Adenine Base Editing Demonstrates Profound Impact on Rare Disease
Researchers at The Jackson Laboratory used adenine base editing to repair the SCN1A R613X mutation that causes Dravet syndrome in mice. A single brain injection corrected roughly 60% of the defective DNA, restoring normal gene expression and dramatically reducing seizures. Treated mice lived significantly longer, with benefits observed both when the edit was delivered at birth and at day 12. The work showcases a potential one‑time genetic cure for a rare, fatal childhood epilepsy and aligns with the FDA’s new Plausible Mechanism Framework for rare‑disease therapies.
Merck's ADC Sac-TMT Gets Its First Global Phase 3 Win Ahead of Schedule
Merck and China‑based Kelun‑Biotech announced that their experimental antibody‑drug conjugate sac‑TMT achieved its primary endpoint in a global Phase 3 trial, completing ahead of schedule. The study enrolled roughly 650 patients with advanced solid tumours across 12 countries and demonstrated a...
Sotatercept Reduced Morbidity in CTD-PAH Analysis: Rogerio Souza, MD, PhD
A pooled analysis of the phase‑3 STELLAR, ZENITH and HYPERION trials shows that sotatercept significantly lowers the risk of first major morbidity or mortality events in patients with connective tissue disease‑associated pulmonary arterial hypertension (CTD‑PAH). The benefit persisted despite most...
FDA Approves Baxdrostat for Inadequately Controlled Hypertension
AstraZeneca’s Baxdrostat (Baxfendy) received FDA approval as the first aldosterone synthase inhibitor for hypertension in the United States. The drug is indicated for patients whose blood pressure remains uncontrolled despite taking at least two other antihypertensive agents. Phase III trials (BaxHTN...
What Comes After Pluvicto? A New and Distinct Prostate Cancer Patient Subpopulation Is Taking Shape
PSMA‑targeted radioligand therapy, exemplified by lutetium‑177 vipivotide tetraxetan (Pluvicto), is now a standard option for metastatic castration‑resistant prostate cancer. However, response durability is limited, with fewer than half of patients achieving meaningful benefit and most eventually progressing. The authors highlight...
Novartis' Pluvicto Cuts PSA Progression Risk in Phase 3 Prostate Cancer Trial
Novartis announced that its radioligand therapy Pluvicto, when added to standard of care, reduced the risk of prostate-specific antigen progression in the Phase III PSMAddition trial for PSMA‑positive metastatic hormone‑sensitive prostate cancer. The data were unveiled at the American Urological Association...
Pittsburgh Researchers Develop Shelf-Stable Artificial Platelets to Stop Severe Bleeding
Researchers at the University of Pittsburgh, in partnership with Case Western Reserve and Haima Therapeutics, have created SynthoPlate, a freeze‑dried artificial platelet that reconstitutes with water to form a clotting agent. The quarter‑sized vial remains stable at room temperature for...
Nanobiotix's Phase‑2 Lung Cancer Trial Shows 85.7% Response Rate
Nanobiotix S.A., with Johnson & Johnson as sponsor, presented Phase‑2 CONVERGE data showing an 85.7% overall response rate and 57.1% complete response in seven stage III inoperable NSCLC patients, suggesting the nanoparticle radioenhancer NBTXR3 could improve outcomes when combined with chemoradiotherapy...
STAT+: Pharmalittle: We’re Reading About U.S. Biotech’s China Problem, a Regeneron Flop, and Much More
U.S. biotech companies have poured roughly $60 billion into Chinese‑origin molecules in the first quarter of 2026, a rate that could double the previous year’s spend and is ten times the 2021 level. The surge is deepening a split in the...
The Ebola Emergency Shines a Light on the Urgent Need for New Vaccines
The Democratic Republic of Congo and Uganda are facing a Bundibugyo Ebola outbreak, with the U.S. CDC reporting 336 suspected cases and 88 deaths as of 17 May. The WHO declared a public‑health emergency of international concern, but its response is...
