Sarepta Therapeutics CEO Doug Ingram is taking the high road.
In remarks Thursday on a quarterly earnings call, Ingram refused to criticize the Food and Drug Administration for rejecting Sarepta’s Duchenne muscular dystrophy drug Vyondys 53.
“We were treated fairly by the division of neurology,” he said, referring to the group of reviewers within the FDA who reviewed Vyondys but turned the drug away in August, citing the risk of infections related to intravenous infusion ports and kidney toxicity seen in animal experiments.
Some Sarepta investors and patient advocates within the Duchenne community have sharply criticized the FDA for rejecting Vyondys, and have prodded Sarepta to be more combative in efforts to overturn the decision. Ingram rejected that strategy, insisting it’s in Sarepta’s best interest to maintain a good working relationship with the FDA.
“We want to build a positive relationship” with the FDA, he said.
But all the kumbaya dished Thursday still doesn’t provide much clarity on Vyondys’s future. A timeline to finding a fix for those Vyondys safety issues was not offered by Ingram on Thursday. He refused to answer questions about whether Sarepta and FDA had scheduled a meeting to discuss the drug’s rejection. He also said no updates would be offered until the company knows how — or if — Vyondys can be approved.
The rejection of Vyondys and its uncertain future is also affecting a third Duchenne drug called casimersen. Sarepta said it would delay seeking approval for casimersen until the Vyondys issue is resolved.
On Sarepta’s program to develop a gene therapy against Duchenne, a Phase 3 clinical trial will start in the middle of 2020. An ongoing Phase 2 study was expanded from 20 to 40 patients, with dosing expected to be completed by the end of the year. Work on expanding Sarepta’s gene therapy manufacturing capacity are underway.
In the third quarter, Exondys 51 sales were $99 million, up 26% year over year. Sarepta raised its Exondys 51 sales guidance for 2019 to a range of $370-380 million from $365-375 million. The company reported a net loss $126.3 million, or $1.70 per share, in the third quarter.