Benzinga | Nov 15, 2021 10:53AM EST

Rocket Pharma Provides Updates from its Phase 1 Clinical Trial of RP-A501 in Danon Disease

--Results demonstrate sustained benefit across clinical, functional and biomarker endpoints in all four patients with long-term follow up--

-- NYHA class improvement (from II to I) in all three patients (two low-dose, one high-dose) with closely monitored immunosuppressive regimen--

--Decreased cardiac wall thickness with improved or stabilized ejection fraction on echocardiogram in all three patients with closely monitored immunosuppressive regimen--

-- Sustained improvement or stabilization in BNP and 6-minute walk test across four patients--

--Sustained cardiac LAMP2B expression greater than 50% by immunohistochemistry and improved cardiac tissue architecture in all three patients with closely monitored immunosuppressive regimen--

--RP-A501 generally well tolerated at low-dose with manageable safety profile--

--Webcast to be held at 8:30 a.m. ET today--