From Decades to Years - AI Could Speed Search for Brain Drugs Hiding in Plain Sight
Scientists at the UK Dementia Research Institute are using artificial intelligence to sift through patient data, voice recordings, eye scans and lab‑grown brain cells, aiming to repurpose existing drugs for neurological conditions such as motor neurone disease (MND). By training machine‑learning models on patterns of disease, the team hopes to identify treatments in years rather than the traditional decade‑long development cycle. The MND‑SMART trial tests several AI‑suggested candidates simultaneously, offering faster clinical feedback. Success could bring affordable, effective therapies to patients much sooner.
Abivax Presents First Quarter 2026 Financial Results and Reports Three-Year Interim Data From Study 108, a Phase 2a/2b Open-Label Extension...
Abivax reported Q1 2026 results, highlighting €491.6 million (≈$540 million) in cash and a runway extending into Q4 2027. The company released three‑year interim data from Study 108, showing that 68% of 130 ulcerative colitis patients remained in clinical remission after 144 weeks of obefazimod...
FDA Approves Hepcludex, First Treatment for Chronic Hepatitis D
The U.S. Food and Drug Administration has granted approval to Hepcludex (bulevirtide‑gmod), marking the first FDA‑cleared therapy for chronic hepatitis D in adults without cirrhosis or with compensated cirrhosis. Approval follows the phase 3 MYR301 trial, which showed a 48% combined response...
Skape Bio Unlocks Generalizable GPCR Drugs Using AI Protein Design
Skape Bio, founded by former UW protein‑design researcher Chris Norn, has unveiled an AI‑driven platform that creates miniprotein therapeutics for G‑protein‑coupled receptors (GPCRs). A recent Nature paper shows functional miniproteins targeting 11 diverse GPCRs, including agonists validated on three receptors....
Experimental mRNA Vaccine May Protect Against Multiple Ebola Viruses
Researchers have created an experimental mRNA vaccine that protects rodents from three orthoebolavirus strains, including the Bundibugyo virus driving the current outbreak in the Democratic Republic of the Congo and Uganda. The study shows the vaccine elicits robust antibody responses...
Expanded Label for Efgartigimod Offers Fast-Acting Treatment for gMG: James F. Howard, MD
On May 11, 2026, the FDA broadened the indication for efgartigimod, approving it for all adult patients with generalized myasthenia gravis, including AChR‑positive, MuSK‑positive, LRP4‑positive, and triple‑seronegative subtypes. The decision follows the phase‑3 ADAPT SERON trial, which enrolled 119 patients...
AstraZeneca Wins EU Backing for Breast Cancer Drug, Splitting with FDA Panel
AstraZeneca’s breast‑cancer therapy, identified as [drug], received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use, clearing the path for EU approval. In contrast, an FDA advisory committee voted against the drug last month,...
OSE’s Tedopi-Keytruda Combo Clears Phase II Ovarian Cancer Hurdle
French biotech OSE Immunotherapeutics announced positive Phase II data for its cancer vaccine Tedopi combined with Merck's Keytruda in platinum‑sensitive recurrent ovarian cancer. The combination improved median progression‑free survival to 4.1 months versus 2.8 months with best supportive care, cutting the risk of...
NIH Researchers Identify Avenue for Enhanced GLP-1-Induced Weight Loss
NIH scientists have mapped how the GLP‑1 agonist semaglutide triggers intracellular signaling in mouse hindbrain neurons, pinpointing cyclic AMP (cAMP) elevation in the area postrema as a key driver of weight loss. The study revealed that cAMP responses differ across...
What Is Immunotherapy and How Does It Treat Cancer and Other Conditions?
Immunotherapy research has surged, with clinical trials climbing from 1,257 between 2006‑2016 to 4,591 in the past decade. Cancer treatments dominate the field, featuring checkpoint inhibitors, CAR‑T cells, and mRNA‑based vaccines now approved for more than 30 tumor types. Researchers...
Cognitive Effects Vary by Therapy for Advanced Prostate Cancer
A phase‑2 ARACOG trial presented at ASCO showed that men with advanced prostate cancer receiving darolutamide experienced significantly less cognitive decline over 24 weeks than those on enzalutamide. The study enrolled 111 patients (median age 71) and evaluated five computer‑based neurocognitive tests,...
The NMPA Grants Conditional Approval to Boehringer Ingelheim’s Hernexeos for 1L HER2-Mutant NSCLC
China's National Medical Products Administration granted conditional approval to Boehringer Ingelheim’s oral HER2‑mutant NSCLC drug Hernexeos (zongertinib). The decision is based on a Phase Ib trial of 74 treatment‑naïve patients that delivered a 75.7% overall response rate and a median...
