The BioPharm Brief: Cancer, Personalized

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry.

Merck and Moderna have reported a significant phase 3 milestone for their individualized cancer vaccine approach. In the INTerpath-001 trial, intismeran autogene, also known as V940 or mRNA-4157, combined with pembrolizumab met its primary endpoint of recurrence-free survival and a key secondary endpoint of distant metastasis-free survival in patients with completely resected stage IIB to IV melanoma. The trial randomized 1,137 patients to receive either the combination or pembrolizumab alone. The companies say the result represents the first positive phase 3 readout for an individualized neoantigen therapy and an mRNA-based cancer therapy. The personalized treatment uses sequencing of a patient's tumor to identify mutations and encodes up to 34 tumor-specific neoantigens into synthetic mRNA, with the goal of generating a targeted T-cell response.

The manufacturing challenge behind personalized cancer vaccines is also moving into the clinic. NTx Bio's NTxscribe CORE platform is being used by Baylor College of Medicine to manufacture mRNA for DOC1021, a personalized double-loaded dendritic cell vaccine from Diakonos Oncology that is now being evaluated in a Phase 1 trial for refractory melanoma. The platform uses continuous-flow technology to amplify tumor-derived mRNA from small surgical biopsies while preserving the full-length range of the patient's tumor transcriptome. The approach is designed to address a major challenge in personalized cell and gene therapies, where manufacturing can be highly manual and variable. DOC1021 is also being developed for glioblastoma and pancreatic ductal adenocarcinoma.

And in glioblastoma, BioArctic and Mesenkia Therapeutics are combining two platforms to explore a new way of getting antibodies into the brain. Their research collaboration pairs BioArctic's BrainTransporter technology with a Mesenkia antibody targeting herpesvirus entry mediator, or HVEM, a protein found on certain glioblastoma tumor cells, including tumor stem cells. BrainTransporter uses transferrin receptor-mediated transport to increase antibody exposure in the brain. In preclinical work, the technology produced up to a 70-fold increase in antibody brain exposure. The companies will now generate and evaluate a candidate in preclinical studies, with further development decisions dependent on those results.

That’s The BioPharm Brief for today.

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Key takeaways:

• Merck and Moderna reported a positive phase 3 readout for an individualized mRNA neoantigen therapy in melanoma.
• NTx Bio's manufacturing platform is now supporting clinical production of a personalized cancer vaccine.
• BioArctic and Mesenkia are taking a brain-delivery approach into glioblastoma research.