Written by John Carroll

Over the last three months Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) subsidiary Akcea has executed a $1.65 billion partnership with Novartis A G (NYSE: NVS) on two of its four drugs and mapped a path to regulators on both sides of the Atlantic after its lead therapy volanesorsen successfully completed a Phase III study. Now Akcea — a specialist in lipid disorders — is shooting for a $100 million IPO, with Novartis coming in to support the move with an extra $50 million to purchase a growing equity stake.

Much of the value in the IPO will be assigned to volanesorsen, which investigators says was successful in treating rare cases of familial chylomicronemia syndrome. Founded in 2015, Akcea plans to go on to market volanesorsen alone in the U.S. and Europe, with its partners at Novartis stepping in on the next two therapies — AKCEA-APO(a)-L_Rx. and AKCEA-APOCIII-L_{Rx —} it plans to steer to regulators. Novartis first, though, has to decide if it wants to pick up options for the drugs and pay for Phase III itself...

In the Phase III investigators reported that:

• a huge drop in triglycerides was experienced among the 33 FCS patients taking the drug, hitting the primary endpoint while
• the placebo arm saw the level of triglycerides go up, helping to illustrate the treatment effect.
• 5 patients were forced out of the trial due to a threatening decline in platelet counts.
• grade 4 thrombocytopenia occurred in three patients, which ended after they stopped dosing.
• there were no withdrawals due to platelet counts after the company began monitoring the side effect, but the safety issue did not pass unnoticed.

The drug is designed to tackle a disease caused by hereditary mutations that inhibit the activity of lipoprotein lipase, needed to break down triglycerides carried by chylomicrons.

In Novartis’ original deal with Akcea in January, the Big Pharma promised $225 million in near-term payments, split between fees, an upfront and an equity stake, and promising $1.13 billion more in development and commercialization milestones.