Tag: United States

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Three things to watch amid Anthropic’s latest feud with the government

This story originally appeared in The Algorithm, our weekly newsletter on AI. To get stories like this in your inbox first, sign up here.

For those of you enjoying your summer unaware of Anthropic’s latest feud with the US government, here’s a recap: In April the company said it had built an AI model called Mythos that was so good at working with code it could pose a global cybersecurity threat. Anthropic gave access to a small group of cybersecurity experts so they could see what they were up against. Then it released a modified version called Fable which it said was safer to the public on Tuesday, June 9. That Friday, the federal government told the company it was a threat to national security and placed export controls on the new release. Anthropic revoked access to both models hours later.

People worried about catastrophic effects of AI—broadly labeled “doomers”—have said for years that the technology poses a threat to humanity and published proposals for how the government should intervene in its development. The doomers just got their government intervention—not over a bioweapon or rogue AI, but in response to an AI model that’s basically just really good at coding. And the result so far looks less like a safety plan than like a superficial reaction.

There’s plenty to dissect about what happened in those few days that led to such drastic action from the government, and it’s notable that Amazon CEO Andy Jassy was the one who told government officials that Fable would be dangerous (Amazon is both invested in Anthropic and building its own competing AI models). It’s also possible this will be a short-lived ban from the government that doesn’t survive legal scrutiny (it’s not clear that Anthropic’s offering access to Fable really counts as “exporting” it, for example). 

But there are ripple effects happening already. 

For one, this is making a whole lot of people not want to rely on American AI companies. TheFrench politician Bruno Retailleau described it as a “wake-up call” that should motivate Europe to build more AI. But any vision of turning Paris into Silicon Valley—touted by many other European leaders following the shutdown of Anthropic’s models—is complicated by one big thing: China. 

Open-source models from China are very capable and incredibly cheap, and they can be downloaded to run on anyone’s servers with no rules or guardrails. (This makes them attractive to companies that don’t want access turned off on the basis of a decision from the White House—but equally attractive to cybercriminals, the type that Anthropic hoped to fend off by building safety guardrails into its models.) 

It’s possible that companies, including those in the US and Europe, will decide that working with Chinese models is just easier, as the skyrocketing of shares in the Chinese startup Zhipu suggests. Playing this forward, is it possible the government’s next drastic decision will be to say that US companies using models from China pose a threat to national security? I wouldn’t write it off. 

Second, it’s possible that shutting off access to Anthropic’s models will leave the country morevulnerable to cybersecurity attacks, not less. Leading cybersecurity experts have said as much in an open letter to the government, writing that access to Anthropic’s models was helping researchers prepare defenses, and that the company’s models are no more dangerous than other leading models that are widely available. Such is the risk of applying the concept of nonproliferation to software—trying to control and restrict dangerous AI models in the manner of the uranium used for nuclear weapons. 

The third thing worth watching is how US lawmakers will react. Remember that following Anthropic’s last feud with the government over how the Pentagon could or could not use its models, a slate of new bills was introduced that would define the limits of military AI.

Right now, the biggest players shaping how AI gets used are the companies and the White House. There’s been much talk about more federal AI regulation, and polling suggests most Americans want it. Lawmakers are still figuring out whether to form rules on how kids use chatbots and are far from a clear answer on the extent to which the government should vet the safety of AI models. But with every drastic action from the White House, the pressure for regulations rises.

To state the obvious, predictions are hard when the administration’s attitudes toward AI  change with the wind. When President Trump took office, he threw out the restrictive rulebook for how to make AI safe and promised to get out of the way of tech companies. The White House has now called the most valuable AI startup a risk to national security once in the spring, and again in summer. What will fall bring?

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AbbVie to Acquire Apogee Therapeutics for $10.9B

SAN DIEGO — AbbVie has agreed to acquire Apogee Therapeutics for $10.9 billion, the companies said today, in a deal designed to bolster the buyer’s pipeline with an atopic dermatitis (AD) candidate set to advance to Phase III trials during the second half of this year, and being positioned as a potential challenger to a top-selling drug.

Apogee’s lead candidate zumilokibart, an IL-13 inhibitor also called APG777, is a long-acting treatment that according to the company holds “pipeline-in-a-product potential” because of the opportunity it has for treating a variety of immunology and inflammation (I&I) diseases for which the drug is under study.

“We continue to believe that Apogee’s zumilokibart is one of the more attractive assets in the I&I space, and its current valuation proves this out,” Edward Nash, a managing director and senior biotechnology analyst with Canaccord Genuity, wrote this morning in a research note. “The company, since its 2022 inception, has continued to deliver strong clinical results for zumilokibart in atopic dermatitis. The BIG [emphasis in original] differentiator for the drug is its potential to be dosed once every three or six months, which was just demonstrated in recently announced updates from the Phase II trial.”

