Today's Pharma Pulse
Ona lands $86.6M Series B, one of Spain’s biggest biotech rounds
Spanish biotech firm Ona announced an $86.6 million Series B financing, ranking among the largest venture rounds in Spain’s life‑science sector this year. The round was led by Seventure Partners with participation from existing backers and new strategic investors. The funding will be used to scale Ona’s AI‑driven drug discovery platform and accelerate its pre‑clinical programs.
Trump Signs Executive Order to Fast‑Track Psychedelic Drugs for Serious Mental Illness
President Donald Trump signed an executive order that compels the FDA to issue National Priority Vouchers to psychedelic drugs with Breakthrough Therapy designations for serious mental illness. The move is intended to eliminate regulatory bottlenecks and speed patient access to treatments that have shown promise in early trials.
Why Aspirin Is Becoming a Weapon Against Cancer
Aspirin, the 4,000‑year‑old painkiller, is now shown to cut colorectal cancer risk in high‑genetic‑risk patients. A 10‑year trial of 861 Lynch‑syndrome participants found a daily 600 mg dose halved cancer incidence, and a lower 75‑100 mg dose appears equally effective. The UK...
CPC Biotech Debuts Unified Bioprocess Fluid Portfolio
CPC Biotech, a new brand under Dover’s Pump Solutions Group, launched by merging CPC’s sterile connector expertise with PSG Biotech’s low‑shear pumps, flow meters and sensors. The unified portfolio offers biopharma manufacturers a single source for connectors, pumps, and measurement...
Atsena Secures DMC Approval for LIGHTHOUSE Trial
Atsena Therapeutics received a recommendation from its independent data monitoring committee to launch the pivotal Part C of the LIGHTHOUSE Phase I/II/III trial for ATSN‑201, its gene‑therapy candidate targeting X‑linked retinoschisis (XLRS). Part C will enroll 76 patients across Europe and North America,...
Celastrol Disrupts Hsc70‑Bim to Boost Longevity, Reduce Fibrosis
Celastrol Targets Hsc70-Bim Interaction as a Novel Senolytic to Extend Lifespan and Mitigate Organ Fibrosis https://t.co/Iv51QlhH5l
Eli Lilly to Acquire Kelonia Therapeutics for ~$7B
Eli Lilly announced a definitive agreement to acquire Kelonia Therapeutics for roughly $7 billion in cash, including an upfront payment of $3.25 billion. The deal brings Kelonia’s in‑vivo gene placement system (iGPS0) and its lead candidate, KLN‑1010, a one‑time IV gene therapy that...
AstraZeneca’s Ultomiris Cuts Urine Protein in Late-Stage Kidney Disease Trial
AstraZeneca announced that its rare‑blood‑disorder drug Ultomiris achieved the primary goal in a late‑stage trial for IgA nephropathy, a rare kidney disease. An interim analysis showed a statistically significant reduction in urine protein by week 34, with effects detectable as early...
Probiotics Shown to Relieve Constipation in Kids: Vietnam RCT
A Vietnam‑based randomized, double‑blind trial found that two Bacillus spore probiotic liquids, LiveSpo Kids and LiveSpo Preg‑Mom, significantly eased functional constipation in preschool children. Over four weeks, the probiotic groups experienced a 3.6‑ to 4‑fold reduction in low‑frequency bowel movements,...
Long‑time Applicants Finally See Chance for Rigorous Testing
Been applying to her office and others (eg welcome trust, nimh, arpa) for years asking to test/demonstrate that this isn’t magical thinking or snake oil. Glad to see things might change.
APC-Deficient Cancer Cells Rely on Single Enzyme for Survival
Researchers have identified aldehyde dehydrogenase 2 (ALDH2) as a critical survival factor for colorectal cancer cells lacking functional APC. Computational screening and lab experiments show that pharmacological inhibition of ALDH2, using agents such as disulfiram, sharply reduces proliferation and induces...
Trial Shows Safety of Novel KIR-CAR T Therapy in Solid Tumors
A Phase I dose‑escalation trial of SynKIR‑110, a novel KIR‑CAR T therapy, demonstrated safety in nine patients with advanced ovarian cancer, mesothelioma or cholangiocarcinoma. The multi‑chain design uses NK‑cell receptors to provide an on/off switch, reducing T‑cell exhaustion and side...
Dr. Kaeberlein's Optispan Podcast Series - Rapamycin and More
The Optispan podcast hosted by Dr. Kaeberlein outlines a translational protocol for 3‑hydroxyanthranilic acid (3HAA), a mouse‑tested longevity molecule. Using FDA BSA scaling, the human equivalent dose (HED) is calculated at roughly 1.1 g per day for a 70‑kg adult. Safety...
