Pending FDA Decisions: 20 Extreme Trades

by: Mike Havrilla

Below are 20 extreme trades among companies with market caps under $500 million from the FDA Calendar (database includes 225 entries as of 6/21/09) which have pending FDA decisions for either new drug products or biological agents that may have a major impact on each of the underlying stock prices. A high risk/reward trading approach to consider for speculative traders is investing a small amount of money that you can afford to lose in a basket of stocks included in Extreme FDA and Clinical Trial Calendar Trades, which are highlighted in periodic articles at Another approach is to buy a basket of stocks well ahead of their expected binary events before the trading crowd arrives and causes an increase in the share price and trading volume.

BioDelivery Sciences (NASDAQ:BDSI): On 6/15/09, BDSI announced that the Company has reached agreement with the FDA on all aspects of its pending NDA for Onsolis (fentanyl buccal soluble film), including the REMS. Although the FDA did not meet its PDUFA action date, the FDA will not be issuing a review extension letter as their action on the NDA is forthcoming. Recent FDA approvals for Entereg, Cimzia, and Nplate provide examples of missed PDUFA action dates for drugs which also required REMS. In these cases, final FDA decisions occurred in a range of 10-30 days after the original PDUFA date, which translates into a possible Onsolis approval announcement at any time from 6/22/09 to 7/13/09.

Acura Pharma (NASDAQ:ACUR): Along with King Pharma (NYSE:KG), ACUR has a pending Acurox (oxycodone + niacin) NDA (priority review) with a PDUFA action date of 6/30/09 for a possible FDA decision. Acurox is an immediate release opioid analgesic, has a proposed indication for relief of moderate-to-severe pain and is designed to deter common methods of misuse and abuse.

Spectrum Pharma (NASDAQ:SPPI): Zevalin (Ibritumomab Tiuxetan) (a radio-immunotherapeutic that specifically targets and kills cancer cells) sBLA to expand FDA-approved label as a first-line consolidation therapy (following a response to first-line treatments) for non-Hodgkin's lymphoma (NHL). On 2/23/09, a three-month PDUFA action date delay to 7/2/09 was announced as SPPI submitted additional data to the FDA, which was classified as a major amendment by the agency.

The FDA accepted Spectrum Pharma's supplemental New Drug Application (sNDA) for FUSILEV (levoleucovorin) for Injection on 3/26/09 in combination with 5-FU containing regimens in advanced metastatic colorectal cancer. The user fee goal (PDUFA) date for the sNDA is October 8, 2009. The FDA approved FUSILEV on March 7, 2008 for rescue after high-dose methotrexate therapy in osteosarcoma. FUSILEV is also indicated to diminish the toxicity and counteract the effects of impaired methotrexate elimination and of inadvertent overdosage of folic acid antagonists. In December 2008, FUSILEV was listed in the National Comprehensive Cancer Network (NCCN) Drugs and Biologic Compendium™ to be used in combination with high-dose methotrexate for the treatment of bone cancer (osteosarcoma and dedifferentiated chrondrosarcoma), and the NCCN Clinical Practice Guidelines in Oncology in Colon Cancer and Rectal Cancer were updated to reflect that FUSILEV is available in the United States.

Labopharm (NASDAQ:DDSS) has a pending NDA for a rapid-onset formulation of trazodone (DDS-04A) filed through the 505(b)(2) pathway as a new formulation of an existing drug for the proposed treatment of major depressive disorder with an estimated PDUFA action date of 7/18/09 for a possible FDA decision.

Advanced Life Sciences (NASDAQ:ADLS): Cethromycin NDA (a once-daily antibiotic for the treatment of community acquired pneumonia - CAP) with an expected PDUFA decision date of 7/31/09. On 6/2/09, ADLS announced that the FDA's Anti-Infective Drugs Advisory Committee voted in the majority that Restanza (cethromycin) demonstrated safety for the outpatient treatment of adults with mild-to-moderate CAP (11 positive, 3 negative, 1 abstaining). However, the committee voted that Restanza did not demonstrate efficacy in the treatment of CAP (3 positive, 11 negative, 1 abstaining).

