Showing posts with label anemia. Show all posts
Showing posts with label anemia. Show all posts
Friday, March 23, 2012
Medgenics gets OK to start Phase IIa trial of their EPODURE biopump...
Article posted 3/23/12 on Proactive Investors.com. Israel-based Medgenics gets Israeli Ministry of Health OK to being a Phase IIa trial of it's EPODURE biopump to treat HCV, anemia & haemophilia. The technology will allow patients to continuously produce and maintain their own proteins without dangerous variations in PK.
Medgenics gets approval to begin Phase IIa clinical study on EPODURE biopump
Fri 11:17 am by Giles Gwinnett
Medgenics has received the green light to begin a Phase IIa clinical study on its EPODURE which is used to treat anaemia.
The company has received clearance from the Israeli Ministry of Health to begin the study, it said.
The firm's biopump system sees a tiny sliver of the patient's tissue taken and modified to carry the gene to enable it to continuously produce the required protein to treat the condition.
It is is developing three applications of the technology - to treat anaemia, hepatitis-C and haemophilia.
The study, announced today, will involve 20 patients and will asess EPODURE's ability to replace months of routine injections for anaemic patients who have end-stage renal failure - or kidney disease.
Each patient will receive an individually targeted initial dose of EPODURE biopumps designed to produce levels of the protein that would replace the routine injections over a period of 4-12 months.
President and CEO of the firm Andrew Pearlman said he expected the phase IIa study to build on the positive clinical results from the earlier Phase I/II study of EPODURE to treat pre-dialysis patients with chronic kidney disease.
"This is the first clinical study that will permit adjustment of the EPODURE dose based on patients' needs as is currently done in standard EPO (erythropoietin) treatment, and will be standard in future EPODURE use.
"This represents a significant change from the fixed-dose study we previously conducted in pre-dialysis patients," he said.
Dr Pearlman added that the company's EPODURE Biopump technology offered potential advantages over current therapy because it enabled patients to continuously produce and deliver their own proteins.
"Sustained delivery of EPO is expected to help keep hemoglobin within the target range to reduce the risks of hemoglobin variability, while avoiding the possible risks posed by EPO concentrations many times the normal physiological range, as observed with EPO administrations.
"EPODURE could potentially be a safer anemia management tool. Additionally, the cost benefits for the management of anemia could be highly significant," he said.
Wednesday, February 8, 2012
Medgenics developing new therapeutic therapy for anemia...
Article by Ray Dirks from Seeking Alpha.com. Although not directly related to Hepatitis C drug development, patients on the current treatments for Hepatitis C carry the risk of developing anemia. Sometimes this requires the administration of a medicines that increases red blood cell production, but is expensive and carries it's own risks, such as red cell aplasia or cardiac events. The Seeking Alpha investiment website covers a new treatment for anemia in development from a company called Medgenics. They have developed a mini-bio pump delivery system that results in sustained levels of erythropoietin naturally with very little side effects and at a fraction of the cost. They are entering phase 1 trials for Hepatitis C treatment-related animeia later this year.
Medgenics' Disruptive Biotechnology Is One To Watch
By Ray Dirks
Medgenics (AMEX:MDGN) has, what I believe to be, the most disruptive biotechnologies of the last 20 years. This company is poised to own the 90 billion plus protein therapy market. We are talking about an enormous indication. Nomura (the big investment bank) calls this one of the top 10 biotech companies to watch over the next 5 years.
Currently the only form of treatment for Anemia is Epogen. Epogen is a synthetic version of the Erythropoietin that increases your red blood cell production. It is a $12B a year product monopolized by Amgen (AMGN) and affects nearly 1-2 billion worldwide.
When you are diagnosed with anemia, you go to your doctor. Your doctor is going to tell you that you have an abnormally low hemoglobin count and he will prescribe an epogen shot to get your hemoglobin into check and into a 10-12 count range which is considered healthy.
These shots cost about $25K a patient per year and are very painfully administered. What is going to occur is a spike of your Epo level from very low up to a 100x, which can lead to high blood pressure, a percentage increase of fatal cardiac events (as per the FDA) and all sorts of side effects. Over the next 48 hours, your Epo will crash back to a low level after which you will be treated with another shot of epogen, and this happens over and over again for the rest of your existence.
