Q: What’s the next “big thing” on the horizon that will significantly alter the pharma/biopharma market, your operations, or the way you do business?

The Next "Big Thing" in the Pharma/Biopharma Market

Dago Caceres, Global Strategy Director, Pharma Solutions, IFF

A: The digitalization of the pharma market is advancing at a speed and scale that has exceeded my expectations. It has reached just about every critical element of the industry — from the way pharma companies conduct clinical trials and R&D work to improvements in quality management and post-marketing monitoring. I’m also seeing more companies implement artificial intelligence–based technology platforms regardless of their size, business model, or position in the value chain.

An important digital trend — that has been exacerbated by the pandemic — is using advanced tools to increase the resilience and predictability of the complex pharma supply chain. For the Pharma Solutions division at IFF, we’re diving right into the digital era with our manufacturing facilities to increase the robustness and consistency of our products. Digitalization not only helps us to optimize production processes but also enhances our product offerings overall. We’re utilizing innovative technologies and advanced statistical techniques for data mining, predictive modeling, and process optimization. With advanced analytical tools, we can tap into the robust data generated during production processes. This data offers new insights to address variability in the manufacturing chain — resulting in more stable processes, fewer supply disruptions ,and greater batch-to-batch consistency, all within strict validated parameters.

Sandra Wisniewski, President, Materials Technologies and Reno Nguyen, Principal Scientist, W. R. Grace & Co.

A: From the perspective of some of our pharma customers, mRNA will be the biggest focus for the next generation vaccines for influenza, yellow fever, HIV, etc.; and new therapies for cystic fibrosis, multiple sclerosis, and different types of cancer.  Of course, mRNA technology cannot work without lipids, which stabilize and ensure delivery of the nucleic acid molecules within human cells. Grace chromatographic silicas will become increasingly important for the purification of the key lipids. 

With major investment in our manufacturing facilities, Grace is well prepared to meet an increased demand for silica gel. Grace also is working on technologies for the purification of mRNA, and we are committed to bolstering collaborations with pharma companies.

Sergey Vlasenko, Ph.D., Associate Vice President Pharma and Biopharma End Markets, Agilent Technologies 

A: Another “big thing” that is happing in the industry is the prioritization of investments into new drug modalities, focusing on disease prevention and curative therapies. For example, the novel mRNA technology platform that enabled the industry to pass the test of rapid pandemic response with flying colors is now being investigated for use in the potential prevention of diseases ranging from difficult-to-eradicate pathogens to cancer. This new mRNA technology platform will likely lead to breakthroughs in gene therapy offering a once-in-a-lifetime correction of defective genes by delivering CRISPR-Cas9 coding mRNA and guide RNA packaged into lipid nanoparticles to target organs.

At the enterprise level, pharma/biopharma companies will also need to continue to invest in programs targeting increased productivity and reduced costs, with digital transformation programs taking central stage. These programs will range from broader adoption of artificial intelligence and machine learning in novel drug design to investments into lab automation and instrument connectivity leading to intelligent end-to-end workflow solutions. The digital transformation of pharma/biopharma lab will improve R&D, operations, business processes, and other enterprise functions.

Kurt Nielsen, President and Chief Executive Officer, Pharmaceutics International, Inc. (Pii)

A: I think the next big thing in healthcare is the integration of the normalization of digital tools to analyze and share ever increasing amounts of data. Not only from integrated and connected  be electronic health records, but also digital delivery of care like telemedicine. In U.S. healthcare, the next big thing is a quantum leap in accessibility to healthcare that is driven by these digital breakthroughs. The digitization of data and the pervasive use of mobile devices, including video calls, makes it easier for people to get the advice and information they need when they need it to take care of themselves better and ultimately prevent disease from impacting their lives — not just providing treatments when needed.

The next big thing for Pii is contributing to the COVID-19 supply chain. This is a massive global health challenge and, given the transmission rates and frequency of new variants emerging, it’s going to be around for a long time. Vaccination rates outside the U.S. are woefully small — less than 1% — which adds to the challenges ahead for the global community. There are 11 billion doses of vaccine needed outside the United States. Pii has an opportunity to be part of that global supply chain to provide initial and booster vaccinations to millions of people.

The other “next big thing” for Pii is onshoring of the pharmaceutical supply chain back to the U.S. It’s been moving east for over 40 years: Europe to India to China. The COVID pandemic has highlighted the need for governments across the globe, including the U.S., to re-evaluate the long, complex supply chains that were put in place to take cost out of the system. That strategy worked when the increasingly globalizing supply chain was serving near-term profit demands in the retail and institutional distribution channels. However, with the rise of populism globally and in the U.S., as well as the democratization of healthcare in the U.S., the U.S. Government has stepped in to provide grants, loans, and contracts to many healthcare businesses to build and revitalize infrastructure in the U.S. But when you talk about providing care to larger numbers of people, it’s a different motive than simply profit. In order to further democratize healthcare, infrastructure is needed onshore: accessible, modern, adaptable, flexible, and ready to perform here in the U.S.

