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Pharmaceutical companies very often have a run-up leading into drug approval dates. I usually try to buy the stocks three to nine months before the anticipated catalyst date to make profit from the run-up. I screened for companies that have an European Medicines Agency (EMA) or a Medicines and Healthcare products Regulatory Agency (MHRA) decision date set for this year. Here is a look at 5 companies that I found.

1. Sucampo Pharmaceuticals (NASDAQ:SCMP) is a global pharmaceutical company focused on the discovery, development and commercialization of proprietary drugs based on prostones. The therapeutic potential of prostones, which occur naturally in the human body as a result of enzymatic catalysis by 15-PGDH of eicosanoids and docosanoids, was first identified by Ryuji Ueno, M.D., Ph.D., Sucampo's Chairman and CEO. Dr. Ueno founded Sucampo Pharmaceuticals in 1996 with Sachiko Kuno, Ph.D., founding CEO and currently Executive Advisor, International Business Development, and a member of the Board of Directors.

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Upcoming MHRA catalyst

In the United Kingdom, Sucampo awaits a final regulatory decision from the Medicines and Healthcare products Regulatory Agency in the third quarter of 2012 for the short-term use of Amitiza (lubiprostone) in the treatment of chronic idiopathic constipation (CIC).

Financials

The company reported the second-quarter financial results on August 7 with the following highlights:

Revenue$16.7 million
Net loss$0.8 million
Cash$88.6 million

Upcoming other catalysts

Management confirmed on August 7 that it continues to pursue the following 2012 Amitiza-related value drivers:

  • In Japan, the company anticipates a pricing decision later this year, to be followed by a comprehensive primary care and specialist launch in the fourth quarter of 2012 with a partner, Abbott Japan.
  • In the U.K., the company awaits regulatory action on the MAA for the treatment of CIC in the third quarter 2012. In Switzerland, the company expects to conclude pricing negotiations with the authorities for an appropriate reimbursement price for CIC.
  • In the U.K. and Switzerland, the company expects to file MAAs for the OIC indication.

Management also confirmed continuing efforts to achieve these 2012 Rescula-related value drivers:

  • In the U.S., obtaining further improvements in the label to fully reflect current scientific understanding in advance of its launch during the fourth quarter 2012.
  • In the E.U. and Switzerland, filing MAAs for the reduction of elevated intraocular pressure in patients with ocular hypertension or chronic open-angle glaucoma.

My analysis

The stock has a $18.75 price target from the Point and Figure chart. I believe the stock could run to $6-$7 after the Amitiza approval in the U.K. this quarter. If there is no U.K. approval, the stock would likely dip below $4.

2. Savient Pharmaceuticals (SVNT) is a specialty biopharmaceutical company focused on developing and commercializing Krystexxa (pegloticase) for the treatment of chronic gout in adult patients refractory to conventional therapy. Savient has exclusively licensed worldwide rights to the technology related to Krystexxa and its uses from Duke University and Mountain View Pharmaceuticals (MVP). Duke developed the recombinant uricase enzyme and MVP developed the PEGylation technology used in the manufacture of Krystexxa. MVP and Duke have been granted U.S. and foreign patents disclosing and claiming the licensed technology and, in addition, Savient owns or co-owns U.S. and foreign patents and patent applications, which collectively form a broad portfolio of patents covering the composition, manufacture and methods of use and administration of Krystexxa. Savient also manufactures and supplies Oxandrin (oxandrolone tablets, USP) CIII in the U.S.

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Upcoming EMA catalyst

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The company is expecting a EU approval of Krystexxa in the second half of 2012 as stated in the investor presentation dated August 16.

Krystexxa is indicated for the treatment of chronic gout in adult patients refractory to conventional therapy, a condition referred to as Refractory Chronic Gout, or RCG. RCG occurs in patients who have failed to normalize serum uric acid and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose or for whom these drugs are contraindicated.

Financials

The company reported the second-quarter financial results on August 8 with the following highlights:

Revenue$4.6 million
Net loss$16.4 million
Cash$142.2 million

Upcoming other milestones

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My analysis

I believe the stock could best $2 level after Krystexxa EU approval this year. If the drug fails to gain approval, I believe we will see the stock back under $1.

3. Gentium (GENT), located in Como, Italy, is a biopharmaceutical company focused on the development and manufacture of drugs to treat and prevent a variety of diseases and conditions, including vascular diseases related to cancer and cancer treatments. Defibrotide, the company's lead product candidate, is an investigational drug that has been granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) and Orphan Medicinal Product Designation by the European Medicines Agency, both to treat and to prevent VOD, as well as Fast Track Designation by the U.S. FDA to treat VOD.

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Upcoming EMA catalyst

The company announced on June 21 that it has submitted its responses to the Day 180 List of Outstanding Issues (LoOIs) received from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) with respect to the company's Marketing Authorization Application (MAA) for Defibrotide to treat and prevent hepatic veno-occlusive disease (VOD) in adults and children undergoing haematopoietic stem cell transplantation therapy. Gentium expects to receive an opinion from the CHMP regarding the approval of Defibrotide during the third quarter of 2012.

