September 18, 2023

The need-to-know this morning

Black Diamond Therapeutics co-founder and CEO David Epstein resigned. Chairman Mark Velleca has been appointed CEO, effective immediately. Epstein will remain on Black Diamond's board.
Lonza CEO Pierre-Alain Ruffieux resigned. He will be replaced on an interim basis by Chairman Albert Baehny until a new CEO is found.
ImmunoGen named Lauren White as its new chief financial officer. She joined from C4 Therapeutics.
The FDA granted priority review to Libmeldy, a gene therapy from Orchard Therapeutics that treats children with metachromatic leukodystrophy, a rare and life-threatening genetic disease. The FDA decision deadline is March 18. The treatment is already approved in Europe.
An experimental blood-thinning medicine developed by privately held Anthos Therapeutics significantly reduced bleeding events compared to Xarelto, a currently approved anticoagulant, achieving the goals of a mid-stage study.

gene editing

New David Liu startup wants to reach difficult tissues with gene therapy

Storied Broad Institute biochemist David Liu is launching a gene editing startup meant to deliver therapies directly to precise sites around the body, STAT has learned. The company, Nvelop, is based on Liu's prior work loading base editors, which can swap out individual base pairs, into viral shells and editing genes in mouse livers, eyes, and nervous systems.

NVelop last year raised $100 million in seed funding, and is being run by Jeff Walsh, a former top executive at Bluebird Bio. It recently recruited Melissa Bonner, Bluebird's head of research.

Using virus-like particles to deliver CRISPR-based therapies and the like could ultimately be more scalable than existing delivery technologies, experts say. But the road could be long, since manufacturing these particles continues to be quite arduous.

cancer

GSK's JAK inhibitor approved for bone marrow cancer

The FDA just approved momelotinib, a JAK inhibitor that GSK acquired last year after buying Sierra Oncology for $1.9 billion. The drug, branded Ojjaara, is now sanctioned to treat intermediate or high-risk myelofibrosis, a blood cancer, in patients with anemia. In a happy twist for GSK, regulators said the medicine could be given to patients regardless of their treatment history, FiercePharma writes. Peak sales could be "comfortably above blockbuster status," according to GSK's chief commercial officer Luke Miles.

Ojjaara's top competitor is Jakafi, a JAK inhibitor made by Incyte that was approved more than a decade ago. In some patients, Jakafi might actually worsen anemia — which creates a gap in the market that GSK hopes to fill with Ojjaara.

psychedelics

Nonprofit behind MDMA therapy will file for FDA approval

MAPS, a nonprofit that makes an MDMA-based therapy for post-traumatic stress disorder, will file to have the drug approved by the FDA. Although MDMA is illegal and the main component of the party drug ecstasy, a number of studies have validated its efficacy in treating PTSD. The decision to file came after a late-stage study published last week showing that MDMA given alongside therapy helped 71.2% of patients no longer qualify for PTSD diagnosis. By comparison, only 46.2% of patients receiving placebo had the same outcome.

MAPS was founded in 1986, and said it hopes that MDMA-assisted therapy will be approved next year — and that it inspires other researchers studying therapeutic psychedelics, Reuters writes.

drug pricing

South Africa says J&J's price for TB drug was 'excessive'

Johnson & Johnson is being probed by South African regulators over the "excessive pricing" of its critically important tuberculosis drug bedaquiline. The South African Competition Commission said in a statement that it had "reasonable suspicion" that the company might have engaged in "exclusionary practices."

Bedaquiline is a lifesaving component of a variety of combination therapies to treat multi drug-resistant tuberculosis. The pill was approved more than a decade ago, but it's still hard for many lower-income countries to get access to it. Initially, J&J was charging $900 for a single course of the drug in low-income countries.

Patient advocacy groups are increasingly putting pressure on J&J, however — so the pharma giant agreed last month to lower the medicine's price by as much as 55% in many low- and middle-income countries. About 1.5 million people die each year from the disease.