Novartis has announced it has signed a licensing and option agreement with Chinese biotech Abogen Biosciences worth up to $7.8bn, gaining worldwide rights to an experimental mRNA-based therapy designed to deplete disease-causing B cells.
Under the agreement, Novartis will pay Abogen $575m upfront for ABO2203, an mRNA-encoded CD19xCD3 T-cell engager (TCE).
The Chinese biotech is eligible for a further $7.2bn in development, regulatory and commercial milestones if Novartis exercises options covering additional programmes and agreed targets are achieved. Abogen may also receive royalties on future product sales.
The transaction remains subject to customary closing conditions, including regulatory clearances.
ABO2203 uses a lipid nanoparticle (LNP) formulation to deliver mRNA encoding a TCE that binds to CD19 on B cells and CD3 on T cells.
Rather than administering a manufactured TCE protein directly, the approach uses the patient's own cells to produce the therapeutic molecule in vivo.
The strategy is being investigated as a potential way of selectively depleting B cells implicated in autoimmune diseases, including immune thrombocytopenia (ITP), lupus and other conditions.
mRNA approach targets B-cell depletion
Early clinical evidence has already supported the approach — research published in Cell last month reported results from three patients with refractory secondary ITP treated with ABO2203.
The study found rapid and complete depletion of peripheral B cells, with sustained depletion in bone marrow and durable platelet recovery during six months of follow-up.
The three patients experienced only grade 1 or 2 adverse events and no cytokine release syndrome — although, it is worth noting the very small patient population, which means substantially more clinical research will be required to establish the therapy's safety and efficacy.
ABO2203 is also being investigated in an early Phase I study in patients with refractory autoimmune diseases.
The trial is designed to assess safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy, with an estimated enrolment of 66 patients.
The programme builds on growing interest in B-cell depletion as a way to reset the immune system.
Conventional CAR-T therapies have generated promising results in severe autoimmune disease, while TCEs offer an alternative approach that could potentially avoid some of the manufacturing complexity associated with cell therapies.
For drug developers and manufacturers, ABO2203 also highlights the expanding therapeutic applications of mRNA and LNP technologies beyond vaccines, with manufacturing shifting towards the production of nucleic-acid drug substances and sophisticated delivery systems.
Novartis looks to Chinese biotech innovation
The agreement is the latest in a series of licensing deals highlighting Novartis' focus on external innovation and China's increasingly important role in global drug development.
In September, Novartis agreed to license a preclinical radioligand therapy from China's BoomRay Pharmaceuticals in a deal potentially worth $900m.
The company has also established collaborations with Chinese biotech Argo Biopharma covering siRNA-based cardiovascular medicines.
The broader trend is being seen across the pharma sector. Global drugmakers are increasingly licensing innovative assets from Chinese developers, with Chinese companies retaining domestic capabilities while securing international partners to fund development and commercialisation.
For Novartis, the Abogen agreement adds an emerging mRNA therapeutic platform and an experimental autoimmune medicine to its pipeline, while giving the Swiss pharma options around further RNA-based programmes.
