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.
CMS-0062-P Deep Dive: What the 2026 Interoperability and Prior Authorization for Drugs Proposed Rule Actually Means for Health Tech Investors...
CMS released the proposed rule CMS-0062-P on April 10, 2026, extending prior‑authorization interoperability to prescription drugs and mandating FHIR‑based API endpoint reporting across Medicare Advantage, Medicaid, CHIP and qualified health plans. The rule sets a comment deadline of June 15, 2026 and targets compliance by October 1, 2027, introducing 24‑hour decision windows for Medicaid/CHIP drugs and 72‑hour (or 24‑hour expedited) windows for QHPs. It requires adoption of updated implementation guides such as CARIN Blue Button 2.2.0 and Da Vinci PDex 2.1.0, while retiring older STU‑2 IGs by January 1, 2028. For health‑tech founders and investors, the rule creates a clear demand signal for FHIR‑native solutions throughout the prior‑authorization stack.
Creative Bioarray Launches PDO-Based Drug‑screening Platform to Speed Oncology Research
Creative Bioarray announced a patient‑derived organoid (PDO) drug‑screening service that spans more than 20 solid‑tumor types and works with 96‑, 384‑ and 1536‑well plates. The platform aims to bridge the gap between 2D cell lines and animal models, giving researchers...
STAT+: Pharmalittle: We’re Reading About a Review of Alzheimer’s Drugs, FDA Interest in Compounded Peptides, and More
A Senate‑Democrats report released ahead of a drug‑pricing hearing shows that companies which signed pricing deals with former President Trump have continued to raise drug prices, with new therapies averaging $353,000 a year and combined profits climbing to $177 billion in...
Wegovy HD Now Available on GoodRx for $399 Per Month Self-Pay
GoodRx has launched a self‑pay option for Novo Nordisk’s Wegovy HD, a 7.2 mg semaglutide injection, priced at $399 per month and scaling to $798 for two months and $1,197 for three months. The higher‑dose formulation serves as an FDA‑approved step‑up...
Aligos Therapeutics Licenses Pevifoscorvir Sodium to Xiamen Amoytop Biotech for Greater China
Aligos Therapeutics signed an exclusive license with Xiamen Amoytop Biotech to develop and commercialize its hepatitis B candidate pevifoscorvir sodium in Greater China. The agreement provides Aligos with a $25 million upfront payment and up to $420 million in milestone fees, plus...
New Lab-Grown Organoids Accurately Mimic Pediatric Brain Tumor Biology
Scientists at St. Jude Children’s Research Hospital have created patient‑derived tumor organoids and organoid xenografts that faithfully recapitulate the genetic, epigenetic and cellular landscape of pediatric brain tumors. The 3D models, validated with DNA methylation, bulk and single‑cell RNA sequencing, and...
MGTX Buys Back XLRP Gene Therapy for $25 M
$MGTX just announced that it has entered into an asset purchase agreement with $JNJ to acquire back its bota-vec Gene Therapy program for the treatment of X-linked retinitis pigmentosa (XLRP) 🧵👇 for an upfront cash payment of $25M & a...
Breakthrough HIV Drug Is Out Of Reach For Many Who Need It Most
Gilead's long‑acting HIV pre‑exposure prophylaxis, lenacapavir, demonstrated almost 100% efficacy in trials and requires only two injections per year. The company can produce up to 10 million doses by 2026 but has pledged just 3 million through the Global Fund and PEPFAR,...
GLP‑1 Drugs Don’t Disproportionately Reduce Functional Muscle, DXA Misleads
1/2) New research suggests GLP-1 weight loss drugs probably do NOT cause disproportionate loss of functional lean muscle. The confusion has arisen from the fact that DXA scans capture and pool a lot of different organs and tissues into the category...
Duchenne Kids Show Dramatic Improvement After Microdystrophin Treatment
A couple of weeks ago when I visited Solid Bio, they showed me some videos of Duchenne kids pre and post microdystrophin treatment that I was amazed by - but they weren't sharable yet. Bo just presented them at Needham...
Interna Therapeutics Collaborates with Daiichi Sankyo to Develop MNM-Based Targeted Delivery Technologies
Interna Therapeutics announced a research collaboration with Daiichi Sankyo’s Boston Research Institute to evaluate its MNM (Molecular Nanoparticle Matrix) technology as a delivery enhancer for targeted therapeutics. The partnership will integrate MNM molecules with Daiichi Sankyo’s targeting approaches, beginning with...
