Radically different
Nature Medicine, Published online: 07 April 2026; doi:10.1038/s41591-026-04307-8
In a phase 1 basket trial, the small interfering RNA zodasiran, targeting ANGPTL3, lowered triglycerides in patients with severe hypertriglyceridemia and lowered both triglycerides and low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolemia.
An experimental HIV prevention pill being developed by Merck could be mass produced for less than $5 per patient a year according to a new analysis. Advocates argue the low cost means the company should find it easier to license the drug so that low- and middle-income countries can gain easy access.
The pill, dubbed MK 8527, is currently undergoing a pair of late-stage clinical trials that are expected to determine whether the medicine can lower HIV transmission when given to people at high risk of infection. The results are due in the latter half of 2027, according to separate postings on ClinicalTrials.gov.
Already, the pill is generating considerable interest after Merck released mid-stage results last summer showing its drug holds promise. In addition to being safe and effective, the study found it could protect against infection, a form of prevention known as pre-exposure prophylaxis or PrEP, within 24 hours after being taken. Merck noted the pill works in a novel way.
Good morning, everyone, and welcome to another working week. We hope the weekend respite was relaxing and invigorating because that oh-too-familiar routine of meetings, deadlines, and the like has returned with a vengeance. You knew this would happen, yes? To cope, we are relying, as always, on cups of stimulation. Our choice today is laced with traces of cocoa. Feel free to join us. Remember, no prescription is required. Meanwhile, here are some tidbits to help you along. Best of luck accomplishing your goals today and, of course, do keep in touch. …
In February, a small biotech company called Kezar Life Sciences reached a breakthrough with the U.S. Food and Drug Administration, agreeing to a plan for a clinical trial it hoped could lead to the approval of its treatment for a rare, debilitating liver disease called autoimmune hepatitis. The problem: The agreement came four months too late, STAT explains. The meeting to discuss trial design, a critical step in the drug development process, had been scheduled for last October. But the FDA abruptly canceled it without explanation. The company could no longer proceed as planned and, without clarity from regulators, its path forward was unclear. Kezar’s investors wanted out, and the biotech was forced to start the process of winding down.
Americans starting weight loss medicines for the first time want lower cost and greater convenience as they consider pills from Novo Nordisk and Eli Lilly, Reuters says, citing seven doctors who specialize in obesity. Novo’s Wegovy pill has been on the market since January, while Lilly’s newly approved Foundayo joins the fray this week. Interviews with the specialists show a promising landscape for oral weight loss drugs as the companies compete for share in the fast-changing obesity treatment market that is seen topping $100 billion a year in the next decade. All seven doctors said they had begun prescribing oral Wegovy, and three said they have prescribed the pill to about 10% of their patients. Of those patients, most are taking a GLP-1 for the first time, rather than switching from injectables, and have not yet reached the highest dose.
Nature Medicine, Published online: 06 April 2026; doi:10.1038/s41591-026-04282-0
In the phase 3 EMN24 IsKia trial, transplant-eligible patients with newly diagnosed multiple myeloma who received isatuximab with carfilzomib, lenalidomide and dexamethasone pretransplant induction and post-transplant consolidation showed higher rates of measurable residual disease negativity after consolidation than patients who received carfilzomib, lenalidomide and dexamethasone.
In February, a small biotech company called Kezar Life Sciences reached a breakthrough with the Food and Drug Administration, agreeing to a plan for a clinical trial it hoped could lead to the approval of its treatment for a rare, debilitating liver disease called autoimmune hepatitis. The problem: The agreement came four months too late.
The meeting to discuss trial design, a critical step in the drug development process, had been scheduled for last October. But the FDA abruptly canceled it without explanation. The company could no longer proceed as planned and, without clarity from regulators, its path forward was unclear. Kezar’s investors wanted out, and the biotech was forced to start the process of winding down.
It laid off most of its staff of about 60 people. Then, it auctioned off its lab equipment and sold much of its office furniture, except for the table and chairs in one conference room it kept in case the company got its meeting with FDA staff.
Last week — after the meeting and the breakthrough happened — the company said it would be sold. Kezar hopes the buyer, Aurinia Pharmaceuticals, will take the drug forward, though how quickly that can happen, if at all, is not guaranteed.
It’s also not clear why Kezar’s initial meeting was canceled. But to CEO Chris Kirk, the chain of events fits a pattern over the past year in which volatility at the FDA — including staff departures and decision-making seen as inconsistent — has ricocheted across the industry, impacting drugmakers. Those impacts can fall disproportionately on small companies, which, unlike major drugmakers, often operate on one financing to the next.
“In my career, I’ve often not agreed with what the FDA has said, but I’ve at least relied on their consistency,” said Kirk, who’s worked in biotech for more than two decades. “That doesn’t appear to be what’s happening now. It feels more stochastic and maybe even capricious, what’s going on at the FDA. And this isn’t good for patients. It’s definitely not good for the biotech ecosystem as a whole.”
Background: Rates of opioid use disorder (OUD) have increased among women over the past 2 decades. Medication treatment for opioid use disorder (MOUD) is effective but underused. Gender-specific treatments for women have been associated with improved substance use outcomes. However, these treatments have not specifically targeted women’s engagement in MOUD, and the impact of existing gender-specific treatments is restricted by in-person delivery. Objective: The aim of this study was to develop a digital intervention to feasibly deliver gender-specific care that addresses the individualized needs of women with OUD to increase engagement in MOUD. Methods: A mixed methods, user-centered design approach was used to inform the development of a digital intervention. In phase 1, qualitative interviews were conducted with women with lived experience of OUD (n=20) and providers who treat women with OUD (n=8). Interviews were recorded, transcribed, and coded for themes. In addition, a larger sample of treatment providers (n=55) completed an online survey to further inform the content of the digital intervention. Phase 2 consisted of designing, beta-testing (n=5), and refining the intervention. Results: The age of women with lived experience ranged from 21 to 59 (mean 38.5, SD 9.4) years; 63% (5/8) of providers interviewed were female participants. The qualitative interview data from women with lived experience and providers were grouped into 6 thematic categories: 3 treatment-related (1) barriers to treatment, (2) facilitators to successful recovery, and (3) important issues to address in treatment, and 3 technology-related (4) positives of using technology as part of treatment, (5) suggested technology features, and (6) barriers to using technology. Across the treatment-related categories, several themes touched on women-specific factors including family responsibilities, abusive partners, stigma, and motivation for treatment (eg, pregnancy). The technology-related categories provided information for designing the features of the intervention, as well as revealing barriers to technology use, which could be helpful in developing implementation strategies. Provider survey participants were primarily female participants (40/55, 73%), with a mean age of 42.5 (SD 12.5) years. Survey data provided additional information on barriers to treatment and suggested technology features. Based on these data and preliminary work, the intervention was created. Minor edits to content and visual design were made in the beta-testing phase. The final version includes a web-based component with 6 topic modules and a mobile component. Topics in the web-based component are presented through infographics, text, videos, and interactive questions. The mobile component includes daily motivational messages, skills practice activities (2/wk), weekly check-ins, and resources (always available). Conclusions: Important themes and suggested features from women with lived experience and providers were incorporated into a digital intervention for women with OUD. Data on feasibility, satisfaction, and engagement with the intervention are currently being collected in phase 3, a pilot randomized controlled trial.
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