Tag: United States

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Obamacare rolls shrank dramatically in many states over the past year, new federal data shows

NEW YORK — States across the country saw steep drops in the number of people covered by the Affordable Care Act over the past year, with Ohio and Oklahoma each losing nearly one-third of enrollees, according to new federal data that provides the first complete 50-state breakdown of sharp enrollment declines following the January expiration of enhanced subsidies.

The data, posted in late June by the Trump administration and first reported on by The Associated Press, reveals how changes in each state’s insured population led to around 2.6 million fewer Americans having Obamacare plans in February compared with the same time last year.

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Structural Biology Reveals New Drug Targets for Rare Neuromuscular Disease

Researchers at the University of California San Diego have uncovered the structural mechanisms that cause congenital myasthenic syndromes (CMS), revealing mutation-specific treatment strategies and identifying an existing antidepressant as a promising candidate for drug repurposing.

The study, published in Nature, explains how disease-causing mutations alter the human acetylcholine receptor (AChR), the protein responsible for converting nerve signals into muscle contraction. The findings also provide a framework for developing precision medicines tailored to the specific mutation carried by individual patients.

CMS is a rare inherited group of neuromuscular disorders that typically appears at birth or early childhood. Mutations in the AChR impair communication between nerves and muscles, leading to muscle weakness, difficulty walking, breathing problems, and, in severe cases, paralysis or death. Although more than 50 disease-causing mutations have been identified, the molecular mechanisms underlying these disorders have remained largely unknown.

To address this question, the investigators determined 12 high-resolution structures of representative mutant AChRs with and without therapeutic compounds. By combining these structural data with functional studies, they identified common mechanisms that explain the two major forms of CMS.

Fast-channel CMS is caused by loss-of-function mutations that reduce receptor opening and weaken neuromuscular transmission. The researchers discovered a previously unknown allosteric binding pocket that can be targeted by positive allosteric modulators (PAMs), compounds that enhance receptor activity without directly activating the channel.

Notably, different PAMs restored receptor function in different patient mutations rather than producing a universal effect. The authors write that this “mutation-specific activity underscores the capability and necessity of tailoring therapies to individual patient genotypes.”

The study also showed that the newly identified drug-binding pocket is largely absent until a PAM binds, creating an opportunity to design more effective compounds that stabilize receptor function. The authors conclude that these findings point to “a new class of allosteric drugs” with potential applications not only in fast-channel CMS but also in disorders such as myasthenia gravis, in which acetylcholine receptor function is compromised.

The team also investigated slow-channel CMS, a gain-of-function disorder in which receptors remain open too long, causing excessive calcium influx and progressive damage at the neuromuscular junction.

Structural analyses revealed that the current therapies quinidine and fluoxetine act through a shared mechanism, blocking the receptor pore despite their distinct chemical structures.

The investigators also evaluated reboxetine, an antidepressant already approved in several countries. Unlike the existing drugs, reboxetine selectively inhibited abnormal receptor activity across multiple slow-channel mutations while largely sparing normal receptor function.

The authors believe that reboxetine’s ability to suppress pathological receptor activity, together with its established clinical safety profile, “positions it as a potential candidate for slow-channel CMS therapy,” although they caution that its known adverse effects warrant careful evaluation in future clinical studies.

Beyond identifying therapeutic opportunities, the study establishes unifying principles of CMS pathogenesis. The researchers found that fast-channel mutations weaken the coupling between acetylcholine binding and channel opening, whereas slow-channel mutations stabilize an abnormally widened, desensitized-like pore. These shared structural mechanisms explain why genetically diverse mutations produce similar clinical symptoms while requiring different therapeutic strategies.

The work also revises understanding of receptor biology. The authors write that their findings “overturn the traditional view of the β subunit as merely a structural scaffold,” instead demonstrating that it plays “a central role in the gating cycle.”

Overall, the researchers conclude that their integrated structural and functional analyses define how CMS mutations disrupt receptor gating, reveal the molecular basis of current and candidate therapies, and identify new druggable sites for intervention. They conclude that the work provides “a roadmap towards the development of safer and more effective, mutation-specific treatments for both fast-channel and slow-channel CMS.”

 

 

The post Structural Biology Reveals New Drug Targets for Rare Neuromuscular Disease appeared first on Inside Precision Medicine.

