10 clinical trials to watch the rest of 2025

Eli Lilly and Novo Nordisk have dominant obesity drug businesses that will be hard for competitors to challenge, not just because of their established medicines Zepbound and Wegovy, but also because of the prospects they are preparing next.
For Lilly, the next drug in line is orforglipron. The therapy is among the most closely watched in all of pharma research, as it could be one of the first oral “incretin” medicines to reach market and, because it’s chemical-based, more easily scaled into mass production.

Initial Phase 3 results Lilly disclosed in April from a trial of people with diabetes — who tend to lose less weight in obesity trials than non-diabetics — were so striking the company’s market value swelled by about $100 billion. The evaluation period in that study was only 40 weeks, raising hopes orforglipron might have greater impact in a longer obesity study that’s expected to produce results later this year.
Any data Lilly reveals will be highly scrutinized. Incretin drugs are associated with gastrointestinal side effects that can cause people to stop therapy, making the safety of newer medicines particularly important.
Detailed diabetes study results that Lilly presented in June and published in The New England Journal of Medicine showed “persistence of both severe and frequent” GI side effects, as well as a plateauing weight loss effect, William Blair analyst Andy Hsieh wrote in a research note. “We are less bullish about the prospects of orforglipron” in obesity as a result, he added. — Jonathan Gardner

Link to trial

Sanofi struck gold with Dupixent. The drug, which Sanofi co-developed with Regeneron Pharmaceuticals, is now marketed for eight autoimmune conditions. It generated nearly $14 billion in sales last year, making it one of the industry’s top-selling products. Wall Street analysts are betting a successor drug the French pharma is developing might also have multibillion-dollar potential, if it can come through in studies that may yield results in the second half of this year.
Called amlitelimab, the therapy came to Sanofi via a 2021 acquisition. Amlitelimab works differently than Dupixent, as it’s designed to block a molecule called OX40L that amplifies T cell-driven inflammation in many inflammatory conditions. Sanofi believes it holds similarly broad potential, with studies underway in eczema, asthma, hidrandenitis suppurativa and multiple other diseases. Sanofi has estimated that, if approved, amlitelimab could generate more than 5 billion euros annually at its peak. Jefferies analysts are projecting more than $8 billion.

That confidence is tied heavily to amlitelimab’s prospects in eczema, where some analysts predict the drug will earn billions of dollars each year as a treatment for people who don’t respond to Dupixent. In a Phase 2 trial, the drug showed potential to generate deep responses, suggesting it could be positioned as a longer-lasting alternative.

Sanofi expects “both to grow very well all the way to the end of patent [protection] by taking up new patients and coexisting with different approaches,” CEO Paul Hudson said on an earnings call in April. Dupixent’s patents expire early next decade.

A similar drug Amgen developed by Amgen disappointed in a Phase 3 eczema trial, however, sparking doubts among investors that amlitelimab’s performance in late-stage testing might not match what was observed in Phase 2. Amlitelimab also missed its mark in a mid-stage trial in asthma, though Sanofi chose to move it into pivotal testing anyway.

Sanofi executives have noted its drug works slightly differently than Amgen’s. Data from late-stage studies was expected next year, but an early look could now come in the second half, as Sanofi has completed study enrollment more quickly than it anticipated.
— Ben Fidler

Link to trials Coast-1 and Shore

Hidradentitis suppurtiva is a chronic inflammatory skin condition that causes painful abscesses and tissue scarring. While estimates of disease prevalence vary widely, it’s historically been underdiagnosed and undertreated. Moonlake Immunotherapeutics, a Swiss biotech, could be in a position to help, if a pair of ongoing Phase 3 studies expected to read out in September succeed.
Moonlake’s antibody drug, sonelokimab, binds to two inflammatory cytokines as well as a protein, albumin, that are involved in hidradentitis suppurtiva. The way in which it attacks those targets, as well as its small molecular size, might help it treat the disease better than available biologics — AbbVie’s Humira, UCB’s Bimzelx and Novartis’ Cosentyx — with less frequent dosing. Data generated so far “suggest an optimal profile” compared to IL-17 inhibitors like Bimzelx and Cosentyx, “based on a compelling balance of strong efficacy and encouraging safety,” wrote Leerink Partners analyst Thomas Smith, in a January report.
Moonlake reportedly rejected a buyout offer from Merck this year, too, indicating pharma companies are watching its progress.

