After several clinical setbacks, Vertex Pharmaceuticals found promising results in its study of a drug to treat a genetic kidney disease called focal segmental glomerulosclerosis (FSGS).
As reported recently by BioPharmaDive, plans are underway to bring the drug to late-stage testing in early 2022 since it has out-performed expectations.
Although Vertex has been successful in developing four drugs to treat cystic fibrosis, in light of some recent setbacks, the company has been unable to convince its investors that it can be just as successful in developing drugs for other diseases.
About the Study
As far back as 2010, Belgium and U.S. researchers found that two variants in the APOL1 gene substantially increased not only the risk of FSGS but the risk of other kidney diseases.
By targeting these two variants, the study investigators discovered a new method of treating FSGS.
Sixteen patients each with two APOL1 variants and FSGS were enrolled in the study. After three months, the patients’ protein levels were reduced by 48%. This reduction surpasses the expectation of Wall Street analysts who had regarded a 30% to 40% lowering of protein as being successful.
Vertex released data showing that protein levels fell continually during the study from weeks one through thirteen. Vertex also confirmed that there were no serious reactions to the new drug and that all side effects were mild or moderate.
About FSGS
Focal segmental glomerulosclerosis is characterized by scar tissue that develops on sections of the kidneys responsible for filtering waste products from blood (glomeruli). The scarring prevents the kidney from filtering blood with the result that protein accumulates in the urine (proteinuria).
The new drug is being developed to target proteins that are encoded by the APOL1 gene.
FSGS can cause symptoms such as high creatinine levels or swelling. The disease may be caused by viral infections or toxicity from drugs. Currently, the only treatments available involve steroids or blood pressure medication, neither of which have proved beneficial.
African Americans are at Greater Risk
There is a greater risk for African-Americans to develop chronic kidney disease as opposed to Americans of European ancestry.
The increased risk can be associated with the two APOL1 genes found in people with African ancestry. The discussion centers around the inheritance factor showing a higher rate of kidney disease in African Americans living in the U.S.
Looking Forward
Although lowering protein levels in urine is associated with the efficacy of a drug, nothing has been established proving what effect lowering protein levels would have on kidney function or even how it would decrease the risk of kidney failure.
Vertex is working on several experimental treatments. The biotech company is confident that its years of experience and its drug-making expertise will result in the development of powerful new treatments.
