Longeveron's Laromestrocel Shows Promise in Phase 2a Trial for Mild Alzheimer's Disease
• Longeveron's Phase 2a CLEAR MIND trial demonstrated that laromestrocel (formerly Lomecel-B) is safe and well-tolerated in patients with mild Alzheimer's disease, with no reported cases of amyloid-related imaging abnormalities.
• The cellular therapy showed statistically significant improvements in cognitive function measured by the Montreal Cognitive Assessment, daily living activities, and preservation of brain volume compared to placebo.
• Laromestrocel has received both Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA, positioning it as potentially the first cellular therapeutic for Alzheimer's disease.
Longeveron's Lomecel-B Nears Full Enrollment in Pivotal HLHS Trial, Shows Promise in Pediatric Heart Disease
• Longeveron's Phase 2b ELPIS II trial for Lomecel-B in Hypoplastic Left Heart Syndrome has reached 90% enrollment, with completion expected in Q2 2025 and potential BLA submission in 2026.
• Phase 1 ELPIS I study demonstrated 100% transplant-free survival up to five years post-Glenn surgery in treated children, compared to historical 80% survival rate.
• The company reported a 237% revenue increase to $2.4 million in 2024, while reducing net losses by 25% to $16.0 million compared to previous year.
Landmark Prevention Trial Launches to Stop Alzheimer's Before Symptoms Begin in Young Adults
• WashU Medicine initiates groundbreaking international trial testing Eli Lilly's remternetug in young adults as young as 18, targeting Alzheimer's prevention up to 25 years before expected symptom onset.
• The Primary Prevention Trial will enroll 240 participants from families with genetic mutations, focusing on removing or preventing amyloid beta plaques before cognitive symptoms develop.
• The $130 million study represents a collaborative effort between academic institutions, pharmaceutical industry, and foundations, with support from NIH, Alzheimer's Association, and private donors.
Alzheimer's Disease Pipeline Shows Promise with 120+ Therapies in Development
• Over 120 Alzheimer's Disease treatment therapies are under development by 110+ companies globally, ranging from preclinical to marketed phases.
• Emerging therapies like NRDN-201, ST-501, and KarXT are in various clinical trial phases, showing potential for significant market impact.
• MapLight Therapeutics initiated a Phase 1 trial for ML-007/PAC, targeting schizophrenia and Alzheimer's disease psychosis, with Phase 2 trials planned.
• The FDA granted conventional approval to Leqembi (lecanemab-irmb), marking the first amyloid beta-directed antibody to transition from accelerated approval.
Advancements in Gene and Cell Therapies Target Diverse Diseases
• Ultragenyx seeks accelerated FDA approval for UX111, a gene therapy for MPSIII, based on Phase 1/2/3 trial data.
• Arbor Biotechnologies' CRISPR-based therapy ABO-101 receives clearance for US trial in Primary Hyperoxaluria Type 1.
• Allogene Therapeutics' ALLO-329 cleared by FDA for Phase 1 trial in rheumatology indications including lupus.
• uniQure progresses in trial for SOD1-ALS gene therapy AMT-162, advancing to the second cohort enrollment.
Longeveron's Lomecel-B Shows Promise in HLHS and Alzheimer's Disease Trials
• Longeveron's ELPIS II trial for HLHS, evaluating Lomecel-B™, has surpassed 80% enrollment and is deemed pivotal by the FDA for BLA submission.
• Positive Phase 2a data from the CLEAR MIND trial, assessing Lomecel-B™ in mild Alzheimer's, was highlighted at the Alzheimer's Association International Conference.
• Longeveron reported a 177% increase in revenue for the first nine months of 2024, driven by contract manufacturing and Bahamas Registry Trial demand.
• The company's current cash reserves are projected to sustain operations through the fourth quarter of 2025, supporting ongoing clinical development.
Longeveron's Lomecel-B Shows Promise in Mild Alzheimer's Disease by Targeting MMP14
• Longeveron presented data at CTAD24 showing Lomecel-B's potential in treating mild Alzheimer's by inhibiting MMP14, offering mechanistic and clinical insights.
• The study found that Lomecel-B's ability to inhibit MMP14 correlates with improved clinical and biomarker outcomes in patients with mild Alzheimer's disease.
• Lomecel-B exhibits immunomodulatory and pro-vascular effects, potentially driven by its MMP14 inhibition, which may protect TIE2 receptor integrity in Alzheimer's patients.
Longeveron's Lomecel-B Shows Long-Term Survival Benefit in HLHS Patients
• Longeveron presented data showing 100% five-year transplant-free survival in HLHS patients treated with Lomecel-B in the ELPIS I study, following Glenn surgery.
• The survival rate with Lomecel-B surpasses historical data from the Single Ventricle Reconstruction Trial, which reported 83% transplant-free survival and a 5.2% heart transplantation rate.
• The ELPIS II Phase 2b trial is ongoing, evaluating Lomecel-B as an adjunct therapy for HLHS, building on the positive results from the ELPIS I trial.
• Lomecel-B has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA, potentially expediting its development for HLHS.
