10 clinical trials to watch in the second half of 2026
The biotechnology sector’s upswing accelerated in the first half. Positive results from anticipated trials in lung cancer, Alzheimer’s and multiple autoimmune conditions could add to the momentum.
The first half of 2026 featured the kind of news biotechnology investors and executives have been yearning for.
Dealmaking is off to a record pace, rewarding the public and private investors backing drug companies. Several biotechs raised $300 million or more in initial public offerings, a sign there’s real appetite for the startups that buckled down during the sector’s lengthy downturn. The regulatory drama that consumed the industry may have eased somewhat, too, thanks to a White House-directed overhaul reportedly aimed at installing more traditional leaders atop the Food and Drug Administration.
Along the way multiple companies reported scientific milestones against pancreatic cancer, hepatitis B and obesity. Several other, crucial readouts lay ahead in lung cancer, Alzheimer’s, infectious disease and more.
Here are 10 to watch in the second half:
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Drugs known as PD-1/VEGF inhibitors are the source of one of the most intense scientific debates in all of oncology research. Landmark study data from partners Akeso and Summit Therapeutics a couple of years ago suggested these medicines might be even more effective than widely used cancer immunotherapies like Merck & Co.’s Keytruda and Bristol Myers Squibb’s Opdivo. But the results accumulated since have left the future role of PD-1/VEGF drugs less clear.
Study findings expected later this year from a trial called “Harmoni-3” trial should go a long way toward settling the argument. Unlike much of the data compiled by Akeso and Summit so far, Harmoni-3 is a global trial rather than a study run only in China. It’s also testing a combination of ivonescimab and chemotherapy against the Keytruda-chemo regimen that’s often used as frontline care, with the goal of definitively showing the new drug can help delay disease progression and death.
Success could upend care in non-small cell lung cancer, one of the world’s most common tumors. It would also ignite optimism that PD-1/VEGF drugs might be similarly impactful against other cancers. And Akeso and Summit have reasons to feel confident. A similarly structured trial only involving patients in China met its objectives, with an ivonescimab-chemo combo extending survival and holding tumors in check longer than a different immunotherapy-chemo pairing. The data were spotlighted in June at the year’s biggest meeting for cancer research.
Some analysts have viewed those results as a sign ivonescimab will be similarly successful in Harmoni-3. Yet Summit shares fell by double digits after the data were presented, as the chorus of skepticism grew louder.
Some critics, for instance, have pointed to important differences between the two studies. In the China-only trial, patients may have had inconsistent access to care after their disease progressed, muddying the survival data. In addition, that study enrolled younger people than the global Harmoni-3 trial while excluding those over 75 years of age — a group that accounts for 33% of the patients in the U.S. who get Keytruda or other drugs like it. Ivonescimab wasn’t clearly beneficial for enrollees between the ages of 65 and 75 in the China study.
The lack of benefit in these older patients “reduces the likelihood” that the China trial “is relevant,” wrote Leerink analyst Daina Graybosch in a June client note. — Jonathan Gardner
Back in late 2023, Bristol Myers Squibb agreed to spend $14 billion acquiring Karuna Therapeutics, to take control of a first-of-its-kind, mind-stabilizing medicine that analysts viewed as a potential blockbuster treatment for schizophrenia.
Fast forward to mid-2026, and revenue from that medicine — sold as Cobenfy — has largely underwhelmed Wall Street. It totaled $155 million across 2025 and $56 million in the first quarter of this year.
Bristol Myers hopes to beef up Cobenfy sales by showing it can also treat Alzheimer’s disease psychosis. With about 7 million Alzheimer’s patients in the U.S., and roughly a quarter to half experiencing related psychosis, the commercial opportunity is vast. A late-stage trial titled “ADEPT-2” will serve as the first major test of whether Bristol Myers can tap into this market.
Results were on track to arrive earlier, but the timeline got pushed back after Bristol Myers said it found “irregularities” in the way the study was conducted at a “small number” of sites. The delay was met with mixed reactions: some analysts were unnerved, whereas some investors appeared encouraged by the experiment forging ahead.