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Your family’s $300 stake in OpenAI

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

OpenAI CEO Sam Altman’s oft-discussed promise that Americans will share in the wealth AI creates was in the news again last week. On Thursday, the Financial Times reported that Altman is in talks with President Trump about giving the US government a 5% stake in OpenAI.

In some ways, Altman’s plan is old news. He wrote about a more radical version of this back in 2021, proposing that all companies above a certain valuation (not just AI companies) pay 2.5% of their market value each year into a fund that sends Americans annual disbursements. In April this year, OpenAI described a narrower proposal that closely resembles what Altman is reportedly discussing with Trump now. And the notion has broad political appeal: Senator Bernie Sanders has proposed giving Americans a 50% stake in top AI companies.

What’s the logic here? For would-be recipients, it’s twofold. First, AI learns directly from human-generated work—books, movies, art—but AI companies generally never pay the authors of that work. A free equity stake could serve as a form of belated compensation. Second, the payout could mitigate the widespread anxiety that AI will cause a collapse of the labor market (even if economists disagree) by providing a safety net. 

How large a safety net is up for debate. Details of OpenAI’s latest proposal are sparse, but let’s say the government were to distribute this equity stake directly to Americans. After its funding round in March the company was valued at $852 billion, making a 5% stake in OpenAI worth about $42.6 billion today (the company is reportedly delaying its IPO until it can reach a $1 trillion evaluation, a tall order given that it’s spending heavily on data centers and still has not turned a profit). Distributing that $42.6 billion equally among the roughly 133 million American households would give each about $320 in equity. But if it were to operate like other wealth funds, the government would not give equity directly to Americans but rather let the fund grow and then share a portion of the returns with everyone, perhaps delivering a bigger payout, if and when AI companies can ever start sustainably turning a profit.

If this dividend does materialize, what’s in it for tech companies? Altman might hope the promise of payouts could help swing public opinion a bit more back toward AI companies. (A majority of Americans don’t trust companies to use AI responsibly and oppose construction of data centers in their area, and half are more concerned than excited about the increased creep of AI into their daily lives.)

But the bigger prize for OpenAI might be that the Trump administration loves making tech deals—like its equity stake in Intel and its share of Nvidia’s sales to China, among others.  Staying on the administration’s good side is pretty essential for AI companies right now (just ask Anthropic). It could mean not having your models deemed a supply chain risk, or getting more help from the White House in stopping your rivals from China. 

My main takeaway is that these plans currently function more as a story than a policy. Altman has been talking about some version of this idea for five years and reportedly pitched it to President Trump soon after he took office, yet there is still little indication that a concrete plan is taking shape. The more ambitious proposal from Sanders is even less likely to gain traction.

But what these plans do reveal is just how up for debate the future of AI still is. Altman drew inspiration for his plan from the Alaska Permanent Fund, which was set up in the 1970s to give Alaskans a share in oil profits. The idea was based on two premises: that oil is a shared resource, and that eventually it will run out. Altman seems happy to concede the first claim about AI. But he’d balk at the second, having promised that AI will generate extraordinary wealth for decades to come. Whether Americans ever receive a check is beside the point; the proposal’s real purpose may be to convince them that the AI boom will be large enough to share.

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STAT+: Online GLP-1 prescriptions are often fast, easy — and low on clinical oversight

A secret shopper study has pulled back the veil on the practices of nearly 50 telehealth sites that prescribe popular GLP-1 medications for weight loss.

Direct-to-consumer telehealth companies have grown rapidly in response to patients’ demand for GLP-1s like semaglutide and tirzepatide, especially after shortages created a market for compounded versions that aren’t approved by the Food and Drug Administration. As online prescribing has grown, clinicians and public health experts have raised concerns about the potential for lax virtual care practices to put patients at risk. 

In a study published Monday in the Journal of the American Medical Association, one researcher at Yale University set out to document those online prescriptions by posing as a patient. The results capture what many Americans have learned in the last few years: It is extraordinarily fast and easy to get a GLP-1 online. 

