2025 was a challenging year for the biopharma industry, marked by slow funding and shifting regulatory positions from the U.S. Food and Drug Administration (FDA). Despite these obstacles, several companies achieved significant clinical milestones in drug development.
UniQure made headlines in September with data showing its gene therapy AMT-130 slowed Huntington’s disease progression by 75% over three years. The therapy also improved functional capacity and reduced a key disease biomarker compared to external comparators. However, five weeks later, uniQure reported that the FDA no longer considered its Phase I/II data sufficient to support a biologics license application. UniQure stated that its timeline for submission is now “unclear.” Stifel analysts described the FDA’s reversal as “a shock,” noting potential broad implications for other gene therapy developers.
In the obesity treatment sector, Metsera attracted attention after reporting that its GLP-1 injectable MET-097i led to an 11.3% weight loss over 12 weeks in a Phase IIa trial of patients without type 2 diabetes. Some participants lost up to 20% of their body weight. Further results showed a placebo-adjusted weight reduction of 14.1% at 28 weeks, which Leerink Partners called “very encouraging.” These findings sparked a high-profile acquisition battle between Pfizer and Novo Nordisk, with Pfizer ultimately acquiring Metsera for $9.2 billion.
Novo Nordisk, despite losing out on Metsera, reported positive mid-stage results for its oral amylin candidate amycretin in type 2 diabetes patients. The pill produced a 7.6% weight loss at 36 weeks versus placebo; the injectable version achieved an 11.9% reduction. BMO Capital Markets described amycretin’s profile as “competitive” and noted no plateau in weight loss during the study period: “Importantly, no weight loss plateau was observed in either administration route, suggesting the potential for additional weight loss at longer time points,” BMO added.
Amycretin also lowered blood glucose levels by up to 1.8%. This success follows recent setbacks for Novo Nordisk’s obesity franchise—its next-generation therapy CagriSema failed to meet investor expectations in late 2024, leading to a sharp drop in market capitalization.
In oncology, BioNTech advanced its PD-1/VEGF bispecific antibody pumitamig (BNT327), acquired through Biotheus, into late-stage development after global Phase II results showed a 76.3% overall response rate among small cell lung cancer patients receiving standard chemotherapy alongside pumitamig; disease control reached 100%. Truist Securities analysts found these results “very reassuring” as they confirmed earlier findings from Chinese trials.
Bristol Myers Squibb partnered with BioNTech on pumitamig this year, committing more than $11 billion—including $3.5 billion upfront—to co-develop and commercialize the asset globally.
Roche returned to prominence in Alzheimer’s research with mid-stage data on trontinemab presented at major conferences this year. In one study, 92% of treated patients reached low amyloid plaque levels on PET scans; fewer than 5% experienced brain swelling or bleeding events linked to treatment. Earlier results from July indicated that most trontinemab-treated patients tested negative for brain amyloid presence and many achieved deep clearance of plaques.
This represents a turnaround for Roche following previous failures with gantenerumab in late-stage Alzheimer’s trials two years ago.
The Alzheimer’s field remains highly active: Eisai continues work on anti-tau therapies despite recent failures by competitors such as Johnson & Johnson's posdinemab candidate. Howard Fillit of the Alzheimer’s Drug Discovery Foundation commented earlier this month: “Forty years of research is finally paying off.”
BioSpace reviewed these developments as part of its ongoing coverage of clinical advances shaping biopharma's future.
