Benitec (BLT.AX) to Wind Down and Terminate Hep C Program
Update on TT-034 Hepatitis C Clinical Trial
- A conference call and webcast on this announcement and the Interim Report will be held on Tuesday March 1 at 8:30am AEDT (Australian Eastern Daylight time) and simultaneously on Monday February 29 at 4.30pm EST (US Eastern Standard time)
SYDNEY, Feb. 26, 2016 /PRNewswire/ -- Benitec Biopharma (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) announced today that it will wind-down its hepatitis C program and terminate it upon completion of patients in Cohort 4 in its Phase I/IIa clinical trial for TT-034.
Benitec's Board made the decision to discontinue the hepatitis C program following a review of the commercial opportunities for TT-034. A number of effective therapies have become available for the treatment of hepatitis C since Benitec commenced its clinical trial in January 2014. In recent months, several competitors have made improvements in the efficacy, delivery and success rates of their product treatments while continuing to reduce pricing and treatment duration.
As a result of this increasing competitive landscape and the time required to get TT-034 to market, TT-034 has generated limited and diminishing partnering interest from pharmaceutical companies. The Board has today concluded that the hepatitis C program does not offer the commercial value necessary to attract a worthwhile partnership deal and, as a result, does not warrant additional expenditure or focus of company resources beyond completion of patients in Cohort 4.
Completing the work with patients in Cohort 4 can provide Benitec with valuable data that supports and validates the company's ddRNAi technology platform and other pipeline programs. Benitec is committed to completing the collection of trial data and monitoring patients through the required long-term safety follow-up period. Final data supporting the primary and secondary endpoints of the study will be reported in CYQ4 2016 when the study is completed.
No significant financial obligation will arise from the discontinuance of the hepatitis C program.
Although the hepatitis C program is being discontinued, it is important to note that TT-034 has been shown to be safe and well tolerated, meeting the primary endpoint of the study and, as such, will assist in other programs.
Benitec's Chief Scientific Officer Dr. David Suhy said, "The TT-034 hepatitis C program is a First-in-Man trial. The data presented to date shows that TT-034 transduces hepatic tissues, expresses the anti-HCV shRNA and has a favorable safety profile with no significant adverse events reported relating to the administration of the study drug. Considering the novel characteristics of the drug, administered in a manner that cannot be withdrawn, we are pleased with the validity that TT-034 has shown in this trial. It has provided solid proof of concept for our ddRNAi platform and our other pipeline programs, particularly our hepatitis B program."
Benitec remains focused on advancing its other pipeline programs, including hepatitis B, age-related macular degeneration (AMD) and oculopharyngeal muscular dystrophy (OPMD). The company believes that each of these programs presents attractive commercial opportunities. In particular, the hepatitis B program is attracting considerable interest from pharmaceutical companies. Based on this interest and anticipated in vivo data, combined with a significant potential market opportunity, Benitec will now prioritise the hepatitis B program as its next candidate for clinical development.
Webcast Information and Conference Call:
The Company will host a live audio webcast and conference call on Tuesday, 1 March at 8:30am AEDT/ Monday, 29 February at 4.30pm EST, to provide an operational and financial update.
To access the live webcast please enter at http://services.choruscall.com/links/bntc160229 into your internet browser. Investors will be able to submit questions in writing via the webcast, to be addressed by Benitec's management during the call. To access the conference call please use the dial in details below.
Conference ID: 418337
US dial in: +1 855 624 0077
Australia dial in: 1800 908 299 or 1800 455 963
All other locations dial: +61 2 9007 8048
Shareholders are encouraged to use the webcast link, as conference call lines are limited.
An archive of the webcast will remain available on Benitec's website for 90 days beginning at approximately 5:30pm EST on 1 March 2016. For further information regarding Benitec and its activities, please contact the persons below, or visit the Benitec website at www.benitec.com
Showing posts with label TT-034. Show all posts
Showing posts with label TT-034. Show all posts
Friday, February 26, 2016
Thursday, October 22, 2015
AASLD - Benitec To Present Interim Data of TT-034 Phase I/IIa Study At Liver Meeting
Benitec Biopharma's Abstract Accepted for Presentation at the AASLD Liver Meeting 2015
SYDNEY, Oct. 22, 2015 /PRNewswire/ -- Benitec Biopharma Limited (NASDAQ: BNTC; NASDAQ: BNTCW; ASX: BLT) is pleased to announce that clinical data on its Phase I/IIa study of TT-034 for hepatitis C will be presented in the 'late-breaking poster' session at The Liver Meeting® 2015, the 66th Annual Meeting of the American Association for the Study of Liver Disease (AASLD) being held in San Francisco on November 13-17, 2015.
The abstract, which has been published on the conference website, details interim results from patients in the first three cohorts in Benitec's Phase I/IIa study of TT-034, a DNA-directed RNA interference agent (ddRNAi).
The abstract, which has been published on the conference website, details interim results from patients in the first three cohorts in Benitec's Phase I/IIa study of TT-034, a DNA-directed RNA interference agent (ddRNAi).
The primary endpoint that this study is monitoring is safety of this first-in-man gene therapy-based gene silencing drug. Key findings include:
The three doses of TT-034 administered to date have been well tolerated in human subjects infected with the hepatitis C virus (HCV) and there have been no reported serious adverse events related to administration of the study drug;
The three doses of TT-034 administered to date have been well tolerated in human subjects infected with the hepatitis C virus (HCV) and there have been no reported serious adverse events related to administration of the study drug;
The initial dose (4E10 vg/kg) resulted in very low levels of transduction as expected.
The second dose (1.25E11 vg/kg) resulted in the detection of substantially higher levels of TT-034 in the hepatocytes, the predominant cell type in the liver, yielding 0.48, 3.65 and 10.44 copies of TT-034
The second dose (1.25E11 vg/kg) resulted in the detection of substantially higher levels of TT-034 in the hepatocytes, the predominant cell type in the liver, yielding 0.48, 3.65 and 10.44 copies of TT-034
DNA per cell in the three patients respectively;
The first subject administered with the third dose (4.00E11 vg/kg) had 17.74 copies of TT-034 per cell, indicating that a significant portion of their hepatocytes may have been transduced, and expression of anti-HCV shRNAs was clearly detected in the transduced hepatocytes.
Benitec's Chief Scientific Officer, Dr David Suhy said, "We are pleased to be given the opportunity to present this interim data at the AASLD Liver Meeting next month. The results show that a single infusion of TT-034 is reaching the liver and that it has a very favourable safety profile at the doses tested to date. In the patient that has received the highest dose to date, we are able to detect that anti-HCV shRNAs have been expressed in the liver without any drug-related serious adverse effects, indicating that so far the trial is achieving its primary outcome. We are pleased with the progress of the trial to date."
