Brincidofovir

Brain cancers are notoriously difficult to treat, with surgery and radiation continuing to be the main options for these malignancies. A rare and aggressive type of brain tumor driven by a particular genetic signature could soon have its first approved drug treatment. Jazz Pharmaceuticals is now committing $935 million to add this prospect to its pipeline. The drug, dordaviprone, was developed by Durham, North Carolina-based Chimerix. The cancer is H3 K27M-mutant diffuse glioma, a high-grade glioma that affects children and young adults. While surgery is a treatment option for brain cancers, it’s usually risky for H3 K27M gliomas because these tumors affect critical parts of the brain and spinal cord. Meanwhile, radiation introduces complication risks and is not curative. “This is a very high unmet need,” Jazz Chief Medical Officer Robert Iannone said, speaking during the company’s presentation Wednesday at TD Cowen’s annual health care conference in Boston. “I’m a pediatric oncologist by training. There really has been no progress in this area. I know from treating these patients, it’s been a very sad situation to have only radiation and surgery as the effective therapy.” Sponsored Post Tackling Messy Provider Data with an NPI-Forward Approach Mike Wirth of ProviderTrust shares insights about the company in this interview at the ViVE 2025 conference in Nashville last month. By ProviderTrust and MedCity News About 2,000 patients are affected by H3 K27M-mutant gliomas annually in the U.S., according to a Chimerix estimate. Dordaviprone is part of a new class of cancer drugs called imipridones. These small molecules target certain G protein-coupled receptors and enzyme targets, altering their activity to lead to cancer cell death. Dordaviprone, known earlier in its development as ONC201, is designed to selectively target mitochondrial protease ClpP and dopamine receptor D2. Phase 2 results showed tumor regression in patients with glioblastoma who exhibited the H3 K27M mutation. In the 50 patients included in the primary efficacy analysis, Chimerix reported a 28% objective response rate and a median duration of response of 10.4 months. A Phase 3 test enrolling patients newly diagnosed with this type of brain cancer could support first-line use of the drug; interim results are expected in the third quarter of 2025. But based on the Phase 2 data, Chimerix is seeking accelerated approval of dordaviprone for recurrent H3 K27M-mutant diffuse glioma. Last month, the FDA accepted the new drug application under priority review, setting an Aug. 18 target date for a regulatory decision. Though cancer is the therapeutic focus of Chimerix, it’s a relatively new area for the company, one of the older biotechs in North Carolina’s Research Triangle. Dordaviprone came from Chimerix’s 2021 cash and stock acquisition of Oncoceutics, a clinical-stage developer of imipridones. Besides the lead drug candidate, the deal also brought ONC206, an imipridone that Chimerix has advanced to Phase 1 testing in central nervous system tumors. Per terms of the Oncoceutics acquisition, that company’s shareholders could receive up to $360 million in milestone payments plus royalties from sales. Chimerix, founded in 2000, began as an antiviral drug developer. For most of the biotech’s history, its focus was brincidofovir, a molecule initially developed for treating cytomegalovirus infection. It failed in that indication, but won FDA approval in 2021 as a treatment for smallpox and is now known by the brand name Tembexa. In 2022, Chimerix sold that drug’s rights to Emergent BioSolutions for $225 million up front and up to $100 million in milestone payments. Chimerix applied the cash toward clinical development of dordaviprone in brain cancer. Sponsored Post Webinar: Tips To Have Your Practice Succeed This Year This on-demand webinar will offer insights and share tips on helping practices run more efficiently. By Elation Health and MedCity News In the past year, Chimerix’s stock price has mostly traded below the $1 mark. Those shares got a significant lift in December after the biotech announced it would seek accelerated approval for dordaviprone. According to acquisition terms disclosed Wednesday, Dublin-based Jazz will pay $8.55 in cash for each Chimerix share, representing a 72% premium to the biotech’s closing stock price on Tuesday. There’s potential for additional financial upside. Because Chimerix’s drug is under priority review, it’s eligible for a rare pediatric disease priority review voucher if the drug is approved. Such vouchers are typically sold to big pharma companies, and they once fetched prices in the range of $100 million. Recent voucher sales have been in the neighborhood of $150 million. The Jazz portfolio currently has five cancer drugs. Chief Financial Officer Philip Johnson described dordaviprone as a “great fit with our oncology business,” adding that the drug can leverage Jazz’s commercial capabilities and offers a patent life extending into 2037 and potentially longer. The Chimerix acquisition fits Jazz’s strategy of acquiring assets for rare opportunities in niche markets, Leerink Partners analyst Marc Goodman wrote in a note to investors. Beyond addressing a high unmet medical need, the deal leverages Jazz’s expertise in rare diseases and oncology. Jazz prefers acquiring late-stage assets that have a potential near-term commercial launch, Goodman said. Leerink is still researching the H3 K27M market opportunity, but he noted that given the size of the patient population for this ultra-rare type of cancer, it makes sense for dordaviprone to fall within the $300,000 to $800,000 price range for orphan drug products. The acquisition has been approved by the boards of both Jazz and Chimerix, but still must meet customary closing conditions including the tender of the majority of the outstanding shares of Chimerix. The companies expect the deal to close in the second quarter of this year.

