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I actually sent Ritu an email to see if she'd respond. Doubt she's even capable of comprehending anything I have to say anyways.
Ritu is very bright, which doesn't mean she is always right. I don't think you should say she would wouldn't be capable of comprehending anything you have to say.
the bankers are salivating over the next financing
YM BIOSCIENCES INC. (TSX:YM; $2.90; AMEX:YMI; US$3.06) – Buy, Speculative Risk
Latest activity in the JAK inhibitor world.
We provide an update on recent developments with JAK inhibitors.
Highlights
· Incyte (Nasdaq:INCY; US$20.43; Not Rated) on track for ruxolitinib (INCB018424, a JAK1 and JAK2 inhibitor) NDA filing in June. Partner, Novartis (NYSE:NVS; US$59.57; Not Rated) plans to submit an MAA to EU regulators at the same time. Incyte hopes to obtain priority review, implying a 6-month review period in the U.S. The company is starting to prepare in the event the FDA requests an Advisory Committee meeting. Full details from two positive phase III studies (COMFORT-I and COMFORT-II) will be presented at ASCO, June 6th.
· Incyte retains its head-of-the-class status. In our view, Incyte is leading the way in setting the bar for what regulators are looking for to approve a JAK inhibitor for myelofibrosis. If ruxolitinib is approved then we expect YM and other developers of JAK inhibitors to have to show similar improvements in spleen size and resolution of constitutional symptoms.
· Onyx (Nasdaq:ONXS; US$36.95; Not Rated) hands back JAK2 inhibitors to S*Bio. S*Bio has regained North American and EU rights to SB1518 and SB1578 and will move the programs forward independently. There has been some criticism that the safety profile of SB1518 is not as attractive as others in the space. We see this news as negative for S*Bio. In our view, Onyx may not have been sufficiently satisfied with the profile of the drug to continue to fund its development.
· CYT387 update at ASCO June 3rd. We expect data from 60 patients treated for at least 3 months to be presented at ASCO. In addition to anemia data, we look for spleen size reductions, symptomatic improvements and updated safety data.
· Safety may be the differentiating factor between the JAK inhibitors. If YM is able to show a beneficial, long-lasting impact on anemia this could be an important differentiating factor.
· YM a long way behind Incyte. We estimate YM is unlikely to start phase III trials until at least mid-2012. The earliest we expect CYT387 to be marketed is 2015, which is at least 3 years behind ruxolitinib.
· CYT387 unpartnered, for now. If YM reports positive data at ASCO, which we expect, we believe management would prefer to structure a regional deal to provide some funding for phase III trials rather than complete a global licensing deal. YM may be able to obtain greater economics over the long-term by conducting its own phase III program, but with added risk. Investors waiting for a potential buyout of the company or a large global deal could be disappointed if these events do not transpire.
· Reiterate Buy rating. In our view, the latest developments in the JAK world are positive for YM. The forthcoming NDA from Incyte has been well-known. There has already been caution expressed around the S*Bio JAK2 inhibitor, so we do not see this as having a negative impact on YM. Conversely, it could be positive in that potential partners now know the level of the bar that is likely to be required, which has been set by Incyte, and they are not willing to compromise with a product that could potentially be inferior in terms of either safety or efficacy. We believe YM shares could rally at the ASCO conference where we expect considerable emphasis and media coverage of the JAK inhibitors as a result of company presentations
The full comment can be found at: www.bloomburton.com/research/YM 20110504.pdf
------------
Bloom Burton & Co. Inc.
65 Front Street East, Suite 300
Toronto, Ontario M5E 1B5
when you have nothing to sell buying back stock is not the answer
They are cutting cost to buy back stocks to excerise options.
In five years when there are no drugs they will be gone, but have made their money
It looks like Pfizer's got quite a future
http://pipeline.corante.com/archives/2011/04/28/pfizer_layoffs_today.php
In February 2009 fossil was selling at 11 dollars a share.
It is now trading at over 95 a share.
company makes almost 4 dollars a share. they make nice watches, nothing special, not technology dominance or i/p
how does the valuation make any sense in a normal market
http://finance.yahoo.com/q?s=FOSL
This market is headed for a huge fall
To prepare you for when you retire and take up texting:
ATD - At the Doctor's
BFF - Best Friends Funeral
BTW - Bring the Wheelchair
BYOT - Bring Your Own Teeth
CBM - Covered by Medicare
CUATSC - See You at the Senior Center
DWI - Driving While Incontinent
FWBB - Friend with Beta Blockers
FWIW - Forgot Where I Was
FYI - Found Your Insulin
GGPBL - Gotta Go, Pacemaker Battery Low
GHA - Got Heartburn Again
HGBM - Had Good Bowel Movement
IMHO - Is My Hearing-Aid On?
LMDO - Laughing My Dentures Out
LOL - Living on Lipitor
LWO - Lawrence Welk's On
OMMR - On My Massage Recliner
OMSG - Oh My! Sorry, Gas
ROFL...CGU - Rolling on the Floor Laughing...Can't get Up!
TTYL - Talk to You Louder
WAITT - Who Am I Talking To?
WTFA - Wet the Furniture Again
WTP - Where's the Prunes
WWNO - Walker Wheels Need Oil
GGLKI - Gotta Go, Laxative Kicking in!
Management is taking a hard line on spending and following some suggestions Barron's made at the end of last year.
It looks like Pfizer management is listening to Barron's suggestion, plus it looks like there was supposed to be something added to this sentence
It could also be two sentences, the beginning of one sentence and the end of another one.
Medgenics:
Just from that description I would think there are some major issues with regards to scale. That's a 2.5 mL "sliver" that is supposed to produce how many times it's weight in protein *and* guarantee secretion of said protein to the circulation so that it reaches a therapeutic level?
so far everyone that has been given the tissue back has produced the protein for a minimum of six months.
And practically, is this any better for the patient than IV infusion? Or is it just a "novel" way of delivery (scale issues aside)?
If you take interferon as an example you get the flu like symptoms because of the peak effect of the injection then at the end of the week you have a trough in the drug effect then it is time for a new injection.
This process should keep the drug effect level reducing the side effects.
http://www.medgenics.com/downloads/Presentation_Medgenics%20ASN%202010%20.pdf
So the bio-pump is actually protein-producing tissue harvested from the person's body?
The tissue is taken from the person's body, then the gene sequence is inserted into the tissue, cleaned and then sent back and reinserted into the person.
The tissue acts as a bio reactor.
this is old but it gives you the science
http://www.medgenics.com/downloads/Medgenics_EIO_10-16-06.pdf
Medgenics, A paradigm shift in protein manufacturing.
Medgenics Announces Exercise by Underwriters of Over-Allotment Option
MDGN 4.05 -0.02
Press Release Source: Medgenics On Tuesday April 19, 2011, 6:26 pm EDT
MISGAV, ISRAEL and VIENNA, VA--(Marketwire - 04/19/11) - Medgenics, Inc. (AMEX:MDGN - News) (AMEX:MDGN.W - News) (AIM: MEDG) (AIM: MEDU), the company that has developed a novel technology for the sustained manufacture and delivery of therapeutic proteins continuously in patients using their own tissue, today announced that, in connection with its previously announced initial public offering in the U.S., the underwriters have exercised their option to purchase additional redeemable Common Stock purchase warrants (the "Warrants") to purchase 369,000 shares of additional Common Stock, $0.0001 par value per share, at the public offering price of $0.46 per Warrant. The Warrants are exercisable at a price of $6.00 each and expire on April 12, 2016. Further details of the Warrants were announced in a press release on April 8, 2011.
With the exercise of this over-allotment option, the net proceeds to Medgenics from the initial public offering are approximately $10.2 million, after deducting underwriting discounts and commissions and offering expenses payable by the Company. The underwriters will continue to have an option, expiring May 21, 2011, to purchase 369,000 additional shares of Common Stock to cover over-allotments.
