Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the
“Company”), a clinical-stage biopharmaceutical company focused on
rare diseases, today provided an update on its pipeline programs as
well as an update on recent corporate developments.
“2023 was a year of tremendous progress for Mereo. Key
milestones in the development of setrusumab for the treatment of OI
included positive data from the Phase 2 portion of the Orbit study,
and initiation of the Phase 3 portion of the Orbit study and Phase
3 Cosmic study by our partner, Ultragenyx,” said Dr. Denise
Scots-Knight, Chief Executive Officer of Mereo. “In addition, we
significantly advanced the development of alvelestat, gaining
valuable clarity on the regulatory path with both the FDA and EMA.
If the proposed Phase 3 study is successful, it could support
submissions for full regulatory approvals for this first-in-class
therapy addressing a major unmet medical need in Alpha-1
Antitrypsin Deficiency-associated Lung Disease. These developments
are expected to further support our ongoing partnering activities
for alvelestat. We look forward to providing further updates on
both setrusumab and alvelestat during the remainder of the year.
With a cash runway into 2026 and several potential important value
inflection points on the horizon in 2024, we believe that Mereo
remains well positioned for long-term growth and success.”
Recent Pipeline Progress
Setrusumab (UX143)
Enrollment in the Phase 3 portion of the Global Phase 2/3 Orbit
study led by Mereo’s partner, Ultragenyx Pharmaceutical Inc.
(NASDAQ: RARE), was initiated in mid-2023 and the study is expected
to be fully enrolled around the end of the first quarter of 2024.
The pivotal Phase 3 portion of the Orbit study will include up to
195 patients aged 5 to <26 years randomized 2:1 to receive
setrusumab or placebo, with a primary efficacy endpoint of
annualized clinical fracture rate excluding fingers, toes, skull
and face. Additionally, Cosmic, a Global Phase 3 open-label,
randomized, active-controlled study in approximately 65 patients
aged 2 to <7 years evaluating setrusumab compared to intravenous
bisphosphonates on reduction in total fracture rate, including
morphometric vertebral fractures, was initiated in the second half
of 2023 and is anticipated to be fully enrolled in the first half
of 2024.
Recent data reported in October 2023, from the 24 patients
enrolled in the Phase 2 portion of the Orbit study with at least 6
months of treatment, showed that treatment with setrusumab reduced
the annualized fracture rate by 67%. This reduction was associated
with continuing large and meaningful improvements in bone mineral
density (BMD). Setrusumab was well tolerated with no drug related
serious adverse events (SAE’s) reported and no reports of
drug-related hypersensitivity. Additional data from the Phase 2
portion of the Orbit study are expected during 2024.
If approved by the U.S. Food and Drug Administration (FDA) and
the European Medicines Agency (EMA), setrusumab would be the first
FDA and EMA-approved treatment for Osteogenesis Imperfecta
(OI).
The Company has received guidance from the EMA and European
Health Technology Assessment (HTA) agencies on its approach to the
design and delivery of a post-Marketing Authorization Evidence
Generation Plan (“SATURN”) and continues to interact with key
decision-makers at European and national levels in Mereo’s
commercial territory to prepare the markets should setrusumab
receive regulatory approval.
Alvelestat (MPH-966)
The Company has continued its interactions with the Division of
Pulmonology, Allergy and Critical Care (DPACC) and the Division of
Clinical Outcome Assessment (DCOA) of the FDA regarding the use of
a Patient Reported Outcome (PRO) instrument as a potential primary
endpoint in its planned Phase 3 study of alvelestat for the
treatment of Alpha-1 Antitrypsin Deficiency-associated Lung Disease
(AATD-LD). These interactions followed an End-of-Phase 2 meeting
for the Company’s ASTRAEUS study in the first quarter of 2023, at
which the FDA and the Company discussed the potential use of the St
George’s Respiratory Questionnaire (SGRQ) Total Score and/or SGRQ
Activity domain as the primary endpoint in a Phase 3 study which,
if successful, could support submissions for full approval in the
U.S.
