Insilico Medicine Receives FDA Orphan Drug Designation for Generative AI Discovered and Designed IPF Drug

Rare Daily Staff

The U.S. Food and Drug Administration granted Insilico Medicine Orphan Drug Designation to its experimental idiopathic pulmonary fibrosis therapy INS018_055, a drug in which artificial intelligence was used to both identify a novel target and to generate a novel small molecule candidate against it.

Idiopathic pulmonary fibrosis (IPF) is a chronic lung disease that causes progressive and irreversible decline in lung function and represents a significant unmet medical need worldwide. As the disease progresses, damage to the patient’s lungs increases and respiratory function is compromised, often with severe consequences.

INS018_055 is a potentially first-in-class small molecule inhibitor discovered by Insilico’s generative AI platform, Pharma.AI, for the treatment of IPF.

INS018_055 is the first wholly owned program of the company in which AI was used both to identify a novel target and to generate novel small molecules. The enrollment for the INS018_055 phase 1 clinical trial was initiated in New Zealand in February 2022 and in China in May 2022. Both of the clinical trials were completed in 2022. Top-line data from the phase 1 clinical trial indicated that the drug candidate has a favorable safety, tolerability, and pharmacokinetic profile. Insilico plans to initiate a global multicenter phase 2 clinical trial in early 2023.

The FDA’s Orphan Drug Designation program supports the development and evaluation of drugs that address rare diseases which affect fewer than 200,000 people in the United States. Receiving orphan drug designation from the FDA facilitates the subsequent development and commercialization of INS018_055 that comes with the designation, including eligibility for federal grants, tax credits for qualified clinical trials, prescription drug user fee exemptions, and a seven-year marketing exclusivity period upon FDA approval.

“Insilico has achieved numerous drug discovery milestones and provided new clinical hope using generative AI,” said Alex Zhavoronkov, founder and CEO of Insilico Medicine. “We are progressing the global clinical development of the program at top speed to allow patients with fibrotic diseases to benefit from this novel therapeutic as soon as possible.”

Photo: Alex Zhavoronkov, founder and CEO of Insilico Medicine