News Feed Item

Jazz Pharmaceuticals Announces Acquisition From Aerial Biopharma Of Rights To A Late Stage Investigational Compound For Excessive Daytime Sleepiness

-- Terms include $125 million upfront payment in cash plus a potential of up to $272 million in milestone payments

-- Investor conference call to be held today, January 13 at 8:30 AM EST/1:30 PM GMT 

DUBLIN, Jan. 13, 2014 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and Aerial BioPharma, LLC (Aerial) today announced that the companies have signed a definitive agreement under which Jazz Pharmaceuticals has acquired rights to ADX-N05, a novel compound in clinical development for the treatment of excessive daytime sleepiness in patients with narcolepsy. 

Under the agreement, a subsidiary of Jazz Pharmaceuticals has acquired worldwide development, manufacturing and commercial rights to ADX-N05, other than in certain countries in Asia where SK Biopharmaceuticals Co., Ltd (SK) retains rights(1). Under the agreement, Aerial will receive an upfront payment of $125 million.  Aerial and SK are eligible to receive milestone payments based on development, regulatory and sales milestones and tiered royalties based on potential future sales.

"ADX-N05 is a strong fit with our specialty focus and continues our commitment to develop and bring to market differentiated treatments for patients with sleep-related disorders," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals plc.  "This acquisition supports our mission to improve care for patients with serious medical conditions. We look forward to accelerating the development of ADX-N05 as part of our long-term strategy of building a development pipeline and expanding our portfolio of commercial products through targeted investments that offer potential new treatment options for patients, with the objective of creating significant value for our shareholders."

"The addition of ADX-N05 to our development pipeline is an excellent strategic fit for Jazz given our strong clinical expertise in sleep and narcolepsy," said Jeffrey Tobias, MD, executive vice president of research and development and chief medical officer of Jazz Pharmaceuticals. "The ADX-N05 Phase 2b results, announced by Aerial in October 2013, demonstrated a robust effect on excessive daytime sleepiness in patients with narcolepsy. Given ADX-N05's demonstrated wake-promoting properties in pre-clinical and clinical studies, including the Phase 2b results, we believe ADX-N05 could also potentially benefit patients whose excessive daytime sleepiness stems from other causes, such as obstructive sleep apnea, where we also intend to pursue Phase 3 clinical trials. We look forward to discussing our development plans with the U.S. Food and Drug Administration and initiating our Phase 3 clinical program for ADX-N05 as quickly as possible."

Excessive daytime sleepiness is a common symptom for patients with narcolepsy and obstructive sleep apnea (OSA).  Despite current therapies, many patients with narcolepsy and OSA continue to experience excessive daytime sleepiness(2,3).  Narcolepsy is a chronic, debilitating, orphan condition that impacts approximately 157,000 people in the United States(4).  Less than half of the estimated 157,000 people living with narcolepsy in the United States have been properly diagnosed and approximately 50,000 patients receive wake-promoting therapies. OSA is a serious chronic sleep disorder in which breathing repeatedly stops and starts during sleep.  People living with excessive daytime sleepiness in narcolepsy and OSA are often inadequately treated with available wake-promoting agents.  In the United States, approximately 500,000 patients receive wake-promoting therapies for excessive daytime sleepiness associated with OSA(5).

Conference Call Information
Jazz Pharmaceuticals will host a conference call and live audio webcast today at 8:30 am EST/1:30 pm GMT to discuss this transaction and related matters. Interested parties may access the live audio webcast and slide presentation via the Investors & Media section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary to listen to the webcast. A replay of the webcast will be archived on the website for one week.

Audio Webcast/Conference Call:
U.S. Dial-In Number: +1 800 510 9691
Outside the U.S. Dial-In Number: +1 617 614 3453
Passcode: 87477314

A replay of the conference call will be available through January 20, 2014 and accessible through one of the following telephone numbers and entering the passcode:

Replay U.S. Dial-In Number: +1 888 286 8010
Replay Outside the U.S. Dial-In Number: +1 617 801 6888
Passcode: 50055465

Jazz Pharmaceuticals Advisor
Cooley LLP served as legal advisor to Jazz on this transaction.

