Chondrial and Zafgen Complete Merger as Larimar Therapeutics and Raise $80 Million

Rare Daily Staff

Rare disease biotech Chondrial Therapeutics completed its reverse merger with Zafgen, to create a publicly traded clinical-stage biotechnology company that will operate under the name Larimar Therapeutics and closed an $80 million private placement.

Shares of Larimar began trading on the Nasdaq Global Market under the ticker symbol “LRMR.”

The financing will be used to fund a pipeline focused on treatments for complex rare diseases that uses an intracellular delivery platform to design fusion proteins targeting rare diseases characterized by deficiencies in intracellular bioactive compounds.

The company’s lead compound, 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.

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 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.

The U.S. Food and Drug Administration has granted CTI-1601, which is currently in a phase 1 study, Rare Pediatric Disease, Fast Track, and Orphan Drug designations. To date, two cohorts of patients have completed the single ascending dose phase 1 clinical trial. Larimar is working to initiate the third cohort in the trial, which is delayed due to the continued impact of the COVID-19 pandemic. Topline results from the phase 1 clinical program are planned for the first half of 2021.

“We are excited to complete this merger and become a publicly traded company as we develop treatments for complex rare diseases using our novel cell penetrating peptide technology platform,” said Carole Ben-Maimon, president and CEO of Larimar. “We believe our lead product candidate, CTI-1601, has the potential to become the first frataxin replacement therapy for patients with Friedreich’s ataxia.”

In conjunction with the reverse merger and public listing, Larimar appointed Joseph Truitt as chair of its Board of Directors. Truitt currently serves as CEO and a board member of Biospecifics Technologies, and prior to that was CEO of Achillion Pharmaceuticals.

In addition, Larimar appointed Nancy Ruiz, chief medical officer. Ruiz brings more than 20 years of global experience in all phases of clinical development and medical affairs, most recently as vice president of clinical development and head of drug safety at Prolong Pharmaceuticals.

Larimar has also appointed Michael Celano as chief financial officer. Celano has more than 15 years of experience as chief operating officer, chief financial officer, and a board member of multiple high-growth life science companies.

Photo: Carole Ben-Maimon, president and CEO of Larimar