Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE), a biopharmaceutical
company focused on the development and commercialization of novel
products for serious rare and ultra-rare diseases, and Mereo
BioPharma Group plc (Nasdaq: MREO, AIM: MPH), a clinical stage
biopharmaceutical company focused on oncology and rare diseases,
today announced a license and collaboration agreement for
setrusumab, a monoclonal antibody in clinical development for
osteogenesis imperfecta (OI). Setrusumab is an investigational
anti-sclerostin fully human monoclonal antibody that has shown the
ability to improve bone production and density leading to greater
bone strength in animal models of OI. Data from a Phase2b of
setrusumab conducted by Mereo demonstrated a dose-dependent
increase in bone formation, density, and strength in adults with
OI.
“Setrusumab is a great complement to Ultragenyx’s product
portfolio and enables us to leverage the broad expertise and
infrastructure we have established in metabolic bone diseases with
Crysvita,” stated Emil D. Kakkis, M.D., Ph.D., Chief Executive
Officer and President of Ultragenyx. “Most importantly, setrusumab
is a promising option for patients with osteogenesis imperfecta,
which is one of the most common genetic bone diseases associated
with frequent bone fractures.”
“Osteogenesis imperfecta is a rare and devastating genetic
disease, with currently no approved therapies. We are proud to
partner with Ultragenyx to continue the development of setrusumab
as potentially the first approved therapy for OI in both children
and adults,” said Dr. Denise Scots-Knight, Chief Executive Officer
of Mereo. “Following the positive data from our Phase 2b ASTEROID
study, we set out to find the right partner and we believe that
Ultragenyx, with its proven track record of successfully developing
and commercializing novel therapies for rare diseases, is ideally
positioned to support the further advancement of our innovative
therapeutic candidate. We look forward to continuing to work
closely with Ultragenyx to make setrusumab available to the OI
community worldwide.”
OI is a group of genetic disorders including types I, III and
IV, of which approximately 85-90% are caused by mutations in the
COL1A1 or COL1A2 genes leading to either a reduced amount of normal
collagen or collagen with abnormal structure and changes in bone
metabolism. Since collagen molecules represent the foundation upon
which bone is formed, these abnormalities lead to increased bone
resorption, reduced bone mass, and bone fragility and weakness.
Although the abnormal or deficient collagen weakens bone, these
collagen abnormalities also set off a maladaptive cascade of bone
remodeling signals that enhance bone resorption, or the breaking
down of bone, with inadequate production of new bone, which
compounds the bone fragility. These genetic defects and their
consequences lead to systemic clinical manifestations such as
decreased bone mass, bone brittleness leading to a high rate of
fractures, including at atypical sites, or bone deformities,
including abnormal spine curvature, as well as pain, decreased
mobility, and short stature. OI affects approximately 60,000
patients in the developed world and has no approved treatments.
Setrusumab is a fully human monoclonal antibody that inhibits
sclerostin, a protein that acts on a key bone-signaling pathway and
inhibits the activity of bone-forming cells. By blocking inhibitory
effects of sclerostin, the anti-sclerostin antibody causes new bone
formation, increased production of collagen, and increased bone
mineral density and strength. Sclerostin inhibition also reduces
excessive bone resorption, further enhancing the impact on bone
density. In various mouse models of OI, the use of anti-sclerostin
antibodies was shown to stimulate bone formation, improve bone mass
and density, reduce bone fragility, increase long bone stiffness
and strength, and reduce the number of fractures. Overall,
improvements in bone mass and strength were enhanced when an
anti-sclerostin antibody was used in combination with
bisphosphonates, the current standard of care in OI.
Mereo has completed the Phase 2b ASTEROID study of setrusumab
across three dose groups monthly for 12 months in 90 adults with OI
types I, III, and IV. Results from the study indicated improvements
in bone mineral density across multiple measures and at multiple
anatomical sites on a dose-dependent basis after 12 months.
Improvements were also observed in serum P1NP (procollagen type I N
propeptide), a biomarker of bone formation and direct measure of
collagen production. The bone mineral density and P1NP results were
consistent across OI types studied. In addition, there was a
dose-dependent improvement in trabecular bone architecture and bone
strength by measuring wrist bone failure load and stiffness. In the
per protocol population at the high dose, there was a trend toward
improvement of ankle failure load and a statistically significant
improvement in ankle bone stiffness. While the study was not
powered to show a difference in fracture rates, there was a trend
toward a reduction in fractures in the highest dose relative to the
lower doses. In the study, setrusumab was generally well tolerated
with no cardiac-related safety concerns observed.
The companies will expand and initially prioritize the
development of setrusumab for pediatric patients with OI.
Development plans are being finalized which may include changes to
current study designs, and will require discussions with regulatory
agencies, for a pediatric Phase 2/3 study that first focuses on
determining the optimal dose based on increases in collagen
production using serum P1NP levels and an acceptable safety
profile. Following determination of the dose, the study is intended
to adapt into a pivotal Phase 3 stage, evaluating fracture
reduction over an estimated 15 to 24 months as the primary endpoint
pending regulatory review. The pediatric Phase 2/3 study is
expected to start in 2021. A separate pivotal study is also being
planned for adults with OI.
Setrusumab has received orphan drug designation from the U.S.
Food and Drug Administration (FDA) and European Medicines Agency
(EMA), rare pediatric disease designation from the FDA, and was
accepted into the EMA’s Priority Medicines program (PRIME).
Under the terms of the collaboration, Ultragenyx will lead
future global development of setrusumab in both pediatric and adult
patients. Mereo granted Ultragenyx an exclusive license to develop
and commercialize setrusumab in the US and rest of the world,
excluding Europe where Mereo retains commercial rights. Each party
will be responsible for post-marketing commitments in their
respective territories.
Ultragenyx will make an upfront payment of $50 million to Mereo
and will fund global development of the program until approval, and
has agreed to pay a total of up to $254 million upon achievement of
certain clinical, regulatory, and commercial milestones. Ultragenyx
will pay tiered double digit percentage royalties to Mereo on net
sales outside of Europe, and Mereo will pay a fixed double digit
percentage royalty to Ultragenyx on net sales in Europe. Under the
terms of its 2015 agreement with Novartis, Mereo will pay Novartis
a percentage of proceeds, subject to certain deductions, with Mereo
receiving a substantial majority of the payments from
Ultragenyx.
The completion of the transaction is subject to
Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR) review
and the satisfaction of other customary closing conditions.
About Osteogenesis Imperfecta
Osteogenesis Imperfecta (OI) is a rare genetic disorder that is
characterized by fragile bones and reduced bone mass resulting in
bones that break easily, loose joints, and weakened teeth. In
severe cases, those with OI may experience hundreds of fractures in
a lifetime. In addition, people with OI often suffer muscle
weakness, early hearing loss, fatigue, curved bones, scoliosis,
respiratory problems, and short stature, leading to significant
effects on overall health and quality of life. The majority of
cases of OI (estimated at approximately 90%) are caused by a
dominant mutation in a gene coding for type I collagen, a key
component of healthy bone. Current treatment of OI is supportive,
focusing on minimizing fractures and maximizing mobility, but to
date, there are no FDA or EU approved treatments. OI is estimated
to affect between 1 in 6,500 and 1 in 30,000 people globally.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing
novel products to patients for the treatment of serious rare and
ultra-rare genetic diseases. The company has built a diverse
portfolio of approved therapies and product candidates aimed at
addressing diseases with high unmet medical need and clear biology
for treatment, for which there are typically no approved therapies
treating the underlying disease.
The company is led by a management team experienced in the
development and commercialization of rare disease therapeutics.
Ultragenyx’s strategy is predicated upon time- and cost-efficient
drug development, with the goal of delivering safe and effective
therapies to patients with the utmost urgency and ensuring majority
access to its therapies for patients who can benefit.
Ultragenyx’s metabolic bone product portfolio includes Crysvita®
(burosumab), which is approved by the FDA for the treatment of
X-linked hypophosphatemia (XLH) in adult and pediatric patients six
months of age and older and for FGF23-related hypophosphatemia in
tumor-induced osteomalacia (TIO) associated with phosphaturic
mesenchymal tumors that cannot be curatively resected or localized
in adults and pediatric patients 2 years of age and older.
For more information on Ultragenyx, please visit the company’s
website at www.ultragenyx.com.
About Mereo BioPharma
Mereo BioPharma is a biopharmaceutical company focused on the
development and commercialization of innovative therapeutics that
aim to improve outcomes for oncology and rare diseases. Mereo's
lead oncology product candidate, etigilimab (Anti-TIGIT), has
completed a Phase 1a dose escalation clinical trial in patients
with advanced solid tumors and has been evaluated in a Phase 1b
study in combination with nivolumab in select tumor types. The
company recently announced initiation of a Phase 1b/2 study of
etigilimab in combination with an anti-PD-1/PDL-1 in a range of
different tumor types. Mereo's rare disease product portfolio
consists of setrusumab, which has completed a Phase 2b dose-ranging
study in adults with osteogenesis imperfecta (OI), as well as
alvelestat, which is being investigated in a Phase 2
proof-of-concept clinical trial in patients with alpha-1
antitrypsin deficiency (AATD) and in a Phase 1b/2 clinical trial in
COVID-19 respiratory disease.
Ultragenyx Forward-Looking Statements
Except for the historical information contained herein, the
matters set forth in this press release, including statements
related to Ultragenyx's expectations and projections regarding its
future operating results and financial performance, anticipated
cost or expense reductions, the timing, progress and plans for
its clinical programs and clinical studies, future regulatory
interactions, and the components and timing of regulatory
submissions are forward-looking statements within the meaning of
the "safe harbor" provisions of the Private Securities Litigation
Reform Act of 1995. Such forward-looking statements involve
substantial risks and uncertainties that could cause our clinical
development programs, collaboration with third parties, future
results, performance or achievements to differ significantly from
those expressed or implied by the forward-looking statements. Such
risks and uncertainties include, among others, the effects from the
COVID-19 pandemic on the company’s clinical activities, business
and operating results, risks related to reliance on third party
partners to conduct certain activities on the company’s behalf,
uncertainty and potential delays related to clinical drug
development, smaller than anticipated market opportunities for the
company’s products and product candidates, manufacturing risks,
competition from other therapies or products, and other matters
that could affect sufficiency of existing cash, cash equivalents
and short-term investments to fund operations, the company’s future
operating results and financial performance, the timing of clinical
trial activities and reporting results from same, and the
availability or commercial potential of Ultragenyx’s products and
drug candidates. Ultragenyx undertakes no obligation to update or
revise any forward-looking statements. For a further description of
the risks and uncertainties that could cause actual results to
differ from those expressed in these forward-looking statements, as
well as risks relating to the business of Ultragenyx in general,
see Ultragenyx's Quarterly Report on Form 10-Q filed with
the Securities and Exchange Commission on October
27, 2020, and its subsequent periodic reports filed with
the Securities and Exchange Commission.
Mereo BioPharma Forward-Looking StatementsThis
Announcement contains "forward-looking statements." All statements
other than statements of historical fact contained in this
Announcement 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 involve assumptions that could cause
actual results to differ materially from the Company's historical
experience and its present expectations or projections.
These forward-looking statements are subject to risks and
uncertainties, including, among other things, those described in
the Company’s latest Annual Report on Form 20-F, Reports on Form
6-K and other documents filed from time to time by the Company with
the United States Securities and Exchange Commission. The Company
wishes to caution investors 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 forward-looking statements, whether as a result of new
information, future events or otherwise, except to the extent
required by law.
Contacts:
Ultragenyx Joshua Higa(415) 475-6370
Mereo |
+44 (0)333 023 7300 |
Denise Scots-Knight, Chief
Executive Officer |
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N+1 Singer (Nominated
Adviser and Broker to Mereo) |
+44 (0)20 7496
3081 |
Phil Davies |
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Will Goode |
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Burns McClellan (US
Investor Relations Adviser to Mereo) |
+01 212 213
0006 |
Lisa Burns |
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Lee Roth |
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FTI Consulting (UK
Public Relations Adviser to Mereo) |
+44 (0)20 3727
1000 |
Simon Conway |
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Ciara Martin |
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Investors |
investors@mereobiopharma.com |
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