Innovation in Research: Opportunities at Vertex Pharmaceuticals, BioNxt Solutions, and BioNTech

Vertex Pharmaceuticals: The Experts with the Gene Scissors

In late 2023, the US Food and Drug Administration (FDA) approved Casgevy, a CRISPR gene therapy from Vertex Pharmaceuticals. The European Medicines Agency (EMA) in Amsterdam followed suit in early 2024. The therapy was developed in collaboration with CRISPR Therapeutics and is now considered a medical milestone. The goal is not merely treatment, but a cure for two severe, hereditary blood disorders: sickle cell anemia and beta-thalassemia. In the process, the patient's own blood stem cells are harvested. In the laboratory, the CRISPR gene-editing tool is then used to edit or deactivate a specific gene (BCL11A). This effectively "reprograms" the body genetically so that it once again produces healthy, fetal hemoglobin. The modified cells are returned to the patient via infusion. The advantages are clear: After a single treatment, patients generally no longer require lifelong, risky blood transfusions and remain free of the extremely painful vascular blockages typical of sickle cell anemia. The success rate is also remarkable, exceeding 90% in the clinical trials. The market for Vertex is vast. In the US and Europe alone, approximately 35,000 patients have been identified as eligible for the therapy. Added to this is a large patient population in countries such as Saudi Arabia and the Persian Gulf. The long-term market potential is therefore estimated at several billion US dollars. After a slow start due to organizational issues, revenue is now picking up significantly. In 2025, Casgevy generated approximately USD 116 million. A steep growth trajectory is expected for the full year, with revenue projected to surpass USD 500 million. However, there are (still) major drawbacks: Casgevy costs USD 2.2 million per patient because it involves a highly complex, individualized procedure. The stem cells must be edited individually for each patient in the laboratory. The process takes several months from cell collection to the actual infusion and billing.

In terms of the numbers, things are going very well for Vertex. Revenue growth to USD 2.99 billion (+8%) was driven primarily by new drugs, which already account for about a quarter of the growth. The core business of cystic fibrosis drugs, such as Trikafta, remains the solid foundation of the US company. Earnings per share came in at USD 4.47, well above analysts' estimates. In addition, the company held cash reserves of over USD 13 billion as of the end of March.

Like most research-intensive US biotech giants, Vertex continues to pay no dividend. Profits are reinvested entirely into its own clinical pipeline or strategic acquisitions. However, share buybacks are ongoing. In the first quarter of 2026 alone, the company repurchased over 741,000 shares for approximately USD 344 million. Management confirmed the full-year 2026 revenue forecast of USD 12.95 to USD 13.10 billion. Vertex is currently valued at around USD 104 billion. The stock has recently been extremely volatile. Starting in early March, it fell by about 15% within eight weeks, only to nearly fully recover from this correction within two months. Vertex is expected to benefit in the coming quarters from the rollout of new drugs and therapies and from successful clinical trial results.

BioNxt Solutions: Oral Dissolvable Film Instead of Injections and Tablets

Not everyone likes injections, but for many diseases, there is currently no other way to deliver the active ingredient into the body. However, there are innovative approaches that can solve this health-related problem—one that is worth billions. The German-Canadian company BioNxt Solutions is currently developing sublingual drug films. These products dissolve directly under the patient's tongue, thereby solving many of the problems facing modern medicine.

At first glance, this may sound trivial, but these issues are enormous when it comes to injections and tablets. For example, weight-loss injections like those from Eli Lilly or modern MS therapies offer a major advantage: 100% of the active ingredient is absorbed. However, they also have major drawbacks, as many people are reluctant to give themselves injections—the psychological barrier is high. In practice, this leads to poor adherence to treatment or even discontinuation of therapy. Last but not least, most modern peptide therapies require a continuous cold chain. A thin film, on the other hand, is very stable and dry, which saves both time and money.

Tablets also have many drawbacks, which is particularly true for patients with neurological conditions such as multiple sclerosis or Parkinson's disease. Due to difficulty swallowing, they can take tablets only with great difficulty or not at all. The active ingredient films from Bionxt Solutions elegantly solve this problem—they do not even require water. Added to this is the issue of how tablets take effect. After ingestion, they pass through the stomach and liver before entering the bloodstream. In the process, part of their effectiveness is lost. The positive effects can be negatively influenced by factors such as previously consumed meals. BioNxt's oral dissolvable film, on the other hand, dissolves in seconds, and the active ingredient diffuses directly through the oral mucosa into the veins. As a result, a much lower dose of the active ingredient is often required to achieve the same therapeutic effects. Last but not least, the risk of gastrointestinal side effects is reduced.