Mark Parry, Technical Director, Intertek Melbourn

A: One of the next big things which is pertinent to us is the increased momentum of inhaled or nasal drug development for delivery of vaccines and complex drugs, including peptides, monoclonal antibodies, oligonucleotides, mRNA, and gene therapies. The potential for patient-friendly drug delivery and new therapeutic pathways for diseases such as cystic fibrosis, asthma and lung cancer, coupled with the advantages presented both by targeted delivery to the lung and systemic delivery for other diseases or treatment pathways, are driving this increased attention. The size of a biologic, susceptibility to stresses and impact on stability, aerodynamic properties, and bioavailability all present challenges. We are already seeing significant drive to develop and innovate both formulation and device design and we anticipate this continuing to be primary focus for us and our development services.

Solution- and suspension-based sprays are inexpensive; however, some devices do not tolerate freeze–thaw cycles, which raises fill-finish concerns. Reconstitution of drug product close to administration or careful device screening and design can circumvent that problem and can also address drug product stability challenges where lyophilization can be used to extend the shelf life. Pressurized metered-dose inhalers (pMDIs) present significant formulation challenges for biologics, and so the focus is primarily on aqueous and solid formulations for pulmonary delivery where particle engineering must be carefully considered for solid or suspension-based products. A nebulizer solution does not require significant particle engineering and is a common target for development for early clinical trials, acute hospital-based treatment regimes, as well as the treatment of pediatric, elderly, ventilated, and sedated patients.

Brian Keesee, Vice President & General Manager, Global Operations and Supply, Clinical Services, PCI Pharma Services

A: There has been a significant increase in the implementation of technology within the pharma and biopharma industries. Wearables have become more popular among consumers, because they help individuals track their daily physical activity and overall health. These tech products have also allowed healthcare professionals to monitor their patients’ vitals and health at home, which has proven extremely beneficial during the pandemic.

I also predict that the U.S. population will expect greater convenience with their drug supply delivery, as more companies make their products and solutions accessible at home. As a society, we are now accustomed to quick and easy at-home delivery for almost everything we need, so it is likely the industry will hear more requests from patients for the “Amazon Prime effect” regarding their prescriptions and lifesaving drugs. Similarly, our industry’s recruitment and talent process will change completely due to remote work. This will lead to more diverse talent pools, as well as more fluid and accelerated advancements than the industry has ever seen.

Manny Soman, Chief Executive Officer, Verista

A: The pandemic has changed our minds and reality. The new normal will be that drugs and devices will be brought to market faster. It used to take 10 years to take a molecule from R&D to manufacturing. Today, that won’t fly, and mankind won’t wait. The concepts of what we’ve traditionally thought of as product velocity and speed-to-market will be shattered. To meet these new demands, organizations must alter their traditional operational and business models.

The main issue facing this industry is: How can facilities be produced faster and to regulatory standards without compromising on quality, safety and efficacy? Even prior to the pandemic, organizations were struggling with speed-to-market from R&D to manufacturing. The key to the puzzle is a well-governed, transparent, and collaborative model that supports dynamic and effective decision-making.

Organizations are looking to end-to-end service providers like Verista to manage, automate, and validate their data, systems, and operations to ensure they comply with regulations, all while facilitating faster and more informed decision making. To achieve this, there is a push toward digital transformation. Legacy processes and systems are being replaced with streamlined digital solutions that enable data to get from R&D and the manufacturing shop floor to decision makers in record time. For example, Verista helped a top 5 pharma client implement Veeva Vault as their regulatory information management systems (RIMS), resulting in faster time to submission. By evaluating and streamlining their processes, they can now complete their fully compliant dossiers faster, meaning their molecules get to market faster too.

Saharsh Davuluri, Vice Chairman and Managing Director, Neuland Laboratories Limited

A: The trend, which has been apparent for some time but which will become more significant, is the rise of targeted therapies leading to a greater number of products coming to the market — though each product by itself is not likely to be very significant in terms of size. We will have to be able to manage more projects while optimizing operations to ensure agility and capability to manage a number of products.

Melissa Sherman, Ph.D., Chief Executive Officer, MOBILion Systems Inc.

A: Traditionally, the treatment of diseases has been led by small molecule drugs, but there’s a mega trend of shifting to large molecule drugs, which are more complex and used to treat a wider range of diseases. Because of this and the rise of new therapeutic systems (in the form of personalized medicines like cell and gene therapies and antibody engineering) across the U.S. pharma market, there is a growing need for traditional analytical tools to enable higher and faster characterization of these more convoluted drug systems and drug products. Right now, the tools available are not meeting the needs of these sophisticated systems.

That’s why high-resolution technologies like our HRIM instrument MOBIE are so important. With so much pressure to diagnose and treat diseases, researchers need to be able to quickly and easily detect and analyze molecules that are indicative of disease and explore biologic systems that reach far beyond genomics. However, to do that, they need systems capable of superior sensitivity to detect even minute traces of many disease-specific biomolecules, and that’s really what’s driving our R&D efforts.

