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

• Seven presentations represent Ipsen's largest presence to date at ASBMR, the world's leading scientific organization for bone health research
• Ipsen's data demonstrate potential advances in understanding fibrodysplasia ossificans progressiva (FOP), including key learnings related to study design, natural history, biomarkers, investigational therapeutic options, and disease-specific patient-reported outcome measures

PARIS, FRANCE, 10 September 2020 - 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 2020Annual 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

For further information:

Media Jess Smith Senior Director, Global Franchise Communications [phone] [e-mail] Financial Community Eugenia Litz Vice President, Investor Relations [phone] [e-mail]	Maryann Quinn Director, Product Communications North America 1-857-529-1151 [e-mail] Myriam Koutchinsky Investor Relations Manager [phone] [e-mail]

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.