....regarding share price, we may see a run to $4.50-$4.75, following the earnings release/webcast. The the real fun will start as folks wait for the binary data....With positive data, the share price will likely soar -- as proof of concept is reached not just for UC, but with improved probability for all other pipeline programs. Market cap should rise $800 to $1 Billion, with a real chance at a buyout. We'll see....impossible to predict.
Most JV agreements call for a joint press release (simultaneous) of clinical trial results.
It's interesting that ATHX are releasing the data for 8-weeks separately (at a different time) from 16-weeks (when results will definitely be know at the time they release 8-week data). Why would they do this? The only conclusion I can logically derive is that they are looking to raise $$$ between the two release dates, and the data are positive.
If the UC data are negative for 8-weeks, why would they wait to deliver bad news again several weeks later (as the CEO has guided) for the 16-week endpoint (double tank for the stock)? If the data are negative for 8 and positive for 16, it wouldn't make sense to tank the shares, wait, and release + 16-week data (some type of delayed immune response). However, the data could theoretically be sig at 8, but not 16 (seems improbable)? Then they would retain the ability to raise funds. It must either be an 8-week +, and/or a +/- 16-week negative.
Incidentally, the trial is small (although complex and well-designed), and I'm sure the data have been QC'd along the way - enrollment took much longer than expected. The data base should be clean and ready to go, with endpoint analyses pre-programmed for the db. Gil has often indicated they'll have the data quickly. Well, March 19 is the 16-week point (not the 8) from the Dec 19 PR indicated full recruitment. Again, why the double results release - the analyses run for both once they unlock the blind, and trigger the stat run.
The only conclusion I can reach, is that the 8-week result must look very good. No mention of the 4-week endpoint. Or, maybe Gil is just too optimistic a cheerleader. We'll see.....
With the addition of international sites to the MulltiStem ischemic stroke program, the company is preparing not only to take advantage of new approval paths ex-US, but also with the FDA. Two sites are now active in the UK.
On the earnings call next week there should be significant developmental news including their AMI and GvHD trial initiation. It would appear likely that PFE would buy this company, or lock them up further developmentally if the UC data are positive. The potential markets are just too large.... And, the milestone would market the beginning of the regen medicine revolution. Analogous market development in cancer immunotherapy is soon to shift to regen medicine, as the cell science advances in a clinically meaningful way. Countries around the world are altering their regulatory paths and cell therapy guidelines to gain advantage.
Several payment milestones should also be announced regarding RAGE and the completion of the UC Phase 2. March 19th market the 16 week endpoint using the press release (Dec-19-14) for enrollment completion - as the marker. It is possible that ATHX and PFE will know the results by the time of the call.
Suspect we'll see a leg up into the earnings call, depending on the biomarket winds... Positive UC data next month/early May (8-week data first) and a new era may have arrived.
Easy answer. Most sites only have 1 or 2 patients. You would need to see the aggregate data for the full picture.There are currently 52 sites for this 140 patient study and the randomization, if I remember correctly is 3:1 Multistem to Placebo.
Actually not an error. The pathogenesis of AMI and GvHD are very different. Classically,GvHD is characterized by selective damage to the liver, skin (rash), mucosa, and the gastrointestinal tract - which was prevented, to the extent reported, by MAPCs. It is the latter which led investigators to hypothesize regarding the efficacy of MAPCs in ulcerative colitis - along with pre-clinical data. A positive result in GvHD is much closer mechanistically to predicting an effect in ulcerative colitis than a positive effect in MI. The company has made several references to the GVHD result an UC in their scientific presentations. We'll know soon if they are correct. Since no P1 study for UC was completed, it is hard to handicap the results -- this is best done by considering the P1 for GvHD along with some compelling preclinical data. A positive result to this binary event is a tide that will lift all boats in regenerative medicine. A potential major milestone and passage to a new era in regenerative medicine if positive. The stock will certainly move much higher than the board has been predicting -- consider the $40-60, in the near term, with a robust result.
There were two separate 100k share dumps today. Not a very sophisticated attempt at dropping the share price' and, poorly telegraphed last night following a sophomoric bear case publication. The bottom-line is that there is an upcoming binary event, and the only suggestive clinical data is from GvHD P1 results. Based on these P1 human results, and pre-clinical data, the odds appear to tip in favor of a positive outcome (at the high dose). It will be what it is...PFE does have 4-week data, as the collections are at 4,8.16 weeks. Close to March 17 should be the final data point for the last patient - perhaps a comment on the earnings webcast?
Here is exactly what you wrote: "Multistem ... but my reducing the inflammation and damage in the penumbra region (part of brain effected by ischemic stroke)." You indicated a direct mechanism. That's incorrect.
What has been shown preclinically (published) is that M2 macrophages (neuroprotective) increase following administration, and these are a result of increases in T-regulatory cell activity in the spleen and plasma. The company has stated their MAPCs do not pass the BBB.
