Larimar Therapeutics receives FDA clearance to proceed with next stage of trial - Ataxia UK

Larimar Therapeutics receives FDA clearance to proceed with next stage of trial

Post Published: July 27, 2023

Larimar Therapeutics is a biotechnology company developing treatments for rare conditions, including Friedreich’s ataxia (FA). They are testing a treatment for FA called CTI-1601, which is a protein replacement therapy designed to deliver the frataxin protein to the cells of people with FA. Frataxin is the protein which is decreased in people with FA. 

CTI-1601 is currently being tested in a Phase 2 clinical trial, taking place in the US. 

In May 2023, they reported that preliminary results from the first stage of this trial were promising (read more here). In the first stage of the trial, participants received a 25mg dose of CTI-1601 or placebo for 4 weeks. The US drug regulators (the FDA) reviewed the data from this part of the trial, and have now given clearance for the next stage of the trial to begin, in which participants will receive either placebo or a higher dose of 50mg of CTI-1601.  

The FDA also gave clearance for Larimar Therapeutics to start their open label extension trial, in which participants will receive 25mg of CTI-1601. The aim of this study is to evaluate the effects of long-term use of CTI-1601. 

In their press release, Larimar Therapeutics also state that they have begun to engage with drug regulators outside of the US, as they prepare to expand their clinical programme to other countries. 

Larimar Therapeutics expects to have results from the 50mg cohort of participants in the first half of 2024, and from the open label extension at the end of 2024. 

Read the full press release here.

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