Dravet syndrome

Stoke Therapeutics to Present Data from the Company’s Dravet Syndrome Clinical Program at the American Epilepsy Society 2023 Annual Meeting

Retrieved on: 
Tuesday, November 21, 2023

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced that presentations related to the Company’s work in Dravet syndrome will be presented at the American Epilepsy Society (AES) 2023 Annual Meeting, taking place December 1 – 5, in Orlando, Florida.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced that presentations related to the Company’s work in Dravet syndrome will be presented at the American Epilepsy Society (AES) 2023 Annual Meeting, taking place December 1 – 5, in Orlando, Florida.
  • The company is advancing STK-001 as potentially the first medicine to address the genetic cause of Dravet syndrome.
  • “People living with Dravet syndrome typically experience high rates of seizures and debilitating effects on neurodevelopment, including behavioral and developmental delays, movement and balance issues, and delayed language and speech, among other life-altering challenges, which current treatments do not adequately address,” said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics.
  • “We look forward to sharing the findings from more than 3 years of clinical studies, including important new data from the longest and largest prospective natural history study of patients with Dravet syndrome, which showed that despite treatment with multiple anti-seizure medicines, most patients continued to experience convulsive seizures over 24 months and that gaps in neurodevelopment persist between children with Dravet syndrome and their neurotypical peers.

Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates

Retrieved on: 
Tuesday, November 7, 2023

These data, along with encouraging feedback from clinicians, support our belief that STK-001 is a disease-modifying approach that is moving treatment beyond seizure management to address the syndrome.

Key Points: 
  • These data, along with encouraging feedback from clinicians, support our belief that STK-001 is a disease-modifying approach that is moving treatment beyond seizure management to address the syndrome.
  • Following an analysis of these data, the Company plans to meet with regulators to discuss a Phase 3 study design.
  • As of September 30, 2023, Stoke had $214.7 million in cash, cash equivalents, and marketable securities, which is anticipated to fund operations to the end of 2025.
  • General and administrative expenses for the nine months ended September 30, 2023, were $30.7 million, compared to $29.5 million for the same period in 2022.

Ovid Therapeutics Reports Third Quarter 2023 Financial Results and Corporate Updates

Retrieved on: 
Friday, November 3, 2023

The remaining 13% interest in these potential future milestone payments and royalties was acquired by Ligand in October 2023.

Key Points: 
  • The remaining 13% interest in these potential future milestone payments and royalties was acquired by Ligand in October 2023.
  • General and administrative expenses were $6.8 million for the three months ended September 30, 2023, as compared to $7.6 million for the same period in 2022.
  • The decrease reflects the impact of headcount reductions conducted as part of organizational changes made in the first half of 2023.
  • Total operating expenses were $12.1 million for the three months ended September 30, 2023, as compared to $12.8 million for the same period last year.

Longboard Pharmaceuticals Provides Corporate Update and Reports Third Quarter 2023 Financial Results

Retrieved on: 
Thursday, November 2, 2023

Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today provided a corporate update and reported financial results for the third quarter ended September 30, 2023.

Key Points: 
  • Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today provided a corporate update and reported financial results for the third quarter ended September 30, 2023.
  • “We look forward to PACIFIC Study topline data in January 2024 evaluating LP352 in people living with Developmental and Epileptic Encephalopathies, or DEEs.
  • DEE caregivers and healthcare providers continue to be frustrated with the lack of new treatment options and remain focused on finding treatment options that balance safety, efficacy and burden.
  • We look forward to Phase 1 SAD data in the first half of 2024,” stated Kevin R. Lind, Longboard’s President and Chief Executive Officer.

Ovid Therapeutics and Ligand Pharmaceuticals Enter into a $30 Million Agreement for a 13% Interest in Soticlestat Royalties and Milestones Extending Ovid’s Cash Runway into 2026

Retrieved on: 
Wednesday, October 18, 2023

In return, Ovid will receive a $30 million payment, less certain reimbursable expenses.

Key Points: 
  • In return, Ovid will receive a $30 million payment, less certain reimbursable expenses.
  • “We are pleased to partner with Ligand which recognizes the potential value of our royalty and milestone payments associated with soticlestat,” said Jeffrey Rona, Chief Business and Financial Officer of Ovid.
  • With this additional, non-dilutive capital infusion from Ligand, Ovid anticipates that its cash runway is expected to last into 2026 and will enable the expansion of its clinical programs.
  • Ovid has no ongoing obligations or costs associated with the development of soticlestat.

