Larimar Therapeutics Created Out of Merger, Will Advance Frataxin Therapy

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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Chondrial Therapeutics has merged with Zafgen, giving rise to Larimar Therapeutics, which will continue working to advance CTI-1601 as a possible frataxin replacement therapy for Friedrich’s ataxia (FA).

Larimar’s arrival came about five months after Zafgen and Chondrial announced they had entered into a merger agreement.

“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,” Carole Ben-Maimon, MD, president and CEO of Larimar, said in a press release.

“We believe our lead product candidate, CTI-1601, has the potential to become the first frataxin replacement therapy for patients with Friedreich’s ataxia. We are honored to have the support of both Chondrial’s and Zafgen’s existing shareholders, as well as new support from such a strong and respected syndicate of investors,” Ben-Maimon added.

Originally developed by Chondrial, CTI-1601 uses a special man-made carrier protein to deliver frataxin — the protein missing in people with FA — to mitochondria, the cell compartments responsible for the production of energy. By replenishing the levels of frataxin, CTI-1601 is expected to improve mitochondria function, with a goal of easing or stopping disease progression.

The safety, tolerability, and pharmacological properties of CTI-1601 are currently being investigated in a Phase 1 trial (NCT04176991) in up to 32 adults with Friedreich’s ataxia.

Patients are being randomly assigned to either increasing single doses of CTI-1601, or to a placebo, both administered by a subcutaneous (under the skin) injection.

The study’s main goal is to assess the number of patients with treatment-emergent adverse events within 70 days. Secondary goals include several evaluations of CTI-1601’s pharmacological properties, including pharmacokinetics (how a medicine moves through and is processed by the body), and pharmacodynamics (how it affects the body).

Dosing began last year, and Larimar reports that two of three patient groups have finished. The study is on a temporarily pause due to the COVID-19 pandemic, and plans are to resume as soon as possible. A third group may still be enrolling at the trial’s single New Jersey site; information is available here. Top-line results are expected in the first half of 2021.

The merger also resulted in a series of staff changes within Larimar, including a new chief medical officer, Nancy Ruiz.

“We are also pleased to welcome our new directors and leadership team members, whose guidance will be instrumental as we transition to a publicly traded company and continue to advance CTI-1601’s clinical development as a potential treatment for patients with Friedreich’s ataxia, a rare disease which currently has no approved medical treatment options,” Ben-Maimon said.

CTI-1601 has received orphan drug, rare pediatric disease and fast track designations by the U.S. Food and Drug Administration.