Larimar Therapeutics Reports Positive Topline Phase 1 Clinical Trial Data Showing Dose-Dependent Increases in Frataxin Levels in Patients with Friedreich's Ataxia
BALA CYNWYD, Pa., May 11, 2021 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for Friedreich’s ataxia (FA) and other complex rare diseases, today announced topline data from its Phase 1 multiple ascending dose (MAD) clinical trial (n=27) evaluating CTI-1601 as a treatment for FA.
FA patients participating in the trial received subcutaneous injections of CTI-1601 or placebo at increasing dose levels and frequencies over a 13-day period. Patients in Cohort 1 were dosed with 25 mg of CTI-1601 or placebo daily for four days, and then every third day until Day 13. Cohort 2 patients were dosed with 50 mg of CTI-1601 or placebo daily for seven days, and then once every other day until Day 13. Patients in Cohort 3 received daily injections of 100 mg CTI-1601 or placebo for thirteen days.
Data show that repeated subcutaneous administration of CTI-1601 resulted in dose-dependent increases in frataxin (FXN) levels from baseline compared to placebo controls. These dose-dependent increases in frataxin levels were seen in all evaluated tissues (buccal cells, skin biopsies, and platelets) with daily dosing. The median change from baseline in frataxin levels observed for each dosing group and tissue are shown in the table below.
*FXN levels from Day 4 and 13 measurements are shown for data derived from Cohort 1. FXN levels from Day 7 and 13 measurements are shown for data derived from Cohorts 2 and 3. Sample collection days varied in each cohort per the clinical trial protocol.
“The ability of CTI-1601 to elevate frataxin in the evaluated tissues strongly supports its continued clinical evaluation, as frataxin levels in buccal cells and blood have been shown to be associated with disease severity in FA patients,” said Nancy M. Ruiz, MD, Chief Medical Officer of Larimar. “Notably, daily subcutaneous CTI-1601 administration resulted in median frataxin levels in buccal cells that are similar to, and even exceed, the levels we would expect to see in phenotypically normal heterozygous carriers (who have frataxin levels that are approximately 50% of unaffected healthy persons). These findings are exciting and represent a critical step forward in CTI-1601’s development as a frataxin replacement therapy for patients with FA. We believe CTI-1601 has the potential to address a critical unmet need, as FA is caused by patients’ inability to produce sufficient amounts of frataxin and current treatment options for this progressive and devastating disease are limited to symptom management. It is also important to note that CTI-1601 is the only drug candidate that we are aware of in clinical development that is designed to supplement frataxin levels in patients with FA, thus addressing the root cause of the disease.”
Safety data from the trial indicate that repeated subcutaneous injections of CTI-1601 were generally well tolerated at doses up to 100 mg administered daily for 13 days. A summary of the safety data, as well as pharmacokinetic data are shown below.
Patients who completed the single ascending dose (SAD) and/or MAD clinical trials are eligible to screen for an open-label extension clinical trial, the Jive study, which Larimar expects to initiate in the second half of 2021. Larimar also expects to initiate a MAD clinical trial in patients under 18 years of age in the second half of 2021. Patients completing this pediatric trial will also be eligible to screen for the Jive study.
Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar, commented, “We are thrilled with these data, which demonstrate proof-of-concept for CTI-1601 and highlight its safety and tolerability profile. Our ability to generate these results amid the pandemic speaks to the commitment of the patients who participated in the trials and the broader FA community. It is also a testament to the great work being done by the Friedreich’s Ataxia Research Alliance and the talent of our employees, partners, and investigators. We extend our sincerest thanks to all of these individuals for the important roles they have played in CTI-1601’s clinical development to date. We look forward to sharing additional quantitative analyses from our Phase 1 clinical trials at a future scientific meeting. We anticipate this data will inform the design of our planned future clinical trials for CTI-1601.”
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About the Phase 1 MAD Clinical Trial
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