FDA grants orphan drug designation to AI-created drug for idiopathic pulmonary fibrosis

The FDA granted orphan drug designation to INS018_055, an anti-fibrotic small molecule inhibitor, for treating patients with idiopathic pulmonary fibrosis, according to a press release from the drug’s manufacturer.

INS018_055 (Insilico Medicine) was identified and generated through Pharma.AI, the company’s artificial intelligence (AI) platform, according to the release.

This orphan drug designation follows a randomized, double-blind, placebo-controlled phase 1 trial conducted in New Zealand, according to an earlier release from the manufacturer. This trial demonstrated that the AI-discovered drug was generally safe and well tolerated in 78 healthy volunteers, with a favorable pharmacokinetic profile.

According to the release, no deaths or serious adverse events occurred, and any mild treatment-related adverse events that patients experienced resolved at the trial’s conclusion.

“We are pleased to announce that Insilico has achieved numerous drug discovery milestones and provided new clinical hope using generative AI,” Alex Zhavoronkov, PhD, Insilico Medicine’s founder and CEO, said in the release. “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.”

The company plans to begin a phase 2 trial of INS018_055 in early 2023, according to the release.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

Reference:

• Insilico Medicine announces positive topline results of the New Zealand phase 1 trial of INS018_055, an AI-designed drug for an AI-discovered target. https://www.globenewswire.com/news-release/2023/01/10/2586249/31533/en/Insilico-Medicine-announces-positive-topline-results-of-the-New-Zealand-Phase-1-trial-of-INS018_055-an-AI-designed-drug-for-an-AI-discovered-target.html#:~:text=New%20York%2C%20Jan.%2010%2C%202023%20%28GLOBE%20NEWSWIRE%29%20--,end-to-end%20AI%20platform%20for%20idiopathic%20pulmonary%20fibrosis%20%28IPF%29. Published Jan. 10, 2023. Accessed Feb. 8, 2023.