Market Overview

Rocket Pharmaceuticals Reports First Quarter 2019 Financial Results and Operational Highlights


– Initiated Phase 1 Trial for FA "Process B" Using a Modified Cell
Enrichment Process, Transduction Enhancers, and Commercial-Grade Vector
Manufacturing and Cell Processing –

– Successful Closing of $90.6 Million Equity Offering to Support
Pipeline –

– On Track for Clinical Data on Two of Four Gene Therapy Programs by
Year End –

Pharmaceuticals, Inc.
(NASDAQ:RCKT) ("Rocket"), a leading
U.S.-based multi-platform clinical-stage gene therapy company, reports
financial results for the quarter ended March 31, 2019, and provides an
update on the Company's recent achievements, as well as upcoming

"Last quarter, we continued to lay the groundwork towards achieving our
objective of having four programs in the clinic by year end," said
Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We
initiated our Phase 1 clinical trial for FA ‘Process B' and are
encouraged by the continued long-term clinical profile of patients
receiving ‘Process A' RP-L102. We are also encouraged by the strong
preclinical data package of RP-A501 as it nears the clinic. In non-human
primate studies, RP-A501 demonstrated robust transduction and LAMP2
protein expression in all four heart chambers, the key target organ for
Danon disease."

"We remain focused on our core strategic goal of bringing transformative
gene therapies to patients with devastating rare diseases, as quickly as
possible. With the closing of our most recent equity raise, we are well
capitalized to advance our pipeline and look forward to providing
updates on two clinical programs by year end," Dr. Shah concluded.

Recent Pipeline and Corporate Updates

  • Presentation of preclinical and clinical updates at the American
    Society of Gene and Cell Therapy (ASGCT) 2019 Annual Meeting.
    ASGCT, Rocket's oral and poster presentations highlighted promising
    data on four gene therapy programs: Fanconi Anemia (FA), Danon
    disease, Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase
    Deficiency (PKD). Oral and poster presentations are available online:
  • Patient dosing commences in Phase 1 clinical study for FA with
    improved "Process B".
    The Center for Definitive and Curative
    Medicine at Stanford is serving as the lead U.S. clinical site.
    "Process B" incorporates a modified cell enrichment process,
    transduction enhancers, and commercial-grade vector manufacturing and
    cell processing. Initial patients have received treatment under
    "Process B" at Stanford under a U.S.-focused Phase 1 study, with
    additional Phase 2 enrollment anticipated in the second half of 2019.
    Preliminary data from the Phase 1 "Process B" study are expected by
    the end of 2019.
  • Partnership with University of California, Los Angeles (UCLA) to
    lead U.S. clinical development efforts for LAD-I and Infantile
    Malignant Osteopetrosis (IMO) programs.
    UCLA and its Eli and
    Edythe Broad Center of Regenerative Medicine and Stem Cell Research is
    serving as the lead U.S. clinical research center for the planned
    registrational clinical trial for LAD-I and also a lead U.S. clinical
    site for IMO.
  • Grant awarded from California Institute for Regenerative Medicine
    CIRM awarded Rocket a $6.5 million grant to support the
    development of RP-L201 for LAD-I. The grant will help fund the U.S.
    Phase 1/2 LAD-I registration-enabling study. The trial will evaluate
    the safety and efficacy of the infusion of autologous hematopoietic
    stem cells transduced with a lentiviral vector encoding the ITGB2
  • Rare Pediatric Disease designation for IMO. Rocket received
    Rare Pediatric Disease designation from the Food and Drug
    Administration (FDA) for RP-L401 for the treatment of IMO. The FDA
    defines a "rare pediatric disease" as a serious and life-threatening
    disease that affects less than 200,000 people in the U.S. that are
    aged between birth to 18 years. The Rare Pediatric Disease designation
    program allows for a Sponsor who receives an approval for a product to
    potentially qualify for a voucher that can be redeemed to receive a
    priority review of a subsequent marketing application for a different
  • Approximately $90.6 million secured in public equity offering.
    Rocket successfully closed an oversubscribed underwritten public
    offering of common stock for gross proceeds of approximately $90.6
    million. Net of expenses, the Company received approximately $86.0
    million in net proceeds.

Anticipated Milestones

  • FA (RP-L102)
    • Initial Phase 1 data under "Process B" (2H19)
    • Regulatory alignment on final endpoints for registration and Phase
      2 study (2H19)
    • Additional data from RP-L102 (2020)
  • Danon Disease (RP-A501)
    • Initiation of Phase 1 study (2Q19)
    • Phase 1 data (2020)
    • Initiate Phase 2/registration-enabling study (2020)
  • LAD-I (RP-L201)
    • Initiation of registration-enabling Phase 1/2 study (2Q19)
    • Initial Phase 1 data (2H19)
    • Additional data from RP-L201 (2020)
  • PKD (RP-L301)
    • Initiation of Phase 1 study (2H19)
    • Data from Phase 1 (2020)
  • IMO (RP-L401)
    • Initiation of clinical study (1H20)

Upcoming Investor Conferences

  • Bank of America Merrill Lynch Global Healthcare Conference 2019. Rocket
    is scheduled to present on Wednesday, May 15, 2019, at 3:40 p.m.
    Pacific Time. Rocket will also participate in a Gene Therapy
    Panel on Wednesday, May 15, 2019, at 4:20 p.m. Pacific Time.

