BioPharma Investor's  Instablog

Interest for Raptor Pharmaceuticals is really picking up in 2011.  Here are the recent headlines for this up and coming Small Cap Pharmaceutical company.  It has a Pipeline for Rare Diseases and I expect 2011 to be a strong year for Raptor Pharmaceuticals if Clinical Trial results are successful.  Results for DR Cysteamine clinical trials is expected in 1Q 2011.  DR Cysteamine has clinical development programs including:

◦Nephropathic Cystinosis, a rare genetic disorder (Phase 3)

◦Non-alcoholic Steatohepatitis (NASH), a metabolic disorder of the liver (Phase 2)

◦Huntington's Disease, an inherited neurodegenerative disease (Phase 2)

Raptor Pharmaceuticals Stock Quote:  RTTP  Google Finance

Read more from their previous 10-K filings for the company's Strategic Development Plan here.

Find out more about their Clinical Pipeline here.

Raptor Pharmaceutical Hires Patrick Reichenberger to Lead Commercial Development of DR Cysteamine for Nephropathic Cystinosis

Contracts in Place for Cambrex Corporation and Patheon, Inc. to Manufacture Active Pharmaceutical Ingredient and Commercial Product, Respectively

January 10, 2011 6:30 AM EST

NOVATO, Calif., Jan. 10, 2011 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTP) announced today the appointment of Patrick Reichenberger to the newly created position of Vice President, Commercial Operations. Additionally, Raptor has engaged Cambrex Corporation (NYSE: CMB) for the manufacturing of the active pharmaceutical ingredient ("API"), cysteamine bitartrate, and Patheon, Inc. (TSX: PTI) to produce the commercial product, DR Cysteamine.

A 20-year industry veteran with significant expertise in commercial operations and product-launch management, Mr. Reichenberger will lead the development and management of Raptor's sales and marketing efforts along with its commercial manufacturing, supply and distribution programs. Prior to joining Raptor, Mr. Reichenberger served as Senior Director of Commercial Development at XOMA, LLC. In this role, he led XOMA's commercial development department with particular emphasis on XOMA 052, an anti-IL1 antibody for diabetes, cardiovascular disease, and Behcet's Uveits, an orphan disease.

Prior to XOMA, Mr. Reichenberger was in charge of marketing, sales and distribution at Questcor Pharmaceuticals where he developed patient access and reimbursement programs supporting the successful commercial re-launch of H.P. Acthar® Gel for the treatment of infantile spasms, an ultra-orphan, pediatric disease. Prior to Questcor, he led commercial and strategic marketing for brands at Genentech, Athena Neurosciences, and Parke-Davis, where he assisted in the launch of Lipitor®.

"The addition of Patrick is particularly appropriate at this time as we work towards a potential commercial launch of DR Cysteamine for nephropathic cystinosis in 2012. His hands-on commercial development expertise will be central to our efforts to build the necessary commercial infrastructure and capability for the near term and as the Company grows in later years." stated Christopher M. Starr, Ph.D., Chief Executive Officer of Raptor.

Mr. Reichenberger stated "My experience in building commercial infrastructures around orphan therapeutics, especially Acthar® Gel, is particularly applicable to Raptor's nephropathic cystinosis program. I am encouraged by the enthusiastic patient and foundation support for Raptor's DR Cysteamine program, and I look forward to working to create a sound patient access program ready for commercial launch. While developing and implementing such a program for nephropathic cystinosis is my top priority at Raptor, I am also very excited about the entire Raptor pipeline. I am impressed with the commercial opportunities at Raptor with a near term program in an orphan indication for nephropathic cystinosis and mid-stage programs in other indications, including Huntington's Disease and NASH. I'm pleased to have the opportunity to use all of my capabilities, from long-term strategic planning to practical marketing and sales, in this position."

Ted Daley, President of Raptor stated "We are very pleased to welcome Patrick to lead our commercial efforts. The addition of Patrick to our leadership team, along with our recently established long-term manufacturing agreements with Cambrex and Patheon, are significant milestones in the continued advancement of our DR Cysteamine programs. Both Cambrex and Patheon have significant expertise working with cysteamine bitartrate. Cambrex has provided us with cysteamine bitartrate for all our clinical studies and, for over 10 years, has been one of the few API manufacturers worldwide that is capable of providing a commercial source of pharmaceutical-grade cysteamine bitartrate. Patheon developed Raptor's DR Cysteamine microbead formulation and currently manufactures our clinical drug supply. We look forward to employing their combined expertise and capabilities in our commercial manufacturer."

About Nephropathic Cystinosis

Nephropathic cystinosis is an inborn metabolic error characterized by the abnormal transport of cystine, an amino acid, out of the lysosomes. Failure to treat nephropathic cystinosis can cause serious health consequences, including renal failure and resultant kidney transplant, growth failure, rickets, photophobia and blindness. Symptom onset typically occurs within the first year of life, when cystine crystals accumulate in various tissues and organs, including the kidneys, brain, liver, thyroid, pancreas, muscles and eyes.

