AI Earnings Predictions for Stoke Therapeutics Inc. (STOK)

EX-99.1

2 stok-ex99_1.htm

EX-99.1

EX-99.1

EX 99.1

Stoke Therapeutics Announces Third Quarter 2026 Financial Results and Provides Business Updates

–Phase 3 EMPEROR study of zorevunersen, an investigational medicine for Dravet syndrome: ~110 patients through Week 28 primary endpoint, ~20 patients through Week 52 and all continuing into open-label treatment period; data readout anticipated in Q3 2027 followed by the planned U.S. NDA submission in H2 2027–

–Successful FDA meeting to align on U.S. NDA submission: Changes in cognition and behavior, a key secondary endpoint in EMPEROR, will be analyzed using a multicomponent assessment of four Vineland-3 subdomains–

–STK-002, an investigational medicine for ADOA: Dose escalation in the Phase 1 OSPREY study continues with dosing of third cohort underway; initial safety and efficacy data anticipated in H1 2027–

–$358.8M in cash, cash equivalents and marketable securities as of September 30, 2026 expected to fund operations into early 2028–

–Webcast and conference call for analysts and investors at 4:30PM Eastern Time today–

BEDFORD, Mass., October 29, 2026 – 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 third quarter ended September 30, 2026, and provided business updates including progress of the global Phase 3 EMPEROR study, ongoing preparations to support regulatory submissions for zorevunersen in the U.S., and the advancement of STK-002 as a potential treatment for people with Autosomal Dominant Optic Atrophy (ADOA).

“The Phase 3 EMPEROR study of zorevunersen continues to progress well. Approximately 110 of 162 enrolled patients have completed their Week 28 visit when the primary endpoint of change in major motor seizure frequency is measured and approximately 20 are through Week 52, the end of the treatment period. All 20 of these patients are continuing into the open-label extension period of the study and to date, no patients have discontinued treatment,” said Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics. “Our NDA planning is on track following our recent discussion with the FDA. Importantly, the key secondary endpoint of changes in patients' cognition and behavior will be measured using a multicomponent assessment of four Vineland-3 subdomains. This multicomponent measure reflects the diversity of effects that people with Dravet syndrome experience and how to best evaluate them in a clinical trial. We look forward to the Phase 3 data readout in the third quarter of 2027 and completing our NDA submission in the second half of 2027.”

Mr. Smith continued, “Our second clinical program, STK-002, targets the protein haploinsufficiency that causes ADOA and therefore may have the potential to improve vision in people living with this severe genetic disease that often leads to blindness. Dosing in the first two cohorts is complete and dosing in the third cohort is underway with the fourth cohort expected to follow in early 2027. Safety

EX 99.1

and efficacy data are anticipated in the first half of 2027. With this progress across the business, we are building Stoke for the future with the team and capabilities to realize the full potential of zorevunersen, our pipeline and our unique scientific approach.”

Program Highlights

Dravet syndrome (zorevunersen)

• Pivotal Phase 3 EMPEROR Study Progress: Global enrollment is now complete. Enrollment of 162 patients in the primary analysis population (the United States, United Kingdom and Japan) was completed in June 2026, and a data readout from these patients is anticipated in the third quarter of 2027. Patients are progressing through the study and as of October 29, 2026:

o All 162 patients are through Week 8 and have received either two 70 mg loading doses of zorevunersen or sham.

o Approximately 130 are through Week 24 and therefore have completed three of the four doses of zorevunersen or sham in the 52-week treatment period.

o Approximately 110 are through Week 28, the time point at which the primary endpoint of change from baseline in log-transformed major motor seizure frequency is measured.

o Approximately 65 are through Week 40 and have therefore completed all doses of zorevunersen or sham in the 52-week treatment period.

o Approximately 20 are through Week 52, the end of the treatment period. All of these patients are continuing into the open-label treatment period.

o No patients have discontinued treatment in the study.

