WuXi AppTec News


WuXi ATU Announces Licensing Agreement with Janssen for TESSA™ Technology

August 11, 2022 – PHILADELPHIA, PA. WuXi Advanced Therapies (WuXi ATU), a wholly owned subsidiary of WuXi AppTec, today announced a licensing agreement with Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson ("Janssen"). Under this agreement, WuXi ATU will license to Janssen its TESSA™ technology, a high-performance system that can produce 10 times more adeno-associated viral (AAV) vectors than traditional AAV manufacturing systems. Janssen will also have access to work on WuXi ATU’s proprietary clonal suspension HEK293 cell line. This agreement was facilitated by Johnson & Johnson Innovation.

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WuXi AppTec Reports Record First-Half Results in 2022

(SHANGHAI, July 26, 2022) — WuXi AppTec (stock code: 603259.SH / 2359.HK), a global company that provides a broad portfolio of R&D and manufacturing services that enable companies in the pharmaceutical, biotech and medical device industries to advance discoveries and deliver groundbreaking treatments to patients, is pleased to announce its financial results for the first half of the year ending June 30, 2022 (“Reporting Period”).

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WuXi AppTec Plans to Build a New Site in Singapore, Better Serving Global Partners and Advancing Healthcare Innovation

SINGAPORE, July 19, 2022 – WuXi AppTec, a leading global provider of R&D and manufacturing services that enable the global pharmaceutical and healthcare industry, today announced a plan to build a new R&D and manufacturing site in Singapore. The company intends to invest up to S$2 billion (approximately US$1.43 billion) to construct and operationalize the site, which aims to further enable global partners to advance healthcare innovations. The investment is expected to be made in stages over the next 10 years, depending on the company’s business needs.

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Innovation that Matters


Delivering on the Promise of New Therapies for Rare Diseases: An Interview with David Walker, Director of Chemistry, Sentinel Oncology

