Biopharmaceutical company Annexon has reported promising results from its Phase I single ascending dose (SAD) and multiple ascending dose (MAD) healthy volunteer study of ANX1502. ANX1502 is a first-in-kind oral, selective small molecule inhibitor that targets the active form of C1s responsible for propagating classical pathway… Read More
A new drug for atopic dermatitis (AD) has demonstrated a positive clinical effect and improvements in various efficacy endpoints after four weeks of treatment. The Phase Ia/IIb study was designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and CCR4 surface receptor occupancy of zelnecirnon in eligible… Read More
Positive Phase I results for a new solid dose prescription cannabidiol (CBD) demonstrate the tolerability and bioavailability of the drug. Neuroscience drug discovery company Sapient Therapeutics successfully completed Part B of the Phase I trial comparing its CBD therapy SAP021 with Epidiolex. The trial… Read More
Insilico Medicine has initiated the first-in-human study for ISM5411, a poly hydroxylase domain (PHD) 1/2 inhibitor for the treatment of inflammatory bowel disease (IBD) designed by its generative AI platform. The company also plans to conduct global, multicentre Phase Ib trials in patients with ulcerative colitis… Read More
The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication. Read More
Data presented at the American Society for Hematology meeting (ASH) show a 70% response rate in last line multiple myeloma patients treated with CellCentric’s inobrodib in combination with pomalidomide (pom) and dexamethasone (dex). Inobrodib is the first in a new class of drug, which disrupts the… Read More
OSE Immunotherapeutics and Nantes University Hospital have revealed positive interim data analysis from the FIRsT Phase I/II study evaluating FR104/VEL-101 immunotherapy in renal transplants. The data was presented the Annual Meeting of the Francophone Society of Transplantation (SFT, Société Francophone de Transplantation) held in Brest, France. Read More
Inipharm has initiated Phase I dosing of its lead candidate, INI-822, which is a small-molecule inhibitor for HSD17B13, a gene implicated in nonalcoholic steatohepatitis (NASH). Loss-of-function variants of HSD17B13 are known to be associated with reduced incidence risk and severity of multiple liver diseases. Read More
A potential schizophrenia treatment, discovered at Vanderbilt University in Tennessee in the US, has been cleared by the US Food and Drug Administration for use in Phase I clinical trials. NMRA-266, an allosteric modulator that works through a mechanism that has been clinically validated in the… Read More
Arcellx has revealed encouraging interim data from its Phase I study of CART-ddBCMA in patients with relapsed or refractory multiple myeloma. The company plans to present new data with a median follow-up of 26.5 months at the 65th American Society of Hematology (ASH) Annual Meeting and… Read More
Asklepios BioPharmaceutical (AskBio), a gene therapy subsidiary of Bayer, has presented first-in-human data from a Phase I trial investigating AB-1002 (also known as NAN-101) for the treatment of patients with congestive heart failure (CHF). The data were shared at the American Heart Association Scientific Sessions, which… Read More
Topline results from a Phase I/II clinical trial have shown that Kiora’s light-restoring small molecule KIO-301 has the potential to meaningfully improve vision in patients with retinitis pigmentosa (RP). The results of the ABACUS study for Kiora’s intravitreal (IVT) molecular photoswitch were presented at the American… Read More
Gate Neurosciences has announced positive topline quantitative EEG (qEEG) biomarker and safety results from its Phase I multiple ascending dose study of apimostinel in healthy volunteers. The results demonstrated a dose-dependent increase in qEEG pharmacodynamic biomarkers of NMDA receptor target activation from baseline, compared with subjects… Read More
Initial data from an ongoing Phase I/II study of HPK1 inhibitor NDI-101150 show clinical benefit in three patients with solid tumours, including one complete response in a patient with renal cell carcinoma. Two patients with pancreatic cancer and renal cell carcinoma, respectively, exhibited prolonged (> six… Read More
UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The UK Medicines and Healthcare Products Regulatory Agency (MHRA), Research Ethics Committee (REC), and Health Research Authority (HRA) have all approved the clinical trial application that was submitted… Read More
The latest episode of the DDW Highlights podcast is now available to listen to below. DDW’s Megan Thomas narrates five key stories of the week to keep DDW subscribers up-to-date on the latest industry updates. The news highlights this week feature a variety of treatment approaches, from… Read More