It's a terrible way to live, costs the system billions and is one of the worst FDA procedures on the market. In fact, Amgen has already received a black box warning because of the side effects and increased risks and is under major scrutiny for reimbursements issues as well.
The holy grail of Anemia treatment is to treat the patient without the infections and without that "spike" in Epo which is deadly in nature and provides a terrible quality of life.
Medgenics has developed a therapeutic system that can provide a dosage of the sustained levels (no spikes) of Erythropoietin naturally with literally no side effects and only two primary clinic visits for a fraction of the money. Let me explain exactly what they do and how they do it:
Medgenics takes a small piece of your skin about the size of a one inch toothpick from your abdomen.
This piece of tissue is then processed in a lab by inserting a small piece of DNA that teaches it to continuously produce the missing protein; which in this case is Erythropoietin.
Once this engineered piece of tissue is making the protein you are missing and at the levels needed, it is injected back under your skin and the result is a mini biobump that produced natural Epogen without the spikes, without the shots, without the side effects or dropouts. And for a fraction of the cost of Amgen's Epogen shots.
The Phase I/II study was done with 20 patients, all people diagnosed with anemia obviously. In all 20 patients you have biopumps producing Erythropoietin safely for as long as 36 months without any follow up, within that 10-12 count and without the injections and their dangerous spikes.
The most compelling thing about this story is that this isn't just a treatment for only Anemia; it is a platform that can be used on ALL protein therapies. In fact the company is entering Phase 1 for Hep C (Interferon) in Q2 of this year. Hepatitis C/Interferon is a 3.5 billion dollar Market this year. Gilead (GILD) paid 11 billion for a HEP C company… Inhibitex (INHX) was taken over for 2.5 billion (another Hep C company).
Medgenics already has an active program for Hemophilia developed with Baxter (BAX), and they will also be starting programs for:
* Growth Failure/growth hormone
* Multiple Sclerosis/Interferon Beta
* Diabetes/Insulin
* many others
In summary you have a company that has:
* $50 million invested
* the most disruptive technology in biotech today
* a management team that is arguably from top to bottom the best I have ever seen. Former head of Stanford Medicine, and an entrepreneur who sold his last company for $600 million
* Baxter collaboration and the potential for many more collaborations on multiple indications
* already finished Phase I/II and entering Phase IIB, this is NOT pre Clinical; this is tested and proven in human
* a ridiculously low valuation relative to its competitors.
* the potential of two probably partnerships in the near term
* a science which is not a one trick pony, it is a platform that covers every protein therapy which is more than a 91 billion dollar market
* largest shareholders are Joel Kanter and Isaac Blech. Joel has just sold Clarisonic for $600 million. Isaac has founded over 35 billion in companies
Enough of the science. Let me walk you through the value proposition of the investment. There are two public companies and one collaboration that I will use as comparisons.
Protalix (PLX) has a platform technology and is at about the same stage of clinical as Medgenics, but is only for an indication of 25,000 patients and trades at a 520 million dollar market cap.
Prolor (PBTH), which has a treatment which only increases the half lives of protein therapies, trades at a 300mm market cap.
And of course Zymogenetics which only has an interferon lasting slightly longer than the usual 5-6 hours for Hep C, was bought by Bristol-Myers Squibb (BMY) for $545 million.
Medgenics' market value is currently approximately $32 million and is clearly poised to be the leader in a multi billion dollar space, and I believe will far exceed the market value of the the companies mentioned in this article.
Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.
Medgenics' Disruptive Biotechnology Is One To Watch
By Ray Dirks
Medgenics (AMEX:MDGN) has, what I believe to be, the most disruptive biotechnologies of the last 20 years. This company is poised to own the 90 billion plus protein therapy market. We are talking about an enormous indication. Nomura (the big investment bank) calls this one of the top 10 biotech companies to watch over the next 5 years.
Currently the only form of treatment for Anemia is Epogen. Epogen is a synthetic version of the Erythropoietin that increases your red blood cell production. It is a $12B a year product monopolized by Amgen (AMGN) and affects nearly 1-2 billion worldwide.
