NEW YORK, Aug. 08, 2019 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (APLT), a clinical-stage biopharmaceutical company developing novel drug candidates in indications of high unmet medical need, today announced the completion of the Single Ascending Dose (SAD) healthy volunteer portion of the Phase 1/2 study of AT-007 in Galactosemia (ACTION-Galactosemia).
The results show that AT-007 was well tolerated, with no drug-related adverse events or dose-limiting toxicities reported. The SAD study treated 4 cohorts of 8 subjects each and explored doses from 0.5mg/kg to 20mg/kg. In addition to safety, AT-007 demonstrated a linear pharmacokinetic (PK) profile, favorable exposure, and half-life consistent with once-daily dosing.
“We are encouraged by the favorable safety profile in healthy volunteers, and we look forward to advancing AT-007 through the MAD study in healthy volunteers and to the Phase 2 portion of the study in patients with Galactosemia,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “Developing drugs for high unmet indications, such as Galactosemia, is critical to our mission at Applied, and we are committed to developing a potential new therapy for patients with no available treatment options.”
AT-007 will advance in parallel to a Phase 1b Multiple Ascending Dose (MAD) study in healthy volunteers (up to 7 consecutive days of treatment), and a Phase 2 study in adults with Galactosemia. Galactosemia patients will be eligible for treatment up to 28 days total (single dose followed by 27 consecutive days of dosing). In addition to safety and PK, the study will determine the ability of AT-007 to suppress toxic accumulation of galactitol in Galactosemia patients. Applied Therapeutics plans to employ recent FDA guidance permitting biomarker-based development in low prevalence, slowly progressing rare metabolic diseases, such as Galactosemia.
“AT-007 marks our second program to move through the clinic, and we are excited to see additional data in support of our technology and development strategy,” said Shoshana Shendelman, PhD, Founder and Chief Executive Officer of Applied Therapeutics. “We look forward to presenting additional data on our Galactosemia program at the American Society for Human Genetics conference this fall.”
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. Galactose is found in foods, but the human body also naturally produces galactose on its own, so dietary restriction can’t prevent complications of disease. It is estimated that the U.S. Galactosemia population is approximately 2,800 patients, based on newborn screening data identifying 2,500 infants through 2014, and the estimated birth rate of 80 patients per year. High levels of galactose circulating in the blood and tissues of Galactosemia patients enables Aldose Reductase to convert galactose to a toxic metabolite, called galactitol, which causes long-term complications ranging from CNS dysfunction to cataracts.
AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in Phase 1/2 development for treatment of Galactosemia. AT-007 has been studied in in an animal model of Galactosemia, which demonstrated that AT-007 reduces toxic galactitol levels and prevents disease complications. Applied Therapeutics is planning 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. The company received Orphan Designation for AT-007 for Galactosemia in May 2019.
About Applied Therapeutics Inc.
Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The company’s lead drug candidate, AT-001, is a novel aldose reductase inhibitor (ARI) that is being developed for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The company plans to initiate a Phase 3 registrational study in DbCM in 2019. Applied Therapeutics is also developing AT-007, a central nervous system penetrant ARI, for the treatment of Galactosemia, a rare pediatric metabolic disease, and initiated a Phase 1/2 clinical trial in June 2019. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of diabetic retinopathy, expected to advance into a Phase 1 study in 2020.
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