Following this exercise of the over-allotment option, Medgenics has sold 2,460,000 shares of Common Stock and 2,829,000 Warrants in its US initial public offering. The Common Stock and the Warrants are listed on NYSE Amex under the trading symbol "MDGN" and "MDGN.W," respectively. These Warrants will not be admitted to trading on the AIM market of the London Stock Exchange.
Roth Capital Partners acted as the sole book-running manager for the initial public offering and Maxim Group LLC acted as co-lead manager.
Medgenics intends to use the proceeds from its initial public offering for product development activities, including clinical trials of its product candidates EPODURE to treat anemia in chronic kidney disease, INFRADURE to treat hepatitis C and HEMODURE to treat hemophilia, for patent maintenance fees and intellectual property support and for working capital and other general corporate purposes, which may include the acquisition or licensing of complementary technologies, products or business.
A registration statement relating to the Common Stock and the Warrants was declared effective by the Securities and Exchange Commission on April 6, 2011. Electronic copies of the prospectus may be obtained, when available, at the Securities and Exchange Commission's website at http://www.sec.gov/ and on the Company's website (www.medgenics.com). Copies of the prospectus may also be obtained from Roth Capital Partners, LLC Equity Capital Markets, 24 Corporate Plaza, Newport Beach, CA 92660, at 800-678-9147 and Rothecm@roth.com.
This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.
About Medgenics
Notes to Editors:
Medgenics is a medical technology and therapeutics company focused on providing sustained protein therapies, having developed its unique tissue-based Biopump platform technology to provide sustained-action protein therapy for the treatment of a range of chronic diseases using the patient's own tissue. The Company has reported proof of concept of the Biopump approach in a Phase I/II clinical trial in its first clinical application, treatment of anemia due to renal failure, where a single administration of Biopumps producing erythropoietin has demonstrated sustained anemia treatment in 14 patients to date, most showing 6 months or more of safe and sustained treatment, and one more than 2 years. The first revenue generating commercial deal was negotiated in late 2009 for development of a Biopump producing clotting Factor VIII to treat hemophilia. The Company intends to seek additional deals with various strategic partners to further develop the Biopump platform technology for a range of clinical applications.
Biopumps are made using needle biopsies taken from the lower layer of the patient's skin under local anesthetic, and processed during 10-14 days to become 30 mm long tissue biofactories, or Biopumps, producing the required protein. The requisite number of Biopumps are injected 1-2mm under the patient's skin, where they heal in place and can provide sustained protein production and delivery for many months. The Company believes that delivery can be reversed or down-dosed by ablation or removal of one or all Biopumps. The Company is developing the Biopump to provide substantially greater safety and reliability in protein treatment in a more cost effective manner than experienced with the existing injected protein therapies. Medgenics currently has three products in development based on this technology, addressing the indications of:
Anemia - using EPODURE, a Biopump producing erythropoietin (EPO)
Hepatitis-C - using INFRADURE - a Biopump producing interferon-alpha (IFN-a)
Hemophilia - using HEMODURE - a Biopump producing clotting Factor VIII
The Company is completing its Phase I/II clinical trial using EPODURE to treat anemia in patients with chronic kidney disease. Designed to produce and deliver a therapeutic dose of EPO steadily for six months or more, EPODURE Biopumps have delivered EPO in all 14 patients treated to date, and provided effective anemia treatment for 6 months or more in most patients treated, with the longest continuing for 29 months or more. The Company is now preparing for a Phase I/II trial of INFRADURE for treatment of hepatitis C, where the treatment has potential to provide the benefits of interferon-alpha treatment, but with much reduced side effects caused by the high concentrations of bolus injections and their resulting side effects. The Company hopes to launch the first INFRADURE trial for hepatitis C during the coming year.
Medgenics intends to develop its innovative products and bring them to market via multiple strategic partnerships with major pharmaceutical and/or medical device companies. In addition to treatments for anemia, hepatitis C and hemophilia, Medgenics plans to develop and/or out-license a pipeline of future Biopump products targeting the large and rapidly growing global protein therapy market, which was estimated to have reached US $95 billion by the end of 2010. Other potential applications of Biopumps producing various proteins include multiple sclerosis, arthritis, pediatric growth hormone deficiency, obesity, and diabetes, as well as rare diseases.
Contact:
For further information, contact:Medgenics, Inc.Dr. Andrew L. Pearlman Phone: +972 4 902 8900
Teva Pharmaceutical (TEVA): Uh Oh - Laquinimod Competitor BG-12 Shows Competitive/Superior Data
Rating HOLD
Price Target $53.00
Price $49.17
Key Takeaway
Biogen announced more specific efficacy data for its Phase III oral MS drug BG-12 that appear superior to that for laquinimod, and this is dampening market enthusiasm for Teva's highest profile pipeline drug. The stock is down >6% today, which we think is probably an overreaction. That said, given overall market concerns on Copaxone and now potentially laquinimod, we remain at Hold.
A shot across the bow. During its 4Q earnings call this morning, Biogen (BIIB, $86.57, Buy) announced data for BG-12, its Ph.3 oral drug for MS, with efficacy data that on the surface appear superior to that for Teva's Ph.3 oral drug laquinimod. As a reminder, BIIB had press released last Monday (ironically, the same day that Teva announced the ALLEGRO Ph.3 data for laquinimod vs. placebo) that BG-12 had hit statistical significance in its DEFINE trial vs. placebo, but specific details around efficacy and safety were not included. The headline efficacy data for BG-12 from BIIB's call today were: ARR reduction vs. placebo of 53% (vs. 23% for laquinimod) and EDSS reduction vs. placebo of 38% (vs. 36% for laquinimod). On first blush, these efficacy data look strong and very competitive to Novartis' (NOVN.VX, CHF51.05, Buy) Gilenya, which is currently the only FDA approved oral pill for MS. Gilenya has similar efficacy data, but carries multiple warnings on its label (for potential cardiovascular, ocular, lung function, liver issues), and this has been viewed as the opportunity for laquinimod -- to come in as a safer oral option. Feedback we had gathered from neurologists while we were at last week's AAN meeting had suggested that despite lower ARR efficacy data for laquinimod, its safety profile along with its once daily oral dosing could make it a potential winner. In our view, today's BG-12 data now throws this in question, with BG-12 potentially stepping up ahead of laquinimod.
Several things to keep in mind. Whereas BG-12's efficacy data look stronger compared to laquinimod, we remind investors that it's always difficult to compare drugs across clinical trials without the benefit of having a true-head-to-head study comparing any two drugs, with differences on efficacy sometimes attributable to differences in clinical trial design and patient populations studied. We also note that there are several differentiating factors between laquinimod and BG-12. First, as it relates to dosing, laquinimod is given once daily, whereas BG-12 is given twice daily or three times daily. Feedback from physicians at AAN on dosing was somewhat mixed, but overall, it was generally viewed that once daily is always preferred. Also, on safety and tolerability, laquinimod seems very clean, suffering from just mild and transient elevations in liver enzymes, and some increases in back pain and abdominal pain. BG-12 shows flushing and GI side effects, and while this appears manageable, without more specific numbers and data, it's difficult for us to draw conclusions at this time. We'd expect to get a more definitive picture on the profiles of laquinimod and BG-12 later this year with data expected from the second of each's Ph.3 clinical trials -- BRAVO for laquinimod (vs. Biogen's Avonex) and CONFIRM for BG-12 (interestingly, vs. Teva's Copaxone).
What to do with the stock? For those bullish on Teva, laquinimod has been viewed as a potential multibillion dollar product that could be a potential successor to Copaxone, which generated >$3BN in sales in 2010 (~20% of Teva's total revenue but perhaps 35-40% of profits), but this new data for BG-12 could put a big dent into this. We'd agree that Teva's valuation at current levels is cheap (~9X PE versus ~12X PE for the peer group). However, in the bigger picture, given continued market uncertainty over the fate of Copaxone -- not only on the competitive front in the MS marketplace but also the risk of potential generics (especially given a September patent trial ) -- and now big questions on the competitiveness of laquinimod, we don't see a particularly strong reason to step up in a major way at these levels.
