Molecure's OATD-01 Clears IDMC Interim Analysis in Phase II Pulmonary Sarcoidosis Trial
Key Insights
The Independent Data Monitoring Committee recommended the KITE Phase II trial continue without protocol changes after reviewing safety and efficacy data from the first 30 patients.
A futility analysis based on unblinded data indicated sufficient probability of achieving the primary endpoint via PET/CT imaging and selected secondary endpoints.
Preliminary blinded biomarker analysis from 23 patients showed inflammatory biomarker trends consistent with OATD-01's expected anti-inflammatory biological response.
Molecure S.A. (search) (WSE: MOC), a clinical-stage biotechnology company headquartered in Warsaw, Poland, announced that the Independent Data Monitoring Committee (IDMC) has recommended the continuation of the global Phase II KITE clinical trial without any changes to the current study protocol. The trial is evaluating OATD-01, a first-in-class CHIT1 (search) inhibitor, in patients with active pulmonary sarcoidosis (search).
The IDMC's recommendation followed a comprehensive review of the complete safety and efficacy dataset from an interim analysis involving the first 30 patients who completed the 12-week treatment period with either OATD-01 or placebo. Based on unblinded data, a futility analysis indicated sufficient probability of achieving the primary endpoint—which relies on differences in PET/CT imaging—as well as selected secondary endpoints.
"The IDMC recommendation is highly reassuring at this stage. The Committee recommended that KITE continue without any changes to the protocol, which suggests that the study has a good chance of achieving its primary efficacy endpoint and meets the required safety criteria," said Dr. Marcin Szumowski, CEO of Molecure S.A. (search) "From a business perspective, the IDMC recommendation, along with the observed biomarker trends and favorable safety profile, strengthens our position in ongoing partnering discussions following the strong interest we received during the BIO International Convention."
Biomarker Signals Point to Anti-Inflammatory Activity
As part of the interim analysis, Molecure examined blinded biomarker data from 23 patients, focusing on inflammatory biomarkers. The observed trends suggest anti-inflammatory activity consistent with OATD-01's expected biological response, though the company cautioned that definitive conclusions cannot be drawn until the study is fully unblinded.
"I am very gratified to hear about the positive IDMC recommendation and the progress in the KITE study," said Daniel Culver, a leading lung sarcoidosis expert from Cleveland Clinic, one of the largest reference centers for pulmonary sarcoidosis (search). "I was also highly impressed with the biomarker data. These data are consistent with the possibility that there are between-group differences related to the study drug for biomarkers of sarcoidosis inflammation. It is exciting to see that a study for a novel mechanism of action and innovative trial design is making good progress."
Dr. Dariusz Stencel, Chief Medical Officer of Molecure, added: "Despite the limitations of the double-blind nature of the study the changes observed in selected inflammatory biomarkers are highly encouraging. These findings reinforce our confidence in the asset's potential for further development and will support further patient recruitment."
Trial Design and Next Steps
The KITE study is a double-blind, placebo-controlled Phase II trial being conducted at nearly 30 sites across the United States, European Union, Norway, and the United Kingdom. The trial evaluates a fixed 25 mg dose of OATD-01 administered over a 12-week treatment period.
Molecure is currently randomizing an additional 20 patients for the second and final interim analysis, which will include a statistical re-evaluation of the required trial size. The positive IDMC recommendation is expected to boost patient recruitment and accelerate ongoing business development discussions for the OATD-01 program.
OATD-01 holds therapeutic potential across multiple interstitial lung diseases, including sarcoidosis and idiopathic pulmonary fibrosis (search), as well as MASH (search). Molecure's broader pipeline also includes OATD-02, a dual arginase inhibitor currently in Phase I development for cancer.