Children's Research Institute, Inc.

TARA-002 demonstrated compelling efficacy and was generally well-tolerated Enrollment in additional cohorts underway with initial results expected in 1H’25 NEW YORK, Sept. 09, 2024 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced completion of the first cohort of the Phase 2 STARBORN-1 trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of pediatric patients with lymphatic malformations (LMs). Enrollment is now underway in additional cohorts. “The initial data is compelling and reflective of the significant benefit observed in previous studies with OK-432, the predecessor of TARA-002,” said Nancy Bauman, MD, Children's National Medical Center: Children's Research Institute, Washington DC, and investigator for the STARBORN-1 trial. “There is a pressing need for an effective therapeutic option for LMs, a rare condition mainly affecting children for which there are currently no U.S. FDA approved agents. I remain excited about the potential for TARA-002 to play a meaningful role in the treatment of these patients and look forward to future learnings from this important study.” Of three patients treated in the first cohort, which enrolled individuals six years to less than 18 years of age, two patients treated with TARA-002 achieved a complete response after receiving one dose of TARA-002; the responses were seen in a patient with a macrocystic lymphatic malformation and a patient with a ranula. The safety and tolerability seen in this cohort was consistent with that of the historical experience with OK-432 and included treatment emergent adverse events (TEAEs) of pain, swelling, fatigue, and body temperature increases. All TEAEs were mild to moderate and resolved. “We are pleased with the progress of our Phase 2 STARBORN-1 trial and the encouraging results we have seen thus far,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We have received positive feedback from our investigators and already have a number of patients on waiting lists for our subsequent cohorts, and expect to share initial results from our next cohort in the first half of 2025.” STARBORN-1 is a Phase 2 single-arm, open-label, prospective clinical trial evaluating the safety and efficacy of intracystic injection of TARA-002 for the treatment of macrocystic and mixed cystic LMs (≥ 50% macrocystic disease) in participants six months to less than 18 years of age. The trial will enroll approximately 30 patients including age de-escalation safety lead-in cohorts of children ages six years to less than18 years, two years to less than six years, and six months to less than two years, who will receive up to four injections of TARA-002 spaced approximately six weeks apart. The primary endpoint of the trial is the proportion of participants with macrocystic and mixed cystic LMs who demonstrate clinical success, defined as having either a complete response (90% to 100% reduction from baseline in total LM volume) or substantial response (60% to less than 90% reduction in total LM volume) as measured by axial imaging. About TARA-002 in LMs TARA-002 is an investigational cell therapy based on the broad immunopotentiator, OK-432, which was originally granted marketing approval by the Japanese Ministry of Health and Welfare as an immunopotentiating cancer therapeutic agent. This cell therapy is currently approved in Japan and Taiwan for LMs and has been used to successfully treat thousands of pediatric patients with this rare condition. In addition, OK-432 was studied in the largest ever conducted Phase 2 trials in LMs, in which the therapy was administered via a now-closed compassionate use program led by the University of Iowa. TARA-002 has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of LMs. About Lymphatic Malformations 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 are 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 three 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. About Protara Therapeutics, Inc. Protara is committed to advancing transformative therapies for people with cancer and rare diseases. Protara’s portfolio includes its lead program, TARA-002, an investigational cell-based therapy being developed for the treatment of non-muscle invasive bladder cancer and lymphatic malformations, and IV Choline Chloride, an investigational phospholipid substrate replacement for patients dependent on parenteral support. For more information, visit www.protaratx.com. Forward-Looking Statements Statements contained 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,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words or expressions referencing future events, conditions or circumstances that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include but are not limited to, statements regarding Protara’s intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: Protara’s business strategy, including its development plans for its product candidates and plans regarding the timing or outcome of existing or future clinical trials; statements related to expectations regarding interactions with the FDA; Protara’s financial position; statements regarding the anticipated safety or efficacy of Protara’s product candidates; and Protara’s outlook for the remainder of the year. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that contribute to the uncertain nature of the forward-looking statements include: risks that Protara’s financial guidance may not be as expected, as well as risks and uncertainties associated with: Protara’s development programs, including the initiation and completion of non-clinical studies and clinical trials and the timing of required filings with the FDA and other regulatory agencies; general market conditions; changes in the competitive landscape; changes in Protara’s strategic and development and commercial plans; Protara’s ability to obtain sufficient financing to fund its strategic plans and commercialization efforts; having to use cash in ways or on timing other than expected; the impact of market volatility on cash reserves; the loss of key members of management; the impact of general U.S. and foreign, economic, industry, market, regulatory, political or public health conditions; and the risks and uncertainties associated with Protara’s business and financial condition in general, including the risks and uncertainties described more fully under the caption “Risk Factors” and elsewhere in Protara's filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such 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. Company Contact:Justine O'MalleyProtara TherapeuticsJustine.OMalley@protaratx.com646-817-2836

