Larimar Therapeutics is a biotechnology company, developing a treatment for Friedreich’s ataxia (FA) called CTI-1601. CTI-1601 replaces frataxin, which is the protein that is reduced in FA. More specifically, CTI-1601 is a fusion protein that delivers human frataxin to cells. Their Phase 2 trial, which is taking place in the US, is designed to identify the best dose of CTI-1601. Larimar Therapeutics recently announced the preliminary results of the first cohort of the trial, which included 13 people with FA.
In this part of the trial, participants were given either a 25 mg injection of CTI-1601 or a placebo daily for 14 days, then every-other-day until day 28. Participants’ frataxin levels were measured in skin cells and in cells found inside the cheek (buccal cells). The results showed that with CTI-1601 treatment there were increases in frataxin levels after 14 days compared to the placebo. All seven of participants who had CTI-1601 treatment had an increase in skin frataxin levels at day 14, compared to none of the four participants who were given a placebo. In buccal cells, five out of seven participants who had CTI-1601 treatment had an increase in frataxin levels at day 14, compared to neither of the two participants given a placebo. Please note that not all 13 participants in the study were included in this analysis as one participant withdrew from the study, and some participants did not have quantifiable levels of frataxin.
The safety data also showed that the treatment was generally well tolerated. No serious adverse events or important medical events were reported. One participant withdrew from the study due an allergic reaction which resolved with standard treatment.
The next step is for Larimar Therapeutics to start an additional cohort of the Phase 2 trial, where participants will be receiving a higher dose of the drug (50 mg). As the US drug regulators (the FDA) previously put a partial clinical hold on the CTI-1601 programme (more information here), the FDA need to grant clearance in order for Larimar Therapeutics to do this. Larimar Therapeutics have submitted the data from the 25 mg cohort to the FDA and have a meeting with them later this quarter. Larimar Therapeutics expect to provide an update on the next steps for their CTI-1601 programme in the third quarter of 2023, after they have received the feedback from their meeting with the FDA.
For more information, read the full press release here.