TARA-002 Delivers Robust Clinical Success in Pediatric Lymphatic Malformations: Interim STARBORN-1 Trial Results Stand Out
Clinical Success Rates Surpass 80%, Marking a Significant Milestone for Pediatric Patients
The latest interim data from Protara Therapeutics’ ongoing Phase 2 STARBORN-1 trial reveals a remarkable response for TARA-002, their investigational therapy for lymphatic malformations (LMs) in pediatric patients. Among the children evaluated, 80% who completed treatment and a full 100% who reached the eight-week assessment achieved clinical success—offering hope for a population with few approved treatment options and high surgical risk.
Most Responders Saw Results After Just One or Two Doses
The trial enrolled twelve patients as of November 12, 2025. Out of these, eight patients had completed the eight-week response assessment, with the majority (7 out of 8) achieving clinical success after just one or two doses of TARA-002. Only a single patient, presenting with a large 1,739 ml macrocystic LM, required all four doses but achieved a complete response by the trial's definition. Importantly, two patients assessed at 32 weeks remained disease-free, highlighting the potential durability of treatment.
| Outcome | Results |
|---|---|
| Clinical success (patients completing treatment) | 80% (8/10) |
| Clinical success (8-week assessment) | 100% (8/8) |
| Macrocystic complete response rate | 83% (5/6) |
| Mixed cystic complete response | 1/1 patient |
| Durable response (32 weeks disease-free) | 2 patients |
Favorable Safety Profile: No Serious Adverse Events Noted
Alongside strong efficacy, TARA-002 showed a favorable safety and tolerability profile. The majority of adverse events (AEs) were mild to moderate, mainly presenting as swelling and fatigue. Notably, there were no serious adverse events reported, and only one patient discontinued due to a moderate (Grade 2) fatigue event. This contrasts favorably with the current treatment landscape, where many children endure invasive surgical procedures or off-label therapies with significant side effects.
TARA-002: A Targeted Option for a Rare, High-Burden Condition
Lymphatic malformations are rare congenital anomalies that frequently present in early childhood, most commonly in the head and neck region. They can cause complications such as airway obstruction, infection, and disfigurement. Despite the clinical need, no FDA-approved treatments currently exist—leaving patients with limited and often risky choices.
TARA-002 is a cell-based therapy derived from a genetically distinct, inactivated strain of Streptococcus pyogenes, developed from the same cell bank as OK-432—the Japanese standard of care for LMs for decades. Its promising results and rare pediatric disease designation highlight its significance as a potentially practice-changing intervention.
Looking Ahead: Conference Call, Further Enrollment, and Regulatory Momentum
Protara Therapeutics is hosting a webcast and conference call to discuss these interim results, offering further detail on both the data and the evolving LM treatment landscape. As the trial continues to enroll and gather more data, the strong early results will be closely watched by the medical and investment communities.
Key Takeaways: High Response Rates and a New Hope for Pediatric LMs
For parents and clinicians dealing with lymphatic malformations, the STARBORN-1 results signal the possibility of an effective, non-surgical therapy with lasting benefit and manageable side effects. With continued monitoring and additional data expected as enrollment progresses, TARA-002 is positioned to fill a critical therapeutic gap and potentially transform care standards for children with this rare condition.
For more information on the ongoing trial, visit clinicaltrials.gov (NCT05871970). Details and a replay of the conference call will be made available on Protara’s investor website.
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