Transcept And Shin Nippon Biomedical Laboratories Announce Global Licensing Agreement For Advanced Acute Migraine Treatment

POINT RICHMOND, Calif. and TOKYO, Sept. 25, 2013 /PRNewswire/ -- Transcept Pharmaceuticals, Inc. (NASDAQ: TSPT), and Shin Nippon Biomedical Laboratories, Ltd ("SNBL"), today announced that the companies have entered into an exclusive worldwide licensing agreement for a novel, rapidly absorbed treatment for acute migraine incorporating dihydroergotamine (DHE) as the active drug.  The agreement grants Transcept the global development and commercialization rights to the product candidate, designated TO-2070, which is expected to offer a new and potentially improved approach to the treatment of acute migraine.  Preclinical data suggest that TO-2070 may offer significant migraine treatment benefits beyond those provided by less convenient and more invasive DHE drug delivery methods, such as injection, liquid nasal sprays or pulmonary inhalation.  TO-2070 is designed to provide these benefits at a relatively low cost via a unique and proprietary nasal powder drug delivery system that can be easily self-administered by patients.

Under the terms of the agreement, Transcept has agreed to pay SNBL an upfront technology license fee of $1 million, development milestones totaling $6.5 million through New Drug Application (NDA) approval and, following the launch of the product, commercialization milestones tied to the achievement of specified annual sales levels totaling up to $35 million.  Additionally, Transcept will pay tiered, low double-digit royalties on annual net sales.  The parties anticipate entering into further agreements under which SNBL would supply Transcept with nasal powder delivery devices and would provide Transcept with certain preclinical and clinical services to support TO-2070 development.

Glenn A. Oclassen, President and CEO of Transcept, stated as follows:  "For more than 55 years, SNBL has been a leader in the field of biopharmaceutical research, and this nasal powder drug delivery system is the latest example of its innovative spirit.  Our TO-2070 agreement enables Transcept and SNBL to work together to develop a potentially important new therapeutic for acute migraine, which is among the most common neurological disorders. There are approximately 30 million migraine cases each year in the United States alone, and we estimate that the worldwide therapeutic market is over $3 billion.  However, current treatment options are not adequate to meet the needs of patients.  We believe that TO-2070 represents a significant potential therapeutic advance that could offer patients and physicians rapid and meaningful relief of migraine symptoms. TO-2070 will offer these advantages in a DHE treatment system that does not have the problems associated with intranasal liquid, injectable or pulmonary delivery, that can be conveniently self-administered, and that has a relatively low manufacturing cost." 

DHE is a well-established acute migraine treatment, especially among migraine specialists, and has been approved for use in the United States since 1946.  However, it has poor oral bioavailability and is not available in an oral dosage form.  Instead, DHE is generally administered via subcutaneous (SC), intramuscular (IM) or intravenous (IV) injection.  It is also available as a liquid nasal spray; however, the performance of that product is limited by an inconvenient administration regimen, slow onset of relief, inconsistent absorption, post-nasal drip and bitter taste.  A DHE product that is delivered via pulmonary inhalation is under review by the Food and Drug Administration.

TO-2070 is based on a novel nasal powder drug delivery technology created by SNBL that combines a muco-adhesive drug carrier with a proprietary nasal powder drug delivery device.  This system is the product of a decade-long SNBL internal development program with the objective of unlocking the significant market potential for nasal drug delivery by overcoming many of the obstacles that have limited its application to date.  Unlike previous nasal drug delivery technologies, the SNBL system is able to consistently deliver drug into the nasal cavity with each administration and hold it in place on the nasal mucosa to enable enhanced absorption into the bloodstream. Delivery of the dry powder drug formulation to the nasal mucosa via the specially designed device nozzle is achieved by simply squeezing the device.  Animal model data have shown pharmacokinetic (PK) results that are similar to subcutaneous and intramuscular drug administration of DHE, suggesting the potential for migraine relief heretofore achievable only with injectable DHE dosage forms.  Furthermore, the easy-to-use delivery device allows patients to self-administer their treatment without the need to manipulate complicated devices, or to visit their physician or emergency room.

"The decision by Transcept to license this promising new migraine therapeutic provides an important validation for our breakthrough drug delivery platform, which we believe is positioned to trigger a renewed focus on the broad potential of nasal drug delivery.  We believe that this new relationship with Transcept, with its track record of successfully advancing a neuroscience product all the way through NDA approval, provides an ideal opportunity to bring our novel technology to market," said Ryoichi Nagata M.D. Ph.D., SNBL CEO and President.

Alan Rapoport, M.D., Clinical Professor of Neurology at the David Geffen School of Medicine at UCLA, Los Angeles, CA, and President of the International Headache Society commented: "DHE has proven to be an excellent treatment for many of the migraine patients who have tried it.  This medication can work quickly depending on how it is administered, provides long-lasting relief, has demonstrated a low migraine recurrence rate and can be effectively administered late in the migraine process.  Despite these important advantages, the therapeutic impact of DHE has been somewhat limited by challenges associated with the effective delivery of the drug.  With this in mind, a novel delivery system that enables the administration of DHE in a convenient way and offers patients rapid and sustained migraine relief, would represent an important advance in the treatment of migraine."

