Univentricular Heart

NEWS RELEASE: Children’s National Hospital, Additional Ventures offer grants for pediatric cardiology devices Grants totaling $300,000 to be distributed among innovators with medical technology solutions WASHINGTON – The Alliance for Pediatric Device Innovation (APDI), a nonprofit consortium led by Children’s National Hospital and funded by the Food and Drug Administration (FDA), announces today it is partnering with Additional Ventures, a nonprofit foundation focused on accelerating research progress and improving clinical care for individuals born with single ventricle heart defects, to solicit proposals and offer grant funding for the development of pediatric devices specifically evaluated and labeled for children with heart conditions. Children with heart conditions face unique challenges that demand specialized medical devices tailored to their specific physiological needs. There is a significant unmet need for pediatric devices designed to monitor and treat young patients effectively in cardiology, interventional cardiology, cardiac surgery and electrophysiology. Current technologies for pediatric heart patients often fall short because they are typically adapted from adult devices and may not provide the accuracy, safety or comfort required for children. Areas of special interest include remote patient monitoring systems that can track vital signs and heart function in real-time, wearable devices designed for continuous monitoring, and minimally invasive technologies for cardiac procedures. Through this request for proposals, APDI seeks innovative solutions that address these gaps, offering the potential to transform care for children with heart conditions by developing advanced, child-centric devices. “Our commitment to advancing pediatric cardiology is stronger than ever. With the launch of this initiative, we are paving the way for innovative pediatric devices that will transform how we monitor and care for children with heart conditions,” said Kolaleh Eskandanian, Ph.D., M.B.A., vice president and chief innovation officer at Children’s National and APDI program director and principal investigator. “This proposal allows us to support projects that show promise in advancing novel devices specifically designed for children and support innovators who need funds for the complex process of bringing new technologies to market.” When approaching device design for this proposal, Dr. Eskandanian adds that innovators should start with a pediatric focus because, if the device works for children, it can be adapted for adults. She notes that developing pediatric device solutions requires an individualized approach with a design that accommodates usability for all age groups. The total award fund of $300,000 will be distributed among innovators with the most promising devices as selected by the judging panel. As part of the collaboration, Additional Ventures and APDI will provide winning companies with support services and technical expertise, including but not limited to regulatory, reimbursement, clinical trials study design and data science services. Interested innovators can learn more and apply for the APDI medical device grant online at innovate4kids.org. The deadline to apply is midnight on September 23, 2024. APDI is one of five nonprofit consortia in the FDA’s Pediatric Device Consortia program that receives funding to provide a platform of services, expertise and grants to support pediatric innovators in bringing medical devices to the market that address the needs of children. Along with Children’s National, APDI members include Johns Hopkins University, CIMIT at Mass General Brigham, Tufts Medical Center, Medstar Health Research Institute, OrthoPediatrics Corp. and MedTech Color. Advancements in pediatric medical devices continue to lag significantly behind those of adults, which is why APDI is focused on helping more pediatric medical device innovations achieve commercialization. For more information on APDI, visit innovate4kids.org. About Children’s National Hospital Children’s National Hospital, based in Washington, D.C., was established in 1870 to help every child grow up stronger. Today, it is the No. 5 children’s hospital in the nation and ranked in all specialties evaluated by U.S. News & World Report. Children’s National is transforming pediatric medicine for all children. The Children’s National Research & Innovation Campus opened in 2021, a first-of-its-kind pediatric hub dedicated to developing new and better ways to care for kids. Children’s National has been designated three times in a row as a Magnet® hospital, demonstrating the highest standards of nursing and patient care delivery. This pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty care locations in the D.C. metropolitan area, including Maryland and Virginia. Children’s National is home to the Children’s National Research Institute and Sheikh Zayed Institute for Pediatric Surgical Innovation. It is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. As a nonprofit, Children’s National relies on generous donors to help ensure that every child receives the care they need. For more information, follow us on Facebook, Instagram and LinkedIn. About Additional Ventures Additional Ventures is a purpose-driven organization leveraging evidence-based research and deep subject matter expertise to make an outsized impact. Our relentless optimism powers bold, high-risk innovations to solve some of humanity’s most complex challenges. Our biomedical research work focuses on a rare form of congenital heart defects called single ventricle heart disease. While this field is in its infancy, with limited knowledge of cause, risk, outcomes, or treatments, we are confident that through coordinated strategic and interdisciplinary work, dynamic teaming, and flexible funding, we can illuminate a functional cure for patients and their families. Learn more about our work at additionalventures.org, LinkedIn, or X. The opinions expressed in this news release are the author’s only and do not necessarily reflect those of Medical Design & Outsourcing or its employees.

