Today's Pharma Pulse
CMS postpones GLP‑1 Medicare pilot, hitting Lilly and Novo shares
The Centers for Medicare & Medicaid Services indefinitely delayed its BALANCE financing model aimed at expanding Medicare coverage for GLP‑1 obesity drugs. The postponement knocked Eli Lilly’s stock down about 2% and Novo Nordisk’s by roughly 4%, with analysts projecting a near‑term revenue loss of up to $500 million for Lilly that could rise to $3.3 billion.
Also developing:
By the numbers: Asahi Kasei acquires German biotech Aicuris for $920.7M
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 tumor‑microenvironment modulators. The lineup offers a snapshot of where oncology drug design is heading. Executives and investors see these early signals as a barometer for the next wave of high‑value pipeline assets.
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 ...
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...
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...
ViewsML Secures $4.9 Million to Help Scientists Virtually Analyze Tissue Samples
ViewsML, a Vancouver AI startup, closed a C$4.9 million seed round (approximately US$3.6 million) to commercialize its virtual biomarker platform. The technology uses artificial intelligence to extract quantitative biomarker data from pathology images without traditional chemical staining. By turning a lab‑bound process...
AB Science: New Publication on Medrxiv Demonstrating Substantial Survival Benefits and Preserved Quality of Life with Masitinib in ALS Patients
AB Science announced a medRxiv preprint showing its tyrosine‑kinase inhibitor masitinib dramatically improves survival for amyotrophic lateral sclerosis (ALS) patients. The data reveal a 5‑year survival rate of 42.3% overall and 52.9% among those treated before complete functional loss, roughly...
Trump, FDA Plan to Expedite Psychedelic Development
President Donald Trump publicly endorsed psychedelic medicines as a breakthrough for severe mental health disorders, pledging to fast‑track their research and development. The White House announcement coincides with the FDA’s plan to create an accelerated review pathway for psychedelic drug...
FDA Grants Priority Review for Padcev Plus Keytruda in Bladder Cancer Treatment
The U.S. Food and Drug Administration has granted Priority Review to the peri‑operative combination of Padcev (enfortumab vedotin) and Keytruda (pembrolizumab) for muscle‑invasive bladder cancer (MIBC). The Phase 3 EV‑304 trial demonstrated a 47% reduction in recurrence, progression or death and a...
Novartis Cuts Two Programs in Cancer-Related Blood Clots
Novartis announced the termination of two late‑stage programs targeting cancer‑related blood clots after a pivotal trial demonstrated inferior efficacy versus existing anticoagulants. The candidates, one of which Novartis out‑licensed in 2019, were being developed to address the high unmet need...
Pharmaceutical Executive Daily: Eli Lilly Acquires Kelonia Therapeutics
Eli Lilly announced a $7 billion deal to acquire Kelonia Therapeutics, adding genetic‑medicine and novel delivery technologies to its pipeline. In parallel, President Trump issued an executive order to speed FDA reviews of psychedelic therapies, signaling a policy shift toward emerging mental‑health...
CRISPR Advances Outpace Ethics in Germline Editing Debate
In 2018, He Jiankui edited the genomes of two human embryos, brought them to term, and went to prison for it. The babies are now children. We don't know how they're doing. Seven years later, base editing and prime editing can...
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...
Viatris Recalls Extended-Release Xanax over Dissolution Test Failure
Viatris has issued a Class II recall of a single batch of its extended‑release Xanax (Xanax XR) after dissolution testing showed the tablets did not meet release specifications. The affected bottles, each containing 60 3‑mg tablets, were manufactured in Ireland and are being...
Funding Boosts AI for 3D Protein and RNA Mapping
Funding will support the development of advanced AI methods to map proteins and RNA structures in three dimensions. https://t.co/L6ZSpg8iMW #infrastructure #IoT #AI #5G #cloud #edge #futureofwork @virginia_tech https://t.co/7K7X1E9NpH
JRT: LSD-Derived Antidepressant Boosts Brain Spines without Hallucinations
One dose: > 46% more dendritic spines in prefrontal cortex > 100x more potent than ketamine as an antidepressant What is it? LSD with a two atom swap. > no trip > no hallucinations > no schizophrenia signature It's called JRT. https://t.co/sozMqlJctc
Substack Live | Flagship Pioneering Announcement: Building on the Code of Life
Flagship Pioneering is hosting a Substack Live session on April 21 at 12:30 pm ET featuring origination partner Jake Rubens. The event will unveil a new initiative that leverages the "code of life" to advance synthetic‑biology‑based therapeutics. Rubens, an MIT‑trained synthetic biologist...
Balaji Predicts Booming Market for Bro Biotech
.@theojaffee asks Balajis for his thoughts on Clavicular, Balajis says he’s cheering for Clavicular because he’s tapping into bro biotech/bro science and that’s set to be a major market. Peptides, etc https://t.co/CgSt6rg1zI
LLY's Second in Vivo CAR‑T Deal via Kelonia Buyout
Kelonia turns #ASH25 late-breaker into buyout, with $LLY's second move into in vivo Car-T. Via @ApexOnco -> https://t.co/NPhdnuhPu3 $AZN $GILD
Elisrasib Shows Strong Response Rates in Advanced KRAS‑G12C Lung Cancer
Elisrasib, a next‑generation KRAS‑G12C inhibitor, produced clinical benefit in a 165‑patient cohort of advanced non‑small cell lung cancer at the AACR 2026 meeting. The oral drug, given at a 600 mg daily dose, showed durable responses in patients previously treated with...
Key Overlooked Factors in Clinical Trial Design
New presentation on things we often forget to consider when designing a clinical trial: https://t.co/BVlSCIHdLu #Statistics #clinicaltrials
Inside the $3.25B Eli Lilly Acquisition: Memo and Deck
Read the memo and slide deck that led to th creation of the company Eli Lilly bought for $3.25 billion today https://t.co/cNoTs7M4YM
FDA Accepts Ultragenyx BLA for UX111 Gene Therapy Targeting Sanfilippo A
Ultragenyx announced that the U.S. Food and Drug Administration has accepted its resubmitted Biologics License Application for UX111, an AAV9 gene therapy for Sanfilippo syndrome Type A. The agency set a PDUFA action date of September 19, positioning UX111 as...
Nektar Jumps as Alopecia Drug Shows Promise
Nektar surges again as alopecia drug shows new promise in extension study https://t.co/UV0ccY4HiR @ByJonGardner $NKTR + 25% $LLY
STAT+: Eli Lilly’s $3.25 Billion Acquisition of Kelonia Therapeutics Caps Startup’s Tortuous Ride
Eli Lilly announced a $3.25 billion acquisition of Kelonia Therapeutics, a small biotech focused on cell therapies for cancer and autoimmune diseases. The deal may include additional payments if Kelonia meets clinical, regulatory, and commercial milestones. Kelonia, formerly Elcano Therapeutics, survived on...
Merck Partner Reveals Bispecific Data; Biogen Pays $100M for Antibody Rights
Merck’s partner Sino Biopharm disclosed first‑in‑human results for its PD‑1×VEGF bispecific antibody, MK‑2010, reporting that six of 11 lung‑cancer patients achieved a response at a low dose. A second cohort showed four responses among nine patients, indicating activity across dosing...