Market Overview

Rocket Pharmaceuticals Reports Second Quarter 2018 Financial Results and Operational Highlights


- Achieved Regulatory Designations for Fanconi Anemia (FA) in the
U.S. and Europe;
Registrational Trial on Track for 2019 -

- Presented Promising Fanconi Anemia Data at ASGCT -

- Adeno-associated Viral Vector Program Disclosure on Track for
Fourth Quarter of 2018 -

Pharmaceuticals, Inc.
(NASDAQ:RCKT) ("Rocket"), a leading U.S.-based
multi-platform gene therapy company, today reported financial results
for the quarter ended June 30, 2018, and provided an update on the
Company's recent achievements, as well as upcoming milestones.

"Rocket made significant progress on our clinical, regulatory and
corporate initiatives in the second quarter," said Gaurav Shah, M.D.,
Chief Executive Officer and President of Rocket. "We are pleased with
the positive clinical data from our FA program that were presented at
ASGCT and look forward to additional data over the next 12-18 months.
The momentum has continued with recent regulatory designations for FA,
including Rare Pediatric Disease from the U.S. Food and Drug
Administration (FDA) and Advanced Therapy Medicinal Product (ATMP) by
the European Medicines Agency (EMA). These positive steps set the stage
nicely for a global registrational study in 2019."

"Our additional gene therapy pipeline programs for devastating rare
diseases remain on track. These include our products for Leukocyte
Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD) and our
undisclosed adeno-associated viral vector (AAV) program. We expect to
disclose the indication and share preclinical data from our AAV program
later this year, and clinical data on up two programs in 2019. Our
progress to date has been a true collaboration between the Rocket team,
our partners, physicians, and the patients we serve. We look forward to
meeting the milestones ahead."

Recent Pipeline and Corporate Updates

  • Rare Pediatric Disease Designation for FA. In July 2018, the
    Company was notified that it received Rare Pediatric Disease
    designation from the FDA for RP-L102 for the treatment of FA Type A.
    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 product.
  • Advanced Therapy Medicinal Product Classification for FA. In
    June 2018, the Company was notified that the EMA classified RP-L102 as
    an ATMP. The ATMP classification recognizes and defines medicines for
    human use that are considered gene-, tissue- or cell-based therapies.
    The key benefit of ATMP classification is the early involvement and
    guidance from the EMA's Committee of Advanced Therapies, which is the
    regulatory reviewing body for gene therapies.
  • Phase 1/2 data of RP-L102 in FA shows promising engraftment and
    chromosomal stability leading to improved bone marrow functionality.
    the ASGCT Annual Meeting in May 2018, updated data from the ongoing
    Phase 1/2 clinical trial of RP-L102 was presented and included data
    from four patients that have been followed for 12-24 months and a
    fifth patient, treated with transduction-enhanced RP-L102, that was
    followed for two months. All patients demonstrated continued
    improvement in engraftment following administration of RP-L102 with
    sustained phenotypic reversals and earlier evidence of gene correction
    seen in higher-dosed patients. The progressive increases of corrected
    versus non-corrected peripheral blood leukocytes indicate the
    potential of RP-L102 to restore the functionality of bone marrow
    hematopoietic stem cells. The one patient that received transduction
    enhanced RP-L102 showed the highest transduction efficiency seen to
    date in all five patients treated, with a preliminary drug product
    vector copy number (VCN) of ~2.5 – 3, and a cell dose considered below
    the threshold level of 500,000K CD34+/kg. Rocket plans to engage with
    regulatory authorities to progress RP-L102 towards a potential global
    registrational study in 2019.
  • Stanford University research collaboration. In May 2018, Rocket
    and the Stanford University School of Medicine announced a strategic
    collaboration to support the advancement of FA and PKD gene therapy
    research. Under the terms of the collaboration agreement, Stanford
    will serve as a lead clinical trial research center in the U.S. for
    the planned FA registrational trial and would also be the lead site
    for PKD clinical trials. The project will also separately evaluate the
    potential for non-myeloablative, non-genotoxic antibody-based
    conditioning regimens as a future development possibility that may be
    applied across bone marrow-derived disorders.
  • Strengthened management team with addition of former FDA Director
    of the Office of Orphan Products Development (OOPD)
    . Gayatri R.
    Rao, M.D., J.D., was appointed Vice President, Regulatory Policy and
    Patient Advocacy, in May 2018. Dr. Rao most recently served as
    Director of the OOPD within the FDA for the last five years where she
    was responsible for implementing statutory programs focused on
    promoting the development of medical products for rare diseases. In
    her new role at Rocket, Dr. Rao will support the development of global
    regulatory policies and strategies, patient advocacy initiatives, and
    rare disease natural history studies.

