(INTRODUCTION - FEW BRIEF WORDS BY ME)
This is my first update to the Alexza Pharmaceuticals Summary Thread. Expect more updates from me in the future. Included in this update is information from Alexza's Q1 2010 Earnings Release and Conference Call released on May 10th and Alexza's May 11th Presentation at the JMP Securities Research Conference.
I encourage everybody to do their own due diligence and make their own decisions about their investments and finances. Nobody knows your own finances and investment comfortability better than yourself. Never take another message board user's word at face value for anything (including my own). Double check. Do your own due diligence. Make your own decisions. If you like what you see, please help me keep this bumped to the top of the first page by making occasional replies. If I made any mistakes, please let me and everybody else here know. If you have anything you with to contribute, post it as a reply. I'll try to keep this summary updated periodically with contributions made by others here and as new information or news becomes known.
Because of the length of the summary and the character restrictions for Yahoo! replies, the summary will be broken up into multiple replies. Each section will be posted directly below the previous section.
(PART ONE - SUMMARY OF AZ-004: ITS NDA FILING)
Alexza Pharmaceuticals (NASDAQ: ALXA) has a drug, AZ-004 (Staccato Loxapine), up for FDA approval. The AZ-004 NDA is a combination drug-device NDA (meaning that both drug and device will be up for approval consideration together using the same New Drug Application). The Prescription Drug User Fee Act (PDUFA) date for this NDA is set by the FDA for October 11, 2010. Since 2004 when its IND was filed, AZ-004 has a 1,600 patient NDA database from 13 clinical trials. The Stacatto device has almost 3,000 administrations during all of its clinical trials.
AZ-004 showed very strong and positive Phase 3 date from two pivotal studies for schizophrenia and bipolar disorder; 658 patients enrolled in two Phase 3 trials. Both Phase 3 trials met their primary and secondary endpoints. Rapid onset was established in both Phase 3 trial patient populations. Both Phase three trials showed a very strong safety profile in which the drug was well tolerated in patients.
Phase III supplemental data provided in May showed strong results and an advantage over the other therapies for agitation in schizophrenia and bipolar disorder patients. This advantage meets an unmet need in this market. All institutional analysts covering this stock are confident of approval as well.
Huge upside...millions of people can be cured if FDA approval..imo
I also wanted to say thanks for the nice summary of ALXA. I saw this stock mentioned on another thread and coming here and reading your summary is a great way for me to start my DD. Thanks for the informative posts.
(PART FOUR - PHASE 3 STUDY FOR SCHIZOPHRENIA AND PHASE 3 STUDY FOR BIPOLAR DISORDER)
Alexza Pharmaceuticals successfully completed two separate Phase 3 trials: a 344-patient trial for schizophrenia and a 314-patient trial for bipolar disorder. In total, the AZ-004 NDA contains efficacy and safety data from more than 1,600 patients and subjects who have been studied in 13 different clinical trials. Both trials met primary and key secondary endpoints. The company submitted an NDA on December 11, 2009 and accepted on February 11, 2010. The FDA has set the Prescription Drug User Fee Act (PDUFA) goal date for October 11, 2010.
In September of 2008, ALXA announced positive results from the first Phase 3 clinical trial of AZ-004 in schizophrenic patients with acute agitation. This Phase 3 clinical trial was an in-clinic, multi-center, randomized, double-blind, placebo-controlled study and tested AZ-004 at two dose levels, 5 mg and 10 mg. It enrolled 344 schizophrenic patients with acute agitation at 24 U.S. clinical centers.
In December of 2008, ALXA announced positive results from the second Phase 3 clinical trial of AZ-004 in bipolar disorder patients with acute agitation. As with the previous clinical trial for schizophrenic patients, this Phase 3 clinical trial for bipolar disorder patients was also an in-clinic, multi-center, randomized, double-blind, placebo-controlled study and tested AZ-004 at two dose levels, 5 mg and 10 mg. It enrolled 314 acutely-agitated patients with bipolar disorder at 17 U.S. clinical centers.