Rare diseases represent a significant unmet medical need, impacting the lives of millions of patients and their caregivers worldwide. At WuXi AppTec, we believe that our ongoing collaborative efforts to raise disease awareness and foster innovative thinking will lead to better and faster breakthrough treatments to address the healthcare challenges of rare diseases. As we continue this exciting journey to bring transformative medicines to patients, we are thrilled to share with you a new interview series from worldwide leading experts, “Delivering on the Promise of New Therapies for Rare Diseases.” Hi David! Thank you for joining our interview. To kick off, could you please introduce yourself? David: Hello, my name’s David Walker and I’m the Director of Chemistry at Sentinel Oncology. Sentinel is a Cambridge, UK-based drug discovery company focused on treating diseases with high levels of unmet medical need. I’m also the technical lead on Sentinel’s program to develop a therapeutic for Fragile X syndrome. As a company which is mostly focused on cancer treatments, what inspired Sentinel to work on Fragile X and what issues are you trying to address? David: I’m often asked why an oncology company is working on Fragile X syndrome! Well, we were driven by the science. Sentinel originally set out with a focus on developing selective inhibitors of the kinase S6K1 to treat triple-negative breast cancer. Triple-negative breast cancer is a particularly invasive subtype of breast cancer accounting for 10 to 20% of all breast cancers. Prognosis is generally poor and treatment options remain limited. One of the reasons for such a poor prognosis is the common formation of brain metastases which evade treatment. It has been published that S6K1 is responsible for driving the metastasis. To address this huge unmet medical need, we set about developing a series of brain penetrant S6K1 inhibitors. Then, in 2012, Professor Eric Klann and Dr. Aditi Bhattacharya at New York University published in the journal Neuron, showing that S6K1 plays a key driving role in the pathology of Fragile X syndrome. In short, Fragile X Syndrome is caused by a mutation of the gene Fmr1, leading to the absence of the protein product FMRP. FMRP acts as a repressor of protein synthesis so that in its absence, protein synthesis is always on and elevated. Klann and others have shown that in Fragile X patients and model mice, S6K1 is over-activated in the brain compared to healthy counterparts, and this drives excessive protein synthesis leading to pathology. S6K1 is a master regulator of protein synthesis and Klann and Bhattacharya showed that genetic deletion of S6K1 in a Fragile X model mouse restores healthy protein synthesis in the brain, as well as correcting associated pathologies. This led us to hypothesize that if a small molecule inhibitor of S6K1 could achieve the same effect, it could potentially be a viable treatment option for Fragile X patients. Indeed, we went on to collaborate with Klann and Bhattacharya to test our S6K1 inhibitor in their Fragile X model mice. This work was published in Neuropsychopharmacology in 2016. In short, our S6K1 inhibitor was able to correct a wide range of deficits in the Fragile X mouse. This included dampening down the activity of S6K1 in brain to healthy levels, decreasing protein synthesis in the brain and correcting neuronal spine morphology. Furthermore, we showed efficacy in behavioral models, including the social novelty test, Y-maze, and audiogenic seizure model. Overall, this strong scientific rationale underpinned our choice to invest in the Fragile X space. We are also driven by the high level of unmet medical need with Fragile X. All the existing therapies of Fragile X treat the symptoms only, whereas with our S6K1 inhibitor, we have the opportunity to treat the underlying disorder itself. What is the greatest value of your modality or technical approach to Fragile X syndrome, compared with existing treatments to these patients? David: Today, the drugs available to Fragile X patients treat symptoms only. Probably, the greatest value of our approach is that we have the potential to treat the underlying root cause of the disease. This in turn has a potential to improve profound aspects, such as cognition. To date, there has never been an S6K1 inhibitor in the clinic for Fragile X syndrome, so our approach is differentiated over what has come before. Could you share with us the current progress of your Fragile X program and its upcoming milestone? David: So, our candidate drug SOL784 has shown promising preclinical effects in several models of Fragile X Syndrome, giving us good confidence in taking this molecule forward. We have plans to put SOL784 into a Phase I clinical trial. Right now, SOL784 is in late stage