PATIENTS
Committed to Making a Difference
At Protara, our mission is simple: to improve lives. We put patients at the heart of everything we do, working side by side with doctors, researchers, and advocacy groups. Together, we are focused on developing new therapies that bring hope and meaningful change for people living with cancer and rare diseases.
Non-Muscle Invasive Bladder Cancer (NMIBC)
Bladder cancer is one of the most common cancers in the U.S. Each year, more than 84,000 people are expected to be diagnosed. About three out of four cases are a type called non-muscle invasive bladder cancer (NMIBC). In NMIBC, the cancer is found in the inner lining of the bladder but has not spread into the bladder wall muscle.
NMIBC often comes back after treatment and up to 80% of patients will experience recurrence. Some people may face disease progression and eventually require major surgery, such as bladder removal. These challenges highlight the urgent need for better, less invasive options.
Protara is developing TARA-002, an investigational therapy designed to activate the body’s immune system to help fight bladder cancer cells. TARA-002 is currently being studied in a Phase 2 clinical trial for patients with high-grade NMIBC who have not responded to, or have not yet received, BCG (a standard treatment).
Lymphatic Malformations (LMs)
Lymphatic malformations are rare, non-cancerous cysts that occur when lymph vessels do not form or drain properly. They are often diagnosed early in life: more than half are found at birth, and 90% are identified by age two. While not malignant, LMs can cause serious challenges such as difficulty breathing, swallowing, or speaking.
Currently, there are no approved therapies for LMs. Surgery is sometimes considered but can carry a high risk of complications, and the malformation often comes back. Families need safer, more effective treatment options.
Protara is studying TARA-002 as a potential therapy for pediatric patients with lymphatic malformations. This cell therapy was developed from the same master cell bank as OK-432, which has already been used for years in Japan, where it is considered a standard of care. We are now evaluating TARA-002 in a Phase 2 clinical trial for children with LMs.
Choline Deficiency
Choline is an essential nutrient that supports liver health, brain function, mood, and muscle control. People who rely on parenteral support (PS) or parenteral nutrition (PN) delivered directly into the bloodstream often do not get enough choline. In fact, a recent study found that approximately 78% of long-term PS patients are choline deficient, and more than half develop liver problems as a result.
Despite clinical guidelines recommending choline supplementation, there are currently no approved intravenous (IV) choline products available for patients who depend on parenteral support.
Protara is developing IV Choline Chloride, an investigational therapy designed to fill this gap. It is being studied in a Phase 2b/3 clinical trial for adolescents and adults with intestinal failure who rely on long-term PS.
