News and Comments

ImmunoGen And The Second Lethal Virus

  Monday, August 30, 2010

Two lethal viruses plague general investors. The euphoria virus infects investors when the market stages sustained rallies. This virus makes investors buy and buy until the balloon becomes overstretched and eventually bursts. The fear virus causes hysteria in susceptible investors when their stocks go south and rush them to sell at ridiculously low prices. The fear virus also provokes vulnerable investors to immediately dispose of their stocks at the hint of unfavorable news, whether or not the negative news is indicative of weakness in the firms’ fundamentals. Healthy, rational investors usually take advantage to accumulate the battered stocks of firms with strong fundamentals at the new bargain price that results from sell-offs by the fearful.  More...

Compugen (CGEN): Advancing the Drug Discovery Process

  Thursday, August 12, 2010

With the evolution of the genomic and proteomic technologies, we highly regard and appreciate the firms that have invested in these technology towards breakthrough discoveries that are used in a therapeutic progress towards healing chronic and deadly diseases. One of these firms has offered us more than hints about what it can accomplish through its thoroughly developed technologies. The firm in question is Compugen (CGEN), is still small as measured by its market cap, but we see it as a future giant based on its achievements. Using its LEADS Platform and other proprietary algorithms and technologies, Compugen was capable of predicting in silico the existence of novel members of the B7/CD28 family of co-stimulatory proteins, and discovering an extracellular region of the predicted (previously unknown) membrane protein in the B7/CD28 family.  More...

VERTEX (VRTX): At the Finish Line

  Monday, August 09, 2010

When an investigational drug proves it can eradicate a virus we call it a breakthrough. The nearest to approval of the expected breakthroughs is Vertex’ (VRTX) drug telaprevir, a specific hepatitis C virus protease inhibitor. The drug was a blessing for the infected victims who had it in clinical trials. It brought a miracle happy ending to what seemed to be an endless nightmare of acute liver inflammation, chronic liver inflammation, liver cirrhosis, liver transplant and possible liver cancer. We expected telaprevir to be greeted by the government and insurance companies, which spend ton of money on the HCV liver problems where treatments that do not work are, nevertheless, paid for.  More...

Onyx (ONXX) Was Right All Along

  Monday, July 26, 2010

In October 2009, Onyx (ONXX) paid $276 million up front to acquire the privately held Proteolix to get to its multiple myeloma drug carfilzomib. It promised $535 million in future payments largely contingent on carfilzomib's approval. As usual, investors did not like Onyx paying money, because investors do not like any company to pay money to develop or acquire state-of-the- art products. When somebody hates something he sees everything in it with pessimism. Onyx put its hands on carfilzomib, which looked very promising for multiple myeloma cases resistant to current treatments. The breakthrough multiple myeloma drugs Velcade (bortezomib) and Revlimid (lenolidamide) could not prevent the cancer from recurring and failed to work at all on a large percentage of recurrent cancer.  More...

HIV/AIDS: Great News With Enormous Promises

  Wednesday, July 14, 2010
When people began to develop acute immune deficiency syndrome and die, horror was the feeling of humans allover the world. Nobody had yet known the cause of the catastrophe and no science could deliver any explanation. When the explanation emerged, the horror became a double horror for those who found out that the culprit is a new virus that has the ability to destroy the immune system and its presence is a death sentence. For a few years, patients died by the thousands without any hope without treatments and with the first generation treatments. The side effects of the combination drugs given at the time were bitter honey that offered sweet unreliable hope and bitter devastation and despair.

It was real horror for the patients, their relatives and friends until the advancement in molecular biology and biotechnology began to yield drugs that turned the desperation into real hope. The improvement on these products by companies like Gilead (GILD) contributed to patients’ well being and to a better prognosis for the devastating disease. HIV patients are now living almost a normal life, but unfortunately, no protective vaccines and no cures have succeeded in reaching the market. All attempts to developing vaccines have failed and all promises of cures have died in experimentation. The mini HIV creature has been capable of evading all scientists’ creative means to prevent it from destroying human immune system cells.