Sonelokimab didn’t demonstrate the kind of clear dose response in mid-stage testing that investors like to see, leading some to question whether its results will be replicated in Phase 3, RBC Capital Markets analyst Brian Abrahams wrote in June. Moonlake’s readout will also come as Cosentyx and Bimzelx, respectively approved in 2023 and 2024, gain greater adoption among patients and physicians. Executives hope to see a delta of at least 20% versus placebo in patients achieving a 75% reduction in disease symptoms — a result that would be “clinically meaningful and competitive” versus other therapies, according to Leerink analysts.

The findings could be worth billions. Bimzelx’s launch trajectory suggests it could be pacing toward blockbuster
status in the U.S. by the end of the year, Cantor Fitzgerald analyst Prakhar Agrawal wrote in May. Even if
sonelokimab looks like a “me-too Bimzelx,” it’s “likely to be a blockbuster drug,” Agrawal wrote. — Ben
Fidler

Link to trials Vela-1 and Vela-2

Roche’s fenebrutinib is one of a newer generation of BTK inhibitors that, unlike their cancer-fighting predecessors, are designed to reach into the brain — potentially making them useful to treat autoimmune diseases like multiple sclerosis. Though these drugs have underwhelmed in clinical testing so far, Wall Street analysts are still penciling them in as future blockbusters. A successful result in a trio of late-stage trials could support that outlook.

Fenebrutinib is the third drug of its kind to near a Phase 3 readout in the “relapsing” form of multiple sclerosis, which is characterized by periodic disease flares. The first two, Sanofi’s tolebrutinib and Merck KGaA’s evobrutinib, failed trials testing them against another MS medicine called Aubagio. (Tolebrutinib did succeed against a rarer type of MS, however.)

Development of each drug has been slowed by signs of potential liver damage in testing, suggesting a risk that could be tied to all BTK blockers. These factors, along with the availability of effective treatments for relapsing MS, have fueled doubts about Roche’s chances with fenebrutinib.

Still, Roche is optimistic. Executives have previously noted how fenebrutinib is different from others in its class because its target binding is “reversible.” Preclinical testing showed fenebrutinib is “more potent and more selective” than others, Roche’s pharma chief Teresa Graham said on a conference call in April. That’s helped Roche bring to Phase 3 what it believes is the “most appropriate dose,” she said.
“We do believe that we just frankly have a better BTK,” Graham said.
Those words will be put to the test shortly. Two trials called FENhance 1 and FENhance 2 are evaluating fenebrutinib in relapsing MS, while FENtrepid is testing the drug in the primary progressive form of the disease. Roche has said to expect initial results by the end of the year.  “[I]t will be interesting to see if greater selectivity or reversibility yields better results,” wrote Leerink Partners analysts, in a May report. — Ben Fidler

Link to trials FENhance 1, FENhance 2 and FENtrepid

RNA editing is a budding corner of genetic medicine. About a dozen small and large companies are pursuing the technology, believing it may prove safer and more flexible than DNA editing. An important proof point could come later this year, when Wave Life Sciences reports new results from a study testing an RNA editing therapy in the rare disease alpha-1 antitrypsin deficiency.

Wave revealed promising initial results from that trial last year. Two people with alpha-1 antitrypsin deficiency, or AATD, who were treated with a single dose produced significant amounts of a protein their bodies normally can’t make. The effects took hold within days, lasted through about two months of follow-up and weren’t accompanied by any serious side effects. The findings surpassed investor expectations and catapulted shares of Wave as well as some of its peers.

