Putting One's Best Foot Forward - the June ReadoutHere is a quote from de Guttadauro from the year end conference call:
"With several patients still receiving treatment, there is an opportunity for a truly impressive endpoint that we could sell ourselves short if we rush into a deal prematurely based on data that could improve over time. With industry leaders such as Pfizer, Roche, Merck Serono, Bristol-Myers Squibb and Insight already familiar with pela thanks to collaborative clinical trials, we intend to run a methodical and competitive process that ensures we reach the best possible outcome for our shareholders."
de Guttadauro, Heineman, and Coffey in their various ways all express confidence in positive results to come from the BRACELET-1 data release in June. Here de Guttadauro speaks of "an opportunity for truly impressive results." This is coming from guys who have real-time information on how the trial is progressing. They are promoting and waiting for the June readout for a reason. They are also being clear with biopharma that they are running a methodical and competitive process. They want BP to know the deal making process they are following. No favourites, no selective release of information, but rather a level playing field for honest competition. ONC/Y is putting its best foot forward so BP will put its best foot forward. They are doing what they can to generate multiple interested parties and to set before them the most compelling data. I wonder if ONC/Y is continually metering out real-time information to interested parties with confidentiality agreements, or if they are holding back some maturing data from them as well until the big reveal in June. That could be smart, but as with so many things I really don't know what is happening.
BTW, the data that will be complete by early June will be the ORR (Objective Response Rate) and the PFS (Progression Free Survival). The ORR is complete responses plus partial responses divided by total evaluable patients ((CRs + PRs)/ total evaluable patients). PFS is the length of time until a patient has more than 20% tumor growth following treatment. Median PFS (mPFS) is the length of time until one-half of the evaluable patients have had more than 20% tumor growth following treatment. As Heineman said both ORR and mPFS are fairly predictive of a positive clinical outcome and should be sufficient to make a decision to initiate registratioin trials. However, the ultimate goal is substantially increased overall survival (aka durability). That will slowly come into focus, but likely not be finalized until sometime in 2024. The clinical trial completion date is given as July 2024, which is two years following the enrollment of the final patient.
Immuno-therapy is unique in that sometimes a mPFS benefit does not materialize, but OS is greatly improved (like in IND213 and in an earlier panc trial). However, from the comments of Coffey, Heineman, and Guttadauro it looks like they are expecting good ORR and mPFS results in June. The June readout is "crucial" as both Coffey and de Guttadauro have said. So they are sticking their necks out and drawing lots of attention to the June readout. Hopefully that means they are rightfully confident of the results.