Astellas Receives CHMP Positive Opinion for Perioperative Padcev + Keytruda for Muscle-Invasive Bladder Cancer (MIBC)
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion on Astellas’ Padcev (enfortumab vedotin) combined with Keytruda (pembrolizumab) for neoadjuvant and adjuvant treatment of cisplatin‑ineligible patients with resectable muscle‑invasive bladder cancer (MIBC). The...
Biogen, Denali to Drop Drug in Non-Genetic Parkinson’s After Mid-Stage Study Flop
Biogen and Denali Therapeutics announced that their LRRK2 inhibitor BIIB122 failed to meet primary endpoints in a Phase 2b trial of early‑stage Parkinson’s disease. The mid‑stage study, which targeted patients without the LRRK2 genetic mutation, showed no statistically significant slowing...
NICE Changes Stance on Genmab's Cervical Cancer Drug
Two months after rejecting Genmab’s Tivdak, the UK health technology body NICE has reversed course, recommending the antibody‑drug conjugate for adults with recurrent or metastatic cervical cancer. The decision follows a revised economic model that incorporates the innovaTV 301 trial results...
Allergan Aesthetics (AbbVie) Reports CHMP Positive Opinion for Boey to Temporarily Improve Glabellar Lines in Adults
Allergan Aesthetics, a unit of AbbVie, received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommending Boey (trenibotulinumtoxinE) for the temporary improvement of moderate-to-severe glabellar lines in adults. The recommendation covers all...
The Problem at the Heart of Drug Discovery: Lexogen & Ochre Bio on the Power of AI on Human Data
Lexogen, an RNA transcriptomics and NGS service provider, teamed up with Ochre Bio, a biotech developing AI‑driven RNA therapies for chronic liver disease. The partnership leverages Lexogen’s high‑throughput sequencing to generate human‑first data that trains Ochre’s predictive models. Together they...
Biogen, Denali Pull Parkinson's Drug After Failed Trial
Biogen has halted development of BIIB122, its LRRK2‑inhibitor partner with Denali, after the phase 2b LUMA trial failed to slow Parkinson’s disease progression despite achieving about 30% target inhibition. The study, which included both LRRK2‑mutated and idiopathic patients, missed its primary...
TrivarX Engages Beyond Drug Development to Progress Stabl-Im Safety Trial
TrivarX announced a partnership with specialist CRO Beyond Drug Development to drive the Phase 1 safety trial of its Stabl‑Im oncology imaging platform. Beyond will handle protocol finalisation, regulatory documentation and site selection as TrivarX prepares to dose healthy volunteers in...
STAT+: Closely Watched Experimental Parkinson’s Drug Fails Key Clinical Trial
Biogen and Denali Therapeutics announced that their experimental Parkinson’s drug, a small‑molecule inhibitor of the LRRK2 protein, failed to slow disease progression in a randomized Phase 2 trial. The study enrolled 648 adults who received either the LRRK2 pill or...
Zentalis Pharmaceuticals to Present Phase 1b MUIR Trial Data Showing Encouraging Clinical Activity and Manageable Safety Profile of Azenosertib Plus...
Zentalis Pharmaceuticals will present Phase 1b MUIR trial results showing that the WEE1 inhibitor azenosertib combined with paclitaxel yields a 39% overall response rate and a median progression‑free survival of 7.3 months in heavily pre‑treated platinum‑resistant ovarian cancer (PROC) patients. The data,...
#ASCO26: Key Head and Neck Cancer Readouts From J&J, Corbus, Summit, Akero and Bicara
At ASCO 2026, Johnson & Johnson, Corbus, Summit, Akero and Bicara unveiled pivotal data on next‑generation therapies for head and neck squamous cell carcinoma. J&J’s pembrolizumab‑plus‑chemotherapy regimen reduced the risk of death by roughly 20% in first‑line patients. Corbus reported...
#ASCO26: New Data Released for VEGF Bispecifics From BioNTech-BMS, Pfizer-3SBio
At ASCO 2026, BioNTech‑BMS and Pfizer‑3SBio unveiled new Phase 1/2 data on their VEGF‑targeting bispecific antibodies. BioNTech‑BMS reported a 45% overall response rate in heavily pre‑treated solid‑tumor patients, while Pfizer‑3SBio’s candidate achieved disease control in 78% of a similar cohort....
STAT+: Merck-Kelun Lung Cancer Drug Cut Risk of Tumor Progression by 65%, ASCO Abstract Shows
Merck and China‑based Kelun‑Biotech announced that their antibody‑drug conjugate sacituzumab tirumotecan (sac‑TMT) reduced the risk of tumor progression by 65% in a Phase 3 trial of untreated advanced non‑small cell lung cancer (NSCLC). The study, conducted in China, also showed an...
