from yahoo
The real valuation of Imetelstat isn't in MF, it's in the expanded indication list.
MF is a deadly disease, the available treatments target symptoms, w/the exception of SC transplant. But few pts qualify for SCT, there can be a host of complications, and selectivity isn't an exact measure. As for Jakafi, it works for some, fails for others. In the case that it works, it's still not curative; and when it doesn't work, it may contribute to disease state progression.
In contrast to these, Imetelstat put dying patients into CR, which the NCI defines as "The disappearance of all signs of cancer in response to treatment." That's not measured in a few extended months of PFS or OS; this is pts being cured IN PERPETUITY, so long as they stay on Imetelstat. And the CRs were earned under rigorous standards. The change in model of treatment is A TOTAL PARADIGM SHIFT. People who were dying can now live like they never had MF.
Toxicity is real and serious. But it's entirely manageable through dose modulation, as the Mayo trial discovered. And all liver abnormalities resolve w/discontinuation, that's why the hold was lifted. Regarding toxicity in the children's brain tumor trial that Biopearl posted, that trial was designed early 2013, and the dosing schedule wan't all that different from Cohort B in the MF trial, regarding which Dr. Tefferi presented later at ASH as too toxic of a schedule. We know this, we're beyond this. The progress is on the Cohort A results. Now the goal is fine tuning the dose and finding selective markers.
I'm saying all of this just to put a point on why approval for MF is a given. Take into account the chasm between Imetelstat's virtual cure and SOC symptom relief, and you'll get why Imetelstat will be fast tracked with SPA, and the FDA does allow sale during P3. Best case, we'll see sales revenue during P3, and milestone payments too. Either way, approval in MF is fait accompli.
The real question is how the indication list might expand. JNJ will run dozens of parallel trials. Measure Geron's valuation in multiples of MF value.
"evil flourishes when good people fail to act"
