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.

Sanofi is interested in Recludix Pharma’s approach to treating inflammation with an oral small molecule that targets a protein thought to be undruggable. If it works, the drug could have safety and dosing advantages over currently available biologic drugs, including blockbuster Sanofi drug Dupixent.

Novartis’s acquisition of DTx Pharma brings a lead program for a rare disease with no FDA-approved therapies. But the deal could also help the pharma giant bring new RNA therapies to new places in the body, a top Novartis executive explained.

Sparsentan failed to meet the main goal of a Phase 3 test in focal segmental glomerulosclerosis (FSGS). Nevertheless, Travere Therapeutics says the totality of data points to patient improvement in the rare kidney disorder and the biotech will talk with regulators about a potential path forward.

Merck is acquiring Prometheus Biosciences and a lead drug candidate in development for inflammatory bowel disorders. The deal comes as Merck looks for drug prospects that could make up for revenue declines facing blockbuster seller Keytruda, which faces patent expirations in coming years.

Function Oncology goes beyond next-generation sequencing with a CRISPR-based technology that identifies targets for a patient’s cancer regardless of whether or not they are mutated. Now out of stealth, the company will present data validating its approach during the American Association for Cancer Research annual meeting.

Two fatalities were reported in an early-stage study testing a Gossamer Bio drug intended to treat a rare cancer affecting the central nervous system. Due to the adverse events and a partial FDA clinical hold, Gossamer has decided to stop work on the program.

The Federal Trade Commission has ordered Illumina to divest Grail, the liquid biopsy company it acquired for more than $7 billion. The agency said Illumina’s proposed remedies are insufficient and the tie-up is likely to reduce competition in the R&D and commercialization of new cancer tests.

Travere Therapeutics drug Filspari won accelerated FDA approval to treat a rare disorder that can lead to kidney failure. The small molecule is also in late-stage testing for another rare kidney disease; analysts say each indication represents a blockbuster opportunity.

Syena, the latest company formed by startup creator Replay, is developing cancer treatments based on a type of immune cell called a natural killer cell. This NK cell therapy technology, licensed from The University of Texas MD Anderson Cancer Center, could overcome many of the limitations facing first-generation cell therapy.

Versant Ventures-backed Belharra Therapeutics has launched with $130 million in funding and a partnership with Roche subsidiary Genentech. The startup’s technology analyzes proteins in the context of the entire cell, enabling it to discover molecules that bind to targets previously deemed undruggable.

Neurocrine Biosciences is paying $175 million up front for rights to a preclinical Voyager Therapeutics gene therapy that could put the biotech in competition with Eli Lilly among other companies. Voyager could earn up to $4.2 billion in milestone payments.

Amgen’s Lumakras became the first FDA-approved drug that addresses the elusive KRAS mutation, but Mirati believes its newly approved therapy, Krazati, could be better. The small molecule’s features include the ability to penetrate into the brain, where it can address cancer that has spread to the central nervous system.

Telaria is developing a gene therapy for the rare skin disorder recessive dystrophic epidermolysis bullosa. The company was formed by Replay, a gene therapy biotech that forms subsidiaries that leverage its platform technologies for writing and delivering genetic medicines.

Capstan Therapeutics, a University of Pennsylvania spinout, is now out of stealth with technology that could enable new medicines that work by engineering cells inside of a patient. Penn biotech pioneers Carl June and Drew Weissman are among the scientific co-founders of this startup, which aims to develop therapies for fibrosis, cancer, inflammation, and genetic diseases.

Neurocrine Biosciences is acquiring Diurnal Group, a U.K-based biotech that has commercialized two endocrine disorder drugs. Neurocrine’s top-selling product is a blockbuster drug for an involuntary muscle movement disorder.

Acadia Pharmaceuticals drug Nuplazid failed to win FDA approval for the treatment of psychosis in Alzheimer’s disease patients. The regulator said that the data submitted were not from an adequate and well-controlled study and the company must run another clinical trial.

Private equity firm KKR teamed up with OMX Ventures to lead the seed investment in Replay, a company developing a suite of technologies that could overcome capacity limitations of adeno-associated viruses used for genetic medicines delivery. Duchenne muscular dystrophy is among the disease targets of the new startup.

Vertex Pharmaceuticals already has a cell therapy candidate for type 1 diabetes but the company says acquiring rival ViaCyte will provide it with complementary assets and technologies. The two companies already share a partner in common: both have therapeutic candidates that use the gene-editing capabilities of CRISPR Therapeutics.

Human Longevity, Inc., a startup founded by genomics pioneer J. Craig Venter, plans to join the public markets via a SPAC merger. If the business combination closes, HLI would receive about $345 million for continued commercialization of its platform that uses genomics, AI, and other technologies for early disease detection and other strategies intended to help people live longer.

Drug prices are on the minds of patients and politicians. The November election is expected to tip the balance of power in Congress and a panel held during the Biotechnology Innovation Organization’s annual meeting discussed what that change could mean for drug price legislation.

Biopharmaceutical companies and public health officials have learned a great deal about pandemic response in the last two years. A panel discussion during the annual meeting of the Biotechnology Innovation Organization explored some of the lessons from Covid-19 and how they can prepare us for the next pandemic.

Bristol Myers Squibb is splashing out $4.1 billion to acquire Turning Point Therapeutics, a clinical-stage biotech whose lead drug is currently in pivotal testing as a treatment for certain lung cancers. Recently reported preliminary data suggest the small molecule could have advantages over currently available drugs in the same class from rivals Pfizer and Roche.

Aspen Neurosciences is developing a cell therapy that uses a patient’s own stem cells to develop a personalized treatment for Parkinson’s disease. The approach is slightly different than that of Bayer, whose experimental Parkinson’s cell therapy is made from stem cells sourced from healthy donors.

Scientists developing new RNA drugs need to understand how proteins bind to these molecules. Eclipsebio’s technology provides that analysis and the biotech startup now has a Series A round of funding to scale up its business.

Creyon Bio is the latest company to launch with artificial intelligence technology for drug R&D. The startup claims its technology can provide sufficient safety and toxicity data to enable a therapy to bypass the animal research currently needed before clinical trials.

Endeavor BioMedicines aims to kill two birds with one stone—that stone being a small molecule drug. Taladegib targets a cell signaling pathway associated with both cancer and idiopathic pulmonary fibrosis, and the biotech will apply its Series B round toward clinical tests of the molecule on both fronts.

The FDA placed the partial hold on the study last November following a patient death suspected to be linked to a known complication of leukemia therapies. Kura Oncology said it has reached agreement with the FDA on a mitigation strategy for that complication.

Blueprint Medicines is paying $250 million up front to acquire Lengo Therapeutics, developer of targeted cancer therapies. The startup’s lead drug candidate is on track to begin human testing in non-small cell lung cancer, and Blueprint believes the small molecule has advantages over new targeted therapies from Johnson & Johnson and Takeda Pharmaceutical.