- Applied Therapeutics, Inc. (APLT) **completed a meeting with the U.S. Food and Drug Administration (FDA)** in the third quarter of 2025 to discuss the potential New Drug Application (NDA) submission for **govorestat** for the treatment of Charcot-Marie-Tooth Sorbitol Dehydrogenase (SORD) Deficiency (CMT-SORD).  **[1697532_1a9c3aa446384f3b9a939cfed1736949_0]** 
- The company is **awaiting official meeting minutes** from the FDA to determine the next steps regarding a potential submission strategy for regulatory approval.  **[1697532_1a9c3aa446384f3b9a939cfed1736949_0]** 
- At this time, a definitive path forward, including under the accelerated approval pathway, **has not yet been determined**.  **[1697532_1a9c3aa446384f3b9a939cfed1736949_0]** 
- Govorestat has received **Orphan Drug Designation** from the FDA for CMT-SORD, a rare disease affecting approximately **3,300 patients in the U.S.** and **4,000 patients in the EU**.  **[1697532_1a9c3aa446384f3b9a939cfed1736949_0]**

Applied Therapeutics Provides Update on Govorestat FDA Meeting

- ABLi Therapeutics will present final results from its **201 Trial** evaluating **risvodetinib** in untreated Parkinson's disease (PD) as a "Clinical Breakthrough Lecture" on **October 8, 2025**, at the International Congress of Parkinson's Disease and Movement Disorders.  **[1697532_248d3309cb27449dbfcfb0018db8a2ad_0]** 
- The 201 Trial, which involved **126 participants**, was the first long-term dosing trial of risvodetinib as a monotherapy in untreated PD, and the presentation will cover primary, secondary, and exploratory endpoint analysis, including the effect on underlying PD pathology.  **[1697532_248d3309cb27449dbfcfb0018db8a2ad_0]** 
- Risvodetinib is described as a **disease-modifying therapy** designed to halt disease progression and reverse functional loss in Parkinson's disease, with intellectual property protection extending beyond **2036**.  **[1697532_248d3309cb27449dbfcfb0018db8a2ad_1]** 
- It was recently the **first monotherapy** shown to improve patient quality of life and reduce underlying synuclein aggregate pathology in a randomized, placebo-controlled clinical trial.  **[1697532_248d3309cb27449dbfcfb0018db8a2ad_1]**

ABLi Therapeutics to Present Final Risvodetinib Trial Results

- Applied Therapeutics, Inc. entered into a **Controlled Equity OfferingSM Sales Agreement** with **Cantor Fitzgerald & Co.** on **August 29, 2025**.  **[1697532_0000950170-25-112224_aplt-ex10_1.htm:0]** **[1697532_0000950170-25-112224_aplt-20250829.htm:1]** 
- This agreement allows the company to offer and sell up to **$75,000,000** in common stock through "at the market" offerings.  **[1697532_0000950170-25-112224_aplt-20250829.htm:1]** 
- Cantor Fitzgerald & Co. will receive a commission of **3.0%** of the aggregate gross proceeds from any shares sold.  **[1697532_0000950170-25-112224_aplt-20250829.htm:1]** **[1697532_0000950170-25-112224_aplt-ex10_1.htm:47]** 
- Concurrently, the company terminated its previous sales agreement with Leerink Partners LLC, effective **August 29, 2025**.  **[1697532_0000950170-25-112224_aplt-20250829.htm:1]** 
- Under the terminated Leerink Partners Sales Agreement, from **August 11, 2023**, to **August 29, 2025**, Applied Therapeutics, Inc. sold **20,615,976 shares** for net proceeds of approximately **$49.3 million**.  **[1697532_0000950170-25-112224_aplt-20250829.htm:2]**

Applied Therapeutics (APLT)