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“This Tiny, Unknown Biotech is About to Unleash Its ‘Holy Grail’ Drug”

Biotech Supertrader teases that "This May be the Most Radical Advance in Medicine in the Last 100 Years"

By Travis Johnson, Stock Gumshoe, January 8, 2014

Robert Morris is helming a biotech-focused stock newsletter that’s called Biotech Supertrader (modesty has no place in the world of newsletter promotions, of course), and I’ve never covered this letter before so I thought I ought to have a look at the latest teaser we’ve been asked about.

Morris, incidentally, has been featured in our pages before — but that was back when he was editor of China Stock Insider at the same publisher. That letter, like almost all China-focused investment newsletters, seems to have disappeared quietly into that good night … which probably tells you that it’s time to invest in China again, since the newsletter publishers are ignoring the Middle Kingdom and rushing out their pitches about biotech and tech stocks. At the time, Morris was teasing NQ Mobile (NQ), which has turned out to be pretty good if you bought it down there in the $6-8 neighborhood (though it’s been a wild ride).

So now what’s he pitching for his Biotech Supertrader?

Well, the destruction of “Man’s deadliest disease”, of course. Here’s how the teaser gets our attention:

“This Tiny, Unknown Biotech is About to Unleash Its ‘Holy Grail’ Drug on Man’s Deadliest Disease

“Their ‘Guided Missile Approach’ Could Save Thousands of Lives Each Year

“It’s about to become the most talked about advancement in cancer treatment in our lifetimes and you can lock in a life-transforming fortune if you act quickly….

“I’m urging my subscribers to load up on this stock NOW….

“I’ve just uncovered a tiny, unknown biotechnology company with a new cancer drug in phase 3 clinical trials which is showing remarkable success at treating several types of cancer.

“Their scientists have found an innovative approach to cancer care which involves a breakthrough in treatment. It goes deep inside the inner workings of our cells.

“Plus, this medicine looks to be many times more effective and with fewer side effects than the chemo, radiation, and drug therapies currently available.”

If there’s one thing that investors know can make them rich and make them feel good about themselves and the world, it’s a cure for cancer — we’ve seen that effective cancer treatments can and do (occasionally) turn little biotech stocks into gigantic successes, so the dream lives on that you’re going to catch one of these lottery tickets and own the next Genentech. Will we be so lucky? Well, let’s see which one he’s pitching:

“When this drug wins FDA approval – which I believe it will – this small company’s $4.16 stock price will go straight to the moon.

“And the market for this drug is absolutely huge!

“You see, this small biotech is targeting its new drug, let’s call it ‘drug S’, at cancers of the blood and bone marrow. And it is already in very promising phase 3 trials for these two types of cancer.

“But here’s where it gets really interesting. It looks like the drug this company is developing will also work on other types of cancer!

“There are positive signs it works on Non-Small Cell Lung Cancer (NSCLC) too. There are 1.1 million people with this type of malignancy. Just in the United States alone there are over 300,000 patients with this disease according to The American Cancer Society. Each desperate for a cure.

“Plus it looks like ‘drug S’ may turn out to be an effective treatment for ovarian Cancer. There are more than 204,000 new cases of ovarian cancer diagnosed worldwide each year with 22,280 of these in the United States according to the National Cancer Institute estimates.”

So … who is it? Thinkolator sez this is Cyclacel Pharmaceuticals (CYCC)

Cyclacel is indeed a little biotech around $4 (it closed at $4.35 yesterday), with a market capitalization of only about $80 million — so be careful, we’re a big enough group here that if just a small percentage of Stock Gumshoe readers got enthused about this stock it could drive the shares up, less than a million dollars worth of shares trade each day (Biotech Supertrader says they limited their readership to 750 people — I don’t know if that’s still their cap or if they’ve hit it, but we’ll have more folks than that reading this free article).

And like many biotech stocks, it’s got some impressive scientists and it’s been losing money for a long time as they’ve been searching for a viable drug (their current lead drug also was a big focus of theirs back when it was in Phase 1 trials five or more years ago, so that’s a good reminder of the time these things take, it’s just starting Phase 3 trials now). It looks like they must have gone public in 2004, when they were about eight years old, and a quick scan of ten years of their financials over at Morningstar indicates that they’ve never generated more than a token amount of revenue (meaning, they’ve probably had some research collaboration payments or partnership funding, but never got a product to market), and have accumulated more than $250 million in losses to date. And had two reverse splits to keep the price from sinking far into penny territory.

