Encoded Therapeutics, Inc.

– Designation recognizes the potential of ETX101 to address a serious condition with high unmet need – SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- #Dravetsyndrome --Encoded Therapeutics, Inc. (“Encoded”), a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to ETX101 for the treatment of SCN1A+ Dravet syndrome. This designation is in addition to Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease Designations, all previously granted by the FDA. “Breakthrough Therapy Designation reflects the FDA’s recognition of both the urgent need for disease-modifying Dravet syndrome treatments and the potential of ETX101 as a meaningful treatment option for this devastating disease,” said Salvador Rico, M.D., Ph.D., Chief Medical Officer of Encoded. “Data from the ongoing Phase 1/2 studies showed durable reduction of seizures and improvements in neurodevelopment following a one-time administration. We are grateful to the FDA for recognizing the potential of ETX101 and look forward to continued collaboration to bring a meaningful new treatment option to the Dravet syndrome community as soon as possible.” Breakthrough Therapy Designation is a process designed to expedite development and review of investigational therapies that are intended to treat a serious or life-threatening condition and for which preliminary clinical evidence indicates the therapy may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints. The designation provides Encoded with more frequent and intensive FDA guidance, including the involvement of senior reviewers, as well as eligibility for rolling and priority review of the marketing application. About Dravet Syndrome Dravet syndrome is a severe genetic developmental and epileptic encephalopathy that begins in infancy and is most commonly caused by loss-of-function variants in the SCN1A gene. The disorder is characterized by frequent, prolonged seizures that are often resistant to treatment, as well as significant cognitive, behavioral, and motor impairments. Individuals with Dravet syndrome face a lifelong disease burden and an increased risk of premature mortality. Current treatment options are largely symptomatic and do not address the underlying genetic cause of the disease. More information about Dravet syndrome can be found at . About ETX101 ETX101 is an investigational AAV9-based gene regulation therapy designed to increase the expression of the SCN1A gene to restore sodium channel function in inhibitory interneurons. By addressing this root mechanism, ETX101 has the potential to treat the full spectrum of Dravet syndrome symptoms, including seizures, communication and cognitive impairment, behavioral issues, and motor dysfunction. The therapy is administered via a single intracerebroventricular (ICV) injection and is designed for long-term benefit. ETX101 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug Designations from the FDA, as well as Orphan Designation from the European Medicines Agency (EMA). About Encoded Therapeutics Encoded Therapeutics is a clinical-stage biotechnology company developing precision genetic medicines to transform the lives of patients with severe neurological disorders. The company’s vector engineering platform enables potent and cell-type-selective regulation of gene expression, allowing for targeted modulation of disease-relevant genes. Encoded is advancing a pipeline of programs across genetic epilepsies and other neurological disorders with significant unmet need. With integrated discovery, development, and manufacturing capabilities, Encoded is positioned to efficiently move programs from concept through the clinic. Encoded is driven by a mission to meaningfully improve the lives of patients and families affected by devastating neurological disorders. For more information, please visit . Contacts Investors/Business Development Jennifer Gorzelany communications@encoded.com Media Lori Rosen lori@redhousecomms.com

