|By Marketwired .||
|November 7, 2012 01:55 AM EST||
SAN DIEGO, CA and LEIDEN, THE NETHERLANDS -- (Marketwire) -- 11/07/12 -- Statistically significant results for primary endpoint of time to beginning of symptom relief
Santarus, Inc. (NASDAQ: SNTS) and Pharming Group NV (NYSE Euronext: PHARM) today announced that their pivotal Phase III clinical study to evaluate the safety and efficacy of the investigational drug RUCONEST® (recombinant human C1 esterase inhibitor) 50 U/kg for the treatment of acute attacks of angioedema in patients with Hereditary Angioedema (HAE) met the primary endpoint of time to beginning of symptom relief.
A statistically significant difference in the time to beginning of symptom relief was observed in the intent-to-treat population (n=75) between RUCONEST and placebo (p=0.031, log-rank test); the median time to beginning of symptom relief was 90 minutes for RUCONEST patients (n=44) and 152 minutes for placebo patients (n=31). The time to beginning of symptom relief was defined as the time from the beginning of infusion of study medication (RUCONEST or placebo) until the beginning of a persistent beneficial effect, based on the patient's responses to a Treatment Effect Questionnaire for the primary attack location.
RUCONEST was generally well tolerated in this Phase III clinical study and the frequency of patients experiencing at least one treatment emergent adverse event in the RUCONEST treated group was less than in the placebo group. Within 72 hours of the completion of infusion of study medication, four RUCONEST patients (7%) experienced six adverse events: sneezing, procedural headache, back pain, skin burning sensation, an increase in fibrin D-dimer and lipoma. Within the 72 hour period four placebo patients (22%) experienced four adverse events: sinus congestion, vasomotor rhinitis, diarrhea and dyspepsia. Thromboembolic events, anaphylaxis, or neutralizing antibodies to C1 inhibitor were not observed in any patient. There was one patient in the RUCONEST group that experienced a serious adverse event (abdominal hernia at Day 79) that was assessed by the investigator as not related to the study drug.
"These positive results are consistent with the efficacy data previously reported from two smaller randomized, controlled clinical studies with RUCONEST in patients with HAE, and we believe the results provide strong support for our proposed dosing regimen of 50 U/kg in treating acute attacks of HAE," said Wendell Wierenga, Ph.D., executive vice president of research and development of Santarus.
"We are very pleased with these pivotal study results and look forward to working with our colleagues at Santarus to prepare and submit the Biologics License Application (BLA) for RUCONEST to the FDA in the first half of 2013," said Bruno Giannetti, M.D., Ph.D., chief operations officer of Pharming. "We anticipate that additional data from this Phase III study will be presented at an appropriate medical meeting in 2013."
Santarus licensed exclusive rights to commercialize RUCONEST in North America for the treatment of acute attacks of HAE as well as other potential future indications from Pharming. Under the terms of the license agreement, a $10 million milestone is now payable to Pharming as a result of the successful achievement of the primary endpoint of the Phase III clinical study. An additional $5 million milestone will be payable to Pharming upon U.S. Food and Drug Administration (FDA) acceptance of the BLA for review.
RUCONEST Phase III Clinical Study (Study 1310)
Pharming conducted Study 1310 with RUCONEST under a Special Protocol Assessment (SPA) agreement with the FDA, and the study is intended to support the submission of a BLA in the U.S.
* In Study 1310, RUCONEST was evaluated for the treatment of acute attacks of angioedema in patients with HAE in an international, multicenter, randomized, placebo-controlled Phase III study comparing a single IV infusion of 50 U/kg of RUCONEST to a saline control with a primary endpoint of time to beginning of symptom relief.
* The time to beginning of symptom relief was defined as the time lapsed from the beginning of the infusion of study medication to the beginning of a persistent beneficial effect based on the patient's responses to a Treatment Effect Questionnaire for the primary attack location.
* A 90 day follow-up period was required for each patient enrolled, or until such time that the patient required open-label treatment for a subsequent attack during the 90 day period. The data from the open-label extension are being collected for additional efficacy and safety analyses.