When you are diagnosed with anemia, you go to your doctor. Your doctor is going to tell you that you have an abnormally low hemoglobin count and he will prescribe an epogen shot to get your hemoglobin into check and into a 10-12 count range which is considered healthy.
These shots cost about $25K a patient per year and are very painfully administered. What is going to occur is a spike of your Epo level from very low up to a 100x, which can lead to high blood pressure, a percentage increase of fatal cardiac events (as per the FDA) and all sorts of side effects. Over the next 48 hours, your Epo will crash back to a low level after which you will be treated with another shot of epogen, and this happens over and over again for the rest of your existence.
It's a terrible way to live, costs the system billions and is one of the worst FDA procedures on the market. In fact, Amgen has already received a black box warning because of the side effects and increased risks and is under major scrutiny for reimbursements issues as well.
The holy grail of Anemia treatment is to treat the patient without the infections and without that "spike" in Epo which is deadly in nature and provides a terrible quality of life.
Medgenics has developed a therapeutic system that can provide a dosage of the sustained levels (no spikes) of Erythropoietin naturally with literally no side effects and only two primary clinic visits for a fraction of the money. Let me explain exactly what they do and how they do it:
Medgenics takes a small piece of your skin about the size of a one inch toothpick from your abdomen.
This piece of tissue is then processed in a lab by inserting a small piece of DNA that teaches it to continuously produce the missing protein; which in this case is Erythropoietin.
Once this engineered piece of tissue is making the protein you are missing and at the levels needed, it is injected back under your skin and the result is a mini biobump that produced natural Epogen without the spikes, without the shots, without the side effects or dropouts. And for a fraction of the cost of Amgen's Epogen shots.
The Phase I/II study was done with 20 patients, all people diagnosed with anemia obviously. In all 20 patients you have biopumps producing Erythropoietin safely for as long as 36 months without any follow up, within that 10-12 count and without the injections and their dangerous spikes.
The most compelling thing about this story is that this isn't just a treatment for only Anemia; it is a platform that can be used on ALL protein therapies. In fact the company is entering Phase 1 for Hep C (Interferon) in Q2 of this year. Hepatitis C/Interferon is a 3.5 billion dollar Market this year. Gilead (GILD) paid 11 billion for a HEP C company… Inhibitex (INHX) was taken over for 2.5 billion (another Hep C company).
Medgenics already has an active program for Hemophilia developed with Baxter (BAX), and they will also be starting programs for:
* Growth Failure/growth hormone
* Multiple Sclerosis/Interferon Beta
* Diabetes/Insulin
* many others
In summary you have a company that has:
* $50 million invested
* the most disruptive technology in biotech today
* a management team that is arguably from top to bottom the best I have ever seen. Former head of Stanford Medicine, and an entrepreneur who sold his last company for $600 million
* Baxter collaboration and the potential for many more collaborations on multiple indications
* already finished Phase I/II and entering Phase IIB, this is NOT pre Clinical; this is tested and proven in human
* a ridiculously low valuation relative to its competitors.
* the potential of two probably partnerships in the near term
* a science which is not a one trick pony, it is a platform that covers every protein therapy which is more than a 91 billion dollar market
* largest shareholders are Joel Kanter and Isaac Blech. Joel has just sold Clarisonic for $600 million. Isaac has founded over 35 billion in companies
Enough of the science. Let me walk you through the value proposition of the investment. There are two public companies and one collaboration that I will use as comparisons.
Protalix (PLX) has a platform technology and is at about the same stage of clinical as Medgenics, but is only for an indication of 25,000 patients and trades at a 520 million dollar market cap.
Prolor (PBTH), which has a treatment which only increases the half lives of protein therapies, trades at a 300mm market cap.
And of course Zymogenetics which only has an interferon lasting slightly longer than the usual 5-6 hours for Hep C, was bought by Bristol-Myers Squibb (BMY) for $545 million.
Medgenics' market value is currently approximately $32 million and is clearly poised to be the leader in a multi billion dollar space, and I believe will far exceed the market value of the the companies mentioned in this article.
Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.
Wednesday, January 26, 2011
Scientists find connection between anemia and ITPA variants.
Scientists have discovered two functional variants in the inosine triphosphatase (ITPA) gene that protect patients with hepatitis C virus (HCV) against anemia brought on by antiviral treatment.
