— Continued to advance clinical gene therapy
programs for the treatment of Danon Disease, Fanconi Anemia (FA),
Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase
Deficiency (PKD) —
— Updates on all four programs to be presented
at the 2022 Annual Meeting of the American Society of Gene and Cell
Therapy (ASGCT) this month; trials on track for 2022 data readouts
—
— Appointed internationally recognized
cardiovascular physician-scientist and biopharmaceutical executive
Fady Malik, M.D., Ph.D., to Board of Directors –
— Named experienced commercial leader Carlos
Martin to Chief Commercial Officer and proven biotech executive
Jessie Yeung to Vice President of Investor Relations and Corporate
Finance —
— Cash position of $346.6M; operational runway
extended into first half of 2024 —
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading
late-stage, clinical biotechnology company advancing an integrated
and sustainable pipeline of genetic therapies for rare childhood
disorders with high unmet need, today reports financial results for
the quarter ending March 31, 2022, and updates from the Company’s
key pipeline developments, business operations and upcoming
milestones.
“I am pleased with our first quarter progress as we maintained
our strong focus on execution across each of our clinical programs,
in-house AAV manufacturing readiness and overall business
operations,” said Gaurav Shah, M.D., Chief Executive Officer of
Rocket Pharma. “We are looking forward to this month’s ASGCT
meeting, where we will present updates from all four clinical
programs. We also remain on track to deliver full data readouts
through the rest of the year as planned.”
Dr. Shah continued, “In the first quarter, we also bolstered our
Company leadership team with the appointments of Fady Malik, M.D.,
Ph.D., an internationally recognized physician-scientist, to our
Board of Directors, global commercial executive Carlos Martin to
Chief Commercial Officer and capital markets expert Jessie Yeung to
Vice President, Investor Relations and Corporate Finance.
Furthermore, we continued to scale up our in-house manufacturing
facility and remain on track for AAV cGMP manufacturing initiation
in Q2.”
“Finally, we extended our cash runway from the second half of
2023 into the first half of 2024 and have a strong balance sheet
with liquidity through potential regulatory filings and approvals,”
said Dr. Shah. “Taken together, we have generated tremendous
momentum with our best-in-class gene therapies, utilizing both
ex-vivo lentiviral and in-vivo AAV platforms, in our pursuit of
cures for patients facing these rare and truly devastating
diseases.”
Key Pipeline and Operational Updates
- Danon, FA, LAD-I and PKD trials have continued to progress
and remain on track. All 2022 milestones remain on track
including potential top-line readouts for Phase 2 trials in LAD-I
and FA in Q2 and Q3, respectively, pediatric efficacy readout for
the Phase 1 Danon Disease trial in Q3, and the Phase 1 readout for
PKD in Q4.
- Updated clinical data to be presented at ASGCT. Updates
from all four clinical programs to be presented at the 2022 Annual
Meeting of the American Society of Gene and Cell Therapy (ASGCT) at
the Walter E. Washington Convention Center in Washington, D.C., May
16-19.
Details for oral presentations are as
follows:
Title: Extended Results from
First-In-Human Clinical Trial of RP-A501 (AAV9:LAMP2B) Gene Therapy
Treatment For Danon Disease Session: Cardiovascular and
Pulmonary Diseases Presenter: Barry Greenberg, M.D., FHFSA,
University of California, San Diego Medical Center, La Jolla, CA
Date: Monday, May 16, 2022 Session Time: 10:15 a.m. –
12:00 p.m. ET Presentation Time: 10:45 a.m. – 11:00 a.m. ET
Location: Room 206 Abstract Number: 24
Title: Ex vivo Lentiviral-mediated
Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated
Results from Global RP-L102 Clinical Trials Session:
Hematopoietic Stem Cell Gene Therapy Presenter: Agnieszka
Czechowicz, M.D., Ph.D., Center for Definitive and Curative
Medicine, Stanford University School of Medicine, Stanford, CA
Date: Monday, May 16, 2022 Session Time: 3:45 p.m. –
5:30 p.m. ET Presentation Time: 4:15 p.m. – 4:30 p.m. ET
Location: Room 202 Abstract Number: 108
Title: Interim Results from an ongoing
Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for
Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I
(LAD-I) Session: Clinical Trials Spotlight Symposium
Presenter: Donald B. Kohn, M.D., Broad Stem Cell Research
Center, UCLA, Los Angeles and recipient of ASGCT’s 2022 Outstanding
Achievement Award Date: Thursday, May 19, 2022 Session
Time: 8:00 a.m. – 9:45 a.m. ET Presentation Time: 8:00
a.m. – 8:15 a.m. ET Location: Ballroom C Abstract
Number: 1188
Details for the poster presentation are as
follows:
Title: Changing the Treatment Paradigm
for Pyruvate Kinase Deficiency with Lentiviral Mediated Gene
Therapy: Interim Results from an Ongoing Global Phase 1 Study
Presenter: Ami Shah, M.D., Center for Definitive and
Curative Medicine, Stanford University School of Medicine,
Stanford, CA Date: Monday, May 16, 2022 Time: 5:30
p.m. – 6:30 p.m. ET Location: Hall D Abstract Number:
357
- In-house AAV current Good Manufacturing Practice (cGMP)
manufacturing initiation anticipated in Q2. The Company’s
state-of-the-art, 103,720 ft2 manufacturing facility in Cranbury,
N.J. is being scaled up to manufacture AAV drug product for a
planned Phase 2 study in Danon Disease.
