October FDA Calendar Stocks: Tricks or Treats?

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 |  Includes: AMGN, ANIP, GSK, GTXI, IMUC, MNK, PRTK
by: Mike Havrilla

Below are six stocks from the BioMedReports.com FDA Calendar with expected catalysts during the month of October, including three pending FDA decisions, updated clinical trial data, and an expected FDA filing to expand the label of an existing product (sNDA).

On 8/13/09, BioSante Pharma (NASDAQ: BPAX) announced positive safety data in its ongoing LibiGel Phase 3 clinical development program, reporting that with over 1,250 women enrolled and almost 825 women-years of exposure in its LibiGel Phase 3 program, there have been no deaths and only five cardiovascular events. This analysis of blinded data indicates a very low cardiovascular event rate has occurred thus far and the studies will continue as planned. BPAX targets submission to the FDA of a new drug application (NDA) by mid-2011.

The Phase 3 Cardiovascular and Breast Cancer Safety Study is a randomized, double-blind, placebo-controlled, multi-center, cardiovascular events and breast cancer study that will enroll between 2,400-3,100 women, exposed to LibiGel or placebo for 12 months. In addition to the Phase 3 safety study, BPAX is conducting two Phase 3 efficacy trials with about 500 surgically menopausal women each for a six-month clinical trial under an FDA approved SPA (special protocol assessment). BPAX expects to issue a safety update on LibiGel by the end of October.

GTx Inc. (NASDAQ: GTXI) has a pending NDA for Acapodene (toremifene) 80 mg with a PDUFA action date of 10/30/09 as part of standard 10-month review cycle. Toremifene 80 mg is an oral selective estrogen receptor modulator which GTXI seeks to market for the prevention of bone fractures in men with prostate cancer on androgen deprivation therapy (ADT). ADT is primary treatment for advanced prostate cancer. In the United States, approximately 700,000 men with prostate cancer are being treated with ADT and an estimated 100,000 initiate ADT each year.

GTXI has already rebounded from its sell-off to below 8 bucks and the current share price in the mid-10s is not compelling enough for me to take on the added risk of holding through the PDUFA date, although there may still be some share price run-up left going into the FDA decision. However, the risks of potential labeling restrictions or other delays by the FDA in addition to pending competition from Amgen’s (NASDAQ: AMGN) denosumab / Prolia (which has a pending decision expected on 19-October) leave me on the sidelines for GTXI.

Amgen has a pending BLA for denosumab (d-mab, proposed brand name Prolia) (injected every 6 months) with a PDUFA action date of 10/19/09 for a possible FDA decision. On 7/27/09, Amgen and GlaxoSmithKline (NYSE: GSK) announced a deal to share commercialization of d-mab for postmenopausal osteoporosis outside the U.S. On 8/13/09, an FDA Advisory Panel unanimously backed the use of d-mab (initially limited to women that have failed other treatments), voting 15-0 on a question that asked if there were a population of postmenopausal women with osteoporosis in which the benefit of treatment with d-mab is likely to outweigh the risks.

The Advisory Panel rejected the use of d-mab to prevent osteoporosis, voting 13-2 against the use of d-mab for women at risk for osteoporosis because of low bone density until long-term safety data is available for the drug. About 10 million Americans are estimated to have osteoporosis while 34 million are at risk due to low bone density. The FDA Panel endorsed use of d-mab for certain prostate cancer patients for treating bone loss from hormone ablation therapy, but rejected use of the drug in women with breast cancer being treated with aromatase inhibitors due to safety concerns. I have no position at this time in Amgen since I prefer to focus on small / micro-cap stocks for more leveraged FDA Calendar trades.

In addition, GTXI announced in mid-September that following a planned safety review, an independent Data Safety Monitoring Board (DSMB) has recommended that the company continue as planned the pivotal Phase 3 clinical trial evaluating toremifene 20 mg for the prevention of prostate cancer in men with high grade prostatic intraepithelial neoplasia (PIN), a precancerous lesion of the prostate. Nearly 1,600 patients with high grade PIN have been enrolled in the three year clinical trial and the primary endpoint is a reduction in prostate cancer incidence. The trial is being conducted under a Special Protocol Assessment with the FDA and the last patient will complete the Phase 3 high-grade PIN clinical trial during 1Q10.