Last month, Apogee announced positive 16-week data from Part B of its Phase II APEX trial (NCT06395948) assessing zumilokibart in moderate-to-severe AD. The trial met its primary and secondary endpoints with high statistical significance, as 65.9% of patients treated with mid-dose zumilokibart achieved EASI-75 (41.9% placebo adjusted).

Based on these results and subject to positive regulatory feedback, Apogee said it planned to move forward in its Phase III trials with the mid-dose, which achieved the best clinical activity of the three doses tested and was well-tolerated.

To support those Phase III trials and continued late phase development and potential commercialization of zumilokibart, Apogee last month entered into a strategic financing for up to $1.3 billion in flexible, non-dilutive total capital.

The capital includes up to $800 million of synthetic royalty funding and access of up to $500 million in senior corporate debt available by mutual consent of Blackstone and Apogee. Blackstone agreed to provide the synthetic royalty funding in exchange for low-to-mid single digit tiered royalties for 15 years on worldwide annual sales of zumilokibart. The royalties decrease with increasing sales, with zero royalties paid out on global annual sales exceeding $8 billion.

Third-largest deal, so far

The $10.9 billion Apogee acquisition is the new third largest biopharma merger-and-acquisition (M&A) deal announced so far this year, behind the €10.7 billion ($12.268 billion) cash buyout offer for Italian-based Recordati being pursued by CVC Capital Partners and Groupe Bruxelles Lambert, which aim to take the company private; and Sun Pharmaceutical Industries’ planned $11.75 billion purchase of Organon, the women’s health drug developer spun out of Merck & Co., in a deal expected to close in early 2027.

The previous third-largest M&A deal this year, now fourth-largest, is GlaxoSmithKline (GSK)’s planned $10.6 billion buyout of Nuvalent,  announced June 9 and expected to close in the third quarter.

For AbbVie, the deal for Apogee adds to its pipeline in I&I, a category the biopharma giant dominated when its multi-indication blockbuster Humira® (adalimumab) was the world’s best-selling drug, before it lost patent exclusivity in the European Union in 2018 and the U.S. in 2023—after which it slipped from the top of GEN’s annual A-Lists of Top 10 Best-Selling Drugs.

However, AbbVie has developed two successful I&I drugs in recent years, Skyrizi® (risankizumab)  and Rinvoq® (upadacitinib)—with Skyrizi ranking No. 6 on GEN’s latest best-selling drugs A-List, generating $17.562 billion in sales last year (up 49.9% from 2024) and $4.483 billion in Q1 2026, up 30.9% from Q1 2025.

“For more than two decades, AbbVie has led and shaped the field of immunology bringing the science, scale and expertise needed to address some of the most complex diseases,” Robert A. Michael, AbbVie’s chairman and CEO, said in a statement. “The acquisition of Apogee further builds on our existing leadership, strengthening our ability to deliver innovative medicines to patients who need better options while also creating significant long-term value for shareholders.”

Apogee investors signaled support for the buyout with a surge of stock buying that sent the company’s shares soaring 47% in early day trading from $90.38 to $132.65 as of 10:21 am ET. AbbVie shares rose 4.5% from $216.49 to $226.24.

Potential Dupixent® challenger

Apogee is positioning zumilokibart as a potential challenger to Dupixent® (dupilumab), the blockbuster drug for AD and other indications that is co-marketed by Sanofi, which records global net sales, and Regeneron Pharmaceuticals.

Dupixent ranked No. 5 among “Top 10 Best-Selling Drugs” as ranked by GEN in a recent A-List, with $18.124 billion (€15.714 billion) in 2025 sales, up 20.2% from the $15.077 billion (€13.072 billion) that the drug racked up in 2024. Dupixent carried that momentum into the first quarter of this year, garnering $4.9 billion (€4.2 billion) in sales as recorded by Sanofi, up 33% from a year earlier. 

However, Dupixent is set to lose key U.S. patent exclusivity in 2031, giving Apogee and other AD drug developers time, they hope, to bring new treatments to market that can successfully compete when Dupixent loses its IP protection.

In addition to AD, zumilokibart is also being developed to treat asthma and eosinophilic esophagitis (EoE). The EoE program is set to advance into mid-stage clinical study as Apogee plans to launch the Phase IIb ELEVATE trial in the second half of this year.

Apogee has generated positive Phase Ib data for zumilokibart in asthma, and is on course to advance that program into the Phase IIb ASPIRE trial, set to launch in the first half of 2027.