* The study enrolled a total of 75 patients who were randomized 3:2 to receive either RUCONEST or saline.
About RUCONEST and Hereditary Angioedema
RUCONEST (INN conestat alfa) is a recombinant version of the human protein C1 esterase inhibitor, and is produced with Pharming's proprietary technology which uses milk from transgenic rabbits. RUCONEST is approved in Europe for the treatment of acute angioedema attacks in patients with HAE, a genetic disorder in which the patient is deficient in or lacks a functional plasma protein C1 esterase inhibitor, resulting in unpredictable and debilitating episodes of intense swelling. The swelling may occur in one or more anatomical areas, including the extremities, face, trunk, genitals, abdomen and upper airway. The frequency and severity of HAE attacks vary and are most serious when they involve laryngeal edema, which can close the upper airway and cause death by asphyxiation. According to the U.S. Hereditary Angioedema Association, epidemiological estimates for HAE range from one in 10,000 to one in 50,000 individuals. RUCONEST is an investigational drug in the U.S. and has been granted orphan drug designation by the FDA both for the treatment of acute attacks of HAE and for prophylactic treatment of HAE.
About Pharming Group NV
Pharming Group NV develops innovative products for the treatment of unmet medical needs. RUCONEST® is a recombinant human C1 esterase inhibitor approved for the treatment of angioedema attacks in patients with HAE in all 27 EU countries plus Norway, Iceland and Liechtenstein, and is distributed in the EU by Swedish Orphan Biovitrum (OMX: SOBI). The product is also being evaluated for various follow-on indications. Pharming's advanced technologies include innovative and validated platforms for the production of protein therapeutics, technology and processes for the purification and formulation of these products. A feasibility study, using the validated transgenic rabbit platform, aimed at the development of recombinant Factor VIII for the treatment of Haemophilia A is underway with partner, Renova Life, Inc. Additional information is available on the Pharming website, www.pharming.com.
Santarus, Inc. is a specialty biopharmaceutical company focused on acquiring, developing and commercializing proprietary products that address the needs of patients treated by physician specialists. The company's current commercial efforts are focused on GLUMETZA® (metformin hydrochloride extended release tablets) and CYCLOSET® (bromocriptine mesylate) tablets, which are indicated as adjuncts to diet and exercise to improve glycemic control in adults with type 2 diabetes, and on FENOGLIDE® (fenofibrate) tablets, which is indicated as an adjunct to diet to reduce high cholesterol. Santarus also sells ZEGERID® (omeprazole/sodium bicarbonate), which is indicated for the treatment of certain gastrointestinal diseases and disorders.
Santarus has a diverse product development pipeline. A New Drug Application for UCERIS (budesonide) for induction of remission of active mild to moderate ulcerative colitis is under review by the U.S. Food and Drug Administration with a response expected in January 2013. The pipeline also includes two late-stage investigational drugs in Phase III clinical studies: RUCONEST® (recombinant human C1 esterase inhibitor) for treatment of acute attacks of hereditary angioedema and rifamycin SV MMX® for treatment of travelers' diarrhea. In addition, the company's investigational monoclonal antibody, SAN-300, is being evaluated in a Phase I clinical program. More information about Santarus is available at www.santarus.com.
Santarus and Pharming caution you that statements included in this press release that are not a description of historical facts are forward-looking statements. The inclusion of forward-looking statements should not be regarded as a representation by Santarus or Pharming that any of its plans or objectives will be achieved. Actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in Santarus and Pharming's businesses, including, without limitation: risks related to the timing for submission of the BLA and whether the FDA will accept the BLA for review following submission and ultimately approve it; whether the FDA will concur with the clinical interpretation of the Phase III study results or the conduct of the study; whether the FDA ultimately will require additional clinical studies or other development programs before accepting the RUCONEST BLA or approving RUCONEST; risks related to Santarus' dependence on Pharming for many functions related to RUCONEST, and Pharming's ability to continue to perform these functions based on its limited financial resources; risks related to the license and supply arrangements between Santarus and Pharming, including the potential for termination of the arrangements; other difficulties or delays in development, testing, manufacturing and marketing of, and obtaining and maintaining regulatory approvals for, Santarus' and Pharming's products; and other risks detailed in prior press releases as well as in public periodic filings with the Securities and Exchange Commission.
