Know What's Happening in Pharma
Know What's Happening in Pharma
Eli Lilly’s oral GLP‑1 pill and triple‑agonist trial reshape obesity drug race
The FDA approved Lilly’s oral GLP‑1 weight‑loss tablet Foundayo (orforglipron), which can be taken without food or water restrictions. In a Phase 3 trial, Lilly’s experimental injectable triple‑hormone agonist retatrutide achieved a 28.7% average body‑weight reduction, outpacing Novo Nordisk’s next‑generation candidates.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma
Bayer announced that its phase‑III PEACE‑3 trial demonstrated a statistically significant overall‑survival benefit for the combination of Xofigo (radium‑223) and enzalutamide versus enzalutamide alone in patients with metastatic castration‑resistant prostate cancer (mCRPC) and bone metastases. Median overall survival extended to 38.2 months compared with 32.6 months, reflecting a 24% reduction in the risk of death. The study also confirmed earlier improvements in radiographic progression‑free survival (19.4 versus 16.4 months). Results will be presented at ASCO GU 2026 and published in the Annals of Oncology.
PMV Pharmaceuticals reported Phase I results of rezatapopt, a small‑molecule p53 reactivator, in 77 patients with advanced solid tumors carrying the TP53 Y220C mutation. The oral drug was generally well tolerated, with few dose‑limiting toxicities, allowing the selection of a...
New: Generate caps a strong month for #biotech IPOs with $400M offering https://t.co/HzslZOPcwM by @gwendolynawu $GENB $AZN $AMGN #IPO
The FDA has warned consumers against buying ULTRA ADVANC3 on Amazon and ULTRA ADVANC3 GOLD on NaturistaRex after laboratory tests revealed hidden prescription drugs. The supplements contain undeclared dexamethasone, diclofenac, and methocarbamol, each carrying serious health risks. The agency advises immediate discontinuation and...
Fulgent Genetics reported Q4 2025 revenue of $84.1 million, a sequential increase and strong year‑over‑year growth across its Precision Diagnostics and Biopharma segments. Non‑GAAP gross margin improved to 44.3% while operating expenses fell, delivering a $0.7 million adjusted EBITDA and a $4.5 million...
Senator Ron Johnson (R‑WI) is urging Congress to draft a “Right to Try 2.0” bill that would force the FDA to grant broader, faster access to experimental therapies for rare diseases. FDA Commissioner Martin Makary pushed back, defending the agency’s...
The U.S. Department of Justice filed an amicus brief supporting AbbVie in its effort to overturn a Colorado statute that prohibits pharmaceutical manufacturers from limiting discounts under the federal 340B drug pricing program when hospitals use contract pharmacies. The Colorado...
Researchers from Japan Tobacco and D‑Wave have unveiled a hybrid framework that merges deep generative models with quantum annealing to design drug‑like molecules. The system introduces a Neural Hash Function that enables binarisation for quantum processing while preserving gradient flow....
The FDA granted accelerated approval to zongertinib (Hernexeos) for adults with unresectable or metastatic non‑squamous NSCLC that carry activating HER2 TKD mutations, extending its use to treatment‑naive patients. The decision rests on the Beamion LUNG‑1 trial, which reported a 76%...
RIKEN scientists have shown that an intrinsically disordered region (IDR) of the DEAD‑box helicase DDX3X confers selective binding to specific mRNA structures, a mechanism uncovered using solution NMR spectroscopy. The discovery clarifies how DDX3X distinguishes target transcripts, linking its specificity...
ARUP Laboratories has unveiled the Innovation Central Laboratory, a dedicated hub for co‑development with pharmaceutical and biotech partners. The facility is designed to validate technologies and move next‑generation diagnostics from concept through to commercial readiness. It will host projects ranging...
The FDA approved KOMZIFTI (ziftomenib) on November 13 2025 as an oral 600 mg capsule for adult patients with relapsed or refractory acute myeloid leukemia (AML) harboring an NPM1 mutation. Approval rests on the single‑arm KO‑MEN‑001 trial, which enrolled 112 participants across seven...
The January 2026 Patent Highlights roundup spotlights a wave of new intellectual‑property activity across several cutting‑edge drug discovery areas. Notable filings include lysine‑directed covalent inhibitor chemotypes, strategies to balance potency with drug‑like properties, refined target‑selection frameworks, dozens of Polθ synthetic‑lethal patents...
The Centers for Medicare & Medicaid Services entered the third year of its Medicare drug price negotiation program, selecting 15 drugs—including, for the first time, therapies covered under Medicare Part B. Patient advocacy groups have until March 1 to submit written comments...