Anti‑amyloid Drugs Show Trivial, Non‑meaningful Cognitive Benefit
The effect of current anti-amyloid drugs vs Alzheimer's disease is "trivial" for cognitive function or dementia severity, not clinically meaningful, from a systematic @cochranecollab review of 17 trials, >20,000 patients https://t.co/9A6PeZOyAt https://t.co/c1PCOYZxws
FDA Approves Kidney Drug Despite No Functional Benefit
This week's Biotech Scorecard newsletter: $TVTX Travere Therapeutics' drug for a rare kidney disease doesn’t improve kidney function. The FDA approved it anyway. https://t.co/63hDsPfOcT
Lilly's Obesity Pill Heads for Diabetes Filing After Heart Risk Trial
Eli Lilly’s newly approved obesity medication, marketed as Foun…, demonstrated a 16% lower incidence of major cardiovascular events compared with a standard insulin regimen in a recent trial. The data, presented by Lilly, suggest the drug not only aids weight loss...
Roche Initiates New Elevidys Trial Targeting EU Approval
Roche to launch another Elevidys trial, with eyes on European approval https://t.co/eAANNiihpt via @DrewQJoseph $SRPT
Phase III Study of Lilly’s Foundayo, Reaffirms Cardiovascular and Overall Safety Profile, Plus Improvements to Cardiometabolic Health
Eli Lilly announced that its oral GLP‑1 drug Foundayo (orforglipron) achieved the primary endpoint in the Phase 3 ACHIEVE‑4 trial, demonstrating non‑inferior major adverse cardiovascular event risk versus insulin glargine. The study, the largest of its kind with over 2,700 participants...
What Happens When You Stop Ozempic or Mounjaro? New Study Reveals Surprising Weight-Loss Results
A Cleveland Clinic analysis of nearly 8,000 Ohio and Florida adults who stopped GLP‑1 injectables—semaglutide (Ozempic/Wegovy) or tirzepatide (Mounjaro/Zepbound)—found minimal weight regain. Patients treated for obesity lost an average of 8.4% of body weight and regained only 0.5% after one...
Phytochemical Blend Boosts Recovery Metrics in Small Study, Researchers Report
Researchers from Loughborough University and the University of Nottingham reported that a nine‑day supplement containing calcium fructoborate, turmeric and pomegranate extract improved pressure‑pain threshold by 21% and enhanced sleep quality in recreational athletes. Funded by VDF FutureCeuticals, the double‑blind trial...
STAT+: Roche to Launch Another Elevidys Trial, with Eyes on European Approval
Roche announced a new Phase 3 trial of Elevidys, the gene‑therapy for Duchenne muscular dystrophy, targeting European approval after a negative EMA review last year. The study will enroll roughly 100 boys in the early stages of the disease and compare...
FDA to Review Easing Restrictions on Peptide Injections Backed by RFK Jr.
The Food and Drug Administration announced a July meeting to consider easing limits on more than half a dozen peptide injections championed by Health Secretary Robert F. Kennedy Jr. The move pits the secretary’s “Make America Healthy Again” agenda against...
LEO Pharma Reports the US FDA’s sNDA Acceptance of Anzupgo for Use in Children with Chronic Hand Eczema
LEO Pharma announced that the U.S. Food and Drug Administration has accepted a supplemental New Drug Application for Anzupgo (delgocitinib) cream targeting adolescents aged 12 to 17 with moderate‑to‑severe chronic hand eczema. The acceptance follows the Phase III DELTA TEEN trial, which...
AscentX Medical’s Dr. Sandhu on a New Approach to Treating GERD
AscentX Medical is developing G125, a regenerative injectable biomaterial designed to reinforce the lower esophageal sphincter in patients with gastroesophageal reflux disease (GERD). The platform delivers a biocompatible scaffold via a patented needle that integrates with tissue, promoting collagen growth...
Stanford Team Generates Light Inside Deep Tissue Using Ultrasound‑Activated Nanoparticles
Stanford scientists have shown that ultrasound can activate specially engineered nanophosphors circulating in the bloodstream to emit light deep inside living tissue. The breakthrough, demonstrated in mice, could replace invasive fiber‑optic probes for optogenetics, imaging and cancer treatment.
Johnson & Johnson’s Experience Navigating The Commissioner’s National Priority Voucher Program
Johnson & Johnson’s hematology division secured FDA approval for a new multiple myeloma regimen—Tecvayli plus Darzalex Faspro—through the FDA Commissioner’s National Priority Voucher pilot. The approval came just 55 days after filing, marking the first blood‑cancer therapy to use the voucher...
Breakthrough Science, Unequal Survival
Recent breakthroughs such as routine stem‑cell transplants and CAR‑T therapy have transformed treatment for several blood cancers, delivering long‑term remission for patients once deemed incurable. Yet blood cancer remains the UK’s third‑largest cancer killer, with 310,000 people living with or...
