Good morning, everyone, and welcome to the middle of the week. Congratulations on making it this far, and remember there are only a few more days until the weekend arrives. So keep plugging away. After all, what are the alternatives? While you ponder the possibilities, we invite you to join us for a needed cuppa stimulation. Our choice today is ginseng peach, a favorite from our pantry. Meanwhile, here is the latest menu of tidbits to help you on your way. We hope you conquer the world and have a wonderful day. And as always, please do stay in touch. We appreciate feedback, tips and criticism. …

In a pair of late-stage trials, an AstraZeneca drug reduced the dangerous flare-ups that worsen COPD, positioning it as a potential new option for the vast number of patients with the debilitating lung condition, STAT explains. According to data released Tuesday, the results also amount to a re-assuring victory for AstraZeneca, which is known for its metronomic success in trials but has seen some notable misses in recent months, most dramatically with a heart disease drug. Analysts and the company have projected that the injectable drug could reap billions in annual sales if approved, both in COPD and in other conditions including asthma. 

The Trump administration permanently filled four key leadership roles at the U.S. Food and Drug Administration on Tuesday, after nominating Heidi Overton to lead the agency late last month, STAT says. Three of the officials are serving in an acting capacity, and will take the same roles permanently. They are Michael Davis, a psychiatrist who will be director of the Center for Drug Evaluation and Research; former Merck official Karim Mikhail, who will be director of the Center for Biologics Evaluation and Research; and Bret Koplow, an attorney who has been at the FDA since 2011 and will serve as director of the Center for Tobacco Products. 

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Residue and stains plastered on production walls, floors, and manufacturing equipment. Corrosion on a product line. An employee wearing open-toed sandals.

These were among the unsanitary conditions found last April by a Food and Drug Administration inspector during a visit to Shoolin Pharma in Gujarat, India, where the company makes active pharmaceutical ingredients for more than a dozen medicines, including erectile dysfunction and anti-convulsant pills, that are sold to compounding pharmacies in the U.S., according to the agency.

The concerns about filth and possible contamination in the production and packaging areas — as well as incomplete laboratory testing records — prompted the agency last month to halt all product shipments by the company into the U.S., the FDA disclosed in an Aug. 18 warning letter that was posted on the FDA web site last week.

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Good morning, everyone, and welcome to another working week. We hope the weekend respite — longer than usual thanks to a holiday on this side of the pond — was relaxing and invigorating. Now, though, 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 a cup of stimulation. Our choice today is butter pecan. Feel free to join us. Remember, no prescription is required. Meanwhile, here are a few items of interest. Best of luck accomplishing your goals, and we hope you conquer the world. And, of course, do keep in touch. …

An experimental Novartis drug for a rare neuromuscular condition failed to improve muscle function in a pivotal study, its second major trial collapse in a matter of days, STAT writes. The two study flops will likely stoke concerns about its pipeline and acquisition strategy as it navigates a major patent cliff, though the company did see a victory with a multiple sclerosis drug last week. The stock was down by as much as 10% in early trading in Switzerland. The Phase 3 HARBOR trial was testing the drug del-desiran in a condition called myotonic dystrophy type 1, known as DM1, which causes progressive muscle stiffness and weakness.

Novo Nordisk stopped two trials of a drug that aims to tame inflammation as a way to improve cardiovascular health, another blow for the approach, STAT notes. The company said it had halted the HERMES and ATHENA trials of its drug, ziltivekimab, after a data monitoring committee found they were unlikely to succeed. Novo informed trial investigators on Friday. The move follows the earlier failure of the drug in the ZEUS trial, which showed that the medicine, despite lowering markers of inflammation, did not reduce the risk of major clinical complications, including deaths and heart attacks.

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Rise and shine, everyone, another busy day is on the way. And it is getting off to a good start here on the Pharmalot campus, where we have clear blue skies and chilly breezes greeting us today. Who could ask for anything more? Actually, we could — it is time to reheat the coffee kettle for another cup of stimulation. Our choice today is strawberry creme, a pantry favorite. Please feel free to join us. Remember, no prescription is required and no rebates are involved. And now, here are your tidbits. Hope you have a productive and meaningful day, and, as always, please do keep in touch. We have adjusted our settings to accept postcards and telegrams. …

Eli Lilly reported that a high dose of its gene-editing therapy reduced cholesterol levels by 62% in participants in a Phase 1 clinical trial, an early but encouraging test of whether a one-time treatment may one day help people seeking to lower their LDL, or “bad,” cholesterol, STAT writes. Lilly acquired the therapy in its $1 billion buyout of Verve Therapeutics last year. There were no treatment-related serious adverse events in the study, a notable finding given that Verve had to shelve its first candidate over safety concerns. Lilly now plans to begin a Phase 2 trial in an unspecified number of patients and will likely need to then run Phase 3 trials in thousands of patients for the treatment to earn approval. 