Two other programs, both of them combination therapies that include zumilokibart, round out Apogee’s pipeline. APG279, a combination of zumilokibart and APG990, an OX40L inhibitor, is an AD candidate now in a Phase I trial (NCT07027527) comparing the the safety, tolerability, and pharmacokinetic (PK) parameters of the combination vs. Dupixent in adults with moderate-to-severe atopic dermatitis (AD).

Apogee cites preclinical studies showing that APG279 has driven closer to JAK-like inhibition of Type 1, 2, and 3 signaling compared to approved or in-development biologics, with the potential for best-in-class dosing and better tolerability in AD and a variety of other I&I diseases.

One-two punch

Apogee reasons that its chances of treating AD are enhanced by a proverbial one-two punch combining deep and sustained inhibition of Type 2 inflammation through zumilokibart’s inhibition of IL-13 with broader inhibition of Type 1-3 inflammation through APG990’s inhibition of OX40L.

The other combination program, APG273, is a preclinical combination of zumilokibart with APG333, a TSLP (thymic stromal lymphopoietin) that is being developed to treat asthma and COPD. Apogee has said it plans to announce additional plans for clinical studies later this year.

“Apogee’s pipeline adds highly differentiated clinical-stage assets, further expanding our robust immunology portfolio in areas of significant patient need, including atopic dermatitis and asthma,” Michael added. With our deep scientific expertise and proven capabilities, we are uniquely positioned to rapidly advance these programs and continue to transform the standard of care in inflammatory diseases.”

AbbVie has agreed to acquire all outstanding shares of Apogee for $135.11 per share cash, a 49.5% premium from the stock’s closing price on Friday.

The boards of AbbVie and Apogee have unanimously approved the transaction, which is expected to close in the third quarter subject to customary closing conditions, including Apogee shareholder approval and receipt of regulatory approvals.

“This transaction reflects the strength of Apogee’s vision, our team’s dedication and the significant progress we’ve made advancing zumilokibart and our differentiated pipeline,” stated Apogee CEO Michael Henderson, MD. “Since our founding, we’ve focused on developing transformative therapies for patients with inflammatory diseases while creating value for shareholders. This transaction delivers substantial shareholder value and positions our programs to reach their full potential.”

“We believe AbbVie can advance zumilokibart and our portfolio while expanding their impact for patients worldwide,”  Henderson added.

The post AbbVie to Acquire Apogee Therapeutics for $10.9B appeared first on GEN – Genetic Engineering and Biotechnology News.

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STAT+: FDA to launch pilot program to speed up early-stage clinical trials

WASHINGTON — Federal health officials announced a pilot program Monday to speed up early-stage clinical trials, which they say will reduce development timelines by six to 12 months, in hopes of encouraging U.S.-based trials and combating Chinese dominance in the field.

The pilot comes as the Food and Drug Administration, through the president’s 2027 fiscal budget, asks Congress to establish a permanent, faster process for the existing Investigational New Drug pathway. That proposal was championed by former FDA Commissioner Marty Makary before he resigned last month, though officials said on a Monday morning call that this program had been in the works since the start of the administration.

In a Fox News op-ed, health secretary Robert F. Kennedy Jr. wrote that the U.S. is “losing ground” against China in clinical research and touted the actions as a way to reverse that trend.

Continue to STAT+ to read the full story…

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Prevalence and Associations of Medical Expenditure Panel Survey–Defined Long COVID Among Adults: Cross-Sectional Study

<strong>Background:</strong> Long COVID is a clinical condition that significantly influences quality of life, productivity, and morbidity in the individuals affected. Much of the research to date has examined medical comorbidities and their associations with long COVID, but there remains a substantial need to understand the social and behavioral factors associated with long COVID. <strong>Objective:</strong> The objective of this study was to investigate the prevalence and associations of Medical Expenditure Panel Survey (MEPS)–defined long COVID among adults in the United States through the application of the Andersen behavioral model. <strong>Methods:</strong> This cross-sectional database study used the 2022 MEPS dataset. Variables in this analysis were organized according to the Andersen behavioral model. The appropriate weighting variable was used to obtain weighted population-based estimates. Between-group differences (ie, those with MEPS-defined long COVID vs those without) were assessed using chi-square tests, and a multivariable binomial logistic regression model was developed to assess the association between each variable and having MEPS-defined long COVID. <strong>Results:</strong> A total of 11,266 individuals were eligible for inclusion in this study. This represented a weighted population of 256,500,584 American adults. Of these 11,266 individuals, 790 (7%; weighted population=18,397,214) had MEPS-defined long COVID, whereas 10,476 (93%; weighted population=238,103,371) did not. Variables identified that were statistically associated with having MEPS-defined long COVID among American adults included 3 predisposing variables (age, sex, and Asian race), 2 enabling variables (marital status and employment status), 3 need variables (number of chronic conditions, health status, and instrumental activity of daily living limitations), 1 personal health practices variable (ever receiving the COVID-19 vaccine), and 1 external environmental variable (south region). <strong>Conclusions:</strong> The prevalence and factors associated with having MEPS-defined long COVID among American adults in this study offer insights to expand our limited understanding of the complex environmental and social factors associated with MEPS-defined long COVID. Further research is required among the long COVID population to better understand and differentiate the causes and consequences of this condition.
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The Download: record-breaking subsea tunnels and flexible data centers

