Today's Pharma Pulse
Ona lands $86.6M Series B, one of Spain’s biggest biotech financings
Spanish biotech Ona announced a $86.6 million Series B round, ranking among the largest venture financings 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 funds will expand Ona’s AI‑driven drug discovery platform and accelerate its pre‑clinical pipeline.
BIO Patient Advocacy Coffee Chat: Act Now to Be Heard on CMS Drug Pricing
The Centers for Medicare & Medicaid Services entered the third year of its Medicare drug price negotiation program, selecting 15 drugs—including, for the first time, therapies covered under Medicare Part B. Patient advocacy groups have until March 1 to submit written comments and can register for roundtables and town halls in April. Advocates emphasized that thorough preparation, data‑driven storytelling, and coordinated patient voices are essential to influence the process. Early results show some negotiated drugs have actually increased out‑of‑pocket costs for patients, raising concerns about access and provider reimbursement.
Makary Misrepresents Unresolved Drug Applications, Conflates Therapies
(Hopefully) Final word today on the Makary $QURE (or was it $RGNX) thing: Bottom line: Makary shouldn't be talking publicly about unresolved drug applications. Uniqure doesn't even have an application submitted; and RegenxBio's CRL letter isn't posted to the FDA's...
Bristol Myers Says ADC Licensed From China Hits Mark in Aggressive Breast Cancer
Bristol Myers Squibb reported that its antibody‑drug conjugate iza‑bren, licensed from Chinese partner SystImmune, achieved statistically significant improvements in progression‑free and overall survival versus chemotherapy in a Phase III trial for advanced triple‑negative breast cancer. The study, conducted in mainland China...
Makary Slams FDA, Defends Rare Disease Drugs Amid Controversy
You've got to watch this Makary interview on CNBC. He attacks a drug, hits the Old FDA and circles all the wagons around Vinay Prasad, who "loves President Trump" and faces a fatwa (WSJ). Makary is getting major props for...
FDA Chief Backs Rare Disease Rejections, Lauds Vinay Prasad
FDA Commissioner Marty Makary defends rare disease drug rejections, Vinay Prasad "Vinay Prasad is a genius,” Makary said. “He’s on loan from UCSF. He’s published over 500 scientific peer-reviewed articles, and some of the drugs they are criticizing him for...
Most Detailed Spatial Atlas yet for Mapping the Pancreatic Tumor Microenvironment
An international team led by the University of Birmingham, University Hospitals Birmingham NHS Foundation Trust and Bristol Myers Squibb has produced the most detailed spatial atlas of pancreatic ductal adenocarcinoma (PDAC) to date. Using spatial transcriptomics and spatial molecular imaging...
![[Comment] Impact of Adding Hormone Therapy to Postoperative Radiotherapy in Prostate Cancer](https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/f1452ec53e5201a4460c9aaf141538d3.webp)
[Comment] Impact of Adding Hormone Therapy to Postoperative Radiotherapy in Prostate Cancer
Adding androgen deprivation therapy (ADT) to definitive radiotherapy improves overall survival for men with high‑risk prostate cancer, but its benefit when combined with postoperative radiotherapy (PORT) after prostatectomy is less clear. Randomised trials such as GETUG‑AFU 16, SPPORT, and the RADICALS‑HD...
Covalent Organic Frameworks Assembled Inside Tumor Cells Trigger Cancer Cell Death and Immune Activation
Researchers at the University of Macau have demonstrated the first in‑situ synthesis of covalent organic frameworks (COFs) inside lysosomes of cancer cells, using acidic pH‑driven imine condensation of TAPB and DMTP. The crystalline UMCOF1 particles rupture lysosomal membranes, liberating ferrous...
FDA’s One Trial Policy Not a Revolution but a Potentially Risky Evolution
The FDA announced it will default to a single pivotal trial for new drug applications, extending a practice long used in oncology and rare‑disease approvals. In 2024, 66% of new molecular entities were cleared based on one trial, signaling a...
ALS Advances Unite Patients and Pharma on Novel Targets, Biomarker Breakthroughs
The latest episode of BioSpace’s Denatured podcast spotlights a growing partnership between ALS patients and pharmaceutical developers, featuring insights from EverythingALS founder Indu Navar and VectorY Therapeutics CMO Dr. Olga Uspenskaya. The discussion highlights how patient‑driven collaborations are accelerating trial timelines...
Pharma Leadership Roundup: Sarepta Therapeutics CEO Announces Retirement, Rivus Pharmaceuticals Appoints New CEO
Sarepta Therapeutics announced that CEO Douglas Ingram will retire by the end of 2026, triggering a formal succession search. The move follows a turbulent 2025 in which the company’s flagship gene therapy Elevidys was linked to two patient deaths, prompting...
