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Over the past 12mo, TRIL has "outperformed" just over half of all bio's. Its -44% return places it 180 of 362 bio's which isn't all that bad considering its lead compound just entered the clinic, there's a complete lack of safety or efficacy data, and the risk-off environment.
When you choose equities, then healthcare, then biotechs, then small caps with a pre-clinical asset, volatility and waiting for data just come with the territory.
Alliqua (ALQA) Snapshot
Key Strengths
Strong revenue growth (+250% in 2015e) from internal, partnered and acquired products (expecting guidance to be raised at 3Q15 conf call)
Increasing margins (approaching 70% on internal products) reflects the continued shift away from historic contract mfr business
Improved balance sheet (recently raised ~$30M at $4.55)
Partner Validation (Celgene has a 15% equity position and both their CMO and Head of CELG Cellular therapeutics sit on the BoD)
Continued on Biotech Values
Full Disclosure: I'm long ALQA. Hopefully, the following can serve as a jumping off point for your dd.
Alliqua (ALQA) Snapshot
Key Strengths
Strong revenue growth (+250% in 2015e) from internal, partnered and acquired products (expecting guidance to be raised at 3Q15 conf call)
Increasing margins (approaching 70% on internal products) reflects the continued shift away from historic contract mfr business
Improved balance sheet (recently raised ~$30M at $4.55)
Partner Validation (Celgene has a 15% equity position and both their CMO and Head of CELG Cellular therapeutics sit on the BoD)
-----------------
Key Risks
50% of Wound care market is controlled by a handful of large players (3M, J&J, Covidien, CR Bard, Smith & Nephew, and ConvaTec)
Integration of the recently acquired Celleration business
Continued MAC reimbursement approval
Rapid growth may pressure cash flow - however the company expects no near-term dilution outside of a possible acquisition
Basically it boils down to execution and ALQA has a strong partner in Celgene and an experienced management team with a track record of building a $1.4B wound care business while running Bristol Meyers' Convatec Unit (which was subsequently spun-out to a Private Equity firm)
Wound care is an $8B global market. ALQA now has products that address 6 of the 10 key categories in the segment. As ALQA continues building a clinical argument for its products within the core DFU/VLU (diabetic foot ulcers, chronic venous ulcers) segment, Revenue is expected to grow by over 80% in 2016. Expansion of the company’s Biovance amniotic tissue platform along with two additional CELG products in 2016 represents significant upside.
Going into 2016, I'm looking for revenue multiples to expand to levels more reflective of ALQA's peer group e.g. OSIR, MDXG
Reduced execution risk as company integrates recent Celleration acquisition
Additional regional Medicare MAC) coverage for Biovance and MIST Therapy
Sales force build out (50+ sales team in place by early 2016) to leverage additional product pipeline
With ALQA currently trading near its 52wk low, the mid $3's represents an attractive entry point and, based on 2016e revenues of $33M @ 5X EV, offers a potential 75% 12mo return from current levels.
It could have been Elk but I heard it was Alpaca...not sure but I think he became a vegan.
"the holding period is suspended for the period during which the stock is covered for purposes of determining the character of gain or loss. "
See: http://www.bbdcpa.com/uncategorized/tax-consequences-associated-with-option-strategies-part-iii/
April 1st was the effective date of last financing. Leerink and Cowen were both underwriters. If you move forward 25 days to account for the quiet period, today was the first day analysts could initiate coverage.
We are coming up on a year since the wheels fell off the ARIA wagon. Considering the stock is actually lower now then it was then, I'm shocked to see the level of support Berger continues to receive from some here. The only explanation that makes sense to me is that they must be suffering from Stockholm syndrome. Let's not forget that at the same time Harvey Berger was telling investors the Iclusig AE's were no big deal, the FDA had already approached the company about the issue. While he was telling investors that there was nothing to see here, he was actually selling millions of dollars in stock and then had the audacity to claim he would buy more if he could. He left every single shareholder holding the bag.
