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Pfizer's Hemophilia B Gene Therapy Candidate Enters Phase III

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Pfizer Inc. (PFE - Free Report) announced that it initiated a phase III, lead-in study of an experimental gene therapy treatment, fidanacogene elaparvovec, for patients with hemophilia B. Fidanacogene elaparvovec, a high-activity human coagulation factor IX gene, is a one-time treatment option.

Pfizer also plans to conduct the next part of the phase III study and the patients who completeat least six months in the lead-in studywill be moved to this pivotal study. Fidanacogene elaparvovec was previously known as SPK-9001/PF-06838435 and was originally developed by Spark Therapeutics . Fidanacogene elaparvovec was transferred by Spark to Pfizer, following positive results announced from an ongoing phase I/II study in May 2018.

The results showed that all 15 patients in the study were able to discontinue routine infusions of factor IX concentrates with no reported serious adverse or thrombotic events. Consequently, Pfizer has now assumed sole responsibility for all subsequent pivotal studies, and regulatory and development activities of any product, resulting from the hemophilia B gene therapy program

Hemophilia B is a rare, genetic bleeding disorder found almost exclusively in males. It causes the blood to take a long time to clot because of a deficiency in one of several blood clotting factors, most notably clotting factor IX, a specific protein in the blood. It is expected that hemophilia B patients if once treated with fidanacogene elaparvovec will be able to produce factor IX themselves, rather than having to regularly inject factor IX.

Year to date, shares of Pfizer have increased 3.7% compared with the industry’s decline of 1.7%.

 

Gene therapy is set to become one of the most promising space in the volatile biotech sector, which continues to grapple with increasing threat from biosimilars. 

Pfizer is also developing other candidates for gene therapy.The company’s mini-dystrophin gene therapy candidate, PF-06939926 is in phase Ib clinical trial, being evaluated in boys with duchenne muscular dystrophy.

We remind investors that Spark, in December 2017, obtained the FDA approval for its lead gene therapy drug, Luxturna (voretigene neparvovec-rzyl), for the treatment of patients with viable retinal cells and confirmed biallelic RPE65 mutation-associated retinal dystrophy.

Luxturna is the first FDA-approved gene therapy for inherited retinal disease and the first adeno-associated virus (AAV) vector gene therapy approved United States. The company has built a pipeline of gene therapy candidates that are directed to the retina, the liver and the central nervous system.

The pipeline includes an ocular program consisting of SPK-7001, targeting choroideremia, currently in phase I/II clinical trials. Its hemophilia programs also consist of SPK-8011 for hemophilia A, currently in phase I/II clinical trials.

BioMarin Pharmaceutical (BMRN - Free Report) is also developing valoctocogene roxaparvovec, an experimental gene therapy for the treatment of patients with severe hemophilia. 

uniQure N.V. is another promising player in this space, which is creating a pipeline of innovative gene therapies that have been developed both internally and through its collaboration with Bristol Myers-Squibb (BMY - Free Report) .

 

Zacks Rank & Stocks to Consider

Pfizer has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank stocks here.

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