Sarepta Therapeutics, Inc. (NASDAQ: SRPT)
Shares of Cambridge, Massachusetts-based Sarepta Therapeutics rocketed on Tuesday morning after the company released encouraging data from an early-phase trial of its experimental gene therapy for treating Duchenne muscular dystrophy (“DMD”) disease.
DMD is a rare genetic disease that wastes the muscles, and is caused by dystrophin absence.
The company said the results showed that the drug considerably increased dystrophin protein in three patients (boys) that had been diagnosed with the disease.
The data collected from the patients showed profound effect of the gene therapy, enough to suggest it may stop or even set back the effects of DMD in some patients.
The principal investigator of the study Dr. Mendell said
“I have been waiting my entire 49-year career to find a therapy that dramatically reduces CK levels and creates significant levels of dystrophin. Although the data are early and preliminary, these results, if they persist and are confirmed in additional patients, will represent an unprecedented advancement in the treatment of DMD.”
“I look forward to treating more patients in the clinical study to generate the data necessary to bring this therapy to patients with DMD, with the goal of dramatically changing the course of the disease,” Mendell added.
The stock’s gain of 55% makes Sarepta Therapeutics the fourth most valuable pharma in Massachusetts, behind Alexion Pharmaceuticals, Biogen, and Vertex Pharmaceuticals.
Sarepta Therapeutics CEO Comments
Sarepta Therapeutics president and chief executive officer Doug Ingram, said, “As a genetic medicine company, our goal is to work with the world’s leading clinicians and scientists to advance scientific discoveries to the clinic and, ultimately, to therapies that profoundly improve and extend the lives of those living with Duchenne muscular dystrophy and other rare, fatal diseases.”
Ingram continued, “Since the discovery of the dystrophin gene in 1986, scientists, clinicians, patient advocates and the biotech ecosystem have tirelessly searched for ways to restore or replace dystrophin and rescue boys with DMD from the damage and early death. Dr. Mendell’s results, if confirmed in additional patients, studies, measures and time points, represent a monumental leap forward in the direction of our goal.”
Sarepta Therapeutics, Inc. Company Profile
Sarepta Therapeutics, Inc. engages in the discovery and development of gene therapy, ribonucleic acid-based therapeutics, as well as other genetic medical approaches for treating rare neuro-muscular diseases.
Sarepta offers a number of disease-modifying therapies proprietary products which include EXONDYS 51, Casimersen, Golodirsen, and SRP-5051.
The drugmaker also has a strategic partnership with Nationwide Children’s Hospital for the research and advancement of micro dystrophin gene therapy program under the license and research option agreement.
It also has a license and research option partnership for the advancement of gene editing CRISPR/Cas9 tech with Duke University for muscular dystrophy.
In addition, it has a license and collaboration agreement with Summit Limited to commercialize products in its utrophin modulator pipeline.
