Protara Therapeutics Announces Completion of First Cohort in Phase 2 STARBORN-1 Trial of TARA-002 in Pediatric Patients with Lymphatic Malformations

Rhea-AI Summary

Protara Therapeutics (Nasdaq: TARA) has completed the first cohort of its Phase 2 STARBORN-1 trial for TARA-002, an investigational cell-based immunopotentiator for treating pediatric lymphatic malformations (LMs). The initial results are promising, with two out of three patients achieving complete responses after a single dose. The safety profile was consistent with historical data, showing mild to moderate, resolvable adverse events.

The trial aims to enroll about 30 patients aged 6 months to 18 years, with initial results from the next cohort expected in 1H'25. The primary endpoint is the proportion of participants demonstrating clinical success, defined as a substantial or complete reduction in LM volume. This development is significant as there are currently no FDA-approved treatments for LMs, a rare condition primarily affecting children.

Positive

• Two out of three patients in the first cohort achieved complete response after one dose of TARA-002
• Safety and tolerability profile consistent with historical experience
• Enrollment in additional cohorts underway with positive investigator feedback
• Strong patient interest with waiting lists for subsequent cohorts

Negative

• Initial results to only three patients in the first cohort
• Full trial results not expected until first half of 2025

Insights

Medical Research Analyst

The completion of the first cohort in Protara's Phase 2 STARBORN-1 trial for TARA-002 marks a significant milestone. The initial efficacy data is particularly noteworthy, with two out of three patients achieving complete response after just one dose. This suggests potential cost-effectiveness and improved patient experience if fewer treatments are needed.

The safety profile aligns with historical data from OK-432, TARA-002's predecessor. While the sample size is small, the consistency in safety is reassuring. The mild to moderate and resolvable nature of the TEAEs is encouraging for pediatric use. However, we'll need to closely monitor larger cohorts for a more comprehensive safety assessment.

The waiting lists for subsequent cohorts indicate high interest from both physicians and patients, potentially signaling a smoother path for recruitment and future market adoption if approved.

Financial Analyst

Protara Therapeutics' progress with TARA-002 could significantly impact its financial outlook. The positive initial results may attract investor attention and potentially increase the company's valuation. However, it's important to note that full results aren't expected until 1H'25, which means a considerable waiting period for more comprehensive data.

The unmet medical need in pediatric lymphatic malformations presents a valuable market opportunity. If TARA-002 maintains its efficacy in larger cohorts, it could become a first-in-class FDA-approved treatment, potentially leading to substantial revenue streams. The high response rate and single-dose efficacy in some patients could translate to higher profit margins and lower treatment costs, enhancing both market penetration and reimbursement potential.

Investors should monitor cash burn rate and potential partnership or licensing deals as the trial progresses.

Pediatric Specialist

The initial results from the STARBORN-1 trial are promising for pediatric patients with lymphatic malformations. The complete response in two out of three patients, especially after a single dose, is remarkable. This could potentially reduce treatment burden on young patients and their families.

The inclusion of different age groups in the trial design is crucial. LMs often present in early childhood, so data on safety and efficacy across various age ranges will be valuable for clinical decision-making. The planned enrollment of patients as young as six months is particularly important.

The primary endpoint focusing on volume reduction is clinically relevant, as it often correlates with symptom improvement. However, future studies should also consider quality of life measures and long-term outcomes to fully assess the impact of TARA-002 on pediatric patients' overall well-being.

• TARA-002 demonstrated compelling efficacy and was generally well-tolerated

• Enrollment in additional cohorts underway with initial results expected in 1H’25
  

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 completion of the first cohort of the Phase 2 STARBORN-1 trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of pediatric patients with lymphatic malformations (LMs). Enrollment is now underway in additional cohorts.