To read the full abstract, please visit: http://www.aasld.org/sites/default/files/TLM-2015-LakeBreakingAbstracts.pdf
This announcement has been prepared for publication in Australia and may not be released in the United States. This announcement does not constitute an offer to sell, or a solicitation of an offer to buy, securities in the United States or any other jurisdiction.
For further information regarding Benitec and its activities, please contact the persons below, or visit the Benitec website at www.benitec.com.
The first subject administered with the third dose (4.00E11 vg/kg) had 17.74 copies of TT-034 per cell, indicating that a significant portion of their hepatocytes may have been transduced, and expression of anti-HCV shRNAs was clearly detected in the transduced hepatocytes.
Benitec's Chief Scientific Officer, Dr David Suhy said, "We are pleased to be given the opportunity to present this interim data at the AASLD Liver Meeting next month. The results show that a single infusion of TT-034 is reaching the liver and that it has a very favourable safety profile at the doses tested to date. In the patient that has received the highest dose to date, we are able to detect that anti-HCV shRNAs have been expressed in the liver without any drug-related serious adverse effects, indicating that so far the trial is achieving its primary outcome. We are pleased with the progress of the trial to date."
To read the full abstract, please visit: http://www.aasld.org/sites/default/files/TLM-2015-LakeBreakingAbstracts.pdf
This announcement has been prepared for publication in Australia and may not be released in the United States. This announcement does not constitute an offer to sell, or a solicitation of an offer to buy, securities in the United States or any other jurisdiction.
For further information regarding Benitec and its activities, please contact the persons below, or visit the Benitec website at www.benitec.com.
Wednesday, September 16, 2015
Benitec Initiates a Fourth Site in Hepatitis C Clinical Trial
Benitec Initiates a Fourth Site in Hepatitis C Clinical Trial
SYDNEY, Sept. 16, 2015 /PRNewswire/ -- Benitec Biopharma Limited (NASDAQ: BNTC; NASDAQ: BNTCW; ASX: BLT) a clinical-stage biotechnology company developing innovative therapeutics based on its gene-silencing technology, DNA-directed RNA interference (ddRNAi), is pleased to announce it has initiated a new site for its ongoing Phase 1/2a TT-034 trial at the Methodist Health System Clinical Research Institute in Dallas, Texas. The site has commenced pre-screening hepatitis C patients and is led by principal investigator Dr. Parvez Mantry, a gastroenterologist and hepatologist.
This brings the total number of trial sites to four, with Benitec already having established sites at the Duke Clinical Research Institute, the University of California San Diego and the Texas Liver Institute.
Benitec CEO and Managing Director Dr. Peter French said, "We are pleased to welcome a fourth site to join our first-in-man trial of TT-034, an innovative therapeutic based on Benitec's gene silencing technology, ddRNAi. The addition of this site reflects the growing interest from the medical community in Benitec's potentially transformational approach to treating and curing hepatitis C. Recruitment and dosing for the trial is proceeding well."
More detail on the TT-034 trial: TT-034 is a ddRNAi-based therapeutic, designed to treat and potentially cure hepatitis C (HCV) with a single administration. TT-034 targets the hepatitis C viral RNA at three separate, highly conserved sites. As such it acts as a "triple therapy" even though it is a monotherapy, and minimises the ability of the virus to mutate and escape the therapy. Once it reaches the liver cells it enters the nucleus and produces three separate short hairpin RNAs continuously for the lifetime of the cell. Thus it has the potential to not only treat the existing HCV infection but to guard against reinfection for months to years without the need to re-treat. It has been extensively tested in pre-clinical in vivo studies and no adverse effects were seen at any therapeutic dose. However, as it is regulated as a gene therapy, the trial design is to primarily ensure that treatment with TT-034 is safe, hence the gradual dose escalation.
SYDNEY, Sept. 16, 2015 /PRNewswire/ -- Benitec Biopharma Limited (NASDAQ: BNTC; NASDAQ: BNTCW; ASX: BLT) a clinical-stage biotechnology company developing innovative therapeutics based on its gene-silencing technology, DNA-directed RNA interference (ddRNAi), is pleased to announce it has initiated a new site for its ongoing Phase 1/2a TT-034 trial at the Methodist Health System Clinical Research Institute in Dallas, Texas. The site has commenced pre-screening hepatitis C patients and is led by principal investigator Dr. Parvez Mantry, a gastroenterologist and hepatologist.
This brings the total number of trial sites to four, with Benitec already having established sites at the Duke Clinical Research Institute, the University of California San Diego and the Texas Liver Institute.
Benitec CEO and Managing Director Dr. Peter French said, "We are pleased to welcome a fourth site to join our first-in-man trial of TT-034, an innovative therapeutic based on Benitec's gene silencing technology, ddRNAi. The addition of this site reflects the growing interest from the medical community in Benitec's potentially transformational approach to treating and curing hepatitis C. Recruitment and dosing for the trial is proceeding well."
More detail on the TT-034 trial: TT-034 is a ddRNAi-based therapeutic, designed to treat and potentially cure hepatitis C (HCV) with a single administration. TT-034 targets the hepatitis C viral RNA at three separate, highly conserved sites. As such it acts as a "triple therapy" even though it is a monotherapy, and minimises the ability of the virus to mutate and escape the therapy. Once it reaches the liver cells it enters the nucleus and produces three separate short hairpin RNAs continuously for the lifetime of the cell. Thus it has the potential to not only treat the existing HCV infection but to guard against reinfection for months to years without the need to re-treat. It has been extensively tested in pre-clinical in vivo studies and no adverse effects were seen at any therapeutic dose. However, as it is regulated as a gene therapy, the trial design is to primarily ensure that treatment with TT-034 is safe, hence the gradual dose escalation.
Monday, May 25, 2015
Benitec Biopharma signs manufacturing deal for hepatitis C treatment
Benitec Biopharma signs manufacturing deal for hepatitis C treatment
Monday, May 25, 2015 by Proactive Investors
Benitec Biopharma (ASX:BLT) has entered into an agreement with Maryland-based Omnia Biologics to manufacture material for its current first-in-man clinical trial for its TT-034 hepatitis C treatment.
This ensures the company has enough clinical material to complete the current trial.
The company is also moving to establish its own scalable manufacturing process in collaboration with third parties to supply large markets it is targeting.
Hepatitis C Treatment
TT-034 is a ddRNAi-based therapeutic that is designed to treat and potentially cure hepatitis C with a single administration.
It targets the hepatitis C viral RNA at three separate, highly conserved sites, which minimises the ability of the virus to mutate and escape the therapy.
Once it reaches the liver cells, it enters the nucleus and produces three separate short hairpin RNAs continuously for the live of the cell.
This has the potential to guard against reinfection for months to years without the need to re-treat.
Proactive Investors Australia is the market leader in producing news, articles and research reports on ASX emerging companies with distribution in Australia, UK, North America and Hong Kong / China.
