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Hello! We’re just a few days out from STAT’s Breakthrough Summit East!
In the meantime, today we learn about China’s progress with CRISPR treatments, and see a new cell therapy approach for graft-versus-host that shows promise.
The need-to-know this morning
- Avidity Biosciences presented updated study results on its RNA-based drug for people with Duchenne muscular dystrophy mutated at exon 44. The drug showed increases in dystrophin production and reductions in a key muscle-damaging protein.
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Curevo Vaccine, a small company developing a new shingles vaccine, announced that the venture capitalist Moncef Slaoui, who previously ran the U.S. government’s Operation Warp Speed Covid vaccine effort, would become its chairman.
- Incyte reported positive results from two Phase 3 studies of its experimental oral medicine povoricitinib in patients with
hidradenitis suppurativa, a chronic, inflammatory skin condition. Incyte shares fell, however, because povoricitinib’s efficacy fell short of expectations relative to approved biologic treatments for the disease.
Orca Bio’s T cell therapy looks promising for GVHD
An experimental T cell therapy, Orca-T, significantly reduced chronic graft-versus-host disease in leukemia patients undergoing stem cell transplant, meeting the primary goal of a Phase 3 study.
In the 187-patient trial, Orca-T, developed by Orca Bio, lowered chronic GVHD risk by 74% compared to standard transplants, with 78% of patients alive without GVHD after one year versus 38% in the control group. Survival at one year was 94% for Orca-T patients, with no increase in relapse rates.
Orca plans to file for FDA approval later this year, STAT’s Adam Feuerstein writes, positioning its therapy as a safer alternative to traditional donor-matched transplants.
12 Chinese companies set to transform gene-editing
The CRISPR competition between U.S. and Chinese biotechs is intensifying, with Chinese firms aggressively advancing treatments for rare diseases, oncology, and blood disorders. While the U.S. leads in some areas like beta thalassemia, Chinese companies are rapidly developing in vivo gene-editing therapies and leveraging a more flexible regulatory environment to accelerate clinical trials.
In the latest feature in our “Who to Know” series, STAT contributor Brian Yang assembles a list of 12 Chinese companies leading the charge in gene-editing innovation.
Firms like HuidaGene, YolTech, and BRL Medicine are pioneering treatments for Duchenne muscular dystrophy, beta thalassemia, and sickle cell disease, while others like GenAssist and MedGenCell focus on muscular and lung cancer therapies.
AstraZeneca buys European cell therapy company
From STAT’s Andrew Joseph: AstraZeneca said this morning it was buying the Belgian cell therapy company EsoBiotec for $425 million upfront, building out another cancer-targeting approach.
EsoBiotec is developing in vivo cell therapies, meaning treatments that can engineer a patient’s immune cells in the body to recognize and attack tumor cells. Current cancer-fighting cell therapies involved removing a patient’s immune cells, modifying them, and then infusing them back into the patient, typically a weeks-long process. If EsoBiotec’s approach works, the therapy would be delivered by an IV injection.
AstraZeneca framed the deal, which also includes up to $575 million in milestone payments, as a way to potentially expand the availability of cell therapies. It also said it may explore the in vivo cell therapy approach in autoimmune diseases.
Under the agreement, EsoBiotec will keep its operations in Belgium as an AstraZeneca subsidiary.
RFK Jr.’s regenerative medicine meeting raises red flags
HHS Secretary Robert F. Kennedy Jr. held a regenerative medicine roundtable on earlier this month whose participants included not only cautious voices from the Alliance for Regenerative Medicine and FDA officials but also advocates for unproven treatments. That sparks concern about potential FDA deregulation of adult stem cell therapies, writes UC Davis cell biology professor Paul Knoepfler, in his new Lab Dish column on regenerative medicine.
“Could this roundtable have included over-exuberant cheerleading for unproven adult stem cells and decreased regulation?” Knoepfler writes.
Discussions reportedly centered on reducing regulatory barriers. While Kennedy claims he doesn’t want a regulatory “Wild West,” transparency issues and the absence of key FDA figures like Peter Marks fuel speculation that looser oversight may prioritize industry interests over patient safety.
Latigo raises $150 million for non-opioid pain drugs
From STAT’s Allison DeAngelis: The pain field is heating up, with a startup raising more money to advance a non-opioid treatment for chronic pain.
Latigo Biotherapeutics recently raised another $150 million from investors, it announced this morning. It’s a rapid-fire recapitalization for the company, which launched 13 months ago with $135 million. The biotech will use the funding to push its two drug candidates through clinical trials, and expand its R&D pipeline.
The two existing drugs designed to silence pain-signaling neurons by blocking a sodium channel called NaV 1.8 — the same approach used by Vertex’s Journavx, which was approved by the FDA in January for acute pain. Latigo’s lead drug is currently being tested in a Phase 2 trial in acute pain, with a Phase 3 trial slated to start by the end of the year.
Latigo has drug candidates for both acute and chronic pain settings, but it’s the latter that’s gotten attention in the industry. In 2021, it was estimated that one in five U.S. adults experienced chronic pain, but there are few treatment options. Latigo hopes to move its chronic pain treatment into a Phase 2 trial by the end of the year.
More reads
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U.K. plan to require drugmakers to pay higher rebates draws criticism from industry group, STAT
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In rebound year for Pfizer, CEO Albert Bourla scored 14% total pay increase to $24.6 million, FiercePharma