Larimar Therapeutics Announces the Dosing of the First Patient in Long-term Open Label Extension Study for Nomlabofusp in Patients with Friedreich’s Ataxia
Larimar Therapeutics initiates an open label extension study for nomlabofusp in patients with Friedreich's ataxia, aiming to assess long-term safety and tissue frataxin levels. The company plans to use data from this study to support a potential BLA submission for accelerated approval by H2 2025, with initial data expected in Q4 2024.
Larimar Therapeutics' initiation of an open label extension (OLE) study for nomlabofusp is a critical step in the drug development process, particularly for a rare disease like Friedreich’s ataxia (FA). The emphasis on long-term safety and tissue frataxin levels is significant, as it addresses the fundamental deficit in FA patients, who have reduced levels of the mitochondrial protein frataxin. A comprehensive understanding of the drug's pharmacokinetics and its impact on pharmacodynamic markers, such as lipid profiles and gene expression, is crucial for assessing its efficacy and safety profile over an extended period.
From a research perspective, the use of a synthetic control arm is innovative and may offer a more robust comparison for evaluating the drug's effectiveness in the absence of a large patient population, which is often the case with rare diseases. The potential use of frataxin as a novel surrogate endpoint could streamline the regulatory approval process, provided it is validated as a reliable indicator of clinical benefit. The anticipation of initial data in Q4 2024 will be a pivotal moment for stakeholders, as it will offer the first glimpse into the drug's long-term impact and could influence Larimar's stock performance and strategic planning.
The strategic move by Larimar to target accelerated approval with a Biologics License Application (BLA) submission for nomlabofusp in H2 2025 is noteworthy for investors. Accelerated approval pathways can significantly reduce the time and cost associated with bringing a drug to market, which is particularly valuable for biotech firms that operate with limited resources and under the pressure of financial sustainability. The successful transition from Phase 2 to the OLE study suggests confidence in nomlabofusp's potential, which could have positive implications for investor sentiment and Larimar's market valuation.
Investors should consider the risks associated with the FDA's partial clinical hold and the need for dose escalation data, as regulatory hurdles can impact development timelines and financial forecasts. The focus on a rare disease market, while often less competitive, requires careful consideration of market size and potential return on investment. The stock market reaction to the initial OLE study data release in Q4 2024 will likely hinge on the balance between safety outcomes and efficacy signals demonstrated by nomlabofusp.
The economic implications of a new treatment for Friedreich’s ataxia, a rare and debilitating disease, extend beyond immediate stock market considerations. The introduction of nomlabofusp could shift the treatment paradigm and potentially reduce long-term healthcare costs associated with managing FA symptoms and complications. The value of such a treatment is amplified in the rare disease space, where patients often have limited options.
Should nomlabofusp prove to be safe and effective, it could create a precedent for using surrogate endpoints like tissue frataxin levels in regulatory approvals for other rare diseases. This could have a broader economic impact by encouraging innovation and investment in treatments for rare diseases, which are often overlooked due to perceived lower profitability. However, the pricing strategy for nomlabofusp will be a critical factor in its economic viability and accessibility, given the challenges of balancing drug development costs with the affordability for patients and payers in a rare disease market.
03/11/2024 - 04:05 PM
Study will inform on long-term safety profile and tissue frataxin levels OLE initiated with 25 mg daily subcutaneous injections of nomlabofusp Frataxin data and safety data from the OLE study are intended to help support a potential Biologics License Application (“BLA”) submission for accelerated approval targeted for H2 2025 Initial data expected in Q4 2024 BALA CYNWYD, Pa., March 11, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced dosing of the first patient in an open label extension (OLE) study evaluating 25 mg daily subcutaneous injections of nomlabofusp in participants with Friedreich’s ataxia (FA). Nomlabofusp (CTI-1601) is a novel protein replacement therapy designed to address the root cause of Friedreich's ataxia (FA) by delivering frataxin to mitochondria.