Continue to STAT+ to read the full story…

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Construct Validation of a Remote Brain Health Assessment Battery to Evaluate Vocational Aptitude and Factors Associated With Cognitive Resilience in the Military: Observational Trial

<strong>Background:</strong> Vocational aptitude and cognitive resilience predict military success, yet current assessments rely on resource-intensive, in-person testing that limits scalability. A brief, self-administered, remotely deployable computerized battery offers a practical solution for large-scale screening and monitoring. <strong>Objective:</strong> This study aims to deploy a set of computerized assessments among National Guard recruits and assess their preliminary construct validity against a standardized aptitude measure and a research-based proxy for cognitive resilience. <strong>Methods:</strong> In this observational study, 267 enlisted service members from the Minnesota Army National Guard participated in 2 complementary ethics-approved observational trials: Office of Naval Research Neuropsychometrics and Advancing Research on Mechanisms of Resilience (ARMOR). National Guard soldiers in ARMOR completed the Armed Forces Qualification Test (AFQT), Penn Computerized Neurocognitive Battery (Penn CNB), and a 20-minute computerized brain health assessment battery (BrainHQ) at separate time points over the course of their military careers. BrainHQ assessments consisted of adaptive psychophysical tasks measuring the speed and accuracy of visual and auditory information processing. The battery assessed decision-making speed, emotion-processing speed, selective attention under speeded conditions, working memory capacity for speeded visual elements, verbal memory and learning of speeded speech, and problem-solving speed. The Penn CNB included nonspeeded neuropsychological assessments of executive function, verbal memory, social cognition, and reasoning. Linear regression evaluated the association between BrainHQ performance and AFQT percentiles, and partial correlations assessed associations between conceptually related BrainHQ and Penn CNB subtests. <strong>Results:</strong> Participants were predominantly young (mean age 19.1 years) and male (178/267, 66.7%). BrainHQ performance was significantly associated with enlistment eligibility and vocational aptitude, as measured by the AFQT (<i>P</i>&lt;.001), after controlling for age and education. The overall model explained 24.4% of the variance in AFQT percentiles (adjusted R2=0.227). The BrainHQ assessment composite was the strongest predictor, uniquely accounting for 19.2% of the variance and supporting the construct validity of aptitude. These associations persisted despite the temporal separation between assessment time points. Quartile analyses showed graded relationships between BrainHQ performance and AFQT eligibility thresholds, with higher BrainHQ performance associated with progressively greater probabilities of meeting higher AFQT benchmarks. Preplanned partial correlations between BrainHQ subtests and standardized neurocognitive measures from the Penn CNB showed significant positive associations (r=0.17-0.25; all <i>P</i>&lt;.001 to .02) with cognitive domains typically associated with cognitive resilience. <strong>Conclusions:</strong> A brief, self-administered, and scalable brain health battery demonstrates associations with military vocational aptitude and with neurocognitive domains associated with cognitive resilience. Future studies should evaluate whether integrating these assessments into current practices predicts success in Basic Combat Training, guides military progression, and supports long-term cognitive screening and monitoring across the Armed Forces.
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Opinion: Stopping doctors from ordering unnecessary diagnostic tests requires a structural fix

American medicine runs more than 14 billion tests a year. While some tests can be lifesaving, many are used at the wrong time or on the wrong patient and are useless or even harmful.

The medical industry has spent enormous effort making more advanced tests but expended little effort learning how to use tests correctly. There is a science for how to better use tests, diagnostic stewardship, but most doctors have never heard of it.

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MH-POWER for College Students on the Spectrum

Conditions: College Students on the Autism Spectrum With Mental Health Challenges

Interventions: Behavioral: The MH-POWER program

Sponsors: University of Texas Rio Grande Valley; The American Occupational Therapy Foundation

Recruiting
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Pregnenolone for Cannabis Use Disorder and Depression

Conditions: Cannabis Use Disorder; Major Depressive Disorder (MDD)

Interventions: Drug: Pregnenolone

Sponsors: University of Texas Southwestern Medical Center; National Institute on Drug Abuse (NIDA); The University of Texas at Dallas

Not yet recruiting
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StockWatch: New UC Data Sparks Smoother Sailing for Abivax

Less than a month after its stock roller-coastered on safety signals associated with its late-stage ulcerative colitis (UC) drug candidate obefazimod, shares of Abivax (Euronext Paris and Nasdaq: ABVX) enjoyed smoother sailing this past week—namely a 63% surge in Europe and a 50% leap in the United States over four trading days, following more positive data that appeared to reassure investors.

Abivax declared that obefazimod “delivered meaningful clinical benefit” to adults with moderately to severely active UC in the ABTECT Maintenance Part 2 supplemental portion of its Phase III UC maintenance program, with 37.2% of induction nonresponders achieving clinical remission and 34.5% achieving endoscopic remission at Week 44 following continued 50 mg treatment. Of those patients, 61.5% also showed clinical response, 48.0% endoscopic improvement, and 44.6% Histologic-Endoscopic Mucosal Improvement (HEMI).