Imo, he's still screwing over shareholders. Denner wanted to increase his stake but Berger prevented him. What kind of CEO makes it HARDER for an investor to own shares in a company? The kind who puts his job above shareholders best interests. ARIA: The stock where losing less is the new winning!
Certainly, we are all responsible for our own investment decisions but it's a lot harder to invest wisely when you have a CEO who's willing to tell half truths. Speaking for myself, I know that his actions made it easier for me to ignore the facts. Well, never again.
There are two paths ARIA can take. Path one, aka Harvey's path, is only for "true believers" hellbent on proving that pona is more than a T315I+salvage drug. This path is very long and has many false summits along the way. Resurrecting pona is not for the faint of heart as it will take many more trials which certainly leads to greater dilution and, at least initially, a lower stock price. The phrase "this is going to hurt but it's for your own good" comes to mind.
The second path, aka Denner way, merely requires that Denner publicly reject Harvey's leadership. On the day that happens I believe the stock will trade above $8 and once ARIA is in "play" I can easily see it trading at $10-$12 with a buyout premium.
If you are an institutional holder and could double your money today versus holding on for years hoping that a smaller piece of a bigger pona pie is worth more, which would you choose?
Either way I wish the old-timers here the best of luck.
I'm not sure Novo Nordisk's decision to return NKG2A to IPH was driven by the Ph1 RA results. When Novo swapped lirilumab back to IPH a few years ago, they acquired the full rights to anti-NKG2D (NN8555)(formerly IPH 2301) which is currently in a Ph2b Inflammation/ Crohn's disease trial. Perhaps Novo felt sufficiently comfortable with the progress of the NKG2D asset in inflammation that it made sense for them to move forward with a 100% owned drug. My bet is that once Novo decided to progress NKG2D, they recognized the best way to leverage their NKG2A asset was to to license it back to IPH. At the same time I think it's interesting that Novo took equity in IPH for the NKG2A asset.
fwiw, there seems to be quite a bit of literature supporting HLA-E expression by tumor cells, most notably in lymphomas, ovarian, gliomas, colon cancer, and melanomas. I was somewhat surprised at how quickly the company is moving forward with Ph2 PoC trials across three indications in both single-agent and combo trials.
This financing is highly dilutive but I believe it significantly changes the risk profile of the company. Frankly, I didn't think they would be able to raise this amount of funding. Also, nice to see some quality names in the deal.
-----------------
STEM CELL THERAPEUTICS COMPLETES $33 MILLION PRIVATE PLACEMENT
Toronto, Canada – December 13, 2013 – Stem Cell Therapeutics Corp. (TSX-V:
SSS; OTCQX: SCTPF), an immuno-oncology company developing cancer stem cellrelated
therapeutics, is pleased to announce that it has raised gross proceeds of $33
million through a private placement of units. The financing proceeds will be used to
advance the Company’s CD47 cancer stem cell program through IND-enabling studies,
manufacturing and phase 1 clinical trials.
“The transformation of Stem Cell Therapeutics into a global competitor in the immunooncology
space requires a value-driving asset backed by world class science, access to
significant capital, experienced leadership, as well as a strong and knowledgeable
investor base. As of today we have all those components,” commented the company’s
CEO, Dr. Niclas Stiernholm.
The financing was led by a prominent U.S. healthcare fund, with participation from
several other premier U.S. healthcare institutional investors, including Special Situations
Funds, Ridgeback Capital, Merlin Nexus, Sabby Capital, venBio, Opaleye Management
and HSMR Advisors. Bloom Burton & Co. acted as lead agent for the private placement.
ROTH Capital Partners, LLC acted as placement agent in the United States.
“The significant investment and validating sponsorship from these reputable life sciencefocused
funds is the result of a concentrated effort to introduce the U.S. investment
community to our CD47 immune checkpoint program since the acquisition of Trillium
Therapeutics in April 2013,” added Dr. Stiernholm.