Ultimately, by providing technology that reduces analysis times from days and hours to minutes and seconds, we’re helping researchers to accelerate the process of bringing safer and more effective drugs to market.

Alex Dunlap, Business Development Manager, Lab Services, Alcami

A: As COVID continues to change the markets and the future of healthcare, I believe that more large molecule products will become the forefront of drug product development, calling for advancements in testing, equipment, and environmental controls.

To support the increasing demand for biologics, Alcami is staying ahead of the curve with continued investment in biotechnology testing by renovating and expanding our biologics infrastructure. Approximately 5,200 ft2 of lab space and 40 scientists are being added to our existing footprint and headcount in North Carolina.  Beyond established capabilities in high-resolution mass spectrometry, ELISA, cell-based bioassays, capillary isoelectric focusing (cIEF), and glycan analysis, Alcami will be introducing new instrumentation and capabilities in support of DS/DP characterization in early-phase development.

The expanded footprint will incorporate three different laboratories; one focusing on chromatography, one housing a wide range of mass spectrometry equipment for characterization and sequencing, and a bioassay lab with a separate cell culture room.

David Levy, Global Product Director, Scitara Corporation

A: Pharma 4.0 was born out of the need for a bespoke approach in the regulated pharmaceutical industry to adopting Industry 4.0 and represents the inevitable next step in its evolution. The success of an organization's digital transformation strategy depends on the volume, accuracy, and quality of the information available in its digital systems.

The foundation of a digital transformation strategy lies in bringing together the vast amounts of data generated by scientific research and development. The goal is to gather, store and retrieve all data in a correct, structured, and useable format — all while maintaining regulatory compliance — and therefore unlock meaningful insights from that data. The potential of this connected approach to enable faster and more accurate decision-making in the drug discovery and development process, and so bring new drugs to market more quickly, is recognized across the pharmaceutical industry.

Within the lab, a significant barrier to success is the lack of proper infrastructure that connects all data sources across a very diverse environment. The “next big thing,” therefore, is a universal digital data connectivity solution unencumbered by legacy systems, data siloes, or manual processes that connects any instrument, resource, app, and software in the lab. Enabling the free flow of data in this way will allow the development of reliable data models that ultimately provide scientists with the insights they need to inform better research outcomes.

Offering a new solution to this decades-old problem, the Scitara Digital Laboratory Exchange (DLX™) was conceived by a team with long experience in the laboratory environment and developed specifically for the scientific lab. The DLX offers a rapid connectivity infrastructure for any instrument in the lab in a fully compliant and auditable cloud-based platform. 

Frank Romanski, Director, Global Head of Marketing, Pharma Solutions, BASF

A: While it may seem cliché, I strongly believe in the disruption potential of digitalization for the pharma industry. Despite the aforementioned innovation power, the pharmaceutical industry still relies very heavily on analogue systems. The ability to create, test, scale-up, and commercialize modern complex formulations and biopharmaceutics takes an enormous team of specialists and experts, and yet, the way we share data, collaborate, and exchange information between entities remains largely on paper. As a supplier of high-quality pharmaceutical ingredients, the volume of analogue paperwork, technical know-how, and compliance documentation that is exchanged with our customers in the industry is astronomical — and this is multiplied further through the use of tollers and contract manufacturing organizations to bring a drug to market. Yet, all of these things within this business can be automated, digitized, and ultimately brought into the 21st century within the next few years. This simplifies an overly complex business, and simultaneously raises the level of accuracy over error-prone analogue transitions — directly impacting patient outcomes. To address this, at BASF we have pioneered ZoomLab™, a complex, proprietary algorithm allowing pharmaceutical formulators in over 91 countries the ability to model and optimize drug formulations, saving months of research time and thousands if not millions in development costs. While our RegXcellence® program allows for instant access and full compliance with the formerly analogue quality, regulatory, and safety data for our products. As a key supplier and integral part of the pharmaceutical value chain, we see digitalization as a true enabler of innovation and compliance, and we are happily leading the industry in building a platform for technical, quality, and regulatory truth. While I’m passionate and excited for quantum computing, we have a lot of “fix the basics” to do in the analogue world of pharma, and this is where I see the next few years going.

Matthew Hewitt, Ph.D., Senior Director, Scientific Services, Cell and Gene Therapy (CGT), Charles River

A: Decentralized manufacturing in cell and gene therapy will become the dominant way commercial cell therapies for oncology and auto-immunity are manufactured. Shipping cell therapies requires ultra-cold-chain logistics with complex interactions. While early commercial approvals in cell therapies were for relatively small patient populations, current shipping standards are untenable in the future as we see approvals for larger patient populations. As additional cell therapies are approved, staffing facilities will become the primary pain point. It will not make sense for every company to build out their own manufacturing capabilities, given already fierce competition for talent, which will ultimately drive up the cost of goods and sold for manufacturing. This will mean any facility that is built must be highly utilized to be profitable and will ultimately keep the cost of therapeutics high. Building out a biopharma industry–CDMO partnered hub-and-spoke decentralized, multi-modal (multi-therapy) manufacturing network is one vision that would maximize utilization while lowering therapeutics costs. This will also allow us to stratify the workforce to better utilize people. There will be challenges, the primary one likely being supply chain logistics, but it’s hoped that, as the field moves to manufacturing using closed, automated, scalable platforms, this will become easier. 