So that’s not unusual, but it means that — as with all developmental-stage biotechs — it’s not about the financials or the fundamentals, it’s about what’s going to happen in their clinical trials and whether things are going well enough that they can continue to finance the trials … which get much more expensive as you progress through Phase 2 and Phase 3.

All I know about them so far is that they say they’ve got enough cash to get through enrollment in their key Phase 3 study for “drug S” (which is sapacitabine) as of September when they last updated their investor presentation, but I know nothing about the science or the competing cancer drugs that are out there or how fabulous this particular one might be, so I asked our favorite medical writer, Doc Gumshoe (who, yes, is not a doctor) to check them out quickly and chime in. Here’s what he could share after looking into them for a few minutes (he’s just looking at the medical stuff, not so much the “investor presentations”):

    Cyclacel’s Prospects

    Cyclacel has three drugs in development at this time, and is involved in eight clinical trials with these drugs, not including two clinical trials that have been terminated. Their top contender is sapacitabine which targets the division of cancer cells. If you can prevent cancer cells from dividing and reproducing, you have the cancer whipped, so targeting cancer cell division (or mitosis, which is the technical term) is a highly promising avenue for treating cancer. However, we need to take note of the fact that sapacitabine is one of a large number of drugs that propose to fight cancer by this method.

    At present, all eight of Cyclacel’s clinical trials involve sapacitabine. Of these, at least one has been completed – a Phase 1 study of the safety and pharmacology of the drug. Four others are current, with no information about results. These are likely Phase 1 or small Phase 2 studies, to assess safety, determine what a correct dose might be, and evaluate whether the drug does what it’s supposed to do in human subjects with the target diseases, which in this case include acute myeloid leukemia (AML), cutaneous T-cell lymphoma, and some advanced solid tumors. Prior to the clinical trials, sapacitabine has demonstrated impressive results in delaying the spread of metastatic liver cancers in mice.

    From what I can gather from public sources (i.e., the NIH Clinical Trials Registry), there is one Phase 3 trial, which started recruiting patients in February of 2013 and is expected to be completed in late 2015. The trial is in elderly patients with AML, and compares alternating cycles of sapacitabine and decitabine with decitabine alone. Decitabine (Dacogen) is FDA-approved for treating AML and also targets cancer cells’ replication by attacking their DNA.

    It is possible that the Phase 3 trial by itself could lead to FDA approval for sapacitabine, depending on the strength of the results. However, that trial would not get the drug approved for use as monotherapy, since it is not being investigated as monotherapy. My guess is that Cyclacel is planning more trials of sapacitabine as monotherapy, perhaps in younger patients. And my further guess is that FDA approval is still quite a long way off.

    Sapacitabine is also in a Phase 3 trial with cyclophosphamide and rituximab for the treatment of relapsed chronic lymphocytic leukemia. Cyclophosphamide (marketed under several trade names) is a well-established chemotherapy agent used in a number of cancers, and has led to remission in many cases; however, it is associated with truly harrowing adverse effects. Rituximab (Rituxan, Genentech) is used not only in cancers but in some autoimmune diseases. And sapacitabine is also being studied in patients with previously-treated non-small-cell lung cancers.

    Although the piece from Biotech Supertrader said that the drug – identified as “drug S” –is also a promising treatment for ovarian cancer, I find no clue that it is being studied in such patients. [ed note: that’s because that “promise” is in the lab still, not in people — they had a press release about this in the Fall, “75% of Ovarian Cancer Patient Samples Highly Sensitive to Sapacitabine”, not studied in patients but on patient samples]

    Cyclacel has two other drugs in development: selicilib and a drug designated as CYC116. One selicilib study has been terminated, and in a second Phase 1 study, selicilib is used with sapacitabine in patients with advanced solid tumors. Remember, however, that Phase 1 studies are many rungs of the ladder below what’s needed to gain FDA approval.