Corey Davis, Ph.D. *, Equity Analyst
(212) 336-7187 cdavis@jefferies.com
Graig Suvannavejh, Ph.D. *, Equity Associate
(212) 284-2170 gsuvannavejh@jefferies.com
Oren G. Livnat *, Equity Associate
(212) 284-2214 olivnat@jefferies.com
* Jefferies & Company, Inc.
If I had the opportunity to burn though 1.2 billion dollars, I could have probably found a drug that might have a chance to get approved also.
now that they cancelled their meetings they can get paid overtime to reschedule their meetings over the next 3months.
I agree with you. They could have gotten a lot in a sale before they bought the private company, now Mancuso does it again.
It keeps his salary rolling in.
CTIC wins appeal, Pixantrone approved, yee haw
April Fools
not really
I am not in the stock, if I had stayed in I would have made a lot on it. I do not understand how the stock has gotten this high because I do not think the sales of lymphoseek will be that high and Riggs is still not ready for prime time.
the new ceo I don't think is any better than bupp. He did nothing as ceo of boston life sciences before he changed its name, to what I don't remember
I am not sure what you are referring to, is it this
Abstract
Engraftment syndrome (ES) has been recognized as an inflammatory condition during neutrophil recovery after hematopoietic stem cell transplantation (HSCT) characterized by noninfectious fever and skin rash. It has been reported to occur frequently after autologous HSCT in children and adults, and has been shown to be an independent risk factor for increased transplant-related mortality (TRM). However, virtually no data exist on its occurrence after allogeneic HSCT in children. To determine incidence, predisposing factors for, and complications of ES in a pediatric transplant cohort, we analyzed 61 consecutive recipients of a myeloablative allogeneic HSCT for the occurrence of ES. Diagnosis of ES was established when children presented with =2 of the following symptoms within 7 days before engraftment: (1) fever >38.0°C, (2) skin rash, (3) weight gain and albumin drop, or (4) dyspnea, hypoxia, and pulmonary infiltrates. Incidence of ES in this cohort was 48% (29 of 61). In a univariate analysis, posttransplant granulocyte-colony stimulating factor (G-CSF) administration (P = .02), and high mononuclear cell count (MNC) (P = .002) were identified as significant risk factors predisposing for the development of ES. In a multiple logistic regression analysis, amphotericin B therapy (P = .009) and high MNC (P = .004) were significant explanatory variables for ES risk. There was a slight trend toward a higher rate of chronic GVHD (cGVHD) in patients with ES (P = .11). However, after a median follow-up of 9.5 years overall survival (OS) (P = .53) and TRM (P = .65) did not differ between the 2 groups. ES presenting with fever, rash, weight gain, and pulmonary symptoms should be recognized as a frequent complication of allogeneic HSCT after myeloablative conditioning in children. Treatment with G-CSF, amphotericin B, and a high nucleated cell count of the graft predisposed for the development of ES in this study. OS and TRM in this cohort were not affected by the occurrence of ES.
Still early data, but must have been good enough for the FDA/EMEA to grant a Ph2/Ph3 trial(not a pivotal trial or SPA though).
you are correct that it isn't an spa but you are incorrect that it is not a pivotal trial
call William Prather. If they meet the primary endpoint they can file, they do not need to run another phase 3.
post 116928
I always assumed Vanda sent someone at the fda a suitcase with cash to get their drug approved.
all it needed was some insider trading as an incentive
http://www.bloomberg.com/news/2011-03-29/sec-says-fda-chemist-reaped-3-6-million-from-insider-trading.html
Liang traded in smaller developmental drug companies, where a government decision would be more likely to have a significant impact on the stock price, the SEC said. He gained more than $1 million trading stock of Vanda Pharmaceuticals Inc. (VNDA) a Rockville, Maryland, firm that rose more than 600 percent a day after the FDA cleared sales of its schizophrenia drug Fanapt in May 2009, according to the lawsuit.
a year ago they had over 7 in cash, so they are trading under cash but the burn is high. this 7.2 million doesn't do much for the burn
I guess he doesn't have to mark to market.
he considers them long term holdings. Maybe he is grandfathered in somehow because his fund was started a long time ago.
is the better offer public and do you have a link to the ten million going to management
take a look at these reports and see if there is anyway a credible analyst would drop coverage
http://www.pluristem.com/national%20report10-1-11.pdf
http://www.pluristem.com/National%20February%203%202011.pdf
now they do not have a financing overhang. This will be one of the premier stem cell companies.
this is a good analysis. Someone must have written it for him.
http://www.cpreports.com/?p=917
they were human
I remember going to a genta presentation when I was just starting to invest in biotech. They had so many before and after melanomas disappearing I thought I was watching a majic show.
most analysts are interested in banking but I had to bring up this case because the termination of coverage came within two months of the analyst upgrade and it was so blatant
Biotechnology For full report in pdf, please click here
VIEW MODEL
sometimes an analyst picks up coverage then the trial fails or is delayed and they drop coverage. the only thing that has happened here is that Pluristem has raised 38 million dollars at 3.25 a share. There is no good excuse for this and any analyst worth his salt would not do this even if Roth told him to. He either lied when he raised the target or he is lying now.
Pluristem Therapeutics, Inc. | PSTI - $2.85 - NASDAQ | Buy
Estimates Changed
Target Price Changed
Stock Data
52 Week Low - High $0.94 - $2.97
Shares Out. (mil) 26.32
Mkt. Cap.(mil) $75.0
3-Mo. Avg. Vol. 223,321
12-Mo.Price Target $4.00
Cash (mil) $1.6
Tot. Debt (mil) NM
Est. 3Yr. EPS Growth NA
Cash (mil): Does not include $5.25 million raised in a private offering in October.
EPS ($)
Yr Jun 2009 --2010E-- --2011E--
Curr Curr Prev
1Q (0.29)A (0.11)A (0.08)A (0.08)A
2Q (0.09)A (0.10)A (0.08)E (0.10)E
3Q (0.17)A (0.13)A (0.10)E (0.11)E
4Q (0.12)A (0.10)A (0.10)E (0.11)E
YEAR (0.63)A (0.44)A (0.35)E (0.41)E
P/E NM NM NM NM
Revenue ($ millions)
Yr Jun 2009 --2010E-- --2011E--
Curr Curr
1Q -- -- --
2Q -- -- --
3Q -- -- --
4Q -- -- --
YEAR -- 0.0E 0.0E
PSTI: Raising Price Target After Discussions With Management
Reiterating BUY rating and raising price target to $4/share. We recently had an opportunity to sit down with PSTI management. Based on our conversation, we believe that we have a clearer picture of the company's prospects.
2010 was a good year for PSTI. The highlight of 2010 for Pluristem was its disclosure of results from its Phase 1 clinical studies (in Europe and the U.S.) of PLX-PAD (a stem cell treatment for critical limb ischemia, a severe manifestation of peripheral arterial disease that can result in limb amputation). In a total of 21 patients, PLX-PAD was found to be safe and well-tolerated. Importantly, 13 of 21 patients (62%) had improvements in blood flow and tissue oxygenation, and 17 of 21 patients (81%) had measured improvements in quality of life (as measured by the King's College Score for Quality of Life assessment). Additionally, 15 of 21 patients (71%) experienced a reduction in pain from baseline.
