RTW Biotech Opp. - Update on Largest Core Portfolio Position
14/09/2023 07:00
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RNS Number : 4384M RTW Biotech Opportunities Ltd 14 September 2023
LEI: 549300Q7EXQQH6KF7Z84
14 September 2023
RTW Biotech Opportunities Ltd
Update on Largest Core Portfolio Position
· Rocket Pharmaceuticals Reaches Alignment with FDA on Pivotal
Phase 2 Trial Design for RP-A501 in Danon Disease
· On 13 September, Rocket also announced an underwritten public
offering of 7,812,500 shares of its common stock
RTW Biotech Opportunities Ltd (the "Company"), a London Stock Exchange-listed
investment fund focused on identifying transformative assets with high growth
potential across the life sciences sector, is pleased to note the announcement
by its largest portfolio company position, Rocket Pharmaceuticals, Inc.
("Rocket"), that it has reached alignment with the U.S. Food and Drug
Administration ("FDA") on the trial design for its global Phase 2 pivotal
trial of RP-A501 for Danon Disease. The global, single-arm, multi-center Phase
2 pivotal trial will evaluate the efficacy and safety of RP-A501 in 12
patients with Danon. To support accelerated approval, the study will assess
the efficacy of RP-A501 as measured by the biomarker-based primary endpoints.
Danon Disease is a fatal inherited cardiomyopathy for which there is currently
no cure.
Roderick Wong, MD, Managing Partner and Chief Investment Officer, RTW
Investments, LP and Chairman of the Board of Directors, Rocket Pharmaceuticals
said:
"This is a significant milestone for Rocket and for patients with Danon
Disease and puts us a step closer to a potential therapy for an otherwise
incurable disease. We're proud to continue to support this important work and
pave the way for future gene therapies in cardiovascular disease."
Rocket was a 10.3% position in the Company as of 31 August 2023. After an
increase of +38.8% in the share price on 13 September, the estimated uptick to
the 31 August NAV is expected to be approximately 3.9% (not including a
purchase of pre-funded warrants by the Company on 13 September, which has not
yet been factored into this estimated figure).
On 13 September, Rocket also announced the pricing of a public offering of
7,812,500 shares of its common stock at a public offering price of $16.00 per
share and to certain investors, including the Company, pre-funded warrants to
purchase 3,126,955 shares of common stock at a price of $15.99 per pre-funded
warrant. The gross proceeds to Rocket from the offering are expected to be
approximately $175 million.
Rocket is a gene therapy company focused on rare childhood diseases. It was
incubated by the Investment Manager, RTW Investments LP, in 2015 and has been
a part of the Company's core portfolio since its launch on 30 October 2019.
The full text of both announcements is contained below.
For further information:
RTW Investments, LP +44 20 7959 6361
Woody Stileman, Managing Director
Krisha McCune, Director, Client Service
Buchanan +44 20 7466 5107
Charles Ryland
Henry Wilson
George Beale
Numis +44 20 7260 1000
Freddie Barnfield
Nathan Brown
Euan Brown
BofA Securities +44 20 7628 1000
Edward Peel
Kieran Millar
Cadarn Capital +44 73 6888 3211
David Harris
Elysium Fund Management Limited +44 (0) 14 8181 0100
Joanna Duquemin Nicolle, Chief Executive Officer
Sadie Morrison, Managing Director
Morgan Stanley Fund Services USA LLC +1 914 225 8885
About RTW Biotech Opportunities Ltd:
RTW Biotech Opportunities Ltd (LSE: RTW & RTWG) is an investment fund
focused on identifying transformative assets with high growth potential across
the biopharmaceutical and medical technology sectors. Driven by a long-term
approach to support innovative businesses, RTW Biotech Opportunities Ltd
invests in companies developing next-generation therapies and technologies
that can significantly improve patients' lives. RTW Biotech Opportunities Ltd
is managed by RTW Investments, LP, a leading healthcare-focused
entrepreneurial investment firm with deep scientific expertise and a strong
track record of supporting companies developing life-changing therapies.
Visit the website at www.rtwfunds.com/rtw-biotech-opportunities-ltd
(https://www.rtwfunds.com/rtw-biotech-opportunities-ltd/) for more
information.