About Cysteamine and DR Cysteamine

DR Cysteamine is Raptor's proprietary enteric-coated, microbead oral formulation of cysteamine bitartrate designed to potentially reduce dosing frequency and gastrointestinal side effects associated with immediate-release cysteamine bitartrate, which is approved for sale by the FDA and EMA to treat nephropathic cystinosis, a rare, genetic lysosomal storage disease.

In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego for the development DR Cysteamine for nephropathic cystinosis and cysteamine for other potential indications including Huntington's Disease, NASH and Batten Disease.

About Raptor Pharmaceutical Corp.

Raptor Pharmaceutical Corp. (Nasdaq: RPTP) ("Raptor") is dedicated to speeding the delivery of new treatment options to patients by working to improve existing therapeutics through the application of highly specialized drug targeting platforms and formulation expertise. Raptor focuses on underserved patient populations where it can have the greatest potential impact. Raptor currently has product candidates in clinical development designed to potentially treat nephropathic cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase ("ALDH2") deficiency, and thrombotic disorder.

Raptor's preclinical programs are based upon bioengineered novel drug candidates and drug-targeting platforms derived from the human receptor-associated protein ("RAP") and related proteins that are designed to target cancer, neurodegenerative disorders and infectious diseases.

For additional information, please visit http://www.raptorpharma.com/.

Wedbush Starts Raptor Pharmaceuticals (RPTP) at Outperform

January 6, 2011 5:05 PM EST

Wedbush initiates coverage on Raptor Pharmaceuticals (NASDAQ: RPTP) with a Outperform rating and $7 price target.

The firm said, "We expect that Phase III data from RPTP’s lead DR Cysteamine program, anticipated in Q1:11, to be an important positive catalyst for the stock. Mature results from the Phase III results, in our opinion, will demonstrate non-inferiority to Cystagon as suggested by previous Phase IIa and IIb studies. Additionally, the drug, a delayed-release formulation of the approved active ingredient cysteamine bitartrate (Cystagon), should offer a significant improvement in quality of life and should demonstrate a more favorable safety profile with fewer side effects, in our opinion."

For more ratings news on Raptor Pharmaceuticals click here and for the rating history of Raptor Pharmaceuticals click here.

Shares of Raptor Pharmaceuticals closed at $3.39 yesterday, with a 52 week range of $1.41-$4.00.

Raptor Pharmaceuticals: A Pipeline With Potential
October 07, 2010
By: Tony Pelz-Seeking Alpha

In my continuing search for “off the beaten track” small cap biotechs with impending catalysts, I came across Raptor Pharmaceuticals (Ticker: RPTP). Despite its checkered past, RTPT possesses an interesting pipeline with numerous near-to-medium term catalysts. According to the Company,

Raptor currently has product candidates in clinical development designed to potentially treat nephropathic cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's Disease, aldehyde dehydrogenase deficiency and a non-opioid solution designed to potentially treat chronic pain.

The Company also has a small roster of pre-clinical products.

Here are the catalysts and associated estimated market values:

•DR Cysteamine (Cystinosis): Phase 3 started June 2010; data expected to be reported 4Q’10/1Q’11; est. market value: $100 million

•DR Cysteamine (NASH): Phase 2a data released 5/2010; IND filing for Phase 2b 1H’11; est. market value: $1.8 billion

•DR Cysteamine (Huntington’s Disease): Phase 2 planned initiation Q3’10; est. market value: $2.1 billion

•Convivia: Out-licensing discussions; est. market value: $1.8 billion

•Tezampanel/NGX-426: Phase 2/3 ready, out-licensing discussions; est. market value: $700 million

The core medium term value drivers of the business going forward are clearly the candidates targeting NASH and Huntington’s. For the NASH indication alone, the Company claims there are currently 7.5 million adults (25 million by 2025) suffering from the disease with no current approved treatments. Investors are obviously cognizant of this market potential as positive results from Phase 2a data released in May 2010 caused the shares to rocket from around $1.50 to over $3.85. In addition, the stock regained all of its losses in just three days following a highly dilutive stock offering back in August; there appears to be a floor to the share price – quite rare. The near term Phase 3 data (Cystinosis) expected in 4Q’10/1Q’11, although targeting a smaller market, should at the very least (if positive) provide some price stability given the expectations that the Company should eventually be positioned to generate organic cash flows in the near to medium term with an approved product – i.e. to survive long enough to get the other “big ticket” products approved.

On the financial side, RPTP maintains a cash balance of around $17.2 million with no debt. Raptor management believes existing cash levels, at current monthly cash burn, will last into December 2011 – this is a decent amount of leeway. The Company currently has around 30.1 million shares (41.9 million fully diluted) outstanding – if the shares begin to run, look out for additional equity raisings and associated dilution.