- Broadened cardiovascular experience of Rocket’s Board of
Directors. The Company announced the appointment of Fady Malik,
M.D., Ph.D. to its Board of Directors. Dr. Malik brings nearly 25
years of experience as an internationally recognized cardiovascular
physician-scientist and highly successful biopharmaceutical
executive. Dr. Malik is Executive Vice President of Research and
Development at Cytokinetics, a late-stage biopharmaceutical
company, where he has worked in a variety of positions since he
joined the founders to launch the company.
- Named Chief Commercial Officer. In March 2022, Carlos
Martin was appointed to Senior Vice President, Chief Commercial
Officer. Mr. Martin brings over 20 years of global commercial
leadership gained at Novartis, Schering Plough and Eli Lilly. Mr.
Martin will lead the go-to-market entry models in the U.S. and EU
and build-out of key commercial capabilities to pave the way for
potential patient access, reimbursement and treatment of Rocket’s
gene therapies.
- Appointed Vice President, Investor Relations & Corporate
Finance. In March 2022, Jessie Yeung joined the Company as Vice
President, Investor Relations. Ms. Yeung brings more than 15 years
of investor relations, corporate finance and capital market
experience across industries including the biopharmaceutical and
financial sectors. Prior to Rocket, Ms. Yeung was the Head of
Corporate Finance and Investor Relations at Legend Biotech. Ms.
Yeung also has experience as an equity research analyst at Bank of
America Merrill Lynch, Wells Fargo, and J.P. Morgan.
- Published peer-reviewed expanded categorization of severe
PKD. “Who should be eligible for gene therapy clinical trials
in red blood cell Pyruvate Kinase Deficiency (PKD)?: Toward an
expanded definition of severe PKD” was published in the American
Journal of Hematology. The publication summarizes the natural
history of the disease and proposes an expanded categorization of
severe PKD to help identify the most optimal patients for ongoing
gene- and cell-based evaluations.
- Recognized Rare Disease Day with an event at NASDAQ Tower in
New York City. On February 28, 2022, Rocket hosted its annual
Rare Disease Day celebration highlighting the theme, “Rare, But Not
Alone.” More than 250 members of the global rare disease community
and Rocket team gathered in person and virtually to hear about the
impact of rare disease and clinical research from patients,
families, advocacy groups and scientific collaborators and
innovators – including Dr. Moris Danon, founder of Danon Disease.
The event concluded with the lighting of the Empire State Building,
as well as other global landmarks, in Rare Disease Day colors.
Anticipated 2022 Milestones
RP-A501 for Danon Disease (AAV)
- Report data from pediatric patient cohort of Phase 1 trial – Q3
2022
- Initiate pivotal Phase 2 trial activities – Q4 2022
RP-L201 for Leukocyte Adhesion Deficiency-I (LVV)
- Report top-line data from pivotal Phase 2 trial – Q2 2022
RP-L102 for Fanconi Anemia (LVV)
- Report top-line data from pivotal Phase 2 trial – Q3 2022
RP-L301 for Pyruvate Kinase Deficiency (LVV)
- Report preliminary Phase 1 data – Q4 2022
- Initiate pivotal Phase 2 trial activities – Q4 2022
Manufacturing Facility in Cranbury, New Jersey
- Achieve in-house AAV current Good Manufacturing Practice (cGMP)
manufacturing readiness – Q2 2022
Upcoming Investor Conference
- Kinnari Patel, Pharm.D., MBA, President and Chief Operating
Officer, will deliver an in-person company presentation at the Bank
of America Securities Healthcare Conference on Wednesday, May 11 at
2:20 p.m. ET at the Encore Hotel in Las Vegas.