“While there’s clearly a scenario whereby ADEPT-2 could still [succeed], none of this suggests an overwhelmingly positive result, and all of our past issues with this trial and setting remain,” Carter Gould, of the investment firm Cantor Fitzgerald, wrote last year in a note to clients.
Should the trial miss its main goal, Bristol Myers is running in parallel two other large studies — “ADEPT-1” and “ADEPT-4” — testing Cobenfy in this setting. Those are also set to produce data by the end of the year.
“Cobenfy’s initial sales ramp in schizophrenia has been modest because its efficacy is no better than the gold-standard generic drugs, so it is critical it succeeds in Alzheimer’s Disease psychosis … and benefits from label expansion,” wrote David Risinger, a Leerink Partners analyst, in a February note. — Jacob Bell
Biotech companies don’t always get a second chance when their first drug suffers a major setback. Yet Celldex Therapeutics was able to not only survive multiple crushing clinical defeats, but reinvent itself. It’ll soon find out whether that comeback tale will yield what some analysts view as a blockbuster-to-be.
That drug has come to be known as barzolvolimab and is now in mid- and late-stage testing across a range of inflammatory conditions seen as large market opportunities. The first pivotal studies from that program — a pair of trials in a debilitating itching disorder called chronic spontaneous urticarias — are set to read out in the fourth quarter.
Chronic urticarias cause hives to form on the legs, arms or elsewhere that come with severe itching, burning or swelling. They can either be “induced” by certain triggers, such as heat or sunlight, or occur spontaneously. Millions of people in the U.S. are estimated to be affected, yet treatments — such as the biologic drug Xolair and antihistamines — take weeks to work if they do at all.
Celldex is one of the companies working on a newer alternative. Its treatment works differently than existing therapies, driving down counts of the “mast” cells implicated in the onset of chronic hives. Earlier testing has suggested it might be more potent, fast-acting and durable than available treatments, though those benefits came alongside concerns the drug might drive neutrophil counts too low in the process.
The two placebo-controlled studies set to read out shortly involve nearly 2,000 patients and are designed to test whether barzolvolimab can help patients who still have symptoms despite treatment with antihistamines or other biologic therapies. The drug’s goal is to tamp down itching and hives over the course of 12 weeks.
Enrollment was completed in those as well as other ongoing studies well ahead of schedule, which Stifel analyst Alex Thompson took as a sign that “severe safety worries [are] off the table,” according to a May research note. Should Celldex succeed, it could unlock “a significant commercial opportunity” not only in urticarias, but “other mast-cell-driven diseases,” wrote Leerink Partners’ Thomas Smith. — Ben Fidler
To say cancer vaccines have a checkered past would be an understatement. Many shots meant to spur the immune system to fight tumors of the brain, lung and kidney showed early promise, only to disappoint in their final tests. Different types of immune-boosting drugs have become commonplace in cancer care instead.
For multiple years now, though, anticipation has been building that a vaccine developed by Moderna and Merck & Co. might change the narrative. Called intismeran, it’s a newer kind of personalized vaccine designed to trigger an immune response against dozens of unique protein flags, or “neoantigens,” on the surface of a patient’s tumor.
Earlier testing showed intismeran amplified the effects of a common immunotherapy when administered to patients with advanced melanoma. Updated results from that trial presented at the American Society of Clinical Oncology meeting suggested the benefits might be long-lasting, too, with investigators reporting that a combination of intismeran and Merck’s Keytruda roughly halved the risk of relapse or death after five years of follow-up.
To some Wall Street analysts, those findings have increased the likelihood that Moderna and Merck’s shot will prove successful in a trial that’s now among the most closely watched in all of oncology research. Called Interpath-001, the study is testing that same intismeran-Keytruda combination against Keytruda alone in the “adjuvant” setting, after surgery, in melanoma.
Yet there are major questions ahead of the readout, which is expected sometime over the next 12 months and potentially as early as this year. For one, the Keytruda-only arm of Moderna and Merck’s study may perform better in the late-stage trial than it did in Phase 2, when the drug had a “relatively weak” showing compared to historical data, wrote William Blair analyst Myles Minter.
