The FDA has some questions for Adverum Biotechnologies about the CMC work related to their lead gene therapy for wet, age-related macular degeneration, and regulators have put a clinical hold on their clinical development program until they get some answers.
The Menlo Park, CA-based biotech $ADVM reported Monday as the market opened that the agency had placed a hold on their drug “in early April” and were now reviewing a response to their query, submitted last week. This was the first chance that investors got to hear about this news from the public company, which reserved word about the FDA action until after they noted they had a green light from the independent data monitoring committee for the recruitment of their second cohort of patients for ADVM-022 — which they say was the chronological order of events.
This is their only clinical-stage program. But the news seems to be sitting well with its shareholders. The stock is up 7% in mid-afternoon trading.
I asked in a followup query to the company why they didn’t file anything with the SEC on the clinical hold when it occurred, or within a few days of the event. I haven’t heard back.
The treatment involves an aflibercept coding sequence which is delivered to the eye via a company-owned vector. Aflibercept is a drug delivered to the eye via injection, making any reduction in treatments something most patients would welcome.
Edward Nash at SunTrust says he’s been in touch with the executive crew at the biotech and noted that under the best case scenario the program would be delayed a month. That, obviously, could stretch out if the FDA takes more time to suss things out.
Adverum was formed out of Avalanche Biotechnologies’ acquisition of Paris-based Annapurna and their merger into a new gene therapy company following some trouble at Avalanche. Late last year the biotech abandoned their then lead, ADVM-043, after researchers determined it was having an insufficient effect on A1AT deficiency.
“We are working with the FDA to resolve this matter as quickly as possible,” said Adverum CEO Leone Patterson in a statement. “We are deeply committed to the development of our novel gene therapy ADVM-022 for patients with wet AMD. In the OPTIC trial, the DMC reviewed the safety data and unanimously agreed that we could proceed to dosing the second cohort. No patient has experienced an SAE, with a follow up period of up to five months. We look forward to sharing 24-week primary and secondary outcomes from the first cohort of patients at a scientific meeting in the second half of this year.”
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