Holden YMB:
Folks,
There are many exciting things about this. It skips p1a SAD and goes right into p1b MAD seeking recommended dose level regimen. As soon as they have this under AD they will roll right into p2a optimization study (optimal number of doses). Again, they roll right into the p2 upon submission of p1b data. Note I didn't say p1a safety data. This is the first ever AD from the very beginning of the study. This is, IMO a HUGE validation that TRiM is a completely safe deliver platform. NO SAE's of which we are aware. This is the first IND ARWR will have filed directly with the FDA before doing off shore early-stage development studies. This is a massive vote of confidence from the FDA and should be celebrated. And when they are done with p2a they will be able to roll right into p2b with only FDA data review. I am assuming they will be study various patient populations and perhaps a pediatric population, but that may be down the road a bit. (Not sure ccRCC is a pediatric cancer) No new IND from data submission to data submission. So Fast Track Designation, PR and BTD are right around the corner. This is going to be the fastest oncology study in history. This will be the first RNAi extrahepatic study ever and they have a strong multi-year lead in that regard. I am sure we will find out other positive bits as PRs and filings continue. Will it be a global study? Likely...Lastly I assume because the company is so effective at trigger design they will be able to branch out and attack many solid tumors. They still need to discover the differing receptors but time to clinic from discover should be VERY fast. I suspect they already have many receptors lined up, but that is speculation.
Dr. Cheddar, unless some exogenous circumstance exists, new oncology molecules always start in Stage 4 cancer. The thinking there is that these people are very likely going to die, have been treated with the highest standard(s) of care for that circumstance and the therapeutic either wasn't effective or the patient built up a tolerance. As long as a drug can achieve whatever safety/efficacy hurdle is embedded in the application then it's OK to inject the drug. If it is as safe as other compounds in really sick people and provides a measurable improvement than the current standard(s) of care, the thought process continues, you can start studies in earlier stages of the disease and eventually pediatric patients. Or if it proves safer and achieves the same level or better efficacy the same holds true. Some cancers are ~exclusively pediatric. It will be most gratifying to see ARWR cure say, Ewing Sarcoma, for example, is almost always a child cancer. It's a painful death sentence and has no cure. Tragic for all involved.
Exciting times!!!
finance.yahoo.com/quote/ARWR/community?p=ARWR
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