Zachary Pitluk, Ph.D., Vice President of Life Sciences and Healthcare, Paradigm4

A: Quantum will be one of the ‘next big things’ in the coming years that will affect the life science industry, accelerating scientific discoveries through the production of higher-resolution data and new computing paradigms.

Quantum microscopes and quantum computing will drive a further explosion in collecting in vivo biological data, solving the problems of resolution either directly via imaging or computationally. While quantum computing is still in its infancy, it holds the potential to elevate our understanding of complex biological phenomena, as well as to revolutionize the way drugs are discovered and developed.

With a digital transformation already underway across pharma, quantum methods will create an increased need for scientists to have their data organized and rapidly accessible in scalable analytics platforms future-proofed to meet emerging requirements.

Anil Kane, Ph.D., Global Head of Technical & Scientific Affairs, Pharma Services, Thermo Fisher Scientific

A: While the COVID-19 pandemic continues to create elements of uncertainty for the pharmaceutical market, it is more important that we continue to reinvent the approach of how to bring lifesaving drugs to market faster without impacting product quality, safety, or efficacy.

There are several takeaways from our experience in response to the pandemic:

  • Supply chains can be at significant risk when there is overreliance on a location that may be vulnerable to disruption. Shifting production locations closer to end markets or in lower-risk countries could be a new approach.
  • To avoid global clinical supply chain disruption, selection of clinical investigators/locations, as well as regional clinical warehouse supply centers, would be key.
  • Increased focus on operational resilience and accelerating initiatives that enable more agility with the use of digital tools and automation.
  • Companies will consider reevaluating their strategies, risk tolerance, and overall network footprint to address these risks. Key decisions include dual sourcing, geographic diversification, or outsourcing to CDMOs.
  • R&D will focus on analytical tools, big data, and artificial intelligence to bring drugs and therapies faster to the clinic. Digitization in clinical trial management, patient recruitment, monitoring, data collection, data analysis, and reporting will utilize digital platforms and remote decentralized units.
  • Digital tools will also enable some key business processes — such as auditing, regulatory inspections or product release. 
Nancy Lurker, Chief Executive Offocer, EyePoint Pharmaceuticals

A: The risk of significant price controls or price negotiations by the U.S. government has the potential to greatly impact all aspects of our industry. If it happens, we are likely to see a significant amount of pipeline programs shut down, particularly in cases where companies may not be able to generate enough money to justify the expense. And one challenge with that is, oftentimes, the maximum potential of a pipeline product isn’t fully discovered or completely understood until it’s used repeatedly in a broad base of patients — which can ultimately reveal new indications or different ways of using a given therapy. If current price-negotiating proposals from Medicare go through, we run the risk that the cutting-edge of innovation will be significantly curtailed. Many smaller pharma and biotech companies will eventually run the risk of going out of business — and power will be consolidated, much like what happened in the tech industry.

Jeff Elton, Ph.D., Chief Executive Officer, ConcertAI

A: With more clinical data being available from more sources with greater veracity and velocity, we can apply new tools — especially those derived from machine learning and artificial intelligence (AI) methodologies.  

We will see AI support the understanding of adverse events, help identify new patient cohorts, optimize clinical trial design, guide us to new clinical sites to accelerate accruals, and actually to avoid the prior generations of health inequities and under-representations. We can create models and AI-enabled optimizations to overcome historical biases. Of course, AI predicated on legacy data can perpetuate bias, but we are getting more sophisticated and purposeful about avoiding this. 

In the coming 3–5 years, AI will not be making clinical decisions, but it will be an integral part of why new medicines are available more rapidly; why we will have more confidence in trial design, realize shorter study execution times, and be able to translate study outcomes into evidence and rules guiding clinical practice decisions.

But you can’t have AI unless you have data — a lot of it, with depth and confidence that it lacks bias. This will cause a change in operating models that the United States is posed for, one that’s already starting within the biopharma innovators. The front-edge of the ‘next big thing’ emerged during the pandemic out of necessity. However, now seen, experienced, and assessed, it is clear the new ways can bend the historical curves of time, cost, and precision for the benefit of biomedical innovators, providers, and patients.

Mike Hopkins, Vice President, Business Development, US, Sterling Pharma Solutions

A: Despite the global disruption resulting from the pandemic, Sterling continued to grow its business and learned how to adapt and work in new ways.

Cannabinoid medicines and antibody–drug conjugates will continue to advance and become more mainstream, while flow chemistry is looking to become adopted more widely as a technology in API manufacturing. Sterling has invested to increase its capabilities in all these areas, as well as looking at further high-growth markets to meet future customer demands.

