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.
Pharmaceutical Executive Daily: Eli Lilly Releases Updated Safety Data for Foundayo
Eli Lilly has released topline safety results from its Phase III Achieve‑4 trial for Foundayo, addressing FDA‑requested evidence on cardiovascular, liver and gastric safety. The FDA’s post‑approval letter, issued on the day of approval, demanded trial data rather than observational studies. Concurrently, ICER and Verdant Research issued a white paper urging tighter controls on surrogate endpoints and faster confirmatory trials. A commentary notes the FDA’s shift to a single‑trial default, highlighting a broader move toward data‑driven drug development.
FDA Scientists Working in Satellite Laboratories Across U.S. Help Prevent Harmful Drugs From Reaching Americans
The FDA’s National Forensic Chemistry Center runs satellite laboratories inside high‑volume international mail hubs in Chicago, New York, Los Angeles, Miami and Honolulu. These compact labs use spectroscopy and mass spectrometry to screen incoming packages for counterfeit, unsafe or novel drug compounds...
RSV Vaccines Work to Prevent Hospitalization
Recent clinical data show that newly approved respiratory syncytial virus (RSV) vaccines dramatically cut hospital admissions. In infants, the vaccine lowered hospitalization risk by roughly 70%, while older adults experienced a 50% reduction in severe cases. The FDA has accelerated...
Roche to Start Phase III Trial to Broaden Access to Elevidys in Duchenne Muscular Dystrophy
Roche announced a global, pivotal Phase III trial of Elevidys, its gene‑therapy for Duchenne muscular dystrophy, targeting roughly 100 early‑ambulatory boys. The 72‑week, placebo‑controlled study will assess change in time‑to‑rise‑from‑floor velocity as the primary efficacy endpoint. Results are intended to bolster...
Study Data Technical Conformance Guide - Technical Specifications Document
The FDA has issued the final Study Data Technical Conformance Guide (Docket FDA-2014-D-0092), outlining technical specifications for electronic study data submissions. The guidance clarifies the agency’s expectations but remains non‑binding, allowing sponsors to use alternative approaches that meet regulatory requirements....
Financial Transparency and Efficiency of the Prescription Drug User Fee Act, Biosimilar User Fee Act, and Generic Drug User Fee...
The FDA announced a public meeting on June 23, 2026 to discuss financial transparency and efficiency of the Prescription Drug User Fee Act (PDUFA VII), Biosimilar User Fee Act (BsUFA III) and Generic Drug User Fee Act (GDUFA III). The session will present five‑year financial...
Batch of Anti-Anxiety Drug Xanax Recalled, F.D.A. Says
The FDA announced a recall of a single batch of Xanax XR, the extended‑release formulation of the popular anti‑anxiety medication. Viatris, the drug’s distributor, is pulling 51 bottles of 3 mg tablets because they may not dissolve properly, potentially altering drug...
From Static Snapshots to Dynamic Protein Modeling
AlphaFold solved protein structure prediction. That's a snapshot. But biology isn't static. Proteins flex, shift, and interact across time. Drug targets have multiple conformations. Cells don't act alone. The next hard problem is capturing that motion, and building models that reflect...
Toward Equitable Access to Cell and Gene Therapies: Rethinking Co-Payments
Cell and gene therapies now command one‑time price tags exceeding $3 million, creating affordability challenges for the U.S. health‑care system. While patient cost sharing represents a tiny slice of total spending, deductibles and coinsurance can still impose thousands of dollars in...
Viatris Recalls 3 Mg Xanax XR Lot Over Dissolution Failure, FDA Class II Alert
Viatris has voluntarily recalled a single lot of its 3 mg extended‑release Xanax tablets after the FDA identified failed dissolution specifications. The Class II recall, covering bottles distributed nationwide between Aug. 27 2024 and May 29 2025, could lead to reduced drug effectiveness but no adverse...
FDA Webinar on the RCT-DUPLICATE Initiative: Emulating Randomized Clinical Trials with Non-Randomized Real-World Data Studies - 04/29/2026
On April 29, 2026 the FDA hosted a free public webinar to update stakeholders on the RCT‑DUPLICATE demonstration project. The initiative compares non‑randomized real‑world data (RWD) studies with randomized controlled trial (RCT) outcomes to assess causal validity. Findings show strong...
