(SHANGHAI, August 13, 2020) — WuXi AppTec Co., Ltd. (stock code: 603259.SH / 2359.HK), a company that provides a broad portfolio of R&D and manufacturing services that enable companies in the pharmaceutical, biotech and medical device industries worldwide to advance discoveries and deliver groundbreaking treatments to patients, is pleased to announce its reviewed financial results for the first half of 2020 (Reporting Period).
SHANGHAI and PHILADELPHIA, June 1, 2020 -- WuXi AppTec, a leading global pharmaceutical and medical device open-access capability and technology platform company, announced today the appointment of Dr. David Chang as Chief Executive Officer of WuXi Advanced Therapies business unit (WuXi ATU), WuXi AppTec’s cell and gene therapy Contract Development and Manufacturing Organization (CDMO). In this new executive role, Dr. Chang will provide strategic, scientific and operational leadership to the company’s global cell and gene therapy R&D and manufacturing services.
May 18, 2020 – Philadelphia, PA - WuXi Advanced Therapies, a leading global Contract Development and Manufacturing Organization (CDMO) offering end-to-end contract services for the advanced therapies industry, announced today the expansion of its service capabilities to offer a fully integrated Closed Process CAR-T Cell Therapy Platform. This new platform will accelerate the timeline for cell and gene therapy development, manufacturing and release while providing greater predictability.
Watch the recording of our 4th episode in our “Collaborations That Transform” webinar series featuring dynamic discussions around how we can work together to meet the unmet needs of Multiple Sclerosis patients in the broader context of neuroscience R&D.
On September 24, join us for "Emerging Opportunities in Multiple Sclerosis and Neurosciences," the fourth episode in our "Collaborations That Transform" webinar series.
Throughout our "Collaborations That Transform" rare disease series, we have seen countless examples of rare disease R&D breakthroughs resulting from great partnerships and global collaboration. Spinal Muscular Atrophy is a rare disease community, which holds no exception to this trend.
Celebrate SMA Awareness with us! Stuart Peltz (Founder and CEO, PTC Therapeutics) shares his perspectives on the dynamics of R&D in the context of the journey that led to the accelerated approval this August of Evrysdi™ (risdiplam), the first oral treatment for SMA. What can we learn from the collaborative partnership between Roche, PTC Therapeutics, and the SMA Foundation?
In the past few years, the field of cell and gene therapy has witnessed major breakthroughs. It has become one of the hottest fields in the biotech industry, with the hope that it will eventually revolutionize the way we treat diseases and, ultimately, become a mainstream treatment option. Cell therapy refers to the transfer of healthy or modified human cells to heal or replace damaged tissue or cells, while gene therapy refers to the modifying of genes or replacing of defect genes with healthy ones to treat, cure or prevent a disease or medical condition. Though distinct, the two are closely associated with each other and often considered one field. Though still in their infancy, cell and gene therapies are developing at an explosive rate. There are currently hundreds of related programs in the clinic. This means that, over the next several years, many cell and gene therapies are likely to enter the market. There are clearly good prospects for the future of cell and gene therapy, but the road ahead contains obstacles as well. These include a prolonged and dynamic R&D cycle, safety issues, and manufacturing cost and limitations. Only by addressing and overcoming these challenges can we unleash the full potential of cell and gene therapy to help patients in need. Dr. David Chang, CEO of WuXi ATU, WuXi AppTec’s cell and gene therapy Contract Development and Manufacturing Organization (CDMO), recently sat down with the WuXi AppTec Content Team to share his observations on cell and gene therapy’s current development and insights into the field’s path forward. Prior to joining WuXi AppTec, Dr. Chang held senior positions at several leading companies, such as Celgene (acquired by Bristol-Myers Squibb in 2019) and Genentech. He also has extensive experience in the biotech and cell and gene therapy industries. What’s the current state of cell and gene therapy globally? Dr. Chang: Although the earliest research occurred decades ago, it is only in recent years that we have been able to bring effective therapies to the market. The biggest and by far most famous breakthrough in cell therapy is of course CAR-T (Chimeric