Actelion might have a shot at breaking into the market for new oral drugs against immune-related disorders. The Swiss biotech ($ATLN) scored positive efficacy data from a midstage trial with patients suffering from plaque psoriasis on its candidate ponesimod. And the company intends to follow up the results in planned late-stage trials.
The 326-patient study nailed its primary goal, with 46% of patients on a 20-mg dose of the oral compound achieving at least a 75% improvement on an index to rate the severity of the skin disease at 16 weeks. Also, 48.1% of patients reached the same goal on a 40-mg dose. Those numbers trumped the 13.4% improvement in patients on placebo.
Actelion appears to have notched another R&D victory with ponesimod, building on the impressive late-stage study results earlier this year with macitentan in patients with pulmonary arterial hypertension (PAH). Macitentan is under FDA review and holds the key to Actelion's future in the competitive market for drugs against PAH, for which its aging blockbuster Tracleer is used.
The biotech faces plenty of competition in the psoriasis arena too. Ponesimod--a selective S1P1 modulator--has now produced positive efficacy results in midstage trials with psoriasis and multiple sclerosis patients. (The market for MS pills has already stoked a rivalry among Novartis ($NVS), Sanofi ($SNY) and Biogen Idec ($BIIB).) Amgen ($AMGN), however, has shown better evidence of efficacy in a midstage trial for its injected drug brodalumab in psoriasis patients, Vontobel analysis Andrew Weiss noted, as quoted by Reuters.
"However, a strength of Actelion's compound is the oral route of administration," Weiss pointed out in the Reuters report.
On the safety front ponesimod's profile was similar to results from a midstage study in MS patients, according to the company. The most common side effects in the psoriasis study were shortness of breath and increased liver enzymes classified as "asymptomatic."
Actelion has had one of the largest R&D budgets among global biotechs as CEO Jean-Paul Clozel has invested big in broadening the scope of the business beyond Tracleer. He made a point to note in the company's release this morning that ponesimod came from his company's discovery work and seems to have a shot at treating multiple immune disorders.
"Actelion will rapidly move forward with the preparation of the pivotal program and discussions with health authorities for the psoriasis indication," Clozel said in a statement.
- here's Actelion's release
- see the Reuters article
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