March 11, 2022 PAO-03-022-RT-01
Alen Guy, Ph.D., Technical Director in the Pharmaceutical Business Group, IMCD
A: In 2022, the Pharma Business group will be putting more choices, solutions, and resources directly in the hands of customers through an innovative approach to our website. We have gone through an extensive digitalization process that we think will bring expertise and capabilities much closer to our customers. One thing all pharma companies seek is the effective treatment or product brought to market as quickly as possible. We can bring value and support to virtually every part of the development, production, and future of pharmaceutical products. The key in our digital age is to get the right balance between direct person-to-person expertise and accessibility for the customer at a time they choose or need. People have finite time to deliver top-quality service, and I think many developers and formulators accept this very well, but I know that IMCD Pharma group and the people we serve have a lot of common ground on what should be readily available. For this reason, our increasingly digitized and collaborative laboratory network will bring enhanced capabilities more seamlessly to our customers through the website-based Solution Centre. This will achieve the milestone of putting the IMCD Pharma group next to the customer as they create solutions for their challenges.
A: Arising from an increasing number of small and medium-sized pharmaceutical and biotech companies without their own production and/or testing facilities, heightened need for sterile injectable formulations, and growing demand for biopharma products, Alcami is undergoing significant growth.
In 2022, our Morrisville, North Carolina, and Charleston, South Carolina, sites are expected to double in number of aseptic fill lines with lyophilization capabilities in support of sterile manufacturing from clinical to commercial scale.
Implementing new operational approaches, including isolator technology, low-loss fill lines, single-use equipment, and terminal sterilizations allows us to increase manufacturing process efficiencies, minimize waste, and reduce costs.
Activities related to the development of COVID-19 therapeutics and vaccines, combined with the continued advancement of other biologic candidates, created deepened demand for bioanalytical testing. 2022 will bring significant enhancement to Alcami bio testing at our 16,000–square foot laboratory in the Durham, NC, site, which is being equipped with new tandem quadrupole and QTOF systems, variable pathlength UV, and ultracentrifugation.
Additionally, a solid-state chemistry center of excellence will occupy our newly built 12,000–square foot laboratory in Morrisville. This space will host thermal analysis with DSC and TGA, along with polymorph, crystallization, and salt form screenings using laser particle size, polarizing light microscopy, X-ray powder diffraction, and ion chromatography.
2022 also brings advancement in our cleanroom services. The acquisition of Masy BioServices expands Alcami's external environmental monitoring program with room and chambers mapping and the SenseAnywhere monitoring system.
We are also strengthening our internal systems by installing NuGenesis by Waters for next-generation, integrated laboratory information-management systems (LIMS).
A: This year begins a new chapter for Vector Laboratories as we move to expand our portfolio and capabilities. Thankfully, we’re not starting from scratch. Following a historic 2021 that saw Vector Laboratories become a standalone company, we now have in place the people, systems, and processes to capitalize on our inherent value.
With everything under one roof, we are planning an ambitious mergers and acquisitions strategy. We’re actively identifying complementary companies to further accelerate our growth strategy. Our goal is to complete one major acquisition, as well as multiple small, large, and transformative add-ons, starting this year.
M&A will be a key driver in our two-pronged product strategy. The first involves expanding our capabilities to better serve customers, not only with catalog products and customizations, but also custom services that result in high-value, bespoke products. The second prong hinges on serving the needs of customers as they move into applied uses, such as diagnostics. We want to broaden Vector’s presence in our existing sectors by continuing to invest in cutting-edge tools that can help our customers accelerate their innovations. We are planning to become market leaders in one new vertical, build out our custom product manufacturing capabilities, and grow commercial supply relationships in 2022.
A: This year, IFF’s Pharma Solutions is looking forward to the 60th anniversary of our flagship brand Avicel®, which pioneered the use of microcrystalline cellulose as a strategic excipient in the formulation of high-quality pharmaceutical products. As the originator and leader in this space, we have the responsibility to continue to supply the highest possible quality to the industry to grow and evolve with our customers. It’s remarkable how we’ve been able to shape and support the pharmaceutical industry with this critical product in the last six decades. Our journey is marked by continuous innovation, starting with our gold standard grades of Avicel® PH-101 and Avicel® PH-102 and then enabling customization to produce a wide range of solutions that are widely used by customers today. Due to our polymer science expertise and deep application know-how, we’ve also been able to create multiple co-processed materials that have enabled the production of more robust and reliable formulations.
Moving forward, we’ll continue to invest and support this product line. We are actively using cutting-edge digital tools like advanced analytics and predictive modeling to further improve the reliability, consistency, and quality of our entire product offering.
A: We have several important milestones on the horizon. One is the continued release of new modules for our digital tools within our industry-leading Virtual Pharma Assistants (VPAs) platform. We continue to focus on improving the customer experience, whether that customer is seeking technical, quality, regulatory, or purchasing information and services. New modules will build on our success and offer a seamless and unique experience to customers large and small.
Next, we are integrating digitalization into our core manufacturing sites; for example, in 2022, we will launch a truly virtual audit experience at our flagship Bishop, Texas, ibuprofen plant — offering all customers an intimate experience with the facility despite lingering travel restrictions from COVID.
I want to also highlight the increasing momentum in the area of sustainability. Customers in 2022 are demanding far more transparency into product carbon footprint (PCF) than in any years in the past, and, as a direct result, we have fully validated the specific PCFs for our ibuprofen portfolio and are actively expanding this initiative for other product lines this year.
Finally, we continue to expand our footprint in the fast-growing biopharma industry, where portfolio expansions and investments will continue to increase our capacity and unique capabilities in the market.
A: 2022 is the first full year after the preliminary FDA guidance for real-world data (RWD) supporting real-world evidence (RWE) for regulatory decisions was released in the fourth quarter of 2021. This is one of those rare inflection points where study designs can now be fundamentally reconsidered and where data from retrospective and prospective approaches can be integrated into a single coherent evidence-generation plan, by study and over the life cycle of a therapeutic program. There’s an emerging “System of Evidence” that offers the potential of accelerating therapeutic programs, allowing more confidence in earlier decisions, and ongoing studies confirming efficacy and safety.
ConcertAI was founded to advance the next generation of RWD and artificial intelligence software-as-a-service (AI SaaS) solutions to bring RWE generation in oncology, hematology, and urological cancers more broadly into practice with new standards of precision, representativeness, and utility across a range of study types. Our 2021 integration of TeraRecon, a leading radiological imaging company, and our partnership with ASCO’s ConcerLinQ program have set us up to bring a multitude of pieces together this year. Our retrospective RWE work will be complemented by our Digital Clinical Trial solution to enable biopharma-sponsored and investigator-initiated clinical trials within their workflows — using clinical data sources throughout. This will bring the entire “System to Evidence” to reality by offering biopharma innovators more options to accelerate needed new medicines and additional evidence guiding treatment decisions to patients and their care providers.
