Brincidofovir

Ongoing mpox outbreak continues to be a global health threat and is driving need for greater therapeutic research and innovationGAITHERSBURG, Md., July 28, 2025 (GLOBE NEWSWIRE) -- Emergent BioSolutions Inc. (NYSE: EBS) today announced the publication of a comprehensive review article, "Brincidofovir in the Era of Mpox," in the peer-reviewed journal Expert Review of Anti-infective Therapy.1 This publication includes an overview of brincidofovir, in vitro and in vivo data, including human case studies of the investigational use of brincidofovir, the current evaluation for treating mpox infections in adults and pediatrics in the Democratic Republic of Congo, and the potential for combination therapy with tecovirimat for immunocompromised patients with severe disease. “We believe in the importance of assessing brincidofovir, an antiviral, for this public health threat and we look forward to advancing our research across mpox as it’s a global disease of increasing concern,” said Simon Lowry, M.D., chief medical officer and head of research and development at Emergent. Mpox, formerly known as monkeypox, continues to pose a significant public health challenge, with ongoing regional and global outbreaks particularly in Central and West Africa. On August 14, 2024, the World Health Organization (WHO) declared the current mpox outbreak a global Public Health Emergency of International Concern (PHEIC).2 This marks the second mpox PHEIC declared by the WHO in the past two years, which followed the regional emergency declaration by the Africa Centres for Disease Control and Prevention (Africa CDC) on August 13, 2024.3 This month, the U.S. Centers for Disease Control and Prevention issued an updated report on the surge of mpox around the world.4 As part of the ongoing effort to identify safe and effective treatments for mpox, brincidofovir is currently being evaluated by the Africa CDC in partnership with PANTHER (PANdemic preparedness plaTform for Health and Emerging infectious Response) in the MpOx Study in Africa (MOSA), a double-blind, placebo-controlled clinical trial for people diagnosed with mpox, which initiated in January 2025. The study has received initial funding from Horizon Europe and Africa CDC. Dr. Lowry continued, “In addition to our engagement with PANTHER on the MOSA trial, this scientific review provides insights into the investigational use of brincidofovir in the treatment of mpox, and we are pleased this publication is available for the broader medical community.” Please see full Prescribing Information for TEMBEXA® (brincidofovir) for additional safety information.About Emergent BioSolutions At Emergent, our mission is to protect and save lives. For over 25 years, we’ve been at work preparing those entrusted with protecting public health. We deliver protective and life-saving solutions for health threats like smallpox, mpox, botulism, Ebola, anthrax and opioid overdose emergencies. To learn more about how we help prepare communities around the world for today’s health challenges and tomorrow’s threats, visit our website and follow us on LinkedIn, X, Instagram, Apple Podcasts and Spotify. Safe Harbor StatementWe generally identify forward-looking statements by using words like “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “plan,” “should,” “will,” “would,” and similar expressions or variations thereof, or the negative thereof, but these terms are not the exclusive means of identifying such statements. Forward-looking statements are based on our current intentions, beliefs, and expectations regarding future events based on information that is currently available. We cannot guarantee that any forward-looking statement will be accurate. Readers should realize that if underlying assumptions prove inaccurate or if known or unknown risks or uncertainties materialize, actual results could differ materially from our expectations. Readers are, therefore, cautioned not to place undue reliance on any forward-looking statement. Any forward-looking statement speaks only as of the date of this press release, and, except as required by law, we do not undertake to update any forward-looking statement to reflect new information, events, or circumstances. Readers should consider this cautionary statement, as well as the risk factors identified in our periodic reports filed with the U.S. Securities and Exchange Commission, when evaluating our forward-looking statements. Investor Contact:Richard S. LindahlExecutive Vice President, CFOlindahlr@ebsi.com Media Contact:Assal HellmerVice President, Communicationsmediarelations@ebsi.com __________________________ 1 https://doi-org.libproxy1.nus.edu.sg/10.1080/14787210.2025.25320292 https://www-who-int.libproxy1.nus.edu.sg/news/item/14-08-2024-who-director-general-declares-mpox-outbreak-a-public-health-emergency-of-international-concern3 https://africacdc.org/news-item/africa-cdc-declares-mpox-a-public-health-emergency-of-continental-security-mobilizing-resources-across-the-continent/ 4 https://www-cdc-gov.libproxy1.nus.edu.sg/mpox/situation-summary/index.html

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.