Phase III trial demonstrates rapid efficacy of Zelrix in treating acute migraine

www.nupathe.comSep 10 2009

NuPathe Inc., a specialty pharmaceutical company developing innovative products for the treatment of neurological and psychiatric diseases, today announced data from the pivotal Phase III trial of Zelrix™, a novel transdermal patch in clinical development for the treatment of acute migraine. Zelrix combines NuPathe’s proprietary SmartRelief™ iontophoretic transdermal technology with sumatriptan, the most prescribed treatment for acute migraine in the United States. The Phase III trial was conducted in 530 adults and was administered in a multi-center, randomized, parallel group, double-blind, placebo-controlled trial, where efficacy and tolerability of Zelrix were compared with placebo.

Zelrix met the primary efficacy endpoint of a statistically significant improvement compared to placebo for pain freedom at two hours after patch application (18 percent vs. 9 percent,>

• Pain relief: 53 percent of patients treated with Zelrix compared with 29 percent for placebo (p<0.0001);
• Nausea free: 84 percent of patients treated with Zelrix compared with 63 percent for placebo (p<0.0001);
• Photophobia free: 51 percent of patients treated with Zelrix compared with 36 percent for placebo>
• Phonophobia free: 55 percent of patients treated with Zelrix compared with 39 percent for placebo>

In addition to meeting the primary and key secondary endpoints, Zelrix demonstrated rapid efficacy and sustained pain relief with lower use of rescue medication:

• Pain relief within one hour: 29 percent of patients treated with Zelrix compared with 19 percent for placebo>
• Nausea free within one hour: 71 percent of patients treated with Zelrix compared with 58 percent for placebo>
• Sustained pain relief (from two to 24 hours): 34 percent of patients treated with Zelrix compared with 21 percent for placebo>
• Use of rescue medication: 40 percent of patients treated with Zelrix compared with 60 percent for placebo (p<0.0001).

Zelrix was well-tolerated in the clinical trial. Skin tolerability was typical of other transdermal products with mild to moderate erythema present upon patch removal. The incidence of triptan-specific adverse events was very low (2 percent for Zelrix). The most common adverse event was application site pain, having been reported by 23 percent of patients with Zelrix compared to 15 percent with placebo. The next most common adverse event was application site tingling, with 12 percent reported with Zelrix compared to 19 percent with placebo. The majority of adverse events were reported as mild and transient.

“The Zelrix patch is a highly-effective, well tolerated, non-oral treatment for migraine,” said Jerome Goldstein, MD, founder of the Headache and Facial Pain Section of the American Academy of Neurology and Director of the San Francisco Headache Clinic and Clinical Research Center. “Treatment-altering nausea, medication side effects, and inconsistent drug absorption are common problems that prevent many patients from effectively treating their migraines. The data from the clinical trial indicates that Zelrix could benefit many migraine patients.”

“Transdermal delivery has been recognized as an ideal approach to treating migraine, but has not been achieved to date,” said Jane Hollingsworth, chief executive officer of NuPathe. “Using NuPathe’s SmartRelief technology, Zelrix is poised to be the first and only transdermal patch approved for the treatment of acute migraine. This breakthrough is an important milestone for NuPathe and millions of migraine sufferers. We plan to file an NDA for Zelrix in 2010 and look forward to bringing this important innovation to patients.”