BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR), a company dedicated to transforming the lives of patients through the development of innovative therapies, has made notable strides in the last twelve months. The company recently reported its full year and Q4 2023 operational and financial results.
Their drug, nomlabofusp, is showing promise in addressing frataxin deficiency, a known cause of Friedreich’s ataxia (FA). This genetic disorder ultimately leads to damage to the nervous system and the heart. The Phase 2 dose exploration study has been a crucial part of its development. The experiment reported top-line data and the successful completion of the Phase 2 dose exploration study.
Surprisingly, the observations have been extremely encouraging. The drug was well tolerated by patients with skin and buccal cell frataxin levels showing a dose-dependent increase. The most significant finding, however, is that skin frataxin levels in patients more than doubled, as a percentage of levels in healthy volunteers, after only 14 days of daily treatment with a 50 mg dose.
This exciting data suggests a profound therapeutic potential of nomlabofusp not only as a treatment but also as an agent to reverse frataxin deficiency, a root cause in Friedreich’s ataxia. The first patient in its OLE (open-label extension study) has also received their dose, marking another key milestone in the study. The OLE study is designed to monitor the long-term safety profile and development of long-term frataxin levels.
Dr. Carole Ben-Maimon, Larimar’s President and CEO, shares that they are “thrilled” with the progress, and that she believes the data positions them “strongly for execution” of upcoming crucial milestones. This, she emphasizes, is particularly relevant given the “urgent unmet needs of the FA community.”
In their ongoing discussions with the FDA, Larimar is working on a potential use of frataxin as a novel surrogate endpoint. This may unlock the potential for accelerated approval. Moreover, the company is planning for a global double-blind placebo-controlled confirmatory study. The study is expected to be initiated before the potential BLA (Biologics License Application) is submitted. The targeted timeline for the submission is the second half of 2025.
Regarding their financial status, Larimar ended 2023 with $86.8 million in cash, cash equivalents, and marketable securities. Following a public offering of common stock the following February, they raised an additional net amount of approximately $161.6 million.
From the financial perspective, the company reported a net loss in Q4 2023 of $13.0 million, which compares to a net loss of $9.4 million in Q4 2022. For the full year, the net loss was $36.9 million, a slight increase from $35.4 million in 2022.
In sum, the year 2023 was filled with significant progress for Larimar Therapeutics, marked by impressive clinical results, pivotal regulatory milestones, and a strengthened balance sheet. As we move further into 2024, industry watchers anticipate continued advancements for this groundbreaking pharmaceutical innovator.
For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and MSN.