News and Comments

ADVENTRIX (ANX): A REVERSE SPLIT WITH SOME EXCITING NEWS.

  Wednesday, April 28, 2010

Despite the emergence of new biological treatments for cancer, Chemotherapy is still essential, and indispensable as a major treatment for malignancies. The adverse events caused by these chemicals were found to be acceptable as a risk versus the great reward of using chemotherapy. The technological evolution that took place in the past decade and the surfacing of state-of-the-art technologies such as nanotechnology, scientists believed possibilities exist now for improving the adverse effect profiles of these therapeutic molecules. Reformulating the existing products has become an increasingly common product lifecycle-management technique. We read that a 2004 report on the U.S. drug market from BCC, Inc. projected that reformulations would grow from 62% of the market in 2003 to 79% in 2008. Finding ways to modify and improve existing products’ side effect profiles has become feasible after being essential but unreachable goal.  More...

AMGEN (AMGN): Practicing Predictive Medicine Will Have Tremendous Benefit on Patients Health And On Healthcare Cost

  Monday, April 19, 2010

Practicing personalized medicine, or predictive medicine is no more a dream. It is real and it is performed at the genetic level. The breakthrough capability is made possible through the effective use of next-generation sequencing technologies provided by genomic firms that have constantly upgraded their gene analysis techniques and programs. Currently, millions of DNA sequences can be read simultaneously in a single experiment. What we are witnessing here is a quantum leap beyond the methods used since the sequencing of the first human genomes, i.e., twenty years ago.

The next-generation sequencing technologies enable the differential diagnosis of the same disease, for example colorectal cancer, based on the cancers’ genetic expressions, rather than on their morphologies. This capability enables the selection of effective treatments based on biomarkers analysis. The revolutionary practices will save patients from torture with ineffective drugs on a case by case basis, hence, save millions of dollars in unnecessary spending on experimentation with investigational and marketed drugs on thousands of patients.  

In the news, Amgen (AMGN) was the first to use the new technologies on colorectal cancer to enable using its drug Vectibix (panitumumab) only on colorectal cancers expected to respond to it. A new biomarker analysis of the pivotal Phase 3 "408" trial of Vectibix® plus best supportive care (BSC) compared to BSC alone used massively parallel, next-generation sequencing technology to investigate whether mutations in nine genes in colorectal cancer are predictive of response to Vectibix in metastatic colorectal cancer (mCRC). Highlighted results were presented at the opening press conference at the American Association for Cancer Research (AACR) 101st Annual Meeting 2010 in Washington, D.C.

Tumor samples from 288 patients, which had previously been analyzed for KRAS exon 2 mutations, were analyzed in this study for mutations in nine genes: KRAS (exon 3), NRAS, BRAF, PIK3CA, PTEN, AKT1, EGFR, beta-catenin (CINN1B) and TP53. All nine genes are either direct or indirect components of the EGFR signaling pathway. The study enabled scientists to learn that Vectibix improves progressive-free survival in patients with KRAS wild-type (WT) tumors and had no effect in patients with KRAS mutant tumors. Mutations in NRAS, another member of the RAS gene family, were associated with lack of response to Vectibix. Patients with both KRAS WT and NRAS WT tumors had improved progressive-free survival receiving Vectibix, compared with those receiving BSC. Further investigation in larger studies is required to determine the predictive value of BRAF mutations.

Commenting on the study, Marc Peeters, M.D., Ph.D., Department of Oncology, Antwerp University Hospital and the study's principal investigator said, "To our knowledge, this is the first time next-generation sequencing has been used to analyze tumor samples from a Phase 3 clinical trial and demonstrate how advancing technologies can be quickly applied to ongoing clinical research. The KRAS gene mutation is a well-established biomarker for a lack of response to anti-EGFR treatment and has played a pivotal role in the advancement of personalized medicine. We are excited to be taking another step forward in the advancement of additional biomarkers with the study results presented today. In addition to the excitement of this being among the first times this technology has been used in Phase 3 research, the superior sensitivity of next-generation sequencing revealed unexpected genotypic complexity in many patient tumors,”

It is known now that one hundred nine tumors have more than one mutant gene, and 20 had more than one mutation in a single gene. 

