https://www.bioworld.com/rss en-us J-Pharma Co. Ltd. is progressing the most clinically advanced L-type amino acid transport 1 (LAT1) inhibitor, nanvuranlat (JPH‑203), in a global phase III Beacon-BTC study of biliary tract cancer following U.S. FDA alignment. http://www.bioworld.com/articles/731921 Thu, 18 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731921-j-pharma-drives-first-cancer-lat1-to-phase-iii-study-with-fda-nod Illustration of cancer in the bile ducts Four years after the U.S. FDA issued a complete response letter for Spero Therapeutics Inc.’s oral antibiotic, tebipenem pivoxil hydrobromide, to treat complicated urinary tract infections (cUTI), including pyelonephritis, the agency approved the drug based on phase III data showing noninferiority to intravenous imipenem-cilastatin. http://www.bioworld.com/articles/731919 Thu, 18 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731919-gsk-speros-oral-antibiotic-utebzi-for-cuti-wins-us-fda-nod FDA Approved stamp with pills Three years on from the rebuff of a U.S. FDA complete response letter, F2G Ltd. now has the data needed to resubmit the NDA for the first novel antifungal drug in more than two decades. Along with partner Shionogi & Co. Ltd., F2G has released positive phase III results for orally administered olorofim, showing noninferiority against I.V.-administered Ambisome (amphotericin B, Gilead Sciences Inc.) in patients with refractory aspergillosis infections, or who were unsuitable for mainstay azole therapy. http://www.bioworld.com/articles/731918 Thu, 18 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731918-f2g-preps-nda-for-first-new-antifungal-in-20-years-on-solid-phase-iii Aspergillus fumigatus The U.S. FDA’s Vaccines and Related Biological Products Advisory Committee voted unanimously in favor of Moderna Inc.’s trivalent mRNA flu vaccine, mRNA-1010, which the company wants to brand as Mflusiva. On the question of whether the benefits of the product outweigh the risks in people ages 50 to 64, balloting was 9-0, and on whether the same is true in those 65 and older, the tally was again 9-0. http://www.bioworld.com/articles/731917 Thu, 18 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731917-table-turned-modernas-high-fi-flu-shot-plays-well-at-adcom Elderly person receiving shot Cenna Biosciences Inc. has obtained IND clearance from the FDA for 8M2D, a first-in-class peptide for the treatment of Alzheimer’s disease. A phase Ia/Ib trial of 8M2D in healthy volunteers and patients with early Alzheimer’s disease is expected to begin dosing late this year. http://www.bioworld.com/articles/732011 Thu, 18 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732011-fda-clears-ind-for-cennas-8m2d-for-alzheimers-disease Beam Therapeutics Inc. has obtained IND clearance from the FDA for BEAM-304 for the treatment of phenylketonuria (PKU). BEAM-304 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct mutations in the phenylalanine hydroxylase (PAH) gene that cause PKU. http://www.bioworld.com/articles/732008 Thu, 18 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732008-beam-therapeutics-beam-304-gains-ind-clearance-for-pku Another day, another about-face by the U.S. FDA on Uniqure NV’s Huntington’s disease gene therapy. But this latest shift brings good news for the company’s AMT-130, for which the FDA says three-year analysis data from the phase I/II study will be acceptable for an accelerated BLA filing, now expected to be submitted in the third quarter. http://www.bioworld.com/articles/731903 Wed, 17 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731903-flexibility-restored-uniqure-plans-for-bla-filing-in-huntingtons Genetic mutation illustration Calidi Biotherapeutics Inc. has received pre-IND regulatory feedback from the FDA on Calidi’s IND-enabling preclinical plans and clinical strategy for CLD-401. The parties agreed on key aspects of the CMC and nonclinical programs, as well as the overall design for the proposed first-in-human study. http://www.bioworld.com/articles/731994 Wed, 17 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731994-calidi-receives-pre-ind-feedback-from-fda-on-cld-401 Oncolytic virus concept illustration On the eve of the June 17 Vaccines and Related Biological Products Advisory Committee (VRBPAC) meeting, which will discuss Moderna Inc.’s mRNA-1010, researchers at Washington University School of Medicine in St. Louis have reported that the vaccine conferred broader and more durable protection than a standard flu shot. http://www.bioworld.com/articles/731989 Wed, 17 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731989-mrna-flu-vaccine-can-activate-broad-germinal-center-response mRNA vaccines are composed of messenger RNA encapsulated in lipid nanoparticles Elixiron Immunotherapeutics Inc. announced positive interim open-label phase II Alzheimer’s disease (AD) study findings of enrupatinib, an oral brain-penetrant colony-stimulating factor 1 receptor inhibitor, with plans to move the asset into a biomarker-based and placebo-controlled study. http://www.bioworld.com/articles/731984 Tue, 16 