This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology.

Inside the world’s deepest and longest subsea road tunnel

—Niall Firth

I’m currently around 1,000 feet beneath the North Sea, in a dark, dank cave. It smells weird. And I’m increasingly aware of the pressure from millions of tons of seawater just above my head.

I’m under the iconic fjords of Norway to visit what will soon become the world’s longest and deepest subsea road tunnel—an exceptional engineering feat that will carry drivers deep beneath the North Sea.

I’m here to understand how you make a 16.6-mile highway that sits 1,280 feet below the sea at its deepest point. And also—at a time when it can feel hard to get anything done—to reassure myself that ambitious engineering is still possible. That we can still make things. 

Step inside Norway’s Rogfast tunnel and see how engineers are making it happen.

This story is from the next edition of our magazine, which is all about engineering. Subscribe now to get a copy when it lands on Wednesday!

Want to get a data center online quickly? Give it some flex.

The AI boom is putting unprecedented pressure on the electric grid. But rather than rushing to build new power plants, companies could find part of the solution right under our noses—or, more precisely, in the transmission lines under our feet and above our heads.

If data centers can limit the power they draw during high-demand stretches, they won’t need to wait for big infrastructure upgrades or build their own off-grid generation.

The idea of flexibility isn’t entirely foreign to grid operators. But a new generation of software could make the process faster, smarter, and more precise for the AI era.

Find out how the challenge of powering AI could lead to a smarter, more flexible grid.

—Amos Zeeberg

The must-reads

I’ve combed the internet to find you today’s most fun/important/scary/fascinating stories about technology.

1 SK Hynix has overtaken Samsung as South Korea’s most valuable company
It’s also now the world’s most valuable memory chipmaker. (Reuters $)
+  And one of the biggest beneficiaries of the global AI boom. (BBC)
+ AI’s need for memory chips is set to skyrocket device prices. (WSJ $)

2 Trump says he no longer views Anthropic as a national security threat
“Well, not now, but a week ago, maybe,” he told The Axios Show. (Axios)
+ He praised the response of Anthropic CEO Dario Amodei. (Reuters $)
+ Anthropic’s IPO outcome could depend on the midterms. (WSJ $)
+ A culture war tactic against Anthropic has backfired. (MIT Technology Review)

3 SpaceX has received the lowest possible ESG rating
Index provider MSCI gave the company a triple C. (Financial Times $)
+ Russia got the same score after invading Ukraine. (Business Times)
+ Elon Musk previously called ESG metrics the “Devil Incarnate.” (CNBC)

4 A Tesla on Autopilot allegedly crashed into a Texas home and killed a woman
The driver said his Tesla Model 3 was in self-driving mode. (NYT $)
+ Tesla’s AI trainers don’t trust its self-driving tech. (Reuters $)

5 Polymarket reportedly paid creators to post fake betting videos
Clips showed them winning big on bets they would have really lost. (WSJ $)
+ Polymarket bets on an Iran deal are fueling insider-trading fears. (Bloomberg $)

6 Physicists have proposed that black holes don’t exist
They may be something much stranger: “gravastars.” (404 Media)
+ This is the first ever photo of a black hole. (MIT Technology Review)

7 A daring space rescue mission is set to launch this week
A spacecraft will try to lift an observatory into a safer orbit. (Space)
+ We’re putting more stuff into space than ever. (MIT Technology Review)

8 Nothing’s next budget phone has been cancelled due to “RAMageddon”
The company said memory prices pushed costs too high. (The Verge $)
+ Buying a used phone makes more sense than ever. (Wired $)

9 A viral doomsday scenario aims to pierce Europe’s AI complacency
It envisions the US and China tearing Europe into pieces. (Guardian)

10 Scientists have invented a way to brew espresso with ultrasonic waves
No hot water required. (Wired $)

Quote of the day

“Even before we start reaping the benefits of AI in our devices, we are already paying the bill.” 