Results from studies performed over the last twenty-five years indicate that KRAS plays an important role in cell growth regulation. In mCRC, EGFR transmits signals through a set of intracellular proteins. Upon reaching the nucleus, these signals instruct the cancer cell to reproduce and metastasize, leading to cancer progression.  Anti-EGFR antibody therapies work by blocking the activation of EGFR, thereby inhibiting downstream events that lead to malignant signaling. However, it is hypothesized that in patients whose tumors harbor a mutated KRAS gene, the KRAS protein is always turned "on," regardless of whether the EGFR has been activated or therapeutically inhibited. KRAS mutations occur in approximately 40 – 50 percent of mCRC patients.

You know what this means? It means that we are giving treatments that inactivate EGFR to  patients who do not benefit from them. These patients amount to half of the cancer inflicted patients. Avoiding this practice promises less pain and torture for those patients and less money spent on futile treatments. In addition, other effective treatments could be pinpointed for those patients.

Again, we remind, the heroes’ behind this huge advancement in addition to Amgen, are the genomic firms. These firms are the backbone of the biotechnology and drug industries. Without them, no revolution in medical sciences can take place. Most of the genomic firms are scientifically sound and are evolving their technologies and kits and moving from research purposes to revolutionizing the medical practices from the diagnosis of diseases, to differential diagnosis of different types of the same diseases. Their sequencing and analysis capabilities  are making possible pinpointing effective therapeutics for each subtype of diseases.

We are no more knocking at the door. Trust us, we are already in.

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ILLUMINA (ILMN): Today’s News Is What We Want To Hear

  Thursday, April 08, 2010

Those who follow on the advancement of biotechnology achievements walk up every day looking for specific news about breakthrough treatments for cancer and chronic devastating diseases, not for just a modest improvement of drugs, or me-too drugs that do little against the diseases and a lot in devastating the healthcare budget. The more we see breakthroughs, the more we get greedy, asking or at least hoping for more treatments that work at the root-cause of diseases, personalized medicine, where patients are protected from treatments that promise better disease management, but are themselves etiologies of new diseases, adding different pathologies and devastation than the diseases they are supposed to treat.

We don’t want today’s news coming from Illumina to be missed by those who are interested in the advancement of molecular biology, computer science and biotechnology in general. We want to bring this news to the patients who suffer the adverse events of their treatments in addition to the devastation of their diseases. We want to bring it to the researchers in pharmaceutical and biotechnology companies who dream about cutting the enormous expenses of clinical trials and shortening the time required to take their molecules from the preclinical laboratory to the market.

In a press release Illumina (ILMN) announced the launch of its VeraCode ADME Core Panel designed to help researchers study genetic predispositions for differential drug response and adverse events. The Panel offers the most complete representation of the key biomarkers associated with drug absorption, distribution, metabolism and excretion (ADME) as standardized by pharmaceutical industry experts in the PharmADME Core List. Researchers using the product can analyze key ADME content in as little as one day, which never happened before.

Dan Masys, M.D., chair of Bioinformatics at Vanderbilt University said, “We selected the ADME Core Panel for its multiplex coverage of the key biomarkers associated with drugs in our initial study, and for its efficiency in processing samples. We are looking for a set of variants that reliably predict an adverse effect from a medication, and have over 80,000 DNA samples with matched, de-identified medical records to support the study.” Dr. Masys is leading the Vanderbilt Electronic Systems for Pharmacogenetic Assessment study using the VeraCode ADME Core Panel to generate an extensive pharmacogenetic database. This database will be widely used to link genotypes to drug response phenotypes extracted from electronic health record data.

What does the VeraCode ADME Core Panel offers? According to the firm’s press release, it offers the following:

The VeraCode ADME Core Panel contains 184 biomarkers in 34 genes, with the highest coverage of the PharmADME Core List. This list, produced by the PharmADME Working Group of industry and academic experts, provides comprehensive coverage of the most biologically relevant biomarkers spanning complex regions of the genome.

Rapid and precise genotyping assay: Using a highly specific and streamlined chemistry, DNA can be genotyped within 8 hours with less than 2.5 hours of hands-on time, enabling researchers to complete studies faster than ever before.

Data precision with an excellent call rate. The supporting software for VeraScan offers a convenient user interface that manages user authentication, logs system activity, and automatically translates genotype data into the star nomenclature used by researchers to analyze pharmacogenetic data.

