Needs for New Medicines and Greater Pharmaceutical R&D Effectiveness

Over the last three decades, pharmaceutical R&D and the great medicines that have resulted from it have brought unprecedented benefits to most people in developed countries. It would not be an exaggeration to say that baby boomers can expect an extra decade of high-quality life based on the fruits of pharma R&D.

Today, however, the pharma industry is under a virtual state of siege. Successfully breaking that siege depends on increasing R&D productivity and success. For a host of converging reasons, success has been elusive. With a few notable exceptions, the great new medicines expected to result from the "-omics" revolution have yet to materialize. More information has not been matched by more knowledge. Enormous sums are being spent on pharma R&D every year and the number of major medicines emerging has been disappointingly low. Societal pressures on pricing and the emergence of high quality generics for many major illnesses puts additional pressures on costly R&D. The diseases for which we seek treatments are progressively more difficult to address, requiring extremely safe drugs and long, costly development campaigns. These challenging diseases include many which are accompaniments of excessive lifestyles and / or the aging process,such as loss of cognitive function, osteoarthritis, atherosclerosis, "diabesity", cancer, Chronic Obstructive Pulmonary Disease, frailty, etc.

Just scaling up the way in which pharma R&D has been performed will not address the challenge. Better ways of realizing the promise of the molecular revolution in medical biology are key to creating the medicines of the future.

The megatrend over the last three decades has been to rely more and more heavily on molecular targets and "deconstructed" systems (studying isolated parts such as proteins or cells) in the quest for new medicines. This has had remarkable benefits, but not without costs. It is now possible to much more efficiently optimize small molecules directed against specific targets. However, the effects of these compounds when studied in animals are often significantly different from what had been hoped. This reflects the influence of the complex, interconnected, often redundant, and highly homeostatic (resistant to change) nature of the biological and biochemical processes in living organisms. The whole often differs from the sum of the parts.

Furthermore, men are not mice; rodents and people last shared a common ancestor when it cowered and hid from dinosaur predators (between 65 and 85 million years ago)! Furthermore, human diseases are far more complex and variable than even the best animal models of disease. No wonder drugs that work wonderfully in pre-clinical studies don't always perform as hoped in human clinical trials or in medical practice. Rather than trying to glean more and more information from more and more preclinical work, it makes great sense to quickly get novel drugs into humans to determine how likely they are likely to produce desired benefits.

How Kinemed Helps Meet These Challenges

KineMed is centrally positioned to accomplish this, since we can provide high quality information about whether or not a particular drug is "working" in man much more quickly and cheaply than alternative approaches.

KineMed's platform offers unique insights into health, disease, and drug action that are potentially game changing for pharma R&D. We do this by providing high quality, decision-relevant information about potential medicines earlier and for less money than is otherwise possible, addressing one of the most vexing aspects of pharma R&D.

One of the most difficult aspects of the drug discovery and development process is the deciding which potential medicines to advance and which to sideline in the face of  incomplete information. These decisions have costly, time-consuming, and potentially opportunity-squandering implications. The absence of timely decision-relevant information is one of the key reasons for the disturbingly high attrition rates which typify current pharma R&D, even in the best companies. This has become apparent to the leadership of all high quality pharmaceutical companies, driving a progressive interest in "Target Validation", "Translational Medicine" and "biomarkers"; they are understandably seeking ways to increase the probability that the conclusions drawn in the pre-clinical phases of R&D are borne out in the clinical phases of development and medical use and to find ways to determine earlier in the costly process whether or not they are on the right track with a particular potential medicine.

This is where KineMed shines! KineMed's founders foresaw this need and have spent the past decade creating, road-testing, and refining, and ultimately validating (largely in collaboration with and funded by a virtual consortium of pharma companies) state-of-the-art approaches to addressing these very issues: identifying quick, relatively inexpensive, and highly informative ways to determine which drugs are future winners and which are future failures. KineMed has done this for drugs acting in a wide range of therapy areas, especially in diseases of the nervous system, cardiovascular and metabolic systems, and connective tissues. KineMed's insights can help determine the answers to two questions key to R&D success earlier and with great accuracy: (1) is the drug directed against a "good" target? and (2) is the drug a "good" compound? The answers powerfully guide the decisions whether or not to progress a specific compound and at what doses and in which patients to achieve what outcomes. Furthermore, the answers can greatly strengthen research activities by validating or disqualifying novel targets earlier in the development process. By allowing better decision making about resource allocations and approaches across a portfolio of compounds, KineMed can have a surprisingly large positive impact on the rate of emergence of best-in-class medicines and dramatically change the productivity of pharmaceutical R&D. This is the heart of the strategic opportunity which KineMed so powerfully addresses.