I am approaching this trade as more of a 1 year + horizon investment. I attempted to build a position with options for about 4 weeks but to no avail; far too illiquid with ridiculous spreads (4) – I would expect spreads to tighten up as word gets out about this Company and its potential. As such, I will be slowly building a common stock position. On that front, I don’t like the technical/chart for the stock. After the May 2010 price spike, the shares have flat lined into a tight range between $2.70 and $3.00 – also, this range has tighten significantly over the past several weeks. Usually, this tells me that the stock is about to make a big move in either direction (i.e. up or down). Thus, I intend to slowly build this position and not put on the entire trade in one lot. As options become more liquid, I may either trade out of the shares into a long synthetic, sell Calls against the position or sell Puts.

Looking forward, I don’t expect the stock to make a huge move on the near term Phase 3 data (if positive). That said, the Company’s other products and associated potential upside appear quite substantial. If these products begin to rack up decent data in the trials, not only will the market take notice (and hence the shares rise) but Raptor could become a decent takeover target.

Raptor Pharma (RPTP) Updates on DR Cysteamine Phase 3, Financials
August 24, 2010 6:49 AM EDT

Raptor Pharmaceutical Corp. (Nasdaq: RPTP), provides updates on timelines related to its clinical programs including the pivotal Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

Financial Updates and Guidance

As of August 24, 2010, the Company has approximately $17.2 million in cash and cash equivalents taking into account approximately $14 million of proceeds, net of placement agent fees and expenses, the Company received in a private placement financing which closed on August 12, 2010. The Company anticipates that its cash and cash equivalents will support planned operations and its planned clinical development programs, as described below, into December 2011.

Cystinosis
Raptor's cystinosis program remains the highest priority for the Company. "Over the next 18 months, the majority of the Company's resources will be devoted to completing what we believe will be the final development phase of this program," said Christopher M. Starr, Ph.D., CEO of Raptor. "Anticipating the successful completion of our ongoing Phase 3 clinical trial in 2010 and NDA submission in 2011, we are building the commercial infrastructure in our anticipation of a commercial launch in 2012."

In November 2009, Raptor completed its Phase 2b clinical trial of DR Cysteamine in cystinosis. DR Cysteamine demonstrated improved tolerability and the potential to reduce total daily dosage and administration frequency compared to immediate-release cysteamine bitartrate.

On June 28, 2010, Raptor enrolled the first patient in its pivotal Phase 3 clinical trial in cystinosis. This trial is designed as a randomized, crossover, outpatient study of the safety, tolerability, pharmacokinetics ("PK") and pharmacodynamics ("PD") of DR Cysteamine compared to immediate-release cysteamine bitartrate in cystinosis patients. The study is being conducted at nine centers: four in the U.S., including Emory University, Children's Memorial Hospital at Northwestern University, Stanford University, and Texas Children's Hospital at Baylor University; and five in the EU including in Hopital Necker-Enfants Malades and Hopital Robert Debré in Paris, Hospices Civils de Lyon in Lyon, France, Centre Hospitalier Universitaire in Montpellier, France and Radboud University Medical Center in Nijmegen, The Netherlands.

"We are very excited to have begun this study and are pleased with the patient support and initial rate of enrollment we are seeing at our sites," said Craig Langman, M.D., the Isaac A. Abt, M.D., Professor of Kidney Diseases at Feinberg School of Medicine, Northwestern University in Chicago and Principal Investigator of the Phase 3 clinical trial. "We currently have three U.S. sites enrolling patients and a fourth to commence early next month. Our European sites will begin enrolling patients by mid-September".

The Company expects to complete patient enrollment in the next few months and anticipates that all patients will have completed the treatment period by the end of the year. The treatment period for each of the anticipated 30 patients in the clinical trial is a total of 9 weeks and all patients will continue on DR Cysteamine in an extension study following the treatment period.

If the results from this trial are successful, Raptor anticipates filing an NDA for DR Cysteamine in cystinosis in mid-2011 and is planning a concurrent filing with the EMA for registration in the EU.

Huntington's Disease

Raptor anticipates that the Phase 2 study using DR Cysteamine in 96 Huntington's Disease patients will start in the third quarter of 2010. The Phase 2 clinical trial will be conducted under a previously announced collaboration agreement with The Centre Hospitalier Universitaire d'Angers ("CHU d'Angers") of Angers, France. Clinical expenses of the study will be covered by a grant from the French government.

Under the collaboration agreement between the Company and CHU d'Angers, Raptor will supply the DR Cysteamine study drug and will retain commercial rights to the clinical trial results. This Phase 2 trial was contemplated based on encouraging preclinical results reported by Institut Curie, CNRS and Inserm scientists that cysteamine prevents death of neurons and increases levels of a brain growth factor called brain-derived neurotrophic factor ("BDNF") in Huntington's Disease models. Reduced levels of BDNF in the brain have been widely reported to be important in the clinical development of Huntington's Disease.

