HOERSHOLM, Denmark—
Santaris Pharma, a clinical-stage company focused on the discovery and development of RNA-targeted therapies, and biopharmaceutical firm
miRagen Therapeutics have joined forces to develop a new class of drugs targeted toward treating cardiovascular disease, the number one cause of death worldwide, claiming 17.1 million lives a year, or 29 percent of all deaths globally.
Under the strategic alliance, announced June 23, Boulder, Colo.-based miRagen will use Santaris Pharma's proprietary locked nucleic acid (LNA) technology to select drug candidates against miRagen's proprietary microRNA targets for the treatment of cardiovascular disease.
In exchange, Danish firm Santaris has received an undisclosed minority interest in miRagen, and stands to receive milestones and royalties related on LNA-base medicines developed as part of the collaboration. Financial terms of the alliance were not disclosed.
miRagen, a leader in developing microRNA-based therapeutics for cardiovascular and muscle disease, is developing single-stranded, LNA-based drug candidates for the treatment of patients with cardiovascular and muscle disease.
"We are pleased that miRagen selected Santaris Pharma A/S as its preferred partner, further validating that our LNA drug platform is the technology-of-choice for developing RNA-targeted medicines," says Soren Tulstrup, Santaris Pharma's president and CEO. "This collaboration is a prime example of two companies leveraging their unique capabilities in developing RNA-targeted medicines and combined expertise in cardiovascular disease to develop new medicines for life-threatening diseases. Santaris Pharma looks forward to adding more alliances with biotechnology companies to its growing list of partners."
Navjot Rai, Santaris' director of global communications, says, "This alliance—the first between the two companies—leverages the unique capabilities of two industry leaders in developing RNA-targeted medicines to treat a range of diseases, including metabolic disorders, infectious and inflammatory diseases, cancer and rare genetic disorders."
"The LNA Drug Platform overcomes the limitations of earlier antisense and siRNA technologies to deliver potent, single-stranded LNA-based drug candidates across a multitude of disease states," Rai says. "The unique combination of small size and very high affinity, which is only achievable with LNA-based drugs, allows this new class of drugs to potently and specifically inhibit RNA targets in many different tissues without the need for complex delivery vehicles. LNA-based drugs are a promising new type of therapy that enables scientists to develop drugs to attack previously inaccessible clinical pathways."
The most important features of LNA-based drugs include excellent specificity, providing optimal targeting; increased affinity to targets providing improved potency; and strong pharmacology upon systemic delivery without complicated delivery vehicles, Rai says.
"Cardiovascular disease represents an enormous burden on global healthcare systems," says William S. Marshall, president and CEO of miRagenTherapeutics. "Principally funded through venture capital investments, miRagen combines world-class leadership in cardiovascular medicine with unprecedented in-house expertise in microRNA biology and chemistry.
"There are several reasons this strategic partnership was attractive to us," William says. "First, Santaris' LNA drug platform provides us with rights to what we view is the best chemistry to develop miRagen's microRNA-targeted therapies. Secondly, this enhances our ability to advance our programs forward."
The fact that the LNA chemistry has been tested in a host of non-clinical safety studies—and is currently being dosed in human clinical trials—also provides a level of validation that is important, he points out.
"Our goal is utilize the LNA drug platform to advance our preclinical programs forward," Marshall says. "We have several programs in preclinical development today, in a variety of cardiovascular and muscle indications. We anticipate that we could enter the clinic with our first cardiovascular indication during the second half of 2011."
MiRagen will participate in a joint research committee, and Santaris Pharma will provide technical expertise and consultation in connection with research and early development activities utilizing the LNA drug platform, he says.
"MicroRNAs have emerged as an important class of small RNAs encoded in the genome," Marshall says. "They act to control the expression of sets of genes and entire pathways and are thus thought of as master regulators of gene expression."
Recent studies have demonstrated that microRNAs are associated with many disease processes, he says. Because they are single molecular entities that dictate the expression of fundamental regulatory pathways, microRNAs represent potential drug targets for controlling many biologic and disease processes.
Like Rai, Marshall is hopeful about the potential of LNA-based drugs, based on their unique combination of small size and very high affinity, and looks forward to their ability to potently and specifically inhibit RNA targets in many different tissues without the need for complex delivery vehicles. All of this, Marhall reiterates, will give scientists the power to go after clinical pathways that they had little or no hope of attacking before.
