BEDFORD, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine, today announced that its Board of Directors has appointed Ian F. Smith as Chief Executive Officer (CEO). Mr. Smith has served as Interim CEO since March 2025 and as a member of Stoke's Board of Directors and advisor to the Company since 2023. He will continue to serve as a Director on the Company's Board of Directors ...

Company Highlights - Aurinia Pharmaceuticals Inc. (AUPH) has seen solid growth for its product LUPKYNIS, with expected net product sales for 2025 ranging from $250 million to $260 million, up from $216.2 million last year [2][3] - Arrowhead Pharmaceuticals Inc. (ARWR) is preparing for its first commercial launch in 2025, with its lead drug candidate Plozasiran under FDA review, decision expected on November 18, 2025 [4][5] - Beta Bionics Inc. (BBNX) reported a 54% growth in second-quarter net sales, with expectations for full-year 2025 net sales to range between $88 million and $93 million, compared to $65.1 million last year [7][8] - Kiniksa Pharmaceuticals International plc (KNSA) anticipates net product revenue for its drug Arcalyst to be between $625 million and $640 million for 2025, up from $417 million in 2024 [10][11] - Ionis Pharmaceuticals Inc. (IONS) generated net product sales of $19 million in Q2 2025, with a supplemental new drug application expected by year-end [12][14] - Stoke Therapeutics Inc. (STOK) is developing Zorevunersen for Dravet syndrome, with a 52-week high of $24.60 reached recently, representing an 82% gain in less than two months [16][17] - Fortress Biotech Inc. (FBIO) is awaiting an FDA decision on its investigational drug CUTX-101 for Menkes disease, expected on September 30, 2025 [18][21] Stock Performance - AUPH stock increased by 168% over 1.5 years, reaching a 52-week high of $12.87 [3] - ARWR stock gained 65% in less than 9 months, hitting a 52-week high of $31.13 [5] - BBNX stock rose nearly 55% in less than 4 months, reaching a 52-week high of $26.66 [8] - KNSA stock saw a 46% increase in less than four months, touching a 52-week high of $37.34 [11] - IONS stock gained 52% in less than two months, reaching a 52-week high of $64.72 [15] - STOK stock increased by 82% in less than two months, achieving a 52-week high of $24.60 [17] - FBIO stock rose by 61% in less than a month, reaching a 52-week high of $3.97 [21]

Summary of Stoke Therapeutics FY Conference Call Company Overview - **Company**: Stoke Therapeutics (NasdaqGS:STOK) - **Focus**: Development of treatments for haploinsufficient diseases, primarily targeting Dravet syndrome, a genetic epilepsy caused by a mutation leading to a lack of NAV 1.1 protein expression [3][4] Core Points and Arguments - **Dravet Syndrome**: A severe genetic epilepsy characterized by early-onset seizures and neurodevelopmental issues, with affected children often not developing neurotypically beyond age two [4][3] - **Mechanism of Action**: The company utilizes an Antisense Oligonucleotide (ASO) to upregulate NAV 1.1 protein, addressing the root cause of Dravet syndrome [4][7] - **Clinical Trials**: Currently in Phase 3 trials, with a significant number of patients pre-screened and a healthy financial position to support the trial [5][39] - **Efficacy Data**: - Approximately 85% median reduction in seizures observed in patients on top of standard care [10][11] - Long-term data shows durable seizure reduction and improvements in cognition and behavior, measured by the Vineland scale [12][14] - **FDA Breakthrough Designation**: Received for the treatment of Dravet syndrome, highlighting the unmet medical need and potential benefits of the therapy [8][49] Important but Overlooked Content - **Patient Demographics**: The treatment shows potential benefits across a broad age range, including older patients, due to neuroplasticity [36][33] - **Trial Design**: The Phase 3 study is a 52-week sham-controlled trial with a primary endpoint focused on seizure reduction and secondary endpoints on cognitive and behavioral improvements [38][41] - **Enrollment Demand**: There has been a logarithmic increase in awareness and demand for the trial, driven by advocacy groups and physician interest [47][48] - **Financial Position**: The company has over $350 million in cash, with Biogen covering 30% of development costs, ensuring strong financial backing for ongoing and future projects [60][61] - **Future Prospects**: Plans to expand research into other haploinsufficient diseases, such as ADOA and Syngap one, indicating a broader pipeline beyond Dravet syndrome [62][61] Conclusion Stoke Therapeutics is positioned strongly in the biotech sector with a promising treatment for Dravet syndrome, backed by robust clinical data and financial resources. The ongoing Phase 3 trial and potential FDA discussions could lead to significant advancements in treatment options for patients with this severe condition.

