Avidity Biosciences’ RNA therapy for facioscapulohumeral muscular dystrophy has preliminary clinical data showing it knocked down expression of a gene that causes this rare disease. More details will be presented at a medical conference this week.

The post Avidity Bio’s RNA Therapy Flashes Early Signs of Treating a Muscular Dystrophy’s Root Cause appeared first on MedCity News.

Skye Bioscience’s nimacimab blocks CB1, the same receptor targeted by an obesity drug candidate now in the hands of Novo Nordisk. But Skye contends its Phase 2-ready antibody drug has several advantages over the small molecule that joined Novo’s pipeline in a deal valued at $1 billion.

The post Skye Bioscience Falls Short in Glaucoma Trial, But It’s Full Steam Ahead for Obesity Program appeared first on MedCity News.

Novo Nordisk’s focus on diabetes and obesity shape its business development work, but the company is also exploring other areas. Top Novo Nordisk M&A executive John McDonald discussed his company’s strategy during a session at the BIO conference in San Diego.

The post At BIO, Novo Nordisk’s Top Dealmaker Talks M&A Strategy and What’s Next After GLP-1s appeared first on MedCity News.

ImmunityBio therapy Anktiva is now FDA approved for treating patients with non-muscle invasive bladder cancer. The novel immunotherapy works by sparking activity from three types of immune cells.

The post FDA Approves Immunotherapy That Can Spare Bladder Cancer Patients From Radical Surgery appeared first on MedCity News.

Cancer drug developer Boundless Bio is a pioneer in therapies that target extrachromosomal DNA. The IPO cash will support its pipeline, including a lead program on track to report preliminary data later this year.

The post Boundless Bio’s ‘BOLD’ IPO Reels In $100M for a New Kind of Cancer Drug appeared first on MedCity News.

Mirador Therapeutics applies machine learning to human data to discover new immunology and inflammation drugs. The startup’s management includes several from the executive team of Prometheus Biosciences, an immunology biotech bought by Merck last year.

The post Precision Medicine Startup Mirador Unveils $400M for R&D of New I&I Drugs appeared first on MedCity News.

Contineum Therapeutics, which emerged from a Verant Ventures startup creation engine, has filed paperwork for an IPO. The biotech’s clinical-stage pipeline spans multiple sclerosis, idiopathic pulmonary fibrosis, and depression.

The post With MS & Fibrosis Trials Planned, J&J-Partnered Contineum Now Preps for IPO appeared first on MedCity News.

With pimavanserin’s Phase 3 failure in schizophrenia, Acadia Pharmaceuticals said it will no longer run clinical trials for the drug, whose lone approval is for treating psychosis from Parkinson’s disease. The FDA previously rebuffed regulatory submissions for the drug in dementia and Alzheimer’s disease.

The post Schizophrenia Trial Failure Ends Acadia’s Efforts to Expand Use of Its Flagship Drug appeared first on MedCity News.

A Janux Therapeutics T cell engager has early clinical data showing improvement in cases of advanced prostate cancer, validating the technology platform that produced the therapy. Janux is now leveraging its soaring stock price to raise $175 million in a stock offering.

The post Janux Rides Its TRACTr to Validation in Prostate Cancer and More Solid Tumors appeared first on MedCity News.

The weight loss achieved by Viking Therapeutics’ obesity drug tops results posted by Eli Lilly’s Zepbound in its pivotal test. But Viking still needs to show its results can hold up in a larger Phase 3 clinical trial.

The post Viking Therapeutics Obesity Drug’s Data Raise Best-in-Class Expectations appeared first on MedCity News.

Startup TrX1 develops cell therapies that function like a particular type of regulatory T cell, or Treg, whose role includes dampening inflammation and inducing long-term tolerance. Based on research from Stanford, TrX1’s lead program is a potential therapy for preventing graft-versus-host disease.

The post Cell Therapy Startup With Stanford Roots Unveils $75M to Seek Inflammation Cures appeared first on MedCity News.

Galapagos is a latecomer to CAR T-therapy, but the drugmaker hopes its technology for producing cell therapies at a clinical site will help it stand out. Galapagos has encouraging preliminary data from two early-stage programs and it just begun Phase 1/2 testing of its third cancer cell therapy.

The post Galapagos Counts On Cocoon to Help It Catch Up in Cancer Cell Therapy appeared first on MedCity News.

Pfizer already reported its experimental drug for hemophilia A and B met a pivotal study’s goals of reducing bleeding episodes. During the annual meeting of the American Society of Hematology, the company presented a full look at the results, which are expected to support regulatory submissions soon.

The post On Heels of ASH Data, Pfizer Eyes FDA Submission for New Hemophilia Drug appeared first on MedCity News.

Ventyx Biosciences is stopping work on its lead drug candidate in plaque psoriasis and psoriatic arthritis. Despite achieving the goals of its Phase 2 test in psoriasis, the results are short of those achieved by big pharmaceutical companies with molecules that address the same target.

The post Ventyx Drops Drug for Psoriasis as Data Look Uncompetitive in Tight TYK2 Field appeared first on MedCity News.

European Union regulators ordered Illumina to divest Grail. Illumina is appealing antitrust findings in Europe and the U.S., but the DNA sequencing giant revealed that divesting all or part of Grail is an option even if it wins both legal challenges.

The post EU Orders Grail Sale; Illumina Continues Appeals But Plans for Multiple Outcomes appeared first on MedCity News.

Acquiring Mirati Therapeutics brings Bristol Myers Squibb Krazati, one of two FDA-approved therapies addressing a KRAS cancer mutation. The deal comes as BMS looks to add revenue-generating products as patent expirations loom for several of its cancer products.

The post Bristol Myers Squibb Bolsters Its Cancer Presence With $4.8B Mirati Acquisition appeared first on MedCity News.

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.

The post Once a Blockbuster Prospect, Is Travere’s Kidney Drug on Track to Become a Bust? appeared first on MedCity News.

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.

The post FDA Rejects ARS Pharma’s Nasal Spray Alternative to Injectable Epinephrine appeared first on MedCity News.

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.

The post Sanofi Puts Up $125M to Partner on Oral Drug That Could Be Dupixent’s Successor appeared first on MedCity News.

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.

The post Here’s Why Novartis Is Paying $500M to Acquire a Preclinical RNA Startup appeared first on MedCity News.

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.

The post Travere Therapeutics Drug for Rare Kidney Disease Falls Short in Pivotal Test appeared first on MedCity News.

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.

The post Merck’s $11B Prometheus Acquisition Brings IBD Drug & Precision Medicine Platform appeared first on MedCity News.

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.

The post Startup Function Oncology Aims to Make CRISPR Part of Next Revolution in Cancer appeared first on MedCity News.

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 post Patient Deaths and Partial Hold Prompt Gossamer Bio to Stop Work on Cancer Drug appeared first on MedCity News.

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.

The post FTC Orders Illumina to Divest Grail, Says Deal Stifles Cancer Test Competition appeared first on MedCity News.

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.

The post FDA Approval of Travere Kidney Disease Drug Sets Up Blockbuster Expectations appeared first on MedCity News.

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.

The post MD Anderson’s Tech Puts Replay on Fast Path to Clinic With TCR NK Cell Therapies appeared first on MedCity News.

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.

The post Scripps Spinout Belharra Unveils $130M to Catch Next Big Wave of Drug Discovery appeared first on MedCity News.

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.

The post Neurocrine Commits $175M to Voyager in New Neuro Gene Therapy Alliance appeared first on MedCity News.

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.

The post FDA Approval of Mirati Lung Cancer Drug Sets Stage for Competition With Amgen appeared first on MedCity News.