development, undergoing a number of preclinical tests necessary for it to enter clinical trials. Let’s talk about the future. In your opinion, where might the next breakthrough in the field come from? David: In my view, the next paradigm shift looks to be the implementation of new measurable outcomes for drugs in Fragile X clinical trials. Some of this is happening already. Clinical development clearly is not easy in this field and there are challenges such as the placebo effect to contend with; however, outcome measures such as the EEG and eye gaze tracking are now finding their place in Fragile X clinical trial design. We are hopeful that an S6K1 inhibitor which treats the underlying pathology of the disease will show meaningful effect under these measures. From your point of view, how will AI transform the field of Fragile X research? David: I think it probably still remains to be seen. There are groups already using AI to predict rational combination therapy for Fragile X syndrome. These combinations are already in the clinic, so we should see results in due course. Could you highlight how collaborations and partnerships affect your Fragile X program and the field? David: Collaborations and partnerships are very important to this field. Ever since Sentinel got into this field, FRAXA has provided tremendous support to us in terms of advice and practical drug testing. This was not our original field of expertise and it’s fair to say that without that relationship, we wouldn’t have taken this program forward. So, huge thanks to Michael Tranfaglia and Patricia Cogram of FRAXA. Our research collaboration with Professor Eric Klann and Dr. Aditi Bhattacharya at New York University was also instrumental in generating proof-of-concept data on our S6K1 inhibitor in Fragile X model mice. We are very grateful to all our collaborators for their support. Finally, we are excited to be in alliance with MSRD to take our Fragile X program forward. In your opinion, what does patient-centric drug development mean for the Fragile X field? David: This is an important question. Patient-centric drug development means understanding the Fragile X patient population and the makeup of patients in any clinical trial. This will have a bearing on the outcome of said trial. There are important factors to consider such as age, sex, and genetic disposition amongst others. For example, in our case, there’s an argument to start treatment with an S6K1 inhibitor in young patients to target the pathology during development. Furthermore, genetic disposition affects severity of disease. For example, males with full mutation are generally considered the most severely affected group. We know from the world of oncology that patient populations tend to be divided into certain subgroups that respond differently to a drug. We’d like to explore that parallel in Fragile X Syndrome. In 2021, the FDA approved 50 new drugs. If we imagine 2030 and think beyond Fragile X, would we collectively be able to achieve 100+ new drug approvals at half of today’s cost? And in achieving so, do you foresee any major gaps to bridge or any upcoming breakthroughs which you are most excited about? David: Well, the drug discovery landscape has changed a lot in the last five years with biologics and gene therapies and others being added to the portfolio. In other words, the landscape is no longer small molecules. So, probably these additional therapies will supplement numbers, but I don’t think that net cost is necessarily the key question – rather, it’s where is the innovation coming from? In my view, nothing will replace the development cost of a drug, and experiments will get more costly as they get more complicated. In terms of major gaps to bridge; well, innovation starts early. It remains difficult to raise early stage finance, and innovators must have access to good finance to get the work done. Breakthroughs rely on high quality new science. There will be new ways to treat disease besides small molecule. AI may help with areas such as repurposing, but we still need to find those new drugs. Thank you very much for your time, and for sharing your valuable insights with us . David: Thank you.     David Walker DIRECTOR OF CHEMISTRY, SENTINEL ONCOLOGY David is a medicinal chemist with over 20 years’ experience in Biotech & Pharma. David has held positions at Pfizer, Cambridge Discovery Chemistry, Millennium Pharmaceuticals & Astex Therapeutics. Whilst at Sentinel Oncology, David has spent over 14 years managing small molecule drug discovery and development projects via an outsourcing model. David is technical lead on Sentinel’s program to develop a therapeutic for Fragile X Syndrome. David is a named inventor on multiple patents and co-author on several scientific publications.

