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Vertex starts gene therapy Casgevy in five patients 

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Vertex starts gene therapy Casgevy in five patients  

From STAT’s Jonathan Wosen: Vertex said in its earnings call yesterday that as of mid-April, it’s collected cells from five patients slated to receive Casgevy, its treatment for sickle cell disease and beta thalassemia that it makes with CRISPR Therapeutics. The update on Casgevy, the first-ever approved CRISPR-based therapy, comes shortly after news reports that Bluebird Bio, which sells a different sickle cell gene therapy, began collecting cells for its first patient last week.

The five patients span regions where Casgevy is currently approved — the U.S., Europe, and the Middle East — but CEO Reshma Kewalramani declined to provide a detailed breakdown of where each patient was located, what condition they had, and where they are in the grueling, months-long process that precedes and follows infusion of the $2.2 million treatment.


Kewalramani also reiterated that she sees blockbuster potential in Vertex’s pain program, including suzetrigine, a non-opioid pain drug that’s under FDA review. The company is already talking with insurers, pharmacists, and pharmacy benefit managers in anticipation of the commercial rollout. COO Stuart Arbuckle said Vertex first plans to target patients who’ve been discharged from health care institutions, a group that accounts for 35% of acute pain prescriptions.

The company’s overall revenue guidance remains unchanged, with Vertex expecting between $10.55 billion to $10.75 billion this year, much of which will be driven by sales of its cystic fibrosis drug Trikafta, which brought in $2.48 billion during the first quarter.

Another big financing round in immunology

From STAT’s Allison DeAngelis: Investors are clamoring for inflammation and immunology companies — just look at the mega-rounds that venture capitalists have raised this year for biotechs developing psoriasis treatments (Exhibits AB, and C). Today, investors at SR One, New Enterprise Associates, Norwest Venture Partners, and Delos Capital led a $200 million Series C for another immune disease company called Zenas BioPharma.

Zenas isn’t focused on psoriasis, though. The company’s lead drug, obexelimab, is being tested in a Phase 3 trial for IgG4-related disease  — a condition that causes lesions or masses in multiple organs — and in multiple Phase 2 trials for multiple sclerosis, lupus, and a form of anemia. The monoclonal antibody targets both CD19 and Fc gamma RII.

Zenas acquired the drug from Xencor in 2021 and has already sold the rights to commercialize the drug in Japan, South Korea, Australia, and other locations to Bristol Myers Squibb.

A distinct genetic form of Alzheimer’s?

The APOE4 variant has long been thought of as a risk factor for Alzheimer’s, but a new study argues that this variant plays an even more important role than scientists had realized and actually causes a distinct form of the disease.

Analyzing data from more than 13,000 people, researchers found that among APOE4 homozygotes, people who carry two copies of the gene variant, nearly all showed biological signs of Alzheimer’s in the brain. They also typically developed dementia and other symptoms, and died sooner, than people with Alzheimer’s who lack the variant.

The authors of this study called for renewed interest in two goals that have long eluded scientists: developing effective therapies that directly target APOE4, and better understanding how the protein derived from this variant is involved in disease.

Read more from Jonathan Wosen on the implications of these findings on Alzheimer’s drug development.

Signs of inequitable Leqembi distribution

new study on real-world prescriptions of Leqembi, the Alzheimer’s drug from Eisai and Biogen, suggests there are already stark disparities in the rollout of the new treatment.

The analysis, conducted by health analytics Truvteta and presented at the ISPOR conference, looked at the period from January 2023 (when the drug got conditional approval) to February of this year.

Researchers found that 1.5% of people who received Leqembi were Black or African American, compared with 10.4% of the overall Alzheimer’s population. Additionally, 1.7% of patients who got Leqembi were Hispanic or Latino, compared with 6.5% of the broad Alzheimer’s population.

There are limitations to the study: The race of 14.7% of the Leqembi patients and the ethnicity of 16.9% were unknown, but, still, the findings point to a trend to watch out for.

Black people are twice as likely, and Hispanic people are 1.5 times more likely, than white people to have Alzheimer’s or other dementias. And there’s been concern that as new drugs come to market, inequitable access to the treatments could further widen disparities.

More reads

  • MDMA-based mental health treatment faces wary US regulator, Financial Times
  • Gossamer sells rights to drug it hopes can rival a new Merck therapy, BioPharma Dive
  • Opinion: The world is relying on the United States to get value-based drug pricing right, STAT