Summary of Stoke Therapeutics Conference Call Company Overview - **Company**: Stoke Therapeutics - **Focus**: Development of treatments for Dravet syndrome, a genetic condition affecting neurodevelopment and seizures [1][2][3] Key Points Leadership Transition - Ian Smith has taken on the role of Interim CEO, having been with the company for approximately two and a half years [2][3] - Smith's background includes significant experience in biotech, particularly with Vertex Pharmaceuticals, where he worked on cystic fibrosis treatments [3] Disease Focus: Dravet Syndrome - Dravet syndrome affects approximately 40,000 individuals across major global regions, with an incidence of about 1 in 15,000 to 16,000 births [6][7] - The disease is characterized by severe seizures and significant neurodevelopmental delays, with many children not developing beyond the cognitive level of a two-year-old [8][9] Treatment Landscape - Current treatments primarily focus on seizure management, with no existing therapies addressing neurodevelopmental aspects [10][12] - Stoke's drug, zorevunersen, aims to modify the disease by restoring functional protein levels in the brain, potentially improving both seizure control and neurodevelopment [10][12] Clinical Data and Efficacy - In clinical studies, zorevunersen has shown a median seizure reduction of 85% when administered at a 70 mg dose, on top of existing anti-seizure medications [13][14] - The drug has demonstrated durability in seizure reduction over a four-year follow-up period, which is critical given the high mortality rate (20%) associated with Dravet syndrome [15][17] - Improvements in neurodevelopmental outcomes have been observed, with patients scoring significantly higher on the Vineland-3 scale compared to baseline [29][31] Phase 3 Study: AMPRO - The AMPRO study is a 52-week trial designed to evaluate the efficacy of zorevunersen, with a primary endpoint focused on seizure reduction [36][37] - The study aims to enroll 170 patients and is currently progressing well, with over 150 patients already in pre-screening [37] Regulatory Considerations - Stoke Therapeutics has received breakthrough therapy designation from the FDA, which may facilitate a faster path to market [39][41] - A meeting with the FDA is anticipated to discuss expedited filing options based on the existing safety and efficacy data [41] Market Potential and Value Proposition - The potential for zorevunersen to be a high-value treatment is emphasized, particularly if it can demonstrate significant improvements in both seizure control and neurodevelopmental outcomes [44][45] - The company aims to position zorevunersen similarly to other high-impact therapies in the market, such as those for cystic fibrosis [45] Future Outlook - The company is focused on advancing the AMPRO study and hopes to bring the treatment closer to patients, with aspirations for regulatory filing in the near future [48] Additional Insights - The emotional and developmental impact of Dravet syndrome on patients and families is highlighted, underscoring the importance of effective treatment options [9][10] - The potential for zorevunersen to change the trajectory of neurodevelopment in affected children is a key motivating factor for the company's mission [35][48]

Core Insights - Zorevunersen shows potential as the first disease-modifying treatment for Dravet syndrome, with significant reductions in seizure frequency and improvements in cognitive and behavioral outcomes over three years of treatment [2][3][12] Efficacy Results - In Phase 1/2a studies, patients receiving initial doses of 70 mg of zorevunersen experienced a median seizure reduction of 84.8% and an increase of eight seizure-free days per 28 days at three months post-treatment [4] - In open-label extension studies, 94% of eligible patients continued treatment, with 77% remaining after three years, sustaining reductions in major motor seizure frequency [5] - Improvements in quality of life were noted, with an 18-point increase in EuroQol Visual Analog Scale (EQ-VAS) scores over three years [6] Safety Profile - Zorevunersen was generally well tolerated, with treatment-emergent adverse events (TEAEs) reported in 30% of patients in Phase 1/2a studies and 53% in open-label extension studies [7] - The most common TEAE was elevated cerebrospinal fluid (CSF) protein levels, occurring in 14% of patients in Phase 1/2a and 44% in open-label extension studies [7] Study Design - The Phase 1/2a studies involved 81 patients aged 2 to 18 with refractory Dravet syndrome, focusing on safety, pharmacokinetics, and seizure frequency [9] - The ongoing EMPEROR Phase 3 study aims to further evaluate zorevunersen's efficacy and safety in a larger population [13] Company Background - Biogen and Stoke Therapeutics are collaborating on the development of zorevunersen, which has received orphan drug designation and Breakthrough Therapy Designation from the FDA [12][14] - Stoke Therapeutics focuses on restoring protein expression through RNA medicine, with zorevunersen being their first investigational product [16]