Suddenly, and unexpectedly, last Thursday morning, we opened our eyes on breathtaking news from the National Institute of Health (NIH). The NIH announced the discovery of two natural antibodies that can prevent 90% of known HIV strains from infecting cells. A study detailing the research was published in the online edition of the journal Science. The unexpected pleasurable news made us feel, for the first time in years, that humans are finally on the most relevant path towards beating HIV at its own game. We felt that we are on the brink of winning the quarter of a century war between the human mind and the invisible mindless HIV creature, which used its built in instinctive savoir-faire in dismantling millions of human immune systems while protecting itself against all kinds of attempts to assassinate it, or prevent it from causing what medical literature call acquired immune deficiency syndrome (AIDS), killing millions of victims. The NIH news hinted that the research would probably lead to the development of far-reaching HIV preventive vaccines and treatments.

Like all stories of breakthrough achievements, the hero behind this story is the privilege restricted to humans known as curiosity. NIH’s scientists’ curiosity has led them to observe that a few HIV-infected patients have not developed AIDS. They called those patients, non-progressors. It was obvious that the lucky few must have something unique in their immune system that would prevent the virus from invading their immune system cells. Motivated still by academic curiosity, the scientists were determined to unearth the reasons behind the strange phenomenon.

Together with other scientists from various medical and research institutions, the NIH team used a novel molecular device that consists of an HIV protein they modified so it would react only with antibodies specific to the CD4 binding site of HIV. The trick worked. Two antibodies, VRC01 and VRC02 did attach to the protein. The scientists then determined the atomic-level molecular structure of VRC01 when attached to the CD4 binding site. The findings enabled them to learn the structure of the discovered natural antibodies. It is important to know that the CD4 binding site of the virus does not mutate, which explains the reason that the discovered natural antibodies are capable on neutralizing the effects of a vast majority of HIV strains. 



The information was sufficient for the scientists to begin designing components of a candidate vaccine that could stimulate the human immune system to make the natural antibodies, which would prevent infection by many HIV strains worldwide. Developing vaccines to produce the natural antibodies face some technological challenges. The leader of the research, Dr. Kwong, and his colleagues explored how this challenges might be addressed by designing vaccine components that could guide the immune system through the stepwise antibody maturation process, hence, facilitating the generation of a VRC01 antibody from its precursors. Another suggested possibility is designing gene therapy where a gene can express one of the antibodies, probably, VRC01. 



The reason for the failure of the naturally occurring antibodies in protecting the majority of patients is that the immune system usually produces them after the patients are infected. In HIV, the virus has the advantage of debilitating the immune system before the protective antibodies go into action.  



Experts’ Comments



Dr. Gary Nabel, the National Institute of Allergy and Infectious Diseases said:



"I am more optimistic about an AIDS vaccine at this point in time than I have been probably in the last 10 years. Two natural antibodies can attach to and neutralize 90 percent of the various mutations of the human immunodeficiency virus that causes AIDS. 



“We have used our knowledge of the structure of a virus—in this case, the outer surface of HIV—to refine molecular tools that pinpoint the vulnerable spot on the virus and guide us to antibodies that attach to this spot, blocking the virus from infecting cells.”

"This is an antibody that evolved after the fact. That is part of the problem we have in dealing with HIV -- once a person becomes infected, the virus always gets ahead of the immune system.  What we are trying to do with a vaccine is to get ahead of the virus."



Anthony S. Fauci, M.D., director of the National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health, said:

“

The discovery of these exceptionally broadly neutralizing antibodies to HIV and the structural analysis that explains how they work are exciting advances that will accelerate our efforts to find a preventive HIV vaccine for global use.” In addition, the technique the teams used to find the new antibodies represents a novel strategy that could be applied to vaccine design for many other infectious diseases.”

Dr. Mascola, the deputy director of the VRC, said:

“The antibodies attach to a virtually unchanging part of the virus, and this explains why they can neutralize such an extraordinary range of HIV strains.”

Dr. Kwong who led the team at the NIH said, 



“The discoveries we have made may overcome the limitations that have long stymied antibody-based HIV vaccine design.”  



Research teams: The researchers included NIAID scientists from the VRC, the Laboratory of Immunoregulation, and the Division of Clinical Research, all in Bethesda, Md.; researchers from Beth Israel Deaconess Medical Center in Boston; Columbia University in New York; Harvard Medical School and Harvard School of Public Health in Boston; The Rockefeller University in New York City; and University of Washington in Seattle.