First Quarter Financial Results
- Cash position. Cash, cash equivalents and investments as
of March 31, 2022, were $346.6 million.
- R&D expenses. Research and development expenses were
$30.8 million for the three months ended March 31, 2022, compared
to $28.3 million for the three months ended March 31, 2021. The
increase in research and development expense was primarily driven
by an increase in compensation and benefits expense due to
increased R&D headcount, an increase in laboratory supplies and
manufacturing development expenses, offset by a decrease in
non-cash stock compensation expense.
- G&A expenses. General and administrative expenses
were $11.7 million for the three months ended March 31, 2022,
compared to $10.9 million for the three months ended March 31,
2021. The increase in general and administrative expenses was
primarily driven by an increase in compensation and benefits
expense due to increased G&A headcount, and an increase in
commercial preparation expenses, offset by a decrease in non-cash
stock compensation expense.
- Net loss. Net loss was $43.0 million or $0.67 per share
(basic and diluted) for the three months ended March 31, 2022,
compared to $40.2 million or $0.65 per share (basic and diluted)
for the three months ended March 31, 2021
- Shares outstanding. 64,522,057 shares of common stock
were outstanding as of March 31, 2022
Financial Guidance
- Cash position. As of March 31, 2022, we had cash, cash
equivalents and investments of $346.6 million. In April 2022, the
Company sold 1.3 million shares of common stock for net proceeds of
$17.3 million under the at-the-market facility. With the
at-the-market facility proceeds and other efficiencies, the Company
expects such resources will be sufficient to fund its operating
expenses and capital expenditure requirements into the first half
of 2024, 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 four
clinical programs as well as future pipeline programs.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an
integrated and sustainable pipeline of genetic therapies that
correct the root cause of complex and rare childhood disorders. The
Company’s platform-agnostic approach enables it to design the best
therapy for each indication, creating potentially transformative
options for patients afflicted with rare genetic diseases. Rocket's
clinical programs using lentiviral vector (LVV)-based gene therapy
are for the treatment of Fanconi Anemia (FA), a difficult to treat
genetic disease that leads to bone marrow failure and potentially
cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric
genetic disorder that causes recurrent and life-threatening
infections which are frequently fatal, and Pyruvate Kinase
Deficiency (PKD), a rare, monogenic red blood cell disorder
resulting in increased red cell destruction and mild to
life-threatening anemia. Rocket’s first clinical program using
adeno-associated virus (AAV)-based gene therapy is for Danon
Disease, a devastating, pediatric heart failure condition. For more
information about Rocket, please visit www.rocketpharma.com.
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 its guidance for 2022 in light of
COVID-19, the safety and effectiveness of product candidates that
Rocket is developing to treat Fanconi Anemia (FA), Leukocyte
Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD),
and Danon Disease, the expected timing and data readouts of
Rocket’s ongoing and planned clinical trials, Rocket’s plans for
the advancement of its Danon Disease program following the lifting
of the FDA’s clinical hold 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 monitor the impact of COVID-19 on its business
operations and take steps to ensure the safety of patients,
families and employees, the interest from patients and families for
participation in each of Rocket’s ongoing trials, our expectations
regarding the delays and impact of COVID-19 on clinical sites,
patient enrollment, trial timelines and data readouts, our
expectations regarding our drug supply for our ongoing and
anticipated trials, 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 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, 2021, filed
February 28, 2022 with the SEC. 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.
Three Months Ended March 31,
2022
2021
Operating expenses: Research and development
$
30,794
$
28,309
General and administrative
11,770
10,913
Total operating expenses
42,564
39,222
Loss from operations
(42,564
)
(39,222
)
Research and development incentives
-
500
Interest expense
(464
)
(1,729
)
Interest and other income net
623
911
(Amortization of premium) accretion of discount on investments -
net
(577
)
(639
)
Total other expense, net
(418
)
(957
)
Net loss
$
(42,982
)
$
(40,179
)
Net loss per share attributable to common stockholders - basic and
diluted
$
(0.67
)
$
(0.65
)
Weighted-average common shares outstanding - basic and diluted
64,509,721
61,574,405
March 31, December 31,
2022
2021
Cash, cash equivalents and investments
346,593
388,740
Total assets
460,150
497,020
Total liabilities
42,530
42,296
Total stockholders' equity
417,620
454,724
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220505005970/en/
Media Kevin Giordano Director, Corporate Communications
kgiordano@rocketpharma.com
Investors Jessie Yeung, M.B.A. Vice President, Investor
Relations and Corporate Finance investors@rocketpharma.com
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