My question what would constitute a slam dunk?
The data
- 52.6% vs. 22.2% - e.g 30% more people did not need oxygen support and were able to move to room air breathing - Quite fantastic! - These may have been the most severe and closer to death.
- 89.5% vs. 66.7% - e.g. 23% more people not requiring supplemental oxygen - pretty darn great for saving on hospital resources and for patients' prognosis
- 73.7% vs. 55.6% - e.g 18% (so close to 20%) more patients were completely discharged from hospital altogether, which reduces massively the burden on hospitals staff as well as resources and shows the extent of opaganib treatment benefit
- 68.0% vs. 46.7% - e.g. 21.3% more people had a greater reduction of oxygen requirement in total across their time in hospital - again great for saving on hospital resources, staff and patient prognosis.
Meanwhile the safety which let us remember was the actual investigated purpose of the trial was excellent.
I consider these together to be indeed slam dunky if you will.
Of course with efficacy not being the intended purpuse of this trial, efficacy significance cannot be deduced from the 40 patient trial of which there we 20 patients in the control and treatment arms.
The upcoming global Phase 2/3 efficacy study interim results on 135 patients and thus approx 67-68 patients in each arm should provide a much clearer picture. And then of course if all goes well, the final 270 sample date expected later this quarter with 135 patients per sample should be very clear.
I don't feel people should be making comparisons between the non-powered efficacy in this small safety trial with the 'data' from the compassionate use results (not trial).
In the compassion use, opaganib was given to 5 patients, and compared to 18 matched-case controls. Those results showed 100% of the 5 patients went from requiring oxygen to room air in the 14 days, compared to 33% of the 18 match controls.
While 100% looks great, it is not realistic in the bigger population, and therefore seeing 52% in this slightly larger trial, is actually a better indication. Let us also note that in this slightly larger trial the % of controls moving to room air also went down significantly by 1/3rd, from 33% down to 22%.
This sample was not powered for efficacy, but the benefits seen are very substantial and striking with no important adverse events, in fact less SAEs were found in the opaganib vs placebo group.
Looking forward the efficacy powered data, but I wouldn't see why based on similar data that regulatory bodies wouldn't approve this.