Regeneron Shares Tumble 6% After Phase 3 Melanoma Trial Misses Statistical Significance
Regeneron Pharmaceuticals reported that its Phase 3 trial of fianlimab plus cemiplimab did not achieve statistical significance for progression‑free survival versus pembrolizumab, prompting a 6.23% drop in the stock during after‑hours trading. The high‑dose arm showed a numeric 5.1‑month PFS...
How Advanced Analytics Partnerships Enhance the Biopharma Value Chain
Strategic biopharma alliances are increasingly embedding multimodal AI across the entire drug‑development pipeline. Partnerships leverage foundation models to sharpen biomarker discovery and patient selection for complex modalities such as antibody‑drug conjugates. Large‑scale genomic and clinical datasets enable virtual cell modeling...
FDA Approves Enhertu for Neoadjuvant and Adjuvant HER2‑Positive Early Breast Cancer
The U.S. Food and Drug Administration has approved Enhertu (fam‑trastuzumab deruxtecan‑nxki) for two new HER2‑positive early‑breast‑cancer indications – neoadjuvant treatment of stage 2‑3 disease and adjuvant therapy for patients with residual invasive disease after standard therapy. The approvals rest on phase 3...
Novartis Reveals More Data Behind Pluvicto Expansion Bid
Novartas reported new PSMAddition trial data showing that adding its radioligand therapy Pluvicto to standard hormonal treatment cuts PSA progression by 58% in hormone‑sensitive metastatic prostate cancer. Deep PSA reductions below 0.2 ng/mL were achieved by 87.4% of patients versus 74.9%...
Amgen’s Rare Disease Drug Tavneos Tied to 20 Deaths in Japan
Amgen’s rare‑disease drug Tavneos has been linked to 20 deaths among roughly 8,500 Japanese patients, primarily due to vanishing bile duct syndrome, a severe form of drug‑induced liver injury. The Japanese distributor Kissei Pharmaceutical has warned doctors against initiating new...
Regeneron Inks a ~$2.3B Deal with Parabilis Medicines to Advance Antibody-Helicon Conjugates Across Multiple Therapeutic Areas
Regeneron has struck a strategic research collaboration with Parabilis Medicines to develop antibody‑Helicon conjugates (AHCs) across several therapeutic areas. The agreement provides Parabilis with $125 million, including $50 million upfront and a $75 million equity‑financing commitment, and makes it eligible for roughly $2.2 billion...
Harbour Posts Preclinical Data on Would-Be Rival to Eli Lilly’s $1.9B Obesity Bet
Harbour BioMed released preclinical data on LET003, an ACVR2A/2B‑targeting monoclonal antibody created with its AI‑enabled Hu‑mAtrIx platform. In mouse and monkey studies the molecule cleared more slowly than comparators and drove a 13.5% increase in lean mass versus a rival...
BIO Supports and Seeks Refinements to FDA’s Plausible Mechanism Framework
The FDA has issued draft guidance introducing a Plausible Mechanism Framework to evaluate safety and efficacy of individualized, disease‑targeted therapies when traditional trials are infeasible. The Biotechnology Innovation Organization (BIO) issued a supportive comment letter, praising the agency’s direction while...
What Psychedelic Clinical Trials Could Teach Psychiatry
Treatment‑resistant depression (TRD) affects roughly one‑third of U.S. adults on antidepressants and remains a high‑need, low‑treatment area. Compass Pathways’ synthetic psilocybin, COMP360, recently achieved its primary endpoints in two Phase III trials—258 patients in COMP005 and 581 in COMP006—showing rapid, durable...
Supercharging Immune Cells May Help Control HIV Long-Term
Scientists have repurposed CAR‑T cell therapy, originally used for cancer, to target HIV. In a small Phase 1 trial, two participants who received the engineered T cells remained off antiretroviral drugs with undetectable viral loads for nearly two years and one...
