Pfizer aims to be the third big pharma with a significant presence in gene therapy. Its plans to initiate this year three Phase 3 trials targeting mutation-driven blood and muscular diseases would make it a large player in this cutting-edge area of medicine.
The difference between Pfizer and its Swiss rivals Novartis and Roche is that its treatments for muscular dystrophy and hemophilia do not look like they will be the first to market. With hopes that gene therapy could be a one-and-done treatment, arriving second could put Pfizer at a disadvantage if eager patients rush for curative therapies.
Having spun of its off-patent drugs business, the pharma is now trying to talk up the "new Pfizer." Its gene therapies are among seven pipeline projects that it cited Tuesday during its year-end earnings call as critical to its strategy of becoming a more innovation-focused company.
Company executives weren't, however, asked to answer how Pfizer views the emerging gene therapy competition. BioMarin Pharmaceutical looks set to get to the market earlier in hemophilia A than Pfizer, while Uniqure in hemophilia B and Sarepta Therapeutics in Duchenne muscular dystrophy appear ahead.
Pfizer's hemophilia A project, the Sangamo Therapeutics-originated SB-525, is up against BioMarin's valrox, which has been submitted to the Food and Drug Administration for an approval decision later this year.
In hemophilia B, fidanacogene elaparvovec, licensed from Roche subsidiary Spark Therapeutics, is in a neck-and-neck race with UniQure's etranacogene dezaparvovec in Phase 3 testing. Duchenne research, meanwhile, is led by Sarepta, which is launching a Phase 3 trial of its drug this year, putting Pfizer's at a disadvantage.
Other than announcing its intent to launch Phase 3 trials in hemophilia A and Duchenne, Pfizer didn't provide much more detail about these clinical programs. Mikael Dolsten, Pfizer's chief scientific officer, said more could be revealed about the DMD program at an upcoming research & development day.
Progress on that project had been delayed after one patient was hospitalized with kidney complications, but Dolsten said trial investigators had dosed additional patients. The Phase 2 will wrap up this spring, and the new data and longer follow-up will help guide a Phase 3 trial design, the company said.
Dolsten also described the hemophilia A project as having a 'best-in-class profile," even though BioMarin's valrox has impressed hematologists with its ability to increase expression of a key blood-clotting protein.
In addition, he said the company hopes it can bring one new gene therapy into its pipeline per year.
Building its drug development portfolio is one reason why the company has chosen not to buy back shares, said CEO Albert Bourla.
He pointed to the company's need in the past to buy back shares to support their valuation because of revenue declines, but now he said the company is in a different strategic position.
"The company is going to have a best-in-class revenue growth story," he said. "We can use the capital to invest in good Phase 2, Phase 3 assets to grow our pipeline."
Read the original post:
- Gene Therapy for Frontotemporal Dementia With a GRN Mutation Gets Fast Track Status - Monthly Prescribing Reference - March 26th, 2020
- Redpin bags A round to advance Chantix-controlled gene therapies - FierceBiotech - March 26th, 2020
- Vycellix and Avectas Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies - BioSpace - March 26th, 2020
- Avectas and Vycellix Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies - BioSpace - March 26th, 2020
- Global Viral Vector and Plasmid DNA Manufacturing Market to Surpass US$ 2,205.6 Million by 2027 - CMI - Yahoo Finance - March 26th, 2020
- Codexis and Takeda partner on gene therapies for rare diseases - Pharmaceutical Technology - March 26th, 2020
- Genprex Strengthens Leadership Team with the Appointment of Two Accomplished Life Science Executives - Yahoo Finance - March 26th, 2020
- Study reveals gene therapy may help in treating cardiac disease - The Siasat Daily - March 26th, 2020
- TCR therapy an attractive alternative to CAR T for immunotherapy - Drug Target Review - March 26th, 2020
