It might have involved moving the goal posts, but Chelsea Therapeutics has finally got the results it wanted from a pivotal trial of lead product Northera, a therapy to treat dangerously low blood pressure (Event - Chelsea hopes new droxidopa trial stands tall, August 24, 2010).
Shares in the company surged by 25% to $6.30 in early trade today, in response to the positive data, and are now trading at their highest level for nearly a year. With enough cash to last only into the first quarter of next year Chelsea could well swiftly capitalise on this healthy gain to top up its coffers, via a share sale.
Earlier today Chelsea said a trial called 301 met its primary endpoint, significantly improving symptoms associated with neurogenic orthostatic hypotension (NOH). This nervous system disorder is associated with low levels of the neurotransmitter norepinephrine, which causes low blood pressure, dizziness, weakness, problems concentrating and fainting episodes after standing up. It is most commonly associated with Parkinson’s disease.
The situation is the exact opposite of one year ago when a previous phase III study flopped, sending Chelsea’s stock plummeting (Chelsea data causes shares to fall over, September 28, 2009).
The failure sent Chelsea back to the drawing board to come up with a new study design for 301, in an attempt to eliminate the strong placebo effect that appeared to derail the previous trial. The primary endpoint was switched from a measure of dizziness to a relative mean change in results of the orthostatic hypotension questionnaire (OHQ), and at the same time the trial was expanded to recruit more people.
In a decision that surprised many analysts at the time, the FDA allowed this trial design change when the 301 study was already fully recruited. Still, the strong top-line results from the 301 study suggest this strategy has paid dividends.
Further pivotal data is still to come from Northera, generically known as droxidopa, a drug already sold for low blood pressure in Japan.
A phase III study conducted solely in Parkinson’s disease patients called 306 is due to report in the second quarter, and with this patient group representing the biggest target market for Northera these results are important. Chelsea today said that the efficacy was seen in all patient types in the 301 study – patients with multiple system atrophy and autonomic failure can also suffer NOH – meaning the omens for 306 are looking good.
Earlier next year data in adults with ADHD are also due.
Analysts are pencilling in sales of Northera, which has orphan drug status, starting in late 2011 and climbing to $193m by 2016, according to consensus data from EvaluatePharma.
Seeking a partner
NOH is a niche indication – Chelsea has estimated that 52,000 prescriptions might be written a year in the US – but for a company of this size, this is a decent franchise.
Whether the company will sell the drug itself, however, is another question.
Chelsea has said it will seek a partner outside the US and consider offers within its home territory. Should the data reading out over the next six months continue to be positive, interested parties should not be too hard to find.