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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.

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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
February 11, 2014 10:21 am

To KindergardenInvestor, and others possibly interested in Gi Dynamics: Yes I have definitely been forced to rethink GIDYL. I have no position in it now, but may take one. My prior objections were, one, that it is an American company that first got publicly traded in Australia. Which makes little sense, as Down Under is hardly an excellent way to access capital markets. The Australian market is less efficient than the US one, less analyzed and covered, and is a place of relative investor skepticism and non-intellectualism when it comes to biotech. As a gastroenterologist, GID’s method disturbed me. It is the endoscopic placement of a plastic sleeve in the duodenum. The sleeve extends 60 cm caudad into the jejunum. My concerns were that this could become detached, could lead to bacterial overgrowth and lead to permanent villous atrophy that would leave patients permanently with a pseudo-short bowel syndrome.

Getting information to my degree of satisfaction on this has not been easy. The method is approved and widely in use in the EU, South America and Australia for obesity and for type 2 DM brought on by obesity. Why it has been so laggard and lolligagging about securing FDA approval is problematic to sort out, I have found. I don’t see that the FDA has turned it down. Studies are ongoing, in particular what appears to be a phase III, comparing the method with sham endoscopy for weight loss, for 12 months study duration. Centers are still enrolling for this study, which means the time frame for results is displaced.

The method is called EndoBarrier. A patient is sedated and scoped. I guidewire is passed through the channel of the endoscope and then advanced into the jejunum. The scope is withdrawn, and over the guidewire the device is deployed. The scope is then placed again in the stomach, and under observation, the device is deployed in the duodenal bulb. Most people are familiar with how bottles of champagne usually have a wire cage around the cork. EndoBarrier has this, an expandable wire cage place in the duodenal bulb right behind the pylorus, and this holds it in place. I do not feel it is acting literally as a barrier to nutrient absorption. It is but 60 cm long, and believe me the small intestine has many more feet in it than that. Instead, what I think is happening is that bile is secreted out the ampulla outside the sleeve, while food stays inside the sleeve. Bile is a detergent that solubilizes fat for absorption. With the device, bile and ingesta do not mix til distal jejunum and so fat absorption is much less efficient.

Patients do lose weight on it. Occasionally some patients seem to have mild upper GI pain with the device in place, but why this is cannot easily be rationalized. The device can easily be removed by sedating the patient, advancing a scope into the stomach, placing a biopsy forcep on the wire cage, and then just pulling scope and forceps and Endobarrier out in one fell swoop up through the mouth.

Realistically I do think it is likely to be approved. Other countries where it is in use are monitoring it, and there just do not appear to be safety issues with it. I earnestly do not understand the big sell-off in shares yesterday. Does anyone out there know anyone, here or abroad, who has had Endobarrier placed? For clinical trials it is being placed at quite a few US centers. Based on phase II, it is quite efficacious for weight loss, and since the study is comparing it with endoscopy in which no device is placed (patients will not know whether they have it), it is likely to make the device look rather good. Outcomes in which patients have easily lost 20 per cent of body mass are typical. Since it is removable, requires no general anesthesia or surgery, and does work, it could easily catch on. Obesity is not going away as regards prevalence. No word on cost of the procedure or device, but it will easily trump lap banding, gastric stapling and formal surgical procedures. It will clearly NOT require specialized training for GI doctors to deploy, as the techniques used are part of basic repertoire, so there should be easy adaptability. I feel people are likely to embrace it.

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roblites
roblites
February 11, 2014 10:22 am

Bot some ZIOP. A lot of buzz lately on BIO types Twitters. But that’s nothing. Look at SEC 8-K Feb 10 on site or news item on other sites. Corporate slide presentation. Very impressive. A totally loaded gun for the cancer bundle made up of many parts.

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karmaswimswami
February 11, 2014 10:37 am

In addition to the closely-watched train of CHTP, the FDA is having another big meeting this week that has not been announced. Its advisory committee regarding NSAIDS will meet to decide what to do about NSAID warnings over vascular event risks. NSAIDS can inhibit both cyclooxygenase 1 and 2, and there is thus concern that they can have pro-thrombotic actions. No tea leaves have been read about whether they are likely to continue wanting warnings for NSAID such as Aleve, but if they do, the bottom line is that it may cast aspersions over that class and potentially feed favorably into the hand of QRx Pharma for MoxDuo approval.

