Vertex, Leqembi, Eisai and Biogen

Want to stay up to date on the science and politics driving biotechnology today? Sign up to receive our biotech newsletter in your inbox.

Good morning, incredibly exciting news from the STAT newsroom – two of our reporters, Casey Ross and Bob Herman (or as we call them, Bob Ross), goods named Pulitzer finalists in investigative reporting!

Their series showed how Medicare Advantage plans’ use of AI was cutting off critical care for seniors. You can read the stories here: Part 1, Part 2, Part 3, Part 4.

Vertex starts gene therapy Casgevy in five patients

STAT’s Jonathan Wosen: Vertex said in its earnings call yesterday that as of mid-April it has collected cells from five patients who will receive Casgevy, the treatment for sickle cell disease and beta-thalassemia that it is making with CRISPR Therapeutics. Shortly afterwards will be the update on Casgevy, the very first approved CRISPR-based therapy news reports that Bluebird Bio, which sells another sickle cell gene therapy, began collecting cells for its first patient last week.

The five patients cover regions where Casgevy is currently approved – the US, Europe and the Middle East – but CEO Reshma Kewalramani declined to provide a detailed breakdown of where each patient was, what condition they were in and where they were in the grueling situation. A months-long process that precedes and follows the infusion of $2.2 million worth of treatment.

Kewalramani also reiterated that she sees blockbuster potential in Vertex’s pain program, which includes suzetrigine, a non-opioid pain drug under FDA review. The company is already in discussions with insurers, pharmacists and pharmacy benefit managers in anticipation of the commercial rollout. COO Stuart Arbuckle said Vertex wants to focus first on patients discharged from health care facilities, a group that accounts for 35% of prescriptions for acute pain.

The company’s overall revenue guidance remains unchanged, with Vertex expecting between $10.55 billion and $10.75 billion this year, much of which will be driven by sales of its cystic fibrosis drug Trikafta, which posted $2 in the first quarter .48 billion.

Another major funding round in immunology

From STAT’s Allison DeAngelis: Investors are clamoring for inflammatory disease and immunology companies – just look at the mega-rounds that venture capitalists have raised this year for biotech companies developing treatments for psoriasis (Exhibits A, B 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.

However, Zenas is not aimed at psoriasis. The company’s lead drug, obexelimab, is being tested in a Phase 3 trial for IgG4-related diseases — a condition that causes lesions or thickenings 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 purchased the drug from Xencor in 2021 and has already done so sold the rights to market the drug in Bristol Myers Squibb in Japan, South Korea, Australia and other locations.

A separate genetic form of Alzheimer’s?

The APOE4 variant has long been considered a risk factor for Alzheimer’s disease, but a new study suggests that this variant plays an even more important role than scientists 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, almost all showed biological signs of Alzheimer’s disease in the brain. They also typically developed dementia and other symptoms, and died earlier than people with Alzheimer’s disease who don’t have 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 diseases.

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

Signs of an unfair Leqembi distribution

a new study on the practice instructions of Leqembi, the Alzheimer’s drug from Eisai and Biogen, suggests that there are already major differences 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 received conditional approval) to February this year.

Researchers found that 1.5% of people who received Leqembi were black or African American, compared to 10.4% of the overall Alzheimer’s population. Additionally, 1.7% of patients receiving Leqembi were Hispanic or Latino, compared to 6.5% of the broader Alzheimer’s population.

There are limitations to the study: the race of 14.7% of Leqembi patients and the ethnicity of 16.9% were unknown, but still the findings indicate a trend we should pay attention to.

Black people are twice as likely, and Hispanic people are 1.5 times more likely than white people, to develop Alzheimer’s disease or other forms of dementia. And there are concerns that as new drugs come to market, unequal access to the treatments could widen the disparities even further.

Read more

• MDMA-based mental health care faces wary US regulator, Financial times
• Gossamer is selling rights to drugs he hopes will rival a new Merck therapy BioPharma Dive
• Opinion: The world is relying on the United States to get value-based drug pricing right, STAT