Latest news with #rareDiseases
Reuters
11 hours ago
- Business
- Reuters
Italy's Chiesi Group plans acquisitions to boost sales to 6 billion euros by 2030
PARMA, Italy June 5 (Reuters) - Italy's Chiesi Group is aiming to significantly increase the number of patients it treats from around 10 million and its sales to 6 billion euros ($6.9 billion) by 2030, helped by acquisitions, its CEO Giuseppe Accogli said in an interview. The family-owned group, which provides treatment for rare diseases and respiratory conditions such as asthma and COPD, is scouting the market to see if there are opportunities to buy a new company to help achieve its ambition of doubling sales, but says there is no rush. "We are looking in respiratory, in specialty pharma and in rare diseases, not only in Italy, but also globally. I think every year is a good year if we find a good asset that will fit strategically with our portfolio," Accogli said. The last major acquisition by Chiesi dates back to 2023 when it bought rare diseases specialist Amryt for some $1.5 billion, shortly before Accogli took the CEO role. He declined to comment on whether the group has approached listed Italian rival Recordati ( opens new tab in the recent past. "I don't think we can share that, but we always look at everything that is available," he said in an interview at the group's headquarters in Parma, northern Italy. Chiesi has no debt and earns around 1 billion euros each year in EBITDA so the company "has firepower to grow both organically and inorganically and to reach between five and six billion euros of sales with our own resources," Accogli underlined. Regarding the sales goal, around 10-15% should come through inorganic opportunities "that we are scouting". The current year has started well and the CEO is confident of ending it with revenue "growth of more than half single digits" compared to 13% growth reached in 2024 at 3.4 billion euros. On the U.S. tariff front, Accogli said that the situation is still very confused. "Many things have been said. Right now, none of them have gone into action, which doesn't mean that they will not. So we keep monitoring and see. But things change by the day." The CEO is aware that meeting patients' growing therapeutic demand may eventually require establishing a manufacturing presence in the U.S to complement existing sites in France, Italy and Brazil but nothing concrete is planned for the moment. ($1 = 0.8754 euros)
Khaleej Times
2 days ago
- Business
- Khaleej Times
Majid and Lynn Jafar honoured by top US university
The University of Massachusetts Chan Medical School has honoured Majid Jafar, CEO of Crescent Petroleum, and his wife Lynn Barghout Jafar, co-founders of the Loulou Foundation, for their work in advancing medical research and innovation and for supporting children with special needs through the High Hopes Therapy Center in the UAE. The honorary doctorate degrees, presented at the university's 2025 Commencement ceremony by Chancellor Michael Collins, highlight their role in accelerating scientific and therapeutic progress in the field of rare diseases — an area that remains significantly underdiagnosed and underserved, with the vast majority of conditions still lacking approved treatments. More than 350 million people are affected by rare diseases worldwide, with 70% of them children. While over 7,000 rare diseases have been identified, 95% still lack approved treatments. Majid Jafar commented: 'We are deeply honoured by this recognition from UMass Chan Medical School. It affirms what can be achieved when purpose-driven collaboration meets urgency — especially in the field of rare diseases, where so many families are still waiting for new treatments. The UAE has long recognised the importance of medical innovation, and we remain committed to contributing to this global effort to improve children's lives and accelerate meaningful change.' Umass Chan Medical School is among the leading medical schools in the United States and last year entered into a strategic partnership agreement with Mohammed bin Rashid University (MBRU) of Medicine and Health Sciences in Dubai. The commencement ceremony also conferred honorary doctorate degrees on Dr Marcia McNutt, President of the U.S. National Academy of Sciences, and Dr Carolyn Clancy, Assistant Under Secretary for Health at the U.S. Veterans Health Administration. Lynn Barghout Jafar, co-founder of the Loulou Foundation and Founder of the High Hopes Therapy Center in Dubai, added: 'When our own daughter was diagnosed with a rare disease, what began as a personal journey quickly became a global mission to connect families and scientists in the pursuit of answers. We are grateful to be part of a growing community that is transforming the future of rare disease research through science, collaboration, and hope'. In March this year Majid and Lynn Jafar personally hosted a Charity Suhoor in support of Al Jalila Foundation's Child Fund in the UAE, raising Dh50 million — the largest donor-led fundraiser since the Foundation's inception in 2013. Held under the theme 'An Evening of Hope', the gathering brought together business leaders and philanthropists to support life-saving medical treatment and research for children in need. In addition to his work with Loulou Foundation which as supported more than 100 research projects in labs around the world since its founding in 2015, Majid Jafar co-chairs the international campaign for Cambridge Children's Hospital, serves on the advisory board of the Oxford-Harrington Rare Disease Centre — chaired by former UK Prime Minister David Cameron — and is a member of the Board of Fellows and co-chairs the Discovery Council at Harvard Medical School. In his professional capacity, in addition to serving as CEO of Crescent Petroleum, the Middle East's oldest private oil and gas company, Majid Jafar is the Board Managing Director of Dana Gas and Vice Chairman of the Crescent Group, headquartered in Sharjah in the UAE. Lynn Barghout Jafar established High Hopes Pediatric Therapy Center in 2017 as an early intervention facility in the UAE that caters to over 300 children with moderate to complex special needs - a place where they can develop, progress, and also have fun under the supervision of the most experienced and specialized therapists.
