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BALA CYNWYD, Pa. - Larimar Therapeutics, Inc. (NASDAQ: LRMR), a biotechnology firm, reported last week on the progress of nomlabofusp, its investigational treatment for Friedreich’s ataxia (FA), at the International Congress for Ataxia Research in London. According to data from the company’s Phase 1 and Phase 2 studies, nomlabofusp has been associated with modified gene expression, improved lipid profiles, and increased frataxin levels in adults with FA.
The studies included 61 adults with FA and showed that a daily dose of 50 mg of nomlabofusp could likely achieve frataxin levels in most patients similar to those found in asymptomatic heterozygous carriers. These findings are significant as frataxin deficiency is the root cause of FA, a debilitating neurodegenerative disorder.
Dr. Carole Ben-Maimon, President and CEO of Larimar, highlighted that the gene expression and lipid profiles in patients with FA improved towards values seen in healthy controls after treatment with nomlabofusp. This suggests potential benefits in metabolic pathways disrupted by the disease.
The clinical research also established that the disease characteristics of the study participants were representative of the broader adult FA population. This is crucial for the development of nomlabofusp, as it supports the dose prediction modeling and aids in selecting appropriate doses for patients across different age groups and baseline characteristics.
Dr. Rusty Clayton, Chief Medical (TASE:PMCN) Officer at Larimar, expressed optimism about the Phase 2 study findings, which indicated a trend toward the normalization of dysregulated gene expression and lipid profiles. These initial data will be further analyzed with additional long-term data from an ongoing open-label extension study.
Larimar plans to provide a program update in mid-December 2024 and is targeting a Biologics License Application submission for the second half of 2025. The company also intends to initiate a pediatric pharmacokinetic trial later this year, expanding the study to include younger patients.
Larimar Therapeutics is committed to developing treatments for rare diseases and is leveraging its intracellular delivery platform to design additional fusion proteins targeting other conditions. The information in this article is based on a press release statement from Larimar Therapeutics.
In other recent news, Larimar Therapeutics has seen significant attention from various analyst firms. Citi has maintained its Buy rating on Larimar, with a steady price target of $14, and is focusing on the upcoming release of initial Open-Label Extension (OLE) data for the company's drug, nomla. Leerink Partners also maintained an Outperform rating on Larimar, with a price target of $25, anticipating key developments from the company's OLE study data. The firm Wedbush assigned an Outperform rating to Larimar, predicting that Larimar's drug, nomlabofusp, could enter the market by FY27 and potentially generate $1,502M in revenue by FY31.
Jones Trading initiated coverage on Larimar with a Buy rating and a price target of $14, forecasting that nomlabofusp could reach $1.3 billion in peak unadjusted sales by 2031. Baird initiated coverage with an Outperform rating and a $16 price target, citing a 60% probability of nomlabofusp gaining approval, possibly with accelerated approval by 2026.
These recent developments underscore a positive outlook on Larimar Therapeutics' prospects within the Friedreich's ataxia treatment landscape. The unique mechanism of action of nomlabofusp, directly targeting the underlying cause of the disease, is seen as a potential competitive edge in the market. All of these evaluations and predictions are from independent analyst firms and are not the opinions of this article.
InvestingPro Insights
Larimar Therapeutics' recent progress with nomlabofusp aligns with some intriguing financial metrics and analyst perspectives. According to InvestingPro data, the company's market capitalization stands at $426.87 million, reflecting investor interest in its potential breakthrough treatments for Friedreich's ataxia.
An InvestingPro Tip indicates that three analysts have revised their earnings upwards for the upcoming period, suggesting growing confidence in Larimar's pipeline and potential market impact of nomlabofusp. This positive sentiment is further supported by the company's impressive 1-year price total return of 103.34%, highlighting strong market performance over the past year.
However, it's important to note that Larimar is currently not profitable, with a negative P/E ratio of -6.59 over the last twelve months. This is not uncommon for biotechnology companies in the development stage, as they often prioritize research and clinical trials over immediate profitability.
Interestingly, another InvestingPro Tip reveals that Larimar holds more cash than debt on its balance sheet, which could provide financial flexibility as the company progresses towards its targeted Biologics License Application submission in the second half of 2025.
For investors seeking a more comprehensive analysis, InvestingPro offers 7 additional tips that could provide valuable insights into Larimar Therapeutics' financial health and market position.
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