Endovascular Therapy Shows Promise for Post‑Thrombotic Syndrome
Endovascular Therapy for Post-Thrombotic Syndrome — A Randomized Trial [Apr 13, 2026] Vedantham et al. for the C-TRACT [Chronic Venous Thrombosis: Relief with Adjunctive Catheter-Directed Therapy] Trial Investigators @NEJM https://t.co/Ayd4Fa0ARl #NCT03250247 #VTE https://t.co/6iD1NUQdgX
Rebuild, Don’t Just Relieve Pain: The Peptide Revolution for Lasting Joint Health
In this episode, host Steve discusses chronic inflammation and joint pain with Dr. Peter McCullough, chief scientific officer of a wellness company. Dr. McCullough explains how traditional pain relievers only mask symptoms, while their peptide‑based products—Regenerate (oral drops) and Therablu (topical...
MRNA Vaccines Activate Unconventional CD8+ T Cells
A recent study published in *Nature Immunology* shows that mRNA COVID‑19 vaccines trigger a previously underappreciated subset of CD8+ T cells with innate‑like characteristics. These unconventional cells, resembling mucosal‑associated invariant T (MAIT) and γδ T cells, expand rapidly after the...
Innovation for a New Era of Cancer Care
The UK’s National Cancer Plan, released earlier this year, prioritises expanding genomic testing, liquid biopsies, and a national inherited cancer registry to modernise cancer pathways. Johnson & Johnson argues that linking these precision diagnostics to innovative therapies is essential for...
Cells Have a Secret 'Courier System' That Could Open Hard-to-Reach Targets for RNA and Gene Therapies
University College Dublin researchers have identified a previously unknown cellular "courier system" in which nanoparticles acquire a protein‑RNA "condensate corona" that shuttles functional biomolecules between cells. The corona forms a stable droplet that protects its cargo, escapes degradation, and delivers...
Decoding HBx–Smc6 Interaction: Advancing HBV Inhibition
A study in Cell Research reveals how hepatitis B virus protein HBx binds the host Smc6 subunit, triggering ubiquitin‑mediated degradation that lifts restriction on cccDNA and sustains infection. Cryo‑EM resolved the interface at near‑atomic resolution, identifying a pocket on Smc6 and...
Nucleai and Sirona Dx to Provide New Proteomics Solution for Pharma Companies
Nucleai and Sirona Dx have formed a partnership to deliver an end‑to‑end spatial proteomics solution for pharmaceutical and biotech companies. The joint offering unifies assay design, high‑quality imaging, and AI‑driven analytics to turn complex tissue data into actionable biological insights....
Phantom Neuro Secures Approval for Muscle-Machine Interface Trial
Phantom Neuro has received regulatory clearance to launch its first‑in‑human trial of the Phantom X muscle‑machine interface in Melbourne, Australia. The early feasibility study, called CYBORG, will enroll up to ten unilateral below‑elbow amputees who will receive a single outpatient implant...
Boehringer Ingelheim and Zai Lab Team up for Dual DLL3 Therapy Study
Boehringer Ingelheim and Zai Lab have launched a Phase Ib/II trial that pairs Boehringer’s DLL3‑directed T‑cell engager obrixtamig with Zai Lab’s DLL3‑targeting ADC zoci. The study will evaluate safety, tolerability and early efficacy in patients with extensive‑stage small‑cell lung cancer and other neuroendocrine...
Boehringer Ingelheim and Zai Lab Team up for Dual DLL3 Therapy Study
German pharma giant Boehringer Ingelheim and China‑based Zai Lab announced a clinical collaboration to evaluate a dual DLL3‑targeting regimen in extensive‑stage small‑cell lung cancer and other neuroendocrine tumors. The Phase Ib/II study will combine Boehringer’s bispecific DLL3/CD3 T‑cell engager obrixtamig with...
Freya Biosciences Advances Microbial Treatment for IVF Implantation Failure
Freya Biosciences announced that its microbial immunotherapy designed to address IVF implantation failure has progressed to a Phase 2 mid‑stage trial after demonstrating safety and early efficacy in healthy volunteers. The therapy leverages modulation of the uterine microbiome to improve endometrial...
AOP Health Reports the US FDA Approval Rapiblyk (Landiolol) for Pediatric Patients with Supraventricular Tachycardia
The U.S. Food and Drug Administration has granted approval for Rapiblyk (landiolol) for pediatric patients from birth through 17 years old suffering from supraventricular tachycardia (SVT). The decision follows the LANDI‑PED trial, which enrolled 60 children and demonstrated a greater...