Of course, if there is damage to the BBB, anything could pass, including a bullet. However, that's not the nature of ischemic stroke -- think TBI. MAPCs have been demonstrated to REDUCE BBB permeability (published) in TBI preclinical models, a good thing, and you are again incorrect.
You can confirm my statements with publications in the Journal of Neuroinflammation. If you take others to task for being imprecise, then you may be too!
Correction dr. sumitchawla. MAPC does not penetrate the BBB. It, hypothetically, has an indirect effect through anti-inflammatory activities which are spleen mediated.
Garr has missed so much guidance his positive comments have little impact. The delay in releasing topline 1B results and the abandoned partnership discussions/negotiations for NSI 189 have investors spooked.
He was definitely encouraging, but the vast majority of his previous encouragement has included missed projections. Particularly on NSI 189, where he is withholding topline results for the 1B. Perhaps he is waiting for the April Feldman update, and will do good news/bad news? It's hard to believe what he says, because he constantly revises guidance. We will just have to wait for actual results. He's claimed 3 principal investigators for the phase 1 NSI 189 program. Gersnick, Johe, and Fava -- which is the real PI. Fava was simply a paid consultant -- will he now be the new PI?
1. NSI 189 "early look very encouraging" - Garr. Proceeding with Phase 2. However, they are going to sit on data another 1-2 months. Again, another revision of guidance upon revision, upon revision.
2. Mass Gen completed their first surgery a week ago for ALS P2
3. Dr. Feldman to present interim P2 in April for ALS P2
4. All patient surgeries for ALS P2 to be complete by March according Garr
5. ALS full data in December.
No new safety signal -- those deaths were noted, I believe, and previously reported in the updated annual report at the 2.5 mg/kg dose; the investigators adjusted the dose down to 2.3 mg/kg. Reduction in PSAs and CTCs in progressing patients would be a positive. Imaging will be available for presentation. There is some preliminary data in the abstract, due to the timing of the trial's close. Kantoff and investigators have noted, what surprises them, is that patients in the P1 are still around...withholding full judgment on the P2 until the presentation. Regarding share price, EXEL is down quite a bit in recent days - lost over a $1, and PGNX was headed the other direction -- we'll see how PGNX does tomorrow. The interim COMET OS trial is unlikely to succeed, IMHO. Bottom-line, unimpressive abstract, but is that because the data were not in....or is there more to the story? I don't know. I do think the EXEL is going to have a tougher go than PGNX. EXEL has been mismanaged...
These patients are already extremely sick, and yes, there is definitely neutropenia with PMSA ADC. But in December 2013, Progenics EXTENDED the trial: "Progenics announced in December 2013 that it has completed enrollment of 83 chemotherapy experienced patients in this trial and has initiated treatment in an additional trial cohort of up to 35 chemotherapy naive patients who have progressed on hormonal therapies."
The DSMB and the investigator IRBs are all on board with this decision. Let's wait for the data; suspect we'll see some decent news.
Note that the PSA reduction was found to be dose dependent, and that P2 utilizes an optimized dose - although safety may be less optimized ;). The patients in P1 were very ill. Please note that they also found a reduction in CTCs and/or bone pain. Again, in the P1, this was found at the higher doses. We should see improved results for the optimized dose in P2.
Yes...talking about PGNX PMSA ADC. From ECCO P1 release:
Professor Daniel Petrylak, who was Professor of Medicine at Columbia University Medical Center (USA) when the phase I trial started and who is now Director of the Prostate Cancer Program/Genitourinary Cancer Program and co-director of the Signal Transduction Program at Yale University Medical Centre (USA), said: “By conjugating the antibody with a chemotherapeutic agent, we hoped that this would lead to more targeted therapy, which would have fewer toxic side-effects and would be more effective against the cancer.”
Prof Petrylak and his colleagues from other US cancer centres recruited 50 patients to the phase I clinical trial. The patients had the most advanced form of prostate cancer, which had spread to the bone and other organs; they had failed hormone therapy and had received up to two previous chemotherapies. The researchers treated them with doses of PSMA ADC at levels ranging from 0.4 to 2.8 mg/kg, by intravenous infusion, over a period of three weeks per cycle, and for up to four treatment cycles. The researchers detected anti-tumour activity among the patients who were treated at the higher doses. About half of the patients who received doses of 1.8 mg/kg or more showed either a 50% or more reduction in PSA levels, or a fall in CTC in the blood to less than five cells per 7.5 ml of blood, or both. The drug was generally well tolerated by patients, although levels of white blood cells were significantly reduced (neutropenia) at the highest dose of 2.8 mg/kg and one patient died. The researchers say the cause of the death is unclear. Prof Petrylak said: “These results show that PSMA ADC has anti-tumour activity in patients who have failed up to two prior chemotherapies and hormone therapy. We have initiated a phase II trial of up to 75 patients in which the recommended dose will be 2.5 mg/kg. This new trial will evaluate responses in PSA and CTC; it will evaluate control of metastases in bone, internal