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arch1
February 11, 2014 12:48 pm

Dr KSS ; as a follow up to PTN have you any thoughts on Sprout pharma ‘Flibanserin’ for HSDD?

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karmaswimswami
February 11, 2014 1:35 pm

Frank: Thanks for asking about flibanserin. My honest view is that Boehringer was wise to divest itself of that product and that Sprout is foolish to waste the $20 million they just raised chasing it. The agent is a serotonin receptor modulator, agonistic at some, antagonistic at others. As a general theme, augmented serotonin activity in the female central nervous system works against sexual arousal, but to me it is a cognitive error to infer the contrapositive of that, that serotonin antagonism may be beneficial. The FDA is seemingly asking Sprout to go back and do phase I again!

In mulling a lot of these agents we have talked about the last 2-3 days, (1)I really think bremelanotide is the best of the field for FSD, (2) it seems reasonable that droxidopa might be trialled for FSD if nothing else surfaces, and (3) if droxidopa is not approved for neurogenic orthostasis, I have to say I wonder if a sustained delivery form of bremelanotide, such as PEG-bremelanotide, might work for that! It raises blood pressure by an incompletely understood central mechanism.

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karmaswimswami
February 11, 2014 1:54 pm
Reply to  karmaswimswami

Frank: You might want see this link:
http://www.aol.com/article/2014/02/06/new-shot-helps-women-achieve-orgasms/20824692/

This physician has promulgated this with no study or control, and without any theoretical basis for how it should work. And charging cash (1500 bucks!) for it. It is pure 100 per cent placebo and this guy deserves jail and FDA sanctions.

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arch1
February 11, 2014 4:20 pm
Reply to  karmaswimswami

DR KSS Thankyou for info & link. lol Reminds me of how kids like to pick at scabs (unexplored why) Connection?

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Lou L
Irregular
Lou L
February 11, 2014 2:22 pm
biocqr
biocqr
February 11, 2014 2:36 pm

I would be curious to hear KSS’s take on NNVC…

NanoViricides, Inc. (www.nanoviricides.com) is a development stage company that is creating special purpose nanomaterials for antiviral therapy. The Company’s novel nanoviricide® class of drug candidates are designed to specifically attack enveloped virus particles and to dismantle them. The Company is developing drugs against a number of viral diseases including H1N1 swine flu, H5N1 bird flu, seasonal Influenza, HIV, oral and genital Herpes, viral diseases of the eye including EKC and herpes keratitis, Hepatitis C, Rabies, Dengue fever, and Ebola virus, among others.

Versatile Platform Technology
A nanoviricide is created by chemically attaching a virus-binding ligand, derived from the binding site of the virus on its cell surface receptor, to a nanomicelle flexible polymer. This binding site does not change significantly when a virus mutates
Tailor-made design and selection of (1) the virus-binding ligand; and (2) the backbone “nanomicelle”, separately, allows us to rapidly optimize drug candidates (a) against a number of viruses; (b) for desired pharmacokinetic characteristics (e.g. sustained effect); and (c) for different routes of administration. This versatility is unmatched in the Industry.
Virus-specific nanoviricides have been created against important viruses such as HIV, Influenza and Bird Flu by choosing highly virus-specific ligands
Broad-spectrum nanoviricides have been created that can bind to possibly as many as 90-95% of known viruses. The Company is developing broad-spectrum nanoviricides to combat several neglected tropical diseases, such as Dengue, Rabies, and Ebola/Marburg. This is similar to antibiotics such as penicillin against bacteria that exploit a feature common to all bacteria.
Our ADIF Technology enables the creation of a virus-specific accurate drug in the field. This enables a rapid, effective response to novel and emerging infectious diseases and bioterrorism agents. We have successfully demonstrated this technology

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KennyG
Guest
KennyG
February 11, 2014 2:57 pm
Reply to  biocqr

“The Company is developing drugs against a number of viral diseases including H1N1 swine flu, H5N1 bird flu, seasonal Influenza, HIV, oral and genital Herpes, viral diseases of the eye including EKC and herpes keratitis, Hepatitis C, Rabies, Dengue fever, and Ebola virus, among others.” WOW!! Is there anything that they claim they can’t cure. Now I know absolutely nothing about this company…but really? A little suspect I would think. But I defer to a more acknowledged input.