Antigen Receptor T-Cell Immunotherapy). In 2017, Novartis’ Kymriah, developed for the treatment of certain types of ALL (acute lymphoblastic leukemia) patients, and Yescarta, developed by Gilead’s Kite Pharma for large B-cell lymphoma, were successively approved by FDA. These approvals demonstrated the potential of such therapies for cancer treatment. Gene therapy has also been gaining momentum for a long time, and over the past five years several drugs have been approved by regulators in Europe and the U.S. to treat severe inherent genetic diseases caused by specific genetic mutations, such as spinal muscular atrophy (SMA) and β-Mediterranean anemia. These recent developments are a great encouragement to the industry— to drug developers, to investors, and of course they are tremendously encouraging to patients as well. Keep in mind that, for any new therapy, a breakthrough from zero to one is the hardest. Given these successes, it’s no surprise that enthusiasm for cell and gene therapy has grown significantly lately, with many innovative companies being involved. The current pace of development is explosive. Hundreds of drugs are in the pipelines around the world, and many of them are already in the late clinical stage. To what extent will these types of therapies change the future of medicine? Dr. Chang: Cell and gene therapies have enabled people to effectively treat and even cure hitherto very difficult-to-treat cancers and rare genetic diseases, thus rewriting the life stories of many patients. I believe that they will become one of the mainstream treatments in the future. Cell therapy has the potential to be one of the most important treatments in our fight against cancer. For example, current CAR-T therapies have proven effective in treating relapsed/refractory ALL. These specific CAR-T therapies can, in theory, be applied to treat a lot of other cancers, and there’s a lot of relevant research going on right now. As for gene therapy, it has been applied to treat a wide range of diseases, such as cardiovascular disease, eye diseases, nervous system diseases, metabolic diseases and solid tumors, in addition to long-standing efforts in genetic blood diseases such as sickle cell disease and hemophilia. For cell and gene therapy to really take off, what challenges and problems need to be tackled? Dr. Chang: The potential is huge, but the challenges should not be underestimated. To keep our forward momentum there are still a series of bottlenecks to overcome. The industry needs to continuously improve R&D pipeline and lifecycle management, optimize the relevant manufacturing and logistics processes, and reduce costs. I personally believe the major bottleneck is not in R&D, but rather in manufacturing as well as in test and release. One of the major issues to consider in cell and gene therapy is how to scale out or up the manufacturing while reducing the cost at the same time. So, what exactly are the manufacturing challenges for cell and gene therapy? Dr. Chang: In gene therapy, there is a big gap in the production of non-replicable lentiviral vectors and AAV vectors. Depending on the genes of interest, production sometimes requires large-scale fermentations to meet a single patient’s treatment needs, making it commercially challenging or even infeasible. Therefore, yields and efficiency of virus vectors manufacturing are a major challenge for the whole industry. Cell therapy also faces the limitations in commercial manufacturing. Autogenous cell therapy is an individualized treatment that activate, modify and expand the patient’s own immune cells, so a single batch is made for one patient only. This means that the manufacturing scale will never be as large as that of traditional small-molecule or biologics products, and thus is much more costly. At the same time, cell therapy, especially allogeneic cell therapy, which utilizes donor cells, faces the challenge of graft-vs-host disease. It’s necessary to ensure that the modified cells transmitted back to the patient do not generate an immune response (similar to what happens with organ transplant rejection). Finally, cell therapy faces strict requirements for timely and reliable transport of labile raw material and cell products, as well as complexity in product testing and release. As an enabling platform for gene and cell therapy, what solutions and advantages does WuXi ATU have to address these challenges? Dr. Chang: Through the establishment of an advanced therapies CDMO platform, we offer real one-stop service to our partners to help with their research, manufacturing, and test and release. Most existing CAR-T manufacturing processes are not closed and thus require substantial