Clinical Trial News

A phase I trial (BrainChild-03) tested B7-H3-specific CAR T cells in children with DIPG, showing feasibility of repeated intracranial dosing, local immune activation, and CAR T cells persistence in CSF, with one patient showing sustained improvement over 12 months.

The ALS drug development pipeline is rich with promising therapies targeting TDP-43 pathology, including Biogen’s BIIB-105, AbbVie/Calico’s ABBV-CLS-7262, Denali’s DNL-343, and QurAlis’ QRL-201. Trials range from Phase 1 to Phase 3, with completion dates extending to 2026. These therapies aim to slow ALS progression by addressing TDP-43 mislocalization and aggregation, a hallmark of ALS pathology. The article also highlights the importance of clinical trials in advancing ALS treatment and the potential for FDA approval of these therapies.

Immix Biopharma's additional NXC-201 clinical data for relapsed/refractory AL Amyloidosis will be presented at the 20th International Myeloma Society Annual Meeting in Athens, Greece, September 27-30, 2023. Dr. Polina Stepensky highlighted progress in providing new treatment options for patients.

The European Commission approved axicabtagene ciloleucel (axi-cel; Yescarta) for treating adult patients with DLBCL or HGBL who relapse within 12 months post-first-line chemoimmunotherapy. Based on the ZUMA-7 trial, axi-cel significantly improved event-free survival over standard care, marking a new treatment era. Axi-cel also showed a consistent safety profile and improved quality of life.

ACELYRIN reported Phase 2b/3 trial data for izokibep showing early improvements in Hidradenitis Suppurativa patients, with Part B results expected Q3 2023. Psoriatic Arthritis trial enrollment completed, top-line data accelerated to Q1 2024. Strong cash position of $823M supports clinical programs. Leadership updates include Ken Lock as Chief Commercial Officer.

Xeltis secured €12.5M from EIC Fund, totaling €44.5M raised. Funds will advance clinical trials for aXess vascular graft for chronic kidney disease patients and coronary artery bypass graft program, utilizing a polymer implant that regenerates patient's tissue.

Wegovy, a weight-loss drug, reduces heart attack risk by 20% in obese individuals with heart disease history, showcasing health benefits beyond weight loss. Despite high costs and limited insurance coverage, experts see potential for broader acceptance and treatment of obesity as a serious illness.

Elacestrant improved progression-free survival in HR-positive, HER2-negative advanced breast cancer patients with ESR1 mutations, leading to updated ASCO guidelines recommending routine ESR1 mutation testing at progression. Blood-based ctDNA testing is preferred for its sensitivity and safety. Other treatments include endocrine therapy with targeted agents for non-ESR1 mutation cases.

Jacobio Pharma's KRAS G12C inhibitor, glecirasib, received breakthrough therapy designation by China's CDE for pancreatic cancer patients with KRAS G12C mutation post-standard care. This designation, based on clinical trial data, aims to expedite development and patient access. Glecirasib also has BTD for NSCLC in China.

The FDA has approved avacincaptad pegol (IZERVAY) for treating geographic atrophy secondary to age-related macular degeneration, marking a significant advancement in addressing this form of vision loss.