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Protara Therapeutics Reports Strong Early Results for Lymphatic Malformation Treatment

MarketDash Editorial Team
18 days ago
Protara Therapeutics released interim data from its Phase 2 trial showing TARA-002, a cell-based therapy, achieved clinical success in 80% of pediatric patients with lymphatic malformations, with most responding after just one or two doses.

Protara Therapeutics Inc. (TARA) shared some encouraging news Wednesday from its ongoing Phase 2 trial testing TARA-002, an investigational cell-based therapy designed to treat a tricky congenital condition in kids.

The trial, called STARBORN-1, is evaluating how well intracystic injections of TARA-002 work in pediatric patients dealing with macrocystic and mixed cystic lymphatic malformations. These are essentially benign but abnormal growths of dilated lymphatic vessels that can pop up anywhere in the body, though they're most commonly found in the head and neck region. The "macrocystic" and "mixed cystic" labels refer to the size of the cysts these malformations create.

The interim analysis covered 12 patients total, each receiving up to four injections of TARA-002 spaced roughly six weeks apart. Here's where things get interesting: among the eight evaluable patients, seven achieved clinical success with just one or two doses. That's pretty remarkable efficiency for a treatment tackling congenital abnormalities.

The one outlier presented with a massive 1,739 ml macrocystic lymphatic malformation and needed all four doses, but ultimately achieved a complete response anyway.

When you zoom out to look at patients who finished therapy, the numbers look solid. Clinical success was achieved in 80% of cases (8 out of 10), and every single patient who made it to the eight-week assessment mark met the clinical success criteria. That's a 100% success rate among completers.

Breaking down the macrocystic cases specifically: 83% (5 of 6 patients) achieved complete response with lymphatic malformation volume dropping by 90-100%. The sixth patient still saw substantial improvement with a 60% to just under 90% reduction. The lone mixed cystic patient treated also achieved complete response. Two patients have now reached the 32-week post-treatment mark and remain disease-free.

Now, clinical trials rarely go perfectly, and this one had its complications. One patient initially deemed a complete response was later diagnosed with a ranula, which is actually a different type of maxillofacial cyst unrelated to lymphatic malformations.

Two patients withdrew before hitting the eight-week assessment. One turned out to be misdiagnosed and actually had a rare cancer form that didn't respond to treatment. The other patient dropped out after seeing notable improvement in their macrocystic condition—they'd received two doses with aspiration volumes dropping from 160 ml at the first dose down to just 10 ml at the second, suggesting real progress.

On the safety front, most adverse events were mild to moderate, with no serious adverse events reported. The most common issues were swelling and fatigue. One patient did discontinue treatment because of Grade 2 fatigue.

Price Action: TARA stock was down 2.88% to $6.555 at last check Wednesday.

Back to News

Protara Therapeutics Reports Strong Early Results for Lymphatic Malformation Treatment

MarketDash Editorial Team
18 days ago
Protara Therapeutics released interim data from its Phase 2 trial showing TARA-002, a cell-based therapy, achieved clinical success in 80% of pediatric patients with lymphatic malformations, with most responding after just one or two doses.

Protara Therapeutics Inc. (TARA) shared some encouraging news Wednesday from its ongoing Phase 2 trial testing TARA-002, an investigational cell-based therapy designed to treat a tricky congenital condition in kids.

The trial, called STARBORN-1, is evaluating how well intracystic injections of TARA-002 work in pediatric patients dealing with macrocystic and mixed cystic lymphatic malformations. These are essentially benign but abnormal growths of dilated lymphatic vessels that can pop up anywhere in the body, though they're most commonly found in the head and neck region. The "macrocystic" and "mixed cystic" labels refer to the size of the cysts these malformations create.

The interim analysis covered 12 patients total, each receiving up to four injections of TARA-002 spaced roughly six weeks apart. Here's where things get interesting: among the eight evaluable patients, seven achieved clinical success with just one or two doses. That's pretty remarkable efficiency for a treatment tackling congenital abnormalities.

The one outlier presented with a massive 1,739 ml macrocystic lymphatic malformation and needed all four doses, but ultimately achieved a complete response anyway.

When you zoom out to look at patients who finished therapy, the numbers look solid. Clinical success was achieved in 80% of cases (8 out of 10), and every single patient who made it to the eight-week assessment mark met the clinical success criteria. That's a 100% success rate among completers.

Breaking down the macrocystic cases specifically: 83% (5 of 6 patients) achieved complete response with lymphatic malformation volume dropping by 90-100%. The sixth patient still saw substantial improvement with a 60% to just under 90% reduction. The lone mixed cystic patient treated also achieved complete response. Two patients have now reached the 32-week post-treatment mark and remain disease-free.

Now, clinical trials rarely go perfectly, and this one had its complications. One patient initially deemed a complete response was later diagnosed with a ranula, which is actually a different type of maxillofacial cyst unrelated to lymphatic malformations.

Two patients withdrew before hitting the eight-week assessment. One turned out to be misdiagnosed and actually had a rare cancer form that didn't respond to treatment. The other patient dropped out after seeing notable improvement in their macrocystic condition—they'd received two doses with aspiration volumes dropping from 160 ml at the first dose down to just 10 ml at the second, suggesting real progress.

On the safety front, most adverse events were mild to moderate, with no serious adverse events reported. The most common issues were swelling and fatigue. One patient did discontinue treatment because of Grade 2 fatigue.

Price Action: TARA stock was down 2.88% to $6.555 at last check Wednesday.