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.

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.

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.

With conscious and ethical use of Generative AI we can forge a future that is empathetic, innovative and can meet all our needs at the right time and place.

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.

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.

The healthcare industry is sitting on a wealth of data gathered from electronic health records, commercial pharmacies, health systems and payers, and health tech companies. So much that it makes up one-third of the world’s data. Pharmaceutical companies need to tap into this type of data to build inclusive clinical trials,

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.

If we want to support people with obesity, the weight loss debate must stop conflating obesity prevention with obesity treatment. Instead, we should focus on the tools that best solve the needs of people where they are – just as we would for cancer or diabetes – by making the most effective treatments possible available.

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.

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.

Drug developers need policies and technological infrastructure to execute on their goal of being patient-focused. At a minimum, this means a compliant way to engage patients and truly hear their voices; at its most effective, patient engagement also meets patients where they are – whenever and wherever that may be.

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.

Novartis became the seventh drugmaker to legally challenge the White House’s Medicare drug pricing negotiation program. The company filed its lawsuit three days after HHS announced the first 10 drugs that were selected for the program — its heart failure medication Entresto was one on the list. HHS can expect to face more lawsuits from the manufacturers of the 10 drugs it named last week, experts have warned.

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.

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.

Despite these wins for many patients, the new law is already impacting the discovery and development of new drugs for people living with orphan diseases. Not only are drugs that could treat more than one disease being disincentivized, small molecule medicines, which play an important role in treating neurological disorders, cancers, and other diseases, may also be disadvantaged by the law.

The Apellis Pharmaceuticals restructuring will save up to $300 million. It comes as the biotech continues its inquiry into the cause of a rare inflammatory complication reported in a small number of patients who received its geographic atrophy drug, Syfovre.

A Bayer cell therapy for Parkinson’s disease has met primary and secondary goals of its first test in humans, and the pharmaceutical giant is now planning for a larger Phase 2 clinical trial expected to begin enrollment in the first half of next year.

The Walt Disney Company excels in designing exceptional service because they are hyper-focused on listening, tracking, designing, and measuring the experiences of guests visiting their properties. We can apply this approach in pharma by enhancing our patient journey maps and go beyond focusing on the key clinical engagement touch points (e.g., doctor visits, prescription pickups) and enriching our insights with the micro-moments surrounding these obvious events.

Researchers at the University of Texas MD Anderson Cancer Center published two studies this week on a new approach that could improve treatment for patients with pancreatic cancer. The preclinical studies showed that combining immunotherapy with a KRAS inhibitor can lead to long-lasting tumor elimination.

Future research should focus on identifying the interactions between the immune system, microbes, and allergic responses; and exploring potential therapeutic strategies targeting the skin microbiome for allergy management.

Rapport Therapeutics, a clinical-stage biotech that was founded last year, raised $150 million in Series B financing, bringing its total funding to date to $250 million. The company’s platform leverages receptor-associated proteins to create neurology drugs that have more focused activity in the brain, going after disease-driving circuits and cell types.