Aerial BioPharma Advisors
Piper Jaffray & Co. served as exclusive financial advisor and Hutchison Law Group served as legal advisor to Aerial on this transaction.

About ADX-N05
ADX-N05 is a novel, investigational compound in clinical development for the treatment of excessive daytime sleepiness in patients with narcolepsy.  While the mechanism of action is not well understood, the molecule has demonstrated wake-promoting properties in pre-clinical and clinical studies.  In two Phase 2 studies, ADX-N05 has demonstrated a statistically significant effect on excessive daytime sleepiness in patients with narcolepsy. 

Results from Aerial's Phase 2a clinical trial evaluating the efficacy and safety of ADX-N05 for the treatment of excessive daytime sleepiness in adult subjects with narcolepsy were presented at the SLEEP 2013 meeting in Baltimore, MD in June 2013(6).  A total of 33 patients were randomized and completed both ADX-N05 and placebo periods in this double-blind, placebo-controlled, multicenter, crossover study.  The primary efficacy endpoint was the change in the Maintenance of Wakefulness Test (MWT); and secondary endpoints included Epworth Sleepiness Scale (ESS) and the Clinical Global Impression-Change (CGI-C).  The study met both the primary and secondary endpoints.  The average sleep latency on the MWT, an objective measure of the severity of excessive daytime sleepiness, was 11.8 minutes longer with ADX-N05 treatment than with placebo (p=0.0002). On the ESS, a subjective patient-completed measure of sleepiness, ADX-N05 was superior to placebo following 1 week (p<0.0001) and 2 weeks (p=0.0002) of treatment. The CGI-C rating, a subjective physician-completed overall improvement scale, showed that at 1 and 2 weeks of treatment, 88% and 76% of subjects improved on ADX-N05 versus 27% and 39%, respectively, on placebo (p<0.001, p=0.0016, respectively). ADX-N05 was generally well-tolerated during the trial.

The Phase 2b clinical trial in excessive daytime sleepiness in narcolepsy enrolled 93 patients.  Patients received treatment with placebo or ADX-N05 for a 12 week treatment period.  The study's primary and secondary endpoints were met (including objective and subjective primary endpoints: MWT and CGI; and secondary endpoints: Epworth Sleepiness Scale and Patient Global Improvement), and replicated the earlier Phase 2a study with highly statistically significant results at both 4 weeks and 12 weeks.  ADX-N05 was generally well tolerated at doses of 150 to 300 mg during the trial.  Results from the Phase 2b study will be submitted for presentation at a future scientific meeting.

ADX-N05 has previously been evaluated in several Phase 1 and Phase 2 studies.  In those studies, ADX-N05 was generally well-tolerated and demonstrated an acceptable safety profile in more than 500 patient exposures.

About Jazz Pharmaceuticals
Jazz Pharmaceuticals plc is a specialty biopharmaceutical company focused on improving patients' lives by identifying, developing and commercializing innovative products that address unmet medical needs.  The company has a diverse portfolio of products in the areas of narcolepsy, oncology, pain and psychiatry.  The company's U.S. marketed products in these areas include: Xyrem® (sodium oxybate) oral solution, Erwinaze® (asparaginase Erwinia chrysanthemi), Prialt® (ziconotide) intrathecal infusion, FazaClo® (clozapine, USP) HD and FazaClo LD.  Outside of the U.S., Jazz Pharmaceuticals also has a number of products marketed by its EUSA Pharma division. For further information, see www.jazzpharmaceuticals.com.

"Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995
This press release contains forward-looking statements, including, but not limited to, statements related to the therapeutic and commercial potential of ADX-N05, planned future discussions with the U.S. Food and Drug Administration concerning Jazz Pharmaceuticals' anticipated development of ADX-N05, potential future clinical trials and other development of ADX-N05 planned to be conducted by the company and the anticipated timing thereof, including the indications that the company plans to pursue, potential commercialization of ADX-N05 by the company, the company's pipeline and portfolio growth strategy and other statements that are not historical facts.  These forward-looking statements are based on Jazz Pharmaceuticals' current expectations and inherently involve significant risks and uncertainties.  Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the difficulty and uncertainty of pharmaceutical product development, including the timing and cost thereof, the risk that results from early clinical trials may not be predictive of results obtained in later and larger Phase 3 clinical trials and the uncertainty of clinical success and regulatory approval; the company's ability to successfully manage the risks associated with integrating ADX-N05 and any other products or product candidates the company may acquire in the future into the company's product portfolio, including the availability of funding to complete the development of, obtain regulatory approval for and commercialize ADX-N05 and any other potential future acquired product candidates; the possibility that the company may fail to realize the anticipated benefits (commercial or otherwise) from ADX-N05; the company's ability to identify and acquire, in-license or develop additional products or product candidates to grow its business; and possible restrictions on the company's ability and flexibility to pursue certain future opportunities as a result of its substantial outstanding debt obligations; as well as risks related to future opportunities and plans; and those other risks detailed from time-to-time under  the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including the Quarterly Report on Form 10-Q for the quarter ended September 30, 2013 and future filings and reports by the company.  Jazz Pharmaceuticals undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.


  1. SK has retained rights to ADX-N05 in Korea, Japan, China, Taiwan, Singapore, Indonesia, India, Philippines, Thailand, Malaysia, Vietnam and Hong Kong.
  2. NA Antic et al. The effect of CPAP in normalizing daytime sleepiness, quality of life, and neurocognitive function in patients with moderate to severe OSA. Sleep. 2011: Jan 1; 34(1):111-9.
  3. C Guilleminault et al. Problems associated with switch to modafinil – a novel alerting agent in narcolepsy. Eur J Neurol.  2000 Jul; 7(4):381-4.
  4. CR Baumann et al. Narcolepsy: Pathophysiology, Diagnosis, and Treatment.  Springer: NY 2011.
  5. SH Launois et al. Current Opinion in Pulmonary Medicine. 19(6):601-608, November 2013.
  6. RK Bogan et al., Poster 0747, Presented at the 27th Annual Meeting of the Associated Professional Sleep Societies June 1-5, 2013, Baltimore, MD.

