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Cytokinetics announced that additional data from Cohort 4 of REDWOOD-HCM - Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM, a Phase 2, open-label clinical trial of aficamten in patients with non-obstructive hypertrophic cardiomyopathy, were presented in a Late Breaking Clinical Trial session at Heart Failure 2023 an International Congress of the European Society of Cardiology taking place online and in Prague, Czech Republic from May 20, 2023 - May 23, 2023. The new data from Cohort 4 build on the previously presented initial data from Cohort 4 in REDWOOD-HCM, including analyses of all 41 patients through the end of the 12-week clinical study, and new data relating to the effect of aficamten on Kansas City Cardiomyopathy Questionnaire Clinical Symptom Score and angina. At 10 weeks, treatment with aficamten was associated with an average improvement in KCCQ-CSS of 10.6 points. Overall, 58% of patients experienced a clinical reduction in symptom burden: 12.5% had a small improvement, 20% had a moderate to large improvement, and 25% had a large to very large improvement. Additionally, 56% of patients demonstrated improvement of greater than or equal to1 New York Heart Association Functional Class. By Week 10, 28% of patients were asymptomatic. Furthermore, in the 14 patients who reported some angina at baseline, there was an average reduction in the Seattle Angina Questionnaire Angina Frequency score of 14.3 points at Week 10, translating to a reduction in angina frequency from daily or weekly, to weekly or monthly. Treatment with aficamten was associated with a mean relative reduction in high-sensitivity cardiac troponin I of 21% by Week 10 with an absolute mean reduction of -24.8 ng/L, and a mean relative reduction in NT-proBNP of 55% by Week 10 with an absolute mean reduction of -870 pg/mL. After the 2-week washout period, NT-proBNP and high-sensitivity troponin I levels returned to baseline levels. As previously reported, aficamten was generally well-tolerated. No adverse events of heart failure were reported. Four patients had serious adverse events, including one previously reported death, and none were attributed to aficamten.

Genentech, a member of Roche, announced results from the Phase II FENopta study evaluating investigational oral fenebrutinib in adults with relapsing forms of multiple sclerosis, or RMS. The study met its primary and secondary endpoints, showing oral fenebrutinib significantly reduced magnetic resonance imaging, or MRI, markers of MS disease activity in the brain compared to placebo. Additionally, pre-clinical data have shown fenebrutinib to be potent and highly selective, and it is the only reversible inhibitor currently in Phase III trials for MS. Fenebrutinib significantly reduced the total number of new gadolinium-enhancing T1 brain lesions compared to placebo, the primary endpoint of the trial. Additionally, fenebrutinib significantly reduced the total number of new or enlarging T2 brain lesions compared to placebo, a secondary endpoint. Furthermore, a higher proportion of patients treated with fenebrutinib were free from any new gadolinium-enhancing T1 brain lesions and new or enlarging T2-weighted brain lesions compared to placebo. T1 lesions, as measured by MRI, are a marker of active inflammation and T2 lesions represent the amount of disease burden or lesion load. The safety profile of fenebrutinib was consistent with previous and ongoing fenebrutinib clinical trials across more than 2,400 people to date. There were no new safety concerns identified in the FENopta study.

Cytokinetics announced that the first participants have been dosed in a Phase 1 randomized, double-blind, placebo-controlled, multi-part, single and multiple ascending dose clinical study of CK-4021586. CK-586 is a cardiac myosin inhibitor in development for the potential treatment of patients with heart failure with preserved ejection fraction. "We are pleased to announce the initiation of the Phase 1 clinical study of CK-586, a cardiac myosin inhibitor with a mechanism of action distinct from aficamten," said Fady I. Malik, M.D., Ph.D., Cytokinetics' Executive Vice President of Research & Development. "As a pioneer in the mechanics of cardiac myosin inhibition, we believe this mechanism may impact the underlying hypercontractility found in certain patients who have heart failure with preserved ejection fraction and look forward to pursuing this clinical hypothesis with CK-586."

Genentech, a member of Roche, announced that the FDA has accepted the company's supplemental Biologics License Application, or sBLA, for Vabysmo for the treatment of macular edema following retinal vein occlusion, or RVO. The sBLA is based on results from the Phase III BALATON and COMINO studies that demonstrated treatment with Vabysmo provided early and sustained improvement in vision, meeting the primary endpoint of non-inferior visual acuity gains at 24 weeks compared to aflibercept. Vabysmo's safety profile was consistent with previous trials.