Novartis bets up to $7.8B on Abogen’s mRNA autoimmune therapy
Novartis is paying China’s Abogen Biosciences $575 million (€512 million) upfront for global rights to ABO2203, an experimental treatment that uses mRNA to make a B-cell-depleting drug inside the patient’s body.
Why it matters: The approach could offer a simpler way to target the B cells that drive some autoimmune diseases. It aims to achieve deep B-cell depletion without collecting and engineering patients’ immune cells, as conventional CAR-T therapies require.
- The deal comes weeks after Novartis paused autoimmune trials of its CAR-T candidate rapcabtagene autoleucel following three patient deaths. ABO2203 gives the Swiss company another approach to pursuing an immune “reset,” although its early results cannot establish whether it will be safer.
How it works: ABO2203 packages mRNA inside lipid nanoparticles. Once delivered, the mRNA instructs the patient’s cells to produce a protein called a T-cell engager. That protein connects CD3 on T cells with CD19 on B cells, directing the T cells to kill the B cells. The underlying mechanism is already used in Amgen’s blood cancer drug Blincyto, but Abogen’s twist is to deliver the instructions for making the engager inside the body.
Zoom in: A study published in Cell reported results in three patients with treatment-resistant secondary immune thrombocytopenia, a condition involving immune-mediated destruction of platelets.
- ABO2203 rapidly depleted B cells in blood, with sustained depletion in bone marrow. Patients showed platelet recovery and reduced disease activity through six months of follow-up. Reported adverse events were Grade 1 or 2, with no cytokine release syndrome, a potentially serious inflammatory reaction commonly seen in CAR-T therapies.
Also notable: The researchers suggest ABO2203 could be administered by subcutaneous injection in an outpatient setting without premedication or lymphodepletion — the chemotherapy used to reduce immune cells before many CAR-T treatments. Those features could make treatment easier to deliver. However, the proposed advantages still need confirmation in larger studies.
The deal: Alongside ABO2203, Novartis gains exclusive options to license additional programs from Abogen’s RNA platform. Abogen could receive up to $7.2 billion (€6.4 billion) in development, regulatory and commercial milestones if all program options are exercised and the relevant milestones are met, plus potential royalties.
What to watch: ABO2203 is being studied in Phase 1 trials in autoimmune diseases and B-cell non-Hodgkin lymphoma. The next question is whether the B-cell depletion and platelet responses seen in three patients translate into durable benefits across larger groups while maintaining a manageable safety profile.