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George
Member
George
February 11, 2014 3:18 pm
Reply to  biocqr

I see all kinds of problems here. They are basically making little balls of receptors for the viruses to attach too. I am making the assumption that binding changes the ‘ball’ in such a way to destroy that particle. Given that works:
1. you need to deliver the ‘balls’ to the virus: ie the blood or the lung. That is IV or inhaled for many of the listed viruses. You either need to be hooked up to an IV (the best of convenience) or take an inhaler. Inhaled drugs have many issues from a technical and safety perspective (Exubera (inhaled insulin)= increased lung cancer). Their flu program is oral and injectable, basically delivering to where the virus isn’t. You need a high blood concentration to get much into the lung, even if you ignore first pass metabolism from the oral (it goes to the liver first before it gets into the general bloodstream)
2. This doesnt stop viral replication in the cells, where you really need to stop it. The cells will make more.
3. you will need ALOT of these particles in the blood (hence IV more than injectable). Yes they are small, but if you have millions of viruses per mL of blood and 3-4 L of blood… you do the math.

If it works, it could be for acute treatment along (limited population) with other treatments, not a standalone. Perhaps pre or post exposure prophylaxis (someone in your house is sick or some sick person just infected you), which is very costly to develop.

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karmaswimswami
February 11, 2014 3:23 pm

It is frankly difficult for me to get interested in NNVC (Nanoviricides). Its agents are sequestrants, partitioners. Viruses do their dirty work inside cells. NNVC’s nanomoieties compartmentalize them when they are extracellular, such as post-lysis. It will take much clinical data to persuade me that this is a helpful approach. The agents do nothing to interfere intracellularly with virus replication, and that is what the virology world is crying out for. Basically NNVC’s compounds are neuraminidase inhibitors with college educations: they do nothing antiviral except sumping viruses away from cells.

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arch1
February 11, 2014 4:34 pm
Reply to  karmaswimswami

DR KSS I thought NNVC had great promise in2009 Bird flu, Ebola, etc. Bought in @ $3 or so, rode rockrt up & right back down ,sideways till 2013 resumed slide down,!st .turn I sold. Slight loss for 4 years. Today looks like resume of old habits,

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karmaswimswami
February 11, 2014 3:27 pm

The FDA has just announced it is maintaining cardiovascular warnings for all NSAIDs, including naproxen (Aleve). All of them inhibit COXes 1 and 2, and the feeling has been that the COX 1 inhibition, by blunting prostacyclin synthesis, is prothrombotic. (And we all know how the COX-2 inhibitors, supposedly oblivious to COX 1, turned out in this regard.) This has moved QRXPY up today. If the utility of NSAIDs for pain is to be limited, opioids will of necessity take a greater role in analgesia.

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KennyG
Guest
KennyG
February 11, 2014 4:04 pm
Reply to  karmaswimswami

Dr. K – – how does this impact a drug like Celebrex which is a Cox2 inhibitor? Also, what is your opinion on Celebrex as a drug of choice for arthritis pain?

David B.
Member
February 11, 2014 6:15 pm
Reply to  karmaswimswami

The addictive properties of opioids bother me a great deal. There are many who get addicted and it wreaks havoc in the life of the individual and this person’s family. What starts out as a seemingly harmless RX from a physician too often ends up starting a vicious cycle of dependence. Opioid withdrawal is terrible, difficult and often unsuccessful. In the most extreme cases this leads to heroin use. Even very intelligent persons can get sucked in–e.g. Philip Seymor Hoffman. Any thoughts KSS?

karmaswimswami
February 11, 2014 3:48 pm

The CDC released a rather interesting report today that West Nile virus, which causes a devastating encephalitis, has cost the US nearly $800 million since it emerged here in 1999. Sanofi has been developing a vaccine, but still in phase II. A small company called MacroGenics had an agent in trials for it. The trial was stopped for failure to enroll patients. NIAID did have IV immunoglobulin in trials for it, but no published results.

Macrogenic (MGNX) went public last fall. It has had the usual biotech growth curve of nearly all these companies, many of which have behaved like first derivatives or high beta versions of the NASDAQ Biotech index. For a company with no products approved, it has a high market cap. Is anybody in this one?

