1. Discontinuation of oral GLP-1 receptor agonist due to unexpected liver damage in one trial participant
On April 14, 2025, Pfizer decided to discontinue the development of danuglipron (PF-06882961), which is an oral GLP-1 receptor agonist for chronic weight management. Initially, the study met its key primary pharmacokinetic objectives and confirmed a formulation and dose that could deliver a competitive efficacy and tolerability profile. However, among the participants, a participant in one of the dose-optimisation studies experienced potential drug-induced liver injury, which resolved after discontinuation of danuglipron. After a thorough consideration, Pfizer has decided to discontinue development of the molecule.
2. Higher survival showed by CD3xCD20 bispecific antibody combination therapy compared to anti-CD20 combination
On April 14, 2025, Roche announced that the European Commission has approved Columvi® (glofitamab) in combination with gemcitabine and oxaliplatin (GemOx) for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL). Columvi is a CD20 and CD3 T-cell engaging bispecific antibody designed to target CD3 on T cells and CD20 on B cells.
The approval is based on results from the pivotal phase III STARGLO study, where Columvi combination therapy demonstrated a statistically significant and clinically meaningful overall survival (OS) improvement versus MabThera®/Rituxan® (rituximab, anti-CD20) and GemOx (R-GemOx). In the primary analysis, there was a 41% reduction in the risk of death in patients treated with Columvi plus GemOx versus R-GemOx. Columvi combination also met its key secondary endpoints, with a 63% reduction in risk of disease worsening or death. Moreover, there is a 25.5-month median OS for people treated with the Columvi combination compared to 12.9 months for people on R-GemOx.
Link: https://www.roche.com/media/releases/med-cor-2025-04-14
3. Updates on trials data and regulatory decision for Camzyos (mavacamten)
On April 14, 2025, BMS announced that the Phase 3 ODYSSEY-HCM trial, which evaluates Camzyos (mavacamten) for the treatment of adult patients with symptomatic New York Heart Association (NYHA) class II-III non-obstructive hypertrophic cardiomyopathy (nHCM) did not meet its dual primary endpoints of changes from baseline to Week 48 compared to placebo in the Kansas City Cardiomyopathy Questionnaire – Clinical Summary Score (KCCQ-23 CSS) and peak oxygen consumption (pVO2). CAMZYOS® (mavacamten) is the first cardiac myosin inhibitor approved in the U.S.
Moreover, on April 17, 2025, BMS, U.S. FDA has updated the prescribing information for CAMZYOS® (mavacamten), simplifying treatment for patients and physicians by reducing the required echo monitoring for eligible patients in the maintenance phase and expanding patient eligibility by reducing contraindications. FDA-approved updates include reduced frequency of echo monitoring from once every 12 weeks to every 6 months.
4. Approval of Dupixent to treat chronic spontaneous urticaria in U.S. for adults and adolescents aged 12 years and older
On April 18, 2025, the U.S. FDA has approved Dupixent (dupilumab) for the treatment of adults and adolescents aged 12 years and older with chronic spontaneous urticaria (CSU) who remain symptomatic even with histamine-1 (H1) antihistamine treatment. Dupixent inhibits the IL-4 and IL-13 signalling pathway. The US approval is based on data from two phase 3 clinical studies assessing Dupixent in addition to standard-of-care antihistamines, compared to antihistamines alone. Both studies met their primary and key secondary endpoints, with Dupixent demonstrating reductions in itch severity and urticaria activity (a composite of itch and hives) compared to placebo at 24 weeks. Additionally, Dupixent also increased the rate of well-controlled disease or complete response compared to placebo at 24 weeks.
Link: https://www.sanofi.com/en/media-room/press-releases/2025/2025-04-18-15-15-00-3064131
5. Oral small-molecule GLP-1 agonist showed reduced blood sugar level
On April 17, 2025, Eli Lilly and Company announced positive Phase 3 results from the ACHIEVE-1 study, evaluating the safety and efficacy of orforglipron compared to placebo in adults with type 2 diabetes and inadequate glycemic control with diet and exercise alone. Orforglipron is a small-molecule GLP-1 receptor agonist that can be taken orally.
In the first Phase 3 trial of the ACHIEVE program, orforglipron met the primary endpoint of higher A1C reduction compared to placebo at 40 weeks. A1C has been reduced by an average of 1.3% to 1.6% from a baseline of 8.0%. In a key secondary endpoint, more than 65% of participants taking the highest dose of orforglipron achieved an A1C less than or equal to 6.5%, which is below the American Diabetes Association's (ADA) defined threshold for diabetes. In an additional key secondary endpoint, participants taking orforglipron lost an average of 16.0 lbs (or 7.9% in terms of percentage) at the highest dose.
6. Leqembi® has been granted marketing authorisation in the European Union
On April 15, 2025, Eisai and Biogen announced that the European Commission (EC) has granted the amyloid-beta (Aβ) monoclonal antibody Leqembi® (lecanemab) marketing authorisation in the European Union. Lecanemab is indicated for the treatment of adult patients with a clinical diagnosis of mild cognitive impairment and mild dementia due to early AD who have no or one copy of the apolipoprotein E ε4 (ApoE ε4*) gene with confirmed amyloid pathology. The EC’s authorisation was based on the Phase 3 data from the global Clarity AD clinical trial, in which it met its primary endpoint and all key secondary endpoints. Lecanemab reduced clinical decline on CDR-SB by 31% at 18 months compared to placebo and a 33% higher maintenance of daily functional abilities.
7. Bemdaneprocel neuron cell replacement therapy is shown to be well-tolerated and safe
On April 16, 2025, BlueRock Therapeutics LP, a clinical-stage cell therapy company wholly owned by Bayer, published Phase 1 clinical trial data for bemdaneprocel in treating Parkinson’s disease. Bemdaneprocel is an investigational cell therapy designed to replace the dopamine-producing neurons that are lost in Parkinson’s disease. At 18 months following surgery, treatment with bemdaneprocel did not cause any serious adverse events related to cell therapy. In addition, the imaging results showed sustained neuron cell engraftment in both high and low dose cohorts even after termination of immunosuppressants. Moreover, in the high-dose cohort, there was an average improvement of 23 points in the Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III OFF scores, suggesting potential motor function benefits.
https://www.nature.com/articles/s41586-025-08845-y
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