DUBLIN, Dec. 7, 2023 /PRNewswire/ -- The "Retinopathy of Prematurity - Epidemiology Forecast- 2032" report has been added to ResearchAndMarkets.com's offering. This "Retinopathy of Prematurity (Retinopathy of Prematurity) - Epidemiology - 2032" report delivers an in-depth understanding of Retinopathy of Prematurity historical and forecasted epidemiology in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. The report provides historical as well as forecasted epidemiology segmented by total incident cases of preterm infants by birth weight (=1,500 g), total incident cases of Retinopathy of Prematurity, and total treated cases (patients who went for initial treatment, patients who went for retreatment) of Retinopathy of Prematurity in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2019 to 2032. KOL Views To keep up with current epidemiology trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on the retinopathy of prematurity evolving preventive treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, drug uptake, along with challenges related to accessibility, including ophthalmologists, HCPs, Physicians, and others. The analysts connected with 10+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Centers such as National Eye Institute, National Institutes of Health, Bethesda, USA, Stanley Manne Children's Research Institute, United Kingdom, MD, Ophthalmology - Oakland, USA, North Shore University Health System, MD, Lurie Children's Hospital of Chicago, etc., were contacted. Their opinion helps understand and validate retinopathy of prematurity epidemiology trends. Key Highlights In the United States, there were around 18,000 incident cases of Retinopathy of Prematurity in 2022. These cases are projected to increase during the study period of 2019-2032. The risk factor for developing Retinopathy of Prematurity (ROP) is the baby's age and weight at birth. Especially children with gestational age at birth below 31 weeks or a birth weight of less than 1,500 g are in danger of developing Retinopathy of Prematurity. Research indicates that the incidence of Retinopathy of Prematurity has been increasing over the years and tends to rise consistently in the US. In France, approximately 30% of preterm infants by birth weight (=1,500g) develop Retinopathy of Prematurity. Advancements in life-preserving technologies have led to increased survival of premature and low birth weight (LBW) babies. As estimated, gestational age (EGA) and LBW are well-known to be inversely related to Retinopathy of Prematurity risk, these developments in neonatal care have increased the population of babies at risk for Retinopathy of Prematurity. The studies examining the incidence of Retinopathy of Prematurity have focused on various groups of premature infants, leading to variations in the calculated ROP incidence rates. This is due to differences in the worldwide screening guidelines, ranging from infants born before 30 to 37 weeks of gestational age (GA) and weighing between 1,000 g and 2,500 g. These divergent criteria make it challenging to compare ROP incidence rates across different studies directly. Approximately 30% of patients require treatment for Retinopathy of Prematurity in Japan. Epidemiology Insights What are the disease risk, burdens, and unmet needs of Retinopathy of Prematurity? What will be the growth opportunities across the 7MM concerning the patient population with Retinopathy of Prematurity? What is the historical and forecasted Retinopathy of Prematurity patient pool in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan? Why are incident cases of preterm infants by birth weight (=1,500 g) declining in some European countries? Scope of the Report The report covers a segment of key events, an executive summary, and a descriptive overview of retinopathy of prematurity, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies. Comprehensive insight into the epidemiology segments and forecasts, disease progression, and treatment guidelines has been provided. The report provides an edge while developing business strategies, understanding trends, expert insights/KOL views, and patient journeys in the 7MM. A detailed review of current challenges in establishing the diagnosis. Retinopathy of Prematurity Report Insights Patient Population Country-wise Epidemiology Distribution Incident cases of retinopathy of prematurity Retinopathy of Prematurity Report Key Strengths Ten Years Forecast The 7MM Coverage Retinopathy of Prematurity Epidemiology Segmentation Retinopathy of Prematurity Report Assessment Current Diagnostic Practices For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo - SOURCE Research and Markets

