Category: Pharma Talks

16 December 2025

1. The M&A environment

Jens: How would you describe the current state of pharma and biotech M&A? Are we returning to pre-2022 dynamics, or has something fundamentally shifted?

Alan: The M&A landscape in biopharma is primarily driven by upcoming losses of exclusivity over the next five to ten years. Big pharma needs to replenish the top line because several important products are going away.

Valuations are also relatively favorable, which is why we’re seeing many bolt-on acquisitions. Buyers tend to focus on later-stage assets, either commercial products or programs with established proof of concept, so they can get to market fairly quickly.

There is also a focus on platform technologies: either because a product can expand into multiple indications, or because the underlying technology can be applied across other parts of the business.

Overall, there’s renewed interest in the sector, and that’s part of why sentiment has improved. However, biotech still faces fundamentals it hasn’t solved: the cost of developing drugs hasn’t improved in years, timelines remain long, and risk hasn’t diminished. At the same time, price control pressures are increasing, and competition from China has not been fully addressed, particularly in the U.S., and to some extent in Europe as well. So yes, there is breathing room due to M&A activity, but the industry should use this period to transform and become more efficient, so it comes out of this window on a stronger foundation.

Jens: I agree. Years ago, when I was co-founding biotech companies, platform technologies weren’t particularly attractive to investors, people focused on single drugs. That seems to have changed, and platform thinking is now also used to de-risk and broaden the opportunity.

Alan: Exactly. There’s a growing realization in large pharma that you need access to large markets. Obesity illustrates that. Look at the valuation multiple of Eli Lilly versus other big pharma, much higher, largely driven by the perceived long-term value of their obesity franchise.

That is pushing companies to prioritize very large opportunities. A product that will never exceed a billion dollars can be hard to justify in a very large organization. As a result, we expect consolidation in rare disease as big pharma exits. Consolidation in specialty pharma is also ongoing.

It will be interesting to see how biotech positions itself to be attractive to different pools of buyers.

2. What makes biotech attractive today

Jens: One major shift I see is evidence-based development: having a clear plan not only for the clinical trial, but also for biomarkers, endpoints, and early payer discussions, so pharma sees lower risk in licensing or acquiring. Ten years ago, many of us weren’t thinking about payers early.

Alan: I agree. One hope I have is that we reach the end of what I call the “biotech mafia”, financial engineering around products or platforms that are overly complex for the public to evaluate, where IPOs were used as a proof point for early exit, leaving less sophisticated investors holding the risk.

Greater access to information may reduce that. The IPO window still hasn’t truly reopened, and we probably need additional interest rate cuts before it does. But with more transparency, capital allocation should improve, though we’ll see.

3. Acquisition logic: diligence, CMC, and market reality

Jens: Today, does the “whole package” matter more, CMC readiness, clinical development quality, strong proof of concept? Has acquisition logic changed compared with 20 years ago?

Alan: These have always been the basic building blocks. If you’re spending hundreds of millions, or even ten million, you should do thorough due diligence to understand risk across all dimensions.

Yet deals still happen under urgency where diligence is limited, and that often comes back to hurt the buyer. We’ve seen situations where buyers paid extremely high multiples and later realized the assets couldn’t be leveraged the way they expected. Those are failures of decision-making and diligence.

Also, especially on the investment side, understanding the ultimate market has become far more important. The earlier you understand payer reality, the better your decision-making becomes.

The old assumption, “if the science is good, someone will figure out how to sell it”, is changing. Commercial practices have tightened. Products must demonstrate meaningful value to patients, and payers need to agree.

Still, we regularly see investments where this market work hasn’t been done. Teams focus on “what’s the next clinical trial” instead of asking: what will competitiveness look like in five or ten years? Who else is coming? What alternative mechanisms or technologies could challenge this? What are payers thinking? Unfortunately, this isn’t systematic enough.

4. Acquisition vs. interest

Jens: What triggers serious acquisition or partnering interest today? What makes a company “deal-ready”?

Alan: It starts with strategy. If you have a clear strategy, you understand what growth you need, at what pace, and whether you need inorganic options versus internal build.

