Protara Highlights Recent Updates and Anticipated 2025 Milestones

Rhea-AI Summary

Protara Therapeutics highlights recent updates and anticipated milestones for 2025. The company reported positive six-month data from the ADVANCED-2 trial of TARA-002 in NMIBC patients, showing a 72% complete response (CR) rate at six months and a favorable safety profile. Data from 12-month evaluable patients is expected mid-2025, with futility analysis results by the end of 2025.

Protara completed a $100 million public offering, extending its financial runway into 2027. The THRIVE-3 registrational trial of IV Choline Chloride in patients dependent on parenteral support is set to begin in the first half of 2025. Initial results from the Phase 2 STARBORN-1 trial of TARA-002 in pediatric LMs patients are expected by the end of the first half of 2025.

The company remains focused on developing therapies for cancer and rare diseases, with ongoing efforts in combination therapies and regulatory alignments for future trials.

Positive

• Reported 72% CR rate at six months in ADVANCED-2 trial of TARA-002 in NMIBC.
• No Grade 2 or greater treatment-related adverse events in the ADVANCED-2 trial.
• Completed $100 million public offering, extending financial runway into 2027.
• THRIVE-3 registrational trial of IV Choline Chloride to begin in 1H 2025.

Negative

• No data or results from the 12-month evaluable patients in ADVANCED-2 trial until mid-2025.
• Results from futility analysis of six-month evaluable BCG-Unresponsive patients expected only by end of 2025.

Insights

Financial Analyst

The $100 million public offering significantly strengthens Protara's financial position, extending cash runway into 2027. With $181.5 million in total liquidity, the company is well-capitalized to advance its clinical programs. The positive interim data from ADVANCED-2 trial showing a 72% complete response rate at six months positions TARA-002 as a potentially valuable asset in the $3.8 billion bladder cancer treatment market.

The registrational trial alignment with FDA guidance for BCG-Unresponsive NMIBC enhances regulatory clarity and potentially accelerates the path to market. Multiple clinical catalysts in 2025, including 12-month NMIBC data and Phase 2 LM results, could drive significant value creation. The company's diversified pipeline across NMIBC, LMs and IV Choline Chloride provides multiple shots on goal.

Medical Research Analyst

The ADVANCED-2 trial data is particularly impressive, with a 72% complete response rate at six months and perfect retention of response from 3 to 6 months in all 9 patients. The clean safety profile with no Grade 2+ treatment-related adverse events is noteworthy for NMIBC treatments. The systemic priming approach and combination therapy exploration could potentially expand TARA-002's therapeutic utility.

The THRIVE-1 study results revealing 78% choline deficiency in PS-dependent patients, with 63% showing liver dysfunction, validates the medical need for IV Choline Chloride. This positions the upcoming THRIVE-3 registrational trial as addressing a critical unmet need in parenteral nutrition.

Market Research Analyst

With multiple clinical readouts in 2025, including pivotal NMIBC data and Phase 2 LM results, Protara is positioned for potential value-driving catalysts. The BCG-Unresponsive NMIBC market represents a significant opportunity, as current treatment options are TARA-002's strong efficacy data and favorable safety profile could support meaningful market penetration.

The Fast Track designation for IV Choline Chloride highlights its potential importance in addressing nutritional deficiencies in PS-dependent patients. The company's strategic focus on both oncology and rare diseases provides diversification and multiple commercialization opportunities. The extended cash runway reduces near-term financing risk and allows for proper execution of clinical programs.

Reported positive six-month data from ADVANCED-2 trial of TARA-002 in patients with NMIBC

Completed approximately $100 million public offering, extending runway into 2027

Initial data from 12-month evaluable NMIBC patients in ADVANCED-2 trial expected in mid-2025; Results from a futility analysis of approximately 25 six-month evaluable BCG-Unresponsive patients expected by the end of 2025

Dosing of first patient in THRIVE-3 registrational trial of IV Choline Chloride in patients dependent on parenteral support expected in 1H 2025

Results from additional cohorts of Phase 2 STARBORN-1 trial of TARA-002 in pediatric LMs patients expected by the end of 1H 2025

NEW YORK, Jan. 13, 2025 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today highlighted recent updates and anticipated 2025 milestones.

