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Chiesi Limited

Everyone of us is different. Everyone of us is Chiesi.

Every choice we make is driven by the concept of creating shared value; using our business as a force for good to positively impact our society and environment.

Chiesi Limited

Committed to patients

For more than 90 years, Chiesi has developed innovative medicines and services for the world’s most vulnerable.

Chiesi Limited

Everyone of us is different. Everyone of us is Chiesi.

Every choice we make is driven by the concept of creating shared value; using our business as a force for good to positively impact our society and environment.

Chiesi Limited

Committed to patients

For more than 90 years, Chiesi has developed innovative medicines and services for the world’s most vulnerable.

COMMITTED TO PATIENTS

For more than 90 years, Chiesi has developed innovative medicines and services for the world’s most vulnerable in respiratory, rare diseases, neonatology and organ transplantation.

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Our survey found that 70% of people living with chronic or genetic health conditions - or who have experienced a premature birth - have faced stigma, judgement or blame due to their health condition.i

Look Beyond the Bias is a Chiesi commitment to challenging harmful biases, sparking national conversation, and reducing health inequalities.

iChiesi UK survey conducted in 2025 of 1,148 people. Data on file.

UK-CHI-2500837 | September 2025

Our survey found that 70% of people living with chronic or genetic health conditions - or who have experienced a premature birth - have faced stigma, judgement or blame due to their health condition.i

Look Beyond the Bias is a Chiesi commitment to challenging harmful biases, sparking national conversation, and reducing health inequalities.

iChiesi UK survey conducted in 2025 of 1,148 people. Data on file.

UK-CHI-2500837 | September 2025

Press releases for media

Highlights

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Chiesi and Bespak partner to advance Carbon Minimal Inhaler production with UK manufacturing site

Highlights Agreement builds on existing Chiesi-Bespak collaboration, reinforcing a trusted relationship as Chiesi works to evolve a portfolio of extrafine formulation Carbon Minimal Inhalers1, ensuring patients retain continuity of care while reducing carbon emissions Bespak's leadership in low-GWP propellant inhaler manufacturing along with significant expansion at its Holmes Chapel site strengthens Chiesi's global supply chain resilience Partnership aligns with the science-based climate commitments of both companies  Parma (Italy) and Holmes Chapel (UK) – 11 March 2026 – Chiesi Group ("Chiesi"), the international research focused biopharmaceutical company and a certified B Corp, and Bespak, the specialist inhalation CDMO focused on pulmonary and nasal drug delivery, today announced an expansion of their long-standing partnership, increasing pressurized metered dose inhaler (pMDI) manufacturing capacity at Bespak's Holmes Chapel site to support the next phase of Chiesi's Carbon Minimal Inhaler (CMI) program. Building on years of collaboration, the agreement reflects a shared long-term vision: delivering lower carbon inhaled therapies through CMIs at scale, without compromising clinical choice or continuity of care for patients. Both companies are committed to addressing climate change through measurable, science-based action. Chiesi's ambition to reach Net Zero targets by 2035, and Bespak's validated decarbonization roadmap, underpin a partnership grounded in shared sustainability principles. Maria Paola Chiesi, Chiesi Group Vice Chair, said: "At Chiesi, sustainability is not an add-on; it is a commitment that guides our strategic choices. We know that inhalers are essential treatments, and that the environmental impact associated with them must be addressed without shifting the burden onto patients. The partnership with Bespak reinforces our efforts to reduce emissions across the value chain, while protecting access, quality and trust. Climate action and patient care must continue to advance hand in hand." To meet the needs of patients and reduce impact on the environment, Chiesi is working to be the only company to offer a portfolio of extrafine formulation Carbon Minimal Inhalers, including both dry powder