A: This year, the first patients in two critical clinical trials further examining investigational EYP-1901 will be dosed, respectively — in our phase II Durasert and Vorolanib in Ophthalmology (DAVIO) study of EYP-1901 for wet age-related macular degeneration (AMD), and in our phase II study of EYP-1901 for diabetic retinopathy.
In 2021, we shared promising findings observed in our phase I DAVIO study of EYP-1901 for wet AMD — interim six-month results demonstrated a positive safety profile, with 53% of patients remaining rescue-free following a single injection of EYP-1901. We hope the forthcoming studies commencing this year will continue to affirm and reinforce these data and the potential of EYP-1901.
A: Possibly the only milestone that matters in 2022 is ensuring that cancer centers of all dimensions can be included in the clinical trial process and, in doing so, offering more trials to more patients as a clinical care option.
In particular, our team’s goal is to reduce the myriad administrative burdens and costs holding all cancer centers back from joining the ranks of academic centers in clinical trial participation.
This is a massively ambitious industry and corporate goal because the real milestone is not necessarily showing that reducing the administrative burdens matters,, but that it can be done significantly and across many cancer centers uniformly. We have proved this somewhat quietly within the consortium we serve. But 2022 demands that we and others like us be more vocal in allowing more healthcare organizations and stakeholders to see it, to test it, and to make it easier for this model to be accepted and adopted.
So, it’s not just having accomplished material success, but doing it visibly so that others can readily adopt the best standardized methods.
There is tremendous urgency, because this year is so unique. The ongoing impact of the pandemic and challenges in staffing and retaining necessary talent are denying all of us the benefits of clinical research that should be further along today than they are. We can, however, change things permanently for the better.
A: We expect to announce a series of key partnerships to further streamline and automate the use of molecular diagnostics in clinical workflows across the United States using a digital platform solution for comprehensive cancer management. Our objective is to illustrate how a digital healthcare platform can unify EMRs, lab information systems, and molecular diagnostics to digitally reflect what the best comprehensive cancer care centers deliver to their patients.
In particular, we want to establish that automating referral management, tumor boards, molecular test results, and prior authorization with revenue cycle management removes significant friction from health systems. We believe that this is the year we will break down therapeutic silos by providing a unified health record, allowing healthcare providers to ensure patients receive precision treatments and opportunities to enroll in clinical trials.
A: Going into 2022, we continue to see high demands in large-scale manufacturing for the delivery of treatments and vaccines involving not just traditional antibodies but also novel technologies, such as mRNA and cell and gene therapies. As a result, we as a CDMO face a challenge to be well-equipped with sufficient capacity and the right facilities to accommodate the increasing needs.
This year, we have announced plans to expand our biopharmaceutical business within our three core drivers: capacity, portfolio, and global expansion. As part of this growth plan at Samsung Biologics, we are currently building Plant 4, which is expected to start cGMP manufacturing in October 2022, and are also scaling up our mRNA drug substance (DS) manufacturing capacity at our current facility, ready for cGMP operations within the earlier part of this year.
The construction of another new facility, Plant 5, is planned to start within this year, where we will offer multimodal products including cell and gene therapies and next-gen vaccines utilizing mRNA, pDNA, and viral vectors, all at a single site.
We are further venturing into securing additional sites within Songdo, for the construction of Plant 6 and Open Innovation Center, and also overseas in multiple locations to maximize both our manufacturing capacity to produce large-scale biologics and to be in close proximity to its global clients.
A: 2022 represents the first year Lonza is entirely focused on supporting the needs of the healthcare industry. This newly defined clarity allows us to concentrate on meeting the needs of our customers irrespective of their size, location, or modality type. We will continue to anticipate challenges related to the ongoing global pandemic and monitor the evolving market needs to remain firmly positioned to deliver solutions for our customers, partners, and investors.
In the past few months, we have continued to focus on our long-term success and responded to the evolving needs of the pharmaceutical industry. We progressed our growth investment strategy and, in 2022, expect to increase our total capital expenditures (CAPEX) to reach 30% of sales. We are committed to furthering our ongoing expansions in manufacturing capacity and capabilities spanning drug substance and drug product development and manufacturing across our existing production bases in China, the rest of Asia, the United States, and Switzerland.
With the biopharmaceutical industry undergoing significant transformation, CDMOs must evolve to meet their clients' changing needs and growth. At Lonza, we understand that our innovation delivers essential advantages for our customers. In 2022, we will continue to focus on finding new manufacturing solutions, such as Ibex®, which allowed the build-out of mRNA facilities in record time to produce Moderna’s Spikevax vaccine. We will further develop offerings spanning novel modalities, such as exosomes, and technologies, including the Cocoon® Platform for automated cell and gene therapy manufacturing and robotic solutions for endotoxin testing.
A: In terms of parenteral drug delivery solutions at Evonik, I think we are in for an exciting time in 2022. We are committed to further fueling the growth of nucleic acid therapies with the supply of lipids, formulation technologies, and manufacturing. Alongside lipids, we are also working with Stanford University on a polymer platform known as CARTs (charge-altering releasable transporters). These synthetic polymers can safely deliver mRNA to cells. A benefit of this platform is that it enables the delivery of mRNA to tissues and organs that are difficult to target with current lipid nanoparticle delivery. This year, we will start to offer formulation services with CARTs — expanding our toolbox of drug delivery technologies for mRNA and other nucleic acids.
Another highlight is the continued roll-out of the new version of our well-known liposome extruder called LIPEX® Flow, which we launched last year. This enhanced extruder enables higher throughput, faster batch processing times, and a broader operating range, thus expanding application opportunities.
A final highlight for this year is an important addition to the VarioSys® Aseptic Fill Line, which was built and integrated by Bausch + Ströbel in 2020 at our parenteral drug manufacturing facility in Birmingham, Alabama. This year we will install an L-flange, which will add even more flexibility to our VarioSys® fill line, allowing us to do double fills, including for aseptic suspension drug products.
A: For WuXi Biologics, the most significant milestones in 2022 will be the official operation and GMP release of both our U.S. site in Cranbury, New Jersey, and our European site in Dundalk, Ireland. With these locations, we will be closer to existing and prospective clients, given the fact that over 50% of our clients are headquartered in the United States or Europe. These milestones will also reinforce our “Global Dual Source” strategy — a commitment to clients that materials can be sourced and products can be manufactured at multiple WuXi Biologics locations across the globe.
As a global contract research, development, and manufacturing organization (CRDMO), we have continually expanded our capacities and capabilities in the United States, Europe, and Asia in response to growing customer demand around the world. With the COVID-19 pandemic, our reach and diversified capacity meant that we could help enable clients' response without being restricted to any particular region.
In 2022, WuXi Biologics will also further establish full, parallel capabilities in our sites across the United States and Europe, so they can provide true one-stop service — from process development to drug substance and drug product manufacturing. Our total cumulative CapEx in the U.S. and European markets is expected to reach USD $1.5 billion this year.
