Aeterna Zentaris (TSX: AEZ, Stock Forum) suffered a major setback in its commercialization of Macrilen™, a novel orally-active ghrelin agonist for use in evaluating adult growth hormone deficiency (“AGHD”) when the company announced receiving the Complete Response Letter (“CRL”) from the U.S. Food and Drug Admisitration (“FDA”) stating that the agency had determined that the New Drug Application (“NDA”) for Macrilen cannot be approved in its present form.
According to the news release, the FDA stated in the CRL that the planned analysis of the company's pivotal trial did not meet its stated primary efficacy objective as agreed to in the Special Protocol Assessment agreement letter between the company and the FDA.
There were also further issues regarding the lack of complete and verifiable source data for determining whether patients were accurately diagnosed with AGHD.
The CRL finished up by stating, “in light of the failed primary analysis and data deficiencies noted, the clinical trial does not by itself support the indication.”
The company will need to demonstrate the efficacy of macimorelin as a diagnostic test for AGHD in a new, confirmatory clinical study in order for the FDA to give the drug a second look.
The CRL also stated that there was a serious event of electrocardiogram QT interval prolongation for which attribution to the drug could not be excluded and therefore a dedicated thorough QT study was required to evaluate the effect of macimorelin on the QT interval.
Investors weren't the least bit pleased with the news and the company's share price took a kick in the teeth. While some longs suggested that a chance remained the company could address the FDA's concerns, others weren't so hopeful.
Commercialization of drugs is a heavily regulated process and rightfully so. As a result the journey from lab to shelf can be a gamble even in the best of scenarios that can not only stymie the drugs eventual release, but holds the possibility of submarining the company.
Aeterna has more cards in the deck, but a majority rest in the preclinical and Phase 2 stages with only one contender at Stage 3. Macrilen was their NDA pony. It will be interesting to see how this setback plays out in the coming months.
Aeterna was in the news recently when the Quebec City-based company announced its Q3 2014 results a day ago.
Shares plummeted 46.58% on the news to $0.78 per share.
Currently there are 63.9m outstanding shares with a market cap of $49.8 million.