Insight Pharma Reports, a division of Cambridge Healthtech Institute

Blood-Brain Barrier: Bridging Options for Drug Discovery and Development
* Strategies to develop small- and large-molecule CNS drugs capable of crossing the blood-brain barrier (BBB)

* Interviews with leading researchers who are aggressively tackling the BBB challenge in CNS drug discovery and development

* Analysis of results from a Blood-Brain Barrier Survey, responded to by a range of companies involved in CNS research and drug discovery/development

CNS diseases are a major focus of the pharmaceutical industry, with CNS drugs representing some of its most successful products. These include Pfizer’s Zoloft (sertraline, for treatment of depression and certain types of anxiety disorders), Lilly’s Cymbalta (duloxetine, for treatment of depression) and Bristol-Myers Squibb’s/Otsuka’s Abilify (aripiprazole, for treatment of bipolar disorder and schizophrenia). However, drug discovery and development researchers experience difficulty developing CNS drugs that complete clinical trials and win regulatory approval—especially drugs which meet major unmet needs in the CNS area, such as Alzheimer’s disease. The vast majority of drugs fail to cross the BBB, which is causing a major bottleneck in successful development of CNS drug candidates.

Cardiotoxicity: Issues, Technologies, and Solutions for the Future
At least 50 companies have a claimed product or service relevant to cardiotoxicity screening, of which 29 have some clear focus on proarrhythmic cardiotoxicity or ion channel screening. This new report offers in-depth analysis of:

* 50 commercial entities that offer cardiotoxicity screening products/services

* The history and status quo of the current regulatory environment pertinent to drug-induced proarrhythmia

* Methods for assessing the potential for drug-induced cardiotoxicity, with a primary focus on proarrhythmia screening

* Drugs associated with cardiotoxicity, factors that may predispose to drug-induced cardiotoxicity, and current/proposed cardioprotective approaches

* A primer on cardiac anatomy/physiology, with particular consideration given to the various ion fluxes that contribute to the cardiac action potential

* Results of an Insight Pharma Reports cardiotoxicity survey undertaken for this report in December 2007

INFLAMMATORY DIRECTIONS Strategies for Six Diseases with Unmet Needs
inflammatory diseases. Delving in-depth into these efforts, this report provides:

* An analysis of six diseases: rheumatoid arthritis, inflammatory bowel disease, psoriasis, lupus, multiple sclerosis, and asthma

* An assessment of existing drug therapies for these diseases

* Discussion of pharmacological R&D strategies being employed by the industry in developing both biological and small-molecule agents for these diseases

* Review of the approximately 200 compounds in clinical development and assessment of particularly noteworthy drug candidates for these diseases

MicroRNAs Commercial Products on the Horizon
With the first diagnostics set to debut within a year, the new research and development field of microRNAs is beginning to reveal its potential. This new report establishes a baseline for observing microRNAs’ maturation, including assessments of:

* The science and analysis of first-generation microRNA commercial applications

* The early adaptors and where they are heading with this emerging technology

* Clinical applications, which will begin in oncology, followed by infectious diseases, neurology, metabolic disorders, and cardiovascular diseases

* The youthfulness of the field of microRNA

* The status of the field, including promises and caveats, companies involved, and pertinent patents and intellectual property

* Companies that are either developing microRNAs as clinical tools, investigating microRNAs in basic research, or supplying reagents, kits, microarrays, bioinformatics tools, and other essentials

Epigenetics Technologies, Applications, and the Commercial Landscape
Epigenetics is one of the fastest-growing areas of the biological sciences. It’s also a budding industry. This new report offers in-depth analysis of:

* The definition, history, and current status of epigenetics

* Current and potential commercial applications

* An epigenetic approach to cancer etiology

* The epigenetics of noncancerous diseases

* DNA methylation and histone modifications as biomarkers

* The companies involved and their products

* Epigenetic therapeutics: demethylating agents, histone modifications, and combinations

* The drivers of the epigenetics industry, plus recent major industry deals and patent applications

* Results of a quantitative online survey of individuals involved in epigenetics R&D

Multiplex Assays in Translational Medicine Technologies, Applications, and Future Directions
The development and growth of assay technologies has pushed translational medicine into a category unto itself. In a broad perspective on this field, this new report:

* Defines translational medicine by giving some historical background as well as providing personal definitions from experts in the field

* Discusses the evolution of assay technologies

* Reviews currently available assay technologies that apply directly to translational medicine

* Describes and evaluates current applications of these technologies

* Provides case studies of clinicians currently using this technology in their research

* Discusses future directions of assay technologies for translational medicine

* Gives input from the FDA on translation medicine and assay technologies

* Provides interviews from experts in the field of both translational medicine and specific assay technologies

* Profiles premier companies active in the field

Molecular Imaging in Drug R and D and Medical Practice Technologies, Applications, Markets
The rapidly emerging field of molecular imaging is poised to open new vistas for basic researchers, scientists working in drug discovery and development, and physicians. This new report delves fully into:

o The history, evolution, and potential of molecular imaging

o The technologies in use today and under development for the future

o Applications in drug development and diagnostic medicine

o Market dynamics that are shaping and reshaping commerce in molecular imaging instruments and reagents

o Regulatory aspects of the field

o Interviews with experts active as either providers or users of molecular imaging

o Results of an online survey of individuals in pharmaceutical and biotechnology companies and institutional researchers

Ophthalmological Therapeutics Pipelines, Delivery Technologies, and Markets
The ophthalmology market, although a relatively small niche in the pharmaceutical industry, is more than worthwhile to track for multiple reasons:

* It is growing dynamically, and will likely continue this development unbroken for the next 2 or 3 decades.

