First sickle cell patient was dosed Oct 14, and the Bluebird should know if the patient is transfusion independent in less than two weeks - plenty of time for an ASH report. While guidance is for 2015, there may be an early surprise?
With another round of data in the coming months, BCLI stands a reasonable chanced for an expanded access program. Results for this program are rapid and interim data may be available following the 3 month endpoint measurement. The first site came online in June. Without surgery, this program can rapidly enroll patients and measure outcomes - far different than Neuralstem. To date there have been few side effects.
The path forward has a good probability playing out as follows:
1) In Vivo results confirmed in American Journal of Ophthalmology - vision improvement in over 50 percent of patients with spectacular results in some. This would appear to be superior to STEM's results, where further vision loss was only prevented in AMD patients. However, there may be room for both these cells..
2) With no current treatment for dry AMD, the market cap should approach $750 million to $1 billion, with a headline that some blind patients can now see. Share price should rise to a corresponding 22 to 39 cents. Approximately 3 to 4x current price. The largest investor opportunity for the next 6 months appears to be in the coming weeks, when the data will be presumably published, and positive.
3) ACTC is approved for either a 30 to 1, or 100 to 1 reverse stock split. I expect that once the data are published, assuming they are positive, the company with reverse 30 to 1. This will bring the share price to the $5 to $6 range, and facilitate the uplisting to NASDAQ. A reasonable assumption is that this will occur in Q.1.15.
4) A definitive phase 2/3 study will be put in place, an the company will receive breakthrough status in December.
5) Stock price may trade $7-9, split adjusted, on this path of milestones.
MRI is still missing - after all those blogs and press discussion by the CEO. The only direct measurement of depression was not significant in the trial - MADRS - yet the headlines screamed success. Even the consultant (that's how he was originally described?), FAVA, was promoted to a co-PI. A promotion, as I'm sure he's glad to help if CUR signs a contract for the phase 2 remote raters facilitated by Mass Gen? Now, that's good psychology.
Now two Brainstorm sites enrolling patients -- and there has been a frenzy of applicants. No surgery and no immunosuppression. Stat sig 2a data.
Garr is still focused on opening his Mexican ALS site, right to try in Colorado etc - things that will not ever happen. PR stunts?
CUR surgery is too invasive and Brainstorm appears to have better results with a therapy that can be administered chronically. And, now they are trying to repeat STEM's work in SCI with the analogous cells. SCI has been an extremely slow clinical investigation, driving one company broke, and not likely to yield any commercial results a decade, if at all.
STEM likely to win with AMD if ACTC doesn't leapfrog them...
You see full 2a Brainstorm data before year-end, and that is likely extremely difficult news for CUR. Not a bright move with $150 million shelf. What a shame.
Current shares outstanding 87 mil. Now add 150 million shares. LOL. Maybe the MRI data are positive - large effect size positive :)
In this case, CUR is unlikely to have a viable product following BCLI current success in 2a, and likely future success.
NSI 189 does not have any definitive data demonstrating its efficacy, and the company has mischaracterized the results, IMHO. Garr, once again, guided for MRI results for a year and the held them, and, after a year of calling Fava a consultant, promoted him to co-PI. All slippery PR moves to raise share price, IMHO. It worked, for awhile, and now the company will likely experience a large pull back, IMHO.
The share price, following the dilution, will provide for the company to continue operations for another decade of losses, if ALS is shelved. Or, more succinctly, salaries for staff. As the company became more well known, Garr's manipulation of the press, IMHO, is more widely known. He will now have to dilute prior to BCLI releasing full results for their 2a program. BCLI is also likely to have a compassionate use program putting further pressure on CUR to the downside. BCLI has the limelight.
Anticipate, dilution and further share price erosion.
Company has already indicated that over 50 percent of the patients improved, or had spectacular results in P1/2. Schwartz was on the agenda at the recent ISSCR conference several weeks ago, but did not present his session. This appears to indicate that the company had anticipated the results would have been available by the time of the conference.
It is reasonably probable that the share price will exceed .24 - $30 billion in world-wide potential with no effective treatment for improving dry AMD. 3x bagger on approval.
The spin factor:
1) NSI 189 MRI data promoted for a year and now unavailable
2) NSI 189 shows no statistically significant efficacy on only direct depression scale used in the study - MADRS. Company misleads by promoting the effect size which is not significant
3) NSI cognitive scale results have an unmatched baseline between placebo an active making a comparison invalid IMHO
4) Company promoted Mexican ALS site
5) Majority of patients in P1 trial did not benefit from treatment - Dr. Feldman claims it is too early to tell if treatment is effective yet company promotes otherwise.
6) CUR promotes anecdotal patient blogs for patients claiming positive results, but other patients claim to be worse off -- benefit is not verified yet.
7) CUR promotes right to try in Colorado, but will never treat a patient there. Stunt will backfire severely with the FDA
8) BCLI treatment has a statistically significant result in their 2a interim and full results are do shortly. The therapy does not require surgery or immunosuppression.
9) Large shelf registration was a large miscalculation.
10) STEM is the likely owner of the relevant patents, and has pursued a much more commercially viable clinical pathway with AMD.
11) Repeating STEMS's program in SCI is a waste of funds - success in this area has been fleeting
Conclusions: CUR is likely to be surpassed by BCLI an any effect appears to be neurotrophic rather than related to engraftment. NSI 189 data are inconclusive - no evidence it works. Company also has conspicuously back-tracked on MRI data. Management, time and again, misguides investors and unethically promotes trial results without fair balance IMHO. BCLI is now the company to watch in ALS. SELL.
Sovaldi sales should just exceed $4 billion, and if these two decisions are positive, look for $120 and a stock split after holding above $100 for 60 days.
STEM was upgraded today at Chardan Capital, and the firm issued a price target of $4.0. They have also indicated that STEM will release their AMD data on June 19 prior to the market opening. Depending on how large the efficacy signal is, this has the potential to be transformative for the company.
No oral presentation involved. Typical Neuralstem spin. According to the abstract for June 24 it does not include MRI data, and Garr, despite all his missed guidance on revealing the data, has not indicated that data will include MRI results. Perhaps it will be presented on the 17th - but again - the company does not confirm... Garr misleads - I would wait for actual results before speculating. MRI doesn't appear likely. Original guidance listed Fava as only a consultant, who post study became a co-PI. Is that ethical?
CFO noted on CC that VA/Military channel is not reporting in IMS RX and represents a very large percentage of the pie. Additionally, Europe is also not in this estimate. Revenue estimates based on scripts fall quite short of actuals. 2nd quarter likely $3.4 to $4.0 billion. The CFO comments have sparked the rally.
1) Fiorino introduced - will make the rounds with US bankers
2) Israeli press profiles a 2nd super responder - a soldier. Company considering translating for US news outlets
3) Slides will not be distributed for 2a results due to pending publication of data by PI. Waiting for final 2 patients to complete follow-up in August
4) Company preclinical models of NurOwn in AZ show consistent effects across the board -- nice pipeline expansion for attracting funding
5) Tech transfer completed at 2 additional manufacturing sites - looking to consolidate an approach
6) Publication of 2a results could qualify for FDA breakthrough status - publication to be in major journal
7) Trials moving forward and company believes their technology is better than competitors - working on improvements to share price
8) No discussion of up-listing or reverse stock split
9) No negative news (other than visibility issues being in Israel) - Fiorino thrown in the fire, but performed well