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Today, we talk lots about clinical trial inconsistencies: How Canada’s trial reporting is not up to snuff, and how companies perpetually want to spin results to make their experimental medicine look more favorable. We also talk about how the ARIA stemming from Alzheimer’s drugs isn’t taken seriously enough, and how state and local governments are suing insulin makers over drug prices.
The need-to-know this morning
• Belgian drugmaker Argenx said its antDesmoid tumor approvalibody medicine failed to achieve the goals of a late-stage study for a platelet-destroying autoimmune disorder — a setback in the company’s efforts to expand the drug’s use.
• A similar antibody for autoimmune diseases being developed by Immunovant, an offshoot of Roivant Sciences, continued to show promising results in an update from a study of healthy volunteers.
• Avidity Biosciences expanded its cardiovascular research partnership with Bristol Myers Squibb. Avidity will receive $100 million in upfront payments from Bristol for licensing rights to five cardiovascular drug targets.
• Novocure, maker of a medical device to treat brain cancer, announced a strategic restructuring to reduce overall costs, including layoffs affecting 13% of its workforce.
Alzheimer’s drugmakers need more ARIA transparency
The new wave of “breakthrough” Alzheimer’s drugs are becoming increasingly accessible to patients, but they all could potentially trigger brain swelling and bleeds. Manufacturers need to be more candid about the risks of amyloid-related imaging abnormalities, or ARIA, opines Madhav Thambisetty, a neurology researcher at John Hopkins University who helped evaluate the controversial aducanumab three years back.
“I am especially worried about about drugmakers’ lack of transparency in fully reporting all details of clinical outcomes related to ARIA,” he writes.
Rates of brain swelling or bleeding from ARIA has ranged in trials from 12% to 35%, compared to 3% to 13% in those receiving placebo. But the studies frame these complications as “mostly mild or even asymptomatic… and often resolving spontaneously,” he writes. But this ignores the reality that about 1% to 2% of patients have more serious effects, such as headaches, seizures, delirium, impaired speech, problems with vision, and muscle weakness.
An updated primer on interpreting clinical trials
Clinical trial data can be quite difficult to untangle, particularly when results can be spun — statistically or otherwise — to look better than they actually are. STAT has published an update to its Guide to Interpreting Clinical Trial Results to help readers parse data and come to reasonable conclusions. The new version contains “all the lesson and pithy advice” of the original report, which was written by STAT’s Adam Feuerstein and the late, great Sharon Begley.
“Spin is eternal,” Adam writes. “Also as long as companies (and scientists) conduct clinical trials, there will be efforts made to bend negative results in a more favorable light… I’m more amazed at how often companies stick to the standard spin playbook in the mistaken belief that, maybe, this time, it will work. It doesn’t.”
FDA approves a new treatment for desmoid tumors
The Food and Drug Administration on Monday approved the first treatment for people with desmoid tumors, a localized cancer that invades soft tissue and muscle. The new drug, called Ogsiveo, is made by Springworks Therapeutics. It’s the company’s first approved medicine.
Desmoid tumors are fibrous growths that start in connective tissue and are often found in the abdomen, arms, legs, and torso. The tumors do not spread to other parts of the body but can cause severe disability and pain in the tissues that they impact, like muscle or nerves. There are no currently approved therapies for desmoid tumors, but doctors often treat patients with chemotherapy, off-label targeted therapies, radiation, and surgery.
Canada has a clinical trial transparency problem
Only 3% of the 3,700 clinical trials conducted exclusively in Canada over the course of 10 years registered prospectively, reported results, and published their findings, a new study shows. Only 48% of the 3,700 trials published their findings, and 12% reported results in a registry. This underscores how clinical trial transparency is an ongoing problem in Canada.
Disclosure was much higher, however, when trials were conducted simultaneously in Canada and at least one other country. By contrast, 75% of these studies reported results in a registry, and 65% published their data. The analysis found the odds of adhering to transparency standards in Canada dropped by 95% when compared to international trials.
“This is a critical issue, as Canada is falling short of its international commitments when it comes to registration and reporting of clinical trials,” the study’s author told STAT. “This shortfall potentially introduces bias into the evidence that healthcare professionals rely on develop new medical guidelines.”
State and local governments suing insulin makers
Local and state governments are suing both insulin makers and PBMs, claiming collusion in driving up prices. Municipalities in New York, Virginia, Maryland, and Ohio, as well as the state of Utah, have filed lawsuits alleging that companies artificially drove up the cost of insulin, making it unaffordable to patients and causing the government to overpay for the medicines.
“We’re trying to use any tactic we can to reduce and recover our costs,” one attorney for a New York county told STAT.
Novo Nordisk, one of the companies targeted, said it “believes that the allegations in the lawsuit are without merit, and we intend to vigorously defend against these claims.”
• GSK’s blood cancer drug meets primary goal in late-stage trial, Reuters
• Roivant ends longshot lupus program after phase 2 fail, FierceBiotech
• Arvinas gets $350 million boost from investors to extend runway into 2027, Endpoints