* It offers scientific challenges that are definitely tough but not insurmountable, with the paths to success already discernible.

* It is a market that analysts and investors can understand. Severe eye diseases are debilitating but not terminal conditions, and therefore most people know at least 1 person with severe vision impairment and can sympathize—which on the whole creates a more favorable basis for investments.

Kinase Therapeutic Pipelines: An Assessment of Targets and Agents in Development
Recent developments reflect the explosion in the number of kinase inhibitors that have entered clinical development in the past few years:

* By the end of 2006 seven kinase inhibitors had reached the market, three in the period December 2005-December 2006.

* Their collective sales exceeded $4 billion.

* Three more kinase inhibitors have been approved in 2007.

* In addition to ongoing studies of approved kinase inhibitors seeking line extensions, a further 11 are in Phase III studies.

* More than 130 kinase inhibitors are reported to be in either Phase I or Phase II clinical development, with 47 reported to be in Phase II studies.

Molecular Diagnostics: A Rapidly Shifting Commercial and Technology Landscape
Molecular diagnostic tests are now used for a wide range of applications, including:

* Human clinical molecular diagnostic testing

* Veterinary molecular diagnostic testing

* Identity testing

* Forensic testing

* Histocompatibility testing

Monoclonal Antibodies: Pipeline Analysis and Competitive Assessment
In the more than 30 years since the first process for creating monoclonal antibodies, or mAbs, was introduced, they have remained a centerpiece of the growing biotechnology industry. Twenty-four therapeutic mAbs have been approved, several of which have attained blockbuster status, with sales reaching the coveted billion-dollar mark and well beyond. Two drugs, Remicade and Rituxan, generated sales of about $4 billion each in 2006, and global sales for this entire portfolio approached $20 billion in that year.

Backup Compound Strategies: Best Practices for Reducing Phase II Risk
Backup compound planning has risen in importance as the percentage of R&D projects focused on unprecedented targets has skyrocketed. Heads of R&D and chemistry, therapeutic area heads, portfolio analysts, and project managers all recognize that there is no surer way to minimize Phase II risk than a well thought-out backup plan.

Adaptive Clinical Trials Innovations in Trial Design Management and Analysis
The pharma industry is gradually coming to realize that the classically structured clinical trial does not offer enough flexibility to make use of continuously emerging knowledge that is generated as the trial progresses. This report is a comprehensive assessment of the benefits, challenges, and accumulated industry experience with regard to adaptive clinical trials. It includes:

* A critique of the structural, conceptual, and ethical issues inherent in the traditional clinical trial.

* An in-depth review, based on actual case studies (e.g., Napo, Genaera, Pfizer, Lilly, Millennium, and various academic institutions) of the use of adaptive and Bayesian approaches in Phases I, II, and III

* An assessment of various hybrid and seamless designs in which the line between trial stages is blurred

* Evolving regulatory positions of FDA, EMEA, and ICH on adaptive designs; industry response and initiatives

* A review of specialized software vendors (e.g., Cytel, Tourtellotte, Pharsight, CTriSoft) and their applications that have emerged to support adaptive designs.

* A CHI Insight Pharma survey of the views and experiences of individuals involved with adaptive designs

* Three future scenarios for the integration of adaptive designs in clinical trials by 2015: (1) United States Leads the Way, (2) Globally Integrated Midphase Revamping, and (3) the Late Phoenix Scenario

* Roundtable interviews with senior executives in industry and consulting who bring decades of combined experience in adaptive and Bayesian clinical trial designs

Next-Generation Sequencing Scientific and Commercial Implications of the $1000 Genome
Current trends and future directions in genome sequencing

A growing number of companies and academic groups are reporting impressive progress in commercializing new sequencing platforms that offer orders of magnitude of improved throughput and cost. The prospect of routine, personalized genome sequencing is suddenly within reach, not to mention spectacular advances in a host of related fields. This new report from CHI Insight Pharma Reports describes and assesses these developing technologies and their applications:

* Pyrosequencing

* Sequencing by synthesis

* Supported oligonucleotide detection (SOLiD)

* Single-molecule sequencing

* Nanopore sequencing

* Optical trapping

Clinical Forecasting A Novel Bayesian Tool for Predicting Phase III Outcomes
In recent years, there has been an explosion in predictive technologies to help researchers select only the most promising candidates for clinical development. The need for such tools is driven by the disastrous economic consequences of late-stage failures, which account for over 60% of all drug terminations. This report describes a powerful and novel predictive tool called Bayesian network modeling and demonstrates its application in clinical forecasting. Among its many potential benefits, clinical forecasting can:

* Reduce drug development costs

* Increase median cumulative 7-year revenue per Phase III trial

* Redirect capital and human resources to development programs with the greatest likelihood of success

* Expose clinical trial subjects to fewer unsafe or ineffective drugs

* Improve the accuracy and decision-making utility of market forecasts (which currently assume that all drugs in the projection period will achieve NDA approval)

* Increase industry’s and society’s confidence in including pediatric subjects in clinical trials