Patient‑Driven Platform Accelerates Multiple Myeloma Research
.@HealthtreeMM Hub: A Patient-Derived, Patient-Driven Clinical Cancer Information Platform Used to Overcome Hurdles & Accelerate Research in Multiple Myeloma [3/10/22] @NSweeneyPhd @AhlstromJenny @FaithEDavies1 @mtmdphd @JCOCCI_ASCO https://t.co/WOwLb9Eozv @myelomacrowd #mmsm https://t.co/6HieAzjSES
New Trial Compares Dara‑Bor‑Dex vs Cy‑Bor‑Dex in Myeloma‑Induced Kidney
EAA241 - Ph 2 RCT Dara-Bor-Dex vs Cy-Bor-Dex in the treatment of Newly Diagnosed Multiple Myeloma with Light Chain Cast Nephropathy (LCCN) [Study activated 8/11/25] @keruakous https://t.co/1NgvVZ3fTA #NCT07085728 #mmsm @eaonc https://t.co/KJfqVVcwEr
FDA Looks East—Here’s What Industry Needs to Know
The FDA’s FY2027 budget proposes $2.5 million and five new full‑time staff to open foreign offices in Hanoi, Vietnam, and Tokyo, Japan, expanding its inspection footprint in East Asia. The request follows FY2026 congressional language urging permanent presence for unannounced inspections...
Daratumumab Boosts Revlimid Efficacy in Smoldering Myeloma
#EAonc EAA173 - Daratumumab to Enhance Therapeutic Effectiveness of Revlimid in Smoldering Myeloma (DETER-SMM) - PI: @nsc_natalie https://t.co/VtBMJUjI5X Activated: Apr 30, 2019 #mmsm @eaonc #NCT03937635 @VincentRK @mweissmdphd https://t.co/SKPUQiSaU4
Phase 3 Trial Tests Daratum
.@SWOG S2213 Ph3 RCT Dara-VC Induction Followed by ASCT or Dara-VCD Consolidation & Daratumumab Maintenance in Pts w/ Newly Diagnosed AL Amyloidosis [Activated: 12/1/23] https://t.co/OizUfJCc2c #mmsm #bmtsm https://t.co/Hq9VlRCTCI
J&J Lifts 2026 Sales Outlook to $100 B, Defying Pricing Headwinds
Johnson & Johnson announced a 2026 sales guidance of $99.5‑$100.5 billion, topping the Street’s $98.9 billion consensus. The raise comes after a $3.05 billion acquisition and a pricing‑cost deal, underscoring strong oncology momentum and operational resilience.
Four Candidates Target Ultra‑Rare Polish PUS3 Syndrome
“We have four drug candidates for patients with an ultra-rare disease with Polish origins, the PUS3 syndrome; one of these candidates will be used in practice,” Lisowski said. https://t.co/GTJbPQSdXQ
When to Stop Myeloma Treatment: MRD Insights
MT @hhashmi87 #IMS25 Great Meet The Expert talk by @bdermanmd on MRD in Myeloma Risk vs benefit Who can stop treatment Active surveillance post discontinuation #mmMRD #mmsm https://t.co/FIDPrE7QRb
FDA Issues Draft Guidance to Standardize Safety Assessment of Genome‑Editing Gene Therapies
The U.S. Food and Drug Administration today released a draft guidance titled “Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next‑Generation Sequencing.” The document sets uniform pre‑clinical and IND requirements for ex vivo and in vivo CRISPR‑based...
MRD‑negative Patients May Stop Myeloma Maintenance Therapy
Discontinuation of maintenance therapy in multiple myeloma guided by multimodal measurable residual disease negativity (MRD2STOP) - @bdermanmd et al. @ajjakubowiak #ASCO24 Abstract 106 https://t.co/FBTY7SnCxK #NCT04108624 #mmsm #mmMRD
ODAC Approves MRD as Early Endpoint for Myeloma Trials
A Historic Turning Point: ODAC Unanimously Votes [4/12/24] in Favor of MRD Testing as an Early Endpoint in Myeloma Clinical Trials to Support Accelerated Approvals of New Treatments [Apr 18, 2024] @IMFmyeloma https://t.co/eDOgIrpVeR #mmMRD #mmsm #ctsm @FDAOncology https://t.co/V4wrNgvTrG
Clinicians Show Mixed Adoption of Myeloma MRD Testing
Clinician attitudes and practices toward measurable residual disease in multiple myeloma [Jun 7, 2020] @bdermanmd @jagoda_jasielec @ajjakubowiak Brit J Haematol https://t.co/8zhxYfYwZQ #mmsm #mmMRD https://t.co/mPtRCjnB9f
Vaccines Vanishing Threatens Global Health Catastrophe
The Horrors That Could Lie Ahead if Vaccines Vanish — @propublica good article on the work of @NathanLo3579 @Stanford my former mentee and amazing colleague https://t.co/n9oUgkvOZ7