|By PR Newswire||
|January 22, 2014 01:00 AM EST||
SOUTH PLAINFIELD, N.J., Jan. 22, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), the SMA Foundation, and Roche (SIX: RO, ROG; OTCQX: RHHBY), announced today that their joint research program in Spinal Muscular Atrophy (SMA) has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers. SMA is a genetic neuromuscular disorder that leads to muscle weakness and mobility impairment and is considered the leading genetic cause of death in infants and toddlers.
Luca Santarelli, Head of Neuroscience, Ophthalmology and Rare Diseases at Roche commented: "The compelling science behind this project and the highly synergistic alliance among Roche, PTC Therapeutics and the SMA Foundation are the groundwork for this research program. Although still early in development, we consider every step towards a therapeutic option in SMA of high importance for patients and families affected by this devastating and currently untreatable condition."
SMA is caused by a missing or defective SMN1 gene, which results in reduced levels of the survival motor neuron (SMN) protein. SMN protein plays a critical role in the survival of neuronal cells in the spinal cord responsible for muscle movements. Reduced levels of SMN protein lead to dysfunction and death of the neuronal cells, making muscles smaller and weaker over time, and can even result in death in the most severe forms of the disease. The orally available small molecule compounds in the program are designed to target the underlying cause of the disease by increasing SMN protein levels in the nervous system, muscles, and other tissues. It is estimated that approximately one in 10,000 children are born with this rare genetic disorder.
"We are delighted with the achievement of this important milestone," commented Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics, Inc. "The innovative approach developed at PTC has benefited tremendously from the scientific partnership and dedication of both Roche and the SMA Foundation. The rapid progress highlights our combined dedication to advance an investigational medicine for SMA as rapidly as possible."
The SMA program was initiated in 2006 by PTC Therapeutics in collaboration with the SMA Foundation, which provided the majority of its funding prior to Roche joining the collaboration.
"This important achievement may pave the way toward a brighter, more hopeful future for children and adults with SMA," said Loren Eng, President of the SMA Foundation. "While we appreciate that the safety studies in healthy volunteers are an early step and much further development will be required, this is a significant accomplishment, and we are grateful to the commitment of our partners in accelerating this program as rapidly as possible."
In November 2011, Roche had gained an exclusive worldwide license to the SMA program. The initiation of the clinical development triggers a $7.5 million payment to PTC from Roche.
About PTC Therapeutics, Inc.
PTC Therapeutics is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are essential to proper cellular function. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has developed proprietary technologies that it applies in its drug discovery activities and which form the basis for collaborations with leading biopharmaceutical companies. For more information on the company, please visit the website www.ptcbio.com.
PTC Therapeutics, Inc. Forward Looking Statements:
Any statements in this press release about future expectations, plans and prospects for PTC, the development of and potential market for PTC's product candidates, our clinical trials for lead compounds targeting SMA, our collaboration in SMA with Roche and the SMA Foundation, our current and planned regulatory submissions, our earlier stage programs, and other statements containing the words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan" "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Forward-looking statements involve substantial risks and uncertainties that could cause our future results, performance or achievements to differ significantly from those expressed or implied by these forward-looking statements. Such risks and uncertainties include, among others, those related to the initiation and conduct of clinical trials, availability of data from clinical trials, expectations for regulatory approvals, our scientific approach and general development progress, the availability or commercial potential of our product candidates and other factors discussed in the "Risk Factors" in the most recent Quarterly Report, which is on file with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent PTC's views only as of the date of this press release. We anticipate that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing PTC's views as of any date subsequent to the date of this press release.
SOURCE PTC Therapeutics, Inc.
SYS-CON Events announced today that EastBanc Technologies will exhibit at SYS-CON's 18th International Cloud Expo®, which will take place on June 7-9, 2016, at the Javits Center in New York City, NY. EastBanc Technologies has been working at the frontier of technology since 1999. Today, the firm provides full-lifecycle software development delivering flexible technology solutions that seamlessly integrate with existing systems – whether on premise or cloud. EastBanc Technologies partners with p...
May. 31, 2016 08:00 PM EDT Reads: 2,507
SYS-CON Events announced today that IBM Cloud Data Services has been named “Bronze Sponsor” of SYS-CON's 18th Cloud Expo, which will take place on June 7-9, 2016, at the Javits Center in New York City, NY. IBM Cloud Data Services offers a portfolio of integrated, best-of-breed cloud data services for developers focused on mobile computing and analytics use cases.
May. 31, 2016 07:15 PM EDT Reads: 1,793
SYS-CON Events announced today that Super Micro Computer, Inc., a global leader in Embedded and IoT solutions, will exhibit at SYS-CON's 18th International Cloud Expo®, which will take place on June 7-9, 2016, at the Javits Center in New York City, NY. Supermicro (NASDAQ: SMCI), the leading innovator in high-performance, high-efficiency server technology, is a premier provider of advanced server Building Block Solutions® for Data Center, Cloud Computing, Enterprise IT, Hadoop/Big Data, HPC and ...
