Phase 1 Trial of CTI-1601, Lab-made Frataxin, Dosing Final Patient Group

A safety and tolerability study of Larimar Therapeutics’ investigational therapy CTI-1601 has started dosing a third and final group of Friedreich’s ataxia (FA) patients, after a temporary pause due to the COVID-19 pandemic.

“We’re pleased that our Phase 1 clinical trial has resumed and we can continue to move forward with our lead product candidate, CTI-1601, which has the potential to become the first frataxin replacement therapy for patients with FA,” Carole Ben-Maimon, MD, Larimar’s president and CEO, said in a press release.

“Our highest priority remains the health of our employees and patients … We have thoughtfully re-engaged with our clinical site to mitigate the safety risks,” Ben-Maimon added.

CTI-1601 (formerly known as TAT Frataxin) is lab-made, modified version of frataxin — the protein lacking in FA. The modified protein is designed to enter mitochondria, the cells’ powerhouses, where it is expected to be processed and integrated into mitochondrial metabolism.

By restoring frataxin levels where needed, CTI-1601 may improve mitochondrial function, ease symptoms, and halt disease progression. Preclinical data in a mouse model of FA showed that CTI-1601 extended mean lifespan by 53% and improved cardiac function.

The therapy has received orphan drug, rare pediatric disease, and fast track designations by the U.S. Food and Drug Administration. These are meant to speed CTI-1601’s clinical development by providing regulatory support and financial benefits, including seven years of marketing exclusivity in the U.S. should the therapy be approved.

The Phase 1 trial (NCT04176991), taking place in New Jersey, is evaluating the safety, tolerability, and pharmacological properties of increasing doses of CTI-1601 in up to 32 adults.

Patients are being randomly assigned to either a single subcutaneous (under the skin) injection of CTI-1601 or a placebo.

The study’s main goal is to assess CTI-1601’s safety and tolerability, while secondary goals include the therapy’s pharmacokinetics — the movement into, through, and out of the body — and pharmacodynamics — its effects on the body.

Patients’ dosing began last year, and two of the three patient groups already completed the trial. With the trial’s delay, top-line results are now expected in the first half of 2021.

The potential frataxin replacement therapy was originally developed by Chondrial Therapeutics, which entered a merger agreement with Zafgen in late 2019, resulting in a new combined company, Larimar.