    CYC116 is an aurora kinase inhibitor, meaning that it blocks the action of an intracellular enzyme that facilitates cancer cell mitosis. This is a promising avenue of cancer treatment, however, the traffic on this avenue is fairly heavy, and includes several other classes of drugs including tyrosine kinase inhibitors, and taxol based agents such as paclitaxel (Taxol, Bristol Myers Squibb); docetaxel (Taxotere, Sanofi-Aventis), Abraxane (a newer formulation of paclitaxel from Celgene) and others.

    CYC116 supposedly also inhibits vascular endothelial growth factor (VEGF), which induces the growth of blood vessels that nourish cancer cells. Inhibiting VEGF is a well-established means of combating cancer, and CYC116 could hardly be characterized as a radically new departure in cancer treatment.

    The one trial involving this agent has been terminated. That, of course, does not mean that development of CYC116 stops dead in its tracks – there are many reasons why a trial can be terminated, and ours is not to speculate without more information.

    Beyond those three drugs, it’s hard to guess what Cyclacel may have up its corporate sleeve. It is certainly true that a successful cancer drug – even if only moderately successful– can be transformational for the biotech that develops the drug. But the drugs that Cyclacel has under development do not appear to this skeptical observer to be radically new departures in cancer treatment.

    It’s important to remember, when trying to estimate the likelihood of a single drug demonstrating sufficient efficacy and safety to gain FDA approval and market share, that the competitive field is vast. As I mentioned earlier, Cyclacel has a total of 8 clinical trials in process at this time.

    For the sake of perspective, it’s worth knowing that at present there are 41,445 cancer trials being conducted. So those are the odds.

So there you have it — it’s almost impossible to find a development-stage biotech whose financials look great or that makes your heart go pit-a-pat over their valuation, especially in a biotech bull market like we’ve seen over the past year or so, and Cyclacel doesn’t jump out as spectacular on that front either, not unless you’re a big believer in the promise of their specific drug. They’re a small stock and they don’t get much attention, other than from the analysts who probably helped them sell shares in secondary offerings in recent years, and there aren’t any major “skin in the game” insiders as far as I can tell (the CEO owns $1 million worth of shares, but he gets paid more than that every year), and there’s only one really focused owner on the institutional side that seems to have any kind of biotech focus (Eastern Capital owns about 7% of the shares, roughly $5 million worth … don’t know much about them).

So I don’t see a lot to make them stand out other than Robert Morris’ apparent enthusiasm for the shares (which certainly goes over the top, he calls his special report “The End of Cancer Worries Forever“), and I don’t know enough about the science to be a believer (though, to be fair, I almost never speculate on developmental biotechs because they’re so hit-driven and I’m not smart enough to be a hit-picker in the sector). It is at least encouraging that they are enrolling patients for Phase 3, and that they probably won’t have to raise more money before they have some indication of how the trial is going, but sometime in the next year or two they’re probably going to have to either get good results from this trial that let them raise cash at a good price, or have promising enough results that some big pharma company wants to jump in and help fund development of “drug S” (or just buy up the whole company, as happens with some regularity when a little biotech gets promising results).

Oh, and they are presenting at an investor conference next week, so maybe they’ll have something interesting to share then. As you can tell, this one doesn’t jump into my cup of tea … but these kinds of stocks almost never do. Sound interesting to you? Interested in the science or the lottery-ticket possibilities of $80-million developmental biotechs? Have any experience with Robert Morris or know whether or not we should consider him a biotech savant? Let us know with a comment below.

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karmaswimswami
March 6, 2014 5:44 pm

On 5 March 2014, Peter French, MD, the CEO of Benitec, added 9939 shares to his holdings. He already held over 300,000 shares plus about 1.5 million options.

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karmaswimswami
March 6, 2014 6:13 pm

David B: Thanks, that’s helpful. And exciting. Diabetic gastroparesis is a HUGE market. Diabetes causes a neuropathy, such that the stomach bloats up from failure to contract. Food can sit there for what seems like days at a time. We use metoclopramide for it. But long term patients get tardive dyskinesia from that. I sometimes scope these patients and plonk botox into the pylorus to relax it open. Sometimes that helps, sometimes not. But this is shrewd for them to go after. That must be a nicotinic agonist. OAB has many agents now. Not sure that is good to chase. The Alzheimer indication: this would join agents such as rivostigmine as pro-cholinergic therapy and should be beneficial in fact. (There is a lore in medicine than smoking helps and prevents Alzheimer’s. We don’t talk about it.)

