10 clinical trials to watch in the first half of 2026
After a lengthy downturn, the biotech industry finally gathered momentum in 2025. Key readouts in obesity, infectious disease and many rare conditions could help it continue.
For the first time in a while, the biotechnology industry starts a new year with momentum.
Large dealmaking has picked up, driving returns for public investors as well as the firms that form and build new drug startups. Multiple biotechs successfully grew sales of new medicines and, as a result, were rewarded with higher market values and lucrative stock offerings. In tandem, the generalist investors that abandoned biotech during a lengthy pullback have, at least for now, returned. A stock index viewed as a barometer of biotech’s health climbed by a third last year, reaching levels not seen since the bubbly days of the pandemic. The first trillion-dollar drugmaker emerged along the way.
Clinical trial results have fueled many of these outcomes, spurring interest from acquirers or investors. This year, other important readouts lay ahead in obesity, infectious diseases, rare disorders and more.
Here are 10 to watch in the first half:
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Lilly could further cement its leading position this year, when a series of studies on a drug called retatrutide produce results.
The drug targets a metabolic pathway called glucagon in addition to the GLP-1 and GIP that Lilly’s fast-selling Zepbound does — a triple-acting effect that could enhance weight loss. Zepbound set a high bar by helping people with obesity lose up to 21% of their body weight over more than a year. But in a Phase 3 trial in a type of knee pain last year, retatrutide delivered even more drastic results, spurring average weight loss of as much as nearly 29% and, in some cases, more than 35%.
Those findings put the obesity field “on notice,” Evercore ISI analyst Umer Raffat wrote in December. But they also weren’t from a study truly designed to maximize weight loss.
Those studies lay ahead, and the first three, titled Triumph-1, -2 and -3, are due to read out toward the middle of the year. The first is a straightforward trial in people with obesity. The second is in people with obesity and diabetes, while the third is in those with obesity and established cardiovascular disease, as defined by a previous heart attack or stroke. Combined, the trials have enrolled more than 5,000 people.
Success in these studies would keep Lilly ahead of a raft of emerging competitors — among them chief rival Novo Nordisk, which is currently trying to win approval of a dual-acting therapy. Novo has its own version of retatrutide in early-stage human trials, meaning it will surely be at least a few years before the company can directly compete with Lilly in the triple-acting obesity drug market. — Jonathan Gardner
Seasonal influenza is one of the world’s most common respiratory infections. Despite the availability of many vaccines and a few acute treatments, global deaths from the flu total anywhere from 290,000 to 650,000 each year, according to the World Health Organization. Protective shots have varying effectiveness and are seeing declining uptake amid a surge in vaccine hesitancy and misinformation, while other therapies are only modestly beneficial and have to be administered quickly once symptoms hit.
This past November, Merck staked more than $9 billion on a potentially new solution. Called CD388, it’s an antiviral that combines a small molecule with a protein fragment. The therapy’s original developer, Cidara Therapeutics, has been evaluating it as a long-acting, preventive treatment for the seasonal flu.
Those results spurred Merck to acquire Cidara in one of 2025’s biggest deals, with CEO Rob Davis saying, on a conference call at the time, that the drug could unlock a $5 billion commercial opportunity. As an antiviral, it’s also notably not under the purview of the influential panel ACIP, which, under new leadership, has proven “chaotic” and leaned “toward conservative recommendations,” Daina Graybosch, an analyst at Leerink Partners, wrote in a November note to clients.
But that’ll only matter if CD388 succeeds in an ongoing, late-stage trial. Called ANCHOR, it’s enrolling about 6,000 participants and measuring CD388’s ability to prevent flu-like illness. Cidara indicated in November that it had reached its enrollment target and intended to conduct an interim analysis in the first quarter. These early looks can yield a stoppage for overwhelming efficacy, clear failure or a decision to expand the trial.
That check will “determine whether we’re on the right path,” Merck’s research chief, Dean Li, said in November. — Ben Fidler
Regenxbio was a latecomer in the biotech industry’s development of gene therapies for Duchenne muscular dystrophy, a progressive and deadly neuromuscular condition. But it could take a substantial leap forward if the results of a Phase 3 trial expected early this year turn up positive.