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731984-elixirons-enrupatinib-shows-upbeat-interim-phase-ii-ad-results Illustration of amyloid plaques in Alzheimer's disease Moderna Inc. will soon find out if the tumult-ridden U.S. FDA’s switcheroo in mid-February will stick regarding mRNA-1010, a prospective new seasonal influenza vaccine. The Vaccines and Related Biological Products Advisory Committee on June 17 takes up the matter of the shot, which trails a curious history. http://www.bioworld.com/articles/731890 Tue, 16 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731890-another-shot-moderna-flu-vaccine-re-upped-in-adcom Syringe in front of Moderna logo Credit: Asiraj - stock.adobe.com Elixiron Immunotherapeutics Inc. announced positive interim open-label phase II Alzheimer’s disease (AD) study findings of enrupatinib, an oral brain-penetrant colony-stimulating factor 1 receptor inhibitor, with plans to move the asset into a biomarker-based and placebo-controlled study. http://www.bioworld.com/articles/731877 Mon, 15 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731877-elixirons-enrupatinib-shows-upbeat-interim-phase-ii-ad-results Illustration of amyloid plaques in Alzheimer's disease Despite some back-and-forth with the U.S. FDA regarding inclusion in the somewhat controversial Commissioner’s National Priority Voucher (CNPV) program, Sanofi SA’s Tzield (teplizumab) gained FDA clearance, expanding use of the CD3-directed monoclonal antibody as a disease-modifying therapy for patients with recently diagnosed stage 3 type 1 diabetes (T1D). http://www.bioworld.com/articles/731874 Mon, 15 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731874-no-cnpv-needed-sanofis-tzield-gains-accelerated-nod-in-stage-3-t1d Vaccine vials and syringe In a repeat move, the U.S. FDA issued yet again a complete response letter (CRL) to Camurus AB for its subcutaneous extended-release injection drug CAM-2029 (octreotide) to treat the rare chronic growth disorder acromegaly. The drug, which expects to be branded Oclaiz in the U.S. upon approval, is called Oczyesa in the EU and the U.K., where it received marketing authorization in 2025. http://www.bioworld.com/articles/731805 Thu, 11 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731805-another-fda-no-for-camurus-monthly-acromegaly-drug-oclaiz FDA icons and doctor Agios Pharmaceuticals Inc.’s potential $665 million deal for Oscotec Inc.’s cevidoplenib, a phase III-ready oral SYK inhibitor to treat immune thrombocytopenia, marks a new chapter of development focused on cancer therapy resistance for Oscotec, CEO Yoon Tae-young said. http://www.bioworld.com/articles/731828 Tue, 09 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731828-disciplined-665m-deal-moves-oscotecs-cevidoplenib-to-agios Deal handshake with coin, chart background Gen1e Lifesciences Inc. has obtained both orphan drug and rare pediatric disease designations from the U.S. FDA for GEn-1123 for the treatment of Duchenne muscular dystrophy (DMD). http://www.bioworld.com/articles/731763 Tue, 09 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731763-gen1e-lifesciences-receives-us-fda-designations-for-gen-1123 Art concept for inflamed human tissue Amphista Therapeutics Ltd. has obtained IND clearance from the FDA for AMX-883, an orally bioavailable degrader of BRD9, for the treatment of acute myeloid leukemia (AML). A phase I trial in patients with relapsed or refractory AML and high-risk myelodysplastic syndrome is expected to begin in the second half of this year. http://www.bioworld.com/articles/731739 Mon, 08 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731739-amphistas-brd9-degrader-gains-ind-clearance-for-aml 3D illustration of acute myeloid leukemia cells The U.S. FDA’s year-old Commissioner’s National Priority Voucher (CNPV) pilot program played to mixed reviews at the agency’s June 4 listening session intended to get various stakeholders’ perspective on the ultra-accelerated review process being offered to qualifying drugs. http://www.bioworld.com/articles/731633 Fri, 05 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731633-fda-gets-earful-on-cnpv-pilot FDA website and logo Credit: Postmodern Studio - stock.adobe.com Agios Pharmaceuticals Inc.’s potential $665 million deal for Oscotec Inc.’s cevidoplenib, a phase III-ready oral SYK inhibitor to treat immune thrombocytopenia, marks a new chapter of development focused on cancer therapy resistance for Oscotec, CEO Yoon Tae-young said. http://www.bioworld.com/articles/731632 Fri, 05 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731632-disciplined-665m-deal-moves-oscotecs-cevidoplenib-to-agios Deal handshake with coin, chart background Sotio Biotech AS’s SOT-106 has been granted orphan drug designation by the FDA for the treatment of osteosarcoma. SOT-106 is a next-generation antibody-drug conjugate targeting leucine-rich repeat-containing 15 (LRRC15), a clinically validated target broadly expressed across sarcoma subtypes and in tumor-associated stroma. http://www.bioworld.com/articles/731653 Thu, 04 