ImmunoCellular Therapeutics (OTC: IMUC.OB) is developing a Dendritic Cell (DC) Based Cancer Antigen Vaccine (ICT107) to Treat Glioblastoma (GBM, the most common / aggressive form of brain cancer). A Phase I GBM trial was initiated in May 2007 with a goal of determining the safety and immune response of patients. The DC vaccine targets six gliomaspecific peptides including targets highly expressed on cancer stem cells. In the study, 19 patients (16 newly diagnosed and 3 recurrent) treated with no serious adverse events and patients received three vaccinations at two weeks apart.

In addition, 43% of newly-diagnosed patients experienced no tumor recurrence with median progression free survival (PFS) of 14.6 months versus just 6.9 months for the standard of care treatment for GBM. Overall PFS in the trial was 12 months while three patients experienced PFS / OS (overall survival) of greater than two years. Initial data was presented earlier this year at ASCO. No grade 3 or 4 toxicities were reported in the Phase 1 trial. The next update on clinical data will be reported at the Congress of Neurological Surgeons on 10/26/09. Potential partnering of ICT107 may occur in 2010 to fund future clinical development as IMUC retains all rights to ICT-121 for in-house clinical development.

On 5/12/09, Questcor Pharma (NASDAQ: QCOR) received notice from the FDA that in order for its supplemental New Drug Application (sNDA) seeking approval to market H.P. Acthar Gel (repository corticotropin injection) for the treatment of infantile spasms (IS) to be considered a Complete Response, QCOR must perform additional statistical analyses relating to data from one study within the filing and submit this data to the Agency. The H.P. Acthar Gel sNDA is a previously submitted application, but on 2/24/09 QCOR announced that the FDA asked them to reformat the sNDA and resubmit before accepting for review to expand use.

QCOR resubmitted the sNDA on 3/17/09, but the FDA requested additional statistical analyses relating to data from one secondary study within the filing. On 9/21/09, QCOR announced its plans to submit a sNDA for H. P. Acthar Gel (repository corticotropin injection or Acthar) for the treatment of IS in October. This notification follows the completion by the Company of additional statistical analyses requested by the FDA. These analyses were conducted on data from one supportive study within the Company's filing.

On 8/2/09, Transcept Pharma (NASDAQ: TSPT) and privately-held Purdue Pharma announced an exclusive deal to commercialize Intermezzo (zolpidem tartrate sublingual tablet) in the U.S. Pending FDA approval, Intermezzo has the potential to be the first prescription sleep aid specifically approved for use in the middle of the night at the time a patient awakens and has difficulty returning to sleep. The PDUFA action date for the Intermezzo NDA is 10/30/09 for an expected FDA decision. The stock price of TSPT has already surged nearly 15X from its 52-week lows of around $1; so although FDA approval appears likely and may cause an additional share price surge, I have chosen not to play this one from a short-term perspective.

Purdue will pay TSPT near-term milestones that include an upfront cash payment of $25 million and an additional payment of up to $30 million based upon the timing of a possible FDA approval of Intermezzo. In addition, TSPT is eligible to receive up to an additional $90 million upon reaching future milestones related to achievement of intellectual property and U.S. net sales targets along with double-digit royalties ranging up to the mid-20% level on U.S. net sales.

The three stocks I own from this group (BPAX, IMUC, QCOR) have the best risk / reward profiles in my opinion for their upcoming catalysts expected during October since they do not carry the added risk of depending on an actual decision by the FDA, which is always subject to risk, delay, labeling restrictions, etc. In addition, IMUC’s CEO, Manish Singh, was bullish on the prospects for updated survival information on ICT-107 at the Rodman conference in September while QCOR is profitable with a strong balance sheet as a play on re-submitting their sNDA and re-starting the review cycle in response to the Agency’s request for conducting additional statistical analyses on the data from one supportive study in the Company’s filing.

In addition, I had the opportunity to conduct a phone interview in late September with BioSante’s President / CEO, Stephen Simes, who stated that the next update for the Phase 3 safety study of LibiGel is expected before the end of October – at which time the Safety Study External Executive Committee will have its first un-blinded look at the data and issue a safety update on the trial. While the data will remain blinded to the Company, Mr. Simes believes that by around mid-2010 partnership talks for LibiGel should begin to gain momentum ahead of final safety data and the anticipated NDA filing in 1H11.

Disclosure: Long BPAX, IMUC.OB, QCOR.

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