Protara Therapeutics Announces Results from THRIVE-1, a Prospective Observational Study Evaluating the Prevalence of Choline Deficiency and Liver Injury in Patients Dependent on Parenteral Support

Rhea-AI Summary

Protara Therapeutics (Nasdaq: TARA) announced results from THRIVE-1, a study evaluating choline deficiency and liver injury in patients dependent on parenteral support (PS). Key findings:

• 78% of PS-dependent patients were choline deficient
• 63% of choline-deficient participants had liver dysfunction

These results underscore the need for intravenous (IV) choline supplementation. Protara is developing IV Choline Chloride as a treatment when oral or enteral nutrition is insufficient. The company plans to initiate a registrational trial in Q1 2025. Currently, there are no approved IV choline products for PS-dependent patients globally, despite recommendations from medical societies.

Positive

• 78% of patients dependent on parenteral support were found to be choline deficient, indicating a significant market opportunity for IV Choline Chloride
• 63% of choline deficient participants showed liver dysfunction, highlighting the medical need for the treatment
• Protara plans to initiate a registrational trial for IV Choline Chloride in Q1 2025, progressing towards potential commercialization
• No approved IV choline products currently exist for PS-dependent patients globally, positioning Protara's treatment as a potential first-in-class therapy

Negative

• The company's pivotal PK trial for IV Choline Chloride is not expected to begin until Q1 2025, indicating a lengthy timeline before potential market approval
• As a clinical-stage company, Protara Therapeutics likely lacks revenue from product sales and may require additional funding to support ongoing research and development

Medical Research Analyst

The THRIVE-1 study results are highly significant for patients dependent on parenteral support (PS). The finding that 78% of PS-dependent patients are choline deficient, with 63% of those showing liver dysfunction, underscores a critical unmet medical need. This data strongly supports the potential value of Protara's IV Choline Chloride therapy.

The study's robust design, involving 78 patients across multiple centers, lends credibility to its findings. The high prevalence of choline deficiency and associated liver complications in this population suggests that IV Choline Chloride could have a substantial impact on patient outcomes. This aligns with recommendations from respected bodies like ASPEN and ESPEN, further validating the potential of this treatment approach.

Financial Analyst

Protara Therapeutics' announcement is promising from an investment perspective. The company is addressing a clear unmet need in a niche market, which could translate to strong demand if IV Choline Chloride gains approval. The planned initiation of the pivotal PK trial in Q1 2025 provides a clear timeline for potential value inflection points.

However, investors should note that the path to market still involves significant clinical and regulatory hurdles. The seamless Phase 2b/3 trial design could potentially accelerate development, but it's important to monitor the progress and results of this study. If successful, Protara could capture a unique market position, potentially leading to robust revenue streams in the future.

Clinical Nutritionist

The THRIVE-1 study results highlight a critical gap in current parenteral nutrition practices. Choline deficiency in 78% of PS-dependent patients is alarming, given its essential role in liver function and overall health. The high prevalence of liver dysfunction (63%) among choline-deficient patients further emphasizes the urgent need for intervention.

IV Choline Chloride could be a game-changer in clinical nutrition for PS-dependent patients. It addresses a fundamental nutritional deficiency that current PS formulations fail to meet adequately. The potential benefits extend beyond liver health to neurological function and muscle integrity, which could significantly improve the quality of life for these patients.

• 78% of patients dependent on parenteral support were choline deficient, with 63% of these patients demonstrating liver dysfunction, including steatosis, cholestasis, and hepatobiliary injury
• IV Choline Chloride could fill a significant unmet medical need for this population
  
• Company expects to dose first patient in pivotal PK trial for IV Choline Chloride in Q1 2025
  

NEW YORK, Sept. 09, 2024 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced the results of THRIVE-1, a prospective, observational study evaluating the prevalence of choline deficiency and liver injury in patients dependent on parenteral support (PS). Results from the study were featured during a poster session at the 46^th European Society for Clinical Nutrition and Metabolism Congress (ESPEN), from September 7, 2024, to September 10, 2024, in Milan.