When a Text Message Tries to Short-Circuit the FDA
A text exchange between Joe Rogan and Donald Trump suggesting a quick FDA green‑light for ibogaine sparked concern about bypassing the rigorous drug‑approval process. The post explains that FDA approval is deliberately methodical, requiring phased clinical trials and safety data,...
AHA Calls on HRSA to Drop 340B Rebate Pilot, Warns of $1B Cost Hit
The American Hospital Association (AHA) has formally asked the Health Resources and Services Administration (HRSA) to abandon its proposed 340B rebate‑model pilot, arguing it would impose more than $1 billion in annual costs on safety‑net hospitals and jeopardize care for millions....
Tozorakimab Hits Primary Endpoint in Phase III MIRANDA COPD Trial
AstraZeneca announced that tozorakimab met the primary endpoint in the Phase III MIRANDA trial, delivering a statistically significant and clinically meaningful drop in moderate‑to‑severe COPD exacerbations. The result positions the IL‑33‑targeting biologic as a potential first‑in‑class therapy for a disease with...
CRISPR Screens Map Human T‑Cell Genes That Promote or Block HIV Infection
Researchers at Gladstone Institutes and UCSF used genome‑wide CRISPR activation and knockout screens in primary human CD4+ T cells to map host genes that either promote or restrict HIV infection. By optimizing infection rates to about 70%, they could perturb...
Trump’s Psychedelic Order Tests Review Norms
BioCentury’s website uses a tiered cookie system to support essential functions, personalization, marketing, advertising, and analytics. Strictly necessary cookies are always active, enabling authentication, registration, and navigation, while functional cookies enhance site features. Marketing and advertising cookies help tailor product...
Four Overlooked AACR Early-Stage Agents Worth Watching
While everyone was watching KRAS, some of AACR’s promising early stage pipeline agents were easy to miss. Here's an analysis on four examples which caught my eye and are well worth watching to see how they develop in the...
New RAS Cancer Drugs Aim to Beat RevMedicines’ Success
April20: Which RAS-based cancer drug developers are working to improve upon the dazzling pancreatic cancer results of @RevMedicines' daraxonrasib, whose stock surged 54%? Read about $RVMD plus updates on $MGTX and $TVTX in my latest StockWatch for @GENbio: https://t.co/9wQA7XsuLW
AACR26 Innovative Early Stage Developments to Watch Out For
At the AACR annual meeting in San Diego, four cutting‑edge oncology programs were showcased in a single session. Each candidate is at or just beyond the threshold for first‑in‑human trials, spanning bispecific antibodies, RNA‑based therapeutics, CRISPR‑edited cell therapies, and novel...
Nektar’s Phase II Alopecia Extension Reveals Late Responders to IL-2 Therapy
Nektar Therapeutics reported results from a Phase II extension study of its interleukin‑2 (IL‑2) therapy for alopecia areata. The data revealed a subset of patients who did not respond initially but achieved meaningful hair regrowth after prolonged exposure, raising the...
Zepzelca Plus Atezolizumab Gets First‑line Approval in Australia and Singapore
Specialised Therapeutics announced regulatory approval of ZEPZELCA (lurbinectedin) combined with atezolizumab as a first‑line maintenance therapy for extensive‑stage small‑cell lung cancer in Australia and Singapore. The approvals, granted under the FDA’s Project Orbis, add a new option for a disease...
Tenofovir Alafenamide Cuts Biological Age in Adults
An FDA-Approved Tenofovir Alafenamide-Based Antiretroviral Therapy Reduces Biological Age in Healthy Adults: First Human Proof-of-Concept for Retrotransposon-Targeted Gerotherapeutics 💫 https://t.co/FFvTmJv2zv https://t.co/DHHQ18nAmy
Novo Nordisk’s Etavopivat Hits Co‑Primary Endpoints in Phase 3 Sickle‑Cell Trial
Novo Nordisk announced that etavopivat, its once‑daily oral pyruvate‑kinase‑R activator, met both co‑primary endpoints in the Phase 3 HIBISCUS trial, lowering annualised vaso‑occlusive crisis rates by 27% and raising hemoglobin response rates to 48.7% versus 7.2% on placebo. The data position...
Personalized CRISPR Therapies Could Soon Reach Thousands — Here’s How
The FDA has proposed a "plausible mechanism" pathway to streamline approval of personalized CRISPR gene‑editing therapies. The new framework would let developers test multiple patient‑specific guide RNAs within a single trial, potentially shrinking the approval timeline from four years to...
