Tag: United States

Posted on

Bespoke ASOs Cut Seizures, Boost Skills in Rare Epilepsy Syndrome

Two children with an ultra-rare epilepsy linked syndrome called SCN2A-developmental and epileptic encephalopathy (DEE) have been treated with personalized antisense oligonucleotide (ASO) therapies for their specific genetic mutations.

Both children, 9 and 14-year old boys, experienced significant improvements from the therapy. They both had less seizures and could cut back or stop some of the anti-seizure medications. They also achieved improvements in language and motor skills, with the older patient walking independently for the first time after being treated.

SCN2A‑DEE is a severe early‑onset epilepsy syndrome caused by pathogenic variants in the SCN2A gene, which encodes a voltage‑gated sodium channel in excitatory neurons. These variants typically lead to DEE with difficult‑to‑control seizures and profound neurodevelopmental impairment and impact around 16,000 people in the U.S.

Like many rare diseases, children with SCN2A‑DEE often have different mutations, albeit in the same gene. “The therapy is deliberately designed to target the individual’s genetic diagnosis,” said principal investigator Olivia Kim‑McManus, MD, associate professor of neurosciences at UC San Diego School of Medicine and director of the Rady Precision Therapeutics Neuro-Interventional Program at Rady Children’s Hospital San Diego, in a press statement. “The ASO modifies genetic expression and what proteins are expressed.”

This study, which is published in Nature Medicine, involved investigation, design, and treatment of the two boys in parallel. To begin with, the researchers designed allele‑selective ASOs targeting heterozygous intronic SNPs in SCN2A to suppress the mutant transcript while sparing the wild-type copy.

They then ran two parallel first‑in‑human N-of-1 trials for the two boys with individualized dosing. The primary endpoints were seizure frequency and neurodevelopment, plus phenotype‑tailored measures like impact on autism-like symptoms, movement problems, and gastrointestinal function.

Notably, the team scanned 19 infants with related SCN2A disorders and found that three of them could be eligible for treatment with the same ASO as the one given to the 14 year old boy in the future, as it is designed to treat a number of different mutations on the SCN2A gene.

Both boys had fewer seizures on the customized drug. The younger child had an estimated 26% reduction and could come off phenytoin treatment without losing overall control, while the older child’s seizures fell by about 90%, with longer seizure‑free stretches and less need for rescue medication.

Families and clinicians also saw improvements in motor function, abnormal movements, communication and gastrointestinal symptoms, and no serious safety signals emerged from the study.

“We’ve seen changes across the board, showing that targeting the root genetic cause can produce measurable improvement,” said Kim‑McManus.

One key improvement was that the older child began to walk on his own for the first time. “Since then, he’s been walking independently,” said Kim-McManus. “When we really think about precision therapy in a personalized way, you can’t get more personalized than that.”

The researchers are now hoping to use a similar approach to treat more children with SCN2A-related disorders as well as other monogenic conditions.

The post Bespoke ASOs Cut Seizures, Boost Skills in Rare Epilepsy Syndrome appeared first on Inside Precision Medicine.

Posted on

Opinion: Science and the public are learning the wrong lessons from Covid

Covid fatigue is real, but policymakers and policy influencers need to wake up to something important: Post-pandemic polarization threatens to lock our memories into attractive, untrue conclusions that will leave us unprotected next time.

When Covid-19 first arrived, there was little historical experience to guide response. After watching catastrophic results and stress on the health care systems in China, Italy, and Iran, American states rapidly invoked many measures simultaneously to combat a virus with proven destructive power: masks, social distancing, and school and business closures. They turned to the 1918 flu pandemic to understand the importance of rapid and society-wide response.

Read the rest…

Posted on

STAT+: Makers of rare disease drugs seek exemption from Trump’s price-cut pilots

WASHINGTON — Biotech companies are lobbying the Trump administration to exclude treatments for rare diseases from programs that lower brand drug prices in Medicare.

The Rare Disease Company Coalition met last week with the White House Office of Management and Budget to discuss two pilot programs that are part of President Trump’s plan to get drugmakers to lower prices in the United States to levels charged in other rich countries, a policy generally referred to as most-favored nation.

