Text size
Pfizer has spent $800 million to build the leading production platform for gene therapies, the company said on Tuesday in its second day of talks to show how it plans to become a fast-growing innovator. Within three years, the drug giant hopes to launch three potential cures for hemophilia and muscular dystrophyworth as much as $4 billion in annual revenue.
We are in an unrivaled position to go to market, said the head of Pfizers rare-disease business, Suneet Varma, in his Tuesday morning presentation. In another Tuesday talk, the companys immunology leaders highlighted coming breakthroughs for the tens of millions who suffer miserable autoimmune skin disorders.
Pfizer (ticker: PFE) is eager to recast itself as a high-multiple growth stock. By year-end, its slower-selling products will be spun off into a business run by Mylan (MYL). From that point, Pfizer thinks it can boost revenue by 6% a year, despite the patent expirations of nearly $20 billion worth of its products in the latter part of this decade. The expected growth will be fired up by 25 product launches, which Pfizer is explaining in two days of online talks.
After rising Monday, Pfizer stock was down 0.3%, at $36.91, in recent trading. The S&P 500 was up 0.4%.
A rare disease may affect only a few hundred thousand people, but there are a lot of these diseases. Cumulatively, they add up to 400 million people worldwide, said Varma, with fewer than 5% having approved treatments. Pfizer believes the rare-disease market will grow at a double-digit annual rate.
Clinical trials are under way for treatments for the bleeding disorders known as hemophilia A and hemophilia B. After treatment, none of the enrolled patients have had bleeding problems, including some in which more than a year has passed since dosing.
Pfizer hopes for approval and launch of its hemophilia B treatment in 2022. Peak annual sales could hit $500 million to $1 billion, says the company. The hemophilia A launch could happen in 2023 and eventually exceed $1 billion in annual sales.
The gene-therapy treatments can require a short hospital stay. But the possibility of a cure will inspire 30% to 40% of hemophilia patients to seek treatment, Pfizer predicts.
A rival in hemophilia A gene therapy, BioMarin Pharmaceutical (BMRN), had a head start over Pfizer in clinical trials. But last month, the U.S. Food and Drug Administration rebuffed BioMarins application for approval and demanded additional follow-up of its patients for a period that will extend through the end of 2021.
The FDA hasnt asked Pfizer to change its design for the hemophilia A trial that the company will begin in a few weeks, said the chief medical officer of the rare-disease unit, Brenda Cooperstone,
There were some worrisome immune reactions among boys in the continuing clinical trial for Pfizers gene therapy for Duchenne muscular dystrophy. But after adjustments in the trials preparatory regime, Pfizer says there have been no more of those events. The treatment showed promising efficacy in early phase trials.
The pivotal Phase 3 muscular dystrophy trial will begin within weeks, with the first data expected in 2022. Pfizer hopes for a launch in 2023 of a muscular dystrophy therapy that would be used by 30,000 people a year in the U.S. and Europe, generating over $2 billion in annual revenue. to reach that goal, Pfizer is racing with Sarepta Therapeutics (SRPT).
In addition to its rare-disease treatments, Pfizer plans to launch four products for autoimmune disorders by 2025. Two of the products would treat atopic dermatitis, which causes painful itching in up to 30 million Americans. Only a portion of those patients get any treatment today.
Based on successful clinical trials, sufferers of the skin disease can look forward to breakthrough treatments, similar to those launched in recent years for psoriasis, says Pfizers inflammation and immunology business head, Richard Blackburn.
Corrections & Amplifications
Pfizers gene-therapy treatments can require short hospital stays. An earlier version of this article incorrectly said the treatments are like bone-marrow transplants, which require months of grueling hospital care.