Monday, May 25, 2015 by Proactive Investors
Benitec Biopharma (ASX:BLT) has entered into an agreement with Maryland-based Omnia Biologics to manufacture material for its current first-in-man clinical trial for its TT-034 hepatitis C treatment.
This ensures the company has enough clinical material to complete the current trial.
The company is also moving to establish its own scalable manufacturing process in collaboration with third parties to supply large markets it is targeting.
Hepatitis C Treatment
TT-034 is a ddRNAi-based therapeutic that is designed to treat and potentially cure hepatitis C with a single administration.
It targets the hepatitis C viral RNA at three separate, highly conserved sites, which minimises the ability of the virus to mutate and escape the therapy.
Once it reaches the liver cells, it enters the nucleus and produces three separate short hairpin RNAs continuously for the live of the cell.
This has the potential to guard against reinfection for months to years without the need to re-treat.
Proactive Investors Australia is the market leader in producing news, articles and research reports on ASX emerging companies with distribution in Australia, UK, North America and Hong Kong / China.
Wednesday, April 29, 2015
Benitec Doses Fifth Patient And Initiates Additional Trial Site For Hepatitis C Trial
Benitec Biopharma Doses Fifth Patient And Initiates Additional Trial Site For Hepatitis C Trial
- Third patient in cohort two dosed
- Total of five patients now dosed
- Third clinical trial site initiated - the Texas Liver Institute
- Future reporting of this trial to be on completion of trial or as a result of a material event
- Total of five patients now dosed
- Third clinical trial site initiated - the Texas Liver Institute
- Future reporting of this trial to be on completion of trial or as a result of a material event
SYDNEY, April 29, 2015 /PRNewswire/ -- Benitec Biopharma (ASX: BLT; OTCPK: BTEBY), a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or "expressed RNAi," is pleased to advise that the fifth patient in the company's 'first in man', Phase I/IIa dose escalation clinical trial of TT-034 for hepatitis C virus (HCV) infection, has today, been dosed at the Duke Clinical Research Unit. The fifth patient is the third and final patient to be dosed in Cohort 2.
The three patients in Cohort 2 received a dose of TT-034 of 1.25 x 10^11 vg/kg, a concentration that is a half log higher than the doses administered in Cohort 1. In line with the trial's primary endpoint of safety, this dose level is still below the concentration expected to inhibit HCV viral replication and data from the second dosing cohort is therefore expected to serve primarily as a further safety assessment.
As with previous patients, the newly dosed patient will be monitored for six weeks and this data will then be reviewed by the Data Safety Monitoring Board (DSMB). Benitec is currently screening patients for inclusion in cohort 3 in anticipation of the DSMB's review.
Benitec has now initiated a third site, the Texas Liver Institute in San Antonio, Texas, and they have started to pre-screen patients for the TT-034 trial.
Following completion of the first two patient cohorts and initiation of a third trial site, Benitec will now move to conventional clinical trial reporting for cohorts 3 through to 5 of the dose escalation study. The company will provide an update to investors (via ASX announcement) should a material event occur or when the trial is completed.
About TT-034
TT-034 is a ddRNAi-based therapeutic, designed to treat and potentially cure hepatitis C (HCV) with a single administration. TT-034 targets the hepatitis C viral RNA at three separate, highly conserved sites. As such it acts as a "triple therapy" even though it is a monotherapy, and minimises the ability of the virus to mutate and escape the therapy. Once it reaches the liver cells it enters the nucleus and produces three separate short hairpin RNAs continuously for the life time of the cell. Thus it has the potential to not only treat the existing HCV infection but to guard against reinfection for months to years without the need to re-treat. It has been extensively tested in pre-clinical in vivo studies and no adverse effects were seen at any therapeutic dose. However, as it is regulated as a gene therapy, the trial design is to primarily ensure that treatment with TT-034 is safe, hence the gradual dose escalation.
The three patients in Cohort 2 received a dose of TT-034 of 1.25 x 10^11 vg/kg, a concentration that is a half log higher than the doses administered in Cohort 1. In line with the trial's primary endpoint of safety, this dose level is still below the concentration expected to inhibit HCV viral replication and data from the second dosing cohort is therefore expected to serve primarily as a further safety assessment.
As with previous patients, the newly dosed patient will be monitored for six weeks and this data will then be reviewed by the Data Safety Monitoring Board (DSMB). Benitec is currently screening patients for inclusion in cohort 3 in anticipation of the DSMB's review.
Benitec has now initiated a third site, the Texas Liver Institute in San Antonio, Texas, and they have started to pre-screen patients for the TT-034 trial.
Following completion of the first two patient cohorts and initiation of a third trial site, Benitec will now move to conventional clinical trial reporting for cohorts 3 through to 5 of the dose escalation study. The company will provide an update to investors (via ASX announcement) should a material event occur or when the trial is completed.
About TT-034
TT-034 is a ddRNAi-based therapeutic, designed to treat and potentially cure hepatitis C (HCV) with a single administration. TT-034 targets the hepatitis C viral RNA at three separate, highly conserved sites. As such it acts as a "triple therapy" even though it is a monotherapy, and minimises the ability of the virus to mutate and escape the therapy. Once it reaches the liver cells it enters the nucleus and produces three separate short hairpin RNAs continuously for the life time of the cell. Thus it has the potential to not only treat the existing HCV infection but to guard against reinfection for months to years without the need to re-treat. It has been extensively tested in pre-clinical in vivo studies and no adverse effects were seen at any therapeutic dose. However, as it is regulated as a gene therapy, the trial design is to primarily ensure that treatment with TT-034 is safe, hence the gradual dose escalation.
Tuesday, April 7, 2015
Benitec Provides Update On Phase I/IIa clinical trial of TT-034 for hepatitis C
Benitec Provides Update On TT-034 Trial
Information contained on this page is provided by an independent third-party content provider. WorldNow and this Station make no warranties or representations in connection therewith. If you have any questions or comments about this page please contact pressreleases@worldnow.com.
SOURCE Benitec Biopharma Limited
- shRNA detected in liver biopsies at levels in line with expectations
- no treatment-related adverse events reported
SYDNEY, April 7, 2015 /PRNewswire/ -- Benitec Biopharma (ASX:BLT; OTCPK:BTEBY), a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or "expressed RNAi," is pleased to advise that laboratory results from liver biopsies in the company's 'first in man', Phase I/IIa clinical trial of TT-034 for hepatitis C confirmed that the trial is proceeding according to expectations.
TT-034 works by producing (in the liver) three silencing short hairpin RNAs (shRNAs), each responsible for targeting a different part of the hepatitis C virus' genome. The expression of the three shRNAs in patients' liver cells is thus an essential requirement for TT-034 to exert a clinical reduction of hepatitis C viral load. The most recent assay of the biopsies confirmed this expression occurred in all three patients dosed to date. These results were obtained from the biopsies of the first two patients in cohort 1 and the first patient in cohort 2. The second patient in cohort 2 has not yet been biopsied, and the third patient in cohort 2 is yet to be dosed due to a personal issue.