inhalers (DPIs) and next generation propellant pMDIs. Chiesi's CMI program is designed to significantly reduce the carbon footprint of pMDIs by up to 90%, through the transition to a next-generation, low global warming potential (GWP) propellant, while maintaining established treatment options and device familiarity for patients. Reinforcing the partnership with Bespak adds industrial scale and resilience to Chiesi's journey, supporting a phased and responsible transition. The expanded collaboration further strengthens Bespak's position at the forefront of the global industry's transition to next-generation low GWP propellants, and its Holmes Chapel site as a specialist pMDI manufacturing hub within the global pharmaceutical supply chain. Positioned in the North West of England's inhalation R&D and manufacturing cluster, the site contributes high value skills, advanced technical expertise and long-term investment in sustainable inhaler manufacturing, for the benefit of patients and healthcare systems worldwide. Giuseppe Accogli, Chiesi Group CEO, said: "This agreement strengthens an already established partnership with Bespak, and is a concrete example of how we translate our ambition into action. By working with trusted partners across our value chain, we can deliver sustainable innovation at scale while ensuring that patients receive their needed therapies." Chris Hirst, Bespak CEO, said: "Our collaboration with Chiesi has grown over time around a shared commitment to patient safety, technical excellence and sustainability. By deepening this partnership, we are accelerating the transition to low carbon pMDIs and reinforcing the UK's role as a center of excellence for sustainable inhalation manufacturing. This is a position being recognized by the wider industry, leading to our Holmes Chapel site being selected as a key source of supply by leading brand owners like Chiesi, cementing our role as a strategic supply chain partner for the next generation propellant inhalers and innovative nasally-delivered therapies." About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company's mission is to improve people's quality of life and act responsibly towards both the community and the environment. By adopting the legal form of Benefit Corporation in Italy, the US, France and Colombia, Chiesi's commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group's research and development center in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For more information, visit chiesi.com or the website of your local Chiesi affiliate. About Bespak Bespak is a specialist inhalation contract development and manufacturing organisation (CDMO) focused on pulmonary and nasal drug delivery. Trusted by the world's leading pharmaceutical companies, Bespak delivers end-to-end capabilities across product development, clinical supply and commercial manufacturing of inhaled therapies for global supply. Headquartered in Holmes Chapel, UK, with specialist manufacturing sites in Holmes Chapel and King's Lynn, UK, Bespak develops and supplies pressurised metered dose inhaler (pMDI) products, valves and actuators, complex dry powder inhaler (DPI) devices, nasal products and devices, and supports emerging inhalation technologies, including innovative soft mist systems. Sustainability underpins every step of how Bespak operates and innovates. The company has taken clear and measurable steps to align with key United Nations (UN) Sustainable Development Goals (SDGs), is a signatory of the United Nations Global Compact (UNGC) and has set approved net-zero and near-term company-wide emissions targets with the Science Based Targets initiative (SBTi). Through collaboration and targeted investment, Bespak is accelerating the industry's transition to more sustainable inhaled medicines. Built on a long history of inhalation experience and ready for the future, Bespak is a long-term innovation partner creating lasting impact for patients and the planet. Press Info Chiesi Group Anna Bonisoli Alquati, Head of Global External Communications Email: mediarelations@chiesi.com Michela Lijoi, Global External Communications Sr. Manager Phone: +39 328.6353044 Email: m.lijoi@chiesi.com Bespak Notch Communications Ltd Email: bespak@notchcommunications.co.uk References In a limited number of countries, including the United Kingdom, non‑extrafine products will also be transitioned to CMI.

