Table of Contents
regulatory approval. BI is responsible for the manufacture of API and fill-and-finish services for both 11b HSD1 and BACE.
of our product candidates are small molecules and are manufactured in reproducible synthetic processes from readily available starting materials. We believe the chemistry is scalable
and will not require unusual equipment in the manufacturing process. We expect to continue to develop product candidates that can be produced cost-effectively at contract manufacturing facilities.
For certain of our unpartnered product candidates, we expect to retain U.S. commercial rights in specialty markets and establish
regional partnerships to commercialize outside the United States. We have not established sales, marketing or product distribution operations because our most advanced product candidates are still in
preclinical or early clinical development. We currently do not have any sales or marketing experience. We plan to establish the required capabilities within an appropriate time frame ahead of any
product approval and commercialization to support a product launch. As per our agreements with BI, we expect them to commence the commercialization activities with their sales and marketing
organization upon regulatory approval for both of the licensed programs.
Government Regulation and Product Approval
Governmental authorities in the United States, at the federal, state and local level, and analogous authorities in other countries
extensively regulate, among other things, the research, development, testing, manufacture, safety surveillance, efficacy, quality control, labeling, packaging, distribution, record keeping, promotion,
storage, advertising, distribution, marketing, sale, export and import, and the reporting of safety and other post-market information of products such as those we are developing. Our product
candidates must be approved by the FDA through the New Drug Application, or NDA, process before they may be legally promoted in the United States and by the European Medicines Agency through the
Marketing Authorization Application, MAA, process before they may be legally marketed in Europe. Our product candidates will be subject to similar requirements in other countries outside of Europe and
the United States prior to marketing in those countries. The process of obtaining regulatory approvals and the subsequent compliance with applicable federal, state, local and foreign statutes and
regulations require the expenditure of substantial time and resources.
United States Government Regulation
In the United States, the FDA regulates drugs under the Federal Food, Drug, and Cosmetic Act, and the FDA's implementing regulations.
An applicant seeking approval to market and distribute a new drug product in the United States must typically undertake the following:
- completion of preclinical laboratory tests, animal studies and formulation studies in compliance with the FDA's good
laboratory practice regulations;
- submission to the FDA of an IND which must take effect before clinical trials may begin;
- approval by an independent institutional review board, or IRB, representing each clinical site before each clinical trial
may be initiated;
- performance of adequate and well-controlled clinical trials in accordance with good clinical practice, or GCP, regulations
to establish the safety and efficacy of the proposed drug product for each indication;
- preparation and submission to the FDA of an NDA;
- review of the product by an FDA advisory committee, where appropriate or if applicable;
- satisfactory completion of one or more FDA inspections of the manufacturing facility or facilities at which the product or
its components are produced to assess compliance with current good