Ensitrelvir Fumaric Acid

Fulcrum ends development of sole clinical candidate, pociredirPraxis' vormatrigine falls short in pivotal seizure studyShionogi gets FDA nod for oral COVID-19 preventativeCEPI promises Moderna up to $50M for Ebola vaccine development Agios nabs Oscotec's SYK inhibitor Fulcrum ends development of sole clinical candidate, pociredirFulcrum Therapeutics' shares crashed close to 50% during after-hours trading on Monday after disclosing it has discontinued its sickle cell disease asset — and only clinical programme — pociredir. The company is planning to launch a comprehensive review to identify any "strategic alternatives" such as a merger, acquisition or business combination.The decision to bin pociredir, an oral small molecule EED inhibitor, was driven by feedback from the FDA. According to Fulcrum, the US regulator had "heightened" concerns about the candidate's risk-benefit profile due to a high rate of secondary haematologic malignancies observed with a mechanistically similar drug, Tazverik (tazemetostat), which Ipsen withdrew from markets in March. Though the two drugs are directed against different targets — pociredir targets EED and Tazverik targets EZH2 — they are both within the PRC2 complex. carries equivalent malignancy risk regardless of the specific subunit engaged.Feedback from the FDA "left no viable regulatory path forward for further clinical development of pociredir," Fulcrum said. The company is now significantly reducing its operating expenses to preserve capital; as of March 31, it had $333.3 million in cash on-hand. Praxis' vormatrigine falls short in pivotal seizure study Praxis Precision Medicines' focal onset seizure (FOS) hopeful vormatrigine has failed the Phase II/III POWER1 study, the company said Monday. The sodium channel inhibitor and functional state modulator did not lead to a statistically significant reduction from baseline in monthly seizure frequency as compared with placebo.  Still, the company — whose shares slipped about 10% during after-hours trade Monday — isn't giving up on vormatrigine yet. CEO Marcio Souza said Praxis was "encouraged by the signal we saw on the higher dose arm, the low discontinuation rate and solid safety profile," adding that the drugmaker is taking "some time to review these results to ensure we have the best path forward for developing vormatrigine and the ongoing POWER2 study." The Phase III POWER2 trial is the second registrational study evaluating vormatrigine in adults with FOS; it's expected complete in the second half, with topline results due in 2027.Shionogi gets FDA nod for oral COVID-19 preventativeThe FDA on Monday approved Shionogi's oral antiviral Xocova (ensitrelvir) for post-exposure prophylaxis (PEP) of COVID‑19 in adults and children aged 12 and up to prevent infection after coming into contact with someone who has the disease. The approval, which came ahead of Xocova's June 16 PDUFA date, covers a five-day treatment regimen for the SARS-CoV-2 main protease inhibitor. Three tablets are taken on the first day, following exposure, and one tablet is taken on days two through five. Xocova's green light is based on the results of the Phase III SCORPIO-PEP trial, which involved 2387 participants. The PEP treatment significantly reduced the risk of symptomatic COVID-19 by 67% in uninfected individuals following exposure to an infected individual after 10 days, compared with placebo. According to Shionogi, SCORPIO-PEP is the first and only Phase III study of an oral antiviral to meet the primary endpoint of preventing COVID-19 following exposure.CEPI promises Moderna up to $50M for Ebola vaccine development  The Coalition for Epidemic Preparedness Innovations (CEPI) is "urgently" backing the development of three Ebola vaccine candidates to combat the viral disease outbreak in the Democratic Republic of the Congo, committing about $60 million in total to the venture. Moderna said Monday that CEPI has pledged up to $50 million to support preclinical development and Phase I testing of its mRNA vaccine candidate. The funds will also go towards manufacturing so the asset can quickly move into large-scale Phase II/III trials if the early safety and immunogenicity data come back positive.  The International AIDS Vaccine Initiative will also receive financial support from CEPI to work on a Bundibugyo vaccine candidate, as will the University of Oxford. Agios nabs Oscotec's SYK inhibitor  Agios Pharmaceuticals licensed exclusive, global rights to cevidoplenib, a next-generation, oral spleen tyrosine kinase (SYK) inhibitor, from South Korea-based drugmaker Oscotec. It plans to launch a Phase III trial evaluating the drug in patients with immune thrombocytopenia (ITP) in the first half of 2028."Cevidoplenib is a next-generation SYK inhibitor uniquely designed to potentially offer improved tolerability and durability compared to first-generation SYK inhibitors," Agios CEO Brian Goff said in a company release Monday. "Backed by clinically meaningful Phase II data, we believe cevidoplenib has the opportunity to become a best-in-class treatment option for ITP."Under the deal, Oscotec will receive $25 million upfront and is eligible for up to $140 million in development and regulatory milestones across up to three indications in the US and Europe, as well as commercial milestone payments and royalties ranging from high single-digit to mid-teen. The firm also has an option to license exclusive development and commercialisation rights to cevidoplenib in South Korea after the Phase III trial reads out.