References:



Wu X et al. Rational design of envelope surface identifies broadly neutralizing human monoclonal antibodies to HIV-1. Science. DOI: 10.1126/science.1187659 (2010).



Zhou T et al. Structural basis for broad and potent neutralization of HIV-1 by antibody VRC01. Science. DOI: 10.1126/science.1192819 (2010). 



Prohost Comments



This is good news. It offered insightful research, very promising results and suggestions for far-reaching treatments. We do not know which one of the drug designers and developers has helped design and develop the molecules used in the NIH experimentation and we do not know who will develop the therapeutic molecules or the vaccine. But we certainly know that these tasks are none of academia or government businesses. They are the specialty of drug developers that have state-of-the-art drug design and monoclonal antibody technology. There is no doubt that the NIH information has brought new possibilities for the drug developers that are interested in conquering HIV.

We take advantage to recognize the fact that the successful NIH research would not have been possible if not for the accumulated data from the previously failed attempts to develop HIV preventive and therapeutic vaccines. Some of the companies that spent a lot of effort and money on their unsuccessful attempts might have failed to survive, but their efforts have not gone to the drain. For scientists, data from failed experiments are cause for improvement. Data about failed trials are teaching treasures. They teach scientists the reasons for previous failures. They alert them against scientific mistakes that could jeopardize their efforts. They open scientists’ minds to scientific and procedural facts that are usually introduced to science after failed trials.

Before we forget, we have to admit that the firms developing the current HIV treatments have rescued millions of patients from agony and death. The world should be grateful to: Gilead (GILD), Vertex (VRTX), Abbott Laboratories (ABT), Bristol-Myers Squibb (BMY) Roche (RHHBY), GlaxoSmithKline (GSK) and any other HIV drug developer anywhere n the world that we might have omitted mentioning.

These firms will continue to offer HIV treatments for years to come and will continue to improve on them. Gilead sciences is the leader of the HIV innovative drugs. Its protease inhibitors and combination drugs have caused a tremendous transformation in HIV victims’ lives and well being.

In related news, a week ago, the U.S. decided upon a new AIDS policy that aim at finding new ways to educate people about HIV. The policy aims also at getting more patients to treatment as fast as possible, urging the FDA to consider reviewing new HIV tests a priority. The guidelines   focus on prevention and on making sure that the patients have access to care and to tests that monitor the infection so they can take their drugs at the right time.

This news is good news for HIV victims and for HIV drug developers. More...

A New State-Of-The-Art Diagnostic Test

  Wednesday, July 14, 2010
The Biotechnology Revolution: A New State-Of-The-Art Diagnostic Test



Genomics are changing the way medicine is being practiced, bringing to the labs state-of-the-art diagnostic tests that do much more than ruling in, or out diseases. The new laboratory tests enable physicians to determine the most effective treatment options for their patients’ diseases, to monitor the disease progression and install the treatment at the right time.  This is no dream any longer, but reality. The latest news about achievements in this domain comes from Life Technologies (LIFE) and Asuragen, which announced yesterday they have achieved CE-marking and commercial launch in Europe of the BCR/ABL1 Quant™ Test that  enables clinicians to monitor and treat patients afflicted with chronic myeloid leukemia (CML).



Asuragen's clinically validated and cGMP manufactured the BCR/ABL1 Quant™ Test, which is  distributed by Life Technologies (LIFE) and runs on its Applied Biosystems’ CE-marked 7500 Fast Dx Real-Time PCR Instrument™. The test for leukemia progression is a quantitative in vitro diagnostic test. It helps doctors determine the most effective treatment options for CML. The test monitors the BCR-ABL1 to ABL1 ratio by reverse transcription quantitative polymerase chain reaction (RT-qPCR) on whole blood or bone marrow of diagnosed Philadelphia chromosome positive chronic myeloid leukemia (CML) patients expressing b2a2, b3a2 or e1a2 fusion transcripts. The test is intended as an aid in the assessment of complete cytogenetic response (CCyR), major molecular response (MMR), minimal residual disease and relapse in CML patients. 



