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Protara Therapeutics Presents Results from Retrospective Analysis of Randomized and Open-Label Studies Evaluating the Safety and Efficacy of OK-432 in Patients with Lymphatic Malformations
Data showed OK-432 was clinically successful in treating lymphatic malformations and support a favorable safety profile NEW YORK, May 12, 2022 (GLOBE
About this update from Protara Therapeutics, Inc.
[{"type":"text","content":"Data showed OK-432 was clinically successful in treating lymphatic malformations and support a favorable safety profile\nNEW YORK, May 12, 2022 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced the results of a retrospective analysis of OK-432, the originator compound for TARA-002, Protara’s investigational therapy in development for the treatment of lymphatic malformations (LMs). LMs are serious, rare, congenital malformations of lymphatic vessels. The results from the analysis, which were presented during a poster presentation at the International Society for the Study of Vascular Anomalies (ISSVA) World Congress 2022, showed that OK-432 was clinically successful and generally well-tolerated in the treatment of both macrocystic and mixed-cystic LMs. “We are pleased to share these compelling results, which are consistent with the robust body of approximately 30 years of patient experience with OK-432,” said Richard Smith, M.D., Department of Otolaryngology, Carver College of Medicine, University of Iowa, and author of the study. “There are currently no FDA-approved treatments for LMs, which are usually diagnosed in early childhood and can lead to serious complications. These data provide continued support for the potential of TARA-002 to ultimately serve as an effective intervention in this highly underserved area.” The retrospective analysis included 246 patients from a Phase 2 randomized study, and 275 patients from an open-label study. The majority of participants in both studies were six months to 18 years of age. In the first study, patients were randomized 2:1 to receive treatment immediately (immediate treatment group [ITG]) or delayed by six months (delayed treatment group [DTG]). In the open-label study, patients received four doses of OK-432 approximately six weeks apart. The primary efficacy endpoint was clinical success (defined as complete [90%-100%] or substantial [60%-89%] reduction in LM volume measured radiographically) in the ITG versus spontaneous resolution of the LM in the DTG. Efficacy was assessed two weeks post-treatment in the randomized study, and one to six months post-treatment in the open-label study. Key findings are summarized below: Approximately 69% of patients...