Continue to STAT+ to read the full story…

Posted on

China’s AI models have Trump’s AI world at war with itself

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

Over the weekend, several current and former advisors to President Donald Trump on AI publicly lobbed insults at the country’s leading AI companies. David Sacks, the president’s AI and crypto “czar” until March, branded Anthropic’s models as “lobotomized” and “woke.” Emil Michael, a top Pentagon official, called OpenAI’s new head of strategic futures a “supreme village idiot.”

It began because no one can agree on what to do about Kimi, a free, open source model that Chinese AI company Moonshot launched last week. It appears to rival the intelligence of models from OpenAI and Anthropic, which are very much not free. 

Kimi and other Chinese models like it pose a real problem for Trump. And they’re dividing the top AI strategists in his orbit into factions. Every time a new smart, free model from China like Kimi gets released, US companies see less reason to fork out money to access models from Anthropic or OpenAI. Given that enthusiasm for these and other AI companies is driving an outsized share of economic growth, China’s AI models create both economic and political problems for the president. They are “a threat for an administration that really doesn’t want more economic bad news,” Anton Leicht, a fellow at the Carnegie Endowment, wrote on X. They’ve already rattled US stocks. 

What is Trump to do? First, consider that this is all happening just a week after New York imposed the country’s first state ban on new data centers. There is growing distrust of AI companies, and I imagine a not-insignificant share of Americans would have little sympathy for OpenAI or Anthropic as they fend off cheaper competitors, and would say it’s not the government’s job to protect their interests.

On this point, they’d see a sliver of agreement (and really just a sliver) with David Sacks, who on July 19 criticized top AI companies that “want the government to eliminate their open source competition.” He has also argued that Chinese AI models have become popular because they come with fewer restrictions on how people can use them (putting aside the built-in state censorship). 

Sacks, however, is out of a job. He no longer has a formal role advising Trump, and his position that more open AI is better has been largely replaced in the administration by one that sees a larger role for government intervention. The thinking behind this view is that because AI models have gotten strong enough to pose threats to national security, the government must control how they’re used. 

This position has fueled the new White House review process that aims to vet AI models’ security before they’re released. Dean Ball, a former Trump AI advisor who now works for OpenAI, criticized it over the weekend as a “de facto licensing regime for frontier AI.” Ball predicted Trump may solve his Chinese open source problem with a bit of soft power, perhaps by making US companies afraid to use models like Kimi. That drew a response from Michael, who, with Secretary of Defense Pete Hegseth, has been the agency’s main liaison with AI companies. Michael called Ball the AI industry’s “supreme village idiot,” bristling at the suggestion that the government would quietly strong-arm companies rather than, as Michael put it, go through “the democratic process not some Deep State scheme.”

Left out of the conversation has been how a model like Kimi got so good in the first place. For much of the Biden administration and even the beginning of Trump’s second administration, keeping China from getting top chips was a priority. Those export controls have loosened—Trump made the controversial decision to allow Nvidia to sell more chips to China, in exchange for the US government taking a cut—and the government has alleged that some chip smuggling has taken place. But China nonetheless has limited computing power, and it’s not clear what chips the company behind Kimi used to train the model. 

It’s possible that the process involved some distillation, a practice in which AI models are trained on the outputs of existing AI models. OpenAI and Anthropic have long complained that Chinese AI companies do this, and they have requested government help to put a stop to it. In April, they got it, when the Trump administration announced a series of efforts to curb the practice.  

But Kimi is out there and free, and it is nearly as good as the Anthropic model the US government deemed so powerful that it was briefly shut down because it threatened national security. The weekend’s sparring suggests many in Trump’s orbit see that as a wake-up call. But nobody can agree on what for.

Posted on

New CVD Risk Tool Boosts Women’s Risk, Lowers Risk for Some Black Adults

A change in the guidelines used to judge a person’s cardiovascular risk in the U.S. means who is classed as ‘at risk’ or not depending on their lipid levels and other factors will change.

Men, non‑Hispanic Black adults, and current smokers are more often moved down, whereas women and people with diabetes would see their risk category upgraded with the new tool suggests research led by Allison Peng, MD, Johns Hopkins University School of Medicine, and colleagues.

The research is published in JAMA and compares risk classification with the new PREVENT-atherosclerotic cardiovascular disease (ASCVD) tool and the 2013 pooled cohort equations, which have been the standard tools for estimating 10-year atherosclerotic cardiovascular disease risk for a long time.

