Applied Therapeutics Reports Initial Pediatric Biomarker Data from ACTION-Galactosemia Kids

Rhea-AI Summary

Applied Therapeutics (Nasdaq: APLT) announced significant progress in its pediatric ACTION-Galactosemia Kids study, showing a 35% reduction in plasma galactitol after 30 days of treatment with AT-007, meeting statistical significance (p<0.001) compared to placebo. The drug was well-tolerated among children aged 2-17. The company is adjusting dosing based on weight and plans to submit a New Drug Application to the FDA in Q4 2021. A baseline analysis revealed a correlation between plasma galactitol levels and disease severity in patients, marking a milestone in understanding Galactosemia.

Positive

• 35% reduction in plasma galactitol after 30 days of AT-007 treatment.
• Statistically significant results (p<0.001) compared to placebo.
• Safety and tolerability of AT-007 confirmed in children aged 2-17.
• Implementation of weight-based dosing for improved treatment optimization.
• Plans for New Drug Application submission to the FDA in Q4 2021.

Negative

• Delay in NDA submission due to adjustments in the study.

Substantial and statistically significant reduction in plasma galactitol

Baseline analysis demonstrates clear relationship between plasma galactitol level and severity of disease in pediatric Galactosemia patients

NEW YORK, Sept. 09, 2021 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today reported initial biomarker data from the pediatric ACTION-Galactosemia Kids study, as well as baseline analysis of the 47 children enrolled in the study.

The results demonstrate a substantial reduction in plasma galactitol of approximately 35% after 30 days of treatment, which was statistically significant (p<0.001) vs. placebo.  AT-007 was safe and well tolerated in children of all ages (2-17).  A pharmacokinetic analysis of AT-007 plasma drug levels at day 30 revealed that dosing could be further optimized by adjusting dose based on weight rather than age.  As such, weight-based dosing brackets have been implemented for the remainder of the study, and a subset of patients have been dose-adjusted. The Company expects to present additional biomarker data at the optimized drug levels pending analysis at day 30.  Due to the time required for these study changes, the Company now plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in Q4 of this year, and has adjusted projected timelines accordingly.

Additionally, analysis of the 47 children in the ACTION-Galactosemia Kids study demonstrated a clear correlation between baseline galactitol level and baseline clinical functional outcomes. Children with higher plasma galactitol levels displayed greater disease severity vs. children with lower plasma galactitol levels at baseline. This data is the first demonstration of correlation of a biochemical biomarker with severity of disease in Galactosemia patients. Full data will be presented at a future medical conference.

“Reduction in toxic galactitol is critically important to patients with Galactosemia,” said Shoshana Shendelman, PhD, CEO, Founder and Chair of the Board of Applied Therapeutics. “AT-007 has previously demonstrated significant reduction in galactitol in adults with Galactosemia. Now, we have demonstrated a significant reduction in children with this devastating rare disease. As the first interventional clinical study in pediatric Galactosemia patients, ACTION-Galactosemia Kids has already provided important insight into disease severity and progression. This new data demonstrating correlation of galactitol level with severity of disease underscores the importance of reducing this toxic metabolite in patients with Galactosemia.”

About Applied Therapeutics

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-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. 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, as well as novel dual PI3k inhibitors in preclinical development for orphan oncology indications.

To learn more, please visit www.appliedtherapeutics.com and follow the company on Twitter @Applied_Tx.

Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, included in this press release regarding strategy, future operations, prospects, plans and objectives of management, including words such as “may,” “will,” “expect,” “anticipate,” “plan,” “intend,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. These include, without limitation, statements regarding (i) the timing of our NDA submission for potential approval of AT-007, which will include data from the ACTION-Galactosemia Kids trial and the 90-day safety data in adults with Galactosemia, (ii) the timing of our rare disease franchise expansion programs in SORD Deficiency, (iii) the timing of the initiation and completion of our clinical trials, including ARISE-HF, (iv) the likelihood that data from our clinical trials will support future development of our product candidates and (v) the likelihood of obtaining regulatory approval of our product candidates. Forward-looking statements in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved.

Such risks and uncertainties include, without limitation, (i) our plans to develop and commercialize our product candidates, (ii) the initiation, timing, progress and results of our current and future preclinical studies and clinical trials and our research and development programs, (iii) our ability to take advantage of expedited regulatory pathways for any of our product candidates, (iv) our estimates regarding expenses, future revenue, capital requirements and needs for additional financing, (v) our ability to successfully acquire or license additional product candidates on reasonable terms, (vi) our ability to maintain and establish collaborations or obtain additional funding, (vii) our ability to obtain regulatory approval of our current and future product candidates, (viii) our expectations regarding the potential market size and the rate and degree of market acceptance of such product candidates, (ix) our ability to fund our working capital requirements and expectations regarding the sufficiency of our capital resources, (x) the implementation of our business model and strategic plans for our business and product candidates, (xi) our intellectual property position and the duration of our patent rights, (xii) developments or disputes concerning our intellectual property or other proprietary rights, (xiii) our expectations regarding government and third-party payor coverage and reimbursement, (xiv) our ability to compete in the markets we serve, (xv) the impact of government laws and regulations and liabilities thereunder, (xvi) developments relating to our competitors and our industry, (xvii) the impact of the COVID-19 pandemic on the timing and progress of our ongoing clinical trials and our business in general and (xviii) other factors that may impact our financial results. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. Although we believe that we have a reasonable basis for each forward-looking statement contained in this press release, we cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur at all. Factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in our filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” contained therein. Except as otherwise required by law, we disclaim any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.

Contacts

Investors:
Maghan Meyers
(212) 600-1902 or
appliedtherapeutics@argotpartners.com

Media:
media@appliedtherapeutics.com

Applied Therapeutics, Inc.

FAQ

What were the results of the ACTION-Galactosemia Kids study for APLT?

The study showed a 35% reduction in plasma galactitol after 30 days of treatment with AT-007, which was statistically significant.

When does Applied Therapeutics plan to submit the New Drug Application for AT-007?

The company plans to submit the NDA to the FDA in Q4 2021.

How was the safety of AT-007 evaluated in the pediatric study?

AT-007 was found to be safe and well tolerated in children aged 2 to 17.

What is the correlation found in the ACTION-Galactosemia Kids study?

The study demonstrated a clear correlation between baseline plasma galactitol levels and disease severity in pediatric patients.

What is the significance of the 35% reduction in galactitol levels?

This reduction is crucial as it indicates a potential therapeutic benefit for patients with Galactosemia.