Business Wire

Ipsen to Present New Insights at ASBMR for Potential Treatment of Ultra-rare Disease Fibrodysplasia Ossificans Progressiva (FOP), Including Global Phase III MOVE Trial Results

Share

Regulatory News:

Ipsen (Euronext: IPN: ADR: IPSEY) today announced that it will share data from the company’s growing Rare Diseases Therapeutic Area portfolio, with seven presentations at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting (September 11-15, 2020). These include the oral presentation of Ipsen’s MOVE trial (during the Novel Therapies for Rare Bone Disease session on Saturday, 12 September [11:00am - 12:15pm ET]), the first and only multicenter Phase III study in fibrodysplasia ossificans progressiva (FOP). The data will be presented by Dr Robert Pignolo, Division of Geriatric Medicine and Gerontology, Department of Internal Medicine, Mayo Clinic, and describes the trial outcomes of the oral investigational therapy palovarotene in reducing new heterotopic ossification (HO) volume in 107 pediatric and adult patients with FOP.1 The MOVE efficacy results were compared with data from untreated patients from Ipsen’s Natural History Study (NHS).2 Safety outcomes from the MOVE trial will also be presented.

“Our passion and commitment to understanding rare diseases has driven this research forward and we’re proud to present these data at the ASBMR 2020 Annual Meeting,” said Howard Mayer, M.D., Executive Vice President and Head of Research and Development at Ipsen. “Collaboration is critical with rare disease research and development and we look forward to continuing to work with key thought leaders, clinicians, the patient advocacy community, and regulatory authorities as we build on our research and develop potential therapeutic agents in rare diseases, including for patients with FOP.”

Additionally, four posters reporting data from the NHS will be presented. The NHS is the largest FOP study of this kind worldwide and is the first global, multicenter, longitudinal study designed to measure disease progression over three years.2 Findings from the NHS reinforce that measuring HO is a viable way to monitor changes in FOP and to assess a potential treatment effect over this time period.

Follow Ipsen on Twitter via @IpsenGroup and keep up to date with ASBMR 2020 conference news and updates by using the hashtag #ASBMR2020.

Overview of all Ipsen presentations at ASBMR 2020 Annual Meeting:

Medicine/disease

Abstract number/title

Study authors

Palovarotene/FOP

Palovarotene (PVO) for Fibrodysplasia Ossificans Progressiva (FOP): Data from the Phase III MOVE Trial

Robert J. Pignolo, Mona Al Mukaddam, Geneviève Baujat, Staffan K. Berglund, Angela M. Cheung, Carmen De Cunto, Patricia Delai, Maja Di Rocco, Nobuhiko Haga, Edward C. Hsiao, Peter Kannu, Richard Keen, Edna E. Mancilla, Donna R. Grogan, Rose Marino, Andrew Strahs, Frederick S. Kaplan

Palovarotene/FOP

Measuring outcomes in ultra-rare bone diseases: Methodology of the palovarotene fibrodysplasia ossificans progressiva (FOP) clinical development program

Robert J. Pignolo, Geneviève Baujat, Matthew A. Brown, Carmen De Cunto, Maja Di Rocco, Edward C. Hsiao, Richard Keen, Mona Al Mukaddam, Andrew Strahs, Donna R. Grogan, Rose Marino, Frederick S. Kaplan

FOP

Medications used by individuals with Fibrodysplasia Ossificans Progressiva (FOP): Data from a global natural history study

Richard Keen, Mona Al Mukaddam, Geneviève Baujat, Carmen De Cunto, Edward C. Hsiao, Robert J. Pignolo, Kathleen Harnett, Rose Marino, Frederick S. Kaplan

FOP

Longitudinal and flare-up-specific biomarkers in fibrodysplasia ossificans progressiva (FOP): Data from a global natural history study

Robert J. Pignolo, Mona Al Mukaddam, Geneviève Baujat, Carmen De Cunto, Edward C. Hsiao, Richard Keen, Kathleen Harnett, Rose Marino, Frederick S. Kaplan

FOP

Use of assistive devices and adaptations by individuals with fibrodysplasia ossificans progressiva (FOP): Data from a global natural history study

Edward C. Hsiao, Mona Al Mukaddam, Geneviève Baujat, Carmen De Cunto, Richard Keen, Robert J. Pignolo, Kathleen Harnett, Rose Marino, Frederick S. Kaplan