In addition to intellectual property licensed from University of California, San Diego ("UCSD") related to its DR Cysteamine programs, Raptor holds exclusive worldwide licenses to Huntington's Disease related intellectual property from the Weizmann Institute of Science in Israel and Japan's Niigata University.

Non-Alcoholic Steatohepatitis ("NASH")
Raptor's management views its DR Cysteamine development program in NASH as an exciting opportunity based on both its potential market size and the possibility of creating partnering relationships. Following encouraging results from its Phase 2a clinical trial of DR Cysteamine in juvenile NASH patients, reported earlier this year, Raptor is moving its NASH program forward in anticipation of a future Phase 2b clinical trial.

In the next six months, the Company will be developing a commercial-ready formulation for this potential indication, designed as a delayed-release, coated compressed tablet dosage form of cysteamine bitartrate, which will be appropriate for the expected dose levels for NASH patients, anticipated to be significantly lower than the DR Cysteamine dosage for cystinosis patients. The Company plans to file an IND with the FDA by mid-2011 for a Phase 2b clinical trial to start when funding for this trial becomes available. In this regard, the Company is exploring partnering opportunities and potential grant support for the Phase 2b clinical trial.

ConviviaTM

The Company completed its first out-licensing agreement of ConviviaTM with Uni Pharma Co., Ltd in Taiwan in June 2010. Raptor has ongoing discussions with other Asian companies to potentially develop ConviviaTM further in various Asian markets.

Stocks Mentioned:  Dendreon, Human Genome Sciences, Eli Lilly, Cerus, Curis, Compugen, Spectrum Pharmaceuticals, Impax Laboratories, Exelixis, Medivation, Seattle Genetics, Corcept, PolyMedix, BioMarin, Endo Pharmaceuticals, DepoMed, Mannkind, Orexigen Therapeutics, AstraZeneca, Clinical Data

Here are the Top Biotech Picks for 2011, Stocks Highlighted at the JP Morgan Healthcare Conference, News on Human Genome Sciences, Dendreon, Bargain Biotech Stocks, and January FDA Catalysts.

5 Bargain Biotech Stocks for 2011

Street Picks

NEW YORK (InvestorPlace) -- On July 8, 2009, I recommended five cutting-edge medical products and biotech stocks. Even factoring in the single dud of the group, you would still have an average return of more than 110%.

Do I have your attention? Good, because I've put together a list of the hottest bargain biotech stocks to buy for 2011. Some of the names will look familiar because I think the stocks still have legs, while others have been swapped out for new names that I think have more potential in the year ahead.

Here are the best biotech stocks to own now:

No. 1: Cerus

Cerus (CERS) developed and markets the INTERCEPT Blood System, which is designed to inactivate blood-borne pathogens in blood components so the blood can be used in transfusions. In other words, it "cleans" donated blood of viruses, bacteria and parasites.

Cerus is pretty much the only game in town with this remarkable technology, and it has gained approval in most large European countries. Why not the United States? Well, management has not stood up to the FDA. The approval has been held up by one member of the FDA even though Cerus hit the primary endpoints in its pivotal Phase III trial and is receiving grants from the Department of Defense.

The FDA should quit dragging its feet eventually. There is no scientific or product risk in this stock. Cerus' system works. My target price is $14 in one to three years.

•Related Article: 10 Best Stocks for 2011

No. 2: Curis

Curis (CRIS) has developed a series of cancer treatments based on a technology that disrupts intercellular signaling in the Hedgehog pathway. Disrupting communication disrupts cell duplication, the foundation of tumor growth.

Curis has more than 20 trials under way with Genentech/Roche and the National Cancer Institute. This year, Genentech will likely report results of a basal cell carcinoma trial for skin cancer, and it has said it will go from this mid-phase trial directly to an application for approval if the results are strong enough.

One success means a volcanic eruption in the stock, as it will prove the core technology is a viable platform for cancer treatments. A failure could put the entire program -- and the company -- in jeopardy. I believe the technology will be a success, which means this $2 stock could be worth $40-plus. If I'm wrong, you will probably be looking at a 50-cent stock. I'd say it's worth the risk.

Related Article: 5 Top Bargain Stocks Less Than $5

No. 3: Compugen

Compugen (CGEN) is the world's leading molecular intellectual property company. Based in Israel, the company has revolutionized the early phases of drug development through a highly automated process of exploring and selecting molecules with the greatest promise to serve as the basis for a particular treatment.

The company licenses its peptides and proteins for a fee to the who's who of the drug industry, and also receives a back-end cut of any drug that makes it to market using its discoveries.

Compugen just announced that it entered into an agreement with Baize Investments under which it will receive $5 million in R&D funding. My target for CGEN is $20 in three to five years.

•Related Article: 5 Dividend Stocks That Will Trump the Dow

No. 4: Spectrum Pharmaceuticals

Spectrum Pharmaceuticals (SPPI) is a commercial-stage biotechnology company with a primary focus in oncology and hematology The company specializes in rescuing treatments abandoned, in development stages, by other companies.