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arch1
February 11, 2014 5:11 pm
Reply to  karmaswimswami

DR KSS ; Altho I live in an isolated area West Nile arrived a few years ago & played havoc with the horse population. A vaccine is now available for horses that seems effective prevention. This area is home to Appaloosa horse & is ancestral home of Nez Perce nation, town of Joseph OR named after Chief Joseph who lived here. Sorry I don’t know name of vaccine maker.

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biocqr
biocqr
February 11, 2014 4:01 pm

PTN/bremalanotide… KSS, Siva.. care to comment?
Adam Feuerstein commented negatively on the P2B trial results released in March 2013…

“Did you see the big red flag waving? Count the “Ns” representing the number of patients in this analysis. 91+85+75+73=324 patients, which is the mITT patient population, with three patients missing. Also notice that even at the highest bremalanotide dose, the mean change in the number of sexual satisfying events is a paltry 0.6, net the placebo effect.

Palatin designed the study to measure bremalanotide’s effect on sexually satisfying events after three months of at-home dosing. We should have been provided data on the 397 patients enrolled and randomized, or the 287 patients who completed the study. What Palatin gives us, instead, is a post-hoc concoction of patients treated with bremalanotide or placebo for only one month.

Why? Palatin doesn’t say, but it’s safe to assume the analysis of the study’s pre-specified primary endpoint was negative. Palatin tries to pass off an irrelevant efficacy analysis as the real deal, but of course, we’re all smarter than that.

Bremalanotide needs to be injected approximately 45 minutes before a woman wants to have sex. That’s not exactly the hottest form of foreplay, which helps explain why so many women chose not to complete three months of at-home dosing.

Palatin intends to move bremalanotide into phase III studies. I’m not optimistic for a positive outcome.”

PTN commentary starts at bottom of page and continues to next page…
http://www.thestreet.com/story/11932328/1/biotech-stock-mailbag-amarin-mannkind-palatin.html

P2B trial poster…
http://www.palatin.com/pdfs/bremelanotide.pdf

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roblites
roblites
February 11, 2014 4:52 pm

I have commented on Innate Parma in above posts. It’s up 2.24 today to 16.49.
IPHYF. It’s being talked about on my Twitter scan. PD-1s is a current interest.
Did lots of DD this weekend on Patrys (KSS mention) – OTC/PATZF….ASZ/PAB. These people are working with ONYX, part of the Amgen family, that is paying for current trial. Sounds good to me.

karmaswimswami
February 11, 2014 5:43 pm

Biocqr: I like Feuerstein but do not share his assessment. I picked apart how this study was done. The SSE score is not what matters, and is prone to horrible subjective recall distortion. Only one number for this trial matters: the FSFI. There were in fact many women who completed the trial to 3 months, and all that matters is placebo versus 1.75 mg sq of bremelanotide. My single biggest trial question is why so many were enrolled but not randomized. That differential here was huge. There was some placebo effect as one would expect for something as elliptical to define and measure as female sexuality. But the FSFI score difference achieved statistical significance, and there was a tendency to accrue effect with study participation duration. When one zeroes in on what aspects of the FSFI were relevant to this study, the arousal and in-the-mood aspects, which is where PTN’s agent purports to act, the differences were even more significant. The agent is not a fix for the total spectrum of FSD, but does seem to act as aphrodisiacal. It gets patients to think about having sexual encounters, and no other agent does.

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karmaswimswami
February 11, 2014 5:53 pm

Roblites: Insofar as I can tell, PATZF hasn’t traded now in some time. Something is very wrong. When I last sussed out what was going on, they were waiting, on data to be presented for their anti-GRP78 mIgM at ASH in December. I have seen no follow-up at all on that, which cannot be good. The method is brilliant from a basic science perspective, but may not actually be working in clinical trials. I need to poke around the PubMed and clintrials databases. At Patrys’s website, it was updated in January, but contains no comment about data presented in December at ASH. Cannot be good. Shares still trade on the ASX, but are fairly inert. It is like an nonresponsive patient: comatose or brain dead? Big difference.

