Stoke Therapeutics Presents Data From Multiple Studies Of Children And Adolescents With Dravet Syndrome At The American Epilepsy Society 2023 Annual Meeting

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology firm devoted to addressing the underlying explanation for extreme illnesses by upregulating protein expression with RNA-based medicines, in the present day introduced highlights from displays of scientific knowledge on the American Epilepsy Society (AES) 2023 Annual Assembly December 1 – 5, in Orlando, Florida. Collectively, these knowledge assist the corporate’s continued progress to develop STK-001 as the primary disease-modifying drugs for the therapy of Dravet syndrome.

“The excellent set of knowledge being introduced at AES are giving us an excellent understanding of how STK-001 works and its potential to deal with not solely seizures, however lots of the non-seizure results of Dravet syndrome,” mentioned Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. “The substantial and sustained reductions in seizure frequency and enhancements in cognition and habits noticed in our STK-001 scientific research give us confidence that we’re addressing the basis explanation for Dravet syndrome. As well as, the correlation between larger STK-001 publicity ranges in mind and reductions in seizure frequency proven in our modeling knowledge gives extra confidence within the scientific advantages noticed amongst sufferers handled with STK-001 at larger doses and for longer durations of time. These findings are in stark distinction to the information from our two-year pure historical past research that present a scarcity of enchancment amongst sufferers who’re taking the very best out there anti-seizure medicines.”

“Dravet syndrome goes far past seizures, and as youngsters develop up, they expertise a fancy array of life-altering challenges, together with developmental delays, motion and steadiness points and delayed language and speech,” mentioned Joseph Sullivan, M.D., FAES, Professor of Neurology and Pediatrics and Director of the Pediatric Epilepsy Heart of Excellence on the College of California San Francisco, and a distinguished researcher in Dravet syndrome. “On common, sufferers enrolled within the BUTTERFLY pure historical past research have been taking 3.5 anti-seizure medicines. Regardless of this, they continued to expertise equally excessive charges of seizure frequency all through the research and fell additional and additional behind their neurotypical friends in points of cognition and habits, together with their potential to speak and use gross motor and tremendous motor expertise. These findings spotlight the vital want for a brand new strategy to treating this illness, one that may enhance the therapy of seizures and transcend that to deal with the debilitating cognitive and behavioral points of this illness.”

Highlights from the Firm’s displays of knowledge on the assembly, embody:

  • BUTTERFLY Pure Historical past Research of Sufferers with Dravet Syndrome Ages 2 to 18: Regardless of therapy with the very best out there anti-seizure medicines, on common, sufferers continued to expertise convulsive seizures over 24 months at comparable frequency to baseline. No statistically vital change from baseline within the majority of Vineland-III measures (a longtime instrument for assessing developmental disabilities) was noticed and the speed of enchancment on a number of scientific measures, together with key domains of the Vineland-III, was considerably under neurotypical friends. Gaps in neurodevelopment continued to widen all through the research amongst sufferers with Dravet syndrome in comparison with their age-matched neurotypical friends.
  • MONARCH & ADMIRAL Interim Analyses: Single and a number of doses of STK-001 as much as 70mg have been typically properly tolerated. The a number of dose 70mg cohort confirmed the best reductions in convulsive seizure frequency, outperforming all decrease dose teams. Sufferers handled with 2 or 3 preliminary doses of 70mg skilled substantial and sustained reductions in convulsive seizure frequency.
  • SWALLOWTAIL & LONGWING Open Label Extension (OLE) research: Roughly 90% of sufferers who accomplished participation in Part 1/2a research of STK-001 enrolled in considered one of these OLE research. A number of doses of STK-001 as much as 45mg given each 4 months have been typically properly tolerated. Along with sturdy reductions in convulsive seizure frequency all through the course of therapy, knowledge indicated substantial enhancements in a number of assessments of cognition and habits over 12 months. These knowledge assist the potential for disease-modification with STK-001.
  • PK Mannequin for STK-001: A relationship between STK-001 mind exposures and convulsive seizure frequency was evaluated primarily based on 72 sufferers handled within the Part 1/2a research (MONARCH and ADMIRAL) and the SWALLOWTAIL OLE research in youngsters and adolescents with Dravet syndrome. The exposure-seizure evaluation demonstrated that larger STK-001 mind publicity results in better reductions in convulsive seizure frequency (R=-0.23, P<0.001).

Particulars of the Firm’s displays will be discovered within the desk under. All displays can be found for obtain on the Stoke Therapeutics web site underneath the Buyers & Information tab.

Title

Presenter

Date

24-Month Evaluation of BUTTERFLY: A Potential, Observational Research to Examine Cognition and Different Non-seizure Comorbidities in Kids & Adolescents with Dravet Syndrome (DS)

Joseph Sullivan, M.D., FAES, Professor of Neurology and Pediatrics and Director of the Pediatric Epilepsy Heart of Excellence on the College of California San Francisco

Poster Quantity: 1.233

Saturday, Dec. 2

12:00 PM EST

Oral Presentation:

Monday, Dec. 4

3:15 PM EST

MONARCH & ADMIRAL: Part 1/2a Research in US & UK Investigating Security and Drug Publicity of STK-001, an Antisense Oligonucleotide (ASO), in Kids & Adolescents with Dravet Syndrome (DS)

Helen Cross, MB ChB, Ph.D., Professor, The Prince of Wales’s Chair of Childhood Epilepsy and Head of the Developmental Neuroscience Programme at College School London Nice Ormond Avenue Institute of Little one Well being, Honorary Guide in Paediatric Neurology, President of the Worldwide League In opposition to Epilepsy

