Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year June 27, 2025 -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for GTX-102 (apazunersen) as a treatment for Angelman syndrome. "FDA Breakthrough Therapy Designation underscores both the urgent need fo

—Neopharmed Gentili will pay BioCryst $250 million upfront for the European assets and rights related to ORLADEYO, and up to $14 million in future milestones— —BioCryst plans to use proceeds to retire all remaining term debt, which will eliminate approximately $70 million of future interest payments over remaining life of the loan— —With this transaction, BioCryst expects to end 2027 with approximately $700 million in cash and no term debt (an increase of $400 million from prior 2027 net cash gu

June 27, 2025 -- Pharvaris (Nasdaq: PHVS), a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to help address unmet needs of those living with bradykinin-mediated diseases such as hereditary angioedema (HAE) and acquired angioedema due to C1 inhibitor deficiency (AAE-C1INH), today announced the acceptance of abstracts for presentation at the US Hereditary Angioedema Association's 2025 National Summit to be held from July 10-13, 2025, in Baltimore, M

ALTB-268 demonstrated therapeutic potential in murine models of colitis and GvHD, supporting its role as an immune checkpoint enhancer in T cell-mediated inflammatory diseases Findings reinforce ALTB-268’s ability to downregulate T cell effector function and restore immune balance June 23, 2025 -- AltruBio Inc. (“AltruBio” or “the Company”), a clinical stage biotech company developing novel immune checkpoint enhancers to address the unmet need in immunological and inflammatory diseases, with a

Sarclisa recommended for EU approval by the CHMP to treat transplant-eligible newly diagnosed multiple myeloma Recommendation based on GMMG-HD7 phase 3 study demonstrating that Sarclisa with VRd induction treatment significantly improved MRD negativity benefit and prolonged PFS compared to VRd alone If approved, it would represent the fourth indication in the EU and second in the front-line setting globally June 23, 2025. The European Medicines Agency’s Committee for Medicinal Products for Huma

June 25, 2025 -- DIMERx, Inc., a late-stage biopharmaceutical company entering growth phase, today announced it has been awarded a UG3/UH3 cooperative agreement (UG3DA061645) for up to $15 million from the National Institute on Drug Abuse (NIDA) to advance its lead drug candidate, DMX-101, as a non-addictive oral therapy for chronic lower back pain. The grant was awarded through the NIH Helping to End Addiction Long-term® Initiative, a trans-agency effort aimed at speeding solutions to the op

June 25, 2025 -- Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, today announced that the IND application submitted by its partner in China, Angel Pharmaceuticals Ltd. (Angel Pharma), has been approved by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) to initiate a Phase 1b/2 clinical trial of soquelitinib for the treatment of patients with moderate-to-severe atopic dermatitis in China. Corvus co-found

- Potential Best-In-Class Bispecific Antibody ATI-052 Targets Both Thymic Stromal Lymphopoietin (TSLP) and Interleukin-4 Receptor (IL-4R) - June 23, 2025 -- Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel product candidates for immuno-inflammatory diseases, today announced that it has initiated a placebo-controlled Phase 1a/1b program for ATI-052, the Company’s potential best-in-class investigational bispecific anti-TSLP/IL-4R an

60% (12/20) complete response rate after treatment with EO2463 in combination with lenalidomide and rituximab (R2) EO2463 plus R2 was well tolerated in patients with follicular and marginal zone lymphoma EO2463 plus R2 combination treatment resulted in higher-than-expected rates of complete remission so far in this study compared to historical data for R2 alone 23 June, 2025，Enterome SA, a clinical-stage company developing first-in-class OncoMimics™ immunotherapies to treat cancer, presented po

The Independent Data Monitoring Committee (“IDMC”) has recommended to continue the study as planned as there are no safety concerns demonstrated in the data reviewed Interim results to date demonstrate GRI-0621 to be safe and well-tolerated in the first 24 patients evaluated 6-week interim biomarker data (n=24) expected in July 2025 and topline data expected in Q3 2025 Currently available treatments for IPF are limited to only two approved drugs that come with significant side-effects, limited p