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Good morning, extremely thrilling information from the STAT newsroom — two of our reporters, Casey Ross and Bob Herman (or as we name them, Bob Ross), have been named Pulitzer finalists in investigative reporting!
Their sequence uncovered how the usage of AI by Medicare Benefit plans lower off crucial look after seniors. You’ll be able to learn the tales right here: Half 1, Half 2, Half 3, Half 4.
Vertex begins gene remedy Casgevy in 5 sufferers
From STAT’s Jonathan Wosen: Vertex mentioned in its earnings name yesterday that as of mid-April, it’s collected cells from 5 sufferers slated to obtain Casgevy, its therapy for sickle cell illness and beta thalassemia that it makes with CRISPR Therapeutics. The replace on Casgevy, the first-ever permitted CRISPR-based remedy, comes shortly after news reports that Bluebird Bio, which sells a distinct sickle cell gene remedy, started gathering cells for its first affected person final week.
The 5 sufferers span areas the place Casgevy is at present permitted — the U.S., Europe, and the Center East — however CEO Reshma Kewalramani declined to offer an in depth breakdown of the place every affected person was situated, what situation they’d, and the place they’re within the grueling, months-long course of that precedes and follows infusion of the $2.2 million therapy.
Kewalramani additionally reiterated that she sees blockbuster potential in Vertex’s ache program, together with suzetrigine, a non-opioid ache drug that’s beneath FDA overview. The corporate is already speaking with insurers, pharmacists, and pharmacy profit managers in anticipation of the business rollout. COO Stuart Arbuckle mentioned Vertex first plans to focus on sufferers who’ve been discharged from well being care establishments, a gaggle that accounts for 35% of acute ache prescriptions.
The corporate’s total income steering stays unchanged, with Vertex anticipating between $10.55 billion to $10.75 billion this 12 months, a lot of which might be pushed by gross sales of its cystic fibrosis drug Trikafta, which introduced in $2.48 billion throughout the first quarter.
One other massive financing spherical in immunology
From STAT’s Allison DeAngelis: Traders are clamoring for irritation and immunology corporations — simply have a look at the mega-rounds that enterprise capitalists have raised this 12 months for biotechs creating psoriasis therapies (Displays A, B, and C). At this time, buyers at SR One, New Enterprise Associates, Norwest Enterprise Companions, and Delos Capital led a $200 million Collection C for one more immune illness firm known as Zenas BioPharma.
Zenas isn’t targeted on psoriasis, although. The corporate’s lead drug, obexelimab, is being examined in a Part 3 trial for IgG4-related illness — a situation that causes lesions or lots in a number of organs — and in a number of Part 2 trials for a number of sclerosis, lupus, and a type of anemia. The monoclonal antibody targets each 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 different areas to Bristol Myers Squibb.
A definite genetic type of Alzheimer’s?
The APOE4 variant has lengthy been regarded as a threat issue for Alzheimer’s, however a brand new research argues that this variant performs an much more necessary position than scientists had realized and truly causes a definite type of the illness.
Analyzing information from greater than 13,000 individuals, researchers discovered that amongst APOE4 homozygotes, individuals who carry two copies of the gene variant, practically all confirmed organic indicators of Alzheimer’s within the mind. In addition they usually developed dementia and different signs, and died sooner, than individuals with Alzheimer’s who lack the variant.
The authors of this research known as for renewed curiosity in two targets which have lengthy eluded scientists: creating efficient therapies that immediately goal APOE4, and higher understanding how the protein derived from this variant is concerned in illness.
Learn extra from Jonathan Wosen on the implications of those findings on Alzheimer’s drug improvement.
Indicators of inequitable Leqembi distribution
A new study on real-world prescriptions of Leqembi, the Alzheimer’s drug from Eisai and Biogen, suggests there are already stark disparities within the rollout of the brand new therapy.
The evaluation, performed by well being analytics Truvteta and offered on the ISPOR convention, appeared on the interval from January 2023 (when the drug acquired conditional approval) to February of this 12 months.
Researchers discovered that 1.5% of people that obtained Leqembi have been Black or African American, in contrast with 10.4% of the general Alzheimer’s inhabitants. Moreover, 1.7% of sufferers who acquired Leqembi have been Hispanic or Latino, in contrast with 6.5% of the broad Alzheimer’s inhabitants.
There are limitations to the research: The race of 14.7% of the Leqembi sufferers and the ethnicity of 16.9% have been unknown, however, nonetheless, the findings level to a pattern to be careful for.
Black persons are twice as possible, and Hispanic persons are 1.5 instances extra possible, than white individuals to have Alzheimer’s or different dementias. And there’s been concern that as new medication come to market, inequitable entry to the therapies may additional widen disparities.
Extra reads
- MDMA-based psychological well being therapy faces cautious US regulator, Financial Times
- Gossamer sells rights to drug it hopes can rival a brand new Merck remedy, BioPharma Dive
- Opinion: The world is counting on the US to get value-based drug pricing proper, STAT