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A question hanging over gene therapies, which attempt a once-and-done fix for genetic diseases, is will their benefits endure? Monday, Sarepta Therapeutics told a scientific conference that the first four children who got its investigational gene therapy, for Duchenne muscular dystrophy, all continued to show better-functioning muscles after two years.

Durability is an important consideration for a onetime treatment, said Sareptas head of gene therapy, Louise Rodino-Klapac. She spoke on a conference call, part of an online version of the annual congress of the World Muscle Society, where scientists reported their progress against the often-deadly muscular dystrophies.

Sarepta (ticker: SRPT) said that a battery of tests that measure daily functions like walking and fatigue showed the children doing even better than they had at the one-year mark. The assessment is an average of 17 tests, and not every child showed improvement on every measure.

Perhaps that is why Sarepta stock slipped 0.5% on the day, to $143.20, in a rising stock market.

Analysts may have also been anxious to hear whether Sarepta had resolved questions raised by the U.S. Food and Drug Administration about the production process to be used in a Phase 3 study of the gene therapy. Agency demands have slowed the timetables of other gene- therapy developers, and its questions for Sarepta are delaying the launch of its pivotal trial.

Chief executive Doug Ingram said Sarepta was ready with a process to produce therapies for a clinical trial and subsequent commercial demand. Were working with the agency right now to resolve any questions that they might have, said Ingram, get their blessing and be starting that trial as soon as is possible.

RBC Capital analyst Brian Abrahams found the data encouraging. He rates Sarepta stock at Outperform, with a price target of $200 a share.

We view the results as continuing to support true functional benefits from the companys gene therapies, Abrahams said in a Monday note. These studies werent controlled, he cautioned, so investors are looking forward to Sareptas results from a randomized, controlled study for which results should be available in the first quarter of 2021.

On Sareptas heels is Pfizer (PFE), with clinical trials of a gene therapy for muscular dystrophy whose benefits it has measured out to one year.

At the conference, Sarepta also showed data from early-stage trials of a gene therapy for another group of inherited muscle diseases, known as Limb-girdle muscular dystrophies. Functional tests of the first three patients showed them maintaining their improvement over 18 months.

CEO Ingram reminded the audienceand perhaps any listening regulatorsthat families are impatient for these muscular dystrophy trials conclusions.

This is truly -- Im not being hyperbolic, but I say in a very real sense, at least from our perspective, a matter of life and death, he said. We have to get this trial started. Kids are waiting for this therapy.

Write to Bill Alpert at william.alpert@barrons.com

The rest is here:
Sareptas First Gene-Therapy Test Patients Look Good, 2 Years Out - Barron's

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