The study found that 78% of patients who are dependent on PS were choline deficient, and that 63% of choline deficient participants had liver dysfunction, including steatosis, cholestasis, and hepatobiliary injury, underscoring the need for intravenous (IV) choline supplementation in this patient population. Protara is developing IV Choline Chloride, an investigational phospholipid substrate replacement therapy, as a source of choline when oral or enteral nutrition is not possible, insufficient, or contraindicated.

“Findings from this study reinforce that there are a significant number of people dependent on PS who may potentially benefit from treatment with IV Choline Chloride,” said Palle Bekker Jeppesen M.D., Ph.D., Clinical Professor, Department of Intestinal Failure and Liver Diseases, Rigshospitalet, Copenhagen University Hospital, Copenhagen, Denmark. “Choline is essential for PS dependent patients, as deficiency can lead to hepatic injury, neuropsychological impairment, muscle damage, and thrombotic abnormalities. Access to an IV formulation of choline has the potential to meaningfully impact patients for whom oral or enteral choline supplementation is not an option.”

“Currently, there are no approved IV choline products for patients dependent on PS globally, despite top professional medical societies in both the U.S. and Europe recommending choline treatment for these patients,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We remain committed to bringing the first approved IV formulation of choline to the PS community and look forward to initiating our registrational trial in the first quarter of 2025.”

THRIVE-1 Study and Results

THRIVE-1 was a multi-center, cross-sectional prospective, observational study involving 78 patients to assess the prevalence of choline deficiency and liver injury in adolescents and adult patients with intestinal failure who are dependent on PS.

78% (61/78) were choline deficient, with 63% (38/60) of these patients demonstrating liver dysfunction including steatosis, cholestasis, and signs of hepatobiliary injury.

IV choline is recommended for patients receiving PS by the American Society for Parenteral and Enteral Nutrition (ASPEN) Recommendations for Changes in Commercially Available Parenteral Multivitamin and Multi–Trace Element Products, as well as by ESPEN in its Guideline on Home Parenteral Nutrition.

Based on feedback from the U.S. Food and Drug Administration, Protara intends to assess the safety and efficacy of IV Choline Chloride in THRIVE-3, a seamless registrational Phase 2b/3 trial with dose confirmation followed by a double-blinded, randomized, placebo-controlled trial in adolescents and adults receiving parenteral support.

About IV Choline Chloride

IV Choline Chloride is an investigational, intravenous phospholipid substrate replacement therapy initially in development for patients receiving parenteral support (PS). Choline is a known important substrate for phospholipids that are critical for healthy liver function and also plays an important role in modulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function, and bone health. PS patients are unable to synthesize choline from enteral nutrition sources, and there are currently no available PS formulations containing choline. Approximately 80 percent of patients dependent on PS are choline-deficient and have some degree of liver damage, which can lead to hepatic failure. In the U.S. alone, there are approximately 40,000 patients on long-term parenteral support who could benefit from an IV formulation of choline. IV Choline Chloride has the potential to become the first U.S. Food and Drug Administration (FDA) approved IV choline formulation for PS patients. IV Choline Chloride has been granted Orphan Drug Designation by the FDA for the prevention of choline deficiency in PS patients. The Company was issued a U.S. patent claiming a choline composition with a term expiring in 2041.

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, 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; 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. 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 development 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
Source: Protara Therapeutics

FAQ

What were the key findings of Protara Therapeutics' THRIVE-1 study for TARA stock?

The THRIVE-1 study found that 78% of patients dependent on parenteral support were choline deficient, and 63% of these patients had liver dysfunction. These findings suggest a significant market opportunity for Protara's IV Choline Chloride treatment.

When does Protara Therapeutics (TARA) plan to start its pivotal trial for IV Choline Chloride?

Protara Therapeutics plans to dose the first patient in its pivotal PK trial for IV Choline Chloride in Q1 2025.

Are there any approved IV choline products for parenteral support-dependent patients?

No, currently there are no approved IV choline products for patients dependent on parenteral support globally, despite recommendations from medical societies in the U.S. and Europe.

What is the potential market for Protara Therapeutics' (TARA) IV Choline Chloride?

Based on the THRIVE-1 study results, 78% of patients dependent on parenteral support were choline deficient, indicating a potentially significant market for IV Choline Chloride as a treatment for this population.