Obtains global rights for treatment option in development for Dravet syndrome, a rare and catastrophic form of epilepsy that begins in infancy
Plans to initiate Phase 3 clinical studies of Brabafen™ in second quarter of 2015
SAN DIEGO, Calif., Oct. 27, 2014 (GLOBE NEWSWIRE) -- Zogenix, Inc. (Nasdaq:ZGNX), a pharmaceutical company developing and commercializing products for the treatment of pain-related and central nervous system (CNS) disorders, today announced that it acquired Brabant Pharma ("Brabant"), a privately-held U.K.-based pharmaceutical company for $20 million in cash and $15 million in stock plus potential future milestone and royalty payments. The acquisition includes worldwide development and commercialization rights to BrabafenTM, low-dose fenfluramine, for the treatment of Dravet syndrome, a rare and catastrophic form of intractable epilepsy that begins in infancy. Brabafen has recently received orphan drug designation in Europe and the U.S. for the treatment of Dravet syndrome.
Children with Dravet syndrome, also known as Severe Myoclonic Epilepsy of Infancy (SMEI), experience frequent, severe and potentially life-threatening seizures that typically start in the first year of life. These seizures do not respond to standard anti-epileptic medications and current treatment options are very limited.
The latest results from an ongoing, long-term clinical study in 15 Dravet syndrome patients treated with Brabafen show that 93% of patients were either seizure-free or had greatly reduced seizure frequency, during an average treatment period of greater than 12 years. The results also demonstrate the safety of Brabafen, which was shown to be well tolerated and treatment side effects were mild and transient for the entire study treatment period.
Joseph Sullivan, M.D, director of the University of California at San Francisco Pediatric Epilepsy Center, said, "Based on the available long-term clinical data, Brabafen has the potential to transform the quality of life for Dravet syndrome patients by eliminating or reducing seizures. The prognosis for seizure control in these patients is poor and current treatment alternatives offer little benefit. However, if the current positive results are demonstrated in controlled Phase 3 clinical trials, Brabafen could be a breakthrough treatment for patients with this catastrophic epilepsy syndrome."
The safety and effectiveness of Brabafen has been evaluated in a continuing, long-term, open-label, study in 15 Dravet syndrome patients. The average duration of treatment in this study is currently more than 12 years, with the longest duration of treatment at more than 26 years. More than two-thirds (67%) of patients were seizure free for at least a year after the latest assessment with an average seizure free period of 5.5 years. The majority (87%) of patients had a greater than 75% reduction in seizure frequency. There were no reports of pulmonary hypertension and there were no deaths. Two patients showed sub-clinical evidence of cardiac valve thickening that was judged to be clinically insignificant following detailed investigation by independent cardiologists. Similar findings spontaneously resolved in a third patient.
"Since securing an exclusive license from the Universities of Antwerp and Leuven in Belgium for the key clinical studies conducted by collaborators Berten Ceulemans, M.D., Ph.D. and Lieven Lagae, M.D., Ph.D., Brabant has made remarkable progress in defining the overall development program and regulatory filing strategy through formal meetings with the U.S. Food and Drug Administration and the European Medicines Agency. Based upon agency feedback, we plan to initiate two Phase 3 studies (of 40 to 60 Dravet syndrome patients per study) in the second quarter of next year in the U.S. and Europe, with top line results potentially available in the first half of 2016," said Stephen Farr, Ph.D., president of Zogenix. Dr. Farr continued, "With the addition of this potential breakthrough product candidate, Brabafen, to our development programs for two abuse deterrent formulations of extended-release hydrocodone and Relday, we believe Zogenix is poised to reach key clinical studies results and regulatory submissions every year from 2015 through 2018."
"This is a transformational acquisition for Zogenix. While we remain engaged in strategic opportunities for Zohydro ER, including pursuit of a co-promotion partner and development of abuse deterrent formulations, the addition of Brabant's Dravet syndrome program significantly enhances our CNS development pipeline," said Roger Hawley, chief executive officer of Zogenix.
"We believe the positive, durable long-term results of Brabafen place us in a leadership position within the Dravet syndrome community. We are fully committed to bringing this promising therapeutic to market for the benefit of these children and their families," added Mr. Hawley.
In addition to the $35 million acquisition cost in cash and Zogenix stock (11,995,202 shares to be registered), the terms of the purchase agreement includes $50 million in potential regulatory milestone payments and up to $45 million in royalty payments on Brabafen sales. The first milestone payment is not anticipated until late 2016 upon submission of the New Drug Application for Brabafen.
Zogenix also signed a non-binding term sheet which, subject to the negotiation and execution of definitive documentation, is anticipated to provide a $20 million term loan plus an additional $4 million in a revolving line of credit. This potential financing is expected to help fund research and development expenses for Brabafen and Relday, and the launch, if approved, of an abuse deterrent formulation of Zohydro ER.
Expenses related to the Phase 3 clinical programs for Brabafen are estimated at $10 million, with an additional $5 million in post-approval clinical expenses. The Company ended the third quarter of 2014 with approximately $50 million in cash plus an additional $8.5 million remaining in escrow from the sale of Sumavel DosePro earlier this year. The Company anticipates combined selling, general and administrative expenses and research and development expenses, including expenses related to the acquisition, for the second half of the year 2014 to be $50 million to $55 million, towards the low end of the full-year guidance range previously provided.
