This morning, New York City-based clinical-stage biopharmaceutical company Applied Therapeutics Inc. (APLT:NASDAQ) announced "positive topline results from the Pivotal Phase 2 portion of the ACTION-Galactosemia study of AT-007, a central nervous system (CNS) penetrant Aldose Reductase inhibitor, in adult Galactosemia patients."
The company explained in the report that "Galactosemia is a rare metabolic disease that affects how the body processes a simple sugar called galactose, and for which there is no known cure or approved treatment available."
The firm stated that the ACTION study is a double-blind trial evaluating the effects of AT-007 in healthy adult Galactosemia patients, and noted that the "key biomarker outcome of the study was reduction in galactitol, an aberrant toxic metabolite of galactose, formed by Aldose Reductase in Galactosemia patients."
According to the report, the data from the study showed that the AT-007 treatment resulted in a statistically significant and robust reduction in plasma galactitol versus placebo in adult Galactosemia patients and that reductions in galactitol were dose dependent. The company indicated that AT-007 was well tolerated, with no drug-related adverse events reported in the 72 healthy volunteers treated in the first part of the trial or in Galactosemia diagnosed patients. Applied Therapeutics believes that AT-007 represents a potentially compelling new therapeutic option for patients with Galactosemia and stated that it expects to file for regulatory approval for the drug in H2/20.
The company's Chief Medical Officer Riccardo Perfetti, MD, PhD, commented, "We are thrilled with these results...Galactosemia is a devastating disease with no treatments currently available. We have long known that dietary restriction alone does not prevent chronic complications of disease. These results provide hope for patients and families that action through drug treatment with AT-007 can potentially change the course of the disease, transforming patients' lives."
Shoshana Shendelman, PhD, founder, CEO and chair of the Board of Applied Therapeutics, added, "The Galactosemia program is an example of our overall strategy to apply technological breakthroughs to areas of high unmet need...By employing new regulatory pathways and using biomarkers early in development, we are able to bring critical drugs to patients who desperately need them quickly and effectively. The ACTION-Galactosemia data presented today is our first pivotal study readout - but it's just the beginning. We are continuing to advance our pipeline of novel drug candidates in other disease indications and look forward to sharing additional successful data readouts in the future."
Applied Therapeutics is headquartered in New York, N.Y., and describes its business as "a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need." In addition to AT-007, the company is developing another lead drug candidate, AT-001, a novel aldose reductase inhibitor that is being developed for the treatment of Diabetic Cardiomyopathy, a fatal fibrosis of the heart.
Applied Therapeutics has a market cap of approximately $484 million with about 18.52 million outstanding shares. APLT shares opened more than 20% higher today at $31.75 (+$5.59, +21.37%) over yesterday's closing price of $6.16 and the company's stock achieved a new 52-week high price this morning of $40.141. The stock has traded today between $31.00 and $40.141 per share and is presently trading at $33.00 (+$6.84, +26.15%).
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