In cohorts 1 and 2, the dose of TT-034 is sub-therapeutic and, therefore, the amount of shRNA produced will not result in reduction of hepatitis C viral load.
Furthermore, to date there have been no treatment-related serious adverse effects (SAEs) in any of the four patients dosed.
More detail on the TT-034 trial
TT-034 is a ddRNAi-based therapeutic, designed to treat and potentially cure hepatitis C (HCV) with a single administration. TT-034 targets the hepatitis C viral RNA at three separate, highly conserved sites. As such it acts as a "triple therapy" even though it is a monotherapy, and minimises the ability of the virus to mutate and escape the therapy. Once it reaches the liver cells it enters the nucleus and produces three separate short hairpin RNAs continuously for the life time of the cell. Thus it has the potential to not only treat the existing HCV infection but to guard against reinfection for months to years without the need to re-treat. It has been extensively tested in pre-clinical in vivo studies and no adverse effects were seen at any therapeutic dose. However, as it is regulated as a gene therapy, the trial design is to primarily ensure that treatment with TT-034 is safe, hence the gradual dose escalation.
About Benitec Biopharma Limited
Benitec Biopharma Limited is an ASX-listed biotechnology company (ASX:BLT; OTC:BTEBY) which has developed a patented gene-silencing technology called ddRNAi or 'expressed RNAi'. Based in Sydney, Australia with labs in Hayward CA (USA) and collaborators and licensees around the world, the company is developing ddRNAi-based therapeutics for chronic and life-threatening human conditions including Hepatitis C and B, drug resistant lung cancer and wet age-related macular degeneration. Benitec has also licensed ddRNAi to other biopharmaceutical companies for applications including HIV/AIDS, Huntington's disease, chronic neuropathic pain and retinitis pigmentosa.
For further information regarding Benitec and its activities, please contact the persons below, or visit the Benitec website at www.benitec.com.
Information contained on this page is provided by an independent third-party content provider. WorldNow and this Station make no warranties or representations in connection therewith. If you have any questions or comments about this page please contact pressreleases@worldnow.com.
SOURCE Benitec Biopharma Limited
- shRNA detected in liver biopsies at levels in line with expectations
- no treatment-related adverse events reported
SYDNEY, April 7, 2015 /PRNewswire/ -- Benitec Biopharma (ASX:BLT; OTCPK:BTEBY), a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or "expressed RNAi," is pleased to advise that laboratory results from liver biopsies in the company's 'first in man', Phase I/IIa clinical trial of TT-034 for hepatitis C confirmed that the trial is proceeding according to expectations.
TT-034 works by producing (in the liver) three silencing short hairpin RNAs (shRNAs), each responsible for targeting a different part of the hepatitis C virus' genome. The expression of the three shRNAs in patients' liver cells is thus an essential requirement for TT-034 to exert a clinical reduction of hepatitis C viral load. The most recent assay of the biopsies confirmed this expression occurred in all three patients dosed to date. These results were obtained from the biopsies of the first two patients in cohort 1 and the first patient in cohort 2. The second patient in cohort 2 has not yet been biopsied, and the third patient in cohort 2 is yet to be dosed due to a personal issue.
In cohorts 1 and 2, the dose of TT-034 is sub-therapeutic and, therefore, the amount of shRNA produced will not result in reduction of hepatitis C viral load.
Furthermore, to date there have been no treatment-related serious adverse effects (SAEs) in any of the four patients dosed.
More detail on the TT-034 trial
TT-034 is a ddRNAi-based therapeutic, designed to treat and potentially cure hepatitis C (HCV) with a single administration. TT-034 targets the hepatitis C viral RNA at three separate, highly conserved sites. As such it acts as a "triple therapy" even though it is a monotherapy, and minimises the ability of the virus to mutate and escape the therapy. Once it reaches the liver cells it enters the nucleus and produces three separate short hairpin RNAs continuously for the life time of the cell. Thus it has the potential to not only treat the existing HCV infection but to guard against reinfection for months to years without the need to re-treat. It has been extensively tested in pre-clinical in vivo studies and no adverse effects were seen at any therapeutic dose. However, as it is regulated as a gene therapy, the trial design is to primarily ensure that treatment with TT-034 is safe, hence the gradual dose escalation.
About Benitec Biopharma Limited
Benitec Biopharma Limited is an ASX-listed biotechnology company (ASX:BLT; OTC:BTEBY) which has developed a patented gene-silencing technology called ddRNAi or 'expressed RNAi'. Based in Sydney, Australia with labs in Hayward CA (USA) and collaborators and licensees around the world, the company is developing ddRNAi-based therapeutics for chronic and life-threatening human conditions including Hepatitis C and B, drug resistant lung cancer and wet age-related macular degeneration. Benitec has also licensed ddRNAi to other biopharmaceutical companies for applications including HIV/AIDS, Huntington's disease, chronic neuropathic pain and retinitis pigmentosa.
For further information regarding Benitec and its activities, please contact the persons below, or visit the Benitec website at www.benitec.com.
Wednesday, March 11, 2015
Benitec Biopharma Doses Fourth Patient In Hepatitis C Trial
Benitec Biopharma Doses Fourth Patient In Hepatitis C Trial
SYDNEY, March 11, 2015 /PRNewswire/ -- Benitec Biopharma (ASX: BLT; OTCPK: BTEBY), a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or "expressed RNAi," today announced that the fourth patient in the company's Phase I/IIa dose escalation clinical trial of its lead program TT-034 for treating hepatitis C was dosed at the Duke Clinical Research Unit. This is the second patient to be dosed in Cohort Two, with the third and final patient in Cohort Two well advanced in their preparation for dosing.
As previously announced, the parallel dosing of these patients follows a positive recommendation from the DSMB's review of the safety data from the first patient in this cohort.
All three patients in Cohort Two receive a dose of 1.25 x 10^11 vg/kg of TT-034, a concentration that is a half-log higher than the dose administered in Cohort One. This dose level is still below the concentration expected to inhibit hepatitis C viral replication and therefore data from Cohort Two are expected to serve primarily as a further safety assessment.
About TT-034
TT-034 is a ddRNAi-based therapeutic, designed to treat and potentially cure hepatitis C (HCV) with a single administration. TT-034 targets the hepatitis C viral RNA at three separate, highly conserved sites. As such it acts as a "triple therapy" even though it is a monotherapy, and minimizes the ability of the virus to mutate and escape the therapy. Once it reaches the liver cells it enters the nucleus and produces three separate short hairpin RNAs continuously for the lifetime of the cell. Thus it has the potential not only to treat the existing HCV infection, but also to guard against reinfection for months to years without the need to re-treat. It has been tested extensively in pre-clinical in vivo studies and no adverse effects were seen at any therapeutic dose. However, as it is regulated as a gene therapy, the trial design is primarily to ensure that treatment with TT-034 is safe, hence the gradual dose escalation.
