PIPELINE

We have established a portfolio of de-risked assets addressing rare diseases and oncology.
Pre-Clinical IND Cleared Phase 1 Phase 2 Phase 3 HEPATOLOGY, GI, METABOLICS Intestinal Failure- Associated Liver Disease (IFALD) **,† Lymphatic Malformations(LMs)* TARA-002 combinations Non-Muscle Invasive Bladder Cancer (NMIBC) IMMUNOLOGY, ONCOLOGY TARA-002 – Lyophilized, inactivated Group A Streptococcus IV Choline Chloride for Injection - Phospholipid Substrate Replacement

* TARA-002 Granted Rare Pediatric Disease Designation for the treatment of LMs
** Granted Orphan Drug and Fast Track Designations by the U.S. FDA
† Phase 1 PK study to be conducted in addition to Phase 3 study

TARA-002

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.

TARA002 MoA

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, tumor necrosis factor (TNF)-alpha, and granulocyte colony-stimulating factor, and granulocyte-macrophage colony-stimulating are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.

Bladder cancer is the 6th most common cancer in the United States, with non-muscle invasive bladder cancer (NMIBC) representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle. There is an urgent need for new treatments to address NMIBC as significant increases in recurrence, progression, and the number of reported patients needing cystectomies are being observed.
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. There are currently no approved therapies to treat LMs.

TARA-002 is currently being studied in a Phase 1 dose-finding, open-label trial in treatment-naïve and treatment-experienced NMIBC patients with high-grade carcinoma in situ and high-grade papillary tumors. Learn more at clinicaltrials.gov

IV Choline Chloride for IFALD
Choline

IV choline chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN) who have intestinal failure-associated liver disease (IFALD).

Choline is a known important substrate for phospholipids that are critical for healthy liver function. 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 often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel / liver transplant. If approved, IV choline chloride would be the first approved therapy for IFALD. It has been granted Orphan Drug Designations (ODDs) by the FDA for the treatment of IFALD and the prevention of choline deficiency in PN patients.

Protara had an end of Phase 2 meeting with the FDA in late 2018 and received the FDA’s support to advance IV choline chloride into a registration-enabling study for the treatment of IFALD.

The mechanism for which choline supplementation reverses steatosis and improves cholestasis is not completely understood. However, clinical studies support that exogenous supplementation of choline can restore normal physiologic levels of choline thereby reversing steatosis by playing an important role in the synthesis of very low-density lipoprotein (VLDL), which is necessary to move fat out of the liver. Additionally, choline supplementation plays a critical role in the production of normal, healthy mixed micelles in bile, which protects against cholestatic injury to the biliary system.

Intestinal failure-associated liver disease (IFALD), which occurs in patients dependent on PN support, is characterized by choline deficiency, hepatic steatosis, cholestasis, and rapid progression of liver disease through to hepatic failure and death in the absence of intestine-liver transplant. IFALD carries a relatively poor prognosis, with a 15-34% death rate within 1-4 years. When IFALD presents with symptoms of liver disease in children, mortality is even higher (23–40%).

IFALD is uniquely characterized by the presence of both steatosis (toxic fat accumulation in liver cells) and cholestasis (damage to the biliary system in the liver) in patients who are chronic (greater than six months) PN users. A patient is considered to have IFALD if she/he is dependent on PN for more than six months (e.g., has chronic intestinal failure); has evidence of steatosis, determined by imaging techniques or histologic assessments; has evidence of cholestasis (e.g., elevated alkaline phosphatase (ALP), elevated bilirubin and/or histology); and may have evidence of ongoing, progressive liver injury on the basis of multiple abnormal liver function tests, in conjunction with findings of fibrosis, cirrhosis, and/or end-stage liver disease (ESLD).

Expanded Access Policy

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 info@protaratx.com.

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