Agree. They're also forecasting infant trial to be fully enrolled in Q2. And they will also update Phase 2 results in Q2. Based on ibrutinib accelerated approval, I'm expecting these to be key events. The FDA wants controlled trial data and the earliest possible approval. Approval before full enrollment doesn't make sense. Why risk randomization if you can get the drug commercially. My expectation is approval 4 months after the last patient is enrolled. So my bold prediction is an accelerated approval by October.
...from last investor conference, the next milestone will be the completion of enrollment for the Phase3 trial later this year. No new data will be presented this year.
From my recollection of the Ibrutinib approval through the breakthrough pathway, the accelerated submission plan was revealed after the Phase 3 trial was fully enrolled. Up to that point, management pretended that no accelerated submission was expected. My sense was this was the "script" choreographed by the FDA. The FDA didn't want a crowd of activists at their door driven by company hype. And the FDA's real goal was to achieve quick approval AND subsequent hard data from a controlled trial. If patients expected quick approval they wouldn't enroll. This is even more of an issue when you have a sham of a sham-controlled study.
me thinks AH speaks 1% truth. Dont worry i like the dark duck after all is said and done
Type 3 patient looking good.
Find this YouTube by Googling this, "New drug for SMA shows profound effects in clinical triall.
I wonder if any Type 1s are sitting unassisted. This would be unprecedented.
The Phase 2 study of mipo showed FLS in every dose group. Treatment ranged from 5 to 13 weeks. SA guy sees no evidence of reduced FLS in new GEN 2.0 compounds even though they showed 0 FLS in multiple studies of similar duration.
Geez. Tough crowd.
Silly people. You see...the way a signicance test works is it looks at both the sample size and variance among patients. So you don't have to argue about whether the differences are meaningful. The test does the math and TELLS you. If the sample is low, but variance is low, then you can bank on the results. There were only 7 patients on drug. But ALL 7 had dramatic changes in triglyceride, HDL (!), and the rest. The significance test tells you to bank on it.
Safety and tolerability is another question.
I really do.
But the immensity of the opportunity keeps reeling my back.
Leukemia lymphoma society prevalence:
~30k CML patients in us = $1B+ Gleevec sales in US alone
Ibrutinib Population 20 Times Bigger:
~130k CLL patients
~500k NHL patients
Living longer = bigger populations in the future
Chronic treatment = each new start is NOT offset by patient going off treatment for long time = fast long growth
Low side effects = backbone for all combinations
Approval timing exceeding expectations
Massive clinical development plan enrolling at record pace.
Rest of world
Other cancer indications
Even if my revenue estimate is off by a factor of 10, PCYC is still a good value. So what's the risk. I'll take the $ later.
Sorry if this message appears too excited.