The FDA delivered its green light more than two weeks early, with the agency originally slated to weigh in on Shionogi’s Xocova application by June 16. After a prior misfire in demonstrating its protease inhibitor ensitrelvir’s potential as a COVID-19 treatment for U.S. patients, Shionogi has secured an FDA green light for the medicine in the prevention setting.Sold under the Xocova moniker—and available in Japan since 2022—Shionogi’s oral drug has been cleared for post-exposure prophylaxis (PEP) of COVID-19 in U.S. adults and adolescents ages 12 and older following contact with someone who has COVID.With the FDA's sign-off, Xocova has earned the distinction of becoming the first oral drug approved in the U.S. to protect against COVID after exposure, Shionogi noted in a June 1 press release.  The drug is taken over five days, beginning with three tablets on day one and just one tablet on the following four. The FDA delivered its green light more than two weeks early, with the agency originally slated to weigh in on Shionogi’s Xocova application by June 16. Beyond currently approved COVID-19 vaccines from Pfizer-BioNTech, Moderna and Novavax, the U.S. has also cleared antiviral treatments in Pfizer’s Paxlovid, Gilead’s Veklury and Merck & Co.’s Lagevrio, all for treatment after symptom onset. Shionogi’s drug, meanwhile, “has the potential to benefit anyone who does not want to get COVID-19,” Frederick Hayden, M.D., professor emeritus of clinical virology at the University of Virginia School of Medicine, said in the company’s release. Aside from use among households, Xocova could offer an appealing prophylactic option in other exposure incidents, including outbreaks in nursing homes and care facilities, and after travel-related contact, Hayden added.  The FDA’s approval hinges on Shionogi’s SCORPIO-PEP trial, in which Xocova curbed the risk of symptomatic COVID-19 by 67% in uninfected people following exposure to someone infected, through 10 days, as weighted against placebo. Shionogi’s trial enrolled 2,387 participants who had a negative local screening test for the SARS-CoV-2 virus and no symptoms at enrollment after being exposed to an infected person in their household. The company’s primary analysis relied on 2,041 household contact participants with a lab-confirmed negative test at baseline. The Osaka-headquartered company noted that its antiviral was generally well tolerated, with headache, diarrhea and cough the most common side effects among patients on Xocova. “Xoxova is the first and only oral option clinically proven to help prevent symptomatic COVID-19 after exposure among study participants regardless of vaccination status or baseline immunity from prior infection,” Nathan McCutcheon, president and CEO of Shionogi’s U.S. arm, said in a statement. With the drug, “people who are exposed to COVID-19 can act early to help protect themselves,” he added. While the heyday of COVID-19 vaccine and drug sales has long waned—as evinced by Pfizer and Moderna’s ongoing earnings pressures—Shionogi feels Xocova still has an important niche to fill as the disease remains highly transmissible. Up to 47% of people who live with someone infected with COVID risk developing it themselves, Shionogi reckons, pointing to CDC estimates that there were some 3.8 million to 12.4 million new COVID cases in the U.S. from the beginning of last October to May 23, 2026. These infections led to hundreds of thousands of outpatient visits, some 240,000 hospitalizations and between 13,000 and 42,000 deaths, according to the data. In Shionogi’s home country of Japan, Xocova also holds a post-exposure prophylaxis approval. The antiviral was originally greenlit under an emergency nod there as a COVID-19 treatment in November 2022 before securing a full approval in that setting in early 2024. The medication is also available in Singapore. With its protection nod, Shionogi has found a new niche in the U.S. market for Xocova, which disappointed in the late-stage SCORPIO-HR trial back in May 2024. In the study, the antiviral failed to chart a statistically significant reduction in time to sustained resolution of 15 common COVID symptoms. That performance caused the study to miss its primary endpoint. Shionogi leadership admitted at the time that the data were mixed, but they stood behind their asset’s potential and pledged to continue working with regulators to make ensitrelvir more broadly available. Shionogi clarified in its approval announcement Monday that Xocova is cleared as a preventative and not on offer as a COVID treatment in the U.S.