The team used data from the National Health and Nutrition Examination Survey, a large ongoing study that interviews and examines a sample of the U.S. population every year and focused on 24,403 adults aged 40 to 79 years who did not already have cardiovascular disease and did not have very high levels of low‑density lipoprotein (LDL) cholesterol.

For each person, the investigators estimated the chance of a heart attack or stroke in the next 10 years using both the old and new calculators, grouped them as low, borderline, intermediate, or high risk under each system, and then asked who moved up or down. They also examined whether this reshuffling would change who is advised to take cholesterol‑lowering medicines such as statins.

The new tool removes race as an input, adds more risk factors such as body mass index and markers of glycemia and kidney disease, and predicts both 10‑ and 30‑year cardiovascular risk, whereas the old tool focused on 10‑year ASCVD and treated race as Black vs. White.

The results show 22% of study participants changed risk category with the new calculator. Around 14% of all adults were placed into a lower‑risk group, while about 7% were moved up. Around 50% of the population continued to be classed as low risk, but the borderline group grew from about 13% to 18%, while the intermediate group shrank from about 25% to 20%, while the high‑risk group did not change much from about 12% to 11% with the new tool.

Key changes show women’s risk increasing and men’s decreasing, factors such as diabetes playing a bigger role in risk estimation and Black adults being generally classed as lower risk.

In the older framework, an intermediate or high risk often directly triggered a statin recommendation. With the new tool, more patients sit in the enlarged borderline group and would be offered additional tests like coronary artery calcium scoring and allowed shared decision‑making about medications and preventive treatment. Despite these shifts, the proportion of adults for whom statins would be recommended was almost unchanged at around 50% before and after.

“Overall, cardiovascular risk reclassification with the 2026…Dyslipidemia Guideline may result in opportunities for consideration of more personalized cardiovascular risk assessment with risk enhancers and coronary artery calcium scoring to guide lipid-lowering therapy and cardiovascular disease prevention,” write the authors.

In an accompanying editorial in the same journal, Philip Greenland, MD, Northwestern University Feinberg School of Medicine, and Karen Lasser, MD, Boston Medical Center, Boston University, note that despite improving risk assessments, many people who are at risk still do not get treatment or reach suggested goals for healthy cholesterol levels.

“A large percentage of U.S. adults aged 30 years or older are candidates for guideline-directed lipid lowering therapy, and many who are currently taking lipid lowering therapy are not reaching new (or previous) low density lipoprotein cholesterol goals. This is a huge public health burden for the U.S. population for a medical condition that should be highly treatable.”

They conclude: “Given the large numbers of people who would be recommended for treatment following a risk-based discussion, these reports signify a major challenge, and so far, a missed opportunity, for the U.S. health care system.”

The post New CVD Risk Tool Boosts Women’s Risk, Lowers Risk for Some Black Adults appeared first on Inside Precision Medicine.

Posted on

STAT+: A most-wanted fugitive appears to have been secretly working as a biotech executive

One of Rhode Island’s most-wanted fugitives — a former doctor convicted of sexual assault but on the run for 20 years — appears to have been living a secret life as a biotech executive. 

Ronald Fischer, 70, was arrested last week by federal and Rhode Island authorities after they tracked and boarded a 56-foot sailboat cruising off the coast of New Jersey. The former anesthesiologist disappeared in 2005 while on trial for first-degree sexual assault.

The boat was registered under the name Richard Graydon, an alias used by Fischer, U.S. Marshals said. That is the same name as a doctor and seasoned drug development executive hired last March by Immix Biopharma, a Los Angeles-based biotech company, as its new chief medical officer.

Continue to STAT+ to read the full story…

Posted on

The Download: AI hiring biases, and weather data sabotage

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.

AI is more likely than humans to form biases when hiring

The next time you apply for a job, AI may screen your résumé before any human sees it. But there’s good reason to question whether AI will judge you fairly. 

We already know that LLMs pick up human biases from their training data. New research suggests they can also develop their own biases from experience—and stereotype job applicants more than humans do.

As AI companies race to build agentic models that remember the tiniest details about users, they may be handing them ammunition for forming those biases. 

Read the full story on AI’s alarming potential to stereotype job applicants.

—Michelle Kim

The risk of weather data sabotage is rising

Every morning, airline dispatchers, grid operators, and farmers around the world make decisions based on weather forecasts. More recently, the forecasts have become relevant for another industry: prediction markets, where people bet money on all kinds of real-world events, including the weather. 

