Chondrial Therapeutics has secured a near $23 million raise, led by VC Deerfield Management, and has taken on Deerfield’s adviser Carole Ben-Maimon as its new president and CEO.
Alongside its cash boost and top exec, the startup has also licensed an investigational candidate, CTI-1601, designed to treat Friedreich's ataxia, from Indiana University Research and Technology Corp. and Wake Forest University Health Sciences.
The funds will be used to push on with the development of CTI-1601 and to boost its exec and management team, with its labs now based at the Science Center in Philadelphia. Chondrial says it will soon ask for an IND by the FDA so it can start a phase 1 test of its new compound.
It added in a statement that it also plans to “advance [a] pipeline of product candidates focused on the treatment of rare mitochondrial diseases.”
New CEO Dr. Ben-Maimon, who has spent a quarter of a century in biopharma management and research, joins Chondrial after serving as an adviser with Deerfield Management.
She said: “The $22.6 million Series A financing, combined with the licensing of CTI-1601, is a watershed moment for Chondrial. I believe the company is in prime position to advance a potentially groundbreaking therapeutic for patients with Friedreich’s ataxia and capitalize on a broader vision of targeting and treating rare, debilitating mitochondrial diseases.
“We are very appreciative of Deerfield Management, which led the financing and will be providing continuing strategic and operational support. Additionally, we look forward to working with the scientific community, the Friedreich’s Ataxia Research Alliance, and the FDA to advance this important therapeutic.”
Discovered by co-founder Dr. Mark Payne while at Wake Forest Baptist Medical Center, CTI-1601 uses a carrier protein to deliver frataxin, the deficient protein in Friedreich’s ataxia, to the mitochondria where researchers believe it is processed to mature frataxin and becomes active in mitochondrial metabolism.
This genetic disease is a debilitating, life-shortening, degenerative neuromuscular disease that occurs in about 5,000 people in the U.S.
People with Friedreich’s ataxia have very low levels of frataxin, so Chondrial's big idea is that by replacing the deficient protein, the mitochondria will resume normal function, patients’ symptoms may be minimized, and disease progression may be held back.
This comes a few months after Horizon Pharma’s Actimmune missed the primary endpoint in a phase 3 trial in the rare neurodegenerative movement disorder, sparking a 20% drop in its stock price.
The study set out to show that interferon gamma-1b could improve outcomes in patients with Friedreich's ataxia, but the drug failed to move the needle.