In patients whose doses were escalated to 50 mg, clinical remission was recaptured in 45.5% of patients who relapsed during ABTECT Maintenance Part 1—a result Abivax said supported a practical dose-escalation strategy for regaining and sustaining disease control over time.

Of special interest to investors, no new safety signals were seen since earlier this month, when Abivax disclosed various malignancies in nine patients among the 580 enrolled in the study. The earlier disclosure triggered price plunges of 44% for both Abivax’s ordinary shares traded on Euronext Paris and the company’s American depositary shares (ADSs) traded on the Nasdaq Global Market.

Established risk factors

The latest data from ABTECT Maintenance Part 2 showed four total cases of non-melanoma skin cancer (NMSC)—two in the study’s 25 mg arm, two in the 50 mg arm: “All occurred in patients with established NMSC risk factors including advanced age, thiopurine use, prior skin cancer history, and failure of multiple prior advanced therapies,” Abivax stated.

Significantly, incidence rates of malignancies (including NMSCs) when adjusted for patient-year exposure were well within the pre-defined background reference ranges based on previous UC studies.

Exposure-adjusted incidence rates (EAIRs) for malignancies excluding NMSC were 0.48 and 0.69 events per 100 person-years (PYs) in the all-active combined (50 mg + 25 mg) and 50 mg cohorts, respectively, and for NMSC were 0.95 and 0.69 events per 100 PYs, in the all-active combined (50 mg + 25 mg) and 50 mg cohorts respectively, all consistent with expected UC background rates

EAIRs for malignancies excluding non-melanoma skin cancer (NMSC) were 0.48 per 100 PYs in the all-active combined (50 mg + 25 mg) cohort, and 0.69 events per 100 PYs in the 50 mg cohort. For NMSC, EAIRs were 0.95 in the all-active combined cohort and 0.69 in the 50 mg cohort. All those results were consistent, Abivax said, with expected UC background rates ranging from 0.30–0.70 for malignancies excluding NMSC, and 0.70–1.40 for NMSC.

“Paradigm-defining treatment”

“The expanded cumulative safety data further strengthens our confidence in the long-term safety profile of obefazimod and reinforces the favorable benefit-risk profile for our program as we prepare for our planned NDA [New Drug Application] submission later this year,” Abivax CEO Marc de Garidel stated. “We believe this growing body of evidence positions obefazimod, if approved, to become a paradigm-defining treatment option for patients living with ulcerative colitis.”

Investors appeared to share de Garidel’s optimism. The data sparked a buying surge among investors, who sent Abivax shares traded on Euronext Paris soaring 39% the day after the announcement, from €83.30 ($94.71) to €115.50 ($131.31) on Tuesday. The shares rose another 1.7% Wednesday, closing at €117.50 ($133.59), then climbed another 9% Thursday to €127.80 ($145.28) before finishing the week with a 6% increase, to €135.80 ($154.38) and a 63% one-week gain.

On Nasdaq, Abivax ADSs surged 50% for the week, consisting of a roughly 39% leap Tuesday from $96.15 to $133.26. From there, shares dipped 0.5% the following day to $132.56, before rebounding 9% Thursday, finishing the Independence Day holiday-shortened week at $144.65.

Wednesday was a shorter trading day than usual since the company requested a temporary, single-day halt. Abivax requested the halt to price an upsized offering of its U.S. American depositary shares (ADSs), which increased from the originally announced $600 million to $800 million—6.4 million ADSs at $125 per ADS, which the company expected would extend its cash runway into the second quarter of 2029.

The offering closed Thursday at $920 million, of which approximately $874.1 million consisted of net proceeds, after underwriters exercised in full their option to purchase 960,000 additional ADSs representing 15% of the total number initially sold in the offering.

The size of the offering appeared, based on investor chatter cited by Stocktwits, an effort to dampen speculation about Abivax being a prime candidate for a buyout; the company appears in GEN’s most recent A-List of Top 10 Takeover Targets of 2026. But the upsizing of the offering rekindled buyout speculation by individual or “retail” investors, the same outlet reported Thursday.

Abivax said it intends to use the net proceeds toward expenses relating to potential commercialization of obefazimod in the United States; clinical R&D expenses, primarily related to UC and Crohn’s disease; and the remainder, if any, for general corporate purposes.