US Speeds Research Into Mind-Altering Drugs — Including Mysterious 'Ibogaine'
On April 18, President Donald Trump signed an executive order to accelerate U.S. research on ibogaine and other psychedelics, directing the FDA to fast‑track review and providing $50 million in federal funding to match state programs. The order aims to ease...
Epigenome Proteins Enable Precise Cellular Engineering Beyond On/Off Control
Epigenome proteins generate distinct gene expression patterns, influencing cellular behavior beyond simple on/off control and offering new avenues for precise cellular engineering. generegulation
HDAC7 Loss Predicts Poor DLBCL Outcomes; Restoration Stops Tumor
Loss of the HDAC7 protein in diffuse large B cell lymphoma is linked to poorer outcomes, while restoring HDAC7 in cancer cells halts tumor growth, highlighting its potential as both a biomarker and therapeutic target. bloodcancer
AI Model 'Reads' Protein Pairs, Unlocking New Insights Into Disease and Drug Discovery
Researchers at the National University of Singapore unveiled a paired protein language model (PPLM) that learns from two interacting proteins simultaneously, a departure from traditional single‑sequence AI approaches. Trained on more than 3 million protein pairs, the model powers three tools—PPLM‑PPI,...
Major Antineoplastic Mechanisms of Combination Ivermectin-Mebendazole
Recent preclinical analyses highlight the anticancer potential of combining ivermectin and mebendazole, two antiparasitic drugs repurposed for oncology. Ivermectin suppresses proliferative signaling pathways such as Wnt/β‑catenin, Akt/mTOR, and STAT3, while mebendazole disrupts microtubule polymerization, inducing G2/M arrest. Together they inhibit...
Lab-Grown Mini Brain Models Offer New Hope for Diagnosing and Treating Alzheimer’s Disease
Johns Hopkins researchers created patient‑derived hindbrain organoids that faithfully reproduce Alzheimer’s molecular hallmarks. Using these mini‑brains, they tested the SSRI escitalopram, uncovering strikingly different serotonin‑signaling responses across individual organoids. Proteomic analysis of extracellular vesicles revealed disease‑related proteins that shifted with...
STAT+: At AACR, a Provocative Use of CAR-T, Merck’s New Thing and Cancer’s Geography Problem
At the AACR 2026 meeting, early‑phase data showed Merck‑partnered CAR‑T therapy Carvykti eliciting deep responses in 20 high‑risk smoldering multiple myeloma patients, suggesting a preventive angle for a precursor disease. The conference also featured Merck’s first glimpse of an oncology...
Promising Longevity Molecule Stalled by Misaligned Incentives
The Longevity Drug You’ve Never Heard Of This is one of those stories that captures both the promise—and the frustration—of longevity science. In this preview episode, Dr. George Sutphin (@senmorteco ), Associate Professor of Molecular and Cellular Biology at the @uarizona @UAZCancer , walks...
Trump Clears Path for Expanded Psychedelic Research to Treat Veterans’ PTSD
President Donald Trump signed an executive order directing the FDA to accelerate reviews of psychedelic therapies and earmarking at least $50 million for ibogaine research aimed at treating veteran PTSD. The order also creates a Right‑to‑Try pathway for severely ill patients...
AACR San Diego 2026: New Drugs on the Horizon
The AACR 2026 Annual Meeting in San Diego unveiled 11 first‑time disclosed oncology candidates spanning small‑molecule degraders, bispecific antibodies, T‑cell engagers and ADCs. Highlights include NEO‑811, a CRBN‑mediated molecular glue targeting HIF‑1β for VHL‑deficient renal cancer, and AZD8359, a STEAP2‑directed T‑cell...
Lilly Acquiring Kelonia In Largest Funded Biotech Startup Purchase In Years
Eli Lilly announced it will acquire Kelonia Therapeutics for up to $7 billion, marking the largest venture‑backed biotech purchase in years. The deal consists of $3.25 billion paid upfront with an additional $3.75 billion tied to clinical, regulatory and commercial milestones. Kelonia, founded only...
Delgocitinib Maintains QOL, Productivity Improvements at 52 Weeks in Chronic Hand Eczema
The open‑label DELTA 3 extension of the phase 3 delgocitinib trials shows that patients with moderate‑to‑severe chronic hand eczema who continued with twice‑daily or as‑needed delgocitinib cream for a full year maintained significant gains in disease‑specific and generic quality‑of‑life scores. Across 472...