The temptation to manipulate weather data to get an edge in these markets, combined with a collective move toward data-driven AI weather forecasting, is starting to put the accuracy of weather predictions at risk. 

As experts in the field, we can foresee scenarios where the risks snowball into far bigger, more systemic problems. 

Find out why the threats to weather data are growing—and how to stay ahead of them.

—Monique Kuglitsch, Jesper Dramsch, Franz G. Kuglitsch, & Andrea Toreti

The must-reads

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

1 SpaceX is negotiating to sell the Pentagon AI compute
It would provide data center capacity worth billions of dollars. (WSJ $)
+ Deepening ties between Elon Musk’s company and the DoD. (Reuters $)
+ Meanwhile, Anthropic is in talks with Meta to acquire compute. (CNBC)
+ The compute explosion is only just beginning. (MIT Technology Review)

2 Trump Media wants $100,000 a month for early access to Trump’s posts
The premium feed is being pitched to trading firms and banks. (FT $)
+ It aims to monetize Trump’s market-moving social media posts. (Reuters $)
+ Critics described the plan as “brazen corruption.” (Guardian)
 
3 ICE shared Medicaid data it wasn’t supposed to have with Palantir
Court filings show the data reached the contractor before being deleted. (NPR)
+ ICE is using data broker tools to identify “unaccompanied minors.” (Wired $) 
 
4 Apple briefly overtook Nvidia as the world’s most valuable company
The iPhone maker’s earnings durability has impressed investors. (Reuters $)
+ While Nvidia’s rise has stalled amid shifting AI bets. (CNBC)
 
5. Politicians are trying to change what chatbots say about them
A new industry has sprung up to help them edit AI outputs. (NYT $)
+ Chatbots can sway voters better than political ads. (MIT Technology Review)
 
6 Washington is opening the door to armed robots
The Pentagon is accelerating AI weapons development. (WP $)
+ “Humans in the loop” in war is an illusion. (MIT Technology Review)
 
7 China’s Moonshot has paused new subscriptions amid surging uptake
Demand for the headline-grabbing Kimi ​K3 has strained capacity. (SCMP)
+ China’s open-source AI is challenging US models. (MIT Technology Review)

8 Lab-grown teeth could soon replace fillings and implants
Scientists believe regenerative medicine could transform dentistry. (BBC)
+ Humanlike “teeth” have been grown in mini pigs. (MIT Technology Review)
 
9 AI slop on birdwatching forums is putting research at risk
It could contaminate records of species. (Guardian)

10 Heart experts have good news for your coffee habit
Roughly five cups per day is fine—and may even be beneficial. (Gizmodo)

Quote of the day

“The most authoritarian government is producing the most egalitarian models, and what should be the most democratic government is breeding companies that are the most authoritarian.” 

—Rayan Krishnan, CEO of Vals AI, a company that evaluates AI performance, gives the New York Times his take on the competition between Chinese and American models.

One More Thing

RICHARD CHANCE


The curious case of the disappearing Lamborghinis

A new wave of theft is rocking the luxury car industry—mixing high tech with old-school chop-shop techniques to snag vehicles while they’re in transport. 

It’s remained under the radar, even as it’s rocked the industry over the past two years. MIT Technology Review identified more than a dozen cases involving high-end vehicles, obtained court records, and spoke to law enforcement, brokers, drivers, and victims in multiple states to reveal how transport fraud is wreaking havoc across the country.

Find out how a new wave of transport fraud is wreaking havoc across the country. 

—Craig Silverman

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.)

+ Here’s the perfect outfit for a heat wave: the world’s first self-cooling clothes.
+ The “unhinged” cake decorators at a Missoula Walmart have become local legends.
+ This visual guide to chili peppers around the world is a lavishly illustrated ode to the world’s hottest fruit.
+ Take a quirky tour of mid-20th-century cinema in this thread of film posters inadvertently photographed by postwar town planners.

Posted on

Opinion: My husband’s suicide shows there’s something very wrong with the U.S. insurance industry

I remember standing outside an exam room, phone vibrating in my breast pocket, when the coroner’s call came through. I hadn’t wanted to believe the neighbor’s frantic text about a body bag removed from my husband’s condo, but the medical examiner’s office on my caller ID was unmistakable. I answered, and sounds fell out of my mouth into the phone: “Hello, this is Dr. Hardison.”

