Some low doses of mepolizumab have shown some benefits, but there was no clear benefit for infants with phenylketonuria. But Pfizer is optimistic about these promising results
In February 2018 Pfizer announced data from two phase 3 clinical trials in children with phenylketonuria, a rare metabolic disorder. Here is a summary of what is known: The data were inconclusive, however, and there was no clear benefit in infants with phenylketonuria. However, some low doses of mepolizumab showed some benefit, although there was no clear benefit for the larger group of infants with phenylketonuria who were treated with metformin, on metformin alone.
Analyzing the data, Pfizer determined that androgens (such as testosterone and oestrogen) had shown to increase phenylketonuria in some infants. In the second trial, a team of 18 Pfizer scientists studied the effects of mepolizumab on phenylketonuria in those children already diagnosed with the disorder. They saw that mepolizumab reduced either its harmful or harmful effects. Although the findings are encouraging, the results did not meet the study’s primary endpoints – an improvement in a biochemical measure of phenylketonuria.
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On the question of infant treatment, Pfizer was able to achieve a mixed result. It showed that mepolizumab reduced the amount of harmful proteins that make infants with phenylketonuria more susceptible to infection. It also decreased the amount of harmful proteins that make infants more susceptible to short-term toxin exposure. The study was not done in adults. And there was no long-term benefit for adults or children with phenylketonuria.
Pfizer has partnered with Biogen to develop Pfizer HSP20, a short-acting inhibitor of the enzyme to target this enzyme activity. The goal is to stop inflammation in tumors. Since the prostate is a notoriously aggressive cancer for men with PNH, the initial finding is promising and in a high-risk population that could delay the eventual onset of the disease.
The message from the new study is that we need to be careful when interpreting the data. We must not ignore details that warrant additional data analysis.