Protara Therapeutics

Protara Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for cancer and rare diseases with significant unmet needs. Founded in 2006 by Jesse Shefferman and Jacqueline Zummo, the company operates under a model centered on advancing a targeted pipeline of investigational treatments through clinical trials to gain regulatory approval and eventual commercialization. As a publicly-traded entity on NASDAQ (TARA), its revenue generation is prospective, contingent on the success of its drug candidates. The company's financial strategy involves securing capital through public offerings to fund extensive research and development operations. A significant financing round in late 2024 provided Protara with a cash runway projected into 2027, de-risking its near-term development goals.

The company's co-founder, CEO, and President, Jesse Shefferman, has a background of over two decades in the biopharma industry, with experience in strategic and financial roles at companies like Retrophin Inc. and Vertex Pharmaceuticals, as well as in investment banking. This experience in rare diseases and finance has shaped Protara's strategy of acquiring and developing late-stage assets. Co-founder Dr. Jacqueline Zummo, the Chief Scientific Operations Officer, brings over 15 years of senior leadership in the biopharma industry, focusing on clinical development and regulatory affairs.

Protara's lead product candidate is TARA-002, an investigational cell therapy for non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs). TARA-002 is an immunopotentiator derived from a distinct strain of *Streptococcus pyogenes*, developed from the same master cell bank as OK-432, a therapy approved in Japan. Its mechanism of action involves stimulating a broad, pro-inflammatory immune response within the tumor microenvironment and inducing immunogenic cell death, which activates both innate and adaptive immunity to fight the cancer. Clinical trials are underway to evaluate its efficacy and safety, with promising early data showing high complete response rates in NMIBC patients.

The second key asset in Protara's portfolio is IV Choline Chloride, an intravenous phospholipid substrate replacement therapy. It is being developed for patients with intestinal failure-associated liver disease (IFALD) who are dependent on parenteral nutrition and unable to absorb sufficient choline, an essential nutrient for liver function. Choline deficiency is a key factor in the development of IFALD, a serious condition that can lead to hepatic failure. IV Choline Chloride, which has received Orphan Drug and Fast Track designations from the FDA, aims to be the first approved therapy for this condition by providing a direct source of this critical nutrient.

Keywords: Protara Therapeutics, TARA-002, IV Choline Chloride, non-muscle invasive bladder cancer, NMIBC, lymphatic malformations, intestinal failure-associated liver disease, IFALD, clinical-stage biopharmaceutical, cell therapy, immunopotentiator, Streptococcus pyogenes, OK-432, phospholipid substrate replacement, parenteral nutrition, choline deficiency, orphan drug, Jesse Shefferman, Jacqueline Zummo, oncology, rare diseases, NASDAQ:TARA