Five Bold Biomedical Innovations to Watch in 2017
New stories emerge daily in the biotechnology sector. Established large pharmaceutical companies are in need of new products, while ambitious small companies require funding to complete product development, fostering numerous collaborations along the way. New clinical trials continually drive the pharmaceutical market, and new regulatory measures propel some companies to success while causing the downfall of others. Such news is abundant and commonplace.
It is particularly intriguing to observe those research-oriented entrepreneurs who have just secured substantial funding. They are striving to transform the core of a certain study into something commercially valuable, or they are driven by the vision of integrating multiple technologies to spark new chemical reactions. In either case, they are personally undertaking risks to push the healthcare industry forward. These individuals typically possess extraordinary insight and groundbreaking ideas, qualities that place them years ahead of some pharmaceutical giants.
Based on past experience, the failure rate of ambitious ideas from small companies is extremely high. Nevertheless, even in failure, they have to some extent pointed out certain directions for the development of the pharmaceutical industry. By summarizing the development trends of biopharmaceutical startups, we have listed below the five areas most likely to spark heated discussion in this field over the coming year.
Alzheimer’s disease is the greatest burden that an aging global population will face. A U.S. study estimates that by 2040, Alzheimer’s patients in the United States will account for one-quarter of the nation’s healthcare expenditures, while the pharmaceutical industry has yet to develop an effective treatment for the disease. The pharmaceutical sector has been investing billions of dollars in researching targeted antibody drugs designed to prevent the formation of beta-amyloid plaques, which are the primary cause of memory and cognitive decline in Alzheimer’s disease.
In addition, other companies are seeking alternative solutions. Yumanity Therapeutics has developed a novel drug discovery engine for Alzheimer’s disease and other neurodegenerative disorders, securing $45 million in funding. Annexon Biosciences, a spin-off from Stanford University, raised $44 million to investigate how to prevent the immune system from eliminating synapses that are essential for neuronal function, thereby aiming to prevent Alzheimer’s disease. Earlier this month, the startup EIP Pharma achieved a remarkable breakthrough: they successfully improved cognition and memory in several patients with mild Alzheimer’s disease using an older anti-inflammatory medication.
The urgent need, the vast market, and the successive failures in the development of anti-amyloid antibody drugs (such as Eli Lilly’s solanezumab) will compel the pharmaceutical industry to reflect and adopt broader approaches to treating neurodegenerative diseases over the coming year.
Chemists aim to design compounds that bind to specific targets, a field known as targeted drug research. Such studies are largely based on static images of molecular drug targets; however, since real-world targets are dynamic, this approach introduces significant inaccuracies. By employing a “movie-like” dynamic visualization to observe the various conformations of protein targets, researchers can gain a more comprehensive understanding of their characteristics.
Two Boston-based companies, Relay Therapeutics and Morphic Therapeutic, have each secured over $50 million in Series A financing. Both are investigating how to better capture the dynamics of certain protein targets to develop more targeted therapies. This approach has also attracted significant attention from major pharmaceutical companies; for instance, Morphic Therapeutic has received funding from Pfizer, GlaxoSmithKline (GSK), and AbbVie, while Relay Therapeutics was founded by Third Rock Ventures and D. E. Shaw Research.
Many people criticize pharmaceutical companies not only for high drug prices but also because some firms are raking in profits while their products fail to address health issues effectively. Certain prescription smoking-cessation products, such as Pfizer’s varenicline tartrate (Chantix) and GlaxoSmithKline’s bupropion hydrochloride (Zyban), have been shown in clinical trials to lack any miraculous efficacy in guaranteeing smoking cessation. While these companies focus intently on promoting their new drugs, researchers emphasize that pharmacotherapy alone is unrealistic; effective smoking cessation requires combining medication with voluntary behavioral changes.
A San Francisco Bay Area startup, Chrono Therapeutics, is researching a comprehensive approach to help people quit smoking by combining medication, digital sensors, and human coaches in a multi-pronged “siege.” This method offers valuable insights for the treatment of various diseases and the cultivation of healthy habits. If Chrono truly succeeds in helping people quit smoking, the outcomes will be far superior to merely prescribing medication and sending patients home to take it on their own.
In recent years, microbiology has swept through the biological sciences community. This is largely attributable to advances in genetic testing tools, which have deepened our understanding of the trillions of gut and skin bacteria residing within the human body. A harmonious relationship with these microbes generally signifies good health; conversely, microbial imbalances can disrupt immune function, leading to autoimmune diseases. Research has also demonstrated a correlation between gut microbiota composition and mental state or mood, opening up intriguing new avenues for treating depression and other central nervous system and psychiatric disorders. The next step will depend on how biotechnology companies and pharmaceutical firms translate microbiome research into concrete drug development efforts.
Seres Therapeutics, a pioneer in the microbiome field, experienced a sharp decline in its stock price following the announcement of a failed clinical trial for Clostridioides difficile infection earlier this year. Nevertheless, capital continues to flow into various microbiome-focused pharmaceutical companies, such as Vedanta Biosciences, Synlogic, and Second Genome. VCBeat has previously reported on Axial Biotherapeutics’ $19.15 million financing round; the company specializes in studying the impact of the microbiome on the “gut-brain axis,” leveraging this approach to develop treatments for autism and Parkinson’s disease.
In many cases, new drugs or therapies emerge through the following pathway: a university makes a novel biomedical discovery and files for a patent; then, companies license the patent from the university to conduct further research. Generally, substantial investment and experimentation are required to transform these early-stage patents into mature products. As universities become increasingly familiar with integrated error-proofing and lean production standards in the pharmaceutical manufacturing process, they are also playing a more active role in subsequent drug development research.
For example, in March 2016, Merck & Co. paid a $20 million upfront fee to acquire from Harvard University the research rights to a small-molecule drug for the treatment of acute myeloid leukemia. Another examples are Juno Therapeutics and Spark Therapeutics, both major players in the fields of cancer immunotherapy and gene therapy, whose foundational technologies were based on intellectual property licensed from external sources.
In the course of interactions between academia and industry, conflicts of interest are becoming increasingly apparent. Nevertheless, since the United States enacted the Bayh-Dole Act in 1980 to encourage the translation of scientific research from laboratories into commercial applications, universities have gradually been striking a balance in technology transfer. It is anticipated that in the coming year, more collaborations will emerge between research institutions and enterprises, enabling academia to secure more favorable deals.