—Francisco Jeronimo, an analyst at IDC, tells CNBC that consumers are covering the costs of the ongoing memory shortage.

One More Thing

Bill Kirwa drives for Wasili, an Uber-style ridesharing company

BRIAN OTIENO


How mobile money supercharged Kenya’s sports betting addiction

As the lorry he’d flagged down lurched through Kenya’s western highlands, Bill Kirwa’s Infinix smartphone dinged with a notification. The bet of 3,500 shillings he’d placed with mobile money—then worth approximately $35—had just turned into nearly $8,500.

Kirwa, now 26, put the windfall to good use, purchasing a car that enabled him to drive for Wasili, an Uber-style ride-hailing service. But he continued gambling, and over time, his losses mounted. In just a few years, he’s effectively erased his big win.  

Kirwa’s experience is hardly unique. Across Africa, the rapid spread of smartphones and mobile money has fueled an explosion in online gambling. But nowhere is the craze as acute as it is in Kenya. Find out why.

—Jonathan W. Rosen

We can still have nice things

A place for comfort, fun, and distraction to brighten up your day. (Got any ideas? Drop me a line.)

+ A clever Bengal cat has seemingly learned to understand English—and talk back.
+ This list of the 100 greatest bird names lovingly captures the quirks of avian taxonomy.
+ Darth Vader’s weird chestplate transforms into a cassette player in these reworked Star Wars clips.
+ Trace the history and evolution of heavy metal music through the interactive genres and playlists of Map of Metal.

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Birth Characteristics Highlight Early-Onset Colorectal Cancer Risk

U.S. researchers have identified several life factors identifiable at birth that impact on a person’s risk of being diagnosed with colorectal cancer (CRC) at a younger age.

Being a man or having Hispanic ethnicity were potential risk factors for colorectal cancer before the age of 50 years, according to the study in Cancer. Among women, being heavier at birth or having an older father also increased this risk.

Conversely, men and women with a foreign-born mother had a significantly decreased risk of having colorectal cancer at this younger age.

“Evaluating demographic, birth, and parental characteristics is important in understanding what’s causing the rising incidence of early-onset colorectal cancer,” said lead author Sunny Siddique, PhD, MPH, from the Yale School of Public Health.

“Our findings warrant future studies aimed to understand the mechanisms through which factors such as male sex, Hispanic ethnicity, birthweight, maternal birthplace, and paternal age may influence risk of early onset colorectal cancer.”

Colorectal cancer is the second most common cancer in the U.S and, while rates have declined among people 50 years of age in the past few decades, they have steadily increased in younger people.

Compared with colorectal cancer that occurs in later years, early-onset disease presents at an advanced stage, both because of its aggressiveness and delays in diagnosis. It is typically found in the rectum and distal colon, a location associated with Western diets.

Siddique and team compared the characteristics of 1221 people diagnosed with colorectal cancer before age 50 with 61,050 matched individuals without cancer, all living in California.

Multivariable analysis revealed that men had a 34% higher risk of early-onset colorectal cancer compared with women. Hispanic ethnicity was also linked with a 43% higher risk compared with being White.

Among women, every half-kilo increase in birthweight was associated with a 10% increase in early-onset disease, while having a father aged 35 years or older was linked with a 56% increase in risk.

Overall, having a mother born outside the U.S. was associated with a 15% lower risk of early-onset colorectal cancer.

Siddique and co-workers note that more than 60% of foreign-born mothers in their study were born in Mexico and they point to other research suggesting that first-generation, foreign-born Hispanic women tend to have healthier diet and less obesity during pregnancy than their U.S.-born counterparts.

The team speculates that the paternal-age finding may relate to increases in rate of de novo mutations among children born to older fathers.

Referring to the raised Hispanic risk, the researchers note that this ethnic group continues to experience barriers to accessing screening and care, including language and cultural competency, income, and a lack of health insurance.

“Per recent recommendations by the U.S. Preventive Services Task Force to initiate CRC screening among average-risk individuals aged 45 years or older, it is important to note that people younger than age 45 years are not eligible for routine screening,” they pointed out.

“Yet, our finding of Hispanic ethnicity being a risk factor for CRC is particularly relevant to this group of young individuals who are experiencing a high incidence of CRC.”

The post Birth Characteristics Highlight Early-Onset Colorectal Cancer Risk appeared first on Inside Precision Medicine.