The next update from that study will be more definitive. In the third quarter, Wave will report results from people who’ve received more than one dose of its therapy, the first data of its kind in humans. Wave believes those results will be “highly informative” for the drug’s path forward, as well as the potential of others in its portfolio, CEO Paul Bolno said on a conference call in May.

Wall Street analysts are paying close attention. It’s unclear whether repeat dosing of an RNA editing therapy will boost effectiveness or come with safety tradeoffs. Wave is also trying to stay ahead of its rivals, many of which are also developing drugs for AATD. One, Korro Bio, will report early study results in 2025.

Wave’s findings will have implications for “Korro and [other] peers in the RNA editing space,” William Blair analyst Myles Minter wrote in May. — Ben Fidler

Link to trial

PCSK9 inhibitors were once seen as the next big thing in heart medicine. Named after the protein they target, these injectable drugs can dramatically lower cholesterol in people with heart disease. However, clinical success didn’t lead to the lofty sales analysts first predicted and their developers struggled for years to convince payers and physicians of the drugs’ worth.
Amgen has had the most success. Its drug Repatha is used as a “secondary prevention” therapy for people who’ve already had heart attacks or strokes and are at high risk of experiencing another. After a slow start, sales surpassed $1 billion in annual sales in 2021 — seven years after Repatha’s initial approval — and reached $2.2 billion last year. “The barriers that once were there in obtaining Repatha have largely dissipated,” said Amgen commercial chief Murdo Gordon, on a conference call in May.
Some Wall Street analysts think Repatha’s growth will accelerate if a study called Vesalius-CV reads out positively in the second half of this year. That study is a “primary prevention” trial in which Repatha is used as the first line of defense against potential heart attacks, strokes or other cardiovascular events. Although some physicians already prescribe Repatha off-label for this use, study success could “drive long-term Repatha growth” until the drug’s patents expire at the end of this decade, Leerink Partners analyst David Risinger wrote in June.

“The addressable market remains vast, with tens of millions of patients globally still not reaching [cholesterol] goals despite available therapies,” Risinger wrote, and “current penetration is very low.”
Many branded and generic cholesterol drugs are available and multiple newer medicines are in advanced testing. A PCSK9 pill from Merck & Co. recently succeeded in a pair of late-stage trials, too. Yet Risinger thinks Merck’s drug will grow the PCSK9 market rather than change Repatha’s trajectory, in part because uptake is still limited. “There is room for competition,” Amgen’s Gordon said in May. — Ben Fidler 

Link to trial

Lyme disease, a bacterial infection spread to humans by tick bites, has become more common, with some estimates suggesting the disease affects about a half a million people annually in the U.S. A vaccine developed by French biotech Valneva and partner Pfizer could help change that trajectory, if successful in a Phase 3 trial due to read outs results in the second half of this year.
Lyme disease can be treated with antibiotics if caught early. But if it isn’t, people can suffer more long-term health complications, such as joint pain, heart inflammation and neuropathy. Some given antibiotics may experience monthslong “post-treatment Lyme disease syndrome,” which causes brain fog and fatigue.

There are no preventive options for Lyme disease, as the only approved vaccine, GSK’s Lymerix, was withdrawn in 2002. Pharma companies have largely shied away from investing ever since, leaving Valneva’s VLA15 as the only vaccine in late-stage clinical development. The company began working with Pfizer in 2020 and brought VLA15 into a Phase 3 trial two years later.

That trial has faced some roadblocks. The two companies pushed back the timing of their expected approval application from 2025 to 2026 after uncovering trial violations by a study site operator that forced them to remove almost half of the participants they initially enrolled. Since then, the trial, which was originally expected to involve 6,000 volunteers, has enrolled double that total, according to a federal database.

The coming results are important for Valneva. The company’s shares have plummeted since struggling to bring forth a COVID-19 shot. The launch of a vaccine for chikungunya virus infections, meanwhile, sputtered amid safety concerns that caused regulators in the U.S. and Europe to limit use.

