Subcutaneous Tepezza Demonstrates Phase III Efficacy in Thyroid Eye Disease
Amgen reported that its subcutaneous formulation of teprotumumab‑trbw (Tepezza) met the primary endpoint in a phase III trial for moderate‑to‑severe active thyroid eye disease, with 76.7% of patients achieving a ≥2 mm proptosis reduction versus 19.6% on placebo. The study also showed a mean proptosis decrease of 3.17 mm compared with 0.80 mm for placebo and improvements across secondary efficacy measures. Safety mirrored the known IV profile, with only mild injection‑site reactions. The data pave the way for a more convenient delivery option that could broaden access.
Artificial Intelligence and Biology: AI’s Potential for Launching a Novel Era for Health and Medicine
Artificial intelligence is reshaping biology by rapidly predicting protein structures and gene variant effects, exemplified by AlphaFold’s Nobel‑winning breakthrough and AlphaGenome’s genome‑wide insights. Researchers are moving beyond correlation‑based models toward hybrid frameworks that combine causal knowledge with multimodal datasets, as...
Life Biosciences Celebrates ER-100 Milestone After Eight Years
Congrats Life Biosciences team. Big thanks to our investors. Well done Jerry McLaughlin, Michael Ringel, Amit Shashank, Sharon Rozenzweig-L, Ivan Kaplan, @chefserenapoon & team. Go ER-100🤞 Not sure we qualify as “startup” after 8 yrs & being in the clinic...
AI-Driven Biology Faces Jagged Data Frontier; Initiative Leads
AI accelerating bio is going to be a jagged frontier driven by availability of relevant data. This is a fantastic initiative to get ahead of that. (see also what OAIF @JacobTref is doing).
High-Dose Wegovy Debuts at $399 for Self-Paying Patients
Novo Nordisk has launched Wegovy HD, a 7.2‑mg semaglutide injection, priced at $399 per month for self‑paying patients. The FDA approved the higher‑dose formulation in March, expanding the company’s weight‑loss portfolio beyond the previous 2.4‑mg limit. The new price is...
Two Cancer Startups Secure $237M for Antibody Innovations
A couple cool cancer drug startups profiled this week in TR. Sidewinder Therapeutics with $137M for bispecific ADCs https://t.co/tJcQwI7C4U and Stipple Bio with $100M for novel targets for antibody drugs. https://t.co/rbXkuaRpoe
Wedbush Flags One NKTR Dosing Interval as Unviable
You'll never guess which of these two dosing intervals Wedbush says "may not be viable" for $NKTR Rezpeg moving forward. Won't mince words - This firm is responsible for the worst sellside work I've ever seen. Sorry to be harsh,...
Daily Multivitamin Linked to Four‑Month Slowing of Biological Aging, Study Finds
Researchers led by Howard D. Sesso published a study in Nature Medicine showing that adults 60 and older who took a daily multivitamin for two years aged biologically about four months less than those on placebo. The finding revives discussion...
Adaptive Coherence Framework Guides Smarter Microbiome Health
We can be smarter about our microbiome health by cueing into host interactions, an "adaptive coherence framework" @cellhostmicrobe https://t.co/mprFgV7Gjq https://t.co/rQiPeK2aJP
Novo Undercuts Lilly with Pricier Wegovy; Insmed Braces for Flop
Novo underprices Lilly with higher-dose Wegovy; Insmed absorbs ‘expected’ study failure https://t.co/jN9GuuOf4s $NVO $LLY $INSM $VRTX $BIIB
Forecasting Protein Aggregation with an Improved Algorithm
Scientists at the Autonomous University of Barcelona have released the fourth generation of their protein‑aggregation forecasting algorithm, which leverages AlphaFold structural data and molecular‑dynamics simulations. The tool lets users evaluate aggregation risk, explore mutations, scan protein families, and assess pH...
2024 NIA‑AA Definition Connects Biomarkers Directly to Care
The 2024 NIA-AA biological definition of Alzheimer’s disease: linking biomarkers to clinical practice https://t.co/00cP1mXXHR https://t.co/VQ59YeIUR1
Genomics Predicts Individual Weight Loss From GLP‑1 Drugs
Genomics of GLP-1 drug response and side effects With genomic and demographic data it's possible to predict magnitude of weight loss response @23andMe https://t.co/iAS2Fjjxig https://t.co/AXsWnZw2Ub
Faster Process Development via “Transfer Learning”
Transfer learning—a form of AI that reuses models trained on historic data—promises to accelerate biopharmaceutical process development. By applying existing predictive models to new, related fermentations, companies can forecast critical metrics such as viable cell density and product titre from...
