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The Food and Drug Administration (FDA) approved Voltaren Arthritis Pain (diclofenac sodium topical gel, 1%) for nonprescription, over-the-counter (OTC) use to treat osteoarthritis (OA)-associated pain. The medication was initially available through prescription; the change is possible through the FDAs prescription (Rx)-to-OTC switch process.

As a result of the Rx-to-OTC switch process, many products sold over-the-counter today use ingredients or dosage strengths that were available only by prescription 30 years ago, said Karen Mahoney, MD, acting deputy director of the Office of Nonprescription Drugs in the FDAs Center for Drug Evaluation and Research, in an FDA press release. Approval of a wider range of nonprescription drugs has the potential to improve public health by increasing the types of drugs consumers can access and use that would otherwise only be available by prescription. This includes providing the millions of people that suffer with joint pain from arthritis daily over-the-counter access to another non-opioid treatment option.

Voltaren Arthritis Pain, formerly known as Voltaren Gel 1%, received initial FDA approval in 2007. It is not intended for immediate relief; patients should allow up to seven days to feel its effects. If patients feel no change after seven days or are still using the product after 21 days, it is recommended that they terminate use and seek medical attention.

Voltaren Arthritis Pain is a non-steroidal anti-inflammatory drug (NSAIDs). A study published in The BMJ linked NSAIDs to increased heart failure risk, with seven posing the most significant risk; one of these NSAIDs was diclofenac.

The Voltaren website describes the drug, Diclofenac sodium is a nonsteroidal anti-inflammatory drug (NSAID) that is often used to treat arthritis pain. It falls in the same class (NSAID) as drugs such as ibuprofen (Advil) and naproxen (Aleve). Diclofenac works by temporarily blocking the production of pain signaling chemicals called prostaglandins.

Unlike Voltaren Arthritis Pain, Biofreeze pain relief gel and Salonpas deep relieving gel are not NSAIDs; they are both considered topical rubefacients. They are not prescribed specifically for OA-related pain. Salonpas is sold OTC, and Biofreeze is available OTC and by prescription.

According to drugs.com, there are no known drug interactions associated with Biofreeze. Eleven drugs are associated with minor interactions in Salonpas. An estimated 111 drugs are known to interact with Voltaren Arthritis Pain Gel: seven major, and 104 moderate.

There are no known disease interactions associated with Biofreeze or Salonpas. Voltaren Arthritis Pain is reportedly known to interact with asthma, renal dysfunction, heart failure, hypertension, and platelet aggregation inhibition.

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Voltaren Arthritis Pain Receives FDA Approval for Over-the-counter Sale: How Does It Compare to Other Topical Treatments? - DocWire News

RICHMOND, Va. -- Virginia lawmakers continue to fine tune legislation that aligns with the states growing medical cannabis program by advancing two Senate bills facilitating the work of caregivers and lab employees.

SB 185 sponsored by Sen. Siobhan Dunnavant, R-Henrico, would allow employees at nursing homes, assisted living facilities and hospices to administer CBD and THC-A oil to residents who have a valid written certification to use the medication. SB 885 from Sen. David W. Marsden, D-Fairfax, would remove criminal liabilities for analytical lab workers who transport and possess both substances during the course of their work.

Marsden also introduced legislation to protect individuals from possession charges for having marijuana in the form of cannabidiol oil or THC-A oil, if they have valid written certification from a practitioner.

In 2019, Dunnavant and Marsden helped pass legislation signed by the governor to reduce restrictions for patient access to the substances (SB 1557 , SB 1719]).

CBD products are used to treat epilepsy and to help with pain management for a variety of ailments. The product can be extracted from hemp, a plant in the cannabis family that is typically low in THC. The non psychoactive version of THC is THC-A; it does not produce a high. THC-A has been used to treat seizures, arthritis and chronic pain. Fibers of the hemp plant are also used in making rope, clothing, paper and other products. Hemp recently became legal [vox.com] at the federal level, and its cultivation is still regulated].

There is a distinction between hemp-derived CBD oil and marijuana-derived CBD oil, namely the level of THC present.

Dunnavant told a Senate panel that the bill is needed so that staff at assisted living facilities can be included as those authorized to store and administer both CBD and THC-A to residents and patients. Registered nurses and licensed practice nurses can legally administer the oils. Last year lawmakers passed legislation protecting school nurses from prosecution for possessing or distributing such oils, in accordance with school board policy.

Several nursing homes and assisted living facilities when contacted said that currently the use of CBD or THC-A are not allowed at their locations and that there are no immediate plans to incorporate such use into the care of their residents or patients.

Marsden sees his bill as an opportunity for further research and development of medical marijuana in Virginia. The state pharmaceutical processors permitted to manufacture and dispense marijuana-derived medications can distribute products with doses that do not exceed 10 milligrams of THC.

If a laboratory is going to handle a drug that is marijuana, they need immunity from prosecution. Marsden said. Even if we go into decriminalization, that still has some civil penalties for it.

Richmonder Brion Scott Turner is glad that steps are being made towards CBD becoming more available. Turner uses CBD to help with his own medical condition.

I use a CBD infused lotion for my psoriasis, Turner said. It gives me relief from the itching and the psoriatic arthritis that comes with it.

Turner has said that most of his friends and family use CBD to help with a variety of ailments from minor headaches to anxiety attacks.

My mother uses CBD for anything from lower back pain, helping with an upset stomach or even migraines, Turner said.

Other cannabis related bills moving through the General Assembly include HB 972 , which would decriminalize simple possession of marijuana down to a civil penalty of no more than $25. The Senate version [lis.virginia.gov] of the bill carries a civil penalty of no more than $50.

HJ 130, currently in the Senate Committee of Rules, would direct the Joint Legislative Audit and Review Commission to study options for the regulation of recreational adult use and medical use of cannabis. SJ 67 , which has passed the House and Senate, directs JLARC to study options and make recommendations for how Virginia should go about the growth, sale and possession of marijuana. JLARCs recommendations are due by July 1, 2022.

Both Dunnavant and Marsdens bills reported out of committee and are headed to the House floor.

By Chip Lauterbach/Capital News Service

Capital News Service is a flagship program of VCUs Robertson School of Media and Culture. Students participating in the program provide state government coverage for Virginias community newspapers and other media outlets, under the supervision of Associate Professor Jeff South.

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Nursing homes can't administer medical cannabis. This bill would change that. - wtvr.com

A SERVICE for people with arthritis, hip and knee conditions, joint and muscle injuries and spinal pain is proving to be a success with around 5,000 patients using it last year.

The main aim of the Integrated Musculoskeletal Service is to ensure that patients are seen by the right person first time. The service is involving and supporting patients to make the right decision, for themselves, about their treatment - for example by highlighting alternative treatments to hip and knee surgery.

The aim of the service is to get it right first time for our patients and to support them to choose the right treatment for their condition, said James Geary, extended scope physiotherapist. Weve had some great feedback from our patients which is positive.

The service is provided by the University Hospitals of Morecambe Bay NHS Foundation Trust which runs Furness General.

Patient feedback has included:

Considerate and quick. Someone who listened well and sorted out the problem. I wanted a good explanation about what was happening with my body and I got it. Thank you.

The team has expanded its specialist clinics for those with musculoskeletal problems across the following sites: Westmorland General Hospital, Ulverston Community Health Centre, Grange Health Centre, Heysham Primary Care Centre, Alfred Barrow Health Centre, Furness General Hospital, Royal Lancaster Infirmary, Millom Health Centre and Queen Victoria Hospital in Morecambe.

This new way of working is another example of Bay Health and Care Partners working together more effectively to provide better care in the community, which will keep people across Morecambe Bay healthier and at home for longer without having to come into hospital.

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Arthritis, muscle and spinal service success in Barrow - NW Evening Mail

The toll-like receptor 4 (TLR4) pathway is not likely arelevant target in treating patients with rheumatoid arthritis (RA) whodemonstrate an inadequate response to methotrexate therapy, according toresearch published in the Annals of the Rheumatic Diseases.

Researchers conducted a phase 2, proof-of-concept, randomized, placebo-controlled, double-blind, international, multicenter study of patients with moderate to severe anticitrullinated protein antibody-positive RA who previously demonstrated an inadequate response to methotrexate therapy. The study included adults with active RA 6 months who fulfilled the 2010 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) criteria.

In total, 250 patients were screened for eligibility and 90were randomly assigned to receive either placebo (n=29) or 5 mg/kg NI-0101 afirst-in-class humanized monoclonal antibody-blocking TLR4 every 2 weeks for12 weeks (n=61). All participants also continued methotrexate therapy. Fromeach group, 57 and 29 patients (from treatment and control group, respectively)completed both the 12-week visit and the follow-up phase through week 24.

Overall, no major differences were noted between groups interms of individual disease parameters, although patients in the NI-0101 grouphad a longer RA duration (8.5 vs 5.4 years) and were younger at the time ofdiagnosis (45.7 vs 51.2 years). The mean C-reactive protein (CRP) level wasalso higher in this group at baseline (18.3 mg/L vs 13.4 mg/L).

Both treatment groups demonstrated similar decreases frombaseline to week 12 disease activity scores in 28 joints with CRP, with nosignificant between-group differences noted. Both Clinical Disease ActivityIndex and Simplified Disease Activity Index scores decreased approximately 40%from baseline to 12 weeks, and the proportion of patients who achieved good ormoderate EULAR responses increased with treatment.

By week 12, 27.6% and 26% of patients in both groups(placebo and NI-0101, respectively) achieved good EULAR responses whereas 55.2%and 53.6%, respectively, achieved EULAR moderate responses. Similarly, nosignificant between-group differences were noted in ACR responses at week 12,with 55.2% in the placebo group and 58.9% in the treatment group achieving improvement of 20% in ACRcriteria responses, 20.7% and 14.3% achieving improvement of 50% in ACR criteria (ACR50)responses, and 10.3% and 10.7% achieving improvement of 70% in ACR criteria responses. Asubgroup analysis found no significant effects on stratification by either CRPor FcRIIa genotype for either disease activity score in 28 joints with CRP orACR50 response.

