In a major shift, Agios Pharmaceuticals will sell a portfolio of cancer medicines that it has spent more than half a decade developing to French drugmaker Servier, pivoting to focus on a blood disease treatment and emerging research into rare genetic disorders.
Servier will pay Agios $1.8 billion in cash upfront for the Cambridge, Massachusetts-based biotech's approved leukemia drug Tibsovo, as well as a pipeline of three experimental cancer therapies. Should the most advanced of those three win U.S. approval, Agios could receive another $200 million.
Approximately 200 employees primarily involved in cancer research or sales at Agios — about 35% of the company's workforce — will receive an offer to work for Servier, the companies said in a statement Monday announcing the deal.
For Agios, the sale is a significant change in focus. Founded in 2007 to explore cellular metabolism and its role in disease, Agios successfully developed two cancer drugs that won U.S. approvals in 2017 and 2018, respectively. The first, called Idhifa, was licensed to Celgene and then absorbed into Bristol Myers Squibb when the pharma bought Celgene last year. Agios markets the second, Tibsovo, sales of which totaled $82 million through the first nine months of 2020.
By most measures in an industry where research failure is the norm, Agios is a success story. But the cancers for which Idhifa and Tibsovo are approved — several types of acute myeloid leukemia — are uncommon and neither drug is expected to become a top-seller.
Agios has also weathered criticism for high spending with little in the way of returns for investors. Shares now trade at about $33 apiece, similar to their price in early 2014. The company has never made an annual profit.
Selling off the cancer business is a reset, but one consistent with Agios' research background, according to company CEO Jackie Fouse.
"Our scientific platform in cellular metabolism has not changed," Fouse said in an interview with BioPharma Dive. "We've seen an acceleration of our research platform around mechanisms that we think can apply outside of oncology."
Foremost in those efforts is a drug called mitapivat, which is designed to turn on an enzyme that red blood cells use to change sugars into energy. Agios is developing the drug for three blood diseases and, in December, reported positive Phase 3 results in the first, a rare hereditary condition known as PK deficiency.
Agios plans to ask the Food and Drug Administration for approval next year, and expects clearance could come in 2022.
"Our return to a commercial-stage company is not that far away," Fouse said.
Importantly, results from earlier-stage studies reported this year suggest the drug could also treat thalassemia and sickle cell disease, which are more common than PK deficiency and represent more lucrative commercial opportunities. Two Phase 3 trials of mitapivat in thalassemia and a third study in sickle cell are planned for 2021.
"Differently than Tibsovo, this is a drug that can come out, ramp up and have this blockbuster potential. Tibsovo is little [more] niche," Fouse said.
A number of other biotech companies are currently developing new drugs for thalassemia and sickle cell, including cutting-edge genetic medicines that replace or edit DNA, and could prove to be significant competition. But Fouse argues small molecule drugs like mitapivat will be needed to treat both diseases, which can vary significantly from patient to patient.
"We see a really nice opportunity to do great things for patients and compete," said Fouse. "We're happy to engage in healthy competition."
Fouse said more than a dozen companies engaged with Agios during the review process, including "several" that made an offer for the biotech's cancer portfolio. Ultimately, Servier, which has recently made a concerted push to broaden its oncology presence, won out with what Fouse called a "very compelling" offer.
The $1.8 billion is a sizable sum — more than half of Agios' current market value — and gives the company enough cash to fund operations through 2025, when Agios expects to first report positive cash flow.
Agios said it plans to return at least $1.2 billion of the upfront payment to shareholders. The rest will bolster the company's balance sheet and help free it from needing to raise money.
"I firmly believe this is the best way for Agios to move into its future and to do great things for patients and I believe our shareholders will embrace that strategic rationale," Fouse said.
Per deal terms, Servier will owe royalties of 5% on sales of Tibsovo in the U.S. and 15% on any sales of vorasidenib, currently in Phase 3 testing for certain types of low-grade gliomas. Two other drugs, dubbed AG-270 and AG-636, will transfer to Servier along with Agios' preclinical cancer research.
The companies expect the deal to close in the second quarter of 2021.