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Yahoo
3 days ago
- Business
- Yahoo
Stoke Therapeutics Reports Second Quarter 2025 Financial Results and Provides Business Updates
– First patient dosed in the Phase 3 EMPEROR study of zorevunersen in patients with Dravet syndrome – – New 3-year zorevunersen OLE data provide additional support for disease modification: continuing and durable reductions in seizures and improvements in cognition and behavior and generally well tolerated – – Phase 1 study of STK-002 initiated in patients with Autosomal Dominant Optic Atrophy (ADOA), the most common inherited optic nerve disorder – – As of June 30, 2025, the Company had $355.0 million in cash, cash equivalents, and marketable securities, anticipated to fund operations to mid-2028 and into launch readiness – – Webcast and conference call for analysts and investors at 4:30PM Eastern Time today – BEDFORD, Mass., August 12, 2025--(BUSINESS WIRE)--Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. The Company today reported financial results for the second quarter ended June 30, 2025 and provided business updates. "This quarter was defined by strong execution that is driving momentum across our business," said Ian F. Smith, Interim Chief Executive Officer and Director of Stoke Therapeutics. "Our Phase 1/2 and open-label extension studies have provided a strong foundational understanding of zorevunersen and support the EMPEROR Phase 3 registrational study design. We continue to generate long-term data that are helping us appreciate the disease modifying potential of zorevunersen. At the same time, we see growing awareness of Dravet syndrome as a severe neurodevelopmental disease, which is bringing attention to our work and a high degree of interest in zorevunersen." Smith continued, "Beyond Dravet, we have initiated clinical development in a second disease area, advancing STK-002 into a Phase 1 study in patients with Autosomal Dominant Optic Atrophy. Without any treatments approved for ADOA, patients are at risk of progressive loss of sight caused primarily by insufficient OPA1 protein levels. Our pre-clinical data support the potential for STK-002 to restore naturally-occurring protein expression to maintain or improve vision in these patients. We look forward to continuing to expand our approach into new disease areas where we believe we can deliver first-in-class, disease modifying medicines for severe genetic diseases." Recent Program Highlights Yesterday, the Company announced that the first patient has been dosed in the global Phase 3 EMPEROR study of zorevunersen for the treatment of Dravet syndrome. Sites have been initiated in the U.S., UK, Japan and are planned for Europe. Today, the Company announced new positive findings from the long-term open-label extension (OLE) studies of zorevunersen in children and adolescents with Dravet syndrome. Substantial and durable reductions in convulsive seizure frequency on top of standard-of-care medicines were observed through three years of zorevunersen treatment. The data also demonstrate continued improvements in cognition and behavior during the 3-year OLE period beyond the initial 9 months of treatment in the Phase 1/2 studies. Today, the Company announced the Phase 1 study (OSPREY) of STK-002 in patients with Autosomal Dominant Optic Atrophy (ADOA) is now underway. In July, the Company presented data at the European Paediatric Neurology Society (EPNS) Congress from an analysis that evaluated the potential effects on cognition and behavior of a dosing regimen similar to the one now being evaluated in Phase 3. (For full details, see the press release). Upcoming Anticipated Milestones The Company plans to present additional data from the zorevunersen clinical development program at upcoming medical congresses in 2025. Lead optimization is underway to identify a clinical candidate for the treatment of SYNGAP-1 in 2026. SYNGAP-1 is a severe and rare genetic neurodevelopmental disease. Second Quarter 2025 Financial Results As of June 30, 2025, the Company had $355.0 million in cash, cash equivalents, and marketable securities, anticipated to fund operations to mid-2028. Revenue recognized for upfront license fees and services provided from the License and Collaboration Agreement with Acadia Pharmaceuticals for the three months ended June 30, 2025 was $10.6 million, compared to $4.8 million, for the same period in 2024. Revenue recognized from the License and Collaboration Agreement with Biogen for the three months ended June 30, 2025 was $3.2 million. There was no revenue for the same period in 2024. Net loss for the three months ended June 30, 2025 was $23.5 million, or $0.40 per share, compared to a net loss of $25.7 million, or $0.46 per share for the same period in 2024. Research and development expenses for the three months ended June 30, 2025 were $25.9 million, compared to $21.1 million for the same period in 2024. General and administrative expenses for the three months ended June 30, 2025 were $15.3 million, compared to $13.0 million for the same period in 2024. Year-to-Date 2025 Financial Results Revenue recognized for upfront license fees and services provided from the License and Collaboration Agreement with Acadia Pharmaceuticals for the six months ended June 30, 2025 was $16.8 million, compared to $9.0 million for the same period in 2024. Revenue recognized from the License and Collaboration Agreement with Biogen for the six months ended June 30, 2025 was $155.6 million. There was no revenue for the same period in 2024. Net income for the six months ended June 30, 2025 was $89.4 million, or $1.50 per diluted share, compared to a net loss of $52.1 million, or $1.02 per share, for the same period in 2024. Research and development expenses for the six months ended June 30, 2025 were $58.5 million, compared to $43.5 million for the same period in 2024. General and administrative expenses for the six months ended June 30, 2025 were $29.9 million, compared to $23.3 million for the same period in 2024. The increase in operating expenses for the three and six month periods ending June 30, 2025 over the same periods