AstraZeneca Reports the US FDA Approval of Baxfendy (Baxdrostat) To Treat Hard-to-Control Hypertension
AstraZeneca’s Baxfendy (baxdrostat) received U.S. FDA approval for use with other antihypertensives in patients with uncontrolled or treatment‑resistant hypertension. The approval is based on the Phase III BaxHTN trial, which enrolled 796 participants and showed significant blood‑pressure reductions versus placebo. In...
Michigan Teen Gets FDA‑Approved Therapy to Delay Insulin Use in Type‑1 Diabetes
Fourteen‑year‑old Grayson Visco of Hudsonville, Michigan, started a teplizumab infusion that can postpone the need for insulin by a median of 2.7 years. The therapy, sold as TZield, received broader FDA clearance in April to treat children as young as...
Goldman Sachs Raises Biogen Target to $250 on 74% Leqembi Sales Surge
Goldman Sachs lifted its price target on Biogen (BIIB) from $238 to $250 after the company's Q1 2026 earnings showed Leqembi sales up 74% year‑over‑year to $168 million. The move underscores growing confidence that the Alzheimer's therapy can sustain a new...
Japan’s MHLW Approves Boehringer’s Jascayd for IPF and PPF
Boehringer Ingelheim’s Jascayd (nerandomilast) has been approved by Japan’s Ministry of Health, Labour and Welfare for treating idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF). It is the first phosphodiesterase 4B inhibitor with antifibrotic and immunomodulatory properties to receive such...
FDA Greenlights Enhertu for Early HER2‑positive Breast Cancer
#AZN Enhertu approved by US FDA both before and after surgery for treating patients with HER2-positive early breast cancer.
Lotte Biologics Expands Antibody Manufacturing Agreement with Ottimo Pharma
Lotte Biologics announced on May 18, 2026 an expanded collaboration with South Korean biotech Ottimo Pharma to manufacture its PD‑1/VEGFR2 dual‑paratopic antibody, OTP‑01. The agreement builds on a June‑2025 deal and adds commercial process development support at Lotte’s Syracuse Bio Campus in...
STAT+: The China Question Is Tearing Biotech Apart
U.S. biotech firms have poured roughly $60 billion into Chinese‑origin molecules in the first quarter of 2026, a rate that could double the previous year’s spend and represents a ten‑fold jump since 2021. The influx of affordable, fast‑developed compounds fuels pipeline...
Genentech Reports FDA Approval of Tecentriq and Tecentriq Hybreza for Adjuvant Muscle-Invasive Bladder Cancer With ctDNA-Guided Treatment
Genentech’s Tecentriq and Tecentriq Hybreza received FDA approval as adjuvant therapies for adults with muscle‑invasive bladder cancer (MIBC) who show circulating tumor DNA (ctDNA) minimal residual disease after cystectomy. The approval is linked to Natera’s Signatera CDx assay, which was...
Spatially Tunable Multiomic Sequencing Using Light-Driven Combinatorial Barcoding of Molecules in Tissues
Researchers introduced Barcoding by Activated Linkage of Indexes (BALI), a light‑driven combinatorial barcoding platform that writes spatial DNA barcodes directly onto diverse biomolecules in tissue sections. The technique lets users define the number, size, shape and resolution of regions, enabling...
Can Revolution’s 'Miracle' Pancreatic Cancer Drug Be Topped? Immuneering, Actuate Say Yes
Revolution Medicines reported Phase 3 data showing its oral RAS inhibitor daraxonrasib more than doubled median overall survival for advanced pancreatic cancer to 13.2 months versus 6.7 months on chemotherapy. The results triggered a 40 % share surge and will support global regulatory filings,...
