LGVN - Longeveron (NASDAQ: LGVN) Reports 100% 5-Year Survival In Trial For Rare Pediatric Disease HLHS (Compared To 80% Survival In Historical Trials) To Market
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By David Willey, Benzinga
Advancements inhealthcare and medicine have helped contribute to the reduction in childmortality rates , plummeting from 185 per 1,000 births acentury ago to just 7 per 1,000 births in 2020. However, some seriouspediatric conditions continue to pose a threat. One is hypoplasticleft heart syndrome (HLHS), a rare but dangerous heart defect wherethe child’s left ventricle is severely underdeveloped.
Hypoplastic left heartsyndrome (HLHS) impacts approximately 1in 3,800 newborns (or about 1,025 live births in the UnitedStates annually). Children born with this condition have a significantrisk of death stemming from the failure of their right heartventricle. The case of T.J.Olsen , son of former NFL star Greg Olsen, who was born withHLHS, illustrates the gravity of this condition. Despite undergoingfour intricate heart surgeries, T.J.'s condition continued todeteriorate, resulting in congestive heart failure. Ultimately, TJ hadto have a life-saving heart transplant at 8 years of age –highlighting the severity of HLHS.
Longeveron (NASDAQ: LGVN), a clinical-stagebiopharmaceutical company focusing on age-related and life-threateningconditions, is developingLomecel-B™, its leading drug candidate, to try to improveoutcomes for children like T.J. who suffer from HLHS. Lomecel-B™ isa cellular medicine known as a medicinal signaling cell (MSC) that canbe derived from the bone marrow of healthy adults. AdministeringLomecel-B™ directly into the heart tissue of the right ventricleduring the 2nd heart surgery that HLHS patients undergo may have thepotential to boost right ventricular function. If effective, thistreatment could result in enhanced overall outcomes and potentiallylessen the need for future heart transplants in the HLHSpopulation.
100% Survival Rate For Phase 1 Trial
Longeveron is advancing its phase 2trial (ELPIS II), studying 38 HLHS patients. The primaryoutcome measure is safety, and efficacy endpoints include the changein right ventricular ejection fraction (RVEF) at 12 monthspost-treatment. The trial will also assess several secondaryendpoints, including changes in right ventricular function andmorphology, changes in clinical outcomes and quality of life as wellas blood biomarkers and safety.
This follows the long-termsuccess of its phase 1 trial (ELPIS I). Longeveron recently releasednew data showing that among the 10 patients in the ELPIS Istudy, none experienced serious adverse events (SAEs), none of themneeded heart transplants, and the group experienced a 100% 5-yearsurvival rate, as compared with approximately 80 survival rate overthe same period of time in historicaltrials.
Sunjay Kaushal , M.D. Ph.D., Principal Investigator ofthe ELPIS I trial, commentedon the results , “There is a major unmet need among childrenwith HLHS, and today’s data highlighting the 100% survival rate ofELPIS I patients up to 5 years post-treatment underscore theopportunity for Lomecel-B™ as a much needed therapeutic innovationfor this patient group.”
Position In Market
Though there are only approximately 1,000 babies born with thiscondition every year, there are opportunities available for drugdevelopment addressing smaller/rare conditions that aren’t alwaysavailable for drugs targeting conditions affecting larger populations:
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The development cost for these studies is significantly reducedas the population size – which can drivethe cost of a drug trial – is much lower for rare conditionslike HLHS than for more common chronic diseases.
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Longeveron may be able to leverage the benefits of the OrphanDrug Designation, Fast-Track designation, and Rare PediatricDesignation Longeveron has received from the Food & DrugAdministration (FDA) for the development and request for approval ofLomecel-B™ in the treatment of HLHS, including potentially beingable to take advantage of a priority review voucher often associatedwith these designations. Notably, Serepta recently soldits priority review voucher for $102 million.