Then there are events you can’t foresee, like a Phase III failure, that suddenly force action. A company may need to acquire or license a stopgap to stabilize while conditions change. That remains a key driver.

The competitive landscape matters on both the buy side and the sell side. Companies ask whether they can realistically be among the top three in an area. If they can’t, it may no longer make sense to keep investing, and divestment becomes the rational move.

For example, we’re working with a company that developed a platform and initially pursued immunology assets, but realized the platform has greater potential in metabolic disease. They refocused resources toward metabolic development and are now selling a portfolio of five early-stage immunology assets, interesting assets, just not strategic for them.

There are also cases driven by external pressure. If investors push for improved profitability or margins, companies may divest lower-margin businesses to improve overall economics and, by extension, valuation multiples.

So it’s a combination of long-term shareholder value creation, strategic discipline, and not missing emerging hyper-growth opportunities. A strong example is Eli Lilly’s GLP-1 strategy: many assume it “just happened,” but leadership has described having a clear strategy in 2017. That kind of foresight is impressive.

5. Why deals stall or fail

Jens: Why do some deals stall or fail, and how can leaders overcome those issues?

Alan: Most often, deals fail due to lack of alignment at the decision-maker level. A mentor once told me: “You’re the conductor on the train, make sure nobody gets off.” That’s accurate. Deals can derail easily.

It’s always easier to say “no” than to say “how do we make this work?” You want people who focus on making the deal work in a way that creates value for both organizations.

Alignment among decision-makers on both sides is critical. You need access to the real decision-makers and confidence that your counterpart truly has their ear. You need active communication back and forth, and ideally you become allies, each working internally to keep your respective organizations aligned. Many deals fail because that alignment work doesn’t happen.

6. Global perspective

Jens: Do you observe differences in how U.S., European, and Asian companies approach M&A valuation and risk? What should CEOs adapt when engaging across regions, especially given China’s competitiveness?

Alan: Culture is very different, and you have to be aware of that.

In Asia, deals typically start with the relationship. You build trust first, and then you do the deal.

In the U.S., it’s often more transactional: you can go straight to the terms and the numbers. You don’t need to be friends to do business.

That affects meeting dynamics. A hard-charging American style can create distrust in Asia if the relationship foundation isn’t there yet. Trust needs to be built.

On risk: in general, Americans are less risk-averse, sometimes because leaders in public companies may only have a three-to-five-year window in their role and want to show impact, transactions included.

Many Asian companies, often family-owned or operating with multi-generational horizons, and to some extent Europe as well, think longer-term. They are more cautious because decisions affect long-term sustainability and, in some cases, future generations.

The downside is that greater caution can mean missing opportunities. It’s the classic trade-off: high risk, high reward.

7. Deal readiness for 2026

Jens: What recommendations would you give CEOs preparing for M&A readiness in 2026? Where should they invest time and resources early?

Alan: The fundamentals are the same: clear data, a well-run operation, and clean financials.

In every deal, there is usually a “skeleton in the closet” somewhere. The key is to surface it, not hide it – and have a mitigation plan. Show the potential acquirer you understand the issue and are actively managing it.

Valuation sophistication is increasing. With scenario tools, including AI-based approaches, you should have a strong understanding of what your company is worth and why.

Clarity of purpose also matters. If you decide to sell, be decisive. Not “we’re exploring,” but: we’ve done the work, we know our value, we know where it comes from, we have a timeline, and we are executing. That gives buyers confidence their resources won’t be wasted.

Finally, be collaborative and transparent. Your fiduciary responsibility is to maximize value, but you do that by being clear about your model and assumptions, not by hiding or embellishing. You are who you are as an organization.

The best way to maximize value is to attract multiple credible parties and create competition. Transparency helps build buyer confidence and broadens the pool. To use an analogy: if you sell a house and show everything openly, more people can get comfortable, and you create competition. Too often companies hide issues, end up with only one buyer, and then fail to maximize value.

8. What will shape the next wave of pharma M&A

Jens: Final question: what scientific fields or business models will shape the next wave of pharma M&A?

