I sold my positions for a profit already. The mid month presentation won't move the share price so you are left with waiting for June data. I predict June data will be positive because of the large license agreement. You may get a 10% move on that.
If Woodcock approves via AA it will be along the lines of "There is some evidence that the desired mRNA is produced as supported by RT-PCR. At this time it is unclear what the minimum level of dystrophin is required for a clinically meaningful benefit but based on expert testimony we have reason to believe even a 1% production is considered meaningful in this disease left without an alternative treatment. We are requesting Sarepta to run confirmatory trials to measure dystrophin levels...." That's what everyone should be investing on. I work in the industry so I'm investing from a policy standpoint. This is unprecedented...so I give it a 90% change of CRL and 10% success. Again, just based on if I was the one with trial results and had submitted the NDA.
You are not objective. The FDA are being objective here. They've stated in simple terms, based on SRPT data we cannot draw any conclusions except for exon skipping. We can't tell 1) the amount of exon skipping 2) the amount of dystrophin production 3) the min. amount of dystrophin needed for a meaningful benefit. The database of DMD shows that dystrophin levels low and high is not a predictor of ability to walk (historical control problematic) and marker not well defined in literature. What every investor is left with is 1) Exon skipping occurs 2) Anecdotal evidence 3) Scientific investigator letter 4) Congress letter. Woodcock may approve via AA based on exon skipping alone because a thorough review of DMD database shows dystrophin marker is not as predictable as original expected. A
Sarepta had their hand held to get through the trial and yet they've failed on every manner for conducting a clinical trial. Sarepta and its study investigators have failed the DMD community. The only thing I can think of that could possibly allow for AA is evidence that exon skipping occurred. A followup trial would be needed to properly measure before and after of dystrophin levels. That's what everyone should be investing on. Is exon skipping enough proof for AA? Or, does dystrophin level need to be proven to get AA? Luck is needed for everyone betting on this one since the panel voted No 7-6.
Sarepta is very very risky. The lone investment thesis for it is that there is no alternative treatment therefore anecdotal evidence (signed expert letter) is enough for AA. The disease is devastating therefore what is the risk/benefit ratio? If it is very small but based on a poorly run and poorly analyzed trial should it still be approved? The FDA's staff repeats in the ADCOM AA cannot be a substitute for substantial evidence.
0.2? Ill be buying more if that's the case. Adam did the same thing before CPXX. Whatever did happen to re-examining sub-groups within a phase 2 or phase 3 and not being punished for it? Biotech investors have become so spoiled as of late. Let's put a 1 B market cap on a fad/unproven science and slowly reduce expectations because that makes SENSE (sarcasm).
I'm willing to risk 5 k on this. If I lose it not a big deal to me. Its a small portion of my portfolio for a potential 5 to 10 time return. Even Adams source admits possible weak effect... if the source was confident he or she would say no effect without covering themselves. Enough doubt by Adam and the source keeps me smiling. Notice the confidence in ctrx failure piece vs mstx. Adam has changed his confidence in mstx failure.
He follows Mr. Market, points out procedural weaknesses (trial design etc) and points out management or BOD's unethical behavior. This is important so I believe he has a place for rooting out biotech frauds and scams. When Adam reviews scientific data though it's usually through his one or two sources. If you disagree with those sources then make your long investment. Adam like us can't tell the future so he doesn't write in the affirmative. There are times when I feel he overreaches and bullies certain companies. The best thing about biotech investing is...Mr. Market is not always right or is caught napping. That's why there will always be 400% gainers now and then. Enjoy.
I think it's funny that Adam thinks Sarepta has better odds of approval than MSTX having a successful phase III. Sarepta by all means has failed 1) trial design 2) trial analysis 3) data integrity 4) biological marker selection 5) negative adcom. The only thing Sarepta has is patient advocacy and anecdotal evidence that wasn't measured in the trial. Whereas MSTX has a several large clinical studies, a proper sub-group analysis, and interim statistical analysis as well a revised phase 3 trial.
No mention of the statistical look at blinded data during enrollment and the treatment hours between NA and Europe patients...a positive.
It takes time yes, at about 80 hours the effect shows up. The goal is to show a 16 hr improvement which the revised primary endpoint will be able to capture. This is what I took from the article 1) Adam compares MSTX to L.C, clever journalism for followup 2) Source investor not shorting 3) Trial data release is near 4) Large exposure - more eyes on MSTX. This is a 10:1 return, putting $1000 down for $10000, if it fails you will still have $500. Well worth the risk.
Relax people. A.F. is clever and wrote his piece with some self doubt/potential positive to write a followup. He got burned in CPXX and won't write in the affirmative. Notice he compared the stock to Leicester ...guess what??? Leicester won the EPL. It's an interesting comparison. Also, I wrote earlier I am reliant on the K.M curves for my investment thesis as well the extension study. Note his source investor did not short this stock which means the source investor thinks it's undervalued and could explode if successful. The K.M curves were significant and the x hr explanation only makes sense if the biology of vepo is clear. In this case, it is not 100% clear why with the half life that pain subsides thereafter, the source investor is only guessing. But, you can hazard an estimate that pain subsides over x amount of time past the vepo administration. Therefore this is why the primary failed in the first phase 3. The second phase 3 is lenient and will be able to detect the 16 hr plus difference. Remember, there is an extension study...people are enrolling which tells you it works.
Sarepta and ccxi right now. I was short sarepta but now have calls and am waiting on a long shot approval. The aa that everyone believes it will receive is hard to get with a negative adcom vote. As well everyone in the fda was negative on the trial protocol trial analysis...only thing to go off of are the expert testimony. The fda needs to decide if bad data should still be accepted even if experts in the field cite anecdotal evidence. Ccxi I'm waiting on classic results avg at 3.2 so I'm going to watch the covering that will start...day 3. Enjoy.
I have similar probabilities and have reviewed all published data. The stock is where it is because of the size of the float and like others have mentioned....past dilution. I see the science as being sound and any reduction of VOC would be beneficial to the patient therefore 16 hr to 24 hr+ would still mean a 500 M market cap company. I am always for a company that runs an extension because it requires work and $ to the company and investigators. This is my core thesis "In particular, for patients who may be hesitant to participate in EPIC because they may be randomly assigned to receive the placebo control, EPIC-E can provide the certainty of receiving vepoloxamer for subsequent crises. EPIC-E also will give study investigators experience administering vepoloxamer to sickle cell patients in an unblinded manner, which we expect will provide valuable feedback for us." There is evidence in that statement that the treatment works therefore an extension was run.
No one thought it was interesting that Vifor Pharma wanted the license agreement completed before CLASSIC data release? And...in addition they plan to pay $25 M at 7.5 per share? That's confidence!
I don't know if it is Cohen or Baker Brothers, but someone is definitely buying and targeting between 3-5.
Hard to say...I'm holding until 5-7 depending on Classic results. I don't usually speculate but this looks like Cohen or Baker Brothers accumulation which is causing a small squeeze.
Wow, lucky me this is still undervalued. I'm buying for the CLASSIC CCX168 trial results which will bring it to $7. This license agreement was a bonus.
A billion is too much. I would think that for the 7 year exclusivity and the only VOC treatment, along with several additional indications, would be near 350-500 M market cap.
Smart people don't short a $0.3 stock. AF needs to back his argument up with available journals that counter the performance of P-188 in vitro etc... Also, he needs to dissect the primary endpoint, is it relevant? Why was it changed from JAMA? I know he hates post-hoc analyses so is biased there, but there's plenty of successful trials that relied on a previous failed trial. SCD treatment responses are different for pediatric vs. adult which bolsters my confidence.