The BCR-ABL1 fusion gene arises from a specific chromosome translocation, known as the Philadelphia chromosome or t (9:22). The resulting BCR/ABL1 fusion transcripts are present in approximately 95% of CML. If present, the expression level of the fusion transcript or its ratio to a reference transcript may be used to monitor disease progress. Monitoring the level of BCR/ABL1 may be helpful for both prognosis and management of Gleevec®, Tasigna®, Sutent® and Sprycell® kinase therapies in patients with leukemia disease.

To read the press release and about the firms go to: 

http://www.lifetechnologies.com/news-gallery/press-releases/2010/asurage-ad-life-techologies-lauch-ce-marked-bcr.html    

Asuragen and Life Technologies have plans to pursue future regulatory clearance for a BCR/ABL1 Quant test in the United States. 



See Also: Illumina (ILMN), Sequenom (SQNM), Clinical Data (CLDA), Compugen (CGEN) More...

Indeed, Roche Filed NDA For T-DM1

  Wednesday, July 07, 2010
The First Conjugated Monoclonal Antibody From ImmunoGen’s (IMGN) Technology To Reach That Stage

In Today’s news, Genentech, a member of the Roche Group, announced that the company has, indeed, submitted a Biologics License Application (BLA) to the FDA for trastuzumab-DM1 (T-DM1). The drug’s technology is licensed from ImmunoGen (IMGN). The BLA is for approval of T-DM1 in people with advanced HER2-positive breast cancer who have previously received multiple HER2-targeted medicines and chemotherapies. This submission is based on the results of a Phase 2 study, which showed T-DM1 shrank tumors in one-third of women who had received on average seven prior medicines for advanced HER2-positive breast cancer.

This submission was expected and Prohost mentioned it time and time again, the latest was in Prohost Letter #303 posted on Prohost website on Tuesday when we wrote: “Seeing patients who became resistant to its drug herceptin respond to T-DM1, Roche became excited and motivated to ask the FDA for an early approval of the drug. Patients who had previously received, on average, seven different drugs for their advanced breast cancer had an objective response to treatment with T-DM1. Among the responders were a substantial number of patients with recurrent disease who became resistant to Roche’s blockbuster drug Herceptin. How could anybody believe that ImmunoGen, which has a pipeline of successfully conjugated drugs, be bad?”

The Prohost Letter wrote, “ImmunoGen’s weapon resides in its technological capability that succeeded for the first time in history in designing and developing safe and effective conjugated monoclonal antibody drugs. ImmunoGen’s lead drug Trastuzumab-DM1 (T-DM1) is a monoclonal antibody against HER2 neu breast cancer loaded with the cancer-cell killing agent, DM1. The drug has demonstrated efficacy in patients suffering from her2 neu recurrent resistant breast cancer patients.”

In Genentech/Roche press release, Hal Barron, M.D., executive vice president, Global Development and chief medical officer said: “While we’ve made great strides in treating HER2-positive breast cancer, there is a group of people whose breast cancer will come back after many treatments, leaving them with very limited options. Data from studies have shown that T-DM1 shrank tumors in these people, so we are excited to have submitted this application to the FDA in hopes of offering a potential new medicine to people with this type of breast cancer.”

T-DM1 is an antibody-drug conjugate (ADC), also known as an armed antibody, being studied for advanced HER2-positive breast cancer. T-DM1 attaches trastuzumab and the chemotherapy DM1 together using a stable linker, which is designed to keep T-DM1 in one piece until it reaches specific cancer cells. The antibody (trastuzumab) binds to the HER2-positive cancer cells, and is thought to block out-of-control signals that make the cancer grow while also calling on the body’s immune system to attack the cells. Then, once T-DM1 is absorbed into those cancer cells, it is designed to destroy them by releasing the DM1.

A Phase 2 study known as TDM4374g, designed to assess single-agent T-DM1 in 110 women with HER2-positive advanced breast cancer whose disease had worsened after receiving at least two prior HER2-targeted treatments Herceptin® (trastuzumab) and lapatinib) in the metastatic setting, as well as an anthracycline, a taxane and capecitabine, demonstrated the following results: 

-- T-DM1 shrank tumors in 33 percent of women with advanced HER2-positive breast cancer that had worsened following treatment with an average of seven prior medicines for metastatic disease.

-- Most side effects were mild (Grade 1-2) and similar to those observed in previous clinical trials of T-DM1.

