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Ipsen today announced that the pivotal Phase II FALKON trial did not meet its primary endpoint of reducing new heterotopic ossification (HO) in adults and children living with fibrodysplasia ossificans progressiva (FOP) vs. placebo, as a result the study will be closed. The investigational medicinal product (fidrisertib) was generally well tolerated, with no safety concerns in the trial.
“These results are disappointing for the FOP community and patients living with this devastating disease,” said Christelle Huguet, PhD, EVP, Head of Research and Development. “However, we do believe that these data will contribute to the growing body of research on FOP, giving new insights into managing this disease for patients and their care providers. FALKON was a tremendous undertaking by Ipsen, continuing our commitment to FOP. FALKON enrolled 113 patients globally, taking over 5 years to reach this critical milestone. We would like to thank the patients, caregivers, the FOP community and KOLs who dedicated their time to the FALKON trial, it has been a serious commitment to help advance our understanding of FOP.”
About FOP
FOP is a genetic condition caused by pathogenic variants of the ALK2 kinase that leads to bone formation in soft and connective tissues, like muscles, tendons and ligaments, a process known as HO. Once formed, HO is irreversible. There are limited treatment options for patients with FOP. The average/median age of diagnosis is 5 years old and ultimately FOP shortens the life expectancy to a median of 56 years, with untimely death caused by bone formation around the ribcage, leading to breathing problems and thoracic insufficiency. FOP has an estimated prevalence of 1.36 per million individuals and approximately 900 people are diagnosed worldwide; however, the number of confirmed cases varies by country.
About fidrisertib
Fidrisertib is an oral, highly selective and potent small molecule inhibitor of pathogenic variants of the ALK2 kinase, the underlying root cause of FOP. Fidrisertib has been designed to address the unliganded ALK2 signal as well as BMP and aberrant activin liganded signal during flare up; impacting both flare-up based and non-flare-up based HO formation. Fidrisertib is administered orally (capsule sprinkled on food or dissolved in water) without change in dose during flare-ups.
About the FALKON Trial
FALKON is the largest Phase II trial in FOP, comprising 3 parts, designed to evaluate the efficacy, safety and tolerability of fidrisertib, as a first-line treatment in pediatric and adult patients with FOP. Part A is a global, multi-center, placebo-controlled, parallel-group, 3-arm trial, in which 113 patients aged 5 years of age or older with the R206H ACVR1 mutation or other FOP variants associated with progressive HO, were enrolled and randomized to receive either high dose-weight based or low dose weight-based fidrisertib or placebo. The primary endpoint is annualized change from baseline in HO volume. In Part B of the double-blind trial, patients continue their dose of fidrisertib, with the patients on placebo in Part A also randomized to receive either high or low dose fidrisertib. Part C is an extension period for all patients who are responding.
About Ipsen
We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries.
Ipsen (ipsen.com) is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY).
Disclaimers and/or forward-looking statements
The forward-looking statements, objectives and targets contained herein are based on Ipsen’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 Ipsen’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 Ipsen’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 Ipsen. 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 medicine 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. Ipsen must face or might face competition from generic medicine 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 Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical 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 medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine 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 healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen’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 Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen 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 Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen 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. Ipsen’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 Ipsen’s latest Universal Registration Document, available online.
Contacts
Investors
Henry Wheeler; E: henry.wheeler[.]ipsen.com - P: +33(0)7 66 47 11 49
Khalid Deojee; E: khalid.deojee[.]ipsen.com - P: +33(0)6 66 01 95 26
Media
Anne Liontas; E: anne.liontas.ext[.]ipsen.com - P: +33(0)7 67 34 72 96
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