Laminar Pharma has reported encouraging interim results from its ongoing clinical trial of LAM561 in newly diagnosed glioblastoma patients, showing marked improvement in progression-free survival (PFS) among MGMT-methylated patients. The membrane lipid therapy-based treatment, when combined with standard chemoradiotherapy, demonstrated particularly promising results in specific patient subgroups.
Significant PFS Improvement in Methylated Patients
In MGMT-methylated patients with RTOG4 scores, LAM561 achieved a median PFS of 86.4 weeks compared to 54.7 weeks in the placebo group (n=39). Even more striking results were observed in RTOG3 methylated patients, where PFS reached 56.7 weeks versus 19 weeks in the placebo arm (n=9). The preliminary hazard ratio of 0.53 suggests a potentially clinically relevant improvement for methylated patients receiving LAM561 with standard of care.
"We are encouraged by the progression-free survival results from this trial in MGMT-methylated patients, which, if reflected by positive overall survival results, may represent a significant advance in the treatment of glioblastoma," stated Dr. Pablo Escribà, CEO of Laminar Pharma.
Trial Design and Current Status
The pivotal Phase 2b/3 double-blind, placebo-controlled trial enrolled 144 patients with newly diagnosed, IDH-wildtype glioblastoma. Following 66 PFS events in November 2024, the independent data monitoring committee recommended:
- Continuation of the trial until 90 overall survival events
- No stoppage for safety or futility concerns
- Unblinding of the study
The trial will continue until late 2026, when final overall survival analysis is expected to be completed.
Safety Profile and Treatment Tolerance
The combination of LAM561 with standard of care demonstrated a favorable safety profile, consistent with previous clinical trials. No new safety signals were observed in the combination arm, suggesting good tolerability of the treatment regimen.
Clinical Context and Market Impact
Glioblastoma remains one of the most challenging forms of brain cancer, affecting over 100,000 people globally each year. Current treatment options offer limited efficacy, with median survival around 14.5 months despite optimal chemoradiation treatment. Only 15% of patients survive two years post-diagnosis, highlighting the urgent need for more effective therapies.
LAM561's mechanism of action involves altering plasma membrane composition in cancer cells, thereby reducing the activity of membrane-associated signaling proteins that promote tumor growth. This novel approach, particularly effective in brain tumors, represents a potential breakthrough in glioblastoma treatment.
Future Implications
While these interim results are promising, particularly for MGMT-methylated patients who represent 35-50% of glioblastoma cases, the final assessment of LAM561's efficacy awaits overall survival data. The drug's potential impact on the glioblastoma treatment landscape could be substantial, especially considering the current limited therapeutic options for this aggressive form of brain cancer.
