Barbet01 schreef op 15 september 2020 15:13:
[...]
Bedankt en eens, WIC.
Als je de conference call uit Q2 er dan op na leest...
Phil Nadeau
Good morning. Thanks for taking my questions. A question on 1972 clinical trials.gov lists the study has completed as of mid July. So should that imply to us that we're going to see data over the next several weeks to months? And secondly, can you talk a little bit about the primary endpoint in the study, how is the study powered on its endpoint of cartilage thickness at this cMTFC? And what's the clinically meaningful difference on that endpoint? Thank you.
Walid Abi-Saab
...
In terms of the, how the study was powered and the primary endpoint. So this is - the study is powered using MRI. As a primary endpoint, the MRI would measure cartilage thickness and the medial portion of the knee. We follow a very rigorous algorithm that is automated. We work with the sort of the best imaging groups out there. And we've learned a lot also from a lot of studies that were conducted in the field, particularly with Furman [ph] where they had a very large database.
The way we powered our study is to be able to detect a reduction in cartilage loss by about 75% over a year period, so essentially, people who have a certain degree of knee osteoarthritis use, on average, about 100 micron over a year and you can measure that very accurately, actually with MRI and with the variability that we expect, we powered the studies to be able to detect a difference between drug and placebo when we reduce this by about 75%.
You asked a very important question, what is the clinically meaningful effect?
Actually the simple answer is, and honest answer is, we don't know. Hope, because simply nobody has demonstrated these kind of changes and linked them to clinical meaningfulness. This is one of the opportunities and also challenges of being at the forefront and treading into uncharted territory.
So we look forward to get the data and see how these will match or aligned with some of the very important endpoints that we measure, including pain, including function, and we will work with our experts to be able to interpret this and also we will work with health authorities to figure out how we can design the subsequent trials to demonstrate indeed that the changes that we see are clinically meaningful to these patients.
...
Barbet01: ... als je deze reactie naast die van de CEO legt van tijdens de catch up waarbij hij naar mijn aanvoelen toch vrij zeker was van primary outcome... dan weet dat me toch ook te verheugen.
Dat ze hierboven al verwijzen naar het hopelijk behalen diverse secondary endpoints, (10/10 is een utopie natuurlijk), en het feit dat dit een first timer is (niemand zou het hen hebben voorgedaan bij succes) dan denk ik dat bij behalen van primary outcome nagenoeg al de rest meegenomen is. Meer zelfs: behalen ze de primary en enkele secondary outcomes dan ligt de lat er meteen (hoog) voor toekomstige concurrenten. Belangrijke nuance volgens mij daarbij is dat hoe meer secondary outcomes gehaald worden, hoe groter de kans dat men nadien 1972 als blockbuster gaan bestempelen. Maar die primary behalen op zich lijkt me gezien de medische noodzaak al een overwinning.
Zeer benieuwd.
Hoe langer ik over die conference call nadenk (en de mini bewijzen die sindsdien reeds geleverd zijn), hoe meer ik geloof dat Onno niet gewoon wou zalven maar net heel zelfverzekerd was dat deze koersdip van tijdelijke aard is. Maar goed... eerst die readouts zien zodat de hoop en verwachtingen waargemaakt worden.