ATAI Fully Acquires Ibogaine Program, Champions Psychedelic Shift
I am a strong believer in ibogaine, which is one of the reasons why @ataibeckley acquired the residual interest in its ibogaine program in Q4 2023 and now owns it 100%. I’m very encouraged to see the administration taking a positive...
FDA Extends Review of Savara’s Molgramostim BLA for PAP
Savara’s inhaled GM‑CSF therapy, molgramostim, received a three‑month FDA review extension, moving the PDUFA target action date to November 22, 2026. The extension follows the agency’s classification of the company’s recent data submissions as a major amendment, but it did not signal...
Indian Researchers Launch Fluorescent MOF Sensor for Fast Nicotine and Cotinine Detection
Researchers at the Institute of Nano Science and Technology (INST) in Mohali have introduced a fluorescent iron metal‑organic framework nanosphere that lights up in the presence of nicotine or its metabolite cotinine. Published in Nanoscale, the “turn‑on” sensor offers visual,...
FDA Grants RMAT Designation to Grace Science’s GS-100 Gene Therapy for NGLY1 Deficiency
Grace Science, LLC received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA for its investigational gene therapy GS-100 targeting NGLY1 deficiency. The designation, based on early Phase 1/2/3 data showing motor and cognitive gains, promises a faster regulatory...
Musterdepot – Zukunft: In Unsicheren Zeiten Auf Die Moderne Infektionsmedizin Setzen
Investors are shifting focus from over‑valued tech names to the neglected anti‑infective sector as 2026 sees a market rotation. High‑priced AI and cloud stocks can tumble on modest earnings misses, prompting fund managers to seek defensive biotech exposure. Modern infection...
Regulatory Actions for April 16, 2026
BioWorld’s April 16, 2026 regulatory snapshot lists a flurry of biopharma and med‑tech actions, ranging from new drug submissions to market approvals and designations. Companies such as Airs, Aligos, AOP, Arbutus, Cala, Dogwood, Immutep, Leadiant, Northstar, Opna, Reach Surgical, Waters and Xspray...
STAT+: Researchers Behind GLP-1 Obesity Drugs Advance New Approach: Drop GLP-1 as a Target
Researchers who helped create GLP‑1 obesity drugs such as Eli Lilly’s Zepbound are now testing a different strategy. A team led by Richard DiMarchi and Matthias Tschöp engineered a molecule that activates GIP and glucagon receptors, showing weight‑loss results in...
First Large Rapamycin Trial Aims to Boost Human Healthspan
Finally—this is the kind of study the field has been waiting for. A 720-person randomized clinical trial testing low-dose rapamycin in adults 65+ is about to get underway at @UAZPharmacy , with the goal of answering a simple but critical question:...
Drug Discovery Expands Beyond AI to New Frontiers
The marked expansion of paths and methods for new drug development including and beyond AI @Joseph_C_Wu @james_y_zou @WuXuekun @ScienceMagazine https://t.co/hTJ4Rt181n https://t.co/H3U5BNR1ER
Planview and Ozmosi: Bringing AI-Powered Insight to Pharmaceutical Portfolio Decisions
Planview has partnered with Ozmosi to embed curated clinical‑trial and market intelligence into its portfolio management platform, enhancing AI‑driven decision‑making for pharmaceutical companies. The integration supplies real‑time external signals, allowing teams to benchmark assumptions, identify pipeline gaps, and monitor competitive...
1q22 Gain Predicts Poor Prognosis in Myeloma
Prognostic significance of acquired 1q22 gain in multiple myeloma [Oct 28, 2021] Hadiyah Y Audil et al. @myelomaMD Am J Hematol https://t.co/qUKCUdnZPH #mmsm #PrecisionMedicine #oncopath HT @EagleMyeloma https://t.co/vd0BVZ6bRq
1q Abnormalities Define Distinct Proteomic Landscape in Myeloma
Proteomic profiling revealed unique disease biology associated with 1q abnormalities in multiple myeloma [Apr 14, 2026] Mangalaparthi et al. @Nature_NPJ https://t.co/XTcJZIGFh6 #mmsm #PrecisionMedicine HT @Myeloma_Doc https://t.co/XyrxJ29elO
FDA Approves Fast-Acting Heart Drug for Children
Austrian firm AOP Health received FDA approval for its fast‑acting IV beta‑blocker landiolol, marketed as Rapiblyk, to treat supraventricular tachycardia in pediatric patients. The decision follows the LANDI‑PED study, which enrolled 60 children and demonstrated more than a 20% reduction...