Redefining Bioprocessing Using Reservoirs of Biochemical Diversity
Scientists have uncovered a trove of heat‑tolerant glycosyltransferase enzymes in the rhizosphere soils of desert plants in Saudi Arabia. These extremophile enzymes, identified through metagenomic analysis, could improve glycosylation steps in biologics manufacturing by operating under high‑temperature, low‑moisture conditions. The...
Plant Molecular Farming Comes of Age
Plant molecular farming (PMF) has evolved into a scalable, GMP‑compatible platform for producing complex biologics, leveraging low‑energy plant growth and advanced transient‑expression systems. Recent advances in sensor technology, AI‑enabled host engineering, and digital manufacturing have lowered infrastructure costs and accelerated...
Bayesian Statistical Analysis (BSA) Demonstration Project
The FDA’s CDER Center for Clinical Trial Innovation (C3TI) has launched a Bayesian Statistical Analysis (BSA) demonstration project to encourage the use of Bayesian methods in simple phase 3 trials. The program lets sponsors collaborate with FDA statisticians to apply Bayesian...
FDA Issues Series of Guidances Under Drug Competition Action Plan
The FDA released three new guidances under its Drug Competition Action Plan to streamline generic drug reviews. A final guidance clarifies the use of Information Requests and Discipline Review Letters, a revised draft updates procedures for aligning ANDA labeling with...
Approved First Generic for Apokyn Injection Cartridges Requires Separately Packaged Pen
The FDA approved Sage Chemical’s generic apomorphine hydrochloride cartridges, the first generic version of Apokyn for treating "off" episodes in advanced Parkinson’s disease. The generic cartridges work only with the proprietary Apokyn Pen, which must be obtained separately through specialty...
FDA Announces Evusheld Is Not Currently Authorized for Emergency Use in the U.S.
The FDA revised Evusheld's Emergency Use Authorization on Jan. 26, 2023, limiting its use to situations where susceptible SARS‑CoV‑2 variants comprise 90% or less of national cases. With current CDC data showing over 90% of circulating variants are resistant, Evusheld...
FDA Updates Sotrovimab Emergency Use Authorization
The U.S. Food and Drug Administration revoked the Emergency Use Authorization for sotrovimab on April 5, 2022, after CDC data showed the Omicron BA.2 sub‑variant accounted for more than 50 % of cases in every HHS region. Earlier in the year the agency...
Advancing Novel Surrogate Endpoints For Rare Disease Drug Development Workshop - 05/18/2026
The FDA is hosting a virtual workshop on May 18, 2026 to advance novel surrogate endpoints for rare disease drug development. The event is part of the Rare Disease Endpoint Advancement (RDEA) Pilot Program, mandated by PDUFA VII and FDORA, which requires up...
New Nasal Flu Vaccine Shows Promise in Mice
Researchers at Georgia State University have engineered an intranasal influenza vaccine that uses cell‑derived extracellular vesicles (EVs) to display inverted hemagglutinin (HA) proteins. The upside‑down HA exposes the conserved stalk region while masking the variable head, prompting cross‑protective immunity. In...
Merck Adjusts Its Vaccine Supply Deal in China; Soleno Withdraws EU Application
Merck and its Chinese partner Zhifei have revised their COVID‑19 vaccine supply agreement, lowering the committed volume and adding a flexible delivery schedule to reflect waning demand. The updated contract preserves Zhifei’s exclusive distribution rights but shifts risk back to...
Jeito Capital, Prominent Biotech Investor, Raises $1.2B for Next Fund
Jeito Capital announced the close of its Jeito II fund, raising €1 billion (about $1.2 billion) – the largest capital raise ever for an independent European biopharma fund. The fund will back 15‑20 clinical‑stage drugmakers, allocating up to €150 million ($162 million) per company across...
Gilead Takes Another Big Swing at Expanding Beyond HIV
Gilead Sciences has earmarked roughly $11 billion this year for three strategic acquisitions aimed at strengthening its cancer and immunology pipelines. The moves signal a decisive pivot away from its historic reliance on HIV therapeutics toward broader biotech markets. The targeted...