- Genprex Bolsters Board of Directors, Appoints Three Biotechnology and Healthcare Industry Leaders - Insurance News Net - March 26th, 2020
- 89bio Appoints Healthcare Industry Veteran Steven Altschuler, MD, as Chairman of the Board of Directors - BioSpace - March 26th, 2020
- Cancer Gene Therapy Market Boosting the Growth Worldwide: Cancer Gene Therapy Market Dynamics and Trends, Efficiencies Forecast 2025 - Daily Science - March 26th, 2020
- Serial child rapist sentenced to up to 126 years in jail; new gene therapy from UPMC could cure diabetes: Tod - PennLive - March 26th, 2020
- Visiongain Report Looks at Opportunities Within the $23bn Cell Therapy Technologies Market - Yahoo Finance - March 26th, 2020
- Gene therapy - Mayo Clinic - March 16th, 2020
- gene therapy | Description, Uses, Examples, & Safety ... - March 16th, 2020
- Gene Therapies: Overcoming the Biggest Hurdles in... - Labiotech.eu - March 16th, 2020
- Almost 400 cell and gene therapies in US pipeline, finds report - European Pharmaceutical Review - March 16th, 2020
- Homology Medicines Announces Peer-Reviewed Publication of HMI-102 Investigational Gene Therapy Demonstrating Restoration of Normal Metabolic Pathway... - March 16th, 2020
- Gene therapy giving Flames executive Snow hope in ALS fight - NHL.com - March 16th, 2020
- Gene Therapy Reverses Heart Failure in Mouse Model - Technology Networks - March 16th, 2020
- UAE's first gene therapy improves eyesight of two Emirati sisters - Gulf News - March 16th, 2020
- Construction of a replication-competent retroviral vector for expression of the VSV-G envelope glycoprotein for cancer gene therapy. - Physician's... - March 16th, 2020
- Week In Review: Nanjing Legend Files To Stage IPO In The U.S. - Seeking Alpha - March 16th, 2020
- Kuur Therapeutics Launches to Develop and Commercialize Off-the-shelf CAR-NKT Cell Therapies Targeting Hematological and Solid Tumors - Business Wire - March 16th, 2020
- Why Soap Works - The New York Times - March 16th, 2020
- Gene Therapy Is Successfully Treating a Common Form of Inherited Blindness - Singularity Hub - March 4th, 2020
- Biogen teams up with Sangamo in gene therapy deal worth up to $2.7B - MedCity News - March 4th, 2020
- Staff: Cell gene therapy sector needs reinforcements - Bioprocess Insider - BioProcess Insider - March 4th, 2020
- CYTOO and AskBio Enter Research Agreement to Screen Gene Therapy Candidates for Rare Muscle Disorder - Yahoo Finance - March 4th, 2020
- Symbiosis on increased demand for viral vectors - BioPharma-Reporter.com - March 4th, 2020
- Prevail Therapeutics Announces Investigational New Drug Application Active for PR006 for the Treatment of Frontotemporal Dementia with GRN Mutation |... - March 4th, 2020
- Prevail Therapeutics Provides Clinical Advancement Update on PR001 for the Treatment of Parkinson's Disease with GBA1 mutationsCompany Will Present at... - March 4th, 2020
- Avectas and CCRM Announce Collaboration to Accelerate Development of Novel Technology for Cell Therapies - Yahoo Finance - March 4th, 2020
- First-Year Lab Experience Gave This Student the Confidence to Aim for a Ph.D. - UVA Today - March 4th, 2020
- Voyager Therapeutics Announces Fourth Quarter and Full Year 2019 Financial Results and Corporate Updates - Yahoo Finance - March 4th, 2020
- How does gene therapy work? - Genetics Home Reference - NIH - February 24th, 2020
- Nationwide Childrens among hospitals leading the way in gene therapy - The Columbus Dispatch - February 24th, 2020
- BioMarin gene therapy won't need an AdComm as it nabs speedy FDA review - FierceBiotech - February 24th, 2020
- Gene therapy to halt rare form of sight loss - BBC News - February 24th, 2020
- With supply tight, Novartis readies gene therapy plant for production - BioPharma Dive - February 24th, 2020
- New Gene Therapies Institute Aims to Address Regulatory Barriers - HealthITAnalytics.com - February 24th, 2020
- Bluebird's gene therapy hits another delay, this time in the US - BioPharma Dive - February 24th, 2020
- First patient undergoes Luxturna gene therapy on NHS - PharmaTimes - February 24th, 2020