Reuters
2 days ago
- Business
- Reuters
Sanofi to buy US biopharma group Blueprint for up to $9.5 billion
PARIS, June 2 (Reuters) - France's Sanofi has agreed to buy U.S.-based Blueprint Medicines Corporation for up to $9.5 billion to boost its position in rare immunology diseases, in the biggest deal struck by a European healthcare company so far this year, according to LSEG data. Blueprint (BPMC.O), opens new tab is a specialist in treatments for systemic mastocytosis, a rare blood disorder. Sign up here. The two companies said on Monday that Sanofi ( opens new tab would initially pay $129.00 per share in cash, or around $9.1 billion. Blueprint shares jumped 27% to $128.74 in premarket trade. Sanofi stock was down about 1%. Sanofi has ramped up research and development spending in recent years, prompting the company to abandon its long-term profit margin targets two years ago, as it seeks to build on the success of its blockbuster drug Dupixent for eczema and other conditions. However, it suffered a setback last week after an experimental drug for patients with a lung condition commonly called "smoker's lung" failed a late-stage trial. The Blueprint acquisition "represents a strategic step forward in our rare and immunology portfolios. It enhances our pipeline and accelerates our transformation into the world's leading immunology company," said Sanofi CEO Paul Hudson. The deal would add to Sanofi's portfolio the rare immunology disease drug Ayvakit, also known as Ayvakyt, approved in the U.S. and the EU, and a promising advanced and early-stage immunology pipeline. Ayvakit is the only approved medicine for advanced and indolent systemic mastocytosis, a rare blood disorder that occurs when the body makes abnormal mast cells - a type of white blood cell. It triggers a continuous allergic response. The acquisition would also bring elenestinib, a next-generation medicine for systemic mastocytosis, as well as BLU-808, a highly selective and potent oral wild-type KIT inhibitor that has the potential to treat a broad range of diseases in immunology. The deal makes "strategic and financial sense", said JP Morgan analysts in a note, noting that Blueprint expects Ayvakit to reach annual sales of around $2 billion by fiscal year 2030. "We see the transaction as a good fit for Sanofi at a sensible valuation, which investors should see as a positive with time," they added. It is the latest in a series of deals by Sanofi. Last month, it announced the $470 million purchase of Vigil Neuroscience (VIGL.O), opens new tab and in January 2024 it struck a $2.2 billion deal for U.S biotech firm Inhibrx. Hudson said the deal complemented Sanofi's recent acquisitions of other early-stage medicines and added that it still retained a sizable capacity for further deals. The company has said it plans to invest at least $20 billion in the United States through 2030 to boost manufacturing and research, joining other drugmakers in responding to President Donald Trump's drive to boost local manufacturing. Besides $129.00 per share in cash, Blueprint shareholders would also receive one non-tradeable contingent value right (CVR) per share, which would entitle the holder to receive two potential milestone payments of $2 and $4 per CVR for the achievement, respectively, of future development and regulatory milestones for BLU-808. The total equity value of the transaction, including potential CVR payments, is $9.5 billion on a fully diluted basis. It is expected to close in the third quarter, pending regulatory and shareholder approvals.
Associated Press
2 days ago
- Business
- Associated Press
National MPS Society Welcomes Sharon King as Chief Operating Officer (COO)
'We are honored to welcome Sharon King as our new Chief Operating Officer. Sharon brings unmatched experience, heartfelt purpose, and a legacy of advocacy that resonates deeply with our mission.'— Terri Klein DURHAM, NC, UNITED STATES, June 3, 2025 / / -- The National MPS Society is delighted to announce that Sharon King has joined the team as our inaugural Chief Operating Officer (COO). Sharon comes to us with over two decades of experience spanning rare disease nonprofits, industry, and patient advocacy. She is a passionate leader who has dedicated her life to serving patient communities and driving meaningful change in the field of rare diseases. Terri L. Klein, President and CEO at the National MPS Society, shared, 'We are honored to welcome Sharon King as our new Chief Operating Officer. Sharon brings unmatched experience, heartfelt purpose, and a legacy of advocacy that resonates deeply with our mission. Her leadership is shaped by her lived experience and a deep understanding of the challenges faced by families with rare diseases. Her ability to lead with both strategy and compassion will strengthen every facet of our work. I am confident that Sharon will help the Society reach new heights as we continue to serve and support our families. We are thrilled to welcome her and look forward to the strength she will bring to the Society.' Sharon is a longtime patient advocate and thought leader in rare disease, known for her ability to unite public officials, researchers, biotech representatives, industry leaders, and other advocates in the pursuit of rare disease treatment development and impactful public policies. Her journey in advocacy began with the founding of Taylor's Tale, an organization she co-founded in honor of her late daughter, Taylor, to raise awareness and funding for CLN1 disease (a form of Batten Disease) research. Under her leadership, Taylor's Tale grew from a grassroots initiative into a force in the fight against rare diseases. Beyond her work with Taylor's Tale, Sharon