Protara Therapeutics Provides Regulatory Update for TARA-002 for the Treatment of Lymphatic Malformations
Protara Therapeutics (Nasdaq: TARA) announced plans for an additional clinical study to support their Biologics License Application for TARA-002, targeting Lymphatic Malformations (LMs). Based on FDA feedback, the company will conduct large-scale GMP manufacturing comparability in late 2021 and initiate a pediatric clinical study pending alignment with the FDA. TARA-002 is derived from the same cell bank as OK-432, an approved treatment in Japan and Taiwan. Protara aims to deliver the first FDA-approved drug for LMs.
- Plans for additional clinical study to support TARA-002 Biologics License Application.
- FDA feedback is guiding the development process.
- TARA-002 has demonstrated initial manufacturing comparability with OK-432.
- Rare Pediatric Disease designation granted by the FDA.
- No FDA-approved treatment currently available for LMs, indicating high unmet need.
Company to conduct additional clinical study to support submission of a Biologics License Application for TARA-002 in Lymphatic Malformations
NEW YORK, April 23, 2021 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical stage company developing transformative therapies for the treatment of cancer and rare diseases with significant unmet needs, today announced a path forward related to TARA-002 for the treatment of Lymphatic Malformations (LMs), which are rare malformations of the lymphatic vasculature for which there is no U.S. Food and Drug Administration (FDA)-approved treatment. Based on feedback from the FDA, the Company intends to complete confirmatory, large-scale, GMP manufacturing comparability in the second half of 2021 and subsequently initiate a clinical study in pediatric LM patients pending alignment with FDA on study design.
“With the benefit of the recent FDA feedback, we will work with the agency to align on a clinical study in pediatric LM patients, which, we believe, combined with the existing dataset for OK-432 (the originator compound for TARA-002), which demonstrated treatment effect and support for strong safety profile in over 500 LM patients, should provide a robust data package for this rare disease,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We have already begun preparation to initiate a clinical study in LMs and we look forward to continued collaboration with FDA to achieve our goal of delivering the first approved medication for LMs to these patients and their physicians.”
TARA-002 is derived from the same cell bank as OK-432, a broad immunopotentiator approved in Japan and Taiwan for the treatment of LMs, where it is currently the standard of care. In 2020, Protara successfully demonstrated initial manufacturing comparability between TARA-002 and the originator compound OK-432, which has been studied in more than 500 patients in one of the largest Phase 2 trials ever conducted in LMs. TARA-002 has been granted Rare Pediatric Disease designation by the FDA for the treatment of LMs.
About Lymphatic Malformations
Lymphatic malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than
About Protara Therapeutics
Protara is committed to identifying and advancing transformative therapies for people with cancer and rare diseases with limited treatment options. Protara’s portfolio includes its lead program, TARA-002, an investigational cell-based therapy being developed for the treatment of non-muscle invasive bladder cancer and lymphatic malformations, and IV Choline Chloride, an investigational phospholipid substrate replacement therapy for the treatment of intestinal failure-associated liver disease. For more information, visit www.protaratx.com
Forward-Looking Statements
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Company Contact:
Justine O’Malley
Protara Therapeutics
Justine.omalley@protaratx.com
646-817-2836
FAQ
What is the purpose of the additional clinical study for TARA-002?
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What is TARA-002 and how is it related to OK-432?
What designation has TARA-002 received from the FDA?