11/03/2026

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Chiesi Global Rare Diseases and Protalix BioTherapeutics Announce European Commission Approval of Additional Dosing Regimen of Every Four Weeks for Elfabrio® (pegunigalsidase alfa)▼

  Approved dosing regimen reduces the burden for eligible patients, their families, and the broader healthcare system by extending infusion interval from every-two-weeks to every-four-weeks for those stable with an enzyme replacement therapy (ERT) With this decision, announced ahead of Fabry Disease Awareness Month in April, Chiesi Global Rare Diseases will work with countries across the EU to support broader access to this additional dosing schedule for the adult Fabry community PARMA, Italy and CARMIEL, Israel – March 9, 2026 – Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, and Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the discovery, development, production and commercialization of innovative therapeutics for rare diseases with significant unmet needs, today announced that the European Commission (EC) has approved the 2mg/kg every-4-weeks (E4W) dosing regimen for pegunigalsidase alfa in adults living with Fabry disease who are stable with an ERT. The EC decision follows the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending the additional dosing regimen. “The European Commission approval for 2mg/kg body weight E4W dosing regimen for pegunigalsidase alfa represents a meaningful advancement for adults living with Fabry disease and their families,” said Giacomo Chiesi, Executive Vice President, Chiesi Global Rare Diseases. “Because Fabry disease requires lifelong treatment, the cadence of therapy inevitably becomes part of everyday life for patients and caregivers. By introducing an option that extends the infusion interval from every two weeks to every four weeks for eligible patients on stable ERT, we are offering families greater flexibility and the possibility to ease the overall burden of treatment. Ultimately, our goal is simple but profound: to help people spend less time managing their disease and more time living their lives. This milestone reflects our commitment to innovation that goes beyond delivering therapies—by listening to and understanding the real experiences of the Fabry community.” "In Fabry disease, long-term treatment decisions must balance disease management with the realities of lifelong therapy," said Prof. Aleš Linhart, DrSc, FESC. "The approval of pegunigalsidase alfa 2mg/kg every-4-weeks provides an additional option that may help reduce cumulative treatment burden for appropriate patients while maintaining continuity of care." “This approval strengthens the treatment landscape for Fabry disease across the European Union by introducing an additional dosing approach that has the potential to enhance long-term care," said Dror Bashan, President and Chief Executive Officer, Protalix BioTherapeutics. “The authorization reflects not only scientific progress, but also a commitment to optimizing care delivery in a way that supports both patients and healthcare systems.” "For many people living with Fabry disease, treatment is a lifelong commitment that impacts nearly every aspect of daily life," said Mary Pavlou, President, Fabry International Network (FIN). "This approval allows for fewer infusion visits, helping reduce the ongoing burden on patients and families, allowing them to spend more time living their lives beyond treatment." The EC approval is informed by results from an open-label, switch-over study, BRIGHT (formally PB-102-F50), designed to assess the adverse-event profile, efficacy, and pharmacokinetics (PK) of the alternative dosing regimen of pegunigalsidase alfa 2 mg/kg E4W for 52 weeks,1 and its ongoing open-label extension study CLI-06657AA1-03 (formerly PB-102-F51).2 Protalix is entitled to a regulatory milestone payment of $25 million from Chiesi in connection with the EC’s approval of the E4W dosing regimen. About Fabry Disease Fabry disease is a rare, inherited lysosomal storage disorder caused by mutations in the GLA gene, which leads to a deficiency of the enzyme alpha-galactosidase A. This deficiency results in an accumulation of a fatty substance called globotriaosylceramide (GL-3) in the body’s cells, affecting the heart, kidneys, skin, nervous system, and other organs.3 Fabry disease can cause a range of serious signs and symptoms, including fatigue, chronic pain, gastrointestinal issues, decreased ability to sweat, progressive kidney failure, heart complications, and increased risk of stroke.4 The condition affects both males and females