2011 could be a better year. We anticipate two significant events for PSTI in Q1. First, we expect that final data from PSTI's Phase 1 studies could be released by the end of Q1: based on positive preliminary results, we believe that this could further demonstrate the potential of PLX-PAD. Second, we expect to learn the result of meetings that PSTI held with the FDA and EMEA on the regulatory path for PLX-PAD. Specifically, there is the possibility that PSTI could move forward with a single pivotal Phase 2/3 clinical study of PLX-PAD: this could result in regulatory submission by the end of 2012, beating our expectations. Additionally, PSTI may initiate a clinical study of PLX-PAD, later in 2011, to treat intermittent claudication—this could increase the market potential of the treatment.
Raising price target to $4/share (from $2/share). Our new price target is based on estimated 2015 EPS of $0.41 (up from $0.16) subject to a 20X P/E (consistent with currently profitable biotech companies), discounted at 20% annually. Our increase in estimated EPS is based on greater confidence in PLX-PAD. See Valuation.
Refer to important disclosure information and rating System Definition at the end of this report. Regulation Analyst Certification ("Reg AC"): The research analyst primarily responsible for the content of this report certifies the following under Reg AC: I hereby certify that all views expressed in this report accurately reflect my personal views about the subject company or companies and its or their securities. I also certify that no part of my compensation was, is or will be, directly or indirectly, related to the specific recommendations or views expressed in this report.
VALUATION
We maintain our BUY rating on shares of PSTI, and raise our price target to $4/share (from $2/share). Our $4/share price target is based on application of a 20x P/E multiple to estimated 2015 EPS of $0.41, discounted at 20% annually.
===============================================================
Pluristem Therapeutics, Inc. | PSTI - $2.56 - NASDAQ | Non-Covered
Sus Andrew Vaino, Ph.D., avaino@roth.com
(949) 720-7102
Sales (800) 933-6830, Trading (800) 933-6820
suspending Coverage
Rating Changed
Stock Data
52 Week Low - High $0.94 - $4.38
Shares Out. (mil) 41.32
Mkt. Cap.(mil) $105.8
3-Mo. Avg. Vol. 908,845
12-Mo.Price Target NA
Cash (mil) NA
Tot. Debt (mil) NA
Est. 3Yr. EPS Growth NA
EPS ($)
Yr Jun 2009 --2010A-- --2011E--
Curr Curr
1Q (0.29)A (0.11)A (0.08)A
2Q (0.09)A (0.10)A NA
3Q (0.17)A (0.13)A NA
4Q (0.12)A (0.10)A NA
YEAR (0.63)A (0.44)A NA
P/E NM NM NA
Revenue ($ millions)
Yr Jun 2009 --2010A-- --2011E--
Curr Curr
1Q -- 0.0A 0.0A
2Q -- 0.0A NA
3Q -- 0.0A NA
4Q -- 0.0A NA
YEAR -- 0.0A NA
PSTI: Suspending Coverage
We are suspending coverage of Pluristem Therapeutics Inc. to focus on other companies in the Biotech space.
Prior to suspending coverage of PSTI, we had a BUY rating with a 12-month target price of $4.00/share.
Consistent with suspending coverage, prior estimates should not be relied upon.
He picked up coverage at the perfect time for Roth because as the post you responded to said they need cash
Vaino terminated coverage of pluristem today after doubling the price target in january to 4 dollars. They raised 38 million dollars which means they won't have a financial overhang for three years which is great. Problem is Roth can't do a financing for three years.
take back what you said about him being a good analyst.
genta has loads of pictures
Transcript of FDA Teleconference on Benlysta:
Capt Beth Fritsch:
Good afternoon and welcome to the health professional briefing on Benlysta. We have identified your organization as a
healthcare professional organization representing practitioners and professional leaders who may be interested in this topic.
We hope this briefing will provide you an opportunity to ask questions directly to FDA staff most familiar with this new
product. I am Beth Fritsch from the office of special health issues and I'll be your moderator for this teleconference.
Before we get started I would like to review how we will proceed. Today's discussion will focus on the primary efficacy and
safety findings for Benlysta which will be lead by Dr. Sarah Okada a clinical team leader from the division of pulmonary,
allergy and rheumatology drug products or DPARP. Immediately following the discussion, there will be a question and
answer session. At the conclusion of the question and answer session I will give concluding remarks. Your phones will be
on mute during the presentation. Prior to the start of the question and answer session the operator will provide instructions
on how to ask questions to FDA's experts. Please be aware this teleconference is being recorded for playback so please
state your name prior to joining the conversation. Speak clearly and directly into your telephone at all times.
Today I have several colleagues from the DPARP with me. As I mentioned Dr. Sarah Okada, the clinical team leader for
Benlysta will be providing you with an overview of the efficacy and safety findings. Dr. Badrul Chowdhury is also with us
today. He's the division director of DPARP. We also have Dr. Sally Seymore who is the deputy director for safety and
DPARP. Thank you for joining us today. At this time Dr. Okada will begin with her overview of Benlysta.
Dr. Okada:
Thank you Captain Fritsch. Good morning everyone. So as mentioned I'm going to just start off by giving a brief overview of
some of the background facts of Benlysta as we reviewed them.
As you know or as you probably know Benlysta or belimumuab is a human IgG1 lambda monoclonal antibody with a new
target, a molecule called a B lymphocyte stimulator, or BLYS, also known in the scientific literature as B cell activating factor
or BAFF. This molecule plays a role in B cell selection, survival and immunoglobulin class switching and by binding BLYS,
or BAFF, Benlysta may act by inhibiting survival of B cells including abnormal B cells and by reducing the differentiation of
those B cells into immunoglobulin producing cells.
The approved dose of Benlysta is 10 mg/kg given at 2 week intervals for the first 3 doses and at four week intervals
thereafter given by intravenous infusion over one hour. It's available as a lyophilized powder in vials for reconstitution with
sterile water for injection and further dilution in normal saline. Dextrose solutions are incompatible with Benlysta. The
limitations of use of this product pertain to primarily gaps in the data given to us by the clinical trials. The efficacy of Benlysta
has not been evaluated in patients with severe active lupus nephritis or severe active central nervous system lupus.
Benlysta has also not been studied in combination with other biologics such as rituximab or IV cyclophosphamide. So the
use of Benlysta is not recommended in these situations primarily just because we do not have the data to suggest that it's
safe and efficacious.
In terms of the basic clinical pharmacology and drug-drug interactions for the product, its pharmacokinetics are typical
monoclonal antibody pharmacokinetics. The terminal half like is approximately 19 days. Age, gender and race did not
appear to effect belimumab pharmacokinetics in the study population. However no formal drug-drug interaction studies have
been conducted with belimumab.
To turn to the efficacy of Benlysta. The initial efficacy was evaluated in a randomized controlled trial called LBSL02. This
involved 449 patients who received 1, 4 or 10 mg/kg of Benlysta plus standard of care and also contained a control group of
patients receiving placebo plus standard of care. The duration of that study was 52 weeks. Patients were required to have a
SELENA-SLEDAI score of greater than or equal to 4 at baseline and a history of autoantibodies, specifically ANA or
anti-dsDNA. However 28% of that study population was autoantibody negative at baseline. The co-primary endpoints for
that study were % change in SELENA-SLEDAI at week 24 and time to first flare over 52 weeks. No significant difference
between treatment groups were observed for any of the primary or secondary endpoints in that study. However exploratory
analysis done on the data from this study suggested that a treatment effect may have been present in the subgroup of
patients who were autoantibody positive and it also helped to inform the development of a new endpoint that the sponsor
proposed, an endpoint called the SLE responder index or SRI. The SRI is a composite endpoint where a patient is defined
as a responder if they have at least a four point reduction in SELENA-SLEDAI score compared to baseline and no
Page 3
worsening in physician global assessment and no new BILAG A or two new BILAG B organ domain scores at the time of the
primary endpoint assessment.
The sponsor conducted, subsequently, two pivotal trials, trials 1056 and 1057 with those learnings in mind. They enrolled
only SLE patients who were autoantibody positive at baseline and they used the proportion of SRI responders at week 52 as
the primary endpoint.
