Rocket Pharmaceuticals (RCKT) Morgan Stanley 24th Annual Global Healthcare Conference summary

Strategic focus and clinical pipeline

• Renewed focus on cardiovascular genetic medicines with three active clinical programs: Danon disease, PKP2, and BAG3, all progressing rapidly.

• Recent FDA clearance allows continuation of the Danon disease trial after a previous safety hold, maintaining a 12-patient, single-arm design with a recalibrated dose and revised immunomodulation regimen.

• Anticipated completion of Danon trial dosing by mid-2027, with a comprehensive program update scheduled for October 6th.

• Strong cash position with runway into Q2 2028 and multiple financing options available.

• KRESLADI for severe LAD-I approved, with launch planned for Q4 and a WAC of $3.9 million.

Clinical trial design, safety, and dosing

• Danon trial uses a recalibrated dose of 3.8 E13 vector genomes/kg, based on improved understanding of product potency and safety.

• Revised immunosuppressive regimen excludes C3 inhibitor, extends rituximab cycles, and uses eculizumab only as needed, resulting in improved safety.

• Patient identification for Danon trial is robust, with more than nine eligible patients identified and recruitment supported by genetic testing and expanded outreach.

• Exclusion criteria include advanced fibrosis (ejection fraction <50%) and neutralizing antibodies to AAV9.

• Durable treatment effects observed, with protein expression and clinical markers sustained up to seven years in some patients.

Regulatory and market insights

• FDA guidance for rare disease gene therapy is increasingly favorable, with collaborative relationships emphasized.

• Danon disease program benefits from pediatric designation, potentially qualifying for an additional PRV at approval.

• PKP2 and BAG3 programs leverage learnings from Danon, with endpoints focused on protein expression, arrhythmias, RV function, and patient-reported outcomes.

• PKP2 addresses a high unmet need in a population of 50,000–140,000 in the US and Europe, with gene therapy showing life-changing results in early patients.

• BAG3 targets dilated cardiomyopathy, with clinical updates expected soon and endpoints aligned with established cardiac measures.