FORT LEE, N.J., March 1, 2024 /PRNewswire/ -- Mezzion Pharmaceuticals is a rare disease company conducting the confirmatory pivotal phase 3 clinical trial in Fontan subjects: The Fontan Udenafil Exercise Longitudinal Assessment Trial- 2, or FUEL-2 trial. The company is pleased to announce Dr. Rahul Rathod, a pediatric cardiology specialist and leader with over 20 years of experience at Boston Children's Hospital, as the Global Principal Investigator of FUEL-2. Continue Reading FUEL-2 Dr. Rathod currently serves as the Associate Chair of Cardiology, Director of the Single Ventricle Program, and Director of the Fontan Clinic at Boston Children's Hospital as well as Associate Professor of Pediatrics at Harvard Medical School. He is an internationally recognized medical expert, researcher, and key opinion leader in the single ventricle heart disease space and Fontan circulation—with more than 80 peer-reviewed publications. Dr. Rathod is the Founder and Executive Director of the Fontan Outcomes Registry Using CMR Examinations (FORCE). The FORCE Registry is a tech-enabled research platform dedicated to improving the quality and longevity of life for single ventricle patients by aggregating global Fontan data, advancing cardiac imaging through big data and analytics, and utilizing machine learning to improve workflows and patient outcomes. FORCE is the largest Fontan dataset in the world. As the Global Principal Investigator for FUEL-2, Dr. Rathod will lead the clinical trial, overseeing clinical protocol, conduct, and operations at all study sites. He will also lead the study analysis and contribute to reporting the results to the Food and Drug Administration (FDA). "FUEL-2 represents a pivotal moment in Fontan research pioneering advancements for our patients. This landmark study delves into the potential of udenafil to empower individuals to reclaim vitality in their daily lives," said Dr. Rathod about the trial. "The Fontan population is one with significant medical complexities and unmet needs; this trial offers the promise of tangible solutions and a powerful tool that can redefine the treatment landscape for Fontan patients." "Dr. Rathod is a preeminent expert in Fontan physiology and the ideal clinical leader to serve as the Global Principal Investigator of FUEL-2. Mezzion is very proud and encouraged to have him as our partner as this pivotal clinical trial progresses," said Dr. John Hariadi, Chief Medical Officer and Senior Vice President of Global Regulatory Affairs at Mezzion. Dr. Hariadi recently joined Mezzion from Johnson & Johnson, where he was senior director for global regulatory affairs. He previously served in several senior medical leadership roles at the FDA and the United States Department of Homeland Security. He will serve as Mezzion's primary liaison to the FUEL-2 clinical research organization, study sites, and investigators. FUEL-2 is a Mezzion Pharmaceuticals-sponsored clinical trial investigating the clinical efficacy and safety of udenafil in teenagers 12-18 years of age who have undergone the Fontan procedure. It is poised to be the world's largest Fontan clinical trial and is set to enroll 438 patients globally. Currently, 22 sites in the U.S. are enrolling or will begin enrolling in the coming weeks. The latest site information for FUEL-2 can be found here. Udenafil is a selective phosphodiesterase type 5 (PDE5) inhibitor and was previously studied in some adolescents with Fontan physiology. PDE5 inhibitors relax the blood vessels and may increase the blood flow to the lungs. This may mean higher blood oxygen levels; if so, individuals may be less tired when exercising or during everyday activities. For more information about the study, please visit FUEL2study.com. About Mezzion Pharma Co., Ltd. Mezzion Pharma Co., Ltd. is headquartered in Korea. Mezzion and its wholly owned subsidiary, Mezzion Pharmaceuticals, Inc., has an administrative office in Fort Lee, New Jersey. Mezzion Pharma is an innovation-driven pharmaceutical company that is focused on discovering, developing, and commercializing novel therapeutics in the field of rare pediatric diseases. Mezzion Pharma is a publicly listed pharmaceutical company in Korea on the Korean stock exchange under (140410:KOSDAQ). 