Alan: AI will drive a lot of transformation. We started this conversation with the need for biotech to become more efficient. AI has the potential to streamline multiple verticals, discovery, manufacturing, regulatory, and clinical, so the process of bringing a drug to market becomes faster and less costly.

Historically, timelines expanded dramatically. There was a time when companies could bring drugs to market far faster than today. While safety and regulatory standards have evolved, we now finally have technologies that could compress timelines and reduce cost.

That enables new business models. If time-to-market and cost-to-market decline, pricing logic may evolve as well. AI can also support greater precision, targeting specific genotypes or subpopulations rather than broad, less efficient approaches.

Today there are roughly 2,000 pharmaceutical products targeting a few hundred mechanisms of action. There is heavy concentration around known biology, intense competition, and significant waste, especially when you’re not among the top three in a category. Over time, AI could help reduce redundancy and improve capital allocation.

This will take time – likely ten to fifteen years – but it’s coming.

Beyond AI, prevention could become more important. Pharma is largely treatment-oriented today; a more prophylactic model could grow.

Finally, global commercialization and collaboration are important. The current system, every country having its own regulatory agency, creates artificial silos and inefficiencies. Greater harmonization would be beneficial.

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Jens: Thank you for your time and input.

13 November 2025

1. Progress of Genethon’s Gene Therapy Programs

Q: How are Genethon’s programs progressing, especially in neuromuscular diseases?

A: Genethon is currently advancing a robust pipeline of gene therapy programs with a strong focus on neuromuscular and metabolic disorders. One of the most significant recent achievements is the progression of the Duchenne muscular dystrophy (DMD) program, GMT-0004, into Phase 3. This represents the most advanced Duchenne gene therapy program in Europe. Early data from the dose-escalation cohorts show not only biochemical improvements, such as a roughly 75% decrease in circulating creatine kinase (CK), but also stabilization of motor function—an important indicator given the relentless progression of Duchenne.

Genethon is also progressing multiple additional neuromuscular programs toward pivotal readiness. In Crigler–Najjar syndrome, one of the longest-running clinical efforts, several treated patients have now been living for almost five years without requiring phototherapy. This represents a profound improvement in quality of life and a durable therapeutic effect.

2. Clinical Impact and Dose Advantages

Q: What differentiates Genethon’s Duchenne gene therapy from other approaches?

A: A key differentiator of GMT-0004 is its dose efficiency. Compared to other leading programs in the Duchenne space, Genethon administers vector at much lower doses while achieving meaningful and durable clinical benefits. Specifically, the program uses a dose approximately four times lower than Sarepta’s SRP-9001 therapy and nearly seven times lower than RGX-202 from Regenxbio/Solid Biosciences. Lower dosing is clinically and commercially significant: it reduces the risk of immune-mediated toxicities, improves overall safety margins, and eases manufacturing demands, all of which contribute to a more scalable and sustainable therapy.

The differentiation is further reinforced by the fact that Sarepta’s Phase 3 trial did not achieve statistical significance on its primary endpoint. Against this backdrop, the stable motor function and biomarker improvements observed in Genethon’s early cohorts — coupled with the consistency of response across multiple clinical centers — provide strong confidence in the program’s therapeutic potential.

3. Challenges in the One-Shot Gene Therapy Model

Q: Why is the one-time gene therapy business model difficult to sustain?

A: While the vision of a one-time curative therapy has driven much of the field’s early excitement, the business model underlying a single-administration therapy is proving difficult to sustain. Because a one-time treatment must recoup the full cost of research, development, manufacturing, and risk within a single dose, pricing pressures inevitably rise to levels that strain payer budgets.

In addition, the biological and clinical reality of many diseases complicates the concept of a single-dose cure. Tissues regenerate, patients grow (especially pediatric patients), vector expression can decline over time, and the disease may continue progressing. These practical considerations create demand for re-dosing — something that most current AAV-based therapies cannot support due to long-lasting immunity.

The future of gene therapy is likely to shift toward re-dosable or chronic administration. This transition is being enabled by innovations such as lipid nanoparticles (LNPs), next-generation capsids with reduced immunogenicity, and improved immune-modulation strategies that may one day allow safe AAV re-dosing. Such solutions would shift gene therapy economics toward more traditional pharmaceutical models.

