Protara Therapeutics is focused on developing innovative therapies for rare diseases and oncology, with two primary investigational products: TARA-002 and IV Choline Chloride. TARA-002 is a cell therapy derived from the broad immunopotentiator OK-432, which is already approved in Japan for various oncological conditions and lymphatic malformations. This therapy is currently being evaluated for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) in the U.S. The mechanism of action for TARA-002 involves activating both innate and adaptive immune responses, leading to the release of pro-inflammatory cytokines and the direct killing of tumor cells. This dual action aims to enhance the body’s antitumor immune response, addressing the urgent need for effective treatments in NMIBC and LMs, which currently lack approved therapies.

IV Choline Chloride is another investigational therapy aimed at addressing choline deficiency in patients on parenteral nutrition (PN). Given that approximately 80% of PN patients are choline deficient, this therapy has the potential to be the first FDA-approved intravenous choline formulation for these patients. Choline plays a critical role in liver function and overall health, and its deficiency can lead to severe liver damage. The proposed mechanism of action for IV Choline Chloride involves restoring normal choline levels, which may help reverse liver steatosis and improve cholestasis. Protara has received Orphan Drug Designation from the FDA for this therapy, highlighting its significance in treating a population with limited options.

United States

Protara Therapeutics, Inc., a clinical-stage company, engages in the identifying and advancing transformative therapies for people with rare and specialty diseases. The company's lead program is TARA-002, an investigational cell therapy for the treatment of lymphatic malformations. It also develops intravenous choline chloride, an investigational phospholipid substrate replacement therapy for the treatment of intestinal failure associated liver disease. The company was formerly known as ArTara Therapeutics, Inc. and changed its name to Protara Therapeutics, Inc. in May 2020. Protara Therapeutics, Inc. is based in New York, New York.

Cancer

Liver Diseases

Lymphatic Diseases

Rare diseases

Protara Therapeutics

Intestinal failure associated liver disease

Liver disease

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

IV Choline chloride

Bladder cancer

Non-muscle invasive bladder cancer

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and LMs for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. 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. Protara has successfully demonstrated manufacturing comparability between TARA-002 and OK-432.

TARA-002

Lymphatic malformations

Solid tumor/s

When TARA-002 is administered, it is hypothesized that innate and &nbsp;adaptive immune cells within the cyst or tumor are activated and produce &nbsp;a strong immune cascade. Neutrophils, monocytes and lymphocytes &nbsp;infiltrate the abnormal cells and various cytokines, including &nbsp;interleukins IL-6, IL-8, IL-12, interferon (IFN)-gamma, tumor necrosis &nbsp;factor (TNF)-alpha, and vascular endothelial growth factor (VEGF) are &nbsp;secreted by immune cells to induce a strong local inflammatory reaction &nbsp;and destroy the abnormal cells.

Protara Therapeutics

Company Details