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. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: Mezzion Pharma's expectations regarding the potential benefits of udenafil; Mezzion Pharma's expectations regarding the anticipated timing of any future clinical trials; Mezzion Pharma's expectations on regulatory submissions for marketing approval of udenafil for the treatment of patients that have undergone the Fontan operation, to improve exercise capacity in the United States, including the timing of these submissions; and Mezzion Pharma's expectations regarding the potential commercial launch of udenafil, including the timing of a potential approval of udenafil. Risks and uncertainties that contribute to the uncertain nature of the forward-looking statements include: the expectation that Mezzion Pharma will need additional funds to finance its operations; Mezzion Pharma's or any of its collaborative partners' ability to initiate and/or complete clinical trials; the unpredictability of the regulatory process; the possibility that Mezzion Pharma's or any of its clinical trials will not be successful; Mezzion Pharma's dependence on the success of udenafil; Mezzion Pharma's reliance on third parties for the manufacture of Mezzion Pharma's udenafil and udenafil tablets; possible regulatory developments in the United States and foreign countries; and Mezzion Pharma's ability to attract and retain senior management personnel. These and other risks and uncertainties are described more fully in Mezzion Pharma's most recent filings with the Statements under the Private Securities Litigation Reform Act: with the exception of the historical information contained in this release, the matters described herein contain forward-looking statements that involve risk and uncertainties that may individually or mutually impact the matters herein described, including but not limited to FDA review and approval, product development and acceptance, manufacturing, competition, and/or other factors, which are outside the control of Mezzion Pharma. All forward-looking statements contained in this press release speak only as of the date on which they were made. Mezzion undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Media Contacts: PR Contact – Philicia Thompson, [email protected], 7047749643 Medical Contact – John Hariadi, [email protected], (443) 699-6746 Mezzion Pharma Co. Ltd. Contact – S.I. Noh, [email protected], +82 2 560 8000 SOURCE Mezzion Pharmaceuticals, Inc

New Study Exposes Bovine Fortification Protocol Is Hurdle to Advancing the Standard of Care, Questions Feasibility of Randomized Controlled Trials in Vulnerable Premature Infant Population DUARTE, Calif., Feb. 6, 2024 /PRNewswire/ -- Optimizing nutrition for premature infants remains an important focus in neonatal care. More than 20 peer-reviewed studies of 5,000+ preterm infants demonstrated that, compared to bovine milk-based fortifiers (BMBF), Prolacta Bioscience's human milk-based fortifiers (HMBF) improve growth and development1-7 and provide clinically significant reductions in comorbidities.1,2,8-20 The health benefits from the use of HMBF have also shown significant annual cost savings for hospitals.8 More than 100,000 critically ill and preterm infants have received Prolacta's human milk-based nutritional products.21 Prolacta's Exclusive Human Milk Diet (EHMD) Protocol, introduced in 2022, is based on data