Based on the long history of use and established safety profile of DHE, and the data from the animal model PK study indicating that TO-2070 has the potential to deliver therapeutic doses of DHE as rapidly as an IM or SC injection, Transcept is planning the first human PK study for the second half of 2014.  This study is expected to be followed by a meeting with FDA to discuss how Transcept intends to pursue a 505(b)(2) regulatory pathway for TO-2070.  Transcept believes that this regulatory strategy provides key development benefits associated with reduced cost, mitigated risk and potentially expedited approval. 

In addition to TO-2070, Transcept and SNBL are evaluating further potential opportunities for new neuroscience products incorporating the SNBL technology that would deliver meaningful clinical benefits to patients.

About Transcept
Transcept Pharmaceuticals, Inc. is a specialty pharmaceutical company focused on the development and commercialization of proprietary products to address important therapeutic needs in the field of neuroscience.  The company's lead development candidate is TO-2070, a novel, rapidly absorbed treatment for acute migraine incorporating dihydroergotamine (DHE) as the active drug.  Preclinical data suggest that TO-2070 may offer significant migraine treatment benefits beyond those provided by less convenient and more invasive DHE drug delivery methods, such as injection, liquid nasal sprays or pulmonary inhalation. 

In addition to advancing TO-2070, Transcept is continuing to evaluate opportunities to supplement its development pipeline with new programs appropriate to its area of expertise.  The company's management team developed Intermezzo^® from concept to its approval by the FDA in 2011.  Purdue holds commercialization and development rights for Intermezzo in the United States. For further information about Transcept, please visit www.transcept.com. For information about Intermezzo, please visit www.MyIntermezzo.com.

About Shin Nippon Biomedical Laboratories (SNBL)
For more than 55 years, SNBL has been a leader in providing development services to biopharmaceutical companies, generating high quality data with the exceptional precision needed to rapidly advance new medical therapies and innovations that improve patients' lives. 

In addition to providing services, SNBL has a Translational Research business, which utilizes its development capabilities to develop value-adding technologies and provides them to the pharmaceutical and medical industry.  Under such business, SNBL has developed μco™ System, a novel intranasal drug delivery technology platform, to provide effective delivery solutions to pharmaceutical products.  For further information about SNBL, please visit http://www.snbl.com. For information about SNBL's nasal drug delivery technology, please visit http://www.snbl-nds.co.jp/en/.

Forward Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties regarding Transcept.  All statements, other than statements of historical facts, included in this press release regarding Transcept's strategy, future operations, future financial position, future revenues, projected expenses, prospects, plans and objectives of management are forward-looking statements.  Examples of such statements include, but are not limited to, statements relating to the following:  TO-2070 offers a new and improved approach to the treatment of acute migraine; the potential for TO-2070 to be superior to existing migraine treatments, including by offering significant benefits at relatively low cost; the benefits and nature of SNBL's nasal drug delivery system with respect to TO-2070 and as compared to existing delivery systems, including convenience, cost, ease of self-administration, safety and speed; the amount, timing and nature of payments by Transcept to SNBL under the TO-2070 licensing agreement; the nature of Transcept's relationship and collaboration with SNBL and market opportunities pursuant to such relationship; Transcept's estimates regarding the potential size of the market for migraine treatments; beliefs regarding the inadequacy of existing treatment options for migraine; the potential market importance of TO-2070 and SNBL's nasal drug delivery system; the occurrence and timing of Transcept's planned FDA meeting and initial human PK study relating to TO-2070; and the potential benefits of Transcept's regulatory strategy. Transcept may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in its forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements.  Various important factors relating to Transcept could cause actual results or events to differ materially from the forward-looking statements that Transcept makes, including the following: achieving acceptance of Intermezzo by physicians, patients and third party payors; Transcept's dependence on its collaboration with Purdue; supplying sufficient quantities of Intermezzo from third party manufacturers and suppliers to meet anticipated market demand; the impact of competitive products and the market for Intermezzo generally; obtaining, maintaining and protecting regulatory exclusivity and intellectual property protection for Intermezzo; Transcept's ability to identify and finance additional product candidates for in-licensing or acquisition; and the ability of Transcept to obtain additional funding, if needed, to support its business activities.   These and other risks are described in greater detail in the "Risk Factors" section of Transcept periodic reports filed with the SEC .  Forward-looking statements do not reflect the potential impact of any future in-licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments Transcept may enter into or make.  Transcept does not assume any obligation to update any forward-looking statements, except as required by law.

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SOURCE Transcept Pharmaceuticals, Inc.