Eli Lilly agreed to buy three early- and mid-stage vaccine developers in deals that could cost up to nearly $4 billion, STAT notes. The drugmaker, which is rich with cash from its booming GLP-1 business, in recent months has picked up companies working on cancer, autoimmune diseases, and sleep disorders, all for under $10 billion each. The new acquisitions — Curevo, LimmaTech Biologics, and Vaccine Company — will give Lilly a much larger footprint in infectious diseases, an area that has not recently been a priority for the company. Lilly framed the move as one that fit with a broader focus on preventing serious health problems.

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And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is unusually busy thanks to a lengthy to-do list that must be tackled before we walk one of the no-longer-so-short people down the aisle. What else? Hard to keep track, but once the chaos subsides, we hope to settle in for another listening party, where the rotation will likely include this, this, this, this and this. And what about you? Spring is in the air, so perhaps this is time to hike a trail, stroll through a park, or take a long drive to nowhere. You could also plan a summer getaway (book now before those fuel costs rise again) or clear out the perennial clutter. If all this is too much or the weather fails to cooperate, you could simply go into zen mode and plan the rest of your life. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you on Tuesday, since there is a long weekend due to a holiday on this side of the pond. …

Biogen and Denali Therapeutics said Thursday that their experimental therapy for Parkinson’s disease failed to slow the degenerative brain disorder in a randomized trial, dealing a substantial blow to a scientific approach that stoked excitement among advocates and academics, STAT explains. In the study, 648 adults with Parkinson’s were randomized to receive either a placebo or a pill targeting a protein called LRRK2. In 2004, researchers discovered that mutations in the LRRK2 gene can cause a rare, inherited form of Parkinson’s. And in 2018, another group of scientists showed that blocking the protein might actually benefit all patients with the disease. The results are a significant setback to the latter idea.

Earlier this month, Genentech offered countless academics and other researchers up to $125,000 in grants to generate papers about several topics that read like key talking points for a trip to Capitol Hill, STAT reports. The company is seeking “rigorous, independent” work that focuses on the potential consequences of U.S. pricing policies on future innovation, the idea that pharmaceutical discovery is a strategic national asset, and the risks surrounding R&D, according to a request for proposals that came with a June 30 deadline for submissions. Such overtures are hardly new, but this particular solicitation appears notable partly because the company is being very direct about seeking research that is designed to address specific points, rather than solicit topics that may — or may not — dovetail with corporate goals.

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And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda includes promenading with the official mascots, escorting Mrs. Pharmalot to a musical happening and visiting a new eatery. We also hope to have yet another listening party, where the rotation will include this, this, this, this and this. And what about you? This may be an opportunity to enjoy the great outdoors, perhaps a stroll by the seashore or a walk in the woods. A long drive in the country may be nice, although it will cost you. Or perhaps simple pleasures such as noshing on a pastry with a hot cup of stimulation will do. You could also catch up on your reading or reach out to someone special. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon…

A flood of cheap copies of Novo Nordisk’s blockbuster weight-loss drug in India is already reshaping the country’s fast-growing obesity medicine market, showing how quickly the patent cliff will affect GLP-1 makers like Eli Lilly, Bloomberg News explains. Within days of dozens of generics hitting the Indian market after Novo’s patent expired locally, the drug’s share in the country’s GLP-1 segment jumped to 33% in March from 25% a month earlier, according to researcher Pharmarack. That gain came at the expense of Lilly’s Mounjaro, which is still under patent protection in India and whose share fell to 64% from 71%. The data marks the first meaningful snapshot of how market dynamics will change for GLP-1 makers as their patents near expiry.

The U.S. Food and Drug Administration withdrew approval of a GSK drug that the Trump administration had promoted as a treatment for autism, adding another twist to the unusual story of a decades-old drug, Bloomberg News informs us. The agency is pulling its approval of Wellcovorin, a branded version of leucovorin from GSK, according to a post in the Federal Register. GSK had requested that the approval be withdrawn. The FDA first approved leucovorin decades ago and it has been used to blunt the side effects of chemotherapy for some cancer patients. In September, Trump administration officials endorsed leucovorin as an autism treatment. Last month, the FDA approved its use for cerebral folate deficiency, which is seen in some people with autism.

