LMP01
Combination therapy with intranasal delivery for glioblastoma
FDA Orphan Drug DesignationThe disease
Glioblastoma is the most common and most aggressive primary brain cancer in adults. Standard treatment has changed little in two decades. Median survival is about 15 months, and nearly all tumors recur. The tumor grows quickly, infiltrates the surrounding brain and adapts to available treatments, while the blood-brain barrier keeps most drugs from reaching it at effective levels.
LMP01 is a combination of two small molecules designed for IDH wild-type glioblastoma, the most common and most lethal form of the disease. Both components are delivered intranasally, an approach designed to reach the brain directly while limiting exposure to the rest of the body.
A dual mechanism
Glioblastoma cells depend on two connected systems to survive and grow. The first is aerobic glycolysis, the altered energy metabolism that fuels rapid tumor growth. The second is RAS and cell-cycle signaling, which drives proliferation. LMP01 acts on both at once, so tumors that adapt to pressure on one pathway may remain vulnerable through the other.
Our preclinical work supports this rationale. Cell-based studies show synergy between the two components, and in vivo and proteomic studies are giving us insight into how the combination acts.
Early human experience
The scientific basis for LMP01 comes from compassionate use. A patient with IDH wild-type glioblastoma, given a few months to live, received both compounds that make up LMP01 over the course of her compassionate use treatment and survived more than ten years with preserved quality of life. Her regimen differed from LMP01, which delivers both components intranasally.
This is a single case and not a clinical trial result. Her outcome, together with what is known about how the two compounds act, led us to build this program, and we are now testing it in controlled preclinical studies.
Development status
- Orphan Drug DesignationGranted by the FDA for LMP01 in malignant glioma.
- PatentOur international (PCT) application has been published, and we are pursuing patent protection in the United States and other key markets.
- PreclinicalFormulation development has narrowed to final candidates. In vivo studies of tolerability, brain exposure and efficacy in glioblastoma models come next.
- Next stepsWe are preparing for early engagement with the FDA ahead of a first-in-human Phase 1 study. We are also in discussions with leading US academic neuro-oncology centers about preclinical and clinical collaboration.
LMP01 facts
Two molecules, two targets
A new combination aimed at two pathways glioblastoma relies on.
Known components
Both components are existing compounds with published human clinical data.
Direct route to the brain
Intranasal delivery is designed to bypass the blood-brain barrier and limit systemic exposure.
Human signal
A compassionate use patient whose treatment included LMP01’s two compounds survived more than ten years with good quality of life.
Orphan Drug Designation
Granted by the FDA for malignant glioma, with development incentives and market exclusivity on approval.
Beyond glioblastoma
The same approach may apply to other brain cancers. See our pipeline.
LMP01 is an investigational therapy and has not been approved by the FDA or any other regulatory authority.