karmaswimswami
February 11, 2014 6:48 pm

David B: Believe me I share your concerns. Present or former addiction is an issue with most of my patients, as that is how they got liver disease. There certainly are patients that do require opioids chronically, but I was among the first to put in force what really are now considered standards of care for such patients. I began making all those needing them sign opioid use contracts with stipulations about “lost” prescriptions, about regular drug screening, about how the results of those drug screenings would be enforced, about how even the slightest rude or threatening comment to staff members would result in termination. I have been threatened by opioid using patients (in two cases barricaded in exam rooms by them), and have to go through my office’s parking lot to check for drug paraphernalia. Keep in mind that there is more than one way that a drug screen in my office can get a patient terminated from care: it has to show the drug specifically that they are on. If that isn’t present, I show them the door. It cannot contain any other controlled substance of any kind. And it cannot contain any illicit substance of any kind. If it does, they are fired on the spot. I will not prescribe opioids for patients and have them smoking pot. Pot is mind-altering and creates a hazard and can contribute to death from opioids. I also make patients bring their medication bottles. If pill counts do not jive with expectations, they are fired. Most states now also have databases online accessible by physicians regarding ALL patients getting controlled substances. If a patient shows up getting something from someone else that is controlled, they are fired. All of this may sound police state like, but it is necessary for safety and good patient care. When you enforce these rules, word gets around and riffraff stays away. There is now a website called opiophile.org where patients blog about what doctors give out what and how “easy” those doctors are. if a doctor is tough, that gets noted on opiophile. One of my clerical staff has a husband who is a police officer. He comes to see her at lunch. Any patient that gets up and heads out the door when he shows up gets noted and gets a termination letter.

As regards MoxDuo, it does have tamper-resistant tech in it. The pills are not crushable with mortar and pestle.

We are in fact seeing a huge problem with resurgent opioid use in the US, no question. The main cause appears to be cheap heroin. I am told that Hamid Karzai’s policies in Afghanistan (Karzai is widely regarded as a heroin addict) have led to massive Afghani supplies. Philip Seymour Hoffman is said to have had a 10-bag per day habit. Before he died, he had gotten $1000 from an ATM, and was found with 5 empty bags and 65 full bags. The stuff is really cheap. IV shooting is at levels not seen since the 70’s, and a huge uptick in new cases of HIV and HCV are what the US has to show for it. Predicting which opioid-prescribed patients will become people with abuse potential, and discerning which of them have authentic pain is a difficult task. Certainly, fMRI imaging will come to a point where it can be routinely invoked to pick out sincere sufferers from poseurs with “back pain.”

The thing I think is most difficult for most people to understand about addiction is this. The really bad addicts like Hoffman are no longer doing it to get high. The dyed-in-the-wool drinker does not drink to get drunk. These people shoot up or drink exclusively to avoid the negatives associated with NOT using. The brain always seeks a balance between neuroexcitation and neurodepression. When one chronically ingests downers, the brain copes with that by churning out large levels of glutamate, an excitatory neurotransmitter. Glutamate storm, the tremors, sweats and seizures one has when glutamate action is unopposed, is what addicts are trying to avoid. When we understand that as a society, I feel that drug policy will become more rational. This is frankly something the moralists and the TV pseuds like Dr. Drew Pinsky do not get. These people really believe that putting a moral spin on things, and subjected patients to Ludovigo-like maneuvers (remember the scene in “Clockwork Orange” when the doctor says to Alex: “Come now my boy, you really MUST leave it to us!). When we take this out of the hands of the execrable 12-steppers and view it as a molecular issue that needs molecular treatment, we will all of us be better off. There happens to be a profoundly effective drug that helps people beat ALL addictions. it is called Campral, acamprosate. It shuts down glutamate release. How many of the detoxers and shrinks and people like Dr. Drew advocate it? NONE. They have too much to lose by fixing the problem! What Lindsay Lohan needs is to be run through a car wash, put on Campral three times a day with supervision, and taken out of the hands of the fixers. She would get her life back and return to being a stellar actress. We once viewed TB and AIDS as metaphorical illnesses too. That didn’t help, and it doesn’t help addicts either.

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arch1
February 11, 2014 10:32 pm
Reply to  karmaswimswami

DR KSS I agree & thank you for your standards of practice. I hear that U.N. showed Afghanistan farmers how to grow more wheat , where-upon finding there is more money in not growing wheat have half million acres of poppies under cultivation. I live in eco-topia state where much of population is on “medical cannabis” & many have portable meth labs altho being squeezed out by imported stuff coming up from south. Genie seems to be out of bottle & not want to go back . I think you have far better solution than law proliferation,’ just to be ignored.

