FDA grants fast track designation to Insilico's AI-designed mesothelioma drug candidate

Insilico Medicine ("Insilico"; HKEX: 3696), a clinical-stage generative artificial intelligence (AI)-driven drug discovery company, today announced that ISM6331, a novel, potential best-in-class pan-TEAD inhibitor driven by Insilico's proprietary AI, has received Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA), for the treatment of adult patients with unresectable malignant pleural mesothelioma whose disease has progressed on or after prior treatment with anti-PD-1 antibody therapy, with or without anti-CTLA-4 antibody therapy, and platinum-based chemotherapy.

According to the FDA's relevant policy, Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. This is the first Fast Track Designation granted to a program in Insilico's AI-driven pipeline, recognizing ISM6331's potential to provide a meaningful therapy where limited options exist or to offer improved clinical efficacy over available treatments. Previously, ISM6331 received Orphan Drug Designation (ODD) for the same indication in June 2024, following the ODD in February 2023 for Rentosertib (ISM001-055), Insilico's lead program currently in Phase III clinical trial for the treatment of idiopathic pulmonary fibrosis (IPF).

With the Fast Track Designation, ISM6331 gains access to regulatory benefits designed to streamline its clinical development path, including enhanced FDA engagement represented by more frequent meetings and written feedback regarding clinical trial design, biomarker strategies, and overall development plans. Additionally, subject to meeting the relevant criteria, the program may qualify for Accelerated Approval, Priority Review and Rolling Review process. Under the Rolling Review process, completed sections of a Biologic License Application (BLA) or New Drug Application (NDA) may be submitted to FDA for review as they become available, rather than waiting for the entire application to be completed.

Receiving Fast Track Designation validates the strong clinical potential of ISM6331, and that is a boost to our confidence in it, on top of promising preclinical results and Phase I first-in-human progresses. Moreover, ISM6331 boasts synergistic anti-tumor effects and potential to overcome drug resistance as combination therapy. We hope to work even more closely with the FDA as we accelerate clinical evaluation of ISM6331 for future development."

Halle Zhang, Ph.D., Vice President, Clinical Development – Oncology, Insilico Medicine

ISM6331 is a potential best-in-class pan-TEAD inhibitor nominated in Jun 2023, with its novel scaffold empowered by Chemistry42, Insilico's proprietary generative chemistry platform. Chemistry42 utilized structure-based drug design strategies to design novel molecules, which were subsequently prioritized through its advanced scoring and reward pipelines.

"Through pan-TEAD inhibition, ISM6331 holds best-in-class potential by aiming to restore balance to the Hippo pathway and prevent the proliferation and survival of tumor cells, and we're proud to see the Chemistry42-driven candidate has its value recognized by the regulatory authorities with both ODD and FTD," said Feng Ren, PhD, co-CEO and Chief Scientific Officer of Insilico Medicine. "At Insilico, we are delivering strategies to revolutionize drug R&D at scale, and the generative AI advantage in efficiency is largely maintained or even enhanced through innovation-friendly schemes like the FTD. We will always be open for global partnership and regulatory support to accelerate our clinical development momentum, and bring this innovative AI-driven option, among others, to patients in urgent need."

Due to the novel scientific rationale and promising first-hand data in preclinical or clinical studies, ISM6331 was previously featured in AACR 2024 Annual Meeting, and its initial Phase I first-in-human clinical data has been accepted for a brief oral presentation at the upcoming ESMO 2026 Congress.