As 2026 begins, a slate of high-stakes clinical readouts—from a pivotal study of Novartis’ cardiovascular candidate pelacarsen to a Phase III test of Eli Lilly’s next-gen Alzheimer’s drug—are poised to reshape therapeutic landscapes.
2025 was marked by several successful clinical trial storylines, such as the battles to deliver the most effective oral GLP-1 drug and the highest weight loss overall—both won by Eli Lilly—and a few notable failures, with an entire class of therapies—the embattled TIGIT—called into question by poor trial results.
Looking ahead to the first half of 2026, numerous trial readouts will be closely watched by analysts, including one that Myles Minter, biotech equity research analyst at William Blair, called the “biggest biotech event of the entire year.”
This particular readout is for pelacarsen, an asset developed by Ionis Pharmaceuticals and licensed to Novartis, which is being explored for the treatment of Lp(a)-driven cardiovascular disease. Similar to other significant readouts expected in H1 2026, there are a lot of analyst eyes on this trial because of the potentially wider impact on biopharma beyond Novartis.
“There are a lot of Big Pharmas working here, and all are running massive outcome studies in either secondary prevention or primary prevention settings,” Minter said. “But we don’t yet know if lowering Lp(a) works at all, so this is the first study to flip that data card.”
In this piece, BioSpace looks at five clinical trials set to grab headlines in the first half of this new year, and their potential impact on the cardiovascular, Alzheimer’s and obesity markets and more.
Novartis’ Pelacarsen to Set The Stage
Novartis will publish the readout from the Phase III Lp(a)HORIZON study of pelacarsen in the first half of 2026. The readout was originally expected a year earlier, in the first half of 2025, but was pushed back due to fewer cardiovascular events occurring in the trial than expected, according to a January announcement by partner Ionis. Rather than an indication of the drug’s effectiveness, Minter suggested the lack of cardiovascular events was more likely due to patients being well-controlled and adherent to their existing treatments, such as statins.
Pelacarsen is an antisense medicine designed to lower Lp(a), an independent, genetically defined risk factor for cardiovascular disease. There is currently a significant interest from many companies to target this pathway because if Lp(a) levels can be reduced by 70%-80%, the hypothesis is that you could potentially prevent people from having a coronary event, Minter explained.
As a result, Amgen, Lilly, AstraZeneca, Merck and more are all targeting this key risk factor, making Novartis’ readout—the first at the Phase III stage—a crucial benchmark for all companies invested in the space, Minter said. By 2033, the market for Lp(a)-targeted therapies is projected to be worth over $8.5 billion.
Will Vertex Outrun Novartis With Povetacicept?
Vertex Pharmaceuticals is awaiting results from the Phase III RAINER trial of povetacicept, which was picked up in the $4.9 billion acquisition of Alpine Immune Sciences in April 2024, shortly after that company released Phase Ib/IIa data for the asset in patients with IgA nephropathy (IgAN).
Interim data from RAINER are expected in the first half of 2026. In addition, Vertex expects to complete the submission for a rolling biologics license application (BLA) for povetacicept in the same timeframe.
The results of the trial are particularly important for Vertex, with several of its newer products—including non-opioid pain medication Journavx and gene therapy Casgevy—struggling for sales. Should RAINER’s results be positive, Leerink Partners noted in an investor report that due to Vertex’s possession of a priority review voucher, povetacicept could be approved for IgAN by the end of 2026.
The resulting six-month review period may be useful to the company, as Novartis is also developing a BAFF-targeting drug, ianalumab, which could potentially be a rival in the broader autoimmune space. Both assets have product-in-a-pipeline potential, targeting immune thrombocytopenia, warm autoimmune hemolytic anemia and lupus nephritis in clinical trials.
Eli Lilly Positions Alzheimer’s Successor in Remternetug
The first half of 2026 is a busy one for Eli Lilly. Not only is the company gearing up for a March PDUFA date for the obesity medicine orforglipron and potential launch that could set the tone for pricing of oral GLP-1 therapies, the company is also expecting top-line Phase III data from its TRAILRUNNER-ALZ 1 study of remternetug in Alzheimer’s disease. Also anticipated in March, Suvannavejh said this is one of the most anticipated readouts in the space right now.