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David B
Guest
February 11, 2014 11:33 pm
Reply to  karmaswimswami

Thank you KSS; I work in the social work field and work with addicts on a regular basis. Thank you for your diligence re. patients and prescriptions. Addiction needs to be less criminalized and more treatment oriented–this would save the U.S taxpayers a boatload of money, but that’s a discussion for another site! I wish there were some other great alternatives to opioids for severe pain as they are quite effective. I think your opiod stock is likely a good investment, but I don’t think I have the heart to invest in it–I’d be too conflicted.

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karmaswimswami
February 11, 2014 7:10 pm

To Brian Jess: i am not ignoring your stocks. Just haven’t gotten to them yet.

I managed to obtain a master list of all the talks given the last two days at the Waldorf biotech symposium. Does anyone know if these as a group were webcast? I checked several webcast providers like veracast and saw no mention. Perhaps a decision was made company by company. Benitec BLT has opened and pushed right on up to A$0.995 and then retreated because that is a psychological barrier. Some retreat is good at this point. It hasn’t encountered much resistance at all on its way up. It needs some to get real momentum to really move into new territory.

karmaswimswami
February 11, 2014 7:38 pm

Seems there was a brouhaha about NNVC set into motion by a disreputable post at Seeking Alpha. SA may have some worthy articles, but at its worst it can be a Sodom and Gomorrah. I recently lodged a formal complaint there about an article writer whose conclusions I strenuously but politely disagreed with, who then went totally rabid on me. They guy claims to be a practicing and experienced family practice doctor who rants every other paragraph about his “God given abilities.” He gave his full name and so I did some background. It took the guy 5 tries to get into medical school, whereupon he went to a really sub-par one, and took 6 years to finish (medical school is 4 years). He started an internship and, 4 months into it, was fired. SA, finger ever on the pulse of its editorial authenticity, will do nothing about his claims.

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David B
Guest
February 11, 2014 11:44 pm
Reply to  karmaswimswami

I love SA but one really has to separate the wheat from the chaff there. The conversations can get out of hand and some authors seem to be arrogant idiots like the one you reference. I have found several who are quite respectful and who research and write well. Two who I respect for their articles and for being responsive and respectful to their readers would be Long Term Bio and The Behavioral Economist. LTB just covers biotech while TBE covers biotech and several other sectors. I am positive that TBE would be 100% respectful–much like you are KSS. He or she has written articles on Stellar, Medifocus, Xencor and several others. On the microcaps, they go SA Pro after about 3 months so you can’t read them without paying quite a bit.

cat_bastet
February 12, 2014 12:29 am
Reply to  David B

I agree with David B re: LongTermBio on SA. But I either have something there set up wrong or don’t understand what ‘following’ means, since I ‘follow’ LTB but never receive any notice of his new articles. Not sure why.

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--MJ
--MJ
February 12, 2014 7:04 pm
Reply to  cat_bastet

I had the same problem, but if you log in to SA and go to your settings, click ‘Email Alerts’, then scroll down to the ‘Daily Emails’ section and check the box for ‘Authors Alerts’ you will receive notices for everyone you follow.

KindergardenInvestor
February 13, 2014 1:20 am
Reply to  --MJ

Thank you, MJ, that was what I was missing!

biocqr
biocqr
February 11, 2014 8:17 pm

KSS, this may be of interest. A few snips provided…
New MIT nanoparticles offer best-ever gene silencing, could help treat liver diseases.
http://web.mit.edu/newsoffice/2014/better-rna-interference-inspired-by-nature-0210.html

The new MIT particles, which encase short strands of RNA within a sphere of fatty molecules and proteins, silence target genes in the liver more efficiently than any previous delivery system, the researchers found in a study of mice.

“What we’re excited about is how it only takes a very small amount of RNA to cause gene knockdown in the whole liver. The effect is specific to the liver — we get no effect in other tissues where you don’t want it,” says Daniel Anderson, the Samuel A. Goldblith Associate Professor of Chemical Engineering and a member of MIT’s Koch Institute for Integrative Cancer Research.

The research team, which included scientists from Alnylam Pharmaceuticals, also found that the nanoparticles could powerfully silence genes in nonhuman primates. The technology has been licensed to a company for commercial development.

