Rocket Pharmaceuticals (RCKT) TD Cowen 46th Annual Health Care Conference summary

Financial position and strategic focus

• Ended last year with $189 million in cash, sufficient to fund operations into Q2 2027.

• Strategic focus shifted to rare cardiac diseases using AAV, with reduced emphasis on LV ex vivo programs.

• Exploring partnerships or externalization for the LV portfolio to maximize patient access and shareholder value.

Key pipeline milestones and clinical updates

• KRESLADI (LAD-I) has a PDUFA date of March 28, 2026, with potential for non-dilutive financing and commercialization infrastructure.

• Danon disease program resumed pivotal phase II trial after FDA hold, with protocol changes to improve safety and dose calibration.

• PKP2 program showed consistent improvements in phase I; pivotal phase II trial design discussions with FDA expected in the second half of the year.

• BAG3 program for dilated cardiomyopathy is preparing to start phase I, with safety as the primary initial focus.

Regulatory and clinical trial design

• FDA interactions have been collaborative, with no current indication of changes to pivotal trial designs for Danon.

• Danon trial design: 12-patient single-arm, primary endpoints are LV mass index and protein expression.

• PKP2 trial design options include single-arm and randomized crossover; trial expected to be appropriately sized for gene therapy.