Novartis suffered a second trial disappointment within a week, after the Swiss pharma group’s potential blockbuster heart drug failed in a final-stage study.
The drug, pelacarsen, did lower levels of lipoprotein(a), a largely genetic risk factor for cardiovascular disease that affects roughly one in five people. However, that reduction did not translate into fewer deaths, heart attacks and strokes compared with placebo, the Swiss drugmaker said late on Sept 4.
The failure marks a major setback to hopes the medicine would open up a new approach to preventing cardiovascular disease. It also comes just days after Novartis temporarily paused trials of an experimental cell therapy for autoimmune diseases after three patients died.
Novartis shares fell as much as 3.9 per cent on Sept 7 in early Zurich trading, the biggest decline in over four months. They have gained 14 per cent so far this year. Amgen, which is developing a similar heart drug, fell almost 7 per cent in post-market trading on Sept 4.
“This questions whether any Lp(a)-lowering therapies can ultimately show a cardiovascular benefit,” according to Michael Leuchten, an analyst at Jefferies. Any negative reaction might be overstated, as even a successful trial would have raised questions over which patients would benefit and how well the drug could compete, he added.
Novartis is counting on a string of new medicines to sustain growth when some of its biggest products lose patent protection. Chief executive officer Vas Narasimhan has said successful late-stage trials could allow the company to raise its forecast for 5 to 6 per cent annual sales growth till 2030.
Analysts had seen pelacarsen as a potential blockbuster, with peak sales estimates ranging from about US$1.5 billion (S$1.9 billion) at UBS to US$5.4 billion at Jefferies, though the investment bank assigned the drug just a 30 per cent probability of success. Novartis had pegged the overall market opportunity at more than US$5 billion.
The next big pipeline test is an experimental injection designed to tackle the genetic cause of DM1, a muscle-wasting disease, rather than just treating its symptoms. The treatment, del-desiran, is also the centrepiece of Novartis’ US$12 billion Avidity acquisition.
New approaches
The two setbacks carry significance beyond the individual drugs. Novartis had positioned both pelacarsen and rapcabtagene autoleucel, or rap-cel, as potential ways to reshape treatment in their respective fields: one by targeting a previously untreatable inherited cardiovascular risk and the other by bringing CAR-T cell therapy into autoimmune disease.
The pelacarsen disappointment follows a mixed week for Novartis last week, when one of its experimental treatments did deliver on promises, providing some relief. The company’s multiple sclerosis pill remibrutinib succeeded in two late-stage trials, sending the shares up by more than 6 per cent on Sept 1.
Novartis disclosed few details of the pelacarsen failure, including how close it came to meeting the trial’s goals or whether some patients benefited more than others. The company said full results will be presented at an upcoming medical meeting. BLOOMBERG
