After rancorous threats and a clinical hold, Applied Therapeutics is set to resume pediatric galactosemia study
After an anonymous report raised red flags over Applied Therapeutics’ integrity in July, saying the company “cherry picked” data and ostensibly prompting a short attack, the biotech accused the authors of the report of fraud and threatened legal action. Then, a month later, the FDA indicated it also had concerns over its lead galactosemia program, slapping a clinical hold on a study testing their candidate in children.
But on Monday, regulators handed down some good news and lifted the hold on the trial, Applied announced. The study is expected to resume “effective immediately,” the company said in a statement.
At the time the hold was placed, Applied offered few clues as to why the FDA pressed the pause button. On Monday, the company said that execs met with the agency to modify the trial in a way that ensures all patients receive a “clinical benefit.”
The program had previously consisted of two separate trials — a dose escalation and biomarker study, followed by a separate long-term clinical outcomes study. Those parts have now been combined into a single, two-part trial that allows each patient in the dose escalation portion to continue into the long-term portion without interrupting their treatments.
Everyone who was already randomized and participating in the study will be eligible to return, and Applied says they’ll be fully enrolled “within a matter of weeks.”
Though last summer’s hold did not affect the patients from the pivotal trial on adults, it pushed back the timeline for a potential approval. Applied had originally shot for an NDA in the second half of 2020, but at the time of the hold pushed back that goal to the first quarter of 2021.
With Monday’s news, Applied $APLT said it plans to submit an NDA in the third quarter of this year. Shares rose 21% in Monday trading.
Applied’s program, dubbed AT-007, inhibits the enzyme aldose reductase and can cross the blood brain barrier to enter the central nervous system, the company says. Patients with galactosemia have trouble breaking down the sugar galactose in their bloodstreams and can suffer from toxic waste that builds up in tissues and organs that cause long-term complications.
In January of last year, they unveiled pivotal data that AT-007 reduced galactose by around 50% when compared to placebo. But the short attack hit the company in July after the full results came out. Among the accusations Applied made included “fabricated graphs and fraudulent data” aimed at manipulating the company’s stock price.
Such attacks are not uncommon in biotech and companies routinely shake them off, but Applied raised more red flags with the actions it took following the report.