Latest

As part of BioPharm International's continuing coverage from the BIO International Convention 2026, Sara Jane Demy discusses signs of renewed investment activity and why inclusive scientific leadership remains essential to advancing drug development.

Today's BioPharm Brief highlights advances in neurology, hematology, and nephrology, featuring new Alzheimer's disease biomarker data, an expanded pediatric approval for von Willebrand disease prophylaxis, and the first dual BAFF and APRIL inhibitor approved for IgA nephropathy.

Vaccinex will present new biomarker data from the Phase 1b/2 SIGNAL-AD trial of pepinemab — a humanized IgG4 monoclonal antibody targeting Semaphorin 4D — at AAIC 2026 in London on July 13, showing that SEMA4D blockade regulates glial biomarkers associated with disease progression in early Alzheimer's disease, while outlining plans for an enlarged Phase 2b SIGNAL-AD2 study.

The FDA has approved an expanded indication for Wilate (von Willebrand Factor/Coagulation Factor VIII Complex, Human) to include routine prophylaxis in children younger than 6 years with von Willebrand disease — the first FDA-approved option for this age group — based on phase 3 WIL-33 trial data showing significantly reduced bleeding episodes in young pediatric patients.

The FDA has granted accelerated approval to Trutakna, a recombinant fusion protein that simultaneously inhibits BAFF and APRIL, for adults with primary IgA nephropathy at risk for disease progression — marking the first approval of a dual BAFF/APRIL inhibitor in the US and a significant advance in the growing wave of targeted biologics for this immune-mediated kidney disease.

The annual gathering in San Diego showcased the industry's pivot toward complex molecules, flexible manufacturing, and human-based drug discovery — while candid conversations about capital access, deal strategy, and preclinical predictability reflected a sector navigating pressure with optimism.

Today's BioPharm Brief explores three different ways biopharma companies are advancing precision medicine, from next generation bispecific antibody drug conjugates and myeloid engager immunotherapy to stem cell derived neuron replacement for Parkinson's disease.

Because antibody-drug conjugates sit at the interface of antibody biology and small-molecule pharmacology, payload-led design brings those disciplines together, earlier.

Kenai Therapeutics has completed enrollment in the Phase 1b/2a REPLACE trial of RNDP-001, an allogeneic iPSC-derived dopaminergic neuron replacement therapy for idiopathic Parkinson's disease, with safety and preliminary efficacy data expected in 2027 — representing a significant step toward a potential disease-modifying cell therapy for a condition affecting more than 10 million people worldwide.

Biocytogen and Whitehawk Therapeutics have announced a global collaboration combining Biocytogen's RenLite bispecific antibody discovery platform with Whitehawk's CPT113-based ADC linker-payload technology to generate bispecific ADC candidates with differentiated tumor-targeting profiles — with Whitehawk targeting new ADC INDs within 12 to 24 months.

The European Commission has approved Tepkinly in combination with lenalidomide and rituximab for adults with relapsed or refractory follicular lymphoma after at least two prior systemic therapies, based on Phase 3 EPCORE FL-1 data showing a 79% reduction in risk of disease progression or death — marking the first bispecific antibody-based combination regimen approved in Europe for this indication.

The FDA has approved a label expansion for Casgevy, the first CRISPR-based gene therapy, lowering the eligible age from 12 to 2 years in sickle cell disease — a move that significantly broadens the potential patient population and marks another milestone in the clinical maturation of CRISPR-based genomic medicine.

Three-year follow-up data from Agenus' Phase 2 trial of botensilimab plus balstilimab in refractory microsatellite-stable metastatic colorectal cancer show a 33% overall survival rate at three years — with evidence of a plateau on the Kaplan-Meier curve beyond two years — representing an unprecedented durability signal in a tumor type historically resistant to immune checkpoint therapy.