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Taking the distress out of ARDS
March 2010
by Jeffrey Bouley  |  Email the author


VIENNA, Austria—Apeiron Biologics AG in early February announced the signing of an agreement granting GlaxoSmithKline (GSK) exclusive rights to APN01 (recombinant human angiotensin converting enzyme 2, rhACE2), an enzyme biotherapeutic currently in Phase I development for the treatment of the acute respiratory distress syndrome (ARDS).

Under the terms of the agreement, Apeiron will receive an upfront payment of around $17 million in cash and equity investment and will receive royalties on net sales. Total milestone payments could reach more than $320 million in the event of launch in multiple indications.

ARDS is a severe injury to the lungs that is associated with a variety of conditions, such as sepsis, aspiration of acidic gastric contents, trauma, postoperative complications, acute pancreatitis and pneumonias. Apeiron reports that according to recent analyses, it affects millions of individuals worldwide each year—as many as one million people in Organisation for Economic Co-operation and Development member countries alone. ARDS has a mortality rate of 30 percent to 50 percent and, as yet, no effective drugs exist to treat this life-threatening condition.

"We are very pleased that we could attract GSK, one of the world's top pharmaceutical companies, to license this exciting project," says Hans Loibner, CEO of Apeiron. "I am convinced that Apeiron's know-how in this area, together with the extensive development and marketing capabilities of GSK, are the best way forward to bring an innovative therapy to patients worldwide."

"When I returned to Austria after several years of basic research in North America, I had the vision of translating some of my research, including that for ACE2, into clinically useful and commercially viable products," recalls Josef Penninger, Apeiron's founder. "For this purpose, I founded Apeiron and was able to attract excellent management. I am impressed to see how quickly and successfully the basic idea was translated into a clinical project with such promising perspectives. GSK is a most welcome partner for further development."

Three days after the announcement, the In Vivo Blog listed the deal as one of its Deals of the Week, noting that "GSK continues to access early stage innovative programs through small, back-end weighted licensing agreements."

"Although this is far from big money for GSK, the upfront payment exceeds the £10 million Apeiron has raised from Austrian and European grants and angel backers," notes Melanie Senior on the blog.

Apeiron is quick to point out, as did the In Vivo Blog, that it is one of the growing number of companies eschewing venture capital funding.

"Remarkably, Apeiron achieved this major success [the deal with GSK] without prior venture capital financing, but only with individual private investors," notes Manfred Reichl, chairman of the supervisory board and himself an angel investor. "This proves that the biotech cluster in Vienna has come a long way and is catching up to global standards in research quality, management capabilities and financing."

GSK launches R&D unit for rare diseases

LONDON—GSK also announced in February that it has formed a new, standalone unit that will focus on the development and commercialization of medicines for rare diseases, as part of its strategy "to deliver more products of value and improve returns in R&D through a focus on areas with a higher probability of success."

GSK said it will leverage existing capabilities and partnerships to establish further in-licensing opportunities related to more than 5,500 rare diseases, of which less than 10 percent are currently being treated. Many of these diseases are genetic in origin, start in childhood and cause lifelong debility and premature death, GSK said.

The leanly operated unit, according to GSK, involves two collaborations it signed with specialist companies last year: Prosensa, which focuses on nucleic acid-based therapeutics, correcting gene expression in diseases with large unmet medical needs; and JCR Pharmaceuticals, a Japanese developer and manufacturer of bioactive products, including enzyme replacement therapies that could, upon approval, be used to treat orphan diseases such as Hunter syndrome, Fabry disease and Gaucher disease.

"The risk associated with product discovery and development in rare diseases is generally lower than other disease areas as disease definitions are very clear, and clinical trials tend to be small with robust endpoints, says Patrick Vallance, GSK's senior vice president of drug discovery. "In most cases, the molecular target is known, making it easier for specialized physicians to diagnose patients." 
Code: E031005



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