has served in key roles that reflect her dedication to improving the lives of rare disease patients and caregivers. As Senior Lead of Advocacy at Aldevron, she worked to educate, connect, and improve understanding across the stakeholder community contributing to groundbreaking advancements. Sharon is a state-appointed member of the N.C Advisory Council on Rare Diseases. The law that established the Council—the nation's first—is named for her daughter. She is also a former member of the ASGCT Patient Education committee and serves on the Emily Whitehead Foundation Board of Directors. Her work reflects her deep understanding of the unique challenges faced by rare disease patients and their caregivers, as well as her unwavering commitment to promoting patient-centered, integrated care networks. Her efforts are grounded in personal experience and driven by a vision of improving lives through collaboration, education, and innovation. As Chief Operating Officer, Sharon King brings a powerful combination of strategic leadership, lived experience, and deep compassion to the National MPS Society. Her expertise will be instrumental in advancing our mission to support individuals and families affected by MPS and ML. With her unwavering commitment to advocacy and innovation, Sharon will help guide our efforts to expand critical services, drive research, and strengthen the communities we serve. This strategic appointment comes at a pivotal moment as the Society works to close the treatment gap for all families impacted by MPS and ML. With Sharon in this vital role, our team is better positioned to move forward with intention—developing new resources, enhancing care pathways, and ensuring families are never alone in their journey from diagnosis to treatment and beyond. The National MPS Society remains a steadfast partner for the road ahead. Together, we will work toward a brighter future for those we serve. Terri Klein National MPS Society +1 919-806-0101 email us here Visit us on social media: LinkedIn Instagram Facebook YouTube Legal Disclaimer: EIN Presswire provides this news content 'as is' without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.
Globe and Mail
3 days ago
- Business
- Globe and Mail
France's Sanofi to buy U.S. biopharma group Blueprint for up to $9.5-billion
France's Sanofi SNY-Q has agreed to buy U.S.-based Blueprint Medicines Corporation BPMC-Q for up to US$9.5-billion to boost its position in rare immunology diseases, in the biggest deal struck by a European health care company so far this year, according to LSEG data. Blueprint is a specialist in treatments for systemic mastocytosis, a rare blood disorder. The two companies said on Monday that Sanofi would initially pay US$129.00 per share in cash, or around US$9.1-billion. Blueprint shares jumped 27 per cent to US$128.74 in premarket trade. Sanofi stock was down about 1 per cent. Sanofi has ramped up research and development spending in recent years, prompting the company to abandon its long-term profit margin targets two years ago, as it seeks to build on the success of its blockbuster drug Dupixent for eczema and other conditions. However, it suffered a setback last week after an experimental drug for patients with a lung condition commonly called 'smoker's lung' failed a late-stage trial. The Blueprint acquisition 'represents a strategic step forward in our rare and immunology portfolios. It enhances our pipeline and accelerates our transformation into the world's leading immunology company,' said Sanofi CEO Paul Hudson. The deal would add to Sanofi's portfolio the rare immunology disease drug Ayvakit, also known as Ayvakyt, approved in the U.S. and the EU, and a promising advanced and early-stage immunology pipeline. Ayvakit is the only approved medicine for advanced and indolent systemic mastocytosis, a rare blood disorder that occurs when the body makes abnormal mast cells – a type of white blood cell. It triggers a continuous allergic response. The acquisition would also bring elenestinib, a next-generation medicine for systemic mastocytosis, as well as BLU-808, a highly selective and potent oral wild-type KIT inhibitor that has the potential to treat a broad range of diseases in immunology. The deal makes 'strategic and financial sense,' said JP Morgan analysts in a note, noting that Blueprint expects Ayvakit to reach annual sales of around $2-billion by fiscal year 2030. 'We see the transaction as a good fit for Sanofi at a sensible valuation, which investors should see as a positive with time,' they added. It is the latest in a series of deals by Sanofi. Last month, it announced the US$470-million purchase of Vigil Neuroscience and in January, 2024, it struck a US$2.2-billion deal for U.S biotech firm Inhibrx. Hudson said the deal complemented Sanofi's recent acquisitions of other early-stage medicines and added that it still retained a sizable capacity for further deals. The company has said it plans to invest at least US$20-billion in the United States through 2030 to boost manufacturing and research, joining other drugmakers in responding to President Donald Trump's drive to boost local manufacturing. Besides US$129.00 per share in cash, Blueprint shareholders would also receive one non-tradeable contingent value right (CVR) per share, which would entitle the holder to receive two potential milestone payments of US$2 and US$4 per CVR for the achievement, respectively, of future development and regulatory milestones for BLU-808. The total equity value of the transaction, including potential CVR payments, is US$9.5-billion on a fully diluted basis. It is expected to close in the third quarter, pending regulatory and shareholder approvals.
