France's largest drug company is in the middle of a transformation. Late last year, shortly after bringing on a new CEO, Sanofi announced plans to exit the heart and diabetes research that had long been its calling card. In its place, the company put greater emphasis on developing drugs for cancer, immune diseases and uncommon blood disorders.
Months later, Sanofi executives say they're optimistic the company will take a leading position in these research areas. But convincing others has been more of a challenge, in part because of Sanofi's mixed track record.
In blood diseases, one of two hemophilia products Sanofi got after spending $12 billion to acquire a company called Bioverativ is now quickly losing ground to rival therapies. In cancer, Sanofi and longtime partner Regeneron were sixth to bring a certain kind of immuno-oncology drug to market, arriving well after heavyweights from Merck & Co. and Bristol Myers Squibb.
Sanofi does have one clear success to tout: Dupixent, an immune system-regulating drug also developed alongside Regeneron. In less than two years, the drug has become Sanofi's biggest seller. It earned more than $2 billion in sales in 2019, and is on track to eventually deliver upwards of 10 billion euros annually, according to the company.
Sanofi executives know they can't rely solely on Dupixent, though. In December, the company outlined six experimental drugs that could be meaningful additions to its business. And on Tuesday, a virtual research and development event helped detail the progress it's made over the past six months.
"We need to look back in a few years and start saying: Did we really change how patients are treated with some of the work we've done? I think the answer is going to be yes," Sanofi CEO Paul Hudson told reporters ahead of the event, which also spotlighted progress the company is making on its coronavirus vaccine research.
One of the six priority drugs is an enzyme inhibitor called venglustat that's currently in mid- to late-stage testing for three different rare diseases. Sanofi said Tuesday that it could submit the drug for approval in each of the diseases in 2023.
Two other priority drugs, named fitusiran and BIVV001, are in late-stage testing as hemophilia treatments. If those studies read out positive, the company envisions submitting the drugs for approval in the second half of 2021 and the first half of 2022, respectively.
Sanofi also called out a drug that wasn't on its December list. Known as THOR-707, the drug was created using the synthetic biology platform that Sanofi acquired through its recent $2.5 billion purchase of San Diego-based Synthorx.
Sanofi disclosed that early data from a small clinical study suggest THOR-707 increases tumor-fighting white blood cells without activating other white blood cells that cause unwanted immune responses. Sanofi expects detailed results from the study by next year.
John Reed, Sanofi's R&D head, claimed this data was what pushed Sanofi to acquire Synthorx, and said the drug could be an anchor for his company's cancer drug portfolio to build around — serving as an " excellent combination partner" both for current treatments like Libtayo and Sarclisa and for future products.
"I think we're picking up momentum," Reed told reporters Monday, pointing to the development of Libtayo, Sarclisa and experimental drugs for breast and lung cancers.
"These are good anchoring positions, but we're rapidly building around that," he added. "Certainly my mission when I came here was to get the company squarely into a competitive position in oncology."
Whether Sanofi can achieve such a position is unclear. Sarclisa just gained approval for multiple myeloma in March, while Libtayo is also a relative newcomer. During the first quarter, Regeneron recorded $75 million in net Libtayo sales, a small fraction of what similar drugs were earning.
Pulling ahead will likely be difficult in other markets too. Hemophilia, for instance, has become very competitive in a short period of time, thanks to a longer-lasting option in Roche's Hemlibra and rapidly advancing gene therapies. BioMarin, a California-based biotech, could receive approval in August for the first one-time hemophilia A treatment, which analysts expect to erode sales of currently marketed drugs.
Still, Sanofi executives see room in the hemophilia market for both BIVV001, a new kind of once-weekly drug developed by Bioverativ, and fitusiran, a once-monthly RNA interference therapy developed by Alnylam Pharmaceuticals.
Specifically, Hudson said that patients who want to be more active will gravitate to BIVV001, while patients who prioritize convenience will find value in fitusiran.
"We know already that Hemlibra, which was progress, is simply not a real monthly," Hudson said. Fitusiran, meanwhile, will "break new ground."
On a broader level, the next few years will be a critical test of Sanofi's ability to develop new drugs without the help of Regeneron, which gave rise to Dupixent and Libtayo, among other drugs. Last month, Sanofi chose to further loosen ties with its collaborator, selling off $11 billion worth of Regeneron shares to have more cash for expanding the company's pipeline through internal R&D and acquisitions.
Reed appeared to confirm this on the call with reporters, noting how Sanofi is putting a "sizable percentage" of its internal research budget into oncology while also supplementing the pipeline with deals each year.
Sanofi now forecasts at least three cancer drug candidates coming into its pipeline every year, according to Reed.
To help it bear the added R&D weight, Sanofi is investing across a variety of drugmaking technologies like the synthetic biology developed at Synthorx. It's a strategy that other companies like Vertex, which has made investments in cutting-edge fields like CRISPR gene editing and messenger RNA technology, have found successful as they chart out the next steps for their pipelines.