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Stoke Therapeutics Announces Plans to Move Forward With Dosing of STK-001 in Children and Adolescents in its Ongoing Phase 1/2a MONARCH Study for Dravet Syndrome
- FDA to Allow an Additional Higher Dose Level in the Single Ascending Dose Portion (Formerly, Part A) of the Study – - New Preclinical Repeat-Dose Data
About this update from Stoke Therapeutics, Inc.
[{"type":"text","content":"\n- FDA to Allow an Additional Higher Dose Level in the Single Ascending Dose Portion (Formerly, Part A) of the Study – \n\n- New Preclinical Repeat-Dose Data Support Company Plans to Evaluate Multiple Ascending Dose Levels in MONARCH, Subject to FDA Review -\n\n- Enrollment and Dosing in MONARCH is Ongoing; Preliminary Data Still Anticipated in 2021 - \n\n BEDFORD, Mass.--(BUSINESS WIRE)--\nStoke Therapeutics, Inc., (Nasdaq: STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced plans to move forward with dosing of STK-001 in children and adolescents in its ongoing Phase 1/2a MONARCH study for Dravet syndrome. Following recent interactions with the U.S. Food and Drug Administration (FDA) related to the partial clinical hold on higher dose levels in the MONARCH study, the FDA will allow the Company to add an additional higher dose level to the single ascending dose (SAD) portion of the study (previously Part A). A total of three dose levels will now be evaluated in this portion of the study: 10 mg, 20 mg and 30 mg. Dosing above 30 mg in this study remains on FDA partial clinical hold.\n\nIn addition, subject to FDA review, the Company is preparing to add a multiple ascending dose (MAD) portion to the MONARCH study, replacing Part B. This decision is based on new preclinical repeat-dose toxicology data, which were reviewed by the FDA as part of ongoing discussions with the Company. There were no adverse effects observed in the non-human primate (NHP) repeat dose study. The Company plans to submit a protocol amendment to the FDA, which will reflect these changes to the SAD and MAD portions of the study.\n\n“There is an urgent need for more effective medicines for people who are living with Dravet syndrome, so we are pleased to be moving ahead quickly with our plans to continue dosing children and adolescents in this important Phase 1/2a study of STK-001,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “We appreciate the FDA’s timely review of our additional data and look forward to evaluating a total of three individual dose levels in the single ascending dose portion of the study. In addition, we are encouraged by preclinical data that demonstrated the ability to safely achieve gre...