AACR 2025: Promising Results in Cancer Therapies and Targeted Treatments

The American Association for Cancer Research (AACR) annual conference has showcased significant advancements in oncology, with several pharmaceutical companies presenting encouraging data for both established and investigational cancer treatments.

Artios' ATR Inhibitor Shows Promise in Solid Tumors

Artios Pharma's oral ATR inhibitor, ART0380, demonstrated positive results in the Phase I/IIa STELLA study. When combined with low-dose irinotecan, ART0380 achieved a 37% confirmed overall response rate (cORR) in patients with advanced or metastatic solid tumors. Notably, in patients negative for the ataxia-telangiectasia mutated (ATM) protein marker, the cORR rose to 50%, with a median response duration of 5.7 months.

The study focused on patients with ATM deficiency, a key marker for replication stress present in up to 25% of high-unmet need solid tumors. ART0380 showed efficacy against eight tumor types, including pancreatic and colorectal cancer, highlighting its potential as a targeted therapy for specific patient populations.

Agenus and Roche Present Contrasting Results in Immunotherapy

Agenus reported promising data from its Phase II NEOASIS trial, evaluating the combination therapy BOT/BAL (botensilimab and balstilimab) in early-stage solid tumors. In patients with mismatch repair-deficient/microsatellite instability-high tumors, the therapy achieved a 90% pathological response rate and a 70% pathological complete response rate. The treatment also showed activity in mismatch repair-proficient/microsatellite stable tumors, with an 80% pathological response rate.

In contrast, Roche's anti-TIGIT therapy tiragolumab failed to meet its primary endpoint in the Phase III SKYSCRAPER-01 trial for non-small cell lung cancer (NSCLC). When combined with the PD-1 blocker Tecentriq, tiragolumab reduced the risk of disease progression or death by 28% compared to Tecentriq alone, but this effect did not reach statistical significance. Despite the setback, Roche emphasized the treatment's "numerical improvements" in progression-free and overall survival.

Advances in Targeted Therapies for NSCLC and Head and Neck Cancer

Boehringer Ingelheim's HER2 tyrosine kinase inhibitor zongertinib showed promising results in the Phase III Beamion LUNG-1 trial for NSCLC patients with HER2 mutations. The drug achieved a 71% objective response rate, with 7% of patients experiencing complete response and 64% partial response. Notably, zongertinib demonstrated intracranial activity in patients with brain metastases, achieving a 41% response rate in this subgroup.

Merck's Keytruda (pembrolizumab) bolstered its case in head and neck cancer with data from the Phase III KEYNOTE-689 study. As part of a perioperative treatment regimen, Keytruda improved event-free survival by 27% compared to standard of care alone in patients with resected locally advanced head and neck squamous cell carcinoma.

These developments underscore the ongoing progress in targeted therapies and immunotherapies across various cancer types, offering new hope for patients with limited treatment options.