STAT+: FDA Rejection Is a Reality Check on Agency Rhetoric
The U.S. Food and Drug Administration rejected a rare‑disease cell therapy that had already secured approval in Europe, despite earlier internal support from the agency. The decision comes under the FDA’s new leadership and has sparked debate over whether the...
FDA Chief Warns Against Approving Risky, Ineffective Rare‑disease Drugs
$QURE down this morning on comments made by FDA Commissioner Marty Makary to CNBC's Becky Quick. In a discussion about rare-disease drug approvals and Vinay Prasad, Makary said this: "I think there has been a bit of an effort to...
Biotech VC Webinar Launches with Top Experts, Thousands Registered
We'll be gearing up today's webinar on biotech venture capital at the top of the hour. Great guests, great topics. If this is a central issue in your life, tune in. Thousands have signed up already. https://t.co/c16y04mFsD
Drug Dealin Ep. 9: Using AI to Find Novel Treatment Options
In Episode 9 of Drug Dealin, hosts Kenny and Shibu demonstrate how patients can use publicly available tools like ClinicalTrials.gov and AI language models to uncover novel treatment options, focusing on a hypothetical Parkinson’s disease case inspired by a TV...
DIY Gut Microbiome Tests Are Unreliable and Flawed
Direct-to-consumer gut microbiome assessments are unreliable, problematic: a review of 7 companies with significant methodologic issues https://t.co/atwmZUNjVE https://t.co/SqlMljuW0y
Orforglipron Outperforms Other Oral GLP‑1s in T2D Trial
Head-to-head trial of the oral GLP-1 drugs in people with T2D. Orforglipron was superior https://t.co/9B7RXIgdJY
Atara Climbs Amid Report of FDA Inconsistency Leading to Cell Therapy’s Rejection
Atara Biotherapeutics’ stock jumped 20% after STAT reported that internal FDA inconsistencies may have driven the rejection of its EBV‑positive PTLD cell therapy, Ebvallo. The FDA denied the U.S. application citing insufficient evidence of effectiveness, yet former reviewers said data...
Progress Toward Off‑the‑Shelf Vaccines for Hereditary Cancer
We're making headway for off the shelf preventive cancer vaccines in people with hereditary forms of cancer, e.g. Lynch syndrome https://t.co/F3LqKS46BQ
Boehringer Bets up to $500m on Sitryx Immunology Programme
Boehringer Ingelheim announced a potential $500 million investment in UK biotech Sitryx to obtain global rights to an undisclosed small‑molecule immunology programme. The partnership covers upfront fees, milestones and royalties, giving Boehringer full responsibility for research, clinical development and commercialization. Sitryx’s...
Post-Conference Highlights | 2026 ViVE | Feb 22–25 | Los Angeles, CA
ViVE 2026 in Los Angeles marked a decisive shift from digital ambition to operational accountability across the health ecosystem. Leaders emphasized that AI adoption now hinges on governance, measurable ROI, and clinician trust, while interoperability is being treated as core...
The Hidden DNA Shape That Could Break Cancer
Researchers have captured the fleeting i‑DNA structure inside living cells, showing it acts as a molecular switch for genes that drive cancer. The study demonstrates that i‑DNA formation and resolution are tightly timed during DNA replication, influencing gene expression and...
Continuous Cardiac Monitoring: Redefining the “End” Of a Clinical Study?
Continuous cardiac monitoring via wearable ECG devices is reshaping clinical trials and post‑market care. Recent studies show that 14‑day monitoring after cardiac surgery uncovered atrial fibrillation in 24% of patients, many of which were missed until three‑month follow‑ups. In epilepsy...
Pfizer Advances Beam’s Gene Editor After Pulling Hemophilia Gene Therapy
Pfizer has signed a global gene‑editing partnership with Beam Therapeutics, reviving its genetics ambitions after withdrawing the hemophilia B therapy Beqvez a year earlier. The agreement includes a $300 million upfront payment and up to $1.05 billion in milestones, granting Pfizer exclusive worldwide...
RVMD Switches PRMT5 Partner From T
$RVMD was collaborating with $TNGX in PRMT5, but now it's working with $BMY (remember that ex MRTX project?). Via @ByMadeleineA @ApexOnco -> https://t.co/mGZjKOiB7p (And $AMGN still dose optimising.)
Cara Medical Reports 510(k) Clearance of CARA System for Noninvasive Cardiac Conduction System Visualization
Cara Medical announced that the U.S. FDA granted 510(k) clearance for its CARA System, a non‑invasive solution that creates patient‑specific 3‑D visualizations of the cardiac conduction system using CTA data. The platform pairs the CARA Metis™ Simulator for pre‑procedural mapping...
