JPM23: Ipsen CEO happy to take on Big Pharma in rare diseases but says gene therapy is rife with risk

With its recent $952 million deal for Albireo, Ipsen joined a growing list of biopharma companies doubling down in rare diseases. And despite increasing Big Pharma investments in the area, Ipsen CEO David Loew believes his company can hold its own as a midsize player.

Ipsen is “not at all afraid” of competition from Big Pharma companies, Loew, a former Sanofi Pasteur chief, said in an interview last week on the sidelines of the J.P. Morgan Healthcare Conference. “What really matters is to be focused, to be excellent in execution.”

The chief executive pointed to Ipsen’s top seller, Somatuline, which is used to treat the rare hormonal disorder acromegaly and the rare cancer-related carcinoid syndrome, as well as endocrine tumors. The drug has been more widely used than Novartis’ Sandostatin, Loew said.

But the biggest problem for Ipsen these days is Somatuline’s loss of patent protection. In the third quarter of 2022, Somatuline sales dropped 10% year-over-year to 312 million euros.

That’s why Ipsen, a company without an internal research engine, has been busy striking deals. Ipsen added 16 new assets over the past 18 months through dealmaking, the company said at JPM.

The Albireo deal is centered on Bylvay, the first approved treatment for progressive familial intrahepatic cholestasis, a rare inherited liver condition. The bile acid modulator is also in clinical testing for other rare liver diseases.

Bylvay is a nice fit with Ipsen's elafibranor, Loew said. Ipsen licensed elafibranor from Genfit late 2021 as part of a long-term collaboration, with a phase 3 readout in previously treated primary biliary cholangitis expected in early 2023. In addition, Ipsen also holds certain ex-U.S. rights to Exelixis’ liver cancer drug Cabometyx.

Although rare diseases and liver disease are “quite attractive” to Ipsen, Loew said Ipsen is “agnostic” in terms of rare disease indications. But there’s one therapy modality that Loew doesn’t want Ipsen to enter just yet.

“What we will not do right now is go on gene therapy, because we think it’s still charged with quite a lot of risk,” Loew said, pointing to multiple setbacks and safety-related hiccups seen in the field recently.

“Science will come at one point ... where gene therapy or gene editing could become a new tool, and then we have to see, what is the time point we would get into this,” Loew said. “But for now, that’s not inside of our strategy.”

Loew joined Ipsen mid-2020 and quickly outlined a vision for the drugmaker to be a “leading global mid-size biopharmaceutical company” with a focus on oncology, rare diseases and neuroscience.

In neuroscience, Loew is more cautious about where Ipsen intends to play. Specifically, he said Ipsen will focus on niche conditions rather than large indications like Alzheimer’s disease. In a blow to Ipsen in this space, its mesdopetam just flunked a phase 2b trial. Ipsen got the drug mid-2021 through a deal with Swiss company IRLAB. The targeted indication is for uncontrolled movements in Parkinson’s disease patients, but the mid-stage study found the drug failed to significantly improve the number of hours participants with Parkinson’s experienced dyskinesia.

Meanwhile, Ipsen also expanded its oncology offerings last year with a $247 million acquisition of Epizyme. The deal gave Ipsen the lymphoma drug Tazverik.

Cell therapies and bispecific T-cell engagers have shown impressive efficacy in lymphoma, but Loew, who once served as Roche’s oncology head, thinks Tazverik has a role to play.

As Loew sees it, oncology has two distinct segments divided by large academic centers and community doctors. CAR-T cell therapies and T-cell bispecific antibodies require specialized knowledge and infrastructure, partly because of the cytokine release syndrome side effect they may trigger. But many patients, especially elderly patients, aren’t fit for these therapies. Tazverik, as an oral treatment, is “ideally placed to play a role there,” Loew said.

Tazverik won an FDA accelerated approval in previously treated follicular lymphoma in 2020, and Ipsen is testing the drug in combination as a second-line treatment.

Editor's Note: The story has been updated to reflect that mesdopetam has failed a mid-stage clinical trial.