It has had a tremendous run based on market introductions and partnerships in the past two years, but now has even greater potential for a blockbuster with a drug called Zevalin for non-Hodgkin's lymphoma. This drug is currently approved as a salvage and adjunct therapy, and the company is in mid-stage trials for the use of Zevalin as a front-line treatment, which would be a much larger market.

The risk in this stock is high. It could be cut in half or worse on bad news from one of several clinical trials. However, successful trial results could take this stock from less than $7 to $32 in one to three years. SPPI could also become a takeover target.

No. 5: Impax Laboratories

The not-so-small generic drug maker Impax Laboratories (IPXL) (NASDAQ: IPXL) has arguably the best manufacturing technology for time-released drugs in the entire generic industry.

Pfizer's (PFE) patent for its $11 billion cholesterol drug Lipitor expires this year, and due to legal actions, it is already known that IPXL has figured out how to make a generic version of the blockbuster drug. I expect it to market it through a partner either in November of this year or six months later in May 2012 due to FDA regulations.

Two other major product introductions are anticipated in 2011: generic Concerta for ADHD and generic Solodyn for bacterial infections, currently with combined sales of $1.8 billion.

My target for the stock is $35-$40 in one to two years. IPXL is also the possible target of an acquirer.

Stocks to Watch at JPMorgan Healthcare Conference

By David Miller Jan 07, 2011 3:00 pm

Next week investors will congregate in California to discuss the future of the biotech industry. Companies to watch include Exelixis, Medivation, and Dendreon.

It’s the time of year for the JPMorgan (JPM) Healthcare Conference. The conference, which runs Monday through Thursday next week in San Francisco, is the surest place to see everyone in biotech each year. The size of the crowd at the conference, held at the Westin St. Francis hotel, is a decent indicator of how hot the biotech sector is each year. This year’s crowd will be 20% above last year's and one of the largest since I started attending in 2003.

Our firm sets up in a suite down the street and has a steady parade of companies through the doors. This is an excellent opportunity to catch up with the companies we already cover, but more importantly meet new companies to add to our coverage universe. Here’s what I’m looking forward to next week

Exelixis (EXEL) recently released seemingly excellent Phase II data in prostate cancer. Shortly thereafter, management announced they were focusing the company’s assets on this program. I’ve looked at Exelixis often since its first appearance at JPM and have always been turned off by its spendthrift ways. This year it seems to be different. I’m excited to sit down with management to chat about both the data and how they're focusing their resources.

Medivation (MDVN) is one of the companies my clients are always asking about. I’ll want to chat about MDV3100, its prostate cancer drug, as well as the other drugs in its pipeline.

Dendreon (DNDN) got most of the big news out of the way in a conference call this morning so they could talk with investors one-on-one without breaking Reg FD. Their EU strategy is clear, they hit 2010 revenue numbers, and reaffirmed 2011 guidance. Once the company announces how they will raise money (and how much) to support the EU expansion, the stock should be in the clear for 2011. This stock was my firm’s top pick for 2011 back in August and is atop many “top biotech investments for 2011” lists.

Seattle Genetics (SGEN) signed a nice technology deal with Pfizer (PFE) Thursday. There's a running argument in the biotech community whether ImmunoGen’s (IMGN) or Seattle Genetic's antibody-drug conjugate technology is better. Both companies can point to clinical success, but Seattle Genetic has been leading in big-dollar deals lately. SGEN traditionally raises money around this time of year. If they forswear doing that in their wall-to-wall meetings with investors next week, it might make a difference to the stock price. The company is due to file a BLA for FDA approval of brentuximab vedotin Q1-2011. I expect approval six months later for both Hodgkin’s Lymphoma and ALCL.

Corcept (CORT) investors were disappointed when excellent data for Corlux in Cushing’s Syndrome was met with sell-the-news declines in the stock price. Management needs to do the fundraising everyone expects and get it done sooner rather than later. We’re hoping they're not shy during their meetings with investors when pointing out why their trial design and data are stronger than data in Cushing’s from Novartis (NVS).

PolyMedix’s (OTC:PYMX) size and OTC listing makes it largely invisible to most investors. Its antibiotic platform is new and potentially very exciting. 2010 saw a steady rise in interest in PolyMedix on the part of investors until the FDA asked for more data before allowing clinical trials to start in the US (Canadian authorities had no issues, so trials continue there). In my chat with them, I’ll be looking to understand the impact on timelines and making sure this type of “unexpected” outcome isn’t repeated.

BioMarin (BMRN) has been a top pick of my firm for years. We’ve been covering it since 2004 when it was trading around $7. This orphan disease-focused company is perennially on the likely-to-be-acquired list, particularly given big pharma’s recent appetite for these companies. This year, BioMarin appears to be energized to put its large cash balance ($440 million) to work advancing its pipeline.

