Protara Therapeutics Reports Q1 2024 Financial Results and Business Update

Protara Therapeutics, Inc., a clinical-stage biopharmaceutical company, disclosed its financial performance for the first quarter ending March 31, 2024, alongside significant progress updates on its ongoing programs. Protara's chief executive, Jesse Shefferman, underscored the company’s advancements in 2024, particularly in their oncology and rare disease portfolios. With cash resources projected to sustain operations into 2026, the company is confident in its strategic direction.

One key highlight was the encouraging data observed in a three-month evaluation of patients with non-muscle invasive bladder cancer (NMIBC) treated with TARA-002. This clinical program revealed a 38% overall complete response (CR) rate among 16 evaluable patients, with 63% CR in patients with carcinoma in situ (CIS) and varying response rates based on Bacillus Calmette-Guérin (BCG) status. The safety profile of TARA-002 was favorable, with no severe treatment-related adverse events reported. Protara plans to present interim data from the ongoing ADVANCED-2 trial in NMIBC patients in the latter half of 2024.

In an important step for their program focused on IV Choline Chloride, Protara has aligned with the FDA on a registration pathway for patients reliant on parenteral nutrition. This broadened the scope from their initial focus on intestinal failure-associated liver disease (IFALD) to include all patients unable to synthesize choline independently. This move could potentially address the needs of an estimated 40,000 patients in the U.S. who depend on long-term parenteral nutrition.

The company continues to enroll pediatric patients in the Phase 2 STARBORN-1 trial, which tests TARA-002 for lymphatic malformations (LMs). This rare condition lacks FDA-approved therapies, and the trial aims to enroll about 30 patients to examine the efficacy of up to four injections of TARA-002. The primary endpoint is a significant reduction in LM volume, measured through clinical success criteria.

Financially, Protara concluded the first quarter of 2024 with $55.2 million in cash, equivalents, and marketable securities. Additionally, a private placement in April 2024 generated approximately $42.0 million in net proceeds, extending operational funding through 2026. Research and development expenses rose to $7.7 million, reflecting increased clinical and personnel-related activities for TARA-002. Conversely, general and administrative expenses saw a reduction to $4.1 million. Overall, the company reported a net loss of $11.1 million for Q1 2024, compared to $9.0 million in the same period the previous year.

TARA-002 is derived from the same master cell bank as OK-432, an immunopotentiator marketed as Picibanil® in Japan. Protara’s research suggests TARA-002 activates immune cells within tumors, leading to an inflammatory response that kills tumor cells and enhances the antitumor immune response.

Bladder cancer remains a significant health issue in the U.S., with NMIBC comprising 80% of bladder cancer cases. Meanwhile, lymphatic malformations are rare congenital anomalies primarily diagnosed in early childhood, often leading to severe health complications.

IV Choline Chloride is being developed as a vital phospholipid substrate for patients on parenteral nutrition, given the absence of choline in current PN formulations. Recognized for its critical role in liver function and overall health, an IV formulation of choline has the potential to significantly benefit PN-dependent patients who are currently choline-deficient.

Protara Therapeutics is dedicated to advancing novel therapies for cancer and rare diseases, with a robust pipeline that includes TARA-002 for NMIBC and LMs, and IV Choline Chloride for patients on parenteral nutrition. The company continues to pursue regulatory and clinical milestones, aiming to address unmet medical needs in these areas.