Biotech Values

[caravon]

From ARQL Q-10:

Daiichi Sankyo recently provided us with the final data set from the MARQUEE trial (with a cut-off date of December 15, 2012), including analyses of the pre-specified sub-groups prescribed in the statistical analysis plan (“SAP”) for the trial. These latest data analyses confirm a statistically significant improvement in progression free survival (“PFS”) in the intent to treat (“ITT”) population (approximately 1000 patients) for patients receiving tivantinib, but the PFS benefit did not carry over to overall survival (“OS”).

These analyses included an exploratory analysis of the MET IHC (immunohistochemistry) sub-group comprised of 445 evaluable patients. Of these, 211 patients were confirmed to be MET-high as defined in the SAP.

The MET high tivantinib group showed a substantial improvement in OS relative to control a benefit which was not seen in the ITT population.

By comparison, 234 patients were confirmed to be MET low, and in this cohort of patients, no difference in OS was observed. PFS in MET-high and MET-low populations were similar.

The c-MET high pts comprised only 47.4% of all ITT patients instead of the 75% Daiichi/ARQL management expected. Consequently, the trial primary endpoint mOS in ITT NSCLC population was doomed from the start of the trial.

Marquee was a successful Ph2 trial instead of being a Ph3 trial Daiichi/ARQL poorly designed.

Finally, I was correct expecting the Tiva efficacy (including PFS and OS) in C-MET high NSCLC patients.