The schedule has many other companies, but I’m most excited to meet with these. JPM is also a chance to meet with clients -- existing and prospective. Frankly, I also enjoy watching all my East Coast friends try to adapt to market life on the West Coast -- up late, up early.

Read coverage from last year's conference: Now Is the Time to Invest in Health Care.

January Stock Catalysts for Biotech and drug Companies

By Brett Chase Jan 04, 2011 2:20 pm

Orexigen, Endo Pharmaceuticals and Clinical Data are among stocks that will move on FDA action.

US officials are expected to act on a number of drug applications this month. The moves will drive trading in companies, especially smaller biotech firms that live and die by Food and Drug Administration approvals. Here’s a look at some important FDA events for January. As always, dates are subject to change.

January 7

The FDA is expected to decide whether Endo Pharmaceuticals’ (ENDP) extended-release, tamper-resistant pill version of painkiller Opana should be approved for sale. Endo’s pill is designed to be crush-proof to help thwart drug abuse. The application is getting a speedy review from regulators. Last month the agency approved a separate application for Endo’s testosterone gel, a decision that boosted the company’s shares.

The FDA also is expected to decide by January 7 whether AstraZeneca’s (AZN) thyroid cancer drug vandetanib should be approved. Last month, a panel of expert government advisers recommended that the drug be approved but also said the treatment should be studied further to evaluate safety.

January 12

Eli Lilly’s (LLY) pancreatic enzyme replacement therapy liprotamase faces an FDA panel. Lilly acquired the treatment with its $180 million purchase of closely held Alnara Pharmaceuticals last year. The FDA rejection in October of Lilly’s diabetes drug Bydureon was a big blow for a company that needs new products to replace older drugs. Lilly hasn’t said much about liprotamase, which treats cystic fibrosis patients and would have competition from drugs already on the market.

January 20

Eli Lilly (LLY) faces another panel that will consider its imaging agent florbetapir, which is aimed at helping diagnose Alzheimer’s disease. Lilly acquired the product through its acquisition of Avid Radiopharmaceuticals in November for $300 million. Lilly agreed to pay up to another $500 million if the product is approved and meets sales goals.

January 22

Clinical Data (CLDA) is expected to receive word from the FDA about approval of its depression drug Vilazodone. While some analysts predicted a potential blockbuster drug, shares of the company dropped 18% in the past month on concerns the drug may not be approved.

January 26

MannKind (MNKD) was supposed to hear from the FDA December 29 on whether its fast-acting inhaled insulin treatment Afrezza would be approved. The agency said it needed “about” four additional weeks to review the application. Afrezza was rejected by the FDA early last year. Analysts are divided on whether the product can be approved this time.

January 30

Abbott Laboratories (ABT) and Depomed (DEPO) are expecting to get an agency decision on the experimental drug DM-1796, for the treatment of pain caused by shingles. Depomed’s stock doubled in the past year. The company licensed the drug to Solvay, which was later acquired by Abbott.

January 31

Orexigen Therapeutics (OREX) should get word on whether its diet pill Contrave will be approved. A panel of FDA advisers surprised the investment world by recommending approval even after agency staff expressed concerns about safety and effectiveness. The advisers recommended a safety study after approval. Don’t be surprised if the FDA decides it needs more time to review this drug. Diet pills are highly controversial and two other weight-loss drugs offered by Arena Pharmaceuticals (ARNA) and Vivus (VVUS) were rejected by the FDA last year.

 On the Rise: Human Genome Sciences

By Brett Chase Jan 11, 2011 1:30 pm

Bulls on the stock say new lupus drug Benlysta will be approved and is a sure blockbuster product.

Even before CEO H. Thomas Watkins promised yesterday to generate billions in new revenue by 2015, investors were warming to Human Genome Sciences (HGSI) again.

Shares of the company are up 12% this year, trading midday Tuesday at $26.75. That’s down from a high of $33.30 last year. But there are still lingering fears that experimental lupus drug Benlysta won’t be approved or use of the treatment will be limited because of concerns about its effectiveness.

The Food and Drug Administration is considering the drug’s approval after a panel of expert advisers in November recommended allowing Benlysta on the market, making it the first new lupus drug in half a century. After delaying its decision last month, the agency is expected to act by early March.

Bulls on this stock believe Benlysta will be a multi-billion dollar blockbuster drug for Human Genome and development partner GlaxoSmithKline (GSK). Success may also make Human Genome a prime takeover candidate for a big drug company.

“We continue to be extremely enthusiastic about the commercial prospects of Benlysta,” says Stifel Nicolaus analyst Maged Shenouda, who rates the stock a buy.

Shenouda, who has a $38 target price for the stock, believes the drug will be approved and it will generate $156 million in sales this year, climbing to $2.9 billion by 2014. After US approval, Shenouda believes the drug also will be cleared for sale in Europe.

Like any good analyst, Shenouda has been doing his homework, contacting medical experts to get a pulse on whether doctors will prescribe the product.

