An experimental gene therapy from Sarepta Therapeutics (SRPT) showed "remarkable" stability at two years in muscular dystrophy patients, an analyst said Friday as SRPT stock edged higher.
The biotech company tested a low dose of its gene therapy, dubbed SRP-9003, in three patients with limb-girdle muscular dystrophy. This form of muscular dystrophy is caused by deficient levels of beta-sarcoglycan, a protein tied to muscle function.
Patients showed an increase in beta-sarcoglycan levels after two years. On average, the protein was expressed in 54% of tissues after two years. That increased from 36% at 60 days after receiving the gene therapy. On a scale measuring muscle function, patients showed an average improvement of 5.7 points at 18 months. That score was still intact at the two-year mark.
Needham analyst Chad Messer said the results showed "remarkable stability."
"The three patients continue to demonstrate improvements from baseline in all functional measures including time-to-rise, four-stair climb, 100-meter walk test and 10-meter walk test," Messer said in a report to clients. He maintained his buy rating and 166 price target on SRPT stock.
SRPT Stock Rises On Gene Therapy Test
In morning trading on today's stock market, SRPT stock edged up a fraction near 83.20. Earlier, shares popped 3.6%.
The long-term functional data "bolsters confidence" in Sarepta's gene therapies, SVB Leerink analyst Joseph Schwartz said in his note to clients.
Sarepta also unveiled results for a group of patients who received a higher dose of the gene therapy a year ago. All three patients showed increases in beta-sarcoglycan and functional improvements. At one year, they showed an average 4-point improvement from baseline on a key scale.
"We believe these new data are incrementally positive, and support the hypothesis that gene therapy can lead to function improvements in patients with muscular diseases," Mizuho Securities analyst Difei Yang said in a note. She has a buy rating and 160 price target on SRPT stock.
Gene Therapies Share A Component
The news helps SRPT stock, which tumbled to a two-year low in January after its experimental gene therapy for Duchenne muscular dystrophy produced mixed results in a test called Study 102. This form of gene therapy treats a condition due to lacking dystrophin protein. The gene therapy known as SRP-9001 produced large amounts of dystrophin, but patients didn't show functional improvements.
But the two gene therapies share a key component, Schwartz said.
"We see incrementally positive read-though from (Thursday's) update not only to Sarepta's broader limb-girdle muscular dystrophy portfolio, but also to Sarepta's SRP-9001, which has been knocked off its pedestal recently following disappointing Phase 2 Study 102 results in January," he said.
Schwartz kept his outperform rating on SRPT stock.
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