***********
The information in this announcement may include forward-looking statements,
which are based on the current expectations and projections about future
events, and in certain cases can be identified by the use of terms such as
"may", "will", "should", "expect", "anticipate", "project", "estimate",
"intend", "continue", "target", "believe" (or the negatives thereon) or other
variations thereon or comparable terminology. These forward-looking
statements, as well as those included in any related materials, are subject to
risks, uncertainties and assumptions about the Company and/or its underlying
investments, including, among other things, the development of the applicable
entity's business, trends in its operating industry, expected use of financing
proceeds and future capital expenditures and acquisitions. In light of these
risks, uncertainties and assumptions, the events in the forward-looking
statements may not occur.
The information contained in this announcement is given at the date of its
publication (unless otherwise marked). No reliance may be placed for any
purpose whatsoever on the information or opinions contained in this
announcement or on its completeness, accuracy or fairness.
Rocket Pharmaceuticals Reaches FDA Alignment on Pivotal Phase 2 Trial Design
for RP-A501 in Danon Disease
September 12, 2023
Final alignment reached on a 12 patient, single-arm, open label study with a
biomarker based co-primary endpoint assessed at 12 months to support
accelerated approval
Co-primary endpoint consisting of LAMP2 protein expression and Left
Ventricular (LV) Mass change from baseline
CRANBURY, N.J.--(BUSINESS WIRE)--Sep. 12, 2023-- Rocket Pharmaceuticals, Inc.
(NASDAQ: RCKT), a leading late-stage biotechnology company advancing an
integrated and sustainable pipeline of genetic therapies for rare disorders
with high unmet need, today announced that alignment has been reached with the
Food and Drug Administration (FDA) on the global Phase 2 pivotal trial of
RP-A501 for Danon Disease. Danon Disease is a uniformly fatal inherited
cardiomyopathy that leads to mortality in the majority of male patients at age
~20 and females at age ~40, and for which there are no approved curative or
disease-modifying therapies. The disease affects an estimated 15,000 to 30,000
patients in the U.S. and Europe.
"I am very excited to announce our alignment with the FDA on our pivotal study
design for RP-A501 for Danon Disease, which reflects the highly collaborative
discussions with the review team and senior management at FDA's Center for
Biologics Evaluation and Research and marks the first-ever regulatory pathway
to approval for a genetic treatment for heart disease. We believe this
milestone sets us on the most efficient and rapid path to delivering this
potentially transformative therapy to Danon Disease patients who would
otherwise progress to heart transplantation or death," said Gaurav Shah, M.D.,
Chief Executive Officer, Rocket Pharma. "I would also like to highlight the
work conducted by our CMC team over the past several years to establish our
in-house cGMP manufacturing capabilities, which has already provided us with
sufficient material for the pivotal study and should support our eventual
commercialization efforts."
Dr. Shah continued "As a one-time potentially curative infusion, RP-A501 has
the potential to restore normal cardiac function and provide a lifetime of
benefit to patients with Danon Disease who have no other viable treatment
options. With today's progress in our Danon Disease program, we believe we are
forging a path to bring curative gene therapies to patients affected by
devastating cardiovascular diseases and broadening the possibilities for
addressing the large array of inherited heart diseases through the promise of
cardiac gene therapy."
Phase 2 Pivotal Trial of RP-A501 for Danon Disease
The global, single-arm, multi-center Phase 2 pivotal trial will evaluate the
efficacy and safety of RP-A501 in 12 patients with Danon Disease, including a
pediatric safety run-in (n=2), with a natural history comparator and a dose
level of 6.7 x 1013 GC/kg.
· To support accelerated approval, the study will assess the
efficacy of RP-A501 as measured by the biomarker-based co-primary endpoint
consisting of improvements in LAMP2 protein expression (≥ Grade 1, as
measured by immunohistochemistry), and reductions in left ventricular (LV)
mass.
· Key secondary endpoint is change in troponin. Additional
secondary endpoints will include natriuretic peptides, Kansas City
Cardiomyopathy Questionnaire (KCCQ), New York Heart Association (NYHA) class,
event free survival to 24 months and treatment emergent safety events. These
endpoints could support full approval with longer-term follow-up.
· A global natural history study will serve as an external
comparator and run concurrently to the Phase 2 pivotal trial.
· In-house manufacturing has been completed with sufficient
high-quality drug product produced to fully supply the Phase 2 pivotal study.
Potency assays have been developed and qualified in accordance with FDA
guidance.
