This Movember, Ipsen once again champions men’s health through our “Get Men Talking” podcast series — a heartfelt initiative encouraging open conversations about prostate cancer and wellbeing.
In this year’s episode, Ipsen’s own Tim Batchelor, now 10 years clear of prostate cancer, sits down with Errol McKeller, fellow survivor and men’s health advocate, and Olympic legend Colin Jackson. Together, they share powerful personal stories, break down stigma, and inspire men everywhere to take charge of their health — because talking can save lives.
The “Get Men Talking” campaign is part of Ipsen’s continued commitment to raising awareness around early screening, prevention, and support for men affected by prostate cancer. Through authentic voices and real experiences, we’re driving an open dialogue that empowers men — and their loved ones — to act early.
👉 Listen to the podcast, share it with your network, and join us in keeping the conversation going.
Together, we can make a difference — one conversation at a time.
Targeting what cannot be seen: Lindsey Rodrigues on the complexity of epigenetics or mutated in a particular cancer. We have to ask, ‘do we have an understanding of how this gene works? Can we design a medicine to treat it or to target it?”’
Lindsey Rodrigues works in the early research space—well before molecules reach the clinic. Her focus is epigenetics, an area where cancer cells are reprogrammed at the level of gene expression, not genetic mutation. “You have these proteins that are responsible for reading, writing, and erasing epigenetic marks,” she says. “And that controls whether a gene is turned on or off. You can’t see it with traditional tools. That’s what makes it so interesting.”
At Ipsen, she leads the biology strategy for early-stage research programs and supports external innovation teams as they evaluate potential assets to add to Ipsen’s pipeline. “You might have two molecules with similar data,” she says, “but very different mechanisms. We’re asking: is this selective? Does it hit the right target? Do we understand the patient population?”
Her background in academic oncology helps guide that analysis. She trained in microfluidics, RNA biology, and CRISPR screening—which is used to run large-scale experiments that show how genes affect a certain trait, disease, or cell behavior. “I try to find something real in the data,” she says. “Especially something robust.”
Lindsey sees epigenetics as a space where good ideas often fail to translate. “It’s a moving goal,” she says. “They are context-dependent. They may work in a cell line, but not in a patient, and the patient population may be narrower than the sponsor originally believed.”
For her, this makes the science more urgent, not less. “There are so many failures,” she says. “But if something works, it’s powerful.”
Her team works cross-functionally from the beginning, engaging with chemistry, clinical, and pharmaceutical development functions to assess what a compound would need to succeed. “We’re not working in isolation,” she says. “We need input across the board.” That collaborative pressure keeps her focused. “We ask: is this a project that we would all be proud to work on? Is this a treatment we would want our families to receive?” Those questions drive her and her team to the finish line of discovery, playing a part in the larger role of medicinal development.
We’re proud to be part of The Liver Meeting 2025 hosted by the American Association for the Study of Liver Disease (AASLD), where leading healthcare professionals, scientists and the patient community come together to discuss the latest advances in scientific and medical innovations and care, across liver diseases.
This year at The Liver Meeting, we have a strong presence, with data from five rare liver diseases being presented as late breaking abstracts, oral presentations, and posters of distinction. These data, covering primary biliary cholangitis (PBC), progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome (ALGS), biliary atresia (BA), and primary sclerosing cholangitis (PSC), advance our understanding of these conditions and demonstrates our continued commitment to research in rare liver conditions and improving patient outcomes.
As science and technology evolve, we are understanding more about the biology of symptoms, exploring disease-modifying potential, and sharing our science so that we can help shape the future of hepatology.
We use our scientific expertise across five disease areas, PBC, PFIC, ALGS, BA and PSC, to develop medicine that have life changing potential for people living with a rare liver disease. Our understanding of these diseases is evolving.
Our growing understanding of the biology behind PBC has led to recognition that fatigue is not just a general feeling of tiredness, but a distinct and independent symptom of the disease, separate from pruritus (severe itching).
As we uncover the full spectrum of symptoms and their underlying mechanisms, management of PBC is becoming more personalized, focusing on what matters most to patients, including quality of life.
Advances in genetic testing have revealed that PFIC can present not only in childhood but also in adulthood, leading to more accurate diagnosis for adult patients and a broader understanding of its clinical spectrum.
In ALGS, the liver is often the most affected organ, with bile duct abnormalities causing bile to build up to harmful levels. About half of children see improvement in bile flow by age five, while the other half experience worsening symptoms and complications.
Pruritus caused by bile acid accumulation is frequently the most debilitating symptom, severely impacting sleep, emotional well-being, and daily life. In some cases, it becomes a key driver for liver transplantation.
