As preparations get underway for transition from a development stage company to a commercial organization, it's time to take a look at the upcoming events at Chelsea Therapeutics International Ltd. (CHTP), a biopharmaceutical development company. Chelsea shares rose as much as 21% in intraday trading on Friday after the company reported new data from a late-stage trial of its most advanced drug candidate, Northera, also known as Droxidopa.
Northera is an orally active synthetic precursor of norepinephrine that is currently in phase III clinical trials for the treatment of symptomatic neurogenic orthostatic hypotension, a blood pressure disorder, prevalent in patients with Parkinson's disease. According to Chelsea, about 300,000 patients suffer from chronic, symptomatic neurogenic orthostatic hypotension in the US and EU.
Though there is no FDA-approved treatment specifically for neurogenic orthostatic hypotension, Shire Pharmaceuticals' Midodrine, approved in 1996, is currently the only FDA-approved therapeutic for the treatment of orthostatic hypotension. However, the drug carries a blackbox warning for the side effect of supine hypertension, along with the statement that Midodrine has not shown benefit to patients' Activities of Daily Living, or symptomatic/functional benefit. Last August, the FDA proposed pulling Midodrine off the market unless two studies to confirm the symptomatic benefit of the drug are successfully completed within a specified timeframe. Mylan Pharmaceuticals, Eon Labs and Impax Laboratories are generic manufacturers of Midodrine.
Neurogenic orthostatic hypotension, or NOH for short, results from a deficient release of norepinephrine, the neurotransmitter used by autonomic nerves to send signals to the blood vessels and the heart. NOH is characterized by a chronic and often debilitating drop in blood pressure upon standing, lightheadedness, dizziness, blurred vision and syncope (fainting).
During 2007, Northera was granted orphan drug status by the FDA for the treatment of symptomatic NOH and was granted orphan medicinal product designation by the European Medicines Agency, or EMA, for the treatment of patients with Pure Autonomic Failure, or PAF, and patients with multiple system atrophy, or MSA.
In Japan, Northera has been approved since 1989 and is marketed by Dainippon Sumitomo Pharma Co., Ltd., for the treatment of symptomatic orthostatic hypotension, freezing of gait in Parkinson's disease and intradialytic hypotension, or IDH.
The phase III trials dubbed 301 and 302, which evaluated Northera, showed that there was a significant improvement in the symptoms of NOH associated with the drug candidate. Northera has also been found to be both safe and very well tolerated in the studies.
A long-term safety extension study of Northera known as Study 303, the results of which were reported last May, has also demonstrated that Northera provides sustained improvements in both blood pressure and symptoms of NOH.
In June 2010, the company initiated a pivotal phase III trial dubbed Study 306, a confirmatory study to support the efficacy of Northera in treating symptomatic NOH associated with Parkinson's disease. With data from an interim analysis of the Study 306, suggesting an extraordinary benefit for the prevention of falls, one of the most common, devastating and costly health risks faced by Parkinson's disease patients, the company modified the study as 306a and 306b in February 2011.
The new data from the Study 306A released last week demonstrated that Northera was associated with a 60% reduction in falls and 52% reduction in falls-related injuries.
The company is in the process of preparing to file a New Drug Application for Northera for the treatment of symptomatic NOH in the third quarter based on combined data from its two previously completed phase III efficacy studies in NOH, Study 301 and Study 302. The company also plans to file a supplemental new drug application intended to expand the future labeling of Northera in the U.S. to include the prevention of falls in NOH associated with Parkinson's disease, based on the results from 306A and 306B studies. Data from the 306B study is anticipated by the second quarter of 2012.
Chelsea is also studying Droxidopa, alone and in combination with Carbidopa, for the treatment of fibromyalgia in a phase II trial. The company initiated a phase II study of Droxidopa in combination with carbidopa in adult attention deficit hyperactivity disorder last February and a phase II study of Droxidopa for the treatment of chronic fatigue syndrome last August.
And now about the other drugs in the company's pipeline - CH-1504 and CH-4051...
Both CH-1504 and CH-4051 are orally available molecules with anti-inflammatory, autoimmune and anti-tumor properties that potently inhibit dihydrofolate reductase, an enzyme required for cell proliferation. These compounds are said to find potential use in treating rheumatoid arthritis, psoriasis, Crohn's disease, ankylosing spondylitis, uveitus, psoriatic arthritis and several different kinds of cancer.
The company reported positive results from a phase II study of CH-1504 in rheumatoid arthritis in March 2009. Additional trials or further investments are not going to be made in the development of CH-1504. But that said, data related to CH-1504 is expected to support the development of additional compounds in Chelsea's portfolio of novel antifolate compounds.
The second compound in the family of novel antifolate compounds is CH-4051, which is being evaluated in a phase II trial for the treatment of rheumatoid arthritis. The 12-week phase II trial of CH-4051 designed to compare the efficacy and tolerability of CH-4051 against Methotrexate in 250 patients with rheumatoid arthritis who are experiencing an inadequate response to Methotrexate treatment was initiated in September 2010. Methotrexate is considered the standard of care in rheumatoid arthritis, both as a monotherapy and in combination with other rheumatoid arthritis treatments.
The trial is designed to have 5 arms - with patients being randomized to receive 0.3 mg, 1.0 mg or 3.0 mg of CH-4051 daily, 3.0 mg of CH-4051 daily in combination with a folate supplement or 20 mg Methotrexate weekly with a folate supplement for 12 weeks following a two-week Methotrexate-washout.
Last month, an independent Data Safety Monitoring Board, or DSMB, reviewed patient safety data in both the 0.3 mg and 1.0 mg CH-4051 cohorts of the phase II trial in rheumatoid arthritis, and recommended the study be continued as planned. The DSMB has also recommended enrollment to be initiated into the 3.0 mg dose groups of the phase II trial of CH-4051.
Chelsea is on track to report blinded interim efficacy data from the phase II study of CH-4051 in the third quarter of 2011. The full results from the trial are expected to be reported during the second quarter of 2012.
A quick look at Chelsea's balance sheet...
Being a development stage company, Chelsea has sustained operating losses and has generated no revenue since inception. As of March 31, 2011, the company had an accumulated deficit of $146.7 million, working capital of roughly $68 million, including cash and cash equivalents of about $37.9 million, short-term investments of $45.1 million and liabilities of $16.1 million. About $40.3 million was raised in a public offering in February 2011.
The capital resources available at March 31, 2011 are expected to last through the second quarter of 2012, sufficient to meet the commercialization activity and expenses related to the anticipated market launch of Northera in the United States.
Shares of Chelsea have thus far hit a 52-week low of $2.73 and a 52-week high of $8.30. The stock closed Friday's trading at $4.75, up 13.10% on a volume of 4.18 million shares.
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