and can present from childhood through adulthood, often with delayed diagnosis or misdiagnosis. While Fabry disease is rare, early detection and access to appropriate treatment — such as enzyme replacement therapy or pharmacological chaperone therapy — are critical in managing symptoms and slowing disease progression.3 About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group’s research and development center in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. About Protalix BioTherapeutics, Inc. Protalix is a biopharmaceutical company focused on the discovery, development, production and commercialization of innovative therapeutics for rare diseases. Protalix has researched, developed and currently manufactures two enzyme replacement therapies that are currently available in multiple markets. These therapies are recombinant therapeutic proteins expressed through Protalix's proprietary plant cell-based expression system, ProCellEx®. ProCellEx is a unique plant cell-based system that enables Protalix to produce recombinant proteins in an industrial-scale manner with no exposure to mammalian cells. Protalix is the first company to gain US Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa, Elelyso®, for the treatment of Gaucher disease, excluding in Brazil, where Protalix retains full rights. Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of pegunigalsidase alfa, which was approved by both the FDA and the EMA in May 2023. Protalix's development pipeline includes, among others, two proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets: PRX–115, a plant cell-expressed recombinant PEGylated uricase for the treatment of uncontrolled gout; and PRX–119, a plant cell-expressed long acting DNase I for the treatment of NETs-related diseases. Protalix BioTherapeutics, Inc. Forward-Looking Statements To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms “anticipate,” “believe,” “estimate,” “expect,” “can,” “continue,” “could,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk and the final results of a clinical trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences include, among others: the risk that the EC will not approve the CHMP’s positive opinion recommending approval of the 2mg/kg every-4-weeks (E4W) dosing regimen for pegunigalsidase alfa in adults with Fabry disease; risks related to the commercialization of pegunigalsidase alfa; risks relating to pegunigalsidase alfa market acceptance, competition, reimbursement and regulatory actions, including as a result of the boxed warning contained in the FDA approval received for the product; delays in the approval or potential rejection of any applications filed with the FDA, EMA or other health regulatory authorities for Protalix' s product candidates, and other risks relating to the review process; the risk that the results of clinical trials will not support the applicable claims of safety or efficacy; ; risks relating to changes to published interim, topline or preliminary data from clinical trials; the inherent risks and uncertainties in developing drug platforms and products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies; and risks relating to changes in healthcare laws, rules and regulations in the United States or elsewhere; and other factors described in our filings with the US Securities and Exchange Commission. The statements in this press release are valid only as of the date hereof and Protalix disclaims any obligation to update this information, except as may be required by law. Chiesi Global Rare Diseases Media Contact Sky Striar LifeSci Communications Email: sstriar@lifescicomms.com Protalix BioTherapeutics, Inc. Investor Contact Mike Moyer, Managing Director LifeSci Advisors Phone: +1-617-308-4306 Email: mmoyer@lifesciadvisors.com References Holida, M, et al., (2024). A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme. Journal of Inherited Metabolic Disease. doi:10.1002/jimd.12795. Bernat et al., (2025). Extending the interval between pegunigalsidase alfa infusions in patients with Fabry disease: five-year interim results from the ongoing BRIGHT51 study Abstract presented at ICIEM Congress 2025. Mehta, A., & Hughes, D. A. (2024). Fabry disease. In M. P. Adam, S. Bick, G. M. Mirzaa, et al. (Eds.), GeneReviews®. University of Washington, Seattle. Cleveland Clinic. (2025, October 9). Fabry disease: Symptoms & causes. UK-CHI-2600129 | March 2026

09/03/2026

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Chiesi Marks Key Net Zero Milestone with its First Carbon Minimal pMDI Product Submission to the MHRA for Clenil® Modulite® (beclometasone)

