Precision Immunology

We are a privately-owned biotech pioneering late-stage therapy an array of immunologic and inflammatory conditions which are driven by myeloid cells, including monocytes and macrophages. We began this journey many years ago and are initially demonstrating proof-of-concept in hematology/oncology in certain leukemias with our lead compound, lenzilumab (LENZ). LENZ is is completing a Phase 2/3 trial which is fully enrolled. LENZ is a potential therapy for patients who have treatment-naive proliferative Chronic MyeloMonocytic Leukemia (CMML). The current study focuses on patients that carry RAS mutations, such as KRAS, NRAS, CBL and PTPN-11. A significant proportion of patients are also carrying another high- risk mutation, ASXL-1.  

LENZ is demonstrating encouraging data in a range of myeloid disorders. Recent epidemiology and independent patient registries from leading centers and governments in 5 different countries lead to an estimated 60,000 patients in the US and European major 5 countries for CMML. The Australian Leukeamia Foundation cites the incidence (number of new patients with the diagnosis per year) of CMML as 2 per 100,000 people in Australia. 

The clinical response rate to LENZ in CMML, when added to current standard of care, so far in studies is more than 80% (using well-accepted clinical measures). This is 4-5 times the current standard of care response. LENZ is demonstrating excellent safety and tolerability in this condition, as it has in all the other conditions in which it has previously studies. Almost 1000 patients have received LENZ in multiple studies and conditions and in many different countries.

Additional encouraging data with LENZ has been presented in other serious, life-threatening myeloid conditions, such as secondary AML and in MDS, significantly expanding the patient population. So far, the rate of transformation to secondary AML from CMML in this high risk CMML population is much less than half of what would be expected from historical control data.

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This positions LENZ as a potential valuable medicine in the fight against a range of serious and life threatening leukemias and associated conditions. Further, research is pointing to myeloid conditions being associated with prior chemotherapy, opening up the possibility of yet further potential uses. While LENZ has not yet been approved for marketing, it is in the final stages of development and further studies in other conditions may follow. The company is preparing to approach various regulatory agencies for advice on the optimal clinical trial pathway to approval for marketing. 

In addition, LENZ has demonstrated promising data in clinical studies as a companion therapy to CAR-T to minimize or eliminate serious and potentially life-threatening side-effects associated with the CAR-T therapy.
A Phase 3 protocol for potential registration for LENZ in this use has been reviewed and signed off by FDA.