Inspire Pharmaceuticals, Inc. - About Development

Development Mission Statement
To meet the unmet medical need; together we investigate, persevere, and succeed.

PRECLINICAL DEVELOPMENT -The study of compounds for a specific therapeutic application that have not yet been tested in humans, but are actively being studied in tissue or animal models.

Organization
After a molecule is determined to be an appropriate product candidate based upon our business strategy, it moves into the development phase of our organization, where extensive testing of both the characteristics of the actual product and the effects it has on humans are conducted. The progression of products through the various stages of development is overseen by the Portfolio Management Board, a group comprised of selected company officers and senior staff.

The Development team works closely with the Research and Preclinical Development team to ensure a smooth and efficient transition to clinical development. Inspire utilizes a project team system, with senior-level project leaders driving for aggressive, high-quality development plans and timelines for every high priority project. Inspire's Portfolio Management Board meets regularly with project leaders to review projects, provide needed support, determine allocation of resources across the teams, and provide strategic direction.

The two functional areas in Development at Inspire are clinical research and biostatistics & data management. The clinical research function is further divided by therapeutic area, with teams dedicated to ophthalmology and respiratory/allergy. Each functional area lends specific technical and strategic expertise to the clinical development of new treatments. All areas work in concert to investigate the potential benefits and risks of new treatments. The process is regulated by national and international regulatory agencies that provide specific guidance on ensuring that the rights of human subjects have been protected as well as all research studies have been conducted appropriately.

There are four phases of clinical research that occur in sequential order: Phase 1, Phase 2, Phase 3, and Phase 4. Information from the earlier phases is used to advance the study of the investigational drug through these phases. At the end of Phase 3, there generally is enough accumulated information to prepare and submit an application to market the investigational drug.

Phase 1 studies are the first studies that evaluate the use of the investigational compound in humans. These studies are generally done across a wide range of potential doses to determine the safety and tolerability of the investigational drug. These studies are not designed to determine the efficacy of the treatment. Generally, the first Phase 1 studies are conducted in <80 healthy subjects who do not have the disease target; however, this is not necessarily the case.

Phase 2 studies (sometimes referred to as proof of concept studies) are the first studies in which patients with the disease of interest are studied. The objective of these studies is to evaluate the safety and tolerability of the investigational drug and to assess whether the drug has the desired clinical effect (benefit) or efficacy. Usually, these studies evaluate a range of doses in a limited number of patients (<300) who take the drug for a limited period of time.

Phase 3 studies are the definitive studies of the safety and efficacy of the investigational drug that primarily support the applications to market the new drug. Phase 3 studies are larger and more expanded versions of Phase 2 studies. By design, these studies are less restrictive than Phase 2 studies and include a wider variety of patients taking a wide variety of other medications. The duration of treatment and the size of the studies is highly variable, depending on the expected use of the medication and the endpoints that are being evaluated.

Phase 4 studies are generally conducted after the drug has been approved for marketing. Phase 4 studies are designed to investigate other potential uses of the drug or to compare the drug to other available treatments. Additionally, Phase 4 studies are also conducted to obtain specific information from selected subgroups of patients or to gain additional safety experience in a huge number of patients.

Page last updated: 10-May-2007