In connection with the offering, the Company issued 157,142,858 units at a price of $0.21
each. The units consisted of either one common share and three-quarters of a common
share purchase warrant (“Common Share Units”) or one Series 1 Non-Voting First Preferred Share and three-quarters of a common share purchase warrant (“Preferred Share Unit”). Of the total Units issued, 79,247,693 units were Common Share Units and 77,895,165 units were Preferred Shares Units. Each whole warrant entitles the holder to purchase one common share at a price of $0.28 at any time prior to expiry on December 13, 2018. Following the offering, the Company has 121,752,380 common shares issued
and outstanding (144,031,618 on a fully diluted basis.
http://bit.ly/18qAIT8
In order to determine the present value of the CELGZ CVR, you need to discount the future cashflows. Using CELG weighted average cost of capital of ~ 7%, Friday's closing price of $1.99 equates to ~ $1.6B in Abraxane sales. I believe CELG internal estimate for Abraxane sales is around $1.8B which would value the CVR ~ $2.65. $2-$2.65 roughly brackets where CELGZ has been trading since the milestone payment. If you think that Abraxane peak sales will be much higher, then the CVR is certainly attractive at current prices; however, it is worth keeping in mind that the further out those sales materialize the less value they'll have today.
p.s. CELG may consider buying-in the CVR at some point
You won't get any argument from me that hitting VEGFR is a concern. However, I'm betting that Iclusig's overall safety profile is in the same ballpark as existing tki's. If it is, and Iclusig delivers a significantly better MMR when compared to sprycel/tasigna (say 55% vs 45%),then the market is significantly underestimating the 1L/2L commercial opportunity. Hopefully MDA's ASH data will provide additional clarity...buckle up!
The ARIA short thesis, as propagated by @adamfeuerstein, would have one believe the EPIC trial's cardiovascular related exclusion criteria was changed in March because of a supposed safety imbalance between the trial arms. What's conveniently ignored by his reporting is that if a significant imbalance between the gleevec and iclusig arms actually did exist, it would have been reported out by the Data Safety Monitoring Board. Very simply, if the DSMB hasn't stopped or changed the trial, then the only reasonable conclusion one can draw is that at least up to now there has been no significant imbalance between the two arms.
Bears also point to the changed EPIC trial exclusion criteria as somehow indicative of Iclusig being a "dirty drug" that causes increased cardiovascular risk. So, let's take a look at the actual changes to the EPIC trial's CV related language and see how it compares to that used by Tasigna in its first-line CML trial:
............................ICLUSIG.....................................................................................TASIGNA...........................
Myocardial infarction, within 6 months prior to randomization...........History of clinically documented myocardial infarction
Unstable angina within 6 months prior to randomization..................History of unstable angina (during the last 12 months)
Congestive heart failure within 6 months prior to randomization.........Clinically significant heart disease/congestive heart failure
Based on the above, at least as far as CV risk is concerned, Tasigna is clearly more restrictive than Iclusig. In fact, Tasigna excludes ANY patients with documented myocardial infarction, while Iclusig allows patients who have had an MI as recently as 6 months. The same is true for congestive heart failure. Ironically, the very changes the shorts use to bolster their claims may actually make it slightly less, not more, likely for a CV event to occur. Why? Enrolling patients with less cardiovascular risk makes an unrelated CV event less likely.
So, what about Sprycel? As far as myocardial infarctions are concerned, Sprycel uses exactly the SAME 6 month exclusion criteria as the Iclusig language. The point is that all of these TKI's have cardiac AE's - the most reasonable explanation is that it's a class effect. Despite these concerns, Tasigna and Sprycel will each have over $1 billion in revenue in 2013. As Biomaven points out cardiac adverse reactions were reported in 7% of 258 patients taking SPRYCELin front-line. To put this in perspective, in the PACE trial which treated much sicker 3/4th line patients, 5% of patients had myocardial ischemic SAEs and the majority of those patients had pre-existing conditions (see ASCO 2013 abstract http://meetinglibrary.asco.org/content/109745-132 )
Finally, the shorts point out that MD Anderson's decision to run an expansion cohort using a 30mg dose must mean there's a safety issue at the 45mg dose. @dsobek did a fine job explaining the fallacy of that logic in his recent article. However, I would also like to point out that the very same investigator, Dr. Jorge Cortes, who is running the 45mg/30mg front-line study is also running a separate 45mg second-line trial. The shorts would have us believe that Dr Cortes would drop the dose in the front-line trial because of safety concerns but then leave both the dose and exclusion criteria in the second-line trial completely unchanged. Not only did Dr Cortes not change the dose in the 2L trial, he kept the unstable angina, myocardial infarction, TIA, stroke or heart failure exclusions at their original 3 months...not even increasing them to the 6 months the bears would have us believe is so disconcerting in the EPIC trial.
