Insilico’s AI-inspired therapy earns orphan drug designation

FDA decision clears way for Phase 2 clinical trial of INS018_055 later this year

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has granted INS018_055 orphan drug designation for the treatment of idiopathic pulmonary fibrosis (IPF), the therapy’s developer Insilico Medicine, announced.

Insilico is expecting to initiate a global Phase 2 clinical trial testing INS018_055 in people with IPF later this year.

Orphan drug designation is given to therapies with the potential to improve care for rare diseases — defined as those affecting fewer than 200,000 people in the U.S. The designation gives Insilico, as the therapy’s developer, certain incentives, including tax credits on qualified clinical testing, exemption from certain fees, and — most notably — a guaranteed right to seven years of market exclusivity if the therapy is ultimately approved.

“The FDA’s orphan drug designation for the IPF indication is an important milestone in the development of INS018_055,” Feng Ren, PhD, co-CEO and chief scientific officer of Insilico, said in a press release.

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“Our commitment to treating patients with idiopathic pulmonary fibrosis is reflected in the FDA’s decision to grant orphan drug designation to INS018_055,” said Sujata Rao MD, Insilico’s senior vice president and head of clinical development. “We are working to advance novel treatment for IPF as we continue clinical investigation into this devastating disease.”

INS018_055, also referred to as ISM001-055, is a first-in-class therapy that Insilico developed using an artificial intelligence (AI) platform. The company’s platform involved assessments to identify a target molecule in the body that could be manipulated to influence disease processes, then designing a therapy to specifically bind to that target for the desired effect.

“INS018_055 is a potential first-in-class drug candidate discovered by Insilico’s generative AI platform,” Ren said.

Insilico conducted a Phase 1 clinical trial (NCT05154240) that tested oral INS018_055 in 78 healthy volunteers. Results announced in January showed the experimental therapy was tolerated well; all reported side effects related to treatment were mild and resolved by the end of the study. Pharmacological data were in line with expectations.

Buoyed by these results, Insilico is expecting to launch a Phase 2a clinical trial early this year that will evaluate INS018_055 in people with IPF. The study is expected to take place at sites around the world.

“Insilico scientists are now further advancing clinical validation and accelerating the project to meet clinical needs and benefit patients worldwide,” Ren said.

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