On October 5th, the Miami-based biotech announced that Lomecel-B™ met its primary safety endpoint and showed a lack of deterioration in cognitive or atrophy signals in approximately 50 patients aged 60 to 85 years old with mild Alzheimer’s disease, marking a major milestone for the company. The successful trial paves the way for more clinical studies of this potentially game-changing treatment.

“We believe these results provide important validation of both the safety and therapeutic potential of Lomecel-B™ in the treatment of Alzheimer’s disease and provide a robust foundation for additional clinical trials in this and other indications,” Wa’el Hashad, CEO of Longeveron, said announcing the trial results. In late December, the company released additional new clinical and biomarker results from the trial that reinforced the earlier top-line findings. Additional analysis of cognitive function and daily living data showed favorable results with Lomecel-B™ over placebo. The drug was shown to slow and in some cases improve certain measurements of cognitive function (MoCA, MMSE). 

In addition to reporting promising results for Lomecel-B™, in October 2023, Longeveron secured $4 million in gross proceeds before expenses from a round of equity financing. In late December, Longeveron closed a previously announced registered direct offering of 1.35 million shares priced at $1.62 per share, raising an additional $2.36 million in gross proceeds. Longeveron plans to use the net proceeds to fund the ongoing clinical and regulatory development of Lomecel-B™ and for capital expenditures, working capital, and general corporate purposes. The company reported that as of the end of the third quarter, it has enough existing cash and short-term investments to cover expenses and capital requirements into the first quarter of 2024.

Some investors are hopeful that Lomecel-B™ could possibly play a role in the future in treating Alzheimer’s disease, which affects about 6.7 million people 65 and older in the U.S. In 2023 alone, Alzheimer’s and other dementias will cost the U.S. $345 billion, according to the Alzheimer’s Association. By 2050, total payments for healthcare, long-term care and hospice for those with dementia are projected to reach nearly $1 trillion. 

Lomecel-B™ appears to target inflammation, and it is believed that inflammation plays a role in the onset and progression of Alzheimer’s disease. Researchers made this discovery after several studies showed that people who took anti-inflammatory drugs like Enbrel and Humira were as much as 50% less likely to develop Alzheimer's disease later in life. These discoveries have in part helped to spur a new class of investigational drugs that aim to treat and cure Alzheimer’s disease by targeting inflammation. Lomecel-B™ is made from medical signaling cells (MSCs) derived from the bone marrow of healthy adult donors. It is believed that these cells travel to sites of damage or inflammation in the body and promote cellular regeneration and repair.  

Lomecel-B™ as a potential treatment for Alzheimer’s isn’t the only possible catalyst on the horizon for Longeveron, and investors may want to watch for more to come. The therapeutic treatment is also in stage 2 trials to treat Hypoplastic Left Heart Syndrome (HLHS) and Aging-related frailty. HLHS is a rare pediatric congenital heart defect that affects about 1,000 babies in the U.S. annually. The left side of the heart fails to develop in patients with HLHS – leading to short-term mortality, delayed development and long-term organ failure. 

Lomecel-B™ is administered directly into the cardiac tissue of the right ventricle for HLHS patients. The goal is to improve cardiac function through the regenerative, pro-vascular and anti-inflammatory effects of MSCs. Results from a phase 1 ELPIS 1 trial showed 100% survival in Lomecel-B™-treated children at up to five years of age. Historically, children with HLHS have about 20% mortality by five years. 

The results were encouraging enough for Lomecel-B™ to be given the Rare Pediatric Disease Designation (RPD), Orphan Drug Designation (ODD) and Fast Track Designation from the FDA. A phase 2 trial in HLHS of 38 patients is currently underway.  In August, the trial surpassed the 50% enrollment threshold.

The company is also in a phase 2 trial of Lomecel-B™ to treat aging-related frailty, which is characterized by mobility disability, weakness, fatigue, weight loss, slowness and low activity. Its current phase 2 trial will include 45 patients in Japan, where nearly a third of the population is over 65.