Leerink Partners’ Mani Foroohar also noted how Interpath-001 includes patients with earlier-stage disease as well as a more “balanced distribution" of people positive for a protein linked to an immunotherapy response. Those factors are likely to “erode [the] benefit” seen in earlier testing, Foroohar wrote.
The financial implications for Moderna and Merck are significant. The promise of Moderna’s oncology work has helped fuel a turnaround over the last year. Merck, meanwhile, is looking to several drugs — intismeran among them — to help boost revenue when Keytruda, its top-selling product, loses patent protection. — Ben Fidler
A deadly heart condition known as transthyretin amyloidosis cardiomyopathy has, in recent years, become one of the more competitive battlegrounds in the biopharmaceutical industry. Three medicines from Pfizer, BridgeBio Pharma and Alnylam Pharmaceuticals are now available, and all are either already blockbuster medicines or soon expected to be. Pfizer’s leading medication, Vyndamax, topped $6 billion in sales last year.
A fourth drug codeveloped by AstraZeneca and Ionis Pharmaceuticals could join that group if a study called “Cardio-TTRansform” produces positive results later this year. Called eplontersen, the drug is already sold as Wainua for another, rarer form of transthyretin amyloidosis that affects the nerves.
Like Alnylam’s Amvuttra, eplontersen is designed to stop production of the protein, TTR, that’s misfolded and causes harm in transthyretin amyloidosis. But eplontersen is a different kind of nucleic acid-based therapy. The study it’s involved in is also larger and designed differently than Amvuttra’s key trial, which could have implications for real-world use of both therapies.
In a recent report, for instance, Leerink’s Foroohar noted how a greater percentage of patients in Ionis and AstraZeneca’s trial were already on other medicines, such as a TTR “stabilizer” like Vyndamax or the “SGLT2 inhibitors” used as adjunctive therapies. That difference might help the companies definitively see whether their therapy can stand out by better benefiting people already on Vyndamax. A statistically significant signal in that group, as well as the overall study population, could boost Ionis shares and pressure Alnylam.
Though comparing drugs across trials is fraught with caveats, investors will also have a close eye on not only how quickly eplontersen appears to have a meaningful impact, but how that effect compares to what was seen in testing of Amvuttra. — Ben Fidler
Biogen, under the leadership of former Sanofi CEO Christopher Viehbacher, has been on a mission over the past several years to expand beyond the high-risk neuroscience projects that accounted for a large portion of the company’s pipeline. A few drugs have become central to that mission, among them an antibody called litifilimab.
A trio of large clinical trials are currently evaluating litifilimab against different kinds of lupus. Two are set to produce results this year. The findings will provide the most concrete evidence yet of whether the drug can meet Wall Street’s blockbuster expectations.
Collectively, the “TOPAZ-1” and “TOPAZ-2” studies have enrolled more than 1,100 people with systemic lupus erythematosus, a more common form of the autoimmune disease that causes inflammation in the organs and tissues. The main goal of each experiment is to see whether Biogen’s therapy is significantly better than a placebo at reducing the severity of the disease, as measured by an index clinicians use to assess lupus symptoms like fevers, headaches, rashes, seizures and muscle weakness.
Andrew Tsai, an analyst at Jefferies, estimates that SLE affects more than 200,000 people in the U.S. While there are a couple marketed medicines, in GSK’s Benlysta and AstraZeneca’s Saphnelo, the disease remains inadequately controlled in many patients. If litifilimab succeeds in SLE as well as another kind of lupus that primarily affects the skin, and for which there are no approved drugs, it could “easily” be a blockbuster product, according to Tsai, who predicts peak sales will surpass $2 billion.
The drug “represents one of [Biogen’s] several late-stage … programs that could help re-accelerate topline growth,” the analyst wrote in a client note this spring. — Jacob Bell
An infection from the bacterium streptococcus pneumoniae can cause a slew of health problems, from mild sinus issues to life-threatening pneumonia and meningitis. That’s made the vaccines that can prevent these pneumococcal diseases among the biggest businesses in the pharmaceutical industry.