BA is a rare liver condition that affects infants, requiring early recognition and diagnosis to improve outcomes.
There is a spectrum of experiences among those affected by BA, reflecting its complexity and varied impact on children and families.
Our understanding of PSC is growing, effective therapeutic options for PSC are very limited, with liver transplant being the only approach that can support people’s long-term outlook.
There is significant need for both patients and doctors to have access to a tolerable and efficacious treatment option.
By partnering with the patient community, we can raise awareness of these diseases and understanding of the patient experience among healthcare professionals.
Catch up on each day’s key moments and breakthroughs with daily updates from Hugo Gomes da Silva, sharing expert commentary and reflections on the science shaping this year’s meeting. New episodes uploaded daily throughout the congress:
Hear directly from key opinion leaders, patient advocates, and Ipsen experts as they discuss new data, evolving perspectives, and the future of RLD care – recorded live at The Liver Meeting 2025. Watch exclusive interviews from the meeting:
Our presence at The Liver Meeting reflects our ongoing commitment to advancing liver research and supporting the global community.
Explore highlights, posters, and expert sessions from previous meetings.
For more information on fatigue in PBC, read here.
Why Liver Disease? A Vision for Change
Liver disease has emerged as a critical focus for the healthcare science community, driven by significant unmet needs—particularly in rare and underserved conditions. The liver plays a central role in human biology, acting as a hub for carbohydrate, lipid, and protein metabolism. It is also the origin of many inherited metabolic disorders, most of which stem from autosomal recessive mutations in single genes. These conditions are more common than many realize, with estimates suggesting that one in every 800 live births is affected by a form of inherited hepatic metabolic disorder.
For decades, liver transplantation has been the only curative option available to these patients. However, the landscape is shifting. Early efforts in diseases such as Primary Biliary Cholangitis (PBC), Progressive Familial Intrahepatic Cholestasis (PFIC), and Alagille syndrome have shown that targeted innovation can deliver first-in-class treatments to patients who previously had few or no options. Companies like Ipsen are leading the way, demonstrating how focused investment in hepatology can set new standards for rare disease innovation and patient care.
Smarter Trial Design: Building the Foundation
As the science evolves, so too does the way we design clinical trials. Across the industry, there is a growing emphasis on smarter, more agile approaches to development. These new models are grounded in real-world patient insights and increasingly enhanced by artificial intelligence and real-world data, which enable more accurate predictive modelling.
Rare diseases, by their nature, present unique challenges due to their complexity and heterogeneity. To address this, the industry is embracing adaptive trial designs, single-arm studies with external controls, and Bayesian methodologies. These approaches not only improve study outcomes but also accelerate the path to approval.
Patient-centricity is also becoming a cornerstone of innovation. The development of patient-reported outcomes (PROs), such as the FDA-recognized pruritus tool developed by Ipsen, reflects a broader commitment to ensuring that the patient voice is embedded in every stage of the development process.
Where We Are Today: Expanding the Liver Portfolio
The liver disease space has expanded dramatically in recent years. What was once a field dominated by symptomatic treatments is now home to a growing portfolio of disease-modifying therapies. Advances in precision medicine, the use of novel clinical endpoints, and value-based profiling are helping to tailor treatments more effectively to individual patients.
At the same time, our understanding of disease biology is deepening. Innovations in proteomics and pathway modulation are opening new avenues for intervention. These scientific advances are increasingly aligned with regulatory preparedness, enabling faster and more efficient development pathways. Ipsen and other forward-thinking companies are helping to drive this alignment, ensuring that innovation is not only possible but also accessible.
Looking Ahead: Gene Editing in Liver Disease
Liver diseases caused by single-gene mutations affect millions of people worldwide. While traditional treatments have focused on managing symptoms, they rarely offer a cure. Gene editing is now emerging as a transformative approach, with technologies like CRISPR/Cas9 enabling gene insertion, correction, and knockdown.
However, the liver presents unique challenges for gene editing due to its low cell division rate and inefficient DNA repair mechanisms. A promising solution is Repair Drive, a novel strategy developed by Rice University and Baylor College of Medicine. This approach enhances gene correction efficiency by giving edited cells a survival advantage, allowing them to outcompete diseased cells during natural liver regeneration. Importantly, Repair Drive is compatible with existing delivery platforms such as adeno-associated viruses (AAV) and lipid nanoparticles.
This innovation could mark a turning point in the treatment of pediatric and early-onset liver conditions, shifting the paradigm from symptom management to curative therapies.
