RARE Daily

Larimar Therapeutics Raises $70 Million After FDA Clears Full Clinical Hold on FA Therapy

September 14, 2022

Larimar Therapeutics raised $70 million in an underwritten public offering to advance the clinical development of its investigational treatment for Friedreich’s ataxia after the U.S. Food and Drug Administration changed its full clinical hold to a partial hold.

The company offered 22.2 million shares of common stock at $3.15 per share, which is equal to the closing price on Nasdaq for Larimar’s common stock on September 13, 2022. Larimar has granted the underwriters a 30-day overallotment option to purchase up to 3.3 million additional shares of common stock at the offering price, less underwriting discounts and commissions.

The FDA cleared the initiation of the 25 mg cohort of a phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in Friedreich’s ataxia (FA) patients. In a written communication to Larimar, the FDA indicated it was lifting its full clinical hold on the CTI-1601 program and imposing a partial hold. The design of the upcoming phase 2 trial is identical to the design proposed by Larimar, with the exception of a requirement for the FDA to review data from the 25 mg cohort prior to escalating the dose in the second cohort. Larimar expects to begin the phase 2 trial in fourth quarter of 2022, with top-line data expected in the second half of 2023.

Friedreich’s ataxia (FA) a is progressive, neurodegenerative condition caused by mutations in the FXN gene. Signs and symptoms usually begin in puberty and lead to progressive impaired muscle coordination, gradual loss of muscle strength and sensation in the arms and legs, muscle stiffness, and impaired speech.

The clinical hold followed notification by the company to the FDA of the death of non-human primates treated with the highest dose levels of CTI-1601 in an ongoing 180-day toxicology study. In the clinical hold letter, the FDA stated it needed a full study report from the ongoing non-human primate study and Larimar may not initiate additional clinical trials until the company has submitted the report and received notification from the agency that additional clinical trials may commence.

CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with Friedreich’s ataxia who are unable to produce enough of this essential protein. Currently in phase 1 clinical trials in the U.S., CTI-1601 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 PRIME designation by the European Medicines Agency.

Author: Rare Daily Staff

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