Scientists Propose Abandoning GLP‑1 as Obesity Target
Researchers behind GLP-1 obesity drugs advance new approach: Drop GLP-1 as a target https://t.co/ADzYb2Zqal via @statnews
FDA Pushes New
First autism, now low T - FDA continues to do its own literature searches and encourage sponsors to apply for new indications (never saw this in prior admins — with the exception of updating old cancer drug labels to include...
Developers Back Alzheimer’s Drugs Despite Report Suggesting Lack of Efficacy
A new Cochrane review of 17 trials involving 20,342 patients concludes that anti‑amyloid drugs for Alzheimer’s disease deliver only trivial or no clinically meaningful cognitive benefit and may increase the risk of amyloid‑related imaging abnormalities (ARIA). Eli Lilly’s donanemab (Kisunla) and...
Replimune Downsizes While MeiraGTx Regains Eye Therapy
Replimune cuts staff; MeiraGTx reacquires eye gene therapy https://t.co/LqGPyNzRA3 $REPL $MGTX $JNJ $BIIB $RVMD $APLS
Third‑generation T‑cell Engagers Advance Cancer Immunotherapy
Cancer immunotherapy keeps revving up. Now onto a 3rd generation of T cell engagers https://t.co/h05f8h3i8L https://t.co/PP5XE8WWDr
STAT+: Cochrane Review Reignites Alzheimer’s Amyloid Wars
The FDA announced it will convene an external advisory panel to revisit rules on compounded peptides, with meetings slated for July and a follow‑up before February 2027. A new Cochrane review has reignited controversy over amyloid‑targeting Alzheimer’s therapies, questioning their...
Tumour Cells Use a Genetic Trick to Become Drug-Resistant
Researchers have identified that many tumor cells evade traditional Mendelian inheritance, enabling them to acquire drug‑resistance traits far faster than previously understood. The genetic maneuver involves non‑standard chromosome segregation and gene amplification, which let cancer cells adapt to chemotherapy pressures....
Pendulum Expands Mayo Clinic Collaboration Into Women’s Health and Dermatology
Pendulum Therapeutics is deepening its partnership with Mayo Clinic to launch interventional microbiome trials in women’s health and dermatology. The new studies will examine bone health in breast‑cancer patients, menopause transition, and the gut‑skin axis, moving beyond associative research. Pendulum...
New Drug Extends Rheumatoid Arthritis Remission Years Post‑Treatment
Breakthrough Drug Delays Rheumatoid Arthritis for Years After Treatment Ends. https://t.co/03MjFzkPTM #research #RheumatoidArthritis #Arthritis #Pharma #healthcare
ICER and Verdant Research Publish White Paper Examining FDA’s Accelerated Approval Pathway
The Institute for Clinical and Economic Review (ICER) and Verdant Research released a white paper urging reforms to the FDA’s accelerated approval pathway. The report highlights persistent issues such as surrogate‑endpoint uncertainty, delayed confirmatory trials, and opaque decision‑making. It proposes...
Acerand Therapeutics Updates the Phase I/II Study of ACE-106 (ACE-86225106), a Highly Selective and Potentially Best-in-Class PARP1 Inhibitor, in Advanced...
Acerand Therapeutics reported updated Phase I/II data for ACE‑106, a highly selective PARP1 inhibitor, in 57 heavily pre‑treated patients with advanced solid tumors. The drug showed no dose‑limiting toxicities and a safety profile that appears better than approved PARP inhibitors....
BMS-986482
Bristol Myers Squibb disclosed BMS-986482, a CRBN‑mediated degrader that targets the IKZF1‑4 transcription factors, at the ACS Spring 2026 First‑Time Disclosures session. The molecule entered a combined Phase 1/2 study aimed at patients with advanced solid tumors, marking BMS’s entry into...
Now Published - OCEANIC-STROKE: Asundexian Prevents Recurrent Strokes, With No Added Bleeding
The phase III OCEANIC‑STROKE trial showed that adding Bayer's factor XIa inhibitor asundexian to standard antiplatelet therapy reduced recurrent ischemic strokes from 8.4% to 6.2% over two years, without raising major bleeding risk. The study enrolled 12,237 patients with recent non‑cardioembolic stroke...
7 Things to Know About the Nonprescription Drug Product with an Additional Condition for Nonprescription Use Final Rule
The FDA’s Center for Drug Evaluation and Research issued a final rule establishing an “Additional Condition for Nonprescription Use” (ACNU) pathway for over‑the‑counter medicines. Under ACNU, a drug can be sold without a prescription but must include an extra consumer‑screening...