Write to Bill Alpert at william.alpert@barrons.com
Read this article:
How Pfizer Plans to Lead the Industry in Gene Therapies - Barron's
- Extracellular vesicles for the delivery of gene therapy - Nature.com - March 9th, 2025
- Around the Helix: Cell and Gene Therapy Company Updates March 5, 2025 - CGTLive - March 9th, 2025
- Inside the secret island where wealthy people go to alter their DNA - Daily Mail - March 9th, 2025
- Regenerons Gene Therapy DB-OTO Trial Shows Promising Hearing Improvement - The Hearing Review - March 9th, 2025
- Global Cell and Gene Therapy Manufacturing Market to Reach ~USD 10 Billion by 2032 | DelveInsight - GlobeNewswire - March 9th, 2025
- College Station gene therapy company partners with nonprofit to develop treatments for rare diseases - KBTX - March 9th, 2025
- World Hearing Day 2025: Looking Back at Progress in Gene Therapy - CGTLive - March 9th, 2025
- Reflecting on a milestone year for cell and gene therapies - Pharmaceutical Technology - March 9th, 2025
- Q&A: Whats Next for Hemophilia Gene Therapy? | Newswise - Newswise - March 9th, 2025
- 'Llife-changing' gene therapy in London partially restores CT child's sight - CT Insider - March 9th, 2025
- The Genesis of Cell Therapy: Bridging Traditional Pharmacology and Gene Therapy - Technology Networks - March 9th, 2025
- Regenxbio at TD Cowen Conference: Gene Therapy Advancements - Investing.com - March 9th, 2025
- Anova Announces First Patient Enrolled to Phase 1/2a Study of DB107 for the Treatment of High-Grade Gliomas - Business Wire - March 9th, 2025
- Apertura Gene Therapy Supports the Broad Institute in Development of Gene Therapy for Prion Disease Using Engineered AAV Capsid Targeting TfR1 for CNS... - March 9th, 2025
- Gene therapy research offers hope for people with chronic kidney disease - Medical Xpress - January 6th, 2025
- Sangamo Therapeutics to Regain Full Rights to Hemophilia A Gene Therapy Program Following Pfizers Decision to Cease Development of Giroctocogene... - January 6th, 2025
- JCR Pharmaceuticals and Modalis Therapeutics Announce Transition to the Next Phase of Joint Research Agreement for Development of Novel Gene Therapy -... - January 6th, 2025
- Gene therapy targets the retina to treat eye disease - Nature.com - January 6th, 2025
- Sangamos Stock Plummets as Pfizer Axes Hemophilia Gene Therapy Pact - BioSpace - January 6th, 2025
- How Increased Use of Gene Therapy Treatment for Sickle Cell Disease Could Affect the Federal Budget - Congressional Budget Office - January 6th, 2025
- The Future of Regulatory Processes in Cell and Gene Therapy - Pharmaceutical Executive - January 6th, 2025
- CGTLive's 2024 Pillars of Progress: Most-Watched Conference Interviews - CGTLive - January 6th, 2025
- Pfizer cuts losses on near-approval hemophilia gene therapy, adding to troubled Sangamo's woes - Fierce Biotech - January 6th, 2025
- JCR Pharmaceuticals and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- JCR and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- Novartis Gene Therapy Shows Promise in Treating SMA - Yahoo Finance - January 6th, 2025
- Gene Therapy Market to Hit Valuation of US$ 42.26 Billion By 2033 | Astute Analytica - Yahoo Finance - January 6th, 2025
- Novartis gene therapy helps children with rare muscle disorder in study - Reuters - January 6th, 2025
- Capricor Puts Rolling BLA for DMD Cardiomyopathy Cell Therapy Deramiocel in Front of the FDA - CGTLive - January 6th, 2025
- Positive data could expand use of Novartis gene therapy for SMA - Yahoo Finance - January 6th, 2025
- Sangamo spirals after Pfizer halts hemophilia A gene therapy partnership - MM+M Online - January 6th, 2025
- Cell Therapy and Gene Therapy CDMO Market to Reach USD 11.11 Billion by 2030 | Discover Growth Trends and Insights | Valuates Reports - PR Newswire - January 6th, 2025
- Struggling With Adoption, Sickle Cell Gene Therapy Manufacturers Embrace CMS Model - News & Insights - January 6th, 2025
- Sangamo Therapeutics to Regain Rights to Gene Therapy Program from Pfizer - Contract Pharma - January 6th, 2025
- Researchers Create Gene Therapy with Potential to Treat Peripheral Pain ... - December 28th, 2024
- How CRISPR Is Changing Cancer Research and Treatment - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - December 28th, 2024
- 100 cell and gene therapy leaders to watch in 2025 - December 28th, 2024
- Can a new gene therapy reverse heart failure? - Futurity - December 28th, 2024
- Sustained visual improvements in LHON patients treated with AAV gene therapy - Medical Xpress - December 28th, 2024
- Nebraska Medicine administers novel gene therapy to first hemophilia ... - December 28th, 2024
