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(State or other jurisdiction of incorporation)
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(Commission File Number)
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(IRS Employer Identification No.)
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
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Title of each class
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Trading
Symbol(s)
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Name of each exchange on which registered
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Item 2.02. |
Results of Operations and Financial Condition.
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Item 9.01. |
Financial Statements and Exhibits.
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Press Release, dated August 9, 2021
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104
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Cover Page Interactive Data File (embedded within the Inline XBRL document).
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Rocket Pharmaceuticals, Inc.
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Date: August 9, 2021
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By:
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/s/ Gaurav Shah, MD
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Gaurav Shah, MD
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Chief Executive Officer and Director
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• |
Progressed toward agreement with FDA on changes to the Phase 1 clinical
trial protocol in Danon Disease. Rocket anticipates trial may resume this quarter. The U.S. Food and Drug Administration (FDA) had
previously requested Rocket pause patient dosing in the Phase 1 clinical trial of RP-A501 and modify the protocol and other supporting documents with revised guidelines for patient selection and management. No new drug-related safety events
were observed in the low- or higher-dose adult cohorts; the previously disclosed SAE of thrombotic microangiopathy, which has since resolved, was reclassified as a SUSAR. All follow-up study activities continue. Longer-term results from the
low (6.7e13) and higher dose (1.1e14) adult cohorts will be reported in the fourth quarter. In agreement with FDA, Rocket will no longer continue dosing patients at the higher dose (1.1e14).
|
• |
Presented positive clinical updates from RP-L102 Fanconi Anemia (FA)
program at ASGCT. Preliminary results from the Phase 1 and 2 trials presented in a poster at ASGCT are from nine pediatric patients. For RP-L102, Rocket’s ex vivo lentiviral gene therapy candidate for FA, increasing evidence of engraftment was observed in at least six of the nine patients, including two patients with at least 15-months of follow-up and four
patients with at least 6-months of follow-up. A highly favorable tolerability profile was also observed with all subjects being treated without conditioning and with no reports of dysplasia. One patient experienced a Grade 2 transient
infusion-related reaction. The full data presented are available here.
|
• |
Published two peer-reviewed studies supporting the natural history and
clinical design of FA clinical trial. “Natural gene therapy by reverse mosaicism leads to improved hematology in Fanconi anemia patients” was published in the American Journal of Hematology. Data strengthen the natural history of Fanconi Anemia and indicate that reverse mosaicism is a good prognostic factor in FA and is associated with more favorable long-term
clinical outcomes. FA mosaicism in hematopoietic cells is a biologic and clinical proof-of principle for autologous gene therapy in FA patients and results provide a compelling rationale for continued clinical evaluation of autologous gene
therapy. Additionally, “Improved Collection of Hematopoietic Stem Cells and Progenitors from Fanconi Anemia Patients for Gene Therapy Purposes” was published in Molecular Therapy: Methods & Clinical Development. Results demonstrate the safety and efficacy of filgrastim and plerixafor for mobilization of hematopoietic stem and progenitor cells (HSPCs) and collection
by leukapheresis in FA patients, offering crucial information for the enrollment of FA patients for gene therapy studies.
|
• |
First patient dosed in the RP-L401 Infantile Malignant Osteopetrosis
(IMO) Phase 1 clinical trial passed away from likely non-RP-L401 gene therapy related pulmonary complications. IMO is a bone marrow-derived disorder associated with severe bone and hematologic manifestations leading to death in the
first decade of life, frequently within the first two years of life, without an allogenic hematopoietic stem cell transplant (HSCT).
|
• |
Presented positive clinical updates from RP-L201 Leukocyte Adhesion
Deficiency-I (LAD-I) program at ASGCT. Phase 1/2 data presented in an oral presentation at ASGCT are from four pediatric patients with severe LAD-I. RP-L201, Rocket’s ex-vivo lentiviral gene therapy candidate showed preliminary activity in all four patients, including one patient with 18-months of follow-up and one patient with 9-months of follow-up. CD18
expression substantially exceeded the 4-10% threshold in all four patients, which is associated with survival into adulthood and consistent with the reversal of severe LAD-I phenotype. Most importantly, all four patients were able to leave
the hospital in the weeks following RP-L201 therapy. The full data presented are available here.
|
• |
Presented positive clinical updates from RP-L301 Pyruvate Kinase
Deficiency (PKD) program at ASGCT. Updated preliminary Phase 1 data presented in an oral presentation at ASGCT are from two patients with significant anemia and transfusion requirements that showed sustained tolerability.