A: In 2022, PCI will continue to expand our sterile fill/finish capabilities, improving the global capacity shortage for sterile drug manufacturing and packaging that began during the pandemic. Through our acquisition of Lyophilization Services of New England, Inc. (LSNE) and the recent addition of three new automated, isolator technology sterile fill/finish lines at two key clinical trial facilities (San Diego, California, and Melbourne, Australia), we are continuing to meet the demand of the ever-growing biologics market and bringing life-changing therapies to patients faster. PCI is already perceived as a pioneer in biologics packaging; with the addition of LSNE, we will cement our reputation as a leading global CDMO that provides an integrated experience to our clients across the entire drug product life cycle.
A: We look forward to the upcoming launch of our SOPHiA Homologous Recombination Deficiency (HRD) Solution, opening new doors for cancer research. HRD is a complex biomarker, important for PARP inhibitors, that helps identify whether cancer patients may respond better to specific treatments. HRD is caused by a cell’s inability to repair double-stranded DNA breaks through the homologous recombination repair (HRR) pathway, causing mutations that lead to the development of certain cancers. One method for quantifying these genomic aberrations is with whole-genome sequencing (WGS). More specifically, the SOPHiA GENETICS method uses a deep learning algorithm that has been specifically trained to recognize patterns of homologous repair deficiency in the genome of cancer samples. The algorithm is designed to produce a quantitative measurement of the level of genomic aberrations present in the sample being tested. Prior to HRD testing, PARP inhibitors — which keep cancer cells from repairing — were among the biggest breakthroughs in oncology in the last 10 years. SOPHiA GENETICS has also partnered with Friends of Cancer Research and others to develop strategies for assessing assay performance and aligning methods for measuring homologous recombination status and its use as a biomarker in clinical care.
A: The Specialty & Custom Chemicals Show: SOCMA recently acquired a trade show, enhancing our mission to support the growth of the specialty chemical sector in North America. Our return to the trade show arena underpins SOCMA’s strategic focus to deliver commercial growth opportunities and maximize business development partnerships for the specialty and fine chemicals industry, which thrives on these connections. As companies work to reconstruct value chains and rethink CapEx projects and other business strategies, it has never been more important for them to come together — in a way that hasn’t been possible in almost two years. In 2021, we celebrated SOCMA’s 100th anniversary, and we are excited to solidify our position as a leader and connector for this innovative industry by delivering this critical avenue for companies to do business in 2022 and beyond.
ChemOps Training: With the lingering effects of the pandemic remaining, SOCMA accelerated the launch of the ChemOps Training program, answering a call for industry-specific, specialized training resources for chemical manufacturing facilities. A solution to many of our members’ top concerns around chemical operator training and onboarding, the program is quickly becoming the go-to source for the specialty chemical industry to safely and effectively train operators. Companies and industry associations alike are implementing ChemOps Training to improve efficiencies in onboarding as sites fill critical production openings.
A: 2022 will be a cornerstone year for Skye Bioscience, as we transition from a preclinical to a clinical-stage company. We are fast approaching the initiation of our first-in-human phase I study in the first half of the year, with topline data expected in the third quarter and the full data readout in the fourth quarter of 2022.
Our phase I study will evaluate the safety and tolerability of SBI-100 in healthy volunteers. Our objective is to check an important box for regulatory authorities by demonstrating that our drug has minimal to no side effects in the eye and no unwanted systemic side effects from THC.
What is more significant, of course, is potentially demonstrating the intraocular pressure–reducing effects of SBI-100 in patients with glaucoma, thus leading to our planned phase II study in the United States At this time, we have completed our pre-IND meeting, which has resulted in valuable guidance from the FDA, providing Skye with a clear path to move forward with an IND submission. Subject to approval, the initiation of its phase II study will follow, with an expected start date in Q4 2022.
A: Last year, we announced our dedicated global pharmaceutical ingredients organizational structure, leveraging our regional teams, international footprint, and strategic relationships with industry-leading suppliers to serve our customers better worldwide.
This year is about achieving a new level of intimacy with our customers and suppliers. We continuously expand our offering — from infrastructure investments to biopharmaceutical portfolio growth, to strengthen our value and service level. We will continue investing in our distribution facilities, upgrading several more of our locations to meet API handling requirements, FDA registration, and ISO 9001 certification. We will also expand our high-purity solvents distribution capability beyond North America to Europe and Latin America by installing filtration lines, purified tank farms, and equipment essential to maintaining product integrity and quality from origin to destination.
As the fast-evolving biopharma medicinal sector grows, we have already onboarded several suppliers to expand our offering with the novel ingredients used by this sector. We will continue expanding our portfolio with the bio-grade excipients and actives that this market uses.
Beyond our product offering, we plan to expand our technical capabilities by opening several Pharmaceutical Ingredient Solution Centers across the United States, Europe, and Latin America. These facilities and the scientists and professionals who lead them will allow us to deliver development solutions, regulatory assistance, and technical application testing. Whether it’s comparative analysis, formulation troubleshooting, or ingredient testing, customers and suppliers will have our labs and experts at their disposal for their innovation needs.
Finally, we take our Environment, Social and Governance (ESG) responsibilities very seriously as a global organization. We've outlined aggressive ESG goals for 2022 and will continue the rollout and investment across our business in low-carbon and resource-efficient technologies.
A: Collaboration is the catalyst for scientific innovation. As such, leaders need to be focused on how they can make the world a better place, together. Think about what your products, solutions, and research bring to the table, as well as what other players in the industry can bring. You may have a gap that another company’s product or research can fill, and vice versa. Each of us have different strengths and weaknesses, and to identify those within ourselves and others will result in strategic partnerships that highlight our strong suits and solve important problems. By promoting industry-wide collaboration, we can all focus on the overall mission of making the world a better place.
A: 2022 marks our 10th year as a company. Looking back at our early days, we had this vision to create simple tools that could provide a robust answer in a field setting to someone who is not an expert in mass spectrometry. Our life sciences and forensics customers have a job to do, and if we can make that job faster, easier, and more economical, then that’s a huge win for them. And it’s exciting to see, on a daily basis, the impact our customers are making in very critical-to-life applications. For example, first responders are using our handheld devices to detect and identify illicit synthetic opioids, reducing the number of fatal overdoses. Researchers are using our desktop devices to help bring new biotherapeutics to market faster in a quest to save lives from debilitating diseases.
While we’re proud of what we’ve accomplished, we’re excited about the next stage of our journey. We went public in December 2020, which has given us an infusion of capital to increase our technology and product investments. We look forward to the next 10 years, helping customers transform the way they’re doing things and making a difference in people’s lives.
A: In 2022, Reify Health is focused on scaling the capabilities we have developed to a large, global customer base and systematically working to increase patient representation in clinical trials along the way.
With our StudyTeam platform, this means continuing to increase adoption among sites, sponsors, and recruitment partners, especially in global markets. We already support clinical research in more than 75 countries, but we can do more to provide research teams in these countries with tools to run clinical trials more efficiently and more effectively.
This year, we will also be working closely with our enterprise sponsor partners to scale utilization of StudyTeam’s Diversity Reporting functionality. Sponsors and sites are limited in the ability to understand how a trial’s inclusion/exclusion criteria and protocol design impact people from diverse demographics through the recruitment process. We recently found that, out of every 100 Black trial candidates, only 23 will eventually enroll — compared to 40 out of 100 white candidates. In 2022, we are digging deeper into data like those to help our partners understand why the industry is failing to engage a more diverse group of people, and, most importantly, what they can do to develop real, nuanced strategies to improve.
