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.

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.

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.

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.

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.

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.

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.

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.

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.

Bristol Myers Squibb drug Reblozyl is now approved as a first-line therapy for anemia caused by myelodysplastic syndromes, a group of blood cancers. It’s the third indication for the drug, which analysts project could top $4 billion in peak sales.

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 founder and CEO of Unlearn.AI seeks to make clinical trials more efficient and less costly by leveraging its Digital Twin technology. If the company is successful, drugs could potentially be brought to market sooner and in a cost effective manner.

Imugene gains global rights to Precision BioSciences’ most advanced program, an allogeneic cell therapy for advanced cases of blood cancer. Precision will now focus on in vivo gene editing therapies, some of which is partnered with Novartis and Eli Lilly.

Oncology is already a big piece of Pfizer’s drug lineup. The portfolio is expanding to multiple myeloma with FDA approval of Elrexfio, a bispecific antibody that targets the cancer protein BCMA.

Zynerba Pharmaceuticals’ lead drug candidate is designed to bring the therapeutic effect of cannabinoids without also causing euphoric effects. The synthetic cannabinoid in a topical gel formulation includes none of the psychoactive compounds from the cannabis plant.

Johnson & Johnson’s Akeega is approved for treating advanced prostate cancer with a BRCA mutation. The drug, which interferes with a way cancer cells repair themselves, will compete against recently approved drugs from Pfizer and partners AstraZeneca and Merck.

Novo Nordisk isn’t putting all of its weight loss drug hopes in the same basket. Its acquisition of Inversago Pharma for up to $1 billion will bring a biotech whose lead drug candidate has early clinical trial data showing significant weight loss.

Johnson & Johnson’s stable of multiple myeloma drugs has grown to five products with the FDA approval of Talvey. The bispecific antibody is the first in a new class of new drugs designed to go after the novel multiple myeloma target GPRC5D.

“Botched math” in calculating clinical data for an autoimmune disease drug is one of the allegations Nektar Therapeutics makes in a legal complaint against Eli Lilly. The biotech claims its former partner tried to disadvantage the asset in order to advance development of another drug it acquired in a billion dollar deal.

Novo Nordisk weight-loss drug Wegovy reduced the risk of major cardiovascular events by 20% in a large clinical trial. The company said the results support expanding the drug’s label, but the looming question is whether the data will persuade payers to cover the pricey chronic therapy.

Vistagen Therapeutics’ fast-acting nasal spray drug, fasedienol, hit primary and secondary goals of an abbreviated late-stage study in social anxiety disorder. Vistagen’s stock price rocketed skyward on the results, which support progression to another Phase 3 study.

Izervay, a drug developed by Astellas Pharma subsidiary Iveric Bio, is now the second approved therapy for the degenerative vision-loss disorder geographic atrophy. The regulatory decision comes as safety concerns emerge around the first therapy for the disease, an Apellis Pharmaceuticals product.

Clinical trial design will be one of the topics discussed at the upcoming INVEST Digital Health conference in Dallas on October 26. THREAD Clinical Trial Design Director Noah Goodson highlighted some of the milestones the sector has seen in recent years.

Long Covid continues to afflict patients and mystify clinicians. The National Institutes of Health is trying to identify potential treatments and it is launching a series of clinical trials that will assess a wide range of therapeutic interventions.