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David B
Guest
March 6, 2014 6:38 pm
Reply to  karmaswimswami

My mother is one of those rare smoking survivors at age 93 (quit at 83 during a hospitalization for a broken bone) and she has managed to avoid Alzheimers. Certainly not a a recommended medical path but if a compound can be developed without the side effects (e.g. lung cancer, bronchitis, heart attack, stroke) that would be great. I may need it as I have never smoked and if my Mom’s genes are dominant I could live to 120! Also a great reason for me to invest wisely now : )
One downside I see with TRGT based on what you just wrote is that the compound which is the furthest along is the OAB one. I suppose this isn’t necessarily all bad as even if it has more competition–if they can prove efficacy and safety it could clear a pathway for their other compounds.

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testpack3
Member
March 6, 2014 7:51 pm
Reply to  David B

Re:BLT and MJGD / Irwin Biotech sales,

The announcement by the Co. of the placing to raise 31m mentoned the USA concert party and, other Australian investors. What if the aforementioned nominees are part of the concert party, hence ‘bought in; at 1.07, and sold at 2.00, or had the equivilant no. of shares on a 2/1.07??

only a thought

Good investing

Mr Sparkle
Mr Sparkle
March 6, 2014 7:06 pm
Reply to  karmaswimswami

Star Scientific (STSI) is another North Carolina based company making a product called Anatabloc that was originally derived from tobacco, but is now synthisized. It was initially marketed as a stop smoking rememdy called CigRx, and users reported all kinds of unexpected benefits, especially related to inflammatory diseases. The stock has not done well recently, especially after the FDA raised the issue that it should be regulated as a pharmaceutical rather than sold as an off-the-shelf nutriceal. I personally have found it very useful in releiving joint pain, and there are lots of favorable testimonials which can be found on Amazon or GNC.

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kmf3124
Member
kmf3124
March 6, 2014 7:28 pm

Cramer had the CEO of Sangamo, SGMO on his show today. Interesting interview after the recent press they have received. Believe this was discussed much earlier in the thread but it gives an interesting perspective on how the market is currently valuing Sangamo’s “breakthrough” technology that only they own. I do not own but took the opportunity to double down on Benitec. Here’s to early retirement all.

Many thanks to Dr KSS and all the others who have provided such a valuable insight in this thread.

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Nick Semuta
Guest
Nick Semuta
March 6, 2014 9:58 pm

FYI:
LA JOLLA, Calif., March 5, 2014 /PRNewswire/ — Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that it has commenced dosing RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 (“miR-122”) in healthy volunteer subjects in a Phase I clinical study. The primary objective of the study is to evaluate safety and tolerability of RG-101 and the secondary objectives are to evaluate pharmacokinetics, viral load reduction and any impact an oral direct-acting antiviral may have on the pharmacokinetics of RG-101.

“We are very pleased to have dosed our first human subject, advancing Regulus into a clinical-stage company,” said Neil W. Gibson, Ph.D., Chief Scientific Officer of Regulus. “We continue to be encouraged by the preclinical data seen to date and believe that RG-101 has the potential to be a best-in-class host factor agent, specifically due to its pan-genotypic properties, including demonstrated efficacy in the hard to treat HCV genotype 3, and the potential for a once monthly dosing regimen. We look forward to reporting data from the Phase I clinical study of RG-101 by the end of this year.”

About the RG-101 Phase I Clinical Study

The Phase I clinical study of RG-101 will have four parts: (i) a single ascending-dose study in healthy volunteer subjects; (ii) a multiple-ascending dose study in healthy volunteer subjects; (iii) a single-dose drug-drug interaction study of RG-101 in combination with an approved oral direct-acting antiviral (“DAA”) in healthy volunteer subjects; and (iv) a single-dose study in HCV patients to assess the safety and viral load reduction, which is designed to demonstrate human proof-of-concept. Up to approximately 100 healthy volunteer subjects and HCV patients are planned to be enrolled in the Phase I study, which is being conducted in the Netherlands.