Nuvo Research (OTCQX:NRIFF) [TSE:NRI] has a pending NDA re-submission for Pennsaid with a PDUFA action date of 8/5/09 for a possible FDA decision. On 6/16/09, Nuvo announced a deal with Covidien (NYSE:COV) granting it exclusive rights to market and sell Pennsaid, and its follow-on product, Pennsaid Plus, in the U.S. Pennsaid and Pennsaid Plus are Nuvo's topical non-steroidal anti-inflammatory drug (NSAID) candidates that deliver diclofenac through the skin directly to the site of pain.

Nuvo receives an up-front, non-refundable payment of US$10 million and is also eligible to receive a US$15 million milestone payment on Pennsaid's approval by the FDA, which will increase to US$20 million if certain labeling criteria are agreed to by the FDA. Nuvo will receive royalties on net U.S. sales of Pennsaid and Pennsaid Plus and is also eligible to receive additional escalating sales milestone payments for the products totaling up to US$100 million. COV assumes responsibility for all future development activities and expenses for Pennsaid Plus, including two Phase 3 clinical trials that are expected to begin in 2010.

NeurogesX (NASDAQ:NGSX): On 6/10/09, NGSX announced that the FDA agreed to its proposed study to evaluate Qutenza in patients with post-herpetic neuralgia (PHN) following pretreatment with an FDA-approved topical anesthetic. As part of its ongoing NDA review, the FDA wants to determine whether pretreatment with an FDA-approved topical anesthetic would provide similar tolerability to the topical agent used as a pretreatment in the clinical development program.The NDA is supported by clinical studies of Qutenza which was applied after a 60-minute pre-treatment with an over-the-counter (OTC) topical anesthetic.

The study protocol involves enrolling about 20 patients with PHN to receive the Qutenza patch application after a 60-minute pretreatment with an FDA-approved topical anesthetic (2.5% lidocaine / 2.5% prilocaine).

Based on the expected short-term nature of the study, NGSX believes that it could potentially complete enrollment, analyze the data, and submit an NDA amendment prior to the assigned PDUFA date. Submission of this amendment during the review cycle may result in an extension of the PDUFA date (likely to be a minor three-month delay from the current PDUFA action date of 8/16/09). NGSX received approval on 5/21/09 to market Qutenza in the EU. Qutenza is a skin patch that is designed to locally deliver a high-concentration (8%) of the active substance capsaicin to provide sustained relief from peripheral nerve pain.

Salix Pharma (NASDAQ:SLXP): The FDA accepted the complete response for Metozolv (metoclopramide) ODT (orally disintegrating tablets) and granted a PDUFA action date of 9/11/09 for a possible decision by the Agency. SLXP is seeking approval for (1) the treatment of short–term therapy (4–12 weeks) for adults with symptomatic, documented gastroesophageal reflux (GERD or heartburn) who fail to respond to conventional therapy and for (2) the relief of symptoms associated with acute and recurrent diabetic gastroparesis (delayed emptying of the stomach).

In early 2009, the FDA issued a class-wide requirement for all manufacturers of metoclopramide in the U.S. to provide a risk evaluation and mitigation strategy (REMS) for their products concerning a well known adverse event of the drug known as tardive dyskinesia. The FDA decided to elevate the warnings surrounding tardive dyskinesia (involuntary, repetitive movements) to a boxed warning on the package insert that requires the issuance of a medication guide to patients and healthcare professionals to better educate as to the proper use of metoclopramide.

ISTA Pharma (NASDAQ:ISTA): On 6/1/09, ISTA announced the Dermatologic and Ophthalmic Drugs Advisory Committee will review ISTA's New Drug Application (NDA) for Bepreve (bepotastine ophthalmic solution) on June 26, 2009. On 11/13/08, ISTA announced it had filed its NDA with the FDA for Bepreve as an eye drop treatment for ocular itching associated with allergic conjunctivitis.

As bepotastine is a new chemical entity in the U.S., ISTA expected an Advisory Committee would be convened for Bepreve. ISTA continues to expect a standard review of ten months and has been given a PDUFA decision date of 9/12/09 for the FDA to complete its review of the pending NDA.

Transcept Pharma (NASDAQ:TSPT): On 6/11/09, TSPT announced that the FDA has informed the Company that it should expect to receive formal notice of a three month extension of the review period for the new drug application (NDA) for Intermezzo (zolpidem tartrate sublingual tablet). The Intermezzo NDA had been assigned a PDUFA action date of 7/30/09.