Trial 1056 enrolled 819 patients primarily in North America and Europe and had a longer control period of 76 weeks to
assess for possible delayed treatment effect in case no effect was seen at the week 52 timepoint. Trial 1057 enrolled 810
patients primarily in Asia and Latin America. The trials had predominately at baseline musculoskeletal and mucocutaneous
manifestations, approximately 82% from mucocutaneous, from musculoskeletal it was 73% in trial 1056 and 59% of patients
in 1057.
The more robust efficacy results were observed in trial 1057. A summary of the primary endpoint results at week 52 showed
that a higher proportion of Benlysta patients compared to placebo treated patients achieved a response as defined by the
SRI at week 52. For trial 1056 it was approximately 43% in the Benlysta 10 mg/kg group vs 34% of patients in the placebo
group. And in trial 1057 it was 58% of patients in the Benlysta 10mg/kg group compared to 44% of patients in the placebo
treated group.
So these two trials did provide independent substantiation of a difference in treatment effect with respect to the SRI and the
differences were statistically significant. Looking at the components of the SRI, the primary efficacy results appeared to be
primarily driven by the four point reduction in the SELENA-SLEDAI. However the no worsening in the physician global
assessment and the no new BILAG A or two new BILAG B domain scores were also consistent.
As you know there were a couple of concerns that we raised at the advisory committee in November of 2010. One of these
concerns was a possible lack of effect or reversal of treatment effect in patients of African American or African heritage.
Now this is a post-hoc subgroup analysis so we interpret the results with caution however in both studies the small subgroup
of patients that were in this racial subgroup experienced a reversal of effect. In trial 1056, which had the most African
heritage patients, 39% of patients in the placebo group responded vs 33% in the Benlysta 10 mg/kg group. The African
heritage subgroup in trial 1057 was even smaller, however similarly the placebo patients had a higher rate of response: 64%
vs. 46% for Benlysta 10 mg/kg. Again I caution that none of these results are intended to connote a statistical significance.
Another concern was the potential lack of efficacy at week 76 demonstrated in study 1056. At week 76 approximately 32%
of placebo treated patients had an SRI response vs. 39% of Benlysta 10 mg/kg treated patients. When comparing these
results to the week 52 results what's apparent is that the rate of response, or the loss of response, was approximately
similar across the treatment groups so it was concerning that there weren't fewer patients who were losing their response in
the Benlysta group. However again these results are primarily descriptive and not intended to connote statistical
significance.
Finally the last two efficacy issues I wanted to discuss are the clinically important aspects of prednisone reduction and a
reduction in incidence of flares. In trials 1056 and 1057 there was an endpoint of evaluating the proportion of patients with
prednisone reduction of at least 25% from baseline to less than 7.5 mg/day. This endpoint was accepted as a clinically
meaningful reduction in prednisone and the assessment compared baseline prednisone use to the last 12 weeks of the
study prior to the primary endpoint assessment. In trial 1056, well, I would just say in both trials there was a suggestion of
prednisone reduction. 13% of placebo patients achieved this level of reduction vs. 17-19% of the belimumab treated patients
in both studies. Looking at the flip side of that coin we also evaluated patients who required an increase in prednisone of
that amount and the two studies were not consistent in that regard with more Benlysta treated patients requiring an increase
in prednisone in study 1056 vs. trial 1057 where Benlysta treated patients actually did require less incremental increase in
prednisone.
Finally for the flare results, again there was a trend although not statistically significant of a higher proportion of placebo
treated patients having a severe flare during the course of the study vs the Benlysta treated groups. In trial 1056 that was
24% of placebo treated patients who experienced severe flare vs. 18% of the Benlysta 10 mg/kg group. And in trial 1057
that was 23% of placebo treated patients vs. 14% of the belimumab 10 mg/kg group.
I'm going to turn now to a brief discussion of the safety issues. For most safety issues there was a numeric imbalance going
against Benlysta in the pivotal trials, actually in both trials 1056, 1057 and trial LBSL02. These safety issues were placed in
Page 4
the warning section of the approved product label and I'm just going to describe them briefly.
First is mortality. Out of 2133 patients in the 3 clinical trials, 14 deaths occurred in the controlled period, 11 of those deaths
were in the Benlysta groups and 3 of those were in the placebo group. Etiologies included infection, cardiovascular disease
and suicide. For serious infections similarly, serious infections occurred in approximately 6% of the Benlysta groups vs 5.2%
of placebo treated patients. The most frequent serious infections included pneumonia, urinary tract infection, cellulitis and
bronchitis. Infections resulting in death occurred in 0.3% of Benlysta patients vs. 0.1% of placebo patients.
For hypersensitivity reactions including anaphylaxis, again there was a numeric imbalance going against Benlysta with 13%
of Benlysta treated patients experiencing a hypersensitivity reaction on the day of infusion vs. 11% of patients receiving
placebo. Anaphylaxis was observed in 0.6% of patients receiving Benlysta and 0.4% of patients receiving placebo.
Approximately 13% of study patients received premedication. With respect to infusion reactions, again an imbalance with a
respect to 17% of patients on Benlysta vs 15% of patients on placebo.
Finally for depression and suicidality, I'd like to note that psychiatric events overall occurred in a higher proportion in
Benlysta treated patients.16% vs. 12% of placebo treated patients. And this inbalance was related primarily to depression
related events. Serious psychiatric events were reported in 0.8% of patients on Benlysta vs. 0.4% of patients on placebo.
Serious depression was reported in 0.4% of patients receiving Benlysta and 0.1% of patients receiving placebo. Two
suicides were reported for patients receiving benlysta in the control period vs. none in the placebo groups.
Finally a brief summary of the post marketing requirements that have been enacted. The first post marketing requirement is
a randomized placebo controlled clinical trial in 5,000 patients which is intended to assess the long term safety profile and
adverse events of special interest including mortality, malignancy, serious and opportunistic infections and depression and
suicidality. Second is a pregnancy registry. Third is a trial to assess the effects of Benlysta on vaccinations. Fourth is a
prerequired pediatric SLE study. And fifth is a product requirement to improve immunogenicity assays.
The sponsor has also agreed to conduct several post-marketing commitments that we felt were important because of the
data gaps in the original data of the submission. These PMCs include one randomized controlled trial in patients with lupus
nephritis. A randomized controlled trial in African-American patients with SLE. And final reports from the long term open
label continuation trials that are ongoing. The details of these specific clinical trials have not yet been submitted so we have
not provided any feedback to the sponsor and we can't really comment on the details for those trials but the specific
submission dates were listed in the approval letter. That's all I have for the briefing summary. I guess we can open it up for
questions now.
Q&A:
Captain Fritsch:
I'm going to ask a question in the mean time until the questions start queueing up. It sounds like one of the trials was
conducted in Asia and Latin America so I'm wondering if you saw any other demographic differences in those populations.
Okada:
You mean with respect to proportion of Caucasian?
Fritsch:
I guess in terms of the African Americans. What you had talked about in the African American population, the drug may or
may not work as well in that population
I'm wondering if you noticed that within any of the other populations Asian of Latin American.
Okada:
That's a good question. There were not consistent trends with respect to the other racial subgroups. The real reason why we
became additionally concerned with the African American subgroup was that both trial subgroups appeared to have a
similar effect. With respect to Asians for example we have data on the Alaskan Native or American Indian subgroup for
example. In trial 1056 this subgroup actually followed a similar pattern to the African subgroup in the sense that the placebo
treated patients had a higher response than the Benlysta 10 mg/kg group did, however these results were not corroborated
in trial 1057. Again it may be an artificial grouping of folks but we didn't necessarily feel like that rose to the level of the
subgroup results are different.
