On January 13, 2025 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported recent updates and anticipated 2025 milestones (Press release, Protara Therapeutics, JAN 13, 2025, View Source [SID1234649671]).

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"Following a highly productive 2024 marked by positive data in our non-muscle invasive bladder cancer (NMIBC) program and with the funds from a successful financing, we are well positioned to accelerate our development programs and deliver on our mission to provide novel therapies to patients impacted by cancer and rare diseases," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "Looking ahead, we expect several key milestones in 2025, including reporting data on 12-month evaluable patients in our ADVANCED-2 trial of TARA-002 in NMIBC mid-year. On the heels of our recently reported positive interim data from six-month evaluable patients, we continue to believe that TARA-002 could represent a meaningful and differentiated addition to the NMIBC treatment paradigm with an attractive product profile for both physicians and patients."

"In addition, we expect to begin the pivotal THRIVE-3 study of intravenous (IV) Choline Chloride in the first half of 2025. We also expect data from our ongoing Phase 2 STARBORN-1 trial of TARA-002 in lymphatic malformations (LMs) by the end of the first half of 2025."

Recent Company Updates and Planned 2025 Milestones

TARA-002 in NMIBC

In December 2024, the Company reported positive interim results from its ongoing Phase 2 open-label ADVANCED-2 trial in in NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-Unresponsive and BCG-Naïve at the 25th Annual Meeting of the Society of Urologic Oncology (SUO) in Dallas, Texas. The complete response (CR) rate across BCG exposures was 72% (13/18) at six months and 70% (14/20) at any time, with 100% (9/9) of patients maintaining a CR from three months to six months. In addition, two of three patients maintained a CR at nine months. TARA-002 showed a favorable safety profile, with no Grade 2 or greater treatment-related adverse events and no treatment discontinuations due to adverse events.
The Company expects to report data on 12-month evaluable patients in the ADVANCED-2 trial in mid-2025 and results from a futility analysis of approximately 25 six-month evaluable BCG-Unresponsive patients are expected by the end of 2025. As previously announced, the BCG-Unresponsive cohort is designed to be registrational in alignment with the 2024 BCG-Unresponsive Non-muscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment Draft Guidance for Industry issued by the U.S. Food and Drug Administration (FDA).
The Company expects to provide an update on the design of its planned BCG-Naïve registrational trial by the end of the first half of 2025 following regulatory alignment.
The Company continues to explore the administration of systemic priming dosing prior to initiation of intravesical administration, as well as combination therapy with TARA-002 in NMIBC patients with CIS. Given TARA-002’s mechanism of action and safety profile, the Company believes it has strong potential for use in combination therapy and is working to finalize various opportunities for the clinical program.
IV Choline Chloride for Patients on Parenteral Support (PS)

The Company expects to commence the THRIVE-3 registrational trial of IV Choline Chloride, an investigational phospholipid substrate replacement, in adolescents and adults on long-term PS when oral or enteral nutrition is not possible, insufficient, or contraindicated, in the first half of 2025. IV Choline Chloride was previously granted Fast Track designation by the FDA as a source of choline for this patient population.
In September 2024, the Company announced results from THRIVE-1, a prospective, observational study, which found that 78% of PS-dependent patients were choline deficient, and 63% of these patients demonstrated liver dysfunction, including steatosis, cholestasis, and hepatobiliary injury.
TARA-002 in LMs

Protara remains on track to report initial results from additional cohorts in the Phase 2 STARBORN-1 trial of TARA-002 in pediatric patients with macrocystic and mixed cystic LMs by the end of the first half of 2025. The Company previously announced completion of the study’s first safety cohort, in which TARA-002 demonstrated encouraging efficacy and was generally well-tolerated.
Financial Guidance

The Company today provided updated financial guidance. Protara believes its approximately $81.5 million of cash, cash equivalents, and investments in marketable debt securities as of September 30, 2024, together with approximately $100 million gross proceeds from its December 2024 public offering, will be sufficient to fund its planned operations into 2027.