A: In 2022, eClinical Solutions will celebrate its 10-year anniversary, marking a decade of helping modernize an industry that looked very different 10 years ago. The industry changes we anticipated have accelerated exponentially in recent years. Clinical trials have become much more complex, incorporating many new types of data, all of which have the opportunity to bring faster and richer insights to the development process.
Today, an average phase III trial includes data from 10 different systems, double the average from 2012. The impacts of working with a much higher volume of data in trials, including those from decentralized trial designs and systems, are numerous and have put stress on the people, processes, and technology associated with data review, integration, and analysis. The current processes and tools in the middle of clinical development have not kept pace with the type of data required to develop personalized medicines. In 2022, the trend to modernize all aspects of the end-to-end clinical data life cycle has hit an inflection point as technology investment in this space continues to grow. More life sciences companies of all sizes will adopt a modern data infrastructure and analytics platform, such as the elluminate Clinical Data Cloud, to liberate data from silos, automate manual processes, and make the work of drug development experts easier, faster, and more efficient.
A: As we begin 2022, we look forward to an exciting year for 9 Meters. We anticipate topline data from our phase II trial of vurolenatide for the treatment of short bowel syndrome (SBS), a rare and debilitating disease resulting in poor absorption of nutrients as a result of losing a significant length of the intestine. Patients suffering with this disorder can face serious nutrient deficiencies and life-altering GI symptoms. We believe that vurolenatide has the potential to address a number of these symptoms and may provide advantages over current treatment approaches, which aren’t appropriate for all types of SBS patients.
We are also anticipating an interim analysis in Q2 for our phase III trial of larazotide, the most advanced drug in development for celiac disease. Currently, there are no treatment options for the disease outside of a strict gluten-free diet, which doesn’t alleviate symptoms for all patients. We expect the results of our trial by the end of this year, and positive data at that stage could represent a breakthrough milestone for celiac disease care.
A: This year, we’re anticipating several milestones for our proprietary technologies that have the capabilities to unlock the potential of personalized TCR T cell therapy for cancer treatments. Within the first half of this year, we will launch the production version of our TCXpress™ platform, which is capable of processing thousands of single T cells directly into functionally expressed T cell receptors (TCRs) within a matter of days, thereby creating extensive libraries for rapid candidate selection. We will use the TCXPress™ production platform to support our discovery work for our internal clinical programs and for strategic discovery partnerships with other immuno-oncology companies.
Alongside this platform, we’ll continue to build upon our virtual patient data with our NEOXpress™ platform to further our personalized solid tumor program. Both TCXpress™ and NEOXpress™ work together to quickly identify the matching TCR/neoantigen pairs from the captured TCR and tumor-specific neoantigen repertoires obtained from patient tumors — a process we refer to as multiplexing.
In Q4 of 2022, we plan on filing an IND with the U.S. FDA for our lead TCR T cell therapy clinical candidate in our TCX-101 pipeline platform. This TCR T cell therapy, directed against HA-1, a minor histocompatibility antigen, will be given to high-risk leukemia patients in the context of allogeneic stem cell transplant to target their leukemia with a lower risk for graft-versus-host-disease.
Overall, we’re excited for our continued growth this year and remain committed to making personalized cancer therapy treatments a reality.
A: This year, we anticipate achieving a critical milestone for the company with the initiation of our pivotal study in the United States to evaluate the safety and performance of the C-Scan® System, our preparation-free, capsule-based colorectal cancer screening test in development to detect precancerous polyps.
C-Scan is designed to address a relevant unmet medical need: the low colorectal cancer screening rates observed globally, which result in almost 1 million deaths annually. Only about two in three adults among the targeted screening population undergo a colonoscopy in the U.S., and lower rates are found in other regions of the world, such as Europe and Asia.
Main barriers to screening include the invasiveness of the colonoscopy procedure and bowel preparation. Low invasiveness tests available or in development, such as fecal tests or liquid biopsy, are primarily designed to detect cancer and demonstrate low sensitivity in detecting pre-cancerous polyps. As such, they do not necessarily provide patients with the time window to pre-empt the disease. We believe there is a large unmet need for a patient-friendly colorectal cancer screening option, such as C-Scan, that can overcome barriers to colonoscopy screening while also enabling detection of polyps before they turn into cancer, versus detecting cancer once it has already appeared.
We look forward to bringing a much-needed colorectal cancer prep-less screening modality to the market in the upcoming years to enable early intervention, cancer prevention, and improved screening adherence worldwide.
A: Last year, we began the phase I/II AMPLIFY-201 trial of ELI-002, a lymph node–targeted therapeutic cancer vaccine. This year, we anticipate sharing data from all three cohorts evaluating a low, medium, and high dose of the vaccine. ELI-002 was developed with the company’s proprietary Amphiphile (AMP) technology that allows delivery of a therapeutic payload directly to the “brain center” of the immune system — the lymph nodes. The payload in ELI-002 includes AMP mKRAS peptides and ELI-004, a proprietary AMP CpG adjuvant. These components “teach” the immune cells in the lymph nodes how to identify specific cancer cells and target them for degradation.
One quarter of solid tumors harbors KRAS mutations, and AMPLIFY-201 is evaluating the safety and efficacy of ELI-002 as a potential treatment for patients with KRAS-driven tumors who have minimal residual tumor cells following surgery to remove the tumor. An investigational in vitro diagnostic device (IVD) is used to detect circulating tumor DNA (ctDNA) to identify patients who show signs of minimal residual disease in their blood before relapse is detected in traditional radiographic scans.
In addition to announcing data on the ELI-002 program, we plan to continue to advance our other pipeline programs, which include another therapeutic vaccine, the clinical stage ELI-004 AMP CpG adjuvant, cell therapy AMPlifiers, and infectious disease vaccines.
We have an exciting year ahead of us and look forward to continuing to unlock the potential of our AMP platform to make a difference in the lives of patients.
A: We have an exciting year ahead of us at Anixa Biosciences, with key milestones for our pipeline of oncology and infectious disease assets. Toward the end of 2021, our prophylactic cancer vaccine targeting triple-negative breast cancer entered a phase I clinical trial at the Cleveland Clinic. Long-term, this vaccine holds the potential to prevent women from developing breast cancer in the first place. We look forward to monitoring progress and sharing further updates on the program later in 2022.
In addition, in the first quarter of this year, we expect to initiate a phase I clinical trial for our CAR-T therapy being developed in partnership with the Moffitt Cancer Center to treat ovarian cancer. If successful, this treatment could be the first effective CAR-T therapy for a solid tumor and a model for how other CAR-T therapies can be developed to better address difficult-to-treat cancers.
We also plan to continue preclinical work for our COVID-19 therapeutic with our partners at MolGenie. Our aim with this program is to design an inexpensive, room temperature–stable oral therapy that is both convenient and highly effective against multiple variants of the virus.
