Bexotegrast for IPF Earns EMA Orphan Drug Designation

Pliant Therapeutics’ experimental therapy gains ground in Europe

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Pliant Therapeutics’ experimental oral anti-fibrotic therapy bexotegrast has received orphan drug designation from the European Medicines Agency (EMA) as a potential treatment for idiopathic pulmonary fibrosis (IPF), the company announced.

EMA gives this designation to therapies that have the potential to improve care for conditions that affect fewer than five of every 10,000 people in the European Union. The designation qualifies Pliant for a series of benefits, including assistance with clinical trials, access to a centralized approval process, and a guarantee of 10 years of market exclusivity should the therapy ultimately be approved.

“This designation acknowledges the unmet need in IPF, as well as the potential of bexotegrast, and represents an important milestone in the clinical development of this novel drug. We look forward to continuing our work with the EMA and regulators around the globe to advance this therapy for patients in need,” Éric Lefebvre, MD, Pliant’s chief medical officer, said in a press release.

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Bexotegrast, formerly known as PLN-74809, is a small molecule designed to block two integrins — cell surface proteins that play a key role in cell adhesion and also are thought to drive fibrosis (tissue scarring) in IPF. The therapy was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) in 2018, and it was given fast track designation earlier this year.

In early Phase 1 clinical trials conducted in healthy volunteers, the therapy was generally well-tolerated and showed promising anti-fibrotic effects.

Pliant now is sponsoring a Phase 2a clinical trial called INTEGRIS-IPF (NCT04396756) to assess the safety and effectiveness of bexotegrast in people with IPF.

Interim data from the study showed the experimental treatment was generally well-tolerated at doses up to 160 mg per day, and suggested it may slow lung function decline when given with or without current IPF standard care therapies.

INTEGRIS-IPF now is testing a higher dose of the therapy (320 mg per day) in a group of about 28 IPF patients. According to Pliant, 12-week data from this higher dose group is expected to be announced in the first quarter of 2023.

“Following the positive interim results from our Phase 2a trial of bexotegrast, we are pleased to receive the EMA’s orphan medicinal product designation,” Lefebvre said.

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