Bristol Myers Squibb drug Augtyro now has a tumor-agnostic approval permitting the drug to be used to treat all solid tumors positive for NTRK gene fusions. Augtyro came from BMS’s $4.1 billion acquisition of Turning Point Therapeutics.

The post New FDA Nod Brings Bristol Myers Squibb Cancer Drug to a Wider Range of Solid Tumors appeared first on MedCity News.

A Pfizer gene therapy for Duchenne muscular dystrophy failed its Phase 3 clinical trial. But analysts draw distinctions between that therapy and Sarepta Therapeutics’ Elevidys, which awaits an FDA decision that could grant it full regulatory approval.

The post Pfizer & Sarepta Gene Therapies Both Failed Phase 3, But Analysts Expect Sarepta Will Win Approval appeared first on MedCity News.

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.

Iqirvo failed as a treatment for the fatty liver disease MASH, but the drug is now FDA approved in primary biliary cholangitis. An Intercept Pharmaceuticals drug already treats this rare liver disease and Gilead Sciences is poised to compete with its PBC drug approaching an FDA decision this summer.

The post Ipsen, Genfit Drug That Missed in MASH Wins FDA Approval in a Much Rarer Liver Disease 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.

An experimental Moderna combination vaccine for influenza and Covid-19 showed in a pivotal study that it can elicit a higher immune response compared to vaccines that are already available to protect against infection from both viruses.

The post Moderna’s Flu & Covid-19 Combination Vaccine Succeeds in Phase 3 Clinical Trial appeared first on MedCity News.

Geron Corporation’s Rytelo is now FDA approved for treating anemia caused by myelodysplastic syndromes. The new drug will compete against Reblozyl, a blockbuster Bristol Myers Squibb medicine.

The post Geron Drug Lands a First-in-Class FDA Approval in Type of Blood Cancer appeared first on MedCity News.

Rapport Therapeutics’ IPO came with a concurrent private placement that boosted the biotech’s cash haul. The new capital will support clinical development of a lead drug with a novel approach to focal epilepsy, a form of the disorder that affects 1.8 million people in the U.S.

The post Rapport’s Stock Market Debut Reaps $154M as Novel Epilepsy Med Heads for Mid-Stage Test appeared first on MedCity News.

GSK’s Blenrep has pivotal clinical trial results showing the multiple myeloma therapy reduced the risk of disease progression or death by nearly 50%. The results were presented Sunday during the annual meeting of the America Society of Clinical Oncology.

The post GSK’s Quest to Bring Myeloma Drug Back to the Market Stops for a Data Drop at ASCO appeared first on MedCity News.

AstraZeneca’s Tagrisso and Imfinzi helped patients live longer in separate pivotal clinical trials evaluating the drugs in two different types of lung cancer. The results will be presented Sunday during the annual meeting of the American Society of Clinical Oncology.

The post At ASCO, AstraZeneca Adds Data Supporting Expanded Use of Its Drugs in Lung Cancer appeared first on MedCity News.

Moderna’s mResvia is now the third FDA-approved RSV vaccine and the first one based on mRNA technology. It will compete for market share against RSV shots from GSK and Pfizer that won their FDA nods last year.

The post FDA Approves Moderna RSV Vaccine, an mRNA Alternative to GSK and Pfizer Products appeared first on MedCity News.

Gilead Sciences reported Trodelvy’s Phase 3 test in metastatic urothelial cancer fell short of showing a statistically significant benefit on the overall survival main goal. The antibody drug conjugate had received accelerated FDA approval for this type of cancer in 2021.

The post Gilead Sciences ADC Flunks Its Confirmatory Test in Advanced Bladder Cancer appeared first on MedCity News.

Merck’s EyeBio acquisition brings a lead program ready for pivotal testing in diabetic macular edema. The therapy could pose competition to Eylea, the blockbuster Regeneron Pharmaceuticals eye drug.

The post Merck Sees Pipeline Diversification Opportunity in $1.3B EyeBio Acquisition appeared first on MedCity News.

Brensocatib, a drug Insmed licensed from AstraZeneca, met the main goal of a Phase 3 test in non-cystic fibrosis bronchiectasis. Insmed plans to seek regulatory approvals in this indication while also continuing to develop the small molecule for other inflammatory disorders.

The post Insmed’s Trial Results Pave Way for FDA Filing in a Lung Disease With No Approved Therapies appeared first on MedCity News.

Johnson & Johnson is acquiring a bispecific antibody that Numab Therapeutics engineered to address two pathways associated with the inflammation and itching of atopic dermatitis. It’s J&J’s second immunology acquisition this month.

The post J&J Adds Another Bispecific Antibody for Atopic Dermatitis With $1.25B Acquisition appeared first on MedCity News.

Biogen’s acquisition of Human Immunology Biosciences brings a drug candidate with potential applications in multiple immune-mediated disorders. The deal comes just prior to the scheduled presentation of clinical data in two of them.

The post Biogen Immunology Strategy Comes Into Focus With $1B HI-Bio Acquisition appeared first on MedCity News.

To win the battle against rare diseases, drug developers must work hand-in-hand with patients.