“Understanding genetic variability associated with drug response and disposition is a key step toward the realization of personalized medicine, and the VeraCode ADME Core Panel will help enable this exciting transformation,” said Jay Flatley, president and CEO of Illumina. “With the cost of bringing a new medicine to market now exceeding $1 billion and over $220 billion spent annually on medications in the U.S., it is critical that we look for savings in all stages of drug development. With its emphasis on rapid operation and high-quality data, the VeraCode ADME Core Panel can help bring safe and effective therapies to patients as quickly as possible.“

That is what we precisely expect from the biogenetic revolution. Far-reaching, but safe drugs, personalized medicine, using computers, instead of torturing animals and bringing down the healthcare cost. You know what? If we don’t bring down the cost of developing new drugs, we will end up not able to use them at the time they reach the market, let alone developing them.

Thank you ILLUMINA.

For more information visit www.illumina.com/VeraCodeADME.

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Sequenom (SQNM): Looking for facts.

  Tuesday, March 23, 2010

After all we wrote about Sequenom, it looks as if we are somehow destined to further tackle this firm. Sequenom’s story is still unfolding. What happened to this firm’s stock this month reflects a general reality that goes beyond Sequenom, on Wall Street and off Wall Street. When some influential leaders decide to become misleaders, sincere observers and followers must develop protective strategies that help them accept or reject what the influential try to feed them. In the stock market, negative speculators have the advantage of possessing a superior weapon called fear, which positive analysts do not really have. Under some circumstances, by definition, negative becomes the synonym of fear. When people fall to fear, they cannot wait for rational explanations of negative speculation. They believe they have no time to lose in resorting to common sense. They feel compelled to escape from the source of their fears. In the stock market, this translates into selling the stock that somebody labeled as bad.

Assessment of public firms by positive and negative analysts is the kinetic energy behind the millions of shares traded daily, which makes the stock market so interesting and attractive. The war between the two opposing groups is excellent for traders who know how to synchronize their buying and selling with optimistic and pessimistic articles. Serious investors, though, those who really care about investing in promising firms, must have a plan to protect themselves from developing unfounded fear or baseless euphoria. The plan is simple. Look for facts that support the claims.

In the past few weeks, investors in SQNM got lost between the positive and negative analysts. The stock was around $5 when some analysts upgraded it, with one putting $16 as his SQNM target price. Investors rushed to buy SQNM. As the stock crossed $8 with very strong momentum, suddenly, negative articles demeaning the stock emerged. We were not convinced. The only fact they cited was the firm’s financials, which had nothing to do with our positive feeling towards the stock. As we said in our previous article, what motivated us to love Sequenom in the first place was our belief that the firm’s promises could generate billions of dollars for its shareholders. These promises are supported by the firm’s successful marketing of three tests; two of them relate to Sequenom’s non-invasive nuclear prenatal program. Moreover, one of Sequenom’s tests has enabled the distinction between maternal and fetal DNA for both male and female fetuses, which led us to believe that there is sufficient validation of the firm’s technologies behind the non-invasive prenatal test program.

After enormous efforts to cause a sell-off of SQNM through incessant negative campaigns of fear, the fear finally supervened. Helped by preprogrammed selling the stock lost around one third of its value. Today, we heard that Lazard Capital Markets analyst Dr. Sean Lavin upgraded his outlook to "Buy" from "Hold" with a $13 price target, citing the potential for the company to create a test for Down syndrome. The stock rebounded fiercely through a different kind of fear this time, i.e. losing a huge investment opportunity. Lavin said the company will likely create a test for Down syndrome with third-party data showing it to be more accurate than current screening. He said the nearly 30 percent drop in the stock value last week is an opportunity for investors. "We anticipate Sequenom finishing development of a Down (syndrome) test this year, running external trials in 2011, and launching a test in late 2011 or in 2012," he said in a note to investors. He said the company has risks, but its target markets are "too immense" and a successful test is too likely. In the U.S., the company could target a market worth more than $4 billion. With all that said, he added, an investment in the stock is "speculative and risky."

