IntellaTurn, LLC

• Ultragenyx secured its first gene therapy approval, with the FDA clearing GENGLYCOS as the first treatment designed to address the underlying cause of the ultra-rare metabolic disorder, glycogen storage disease type Ia (GSDIa).

• GSDIa is caused by a deficiency in glucose-6-phosphatase, an enzyme that causes the liver to release glucose into the bloodstream during fasting. Without it, patients can experience potentially life-threatening hypoglycaemia and must maintain strict dietary regimens, including frequent doses of raw cornstarch to keep blood sugar levels stable.

• GENGLYCOS is designed to address the root cause of the genetic defect. The one-time treatment uses an AAV8 vector to deliver a functional copy of the affected gene to the patient’s liver cells, with the aim of restoring the liver’s ability to regulate glucose. (Pharmaphorum)

• After more than 10 years in development, Regeneron secured a landmark approval for its monoclonal antibody garetosmab, now commercially known as Pasatru.

• The drug has become the second cleared by the FDA to treat fibrodysplasia ossificans progressiva (FOP) and the first to reduce clinician-assessed flare-ups in adults with the disease.

• Pasatru, given intravenously every four weeks, is designed to block what is believed to be the key protein behind the FOP disease process—activin A—which was pinpointed by Regeneron scientists as a possible target for treating FOP more than a decade ago.

• FOP is an ultrarare genetic disorder—there are roughly 900 cases worldwide—wherein the body’s soft tissues progressively turn to bone. It’s marked by painful flare-ups: hot and swollen masses in the tissue that precede the permanent conversion of muscle, tendons and ligaments to bone. (Fierce Pharma)

• The FDA granted an accelerated approval to Bristol Myers Squibb’s Zenbexus, a decision that could help the drugmaker sustain a multiple myeloma franchise in decline due to generic competition.

• The approval is a “milestone” for medicines like Zenbexus, which are called “CELMoDs” and harness the body’s waste disposal system to trash diseased cells, wrote William Blair analyst Matt Phipps wrote in a note to clients.

• Bristol Myers has multiple CELMoDs in development, including a second myeloma treatment nearing approval. Inherited through an acquisition of Celgene, that portfolio is “underappreciated by investors,” wrote RBC Capital Markets analyst Trung Huynh.

• The clearance makes Zenbexus available for second-line use alongside Johnson & Johnson’s Darzalex and the steroid dexamethasone. (Biopharma Dive)

• President Donald Trump announced Heidi Overton as his pick for FDA commissioner in a social media post Wednesday.

• Overton, who has both a medical degree and PhD but no direct regulatory experience, inherits an FDA that was in flux but has since stabilized in recent months.

• It’s unclear how she might lead the agency, and her policy stances outlined in previously published papers and briefs span much of healthcare.

• Over the years, she’s advocated for increased hospital transparency, changes to how doctors prescribe opioids and more oversight of the abortion pill.

• Overton is currently the deputy director of the Domestic Policy Council. The organization has helped shape some of the most- favored-nation drug pricing negotiations between large pharma companies and the administration, alongside CMS.

• The most notable chapter of her career was the five and a half years spent at the America First Policy Institute (AFPI), a Trump-aligned think tank. While there, Overton co-authored a number of policy papers including ones on hospital price transparency and vaccine mandates. (Endpoints)

• Moderna and Merck shares soared after the companies said a personalized mRNA treatment hit its target of preventing skin cancer from recurring or spreading in a late-stage clinical trial.

• The results come at a pivotal moment for Moderna and mRNA research, after Trump administration health officials cut federal funding for new vaccines and asserted the technology poses more risks than rewards.

  • The customized therapy, which could see a commercial launch next year, could also be adapted for other cancers of the kidney, bladder and pancreas.

• The results could lead to a remarkable turnaround for Moderna, whose market value plummeted more than 90% after demand for its COVID vaccine declined post-pandemic.

• "People will experience this as ... the COVID company now [takes on] cancer," Moderna president Stephen Hoge said.

"But for those of us that live in it, it's like the cancer company that pivoted on a dime to help with COVID has pivoted back and really finally come home." (Axios)

• A once high-flying Boston-area drug company mounted somewhat of a comeback, reporting positive results from a clinical trial testing a potentially first-of-its-kind treatment for the dangerously low blood sugar levels some patients experience after bariatric surgery.

• Amylyx Pharmaceuticals said its medicine, known as avexitide, hit the main goal of the late-stage study by spurring a 55% reduction in serious to severe hypoglycemic events compared to a placebo.

  • Such events starve the brain of the sugar it needs for fuel, leading to confusion, dizziness, slurred speech and loss of coordination. In more dire cases, patients can faint, have seizures or become comatose.

• According to Amylyx, its medicine also appeared safe. (Biopharma Dive)

• That would make it one of the largest, if not the largest, going-public transactions in industry history, topping Parabilis Medicines’ $670 million Nasdaq debut in June.

• And two more companies just chose this avenue: Redx Pharma on Friday and Slate Medicines on Monday.

• The current renaissance has involved 17 drug developers so far in 2026, which almost equals this year’s 19 biotech IPOs.

• It’s the most active year since 2018, according to Raymond James bankers.

• The device is authorized for use in outpatient settings and must be operated under the oversight of a supervisor trained in phlebotomy.

• One phlebotomist can manage up to three Aletta devices simultaneously, which may help address the current phlebotomist shortage in the US.

• Once a vein is identified, the system applies a tourniquet, prepares the skin, inserts and disposes of the needle, changes collection tubes, and applies a bandage.

• The supervising phlebotomist confirms that collection tubes are filled in the correct order and verifies that all tubes are sufficiently full following the procedure.