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QAIAx (AIhealth4U) – AI Public Health Central: Microcity-A (re Quantum AI Agency Aka AI City Hall Project, UPSTO App Nos. 64/074,526, 64/063,557, 63/903,181, 63/729,428

Conditions: Asperger’s Disorder; Asperger Disorder; Autism Disorder; Autism; ADHD – Attention Deficit Disorder With Hyperactivity; ADHD; ASD; Alcohol Abuse/Dependence; Alcohol Addiction; Alcohol and Other Drug Use Disorders; Alcohol and Other Substance Use Prevention; Gambling Addiction; Gambling Disorder; Sex Abuse; Sex Behavior; Sex Crimes; Sex Disorder; Sex Disorders; Gender Dysphoria, Adult; Eating Behavior Disorders; Narcotic-Related Disorders; Narcotic Addiction; Narcissism; Psychiatric Disorder; Psychedelic Effects in Healthy Volunteers; Psychedelic Experiences; Psychedelic Drug Dependence; Marijuana Use Disorder; Marijuana Abuse and Dependence; Smoking (Tobacco) Addiction; Smoking Among Youth; Smoking Abstinence; Abstinence, Sex; Opiate Substitution Treatment; Opioid Abuse (Disorder); Opioid Abuse and Addiction; Cocaine Abuse; MDMA (‘Ecstasy’); Addiction Disorders; Homeless and Low Incomes People, Refugees; Homelessness; Reliability and Validity; Anger Problems; Child Abuse, Sexual

Interventions: Behavioral: AI City Hall Project (AIhealth4u – Public Health Central); Behavioral: AI City Hall Project (QAIAx Microcity A – AI Public Health Central)

Sponsors: Veterans Recovery Network Inc.; U.S. Special Operations Command; Central Virginia VA Health Care System; AI-119 Vulcan Project Research & Educational Technology Company (fka Henry Nanpei Academy Project)

Not yet recruiting
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StockWatch: Positive Phase III Data Sells Investors on Intellia

A second positive Phase III data readout in two months is a major reason why Intellia Therapeutics (Nasdaq: NTLA) shares have soared more than 76% over the past six months—including a 29% surge this past week that followed the CRISPR gene editing therapy developer’s lead pipeline candidate lonvoguran ziclumeran (lonvo-z) meeting three key secondary endpoints in a pivotal study in patients with hereditary angioedema (HAE).

Lonvo-z met the Phase III HAELO trial’s (NCT06634420) primary endpoint, Intellia announced back in April, by showing an 87% reduction (p<0.0001) in mean monthly attacks in the lonvo-z arm vs. the placebo arm during the efficacy evaluation period (weeks 5-28). Lonvo-z also aced the trial’s key secondary endpoint by showing that 62% of the 52 patients in the lonvo-z arm were entirely attack-free and therapy-free for the six-month efficacy evaluation period, vs. just 11% of the 28 patients in the placebo arm (p<0.0001).

On June 13, Intellia presented and published additional data showing lonvo-z to have achieved positive results on three other key secondary endpoints:

  • Monthly rate of attacks requiring on-demand treatment, Weeks 5–28, mean (0.19 vs. 1.79, 95% CI).
  • Monthly rate of moderate/severe attacks, Weeks 5–28, mean (0.11 vs. 1.23, 95% CI).
  • Change from baseline to Week 28 in AE-QoL total score, mean ( — 23.51 vs. — 6.47, 95% CI).

Intellia presented the data at the European Academy of Allergy & Clinical Immunology (EAACI) Annual Congress 2026 in Istanbul, Türkiye, and published the results in The New England Journal of Medicine.

The data was strong enough for Intellia to continue the rolling Biologics License Application (BLA) submission that it began in April. The company expects to complete the BLA filing by year’s end and hopes to gain FDA approval and launch lonvo-z in the first half of 2027.

“Super pleased”

“We were super pleased with the results that we saw,” John Leonard, MD, Intellia’s president and CEO, told GEN. “We’re essentially replicating what we’ve seen throughout the program, where most of the patients reached a status of no attacks, no therapies over the course of this extended observation period. For those patients who didn’t, they appeared to be on their way to reaching that kind of a state. And critically important was that every single patient who got the drug was off long-term prophylaxis.”

“Across the board, and across all subgroups, the drug performed extremely well. So, we think it speaks to physicians, it’s going to speak to payers in terms of how they think about the drug and its ultimate, excellent utility,” Leonard said.

Investors and analysts appeared to share that enthusiasm this past week, as Intellia’s stock price rose over three of the four trading sessions following the release of data on the secondary endpoints. Intellia shares jumped 23% on June 15, the first trading day since the news, rising from $12.11 to $14.92. After a day of profit-taking that saw shares dip 2.5%, to $14.55, Intellia’s stock resumed its upward trajectory, rising nearly 4.5% to $15.20 on Wednesday, then another 3% Thursday, closing the week at $15.67. Markets were closed on Friday for the Juneteenth holiday.