4. Ultra-Rare Diseases and New Funding Models

Q: What makes ultra-rare diseases especially challenging?

A: Approximately 85% of rare diseases fall into the category of ultra-rare disorders, each affecting fewer than one in 100,000 individuals. While the scientific rationale for treating these conditions is often strong, they are generally unattractive markets for pharmaceutical companies and venture investors, who require larger patient populations for meaningful return on investment.

For patient organizations like AFM-Téléthon, which founded Genethon, this creates a profound ethical dilemma. Families affected by ultra-rare diseases often have no available treatment options and struggle even to access clinical trials. Clinical development for ultra-rare diseases should be publicly reimbursed, as trial participation itself represents the only therapeutic pathway for these patients.

He stresses that expecting charities to shoulder full late-stage development costs is not viable. Government-level intervention and alternative funding models are needed to ensure equitable access.

5. Regulatory and Reimbursement Landscape

Q: How do regulators and payers shape the development pathway for gene therapies?

A: Regulatory agencies such as the EMA and FDA have demonstrated strong openness to innovation in the gene therapy space. Europe is not inherently more restrictive than the U.S.; in fact, several ATMPs reached European markets before being approved in the United States.

The more significant barrier is reimbursement. Given the high price of ATMPs, payers require rigorous evidence demonstrating superiority over standard of care, long-term functional benefit, and durable biological effect. Randomized, placebo-controlled trials, long-term follow-ups, and real-world data are essential components of the evidence package.

6. Partnership Strategy and Access to Patients

Q: How does Genethon approach partnerships and collaborations?

A: Genethon evaluates partnerships through a single primary lens: would a collaboration accelerate patient access? This reflects Genethon’s origins as a patient-driven research organization. When partnerships can shorten development timelines or enhance global reach, they are actively pursued. However, for ultra-rare diseases, commercial partnerships are often infeasible due to limited market potential.

7. Role of Artificial Intelligence in Gene Therapy

Q: How is AI transforming Genethon’s approach to research and development?

A: Artificial intelligence is playing an increasingly important role in Genethon’s R&D. The organization recently published work in Nature Communications demonstrating the ability to design novel AAV capsids with organ-specific tropism using AI-driven approaches.

AI is also being applied in manufacturing, where predictive models may eventually enable digital bioreactor concepts. In clinical research, Genethon is using AI to develop rare-disease patient data hubs with French hospitals, improving the understanding of disease trajectories and enabling more objective monitoring of therapeutic response.

8. Europe’s Competitive Position in Gene Therapy

Q: How competitive is Europe globally in gene therapy innovation?

A: Europe has longstanding roots in the field — including the world’s first successful gene therapy trial in Paris for the ‘Bubble Babies’ syndrome. Europe today benefits from a mature scientific ecosystem, industrial capabilities, and long-term government initiatives.

One of the most significant initiatives is ‘France 2030,’ which established five national bioclusters targeting key therapeutic areas. Genother [the gene therapy biocluster chaired by Revah] received €140 million in funding and brings together manufacturing platforms, clinical networks, and training infrastructures. Europe is increasingly attracting talent from the U.S., reflecting strong scientific momentum and greater financial stability.

9. Leadership Insights for Biotech Innovators

Q: What mindset and skills should biotech leaders develop?

A: Successful biotech leaders must be both scientifically visionary and operationally grounded. They should be deeply passionate about science yet avoid falling in love with their own hypotheses. Equally important is a thorough understanding of CMC, regulatory strategy, manufacturability, and patient pathways.

Leadership in gene therapy requires balancing bold innovation with practical execution — ensuring that great science can ultimately become a real, manufacturable, reimbursable therapy for patients.

28.10.2025

1. Review  & Context

Jens Kurth:
Leadership today isn’t defined by control, but by connection. Over the past years, organizations have been forced to reinvent how they lead and collaborate. What, in your view, has changed most in leadership?

Udo Fichtner:
Two trends are certainly dominant. The pandemic has made remote work popular, and technology – especially AI – has an impact. Both trends change the requirements for great leadership. Unfortunately, they do not necessarily change the actual leadership behavior in many cases.

