AI Earnings Predictions for Stoke Therapeutics Inc. (STOK)

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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."

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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

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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

8-K

false 0001623526 0001623526 2026-01-11 2026-01-11

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of Report (Date of earliest event reported): January 11, 2026

Stoke Therapeutics, Inc. (Exact name of Registrant as Specified in Its Charter)

Delaware

001-38938

47-1144582

(State or Other Jurisdiction of Incorporation)

(Commission File Number)

(IRS Employer Identification No.)

45 Wiggins Ave

Bedford, Massachusetts

01730

(Address of Principal Executive Offices)

(Zip Code) Registrant’s Telephone Number, Including Area Code: (781) 430-8200

(Former Name or Former Address, if Changed Since Last Report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

Trading Symbol(s)

Name of each exchange on which registered

Common Stock, $0.0001 par value per share

STOK

Nasdaq Global Select Market Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter). Emerging growth company ☐ If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Item 2.02 Results of Operations and Financial Condition.

On January 11, 2026, Stoke Therapeutics, Inc. (the “Company”) issued a press release (the “Press Release”) announcing updates to timelines for the completion of enrollment and Phase 3 data readout from its ongoing EMPEROR study and that the Company expects to report that it had approximately $391.7 million in cash, cash equivalents and marketable securities as of December 31, 2025, which, when combined with eligible proceeds from the Biogen collaboration, is anticipated to fund the Company’s operations into 2028. The Company’s audited financial statements for the fiscal year ended December 31, 2025, are not yet available. Accordingly, the preliminary financial information included in the Press Release is an estimate subject to the completion of the Company’s financial closing procedures and any adjustments that may result from the completion of the audit of the Company’s financial statements. The preliminary financial information may differ materially from the actual results that will be reflected in the Company’s audited financial statements when they are completed and publicly disclosed. The information in this Item 2.02 shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1924, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference into any other filing under the Exchange Act or the Securities Act of 1933, as amended (the “Securities Act”), except as expressly set forth by specific reference in such a filing.

Item 7.01 Regulation FD.

On January 11, 2026, the Company issued the Press Release, a copy of which is furnished hereto as Exhibit 99.1 The information furnished with this report, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference into any other filing under the Exchange Act or the Securities Act, except as expressly set forth by specific reference in such a filing.

Item 8.01 Other Events.

The Press Release provides an update related to the design of the Company’s Phase 3 clinical trial of zorevunersen, including the following: Phase 3 Study of Zorevunersen

•

The Company expects to complete enrollment of 150 patients in the second quarter of 2026, with a data readout in mid-2027 that is anticipated to support the submission of a New Drug Application (an “NDA”) to the U.S. Food and Drug Administration (the “FDA”). The Company plans to initiate a rolling NDA submission in the first half of 2027.

•

The Company held a multidisciplinary meeting with the FDA to discuss the ongoing clinical development of zorevunersen, including the exploration of potential expedited regulatory pathways. No immediate changes to the zorevunersen development program we