Core Insights - Biogen Inc. and Stoke Therapeutics announced new clinical data for zorevunersen, an investigational treatment for Dravet syndrome, to be presented at the 36th International Epilepsy Congress [1][2] - Zorevunersen is being evaluated in the EMPEROR Phase 3 study, which aims to demonstrate its potential as a disease-modifying therapy [1][8] Company Overview - Biogen is a leading biotechnology company focused on innovative science to deliver new medicines and create shareholder value [9] - Stoke Therapeutics specializes in RNA medicine and is developing zorevunersen to restore protein expression in patients with Dravet syndrome [11] Clinical Data and Study Details - New clinical data show substantial and durable reductions in seizures and improvements in cognition and behavior for patients receiving zorevunersen alongside standard anti-seizure medications [2][4] - The EMPEROR Phase 3 study is a global, double-blind, sham-controlled trial evaluating zorevunersen's efficacy and safety in children with Dravet syndrome [8] Presentation Information - Presentations at the IEC will include data on zorevunersen's potential as a disease-modifying therapy and improvements in seizure-free days and quality of life [2][4] - Key presentations will be made by leading experts in the field, highlighting the significance of zorevunersen in treating Dravet syndrome [5][2]

Core Viewpoint - Purcell & Lefkowitz LLP is investigating Stoke Therapeutics, Inc. to determine if the company's directors breached their fiduciary duties related to recent corporate actions [1]. Group 1 - The investigation is on behalf of Stoke Therapeutics's shareholders [1]. - The law firm specializes in representing shareholders affected by securities fraud and breaches of fiduciary duty [3]. - Shareholders interested in their rights and options can contact the firm for more information [2].

Summary of Stokes Therapeutics Conference Call Company Overview - **Company**: Stokes Therapeutics - **Industry**: Biotechnology, specifically focusing on genetic diseases such as Dravet syndrome and ADOA (autosomal dominant optic atrophy) [5][51] Key Points and Arguments 1. **Dravet Syndrome Treatment**: - Stokes Therapeutics is developing a disease-modifying medicine for Dravet syndrome, showing significant reductions in seizures over three to four years of treatment [5][6] - The medicine also demonstrates cognitive and behavioral benefits, improving neurodevelopment in affected children [6][7] - The mechanism involves upregulating Nav 1.1 in the brain, addressing the root cause of Dravet syndrome [7] 2. **Phase III Program**: - The company has commenced a Phase III clinical trial with 150 patients already in pre-screening, aiming for full recruitment by the second half of next year [9][13][36] - The trial is designed to measure seizure reductions and cognitive benefits over a 52-week period [36][42] 3. **FDA Interaction and Breakthrough Designation**: - The FDA granted breakthrough designation for the treatment, acknowledging its potential to address both seizures and cognitive challenges in Dravet syndrome [11] - Stokes plans to discuss data with the FDA in the second half of the year to expedite patient access [12][21] 4. **Financial Position**: - The company ended the quarter with $355 million in capital and is fully funded through mid-2028, covering the Phase III trial and beyond [8][9] 5. **Partnership with Biogen**: - Stokes has a collaboration with Biogen, which funds 30% of the Dravet development and provides infrastructure for faster patient access outside North America [25][27] - The partnership aims to leverage Biogen's existing market presence to expedite the delivery of the medicine globally [27][30] 6. **ADOA Program**: - Stokes is initiating a clinical program for ADOA, focusing on improving vision by upregulating OPA1 and enhancing mitochondrial function [8][51] - Preclinical data from nonhuman primates showed improved vision, prompting the move into clinical trials [52][53] Additional Important Content - **Clinical Data and Biomarkers**: - The company does not have a measurable biomarker for NAV1.1, which complicates the pathway for accelerated approval [19][20] - Clinical data shows significant improvements in Vineland scores, particularly in expressive and receptive communication, which are critical for assessing treatment efficacy [45][46] - **Safety Profile**: - Over 800 doses have been administered with no significant clinical manifestations of elevated cerebrospinal fluid levels observed [50][51] - **Regulatory Landscape**: - The company remains optimistic about regulatory flexibility from the FDA, especially for rare pediatric diseases with significant unmet needs [14][15] - **Global Market Strategy**: - The partnership with Biogen allows Stokes to access over 70 markets globally, enhancing their ability to deliver the treatment quickly post-approval [27][30] This summary encapsulates the critical insights from the conference call, highlighting the company's strategic direction, clinical advancements, and market positioning.