in 2024 primarily relate to increases in costs associated with an increase in personnel and launch readiness expense. Stoke Webcast and Conference Call for Analysts and InvestorsStoke management will host a webcast and conference call for analysts and investors on Tuesday, August 12, 2025, at 4:30pm Eastern Time. The webcast will be available on the Investors & News section of Stoke's website at Research analysts who plan to join the call and participate in the Q&A session may register here to receive the dial-in details and a unique PIN. All other participants are invited to access the listen-only webcast by clicking here. A replay of the webcast will be archived and available for at least 90 days following the event. About Dravet SyndromeDravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. More than 90 percent of patients continue to experience seizures despite treatment with the best available anti-seizure medicines. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan.1 About ZorevunersenZorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights. About Autosomal Dominant Optic Atrophy (ADOA)Autosomal dominant optic atrophy (ADOA) is the most common inherited optic nerve disorder. It is a rare disease that causes progressive and irreversible vision loss in both eyes starting in the first decade of life. Severity can vary and the rate of vision loss can be difficult to predict. Roughly half of people with ADOA fail driving standards and up to 46% are registered as legally blind. More than 400 different OPA1 mutations have been reported in people diagnosed with ADOA. ADOA affects approximately one in 30,000 people globally with a higher incidence in Denmark of one in 10,000 due to a founder effect. Currently there is no approved treatment for people living with ADOA. About STK-002STK-002 is a proprietary antisense oligonucleotide (ASO) in clinical development for the treatment of Autosomal Dominant Optic Atrophy (ADOA). ADOA causes progressive and irreversible vision loss in both eyes starting in the first decade of life. Stoke believes that STK-002 has the potential to be the first disease-modifying therapy for people living with ADOA. An estimated 65% to 90% of cases are caused by mutations in the OPA1 gene, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein expression and disease manifestation. STK-002 is designed to upregulate OPA1 protein expression by leveraging the non-mutant (wild-type) copy of the OPA1 gene to restore OPA1 protein expression with the aim to maintain or improve vision in patients with ADOA. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-002. STK-002 has been granted orphan drug designation by the FDA as a potential new treatment for ADOA. A Phase 1 study (OSPREY) of STK-002 in patients with ADOA is now underway. About Stoke TherapeuticsStoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke's proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the Company's quarterly results and cash runway; its future operating results and current or future financial position and liquidity; the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all; the design, timing and results of clinical studies, data readouts, regulatory decisions and other presentations for zorevunersen and STK-002; the ability of STK-002 to treat the underlying causes of Autosomal Dominant Optic Atrophy (ADOA) and maintain or improve vision; our expectations, plans, aspirations and goals, including those related to the potential of zorevunersen and our collaborations with Biogen and Acadia. Statements including words such as "anticipate," "expect," "plan," "will," or "may" and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause the Company's results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: the Company's ability to advance, obtain regulatory approval and ultimately commercialize its product candidates; that if Biogen were to breach or terminate the collaboration, the Company would not obtain the anticipated financial or other benefits; the possibility that the Company and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; the Company's ability to protect its intellectual property; the Company's ability to fund development activities and achieve development goals through mid-2028; and the other risks and uncertainties described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the Company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. Reference: Based on Stoke Therapeutics' preliminary estimates, which scaled annual incidence to prevalence using country-specific live birth rates over the past 85 years and adjusted for Dravet-specific mortality. The estimate is based on incidence rates published by Wu et al., Pediatrics, 2015. Financial Tables Follow Stoke Therapeutics, Inc. and subsidiary Consolidated balance sheets (in thousands, except share and per share amounts) June 30, December 31, 2025 2024 Assets Current assets: Cash and cash equivalents $ 101,472 $ 127,983 Marketable securities - current 146,236 88,916 Prepaid expenses 13,694 11,117 Restricted cash - current 75 75 Interest receivable 1,622 700 Other current assets 6,871 3,965 Total current assets $ 269,970 $ 232,756 Marketable securities - long-term 107,256 29,824 Restricted cash - long-term 721 721 Operating lease right-of-use assets 3,218 4,345 Property and equipment, net 3,343 3,909 Total assets $ 384,508 $ 271,555 Liabilities and stockholders' equity Current liabilities: Accounts payable $ 4,313 $ 2,498 Accrued and other current liabilities 25,616 18,567 Deferred revenue - current portion 8,749 18,991 Total current liabilities $ 38,678 $ 40,056 Deferred revenue - net of current portion 9,632 — Other long term liabilities 1,255 2,478 Total long term liabilities 10,887 2,478 Total liabilities $ 49,565 $ 42,534 Stockholders' equity Common stock, par value of $0.0001 per share; 300,000,000 shares authorized, 54,723,455 and 54,032,826 shares issued and outstanding as of June 30, 2025 and December 31, 2024, respectively 5 5 Additional paid-in capital 736,276 719,997 Accumulated other comprehensive income (loss) 96 (151 ) Accumulated deficit (401,434 ) (490,830 ) Total stockholders' equity $ 334,943 $ 229,021 Total liabilities and stockholders' equity $ 384,508 $ 271,555 Stoke Therapeutics, Inc. and subsidiary Consolidated statements of operations and comprehensive income (loss) (in thousands, except share and per share amounts) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024 Revenue $ 13,817 $ 4,831 $ 172,386 $ 9,048 Operating expenses: Research and development 25,855 21,136 58,531 43,504 General and administrative 15,262 13,037 29,915 23,258 Total operating expenses 41,117 34,173 88,446 66,762 Income (loss) from operations (27,300 ) (29,342 ) 83,940 (57,714 ) Other income (expense): Interest income (expense), net 3,789 3,695 6,678 6,121 Other income (expense), net 28 (48 ) 57 (476 ) Total other income (expense) 3,817 3,647 6,735 5,645 Income (loss) before income taxes $ (23,483 ) $ (25,695 ) $ 90,675 $ (52,069 ) Provision for income taxes — — 1,278 — Net income (loss) $ (23,483 ) $ (25,695 ) $ 89,397 $ (52,069 ) Net income (loss) per share: Basic $ (0.40 ) $ (0.46 ) $ 1.54 $ (1.02 ) Diluted (0.40 ) (0.46 ) $ 1.50 (1.02 ) Weighted-average common shares outstanding: Basic 58,353,855 55,765,948 $ 58,109,622 51,288,222 Diluted 58,353,855 55,765,948 $ 59,681,472 51,288,222 Comprehensive income (loss): Net income (loss) $ (23,483 ) $ (25,695 ) $ 89,397 $ (52,069 ) Other comprehensive gain (loss): Unrealized gain (loss) on marketable securities 200 (15 ) 247 9 Total other comprehensive gain (loss) $ 200 $ (15 ) $ 247 $ 9 Comprehensive income (loss) $ (23,283 ) $ (25,710 ) $ 89,644 $ (52,060 ) View source version on Contacts Stoke Media & Investor Contacts: Dawn KalmarChief Communications Officerdkalmar@ 781-303-8302Doug SnowDirector, Communications & Investor RelationsIR@ 508-642-6485 Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
3 days ago
- Business
- Business Wire
Stoke Therapeutics Reports Second Quarter 2025 Financial Results and Provides Business Updates
BEDFORD, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. The Company today reported financial results for the second quarter ended June 30, 2025 and provided business updates. 'This quarter was defined by strong execution that is driving momentum across our business,' said Ian F. Smith, Interim Chief Executive Officer and Director of Stoke Therapeutics. 'Our Phase 1/2 and open-label extension studies have provided a strong foundational understanding of zorevunersen and support the EMPEROR Phase 3 registrational study design. We continue to generate long-term data that are helping us appreciate the disease modifying potential of zorevunersen. At the same time, we see growing awareness of Dravet syndrome as a severe neurodevelopmental disease, which is bringing attention to our work and a high degree of interest in zorevunersen.' Smith continued, 'Beyond Dravet, we have initiated clinical development in a second disease area, advancing STK-002 into a Phase 1 study in patients with Autosomal Dominant Optic Atrophy. Without any treatments approved for ADOA, patients are at risk of progressive loss of sight caused primarily by insufficient OPA1 protein levels. Our pre-clinical data support the potential for STK-002 to restore naturally-occurring protein expression to maintain or improve vision in these patients. We look forward to continuing to expand our approach into new disease areas where we believe we can deliver first-in-class, disease modifying medicines for severe genetic diseases.' Recent Program Highlights Yesterday, the Company announced that the first patient has been dosed in the global Phase 3 EMPEROR study of zorevunersen for the treatment of Dravet syndrome. Sites have been initiated in the U.S., UK, Japan and are planned for Europe. Today, the Company announced new positive findings from the long-term open-label extension (OLE) studies of zorevunersen in children and adolescents with Dravet syndrome. Substantial and durable reductions in convulsive seizure frequency on top of standard-of-care medicines were observed through three years of zorevunersen treatment. The data also demonstrate continued improvements in cognition and behavior during the 3-year OLE period beyond the initial 9 months of treatment in the Phase 1/2 studies. Today, the Company announced the Phase 1 study (OSPREY) of STK-002 in patients with Autosomal Dominant Optic Atrophy (ADOA) is now underway. In July, the Company presented data at the European Paediatric Neurology Society (EPNS) Congress from an analysis that evaluated the potential effects on cognition and behavior of a dosing regimen similar to the one now being evaluated in Phase 3. (For full details, see the press release). Upcoming Anticipated Milestones The Company plans to present additional data from the zorevunersen clinical development program at upcoming medical congresses in 2025. Lead optimization is underway to identify a clinical candidate for the treatment of SYNGAP-1 in 2026. SYNGAP-1 is a severe and rare genetic neurodevelopmental disease. Second Quarter 2025 Financial Results As of June 30, 2025, the Company had $355.0 million in cash, cash equivalents, and marketable securities, anticipated to fund operations to mid-2028. Revenue recognized for upfront license fees and services provided from the License and Collaboration Agreement with Acadia Pharmaceuticals for the three months ended June 30, 2025 was $10.6 million, compared to $4.8 million, for the same period in 2024. Revenue recognized from the License and Collaboration Agreement with Biogen for the three months ended June 30, 2025 was $3.2 million. There was no revenue for the same period in 2024. Net loss for the three months ended June 30, 2025 was $23.5 million, or $0.40 per share, compared to a net loss of $25.7 million, or $0.46 per share for the same period in 2024. Research and development expenses for the three months ended June 30, 2025 were $25.9 million, compared to $21.1 million for the same period in 2024. General and administrative expenses for the three months ended June 30, 2025 were $15.3 million, compared to $13.0 million for the same period in 2024. Year-to-Date 2025 Financial Results Revenue recognized for upfront