About RG-101 for the Treatment of HCV

RG-101 is a key program in Regulus’ ‘Clinical Map Initiative’, which outlines certain corporate goals to advance its microRNA therapeutics pipeline over the next several years. Under its ‘Clinical Map Initiative’, Regulus expects to demonstrate human proof of concept results in the Phase I clinical study of RG-101 by the end of 2014.

The most abundant microRNA in hepatocytes is miR-122 and is a critical host factor for survival and replication of all know HCV genotypes. RG-101 is a novel anti-miR-122 oligonucleotide therapeutic that is effectively targeted to hepatocytes for the treatment of HCV through conjugation to GalNAc, a carbohydrate-based chemistry approach for asialoglycoprotein receptor-mediated delivery of oligonucleotides to hepatocyte cells of the liver. Utilizing the GalNAc conjugate chemistry has significantly improved the potency of the active oligonucleotide of RG-101 by achieving targeted delivery of the oligonucleotide to the infected hepatocytes. Regulus has presented data evaluating RG-101 for in vitro and in vivo potency, pharmacokinetic/pharmacodynamics, toxicology and safety pharmacology and inhibition of HCV replication. Regulus has also tested RG-101 for efficacy in a human chimeric liver mouse model infected with HCV genotypes 1a and 3a. In this model, up to a 2 log reduction in HCV viral load titer was observed in both genotypes after a single dose of RG-101. The duration of action observed for RG-101 supports the potential for a once-a-month dosing regimen. To date, RG-101 has demonstrated an excellent preclinical safety profile and has been well tolerated.

About Regulus

Regulus Therapeutics Inc. (NASDAQ:RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus is uniquely positioned to leverage a mature therapeutic platform that harnesses the oligonucleotide drug discovery and development expertise of Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc., which founded the company. Regulus has a well-balanced microRNA therapeutic pipeline entering clinical development, an emerging microRNA biomarkers platform to support its therapeutic programs, and a rich intellectual property estate to retain its leadership in the microRNA field. Regulus intends to focus its proprietary efforts on developing microRNA therapeutics for oncology indications and orphan diseases and is currently advancing several programs toward clinical development in oncology, fibrosis and metabolic diseases. Specifically, Regulus is developing RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport Syndrome, a life-threatening kidney disease driven by genetic mutations with no approved therapy, and RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of chronic hepatitis C virus infection. Regulus’ commitment to innovation and its leadership in the microRNA field have enabled the formation of strategic alliances with AstraZeneca, GlaxoSmithKline and Sanofi. In addition, the Company has established Regulus microMarkers(TM), a research and development division focused on identifying microRNAs as biomarkers of human disease, which is designed to support its therapeutic pipeline, collaborators and strategic partners.

For more information, please visit http://www.regulusrx.com.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with Regulus’ expectations regarding future therapeutic and commercial potential of Regulus’ business plans, including the belief that RG-101 is the best-in-class anti-HCV host factor agent and its expectations regarding future clinical studies, and technologies and intellectual property related to microRNA therapeutics being discovered and developed by Regulus. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus’ current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Regulus’ programs are described in additional detail in Regulus’ SEC filings. All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

SOURCE Regulus Therapeutics Inc.

/CONTACT: Amy Conrad, Director, Investor Relations and Corporate Communications, aconrad@regulusrx.com, 858-202-6321; Media: Liz Bryan, Spectrum Science, lbryan@spectrumscience.com, 202-955-6222 x2526

/Web site: http://www.regulusrx.com

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Nick Semuta
Guest
Nick Semuta
March 6, 2014 10:03 pm
Reply to  Nick Semuta

SP $10.57..MC $441M….Nasdaq Listed…..Bode well for Benitec’s near future.

Nick Semuta
Guest
Nick Semuta
March 6, 2014 10:27 pm

Doc : You had some interest back in post #75 re: RGLS. I doubt their treatment would be any cheaper than benitecs?

Nick Semuta
Guest
Nick Semuta
March 6, 2014 10:39 pm
Reply to  Nick Semuta

It’s obviously less elegant, being a one month treatment.

tanglewood
March 6, 2014 10:55 pm

Hi Booya (maybe you are Cramer) re your reply on March 5th to the original post #10 dated 1/8/2014 on Sangamo Biosciences Inc. (SGMO) and their zinc finger. It’s better to post as a comment rather than a reply since replies get placed with their respective comments and are difficult to find. I wouldn’t have found it, if I wasn’t re-reading this thread to develop a watch list. That stock was 13.65 on Jan 8, 2014 and now 2 months later, it is 22.96. Maybe Dr. KSS can comment on this stock. It is just mind-boggling the number of biotech stocks that have taken off in the past 6 months.

