Castleman Disease

PHILADELPHIA, Oct. 9, 2024 /PRNewswire/ -- Every Cure, an AI-powered nonprofit biotech organization, has received a five-year $60 million commitment through TED's Audacious Project to advance promising repurposed treatments to patients around the world. This support will enable Every Cure to evaluate the most promising treatments identified by its ARPA-H-funded AI platform in laboratory and clinical studies, with the goal of democratizing access to safe and effective therapies for patients worldwide. Over the next five years, Every Cure will collaborate with patient organizations and stakeholders linked to the most promising drug repurposing opportunities, to ensure that these treatments reach the patients that need them the most. Ultimately, Every Cure aims to transform the global treatment landscape, improve health outcomes, and enhance the quality of life for countless individuals. Every Cure was founded in 2022 by Dr. David Fajgenbaum, Dr. Grant Mitchell, and Tracey Sikora to amplify the impact they had been making at the University of Pennsylvania in repurposing drugs for rare diseases, including the successful repurposing of a drug for Castleman disease that ultimately saved Dr. Fajgenbaum's life. "This is such a major step in our journey to unlock the life-saving potential of existing drugs," said Dr. David Fajgenbaum, President of Every Cure. "I'm alive because of a repurposed drug and we've been able to save the lives of other patients with repurposed drugs over the last 10 years. This funding will help us to save many more lives all around the world." In parallel to the repurposing efforts led by Dr. Fajgenbaum at the University of Pennsylvania, Dr. Mitchell and his colleagues on Every Cure's technology team were pioneering the use of machine learning for drug discovery in partnership with biopharmaceutical companies, and they are now poised to scale these innovative approaches. "We believe that repurposing existing treatments is the fastest and most efficient approach to addressing critical health challenges and maximizing the return on investment for saving lives," said Dr. Grant Mitchell, CEO of Every Cure. Of the world's approximately ~18,500 recognized diseases, only ~4,000 have FDA approved treatments, leaving hundreds of millions of people worldwide without effective therapies for their conditions. Strikingly, over 80% of currently FDA-approved treatments are generic or off-patent and could hold the key to new treatments. Tragically, systemic barriers and a lack of financial incentives hinder further research on these medications, resulting in these life-saving drugs remaining underutilized on pharmacy shelves while patients continue to suffer from limited or no treatment options. To address these challenges, Every Cure systematically identifies and advances promising repurposing opportunities by leveraging its comprehensive AI data engine, which generates predictive efficacy scores for FDA-approved drugs across all diseases. The funding catalyzed through The Audacious Project will strengthen this capability by supporting essential research and clinical initiatives, ensuring rapid access to identified therapies for patients in need, and accelerating the path from discovery to treatment. The Audacious Project is a collaborative funding initiative housed within TED that empowers changemakers to transform their innovative ideas for solving the world's greatest challenges into viable multi-year initiatives. Each year, they partner with visionary philanthropists to launch these initiatives into the world. In addition to financial backing, support through TED's Audacious Project will significantly raise awareness of Every Cure's innovative drug repurposing approach, fostering collaboration and advancing their groundbreaking efforts. This invaluable support is essential as Every Cure seeks to engage a wider audience committed to improving access to effective treatments for patients around the globe. "We are thrilled to support Every Cure's visionary mission to save lives by finding new uses for generic and off-patent drugs," said Anna Verghese, Executive Director of The Audacious Project. "With the funding catalyzed through The Audacious Project, Every Cure has the potential to change the lives of the 300 million people lacking access to safe and effective treatments." Ultimately, Every Cure aims to raise over $220 million to support this historic, first-of-its-kind initiative dedicated to systematically identifying and unlocking hidden cures. The Audacious Project's investment in Every Cure builds on an additional $48 million, three-year contract with ARPA-H, a U.S. federal agency that supports impactful biomedical and health initiatives. This landmark funding through The Audacious Project will further empower Every Cure to leverage the outcomes of the ARPA-H contract, enhancing its organizational efforts for global impact. The investments from ARPA-H and the Audacious donors, such as Acton Family Giving; Laura and John Arnold; Chris Anderson and Jacqueline Novogratz; Reed Hastings; High Tide Foundation; Lyda Hill Philanthropies; Jeff and Marieke Rothschild; Sea Grape Foundation; and Valhalla Foundation, bring Every Cure's current fundraising total to approximately $110 million. Media Contact: Jamie Babin [email protected] About Every Cure: Every Cure is a nonprofit biotech that utilizes AI to unleash the potential of every approved medicine to treat every disease it