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By David Willey, Benzinga
Advancements in healthcare and medicine have helped contribute to the reduction in child mortality rates, plummeting from 185 per 1,000 births a century ago to just 7 per 1,000 births in 2020. However, some serious pediatric conditions continue to pose a threat. One is hypoplastic left heart syndrome (HLHS), a rare but dangerous heart defect where the child’s left ventricle is severely underdeveloped.
Hypoplastic left heart syndrome (HLHS) impacts approximately 1 in 3,800 newborns (or about 1,025 live births in the United States annually). Children born with this condition have a significant risk of death stemming from the failure of their right heart ventricle. The case of T.J. Olsen, son of former NFL star Greg Olsen, who was born with HLHS, illustrates the gravity of this condition. Despite undergoing four intricate heart surgeries, T.J.'s condition continued to deteriorate, resulting in congestive heart failure. Ultimately, TJ had to have a life-saving heart transplant at 8 years of age – highlighting the severity of HLHS.
Longeveron (NASDAQ: LGVN), a clinical-stage biopharmaceutical company focusing on age-related and life-threatening conditions, is developing Lomecel-B™, its leading drug candidate, to try to improve outcomes for children like T.J. who suffer from HLHS. Lomecel-B™ is a cellular medicine known as a medicinal signaling cell (MSC) that can be derived from the bone marrow of healthy adults. Administering Lomecel-B™ directly into the heart tissue of the right ventricle during the 2nd heart surgery that HLHS patients undergo may have the potential to boost right ventricular function. If effective, this treatment could result in enhanced overall outcomes and potentially lessen the need for future heart transplants in the HLHS population.
100% Survival Rate For Phase 1 TrialLongeveron is advancing its phase 2 trial (ELPIS II), studying 38 HLHS patients. The primary outcome measure is safety, and efficacy endpoints include the change in right ventricular ejection fraction (RVEF) at 12 months post-treatment. The trial will also assess several secondary endpoints, including changes in right ventricular function and morphology, changes in clinical outcomes and quality of life as well as blood biomarkers and safety.
This follows the long-term success of its phase 1 trial (ELPIS I). Longeveron recently released new data showing that among the 10 patients in the ELPIS I study, none experienced serious adverse events (SAEs), none of them needed heart transplants, and the group experienced a 100% 5-year survival rate, as compared with approximately 80 survival rate over the same period of time in historical trials.
Sunjay Kaushal, M.D. Ph.D., Principal Investigator of the ELPIS I trial, commented on the results, “There is a major unmet need among children with HLHS, and today’s data highlighting the 100% survival rate of ELPIS I patients up to 5 years post-treatment underscore the opportunity for Lomecel-B™ as a much needed therapeutic innovation for this patient group.”
Position In MarketThough there are only approximately 1,000 babies born with this condition every year, there are opportunities available for drug development addressing smaller/rare conditions that aren’t always available for drugs targeting conditions affecting larger populations:
The development cost for these studies is significantly reduced as the population size – which can drive the cost of a drug trial – is much lower for rare conditions like HLHS than for more common chronic diseases.
Longeveron may be able to leverage the benefits of the Orphan Drug Designation, Fast-Track designation, and Rare Pediatric Designation Longeveron has received from the Food & Drug Administration (FDA) for the development and request for approval of Lomecel-B™ in the treatment of HLHS, including potentially being able to take advantage of a priority review voucher often associated with these designations. Notably, Serepta recently sold its priority review voucher for $102 million.
Longeveron has the advantage of in-house manufacturing capabilities, including its state-of-the-art cGMP facility based in Miami. Because the market size is relatively small, Longeveron anticipates it can produce and distribute its own off-the-shelf Lomebel-B™ product without needing to scale up production beyond its current capacity for the potential HLHS market size.
Other companies involved in treating pediatric diseases include Moderna (NASDAQ: MRNA) and Amgen (NASDAQ: AMGN).
Want to read more about how Longeveron is taking on HLHS? Check out its website.
Other References:
1 Newburger et al. Circulation (2018) 137:2246-2253.
2 Newburger et al. Circulation (2014) 129:2013-2020.
3 Ohye et al. N Engl J Med (2010) 362:1980-1992.
We are a clinical-stage biotechnology company developing cellular therapies for aging-related and life-threatening conditions.Our lead investigational product is Lomecel-B™, which is derived from culture-expanded medicinal signaling cells (MSCs) that are sourced from bone marrow of young healthy adult donors. We believe that by using the same cells that promote tissue repair, organ maintenance, and immune system function, we can develop safe and effective therapies for some of the most difficult diseases and conditions associated with aging.
Forward-Looking Statements
Certain statements in this press release that are not historical facts are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which reflect management's current expectations, assumptions, and estimates of future operations, performance and economic conditions, and involve risks and uncertainties that could cause actual results to differ materially from those anticipated by the statements made herein. Forward-looking statements are generally identifiable by the use of forward-looking terminology such as "believe," "expects," "may," "looks to," "will," "should," "plan," "intend," "on condition," "target," "see," "potential," "estimates," "preliminary," or "anticipates" or the negative thereof or comparable terminology, or by discussion of strategy or goals or other future events, circumstances, or effects. Factors that could cause actual results to differ materially from those expressed or implied in any forward-looking statements in this release include, but are not limited to, statements regarding the offer and sale of securities, the terms of the offering, about the ability of Longeveron’s clinical trials to demonstrate safety and efficacy of the Company’s product candidates, and other positive results; the timing and focus of the Company’s ongoing and future preclinical studies and clinical trials and the reporting of data from those studies and trials; the size of the market opportunity for the Company’s product candidates, including its estimates of the number of patients who suffer from the diseases being targeted; the success of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of the Company’s product candidates; the Company’s ability to obtain and maintain regulatory approval of its product candidates in the U.S., Japan and other jurisdictions; the Company’s plans relating to the further development of its product candidates, including additional disease states or indications it may pursue; the Company’s plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available and its ability to avoid infringing the intellectual property rights of others; the need to hire additional personnel and the Company’s ability to attract and retain such personnel; the Company’s estimates regarding expenses, future revenue, capital requirements and needs for additional financing; the Company’s need to raise additional capital, and the difficulties it may face in obtaining access to capital, and the dilutive impact it may have on its investors; the Company’s financial performance, and the period over which it estimates its existing cash and cash equivalents will be sufficient to fund its future operating expenses and capital expenditure requirements. Additionally, Longeveron makes no assurance that any public offering of its securities as described herein will occur at all, or that any such transaction will occur on the timelines, in the manner or on the terms anticipated due to numerous factors. Further information relating to factors that may impact the Company's results and forward-looking statements are disclosed in the Company's filings with the Securities and Exchange Commission, including Longeveron’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on March 14, 2023. The forward-looking statements contained in this press release are made as of the date of this press release, and the Company disclaims any intention or obligation, other than imposed by law, to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.
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