Shorting biotechs is something I rarely, rarely do. But, IMO, RPTP would be an excellent candidate. Biotech valuations across the board are wacked, and the market can't keep going up incessantly can it? But to go up on the back of clearly negative trial results is a real trick - and a real opportunity to short for those willing to do so. Could have said the same about PRAN when it spiked post-data release. In general though, I much prefer to have cash on the sidelines and buy things I like at cheaper prices - if the biotech market does cool off.
No, because failing to meet the primary efficacy endpoint is bad news. Not to mention the real possibility that it caused harm to at least a subset of patients. Those conclusion require only basic literacy, not insight.
Personally, I thought the results were quite poor. Amazing to me how well (some) biotechs these days spin the results in such a way that much of the public buys into it and the stock goes up. PRAN was worse, but RPTP wasn't much better. Interested if anyone can really make a bullish case for the Ph2 results 'cause I thought they were awful.
Congrats if you bought in at $1.53, that's a better return than I've ever had. Don't fall in love with the science - the data need to support it. Essentially, my beef with the stats issue is I don't believe PRAN used the Bonferroni correction when reporting stats. FWIW, I wasn't real happy with how RPTP reported their results today. They were a bit more straightforward, but they spun the trial as if it were a rousing success when it was anything but - not to mention a small-sized Ph2 done in one country overseas. I think PRAN and RPTP (and, unfortunately, many others) are issuing "Propaganda Releases" rather than "Press Releases". I think it is very close to unethical.
Doesn't it concern anyone that PRAN is deeply negative on a day when virtually every biotech except ONTX is "green". Do you really think this is the result of market manipulation or Wall Street ignorance? In LAYMAN'S TERMS, this is not good! And the p-value explanation was ridiculous - when you do multiple tests for significance, you have to control for the fact that one is increasingly likely to find a "false positive" signal for significance. So, when you're looking at SO MANY interactions (i.e., dosage x test x time tested, etc.), to obtain a single finding at p
And the rational investor tries their best to not let their biases skew their perception of new data/information. I have, unfortunately, violated that law on more than one occasion, but the "rational investor" tries to learn from experience. The data were poor, and did not inspire any faith. It is not typical that a stock tanks the day after an important press release. To attribute the recent price action to anything but a ringing lack of confidence in PRAN and the HD results is, IMHO, irrational.
Post-euphoria, the sober reality sets in. The efficacy results were slim-to-none. That is PRAN's own conclusion. They chose to spin it in a very misleading way (my subjective judgement). If those in the know really thought PRAN had a valuable drug for HD, the stock price would reflect this. And I don't just mean today's action. The price is more than 30% of the high, when the biotech market has performed much better and after the release of the HD data.
The stock is traded on NASDAQ, so their press releases to American media outlets should be more inclusive. A drug for HD is never going to be approved based soley on safety, so I would still maintain that their news release was highly slanted and misleading. Agree with your other points. Unfortunately, the number of biotechs who "data-mined" Ph2 data in order to justify running a Ph3 that ultimately failed are too numerous to count. Some Ph3s (e.g., KERX w perifosine) had to be stopped because harm was being done to patients. Everyone is rooting for the HD patients. I just can't support what PRANA is doing.
What "gives" is scientific integrity and transparency. What "gives" is that the efficacy results are quite poor and they don't want anyone to question them or interfere with the pep rally of a CC they're going to throw.
Then why not share/include the full efficacy results in the press release sent out to the newswires vs. what was contained only on PRAN's website. It's hard to see how scientists (not that you or PRAN are the only ones doing it) forgo scientific integrity in service of spinning data in the most favorable way, aligned with corporate goals of promotion.
It would be nice if people read this before commenting. I haven't edited the info at all or simply taken some quotes out of context. Ask yourselves, why wasn't this info released to the news agencies? Why did one have to link to it, then scroll down to the bottom of the news release on their website?
Secondary Objectives: Efficacy
Main/Primary Efficacy Endpoint: Cognition
Main Composite Cognition z-score and Exploratory Composite Cognition z-score. No statistically significant changes.
Executive Function Composite z-score: PBT2 250mg showed a significant improvement at 12 weeks (p=0.005) and trend at 26 weeks (p=0.069) compared with placebo. On a pre-specified subgroup analysis of early-stage HD (TFC 11-13), the change in Executive Function Composite z-score from baseline at 26 weeks was significantly improved in participants receiving 250mg PBT2 compared with placebo (p=0.038).
Of the two tests within the Executive Function Composite, there was a statistically significant improvement in the Trail Making Test Part B after 12 weeks of treatment compared with placebo (p
You should know better than anyone then that their statistical methods are highly questionable and misleading. I have a Ph.D. in the social sciences as well, and have been a biotech investor for 20+ years. Bottom line, PRAN is torturing the data and the way they spin it. Again, I am extremely confident that other companies will see it this way - none are going to give them an upfront payment and fund Ph3 trials based on such flimsy efficacy data. And the FDA - if PRAN were to ever progress to the point of submitting an NDA - is going to be a lot tougher on them than I have been. The key line is BURIED in the extended news release on their website. Here, I am cutting & pasting from their release:
Main Composite Cognition z-score and Exploratory Composite Cognition z-score. No statistically significant changes.
