Rocket Pharmaceuticals Reports Full Year 2017 Financial Results and Operational Highlights
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- Enters 2018 as a
- Multiple Milestones Expected in 2018, with Additional Data in Fanconi Anemia, Disclosure of AAV Program, and Several Programs Advancing Toward the Clinic -
“2017 was an important year for Rocket. We advanced our pipeline of gene
therapy programs focused on life-threatening and devastating pediatric
diseases, and we implemented strategic initiatives that have
strengthened our organization,” said
“With the successful closing of our merger with
Recent Pipeline and Corporate Updates
Promising preliminary patient data reported for RP-L102, a Phase
1/2 lentiviral vector (LVV)-based gene therapy for FA. At the
European Society of Gene and Cell Therapy’s Annual Meeting in
October 2017, Rocket’s collaboration partners at the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spainpresented interim results from the ongoing Phase 1/2 trial of RP-L102 in FA. Data presented in FA patients, without the use of conditioning, showed promising in vivo engraftment and stabilization of blood counts despite declining counts in the months to years preceding therapy.
Studies of FA gene therapy product, RP-L101, utilizing new gene
modified cell product manufacturing process underway. At the
American Society of HematologyAnnual Meeting in December 2017, Rocket’s collaboration partners at the Fred Hutchinson Cancer Research Centerpresented data from a pilot clinical trial assessing the feasibility of a novel lineage depletion protocol for hematopoietic stem cell (HSC) enrichment and subsequent transduction with the LVV-based gene therapy, RP-L101. Collection, lentiviral transduction and re-infusion of gene modified cells was shown to effectively produce long-term engraftment in mice. Patient treatment with this novel protocol is underway.
- Three additional LVV-based gene therapy pipeline programs in rare bone marrow disorders advance towards the clinic. LAD-I and PKD programs continue to move forward in preclinical IND-enabling studies. An additional program targeting Infantile Malignant Osteopetrosis (IMO) is also in preclinical development. Similar to FA, these diseases can lead to serious complications and have very limited long-term safe and effective treatment options.
Rocket commenced trading on the
NASDAQ Global Marketunder the symbol “RCKT”, following the successful closing of its with merger with Inotek Pharmaceuticals Corporation(Inotek) in January. In conjunction with the completion of the merger, the Company announced its executive management team will be led by Dr. Shah and will consist of: Jonathan Schwartz, M.D., Chief Medical Officer, and Kinnari Patel, Pharm.D., MBA, Chief Operating Officer.
Rocket completed an oversubscribed underwritten public offering of
6,325,000 shares of its common stock in
January 2018. Net proceeds to Rocket from the offering were approximately $78.8 millionafter deducting underwriting discounts and commissions.
- Updated FA patient data (2Q18)
- Preclinical data and disclosure of the AAV-based gene therapy program (2H18)
Targetrolling IMPD filing for the LAD-I program (4Q18)
- Target IND filing for the AAV-based program (next 12 months)
Targetrolling IMPD filing for the PKD program (next 12 months)
The foregoing financial results presented for the year ended
December 31, 2017represent Rocket Pharmaceuticals Ltd.as a standalone private company, as the merger with Inotek closed on January 4, 2018. The financial statements for the first quarter 2018 will reflect the financial results as a merged public company.
Research and development expenses were
$14.9 millionfor the year ended December 31, 2017, compared to $6.0 millionfor the year ended December 31, 2016. General and administrative expenses were $4.9 millionfor the year ended December 31, 2017, compared to $1.6 millionfor the year ended December 31, 2016.
Net loss was
$19.6 millionor $(219.49)per share (basic and diluted) for the year ended December 31, 2017, compared to $7.6 millionor $(84.43)per share (basic and diluted) for the year ended December 31, 2016.
Cash as of
December 31, 2017for Rocket Pharmaceuticals Ltd.was $18.1 million. Following the merger with Inotek and the January 2018offering of common stock, cash, cash equivalents and short-term investments as of February 28, 2018totaled approximately $185 million, which includes a $52 millionfully convertible debenture which expires in 2021.
The standalone historical financial statements of Inotek for the year
December 31, 2017are presented in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2017.
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
products 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, 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
Claudine Prowse, Ph.D.
SVP Corporate Development and IRO
Rocket Pharmaceuticals, Inc.
The Alexandria Center for Life Science
430 East 29 Street, Suite 1040
New York, NY 10016