Poster Quantity: 1.276

Saturday, Dec. 2

12:00 PM EST

SWALLOWTAIL & LONGWING: Open-Label Extension (OLE) Research for Kids and Adolescents with Dravet Syndrome (DS) who Beforehand Participated in a Research of Antisense Oligonucleotide (ASO) STK-001

Archana Desurkar M.D., Guide Paediatric Neurologist at Sheffield Kids’s Hospital Nationwide Well being Service Basis Belief

Poster Quantity: 1.279

Saturday, Dec. 2

12:00 PM EST

Utilization of a Pharmacokinetic (PK) Mannequin for STK-001 in Sufferers with Dravet Syndrome (DS) To Assist Number of Dosing Regimens in Clinic

Meena, Ph.D., Senior Vice President of Translational DMPK and Scientific Pharmacology at Stoke Therapeutics

Poster Quantity: 3.110

Monday, Dec. 4

12:00 PM EST

Dravet syndrome is a extreme and progressive genetic epilepsy characterised by frequent, extended and refractory seizures, starting throughout the first 12 months of life. Dravet syndrome is tough to deal with and has a poor long-term prognosis. Problems of the illness typically contribute to a poor high quality of life for sufferers and their caregivers. The results of the illness transcend seizures and sometimes embody mental incapacity, developmental delays, motion and steadiness points, language and speech disturbances, progress defects, sleep abnormalities, disruptions of the autonomic nervous system and temper issues. The illness is classed as a developmental and epileptic encephalopathy because of the developmental delays and cognitive impairment related to the illness. In contrast with the final epilepsy inhabitants, individuals dwelling with Dravet syndrome have the next danger of sudden sudden dying in epilepsy, or SUDEP. There aren’t any permitted disease-modifying therapies for individuals dwelling with Dravet syndrome. One out of 16,000 infants are born with Dravet syndrome, which isn’t concentrated in a selected geographic space or ethnic group.

STK-001 is an investigational new drugs for the therapy of Dravet syndrome at the moment being evaluated in ongoing scientific trials. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the primary disease-modifying remedy to deal with the genetic explanation for Dravet syndrome. STK-001 is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to revive physiological NaV1.1 ranges, thereby lowering each incidence of seizures and vital non-seizure comorbidities. STK-001 has been granted orphan drug designation by the FDA and the EMA, and uncommon pediatric illness designation by the FDA as a possible new therapy for Dravet syndrome.

The MONARCH research is a Part 1/2a open-label research of youngsters and adolescents ages 2 to 18 who’ve a longtime prognosis of Dravet syndrome and have proof of a genetic mutation within the SCN1A gene. The first aims for the research are to evaluate the protection and tolerability of STK-001, in addition to to find out the pharmacokinetics in plasma and publicity in cerebrospinal fluid. A secondary goal is to evaluate the efficacy as an adjunctive antiepileptic therapy with respect to the proportion change from baseline in convulsive seizure frequency. Stoke additionally intends to measure non-seizure points of the illness, equivalent to high quality of life, as secondary endpoints. Extra details about the MONARCH research will be discovered at https://www.monarchstudy.com/.

Sufferers who participated within the MONARCH research and meet research entry standards are eligible to proceed therapy in SWALLOWTAIL, an open-label extension (OLE) research designed to guage the long-term security and tolerability of repeat doses of STK-001. We anticipate that SWALLOWTAIL can even present worthwhile data on the preliminary results of STK-001 on seizures together with non-seizure points of the illness, equivalent to high quality of life and cognition.

Enrollment and dosing in SWALLOWTAIL are ongoing.

The ADMIRAL research is a Part 1/2a open-label research of youngsters and adolescents ages 2 to <18 who’ve a longtime prognosis of Dravet syndrome and have proof of a genetic mutation within the SCN1A gene. The first aims for the research are to evaluate the protection and tolerability of a number of doses of STK-001, in addition to to find out the pharmacokinetics in plasma and publicity in cerebrospinal fluid. A secondary goal is to evaluate the impact of a number of doses of STK-001 as an adjunctive antiepileptic therapy with respect to the proportion change from baseline in convulsive seizure frequency. Stoke additionally intends to measure non-seizure points of the illness, equivalent to total scientific standing and high quality of life, as secondary endpoints.

Sufferers who participated within the ADMIRAL research and meet research entry standards are eligible to proceed therapy in LONGWING, an open-label extension (OLE) research designed to guage the long-term security and tolerability of repeat doses of STK-001. We anticipate that LONGWING can even present worthwhile data on the preliminary results of STK-001 on seizures together with non-seizure points of the illness, equivalent to high quality of life and cognition.

Enrollment and dosing in LONGWING are ongoing.

The BUTTERFLY research is a multicenter, longitudinal, potential, observational research of youngsters and adolescents ages 2 to 18 who’ve been identified with Dravet syndrome because of an SCN1A gene mutation. This observational research was designed to guage neurodevelopmental standing and alter from baseline to 24 months. Secondary and exploratory endpoints within the research evaluated modifications in different illness measures, together with seizures and extra non-seizure comorbidities. No investigational medicines or different therapies have been supplied. Members continued to obtain their ordinary care, together with anti-seizure medicines, and have been noticed by a group of medical doctors and nurses over time for as much as two years. The research was performed at roughly 20 websites in the US.

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