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Pfizer Starts Phase Ib for Duchenne Muscular Dystrophy Drug

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Pfizer Inc. PFE has announced initiation of dosing in a phase Ib study, evaluating its mini-dystrophin gene therapy candidate, PF-06939926, for the treatment of boys aged from five to 12 years with duchenne muscular dystrophy (DMD). Notably, DMD is a genetic disease characterized by muscle degeneration and weakness. The company anticipates initial data from the study in the first half of 2019.

The phase Ib study is an open-label, non-randomized, ascending dose assessment, to be conducted on approximately 12 boys with DMD. The study is designed to evaluate the safety and tolerability of PF-06939926 and also assess muscle strength, quality and function in a given patient population.

Shares of Pfizer were up almost 1.5% on Apr 12, following the above news release. However, the stock has gained 7.2% in the past year, marginally underperforming the industry’s increase of 7.4%.


We remind investors that PF-06939926 is added to Pfizer’s portfolio following the acquisition of Bamboo Therapeutics in August 2016. The acquisition complements Pfizer’s rare disease portfolio and enhanced its leadership position in gene therapy.

Additionally, last year, Pfizer entered into a collaboration agreement with other companies for advancing gene therapy programs. Notably, in May 2017, Pfizer inked agreement deal with Sangamo Therapeutics to develop and commercialize gene therapy programs for Hemophilia A. Meanwhile, last November, the company amended its contract with Spark Therapeutics ONCE to advance a Hemophilia B gene therapy candidate, which is being evaluated in a phase I/II study.

Nonetheless, this April 2018, Pfizer announced an alliance with private biotech, Allogene Therapeutics, to expedite the development of its allogeneic CAR-T candidate, UCART19, for the treatment of CD19-expressing hematological malignancies.

Notably, DMD is one of the most common fatal genetic disorders affecting children around the world. It is a devastating and incurable muscle-wasting disease. Per data provided by the National Human Genome Research Institute, DMD is a devastating and debilitating disease affecting up to one in 3,500-5,000 male births globally. Thus, there is a significant unmet need for DMD treatments.

At present, two approved therapies for the disease in the U.S. market are Serepta Therapeutics’ SRPT Exondys 51 (eteplirsen) and Marathon Pharmaceuticals’ Emflaza (deflazacort), the first therapy approved for DMD in the country.

Importantly, Pfizer is not the only company working on developing treatments for DMD. Other companies also involved in developing DMD therapies include Catabasis, Summit, PTC Therapeutics, BioMarin BMRN et al.

Zacks Rank

Pfizer carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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