The first Trial Watch: the CMT drug trials under way or changed since 2024, from a Novartis CMT1A study recruiting only in Canada to a cancelled govorestat trial, with a plain-English note on each.

After extending its review period to evaluate additional submissions, the FDA ultimately denied Applied Therapeutics’ govorestat for galactosemia, citing “deficiencies” with the application. The biote...

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RBC downgrades Applied Therapeutics as FDA rejects Govorestat, hacks price target

Cycle Pharmaceuticals has completed its acquisition of Applied Therapeutics (NASDAQ: APLD), strengthening Cycle’s position in rare metabolic and neurological

Investors of Applied Therapeutics, Inc. are urged to join a class action lawsuit for alleged securities law violations during the trial period of their drug candidate govorestat.

The FDA's denial of Govorestat, the sole potential treatment for Classic Galactosemia, leaves patients and families in despair. Advocacy continues for their reconsideration.

Applied Therapeutics (Nasdaq: APLT) announced that its Board has initiated a process to explore strategic alternatives including mergers, acquisitions, partnerships, licensing or other transactions to maximize shareholder value.The Company said it reduced its workforce by approximately 46% and is implementing other cost‑containment and cash conservation measures to preserve runway. The Board completed a Type C meeting with the FDA on govorestat for CMT‑SORD in Q3 2025 and expects to request an additional Type C to discuss a potential Phase 3 design. The Company also accepted FDA written feedback on govorestat data for Classic Galactosemia and is evaluating next steps. No timetable or assurance of a transaction was provided.

– Update on regulatory strategy for govorestat for the treatment of CMT-SORD following receipt of Type C meeting minutes – Meeting scheduled with the FDA in 4Q 2025 to discuss govorestat for the treatment of Classic Galactosemia – New data on govorestat for the treatment of PMM2-CDG published

Applied Therapeutics (NASDAQ:APLT) reported Q2 2025 financial results and significant progress across its rare disease pipeline. The company's lead program, govorestat for CMT-SORD treatment, advances with an upcoming FDA meeting in Q3 2025 to discuss potential NDA submission. The company presented promising 24-month clinical data showing slowed disease progression and launched a sponsored Sorbitol Assay program for patient identification.Financial highlights include cash position of $30.4M as of June 30, 2025, down from $79.4M in December 2024. Q2 2025 resulted in a net loss of $21.3M ($0.15 per share). The company secured an out-licensing agreement with Biossil for AT-001 and strengthened its leadership team with key promotions.

Applied Therapeutics (NASDAQ: APLT) has announced key leadership changes with the promotion of Dr. Evan Bailey to Chief Medical Officer (CMO) and Dottie Caplan to Executive Vice President of Patient Advocacy and Government Affairs. Dr. Bailey, previously SVP of Clinical Development, succeeds Dr. Riccardo Perfetti and will lead clinical development and medical affairs activities for govorestat in late-stage development programs. Dr. Bailey brings extensive experience from Vertex Pharmaceuticals, where he led the TRIKAFTA launch for Cystic Fibrosis. Dottie Caplan, formerly SVP of Patient Advocacy and Engagement, will expand her role to include regulatory, legislative, and public policy functions. Both appointments are strategic moves to advance Applied's rare disease treatment portfolio, particularly focusing on govorestat's development for Classic Galactosemia, CMT-SORD, and PMM2-CDG.

Net loss improves 74% YoY as Applied Therapeutics prepares to unveil crucial Phase 3 trial data for rare disease drug govorestat. See full financial details.

Applied Therapeutics (NASDAQ: APLT) announced that full 12-month clinical data from the INSPIRE Phase 3 trial of govorestat (AT-007) has been accepted for a late-breaking oral presentation at the Peripheral Nerve Society (PNS) 2025 Annual Meeting in Edinburgh, Scotland. The presentation will also include new topline 18-month and 24-month data from patients before their transition to the open-label extension study.The INSPIRE Phase 3 trial was a double-blind, placebo-controlled registrational study evaluating govorestat's effect in patients with Sorbitol Dehydrogenase (SORD) Deficiency, a subtype of Charcot-Marie-Tooth (CMT) disease. The company previously released topline results from the 12-month group-level data analysis in February 2024. ["-"]

Latest earnings reveal stronger cash position and reduced losses, while new leadership team tackles FDA response for govorestat program. Full analysis inside.

Applied Therapeutics faces setback as FDA issues Complete Response Letter for govorestat, citing clinical deficiencies. Company plans to discuss resubmission while advancing SORD program.

Explore FDA flexibility in medical care as a mother shares her daughter's inspiring journey through health challenges.

The US Food and Drug Administration has declined to approve Applied Therapeutics’ govorestat for classic galactosemia.