Thursday, biopharmaceutical company Chelsea Therapeutics (CHTP) announced top-line results from the first of two late-stage trials of Droxidopa for the treatment of symptomatic neurogenic orthostatic hypotension, stating that the study did not meet statistical significance on primary endpoint. Shares of the company plunged more than 65% in regular trading hours.
Symptomatic NOH is a neurogenic disorder resulting from a deficient release of norepinephrine, the neurotransmitter used by sympathetic autonomic nerves to send signals to the blood vessels and the heart, thereby resulting in decreased blood pressure when a person assumes a standing position. The disorder is characterized by dizziness, weakness, blurred vision and fatigue.
Droxidopa, an orally active synthetic precursor of norepinephrine, increases the supply of norepinephrine available for delivery to its receptors to improve orthostatic blood pressure and alleviate symptoms of orthostatic hypotension.
Chelsea noted that while Study 302 showed that Droxidopa showed a strong symptomatic benefit during the trial, a preliminary review of the data indicates it did not show a statistically significant improvement relative to placebo, as measured by the mean score of Item 1, which includes dizziness or light-headedness, of Orthostatic Hypotension Symptom Assessment, or OHSA, during the double-blind phase of trial, which is the study's primary endpoint.
Droxidopa was found to be safe and well tolerated at all dose levels, with no significant adverse events or treatment related withdrawals in the Droxidopa arm.
"While the outcome on Item 1 of the OHSA scale did not meet the company's expectations, our preliminary look at each of the secondary symptomatic outcome measures was encouraging and supportive of the therapeutic benefit of Droxidopa in neurogenic orthostatic hypotension," said president and chief executive officer Simon Pedder. "In addition, the benefits of Droxidopa, as measured by both BP and item 1 of the OHSA scale, appeared to persist to some extent despite absence of therapy, raising potential questions regarding the suitability of this type of trial design for an NOH study."
The study was a double-blind, placebo controlled withdrawal-design study in which all patients underwent an initial open-label dose titration. The 101 patients enrolled in the blinded study had a mean score on Item 1 of the OHSA scale of 2.1 at randomization.
At the end of the 14-day blinded treatment period, patients in placebo arm had an average OHSA score of 4.0, or a mean change, or increase, of 1.9 units from randomization. Patients in Droxidopa arm of the trial had a mean score of 3.5 at the end of the two-week treatment period, reflecting a mean change from randomization of 1.3 units resulting in a 0.6 unit difference between arms.
Chelsea noted that its preliminary analysis showed that nearly every secondary symptomatic endpoint in the trial was either supportive or strongly supportive of Droxidopa's therapeutic benefit, including standing short time and long-time, and also the composite orthostatic hypotension activities of daily living composite score all of which were statistically significantly in favor of Droxidopa.
Chelsea said it remains hopeful that the results of the study may provide a better opportunity to clearly demonstrate the efficacy of Droxidopa in the indication.
CHTP is currently trading at $2.42 per share, down 65.53%, on the Nasdaq.
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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.