Where face-to-face meetings are not possible, ensuring transparency and the sharing of information through effective IT communication interfaces becomes even more important in building trusting customer partnerships. Sterling also recognizes the need for continuous investment in people and skills with a focus on customer service, building on its core values of transparency, caring, and reliability.

Sterling is expanding capabilities and capacity at each of its four sites in the U.S. and the UK, but more importantly, its CEO and management team are taking time to ensure that all of Sterling’s 800+ employees work by the company’s values in every decision made.

Through investment in equipment, infrastructure, and people, Sterling is striving to enhance its customers’ experiences and ensure its market position as a trusted partner of choice for API development.

Nate Mccutcheon, Executive Vice President and Chief Operations Officer, Shionogi

A: There are many exciting new innovative research programs that have the potential to change treatment in many important therapeutic areas. We are also excited about the meaningful advancements the role of technology can play in our industry.

However, one area of particular concern we all need to be very aware of is antimicrobial resistance (AMR). We believe this is a real and immediate global threat, and significant advancements must be made to prevent this from becoming the “next big thing” globally.

The damaging effects caused by resistant pathogens are already responsible for an estimated 700,000 deaths per year globally, and future projections of the impact of unresolved AMR infections surpass the projected number of deaths caused by cancer by 2050. AMR could also have a substantial impact on the global economy, in the form of both direct and indirect costs to society.

In the future, a lack of effective antibiotics could make routine medical interventions extremely challenging or even impossible.

For these reasons, we believe AMR must be regarded as a global, regional, and national priority for health organizations and governments, to be addressed with the utmost urgency on a global scale.

Maaike Everts, Ph.D., Director Strategic Marketing Parenteral Drug Delivery Solutions, Evonik

A: The recent advances in mRNA technologies driven by the COVID-19 pandemic has brought gene therapy — previously thought to be pharmaceuticals of the future — into the here and now. In response to the demand for the mRNA COVID-19 vaccines, Evonik rapidly built out capacity for lipid manufacturing and expanded production of parts of our portfolio, such as the plant-based cholesterol PhytoChol. In the long-term, we are investing in technologies that complement and go beyond lipid nanoparticles (LNPs). An example of such investment is a recent collaboration we started with Stanford University to develop a polymer-based delivery system for mRNA.

As gene therapy and related technologies become more widespread, managing complex or challenging supply chains — such as those requiring extremely cold storage — will be paramount. Lyophilization is a method of obtaining stable drug products for fragile (bio)pharmaceutical substances. We are therefore planning for more intensive use of our VarioSys® fill line with lyophilized products.

Marie E. Lamont, President and Chief Operations Officer, Inteliquet

A: Precision and targeted medicine is here and, while genetic testing has been partially adopted, our full power to unlock discoveries, providing the best and most effective treatment, won’t be achieved until standard of care evolves. Full testing, protecting patient privacy, should occur earlier in a patient’s care and disease journey. Testing occurs too late to have the most impact on care and treatment, and testing is not yet comprehensive for all patients. This wonderful tech and data are meant to help patients, and as a society we are not yet fully using it for the best interest of the patient.

Take this down a step. Clinical research no longer needs to be limited to legacy ways of matching patients to clinical trials, to academic centers, or by patient access to clinical trials when first diagnosed. Unlock the next generation of novel therapeutics. Bring together patients regardless of treatment location. Open opportunities to participate in research at the beginning of the patient journey. The immensity of the administrative process is painful, as finding the genetic details to match a patient has been manual. A new focus on genetic testing, improved global data infrastructure, and easy-to-implement software platforms means patients who were once outside the clinical research process can be included. 

These are important steps along the treatment and innovation continuum that move novel therapeutics and discoveries faster through development and into the hands of treating physicians and their patients and impact our global society as a whole.

Yan Zhang, Ph.D., Chief Executive Officer, Mission Bio

A: Genomics data is only as powerful as it can be interpreted, which is why the integration of genomics into our electronic medical records is going to be a huge step toward maximizing the clinical benefit. Soon, it will be routine for doctors and patients to have access to genomic data, with the potential to guide every medical decision. The low-hanging fruit is better diagnoses for rare genetic disorders, which affect 25 million undertreated Americans. As new research expands understanding of the links between genetics and more common diseases, doctors will be able to continuously improve how they guide patients toward better treatments and disease prevention.

Electronic medical records are an opportunity to leverage genomics in ways that can help every patient. They’ll connect genotype and phenotype in a real-world setting, which can open new insights for disease prediction, prevention, and early treatment. Therapeutics companies will be able to design and test combinations of treatments highly specific to patients based on their genetic likelihood of success. Diagnostics companies will use new insights to design tests that can stratify patients for specific cancer or cardiac disease screenings based on their predisposed risks. Doctors will be better able to guide patients to the right tests at the right time and ultimately to medicines with less risk of side effects. Patients will get treated for diseases before symptoms escalate.

