BALA CYNWYD, Pa., Nov. 10, 2020 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Nasdaq:LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today reported its third quarter 2020 operating and financial results.

“I am very pleased with the progress Larimar has made over the past few months, as we achieved key clinical, regulatory, and corporate milestones that have left us well positioned for continued growth,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar Therapeutics. “In the third quarter, we continued to advance our lead program in development for Friedreich’s ataxia (FA), resuming our Phase 1 trials evaluating CTI-1601 in patients with FA. Though the trials were delayed due to the impact of the COVID-19 pandemic, they are now on track for topline results in the first half of 2021.”

Dr. Ben-Maimon continued, “Alongside this clinical achievement, we also complemented previous regulatory designations from the U.S. Food and Drug Administration (FDA) with an orphan drug designation from the European Commission and strengthened Larimar’s leadership team with the formation of a Scientific Advisory Board (SAB). Members of the SAB are key opinion leaders in the fields of rare disease, pediatrics, and mitochondrial disease who will provide strategic scientific guidance as we build our pipeline.”

Third Quarter and Subsequent Highlights

• In July 2020, Larimar resumed its Phase 1 clinical trials to evaluate the safety and tolerability of CTI-1601 for the treatment of FA with the dosing of its third cohort. The trials were previously delayed due to the impact of the COVID-19 pandemic. Topline data from the trials are expected in the first half of 2021.
• In August 2020, the European Commission granted an orphan drug designation for CTI-1601 for the treatment of FA. This designation complements previously received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA.
• In October 2020, Larimar announced the formation of its SAB. The SAB will provide strategic scientific guidance to company management and is comprised of key opinion leaders in the fields of rare disease, pediatrics, and mitochondrial disease. Members of the SAB include: Russell (Rusty) Clayton, DO; Marni J. Falk, MD; Giovanni Manfredi, MD, PhD; Mark Payne, MD; and Marshall Summar, MD.

Third Quarter 2020 Financial Results

As of September 30, 2020, the Company had cash, cash equivalents, and marketable debt securities totaling $102.3 million.

The Company reported a net loss for the third quarter of 2020 of $10.3 million, or $0.64 per share, compared to a net loss of $8.6 million, or $1.42 per share, for the third quarter of 2019.

Research and development expenses for the third quarter of 2020 were $6.9 million compared to $8.0 million for the third quarter of 2019. The decrease in research and development expenses compared to the prior year period was primarily driven by lower clinical supply manufacturing costs and toxicology studies partially offset by an increase in external clinical trial expenditures, an increase in personnel related costs due to headcount additions in our research and development functions and an increase in stock-based compensation expense associated with stock option grants made in July 2020.

General and administrative expenses for the third quarter of 2020 were $3.4 million, compared to $0.6 million for the third quarter of 2019. The increase in general and administrative expenses as compared to the prior year period was primarily driven by an increase in professional fees and insurance costs that are primarily due to the costs of operating as a public company, an increase in personnel related costs due to increased headcount, an increase in stock-based compensation associated with stock option grants made in July 2020 and an increase in facilities costs.

About CTI-1601
CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with Friedreich’s ataxia (FA) who are unable to produce enough of this essential protein. Currently in Phase 1 clinical trials in the U.S., CTI-1601 has been granted Rare Pediatric Disease designation, Fast Track designation and Orphan Drug designation by the U.S. Food and Drug Administration (FDA) and orphan drug designation by the European Commission. Topline results from the Phase 1 clinical program are planned for the first half of 2021.

About Friedreich’s ataxia
Friedreich’s ataxia (FA) is a rare, progressive, multi-symptom genetic disease that typically presents in mid-childhood and affects the functioning of multiple organs and systems. The most common inherited ataxia, FA is a debilitating neurodegenerative disease resulting in multiple symptoms including progressive neurologic and cardiac dysfunction – poor coordination of legs and arms, progressive loss of the ability to walk, generalized weakness, loss of sensation, scoliosis, diabetes, and cardiomyopathy as well as impaired vision, hearing, and speech. FA affects an estimated 4,000-5,000 individuals living in the United States and approximately 20,000 in the European Economic Area and United Kingdom. FA results from a deficiency of the mitochondrial protein, frataxin (FXN), which is found in cells throughout the body. To date, there are no medical treatment options approved for patients with FA.

About Larimar Therapeutics
Larimar Therapeutics, Inc. (Nasdaq:LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The company’s lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program in the U.S. as a potential treatment for Friedreich’s ataxia (FA). Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds. For more information, please visit: https://larimartx.com.