Like Elevidys, the only marketed gene therapy for Duchenne, Regenxbio’s RGX-202 is designed to produce microdystrophin, a tiny form of the muscular shock absorber Duchenne patients lack. But the therapy is packaged into a different type of microscopic virus, and produces a larger version of the protein that more closely resembles normal dystrophin. The company expects those distinctions to lead to “improved function,” CEO Curran Simpson told BioPharma Dive in 2024.
Results from an early, small trial hinted at such potential. Treated patients showed improvements on multiple measures of muscle function from the study’s start, as well as when compared to historical data, after nine to 12 months of follow-up. And in that study, all patients treated with the dose selected for the Phase 3 trial reached a threshold for microdystrophin production needed for an “accelerated” approval. Regenxbio need only show that patients in its late-stage study safely hit that same mark after three months to declare success.
Regenxbio, for its part, reiterated in November its alignment with the FDA and noted how its regulatory strategy is to show a differentiated safety profile from Elevidys. A pre-approval filing meeting with the FDA will likely come around the time it reveals study results. — Ben Fidler
Editor’s note: This entry was updated June 29, 2025. The trial readout is still pending.
A large clinical trial in its final stages could soon prove whether a protein particle known as Lp(a) will join cardiologists’ checklist for treating heart risk alongside LDL cholesterol and triglycerides.
Run by Novartis, the study is testing an RNA medicine called pelacarsen in people with established cardiovascular disease and elevated levels of Lp(a). Genetic studies have linked high Lp(a) to greater heart risk, while past testing by Novartis and others has shown so-called antisense therapies are effective at lowering Lp(a). Novartis’ trial, which began in late 2019 and has enrolled more than 8,000 people, will go a long way toward showing whether tamping down Lp(a) can reduce major cardiovascular events like heart attacks and stroke.
If successful, the study could position Novartis to seek approval of pelacarsen, a drug it expects to eventually bring in multibillion-dollar sales each year. And it could boost the confidence of rivals Amgen and Eli Lilly that drugs they have in testing could do something similar.
Novartis’ study is event driven, which means that a certain number of heart events must occur before investigators can calculate whether pelacarsen has a heart benefit. While the company previously expected results in 2025, it now anticipates data coming this year. — Ned Pagliarulo
Intellia Therapeutics was one of the first biotech companies built around the Nobel prize-winning, gene editing technology known as CRISPR. But, while Intellia’s clinical progress has at times displayed CRISPR’s massive therapeutic potential, the company has burned through more than $2 billion since inception and twice laid off staff amid shifting research plans.
Intellia still has a shot at success, though. In the first half of 2026, it’s expecting results from a Phase 3 trial testing a possible treatment for a rare condition called hereditary angioedema. That therapy, named lonvo-z for short, has shown the potential to durably reduce the rates of the swelling attacks that characterize the condition.
Intellia has billed this therapy as a possible “functional cure” that might alleviate the need for the frequent injections or pills used to prevent or treat symptoms. It also, so far, hasn’t been beset by the same kind of liver-related issues as another program, nex-z, which is being developed for a type of genetic heart condition.
The results Intellia accrued so far suggest a “good probability of success” in the trial and, assuming an approval afterwards, a “competitive commercial profile,” wrote Evercore ISI analyst Jonathan Miller.
Intellia has pointed to important, technical differences between lonvo-z and nex-z — such as the genes they target and the RNA that “guides” them to it — that could lead to different outcomes. The HAE patients receiving lonvo-z are also typically younger and healthier than those getting nex-z.
“[W]e view lonvo-z and nex-z as absolutely distinct from each other and the patient experience thus far aligns with that,” said CEO John Leonard, on a November conference call, adding that the company expects lonvo-z to “redefine the HAE treatment landscape.”
Yet history isn’t on Intellia’s side. Genetic medicines have struggled to sell in areas where, as with HAE, multiple effective medicines exist. Because of those options, Intellia may need to prove exceptionally potent, durable and safe to entice patients and physicians to try it instead of existing options.
Miller, of Evercore, projects sales would top out at $500 million annually in peak years, given the way gene therapy launches have gone so far. — Ben Fidler
Vertex Pharmaceuticals is best known for a cystic fibrosis drug business that now regularly brings in more than $10 billion each year. But pressure to recreate that success elsewhere is intensifying, as a pain medicine it recently brought to market may not be the blockbuster it once hoped.
