Longeveron LLC — Department of Health and Human Services SBIR Phase I: NIA

Description

Aging Frailty is a biologically driven geriatric syndrome of multisystem physiological decline that is distinct from normal aging and disproportionately increases vulnerability to adverse clinical outcomesFrailty has an overall prevalence ofof thoseyears and olderand there is growing awareness in the geriatric community to diagnose and treat this conditionas it is not an inevitable consequence of agingThe American Geriatrics Society has published aGeriatrics Evaluation andampManagement Tools Frailtyto aid practicing physicians in the diagnosis of Aging Frailtyand frailty dedicated societies and journals now exist due to the urgent need to develop effective interventionThere are no FDA approved therapies for treating Aging Frailtybut a biologically driven cell based therapy could have a major beneficial impactThe pathophysiology of Aging Frailty includes an accentuated pro inflammatory state that can promote systemic tissue damageincluding muscle atrophyMesenchymal stem cellsMSCshave potent antiinflammatory and pro regenerative propertiesmaking them an ideal therapeutic candidate for Aging FrailtyLongeveron is a manufacturer of a proprietary formulation of allogeneic MSCscalled LMSCswhich are being evaluated for therapeutic efficacy to treat Aging Frailty in this Phaseb trialWe recently completed a Phaseclinical trial to demonstrate the safety and tolerability of this approach for treating Aging FrailtyThat trial not only demonstrated the high safety profile of this therapeutic approachit provided provisional data showing efficacyThe Phaseb trial of this proposal represents the next step in clinical development of LMSCs as a therapeutic candidate for Aging FrailtyThis trial has been designed with guidance from FDA and international leaders in geriatricswhich was used to define enrollment criteria and endpointsFDA guidance for this clinical program is that a registrational endpoint would need to be a composite of surrogates for long term clinical outcomese gfallsfractureshospitalizationsinstitutionalizationand deathSpecificallythis composite would entail endpoints indomains relevant to Aging Frailtya metric of functional capacitya patient reported outcomePROand a biomarkerThis Phaseb trial is designed to examine endpoints in thesedomains in order to develop a composite endpoint to ultimately test prospectively in a Phasestudy for registrational purposesThis proposal addresses a truly novel application of cell based therapy that could become the first FDAapproved treatment for Aging FrailtyWe believe that Aging Frailty is an outstanding candidate indication for LMSC therapysupported by mechanism of actionpreclinical dataand Longeveron s Phaseclinical dataand has gained the support and endorsement of key opinion leaders in the fieldAccordinglythis study is highly timelyand has a high probability of making an extraordinary impact on a major unmet needas well as on regenerative medicine Aging Frailty is a large and growing unmet medical need that affects aboutof those ageyears and olderIt is a biologically driven geriatric syndrome that is distinct from normal agingand disproportionately increases vulnerability to adverse clinical outcomesThere are no FDA approved therapies for treating Aging FrailtyThe pathophysiology of Aging Frailty includes an accentuated pro inflammatory state that can promote systemic tissue damageincluding muscle atrophyMesenchymal stem cellsMSCshave potent anti inflammatory and pro regenerative propertiesmaking them an ideal therapeutic candidate for Aging FrailtyIn this clinical studyLongeveron will test a proprietary formulation of allogeneic MSCscalled LMSCsfor therapeutic efficacy to treat Aging FrailtyThis study is highly timelyand has a high probability of making an extraordinary impact on a major unmet needas well as on regenerative medicine