• Successful Meeting with the FDA: Stoke recently met with the U.S. Food and Drug Administration (FDA) to align on key aspects of the planned U.S. New Drug Application (NDA) for zorevunersen. Stoke plans to continue actively engaging wit

EX-99.1

2 stok-ex99_1.htm

EX-99.1

EX-99.1

EX 99.1

Stoke Therapeutics Announces Second Quarter 2026 Financial Results and Provides Business Updates

– Phase 3 EMPEROR study of zorevunersen in Dravet syndrome: Enrollment of 162 patients complete with data readout anticipated in Q3 2027–

– Pre-NDA meeting with FDA scheduled for H2 2026; rolling U.S. NDA submission planned to initiate in Q1 2027 –

– Phase 1 OSPREY study of STK-002 in patients with ADOA: Dosing complete in sentinel cohort; dose escalation continuing and initial data anticipated in H1 2027–

– $420M in cash, cash equivalents and marketable securities based on $354.3M as of June 30, 2026 and $65.7M from a subsequent ATM sale; expected to fund operations through to potential U.S. commercialization of zorevunersen in early 2028 –

–Webcast and conference call for analysts and investors at 4:30PM Eastern Time today–

BEDFORD, Mass., August 3, 2026 – 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, 2026, and provided business updates including progress of the global Phase 3 EMPEROR study, preparation for a rolling New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) and the advancement of STK-002 as a potential treatment for Autosomal Dominant Optic Atrophy (ADOA).

Stoke will participate in a pre-NDA meeting with the FDA during the second half of 2026 as the Company prepares to initiate the planned rolling NDA submission for zorevunersen in the first quarter of 2027. With the June 2026 completion of enrollment of 162 patients into EMPEROR, a Phase 3 readout is anticipated in the third quarter of 2027. These data are expected to complete the rolling U.S. NDA submission in the second half of 2027. Beyond zorevunersen, dose escalation is underway in the Phase 1 OSPREY study of STK-002 in patients with ADOA, the most common inherited optic nerve disorder. Safety and efficacy data are anticipated in the first half of 2027.

"We have made significant progress across our business this year, including the rapid enrollment of 162 patients into the Phase 3 EMPEROR study and progression of these patients through key study milestones with the first patients now approaching the end of the 52-week treatment period,” said Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics. “With no treatment discontinuations to date in EMPEROR, and supportive four-year safety and efficacy data from the ongoing open-label extension studies, we have confidence in the potential of zorevunersen to change the course of Dravet syndrome. We look forward to our upcoming pre-NDA meeting with the FDA to align on the overall data package content and timing, as well as the statistical analysis plan, as we prepare to initiate our rolling U.S. NDA submission in the first quarter of 2027."

EX 99.1

Mr. Smith continued, “Beyond Dravet syndrome, we are advancing our pipeline. Dose escalation continues in our Phase 1 study of STK-002 in patients with ADOA and initial data are anticipated in the first half of 2027 to guide future clinical development. We are also expanding our early research efforts to identify new haploinsufficient disease targets, and we recently strengthened our leadership team with the addition of Tom McCauley as Chief Scientific Officer to help guide this next phase of platform expansion and translational research. In parallel we continue to enhance our commercial capabilities, enabled by our strong financial position of approximately $420 million that will take us through to a potential U.S. launch of zorevunersen by early 2028 and support the Company’s next phase of growth."

Program Highlights

Dravet syndrome (zorevunersen)

• Pivotal Phase 3 EMPEROR study progress:

o U.S., UK and Japan: In June, Stoke announced completion of enrollment of 162 patients in the U.S., UK and Japan where sham control is administered via lumbar puncture (LP). This is the planned primary analysis population for the U.S. NDA. Patients are consistently progressing through the study. As of July 31, 2026:

▪ Approximately 145 are through Week 8 and have received either two 70 mg loading doses of zorevunersen or sham.

▪ Approximately 80 are through Week 24 and therefore have one dose of zorevunersen or sham remaining in the 52-week treatment period.

▪ Approximately 60 are through Week 28, the time point at which the primary endpoint of change in major motor seizure frequency will be measured.

▪ The first patients are expected to reach Week 52 in August 2026.

▪ No patients have discontinued treatment in the study.

