|By PR Newswire||
|February 18, 2014 06:02 AM EST||
MELBOURNE, Australia and NEW YORK, Feb. 18, 2014 /PRNewswire/ --
- Primary endpoints of safety and tolerability met.
- Secondary endpoint: Statistically significant improvement in a measure of executive function(cognition) in research participants administered 250mg PBT2 daily (p=0.042).
- PBT2 250mg was also associated with a favourable signal in functional capacity.
- Preliminary evidence suggests PBT2 250mg reduced atrophy of brain tissue in areas affected in Huntington disease, seen in a pilot imaging sub-study.
- Company plans to advance PBT2 to a confirmatory Phase 3 clinical trial.
- Prana to host investor conference call and webcast today at 5:30pm ET; See access information below
Prana Biotechnology (ASX:PBT; NASDAQ:PRAN) today announced that its Phase 2 REACH2HD clinical trial investigating PBT2 as a treatment for Huntington disease met its primary safety endpoint and achieved statistically significant improvement in a measure of executive function (cognition), which comprised part of the study's main efficacy outcome. Prana plans to advance PBT2 into a confirmatory Phase 3 clinical trial that could allow PBT2 to be approved for the treatment of Huntington disease.
Dr. Ray Dorsey, Professor of Neurology at the University of Rochester and the Principal Investigator on the trial added: "We are very pleased that the results of the Reach2HD study have shown that PBT2 is well tolerated and generally safe over six months in individuals with early to mid-stage Huntington disease."
"In addition, the results indicated a significant benefit on cognition that is consistent with the previous trial in Alzheimer's disease and is accompanied by an encouraging finding in functional capacity. We are very thankful for the involvement of the research participants and investigators in this study and look forward to future trials of this promising therapy for one of the cardinal features of Huntington disease."
Reach2HD is a double-blind, placebo-controlled study was conducted by the Huntington Study Group at research sites in the United States and Australia. The study enrolled 109 individuals with Huntington disease who were randomly assigned to receive daily doses of either PBT2 250mg, PBT2 100mg, or placebo for 26 weeks.
The primary endpoint of the study was met. In this study, PBT2 was safe and well tolerated. Ninety-five percent (104 of 109) of participants completed the study on their assigned dose. There were no substantial differences in adverse events across the two PBT2 dose groups and the placebo group. Only one of the ten reported serious adverse events was deemed by the clinical site investigator to be related to drug treatment.
An independent Data Safety Monitoring Board met on five occasions over the course of the trial and on each occasion recommended that the trial continue as per the original protocol.
The effects of PBT2 were tested on cognition, motor performance, behaviour and functional capacity, of which cognition was pre-specified as the main efficacy outcome.
There was a statistically significant improvement in performance on the Trail Making Test Part B, in the PBT2 250mg group compared to placebo at both 12 (p<0.001) and 26 weeks (p=0.042). Trail Making Test Part B is a measure of executive function (e.g., ability to plan activities), which is impaired early in the course of Huntington disease and is also affected in Alzheimer's disease.
Given the evidence from an earlier trial that showed that PBT2 improved executive function in Alzheimer's disease patients, the Reach2HD trial included a plan to assess the effects of PBT2 on an Executive Function Composite z-score that included the Trail Making Test Part B. There was a statistically significant improvement in this z-score (p=0.038) in a pre-specified analysis of Reach2HD participants with early stage Huntington disease, as measured by their Total Functioning Capacity score. Across all participants, which comprised both early and mid-stage patients, there was a trend to improvement (p=0.069).
Dr. Rudy Tanzi, Professor of Neurology at Harvard Medical School and Prana's Chief Scientific Advisor, said: "The observation of significant improvement in executive function with PBT2 in this clinical trial for Huntington disease and the previously reported Alzheimer's trial, suggests a common mechanism for neurodegeneration in these diseases based on metal interactions. In my opinion, these findings significantly elevate the potential for PBT2 as an effective therapy for both Huntington disease and Alzheimer's disease."
The improvement in executive function was accompanied by a small but favourable signal in a key measure of functional capacity.