More trials of T-DM1 are planned for the drug, either alone or in combination with other medicines.  An ongoing Phase 3 trial, known as EMILIA, is comparing T-DM1 to lapatinib in combination with capecitabine in people with advanced HER2-positive breast cancer whose disease has worsened after receiving initial treatment. Also, a planned Phase 3 study, MARIANNE, will compare both T-DM1 alone and T-DM1 in combination with pertuzumab to Herceptin in combination with a taxane chemotherapy in people with advanced HER2-positive breast cancer who have not been previously treated for advanced disease.

Genentech has opened a T-DM1 Patient Access Study in the United States to provide a specific group of people with advanced HER2-positive breast cancer access to T-DM1 while Genentech seeks U.S. approval.

Genentech licenses technology for T-DM1 from ImmunoGen (IMGN).

For the complete press release click: http://www.roche.com/media/media_releases/med-cor-2010-07-07.htm More...

EXELIXIS: Fear based on mind reading vs. reality.

  Monday, June 28, 2010
Following Exelixis (EXEL) press release, which announced that it has regained full rights from Bristol-Myers Squibb (BMY) to its cancer drug XL184, the firm’s stock plummeted. In the press release, Exelixis stated that BMS’ priorities made it difficult for BMS to align on the scope, breadth and pace of the ongoing clinical development of XL184 as agreed upon in the original agreement. Those who instigated the sell-off did not buy Exelixis’ explanation and were more comfortable with the notion that BMS decided to walk away from XL184, probably because of bad news about the drug. Their reasoning was that a giant pharmaceutical company like BMS, which focuses on building an impressive advanced oncology pipeline would not let a promising multi-targeted cancer drug in late phase trials slip from its hands without a significant reason. BMS did not allude to any bad reason or any reason at all for its decision. It did not comment on Exelixis’ explanation and did not deny it. 

Although investors’ speculation and their spontaneous response, instigating the selling of the stock, are not irrational, they were not supported by evidence-based knowledge, or facts. Their best speculation was that if BMS doubts the drug’s promises the least of what they can do is the same as BMS did, i.e., abandoning investing in Exelixis. The sell-off was triggered by a pyramid of suppositions that have no concrete proof.  

Now that the acute fear has subsided and Exelixis stock price bottomed, other investors and, probably, some of those who sold the stock, might like to take a deep breath and reexamine the whole situation in a more professional and rational way. It is not unreasonable for investors to try to find out good investment opportunities in bottomed stocks.

To find out whether the stock selling based on the sellers’ reading of BMS’ mind is correct, or a mistake, we resorted to history, which tells that many large pharmaceutical firms have abandoned the development of drugs that have become best sellers when other firms continued developing them. Looking at the drug itself and the history of its performance in clinical trials, we see XL184 as not only the most advanced product in Exelixis pipeline, but is also the most advanced MET inhibitor. MET pathway has long been recognized as a master switch and drug target in cancer progression. MET is mutationally activated in hereditary and sporadic papillary renal cell carcinoma and some head and neck cancers. It is either over-expressed or activated in the absence of mutation in glioblastomas, breast carcinomas, gastric cancers and other solid tumors. MET amplification has been demonstrated in some NSCLCs.

XL184 also targets and inhibits VEGFR2, which leads to starving the tumors of oxygen through the antiangiogenic effect. Expression of VEGF occurs in various cancers and has been associated with prognostic significance. Targeting the VEGF receptor has already demonstrated efficacy as anti-cancer strategy in multiple tumors. Dual targeting of MET and VEGFR2 blocks two of the major mechanisms tumors use to overcome hypoxia.

In addition, XL184 inhibits RET, which is identified in multiple endocrine tumors and familial medullary thyroid carcinoma. Activated RET is involved in regulating cell proliferation, migration, differentiation, and survival. It is activated in papillary thyroid cancer (PTC) and in both familial and sporadic forms of medullary thyroid cancer (MTC).

These unique combination of actions makes XL184 capable of causing antiangiogenic, antiproliferative, and antiinvasive effects in tumors – all have been confirmed in preclinical cancer models and in some clinical trial results. 

