Innovations Advanced by M&A: Breakthroughs for a Rare Blood Disease

Jun 26, 2024 | Blog Post, Innovations Advanced by M&A

This blog is another installment in a series, Innovations Advanced by M&A, that underscores the importance of mergers and acquisitions in ensuring a competitive and vibrant biopharmaceutical landscape for patients. You can find previous blogs in this series here.

Life sciences companies of all sizes count on mergers and acquisitions (M&A) to be able to connect their promising breakthroughs with the resources, expertise and infrastructure needed to develop new medicines and deliver them to patients. In one of many examples from across the United States’ vibrant life sciences ecosystem, the merger between Alexion Pharmaceuticals and AstraZeneca helped accelerate the development and global distribution of transformative treatments for paroxysmal nocturnal hemoglobinuria (PNH) – a rare and life-threatening blood disease.

Alexion was born out of a conversation between Yale physician Leonard Bell and his friend, Steven Squinto, in the ice cream aisle of a New Haven, CT, grocery store in 1992. The pair believed that therapies which blocked the complement system – a complex set of enzymes within the immune system – represented a potentially transformative approach to treating many inflammatory conditions. Although Alexion researchers found the effect of this new class of medications in treating more common ailments like rheumatoid arthritis and kidney disease was underwhelming, they quickly discovered a niche for this promising science: treating rare diseases like PNH.

PNH affects just 3,000-6,000 people in the United States. It is a genetic condition which makes red blood cells susceptible to attacks by the complement system, breaking them apart and releasing “free” hemoglobin into the bloodstream. Hemoglobin is an important protein that allows red blood cells to transport oxygen and gives blood its characteristic red color. However, when hemoglobin is separated from red blood cells, it can cause life-altering, even life-threatening, complications for patients with PNH, including fatigue, anemia, blood clots and kidney disease.

15 years after Alexion first set out to bring a new medicine to patients, the company’s leading complement inhibitor, Soliris, was approved in 2007 as the first disease-modifying treatment for PNH. A decade later, Alexion secured FDA approval for Ultomiris, a similar complement inhibitor that is administered less frequently than Soliris. Together, these therapies have contributed to a dramatic increase in life expectancy and quality of life for those living with PNH. 

Despite these successes, the small and widely dispersed PNH patient population led to challenges for Alexion’s ability to reach those who would benefit most from their innovative therapies. In an effort to expand the global reach of their pipeline, Alexion was acquired by AstraZeneca in 2020.

“Combining AstraZeneca’s capabilities in precision medicine and Alexion’s expertise in rare-disease development and commercialization will enable the new company to develop a portfolio of medicines addressing the large unmet needs of patients suffering from rare diseases.”

– AstraZeneca, 2020 (press release)

Today, Alexion continues to operate as a wholly owned subsidiary of AstraZeneca and remains focused on bringing new treatments to patients living with rare diseases. By combining Alexion’s existing scientific expertise with AstraZeneca’s established resources and distribution infrastructure, the merger has allowed the companies to more effectively reach people living with PNH.

For example, building on Alexion’s initial success in complement inhibitors, the combined company recently secured FDA approval for Voydeya, a first-in-class Factor D inhibitor to treat extravascular hemolysis – a serious complication that affects just a narrow subset of patients undergoing treatment for PNH.

“For nearly 30 years Alexion has worked to develop and deliver transformative medicines to patients around the world with rare and devastating diseases…  We bring to AstraZeneca a strong portfolio, innovative rare disease pipeline, a talented global workforce and strong manufacturing capabilities in biologics. We remain committed to continuing to serve the patients who rely on our medicines and firmly believe the combined organization will be well positioned to accelerate innovation…”

Dr. Ludwig Hantson, CEO, Alexion

Today, there are more than 7,000 known rare diseases, yet just 5% currently have an FDA-approved treatment. As illustrated by the merger between Alexion and AstraZeneca, M&A plays a vital role in allowing life sciences companies to leverage their complementary resources, infrastructure and expertise to bring new innovations to patients – particularly in the face of the unique challenges associated with developing and delivering rare disease therapies. Therefore, policymakers must take a balanced and bipartisan approach to merger enforcement and preserve this vital avenue for companies to bring new treatments and cures to the patients who need them most.