Dr. Ira Shoulson, Professor of Neurology at Georgetown University and Chair of the Huntington Study Group, who was not involved in the trial and acts as an advisor to Prana, added: "In the Reach2HD trial, the improvement in executive function performance was also accompanied by a favourable signal of a slowing of functional decline, as measured by the Total Functional Capacity score. This is the first time we have observed dose-related slowing in functional decline over a six month period of treatment – which taken together with the safety reassurance – will provide genuine optimism for the Huntington disease community to support a larger confirmatory trial of PBT2 in Huntington disease."
Finally, as Huntington disease and other neurodegenerative disorders progress, there is a gradual loss of brain tissue or atrophy. In Reach2HD, brain imaging using magnetic resonance imaging (MRI) was performed in a small subset of patients (n=6) to map anatomical changes in brain structure. In the combined PBT2 groups (n=4) a reduction in atrophy of brain tissue in regions of the brain known to be affected by Huntington disease was observed compared to the placebo group.
Dr. Diana Rosas, Associate Professor of Neurology at Harvard Medical School and the study's co-Principal Investigator who conducted the imaging sub-study commented: "Despite the very small number of patients in the sub-study, the data are suggestive of a beneficial effect of PBT2 in regions of the brain that are known to be vulnerable to Huntington disease."
Investor Conference Call Information:
Prana will host an investor conference call and webcast this evening at 5:30pm Eastern Time to discuss the study results. Investors in the United States may access the conference call by dialing 1 (855) 293-1544; conference ID: 58817510. The live webcast may be accessed here. Additional conference call information is available at www.pranabio.com, including Australian and international conference numbers.
Due to the expected high number of participants on the call, we recommend you commence registration for the event 15 minutes prior. A recording of the call will be available within 4 hours of its conclusion and will remain available for three months. The archived recording can be accessed here.
Global Investor Relations Lead
Investor Relations (USA)
Media Relations (Australia)
Media Relations (US)
SOURCE Tiberend Strategic Advisors, Inc.
When it comes to cloud computing, the ability to turn massive amounts of compute cores on and off on demand sounds attractive to IT staff, who need to manage peaks and valleys in user activity. With cloud bursting, the majority of the data can stay on premises while tapping into compute from public cloud providers, reducing risk and minimizing need to move large files. In his session at 18th Cloud Expo, Scott Jeschonek, Director of Product Management at Avere Systems, discussed the IT and busin...
Dec. 6, 2016 12:30 AM EST Reads: 3,853
According to Forrester Research, every business will become either a digital predator or digital prey by 2020. To avoid demise, organizations must rapidly create new sources of value in their end-to-end customer experiences. True digital predators also must break down information and process silos and extend digital transformation initiatives to empower employees with the digital resources needed to win, serve, and retain customers.
Dec. 6, 2016 12:30 AM EST Reads: 1,187
The IoT is changing the way enterprises conduct business. In his session at @ThingsExpo, Eric Hoffman, Vice President at EastBanc Technologies, discussed how businesses can gain an edge over competitors by empowering consumers to take control through IoT. He cited examples such as a Washington, D.C.-based sports club that leveraged IoT and the cloud to develop a comprehensive booking system. He also highlighted how IoT can revitalize and restore outdated business models, making them profitable ...
Dec. 6, 2016 12:30 AM EST Reads: 4,564
In his general session at 19th Cloud Expo, Manish Dixit, VP of Product and Engineering at Dice, discussed how Dice leverages data insights and tools to help both tech professionals and recruiters better understand how skills relate to each other and which skills are in high demand using interactive visualizations and salary indicator tools to maximize earning potential. Manish Dixit is VP of Product and Engineering at Dice. As the leader of the Product, Engineering and Data Sciences team at D...
Dec. 6, 2016 12:00 AM EST Reads: 847
SaaS companies can greatly expand revenue potential by pushing beyond their own borders. The challenge is how to do this without degrading service quality. In his session at 18th Cloud Expo, Adam Rogers, Managing Director at Anexia, discussed how IaaS providers with a global presence and both virtual and dedicated infrastructure can help companies expand their service footprint with low “go-to-market” costs.
Dec. 5, 2016 11:45 PM EST Reads: 4,282
Get deep visibility into the performance of your databases and expert advice for performance optimization and tuning. You can't get application performance without database performance. Give everyone on the team a comprehensive view of how every aspect of the system affects performance across SQL database operations, host server and OS, virtualization resources and storage I/O. Quickly find bottlenecks and troubleshoot complex problems.
