Emerald BioStructures’ Structural Biology Expertise Supports Advancement of Three New Drug Candidates to Clinical Developm

BAINBRIDGE ISLAND, Wash.--(BUSINESS WIRE)-- Emerald BioStructures, a leading provider of integrated structural biology services, today announced that protein crystal structures determined at Emerald BioStructures have supported the advancement of three drug candidates to clinical trials, for leukemia, asthma, and spinal muscular atrophy. 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.”

About Emerald BioStructures’ Structure-based Drug Design

The lead discovery program at Emerald BioStructures generates drug-like compounds via a high-throughput pipeline for rapidly generating ligand-protein co-crystal X-ray structures. The process integrates X-ray crystallography, NMR, SPR, and computational modeling to clients’ target proteins and small molecule ligands. Emerald’s structural data and collaborative approach have proven to be extremely valuable in helping our clients identify successful drug candidates.

About Emerald BioStructures

Emerald BioStructures is an integrated gene-to-structure contract research organization that provides collaborative drug discovery services to pharmaceutical companies, biotechnology companies, academic institutions, and government facilities. The company operates a high-throughput platform leveraged for fragment-based lead discovery and structure-based drug design. Emerald’s work provides a solid foundation for the discovery of highly selective, efficacious drugs. www.emeraldbiostructures.com

About Deciphera Pharmaceuticals

Deciphera Pharmaceuticals, LLC was established in 2003 as a drug discovery and development company with the mission to design, optimize and introduce “best-in-class” small molecule Switch Inhibitors of protein kinases for human clinical trials and the global pharmaceutical marketplace through the use of its proprietary drug discovery technology platform, Phylomechanics. www.deciphera.com

About N30 Pharmaceuticals

N30 Pharma is a privately held biopharmaceutical company headquartered in Boulder, Colorado. N30 Pharma focuses on the discovery and development of proprietary drugs that provide therapeutic advantage over current treatment for major human diseases such as asthma, COPD and IBD. The Company's strategy is to focus upon discovery research and early-stage clinical development, and then establish one or more partnerships with leading pharmaceutical companies to further the advancement of its lead clinical candidates. www.n30pharma.com

About Families of Spinal Muscular Atrophy

Families of SMA is a non-profit 501(c)3 tax exempt organization with 30 Chapters throughout the United States and over 70,000 members and supporters. Families of SMA funds and directs the leading SMA research programs. The successful results and progress from basic research to drug discovery programs to clinical trials provide real hope for families and patients. www.curesma.org


For Emerald BioStructures:
Lance Stewart, 206-780-8911
[email protected]
For Media:
MacDougall Biomedical Communications
Douglas MacDougall or Jennifer Conrad, 781-235-3060

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