May. 31, 2016 07:15 PM EDT Reads: 1,003
The Internet of Things (IoT) is growing rapidly by extending current technologies, products and networks. By 2020, Cisco estimates there will be 50 billion connected devices. Gartner has forecast revenues of over $300 billion, just to IoT suppliers. Now is the time to figure out how you’ll make money – not just create innovative products. With hundreds of new products and companies jumping into the IoT fray every month, there’s no shortage of innovation. Despite this, McKinsey/VisionMobile data...
May. 31, 2016 07:15 PM EDT Reads: 1,898
18th Cloud Expo, taking place June 7-9, 2016, at the Javits Center in New York City, NY, will feature technical sessions from a rock star conference faculty and the leading industry players in the world. Cloud computing is now being embraced by a majority of enterprises of all sizes. Yesterday's debate about public vs. private has transformed into the reality of hybrid cloud: a recent survey shows that 74% of enterprises have a hybrid cloud strategy. Meanwhile, 94% of enterprises are using some...
May. 31, 2016 07:00 PM EDT Reads: 3,331
We’ve been doing it for years, decades for some. How many websites have you created accounts on? Your bank, your credit card companies, social media sites, hotels and travel sites, online shopping sites, and that’s just the start. We do it often without even thinking about it, quickly entering our personal information, our data, in a plethora of systems. Sometimes we’re not even aware of the information we are providing. It could be very personal information (think of the security questions you ...
May. 31, 2016 06:45 PM EDT Reads: 1,144
@DevOpsSummit taking place June 7-9, 2016 at Javits Center, New York City, and Nov 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with the 18th International @CloudExpo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world.
May. 31, 2016 06:45 PM EDT Reads: 3,240
The cloud era has reached the stage where it is no longer a question of whether a company should migrate, but when. Enterprises have embraced the outsourcing of where their various applications are stored and who manages them, saving significant investment along the way. Plus, the cloud has become a defining competitive edge. Companies that fail to successfully adapt risk failure. The media, of course, continues to extol the virtues of the cloud, including how easy it is to get there. Migrating...
May. 31, 2016 06:30 PM EDT Reads: 894
In today's enterprise, digital transformation represents organizational change even more so than technology change, as customer preferences and behavior drive end-to-end transformation across lines of business as well as IT. To capitalize on the ubiquitous disruption driving this transformation, companies must be able to innovate at an increasingly rapid pace. Traditional approaches for driving innovation are now woefully inadequate for keeping up with the breadth of disruption and change facin...
May. 31, 2016 05:45 PM EDT Reads: 1,785
As machines are increasingly connected to the internet, it’s becoming easier to discover the numerous ways Industrial IoT (IIoT) is helping to shape the business world. This is exactly why we have decided to take a closer look at this pervasive movement and to examine the desire to connect more things! Now if you need a refresher on IIoT and how it is changing the world, take a moment and listen to Greg Gorbach with ARC Advisory Group. Gorbach believes, "IIoT will significantly change the worl...
May. 31, 2016 05:00 PM EDT Reads: 2,148
WebRTC is bringing significant change to the communications landscape that will bridge the worlds of web and telephony, making the Internet the new standard for communications. Cloud9 took the road less traveled and used WebRTC to create a downloadable enterprise-grade communications platform that is changing the communication dynamic in the financial sector. In his session at @ThingsExpo, Leo Papadopoulos, CTO of Cloud9, will discuss the importance of WebRTC and how it enables companies to fo...
May. 31, 2016 04:45 PM EDT Reads: 2,660
"What we see what happens when you have a completely networked society and the potential to now drive the value creation and the collaboration and the ecosystems that are possible when you start to be able to connect people and industries together in ways that have never been possible before," explained Esmeralda Swartz, VP of Marketing Enterprise & Cloud at Ericsson, in this SYS-CON.tv interview at @ThingsExpo, held November 3-5, 2015, at the Santa Clara Convention Center in Santa Clara, CA.
May. 31, 2016 04:45 PM EDT Reads: 1,904
How will your company move to the cloud while ensuring a solid security posture? Organizations from small to large are increasingly adopting cloud solutions to deliver essential business services at a much lower cost. According to cyber security experts, the frequency and severity of cyber-attacks are on the rise, causing alarm to businesses and customers across a variety of industries. To defend against exploits like these, a company must adopt a comprehensive security defense strategy that is ...
May. 31, 2016 04:30 PM EDT Reads: 974
SYS-CON Events announced today that AppNeta, the leader in performance insight for business-critical web applications, will exhibit and present at SYS-CON's @DevOpsSummit at Cloud Expo New York, which will take place on June 7-9, 2016, at the Javits Center in New York City, NY. AppNeta is the only application performance monitoring (APM) company to provide solutions for all applications – applications you develop internally, business-critical SaaS applications you use and the networks that deli...
May. 31, 2016 04:00 PM EDT Reads: 2,730
SYS-CON Events announced today that Zerto will exhibit at SYS-CON's 18th International Cloud Expo®, which will take place on June 7-9, 2016, at the Javits Center in New York City, NY. Zerto is committed to keeping enterprise and cloud IT running 24/7 by providing innovative, simple, reliable and scalable business continuity software solutions. Through the Zerto Cloud Continuity Platform™, organizations can seamlessly move and protect virtualized workloads between public, private and hybrid clou...
May. 31, 2016 03:50 PM EDT Reads: 309