Secondary Objective: Efficacy
Motor, Behaviour, Function, Global Endpoints
No significant changes were seen in motor, functional, behavioural or global assessments in either PBT2 treatment group compared to placebo over the 26 week treatment period.
A small but positive signal in TFC was observed on the 13 point scale across the PBT2 groups relative to placebo (mean changes from baseline:
250mg PBT2 = -0.3; 100mg PBT2 = -0.5; placebo= -0.6.
Secondary Objective: Efficacy–Biomarker Endpoint
There were no significant changes in the urine or blood biomarkers assessed at week 26 with PBT2 treatment compared to placebo.
Does it bother any longs that the company's own press release - buried in the small print on the copy on its website, but nowhere to be found in the PR released to the newswires - concludes that there was no statistically significant difference between drug and placebo in terms of efficacy. Now, when you look at multiple measures at multiple time points with multiple doses with multiple patient groups, odds are you'll find something that is p
I retain 50% of my original stake in CUR and remain very bullish on the ALS trial. However, I see no reason to be optimistic about NSI-189. Seems to me that if they had stellar results, they would have released them long ago. More tests, more data crunching are not great signs to me. Also, everyone needs to quit kidding themselves, management would have loved to partner the drug - their long-stated intention until recently. I just don't think there is any interest in it - or the proposed deal terms aren't great. CUR will sink or swim based on the ALS results. NSI-189 is an afterthought - limited data, hugely expensive to develop, no partner interest and many, many years from market - if it would ever get there.
Did you listen to the OREX JMP presentation? I feel much better about OREX extending Contrave into diabetic populations where they have published some data than ARNA extending Belviq into smoking cessation w/o any data (and a far smaller market).
Give me a break. I already specified the multiple timelines that management missed. Go back and listen to or view their archived presentations. I also didn't specifically say CUR share price is due for a correction, but that I am betting the biotech sector is due for one. And CUR became overweighted in my portfolio because I thought it was a great value at 1.4 and it subsequently appreciated well over 100% - may you have similar "problems". Too, don't know if you're active on Twitter, but when Neuralstem PR tweets about a patient's blog, a patient in a current trial where outcomes are unknown, I think that is a bit much. Finally, I remain long CUR, don't own other stem cell companies, and have been investing in biotech for over 20 years. That experience gives rise to my concerns about some actions of current management. Ultimately, in CUR's case, I am investing because of the ALS drug and its compelling, though early-stage data.
1) While I am very much expecting there to be a "correction" in biotech stocks, I view OREX as one of the few true value plays in biotech. Their Enterprise Value (MC + LTD - Cash) is quite good, and will look much better once Takeda milestones (and possible monies from a EUR and/or ROW partnership) kick in. Needless to say, I think their EV is much more inviting than that of VVUS or ARNA
2) The Light Study results are going to allow them to dominate ARNA and VVUS in Eur/ROW countries - if the other 2 ever do get approved - and I didn't realize how lucrative those territories can be until I listened to their JMP presentation yesterday. While they won't receive significant revenue from ex-USA countries until 2016 or so, I believe "the Street" is undervaluing this income stream.
Sentiment: Strong Buy
Missing timelines absolutely affects how "the Street" perceives management. As it is, they are really coming close to pushing other (ethical) boundaries by promoting the "tweets" of ALS study participants. Remember, management was providing the timelines, not some analyst. NSI-189 is a long-shot, plain and simple. And, at least in depression, would require resources for trials and marketing that are way, way beyond the reach of CUR.
Personally, I've sold 40% of my stake for the following reasons: CUR was becoming a very top-heavy holding in my small portfolio; I'm very leery of the valuations for most biotechs currently and am planning for the possibility of there being a "correction" soon; I do believe there are a variety of red flags with management. Why didn't I sell the other 60%? Because I am willing to wait for, at least, the interim ALS results in April and see what the data look like.
I've been a biotech investor for a long time, bought into CUR at $1.48, and have followed the company for a couple of years. I stand by what I said. The worst thing any company can do is over-promise and under-deliver. If you've heard the company present, they missed publicly stated timelines for several trials (e.g., Mexico City ALS trial, both SCI trials, results/partnering for NS-189). Those are objective facts. Subjectively, I consider them to be "red flags", and I'm also not thrilled with their latest financing - neither the timing nor the institution involved.
I am invested in CUR because of the promise of the ALS drug and the published data to date. To their credit, they HAVE delivered on their internal milestones/goals for this program. The outcome of the Ph2 trial will likely be one of those "binary" events for a small-cap biotech.