April 20, 2024

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[THE INVESTOR] Crystal Genomics said on Dec. 27 that its leukemia therapy has received orphan drug designation from the US Food and Drug Administration which will spur development of the lead candidate.

CG026806 is a non-covalent small molecule inhibitor of Bruton’s tyrosine kinase and FMS-like tyrosine kinase 3 to treat patients with acute myeloid leukemia, a type of cancer that starts in the blood-forming cells of the bone marrow. The drug is currently in pre-clinical development in partnership with Canada-based Aptose Bioscience. 

Crytal Genomics CEO Cho Joong-myung

“With the orphan status, we can cut the development period of the drug by about two years compared to non-designated therapies,” a Crystal Genomics official said. “We will continue to work closely with Aptose Bioscience for its successful development.”

Under a 360 billion won (US$334.97 million) worth licensing deal in June 2016, Aptose Bioscience is undertaking Investigational New Drug enabling studies and expects to exercise its option to develop and commercialize CG026806 under the agreement and initiate phase 1 clinical trials by mid-2018.

“The results from non-clinical studies that we and our research collaborators have generated are promising and give reason for our eagerness to begin clinical trials in both acute myeloid leukemia and B-Cell malignancies in 2018,” Aptose Bioscience CEO William G. Rice.

According to the Korean company, Aptose Biosceince secured sufficient funds for CG026806’s clinical programs as it entered into a US$15.5 million common share purchase agreement with Aspire Capital Fund on Oct. 30.

In November, Crystal Genomics filed a wide range of patents involving CG026806 in China, which claimed the CG026806 compound, pharmaceutical compositions, and methods of treating various diseases. The patent will provide protection to Crystal Genomics’ rights to the potential drug in the case of out-licensing deals in Korea and China. The patent expires on Dec. 26, 2033.

Acute myeloid leukemia is the most common type of acute leukemia among adults, with an annual incidence of approximately 21,000 patients and causing more than 10,000 deaths each year in the US.

Orphan Drug Designation provides seven years of market exclusivity if the drug candidate receives regulatory approval, together with tax credits for qualified clinical trial costs, exemptions from certain FDA application fees, and assistance in design of clinical trials.

By Park Han-na (hnpark@heraldcorp.com)