10 Trades on FDA Binary Events

by: Mike Havrilla

Below is a selection of 10 companies with pending FDA decisions that are expected through the end of October, with a focus on small-cap companies with market caps of approximately $1 billion or less:

1) Savient Pharma (OTC:SVNT) $14.64 $990M

FDA Decision (BLA Class 2 Re-submission)

Krystexxa (pegloticase) (PEGylated uricase enzyme)

CRL issued by FDA 7/31/09, seeking approval as chronic gout treatment in patients that do not respond to conventional treatments, FDA accepted BLA resubmission as Class 2 response (six-month review) with PDUFA action goal date 9/14/10, submitted six-month stability data to FDA on 7/28/10 and plans to sell company post-approval.

2) Alexza Pharma (NASDAQ:ALXA) $3.16 $167M

FDA Decision (NDA) Staccato loxapine (AZ-004)

AZ-004 is an inhalation product candidate being developed for the rapid treatment of agitation in patients with schizophrenia or bipolar disorder, partnered with Biovail (BVF) in the U.S. and Canada, PDUFA action goal date of 10/11/10 under standard, 10-month review.

3) Alkermes (NASDAQ:ALKS) $13.98 $1.3B

FDA Advisory Panel (9/16/10), FDA Decision (sNDA) Priority Review (10/12/10)

VIVITROL (naltrexone extended release, once-monthly injectable suspension)

On 5/25/10, announced FDA accepted sNDA submission with a priority review (six-month) designation and PDUFA action goal date of 10/12/10, currently approved for the treatment of alcohol dependence and seeking approval for opioid dependence, FDA Advisory Panel scheduled for 9/16/10 to review sNDA.

4) Amylin Pharma (AMLN) $21.90 $3.1B

FDA Decision (NDA) Class 2 Resubmission

BYDUREON (exenatide for extended-release injectable suspension) (once-weekly administration)

ALKS due to receive 8% royalties on first 40M units sold per year, then 5.5% royalties thereafter for its drug delivery technology

CRL reply submitted on 4/22/10 and accepted 5/6/10 for Class 2 (six-month) review and PDUFA action goal date of 10/22/10, addresses product labeling, clarification of manufacturing, and Risk Evaluation and Mitigation Strategy (REMS).

5) Arena Pharma (NASDAQ:ARNA) $7.01 $787M

9/16/10 FDA Advisory Panel Meeting (9/16/10) FDA Decision (NDA) (10/22/10)

Lorcaserin (a selective serotonin 2C receptor agonist) Pending FDA decision for experimental weight loss drug, Endocrinologic and Metabolic Drugs FDA Advisory meeting scheduled to review NDA on 9/16/10, PDUFA action goal date of 10/22/10 under a standard, 10-month review period, exclusive US mktg deal with Eisai (OTCPK:ESALY) on 7/1/10 incldg $50M upfront.

6) Vivus (NASDAQ:VVUS) $6.31 $512M

Clinical Trial (Two-Year Data, SEQUEL study) (3Q10), FDA Decision (NDA) (10/28/10), MAA/EMA (Europe) New Filing

QNEXA (phentermine plus topiramate) (once-daily combo weight loss / obesity drug candidate)

On 7/15/10, Endocrinologic and Metabolic Drugs FDA Advisory Panel voted 10-6 against whether the overall risk-benefit of QNEXA is favorable to support approval, PDUFA action goal date is 10/28/10 under a standard 10-month review, SEQUELextension study results (subset of patients from 56-week pivotal CONQUER study) expected 3Q10 to provide long-term (two-year) safety / efficacy data and support MAA filing in Europe.

7) Jazz Pharma (NASDAQ:JAZZ) $9.86 $383M

FDA Decision (NDA) for JZP-6 (sodium oxybate oral solution)

On 8/20/10, Arthritis & Drug Safety / Risk FDA Advisory Panel voted 20 to 2 against recommending approval for the treatment of fibromyalgia, PDUFA action goal date of 10/11/10 under a standard 10-month review period, currently marketed to treat narcolepsy under a REMS / limited distribution program.

8) ISTA Pharma (ISTA) $3.14 $105M

FDA Decision (sNDA) XiDay (bromfenac) once-daily eye drops

Xibrom is currently marketed as an eye drop used twice-daily beginning 24 hours after cataract surgery, this sNDA filing is to request change to product label for once-daily use following cataract surgery as XiDay, expected FDA decision by 10/16/10 under standard 10-month review period.

9) Avanir Pharma (NASDAQ:AVNR) $2.77 $260M

FDA Decision (NDA) Class 2 Resubmission

ZENVIA (dextro-methorphan + quinidine)

Seeking approval for treatment of patients with neurological condition known as pseudobulbar affect (PBA), announced 5/17/10 that FDA accepted NDA resubmission with a Class 2 (six-month) review period and new PDUFA action goal date of 10/30/10.

10) Biodel (BIOD) $3.99 $105M

FDA Decision (NDA) for LINJETA (new proposed trade name) (rapid-acting insulin formulation)

On 8/24/10, SEC 8K filing indicated AMRI contract manufacturing site (one of two sites that made product vials for NDA) received FDA warning letter, BIOD plans to commercialize disposable pen version and expects to submit as sNDA early 2011 with different manufacturer (Wockhardt), PDUFA action goal date of 10/30/10.

Disclosure: Long ALKS