Rare Daily Staff

Zafgen and Chondrial Therapeutics have entered into a definitive merger agreement under which Chondrial will become a wholly-owned subsidiary of Zafgen and the stockholders of Chondrial Therapeutics will become the majority owners of Zafgen’s outstanding common stock upon the close of the merger.

The proposed merger will result in a combined publicly traded, clinical-stage biopharmaceutical company operating under a new name, Larimar Therapeutics.

“We are incredibly pleased to announce this proposed merger, as, once completed, we anticipate it will provide significant resources to advance CTI-1601, our novel therapeutic being developed for patients with Friedreich’s ataxia and expand our efforts on the development of additional potential treatments for other rare diseases,” said Carole Ben-Maimon, president and CEO of Chondrial Therapeutics. “Our current programs are based on our proprietary novel protein replacement therapy platform, which includes a cell penetrating peptide technology that allows the intracellular delivery of bioactive cargos. With this strong scientific foundation and company evolution, we believe we are well positioned to address complex rare diseases where patients are in need of innovative treatments.”

The merger announcement comes as the U.S. Food and Drug Administration granted Chondrial Therapeutics Rare Pediatric Disease designation and Fast Track designation for its experimental Friedreich’s ataxia therapy CTI-1601 as it begins a phase 1 clinical trial to evaluate its safety and tolerability.

Friedreich’s ataxia (FA) is a debilitating, life-shortening, progressive, rare, genetic disease that affects multiple body systems, particularly the brain and heart. It is the most common ataxia and is caused by a deficiency of a key protein, frataxin, which is normally present in the mitochondria of all tissues. Children and young adults progressively lose the ability to walk and speak, and their hearts become very thick and weak, and can eventually fail. There are currently no approved treatments and no cure for FA.

CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with FA, where researchers believe it is processed to mature frataxin and becomes active in mitochondrial metabolism. Due to a genetic abnormality, patients with FA are unable to produce enough of this essential protein. CTI-1601 is the only frataxin replacement therapy in clinical development. The FDA has granted Orphan Drug designation to CTI-1601.

The double-blind, placebo-controlled trial will enroll adult patients over age 18 with FA. It will evaluate the safety, tolerability and pharmacokinetics of single ascending doses of subcutaneously administered CTI-1601. Topline data from the Phase 1 clinical program are expected by the end of 2020.  

Fast Track Designation is intended to expedite review and facilitate development of drugs which are being developed to treat a serious or life-threatening condition and demonstrate the potential to fill an unmet medical need. The Rare Pediatric Disease designation means that Chondrial may qualify for a voucher if CTI-1601 is approved. The vouchers can be used to reduce the review time of a marketing application to six months from 10 months.

The vouchers are potentially lucrative because they are transferable. Most recently, Swedish Orphan Biovitrum sold its Priority Review voucher to AstraZeneca for $95 million.

Photo: Carole Ben-Maimon, president and CEO of Chondrial Therapeutics

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