The results of both clinical trials for schizophrenia and bipolar disorder showed very positive results. Both doses of AZ-004 (5 and 10 mg) met the primary and key secondary endpoints of the studies, with highly statistically significant reductions in agitation, as compared to placebo. The primary endpoint was reduction in agitation as measured by the PEC at 2 hours. The key secondary endpoint was reduction in agitation as measured by the CGI-I at 2 hours. Additionally, the 10 mg dose of AZ-004 exhibited a rapid onset of effect, with statistically significant reductions in agitation at 10 minutes post-dose, the first time point measured. The reduction of agitation was sustained through the 24-hour study period. The 10 mg dose sustained this statistically significant improvement at all measurement time points throughout the 24-hour study period, compared to placebo. In both studies, the administration of AZ-004 was generally safe and well tolerated. In 2009, Alexza initiated and completed five non-pivotal safety and NDA-supporting studies for AZ-004. Alexza believes these data, along with data from the other efficacy and safety trials conducted with AZ-004, adequately demonstrate the efficacy and safety of AZ-004 for the proposed indication.
(PART FIVE - THE COMPANY FINANCES)
During their most recent Earnings Release and Conference Call on May 11, 2010, ALXA announced that they currently have enough money to fund their planned operations through the second quarter of 2011. If milestones are met under the Biovail Corporation deal, they will have enough money to fund their operations into 2012. As of March 31, 2010, ALXA has 40 million dollars in consolidated cash, cash equivalents and marketable securities. On May 5, 2010, ALXA executed a 15 million dollar loan with Hercules Technology Growth Capital. ALXA is also eligible for up to 90 million dollars in milestones payments from Biovail Laboratories, explained below.
Alexza Pharmaceuticals partnered with Biovail Corporation, Canada's single largest pharmaceutical company, operating internationally in all aspects of pharmaceutical products. In February 2010, Alexza established a collaboration with Biovail Laboratories International SRL, a subsidiary of Biovail Corporation, to develop and commercialize AZ-004 in the U.S. and Canada. Under the terms of the collaboration, Alexza is entitled to receive an upfront cash payment of $40 million, up to $90 million in potential milestone payments contingent on the successful approval of the AZ-004 NDA and other milestones.
(PART SIX - COMPANY EXECUTIVES)
Alexza Pharmaceuticals President and CEO, Thomas B. King, has over 16-years of experience working in the pharmaceutical industry. He has been President and CEO of Alexza Pharmaceuticals since 2003 (being with the company every step of the way for AZ-004 since its IND filing in 2004). Before he joined Alexza Pharmaceuticals, Mr. King served as President, Chief Executive Officer and a member of the board of directors of Cognetix, Inc., a biopharmaceutical development company. In addition, from January 1994 to February 2001, Mr. King held various senior executive positions at Anesta Corporation, a publicly-traded pharmaceutical company, including President and Chief Executive Officer from January 1997 to October 2000, and was a member of the board of directors until it was acquired by Cephalon, Inc., a publicly-traded biopharmaceutical company. Mr. King is a member of the board of directors of Achaogen, Inc., a privately-held biotechnology company.
Alexza Pharmaceuticals CFO August J. Moretti has served as ALXA's Senior Vice President and Chief Financial Officer since February 2005 and as their Secretary since December 2005. From August 2004 to February 2005, Mr. Moretti was their part time Chief Financial Officer. From January 2001 to January 2005, Mr. Moretti served as Chief Financial Officer and General Counsel at Alavita, Inc. (formerly known as SurroMed, Inc.), a biotechnology company. From January 1982 to December 2000, Mr. Moretti was a member of Heller Ehrman White & McAuliffe LLP, an international law firm.
(PART SEVEN - OTHER INFORMATION)
Alexza is able to manufacture 7 million units of AZ-004 a year at their facility in Mountain View, California. ALXA is expected to begin purchasing manufacturing and raw components for AZ-004 in a few months.
Alexza is currently engages in ongoing discussion with multiple parties about selling AZ-004 outside of North America. They are planning to file for AZ-004 for approval in Europe and hope to do by in early 2011.
ALXA is looking to put other product candidates of theirs back into development. They plan to advance at least one product candidate this year.
In additional to ALXA's current 6 product candidates currently with INDs and clinical trials, there are 5 other compounds that haven't been publicly announced that are viable for use of the Staccato System, which Alexza is currently eventuating (along with other compounds).
(PART THREE - AZ-004'S ADVANTAGES AND HOW IT WORKS)
Alexza is developing AZ-004 to potentially offer an acute agitation treatment option that provides a fast onset of effect, that is noninvasive and safer to administer than injections, and that allows patients to be active participants in choosing acceptable treatment options.