A positive study result and approval would open up a market opportunity estimated to be worth more than $1 billion annually, Jefferies analyst Maury Raycroft wrote in a June report. Valneva would also be eligible for up to $143 million in future payouts and sales royalties ranging from 14% to 22%, Raycroft noted. —
 Delilah Alvarado

Link to trial

Since selling its migraine drug business for nearly $12 billion, Biohaven has been searching for its next big franchise. As part of that hunt, the company made a $100 million bet on the science coming out of a Pittsburgh-based biotech firm. The next key test of that science is a major depression study set to produce results toward the end of this year.
The study is evaluating a medicine designed to block “ion channels” — tube-shaped proteins that, through the rush of charged particles, allow cells to communicate with each other. This medicine, now known as BHV-7000, specifically targets two kinds of potassium ion channels that are located in the nervous system and regulate neuron activity and excitability. Biohaven had previously evaluated BHV-7000 in patients with bipolar disorder, but that trial did not succeed.

The company acquired the drug through a deal with Knopp Biosciences. While ion channel research is famously difficult, recent breakthroughs and promising data readouts had, by 2022, convinced the Biohaven team the time was right to “pounce” on this area of development, according to CEO Vlad Coric. The company is now investigating whether BHV-7000 can be useful against major depression and two kinds of epilepsy. It also has another drug targeting a different type of ion channel, potentially for the treatment of migraines.
“Ion channels really have the potential for us to change the way we treat neuropsychiatric and neurological disorders,” Coric said last year. It’s a research field “ripe for moving quickly.”

Success there could help Biohaven rebound after a series of recent pipeline setbacks, as well as reverse a stock slide that’s seen the company lose more than half of its share value this year. — Jacob Bell

Link to trial

Editor’s note: Moderna read out results from this trial in June.
Moderna rocketed to biotech stardom during the COVID-19 pandemic. One of the first companies to successfully develop a COVID shot in record time, Moderna’s market capitalization surged past $100 billion in 2021. But slowing vaccine sales and a dramatic shift in the regulatory landscape have caused Moderna to lose over 90% of its share value since, raising questions about its future.
A combination shot Moderna is developing for flu and COVID is an important part of its plans. Dubbed mRNA-1083, it’s the furthest along in clinical development of any shot like it. That vaccine is “a key pillar of the bull thesis for [Moderna] and path to profitability later this decade,” Leerink Partners analyst Mani Foroohar wrote in a note to clients earlier this month.

Yet the approval path for mRNA-1083 is longer than Moderna originally anticipated. New FDA leaders Martin Makary and Vinay Prasad have set stricter vaccine standards. As a result, Moderna, which had already claimed success in a late-stage, can’t get regulatory clearance for mRNA-1083 without positive Phase 3 results for an influenza vaccine, mRNA-1010, that contains the same flu component as the combo. The company pulled its combo application in May, heightening the importance of the flu readout expected this summer.

At an investor conference in June, Moderna president Stephen Hoge noted the company intends to file for approval of both the standalone flu shot, as well as the combination vaccine, if the mRNA-1010 study is successful. “We think the bigger market is going to be for the combo,” Hoge said, but “we want both options available.”
Some Wall Street analysts aren’t sold on Moderna’s optimism. Foroohar, for instance, is skeptical of the “commercial viability” of mRNA-1083 given the FDA’s newer COVID vaccine guidelines and uncertainty about which framework a “combo vaccine would adhere to.” — Delilah Alvarado

Link to trial

Editor’s note: This entry was updated June 29, 2025. The trial readout is still pending.
Diabetes is a disease of both the cardiovascular and metabolic systems, doubling the risk of heart problems and making people vulnerable to other poor health outcomes. As a result, regulators want to ensure new diabetes medicines don’t compound risks to cardiovascular health. Increasingly, they’re also looking for proof drugs can be protective, too.
Developers of medicines known as “incretins” have been putting their therapies to such a test. Already, Novo showed its weight loss drug Wegovy can prevent an array of heart issues, evidence it has used to unlock broader insurance coverage. Next in line is Eli Lilly, which could have results from a cardiovascular outcomes trial of its own this year.

