Exhibit 99.2
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Larimar Therapeutics Announces Completion of Dosing of the Single Ascending Dose Clinical Trial in Friedreich’s Ataxia Patients and Provides Program Update
| • | Preliminary data suggest that single subcutaneous injections of CTI-1601 were well tolerated at doses up to 100 mg |
| • | Company remains on track to report topline data from placebo-controlled single and multiple ascending dose clinical trials in Q2 2021 |
Bala Cynwyd, PA, December 8, 2020 – Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced the completion of dosing from the Company’s Phase 1 single ascending dose (SAD) clinical trial (n=28) evaluating CTI-1601 as a treatment for Friedreich’s ataxia (FA) and provided additional updates regarding the status of an ongoing multiple ascending dose (MAD) clinical trial, the timing of Phase 1 topline results, and future activities planned for 2021.
A Safety Review Committee reviewed preliminary blinded data after each cohort of the placebo-controlled SAD clinical trial and recommended continuation of the trial. Dosing has been completed and based on preliminary data, single subcutaneous injections of CTI-1601 at doses up to 100 mg are thought to have been well tolerated. Injection site adverse events were mild and transient, and no serious adverse events were reported. Analysis of clinical trial results remains ongoing.
“At all doses tested, the Phase 1 SAD data support further investigation of CTI-1601,” said Nancy M. Ruiz, MD, Chief Medical Officer of Larimar. “FA is a rare and progressive disease that has a devastating impact on patient quality of life and mortality. Current treatment options are limited to symptom management. These preliminary Phase 1 findings represent a critical step forward in developing CTI-1601 as a potential frataxin replacement therapy for patients with FA and we look forward to its continued advancement through the clinic.”
Eligible participants from the SAD trial may enroll in an ongoing double-blind, placebo-controlled, MAD clinical trial. To date, dosing of the first two MAD cohorts has been completed, with dosing of the third cohort expected to begin in the first quarter of 2021. Larimar expects to report unblinded topline data from both the SAD and MAD trials in the second quarter of 2021.
Patients completing the SAD and/or MAD clinical trials are eligible to screen for an open-label extension clinical trial, which is expected 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.
Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar, commented, “That our Phase 1 program remains on track for topline data in the second quarter of 2021 despite the industry-wide challenges posed by COVID-19 is a testament to the talent and unwavering commitment of our employees, partners, and investigators. Their hard work has allowed us to advance CTI-1601’s clinical development without compromising the safety of our patients. These patients have shown a steadfast dedication to our clinical trials despite the pandemic, and for this we are extremely grateful. Looking forward, the safety of patients and employees will remain our top priority as we work to execute on clinical and corporate milestones in pursuit of our goal of developing this FA therapy.”