Hello! Hope you had a restful and reflective Indigenous Peoples’ Day. Today, we talk about how the autoimmune disease space is hotter than ever, see more gene therapy contraction, and the CEO of a generics giant talks about how elections affect industry.
The need-to-know this morning
Investment in autoimmune disease is soaring
Investment has surged this year in autoimmune disease: More than $1.7 billion has been committed to this space in the first half of the year alone. That surpasses investment in the ultra-hot spaces like cardiometabolic disease and obesity.
The influx, STAT’s Allison DeAngelis writes, is driven largely by breakthroughs in CAR-T therapy, particularly its efficacy in lupus. Cancer companies now are realizing the potency of their platforms in autoimmunity, and pivoting.
Concerns still remain about CAR-T’s toxicity and fertility risks, which has prompted some researchers to pursue other approaches like T-cell engagers instead — and there’s thought that next-gen immunotherapies could offer even more promise.
Another high-profile gene therapy retrenches
The high-profile “jumping gene” editing biotech SalioGen Therapeutics will lay off some of its employees and is scaling back its ambitions amid the broader downturn in the sector, STAT’s Jason Mast writes. The company launched in 2021 with technology meant to insert full-length genes into patient genomes, with a focus on diseases like cystic fibrosis and Stargardt disease. It has raised about $135 million to date.
But SalioGen isn’t alone. Hurdles in development and manufacturing complexities, along with the fizzling of investment in gene editing, has led to retrenchment in similar companies. Tome Biosciences, for instance, signaled in August that it would shut down — less than a year after announcing a $213 million raise.
Sandoz CEO weighs in on why global elections matter
It’s not just the U.S. counting down to a major election. More than 75 countries are soon headed to the polls, Sandoz CEO Richard Saynor points out. He sees two key themes in the forthcoming elections: heightened interest in protectionist economic policies and concerns about inflation.
“Protectionist policies,” Saynor opines, “will work against the goal of expanding patient access to essential medicines.”
The key to allowing patients globally to access medicines — particularly generic and biosimilar drugs — is to maintain strong global supply chains, he says. Saynor proposes ways for governments to reduce health care costs, stabilize supply chains, and maintain patient access to medicine.
Lundbeck buys Longboard for $2.6 billion
Lundbeck is buying Longboard Pharmaceuticals for $2.6 billion, or $60 a share — a 54% premium over Longboard’s stock price, STAT’s Andrew Joseph writes. Just a year ago, Longboard’s stock was only around $5 — but has since surged thanks to positive Phase 3 results from its lead durg, bexicaserin, which has been found to reduce seizures in some forms of epilepsy.
This is a major part of Lundbeck’s aim to expand its neuro-rare franchise, CEO Charl van Zyl said in a statement. If bexicaserin wins approval, it could launch in 2028 and see peak sales between $1.5 billion and $2 billion.
Jasper looks to rival Genentech and Novartis’ Xolair
Jasper Therapeutics, a biotech seeking to make stem cell transplants less taxing and to treat immune-mediated diseases, reported that its experimental drug helped patients with a chronic skin condition in an initial and closely watched clinical trial.
In a small study, 14 of 15 patients given an injection of the drug, briquilimab, showed improvements in symptoms of chronic inducible urticaria, a disease defined by red, itchy welts.
Yesterday’s announcement was enough to send Jasper’s stock up 10%. The spike was fueled in part by the possibility that Jasper’s drug could compete with Xolair, a blockbuster sold by Novartis and Genentech that is already approved for chronic hives, STAT’s Jonathan Wosen notes.
More reads
- In stark reversal, FDA to reconsider removal of Eli Lilly weight loss drug from shortage list, STAT
- Why I’m wary of the new schizophrenia miracle drug, STAT
- Pfizer details results from failed Phase 3 trial of Duchenne muscular dystrophy gene therapy, Endpoints
- Demis Hassabis’s drug discovery start-up accelerates spending to ‘solve’ diseases, Financial Times
- Novartis channels Gargamel, terminating midphase SMURF1 trial, FierceBiotech