STOK — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-11-03

• Presentations of new clinical data for zorevunersen at medical congresses have contributed to greater understanding of its disease-modifying potential that is highly differentiated from any currently available treatments. We, together with Biogen, recently presented long-term clinical data at the 16…

• An additional cohort of 34 patients in Europe completed enrollment in August 2026 and an additional cohort of 5 patients in China completed enrollment in October 2026. Global enrollment in the clinical trial is now closed.

• In October 2026, we announced the completion of a successful meeting with the FDA intended to align on key aspects of the planned submission of an NDA for zorevunersen. Following the discussion, we plan to use a multicomponent assessment of four prespecified Vineland-3 subdomains to evaluate changes…

• We are also pursuing treatment for Autosomal Dominant Optic Atrophy (ADOA), the most common inherited optic nerve disorder, with STK-002. In February 2026, the first patient was dosed in the Phase 1 OSPREY study of STK-002. As of October 2026, all eight planned clinical trial sites are active in the…

• On September 23, 2026, we announced the appointment of Bo Cumbo to our Board of Directors. Mr. Cumbo brings deep experience commercializing new medicines for people with rare genetic diseases. Concurrent with Mr. Cumbo’s appointment, Edward Kaye, M.D., retired from his role as a Director on our Boar…

Text removed vs the prior filing · source: 10-Q · 2026-08-03

• Presentations of new clinical data for zorevunersen at medical congresses have contributed to greater understanding of its disease-modifying potential that is highly differentiated from any currently available treatments. The Company will continue its educational efforts at major neurology and epile…

• An additional cohort of approximately 30 patients is currently being enrolled in Europe, where the sham control is administered via a needle prick (NP).  Screening for this cohort has now closed, and enrollment is expected to be completed in August 2026.

• Site activation and screening are also underway in China, where zorevunersen has been granted Breakthrough Therapy Status. Enrollment is anticipated to complete in the second half of 2026.

• Data from the additional patients in Europe and China are not planned for inclusion in the U.S. NDA submission to the FDA.

• In August 2026, we announced that Stoke intends to participate in a pre-NDA meeting with the FDA during the second half of 2026 in preparation for the rolling NDA submission planned to initiate in the first quarter of 2027. Among other items, this meeting is intended to achieve alignment on the data…

Text added vs the prior filing · source: 10-Q · 2026-11-03

• for Medicare and Medicaid Innovation, two mandatory MFN demonstration models under Medicare Parts B and D, respectively. In September 2026, CMS finalized the Part B model, the Global Benchmark for Efficient Drug Pricing (GLOBE) Model, which is scheduled to begin on January 1, 2027, and excludes drug…

• The orphan drug exclusivity contained in the Orphan Drug Act has been the subject of recent scrutiny from the press, from some members of Congress and from some in the medical community. Furthermore, the FDA’s interpretations of the Orphan Drug Act have not been successfully challenged in court and …

• Many of our potential competitors, alone or with their strategic partners, have substantially greater financial, technical and other resources than we do, such as larger research and development, clinical, marketing and manufacturing organizations. Mergers and acquisitions in the biotechnology and p…

• smaller number of competitors. Our commercial opportunity could be reduced or eliminated if competitors develop and commercialize products that are safer, more effective, have fewer or less severe side effects, are more convenient or are less expensive than any product candidates that we may develop…

• We currently have a limited marketing organization in the U.S., and we do not yet have any organization for the sales or distribution of zorevunersen, STK-002 or our future product candidates. To market any products that may be approved, we must build our sales, marketing, managerial and other non-t…

Text removed vs the prior filing · source: 10-Q · 2026-08-03

• for Medicare and Medicaid Innovation, two mandatory MFN demonstration models under Medicare Parts B and D, respectively. If these rules or other MFN pricing rules are finalized, they are likely to mandate reduced prices of at least some drugs in the United States, if they are also sold in comparator…

• The orphan drug exclusivity contained in the Orphan Drug Act has been the subject of recent scrutiny from the press, from some members of Congress and from some in the medical community. Furthermore, the FDA’s interpretations of the Orphan Drug Act have

• not been successfully challenged in court and future court decisions could continue that trend. There can be no assurances that the exclusivity granted to orphan drugs approved by the FDA will not be modified in the future, or as to how any such changes might affect our products, if approved.

• Many of our potential competitors, alone or with their strategic partners, have substantially greater financial, technical and other resources than we do, such as larger research and development, clinical, marketing and manufacturing organizations. Mergers and acquisitions in the biotechnology and p…

• We currently have a limited marketing organization in the U.S., and we do not yet have any organization for the sales or distribution of zorevunersen, STK-002 or our future product candidates. To market any products that may be approved, we must build our sales, marketing, managerial and other non-t…

Text added vs the prior filing · source: 10-Q · 2026-11-03

• (2) On September 22, 2026, Edward Kaye notified the Company of his resignation from the board of directors of the Company, effective September 23, 2026.

Text removed vs the prior filing · source: 10-Q · 2026-08-03

• Each new Rule 10b5-1 Plan that was adopted in the above table includes a representation from the director or officer, respectively, to the broker administering the plan that such director or officer was not in possession of any material nonpublic information regarding the Company or the securities s…

• Once executed, transactions under a Rule 10b5-1 Plan adopted during the period described above will be disclosed publicly through Form 4 and/or Form 144 filings with the SEC in accordance with applicable securities laws, rules, and regulations. Except as may be required by law, we do not undertake a…