New trial results for GSK’s oral gepotidacin, a potential first-in-class oral antibiotic, has shown that it was non-inferior with 92.6 percent success rates in patients with uncomplicated urogenital gonorrhoea (GC). “These results highlight the potential of gepotidacin as a new oral treatment option given the rising incidence of gonorrhoea worldwide” In the pivotal EAGLE-1 Phase III trial, comparatively, intramuscular ceftriaxone (500mg) plus oral azithromycin (1,000mg) combined therapy, a leading combination treatment regimen for gonorrhoea, provided success rates of 91.2 percent. Gepotida ..read more

Roche’s twice-yearly, 10-minute subcutaneous injection of OCREVUS ® (ocrelizumab) has shown significant promise for patients with either with relapsing or primary progressive multiple sclerosis (RMS or PPMS). One-year data from the Phase III OCARINA II trial demonstrated a near-complete suppression of relapse activity (97.2 percent had no relapse) in patients through 48 weeks of the treatment. About OCREVUS The monoclonal antibody is designed to target CD20-positive B cells. Roche stated that the subcutaneous formulation of the treatment facilitated “rapid and sustained B-cell depletion in th ..read more

Novartis has released new data from the first and only Phase III study to demonstrate significant proteinuria reduction by targeting the complement system in patients with the rare kidney disease IgA nephropathy (IgAN). Pre-specified interim analysis results from the Phase III APPLAUSE-IgAN clinical trial were presented at the 2024 World Congress of Nephrology (WCN). Fabhalta ® (iptacopan) is a Factor B inhibitor of the alternative complement pathway. It was investigated as a twice-daily oral treatment in the trial. The small molecule treatment enabled a 38.3 percent proteinuri ..read more

Positive data from MaaT Pharma’s Microbiome Ecosystem TherapyTM (MET) MaaT013 as a treatment for acute graft-versus-host disease (aGvHD), is set to be presented at the 2024 European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting. Based on the long-term survival data, at 18 months MaaT013 enabled 42 percent overall survival in all patients and 58 percent in responder patients. “MaaT013 shows remarkable efficacy at 18 months, yielding more complete responses in aGvHD patients who have shown resistance to current treatments, as compared to other available therapies. This effe ..read more

The first-of-its-kind global Phase II study for vosoritide has demonstrated that children with hypochondroplasia, a genetic cause of short stature in children, achieved an increased average growth rate of 1.8 cm annually. [vosoritide] is the first medicine that has been developed to specifically target the pathway involved in hypochondroplasia” “This is the first medicine that has been developed to specifically target the pathway involved in hypochondroplasia,” commented Dr Andrew Dauber, Chief of Endocrinology at Children’s National Hospital in the US. The peptide-based treatment was ..read more

There has been a “notable” rise in licensing agreements for innovator drugs incorporating clustered regularly interspaced short palindromic repeats (CRISPR)-based technology for gene therapies over the past five years, according to data and analytics firm GlobalData. These agreements have amassed a total deal value of $21 billion. Of note, between 2020 to 2022, there was a “remarkable surge” in deal worth. For agreements relating to or involving treatments for haematological disorders, the total deal value reached $1.8 billion, the research found. For instance, the approval of Casgevy in the ..read more

A new clinical study has demonstrated that when administered immediately post-childbirth, one low dose injection of esketamine reduced major depressive episodes in individuals with prenatal depression. Based on the data, the researchers concluded that low dose esketamine should be considered in mothers with symptoms of prenatal depression. In mothers with prenatal depressive symptoms, a single low dose of esketamine given shortly after childbirth decreased major depressive episodes at 42 days postpartum by about three quarters, the findings showed.  As such, the investigators estimated t ..read more

First evidence of clinical activity in the brain for a targeted protein degrader has been demonstrated with NX-5948, an orally available, selective degrader of Bruton’s tyrosine kinase (BTK). The small molecule anti-cancer treatment from Nurix Therapeutics is currently being investigated in a Phase I clinical trial in patients with relapsed or refractory B cell malignancies. NX-5948: a novel small molecule treatment One case study demonstrated partial response with the BTK degrader in chronic lymphocytic leukaemia with secondary central nervous system (CNS) involvement and disease progression ..read more

Over the past decade, there has been an increase in discussions involving industry stakeholders, regulatory agencies, and physicians about developments in the emerging microbiome therapeutics industry. The field initially triggered inquiries and these conversations have since evolved, indicating a noticeable positive shift in attitudes over time.  The recent FDA approvals of two microbiome products in the US, in November 2022 and April 2023,1-2 represent a significant milestone, maturing the industry and paving the way for advancement and regulatory clearance of additional microbiom ..read more

Interim data from a Phase I/II clinical trial suggest that mRNA-3927, an investigational mRNA therapy from Moderna, could be a promising treatment for propionic acidaemia. A 70 percent reduction in the risk of metabolic decompensation events was reported by eight participants in the 12-month pretreatment period, according to the trial results published in Nature. “We are excited to share the first published clinical data utilising an mRNA therapy for intracellular protein replacement,” shared Dr Kyle Holen, Moderna’s Senior Vice President and Head of Development, Therapeutics and Oncology. A ..read more