Actelion drug cuts brain lesions in MS patients

Actelion's experimental multiple sclerosis drug ponesimod met its main goal in a mid-stage trial, giving the Swiss biotech's pipeline a much-needed boost.

Actelion has had a bumpy ride after a string of product setbacks last year, and it has faced tough criticism from its main shareholder Elliott Advisors, which urged the group to consider putting itself up for sale.

Europe's largest pure-play biotech group said on Tuesday its selective S1P1 receptor agonist, ponesimod, cut the number of new active inflammatory lesions in the brain in a Phase IIb dose-finding study in patients with relapsing-remitting multiple sclerosis.

At 0856 GMT, Actelion shares were down 0.3 percent, outperforming a 1.2 percent drop in the European healthcare index.

"The product, assuming competitive efficacy, has a profile that would position it well in the market and as such we believe it has blockbuster potential," Nomura analyst Samir Devani said.

Actelion said 464 patients were enrolled, making this the largest ever dose-finding study conducted in this autoimmune disorder of the central nervous system.

Actelion said it would now move ponesimod to the final stage of clinical development, and analysts at Jefferies said the mid-stage data could pave the way for a partnership deal.

"Given the number of emerging oral MS drugs, together with Actelion's relative inexperience conducting large pivotal CNS trials and finite financial resources, we believe a partnership deal for ponesimod could be announced within the next 6-12 months now that the Phase IIb data are in-house," the Jefferies analysts said.

Novartis has seen strong demand for its recently launched its MS pill Gilenya, which rivals older treatments such as Biogen Idec's Tysabri.

Biogen is now developing its own oral MS drug, BG-12, which, if approved, could help it to take the lead in the oral MS drug market. Teva Pharmaceutical Industries is also developing experimental pill laquinimod.

Actelion is trying to cut its dependence on key drug Tracleer, which treats a rare heart and lung disorder and rakes in more than $1 billion a year, but is now starting to face competition from Gilead Sciences' Letairis.

Investors are now awaiting data from a phase II trial of macitentan in lung fibrosis for more insight into its safety profile. Macitentan is being billed as Tracleer's successor, and results of a late-stage study in pulmonary arterial hypertension (PAH) are due in the first half of 2012.