The post Allying With Patients to Make Every Cure Possible appeared first on MedCity News.

The PTC Therapeutics drug Translarna failed its confirmatory study in Duchenne muscular dystrophy. Analysts say the European Commission’s decision to not adopt the Committee on Medicinal Products for Human Use’s negative opinion on the drug is unusual, if not unprecedented.

The post European Commission Decision Is a Reprieve for PTC Therapeutics’ Rare Muscle Disease Drug appeared first on MedCity News.

Rapport Therapeutics’ lead program could offer more targeted epilepsy treatment compared to currently available anti-seizure medications, which introduce many side effects. The IPO will support plans to develop the oral small molecule in focal epilepsy as well as peripheral neuropathic pain and bipolar disorder.

The post CNS-Focused Rapport Therapeutics Preps IPO to Back Phase 2-Ready Epilepsy Drug appeared first on MedCity News.

Amgen’s Imdelltra has FDA approval in extensive-stage small cell lung cancer, making it the first bispecific T cell engager approved for treating this type of cancer. Analysts project the new Amgen drug will become a blockbuster seller.

The post Amgen Drug for Aggressive Type of Lung Cancer Wins Accelerated FDA Approval appeared first on MedCity News.

Johnson & Johnson’s Proteologix acquisition adds a pipeline of bispecific antibodies, including an experimental treatment for asthma and atopic dermatitis. It’s the latest M&A deal involving molecules addressing TSLP, a key signaling protein involved in inflammation.

The post J&J Joins Chase for Hot Immunology Target With $850M Proteologix Acquisition appeared first on MedCity News.

Blackstone Life Sciences startup Uniquity Bio emerged from stealth with an in-licensed drug candidate that could treat a wide range of immunology and inflammation indications. The antibody’s target puts it in competition with drugs from AstraZeneca, Pfizer, and Sanofi.

The post Blackstone Unveils Immunology Startup With $300M and a Phase 2-Ready Drug From Merck appeared first on MedCity News.

Takeda Pharmaceutical has secured an option on an Alzheimer’s disease immunotherapy from AC Immune that is on track to report its first Phase 2 data later this quarter. Exercising the option would make Takeda responsible for Phase 3 testing of the therapy, putting AC Immune in line for up to $2.1 billion in milestone payments.

The post Takeda Commits $100M for Chance to Bring Immunotherapy to Alzheimer’s Disease appeared first on MedCity News.

Eli Lilly-partnered Lycia Therapeutics will use its Series C financing to continue developing its internal pipeline of protein-degrading drugs. The startup is working to reach the clinic with drugs for autoimmune and inflammatory disorders.

The post Lycia Therapeutics Lands $106M for Protein Degraders That Treat Autoimmune & Inflammatory Diseases appeared first on MedCity News.

Amgen is confident its injectable obesity drug would have a differentiated profile compared to medications now on the market from Novo Nordisk and Eli Lilly and it’s now preparing for Phase 3 testing. But an oral obesity drug in Amgen’s pipeline will not advance beyond Phase 1.

The post Amgen Puts Its Weight Behind Obesity Drug With Potential Edge Over Lilly, Novo Nordisk Meds appeared first on MedCity News.

Pepper Bio has licensed rights to lerociclib, a molecule that G1 Therapeutics designed to offer dosing and safety advantages over approved therapies in the same drug class. EQRx previously held rights to the drug, but returned them after the company was acquired last year.

The post Pepper Bio’s ‘Google Maps for Drug Discovery’ Finds Way to Potential New Liver Cancer Drug appeared first on MedCity News.

The FDA approved X4 Pharmaceuticals drug Xolremdi for treating WHIM syndrome. The molecule, which addresses the underlying cause of this rare immunodeficiency, was licensed from Sanofi’s Genzyme subsidiary.

The post Drug Licensed from Sanofi Becomes First FDA-Approved Therapy for Ultra-Rare Primary Immunodeficiency appeared first on MedCity News.

Ono Pharmaceutical’s acquisition of Deciphera Pharmaceuticals adds to a cancer drug pipeline that already spans multiple modalities. Deciphera develops drugs that target the on-off switch of enzymes that drive cancer cell growth.

The post Ono Pharma’s Plan to Become a Global Player Picks Up a New Piece With $2.4B Deciphera Acquisition appeared first on MedCity News.

The FDA has approved Beqvez, a Pfizer gene therapy developed for moderate-to-severe hemophilia B. The one-time treatment carries a $3.5 million price tag, the same as a CSL Behring gene therapy already available for treating the inherited bleeding disorder.

The post Pfizer’s First Gene Therapy Approval Sets Up a Showdown With CSL in Hemophilia B appeared first on MedCity News.

Day One Biopharmaceuticals drug Ojemda is now FDA-approved for advanced pediatric low-grade glioma, the most common type of brain cancer in children. The regulatory decision for Ojemda covers a broader swath of patients than a drug combination from Novartis approved for treating this childhood cancer.

The post FDA Drug Approval Marks a New Day for Treating Pediatric Brain Cancer appeared first on MedCity News.