That’s what sincere analysts should say – to mention the risk and the reward. In Sequenom’s case, the reward is huge. The successful outcome of Sequenom’s tests will not stop at the two that have been launched and the expected successful outcome of the one for Down syndrome, but open the door to many tests, which would be big.
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Investors’ Rush To Buy ImmunoGen IMGN

  Friday, March 19, 2010

IMGN soared on Thursday, as investors did not miss the parts related to ImmunoGen in the article Robert Langreth, a senior editor at Forbes wrote about the pharmaceutical company Roche’s status and plans. In the article, Severin Schwan, Roche’s chief executive remind of his firm’s dominant position in the cancer drug market, stressing the fact that Roche’s cancer drug Avastin is hard to beat, regardless of what other pharmaceutical companies claim about their upcoming competing drugs. “In cancer, we are at the beginning,” Schwan told Forbes in an interview yesterday. “The barrier to beat our established drugs is very high. It is extremely difficult to find something better than Avastin whose sales approached $6 billion last year.”

The part related to ImmunoGen, though, came when the article described Roche’s plans. He wrote, “Roche is testing drugs called armed antibodies that combined targeted therapy with traditional toxic cell-killing drugs. The idea is that the antibody will deliver the toxic chemotherapy directly to the tumor, maximizing effectiveness and limiting side effects.” Is this not ImmunoGen’s drug? Indeed it is as investors who read the article confirmed their notion when it mentioned the armed antibody by name “T-DM1”, described it as much as possible, and confirmed that ImmunoGen is the developer of the drug.

Motivating IMGN’s rally yesterday is the part where the writer said, “Roche is in late stage testing of T-DM1 that chemically links Herceptin to a potent chemotherapy toxin. If the drug works, patients will take one drug for breast cancer instead of two or three. Roche, presumably, could charge a higher price for the two-in-one combo, especially if it has fewer side effects.”

Further exciting ImmunoGen’s fans was the part of the text that said, “Schwan swears there are no big more mergers in Roche’s future because we think it destroys a lot of value. We are going for targeted midsize acquisitions.”

Well, well, well. Reading the article’s description of ImmunoGen’s T-DM1 drug was enough for informed investors to connect the dots. ImmunoGen typically fits the description of what Roche is interested in. If taken over, the firm, which is a small size, the premium to be paid in case of acquisition would turn it into a midsize firm. In addition, investors know that ImmunoGen is not a single cancer drug firm, but has a pipeline of several conjugated monoclonal antibodies developed for several cancers. More important, it owns the TAP technology that made possible what defied other firms’ previous attempts, i.e., designing and developing monoclonal antibodies that carry highly toxic payloads, yet leave the normal cells intact because they released their toxic ammunition only inside the cancer cells.

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SEQUENOM (SQNM): Does Anyone Really Believe That Missing Analysts Expectations By Five Cents Should

  Wednesday, March 17, 2010

SEQUENOM (SQNM): Does Anyone Really Believe That Missing Analysts Expectations By Five Cents Should Cause a Sell-Off In A Fundamentally Sound Development-Stage Biotechnology Firm’s Shares?

To those who keep downgrading Sequenom we say, we would love to be your disciples on condition that you do not try to convince us that development-stage firms deserve to be downgraded and their stocks crushed because their quarterly reports show a loss of five cents more than analysts’ expectations. We will become your faithful followers if you tell us in straight sentences that the firm’s scientific fundamentals are dismal – its technologies are not working, its programs are not advancing and its future is bleak, which you certainly did not do yet.

We will do exactly what you recommend if you tell us something more convincing than the story of the five cents. Here is some help:

A few months ago, Sequenom has launched SensiGene™ Cystic Fibrosis (CF) Carrier Screening test.

- Do you believe the test has been launched?
- Do you believe the test is not working? If so, then explain why
- Will the test not sell? If so, why?

In Sequenom’s non-invasive nuclear prenatal program, the firm has launched SensiGene Fetal RHD Genotyping test, which was its first clinical laboratory test to be accomplished through its SEQureDx™ technology.

- Do you believe the test has been launched?
- If so, then do you believe the test is superfluous? If so, then could you please explain why do you think so?

In the same prenatal program, Sequenom has launched another test, the non-invasive SensiGene™ Fetal (XY) (Fetal Sex Determination) test, which is also powered by SEQureDx™ technology.

