NEW YORK – Sarepta Therapeutics on Tuesday said it has begun screening patients with limb-girdle muscular dystrophy for enrollment in the Phase III EMERGENE trial of its gene therapy SRP-9003, or bidridistrogene xeboparvovec.
The pivotal trial, which Sarepta CEO and President Doug Ingram discussed last week at the JP Morgan Healthcare Conference, will focus specifically on limb-girdle muscular dystrophy Type 2E (LGMD2E). Current medications for this rare, genetic neuromuscular condition, also known as a beta-sarcoglycanopathy, only manage symptoms but aren't disease modifying. SRP-9003 is designed to deliver a full-length beta-sarcoglycan transgene and uses an MHCK7 promoter, which is expressed in the heart and is a key protein in patients with this condition who are at risk of dying from pulmonary and cardiac problems.
The EMERGENE study will enroll 15 patients with LGMD2E who are at least 4 years old. The trial has a six-month natural history lead-in period, after which Sarepta will track patients' beta-sarcoglycan expression 60 days after receiving SRP-9003. Researchers will also measure patients' clinical functions through 60 months post-treatment and assess the gene therapy's safety.
In a statement, Louise Rodino-Klapac, executive VP, CSO, and head of R&D at Sarepta, said that in an earlier development program, SRP-9003 showed "significant protein expression" in patients 12 weeks and two years after treatment, and the gene therapy also appeared to slow disease progression and improve patients' mobility and quality of life.
In addition to SRP-9003, Sarepta has gene therapy programs in several other limb-girdle muscular dystrophy subtypes that account for more than 70 percent of cases. "In addition to its importance for the LGMD2E community, EMERGENE will inform the clinical development of other programs for LGMD in Sarepta's pipeline while serving as a pathfinder for viable regulatory pathways to support the development of gene therapies to treat ultra rare diseases," Rodino-Klapac said.