Clearly, the 30mg dose expansion cohort is exploratory. In practice, dose titration is common with both sprycel and tasigna so it only makes sense to explore Iclusig at lower doses in small n, IST led trials. But, instead of taking my word for it, thanks to the interwebs, you can listen to Cortes' 2012 ASH presentation and hear what he thinks about Iclusig yourself
I hadn't seen this one. Do you or Peter have any thoughts on Stem Cell Therapeutics Patent/IP as it relates to Stanford's? SCTPF's CEO recently said that Stanford's patents are particularly broad and might even squeeze out InhibRx. He suggested that this was the reason SCTPF went with the natural ligand to CD47, however, it is unclear to me whether the MOA between the two approaches is really all that different. Any thoughts/comments would be appreciated.
One clarification, the second-line patients I referenced achieved a 53% MMR in 15 months while the EPIC endpoint is actually 12 month MMR.
Since CD47 is also expressed on healthy cells, a concern is that anti-CD47 will also block healthy cells from expressing CD47 and consequently they will be "eaten" by mistake. The hope is that the lack of calreticulin expression in healthy cells will allow the anti-CD47 drugs being developed by Stanford and SCTPF to safely initiate phagocytosis in cancer cells. Calreticulin is a pro-phagocytic molecule that is highly expressed on the surface of several types of human cancer cells, including acute myeloid and lymphoblastic leukemias. Importantly, calreticulin is not expressed on healthy cells. If CD47 is the "don't eat me" signal then think of calreticulin as the "eat me" signal. It appears that in order for phagocytosis to occur requires both the CD47 "don't eat me" signal be turned off and a calreticulin "eat me" signal to be turned on. The hope is that turning off the "don't eat me" signal in healthy cells alone won't be sufficient to initiate phagocytosis because healthy cells don't express the calreticulin "eat me" signal.
With 3Q GDP likely under 2% and inflation risk nowhere in sight, I think the Fed is tilting at windmills. Based on the data already in hand, it would be hard to justify tapering before year-end let alone September.
Peter, your points are well-taken. In the end, I concluded that Stanford's progress would at least validate the target. My bet is that a partnership may be possible as soon as Stanford validates CD47 in the clinic and SCTPF has IND-enabling studies in hand.
Stem Cell Therapeutics (SCTPF) is certainly highly speculative. In fact, it's all the things I typically avoid in an investment--- it's an under-capitalized, Canadian penny stock with little data. The company is just beginning CD47 IND-enabling studies and will need to raise millions of dollars before it is able to bring a drug to the clinic. However, even with all those negatives, I decided to take a position.
CD47 could be a very important oncology target. In order for cancer to survive, it first has to find a way to evade the innate immune system, specifically, macrophages. Macrophages clear the body of dead/dying/damaged cells. Basically, macrophages have a signal regulating protein on their cell surface called SIRPa. Healthy cells express CD47 which binds to SIRPa and transmits a "do not eat me" signal to the macrophages. If a cell doesn't express sufficient CD47 then the macrophage engulfs the cell in a process known as phagocytosis. Some cancers - so far mainly hematological such as leukemia and lymphoma - have found a way to avoid phagocytosis by expressing CD47 on their own surfaces, in essence, tricking macrophages into leaving them alone. Both Stanford and Stem Cell Therapeutics (SCTPF) are developing an anti-CD47 fusion protein that prevents cancer cells from usurping this process.
If anyone's interested, here's the research I relied on to make my risk/reward assessment.