“Based on multiple clinician discussions, we believe Benlysta’s efficacy and safety profile will drive broad adoption,” he says.

He bases his 2014 revenue estimate on Benlysta taking about 20% of the lupus drug market in the US and more than 14% in Europe.

What’s more, Human Genome will be an attractive takeover candidate, he says.

The knocks on the drug: Limited effectiveness, particularly in African-American patients. But the safety concerns are minimal and this is a disease that hasn’t been treated with a new drug for a very long time, not to mention, lupus is a horribly painful condition.

The label may even carry disclaimers, stating -- for instance -- that Benlysta showed less of a benefit in black patients, Leerink Swann analyst Joseph Schwartz says in a note last month.

“We believe the revenue impact of such statements would be minimal, considering the lack of other attractive treatment options,” says Schwartz, who rates the stock a buy and has a $35 a share price target.

In case there was any doubt, CEO Watkins told analysts and investors at a conference yesterday that 2011 is a turning point for the money-losing company. He says a sales team is in place for a near-term drug launch.

“Based on the opportunities we have in hand and our expectation of US and European regulatory approvals of Benlysta, we believe Human Genome Sciences will achieve sustainable multi-billion dollar annual revenues by 2015,” Watkins said. This year “will be a game-changing year for us assuming approval and launch of Benlysta.”

Dendreon's Provenge Overhang

By Brett Chase Jan 07, 2011 1:40 pm

Signs are positive for Dendreon moving ahead with its prostate cancer vaccine Provenge in the US and eventually Europe. Medicare reimbursement however, continues to hold back the stock.

Dendreon (DNDN) is moving ahead with plans to sell its prostate cancer vaccine Provenge in Europe, a positive signal for a stock that’s been beat up over reimbursement worries in the US.

Dendreon will apply either late this year or early 2012 to sell Provenge in Europe. Company executives say they won’t be required to conduct any new human studies to get approval there -- another positive. If all goes as planned, Dendreon will get European clearance by 2013.

Despite the positives, Dendreon’s shares aren’t exactly rallying on the news. The stock rose about 4% to $36.93 in midday trading. The shares are down 4% over the past three months and down almost a third from their high last year as concerns over the company’s capacity to make enough Provenge and insurance reimbursement questions spooked some investors.

In November, a panel of experts advising Medicare and Medicaid recommended that the government should reimburse patients who receive the vaccine. The agency that oversees the two public health plans ordered the review after Provenge was approved for sale in the US last year. The Centers for Medicare and Medicaid Services says it will draft guidance on paying for the treatment by March and will make a decision by June 30. Provenge patients are currently being reimbursed by government and private insurance.

Dendreon reiterated an earlier estimate that Provenge sales will be as much as $400 million this year and the company will continue to lose money as it spends to produce and sell the vaccine and expand to Europe. (Provenge revenue was $48 million last year.) Dendreon plans to build a plant in Germany and initially will contract with another company to produce Provenge. That’s, of course, contingent on Provenge winning EU approval.

The bulls on this stock think Dendreon is way undervalued.

Robert W. Baird analyst Christopher Raymond has a price target of $56 a share. The stock closed above $54 after receiving approval last year.

“Now may be the best time to get involved in this name, ahead of Dendreon’s planned capacity expansions and in particular ahead of additional or more substantive signals of strong commercial uptake,” Raymond says in a note yesterday.

Provenge is a new type of treatment. It’s a vaccine that turns a patient’s immune system against cancer. In men with advanced prostate cancer it’s been shown to extend their life an average of four months. A number of analysts predict the treatment eventually will exceed $1 billion in annual sales.

“What we’re in is a golden era for patients with prostate cancer,” Dendreon CEO Mitchell Gold said on a call with investors and analysts this morning.

This wonder drug comes with a big price tag: $93,000 per treatment. Conspiracy theorists believe the government’s review of the treatment is related to sticker shock. But Medicare’s analysis isn’t supposed to consider price. The review in November was over safety and effectiveness.

Pricing the product in Europe will be an issue as well. But that’s a challenge every drug maker faces as a number of countries have price controls.

Gold says demand continues to be strong and plans for production and sales are on track. The company has 100 sales people and production capacity will increase tenfold this year as plants in New Jersey, Los Angeles and Atlanta come on line.

The only overhang on the stock seems to be the Medicare issue. A bet on Dendreon continues to be a call on whether insurance reimbursement is upheld. So far, the signs point to that happening.

So Pfizer is increasing their portfolio with King Pharmaceuticals, a maker of veterinary meds and pain therapies.  In addition to this news, another company came into the spotlight for pain management therapies.  This company is Pain Therapeutics (PTIE).  Pain Therapeutics is in clinical trials now in collaboration with King Pharmaceuticals so look for Pfizer to kick in some clinical trial expertise with Pain Therapeutics (PTIE).  Additional companies involved in pain therapies is Endo Pharmaceuticals.  I highly suggest buying Endo as a buy right now.  Since June 2010 Endo's stock has risen form 20.22 to its current trading at 34.99.  I suggest a limit order to buy at some discount.  Another Pain therapies stock is Watson Pharmaceuticals, a generic drug maker. 