“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.
  

About Protara Therapeutics, Inc.

Protara is a clinical-stage biotechnology company committed to advancing transformative therapies for people with cancer and rare diseases. Protara’s portfolio includes its lead candidate, TARA-002, an investigational cell-based therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs). The Company is evaluating TARA-002 in an ongoing Phase 2 trial in NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin (BCG), as well as a Phase 2 trial in pediatric patients with LMs. Additionally, Protara is developing IV Choline Chloride, an investigational phospholipid substrate replacement for patients on parenteral support who are otherwise unable to meet their choline needs via oral or enteral routes. For more information, visit www.protaratx.com.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words or expressions referencing future events, conditions or circumstances that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include but are not limited to, statements regarding Protara’s intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: Protara’s business strategy, including its development plans for its product candidates and plans regarding the timing or outcome of existing or future clinical trials (including reporting initial data from 12-month evaluable patients in mid-2025); statements related to expectations regarding interactions with the FDA; Protara’s financial position; statements regarding the anticipated safety or efficacy of Protara’s product candidates; and Protara’s outlook for the remainder of the year and future periods. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that contribute to the uncertain nature of the forward-looking statements include: risks that Protara’s financial guidance may not be as expected, as well as risks and uncertainties associated with: Protara’s development programs, including the initiation and completion of non-clinical studies and clinical trials and the timing of required filings with the FDA and other regulatory agencies; general market conditions; changes in the competitive landscape; changes in Protara’s strategic and commercial plans; Protara’s ability to obtain sufficient financing to fund its strategic plans and commercialization efforts; having to use cash in ways or on timing other than expected; the impact of market volatility on cash reserves; failure to attract and retain management and key personnel; the impact of general U.S. and foreign, economic, industry, market, regulatory, political or public health conditions; and the risks and uncertainties associated with Protara’s business and financial condition in general, including the risks and uncertainties described more fully under the caption “Risk Factors” and elsewhere in Protara's filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Protara undertakes no obligation to update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise, except as required by law.

Company Contact:

Justine O'Malley
Protara Therapeutics
Justine.OMalley@protaratx.com
646-817-2836

FAQ

What does the TARA-002 six-month data mean for NMIBC patients?

The six-month data from the ADVANCED-2 trial of TARA-002 in NMIBC patients showed a 72% complete response rate, indicating a promising treatment option with a favorable safety profile.

When is Protara expected to report 12-month data for TARA-002 in NMIBC?

Protara expects to report data on 12-month evaluable patients in the ADVANCED-2 trial of TARA-002 in NMIBC by mid-2025.

How will the $100 million public offering impact Protara's financial stability?

The $100 million public offering extends Protara's financial runway into 2027, providing sufficient funds to support planned operations and development programs.

When will the THRIVE-3 trial of IV Choline Chloride begin?

The THRIVE-3 registrational trial of IV Choline Chloride is expected to begin in the first half of 2025.

What are the anticipated milestones for Protara in 2025?

Anticipated milestones include reporting 12-month data for TARA-002 in NMIBC mid-2025, results from futility analysis of BCG-Unresponsive patients by end of 2025, and initial results from the STARBORN-1 trial of TARA-002 in pediatric LMs by the end of the first half of 2025.

What is the significance of the THRIVE-3 trial for Protara?

The THRIVE-3 trial is a pivotal study for IV Choline Chloride in patients dependent on parenteral support, potentially addressing a significant unmet need for this patient population.

What were the results from the THRIVE-1 study?

The THRIVE-1 study found that 78% of PS-dependent patients were choline deficient, with 63% demonstrating liver dysfunction, highlighting the need for IV Choline Chloride treatment.