NEW YORK--(BUSINESS WIRE)--ArTara Therapeutics, a clinical-stage biopharmaceutical company developing treatments for rare diseases with significant unmet therapeutic needs, has initiated development of TARA-002, a toll-like receptor 4 agonist (TLR4a), for the potential treatment of lymphangiomas (Lymphatic Malformations [LMs]). This is ArTara’s second pipeline program and the first to focus in the immunology and oncology therapeutic area.
TARA-002 is an investigational cellular therapy based on OK-432, an immunostimulant, currently approved in Japan and Taiwan, that has been the standard of care for LMs for over 20 years.
Lymphatic malformations are localized anomalies of the lymphatic vasculature that develop in utero or in infants, most commonly in the head and neck. LMs are estimated to occur in 1 out of 4,000 live births.1 The only approved treatment for LMs in the US and Europe is surgical excision, which carries high rates of recurrence (55%) and surgical complications (33%).2
A randomized, Phase-2 study of OK-432, led by Dr. Richard Smith of the University of Iowa, demonstrated a clinically successful outcome in 94% of patients with macrocystic LMs and 63% of patients with mixed macrocystic-microcystic LMs.3
“We are enthusiastic about the potential of TARA-002 to change the lives of pediatric patients around the world suffering from LMs,” said Dr. Julio Casoy, ArTara’s Chief Medical Officer. “We also hope to capitalize on this mechanism’s long history in oncology outside the US as there are hundreds of thousands of cancer patients who could benefit from TARA-002’s intriguing TLR4 activation properties.”
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.
In partnership with Novex Innovations, a leading biologics CDMO, ArTara will build a US-based manufacturing facility for TARA-002 and initiate regulatory activities in the US to support marketing authorization for TARA-002. “We are grateful to Dr. Richard Smith and the University of Iowa for their collaboration and generosity of time as we build on their excellent work with this mechanism in LMs,” said Jesse Shefferman, ArTara’s Chief Executive Officer. “Additionally, we look forward to working with the Novex team as, together, we build a US-based facility to bring TARA-002 into the future.”
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.
About ArTara Therapeutics
ArTara is a rare diseases therapeutics company focused on optimizing product candidates for patients suffering from rare diseases where there is a significant unmet need. ArTara’s current development programs focus on rare diseases of structural and connective tissues as well as rare hepatology/gastrointestinal and metabolic disorders with further programs in immunology, beginning with investigational candidate TARA-002 for the potential treatment of Lymphatic Malformations.
For more information, visit http://www.artaratx.com/.
1. Kennedy TL, et al. Cystic hygroma/lymphangioma: a rational approach to management. Laryngoscope. 2001;111:1929-1937.
2. Ha J, et al. A review of the management of lymphangiomas. Curr Ped Rev. 2014;10:238-248.
3. Smith R, et al. Efficacy and safety of OK-432 immunotherapy of lymphatic malformations. Laryngoscope. 2009;119:107-115.