possibly can. Every Cure believes that repurposing medicines represents the fastest and most efficient way to tackle critical health challenges all while optimizing the return on investment in the effort to save lives. Unfortunately, systemic barriers often hinder effective repurposing, leaving patients without access to available potential treatments. To tackle these challenges, Every Cure leverages its AI platform to efficiently identify and evaluate drug repurposing opportunities across all drugs and diseases. This process is guided by objective data and insights from global experts, patient advocacy groups, and research foundations, ensuring a comprehensive strategy that maximizes the potential for discovering effective repurposed treatments. Every Cure aims to update clinical guidelines and may conduct clinical trials as part of its mission to ensure effective therapies reach those in need. Every Cure proudly announced its launch in September 2022 at the Clinton Global Initiative. About The Audacious Project: Launched in April 2018, The Audacious Project is a collaborative funding initiative that's catalyzing social impact on a grand scale. Housed at TED, the nonprofit devoted to ideas, and with support from The Bridgespan Group, The Audacious Project convenes funders and social entrepreneurs with the goal of supporting bold solutions to the world's most urgent challenges. The funding collective is made up of respected organizations and individuals in philanthropy, including Bill & Melinda Gates Foundation, ELMA Philanthropies, Emerson Collective, MacKenzie Scott, Skoll Foundation, Valhalla Foundation, and more. Each year The Audacious Project supports a new cohort. The 2024 grantees are Equipo Argentino de Antropología Forense, Every Cure, Food4Education, Global Methane Hub, Great Barrier Reef Foundation, Inkomoko, MapBiomas, Project Canary, Scaling Safety, and Transcend Education. Learn more at AudaciousProject.org. For more information on Every Cure's initiatives and to learn how you can contribute, visit EveryCure.org. For further information or to express interest in partnering with Every Cure, please contact us at [email protected] or connect with us on Twitter, LinkedIn, Facebook, and Instagram. SOURCE Every Cure WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Sanofi’s Enjaymo, the only approved drug therapy for a rare and debilitating blood disorder, is being acquired by Recordati in a deal that helps the Italian company build its rare disease portfolio and expand its commercial reach in key markets. Recordati is paying $825 million up front for global rights to Enjaymo, a drug that is approved in the U.S., Japan, and Europe. If the drug achieves sales milestones, the company could pay out up to $275 million more, according to deal terms announced Friday. Sanofi and Recordati expect to complete the transaction by the end of this year. Enjaymo treats cold agglutinin disease, a condition in which the immune system attacks red blood cells and causes them to rupture. This process happens at cold temperatures, giving the disease its name. At these temperatures, antibodies that normally attack bacteria instead attach to red blood cells, causing them to bind together, or agglutinate. The red blood cells are destroyed by the immune system, leading to anemia. presented by Health Tech What’s Keeping Healthcare CIOs Up at Night: How to Improve Patient Satisfaction by Handling Calls More Efficiently Health systems have spent billions on portals while investments in modernizing the voice channel — the dominant preference of healthcare consumers — have taken a backseat. By Stephanie Baum The resulting anemia can be managed with blood transfusions. Enjaymo takes a different approach by interfering with the process that leads to red blood cell destruction in cold agglutin disease. The drug, an intravenously infused monoclonal antibody, is designed to selectively target and block a complement system protein called C1. This in turn stops immune system antibodies from attaching to immune cells. The FDA approved Enjaymo in 2022. Approvals in Japan and Europe soon followed. In these three markets, the companies estimate cold agglutin disease affects between 11,000 and 12,000 patients. Recordati, which is headquartered in Milan, is the latest stop in a long journey for Enjaymo. The drug was initially developed by True North Therapeutics, which itself was acquired by Bioverativ in 2017. In 2018, Sanofi paid $11.6 billion to buy Bioverativ. Enjaymo generated €72 million (about $79 million) in sales last year, a 240% increase over the product’s sales in 2022, according to Sanofi’s annual report. Sales for Enjaymo are continuing to grow, but it’s not on track to become a blockbuster seller. Recordati said that in the 12 months ending in August, the drug accounted for €100 million (about $109 million) in revenue. The company projects the product’s peak sales could reach €250 million to €300 million (about $274 million to $328 million). Recordati said its milestone payment to Sanofi is tied to the achievement of net sales at or above the top end of peak year sales expectations. Rare disease accounts for about 34% of Recordati’s more than €2 billion in annual revenue, according to a September investor presentation. Most of those rare disease sales are in the U.S. Much of the company’s rare disease portfolio is comprised of assets from its 2022 acquisition of EUSA Pharma, a developer of drugs for rare cancers. Sponsored