Shares of clinical-stage biopharmaceutical company, Applied Therapeutics Inc. APLT, surged 34.48% on the announcement of positive top-line results from the mid-stage study in adult galactosemia patients on pipeline candidate, AT-007.
ACTION-Galactosemia is a double-blind, placebo-controlled trial, evaluating the safety and pharmacokinetics of AT-007 in healthy volunteers. The study also evaluated the safety, pharmacokinetics and biomarker effects of the candidate in adult Galactosemia patients over 28 days of once-daily oral dosing.
AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI), being developed for the treatment of galactosemia, which is a rare metabolic disease. The key biomarker outcome of the study was a reduction in galactitol.
The top-line results from the phase II portion of the ACTION-Galactosemia study showed that treatment with AT-007 resulted in a statistically significant and robust reduction in plasma galactitol versus placebo in adult galactosemia patients. Moreover, reductions in galactitol were dose dependent, with higher concentrations of AT-007 resulting in a greater magnitude of reduction in galactitol. The candidate was well tolerated, with no drug-related adverse events noted to date in galactosemia patients or the 72 healthy volunteers treated in part 1 of the trial.
Applied Therapeutics will continue to evaluate AT-007’s long-term safety in adult galactosemia patients. The company also plans to initiate a pediatric study in 2020.
We remind investors that Applied Therapeutics is conducting a biomarker-based development program in patients with galactosemia, based on the recently released draft industry guidance on drug development for low prevalence, slowly progressing rare metabolic diseases.
Based on the encouraging results, Applied Therapeutics expects to file for regulatory approval of AT-007 in the second half of 2020. The successful development of the candidate will significantly boost the company’s performance in the long haul.
Shares of the company have skyrocketed 260.8% in the past year compared with the industry’s 2.9% growth.
We note that Applied Therapeutics obtained Orphan Designation for AT-007 in galactosemia in May 2019.
Apart from AT-007, the company has other candidates in its pipeline. Its lead drug candidate, AT-001, is a novel ARI being developed for the treatment of diabetic cardiomyopathy, a fatal fibrosis of the heart. The preclinical pipeline also includes AT-003 for the treatment of diabetic retinopathy. The candidate is expected to advance into a phase I study this year.
Zacks Rank & Stocks to Consider
Applied Therapeutics currently carries a Zacks Rank #4 (Sell). A few better-ranked stocks in the biotech space are Amarin Corp. AMRN, Epizyme, Inc. EPZM and Celsion Corp. CLSN. All three stocks carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Earnings per share estimates for Amarin have increased 3 cents for 2020 in the past 30 days.
Loss per share estimates for Epizyme have narrowed 2 cents for 2020 in the past 60 days.
Loss per share estimates for Celsion have narrowed 11 cents for 2020 in the past 60 days.
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