About Benitec Biopharma Limited:
Benitec Biopharma Limited is an ASX-listed biotechnology company (ASX:BLT; OTC:BTEBY) that has developed a patented gene-silencing technology called ddRNAi or 'expressed RNAi'. Based in Sydney, Australia, with labs inHayward, CA (USA), and collaborators and licensees around the world, the company is developing ddRNAi-based therapeutics for chronic and life-threatening human conditions including Hepatitis C and B, drug resistant lung cancer and wet Age-related Macular Degeneration (AMD). Benitec has also licensed ddRNAi to other biopharmaceutical companies for applications including HIV/AIDS, Huntington's disease, chronic neuropathic pain and retinitis pigmentosa.
For further information regarding Benitec and its activities, please contact the persons below, or visit the Benitec website at www.benitec.com.
SYDNEY, March 11, 2015 /PRNewswire/ -- Benitec Biopharma (ASX: BLT; OTCPK: BTEBY), a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or "expressed RNAi," today announced that the fourth patient in the company's Phase I/IIa dose escalation clinical trial of its lead program TT-034 for treating hepatitis C was dosed at the Duke Clinical Research Unit. This is the second patient to be dosed in Cohort Two, with the third and final patient in Cohort Two well advanced in their preparation for dosing.
As previously announced, the parallel dosing of these patients follows a positive recommendation from the DSMB's review of the safety data from the first patient in this cohort.
All three patients in Cohort Two receive a dose of 1.25 x 10^11 vg/kg of TT-034, a concentration that is a half-log higher than the dose administered in Cohort One. This dose level is still below the concentration expected to inhibit hepatitis C viral replication and therefore data from Cohort Two are expected to serve primarily as a further safety assessment.
About TT-034
TT-034 is a ddRNAi-based therapeutic, designed to treat and potentially cure hepatitis C (HCV) with a single administration. TT-034 targets the hepatitis C viral RNA at three separate, highly conserved sites. As such it acts as a "triple therapy" even though it is a monotherapy, and minimizes the ability of the virus to mutate and escape the therapy. Once it reaches the liver cells it enters the nucleus and produces three separate short hairpin RNAs continuously for the lifetime of the cell. Thus it has the potential not only to treat the existing HCV infection, but also to guard against reinfection for months to years without the need to re-treat. It has been tested extensively in pre-clinical in vivo studies and no adverse effects were seen at any therapeutic dose. However, as it is regulated as a gene therapy, the trial design is primarily to ensure that treatment with TT-034 is safe, hence the gradual dose escalation.
About Benitec Biopharma Limited:
Benitec Biopharma Limited is an ASX-listed biotechnology company (ASX:BLT; OTC:BTEBY) that has developed a patented gene-silencing technology called ddRNAi or 'expressed RNAi'. Based in Sydney, Australia, with labs inHayward, CA (USA), and collaborators and licensees around the world, the company is developing ddRNAi-based therapeutics for chronic and life-threatening human conditions including Hepatitis C and B, drug resistant lung cancer and wet Age-related Macular Degeneration (AMD). Benitec has also licensed ddRNAi to other biopharmaceutical companies for applications including HIV/AIDS, Huntington's disease, chronic neuropathic pain and retinitis pigmentosa.
For further information regarding Benitec and its activities, please contact the persons below, or visit the Benitec website at www.benitec.com.
Wednesday, January 7, 2015
Benitec Advances Hepatitis C Clinical Trial
Benitec Advances Hepatitis C Clinical Trial
SYDNEY, Jan. 7, 2015 /PRNewswire/ -- Benitec Biopharma Limited (ASX: BLT, OTC: BTEBY) is pleased to advise that the third patient in its Phase I/IIa clinical trial of TT-034 for hepatitis C was dosed earlier today at the Duke Clinical Research Unit (USA). This is a significant step for this "first in man" study, and follows review of the collective data from the first two patients by the independent Data Safety Monitoring Board (DSMB). The DSMB determined that the patients from the first dosing cohort were clear of any significant treatment-related adverse events.
The newly dosed patient is the first to receive the increased dose of TT-034 (1.25 x 10^11 vg/kg, a concentration that is a half log higher than the doses administered in the first cohort). While TT-034 is designed as a potential "one-shot" cure for hepatitis C, the current dose is still below that expected to inhibit viral replication and data from the second dosing cohort are therefore expected to serve primarily as a further safety assessment.
As with previous patients, the newly dosed patient will be monitored for six weeks and results will be reviewed by the DSMB. Should the results indicate appropriate safety outcomes, the DSMB is expected to recommend that the remaining two patients in the second cohort be dosed. It is aimed to dose both at approximately the same time. The trial sites at Duke Clinical Research Unit and University of California San Diego have identified a number of patients who have passed initial screening who can be prepared in anticipation of this outcome.
About TT-034
TT-034 is a ddRNAi-based therapeutic, designed to treat and potentially cure hepatitis C (HCV) with a single administration. TT-034 targets the hepatitis C viral RNA at three separate, highly conserved sites. As such it acts as a "triple therapy" even though it is a monotherapy, and minimizes the ability of the virus to mutate and escape the therapy. Once it reaches the liver cells, it enters the nucleus and produces three separate short hairpin RNAs continuously for the lifetime of the cell. Thus TT-034 has the potential to not only treat the existing HCV infection, but also to guard against reinfection for months to years without the need to re-treat. TT-034 safety and efficacy has been tested extensively in pre-clinical in vivo studies with no adverse effects observed at therapeutic doses.
worldnow.com.
SYDNEY, Jan. 7, 2015 /PRNewswire/ -- Benitec Biopharma Limited (ASX: BLT, OTC: BTEBY) is pleased to advise that the third patient in its Phase I/IIa clinical trial of TT-034 for hepatitis C was dosed earlier today at the Duke Clinical Research Unit (USA). This is a significant step for this "first in man" study, and follows review of the collective data from the first two patients by the independent Data Safety Monitoring Board (DSMB). The DSMB determined that the patients from the first dosing cohort were clear of any significant treatment-related adverse events.
The newly dosed patient is the first to receive the increased dose of TT-034 (1.25 x 10^11 vg/kg, a concentration that is a half log higher than the doses administered in the first cohort). While TT-034 is designed as a potential "one-shot" cure for hepatitis C, the current dose is still below that expected to inhibit viral replication and data from the second dosing cohort are therefore expected to serve primarily as a further safety assessment.