The Next Frontier: Regenerating the Liver
Beyond gene editing lies an even more ambitious goal: regenerating liver tissue itself. Cellular regenerative therapies represent the next frontier in hepatology, moving us from treatment to true restoration.
Emerging technologies are exploring bio-delivery systems that can transform liver cells into temporary implantable, tissue-like therapies. One such example is the work being done by Dimension Bio, which is pioneering what it calls “tissue therapeutics.” These therapies have the potential to delay——the need for liver transplantation in cases of liver failure.
This vision of regenerative medicine challenges us to think differently. Are our regulatory and reimbursement systems ready to support such transformative innovation? The answer to that question will shape the future of care.
The System Challenge: Innovation vs. Infrastructure
While scientific progress is accelerating, healthcare infrastructure is struggling to keep pace. Regulatory frameworks are often slow to accommodate novel endpoints and adaptive trial designs. Reimbursement pathways, too, are frequently misaligned with the speed and scope of scientific breakthroughs.
Without systemic alignment, even the most promising therapies risk being delayed or rendered inaccessible. The challenge is not just to innovate, but to ensure that innovation reaches the patients who need it most.
Bridging the Gap: Proactive Engagement
To overcome these barriers, industry leaders are taking a more proactive role in shaping the future of healthcare. This includes engaging with regulators and payers to advocate for adaptive trial models, novel endpoints, and more flexible reimbursement structures.
Collaboration is key. By working together—across industry, regulatory bodies, payers, and patient advocacy groups—we can create an ecosystem that supports innovation from bench to bedside.
Shared Values: Curiosity, Courage, Commitment
At the heart of this transformation are the values that drive the sector forward. Curiosity fuels the exploration of new scientific frontiers. Courage empowers us to challenge conventional thinking. And commitment ensures that we never lose sight of the patients we serve—especially those in vulnerable and underserved communities.
Ipsen exemplifies how these values can be brought to life, combining scientific excellence with advocacy to elevate the voices of patients who are too often overlooked.
Redefining What’s Possible
The journey in liver disease is about more than developing new medicines. It’s about reshaping the future of care. By pushing the boundaries of clinical science and advocating for systemic reform, we can ensure that innovation is not only achieved but also delivered.
The future of hepatology will be defined by how well science and systems evolve together. If we get this right, we won’t just treat liver disease—we’ll redefine what’s possible for generations to come.
We are proud to announce our robust performance across all therapeutic areas in the first nine months of 2025, underscoring our commitment to delivering transformative medicines to patients worldwide.
A Strong Year-to-Date Performance
Total sales rose by 12.1% at constant exchange rates. Growth was driven across all three therapeutic areas:
This performance has prompted an upgrade to our full-year 2025 guidance, now forecasting total sales growth of around 10% total sales growth* and a core operating margin of around 35%.
Strategic Expansion in Oncology
We also announced our intention to acquire ImCheck Therapeutics, a pioneering French biotech specializing in next-generation immuno-oncology. The acquisition centers on ICT01, a first-in-class monoclonal antibody targeting BTN3A1, currently in Phase I/II trials for first-line unfit acute myeloid leukemia (AML). Early data show unprecedented response rates, nearly double those of standard care.
This move reinforces Ipsen’s nearly 40-year legacy in Oncology, expanding our pipeline into hematology-oncology and addressing cancers with high unmet medical needs.
Looking Ahead
As we enter the final stretch of 2025, we remain focused on delivering innovative therapies to patients who need them most. Our continued growth and strategic investments reflect our belief that everyone deserves a life fully lived.
*At constant exchange rates
Every person’s experience living with neuroendocrine tumors (NETs) is unique and for Melanie, coming to terms with a diagnosis of lung NETs is an ongoing journey, building knowledge and understanding of how to live with this complex and often unknown form of cancer.
Getting a diagnosis
As is so often the case for many people, Melanie’s diagnosis came by chance following a routine check-up for a cough. When a subsequent x-ray revealed some shadowing on her lungs, her GP initially diagnosed her with lung cancer, with a very poor prognosis, something she wasn’t told in the appointment, but discovered in her notes. This caused immense distress at the thought of what this would mean for her future. However, it was only after a biopsy was conducted that the true nature of her diagnosis was revealed: Melanie was in fact living with an advanced form of lung NETs, a term she had never heard of before.
The quest for knowledge
NETs are a relatively uncommon form of cancer that affects the cells of your neuroendocrine system.1 These cells are found throughout your body, but NETs most often occur in the lungs, stomach, or pancreas.2 Most forms of NETs grow slowly and are less aggressive,3 a point the doctor was keen to emphasize to Melanie. However, being diagnosed with cancer of any form is life changing.