We’re proud of the foundational work that we’ve accomplished in 2021 and look forward to continuing to bring our therapies forward to meet persistent unmet needs in cancer and COVID-19.
A: We anticipate completing the phase Ib clinical trial evaluating our lead drug candidate neihulizumab (AbGn-168H), a novel immune checkpoint agonist antibody targeting PSGL-1, in steroid-refractory acute graft-versus-host disease. Supported by the U.S. FDA with Orphan Drug and Fast Track Designation, we look forward to developing a safer and more durable drug in this population of high unmet medical need where patients suffer and fail previous lines of therapy safety.
We will be performing toxicology studies which will enable us to file an IND for our next-generation PSGL-1–targeting asset, leiolizumab (AbGn-268), which uses the same binding domain as neihulizumab but is engineered to provide convenient, patient-friendly subcutaneous dosing with higher efficacy. We will continue to progress our ongoing studies of leiolizumab (AbGn-268) in other indications, such as ulcerative colitis, Crohn’s disease, alopecia areata, vitiligo, and beyond.
Additionally, we will be focusing on generating additional follow-on molecules in our research and development work, as well as business development and licensing in complementary immune-modulating candidates.
A: In 2022, we look forward to a historic year for Omega Therapeutics and the industry, where we will bring programmable epigenetic medicines into human trials for the first time. Additionally, we have several key milestones that highlight the precision and flexibility of our OMEGA Epigenomic Programing™ platform and its potential to address a broad range of indications.
We anticipate filing an IND for the industry’s first epigenomic controller, OTX-2002, for the treatment of hepatocellular carcinoma (HCC) in the first half of the year. OTX-2002 is a modular and programmable mRNA therapeutic that targets a historically undruggable gene, MYC, through epigenetic modulation. As background, MYC is an oncogene found in >50% of cancers, including HCC. Despite its ubiquity among cancers, current approaches have failed to target this gene effectively. We believe, that through our epigenetic programming platform, we could offer new solutions to the challenges that have limited our industry’s ability to target the biological root of a disease.
Beyond OTX-2002, we are planning to select additional development candidates in the first half of 2022 to expand our clinical portfolio in oncology and beyond. We also expect to unveil exciting new preclinical data through multiple conference presentations that highlight the flexibility and therapeutic potential of our approach.
Cumulatively, 2022 represents a major transition for the company to the next stage of its evolution. We’re excited to be able to share the progress we’ve made and showcase the broad applicability of our platform in oncology, immunology, regenerative medicine, and multigenic diseases.
A: We expect 2022 to be a year rich with clinical data as we advance our four wholly owned and internally developed drug candidates in phase I clinical trials in hematologic malignancies and solid tumors. Nurix’s drug candidates are designed to modulate proteins by either harnessing or inhibiting a class of enzymes known as E3 ligases that orchestrate the body’s natural process of protein degradation within the cell.
NX-2127, the lead drug candidate in our protein degradation portfolio, is being evaluated in a phase Ia/Ib clinical trial in adults with relapsed or refractory B cell malignancies. We have demonstrated its ability to rapidly degrade Bruton’s tyrosine kinase (BTK) in the initial patients treated in the dose-escalation phase Ia trial and expect to report additional data from this first-in-human trial in the second half of 2022.
NX-1607, the lead drug candidate in our E3 ligase inhibitor portfolio, is a first-in-class inhibitor of the ligase known as Casitas B-lineage lymphoma proto-oncogene-B (CBL-B), which is being evaluated in an ongoing phase I trial in multiple solid tumor indications. We expect to have initial safety and PK/PD data from the dose-escalation portion of the phase I trial in mid-2022.
Finally, Nurix is the first company to combine targeted protein modulation with cell therapy in DeTIL-2055, an autologous T cell product stimulated and enhanced with the addition of a novel CBL-B inhibitor, NX-0255. We anticipate reporting initial findings from the ongoing phase I trial in the second half of 2022.
A: In 2022, we anticipate several company milestones, including clinical trial initiations and data readouts, digital medicine studies, and corporate growth. We kicked off 2022 with U.S. FDA clearance of a phase IIb trial, expected to start early this year, evaluating our pharmacologically optimized form of LSD, MM-120, as a treatment for general anxiety disorder. This is an industry milestone, as it marks the first commercial study of LSD in over 40 years. We expect to announce topline results from our phase I trial and initiate phase IIa for our 18-MC compound, an ibogaine derivative used to treat opioid withdrawal. We also plan to initiate phase IIa trials of our optimized form of LSD for acute and chronic pain indications, as well as a phase I trial of R(–)-MDMA for autism spectrum disorder.
Alongside our drug development milestones, we are looking to complete our first study of MindMed’s Session Monitoring System, a digital platform that supports the passive collection of sensory data during a consciousness-altering therapeutic session. We more than quadrupled our head count in 2021 and anticipate at least doubling in size in 2022 as we advance our drug development and digital medicine pipelines. To do so, we hope to secure additional equity funding from institutional investors with a shared long-term vision and belief in MindMed’s mission, leadership team, and execution strategy.
A: In 2022, we will continue demonstrating the power and versatility of our immune-educating delivery platform, the DPX® technology, to create novel immunotherapies. DPX is being developed for a variety of therapeutic areas (e.g., oncology) where generation of a long-lasting, target-specific immune response is expected to mitigate disease. Recently, we obtained promising clinical results with our lead compound, maveropepimut-S (MVP-S), in several hard-to-treat cancers. This year, we look forward to confirming these results in larger cohorts while expanding our clinical pipeline.
Later this year, we anticipate communicating preliminary results from our phase IIb clinical trial, VITALIZE, evaluating the benefit of MVP-S and intermittent, low-dose cyclophosphamide (CPA) in combination with KEYTRUDA® in relapsed/refractory diffuse large B cell lymphoma (r/r DLBCL).
We also predict sharing topline data from our phase II basket trial showing clinical benefit in patients with advanced, metastatic bladder cancer and treated with MVP-S, intermittent low-dose CPA in combination with KEYTRUDA at an upcoming scientific conference. MVP-S is also being evaluated for the first time in a neoadjuvant setting in a phase Ib study in women with HR+/HER2– breast cancer, where topline results will be collected in the second half of 2022.
In 2022, we are initiating two new clinical trials: AVALON, a phase IIb trial in ovarian cancer evaluating MVP-S to confirm results observed in a previous study (DeCidE1); and a phase Ib trial in bladder cancer evaluating our dual antigen–targeted T cell immunotherapeutic compound, DPX-SurMAGE. Through these milestones, we aim to continue moving MVP-S forward to registration.
A: This year holds significant momentum for Immunome as we continue to advance the discovery and development of antibody-based therapies for areas of high unmet need, with a focus on oncology and infectious diseases. As new COVID-19 variants emerge and we continue to grapple with the potential of COVID-19 becoming an endemic disease, it is a top priority for us to leverage our discovery engine to develop cutting-edge antibody therapies that can fight this virus. We recently submitted our first investigational new drug (IND) application to advance our three-antibody cocktail. In preclinical testing conducted to date, our cocktail demonstrates an ability to neutralize SARS-CoV-2 variants of concern, including Omicron. We look forward to communicating additional data as it becomes available.
