NurOwn, Brainstorm Cell Therapeutics’ experimental stem cell therapy for ALS, did not win the backing of an independent panel of FDA advisors. Many advisory committee members said they want to see more data from another clinical trial, the same guidance the FDA has given the biotech for nearly three years.

Ionis Pharmaceuticals’ olezarsen has Phase 3 results showing the therapy handily beat a placebo at reducing fat levels in the blood due to a rare, inherited metabolic disorder with no FDA-approved drugs. Ionis plans early 2024 submissions for what could become the first medicine it commercializes without a partner.

Intercept Pharmaceuticals’ acquisition by Alfasigma comes three months after the FDA again rejected the biotech’s drug as a treatment for the fatty liver disease NASH. But Intercept still has rare liver disease assets, and Italy-based Alfasigma says acquiring the company will help it expand in gastroenterology and hepatology.

Novo Nordisk is paying Valo Health $60 million up front to gain three preclinical cardiovascular disease programs. Milestone payments for those programs and others covered under the artificial intelligence drug discovery pact could reach up to $2.7 billion.

Scynexis said no adverse events or contamination problems have been reported for Brexafemme, an antifungal approved for treating vaginal yeast infections. The biotech said the risk of cross-contamination stems from the processes of the small molecule’s third-party manufacturer.

Syntax Bio’s technology platform uses CRISPR to direct stem cells to become a desired cell type in a process that’s more scalable and less expensive than current methods. The startup is raising a Series A round of financing to further demonstrate the potential of its technology.

Standard newborn screening can detect dozens of inherited diseases, but genomics technology enables testing for hundreds of them. A North Carolina program evaluating the feasibility of bringing genomics to newborn screening is expanding with an initial goal of testing 5,000 babies in the next year.

Seven months after Travere Therapeutics’ flagship drug received accelerated FDA approval in a rare kidney disease, the molecule has fallen short of the key goal of its confirmatory test. It’s the small molecule’s second Phase 3 failure this year.

Specific targets were not disclosed, but Merck KGaA is betting the artificial intelligence technologies of Exscientia and BenevolentAI will discover into new drugs for cancer, neurology, and immunology.

ARS Pharmaceuticals frames its intranasal epinephrine spray as a needle-free alternative to products such as EpiPen. Though this spray won the backing of an FDA advisory committee, the agency is now requiring that ARS Pharma run another study to support a regulatory submission.

BeiGene welcomes the return of the immunotherapy’s rights as an opportunity to make it the backbone of its solid tumors strategy. But Novartis cedes an opportunity to gain a competitor to established cancer immunotherapies from Merck and Bristol Myers Squibb.

Magnet Biomedicine builds on decades of molecular glue research from its scientific co-founder, Harvard University Professor Stuart Schreiber. The startup is developing drugs that take these glues beyond the biotech industry’s current focus on targeted protein degradation.

The Coalition for Epidemic Preparedness Innovations, or CEPI, has pledged up to $90 million to BioNTech’s messenger RNA vaccine candidates for mpox. This research could help advance the scientific understanding of the application of mRNA technology to Orthopoxviruses, which includes mpox, formerly known as monkeypox.

FDA approval of GSK’s Ojjaara in myelofibrosis introduces a new competitor to blockbuster Incyte drug Jakafi. Ojjaara was part of GSK’s $1.9 billion acquisition of Sierra Oncology last year.

RayzeBio’s IPO will support pivotal testing of a targeted radiopharmaceutical for cancer patients who progress after treatment with Novartis’s Lutathera. Neumora will apply its IPO cash toward Phase 3 testing of a depression drug with a novel mechanism of action.

Allogeneic cell therapy hasn’t caught up to autologous cell therapy yet, and the disappointing data so far has soured the entire allogeneic field for at least one venture capitalist. The cell therapy debate was one of the topics covered during a panel discussion at the Biopharm America conference.

Pharmaceutical industry observers say CVS Health’s creation of Cordavis, a new biosimilar drugs company, is part of a strategy to make its move into this market as profitable as possible.

Avalon BioVentures unveiled new portfolio company Arialys Therapeutics. The startup’s lead program, an antibody from Astellas Pharma, is a potential treatment for anti-NMDA receptor encephalitis (ANRE), a rare autoimmune inflammatory disorder.

The FDA said the updated messenger RNA vaccines from Pfizer/BioNTech and Moderna will protect against the omicron subvariants now prevalent in circulation. Left out of the FDA decision is Novavax, whose protein-based vaccine is still under regulatory review.

Moderna and Immatics aim to see if the combination of their respective technologies can lead to better cancer treatments spanning multiple therapeutic modalities. Milestone payments to immunotherapy developer Immatics could top $1.7 billion.

Digital therapeutics developer Click Therapeutics is partnering with Indivior to develop a mobile app for substance use disorder. Indivior is now Click’s third development partner, following deals with Boehringer Ingelheim and Otsuka Pharmaceutical.

Seagen and Nurix are bringing their respective technologies together to develop a new class of cancer drugs called degrader antibody conjugates. Nurix CEO Arthur Sands says the multi-year, multi-target collaboration expands the reach of each company’s technology.

By revealing how tumors interact with immune cells, Noetik aims to identify new targets for new cancer drugs. The startup and its artificial intelligence-driven platform are supported by $14 million in seed financing.

Gene therapy biotech Kriya Therapeutics has acquired Tramontane Therapeutics, a startup focused on metabolic diseases. Tramontane brings a gene therapy that’s a potential one-time treatment for the fatty liver disease NASH.

Peanut allergy therapy Palforzia, which Nestlé acquired in a multi-billion dollar deal, has been sold to allergy product company Stallergenes Greer. Palforzia was the first FDA-approved drug for peanut allergy, but its sales were hampered by the Covid-19 pandemic.

Startup Star Therapeutics will use the Series B funding to support clinical development of a drug candidate for von Willebrand disease, a bleeding disorder. The capital will also support the formation of new biotech subsidiaries developing antibodies with therapeutic applications in hematology and immunology.

Intarcia Therapeutics’ GLP-1 agonist product candidate for type 2 diabetes now belongs to i2o Therapeutics, which will make the treatment’s case for approval at an upcoming FDA advisory committee meeting. The startup’s $46 million Series A financing is one of several recently announced funding rounds.

Amgen and the Federal Trade Commission are settling the lawsuit the regulator filed to block the pharmaceutical giant’s $28 billion Horizon Therapeutics acquisition. As part of the settlement, Amgen agrees not to “bundle” its products with Horizon’s drugs in negotiations with health plans.

Legislation introduced by Utah Senator Mike Lee would eliminate testing to show a biosimilar can be substituted for a reference biologic product. Lee contends the current testing requirement adds costs and delays market access for these lower-cost biological medicines.

Novo Nordisk is expanding its cardiometabolic drug pipeline with the acquisition of Embark Biotech, a startup developing drugs for a novel target that can suppress appetite and boost calorie burning in cells. Embark’s science comes University of Copenhagen research on a novel fat cell target.