TopoTarget is a European biotech company that licensed the histone deacetylase (HDAC) inhibitor belinostat to Spectrum Pharma $SPPI. In June 2012 Topo released top-line data for the clinical trial in cancer of unknown primary (CUP) - belinostat failed to achieve the primary endpoint of an increase in progression-free survival (PFS). A secondary endpoint of objective response rate (ORR) was positive and statistically significant.
The company then held a conference call to explain the results and justify to shareholders why they shouldn't view this as a failure. They even went so far as to release a further press release in August 2012 with some new data-mining. This sort of behavior is typical of small biotech companies heavily reliant on the fate of a single drug, and diligent biotech investors must recognize this and not invest in a company advancing a drug out of necessity or convenience vs. actual clinical and commercial potential.
My notes from the conference call continue below the jump.
The company then held a conference call to explain the results and justify to shareholders why they shouldn't view this as a failure. They even went so far as to release a further press release in August 2012 with some new data-mining. This sort of behavior is typical of small biotech companies heavily reliant on the fate of a single drug, and diligent biotech investors must recognize this and not invest in a company advancing a drug out of necessity or convenience vs. actual clinical and commercial potential.
My notes from the conference call continue below the jump.
why was a benefit seen in ORR but not PFS?
<p><span >claimtoken-50c538f272550</span></p>
- heterogeneous patient population
- several factors, will have to look at subgroups
- we are taking the ORR data as evidence for clear clinical signal
- per protocol vs ITT has some impact but that doesn't give a different result
- We are in dialogue with clinical experts to evaluate the results and see what patient population would benefit most for future trials.
- Belinostat+Cap has biological activity and was well tolerated. promising signal as treatment modailty for solid tumors.
- We also have signals in ovarian and bladder cancer
- responses occur early but dont last very long. but in maintenance treatment there is obviously a benefit
- we are finalizing our PTCL trial and complete top-line results will be available later on this year.
- 7% CR, 36% PR on belinostat arm
- no reason for concern on safety
- thrombosis patients (n=6) all had local explanantions
- "fully warranted" to continue development of belinostat in solid tumors: lots of preclinical and clinical data that is encouraging us
<p><span >claimtoken-50c538f272550</span></p>
RSS Feed