Stoke Therapeutics Appoints Ian Smith to its Board of Directors

Retrieved on: 
Wednesday, September 20, 2023

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced the appointment of Ian Smith to its Board of Directors effective September 19.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced the appointment of Ian Smith to its Board of Directors effective September 19.
  • “Ian brings more than two decades of significant executive management experience in the life sciences industry, with a strong track record of successfully building valuable biotech businesses,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics.
  • “I am pleased to join Stoke’s Board of Directors and look forward to working with the team to help them achieve their goals.
  • Together, I believe we have the opportunity to fundamentally change the way certain diseases are treated, starting with Dravet syndrome.”
    Ian F. Smith is a director of Stoke Therapeutics.

Stoke Therapeutics Presents Data Related to the Ongoing Clinical Development of STK-001 for the Treatment of Dravet Syndrome at the 35th International Epilepsy Congress

Retrieved on: 
Tuesday, September 5, 2023

Four posters from the Company’s work in Dravet syndrome are being presented at the International Epilepsy Congress (IEC) 2023, September 2-6, in Dublin, Ireland.

Key Points: 
  • Four posters from the Company’s work in Dravet syndrome are being presented at the International Epilepsy Congress (IEC) 2023, September 2-6, in Dublin, Ireland.
  • Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures beginning within the first year of life.
  • The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease.
  • All presentations are available for download on the Stoke Therapeutics website under the Investors & News tab.

Researchers Use the Simulations Plus DILIsym® Platform to Predict Safety of Combined CBD and VPA Treatment

Retrieved on: 
Tuesday, August 15, 2023

CBD is an FDA-approved treatment for seizures in patients diagnosed with Dravet syndrome, Lennox-Gastaut syndrome, and tuberous sclerosis complex.

Key Points: 
  • CBD is an FDA-approved treatment for seizures in patients diagnosed with Dravet syndrome, Lennox-Gastaut syndrome, and tuberous sclerosis complex.
  • During the pre-approval clinical trials, treatment with CBD was occasionally associated with elevation in blood levels of ALT, a marker of liver injury, but this was much more common in patients already receiving treatment with VPA for seizures.
  • To identify potential underlying mechanisms, researchers used DILIsym to predict ALT levels in simulated populations that were treated with CBD alone, treated with VPA alone, and then treated with VPA before they were treated with CBD.
  • DILIsym predicted dose-dependent ALT elevations for CBD treatment and ALT elevations for VPA treatment when each was given alone, but the combined VPA and CBD treatment protocol mimicking the clinical trials did not increase the incidence of ALT elevations relative to CBD treatment on its own.

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates

Retrieved on: 
Monday, August 7, 2023

“We recently shared positive new data supporting the potential for STK-001 as the first disease-modifying treatment for Dravet syndrome.

Key Points: 
  • “We recently shared positive new data supporting the potential for STK-001 as the first disease-modifying treatment for Dravet syndrome.
  • Single and multiple doses of STK-001 from 10mg up to 70mg have been generally well tolerated.
  • During the quarter ended June 30, 2023, Stoke updated its estimate of the total effort it expected to expend to satisfy its performance obligations under the Acadia collaboration.
  • As a result, Stoke recorded a cumulative catch-up adjustment of $(5.3) million which resulted in a reversal of revenue during the quarter ended June 30, 2023.

Ovid Therapeutics Reports Second Quarter 2023 Financial Results and Corporate Updates

Retrieved on: 
Friday, August 4, 2023

Completed single ascending dose (SAD) portion of Phase 1 trial evaluating OV329, a potential next-generation GABA-aminotransferase inhibitor

Key Points: 
  • Completed single ascending dose (SAD) portion of Phase 1 trial evaluating OV329, a potential next-generation GABA-aminotransferase inhibitor
    NEW YORK, Aug. 04, 2023 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare epilepsies and seizure-related disorders, today reported business updates and financial results for the second quarter ended June 30, 2023.
  • “In the second quarter, we accomplished a strategic milestone for Ovid.
  • In the second quarter 2023, Ovid executed several non-routine non-operating cash expenditures.
  • Second quarter expenses included severance costs following a headcount reduction that primarily impacted roles within Ovid's G&A functions in June 2023.