Like lipoprotein nanoparticles, the MIT team’s new lipopeptide particles are spheres whose outer membranes are composed of long chains with a fatty lipid tail that faces into the particle. In the new particles, the head of the chain, which faces outward, is an amino acid (the building blocks of proteins). Strands of siRNA are carried inside the sphere, surrounded by more lipopeptide molecules. Molecules of cholesterol embedded in the membrane and an outer coating of the polymer PEG help to stabilize the structure.

The researchers tuned the particles’ chemical properties, which determine their behavior, by varying the amino acids included in the particles. There are 21 amino acids found in multicellular organisms; the researchers created about 60 lipopeptide particles, each containing a different amino acid linked with one of three chemical groups — an acrylate, an aldehyde, or an epoxide. These groups also contribute to the particles’ behavior.

David Putnam, an associate professor of biomedical engineering at Cornell University, says he is impressed with the team’s approach to mimicking how the body transports fatty molecules with lipoprotein particles.

“They hijacked that machinery and made something that looks like the lipoprotein structures and will carry siRNA straight to the liver. They’re building on Mother Nature and making it as efficient as possible,” says Putnam, who was not part of the research team.

The researchers then tested the particles’ ability to shut off the gene for a blood clotting protein called Factor VII, which is produced in the liver by cells called hepatocytes. Measuring Factor VII levels in the bloodstream reveals how effective the siRNA silencing is.

In that initial screen, the most efficient particle contained the amino acid lysine linked to an epoxide, so the researchers created an additional 43 nanoparticles similar to that one, for further testing. The best of these compounds, known as cKK-E12, achieved gene silencing five times more efficiently than that achieved with any previous siRNA delivery vehicle.

In a separate experiment, the researchers delivered siRNA to block a tumor suppressor gene that is expressed in all body tissues. They found that siRNA delivery was very specific to the liver, which should minimize the risk of off-target side effects.

“That’s important because we don’t want the material to silence all the targets in the human body,” Dong says. “If we want to treat patients with liver disease, we only want to silence targets in the liver, not other cell types.”

In tests in nonhuman primates, the researchers found that the particles could effectively silence a gene called TTR (transthyretin), which has been implicated in diseases including senile systemic amyloidosis, familial amyloid polyneuropathy, and familial amyloid cardiomyopathy.

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newby3867
newby3867
February 11, 2014 8:56 pm

Dr KSS any thoughts on Ventrus Bioscience (VTUS) and their upcoming phase 3 clinical trials with VEN-307 for gastrointestinal disorders.Data is due sometime soon.Also after the sell off and the story on Seeking Alpha which effected CTIX negative,are you still a believer?Looks like a good entry point to me.Thanks,Glenn

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cat_bastet
February 12, 2014 12:23 am

Any opinions from the medical professionals here on this article
http://tinyurl.com/mz5cmvu
on FierceBiotech today? It’s a brief about the ‘future of electroceuticals’. Way above my pay grade but it sounded interesting. Here’s the first paragraph:
“Looking beyond the small-molecule drugs and biologic treatments that have dominated therapeutic development over the past generation, GlaxoSmithKline’s ($GSK) all-encompassing R&D department is trying to get a jump on the future of medicine, and research chief Moncef Slaoui is betting that there’s a great deal of promise in drug-mimicking electronics.”

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arch1
February 12, 2014 3:28 am
Reply to  cat_bastet

Kerry I have no special insight but in my opinion there is much to be done in field of electricity/light/magnetism gravity spectrum; for sake of simplicity lets call that wave energy. From an engineering standpoint the human organism is an electro-chemical engine. Much work has been done on chemical side with much left to do . Electro side was dis-credited by much quackery last century. Just now starting to look at role in treatment,such as blue light therapy.It is well known we are affected by wave-energy ie. melanoma from over exposure to ultra-violet radiation. There has been ongoing battle between linear & threshold radiation dosage & recent exposure of early day fraud by linear advocates. There is “J” curve for instance Where starting from zero exposure organism benefits from exposure for a while before point where injury can begin to be observed. Also true in toxicity exposure. ” What doesn’t kill me makes me stronger” has some validity it seems. All that said I think investment opportunity is far away.IMHO

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arch1
February 12, 2014 3:47 am
Reply to  arch1

As addition we know far more about light spectra frequency on plants & also gravity influence than we know about same on animals.. I have conjecture that magnetism is
somehow expression of zero frequency electric flow but can think of no science to prove it.
I am only simple self with much curiosity; advice do not take too seriously.

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