As with previous patients, the newly dosed patient will be monitored for six weeks and results will be reviewed by the DSMB. Should the results indicate appropriate safety outcomes, the DSMB is expected to recommend that the remaining two patients in the second cohort be dosed. It is aimed to dose both at approximately the same time. The trial sites at Duke Clinical Research Unit and University of California San Diego have identified a number of patients who have passed initial screening who can be prepared in anticipation of this outcome.
About TT-034
TT-034 is a ddRNAi-based therapeutic, designed to treat and potentially cure hepatitis C (HCV) with a single administration. TT-034 targets the hepatitis C viral RNA at three separate, highly conserved sites. As such it acts as a "triple therapy" even though it is a monotherapy, and minimizes the ability of the virus to mutate and escape the therapy. Once it reaches the liver cells, it enters the nucleus and produces three separate short hairpin RNAs continuously for the lifetime of the cell. Thus TT-034 has the potential to not only treat the existing HCV infection, but also to guard against reinfection for months to years without the need to re-treat. TT-034 safety and efficacy has been tested extensively in pre-clinical in vivo studies with no adverse effects observed at therapeutic doses.
Tuesday, February 18, 2014
University of Westminster develops groundbreaking method to test hepatitis C cure
Researchers at the University of Westminster have developed a groundbreaking method which can be used to test a new innovative cure for hepatitis C, a liver disease caused by the hepatitis C virus (HCV).
Biomedical Sciences 17 February 2014
The cure is the first of its kind ever to be tested in humans and comes in the form of a drug based on gene therapy which is under development by the Australian company Benitec Biopharma.
Around 150 million people worldwide are infected with hepatitis C, and more than 350,000 people die every year from hepatitis C related liver diseases. Hepatitis C is one of the leading causes of liver cirrhosis and cancer, and one of the most common and seriously infectious conditions in the world (according to the World Health Organisation (WHO)).
Although treatments are already available for hepatitis C, these are lengthy, have low chances of success, cause significant side-effects, or the virus is already becoming resistant. The new drug, TT-034, developed by Benitec Biopharma, is based on the biological mechanism for which the Nobel Prize in Physiology or Medicine was awarded in 2006. Unlike anything else currently available to patients, this treatment works with a single injection to directly destroy the hepatitis C virus and remove the infection. The drug is currently undergoing clinical trials in the US with results expected in the coming months.
Dr Sterghios A. Moschos, MSB, Director of Westminster Genomic Services at the University of Westminster, developed the comprehensive and innovative method by adapting state-of-the-art genome sequencing technologies to show exactly how the new drug works. The research was conducted in collaboration with the European Bioinformatics Institute and Benitec Biopharma.
Dr Moschos said: “Our entirely new method to test the new drug has had a major impact on building robust confidence in this innovative therapy. For the first time ever we have shown that there are more ways to hit the hepatitis C infection than previously thought possible, and that this treatment works like a combination of multiple drugs. Our approach has helped Benitec Biopharma, to obtain permission to start clinical trials much earlier than we expected. This is unprecedented for gene therapy, particularly for a disease for which treatments already exist.”
For further information you can read the research paper published on Molecular Therapy Nucleic Acids at nature.com.
Source
Biomedical Sciences 17 February 2014
The cure is the first of its kind ever to be tested in humans and comes in the form of a drug based on gene therapy which is under development by the Australian company Benitec Biopharma.
Around 150 million people worldwide are infected with hepatitis C, and more than 350,000 people die every year from hepatitis C related liver diseases. Hepatitis C is one of the leading causes of liver cirrhosis and cancer, and one of the most common and seriously infectious conditions in the world (according to the World Health Organisation (WHO)).
Although treatments are already available for hepatitis C, these are lengthy, have low chances of success, cause significant side-effects, or the virus is already becoming resistant. The new drug, TT-034, developed by Benitec Biopharma, is based on the biological mechanism for which the Nobel Prize in Physiology or Medicine was awarded in 2006. Unlike anything else currently available to patients, this treatment works with a single injection to directly destroy the hepatitis C virus and remove the infection. The drug is currently undergoing clinical trials in the US with results expected in the coming months.
Dr Sterghios A. Moschos, MSB, Director of Westminster Genomic Services at the University of Westminster, developed the comprehensive and innovative method by adapting state-of-the-art genome sequencing technologies to show exactly how the new drug works. The research was conducted in collaboration with the European Bioinformatics Institute and Benitec Biopharma.
Dr Moschos said: “Our entirely new method to test the new drug has had a major impact on building robust confidence in this innovative therapy. For the first time ever we have shown that there are more ways to hit the hepatitis C infection than previously thought possible, and that this treatment works like a combination of multiple drugs. Our approach has helped Benitec Biopharma, to obtain permission to start clinical trials much earlier than we expected. This is unprecedented for gene therapy, particularly for a disease for which treatments already exist.”
For further information you can read the research paper published on Molecular Therapy Nucleic Acids at nature.com.
Source
Tuesday, January 14, 2014
Benitec Proceeds With ‘First In Man’ Trial for TT - 034: A Single Injection To Treat Hepatitis C
Hepatitis C Trial To Proceed
1/14/2014 9:04:01 AM
Sydney Australia, 14th January 2014: RNAi - based therapeutics company Benitec Biopharma Limited (ASX: BLT) today announced that the US Food and Drug Administration (FDA) has advised the Company that it may proceed with it s ‘first in man’ clinical trial for TT - 034, a ddRNAi - based therapeutic, designed to treat Hepatitis C with a single injection .
This follows the FDA’s review of Benitec’s Investigational New Drug (IND) application, which was filed on 6 December 2013.
Benitec Biopharma’s CEO and Managing Director, Peter French said, “We are very pleased with this outcome which establishes Benitec as a clinical stage company .”
Excerpt From:March 2013/ Benitec Selects the Duke Clinical Research Unit as a Site for Hepatitis C Phase I/II Clinical Trial
About TT-034
For further information, please contact the persons outlined below, or visit the Benitec website at www.benitec.com
Company
Carl Stubbings
Chief Business Officer
Tel: +61 (2) 9555 6986
Email:
cstubbings@benitec.com
Investor relations
Jane Lowe
Buchan Consulting
Te
l: +61 (2) 9237 2807
Email:
jlowe@buchanwe.com.au
About Benitec Biopharma Limited:
Benitec Biopharma Limited is an ASX - listed biotechnology company (ASX Code: BLT) based in Sydney, Australia. The company has a pipeline of in - house and partnered therapeutic programs based on its patented ge ne - silencing technology, ddRNAi . Benitec is developing treatments for chronic and life - threatening human conditions such as Hepatitis C , Hepatitis B, wet age - related macular degeneration, cancer - associated pain, drug resistant lung cancer and oculopharyngeal muscular dystrophy based on this technology. In addition, Benitec has licensed ddRNAi technology to other biopharmaceutical companies who are progressing their programs t owards the clinic for applications including HIV/AIDS, retinitis pigmentosa and Huntington’s disease. For more information on Benitec refer to the Company’s website at www.benitec.com .
