TARA-002 is our lead program and first to focus in the immunology and oncology therapeutics area. Tara-002 is a cellular therapy based on OK-432, a genetically distinct strain of Streptococcus Pyogenes which is rendered nonvirulent through a proprietary manufacturing process. This process retains the Streptococcus antigen properties while eliminating the infectious properties of the bacteria.
TARA-002’s predecessor treatment, OK-432, has been the standard of care in Japan for LMs for over 25 years and has demonstrated statistical and medical superiority compared to surgery in a multi-center study conducted in the US through the University of Iowa.
In addition to its use in LMs, OK-432’s attenuated Streptococcus Pyogenes modality has been approved in Japan as a potent biologic response modifier (BRM) in adjunctive use with first-line oncology therapies in gastric and lung cancer, and as a direct agent in Thyroid, head & neck tumors and for cancerous pleural effusion and ascites.
ArTara has acquired the Investigational New Drug Application (IND) and the Orphan Drug Designation for OK-432, as well as exclusive access to the data from the US-based, multicenter study in LMs.
Lymphatic malformations are rare, non-malignant, lesions consisting of dilated, lymphatic fluid-filled sacs caused by the abnormal development of the lymphatic endothelial system. The majority of LMs present from birth and are usually observed in-utero during the second trimester. They usually present as antero-lateral cervical masses and are estimated to occur in 1 out of 4,000 live births. The only approved treatment for LMs in the US and Europe is surgical excision, which carries high rates of recurrence and surgical complications (damage to nerves, scarring, and deformity).
IV CHOLINE CHLORIDE
IV Choline Chloride, our first program, has shown promising results in two Phase 2 studies in IFALD (Intestinal Failure Associated Liver Disease). IV Choline is a replacement therapy for patients on PN with choline deficiency who exhibit symptoms of liver impairment. We are developing IV Choline in partnership with Dr. Alan Buchman, a leader in the field of gastroenterology and a key voice for the potential of choline as a therapy for patients with IFALD.
ArTara’s IV Choline Chloride is protected by intellectual property through 2035 and has been awarded Orphan Drug Designation by the US FDA. Other intellectual property protections are in process, as well as Orphan Drug Designation applications in other key geographies.
IFALD (intestinal failure associated liver disease) is a potentially fatal liver disease that afflicts people who survive by receiving parenteral nutrition (PN). Through PN, patients with a missing or nonfunctional gastrointestinal tract receive the nutrition needed to support life. Up to 65% of all PN patients develop IFALD, in part due to deficiencies and imbalances in PN ingredients. There are as many as 25,000 PN patients in the US and ~10,000 in the EU. IFALD is characterized by accumulation of triglycerides in the liver leading to liver injury, cholestasis, and eventually a need for dual liver/intestine transplant, or death. There are no approved therapies for IFALD.