Filing of the Clinical Trial Application (CTA)/Investigational Medicinal
Product Dossier (IMPD) for RP-A501 to enable initiation of EU study activities
is anticipated in the third quarter of this year. Additionally, Rocket has
secured an ICD-10 code from CMS for LAMP2 deficiency in Danon Disease
About RP-A501
RP-A501 is Rocket's investigational gene therapy product for the treatment of
Danon Disease and the first gene therapy for a cardiovascular condition to
demonstrate safety and efficacy in clinical studies. Danon Disease is caused
by mutations in the LAMP2 gene. RP-A501 consists of a recombinant
adeno-associated serotype 9 (AAV9) capsid containing a full-length, wild-type
version of the human LAMP2B transgene (AAV9.LAMP2B) which, when inserted into
heart cells harboring mutations in the endogenous LAMP2 gene, has the
potential to fully restore cardiac function at its root. RP-A501 represents a
single dose treatment and is administered as an intravenous (IV) infusion. In
preclinical and clinical studies, AAV9.LAMP2B has been shown to target cardiac
cells (cardiomyocytes) and deliver the functional LAMP2B gene to heart tissue,
which ultimately leads to improved cardiac structure and function in patients.
About Danon Disease
Danon Disease is a rare X-linked inherited disorder caused by mutations in the
gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important
mediator of autophagy. This results in accumulation of autophagosomes and
glycogen, particularly in cardiac muscle and other tissues, which ultimately
leads to heart failure, and for male patients, frequent death during
adolescence or early adulthood. It is estimated to have a prevalence of 15,000
to 30,000 patients in the U.S. and Europe. The only available treatment option
for Danon Disease is cardiac transplantation, which is associated with
substantial complications and is not considered curative. There is a high
unmet medical need for patients with Danon Disease.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and
sustainable pipeline of investigational genetic therapies designed to 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. Rocket also has
received IND clearance for the AAV-based gene therapy program for
PKP2-arrhythmogenic cardiomyopathy (ACM) and is advancing a preclinical
program for BAG3-associated dilated cardiomyopathy (DCM). For more information
about Rocket, please visit www.rocketpharma.com (http://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 the safety and effectiveness of product candidates that Rocket is
developing to treat Danon Disease (DD), the expected timing and data readouts
of Rocket's ongoing and planned clinical trials, the expected timing and
outcome of Rocket's regulatory interactions and planned submissions, Rocket's
plans for the advancement of its Danon Disease program, including its planned
pivotal trial, 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, 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, our ability to submit regulatory filings with the U.S. Food and
Drug Administration (FDA) and to obtain and maintain FDA or other regulatory
authority approval of our product candidates, Rocket's dependence on third
parties for development, manufacture, marketing, sales and distribution of
product candidates, the outcome of litigation, our competitors' activities,
including decisions as to the timing of competing product launches, pricing
and discounting, our integration of an acquired business, which involves a
number of risks, including the possibility that the integration process could
result in the loss of key employees, the disruption of our ongoing business,
or inconsistencies in standards, controls, procedures, or policies, our
ability to successfully develop and commercialize any technology that we may
in-license or products we may acquire and any 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, 2022,
filed February 28, 2023 with the SEC and subsequent filings with the SEC
including our Quarterly Reports on Form 10-Q. 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.
View source version on businesswire.com:
https://www.businesswire.com/news/home/20230912416941/en/
(https://www.businesswire.com/news/home/20230912416941/en/)
Media
Kevin Giordano
kgiordano@rocketpharma.com
Investors
Brooks Rahmer
investors@rocketpharma.com
Source: Rocket Pharmaceuticals, Inc
Rocket Pharmaceuticals Announces Pricing of Public Offering of Common Stock
Sep 13, 2023 |
CRANBURY, N.J.--(BUSINESS WIRE)--Sep. 13, 2023-- Rocket Pharmaceuticals, Inc.
(NASDAQ: RCKT), a leading late-stage biotechnology company advancing an
integrated and sustainable pipeline of investigational genetic therapies for
rare childhood disorders with high unmet need, announced today the pricing of
an underwritten public offering of (i) 7,812,500 shares of its common stock at
a public offering price of $16.00 per share and (ii) to certain investors,
pre-funded warrants to purchase 3,126,955 shares of common stock at a price of
$15.99 per pre-funded warrant, which represents the per share public offering
price for the common stock less the $0.01 per share exercise price for each
such pre-funded warrant. The gross proceeds to Rocket from the offering,
before deducting underwriting discounts and commissions and offering expenses,
are expected to be approximately $175 million. All shares and pre-funded
warrants in the offering are to be sold by Rocket. In addition, Rocket has
granted the underwriters a 30-day option to purchase up to an additional
1,640,918 shares of its common stock. The offering is expected to close on or
about September 15, 2023, subject to customary closing conditions.
