Unfazed by recent cardio setbacks, Novartis takes another PhIII challenge head-on with Akcea drug
In the latest move of its long wrestle with the giant cardio market, Novartis is mounting a late-stage campaign for a lipoprotein(a)-lowering drug developed by Akcea.
The pharma giant is going for an option to take over Phase III development three months after the closely held Ionis affiliate unveiled Phase II data that showed its drug, then called AKCEA-APO(a)-LRx, could slash Lp(a) levels significantly in at-risk cardio patients.
There’s no mention of any financials in the press release, but according to the terms spelled out in their 2017 pact, Akcea stands to pocket $150 million for the license fee.
Novartis is now projecting big things for the asset, which it has renamed TQJ230.
“No treatments are currently available to substantially lower Lp(a). People with this inherited risk factor are facing cardiovascular risks that cannot be addressed effectively with lifestyle changes,” said John Tsai, head of global drug development. “We’re excited about the novel, RNA-targeting approach that could be a game-changer for people with elevated Lp(a). If our Phase 3 trial succeeds, we expect that TQJ230 will become the leading treatment option and another pillar of our longstanding commitment to re-imagining cardiovascular medicine.”
This will mark another major cardio effort after canakinumab, a key anti-inflammatory drug that Tsai’s predecessor (and current CEO) Vas Narasimhan touted as a blockbuster in the making, only to pivot quickly to cancer when the FDA batted the application back. While Novartis supplied evidence that its drug reduced the likelihood of major cardiovascular events like heart attack or stroke in a large subgroup of patients, it didn’t quite make the cut as a targeted therapy.
And that, in turn, followed the collapse of its attempt to get its other cardio drug serelaxin approved.
For a potential mass market indication like elevated Lp(a), having a fairly clean safety profile is critical, Stifel analysts wrote in a note calling the opt-in a “validating event” for Ionis/Akcea’s platform tech, which is designed to augment the potency of antisense drugs.
From Novartis’ vantage point, we’d imagine that ahead of executing the Akcea deal, they were already comfortable with the potential benefits of lowering Lp(a) – thus the ph2, and the decision to opt-in, was likely largely about whether or not a clean-enough profile was established. (…) while the ph2 data weren’t pristine, elimination of thrombocytopenia and renal function issues suggests that LICA can go a long way in widening the therapeutic index of antisense drugs.
TQJ230 will now test the theory surrounding Lp(a) as a biomarker for cardio risk — and Novartis’ ability to turn hype into real commercial results.
At the American Heart Association Scientific Sessions last November, Akcea reported that around 98% of patients in the 20mg weekly cohort and 81% in the 60mg every four weeks group achieved “clinically significant reductions in Lp(a) levels bringing them below the recommended threshold of risk for CVD events (<50 mg/dL).”