2022 was a year of focus, momentum and growth at Ipsen. Learn about the progress we made and download the 2022 Integrated Annual Report here.

Diana was a full-time journalist when, at the age of 34, she experienced a stroke that impacted her movement and left her with spasticity.

“I didn’t know if I would ever walk again,” Diana says, “or if I could feed myself.” She suffered from intense pain and had difficulty moving. But despite the long road ahead, she was grateful to be alive.

Today, eight years later, Diana lives a full and busy life. She drives, cooks, does her own makeup and plays games with her family. She credits the love of her husband and children, alongside treatment and therapy, for her incredible progress.

“I discovered that I could be strong and overcome difficulties and even give strength to other people who are going through what I have,” Diana says. “To be able to walk again today, hand-in-hand with my husband, makes me very proud.”

Steve has been diagnosed with kidney cancer twice. After initial success with a nephrectomy, his cancer returned in January 2018. This time, his doctor said there was no treatment available. “He advised I would probably be put on life-extending drugs,” Steve says. “I asked how long they would last, and he said maybe four years.”

Steve was scared to think that he might not be there for his family. He learned about an alternative treatment option and today, he is in long-term remission.

During his illness, Steve found support from groups like Action Kidney Cancer. He now works as group counselor to support others.

“I wouldn’t be here today without the research,” he says. “It gives me the opportunity to be with my children, my wife, my family, as we grow.”

Collette spent almost 20 years going back and forth between different doctors and treatments, before she was finally diagnosed with PBC.

“In those days there was no treatment; there was nothing,” she says. “I was told I had five to seven years or the option of a liver transplant.”

Given the lack of information, she put a listing in a national newspaper to find out if there were others living with PBC. She was inundated with responses from people who suffered personally or who had loved ones living with the disease.

Determined to spread the word, she founded The PBC Foundation, dedicated to providing support and information to those affected by PBC.

“To know that my situation has helped others in their PBC journey, is very precious to me,” she says. “And thanks to receiving treatment, I can now live my life.”

In September 2012, when she was 21, Caitlin received news every child dreads: her father had been diagnosed with pancreatic cancer and had been given three months to live.

Defying the odds, her father’s cancer initially went into remission after surgery. It came back three years later, and Caitlin moved home to help with his care. “Often,” she says, “you’re so focused on your loved one that you don’t think about how it’s impacting you. His health became our number one priority. At the time, I was in survival mode myself.”

The experience taught Caitlin the importance of accepting help as a caregiver. “The best way to care for the person you love is to make sure you’re getting the care you need.” Driven by the desire to help people going through the same challenges, she got involved with advocacy group PanCAN following her father’s passing.

“My dad always said three things make a good day,” Caitlin says. “Help someone, learn something new and get one thing done.”

The rare disease community continues to strive for innovative solutions to meet patient needs and ensure no person living with a rare disease is left behind.  

To mark Rare Disease Day 2023, Yann Le Cam, Chief Executive Officer of EURORDIS and Ipsen’s Jennifer Schranz, Global Head of Rare Disease, Research and Development, connected to discuss EURORDIS’ key focus areas including, shaping rare disease policy in Europe to put patients at the center and prioritizing a holistic patient-centered approach.  

Watch the video below containing an excerpt from this discussion, focusing on driving innovation, the power of early diagnosis, and the importance of ensuring the patient voice is heard to ensure adequate medical and social care is provided.   

This year marks the 17^th anniversary since the discovery of the ACVR1 gene was announced, a major scientific milestone in fibrodysplasia ossificans progressiva (FOP) research that helped us understand what causes the condition. On April 23, we marked FOP Awareness Day to honor this important anniversary, raise awareness of this ultra-rare genetic condition, and celebrate the strength of the FOP community, who face challenges every single day that most of us would find unimaginable. At Ipsen, we recognize the importance of advocating for the unique and complex needs of people living with FOP and although FOP Awareness Day is one very important day, we will never lose sight of the fact that the FOP community deserves our ongoing and long-term commitment every day. In order to succeed in addressing the significant unmet needs that exist, working together with the FOP community is of paramount importance.

We believe it is our responsibility to listen to the first-hand experiences of the FOP community in order to drive positive change. One way we can do this is through studies that truly put those living with FOP at the center of our research, such as burden of illness surveys and natural history studies, which are vital tools to expand our knowledge in rare diseases. Research like this allows us to design clinical trials that explore those aspects of a disease that are the most impactful and relevant to patients, such as disease progression, physical, social, emotional, and quality of life outcomes.