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tanglewood
March 6, 2014 11:08 pm
Reply to  tanglewood

Sorry Dr KSS, you already commented on Sangamo at #194. I hadn’t refreshed my screen.

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jamespaul108
jamespaul108
March 6, 2014 11:23 pm

There was a fascinating article on nicotine in the March 2014 issue of Discover magazine. “The first hint of nicotine’s curious benefits came from a study published in 1996 by Harold Kahn…At any age, smokers were 11 times as likely to have died of lung cancer as nonsmokers…But…one oddball jumped out: Death due to Parkinson’s disease occurred at least three times as often in nonsmokers as in smokers.” “Nicotine has separate mechanisms by which it may protect brain cells, aside from its influence on dopamine…One of the functions of nicotinic receptors it to moderate the entry of calcium into cells. The presence of nicotine increases the amount of intracellular calcium, which appears to improve cellular survival.” “The neuroprotective effects of nicotine were studied in a randomized clinical trial of 67 subjects in the early stages of Alzheimer’s disease…The results found ‘significant nicotine-associated improvements in attention, memory and psychomotor speed’…”
The article also says it’s hard to get lab animals hooked on nicotine alone, although giving rats nicotine with “five other chemicals found in tobacco…significantly increased rats’ … self-administration of the mix”.

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tanglewood
March 6, 2014 11:28 pm

I would love to hear Dr KSS’s opinion on the very controversial and volatile Questcor Pharmaceuticals (QCOR). Is it a ruse?

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Rosenmeyer
Rosenmeyer
March 6, 2014 11:55 pm

thank you dr. kss & everybody else-a lot of honest & diligent work are contained in this thread. As far as Benitec goes-it is good to see the fever pitch for the stock steady-things imo got way too euphoric -i have a large position for me-but I realize I could lose most of my investment or I could see excellent appreciation-but the KEY is it has not been tested in humans (although it may have already been dosed in one patient) we have to be very patient -not obsess & simply wait for news (I know that is difficult)-I love the risk/reward here but I am treating my stock like it is a call-which COULD multiply substantially or lose most of its value- all other views are welcome

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davidbelan
davidbelan
March 7, 2014 6:28 am

I thank you Dr Karmaswimswami very much for (as always) the thorough explanation about Biotron (BITRF). I tried to grab some more Benitec shares yesterday but due to my working the night shift at a Monoclonal Antibody manufacturer (Downstream Lead Manufacturing Tech at MedImmune (AZ Biologics) in Maryland) I sometimes can’t pay enough attention to my investment options during the day when I need to be sleeping… I was also waiting for a couple of other trades to clear so that I had the available cash funds in the trading account to buy a slightly more significant (at least to my account) volume of shares.

Speaking of working at MedImmune (now AZ Biologics), if there are any set it and forget it very long term investors out there, buying shares of AZN or Merck on a dip would be a good long term investment. AZ Biologics, through their MedImmune acquisition and other strategic acquisitions, has positioned itself to have several BLA’s per year through the year 2020 and beyond. The shares of AZN have already appreciated about 25% in the past 6 months but I feel that this is just the tip of the iceberg as far as their Biologics arm. They are also currently partnered with Merck as part of their Trusted Partner Network and are making Merck’s (FDA breakthrough designation) drug Lambrolizumab and expected to make approximately 40 full scale batches this year alone due to the wide application success that the drug is showing in trials. It truly looks like an amazing drug for a great deal of different cancers. I am very proud to be able to work on such an amazing project.

I know you have covered INOVIO slightly here and there in this feed but I was wondering what you think about near term share value appreciation assuming that their Lead drug candidate shows positive results. I know that their trials are showing great promise for widespread application due to the nature of the synthetic DNA vaccine approach they are pursuing and I know that their pivotal trial is due to conclude on April 1, 2014 (hopefully we investors are not the fools)… The reason I ask is that I recently purchased several Aug 2014 $5.00 Call Options for INO and I was wondering what you thought of them overall, and in the short term specifically.

