Biohaven Ltd. has reported encouraging preliminary clinical data from its oncology development programs, showcasing the potential of its next-generation antibody drug conjugate (ADC) platform incorporating the proprietary TopoIx payload. The company presented updates at its 2025 R&D Day, demonstrating clinical progress with two innovative ADCs targeting different cancer pathways.
BHV-1510 Shows Promise in Combination Therapy
The company's novel Trop2-directed ADC, BHV-1510, demonstrated encouraging preliminary clinical activity both as monotherapy and in combination with Regeneron's anti-PD-1 antibody cemiplimab. In the ongoing Phase 1 study, the combination showed remarkable anti-tumor activity, with tumor shrinkage observed in the first 6 out of 6 patients treated, including confirmed partial responses and activity in patients with brain metastases.
Notably, the majority of patients treated with the combination had previously failed anti-PD-1/PD-L1 therapies, suggesting potential for BHV-1510 to overcome resistance mechanisms. As monotherapy, tumor reductions including partial responses were observed in patients who had failed standard of care therapies.
"The early clinical data with BHV-1510 dosed in patients who failed standard of care treatment are highly encouraging, particularly the observed potential synergy with anti-PD-1 therapy," commented Nushmia Khokhar, M.D., Chief Medical Officer of Oncology at Biohaven. "These findings, combined with the promising efficacy and tolerability profile of our novel TopoIx payload and stable linker technology, support the potential of BHV-1510 to advance into earlier lines of therapy for challenging tumor types."
Favorable Safety Profile Observed
BHV-1510 demonstrated a favorable pharmacokinetic profile with very low levels of free payload. The safety profile was encouraging, with stomatitis as the main toxicity observed in the Phase 1 study—an expected on-target Trop2 class toxicity that proved manageable. Importantly, there were no cases of payload-associated interstitial lung disease (ILD), and low rates of gastrointestinal and hematologic toxicities were observed. The combination with cemiplimab was well tolerated with no dose limiting toxicity reported in initial cohorts.
First-in-Class FGFR3 ADC Enters Clinical Testing
Biohaven announced the first patient dosing in the Phase 1 study of BHV-1530, a potential first-in-class fibroblast growth factor receptor 3 (FGFR3)-directed ADC utilizing the proprietary TopoIx payload. BHV-1530 targets cancers driven by FGFR3 alterations and/or upregulated FGFR3 protein expression, including urothelial cancers and other solid tumors.
FGFR3 represents a clinically validated target in oncology, with one small molecule inhibitor (erdafitinib) currently approved. However, there are no FGFR3 ADCs beyond BHV-1530 in advanced clinical testing, positioning this as a potentially first-in-class therapeutic approach.
"We are excited to partner with Biohaven and dose the first patient on this important study," stated Michael Song, M.D., Ph.D., Principal Investigator and leading medical oncologist and hematologist at NEXT Oncology. "This is an exciting, validated target with potential to extend therapeutic benefit to several FGFR3 driven tumors."
Platform Technology and Strategic Collaborations
The early clinical data aligns with BHV-1510's preclinical profile, which demonstrated high ADC stability, differentiated safety and efficacy, immunogenic cell death, and anti-PD-1 synergism. Biohaven is advancing additional innovative technologies through strategic collaborations with Merus and GeneQuantum, leveraging the company's differentiated ADC platform against undisclosed novel validated and emerging high-value targets.
The preclinical programs incorporate the TopoIx payload, which demonstrated immunogenic cell death and synergistic efficacy with PD-1/PD-L1 checkpoint inhibitors in laboratory studies.
"We are excited to be in the clinic with two innovative ADCs and to share the early clinical experience with the first of our two programs, demonstrating the potential of our oncology portfolio to deliver a wide range of optimized, next-generation ADCs," commented Brian Lestini, M.D., Ph.D., President of Oncology at Biohaven. "Together with our collaborations with Merus and GeneQuantum as well as licensed conjugation technology from Yale University, Biohaven's platform has the potential to generate multiple differentiated mono- and bispecific ADC therapies with greater potency and selectivity over currently available ADC approaches."
