The FDA has hit Applied Therapeutics with a partial clinical hold for its experimental galactosemia drug AT-007.
This comes around six weeks after a short report attacking the New York biotech floated onto Twitter and through a blog from Biotech Research Partners. It wasn’t as well covered as some of these reports but apparently still did a number on Applied Therapeutics, which saw its shares drop around 6% in the build-up to the July Fourth weekend.
The report questioned the biotech’s trial data and “integrity” issues from one of its latest studies, questioning whether it can really gain approval and alleging the company is cutting corners. It specifically called out AT-007.
Humanized Mouse Models for Drug Discovery: The NOG Portfolio
Human immune system mouse models are leading to breakthroughs in a wide range of research applications. In this white paper, explore the NOG portfolio and the unique benefits of each model to determine the appropriate choice for your study.
“In our opinion, the company is doing more than just leveraging a permissive FDA—we think they are cutting corners.” It said one of its main concerns is that the current trial size is “substantially smaller than originally planned” and that a patient was enrolled twice in the same trial.
Seeing the report, and the fallout for its shares, Applied hit back, saying it had “uncovered fraudulent attempts to manipulate the Applied Therapeutics stock,” hitting back hard on the attack.
But now the FDA has taken aim at the drug, hitting it with a partial hold on its registrational trial in children, which started in mid-June. The FDA is demanding “additional technical information relating to ensuring that every patient in the study has access to the prospect of direct benefit of the drug,” the biotech said in a SEC filing (but not a press release).
It did not go into further detail, except to say that the hold “does not relate to concerns regarding the safety profile of AT-007.” It plans to submit the supporting technical information “promptly,” and hopes for a resolution within 30 days.
This will not affect its adult part of the trial, known as the ACTION-Galactosemia study, which is in the long-term extension phase. But because of this, it now expects to file for an NDA for the drug in the first quarter of next year, after initially guiding it for the second half of this year.
The biotech was down 16% Monday on the news.
Applied has been working on a series of drugs that target aldose reductase, an enzyme that converts glucose into sorbitol, a sugar alcohol involved in multiple diseases including complications of diabetes.
Previous efforts to block aldose reductase have had limited success as they have not been selective or specific enough, but in its SEC-1 filing for its IPO last year, the company said it designed its drugs to overcome these challenges.
Its lead program, AT-001, in diabetic cardiomyopathy has been in a mid- to late-stage study (and is also now being used in critical COVID-19 patients), with its second being AT-007.
Its target, galactosemia, is a rare genetic disorder that affects how the body processes galactose, a simple sugar.
Galactose is produced in the body at low levels and, along with glucose, makes up lactose, found in milk and dairy products. There are a few types of galactosemia, which differ in severity and symptoms. But Type 1, the most severe kind, can be fatal if not caught early in life.