Chiesi announces its first carbon minimal pressurised metered dose inhaler (pMDI) portfolio submission to the UK Medicines and Healthcare products Regulatory Agency (MHRA) for beclometasone (100mcg and 200mcg). Chiesi is the market leader for inhaled therapies for respiratory conditions in the UK1 and intendes to replace the current propellant, hydrofluoroalkane (HFA) 134a, with the next-generation propellant, HFA-152a, across its pMDI inhaler portfolio. This shift is expected to reduce the carbon footprint of Chiesi pMDI inhalers by up to 90%.2 Manchester, UK – 8th December 2025 – Chiesi UK and Ireland has today announced it has submitted beclometasone (100mcg and 200mcg), formulated with a next generation propellant (HFA-152a), to the MHRA. Beclometasone will be the first in Chiesi’s global pMDI product portfolio to transition to the next generation propellant, and marks a significant step forward in Chiesi’s ambition to achieve Net Zero emissions by 2035.   “We are proud to be the market leader for inhaled respiratory therapies in the UK,1driving solutions that benefit both patients and the planet. The submission to the MHRA for use of a next generation propellant within our established beclometasone product marks the first regulatory submission for Chiesi’s carbon minimal inhaler programme anywhere in the world.” said Ralph Blom, General Manager, Chiesi UK and Ireland. “We continue to invest in our inhaler technology and our intention is to fully transition our pMDI portfolio to the new propellant by the end of 2027 in the UK. Our priority is to ensure continued clinical benefit while dramatically reducing our environmental impact.”   Pharmacokinetic studies have demonstrated therapeutic equivalence and tolerability of products formulated with HFA-152a, versus those formulated with HFA-134a, across Chiesi’s pMDI portfolio. Transitioning to the next generation propellant will reduce the carbon footprint of the company’s pMDIs by up to 90% compared to the current ones, while ensuring that patients maintain access to the device that is most effective for them.Following Chiesi’s transition of its pMDI portfolio to the next generation propellant, the NHS’ inhaler carbon footprint is expected to reduce by around 22% (based on current prescribing trends).3 Sarah Rust, Practice Nurse Lead, St Andrews Medical Centre, Salford, commented: “Transitioning to a lower-carbon propellant means we will be able to continue delivering effective care for people who rely on beclometasone, while taking meaningful steps to reduce the environmental impact of pMDIs. For the respiratory community, ensuring patient access to the right treatment remains a priority - and this development with the MHRA moves us forward, supporting both clinical outcomes and the NHS’ sustainability goals.”   Among inhaler types, pMDIs are the most widely used across the UK, accounting for around 70% of the 60 million inhalers prescribed each year.4,5 Today’s milestone was fuelled by Chiesi’s €400 million investment programme to reduce the carbon footprint of its pMDIs – a central part of the company’s commitment to becoming Net Zero by 2035.6 Beclometasone marks the first product to transition to the next generation propellant, with other Chiesi pMDIs to be submitted for regulatory review in the coming months. Darshan Negandhi MRPharmS, PGCert, MAPCPharm, IPresc, Morden PCN Clinical Pharmacist and Trainer, CPPE Tutor - London, added: “Through the introduction of a more sustainable propellant into beclometasone, we’ll be able to uphold high standards of treatment without compromising on environmental responsibility. The transition of the Chiesi pMDI portfolio to the next generation propellant is a positive milestone, and will enable patients to benefit from therapeutic options that positively contribute to the NHS commitment to greener healthcare.”   About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi’s commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we’re part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With over 85 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,000 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For further information please visit www.chiesi.uk.com. Media contact Yasmin Ghariani, Chiesi UK Head of Communications Phone: (+44) 161 488 5555 Email: y.ghariani@chiesi.com References IQVIA BPI HPA, units, MAT October 2025. Panigone S. et al 2020, BMJ Open Respiratory Research: Environmental impact of inhalers for respiratory diseases: decreasing the carbon footprint while preserving patient-tailored treatment. https://doi.org/10.1136/bmjresp-2020-000571. Chiesi. Data on file. NHS England, 2023. Greener NHS: Delivering high quality, low carbon respiratory care. [online] Available at: https://www.england.nhs.uk/greenernhs/2023/02/blog-delivering-high-quality-low-carbon-respiratory-care/. [Last Accessed: December 2025]. Asthma and Lung UK, 2023. How inhalers affect the environment. [online] Available at: https://www.asthmaandlung.org.uk/symptoms-tests-treatments/treatments/inhaler-choices/how-inhalers-affect-environment. [Last Accessed: December 2025]. Chiesi. Data on file.   UK-CHI-2600030 | January 2026 

03/02/2026

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Procysbi® (gastro-resistant mercaptamine) recommended for use in NHS Scotland for the treatment of nephropathic cystinosis¹

