Benzinga | May 11, 2021 09:36AM EDT

Larimar Therapeutics Reports Positive CTI-1601 Data From Friedreich's Ataxia Trial

* Larimar Therapeutics Inc (NASDAQ: LRMR) has announced topline data from its Phase 1 multiple ascending dose trial evaluating CTI-1601 as a treatment for Friedreich's Ataxia (FA).

* FA is a genetic, progressive, neurodegenerative movement disorder with a typical onset between 10 and 15 years. It causes difficulty walking, a loss of sensation in the arms and legs, and impaired speech that worsens over time.

* 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.

* Cohort 3 (100 mg, n=7) of the treatment arm showed a change of 2.64 pg/?g total protein in buccal cells from baseline at day 13 compared to a mere 0.35 pg/?g total protein in the placebo group.

* Cohort 3 of the treatment arm showed a change of 3.55 pg/?g total protein in platelets from baseline at day 13 compared to negative 0.65 pg/?g total protein in the placebo group.

* Data shows that frataxin levels achieved in buccal cells following daily 50 mg and 100 mg CTI-1601 were at or more than those that would be expected in phenotypically normal heterozygous carriers.

* Safety data indicate that repeated subcutaneous injections of CTI-1601 were generally well tolerated at doses up to 100 mg administered daily for 13 days.

* The most common adverse events were mild and moderate injection site reactions.

* Patients who completed the single ascending dose (SAD) or MAD clinical trials are eligible to screen for an open-label extension clinical trial, the Jive study, which is expected to start in the second half of 2021.

* Larimar 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.

* CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria.

* Larimar will host a conference call and webcast today at 8:00 a.m. ET.

* Price Action: LRMR shares are trading 17.4% lower at $10.83 on the last check Tuesday.