- Alexion Pharmaceuticals announced Wednesday it will acquire Syntimmune, paying $400 million upfront and lining up to $800 million in milestone payments in a deal that will expand the Massachusetts-based pharma's drug pipeline.
- Syntimmune's sole drug candidate is SYNT001, a clinical-stage antibody therapy with a lead indication for a rare hematology disease. Alexion executives said on a Wednesday morning conference the company will announce a second indication for testing in 2019.
- Pending regulatory approval, the deal is expected to close in the fourth quarter of 2018. Alexion's stock jumped 5% in the first hours of trading Wednesday.
This deal is a big bet on SYNT001 and, more broadly, the anti-FcRn drug class.
"We are excited to help shape the FcRn landscape," CEO Ludwig Hantson said on the Sept. 26 call.
Hantson said Alexion sees "potential for broad application across a number of indications" for SYNT001. A related company presentation listed potential indications in neurology, hematology, nephrology, rheumatology and dermatology.
While a second indication will be announced next year, SYNT001 will keep a lead indication in warm autoimmune hemolytic anemia (WAIHA).
WAIHA affects roughly 65,000 people in the U.S. and major European markets. There are no approved treatments for the rare disease, and the anemia and other complications can be life-threatening.
An ongoing Phase 1b/2a trial is expected to readout in the first half of 2019, execs said.
Alexion R&D head John Orloff gave interim safety data from that study Wednesday, saying there have been no severe adverse events reported so far. The most common adverse event was headache, typical for the drug class according to Orloff, and all cases were mild or moderate.
The acquisition bolsters Alexion's pipeline, which was a top priority of Hantson's for 2018. With that as an aim, the biotech spent $855 million in April of this year to buy Wilson Therapeutics and its experimental treatment for Wilson's disease.
Alexion's only other drug candidates are a long-acting C5 inhibitor called ALXN1210 and a preclinical C6 complement inhibitor.
The company has three rare disease drugs on market, led by its blockbuster Soliris (eculizumab).
In other news for Alexion, the company released Phase 3 data Monday on Soliris for neuromyelitis optica (NMO), showing a 94% reduction in risk of relapse compared to placebo. The strong efficacy primes the drug for a fourth indication by mid-to-late 2019. Market consensus sales figures have Soliris growing in NMO to $389 million by 2023.
Investment bank Jefferies had a more tempered view of the Soliris data, arguing high pricing will lead to "quite limited" use in NMO. Analyst Eun Yang pegged Soliris NMO sales to reach $242 million by 2023, more than a third below the consensus forecast, according to a Sept. 24 Jefferies note to investors.
Overall, while consensus sales for Soliris are expected to grow to $5.4 billion by 2023, Jefferies predicts they will be about 30% lower at $3.8 billion.
Correction: This article has been updated to reflect Alexion's recent move from Connecticut to Massachusetts.