In addition to our expected milestones in our infectious disease program, Immunome is also making substantial progress in our lead oncology program, IMM-ONC-01, an antibody targeting the novel, innate immune checkpoint IL-38. We believe, based on IL-38 expression in multiple solid tumors of high unmet clinical need, that this checkpoint may have a significant therapeutic role in oncology. We are actively working toward filing an IND in the second half of 2022. In parallel, we are advancing new oncology preclinical candidates and look forward to further demonstrating the potential of our platform in harnessing the power of human immune response.
A: This is an exciting year for X4, as we are anticipating topline data from our fully enrolled phase III trial evaluating our lead drug candidate, mavorixafor, in people with the rare immunodeficiency WHIM syndrome. As a first-in-class oral antagonist of the chemokine receptor CXCR4, mavorixafor has the potential to become the first disease-modifying therapy for these patients, given its ability to mobilize white blood cells from the bone marrow into the blood. Data from our ongoing phase II trial to date continue to show positive safety and efficacy, including durable increases in multiple white blood cell counts and improvements in infections and warts, characteristic symptoms of WHIM patients. Data from both trials will support a later NDA submission to the U.S. FDA.
On the primary immunodeficiency front, we also anticipate releasing additional data from our ongoing phase Ib trial in chronic neutropenia, in addition to continuing to explore other indications to target the broader chronic neutropenia landscape, given recent data that showed the potential of mavorixafor to increase white blood cell counts across multiple disease states, independently of CXCR4mutation status.
Finally, we anticipate releasing additional interim data from our ongoing phase Ib study of mavorixafor for the treatment of people with the rare non-Hodgkin’s lymphoma, Waldenstrom’s macroglobulinemia, who carry the CXCR4 mutation. These patients have more severe disease than those without the mutation and respond worse to the currently approved drug, ibrutinib. Data so far have shown the potential of mavorixafor–ibrutinib combination to improve clinical responses of these patients.
A: We look forward to another year of advancing TFF Pharma’s mission to leverage our Thin Film Freezing technology and transform medicines for better efficacy, safety, and stability.
We are entering phase II studies for our Inhaled Voriconazole Powder and Inhaled Tacrolimus Powder programs, which could improve standard of care for invasive pulmonary aspergillosis and immune rejection from lung transplant, respectively. In the near term, we are focusing on our internal programs, including Inhaled Voriconazole Powder, Inhaled Tacrolimus Powder, and our Inhaled Niclosamide Powder, an antiviral which is a potent inhibitor of multiple infections, including SARS-CoV-2, and is moving quickly toward phase II studies. We also expect to share updates on our inhalable COVID-19 antibody treatment, AUG-3387, which is being developed in collaboration with Augmenta Bioworks.
Beyond our internal pipeline, we are continuing to pursue our many R&D collaborations with academia, pharmaceutical companies, and the government, where our technology has potential to provide a differentiating impact for patients. Our Thin Film Freezing technology is versatile and is differentiated by the applicability to convert a broad array of molecules, including biologics, such as mRNA vaccines, to a dry powder that remains shelf-stable and can be administered through a standard inhaler, intranasally, or intra-ocularly to target affected organs more directly. This broad potential has garnered ongoing interest from companies, the government, and academic organizations alike, and we will continue to identify licensing partners to ultimately commercialize our assets.
A: Selecta had a productive 2021 with the release of human data showing ImmTOR’s potential to enable redosing and change gene therapy’s “once and done” treatment paradigm, the submission of our first gene therapy IND to treat Methylmalonic Acidemia, the enrollment completion of our DISSOLVE I clinical study, and numerous business development transactions.
Building on this momentum, we anticipate several exciting and important clinical milestones in 2022, including: topline data from the phase III DISSOLVE program, our partnered program in chronic refractory gout, a severe form of inflammatory arthritis; the initiation of our SEL-302 gene therapy trial in methylmalonic acidemia, a rare metabolic disease that affects the body’s ability to metabolize certain amino acids and fats; and the advancement of our IgG protease (Xork) toward the clinic with the hope of making those with pre-existing immunity eligible for treatment with AAV gene therapies.
Additionally, with the exciting preclinical data we released in January showing strong synergistic effects when ImmTOR is combined with an IL-2 agonist, one of Selecta’s top priorities is the rapid advancement of our next-generation ImmTOR platform toward the clinic. This combination, with its potential to induce and expand both regulatory T cells and antigen-specific regulatory T cells, has the potential to improve the ImmTOR platform across our three product pillars: Tolerogenic Therapies for Autoimmune Disease, Gene Therapies, and Enzyme Therapies.
With our expected milestones in 2022, we believe that this will be the year that we solidify our position as the leader in Precision Immune Tolerance.
A: Last year was a landmark year for SCYNEXIS. We received U.S. FDA approval for our first commercial product to treat vulvovaginal candidiasis (vaginal yeast infection) in the second quarter of 2021. I anticipate a similarly exciting year in 2022. In February, we announced positive topline data from CANDLE, a phase III study evaluating oral ibrexafungerp for the prevention of recurrent vaginal yeast infections. With these data, we plan to file a supplemental NDA by the end of Q2 and anticipate possible approval by the end of the year to give doctors a new option when treating their patients. Additionally, we will have the opportunity to showcase new data for ibrexafungerp as a versatile antifungal for a broad range of infections with interim analysis of FURI and CARES — two studies that are evaluating oral ibrexafungerp for patients with difficult-to-treat fungal infections, including those caused by resistant strains. We also plan to enroll our first patient in MARIO, a phase III study to evaluate oral ibrexafungerp in patients suffering from invasive candidiasis. Oral ibrexafungerp would serve as a step-down from IV therapy, with the goal of allowing patients to leave the hospital earlier and continue treatment with a potent oral fungicidal therapy. These milestones highlight our efforts to bring to market meaningful and innovative solutions in the fight against a broad range of fungal infections.
A: 2022 will be another productive year for Revolo as we continue our efforts to revolutionize autoimmune and allergic disease treatment with drug product candidates that reset the immune system to achieve superior long-term disease remission without immunosuppression.
On the clinical front, we anticipate clinical readouts from two ongoing phase II clinical trials evaluating our lead immune-resetting drug candidate, ‘1104, in eosinophilic esophagitis (EoE), and allergen sensitivity. EoE is a chronic rare allergic inflammatory disease that results in thickening of the esophageal walls and difficulty swallowing, which has really impactful consequences to daily life for these suffering patients. Currently, there are no approved treatments available, and we believe ‘1104 has the potential to offer a truly life-changing option.
We also plan to begin two phase II trials to explore the potential of our second immune-resetting drug candidate, ‘1805, for the treatment of patients with moderate-to-severe rheumatoid arthritis (RA) and non-infectious uveitis (NIU), a chronic inflammatory condition of the eye that is the leading cause of adult blindness in Western countries. Data from a previous phase IIa trial in RA demonstrated ‘1805’s potential as a safe and effective treatment for these patients, with sustained remission for 12 weeks following a single intravenous dose, and without immune suppression. We look forward to continuing to advance our candidates to bring what could be revolutionary therapies to those in great need.