A pharmacokinetics profile of NI-0101 showed expectedconcentrations and elimination consistent with simulations. NI-0101concentrations were maintained above the targeted threshold of 10,000 ng/mL ina majority of patients. In terms of pharmacodynamics, no significantdifferences between treatment groups were noted for all evaluated biomarkers.

NI-0101 infusions every 2 weeks demonstrated an acceptablesafety and tolerability profile in patients with RA, with similar proportionsof treatment-emergent adverse events occurring in both the placebo andtreatment groups (51.7% and 52.5%, respectively). The most frequently reportedadverse events were infections (13.8% in the placebo group and 11.5% in theNI-0101 group).

The lack of significant effect of NI-0101 in thiswell-controlled prospective clinical trial indicates that blocking the TLR4pathway alone is unlikely to benefit patients with established RA, theresearchers concluded. The good NI-0101 safety and [pharmacokinetic]profiles support further exploration in other diseases.

Disclosure: Thisclinical trial was supported by Novimmune SA. Please see the original referencefor a full list of authors disclosures.

Reference

Monnet E, Choy EH, McInnes I, et al. Efficacy and safety of NI-0101, an anti-toll-like receptor 4 monoclonal antibody, in patients with rheumatoid arthritis after inadequate response to methotrexate: aphase II study [published online December 31, 2019]. Ann Rheum Dis. doi: 10.1136/annrheumdis-2019-216487

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TLR4 Blockade May Not Treat RA With Inadequate Response to MTX - Rheumatology Advisor

The U.S. Food and Drug Administration has approved the use of an intravenous form of the pain reliever meloxicam (Anjeso, Baudax Bio) for the relief of moderate to severe pain.

Meloxicam is a nonsteroidal anti-inflammatory drug (NSAID) currently available in tablet form as Mobic by Boehringer Ingelheim. It is approved for the relief of pain associated with osteoarthritis and rheumatoid arthritis in adults, and in children two years and older who have juvenile rheumatoid arthritis.

Now, it can be administered as a once a day, 24-hour intravenous NSAID treatment for patients with moderate to severe pain.

The active ingredient in meloxicam is a long-acting, preferential COX-2 inhibitor with analgesic, anti-inflammatory and antipyretic activities, which inhibits COX-2 thereby reducing prostaglandin biosynthesis.

The approval of Anjeso marks a major advancement in the treatment landscape for managing moderate to severe pain. With our nation currently in the midst of a national opioid epidemic, we are thrilled to be able to offer a novel, non-opioid therapeutic option with the potential to meaningfully impact the acute pain treatment paradigm," said Gerri Henwood, president and chief executive officer of Baudax Bio, in a press statement.

The company cited the success several mid to late stage clinical trials on Anjeso, plus a recent phase three safety trial that showed the treatment was well tolerated as an option to opioids.

While traditional opioid medications have proven effective at providing pain relief, the associated adverse side effects, including sedation and respiratory depression, have driven physicians to employ a multi-modal approach to treating post-operative pain. With 24-hour, durable pain relief and a safety profile comparable to placebo, Anjeso has the potential to serve as a meaningfully differentiated analgesic alternative, said the University of Miami's Keith Candiotti, M.D., in a written statement.

The Anjeso approval is supported by two phase three efficacy studies, one double-blind, placebo-controlled phase three safety study and four phase two clinical studies. A randomized multicenter, double-blind, placebo-controlled phase three study of 379 orthopedic surgery patients showed that patients receiving once-daily intravenous meloxicam 30 mg for seven days required fewer opioids. Total opioid consumption (36.8 mg versus 50.3 mg IV morphine equivalent dose) and opioid consumption from zero to 24 hours, 2448 hours, zero to 48 hours, and zero to 72 hours were statistically significantly lower in the meloxicam treatment group. The side effects were reported as mild to moderate and affected 65 percent of patients.

The most common adverse reactions reported in at least 2 percent of patients treated with Anjeso and at a greater frequency than placebo included: constipation, gamma-glutamyl transferase increased and anemia.

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FDA Approves Opioid Treatment Option - Rheumatology Network

Galapagos(NASDAQ:GLPG) galloped to all-time highs heading into this week. Although a pullback in the overall market has caused the biotech to give up some of its gains, Galapagos is still up close to 20% year to date and up around 150% over the past 12 months.

Some investors could view Galapagos as valued at a steep premium after its big run-up. But is it really too late to buy the high-flying biotech stock?

Image source: Getty Images.

The primary catalyst behind Galapagos' meteoric rise over the last year was its major collaboration deal signed with Gilead Sciences (NASDAQ:GILD) in July last year. Gilead forked over $5.1 billion for the 10-year agreement, with a $3.95 billion upfront payment and a $1.1 billion equity investment in Galapagos.

Gilead and Galapagos were already partnering on immunology drug filgotinib. The new deal gave Gilead the rights to Galapagos' other late-stage pipeline candidate, idiopathic pulmonary fibrosis (IPF) drug GLPG1690. In addition, Gilead can exercise an option to license any of Galapagos' other candidates.

You can attribute Gilead's interest in Galapagos to the tremendous promise for filgotinib. The experimental drug sailed through late-stage clinical studies targeting rheumatoid arthritis with flying colors. Gilead and Galapagos filed for U.S. and European regulatory approvals for filgotinib in treating rheumatoid arthritis in 2019. Approvals are anticipated later this year in the indication.

And that could be just the start. Gilead and Galapagos are also evaluating filgotinib in other late-stage clinical studies in treating Crohn's disease, psoriatic arthritis, and ulcerative colitis. It's also in phase 2 clinical studies targetingankylosing spondylitis and other inflammatory diseases.

Just how successful filgotinib could be if it wins approval remains to be seen. But peak annual sales of close to $3 billion in treating rheumatoid arthritis and another $3 billion in treating other immunology indications could be possible. Filgotinib's safety profile and convenience (it's an oral medication instead of an injection) could boost its commercial success.

That kind of market potential might make Galapagos' current market cap of under $16 billion seem like a steal. However, it's important to remember that the biotech won't rake in all of the money that filgotinib could make.

Galapagos will market filgotinib on its own inBelgium,the NetherlandsandLuxembourg. It willsplit profits generated by filgotinib equally with Gilead in France,Germany,Italy,Spain, and theUnited Kingdom. In other countries, Galapagos stands to receive tiered royalties between 20% and 30%.

Based on AbbVie'sexperience with blockbuster drug Humira prior to it losing exclusivity in Europe, I expect somewhere around two-thirds of filgotinib's sales will be made in the U.S. If we use a peak annual sales estimate of $6 billion, that would give Galapagos a maximum of $1.2 billion from U.S. sales of the drug. Outside of the U.S., my back-of-the-napkin estimate is that Galapagos would make a little under $1 billion annually.

It's more difficult to predict the financial impact for Galapagos' other drugs. Galapagos thinks that the global market for IPF could be $5 billion by 2025. If we assumedGLPG1690 could capture half of that market, Galapagos would probably make around $750 million annually at peak sales due to its licensing deal with Gilead.

My numbers are admittedly very rough. However, I think that peak revenue from filgotinib andGLPG1690 could bring in somewhere in the ballpark of $3 billion for Galapagos in the future. The company's other earlier-stage programs could boost its sales. In addition, Galapagos is eligible to receive some hefty milestone payments from Gilead if all goes well.

Still, though, we're looking at a stock that currently trades at more than five times sales that it might achieve sometime in the future. I like the potential for Galapagos' products. However, I think that there are other biotech stocks with more room to run. My view is that it is a little too late to jump on the Galapagos bandwagon.

Excerpt from:
Is It Too Late to Buy High-Flying Galapagos? - Motley Fool

A new study examined whether season has an impact on symptoms of fatigue, pain, and dryness in primary Sjgrens syndrome.

Seasonality in rheumatic diseases is an issue frequently perceived and voiced by patients. Several studies have identified weather-related flares in rheumatoid arthritis (RA). Weather conditions might also influence pain and function in osteoarthritis, and a seasonal pattern in gout incidence has been described, the researchers wrote.

However, seasonality has not been investigated in primary Sjgrens syndrome (pSS) yet.

The present study evaluated patient data from the French nationwide multicenter pSS cohort Assessment of Systemic Signs and Evolution in Sjgrens Syndrome (ASSESS) (n=395). ASSESS was created in 2006 and houses five-year prospective follow-up data as well as data from three randomized, placebo-controlled trials of infliximab (TRIPSS) (n=103; 22 weeks of follow-up; seven visits), rituximab (TEARS) (n=120; 24 weeks of follow-up; six visits), and hydroxychloroquine (JOQUER) (n=120; 48 weeks of follow-up; four visits). In each study, visits included data collection on visual analog scale (VAS) scores for pain, fatigue, and dryness. In the ASSESS group, objective assessments of dryness were taken at baseline and again at two and five years. Data were assessed by the day, month of the year, and season.

Final analysis included 632 Sjgrens syndrome patients and a total of 2,858 VASs observations spanning the four studies. VASs collected for Sjgrens syndrome patients by season were: spring, 744; summer, 584; fall, 848; and winter, 682. VAS scores for pain were as follows: spring, 52.2; summer, 55.1; fall, 51.0; and winter, 51.7. VAS fatigue scores were 61.9, 62.2, 60.0, and 61.9, respectively; VAS scores for dryness were 58.9, 61.2, 56.9, and 57.9, respectively.

The EULAR Sjgrens Syndrome Patient Reported Index scores did not significantly differ by season: spring, 57.7; summer, 59.5; fall, 55.9; and winter, 57.2.

The results of the study were published in Arthritis Research &Therapy.

The researchers concluded, This first large study on seasonality in [primary Sjgrens syndrome] provides new evidence that fatigue, pain and dryness, as well as the ESSPRI score, do not have meaningful fluctuations according to months or seasons. In [primary Sjgrens syndrome], seasonality does not affect patient-reported outcomes (PROs) on fatigue, pain and dryness.

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Tis the Season? Study Examines Effect of Time of Year on Primary Sjgrens Syndrome Symptoms - DocWire News

Compelling, controversial, and complicated. Thats how Kent Allen, DVM, of Virginia Equine Imaging, in The Plains, describes the equine back. The veterinary community continually debates equine back pain, which he said can be difficult to diagnose with standard approaches and imaging techniques.

