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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 (Nasdaq: STOK), a biotechnology company focused on RNA medicine for protein expression restoration, has announced its upcoming participation in the 43rd Annual J.P. Morgan Healthcare Conference. The company's management will deliver a presentation on Wednesday, January 15, 2025, at 6:00 p.m. ET (3:00 p.m. PT).
The presentation will be accessible through a live audio webcast on the Investors & News section of Stoke's website. Interested parties can access the webcast through the company's investor relations portal, and a replay will remain available for 30 days following the presentation.
Stoke Therapeutics (Nasdaq: STOK) has achieved alignment with FDA, EMA, and PMDA for its Phase 3 EMPEROR study of zorevunersen, potentially the first disease-modifying treatment for Dravet syndrome. The study will evaluate two loading doses of 70mg followed by two maintenance doses of 45mg over 52 weeks versus sham in patients aged 2 to <18 years.
The primary endpoint will measure reduction in major motor seizure frequency, while key secondary endpoints include improvements in cognition and behavior. Recent clinical data showed an 87% median reduction in convulsive seizure frequency at month eight in treated patients. The Phase 3 study, planned to start in mid-2025, will enroll approximately 150 patients globally, with data expected by end of 2027.
Zorevunersen has received FDA Breakthrough Therapy Designation, which may expedite development and review. The treatment has been generally well tolerated, with over 600 doses administered to patients, some remaining on treatment for more than three years.
Stoke Therapeutics (NASDAQ: STOK) has announced a webcast and conference call scheduled for January 7, 2025, at 8:00am Eastern Time to discuss successful regulatory alignment regarding a Phase 3 study of zorevunersen. The drug is being developed as potentially the first disease-modifying treatment for Dravet syndrome.
The company will host the event for investors and analysts, with research analysts having the opportunity to participate in a Q&A session through registration. The webcast will be accessible through Stoke's investor relations website, and a replay will be available for at least 90 days following the event.
Stoke Therapeutics (NASDAQ: STOK) announced new data from studies of zorevunersen for Dravet syndrome treatment. Nine patients treated with 2-3 doses of 70mg followed by 45mg maintenance dosing showed substantial seizure reductions, with an 87% median reduction at month eight. Patients demonstrated continuous improvements in cognition and behavior through 2 years of treatment.
The drug was generally well-tolerated across Phase 1/2a and OLE studies, with 81 patients treated. Safety findings showed 30% experienced treatment-related adverse events, mainly CSF protein elevations and procedural vomiting. The data support the company's proposed Phase 3 registrational study regimen.
Stoke Therapeutics has received FDA Breakthrough Therapy Designation for zorevunersen in treating Dravet syndrome with confirmed SCN1A gene mutations. Clinical data from Phase 1/2a and open-label extension studies showed substantial and sustained reductions in seizure frequency, along with improvements in cognition and behavior, while being generally well-tolerated. Over 600 doses have been administered, with some patients on treatment for more than three years.
The company plans to provide updates on its global Phase 3 registrational study by year-end. The Breakthrough Therapy designation grants zorevunersen access to Fast Track features, intensive FDA guidance, and organizational commitment from senior FDA managers.
Stoke Therapeutics (NASDAQ: STOK) announced upcoming presentations at the American Epilepsy Society 2024 Annual Meeting featuring new data on zorevunersen for Dravet syndrome. The data includes results from nine patients who received initial 70mg doses followed by 45mg maintenance doses, showing durable seizure reductions and improvements in multiple Vineland-3 subdomains through 24 months.
The company will present multiple posters and host a corporate symposium discussing the potential of disease-modifying treatments. Additionally, Stoke will conduct a virtual event for investors and analysts on December 9, featuring discussions with leading clinicians and patient advocates about the treatment landscape and real-world impacts of a disease-modifying medicine.
Stoke Therapeutics (NASDAQ: STOK) reported Q3 2024 financial results and business updates. The company ended Q3 with $269.2 million in cash and equivalents. Revenue was $4.9 million, up from $3.3 million in Q3 2023. Net loss was $26.4 million ($0.47 per share) compared to $24.5 million ($0.55 per share) last year. The company plans to share new data on zorevunersen's dosing regimen and complete regulatory discussions for Phase 3 study design by year-end. The FDA has removed the Partial Clinical Hold on zorevunersen, their lead program for Dravet syndrome.
Stoke Therapeutics (NASDAQ: STOK) presented promising data on zorevunersen (STK-001) for Dravet syndrome at the 15th European Epilepsy Congress. Key findings include:
1. Substantial seizure reductions: 85% median reduction at 3 months and 74% at 6 months after treatment.
2. Improvements in cognition and behavior within the first year of treatment, with additional increases as treatment continued.
3. Generally well-tolerated across studies.
4. Natural history study showed high seizure rates and developmental plateaus in Dravet patients despite standard treatments.
These results support zorevunersen's potential as the first disease-modifying treatment for Dravet syndrome, paving the way for a Phase 3 registrational study.
Stoke Therapeutics (Nasdaq: STOK) has appointed Eric Olson as Chief Business Officer. With two decades of experience in life sciences business development and strategy, Olson brings expertise in rare diseases and RNA therapeutics. He will lead the company's global corporate strategy and business development efforts.
Olson's appointment aims to expand Stoke's first-in-class, disease-modifying approach to treating genetic diseases by upregulating protein expression with RNA-based medicines. His experience includes leading over $15 billion in transactions for major biopharma companies, most recently as VP of Business Development at Alnylam Pharmaceuticals.
The company sees this hire as instrumental in driving its next growth phase, particularly in light of recent landmark data for zorevunersen in Dravet syndrome treatment. Stoke aims to leverage Olson's expertise to expand its TANGO platform to other severe diseases of the central nervous system and eye.
Stoke Therapeutics (Nasdaq: STOK), a biotechnology company focused on RNA medicine to restore protein expression, has announced its participation in two upcoming investor conferences in September 2024. The company will present at the H.C. Wainwright 26th Annual Global Investment Conference on September 10 at 12:00 p.m. ET and the 2024 Cantor Fitzgerald Global Healthcare Conference on September 17 at 11:30 a.m. ET.
These presentations offer investors an opportunity to gain insights into Stoke's progress and strategy. Live webcasts of both events will be available, with archived replays accessible on the company's investor relations website. This engagement with the investment community underscores Stoke's commitment to transparency and shareholder communication.