Deep dive into Actelion's Uptravi data calms investors as FDA decision date nears

Actelion (VTX:ATLN) has presented detailed data on the drug it hopes will cement its position in the pulmonary arterial hypertension (PAH) market, Uptravi. The shock-free data drove a small uptick in Actelion's stock, but fell short of being the blockbuster-guaranteeing release some were hoping to see.

Actelion CEO Jean-Paul Clozel

Basel, Switzerland-based Actelion presented the data at American College of Cardiology (ACC) this week, almost 9 months after it posted top-line results. The deeper dive into the data at ACC showed declines in disease progression and PAH-related hospitalizations underpinned the 40% drop in risk reduction that caused so much excitement back in June. Actelion also confirmed the fall in risk reduction was consistent across all subgroups.

When paired to safety data that were comparable to the placebo--although adverse event-related discontinuations were twice as common in the treatment arm--the results reassured analysts Uptravi is on track to win approval on both sides of the Atlantic in 2016. "Uptravi could be the first oral prostacyclin approved with a morbidity-mortality benefit and label allowing combination use. We forecast $1.4 billion worldwide peak sales," Jefferies analyst Peter Welford wrote in an investor note.

Welford downplayed issues that gave other analysts--notably Bank Sarasin's Chi Tran-Brändli--reason to question whether Uptravi is the drug to secure Actelion's position as a leader in the PAH market. Tran-Brändli picked out data on the 6-minute walk test (6MWT)--which physicians use as a surrogate assessment of efficacy--as a cause for concern, Neue Zürcher Zeitung reports. For Welford, the data were strong enough to for Uptravi to compete with United Therapeutics' ($UTHR) Orenitram.

The response of regulators and physicians to such details will dictate whether the mooted triple-therapy combination of Actelion's Uptravi and Opsumit with a phosphodiesterase type 5 inhibitor--such as generic Viagra--will win favor. Welford thinks early signs are good. "We were somewhat surprised at ACC by physicians' likely willingness to progressively adopt triple therapy as the standard-of-care for PAH patients," he wrote.

- read the abstract
- here's the release
- and Neue Zürcher Zeitung's article (German)

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