re:"A lot of parents of children demanded for DMD medicine from SRPT in FDA meeting as there is no alternative medicine."
re: " Well FDA did not listen - they demanded more evidence."
I don't believe that's accurate. So it isn't really a good comparison with AVXL in regard to future FDA approval.
As I recall, the FDA's main objection was that SRPT's approach was to alter the patient's DNA, an irreversible process. Hacking a person's DNA is tricky and not exactly safe. Moreover, they do not repair the code, just in effect, delete the bad part resulting in a truncated protein that is kind-of like the one the cell was supposed to produce, and hope that it is better than nothing. Moreover, the the targetted exon, which might be likened to subroutine data they are skipping over, is only relevent in 13% of the patient population. Also, results so far indicate that the cells seem to be reluctant to produce enough of the new protein, not as much as expected. They may be missing something there. So, the evidence is that it's not a very good solution for DMD and the best that could be said is that it needs more work. Considering the evidence, one would tend to side with the FDA in this instance.
Just to clarify what we are talking about:
"Duchenne Muscular Dystrophy is an X-linked recessive form of muscular dystrophy, affecting around 1 in 3,600 boys, which results in muscle degeneration and premature death. The disorder is caused by a mutation in the gene dystrophin, located on the human X chromosome, which codes for the protein dystrophin"
re:"A lot of parents of children demanded for DMD medicine from SRPT in FDA meeting as there is no alternative medicine. "
fwiw, Actually, there is an apparently largely unknown underdog in p3 trials that imo has a much more promising solution. But it's a long slog, because, after the initial diagnosis, it takes several years for the dibilitating and ultimately fatal symptoms to manifest. It has occupied a small place in my portfolio next to AVXL for some time. I'm not going to mention it by name but if someone does some DD they can find it.
Muscle fibers need to be able to slide over each other when the muscle moves otherwise the stick and can basically tear themselves apart. The kids are initially ok because the cells produce utrophin, a soft more grease-like lubricating substance. After muscle growth is complete, the cells are supposed to switch over to dystrophin, which is a more hard and permanent coating, which could be likened to teflon. With DMD, the dystrophin doesn't happen and because utrophin production has ceased, the existing utrophin wears away, gradually disappears. Then the patient is in trouble.
From the competitor's website:
"Our utrophin modulation approach aims to use small molecule drugs to maintain the production of utrophin to compensate for the absence of dystrophin in DMD patients and so protect healthy muscle function. A significant advantage of utrophin modulation is that it is independent of the underlying genetic fault and therefore has the potential to treat 100% of DMD patients."
Gee, AVXL uses small molecule drugs don't they? Also they don't harm or otherwise irreversably alter the cells DNA or anything like that. Obviously differences, different diseases, but a much better overall approach with much better prospects for success. So thay have that in common.
So far, the company hasn't come under media attack. Maybe the daily volume has been too low with its wide spreads are unattractive for shorting . I don't know, I was a little reluctant to even mention it here. I hope it stays under the radar of being a target for "unusual trading activity" like has occurred with AVXL.