Financial Data and Key Metrics Changes - The company ended the second quarter with $355 million in cash, cash equivalents, and marketable securities, expected to fund operations through mid-2028 [42] - Total revenue for the quarter was $13.8 million, driven by collaborations with ACADIA and Biogen, with expectations for revenue from Biogen to increase [43] - The net loss for the quarter was $23.5 million, or $0.40 per share, slightly improved from the prior year despite a $6.9 million year-over-year increase in operating expenses [43] Business Line Data and Key Metrics Changes - The Phase III EMPORER study for Dravet syndrome is underway, with the first patient dosed and strong enrollment anticipated due to high awareness and urgent patient need [5][21] - The company has advanced STK002 into Phase I clinical development for autosomal dominant optic atrophy (ADOA), indicating a growing pipeline [7][35] Market Data and Key Metrics Changes - The collaboration with Biogen enhances the company's ability to deliver zurivanersen globally and strengthens its balance sheet [8] - The company estimates approximately 13,000 patients currently living with ADOA across key geographies, indicating a significant market opportunity [38] Company Strategy and Development Direction - The key priority remains the development of zurivanersen for Dravet syndrome, aiming to deliver a disease-modifying medicine [4] - The company is focused on establishing internal capabilities and enhancing leadership to support growth and value creation [9] - The strategic collaboration with Biogen is expected to provide global expertise in commercializing high-value disease-modifying medicines [8] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term potential of zurivanersen to modify the course of Dravet syndrome, supported by positive data from ongoing studies [7][31] - The company is committed to advancing zurivanersen to patients as quickly as possible, leveraging breakthrough therapy designation from the FDA [50][52] Other Important Information - The company has seen a favorable safety profile for zurivanersen, with no clinical manifestations associated with elevated CSF protein levels observed in patients [32][94] - The Vineland-three assessment is being used to measure changes in cognition and behavior in patients with Dravet syndrome, with significant improvements noted over time [28][30] Q&A Session Summary Question: Can you help us understand the potential for accelerated approval for zurivanersen? - Management confirmed that zurivanersen has breakthrough designation and they are collecting further data to discuss with the FDA in the second half of the year [50][52] Question: What is the magnitude of cognition and behavior improvements in the Vineland-three data? - Management noted that changes in cognition and behavior are clinically meaningful, with caregivers identifying even small improvements as significant [62][63] Question: Can you explain the data used to inform the powering assumptions for the Phase III EMPOR study? - Management indicated that data from previous studies and natural history studies were used to inform the powering assumptions, ensuring robust statistical significance [70][72] Question: Are there trends in seizure reduction and neurodevelopmental benefits among different age groups? - Management acknowledged variability in responses but emphasized the importance of treating younger patients to potentially change the course of their development [86][87] Question: Can you discuss the higher incidence of CSF protein elevations in the OLE study? - Management clarified that elevated CSF protein levels are a laboratory finding and have not been associated with clinical effects, supporting the safety profile for moving into Phase III [92][94] Question: What prompted the decision to explore STK-two in ADOA? - Management explained that a thorough evaluation of the opportunity in ADOA, including promising nonhuman primate data, led to the decision to pursue clinical studies [98][100]

Zorevunersen for Dravet Syndrome - Stoke Therapeutics is developing zorevunersen as a potential disease-modifying medicine for Dravet syndrome [16] - Initial 70mg doses of zorevunersen demonstrated substantial and sustained reductions in convulsive seizure frequency [18] - Ongoing zorevunersen treatment showed substantial and durable reductions in major motor seizure frequency in OLE studies [21] - 36-month OLE data shows continuing improvements in cognition and behavior with zorevunersen treatment [56] - The EMPEROR Phase 3 study is designed to assess disease modification in Dravet syndrome with a dosing regimen of 2x70mg followed by 2x45mg over a 52-week treatment period [31, 32] - The EMPEROR Phase 3 study has dosed its first patient in August 2025 and anticipates data in 2H 2027, randomizing approximately 170 patients across ~70 global sites [35] STK-002 for Autosomal Dominant Optic Atrophy (ADOA) - Approximately 65-90% of ADOA cases are caused by mutations in one allele of the OPA1 gene, leading to haploinsufficiency [64] - Up to 46% of ADOA patients are registered as legally blind [65] - Preclinical findings support further development of STK-002, showing increased OPA1 protein and ATP-linked respiration [70] - A Phase 1 study of STK-002 in ADOA patients is underway in the UK, involving 21 patients aged ≥18 to <55 years receiving single ascending doses (0.1, 0.3, 0.5, & 0.7 mg/eye) [76]