Fabian Gerlinghaus, Co-founder and Chief Executive Officer, Cellares

A: One of the biggest impacts to the cell therapy market will be the pace of progress of allogeneic products. Marketed autologous cell therapies are powerful but made from each patient’s own cells in a time-consuming, costly, and complex process –– although automation is improving each of these.

Off-the-shelf allogeneic cell therapies are now in development that may push the industry  forward. Allogeneic therapies are derived from healthy donors rather than the patients, meaning they can be manufactured and stored, ready for administration to the patient at any time. Ideally, allogeneic products can be manufactured at a larger scale and at lower cost, opening the door to treat many more patients than the current made-to-order products.

Most of the approved cell therapies today are chimeric antigen receptor (CAR) T cell therapies, which have shown tremendous promise in hematologic cancers. With the FDA approval of Abecma® this year for patients with multiple myeloma, the class is beginning to expand into more clinical indications and larger patient populations. Still, everyone is waiting for the allogeneic leap, when cell therapies can finally be manufactured at a larger scale and treat an order-of-magnitude more patients, and, critically, at much lower cost.

At Cellares, we are well prepared to support this jump. With our automated technologies that support manufacturing at scale, we have the potential to bring these lifesaving treatments to millions of patients in need. Exactly how quickly these therapies will be ready for approval remains an open question, but we’ll be ready when they are.

Charles Christy, Head of Commercial Solutions Ibex® Dedicate, Lonza

A: The global COVID-19 pandemic has served as a wake-up call and altered the way the pharma manufacturing industry works and is viewed globally. The old binary choice between insource or outsource is being replaced by a more flexible collaboration model. The drug development pipeline is also changing; small, emerging, or even virtual biotech companies now bring the majority of early-phase drug candidates. These companies have limited in-house capabilities and need to outsource development and manufacturing.

With an increasing number of companies looking for flexibility and relying on outsourcing parts of their development and manufacturing processes to CDMOs, speed, access to manufacturing capacity, and collaborative business models to manage risk and uncertainty will soon take center stage.

Infectious disease outbreaks are very hard to predict, which increases the emphasis on speed to market. In addition, what has become clear is the diversity of biological approaches that have been developed, from novel monoclonal antibodies to viral vectors, cell and gene therapy, to the breakthrough of mRNA.

Our business needs to adapt fast and provide capacity across all modalities. Providing expertise, process development, fast scale-up, and commercial high-volume capacity as soon as the need arises is becoming more critical.

Lonza has a track record of scaling up and commercializing novel therapies. Our unique flexible offering, Ibex®Solutions, spans the complete product life cycle of a biopharmaceutical — from preclinical to commercial stages, from drug substance to drug product, all in one location. The manufacturing space already includes utilities and supporting infrastructure and is ready to be fitted out for any technology and scale, thus saving at least 12–18 months in building a new manufacturing line.

In summary, we can expect major changes to our business that are being challenged to move at a faster and faster pace and provide capacity for manufacturing of standard and novel modalities. We need to give customers the flexibility to manage supply, addressing drug development uncertainty and market demand changes. The goal will be to help new medicines reach patients faster, enabling a healthier world.

Glenn Mattes, President, Chief Executive Officer, and Director, TFF Pharmaceuticals, Inc.

A: The global pandemic changed the pharma landscape in terms of speed to market, storage, and distribution, and we believe this revolution in the industry is here to stay and will continue to focus on drug delivery mechanisms that enable easier access, both from a manufacturing perspective and platform development perspective.  Developing technologies that can be applicable to a variety of biological products and chemical entities can reduce cost in addition to facilitating rapid scale-up and rollout.  Within the COVID space and other indications, there is an increasing trend of using small molecules and biologics. Administration of large, complex molecules via injection is also challenging, as they do not directly target the initial site of infection. Liquid injections are subject to the difficulties of cold-chain handling, storage, and degradation.  While injectables have been the current standard for vaccines and therapies, we believe that alternate routes of administration for more targeted delivery will not only change the drug delivery field but address dosage issues, resulting in improved efficacy and safety profiles.  We may see an increase in technologies and mechanisms of delivery using alternative methods of drug administration. 

Pavan Cheruvu, M.D., President and Chief Executive Officer, Sio Gene Therapies

A: Our next great achievement as an industry will be bringing gene therapies to much larger patient populations than we’ve seen before.

Advancements in gene therapy have given us the tools to address diseases that we once thought were untouchable. Several gene therapy products have already been approved by the FDA, with monogenic diseases as the primary targets.

Many monogenic diseases are a natural fit for viral vector gene therapy, which replace and restore a missing gene. As a company at the forefront of new advances in genetic medicines, we believe that gene therapy will soon be extended beyond monogenic disease and begin to address multifactorial conditions that represent a significant portion of global disease burden. We are now beginning to see viral vectors as yet another safe and effective mode of delivery, and with advances in technology, it will no longer matter whether a disease is monogenic or multifactorial. I predict that this change in our mindset regarding viral vectors will enable gene therapies to benefit and access much larger patient populations.

