StemCell Therapy

The hottest biotechs in the field of hemophilia are stealing the show at this years edition of the EAHAD hemophilia congress in Paris.

A disorder for which no cure is available, hemophilia is caused by absent or defective genes coding for blood clotting factors, turning simple injuries intohealth risks and causing spontaneous bleeding. Researchers and companies worldwide working to improve hemophilia therapy are meeting this week in Paris for the10th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).

We recently reviewed the latest advances in hemophilia, a field teeming with innovative solutions and technology.Among the most interesting presented at the congress are new results on gene therapy and RNAi, a pen to treat hemophilia and many companies fighting to reduce thedosing frequency of prophylactic therapy. On top of that, Shire has reported that current estimates of people suffering from the disease could be completely wrong

Novo Nordisk ismaking plans to use its famous insulin pens to deliver hemophilia drugs. According to astudy evaluating user experience with the pens presented at the congress, participants liked the device as it is easy to use, well designed, more portable and involving fewer steps than their current kits for hemophilia.

The ultimate goal of the Danish company is to use its FlexTouch pen to deliverconcizumab, an antibody againsttissue factor pathway inhibitor (TFPI) currently in Phase I for both hemophilia A and B.

Shire has presented a study revealing that the incidence of hemophilia could be more than three times higherthan current estimates. It also showed that only 25% of hemophiliacs receive adequate treatment. These findings might push efforts to put an end to this situation and stimulate market growth.

Shirepresented positive results from a Phase II/III trial for Adynovate (BAX 855) in children with hemophilia A. Interestingly, the company also showed early stage in vitro results for combination therapies with a biosimilar of Roches emicizumab (ACE910). It looks like the antibody, not yet in the market, already has strong competitors getting ready for when its patent expires.

Sobi, inSweden, has co-developed recombinant clotting factors with an extended half-life in partnership with Biogens spin-offBioverativ. To do so, they fuse the clotting factor to the Fc portion immunoglobulin G1 proteins.

The team has presented positive long-term safety and efficacy results forEloctatein hemophilia A andAlprolixin hemophilia B. Both are already in the market and reduce dosing frequency to weekly injections.

OPKO Biologics, in Israel, follows a strategy similar to Sobis. ItsCTP technologyextends the half-life of proteins by fusing them with theC-terminal peptide of human chorionic gonadotropin (hCG).

The company has presented data forMOD-5014(FVIIa-CTP) supporting the advance intoPhase II/IIItrials. The drug is intended for delivery twice a week, which is double of that from Sobis products.

Spark Therapeuticsis one of the leaders in the development ofgene therapy for hemophiliaanduniQures main competitor. The American company will report results from itsPhase I/IItrial forSPK-9001in hemophilia B showing sustained activity of the therapy after12 weeks, with only one reported bleeding.

Despite good results, Spark is facing strong competition from the DutchuniQure. Its gene therapyAMT-060has already shown sustained effects for at least52 weeksin a patient subpopulation. Both companies nowhavebreakthrough designation from the FDA and therace to reach the market is tight.

Sanofis partner, Alnylam, is conducting clinical trials across the UK, Switzerland and Bulgaria to test its unique RNAi technology for hemophilia. Its candidate fitusiran, which blocks antithrombin to improve clotting,is proving safe and effective inPhase I/IItrials.

This unique treatment has the potential to reduce dosing to a monthly basis and is suitable for patients with both hemophilia A and B, also including those that have developed resistanceto standard treatments.

Among the companies presenting are many others includingGenentech, Bayer and Catalyst Biosciences. The sheer number of innovative approaches under development is great news. Such a wide arrange of solutions could provide a better quality of life for hemophilia patients, each treatment suited for the particular needs of differentpatients. Especially now that, thanks to Shire, scientists know the number of patients suffering from the condition could actually be much higher.

Images from Sashkin, nobeastsofierce, Roberta Canu, Mond Duang,Art tools, LeonP,Pakpoom Nunjui /Shutterstock.com

Read more:
Gene Therapy, RNA and Pens at European Hemophilia Congress in Paris - Labiotech.eu (blog)

This entry was posted on February 6, 2017 at 9:47 am and is filed under Gene therapy. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |