Ipsen’s palovarotene clinical program in fibrodysplasia ossificans progressiva reaches prespecified interim analysis futili...
January 24 2020 - 1:00AM
Business Wire
Ipsen has decided to pause dosing in the
palovarotene trials
Based on encouraging therapeutic activity
signals observed in preliminary post-hoc analyses and
recommendations from the Independent Data Monitoring Committee,
Ipsen to conduct further assessment of the complete data set and
work with the regulatory authorities to determine the path
forward
Regulatory News:
Ipsen (Euronext: IPN; ADR: IPSEY) today announced the decision
to pause dosing patients in the global Phase III (PVO-1A-301) study
designed to evaluate the efficacy and safety of palovarotene in
patients with fibrodysplasia ossificans progressiva (FOP), as well
as the ongoing Phase II (PVO-1A-202/204) extension studies. In both
the Phase III and Phase II extension studies, palovarotene is dosed
both chronically (daily) and episodically (during flare-ups). The
decision to pause dosing patients in the trial is based on results
of a futility analysis reviewed by the Independent Data Monitoring
Committee (IDMC) as part of the prespecified interim analysis. The
results of a futility analysis indicated that the Phase III FOP
trial was unlikely to meet its primary efficacy endpoint
(annualized change in new HO volume as compared with Natural
History Studyi) upon completion.
Despite the results of the prespecified interim analysis,
signals of encouraging therapeutic activity were observed in
preliminary post-hoc analyses of the Phase III trial and shared
with and acknowledged by the IDMC which is recommending not to
discontinue the study. In its recommendations, the IDMC notes
highly disparate results precluding a confident conclusion about
futility. The IDMC also points out that the protocol-prespecified
model may have negatively affected the efficacy analysis and
shifted the statistical conclusion from significant therapeutic
benefit to showing futility of the treatment. The FDA partial
clinical hold for the pediatric population under the age of 14 for
FOP and multiple osteochondromas (MO) issued on 4 December 2019,
remains in effect.
Ipsen will pause dosing patients in the trials and conduct
further assessment of the complete data set. Based on the IDMC’s
observations and recommendations, Ipsen will discuss these findings
with regulatory authorities to determine the path forward for the
palovarotene program in FOP. Ipsen will collaborate and consult
with the patients, investigators, ethics committees and regulatory
authorities to define next steps for the program, in the best
interests of patients, whilst ensuring consent of all parties
involved.
“While the study has met prespecified statistical futility, we
are encouraged by the results observed in the preliminary post-hoc
analyses and look forward to discussing these with regulators as
quickly as possible to determine the next steps for the
palovarotene program,” said Aymeric Le Chatelier, Chief Executive
Officer at Ipsen. “We gratefully acknowledge the ongoing support
and trust from patients, their families and the healthcare
professionals involved in these trials. We are deeply committed to
drug development in the area of rare and ultra-rare diseases where
there are multiple high unmet medical needs and often a limited
understanding of the disease itself.”
Ipsen is currently assessing the financial implications of these
developments, including the financial outlook for 2022, and will
present in February updated views together with Full Year 2019
results.
About palovarotene
Palovarotene is a RARγ agonist being developed as a potential
treatment for patients with ultra-rare and debilitating bone
diseases, including fibrodysplasia ossificans progressiva (FOP) and
multiple osteochondromas (MO), as well as other conditions
including dry eye disease. Palovarotene, which had rare pediatric
disease and breakthrough therapy designations for the treatment of
an ultra-rare bone disorder, was acquired by Ipsen through the
acquisition in April 2019 of Clementia Pharmaceuticals.
About Ipsen
Ipsen is a global specialty-driven biopharmaceutical group
focused on innovation and Specialty Care. The Group develops and
commercializes innovative medicines in three key therapeutic areas:
Oncology, Neuroscience and Rare Diseases. Its commitment to
oncology is exemplified through its growing portfolio of key
therapies for prostate cancer, neuroendocrine tumors, renal cell
carcinoma and pancreatic cancer. Ipsen also has a well-established
Consumer Healthcare business. With total sales over €2.2 billion in
2018, Ipsen sells more than 20 drugs in over 115 countries, with a
direct commercial presence in more than 30 countries. Ipsen’s
R&D is focused on its innovative and differentiated
technological platforms located in the heart of the leading
biotechnological and life sciences hubs (Paris-Saclay, France;
Oxford, UK; Cambridge, US). The Group has about 5,800 employees
worldwide. Ipsen is listed in Paris (Euronext: IPN) and in the
United States through a Sponsored Level I American Depositary
Receipt program (ADR: IPSEY). For more information on Ipsen, visit
www.ipsen.com.
Ipsen—Cautionary Note Regarding Forward-Looking
Statements
The forward-looking statements, objectives and targets contained
herein are based on the Group’s management strategy, current views
and assumptions. Such statements involve known and unknown risks
and uncertainties that may cause actual results, performance or
events to differ materially from those anticipated herein. All of
the above risks could affect the Group’s future ability to achieve
its financial targets, which were set assuming reasonable
macroeconomic conditions based on the information available today.
Use of the words "believes", "anticipates" and "expects" and
similar expressions are intended to identify forward-looking
statements, including the Group’s expectations regarding future
events, including regulatory filings and determinations. Moreover,
the targets described in this document were prepared without taking
into account external growth assumptions and potential future
acquisitions, which may alter these parameters. These objectives
are based on data and assumptions regarded as reasonable by the
Group. These targets depend on conditions or facts likely to happen
in the future, and not exclusively on historical data. Actual
results may depart significantly from these targets given the
occurrence of certain risks and uncertainties, notably the fact
that a promising product in early development phase or clinical
trial may end up never being launched on the market or reaching its
commercial targets, notably for regulatory or competition reasons.
The Group must face or might face competition from generic products
that might translate into a loss of market share. Furthermore, the
Research and Development process involves several stages each of
which involves the substantial risk that the Group may fail to
achieve its objectives and be forced to abandon its efforts with
regards to a product in which it has invested significant sums.
Therefore, the Group cannot be certain that favorable results
obtained during pre-clinical trials will be confirmed subsequently
during clinical trials, or that the results of clinical trials will
be sufficient to demonstrate the safe and effective nature of the
product concerned. There can be no guarantees a product will
receive the necessary regulatory approvals or that the product will
prove to be commercially successful. If underlying assumptions
prove inaccurate or risks or uncertainties materialize, actual
results may differ materially from those set forth in the
forward-looking statements. Other risks and uncertainties include
but are not limited to, general industry conditions and
competition; general economic factors, including interest rate and
currency exchange rate fluctuations; the impact of pharmaceutical
industry regulation and health care legislation; global trends
toward health care cost containment; technological advances, new
products and patents attained by competitors; challenges inherent
in new product development, including obtaining regulatory
approval; the Group's ability to accurately predict future market
conditions; manufacturing difficulties or delays; financial
instability of international economies and sovereign risk;
dependence on the effectiveness of the Group’s patents and other
protections for innovative products; and the exposure to
litigation, including patent litigation, and/or regulatory actions.
The Group also depends on third parties to develop and market some
of its products which could potentially generate substantial
royalties; these partners could behave in such ways which could
cause damage to the Group’s activities and financial results. The
Group cannot be certain that its partners will fulfil their
obligations. It might be unable to obtain any benefit from those
agreements. A default by any of the Group’s partners could generate
lower revenues than expected. Such situations could have a negative
impact on the Group’s business, financial position or performance.
The Group expressly disclaims any obligation or undertaking to
update or revise any forward-looking statements, targets or
estimates contained in this press release to reflect any change in
events, conditions, assumptions or circumstances on which any such
statements are based, unless so required by applicable law. The
Group’s business is subject to the risk factors outlined in its
registration documents filed with the French Autorité des Marchés
Financiers. The risks and uncertainties set out are not exhaustive
and the reader is advised to refer to the Group’s 2018 Registration
Document available on its website (www.ipsen.com).
i *Prospective Natural History Study (NHS), the first
multi-center, non-interventional, two-part longitudinal study
designed to measure disease progression over three years in
patients with FOP.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200123005883/en/
For further information:
Media Christian Marcoux, M.Sc. Senior Vice President,
Global Communications +33 (0)1 58 33 67 94
christian.marcoux@ipsen.com
David Caruba Director, Global Communications +1 (857) 998-7036
david.caruba@ipsen.com
Financial Community Eugenia Litz Vice President, Investor
Relations +44 (0) 1753 627721 eugenia.litz@ipsen.com
Myriam Koutchinsky Investor Relations Manager +33 (0)1 58 33 51
04 myriam.koutchinsky@ipsen.com
Ipsen (EU:IPN)
Historical Stock Chart
From Apr 2024 to May 2024
Ipsen (EU:IPN)
Historical Stock Chart
From May 2023 to May 2024