- Pfizer will pay $250 million to secure rights to a Phase 2 lipid-lowering drug developed by Akcea Therapeutics as a treatment for elevated triglycerides, liver disease and diabetes.
- An affiliate of Ionis Pharmaceuticals, Akcea is pursuing a partnership with the experimental drug, called AKCEA-ANGPTL3-LRx, because of Pfizer's broader commercial reach — useful for diseases that affect millions of patients, rather than the narrower groups targeted by Akcea's on-market rare disease drugs.
- Akcea shares rose 32% Monday to trade above $20 a piece, returning them to near the levels they were before three top executives abruptly exited the company last month.
Like the rest of the Ionis and Akcea's pipeline, AKCEA-ANGPTL3-LRx treats patients by disrupting the body's production of proteins associated with a disease by preventing expression and translation by messenger RNA.
ANGPTL3, or angiopoietin-like 3 protein, regulates triglycerides, cholesterol, glucose and energy metabolism. Research has found that patients with a genetic mutation inhibiting ANGPTL3 have reduced low-density lipoprotein cholesterol and triglycerides.
Akcea's drug candidate has completed trials in patients with familial partial lipodystrophy and familial chylomicronemia syndrome (FCS), and is currently in a Phase 2 trial in patients with hypertriglyceridemia, Type 2 diabetes, and nonalcoholic fatty liver disease who need to lower their triglyceride levels.
The $250 million upfront fee will be shared by Akcea and parent Ionis, with Akcea paying its obligation by issuing Ionis more shares. This will increase Ionis' 75% stake in the company by about 1%.
Another $1.3 billion in payments are on offer from Pfizer upon achievement of development, regulatory and sales milestones, along with double-digit sales royalties, also to be divided between Akcea and its parent. Akcea retained some option rights to co-commercialize the drug with Pfizer. The big pharma will assume development costs after completion of Phase 2 testing.
Akcea successfully won approval of two drugs: Tegsedi (inotersen) for hATTR amyloidosis in the U.S. and Europe, and Waylivra (volanesorsen) for FCS, although the latter of the two was rejected by the FDA. Waylivra has not been launched in Europe yet, and Tegsedi is in a hard-fought battle for share against Alnylam's Onpattro and Pfizer's emerging Vyndaqel.
Akcea had $296 million in cash, equivalents and short-term investments as of June 30. The $250 million upfront fee should help Akcea continue to fund the launch of the two products as well as its research and development. The company spent $95 million on selling, general and administrative functions in the first six months of 2019, along with $120 million on R&D.