(Hopefully) Final word today on the Makary $QURE (or was it $RGNX) thing: Bottom line: Makary shouldn't be talking publicly about unresolved drug applications. Uniqure doesn't even have an application submitted; and RegenxBio's CRL letter isn't posted to the FDA's...
Bristol Myers Squibb reported that its antibody‑drug conjugate iza‑bren, licensed from Chinese partner SystImmune, achieved statistically significant improvements in progression‑free and overall survival versus chemotherapy in a Phase III trial for advanced triple‑negative breast cancer. The study, conducted in mainland China...
You've got to watch this Makary interview on CNBC. He attacks a drug, hits the Old FDA and circles all the wagons around Vinay Prasad, who "loves President Trump" and faces a fatwa (WSJ). Makary is getting major props for...
FDA Commissioner Marty Makary defends rare disease drug rejections, Vinay Prasad "Vinay Prasad is a genius,” Makary said. “He’s on loan from UCSF. He’s published over 500 scientific peer-reviewed articles, and some of the drugs they are criticizing him for...
An international team led by the University of Birmingham, University Hospitals Birmingham NHS Foundation Trust and Bristol Myers Squibb has produced the most detailed spatial atlas of pancreatic ductal adenocarcinoma (PDAC) to date. Using spatial transcriptomics and spatial molecular imaging...
![[Comment] Impact of Adding Hormone Therapy to Postoperative Radiotherapy in Prostate Cancer](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/f1452ec53e5201a4460c9aaf141538d3.webp)
Adding androgen deprivation therapy (ADT) to definitive radiotherapy improves overall survival for men with high‑risk prostate cancer, but its benefit when combined with postoperative radiotherapy (PORT) after prostatectomy is less clear. Randomised trials such as GETUG‑AFU 16, SPPORT, and the RADICALS‑HD...
Researchers at the University of Macau have demonstrated the first in‑situ synthesis of covalent organic frameworks (COFs) inside lysosomes of cancer cells, using acidic pH‑driven imine condensation of TAPB and DMTP. The crystalline UMCOF1 particles rupture lysosomal membranes, liberating ferrous...
The FDA announced it will default to a single pivotal trial for new drug applications, extending a practice long used in oncology and rare‑disease approvals. In 2024, 66% of new molecular entities were cleared based on one trial, signaling a...
The latest episode of BioSpace’s Denatured podcast spotlights a growing partnership between ALS patients and pharmaceutical developers, featuring insights from EverythingALS founder Indu Navar and VectorY Therapeutics CMO Dr. Olga Uspenskaya. The discussion highlights how patient‑driven collaborations are accelerating trial timelines...
Sarepta Therapeutics announced that CEO Douglas Ingram will retire by the end of 2026, triggering a formal succession search. The move follows a turbulent 2025 in which the company’s flagship gene therapy Elevidys was linked to two patient deaths, prompting...
The U.S. Food and Drug Administration rejected a rare‑disease cell therapy that had already secured approval in Europe, despite earlier internal support from the agency. The decision comes under the FDA’s new leadership and has sparked debate over whether the...
$QURE down this morning on comments made by FDA Commissioner Marty Makary to CNBC's Becky Quick. In a discussion about rare-disease drug approvals and Vinay Prasad, Makary said this: "I think there has been a bit of an effort to...
We'll be gearing up today's webinar on biotech venture capital at the top of the hour. Great guests, great topics. If this is a central issue in your life, tune in. Thousands have signed up already. https://t.co/c16y04mFsD
In Episode 9 of Drug Dealin, hosts Kenny and Shibu demonstrate how patients can use publicly available tools like ClinicalTrials.gov and AI language models to uncover novel treatment options, focusing on a hypothetical Parkinson’s disease case inspired by a TV...
Direct-to-consumer gut microbiome assessments are unreliable, problematic: a review of 7 companies with significant methodologic issues https://t.co/atwmZUNjVE https://t.co/SqlMljuW0y
Head-to-head trial of the oral GLP-1 drugs in people with T2D. Orforglipron was superior https://t.co/9B7RXIgdJY
Atara Biotherapeutics’ stock jumped 20% after STAT reported that internal FDA inconsistencies may have driven the rejection of its EBV‑positive PTLD cell therapy, Ebvallo. The FDA denied the U.S. application citing insufficient evidence of effectiveness, yet former reviewers said data...
We're making headway for off the shelf preventive cancer vaccines in people with hereditary forms of cancer, e.g. Lynch syndrome https://t.co/F3LqKS46BQ
Boehringer Ingelheim announced a potential $500 million investment in UK biotech Sitryx to obtain global rights to an undisclosed small‑molecule immunology programme. The partnership covers upfront fees, milestones and royalties, giving Boehringer full responsibility for research, clinical development and commercialization. Sitryx’s...
