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Larimar treatments (Nasdaq: LMR) have had a few hiccups since I last wrote about this biotechnology. This was before Seeking alpha matter I wrote with the title “Larimar: Frederick’s ataxia drug With great potential“In this article, I pointed out that its drug nomlabofusp has already proven its ability to raise frataxin levels in patients with FA. However, since then, there have been some developments that have put this company on track for a better launch. Shot on getting approval for this particular drug, one of the key developments, which I think is very liberating for this biotechnology, is that the FDA has lifted the partial clinical block on this program below, but it’s all starting to fully be the clinical hurdle in 2021. And so far filled with a partial stop, he was only able to To dose patients with 25 mg nomlabofusp for FA.
With absolutely no restrictions from the US Food and Drug Administration (FDA), Larimar is free to test up to 50 mg doses of this drug now to treat these patients. Interim data from an open-label study, using nomlabofusp for FA, is expected in the fourth quarter of 2024. The second development, which should interest investors, is that the FDA has chosen to develop this FA indication to be part of its pilot program known as START. This program will select only 6 candidates from across this country to be tested as part of this pilot program. This is a great honor, and this program is specifically designed to accelerate the development of candidates targeting rare diseases with unmet medical needs. With several new key developments for nomlabofusp’s accelerated clinical development for FA, as well as interim data from the open-label study expected in Q4 2024, I believe investors could benefit from any potential gains made.
Nomalapusb for the treatment of patients with Friedreich’s ataxia
This company is developing the use of nomlabofusp to treat patients with Friedreich’s ataxia. This program is currently being developed in a phase II study program to treat these patients. Friedreich’s ataxia (FA) It is a type of disorder characterized as a rare and inherited neurodegenerative disease in which patients’ nerves are greatly affected. In fact, there are three parts of the body to which this disease causes problems: the spinal cord, the peripheral nerves, and the cerebellar part of the brain. One thing to note is that it greatly affects children and teenagers and causes them fatigue, frequent falls, as well as inability to coordinate movements. The global Frederick Ataxia market is expected to be It could reach $2.06 billion by 2030. The fact is that the FXN gene, which these patients have defective forms of, is responsible for creating a protein known as frataxin. Without the protein frataxin in place, the mitochondria it houses cannot produce energy or power organs. In contrast, patients with low levels of the protein frataxin cannot perform voluntary movements or other necessary functions. This is where Nomlaphosp comes in, as it is a recombinant fusion protein that is supposed to deliver frataxin to mitochondria. By doing so, it is believed that the course of the disease can be changed. In the previous article I wrote titled Above at the Beginning, I noted that a phase 1 study showed that this treatment was able to increase frataxin levels in a dose-dependent manner. If this theory is correct, removal of the partial clinical hold and subsequent testing of higher doses of 50 mg would mean that frataxin levels should be significantly increased.
Speaking of testing the higher 50 mg dose of nomalapusb, this is only possible because of the first development that I wanted to touch on as part of this update. That is, it was announced on May 20, 2024 that the Food and Drug Administration had obtained this Remove partial clinical evidence for this drug To treat this category of patients. The reason this is possible is because the company provided Phase 2 dose expansion data, which demonstrated that both 25mg and 50mg of this treatment are safe and tolerable over 4 weeks. In essence, patients were given these two doses of nomlaphosp every day for 14 days and then every other day until day 28. It was noted that no safety issues were found at all. The only drawback I see is that doses higher than 50mg will not be tested immediately. The plan is to start with long-term doses of 25 mg of nomalapusb, then if all goes well, move up to higher doses of 50 mg. Why is he doing that? This is because she wants to see how the pharmacodynamics work for the 25 mg dose first, before moving on to higher doses. Not only that, but although there is a small chance, it may be possible to move up to a much higher dose beyond 50 mg. The problem is that it won’t be able to do that until it sends new data to the FDA. From there, the agency will decide whether higher doses are possible.