Allen knows equine backs well. He has extensive experience treating high-performance horses in his practice and leadership roles at the Olympic, Pan American, and World Equestrian Games. Hes presented diagnostic and treatment methods for many International Society of Equine Locomotor Pathology (ISELP, for which he serves as vice president and executive director) modules around the world and at the American Association of Equine Practitioners convention.

Allen shared his back-pain workup and treatment recommendations with equine veterinarians at the 11th annual Northeast Association of Equine Practitioners (NEAEP) symposium, held Sept. 25-28, 2019, in Saratoga Springs, New York.

First Allen reviewed spinal anatomy, reminding veterinarians about variation in the thoracic and lumbar vertebrae running from the withers to the pelvis. Typically, horses have 18 thoracic (sometimes 19 and rarely 17) and six (occasionally five) lumbar vertebrae. He doesnt use markers on back radiographs to identify specific vertebrae, opting rather to look for the anticlinalthe vertebra at which the spine orientation changeswhich is usually the 15th thoracic vertebrae (T15, though occasionally T14).

T12 is usually the base of the withers, he explained, and the anticlinal is usually T15. Veterinarians can identify the thoracolumbar junction (between the thoracic and lumbar vertebrae) by looking at the change in rib curvature and the transverse processes, which are the protrusions from the sides of the vertebrae. Other helpful anatomical landmarks include the tuber coxaethe point of the hipwhich blocks veterinarians radiographic view of L4-6 (sometimes 5-6, he said). Thats also where articular processes begin to appear more upright.He pointed out the fused mammillary processes, where horses get arthritic change, off the articular processes and in the facet jointwhere the vertebral bodies come together.

We want to understand (the anatomy) before we start trying to diagnose it and treat it, he said, emphasizing that vets also must thoroughly understand the epaxial musculature, the multifidus muscle, longissimus muscle, intraspinous ligament (in which you can see some bony changes that arent usually associated with pathologydisease or damage), and the supraspinous ligament (which can be injured but usually a relatively minor issue thats easy to treat).

The multifidus, which attaches on the articular areas right beside the mammillary processes and reaches forward two or three vertebrae, is crucial because it is responsible for balancing and tensioning that back, he said. And, so, the more you fatigue or have pathology in the back and the more you get multifidus loss of the musculature, the more pressure and weight you put on the longissimus and the more the entire system fails.

Typical back problems Allen and his colleagues see in their practice include:

Bucking is common in horses with back pain, Allen said. Vets should ask themselves, Is it lame? Does the lameness have anything to do with the back (which is rare)? Usually its a decreased performance complaint.

Allen performs a detailed clinical exam on each horse fitting this description; he palpates the back and mobilizes its structures in a dynamic exam, watching the horse walk and jog. He emphasized that horses with back pain come in all shapes and sizes, and some might be very stoic and not show signs of problems until theyre performing at a very high level, sometimes even very late in their athletic careers.

These horses can jump, they can do their jobs, said Allen, citing an upper-level equine athlete he treatedone with significant overriding dorsal spinous processes that had gone undetected until the horse developed severe back pain late in his eventing career. Once we figured out his problem and gave him some pain relief, he looked a lot better doing his job.

Other horses are more demonstrative and will take your kneecap off if you press around on their backs, he said.

For horses not about to kick you in the kneecap, said Allen, he uses a surcingle weighted with 60 pounds to evaluate pain level while the horse is longed in a round pen. I get to see (the horse) move without the weighted surcingle and with the weighted surcingle without the riders influence, he said.

In videos he showed how a horse wearing the weighted surcingle stiffened, accentuating his lameness.

We use this a lot; we dont go through a day without putting this on several horses, he said.

Once Allen and his colleagues have determined without a doubt the horse is experiencing back pain, he turns to imaging, beginning with lateral radiographic views of the spine. He also uses nuclear scintigraphy (bone scans). But he cautioned that any imaging can reveal irrelevant dorsal articular remodeling. In other words, he and other veterinarians see damage in many backs that never creates pain.

In fact, several good studies show there are a lot of orthopedic back problems in horses that show no pain in the back, he said. And so youre left wondering if these horses have pathology and theyre not back sore, then what the heck are you doing X raying them and bone scanning them if theres no correlation between the imaging and the clinical? And the answer is, its really hard to figure it out.

You have got to tie the clinical to the imaging, he added, telling veterinarians to first ask themselves in back pain cases how the injury happened. (Researchers) proved years ago that kissing spines without a doubt is developmental, and it worsens with time. Now, no one has really proven one way or another about the facet arthritis, but I suspect its the exact same thing.

Whether a horse experiences and shows pain from the pathology depends on what the horse is asked to do, he said. For example, in one study veterinarians found severe bone pathology in a group of racehorses euthanized for reasons besides back pain. But, had the horses all become jumpers, many might have exhibited pain they didnt show as racehorses, he reasoned.

The question is, does the horse have the developmental abnormalities, which most of the time were never going to know, is it old enough to have this problem, does it have a job that is going to exacerbate the problem? he said. And, then, you as clinicians can start putting the picture together.

Additionally, Allen said he and his colleagues commonly see horses that are both lame and back sore, generally for separate problems requiring treatment.

Once Allen has confirmed the pathology in the back is, in fact, the cause of the pain, he applies the following treatments:

Secondary treatments include light ridden or in-hand work for short periods. In fact, Allen said its key to avoid long rest periods, opting instead to avoid muscle wasting by returning horses to work.

Extracorporeal shock wave therapy is a big part of his treatment approach. What were really doing with it is quite different from what were doing with treating a proximal suspensory (ligament injury in the leg), he said. There, were trying to stimulate all kinds of healing pathways . Here (with backs) what were trying to do is downregulate pain receptors normal bone has very few pain receptors in it, but arthritic bone has a lot.

We tend to use 1,000 to 2,000 pulses, depending on whether we know where the problem is, and typically we repeat it at five- to six-month intervals, but its variable depending on the patient, he added. Weve been very successful with managing these upper-level athletes with ESWT.

Allen and his colleagues commonly use ESWT and mesotherapy synergistically if the back injury is soft-tissue-related only.

As for other treatment approaches, Allen referenced a study Denoix et al. conducted with the bisphosphonate tiludronate disodium (Tildren). It maybe wasnt the greatest in statistical significance, but 80% of the horses treated with Tildren showed improvement by Day 60, compared with 50% of the control group, he said. So definitely on arthritis in that area, this is a study that you can point at and say, Well, theres a rationale for giving these horses Tildren.

Allen said while several studies support acupuncture for treating backs, this approach as well as chiropractic dont last very long. They last about three weeks, he said. We need something that lasts months and months, or longer.

In an unpublished study Allen and his colleagues looked at 314 horses presented for back pain. Of those, 57 were also presented for lameness. When veterinarians examined the horses, they considered only 32% as lame, but 92% as positive to back palpation and 90% as having a positive response to the weighted surcingle. Some other notes he made:

I dont understand why we want to rush to do surgery because you can manage these horses very effectively with medical therapy, he said. Medical management of back pain and pathology is very successful at keeping these horses going and going at their level of work, not dropping down and doing something lesser.

Theres one exception to that the horses that have already presented with avoidance behavior, he added. And if that avoidance behavior includes bucking the riders off now you have a physical problem and a mental problem. Once that horse has decided that his default response to back pain is bucking the rider off, you can treat that horse and you can be very successful. But what you dont know is at what moment four months from now thats all going to wear off and that horse is going to turn into a horse trying to be the star of the Calgary Stampede.

His biggest take-home message? Veterinarians must see horses with back pain in the field, diagnose them, and start medical management early. If you wait till they start bucking, the prognosis plummets, he said.

The (combination of) technology and the education is there to do it now, he added. It wasnt 15 years ago, but it is now. And, so, you can X ray the dorsal spinous processes, you can have (horses) sent to a clinic and you can X ray the entire spine and know what youre looking at, you can do bone scans, and you can learn to ultrasound these articular processes and the osteoarthritis. These horses dont have to keep going, and you have to educate your riders that when this horse starts getting back sore, weve got to see it and weve got to figure it out.

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Equine Back Pain: Dr. Kent Allen's Bag of Diagnostic and Treatment Tricks - TheHorse.com

Diabetes is characterized by trouble producing or managing insulin, and one emerging treatment involves converting stem cells into beta cells that secrete the hormone. Now, scientists have developed a more efficient method of doing just that, and found that implanting these cells in diabetic mice functionally cured them of the disease.

The study builds on past research by the same team, led by Jeffrey Millman at Washington University. The researchers have previously shown that infusing mice with these cells works to treat diabetes, but the new work has had even more impressive results.

These mice had very severe diabetes with blood sugar readings of more than 500 milligrams per deciliter of blood levels that could be fatal for a person and when we gave the mice the insulin-secreting cells, within two weeks their blood glucose levels had returned to normal and stayed that way for many months, says Millman.

Insulin is normally produced by beta cells in the pancreas, but in people with diabetes these cells dont produce enough of the hormone. The condition is usually managed by directly injecting insulin into the bloodstream when its needed. But in recent years, researchers have found ways to convert human stem cells into beta cells, which can pick up the slack and produce more insulin.

In the new study, the team improved that technique. Usually when converting stem cells into a specific type of cell, a few random mistakes are made, so some other types of cells end up in the mix. These are harmless, but arent exactly pulling their weight for the job at hand.

The more off-target cells you get, the less therapeutically relevant cells you have, says Millman. You need about a billion beta cells to cure a person of diabetes. But if a quarter of the cells you make are actually liver cells or other pancreas cells, instead of needing a billion cells, youll need 1.25 billion cells. It makes curing the disease 25 percent more difficult.

So, the new method was focused on reducing those unwanted extras. By targeting the cytoskeleton, the underlying structure that gives cells their shape, the team was able to not only produce a higher percentage of beta cells, but they also functioned better.

Its a completely different approach, fundamentally different in the way we go about it, said Millman. Previously, we would identify various proteins and factors and sprinkle them on the cells to see what would happen. As we have better understood the signals, weve been able to make that process less random.

When these new-and-improved beta cells were infused into diabetic mice, their blood sugar levels were stabilized, rendering the diabetes functionally cured for up to nine months.