Dec. 5, 2016 10:45 PM EST Reads: 2,069
"We are the public cloud providers. We are currently providing 50% of the resources they need for doing e-commerce business in China and we are hosting about 60% of mobile gaming in China," explained Yi Zheng, CPO and VP of Engineering at CDS Global Cloud, in this SYS-CON.tv interview at 19th Cloud Expo, held November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA.
Dec. 5, 2016 10:30 PM EST Reads: 972
"Once customers get a year into their IoT deployments, they start to realize that they may have been shortsighted in the ways they built out their deployment and the key thing I see a lot of people looking at is - how can I take equipment data, pull it back in an IoT solution and show it in a dashboard," stated Dave McCarthy, Director of Products at Bsquare Corporation, in this SYS-CON.tv interview at @ThingsExpo, held November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA.
Dec. 5, 2016 10:30 PM EST Reads: 1,050
Predictive analytics tools monitor, report, and troubleshoot in order to make proactive decisions about the health, performance, and utilization of storage. Most enterprises combine cloud and on-premise storage, resulting in blended environments of physical, virtual, cloud, and other platforms, which justifies more sophisticated storage analytics. In his session at 18th Cloud Expo, Peter McCallum, Vice President of Datacenter Solutions at FalconStor, discussed using predictive analytics to mon...
Dec. 5, 2016 09:00 PM EST Reads: 4,953
The Internet of Things will challenge the status quo of how IT and development organizations operate. Or will it? Certainly the fog layer of IoT requires special insights about data ontology, security and transactional integrity. But the developmental challenges are the same: People, Process and Platform and how we integrate our thinking to solve complicated problems. In his session at 19th Cloud Expo, Craig Sproule, CEO of Metavine, demonstrated how to move beyond today's coding paradigm and sh...
Dec. 5, 2016 08:45 PM EST Reads: 491
Today we can collect lots and lots of performance data. We build beautiful dashboards and even have fancy query languages to access and transform the data. Still performance data is a secret language only a couple of people understand. The more business becomes digital the more stakeholders are interested in this data including how it relates to business. Some of these people have never used a monitoring tool before. They have a question on their mind like “How is my application doing” but no id...
Dec. 5, 2016 07:45 PM EST Reads: 2,224
@GonzalezCarmen has been ranked the Number One Influencer and @ThingsExpo has been named the Number One Brand in the “M2M 2016: Top 100 Influencers and Brands” by Onalytica. Onalytica analyzed tweets over the last 6 months mentioning the keywords M2M OR “Machine to Machine.” They then identified the top 100 most influential brands and individuals leading the discussion on Twitter.
Dec. 5, 2016 07:45 PM EST Reads: 2,087
IoT is rapidly changing the way enterprises are using data to improve business decision-making. In order to derive business value, organizations must unlock insights from the data gathered and then act on these. In their session at @ThingsExpo, Eric Hoffman, Vice President at EastBanc Technologies, and Peter Shashkin, Head of Development Department at EastBanc Technologies, discussed how one organization leveraged IoT, cloud technology and data analysis to improve customer experiences and effici...
Dec. 5, 2016 07:15 PM EST Reads: 5,051
DevOps is being widely accepted (if not fully adopted) as essential in enterprise IT. But as Enterprise DevOps gains maturity, expands scope, and increases velocity, the need for data-driven decisions across teams becomes more acute. DevOps teams in any modern business must wrangle the ‘digital exhaust’ from the delivery toolchain, "pervasive" and "cognitive" computing, APIs and services, mobile devices and applications, the Internet of Things, and now even blockchain. In this power panel at @...
Dec. 5, 2016 07:15 PM EST Reads: 388
In his session at 19th Cloud Expo, Claude Remillard, Principal Program Manager in Developer Division at Microsoft, contrasted how his team used config as code and immutable patterns for continuous delivery of microservices and apps to the cloud. He showed how the immutable patterns helps developers do away with most of the complexity of config as code-enabling scenarios such as rollback, zero downtime upgrades with far greater simplicity. He also demoed building immutable pipelines in the cloud ...
Dec. 5, 2016 07:00 PM EST Reads: 1,816