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
SYS-CON Events announced today that eCube Systems, the leading provider of modern development tools and best practices for Continuous Integration on OpenVMS, will exhibit 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. eCube Systems offers a family of middleware products and development tools that maximize return on technology investment by leveraging existing technical equity to meet evolving business needs. ...
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.
Amazon has gradually rolled out parts of its IoT offerings, but these are just the tip of the iceberg. In addition to optimizing their backend AWS offerings, Amazon is laying the ground work to be a major force in IoT - especially in the connected home and office. In his session at @ThingsExpo, Chris Kocher, founder and managing director of Grey Heron, explained how Amazon is extending its reach to become a major force in IoT by building on its dominant cloud IoT platform, its Dash Button strat...
Without lifecycle traceability and visibility across the tool chain, stakeholders from Planning-to-Ops have limited insight and answers to who, what, when, why and how across the DevOps lifecycle. This impacts the ability to deliver high quality software at the needed velocity to drive positive business outcomes. In his general session at @DevOpsSummit at 19th Cloud Expo, Eric Robertson, General Manager at CollabNet, will discuss how customers are able to achieve a level of transparency that e...
Two weeks ago (November 3-5), I attended the Cloud Expo Silicon Valley as a speaker, where I presented on the security and privacy due diligence requirements for cloud solutions. Cloud security is a topical issue for every CIO, CISO, and technology buyer. Decision-makers are always looking for insights on how to mitigate the security risks of implementing and using cloud solutions. Based on the presentation topics covered at the conference, as well as the general discussions heard between sessi...
For basic one-to-one voice or video calling solutions, WebRTC has proven to be a very powerful technology. Although WebRTC’s core functionality is to provide secure, real-time p2p media streaming, leveraging native platform features and server-side components brings up new communication capabilities for web and native mobile applications, allowing for advanced multi-user use cases such as video broadcasting, conferencing, and media recording.
Fifty billion connected devices and still no winning protocols standards. HTTP, WebSockets, MQTT, and CoAP seem to be leading in the IoT protocol race at the moment but many more protocols are getting introduced on a regular basis. Each protocol has its pros and cons depending on the nature of the communications. Does there really need to be only one protocol to rule them all? Of course not. In his session at @ThingsExpo, Chris Matthieu, co-founder and CTO of Octoblu, walk you through how Oct...
DevOps is speeding towards the IT world like a freight train and the hype around it is deafening. There is no reason to be afraid of this change as it is the natural reaction to the agile movement that revolutionized development just a few years ago. By definition, DevOps is the natural alignment of IT performance to business profitability. The relevance of this has yet to be quantified but it has been suggested that the route to the CEO’s chair will come from the IT leaders that successfully ma...
Major trends and emerging technologies – from virtual reality and IoT, to Big Data and algorithms – are helping organizations innovate in the digital era. However, to create real business value, IT must think beyond the ‘what’ of digital transformation to the ‘how’ to harness emerging trends, innovation and disruption. Architecture is the key that underpins and ties all these efforts together. In the digital age, it’s important to invest in architecture, extend the enterprise footprint to the cl...
Almost everyone sees the potential of Internet of Things but how can businesses truly unlock that potential. The key will be in the ability to discover business insight in the midst of an ocean of Big Data generated from billions of embedded devices via Systems of Discover. Businesses will also need to ensure that they can sustain that insight by leveraging the cloud for global reach, scale and elasticity.
@DevOpsSummit has been named the ‘Top DevOps Influencer' by iTrend. iTrend processes millions of conversations, tweets, interactions, news articles, press releases, blog posts - and extract meaning form them and analyzes mobile and desktop software platforms used to communicate, various metadata (such as geo location), and automation tools. In overall placement, @DevOpsSummit ranked as the number one ‘DevOps Influencer' followed by @CloudExpo at third, and @MicroservicesE at 24th.
A critical component of any IoT project is what to do with all the data being generated. This data needs to be captured, processed, structured, and stored in a way to facilitate different kinds of queries. Traditional data warehouse and analytical systems are mature technologies that can be used to handle certain kinds of queries, but they are not always well suited to many problems, particularly when there is a need for real-time insights.
One of biggest questions about Big Data is “How do we harness all that information for business use quickly and effectively?” Geographic Information Systems (GIS) or spatial technology is about more than making maps, but adding critical context and meaning to data of all types, coming from all different channels – even sensors. In his session at @ThingsExpo, William (Bill) Meehan, director of utility solutions for Esri, will take a closer look at the current state of spatial technology and ar...
Without lifecycle traceability and visibility across the tool chain, stakeholders from Planning-to-Ops have limited insight and answers to who, what, when, why and how across the DevOps lifecycle. This impacts the ability to deliver high quality software at the needed velocity to drive positive business outcomes. In his session at @DevOpsSummit 19th Cloud Expo, Eric Robertson, General Manager at CollabNet, will show how customers are able to achieve a level of transparency that enables everyon...
Explosive growth in connected devices. Enormous amounts of data for collection and analysis. Critical use of data for split-second decision making and actionable information. All three are factors in making the Internet of Things a reality. Yet, any one factor would have an IT organization pondering its infrastructure strategy. How should your organization enhance its IT framework to enable an Internet of Things implementation? In his session at @ThingsExpo, James Kirkland, Red Hat's Chief Arch...