Tag: Larimar Therapeutics

Larimar Therapeutics, Inc. (NASDAQ: LRMR) saw a 17.19% increase in after-hours trading on Monday, reaching $8.52, following a positive regulatory update. Investor excitement over recent advancements in the company’s clinical trials is reflected in this notable boost.

FDA Lifts Partial Hold On Nomlabofusp Program

Larimar Therapeutics (LRMR) announced that the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold on its nomlabofusp (CTI-1601) clinical program. Nomlabofusp, a pioneering protein replacement therapy, is being developed to treat Friedreich’s Ataxia (FA).

This therapeutic approach aims to address the underlying cause of FA by delivering frataxin directly to mitochondria. The FDA made its judgment after carefully examining the information from the recently finished four-week Phase 2 dosage exploration trial for Larimar, which was placebo-controlled.

Two cohorts participated in the trial; they were given dosages of 25 mg and 50 mg, respectively, and were dosed daily for the first 14 days, then every other day until day 28. Larimar was able to move forward with increasing the dosage in its current Open-Label Extension (OLE) trial to 50 mg as a result of this evaluation.

Promising Interim Data And Future Plans

The OLE study, which is currently evaluating the long-term safety and pharmacokinetics of nomlabofusp, will initially focus on 25 mg daily subcutaneous injections, either self-administered or administered by a caregiver. Larimar plans to escalate to a 50 mg dose after further characterizing frataxin pharmacodynamics (PD) at the 25 mg level. Should there be a need to escalate beyond 50 mg, additional data will be required for FDA review.

Nomlabofusp was generally well tolerated, with a predictable pharmacokinetic profile and dose-dependent increases in skin and buccal cell frataxin levels, according to the Phase 2 research. Notably, patients in the 50 mg cohort achieved significant increases in frataxin levels, with some reaching over 50% of the average levels observed in healthy individuals.

Interim data from the OLE study are anticipated in the fourth quarter of 2024, which will provide further insights into the long-term effects of nomlabofusp in patients with FA. This data is crucial for determining the future trajectory of the drug’s development and potential FDA approval for broader use.

Tuesday saw a notable increase in the price of Larimar Therapeutics, Inc. (NASDAQ: LRMR) shares during extended trading. After closing at $8.74 in the regular session, the company’s stock gained a significant 11.62% during the post-market period, reaching $10.05 on the US stock charts. The increase in LRMR shares came after a calculated move in equity.

With the opportunity for eligible investors to acquire pre-funded warrants for the acquisition of common stock shares, Larimar (LRMR) announced the commencement of its underwritten public offering of its common stock. The business also intends to provide the underwriters a 30-day window during which they can buy up to 15% more of the securities that will be offered in the planned offering.

Larimar will supply all of the common stock and pre-funded warrants for the offering, subject to the state of the market and other considerations. The participation of respected entities like Leerink Partners, Citigroup, and Guggenheim Securities as joint bookrunning managers, along with LifeSci Capital as lead manager, emphasizes the importance of this offering.

The intended use of the net proceeds is aimed at enhancing the development of nomlabofusp (CTI-1601) and other pipeline candidates, as well as addressing working capital requirements and general corporate expenses, particularly research and development efforts.

Recently, Larimar unveiled encouraging top-line data and the successful conclusion of its four-week, placebo-controlled Phase 2 dose exploration study concerning nomlabofusp (CTI-1601) in individuals affected by Friedreich’s ataxia (FA). The observed dose-response patterns and elevation in FXN levels within peripheral tissues further validate the therapeutic promise of nomlabofusp in tackling FXN deficiency, the underlying pathology of FA.

Notably, patients administered with 50 mg of nomlabofusp showcased initial FXN skin levels below 17% of the average healthy volunteer level. However, after a 14-day course of daily treatment, all patients with quantifiable baseline levels displayed FXN skin levels surpassing 33% of the average healthy volunteer level, with three patients achieving levels exceeding 50%.

Larimar’s recent market activities coupled with its promising therapeutic advancements underscore its commitment to addressing critical medical needs and fortifying its position within the biopharmaceutical landscape.