Jazz Pharmaceuticals Inc. submitted a new drug application for JZP-6 (sodium oxybate oral solution, also known as Xyrem) in fibromyalgia based on stellar Phase III data that sent the company's shares shooting up a whopping 300 percent in June.

And the stock has continued to climb. Since June 1, shares of the Palo Alto, Calif.-based firm have risen more than 700 percent, and cash-strapped Jazz took advantage, adding much-needed cash through a $7 million private placement in July. The firm ended the third quarter with about $21.4 million in cash, equivalents and marketable securities.

Shares (NASDAQ:JAZZ) closed Tuesday at $8.15, down 34 cents.

The NDA submission included data from two Phase III trials, both of which met their primary endpoint of showing that JZP-6 significantly decreased pain and fatigue, and also improved daily function, patient global impression of change and sleep quality in subjects suffering fibromyalgia, a musculoskeletal disorder characterized by widespread pain.

Data from the first three-month study in 548 patients showed that 54.2 percent of those treated with JZP-6 at 4.5 g per night and 58.5 percent treated with 6 g per night had significantly greater reduction in pain - defined clinically as greater than 30 percent - compared to 35.2 percent of patients in the placebo arm. (See BioWorld Today, June 16, 2009.)

The second study showed similar statistical significance, with 35 percent of patients in each of the JZP-6 treatment arms reporting pain relief compared to 20 percent of patients on placebo. (See BioWorld Today, June 26, 2009.)

If approved, JZP-6 would go up against pharma heavyweights Pfizer Inc., which markets Lyrica (pregabalin) and Eli Lilly and Co., which sells Cymbalta (duloxetine hydrochloride). An estimated 6 million people in the U.S. have fibromyalgia.

In Europe, partner UCB SA, of Brussels, Belgium, will handle the regulatory filing, though the path forward may be less clear overseas, given that the Committee for Medicinal Products for Human Use raised concerns about limited treatment effect with both Pfizer's and Lilly's drugs.

Jazz already markets JZP-6 as Xyrem to treat excessive daytime sleepiness and cataplexy. For the third quarter, Xyrem sales totaled about $25 million.

Alexza Files NDA, Seeks Partner for AZ-004

The lead candidate from Alexza Pharmaceuticals Inc.'s Staccato technology program made it to the FDA ahead of schedule, and the Mountain View, Calif.-based firm hopes to secure a partner for the drug, AZ-004, a version of loxapine to treat agitation in schizophrenic and bipolar patients, by the end of 2010.

The NDA was based on efficacy and safety data from more than 1,600 patients, including two pivotal trials in which AZ-004 met the primary and key secondary endpoints with statistically significant reductions in agitation vs. placebo.

Alexza is hoping the rapid onset provided by the Staccato system, as well as its administration via inhalation rather than intramuscular injection, will give it an edge in the agitation market.

Assuming a standard 10-month review, AZ-004 could hit the market in 2011. Piper Jaffray analyst Edward Tenthoff is "conservatively" forecasting sales of $218 million by 2015, based on a price of $25 per dose.

In the U.S., about 2.4 million people are diagnosed with schizophrenia and about 5.7 million are bipolar. Of those, more than 90 percent are likely to experience agitation, and Alexza said its market research showed that those patients experienced an average of 11 to 12 episodes of acute agitation each year.

Alexza, which regained full rights to AZ-004 (along with AZ0104 and AZ-002) from its acquisition of the Symphony Allegro joint venture from Symphony Capital, is seeking to partner the drug with a firm that has a sales force capable of reaching the psychiatry market, as well as the hospital market since about half of agitation episodes are treated in emergency settings. (See BioWorld Today, June 17, 2009.)

Earlier in the pipeline, the company is developing a Staccato version of prochlorperazine, AZ-001, which has completed Phase II testing in migraine and awaits a partner, and AZ-003, a Staccato fentanyl product in Phase I testing for breakthrough pain.

Alexza ended the third quarter with about $14.7 million, though it added another $19.1 million in an October financing. Those funds, along with proceeds from an employee stock purchase plan and option exercises, are expected to carry the company into the second half of 2010.

Shares of Alexza (NASDAQ:ALXA) gained 26 cents to close Tuesday at $2.55.