The second update I wanted to highlight, and what I think will really move this program forward, is that nomlabofusp’s use of FA It was selected to be part of the FDA’s experimental program known as START. What is this program, and why is this a major development? This START program is ideal, because it only launched in September of 2023. Not only that, it is built on giving drugs the ability to go on an accelerated development path. This means more frequent interactions with the US Food and Drug Administration (FDA) and the ability to prepare applications that will be used to ultimately gain regulatory approval for this candidate. I find it very good that Larimar was able to get this recognition from the FDA. Especially when you take into consideration that there are only 6 companies that will be selected as part of this pilot program. The stock is up almost 100% since the last time I wrote about this. However, I believe further upside is possible as there are two important catalysts for investors to look forward to. The first catalyst includes the release of interim data from the phase 2 open-label study, using nomalapufosb to treat patients with Friedreich’s ataxia, expected in the fourth quarter of 2024. If the data from this study turn out to be encouraging, combined with other positive developments from it, it is expected that A Biologics License Application (BLA) for nomlabofusp for FA is submitted in the second half of 2025. However, it is important to note that such timing for a BLA submission may only be possible if an accelerated approval pathway is granted to Larimar for nomlabofusp.
Finance
According to the 10-Q SEC Filing, Larimar Therapeutics had $239 million in cash, cash equivalents, and marketable securities as of March 31, 2024. The reason it has cash on hand is because it was able to raise additional cash through the offering. This was concerning Obtaining $161.8 million in cash, after expenses, through a public offering of common shares. It sold an aggregate of 19,736,842 shares of its common stock, along with the underwriters’ option to purchase 2,574,370 shares of stock, at the same offer price of $8.74 per share. Why was this show in February 2024 such an important event? This is because it has now expanded its cash runway significantly. It believes it has enough cash on hand to fund its operations through 2026. Its cash burn per quarter is $16.7 million.
Risks to business
There are several risks that investors should be aware of before investing in Larimar Therapeutics. The first risk to consider will be related to the open-label phase 2 study, which uses nomlabofusp to treat patients with Friedreich’s ataxia. Interim data from this phase 2 study is expected to be released in the fourth quarter of 2024 and there is no guarantee that the data will be good. Additionally, the goal is to eventually expand to higher doses of 50 mg for this treatment. There is no way to know whether higher doses will result in increased frataxin levels in FA patients.
The second risk to consider is related to the removal of the partial clinical suspension of the Nomalapusb program for the treatment of patients with Friedreich’s ataxia. While a 4-week study showed that 25 mg and 50 mg of this treatment were safe and tolerable, this does not mean that there may not be any safety issues that could arise later. Therefore, it is entirely possible that some type of clinical hold could be placed back on this program if there is a significant safety event observed through clinical testing.
The third risk to consider is related to potential competitors in the field. As I mentioned in the previous article, Biogen (BIIB) It paid a huge sum to acquire Reata Pharmaceuticals. There are three other companies in various stages of clinical development that are using other drugs to treat these patients with FA. These other companies are as follows: PTC treatments (PTCT), Designing treatments (DSGN) and Lexio treatments (lexio). While all of these companies may eventually be competitors, there is one thing they have been unable to achieve, which is targeting the underlying cause of FA. Nomlabofusp is the first and only protein replacement therapy to help these patients. For this reason, the FDA may eventually allow the use of the surrogate label for increased frataxine levels as part of the accelerated approval pathway for U.S. marketing approval for nomlabofusp. It remains to be seen whether Larimar will achieve this goal, but if it does, it will have a significant competitive advantage over all of these other companies.
Conclusion
Larimar has had a bit of a bumpy road, especially when you consider that the FDA’s nomlabofusp was placed on a full hold by the FDA and then a partial clinical hold afterward. However, the removal of the partial clinical hold now puts it on a smoother path to success. What remains unknown is whether long-term data show that FA patients can tolerate higher doses of 50 mg nomlabofusp without any undue toxicity. I still rely on the long-term prospects of this biotechnology, primarily because it targets the underlying cause of FA, not like other treatments that don’t. If the theory of increasing frataxin levels with nomlabofusp is correct with further testing, it will be on its way to capturing a significant portion of the FA market. No matter how long or how good Biogen is Skylcares It does in sales over the next few years.
