TorreyPines Therapeutics, Inc. (Nasdaq: TPTX) today announced the initiation of a Phase IIb clinical trial for its lead product candidate, tezampanel, a novel pain compound in development for the treatment of acute migraine. An AMPA/kainate (AK) receptor antagonist, tezampanel offers a non-narcotic, non-vascular approach to the management of headache pain and represents a potentially promising alternative to current migraine treatments.
The double-blind, placebo-controlled, parallel-group study will enroll approximately 300 patients and treat a single migraine attack, with or without aura. Equal numbers of patients will be randomized to one of four arms and receive a 40 mg, 70 mg, or 100 mg single subcutaneous dose of tezampanel or placebo. The primary efficacy endpoint is headache pain relief at two hours post-dose. Secondary efficacy endpoints include pain free at two hours, sustained pain relief and sustained pain free at 24 hours, and headache recurrence and relapse. Additional measures include assessments of functional disability and patient satisfaction, relief of migraine-associated symptoms such as nausea, vomiting, photophobia (sensitivity to light) and phonophobia (sensitivity to sound), as well as various assessments that characterize speed of onset. Safety, tolerability and pharmacokinetics will also be evaluated. The study will be conducted in approximately 25 centers in the U.S.
“The initiation of our Phase IIb trial with tezampanel is an important milestone for TorreyPines and a promising development for the millions of people who suffer with acute migraine,” said Neil Kurtz, M.D., President and Chief Executive Officer of TorreyPines. “We believe that tezampanel will not only relieve the acute pain associated with migraine, but also address the underlying mechanisms that precipitate the migraine. Tezampanel has the potential to offer an important new mechanism of action to the treatment of migraine.”
In previous clinical trials, tezampanel has been administered to more than 200 healthy adult volunteers or patients. In five Phase IIa, placebo-controlled trials, tezampanel demonstrated proof of concept in multiple pain models. In a placebo and active-controlled trial in patients with acute migraine, the compound, administered intravenously, achieved statistical significance in all primary and secondary endpoints traditionally required for regulatory approval. These endpoints included pain relief at two hours, pain-free at two hours and relief of nausea, photophobia and phonophobia.
TorreyPines’ follow-on compound, NGX426, is an oral prodrug of tezampanel and is in Phase I testing. Both compounds may effectively relieve severe and persistent pain through a novel mechanism that may not impart the side effects and risks associated with currently available migraine and other chronic pain treatments.
About AK Receptor Antagonists
AK receptor antagonists selectively block transmission of pain signals mediated through the activation of a subtype of glutamate receptors. These receptors play a critical role in the development of central sensitization phenomena — a key component of many pain syndromes, including migraine and persistent pain states such as chronic neuropathic pain. Because they do not block opioid receptors, constrict blood vessels or interact with systems external to the central nervous system at dosages that are therapeutically relevant, the safety profile of AK antagonists may offer important advantages over existing drugs.
Migraine is a painful neurological condition characterized by an intense and disabling episodic headache. According to the National Headache Foundation, an estimated 30 million people in the U.S. suffer from migraines. Approximately 25 percent of migraine sufferers endure at least one attack per week. In addition to headache pain, migraine attacks are frequently accompanied by sensitivity to light (photophobia) and sound (phonophobia), nausea and vomiting. Although once thought to be primarily caused by vascular changes in the brain, researchers now consider migraine a neurological disorder. In the U.S., the prescription migraine market is approximately $2 billion, with the market leader, Imitrex(R), accounting for approximately half of all sales.
About TorreyPines Therapeutics
TorreyPines Therapeutics, Inc. is a clinical stage biopharmaceutical company that discovers and develops small molecule drugs to treat diseases and disorders of the central nervous system (CNS). Led by an accomplished management team, TorreyPines is leveraging novel drug targets and technologies to potentially deliver new CNS therapies for migraine; chronic pain, including neuropathic pain; and cognitive disorders, including Alzheimer’s disease and cognitive impairment associated with schizophrenia. Further information is available at http://www.torreypinestherapeutics.com.
This press release contains forward-looking statements or predictions. Such forward-looking statements include statements regarding the potential for tezampanel and NGX426 as treatments for migraine and other pain indications and the potential of the company’s product candidates to treat certain diseases and disorders. Such statements are subject to numerous factors, risks, and uncertainties that may cause actual events or results to differ materially from the combined company’s current expectations. Statements regarding TorreyPines Therapeutics’ product candidates are subject to risks and uncertainties regarding development, regulatory approval and commercialization, including whether any preclinical studies or clinical trials, either ongoing or conducted in the future, will prove successful, and if successful, whether the results can be replicated; whether safety and efficacy profiles of any of its drug candidates will be established, or if established, will remain the same, be better or worse in future clinical trials, if any; whether pre-clinical results will be substantiated by ongoing or future clinical trials, if any, or whether any of its drug candidates will be able to improve the signs or symptoms of their respective clinical indication; whether any of its drug candidates will support an NDA filing, will be approved by the FDA or its equivalent, or if approved, will prove competitive in the market; or whether the necessary financing to support its drug development programs will be available. Actual results may differ materially from the above forward-looking statements due to a number of other important factors including that TorreyPines Therapeutics may not be able to execute its integration strategies or realize the expected benefits of its recently completed merger with Axonyx, Inc. These and other risks which may impact management’s expectations are described in greater detail in the registration statement on Form S-4, as amended, as filed with the Securities and Exchange Commission and Axonyx’s other SEC reports. TorreyPines Therapeutics undertakes no obligation to publicly release the result of any revisions to such forward-looking statements that may be made to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.
TorreyPines Therapeutics, Inc http://www.torreypinestherapeutics.com