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731653-sotio-biotechs-sot-106-designated-orphan-drug-for-osteosarcoma Illustration of bone cancer in the knee Hanmi Pharmaceutical Co. Ltd. secured a $1.26 billion deal with Eli Lilly and Co. to out-license ex-Korea rights to sonefpeglutide (HM-15912), a Lapscovery-based glucagon-like peptide-2 analog in development for multiple indications, including an ongoing phase II study of short bowel syndrome. It was one of two billion-dollar Asian company deals signed by Lilly on June 1, with the second transaction involving Haisco Pharmaceutical Group Co. Ltd., of Beijing. http://www.bioworld.com/articles/731645 Tue, 02 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731645-hanmi-haisco-win-billion-dollar-partnerships-with-eli-lilly Hanmi signage Credit: Hanmi Pharmaceutical Co. Ltd. Shares of Fulcrum Therapeutics Inc. (NASDAQ:FULC) fell 54% after the company said it is discontinuing work on sickle cell disease (SCD) candidate pociredir, its only clinical-stage candidate, and reviewing strategic alternatives in the wake of the U.S. FDA’s safety concerns regarding the drug target. http://www.bioworld.com/articles/731532 Tue, 02 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731532-fulcrum-drops-work-on-scd-drug-pociredir-stock-plummets Sickle cell illustration Rather than reinventing the wheel for every gene therapy that uses genome editing, the U.S. FDA is advising sponsors on leveraging existing knowledge, be it publicly available or platform-based, to more efficiently advance their products across multiple stages of development. http://www.bioworld.com/articles/731529 Tue, 02 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731529-fda-advises-leveraging-whats-already-known-in-gene-therapy-r-and-d DNA double helix illustration with section being removed in red Iovance Biotherapeutics Inc. has received IND clearance from the FDA for IOV-5001, a next-generation interleukin-12 (IL-12)-tethered tumor-infiltrating lymphocyte (TIL) therapy. http://www.bioworld.com/articles/731580 Tue, 02 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731580-fda-approves-ind-for-iovances-til-therapy-iov-5001 Microscope with laptop displaying histology image. Voyager Therapeutics Inc. has obtained IND clearance from the FDA for VY-1706, the company’s investigational gene therapy for the treatment of Alzheimer’s disease. http://www.bioworld.com/articles/731578 Tue, 02 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731578-voyagers-tau-targeted-gene-therapy-vy-1706-gains-ind-approval Tau neuron illustration Tau protein accumulating on neuron. Credit: Kenneth S. Kosik, University of California - Santa Barbara The U.S. FDA approved Shionogi & Co. Ltd.’s Xocova (ensitrelvir) as the first oral post-exposure prophylactic option in the U.S. to prevent COVID-19, with the decision coming ahead of a PDUFA target date of June 16. http://www.bioworld.com/articles/731531 Tue, 02 Jun 2026 08:00:00 -0400 https://www.bioworld.com/articles/731531-fda-clears-shionogis-xocova-as-first-covid-19-prevention-pill Xocova blister pack Credit: Shionogi & Co. Ltd. Hanmi Pharmaceutical Co. Ltd. secured a $1.26 billion deal with Eli Lilly and Co. to out-license ex-Korea rights to sonefpeglutide (HM-15912), a Lapscovery-based glucagon-like peptide-2 analog in development for multiple indications, including an ongoing phase II study of short bowel syndrome. It was one of two billion-dollar Asian company deals signed by Lilly on June 1, with the second transaction involving Haisco Pharmaceutical Group Co. Ltd., of Beijing. http://www.bioworld.com/articles/731459 Mon, 01 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731459-hanmi-haisco-win-billion-dollar-partnerships-with-eli-lilly Hanmi signage Credit: Hanmi Pharmaceutical Co. Ltd. Industry watchers will be tracking closely Replimune Group Inc.’s latest attempt to secure U.S. FDA approval of its advanced melanoma candidate, RP-1 (vusolimogene oderparepvec). The company announced Friday it had reached alignment for resubmitting the twice-rejected BLA with the agency, which it said has pledged to prioritize the review. http://www.bioworld.com/articles/731453 Fri, 29 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731453-replimune-tries-again-with-melanoma-drug-post-fda-shakeup Skin exam The FDA has accepted the IND application from Rest Therapeutics SAS for RST-101, the company’s lead investigational candidate for the early treatment of post-traumatic stress disorder (PTSD). http://www.bioworld.com/articles/731494 Fri, 29 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731494-fda-accepts-ind-for-rest-therapeutics-early-ptsd-candidate PTSD chalk silhouette, man holding head Multiple updates on TROP2-directed antibody-drug conjugates (ADCs) for lung and breast cancers highlight both progress made and opportunities for improvement for the targeted class of cancer therapeutics. http://www.bioworld.com/articles/731648 Thu, 28 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731648-trop2-adcs-progress-into-first-line-for-lung-breast-cancers Floating antibody drug conjugates