showing that hospitals with the best outcomes start fortification as early as day one and advance feeds quickly.1,7,22 Yet, in contrast to this protocol and more than a decade of extensive clinical evidence, two small randomized controlled trials (RCTs) — Jensen with 228 infants and O'Connor with 127 infants — found no statistically significant difference in mortality and morbidity when comparing HMBF to BMBF. While the RCTs were intended to provide a head-to-head comparison between fortifier products, the feeding protocols fundamentally differed. HMBF recommendations for best outcomes regarding the day fortification should begin and the speed at which feeds are advanced were not followed in either study. Both RCTs were designed more than six years ago with fortification protocols intended to mitigate the risks of BMBF, which is known to cause feeding intolerance and severe complications in preterm infants, including sepsis and necrotizing enterocolitis (NEC), a leading cause of preemie death.23 Ethical Boundaries Limit RCT Feasibility The ability to fortify early and advance feeds quickly with HMBF raises an important ethical issue for RCT design. The known risks of BMBF established the decades-old standard of care in the neonatal intensive care unit (NICU) to delay fortification until preterm infants can tolerate the risks of the foreign protein. Given these known risks, randomizing extremely premature infants to day-one fortification with BMBF would unjustly endanger this vulnerable patient population. In seeking to mitigate BMBF's risk, the protocols used in the RCTs demonstrated severely delayed EHMD implementation, thus reducing the observable EHMD benefits. Yet, despite feeding protocols that were incongruent with established EHMD guidelines, both the RCTs showed lower morbidity and mortality rates for infants in the HMBF arms, which are clinically significant findings: While significantly underpowered, the Jensen study showed the HMBF death rate was 47% lower than BMBF.24 After adjusting for gestational age, the HMBF group in the Jensen study had decreased odds of bronchopulmonary dysplasia (BPD) at 36 weeks (p = 0.049).24 The O'Connor study demonstrated a 50% reduction in late-onset sepsis and a statistically significant reduction in retinopathy of prematurity (ROP) in infants receiving HMBF.13 "As clinicians, we must review studies with a critical lens and truly consider the body of evidence on topics critical to the well-being of our patients," said Melinda Elliott, MD, FAAP, practicing neonatologist and chief medical officer for Prolacta. "I am convinced about the advantages of HMBF for critically ill and premature babies because I witnessed the impact with my own patients in Baltimore. What began as an initiative to decrease the incidence of NEC in our unit quickly became a unit-wide initiative to provide a clearly superior standard of care." Real-World Evidence Demonstrates Improved Outcomes and Reduced Costs Extensive real-world data affirm EHMD adoption enables critical health improvements for premature infants and major cost reductions for hospitals. Analysis of 2019-2022 data from 3,000+ patients at 60+ U.S. hospitals found EHMD implementation improved health outcomes and reduced costs, generating a 2.6X dollar-for-dollar return on investment.25 Similarly, a 2023 peer-reviewed report found EHMD implementation resulted in annual cost savings of $500,000 to $3.4 million per hospital from a reduction in comorbidities and shorter lengths of stay among very low birth weight infants.8 The body of real-world evidence in support of HMBF is substantial. It has been clinically proven in more than 20 peer-reviewed clinical studies that compared to BMBF, HMBF when used as part of an Exclusive Human Milk Diet demonstrated: Lower mortality and morbidity14,16,19 Reduced incidence