Lilly’s trial has enrolled more than 13,000 people with diabetes and is evaluating whether tirzepatide, the incretin drug it sells as Mounjaro for diabetes and Zepbound for obesity, can help heart health. Unlike other, similar studies that tested a medicine against a placebo, Lilly is pitting tirzepatide against another diabetes drug it sells, Trulicity, which itself is effective at aiding cardiovascular health. Results are expected by the third quarter.
While the findings will provide another data point to compare Lilly’s incretin therapies to Novo’s, they could also signal how well Zepbound may fare in another trial of people with obesity. That study is measuring whether Zepbound can prevent a broader group of heart-related complications as well as death from any cause, and is expected to produce data in 2027. — Jonathan Gardner
Link to trial

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 next year. — Ned Pagliarulo

Link to trial

Editor’s note: This entry was updated June 29, 2025. The trial readout is still pending.

Entyvio, an injectable drug for inflammatory bowel disease, has become one of Takeda’s top-selling medicines since its approval more than 10 years ago. Eli Lilly hopes a recent acquisition will yield a more convenient alternative. A trial called Emerald-2 could indicate whether the money was well spent.
Last March, Lilly paid $3.2 billion for Morphic Therapeutic, giving the company an experimental medicine called MORF-057. Like Entyvio, Morphic’s drug targets a specific type of “integrin” protein that triggers inflammation in the intestine. But MORF-057 is a pill; Entyvio is administered via an intravenous infusion or under-the-skin shot.
An earlier, open-label study completed by Morphic suggested MORF-057 could be roughly as effective as Entyvio. More definitive evidence, though, will come from placebo-controlled mid-stage trials, the furthest along of which, Emerald-2, is in ulcerative colitis.

Positive results could help validate Lilly’s bet on Morphic and put the company on track to compete with oral IBD medicines from Pfizer and Bristol Myers Squibb.

The program’s progress could also contribute to a shift in treatment for immune conditions, which have historically been treated by injectable medicines. “[T]he move towards oral options is becoming increasingly evident,” Stifel analysts Alex Thompson and Paul Matteis wrote in a report initiating coverage of Morphic two years ago. They estimated MORF-057, if eventually approved, could generate more than $2 billion in yearly peak sales. — Gwendolyn Wu

Link to trial

Editor’s note: This entry was updated June 29, 2025. The trial readout is still pending.

Incretin drugs have been so successful in clinical testing they’re akin to a pharmaceutical Swiss Army knife. Already, medicines from Novo Nordisk and Eli Lilly approved to treat obesity and diabetes have proven beneficial for heart failure, sleep apnea and kidney disease.

A trial expected to produce results later this year could determine whether incretins might even play a role treating Alzheimer’s disease.
Known as Evoke, the study has been testing an oral version of semaglutide, the active ingredient in Novo’s Ozempic and Wegovy, in more than 1,800 people with early Alzheimer’s over two years. Novo aims to show semaglutide can help slow the cognitive decline associated with the condition.
A company spokesperson told BioPharma Dive results should be available sometime in 2025. A federal database lists the trial’s primary completion as September.

If results are positive, semaglutide could force researchers to consider how targeting gut hormones affect the brain and, possibly, change Alzheimer’s care.

The only treatments shown in testing to alter Alzheimer’s course, Biogen’s Leqembi and Eli Lilly’s Kisunla, have only modest effects on cognition. They have so far been used sparingly because of side effects that include brain swelling.
As a daily pill patients could take at home, oral semaglutide would be more convenient than Leqembi and Kisunla, which require periodic trips to healthcare facilities for lengthy infusions. — Jonathan Gardner

Link to trial

Editor’s note: Vertex said in May that it temporarily paused a portion of this study to investigate a “tolerability” issue. This entry was updated accordingly on June 29, 2025.