One drug Vertex is developing for a rare kidney disease could be part of the solution. Called povetacicept, Vertex acquired it in a roughly $5 billion buyout almost years ago. Povetacicept has since been advanced into a late-stage trial in IgA nephropathy, a progressive condition that can lead to kidney failure.
In pursuing IgA nephropathy, Vertex is attempting to catch and surpass more established competitors. An Otsuka Pharmaceutical drug that works similarly to povetacicept was cleared by U.S. regulators in November, and Vera Therapeutics has already submitted another, comparable therapy to the FDA as well. Yet Vertex executives have claimed their drug has “best-in-class potential,” pointing to a dual-targeting mechanism that Otsuka’s medicine doesn’t possess, and possible efficacy and dosing advantages compared to Vera’s treatment.
Those claims will be put to the test shortly, as an interim check of Vertex’s Phase 3 study in early 2026 could herald an “accelerated” approval filing in the first half. If positive, the findings could also support Vertex’s conviction that povetacicept is a “pipeline in a product,” as it’s seen as having the potential to treat multiple inflammatory conditions. The drug has advanced into late-stage testing for another kidney condition, primary membranous nephropathy, too.
“We have to see the interim analysis data, but assuming it holds up, I think it's a truly exciting opportunity for '26 and beyond,” said David Altshuler, the company’s retiring executive vice president and chief scientific officer, at an investor conference in December. — Ben Fidler
Sarepta Therapeutics’ fortunes nosedived over the last couple years. Safety concerns dimmed the once bright commercial outlook for Elevidys, leading the company to suspend financial guidance, lay off staff and trim its pipeline. Two other Sarepta Duchenne drugs failed confirmatory trials, too, raising questions about the sustainability of its core business. Shares now trade at a small fraction of the more than $160 they were once worth.
Those setbacks have heightened the importance of a partnership Sarepta has with Arrowhead Pharmaceuticals. Forged in November 2024, the deal handed Sarepta rights to an array of RNA-based medicines that Arrowhead had been developing for diseases of the muscles, central nervous system and lungs. Four are in clinical testing, and the first two are expected to produce results early this year.
One drug, SRP-1001, is being tested in a progressive neuromuscular condition called facioscapulohumeral muscular dystrophy. The other, SRP-1003, is for a form of myotonic dystrophy. In both cases, Sarepta and Arrowhead will be presenting preliminary results showing the drugs’ safety and potential to work as intended.
For SRP-1001, that means lowering production of an aberrant protein in skeletal muscle called DUX4. The goal with SRP-1003, meanwhile, is to essentially eliminate a type of mutated messenger molecule that makes harmful proteins in people with myotonic dystrophy.
The results have important implications for Sarepta. The company’s financial struggles already caused it to push off debt payments and sell Arrowhead shares just to ensure it can meet obligations under the existing partnership. A positive outcome — such as implications that these drugs might be superior to other, similar medicines in development — would provide the company with a “lifeline” and “much-needed lift,” wrote Leerink Partners’ Joseph Schwartz, last September. — Ben Fidler
Long dismissed by the broader pharmaceutical industry, psychedelics have recently become one of the most exciting areas in drug development. And there, London-based Compass Pathways is considered a frontrunner.
The company disclosed in June that its proprietary version of psilocybin — a psychedelic compound found in many mushroom species — had hit the main goal of a late-stage study titled “COMP005.” Compared to a placebo, a single dose of the drug significantly reduced symptoms of hard-to-treat depression six weeks into the experiment.
Compass expects that, in the first few months of next year, not only will it have 26-week data from COMP005, but also initial results from a second Phase 3 trial that enrolled faster than anticipated. This “COMP006” study recruited 585 participants with treatment-resistant depression.
How COMP006 reads out is sure to have a major effect on Compass and potentially the broader psychedelics field. The company highlighted during its latest earnings call that the speedy enrollment, along with positive feedback from the FDA, has accelerated its commercialization plans by nine to 12 months. The drug could therefore receive approval by mid-2027, according to Leonid Timashev, an analyst at RBC Capital Markets.