You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and neither Santarus nor Pharming undertakes any obligation to revise or update this news release to reflect events or circumstances after the date hereof. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
Santarus®, FENOGLIDE®, UCERIS, and ZEGERID® are trademarks of Santarus, Inc. GLUMETZA® is a trademark of Biovail Laboratories International S.r.l. licensed exclusively in the United States to Depomed, Inc. CYCLOSET® is a trademark of VeroScience LLC. MMX® is a trademark of Cosmo Technologies Limited. RUCONEST® is a trademark of Pharming Group N.V.
Press release (PDF): http://hugin.info/132866/R/1655680/535073.pdf
This announcement is distributed by Thomson Reuters on behalf of Thomson Reuters clients. The owner of this announcement warrants that:
(i) the releases contained herein are protected by copyright and other applicable laws; and
(ii) they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Pharming Group N.V. via Thomson Reuters ONE
Martha L. Hough
VP Finance & Investor Relations
+1 (858) 314-5824
Debra P. Crawford
Chief Financial Officer
+1 (858) 314-5708
Sijmen de Vries
Tel: +31 (0)71 524 7400
Westwicke Partners, LLC
Stefan Loren, Ph.D.
+1 (858) 356-5930
+1 (858) 356-5932
Julia Phillips/John Dineen
Tel: +44 (0)207 269 7193
The pace of innovation, vendor lock-in, production sustainability, cost-effectiveness, and managing risk… In his session at 18th Cloud Expo, Dan Choquette, Founder of RackN, discussed how CIOs are challenged finding the balance of finding the right tools, technology and operational model that serves the business the best. He also discussed how clouds, open source software and infrastructure solutions have benefits but also drawbacks and how workload and operational portability between vendors an...
Jan. 23, 2017 03:30 AM EST Reads: 5,105
Adding public cloud resources to an existing application can be a daunting process. The tools that you currently use to manage the software and hardware outside the cloud aren’t always the best tools to efficiently grow into the cloud. All of the major configuration management tools have cloud orchestration plugins that can be leveraged, but there are also cloud-native tools that can dramatically improve the efficiency of managing your application lifecycle. In his session at 18th Cloud Expo, ...
Jan. 23, 2017 03:00 AM EST Reads: 6,169
Web Real-Time Communication APIs have quickly revolutionized what browsers are capable of. In addition to video and audio streams, we can now bi-directionally send arbitrary data over WebRTC's PeerConnection Data Channels. With the advent of Progressive Web Apps and new hardware APIs such as WebBluetooh and WebUSB, we can finally enable users to stitch together the Internet of Things directly from their browsers while communicating privately and securely in a decentralized way.
Jan. 23, 2017 03:00 AM EST Reads: 1,002
With the proliferation of both SQL and NoSQL databases, organizations can now target specific fit-for-purpose database tools for their different application needs regarding scalability, ease of use, ACID support, etc. Platform as a Service offerings make this even easier now, enabling developers to roll out their own database infrastructure in minutes with minimal management overhead. However, this same amount of flexibility also comes with the challenges of picking the right tool, on the right ...
Jan. 23, 2017 02:45 AM EST Reads: 5,415
Data is an unusual currency; it is not restricted by the same transactional limitations as money or people. In fact, the more that you leverage your data across multiple business use cases, the more valuable it becomes to the organization. And the same can be said about the organization’s analytics. In his session at 19th Cloud Expo, Bill Schmarzo, CTO for the Big Data Practice at Dell EMC, introduced a methodology for capturing, enriching and sharing data (and analytics) across the organization...
Jan. 23, 2017 02:30 AM EST Reads: 3,372
"Tintri was started in 2008 with the express purpose of building a storage appliance that is ideal for virtualized environments. We support a lot of different hypervisor platforms from VMware to OpenStack to Hyper-V," explained Dan Florea, Director of Product Management at Tintri, in this SYS-CON.tv interview at 18th Cloud Expo, held June 7-9, 2016, at the Javits Center in New York City, NY.
