Structural Biology Expertise Supports Advancement of Three New Drug Candidates

This milestone brings the total count of Emerald’s collaborator INDs (investigational new drugs) to eight, with six of these in clinical trials, and makes Emerald BioStructures one of the most prolific structure-based drug design contract service organizations in the world.

Targeted drug design enables the progress of the most promising candidates for clinical development and increases chances of clinical success because it reveals molecules with desirable properties, such as high solubility, low molecular weight, and other drug-like qualities. “The Emerald team congratulates our colleagues at Deciphera, N30, and Families of Spinal Muscular Atrophy and its development partner RepliGen for their tremendous achievements to bring breakthrough new drug candidates into clinical trials. We feel privileged to have had the opportunity to provide high resolution structural insight to guide the development of these clinical-stage drug candidates,” said Lance Stewart, CEO of Emerald BioStructures.

The three drug candidates include: DCC-2036, being developed for the treatment of a resistant form of leukemia and in trials sponsored by Deciphera Pharmaceuticals and researchers at Tufts Medical Center, M.D. Anderson, and the University of Michigan; N6022, being developed for the treatment of asthma and in trials sponsored by N30 Pharmaceuticals LLC; and RG3039, being developed for the treatment of spinal muscular atrophy and in trials sponsored by RepliGen Corporation in cooperation with Families of Spinal Muscular Atrophy.

"The power in designing drugs based on structures of biological targets is that we’re able to rapidly discover novel therapies that affect the molecular mechanisms responsible for the disorder,” said Gary Rosenthal, Ph.D, Executive Vice President Research at N30 Pharmaceuticals. Their investigational new drug is the first GSNOR inhibitor to enter human clinical development. The March 2011 issue of American Chemical Society Medicinal Chemistry Letters documents this work in a paper titled, “Discovery of S-Nitrosoglutathione Reductase Inhibitors: Potential Agents for the Treatment of Asthma and Other Inflammatory Diseases.”

“The molecular targeting performed at Emerald allowed us to tailor our drug candidate DCC-2036 to the specific mutation that causes resistance to traditional therapies for this type of leukemia,” said Daniel Flynn, CEO of Deciphera. “Structure-based drug design is promising in its efficiency and Emerald offers best-in-class structural biology services.” The Deciphera drug candidate is being developed to treat individuals with leukemia who have developed a mutant enzyme that leads to a relapse that is resistant to all currently marketed therapies. DCC-2036 blocks this mutation at the molecular level. The work is documented in a paper titled, “Conformational Control Inhibition of the BCR-ABL1 Tyrosine Kinase, Including the Gatekeeper T315I Mutant, by the Switch-Control Inhibitor DCC-2036” in the April 2011 issue of the publication Cancer Cell.

“Emerald’s structural insights helped us to identify an important target called ‘DcpS‘ which plays a role in gene expression. Our drug candidate inhibits DcpS and improves the activity of a key gene involved in spinal muscular atrophy,” said Jill Jarecki, Ph.D., Research Director of Families of SMA. This drug candidate has the potential to treat SMA, which is a genetic disorder affecting about 1 in 6,000 births per year and is the leading genetic cause of death in children under the age of two. Emerald’s work on this program is documented in the peer-reviewed paper, “DcpS as a therapeutic target for spinal muscular atrophy.”