Roche snaps up Santaris

BASEL, Switzerland—Back in January, Roche and Copenhagen, Denmark-based Santaris Pharma A/S signed a license agreement to discover and develop novel medicines, a deal that has since been concluded. Roche apparently liked what it saw, because the pharmaceutical giant has now announced plans to acquire Santaris in a deal that could be worth up to $450 million.

“Today there are many disease targets that are very challenging or even impossible to reach with small molecules or antibodies,” John C. Reed, head of Roche Pharma Research and Early Development, noted in a news release. “We believe the LNA platform provides the means to efficiently discover and develop an important new class of medicines that may address the significant needs of patients across multiple therapeutic areas.”

Per the terms of the transaction, Roche will pay $250 million up front to Santaris Pharma shareholders, with the potential for additional contingent payments of up to $200 million if certain predetermined milestones are met. Following the close of the acquisition, which is subject to customary closing conditions, Roche plans to maintain Santaris Pharma’s Denmark operations, though the site will be renamed Roche Innovation Center Copenhagen.

“Roche and Santaris Pharma have complementary capabilities that will help us realize breakthrough medicines,” J. Donald deBethizy, president and CEO of Santaris, commented in a statement. “The acquisition combines Santaris Pharma’s next-generation antisense technology and LNA expertise with Roche’s deep experience in disease biology, chemistry, drug safety, drug formulation, delivery and development.”

Roche and Santaris Pharma’s January license agreement was focused on using Santaris’ proprietary Locked Nucleic Acid (LNA) platform for the discovery and development of novel RNA-targeted medicines in multiple disease areas, and Santaris received an upfront payment of $10 million under the deal. The company announced a similar agreement the next day with GlaxoSmithKline as well, which Dr. Henrik Ørum, Santaris’ chief scientific officer and vice president of business development, noted was “the sixth partner deal we have concluded in the past 12 months with pharmaceutical and biotech companies.”

“The acquisition of Santaris provides the unique opportunity to develop new Roche molecules by merging our deep expertise in disease biology, target selection, toxicology, formulation and drug delivery with the most extensive experience in designing and producing LNA oligonucleotides, a technology that we believe will redefine RNA-targeting medicine in the future,” says Stepan Kracala, a media spokesperson for Roche Communications. “RNA-targeting medicines represent a new therapeutic modality, an emerging class of medicines, designed to access disease targets in the body that cannot be approached by traditional small molecules, antibodies or other therapeutic proteins. RNA-targeting medicines continue to be an emerging field that may address unmet patient need, R&D productivity and overall development speed.”

Traditional approaches to RNA-targeted therapies, Santaris Pharma explains on its website, consist of single-stranded approaches (generally known as antisense) and double-stranded approaches (generally referred to as siRNA). These both have their own issues, however, as “a significant limitation of antisense has been low affinity for its RNA target, leading to insufficient potency and narrow therapeutic index in animals and humans. The number one disadvantage with siRNA compounds has been their relatively large molecule size, leading to poor cellular uptake and the need for complex delivery vehicles that can sometimes be associated with toxicity.”

The LNA platform combines Santaris Pharma’s proprietary LNA chemistry with its targeted drug discovery capabilities to rapidly provide drug candidates against both mRNA and microRNA. The platform is meant to bypass the limitations of previous antisense and siRNA technologies, offering small size, high binding affinity and metabolic stability to create a new class of drug candidates that can potently and specifically affect RNA targets in a variety of different tissues without complex delivery vehicles.

“LNA-based medicines represent a new technology that has overcome the limitations of earlier siRNA programs,” Kracala explains. “LNA technology is particularly exciting because it allows for an emerging class of drug candidates that potently and specifically bind and inhibit RNA targets in many different tissues without the need for complex delivery vehicles.”

Roche expects this technology to have potential in a number of areas, says Kracala, who notes that “Without the need for a complicated delivery mechanism, LNA-based medicines open new opportunities to address previously undruggable targets and may finally offer solutions for patients across many of our therapeutic areas, including oncology, ophthalmology, neurosciences, infectious diseases and rare diseases.”

The Santaris Pharma deal was, at the time of the announcement, the second for the Roche group in as many months, as the company announced in July that Genentech would be acquiring Seragon Pharmaceuticals Inc. in a transaction that could be worth $1.725 billion all told. As noted in our article “A deep breath for an $8.3-billion plunge” (also part of the September issue with this article), of course, the planned acquisition of InterMune makes that a trio of deals.