INS018_055, a potential treatment for idiopathic pulmonary fibrosis (IPF), has received the orphan drug designation from the US Food and Drug Administration (FDA), Insilico Medicine announced.
“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,” said Alex Zhavoronkov, PhD, CEO and founder of Insilico Medicine.
The novel treatment was developed using clinical-stage end-to-end generative artificial intelligence (AI) technology. It was the company’s first project that fully relied on AI to identify potential targets and craft the small molecules.
The project was first announced in February 2021 as a preclinical study. A year later, the phase 1 clinical trial began in China and New Zealand. Results from the trial demonstrated a promising pharmacokinetic profile with acceptable safety and tolerability.
Read more about IPF therapies
INS018_055 is a small molecule inhibitor, currently the first in its class. Novel therapeutic options for IPF represent important advances since current treatment schemes do not yield optimal results and lack long-term benefits.
IPF is a chronic, progressive disease that causes irreversible damage to the lung tissue and leads to a decline in respiratory function, which translates into poor quality of life and high morbidity.
The orphan drug designation encourages and facilitates the creation, development, and testing of new medications for diseases that affect 200,000 people or less in the US and facilitates further studies and trials.
The developers announced the next step in the testing of INS018_055 is a global multicenter phase 2 clinical trial, which they plan to initiate early in 2023.
Insilico Medicine receives FDA orphan drug designation for generative AI discovered and designed drug for idiopathic pulmonary fibrosis. News release. Insilico Medicine; February 8, 2023.