Larimar Therapeutics begins dosing adolescents with Friedreich's ataxia in a pharmacokinetic study, expanding to younger children in 2025.
Quiver AI Summary
Larimar Therapeutics has initiated dosing for adolescents aged 12-17 in its pediatric pharmacokinetic (PK) run-in study for nomlabofusp, a potential treatment for Friedreich’s ataxia. Adolescents who complete this study will be eligible to participate in the ongoing open label extension (OLE) study. The company is also planning to start a study cohort for children aged 2-11 in the first half of 2025. Preliminary safety and exposure data for these groups will be analyzed to determine their transition into the OLE study. Long-term data for adult participants and available adolescent PK data are expected to be reported in mid-2025. The OLE study will assess the safety, tolerability, and therapeutic effects of nomlabofusp over an extended period.
Potential Positives
- Dosing of adolescents aged 12-17 has commenced in the pediatric pharmacokinetic run-in study for Friedreich’s ataxia, indicating progress in clinical trials.
- The company is actively enrolling adolescents and plans to expand by initiating a cohort of children aged 2-11 years old in the first half of 2025, demonstrating commitment to pediatric research.
- Long-term data from adults and the available pediatric data are expected to be reported in mid-2025, which could provide valuable insights into the treatment’s effectiveness and safety.
- LARIMAR is leveraging its intracellular delivery platform to potentially expand its product pipeline targeting other rare diseases, which could enhance its market position and growth prospects.
Potential Negatives
- Reliance on forward-looking statements indicates significant uncertainty regarding the success and timeline of drug development, potentially affecting investor confidence.
- Delayed access to long-term data for both adults and adolescents may hinder the perceived progress of the drug's development, impacting recruitment and investment interest.
- Increased scrutiny surrounding the clinical trial process, especially when involving vulnerable populations such as children, poses ethical challenges and potential reputational risks for the company.
FAQ
What is the current stage of Larimar's pediatric PK run-in study?
Larimar's pediatric PK run-in study is enrolling adolescents aged 12-17 years old and preparing to include children aged 2-11 in 2025.
What does the ongoing open label extension (OLE) study assess?
The OLE study evaluates the safety, tolerability, pharmacokinetics, and FXN levels after long-term administration of nomlabofusp.
When will Larimar report long-term data from the OLE study?
Long-term 50 mg data from adults and adolescent PK run-in data are expected to be reported in mid-2025.
Who is eligible to participate in the OLE study?
Participants from the PK run-in study who complete the assessment of safety and pharmacokinetics will be eligible for the OLE study.
What is nomlabofusp developed for?
Nomlabofusp is being developed as a potential treatment for Friedreich's ataxia, a rare genetic disorder affecting coordination and movement.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
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Full Release
- Adolescents receive a weight-based dose equivalent to the 50 mg adult dose
- Adolescents 12-17 years old who complete participation in the pharmacokinetic (PK) run-in study will be eligible to screen in the ongoing open label extension (OLE) study
- Larimar is continuing to enroll adolescent patients and plans to initiate a cohort of children 2-11 years old in 1H 2025
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Long-term 50 mg data from adults in OLE study and available adolescent PK run-in data expected mid-2025
BALA CYNWYD, Pa., Jan. 23, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that dosing of adolescents 12-17 years old has started in the Company’s pediatric PK run-in study for patients with Friedreich’s ataxia (FA).
“Dosing adolescents is the first step in evaluating the safety and PK of nomlabofusp in pediatric patients with FA. We continue to enroll adolescents in our first cohort. This cohort will be followed by a second cohort of children 2-11 years old. We expect to transition both the adolescents and children into the ongoing OLE study after assessing safety and exposure data from each successive cohort,” said Dr. Rusty Clayton, Chief Medical Officer of Larimar. “We look forward to reporting long-term 50 mg data in adults from our OLE study, as well as available data from adolescents completing the pediatric PK run-in study, in mid- 2025.”
Study participants in the PK run-in study are randomized 2:1 to receive either nomlabofusp or placebo daily for seven days. Following assessment of safety and PK data of each successive cohort, participants will be eligible to screen for the OLE study. A cohort of children (2- 11 years old) is planned to initiate in the first half of 2025.
The OLE study is evaluating the safety and tolerability, PK, and FXN levels in buccal and skin cells, along with exploratory pharmacodynamic markers (lipid profiles and gene expression) and clinical outcome measures, following long-term subcutaneous administration of nomlabofusp.
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, 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 and other planned product candidates, Larimar’s planned research and development efforts, including the timing of its nomlabofusp clinical trials, interactions and filings with the FDA, expectations regarding potential for accelerated approval or accelerated access and time to market 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 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 and commercialization 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
[email protected]
(212) 915-2569
Company Contact:
Michael Celano
Chief Financial Officer
[email protected]
(484) 414-2715