of feeding intolerance15 Achieved adequate growth1,2,7 Reduced incidence of BPD1,12,14,15 Reduced incidence of ROP12,14,15 Reduced late-onset sepsis incidence and evaluations12-14 Reduced risk of NEC14,15,20 Improved long-term outcomes such as neurodevelopment4,5 Shortened stays in the NICU15 Reduced hospital costs8,15,25,26 Achieved better growth in term infants recovering from surgery for single ventricle physiology (SVP)27 and congenital gastrointestinal disorders (CGIDs)28 About Prolacta Bioscience Prolacta Bioscience® is a global life sciences company dedicated to Advancing the Science of Human Milk® to improve health outcomes for critically ill and premature infants. More than 100,00021 extremely premature infants worldwide have benefited from Prolacta's human milk-based products, which have been evaluated in more than 20 peer-reviewed clinical studies. Hospitals adopting Prolacta's Exclusive Human Milk Diet realize a 2.6X return on investment.25 Operating the world's first pharmaceutical-grade human milk processing facilities, Prolacta maintains the industry's strictest quality and safety standards, with over 20 validated tests for screening and testing human milk. Prolacta's manufacturing process uses vat pasteurization to ensure pathogen inactivation while protecting nutritional composition and bioactivity. Learn more at , on X, Instagram, Facebook, and LinkedIn. Media Contact: Loren Kosmont [email protected] 310-721-9444 References Huston R, Lee M, Rider E, et al. Early fortification of enteral feedings for infants <1250 grams birth weight receiving a human milk diet including human milk-based fortifier. J Neonatal Perinatal Med. 2020;13(2):215-221. doi:10.3233/NPM-190300 Huston RK, Markell AM, McCulley EA, Gardiner SK, Sweeney SL. Improving growth for infants ≤1250 grams receiving an exclusive human milk diet. Nutr Clin Pract. 2018;33(5):671-678. doi:10.1002/ncp.10054 Gates A, Thompson AB, Marin T, Waller JL, Patel J, Stansfield BK. Novel multinutrient human milk-based human milk fortifier promotes growth and tolerance in premature infants. J Parenter Enteral Nutr. 2022;46(4):817-827. doi:10.1002/jpen.2249 Bergner EM, Shypailo R, Visuthranukul C, et al. Growth, body composition, and neurodevelopmental outcomes at 2 years among preterm infants fed an exclusive human milk diet in the neonatal intensive care unit: a pilot study. Breastfeed Med. 2020. 15(5):304-311. doi:10.1089/bfm.2019.0210 Rahman A, Kase J, Murray Y, et al. Neurodevelopmental outcome of extremely low birth weight infants fed an exclusive human milk diet is not affected by growth velocity. Breastfeed Med. 2020;15(6):362-369. doi:10.1089/bfm.2019.0214 Sandhu A, Fast S, Bonnar K, Baier RJ, Narvey M. Human-based human milk fortifier as rescue therapy in very low birth weight infants demonstrating intolerance to bovine-based human milk fortifier. Breastfeed Med. 2017;12(9):570-573. doi:10.1089/bfm.2017.0017 Hair AB, Hawthorne KM, Chetta KE, Abrams SA. Human milk feeding supports adequate growth in infants ≤ 1250 grams birth weight. BMC Res Notes. 2013;6:459. Published 2013 Nov 13. doi:10.1186/1756-0500-6-459 Swanson JR, Becker A, Fox J, et al. Implementing an exclusive human milk diet for preterm infants: real-world experience in diverse NICUs. BMC Pediatr. 2023;23(1). doi.org/10.1186/s12887-023-04047-5 Osmanova M, Müller MJ, Habisch B, Hippe A, Seeliger S. Nutrition of infants with very low birth weight using human and bovine based milk fortifier: benefits and costs. Neonat Pediatr Med. 2021;S10:003. Hanford J, Mannebach K, Ohler A, Patten M, Pardalos J. Rates of comorbidities in very low birth weight infants fed an exclusive human milk diet versus a bovine supplemented diet. Breastfeed Med. 2021;16(10):814-820. doi:10.1089/bfm.2020.0345 Carome K, Rahman A, Parvez B. Exclusive human milk diet reduces incidence of severe intraventricular hemorrhage in extremely low birth weight infants. J Perinatol. 