Rocket Pharmaceuticals (RCKT) investor relations material

Regulatory and clinical development updates

• Danon disease program will dose three patients in H1 2026, followed by FDA discussions on trial continuation in H2 2026.

• PKP2 program reached agreement with FDA on dosing; study design discussions ongoing with regulatory clarity expected in 2026.

• Danon disease update to include safety, epidemiology, and FDA guidance in the second half of 2026.

• PKP2 pivotal trial endpoints may combine arrhythmias, right ventricular function, and clinical outcomes; patient selection will target moderately progressed individuals.

• BAG3 program IND cleared for mid-2026; phase I trial to begin with dose escalation based on preclinical and prior program experience.

Commercial and operational strategy

• KRESLADI (LAD-I) PDUFA date is March 28, 2026, with plans for immediate commercial readiness and patient access.

• Commercial infrastructure and experience from KRESLADI launch will support future AAV-based cardiac programs.

• Operational restructuring completed; company has ~200 staff and is prepared for internal AAV manufacturing and commercial scaling.

• Focus has shifted from lentiviral to AAV pipeline, with resources prioritized for rare cardiac genetic therapies.

Market and epidemiology insights

• Danon disease awareness is increasing; LAMP2 gene added to cardiomyopathy panels only in mid-2010s, with ICD-10 code established recently.

• Genotype-phenotype correlation in Danon is strong, especially in males, aiding diagnosis and patient identification.

• Genetic testing for cardiomyopathies is more widespread in Europe and Japan than in the US; US testing rates remain low but are expected to rise.