Preliminary activity, measured by peripheral blood VCN levels, was observed in both patients during the initial 9-months and 3-months post-treatment, respectively. Durable normalization of hemoglobin levels were observed, from an average
baseline of ~7.4 grams (g)/deciliter (dL) to 13.1 g/dL at 9-months post treatment in the first patient and from a baseline of ~7.0 g/dL to 14.4 g/dL at 6-months post treatment in the second patient. The Phase 1 trial continues to enroll
patients with longer-term data expected in the fourth quarter. The full data presented are available here.
|
• |
Fanconi Anemia (RP-L102)
|
o |
Updated “Process B” data (Q4 2021)
|
• |
LAD-I (RP-L201)
|
o |
Longer-term Phase 2 data (Q4 2021)
|
• |
Danon Disease (RP-A501)
|
o |
Longer-term Phase 1 data (Q4 2021)
|
• |
PKD (RP-L301)
|
o |
Longer-term Phase 1 data (Q4 2021)
|
• |
IMO (RP-L401)
|
o |
Phase 1 clinical update (Q4 2021)
|
• |
Citi’s 16th Annual BioPharma Virtual Conference, Sept. 8-10, 2021
|
• |
Cash position. Cash, cash equivalents and investments as of June
30, 2021 were $426.8 million.
|
• |
R&D expenses. Research and development expenses were $24.8
million for the three months ended June 30, 2021, compared to $16.7 million for the three months ended June 30, 2020, due to an increase in compensation and benefits expense resulting from increased R&D headcount, an increase in non-cash
stock compensation expense, an increase in manufacturing and development costs, and an increase in clinical trials expense.
|
• |
G&A expenses. General and administrative expenses were $9.3
million for the three months ended June 30, 2021, compared to $6.8 million for the three months ended June 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 office and administrative costs.
|
• |
Net loss. Net loss was $34.5 million or $0.55 per share (basic
and diluted) for the three months ended June 30, 2021, compared to $25.0 million or $0.45 per share (basic and diluted) for the three months ended June 30, 2020.
|
• |
Shares outstanding. 63,448,069 shares of common stock were
outstanding as of June 30, 2021.
|
• |
Rocket expects its balance in cash, cash equivalents and investments of $426.8 million as of June 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.
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Three Months Ended June 30,
|
Six Months Ended June 30,
|
|||||||||||||||
2021
|
2020
|
2021
|
2020
|
|||||||||||||
Operating expenses:
|
||||||||||||||||
Research and development
|
$
|
24,798
|
$
|
16,731
|
$
|
53,340
|
$
|
33,687
|
||||||||
General and administrative
|
9,250
|
6,828
|
19,930
|
13,990
|
||||||||||||
Total operating expenses
|
34,048
|
23,559
|
73,270
|
47,677
|
||||||||||||
Loss from operations
|
(34,048
|
)
|
(23,559
|
)
|
(73,270
|
)
|
(47,677
|
)
|
||||||||
Research and development incentives
|
-
|
-
|
500
|
-
|
||||||||||||
Interest expense
|
(251
|
)
|
(1,786
|
)
|
(1,980
|
)
|
(3,360
|
)
|
||||||||
Interest and other income net
|
501
|
429
|
1,412
|
1,395
|
||||||||||||
Amortization of premium on investments - net
|
(727
|
)
|
(124
|
)
|
(1,366
|
)
|
(62
|
)
|
||||||||
Net loss
|
$
|
(34,525
|
)
|
$
|
(25,040
|
)
|
$
|
(74,704
|
)
|
$
|
(49,704
|
)
|
||||
Net loss per share attributable to common shareholders - basic and diluted
|
$
|
(0.55
|
)
|
$
|
(0.45
|
)
|
$
|
(1.20
|
)
|
$
|
(0.90
|
)
|
||||
Weighted-average common shares outstanding - basic and diluted
|
63,061,232
|
55,158,459
|
62,321,926
|
55,020,789
|
||||||||||||
June 30,
|
December 31,
|
|||||||||||||||
2021
|
2020
|
|||||||||||||||
Cash, cash equivalents and investments
|
426,830
|
482,719
|
||||||||||||||
Total assets
|
535,154
|
590,824
|
||||||||||||||
Total liabilities
|
45,717
|
87,305
|
||||||||||||||
Total stockholders' equity
|
489,437
|
503,519
|
Media
|
Investors
|
Kevin Giordano
|
Mayur Kasetty, M.D.
|
Director, Corporate Communications
|
Director, Business Development & Operations
|
kgiordano@rocketpharma.com
|
investors@rocketpharma.com
|