A: The milestones that Recce Pharmaceuticals achieved in the last year have given us significant momentum heading into 2022 as we continue to advance the research and development of our new class of synthetic anti-infectives against a variety of bacterial pathogens, including their antimicrobial-resistant forms. After receiving ethics approval to start a phase I intravenous (IV) clinical trial of our lead synthetic polymer compound, RECCE® 327 (R327), we kicked off the new year with positive safety data, demonstrating R327 to be safe and well tolerated in healthy subjects up to 150 mg. Based on these results, we look forward to sharing further clinical updates regarding the remaining cohorts and testing subjects with ascending doses of R327, which is on track for Q2 2022. We are also making substantial progress in our phase I/II clinical trial designed to assess the safety and efficacy of the topical application of R327 for patients with burn wound infections. Visible infection reduction has occurred in all patients to date within the first 24 hours of treatment, including efficacy against a range of both gram-positive and gram-negative bacteria. With further clinical data expected to be available early this year, 2022 will see R327 continue to advance in ongoing in-human trials while also developing throughout preclinical studies. We look forward to further demonstrating the exciting potential of our anti-infective pipeline to assist in the fight against deadly drug-resistant pathogens.
A: 2022 marks an important year for Rain as we continue to evaluate our lead product candidate, milademetan, in multiple clinical trials and explore its therapeutic potential in new, broader indications alone and in combination with validated treatment strategies. Our initial approach was to commence and evaluate milademetan in a registrational de-differentiated liposarcoma clinical study based on strong prior clinical data. However, we see immense potential for milademetan to help a wide range of patients suffering from cancer. We expect to report interim analysis from our second trial, the MDM2-amplified phase II basket trial (MANTRA-2) later in 2022, evaluating milademetan in patients that exhibit wild-type p53 and MDM2-amplified advanced solid tumors. This year, we also plan to dose the first patient in our phase II MANTRA-3 trial evaluating milademetan as a monotherapy for the treatment of patients with Merkel cell carcinoma that are refractory to immune checkpoint inhibition. Finally, we recently formed a collaboration with Roche to evaluate milademetan in combination with Roche’s anti-PD-L1 antibody, atezolizumab, for the treatment of patients in a specific genetically selected population — patients that have lost function of CDKN2A — in a phase I trial. We expect 2022 to be the year when Rain begins to release milademetan clinical data, from which a multitude of clinical data will be represented over multiple quarters. Overall, we are excited for what 2022 holds for Rain as we continue to make progress toward bringing new therapeutics to patients with cancer.
A: At MilliporeSigma, we have set critical milestones to address our customers’ needs. We are committed to innovating and acting on trends impacting life and health with science. Let me highlight three key areas.
First, given the increasing trend in outsourcing services and the rising demand for both traditional and novel modalities, we have set up a global, fully integrated multi-modality CDMO services business. This year, we expect to take significant steps to scale our businesses and provide an end-to-end offering across modalities to help customers accelerate their lifesaving therapies to and through the clinic.
Second, our customer expectations are shifting, as shaped by their own digital experience. We are moving to provide a more seamless experience across labs, including academic, pharma, biotech, testing, and other labs, through innovation in our industry-leading eCommerce platform, sigmaaldrich.com. We expect that these changes for desktop and mobile platforms will provide a best-in-class customer experience. In addition, we will broaden our eCommerce offering to include select, high-value third-party suppliers that complement our leading portfolio to build a seamless experience for customers.
Third, we will continue to focus on delivering the company’s leading product offering for pharmaceutical development and manufacturing, including filtration devices, chromatography resins, single-use assemblies and systems, processing chemicals, and excipients. Significant investment in our manufacturing capacity across our portfolio will become operational this year; one example is the production of Mobius® Single-Use Assemblies in Molsheim, France, a critical product used to manufacture vaccines, including for COVID-19, and other lifesaving therapies. Bringing additional single-use capacity online in the United States, at our single-use Center of Excellence in Danvers, Massachusetts, and establishing single-use at our Molsheim site will shorten the lead time for custom assemblies for global customers. We recognize that innovation is also accelerating across the life science ecosystem in APAC, and we are continuing our commitment to ensure that our leading portfolio is available to customers where and when they need it.
A: Despite the unexpected nature of the past two years during the pandemic, Sterling’s strategic plan to grow its business saw us undertake a number of investments in areas that will either come online or reach a milestone this year.
First, we acquired ADC Biotechnology, a UK-based bioconjugation development services business specializing in antibody–drug conjugates (ADCs). Since the acquisition, we have invested to grow and develop the technical services team and expanded the analytical services team at the site in Deeside, Wales. This year, we will complete a project to establish cGMP bioconjugation and ADC manufacturing at Deeside, allowing us to offer end-to-end development services in this fast-expanding area of the industry.
Additionally, we have made a number of investments in other key technologies, such as flow chemistry and biocatalysis, but most importantly in capacity, to support what is a growing need of high-quality, small molecule API manufacturing. This year will see further capacity being added to our manufacturing network, potentially through a combination of strategic capital projects and/or additional acquisitions.
Sterling has also undertaken an initiative to increase the sustainability of our Dudley, UK, facility, with a goal of reducing carbon emissions by 50%. This year, an anaerobic digestion plant will be commissioned that will convert waste into a usable energy source and aims to reduce waste treatment emissions by 65% and carbon dioxide emissions by over 7,000 tons.
We also continue to align our culture, processes, and systems across our four sites, ensuring our continued commitment to service excellence for customers, as well as investing in our people and the opportunities we provide for them to grow with the organization.
A: Following a strong 2021, SOTIO anticipates several key milestones to continue building out our robust clinical pipeline. This year, we are advancing three novel programs through phase I. BOXR1030 is our lead CAR-T program for the treatment of various solid tumors expressing GPC3. It’s based on the proprietary BOXR platform that aims to enhance the fitness of T cells in the hostile tumor microenvironment, a long-standing challenge for traditional CAR-T therapies. The study is scheduled to start early 2022. SOT102 is a novel antibody–drug conjugate (ADC) with a best-in-class potential for Claudin 18.2–targeting therapies. We expect to start a phase I dose escalation in patients with gastric and pancreatic cancer in April 2022. Finally, we plan to initiate a phase I study with our first IL-15–based immunocytokine using a PD-1 inhibitor as the targeting arm by the end of 2022.
In parallel with the initiation of these three clinical trials, we are also initiating our phase II AURELIO-04 study in collaboration with Merck. The study will evaluate the combination of our IL-15 superagonist, SOT101 and MSD’s KEYTRUDA® (pembrolizumab) in patients with selected advanced/refractory solid tumors. The study is expected to treat up to 300 patients with a combination of SOT101 and a standard dose of KEYTRUDA and will enroll patients in the United States and selected European countries across six different indications.