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roblites
roblites
March 7, 2014 9:19 am

RNN down pre market. $1.30. No news I can find.

rodj1201
rodj1201
March 7, 2014 9:19 am
KennyG
Guest
KennyG
March 7, 2014 9:36 am
Reply to  rodj1201

Unfortunately AF has a huge following and his opinion often makes or breaks a stock. Personally I am not a fan of his or anything associated with Cramer and company.

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$$ViaTheHelix
$$ViaTheHelix
March 7, 2014 9:43 am
Reply to  rodj1201

Essentially says Rexahn is like kissing your sister -a second time!

greenfire67
Irregular
March 7, 2014 10:05 am

ATTN: Linda Kulow, Joseph Rotondi, Glen Newberry
NVLX has been discussed several times here. Ctrl+F – then type nvlx. Hit next to find the next post. #239/40/41, 860/64/68/73
It’s a pumper. I’d like to add that they use a synthetic form of thc, cannibidol, which is extremely toxic, to the point that it will kill you, unlike the natural form of thc. I believe this is due to the synthetic form is a double mirror of itself, and has no natural properties and thus toxic. The natural form binds to cannibidoid receptors, as the synthetic form does not.

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jamespaul108
jamespaul108
March 7, 2014 10:56 am
Reply to  greenfire67

Cannabidiol (CBD) is naturally produced by cannabis plants and is said to have some medicinal properties according to wikipedia. It is non-psychoactive. http://en.wikipedia.org/wiki/Cannabidiol

However, stay away from Nuvilex – see Dr. KSS’s comments.

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greenfire67
Irregular
March 7, 2014 12:19 pm
Reply to  jamespaul108

James,
There are several cannibidoids in cannabis. THC, CBC, CBD, CBG’S, ect, ect.
I believe all studies are using the synthetic form, which is extremely toxic. People have died from using it. (used for pain and siezure management) Again, it is a double mirror of the natural form, meaning you can overdose quite easily.
If a legitimate co. started a trial using the natural form, (verifiable) then I would look further into such a study. Until then, I’m out.

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jamespaul108
jamespaul108
March 7, 2014 12:48 pm
Reply to  greenfire67

I see. I agree, I’m staying far away from NVLX.

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ockrazor
ockrazor
March 7, 2014 10:16 am

DMPI keeps on keeping on. I have asked before and I will ask again as the decision is huge has anyone look at EXAS. Any thoughts out there? Dr. KSS any thoughts. This is a major decision.

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jlw3262
Member
jlw3262
March 7, 2014 11:32 am

Regarding Palatin PTN- I have followed very carefully the discussion of this drug for female sexual disfunction. I really want it to work out ( for a variety of reasons) but I have one major concern that I am having a hard time getting over. As I understand it, the delivery method is via a shot- with a needle- not orally (like viagra, et al). And this needs to be done before every use. It just strikes me as a limiting factor. Granted, I’m not the target market; but it is hard for me to imagine millions of women giving themselves shots before dates in order to feel more amarous. Am I just missing something? Again, I would love to believe. And it sounds like a neat technology for a real problem. But the delivery method and the acute activity of the drug just seem limiting. Maybe it’s just my obvious ignorance. Can some of you with more knowledge or insight please weigh in?

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$$ViaTheHelix
$$ViaTheHelix
March 7, 2014 5:16 pm
Reply to  jlw3262

I too am confused. If the self dosing in women is to be via injection that seems to be a non-starter for some late-term sex. Is subcutaneous the only means of administering bremelanotide being tested? I understand absorption being slower, but how slow is it when you don’t want to inject yourself?
Thanks

bravorob24
bravorob24
March 7, 2014 5:31 pm
Reply to  jlw3262

I can’t say I have any more knowledge on the subject but based on the information I have been able to gather I have reached the same conclusion you have, Larry. Due to increased blood pressure concerns when administered intranasally it will be administered subcutaneously using what they call an “autoinjector”. It does concern me, given the stigma of direct injection, whether painless or not. Below is a related announcement from Palatin last year.

October 28, 2013 – Palatin Technologies, Inc. (NYSE MKT: PTN) today announced the successful completion of a clinical trial designed to demonstrate equivalence of subcutaneously administered bremelanotide via autoinjector compared to pre-filled syringe administration. Bioequivalence was achieved in this clinical trial. Palatin used pre-filled syringes in its Phase 2 clinical trials, but will use the single-dose disposable autoinjector in its planned Phase 3 clinical trials and for commercialization.

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bwd1up
bwd1up
March 7, 2014 12:13 pm

i normally would not bother anyone with an email teaser, but i’m betting the Team here will know this company she is teasing (Hilary Kramer). I only pasted some info that may help determine the company. Also, she says SP is 3.55.

Stage Set for Massive Run-Up:
• Shares up 270.6% in last 12 months… yet still ‘deep value priced’ at only $3.55 per share
• Phase 1 (complete) => Lead vaccine displays potentially revolutionary ‘shutdown effect’ on spread of cervical cancer. Of 18 patients tested in phase 1… all 18 showed an immune response!
• Phase 2 (results out mid-2014) => Optimism rife for results of larger study. Positive news could have a major impact on the stock price.
Friend, I don’t often lose my calm reserve. But know what I think? We could be staring at the long-awaited ‘miracle cure’ for at least some forms of cancer.
WHAT’S COVERED?
The Company’s clinical programs include cervical dysplasia (therapeutic), avian influenza (preventive), prostate cancer (therapeutic), leukemia (therapeutic), hepatitis C virus (HCV) and HIV vaccines.
Good news for everyone… but especially for savvy investors like us.
First, because our Pennsylvania-based drug company has the exclusive patent

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greenfire67
Irregular
March 7, 2014 12:24 pm
Reply to  bwd1up

Bradley,
Lost my butt following Hillary. Just another pumper letter.

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Subramania Kaushik
Subramania Kaushik
March 7, 2014 12:19 pm

Bradley, I believe your friend is talking about INO!

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KennyG
Guest
KennyG
March 7, 2014 12:32 pm

That is correct…this is INO

David B
Guest
March 7, 2014 12:51 pm

Would love to see a KSS and Adam Feuerstein face off on CNBC over Rexahn : )
Very doubtful though that Kramer et al. would have the cajones to allow that to happen. They don’t want their windbag to be exposed.

Lou L
Irregular
Lou L
March 7, 2014 1:19 pm

Added last night more Benitec in BLT from Aux Exchange. Long in BNIKF already. It took 6 partials to fill the order : 5382+688+744+1519+889+778=10,000. Here in the U.S., lots are usually in multiple of 100’s. Odd shares are usually from employees or insiders acquired via fix $$/disposed with share deduction for tax withheld. May be they traded differently Down Under? Can anyone help?

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Lou L
Irregular
Lou L
March 7, 2014 1:21 pm

Dr. Karma, can you comment on InterMune(ITMN) again? Reuters today says “ITMN is attracting takeover interest from several pharmaceutical companies, according to people familiar with the matter”. I have large position in May 30 put.
http://www.reuters.com/article/2014/03/07/us-intermune-deal-idUSBREA2617G20140307?type=companyNews

karmaswimswami
March 7, 2014 5:31 pm
Reply to  Lou L

Lou: it’s hard to know really. The only reason anyone would want the company is to get pirfenidone, as the company is not developing anything else. This is a bloated overpriced way to get at a drug that is AT BEST a mediocre one. Pirfenidone is an an immunosuppressive that may slightly slow down the process of scarring in IPF patients with advanced disease….and cause them to be more likely to get infections in the process. It does not change the outcomes—-death or lung transplant. And this whole area is very vulnerable because someone is going to wise up, devise a way to give anti CTGF siRNA into lungs and fix IPF. The market has behaved very irrationally about Intermune for a long time, and may continue to do so. It is the kind of thing I would hate to be in because I would not be able to sleep at night. Questionable company, very questionable product…AND it is NOT FDA approved (yet, if ever). There are definitely going to be objections to approving it. The drug just causes a dead cat bounce effect in IPF patients.

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Lou L
Irregular
Lou L
March 7, 2014 9:09 pm
Reply to  karmaswimswami

Dr. Karma, thanks for your comment. ITMN has unusual high institutions ownership – 97.5%. Yet majority of the independent analysts are bearish on the stock. My put position was in black prior to today’s run up. I am hoping when institutions start to sell, it will tank.

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