CVS Health Sued Over Alleged Scheme to Siphon 340B Drug Program Savings
Three major hospital systems sued CVS Health and its affiliates, alleging the company diverted roughly $250 million in savings from the federal 340B Drug Pricing Program between 2020 and 2025. The lawsuits claim CVS Specialty and its PBM unit paid hospitals...
Eli Lilly’s Triple Combo Obesity Drug Tops 28% Weight Loss in a Pivotal Trial
Eli Lilly’s experimental obesity drug retatrutide delivered an average 28.3% weight loss – roughly 70 pounds – in its Phase 3 trial, with the highest dose leading the result. The study enrolled 2,339 participants without diabetes and tested three escalating doses, each outperforming...
Cytokine‑Armored CAR T Cells Overcome Antigen Heterogeneity in Glioma Model
UCLA researchers have engineered cytokine‑armored CAR‑T cells that secrete IL‑12 and a decoy‑resistant form of IL‑18 (DR‑18) to treat glioblastoma in mouse models. The IL‑12/DR‑18 combination enabled CAR‑T cells to eradicate tumors even when cancer cells lacked the targeted IL‑13Rα2...
New Eye Drop Formulation Shows Promise for Dry Eye Disease
Researchers at Baylor College of Medicine and Okayama University have created a water‑soluble rexinoid eye‑drop, NEt‑3IB, that boosts resident macrophage function and mitigates dry‑eye pathology in mouse models. The formulation significantly lowered ocular inflammation, preserved corneal barrier integrity and goblet...
Vitamin B12 Analog Targets Deadly Brain Cancer Cells
Researchers at Nitric Oxide Services and Cleveland Clinic have demonstrated that nitrosylcobalamin, a nitric‑oxide‑releasing vitamin B12 analog, can penetrate the blood‑brain barrier and preferentially accumulate in glioblastoma tissue in rat models. Pharmacokinetic data show sustained tumor nitrate levels for at...
Targeting Tumor-Specific Inflammatory Process May Prevent Drug Resistance in Colon Cancer
A preclinical study from Weill Cornell Medicine and MD Anderson reveals that colorectal tumors with KRAS mutations develop resistance to KRAS inhibitors primarily through a tumor‑specific inflammatory response rather than additional KRAS mutations. The researchers observed early up‑regulation of inflammatory...
Financings for May 21, 2026
BioWorld reported three major developments on May 21, 2026. Researchers unveiled a “detargeted” gene‑therapy platform that enhances enzyme activity and reduces off‑target effects for Pompe disease. The World Health Organization declared the Ebola outbreak in the Democratic Republic of Congo a public‑health...
BioMarin Notches Win in Study that Could Expand Use of Top-Selling Medicine
BioMarin announced positive Phase 3 data showing its blockbuster drug Voxzogo accelerates growth in children with hypochondroplasia, a milder form of dwarfism. The trial reported significant gains in standing height and arm span after one year versus placebo. Analysts estimate the...
The Multiple Myeloma Revolution Happening Right Now: Swarup Kumar, MD
Swarup Kumar, MD highlights that bispecific antibodies are driving multiple myeloma cure rates up to 30‑40%, a stark rise from the historic sub‑10% figure. He notes that real‑world outcomes, while promising, still trail slightly behind trial data, underscoring the need...
Fixing Medication Adherence In Clinical Trials: How Technology Stops Data Corruption
Medication adherence in clinical trials is notoriously difficult to measure, with self‑reports often exceeding 90% while biomarker data reveal far lower rates. Traditional methods such as dosing diaries and pill counts are vulnerable to patient error and intentional manipulation, compromising...
Avaí Bio, Austrianova Advance Α-Klotho Cell Therapy Manufacturing Step
Avaí Bio and Austrianova have finished a GMP‑compliant master cell bank (MCB) of genetically engineered cells that overexpress the anti‑aging protein α‑Klotho. The bank will now undergo independent viral and adventitious‑agent testing before a working cell bank is created for...
How Eli Lilly's Next-Gen Obesity Drug Is 'Raising The Bar' In Weight Loss
Eli Lilly’s next‑generation obesity drug retatrutide delivered dramatic weight loss in the Phase 2 Triumph‑1 trial, with participants shedding an average 28.3% of body weight (about 70 lb) after 80 weeks and up to 30.3% (≈85 lb) in a 104‑week extension. Nearly half of...
Label Change Helps Remove Barriers to Broader Use of Epcoritamab in DLBCL, Sharman Says
The FDA revised the label for epcoritamab (Epkinly) on April 1, 2026, allowing the first full 48‑mg dose to be administered in an outpatient setting for relapsed/refractory diffuse large B‑cell lymphoma (DLBCL). The change follows interim data from the EPCORE NHL‑6 study,...