license fees and services provided from the License and Collaboration Agreement with Acadia Pharmaceuticals for the six months ended June 30, 2025 was $16.8 million, compared to $9.0 million for the same period in 2024. Revenue recognized from the License and Collaboration Agreement with Biogen for the six months ended June 30, 2025 was $155.6 million. There was no revenue for the same period in 2024. Net income for the six months ended June 30, 2025 was $89.4 million, or $1.50 per diluted share, compared to a net loss of $52.1 million, or $1.02 per share, for the same period in 2024. Research and development expenses for the six months ended June 30, 2025 were $58.5 million, compared to $43.5 million for the same period in 2024. General and administrative expenses for the six months ended June 30, 2025 were $29.9 million, compared to $23.3 million for the same period in 2024. The increase in operating expenses for the three and six month periods ending June 30, 2025 over the same periods in 2024 primarily relate to increases in costs associated with an increase in personnel and launch readiness expense. Stoke Webcast and Conference Call for Analysts and Investors Stoke management will host a webcast and conference call for analysts and investors on Tuesday, August 12, 2025, at 4:30pm Eastern Time. The webcast will be available on the Investors & News section of Stoke's website at Research analysts who plan to join the call and participate in the Q&A session may register here to receive the dial-in details and a unique PIN. All other participants are invited to access the listen-only webcast by clicking here. A replay of the webcast will be archived and available for at least 90 days following the event. About Dravet Syndrome Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. More than 90 percent of patients continue to experience seizures despite treatment with the best available anti-seizure medicines. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan. 1 About Zorevunersen Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights. About Autosomal Dominant Optic Atrophy (ADOA) Autosomal dominant optic atrophy (ADOA) is the most common inherited optic nerve disorder. It is a rare disease that causes progressive and irreversible vision loss in both eyes starting in the first decade of life. Severity can vary and the rate of vision loss can be difficult to predict. Roughly half of people with ADOA fail driving standards and up to 46% are registered as legally blind. More than 400 different OPA1 mutations have been reported in people diagnosed with ADOA. ADOA affects approximately one in 30,000 people globally with a higher incidence in Denmark of one in 10,000 due to a founder effect. Currently there is no approved treatment for people living with ADOA. About STK-002 STK-002 is a proprietary antisense oligonucleotide (ASO) in clinical development for the treatment of Autosomal Dominant Optic Atrophy (ADOA). ADOA causes progressive and irreversible vision loss in both eyes starting in the first decade of life. Stoke believes that STK-002 has the potential to be the first disease-modifying therapy for people living with ADOA. An estimated 65% to 90% of cases are caused by mutations in the OPA1 gene, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein expression and disease manifestation. STK-002 is designed to upregulate OPA1 protein expression by leveraging the non-mutant (wild-type) copy of the OPA1 gene to restore OPA1 protein expression with the aim to maintain or improve vision in patients with ADOA. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-002. STK-002 has been granted orphan drug designation by the FDA as a potential new treatment for ADOA. A Phase 1 study (OSPREY) of STK-002 in patients with ADOA is now underway. About Stoke Therapeutics Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke's proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the Company's quarterly results and cash runway; its future operating results and current or future financial position and liquidity; the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all; the design, timing and results of clinical studies, data readouts, regulatory decisions and other presentations for zorevunersen and STK-002; the ability of STK-002 to treat the underlying causes of Autosomal Dominant Optic Atrophy (ADOA) and maintain or improve vision; our expectations, plans, aspirations and goals, including those related to the potential of zorevunersen and our collaborations with Biogen and Acadia. Statements including words such as 'anticipate,' 'expect,' 'plan,' 'will,' or 'may' and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause the Company's results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: the Company's ability to advance, obtain regulatory approval and ultimately commercialize its product candidates; that if Biogen were to breach or terminate the collaboration, the Company would not obtain the anticipated financial or other benefits; the possibility that the Company and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; the Company's ability to protect its intellectual property; the Company's ability to fund development activities and achieve development goals through mid-2028; and the other risks and uncertainties described under the heading 'Risk Factors' in the Company's Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the Company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. Reference: Based on Stoke Therapeutics' preliminary estimates, which scaled annual incidence to prevalence using country-specific live birth rates over the past 85 years and adjusted for Dravet-specific mortality. The estimate is based on incidence rates published by . Financial Tables Follow Stoke Therapeutics, Inc. and subsidiary Consolidated balance sheets (in thousands, except share and per share amounts) June 30, December 31, 2025 2024 Assets Current assets: Cash and cash equivalents $ 101,472 $ 127,983 Marketable securities - current 146,236 88,916 Prepaid expenses 13,694 11,117 Restricted cash - current 75 75 Interest receivable 1,622 700 Other current assets 