October 23, 2023

The need-to-know this morning

The "Roi" in Roivant Sciences stands for "return on investment" and with a blockbuster deal announced Monday, the biotech certainly delivered. Last December, it essentially acquired an experimental drug for inflammatory bowel diseases from Pfizer for $45 million and a commitment to invest in the drug's development. Less than a year later, Roivant is selling the drug, called RVT-3101, to Roche for at least $7.1 billion.

CRISPR

FDA clears Verve for U.S. study of CRISPR treatment

Verve Therapeutics said Monday that it had received clearance from the Food and Drug Administration to conduct a clinical trial in the U.S. of its experimental, gene-editing treatment for a common form of heart disease.

The FDA's action removes a clinical hold on Verve's CRISPR-based therapy, called VERVE-101, that was placed on it last November. Verve now intends to recruit participants from U.S. sites into a Phase 1 study that's been underway since last year in New Zealand and the United Kingdom.

podcast

Roche drug effective for early ALK-positive lung cancer

New data shows that alectinib, a Roche cancer drug, could be helpful in treating earlier stages of ALK-positive lung cancer. The medicine reduced the risk of recurrence or death by 76% compared to chemotherapy for early forms of the disease, new Phase 3 presented at ESMO show. The drug is already approved for metastatic forms of ALK-positive cancers, which account for 4%-5% of all lung cancers.

"This is an absolutely amazing data set," said Solange Peters, a Swiss oncologist who once served as president of ESMO.

Alectinib, sold under the trade name Alecensa, works by inhibiting the protein that drives tumor growth in patients with ALK-positive lung cancer. The drug not only improved disease-free survival when compared to chemo, but it reduced the risk of brain metastasis.

achondroplasia

BioMarin's dwarfism drug gets expanded approval

The FDA just expanded the label for BioMarin's dwarfism drug. Now, the once-daily injection will be available to children under 5 with achondroplasia, Reuters writes. The drug, Voxzogo, achieved an accelerated approval in 2021 to treat the genetic disorder in children over 5. This expanded use is based on trial data that showed the drug was similarly safe and effective in younger children.

BioMarin said that Voxzogo brought in $169.2 million in sales last year. This year, sales are expected to hit $440 million. There continues to be a great deal of controversy around drugs that treat achondroplasia, Nature wrote in August. But companies like BridgeBio and Ascendis Pharma are developing their own assets to compete with BioMarin in the dwarfism space.

dengue fever

Antiviral pill for dengue shows early efficacy

Johnson & Johnson is developing an antiviral pill that could prevent dengue fever, Forbes writes. Early data presented at the American Society of Tropical Medicine & Hygiene meeting demonstrated JNJ-1802's efficacy in a challenge trial. The company gave participants either the drug or an inert placebo for 26 days, then injected them with a strain of the dengue virus.

Participants were monitored over 85 days for signs of infection, including immune response and virus levels in the blood. While all five people in the placebo group showed detectable levels of the virus, six out of 10 participants who received a high dose of the drug showed no signs of infection. The drug "is the first ever to show antiviral activity against dengue," one exec leading the drug's development told Reuters.

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