In the third quarter of 2023, the Company held an additional
meeting with the FDA (DPACC and the DCOA) and analyzed additional
data from the investigator-led Phase 2 ATALANTa study, which was
funded by the National Center for Advancing Translational Sciences
(NCATS) through the National Institutes of Health (NIH)-Industry
Program for discovering new therapeutic uses for existing
molecules, and led by Professor Mark Dransfield from the University
of Alabama at Birmingham.
Data from both the ATALANTa and ASTRAEUS studies support the use
of the SGRQ Total Score as the primary endpoint in the proposed
Phase 3 study based on benefits observed in patients with the
severe Pi*ZZ phenotype, including those who are in the early stages
of the lung disease. In some countries, these patients are
currently not eligible for, or not treated with, augmentation
therapy. Following additional FDA interactions in the fourth
quarter of 2023, subsequent to the DCOA meeting, the Company has
aligned on a Phase 3 study design using SGRQ Total Score as the
primary endpoint with a functional assessment as a key secondary
endpoint which, if the study is successful, is expected to support
submissions for full regulatory approval in the U.S. Inclusion of
patients with earlier and later stage lung disease progression in
the planned registrational study could increase the addressable
patient population for alvelestat.
In Europe, the Company has received guidance from the EMA on the
primary endpoint for its Phase 3 clinical trial of alvelestat for
the treatment of AATD-LD. The EMA has indicated that lung density
by computed tomography (CT) scan with a relaxed p value (p<0.1)
may be sufficient for full regulatory approval if the study is
successful.
The Company remains engaged with multiple potential partners for
the development and potential commercialization of alvelestat and
expects to provide further details on these efforts in 2024.
Leflutrozole
The Company recently entered into an exclusive global license
agreement with ReproNovo SA (the “License Agreement”) for the
development and commercialization of leflutrozole, a non-steroidal
aromatase inhibitor. Under the terms of the License Agreement,
ReproNovo, a reproductive medicine company, is responsible for all
future development and commercialization of leflutrozole. Mereo
received an upfront payment and will be eligible to receive up to
$64.25 million in future clinical, regulatory and commercial
milestones as well as tiered mid-single digit royalties on global
annual net sales of leflutrozole.
Cash Runway Guidance and Financial Reporting
Update
The Company continues to expect its existing cash and short-term
deposits, excluding income from existing or potential partnerships,
will enable funding of its currently committed clinical trials,
operating expenses and capital expenditure requirements into
2026.
Effective January 1, 2024, the Company began complying with and
reporting under the SEC rules and Nasdaq listing requirements
applicable to U.S. domestic filers.
About Mereo BioPharma
Mereo BioPharma is a biopharmaceutical company focused on the
development of innovative therapeutics for rare diseases. The
Company has two rare disease product candidates, setrusumab for the
treatment of Osteogenesis Imperfecta (OI) and alvelestat primarily
for the treatment of severe Alpha-1 Antitrypsin
Deficiency-associated Lung Disease (AATD-LD). The Company’s
partner, Ultragenyx Pharmaceutical Inc., initiated a pivotal Phase
2/3 pediatric study in young adults (5 to <26 years old) for
setrusumab in OI and a Phase 3 study in pediatric patients (2 to
<7 years old) in mid-2023. The partnership with Ultragenyx
includes potential milestone payments of up to $245 million
(following the recent $9 million milestone) and royalties to Mereo
on commercial sales in Ultragenyx territories. Mereo has retained
EU and UK commercial rights and will pay Ultragenyx royalties on
commercial sales in those territories. Setrusumab has received
orphan designation for OI from the EMA and FDA, PRIME designation
from the EMA and has pediatric disease designation from the FDA.
Alvelestat has received U.S. Orphan Drug Designation for the
treatment of AATD, Fast Track designation from the FDA, and two
Phase 2 proof-of-concept studies have been completed in North
America and Europe. In addition to the rare disease programs, Mereo
has two oncology product candidates in clinical development.
Etigilimab (anti-TIGIT) has completed a Phase 1b/2 basket study
evaluating its safety and efficacy in combination with an anti-PD-1
in a range of tumor types including three rare tumors and three
gynecological carcinomas - cervical, ovarian, and endometrial and
is in an ongoing Phase 1b/2 investigator led study at the MD
Anderson Cancer Center in clear cell ovarian cancer; navicixizumab,
for the treatment of late line ovarian cancer, has completed a
Phase 1 study and has been partnered with OncXerna Therapeutics,
Inc. in a global licensing agreement that includes payments of up
to $300 million in milestones and royalties. Mereo has entered into
an exclusive global license agreement with ReproNovo SA for the
development and commercialization of leflutrozole, a non-steroidal
aromatase inhibitor. Under the terms of the agreement, ReproNovo, a
reproductive medicine company, is responsible for all future
development and commercialization of leflutrozole.
For more information on Mereo BioPharma, please visit
www.mereobiopharma.com.
Forward-Looking Statements
This press release contains “forward-looking statements” that
involve substantial risks and uncertainties. All statements other
than statements of historical fact contained herein are
forward-looking statements within the meaning of Section 27A of the
United States Securities Act of 1933, as amended, and Section 21E
of the United States Securities Exchange Act of 1934, as amended.
Forward-looking statements usually relate to future events and
anticipated revenues, earnings, cash flows or other aspects of our
operations or operating results. Forward-looking statements are
often identified by the words “believe,” “expect,” “anticipate,”
“plan,” “intend,” “foresee,” “should,” “would,” “could,” “may,”
“estimate,” “outlook” and similar expressions, including the
negative thereof. The absence of these words, however, does not
mean that the statements are not forward-looking. These
forward-looking statements are based on the Company’s current
expectations, beliefs and assumptions concerning future
developments and business conditions and their potential effect on
the Company. While management believes that these forward-looking
statements are reasonable as and when made, there can be no
assurance that future developments affecting the Company will be
those that it anticipates.
All of the Company’s forward-looking statements involve known
and unknown risks and uncertainties some of which are significant
or beyond its control and assumptions that could cause actual
results to differ materially from the Company’s historical
experience and its present expectations or projections. Such risks
and uncertainties include, among others, the uncertainties inherent
in the clinical development process; the Company’s reliance on
third parties to conduct and provide funding for its clinical
trials; the Company’s dependence on enrollment of patients in its
clinical trials; and the Company’s dependence on its key
executives. You should carefully consider the foregoing factors and
the other risks and uncertainties that affect the Company’s
business, including those described in the “Risk Factors” section
of its latest Annual Report on Form 20-F, as well as discussions of
potential risks, uncertainties, and other important factors in the
Company’s subsequent filings with the Securities and Exchange
Commission. The Company wishes to caution you not to place undue
reliance on any forward-looking statements, which speak only as of
the date hereof. The Company undertakes no obligation to publicly
update or revise any of our forward-looking statements after the
date they are made, whether as a result of new information, future
events or otherwise, except to the extent required by law.
Mereo BioPharma Contacts: |
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Mereo |
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+44 (0)333 023
7300 |
Denise Scots-Knight, Chief
Executive Officer |
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Christine Fox, Chief Financial
Officer |
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Burns McClellan
(Investor Relations Adviser to Mereo) |
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+01 646 930
4406 |
Lee Roth |
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Investors |
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investors@mereobiopharma.com |
Ultragenyx Pharmaceutical (NASDAQ:RARE)
過去 株価チャート
から 5 2024 まで 6 2024
Ultragenyx Pharmaceutical (NASDAQ:RARE)
過去 株価チャート
から 6 2023 まで 6 2024