Of course, at this stage its just an animal trial, so the results may not translate to humans any time soon, if ever. But the researchers plan to continue the work by testing the cells in larger animals over longer periods, with hopes of one day getting the treatment ready for human clinical trials.

The research was published in the journal Nature Biotechnology.

Source: Washington University

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Mice with diabetes "functionally cured" using new stem cell therapy - New Atlas

The study builds on past research by the same team, led by Jeffrey Millman at Washington University. The researchers have previously shown that infusing mice with these cells works to treat diabetes, but the new work has had even more impressive results.

These mice had very severe diabetes with blood sugar readings of more than 500 milligrams per deciliter of blood levels that could be fatal for a person and when we gave the mice the insulin-secreting cells, within two weeks their blood glucose levels had returned to normal and stayed that way for many months, says Millman.

Insulin is normally produced by beta cells in the pancreas, but in people with diabetes these cells dont produce enough of the hormone. The condition is usually managed by directly injecting insulin into the bloodstream when its needed. But in recent years, researchers have found ways to convert human stem cells into beta cells, which can pick up the slack and produce more insulin.

In the new study, the team improved that technique. Usually when converting stem cells into a specific type of cell, a few random mistakes are made, so some other types of cells end up in the mix. These are harmless, but arent exactly pulling their weight for the job at hand.

The more off-target cells you get, the less therapeutically relevant cells you have, says Millman. You need about a billion beta cells to cure a person of diabetes. But if a quarter of the cells you make are actually liver cells or other pancreas cells, instead of needing a billion cells, youll need 1.25 billion cells. It makes curing the disease 25 percent more difficult.

So, the new method was focused on reducing those unwanted extras. By targeting the cytoskeleton, the underlying structure that gives cells their shape, the team was able to not only produce a higher percentage of beta cells, but they also functioned better.

Its a completely different approach, fundamentally different in the way we go about it, said Millman. Previously, we would identify various proteins and factors and sprinkle them on the cells to see what would happen.

As we have better understood the signals, weve been able to make that process less random.

When these new-and-improved beta cells were infused into diabetic mice, their blood sugar levels were stabilized, rendering the diabetes functionally cured for up to nine months.

Of course, at this stage its just an animal trial, so the results may not translate to humans any time soon, if ever. But the researchers plan to continue the work by testing the cells in larger animals over longer periods, with hopes of one day getting the treatment ready for human clinical trials.

The research was published in the journal Nature Biotechnology.

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Mice with diabetes functionally cured using new stem cell therapy - Daily Times

The latest version of the 2020 market study on Cancer Stem Cell Therapy Market comprising 97 with market data Tables, Charts, Graphs, and Figures which are easy to understand with showcased in-depth analysis.

The global Cancer Stem Cell Therapy market size is estimated at xxx million USD with a CAGR xx% from 2015-2019 and is expected to reach xxx Million USD in 2020 with a CAGR xx% from 2020 to 2025. The report begins from overview of Industry Chain structure, and describes industry environment, then analyses market size and forecast of Cancer Stem Cell Therapy by product, region and application, in addition, this report introduces market competition situation among the vendors and company profile, besides, market price analysis and value chain features are covered in this report.

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As per the research and study, the market has settled its presence worldwide. Cancer Stem Cell Therapy Market Research study offers a comprehensive evaluation of the Market and comprises a future trend, current growth factors, focused opinions, details, and industry certified market data.

Glancing to 2020, the global market expected to be a significant year for Cancer Stem Cell Therapy Market in terms of growth and revenue.

Almost all companies who are listed or profiled are being to upgrade their applications for end-user experience and setting up their permanent base in 2020. This report focused and concentrate on these companies including AVIVA BioSciences, AdnaGen, Advanced Cell Diagnostics, Silicon Biosystems.

Furthermore, the research contributes an in-depth overview of regional level break-up categorized as likely leading growth rate territory, countries with the highest market share in past and current scenario. Some of the geographical break-up incorporated in the study are AVIVA BioSciences, AdnaGen, Advanced Cell Diagnostics, Silicon Biosystems.

With the Cancer Stem Cell Therapy market forecast to expand CAGR% in 2020 and with X-X-X-X supposed to be a big beneficiary, it is better positioned than Z-Z-Z-Z for 2020.

According to the AMR market study, Recent trends in consumer preferences market segments such as type, application will be more challenging. Cancer Stem Cell Therapy market segment sales will traverse the $$ mark in 2020.

Unlike classified segments successful in the industry such as by Type (Autologous Stem Cell Transplants, Allogeneic Stem Cell Transplants, Syngeneic Stem Cell Transplants, Others) and by End-Users/Application (Hospital, Clinic, Medical Research Institution, Others).

The 2020 version of the Cancer Stem Cell Therapy market study is a further split down / narrowed to highlight the latest emerging twist of the industry.

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With using the latest technology and analysis on demand-side, Key players are getting in consumer behavior and their changing preferences.

Again, big investment firms or giants are willing to put more capital to get a key players performance in the market for new applications or products.

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Global Cancer Stem Cell Therapy Market 2020: What Are The Key Trends In Market? - Chronicles 99

R3 Stem Cell Heroes Program - Free Regenerative Procedures for Veterans

LAS VEGAS (PRWEB) February 20, 2020

R3 Stem Cell, the nation's leader in regenerative therapies and provider training, announced that its Heroes Program was able to complete 70 complimentary stem cell procedures on military veterans in 2019. The veterans traveled from all over the US to receive the regenerative therapies at no cost.

At the R3Stem Cell Training Course for doctors, providers of all kinds learn how to perform regenerative procedures in a supervised setting. This includes stem cell and exosome procedures on military veterans of all ages and from all branches of service. Any veteran can be nominated (or nominate themselves), and the pain being experienced does not have to be a result of a service related injury.

According to R3 Stem Cell CEO David Greene, MD, MBA, "When we hear the stories of these Veterans and what they have done for America, it's so exciting to be able to do something to help improve their quality of life. Especially Veterans of the Vietnam War, all they wanted to do was protect our country. To see how well they do afterwards and then refer in their Veteran friends for procedures, it's amazing!"

The chosen Veterans receive an evaluation by the Board Certified doctors who are trainers, and then procedures include whatever amount of regenerative biologic deemed appropriate. The Veteran patients are followed up the R3 staff, and invited back for additional therapies when necessary.

To date over 70 Veterans have successfully receive regenerative therapies in the program. Over a dozen patients are treated at each workshop, with more opportunities upcoming. Added Dr. Greene, "Our stem cell courses are extremely hands on an providers love it. They have been increasing in popularity continuously, which allows us to treat more Veterans!"

For those who would like to nominate a Veteran for the Heroes Program to receive free stem cell therapy, simply visit https://r3stemcell.com/hero or call (844) GET-STEM.

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R3 Stem Cell Heroes Program Performs Complimentary Regenerative Therapies on 70 Veterans in 2019 - PR Web

Although radiation is successfully used to treat breast cancer, inflammation caused as a side-effect of radiation may have an adverse effect, promoting the survival of triple-negative breast cancer cells.

This is the conclusion of a study by Jennifer Sims-Mourtada, Ph.D., director of Translational Breast Cancer Research at ChristianaCares Helen F. Graham Cancer Center & Research Institute, published online in the International Journal of Radiation Biology.[1]

Triple-negative breast cancer is cancer that tests negative for estrogen receptors, progesterone receptors, and excess human epidermal growth factor receptor 2 (HER2) protein. As a result, the growth of this cancer is not fueled by the hormones estrogen and progesterone, or by the HER2 protein. These cancers tend to be more common in women under age 40, who are African-American, Latina, or who carry a mutated BRCA1 gene.

Triple-negative breast cancer accounts for 15-20% of all breast cancers and is faster growing than other types of breast cancers.

Sims-Mourtadas latest study helps scientists to better understand the mechanisms behind the development of this aggressive and hard-to-treat cancer. It shows that inflammation caused by radiation can trigger stem-cell-like characteristics in non-stem breast cancer cells.[1]

The good and the badThis is the good and the bad of radiation, Sims-Mourtada noted. We know radiation-induced inflammation can help the immune system to kill tumor cells thats good but also it can protect cancer stem cells in some cases, and thats bad.

Whats exciting about these findings is were learning more and more that the environment the tumor is in its microenvironment is very important. Historically, research has focused on the genetic defects in the tumor cells. Were now also looking at the larger microenvironment and its contribution to cancer, she added.

My work focuses on cancer stem cells and their origination. [These cells] exist in many cancers, but theyre particularly elusive in triple-negative breast cancer. Their abnormal growth capacity and survival mechanisms make them resistant to radiation and chemotherapy and help drive tumor growth, Sims-Mourtada explained.

The researchers applied radiation to triple-negative breast cancer stem cells and to non-stem cells. In both cases, they found radiation-induced an inflammatory response that activated the Il-6/Stat3 pathway, which plays a significant role in the growth and survival of cancer stem cells in triple-negative breast cancers. They also found that inhibiting STAT3 blocks the creation of cancer stem cells. As yet unclear is the role IL-6/STAT3 plays in transforming a non-stem cell to a stem-cell.

DelawareFor women living in Delaware, Sims-Mourtadas research is especially urgent: The rates of triple-negative breast cancer in the state are the highest nationwide.

At ChristianaCare, we are advancing cancer research to help people in our community today, while we also advance the fight against cancer nationwide, said Nicholas J. Petrelli, M.D., Bank of America endowed medical director of the Helen F. Graham Cancer Center & Research Institute.

Sims-Mourtadas research is a dramatic step toward better treatments for triple-negative breast cancer, Petrelli concluded.

To advance her research on inflammation, last year Sims-Mourtada received a US $ 659,538 grant from the Lisa Dean Moseley Foundation. The three-year grant will enable her and her team at the Cawley Center for Translational Cancer Research to continue investigating the role of cells immediately around a tumor in spurring the growth of triple-negative breast cancer and possible therapy for this particularly difficult to treat cancer.

Our next step is to understand the inflammatory response and how we might inhibit it to keep new cancer stem cells from developing, Sims-Mourtada said.

The researchers previously identified an anti-inflammatory drug, currently used to treat rheumatoid arthritis, that has the potential to target and inhibit the growth of cancer stem cells and triple-negative breast cancer tumors. That research could set the stage for clinical investigation of the drug, alone or in combination with chemotherapy, to improve outcomes for patients with triple-negative breast cancer.

