Longeveron Inc. (NASDAQ: LGVN) is a small-cap biotechnology that should be on everyone’s radar. While still speculative, it is using cell therapies to target the broad market space of Alzheimer’s treatment. Specifically, he is using Lomecel-B for see if it can become a disease-modifying agent for these patients.
There was some published data from a phase 1 study showing that treatment with this drug was safe and helped patients achieve slower cognitive decline than placebo. The Phase 2 study is already recruiting patients and this is a huge program for biotechnology.
Although Longeveron fails in Alzheimer’s (AD), he still has more shots on goal using Lomecel-B for acute respiratory distress syndrome (ARDS) in Covid-19 patients and then in treating a rare disease known as Hypoplastic Left Syndrome. heart. Both of these programs are progressing into a phase 1 and a phase 2 respectively.
Finally, there is another indication in the pipeline that is risky, but great if it pays off. This would mean using Lomecel-B for the frailty of aging. A phase 1/2 study called HERA is expected to have the results published by the first half of 2022. This is risky, just like the Alzheimer’s indication, but it’s nice to see that Longeveron has four indications in the pipeline in progress.
Lomecel-B for the treatment of patients with Alzheimer’s disease
The first program in the pipeline is Lomecel-B, which was developed to treat patients with Alzheimer’s disease. Alzheimer’s disease is a terrible disease for which there is cognitive loss. These patients have memory loss and therefore also lose the ability to perform simple tasks.
There is currently no cure, and currently approved medications don’t help much, if at all. Aricept is one such standard of care drug (SOC) for Alzheimer’s, but it aims to increase acetylcholine levels in the brain by preventing acetylcholine from breaking down in the first place. It is a very broad market opportunity. It is expected that the global Alzheimer’s disease (AD) market. it could reach $ 25 billion by 2027.
The first drug approved for Alzheimer’s disease (AD) as a modifying agent is Aduhelm. Two main issues with Aduhelm are that he had a possible safety issue with hemorrhage swelling in the brain, and the FDA approval was surrounded by huge controversy. matter of fact, Biogen (BIIB) has withdrawn the application to receive approval for the drug in AD in Europe. Sales can’t really improve for that either, especially since the Center for Medicare and Medicaid Services said will not pay for Aduhelm outside of clinical trials. Essentially, Biogen has to rely on another drug for this indication known as Lecanamab. The data from this study will come out later in the fall of this year, so it’s important to see how it turns out. Regardless, this means that these AD patients need a new treatment option.
Longeveron hopes that Lomecel-B as a cell therapy can help AD patients improve in several ways. It remains to be seen how well it performs in the clinic, but the use of this therapy is being studied in a Phase 2a study that started in December 2021. This is a double-blind, randomized, controlled study with placebo that is trying to explore Lomecel-B for mild AD. One positive thing I like about this study is that Longeveron was able to implement the ability to test single and multiple doses of Lomecel-B for these patients. This flexibility is good, because if a single dose does not achieve the desired effectiveness, it is possible that multiple doses of it work better.
The ability to test multiple doses I believe stems from the excellent safety profile observed in a previous phase 1 study. This was a study using low dose Lomecel-B to treat these patients with mild AD. It was shown that Longeveron’s infusion of this cell therapy did not cause any security problems related to the treatment. Even better, there was no trace of a serious problem associated with other classes of Alzheimer’s drugs being investigated. These other drugs caused something known as ARIA.
What is ARIA? ARIA stands for amyloid-related imaging abnormalities. That is, monoclonal antibodies and other drug classes may have caused problems such as cerebral microhemorrhage or vasogenic edema. The bottom line is that doctors need to monitor such abnormalities that can occur with this class of drugs using magnetic resonance imaging. Longeveron doesn’t have to worry about that at all. Second, she saw some evidence of being able to help patients achieve slower decline in cognitive status.
An important cognition endpoint of this phase 1 study is known as the Mini Mental State Exam (MMSE), which is used to assess a person’s cognitive abilities. Patients who took Lomecel-B experienced slower cognitive decline than those who took placebo. Clearly not complete tangible evidence, but at least it has shown cell therapy to be quite active.
The good news is that ongoing phase 2a data should eventually shed some light on whether or not Lomecel-B is appropriate for treating patients with mild AD. A total of 48 patients will be enrolled in this study and updates are expected from the company on the completion of the recruitment study later. According to ClinicalTrials website, this is estimated to have a primary completion date of September 29, 2023. Of course, this is not the final date because it depends on how quickly these patients are ultimately recruited and then treated in this study.
According to Deposit 10-K SEC, Longeveron had cash and cash equivalents of $ 25.7 million, short-term investments of $ 9.4 million and working capital of $ 32.7 million as of December 31, 2021. It raised cash primarily through its IPO in 2021 and then the subsequent offers he issued. For its IPO, it raised about $ 26.7 million in net proceeds and then another $ 18.6 million from a 2021 PIPE offering.
The company believes it has enough liquidity on hand to finance its operations in 2024. I think it is well capitalized at the moment, but has already prepared to raise additional cash if needed. He filed a Form S-3 with the SEC to possibly use a shelf of mixed titles later if needed. It is effective with the SEC and you may choose to raise up to $ 50,000,000 from time to time through the sale of Class A common stock, preferred stock, warrants and / or purchase agreements.
Risks for businesses
The biggest risk to the company, I believe, relates to the Alzheimer’s study it is conducting. Why? Because there is a 99% trial failure rate in this space. Although it has shown that Lomecel-B has an excellent safety profile and some efficacy at the start, there is no guarantee that the Phase 2 study will ultimately be successful. I think this is an obstacle to consider for the AD space. This is because Biogen did not go well with Aduhelm’s FDA approval.
The conclusion is that Longeveron’s drug Lomecel-B must provide sufficient evidence of cognitive improvement. If not, it may not work well even with FDA approval. The good news is that Lomecel-B is applied to many other indications. In fact, it is being studied for use in patients with ARDS due to Covid-19, frailty of aging, and hypoplastic left heart syndrome.
Financials are not at risk at the moment, but I would say that I would not entirely rule out a cash increase earlier than expected. The fact that Longeveron submitted an S-3 form means that he plans to eventually use it later. I would argue that if the stock trades higher on good news, it will benefit from using this type of cash increase almost immediately.
The bottom line is that Longeveron is a great speculative biotech to buy. This is because it targets the huge space of Alzheimer’s disease. He is on the right track at the moment, because the safety profile was excellent. While there was some hint of efficacy, more tests on far more patients will be needed to determine if this was not a one-time event. At least cognitive decline slowed more in the Lomecel-B group than in the placebo group.
However, this effect must ultimately be achieved with statistical significance. It has many other great market opportunities such as ARDS targeting due to Covid-19 and the fragility of aging. He is even using Lomecel-B to treat a rare disease known as hypoplastic left heart syndrome. As for a catalyst that investors can expect, one could be the release of the results of the phase 2 study using Lomecel-B for the frailty of aging. Data from this study is expected to be published in the first half of 2022.