Protara Therapeutics Provides Comparability and U.S. Regulatory Updates for TARA-002 Supporting Advancement in Oncology and Rare Disease Indications
- Company Demonstrates Initial Comparability Between TARA-002 and OK-432, Advancing to Final GMP Comparability Runs -
- Reaches Alignment with FDA on Development Path Forward for TARA-002 in Non-Muscle Invasive Bladder Cancer; Company Plans to Initiate Clinical Trials in 2021 -
- Company Expects to Request Meeting with FDA by Year End to Discuss Path to BLA for TARA-002 in Lymphatic Malformations -
“The outcome of our recent interaction with the FDA represents a significant milestone for the TARA-002 program, confirming initial comparability between TARA-002 and OK-432 and expanding the potential market opportunity of TARA-002 beyond LMs to include a potentially significant oncology indication,” said
FDA Confirmed Initial Comparability Between TARA-002 and OK-432; Final GMP Comparability Expected to be Completed in Mid-2021
Following a recent pre-Investigational New Drug (IND) engagement with the
Clinical Development Path in NMIBC
In addition, the Company reached alignment with the FDA on a proposed clinical development plan to evaluate TARA-002 in patients with NMIBC. Advancement into the clinic will be supported by existing and ongoing non-clinical studies as well as the historical safety and efficacy data for OK-432.
Subject to the successful completion of select non-clinical studies to characterize local toxicity of intravesical administration of TARA-002 as well as acceptance of an IND filing, the Company plans to commence a Phase 1 study in 2021 to assess the safety and tolerability of TARA-002 in patients with NMIBC, including patients with carcinoma in situ (CIS), with results expected in 2022.
The Phase 2 development program, which Protara plans to commence in 2022, is expected to include NMIBC patients with CIS +/- Ta and/or T1 papillary tumors and high-grade Ta and/or T1 papillary tumors without CIS.
Regulatory Path in LMs
Protara plans to request a meeting with the
TARA-002 is an investigational cell therapy in development for the treatment of lymphatic malformations (LMs) and non-muscle invasive bladder cancer (NMIBC). TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil® in
When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-6, IL-8, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, and vascular endothelial growth factor (VEGF) are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells. TARA-002 has been granted Rare Pediatric Disease Designation by the
About Non-Muscle Invasive Bladder Cancer
Bladder cancer is the 6th most common cancer in
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 present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of 2 years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.
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.
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Source: Protara Therapeutics