CD47 Overview
Blocking the CD47 "don't-eat-me" signal through the use of anti-CD47 antibodies http://stemcell.stanford.edu/CD47/
CD47 plus Rituximab boost the destruction of cancer cells http://med.stanford.edu/ism/2013/june/sirp-0610.html
CD47 plus Rituximab synergize to trigger the host’s own immune system to eliminate the cancer http://med.stanford.edu/ism/2010/september/cd47.html
CD47 activates CD8+ T cells to attack the cancer cells http://med.stanford.edu/ism/2013/may/cd47.html
Stem Cell Therapeutics/Trillium http://www.stemcellthera.com
July 2013 Corporate Presentation http://www.stemcellthera.com/Documents/Presentations/SCTPresentation_SCTCorpNCQ3webx.pdf
Targeting SIRPa in cancer http://www.landesbioscience.com/journals/oncoimmunology/2012ONCOIMM0376.pdf
SCT Patent: Compositions and methods for treating hematologic cancers targeting the SIRP - CD47 interaction http://www.google.com/patents/EP2429574A1?cl=en
SCT Patent: Modulation of SIRPa - CD47 Interaction for Increasing Hematopoietic Stem Cell Engraftment and Compounds therefor https://www.google.com/patents/WO2009046541A1?cl=en
March 2013 Prospectus https://www.otciq.com/otciq/ajax/showFinancialReportById.pdf?id=104032
Abstracts/Scientific Review
CD47 is an adverse prognostic factor and therapeutic antibody target on AML stem cells http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2726837/pdf/nihms122682.pdf
SIRPa Inhibits Growth and Induces Programmed Cell Death in AML http://www.plosone.org/article/fetchObject.action?uri=info%3Adoi%2F10.1371%2Fjournal.pone.0052143&representation=PDF
Anti-CD47 antibody synergizes with rituximab in NHL http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2943345/pdf/nihms227474.pdf
Anti-CD47 antibody primes an effective anti-tumor T-cell response http://www.pnas.org/content/early/2013/05/17/1305569110.full.pdf
The CD47 (SIRPa)is a therapeutic target for human solid tumors http://www.pnas.org/content/early/2012/03/20/1121623109.full.pdf
SIRPa is the key CD47 binding partner modulating stem cell survival http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3457732/pdf/JEM_20120502.pdf
Issues to Research:
Stanford Patent: Methods for manipulating phagocytosis mediated by CD47 http://www.google.com/patents/US20110014119
Will combining anti-CD20 and anti-CD47 antibodies overstimulate the immune system? http://www.nature.com/scibx/journal/v3/n37/pdf/scibx.2010.1109.pdf
Most normal cell don’t display calreticulin and are therefore not depleted when exposed to a blocking anti-CD47 antibody http://med.stanford.edu/ism/2010/december/crt-signal.html
Calreticulin minimally expressed on most normal cells http://www.ncbi.nlm.nih.gov/pubmed/21178137
Is targeting of CD47-SIRPa enough for treating hematopoietic malignancy? http://bloodjournal.hematologylibrary.org/content/119/18/4333.full?ijkey=4eb4ef03d1850517aa59542815e25852e0473e6c&keytype2=tf_ipsecsha
Response: Is targeting of CD47-SIRPa enough for treating hematopoietic malignancy? http://bloodjournal.hematologylibrary.org/content/119/18/4334.full?ijkey=ee4a58f5d1c40227e80fc4dcde146b7f2b57d196&keytype2=tf_ipsecsha
ARIA AP26113 Update included in "Abstracts not to be missed" at upcoming ESMO/ECCO Conference http://bit.ly/14Vf5pK
Abstract Titles for the upcoming ESMO/ECCO Conference are now available online http://bit.ly/17ye45G
I was very interested in EYEGUARD A/C expanding the market beyond Behçet's...with those trials now at least a year off, I sold my position at $4.86 from ~$2.60.
Dose titration in front-line makes a lot of sense..glad to see Cortes is exploring the 30mg possibility. I suspect some front-line patients will need less than 45mg, so it can only help to have data that possibly shows the same benefit is achievable at 30mg but with fewer side effects.