At ASCO 2010 Annual Meeting, the developers reported that in phase 1 trials on patients with  medullary thyroid cancer, XL184 demonstrated a 29% response rate, with a median duration of response that had not yet been reached, with a range of 4 to 35+ months. They also reported that in a phase 2 clinical trial on patients with the nasty glioblastoma, XL184 demonstrated a 30% response rate when dosed at 125 mg daily, with a median duration of response of 5.1 months. Data were presented showing that the drug demonstrated objective responses in patients with refractory melanoma, non-small cell lung cancer (NSCLC) (both as a single agent and in combination with erlotinib), hepatocellular carcinoma, prostate and ovarian cancers in an ongoing adaptive randomized discontinuation trial (RDT).

The above results were announced by the developing firms prior to their divorce. They are encouraging.  Do we have any concrete reason to doubt them? We personally don’t. All what we see now is a unique drug that targets three very important pathways of a multitude of cancers. About future plans, the drug will be in the hands of the FDA for approval of its first indication, thyroid cancer, in 2011. Exelixis expects to initiate a phase 3 pivotal trial in recurrent glioblastoma in the year-end 2010 time frame and to prioritize tumor types from the RDT in 2011 to add more cancers to the drug indications.

This is how we see the story. More...

Regeneron: Arcalyst is the missing link in the treatment of gout

  Friday, June 11, 2010
Regeneron’s (REGN) Phase 3 study in gout patients initiating allopurinol therapy to lower their uric acid levels showed that the company’s drug Arcalyst (rilonacept), known as IL-1 Trap, prevented gout attacks, as measured by the primary endpoint of the number of gout flares per patient over the 16 week treatment period.

Is this s good, or bad news?

Judging from investors’ reaction to the news, one would believe the clinical trial results are a failure. However, judging based on understanding the disease, its causes, complications, symptoms and what current conventional treatments are accomplishing, or lacking would prove that Arcalyst might be the missing link in the treatment of gout. The drug neutralizes a major mediator of the severe attacks of inflammatory symptom flare up the patients suffer from after taking the uric acid lowering drugs, which is the fundamental treatment of gout. That’s what makes the bulk of patients who abandon the treatments that deal with the cause of disease, i.e., elevated uric acid. of the cause of disease. No conventional drug taken at a safe dose has been effective in preventing the flare up of the symptoms, which take their toll on patients and probably lead to worsening of the disease. It is easier for gossipers to perpetrate skepticism towards Regeneron’s drug Arcalyst if the listeners do not understand what gout is and what treatments are required for its successful management. The fact is that, gout requires more than two classes of drugs to stop the main cause of this disease, control its disabling symptoms, and prevent the attacks, thus, the consequent complications if the disease is not well treated.  

Gout occurs in patients who have elevated levels of uric acid. The uric acid is deposited in the joints and other body tissues. In the joints uric acid causes joint inflammation, joint pain, stiffness swelling, redness and heat. Deposited in soft tissues, uric acid causes the same it does the joints. Generally, patients experience flare up of symptoms that no drug is yet capable of preventing. The current management of gout aims first at lowering uric acid by either decreasing its formation or increasing its elimination from the body with uric acid-lowering drugs. Drugs exist that can tackle one or the other of the two actions. For example, the drug Allopurinol, decreases uric acid formation and  Probenecid helps the body eliminate excess uric acid through the kidneys and into the urine. The problem is that controlling the level of uric acid does not stop all the symptoms right away but the pain and suffering might increase and persist for long. The reason is that the breakup of uric acid crystals boosts inflammatory mediators, especially interleukin-1 (IL-1), which causes the flare. IL-1 is targeted for inhibition by Regeneron’s drug Arcalyst. The flare the patients experience after the treatment makes it difficult for them to adhere to uric acid-lowering therapies and discontinue the treatment within the first few months of therapy. That’s why Arcalyst is badly needed.

Currently, physicians are using the anti-inflammatory drug colchicine to calm down inflammation. In many cases, however, the dose required for the control of severe inflammatory symptoms is high and must be taken for a long time, which makes its side effects prohibiting. Patients would suffer severe diarrhea, abdominal cramps, nausea, and vomiting. Non-steroid anti-inflammatory drugs are used also, but in many cases they do not control the severe inflammatory symptoms during the flare, unless very high doses are given for a long time. Long-term use of high dose can provoke grave side effects, including peptic ulcer, kidney failure and liver failure. Steroids are resorted to, yet, again, their long-term high dose use could result in detrimental side effects, which go from immune system suppression, to osteoporosis, bone fracture, endocrine system deregulation, difficulty of wound healing, diabetes and the list goes on and on.

