What Applied Therapeutics does

Applied Therapeutics, Inc. was a clinical-stage biopharmaceutical company that developed govorestat (AT-007), a next-generation, CNS-penetrant, highly selective Aldose Reductase Inhibitor, targeting rare metabolic and neurological diseases with no FDA-approved treatment options — specifically Classic Galactosemia, Sorbitol Dehydrogenase (SORD) Deficiency, and Phosphomannomutase 2 Deficiency (PMM2-CDG). The company pursued regulatory designations that would accelerate development, including FDA Orphan Drug status across all three indications, EMA Orphan Medicinal Product Designations for Galactosemia and SORD Deficiency, Pediatric Rare Disease designations, and FDA Fast Track designation for Galactosemia. The technology platform centered on applying selective ARI pharmacology to CNS-penetrant oral therapy, supported by Phase 3 readouts in the ACTION-Galactosemia Kids study and the INSPIRE trial in SORD Deficiency.

The company was pre-revenue and operated as a clinical development organization, sustaining operations through repeated equity raises totaling more than $400 million cumulatively across private and public rounds, including a $100 million PIPE in February 2024. In November 2024, the FDA issued a Complete Response Letter for the Galactosemia NDA, materially impairing the lead commercial pathway. This was followed by a 46% workforce reduction in November 2025 and acquisition by Cycle Group Holdings Limited, completed in February 2026 at approximately $14.3 million total consideration ($0.088 per share plus contingent value rights). Applied Therapeutics is now an indirect wholly owned subsidiary of Cycle Group Holdings, delisted from Nasdaq. In April 2026, the company announced discontinuation of all active Applied-sponsored govorestat clinical studies and ended the Expanded Access Program in order to re-evaluate development strategy under new ownership.

Name

Applied Therapeutics

Legal name

Applied Therapeutics, Inc.

Website

https://appliedtherapeutics.com

Company type

Private

Founded year

2016

Operating status

Acquired

Headcount range

11–50 employees

Short description

Applied Therapeutics is a clinical-stage biopharmaceutical company that develops govorestat, a CNS-penetrant Aldose Reductase Inhibitor, for rare metabolic and neurological diseases including Classic Galactosemia, SORD Deficiency, and PMM2-CDG; acquired by Cycle Group Holdings in February 2026.

Ownership category

akta.pro rank

Product category

Rare Disease Biopharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN)

akta.pro secondary industries

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA), Rare Cardiovascular & Vascular Disorder Therapies (HLAIAIAH)

Keywords

• Rare disease therapeutics
• Aldose reductase inhibitors
• Clinical-stage biopharmaceuticals
• Metabolic disease treatments
• Neurological disease drugs

Headquarters

HQ city

New York

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Others

Revenue model

1. Pharmaceutical Product Sales: Pre-revenue clinical-stage biopharmaceutical company. Revenue would be generated through sales of govorestat and other pipeline products upon regulatory approval. The company was acquired by Cycle Group Holdings in February 2026.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels3 records

Core offering

Applied Therapeutics is a clinical-stage biopharmaceutical company developing govorestat (AT-007), a CNS-penetrant next-generation Aldose Reductase Inhibitor (ARI) for the treatment of rare metabolic and neurological diseases including Classic Galactosemia, SORD Deficiency, and PMM2-CDG. The company leverages expedited regulatory pathways including Orphan Drug, Fast Track, and Pediatric Rare Disease designations to advance its pipeline.

Product overview

Applied Therapeutics is a clinical-stage biopharmaceutical company developing treatments for rare metabolic and neurological diseases. The company's primary product is govorestat (AT-007), a CNS-penetrant Aldose Reductase Inhibitor targeting multiple rare conditions including Classic Galactosemia, CMT-SORD, and PMM2-CDG. The company leverages technological advances to target molecules and pathways with proven roles in disease biology, using expedited regulatory pathways where possible. Following acquisition by Cycle Group Holdings Limited in February 2026, Applied Therapeutics operates as a wholly owned subsidiary focused on advancing the govorestat pipeline.

Differentiator

Problem solved

Functional benefit

Products and services

• Govorestat (AT-007) Govorestat (AT-007) is a next-generation, potent and selective oral Aldose Reductase Inhibitor (ARI) being developed for the treatment of multiple rare diseases including Classic Galactosemia, Sorbitol Dehydrogenase (SORD) Deficiency, and Phosphomannomutase 2 Deficiency (PMM2-CDG). It is CNS-penetrant with a highly selective mechanism of action that avoids the off-target effects of historical ARIs, and has received Orphan Drug designation from FDA and EMA for multiple indications.

Quantifiable outcome

• Phase 3 ACTION-Galactosemia Kids study demonstrated clinical benefit on activities of daily living, behavioral symptoms, cognition, fine motor skills and tremor in pediatric patients aged 2-17
• +2 more outcomes

Named customers3 records

Segments1 record

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Scale indicators7 records

Recent moves6 records

Expansion highlights3 records

Direct peers

• Cycle Pharmaceuticals: Specialty rare disease pharmaceutical company and now the parent of Applied Therapeutics; directly comparable as a rare-disease-focused commercial-stage company targeting similar patient populations and distribution channels.
• Ultragenyx Pharmaceutical: Clinical-to-commercial rare disease biotech with multiple orphan programs in metabolic and neurological diseases; closely comparable business model of targeting rare genetic disorders with high unmet need.
• BioMarin Pharmaceutical: Rare disease biopharma with deep pediatric and inherited metabolic disease franchise; directly comparable in commercial strategy for ultra-rare enzyme and metabolic disorder therapeutics.
• Recordati Rare Diseases: Specialty rare disease unit of Recordati focused on metabolic and neurological rare disorders; comparable in go-to-market model for ultra-rare pediatric and metabolic disease therapies.
• Travere Therapeutics: Rare disease biopharma developing therapies for serious rare metabolic and neurological conditions; comparable in business model and patient population targeting.
• Sarepta Therapeutics: Genetic rare disease company focused on neuromuscular and inherited disorders; comparable in CNS-penetrant rare disease drug development and orphan regulatory strategy.

Broad incumbents

• Alexion (AstraZeneca Rare Disease): Established rare disease commercial leader (now part of AstraZeneca) with multiple orphan drugs in metabolic, neurological, and genetic diseases; comparable in commercial model and patient access infrastructure.
• Genzyme (Sanofi): Pioneering rare disease commercial franchise within Sanofi; comparable in rare metabolic disease commercialization, specialty distribution, and orphan drug lifecycle management.

Emerging players

• BridgeBio Pharma: Genetic disease-focused biopharma with pipeline assets in rare metabolic and neurological conditions; comparable in targeting genetically defined rare diseases with high unmet need.
• Taysha Gene Therapies: Clinical-stage rare disease company developing CNS-penetrant therapies for genetic neurological disorders; comparable in CNS-targeted rare disease drug development approach.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat3 records

Key risks6 records

Key highlights5 records

Customer concentration

• Xx.com/applied_tx

Revenue estimate

Valuation estimate

Number of profiles

Profiles2 records

Funding overview

Funding rounds8 records

Investors13 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Applied Therapeutics