AI Uncovers Significant Misdiagnoses in Carcinoma Type, Study Shows
Caris Life Sciences published a JAMA Network Open study showing its GPSai algorithm identified misdiagnoses in lung squamous cell carcinoma (SCC). Among nearly 4,000 cases, 123 (3.1%) were re‑classified as metastases from other primary sites. The AI model combined molecular,...
New Light Shed on Who Benefits Most From Weight-Loss Jabs
A new study published in Nature examined 15,000 people who used GLP‑1 weight‑loss drugs such as Wegovy and Mounjaro and found that two genetic variants linked to appetite and digestion significantly boost weight loss and increase nausea risk. Carriers of...
Genomic Drivers of Venetoclax Resistance in T(11;14) Myeloma
Genomic mechanisms of resistance to venetoclax in multiple myeloma with t(11;14)(CCND1;IGH) [Apr 2, 2026] @MKaddouraMD et al. @lbaughn @BloodPortfolio https://t.co/qmI6giPG6K #mmsm #PrecisionMedicine https://t.co/hpwUcavTg9
New Gene Therapy May Help Protect the Hearts of Patients with Friedreich's Ataxia
Lexeo Therapeutics reported early-phase results for its investigational gene therapy AAVrh.10hFXN (LX2006) in Friedreich’s ataxia–associated cardiomyopathy. In a phase 1 trial of 17 adults, a single intravenous infusion was well tolerated and produced signals of cardiac improvement, while neurological scores remained...
Wildflower Once Used to Treat Wounds and Sore Throats Shows Promise in Fighting Dangerous Superbugs
Researchers at Irish universities have demonstrated that extracts from the wildflower tormentil (Potentilla erecta) possess strong antimicrobial activity against multidrug‑resistant bacteria, including strains that cause pneumonia and urinary‑tract infections. The study identified ellagic acid and agrimoniin as the key compounds...
Lab-Grown Pineal Gland Organoids Produce Melatonin, Offering a New Sleep Model
Researchers at Yale School of Medicine have engineered human pineal gland organoids that synthesize and release melatonin. By coupling these organoids with a nerve‑cell assembloid, they demonstrated stimulus‑dependent hormone secretion and successfully restored melatonin production in mice lacking a native...
RFK Jr. Pushes to Overturn Peptide Ban, Officials Dispute Claims
RFK Jr. May Reverse a Peptide Ban He Calls “Illegal.” Former FDA Officials Say He Mischaracterized Their Work. https://t.co/glqreTHvKA https://t.co/Vi4OTryX3p
Pemivibart Shows Safety, Prevents COVID-19 in CLL Subset in Phase 3 CANOPY Trial
A phase 3 CANOPY subset analysis evaluated pemivibart, a recombinant IgG1 monoclonal antibody, for pre‑exposure COVID‑19 prophylaxis in patients with chronic lymphocytic leukemia (CLL). Among 29 CLL participants, none developed symptomatic SARS‑CoV‑2 infection over a 180‑day follow‑up, and the safety profile...
Childhood Dementia Explained by Synaptic Dysfunction, Opens New Therapies
Researchers at Flinders University used human iPSC-derived cortical neurons to model Sanfilippo syndrome, revealing that excitatory synapses become hyperactive early in development. This chronic overactivity mirrors the hyperactivity and sleep disturbances observed in affected children and appears to drive cognitive...
Lymph-Node CD8+ T Cells Amplify PD-1 Blockade in GI Cancer
PD-1 antibody-bound progenitor-exhausted CD8+ T cells in lymph nodes boost PD-1-blockade anti-tumor immunity in gastrointestinal cancer https://t.co/VKoxzy6oUq https://t.co/cQG2sLWtL2
Post-HSCT Gilteritinib May Improve Outcomes in R/R FLT3-Mutated AML
A systematic review of eight studies suggests that post‑transplant gilteritinib maintenance may markedly improve survival for patients with relapsed or refractory FLT3‑mutated acute myeloid leukemia. One‑year overall survival rates ranged from 72.3% to 100%, while two‑year overall survival hovered around...
