DANA-FARBER CANCER INSTITUTE

X4 Pharmaceuticals reported $0.6M net product revenue for Q3 2024, with a net loss of $(36.7)M. The company launched XOLREMDI for WHIM syndrome in the U.S. and plans to submit an MAA to the EMA by early 2025. X4 anticipates continued operating losses and negative cash flows, requiring additional capital. Challenges include reliance on XOLREMDI, potential market size uncertainty, and regulatory compliance risks.

Valo Health appoints Brian M. Alexander as CEO and CEO-Partner of Flagship Pioneering, elevates Brett Blackman to COO, and adds Travis Wilson to its Board of Directors.

Chimerix to present updated RANO 2.0 response assessment of dordaviprone in recurrent H3 K27M-mutant diffuse midline glioma at the 2024 SNO Annual Meeting, showing an overall response rate of 28.0%.

Cancer care institutions develop patient-partnered research initiatives, facing challenges like complex consent processes, data sharing, and EHR interoperability. The PE-CGS Network aims to engage patients directly in research, focusing on rare cancers and underrepresented populations, using culturally sensitive communication and digital platforms. Despite efforts, barriers persist, including lengthy consent forms and data transfer difficulties between institutions, prompting ongoing initiatives to improve patient engagement and data accessibility.

Chimerix announced updated Phase 2 results for dordaviprone in recurrent H3 K27M-mutant diffuse midline glioma, showing a 28.0% overall response rate, median time to response of 4.6 months, and median duration of response of 10.4 months using RANO 2.0 criteria. The company plans to include these results in their upcoming New Drug Application to Australian regulators and will present the findings at the 2024 SNO Annual Meeting.

Recent studies in The ASCO Post include: KEYNOTE-522 showing pembrolizumab plus chemotherapy benefits in early-stage triple-negative breast cancer; WHO review finding no link between mobile phone use and brain cancer; TiNivo-2 trial reporting no progression-free survival benefit from nivolumab plus tivozanib in metastatic renal cell carcinoma; SOLARIS trial finding no progression-free survival benefit from high-dose vitamin D3 in metastatic colorectal cancer; American Cancer Society report highlighting rising breast cancer incidence in key subgroups; retrospective study on adjuvant chemotherapy in pancreatic ductal adenocarcinoma; genetic study identifying unique prostate cancer risk factors in African men; and first-in-human trial of puxitatug samrotecan showing manageable safety profile and preliminary efficacy in advanced solid tumors.

The phase 3 KEYVIBE-008 trial found that co-formulated vibostolimab and pembrolizumab plus etoposide and platinum did not meet the primary end point of overall survival (OS) in extensive-stage small cell lung cancer (ES-SCLC) compared to atezolizumab plus EP. Median OS was 11.5 months vs 12.9 months, and median progression-free survival (PFS) was 5.3 months vs 4.5 months. The trial was discontinued due to the benefit/risk profile, though investigation continues in non–small cell lung cancer (NSCLC).

PD-L1 expression predicts better outcomes with nivolumab and ipilimumab in ccRCC, while high KIM-1 expression worsens outcomes, especially with nivolumab alone or with ipilimumab. High fibroblast levels in the tumor microenvironment shorten DFS with nivolumab plus ipilimumab compared to nivolumab alone. KIM-1 may serve as a minimally invasive biomarker for RCC recurrence.