Stoke Therapeutics, Inc. - STOK STOCK NEWS

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.

Stoke Therapeutics (NASDAQ: STOK) will present data from its Dravet syndrome clinical program at the 15th European Epilepsy Congress in Rome. The company is developing zorevunersen (STK-001), an antisense oligonucleotide, as the first potential medicine to address the genetic cause of Dravet syndrome.

Key presentations include:

• A platform session on MONARCH and ADMIRAL Phase 1/2a studies
• A poster on the 24-month analysis of the BUTTERFLY observational study
• A poster on SWALLOWTAIL and LONGWING open-label extension studies

Dr. Barry Ticho, Chief Medical Officer, emphasized the potential of zorevunersen to change Dravet syndrome treatment by addressing the root cause. The company is preparing for a Phase 3 registrational study of zorevunersen.

Stoke Therapeutics (Nasdaq: STOK), a biotechnology company focused on RNA medicine, has granted stock options to three new employees as an inducement for their employment. The grants, effective August 15, 2024, include options to purchase a total of 48,400 shares of common stock at an exercise price of $14.25 per share, matching the closing price on the grant date. These options will vest over 4 years, with 25% vesting after one year and the remainder vesting monthly over the following 36 months. The options have a 10-year term and are subject to the company's 2023 Inducement Plan and individual stock option agreements.

Stoke Therapeutics (NASDAQ: STOK) reported Q2 2024 financial results and provided business updates. Key highlights include:

1. FDA removed Partial Clinical Hold on zorevunersen for Dravet syndrome
2. Phase 3 registrational study plans update expected in H2 2024
3. Phase 1 study (OSPREY) of STK-002 for ADOA to begin this year
4. $282.0 million in cash and equivalents as of June 30, 2024
5. Q2 2024 revenue: $4.8 million; Net loss: $25.7 million ($0.46 per share)
6. R&D expenses: $21.1 million; G&A expenses: $13.0 million

The company continues to advance its RNA medicine pipeline, with zorevunersen showing promising results in reducing seizures and improving cognition in Dravet syndrome patients.