Chimerix

DelveInsight's 2025 assessment reveals 15+ companies are developing over 20 cytomegalovirus (CMV) infection treatment therapies across various clinical development phases.

• Jazz Pharmaceuticals reported total revenues of $898 million in Q1 2025, with Xywav and Epidiolex showing strong year-over-year growth of 9% and 10% respectively. • The company submitted a supplemental New Drug Application for Zepzelca in combination with atezolizumab as maintenance therapy for first-line extensive-stage small cell lung cancer, based on promising Phase 3 trial results. • Jazz completed the acquisition of Chimerix, adding dordaviprone to its late-stage pipeline for H3 K27M-mutant diffuse glioma, with an FDA PDUFA target date of August 18, 2025.

• DelveInsight's latest report reveals a robust pipeline with 110+ companies developing 120+ therapies for acute myeloid leukemia (AML), showing significant investment in this aggressive blood cancer. • Several promising candidates are advancing through clinical trials, including GlycoMimetics' uproleselan in Phase III, BioSight's aspacytarabine (BST-236) in Phase II, and novel approaches like Senti Biosciences' logic-gated CAR-NK cell therapy. • Recent developments include Moleculin Biotech's Phase III MIRACLE trial for annamycin, Qurient's adrixetinib IND approval, and Rigel Pharmaceuticals' trial of REZLIDHIA in combination therapy for IDH1-mutated AML.

Jazz Pharmaceuticals has announced the strategic acquisition of Chimerix, expanding its portfolio with a promising late-stage drug candidate.

Jazz Pharmaceuticals announces acquisition of Chimerix for $935 million, gaining dordaviprone, a first-in-class treatment for rare H3 K27M-mutant diffuse glioma with FDA Priority Review status.

• Q32 Bio's bempikibart showed promise in Phase IIa trial for alopecia areata (AA), achieving a 16% mean reduction in SALT score at week 24 compared to placebo. • However, bempikibart failed its Phase IIa trial for atopic dermatitis (AD), with placebo showing slightly better improvement in EASI scores, leading to investor selloff. • BioAge Labs halted its Phase II STRIDES trial of azelaprag due to liver transaminitis, causing stock downgrade and concerns about the drug's safety profile at higher doses. • Candel Therapeutics and uniQure saw stock surges following positive trial results and FDA agreement, respectively, while CervoMed and Chimerix also experienced significant stock movements.

• Chimerix has submitted a New Drug Application (NDA) to the FDA for dordaviprone, targeting recurrent H3 K27M-mutant diffuse glioma, a highly aggressive brain tumor. • The NDA is supported by Phase 2 data showing a 28% objective response rate and a median duration of response of 10.4 months with dordaviprone. • Chimerix is requesting Priority Review, potentially shortening the review period to six months, with a possible launch in Q3 2025 if approved. • Dordaviprone has Rare Pediatric Disease Designation, making Chimerix eligible for a Rare Pediatric Disease Priority Review Voucher.

• The FDA has accepted and granted priority review to Chimerix's new drug application for dordaviprone, a treatment for recurrent H3 K27M-mutant diffuse glioma. • Dordaviprone, a first-in-class imipridone, targets mitochondrial protease ClpP and DRD2, offering a novel approach to treating this aggressive brain cancer. • The FDA has set a PDUFA target action date of August 18, 2025, and does not currently plan to hold an advisory committee meeting. • Clinical data from pooled analyses showed a 20% overall response rate and a median duration of response of 11.2 months, supporting the application.

• Dordaviprone demonstrates a 28% overall response rate in recurrent H3 K27M-mutant diffuse midline glioma using RANO 2.0 criteria. • The median time to response with dordaviprone was 4.6 months, with a median duration of response of 10.4 months. • Chimerix plans to include these updated RANO 2.0 results in a New Drug Application to Australian regulators. • Further presentations at SNO 2024 will cover additional research on dordaviprone and ONC206 in high-grade gliomas.

• Chimerix announced that dordaviprone demonstrated a 28% overall response rate in patients with recurrent H3 K27M-mutant diffuse midline glioma. • The Phase 2 trial data, assessed using RANO 2.0 criteria, showed a median time to response of 4.6 months and a median duration of response of 10.4 months. • Chimerix plans to submit a New Drug Application to Australian regulators based on these updated results, marking a significant regulatory milestone. • The findings will be presented at the 2024 Society for Neuro-Oncology Annual Meeting, highlighting the drug's potential in treating this aggressive brain tumor.