Binary events like clinical trial results and FDA decision are typical in biotech firms, and can cause significant swings in share price, particularly if they are smaller companies.
Let's take a look at some of the catalysts that are in store for biopharmaceutical company Raptor Pharmaceutical Corp. (RPTP).
Raptor is focused on speeding the delivery of new treatment options to patients by working to improve existing therapeutics. The company's lead product candidate is DR (delayed release) Cysteamine, which is being evaluated for potential indications, including Nephropathic Cystinosis, Non-alcoholic Steatohepatitis and Huntington's 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, Non-alcoholic Steatohepatitis or NASH for short and Batten Disease.
DR Cysteamine is Raptor's proprietary enteric-coated, microbead oral formulation of Mylan Inc.'s (MYL) Cystagon, which contains the active ingredient cysteamine bitartrate. Cystagon is approved by the FDA and EMA to treat cystinosis, and is the standard of care for the cystinosis treatment.
Cystinosis is a rare inherited disorder characterized by the accumulation of cystine within body cells, leading to impaired cellular function. This disorder causes kidney failure, growth retardation vision problems and sometimes even death.
Cystagon, the standard of care for the cystinosis treatment, has significant compliance issues and the disease is not well managed in majority of the patients. DR Cysteamine is designed to potentially mitigate gastrointestinal side effects and the difficulties of frequent dosing associated with the Cysteamine therapy.
DR Cysteamine for nephropathic cystinosis is expected to be dosed twice daily, compared to Cystagon, which requires dosing every six hours. In addition, DR Cysteamine is designed to pass through the stomach and deliver the drug directly to the small intestine, where it may be more easily absorbed into the bloodstream and may result in fewer gastrointestinal side effects, according to the company.
The company commenced the phase III trial of DR Cysteamine for the potential treatment of cystinosis last June. The study compares the safety and tolerability of every 12-hour DR Cysteamine to Cystagon (immediate-release cysteamine bitartrate), the current standard of care, in nephropathic cystinosis patients.
The primary endpoint of the study is the steady-state white blood cell cystine levels of patients taking DR Cysteamine compared to Cystagon. Secondary endpoints are the safety and tolerability of DR Cysteamine and the comparability of steady-state pharmacokinetics of DR Cysteamine and Cystagon in nephropathic cystinosis patients. The cystinosis population is about 500 in the U.S. and 2,000 worldwide. The potential market for cystinosis treatment is estimated to be more than $100 million.
The company is slated to report top line data from the phase III clinical trial of DR Cysteamine for nephropathic cystinosis sometime within the second quarter of 2011.
DR Cysteamine is also under phase II stage of development in two trials where it is being evaluated as a potential treatment for Non-alcoholic Steatohepatitis and Huntington's Disease.
Non-alcoholic Steatohepatitis is a progressive liver disease, with a 25% incidence in obese patients. About 75 million adults in the U.S. have NASH , which can cause cirrhosis, liver failure and end-stage liver disease.
Currently there are no treatment options for NASH. The disease is managed with lifestyle changes such as diet, exercise and weight reduction. The anticipated market potential in the U.S for the treatment of NASH is about $2 billion.
The phase 2a NASH study was encouraging and the trial data which was positive was announced last May. A pre-IND meeting with the FDA for a phase 2b study is planned for the first half of 2011.
Huntington's Disease is yet another indication for which DR Cysteamine is being evaluated as a potential treatment. Huntington's Disease is a rare hereditary condition caused by a defective gene. This gene makes an abnormal protein which leads to the degeneration of certain nerve cells in the brain.
There is a high unmet need in the current Huntington's disease therapeutics market in terms of both safety and efficacy profile. According to market research firm GlobalData, the global Huntington's disease therapeutics market, which was valued at $142 million in 2009, is expected to increase to $2 billion by 2017.
The phase II trial of DR Cysteamine in Huntington's Disease was initiated last October and the enrollment is expected to be completed by the second quarter of this year.
Raptor's other clinical development programs include, phase II drug candidate Convivia for the potential management of acetaldehyde toxicity due to ALDH2 deficiency, an inherited metabolic disorder, and phase I candidate oral Tezampanel, a non-opioid glutamate receptor antagonist as a potential anti-platelet agent.
Uni Pharma Co., Ltd. has an exclusive license to commercialize Convivia in Taiwan. Raptor continues to seek potential partners in other Asian countries to continue clinical development of Convivia in those countries.
The company's preclinical product candidates include, receptor-associated protein, or RAP, platform consisting of HepTide for the potential treatment of primary liver cancer and other liver diseases; and NeuroTrans to potentially deliver therapeutics across the blood-brain barrier for treatment of a variety of neurological diseases (licensed to Hoffman - La Roche), and mesoderm development protein, or Mesd, platform consisting of WntTide for the potential treatment of breast cancer.
Being an early development stage company, Raptor has not generated any revenues to date, and as of February 28, 2011, had an accumulated deficit of approximately $53.9 million. According to the company, cash and cash equivalents as of March 31, 2011 of approximately $16.0 million will be sufficient to meet its obligations into the first calendar quarter of 2012.
In a SEC filing made on April 14, 2011, it has been revealed that Raptor plans to continue to review strategic partnerships, collaborations and potential equity sales as a potential means to fund its preclinical and clinical programs beyond the first calendar quarter of 2012.
RPTP has thus far hit a 52-week low of $1.41 and a 52-week high of $4.04. The stock closed Monday's trading at $3.44 - below the 50-day moving average of $3.48 but above the 20-day moving average of $3.39.
With several catalysts coming up in the near term, Raptor is a stock to keep an eye on. Stay tuned...
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June 05, 2026 16:18 ET A busy week for economic news flow saw a slew of reports being released that reflected the trends in the U.S. labor market. In Europe, economic growth and inflation data gained attention as the European Central Bank and Bank of England head for policy session later in the month. In Asia, the monetary policy session of the Indian central bank was in focus as the country, a major oil importer, reels under the pressures of a weaker rupee and rising inflation.