Thx but there really isn't much to say until we have some front-line data. Hopefully we'll get a precursor to the EPIC trial at ASH from the MDAnderson IST. If the 12 month MMR is as I expect (58%+) and the safety profile is comparable to sprycel/tasigna, then today's arguments will finally be put to rest. Generic gleevec will offer the best value while Iclusig will be best-in-class...both will do well imo at the expense of tasigna/sprycel.
I think a lot of folks missed this one...it's hard to drive looking through your rear view mirror ;)
Xoma's EOA Ph2 data is now expected in Oct.
XOMA Completes Enrollment in Phase 2 Proof-of-Concept Gevokizumab Trial in Patients With Erosive Osteoarthritis of the Hand
BERKELEY, Calif., July 22, 2013 (GLOBE NEWSWIRE) -- XOMA Corporation (Nasdaq:XOMA), a leader in the discovery and development of therapeutic antibodies, today confirmed it has completed patient enrollment in the Company's Phase 2 proof-of-concept (POC) study designed to evaluate the potential for gevokizumab to improve pain symptoms, physical function and structural abnormalities in patients with active inflammatory, erosive osteoarthritis of the hand (EOA) and elevated C-reactive protein (CRP) levels. XOMA's study enrolled approximately 90 patients who were randomized 2:1 to receive 60mg of gevokizumab dosed subcutaneously once monthly or placebo. The study was designed and powered to detect a significant improvement from baseline versus placebo in the mean Australian/Canadian Hand Osteoarthritis Index (AUSCAN™) pain score in the target hand at Day 84. The study also is capturing multiple outcome measures, including pain, stiffness, physical function, X-ray, radiographic and MRI changes, as well as changes in CRP and concomitant acetaminophen use, at three and six months. The Company anticipates having preliminary top-line data for the AUSCAN score in October.
XOMA also provided an update on the EOA study in patients with non-elevated CRP. This study was opened for enrollment in May, and investigators have enrolled approximately 40 patients of the targeted 90 who had qualified for the original EOA study with the exception that they did not have elevated CRP levels. This supplemental study will help inform the design of the potential Phase 3 studies of gevokizumab in EOA.
http://investors.xoma.com/releasedetail.cfm?ReleaseID=779233
According to the Credit Suisse report, "Gleevec/Glivec is used much more frequently in the first line setting in the EU than the US (74% vs. 46%)." Using those percentages, until the EPIC results are in, Iclusig only has the possibility of being used in second-line 26% of the time in the EU (other than T315I).
"This is not an unfair report and is not biased to favor Novartis in some way."
I agree, whole-heartedly.
Basically, the question Credit Suisse asked was 'if Iclusig is only marginally better would you continue to prescribe Sprycel/Tasigna?' To no great surprise, most answered yes.
The one question I'm most interested in is 'if the 12 month, EPIC MMR is significantly better than the results achieved by Sprycel/Tasigna, would you prescribe Iclusig in a first or second-line setting?' I suspect that question would have a much different result than the one seen in the Credit Suisse survey.
I've never implicitly handicapped the three trials. I merely pointed out that, of the 3 trials, Eyeguard B is the only one with Ph2 results which introduces an additional risk factor to the investment decision. For the record, I agree with your and iwfal's assessment.
To clarify my earlier post, I am drawing a distinction between corporate currency risk (which among other factors is dependent on whether a product is sold in a customers local currency and repatriated) and the currency risk associated with investing directly in a non-US corporation as compared to using its ADR. The former represents translation risk which hits a company's P&L while the latter is a transactional risk that hits your wallet.
You are right, you can't, but I was attempting to answer a different question. You can control whether you choose to settle your trade in dollars (GLPYY) or euros (GLPG.BR). You can also control the timing of your purchase and sale. I haven't run the numbers yet but just eyeballing it, if you were to convert your shares back into USD today, the gain in GLPG.BR's stock price over the last few weeks looks like it was completely offset by the the stronger dollar. In comparison, the ADRs are up ~ 10% over the same time period.