FOP

A global natural history study of fibrodysplasia ossificans progressiva (FOP): 12-month outcomes

Mona Al Mukaddam, Robert J. Pignolo, Geneviève Baujat, Matthew A. Brown, Carmen De Cunto, Maja Di Rocco, Edward C. Hsiao, Richard Keen, Kim-Hanh Le Quan Sang, Andrew Strahs, Rose Marino, Frederick S. Kaplan

FOP

Validity and reliability of the fibrodysplasia ossificans progressiva physical function questionnaire (FOP-PFQ), a patient-reported, disease-specific measure

Robert J. Pignolo, Miriam Kimel, John Whalen, Ariane Kawata, Dennis Revicki, Rose Marino, Frederick S. Kaplan

About palovarotene

Palovarotene is an oral investigational, selective RARγ agonist being developed as a potential treatment for patients with the debilitating ultra-rare, genetic disorder fibrodysplasia ossificans progressiva (FOP). Palovarotene, which had rare pediatric disease and breakthrough therapy designations for the treatment of FOP, was acquired by Ipsen through the acquisition of Clementia Pharmaceuticals in April 2019.

About fibrodysplasia ossificans progressiva (FOP)

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, genetic disorder characterized by bone that forms outside the normal skeleton, in muscles, tendons or soft tissue.3 FOP is among the rarest of human diseases, and while there are approximately 1,000 described cases globally, the reported prevalence of FOP is estimated at approximately 1.36 per million individuals.4,5

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. Ipsen also has a well-established Consumer Healthcare business. With total sales over €2.5 billion in 2019, 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’s Forward Looking Statement

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, and the outcome of this study or other studies. 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 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 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 6 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 2019 Universal Registration Document available on its website (www.ipsen.com).

References

  1. Pignolo R et al. Palovarotene (PVO) for fibrodysplasia ossificans progressiva (FOP): Data from the phase III MOVE trial. ASBMR September 2020.
  2. Al Mukaddam M et al. A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP): 12-Month Outcomes. J Endocr Soc. 2020;4 (Supplement 1):OR29-05
  3. The Medical Management of Fibrodysplasia Ossificans Progressiva: Current Treatment Considerations, IFOPA. Accessed: May 2020. Available: http://fundacionfop.org.ar/wp-content/uploads/2019/05/GUIDELINES-May-2019.pdf
  4. Lilijesthrom M & Bogard B. The Global Known FOP Population. Presented at the FOP Drug Development Forum. Boston, MA; 2016.
  5. Baujat et al. Prevalence of fibrodysplasia ossificans progressiva (FOP) in France: an estimate based on a record linkage of two national databases. Orphanet Journal of Rare Diseases. 2017; 12:123.

Contact information

Media
Jess Smith
Senior Director, Global Franchise Communications
+44 (0) 7557 267634
jess.smith@ipsen.com

Maryann Quinn
Director, Product Communications
North America
1-857-529-1151
Maryann.quinn@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

About Business Wire

Business Wire
Business Wire



Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Swiss Medical Group Holding Acquires CoreMedica Europe24.9.2020 17:54:00 CESTPress release

Swiss Medical Group (SMG) Holding Luxembourg S.A., leading the development of STARKS Age-Management in Europe and Asia, announced today the acquisition of CoreMedica Europe, to expand in Europe and the rest of the world. CoreMedica Europe recently made the headlines in Switzerland for combining a COVID-19 Screening Test and Immunity profile with its existing home self-collection kit that allows customers to collect a few drops of blood and send them by mail to CoreMedica Europe’saccredited laboratory in Geneva to screen the anti-SARS-COV-2 IgG antibody and assess any previous infection to SARS-COV-2, the virus responsible for COVID disease, and analyze a set of 5 key biomarkers known to support the immune system (vitamin D, zinc, magnesium, copper, selenium) so as to receive personalized actionable recommendations to help patients build up their natural defenses against the effects of a next potential viral infection. “In this epidemic and containment context the essential objective o

Esri and AfroChampions Launch Partnership to Promote GIS in Africa24.9.2020 15:52:00 CESTPress release

