Insilico Medicine secures IND clearance in China for inhaled Rentosertib, advancing a Phase I study in Idiopathic Pulmonary Fibrosis.
Written By: Fariha Sameen, PharmD
Reviewed By: Pharmacally Editorial Team
Insilico Medicine announced that China’s Center for Drug Evaluation (CDE) has granted Investigational New Drug (IND) clearance for an inhalation formulation of Rentosertib (ISM001‑055) for the treatment of Idiopathic Pulmonary Fibrosis (IPF). This marks the 13th program from the company’s AI‑driven pipeline to reach the IND stage, and CDE has also granted the inhalation formulation Breakthrough Therapy Designation under China’s regulatory framework.
The clearance supports a Phase I clinical study to evaluate the safety, tolerability, and pharmacokinetic (PK) profiles of inhaled Rentosertib in approximately 80 participants. The trial will consist of two parts: (1) a randomized, double‑blind, placebo‑controlled component in healthy volunteers with single‑ascending dose (SAD) and multiple‑ascending dose (MAD) cohorts; and (2) an open‑label evaluation in patients with IPF receiving multiple doses.
The inhalation formulation is designed to deliver Rentosertib directly to the lungs, aiming to increase local pulmonary exposure while reducing systemic exposure. In preclinical studies, the inhaled form has demonstrated antifibrotic and anti‑inflammatory activity, along with favorable pharmacokinetic and safety profiles. Earlier clinical data with the oral formulation showed good tolerability and a dose‑dependent efficacy signal, supporting continued development of both the oral and inhalation routes.
Feng Ren, Co‑CEO and Chief Scientific Officer of Insilico Medicine, stated that the IND clearance reflects confidence in Rentosertib’s mechanism and clinical potential, citing prior Phase IIa data that demonstrated favorable tolerability and pharmacokinetics. He added that the inhalation formulation is intended to enhance lung‑targeted delivery while minimizing systemic exposure, and confirmed that the oral program remains on track to advance into Phase III trials later this year.
Alex Zhavoronkov, Founder and Chief Executive Officer of Insilico Medicine, noted that the milestone underscores the scalability and reproducibility of the company’s AI‑driven drug discovery approach, powered by its Pharma.AI platform which integrates target identification, molecule design, and clinical‑development support. Insilico reported broader pipeline progress, including 13 IND‑cleared programs, 30 preclinical candidates, and three ongoing Phase II trials, with internal early‑discovery timelines reduced to approximately 12–18 months compared with conventional approaches.
Idiopathic Pulmonary Fibrosis is a progressive lung disease affecting an estimated 5 million people worldwide and is associated with a median survival of 3–4 years. Current therapies can slow disease progression but do not reverse it, highlighting the need for more effective, mechanism‑based treatments.
Rentosertib, a small‑molecule TNIK inhibitor discovered using generative AI, has received Orphan Drug Designation from the U.S. FDA and Breakthrough Therapy Designation from China’s CDE. The program advanced from target identification through early clinical development in approximately 18–30 months, with only a limited number of synthesized compounds, underscoring the efficiency gained from Insilico’s AI‑enabled platform.
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