open, manual manipulation under a biosafety hood, which may lead to errors, high costs, and risk of contamination. To specifically address these problems, WuXi ATU has recently introduced an advanced closed automated CAR-T platform in its base in Philadelphia to meet the manufacturing needs of our partners. Relying on our two bases respectively in the US and China, we can not only meet the diverse manufacturing needs of customers from different regions, but also ensure the timeliness of transportation that CAR-T requires. In gene therapy manufacturing, currently, we have established high-yield, highly scalable AAV and lentiviral platforms which can supply cost-effective and commercially feasible viral vectors in a timely manner. WuXi ATU also has the capability to provide international standard oncolytic virus product development and manufacturing services to customers worldwide. In November, we signed a strategic partnership agreement with Gene Medicine, a Korean gene therapy biotech, to provide services covering oncolytic virus product development, manufacturing and clinical trials for its key programs. In addition to the mainstream approaches of introducing genes into cells, we are also establishing more efficient and promising non-viral technologies such as electroporation, which utilizes electric fields to allow genes to pass through the cell membrane. Most importantly, WuXi ATU also provides the integrated analytical method development and testing services to our manufacturing clients. With the complexity of testing and releasing advanced therapies products in a timely and reliable manner, this integrated testing and releasing service will greatly support our clients to supply their products to meet either clinical or commercial needs. Through improving efficiency and reducing costs, we’ll support our clients to enhance the accessibility of cell and gene therapy – and help more of our partners’ innovative therapies to reach patients as broadly as possible as soon as possible. The rapid development of cell and gene therapy has attracted a lot of outside attention. People have high expectations. From the perspective of someone on the inside, what do you see when you look at the near future? Dr. Chang: There is no denying that cell and gene therapy still face challenges. Actually, every new therapy faces or faced a similar situation. Even regulators need to learn alongside industry. This process is both normal and necessary. I believe that in another five to ten years, with the accumulation of relevant knowledge and experience, the whole industry will become more mature, efficient and standardized. I firmly believe that the future for cell and gene therapy is very bright, and WuXi ATU stands ready to join hands with global innovators to move it to the next stage.
At TG Therapeutics in New York City the company didn’t start out looking for new therapies for Multiple Sclerosis (MS). TG began as an oncology focused company but as the data came in from their research the company and President and CEO Michael Weiss believed their assets could be applied in additional disease spaces and are developing a new therapy for MS. More than 2.5 million people suffer from MS worldwide. It is a long-lasting disease that can affect your brain, spinal cord, and the optic nerves in your eyes. It can cause problems with vision balance, muscle control, and other basic body functions. MS is a rare autoimmune disease. Normally, antibodies produced by the immune system help protect the body against viruses, bacteria, and other foreign substances. In people who have MS, the immune system attacks the body. There is no known cure for MS, but Mr. Weiss believes that real progress has been made in developing new treatment options for MS sufferers, and that TG may be offering its own new therapy for MS. WuXi AppTec Communications as part of its special series on MS spoke to Mr. Weiss about the advanced therapeutic approaches TG is developing to treat this debilitating disease. Mr. Weiss founded and has served as the Company’s Executive Chairman, President and CEO since December 2011. Prior to TG, Mr. Weiss founded and was the CEO of ACCESS Oncology (1999) which merged with Keryx Biopharmaceuticals (2004) for which he served as Chairman and CEO until mid-2009. Previously, Mr. Weiss spent several years as a lawyer and then investor in biotech venture capital. Mr. Weiss continues to have a passion for helping early stage biotech companies and serves as Vice Chairman of Fortress Biotech and Chairman of Checkpoint Therapeutics and Mustang Bio. WuXi AppTec: Many biotech companies are involved in cancer research; how did you chose to target MS as well? Michael Weiss: At TG our