I recently started a small position in the ADR, GLPYY. If you are converting $'s to make your purchase, an additional consideration is that GLPG.BR, unlike the GLPYY, has currency risk. In the last few weeks, the $USD has strengthened considerably versus the $EUR resulting in ~ 7% greater purchasing power. If i was making my purchase in the next few weeks, I'd probably bet on the $USD continuing to strengthen and buy GLPG.BR with it's greater liquidity.
Somewhat perversely, the narrower EU label (sprycel/tasigna failures) may actual help Iclusig's premium pricing strategy.
SL-401 receives Orphan Drug designation in BPDCN. A pivotal registration trial is expected to start in early 2014.
NEW YORK, June 10, 2013 (GLOBE NEWSWIRE) -- Stemline Therapeutics, Inc. (STML) today announced that SL-401 has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive hematologic malignancy for which there is no effective treatment. SL-401 also has Orphan Drug status for the treatment of acute myeloid leukemia (AML).
SL-401 is a novel targeted therapy directed to the interleukin-3 receptor (IL-3R) present on tumor bulk and cancer stem cells (CSCs) of multiple hematologic cancer indications. SL-401 has demonstrated single agent clinical activity in patients with advanced hematologic cancers, including BPDCN, AML, and myelodysplastic syndrome (MDS).
"We are focused on the rapid development of SL-401 due to its potential for the treatment of IL-3R-expressing hematologic malignancies," commented Eric K. Rowinsky, M.D., Chief Medical Officer and Head of Research and Development at Stemline. "SL-401 is demonstrating robust clinical activity in heavily-pretreated patients with BPDCN who are refractory to available therapies, including high-dose chemotherapy and allogeneic stem cell transplantation. This Orphan Drug designation provides us with a number of benefits that further strengthen our SL-401 program. Stemline will progress SL-401 into pivotal trials in BPDCN and AML in the near-term, and we will continue to evaluate SL-401 in a wide range of additional IL-3R-expressing hematopoietic malignancies."
About Orphan Drug Designation:
Orphan Drug designation is granted by the FDA's Office of Orphan Products Development for drugs that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer U.S. patients annually. Orphan Drug designation qualifies a company for several benefits under the Orphan Drug Act of 1983. The benefits apply across all stages of drug development and include accelerated approval process; seven years of market exclusivity following marketing approval; tax credits on U.S. clinical trials; eligibility for Orphan Drug grants; and waiver of Prescription Drug User Fee Act (PDUFA) and certain other administrative fees.
About Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN):
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive hematologic cancer for which there is no effective treatment, carries a poor prognosis and represents an unmet medical need. BPDCN had previous names, including blastic NK cell lymphoma and agranular CD4+/CD56+ hematodermic neoplasm, until 2008 at which time the World Health Organization (WHO) renamed this disease BPDCN. The disease most commonly affects middle-aged and older patients and is approximately three times more common in men than women. BPDCN derives from plasmacytoid dendritic cells, which are specialized cells of the immune system. BPDCN cells express high levels of IL-3R. BPDCN, which has features of both leukemias as well as lymphomas, typically presents with skin lesions, and often also involves the bone marrow and blood, and can also involve the spleen and lymph nodes. BPDCN proliferation in the bone marrow results in decreased blood cell counts, which can lead to serious infections, anemia, bleeding, and invariably death. Although BPDCN can be controlled for brief periods with standard chemotherapy, including high-dose chemotherapy with bone marrow transplantation, overall prognosis remains poor and the median overall survival from diagnosis is approximately 12 months. There are currently no approved therapies for BPDCN, and an optimal therapeutic regimen for BPDCN has not yet been established.
About Stemline:
Stemline Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing novel oncology therapeutics that target both cancer stem cells (CSCs) as well as the tumor bulk. Stemline's clinical candidates, SL-401 and SL-701, have demonstrated clinical activity, including durable complete responses (CRs), in Phase 1/2 studies of patients with advanced hematological and brain cancer, respectively. For more information about Stemline Therapeutics, visit www.stemline.com.