6,871 3,965 Total current assets $ 269,970 $ 232,756 Marketable securities - long-term 107,256 29,824 Restricted cash - long-term 721 721 Operating lease right-of-use assets 3,218 4,345 Property and equipment, net 3,343 3,909 Total assets $ 384,508 $ 271,555 Liabilities and stockholders' equity Current liabilities: Accounts payable $ 4,313 $ 2,498 Accrued and other current liabilities 25,616 18,567 Deferred revenue - current portion 8,749 18,991 Total current liabilities $ 38,678 $ 40,056 Deferred revenue - net of current portion 9,632 — Other long term liabilities 1,255 2,478 Total long term liabilities 10,887 2,478 Total liabilities $ 49,565 $ 42,534 Stockholders' equity Common stock, par value of $0.0001 per share; 300,000,000 shares authorized, 54,723,455 and 54,032,826 shares issued and outstanding as of June 30, 2025 and December 31, 2024, respectively 5 5 Additional paid-in capital 736,276 719,997 Accumulated other comprehensive income (loss) 96 (151 ) Accumulated deficit (401,434 ) (490,830 ) Total stockholders' equity $ 334,943 $ 229,021 Total liabilities and stockholders' equity $ 384,508 $ 271,555 Expand Stoke Therapeutics, Inc. and subsidiary Consolidated statements of operations and comprehensive income (loss) (in thousands, except share and per share amounts) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024 Revenue $ 13,817 $ 4,831 $ 172,386 $ 9,048 Operating expenses: Research and development 25,855 21,136 58,531 43,504 General and administrative 15,262 13,037 29,915 23,258 Total operating expenses 41,117 34,173 88,446 66,762 Income (loss) from operations (27,300 ) (29,342 ) 83,940 (57,714 ) Other income (expense): Interest income (expense), net 3,789 3,695 6,678 6,121 Other income (expense), net 28 (48 ) 57 (476 ) Total other income (expense) 3,817 3,647 6,735 5,645 Income (loss) before income taxes $ (23,483 ) $ (25,695 ) $ 90,675 $ (52,069 ) Provision for income taxes — — 1,278 — Net income (loss) $ (23,483 ) $ (25,695 ) $ 89,397 $ (52,069 ) Net income (loss) per share: Basic $ (0.40 ) $ (0.46 ) $ 1.54 $ (1.02 ) Diluted (0.40 ) (0.46 ) $ 1.50 (1.02 ) Weighted-average common shares outstanding: Basic 58,353,855 55,765,948 $ 58,109,622 51,288,222 Diluted 58,353,855 55,765,948 $ 59,681,472 51,288,222 Comprehensive income (loss): Net income (loss) $ (23,483 ) $ (25,695 ) $ 89,397 $ (52,069 ) Other comprehensive gain (loss): Unrealized gain (loss) on marketable securities 200 (15 ) 247 9 Total other comprehensive gain (loss) $ 200 $ (15 ) $ 247 $ 9 Comprehensive income (loss) $ (23,283 ) $ (25,710 ) $ 89,644 $ (52,060 ) Expand
Business Wire
5 days ago
- Business
- Business Wire
Stoke Therapeutics and Biogen Announce First Patient Dosed in Phase 3 EMPEROR Study of Zorevunersen, a Potential Disease-Modifying Treatment for Dravet Syndrome
BEDFORD, Mass., & CAMBRIDGE, 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, and Biogen Inc. (Nasdaq: BIIB), today announced that the first patient has been dosed in the global Phase 3 EMPEROR study of zorevunersen for the treatment of Dravet syndrome. Zorevunersen, an investigational antisense oligonucleotide, has the potential to be the first disease-modifying treatment for Dravet syndrome. 'Our Phase 1/2 and open-label extension studies have provided a large dataset to support our understanding of zorevunersen and guide the EMPEROR study design, including dosing, duration and selection and powering of the endpoints,' said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. 'Given the severity of this disease and the limitations of current treatments, the substantial and durable reductions in seizures and continuing improvements in cognition and behavior support our belief that zorevunersen may improve outcomes for patients with Dravet syndrome.' 'The initiation of the EMPEROR study is a critical milestone in zorevunersen's development,' said Katherine Dawson, M.D., Head of the Therapeutics Development Unit at Biogen. 'Despite treatment with available anti-seizure medicines, no approved medications currently address the underlying cognitive and behavioral aspects of this rare, genetic disease. Together with Stoke, we look forward to working in collaboration with the hope of bringing forward zorevunersen as the first disease-modifying treatment option, if approved, for Dravet syndrome.' EMPEROR Pivotal Phase 3 Design Summary Anticipated Enrollment: Patients with Dravet syndrome between the ages of 2 and up to 18 years of age with a confirmed variant in the SCN1A gene not associated with gain of function. Duration: Following an 8-week baseline period, participants will be randomized 1:1 to receive either zorevunersen or sham for a 52-week treatment period. Treatment Arms: Patients in the active treatment arm will receive two 70mg loading doses (Day 1 and Week 8) followed by two 45mg maintenance doses (Week 24 and Week 40). All patients will continue to receive standard of care medicines throughout the study. Primary Endpoint: Change in major motor seizure frequency measured at Week 28. Key Secondary Endpoints: Change in major motor seizure frequency measured at Week 52. Change in behavior and cognition as measured by the Vineland-3 subdomains, including expressive communication, receptive communication, interpersonal relationships, coping skills and personal skills measured at Week 52. Eligible participants will be offered ongoing treatment with zorevunersen as part of an open-label extension (OLE) study. 'Dravet syndrome is one of the most well studied genetic epilepsies so we know the significant and life-altering effects it can have on patients and their caregivers,' said Joseph Sullivan, M.D., FAES, principal investigator of the study and Professor of Neurology and Pediatrics and Director of the Pediatric Epilepsy Center of Excellence at the University of California San Francisco. 