University Health Network

Pozelimab combined with cemdisiran shows superior control of intravascular hemolysis in PNH patients compared to Ultomiris, with 96% achieving target LDH levels versus 80%. The combination offers potential for self-administration with infrequent dosing, indicating a promising treatment for PNH and other complement-mediated diseases.

Pozelimab combined with cemdisiran shows superior control of intravascular hemolysis in PNH patients compared to Ultomiris, with lower LDH levels and potential for self-administration. Ongoing Phase 3 trials and extensions aim to confirm its efficacy and safety, offering hope for better PNH treatment.

Top hospitals in North America, including University of Minnesota, Toronto Western Hospital, UCSF Medical Center, Kaizen Brain Center, and University of South Florida, are conducting clinical trials on trigeminal neuralgia, aiming to improve treatment and quality of life for patients suffering from this severe facial pain condition.

Rebecca Roland's family struggles to find Teva-Clobazam, her father's epilepsy medication, due to a shortage in Canada extending to April 2025. The controlled substance's scarcity forces constant pharmacy searches and concerns over potential breakthrough seizures. Health Canada is working with manufacturers to mitigate the shortage, but alternatives pose risks.

Despite headwinds, the techbio sector continues to attract significant investment and drive innovation, exemplified by the 2024 launch of Xaira, backed by over $1 billion, aiming to design novel therapeutic proteins using advanced AI. The sector is maturing through strategic consolidation, significant capital deployment, technical diversification, and steady clinical validation, as illustrated by 25 techbio organizations, including notable players like Isomorphic Labs, Generate:Biomedicines, Recursion Pharmaceuticals, and Schrödinger Inc., all leveraging AI for drug discovery. Emerging players such as Archon Biosciences, AION Labs, and Antiverse are also making strides, while established companies like Insilico Medicine and Ginkgo Bioworks continue to expand their platforms and partnerships.

Regeneron Pharmaceuticals reported positive phase 3 trial results for pozelimab plus cemdisiran in treating paroxysmal nocturnal haemoglobinuria (PNH), showing superior efficacy over standard treatments. The combination achieved higher rates of LDH control and normalization, indicating significant progress in PNH treatment and potential for broader application in complement-mediated diseases.

Phase 3 trial data showed the poze-cemdi combination treatment achieved better disease control in PNH patients, measured by LDH levels, compared to ravulizumab. Poze-cemdi, combining an antibody and siRNA targeting C5, demonstrated superior LDH control and normalization, with potential for self-administration, indicating a promising treatment approach for PNH and other complement-mediated diseases.

Regeneron's pozelimab and cemdisiran (poze-cemdi) in PNH showed 96% LDH control vs 80% with ravulizumab, 93% LDH normalization vs 65% with ravulizumab, and 84% LDH decrease at week 26. Safety profile similar to ravulizumab, with 84% TEAEs vs 87% for ravulizumab. Subcutaneous delivery every 4 weeks with potential for self-administration.

Phase 3 trial data shows poze-cemdi combination treatment outperforms ravulizumab in controlling PNH, with better LDH level management. Poze-cemdi, a first-in-class treatment combining an antibody and siRNA targeting C5, offers potential for self-administration and improved patient outcomes.

Phase 3 trial data showed the poze-cemdi combination treatment outperformed standard ravulizumab in controlling PNH, with 96% achieving LDH control vs. 80% on ravulizumab. Poze-cemdi offers potential for self-administration, marking a significant advancement in PNH treatment.