First Quarter 2019 Financial Results

  • Cash position. Cash, cash equivalents and investments as of
    March 31, 2019, were $196.6 million.
  • Debt. Our balance sheet includes a $52.0 million fully
    convertible debenture which matures in August of 2021.
  • R&D expenses. Research and development expenses were $15.1
    million for the three months ended March 31, 2019, compared to $5.7
    million for the three months ended March 31, 2018. The increase was
    primarily driven by increases in manufacturing and process development
    expenses of $5.5 million and research agreement expenses of $1.9
  • G&A expenses. General and administrative expenses were $3.8
    million for the three months ended March 31, 2019, compared to $8.7
    million for the three months ended March 31, 2018. The decrease was
    primarily due to certain stock compensation and severance termination
    expenses in Q-1, 2018 related to the reverse merger with Inotek
    Pharmaceuticals Corporation.
  • Net loss. Net loss was $19.5 million or $0.43 per share (basic
    and diluted) for the three months ended March 31, 2019, compared
    to $15.3 million or $0.42 per share (basic and diluted) for the three
    months ended March 31, 2018.
  • Shares outstanding. 45,114,437 million shares of common stock
    were outstanding as of March 31, 2019.

Financial Guidance

  • Cash position. As of March 31, 2019, we had cash, cash equivalents and
    investments of $196.6 million. On April 18, 2019, Rocket closed a
    public offering of 5,175,000 shares of common stock and received net
    proceeds of approximately $86.0 million. Considering the net proceeds
    from the public offering, Rocket expects such resources will be
    sufficient to fund its operations into the first half of 2021. As of
    April 30, 2019, we had cash, cash equivalents and investments of
    $273.1 million.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket") is an emerging,
clinical-stage biotechnology company focused on developing
first-in-class gene therapy treatment options for rare, devastating
diseases. Rocket's multi-platform development approach applies the
well-established lentiviral vector (LVV) and adeno-associated viral
vector (AAV) gene therapy platforms. Rocket's lead clinical program is a
LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a
difficult to treat genetic disease that leads to bone marrow failure and
potentially cancer. Rocket's additional pipeline programs for bone
marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD),
Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant
Osteopetrosis (IMO). Rocket is also developing an AAV-based gene therapy
program for a devastating, pediatric heart failure indication, Danon
disease. For more information about Rocket, please visit

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future
expectations, plans and prospects, including without limitation,
Rocket's expectations regarding the safety, effectiveness and timing of
product candidates that Rocket may develop, to treat Fanconi Anemia
(FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase
Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon
disease, and the safety, effectiveness and timing of related
pre-clinical studies and clinical trials, may constitute forward-looking
statements for the purposes of the safe harbor provisions under the
Private Securities Litigation Reform Act of 1995 and other federal
securities laws and are subject to substantial risks, uncertainties and
assumptions. You should not place reliance on these forward-looking
statements, which often include words such as "believe," "expect,"
"anticipate," "intend," "plan," "will give," "estimate," "seek," "will,"
"may," "suggest" or similar terms, variations of such terms or the
negative of those terms. Although Rocket believes that the expectations
reflected in the forward-looking statements are reasonable, Rocket
cannot guarantee such outcomes. Actual results may differ materially
from those indicated by these forward-looking statements as a result of
various important factors, including, without limitation, Rocket's
ability to successfully demonstrate the efficacy and safety of such
products and pre-clinical studies and clinical trials, its gene therapy
programs, the preclinical and clinical results for its product
candidates, which may not support further development and marketing
approval, the potential advantages of Rocket's product candidates,
actions of regulatory agencies, which may affect the initiation, timing
and progress of pre-clinical studies and clinical trials of its product
candidates, Rocket's and its licensors ability to obtain, maintain and
protect its and their respective intellectual property, the timing, cost
or other aspects of a potential commercial launch of Rocket's product
candidates, Rocket's ability to manage operating expenses, Rocket's
ability to obtain additional funding to support its business activities
and establish and maintain strategic business alliances and new business
initiatives, Rocket's dependence on third parties for development,
manufacture, marketing, sales and distribution of product candidates,
the outcome of litigation, and unexpected expenditures, as well as those
risks more fully discussed in the section entitled "Risk Factors" in
Rocket's Annual Report on Form 10-K for the year ended December 31,
2018. Accordingly, you should not place undue reliance on these
forward-looking statements. All such statements speak only as of the
date made, and Rocket undertakes no obligation to update or revise
publicly any forward-looking statements, whether as a result of new
information, future events or otherwise.

Selected Financial Information
Operating Results:
(amounts in thousands, except share and per share data)
    Three Months Ended March 31,
  2019     2018  
Revenue $ - $ -
Operating expenses:
Research and development 15,137 5,743
General and administrative   3,808     8,662  
Total operating expenses   18,945     14,405  
Loss from operations (18,945 ) (14,405 )
Research and development incentives 250 186
Interest expense (1,604 ) (1,427 )
Interest and other income net 601 288
Accretion of discount on investments   247     15  
Net loss $ (19,451 ) $ (15,343 )
Net loss per share attributable to common shareholders - basic and
$ (0.43 ) $ (0.42 )
Weighted-average common shares outstanding - basic and diluted   45,122,815     36,137,120  
Selected Balance Sheet Information
(amounts in thousands)
March 31, December 31,
  2019     2018  
Cash, cash equivalents and investments 196,590 213,132
Total assets 242,138 251,313
Total liabilities 65,061 57,276
Total shareholders' equity 177,077 194,037

View Comments and Join the Discussion!