'Providing additional relief from seizures remains an important clinical outcome, but the potential to address the underlying genetic cause to also address neurodevelopmental symptoms signals a fundamentally new way of treating the disease. The urgent need for treatments is evident in the high degree of interest in the EMPEROR study.' The EMPEROR clinical trial has initiated in the United States, United Kingdom, Japan and is planned for Europe. For more information on the EMPEROR study, please visit and About Stoke Therapeutics Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for the Company's proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit About Biogen Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients' lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at Follow us on social media - Facebook, LinkedIn, X, YouTube. About Dravet Syndrome Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. More than 90 percent of patients continue to experience seizures despite treatment with the best available anti-seizure medicines. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan. 1 About Zorevunersen Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights. About the EMPEROR Study The EMPEROR Phase 3 Study (NCT06872125) is a global, double-blind, sham-controlled study evaluating the efficacy, safety and tolerability of zorevunersen in children ages 2 to <18 with Dravet syndrome with a confirmed variant in the SCN1A gene not associated with gain of function. The trial is expected to enroll participants across the United States, Japan, United Kingdom and European Union, with participants being randomized 1:1 to receive either zorevunersen via intrathecal administration or a sham comparator for a 52-week treatment period following an 8-week baseline period. Following the completion of the study, eligible participants will be offered ongoing treatment with zorevunersen as part of an OLE study. The primary endpoint of the study is percent change from baseline in major motor seizure frequency at week 28 in patients receiving zorevunersen as compared to sham. The key secondary endpoints are the durability of effect on major motor seizure frequency and improvements in behavior and cognition as measured by Vineland-3 subdomains, including expressive communication, receptive communication, interpersonal relationships, coping skills and personal skills. Additional endpoints include safety, Clinician Global Impression of Change (CGI-C), Caregiver Global Impression of Change (CaGI-C), and the Bayley Scales of Infant Development (BSID-IV). For more information, visit Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all; the design, timing and results of the Phase 3 EMPEROR study; and the expectations and potential benefits of Stoke's collaboration with Biogen. Statements including words such as 'anticipate,' 'could,' 'expect,' 'plan,' 'will,' or 'may' and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause Stoke's results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: Stoke's ability to advance, obtain regulatory approval and ultimately commercialize its product candidates; that if Stoke's collaborators were to breach or terminate their agreements, it would not obtain the anticipated financial or other benefits; the possibility that Stoke and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; Stoke's ability to protect its intellectual property; Stoke's ability to fund development activities and achieve development goals through mid-2028; and the other risks and uncertainties described under the heading 'Risk Factors' in Stoke's Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and Stoke undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. Biogen Safe Harbor This news release contains forward-looking statements, including relating to the potential, benefits, safety and efficacy of Zorevunersen; the potential of Biogen's commercial business and pipeline programs, including Zorevunersen; the anticipated benefits and potential of Biogen's collaboration arrangement with Stoke Therapeutics; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by such words as 'aim,' 'anticipate,' 'assume,' 'believe,' 'contemplate,' 'continue,' 'could,' 'estimate,' 'expect,' 'forecast,' 'goal,' 'guidance,' 'hope,' 'intend,' 'may,' 'objective,' 'plan,' 'possible,' 'potential,' 'predict,' 'project,' 'prospect,' 'should,' 'target,' 'will,' 'would,' and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. These forward-looking statements are based on management's current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward-looking statements will be realized in whole or in part. We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to be materially different from those stated or implied in this document, including, among others, uncertainty of long-term success in developing, licensing, or acquiring other product candidates or additional indications for existing products; expectations, plans and prospects relating to product approvals, approvals of additional indications for our existing products, sales, pricing, growth, reimbursement and launch of our marketed and pipeline products; our ability to effectively implement our corporate strategy; the successful execution of our strategic and growth initiatives, including acquisitions; the risk that positive results in a clinical trial may not be replicated in subsequent or confirmatory trials or success in early stage clinical trials may not be predictive of results in later stage or large scale clinical trials or trials in other potential indications; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events, restrictions on use with our products, or product liability claims; and any other risks and uncertainties that are described in other reports we have filed with the U.S. Securities and Exchange Commission. These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, and in our subsequent reports on Form 10-Q and Form 10-K, in each case including in the sections thereof captioned 'Note Regarding Forward-Looking Statements' and 'Item 1A. Risk Factors,' and in our subsequent reports on Form 8-K. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise. Reference: Based on Stoke Therapeutics' preliminary estimates, which scaled annual incidence to prevalence using country-specific live birth rates over the past 85 years and adjusted for Dravet-specific mortality. The estimate is based on incidence rates published by .
