Rocket Pharmaceuticals Inc at Guggenheim Genomic Medicines and Rare Disease Day (Virtual) Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Appreciate that overview. So given that Rocket's portfolio spans lentiviral vectors on one side and AAV on the other side, can you walk us through the CMC and regulatory hurdles that is unique to each of these and given this is like the area where a lot of your peers have stumbled in 2021 and 2022?

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Thank you for that. So you did mention the next wave of programs. You directed us to think more in terms of Danon disease at least from a commercial opportunity. So are you tilting more towards AAV going forward? And from a capsid perspective, is this going to be leveraged to AAV9, or are you going to sort of expand beyond AAV9?

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Got it. You did mention Danon, a regulatory milestone catalyst sometime middle of the year or back end of the year; I'm not exactly sure. But what additional information, from a company perspective, do you need before cementing the regulatory interactions?

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Got it. You just mentioned natural history. I believe by the time you meet with the FDA, you could have about 200 patients-ish on the natural history database. So if you look at across, say, the wall thickness, BNP, survival, et cetera, how would you characterize the available interventional clinical data that you've generated so far?

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Got it. And so what is -- based on your prior interactions with the agency, what is their position on protein expression in Danon's disease or to your earlier introduction from a pivotal study structuring? Is it a composite cardiovascular endpoint with -- in a protein expression as a key secondary endpoint or could that be a primary endpoint as well?

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : And internally, within your discussions with thought leaders and stuff, is there a point of no return for these patients since you said 19 is the median life expectancy in male Danon patients? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 01, 2022 / 12:00PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Guggenheim Genomic Medicines and Rare Disease Day (Virtual)

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Got it. And in terms of follow-up that you think would be required for an approval, what should that look like?

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Got it. And is this disease, based on all the natural history that you have, sort of a biphasic in the sense there's a plateau phase and then there is a very quick downward spiral? And you exclude the class 4 patients, but you can still enroll 2 and 3 in that downward slope?

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Got it. So in the low-dose cohort, was there a direct correlation between vector copy numbers, protein expression, and clinical measures? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 01, 2022 / 12:00PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Guggenheim Genomic Medicines and Rare Disease Day (Virtual)

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Got it. So durability should not be -- or it shouldn't be a concern. But in -- what's the limit of that durability at least from our animal data or anything that you had since the first patient was treated?

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : If you can get 10 years, you come from an oncology setting, 10 years is taking HER2 patients and giving her Herceptin, right? So Danon is phenomenal. And obviously down the road, you can think about maybe different vectors or getting rid of neutralizing antibodies or to eliminate cellular reactivity and come and treat again. So 10 years would be phenomenal. Cool. So I think I already asked you about the class 4 patients and point of no return. But what about female subjects? They do have slightly slower disease progression. And then what would be the relevant clinical hurdles in that setting?

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Got it. And in terms of identifying these patients, what are you doing to screen for Danon subjects?

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Got it. And of the target commercial population, how many have been identified? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 01, 2022 / 12:00PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Guggenheim Genomic Medicines and Rare Disease Day (Virtual)

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Got it. So while you're prepping for the Danon pivotal registration study, you'll be also in a position to launch the Fanconi Anemia program. So walk us through some of the commercial readiness activities that are already happened or happening between now and year end.

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Got it. And on the payment models, this is going to be across the spectrum, a sticker-shock kind of price for a lot of -- appears, especially in Europe. Now I've heard of different models. I was talking to BioMarin in a fireside chat yesterday. They're basically saying they are further along [when] -- their hemophilia A program. It's going to be a pay up -- upfront payment. But there is going to be a clawback provision, sort of, made adequately -- provisions being made on the balance sheet. So as the effect fades or patients have to go in and get replacement therapy, X amount goes to -- for that over a three- to five-year span, and that would be it. How are you guys thinking about pricing because it's -- everybody will have some unique challenges here? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 01, 2022 / 12:00PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Guggenheim Genomic Medicines and Rare Disease Day (Virtual)

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Awesome. Any final thoughts that you want to leave the audience with for 2022? Looks like a big year for you, both regulatory and clinical readout perspective.

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Awesome. I appreciate the time, Kinnari, and very -- good luck to you and the rest of the Rocket team. And hopefully, we could catch up pretty soon after the conference and stuff.

Question: Debjit Chattopadhyay - Guggenheim Securities, LLC - Analyst : Thank you so much. Appreciate your time. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 01, 2022 / 12:00PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Guggenheim Genomic Medicines and Rare Disease Day (Virtual)