Five years from now, we will drastically increase the number and type of diseases we can target with gene therapy — ranging from orphan diseases to indications like Parkinson’s disease and diabetes. The first successes of “larger-market” gene therapies in clinical trials will spur improved access to life-changing treatments and drive even greater innovation in this frontier of biotechnology.

Ian Walters MD, Chief Executive Officer and Director, Portage Biotech

A: We are moving past a one-size-fits-all approach for disease treatment generally, but especially in the field of oncology. I expect that the “next big thing” that is going to significantly alter the biopharma industry is the advent of newer, more evolved precision technologies that allow physicians to match the right medicine to the right patient. Previously, we didn’t have access to the necessary tools to profile which treatments would be better for specific patients. As a result, we developed medicines that had broad-reaching effects, but many patients who receive these treatments don’t see an adequate benefit. Today, we have more tools in the toolbox to accelerate the development of safer and more effective medicines.

We’ve also seen a greater push to identify which patients would respond best to which treatments and have started to see a greater need for profiling and more specific diagnostic technology, which ultimately should lead to soaring efficacy rates. This shift will drastically shift how drug developers identify, select, and use drug candidates going forward, so represents a major change but will also — hopefully — result in a major benefit to patients. It also changes the cost–benefit equation for developing drugs.  If you have a highly effective treatment, you need fewer patients to prove its benefit (translates to less expensive faster clinical trials).  A large part of what is going to push this shift forward is the increasing recognition of combination therapies as the most effective path to precision cancer treatment. We will continue to see customized treatment plans for patients that include multiple therapies tailored to fit specific patients’ needs.

At Portage Biotech, we’re taking all of these changes into account as we advance our novel immuno-oncology therapies through the clinic. It’s an exciting time to be in this field, and I look forward to continuing to collaborate with other experts as we work toward a brighter future for people with cancer.

Andrew Hall, Interim Chief Executive Officer and Chief Business Officer, IMV

A: Patients deserve therapies that preserve their quality of life. For difficult-to-treat diseases, current treatments often result in broad immunosuppression, non-disease specific, or non-selective modulation of immune function. These side effects can ultimately compromise patients’ health and quality of life and lead to treatment discontinuation. The advancement of immunotherapies with improved precision and long-lasting therapeutic potency that do not induce harsh and systemic adverse events have the potential to revolutionize treatment and, therefore, the biotech market.

Targeted drug therapies preserving a good safety profile that are easy to administer in an office setting and remain cost-effective is the next game changer in cancer therapies. For difficult-to-treat cancers, strategies focusing on generating a targeted and sustained immune response against tumor cells is a driving force in the development of novel solutions. Purposeful, precise activation of the immune system against tumor cells by these newer treatment options has shown unique advantages, such as efficacy in vulnerable patient populations or in patients that are non-responsive and even refractory through other lines of treatment.

The biotech industry needs to push the boundaries beyond precision medicine by focusing on patients’ quality of life, and this is changing the way many companies do business, forcing researchers and investors to concentrate their efforts on treatment options that also improve patients’ experience while undergoing therapy. At IMV, our DPX technology allows us to align our efforts on this vision to create novel immunotherapies to re-train cancer patient's own immune system for relentless, persistent disease control and eradication without the traditional toxicities.

Jeffrey Stein, Ph.D., President and Chief Executive Officer, Cidara

A: With the COVID pandemic still a global crisis, the biopharma market is in a persistent battle to keep up. While it seems hard to imagine anything positive coming from the pandemic, its unpredictability has forced us to make significant improvements. These include efficient decision making, hiring remote talent, and improvements in development timelines. While we have made great strides in these areas, COVID has also highlighted a key need that has remained unmet long before it — universal viral protection.

For the millions of people globally with weakened immune systems, there is a massive drop-off in vaccine effectiveness. This is not unique to COVID, as other vaccines, such as the seasonal flu vaccine, also protect a staggeringly low percentage of those with compromised immune systems. Additionally, as evidenced by COVID and seasonal influenza, vaccine effectiveness in general often wanes over time due to viral variation — losing protection for even those with robust natural immunity. Monoclonal antibodies for viral diseases have not provided adequate effectiveness, either.

I predict the “next big thing” will be the development of true universal protection, not in the form of a vaccine. Our company has seen preclinical success with our Cloudbreak® platform to provide universal protection for all viral strains. Now the race to usher in a new era of viral protection is being pushed to the forefront. We anticipate that, in the next decade, we will have begun reshaping the antiviral therapeutic landscape and create a world in which universal protection is available for everyone. 

Marco Taglietti, M.D., Chief Executive Officer, SCYNEXIS

A: There are two significant “next big things” ahead to highlight:

First, a coming positive force on the horizon is the increased adoption of in silico models in infectious disease drug discovery and development to accelerate the identification of new promising treatments. Spurred by advancements in artificial intelligence and machine learning, in silico approaches may help bypass the slower and more expensive “classic” approaches. We are approaching a point where we will be able to better leverage cumulative understanding of medicine and biology. Ultimately, this approach may allow for more efficient and cost-effective drug development and even potentially lead to new innovative treatments that may have never been identified without the help of advanced computational methods.