Data from this cohort are anticipated in the third quarter of 2027 and are expected to

EX-99.1

2 stok-ex99_1.htm

EX-99.1

EX-99.1

Stoke Therapeutics Announces First Quarter 2026 Financial Results and Provides Business Updates

– New 4-year longitudinal data from the Phase 1/2a open-label extension (OLE) studies provide additional support for the disease-modifying potential of zorevunersen, an investigational medicine for the treatment of Dravet syndrome –

– Statistically significant improvements in cognition and behavior demonstrated at 1, 2, 3 and 4 years of treatment compared to OLE baseline, in addition to continued durability in seizure reductions –

– Zorevunersen generally well tolerated, with some patients treated for more than 5 years –

– Enrollment of approximately 150 patients into the Phase 3 EMPEROR study expected to complete in June 2026 to support a data readout in mid-2027; these data are anticipated to complete the rolling U.S. NDA submission planned to initiate in first quarter 2027 –

– As of March 31, 2026, the Company had $411.0 million in cash, cash equivalents and marketable securities expected to fund operations into 2028, including $80.7 million raised through selective ATM sales in first quarter 2026 –

– Webcast and conference call for analysts and investors at 4:30PM Eastern Time today –

BEDFORD, Mass., May 7, 2026 – 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 with Biogen (Nasdaq: BIIB) as a first-in-class potential disease-modifying treatment for Dravet syndrome. The Company today reported financial results for the first quarter ended March 31, 2026, and announced new 4-year longitudinal data from the ongoing Phase 1/2a open-label extension (OLE) studies that provide additional support for zorevunersen as a potential disease-modifying treatment for Dravet syndrome. Statistically significant improvements were demonstrated in cognition and behavior at 1, 2, 3 and 4 years of treatment compared to OLE baseline. Reductions in major motor seizure frequency were observed through 4 years of treatment in patients taking standard anti-seizure medicines (ASMs). Zorevunersen continues to be generally well tolerated, with some patients treated for more than 5 years in the Phase 1/2a and ongoing OLE studies.

The Company also announced an update on progress of the global Phase 3 EMPEROR study. Enrollment of approximately 150 patients in the U.S., UK and Japan is expected to complete in June 2026 to support a data readout in mid-2027. These data are anticipated to complete the rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) planned to initiate in the first quarter of 2027.

“These new 4-year OLE data suggest that zorevunersen may change the course of Dravet syndrome by providing children with durable reductions in seizures and the possibility of a more neurotypical development path. Together with the Phase 1/2a results, the ongoing extension studies offer 5 years of clinical data, providing a unique opportunity to understand the long-term benefits and safety of zorevunersen,” said Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics.

Mr. Smith continued, “We look forward to results from our pivotal Phase 3 study, which is on track to complete enrollment of approximately 150 patients in June to support a data readout in mid-2027. As we continue to see this study through to completion, our focus turns increasingly to commercial preparedness and bringing zorevunersen to patients while also building our pipeline for the future. All of this is supported by our strong financial position taking us through to a potential U.S. launch in early 2028.”

Program Highlights

Dravet syndrome (zorevunersen)

· New 4-year safety and efficacy data from OLE studies: Following treatment in the Phase 1/2a studies 93% (75/81) patients continued treatment in one of two OLE studies. As of the 4-year data cutoff, 77% (58/75) patients remained in these studies. The new 4-year data announced today showed that patients treated with zorevunersen on top of standard anti-seizure medicines continued to experience durable reductions in seizures and ongoing improvements in cognition and behavior. Zorevunersen continues to be generally well tolerated, with some patients treated for more than 5 years in the Phase 1/2a and ongoing OLE studies in which more than 850 doses have been administered. Elevated CSF protein lab values occurred in approximately 94% of patients of which 59% have been classified as a treatment-emergent adverse event. Importantly, no serious or severe clinical manifestations have been associated with CSF protein elevations. There have been no reports of hydrocephalus.

· Pivotal Phase 3 EMPEROR study progress:

o U.S., UK and Japan: New patient entry into screening is now closed. As of May 5, approximately 130 patients had been randomized to zorevuner