Rocket Pharma: Positive Updates from Phase 1 Trial for Heart Failure Gene Therapy

 Pediatric efficacy data for both patients show initial improvements across clinical, functional and biomarker endpoints with six to nine months of follow-up; positive results including protein expression obtained at three and six months consistent with adult cohorts at similar timeframe—

— Results demonstrate sustained clinical benefit across all parameters in adult patients with up to 36 months of follow-up—

— All adult and pediatric patients with closely monitored immunomodulatory regimen showed improvement in New York Health Association (NYHA) class (from II to I) with follow-up of six to 36 months; patients are no longer afflicted with cardiac disease symptoms during regular activity or cardiac-related limitations in physical activity—

— RP-A501 was generally well tolerated with manageable safety profile across pediatric and adult cohorts—

— Strength of clinical data presented to date expected to support Phase 2 pivotal study; FDA feedback on pivotal study design and endpoints anticipated later this year—

— Webcast to be held at 8:00 a.m. ET today, Sept. 30—

Company management will discuss the Danon Disease data via webcast today, Sept. 30, 2022, at 8:00 a.m. ET. To access the webcast, please register online at: https://ir.rocketpharma.com/events-presentations. Participants are requested to register a minimum of 15 minutes before the start of the call.

A simultaneous webcast of the presentation will be available under "Events" in the Investors section of the Company’s website at: https://ir.rocketpharma.com/. The webcast replay will be available on the Rocket website upon completion of the event.

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