- Again we ask: Do you believe the firm has launched this test? Do you believe it is safe, accurate and highly specific and has the same accuracy reading the fetus sex early in pregnancy? If not, please explain.

- Do you believe that the test enabled the distinction between maternal and fetal DNA for both male and female fetuses? If so, then do you believe that the test validates the firm’s technologies behind the non-invasive prenatal test program?

Yes, we are ready to follow your negative recommendation about this biotech firm, but first, please tell us what you know and think about Sequenom’s MassARRAY® genotyping system? Do you like it? Do you believe that this system and other few similar systems are making a historical move into the clinical laboratory?

Did you know that by using Sequenom MassARRAY® genotyping, Children’s Hospital Oakland Research Institute and the University of Minnesota Cancer Center were able to find out that donors who have natural killer cell immunoglobulin-like receptor (KIR) haplotypes, would significantly improve treatment in patients with acute myelogenous leukemia (AML) who received hematopoietic cell transplantation (HCT) therapy. As a matter of fact, the AML patients who received T-replete hematopoietic cell transplantation therapy1 based on Sequenom MassARRAY® genotyping had significant survival benefit.

1. The complete results from the study are published in Blood (Volume 113, Number 3), the journal of the American Society of Hematology.

If you still believe that the five cent story that ate over 25% of SQNM’s price in two days based on your preaching is more important than the above story, then here is another story: Using an assay exclusively licensed from Sequenom, researchers published a study by the University of Michigan, which demonstrates that around 15% of women determined to be negative human papillomavirus (HPV) in the cervix with the standard HPV DNA test may actually be infected with the virus. Sequenom assay was able to detect the presence of high-risk HPV in 46.7% of women who tested negative by the commonly used Hybrid Capture 2 (HC2) test.

WOW!

You know what? Those who continue to believe that the missed five cents in the quarterly results of a firm that is still building its programs cannot be but irrational. You know how we know? Because the missed five cents are spent on the firm’s programs. Development-stage firms have to spend on building their programs before they cash in on them. By the way, the aforementioned test is another example of how the genomic knowledge is entering the clinic through new advanced DNA sequencers. In the meantime, it further validates Sequenom’s technology.

Sequenom is in the process of researching and evaluating a potential molecular diagnostic for the assessment of risk in developing age-related macular degeneration (AMD).

With regard to the T21 (Down syndrome) test, Sequenom said it is committed to the development of the noninvasive T21 test.

Prohost Word: Until the bearish on Sequenom convince us that they are not playing a game, and their concerns about Sequenom’s science, technology and products are real, we continue to believe that the future of medicine and agriculture is now in the hands of the genomic group. Sequenom is, indeed, a member of this group with advanced standing. The genomic firms’ technologies, tools and discoveries are the engines of the revolution in medicine and in the discovery and development of breakthrough drugs. (Read the article entitled Genomics in Prohost Newsletter for subscribers only.)

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INTERMUNE: Pulmonary Fibrosis Drug Will Open the Door to A large Product Pipeline

  Thursday, March 11, 2010
The FDA Pulmonary-Allergy Drugs Advisory Committee (PADAC) voted 9-3 to recommend approval of Esbiet® (pirfenidone) developed by the biotech firm Intermune (ITMN) for idiopathic pulmonary fibrosis (IPF). The same drug is approved in Japan for IPF and sold under the trade name Pirespa® by Shionogi in that country. Both Intermune and Shionogi had licensed pirfenidone from Marnac and its co-licensor, KDL GmbH.  More...

ISIS (ISIS): Lou Gehrigs Disease Antisense Drug Has Reached Clinical Trials

  Monday, March 08, 2010
Isis initiated a Phase 1 study of its antisense drug ISIS-SOD1Rx in patients with an inherited, aggressive form of Lou Gehrig\'s disease also known as familial amyotrophic lateral sclerosis. (ALS). As ISIS-SOD1Rx selectively inhibits the production of SOD1, it is expected to benefit the cases of familial ALS that are related to mutant forms of superoxide dismutase, or SOD1, which constitutes approximately 20% of ALS patients suffering from familial ALS.  More...

ON SEQUENOM (SQNM)

  Tuesday, March 02, 2010

On September 22, Prohost wrote:  More...

Time To Appreciate ISIS PHARMACEUTICALS (ISIS)

  Tuesday, March 02, 2010

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