Encoded Therapeutics’ lead asset ETX101 is a gene therapy being tested for Dravet syndrome. Phase 1/2 data showed substantial reductions in seizures.
Encoded Therapeutics has closed a $275 million series F round to help advance its investigational Dravet syndrome therapy into pivotal development.
Much of the proceeds will go toward Encoded’s lead asset ETX101, according to a Wednesday release. The therapy is being tested as a one-time disease-modifying gene regulator for children with Dravet syndrome who carry SCN1A mutations.
ETX101 is currently being studied among young children in the Phase 1/2 POLARIS program. After 52 weeks of observation, a single dose of EXT101 demonstrated a median of 79% reduction in monthly countable seizures for three patients, while five patients experienced a median 89% reduction, according to data shared Tuesday.
Encoded also touted “progressive gains” in cognitive and adaptive behavior following ETX101 treatment.
The California biotech will funnel the new cash into a pivotal trial of infants and young children, plus launch an expansion for children up to the age of 18 years. The company believes ETX101 has the ability to “meaningfully alter the course of Dravet syndrome,” he said in a Wednesday statement.
Dravet syndrome is a rare epileptic disease that onsets in infancy and continues throughout a patient’s life, accumulating symptoms such as frequent and prolonged seizures, developmental delays and motor problems, among others. Most patients with Dravet carry mutations in the SCN1A gene, which encodes a sodium channel protein and is associated with other seizure-related disorders.
Encoded’s ETX101 is designed to control the expression of this gene in specific cells to potentially address motor, cognitive, behavioral and developmental symptoms of Dravet.
Aside from ETX101, Encoded will also use its series F raise to advance its other pipeline assets, including the post-amputation neuroma pain therapy ETC301 toward an investigational new drug (IND) application next year. The biotech will also set aside some of its proceeds for the commercial scale-up of internal manufacturing capabilities.
The fundraising was co-led by GV (Google Ventures) and an undisclosed healthcare fund, with participation from ARCH Venture Partners, Farallon Capital Management, RTW Investments and others.
Encoded joins a flurry of biotechs that have announced funding efforts this week. On Tuesday, BrainChild Bio bagged $116 million in its series A round to help push into pivotal development for its glioma drug candidate. That same day, Moonwalk Biosciences brought in $70 million to advance its RNAi obesity therapy to clinical development.
Also on Tuesday, Caspian Therapeutics spun out of Kura Oncology with $50 million in starting capital to advance disease-modifying treatments for diabetes. Meanwhile, Solstice Oncology brought in $225 million in series A proceeds to develop a CTLA-4 therapy for cancer.