I am an emergency physician living in the U.S., and we had one of the top commercial health insurance plans. When my husband, Randy, became suicidal for the first time in his life, I thought I could find him the best care, our insurance would pay for it, and he would get better. Only one of those three things actually happened.

Read the rest…

Posted on

StockWatch: Lilly’s Up-to-$3.8B Deal for AtaiBeckley a Good Trip for Psychedelic Drugs, Analysts and Investors Agree

It wasn’t too long ago that biopharma giants stayed away from developing psychedelic drugs—but positive clinical data plus a friendlier regulatory climate in Washington have prompted the largest drug developers to embrace the field.

The latest and most telling example of pharma embracing psych drugs came when Eli Lilly (NYSE: LLY) announced that it agreed to acquire AtaiBeckley (Nasdaq: ATAI) for up to $3.8 billion—of which Lilly will pay $2.8 billion upfront. The deal, set to close in the third quarter, expands Lilly’s neuroscience portfolio by adding the pipeline of AtaiBeckley led by BPL-003 (mebufotenin benzoate), a Phase III candidate for treatment-resistant depression (TRD) that is a synthetic form of 5-MeO-DMT administered intranasally. BPL-003 has been granted the FDA’s Breakthrough Therapy designation.

BPL-003 wowed analysts and others back in April after AtaiBeckley published positive data from a Phase IIa trial (NCT05660642) showing that a single intranasal dose of BPL-003 led to rapid and sustained reductions in Montgomery-Åsberg Depression Rating Scale (MADRS) scores from baseline in 12 TRD patients who remained on stable SSRI therapy throughout the study. Both the six patients dosed at 10 mg and six at 12 mg showed a 66.7% antidepressant response rate (defined as ≥50% reduction from baseline MADRS score) at Day 2, with five of six participants in the 10 mg cohort (83%) and four of six in the 12 mg cohort (66.7%) maintaining their response at Week 12.

“Especially with progress on BPL-003, we see the company as positioning itself well to becoming a significant player in the mental health therapeutics space,” Sumant Kulkarni, a senior analyst covering biotechnology with Canaccord Genuity, wrote on news of the positive data, adding: “We also still see this space as large enough to accommodate multiple approaches/competitors.”

$3.7B in projected peak sales

Kulkarni also raised Canaccord Genuity’s peak unadjusted U.S. sales forecast for BPL-003 to $3.7 billion by 2036 from $2 billion, after revising the firm’s model by raising the list price from $20,000 to $30,000 per annual treatment course (not accounting for insurance coverage), about the same price as Spravato® (esketamine), also a nasal spray marketed by Johnson & Johnson (NYSE: JNJ) for TRD plus some depressive symptoms in adults with major depressive disorder (MDD).

Spravato, a noncompetitive N-methyl D-aspartate (NMDA) receptor antagonist, crossed the $1 billion sales threshold during the second quarter, as it generated $584 million, up 25% quarter-over-quarter from $464 million in Q1—and up 43% from $734 million in the first half of 2025.

“Sales are tracking to reach annual sales guidance of $3-3.5B+ by 2027–28,” Jefferies equity analyst Andrew Tsai wrote in a research note focused on J&J’s second-quarter results. “Spravato’s trajectory supports the notion psychedelics can be commercially viable in hard-to-treat mental health disorders, by leveraging JNJ’s infrastructure.”

Given the data for BPL-003, Lilly got a bargain, Tsai wrote in a separate note on the Lilly-AtaiBeckley acquisition.

“We think the deal heavily favors LLY, as ATAI’s lead asset BPL-003 (intranasal 5-MeO-DMT) should have multibillion dollar peak sales potential,” Tsai wrote, rather than the $1 billion-plus that he thinks was implied by the deal price.

Tsai and Jefferies had previously forecast peak sales of between $1 billion and $2 billion—a range he said was “arguably conservative” since BPL-003 could, if it aces its Phase III trial, show superiority to Spravato, which is on track to reach up to $5 billion-plus in peak sales.

Positive implications

“At the same time, we appreciate LLY has significantly more resources to maximize the long-term value of ATAI’s psychedelic assets. In any case, the deal has (+) [positive] implications for the entire psychedelic space,” Tsai added.