“The initial data is compelling and reflective of the significant benefit observed in previous studies with OK-432, the predecessor of TARA-002,” said Nancy Bauman, MD, Children's National Medical Center: Children's Research Institute, Washington DC, and investigator for the STARBORN-1 trial. “There is a pressing need for an effective therapeutic option for LMs, a rare condition mainly affecting children for which there are currently no U.S. FDA approved agents. I remain excited about the potential for TARA-002 to play a meaningful role in the treatment of these patients and look forward to future learnings from this important study.”

Of three patients treated in the first cohort, which enrolled individuals six years to less than 18 years of age, two patients treated with TARA-002 achieved a complete response after receiving one dose of TARA-002; the responses were seen in a patient with a macrocystic lymphatic malformation and a patient with a ranula. The safety and tolerability seen in this cohort was consistent with that of the historical experience with OK-432 and included treatment emergent adverse events (TEAEs) of pain, swelling, fatigue, and body temperature increases. All TEAEs were mild to moderate and resolved.

“We are pleased with the progress of our Phase 2 STARBORN-1 trial and the encouraging results we have seen thus far,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We have received positive feedback from our investigators and already have a number of patients on waiting lists for our subsequent cohorts, and expect to share initial results from our next cohort in the first half of 2025.”

STARBORN-1 is a Phase 2 single-arm, open-label, prospective clinical trial evaluating the safety and efficacy of intracystic injection of TARA-002 for the treatment of macrocystic and mixed cystic LMs (≥ 50% macrocystic disease) in participants six months to less than 18 years of age. The trial will enroll approximately 30 patients including age de-escalation safety lead-in cohorts of children ages six years to less than18 years, two years to less than six years, and six months to less than two years, who will receive up to four injections of TARA-002 spaced approximately six weeks apart.

The primary endpoint of the trial is the proportion of participants with macrocystic and mixed cystic LMs who demonstrate clinical success, defined as having either a complete response (90% to 100% reduction from baseline in total LM volume) or substantial response (60% to less than 90% reduction in total LM volume) as measured by axial imaging.

About TARA-002 in LMs

TARA-002 is an investigational cell therapy based on the broad immunopotentiator, OK-432, which was originally granted marketing approval by the Japanese Ministry of Health and Welfare as an immunopotentiating cancer therapeutic agent. This cell therapy is currently approved in Japan and Taiwan for LMs and has been used to successfully treat thousands of pediatric patients with this rare condition. In addition, OK-432 was studied in the largest ever conducted Phase 2 trials in LMs, in which the therapy was administered via a now-closed compassionate use program led by the University of Iowa.

TARA-002 has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of LMs.

About Lymphatic Malformations

Lymphatic malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of three years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection; and cosmetic and other functional disabilities.

About Protara Therapeutics, Inc.

Protara is committed to advancing transformative therapies for people with cancer and rare diseases. Protara’s portfolio includes its lead program, TARA-002, an investigational cell-based therapy being developed for the treatment of non-muscle invasive bladder cancer and lymphatic malformations, and IV Choline Chloride, an investigational phospholipid substrate replacement for patients dependent on parenteral support. 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; the loss of key members of management; 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 are the initial results of Protara Therapeutics' STARBORN-1 trial for TARA-002?

In the first cohort of three patients aged 6-18 years, two achieved complete response after one dose of TARA-002. The treatment was generally well-tolerated with mild to moderate adverse events that resolved.

When are the next results expected for Protara Therapeutics' TARA-002 trial?

Protara Therapeutics expects to share initial results from the next cohort of the STARBORN-1 trial in the first half of 2025.

What is the primary endpoint of the STARBORN-1 trial for TARA-002?

The primary endpoint is the proportion of participants who demonstrate clinical success, defined as either a complete response (90-100% reduction in LM volume) or substantial response (60-90% reduction in LM volume) as measured by axial imaging.

How many patients will the STARBORN-1 trial for TARA-002 enroll?

The STARBORN-1 trial aims to enroll approximately 30 patients, including age de-escalation safety lead-in cohorts of children from 6 months to less than 18 years old.