Encoded raises $275m for Dravet therapy and other financings

Right after reporting encouraging data with its Dravet syndrome gene therapy, Encoded Therapeutics has raised an impressive $275 million to take the programme into a pivotal trial.

At the European Epilepsy Congress (EEC) this week, Encoded showcased results of the phase 1/2 POLARIS trial of ETX101 in Dravet syndrome associated with mutations in the SCN1A gene, including a big reduction in seizure frequency – compared to historical controls – in the 12 months after the one-shot therapy.

Dravet syndrome is a severe form of developmental and epileptic encephalopathy which begins in infancy and causes prolonged seizures that do not respond well to current anticonvulsant drugs, as well as behavioural and developmental delays.

Treatment with ETX101 was also associated with "encouraging" developmental gains among subjects in POLARIS, according to the South San Francisco company.

The new financing, co-led by GV and another unidentified healthcare fund, will also fund the scale-up of manufacturing for ETX101 for forthcoming trials, as well as development of a second drug candidate – codenamed ETX301 – that Encoded has in preclinical testing for post-amputation neuroma pain.

The financing was also supported by ARCH Venture Partners, Braidwell LP, Farallon Capital Management, Illumina Ventures, Invus, Janus Henderson Investors, Matrix Capital Management, Nolan Capital, RTW Investments, SoftBank Vision Fund 2, and Venrock.

Other recent financing rounds

Also this week, Seattle startup Brainchild Bio closed a $116 million Series A round earmarked for clinical trials of its CAR-T therapies for central nervous system cancers, headed by BCB-276, a B7-H3-targeted therapy for rare and aggressive paediatric tumour diffuse intrinsic pontine glioma (DIPG). The therapy is currently in the phase 2 ILLUMINATE study, with results due in 2028.

An undisclosed private family fund and foundation led the round, with participation from the company's seed investor, Seattle Children's Hospital, and new investor WRF Capital.

South San Francisco-based Moonwalk Biosciences, which specialises in RNA interference drugs for obesity and cardiometabolic diseases, added to its coffers with a $70 million second round co-led by Alpha Wave and YK Bioventures and backed by Eli Lilly, Gaorong Ventures, and existing investors ARCH Venture Partners, Khosla Ventures and Future Ventures.

Armed with the new cash, Moonwalk plans to take its lead drug, MW101 for obesity, into phase 1 testing before the end of next year. The drug, which uses adipose-selective delivery chemistry licensed from China's Suzhou Siran Biotechnology, acts via an undisclosed, non-incretin mechanism that could differentiate it from GLP-1 agonist therapies for weight loss.

TwoStep Therapeutics raised $62.5 million in a Series A, providing the resources needed to take its lead peptide-drug conjugate programme, TS-104, into clinical testing for solid tumours. According to San Carlos, California-based TwoStep, TS-104 uses a polyspecific integrin-binding peptide (PIP) that binds to multiple integrins found on solid tumours, delivering a cell-killing MMAE payload.

Insight Partners and Medical Excellence Capital acted as co-lead investors, with Merck KGaA's VC arm M Ventures and Pfizer Ventures also taking a prominent role, with existing backers NFX, 2048 Ventures, and Stanford University also participating.

Kura Oncology has spun out a new biotech, Caspian Therapeutics, that will develop its menin inhibitor technology outside cancer with the help of $50 million in financing from BVF Partners, supported by Eli Lilly, the T1D Fund, Invus, Montanova Capital, and Kura – which is retaining a half-share in the startup.

Caspian's lead asset is KO-7246, a menin inhibitor currently in preclinical development that could have potential in type 1 and type 2 diabetes and other cardiometabolic disorders by encouraging the expansion of insulin-producing beta cells in the pancreas. The new San Diego biotech said it plans to file for approval to start human trials of KO-7246 "as soon as practicable."

Finally this week, New Haven, Connecticut's Cloverleaf Bio has emerged with $33 million in seed financing and a mission to develop a novel class of engineered transfer RNA (tRNA) based payloads for cancer. Its lead programmes – both in preclinical development – are CLB-001, an inhibitory tRNA asset targeting hepatocellular carcinoma, and an antibody-tRNA conjugate (ATC) asset for colorectal cancer.

The round was led by 4BIO Capital with backup from AbbVie Ventures, Eli Lilly, and Boehringer Ingelheim Venture Fund, alongside Draper Associates, Mission BioCapital, and American Cancer Society BrightEdge.