- Gene Therapy for Cardiomyopathies Presents Promising Alternative to Current Treatment - Managed Healthcare Executive - December 28th, 2024
- Stem Cell Transplantation Still the Main Treatment Option for Beta-Thalassemia - Medpage Today - December 28th, 2024
- Caribou Overhyped Gene-Therapy Testing, Investor Class Suit Says - Bloomberg Law - December 28th, 2024
- WuXi AppTec sells off cell and gene therapy operations in US, UK - FirstWord Pharma - December 28th, 2024
- Top 5 Print Publication Articles of 2024 - Managed Healthcare Executive - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - Medpage Today - December 28th, 2024
- UPenn gene therapy pioneers biotech gets $34 million in funding - The Philadelphia Inquirer - December 28th, 2024
- PHC Corporation to present LiCellGrow at Advanced Therapies Week 2025 - Drug Target Review - December 28th, 2024
- The Evolution of Cell & Gene Therapy: Development and Manufacturing Insights and the Role of CDMOs - Pharmaceutical Technology Magazine - December 28th, 2024
- Pig kidney transplants, new schizophrenia drug: Here are 5 of the biggest medical breakthroughs in 2024 - ABC News - December 28th, 2024
- Cell Therapy Manufacturing Trends And Advancements Continuing In 2025 - BioProcess Online - December 28th, 2024
- Can Gene Therapy Treat Chronic Pain? - LabRoots - December 28th, 2024
- Driving innovation: India's foray into gene and cell therapies - The Economic Times - December 28th, 2024
- Governor Hochul Celebrates the Opening Of New York's First Cell and Gene Therapy Hub at Roswell Park Comprehensive Cancer Center in Buffalo - PR Web - December 19th, 2024
- GenSight Biologics Provides Update on Regulatory Discussions and Financial Situation - Business Wire - December 19th, 2024
- Atsena completes dosing in part A of X-linked retinoschisis gene therapy trial - Healio - December 19th, 2024
- Astellas and Sangamo Therapeutics Announce Capsid License Agreement to Deliver Genomic Medicines for Neurological Diseases - StreetInsider.com - December 19th, 2024
- Ring Therapeutics lays off just under half of staff in 2nd wave of cuts this year, CEO set to step down - Fierce Biotech - December 19th, 2024
- Gov. Hochul celebrates opening of first cell and gene therapy hub in NYS - WIVB.com - News 4 - December 19th, 2024
- Muscular Dystrophy Association and Coalition to Cure - GlobeNewswire - December 19th, 2024
- Atsena Therapeutics Announces Dosing Completed in Part A of - GlobeNewswire - December 19th, 2024
- 'A milestone moment': Roswell Park celebrates opening New York's first cell and gene therapy hub - WKBW 7 News Buffalo - December 19th, 2024
- Gene therapy to prevent stillbirth and premature delivery developed - News-Medical.Net - December 19th, 2024
- Breaking through the blood-brain barrier - Science - December 19th, 2024
- Cell therapy weekly: partnerships for advancing cell and gene therapies - RegMedNet - December 19th, 2024
- Roswell Park Opens Cell, Gene Therapy Hub - WGRZ.com - December 19th, 2024
- Cartherics gets $300k grant to advance Cell and Gene Therapy development - ETHealthWorld - December 19th, 2024
- ELMCRx Solutions Offers Cell & Gene Therapy Support Through Partnership with Emerging Therapy Solutions (ETS) - Business Wire - December 19th, 2024
- Fueling the Future of Gene Therapies with Manufacturing Innovation, Upcoming Webinar Hosted by Xtalks - PR Web - December 19th, 2024
- Concinnity secures 3M Seed funding to advance AI-driven gene therapy safety - Tech.eu - December 19th, 2024
- Viral Vectors-Based Gene Therapy for Non-Human Primates Market to Reach Over USD 92.76 Million by 2034 - EIN News - December 19th, 2024
- The pharma industry's silence on RFK Jr., and efforts by parents to develop gene therapies for their children - STAT - December 19th, 2024
- Tenaya reports positive early data on heart gene therapy - Investing.com - December 19th, 2024
- Unraveling The Complexity Of Cell Therapy: Advancements And Challenges - Life Science Leader Magazine - November 27th, 2024
- Novartis wagers more than $1B on gene therapies for the nervous system - BioPharma Dive - November 27th, 2024
- Gene therapy for geographic atrophy in age-related macular degeneration: current insights - Nature.com - November 27th, 2024
- Novartis buys gene therapy startup Kate Therapeutics, joining pursuit of muscular dystrophy treatment - STAT - November 27th, 2024
- At MGB's gene therapy institute, effort to win first venture capital investments continues - The Business Journals - November 27th, 2024
- Neurogene reports death of Rett patient left in critical condition by high dose of gene therapy - Fierce Biotech - November 27th, 2024