Procysbi® (gastro-resistant mercaptamine) has been accepted for use within NHS Scotland for the treatment of proven nephropathic cystinosis, providing a new treatment option for this ultra-rare condition.1 Nephropathic cystinosis is typically diagnosed in infants and children, affecting around 1 in 100,000 – 200,000 people globally.2,3 While gastro-resistant mercaptamine is now available to eligible patients in Scotland and Wales – the treatment remains unavailable in England via the NHS. Manchester, UK – 10 November 2025 – Chiesi UK and Ireland today welcome the Scottish Medicines Consortium’s (SMC) decision to recommend gastro-resistant mercaptamine (a cystine-depleting agent) for use within NHS Scotland for the treatment of people living with nephropathic cystinosis.1 This recommendation follows a detailed appraisal process, where clinicians and patient organisations highlighted the significant burden of currently available treatment and the potential for gastro-resistant mercaptamine to address significant unmet needs in the day-to-day management of the condition. Care for people with nephropathic cystinosis is often complex, time-consuming and demanding for both patients and their families. Until now, treatment options have been limited, requiring multiple daily doses. This can be particularly challenging for children and teenagers, disrupting sleep, schooling and social life, placing additional strain on families and caregivers. Ben Reynolds, Consultant Paediatric Nephrologist, Royal Hospital for Children, Glasgow, said: “The availability of mercaptamine bitartrate in Scotland represents an important step forward in expanding treatment choice for people living with nephropathic cystinosis. It gives clinicians greater flexibility to individualise care, while decisions should continue to be guided by each patient’s clinical profile and circumstance.” Nephropathic cystinosis is a rare, progressive, life-limiting condition that is usually diagnosed in infants and children, affecting around 1 in 100,000 – 200,000 people globally.1,3 The disease is caused by the accumulation of cystine – an amino acid – within cells, leading to tissue and organ damage, particularly in the kidneys.4 David Garzón, Senior Director, Rare Diseases at Chiesi UK and Ireland, added: “We welcome this positive development for patients in Scotland, however we recognise that work must continue to ensure equitable access across the UK. Delayed-release mercaptamine bitartrate has been available to patients in Wales since 2021 and in Ireland for several years, yet in England the process remains stalled. As a result, England remains the only nation in the UK where eligible patients cannot routinely access this medicine, highlighting a growing postcode lottery in care for people living with cystinosis.” Gastro-resistant mercaptamine has been approved across the UK and Europe including availability in France, Italy, Germany and Ireland. It has been available via the NHS in Wales since 2021. The treatment remains unavailable in England, where routine access is still pending. Funding for NHS use in Scotland is expected within 60 days of publication of SMC advice. Comments from the Patient Community Alex Hutchison, Scottish Trustee of Cystinosis Foundation UK, said: “This approval marks a huge moment for people living with cystinosis in Scotland. Having the choice to move from a gruelling six-hourly medication schedule to a more manageable twelve-hourly routine will have a significant impact on quality of life, as well as potentially bringing about clinical improvements for patients. For families, young people, and adults navigating the relentless demands of this rare condition, this decision is an important step forward. We’re proud to have worked both with and for the cystinosis community in advocating for this change.” Laura Smith Van Carroll, Head of Insight & Advocacy at Metabolic Support UK, said: “After many years of hard work and perseverance, we are thrilled to see a positive reimbursement decision for gastro-resistant mercaptamine in Scotland. This means so much to the cystinosis community, who have waited far too long for fair access to a treatment that can truly make a difference to daily life. Cystinosis may be rare, but its impact is immense - the current six-hourly treatment disrupts sleep, family life and wellbeing every single day. Gastro-resistant mercaptamine offers the chance for people living with cystinosis, and their carers, to reclaim normality and rest, without compromising on treatment. We are incredibly proud of the community’s voice, through our surveys and advocacy, which helped bring this evidence to light and made sure their experiences were heard.” “This is positive news for people living with this rare disease and their families.” explained Alison Railton, Director of Policy and Public Affairs at Kidney Research UK. “Having access to this treatment could make a meaningful difference, particularly for children and their parents. The twice-daily dosing means families no longer need to wake during the night, supporting a more consistent night’s sleep. We believe everyone living with cystinosis should have access to the most effective treatments available to prevent them from developing kidney disease and eventually needing dialysis or a kidney transplant.” About Procysbi® (gastro-resistant mercaptamine) Procysbi (gastro-resistant mercaptamine) is a beaded, enteric-coated, delayed-release form of cysteamine, in which the microspheronised beads are further encapsulated in hard gelatin to allow oral administration every 12 hours.5 The treatment is administered twice-daily, aiming to reduce treatment burden for patients and carers.5 Gastro-resistant mercaptamine is available in either 25 mg or 75 mg gastro-resistant hard capsules of cysteamine.6 Therapy should be initiated as early as possible following diagnosis and continued throughout life.7 About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi’s commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With over 85 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,000 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For further information please visit www.chiesi.uk.com. Media contacts Yasmin Ghariani, Chiesi UK and Ireland Head of External Communications Phone: (+44) 161 488 5555 Email: y.ghariani@chiesi.com References Scottish Medicines Consortium. Available at: https://scottishmedicines.org.uk/medicinesadvice/mercaptamine-procysbi-3rd-resubmission-smc2824/ [Last accessed November 2025]. National Kidney Foundation. Nephropathic Cystinosis. Available at: https://www.kidney.org/kidneytopics/nephropathic-cystinosis [Last accessed November 2025]. National Organization for Rare Disorders (NORD). Cystinosis. Available at: https://rarediseases.org/rare-diseases/cystinosis/ [Last accessed November 2025]. Cherqui S. Cysteamine therapy: a treatment for cystinosis, not a cure. Kidney Int 2012;81:127-9. Langman CB, et al. A Randomized Controlled Crossover Trial with Delayed-Release Cysteamine Bitartrate in Nephropathic Cystinosis: Effectiveness on White Blood Cell Cystine Levels and Comparison of Safety. Clin J Am Soc Nephrol. 2012;7:1112–20. [Erratum in Clin J Am Soc Nephrol. 2013;8:468]. All Wales Medicines Strategy Group, Final Appraisal Recommendation Advice number: 0922, April 2022. Ariceta G, Giordano V, Santos F. Effects of long-term cysteamine treatment in patients with cystinosis. Pediatr Nephrol. 2019 Apr;34(4):571-578. UK-PCB-2500005 | November 2025