Longeveron's Lomecel-B Shows Long-Term Survival Benefit in HLHS Patients
• Lomecel-B demonstrated 100% five-year transplant-free survival in HLHS patients post-Glenn surgery in the ELPIS I follow-up study, compared to 83% in the SVR trial.
• The ELPIS I trial met its primary safety endpoint, showing 100% survival and expected growth rates one year after Lomecel-B treatment in HLHS patients.
• Longeveron is currently conducting the ELPIS II Phase 2b trial to further evaluate Lomecel-B as an adjunct therapy for HLHS, building on the positive ELPIS I results.
• Lomecel-B has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA for HLHS, facilitating its development and potential approval.
Longeveron's Lomecel-B Data in Alzheimer's Disease to be Presented at CTAD24
• Longeveron's Lomecel-B data, focusing on MMP14 inhibition, will be presented at the Clinical Trials on Alzheimer's Disease Conference (CTAD24).
• The presentation will highlight Lomecel-B's bioactivity in treating mild Alzheimer's disease, offering mechanistic and clinical insights.
• Lomecel-B, derived from medicinal signaling cells, shows potential for anti-inflammatory and regenerative responses in aging-related diseases.
• The abstract will be published in the Journal of Prevention of Alzheimer's Disease, the official journal of the CTAD conference.
Longeveron's Lomecel-B Shows Promising 5-Year Transplant-Free Survival in HLHS Patients
• Longeveron's Lomecel-B demonstrates improved long-term transplant-free survival in infants with Hypoplastic Left Heart Syndrome (HLHS) over five years.
• The data, from the ELPIS I follow-on study, will be presented at the Congenital Heart Surgeons’ Society (CHSS) 51st Annual Meeting.
• Longeveron is currently conducting the ELPIS II Phase 2b clinical trial to further evaluate Lomecel-B as an adjunct therapy for HLHS.
• Lomecel-B has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the U.S. FDA for the HLHS program.
Cell and Gene Therapies Emerge as Promising Avenues for Alzheimer's Disease Treatment
• Several cell and gene therapies are under investigation for Alzheimer's disease, with some showing early signs of efficacy in clinical trials.
• NKGen's SNK01, an autologous NK cell therapy, demonstrated encouraging clinical activity and tolerability in a Phase 1 trial, with improvements in ADCOMS scores and CSF α-syn levels.
• Longeveron's Lomecel-B, an allogeneic MSC therapy, showed statistically significant improvements in ADCS-ADL scores and trends of improvement on MMSE in a Phase 2 trial.
• Beyond amyloid-targeting antibodies, researchers are exploring diverse targets like tau, inflammation, and viral proteins, with drug repurposing strategies also gaining traction.
Longeveron to Explore Partnerships for Alzheimer's Cell Therapy at Cell & Gene Meeting
• Longeveron will attend the Cell & Gene Meeting on the Mesa to seek partnerships for its Alzheimer's cell therapy program.
• The company's Lomecel-B™ showed promising Phase 2a results, slowing disease worsening in mild Alzheimer's patients.
• Lomecel-B™ has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA.
• Longeveron also highlights its contract development and manufacturing capabilities at its cGMP facility.
Longeveron Achieves FDA Alignment on Lomecel-B Pathway for Hypoplastic Left Heart Syndrome
• Longeveron secured FDA agreement on key endpoints for its Phase 2b ELPIS II trial of Lomecel-B™ in Hypoplastic Left Heart Syndrome (HLHS).
• The FDA indicated that a successful ELPIS II trial, meeting specific conditions, could support a Biological License Application (BLA) submission for Lomecel-B™.
• Lomecel-B™ has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA for HLHS treatment.
• ELPIS II builds upon positive results from ELPIS I, which demonstrated 100% transplant-free survival up to five years in treated children.
Cell and Gene Therapies Show Promise in Alzheimer's Disease Treatment
• Longeveron's Lomecel-B, an allogeneic medicinal signaling cell therapy, has shown promise in Phase IIa trials by slowing disease progression in Alzheimer's patients.
• Lexeo Therapeutics is developing gene therapies targeting APOE alleles, with LX1001 in Phase I/II trials, focusing on the 40-50% of Alzheimer's patients with APOE4.
• Early-stage companies like Regeneration Biomedical are also exploring stem cell therapies, showing cognitive improvements in late-stage Alzheimer's patients in Phase I trials.
• Cell and gene therapies offer a potential advantage over existing treatments by potentially reducing adverse events, though scalability and trial complexities remain challenges.
Lomecel-B Shows Promise in Phase IIa Alzheimer's Trial
• Lomecel-B demonstrated a slowing of disease worsening compared to placebo in a Phase IIa clinical trial for mild Alzheimer's Disease, marking a potential advancement in treatment.
• The trial data indicated that Lomecel-B has a strong safety profile, with no incidence of hypersensitivity or infusion-related reactions observed in both single and multiple dosing.
• Significant improvements were seen in the Alzheimer's Disease Cooperative Study Activities of Daily Living (ADCS-ADL) scale, suggesting a positive impact on patients' daily functions.
• Lomecel-B minimized brain volume loss in key areas affected by Alzheimer's, including the hippocampus, with statistically significant results for left hippocampal volume relative to placebo.
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