Among pharma giants joining J&J in embracing psychedelic drug development in recent years:

  • AbbVie (NYSE: ABBV), which last year acquired the lead pipeline program of privately held Gilgamesh Pharmaceuticals, the moderate-to-severe MDD candidate bretisilocin (GM-2505), for up to $1.2 billion.
  • Otsuka Holdings (Tokyo Stock Exchange: 4578), which in 2023 acquired Mindset Pharma, a Canadian psych drug developer focused on psychiatric and neurological disorders, for C$80 million ($56 million).

With its deal for AtaiBeckley, Lilly becomes the latest pharma giant to perceive the positive implications Tsai cited.

“Treatment-resistant depression persists even after multiple treatments have failed. Millions of people are still searching for relief and desperately need a therapy that works,” Carole Ho, executive vice president and president, Lilly Neuroscience, said in a statement. “Advancing AtaiBeckley’s investigational therapies gives us a real chance to change that.”

Investors agreed with Lilly, giving the pharma a 1% increase Thursday, the day the acquisition was announced, from $1,156.63 to $1,169.17—no small feat since buyers typically stay flat or see their shares slide after announcing an acquisition. And not surprisingly, AtaiBeckley investors were enthusiastic about the deal, as its stock leaped 33% from $5.36 to $7.15. On Friday, Lilly inched up 0.8% to $1,178.58 while AtaiBeckley rose 1% to $7.22.

The AtaiBeckley buyout is Lilly’s eighth announced acquisition of a smaller biopharma this year.

Lilly is acquiring three infectious diseases vaccine developers—Vaccine Company for up to $1.55 billion, Curevo for up to $1.5 billion, and LimmaTech Biologics for up to $780 million—as well as in vivo chimeric antigen receptor T-cell (CAR T) developer Kelonia Therapeutics for up to $7 billion); JAK inhibitor developer Ajax Therapeutics for up to $2.3 billion; next-generation dual-payload antibody-drug conjugate (ADC) developer CrossBridge Bio for up to $300 million; and nonviral DNA delivery-focused drug developer Engage Biologics for up to $202 million cash.

The deal spree reflects Lilly’s desire to capitalize on the billions of dollars it is generating from sales of its obesity and diabetes drugs based on glucagon-like peptide 1 (GLP-1) receptor agonists alone or in tandem with a glucose-dependent insulinotropic polypeptide (GIP).

“If we see great ideas that we think we can use to help people that need them, of course we’ll do deals,” Daniel M. Skovronsky, MD, PhD, Lilly’s chief scientific and product officer and president of Lilly Research Laboratories, said on CNBC.

“Positive development”

David Risinger, a senior managing director and senior research analyst covering diversified biopharmaceuticals at Leerink Partners, said his firm viewed Lilly’s buyout of AtaiBeckley “as a positive development because it enhances LLY’s pipeline of potential neuroscience blockbuster candidates.”

That pipeline is led by five Phase III programs involving four drugs, none of them a psychedelic. Two of the programs belong to brenipatide, a dual agonist of both the GIP and GLP-1 receptors. Brenipatide is being developed for both MDD and alcohol use disorder.

Also in Lilly’s late-stage neuroscience pipeline are:

  • Donanemab, which binds to deposited amyloid plaque in the brain and is being studied for the treatment of cognitively unimpaired Alzheimer’s disease.
  • Ixoberogene Soroparvovec (Ixo-Vec), an intravitreal gene therapy being studied as a single one-time treatment for vision loss associated with neovascular (wet) age-related macular degeneration (AMD).
  • Remternetug (LY3372993), which also binds to deposited amyloid plaque in the brain and is under study as a treatment of cognitively unimpaired/mild cognitive impairment due to Alzheimer’s disease, with potential for subcutaneous delivery.

In addition, AtaiBeckley “would provide ​differentiated exposure in psychiatry and reinforce [Lilly’s] ​broader effort to diversify beyond ​its cornerstone cardiometabolic franchise,” observed Evan David Seigerman, a managing director and head of healthcare research at BMO Capital Markets, as reported by Reuters.

AtaiBeckley was formed last November by the merger of atai Life Sciences and Beckley Psytech. The company’s stock has nearly doubled, soaring 98% over the past six months from $3.64 on January 16.