Plus, news about Bicara Therapeutics, Boehringer Ingelheim, Gubra, AbbVie, OSE Immunotherapeutics, Encoded Therapeutics, UCB and Immix Biopharma: 🤝 Two other companies were interested in Mersana: Day One wasn’t the only company to submit offers to acquire the ADC biotech, according to a regulatory document outlining their up to $285 million deal last month. The Friday evening SEC filing details how an undisclosed “Party A” submitted multiple offers to collaborate on Mersana’s B7-H4-targeted ADC, nicknamed Emi-Le. Another unnamed company, referred to as “Party B,” revised its offer to acquire Mersana multiple times. But Day One, which had backed out of deal discussions at one point, came out over the top with a higher upfront offer of $129 million. — Kyle LaHucik 🏎️ Another SPAC from Bihua Chen and Cormorant: The Cormorant leader and her investment firm are revving up the engines on Helix Acquisition Corp. III, with plans for a $125 million IPO outlined in a Monday morning SEC filing . The special purpose acquisition company (SPAC) will focus on finding a biotech company to take public. Cormorant’s prior two blank-check companies took BridgeBio Oncology Therapeutics and MoonLake Immunotherapeutics onto the Nasdaq. SPACs are seeing a somewhat of a comeback, with cancer-testing startup Freenome inking a SPAC deal last week. — Kyle LaHucik 📊 Bicara Therapeutics’ data reassures: An expansion cohort in a Phase 1b trial studied 750 mg weekly of ficerafusp alfa in a combo approach with Keytruda in first-line HPV-negative recurrent/metastatic head and neck squamous cell carcinoma. At ESMO Asia, the biotech said Saturday that its approach offered 57% confirmed overall response rate and that 10% of patients had a complete response. In a Sunday note, TD Cowen analysts wrote that the results “significantly derisks” the company’s pivotal Phase 2/3 trial studying the 750 mg and 1,500 mg doses . — Reynald Castaneda ⏩ Boehringer progresses Gubra’s obesity triple-agonist: The German pharma giant will move the candidate, called BI 3034701, into mid-stage development. The decision comes after “a favorable safety and tolerability profile” with “encouraging weight loss” in a Phase 1 in healthy volunteers. The partners did not disclose the Phase 1 data. Boehringer Ingelheim licensed the drug from the small Danish biotech. Boehringer axed a different obesity candidate in their collaboration last year. Meanwhile, Gubra’s amylin program serves as the anchor of AbbVie’s foray into obesity . — Kyle LaHucik ✍️ AbbVie adjusts OSE Immunotherapeutics deal: The Nantes, France-based biotech will now be responsible for Phase 1 development of a monoclonal antibody for chronic and severe inflammation. AbbVie can still decide to advance ABBV-230 after that Phase 1. OSE will no longer get a milestone payment for starting the Phase 1. That was part of an overall $665 million biobucks package for the $48 million upfront deal disclosed in February 2024. — Kyle LaHucik 📁 Encoded Therapeutics report data from POLARIS: The program includes three studies in the US, UK and Australia that are investigating ETX101 in patients aged 6 months to 7 years old who have Dravet syndrome because of SCN1A gene variants. The company said Friday that in 19 patients, the therapy led to “dose-dependent and sustained reduction” in a measure called monthly countable seizure frequency. — Reynald Castaneda 🏀 UCB’s Fintepla scores Phase 3 win in rare disease: The biotech’s drug produced a “statistically significant reduction” in countable motor seizure frequency in patients with a rare disease called CDKL5 deficiency disorder. It cut the frequency of seizures by about 48% from baseline, compared with 3% for placebo. UCB plans to file the data with regulators. Fintepla is already approved in the US for Dravet syndrome and Lennox-Gastaut syndrome. — Ayisha Sharma 💰 Immix Biopharma’s $100M offering: The biotech is looking to sell about 19 million shares at $5.10 apiece. This comes after Immix presented Phase 2 data for NXC-201 at ASH in relapsed/refractory AL amyloidosis. — Reynald Castaneda

Encoded Therapeutics expects to start a pivotal study of its Dravet syndrome gene therapy ETX101 in 2026. \n Encoded Therapeutics has linked its Dravet syndrome gene therapy ETX101 to a 78% reduction in seizures, keeping the biotech on track to start a pivotal study next year.The Californian company’s Polaris program is assessing ETX101 in three open-label, dose-escalation trials. Each study is enrolling children aged 6 months to 7 years with SCN1A-positive Dravet syndrome. The AAV9-based therapy is designed to improve outcomes in the patient population by boosting expression of the SCN1A gene that is implicated in most cases of the epileptic encephalopathy condition. Encoded arrived at the American Epilepsy Society Annual Meeting Friday with data on 19 patients who received one of four doses of ETX101. The biotech only has efficacy data for the 10 patients treated at the first three dose levels. No treatment-related serious adverse events were reported at any of the four doses, the company noted. At the third dose, Encoded reported a 78% median reduction in monthly countable seizure frequency. The figure comes from seven months of monitoring of three patients who were receiving anti-seizure medications at baseline. Encoded said the assessment covered a period that is typically associated with increasing seizure burden in young children. The biotech also shared a look at neurodevelopmental endpoints. All four of the children on the two lowest doses with 52 weeks of follow-up have shown “meaningful positive divergence” from untreated children in a natural history study, Encoded said. Receptive and expressive communication and motor function were the most notable improvements, followed by self-care and social interaction.Four of the five children treated before two years of age “showed substantial acceleration of cognitive skill acquisition as early as 16 weeks, with progressive gains through 52 weeks of observation,” Encoded said. The biotech called the rate of cognitive development “an important deviation from the developmental slowing and eventual plateauing” seen in its natural history study.The suggestion that ETX101 is most beneficial to children aged under 2 years has implications for the positioning of the gene therapy. While Dravet typically begins in the first year of life, Stoke Therapeutics is enrolling people aged 2 to 18 years in a phase 3 trial of its Biogen-partnered antisense oligonucleotide zorevunersen. The candidate is designed to increase levels of productive SCN1A messenger RNA. Stoke has a lead over Encoded, putting zorevunersen on course to be the first disease-modifying Dravet drug. Encoded expects to start a pivotal study in 2026. The biotech’s calendar for next year also includes reporting efficacy data from the fourth and final dose cohort. With the top dose appearing safe and tolerable, the readout could position Encoded to surpass the efficacy seen so far.Based on Encoded’s February update, the biotech will need to raise money or ink a partnership to fund the pivotal program. The company’s cash runway ran into the third quarter of 2026 as of the update. The runway reflected a 29% reduction in Encoded’s head count and decision to focus resources on ETX101.