“We are pleased to dose the first patient in our OLE study, further advancing the nomlabofusp clinical program and building on the successful completion of our Phase 2 dose escalation study. Importantly, the OLE study will inform on the long-term safety profile and tissue frataxin levels and provide a first look at real-life experience with self-administration by patients or administration by a caregiver. Participants who completed treatment in the recent Phase 2 dose exploration trial, or who previously completed a prior Phase 1 clinical trial of nomlabofusp are potentially eligible to screen for the OLE. Based on our Phase 1 and Phase 2 findings, we expect to continue daily dosing throughout the study,” said Carole Ben-Maimon, MD, President, and Chief Executive Officer of Larimar. “In February we announced that we intend to pursue frataxin as a potential novel surrogate endpoint to support accelerated approval. The frataxin data, supportive pharmacodynamics and clinical outcomes information and safety data from the OLE study, along with additional nonclinical pharmacology information will be used to help support a potential BLA submission for accelerated approval targeted for the second half of 2025. We look forward to reporting initial data from the OLE study in the fourth quarter of 2024.”
Larimar’s Phase 2 OLE study will initially evaluate daily subcutaneous injections of 25 mg of nomlabofusp self-administered or administered by a caregiver. Key study objectives of the OLE study include safety and tolerability, pharmacokinetics, and tissue frataxin levels in peripheral tissues as well as other exploratory pharmacodynamic markers (lipid profiles and gene expression data) following long-term subcutaneous administration of nomlabofusp. Clinical measures collected during the trial will be compared to data from a synthetic control arm derived from participants in the Friedreich’s Ataxia Clinical Outcome Measures Study (FACOMS) database. To escalate the dose in the OLE study, data from the 50 mg cohort of the Phase 2 dose exploration study, as well as available data from the 25 mg dose in the OLE study, will be submitted for FDA review due to the continued partial clinical hold. Initial data from the OLE study is expected in Q4 2024.
About Nomlabofusp (CTI-1601)
About Larimar Therapeutics https://larimartx.com .
Forward-Looking Statements
In some cases, you can identify forward-looking statements by the words “may,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “ongoing” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties and other factors that may cause actual results, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. These risks, uncertainties and other factors include, among others, the success, cost and timing of Larimar’s product development activities, nonclinical studies and clinical trials, including nomlabofusp clinical milestones and continued interactions with the FDA; that preliminary clinical trial results may differ from final clinical trial results, that earlier non-clinical and clinical data and testing of nomlabofusp may not be predictive of the results or success of later clinical trials, and assessments; that the FDA may not ultimately agree with Larimar’s nomlabofusp development strategy; the potential impact of public health crises on Larimar’s future clinical trials, manufacturing, regulatory, nonclinical study timelines and operations, and general economic conditions; Larimar’s ability and the ability of third-party manufacturers Larimar engages, to optimize and scale nomlabofusp’s manufacturing process; Larimar’s ability to obtain regulatory approvals for nomlabofusp and future product candidates; Larimar’s ability to develop sales and marketing capabilities, whether alone or with potential future collaborators, and to successfully commercialize any approved product candidates; Larimar’s ability to raise the necessary capital to conduct its product development activities; and other risks described in the filings made by Larimar with the Securities and Exchange Commission (SEC), including but not limited to Larimar’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov . These forward-looking statements are based on a combination of facts and factors currently known by Larimar and its projections of the future, about which it cannot be certain. As a result, the forward-looking statements may not prove to be accurate. The forward-looking statements in this press release represent Larimar’s management’s views only as of the date hereof. Larimar undertakes no obligation to update any forward-looking statements for any reason, except as required by law.
Investor Contact: jallaire@lifesciadvisors.com
Company Contact: mcelano@larimartx.com
What is the purpose of Larimar's open label extension (OLE) study?
The OLE study aims to evaluate the long-term safety profile and tissue frataxin levels in patients with Friedreich's ataxia receiving nomlabofusp.
What is nomlabofusp and how does it work?
Nomlabofusp (CTI-1601) is a novel protein replacement therapy designed to deliver frataxin to mitochondria, addressing the root cause of Friedreich's ataxia.
What will the data from the OLE study be used for?
The data from the OLE study will support a potential Biologics License Application (BLA) submission for accelerated approval targeted for H2 2025.
When is the initial data from the OLE study expected?
Initial data from the OLE study is anticipated to be reported in Q4 2024.
What are the key study objectives of Larimar's OLE study?
Key study objectives include assessing safety, tolerability, pharmacokinetics, tissue frataxin levels, and other exploratory pharmacodynamic markers of nomlabofusp.