For many years, the market for pneumococcal shots has been dominated by Pfizer’s “Prevnar” franchise, which began with a 13-strain option in 2010 and expanded when the company introduced a 20-strain alternative more than a decade later. While others, such as Merck & Co. and GSK, have invested in alternatives in the hopes of grabbing a bigger share, they’ve yet to topple a market-leading business that generated more than $6 billion in sales last year.
One biotech, however, has a chance to achieve what others haven’t. Called Vaxcyte, it’s brought into late-stage testing a next-generation vaccine, VAX-31, designed to cover 31 bacterial strains.
In earlier testing, VAX-31 was able to match the effectiveness of Prevnar 20 in protecting against 20 common strains of bacteria. It’s now being evaluated against Prevnar 20 and Merck’s 21-strain shot Capvaxive in a Phase 3 trial, where VAX-31’s objective is to prove statistically non-inferior to the licensed vaccines. The result is one of the biotech sector’s most highly anticipated among investors. In a June note to clients, Mizuho Securities analyst Salim Syed referred to the coming readout as a “‘must-play’ catalyst for biotech investors.”
One reason for the intense interest among Wall Street analysts is the dearth of close competition. Jefferies analyst Roger Song noted in May that next-generation pneumococcal vaccine programs in development at Sanofi, GSK, Inventprise and Pfizer have recently taken a step back. Their stumbles have left VAX-31 as the only “visible near-term” vaccine targeting more than 30 strains, Song wrote.
Yet there is some concern about how VAX-31 might fare. In early May, Vaxcyte shares fell by double digits when executives implied the shot might “miss” against some strains covered by Capvaxive, Mizuho’s Syed wrote in a note at the time. Syed argued that targeting 31 strains gives VAX-31 a “huge” buffer, and other vaccines that have come up short against specific strains have gone on to win approvals.
In a separate note, Leerink analyst David Risinger pointed out that, per a study protocol the FDA has already agreed to, a true “miss” on a particular strain would only occur if VAX-31 performs worse than both Prevnar 20 and Capvaxive. — Delilah Alvarado
Oral immune drugs known as TYK2 inhibitors have seen a resurgence of late. The first to get to market, Bristol Myers Squibb’s Sotyktu, hasn’t been the big seller the company had once hoped. But newer, optimized versions are showing they might be more effective — giving them the chance to fulfill the drug class’ lofty sales potential.
A notable test lay ahead. Later this year, Takeda could report results from a pair of Phase 2 studies testing its TYK2 drug zasocitinib in inflammatory bowel disease. Those trials are important for Takeda, which has stressed a focus on immunology and paid $4 billion to acquire zasocitinib. The drug has already come through in late-stage testing in psoriasis, and could be headed toward approvals in both that indication and psoriatic arthritis. Zasocitinib’s prospects would shoot even higher if it can succeed in IBD, where there is a “dearth of oral options,” R&D chief Andy Plump said on a conference call in May.
Multiple TYK2 drugs, including Sotyktu, have fallen short in testing in IBD. Zasocitinib is more selective than Sotyktu, though, suggesting dosing could be safely cranked up higher than with Bristol Myers’ medicine. On a conference call in January, Plump argued “higher exposures will be necessary for efficacy” in the two main forms of IBD, and that Takeda has “significant upwards headroom” with zasocitinib. Analysts at Leerink have suggested that study success could fuel optimism for the entire “next-gen” class of TYK2 drugs.
“The supporting rationale for efficacy in IBD is quite strong,” Plump said. “But of course, we’re going to need to see what those data look like.” — Ben Fidler
Novartis made one of the biggest acquisition bets in its history when it snapped up Avidity Biosciences for $12 billion last year. Centered around a trio of drugs for neuromuscular conditions, the deal was a calculated gamble Avidity had found an effective way to send RNA drugs into hard-to-reach muscle tissue, unlocking what Novartis views as multibillion-dollar sales opportunities in the years ahead.