4 Spaces Primed for the Next Wave of Gene Therapies
The FDA’s recent approval of Regeneron’s Otarmeni for hereditary hearing loss marks the first non‑oncology gene‑therapy clearance, underscoring a shift toward treating central‑nervous‑system, ophthalmic, cardiovascular and muscular disorders. Companies such as Lexeo, Lilly/AskBio, uniQure, REGENXBIO/AbbVie, Tenaya, Medera and Sarepta are...
This Startup Aims to Upend Biologic Drug Production With Implantable ‘Cell Factories’
Duracyte, a Rice‑University spin‑out, is developing an implantable "cell factory" that produces therapeutic proteins inside patients, eliminating the need for traditional biologic manufacturing and injection pens. The capsule‑sized device houses genetically engineered human cells, supplies them with nutrients from the...
Isomorphic Labs Raises $2.1 B to Expand AI-Driven Drug Discovery Platform
Isomorphic Labs announced a $2.1 billion financing round led by Thrive Capital, with participation from Alphabet, GV, CapitalG, MGX, Temasek and the UK Sovereign AI Fund. The capital will accelerate development of its IsoDDE AI drug design engine and aim to...
Cas9+ Conditionally Immortalized Neutrophil Progenitors as a Tool for Genome Wide CRISPR Screening for Neutrophil Differentiation and Function
Researchers at UC Berkeley have engineered a Cas9‑expressing, estrogen‑regulated Hoxb8 neutrophil progenitor line that can be genetically edited and differentiated into functional neutrophils both in vitro and in vivo. The platform enables forward and reverse genetics, demonstrated by a pooled...
AI Tool in Radiotherapy Advances Global Fight to Eradicate Cervical Cancer
A collaborative consortium has launched an AI‑driven radiotherapy planning platform that automates contouring and dose‑optimization for cervical cancer. Trained on more than 10,000 patient scans, the tool generates treatment plans in minutes, cutting planning time by roughly 40% while preserving...
Alcolase Raises €1.5M to Tackle Alcohol Intolerance with Enzyme Technology
Danish biotech Alcolase secured €1.5 million (about $1.6 million) from investors including Ada Ventures, Delphinus VC, Antler and angel groups. The startup is developing a liposome‑encapsulated enzyme that breaks down alcohol in the stomach, aiming to help the roughly 540 million East Asians...
Weight-Loss Drugs Tied to Lower Death, Recurrence Risk After Breast Cancer
A retrospective cohort study of more than 840,000 breast‑cancer patients diagnosed between 2006 and 2023 found that use of GLP‑1 receptor agonists—drugs approved for type‑2 diabetes and obesity—was associated with a lower risk of death and cancer recurrence over a...
Alpha Cognition Posts 40% Q1 Revenue Jump, Expands Field Team
Alpha Cognition (NASDAQ:ACOG) announced a 40% sequential increase in Q1 2026 revenue to $3.5 million, driven by expanding prescriber adoption of its Alzheimer’s drug ZUNVEYL. The company also completed a 60‑person customer‑facing field team, signaling a push to accelerate market penetration.
Denali Therapeutics Secures FDA Approval for AVLAYAH, First Gene Therapy for Hunter Syndrome
Denali Therapeutics announced that the FDA granted accelerated approval to AVLAYAH, the first gene‑therapy targeting the neurologic manifestations of Hunter syndrome. The company said early commercial activity is already exceeding its launch forecasts, signaling strong physician and payer interest in...
Study Maps Brain Immune Cells to Block Melanoma Metastasis
Yale researchers are targeting the brain's native immune cells to stop melanoma from forming deadly brain metastases. Their work focuses on the IL‑4 signaling axis, which overactivates myeloid cells and creates a pre‑metastatic niche that shields tumor cells from standard...