10/11/2025

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Chiesi Launches 'Look Beyond the Bias' with Olympian Laura Kenny, Patient Groups and Healthcare Professionals, as 70% Report Hiding Health Conditions Due to Fears of Judgement

New survey data from over 1,000 people across the UK reveals that 70% have experienced stigma, judgement or blame based on their health condition – with over half avoiding medical help as a result1 ‘Look Beyond the Bias’ aims to tackle harmful biases, spark national conversation, and reduce health inequalities The campaign highlights the damaging impact of bias – silencing people, delaying diagnosis and leaving certain conditions overlooked, with serious consequences Manchester, UK - Monday 6th October - Chiesi UK and Ireland today launch Look Beyond the Bias, to shine a light on the harmful impact of stigma and bias faced by people living with health conditions. The campaign is informed by new UK survey data from 1,148 people living with chronic or rare conditions, including: asthma, chronic obstructive pulmonary disease (COPD), rare diseases, those who have undergone organ transplants and parents of children born prematurely. It demonstrates a stark warning: bias may impact access to fair and inclusive care. Key findings from the survey include: 70% have experienced stigma, judgement or blame based on their health condition1 57% have avoided seeking medical support due to stigma or bias1 70% have isolated themselves from loved ones for fear of judgement1 82% of young adults have experienced bias - almost twice the rate of over-55s1 79% say they’ve played down the impact of their condition1 Over 60% of people have hidden their condition from colleagues or their employer1 The campaign shows how stigma and bias cut across every part of life, with those surveyed saying it plays a role in delaying diagnosis, limiting research funding and leaves some conditions overlooked and deprioritised.1 This is particularly true for conditions such as COPD and asthma, where the UK has poorer disease outcomes than in other comparable countries, high rates of emergency admissions, falling diagnosis rates, and significant preventable deaths. , In liver disease, deaths also continue to rise, now exceeding 11,000 annually - four times higher than 50 years ago.4 Dame Laura Kenny comments: “After being diagnosed with asthma at a young age, I experienced the same assumptions that many people do when diagnosed with respiratory conditions. Chiesi’s Look Beyond the Bias campaign highlights how stigma can affect so many of us, and why it’s so important to speak up. Health related bias shouldn’t be a barrier for anyone and by shining a light on it through this campaign, we can help ensure that everyone feels valued and empowered with the confidence to get the care and support they deserve.” Look Beyond the Bias brings together patient organisations, healthcare professionals and advocacy partners to call out harmful assumptions, elevate patient voices and advocate for inclusive, equitable care. The campaign features powerful stories and imagery from people living with health conditions, showing the real impact of stigma on health, work and everyday life. Jonathan Blades, Director of External Affairs at Asthma + Lung UK, said: “Stigma around lung conditions is too often ignored, but the damage it causes is far reaching. Our research shows people with lung conditions often feel judged as lazy or unfit, avoided on public transport because of their cough, or too embarrassed or guilty to seek the medical help they need. With 12 million people in the UK expected to develop a lung condition in their lifetime, we must break down these barriers so everyone can access the care they need and deserve.” The launch of Look Beyond the Bias marks the start of a long-term commitment to tackling the stigma uncovered in the survey. Chiesi is working with the NHS, patient groups and the wider healthcare community to champion more inclusive approaches to care. This includes initiatives such as the FRONTIER clinic in Hull and the COMET clinic in Wirral, which support earlier diagnosis of COPD in communities facing high risk and social deprivation. Ralph Blom, General Manager, Chiesi UK and Ireland, commented: “Look Beyond the Bias is our commitment to tackling harmful biases that exist in the UK that are deeply affecting the patients we serve. The stigma, judgement and blame that people feel on a regular basis is widespread and deep-rooted, and we’re proud to be working with partners across the community and NHS to address this challenge. At Chiesi, we feel strongly that no one should be made to feel ashamed, dismissed or blamed because of their health, and this belief sits at the heart of Look Beyond the Bias.” For more information about Look Beyond the Bias, please visit www.chiesi.uk.com/look-beyond-the-bias. About Look Beyond the Bias Look Beyond the Bias has been developed in response to national research conducted by Chiesi UK and Ireland which reveals just how deeply bias is embedded into the everyday lives of people living with chronic and genetic diseases. In a survey of 1,148 people across the UK, 70% said they had experienced stigma, judgement or blame due to their health condition.1 Bias was reported across a wide range of conditions, from asthma and COPD to premature birth, rare diseases and liver and kidney transplant, shaping not only how people are perceived, but also what support they access and how their conditions are systematically deprioritised.1 This bias has tangible consequences, leading to poorer health outcomes, increased strain on NHS resources and wider economic impacts such as unemployment.5,6,7 For further information, please visit www.chiesi.uk.com/look-beyond-the-bias. About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi’s commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we’re part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With over 85 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,000 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For further information, please visit www.chiesi.uk.com. Media contacts Yasmin Ghariani, Chiesi UK Head of External Communications Phone: (+44) 161 488 5555 Email: y.ghariani@chiesi.com References Chiesi UK survey conducted in 2025 of 1,148 people. Data on file. Asthma + Lung UK. Asthma care crisis: charity sounds siren as asthma death toll rises. Available at: https://www.asthmaandlung.org.uk/media/press-releases/asthma-care-crisis-charity-sounds-siren-asthma-death-toll-rises. Last accessed September 2025. Asthma + Lung UK. Lung conditions kill more people in the UK than anywhere else in Western Europe. Available at: https://www.asthmaandlung.org.uk/media/press-releases/lung-conditions-kill-more-people-uk-anywhere-western-europe. Last accessed September 2025. British Liver Trust. Liver disease in numbers. Available at: https://britishlivertrust.org.uk/information-and-support/statistics/. Last accessed September 2025. Mathioudakis AG, Ananth S, Vestbo J. Stigma: an unmet public health priority in COPD. The Lancet Respiratory Medicine. Available at: https://www.thelancet.com/journals/lanres/article/PIIS2213-2600(21)00316-7/fulltext. Last accessed September 2025. Thomas, T. (2024, July 23). ‘National disgrace’: Black mothers in England twice as likely to have NHS birth investigated. The Guardian. Available at: https://www.theguardian.com/society/article/2024/jul/23/national-disgrace-black-mothers-in-england-twice-as-likely-to-have-nhs-birth-investigated. Last accessed September 2025. Van Beukering IE, Smits SJC, Janssens KME, Bogaers RI, Joosen MCW, Bakker M, van Weeghel J, Brouwers EPM. In what ways does health related stigma affect sustainable employment and well-being at work? A systematic review. Journal of Occupational Rehabilitation. 2022;32:365–379. Available at: https://link.springer.com/article/10.1007/s10926-021-09998-z.Last accessed September 2025. UK-CHI-2500840 | September 2025

07/10/2025

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Chiesi Group and Arbor Biotechnologies Announce a Global Strategic Partnership to Develop Novel Rare Disease Gene Editing Programmes