Jens Kurth:
You’ve also worked across multiple industries. Which experiences from other sectors can be surprisingly well transferred to pharmaceuticals or life sciences?

Udo Fichtner:
There are industries out there that appear to be more competitive and just a lot faster. Innovation cycles in some sectors are much shorter, which strongly shapes their corporate culture. Sometimes, more agility and flexibility would be desirable in pharmaceuticals and life sciences. It’s not easy, though, to transfer this spirit into a culture that has been immensely successful in a “protected” environment like pharma.

2. HR as a Lever for Transformation

Jens Kurth:
Transformation often starts with technology but fails on the human side. Where does change most often break down – and why?

Udo Fichtner:
Let me quote two people who are hitting the nail on the head. Michael Kramarsch, founder of hkp, once said: “Transformation is never technology but always human behavior.” And my long-term companion and dear friend Steffen Fischer, CHRO of ifm electronics, with whom I founded the “AI-HR-Lab” back in 2018, added: “The winner is not the one using the best tools, but the one managing the best human–machine interaction.” That says it all.

Jens Kurth:
And what does it take for leaders not only to change things, but to truly move people?

Udo Fichtner:
Know your people. Trust your people. Treat them like adults. And communicate, communicate, communicate. One individual is thrilled by a simple “There is change coming up, follow me” – and doesn’t need much more to buy in. Another needs careful explanation, over and over again. Some are fine with an email or circular announcement, others prefer a video or twenty individual conversations. Remember: know your people! It sounds very simple, but it requires true leadership skills.

Jens Kurth:
True movement happens when leadership meets empathy. The best transformation strategies fail if they forget the human cadence behind the change.

3. Leadership & Culture in Transition

Jens Kurth:
Creating a culture that combines innovation, responsibility, and courage is a challenge – especially in regulated environments. How can HR help to enable this balance?

Udo Fichtner:
It’s a tough one. In a recent project, I supported a company that transformed from a “normal” manufacturer into a CDMO – a contract development and manufacturing organization. We had to implement stringent documentation processes and regular training initiatives to become audit-ready as a first step. What it takes from there to create a sustainably innovative, responsible, and courageous corporate culture in a regulated environment – frankly, I don’t know. It sounds almost like a contradiction in itself: a regulated environment that supports innovation and courage.

Jens Kurth:
That tension defines much of the life sciences world – compliance and curiosity co-existing uneasily. Perhaps the answer lies in creating “safe spaces for courage,” where boundaries are clear, but within them, teams can experiment without fear.

Jens Kurth:
And what role does trust play in transformation – and how can it be anchored in organizations?

Udo Fichtner:
Trust is of the essence. In my experience, there is only one effective way to establish a culture of mutual trust: as a leader, show vulnerability, admit mistakes, talk about them, celebrate them. This ensures that people in your team learn that mistakes are human, can be admitted easily, and serve as a valuable source for learning and development. No finger pointing, no hiding, no politics. Such a culture allows constructive and healthy conflict about issues rather than people. This is the basis for successful transformation processes.

4. Outlook & Personal Reflection

Jens Kurth:
If you were advising a young HR team in a biotech or health-tech start-up – what advice would you give them?

Udo Fichtner:
Learn about data. Learn with data. Learn from data. Data will be King. Use data to support (or argue) people decisions. Data literacy is becoming the game changer for HR.

Jens Kurth:
Data-driven HR can turn intuition into insight – but only when empathy remains part of the equation. Numbers may guide decisions, but culture still determines whether they succeed.

And finally: what does “good leadership” mean to you today – compared to ten years ago?

Udo Fichtner:
I don’t think the fundamentals have changed over the past ten years. It’s still about vision, clarity, integrity, empathy, communication, accountability, adaptability, mutual trust, setting direction, decisiveness, resilience, and learning. Ten years ago – today – and ten years from now. Some of the tools will change, yet the fundamentals won’t.

Jens Kurth:
A timeless definition – and perhaps the best summary of our conversation. Technology will evolve, business models will shift, but the human essence of leadership remains unchanged.

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