While Melanie finally had an answer, the uncommon nature of NETs, especially lung NETs, meant that information about the disease was scarce. Yet, with approximately 35 in every 100,000 people worldwide living with NETs,1 and lung NETs accounting for 27% of all cases,2 this type of cancer is far more prevalent than many realize. In order to seek out more information, Melanie sought a second opinion from a professor at a NETs Center of Excellence, who had the expertise to discuss the complexities of her diagnosis.
While Melanie was reassured that her NETs could be treated, it wasn’t operable. This meant she had to learn to live with it, which is a continually evolving process – and to do so, she had to gain a deeper understanding of this complex disease. Melanie credits a national patient advocacy group for being her crucial support network, offering understanding from others facing similar journeys and providing a hub of information to turn to.
“Everyone is completely and utterly different. But at the same time, incredibly supportive and positive and, even on the really hard days, there is love and you feel like you’re part of a big family.”
Being seen and understood
Having an uncommon cancer can feel isolating. While Melanie feels physically well most of the time, she still shoulders the burden of living with a form of cancer where care options are difficult to navigate and the future is unknown. Recently, Melanie attended appointments with a friend as they were diagnosed with a more common type of cancer highlighting the disparity in care between cancer types. The medical knowledge, funding and specialist support experienced by those with less common cancers can be stark; from a lack of dedicated nursing staff, timely scan results, patient information, support group awareness and even funded parking at hospital car parks.
A long-term outlook on life
Melanie has accepted that her NETs may grow, change, shrink, or spread, but she is still determined to live well; enjoying activities for what they are, not just associating them with providing relief from her diagnosis. Her job as a gardener and recently discovered love of sea swimming means she can continue to enjoy her passion for the outdoors. Learning to live with NETs is an evolving journey, one Melanie is taking one step at a time.
22 October 2025 – 1 mins read
PARIS, FRANCE, 22 October 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents its sales for the year to date and the third quarter of 2025.
Ipsen’s financial results for the first nine months of 2025 were published on 22 October 2025.
An investor and analyst conference call took place on the same day.
Webcast RecordingWe 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.
Discover Ipsen, our medicines and key figures, our objectives and our strategy.
If you are experiencing difficulties registering for the investor and analyst conference call, please contact: khalid.deojee@ipsen.com
Hugo Gomes da Silva, SVP Global Medical Affairs (Rare Disease) at Ipsen, shares powerful insights into the evolving challenges families face throughout their PFIC journey—and highlights the critical importance of trusted information and community support.
As we mark PFIC Awareness Day on October 5, I reflect on what I have heard from those living with Progressive Familial Intrahepatic Cholestasis (PFIC) and how the disease impacts them not just on one day, but every single day. PFIC is a rare genetic liver disease, often diagnosed in infancy or childhood, that can cause progressive liver damage, severe itching, and has a profound impact on quality of life.
This year’s theme, Stronger Together, speaks to the power of unity, empathy, and shared knowledge within this community. For families, this theme represents not only awareness of the condition itself, but also the reassurance that no one should face PFIC alone.
I have had conversations with those parents who embody this spirit of strength and advocacy. One of them is Emily Ventura, co-founder of the PFIC Network and mother to Cedar, who lives with PFIC. She remembers how difficult it was in the early days of her daughter’s diagnosis: “Learning what PFIC really meant for my daughter was overwhelming. I felt lost and unsure of where to find reliable guidance.”
What helped Emily most was finding a community of families who had walked the same path, transforming her fear into confidence. Inspired by this, she went on to co-found the PFIC Network, which provides both trustworthy information and a safe space for families to connect.
Francesca faced a similar journey when her daughter, Eva Luna, was diagnosed with PFIC as an infant. The first months were filled with uncertainty and fear as they tried to understand what the diagnosis meant. One of the most difficult challenges was managing Eva Luna’s persistent itch, which Francesca described as “like a monster” that disrupted family life. Helping her daughter feel comfortable and live a happy, active childhood became the focus of their journey. Many other families share this challenge of balancing symptom management with daily life and hope for the future.
For Francesca, connecting with other mothers gave her hope and perspective: “It was very important to me to be in touch with the other mothers around the world. It gave me a lot of hope, because I saw their children grow, go to school and even live a normal life.” This led her to set up PFIC Italia, which not only supports those in Italy, but has international reach.
Emily’s and Francesca’s journeys show us that behind every diagnosis is a family searching for clarity, support, and the reassurance of not being alone. To navigate PFIC, families need both knowledge that empowers them and a community that gives them strength.