Business Wire
10-07-2025
- Health
- Business Wire
Stoke Therapeutics and Biogen Announce Presentation of Data from Studies of Zorevunersen, an Investigational Medicine for Dravet syndrome, at the 16th European Paediatric Neurology Society (EPNS) Congress
BEDFORD, Mass., & CAMBRIDGE, 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, and Biogen Inc. (Nasdaq: BIIB) today announced the presentation of data from an analysis that informed the design of the Phase 3 EMPEROR study and evaluated the potential effects of the Phase 3 zorevunersen dosing regimen. The data are complementary to previously reported data from a broader cohort of patients treated with zorevunersen in the Phase 1/2a and open label extension (OLE) studies that showed improvements within the first 9 months and continuing improvements through an additional two years. The new analysis is best aligned with the timing and dosing regimen that will be evaluated in the pivotal Phase 3 EMPEROR study and showed improvements in multiple measures of cognition and behavior at Week 68. The results contrasted with findings from a natural history study in which patients with Dravet syndrome were treated with standard of care medicines. Zorevunersen is in development as a first-in-class potential disease modifying treatment for Dravet syndrome. Results were presented as part of the Epilepsy II session at the 16th European Paediatric Neurology Society (EPNS) Congress. "Dravet syndrome is a complex neurodevelopmental disorder that has significant impacts on patients and their families,' said Dr. Andreas Brunklaus, Consultant Paediatric Neurologist at the Royal Hospital for Children in Glasgow, Honorary Professor at the University of Glasgow, and a zorevunersen study investigator. 'Natural history data shows the limitations of treating this disease with anti-seizure medicines. The zorevunersen data give us early evidence that this new genetically-targeted approach could address the underlying cause of Dravet syndrome, resulting in additional seizure control and offer patients the opportunity to experience improvements in cognition and behavior." Previously presented data from the two Phase 1/2a and OLE studies showed substantial and durable reductions in major motor seizure frequency on top of a background of standard anti-seizure medicines and improvements in multiple measures of cognition and behavior through two years of treatment in the OLE studies. Data indicated responses may be better among patients who were treated with loading doses of 70mg followed by maintenance doses of 45mg. Zorevunersen was generally well-tolerated across these studies. "Effects on behavior and cognition are a key secondary endpoint in our Phase 3 EMPEROR study," said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. 'Feedback from caregivers and clinicians, and analyses like this one, give us insight into which assessments have the greatest potential to demonstrate meaningful effects for patients within the year-long treatment period.' 'Most patients with Dravet syndrome continue to experience seizures despite treatment with the best available anti-seizure medicines, and there are currently no medications approved that address the underlying cognitive and behavioral aspects of the disease,' said Katherine Dawson, M.D., Head of the Therapeutics Development Unit at Biogen. 'We look forward to continuing to work together to advance zorevunersen as a potential first-in-class disease modifying medicine for Dravet syndrome.' Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) that is characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. There are no approved disease-modifying therapies for people living with Dravet syndrome. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the United States (~16,000), United Kingdom, EU-4 (Germany, France, Italy, Spain) and Japan. 1 Description of the Modeled Analysis A mixed-effects model for repeated measures (MMRM) analysis was used to evaluate the potential effects of the Phase 3 zorevunersen dosing regimen on patient cognition and behavior at Week 68. The model was developed using clinical data from patients in the Phase 1/2a ADMIRAL study and the LONGWING OLE study. An analysis of patients who received a total cumulative dose consistent with the Phase 3 EMPEROR regimen of two loading doses of 70mg followed by two maintenance doses of 45mg, showed improvements in cognition and behavior. The analysis was performed to inform the design of the Phase 3 EMPEROR study. Baseline covariates for patients followed in the BUTTERFLY natural history study were matched to the selected ADMIRAL patient population. Improvements in patients treated with zorevunersen contrasted with findings from BUTTERFLY. These data support the selection of five sub-domains of the Vineland-3 Adaptive Behavior Scales, including Receptive Communication, Expressive Communication, Interpersonal Relationships, Coping Skills, and Personal Skills now under evaluation as key secondary endpoints in the Phase 3 EMPEROR study. Details of the presentation are as follows: Title: Zorevunersen demonstrates potential as a disease modifying therapy in patients with Dravet syndrome through durable seizure reduction and improvements in cognition, behavior, and functioning with up to 24 months of maintenance dosing in open-label extension studies Presenter: Andreas Brunklaus, M.D., Consultant Paediatric Neurologist at the Royal Hospital for Children, Glasgow, Honorary Professor at the University of Glasgow Session: Epilepsy II Date/Time: Thursday, July 10, 11:51 AM CEST Location: Saal 5, International Congress Center München, Germany The presentation will be available for download on the Stoke Therapeutics website under the Investors & News tab. About Dravet Syndrome Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. More than 90 percent of patients continue to experience seizures despite treatment with the best available anti-seizure medicines. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan. 1 About Zorevunersen Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights. About Stoke Therapeutics Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke's proprietary approach. