A Phase 2/3 Multicenter, Randomized, Double-blind, Placebo-controlled and Open-label Extension Trial to Evaluate the Efficacy and Safety of Aficamten in a Pediatric Population with Symptomatic Obstructive Hypertrophic Cardiomyopathy

SYMPTOMATIC OBSTRUCTIVE HYPERTROPHIC CARDIOMYOPATHY

P1: Change in Valsalva LVOT-G while maintaining LVEF ≥ 55% from baseline to Week 12, P2: Participant incidence of AEs and SAEs through End of Study

P1: Change in resting LVOT-G from baseline to Week 12, P1: All participants: Observed Ctrough of aficamten over the 12-week treatment period Intensive PK substudy: Observed Cmax, tmax, AUCtau, and Ctrough for aficamten, P1: Change in NT-proBNP from baseline to Week 12 Change in hs-cTnI from baseline to Week 12, P1: Change in NYHA Functional Class from baseline to Week 12, P1: Proportion of participants with ≥ 1 class improvement in NYHA Functional Class from baseline to Week 12, P2: Change in the following measurements at 12-week intervals from Week 14 (baseline in OLE) through end of treatment: − Peak LVOT-G at rest and with Valsalva provocation, P2: Change in the following measurements at 12-week intervals from Week 14 (baseline in OLE) through end of treatment: Proportion of participants with resting LVOT-G < 30 mmHg, P2: Change in the following measurements at 12-week intervals from Week 14 (baseline in OLE) through end of treatment: − Proportion of participants with Valsalva LVOT-G < 50 mmHg, P2: Change in the following measurements at 12-week intervals from Week 14 (baseline in OLE) through end of treatment: − Proportion of participants with Valsalva LVOT-G < 30 mmHg, P2: Change in the following measurements at 12-week intervals from Week 14 (baseline in OLE) through end of treatment: − Proportion of participants with LVEF ≥ 50%, resting LVOT-G < 30 mmHg, and Valsalva LVOT-G < 50 mmHg, P2: Time to the following event through last follow-up: − First resting LVOT-G < 30 mmHg − First Valsalva LVOT-G < 50 mmHg − First Valsalva LVOT-G < 30 mmHg − First LVEF ≥ 50%, resting LVOT-G < 30 mmHg, and Valsalva LVOT-G < 50 mmHg, P2: Change in NYHA Functional Class from Week 14 to end of treatment, P2: Proportion of participants with ≥ 1 class improvement in NYHA Functional Class from Week 14 to end of treatment

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