Under this revised timeline, TSPT now anticipates action from the FDA on the NDA on or before 10/31/09. In the normal course of the Intermezzo NDA review, the FDA previously requested additional information regarding middle of the night dosing instructions. As both the request and the TSPT response occurred late in the review cycle, the FDA has informed the company that it will extend the NDA review cycle by three months to consider the new information.

Dyax Corp. (NASDAQ:DYAX): On 6/8/09, DYAX announced today that the FDA accepted the Company’s submission in response to the FDA’s March 2009 Complete Response Letter (CRL), which outlined requirements for approval of DX-88 for the treatment of acute attacks of hereditary angioedema (HAE).

In connection with the acceptance, the FDA assigned Dyax’s BLA a new PDUFA action date of 12/1/09, which represents a six-month, Class 2 Review. In the CRL received 3/25/09, the FDA requested submission of a Risk Evaluation and Mitigation Strategy (REMS) and additional information with respect to the chemistry, manufacturing and controls (CMC) section of the BLA. Dyax believes these issues are fully addressed in its reply, which was submitted 6/1/09.

Electro-Optical Sciences (NASDAQ:MELA): On 6/4/09, MELA announced the submission to the FDA of its Premarket Approval (PMA) application for MelaFind, a non-invasive, point-of-care instrument to assist in the early diagnosis of melanoma, the leading cause of death from skin cancer. Positive top line data from the MelaFind pivotal study, the largest prospective clinical study ever conducted in melanoma detection, were announced in February 2009 and subsequently presented at several major international dermatology meetings in March and May.

The Company's final analysis of the data demonstrated that for all subgroups analyzed, the sensitivity of MelaFind was greater than 95% (lower confidence bound) and MelaFind specificity was statistically significantly higher than that of study clinicians. As announced previously, the FDA has granted expedited review (six months) for the MelaFind PMA, resulting in an estimated decision date deadline of 12/4/09.

Somaxon Pharma (NASDAQ:SOMX): On 6/4/09, SOMX announced that it has resubmitted its New Drug Application (NDA) to the FDA for Silenor (doxepin) for the treatment of insomnia in response to a 2/25/09 Complete Response Letter (CRL) and following a 4/6/09 meeting with the FDA. The resubmission includes additional statistical analyses of the Company’s clinical data relating to the durability of subjective sleep maintenance efficacy. It also includes the results of the Company’s completed clinical trial of doxepin that evaluated the potential for electrocardiogram (ECG) effects.

The results of that clinical trial demonstrated that Silenor had no effect on QT interval prolongation when administered at 6 mg or under exaggerated exposure conditions of 50 mg. The FDA has indicated that the review cycle for the resubmission will be six months (a Class 2 Review designation) for an estimated decision date of 12/4/09 on the Silenor NDA resubmission.

Santarus Pharma (NASDAQ:SNTS): On 4/6/09, SNTS filed NDA for a new tablet formulation to add to its ZEGERID® family of branded prescription pharmaceutical products. Pursuant to Prescription Drug User Fee Act (PDUFA) guidelines, Santarus expects the FDA will complete its review or otherwise respond to the NDA by December 4, 2009. The NDA was submitted under section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act. In connection with the FDA’s acceptance for filing of the NDA for a new tablet formulation, Santarus is providing notice to the NDA holder for Prilosec® (omeprazole) delayed-release capsules and related patent holders that the new tablet formulation does not infringe the patents listed in the Orange Book for Prilosec or that those patents are invalid.

Vion Pharma (OTC:VION): Onrigin (laromustine) Injection (formerly known as Cloretazine or VNP40101M) NDA requesting approval as a single agent for remission induction treatment for patients age 60 and older with de novo poor-risk acute myeloid leukemia (AML). The NDA will receive a standard (10-month) review by the FDA with a PDUFA decision date deadline of 12/12/09.

Pharmaxis (NASDAQ:PXSL): Pharmaxis has a pending NDA for Aridol as a mannitol bronchial challenge test which is designed to help in the correct diagnosis and assessment of asthma. There is currently no FDA-approved dry powder bronchial challenge tests available in the U.S. and Aridol is the first test of this kind that comes in a ready-to-use format. Pharmaxis is seeking approval for Aridol for "the assessment of bronchial hyperresponsiveness to aid in the diagnosis of patients with symptoms of or suggestive of asthma." 12/27/09 is the PDUFA action date for an expected FDA decision.

