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Good morning, everybody. Damian right here with some breathlessly anticipated information from Vertex Prescription drugs, an exit interview from an FDA lifer, and the debut of the $100 genome.
The necessity-to-know this morning
- Pfizer reported fourth-quarter and 2023 earnings, and reaffirmed 2024 steering.
- Kura Oncology reported no cases of differentiation syndrome, a intently tracked aspect impact, in a brand new research that mixes its experimental menin inhibitor, known as ziftomenib, with chemotherapy in sufferers with acute myeloid leukemia. Excessive charges of differentiation syndrome reported in earlier ziftomenib monotherapy research had been a priority.
- Regeneron Prescription drugs is spending $5 million to acquire 2Seventy Bio’s pipeline of cell therapies for most cancers and different illnesses. A smaller 2Seventy will carry on, supporting the advertising and marketing and additional improvement of Abecma, a CAR-T for a number of myeloma partnered with Bristol Myers Squibb.
- Gilead Sciences increased its ownership stake in Arcus Biosciences. The 2 corporations are collectively growing an anti-TIGIT antibody for most cancers.
Vertex’s ache drug hits in Part 3
A novel ache medication from Vertex Prescription drugs met its main targets in three Part 3 research, setting the stage for an FDA utility that might dramatically change the corporate’s fortunes.
As STAT’s Jonathan Wosen reviews, Vertex’s drug, VX-548, considerably outperformed placebo in research enrolling sufferers with acute ache following surgical procedure. The drug missed its secondary objective of proving superior to a mix of acetaminophen and hydrocodone, a generally prescribed opioid.
Vertex will submit the outcomes, amongst 2024’s most hotly anticipated readouts, to the FDA by the center of this yr, the corporate stated. If the whole lot goes to plan, Vertex’s oral, non-addictive ache medication might change into a multibillion-dollar product, analysts have stated, reworking the corporate within the course of.
How an immunological Computer virus might deal with illness
For thousands and thousands of sufferers with autoimmune illnesses, the physique’s pure defenses errantly assault wholesome tissues. One startup’s plan to intervene includes disguising medicine as bodily trash and tricking the immune system into dragging that Computer virus to its organic targets.
As STAT’s Allison DeAngelis reviews, the corporate is named Cour Prescription drugs, and it simply raised a $105 million Sequence A spherical to flesh out its foundational concept. The know-how includes utilizing proprietary polymer nanoparticles to cloak molecules designed to sensitize the immune system. As soon as these nanoparticles attain the liver, the drug works just like the inverse of a vaccine, telling the physique to cease preventing in opposition to its personal cells.
Cour is already in Part 2 improvement with remedies for celiac illness and a uncommon liver dysfunction known as main biliary cholangitis. The corporate can also be at work on remedies for kind 1 diabetes and the neuromuscular illness myasthenia gravis.
The Janet Woodcock exit interview
After greater than three many years on the FDA, together with a prolonged stint as its strongest regulator of latest medicine, Janet Woodcock is retiring from the company and within the course of ending an period.
Woodcock spoke to STAT’s Sarah Owermohle for a wide-ranging exit interview, addressing the FDA’s usually tumultuous relationship with Congress, the company’s future in a altering society, and, after all, the continued controversy over which medicine deserve accelerated approval and which don’t.
“I have a tendency to have a look at outcomes,” Woodcock stated. “What have been the outcomes of that aggressive posture towards getting new most cancers therapies — significantly focused most cancers therapies — on the market to sufferers? Some individuals may say … there have been some that didn’t work out. Nicely, that’s how accelerated approval is about up. Is that mistaken? What’s the choice? You maintain again all of those till you will have definitive proof? Primarily based on the outcomes, you’d have lots of people who wouldn’t be alive. Was that price it to have all that certainty?”
The ‘darkish horse’ startup with a $100 genome
Greater than a yr after promising to roil the marketplace for sequencing with a $100 genome, the privately held Ultima Genomics is launching a line of devices meant to problem Illumina’s near-monopoly.
As STAT’s Jonathan Wosen reviews, the sequencer, known as the UG 100, will price $1.5 million and browse as much as 20,000 human genomes a yr. Factoring in the price of reagents, that works out to $100 per genome, based on Ultima, which plans to launch the sequencer at a scientific convention on Feb. 5.
The query for Ultima, and the entire lots of the companies attempting to unseat Illumina, is whether or not it will probably win over clients on the drug corporations, hospitals, and analysis establishments that deal with many of the world’s genomic sequencing. The corporate has raised greater than $600 million in enterprise funding since its inception, tapping a syndicate that features Andreessen Horowitz and Khosla Ventures.
- Can Wegovy deal with melancholy in addition to weight problems? New analysis seems to be to GLP-1 medicine for psychological sicknesses, STAT
- Pharma worth minimize proposals from US authorities might be steep, analysts say, Reuters
- European regulators tout their accomplishments in opposition to pharma’s anticompetitive practices, STAT
- Scientists doc first-ever transmitted Alzheimer’s instances, tied to no-longer-used medical process, STAT