Rocket Pharmaceuticals, Inc.

Welcome to the Rocket Pharmaceuticals Incorporated Investor Conference Call. My name is Vanessa, and I will be your operator for today's call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session. If you have a question, you can enter the queue by pressing star, then 1 on your touch-tone phone. I will now turn the call over to your host, Mayura Cassetti, Director of Business Development and Operations.

Thank you, Vanessa. Good afternoon, everyone. This is Meir Cassetti, Director of Business Development and Operations and Investor Relations Lead at Rocket Pharmaceuticals. Thank you for joining us. The purpose of this call is to share and discuss key updates on our clinical programs. Before we begin, I would like to briefly discuss the use of forward-looking statements on this conference call. Statements we make on this call may include statements which are not historical facts and are considered forward-looking within the meaning of the securities laws and which are usually identified by the use of words such as anticipates, believes, estimates, expects, intends, may, plans, projects, seeks, should, will, and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the State Harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act, and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies, and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies, and prospects, as reflected in or suggested by those forward-looking statements, are reasonable, we can give no assurance that the plans, intentions, expectations, or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including without limitation, those set forth in our earnings release issued earlier today, and in item 1A, risk factors of our annual report on Form 10-K for the year ended December 31st, 2020, and as updated by our subsequently filed quarterly reports on Form 10-Q and our other SEC filings. We assume no obligation to publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise. Participating on today's call on behalf of Rocket are Dr. Gaurav Shyam, Chief Executive Officer, Dr. Jonathan Schwartz, Chief Medical Officer and Clinical Development Senior Vice President, Dr. Gayathri Rao, Chief Development Officer and Head of Regulatory Policy, Carlos Garcia, Chief Financial Officer, and Claudine Prowse, Senior Vice President of Strategy and Corporate Development. There will be a question and answer session at the end of this call in which we will all participate. I will now turn the call over to Gaurav.

Thank you, Mayur, and thank you, everyone, for joining us today. We have several updates to take you through. Let me highlight the four key take-homes for today. First, regarding the Danube clinical home. we are reiterating our previous guidance that based on our most recent FDA interactions and exercising the best of our judgment and estimation, we continue to anticipate resuming the trial this quarter acutely. Second, again, the low dose is demonstrating increasing and durable benefits. We will go over key data points, including some new ones in a minute. Third, For Dannon, we are removing the high dose from future dosing plans. Four, panconi anemia, LAD-1, and PKD remain on track, and we will provide further clinical updates in Q4. Those are the key takeaways. We'll now get into some more details, starting with Dannon. We've been working very closely with the FDA over the last several weeks, and through our discussions, we have agreed to modify our immune suppression and complement inhibition protocols to bolster safety guardrails. In addition to these protocol updates, we've had a chance to review updated low-dose data sets with the FDA that Rocket believes is supportive of its potential as a viable phase 2 dose. Notably, photographing evidence for all three low-dose patients showed improvements, meaning decrease in autophagic vacuoles, a hallmark of Danden disease pathology, as assessed by electron microscopy of cardiac tissue via endomyocardial biopsy. Additionally, two of the three low-dose patients with closely monitored immunosuppressive regimen compliance demonstrated improvements in New York Heart Association class from 2 to 1, which translates to no impairment of function. These patients also demonstrated substantial improvement of a key marker of heart failure, BMP, which decreased by 75% and 79% versus baseline levels, as well as improvements in cardiac output by 35% to 62% compared to baseline, as measured by invasive hemodynamics. All three treated low-dose patients continue to demonstrate stabilization or improvements in BMP six-minute walk test, as well as New York Heart Association class. Given the activity observed in patients in the low-dose cohort, and importantly to mitigate associated safety concerns in the E14 range, we have decided to forego pursuit of the higher doses, meaning 1.1 E14 vector genomes per kilogram or higher. This is a decision that was made in agreement with the FDA and allows us to focus fully on the low-dose moving forward, the 6.7 to 13, also reduces the total number of patients needed in our phase one study, and potentially allows for more rapid progression to phase two. Now, as disclosed in December of 2020, one patient in the high-dose cohort who was the heaviest patient treated to date and had highly advanced disease developed complement mediated thrombotic microangiopathy which resolved fully with transient hemodialysis. This patient continued to have progressive disease considered unrelated to gene therapy by the trial investigator, as well as his transplant cardiologist, and successfully went on to receive a heart transplant. The patient is currently doing well clinically and reports resolution of his baseline biopsy that was present prior to treatment. Analysis of the explanted heart demonstrates fibrosis that was consistent with end-stage data disease. Our belief has always been that the onset of fibrosis in the year or so prior to transplant could diminish the efficacy of gene therapy, and this patient exemplifies the importance of earlier intervention in this disorder. We at Rocket do not consider this a safety issue, and it is not considered related to the whole. We believe It does highlight the importance of the right timing in order for gene therapy to be fully effective. In discussions with the FDA on this case, we have refined our eligibility criteria to focus on patients earlier in disease. Now, importantly, as of this past week, we have submitted all requested changes to FDA and have confirmation that we have agreement on the updated protocol. As mentioned, we expect that we can resume the trial in Q3 with the revised eligibility criteria in place and refined safety measures in place. Moving forward, we have inbound interest from more than 20 patients for participation in the trial, and we look forward to progressing rapidly toward Phase 1 completion and the Phase 2 registration trial. One final point on damage. Throughout the duration of this hold, we have had an exceptionally collaborative discussion and dialogue with the agency. And with our confidence in the low dose and the modifications to our clinical trial protocol, we are truly excited about the prospects of our DANID program and look forward to presenting longer term data on both the low and higher dose patients in the fourth quarter of this year. Now turning to our lentiviral programs. We've provided updates to ASGCT for our Fanconi anemia, LAD1, and PKD programs continue our momentum toward regulatory violence. For our final LENTI program, we are deeply saddened that the first patient treated in our infantile malignant osteocatrosis phase one trial has passed away from likely non-gene therapy related pulmonary complications with autopsy confirmed evidence of pulmonary hemorrhage That was very likely related to thrombocytopenia following conditioning therapy. It also related to underlying osteopetrosis. Of note, pulmonary complications occur more commonly in osteopetrosis patients relative to many other non-malignant immunologic diseases, especially in those patients who are undergoing transplant. Consistent with the protocol, we have paused enrollment, pending a comprehensive evaluation in collaboration with an independent data monitoring committee. We look forward to providing updates on all of our programs in the fourth quarter of 2021. And finally, as many of you may know, Claudine Krause will be transitioning out of Rocket to take on a CFO role at another company. Claudine has been with Rocket for three and a half years and has been an integral part of our growth story from the days of Inateck for those who were there then, who were private to a mid-cap public company. While we are, and I personally am sad to see Claudine leave us, we are tremendously excited for her and sincerely thank her for her contributions here at Rocket. And I'll pass it to Claudine.