“Going mainstream”

“Psychedelic Medicine is going mainstream,” declared Steve Jurvetson, co-founder of Future Ventures, in a post on X. Jurvetson and Future were among early investors, along with Peter Thiel in atai Life Sciences.

AtaiBeckley is one of numerous psychedelic drug developers to show significant six-month gains since January: As of Friday’s closing bell, Compass Pathways (Nasdaq: CMPS) shares jumped 68% to $12.35, GH Research ballooned 69% to $28.71, while Definium Therapeutics (Nasdaq: DFTX) nearly tripled, zooming 194% to $44.29.

Interestingly, those three companies did not get a solid bounce from AtaiBeckley’s acquisition by Lilly. Since the deal was announced Thursday, Compass fell 7% from $13.31 pre-announcement, Definium dipped 3% from $45.66. GH rose 8% Thursday from $26.92 to $29.13, before sliding 1.4% the following day.

Bucking the trend was Cybin, d/b/a Helus Pharma (Nasdaq: HELP), which has climbed 11% since the Lilly-AtaiBeckley announcement, from $6.51 to $7.25. Its shares have slumped 6% since January—but soared 58% over the past month on positive news, such as the 88%+ enrollment rate of patients in Helus’ Phase III APPROACH pivotal trial (NCT06564818) of HLP003 in MDD, on track for topline data readout in Q4 2026.

“We see the potential for 150–200% upside [jump in stock price] if Phase III data in 4Q26 are positive,” Kulkarni wrote, making it the largest potential jump among psychedelic drug developers.

In addition to favorable data, the stock surges also reflect actions by President Donald J. Trump’s administration to encourage psychedelic drug development. In April, President Trump signed Executive Order 14401, directing the FDA and other federal agencies to accelerate research and improve access to psychedelic drugs, citing their potential as promising treatments for serious mental illnesses.

And on July 13, the FDA published “Psychedelic Drugs: Considerations for Clinical Investigations,” a final guidance designed to provide general considerations for developers of psych drugs, with recommendations for how to conduct clinical trials for the treatments.

“Rather than providing specific recommendations on study design, this guidance will present foundational constructs that all sponsors studying the therapeutic potential of psychedelic drugs, including sponsors without commercial drug development as primary interest (e.g., academic researchers), should consider,” the FDA wrote in the final guidance. “Sponsors are encouraged to request meetings with FDA for advice on a specific drug development program.”

Leaders and laggards

  • Q32 Bio (Nasdaq: QTTB) shares nearly doubled, leaping 91% from $11.21 to $21.38 July 13 after the autoimmune and inflammatory disease drug developer announced positive 36-week topline results from Part B of the Phase IIa SIGNAL-AA trial (NCT06018428) assessing bempikibart in patients with severe or very severe alopecia areata. Q32 said it saw clinically meaningful efficacy data on the primary endpoint of mean percent change from baseline in SALT score, with a reduction from baseline of 35.3% in the prespecified modified intent to treat (mITT) analysis. The company also reported that 40.0% of patients (10/25) achieved SALT-20 response at Week 36 in the mITT analysis, while 30.3% of patients (10/33) achieved SALT-20 response at Week 36 in the ITT analysis of all enrolled patients.
  • Veradermics (NYSE: MANE) shares yo-yoed this past week, climbing 12% from $110.17 to $123.70 Wednesday after the pattern hair loss drug developer announced positive topline results from its open-label Phase II Study 207 trial (NCT06527365) assessing VDPHL01, an extended-release oral minoxidil formulation, in women with mild-to-moderate pattern hair loss. Veradermics said most study participants reported improved hair coverage at Month 2, with approximately 88.9% of patients dosed once daily and 90.0% dosed twice daily reporting “improved” or “much improved” outcomes at Month 6. Participants dosed once daily showed a mean increase in non-vellus target area hair count (TAHC) of 22.7 hairs/cm² at Month 6, an average that rose to 23.3 hairs/cm² in twice daily dosed patients. The mini surge was short-lived, however, as investors more than gave back the gain, selling off shares to send them tumbling 14% to $105.83 Thursday amid possible investor questions about whether the good clinical news was already reflected in the stock price.

The post StockWatch: Lilly’s Up-to-$3.8B Deal for AtaiBeckley a Good Trip for Psychedelic Drugs, Analysts and Investors Agree appeared first on GEN – Genetic Engineering and Biotechnology News.