AZ-004 is a combination drug and device. It used Alexza's proprietary technology, the Staccato System. The Staccato system vaporizes unformulated drug to form a condensation aerosol that allows rapid systemic drug delivery through deep lung inhalation. The drug is quickly absorbed through the lungs into the bloodstream, providing speed of therapeutic onset that is comparable to intravenous administration, but with greater ease, patient comfort and convenience.
The Staccato System is used to deliver the drug Loxapine into the patient's lungs. This process works due to the heating element contained within the Staccato System inhalers. The heating element is coated with a thin layer of Loxapine. Before use, the patient triggers the heating element, which vaporizes the Loxapine, allowing the patient to inhale it. The Loxapine is then rapidly absorbed through the lungs at a rate typically faster than oral and intravenous mediations. Exactly the same dose with each use of the Staccato device; not seen with other inhalation technology. There are no additives when it comes to the Stacatto device. Everything that goes into the lungs is the pure drug and nothing else (good from a safety perspective).
The device has the potential to change the treatment practices for acute agitation. AZ-004 meets the American Association for Emergency Psychiatry (AAEP) Treatment Guidelines for speed, predictability of onset and patient friendly ease of administration. AZ-004 advantages allows doctors, nurses and caregivers to being able to quickly, comfortably, easily and reliably calm down agitated patients.
Clinical trial data indicate that Alexza’s unique Staccato technology generates consistent distribution of aerosolized drug particles of 1 to 5 microns (the ideal size for absorption in deep lung tissue), providing peak plasma concentrations as quickly as an intravenous injection and rapid onset of therapeutic relief.
In addition to Loxapine, the devices have been tested with over 200 FDA-approved that also has the ability to be used in such a manner with the Staccato System. Currently ALXA is engaging in clinical trials with 5 additional drugs; two of which have completed Phase 2 trials, one currently in Phase 2 trial right now and two more which have finished Phase 1 trials. These 5 additional drugs include treatment for migraine headaches, breakthrough pain, acute pain attacks, and insomnia. Since the filing of its first IND in 2004, Alexza has dosed more than 2,600 patient subjects have been administered about 3,000 times and with the Staccato device in 22 different clinical trials under six different INDs for Staccato-based product candidates.
(PART TWO - SUMMARY OF AZ-004: ITS PURPOSE
AZ-004 targets the 22 billion dollar worldwide antipsychotic market (15 billion dollars in the United States alone). AZ-004 is indicated for the treatment of agitation associated with schizophrenia or bipolar disorder. There are 2.4 million schizophrenia patients and 5.7 million bipolar disorder patients in the United States; agitation is a common and severe symptom of both diseases. AZ-004 has a possible $300 to 500 million dollar market for the bipolar and schizophrenia population in just the United States.
Acute agitation, characterized by unpleasant arousal, tension, irritability and hostility, is one of the most common and severe symptoms of many major psychiatric disorders, including schizophrenia and bipolar disorder. AZ-004 meets and fulfills an unmet medical need. Generally disease treatment is for the disease directly, not the agitation symptoms. Agitation is the continuation and escalation of a disease that has not been focused on until now with AZ-004.
According to the National Institute of Mental Health (NIMH), bipolar disorder affects about 5.7 million American adults while schizophrenia afflicts about 2.4 million people in the United States. Market research among physicians and health-care providers indicates that over 90% of these patients will experience agitation during their lifetime and that about 70% of those who experience agitation will have one to six episodes per year. Market research studies with schizophrenia patient caregivers and bipolar patients indicate these patients currently experience an average of 11 to 12 episodes of acute agitation each year. Of which, Market research indicates that approximately 50% of treated acute agitation episodes are treated in emergency settings, another approximately 35% of the treated agitation episodes suffered by schizophrenic and bipolar patients are treated in an inpatient setting (hospital and long-term residential settings), and approximately 15% are treated in a physician's office.
Agitation episodes are currently treated about 55% of the time with oral antipsychotics and about 45% of the time with intra-muscular, or IM, injections. Oral medications work relatively slowly but are easy to administer, painless and are less threatening to patients. IM injections have a faster onset of action and a higher predictability of drug effect, but because they are invasive, IM injections are usually the treatment option of last-resort. Currently, no non-invasive therapies are available that work faster than 30 minutes to help agitated patients in need of treatment. This is where AZ-004 comes into play. AZ-004 is an easy to administer and painless way to administer relief using a fast method.