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Top of the morning to you, and a fine one it is. Sunny skies and mild breezes are enveloping the Pharmalot campus once again. And to celebrate, we are brewing still more cups of stimulation and inviting you to join us. Our choice today is Jack Daniel’s. Yes, this is a real thing. And remember, a prescription is not required. So no need to mess with rebates, coupons, or TrumpRx. Meanwhile, here are a few items of interest. Hope you have a smashing day and conquer the world. And of course, do stay in touch. We appreciate feedback, criticism and tips. …

Steve Ubl, the chief executive of Pharmaceutical Research and Manufacturers of ​America, plans to step down at the end of the ‌year, after more than a decade of leading the main trade group for brand-name drug makers, STAT notes. Ubl led the organization during tumultuous times that included the Covid-19 pandemic and aggressive political attacks on prescription drug pricing. Democrats passed a law directing Medicare to negotiate drug prices and the Trump administration struck  voluntary deals with individual drugmakers aimed at lowering U.S. prices to levels in other high-income countries. The next head of PhRMA will face increasing political pressure on prices and an increasingly populist mood. 

Specific changes in two genes appear to help predict whether patients will lose substantial weight on GLP-1 drugs used to treat obesity — and whether the drugs will cause nausea or vomiting, which are some of their most common side effects, STAT writes, citing a paper in Nature. “I think we have proof of concept here that genetics is playing a role in terms of GLP-1 efficacy and side effects,” said Adam Auton, a vice president at the 23andMe Research Institute and the senior author on the paper. Outside researchers were impressed and intrigued by the findings, but some doubted whether the genetic results would impact patient care. Still, consumers who use what 23andMe calls its Total Health platform will have access to information about these genes and what they predict about GLP-1 use.

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Rise and shine, everyone, another busy day is on the way. And it is getting off to a good start here on the Pharmalot campus, where clear blue skies and comfortable breezes are greeting us. Who could ask for anything more? Actually, we could — it is time to reheat the kettle for another cuppa stimulation. Our choice today is ginger peach. And here is a helpful tip — a teaspoon of honey enhances the flavors splendidly. Of course, you are invited to join us. For the full experience, we are now hawking replicas — take a look. Meanwhile, here are a few tidbits to help you along. As always, do keep in touch. We appreciate feedback, criticism, and tips. …

The U.S. Food and Drug Administration used the president’s budget to propose policies aimed at encouraging domestic development and manufacturing of drugs, STAT notes. FDA Commissioner Marty Makary has said the agency needs “giant, big ideas” to counter China’s dominance in early-stage clinical development of drugs. Among the FDA’s ideas are proposals to make it easier to run early-stage trials in the U.S. and to hand an advantage to U.S.-based generics manufacturers. The Trump administration has been using a variety of policy levers to try and bring drug manufacturing to the U.S. One of the legislative proposals in the FDA’s budget justification would let domestic manufacturers of generic drugs challenge brand drug patents a month before foreign companies, a major advantage in an intensely competitive process. 

Two more drugmakers, AbbVie and Genentech, will officially start selling their medicines on the TrumpRx website, CBS News tells us. Abbvie, which struck a deal with the Trump administration in January to cut the cost of certain medicines, will sell Humira, a popular medication used to treat rheumatoid arthritis, Crohn’s disease, and ulcerative colitis, on the site at an 86% discount. The prescription prices on the site, however, are only available to patients who are uninsured, or whose insurance does not cover it, and who must pay the full list price out of pocket. Those with insurance coverage generally pay lower prices already. TrumpRx now sells over 61 drugs at a lower price, up from about 40 when the website went live in February. 

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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.

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For the second time in two years, a bill is moving through the Colorado legislature that would exempt orphan drugs, which are used to treat rare diseases, from pricing caps that might be pursued by the state’s Prescription Drug Affordability Board — a panel whose work is being closely watched elsewhere in the country.

The effort reflects concerns that patients may lose access to these drugs if pharmaceutical companies halt sales of such treatments in the state. But opponents argue exemptions would unnecessarily extend to numerous big-selling medicines for common conditions that — thanks to regulatory endorsements — also happen to have an orphan designation.

As a result, consumer advocates complain the maneuver would only increase the risk that countless patients could have trouble paying for a wide variety of medicines. They further argue that the legislation would preserve profits for drug companies at the expense of the state government — and its taxpayers — as it tries to cope with budgetary strains.

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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. 

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