Novartis is enlisting the help of Generate:Biomedicines and its artificial intelligence platform to generate drugs for multiple undisclosed targets. Generate will receive $65 million up front, which includes an equity investment of $15 million, and is eligible to receive more than $1 billion in milestone payments, as well as tiered royalties up to the low double-digits on sales of drugs resulting from the collaboration.

Biogen and UCB stay the course for phase III lupus success

Despite a phase IIa failure in 2018 for lupus, Biogen Inc. and UCB Inc. maintained their collaboration and now have new positive data for their anti-CD40L candidate. Top-line phase III data show dapirolizumab pegol, along with the standard of care, met the primary endpoint in demonstrating an improvement in moderate to severe systemic lupus erythematosus compared to placebo at 48 weeks. With these data in hand, the two companies are beginning a second phase III before the year ends. Biogen also has litifilimab, a phase II “home-grown asset” also known as BIIB-059. Litifilimab is a first-in-class, humanized IgG1 monoclonal antibody targeting blood dendritic cell antigen 2 for cutaneous lupus erythematosus.

Wave Life climbs on WVE-N531 phase II Duchenne data

Interim phase II data of Wave Life Sciences Ltd.’s oligonucleotide, WVE-N531, revealed impressive dystrophin expression, solid safety, and the potential for once-monthly dosing for boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping. The findings not only validate the Cambridge, Mass.-based company’s platform and pipeline, but it drove Wave Life’s stock (NASDAQ:WVE) up by 51% in early trading Sept. 24 to $8.07. The data also suggest the company’s candidate could become a best-in-class exon skipper, as initial data appear better than that seen from competitors Sarepta Therapeutics Inc. and Dyne Therapeutics Inc. Wave Life expects a final readout in the first quarter of 2025, when it will also seek U.S. FDA guidance over a potential accelerated approval filing.

ECNP 2024: At both ends of life, improving cognition in Down syndrome

At the 2024 European College of Neuropsychopharmacology (ECNP) Congress, researchers have presented research that could lead to ways to boost brain development and prevent neurodegeneration in individuals with Down syndrome. Most individuals with Down syndrome have a degree of intellectual disability that prevents them from living independently. And because they have three copies of the amyloid precursor protein (APP) gene, which is located on chromosome 21, people with Down syndrome have very high levels of plaques early in life. By age 55, 80% of them also have clinical dementia. On the average, individuals with Down syndrome develop AD slightly earlier than those with familial AD. At the ECNP Congress, investigators tackled both issues in a symposium on “Down syndrome: from early brain development to adult-occurring neurodegeneration.”

Ozempic, Wegovy pricing confab could be in the cards

While in the hot seat today at a U.S. Senate Health, Education, Labor and Pensions Committee, Novo Nordisk A/S President and CEO Lars Fruergaard Jørgensen said he would sit down with Sen. Bernie Sanders (I-Vt.) and the three largest pharmacy benefit managers (PBMs) to discuss lowering the list prices for the company’s popular diabetes and weight-loss drugs, Ozempic and Wegovy. But he seemed a bit skeptical about the PBMs’ written commitment to Sanders, saying any change needs to be handed to the patients, and not just the PBMs’ insurance companies. He also noted that when Novo Nordisk cut the list price of a product before, the PBMs dropped it from their formularies.

Telix expands US radiopharma footprint with $250M RLS buy

Radiopharmaceutical company Telix Pharmaceuticals Ltd. is continuing its buying spree to shore up dominance in the radiopharmaceutical market with its latest acquisition of RLS Radiopharmacies for $250 million to expand its U.S. manufacturing presence and establish a next-generation radiometal production network. Under terms of the deal, Telix will pay RLS $230 million up front and an additional $20 million if certain milestones are met. The deal is expected to close in the first quarter of 2025.

BMS, Merck, Beigene PD-1s to come under scrutiny by ODAC

Briefing documents related to the Sept. 26 meeting of the U.S. FDA’s Oncologic Drugs Advisory Committee meeting were made public, providing a peek into the panel’s discussion of the PD-1 inhibitor class. Under scrutiny are compounds for advanced gastric and gastroesophageal junction adenocarcinoma and in esophageal squamous cell carcinoma. At issue, as the FDA said in an August Federal Register filing, is whether approval of the drugs should be restricted in accordance with PD-L1 expression. In the crosshairs are Bristol Myers Squibb Co.’s Opdivo (nivolumab, given the first go-ahead in December 2014) and Yervoy (ipilimumab, first cleared in March 2011), along with Merck & Co.’s Keytruda (pembrolizumab, greenlighted in September 2014). ODAC members will take a look, too, at Beigene Ltd.’s BLA related to Tevimbra (tislelizumab).

Also in the news

Acadia, Aligos, Antengene, Assembly, Astrazeneca, Bioatla, Biocity, Biohaven, Black Diamond, Context, Convergent, Crown, Diamyd, Elevation Oncology, Entrada, Genfit, Glaukos, Ideaya, Immusoft, Innate, Ipsen, Lantern, Leo, Medincell, MSD, Neuraxpharm, Nicox, Oryzon, Realta, Repare, Revance, Sanofi, Sensorion, Skye, Sumitomo, Telix, Teva, UCB, Uniqure