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit About Biogen Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients' lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at Follow us on social media - Facebook, LinkedIn, X, YouTube. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all; and the design, timing and results of the Phase 3 EMPEROR study. Statements including words such as 'anticipate,' 'could,' 'expect,' 'plan,' 'will,' or 'may' and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause Stoke's results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: Stoke's ability to advance, obtain regulatory approval and ultimately commercialize its product candidates; that if Stoke's collaborators were to breach or terminate their agreements, it would not obtain the anticipated financial or other benefits; the possibility that Stoke and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; Stoke's ability to protect its intellectual property; Stoke's ability to fund development activities and achieve development goals through mid-2028; and the other risks and uncertainties described under the heading 'Risk Factors' in Stoke's Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and Stoke undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. Biogen Safe Harbor This press release contains forward-looking statements, relating to: our strategy and plans; the potential of, and expectations for, our commercial business and pipeline programs; clinical development programs, clinical trials, and data readouts and presentations; regulatory discussions, submissions, filings, and approvals; the potential benefits, safety, and efficacy of our and our collaboration partners' products and investigational therapies; and actions to improve the risk profile and productivity of R&D pipeline, collaborations, and business development activities. These forward-looking statements may be accompanied by such words as 'aim,' 'anticipate,' 'assume,' 'believe,' 'contemplate,' 'continue,' 'could,' 'estimate,' 'expect,' 'forecast,' 'goal,' 'guidance,' 'hope,' 'intend,' 'may,' 'objective,' 'outlook,' 'plan,' 'possible,' 'potential,' 'predict,' 'project,' 'prospect,' 'should,' 'target,' 'will,' 'would,' and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. This press release includes, among others, forward-looking statements including relating to: the ability of zorevunersen to treat the underlying causes of Dravet syndrome, the design, timing and results of the Phase 3 EMPEROR study and the potential effects of the Phase 3 zorevunersen dosing regimen. These forward-looking statements are based on management's current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward looking statements will be realized in whole or in part. We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to be materially different from those stated or implied in this document. These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q , in each case including in the sections thereof captioned 'Note Regarding Forward-Looking Statements' and 'Item 1A. Risk Factors,' and in our subsequent reports on Form 8-K. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise. Reference: Based on Stoke Therapeutics' preliminary estimates, which scaled annual incidence to prevalence using country-specific live birth rates over the past 85 years and adjusted for Dravet-specific mortality. The estimate is based on incidence rates published by .
Yahoo
18-02-2025
- Business
- Yahoo
Biogen Strengthens Pipeline With Stoke Therapeutic's Dravet Syndrome Candidate Via Multi-Million Dollar Deal
On Tuesday, Biogen Inc. (NASDAQ:BIIB) and Stoke Therapeutics, Inc. (NASDAQ:STOK) collaborated to develop and commercialize zorevunersen for Dravet syndrome in all territories outside the United States, Canada, and Mexico. Zorevunersen is an investigational antisense oligonucleotide (ASO) that targets the SCN1A gene, the underlying cause of most cases of Dravet syndrome. Stoke recently announced plans to initiate a global Phase 3 registrational study of zorevunersen (EMPEROR) following the alignment with regulatory agencies in the United States, Europe, and Japan. Also Read: The study is on track to initiate in the second quarter of 2025, with a pivotal data readout expected in the second half of 2027, which is anticipated to support global regulatory filings. Stoke will continue to lead global development and retain exclusive development and commercialization rights for zorevunersen in the U.S., Canada, and Mexico. Biogen receives exclusive rights to commercialize zorevunersen in the rest of the world. Stoke will receive an upfront payment of $165 million. The parties will share external clinical development costs for zorevunersen (30% Biogen; 70% Stoke). Additionally, Stoke may receive up to $385 million in development and commercial milestone payments. Stoke will also be eligible to receive tiered royalties on potential net sales in the Biogen territory, ranging from low double digits to high teens. Stoke has also granted Biogen an option to license rights outside of the U.S., Canada, and Mexico to certain future follow-on ASO products targeting SCN1A, in exchange for separate milestone, cost sharing, and royalty considerations. 'Additionally, this collaboration provides cash flows, that when combined with Stoke's financial position, support the company through to mid-2028,' said Edward Kaye, CEO of Stoke Therapeutics. Price Action: At last check Tuesday, BIIB was down 0.79% at $136.24, and STOK was down 3.15% at $10.34. Read Next:Up Next: Transform your trading with Benzinga Edge's one-of-a-kind market trade ideas and tools. Click now to access unique insights that can set you ahead in today's competitive market. Get the latest stock analysis from Benzinga? BIOGEN (BIIB): Free Stock Analysis Report This article Biogen Strengthens Pipeline With Stoke Therapeutic's Dravet Syndrome Candidate Via Multi-Million Dollar Deal originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved.
