Vertex Pharmaceuticals is a global biotechnology company that invests in scientific innovation to create transformative medicines for people living with serious diseases. It is best known for its innovative treatments for cystic fibrosis (CF), a life-threatening condition that causes sticky, tar-like mucus to build up in the lungs and other organs. But the model it developed for research in CF has led to the development of other innovative treatments.
Its success stems from an unwavering commitment to research and development (R&D) and understanding of causal human biology to course correct a disease. The company believes that the true value in the industry lies in scientific innovation, which is why three out of every five Vertex employees work in R&D.1
David Altshuler, Vertex’s chief scientific officer for ten years, says: “Every dollar we don’t invest in sales and marketing is another dollar we can invest in R&D. At Vertex, everything started with our corporate strategy, we made sure it was designed to enable serial innovation.” This is not hyperbole. Innovative medicines are defined as those with an active substance or combination of active substances that have not been authorised before. Vertex has launched five since 2012. The total will be seven if the US Food and Drugs Administration (FDA) approves its two newest potential treatments.
Transformative advances in cystic fibrosis treatment
Vertex’s pioneering CF programme followed the 1989 discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which controls salt and water flowing in and out of cells. Affecting tens of thousands of people worldwide, CF is caused by a defective or missing CFTR protein.
The CFTR discovery generated global excitement as the first disease-causing gene to be identified in any condition. But, as Altshuler points out, the following decades highlighted the wide gulf between identifying a genetic problem and knowing how to fix it.
We are relentlessly working to bring more medicines to patients. We are already working on programmes that are not just for 2025, but 2030 and 2040
Pre-existing medicines such as antibiotics and steroids treated the symptoms of CF such as infections and inflammation, but the R&D vision was to address the genetic defect by addressing the underlying cause of CF by restoring the function of the CFTR protein. Developing its CF therapies, Vertex screened more than one million molecules, designed and synthesised more than 30,000 molecules and ran more than 150 clinical trials in over 10,000 patients.2
In 2012, the European Medicines Agency (EMA) approved Vertex’s first medicine targeted at the underlying cause of CF; it treated 4% of the patient population.3 But R&D laid the foundations for a string of three more transformational therapies. By 2019 the company had developed four medicines that were capable of treating approximately 90% of CF patients. Given this progress, they are also looking to develop an mRNA therapy, to support the less than 10% of patients with CF who do not benefit from small molecule CFTR modulator therapies.
There are few comparable recent advancements - the development of powerful HIV drugs in the 1990s is one. HIV/AIDS had been a death sentence. Like the CFTR medicines, the new HIV therapies did not cure, but they turned a terminal illness into a chronic, manageable one.4 Between the 1970s and 1990 life expectancy rose to about 30 due to nucleoside reverse transcriptase inhibitors.
Altshuler says: “Innovative treatments for cystic fibrosis have changed patient outcomes over time. Estimates have shown that the median age of survival, for patients with CF relying on supportive care alone, would be 38 years.5 With early adoption of new standard of care therapies before the age of 12, some patients could live up to their 80s.”6
Vertex has applied its unique R&D strategy to discover potential treatments for other serious diseases:
- Sickle cell disease & transfusion-dependent beta thalassemia: both diseases are serious and lifelong inherited blood diseases, which can cause damage to, or failure of, multiple organs in the body. They are caused by mutations in the HBB gene and require lifelong treatment, ultimately leading to a decreased quality of life and reduced life expectancy.7
- Pain: there hasn’t been a new class of acute pain medicine in more than 20 years and Vertex believes it can redefine the treatment of pain with a new mechanism of action. Vertex is investigating small molecules that aim to inhibit selected sensory nerves and prevent pain signals from travelling to the brain to potentially alleviate both acute and chronic pain.
- APOL1-mediated kidney disease: a genetic condition linked to two variants of the APOL1 gene. It can cause rapid progression to kidney failure, characterised by fatigue, lower limb swelling and weight gain.8
- Type 1 diabetes: linked to genetic, immune and environmental factors, type 1 diabetes results in an absence of insulin producing cells. Vertex is investigating multiple treatment approaches.9
Vertex strives to take on the impossible, it is developing the first non-opioid acute pain treatment in more than two decades, and has set a goal to change the paradigm of pain management. “We’re incredibly focused on serial innovation. If something has never been done before, many companies will say it cannot be done, but at Vertex, we see it as an opportunity,” says Altshuler .
“Vertex is in a very interesting and exciting position. Each step forward requires more energy and even more hard work from our world-class team. Over the last 10 years, we have shown that we’re not only able to discover and develop medicines for CF. We have shown that our R&D strategy can work. But of course, we are relentlessly working to bring more medicines to patients. We are already working on programmes that are not just for 2025, but 2030 and 2040. We plan to keep helping patients decades from now.”
For more information please visit vrtx.com/en-gb
References:
1 2023-Vertex-Corporate-Responsibility-Report. Available-at:-https://www.vrtx.com/sites/global/files/2023_Vertex_Corporate_ Responsibility_Report.pdf
2 Vertex Pharmaceuticals: Humanizing drug discovery. Available at: https://www.nature.com/articles/d42473-020-00303-9.
3 Vertex Receives European Approval for ivacaftor, the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People With a Specific Genetic Mutation (G551D). Available-at:-https://investors.vrtx.com/news-releases/news-release-details/vertex-receives-european-approval-kalydecotm-ivacaftor-first
4 Alum EU et al. Toward a cure - Advancing HIV/AIDs treatment modalities beyond antiretroviral therapy: A Review. Medicine (Baltimore). 2024 Jul 5;103(27):e38768.
5 National Institute for Health and Care Excellence (NICE), Jul 24. Ivacaftor-tezacaftor-elexacaftor,tezacaftor-ivacaftor and lumacaftor-ivacaftor for treating cystic fibrosis. Available at: https://www.nice.org.uk/guidance/ta988/resources/ivacaftortezacaftorelexacaftor-tezacaftorivacaftor-and-lumacaftorivacaftor-for-treating-cystic-fibrosis-pdf-82615917119173
6 Lopez A et al. Elexacaftor/tezacaftor/ivacaftor projected survival and long-term health outcomes in people with cystic fibrosis homozygous for F508del. J Cyst Fibros. 2023 Jul;22(4):607-614.
7 Angastiniotis M, Lobitz S. Thalassemias: An Overview. Int J Neonatal Screen. 2019 Mar 20;5(1):16.
8 Pollak MR, Friedman DJ. APOL1 and APOL1-Associated Kidney Disease: A Common Disease, an Unusual Disease Gene - Proceedings of the Henry Shavelle Professorship. Glomerular Dis. 2023 Jan 25;3(1):75-87.
9 Holt et al. The management of type 1 diabetes in adults. A consensus report by the American Diabetes Association (ADA) and the European Association for the Study of Diabetes (EASD). Diabetologia. 2021.
