We have established a portfolio of de-risked assets addressing rare diseases and oncology.

* TARA-002 Granted Rare Pediatric Disease Designation for the treatment of LMs
** Granted Orphan Drug Designations by the U.S. FDA


TARA-002 is an investigational cell therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs). TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil® in Japan and Taiwan by Chugai Pharmaceutical Co., Ltd.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a pro-inflammatory response with release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma, IL-1b, IL-6, IL-12, granulocyte-macrophage colony-stimulating factor (GM-CSF) and natural killer cells. TARA-002 also directly kills tumor cells and triggers a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response.

Bladder cancer is the 6th most common cancer in the United States, with non-muscle invasive bladder cancer (NMIBC) representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle. There is an urgent need for new treatments to address NMIBC as significant increases in recurrence, progression, and the number of reported patients needing cystectomies are being observed.
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 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 2 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. There are currently no approved therapies to treat LMs.

NMIBC: TARA-002 is currently being studied in a Phase 2 open-label clinical trial in NMIBC patients with high-grade carcinoma in situ.


LMs: TARA-002 is currently being studied in a Phase 2 open-label clinical trial for the treatment of macrocystic and mixed cystic LMs in pediatric patients.

IV Choline Chloride

IV Choline Chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN).

Choline is a known important substrate for phospholipids that are critical for healthy liver function and also plays an important role in modulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function, and bone health. PN patients are unable to synthesize choline from enteral nutrition sources, and there are currently no available PN formulations containing choline. Approximately 80 percent of PN-dependent patients are choline-deficient and have some degree of liver damage, which can lead to hepatic failure. There are currently no available PN formulations containing choline. In the U.S. alone, there are approximately 40,000 patients on long-term parenteral nutrition who would benefit from an IV formulation of choline. IV Choline Chloride has the potential to become the first FDA approved IV choline formulation for PN patients. IV Choline Chloride has been granted Orphan Drug Designation by the FDA for the prevention of choline deficiency in PN patients. The Company was issued a U.S. patent claiming a choline composition with a term expiring in 2041.

The mechanism for which choline supplementation reverses steatosis and improves cholestasis is not completely understood. However, clinical studies support that exogenous supplementation of choline can restore normal physiologic levels of choline thereby reversing steatosis by playing an important role in the synthesis of very low-density lipoprotein (VLDL), which is necessary to move fat out of the liver. Additionally, choline supplementation plays a critical role in the production of normal, healthy mixed micelles in bile, which protects against cholestatic injury to the biliary system.

Expanded Access Policy

Protara is committed to identifying and advancing transformative therapies for the treatment of cancer and rare diseases with significant unmet needs.

Protara advances its investigational therapies, TARA-002 and IV Choline Chloride, by continuing to study them in clinical trials designed to evaluate potential efficacy, safety, and tolerability. Additional information about our ongoing clinical trials can be found on ClinicalTrials.gov.

We maintain that the best way to make our investigational therapies available to patients is by enrolling and completing all of our clinical trials and, if those trials are successful, pursuing regulatory approval. However, in circumstances where patients are not eligible for our clinical trials and may not have options for alternative therapies, Protara evaluates requests to make its medicines available via expanded access for eligible patients. Expanded access, also called compassionate use, makes an investigational product available for treatment outside of clinical trials when no comparable or satisfactory alternative therapy option is available.

Expanded access will only be considered when certain conditions are met. These conditions include the following:

  • The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option.
  • The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition.
  • The patient is not eligible or cannot participate in a clinical trial.
  • A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available.
  • Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access.
  • Adequate supply of the investigational drug is available.
  • Expanded access must be permitted by, and run in accordance with, applicable laws.
  • The treating physician making the request is licensed and qualified to administer the investigational medicine and agrees to comply with Protara’s requirements and local regulations governing expanded access and to adhere to applicable laws and regulations.

All questions and requests regarding Protara’s expanded access program should be submitted to the company by email to Patients@Protaratx.com. We regularly monitor this mailbox and aim to acknowledge and respond to each submitted request.

Protara will continually assess our expanded access policy and provide updates to this website as needed.

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