Since December 18, 2025, when Intellia shares closed at $8.88, the stock has soared nearly 76.5%, accounting for most of its one-year gain of 62%. Lonvo-z accounted for three of Intellia’s four stock price peaks in 2026: The dosing of the first patient in HAELO, announced January 22, led the stock to climb 13%, from $14.03 to $15.90.

Shares surged 12% March 2, from $13.78 to $15.44, when the FDA lifted a clinical hold on the company’s Phase III MAGNITUDE trial (NCT06128629) assessing nexiguran ziclumeran (nex-z) in transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The FDA imposed the hold after an elderly patient died during a study of Nex-z, an in vivo CRISPR-based therapy developed in partnership with Regeneron Pharmaceuticals (Nasdaq: REGN) to treat ATTR-CM by inactivating the TTR gene.

The third peak, an 8% gain from $15.31 to $16.57 on April 22, followed Intellia reporting positive data for lonvo-z, showing that it met HAELO’s primary endpoint, while the fourth peak followed the secondary endpoint announcement.

Misperceived market

“Hereditary angioedema has, in the last 10 to 15 years, had a variety of therapies arrive that are better than the ones that were there 20 years ago. Twenty years ago, circumstances were pretty grim for patients with HAE,” Leonard recalled. “I think some investors have looked at this incorrectly as a satisfied market, only because there are other therapies available.”

Among those therapies are three that won FDA approval last year. Last August, the agency approved Dawnzera® (donidalorsen), a prekallikrein-directed antisense oligonucleotide designed to prevent HAE attacks in patients ages 12+, marketed by Ionis Pharmaceuticals (Nasdaq: IONS). A month earlier, the FDA authorized Ekterly® (sebetralstat), a plasma kallikrein inhibitor indicated for the treatment of acute attacks of HAE in patients ages 12+, marketed in the United States by KalVista Pharmaceuticals (Nasdaq: KALV).

And in June 2025, the FDA approved Andembry® (garadacimab-gxii), an activated Factor XII (FXIIa) inhibitor (monoclonal antibody) and the first long-term prophylactic HAE treatment designed to target Factor XIIa, administered as a once-monthly subcutaneous injection for patients ages 12+, marketed by CSL Behring, the largest business unit of Australian-owned CSL (ASX: CSL).

“What we’re showing is that a lot of efficacy and a lot of utility has been left on the table, and that it’s possible for patients to get pretty close to something resembling a normal person who does not have HAE and all of the things, benefits that come with that,” Leonard said. “As people have looked at the data more completely, I think they’re seeing more and more that that’s the case, and maybe some of those original premises that they had are not quite correct.”

In research notes, three analysts said Intellia’s latest data strengthened the company’s future case to regulators for pursuing approval of lonvo-z as a one-time HAE treatment.

“We view these data as furthering Intellia’s case for regulatory approval following its expected completion of a rolling BLA,” Myles R. Minter, PhD, a partner and biotechnology analyst with William Blair, wrote June 15.

A day earlier, Jefferies equity analyst Maury Raycroft, PhD, commented that Intellia’s latest data will help lonvo-z gain more than a foothold in the HAE market.

“Positive implications”

“Big picture, we believe total HAE data have positive implications for commercial positioning” of lonvo-z, Raycroft wrote. “Editing is expected to be durable (we have seen longer term ph.I/II data out to 3-yrs); therefore, NTLA’s approach could eliminate need for lifelong chronic tx [therapy], justifying the value proposition of a 1X tx, despite competition in a crowded HAE space.”

Raycroft cited market research from Intellia showing that 64% of surveyed patients on LTPs [long-term prophylaxis drugs] are extremely likely to transition to a one-time therapy, while 54% surveyed docs expressed intent to prescribe such a treatment.

Mani Foroohar, MD, senior managing director, genetic medicines, and a senior research analyst with Leerink Partners, said Intellia’s latest results “again demonstrate lonvo-z’s clean safety and best-in-class efficacy and convenience.”

Writing in NEJM, the team of HAELO investigators reported no serious adverse events in patients treated with lonvo-z: “The most common adverse reactions were infusion-related reactions, which were generally transient and resolved without intervention. Elevated levels of serum aspartate aminotransferase and alanine aminotransferase, which occurred in approximately 10 to 15% of patients treated with lonvo-z, were transient, asymptomatic, and resolved without intervention.”

Foroohar sided with optimistic investors over their pessimistic counterparts in arguing that patients will warm up to a one-time treatment, though it will likely be costlier than current therapies.

