What Genoscience Pharma does

Genoscience Pharma is a privately held, clinical-stage biopharmaceutical company headquartered in Marseille, France (10 Rue d'Iéna, 13006 Marseille) that develops lysosomotropic small-molecule drug candidates targeting lysosomal functions implicated in cancer growth, spread, and resistance. The company was founded by Prof. Philippe Halfon and originated in antiviral drug development (initially against Hepatitis C, including the BL-8020 and BL-8030 programs out-licensed to BioLineRx in 2012), before pivoting to oncology. Its lead asset, GNS561/ezurpimtrostat, is a first-in-class orally bioavailable inhibitor of palmitoyl-protein thioesterase 1 (PPT1) that disrupts late-stage autophagy and induces lysosomal cell death; the recommended Phase 2 dose has been established at 200 mg twice daily with a favorable safety profile (no DLT observed across 26 evaluable patients) and a striking liver-to-plasma concentration ratio of approximately 9,559. The pipeline focuses on primary liver cancers—hepatocellular carcinoma (HCC) and intrahepatic cholangiocarcinoma (iCCA)—as well as secondary liver cancers (pancreatic and colorectal cancer with liver metastasis), and an additional idiopathic pulmonary fibrosis program has been added via PROTAC technology.

The company makes money primarily through out-licensing and milestone/royalty arrangements rather than direct product sales. The pivotal commercial vehicle is the April 2025 exclusive global licensing deal with Genfit covering GNS561 for all oncology indications, which layers on top of an earlier December 2021 cholangiocarcinoma license to Genfit covering the US, Canada, and Europe. Secondary revenue comes from non-dilutive French and European public funding: grants and refundable advances from BPIFrance and the Provence-Alpes-Côte d'Azur region, the French government's Programme d'Investissements d'Avenirs, and the European Commission's FEDER program (including the 2025 Gento-Fibrose Pulmonaire project). Genoscience has secured FDA Orphan Drug Designation for ezurpimtrostat in both HCC (February 2023) and cholangiocarcinoma (September 2022, to Genfit), operates a Phase 2b ABE-LIVER trial in first-line HCC in combination with atezolizumab/bevacizumab, and conducts clinical operations across France, Belgium (Jules Bordet Institute), and the United States (Memorial Sloan Kettering). The company holds ISO 9001:2015 certification for its R&D and clinical activities.

Name

Genoscience Pharma

Legal name

Genoscience Pharma

Website

https://genosciencepharma.com

Company type

Private

Founded year

2007

Operating status

Operating

Headcount range

11–50 employees

Short description

Genoscience Pharma is a privately held, clinical-stage French biotech developing first-in-class lysosomotropic small molecules—lead asset GNS561/ezurpimtrostat, a PPT1 inhibitor—for primary and secondary liver cancers and idiopathic pulmonary fibrosis, generating revenue through out-licensing (Genfit) and public grants.

Ownership category

akta.pro rank

Product category

Clinical-stage Oncology Biopharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Pharmaceutical Preparation Manufacturing (325412)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

Keywords

• Oncology drug development
• Lysosomotropic therapeutics
• Autophagy modulation
• Hepatocellular carcinoma treatment
• Clinical-stage biopharmaceuticals

Headquarters

HQ city

Marseille-en-beauvaisis

HQ country

France

HQ region

Europe

Offices6 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales

Revenue model

1. Out-licensing and milestone payments: Genoscience Pharma out-licenses GNS561 to partners like Genfit for specific territories and indications, receiving upfront payments, milestone-based compensation, and royalties on future sales while retaining global development and commercialization rights for other territories
2. Government grants and funding: Received grants and refundable advances from French institutions including BPIFrance and PACA region, plus European Commission FEDER funding for idiopathic pulmonary fibrosis research

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

Genoscience Pharma is a clinical-stage biopharmaceutical company developing orally bioavailable small molecule drug candidates that target lysosomal functions to disrupt cancer cell survival, growth, and resistance. Its lead asset, GNS561 (ezurpimtrostat), is a first-in-class PPT1 inhibitor being advanced through Phase 1b/2a and Phase 2b (ABE-LIVER) trials in hepatocellular carcinoma, intrahepatic cholangiocarcinoma, and pancreatic/colorectal cancers with liver metastasis, with non-core indications out-licensed to Genfit.

Product overview

Genoscience Pharma is a clinical-stage biopharmaceutical company developing unique small molecule drug candidates that target lysosomal functions for cancer treatment. The company's main asset is GNS561 (ezurpimtrostat), a first-in-class PPT1 inhibitor administered orally that disrupts autophagy in cancer cells. The company retains global development and commercialization rights for GNS561 and has out-licensed the drug for cholangiocarcinoma to Genfit. Genoscience also historically developed antiviral candidates including BL-8030 and BL-8020 for hepatitis C, which were out-licensed to BioLineRx. The pipeline focuses on primary liver cancers (hepatocellular carcinoma, intrahepatic cholangiocarcinoma) and secondary liver cancers (pancreatic and colorectal cancer with liver metastasis).

