On November 3, 2021 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported its financial results for the quarter ending September 30, 2021 and updates on the Company’s key pipeline developments, business operations, and upcoming milestones (Press release, Rocket Pharmaceuticals, NOV 3, 2021, View Source [SID1234594271]).

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"We are excited about the strong progress we made in the third quarter as we initiated treatment in our trial of RP-A501 for Danon Disease in the low-dose (6.7e13 vg/kg) pediatric patient cohort," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "I am proud of our team’s collaboration with the FDA, through which we were able to swiftly resolve the clinical hold on the Danon trial and resume this highly important work on behalf of Danon patients. We are gearing up for the remainder of 2021 and look forward to reporting a comprehensive clinical update on our Phase I trial in Danon Disease this month at the AHA Scientific Sessions as well as key updates on all five of our first-in-class gene therapy programs throughout the fourth quarter."

Dr. Shah continued, "We are equally excited about the progress of our LAD-I program, where we completed treatment for all nine patients in the RP-L201 Phase 1/2 clinical trial and presented positive interim data updates on the initial seven patients at the ESGCT Congress in October. Based on the data presented from these seven patients, RP-L201 continues to demonstrate a favorable safety profile and preliminary clinical benefit in patients with severe LAD-I. We will share additional clinical data from the LAD-I trial at the 63rd ASH (Free ASH Whitepaper) Annual Meeting in December, where we will also report clinical updates on our Fanconi Anemia and PKD programs. I am proud of the Rocket team’s unwavering dedication to developing and bringing life-changing curative therapies to patients with rare diseases."

Key Pipeline and Operational Updates

Danon Disease:

Initiated pediatric patient treatment in Phase 1 trial of RP-A501 for the treatment of Danon Disease. Rocket has resumed patient enrollment and initiated treatment in the low-dose (6.7e13 vg/kg) pediatric patient cohort. A comprehensive clinical update is anticipated at the American Heart Association (AHA) Scientific Sessions 2021 being held virtually November 13-15, 2021.
Presented previously disclosed data from ongoing RP-A501 Phase 1 trial in Danon Disease at HFSA. The late-breaking oral presentation at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021 overviewed data from the low-dose (6.7e13 vg/kg) adult cohort which demonstrated RP-A501 was well tolerated and showed progressive and durable clinical benefit.
Leukocyte Adhesion Deficiency-I (LAD-I):

Presented positive interim data updates from RP-L201 LAD-I trial at ESGCT. The oral presentation included data from the initial seven patients with severe LAD-I who were treated with RP-L201 in the Phase 1/2 trial. The safety profile of RP-L201 appears favorable with all infusions well tolerated and no drug product-related serious adverse events. Preliminary efficacy was evident in all seven patients, including two patients with at least 12 months of follow-up. All seven patients demonstrated durable neutrophil CD18 expression that exceeded the 4-10% threshold associated with survival into adulthood and consistent with reversal of the severe LAD-I phenotype. Peripheral blood vector copy number (VCN) levels have been stable and in the 0.5 – 2.5 copy per genome range. No patients have had LAD-I related infections requiring hospitalization subsequent to hematopoietic reconstitution post RP-L201. Additional clinical data are anticipated at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held December 11-14, 2021.
Fanconi Anemia (FA):

Presented previously disclosed clinical data from RP-L102 Fanconi Anemia (FA) program at ESGCT. The oral presentation included data for nine pediatric patients treated with RP-L102, Rocket’s ex vivo lentiviral gene therapy candidate, in the ongoing clinical trials. RP-L102 demonstrated a highly favorable safety profile with all subjects being treated without conditioning and with no sign of dysplasia or other concerning features. RP-L102 showed evidence of preliminary engraftment in at least six of nine patients. A clinical update is anticipated at the 63rd ASH (Free ASH Whitepaper) Annual Meeting being held December 11-14, 2021.
Pyruvate Kinase Deficiency (PKD):

Presented incremental updates from RP-L301 Pyruvate Kinase Deficiency (PKD) program at ESGCT. The oral presentation included data from two adult patients treated with RP-L301, Rocket’s ex vivo lentiviral gene therapy candidate, in the ongoing Phase 1 trial. The safety profile of RP-L301 appears favorable with no infusion-related serious adverse events at up to 9 months post-infusion. Both patients have normalized hemoglobin, improved hemolysis markers and no red blood cell transfusion requirements post-engraftment, as well as no hospitalizations post-hospital discharge. A clinical update is anticipated at the 63rd ASH (Free ASH Whitepaper) Annual Meeting being held December 11-14, 2021.
Published peer-reviewed studies supporting scientific rationale for clinical results observed to date in RP-L301 trial. "Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment" was published in Molecular Therapy: Methods & Clinical Development. The studies demonstrate that in the murine model of PKD, reversion of the phenotype is seen when at least 20% of hematopoietic stem/progenitors are corrected, with comprehensive reversion seen when at least 30% of these progenitors are corrected. This provides meaningful scientific rationale for the clinical results that have been observed to date for the first two patients who have received RP-L301.
Infantile Malignant Osteopetrosis (IMO):

Presented preclinical data supporting ongoing RP-L401 Infantile Malignant Osteopetrosis (IMO) Phase 1 trial at ESGCT. The Phase 1 trial is designed to assess safety and tolerability, as well as preliminary efficacy, of RP-L401, Rocket’s ex vivo lentiviral gene therapy candidate. A clinical update on the Phase 1 trial is anticipated later in the fourth quarter.
Anticipated Milestones

Fanconi Anemia (RP-L102)
Updated "Process B" data (Q4 2021)
LAD-I (RP-L201)
Longer-term Phase 2 data (Q4 2021)
Danon Disease (RP-A501)
Longer-term Phase 1 data (Q4 2021)
PKD (RP-L301)
Longer-term Phase 1 data (Q4 2021)
IMO (RP-L401)
Phase 1 clinical update (Q4 2021)
Third Quarter Financial Results

Cash position. Cash, cash equivalents and investments as of September 30, 2021, were $421.5 million.
R&D expenses. Research and development expenses were $40.0 million for the three months ended September 30, 2021, compared to $21.7 million for the three months ended September 30, 2020, due to increase in manufacturing and development costs, an increase in new research agreements of $7.6 million in non-cash expenses, increases in compensation and benefits due to increased R&D headcount, and an increase in non-cash stock compensation expense.
G&A expenses. General and administrative expenses were $9.7 million for the three months ended September 30, 2021, compared to $5.7 million for the three months ended September 30, 2020, due to an increase in non-cash stock compensation expense, an increase in compensation and benefits expense due to increased G&A headcount and an increase in commercial preparation expenses.
Net loss. Net loss was $50.1 million or $0.79 per share (basic and diluted) for the three months ended September 30, 2021, compared to $29.1 million or $0.53 per share (basic and diluted) for the three months ended September 30, 2020.
Shares outstanding. 64,442,601 shares of common stock were outstanding as of September 30, 2021.
Financial Guidance

Rocket expects its balance in cash, cash equivalents and investments of $421.5 million as of September 30, 2021 to fund its operations into the second half of 2023, including the continued buildout and initiation of AAV cGMP manufacturing capabilities at our Cranbury, New Jersey R&D and manufacturing facility and continued development of our five clinical programs.