Protara Therapeutics disclosed that its Phase 2 STARBORN‑1 study, which evaluates the intracystic injection of TARA‑002 in children with macrocystic and mixed cystic lymphatic malformations, achieved a 100 % clinical success rate among the eight patients who were evaluable at the eight‑week assessment. The trial enrolled 12 patients, and all eight evaluable patients met the primary endpoint of a ≥60 % reduction in total lesion volume. Eight of the ten patients who completed the full treatment course also achieved this threshold, and one patient required four doses to reach a complete response (≥90 % reduction).
Safety data were favorable: the majority of adverse events were mild to moderate, with swelling and fatigue the most common. No serious adverse events were reported, and only one patient discontinued treatment because of a Grade 2 fatigue event. The data cutoff for the analysis was November 12, 2025, and the results are interim, pending further follow‑up.
TARA‑002 is derived from the same master cell bank as OK‑432, a therapy that has been used in Japan for three decades to treat lymphatic malformations. Protara’s drug has received a Rare Pediatric Disease designation from the FDA, positioning it for potential priority review and market exclusivity if it gains approval. Because no approved therapies exist for this condition, the trial results represent a significant de‑risking event for the company’s pipeline and open a sizable unmet‑need market.
The positive interim data support Protara’s strategy to bring a first‑in‑class therapy to a rare pediatric population. The company’s cash runway extends to mid‑2027, but the trial’s small sample size and interim nature mean that additional capital will likely be required to advance the program through later‑stage studies and regulatory submissions. The results also reinforce the broader pipeline, which includes programs for non‑muscle invasive bladder cancer and an intravenous choline chloride formulation.
The market reaction to the announcement was mixed, with some reports noting a surge in interest while others reported a decline. Analysts have reiterated their positive outlook on the company’s prospects, but no specific price targets or ratings are disclosed in this report.
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