Very interesting, esp. the development plans of SMN, TTR, ApoC-III
possible new drug targets with splicing technology:
Duchenne muscular dystrophy,Thalassemia,Progeria,Neurofibromatosis type 1, Cystic fibrosis, Pheochromocytoma, Long QT syndrome, Familial dementia
ApoC-III : Phase II Proof of Concept data -- early 2013/Phase III initiation 2013/potential commercial launch 2016 --- significant licensing opportunity in 2013
-- 2012 clinical data events: CRP, GCGR,GCCR and FGFR$
The analysts ask the same old questions and the company shields a little. What I glean from this conference is, big pharma is putting up big money to accelerate certain aspects of certain ISIS programs. ISIS is leaping to phase III from Phase I. Impressive and more so, because big pharma is paying for it. Very simple to read for yourself. Big pharma has its own access to the data and understands the implications. Antisense technology and drug technology will be used to modulate the lipid profile of possibly millions of people, as a function of time. If this unfolds well, the numbers are not 3000/6000 patients with X or Y abnormality, it will become a preventative and early maintenance issue and technology platform. The numbers will be much larger and big pharma knows this.
All the analysts know is to poke around for numbers. They ask the wrong questions as they are too focused on finding something to hype, short term. The long term aspects of this work look astonishing and big pharma knows this. Lipid management will become a center stage issue, bigger than proper nutrition, diet, exercise, this is beating the genetic primer.
Consider the blue whale. He starts his life about the same size and weight as a human, as a single fertilized cell. His genetics makes him grow a little bigger than a human and carry a bit more lipid content. A blue whale needs its lipids for obvious uses. Buoyancy, insulation, energy store and retrieve. A human, with our current technology base, now has a very different lipid distribution and content *need* than what nature provides, and as we know from statistics, if you can manage this to perfection, long term, the health risks for/to your organs are reduced, heart being first and foremost. This genetic *tuning* opportunity sits side by side with drug management of genetic-driven conditions. However, genetics are not the only factor at play. Life style is also at play here. And yet, the genetic aspect still plays in heavily for human-kind at all times and all ages.
This is a pathway to genetic management (antisense) of lipid type and distribution in human subjects, regardless of what they eat, how they diet or how well they exercise. At least, within a reasonable range of expectation. And for the extreme situation, such as genetic abnormality, this pathway is possibly the sole source for some conditions. If the future holds the promise of genetic management of health, then this would explain, at least in part, why big pharma is investing. And these analysts have not caught on at all, based on their short-sighted questions. I do see and agree, that ISIS needs to get the product into the market and prove its point. The efficacy they get to guard in trials, has to become known to investors as *provable* and so, as the pathway unfolds, credibility and confidence builds. Frankly, the chart shows a broken past. Is it finally time to emerge with something that really works? Big pharma thinks so. That's the key here. Not what analysts think. What big pharma knows. And in fairness, big pharma gets to see more.
But analysts are moving stocks! Sarepta with only one Phase II drug has the same company value as Isis that´s incredible. The presentation slides stands out because they show the money! Besides the already partnered drugs there are seven drugs with Phase 2 efficacy data expected in 2013/2014 with significant new partnership opportunities. ApoC-III in early 2013 is just the beginning.
In the next six weeks we can expect clinical data on four drugs (CRP, GCGR,GCCR and FGFR4)
as well as 2-4 new drugs into the pipeline. Unfortunatly all these impressive pipeline developments has been overshadowed by Kynamro.