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Longeveron has the advantage of in-house manufacturingcapabilities , including its state-of-the-art cGMP facilitybased in Miami. Because the market size is relatively small,Longeveron anticipates it can produce and distribute its ownoff-the-shelf Lomebel-B™ product without needing to scale upproduction beyond its current capacity for the potential HLHS marketsize.
Other companies involved in treating pediatricdiseases include Moderna (NASDAQ: MRNA) and Amgen (NASDAQ: AMGN).
Want to readmore about how Longeveron is taking on HLHS? Check out itswebsite .
Other References:
1 Newburgeret al. Circulation (2018) 137:2246-2253.
2 Newburgeret al. Circulation (2014) 129:2013-2020.
3 Ohye et al. N Engl J Med (2010)362:1980-1992.
We are a clinical-stage biotechnologycompany developing cellular therapies for aging-related andlife-threatening conditions.Our lead investigational product isLomecel-B™, which is derived from culture-expanded medicinalsignaling cells (MSCs) that are sourced from bone marrow of younghealthy adult donors. We believe that by using the same cells thatpromote tissue repair, organ maintenance, and immune system function,we can develop safe and effective therapies for some of the mostdifficult diseases and conditions associated withaging.
Forward-Looking Statements
Certainstatements in this press release that are not historical facts areforward-looking statements made pursuant to the safe harbor provisionsof the Private Securities Litigation Reform Act of 1995, which reflectmanagement's current expectations, assumptions, and estimates offuture operations, performance and economic conditions, and involverisks and uncertainties that could cause actual results to differmaterially from those anticipated by the statements made herein.Forward-looking statements are generally identifiable by the use offorward-looking terminology such as "believe,""expects," "may," "looks to," "will,""should," "plan," "intend," "oncondition," "target," "see," "potential,""estimates," "preliminary," or "anticipates"or the negative thereof or comparable terminology, or by discussion ofstrategy or goals or other future events, circumstances, or effects.Factors that could cause actual results to differ materially fromthose expressed or implied in any forward-looking statements in thisrelease include, but are not limited to, statements regarding theoffer and sale of securities, the terms of the offering, about theability of Longeveron’s clinical trials to demonstrate safety andefficacy of the Company’s product candidates, and other positiveresults; the timing and focus of the Company’s ongoing and futurepreclinical studies and clinical trials and the reporting of data fromthose studies and trials; the size of the market opportunity for theCompany’s product candidates, including its estimates of the numberof patients who suffer from the diseases being targeted; the successof competing therapies that are or may become available; thebeneficial characteristics, safety, efficacy and therapeutic effectsof the Company’s product candidates; the Company’s ability toobtain and maintain regulatory approval of its product candidates inthe U.S., Japan and other jurisdictions; the Company’s plansrelating to the further development of its product candidates,including additional disease states or indications it may pursue; theCompany’s plans and ability to obtain or protect intellectualproperty rights, including extensions of existing patent terms whereavailable and its ability to avoid infringing the intellectualproperty rights of others; the need to hire additional personnel andthe Company’s ability to attract and retain such personnel; theCompany’s estimates regarding expenses, future revenue, capitalrequirements and needs for additional financing; the Company’s needto raise additional capital, and the difficulties it may face inobtaining access to capital, and the dilutive impact it may have onits investors; the Company’s financial performance, and the periodover which it estimates its existing cash and cash equivalents will besufficient to fund its future operating expenses and capitalexpenditure requirements. Additionally, Longeveron makes no assurancethat any public offering of its securities as described herein willoccur at all, or that any such transaction will occur on thetimelines, in the manner or on the terms anticipated due to numerousfactors. Further information relating to factors that may impact theCompany's results and forward-looking statements are disclosed inthe Company's filings with the Securities and Exchange Commission,including Longeveron’s Annual Report on Form 10-K for the year endedDecember 31, 2022, filed with the SEC on March 14, 2023. Theforward-looking statements contained in this press release are made asof the date of this press release, and the Company disclaims anyintention or obligation, other than imposed by law, to update orrevise any forward-looking statements, whether as a result of newinformation, future events, or otherwise.
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