In a note to clients, Timashev wrote that he and his team view Compass as the “highest quality psychedelics story in the space.”
Some investors haven’t bought into that story just yet. Compass’ share price sunk by roughly half with the release of the COMP005 data. Though shares more than rebounded in the months that followed, they again dipped by the double-digits again after the company’s third quarter earnings.
The psilocybin program is “garnering considerable interest and support from specialists, patients and even strategics. It's just investors who have yet to figure this all out,” wrote Cantor Fitzgerald analyst Josh Schimmer in an early November note to clients. — Jacob Bell
Cytokinetics finally secured its first product approval in its nearly three decade-old history last month when the FDA approved its drug for a progressive heart condition. But a coming study result could separate Cytokinetics from its top competitor, and open up that medication to many other people with the disease.
Myqorzo is now one of two drugs, along with Bristol Myers Squibb’s Camzyos, available for hypertrophic cardiomyopathy, a chronic condition characterized by a deadly stiffening of the heart muscle. Both are so-called cardiac myosin inhibitors designed to make the heart’s contractions less forceful. They also now duel for market share among the same patients with the “obstructive” and more common form of the condition. Sales of Camzyos totalled $714 million in the first nine months of 2025 and were expected to top $1 billion for the year.
Last year, though, Camzyos failed in a study testing it in the estimated one-third of people with the “non-obstructive” form. That version of the disease doesn’t impede or “obstruct” blood flow, like its more common counterpart, but can still put people at the risk of poor health outcomes. Camzyos’ setback stirred debate that other drugs like it might similarly struggle. A study investigator at the time claimed Bristol Myers’ failure indicated obstructive and non-obstructive HCM are “two unique diseases.”
Still, some analysts have argued otherwise. Camzyos requires individualized dosing adjustments, and patients whose dosing regimens weren’t interrupted in some way performed better in Bristol Myers’ trial, wrote Stifel’s James Condulis. That suggests a medicine like Myqorzo, which has some different characteristics and a wider potential range of safe and effective doses, should “have a better shot,” he wrote.
Camzyos “isn’t the right tool for the job,” Condulis concluded, leaving Myqorzo with a “credible shot” to be “the best — and only — drug for the entire HCM spectrum.”
The answer will come in the second quarter, when Cytokinetics discloses findings from a study called Acacia-HCM. That trial is testing Myqorzo in an estimated 500 patients with non-obstructive HCM, and evaluating its ability to meaningfully improve peak oxygen consumption as well as scores on an assessment of heart health after 72 weeks. — Ben Fidler
Several companies have challenged Vertex Pharmaceuticals’ dominance in treating cystic fibrosis, only to come up short. Sionna Therapeutics is hoping for better luck, and study results expected in the middle of this year will give important insight into how it might fare.
Sionna raised hundreds of millions of dollars in private and public funding to amass and advance a portfolio of medicines it believes can one-up Vertex. The company designed its therapies to stabilize a tough-to-drug region of the “CFTR” protein that’s defective in cystic fibrosis patients, an approach it thinks could restore lung function better than Vertex’s medicines. Sionna also acquired a trio of prospects from AbbVie for use in multi-drug regimens.
The biotech has been pursuing two strategies in parallel. One involves testing whether the addition of a Sionna “stabilizer” to Vertex’s top-selling medicine, Trikafta, can drive better outcomes than Trikafta alone. A Phase 2 study there is currently underway. The other plan is to evaluate if its own in-house combinations can eventually prove superior to Trikafta. An early-stage trial started in August.
Results from both studies are coming shortly. And in anticipation of those readouts, Sionna shares have been on an extended run that’s given the company a market value nearing $2 billion. To the team of analysts at Stifel, the stock surge is a reflection of “better than expected” Phase 1 results and anticipation that further testing will prove “there’s residual unmet need” in cystic fibrosis.
Others have expressed caution, though. In December, RBC Capital Markets’ Brian Abrahams wrote that Sionna has been given “too optimistic a valuation” given the risks ahead in a “historically challenging space.” Abrahams also warned that Trikafta appeared to underperform in certain Sionna preclinical tests, which may raise questions about how “translatable” those experiments are to the company’s ongoing studies. — Ben Fidler
