TAIPEI, Taiwan – Taiwan's government-funded Development Center for Biotechnology (DCB) said this week that its mTOR inhibitor cancer drug candidate, DCBCI0901, developed in-house and licensed out in 2011 to an alliance of four local pharmaceutical companies, received approval from the FDA and the TFDA to begin phase I trials.
The center, together with members of that alliance – Standard Chemical & Pharmaceutical Co. Ltd., of Tainan; Yungshin Global Holding Corp., of Taichung; China Chemical & Pharmaceutical Co., of Taipei; and Intech Biopharm, a subsidiary of Synmosa Biopharma Corporation, also of Taipei – and other research partners, including National Taiwan University Medical School, had spent the last four years completing toxicology, process R&D and other preparatory tasks getting the drug ready for trial approval in humans. The phase I trial under the authority of both the FDA and TFDA, Taiwan's drug regulatory body, will be conducted at the medical centers of some of Taiwan's top hospitals, including National Taiwan University Hospital, National Cheng Kung University Hospital, Taipei General Hospital and others.
MTOR is involved in cell signaling pathways that control cell proliferation. Inhibiting those pathways can suppress the growth of cancer cells or even activate apoptosis. The drug candidate developed by the center is considered a next-generation mTOR inhibitor, targeting both the mTORC1 and mTORC2 complexes, as well as a third target, PI3K. Additionally, studies undertaken at the National Taiwan University Medical School revealed that DCBCI0901 also showed significant inhibitory activity on drug-resistant lung cancer cells.
With mTOR inhibitors targeting solid cancer types, the alliance has lung, breast and prostate cancers in its sights for future clinical trials.
The drug initially was developed at the Institute of Pharmaceutics, the DCB's small-molecule drug development unit, explained DCB vice president Kuo Mei-hui. "While this unit has successfully developed several botanic drugs through to FDA IND [investigational new drug application] approval, DCBCI0901 is the first small-molecule drug candidate to receive such approval," Kuo said.
Patents for the drug have been filed and secured in major markets, including Taiwan, Japan, the U.S. and Europe, 16 countries in total.
The forming of the alliance was first announced in May 2011, and was unusual for Taiwan in that the DCB wasn't licensing the drug to one company but to four. The stated reason was to spread the benefit and risk as widely as possible and try to lift the local pharma sector as a whole rather than just allowing one company to receive all the future benefit.
"The alliance will share responsibility for all aspects of the drug's development, allocating responsibility for API manufacture, dosage formulation and so on, amongst themselves," Kuo added.
While the DCB picked up the reported NT$250 million (US$8.3 million) tab in preclinical development costs, while also receiving an up-front licensing fee of NT$25 million from the four companies, clinical development costs going forward will be the responsibility of the alliance. In recent years, Taiwan's Ministry of Economic Affairs has been generous with grants supporting clinical trials for locally developed drugs, even those in-lincensed from abroad, and grant applications already filed by the alliance look likely to be approved.
The market for cancer drugs is huge, with 14 million diagnosed cases worldwide every year and global sales of cancer drugs totaling $61.6 billion in 2012 and projected to be between $74 billion to $84 billion by 2017, according to figures cited by DCB.
While this was a joint announcement made by both DCB and the alliance, the drug is fully in the hands of the alliance from here, according to the center, and it will be up to the member companies whether to continue with further clinical trials or license the drug out to another company for its future development.