Post Get Ready for HLTH 2024: A Preview Guide HLTH 2024, scheduled for October 20-23 at the Venetian Expo Center in Las Vegas, will highlight new programming, a physicians roundtable, generative AI, the shifting landscapes of telemedicine, retail health, women's health, and lots more. By Stephanie Baum In a prepared statement, Recordati CEO Rob Koremans said Enjaymo is particularly complementary to one of those products, Sylvant. This drug, an antibody designed to block the inflammatory signaling protein IL-6, has approvals in more than 40 countries for treating idiopathic multicentric Castleman disease, a rare disorder characterized by abnormal growth of the lymph nodes. While this disease is not cancer, its progression is similar to that of lymphoma, a cancer of the lymph nodes. Last year, Recordati began exploring the development of Sylvant for other diseases driven by IL-6 signaling. “Enjaymo further expands our rare diseases footprint in the U.S., Japan and Europe, and will contribute positively to both our top and bottom lines,” Koremans said. “Most importantly, with a strong clinical profile and as the only product approved for the treatment of [cold agglutinin disease], Enjaymo addresses a serious unmet medical need for patients living with this debilitating disease.” Image by istocksdaily, via Getty Images

Researchers have developed a model of Kaposi sarcoma that could expedite the development of new drugs to treat the disease, which is the most common cancer in people living with HIV. Researchers at UNC Lineberger Comprehensive Cancer Center, after decades of research efforts, have developed a mouse model of Kaposi sarcoma that could be key to the development of new drugs to treat the disease. Kaposi sarcoma is a cancer that is the most common cancer in people living with HIV. The findings appeared in Cell Host & Microbe. "This is an important development as we have created the first animal model ever of Kaposi sarcoma. Animal models are essential to move new drugs from the laboratory bench into clinical trials," said UNC Lineberger's Dirk Dittmer, PhD, senior corresponding author, co-leader of the UNC Lineberger Virology Research Program and director of the UNC Viral Genomics Core. "Before this, only repurposed drugs from other cancers were used to treat Kaposi sarcoma, but now we can start investigating entirely new compounds to help treat what can be a lethal cancer." About 20% of all human cancers arise from viruses or require viral infection as an essential cofactor. The Kaposi sarcoma-associated herpes virus (KSHV) was discovered in 1994 and is associated with Kaposi's sarcoma as well as B-cell cancers. KSHV-associated diseases affect internal organs and are ultimately fatal. In the U.S., the diseases are found primarily in immunosuppressed people such as those who are HIV-positive or are transplant patients. Worldwide, an estimated 34,270 cases of Kaposi sarcoma were diagnosed, and 15,086 deaths reported in 2020, with twice as many cases and deaths occurring in men compared to women. Africa accounted for 73% of new cases and 86.6% of the deaths from Kaposi sarcoma worldwide. The disease is endemic, and not HIV-related, in some southern and eastern African countries. Aside from animal models, one way to study cancer is to look at tumor cells in the lab. But according to Dittmer, Kaposi sarcoma tumor cells are very finicky and dependent on signaling molecules and blood supply, which is why they don't survive in a laboratory culture dish. Therefore, researchers have been focusing on developing animal models that would mimic, as closely as possible, the disease in humans. One of the challenges the researchers faced in developing their model was the fact that two types of genes are transcribed into proteins in the Kaposi sarcoma mouse model. Normally when a virus infects a cell, the cell dies as the virus replicates, which is a process called cell lysis; the genes that are needed for the virus to propagate itself are lytic genes. Cancer viruses are different as they enter a quiet state, called latency, where only the genes that help the infected cell survive are expressed. The mouse model the researchers developed is complex as a bit of both types of genes were needed. Cervical cancer and its related virus, HPV (human papilloma virus), offers a good comparison for the challenge of developing a Kaposi sarcoma mouse model. The KSHV genome is 20 times larger than HPV. HPV has two cancer-causing genes, E6 and E7, so to mimic the disease in animals, researchers only needed to design two mice, one for each gene. KSHV may have as many as 10 cancer-causing genes that all work together so it would be way too difficult to develop that many mice, hence the virtue of their single model, Dittmer noted. "Another of the key virtues of our new mouse model is that it helps us understand angiogenesis, or new blood vessel formation. Without angiogenesis cancer cells are deprived of oxygen and die," Dittmer said. "In this mouse model, we can study angiogenesis blocking drugs better than ever before. If new drugs work against Kaposi sarcoma, they will also likely work against lesser-angiogenic tumors, which would be a major plus." For next steps, the researchers hope that others will pursue drug and vaccine development based on a new understanding of fundamental aspects of KSHV provided by their mouse model, including possible development of a much-needed primate model for human KSHV and Kaposi sarcoma.