Pfizer’s Lorbrena, Ultragenyx, Ionis, Sarepta

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Nowadays we see that gene therapy is succeeding in helping patients with a rare liver disease maintain their blood sugar levels. We see an Ionis drug doing better than already approved competitors in a rare swelling disease, and we see some conflicts of interest from doctors on X.

ASCO also starts today! If you haven’t already, sign up for our flash ASCO newsletter here. STAT’s Matt Herper, Adam Feuerstein, Angus Chen and Elaine Chen will report from McCormick Place in Chicago.

The must-know this morning

• Advisors to the European Medicines Agency recommended the approval of a gene therapy for hemophilia B from Pfizer.
• Gilead Sciences says its cancer drug Trodelvy failed to improve survival in patients with advanced metastatic bladder cancer. The phase 3 TROPiCS-04 study aimed to confirm the benefit of Trodelvy in bladder cancer patients. The drug received accelerated approval in 2021.

Pfizer’s Lorbrena reduces progression of rare lung cancer by 81%

New data presented at ASCO on the long-approved Pfizer drug Lorbrena is remarkable: The daily pill reduced disease progression in non-small cell lung cancer by 81% over five years. That means that 60% of patients taking the drug at that time were alive and did not see their tumors grow, compared to 8% who received Xalkori, another cancer drug from Pfizer.

Lorbrena was approved in 2001 for non-small cell lung cancer. It’s not exactly a top seller; last year it grossed $575 million, accounting for just 1% of the drug giant’s annual revenue. So it will be interesting if the updated data will boost sales of both Pfizer drugs.

Read more.

Ultragenyx gene therapy succeeds in late-stage research

A gene therapy developed by Ultragenyx for a rare liver disease was successful in a Phase 3 trial, paving the way for possible approval. The condition, called glycogen disease type 1A, is caused by a genetic mutation that prevents patients from maintaining blood sugar levels. Although once fatal, it is now managed with regular doses of cornstarch, but it is crucial that patients do not miss even a single dose.

A study of 49 patients found that those who received Ultragenyx therapy were able to consume 41% less cornstarch than at the start of the study. By comparison, patients in the placebo arm took only 10% less. While statistically significant, the results are less profound than a previous study, which saw a 72% decrease in patient needs. The gene therapy also failed on a secondary endpoint.

Read more.

The Ionis drug outperforms other angioedema medications on the market

Donidalorsen, an RNA-targeted treatment for hereditary angioedema made by Ionis Pharmaceuticals, has helped improve patients’ symptoms in a few late-stage studies — even for those taking medications for the disease that have already been approved. After 25 weeks, patients who received the drug monthly had 81% fewer attacks of severe swelling compared to placebo; when patients received the drug every two months, rates were still down 55%.

Another study found that as time went on, donidalorsen’s benefits increased: swelling attacks decreased by 92% to 93%, regardless of how often patients were dosed. Interestingly, patients who switched to donidalorsen from existing therapies from competitors like Takeda and BioCryst Pharmaceuticals saw their swelling attack rates drop by 62% compared to their previous treatment.

Ionis will use this data to apply for FDA approval by the end of the year. Otsuka, which worked with Ionis for European rights to the drug, will also apply for approval there this year.

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An ASCO preview and another failed Duchenne test

Which presentations are worth seeing at ASCO this weekend? What is exon skipping? And how do you pronounce bronchiectasis?

This week on ‘The Readout LOUD’ we preview some of the research that will be presented at the American Society of Clinical Oncology conference in Chicago. We also discuss the latest news in health and life sciences, including a milestone in lung disease research and development and a failed trial to confirm Duchenne muscular dystrophy.

Listen.

Doctors on X who approved drugs were paid by the manufacturers

Many doctors weigh in on X – formerly Twitter – and often endorse the prescription medications they rely on most. But a new study finds it’s likely that many have been paid at some point by the makers of these drugs.

The JAMA analysis found that 28 physicians were followed on X and 26 physicians received at least one payment. And the average was more than $27,400 for things like food and drinks, speaking, consulting or travel. In 24 cases, the payments were linked to a drug or device that the doctors actively endorsed on social media.

Interestingly, two-thirds of the physicians who made these recommendations did not score highly on an index used to measure academic productivity, writes STAT’s Ed Silverman. Nearly half of physicians did not disclose compensation from manufacturers. The study authors are concerned that “the conflict of interest… may not be apparent to the general social media audience” because financial ties “were not consistently disclosed in posts.”

Read more.

Read more

• Did Sarepta Therapeutics make $4 billion on exon-skipping drugs that don’t work? STAT
• Summit shares rise after cancer drug surpasses Merck’s Keytruda Bloomberg
• Wegovy maker Novo Nordisk sues nine spas, clinics and pharmacies over counterfeit drugs, Reuters
• ADHD game developer Akili Interactive is being taken private after a $34 million sale FierceBiotech