1/14/2014 9:04:01 AM
Sydney Australia, 14th January 2014: RNAi - based therapeutics company Benitec Biopharma Limited (ASX: BLT) today announced that the US Food and Drug Administration (FDA) has advised the Company that it may proceed with it s ‘first in man’ clinical trial for TT - 034, a ddRNAi - based therapeutic, designed to treat Hepatitis C with a single injection .
This follows the FDA’s review of Benitec’s Investigational New Drug (IND) application, which was filed on 6 December 2013.
Benitec Biopharma’s CEO and Managing Director, Peter French said, “We are very pleased with this outcome which establishes Benitec as a clinical stage company .”
Excerpt From:March 2013/ Benitec Selects the Duke Clinical Research Unit as a Site for Hepatitis C Phase I/II Clinical Trial
About TT-034
TT-034 is a potentially transformative therapeutic that is intended to provide a "one-shot-cure" for Hepatitis C with a single injection. TT-034 works through RNA interference (RNAi), which is a naturally occurring regulatory process in cells that acts to "silence" genes after they have been transcribed from DNA into messenger RNA. Benitec's proprietary ddRNAi approach involves the introduction of a DNA vector that produces short hairpin RNAs (shRNAs) that are processed by the cell into siRNAs. This approach emulates the cell's own gene silencing mechanism and provides long term activity (months). Moreover, the virus vector used to deliver the TT-034 construct, an engineered non-replicating adeno-associated virus (AAV8), targets almost exclusively liver cells (where HCV replicates). TT-034 is further designed to prevent viral escape through mutations (a major problem for most HCV drugs) by using three different shRNAs to simultaneously target three separate highly conserved regions in the HCV genome. In mice and monkeys, TT0-034 has been shown to transduce 100% of hepatocytes in the liver and provide high shRNA activity for 180 days (the duration of the studies), without adverse effects.
For further information, please contact the persons outlined below, or visit the Benitec website at www.benitec.com
Company
Carl Stubbings
Chief Business Officer
Tel: +61 (2) 9555 6986
Email:
cstubbings@benitec.com
Investor relations
Jane Lowe
Buchan Consulting
Te
l: +61 (2) 9237 2807
Email:
jlowe@buchanwe.com.au
About Benitec Biopharma Limited:
Benitec Biopharma Limited is an ASX - listed biotechnology company (ASX Code: BLT) based in Sydney, Australia. The company has a pipeline of in - house and partnered therapeutic programs based on its patented ge ne - silencing technology, ddRNAi . Benitec is developing treatments for chronic and life - threatening human conditions such as Hepatitis C , Hepatitis B, wet age - related macular degeneration, cancer - associated pain, drug resistant lung cancer and oculopharyngeal muscular dystrophy based on this technology. In addition, Benitec has licensed ddRNAi technology to other biopharmaceutical companies who are progressing their programs t owards the clinic for applications including HIV/AIDS, retinitis pigmentosa and Huntington’s disease. For more information on Benitec refer to the Company’s website at www.benitec.com .
Thursday, March 21, 2013
Benitec Selects the Duke Clinical Research Unit as a Site for Hepatitis C Phase I/II Clinical Trial
March 21, 2013, 7:00 p.m. EDT
Benitec Selects the Duke Clinical Research Unit as a Site for Hepatitis C Phase I/II Clinical Trial
SYDNEY and DURHAM, N.C., March 21, 2013 /PRNewswire via COMTEX/ -- RNAi-based therapeutics company Benitec Biopharma Limited (asx code:BLT) today announced the selection of the Duke Clinical Research Unit, the early phase unit of the Duke Clinical Research Institute (DCRI), Durham, North Carolina, USA as a site for its upcoming phase I/II first-in-man trial for TT-034 in Hepatitis C. TT-034 is being developed as a potential "one-shot-cure" for Hepatitis C (HCV).
"We are very excited to be working with Duke, a world renowned research institution with significant experience in this area," said Peter French, Ph.D., Chief Executive Officer of Benitec. "The TT-034 trial marks the transition of Benitec to a clinical stage company. We expect that positive results from the trial will provide a value inflection point for the company, and also be a validation for our ddRNAi technology as an effective platform for therapeutics."
The phase I/II clinical trial is an open-label dose escalation study to evaluate the safety and activity of single doses of TT-034 in patients with chronic HCV genotype 1 infection who have failed previous treatments. The trial is expected to involve 14 patients in 5 sequential dose cohorts. Additional consolidation cohorts may be added during the study to confirm the results of the trial. The primary safety endpoints are dose limiting adverse events. The primary activity end points are serum viral load reduction and degree of hepatocyte transduction (measured through liver biopsies). There is a pre-specified interim read on safety and activity within months of trial commencement. The clinical trial is expected to begin enrolling patients during the second half of 2013.
Duke's principal investigator for the study will be Keyur Patel, M.D. Dr Patel has previous experience with oligonucleotide therapeutics in HCV, is a recipient of the prestigious American Association for the Study of Liver Diseases (AASLD) Shelia Sherlock Clinical and Translational Research award and has over 100 citations in peer-reviewed publications.
"TT-034 is a potentially transformative new treatment," Dr. Patel commented. "A therapeutic that could cure an HCV patient with a single injection would obviously be a big step forward compared to even the best treatments that are currently on the horizon, as they all involve comparatively lengthy regimens with a combination of several drugs."
About TT-034
TT-034 is a potentially transformative therapeutic that is intended to provide a "one-shot-cure" for Hepatitis C with a single injection. TT-034 works through RNA interference (RNAi), which is a naturally occurring regulatory process in cells that acts to "silence" genes after they have been transcribed from DNA into messenger RNA. Benitec's proprietary ddRNAi approach involves the introduction of a DNA vector that produces short hairpin RNAs (shRNAs) that are processed by the cell into siRNAs. This approach emulates the cell's own gene silencing mechanism and provides long term activity (months). Moreover, the virus vector used to deliver the TT-034 construct, an engineered non-replicating adeno-associated virus (AAV8), targets almost exclusively liver cells (where HCV replicates). TT-034 is further designed to prevent viral escape through mutations (a major problem for most HCV drugs) by using three different shRNAs to simultaneously target three separate highly conserved regions in the HCV genome. In mice and monkeys, TT0-034 has been shown to transduce 100% of hepatocytes in the liver and provide high shRNA activity for 180 days (the duration of the studies), without adverse effects.
