Larimar Therapeutics reports Q2 EPS (19c), consensus (19c)

“Advancing our 50 mg cohort in our Phase 2 study and initiating our OLE study following FDA review of unblinded Phase 2 data from our 25 mg cohort are major advances in the development of CTI-1601 as potentially the first therapy designed to increase frataxin levels and address the underlying deficiency driving FA’s devastating clinical course,” said Carole Ben-Maimon, MD, President, and Chief Executive Officer of Larimar. “We are pleased to see that 25 mg was generally well tolerated and increased frataxin levels when dosed daily for 14 days. We are also very excited to now be able to initiate long-term dosing in the OLE trial in patients who participated in our Phase 1 or Phase 2 studies. The ability to follow patients in a long-term study that will assess increases in frataxin over months is very important to moving the development program forward. As we look ahead, we are focused on initiating our 50 mg cohort shortly, and plan to initiate our OLE trial in the first quarter of 2024. We expect to report top-line safety, pharmacokinetic, and frataxin data from the 50 mg cohort of our Phase 2 trial in the first half of 2024, and interim data from the OLE trial in the fourth quarter of 2024. Importantly, these trials will inform our future dose and dose regimen selection, as well as help to explore the long-term safety and pharmacokinetic and pharmacodynamic profiles of CTI-1601. With these key milestones ahead of us, coupled with our robust clinical dataset, we believe we are well-positioned to further advance the development of CTI-1601, with its unique mechanism of action and potential to be disease modifying for patients with FA.”