Page 5
James Powell:
My question is, with respect to the African Americans in the clinical trial, you mentioned lower numbers, can you tell me
about those lower numbers and also related to that related to the safety profile can you tell me whether or not there was a
differential effect in African Americans with respect to safety observations?
Okada:
So the exact numbers that I have, for trial 1056, there were a total of 39 African Americans or African heritage patients in the
placebo group, 15 of whom responded. 40 patients in the 1 mg/kg group, 12 of whom responded and 39 in the 10mg/kg
group, 13 of whom responded. In trial 1057 that number is a lot smaller, there were 11 patients in the placebo group, 7 of
whom responded, there were 8 patients in the 1 mg/kg group 3 of whom responded and 11 patients in the 10 mg/kg group,
5 of whom responded. So we're talking about relatively small numbers.
James Powell:
And with respect to safety was there a differential effect?
Okada:
With respect to safety there didn't appear to be a differential effect. That went a long way to ultimately not being stronger in
the label against use in that subgroup. We're a little concerned about being too definitive based on subgroup results but we
didn't have any corroborating safety concern.
James Powell:
If it's possible for one more question can you tell me if there's any studies with respect to the particular antigen in African
American vs other patients that was used to create the antibody.
Okada:
That's a really good question. I don't think most of the studies looking at the serum levels of BLYS of BAFF, I don't think
there was any attempt to do racial subgrouping in any of those studies so I don't have that data.
Fritsch:
One of the post marketing commitments had to do with the use of Benlysta on vaccines, I'm wondering if for example, could
a person taking Benlysta also get a seasonal influenza vaccine this year, would that be contraindicated or allowed.
Okada:
The main contraindication with respect to vaccines is to not use Benlysta with live vaccines, so the flu shot should be just
fine. What we can't really comment on is does Benlysta impair vaccine responses. That's why we're asking for a post
marketing study to assess that. In theory the Human Genome Sciences suggests that there may be less of an impact on
vaccine responses because they're targeting a very specific subgroup of B cells so there shouldn't be an impaired response
but we'll have to see when the data actually come in.
Ann Santa-Donato:
Thank you. I'm from the association of Women's Health Obstetrics and Neonatal nurses and my question is have any
studies been done, animal studies been done on the efficacy of this drug for use in pregnancy.
Okada:
Benlysta is pregnancy category C. I'm trying to pull up the pregnancy section of the label real quick. Either way we wouldn't
really be able to comment on the efficacy. Animal studies don't always reflect human safety in this respect so we're a little
leery even if we have some the animal data to make the conclusion that it could be safe to use in pregnancy. I should
mention that there were approximately 47 pregnancies in the clinical trials occurring in lupus patients. They were told to use
a double barrier method of contraception but we know how things happen. And in those pregnancies there wasn't a specific
pattern that we could see in terms of potential problems however there was a scattering of spontaneous pregnancy loss. At
the moment we can't really say that it is safe so that's why we encourage anyone that gets pregnant while they're taking
Benlysta to register with the Human Genome Sciences Benlysta pregnancy registry.
Ann Santa-Donato:
So is it the recommendation that if a women becomes pregnant while taking the pregnancy should she discontinue or
consult with the healthcare provider about risk vs. benefit.
Page 6
Okada:
This is an infused medication so the healthcare provider is going to be involved in that decision at each visit but she should
consult with her healthcare provider. A lot of times it does come down to individual response and a willingness to accept the
risks
Lenore Coleman:
Yes this is Dr. Coleman. I have, I guess, a question and then a comment. Since based on the data it appears as though
African Americans are at a 3 fold increased risk of this in terms of higher incidence yet the drug does not appear based
upon the data that you reported, to not only not be efficacious but there is no way for us to be even assured of its safety.
What steps are being taken in terms of the promotion of this drug to physicians nationally regarding to lack of African
American patients that participated in the study and your concerns. My second is a comment. I think that this really points to
the fact that even though your recommendations that suggest that in chronic diseases where there is a preponderance of
patients in a particular ethnic group that has this disease, manufacturers are still not recruiting a proportion of minority
patients enough to assure not only efficacy but safety for use of products in the United States.
Okada:
With respect to the marketing, I can say that we do review the marketing materials. Our division of drug advertising and
marketing does that in conjunction with the review divisions and obviously any attempt to target that population would be
frowned upon by us and likely stopped. However I'm not aware and I sort of doubt that the company is going to try to market
to that subgroup given that we've already included in the label the potential reversal of treatment effect in that subgroup.
With respect to your comments I agree with you. I'm not quite sure why we didn't get more African American patients in trial
1056. It's a little baffling to us, it might be that because African American patients predominantly do experience renal
disease and patients with active renal disease were purposefully excluded from that trial. I think we're trying to address is by
the post-marketing commitment that the company has agreed to conduct a trial in African American patients. I'd like to also
recognize Dr. Rosemary Needer who is the primary medical officer involved in the application. Did you have an additional
comment?
Needer:
No I just want to say in defense of the sponsor, they did try to recruit as many African American patients as Dr. Okada did
allude to; the trial entry criteria prohibited entry of people who had major organ involvement such as lupus nephritis and
cerebritis
As you know African American patients tend to have a worse disease and that therefore a large number of them were not
eligible to participate in the clinical trial as they were designed.
Fritsch:
Thank you. This concludes the question and answer session of today's briefing. Just a reminder a replay of this
teleconference will be available at 866-513-4388 one hour after the call until April 14. No passcode is required. I want to
thank all of the attendees for their participation on such short notice. I hope this exchange was informative and has taken us
one step closer to providing timely information for you to inform y
SCOLR Pharma, Inc. Receives Deficiency Letter from FDA on Pseudoephedrine Application
I am pretty sure this company filed tha anda over two years ago. Someone must have found the study conduct under a refrigerator at the fda that was moved, because something spilled on the floor and they wanted to clean up under the fridge.
SCOLR PHARMA INC Related Quotes
Symbol Price Change
SCLR.OB 0.17 0.00
Press Release Source: SCOLR Pharma, Inc. On Friday March 11, 2011, 5:14 pm EST
BOTHELL, Wash., March 11, 2011 /PRNewswire/ -- SCOLR Pharma, Inc. (OTC Bulletin Board:SCLR.ob - News) today announced it has received a letter from the Food and Drug Administration (FDA) identifying deficiencies regarding its Abbreviated New Drug Application (ANDA) for extended-release pseudoephedrine.
The Company said the letter identified a number of concerns that the FDA had with the conduct of the bioequivalence study submitted by SCOLR to support the application. The deficiencies cited relate to the design and conduct of the study. None of the issues raised relate to the product formulation. Due to these deficiencies, the agency said it is unable to approve the ANDA application at this time.
Stephen J. Turner, SCOLR Pharma's President and CEO, said: "Although we are extremely disappointed with this situation, we are currently evaluating the deficiencies identified by the FDA to determine the most appropriate course of action. We remain confident in our pseudoephedrine product and intend to rectify the FDA's concerns."
About SCOLR Pharma:
Based in Bothell, Washington, SCOLR Pharma, Inc. is a specialty pharmaceutical company focused on applying its formulation expertise and patented CDT platforms to develop novel prescription pharmaceutical, over-the-counter (OTC), and nutritional products. Our CDT drug delivery platforms are based on multiple issued and pending patents and other intellectual property for the programmed release or enhanced performance of active pharmaceutical ingredients and nutritional products. For more information on SCOLR Pharma, please call 425-368-1050 or visit http://www.scolr.com/.