Leerink Partners, Morgan Stanley, Piper Sandler, and Guggenheim Securities were joint bookrunning managers for the offering, while LifeSci Capital acted as a passive bookrunning manager and Van Lanschot Kempen as the lead manager.

“Response rates (clinical & endoscopic remission) in this portion also appear compelling, especially given the refractory nature of patients in this subset, reaffirming obe’s best-in-disease efficacy,” Thomas J. Smith, senior managing director, immunology and metabolism, and a senior research analyst with Leerink Partners, commented in a research note.

“Should allay investor concerns”

“We believe this update further de-risks obe’s profile in UC and Crohn’s and should allay investor concerns following Part 1 maintenance data released earlier this month,” Smith added.

Smith raised his firm’s 12-month price target on Abivax shares 6%, from $140 to $148.

Two other firms also raised their price targets on Abivax stock:

  • BTIG (Julian Harrison)—Up 17%, from $150 to $175, maintaining “Buy” rating.
  • Wedbush Securities (David Nierengarten)—Up 22% from $90 to $110, maintaining “Neutral” rating.

Even more positive feedback on the latest data came from Faisal Khurshid, a managing director and equity research analyst with Jefferies. Khurshid upgraded his firm’s rating on Abivax’s stock from “Hold” to “Buy,” and boosted Jefferies’ price target 46%, from $108 to $158.

On June 1, Khurshid downgraded Jefferies’ rating on Abivax from “Buy” to “Hold,” citing the safety concerns he said have since been addressed.

“Mgmt. did a nice job addressing investor concerns w/ how they presented the safety data [June 29] vs. the Part 1 update. On top of that, the efficacy profile strengthens w/ each add’l piece of data,” Khurshid observed.

He also cautioned: “There is still risk on cash runway, catalyst path, and commercial needs for a pot’l standalone launch. But ultimately, good data should generate value.”

The biggest outstanding risk for Abivax, Khurshid wrote, is the need for significant resources associated with a commercial launch for an indication in inflammatory bowel disease (IBD): “We still think pot’l of asset better realized w/ a strategic partner.”

Obefazimod is a small molecule upregulator of miR-124, an anti-inflammatory microRNA. It enhances the selective splicing of a single long noncoding RNA to generate miR-124, which downregulates cytokines and chemokines shown to promote inflammation, including tumor necrosis factor (TNF) alpha, IL-6, monocyte chemoattractant protein-1 (MCP-1), and IL-17, as well as Th17+ cells.

Under its former name ABX464, obefazimod was initially developed against HIV but was repurposed to fight inflammatory conditions based on its anti-inflammatory effect.

Leaders and laggards

  • Elicio Therapeutics (Nasdaq: ELTX) shares tumbled 37% from $5.14 to $3.22 Thursday after the developer of immunotherapies for high-prevalence cancers said it entered into a definitive securities purchase agreement led by two new “fundamental institutional investors” with participation from a large existing shareholder—all undisclosed—to purchase 4,380,313 shares of Elicio common stock through a registered direct offering. The offering is expected to result in gross proceeds of approximately $15 million before deducting placement agents’ fees and other expenses, Elicio said. Titan Partners, a division of American Capital Partners, is acting as lead placement agent while B. Riley Securities is acting as co-placement agent.
  • Takeda Pharmaceutical (Tokyo Stock Exchange: 4502) shares increased 2.4% from ¥5,150 ($31.91) to ¥5,274 ($32.68) Thursday and rose another 1.6% to ¥5,359 ($33.20) after the pharma announced an up to $600 million artificial intelligence (AI)-based drug discovery collaboration with Insilico Medicine (Hong Kong Exchange: 3696.HK). Insilico agreed to use its end-to-end platform in leading AI-driven discovery to identify molecules meeting predefined scientific and early development criteria, while Takeda agreed to apply its global development capabilities to advance selected candidates through clinical validation across its therapeutic areas. Takeda gained exclusive worldwide rights to develop, manufacture, and commercialize novel therapeutics selected through the collaboration. Takeda’s American depositary shares (NYSE: TAK) rose 5% from $15.95 to $16.77 Thursday (U.S. markets were closed Friday for the Independence Day holiday). Insilico shares fell 4.1% from HKD 39.62 ($4.97) to an even HKD 38 ($4.77) Thursday and slid 1.6% to HKD 37.38 ($4.66) Friday.

The post StockWatch: New UC Data Sparks Smoother Sailing for Abivax appeared first on GEN – Genetic Engineering and Biotechnology News.