number one goal and priority is to create the best possible treatment solutions for patients with B-cell diseases. While we began with a focus on oncology, we’ve known that many autoimmune diseases also result from aberrant B-cell activity, so from very early on we had a vision to also bring our drug candidates into autoimmune disease. When we saw the early data evaluating CD20s in MS we knew this was a great place to start helping patients. WuXi AppTec: What makes MS so difficult to develop effective therapies for? Michael Weiss: One of the biggest challenges in treating MS is that the cause remains unknown. It’s still unclear exactly why this autoimmune CNS disease develops in some people and not in others Most available data points to some combination of genetic and environmental factors sharing the responsibility. In addition, signs and symptoms of MS patients vary widely. That said, while this lack of critical information has so far made targeting the root cause of MS extremely difficult, we believe CD20 treatment is extremely beneficial to patients and is a significant step forward for the treatment landscape. WuXi AppTec: Have you encountered any patient recruitment challenges in your MS program that were different from your experience with other trials you’ve conducted in the past? Michael Weiss: The ULTIMATE trials enrolled incredibly well with a total of approximately 1100 patients across both trials and target enrollment achieved approximately 9 months ahead of schedule. WuXi AppTec: How much progress has been made in MS drug research and development over the last 20 years? Michael Weiss: Progress has been significant to say the least. Think about it: 30 years ago, there were no approved MS drugs in the United States at all. Ten years ago, there were still no oral treatments available. The use of anti-CD20 monoclonal antibodies in treating MS was only first approved in 2017. We’re witnessing several MS treatments in development, and while a true cure remains elusive, I have hope this research will serve to further advance novel therapies and provide new treatment options for the millions of MS patients worldwide. WuXi AppTec: We have been talking about MS research in general. I would like to switch to your specific drug program. What phase is it in? What is its MOA? And what results have you seen so far? And will it treat all stages of the disease? Michael Weiss: Our ULTIMATE I and ULTIMATE II Phase 3 trials in relapsing forms of multiple sclerosis (RMS) are two independent, global, randomized, multi-center, double-blinded, double-dummy, active controlled trials comparing ublituximab to oral teriflunomide. The primary endpoint for each study is annualized relapse rate following 96 weeks of treatment and the trials are designed to support submission for full approval of ublituximab in relapsing forms of MS. As I had touched on earlier, ublituximab is a novel glycoengineered monoclonal antibody that targets a specific and unique epitope on the CD20 antigen found on mature B-lymphocytes. Its mechanism of action is similar to the approved anti-CD20 monoclonal antibodies, but the innovation of ublituximab is that it has been bioengineered to remove certain naturally occurring sugar molecules from the anti-CD20 antibody in a process called glycoengineering. By removing the sugar molecules we enhance the potency of ublituximab, with data showing 50-100x greater activity than non-bioengineered anti-CD20 antibodies. The ULTIMATE I & II trials are fully enrolled and being conducted under Special Protocol Assessment with the FDA. We are targeting top line data from these trials in the second half of this year. WuXi AppTec: Why do you think your approach can be more successful than other companies’ drug programs in this disease? How will it fit in the MS market space? Michael Weiss: Right now, we expect that ublituximab will be the third CD20 to market, following ocrelizumab, which is currently selling, and ofatumumab, which is expected to enter the market in the next six months or so. To my knowledge, there are no other CD20s in development or late-stage development for MS. We believe ublituximab may offer several advantages over the other CD20’s for MS, including the convenience of being a one-hour infusion every six months, versus the current 3-4 hour standard of care with ocrelizumab, at what we expect to be a competitive price designed to optimize patient access. WuXi AppTec: Sounds very promising! Finally, when do you think your drug may be available to patients? Michael Weiss: Assuming all goes well, we plan to possibly prepare a regulatory filing next year. If that is the case, we may be looking at FDA approval in late 2021/2022.