Acute kidney injury (AKI) kills 90,000 people/yr in the US alone. There is no specific treatment, only supportive care. Considering its importance, there is relatively little research or progress; it is a neglected disease. AKI has many causes: sepsis, ischemia, major surgery, toxins, etc. AKI usually occurs without warning, so we need a treatment that works after the injury. AKI also causes or exacerbates chronic kidney disease.
Other groups have used various high throughput genomic and proteomic approaches to catalog the differences between normal and injured kidneys, including at the single-cell level. As far as we are aware, none of these approaches have led to any effective therapies for AKI.
Our solution is unique, first in class pharmaceutical, which works after the insult to the kidney. AKI has high mortality and high incidence of long-term effects on survivors, which will justify a high cost of treatment. We expect that our treatment will be short, about two weeks. The comparable price for similar biologics averages about $20,000 in the U.S., though less in other markets. There are about 800,000 patients in the U.S. who are diagnosed with AKI annually. Assuming $20,000 per patient and 25% market penetration = $4B U.S. sales. In the developed parts of Europe and Asia, there are about 2 million people diagnosed per year. Assuming $5000 per patient and 25% penetration = $2.5B Europe and Asia sales. Total world sales = $6.5B annually (projected).
No therapies specific for AKI have been approved by the FDA or European Medicines Agency. Only a few possible competitors are known to be under development.
Quark Pharmaceuticals QPI-1002 (I5NP) is a siRNA targeting p53 to prevent apoptosis. This drug is in a Phase 3 trial for AKI following cardiac surgery. The drug must be given very close to the time of injury, such as occurs in AKI due to cardiac injury; this indication is a small fraction of the total AKI population.
AM Pharma Recombinant Alkaline Phosphatase for AKI due to sepsis, which is a much more common cause of AKI, failed a Phase 2 trial, as reported in November 2018 in JAMA.
We have been studying the cell biology of kidney cells for decades and have developed special expertise. In particular, we have grown kidney tubule cells as 3D organoids that closely resemble the in vivo situation.
We look for proteins that are secreted into the central lumen of injured organoids, as such proteins are likely to be essential for repair.
We value data from the experimental group and control group examined under the same situation. We study factors that have mosaic patterns comparing to controls for their biological significance.
We appreciate the complexity of biology. We develop treatments following the inherent complexity.
We have assembled a team of scientists and innovators recognized for their pioneering work in developing and delivering new treatments to patients. We aim to create precision therapies for the most challenging diseases.
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