|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 essence of data analysis involves setting up data pipelines that consist of several operations that are chained together – starting from data collection, data quality checks, data integration, data analysis and data visualization (including the setting up of interaction paths in that visualization). In our opinion, the challenges stem from the technology diversity at each stage of the data pipeline as well as the lack of process around the analysis.
May. 24, 2016 11:30 PM EDT Reads: 997
Many banks and financial institutions are experimenting with containers in development environments, but when will they move into production? Containers are seen as the key to achieving the ultimate in information technology flexibility and agility. Containers work on both public and private clouds, and make it easy to build and deploy applications. The challenge for regulated industries is the cost and complexity of container security compliance. VM security compliance is already challenging, ...
May. 24, 2016 11:15 PM EDT Reads: 907
As cloud and storage projections continue to rise, the number of organizations moving to the cloud is escalating and it is clear cloud storage is here to stay. However, is it secure? Data is the lifeblood for government entities, countries, cloud service providers and enterprises alike and losing or exposing that data can have disastrous results. There are new concepts for data storage on the horizon that will deliver secure solutions for storing and moving sensitive data around the world. ...
May. 24, 2016 11:15 PM EDT Reads: 956
In his session at 18th Cloud Expo, Bruce Swann, Senior Product Marketing Manager at Adobe, will discuss how the Adobe Marketing Cloud can help marketers embrace opportunities for personalized, relevant and real-time customer engagement across offline (direct mail, point of sale, call center) and digital (email, website, SMS, mobile apps, social networks, connected objects). Bruce Swann has more than 15 years of experience working with digital marketing disciplines like web analytics, social med...
May. 24, 2016 10:45 PM EDT Reads: 1,041
Designing IoT applications is complex, but deploying them in a scalable fashion is even more complex. A scalable, API first IaaS cloud is a good start, but in order to understand the various components specific to deploying IoT applications, one needs to understand the architecture of these applications and figure out how to scale these components independently. In his session at @ThingsExpo, Nara Rajagopalan is CEO of Accelerite, will discuss the fundamental architecture of IoT applications, ...
May. 24, 2016 10:45 PM EDT Reads: 693
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. 24, 2016 09:00 PM EDT Reads: 1,789
Enterprise networks are complex. Moreover, they were designed and deployed to meet a specific set of business requirements at a specific point in time. But, the adoption of cloud services, new business applications and intensifying security policies, among other factors, require IT organizations to continuously deploy configuration changes. Therefore, enterprises are looking for better ways to automate the management of their networks while still leveraging existing capabilities, optimizing perf...
May. 24, 2016 08:45 PM EDT Reads: 924
What a difference a year makes. Organizations aren’t just talking about IoT possibilities, it is now baked into their core business strategy. With IoT, billions of devices generating data from different companies on different networks around the globe need to interact. From efficiency to better customer insights to completely new business models, IoT will turn traditional business models upside down. In the new customer-centric age, the key to success is delivering critical services and apps wit...
May. 24, 2016 06:30 PM EDT Reads: 736
With major technology companies and startups seriously embracing IoT strategies, now is the perfect time to attend @ThingsExpo 2016 in New York and Silicon Valley. Learn what is going on, contribute to the discussions, and ensure that your enterprise is as "IoT-Ready" as it can be! Internet of @ThingsExpo, taking place Nov 3-5, 2015, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with 17th Cloud Expo and will feature technical sessions from a rock star conference faculty ...
May. 24, 2016 06:00 PM EDT Reads: 4,645
The 19th International Cloud Expo has announced that its Call for Papers is open. Cloud Expo, to be held November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA, brings together Cloud Computing, Big Data, Internet of Things, DevOps, Containers, Microservices and WebRTC to one location. With cloud computing driving a higher percentage of enterprise IT budgets every year, it becomes increasingly important to plant your flag in this fast-expanding business opportunity. Submit y...
May. 24, 2016 05:00 PM EDT Reads: 1,840
Cloud computing changed data analytics for good. It enabled companies to drastically decrease resources and architecture previously assigned with business intelligence departments. It also enabled laymen to run advanced business analytics. Cloud was also the architecture of choice for storing and processing big data. Data piling is a continuous process, which is going to explode with emerging Internet of Things concept. Answer to this issue developers found in new concept called fog computing. ...
May. 24, 2016 04:15 PM EDT Reads: 1,819
There are several IoTs: the Industrial Internet, Consumer Wearables, Wearables and Healthcare, Supply Chains, and the movement toward Smart Grids, Cities, Regions, and Nations. There are competing communications standards every step of the way, a bewildering array of sensors and devices, and an entire world of competing data analytics platforms. To some this appears to be chaos. In this power panel at @ThingsExpo, moderated by Conference Chair Roger Strukhoff, panelists will discuss the vast to...
May. 24, 2016 04:00 PM EDT Reads: 2,351
Internet of @ThingsExpo, taking place November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with the 19th International Cloud Expo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world and ThingsExpo New York Call for Papers is now open.
May. 24, 2016 04:00 PM EDT Reads: 1,679
SYS-CON Events announced today that Enzu, a leading provider of cloud hosting 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. Enzu’s mission is to be the leading provider of enterprise cloud solutions worldwide. Enzu enables online businesses to use its IT infrastructure to their competitive advantage. By offering a suite of proven hosting and management services, Enzu wants companies to foc...
May. 24, 2016 02:15 PM EDT Reads: 2,073
SYS-CON Events announced today the How to Create Angular 2 Clients for the Cloud Workshop, being held June 7, 2016, in conjunction with 18th Cloud Expo | @ThingsExpo, at the Javits Center in New York, NY. Angular 2 is a complete re-write of the popular framework AngularJS. Programming in Angular 2 is greatly simplified. Now it’s a component-based well-performing framework. The immersive one-day workshop led by Yakov Fain, a Java Champion and a co-founder of the IT consultancy Farata Systems and...
May. 24, 2016 02:00 PM EDT Reads: 3,842