It would be impossible for most of us to understand what someone living with an ultra-rare, chronic, painful and progressive condition like FOP is going through.^1,2 What we do know, however, is that FOP causes ongoing, irreversible bone growth in soft tissues outside of the normal skeletal system, which is known as heterotopic ossification (HO).³ This new bone growth leads to joints becoming immobile, causing progressive loss of movement – many people with FOP need to use a wheelchair by 30 years of age.^2,4 Extra bone growth associated with FOP can also cause the jaw to lock, creating difficulty eating and speaking. Additionally, restrictions in the chest can lead to cardiorespiratory failure and ultimately lead to many people living with FOP to have a reduced life expectancy.⁵

The closest we can get to understanding the realities of living with a rare disease like FOP, is by actively listening to patients about the true impact that it has across every aspect of their lives. By ensuring the patient voice is embedded throughout the end-to-end process of clinical development, including dissemination of findings, the resulting research can be used to bring meaningful change.

Refining rare disease research

Research in rare and ultra-rare diseases brings challenges that are different to those in other disease areas – these include very small patient populations that make it difficult to find individuals who are willing and able to participate in research,⁶ lack of wide-spread disease specific knowledge⁶ and expertise to build upon,^4,7 clinical trial complexity with a lack of known and agreed upon endpoints,⁶ high research and development costs⁸ with a notable failure rate,⁹ and non-existent effective frameworks for assessing value.⁶

To try to break down some of these barriers and better understand the day-to-day realities for people living with FOP, their family members and caregivers, as well as the healthcare professionals (HCPs) who support them, we designed two complementary projects: a prospective, natural history study and a burden of illness survey. Both studies were created in partnership with the FOP community – who not only helped to design them, but who continue to be pivotal as we work to understand and disemminate the insights.

Firstly, the Natural History Study — a first-of-its-kind in FOP. Previously, there was limited research in this area, so the study was designed to more comprehensively describe the natural disease progression and was the first time a global, prospective, longitudinal evaluation of FOP has been carried out, with data collected over a period of 36 months. Findings demonstrated the debilitating impact and progressive nature of FOP, and key insights include:

As a complement to this, we also partnered with FOP community advisors to carry out the first international FOP Burden of Illness (Bol) survey to capture the multifaceted impact of FOP, including the effect on families and caregivers. The Bol survey revealed the extent of the social, economic and quality of life impact of FOP, with findings demonstrating:

Driven by the voices of FOP

At Ipsen, our teams are working everyday to support those in the rare disease community. But the only way we can truly begin to comprehend what life is like for someone living with a rare disease, is by asking them. The Natural History Study and the Bol survey were important tools that allowed us to ask questions and listen to the community, to try to understand their experiences. We would like to personally thank the FOP community who helped make these studies impactful and who have provided opportunities for better understanding and the sharing of these powerful insights.

Only through true understanding can we continue to make the right decisions to support the FOP and rare disease communities.

References

1. Baujat, G et al., 2017, Prevalence of fibrodysplasia ossificans progressiva (FOP) in France: an estimate based on a record linkage of two national databases, Orphanet Journal of Rare Diseases, vol.12, no.1, pp.1-9.

2. Connor JM, Evans DA. Fibrodysplasia ossificans progressiva. The clinical features and natural history of 34 patients. J Bone Joint Surg Br. 1982;64(1):76-83.

3. Kaplan FS, et al. 1993, The Histopathology of Fibrodysplasia Ossificans Progressiva. J Bone Joint Surg Am, Vol 75, no. 2, pp. 220-30.

4. Pignolo, R. et al., 2020. Self-reported baseline phenotypes from the International Fibrodysplasia Ossificans Progressiva (FOP) Association Global Registry. Bone, 134, p.115274.

5. Kaplan FS, Zasloff MA, Kitterman JA, et al. Early mortality and cardiorespiratory failure in patients with fibrodysplasia ossificans progressiva. J Bone Joint Surg Am. 2010;92(3):686–691.

6. Neez E, Gentilini A, Hutchings A.  2021. Addressing unmet needs in extremely rare and paediatric-onset diseases: how the biopharmaceutical innovation model can help identify current issues and find potential solutions.  EFPIA, viewed 28 September 2022, https://dolon.com/rare-knowledge/publications/addressing-unmet-needs-in-extremely-rare-and-paediatric-onset-diseases.

7. Qi, Z., Luan, J., Zhou, X., Cui, Y., & Han, J. 2017. Fibrodysplasia ossificans progressiva: Basic understanding and experimental models. Intractable & rare diseases research, 6(4), 242–248. https://doi.org/10.5582/irdr.2017.01055.

