Curated news and analysis on clinical trials, drug approvals, and medical research.
Aquestive Therapeutics disclosed FDA deficiencies in its Anaphylm NDA, halting labeling discussions and causing a 37% stock drop. The setback does not represent a final rejection, leaving a path for resubmission.
A study finds that white matter disease detected on CT scans signals a higher risk for stroke or dementia compared to MRI-only findings. CT-detected WMD may represent more advanced cerebrovascular injury, highlighting the need for modality-specific interpretation of incidentally discovered brain abnormalities.
Cue Biopharma appointed Dominic Borie as chief medical officer and R&D head as it advances CUE-221 (Phase 2 data expected Q3 2026) and CUE-401 (Phase 1 start Q4 2026). He brings over 20 years of immunology and clinical development experience. The company recently raised $50M to support clinical development.
International expert workshops have identified priority therapeutic targets for medulloblastoma and rhabdoid tumours, two rare childhood cancers. For medulloblastoma, key priorities include SRC degradation, c-MYC/MYCN inhibition, and B7-H3 targeted approaches. For rhabdoid tumours, DCAF5 and MDM2 were highlighted, with EZH2 degraders showing potential.
Two structural biology studies reveal new details about bacterial transcription factors NrdR and CcpA, highlighting their potential as selective drug targets for novel antimicrobials that could circumvent existing resistance mechanisms.
A clinical trial showed that digital symptom check-ins improved quality of life for advanced cancer patients, especially underserved groups. Researchers are also advancing survivorship care and investigating ageing biology to improve recovery in older patients.
The FDA accepted Bristol Myers Squibb’s NDAs for iberdomide and mezigdomide, two CELMoD agents for relapsed/refractory multiple myeloma. Iberdomide has Priority Review and a PDUFA date of Aug. 17, 2026; mezigdomide’s is May 13, 2027. The filings are backed by Phase 3 data, including a 52% PFS risk reduction for mezigdomide.
Serina Therapeutics completes sentinel dosing in Cohort 1 of its Phase Ib registrational trial of SER-252 for advanced Parkinson's disease. Safety review recommends continuation; enrollment and dosing ongoing. FDA alignment on 505(b)(2) pathway and registrational status.
Newron Pharmaceuticals has had a constructive FDA meeting about the clinical hold on its evenamide Phase III ENIGMA-TRS 2 trial and also secured up to €38 million in funding to advance the program. The company plans to propose changes to address the FDA’s concerns, while the financing extends its cash runway beyond key 12-week results.
A new antibody-drug conjugate targeting IL1RAP has shown potent preclinical activity against Ewing sarcoma and other fusion-driven cancers, with minimal off-target toxicity. Researchers at UBC report tumor eradication and reduced metastasis, with clinical trials being planned.
Two Toronto men face charges after an OPP drug investigation in Dryden, Ont. Police seized cash and evidence during a raid on Clearwater Crescent. The case involves multiple charges including proceeds of crime over $5,000 and resisting arrest.
Savara resubmits BLA with near-term FDA decision expected; Sarepta reports $331M in Q1 net product revenue but Elevidys sales continue to decline; AstraZeneca stock falls ahead of July 2026 earnings release.
Fast-track residential developments in Queenstown face criticism over unmanaged cumulative traffic and infrastructure impacts, with public input largely sidelined. A surge in applications for unplanned areas has sparked debate about the process's effectiveness.
CSPC Pharmaceutical Group achieved a $25 million R&D milestone payment in its obesity drug alliance with AstraZeneca, part of a record $18.5 billion deal. The collaboration centers on long-acting GLP-1 assets, including a Phase I candidate, but CSPC shares declined on competitive and governance concerns.
A new study reveals that a second pregnancy uniquely alters the female brain, with distinct changes in attention and sensory networks compared to the first pregnancy. The research also links brain changes to mother-child bonding and peripartum depression.
The GE HealthCare-Mayo MI-BET collaboration advances biomarker-guided prostate cancer therapy. The precision oncology market, valued at $122.6B in 2025, is projected to reach $317.2B by 2034, fueled by genomics and targeted treatments.
FDA approved subcutaneous isatuximab-irfc for multiple myeloma. Phase 3 EMN24 trial showed 77% MRD negativity with Isa-KRd quadruplet therapy. Phase 2 study showed 77% hematologic response rate in relapsed/refractory AL amyloidosis.
Phase I CARLYSLE trial data show CAR-T therapy obe-cel induces remission in severe refractory lupus with a favorable safety profile. Separate case report documents two successful pregnancies after dual-target CAR-T therapy with no transmission to infants.
Keenova's Xiaflex met its primary and secondary endpoints in a Phase 3 trial for plantar fibromatosis. The company plans to file for FDA approval before year-end. A separate real-world evidence manuscript highlighted the lack of approved nonsurgical options for the condition.
Phase III data show deramiocel slowed upper limb decline by 54% in Duchenne muscular dystrophy. The FDA is re-evaluating the BLA with HOPE-3 results, with a submission of the clinical study report expected in February 2026.