NEW YORK, Oct. 23, 2023 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced that dosing is now underway in the Phase 2 STARBORN-1 trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of pediatric patients with lymphatic malformations (LMs). “There is a pressing need for an effective FDA-approved therapy to treat LMs, a rare condition mainly affecting children. There are currently no such agents that are FDA approved specifically for this indication,” said Nancy Bauman, MD, Children's National Medical Center: Children's Research Institute, Washington DC, and investigator for the STARBORN-1 trial. “Having witnessed first-hand in prior studies the significant benefit observed with OK-432, the predecessor of TARA-002, I firmly believe that TARA-002 has the potential to play a meaningful role in this vulnerable patient population.” STARBORN-1 is a Phase 2 single-arm, open-label, prospective clinical trial to evaluate the safety and efficacy of intracystic injection of TARA-002 for the treatment of macrocystic and mixed cystic LMs (≥ 50% macrocystic disease) in participants six months to less than 18 years of age. Including an age de-escalation safety lead-in, the trial will enroll approximately 30 patients who will receive up to four injections of TARA-002 spaced approximately six weeks apart. “Initiation of the STARBORN-1 trial marks an important step forward for our LMs program and, more importantly, the young patients impacted by this rare condition,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We are committed to bringing TARA-002 to patients suffering from this devastating disease and look forward to advancing this study.” The primary endpoint of the trial is the proportion of participants with macrocystic and mixed cystic LMs who demonstrate clinical success, defined as having either a complete response (90% to 100% reduction from baseline in total LM volume) or substantial response (60% to less than 90% reduction in total LM volume) as measured by axial imaging. TARA-002 is an investigational cell therapy based on the broad immunopotentiator, OK-432, which was originally granted marketing approval by the Japanese Ministry of Health and Welfare as an immunopotentiating cancer therapeutic agent. This cell therapy is currently approved in Japan and Taiwan for LMs and has been used to successfully treat thousands of pediatric patients with this rare condition. In addition, OK-432 was studied in the largest ever conducted Phase 2 trials in LMs, in which the therapy was administered via a now-closed compassionate use program led by the University of Iowa. TARA-002 has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of LMs. About Lymphatic Malformations 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 are 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 three 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. About TARA-002 in LMs TARA-002 is an investigational cell therapy in development for the treatment of non-muscle invasive bladder cancer and of LMs for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. 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 approved in Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432. When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-2, IL-6, IL-8, IL-10, IL-12, interferon (IFN)-gamma, and tumor necrosis factor (TNF)-alpha are secreted by immune cells to induce a strong inflammatory reaction and destroy the abnormal cells. About Protara Therapeutics, Inc. Protara is committed to advancing transformative therapies for people with cancer and rare diseases. Protara’s portfolio includes its lead program, TARA-002, an investigational cell-based therapy being developed for the treatment of non-muscle invasive bladder cancer and lymphatic malformations, and IV Choline Chloride, an investigational phospholipid substrate replacement for patients dependent on parenteral nutrition. For more information, visit . Forward-Looking Statements Statements contained 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,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words or expressions referencing future events, conditions or circumstances that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include but are not limited to, statements regarding Protara’s intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: Protara’s business strategy, including its development plans for its product candidates and plans regarding the timing or outcome of existing or future clinical trials; statements related to expectations regarding interactions with the FDA; Protara’s financial position; statements regarding the anticipated safety or efficacy of Protara’s product candidates; and Protara’s outlook for the remainder of the year. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that contribute to the uncertain nature of the forward-looking statements include: risks that Protara’s financial guidance may not be as expected, as well as risks and uncertainties associated with: Protara’s development programs, including the initiation and completion of non-clinical studies and clinical trials and the timing of required filings with the FDA and other regulatory agencies; general market conditions; changes in the competitive landscape; changes in Protara’s strategic and commercial plans; Protara’s ability to obtain sufficient financing to fund its strategic plans and commercialization efforts; having to use cash in ways or on timing other than expected; the impact of market volatility on cash reserves; the loss of key members of management; the impact of general U.S. and foreign, economic, industry, market, regulatory, political or public health conditions; and the risks and uncertainties associated with Protara’s business and financial condition in general, including the risks and uncertainties described more fully under the caption “Risk Factors” and elsewhere in Protara's filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such 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. Company Contact: Justine O'Malley Protara Therapeutics Justine.OMalley@protaratx.com 646-817-2836