ViVE 2026 in Los Angeles marked a decisive shift from digital ambition to operational accountability across the health ecosystem. Leaders emphasized that AI adoption now hinges on governance, measurable ROI, and clinician trust, while interoperability is being treated as core...
Researchers have captured the fleeting i‑DNA structure inside living cells, showing it acts as a molecular switch for genes that drive cancer. The study demonstrates that i‑DNA formation and resolution are tightly timed during DNA replication, influencing gene expression and...
Continuous cardiac monitoring via wearable ECG devices is reshaping clinical trials and post‑market care. Recent studies show that 14‑day monitoring after cardiac surgery uncovered atrial fibrillation in 24% of patients, many of which were missed until three‑month follow‑ups. In epilepsy...
Pfizer has signed a global gene‑editing partnership with Beam Therapeutics, reviving its genetics ambitions after withdrawing the hemophilia B therapy Beqvez a year earlier. The agreement includes a $300 million upfront payment and up to $1.05 billion in milestones, granting Pfizer exclusive worldwide...
$RVMD was collaborating with $TNGX in PRMT5, but now it's working with $BMY (remember that ex MRTX project?). Via @ByMadeleineA @ApexOnco -> https://t.co/mGZjKOiB7p (And $AMGN still dose optimising.)
Cara Medical announced that the U.S. FDA granted 510(k) clearance for its CARA System, a non‑invasive solution that creates patient‑specific 3‑D visualizations of the cardiac conduction system using CTA data. The platform pairs the CARA Metis™ Simulator for pre‑procedural mapping...
Takeda is leveraging real‑world evidence (RWE) and human‑focused AI to reshape clinical trial design, patient access, and global market strategies. Chief Medical Officer Awny Farajallah highlights three pillars: robust evidence generation, capability building, and early patient access. RWE is used...
![[Comment] Oral GLP-1 Receptor Agonists: Competition for Efficacy and Tolerability](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/ce10e510d7294b419a4a07dfad918da4.webp)
Oral GLP-1 receptor agonists have moved from injectable peptides to a tablet form, highlighted by oral semaglutide combined with the absorption enhancer SNAC. The formulation requires patients to fast overnight, take the dose with a small amount of water, and...
GlaxoSmithKline announced that China’s National Medical Products Administration has accepted the New Drug Application for linerixibat and placed it under priority review for treating cholestatic pruritus in primary biliary cholangitis (PBC). The filing is backed by the Phase III GLISTEN trial,...
Buzz Health President Joseph Kleiman says TrumpRx is advancing drug‑price transparency by giving consumers early visibility into prescription costs. He notes that real‑time pricing can help patients compare options but also warns that prices are dynamic and may change before...
Otsuka America became the first major pharmaceutical company to back psychedelic‑inspired medicines in 2020 through a partnership with COMPASS Pathways, and deepened that commitment by acquiring Mindset in 2023. The company is applying its long‑standing CNS expertise to develop serotonergic...
Merck Animal Health announced that the U.S. FDA has approved Numelvi (atinvicitinib) for controlling pruritus linked to allergic dermatitis in dogs six months and older, with commercial availability slated for spring 2026. Numelvi is a second‑generation JAK inhibitor that demonstrates...
The FDA has rejected Regenxbio’s gene therapy for mucopolysaccharidosis type 2, citing a need for additional data that could postpone approval by years. This decision adds to at least four other recent rejections of cell or gene therapies targeting deadly rare...
Mahzi Therapeutics has administered the first dose of its investigational gene therapy MZ-1866 in the Phase I/II UNITE study for Pitt‑Hopkins syndrome. The open‑label, multicentre trial will enroll roughly 12 genetically confirmed patients across sites in Spain, Israel and the...
Women’s HealthX (WoHX) will convene 750 pharma, biotech, hospital, insurer, startup and government leaders in Boston on December 3‑4, 2026. The two‑day summit aims to close the sex‑difference data gap by showcasing evidence‑based technologies, AI tools and digital therapeutics across the...
The Trump administration launched TrumpRx, a website aggregating manufacturer discounts on 43 brand‑name drugs and touting presidential credit for lower patient costs. Early analysis shows that 20 of the listed products already have cheaper generic versions, and the portal deliberately...
argenx announced topline Phase III ADAPT OCULUS data for Vyvgart (efgartigimod alfa + hyaluronidase‑qvfc) in adults with ocular myasthenia gravis. The trial met its primary endpoint, delivering a 4.04‑point improvement in Myasthenia Impairment Index PRO ocular scores versus 1.99 points for placebo at...