Vertex changed the treatment of cystic fibrosis. Since 2012, the company has brought five drugs to market that can help about 90% of people with the lung disease. Those drugs generated nearly $10 billion in sales in 2023 and have turned Vertex into one of the industry’s most valuable companies.
Yet Vertex is still working to cement its CF dominance. One way it’s trying is through a partnership with messenger RNA specialist Moderna.

Vertex and Moderna have been working since 2016 to develop an mRNA-based therapy for CF that’s sprayed into the lungs. That goal has proven elusive for RNA medicines, as the various defenses the body uses to keep drugs out of the lungs — or remove them if they get there — make inhalable therapies difficult. Several drugmakers have struggled in the past. It took Vertex and Moderna six years and two collaborations to bring their first prospect, mRNA-522, into clinical testing. They’ve cited delivery challenges as a main reason why.
Vertex says mRNA-522 could be an option for the estimated 5,000 people globally who can’t take its current medicines because their bodies don’t make a key protein, CFTR, that prevents sticky mucus from accumulating in the lungs. MRNA-522 is supposed to work by sending an mRNA blueprint for the protein directly to lung cells, which then make CFTR.

Initial safety and efficacy results from a small, early-stage study were expected in the first half of the year, before Vertex moved to pause the program. They were meant to include results from a single-dose portion of the trial, as well as findings in people who’ve received more than one dose. — Ben Fidler

Link to trial

Editor’s note: Arvinas and Pfizer read out results rom this study in March, and presented full data in June.

Targeted protein degradation, a way of coercing cells into junking troublesome proteins, has taken off in the last decade. Proponents view the approach as a way to hit difficult drug targets, opening up proteins thought to be “undruggable.” Proof of protein degradation’s value could come this year, when one of the field’s leaders, Arvinas, reports results from a Phase 3 trial.

Arvinas was founded in 2013 by Craig Crews, a Yale University scientist and key contributor to targeted protein degradation research. The company went public five years later and has since formed multiple partnerships. That progress has put Arvinas at the front of the industry’s chase of protein-degrading medicines.

Arvinas sees its lead drug, vepdegestrant, as a potential backbone therapy for a common form of breast cancer. It’s an oral medicine designed to degrade estrogen receptor proteins more potently than fulvestrant, which is administered via injection and widely prescribed for breast cancer patients whose disease is driven by hormones. A pair of Phase 3 trials are currently underway and two more are planned. The first, set to read out in the first quarter, tests vepdegestrant against fulvestrant in advanced, second-line breast cancer.
Arvinas faces competition as well as investor skepticism, though. One oral estrogen receptor degrader was approved in 2021, but only for tumors with so-called ESR1 mutations. Another from Eli Lilly could follow. Their success raises the bar for Arvinas, making the drug’s “competitive differentiation and commercial relevance outweigh a statistical data win,” Stifel analyst Bradley Canino wrote in October. — Ben Fidler

Link to trial

Editor’s note: Vera Therapeutics disclosed results
from this trial in June.

Immune drug research has attracted significant attention from drugmakers and investors lately, as available therapies either bluntly suppress the body’s defenses or leave room for more effective or convenient options. One example is a surge in drug development activity around an autoimmune condition called IgA nephropathy, or IgAN, for which a coming study readout from biotech Vera Therapeutics may have important implications.

Some estimates hold 130,000 to 150,000 people in the U.S. have IgAN, a progressive disease that damages the kidneys. Though several treatments are approved, they don’t wholly stop deterioration of patients’ kidney function. That need, combined with a better understanding of the disease’s biology, has led to a handful of acquisitions involving IgAN drug developers. Drugs launched by Travere Therapeutics and Calliditas Therapeutics have had fast starts, too, which “bodes well” for therapies with “superior value propositions,” Raymond James analyst Ryan Deschner wrote in November.

One of those therapies is Vera’s atacicept. Like several others now in testing, atacicept targets a pair of cytokines called BAFF and April that stimulate production of damaging autoantibodies. In a Phase 2 trial, the drug stabilized a key marker of disease progression so well after two years that results appeared “indistinguishable from [those] of patients with healthy kidneys,” added Deschner, who believes the company is setting a “high bar” for other competitors.