- First Patients Begin Gene Therapy Treatment for Blindness through NHS - Interesting Engineering - February 24th, 2020
- Spark R&D chief High exits in wake of Roche takeover - FierceBiotech - February 24th, 2020
- Why So Many Biotechs Are Scrambling to Develop a Drug for the Same Rare Disease - The Motley Fool - February 24th, 2020
- 3 Cancer Treatment Stocks to Buy Right Now - The Motley Fool - February 24th, 2020
- The Most Important Stock to Own Over the Next Decade - Nasdaq - February 24th, 2020
- FDA Continues Strong Support of Innovation in Development of Gene Therapy Products - FDA.gov - February 2nd, 2020
- Interpreting Sameness Gene Therapy Products Orphan Drug Regulations - FDA.gov - February 2nd, 2020
- Global Gene Therapy Industry Outlook 2020-2024 - Strong Product Pipeline Gives Rise to Lucrative Growth Opportunities - P&T Community - February 2nd, 2020
- FDA guidance on gene therapies development and manufacturing - BioPharma-Reporter.com - February 2nd, 2020
- Devastation, then hope: Platteville baby first in state treated for rare condition after screening - Madison.com - February 2nd, 2020
- Studies target unilateral gene therapy injection - Ophthalmology Times - February 2nd, 2020
- Otonomy Presents Preclinical Results for GJB2 Gene Therapy Collaboration and Cisplatin Otoprotection Program - Yahoo Finance - February 2nd, 2020
- Eyeing a trio of trial initiations, Jim Wilson's gene therapy startup woos Bruce Goldsmith from Deerfield as CEO - Endpoints News - February 2nd, 2020
- The FDA unveils a new regulatory framework to speed along gene therapies, rewarding the leading players - Endpoints News - February 2nd, 2020
- Gene Therapies Make it to Clinical Trials - Discover Magazine - December 31st, 2019
- Experimental Gene Therapy Shows Promise for Preventing and Treating Lou Gehrig's Disease in Mice - BioSpace - December 31st, 2019
- 2019: The year gene therapy came of age - INQUIRER.net - December 31st, 2019
- BLA Submitted for Gene Therapy to Treat Hemophilia A - Monthly Prescribing Reference - December 31st, 2019
- First Alzheimer's Disease Gene Therapy Human Study Provided by Maximum Life Foundation, Offers 10 Free Therapies for Qualifying Patients - Benzinga - December 31st, 2019
- Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research - News18 - December 31st, 2019
- Gene therapy to conquering hepatitis C: A decade of medical breakthroughs - Business Standard - December 31st, 2019
- What were the biggest biopharma stories of 2019? - MedCity News - December 31st, 2019
- Global Gene Therapy Market 2019 Revenue, Opportunity, Forecast and Value Chain 2025 - Market Research Sheets - December 31st, 2019
- Manufacturing: the next breakthrough in gene therapy - STAT - December 23rd, 2019
- Novartis in talks with patients upset about lottery-like gene therapy giveaway - Reuters - December 23rd, 2019
- Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options - Xconomy - December 23rd, 2019
- Gene Therapy for Sickle-Cell Anemia Looks Promisingbut It's Riddled With Controversy - Singularity Hub - December 23rd, 2019
- Making advanced therapies takes industrializing personalization - STAT - December 23rd, 2019
- Dyno Therapeutics Launches to Improve Viral Vectors for Gene Therapy - BioSpace - December 23rd, 2019
- New Gene Therapy Method May Open BRAVE New World in Parkinson's - Parkinson's News Today - December 23rd, 2019
- Pharma's gene and cell therapy ambitions will kick into high gear in 2020despite some major hurdles - FiercePharma - December 23rd, 2019
- Viewpoint: EU should take a lead in enforcing the corporate social responsibility of gene therapy manufacturers - Science Business - December 23rd, 2019
- Takeda Presents Data for Hemophilia A and B Gene Therapy Optimization - Hemophilia News Today - December 23rd, 2019
- GenScript Biotech to Host Global Forum on Cell and Gene Therapy and the Booming China Market During JPM Week - PRNewswire - December 23rd, 2019
- The gene therapy research that could save a family of four - News - The University of Sydney - December 23rd, 2019
- Waning treatment is a warning for all 'one-and-done' therapies - STAT - December 23rd, 2019