FDA clears OATD-01 Phase 2 pulmonary sarcoidosis clinical trial

Phase 1 trial showed OATD-01 was safe, suppressed CHIT1 enzyme

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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The U.S. Food and Drug Administration (FDA) has greenlit a month-old request by Molecure to begin a Phase 2 clinical trial of its investigational oral therapy OATD-01 in people with pulmonary sarcoidosis.

The proof-of-concept study, which is ready to launch, according to Molecure, will be the first to test OATD-01 in people with the lung disease. The first patients are expected to start treatment by the end of the year and the company plans to ask European authorities to expand Phase 2 testing to Europe, including Poland, where it’s based. Molecure was previously known as OncoArendi Therapeutics.

“We are extremely excited to share this great news – not only for us, but for every patient looking for a better and more effective way of treating sarcoidosis,” Marcin Szumowski, Molecure’s CEO, said in a company press release. “This marks the beginning of a new chapter in the development of our flagship project, as we enter human proof-of-concept studies and begin treating pulmonary sarcoidosis patients with OATD-01.”

The Phase 2 trials are expected to conclude in mid-2025.

Pulmonary sarcoidosis is a type of sarcoidosis wherein small clumps of inflammatory cells, or granulomas, accumulate in the lungs, causing inflammation and tissue scarring, or fibrosis.

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What is OATD-01?

OATD-01 is a small molecule that blocks the activity of chitotriosidase (CHIT1), an enzyme involved in several immune functions and found at high levels in the blood and lungs of sarcoidosis patients. Greater CHIT1 activity has been linked to higher granuloma burden and disease activity.

By suppressing the enzyme, Molecure believes OATD-01 may slow the progression of pulmonary sarcoidosis and other lung diseases where it’s been implicated. Preclinical studies have demonstrated the molecule can ease inflammation and reduce the number of lung granulomas in mouse models of sarcoidosis-like disease.

“As we have confirmed in a range of preclinical studies, OATD-01 has the ability to modulate macrophage activity, meaning it has the potential to be disease modifying and treat various inflammatory and fibrotic diseases which develop based on a similar molecular mechanism,” Szumowski said. Macrophages are a type of immune cell involved in sarcoidosis and inflammatory and fibrotic processes.

Data from a Phase 1 trial in healthy volunteers indicated OATD-01 was safe and could strongly suppress CHIT1.

In the multicenter Phase 2 trial, up to 90 patients will be randomly assigned to receive either OATD-01 or a placebo, once a day for at least 12 weeks, or about three months. The trial’s goal will be to determine the extent to which OATD-01 can reduce granuloma-associated inflammation in lung tissue after three months. This will be assessed with PET and CT scans.

In an effort to facilitate its clinical program, Molecure has been working to build relationships with clinical experts specializing in lung disease, as well as other patient-focused foundations and organizations. The company has also participated in recent seminars and conferences toward that goal.

“We are confident that that this will help us get more U.S. and EU research faculties involved in our trials, resulting in faster patient recruitment,” Szumowski said.

OATD-01 has earned orphan drug status in the U.S. for treating pulmonary sarcoidosis and idiopathic pulmonary fibrosis, another type of chronic lung disease. The designation offers financial incentives and regulatory support to speed a therapy’s clinical development.