Rocket Pharmaceuticals (RCKT) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Pipeline overview and program updates

• Six gene therapy programs are in the pipeline, with five disclosed and in the clinic, and a sixth entering soon.

• Programs include Fanconi anemia, pyruvate kinase deficiency, leukocyte adhesion deficiency I (LAD-I), Danon disease, PKP2 arrhythmogenic cardiomyopathy, and BAG3 dilated cardiomyopathy.

• BAG3 program is expected to enter the clinic next year.

Danon disease program

• Danon trial completed enrollment; 12-patient, single-arm design with a 12-month endpoint measuring protein expression and LV mass index improvement.

• Response defined as increase in protein expression and ≥10% improvement in LV mass index; both measured at one year.

• Prevalence estimated at 15,000–30,000 in US and Europe, with higher prevalence in females due to longer survival.

• Only about 20% of patients are currently diagnosed; efforts underway to increase diagnosis via genetic testing and outreach.

• Plans to initiate a female-focused clinical study within the next year, with regulatory discussions in the next six months.

PKP2 and BAG3 cardiomyopathy programs

• PKP2 is in Phase I, focusing on safety and identifying pivotal endpoints; endpoints include arrhythmia reduction, EKG/Holter monitoring, and patient quality of life.

• BAG3 program will enter the clinic next year; both programs will assess if small increases in protein expression yield clinical benefit.