This press release contains forward-looking statements (statements which are not historical facts) within the meaning of the Private Securities Litigation Reform Act of 1995, including statements concerning next steps and intended actions with respect to our pseudoephedrine product. These forward-looking statements involve risks and uncertainties, including activities, events or developments that we expect, believe or anticipate will or may occur in the future. A number of factors could cause actual results to differ from those indicated in the forward-looking statements, including our ability to fund any additional or revised trial studies required to address the FDA's deficiency notice, and the time required for any additional or revised studies. Additional assumptions, risks and uncertainties are described in detail in our registration statements, reports and other filings with the Securities and Exchange Commission, including the "Risk Factors" set forth in our Annual Report on Form 10-K, as supplemented by our quarterly reports on Form 10-Q. Such filings are available on our website or at www.sec.gov. You are cautioned that such statements are not guarantees of future performance and that actual result or developments may differ materially from those set forth in the forward-looking statements. We undertake no obligation to publicly update or revise forward-looking statements to reflect subsequent events or circumstance.
Contacts:
Investor Relations:
SCOLR Pharma, Inc.
425.368.1050
I heard it from a board member. was before they were in the financial shape that ended to their downfall
I never said the bondholder weren't entitled. It should have never gotten that far
BUY OR SELL-Placenta vs bone marrow in battle to save limbs
http://www.forexyard.com/en/news/BUY-OR-SELL-Placenta-vs-bone-marrow-in-battle-to-save-limbs-2011-03-10T181915Z
Thursday March 10, 2011 03:19:18 PM GMT
BUYSELL/STEMCELL-CLI (RPT)* CLI results in 160,000 U.S. amputations a year
* Potential U.S. market worth over $2 bln
* Different approaches, different therapies
By Anand Basu and Shravya Jain
BANGALORE, March 10 (Reuters) - Two stem cell research firms are going head-to-head in the race to develop a first treatment for critical limb ischemia (CLI), where severely blocked arteries cut the flow of blood to hands and legs.
CLI afflicts about 1 million Americans and results in 160,000 amputations each year.
The battle pits Aastrom Biosciences against Pluristem Therapeutics , and focuses on two different approaches to find a treatment in a market potentially worth more than $2 billion a year, according to Vical .
There are currently no drugs approved by the U.S. Food and Drug Administration (FDA) for CLI, and companies including Vical and Sanofi-Aventis have seen experimental medicines fail late-stage trials.
Ann Arbor, Michigan-based Aastrom has been the leading candidate to produce a breakthrough, but is struggling with a late-stage trial design and doubts over the efficacy of its approach.
The autologous therapy it favours processes stem cells from a patient's bone marrow and injects them into the affected limb.
Israel-based Pluristem, instead, processes stem cells from donors' placenta -- an allogeneic therapy better suited for treating CLI, according to several researchers and analysts.
It plans to start a mid-stage trial this year.
"I believe the Pluristem approach is much better than Aastrom's," said National Securities Analyst Jason Kolbert.
"There's a wealth of anecdotal information that suggests young (placental) cells are more potent and vibrant than autologous cells from older patients with co-morbidities."
Some researchers favour using younger cells from placenta, as cells from bone marrow are less potent and can lose their ability to form new blood vessels with ageing.
"Stem cells derived from bone marrow will not be young, as the average age of CLI patients is around 65-70," said Dr. Amish Raval, Assistant Professor at the University of Wisconsin School of Medicine and Public Health. He is principal investigator in clinical trials at the university testing autologous cells.
Aastrom CEO Tim Mayleben, however, says that although the cells are taken from elderly patients, they are processed for 12 days and produce a potent product, proven in clinical studies.
"I don't think there's any data in the scientific literature to suggest patient's bone marrow is older and thus not as potent, but certainly it's talked about in scientific circles," he told Reuters.
IN THE BANK
Also, the processed cells produced by Pluristem's approach are off-the-shelf and ready-to use, while it can take two weeks to process stem cells taken from a patient's bone marrow.
"Unlike autologous cells, you can sort and bank allogeneic cells," said Michael Murphy, clinical director of the stem cell center at Indiana University School Of Medicine.
Cells are also more easily available using donated placentas rather than bone marrow.
"Allogeneic cells offer more promise for utility in clinical application as they can be collected, expanded, banked and are immediately ready for administration, which is critical for CLI treatment," said Murphy at Indiana University.
Murphy, who has previously worked for Aastrom, is principal investigator of a late-stage trial using autologous therapy for CLI, sponsored by privately owned U.S. orthopaedics group Biomet Biologics, and an early-stage trial using allogeneic therapy, sponsored by Medistem .
There are benefits from the autologous approach, however, with a very low risk of immune rejection since cells come from the patient.
"Autologous will have better safety data and is likely to have less long-term clinical monitoring and low or zero worry about tumours and any immune rejection," said Scimitar Equity analyst Henry McCusker.
Aastrom's Mayleben agrees.
"Cells derived from bone marrow are incredibly safe and very efficacious. We've treated 400 patients with our autologous cell therapies and we have never experienced any immune rejection," he said.
"I think it's a little early to compare and contrast different approaches. At the end of the day, what really matter is, does it work?" (Reporting by Anand Basu and Shravya Jain in Bangalore, Editing by Ian Geoghegan)
--------------------------------------------------------------------------------
The bond holders will own the drug and make a deal to partner the drugs.
The management totally screwed this up. I have been told that they were dealing with a large pharma and the ARYX was acting like they were in control and were disrepectful to the pharma guys.
the pharma guys told them to stick it.
A Tale of Two Stem Cell Platforms
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Written by Ray Dirks
Tuesday, 08 March 2011 03:31
0diggsdigg
I recently attended an investor presentation by Mesoblast. It is a fine company and their technology has been validated by a 130 million dollar upfront payment from Cephalon and an additional equity investment by Cephalon of about 107 million dollars. The deal also involves a possible 1.5 billion dollars in clinical milestones for three indications that Cephalon has licensed and Cephalon will be paying all of the clinical trial costs in these indications.
After the presentation I am more convinced than ever that Pluristem Therapeutics (Nasdaq: PSTI) is tremendously undervalued. Mesoblast's market capitalization is over 1.8 billion dollars compared to Pluristem's valuation of about 100 million dollars.
PLURISTEM THERA
2.29 - (0.00%)
Intraday | 3 Month | 6 Month | 1 Year
Quotes delayed at least 20 mins.
The last time I wrote about Pluristem they were a company that had just gotten approval to run a pivotal trial in the indication of Critical Limb Ischemia by both the FDA and EMA. They had one problem, they only had a few million dollars in the bank. Since that time the company has raised over 40 million dollars from warrant exercises and an equity raise, priced at $3.25. Based on their financial position the company is a better value today than it was back in February. They are one of the few biotech companies in the enviable position of having 3 to 4 years of cash.
Mesoblast's technology platform is all about adult mesenchymal precursor cells. About one out of every 100,000 cells removed from a healthy volunteer's bone marrow, not a pleasant experience, will be the cell used in their studies and commercialization process. Pluristem gets their mesenchymol like stromal cells from the placenta which is considered a medical waste and very easy to get access to.
Both companies claim that their cells are extremely potent with no immune reactions. The cells also avoid the ethical and safety issues of embryonic stem cells, and are backed by strong intellectual property protection. Both companies have off the shelf products, claim batch to batch consistency and say their cells can be expanded in large numbers without affecting potency. These are terrific assets because this means a low cost of goods with no supply constraints on approval making both technologies perfect for large pharma because they will have drug like properties and high margin products. The mechanism of action of each of the company’s products is immune modulation as opposed to most stem cells that work by cell differentiation. In fact in animal models within sixty days of delivery of cells you can no longer find the cells in the tissue where the cells were delivered. The companies haven't done this in humans because you can't take biopsies of these areas in humans.
As I said earlier both companies say their drugs are non-immunogenic. As far as I am concerned Pluristem has done more to prove this. Mesoblast has never given more than one injection to any one individual. In Pluristem's trial in Critical Limb Ischemia they gave repeat doses from the same placenta two weeks after the initial dose to many of their patients in an effort to exhibit that their cells do not have an immunogenicity issue. So far so good. There were no problems. In fact in their upcoming phase three trial a dose four months after the first injection is part of the protocol and it is believed this should help the efficacy of the stem cell treatment to increase the length of treatment effect.
