The Galien Forum USA 2020

The Future of Therapeutics: What Novel Modalities Will Be Drugs? What Vaccines and Technologies Will We Need?

Medicines science today faces a challenge on two fronts:  how to develop and personalize a vast array of new insights into the biological origins of complex non-communicable diseases – many of which promise real cures – while fighting a resurgent viral pandemic where the ability to control its severity and spread remains little better than a century ago.  Deaths worldwide from COVID-19 are likely to reach 1.5m by the end of this year, a grim statistic that will put infectious diseases firmly back in the top ranks as a priority for drug, device and vaccines research for the duration of the decade.

Coping with these challenges will benefit from new platform technologies that rely on a systems approach to biology, which through use of computational and mathematical analysis allows for enhanced understanding of a more diverse array of cellular functions, leading in turn to a much larger number of druggable targets against disease.  Systems biology has also spurred the growth of a complementary process for drug development known as translational medicine, which seeks to leverage basic research through multidisciplinary collaborations between researchers and clinicians with the goal of quickly turning these druggable targets into therapeutic breakthroughs that work safely at the level of the individual patient.

Overall, the industry is moving from a “one disease-one target-one drug” approach to a more customized focus on novel bioactive compounds that combine multi-target properties in a single molecule or protein.   The strategy is squarely in line with the trajectory of unmet medical need, with complex conditions like cancer, stroke, neurodegenerative disorders, COPD and respiratory infections topping the World Health Organization (WHO) list of major killers to 2030.

In the drugs space, researchers will continue to build on learnings from the first wave of cellular and gene drug therapies, with approximately 20 such products now approved for clinical use; dozens more are in late stage trials or pending FDA approval.  A key area primed for future growth is allogeneic cell therapy, which seeks to expand the current process of extracting, modifying and replacing human cells with a much faster method based on manufacturing cells synthetically, in mass quantities. This would significantly expand the range and scope of these potentially curative treatments, assuming immune response complications can be resolved safely.   Cell therapy is also being applied against major chronic conditions like diabetes, where studies are underway on reversing the immune response in type one patients that results in the premature death or depletion of beta cells responsible for producing insulin in the pancreas.

Another example of the potential gains from today’s systems biology approach is in precision cardiology, where genomic data can be applied to stratify risk for individuals susceptible to coronary artery disease – currently, the number one killer worldwide – enabling health providers to save lives by catching a genetic predisposition early in life.  And in COVID-19, the potential exists to generate stem cells to replace damaged cells in the lung incurred by the acute respiratory distress syndrome that accounts for the pandemic’s high rate of mortality.

In vaccines, future therapeutic innovations are likely in delivery technologies and also in new vaccines to treat a host of conditions unrelated to communicable infections, like cancer. Surprisingly, 101 vaccines for therapeutic application are currently in development for various cancers, second only to the 137 underway to prevent infectious diseases.  In addition to a universal influenza vaccine, supported by the Bill & Melinda Gates Foundation, progress is pending on an adult vaccine for TB as well as one against HIV; one vaccine to confer immunity against the nearly two dozen strains of pneumococcal pneumonia; and protection for the respiratory syncytial virus, a persistent hazard in infants.

Likewise, the global race for a COVID-19 solution is propelling advances in vaccine design, such as the messenger mRNA technology which avoids using viral particles, thus making a mRNA vaccine both safer for patients and faster and cheaper to manufacture than the traditional live or attenuated virus model. Likewise, work is accelerating on novel non-needle vaccine delivery technologies like micro-patches that can be attached directly by the patient without specialist assistance. All these technologies are geared to making immunization cheaper, less cumbersome and more accessible in hard to reach areas with limited health resources.

The following group of experts, chaired by Pfizer Chief Scientific Officer and President, Worldwide Research, Development and Medical, Dr. Mikael Dolsten, will address the questions surrounding the future of therapeutics in detail, with emphasis on structural transformations in research discovery and development as well as the progress against current standard of clinical care, highlighting the real benefits to today’s – and tomorrow’s – patients.

Penny Heaton, Chief Executive Officer, Bill & Melinda Gates Medical Research Institute

Mads Krogsgaard Thomsen, Executive Vice President and Chief Scientific Officer, Novo Nordisk

John Maraganore, Chief Executive Officer, Alnylam Pharmaceuticals

Jeff Spaeder, MD, Global Chief Medical & Scientific Officer, IQVIA

George Scangos, Chief Executive Officer, Vir Biotechnology Inc.