How Trump's Psychedelics Executive Order Could Unlock Stalled Cannabis Reform
President Donald Trump signed an executive order that accelerates research, clinical trials, and Right‑to‑Try access for psychedelics such as psilocybin, MDMA and ibogaine, while leaving their scheduling unchanged. The order follows a prior, stalled effort to reschedule cannabis, highlighting the...
The ROI of Beating Cancer
A small early‑stage trial showed that a personalized mRNA vaccine triggered an immune response and extended survival for pancreatic cancer patients, a disease that kills over 90% within five years. Economists estimate that between 1988 and 2000, cancer detection and...
Lonvi’s New Capsule Boosts Mouse Lifespan by 9% and Extends Survival 64% in Trials
Lonvi announced that its experimental capsule, built around the grape‑seed compound procyanidin C1, increased overall mouse lifespan by 9.4% and extended survival by 64% from the first day of treatment. The claim, posted on the company’s website and amplified by...
A Protein Engineering Method May Lead to More Exact Cancer Treatments
Researchers at the University of Texas at Dallas unveiled ProSSpeC, a machine‑learning model that predicts protease substrate specificity by mining evolutionary data from thousands of related enzymes. The model identified engineered synthetic proteases that outperformed the commonly used tobacco etch...
Sihuan Pharmaceutical Reports Positive Phase III Data for Dirozalkib in ALK‑Positive NSCLC
Sihuan Pharmaceutical announced on April 20, 2026 that its ALK inhibitor Dirozalkib met primary endpoints in the Phase III DIAMOND‑2 trial for first‑line treatment of ALK‑positive advanced non‑small cell lung cancer. The data, presented at the 2026 AACR meeting, could...
Anktiva Needs Trial Data Before FDA Approval
For the umpteenth time, these posters have zero relevance to Anktiva unless, or until, $IBRX conducts/completes randomized clinical trials that demonstrate the drug improves outcomes for cancer patients w/ lymphopenia. @LoriMills4CA42 and the rest of you can idolize @DrPatrick ...
OpenAI Launches GPT Rosalind for Life Sciences Research
OpenAI has unveiled GPT Rosalind, a large‑language model tuned for life‑sciences research, designed to accelerate early‑stage drug discovery by automating literature review, evidence synthesis, hypothesis generation, and experimental planning. The model outperforms GPT‑5.4 on multiple chemistry, protein‑engineering and genomics benchmarks, and...
The BioPharm Brief: Oncology Innovations Continue with Durable Survival, In Vivo CAR-T, and Combination Therapies
Immunocore reported five‑year overall survival data confirming tebentafusp’s durable benefit in metastatic uveal melanoma, the first T‑cell receptor‑based therapy to show a clear survival advantage in this rare cancer. Eli Lilly announced the acquisition of Kelonia Therapeutics to accelerate in‑vivo CAR‑T...
Formulary Rules Favor Costlier Drug, Creating Unnecessary Hassle
So I need to take esomeprazole (Nexium) instead of omeprazole (Prilosec). They are both generic - at @costplusdrugs they cost $5.86 and $6.34 respectively (so the eso version is actually cheaper). But on the Blue Cross Medicare formulary, they only...
Gene Therapy Works Best for Deaf Children Aged 5‑8
AAV gene therapy for autosomal recessive deafness 9: a single-arm trial 🌟“Gene therapy for congenital deafness has shown promising results in children but lacks data in older populations… . An age-dependent therapeutic effect was observed, with optimal outcomes in 5- to...
10x Genomics Launches Atera, a SaaS Platform for Scalable Single‑Cell Spatial Biology
10x Genomics announced Atera, a cloud‑based, subscription‑driven platform that provides whole‑transcriptome spatial analysis with single‑cell sensitivity at scale. Debuted at the AACR 2026 meeting, the service signals a shift toward SaaS models in specialized biotech research.
FDA Grants Priority Review for Merck's KEYTRUDA‑Padcev Combo in Muscle‑Invasive Bladder Cancer
Merck announced that the FDA has placed its KEYTRUDA (pembrolizumab) and KEYTRUDA QLEX (pembrolizumab‑berahyaluronidase) plus Padcev (enfortumab vedotin) combos for cisplatin‑eligible muscle‑invasive bladder cancer under priority review, with a PDUFA decision due Aug 17, 2026. The move follows Phase 3 KEYNOTE‑B15 data showing...
STAT+: In Early Trial, CAR-T Results Raise Hope of Preventing Multiple Myeloma in High-Risk Patients
A phase‑I trial of CAR‑T therapy in high‑risk smoldering multiple myeloma reported that all 20 participants achieved undetectable disease after treatment. The results, presented at the AACR meeting, suggest the possibility of preventing progression to active myeloma, a deeper response...