J.P. Morgan, Morgan Stanley, Leerink Partners and TD Cowen are acting as joint
book-running managers, and LifeSci Capital is acting as lead manager for the
offering.
The shares and pre-funded warrants are being offered by Rocket pursuant to an
effective shelf registration statement that was previously filed with the U.S.
Securities and Exchange Commission (the "SEC"). The offering is being made
only by means of a written prospectus and prospectus supplement that form a
part of the registration statement. A preliminary prospectus supplement
relating to and describing the terms of the offering was filed with the SEC
and is available on the SEC's website at www.sec.gov
(https://cts.businesswire.com/ct/CT?id=smartlink&url=http%3A%2F%2Fwww.sec.gov&esheet=53553709&newsitemid=20230912393985&lan=en-US&anchor=www.sec.gov&index=1&md5=f38a1e752441f80211d5cb87adad9d95)
.
When available, copies of the final prospectus supplement relating to the
offering may be obtained from J.P. Morgan Securities LLC, c/o Broadridge
Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, by
telephone at (866) 803-9204, or by email at prospectus-eqfi@jpmorganchase.com
(mailto:prospectus-eqfi@jpmorganchase.com) ; Morgan Stanley & Co. LLC, 180
Varick Street, 2nd Floor, New York, NY 10014, by telephone at (866) 718-1649
or by email at Prospectus@morganstanley.com
(mailto:Prospectus@morganstanley.com) ; Leerink Partners LLC, Syndicate
Department, 53 State Street, 40th Floor, Boston, MA 02109, or by telephone at
(800) 808-7525 ext. 6105 or by email at syndicate@leerink.com
(mailto:syndicate@leerink.com) ; or Cowen and Company, LLC, 599 Lexington
Avenue, New York, NY 10022, by telephone at (833) 297-2926 or by email at
Prospectus_ECM@cowen.com (mailto:Prospectus_ECM@cowen.com) . You may also
obtain a copy of this document free of charge by visiting the SEC's website at
www.sec.gov
(https://cts.businesswire.com/ct/CT?id=smartlink&url=http%3A%2F%2Fwww.sec.gov&esheet=53553709&newsitemid=20230912393985&lan=en-US&anchor=www.sec.gov&index=2&md5=a3ac8fbec78824e4ac35308402846263)
.
This press release shall not constitute an offer to sell or a solicitation of
an offer to buy these securities, nor shall there be any sale of these
securities in any state or jurisdiction in which such offer, solicitation or
sale would be unlawful prior to the registration or qualification under the
securities laws of any such state or jurisdiction.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and
sustainable pipeline of investigational genetic therapies designed to 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.
Rocket Cautionary Statement Regarding Forward-Looking Statements
Various statements in this release concerning the timing and completion of the
public offering on the anticipated terms or at all may constitute
forward-looking statements for the purposes of the safe harbor provisions
under the Private Securities Litigation Reform Act of 1995, as amended, and
other federal securities laws. All such forward-looking statements are based
on management's current expectations of future events and are subject to a
number of substantial risks and uncertainties, many of which are outside
Rocket's control, that could cause actual results to differ materially and
adversely from those set forth in or implied by such forward-looking
statements. These risks and uncertainties include fluctuations in Rocket's
stock price, changes in market conditions and satisfaction of customary
closing conditions related to the public offering, as well as those risks more
fully discussed in the section entitled "Risk Factors" in the prospectus
supplement and registration statement referenced above, Rocket's Annual Report
on Form 10-K for the year ended December 31, 2022, filed February 28, 2023
with the SEC and subsequent filings with the SEC including our Quarterly
Reports on Form 10-Q and Current Reports on Form 8-K. There can be no
assurance that Rocket will be able to complete the public offering on the
anticipated terms. 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, unless required by law.
View source version on businesswire.com (http://businesswire.com/) :
https://www.businesswire.com/news/home/20230912393985/en/
(https://www.businesswire.com/news/home/20230912393985/en/)
Media
Kevin Giordano
kgiordano@rocketpharma.com (mailto:kgiordano@rocketpharma.com)
Investors
Brooks Rahmer
investors@rocketpharma.com (mailto:investors@rocketpharma.com)
Source: Rocket Pharmaceuticals, Inc.
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