Bears and bulls

“Bears argue limited patient demand to move up the innovation curve in a market with several approved treatments. We take the other side of this and see onetime therapy (vs lifetime chronic dosing) and patient desire to be attack-free as potent tailwinds to adoption,” Foroohar wrote. “Longer follow-up and crossover data (caveat – small n [number of patients studied]) provide an early glimpse at the improving profile of lonvo-z over time. We look to more data ahead of 1H27 launch to further educate physicians/patients.

“Subgroup analyses demonstrate clear benefit across all patient populations (prior LTP use, historical attack severity/frequency, etc.), supporting broad uptake as SoC [standard of care] across HAE—recognizing this will take time to play out as physicians gain comfort with this (likely) first approved in vivo gene editing therapy,” Foroohar added.

Intellia has not set a price for lonvo-z.

“We have said publicly we’re not going to set any new records beyond prices that have been precedented,” Leonard said.

HAE patients, he continued, “are some of the most costly patients that payers have. They’re small in number, but high in cost, with the therapies they take and their healthcare resource utilization exceeding $1 million a year.

“When you consider that these are patients that are oftentimes treated, or first diagnosed in adolescence or young adulthood, the lifetime costs are frighteningly high,” Leonard explained. “We are confident that, and we have this as an intended outcome, that we will save lifetime health, resources in very, very substantial terms, in a way that payers see and can recognize. We want to make it easy for patients to get onto the therapy, and we want to make it very competitive, cost-competitive for physicians taking care of them.”

Leaders and laggards

  • Elicio Therapeutics (Nasdaq: ELTX) shares plunged 72.5% from $14.85 to $4.08 on June 15 after the developer of immunotherapies to treat high-prevalence cancers said it was evaluating multiple strategic financing and partnership opportunities to advance its planned Phase III adjuvant pancreatic cancer immunotherapies program and broader AMP platform. The action came after ELI-002 7P, a 7-peptide formulation of its lead candidate ELI-002, failed the Phase II AMPLIFY-7P trial (NCT05726864) in patients with mKRAS-driven pancreatic ductal adenocarcinoma (PDAC). ELI-002 7P missed the pre-specified primary endpoint of disease-free survival (DFS) in the intent-to-treat population. Elicio said the ELI-002 7P arm had a higher proportion of R1 resected (higher residual disease) patients vs. the observation arm (19% vs. 10%), and that post-hoc analyses showed significant DFS improvement (R0: HR 0.65, p=0.048) in the 121 lower residual disease (R0 completely resected) patients, a subgroup representing approximately 84% of enrolled patients. Elicio said the trial results will shape a Phase III strategy focused on a defined R0 resected population and additional ELI-002 7P dosing.
  • Neumora Therapeutics (Nasdaq: NMRA) shares plummeted 49% from $1.78 to 91 cents on June 15 after the brain disease drug developer said it was chopping its workforce by approximately 35% or about 34 jobs, ending development of its major depressive disorder (MDD) candidate navacaprant, and refocusing on advancing the rest of its pipeline. The actions came after navacaprant missed statistical significance on the primary and key secondary endpoints of the Phase III KOASTAL-2 trial (NCT06058013) and KOASTAL-3 trial (NCT06058039) in MDD. The primary endpoint was the change from baseline to week 6 on the Montgomery-Åsberg Depression Rating Scale (MADRS).  Neumora projected the job cuts would save it approximately $10 million annually, to be partially offset this year by approximately $2 million in one-time restructuring costs. Neumora said current cash and cash equivalents are expected to provide runway into Q3 2027, including multiple expected key clinical milestones. Neumora’s pipeline includes NMRA-511 in Alzheimer’s disease agitation, NMRA-898 in schizophrenia, and NMRA-215 in cardiometabolic disease.
  • uniQure (Nasdaq: QURE) shares zoomed 78% from $26.99 to $48.16 Wednesday after the gene therapy developer announced the FDA’s revised position that a three-year analysis from its two-trial, Phase I/II study (United States, NCT04120493, and Europe (NCT05243017) of AMT-130 in Huntington’s disease was now acceptable as the primary basis of a Biologics License Application (BLA) for accelerated approval of the gene therapy. Researchers hailed “game-changing” data last year showing significant slowing of Huntington’s disease (HD) progression, but the FDA disagreed while its Center for Biologics Evaluation and Research (CBER) was headed by Vinayak (Vinay) Prasad, MD, who resigned in April. uniQure said the FDA seeks to align on the confirmatory study design prior to the BLA submission, including considering allowing concurrent control on standard-of-care therapy instead of a sham procedure. “FDA communicated that they would work as expeditiously as possible with uniQure on this effort. The company is committed to conducting the confirmatory study without delay and expects to further align with the FDA on the details of such a study prior to BLA submission,” uniQure stated.

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