Senior Staff Writer

Licensing a pulmonary arterial hypertension program from a Swiss company, Biogen Idec Inc. has stepped into a new therapeutic area to address a frequently undiagnosed disease that may affect up to 200,000 people worldwide.

From Basel, Switzerland-based mondoBiotech AG, the company is getting U.S. and European rights to Aviptadil, a synthetically produced human peptide that has orphan drug status in both territories.

The deal calls for mondoBiotech to receive a $7.5 million up-front payment and up to $30 million in development and commercialization milestone payments for the PAH indication. In addition to royalties on commercial sales, the Swiss company also could receive a $5 million equity investment from Cambridge, Mass.-based Biogen Idec once it conducts an initial public offering.

Fabio Cavalli, mondoBiotech's CEO, classified the deal as his company's "biggest collaboration" to date, followed by a 2002 licensing agreement with InterMune Inc.

Biogen Idec has primarily focused its resources on the areas of oncology, neurology and immunology. Aviptadil represents a "recent move" toward a cardiovascular indication, said Jose Juves, the company's director of public affairs.

"We're looking at new therapeutic areas where we can leverage our global capabilities in serving specialty markets," Juves told BioWorld Today. "And we believe PAH is a good strategic fit really for two reasons. One, it is an area of high unmet need, and [two], it's served by a small, focused population of PAH experts."

Aviptadil is the first PAH drug candidate to receive parallel scientific advice for clinical development from the FDA and the European Medicines Agency - meaning the same clinical work may fulfill regulatory requirements of both territories. Juves said it's too early to say when those filings for approval might occur.

Biogen Idec is in charge of global manufacturing, clinical development, regulatory approval and commercialization of Aviptadil, and intends to start additional clinical work in 2007, probably "a larger Phase II trial," Juves said.

The inhaled product already has achieved its primary efficacy endpoint, showing an improvement in physical exercise as measured by an increased walking distance with three or six months of treatment in an open-label Phase II study. No significant adverse events were seen in the eight PAH patients that participated in the trial.

"The data from the Phase II trial looked promising," Juves said, later adding that the drug appeared to be "well tolerated, and the mechanism of action does not overlap with already approved oral therapies."

Cavalli noted that Aviptadil may be used in combination with other PAH therapies, as researchers are "ready to find a better solution" for the disease.

Aviptadil is a synthetically produced human hormone found in abundance within the human lung, but lacking in PAH patients. By replacing the hormone, the drug appears to reduce the increased pressure on the pulmonary arteries that characterizes PAH. Due to the pressure, patients who suffer from the disease experience a shortness of breath, fatigue, chest pain and eventually heart failure and death. PAH can be idiopathic or familial and often is secondary to other conditions such as scleroderma, congenital heart disease and HIV/AIDS.

The indication has challenged some drug development companies, such as Houston-based Encysive Pharmaceuticals Inc., which received a second approvable letter from the FDA in July for its PAH candidate, Thelin (sitaxsentan). The agency is working with the company to address an undisclosed issue that might require additional trials. Thelin received marketing approval in Europe last month. (See BioWorld Today, July 26, 2006.)

The current PAH market includes approved drugs such as Tracleer (bosentan, Actelion Ltd.) and Revatio (sildenafil, Pfizer Inc.). Tracleer, which is marketed for PAH in 22 countries, brought Allschwil, Switzerland-based Actelion sales of CHF417.9 million (US$335.3 million) for the first half of 2006. Cleared for marketing in the U.S. in November 2001, it was the first oral drug approved for PAH.

New York-based Pfizer received FDA approval in June 2005 for its oral drug Revatio, which uses the same active ingredient (sildenafil citrate) found in the company's erectile dysfunction drug, Viagra. And Myogen Inc., of Denver, is in Phase III development with its treatment for PAH, ambrisentan. United Therapeutics Corp., of Silver Springs, Md., also is working in the space, developing UT-15C, which it licensed from Rockville, Md.-based Supernus Pharmaceuticals Inc. The product is an oral formulation of treprostinil, the active ingredient in UTC's Remodulin, approved for subcutaneous and intravenous treatment of PAH. (See BioWorld Today, June 14, 2006.)

MondoBiotech, which was founded in 2001, has plans to go public in Zurich during "the first half of 2007," Cavalli said. A successful IPO would trigger Biogen Idec's $5 million equity investment in the company.

Aside from PAH, mondoBiotech is conducting clinical trials in pulmonary sarcoidosis and acute respiratory distress syndrome with Aviptadil. The company also has out-licensed rights to interferon gamma-1b to InterMune Inc., of Brisbane, Calif., for idiopathic pulmonary fibrosis and asthma.

Biogen Idec currently does not have an interest in the additional Aviptadil indications. "Right now, we're focused on PAH," Juves said.

Biogen Idec's stock (NASDAQ:BIIB) fell 28 cents Thursday to close at $43.39.