Raptor Pharmaceuticals in 2011

Ball and stick model of the cysteamine molecule.Cysteamine Image via Wikipedia
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.

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.


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.

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