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Delivering on the Promise of New Modalities: An interview with Elvire Gouze, CEO & Founder, Innoskel SAS, France

As part of WuXi AppTec’s ongoing efforts to collaboratively foster new thinking and actionable approaches in advancing breakthroughs for patients, we have launched a new interview series in 2022 – “Delivering on the Promise of New Modalities” – so leading voices of R&D can share how their approaches are addressing the barriers standing in the way of breakthroughs. The next installment of our interview series features Elvire Gouze, CEO & Founder of Innoskel SAS, France, a bioscience platform company developing transformative gene therapies for the unmet needs of individuals with rare bone disorders. When Innoskel launched at the end of 2020, they achieved Series A Financing to the tune of €20 million to advance the company’s lead program clinical stage operations. Innoskel is developing treatment options for a group of rare skeletal disorders collectively known as type 2 collagenopathies that affect the structure of the body’s connective tissues (collagen). Earlier this year, the company announced the enrollment of the first patient in a study, which will evaluate the course of type II collagen disorders in children with short stature. Thank you for taking the time to join us today, Elvire! We were excited to see early progress announced for your clinical trials earlier this year. For rare bone disorders, what are challenges in current therapeutic interventions, or current new modality solutions? Elvire: Innoskel is developing innovative treatments for rare bone disorders. These diseases affect the mechanisms of bone growth, mainly endochondral bone growth, and the challenge is to effectively target the growth plates of the bones of affected children while limiting off target distribution. There is no current modality for the diseases requiring gene therapy that have been shown to be effective at restoring bone growth. Indeed, common modalities, such as AAV or lipid nano particles, are known to be ineffective in targeting growth plates. Our approach is unique and a combination of the gene therapy modality, route of administration and proprietary promoter sequences allows us to have safety advantages combined with a directed biodistribution to the target tissues. How is your gene therapy approach helping to address this challenge? Is your technology differentiated from existing approaches? Elvire: Innoskel develops a lentiviral vector because of the ability of lentivirus to transduce non-dividing cells brings a significant advantage allowing genetic modification of the targeted proliferating cells and transmission to the daughter cells after cellular division. We have to pave the way with regulatory agencies and payers to address these diseases with a lentiviral vector administered systemically. The durability of the effect on bone growth is expected to be permanent – nevertheless, it is anticipated that due to bone growth and chondrocyte turnover, the duration of the LVV activity may be limited to 2-3 years requiring repeat administration. Innoskel is considering an immunomodulation regimen that should allow tempering down the immune reaction to the lentiviral vector and would allow for later redosing. In your opinion, what’s the biggest barrier to realizing the full potential of your LVV based gene therapies? Elvire: Manufacturing is a critical aspect of therapeutic intervention, and one must anticipate the technical challenges to accelerate treatment access for the patients. Indeed, current lentiviral vector production standards need to be optimized to reach a yield and product quality compatible with in vivo systemic administration. To solve these challenges and bring the therapies to global patients, how important is global collaboration to your company? Elvire: Global collaboration is critical for Innoskel. Indeed, when developing an innovative treatment for rare pediatric disorders, collaboration between companies, big pharmas and patient organizations is the only way to develop treatments and achieve patient access successfully and rapidly. Global collaboration is especially important in the rare disease area, where the best experts of these diseases are scarce and where the deep understanding of these diseases and their impacts on the life of patients is limited. It is essential to bring experts and patient representatives together to accelerate the development of therapies that are truly life-transforming and meaningful to patients. Worldwide databases and international collaboration are also critical to identify the few patients that could benefit from the treatment as quickly as possible, as we know that the therapeutic window before irreversible damages are done is tight in most cases. Looking for the best experts, wherever they are, allows us to be agile, creative, relevant and efficient. For Innoskel, the global collaboration approach is the best and only way to truly commit to patients to do the best we can to answer their high unmet needs. Thanks Elvire, really enjoy the discussion. If we were to gather here again in 10 or 15 years’ time, what do you think we’re going to be talking about in terms of what we have already achieved in the industry? What do you think are going to be some of the solutions going forward? Elvire: The real challenge for the next 10-15 years is to keep alive the interest of the pharmaceutical sector for rare diseases. Altogether rare diseases affect more than 400 million people worldwide. However, 95% of rare diseases have no approved treatment. The disinterest from the rare disease sector would be catastrophic and as a company, we need to be innovative to build a significant market case by regrouping diseases when possible. There are some solutions going forward such as the implementation of basket trials, allowing for the investigation of multiple rare diseases simultaneously with a single treatment intervention. Adaptive trial design is another solution that can be envisioned to accelerate the development of drug candidates for rare disease with high unmet needs.   Elvire Gouze, PhD CEO & Founder, Innoskel SAS, France Elvire Gouze is CEO and Founder of Innoskel. She is a distinguished researcher whose career represents a rare combination of scientific excellence, drug developmentand support for the patient community. Dr. Gouze has a proven track record of progressing the development of an innovative therapeutic pipeline, as seen with her first venture TherAchon, a biotech company focusing on achondroplasia, a rare bone disease, which was acquired at Phase 1 stage for $810M by Pfizer in May 2019. She holds a PhD in molecular pharmacology. She performed her post-doctoral training at Harvard Medical School, where she later became an Instructor in Orthopedic surgery in the Center for Molecular Orthopedics. Elvire Gouze was later appointed Assistant Professor at the University of Florida and in 2009 became Team Leader at Inserm Nice, France. Dr Gouze won twice the prestigious EY Entrepreneur of the year in the Startup category, PACA area, France.

