TIDMRTW
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
Joanna Duquemin Nicolle, Chief Executive Officer
Sadie Morrison, Managing Director +44 (0) 14 8181 0100
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 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.
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/
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 .
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 ; 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 ; 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 ; 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 . You may also obtain a copy of
this document free of charge by visiting the SEC's website at
www.sec.gov .
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 :
https://www.businesswire.com/news/home/20230912393985/en/
Media
Kevin Giordano
kgiordano@rocketpharma.com
Investors
Brooks Rahmer
investors@rocketpharma.com
Source: Rocket Pharmaceuticals, Inc.
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