Differentiator

Problem solved

Functional benefit

Products and services

• GNS561 (Ezurpimtrostat) First-in-class oral small molecule drug candidate that blocks cancer cell proliferation by inhibiting palmitoyl-protein thioesterase 1 (PPT1), causing late-stage autophagy inhibition and dose-dependent build-up of enlarged lysosomes; recommended Phase II dose is 200mg BID; under development for hepatocellular carcinoma, intrahepatic cholangiocarcinoma, pancreatic cancer with liver metastasis, and colorectal cancer with liver metastasis.
• BL-8020 Oral Hepatitis C treatment developed by Prof. Philippe Halfon that acts via inhibition of HCV-induced autophagy, showing synergistic effects when combined with other anti-HCV agents; exclusively out-licensed to BioLineRx.
• BL-8030 Second-generation orally bioavailable NS3 protease inhibitor for Hepatitis C with activity across multiple HCV genotypes and an improved resistance profile; licensed to BioLineRx via RFS Pharma collaboration.

Quantifiable outcome

• Phase 1b: RP2D established at 200mg BID with favorable safety profile - no dose-limiting toxicity observed across 26 evaluable patients
• +5 more outcomes

Named customers6 records

Segments4 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Six partnerships are on record, tiered core and minor.

• Yissum Research Development Company (Hebrew University of Jerusalem)coreStrategic or Co-development Partner · 19 April 2023New research agreement to expand PROTAC technology development on investigative Genoscience agents. Professor Raphael Benhamou leads research on targeted degradation therapy. PROTAC technology can increase potency and selectivity of small molecule drugs and overcome cancer resistance.
• RFS PharmaminorStrategic or Co-development Partner · 19 June 2007Joint venture to discover and develop protease inhibitors for life-threatening viral infections. Combined Genoscience's molecular modeling capabilities with RFS Pharma's pharmacology and drug development expertise. First joint venture with a US company.
• Grenoble University HospitalcoreStrategic or Co-development PartnerTrial sponsor for Phase 2b ABE-LIVER clinical trial evaluating ezurpimtrostat (GNS561) with atezolizumab/bevacizumab for first-line HCC treatment. First patient treated January 2, 2023.
• Memorial Sloan Kettering Cancer CentercoreStrategic or Co-development PartnerClinical trial site and co-principal investigator (Pr Ghassan K. Abou-Alfa) for Phase 1b/2a study in US. One of five clinical centers participating in patient recruitment.
• Jules Bordet InstitutecoreStrategic or Co-development PartnerLead investigator site (Pr Ahmad Awada) for first-in-human dosing and international Phase 1b/2a clinical trial in Belgium.
• Centre Leon BerardminorStrategic or Co-development PartnerCoordinating center for IMMUNONCOVID-20 Phase 2 study in COVID-19 patients with advanced or metastatic cancer under Dr. Philippe Cassier.

Scale indicators7 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Transgene: French clinical-stage biopharmaceutical company (Strasbourg) developing virus-based immunotherapies for solid tumors. Comparable as a French clinical-stage oncology biotech with multi-indication pipeline programs.
• Innate Pharma: French clinical-stage immuno-oncology company developing antibody-based therapeutics. Comparable in stage, location (Marseille/Paris), size, and oncology focus to Genoscience, serving as a benchmark for European clinical-stage oncology biotechs.
• Genfit: French clinical-stage biopharmaceutical company developing therapeutics for liver diseases and oncology. Acquired exclusive rights to develop and commercialize GNS561 in all oncology indications in April 2025, making it both a direct peer and the primary commercialization partner.
• BioLineRx: Israeli clinical-stage biotech that licensed BL-8020 and BL-8030 from Genoscience in 2012 for Hepatitis C development. Strategic licensee partner with comparable size, clinical-stage profile, and oncology/inflammatory disease focus.
• AB Science: French clinical-stage pharmaceutical company developing tyrosine kinase inhibitors (masitinib) for oncology, inflammatory, and neurological diseases. Comparable as a Paris-based French clinical-stage biotech with multiple indication programs.
• Puma Biotechnology: US oncology company (Nerlynx) led by Alan Auerbach and backed by Robert Duggan/Mahkam Zanganeh — board members shared with Genoscience. Comparable clinical-stage/specialty oncology biotech with similar board DNA and orphan-style indication focus.
• Onxeo: French clinical-stage oncology biotech focused on DNA-targeting and decoy-olecule therapeutics. Comparable in profile: European-based, clinical-stage, private-to-listed oncology biotech with limited commercialization footprint.

Broad incumbents

• AstraZeneca: Global biopharmaceutical leader with Imfinzi (durvalumab) approved for HCC and a major position in liver cancer immunotherapy combinations. Provides competitive and partnering context for Genoscience's HCC combination therapy strategy with checkpoint inhibitors.
• Incyte Corporation: Established US biopharmaceutical company with pemigatinib (Pemazyre) approved for cholangiocarcinoma — directly competing with Genoscience's licensed CCA program. Provides competitive context for what GNS561's commercial trajectory could resemble in CCA.
• Servier: French-headquartered global pharmaceutical company with Tibsovo (ivosidenib) approved for IDH1-mutated cholangiocarcinoma. Directly competes with Genoscience's licensed CCA indication and represents a likely competitor partner for out-licensing transactions.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat3 records

Key risks5 records

Key highlights7 records

Customer concentration

• Xx.com/GenoscienceP
• LinkedInlinkedin.com/company/genosciencepharma

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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

Frequently asked questions about Genoscience Pharma