My opinion on this is that Pain Therapeutics could be slightly overvalued on the hyped news, and although it may seem attractive to buy right now, I suggest waiting for the pullback as it will be years before these clinical trials are actually approved by the FDA.  Also, I am a little cautious placing a buy on Pfizer right now as they will be losing patents on Lipitor next year and other brand name drugs will become available as generics.  I might actually place Pfizer's stock right now on hold or sell, as earnings could be lower than expected.  Just because everyone is doing it does not make it a buy in my opinion. I actually do not necessarily see King Pharmaceuticals as such a hot buy with only 4.85% profit margin and 18M in profits on 380M in revenue.  But given that layoff will presume who knows. 

Stick to your guns and remain a cautious investor and good things will come.  I apply Warren Buffett's principles to the BioPharma Stock World and like purchasing stocks at a discount.  You should be searching for the next Pfizer and not chasing after something that is overly hyped.  Just my opinion.

Stocks mentioned in this article

Pfizer--(PFE)
King Pharmaceuticals --(KG)
Endo Pharmaceuticals --(ENDP)
Pain Therapeutics --(PTIE)  Up 18.56% today on the news
Watson Pharmaceuticals--(WPI)

The Street:   Biotech

NEW YORK (TheStreet) -- Pain relief became the theme of Tuesday's trading session in biotech land, after Pfizer's(PFE) $3.6 billion deal to acquire King Pharmaceuticals(KG), a pain-reliever specialist, came to light before the opening bell.

The news drove King's stock to the cusp of Pfizer's offer price -- the equivalent of $14.25 a share, a 40% premium to the Monday close. In morning trading Tuesday, shares of the Bristol, Tenn.-based King, which also does a robust business in veterinary medicine, were trading at $14.15, up 39.5%, or $4.01, on enormous volume.

The deal also helped the shares of an early-stage painkiller developer with a rather literal name: Pain Therapeutics(PTIE), which has teamed with King on the development of Pain's lead drug candidate, called Remoxy, currently in clinical trials.

The two companies also have a licensing agreement for Remoxy, a "strong painkiller," Pain says in its press materials, the main selling point of which appears to be that it has a "unique formulation designed to reduce potential risks of unintended use." In other words, the Remoxy recipe, delivered as a capsule, makes it hard for users to binge on the drug and get addicted to its painkiller qualities.

Investors and traders were apparently excited by the notion of a global Pfizer sales and marketing force pushing Remoxy into the dreamed-for blockbuster realm. King was slated to submit a new drug application for Remoxy with the Food and Drug Administration sometime during the fourth quarter.

Pain shares were jumping nearly 20% to $7.43 in brisk trading.

For the most part Tuesday, biotech stocks were, however, in the red, following the broader market lower. The Nasdaq Biotechnology Index was down 1.78 points to 906.61.

Alexza Pharmaceuticals(ALXA) stock continued to decline after Monday's bloodbath, sparked by the FDA's decision to deny approval of the company's schizophrenia treatment.

In morning action Tuesday, Alexza shares were moving at $1.30, down 8 cents, or nearly 6%, on heavier-than-average volume. Investors wiped out half the company's market value during the previous session.

Elsewhere in the world of experimental tinctures, Geron (GERN) stock continued to climb in the wake of the stem-cell drug developer's historic launch of human clinical trials Monday.

The stock was changing hands at $6.06 in recent trades, up 7% from the close in the previous session, when they added 6.4%. Volume in Geron shares Tuesday morning was heavy again, surpassing 2 million before 11 a.m. EDT, double the daily average.-- Written by Scott Eden in New York

TheStreet:  Drugs

NEW YORK (TheStreet) -- Pfizer(PFE) is buying King Pharmaceuticals(KG) for $3.6 billion in cash to bolster its presence in the pain relief market, the company said Tuesday.

The deal values King at $14.25 a share, or a 40% premium over King's closing price of $10.15 a share Monday. Pfizer said the acquisition will have no material impact on 2010 earnings but will add approximately 2 cents a share to the drugmaker's estimated 2011 and 2012 earnings.

King is a specialty pharmaceutical company that specializes in pain drugs, including Embeda, an opioid designed with features that make it resistant to abuse and misuse. King earned $1.10 a share in 2009 on total revenue of $1.78 billion but is expected to earn 68 cents a share on $1.48 billion this year, according to Thomson Reuters.

Pfizer said the King acquisition allows the company to expand its current pain offerings, which include Lyrica and Celebrex.

Pfizer shares were down 5 cents to $17.33 in premarket trading Tuesday.   They
have since gained in value to 17.48. 

--Written by Adam Feuerstein in Boston.