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1. U.S. FDA Approves ABRYSVO™, Pfizer’s Vaccine for the Prevention of Respiratory Syncytial Virus (RSV) in Older Adults
Pfizer announced that the U.S. FDA has approved ABRYSVO™, the company’s bivalent RSV prefusion F (RSVpreF) vaccine, for the prevention of lower respiratory tract disease caused by RSV in individuals 60 years and older. The FDA’s decision is based on the data from the pivotal Phase 3 clinical trial (NCT05035212) RENOIR. At the interim analysis, the vaccine efficacy for RSV-associated lower respiratory tract illness with at least two signs or symptoms was 66.7%, and the efficacy for RSV-associated lower respiratory tract illness with at least three signs or symptoms was 85.7%.
2 Press Release: Positive Phase 2 data of novel investigational anti-CD40L antibody frexalimab show significantly reduced disease activity in relapsing multiple sclerosis
Sanofi annoucned phase 2 data of its anti-CD40L antibody frexalimab in patients with relapsing multiple sclerosis. Following 12 weeks of therapy, the number of new gadolinium-enhancing (GdE) T1-lesions was reduced by 89% and 79% in the higher- and lower-dose treatment arms, respectively, compared with placebo, meeting the study’s primary endpoint. Sanofi plans to initiate pivotal trials in multiple sclerosis in early 2024.
3 Rocket Pharmaceuticals Receives European Medicines Agency (EMA) Priority Medicines (PRIME) Designation for RP-A501 Gene Therapy for Danon Disease
Rocket Pharmaceuticals announced that the EMA has granted Priority Medicines (PRIME) designation to RP-A501, an AAV-based gene therapy for the treatment of Danon Disease. PRIME designation was granted based on positive safety and efficacy data from the Phase 1 clinical trial of RP-A501 in patients with Danon Disease. The data demonstrated consistent and robust improvements in multiple clinical and highly relevant laboratory parameters including LAMP-2 protein expression, reduced autophagic vacuoles, BNP, high sensitivity troponin I, and left ventricular mass and wall thickness. In addition, there was improvement in symptoms, as assessed by NYHA class and quality of life, as measured by the KCCQ.
4 Enfortumab vedotin in the previously treated advanced head and neck cancer (HNC) cohort of EV-202.
Astellas presented the phase 2 data of enfortumab vedotin in patients with head and neck squamous cell carcinoma. As of April 11, 2022, DCR was 56.5% (n = 26). Median time to respond was 1.74 mo. Median PFS and OS were 3.94 and 5.98 mo, respectively. Median DOR was not reached. Common AEs were fatigue, alopecia, and peripheral sensory neuropathy (28.3% for each; n = 13). Grade ≥3 AEs occurring in > 1 pt were anemia (n = 3), decreased neutrophil count (n = 2), and malignant neoplasm progression (disease progression of HNC; n = 2). Of treatment-related AEs of special interest for EV, skin reactions occurred in 45.7% of pts, peripheral neuropathy in 32.6%, and hyperglycemia in 4.3%.
5 Head-to-Head Comparison of TLL-018 and Tofacitinib in Patients with Active Rheumatoid Arthritis: Interim Results from a Phase IIa Study
Highlight Pharma Presents data from the phase 2a trial of TLL-018 in patients with rheumatoid arthritis. At week 12, ACR50 response rates in TLL-018 treated groups [10mg, 20mg and 30mg, 48.0% (95% CI, 28.42 - 67.58), 65.4% (95% CI, 47.10 - 83.67), 72.0% (95% CI, 54.40 - 89.60), respectively] were higher than that for tofacitinib [41.7% (21.94 - 61.39)]. TLL-018 20 and 30mg were statistically superior to tofacitinib (p<0.05). Proportions of patients achieving clinical remission (DAS28-CRP<2.6) at week 12 were 39.1%, 34.8%, 54.5% and 17.4% at week 12 for the 10, 20, 30mg TLL-018 and tofacitinib, respectively. TLL-018 20 and 30mg demonstrated high efficacy in patients who had prior bDMARDs, achieving ACR50 rates of >66%.
6 Glyscend Therapeutics Announces Positive Topline Phase 2a Clinical Results and Progress with GLY-200, a First-in-Class Treatment for Type 2 Diabetes and Obesity
Glyscend Therapeutics announced clinical progress with its lead candidate, GLY-200, for the treatment of type 2 diabetes (T2D) and obesity. GLY-200 is a proprietary mucin-complexing polymer designed to enhance the natural mucus barrier in the duodenum (the upper small intestine). Phase 2a data showed that GLY-200 treatment led to significant reductions in postprandial glucose and body weight in patients with T2D. In addition, GLY-200 was well-tolerated with no treatment-related safety signals, and pharmacokinetic data are consistent with a gut-restricted non-absorbed polymer-drug.
7 Coave Therapeutics announces Positive 12-month Data from Ongoing Phase I/II Clinical Trial of CTx-PDE6b in Patients with Retinitis Pigmentosa Caused by Bi-allelic Mutations in PDE6b
Coave Therapeutics announces the results from the Phase I/II trial of its gene therapy, CTx-PDE6b, for retinitis pigmentosa (RP) caused by bi-allelic mutations in the PDE6B gene (PDE6b RP). Following the 12-month study period, both doses were well tolerated (n=17). A subgroup of patients (n=6) with less advanced disease who received the higher dose showed positive efficacy results, consistently measured across all five clinical endpoints (BCVA, Visual Field, Microperimetry, Full Field Sensitivity Test, and Mobility test) and the retinal anatomical evaluation by OCT. In this sub-group, using microperimetry, a significant favorable progression of sensitivity of the four central loci of the retina was observed in the treated eyes compared to the untreated eyes.
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