Reference[1] Arnold KM, Opdenaker LM, Flynn NJ, Appeah DK, Sims-Mourtada J. Radiation induces an inflammatory response that results in STAT3-dependent changes in cellular plasticity and radioresistance of breast cancer stem-like cells [published online ahead of print, 2020 Jan 6]. Int J Radiat Biol. 2020;114. doi:10.1080/09553002.2020.1705423 [Pubmed][Article]

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Radiation, Inflammation and Triple-Negative Breast Cancer: A Study - OncoZine

The most popular stories of the week onAJMC.

The journalOncogenerecently publishedresultsof an investigation into the drivers behind triple-negativebreast cancer. Because the disease has such high recurrence and low survival rates, investigators from Manchester, Glasgow, and Sheffield universities sought to better understand what triggers, and arrests, the growth of this subtype of breast cancer.

Stem Cells Prove Effective in Treating MS Neural Damage

Several clinical trials have found mesenchymal stem cell therapy effective in treating neural damage in patients with multiple sclerosis (MS), according to a review published inStem Cell Investigation.

Youth Newly Diagnosed With HIV Have Advanced Infection, Higher Viral Loads

Close to 80% of HIV-positive individuals are shown to be virally suppressed through their most recent test results, according to data from 2016 through 2018, as well as 32% to 63% of adults older than 24 years. Youth with a new HIV diagnosis, however, come in at only 12%.

DC Circuit Panel Rejects Arkansas Medicaid Work Rules

Writing for the panel, Judge David Sentelle said HHS Secretary Alex Azar ignored predictions that thousands of people would lose their healthcare coverage.

Much of MS Cost Burden Falls on Patients' Shoulders

A British study shows nonmedical costs make up a significant portion of the annual expense generated by MS. Most of those nonmedical costs are not covered by insurance.

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Top 5 AJMC Stories of the Week: February 22, 2020 - AJMC.com Managed Markets Network

Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, today announced that the company is scheduled to participate in a fireside chat at the 40th annual Cowen Health Care Conference on Monday, March 2nd, 2020, at 4:10 p.m. ET at the Boston Marriott Copley Place.

A live webcast of the fireside chat can be accessed on the Magenta Therapeutics website at https://investor.magentatx.com/events-and-presentations. The webcast replay will be available for 90 days following the event.

About Magenta TherapeuticsMagenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking StatementThis press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "will," "could", "should," "expects," "intends," "plans," "anticipates," "believes," "estimates," "predicts," "projects," "seeks," "endeavor," "potential," "continue" or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption "Risk Factors" in Magentas Registration Statement on Form S-1, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200226005256/en/

Contacts

Manisha Pai, Vice President, Communications & Investor Relations617-510-9193mpai@magentatx.com

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Magenta Therapeutics to Participate in Cowen Health Care Conference on Monday, March 2nd in Boston - Yahoo Finance

The market expects Fate Therapeutics (FATE) to deliver a year-over-year decline in earnings on higher revenues when it reports results for the quarter ended December 2019. This widely-known consensus outlook is important in assessing the company's earnings picture, but a powerful factor that might influence its near-term stock price is how the actual results compare to these estimates.

The stock might move higher if these key numbers top expectations in the upcoming earnings report. On the other hand, if they miss, the stock may move lower.

While management's discussion of business conditions on the earnings call will mostly determine the sustainability of the immediate price change and future earnings expectations, it's worth having a handicapping insight into the odds of a positive EPS surprise.

Zacks Consensus Estimate

This clinical-stage biotech company that develops stem cell treatments is expected to post quarterly loss of $0.39 per share in its upcoming report, which represents a year-over-year change of -56%.

Revenues are expected to be $1.84 million, up 10.8% from the year-ago quarter.

Estimate Revisions Trend

The consensus EPS estimate for the quarter has remained unchanged over the last 30 days. This is essentially a reflection of how the covering analysts have collectively reassessed their initial estimates over this period.

Investors should keep in mind that an aggregate change may not always reflect the direction of estimate revisions by each of the covering analysts.

Price, Consensus and EPS Surprise

Earnings Whisper

Estimate revisions ahead of a company's earnings release offer clues to the business conditions for the period whose results are coming out. Our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction) -- has this insight at its core.

The Zacks Earnings ESP compares the Most Accurate Estimate to the Zacks Consensus Estimate for the quarter; the Most Accurate Estimate is a more recent version of the Zacks Consensus EPS estimate. The idea here is that analysts revising their estimates right before an earnings release have the latest information, which could potentially be more accurate than what they and others contributing to the consensus had predicted earlier.

Thus, a positive or negative Earnings ESP reading theoretically indicates the likely deviation of the actual earnings from the consensus estimate. However, the model's predictive power is significant for positive ESP readings only.

A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive surprise nearly 70% of the time, and a solid Zacks Rank actually increases the predictive power of Earnings ESP.

Please note that a negative Earnings ESP reading is not indicative of an earnings miss. Our research shows that it is difficult to predict an earnings beat with any degree of confidence for stocks with negative Earnings ESP readings and/or Zacks Rank of 4 (Sell) or 5 (Strong Sell).

How Have the Numbers Shaped Up for Fate Therapeutics?

For Fate Therapeutics, the Most Accurate Estimate is the same as the Zacks Consensus Estimate, suggesting that there are no recent analyst views which differ from what have been considered to derive the consensus estimate. This has resulted in an Earnings ESP of 0%.

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On the other hand, the stock currently carries a Zacks Rank of #3.

So, this combination makes it difficult to conclusively predict that Fate Therapeutics will beat the consensus EPS estimate.

Does Earnings Surprise History Hold Any Clue?

Analysts often consider to what extent a company has been able to match consensus estimates in the past while calculating their estimates for its future earnings. So, it's worth taking a look at the surprise history for gauging its influence on the upcoming number.

For the last reported quarter, it was expected that Fate Therapeutics would post a loss of $0.38 per share when it actually produced a loss of $0.40, delivering a surprise of -5.26%.

The company has not been able to beat consensus EPS estimates in any of the last four quarters.

Bottom Line

An earnings beat or miss may not be the sole basis for a stock moving higher or lower. Many stocks end up losing ground despite an earnings beat due to other factors that disappoint investors. Similarly, unforeseen catalysts help a number of stocks gain despite an earnings miss.

That said, betting on stocks that are expected to beat earnings expectations does increase the odds of success. This is why it's worth checking a company's Earnings ESP and Zacks Rank ahead of its quarterly release. Make sure to utilize our Earnings ESP Filter to uncover the best stocks to buy or sell before they've reported.

Fate Therapeutics doesn't appear a compelling earnings-beat candidate. However, investors should pay attention to other factors too for betting on this stock or staying away from it ahead of its earnings release.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportFate Therapeutics, Inc. (FATE) : Free Stock Analysis ReportTo read this article on Zacks.com click here.

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Analysts Estimate Fate Therapeutics (FATE) to Report a Decline in Earnings: What to Look Out for - Yahoo Finance

With the success of CAR T-cell therapies and other innovative medicines in this area, immuno-oncology has become a buzzword in biotech, with more and more companies starting up in Europe and elsewhere. With so many players involved, it can be hard to know who is doing what. Here are 20 European companies we think stand out and are leading the revolution in cancer medicine.

Although immunotherapies to treat cancer have been around for decades, new approaches are popping up every other day. After all, enlisting the immune system to fight cancer remains an extremely promising therapeutic strategy. There are so many companies focusing on different kinds of immunotherapies that it was quite a challenge to choose our favorites. We therefore enlisted the help of experts in the industry to select the following 20 immuno-oncology companies in Europe as our top picks. As ever, these are in no particular order.

Founded in Abingdon, UK, in 2008, Adaptimmune develops enhanced T-cell therapies that support the immune system in detecting and targeting cancer cells. Interestingly, its SPEAR T cell technology can target a variety of solid tumors a feat that is not easy to achieve, as solid tumors have a highly complex tumor microenvironment that many immunotherapies find difficult to penetrate.

The companys technology can identify and select T cell receptors (TCRs) that are more likely to improve the patients immune response and result in the effective binding of cancer cells. In January 2020, Adaptimmune and Japanese company Astellas agreed to co-develop and co-commercialize stem-cell-derived allogeneic chimeric antigen receptor (CAR)-T and TCR T cell therapies, which means that they not only treat one specific patient but a large number of patients with a particular cancer type. The deal was worth 806M.

ADC Therapeutics works in the area of antibody-drug conjugates (ADCs). Since being founded in 2011 in Lausanne, Switzerland, ADC Therapeutics has raised 514M ($558M) to advance its immuno-oncology therapies. The companys proprietary ADCs combine monoclonal antibodies that are specific to targets on the surface of tumor cells with a specific class of toxins called pyrrolobenzodiazepine (PBD) dimers.

The PBD dimer is released inside the tumor cells and binds to the tumor DNA, which blocks the division of the tumor cells and eventually kills them. ADC Therapeutics currently has two ADCs for liquid tumors in pivotal phase II studies, and one ADC targeting solid tumors in phase Ib. In January 2020, the company announced the positive results of its phase II trial of its leading ADC in patients with relapsed or refractory diffuse large B cell lymphoma.

Yet another direction in the treatment of cancer via the immune system is taken by Heidelberg-based company Affimed Therapeutics. Founded in 2000, the company focuses on the innate immune system. Using its ROCK platform, Affimed develops multi-specific antibodies called cell engagers.

Innate cell engagers can bind to the patients innate immune cells, including natural killer cells and macrophages, while simultaneously binding to specific tumor cells. As a result, the patients own immune systems can attack and kill the cancer cells. The companys lead candidate is currently in phase II for a number of liquid tumors.

Based in London, UK, and founded in 2014 as a spin-out from the University College London, Autolus Therapeutics uses CARs and TCRs to reprogram T cells so they recognize and consequently kill tumor cells. In January 2020, the company revealed the closure of its public offering on the Nasdaq with net proceeds of 68.4M ($74.2M).

Autolus other news in January was the announcement of the clinical progress of its next-generation programmed T cell therapy. It is currently in Phase I/II for the treatment of adults with relapsed or refractory diffuse large B cell lymphoma. The results showed no neurotoxicity and a manageable safety profile.

