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• An FDA advisory panel vote of 9-1 to recommend the approval of Mesoblast Limited’s graft versus host disease (GVHD) cell therapy was a cruel tease for the Australian company.

• More than four years later—and after two rejections from the agency—Mesoblast has finally gained an endorsement from the U.S. regulator for its first approved drug.

• Yesterday afternoon, the FDA signed off on Ryoncil (remestemcel-L), an off-the-shelf cell therapy to treat patients 2 months and older who have acute GVHD and don’t respond to steroids.

• Ryoncil becomes the first FDA-approved treatment to use mesenchymal stromal cells (MSC) in any indication. MSCs are taken from the bone marrow of unrelated donors and can differentiate into a variety of cell types.

• For as long as Mesoblast waited, the nod came three weeks ahead of the FDA's target date for an approval decision.

• “It gives everybody a lovely Christmas present—and a happy new year,” Mesoblast CEO Silviu Itescu, who founded the company 20 years ago, said on a conference call. (Fierce Biotech)

• After seven decades with no advances in the treatment of the genetic disorder classic congenital adrenal hyperplasia (CAH), Neurocrine Biosciences has scored FDA approval for its first-in-class, twice-daily oral medicine Crenessity (crinecerfont).

• The approval came nearly two weeks before its FDA target date and is termed as “paradigm shifting” by the San Diego-based company. The selective oral corticotropin-releasing factor type 1 receptor (CRF) antagonist can be used by CAH patients age 4 and older.

• Serving as an add-on to glucocorticoid replacement therapies, Crenessity reduces excess adrenocorticotropic hormone (ACTH) and downstream adrenal androgen production, allowing for glucocorticoid dose reduction.

• It’s a key breakthrough for CAH patients as long-term use of glucocorticoid replacement therapies at high doses can cause weight gain, heart problems and bone loss. (Fierce Pharma)

• The FDA has blessed two previously approved medicines—one a cream and the other an injection—to treat atopic dermatitis (AD). Both products now will be available to a significantly larger patient population as roughly 26 million in the U.S. have the disorder, which is also known as eczema.

• Organon’s Vtama, a topical cream originally approved for plaque psoriasis in 2022, is now cleared to treat AD, the company said. The nod came three months after New Jersey-based Organon acquired the product in a $1.2 billion takeover of Dermavant.

• Similarly, the FDA gave a thumbs-up to Galderma’s Nemluvio to treat AD. The drug, a monthly subcutaneous injection, was previously endorsed by the U.S. regulator for prurigo nodularis. (Fierce Pharma)

• How do you get a bunch of investors on Wall Street to understand the importance of manufacturing? If you’re Thijs Spoor, CEO of radiopharma biotech Perspective Therapeutics, you take them straight to the factory.

• His company is developing radiopharmaceuticals, an area of drug development that has seen frenzied Big Pharma dealmaking.

• But the complex manufacturing processes required to turn radioactive isotopes into therapeutic agents has the potential to scare off investors who don’t understand how a company will scale, or how the therapy will get from A to B when a patient needs it, Spoor explained.

• So despite being in the early stages of clinical development, Perceptive is centering manufacturing in its pitches—and the decommissioning of a manufacturing facility provided the perfect opportunity.

“We finally got investors over the hump by literally doing tours of our manufacturing site. We said, don’t look at pictures, just come on in,” Spoor said.

“We invited everyone to go and touch and feel the manipulators and try it out. And we left with some extraordinary converts who all said, yeah, we didn’t get it. Now we totally get it.”

• Viridian Therapeutics’ stock jumped Monday after the company unveiled another Phase 3 look at its potential competitor to Amgen’s Tepezza in thyroid eye disease.

• Veligrotug met all its primary and secondary endpoints in chronic thyroid eye disease patients, Viridian announced. TED occurs in two phases: an active phase when symptoms tend to worsen, and a chronic phase when symptoms stabilize but patients may still experience some eye changes.

• Of chronic patients who received veligrotug in the Phase 3 THRIVE-2 study, 56% had a reduction in proptosis, or bulging of the eyes — a hallmark of the disease. In the placebo group, 8% of patients saw a response. The results on this measure—the trial’s primary endpoint—were statistically significant, the company said in its announcement.

• Veligrotug also met the secondary endpoint of improvement in diplopia, or double vision. In the veligrotug arm, 56% of patients had a diplopia response and 32% achieved complete resolution of diplopia, compared to 14% of placebo patients who saw a complete resolution. (Endpoints)

• An experimental drug co-developed by Teva and Sanofi met its main objectives in a mid-stage clinical trial in inflammatory bowel disease, showing what the partners claimed to be “best-in-class potential.”

• Around half of people with ulcerative colitis who received a high dose of the drug, duvakitug, in a Phase 2 trial had their symptoms resolve after 14 weeks, versus just over 20% of those on placebo. A similar, roughly 48% of Crohn’s disease patients given a high dose of duvakitug experienced meaningful improvement on a measure of disease severity, compared to 13% of people on placebo, Sanofi and Teva said.

• The companies didn’t provide details, which will be presented at a medical meeting next year. Still, they said the drug’s effects were consistent across subgroups and rates of treatment-related side effects were similar between both study groups. (Biopharma Dive)

• Novo Holdings, the parent company of Novo Nordisk, can proceed with its planned $16.5 billion acquisition of Catalent, a leading contract drug manufacturer, after U.S. regulators declined to challenge the deal following months of scrutiny.

• The deal had generated enormous interest over concerns that competition in the pharmaceutical industry could be harmed.

• Indeed, the move was prompted by sporadic shortages of one of the world’s hottest-selling medications—Novo Nordisk’s weight loss treatment Wegovy—and designed, in part, to solve what has been a critical and seemingly intractable problem for Novo Nordisk. (STAT News)

• But this year the world got a glimpse of what might be the next best thing: an injectable drug that protects people for 6 months with each shot.

• Any doubts about the finding disappeared 3 months later when a similar trial, conducted across four continents, reported 99.9% efficacy in gender diverse people who have sex with men.

“It has the potential, if we can do it right, which means going big and getting it out there,” says Linda-Gail Bekker, an infectious disease specialist at the University of Cape Town who led one of the two efficacy trials for the drug’s maker, Gilead Sciences.

• The off-the-charts success of the drug as PrEP sprang from a basic research advance: a new understanding of the structure and function of HIV’s capsid protein, which lenacapavir targets.

• Many other viruses have their own capsid proteins, which form a shell around their genetic material, so this drug’s triumph raises the exciting prospect that similar capsid inhibitors could fight other viral diseases.