LJPC
La Jolla posts mixed bag of phase 3 data, raising hopes of FDA approval but calling commercial potential into question
“With positive primary outcome, we continue to view FDA approvability as high. However, given [the] lack of [statistically significant] mortality benefits and total organ function improvement—except for catecholamine use reduction—its commercial potential could be debatable
The questions about the commercial prospects of LJPC-501 stem from its performance against the secondary endpoints flagged up by Yang. By seven days into the study, 29% of people who received LJPC-501 had died. That compared to 35% in the placebo arm. After 28 days, the figures for the treatment and placebo arms were 46% and 54%, respectively. The difference fell short of statistical significance.
http://www.fiercebiotech.com/biotech/la-jolla-posts-mixed-bag-phase-3-data-raising-hopes-fda-approval-but-calling-commercial
Y luego el artículo del Bastardo:
LJPC-501 demonstrated a 22% reduction in the risk of death after seven days compared to placebo, but the difference was not statistically significant. After seven days, 29% of the LJPC-501 patients died compared to 35% of placebo patients.
"The mortality data suggest the need for larger trials to test whether angiotensin II [LJPC-501] therapy increases survival in this very sick group of patients," he added.
...it's a mistake to brush off the significance of having another class of drug to raise blood pressure in critically ill shock patients.
"What it means for the bedside clinician is we have now completed this jigsaw puzzle of three different pieces -- catecholamines, vasopressin and now angiotensin -- needed for a robust blood pressure response. With all three, we should be able to achieve a system where we can keep them balance so we can actually do good service to the human body. We supply patients with what is needed, keep their blood pressure normal, prevent organ injury and then fix the source of the problem," said Khanna.
If LJPC-501 is approved and widely adopted by hospital emergency rooms and intensive care units to treat the most at-risk distributive shock patients, peak sales could reach $500 million annually, according to Cowen analyst Phil Nadeau.
"Our consultants says there is a desperate need for new options in the CRH patient population, and that any agent that demonstrates an ability to increase blood pressure will be adopted," Nadeau wrote in a recent research note. CRH refers to distributive shock patients who don't respond to first-line treatment with catecholamines.
Last March, soon after La Jolla announced initial blood-pressure results from the LJPC-501 study, two experts weighed in more skeptically about the utility of the drug absent significant improvement in outcomes.
Dr. Jim Russell, chair of the ATHOS-3 data safety monitoring board, told BioCentury, "Clinicians already have a number of choices. I don't think uptake in the market will be very big without further studies. In particular, I think we probably need a larger study powered for a clinically meaningful endpoint such as organ dysfunction, or mortality, or both. We need to see at minimum a significant increase in vital organ function. Many clinicians would say you need to see a significant decrease in mortality."
Similarly, Dr. Scott Manaker of the University of Pennsylvania told BioCentury in March, "I'm not sure why one would use angiotensin II to simply increase blood pressure if it's not going to lead to improved long-term outcomes."
https://www.thestreet.com/story/14144059/1/mixed-outcomes-for-la-jolla-pharma-shock-drug-fuels-debate-over-sales-potential.html
PS: yo también contenta con las RDUS
«Después de nada, o después de todo/ supe que todo no era más que nada.»