That view will be put to the test in the second quarter, when Vera expects to reveal topline results from a Phase 3 study called Origin 3. The findings could support an approval application and will impact drugmakers like Biogen and Vertex, the latter of which bought Alpine Immune Sciences in 2024 for a drug that also targets BAFF and April. — Ben Fidler

Link to trial

Editor’s note: Beam Therapeutics read out results from this trial in March.

Beam Therapeutics has been in the headlines for an experimental sickle cell disease therapy that could offer lasting benefit via a single infusion. It’s similar in concept to Casgevy, an approved medicine from Vertex Pharmaceuticals, but uses a different form of DNA modification known as base editing to accomplish its goal. Beam thinks it may work more quickly and efficiently than Casgevy.

But even more important to Beam may be another medicine that’s not quite as far along in testing. Unlike its sickle cell therapy, this medicine works by editing genes directly in the body, rather than being applied to a patient’s cells in a laboratory. It’s designed to correct a mutation that causes severe alpha-1 antitrypsin deficiency, a genetic disease that damages the lungs and liver.

Alpha-1 antitrypsin deficiency has become a top target for drugmakers, which are attempting to treat it with a range of different drug types. Base editing holds potential to deliver powerful, life-long benefits.

But Beam needs to prove it can work. It will offer initial clinical data from several study cohorts in the first half of 2025 that will give at least some indication of its medicine’s efficacy and, most importantly at this stage, safety. — Ned Pagliarulo
Link to trial

Editor’s note: Compass Pathways read out results from this trial in June.
It’s been a decades-long mission by some researchers to strip the tie-dye veneer from the field of psychedelics and show these drugs can be legitimate treatments for various diseases.
Yet, the first major swing at getting the Food and Drug Administration to approve a psychedelic ended in failure. Last August, the agency rejected a request from one of the field’s most talked-about companies, which had hoped to get MDMA — also known as the party drug ecstasy — cleared as a therapy aid for people with post-traumatic stress disorder.

That rejection could put more pressure on other developers, like U.K.-based Compass Pathways, to succeed with their programs. Compass has been testing a synthetic form of psilocybin, a compound found in mushrooms, against a range of mood and behavioral disorders. The drug’s biggest proving point so far is a late-stage study in treatment-resistant depression.

Compass had previously expected data toward the end of 2024. But enrollment challenges caused delays, which then led the company to lay off 30% of its workforce.

In a December note to clients, TD Cowen analyst Ritu Bural wrote that while the delayed timeline is disappointing, Compass’ choices were “highly rational to maintain study integrity and adequately address key FDA considerations on psychedelic trial design/conduct.” Bural added that her team still sees the trial as having a “high likelihood of success” since its design mirrors an earlier, successful mid-stage study. — Jacob Bell

Link to trial

Editor’s note: Vertex Therapeutics read out results from this trial in April.
Ambition is nothing new among biotech companies, which regularly promise new and better human medicines will emerge from experiments they’ve run in petri dishes and mice. But even so, Verve Therapeutics has set a bold target to prevent heart attacks for life with a single drug infusion.

Verve’s aspirations are rooted in a precise form of CRISPR gene editing that allows it to switch off genes associated with high cholesterol. Its first clinical trial attempt showed “proof of principle,” establishing that base editing a gene called PCSK9 could sharply lower cholesterol in people who have genetically high levels.
But concerning laboratory test results in one study participant, while resolved, led Verve to pivot to a back-up version of its therapy, which uses a different particle to deliver the same editing machinery to the right cells in the body. Initial data from a new study of this successor drug are due in the first half of 2025.

When they come, the results will inform Verve’s strategy for targeting the PCSK9 gene moving forward. They could also trigger Eli Lilly to decide whether it wants to use an option it holds to license the therapy under a partnership the two companies struck in 2023.

More broadly, the data are likely to shift investor sentiment around the therapeutic promise of using base editing to treat heart disease. — Ned Pagliarulo

Link to trial