Electronic Submission of Adverse Event Reports to FDA Adverse Event Reporting System (FAERS) Using International Council for Harmonisation (ICH) E2B(R3)...
The U.S. Food and Drug Administration held two public meetings on April 4 and November 7, 2023 to outline upgrades to electronic adverse event reporting using the ICH E2B(R3) standard. The sessions targeted both pre‑market and post‑market safety surveillance programs managed by CDER and...
Promega to Showcase Oncology Research Tools and Companion Diagnostics at AACR Annual Meeting 2026
Promega will showcase a suite of oncology‑focused tools at the AACR Annual Meeting in San Diego, including its Lumit® hKi‑67 proliferation assay, the TarSeer™ BRETSA™ target‑engagement platform, and FDA‑cleared OncoMate® MSI companion diagnostic. The company also unveiled pre‑configured automated nucleic‑acid...
Eltrombopag Proves More Cost-Effective than IVIG Perioperatively
Cost-effectiveness of eltrombopag vs intravenous immunoglobulin for the perioperative management of immune thrombocytopenia [Jan 31, 2022] @ThisIsManraj et al. @BloodJournal https://t.co/2hr1CFmUro #ITP https://t.co/Fz6ZRjJHlk
CM313 Shows Promise as New ITP Therapy
A Novel Anti-CD38 Monoclonal Antibody [CM313] for Treating Immune Thrombocytopenia [Jun 19, 2024] Chen et al. @NEJM https://t.co/o9wf1ogUi4 #NCT05694767 #ITP https://t.co/aSXzpOHOtF
Retatrutide Vs. Zepbound: How Does Lilly’s Next-Gen Obesity Drug Compare to Tirzepatide?
Eli Lilly’s investigational triple‑agonist retatrutide is delivering weight‑loss results that eclipse its approved dual‑agonist Zepbound (tirzepatide), with Phase III trials reporting up to 28.7% body‑weight reduction over 68 weeks and HbA1c drops of about 2.0%. Zepbound, already a blockbuster generating roughly...
Fostamatinib Shows 79% Response, 54% Complete Remission in ITP
Fostamatinib [Syk inhib] effectiveness and safety for immune thrombocytopenia in clinical practice [August 8, 2024] @tomasjosegonza2 et al. @BloodPortfolio https://t.co/wrctRVBZsJ #ITP 79% RR, 53.6% CR https://t.co/OJfMY0DMah
Rilzabrutinib Shows Safety and Efficacy in Chronic ITP
Safety and efficacy of rilzabrutinib vs placebo in adults with [persistent/chronic] immune thrombocytopenia: the phase 3 LUNA3 study [Jun 12, 2025] Kuter et al. @BloodPortfolio https://t.co/hhnzXg3bqu #NCT04562766 #ITP https://t.co/IkjLnu1kTd
Proteins.1 Launches with €4.7m to Make Protein Detection as Easy as PCR
Proteins.1, a Finnish spin‑off, announced a €4.7 million (~$5.1 million) pre‑seed round to commercialise a PCR‑style protein amplification platform. The enzyme‑free, solid‑state technology uses magnetic cycling and thin‑film transistors to read a single captured protein repeatedly, delivering up to 1,000× greater sensitivity...
Daratumumab Shows Safety and Efficacy in ITP
Safety and Efficacy of Daratumumab in Immune Thrombocytopenia (#ITP) [Aug 5, 2025] Tsykunova et al. @BloodPortfolio https://t.co/GHyJGG7YSe #NCT04703621 https://t.co/pBgjzTCGQl
Ianalumab Combined with Eltromb
Ianalumab [VAY736] plus Eltrombopag in Immune Thrombocytopenia [Dec 9, 2025] @CukerMd et al. for the VAYHIT2 Investigators @NEJM https://t.co/99urXh8agT #NCT05653219 #ITP https://t.co/qKKhwv4ofR
MitoCatch Boosts Mitochondrial Transplants, Offering New Hope for LHON Therapy
Ayupov and colleagues announced MitoCatch, a protein‑binder system that efficiently delivers therapeutic mitochondria to Leber’s hereditary optic neuropathy (LHON) neurons, markedly improving cellular health and survival. The breakthrough could reshape biohacking strategies aimed at cellular rejuvenation.
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...