October has been called the Super Bowl or World Cup of biotech approvals in the investing world. Whatever you want to call it, investors will be watching closely as many companies will have their drugs reviewed by the FDA for approval. This is often-times a turning point in a small biotech company's history in regards to whether or not they get approved or denied. In a previous article I focused on the recent financing and how that will fuel the future growth of Alexza Pharmaceuticals (ALXA). This piece will focus on key findings from a recent conference call that makes a strong case for the growth of Alexa heading into their Oct 11th PDUFA date for AZ-004 (Staccato Loxapine).
There are often many questions that can come up prior to an approval date regarding efficacy or safety. These questions are most often addressed by an Advisory Panel of the FDA. An advisory panel is becoming more common for companies as these are often required before they await approval from the FDA. The advisory panel usually addresses potential issues regarding safety or efficacy. There have already been at least a half dozen companies before the end of 2010 that have gone before or will have their product undergo the scrutiny of an advisory panel prior to the FDA for approval of their drug or device. Vivus (VVUS) went before an FDA panel on July 15th for their drug Qnexa for obesity and it was voted 10-6 not approvable. Mela (MELA) had their advisory panel meeting rescheduled after it was originally scheduled for August 26th for their medical device for melanoma called the MELAFIND. The FDA had pushed the MELA meeting back to November as they needed time to assemble their advisory panel. Questcor (QCOR) was notified on June 11th that they had 2 months to prepare for an FDA meeting for Sept 11th where the FDA would decide on their drug, Athcar, for the treatment of infantile spasms. This FDA meeting was pushed back because the FDA stated they needed more time to review information regarding labeling and potential post-approval commitments that they solicited from Questcor. This is also after the May 6th Advisory Committee to the Division of Peripheral & Central Nervous System Drugs of the FDA voted in support of Athcar for approval. Arena Pharmaceuticals (ARNA) has an Advisory Panel Meeting for September 16th to discuss Locaserin for the treatment of obesity and their PDUFA date is Oct 22nd. Jazz (JAZZ) has an advisory committee meeting scheduled with the FDA on August 20th to discuss their drug Xyrem in fibromyalgia. Their scheduled PDUFA date is Oct. 11th. What does this Oct 11th date have to do with Alexza Pharmaceuticals? The importance is that date is the PDUFA date for Alexa's AZ-004 (Staccato Loxapine) where they will seek an indication for the rapid treatment of agitation associated with schizophrenia or bipolar disorder.
As an investor can clearly see, getting past the FDA hurdle of an Advisory Panel Meeting before your scheduled PDUFA date is a challenge. With less than 60 days from their PDUFA date, Alexa has cleared that hurdle. There were key questions and potential concerns answered in a recent Merrill Lynch/Bank of America investor conference (Aug 13th) that were cleared up for investors in regards to AZ-004 (Staccato Loxapine). These questions were important to address as we move closer to the PDUFA date of October 11th.
The first question focused on if there would be an advisory panel meeting for AZ-004 because this was a new drug/device combo. This would be a combination of the Staccato delivery system being used to deliver an older but effective drug in Loxapine.
In their Pre-NDA meeting, the FDA staff sent a clear signal to Alexa that there would not be an advisory panel meeting for AZ-004. There has been no indication there will be a meeting, and a meeting usually requires advance notice, like JAZZ had for their advisory panel meeting for Aug 20th. The FDA reserves the right to change their mind but it is unlikely there will be a change given the amount of time it takes to convene a panel and how far ahead you have to schedule to invite panel members. A panel is usually convened when there is a “tight walk” between efficacy and& safety of a clinical trial or there is a clinical endpoint that needs to be discussed or clarified. That has not been the case with AZ-004.
Alexza had an extensive end of Phase 2 meeting with the FDA regarding AZ-004 (Staccato Loxapine) in 2009 and has had about 25 meetings with the FDA regarding the different developmental phases of their programs, according to their CFO. These meeting included pre-IND meetings, end of Phase II mtngs, and pre-NDA meetings for various programs.
The second question was with AZ-004 being the first CNS drug delivered to the deep lung, if there was any skepticism with the FDA around that issue and what division of the FDA had been involved. The FDA pulmonary division has been involved and Alexza has worked through the division of combination products, as they had done early on from the start. The division made a decision early on that Alexza's products were being reviewed as a "drug" and not a combo of drug/device so all of their applications would be filed as NDAs. Their application is reviewed by the toxicology and pulmonary groups in the division of psychiatry. That is headed up by Dr. Tom Laughren.
The third question focused on whether Alexza prepared a REMS for AZ-004. Alexza did not submit a REMS. It was discussed that it had not because the Staccato is a single-dose device and Loxapine is not a drug of abuse or a scheduled drug. Also, the side effect profile is very benign, with a very small incidence of insomnolence in the clinical trials but not much else. If the FDA approves AZ-004 and decides to restrict it to the inpatient setting, that will provide even additional support from the FDA in terms of overall safety, as all patients would be medically supervised when using AZ-004. All of the clinical work with AZ-004 has been in a medically supervised environment, be it the physician's office, clinic, or emergency room. The key is that the FDA has options and does not have their back up against the wall with AZ-004. I will focus on the clinical data as well as delivery system in future articles.
Alexza is definitely gearing up for approval of AZ-004 with their partner Biovail (BVF) who has commercial responsibility for the U.S. and Canada. Alexza was attracted to Biovail because of their commitment to CNS and the psychiatry space in medicine. Wellbutrin did 180 million in sales for Biovail in 2009, which was its largest sales producer, and they believe that AZ-004 will be a major contributor to their growth story in 2010. Many investors believe that Alexxa will be a solid growth stock in the months to come.
Disclosure: Long ALXA