PIK3CA- and MAP2K-activated squamous cell carcinoma
A novel combination of small-molecule therapeutics for the treatment of advanced squamous cell carcinoma (SCC) tumors that simultaneously harbor genetic alterations activating the proto-oncogenes, PIK3CA and MAPK2K1/2. Genomate population-level clinicogenomic analysis revealed that 0,64% of SCC cases fall into this category. There are approximately 1.5-2 million newly diagnosed SCCs per year in the US and approximately 4–5 million worldwide. Currently there is no FDA-approved targeted therapy for this molecular subtype. Standard chemotherapies have limited efficacies for advanced cases.
This asset is the combination of two repurposed small-molecule therapeutics. One of the molecules has no remaining basic molecule exclusivity, the still existing patents are for formulation and treatment-use. Core-protection milestones for the other molecule are 2027 in the U.S. and 2029 in Europe, with additional formulation and treatment-use patents extend beyond those dates.
An off-label, in-patient application of the asset demonstrated safety and efficacy for the indication. In one case study, the combination was used in another indication and no clinical toxicities or hypoglycemia were reported. Various lines of preclinical evidence also support antitumor activity. The asset is supported by a compelling data package demonstrating a high probability of regulatory success.
Non-small cell lung cancer with rare EGFR mutations
This asset is the repurposing of a small-molecule receptor tyrosine kinase inhibitor therapeutic for non-small cell lung cancer (NSCLC) with rare EGFR mutations, such as exon 20 insertions. Genomate population-level clinicogenomic analysis revealed that 6.07% of NSCLC cases harbor rare mutations. There are approximately 200,000 newly diagnosed NSCLCs per year in the US and approximately 2.2 million worldwide. Currently, aminvantanab, a monoclonal antibody is an FDA-approved targeted treatment for EGFR exon 20 mutant NSCLC. The asset provides an orally administered alternative option.
The principal compound patent and the associated six-month pediatric exclusivity expired in 2026. Additional patents covering particular forms, formulations, and uses remain listed. As the asset has been extensively used in solid tumor indications, its toxicity profile is well documented and manageable, significantly derisking repurposing. There are several published case reports demonstrating the efficacy of the asset in NSCLC with EGFR exon 20 insertions, as well as reports involving other rare EGFR variants. The asset is supported by a compelling data package demonstrating a high probability of regulatory success.
Lung cancer with NRG1 translocations
This asset is the repurposing of a small-molecule receptor tyrosine kinase inhibitor therapeutic for lung cancer with NRG1 translocations. Genomate population-level clinicogenomic analysis revealed that NRG1 translocations are significantly overrepresented in NSCLC cases without effective current FDA-approved molecularly targeted treatment options. 0.8 % of NSCLC cases harbor NRG1 translocations, and this molecular alteration is substantially enriched in certain rare histologies, e.g., invasive mucinous adenocarcinoma (IMA) (28%). There are approximately 200,000 newly diagnosed NSCLCs per year in the US and approximately 2.2 million worldwide. Currently, zenocutuzumab-zbco (Bizengri) is the only FDA-approved treatment for NRG1 fusion-positive NSCLC. Bizengri is a bispecific antibody, administered as an intravenous infusion. The asset provides an orally administered alternative option.
The principal compound patent and the associated six-month pediatric exclusivity expired in 2026. Additional patents covering particular forms, formulations, and uses remain listed. As the asset has been extensively used in solid tumor indications, its toxicity profile is well documented and manageable, significantly derisking repurposing.
Multiple case reports and small series document afatinib activity in NRG1 translocation-positive lung adenocarcinoma, including prolonged responses. The asset is supported by a compelling data package demonstrating a high probability of regulatory success.
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