M11 Template: Clinical Electronic Structured Harmonised Protocol (CeSHarP)
The ICH has issued the M11 Template — Clinical Electronic Structured Harmonised Protocol (CeSHarP) – a draft guidance released in June 2025. The template standardizes the format, table of contents, and common headers for clinical trial protocols, while the accompanying Technical Specification defines...
Eli Lilly Reports P-III (TRIUMPH-1) Trial Data on Retatrutide for Weight Management
Eli Lilly’s phase‑III TRIUMPH‑1 trial showed its triple‑agonist retatrutide produced dramatic weight loss in adults with obesity and at least one weight‑related comorbidity. Across 80‑week endpoints, the 12 mg dose cut mean body weight by 28.3% and waist circumference by 24.1 cm, with...
Infex Chases After Insmed with Bronchiectasis Trial Win
Infex Therapeutics announced that its anti‑PcrV antibody RESP‑X (INFEX702) successfully completed a Phase 2a trial in patients with non‑cystic fibrosis bronchiectasis (NCFB) colonised by Pseudomonas aeruginosa. The study demonstrated early efficacy signals, a favorable safety profile and a 28.8‑day half‑life supporting...
Bayer Gets Swift Review for Kerendia in Type 1 Diabetes
Bayer has applied to the FDA for a label extension of its mineralocorticoid receptor antagonist Kerendia to treat chronic kidney disease in type 1 diabetes patients. The agency granted priority review, promising a decision within six months. Phase 3 FINE‑ONE data showed...
ATS 2026: Long-Term Data Show Durable Benefit of Trikafta for Cystic Fibrosis
At the 2026 American Thoracic Society conference, a German single‑centre cohort of 106 adults with cystic fibrosis and at least one F508del allele reported four‑year outcomes on Trikafta (elexacaftor/tezacaftor/ivacaftor). Patients showed a 0.5 L (15.6%) rise in FEV1, a 28.9% drop...
Quality by Design: The Impact of Disconnected Systems on Clinical Data
Clinical trials are increasingly digital, but the rapid addition of tools like EDC, ePRO, safety databases, CTMS, eTMF and RBQM often results in fragmented, poorly integrated systems. These disconnections force duplicate data entry, create inconsistent audit trails, and raise the...
Infex Reports Phase IIa Win for Anti-Pseudomonas Antibody
Infex Therapeutics announced positive Phase IIa results for RESP‑X, its first‑in‑class anti‑virulence monoclonal antibody targeting PcrV in Pseudomonas‑colonised non‑cystic fibrosis bronchiectasis. The randomized, double‑blind trial demonstrated safety, tolerability and a 28.8‑day half‑life supporting quarterly dosing. While the study was not powered...
Eli Lilly Acquires Engage Biologics for ~$202M
Eli Lilly announced the acquisition of Engage Biologics for roughly $202 million in cash, integrating Engage’s Tethosome non‑viral DNA delivery platform with Lilly’s growing genetic‑medicine portfolio. The deal includes an upfront payment and milestone‑based earn‑outs tied to development progress. Tethosome combines engineered DNA...
Guidance, Compliance, & Regulatory Information
The FDA’s Guidance, Compliance, & Regulatory Information hub aggregates key resources for drug manufacturers, ranging from compliance programs and inspection protocols to labeling and generic development guidance. Recent additions include an Environmental Impact Statement focused on sunscreen drug products and...
Collaborative Drug Discovery Inks Deal with Eli Lilly to Accelerate Biotech Innovation
Eli Lilly’s TuneLab AI platform will be embedded into Collaborative Drug Discovery’s (CDD) Vault system, giving biotech companies access to Lilly’s proprietary ADMET predictive models. The integration will appear in both the core and AI modules of CDD Vault, allowing researchers...
FDA Offers New Details On Real-Time Clinical Trial Pilot, But Questions Remain
The FDA released new operational details for its real‑time clinical‑trial pilot, clarifying that raw patient‑level data will not be transmitted directly to the agency. Instead, sponsors must use their own data‑capture systems to send predefined safety, efficacy, dosing, or operational...
STAT+: Biotech Execs, Academic Expert Lament Impact of FDA Turnover on Rare Disease Drug Development
Biotech leaders and an academic expert warned that recent turnover among senior FDA officials is creating heightened uncertainty for rare‑disease drug developers. At the STAT Breakthrough Summit West, Mahzi Therapeutics CEO Yael Weiss described constant investor inquiries and a “roller‑coaster”...
Mutating Antibodies for Easier Drug-Conjugate Manufacturing
Scientists at Johns Hopkins University have engineered a general‑purpose antibody by mutating its fragment crystallizable (Fc) region, creating up to four new attachment sites for molecules such as drugs, dyes, or nanoparticles. Six precise Fc mutations enable consistent, site‑specific conjugation,...