So, the most important results from Arcalyst phase 3 study is the one, which confirms that the concomitant use of the drug with uric acid-lowering therapy for the first several months may help avoid gout flares. The use of Arcalyst is expected to improve patients’ adherence to uric acid lowering drugs, sustain their wellbeing and improve the outcome of their disease. Is this bad news? More...

The fruits of the Human Genome Project Are Ripening

  Friday, June 04, 2010
In June 2009, Illumina announced the launch of its individual genome sequencing service. The service is built around physician-patient consultation, with a physician’s order required to initiate the process. Sequencing is performed in Illumina’s CLIA-certified, CAP-accredited laboratory, following a rigorous process that focuses on physician involvement, patient privacy and data quality. The news was big, but what makes such milestones in the medical fields achievable is individuals’ serious consideration, which is possible only through offering them explanation of the purpose of the newly suggested process. In the case of individual genome sequencing (IGS), individuals should know more about sequencing, its purpose, the results and the impact on their protection and treatment, in case they are diagnosed with a sickness. The June 2009 announcement of the sequencing service, Illumina was not crystal clear on both the clinical utility of the sequencing and its cost.    

Today, Illumina (ILMN) announced the price of IGS. The details of the clinical utility of the sequencing is mostly left for physicians to elaborte on, as they are the decision makers with regard to IGS. The price decided upon is $19,500 for individual genome sequencing service and $14,500 per genome for groups of five or more participants using the same physician. In other words, to help lower the cost for clients, a physician can order five genome sequencing at a time thus saving $5000 for each individual. This is not all. Patients who suffer from serious medical conditions for whom IGS could provide potential direct clinical value the cost will be $9,500 per genome. In all cases, the service requires individuals to follow Illumina’s physician-mediated process, which involves pre-service consultation, consent, and a seven-day cooling off period, with final genome data returned to the physician.

There is no doubt that the announced price of IGS service, especially for disease-threatened patients helps opening the door to a new era where the diagnostic procedures will not only diagnose the pathology, but also its origin, i.e., the root-cause of the disease. Also, the new cost, $9,500, will definitely be a factor in making things happen. This price is much lower than the $25,000 suggested in the past. This step will open the door to therapeutics and therapeutic procedures through emerging technologies such as gene editing through antisense, RNA interference (RNAi, microRNAi and gene switching). Serious biotech companies specialized in these disciplines include ISIS (ISIS), which uses its antisense products for gene-editing, in addition to its microRNA technology developed through a Regulus Therapeutics, a company it established through a joint venture with Alnylam (ALNY), which is also specialized in RNA interference technology. Other firms include, RXI Pharmaceuticals (RXII), which is also specialized in the same. 

We expect the price of individual genome sequencing (IGS) will further drop through more technological advancements, which have been a major factor in driving the cost down in the first place. The lowering of the price will definitely lead to increasing the number of individual genome sequences, which will be compared them to those of the public databases being populated by academia. The content of the databases will be enriched and the entire field accelerated. That’s what Illumina believes would be the case. Moreover, Illumina intends to create the World Genome Registry, a web-based resource for those who have been sequenced to record the date they were sequenced, at what coverage and with which technology platform. This web site will keep the consumer genetics community updated on the current global numbers and status of whole-genome human sequences being generated. The company intends to create this resource and then turn it over to the community once it is established.

Illumina’s (IGS) service provides valuable information regarding individual genetic makeup. Although curiosity could be a driven motive for some people to ask for sequencing their genomes, a good motive should be health-driven such as existing illnesses or family history of diseases. Once the sequencing has been completed, geneticist physicians and the patients’ physicians will review the results and help the individual formulate an action plan, which may include ordering more clinically tests to further investigate the health concerns. Once found vulnerable to developing a disease, or sick, patients will have the chance to receive a much better protective and preventive measures, or, in case of sicknesses, the treatments that work for them, not against their genetic constructs. That’s why we reiterate that the biotech firms specialized in genomics are the heart of the biotechnology industry. We expect more from the genomics group and we definitely expect much more from Illumina. 

Disclosure: No position. More...

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