Mesoblast said in their presentation that if their drug were to receive approval their contract manufacturer, would have to build a bricks and mortar facility in a tax advantaged area like Singapore to produce their product in a commercial scale. This is a concern to me because even if their pivotal trial is acceptable this could be an issue with the FDA. Many drug approvals are held up because of issues with the CMC section of the drug approval process. In this case the FDA might have an issue with the fact that the drug produced for the phase 3 study would be produced at a different manufacturing facility with a different cell bank, than what will be used in the manufacturing of the commercial facility. Pluristem in contrast is doing a very intelligent thing. They are manufacturing their clinical trial batches in the same facility and bioreactor that they will be using for the commercial launch of the drug, if they have a successful clinical trial. This is an incredibly important fact that I do not think most people following the companies have realized.
If you put these things together I think Pluristem has some advantages to Mesoblast but even if you say the products are the same or the differences are immaterial the Pluristem market value comparison to that of Mesoblast just doesn't make any sense and we should see a rise in Pluristem's valuation to fill that gap. I also have to believe that because of the simplicity and intellectual property around Pluristem's manufacturing it will not be long before "big pharma" takes a serious look at Pluristem and one of those companies commits to a similar licensing deal with Pluristem, that Cephalon offered to, and was accepted by Mesoblast.
MSB’s market value is currently approximately $1.8 Billion while PSTI’s market value is approximately $100-120 Million. As PSTI enters clinical trials for the other indications with their proprietary placental-expanded (PLX) cells, and for the reasons I point out in this report, their market value should reasonably approach the market value of MSB. Ergo, my opinion to aggressively accumulate PSTI’s Stock.
A Tale of Two Stem Cell Platforms
| Print | E-mail
Written by Ray Dirks
Tuesday, 08 March 2011 03:31
I recently attended an investor presentation by Mesoblast. It is a fine company and their technology has been validated by a 130 million dollar upfront payment from Cephalon and an additional equity investment by Cephalon of about 107 million dollars. The deal also involves a possible 1.5 billion dollars in clinical milestones for three indications that Cephalon has licensed and Cephalon will be paying all of the clinical trial costs in these indications.
After the presentation I am more convinced than ever that Pluristem Therapeutics (Nasdaq: PSTI) is tremendously undervalued. Mesoblast's market capitalization is over 1.8 billion dollars compared to Pluristem's valuation of about 100 million dollars.
PLURISTEM THERA
2.29 - (0.00%)
Intraday | 3 Month | 6 Month | 1 Year
Quotes delayed at least 20 mins.
The last time I wrote about Pluristem they were a company that had just gotten approval to run a pivotal trial in the indication of Critical Limb Ischemia by both the FDA and EMA. They had one problem, they only had a few million dollars in the bank. Since that time the company has raised over 40 million dollars from warrant exercises and an equity raise, priced at $3.25. Based on their financial position the company is a better value today than it was back in February. They are one of the few biotech companies in the enviable position of having 3 to 4 years of cash.
Mesoblast's technology platform is all about adult mesenchymal precursor cells. About one out of every 100,000 cells removed from a healthy volunteer's bone marrow, not a pleasant experience, will be the cell used in their studies and commercialization process. Pluristem gets their mesenchymol like stromal cells from the placenta which is considered a medical waste and very easy to get access to.
Both companies claim that their cells are extremely potent with no immune reactions. The cells also avoid the ethical and safety issues of embryonic stem cells, and are backed by strong intellectual property protection. Both companies have off the shelf products, claim batch to batch consistency and say their cells can be expanded in large numbers without affecting potency. These are terrific assets because this means a low cost of goods with no supply constraints on approval making both technologies perfect for large pharma because they will have drug like properties and high margin products. The mechanism of action of each of the company’s products is immune modulation as opposed to most stem cells that work by cell differentiation. In fact in animal models within sixty days of delivery of cells you can no longer find the cells in the tissue where the cells were delivered. The companies haven't done this in humans because you can't take biopsies of these areas in humans.
As I said earlier both companies say their drugs are non-immunogenic. As far as I am concerned Pluristem has done more to prove this. Mesoblast has never given more than one injection to any one individual. In Pluristem's trial in Critical Limb Ischemia they gave repeat doses from the same placenta two weeks after the initial dose to many of their patients in an effort to exhibit that their cells do not have an immunogenicity issue. So far so good. There were no problems. In fact in their upcoming phase three trial a dose four months after the first injection is part of the protocol and it is believed this should help the efficacy of the stem cell treatment to increase the length of treatment effect.
Mesoblast said in their presentation that if their drug were to receive approval their contract manufacturer, would have to build a bricks and mortar facility in a tax advantaged area like Singapore to produce their product in a commercial scale. This is a concern to me because even if their pivotal trial is acceptable this could be an issue with the FDA. Many drug approvals are held up because of issues with the CMC section of the drug approval process. In this case the FDA might have an issue with the fact that the drug produced for the phase 3 study would be produced at a different manufacturing facility with a different cell bank, than what will be used in the manufacturing of the commercial facility. Pluristem in contrast is doing a very intelligent thing. They are manufacturing their clinical trial batches in the same facility and bioreactor that they will be using for the commercial launch of the drug, if they have a successful clinical trial. This is an incredibly important fact that I do not think most people following the companies have realized.
If you put these things together I think Pluristem has some advantages to Mesoblast but even if you say the products are the same or the differences are immaterial the Pluristem market value comparison to that of Mesoblast just doesn't make any sense and we should see a rise in Pluristem's valuation to fill that gap. I also have to believe that because of the simplicity and intellectual property around Pluristem's manufacturing it will not be long before "big pharma" takes a serious look at Pluristem and one of those companies commits to a similar licensing deal with Pluristem, that Cephalon offered to, and was accepted by Mesoblast.
MSB’s market value is currently approximately $1.8 Billion while PSTI’s market value is approximately $100-120 Million. As PSTI enters clinical trials for the other indications with their proprietary placental-expanded (PLX) cells, and for the reasons I point out in this report, their market value should reasonably approach the market value of MSB. Ergo, my opinion to aggressively accumulate PSTI’s Stock.
From Barclays, We are reiterating our 1-Overweight rating on REGN following publication of a retrospective review of Lucentis vs Avastin safety in AMD and ahead of prospective data expected from the CATT study in 1H11. With some concern regarding implications of upcoming CATT data for the brand anti-VEGF market results of the retrospective analysis suggest potential incremental cardiovascular risk with Avastin. Results are consistent with prior Medicare claims data as well as feedback from retinal experts and if replicated in CATT could result in actual brand expansion in AMD where we expect REGNs VEGF-TRAP eye (VTE) to dominate.
The journal Ophthalmologica published online a retrospective analysis of arterial thromboembolic events (ATEs) in patients treated for AMD with Avastin vs Lucentis. The study by Carneiro et al involved a chart review of 378 consecutive patients treated with anti-VEGF monotherapy between December 2006 and January 2010 at Hospital Sao Joao in Portugal. Results suggested a significantly greater number of ATEs in patients treated with Avastin at 12.4% (12/97) vs Lucentis at 1.4% (3/219), p<0.00001. Review of baseline characteristics across groups suggests balance across age and visual acuity but with longer days of follow up and total injections with Avastin. Subsequent analysis controlling for a fixed follow time period and comparable injection burden suggests persistent of adverse Avastin trends.
Results from Portugal are supported by a recent larger review of 146,942 Medicare beneficiairies in the US where 19,026 patients receiving Lucentis and 21,815 patients receiving Avastin were seen to have differences in risk for stroke and mortality with hazard ratios much lower for Lucentis. Retinal expert feedback has suggested that potential systemic effects of Avastin are greater than with Lucentis, and we expect adverse trends in CATT to support renewed anti-VEGF brand dominance.