BioNTech has a much larger pipeline that contains CARs, TCRs, recombinant cytokines, and optimized mRNA. Founded in Mainz, Germany, in 2008, the company is one of the largest biotechs in Europe. Its pipeline is full to bursting with a variety of immuno-oncology therapies based on mRNA, antibodies, and CAR-T cells.

In July 2019, BioNTech raised one of the biggest ever funding rounds of a European biotech with a whopping 290M Series B. However, in October of that same year, the companys Nasdaq IPO, which was initially estimated to raise around 725M, resulted in pricing at only 141M due to poor market conditions in the US.

Based in Paris, France, and founded in 1999, Cellectis is also focusing on CART-cell therapy. It is working on a novel type of CART therapy known as Universal CARTs (UCARTs). Unlike the two approved CAR-T therapies, Novartis Kymriah and Gileads Yescarta, UCARTs are allogeneic so rather than being personalized, they can treat a large number of patients with a particular cancer type.

In November 2019, Cellectis announced that it had won the European patent challenge for the use of CRISPR-Cas9 for gene editing T cells. This process will help the company further develop its UCART platform. To date, six of its main UCART product candidates are in Phase I clinical trials. For three of these programs, Cellectis has partnered with Servier and Allogene.

As another biotech giant, Celyad is also working in the field of CART-cell therapies but is focusing on the NKG2D receptor. Founded in 2004 in Mont-Saint Guibert, Belgium, the companys lead immuno-oncology candidate uses the NKG2D receptor. NKG2D can bind to eight different ligands that are naturally found on cancer cells present in 80% of solid and liquid malignancies.

Something cool about NKG2D is that the ligands that it recognizes are also expressed by the tumor microenvironment, which comprises the blood vessels that support the tumor and the cells that make sure the tumor can evade the immune system. As a result, the lead candidate can target and kill the tumor as well as cleaning up its microenvironment. The candidate is currently in phase I.

Spun-off from the Babraham Institute in Cambridge, UK, in 2007, Crescendo Biologics develops targeted T-cell enhancing therapeutics with the aim of tackling difficult-to-treat cancers. The companys Humabody therapies are small, multi-specific antibody fragments that can penetrate tumor tissue in a way that conventional antibodies cannot.

Crescendo Biologics lead proprietary candidate is a bispecific T-cell engager targeting the prostate-specific membrane antigen. It can selectively activate tumor-specific T cells within the tumor microenvironment. It is currently advancing towards clinical development.

As a spring chicken among other European immuno-oncology companies, we felt Ervaxx deserved a mention here because it develops cancer vaccines and cell therapies targeting dark antigens normally silent genes that are expressed in cancer cells. Founded in 2017 in London, UK, Ervaxx recently made headlines when it got its hands on the license of an exciting new preclinical stage universal cancer immunotherapy.

In early January 2020, a group of researchers at Cardiff University discovered a new type of T cells that have the potential to attack a wide range of cancers, such as leukemia and melanoma. Ervaxx is continuously expanding its repertoire of Dark Antigens. Using these, the company is working on a pipeline of off-the-shelf cancer vaccines and TCR-based therapies.

Founded in 2008 in Copenhagen, Denmark, Evaxion takes a very different approach to fighting cancer: using artificial intelligence (AI) to develop immunotherapies. The company has developed two AI platforms that can identify and optimize epitopes and antigens that are capable of eliciting strong immune responses against cancer and infectious diseases.

The companys PIONEER platform is used for the development of personalized cancer immunotherapies. In April 2019, it announced the beginning of phase I with the dosing of the first patient with its lead candidate, a therapeutic vaccine. Evaxion closed the year by successfully raising 16M ($17M).

GammaDelta Therapeutics, founded in 2016, takes another approach to immuno-oncology therapies. The London-based company focuses on gamma delta T cells. Unlike alpha beta T cells which are commonly used in CAR-T therapy, gamma delta T cells are a part of the innate immune system. In short, this means that gamma delta T cells are already pre-programmed to identify and kill cells that are modified by cancer. The company is currently advancing its allogeneic gamma delta T cell therapies through preclinical development.

In October 2019, GammaDelta Therapeutics announced the formation of the spinout company Adaptate Biotherapeutics. Adaptate will be developing therapeutic antibodies that can modulate the activity of gamma delta T cells with the aim of triggering an immune response to fight cancer.

Danish biotech Genmab had an exciting start to the new year. In January 2020, one of the companys lead candidates daratumumab, a monoclonal antibody, received marketing authorization by the EMA. Marketed as DARZALEX, it can be used in combination with bortezomib, thalidomide, and dexamethasone to treat multiple myeloma.

Founded in Copenhagen in 1999, Genmab focuses on the development of antibody therapies against cancer. In December 2019, Genmab signed a deal with German biotech CureVac to develop mRNA drugs that can produce antibodies within the patient, which is an exciting development.

Heidelberg Pharma, formerly known as Wilex, was founded in 1997 in Heidelberg, Germany. The company focuses on ADCs. It has an exclusive license agreement with the Max Delbrck Center for Molecular Medicine in the Helmholtz Association in Berlin that includes a number of surface proteins found in multiple myeloma cells called B cell maturation antigen (BCMA).

Heidelberg Pharmas lead candidate targets BCMA and is currently advancing through the preclinical stage. It is an ADC that consists of a BCMA antibody, a specific linker, and the toxin amanitin, one of the deadliest toxins found in several mushroom species.

Founded in 2011, Austrian company Hookipa Pharma engineers arenaviruses to deliver tumor-specific genes to dendritic cells. Dendritic cells naturally activate killer T cells by delivering the tumor-specific antigens to them, which triggers an immune response. HOOKIPA Pharmas co-founder, Rolf Zinkernagel, actually received the Nobel Prize for Physiology or Medicine in 1996 for his work on how killer T cells can recognize virus-infected cells.

Hookipas leading immuno-oncology therapy is currently progressing through phase I. In February 2019, the company closed a Series D financing round for 33.2M ($37.4M). The funding is being used to boost the clinical development of its immunotherapies for cancer and infectious diseases based on its proprietary arenavirus platform.

This French company develops antibody therapies to fight cancer. Marseille-based ImCheck has developed a pipeline of monoclonal antibodies that target specific checkpoint molecules of the butyrophilin (BTN) family, which engage gamma delta T cells. As part of the innate immune system, gamma delta T cells fight cancer cells naturally.

ImChecks lead drug candidate, an anti-BTN3 antibody that targets solid tumors, is expected to enter phase I in 2020. In order to fund this trial and further boost the development of immunotherapies, ImCheck closed a series B funding round in December 2019 worth 48M.

Immatics was founded in 2000 in Tbingen, Germany. The company develops personalized immuno-oncology therapies by engineering patient T cells to express TCRs, which specifically target the patients cancer. Unlike CAR-T therapies that generally target surface proteins, Immatics immunotherapy can also target proteins within cancer cells, which makes the cancer cells more vulnerable and accessible to the immune system.

In August 2019, American company Celgene and Immatics signed a deal worth more than 1.35B ($1.5B) to further develop its immuno-oncology therapies. In three cancer immunotherapy programs, Immatics will develop leading cell immunotherapy candidates for solid tumors, while Celgene has the option to co-develop the candidates or take over the development altogether.

Based in Oxfordshire, UK, and founded in 2008, Immunocore also focuses on TCRs. Its ImmTAC (immune mobilizing monoclonal TCRs against cancer) technology is based on a new class of bi-specific biologics that can activate a specific T cell response in cancer cells.

In December 2019, Immunocore announced the start of the first-in-human clinical trial of its third bispecific, which was developed using the ImmTAC technology platform. Immunocore has collaborations with a number of partners, including AstraZeneca, Genentech, GSK, and Eli Lilli.

Medigene also focuses on TCRs. However, this company develops modified TCRs called TCR-Ts, as well as dendritic cell vaccines (DCs), and T cell-specific antibodies (TABs).

In January 2020, Medigene announced positive results after a two-year phase I/II for its DC vaccine in patients with acute myeloid leukemia (AML). Moreover, its leading TCR-T candidate is currently being prepared for phase II clinical trials. Medigene was founded in 1994 in Martinsried, Germany.

As a neighbor of Medigene, MorphoSys is also based in Martinsried, Germany. It was founded in 1992. The company is one of the very few European biotechs valued at over 1B. MorphoSys develops antibody therapies for numerous conditions.

The companys lead immuno-oncology candidate tafasitamab is a humanized monoclonal antibody that targets CD19, an antigen mainly expressed by various B cell-derived blood cancers, such as non-Hodgkins lymphoma, diffuse large B cell lymphoma, and chronic lymphocytic leukemia. This candidate is being co-commercialized by Incyte. Other partners of MorphoSys include Novartis, Roche, GSK, and Roche.

Another company focusing on ADCs is NBE Therapeutics. This companys goal is to target solid tumors. Based in Basel, Switzerland, and founded in 2012, NBE Therapeutics has developed a technology that uses an enzyme to attach small molecule drugs to monoclonal antibodies. This approach is different from conventional ADCs that are usually generated using a chemical junction, which can be unstable at times.

The companys lead candidate, an ADC for the treatment of triple-negative breast cancer, lung, and ovarian cancer, is expected to reach phase I in 2020.

Images via Elena Resko & Shutterstock.com

Originally posted here:
Meet 20 European Immuno-Oncology Companies that are Fighting... - Labiotech.eu

Cells constitute discrete units of biological function and serve as starting points in a myriad of studies to identify and map many of the basic biochemical and physical processes of life. Cell analysis is a wide term that can be applied to a range of different technologies, the technology used depends on experimental needs. Compiling data sets from multiple cell analysis investigations allows scientists to better understand, predict, and ultimately influence the factors that underlie cell health, function, death, and proliferation.