Esri, the global leader in location intelligence, today announced a joint initiative with AfroChampions, a Pan-African nonprofit that aims to promote policies that foster private-public collaboration for Africa's economic transformation. The goal of the initiative is to engage leaders in business, governments, the African Union, and other regional economic communities through dialogue and potential partnership building in applying geospatial technology and solutions. This new partnership with AfroChampions seeks to contribute to sustainable economic development in Africa and promote the benefits of a shared geospatial infrastructure throughout the continent. As a majority of Africans still live in rural areas, geographic information system (GIS) technology can create new opportunities for growth, especially in critical fields such as health and telemedicine, land management, agriculture, and mobility. The powerful mapping and data analytics that GIS provides forms a foundation for some

DXC Technology Announces Leadership Appointments to Support the “new DXC"24.9.2020 15:20:00 CESTPress release

DXC Technology (NYSE:DXC) today presented its latest leadership appointments further strengthening the DXC leadership team, the majority being new to DXC within the last year. This team is bringing the “new DXC,” which is focused on its customers and its people, to the market as it executes the company’s transformation journey. DXC Customer Portfolio Leadership Over the past year, CEO Mike Salvino has recruited new IT services industry leaders who bring formidable experience delivering for customers, inspiring people, and running and growing businesses. DXC announces the following appointments and changes to the team that will run DXC’s business and lead execution of the DXC transformation journey: Americas will be led byJim Brady and David Swift. Jim joined DXC in June 2020 from Accumen, where he was the COO and previously worked at Accenture and Honeywell. David Swift joined DXC in November 2019 from Accolade where he was Chief Service Officer and previously worked at Aon Hewitt and

Statement on One-Year Anniversary of Women’s Entrepreneurship Accelerator24.9.2020 15:07:00 CESTPress release

Please find the statement below from Women’s Entrepreneurship Accelerator leadership regarding the one-year anniversary of the ground-breaking initiative. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200924005283/en/ Anita Bhatia, Deputy Executive Director, UN Women One year ago, we committed to educating and empowering ten million women entrepreneurs over the next ten years through the Women’s Entrepreneurship Accelerator (WEA). We began to lay the groundwork for a systemic transformation to break down the barriers keeping women from achieving financial independence and contributing to a better future for themselves and their communities. Today, in our unprecedented reality, women find themselves at history’s crossroads. They are disproportionately harmed by the economic devastation wrought by COVID-19 while simultaneously holding the keys to recovery. Women make up 39% of global employment but have accounted for 54% of o

Airship Acquires Conversational Commerce Pioneer ReplyBuy to Accelerate Expansion Into Mobile Commerce24.9.2020 15:00:00 CESTPress release

Customer engagement company Airship today announced that it has acquired ReplyBuy, a company that pioneered fully integrated solutions for payments over SMS. This move extends Airship’s platform beyond customer engagement to directly enable commerce, as well as power one-to-one customer conversations via two-way SMS. In addition, Airship plans to extend ReplyBuy’s technology to provide turnkey commerce and payments solutions across more marketing channels, messaging platforms and industry verticals. The acquisition will provide companies of all sizes a single platform to create, manage and optimize cross-channel customer interactions and drive greater revenue more quickly. Airship’s real-time mobile data, predictive AI and unified approach to customer journeys ensures relevant messaging at the optimal time on customers’ preferred channels across app and web notifications, SMS, email, mobile wallet and more. Subsequent digital actions customers take, now including purchases and conversa

Careismatic Brands Launches “19 Days of Gratitude” Global Campaign to Support Nurses and the DAISY Foundation24.9.2020 15:00:00 CESTPress release

Careismatic Brands, the world’s largest designer and manufacturer of medical apparel, today announced the launch of a global tribute and charitable fundraising campaign, “19 Days of Gratitude,” spotlighting the heroism of nurses as they continue to battle COVID-19 on the front lines. Launching in a year that the World Health Organization has deemed “The International Year of The Nurse and Midwife,” and timed to the 200th anniversary of Florence Nightingale’s birth, the new campaign will celebrate nurses by revealing 19 stories that showcase frontline nurses from around the world over 19 consecutive days. The campaign will debut on careismatic.com/TributeToCourage and across Careismatic’s various social and digital channels, to celebrate the nursing community and raise money for the DAISY (Diseases Attacking the Immune System) Foundation, a worldwide organization recognizing the incredible work and sacrifice of nurses. This press release features multimedia. View the full release here:

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom