PureTech Health (PRTC) Leerink Global Healthcare Conference 2025 summary

Company overview and R&D model

• Employs a hub-and-spoke R&D model, focusing on clinically validated molecules with unmet patient needs.

• Achieved over 80% clinical trial success rate in the past 15 years.

• Generates capital through equity, milestones, and royalties from Founded Entities.

• Maintains a strong balance sheet, ending 2024 with $365 million.

• Prioritizes rigorous de-risking and early validation in product development.

Key pipeline achievements and financial highlights

• Reported unprecedented phase II-B data for deupirfenidone in idiopathic pulmonary fibrosis (IPF).

• Karuna Therapeutics, a Founded Entity, was acquired for $14 billion; COBENFY, invented at PureTech, received FDA approval for schizophrenia.

• Launched Seaport Therapeutics, raising $325 million to advance CNS programs.

• Deupirfenidone program demonstrated stabilization of lung function at 26 weeks and durable effect at 52 weeks.

• Holds broad IP protection for deupirfenidone through 2043.

Clinical data and future plans for deupirfenidone

• Phase II-B trial showed dose-dependent efficacy, with the highest dose achieving lung function decline similar to healthy older adults.

• High-dose arm (825 mg) showed better efficacy and favorable GI tolerability compared to pirfenidone.

• Plans to initiate phase III trial for IPF by end of 2025, with regulatory discussions mid-year.

• Exploring financing options for phase III, including royalty-linked and regional deals.

• Aims to retain US rights and potentially partner ex-US rights.