CSL Limited Annual Report 2021/22 31 Why We Need to ‘Disrupt’ Ourselves Patients, Public Health, Our Employees and the CSL business are counting on it To be an innovator means that, at times, you will disrupt the status quo and challenge orthodoxies to achieve better outcomes. At CSL, we have a history of disrupting ‘the way things are’. Equally, we are not afraid to also disrupt ourselves if it means an even better experience or outcome for patients and public health. ‘ As a key driver of CSL’s future growth, R&D’s job is to create the pipeline and capabilities necessary to help the CSL Behring and CSL Seqirus businesses grow in the decades ahead. We need to provide our businesses with the scientific platforms, products, skills, and expertise that will meet the future health needs of patients and the general public. This means that sometimes, our innovation will disrupt our own offerings or ways of working in order to better address the needs of those we serve.’ Dr William Mezzanotte, Executive Vice President, Head of Research & Development and Chief Medical Officer We need to look no further than CSL’s advancements in haemophilia B, a rare disorder where blood doesn’t clot normally due to not having sufficient factor IX. Decades ago, CSL stepped up and introduced plasmaderived factor replacement therapies – which, at the time, significantly transformed the lives of people living with this rare bleeding disorder. Despite having to be routinely intravenously infused, and having to monitor their activities, haemophilia B patients found this medicine provided a benefit by helping them to lead fuller, more active lives. Nevertheless, we knew we could do better. In 2016 we received our first approval for IDELVION®, our long-acting recombinant factor IX albumin fusion protein for the treatment of haemophilia B. IDELVION® is also infused intravenously, but patients who are well-controlled on this regimen may potentially switch to a 14-day dosing interval allowing them to better fit dosing into their schedules. Moreover, this treatment reduces breakthrough bleeds and gives patients the ability to lead a more active life. IDELVION® is the standard of care in several countries around the world, however, there is still an unmet need for many patients. Which brings us to today, and our quest to bring the promising etranacogene dezaparvovec, also known as CSL222, to the market. Etranacogene dezaparvovec is an adeno-associated virus vector serotype 5-based (AAV5) gene therapy that is specifically designed to enable near-normal blood-clotting ability by addressing the underlying cause of haemophilia B – a faulty gene that causes a deficiency in clotting factor IX. In clinical studies, etranacogene dezaparvovec, after a single, one-time infusion, has been shown to significantly reduce the rate of annual bleeds in patients with haemophilia B, compared to when these patients were receiving recombinant factor IX therapy alone. If approved, etranacogene dezaparvovec would be the first ever gene therapy treatment option for the haemophilia B community and enable a major change to the lives of those patients who are appropriate for the therapy. ‘ Etranacogene dezaparvovec, potentially the first gene therapy approved for haemophilia B, further demonstrates CSL’s mission to relentlessly pursue innovative and disruptive technologies when it benefits patients with rare and serious disease. This is what it means to truly Deliver on Our Promise.’ Dr William Mezzanotte, Executive Vice President, Head of Research & Development and Chief Medical Officer Patients with hereditary angioedema (HAE) can expect the same type of dedication from us. From the ground-breaking BERINERT®, designed to halt HAE attacks, to the disruptive HAEGARDA® which results in near-elimination of HAE attacks, CSL is moving one step further as we study garadacimab, an anti-factor XIIa monoclonal antibody. In Phase II studies, this home-grown therapy produced similar efficacy for HAE patients as does HAEGARDA® but with a less frequent, once-monthly administration schedule and with the additional patient-friendly benefit of an autoinjector for easier administration. CSL also disrupts in other areas where we already have a lot of experience and world-class capabilities. Egg-based vaccine manufacturing is the most common way that influenza vaccines are made, with CSL producing them since the 1940’s. However, newer technologies such as cell-based vaccines, offer a modern, efficient and scalable alternative to traditional egg-based manufacturing for seasonal influenza vaccine production and rapid pandemic response. As the largest cell-based influenza vaccine producer in the world, CSL has been able to accelerate production from pilot scale to industrial scale. Adding an adjuvant to both egg-based and cell-based vaccines is intended to make these vaccines more effective. While we have world-class capabilities in cell-based and adjuvanted vaccines, CSL continues to innovate with a self-amplifying messenger RNA (sa-mRNA) technology platform – the next generation of mRNA technology. During the COVID-19 outbreak, mRNA technology was thrust into the spotlight for its role in fighting the pandemic; sa-mRNA takes the technology one step further. It could be beneficial in both pandemic response and to help prevent seasonal influenza more effectively and consistently, a major advantage for public health. In fact, to help expedite our work in this exciting area, CSL is advancing a new R&D campus for sa-mRNA in Waltham, Massachusetts, in the US, that will serve as the company’s central R&D site for current and future vaccine design, and collaborations with stakeholders from across the industry and academia. CSL will continue to explore ways to innovate and improve medicines for patients and public health, even when we are successful. That is what we have done for patients with haemophilia B and hereditary angioedema, and with our influenza vaccines and what we plan to do for others. It’s an exciting time to be in the business of disruptive innovation for patients, public health, our employees and the CSL business as CSL R&D drives forward with this approach.
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