This intelligence report provides a comprehensive analysis of the Global Cell Analysis Market. This includes Investigation of past progress, ongoing market scenarios, and future prospects. Data True to market on the products, strategies and market share of leading companies of this particular market are mentioned. Its a 360-degree overview of the global markets competitive landscape. The report further predicts the size and valuation of the global market during the forecast period. Some of the key players profiled in the study are Thermo Fisher Scientific, Inc. (United States), Danaher Corporation (United States), The Merck KGaA Group (Germany), Becton, Dickinson and Company (United States), GE Healthcare (United States), Agilent Technologies, Inc. (United States), Olympus Corporation (Japan), PerkinElmer, Inc. (United States), Promega Corporation (United States), Sysmex Corporation (Japan) and AbbVie, Inc.(United States) are some of the key players profiled in the study. Additionally, the Players which are also part of the research are Lonza Group (Switzerland), Tecan Group (Switzerland), Miltenyi Biotec (Germany), Stemline Therapeutics, Inc.(United States), Fujifilm Irvine Scientific (United States), STEMCELL Technologies Inc. (Canada), Sino Biological Inc. (United States), BIOTIME, Inc. (United States), Miltenyi Biotec (Germany), PromoCell GmbH (Germany), Carl Zeiss (Germany), Bio-Rad Laboratories Inc (United States), MacroGenics, Inc. (United States), OncoMed Pharmaceuticals, Inc. (United States) and Bionomics (Australia).

Free Sample Report + All Related Graphs & Charts @: https://www.advancemarketanalytics.com/sample-report/124696-global-cell-analysis-market

Market Trend

Market Drivers

Restraints

Opportunities

Each segment and sub-segment is analyzed in the research report. The competitive landscape of the market has been elaborated by studying a number of factors such as the best manufacturers, prices and revenues. Global Cell Analysis Market is accessible to readers in a logical, wise format. Driving and restraining factors are listed in this study report to help you understand the positive and negative aspects in front of your business.

This study mainly helps understand which market segments or Region or Country they should focus in coming years to channelize their efforts and investments to maximize growth and profitability. The report presents the market competitive landscape and a consistent in depth analysis of the major vendor/key players in the market.Furthermore, the years considered for the study are as follows:Historical year 2013-2017Base year 2018Forecast period** 2019 to 2025 [** unless otherwise stated]

**Moreover, it will also include the opportunities available in micro markets for stakeholders to invest, detailed analysis of competitive landscape and product services of key players.The Global Cell Analysis segments and Market Data Break Down are illuminated below:Type (Flow Cytometry Products (Reagents and Consumables, Instruments, Accessories, and Software), QPCR Products (Reagents and Consumables, and Instruments), Cell Microarrays (Consumables, and Instruments), Microscopes (Electron, Inverted, Stereo, and Near-field Scanning Optical), Spectrophotometers (Single-mode Readers, and Multi-mode Readers), Cell Counters (Automated Cell Counters, Hemocytometers, and Manual Cell Counters), HCS Systems, Others)

Application (Cell Culture, Cell Imaging, Cell Isolation and Expansion, Cell Signaling Pathways, Cell Structure, Cell Tracing & Tracking, Cell Function Assays, Stem Cell Research, Others), End Use Verticals (Hospitals and Diagnostic Laboratories, Pharmaceutical & Biotechnology Companies, Research Institutes, Cell Culture Collection Repositories, Others), Process (Cell Identification, Cell Viability, Cell Signaling Pathways/Signal Transduction, Cell Proliferation, Cell Counting and Quality Control, Cell Interaction, Target Identification and Validation, Single-cell Analysis)

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Enquire for customization in Report @ https://www.advancemarketanalytics.com/enquiry-before-buy/124696-global-cell-analysis-marketRegion Included are: North America, Europe, Asia Pacific, Oceania, South America, Middle East & Africa

Country Level Break-Up: United States, Canada, Mexico, Brazil, Argentina, Colombia, Chile, South Africa, Nigeria, Tunisia, Morocco, Germany, United Kingdom (UK), the Netherlands, Spain, Italy, Belgium, Austria, Turkey, Russia, France, Poland, Israel, United Arab Emirates, Qatar, Saudi Arabia, China, Japan, Taiwan, South Korea, Singapore, India, Australia and New Zealand etc.

Objectives of the Study

Read Detailed Index of full Research Study at @ https://www.advancemarketanalytics.com/reports/124696-global-cell-analysis-market

Strategic Points Covered in Table of Content of Global Cell Analysis Market:

Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the Cell Analysis market

Chapter 2: Exclusive Summary the basic information of the Cell Analysis Market.

Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges of the Cell Analysis

Chapter 4: Presenting the Cell Analysis Market Factor Analysis Porters Five Forces, Supply/Value Chain, PESTEL analysis, Market Entropy, Patent/Trademark Analysis.

Chapter 5: Displaying the by Type, End User and Region 2013-2018

Chapter 6: Evaluating the leading manufacturers of the Cell Analysis market which consists of its Competitive Landscape, Peer Group Analysis, BCG Matrix & Company Profile

Chapter 7: To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries in these various regions.

Chapter 8 & 9: Displaying the Appendix, Methodology and Data SourceKey questions answered

Definitively, this report will give you an unmistakable perspective on every single reality of the market without a need to allude to some other research report or an information source. Our report will give all of you the realities about the past, present, and eventual fate of the concerned Market.

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

About Author:

Advance Market Analytics is Global leaders of Market Research Industry provides the quantified B2B research to Fortune 500 companies on high growth emerging opportunities which will impact more than 80% of worldwide companies revenues.

Our Analyst is tracking high growth study with detailed statistical and in-depth analysis of market trends & dynamics that provide a complete overview of the industry. We follow an extensive research methodology coupled with critical insights related industry factors and market forces to generate the best value for our clients. We Provides reliable primary and secondary data sources, our analysts and consultants derive informative and usable data suited for our clients business needs. The research study enable clients to meet varied market objectives a from global footprint expansion to supply chain optimization and from competitor profiling to M&As.

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See the article here:
Cell Analysis Market is Expected to See Growth Rate of 6.96% - Nyse Nasdaq Live

A novel approach to removing salt from water, inspired by mangrove trees, has been revealed by researchers who say the system could offer an unusual approach to clearing up flood water.

Mangroves, like other trees, employ a system of water transport: it is thought evaporation of moisture from their leaves produces a negative pressure in their water-conducting tissues that helps to draw water into their roots and up their trunks.

This transport relies on surface tension, the fact that water molecules like to interact with the walls of the tissues, and that water molecules exert a tug on each other.

Salty water can damage most plants, but mangroves can thrive in salty conditions because they have adaptations including cell membranes that prevent salts from passing through in an uncontrolled manner, as well as cell walls that contain a waxy substance.

The upshot is that a mangrove essentially desalinates the water from its surroundings.

Now, writing in the journal Science Advances, researchers say they have reproduced this process in a synthetic system, using a polymer membrane that filters out salts as the root, a finely porous silica filter as the stem, and leaves based on either a hydrogel-filled membrane or aluminium oxide featuring tiny pores.

In our particular demonstration, through simple evaporation, a huge negative pressure was generated to drive water flow through a semi-permeable, reverse-osmosis membrane, thereby desalinating the water, said Dr Jay Werber and Dr Jongho Lee, co-authors of the study, who carried out the work at Yale University. In the industrial process, large, high-pressure pumps and loads of electricity are required to generate these high pressures to drive flow and desalination.

Importantly, they note, their system works without the production of air-bubbles which can block the flow thanks to the use of the membrane and tiny pores in the silica stem.

The team add that replicating the natural process adds support to the theory of how water transport in mangrove trees occurs.

However, they note that the system is not a practical way to desalinate water, not least since a large amount of heat would be needed.

The energy that drives desalination in our device is absorbed from the environment: basically, heat is absorbed to drive evaporation, said Werber and Lee. In a small-scale device, this energy is essentially free, meaning that it isnt provided as electricity or generated heat, similar to how drying clothes on a clothes line doesnt take added energy. However, trying to scale this up to large volumes would be very challenging.

But they offer an alternative suggestion: incorporate the system into buildings to turn them into giant sponges, offering a novel way to handle stormwater and reduce flood damage.

In this scenario, the buildings themselves would soak up excess groundwater and evaporate the water from their walls and roofs, the authors write.

And there is a bonus: The evaporation of water from the building walls would additionally provide passive cooling, the team write.

Prof Marc-Olivier Coppens, director of the UCL Centre for Nature-Inspired Engineering, who was not involved in the study, described the teams synthetic mangrove device as remarkable, but said further developments would be needed.

The proposed device is a creative, exciting development; however, the application of this principle is still early stages, he said. Higher stability for longer times than those tested here, with less salt build-up, but also the possibility to be used for real seawater, containing more [chemical] species, would be necessary for practical use.

He added that the idea of using such synthetic mangroves for stormwater management was exciting. He said: It remains to be seen whether sufficient fluxes and total flows can be achieved for this application, but it is a compelling application.

Link:
Device inspired by mangroves could help clear up flood water - The Guardian

Investment Thesis

I last posted about TG Therapeutics (TGTX) in November last year after the company published long-awaited positive trial data concerning flagship drugs umbralisib and ublituximab, which caused a small uptick in the share price from ~$5 to ~$9.

I felt that TG was edging closer to commercialising its first drug and recommended its stock as a speculative investment - since then the share price has continued to grow and has reached $16 at the time of posting this update on further positive news flow. The next few months promise to be a critical period for the company.

Data is expected in the coming days from a pivotal trial - UNITY CLL - in which umbralisib and ublituximab are being used in combination (referred to as "U2") to treat chronic lymphocytic leukemia ("CLL"). The randomised trial - which began in November 2015 and has enrolled ~420 patients - compares U2 against a combination of Obinutuzumab and Chlorambucil with the primary outcome measure being progression-free survival ("PFS") assessed over 3 years and the secondary outcome being overall response rates ("ORR"). The company is waiting for the PFS data before deciding whether to push for accelerated approval from the FDA for the treatment.

What makes the data from this trial all the more significant is the impact it will have on a phase 1/2a triple combination trial TG is conducting where U2 is being combined with a third indication targeting CLL, venetoclax. Early data from this trial released in December suggested ORR of 87% after U2 induction and before the use of venetoclax, and the U2 induction was also shown to reduce risk of tumor lysis syndrome. After venetoclax was introduced a 100% ORR was achieved as well as a 44% complete response ("CR"). After 12 cycles of therapy 7 of 9 patients tested had undetectable minimal residue disease and stopped therapy.

