Protara Therapeutics presents the results of a retrospective
NEW YORK, May 12, 2022 (GLOBE NEWSWIRE) — Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced the results of a retrospective analysis of OK-432, the parent compound of TARA-002, Protara’s experimental therapy in development for the treatment of lymphatic malformations (LM). LMs are serious and rare congenital malformations of the lymphatic vessels. The results of the analysis, which were presented in a poster presentation at the 2022 International Society for the Study of Vascular Anomalies (ISSVA) World Congress, showed that OK-432 was clinically effective and generally well tolerated in the treatment of macrocystic diseases and mixed cystic LM.
“We are delighted to share these compelling results, which are consistent with the solid body of approximately 30 years of patient experience with OK-432,” said Richard Smith, MD, Department of Otorhinolaryngology, Carver College of Medicine, University of Iowa, and study author. “There are currently no FDA-approved treatments for ML, which are typically diagnosed in early childhood and can lead to serious complications. These data support the potential of TARA-002 to ultimately serve as an effective intervention. in this very underserved area.
The retrospective analysis included 246 patients from a randomized phase 2 study and 275 patients from an open-label study. The majority of participants in both studies were between six months and 18 years old. In the first study, patients were randomized 2:1 to receive treatment immediately (immediate treatment group [ITG]) or delayed by six months (delayed treatment group [DTG]). In the open-label study, patients received four doses of OK-432 approximately six weeks apart. The primary efficacy endpoint was clinical success (defined as [90%-100%] or substantial [60%-89%] reduction in LM volume measured radiographically) in ITG compared to spontaneous resolution of LM in DTG. Efficacy was assessed two weeks after treatment in the randomized study and one to six months after treatment in the open-label study.
The main conclusions are summarized below:
- Approximately 69% of patients in the randomized ITG study achieved clinical success after six months, while only 7.5% of DTG patients showed spontaneous resolution of LMs during the same period (p
- 73.1% of patients in the open-label study achieved clinical success.
- In the randomized and open-label studies, 10 of 219 (4.6%) and 5 of 275 (1.8%) subjects, respectively, experienced serious treatment-emergent adverse events that were assessed by the investigator as related to the study drug. The most serious adverse events (SAEs) were airway obstruction and facial paralysis due to post-injection swelling which required tracheotomy and hospitalization. Both of these events have been reported as resolved. SAE related to OK-432 led to discontinuation (proptosis of the eye).
- Local/systemic reactions peaked within the first few days and resolved within two weeks.
- Patients were followed up to three years after treatment without significant safety concerns.
“We are encouraged by the growing amount of positive data supporting OK-432 to help patients with LD,” said Jesse Shefferman, CEO of Protara Therapeutics. “We look forward to using the robust data set for OK-432 to support further development of TARA-002 as we work toward our goal of delivering the first approved drug for SCI in the United States”
TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and SCI for which it has received Rare Pediatric Disease Designation from the United States Food and Drug Administration. TARA-002 was developed from the same genetically distinct group A master cell bank Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil® in Japan and Taiwan by Chugai Pharmaceutical Co., Ltd. Protara successfully demonstrated manufacturing comparability between TARA-002 and OK-432.
When TARA-002 is administered, it is believed that the innate and adaptive immune cells of the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate abnormal cells and various cytokines, including the interleukins IL-2, IL-6, IL-8, IL-10, IL-12, interferon (IFN)-gamma, factor necrosis (TNF)-alpha, granulocyte-colony stimulating factor and granulocyte-macrophage colony-stimulating factor, are secreted by immune cells to induce a strong local inflammatory response and destroy abnormal cells.
About Lymphatic Malformations
Lymphatic malformations (LM) are rare birth defects of the lymphatic vessels resulting in the inability of these structures to connect or drain into the venous system. Most MLs are present in the head and neck region and are diagnosed in infancy during the period of active lymphatic growth, with over 50% detected at birth and 90% diagnosed by age three. . The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheotomy dependency; intralesional bleeding; impact on critical structures, including nerves, vessels, lymphatics; recurrent infection and aesthetic and other functional impairments.
About Protara Therapeutics, Inc.
Protara is committed to identifying and advancing transformative therapies for people with cancer and rare diseases with limited treatment options. Protara’s portfolio includes its lead program, TARA-002, an investigational cell therapy in development for the treatment of non-muscle invasive bladder cancer and lymphatic malformations, and IV Choline Chloride, an investigational replacement therapy of phospholipid substrate for the treatment of intestinal failure – associated liver disease. For more information, visit www.protaratx.com.
Statements in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as ” predicts”, “believes”, “potential”, “proposed”, “continues”, “designs”, “estimates”, “anticipates”, “expects”, “plans”, “intends”, “may”, “could”, “could”, “shall”, “should” or other words or expressions referring to future events, conditions or circumstances that convey uncertainty of future events or results to identify such forward-looking statements These forward-looking statements include, but are not limited to, statements regarding Protara’s current intentions, beliefs, projections, prospects, analyzes or expectations regarding, among other things: Protara’s business strategy, including s development plans for its product candidates and plans regarding the timing or results of existing or future clinical trials; statements regarding expectations regarding interactions with the FDA, including potential alignment with the FDA on a development pathway for TARA-002 in pediatric LM patients; the financial condition of Protara; statements regarding the safety or anticipated efficacy of Protara’s product candidates; and Protara’s outlook for the rest of the year. Because these statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by these forward-looking statements. Factors that contribute to the uncertain nature of forward-looking statements include: the risks that Protara’s financial forecasts will not meet expectations, and the risks and uncertainties associated with: Protara’s development programs, including the launch and completion of nonclinical studies and clinical trials and timing of required filings with the FDA and other regulatory agencies; the impact of the COVID-19 pandemic on Protara’s business and the global economy as well as the impact on contract research organizations, study sites or other clinical partners of Protara; general market conditions; changes in the competitive landscape; changes in Protara’s strategic and business plans; Protara’s ability to obtain sufficient financing to fund its strategic plans and marketing efforts; having to use the money in a manner or at a time other than intended; the impact of market volatility on cash reserves; the loss of key members of management; the impact of general US and foreign economic, industry, market, regulatory or political conditions; and the risks and uncertainties associated with Protara’s business and financial condition generally, including the risks and uncertainties described in greater detail under “Risk Factors” and elsewhere in Protara’s filings and reports with of the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date they are made and are based on management’s assumptions and estimates as of that date. Protara undertakes no obligation to update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise, except as required by law.