Larimar Therapeutics Announces FDA has Removed Partial Clinical Hold for Nomlabofusp Program in Friedreich’s Ataxia
Larimar Therapeutics (Nasdaq: LRMR) announced that the FDA has removed the partial clinical hold on their nomlabofusp (CTI-1601) program, aimed at treating Friedreich’s Ataxia (FA). This decision followed the review of data from a recently completed Phase 2 dose exploration study, which included 25 mg and 50 mg cohorts. The study showed that nomlabofusp was well-tolerated and demonstrated dose-dependent increases in frataxin levels. Larimar plans to escalate the dose to 50 mg in the ongoing open-label extension (OLE) study. Interim data from this study is expected in Q4 2024, with a Biologics License Application (BLA) submission targeted for the second half of 2025.
- FDA removed partial clinical hold, allowing dose escalation to 50 mg.
- Phase 2 study showed nomlabofusp was well-tolerated.
- Phase 2 study demonstrated dose-dependent increases in frataxin levels.
- Interim data from the ongoing OLE study expected in Q4 2024.
- Biologics License Application (BLA) submission targeted for 2H 2025.
- Further dose escalation above 50 mg requires additional FDA review.
- Interim data from the ongoing OLE study won't be available until Q4 2024, which could delay stakeholder decision-making.
Insights
The FDA's removal of the partial clinical hold on Larimar Therapeutics' nomlabofusp program is a significant milestone. This decision validates the progress made in their Phase 2 trial and signals confidence in the safety and potential efficacy of the therapy. For investors, this is a strong indicator that Larimar is on a clear path towards advancing their treatment for Friedreich’s Ataxia (FA).
Financially, this can translate into enhanced company valuation and increased investor confidence, potentially leading to a rise in stock price. The projected interim data release in Q4 2024 and subsequent BLA submission in H2 2025 are key milestones to watch. These events could act as catalysts for the stock, driving investor interest and potentially leading to significant price movements around these dates.
Moreover, the predictable pharmacokinetic profile and dose-dependent increase in frataxin levels enhance the therapy’s attractiveness, potentially making it a competitive treatment in the FA market. However, the road ahead will require thorough monitoring of upcoming trial data to ensure the efficacy and safety profile is robust enough for eventual market approval.
The clearance of the partial clinical hold by the FDA suggests that nomlabofusp has shown promising results in the Phase 2 trial, particularly in terms of safety and pharmacodynamics. The treatment's ability to increase frataxin levels, a critical factor in FA, is a noteworthy development.
In scientific terms, achieving frataxin levels in skin cells over 33% of the average observed in healthy volunteers is a meaningful outcome. This indicates the therapy's potential to address the root cause of FA. The planned dose escalation to 50 mg and the ongoing evaluation of long-term safety are important steps in confirming the therapy’s viability.
However, the novel nature of this protein replacement therapy necessitates careful observation of the long-term safety data from the OLE study, particularly as the dosage increases. The biopharmaceutical industry will closely observe Larimar’s approach and results, as successful outcomes could pave the way for similar treatments for other rare diseases.
- Food and Drug Administration (FDA) removed partial clinical hold following review of Phase 2 dose exploration study data
- Ongoing open label extension (OLE) study initially evaluating 25 mg; Larimar plans to dose escalate to 50 mg following further characterization of frataxin pharmacodynamics (PD) at the 25 mg dose
- Interim data from OLE study remains on track for Q4 2024
- Biologics License Application (BLA) submission targeted for 2H 2025
BALA CYNWYD, Pa., May 20, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced the U.S. FDA has removed the partial clinical hold previously placed on the company's nomlabofusp (CTI-1601) clinical program. Nomlabofusp is currently in development for the treatment of patients with Friedreich’s Ataxia (FA). Nomlabofusp is a novel protein replacement therapy designed to address the root cause of FA by delivering frataxin to mitochondria. The FDA removed the partial clinical hold after a review of data from the Company’s recently completed four-week, placebo-controlled Phase 2 dose exploration study. The review included data from both the 25 mg and 50 mg cohorts in patients who received daily dosing of nomlabofusp for 14 days followed by every other day dosing until day 28.
“We are very excited the FDA has removed the partial clinical hold on our nomlabofusp program following review of our Phase 2 data. Helping patients with FA is our top priority and we appreciate the attention and thorough review by the FDA of all submitted data,” said Carole Ben-Maimon, MD, President, and Chief Executive Officer of Larimar. “Importantly, we are now cleared to dose escalate to the 50 mg dose in our ongoing OLE study which we plan to do following further characterization of frataxin PD at the 25 mg dose. The OLE study is evaluating the long-term safety as well as frataxin levels following daily administration of nomlabofusp and we look forward to interim data in the fourth quarter of the year.”
In the Phase 2 dose exploration study, nomlabofusp was generally well-tolerated throughout the four-week treatment period. Nomlabofusp had a predictable pharmacokinetic profile and demonstrated dose-dependent increases in frataxin levels in skin and buccal cells. All patients with quantifiable levels at baseline and Day 14 in the 50 mg cohort achieved frataxin levels in skin cells over
The long-term safety and tolerability, pharmacokinetics, and frataxin levels in peripheral tissues following nomlabofusp are currently being evaluated in the ongoing OLE study in patients with FA. The OLE study will initially evaluate daily subcutaneous injections of 25 mg of nomlabofusp self-administered or administered by a caregiver. Larimar plans to dose escalate to 50 mg in the OLE study following additional characterization of frataxin PD at the 25 mg dose. Further dose escalation above 50 mg, if necessary, would require submission of additional data for FDA review to support the increased dose. Interim data from the OLE study is expected in the fourth quarter of 2024.
About Nomlabofusp (CTI-1601)
Nomlabofusp is a recombinant fusion protein intended to deliver human frataxin to the mitochondria of patients with Friedreich’s ataxia who are unable to produce enough of this essential protein. Nomlabofusp has been granted Rare Pediatric Disease designation, Fast Track designation and Orphan Drug designation by the U.S. Food and Drug Administration (FDA), Orphan Drug Designation by the European Commission, and a PRIME designation by the European Medicines Agency.
About Larimar Therapeutics
Larimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. Larimar’s lead compound, nomlabofusp (CTI-1601), is being developed as a potential treatment for Friedreich's ataxia. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds. For more information, please visit: https://larimartx.com.
Forward-Looking Statements
This press release contains forward-looking statements that are based on Larimar’s management’s beliefs and assumptions and on information currently available to management. All statements contained in this release other than statements of historical fact are forward-looking statements, including but not limited to statements regarding Larimar’s ability to develop and commercialize nomlabofusp (also known as CTI-1601) and other planned product candidates, Larimar’s planned research and development efforts, including the timing of its nomlabofusp clinical trials, interactions with the FDA and overall development plan and other matters regarding Larimar’s business strategies, ability to raise capital, use of capital, results of operations and financial position, and plans and objectives for future operations.
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 nomabofusp 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:
Joyce Allaire
LifeSci Advisors
jallaire@lifesciadvisors.com
(212) 915-2569
Company Contact:
Michael Celano
Chief Financial Officer
mcelano@larimartx.com
(484) 414-2715
FAQ
What recent action did the FDA take regarding Larimar's nomlabofusp program?
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