An anti-amyloid antibody positioned as the successor to Lilly’s Kisunla—approved by the FDA in 2024—remternetug is being trialed in more than 1,600 patients with early, symptomatic Alzheimer’s. The trial is assessing both subcutaneous and intravenous formulations of the drug, with the primary outcome measure focused on amyloid plaque clearance.
The subcutaneous format could offer advantages over intravenous delivery, including patient and caregiver preference, Dawn Brooks, Lilly’s global development leader for Alzheimer’s disease, told BioSpace in 2023. The company is also testing whether delivery via subcutaneous injections could alter the risk of developing amyloid-related imaging abnormalities.
Phase I data presented in April 2023 at the International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD 2023) showed that IV remternetug lowered beta-amyloid plaques after 85 days in all but the lowest dosing regimens, compared with placebo, in patients with mild to moderate dementia due to Alzheimer’s.
“Even in Phase I, we’re seeing very robust results across a range of doses,” Brooks told BioSpace at the time.
Ocular Challenges Big Pharma With Axpaxli
Ocular Therapeutix is set to report data next year from the Phase III SOL-1 trial of its treatment Axpaxli in wet age-related macular degeneration (wet AMD).
Axpaxli, a tyrosine kinase inhibitor (TKI) with anti-angiogenic properties, is dosed every six months, which would make it a “paradigm change in treatment,” for wet AMD, Graig Suvannavejh, managing director of Equity Research at Mizuho Securities, told BioSpace.
Ocular is in a race to market with EyePoint Pharmaceuticals, which is also developing a drug that can be dosed every six months. However, EyePoint’s data readout is expected to occur mid-way through 2026, giving Ocular a slight advantage, if its trial is successful.
Last month, Ocular said it would submit a new drug application (NDA) to the FDA shortly after the trial reads out in the first quarter of 2026.
Regardless, a positive result for both companies could immediately have a material impact, Suvannavejh stated. “They’re not partnered with any large pharmaceutical company, so . . . if the data across both programs end up being positive, either one or potentially both, will get acquired by someone else who has a presence in the space,” he predicted.
Regeneron, with partner Bayer, and Roche are the current market leaders, with Eylea and Vabysmo, respectively, both exceeding $1 billion in sales in the third quarter of 2025. In total, the wet AMD market is worth between $14 billion and $15 billion annually, Suvannavejh said, leaving plenty of dollars on the table for new players.
Wave’s WVE-007 Could Change Obesity Landscape
Generating considerable excitement among analysts last month were interim Phase I data from Wave Life Sciences’ weight loss therapy WVE-007—results that sent the company’s stock soaring approximately 80% in the immediate aftermath.
Wave’s data, announced Dec. 8, showed a 4.5% reduction in total body fat, a 9.2% reduction in visceral fat and a 0.9% increase in lean mass with the 240 mg dose—the lowest therapeutic dose—of WVE-007 after three months. The latter point could be crucial, as one of the noted drawbacks of existing GLP-1 obesity treatments is the potential for muscle loss.
Truist Securities analysts in a note at the time said the update “fundamentally changes the outlook for obesity landscape in a very disruptive manner and for the better.”
And Wave isn’t done yet. The company is gearing up for a catalyst-rich first half of 2026, with six-month data from the 240 mg single-dose cohort and three-month follow-up data from the 400 mg single-dose cohort expected in the first quarter. Then, in Q2, Wave plans to release results from the 400 mg cohort and three-month follow-up data from the 600 mg cohort.
WVE-007 represents a vastly different approach to rivals in the obesity space, with the investigational therapy delivered via a single RNAi injection to silence mRNA encoding the INHBE protein. People with a “protective” loss-of-function mutation in one copy of the INHBE gene have “healthier body composition,” and a better cardiometabolic profile, according to Wave.
This dose was observed to achieve fat loss on par with GLP-1s, Wave CEO Paul Bolno said in a statement. The company expects to be able to deliver the treatment once- to twice-yearly, as well as potentially using the drug as an add-on therapy to incretins, he added.