In early June, Novartis reported that one drug in the deal appeared to be working as intended in a small study in a rare neuromuscular disorder called facioscapulohumeral muscular dystrophy. Those data “at least partially validate[s] Novartis' decision to acquire Avidity,” Jefferies analyst Michael Leuchten wrote in a client note at the time. But the company could gain much more reassurance if a late-stage study called “Harbor” produces positive results later this year.
That trial is evaluating an Avidity-discovered candidate known as del-desiran in people with myotonic dystrophy type 1, a progressive, muscle-wasting condition that affects an estimated 80,000 people in the U.S. Like Avidity’s other medicines, del-desiran is a conjugated medicine consisting of an RNA molecule fused to a targeting antibody. The drug is designed to get into muscle cells and lower levels of a faulty mutant messenger RNA that drives the condition. The hope is it might alter disease progression, rather than only manage symptoms as existing off-label treatments do.
Novartis is evaluating whether about a year of del-desiran treatment will lead to a statistical difference, compared to a placebo, on measures of muscle function such as how long it takes for a person to open their hands, or walk or run 10 meters.
A positive readout from the Harbor trial could keep Novartis ahead of a key competitor in Dyne Therapeutics, which has a rival RNA-based medicine in Phase 3 testing. Dyne is pursuing a different strategy than Novartis, as it intends to seek an “accelerated approval” if early data from that trial turn up positive next year. Novartis, by comparison, believes it can pressure the coming competition by using more substantive muscle function data to support a standard clearance, CEO Vas Narasimhan has said.
“Once that happens, we would make it harder for accelerated approvals to ultimately happen in the field as historical precedents have shown. So I think being first to market with a key, compelling profile is going to be very attractive,” Narasimhan said in an April earnings call.
Novartis previously projected that del-desiran could reach as much as $6 billion in peak annual sales if successful in testing. — Gwendolyn Wu
The race to make a better blood thinner is reaching its climax. Two readouts from partners Bristol Myers Squibb and Johnson & Johnson could go a long way toward deciding its outcome.
The drug the two companies are developing is known as milvexian. It’s one of multiple emerging medications targeting a clotting protein called Factor XI, and that have long been seen by proponents as a possibly safer alternative to existing therapies like Xarelto and Eliquis.
Study results for Factor XI inhibitors have been somewhat mixed so far, and milvexian failed a late-stage trial in acute coronary syndrome last November. But at the time, Bristol Myers and J&J were adamant that the drug still had “multibillion-dollar potential” and pointed to a pair of ongoing Phase 3 trials as evidence. Those two studies could read out results later this year.
Combined, the two trials have enrolled more than 30,000 participants and seek to prove milvexian can prevent strokes in people with irregular heartbeats or who’ve already had a stroke. In those with irregular heartbeats, known as atrial fibrillation, the experimental drug is being tested against Eliquis. Milvexian is being compared to a placebo in the stroke prevention study.
Eliquis was able to displace warfarin as the leading stroke prevention drug because it was more effective and recipients had fewer dangerous bleeding incidents. Eliquis was also more convenient, offering twice daily dosing that didn’t need to be individually tailored in the way warfarin did.
To succeed in atrial fibrillation, milvexian has to prove clinically similar, or non-inferior, to Eliquis at preventing strokes while lowering the risk of bleeding side effects. In an investor conference, Tom Cavanaugh, J&J’s group chairman for North American innovative medicine, said a 30% to 40% relative risk reduction would be “absolutely significant.”
In the secondary stroke prevention trial, Bristol Myers and J&J aim to show that a regimen involving milvexian and drugs like Brilinta or Effient is superior to the latter medicines alone at warding off additional strokes. They have reason to be optimistic, as a rival drug from Bayer reduced the relative risk of a recurrent stroke by 26% without a meaningful increase in bleeding risk in a different study.
Some Wall Street analysts have expressed confidence, too. In acute coronary syndrome, where milvexian stumbled, blood thinners aren’t commonly used. They are in stroke prevention, where outside trial investigators recommended last year that milvexian’s testing should continue. — Jonathan Gardner
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 36 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
