* TARA-002 Granted Rare Pediatric Disease Designation for the treatment of LMs
** Granted Orphan Drug Designations by the U.S. FDA
TARA-002 is an investigational cell therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs). 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 Japan and Taiwan by Chugai Pharmaceutical Co., Ltd.
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-2, IL-6, IL-8, IL-10, IL-12, interferon (IFN)-gamma, and tumor necrosis factor (TNF)-alpha are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.
IV choline chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy in development for patients receiving parenteral nutrition (PN).
Choline is an important substrate for phospholipids that are critical for healthy metabolism. It plays important roles in modulating gene expression, cell membrane signaling, lipid transport and absorption, liver health, brain development and neurotransmission, muscle function and bone health. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients can experience a number of health consequences, including hepatic injury, neuropsychological impairment (including memory issues), and muscle damage, as well as thrombotic abnormalities. If approved, IV choline chloride would be the first approved therapy for choline deficiency in PN patients. It has been granted Orphan Drug Designation (ODD) by the FDA for the prevention and/or treatment of choline deficiency in patients on PN.
Protara is committed to identifying and advancing transformative therapies for the treatment of cancer and rare diseases with significant unmet needs.
Expanded access, also called compassionate use, makes an investigational product available for treatment outside of clinical trials when no comparable or satisfactory alternative therapy option is available.
Protara is committed to advancing its investigational therapies, TARA-002 and IV Choline Chloride, by continuing to study them in clinical trials designed to confirm potential safety, tolerability and efficacy. We believe the best way to make our therapies available to patients is by enrolling and completing all of our clinical trials and, if those trials are successful, pursuing regulatory approval. Protara is not making its medicines available via expanded access at this time. We are focused on advancing our clinical development programs and more information about our clinical trials can be found on ClinicalTrials.gov when those trials are underway. We believe that participation in our clinical trials will be the most appropriate way to access our investigational medicines prior to their approval.
Protara will continually assess our current expanded access policy and provide updates to this website. We look forward to collaborating with the U.S. FDA and making these important therapies available to patients. Please contact us with any questions at firstname.lastname@example.org.