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Delivering on the Promise of New Therapies for Rare Diseases: An Interview with Ralph Laufer, CSO of Lysogene

Rare diseases represent a significant unmet medical need, impacting the lives of millions of patients and their caregivers worldwide. At WuXi AppTec, we believe that our ongoing collaborative efforts to raise disease awareness and foster innovative thinking will lead to better and faster breakthrough treatments to address the healthcare challenges of rare diseases. As we continue on this exciting journey to bring transformational medicines to patients, we are thrilled to share with you a new interview series from worldwide leading experts, “Delivering on the Promise of New Therapies for Rare Diseases.” Thank you for taking the time to join us, Ralph! Before we start, could you please introduce yourself for our audience? Ralph: My name is Ralph Laufer. I’m the Chief Scientific Officer of Lysogene, which is a gene therapy company located in Paris, France, and we are developing gene therapies for neurological diseases. At Lysogene, we have two clinical stage programs for the treatment of rare neurodegenerative lysosomal storage diseases, namely MPS IIIA, also known as Sanfilippo disease and GM1 gangliosidosis, as well as several discovery and preclinical stage programs that are focusing on Fragile X syndrome, Gaucher disease and related diseases, as well as neurodegenerative diseases. What inspired your work in the Fragile X field and what issues are you trying to address? Ralph: Lysogene’s mission is to develop novel gene therapy treatments for neurological diseases, and in particular rare genetic diseases, such as Fragile X. In 2018, we initiated a collaboration with the lab of Dr. Herve Moine from the IGBMC in Strasbourg, France, based on his discovery of a novel downstream target of FMR1 in neurons – the messenger RNA of an enzyme called diacylglycerol kinase kappa. We also call it DGKk in short. And the goal of this collaboration was to evaluate the therapeutic effect of DGKk delivered into the brain using an AAV vector in a mouse model of Fragile X. What approaches are you taking to gene therapies? Ralph: So Lysogene’s approach is gene therapy; and the most widely used gene therapy approach, also known as gene transfer or gene addition, involves delivery of a functional gene to substitute a missing or dysfunctional endogenous gene. Predominantly, this approach relies on the use of an innocuous and replication-deficient viral vector to transduce human cells, such as the adeno-associated virus or AAV vector. However, in the case of Fragile X syndrome, a gene transfer approach using the FMR1 gene presents several important challenges. First of all, FMR1 expression dosage is critical for normal cell condition and neuronal function, and duplications of the FMR1 gene are identified causes of intellectual deficiency. Therefore it would be very difficult to administer a gene therapy treatment which would require such a tight regulation of gene dosage. Secondly, the blood brain barrier, which is constituted by a barrier of cells that surround blood vessels in the central nervous system, impedes crossing of AAV vectors from the bloodstream into the brain parenchyma which is the site where pathology occurs. And therefore, Lysogene’s approach is to target a downstream effector of FMR1, the DGKk gene that I mentioned earlier, as well as to deliver the AAV vector carrying the therapeutic transgene directly into the CNS, bypassing the blood brain barrier. What is the greatest and differentiated value of your modality or technical approach to the treatment of Fragile X syndrome? Ralph: The current treatment for Fragile X remains largely insufficient and the care is primarily symptomatic, focusing on disease management without addressing the primary underlying mechanisms of disease. Importantly, current treatments do not constitute a solution for intellectual disability, which is one of the main features of Fragile X. We believe that focusing on DGKk, which is an immediate downstream target of FMR1 and neurons, offers the opportunity to correct one of the major disease mechanisms, and therefore has the potential of being disease modifying. Could you share with us your progress on this program so far, and what is the next milestone? Ralph: The approach we have taken is based on the identification by Dr. Herve Moine, our collaborator from the IGBMC, of DGKk, as the most proximal messenger RNA target of FMRP neurons. The absence of FMRP leads to the absence of DGKk, and the latter mimics the effect of FMR1-knockdown, both in vitro and in vivo. So to tackle this problem, we constructed an AAV that carries an FMRP-independent variant of DGKk, and we administered this vector directly into the brain of FMR1 knockout mice, which is a well-known animal model of Fragile X syndrome. And when we did this, it led to the correction of all behavioral abnormalities observed in the knockout mice relative to healthy wild-type mice. Also, treatment with this AAV was not associated with any overt toxicity in the mice. These findings were recently published in EMBO Molecular Medicine. We believe on the basis of these findings that gene therapy with DGKk holds considerable promise as a treatment option for Fragile X, and we are currently performing additional non-clinical studies to advance this candidate therapy into the clinic. In your opinion, are there any innovative collaborations or partnership models that the Fragile X community can pursue in order to advance the field faster? Ralph: I would say our collaboration with the lab of Dr. Moine at the IGBMC, as well as with Conectus, which is the technology transfer organization of the Alsace region here in France, were crucially important for the success of this program. The program relies on the combined know-how and expertise of one of the leading academic labs with expertise in Fragile X, molecular biology, and a gene therapy company such as Lysogene, which has expertise in AAV vector design, as well as nonclinical and clinical development of gene therapies, and regulatory affairs in the field. This type of academy-industry partnership, with very close collaboration between the scientists, joint project teams, regular meetings, and joint design of experiments, is in my opinion, a very efficient way to advance basic research into the clinic. Finally, what does patient-centric drug development mean for the Fragile X field? Ralph: For Fragile X, patient-centric drug development really means focusing on clinical outcomes that matter to patients. In drug development, there is an increasing focus on assessing patient and caregiver experiences and preferences. This is important for designing clinical trials that will have clinically meaningful outcome measures, but it is also important during nonclinical development, where we try to identify suitable animal models with phenotypes that can either reproduce or be related to a clinical relevant symptom or biomarker. Thank you very much for your participation and sharing your valuable insights with us. Ralph: Thank you.     Ralph Laufer CSO, LYSOGENE Dr. Ralph Laufer is the Chief Scientific Officer at Lysogene, a gene therapy company based in Paris, focusing on the development of innovative treatments for neurological diseases. Before joining Lysogene in 2018, Dr. Laufer was Senior Vice President and Head of Discovery and Product Development at Teva Pharmaceutical Industries. Prior to that, he held the positions of Scientific Director of IRBM Science Park, a drug discovery partnering organization in Rome, Italy, and Head of Pharmacology at IRBM-Merck Research Laboratories Rome. He is the recipient of the American Chemical Society 2013 Heroes of Chemistry Award for his role in the discovery and development of Isentress (raltegravir), the first integrase inhibitor approved for use in HIV infected patients. Dr. Laufer’s scientific achievements include the discovery of the tachykinin NK-3 receptor and the anti-obesity activity of ciliary neurotrophic factor. He is the author of over 90 peer-reviewed articles and inventor of more than 20 patents. Dr. Laufer obtained his PhD in Biochemistry (summa cum laude) and M.Sc. in Chemistry from the Hebrew University of Jerusalem. He conducted postdoctoral training at the Institut Pasteur in Paris.

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