- Exclusive collaboration and license agreement for the development and global commercialisation of Arbor’s clinical-stage gene editing programme, ABO-101 for primary hyperoxaluria type 1 (PH1) - Multitarget research collaboration and option agreement for Arbor’s innovative gene editing technologies for the development of additional rare liver disease targets - Arbor eligible to receive up to $115M in upfront and near-term payments, along with $2B in potential development, regulatory and commercial milestones and low double-digit tiered royalties for products licensed under the agreements PARMA, ITALY & CAMBRIDGE, MA, USA – October 6, 2025 - Chiesi Group, an international research-oriented biopharmaceutical company, together with Arbor Biotechnologies, Inc., a next-generation biotechnology company discovering and developing innovative approaches in genetic medicines, today announced an exclusive global collaboration and license agreement for Arbor’s clinical stage programme ABO-101 in PH1 together with a multitarget option agreement for the use of Arbor’s advanced gene editing platform technology to develop novel liver targeted therapies for rare diseases. At Chiesi Group, these programmes are spearheaded by Chiesi Global Rare Diseases, the Group’s dedicated business unit focused on research, development, and commercialisation of therapies for rare and ultra-rare conditions. "This collaboration marks a transformative moment—not just for us, but for the entire rare disease community. It reflects our commitment to working towards more comprehensive therapeutic options. Achieving this means looking beyond current approaches and exploring the potential of gene editing." said Giacomo Chiesi, Executive Vice President, Chiesi Global Rare Diseases. "While this path holds immense promise, we know there is still a long journey ahead, and much to learn. That’s why we are proud to partner with Arbor Biotechnologies, a leader in the gene editing space with proven expertise in clinical development. This strategic collaboration brings together complementary strengths and reinforces our unwavering mission: to bring lasting hope to patients and their families through meaningful innovation.” “We're proud to join forces with Chiesi, a company that shares our deep commitment to improving outcomes for patients with rare and life-threatening diseases. Chiesi brings a strong track record in rare disease innovation, combined with our platform of advanced gene editors, we aim to deliver significant solutions that can redefine care for patients living with PH1 and other rare genetic diseases," said Devyn Smith, PhD, CEO of Arbor Biotechnologies. Under the terms of the transaction, Chiesi will receive exclusive rights to develop and commercialise ABO-101 for PH1 and the option to leverage Arbor’s knockout (KO) and reverse transcriptase (RT) editing technology to advance additional rare disease targets, providing Arbor upfront and near-term payments of up to $115M. In addition, Arbor is eligible to receive up to $2B in total milestone payments and up to low double-digit tiered royalties. Arbor and Chiesi will collaborate on the ongoing Phase 1/2 redePHine (NCT06839235) clinical study of ABO-101 in PH1. “Genomic medicines offer vast potential to impact the lives of patients around the world, especially those living with rare genetic diseases. We look forward to partnering with Chiesi’s experienced and committed team to help accelerate ABO101 in the clinic and advance development of liver-targeted gene editing therapeutics for patients with PH1 and other rare diseases,” said Dan Ory, MD, Chief Medical Officer of Arbor Biotechnologies. About Primary Hyperoxaluria Type I (PH1) Primary hyperoxaluria type 1 is an ultra-rare lifelong genetic disease, where a mutation in the AGXT gene leads to an enzyme deficiency in the liver resulting in an overproduction of oxalate by the liver and eventual buildup of oxalate crystals in the kidney and other organ systems. As the disease progresses, it can cause recurring kidney stones, kidney damage and eventually lead to end-stage kidney disease (ESKD) and systemic oxalosis. While two siRNA therapies are available for lifelong treatment, the only cure for PH1 is a dual liver and kidney transplant. About ABO-101 ABO-101 is a novel, investigational gene editing medicine designed to be a onetime liver-directed gene editing treatment that potentially results in a permanent loss of function of the HAO1 gene in the liver to reduce PH1-associated oxalate production. ABO-101 is currently being evaluated for PH1 in the redePHine Phase 1/2 clinical study (NCT06839235). ABO-101 consists of a lipid nanoparticle (LNP), licensed from Acuitas Therapeutics, encapsulating messenger RNA expressing a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA which specifically targets the human HAO1 gene. ABO-101 has not been approved for any use by the FDA or any other regulatory agency. About the redePHine Study The Phase 1/2 redePHine study is an open label global multi-centre dose escalation study to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of a single dose of ABO-101 in participants with primary hyperoxaluria type 1. The trial will consist of 2 Study Periods. During the first Study Period, there will be 2 parts. In Part A, adult participants will be treated with a single ascending dose to identify a recommended dose. In Part B, paediatric participants will be treated with the recommended dose. Following the first Study Period, participants will start Study Period 2, a long-term monitoring programme to comply with local and national requirements. About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment. By changing its UK-CHI-2500941 | September 2025 legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. About Chiesi Global Rare Diseases Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system. About Arbor Biotechnologies, Inc. Arbor Biotechnologies, a clinical stage, next-generation gene editing company based in Cambridge, MA, is advancing a pipeline of novel gene editing therapeutics to address a wide range of genetic conditions – from the ultra-rare to the most common genetic diseases. The company’s unique suite of optimized gene editors, which is capable of approaches ranging from gene knockout, excisions, reverse transcriptase editing, and large gene insertion, goes beyond the limitations of early editing technologies to unlock access to new gene targets and has fueled a robust pipeline of first-in-class assets focused on diseases of high unmet need. With Arbor’s clinical programme, ABO-101 for the treatment of primary hyperoxaluria type 1, the company continues to focus its research and development efforts on genomic diseases of the liver and CNS for which there are no existing functional cures. Chiesi Global Rare Diseases Media Contact Sky Striar LifeSci Communications sstriar@lifescicomms.com Arbor Biotechnologies Media Contact: Peg Rusconi Deerfield Group prusconi@deerfieldgroup.com UK-CHI-2500941 | September 2025

07/10/2025

COMMITTED TO A SUSTAINABLE FUTURE

Sustainability has been woven into our company mission for more than 15 years. As a B Corp certified company since 2019, we're part of a global community using our business as a force for good, and we're committed to becoming Net Zero by 2035.

We report on our progress annually as part of one single publication demonstrating our financial, social and environmental data.

Details of Chiesi’s clinical trials can be found at www.clinicaltrials.gov

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