Second, a potential major challenge to the biotech industry’s ability to innovate is the possible introduction of new legislation and/or government reimbursement policies that may negatively impact the financial health of pharma and biotech companies, impacting their ability to develop new treatments. This is especially critical in the case of small biotech companies, which are the backbone of the U.S. R&D ecosystem. It is important for regulators, payers, physicians, patients, and legislation/policy sponsors to work together to identify a path forward that strikes a balance between providing patients affordable access to live-saving medicines without hampering the ability of small biotechs to pursue breakthrough scientific innovations. Although there is no easy solution, any new policies being considered should be measured against the potential impact on small biotech companies, as well as the overall healthcare ecosystem.

Prakash Pandian, Chief Information Officer, Curia (formerly AMRI)

A: For me, it’s two words: digital transformation. That is applying AI and machine learning approaches to drive innovation in R&D and to speed up drug discovery and development and, ultimately, manufacturing. We’ve seen some preliminary examples of this throughout the pandemic. We were applying in-line process controls to optimize real-time production parameters, giving us higher yields and excellent right-first-time metrics, which is differentiating for any company. Digital transformation, I believe, will be a big trend in our industry. We’re seeing some of it already; I don’t think it’s that far in the future.

Curia is transforming. At the end of the day, in our business, as a CDMO partner to pharma and biopharma, it’s all about safe and reliable supply. Our goal is to delight our customers with reliable supply and intelligent partnership — taking them from curiosity to cure. A big part of that is our embrace of digital transformation — that’s an area of focus for us at Curia — to stay ahead of that “next, big thing.”

Talat Imran, Chief Executive Officer, Rani Therapeutics

A: There are so many developments happening in our industry. One that is particularly exciting is what I’ll call “novel modalities.” The first novel modality on the horizon is in therapeutics, and, more specifically, medicines that actually cure a disease versus simply treating it. Methods like CRISPR and gene therapy are examples of novel approaches that may prove to be curative for patients with debilitating chronic conditions. Another example is mRNA technology, which could change how we vaccinate against different diseases. COVID-19 is just the beginning as companies like Moderna and BioNTech are looking at vaccines to treat and cure a variety of diseases.

Another novel modality is drug delivery. A lot of drugs work well, but are difficult to use, are painful, or are inconvenient for the patient. For example, our company, Rani Therapeutics, is trying to solve a long-standing problem: how to convert drugs traditionally administered by injection into a pill. Rani takes traditionally injected medications like octreotide and insulin and loads them into the RaniPill. The RaniPill, after being swallowed, travels through the stomach and into the intestines, where it delivers the drug directly into the intestinal wall. Denali Therapeutics is going after another novel modality: crossing the blood–brain barrier to better target neurodegenerative diseases. These are just two examples that I believe will be meaningful for our industry, and most importantly, transformative for patients.

Omid Farokhzad, M.D., Chairman and Chief Executive Officer, Seer Bio  

A: We believe it is emergence of large-scale, unbiased, and deep proteomics. We’re on our way to mapping the proteome more deeply, which is going to unlock new possibilities for disease biology, detection, and therapies. In recent decades, huge leaps in genomic discovery led to rapid genetic testing, broader knowledge of genetic drivers of disease, and new classes of therapeutics, like complex biological molecules and cell therapies. As we integrate proteomics, genomics, and multi-omics approaches with machine learning and data science, scientists will gain more comprehensive insights leading to earlier disease detection, more curative therapies, and more personalized medicine.

The convergence of proteomics with other data sets will further change the course of disease treatment. By more deeply exploring biology’s “machinery” and all of its variation — a new gateway to insights human health and disease will be opened.

Chronic diseases like neurodegenerative disorders are extremely difficult to treat in part because of how they present: by the time symptoms surface, the damage is already irreversible. But new insights into the proteome could lead to earlier opportunities to intervene, perhaps even with existing therapies.

In cancer, the heterogeneity of disease has long prevented one-size-fits-all therapeutic approaches. But we’re on our way to understanding the specific biological pathways to target for specific cancers in specific patients, which will lead to novel, curative approaches.

Read Part 1: The U.S. Pharma/Biopharma Industry

Dago Caceres

Dago is the Global Strategic Marketing Director for the Pharma Solutions platform of the DuPont Nutrition & Biosciences business unit. In this role, Dago is in charge of developing and implementing the global pharma strategy for the business to achieve sustainable, long-term growth. Prior to that, Dago has held multiple commercial, marketing, and business development roles primarily focused to the Pharmaceutical market for companies like Dow Chemical, FMC, and Rohm & Haas. Dago holds a Chemical Engineering degree from the National University of Colombia and an International MBA from the Moore School of Business, at the University of South Carolina.

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