At Ipsen, my colleagues and I are committed to listening to families and standing alongside them. PFIC Awareness Day reminds us that advancing science is only part of the solution; solidarity and support are equally vital. By standing stronger together, we can help families feel informed, empowered, and never alone on their journey. The PFIC Network is a trusted source for families, bringing together the full spectrum of information, tips, and real experiences in one place.
To learn more about PFIC, visit the PFIC Network’s website: PFIC.org and the PFIC Italia Network
“We collaborate with our medical and chemical colleagues doing drug discovery, taking a look at the current process, and thinking about the best ways to improve efficiency and minimising environmental impact.”
Nathan McCormack focuses on the earliest stages of active pharmaceutical ingredient (API) development. This involves working alongside medicinal chemistry colleagues to identify new molecules, then working to make them practical for scale-up and manufacturing. Part of the API team in Dublin, Nathan supports new molecules for the three Ipsen therapeutic areas of neuroscience, rare disease, and oncology, partnering closely with medicinal chemistry colleagues to assess how a compound is made—and how that process will hold up under large-scale constraints. “What you use on a small scale may not necessarily translate,” he says. “In terms of safety, it’s something we have to think about on a manufacturing scale.”
Nathan’s job is not just about synthesis. He and his colleagues evaluate the feasibility of every reaction route, identifying risks and opportunities early. “We’ll support the supply of this API through preclinical and clinical stages,” he explains, “until we reach commercial manufacture.”
That role also includes tracking what happens to a compound after it enters the body. “We work closely with our external partners,” says Nathan. These partnerships play a large role in innovation and building better processes for sustainability. What excites Nathan most is driving to improve sustainability and environmental footprint. Ipsen is a member of the American Chemical Society Green Chemistry Institute Pharmaceutical Roundtable (ACS GCI PR), a collaboration with other pharmaceutical companies that Nathan and his coworkers see as critical. “We’re already taking action—benchmarking, analyzing, and changing what we do.” This is done to reduce environmental impact while maximizing scientific discovery.
That work also includes partnerships with academic labs. “They come up with some of the best innovations,” he says. “And we can help them adapt it for industrial applications.” For Nathan, sustainability is not a side project—it is the future of drug development.
Naomi Binoche, VP Head of Strategic Alliance Management, shares insights on how pharma partnerships are key to driving success and why celebrating every milestone along the way matters.
This theme was further explored at the ASAP Global Alliance Summit in Amsterdam recently, where Naomi co-presented the session “Living and Growing an Inherited Alliance – a Journey full of Twists and Turns.” The presentation highlighted how Ipsen and Servier overcame the challenges of an inherited partnership to build a successful alliance – a journey that reflected the resilience and adaptability required in the pharma landscape.
“For me, success is not just about big breakthroughs. In nearly 10 years with Ipsen, I’ve learned it’s just as much about building trusted partnerships and celebrating every milestone along the way. We thrive by working together – it’s what drives innovation across our therapeutic areas and ultimately helps us deliver transformational medicines to patients worldwide.
Moving forward together can be transformational. When combining Ipsen’s global reach, size, and expertise with our partners’ cutting-edge research we are able to consistently push new and exciting programs through the pipeline. I’ve been very fortunate to contribute to many successful partnerships that have advanced our programs; moving them from pre-clinical development, through regulatory approval, and finally into the market where they can benefit those in need.
What fulfils me every day is the diversity of partners we work with: from the US-listed biotech to the family-owned business in Europe; the ambitious young enterprise in China, to the established industry leader. Each brings its own unique challenges and opportunities, and it is this variety that makes my day – every single day!
Turning a molecule into a medicine is a long and complex journey, demanding years of dedication and collaboration. That’s why it’s so important to pause and celebrate every win along the way. These shared milestones – whether operational, strategic, or relational – are the heartbeat of alliance success, helping to build trust and momentum over time.
An alliance isn’t just about the initial collaboration or license agreement. It’s about all the supporting pieces that go alongside. Pharmacovigilance, quality, and data protection, manufacturing, supply, and life-cycle opportunities and more. Each achievement is something we secure together with our partners over time. These milestones might not make the headlines, but for me and my team, they’re standout moments that reflect the strength of our collaboration.
Of course, the journey doesn’t stop after bringing a medicine to market. Keeping our medicines available in over 100 countries takes enormous effort, with teams around the world working together every day to make manufacturing and distribution happen. This is where our long-term alliances really shine. Some of our partnerships span over 30 years, with off-patent molecules still achieving double-digit growth in certain regions.
Celebrating each milestone with our partners, especially during what can be a long and challenging journey, fuels our motivation to push programs forward and ensure medicines remain accessible. In the end, making a real impact for patients is what matters — and that’s always worth celebrating.”
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