Veno-occlusive disease is a potentially life-threatening condition, which typically occurs as a significant complication of stem cell transplantation. Certain high-dose conditioning regimens used as part of stem cell transplantation can damage the lining cells of hepatic blood vessels and result in VOD, a blockage of the small veins in the liver that leads to liver failure and can result in significant dysfunction in other organs such as the kidneys and lungs (so-called severe VOD). Stem cell transplantation is a frequently used treatment modality following high-dose chemotherapy and radiation therapy for hematologic cancers and other conditions in both adults and children. At present there is no approved agent for the treatment or prevention of VOD in the United States or the European Union.

Financials

The company reported the first-quarter financial results on May 17 with the following highlights:

Revenue$7.5 million
Net loss$1.2 million
Cash$14.2 million

Salvatore Calabrese, SVP & Chief Financial Officer of Gentium commented on May 17:

"We are pleased to report that Defibrotide usage increased by 28% compared to the prior-year period. The company continues to be cash flow positive despite higher expenditures incurred with activities related to expansion of the company's commercial infrastructure and the hiring of contract research organizations and scientific and regulatory consultants to assist and support the FDA and EMA regulatory approval processes."

My analysis

The stock has a $18 price target from the Point and Figure chart. I believe the stock could challenge $15 level after EU approval of Defibrotide later this year. If the drug fails to gain approval, the stock will likely dip below $8.

4. Horizon Pharma (NASDAQ:HZNP) is a biopharmaceutical company that is developing and commercializing innovative medicines to target unmet therapeutic needs in arthritis, pain and inflammatory diseases.

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Upcoming EMA catalyst

The company is expecting decision on Duexis MAA in the fourth quarter of 2012. Duexis, a combination of the NSAID ibuprofen and the histamine H2-receptor antagonist famotidine, is indicated for the relief of signs and symptoms of rheumatoid arthritis and osteoarthritis and to decrease the risk of developing upper gastrointestinal ulcers in patients who are taking ibuprofen for those indications. Duexis is already approved in the U.S.

Financials

The company reported the second-quarter financial results on August 10 with the following highlights:

Revenue$3.8 million
Net loss$22.8 million
Cash$63.5 million

My analysis

The stock has a $18.75 price target from the Point and Figure chart. If Duexis is to gain EU approval, I believe the stock could challenge $8 level. If the drug is not approved, we could dip below $4.

5. Alexza (NASDAQ:ALXA) is a pharmaceutical company focused on the research, development and commercialization of novel, proprietary products for the acute treatment of central nervous system conditions. Alexza's technology, the Staccato system, vaporizes unformulated drug to form a condensation aerosol that, when inhaled, allows for rapid systemic drug delivery through deep lung inhalation. With the Staccato system, a 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.

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Upcoming EMA Catalyst

In May, Alexza and its European corporate partner, Grupo Ferrer, met with the Rapporteur, Co-Rapporteur and EMA, to further understand specifics of the major objections raised in the Day 120 List of Questions, which were received by the company in March from the Committee for Medicinal Products for Human Use. In July, Alexza submitted its responses regarding the Adasuve Marketing Authorization Application to the European Medicines Agency. The responses submitted in July are intended to address the questions outlined in the Day 120 List of Questions. According to the published EMA timetables, Alexza expects to receive the Day 180 List of Outstanding Issues for the Adasuve MAA in late September 2012, and Alexza currently projects that it will receive the Day 210 CHMP Opinion for the Adasuve MAA in December 2012.

Adasuve (Staccato loxapine) is Alexza's lead program, which is being developed for the acute treatment of agitation associated with schizophrenia or bipolar I disorder in adults. Alexza completed and announced positive results from both of its Phase 3 clinical trials and initially submitted the Adasuve NDA in December 2009. In October 2010, the company received a CRL from the FDA regarding the application. The company completed an end-of-review meeting with the FDA in December 2010, a REMS guidance meeting with the FDA in April 2011, and the Adasuve NDA was resubmitted in August 2011. In December 2011, the Adasuve NDA was the subject of an advisory committee meeting. In January 2012, Alexza received notice of a 90-day extension of the PDUFA goal date, from February 4, 2012 to May 4, 2012. In May 2012, Alexza received a second CRL from the FDA regarding the Adasuve NDA and on June 22, 2012, the company resubmitted the Adasuve NDA. The resubmitted Adasuve NDA has a PDUFA goal date of December 21, 2012.

Financials

The company reported the second-quarter financial results on August 8 with the following highlights:

Revenue$0.7 million
Net loss$7.0 million
Cash$18.5 million

My analysis

If the company can gain both the U.S. and the EU approvals for Adasuve, I believe the stock could trade above $10. Alexza has already received a CRL from the FDA twice regarding Adasuve. I would give a 30% chance that Alexza receives a third CRL.

Source: 5 Pharmaceutical Companies With EMA Or MHRA Catalysts This Year