Conference Call & Webcast
Zogenix will hold a conference call today, October 27, 2014 at 4:30 p.m. ET to discuss the acquisition of Brabant Pharma. To participate, please dial (877) 417-5253 (U.S.) or (315) 625-3082 (International); participant pass code: 24426098. To access the live webcast, please visit the Zogenix Investor Relations website at http://ir.zogenix.com.
A replay of the conference call will be available beginning October 27, 2014 at 7:30 p.m. ET until November 3, 2014, by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (International); pass code: 24426098. A replay of the webcast will also be accessible on the Investor Relations website for one month, through November 27, 2014.
Discussion during the conference call may include forward-looking statements and any comments the Company may make about its future plans or prospects in response to questions from participants on the conference call.
Brabafen is low-dose fenfluramine currently under evaluation for the reduction of seizures in Dravet syndrome patients. Fenfluramine was originally developed and approved as an anoretic agent for the treatment of obesity. Although fenfluramine was withdrawn from the market in 1997 due to risk in the treated patient population of serious heart valve defects, preclinical and clinical evidence of the drug's ability to abolish epileptic seizures had previously been described. In Belgium, under a Royal Decree, the anticonvulsive effects of fenfluramine continued to be evaluated using the limited supply of drug in a clinical trial involving a small group of patients diagnosed with Dravet syndrome1. The study is continuing today with fenfluramine sourced by Brabant Pharma, manufactured from a synthetic process consistent with current regulatory standards for drug substances. Brabafen has been granted orphan drug designation by regulatory agencies in the U.S. and Europe. Phase 3 clinical studies are expected to be initiated by Zogenix in the second quarter of 2015.
About Dravet Syndrome
Dravet syndrome (also known as Severe Myoclonic Epilepsy of Infancy) is a rare, severe and therapy-resistant form of epilepsy most often caused by an identifiable gene defect that results in abnormal functioning of a sodium channel in the brain. Children with Dravet syndrome experience severe, long-lasting, fever-related seizures in the first year of life. Other seizures typically arise later, including myoclonus (involuntary muscle spasms) and status epilepticus (prolonged seizures), which often result in severe cognitive and developmental impairment. Episodes of status epilepticus require immediate emergency care and can be fatal.
Individuals with Dravet syndrome face a higher incidence of SUDEP (sudden unexplained death in epilepsy) and have associated conditions, which also require proper treatment and management. Children with Dravet syndrome do not outgrow this condition and it affects every aspect of their daily lives.
Zogenix, Inc. (Nasdaq:ZGNX) is a pharmaceutical company committed to developing and commercializing therapies that address specific clinical needs for people living with pain-related conditions and CNS disorders who need innovative treatment alternatives to help them return to normal daily functioning.
For more information, visit www.zogenix.com.
Zogenix cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. Words such as "believes," "indicates," "will," "intends," "suggests," "designed" and similar expressions are intended to identify forward-looking statements. These statements are based on the company's current beliefs and expectations. These forward-looking statements include statements regarding: the timing of the commencement of Phase 3 clinical trials for Brabafen; the receipt of top-line results from these clinical trials, and the potential timing of an NDA submission for Brabafen and related milestone payments; the potential for Brabafen to be a breakthrough treatment for Dravet syndrome; the expected timing of, and Zogenix's ability to achieve, key clinical milestones and regulatory submissions for Zogenix's development pipeline; the ability to secure debt financing and Zogenix's plans with respect to any proceeds from such financing; and the potential to secure co-promotion partners for, and develop and receive approval of an abuse deterrent formulation of, Zohydro ER; estimated clinical expenses for Brabafen; and expense guidance for 2014. Actual results may differ from those set forth in this release due to the risk and uncertainties inherent in Zogenix's business, including, without limitation: risks associated with the acquisition of Brabant and integration of Brabant's operations into Zogenix's business, including an increase in near and long-term expenditures, exposure to unknown liabilities and diversion of Zogenix's management's time and attention; the inherent risks of clinical development of an abuse deterrent formulation of Zohydro ER, Brabafen and Relday, and Zogenix's dependence on third parties in such development; the potential that earlier clinical trials may not be predictive of future results; unexpected adverse side effects or inadequate therapeutic efficacy of Zogenix's product candidates that could limit approval and/or commercialization, or that could result in recalls or product liability claims; other difficulties or delays relating to the development, testing, manufacturing and marketing of and obtaining regulatory approval for an abuse deterrent formulation of Zohydro ER; difficulties in identifying, negotiating and carrying out a co-promotions agreement for Zohydro ER; Zogenix's and its licensors' ability to obtain and maintain intellectual property protection for its product candidates; the risk that Zogenix will be unable to negotiate and enter into definitive agreements for a potential debt financing, on acceptable terms, or at all; Zogenix's ability to raise additional funding that it may need to continue to pursue its commercial and business development plans; fluctuations and volatility in Zogenix's stock price, including as a result of the issuance and possible resale of the shares granted as partial consideration for the acquisition of Brabant; competition from other pharmaceutical or biotechnology companies; and other risks detailed in Zogenix's 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, and Zogenix undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
1Ceulemans, Berten, et al. Successful use of fenfluramine as an add-on treatment for Dravet syndrome. EPILEPSIA. July 2012; 53(7):1131-1139.
Brabafen is a trademark of Brabant Pharma
CONTACT: Investors and Media
Zack Kubow | The Ruth Group
646.536.7020 | [email protected]