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Pharmaceutical companies with a set Phase III data release date very often have a run-up leading into the release date. I usually try to buy the stocks 3-9 months before the catalyst date to make profit from the run-up. I screened for companies that have a Phase III data release date set during the next 3-9 months. Here is a look at 5 companies that I found.

1. BioMarin (NASDAQ:BMRN) develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan (sapropterin dihydrochloride) Tablets, for phenylketonuria [PKU], developed in partnership with Merck Serono (NYSE:MRK), a division of Merck KGaA of Darmstadt, Germany; and Firdapse(amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome [LEMS]. Product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA, amifampridine phosphate (3,4-diaminopyridine phosphate), which is currently in Phase III clinical development for the treatment of LEMS in the U.S., PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase [PARP] inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, and BMN-111, a modified C-nutriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia.

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Upcoming Phase III catalysts

Hank Fuchs, Executive Vice President & Chief Medical Officer, commented during the second quarter earnings call:

Starting with the GALNS MPS IVA, the last patient in the Phase III trial is due to complete in Q3 and we are on track to report top line results in Q4 and to potentially file the first market authorization application in Q1 2013. GALNS is our absolute highest priority as a company and we have allotted every necessary resource to the timely analysis of data, release of results and the potential filing of market authorization applications.

BMN-110: GALNS for MPS IVA (Morquio Syndrome)

Morquio syndrome is an inherited, autosomal recessive disease caused by a deficiency of a particular lysosomal enzyme, N-acetylgalactosamine-6 sulfatase [GALNS]. Deficiency of the enzyme results in excessive lysosomal storage of keratan sulfate in many tissues and organs. This accumulation causes systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the thorax impairs respiratory function, and malformation of neck vertebrae and ligament weakness causes cervical spinal instability and, potentially, cord compression. Other symptoms may include hearing loss, corneal clouding, and heart valve disease. Morquio A syndrome is estimated to occur in 1 in 200,000 to 300,000 live births.

BioMarin's proposed treatment for Morquio A syndrome is enzyme replacement of GALNS, which is expected to result in clearance of keratan sulfate from the lysosome. This should halt the progression of the disease and may ameliorate some symptoms.

Financials

The company reported the second-quarter financial results on August 1 with the following highlights:

Revenue$124.0 million
GAAP Net Loss$32.0 million
Cash$524.6 million

Outlook

2012 Guidance

Revenue$475 million to $510 million
Net Cash Usage$40 million to $50 million
Cash Balance (End Of Year)$475 million to $485 million

Anticipated Upcoming Milestones

  • 3Q 2012:Results for Phase II trial for PEG-PAL for PKU
  • 3Q 2012:Results for Phase I trial for BMN-111 for achondroplasia in healthy volunteers
  • 4Q 2012:Results for Phase III trial for GALNS for MPS IVA
  • 4Q 2012:Results for Phase I/II trial for BMN-673 for solid tumors
  • 1Q 2013:Market authorization application filing for GALNS for MPS IVA
  • 1Q 2013:Results for Phase I/II trial for BMN-701 for Pompe disease
  • 1H 2013:IND filing for BMN-190 for LINCL (Batten disease)
  • 2Q 2013:Potential initiation of Phase III trial for PEG-PAL for PKU
  • 2Q 2013:Results for PKU-016 Kuvan neurocognitive study
  • 2H 2013:Potential initiation of Phase III trial for BMN-673 for solid tumors
  • 4Q 2013: Potential FDA approval of GALNS for MPS IVA

My analysis

The stock has a $54 price target from the Point and Figure chart. I believe we could reach $50 after positive Phase III results for GALNS later this year and $60 after FDA approval of GALNS in the fourth quarter 2013. The stock has seen steady insider selling this year.

2. Æterna Zentaris (NASDAQ:AEZS) is an oncology and endocrinology drug development company currently investigating treatments for various unmet medical needs. The company's pipeline encompasses compounds at all stages of development, from drug discovery through to marketed products.

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Upcoming Phase III catalysts

Current Phase 3 trial in multiple myeloma with perifosine in combination with bortezomib (Velcade) and dexamethasone, under SPA reached with the FDA. The FDA also granted periosine orphan-drug designation in September 2009 as well as Fast Track designation in December 2009. Furthermore, on March 1, 2010, perifosine received a positive opinion for orphan medicinal product designation in this same indication from the Committee for orphan medicinal products of European Medicines Agency which also issued positive Scientific Advice for perifosine in multiple myeloma. Results from an interim analysis for this trial are expected in Q1-2013.

Financials

The company reported the second-quarter financial results on August 14 with the following highlights:

Revenue$7.5 million
Net income$4.5 million
Cash$39.8 million

The average cash burn rate has been $2.17 million per month.