About the Duke Clinical Research Unit:
The Duke Clinical Research Unit (DCRU) is a state-of-the-art research facility located within the Duke Medicine campus that provides infrastructure support to sponsors and investigators who are testing new drug candidates and other cutting-edge therapies, or seeking to identify and validate novel biomarkers. The DCRU has more than 20 years of early-phase clinical trial experience and has successfully conducted more than 150 early-phase studies, including 80 phase 1 studies. The experience, support systems and infrastructure of the DCRU enable provision of the highest level of program management and services for early-phase clinical trials, including quality processes, accurate reporting, and regulatory expertise. The DCRU combines the clinical expertise and scientific leadership of one of the most prestigious academic medical centres in the world with the operational capabilities of a full-service contract research organization. The DCRU is part of the Duke Clinical Research Institute. The DCRI is a comprehensive academic research organization and the only one of its kind that can offer all the services of a commercial contract research organization with the academic credibility and expertise of an academic medical center.
About Benitec Biopharma Limited:
Benitec Biopharma Limited (asx code:BLT), based in Sydney, Australia, has a pipeline of in-house and partnered therapeutic programs based on its patented gene-silencing technology, ddRNAi. Benitec is developing treatments for chronic and life-threatening human conditions. Its most advanced program is TT-034 for the treatment of chronic HCV infection. Benitec has licensed ddRNAi technology to other biopharmaceutical companies who are advancing their programs toward the clinic for applications including HIV/AIDS, retinitis pigmentosa and Huntington's disease. For more information on Benitec refer to the Company's website at www.benitec.com.
For more information please contact:
For Duke Clinical Research Unit:Barry Mangum, PharmD | Director Clinical Pharmacology Phone: 919 210 8099 | Barry.Mangum@duke.edu | www.dcru.org
For Benitec:Dr Peter French | Chief Executive Officer Phone: +61 (02) 9555 6986 | pfrench@benitec.com | www.benitec.com
SOURCE Benitec Biopharma Limited
Copyright (C) 2013 PR Newswire. All rights reserved
Benitec Selects the Duke Clinical Research Unit as a Site for Hepatitis C Phase I/II Clinical Trial
SYDNEY and DURHAM, N.C., March 21, 2013 /PRNewswire via COMTEX/ -- RNAi-based therapeutics company Benitec Biopharma Limited (asx code:BLT) today announced the selection of the Duke Clinical Research Unit, the early phase unit of the Duke Clinical Research Institute (DCRI), Durham, North Carolina, USA as a site for its upcoming phase I/II first-in-man trial for TT-034 in Hepatitis C. TT-034 is being developed as a potential "one-shot-cure" for Hepatitis C (HCV).
"We are very excited to be working with Duke, a world renowned research institution with significant experience in this area," said Peter French, Ph.D., Chief Executive Officer of Benitec. "The TT-034 trial marks the transition of Benitec to a clinical stage company. We expect that positive results from the trial will provide a value inflection point for the company, and also be a validation for our ddRNAi technology as an effective platform for therapeutics."
The phase I/II clinical trial is an open-label dose escalation study to evaluate the safety and activity of single doses of TT-034 in patients with chronic HCV genotype 1 infection who have failed previous treatments. The trial is expected to involve 14 patients in 5 sequential dose cohorts. Additional consolidation cohorts may be added during the study to confirm the results of the trial. The primary safety endpoints are dose limiting adverse events. The primary activity end points are serum viral load reduction and degree of hepatocyte transduction (measured through liver biopsies). There is a pre-specified interim read on safety and activity within months of trial commencement. The clinical trial is expected to begin enrolling patients during the second half of 2013.
Duke's principal investigator for the study will be Keyur Patel, M.D. Dr Patel has previous experience with oligonucleotide therapeutics in HCV, is a recipient of the prestigious American Association for the Study of Liver Diseases (AASLD) Shelia Sherlock Clinical and Translational Research award and has over 100 citations in peer-reviewed publications.
"TT-034 is a potentially transformative new treatment," Dr. Patel commented. "A therapeutic that could cure an HCV patient with a single injection would obviously be a big step forward compared to even the best treatments that are currently on the horizon, as they all involve comparatively lengthy regimens with a combination of several drugs."
About TT-034
TT-034 is a potentially transformative therapeutic that is intended to provide a "one-shot-cure" for Hepatitis C with a single injection. TT-034 works through RNA interference (RNAi), which is a naturally occurring regulatory process in cells that acts to "silence" genes after they have been transcribed from DNA into messenger RNA. Benitec's proprietary ddRNAi approach involves the introduction of a DNA vector that produces short hairpin RNAs (shRNAs) that are processed by the cell into siRNAs. This approach emulates the cell's own gene silencing mechanism and provides long term activity (months). Moreover, the virus vector used to deliver the TT-034 construct, an engineered non-replicating adeno-associated virus (AAV8), targets almost exclusively liver cells (where HCV replicates). TT-034 is further designed to prevent viral escape through mutations (a major problem for most HCV drugs) by using three different shRNAs to simultaneously target three separate highly conserved regions in the HCV genome. In mice and monkeys, TT0-034 has been shown to transduce 100% of hepatocytes in the liver and provide high shRNA activity for 180 days (the duration of the studies), without adverse effects.
About the Duke Clinical Research Unit:
The Duke Clinical Research Unit (DCRU) is a state-of-the-art research facility located within the Duke Medicine campus that provides infrastructure support to sponsors and investigators who are testing new drug candidates and other cutting-edge therapies, or seeking to identify and validate novel biomarkers. The DCRU has more than 20 years of early-phase clinical trial experience and has successfully conducted more than 150 early-phase studies, including 80 phase 1 studies. The experience, support systems and infrastructure of the DCRU enable provision of the highest level of program management and services for early-phase clinical trials, including quality processes, accurate reporting, and regulatory expertise. The DCRU combines the clinical expertise and scientific leadership of one of the most prestigious academic medical centres in the world with the operational capabilities of a full-service contract research organization. The DCRU is part of the Duke Clinical Research Institute. The DCRI is a comprehensive academic research organization and the only one of its kind that can offer all the services of a commercial contract research organization with the academic credibility and expertise of an academic medical center.
About Benitec Biopharma Limited:
Benitec Biopharma Limited (asx code:BLT), based in Sydney, Australia, has a pipeline of in-house and partnered therapeutic programs based on its patented gene-silencing technology, ddRNAi. Benitec is developing treatments for chronic and life-threatening human conditions. Its most advanced program is TT-034 for the treatment of chronic HCV infection. Benitec has licensed ddRNAi technology to other biopharmaceutical companies who are advancing their programs toward the clinic for applications including HIV/AIDS, retinitis pigmentosa and Huntington's disease. For more information on Benitec refer to the Company's website at www.benitec.com.
For more information please contact:
For Duke Clinical Research Unit:Barry Mangum, PharmD | Director Clinical Pharmacology Phone: 919 210 8099 | Barry.Mangum@duke.edu | www.dcru.org
For Benitec:Dr Peter French | Chief Executive Officer Phone: +61 (02) 9555 6986 | pfrench@benitec.com | www.benitec.com
SOURCE Benitec Biopharma Limited
Copyright (C) 2013 PR Newswire. All rights reserved
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