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Stoke Therapeutics, Inc. (NASDAQ: STOK) is a pioneering biotechnology company dedicated to addressing the root causes of severe genetic diseases. With a focus on developing antisense oligonucleotide (ASO) medicines, Stoke aims to increase gene expression by targeting RNA splicing. This innovative approach is designed to upregulate protein production, offering potential treatments for conditions that currently have limited therapeutic options.
One of the key technologies driving Stoke's mission is its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform. This advanced method allows for precise modulation of RNA to restore protein levels, providing a novel way to treat genetic disorders. The company's lead candidate, STK-001, is currently in clinical trials for the treatment of Dravet syndrome, a severe form of genetic epilepsy characterized by frequent and prolonged seizures. This promising candidate exemplifies Stoke's commitment to transforming the lives of patients with debilitating conditions.
Beyond Dravet syndrome, Stoke Therapeutics is actively exploring the application of its ASO platform to other severe genetic diseases. The company’s robust pipeline and strategic partnerships underscore its potential to deliver innovative therapies that address significant unmet medical needs.
Financially, Stoke Therapeutics has maintained a strong position, attracting strategic investments and partnering with leading research institutions to advance its clinical programs. The company primarily operates within the United States, leveraging its expertise and innovative technology to push the boundaries of genetic medicine.
For investors and stakeholders, Stoke Therapeutics represents a unique opportunity within the biotechnology sector, with its cutting-edge approach to genetic disease treatment and a clear focus on clinical and commercial milestones. Stay tuned for the latest updates and developments from Stoke Therapeutics as it continues to make significant strides in the field of genetic medicine.
Stoke Therapeutics has reported promising interim results from clinical studies of STK-001, showing a 55% median reduction in convulsive seizure frequency among patients treated with three doses of 45mg. All but one of the patients showed a positive response. The treatment was well-tolerated with mild to moderate adverse events reported due to the study drug. Data from the ongoing SWALLOWTAIL study indicates sustained reductions in seizure frequency and improved non-seizure related measures, marking potential progress in the treatment of Dravet syndrome.
Stoke Therapeutics (Nasdaq: STOK) announced that seven abstracts on Dravet syndrome will be presented at the American Epilepsy Society (AES) 2022 Annual Meeting in Nashville, Tennessee, from December 2-6. The abstracts include findings from the Phase 1/2a studies, MONARCH and ADMIRAL, highlighting significant reductions in seizure frequency with the investigational drug STK-001. The company aims to develop STK-001 as the first disease-modifying therapy for Dravet syndrome, targeting the underlying genetic cause of this severe epilepsy.
Stoke Therapeutics (Nasdaq: STOK) reported encouraging interim results from ongoing Phase 1/2a studies of STK-001, a treatment for Dravet syndrome, showing a 55% median reduction in convulsive seizure frequency with three doses of 45mg. The drug was well-tolerated, with 74% of patients experiencing reduced seizure frequency. As of September 30, 2022, the company had $252.2 million in cash, funding operations into 2025. The company plans to report more data in 2023 and has received FDA Rare Pediatric Disease Designation for STK-001.
Stoke Therapeutics, Inc. (NASDAQ: STOK) announced a webinar and conference call for analysts and investors on November 14, 2022, at 8:30 a.m. ET. This event will present interim analysis data from Phase 1/2a studies involving STK-001, a treatment aimed at children and adolescents with Dravet syndrome. Participants can join by dialing the provided numbers or accessing the live broadcast on Stoke's website. An archived replay will be available for 90 days post-event.
Stoke Therapeutics announced the enrollment of the first patient in its FALCON study, focusing on autosomal dominant optic atrophy (ADOA), a rare genetic disorder causing severe vision loss. The study aims to evaluate disease progression and support the development of STK-002, a potential therapy targeting the underlying cause of ADOA. ADOA affects approximately one in 30,000 people globally, with no approved treatments currently available. The FALCON study will enroll about 45 patients across 10 sites in the U.S., U.K., Italy, and Denmark.
Stoke Therapeutics (Nasdaq: STOK) reported its Q2 2022 financial results, showing revenue of $3.2 million from a License and Collaboration Agreement, compared to none in 2021. The net loss for the quarter was $24.7 million, with R&D expenses rising to $18.4 million. The company has approximately $276.2 million in cash, expected to fund operations into 2025. In Q4, interim data from clinical studies on STK-001 in Dravet syndrome will be announced, focusing on safety and seizure frequency. Additionally, STK-002 received FDA orphan drug designation for Autosomal Dominant Optic Atrophy.
Stoke Therapeutics (Nasdaq: STOK) announced its participation in the 2022 Jefferies Healthcare Conference on June 8, 2022, at 4:00 p.m. ET. The presentation will be in a fireside chat format, accessible via a live webcast through Stoke's Investors & News section. Stoke focuses on RNA-based medicines to address severe diseases, with their first compound, STK-001, in clinical testing for Dravet syndrome. For more info, visit stoke therapeutics.
Stoke Therapeutics reported financial results for Q1 2022, with a net loss of $24.6 million, or $0.66 per share, compared to $16.8 million, or $0.46 per share, in Q1 2021.
The company continues to advance STK-001 in clinical trials for Dravet syndrome, with data readouts expected in 2H 2022. They also announced progress in developing STK-002 for Autosomal Dominant Optic Atrophy (ADOA) and have $293.8 million in cash to fund operations through 2025.
Stoke Therapeutics presented promising preclinical data at the ARVO Annual Meeting, showcasing STK-002 as a potential disease-modifying treatment for Autosomal Dominant Optic Atrophy (ADOA), the most common inherited optic nerve disorder. The data revealed sustained increases in OPA1 protein levels in the retinal tissue of non-human primates following intravitreal administration of STK-002. Significant protein increases were observed even up to eight weeks post-injection, demonstrating the drug's potential to address the underlying cause of ADOA, which affects roughly 1 in 30,000 globally.
Stoke Therapeutics (Nasdaq: STOK) announced new preclinical data supporting the development of STK-002 for treating autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. This proprietary antisense oligonucleotide is designed to increase OPA-1 protein expression to halt or reverse vision loss. The findings will be presented at the ARVO 2022 Annual Meeting in Denver from May 1-4, 2022. Currently, ADOA affects approximately one in 30,000 people globally, with no approved treatment available.