According to a recent TG coporate presentation 115,000 Americans are affected by CLL with 20,000 patients being diagnosed with the disease every year. The size of the treatment market for CLL is estimated to reach ~$10bn by 2025 growing at a CAGR of 15%. TG believes that there is significant unmet need in this market and therefore should U2 receive approval as a stand-alone treatment for relapsed patients who have not responded to prior treatment or in combination with other drugs such as venetoclax as a first line treatment then TG's share price is likely to soar on the prospect of global drug sales in the triple-digit millions.

Besides U2 TG is progressing ublituximab through phase 3 trials as a potential treatment for multiple sclerosis ("MS") compared with oral teriflunomide with top line data expected sometime in 2020, and through phase 3 trials in combination with ibrutinib for treatment of high-risk CLL. Umbrasilib could soon win approval as a stand-alone treatment for MZL and FL after management submitted a joint NDA to the FDA in January. Other promising candidates are also in the early phases of clinical testing.

The company recently completed a $50m direct public offering of common stock to a single investor which has eased perennial fears over TG's precarious funding runway, and as such, a stellar 2020 could conceivably be in prospect.

It's worth remembering however that 1 bad trial result could seriously undermine TG's progress and decimate the share price. The fact that TG does not have any development partners for its drug candidates programs in the US and does not seem to be an acquisition target at present can be read either as an encouraging sign (TG can go it alone and earn bigger profits) or as slightly suspicious (do pharma insiders know something investors don't?).

On balance however, just as I did in November, I am prepared to give management the benefit of the doubt. One thing that is for certain is that the company's fortunes (and share price) are about to change significantly whether for better or for worse. After taking a deep breath, I remain bullish.

TG Therapeutics has a somewhat unique approach to drug discovery - founded in 2012 the company is focused on B-cell mediated diseases. B-cells are produced by hematopoietic stem cells in bone marrow from where they migrate to the spleen, lymph nodes or other tissues forming immunocompetent B-cells capable of initiating an immune response when activated by cell antigens. B-cells however can become cancerous causing diseases such as non-Hodgkin's lymphoma ("NHL") or CLL, or become aberrant causing auto-immune diseases such as MS, rheumatoid arthritis ("RA") or systemic lupus erythematosus ("SLE").

TG's strategy is to identify validated targets against B-cell mediated diseases and then conduct global searches for and acquire "best-in-class" drug candidates in early stages of development - preferably with human clinical proof-of-concept ("POC") from a network of academic research centres or biotech drug development companies. Having acquired a candidate the company then looks to use its proprietary technology platform to develop multiple complementary candidates that can be used either as single treatments or in combination.

The 2 drugs are TG's most advanced candidates. Ublituximab is an anti-CD20 monoclonal antibody, similar to commercialised drugs such as Rituximab, Ofatumumab or Obinutuzumab. CD20 tends to be over-expressed in cancerous or malignant B-cells - anti CD20 antibodies can attach themselves to these cells and trigger immune reactions - such as antibody dependent cellular cytotoxicity or phagocytosis - that result in the death of the malignant cell.

TG has bioengineered ublituximab using a process known as glyco-engineering so that certain naturally occurring sugar molecules have been removed from the antibody which the company says increases its potency and shortens drug-infusion times.

Umbralisib is a PI3K delta and CK1 epsilon inhibitor taken orally once a day. PI3K delta is part of a family of enzymes involved in various cell processes such as proliferation and immunity which tends to be implicated in B-cell related lymphomas, whilst inhibiting CK1 epsilon is also suggested to have anti-cancer effects and modulate adverse T-cell activity.

TG initiated this in January after data from a phase 2b study - Unity-NHL - of 72 patients with MZL and 118 with FL showed that the number of positive responses to umbrailsib as a single treatment was above the study threshold of 40%. Preliminary efficacy data from the first 42 patients with MZL also showed 52% of patients who had failed to respond to prior treatment responded to umbralisib and an additional 36% attained stable disease. Overall, umbralisib was well tolerated by patients - 10 patients discontinued treatment due to adverse events ("AEs") possibly related to the drug's side-effects, no deaths were reported and only 3 patients demonstrated grade 3 infections.

A rolling submission allows TG to file sections of the NDA on an ongoing basis, which is an unusual concession from the FDA, as is the permission to file one NDA for 2 separate indications (MZL and FL) - but umbralisib's prior breakthrough drug designation seems to have made this possible. The NDA should be submitted during the first half of 2020 moving the company one step closer to commercialisation.

The data behind TG's NDA submission for umbralisib. Source: TG Therapeutics analyst day presentation Jan 2020.

22,500 new cases of MZL or FL are diagnosed per annum (according to the American Cancer Society, quoted in TG's JPM Healthcare conference presentation), with ~6,000 - 10,000 relapsed indolent patients needing treatment per annum.

Ublituximab is now progressing through 2 fully-enrolled (500 patients) randomised phase 3 trials - ULTIMATE 1 and ULTIMATE 2 - where the drug is being evaluated against oral treatment Teriflunomide (marketed by Sanofi as Aubagio, Teriflunomide makes sales of ~$430m per quarter) with topline data expected in 2H 2020. During its phase 2 MS trials ublituximab patients' annualized relapse rates of 0.05 were favourably comparable to treatments Ocrelizumab (0.13), Rebif (0.36) and the placebo (0.64), whilst no T1 GD-enhancing lesions were present at weeks 24 and 48 of the trial compared to a baseline of 3.63.

TG estimates the global market for MS treatment will exceed $30bn by 2025 with the only other anti-CD20 approved treatment, Ocrelizumab (marketed as Ocrevus by Roche) currently making more than $2bn in annual sales (and on track to reach $4bn by 2025). Should approval be granted to ublituximab the drug will compete in a crowded field (as shown by the table below) but TG management believes that the convenience of the bi-annual, one-hour duration infusion, efficacy and pricing will translate to a $1 - $2bn market opportunity.

MS treatment options. Source: TG Therapeutics analyst day presentation Jan 2020.

TG Therapeutics drug candidate pipeline. Source: TG Website.

TG is progressing a number of other candidates with similar indications to umbrasilib and ublituximab.

TG 1701 is a novel covalent Brutons tyrosine kinase ("BTK") inhibitor that targets the B-cell receptor signalling pathway that helps to proliferate and protect B-lymphocytes. The drug has entered a phase 1 trial where it will be evaluated both as a single agent and in triple combination alongside U2, an interesting prospect for the company since success would enable them to provide an entirely in-house triple combination therapy.

TG-1501, a monoclonal antibody that attaches to Programmed Death-Ligand 1 (PD-L1) blocking the signals that cancerous cells use to protect themselves from the body's natural immune system. Similarly to TG-1701 the drug is in single agent and combination trials with other TG candidates.

Finally, TG-1801 is an ANTI-CD47/CD19 monoclonal antibody. CD47, is expressed by cancerous cells in order to express a "don't eat me" signal to avoid elimination through phagocytosis whilst CD19 is used by B-tumor cells to make up for a loss of CD20 and continue to resist treatment. TG-1801 has also entered a phase 1 trial.

In Q319 TG made a loss of $59.9m compared to a net loss of $34m over the same period in 2018. The difference was put down to manufacturing and CMC costs but the company says they decreased trial costs by $3.8m in the quarter and expect this reduction to continue into 2020. CFO Sean Power told analysts on the Q3 earnings call that management expects to reduce cash burn from Q3's $33m to $25m - $30m per quarter going forward, and that the cash position (at the time) of $96m would be sufficient to see the company through 2020 and the conclusion of the critical ongoing phase 3 trials.

The $50m raise completed in December will probably be viewed by investors as good business since the share price has subsequently risen and therefore the effects of dilution have been minimised. Still management has made repeated use of at-the-market offerings ("ATMs") with the most recent being a $24m raise at the end of Q319 - and further use of their mixed shelf offering may start to try backers' patience.

The upside for investors is that, besides several minor licensing agreements TG has resisted the temptation to enter into collaboration agreements with big-pharma concerns based on development milestones and post commercialisation revenue sharing, and therefore retains exclusive rights to all of its drug candidates.

TG clearly believes that commercialisation is imminent having appointed a new Chief Commercial Officer, Adam Waldman, who joins from Celgene where he was Head of Hematology-Oncology Marketing. Waldman has extensive experience building sales and marketing teams and will be expected to deliver the launch programs that TG will hope are initiated before the end of this year.

The upcoming results, firstly from the UNITY-CLL trial, and subsequently from ublituximab's MS trial will dictate whether or not TG proves to be a good investment or whether investors who have endured years of frustration will have to accept further short-to-medium term disappointment.

Management has done well in my view to progress different drug candidates to the near-conclusion of pivotal trials, win breakthrough drug designations and submit NDAs to the FDA that stand a good chance of success. It has taken time admittedly and the share price has suffered but it feels to me as though the company now has some momentum.

In my view, the signs are promising enough to suggest that TG will have a commercialised drug - and possibly 2 - by the end of the year. Should they be approved both drugs - perhaps ublituximab for MS more so than Umbrasilib for MZL and FL - will have sizeable addressable markets, and the prospect of an approval for treatment of CLL (U2 + venetoclax or even U2 + proprietary TG-1701) is a juicy one given the size of the market.

If the upcoming UNITY-CLL data is positive TG will surely make an attractive acquisition target for a big pharma - and the figures involved could be large - witness the $9.7bn Novartis recently paid Medicines Company for a single RNA therapy. Therefore I would put at least a 25-50% premium on TG's current price and look at a price of $21+, which is below analyst consensus targets of ~$25, and significantly below what TG could achieve if the market, as expected, reacts buoyantly to good news. With more candidates in development and because the company addresses a range of indications, the worst case scenario (in the event of poorly received data) is not catastrophic either.

Long-term, management will have to prove it has what it takes to successfully commercialise an approved drug and generate sales volumes that justify the high cash burn investors have endured for years. But that is for a different day. In the short term I remain bullish and see this as a potentially exciting time to be holding TG stock, although the usual biotech investing caveats still apply. Things can de-escalate quickly when data is not up to scratch.

Disclosure: I am/we are long TGTX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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TG Therapeutics Edges Closer To Commercialization And Potential Share Price Gain - Seeking Alpha