The ability to identify those patients protected against treatment-induced anemia will ensure completion of antiviral therapy and successful elimination of the virus.
Alessandra Mangia, from Casa Sollievo della Sofferenza Hospital in Italy, and colleagues evaluated the association between ITPA variants and anemia in a cohort of 238 Caucasian patients treated with variable pegIFN and weight-based doses of RBV. The research team found that the ITPA variants were strongly and independently associated with protection from anemia, but did not provide an increase in sustained virological response.
"When anemia develops only four weeks after the start of treatment, physicians are required to immediately reduce ribavirin dosages. This early reduction will affect the overall duration of treatment which, with the combination of pegIFN and RBV, lasts 24 weeks for patients infected with HCV genotypes two and 3 (G2/3) and 48 weeks for patients with HCV genotype one (G1) infection. Currently, only the use of the drug erythropoietin (EPO)-an expensive drug that due to its high cost cannot be reimbursed in several countries-might prevent unsuccessful antiviral treatment in these cases," explained Mangia.
"Our findings demonstrated that ITPA variants are strongly associated with protection from week four anemia and help us in selecting in advance who will need early ribavirin dose reduction and possibly supportive EPO treatment. This may lead to a more rational use of economical resources and to an individualized use of supportive EPO treatment," concluded Mangia.
"Patients with a genetic profile that included the two ITPA variants may be safely administered higher doses of RBV, increasing the likelihood of HCV elimination after treatment-an important finding given that to achieve viral clearance high dosages of RBV need to be used in the early phases of treatment."
A related study led by Fumitaka Suzuki, from Toranomon Hospital in Japan found similar results in its cohort of 61 Japanese patients with HCV.
Patients in this study received a triple therapy of pegINF, RBV and the protease inhibitor, telaprevir. Suzuki and colleagues found that ITPA variants impacted blood levels; however a sustained virological response could be achieved with careful monitoring of anemia and prompt adjustment of RBV dose. The authors suggest that future investigation of the influence of ITPA gene variants on RBV-induced anemia are needed on larger scales and on patients of various ethnicities.
The findings will be published in Hepatology.
The ability to identify those patients protected against treatment-induced anemia will ensure completion of antiviral therapy and successful elimination of the virus.
Alessandra Mangia, from Casa Sollievo della Sofferenza Hospital in Italy, and colleagues evaluated the association between ITPA variants and anemia in a cohort of 238 Caucasian patients treated with variable pegIFN and weight-based doses of RBV. The research team found that the ITPA variants were strongly and independently associated with protection from anemia, but did not provide an increase in sustained virological response.
"When anemia develops only four weeks after the start of treatment, physicians are required to immediately reduce ribavirin dosages. This early reduction will affect the overall duration of treatment which, with the combination of pegIFN and RBV, lasts 24 weeks for patients infected with HCV genotypes two and 3 (G2/3) and 48 weeks for patients with HCV genotype one (G1) infection. Currently, only the use of the drug erythropoietin (EPO)-an expensive drug that due to its high cost cannot be reimbursed in several countries-might prevent unsuccessful antiviral treatment in these cases," explained Mangia.
"Our findings demonstrated that ITPA variants are strongly associated with protection from week four anemia and help us in selecting in advance who will need early ribavirin dose reduction and possibly supportive EPO treatment. This may lead to a more rational use of economical resources and to an individualized use of supportive EPO treatment," concluded Mangia.
"Patients with a genetic profile that included the two ITPA variants may be safely administered higher doses of RBV, increasing the likelihood of HCV elimination after treatment-an important finding given that to achieve viral clearance high dosages of RBV need to be used in the early phases of treatment."
A related study led by Fumitaka Suzuki, from Toranomon Hospital in Japan found similar results in its cohort of 61 Japanese patients with HCV.
Patients in this study received a triple therapy of pegINF, RBV and the protease inhibitor, telaprevir. Suzuki and colleagues found that ITPA variants impacted blood levels; however a sustained virological response could be achieved with careful monitoring of anemia and prompt adjustment of RBV dose. The authors suggest that future investigation of the influence of ITPA gene variants on RBV-induced anemia are needed on larger scales and on patients of various ethnicities.
The findings will be published in Hepatology.
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