IntelGenx (OTC:IGXT): On 4/7/09, IGXT and Cary Pharma (privately held) announced that a New Drug Application (NDA) was submitted to the FDA for their antidepressant (CPI-300), which represents a new strength of a leading antidepressant (name not disclosed) that will provide a more convenient dosing option to patients with major depressive disorder ("MDD"). IGXT and Cary Pharma entered an agreement in November 2007 to jointly develop and commercialize CPI-300 using IntelGenx's proprietary oral delivery technology. CPI-300 was formulated using IntelGenx's proprietary controlled release technology. It contains a new strength of a widely prescribed antidepressant. CPI-300 was developed to broaden the dosing options for physicians while providing a more convenient dosing option for patients requiring a higher strength of the products currently available.

Under the terms of the Collaborative Agreement, IGXT raised $2 million in March 2008 to fund completion of the product development and Cary Pharma acted as the applicant for the submission of the NDA. Upon commercialization of the product, IGXT and Cary Pharma would share profits. The estimated PDUFA action date for a standard, 10-month review is 2/7/2010 for a possible FDA decision on the CPI-300 NDA.

Cadence Pharma (NASDAQ:CADX): On 5/14/09, CADX announced the 505(b)(2) NDA submission for Acetavance (the Company's proprietary intravenous formulation of acetaminophen) for the treatment of acute pain and fever in adults and children. If approved, Acetavance would become the first new intravenous analgesic approved in the U.S. in more than 20 years and the only approved intravenous agent for the treatment of fever in this country.

The NDA filing includes data from one pivotal clinical trial for the treatment of acute pain in patients following orthopedic surger, one pivotal clinical trial for the treatment of endotoxin-induced fever, and is also supported by data from a total of nine placebo-controlled clinical trials, four active-controlled clinical trials, and seven other safety or pharmacokinetic clinical trials. The submission includes safety data from over 1,400 patients who received Acetavance in clinical trials, including 350 children, and data from safety reports that collectively represent more than 53 million patient exposures to intravenous acetaminophen in countries outside the United States. 3/14/10 is the PDUFA decision date for a standard, 10-month review by the FDA.

A.P. Pharma (NASDAQ:APPA): On 5/18/09, APPA announced that it has submitted a NDA for its lead product (APF530) to the FDA for the prevention of chemotherapy-induced nausea and vomiting. APF530 is a long-acting formulation of granisetron that utilizes the Company’s proprietary Biochronomer drug delivery system. The NDA was submitted under section 505(b)(2) of the Federal Food, Drug and Cosmetic Act, whereby the Company can rely upon the FDA’s prior safety and efficacy findings for APF530's active ingredient, granisetron (which is already approved and marketed as Kytril). The FDA is expected to determine whether to accept the NDA for filing within 60 days, and to notify the Company of its determination within 14 days thereafter.

If the NDA is accepted for filing, under the Prescription Drug User Fee Act (PDUFA) guidelines, it is expected that the FDA will complete its review and provide an action letter with respect to the NDA within 10 months following NDA submission.

Human Genome Sciences (NASDAQ:HGSI): On 5/21/09, HGSI announced that it submitted a Biologics License Application (BLA) to the FDA for its human monoclonal antibody drug ABthrax (raxibacumab) for the treatment of inhalation anthrax. Raxibacumab is a first-in-class treatment for anthrax, and is being developed under a contract entered into in 2006 with the Biomedical Advanced Research and Development Authority of the Office of the Assistant Secretary for Preparedness and Response, U.S. Department of Health and Human Services (HHS).

In the first quarter of 2009, under the BARDA contract, HGS achieved its first product sales by initiating the delivery of 20,000 doses of raxibacumab to the U.S. Strategic National Stockpile for emergency use in the treatment of inhalation anthrax. HGS recognized $153.8 million in raxibacumab revenue in the first quarter, including $127.8 million in product sales.

The Company has now completed delivery and expects to recognize at least $8.0 million in additional raxibacumab revenue in the second quarter of 2009. The BLA is also being filed under the BARDA contract, and HGS will receive $10 million from the U.S. Government upon FDA licensure of raxibacumab. 3/21/10 is the estimated PDUFA decision date for a standard, 10-month review by the FDA.

Disclosure: Long BDSI.