Stoke Therapeutics to Host Webcast to Discuss Successful Global Regulatory Alignment for a Phase 3 Study of Zorevunersen as Potentially the First Disease Modifying Medicine for Dravet Syndrome

Rhea-AI Summary

Stoke Therapeutics (NASDAQ: STOK) has announced a webcast and conference call scheduled for January 7, 2025, at 8:00am Eastern Time to discuss successful regulatory alignment regarding a Phase 3 study of zorevunersen. The drug is being developed as potentially the first disease-modifying treatment for Dravet syndrome.

The company will host the event for investors and analysts, with research analysts having the opportunity to participate in a Q&A session through registration. The webcast will be accessible through Stoke's investor relations website, and a replay will be available for at least 90 days following the event.

Positive

• Achieved successful alignment with global regulatory agencies for Phase 3 study
• Potential first-in-class disease-modifying treatment for Dravet syndrome
• Advanced drug candidate zorevunersen to Phase 3 development stage

Negative

• None.

BEDFORD, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today announced that its management team will host a webcast and conference call for investors and analysts to discuss successful alignment with global regulatory agencies related to a Phase 3 study of zorevunersen as potentially the first disease-modifying medicine for the treatment of Dravet syndrome.

The webcast will be conducted Tuesday, January 7, 2025 at 8:00am Eastern Time and can be accessed from the Investors & News section of Stoke’s website at https://investor.stoketherapeutics.com/. 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. For all others, the listen-only webcast can be accessed by clicking here. A replay of the webcast will be archived and available for at least 90 days following the event.

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 protein levels. Stoke’s first compound, zorevunersen (STK-001), is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Dravet syndrome is one of many diseases caused by a haploinsufficiency, in which a loss of ~50% of normal protein levels leads to disease. Stoke is pursuing the development of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. Stoke’s initial focus is haploinsufficiencies and diseases of the central nervous system and the eye, although proof of concept has been demonstrated in other organs, tissues, and systems, supporting its belief in the broad potential for its proprietary approach. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/.

About Zorevunersen

Zorevunersen is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials. Stoke believes that zorevunersen, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. Zorevunersen is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. Zorevunersen 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20250106021231/en/

Stoke Media & Investor Contacts:

Dawn Kalmar

Chief Communications Officer

dkalmar@stoketherapeutics.com

781-303-8302



Doug Snow

Director, Communications & Investor Relations

IR@stoketherapeutics.com

508-642-6485

Source: Stoke Therapeutics, Inc.

FAQ

What is the purpose of STOK's January 7, 2025 webcast?

The webcast will discuss successful regulatory alignment for a Phase 3 study of zorevunersen as a potential first disease-modifying treatment for Dravet syndrome.

What stage is STOK's zorevunersen drug development currently in?

Zorevunersen is advancing to Phase 3 clinical trials following successful alignment with global regulatory agencies.

How can analysts participate in STOK's January 2025 webcast Q&A session?

Research analysts can register to receive dial-in details and a unique PIN to participate in the Q&A session.

What makes STOK's zorevunersen unique in Dravet syndrome treatment?

Zorevunersen is being developed as potentially the first disease-modifying medicine for Dravet syndrome.