8. Jayasundara K, et al 2019, ‘Estimating the clinical cost of drug development for orphan versus non-orphan drugs.’, Orphanet Journal of Rare Diseases, Vol.14, no. 12.

9. Wong CM, et al 2019, ‘Estimation of clinical trial success rates and related parameters’ Biostatistics, Vol. 20, no. 2, pp. 273–286.

10. Pignolo et al. The Natural History of Fibrodysplasia Ossificans Progressiva: A Prospective, Global, 36-Month Study. Genetics in Medicine. 2022. https://doi.org/10.1016/j.gim.2022.08.013.

11. Mukaddam MA. et al. 2022. The impact of fibrodysplasia ossificans progressive (FOP) on patients and their family members: results from an international burden of illness survey, Expert Review of Pharmacoeconomics & Outcomes Research. DOI: 10.1080/14737167.2022.2115360.

Learn how to engage with our campaign and help protect your liver health. Every year, Liver Cancer Awareness Month (LCAM) is an opportunity to bring the sixth most prevalent cancer globally to the forefront of conversation, promoting awareness and the importance of early diagnosis.¹ Hepatocellular carcinoma (HCC), the most common type of primary liver cancer¹, is projected to become the third leading cause of cancer-related death by 2030, making it an area of urgent and growing need.

This is why, in 2021, we are continuing our partnership with the European Liver Patients Association (ELPA), with the goal of encouraging everyone to ask themselves, “Am I at risk of liver cancer?” Building on the momentum from last year’s campaign, we are excited to announce that Liv the Liver and her family of organs are returning with a new episode, accompanied by activities to highlight the importance of acting upon risk factors and seeking medical advice.

We encourage everyone to Rethink Liver Cancer this month!

References

1. Llovet, J.M. et al. Hepatocellular carcinoma. Nat Rev Dis Primers 7, 6 (2021). https://doi.org/10.1038/s41572-020-00240-3
2. Wang W. and Wei C. Advances in the early diagnosis of hepatocellular carcinoma. Genes & Diseases. 2020. https://doi.org/10.1016/j.gendis.2020.01.014

Hear what Ipsen CEO David Loew has to say about our strong full year and Q4 2022 results.

Ipsen leaders will attend the J.P. Morgan Healthcare Conference from January 9-12, 2023, in San Francisco, California. This event will bring together biotech companies, clinical researchers, investors and analysts from all over the world to learn about healthcare’s latest innovations.

Ipsen has set aside more than €3 billion to invest in partnerships and research, and we look forward to connecting with current and future collaborators at the conference. We are also organizing meetings with investors and analysts.

A winning strategy

Ipsen brings innovative treatments to patients living with hard-to-treat conditions. Our brands hold top positions in more than 100 markets worldwide. We have a clear focus, global footprint and proven expertise in getting products to patients.

Our strategic position yields tangible benefits. Ipsen’s sales were up 9.5% for the first three quarters of 2022, and we enjoy high profitability and strong cashflow. Our approach to partnering and external innovation empowered us to add 16 new assets to our pipeline over the past 18 months. Most recently, we acquired the biotech Epizyme.

Divesting our consumer healthcare business has enabled us to focus on specialty care, and strengthen our brands and pipeline in our three therapeutic areas: oncology, rare disease and neuroscience.

Partnering for patients

Ipsen knows great partnerships bring treatments to patients faster, which is why they are central to our strategy. Our commitment to improving the lives and health of patients guides our external innovation decisions.

Philippe Lopes-Fernandes, EVP, Chief Business Officer, and head of the Global Partnering team will attend the conference. The team will be available in San Francisco to meet with like-minded partners working in Ipsen’s three therapeutic areas. We are interested in assets across all stages of development.

CBO Philippe Lopes-Fernandes will participate in two key panels:

• “Headwinds or Not: Moving Forward Despite the Challenging Environment“, Biotech Showcase: The Investor Conference for Innovators, on January 9, and
• “Firepower M&A 2023: Biopharma“, McDermott and EY at the 2023 J.P. Morgan Healthcare Conference, on January 10.

Invested in progress

This event also brings together some of the market’s top investors and analysts. CEO David Loew, CFO Aymeric Le Chatelier and VP Investor Relations Craig Marks are attending the conference, alongside the partnering team and other representatives. On January 11, David Loew will deliver a presentation on Ipsen’s strategic progress. He will outline our changing business approach, as well as the recent successes our new strategy has brought. His talk will be followed by a Q&A session.

For more information on Ipsen, please visit our page for investors.

We look forward to meeting with our industry’s leading players in the new year. We invite interested stakeholders to contact us and arrange a meeting ahead of the event.