SoCal's Ionis Pharma Stock Jolted by $1.6 Billion Cardio Drug Deal With This Big Pharma

January 6, 2017
By Alex Keown, BioSpace.com Breaking News Staff

CARLSBAD, Calif. – Shares of Ionis Pharmaceuticals are up more than 8 percent in pre-market trading after the company announced it entered into an agreement with Novartis that could be worth more than $1 billion.

Ionis and its subsidiary Akcea Therapeutics, will develop treatments targeting high levels of lipoprotein to reduce cardiovascular use with AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx, Novartis’ therapies that could reduce lipoprotein by 90 percent.

In a Phase I/IIa study in healthy volunteers with elevated Lipoprotein(a), AKCEA-APO(a)-LRx produced “significant and sustained reductions in Lp(a) of up to 97 percent,” the company said. ApoC-III is a protein produced in the liver that plays a central role in the regulation of serum triglycerides. It is currently being evaluated in a Phase I/IIa study in healthy volunteers with elevated triglycerides, according to company information.

Under terms of the deal, Ionis and Akcea are eligible to receive $225 million in near-term payments, including an immediate $75 million up-front option payment and a $100 million equity investment in Ionis. For each drug, upon option exercise, Novartis will pay Ionis and Akcea a $150 million license fee. The Swiss-based company will also initiate a global Phase III cardiovascular outcome study in a high-risk population and will be responsible for worldwide development and commercialization activities. Ionis and Akcea are also eligible to receive up to $315 million and $265 million in development and regulatory milestone payments for AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx, respectively, as well as up to $285 million and $265 million in commercialization milestone payments, for each drug, respectively, the company said in its statement.

When licensing fees, royalties and milestone payments are factored in, the deal could be worth more than $1 billion for Ionis, if both drugs are licensed and successfully commercialized, the company said.

“We are advancing our pipeline of novel drugs to treat previously inadequately treated lipid disorders by pursuing indications that drive the greatest near- and long-term value. This strategic partnership allows us to move more rapidly to Phase III cardiovascular outcomes studies with both therapies than our original development plan. Our ability to benefit from Novartis' global commercialization resources and complement them with Akcea's specialized sales force focused on lipid specialists should allow us to maximize the commercial potential of each drug,” Paula Soteropoulos, chief executive officer at Akcea Therapeutics, said in a statement.

Ionis said it will conduct a Phase II dose-ranging study for each drug, to choose the optimal dose and evaluate alternative dose schedules, such as monthly dosing, for a planned Phase III study.

Ionis is no stranger to developing strong partnerships with other pharma companies. Last year the company received a $75 million payment from Biogen for successful Phase III data of their joint investigational treatment for spinal muscular atrophy. In December, the U.S. Food and Drug Administration approved Spinraza, which is the first drug of its type approved for the rare disease.

In July 2016, the company struck a deal worth up to $810 million with Janssen Biotech for the development of its oral gastrointestinal treatment, IONIS-JBI1-2.5. IONIS-JBI1-2.5, is antisense drug designed to locally inhibit an undisclosed target in the gastrointestinal (GI) tract. This is the first time the two companies have collaborated.

Ionis, formerly known as Isis, has several RNA-targeting drugs in late-stage development, including volanesorsen for patients with either familial chylomicronemia syndrome or familial partial lipodystrophy.