- Global Blood Therapeutics, a California-based drug company, said Tuesday that it has licensed two sickle cell disease programs from the French pharmaceutical giant Sanofi.
- Sickle cell is an inherited disorder that causes red blood cells to take on a crescent shape and a hard, sticky structure. These misshapen cells then give rise to long-term health complications and lowered life expectancy. Currently, just a small number of drugs are approved to treat sickle cell, with one of the newest, Oxbryta, coming from Global Blood.
- Tuesday's deal further expands Global Blood's work, giving it access to research programs that harness new ways to stop cells from sickling as well as reduce inflammation and oxidative stress. Per deal terms, Sanofi will get an unspecified, initial payment and could take home around $350 million more if the drugs hit certain milestones. Sanofi is also eligible for single-digit tiered royalties on worldwide net sales of any resulting products.
Sickle cell is one of the most common inherited blood disorders, affecting an estimated 100,000 people in the United States and millions more across the globe. But that large number of patients stands in stark contrast with the relative scarcity of treatment options — a dichotomy that many say is emblematic of the racial inequalities so often found in healthcare.
Oxbryta received approval to treat sickle cell in late 2019, as did a Novartis medicine called Adakveo. And in 2017, the Food and Drug Administration authorized an oral powder meant to reduce severe complications associated with the disease. Before that powder, though, it had been nearly 20 years since the FDA cleared a new sickle cell drug for market.
And in spite of the recent progress, there remains a desperate need for more treatments and better access to them.
"Even with today's best available care, [sickle cell] continues to drive premature deaths and disability," Novartis said last month, while announcing it had received funding from the Bill & Melinda Gates Foundation to develop a sickle cell gene therapy that can be accessible for people living outside wealthy countries. Several advanced gene therapy and gene editing programs are being developed by Bluebird Bio, CRISPR Therapeutics, and others.
For Global Blood, the approval of Oxbryta was a landmark moment, handing the company its first marketed product. Yet, the drug's launch has been hindered by the coronavirus pandemic. Global Blood recorded $124 million in net sales last year, which wasn't enough to offset the company's $367 million worth of operating costs.
"Given there was a significant surge in COVID-19 cases [late last year], we weren't expecting this to be a breakout quarter for Oxbryta," wrote Benjamin Burnett, an analyst at Stifel, in a recent note to investors. After speaking to doctors, though, Burnett's team expects "once conditions improve, so too will sales."
In the meantime, Global Blood will be working on its pipeline programs. The company is currently running a couple late-stage studies that look at Oxbryta's effects in younger patients and on certain health measures, like daily physical activity.
Global Blood also expects two pivotal tests of inclacumab, a drug it licensed from Roche in 2018, to begin before the end of June. Inclacumab is meant to lower the number of times sickle cell patients experience episodes of extreme pain called vaso-occlusive crises.
Global Blood's pipeline is now further expanding with the addition of the two Sanofi programs, which the company claims could by complementary to Oxbryta.
"We envision a future in which sickle cell disease is a well-managed condition with the potential for a functional cure in the form of patient-friendly oral therapies," said Jung Choi, Global Blood's chief business and strategy officer, in a statement. "These novel discovery programs represent promising approaches that we believe may have the potential to lead to meaningful improvements for patients."
Global Blood shares were down 2% late Tuesday morning, to trade at just over $45 apiece.