Partnerships and Pipeline
Every time we innovate to help each person live better...
Our ambition is to be a partner of choice for biotechs bringing innovative therapies to market. Our focus is breakthrough therapies for diseases with high unmet needs, especially in oncology and rare diseases. We are actively identifying potential therapies to in-license and complement our growing pipeline.
As part of Pierre Fabre Laboratories, we bring six decades of worldwide experience in pharmaceutical commercialization, while our agile corporate structure enables us to develop and implement at biotech speed. Most of all, we emphasize creating long-term value for patients and society.
Innovative precision treatments
We build our pipeline through both research and development (R&D) and business development and licensing (BD&L). In both domains, oncology is a priority focus, accounting for over 80% of total investment.
Our current portfolio consists of one commercial products, one product in registration, one product in Phase II development, and two products in Phase I development.
Disease Area:
Dermatology
Indication:
Proliferating infantile hemangioma requiring systemic therapy
- Beta-blocker
- Blocks beta-adrenergic receptors
- Administered as an oral solution
- Received FDA Approval in March 2014
Development Partner:
Approved
Development
Partner:
Tabelecleucel
Disease Area:
Hematology
Indication:
Relapsed or refractory Epstein-Barr virus+ post-transplant lymphoproliferative disorder (EBV+ PTLD)
- Cell therapy
- Allogeneic T-cell therapy where immune cells from a healthy donor are used to target and destroy EBV-infected B cells
- Administered through IV Injection
- Clinicaltrials.gov ID: NCT03394365
Development Partner:
Registration
Development
Partner:
ER004
Disease Area:
Genetic Disorder
Indication:
Hypohidrotic ectodermal dysplasia (XLHED)
- Protein replacement therapy
- In-utero therapy designed to replace the function of endogenous ectodysplasin A1 (EDA1), which is a protein key to the normal development of ectodermal structures in fetuses
- Administered through intra-amniotic injection
- First patient enrolled in the Edelife trial in April 2022
- Clinicaltrials.gov ID: NCT04980638
Development Partner:
Phase II
Development
Partner:
Exarafenib
Disease Area:
Solid Tumor
Indication:
NRAS mutant melanoma
- Targeted therapy
- Pan-RAF small molecule inhibitor that targets BRAF class I, II, and III and NRAS alterations, which are known drivers of disease in patients with melanoma
- Administered orally
- First patient enrolled in August 2021
- Pierre Fabre and Kinnate entered into an Asset Purchase Agreement for Exarafenib in March 2024
- Clinicaltrials.gov ID: NCT04913285
Development Partner:
Phase I
Development
Partner:
PFL-002 / VERT-002
Disease Area:
Solid Tumor
Indication:
Non-small cell lung cancer (NSCLC) with mutation or amplification of MET
- Targeted therapy
- Monoclonal antibody acting as a degrader of c-MET, which is a known driver of disease in patients with MET mutations or amplification in NSCLC
- Investigational New Drug (IND) application filed to the FDA in June 2024
- First patient enrolled in October 2024
- Clinicaltrials.gov ID: NCT06669117
Phase I
PFL-721
(formerly STX-721)
Disease Area:
Solid Tumor
Indication:
NSCLC with EGFR exon 20 insertion mutation
- Targeted therapy
- Mutant-specific EGFR inhibitor that is highly selective for exon 20 insertion mutations, which account for about 4-10% of all EGFR mutations in NSCLC
- Administered orally
- Clinicaltrials.gov ID: NCT06043817
Development Partner:
Phase I
Development
Partner:
PFL-241
(formerly STX-241)
Disease Area:
Solid Tumor
Indication:
NSCLC with EGFR exon 19/21 + C797S mutations
- Targeted therapy
- Mutant-specific, brain-penetrant, 4th-generation EGFR inhibitor that selectively inhibits the C797S resistance mutation co-occurring with EGFR exon 19 deletion or exon 21 mutation, which emerges in about 12.5% of patients receiving therapy with 3rd-generation EGFR inhibitors
- Administered orally
- Clinicaltrials.gov ID: NCT06567015
Development Partner:
Phase I
Development
Partner: