In the midst of regulatory and political upheaval, biopharma’s R&D engine kept running, churning out highs and lows in equal parts. Here are some of this year’s most glorious clinical trial victories.
2025 continued a difficult stretch for biopharma, as funding continued to crawl for much of the year before showing signs of life in the fourth quarter—and several companies experienced regulatory whiplash as the FDA seemingly reversed positions it had only recently communicated.
Add to this an FDA that is hemorrhaging institutional knowledge and a health secretary consistently communicating scientifically questionable positions and it’s been a rollercoaster of a year.
Still, the industry perseveres. Drugmakers continued to advance their investigational products, braving the turbulent drug development waters in the hopes of bringing an innovative therapy to the market. And while this effort often ends in failure—90% of investigational drugs flunk out in clinical trials—several companies, including uniQure, Roche and Novo Nordisk clinched key clinical victories in challenging and lucrative indications.
In this piece, BioSpace looks back at some of the biggest trial successes this year, reviewing not only the data but also their broader impact on the company and industry as a whole.
UniQure Scores Historic Huntington’s Win, Despite FDA Pushback
On Sept. 24, uniQure announced that its gene therapy slowed the progression of Huntington’s disease by 75% at the three-year mark. Aside from disease progression, AMT-130 also hit key secondary endpoints, improving functional capacity and lowering the concentrations of a key disease biomarker versus outside comparators.
The data exceeded analyst expectations and could pave the way for the first genetic treatment for the devastating neurodegenerative disorder—barring any regulatory roadblocks.
But that’s exactly what uniQure ran into in just five weeks later. Last month, despite having aligned with the FDA on the protocols and analytical models used in the pivotal study, uniQure revealed that the regulator “no longer agrees” that Phase I/II data for AMT-130 is “adequate to provide the primary evidence in support of a [biologics license application] submission.”
UniQure had been planning to file a BLA early in 2026, but following the FDA’s seeming reversal, the timeline for submission is now “unclear,” the biotech said in November.
In a Nov. 3 note, Stifel analysts called the FDA’s about-face “a shock,” adding that the decision could have far-reaching stock implications not just for uniQure but also for “other development stage companies in the gene therapy space.”
Elsewhere in the Huntington’s space, PTC therapeutics reported positive Huntington’s data in May from a Phase II study of its small-molecule drug PTC518, though analyst reaction has been mixed. While the drug elicited a 23% reduction in the huntingtin protein, William Blair in a May 5 note said that a link between the biomarker and functional remains to be proven. The analysts also flagged the lack of functional benefit in patients with later-stage disease.
Metsera’s Weight-Loss Win-Streak Sparks Big Pharma Tug-of-War
Rising obesity star Metsera captured the industry’s attention in September last year when it announced that its GLP-1 injectable MET-097i achieved 7.5% weight-loss at 36 days in an early study. This year, the New York biotech proved that its success wasn’t a fluke—and secured a nearly $10 billion buyout for its efforts.
Metsera kicked off 2025 with more data for MET-097i, this time from a Phase IIa trial involving 120 patients with overweight or obesity, but without type 2 diabetes. After 12 weekly doses, patients on the GLP-1 injection dropped 11.3% of their body weight versus placebo. Some patients lost as much as 20% of their weight, the biotech said at the time, adding that it did not observe any plateauing effect.
Metsera followed this up with another mid-stage data drop in late September, touting a 14.1% placebo-adjusted weight reduction with MET-097i at 28 weeks—findings that Leerink Partners in a Sept. 30 note called “very encouraging.” Eli Lilly’s blockbuster medicine, tirzepatide,“achieved about a 13% placebo-adjusted weight-loss” at a similar length of follow-up, the analysts noted.
Late-stage development for MET-097i is slated for late-stage development within the year.
Beyond MET-097i, Metsera’s ultra-long-acting amylin injectable MET-233i induced 8.4% weight-loss versus placebo at 36 days in a Phase I study that read out in June. Some patients on MET-233i saw a 10.2% reduction in body weight.
These results were enough to ignite one of the most high-profile—if not messiest—biopharma bidding war—in recent history. Pfizer in mid-September announced that it would acquire the biotech for $4.9 billion, setting the pharma up for an obesity comeback after suffering several failed assets. A month later, however, Novo Nordisk submitted a rival bid worth $8.5 billion.
This intrusion—which Metsera seemed to welcome, calling the offer “superior” to Pfizer’s—set off a very public back-and-forth between the companies, involving lawsuits from the New York pharma. Ultimately, Pfizer prevailed, upping its offer to $9.2 billion to definitively win Metsera’s hand in corporate marriage.
Novo Bounces Back With Phase II Amycretin Win
Despite losing out on Metsera , Novo Nordisk nevertheless notched a crucial victory last month as its oral amylin candidate amycretin demonstrated a “competitive” profile in a mid-stage diabetes study.
In a Phase II trial of nearly 450 patients with type 2 diabetes, Novo’s amylin pill led to 7.6% weight loss at 36 weeks as compared with placebo. The subcutaneous injection, meanwhile, cut 11.9% off patients’ body weight. BMO Capital Markets called this efficacy profile “competitive,” noting amycretin’s “potentially differentiated profile” in the increasingly crowded space of oral obesity treatments.
“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 elicited a 1.5% decrease in blood glucose levels, as represented by HbA1c at the same time point. A subcutaneous formulation of the drug, meanwhile, elicited a 1.8% HbA1c drop.
Amycretin’s Phase II win helps deliver some vindication for Novo’s obesity franchise, which in recent months has been struggling both clinically and commercially. Its blockbuster drug Wegovy, for instance, has been ceding market share to Lilly’s Zepbound, despite breeching the market first. Novo has blamed compounders for Wegovy’s shaky commercial footing, but analysts are skeptical.
During the company’s first quarter earnings call in May, Peter Verdult of BNP Paribas floated a difficult theory: “Is it really only about the compounders or do we have to consider your nearest branded competitor doing a better job?”
The Danish pharma has also been struggling clinically. In December last year, its next-generation weight-loss therapy CagriSema delivered disappointing outcomes. The asset, which combines semaglutide with amylin analog cagrilintide, met its primary endpoint in the Phase III REDEFINE 1 study, but its 22.7% weight-reduction fell short of investor expectations—which Novo itself set—erasing some $72 billion off the company’s market cap.
Meanwhile, a day before the amycretin readout last month, Novo reported that semaglutide was unable to significantly slow Alzheimer’s disease progression in the Phase III EVOKE and EVOKE+ studies. While expectations for this program had not been high, the data drop still knocked some 10% off Novo’s shares in its aftermath.
BMS/BioNTech Eye PD-1/VEGF Leadership With Geographically Consistent Data
One of the most closely watched—not to mention most clinically promising—trends in cancer today is the rise of PD-1/VEGF inhibition.
Achieved through the use of bispecific antibodies that bind and block both receptors, the PD-1/VEGF approach rose to prominence in September last year, when Summit Therapeutics and its Chinese partner Akeso claimed victory over Merck’s mega-blockbuster Keytruda. In a late-stage non-small cell lung cancer trial, the partners’ ivonescimab achieved nearly 50% better progression-free survival than the cornerstone cancer therapy.
BioNTech jumped into the space a few months later, acquiring Biotheus for up to $950 million and gaining access to its PD-1/VEGF bispecific BNT327. This investment has paid off nicely for BioNTech. In September, the company reported data from a global Phase II trial showing that BNT327, which has since been named pumitamig, given alongside standard chemotherapy, elicited a 76.3% overall response rate (ORR) in patients with extensive-stage small cell lung cancer. Disease control rate hit 100%.
In a Sept. 8 note, analysts at Truist Securities said that these data were “very reassuring” since they “confirmed the treatment effect seen in China.” Analysts were referring to a Phase II readout from pumitamig in March, which demonstrated a confirmed ORR of 85.4% in Chinese patients. Disease control rate in this trial was 97.9%.
Encouraged by these data, BioNTech has taken pumitamig into late-stage development in the same indication, for which it is running the Phase III ROSETTA-LUNG-01 trial. To establish the antibody’s efficacy across different patient populations, the study is enrolling across different geographies, including the U.S., China, Australia and Korea. ROSETTA-LUNG-01 is set to complete in March 2029.
Aside from performing well in the clinic, pumitamig has also helped BioNTech garner hefty support from the industry’s heavy hitters. In June, Bristol Myers Squibb put more than $11 billion on the line—including $3.5 billion upfront and up to $7.6 billion in milestones—for the right to co-develop and co-commercialize the asset.
Roche Makes Alzheimer’s Comeback With Mid-Stage Win
Neuroscience has classically been a challenging space for biopharma, and 2025 was no different. With many companies posting clinical failures in this space—Roche secured a key victory this year in one of the most intractable indications: Alzheimer’s disease.
At the 2025 Clinical Trials on Alzheimer’s Disease (CTAD) meeting earlier this month, the pharma presented data from its ongoing Phase I/II Brainshuttle AD study demonstrating that 92% of patients treated with its next-gen anti-amyloid antibody trontinemab achieved amyloid levels lower than 24-centiloid, a key threshold determining the presence of amyloid plaques on a PET scan. Trontinemab also showed a potential benefit on the tau accumulation in the brain.
As for safety, amyloid-related imaging abnormalities suggestive of brain swelling or bleeding occurred in fewer than 5% of treated patients.
These findings echo an earlier data drop during the 2025 Alzheimer’s Association International Conference (AAIC) in July. In a presentation at the meeting, Roche reported that 91% of trontinemab-treated patients tested negative on a PET scan for amyloid in the brain, while 72% achieved “deep clearance” of amyloid clumps.
Trontinemab represents an Alzheimer’s comeback for Roche. In late 2022, Roche’s gantenerumab, likewise an anti-amyloid antibody failed two Phase III studies, forcing the pharma to pull the plug on the program.
Meanwhile, at-home administration of anti-amyloid drug Leqembi is becoming a reality, companies like Eisai continue to advance candidates against tau, and 30-plus trials are underway against what Alzheimer’s Drug Discovery Foundation Co-founder and Chief Science Officer Howard Fillit recently told BioSpace earlier this month is the industry’s hottest target: inflammation. “Forty years of research is finally paying off,” he said.