Bausch + Lomb Receives FDA 510(k) Clearance for Bi-Blade+™ Dual-Port Vitrectomy Cutter and Adaptive Fluidics™ Advanced Update
Bausch + Lomb received FDA 510(k) clearance for its Bi‑Blade™+ dual‑port vitrectomy cutter and the Adaptive Fluidics update on the Stellaris Elite system. The new cutter operates at 25,000 cuts per minute, a 66% speed boost over the prior model, and delivers a...
Sidewinder Therapeutics Raises $137 Million to Advance Bispecific ADCs
Sidewinder Therapeutics announced a $137 million Series B round, surpassing expectations and bringing its total capital to $162 million. The round was led by Frazier Life Sciences and Novartis Venture Fund, with participation from OrbiMed, DCVC Bio, Goldman Sachs, and others. The funding...
A New Way To Target Metastatic Cancer
Researchers have unveiled a protein‑based delivery platform that homes to lymph nodes and releases an immune‑activating antibody only in the presence of metastatic cancer. The two‑step system first accumulates in nodes after bloodstream injection, then opens in the tumor’s chemical...
Noninvasive Stool DNA Testing May Outperform Colonoscopy Long-Term in Real-World CRC Screening
A new microsimulation study published in the Journal of Medical Economics finds that three rounds of next‑generation multitarget stool DNA (mt‑sDNA) testing over ten years outperform a single colonoscopy in real‑world colorectal cancer (CRC) screening. Higher patient adherence—72% versus 38%...
Can Biopeptides Reclaim Weight Loss From GLP‑1 Drugs?
Biotech startup Lembas is developing biopeptide ingredients that trigger satiety pathways, positioning them as a functional‑food complement to GLP‑1 weight‑loss drugs rather than a replacement. The short, bioavailable peptides aim to activate a cascade of gut hormones, delivering six to...
Wim Hof Method Cuts Inflammation in Multiple Sclerosis Trial
A randomized pilot study of 60 multiple sclerosis patients showed that a 12‑week Wim Hof Method program significantly reduced blood inflammatory markers, matching the effect of a structured lifestyle intervention. Researchers say the findings support the method as a safe...
Life Biosciences to Begin First Human Partial Reprogramming Trial in 2026
Life Biosciences, the Boston biotech co‑founded by David Sinclair, announced that its partial cellular reprogramming therapy will enter a first‑in‑human trial in 2026. The study will enroll 18 participants with glaucoma or non‑arteritic anterior ischemic optic neuropathy (NAION) and will...
NRH Prevents Age‑related Hearing Loss via Sirt3‑mediated Ferroptosis Suppression
NRH attenuates age-related hearing loss by suppressing cochlear ferroptosis and cellular senescence via Sirt3 activation https://t.co/MOqQblWYNM https://t.co/uhSMXRIxDK
Researchers Develop Graphene Nanodrum and AI Platform for Rapid Single-Cell Bacterial ID and Antibiotic Testing
Researchers at TU Delft, its spin‑off SoundCell, and Reinier Haga MDC have created a graphene‑based nanodrum platform that reads the nanomotion of individual bacteria and feeds the data to AI models for rapid identification and antibiotic susceptibility testing. The label‑free...
Extracellular Vesicles: A Growing Pipeline Still Searching for Validation
Extracellular vesicles (EVs), once hailed as natural delivery vehicles, have generated a sizable pipeline but no approved therapeutics yet. More than 90 clinical studies are evaluating both native MSC‑derived vesicles and engineered platforms for regeneration, gene editing, and vaccines. Companies...
MRNA Pioneer Katalin Karikó to Give Johns Hopkins Commencement Address
Nobel laureate Katalin Karikó will deliver the commencement address at Johns Hopkins on May 21. Karikó, whose mRNA work underpins the Pfizer/BioNTech and Moderna COVID‑19 vaccines, will receive a Doctor of Humane Letters. Her career, marked by early setbacks and decades‑long...
SLAS Announces the Cohort for Innovation AveNEW at SLAS Europe 2026
SLAS announced the twelve startups selected for its Innovation Ave NEW program at the SLAS Europe 2026 conference in Vienna. The cohort, representing companies from seven countries, will showcase new laboratory‑automation and discovery technologies on a dedicated exhibition floor. SLAS will cover...
Controversial Drugs Once Feared, Now Offer Hope
A decade ago, these drugs tore apart the FDA. Today, they might be some patients’ best hope Another wonderful piece from @Jasonmmast https://t.co/zOvLrdAPKi via @statnews