Jan. 23, 2017 02:30 AM EST Reads: 4,998
Manufacturers are embracing the Industrial Internet the same way consumers are leveraging Fitbits – to improve overall health and wellness. Both can provide consistent measurement, visibility, and suggest performance improvements customized to help reach goals. Fitbit users can view real-time data and make adjustments to increase their activity. In his session at @ThingsExpo, Mark Bernardo Professional Services Leader, Americas, at GE Digital, discussed how leveraging the Industrial Internet and...
Jan. 23, 2017 02:00 AM EST Reads: 6,637
Data is the fuel that drives the machine learning algorithmic engines and ultimately provides the business value. In his session at 20th Cloud Expo, Ed Featherston, director/senior enterprise architect at Collaborative Consulting, will discuss the key considerations around quality, volume, timeliness, and pedigree that must be dealt with in order to properly fuel that engine.
Jan. 23, 2017 01:45 AM EST Reads: 2,758
The speed of software changes in growing and large scale rapid-paced DevOps environments presents a challenge for continuous testing. Many organizations struggle to get this right. Practices that work for small scale continuous testing may not be sufficient as the requirements grow. In his session at DevOps Summit, Marc Hornbeek, Sr. Solutions Architect of DevOps continuous test solutions at Spirent Communications, explained the best practices of continuous testing at high scale, which is rele...
Jan. 23, 2017 12:45 AM EST Reads: 4,650
Containers have changed the mind of IT in DevOps. They enable developers to work with dev, test, stage and production environments identically. Containers provide the right abstraction for microservices and many cloud platforms have integrated them into deployment pipelines. DevOps and Containers together help companies to achieve their business goals faster and more effectively. In his session at DevOps Summit, Ruslan Synytsky, CEO and Co-founder of Jelastic, reviewed the current landscape of D...
Jan. 23, 2017 12:15 AM EST Reads: 5,147
Why do your mobile transformations need to happen today? Mobile is the strategy that enterprise transformation centers on to drive customer engagement. In his general session at @ThingsExpo, Roger Woods, Director, Mobile Product & Strategy – Adobe Marketing Cloud, covered key IoT and mobile trends that are forcing mobile transformation, key components of a solid mobile strategy and explored how brands are effectively driving mobile change throughout the enterprise.
Jan. 23, 2017 12:00 AM EST Reads: 5,882
Due of the rise of Hadoop, many enterprises are now deploying their first small clusters of 10 to 20 servers. At this small scale, the complexity of operating the cluster looks and feels like general data center servers. It is not until the clusters scale, as they inevitably do, when the pain caused by the exponential complexity becomes apparent. We've seen this problem occur time and time again. In his session at Big Data Expo, Greg Bruno, Vice President of Engineering and co-founder of StackIQ...
Jan. 22, 2017 11:45 PM EST Reads: 7,924
IoT is at the core or many Digital Transformation initiatives with the goal of re-inventing a company's business model. We all agree that collecting relevant IoT data will result in massive amounts of data needing to be stored. However, with the rapid development of IoT devices and ongoing business model transformation, we are not able to predict the volume and growth of IoT data. And with the lack of IoT history, traditional methods of IT and infrastructure planning based on the past do not app...
Jan. 22, 2017 11:00 PM EST Reads: 1,033
One of the hottest areas in cloud right now is DRaaS and related offerings. In his session at 16th Cloud Expo, Dale Levesque, Disaster Recovery Product Manager with Windstream's Cloud and Data Center Marketing team, will discuss the benefits of the cloud model, which far outweigh the traditional approach, and how enterprises need to ensure that their needs are properly being met.
Jan. 22, 2017 11:00 PM EST Reads: 4,569
Up until last year, enterprises that were looking into cloud services usually undertook a long-term pilot with one of the large cloud providers, running test and dev workloads in the cloud. With cloud’s transition to mainstream adoption in 2015, and with enterprises migrating more and more workloads into the cloud and in between public and private environments, the single-provider approach must be revisited. In his session at 18th Cloud Expo, Yoav Mor, multi-cloud solution evangelist at Cloudy...
Jan. 22, 2017 08:30 PM EST Reads: 4,698