Anticipated Milestones

  • Preclinical data and disclosure of the AAV-based gene therapy program
  • Investigational Medicinal Product Dossier (IMPD) filing in Spain for
    the LAD-I program (4Q18)
  • IMPD filing in Spain for the PKD program (Early 2019)
  • Additional FA patient data (Next 12-18 months)
  • Investigational New Drug (IND) application filing in the U.S. for the
    AAV-based program (2019)
  • IND application filing in the U.S. for the FA program (2019)

September Conferences

  • Citi's 13th Annual Biotech Conference – September 5-6, 2018
    in Boston, MA
  • Morgan Stanley 16th Annual Global Healthcare Conference –
    September 12-14, 2018 in New York, NY
  • Oppenheimer Specialty Pharma & Rare Disease Fall Summit – September
    25-26 in New York, NY
  • Jefferies Gene Therapy and Editing Summit – September 27, 2018 in New
    York, NY

Second Quarter 2018 Financial Results

  • Cash position. Cash, cash equivalents and investments as of
    June 30, 2018, were $171.5 million, which includes a $52.0 million
    fully convertible debenture which expires in 2021.
  • R&D expenses. Research and development expenses were $10.8
    million and $16.5 million for the three and six months ended June 30,
    2018, compared to $2.8 million and $5.1 million for the three and six
    months ended June 30, 2017.
  • G&A expenses. General and administrative expenses were $4.1
    million and $12.8 million for the three and six months ended June 30,
    2018, compared to $0.7 million and $1.3 million for the three and six
    months ended June 30, 2017.
  • Net loss. Net loss was $15.8 million and $31.1 million or
    $(0.40) and $(0.82) per share (basic and diluted) for the three and
    six months ended June 30, 2018, compared to $3.3 million and $6.2
    million or $(0.49) and $(0.91) per share (basic and diluted) for the
    three and six months ended June 30, 2017.
  • Shares outstanding. Approximately 39.5 million shares of common
    stock were outstanding as of June 30, 2018.

Financial Guidance

  • Cash position. Based on its current operating plan, Rocket
    expects its cash, cash equivalents and investments as of June 30,
    2018, will be sufficient to run its operations into 2020.

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. Preclinical studies of additional bone
marrow-derived disorders are ongoing and target 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 an undisclosed rare pediatric disease. For more
information about Rocket, please visit

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, including in collaboration
with academic partners, to treat Fanconi Anemia (FA), Leukocyte Adhesion
Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD) and Infantile
Malignant Osteopetrosis (IMO), 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, Rocket's ability to commence a registrational study in FA
within the projected time periods, 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, 2017. 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 June 30, Six Months Ended June 30,
  2018     2017     2018     2017  
Revenue $ - $ - $ - $ -
Operating expenses:
Research and development 10,772 $ 2,819 16,525 5,104
General and administrative   4,100     702     12,752     1,287  
Total operating expenses   14,872     3,521     29,277     6,391  
Loss from operations (14,872 ) (3,521 ) (29,277 ) (6,391 )
Research and development incentives - 192 186 192
Interest expense (1,363 ) - (2,834 ) -
Interest income 473 - 805 -
Other income   (5 )   -     10     -  
Net loss $ (15,767 ) $ (3,329 ) $ (31,110 ) $ (6,199 )
Net loss per share attributable to common shareholders - basic and
$ (0.40 ) $ (0.49 ) $ (0.82 ) $ (0.91 )
Weighted-average common shares outstanding - basic and diluted   39,483,006     6,795,627     37,954,972     6,795,627  
Selected Balance Sheet Information
(amounts in thousands)
June 30, December 31,
  2018     2017  
Cash, cash equivalents and investments 171,466 18,142
Total assets 207,574 20,147
Total liabilities 50,498 4,628
Total shareholders' equity 157,076 15,519

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