I’m looking forward to another strong year from SOTIO as we continue to develop the next generation of potent immunotherapies for patients with cancer.
A: We will continue to support our customers in the context of the COVID-19 pandemic. Every second production site for COVID-19 vaccines worldwide now has process and packaging technology from Syntegon. We are currently seeing a change in vaccine production from vials to syringes and have already supplied first solutions to customers in China. An important highlight is definitely Achema 2022. It is the first major pharma trade show where customers can experience the new Syntegon brand live. We will introduce our Versynta FFP for small-batch applications, as well as Versynta microBatch here for the first time.
A: At Sino Biological, we have numerous milestones we intend to reach in 2022. Our main efforts will focus on product line availability and expansion and include the initiation of research and development in Taizhou and Suzhou, China, and product manufacturing/assembly in the United States. In addition, we will increase our infectious disease product offering as well as warehousing capacity and inventory levels in the United States and Europe for more efficient delivery to our clients in these regions. We will also expand our direct sales force in the United States, Europe, and China and complete at least one company acquisition, allowing for access to new technologies and talent.
A: As an organization, it is starting to feel like we have overcome the disruption that arose from the COVID-19 pandemic these past two years, and we are looking forward to maintaining our growth momentum during 2022. We continue to win new customers and broaden the portfolio of exciting new molecules that we are helping to develop. In response to this increasing demand, we will conclude major facility expansion programs at our key operating sites in both the U.S. and U.K. in the next 12 months. These expansions will deliver new capabilities that can be fully integrated into our services portfolio and increase capacity for our existing services — all of which are part of our continued commitment to expand and innovate our offering to support our customer’s drug programs.
This year also marks the 15th anniversary of our flagship Translational Pharmaceutics™ platform, arguably our most impactful innovation to date. In the intervening years, we have delivered hundreds of integrated drug development programs for our customers, proving that the acceleration of development timelines can be achieved with our unique approach. We’ve replicated the platform in the U.S., providing our customers with a choice of which territory they would like their work to be undertaken. During this year, we are focused on demonstrating how we can integrate drug substance synthesis and manufacturing into the Translational Pharmaceutics platform, which will represent another significant achievement in our mission to accelerate the development of new medicines for patients in need.
A: There are a few key milestones that Corning has on our 2022 roadmap to support our customers. As a leader at the forefront of 3D cell culture, we are continuing to evolve our product offerings for these advanced techniques in semi-automated and automated ways to allow for more widespread adoption, including in areas like high throughput screening applications. New tools like the Corning Matribot Bioprinter, Elplasia plates, Cell Counter, and Matrigel Matrix-3D plates help scientists reduce manual steps, improve consistency, and support more physiologically relevant models.
Cell and gene therapies continue to be a growing area in life sciences, and, to accelerate the path from the research bench to clinical treatments, Corning is continuing to develop and launch new solutions to support bioproduction. Our latest addition is the Ascent® Fixed Bed Reactor (FBR) System, which is designed to combine the greater yield efficiency, flexibility, and viable cell harvest capability of adherent bioproduction platforms with the scale, automation, and control of suspension manufacturing systems. The Ascent FBR System joins the full upstream workflow that Corning offers, which can support customers from vial thaw to seed-train scale-up and now to production scale across several advanced therapy applications.
A: The themes of 2022 for AGC Biologics are investment and ingenuity.
Through our investments and ingenuity, we are laser-focused on three key milestones as a company. First is enhancing and growing our cell and gene therapy services and capacities. The second is building upon our reputation in the protein biologics space. And the third is adopting and developing new processes and technologies to bring products to market even faster and more effectively.
For cell and gene therapy specifically, we are one of the fast-growing CDMOs, with some of the brightest minds in the industry, thanks to facility acquisitions in Longmont, Colorado, in 2021 and Milan, Italy, in 2020. The Longmont site coming online gives us even more global capacity to offer immediately to developers, at a time when there are industry-wide shortages. Our Milan site is also expanding its capacity and developing a new revolutionary viral vector suspension system.
Though we are expanding in those areas, we aren’t slowing down in our biologics protein work. We are expanding capacity at our largescale Boulder, Colorado, mammalian facility, our mammalian and plasmid facility in Heidelberg, Germany, and the Seattle, Washington facility’s mammalian and microbial capabilities. We are on a mission to grow our knowledge and services within this area to ensure we utilize the latest technology and processes to develop and manufacture substances.
For the underlying support of both these service areas, we are building new technology platforms and procedures that will improve every aspect of how we bring a product to market in 2022.
Dr. Alen Guy is the Technical Director in the Pharmaceutical Business Group of IMCD. He has previously held senior positions in excipient manufacturers and drug delivery companies. He holds a Ph.D. in analytical chemistry. At IMCD, he leads a team of more than 25 specialists and scientists and five Technical Centers. Dr. Guy has presented at numerous Drug Delivery and Pharmaceutical conferences on topics such as co-processing of active ingredients and excipients, excipient innovation, orally disintegrating tablet technology, taste-masking, and solid dose development. In recent years, Dr. Guy has particularly focused on the development challenges for products in pediatric, aging population, and long-term care patient groups.
Many of the change agents I have seen in 2019 are derived from changes in regulatory law, commercial downscaling, and impact from patent expiry strategies. The largest external regulatory change came from the issuance of the long-awaited EMEA Annex I, clarifying which technologies are required and acceptable, when and why.
The change in operational focus, from clinical scale-up to commercial scale-down, is enabling use of smaller, modular, flexible fillers with self-contained isolators. In parallel with the approval of biosimilars and biobetters, there is strong industry focus on individualized micro-batches, for CAR-T solutions and gene therapy products. The use of process automation and robotics have increased in all fill-finish unit operations. Widespread implementation of ready-to-use/ready-to-sterilize components and single-use (SUT) in upstream and downstream (SUS) through final fill designs have changed how facilities are planned, reducing plant size and changing warehouse space to accommodate densely packaged plastics goods.
Filling modalities have also been changing; bags that can be mated to lock-luer fittings with pre-sterilized needles and blow-fill-seal/form-fill-seal are re-emerging as processes that offer potential unit cost reduction. Traditional vial and syringe container designs are also changing as suppliers improve standardize offerings while having options including clear plastics.
The most exciting technological or scientific advancement that has influenced our business strategy in 2019 is our novel epigenetic regulator program. Unlike gene therapies, which target and modify DNA directly by inserting specific genes into patient’s cells, epigenetic regulators control or modify gene expression through processes that do not alter the sequence of DNA directly. Our lead asset DUR-928 is a small endogenous molecule that plays an important role in regulating cellular functions such as lipid homeostasis, inflammation and cell survival, crucial pathways involved in many acute and chronic diseases. DUR-928 has shown positive results in a phase IIa trial for the treatment of alcoholic hepatitis, a devastating acute condition with high mortality rates and limited therapeutic options. We are also advancing programs in other indications that could benefit from DUR-928, such as non-alcoholic steatohepatitis (NASH) or psoriasis. We believe that epigenetic regulation is a powerful and untapped treatment approach for many challenging diseases.