ADDIS ABABA, Ethiopia and KINSHASA, Democratic Republic of the Congo and WASHINGTON and PARIS, Jan. 16, 2025 (GLOBE NEWSWIRE) -- Africa CDC’s support for the MOSA, a pan-African randomized platform adaptive trial for the MpOx Study, adding to the initial EU funding, has enabled the enrollment of the first patients at Mbandaka Hospital in Equateur Province, Democratic Republic of Congo (DRC). These patients have been randomized in this pivotal clinical trial, marking a critical step in addressing the Mpox health threat. As Clade Ib cases emerge outside Africa and Clade I and II cases continue to be reported across the continent, Africa CDC, INRB, and PANTHER are leading efforts to test promising therapeutic options to combat this persistent health threat. Declared a Public Health Emergency of Continental Security on August 13, 2024, Mpox remains a significant health threat across Africa, particularly among vulnerable populations such as women, children, and individuals living with HIV. Despite the pressing need, no therapeutic has been approved to treat Mpox in the United States or Africa. Recent results from the PALM 007 and STOMP studies showed that tecovirimat did not show the expected effect on lesion resolution, underscoring the need for additional therapeutic options. The MOSA trial demonstrates Africa’s leadership in addressing this urgent healthcare gap. The study will evaluate different antivirals, either alone or in combination. It will start by evaluating the safety and efficacy of brincidofovir. Brincidofovir is an antiviral from the global life sciences company Emergent BioSolutions. In the United States it is available under a single-patient emergency use Investigational New Drug (IND) for Mpox. The safety and efficacy of brincidofovir to treat Mpox in humans has not been established in double-blind, placebo-controlled studies for Mpox. In the MpOx Study, patients will receive either brincidofovir or a matching placebo in a liquid oral formulation, administered once-a-week, for 2 weeks. A first interim analysis is expected by the end of Q1 2025. “Africa is not just responding to the Mpox outbreak; we are leading the charge in finding solutions by spearheading research and development for Mpox therapeutics,” said Africa CDC Director General, Dr. Jean Kaseya. “The MpOx Study represents a groundbreaking step toward developing a treatment. This effort goes beyond research—it’s about African ownership and leadership in addressing our continent's health challenges through vital, innovative research.” Sponsored by PANTHER, a key research partner of Africa CDC, the MOSA trial builds upon initial funding provided by the European Union’s Horizon Europe program. The study protocol was reviewed through the AVAREF collaborative process in 2023, ensuring it meets the highest ethical and scientific standards. The innovative study design includes pre-planned interim analyses, allowing for rapid decision-making should early signs of either futility or success be observed. “This milestone highlights the vital role of research preparedness and response. Funded initially by Horizon Europe as part of the emergency response to the mpox outbreak in 2022, I am delighted to see MOSA trial fully operational now to advance treatments for mpox.” Marc Lemaître, European Commission, Director-General for Research and Innovation. “We are proud of this important milestone illustrating PANTHER’s mission in addressing continental epidemic priorities,” said Prof. Samba Ousmane Sow, Director General of the Centre for Vaccine Development in Mali (CVD-Mali) and President of the Board of PANTHER. “We hope to contribute to Africa CDC’s response to the current crisis while also deriving concrete lessons to better prepare for future ones.” As Africa continues to report rising Mpox cases, particularly in the DRC, the MOSA trial seeks to provide critical data on potential treatment options. The study will recruit both children and adults, focusing on those most at risk of severe outcomes, especially in remote areas. “We look forward to advancing MOSA, another critical research project that complements ongoing studies on transmission, vaccines and social sciences,” said Prof Placide Mbala, Principal Investigator for the study in the DRC and Head of the Department of Epidemiology and Global Health at INRB. “Mpox continues to be a global threat to public health, creating a need for treatments and vaccines,” said Simon Lowry, M.D., chief medical officer, head of research and development, Emergent. “We applaud Africa CDC and PANTHER for their work progressing the ‘MpOx Study in Africa’ (MOSA) and advancing research for brincidofovir.” The U.S. FDA Prescribing Information for TEMBEXA® (brincidofovir) is provided for additional safety information here. About the Pandemic Preparedness Platform for Health and Emerging Infections Response (PANTHER) PANTHER is the first African-led pandemic preparedness platform for health and emerging infection response. Bringing together leading African and global researchers and public health teams, it aims to create regional hubs and clinical research platforms to support preparedness and rapid response to emerging infectious diseases globally, particularly in Africa. For more information, visit https://pantherhealth.org. PANTHER is sponsoring MOSA as part of the MPX-RESPONSE Project that has received funding from the European Union’s Horizon Europe Research and Innovation programme under grant agreement 101115188 About Africa Centres for Disease Control and Prevention (Africa CDC)The Africa Centres for Disease Control and Prevention (Africa CDC) is a public health agency of the African Union. It is autonomous and supports member states in strengthening health systems. It also helps improve disease surveillance, emergency response, and disease control. Learn more at: http://www.africacdc.org and connect with us on LinkedIn, Twitter, Facebook and YouTube. About Emergent BioSolutions At Emergent, our mission is to protect and enhance life. For over 25 years, we’ve been at work defending people from things we hope will never happen—so we are prepared just in case they ever do. We provide solutions for complex and urgent public health threats through a portfolio of vaccines and therapeutics that we develop and manufacture for governments and consumers. We also offer a range of integrated contract development and manufacturing services for pharmaceutical and biotechnology customers. To learn more about how we help protect public health, visit our website and follow us on LinkedIn, X, Instagram, Apple Podcasts and Spotify. Media Contacts: Margaret Edwin | Director of Communication and Public Information | Africa CDC EdwinM@africacdc.org Jessica Ilunga | Communication Officer | PANTHEREmail: Media@pantherhealth.orgWebsite: www.pantherhealth.orgTwitter | LinkedIn Assal Hellmer | Vice President, Communications | Emergent BioSolutions Email: mediarelations@ebsi.com A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/683aea7c-60d3-4af9-8964-1d0031f84727