Epicrispr Biotechnologies has raised $90 million in a series C round backed by investors including Sanofi Ventures, positioning the biotech to advance a pipeline of epigenetic medicines led by an early-phase facioscapulohumeral muscular dystrophy (FSHD) prospect.
San Francisco-based Epicrispr began a phase 1/2 trial of the FSHD drug candidate, EPI-321, last year. A recent look at data from the trial showed increases in lean muscle mass in three patients with FSHD, a rare muscle degeneration disease. Six months after receiving a single intravenous infusion of EPI-321, the patients had gained between 0.5 and 1.3 pounds of lean muscle mass.
The interim readout provided preliminary clinical support for Epicrispr’s theory that suppressing DUX4 expression will improve outcomes in FSHD. EPI-321 uses a viral vector to get genetic material encoding an epigenetic editor to muscle cells, repressing the expression of toxic DUX4 protein.
Boosted by the readout, Epicrispr, a 2023 Fierce 15 honoree, has reeled in $90 million in a series C round co-led by Octagon Capital and Janus Henderson Investors. Multiple other investors, including Sanofi's VC arm, contributed to the financing round. The funding comes 16 months after Epicrispr raised $68 million to advance EPI-321 into the clinic.
The series C round cements Epicrispr as a rider in the race to treat FSHD, which currently lacks approved therapies. Novartis joined the race in February, when it completed its $12 billion takeover of Avidity Biosciences and gained control of its phase 3 antibody-oligonucleotide conjugate delpacibart braxlosiran. Meanwhile, Sarepta Therapeutics has an siRNA prospect in phase 1/2, and Scholar Rock recently started a phase 2 trial.
Epicrispr’s approach sets it apart from the other clinical-phase FSHD assets, although its focus on using epigenetic modification to modulate protein expression without directly editing DNA is shared by a small band of biotechs. NChroma Bio and Tune Therapeutics are developing epigenetic treatments for chronic hepatitis B.