Upcoming (other) milestones

AEZS-108

  • SPA filing Phase 3 in endometrial cancer
  • Initiate triple-negative breast cancer Phase 2 study
  • Update on ongoing Phase 2 studies in castration- and taxane-resistant prostate cancer and refractory bladder cancer
  • Update on ongoing companion diagnostic program

AEZS-130

  • Fast-track and rolling submission request to FDA
  • File NDA as diagnostic test for AGHD in the U.S.
  • Update on Phase 2A study in cancer-induced cachexia

AEZS-120

  • File CTA for Phase 1 trial in prostate cancer

My analysis

I believe the stock could have a run-up leading into the interim Phase III data due in Q1-2013. If the data is positive we could reach $0.80 - $1 levels.

3. NewLink Genetics Corporation (NASDAQ:NLNK) is a late-stage, biopharmaceutical company focused on discovering, developing, and commercializing novel immunotherapeutic products to improve cancer treatment options for patients and physicians. The company's portfolio includes both "off-the-shelf" biologic and small-molecule immunotherapy product candidates intended to address large, unmet oncology indications, including treatment of pancreatic cancer, non-small cell lung cancer [NSCLC], and melanoma. The company believes its technologies may also be useful in the field of infectious disease.

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Upcoming Phase III catalysts

Dr. Charles Link, Chairman and Chief Executive Officer of NewLink commented on August 14:

Data from the Phase 2 study of our HyperAcute pancreatic cancer immunotherapy was successfully presented at two major conferences and continues to support our Phase 3 study design. We are well past the halfway point in our pivotal trial in pancreatic cancer and we expect to reach the triggering point for our first interm analysis in the first quarter of 2013 and to complete patient enrollment in 2013.

Financials

The company reported the second-quarter financial results on August 14 with the following highlights:

Revenue$0.6 million
Net loss$6.3 million
Cash$31.1 million

NewLink expects to end 2012 with about $20 million in cash, cash equivalents and marketable securities.

My analysis

I believe the stock could best the $20 mark after positive interim data from the Phase 3 study in the first quarter of 2013. There has been one larger insider sell in June 2012.

4. Durata Therapeutics (NASDAQ:DRTX) is a pharmaceutical company focused on the development and commercialization of therapeutic solutions for physicians and providers to advance patient care in infectious disease. Durata is currently enrolling patients in two global Phase 3 clinical trials with its lead product candidate, dalbavancin, for the treatment of patients with acute bacterial skin and skin structure infections.

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Upcoming Phase III catalysts

The company expects to complete two Phase 3 clinical trials and have initial, top-line data available in the beginning of 2013. If the company's ongoing Phase 3 clinical trials are successful, the company plans to submit a New Drug Application to the FDA in the first half of 2013 and a marketing authorization application to the EMA in the second half of 2013.

To date, Dalbavancin has completed a total of fifteen Phase 3, Phase 2 and Phase 1 clinical trials, over approximately ten years, in which more than 1,000 patients have been dosed with dalbavancin.

Financials

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

Net loss$8.3 million
Cash$25.3 million

After the end of the first quarter the company announced on July 24 the closing of its initial public offering of 8,625,000 shares of common stock at a public offering price of $9.00 per share, before underwriting discounts.

My analysis

I believe the stock could best the $10 level after positive Phase III data early in 2013. There was one insider buy filed on August 16.

5. Rigel Pharmaceuticals (NASDAQ:RIGL) is a clinical-stage drug development company that discovers and develops novel, small-molecule drugs for the treatment of inflammatory and autoimmune diseases, as well as muscle disorders. Rigel's pioneering research focuses on intracellular signaling pathways and related targets that are critical to disease mechanisms. Rigel's productivity has resulted in strategic collaborations with large pharmaceutical partners to develop and market its product candidates. Current product development programs include fostamatinib, an oral SYK inhibitor that is in Phase 3 clinical trials for rheumatoid arthritis with its partner AstraZeneca (NYSE:AZN); R343, an inhaled SYK inhibitor that has completed Phase 1 clinical trials for asthma; R333, a topical JAK/SYK inhibitor for discoid lupus; and R548, an oral JAK3 inhibitor for the treatment of transplant rejection and other immune disorders.

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Upcoming Phase III catalysts

AstraZeneca expects to report Phase 3 results from OSKIRA-1, OSKIRA-2, and OSKIRA-3 in the first half of 2013. They also expect to report data from OSKIRA-4 (a Phase 2b monotherapy study) by late 2012. In addition, AstraZeneca has stated that they expect to file a New Drug Application with the U.S. Food and Drug Administration for fostamatinib in the second half of 2013.

Financials

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

Revenue$1.5 million
Net loss$24.7 million
Cash$202.6 million

Rigel expects to end 2012 with cash and investments in excess of $145.0 million, which is expected to be sufficient to fund operations into 2014.

Upcoming (other) milestones

James M. Gower, chairman and chief executive officer of Rigel commented on August 7:

Rigel continues to advance its development programs with the commencement of our newest partnership with AstraZeneca to develop an inhalable asthma treatment, and our anticipated Phase 2 launches of R343 and R333 later this month.

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My analysis

The stock has a $18 price target from the Point and Figure chart. I believe the target price is achievable after positive Phase III results in the first half of 2013.

Source: 5 Pharmaceutical Companies With Phase III Clinical Study Catalysts During The Next 3 - 9 Months