The study, which randomly assigned 12 boys to a couple different doses of the AVI drug or a placebo, showed the boys were able to produce about 22.5 percent of normal dystrophin levels after 24 weeks on the therapy, while there was no increase on the placebo. No serious adverse events were reported in patients on the experimental treatment, and no patients dropped out of the study, AVI said.On the downside, their was no improvement with the higher dose and worst of all, the boost in dystrophin had no clinical benefit. Researchers saw no difference in patients’ walking ability in a standard 6-minute walk test.
The study’s lead investigator hailed the finding as a “major advance”.Opposite of Dendreon, Avi has evidence that they can make the body do something very specific. Dendreon made the claim that they could elicit an immune response resulting in tumor cell death, yet they could not provide evidence of that MOA. Avi made the claim that they could make the body produce dystrophin and they provided information claiming that they did. Yet Dendreon provided a clinical benefit to their unsubstantiated MOA. Avi could not provide evidence of clinical benefit to their substantiated MOA.
From a scientific standpoint it is indeed interesting that a piece of RNA can accomplish this result. Eteplirsen is the therapeutic candidate for DMD and uses their core PMO chemistry applied as a splice switching oligomer (SSO). The drug is intended to skip exon 51 of the dystrophin gene. By skipping exon 51, eteplirsen may restore the gene’s ability to make a shorter – but still functional – form of dystrophin. From this point one would think that the scientific community would begin conducting research into skipping exons to restore or improve gene function. What we do in biotech is swing for a home run however. In this case I don't think we have much of a chance. Are we altering our DNA or are we throwing nucleic acids at the body and hoping the DNA will know what to do with it?
The investor community gave the major advance a thumbs down. The stock went down as much as dystrophin went up, 25%. This could help investors understand such claims of 25% changes. If the stock were worth $100 they would have lost $25 per share. Instead, after a 25% loss, the stock was worth $1.16, a loss of 38 cents. In terms of what matters, the percentage of change is less important than the consequences of change.
When dealing with our DNA, we are dealing with evolution. Can we change millions of years of selection that has produced human beings destined to live short difficult lives? Of course we should be trying. But is this the best way? A for profit effort hell bent on surviving one month to the next? The desperation in the careers of the Avi scientific staff leads to certain outcomes that ignore the sad results of this trial. In a fair world the science would be handed over to an academic lab to learn more about what happened. The expense of moving on with more clinical trials, the suffering of the patients through this invasive research on their muscles is not warranted. It is time to get back to learning about our DNA and to move away from trying to make it stop doing something evolution has somehow allowed.