Taking the distress out of ARDS

Apeiron and GSK sign an exclusive license agreement for Apeiron’s therapeutic enzyme project APN01

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

Under the terms of the agreement, Apeiron will receive anupfront payment of around $17 million in cash and equity investment and willreceive 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 witha variety of conditions, such as sepsis, aspiration of acidic gastric contents,trauma, postoperative complications, acute pancreatitis and pneumonias. Apeironreports that according to recent analyses, it affects millions of individualsworldwide each year—as many as one million people in Organisation for EconomicCo-operation and Development member countries alone. ARDS has a mortality rateof 30 percent to 50 percent and, as yet, no effective drugs exist to treat thislife-threatening condition.

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

"When I returned to Austria after several years of basicresearch in North America, I had the vision of translating some of my research,including that for ACE2, into clinically useful and commercially viableproducts," recalls Josef Penninger, Apeiron's founder. "For this purpose, Ifounded Apeiron and was able to attract excellent management. I am impressed tosee how quickly and successfully the basic idea was translated into a clinicalproject with such promising perspectives. GSK is a most welcome partner forfurther 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 throughsmall, back-end weighted licensing agreements."

"Although this is far from big money for GSK, the upfrontpayment exceeds the £10 million Apeiron has raised from Austrian and Europeangrants 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 companieseschewing venture capital funding.

"Remarkably, Apeiron achieved this major success [the dealwith GSK] without prior venture capital financing, but only with individualprivate investors," notes Manfred Reichl, chairman of the supervisory board andhimself an angel investor. "This proves that the biotech cluster in Vienna hascome 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 anew, standalone unit that will focus on the development and commercializationof medicines for rare diseases, as part of its strategy "to deliver moreproducts of value and improve returns in R&D through a focus on areas witha higher probability of success."

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

The leanly operated unit, according to GSK, involves twocollaborations it signed with specialist companies last year: Prosensa, whichfocuses on nucleic acid-based therapeutics, correcting gene expression indiseases with large unmet medical needs; and JCR Pharmaceuticals, a Japanesedeveloper and manufacturer of bioactive products, including enzyme replacementtherapies that could, upon approval, be used to treat orphan diseases such asHunter syndrome, Fabry disease and Gaucher disease.

"The risk associated with product discovery and development in rarediseases is generally lower than other disease areas as disease definitions arevery clear, and clinical trials tend to be small with robust endpoints, saysPatrick Vallance, GSK's senior vice president of drug discovery. "In mostcases, the molecular target is known, making it easier for specializedphysicians to diagnose patients." 

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