However, as we all know, money plays a crucial role in healthcare systems. And here, too, a thin film can work wonders. For example, the University of Missouri conducted systematic cost-benefit analyses comparing active-ingredient films with injectable treatments and calculating real-world costs over the course of treatment, including doctor visits, lost work time, and materials. According to these analyses, the sublingual delivery method is more than 50% less expensive for the healthcare system, primarily because it eliminates the need for doctor's appointments and the complex logistics associated with injectable therapies.

BioNxt Solutions thus has a game-changer on its hands to help patients and establish itself in markets worth billions. The company has already secured its position by obtaining a European unitary patent (EP4539857) covering all 18 member states. Additionally, eight more countries in Eurasia are included. For the US market—the world's largest and most expensive—a fast-track procedure has already been initiated.

To kick things off, BioNxt Solutions is collaborating with its German partner Gen-Plus on a GLP-1 active ingredient film. This is the class of substances behind the weight-loss drugs Ozempic and Wegovy. This market is currently the most dynamic in the world.** The first phase involves six to nine months of formulation work and proof-of-concept studies. At the same time, the company is preparing for a human bioequivalence study with cladribine (BNT23001) for multiple sclerosis. The company is also exploring applications for psychedelic compounds.

As a result, BioNxt Solutions has several strings to its bow to establish its technology. The company is currently valued at just CAD 48 million on the stock market. After peaking in mid-2025, the share price has fallen to nearly one-third of its previous level. At the current price level, the company also completed a capital increase that raised CAD 2 million. The stock therefore currently offers bold investors the opportunity to buy in at a low price.

At the 17th IIF, CEO Hugh Rogers and Head of R&D Wolfgang Wagner outlined BioNxt Solutions' strategy.

https://www.youtube.com/watch?v=C_jKcnFSPe8

BioNTech: Cancer Is the Target

From the very beginning, BioNTech wanted to defeat cancer. mRNA technology was chosen as the method of choice. But then the pandemic intervened. This detour, however, has proven to be extremely lucrative. Today, thanks to the Comirnaty vaccine, the Mainz-based company has well-stocked coffers holding more than EUR 16 billion in cash and securities. With these funds, BioNTech aims to bring cancer drugs for ten indications to market by 2030. There is likely hardly any other biotech company today with these financial resources and such a large pipeline.

The focus is squarely on the oncology pipeline. Among the most promising candidates is BNT327. It is a bispecific antibody targeting PD-L1 and VEGF-A that is currently being tested in Phase 2 and Phase 3 trials for various tumours, including breast and lung cancer. Internally, it is considered the most important project for future combination therapies. Over the course of the year, initial small-scale efficacy and safety updates from the early Phase 1/2 combination studies are expected. In addition, BioNTech is currently preparing to launch large-scale, global registration studies (Phase 3) to expand the data set beyond the Chinese data collected so far.

In addition, the company is developing BNT122, a personalized mRNA cancer vaccine, in collaboration with the US company Genentech. It is currently in Phase 2 trials for the treatment of colorectal cancer and pancreatic cancer. Last but not least, a therapeutic cancer vaccine against advanced melanoma (BNT111)—commonly known as "black skin cancer"—is also in advanced clinical trials.

BioNTech's stock suffered a major setback in the spring after co-founders Uğur Şahin and Özlem Türeci announced they would depart the company by the end of 2026. Since then, the share has been attempting to find a bottom. The current market capitalization of approximately EUR 19.5 billion exceeds the company's cash reserves by more than EUR 2 billion. Investors who buy in now are betting on success in oncology. The potential is enormous: the markets for breast and lung cancer are estimated to total more than EUR 70 billion. They are expected to roughly double by 2033.

With Vertex Pharmaceuticals, investors are betting on one of the most innovative biotech companies with a solid pipeline. The German company BioNTech also operates in this category, albeit on a significantly smaller scale in terms of market capitalization. Its broad cancer pipeline could revolutionize the market. With the small-cap company BioNxt Solutions, investors are betting on a technology that could replace injections and tablets with active-ingredient films. Given its low market capitalization, the potential is enormous.