Zusduri Demonstrates Real-World Efficacy for Low-Grade UTUC
UroGen’s Zusduri shows strong real-world results for low-grade upper tract urothelial cancer, matching clinical trial efficacy. A promising kidney-sparing option, though durability and reimbursement remain key hurdles. Urology
Kailera Therapeutics IPO Priced at $16, Opens at $26 Targeting Obesity Market
Kailera Therapeutics priced its initial public offering at $16 per share and opened at $26 on April 17, raising capital to advance its GLP‑1 obesity drug pipeline. The move places the company directly against industry giants Eli Lilly and Novo Nordisk as...
Synthesis and In-Vitro Cytotoxicity Evaluation of 1,2,3-Triazole Incorporated 1,3,4- Oxadiazole-Imidazole-Pyridine Derivatives as Anticancer Agents
Researchers synthesized a novel series of 1,2,3‑triazole‑incorporated 1,3,4‑oxadiazole‑imidazole‑pyridine compounds (27a‑j) and characterized them fully. In vitro cytotoxicity testing against MCF‑7, A549, Colo‑205 and A2780 cell lines showed that three analogues—27a, 27b and 27i—outperformed the reference drug etoposide. Compound 27a was...
Acuta Capital Partners Invests $4.2 M for 1.35% Stake in Erasca’s Targeted Cancer Pipeline
Acuta Capital Partners, LLC acquired 354,575 shares of Erasca Inc. for an estimated $4.19 million, giving the fund a 1.35% ownership stake. The investment underscores growing investor interest in Erasca’s RAS/MAPK‑focused cancer therapeutics despite recent clinical setbacks and a steep cash...
Ipsen's Corabotase Shows 66% Improvement in Glabellar Lines and 82% Patient Satisfaction
Ipsen presented Phase II data for its recombinant neuroinhibitor corabotase at the Scale Symposium, reporting a 66% composite response at week 4 and a 60.8% sustained effect at week 24 in moderate‑to‑severe glabellar lines. Patient satisfaction reached 82.8%, positioning the drug as...
Regeneron’s Phase 3 Melanoma Trial Misses PFS Goal, Shows 5‑Month Numeric Gain
Regeneron Pharmaceuticals reported that its Phase 3 trial of fianlimab (LAG‑3 inhibitor) combined with cemiplimab (PD‑1 inhibitor) did not achieve statistical significance for progression‑free survival versus pembrolizumab, despite a 5.1‑month median PFS advantage. The data reshape expectations for LAG‑3‑targeted regimens...
Co‑Diagnostics Q1 2026 Revenue Jumps to $146K as It Pushes FDA Filing and Global Expansion
Co‑Diagnostics posted Q1 2026 revenue of $146,000, up from $50,000 a year earlier, but widened its net loss to $9.1 million as it ramps R&D and prepares an FDA submission for its upper‑respiratory multiplex test. The company also highlighted a $13 billion...
FDA OKs Daiichi Sankyo/AstraZeneca’s Enhertu for Two HER2‑Positive Breast Cancer Indications
The U.S. Food and Drug Administration granted approval for fam‑trastuzumab deruxtecan‑nxki, marketed as Enhertu, to treat two HER2‑positive breast cancer indications. The antibody‑drug conjugate, co‑developed by Daiichi Sankyo and AstraZeneca, now covers early‑stage and metastatic disease, widening its market reach.
Tango Therapeutics Shares Plunge 17% as Q1 Loss Widens and Collaboration Revenue Vanishes
Tango Therapeutics (TNGX) saw its shares tumble 16.93% to $20.66 after reporting a first‑quarter net loss of $45.5 million and a drop in collaboration revenue to zero. The sharp decline highlights investor anxiety over the company's cash‑burn and pipeline outlook.
Enveric Biosciences Posts $1.6M Q1 Loss, Raises $16.7M as EB-003 Enters Preclinical Trials
Enveric Biosciences announced a $1.63 million net loss for Q1 2026 but bolstered its balance sheet with $16.7 million from a private offering and placement. The capital infusion supports the pre‑clinical advancement of EB-003, its lead non‑hallucinogenic neuroplastogen targeting neurological disorders.