Stemline's June 2013 Corporate Presentation can be downloaded here
ABBV, ARRY, CRIS, GILD, GLPG, INCY, SRPT are among the companies presenting at the June 18-19 Wells Fargo Healthcare Conference. A complete list can be downloaded here http://bit.ly/11FdxKK (pdf)
XOMA ReadMeFirst
(Update: Ph2 Gevokizumab trial in pyoderma gangrenosum, Competition Update)
Hopefully, this RMF will save folks some time and make it easier to research the company. It is meant to be a convenient jumping off point but, of course, it's not a substitute for your own dd.
Company Overview
#msg-87804025 XOMA’s May 10, 2013 Credit Suisse Presentation
#msg-87745998 Q1 2013 Results and CC Notes
#msg-83090011 Archived Company Presentations
#msg-83090054 Credit Suisse Investment Thesis
#msg-83090160 Servier Partnership Terms
#msg-83091150 Recent Financings and Baker Bros Investment
#msg-85039491 RBC Capital Markets Global Healthcare Conference Notes
#msg-85514239 XOMA Major Shareholders
XOMA 052 (Gevokizumab) Overview
#msg-83090144 XOMA/Servier to commercialize gevokizumab (XOMA 052) for the treatment of multiple inflammatory disorders
#msg-83091229 IL-1B has emerged as a therapeutic target for inflammatory disorders
#msg-83091258 Is Gevokizumab a “best-in-class” anti-IL-1ß therapeutic antibody?
#msg-83090127 Current Clinical Trials
Uveitis Program
#msg-83091071 Uveitis Market Forecast
#msg-83090431 Uveitis of Behçet’s Disease Pilot Trial Results
#msg-83090221 FDA Grants Orphan Drug Status to Gevokizumab
#msg-83090262 Servier Initiates Behçet's Phase 3 Clinical Trial (EYEGUARD-B)
#msg-83090953 Phase 3 clinical trial in patients with non-infectious uveitis intermediate, posterior, or pan-uveitis (NIU) (EYEGUARD-C)
Proof of Concept Program
#msg-88960499 Ph2 Gevokizumab trial in pyoderma gangrenosum
#msg-83106427 Phase 2 Study of gevokizumab in moderate to severe acne vulgaris
#msg-83122761 Results of Phase 2 Study for Moderate to Severe Acne Vulgaris
#msg-83091380 Phase 2 study of gevokizumab in active inflammatory, erosive osteoarthritis of the hand
#msg-83091342 Non-Infectious Anterior Scleritis selected as next PoC candidate
Diabetes Program
#msg-83090114 Phase 2b trial of XOMA 052 in Type 2 diabetes patients did not achieve the primary endpoint
#msg-83091405 XOMA Discovers Two New Classes of Insulin Receptor-Regulating Antibodies
Cardiovascular Program
#msg-83091327 PoC Gevokizumab Study in Patients With a History of Acute Coronary Syndrome
Aceon Program (Perindopril/Amlodipine)
#msg-83091483 Aceon Phase 3 PATH Trial Meets Primary Endpoint
#msg-83091538 Credit Suisse' Take on the Aceon Opportunity
Competition
#msg-87832678 Competing Ph3 trials in non-anterior uveitis
#msg-87833327 ABBV is testing Humira in two phase-3 uveitis studies
#msg-83091092 Lux Biosciences declares intent to halt application efforts for uveitis treatment
#msg-83091763 Eyegate Pharma's pivotal Phase III study of EGP-437 in patients with anterior uveitis
#msg-83091599 XBiotech Announces Positive Phase II Results in Acne Vulgaris
Not sure where you got the rant impression, lol. fwiw, I think it was smart for Berger to try to set the 12mo MMR bar at 50 (even though I believe it will come in higher). As far as the availability of T790M data at ESMO, the desire to get the highest dose possible likely delayed the start of the Ph2 cohorts by a couple of months so I'm not sure what kind of duration of response data will be available but, at this point, expectations are so low that anything positive could lead to an upside surprise.