2021;41(3):535-543. doi:10.1038/s41372-020-00834-5 Delaney Manthe E, Perks PH, Swanson JR. Team-based implementation of an exclusive human milk diet. Adv Neonatal Care. 2019;19(6):460-467. doi:10.1097/ANC.0000000000000676 O'Connor DL, Kiss A, Tomlinson C, et al. Nutrient enrichment of human milk with human and bovine milk-based fortifiers for infants born weighing <1250 g: a randomized clinical trial [published correction appears in Am J Clin Nutr. 2019 Aug 1;110(2):529] [published correction appears in Am J Clin Nutr. 2020 May 1;111(5):1112]. Am J Clin Nutr. 2018;108(1):108-116. doi:10.1093/ajcn/nqy067 Hair AB, Peluso AM, Hawthorne KM, et al. Beyond necrotizing enterocolitis prevention: improving outcomes with an exclusive human milk-based diet [published correction appears in Breastfeed Med. 2017 Dec;12 (10):663]. Breastfeed Med. 2016;11(2):70-74. doi:10.1089/bfm.2015.0134 Assad M, Elliott MJ, Abraham JH. Decreased cost and improved feeding tolerance in VLBW infants fed an exclusive human milk diet. J Perinatol. 2016;36(3):216-220. doi:10.1038/jp.2015.168 Abrams SA, Schanler RJ, Lee ML, Rechtman DJ. Greater mortality and morbidity in extremely preterm infants fed a diet containing cow milk protein products. Breastfeed Med. 2014;9(6):281-285. doi:10.1089/bfm.2014.0024 Herrmann K, Carroll K. An exclusively human milk diet reduces necrotizing enterocolitis. Breastfeed Med. 2014;9(4):184-190. doi:10.1089/bfm.2013.0121 Huston RK, Markell AM, McCulley EA, et al. Decreasing necrotizing enterocolitis and gastrointestinal bleeding in the neonatal intensive care unit: the role of donor human milk and exclusive human milk diets in infants ≤1500 g birth weight. Infant Child Adolesc Nutr. 2014;6(2):86-93. doi:10.1177/1941406413519267 Cristofalo EA, Schanler RJ, Blanco CL, et al. Randomized trial of exclusive human milk versus preterm formula diets in extremely premature infants. J Pediatr. 2013;163(6):1592-1595.e1. doi:10.1016/j.jpeds.2013.07.011 Sullivan S, Schanler RJ, Kim JH, et al. An exclusively human milk-based diet is associated with a lower rate of necrotizing enterocolitis than a diet of human milk and bovine milk-based products. J Pediatr. 2010;156(4):562-7.e1. doi:10.1016/j.jpeds.2009.10.040 Data on file; estimated number of infants fed Prolacta's products from January 2007 to August 2023. Salas AA, Gunawan E, Nguyen K, et al. Early human milk fortification in infants born extremely preterm: a randomized trial. Pediatrics. 2023;152(3):e2023061603. doi:10.1542/peds.2023-061603 Ginglen JG, Butki N. Necrotizing Enterocolitis. StatPearls Publishing [Internet]. 2023. Available from: Jensen GB, Domellöf M, Ahlsson F, Elfvin A, Navér L, Abrahamsson T. Effect of human milk-based fortification in extremely preterm infants fed exclusively with breast milk: a randomised controlled trial. eClinicalMedicine. 2023. . 1016/j.eclinm.2023. 102375 Data on file; hospital-provided outcomes analysis from 2019 to 2022. Ganapathy V, Hay JW, Kim JH. Costs of necrotizing enterocolitis and cost-effectiveness of exclusively human milk-based products in feeding extremely premature infants. Breastfeed Med. 2012;7(1):29-37. doi:10.1089/bfm.2011.0002 Blanco CL, Hair A, Justice LB, Roddy D, Bonagurio K, Williams PK, Machado D, Marino BS, Chi A, Takao C, Gordon EE, Ashrafi A, Cacho N, Pruetz JD, Costello JM, Cooper DS, & Cardiac Neonate Nutrition Study Group. A randomized trial of an exclusive human milk diet in neonates with single ventricle physiology. J Pediatr. 2022;256: 105–112. doi.org/10.1016/j.jpeds.2022.11.043 Karpen, H. Impact of an exclusive human milk diet in neonates with congenital gastrointestinal disorders: initial clinical results. Oral presentation at ISHMSI, Vienna, 2022 available at: SOURCE Prolacta Bioscience