How Takeda Is Harnessing RWE, Patient Insights and Human-Focused AI to Advance Global Access
Takeda is leveraging real‑world evidence (RWE) and human‑focused AI to reshape clinical trial design, patient access, and global market strategies. Chief Medical Officer Awny Farajallah highlights three pillars: robust evidence generation, capability building, and early patient access. RWE is used...
![[Comment] Oral GLP-1 Receptor Agonists: Competition for Efficacy and Tolerability](https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/ce10e510d7294b419a4a07dfad918da4.webp)
[Comment] Oral GLP-1 Receptor Agonists: Competition for Efficacy and Tolerability
Oral GLP-1 receptor agonists have moved from injectable peptides to a tablet form, highlighted by oral semaglutide combined with the absorption enhancer SNAC. The formulation requires patients to fast overnight, take the dose with a small amount of water, and...
GSK Reports the NMPA’s NDA Acceptance with Priority Review of Linerixibat for Cholestatic Pruritus
GlaxoSmithKline announced that China’s National Medical Products Administration has accepted the New Drug Application for linerixibat and placed it under priority review for treating cholestatic pruritus in primary biliary cholangitis (PBC). The filing is backed by the Phase III GLISTEN trial,...
What Does the Rollout of Trump Rx Reveal?
Buzz Health President Joseph Kleiman says TrumpRx is advancing drug‑price transparency by giving consumers early visibility into prescription costs. He notes that real‑time pricing can help patients compare options but also warns that prices are dynamic and may change before...
Leading in the Psychedelic Space: Q&A with Tarek Rabah
Otsuka America became the first major pharmaceutical company to back psychedelic‑inspired medicines in 2020 through a partnership with COMPASS Pathways, and deepened that commitment by acquiring Mindset in 2023. The company is applying its long‑standing CNS expertise to develop serotonergic...
Merck Animal Health’s Numelvi Secures the FDA Approval for Control of Pruritus Associated with Allergic Dermatitis in Dogs
Merck Animal Health announced that the U.S. FDA has approved Numelvi (atinvicitinib) for controlling pruritus linked to allergic dermatitis in dogs six months and older, with commercial availability slated for spring 2026. Numelvi is a second‑generation JAK inhibitor that demonstrates...
STAT+: Rare Disease Advocates Fume over FDA’s Mixed Signals
The FDA has rejected Regenxbio’s gene therapy for mucopolysaccharidosis type 2, citing a need for additional data that could postpone approval by years. This decision adds to at least four other recent rejections of cell or gene therapies targeting deadly rare...
Mahzi Doses First Patient in MZ-1866 Trial for Pitt Hopkins Syndrome
Mahzi Therapeutics has administered the first dose of its investigational gene therapy MZ-1866 in the Phase I/II UNITE study for Pitt‑Hopkins syndrome. The open‑label, multicentre trial will enroll roughly 12 genetically confirmed patients across sites in Spain, Israel and the...
Women’s HealthX Launches in Boston This December to Transform Women’s Health Through Data and Science
Women’s HealthX (WoHX) will convene 750 pharma, biotech, hospital, insurer, startup and government leaders in Boston on December 3‑4, 2026. The two‑day summit aims to close the sex‑difference data gap by showcasing evidence‑based technologies, AI tools and digital therapeutics across the...
With TrumpRx Is America Reforming Its Pharmaceutical Market, or Simply Rebranding Prices?
The Trump administration launched TrumpRx, a website aggregating manufacturer discounts on 43 brand‑name drugs and touting presidential credit for lower patient costs. Early analysis shows that 20 of the listed products already have cheaper generic versions, and the portal deliberately...
Argenx Reports Topline P-III (ADAPT OCULUS) Trial Data on Vyvgart in Ocular Myasthenia Gravis (MG)
argenx announced topline Phase III ADAPT OCULUS data for Vyvgart (efgartigimod alfa + hyaluronidase‑qvfc) in adults with ocular myasthenia gravis. The trial met its primary endpoint, delivering a 4.04‑point improvement in Myasthenia Impairment Index PRO ocular scores versus 1.99 points for placebo at...
CDSCO Eases Drug Testing Approvals, New Fast-Track System From June 1, 2026
The Central Drugs Standard Control Organization (CDSCO) will issue No Objection Certificates (NOC) for drug‑sample testing immediately upon receipt of applications, eliminating the prior detailed specification review. Companies can begin laboratory testing at designated Indian Pharmacopoeia Commission and other central...
Surprise! Fifth Circuit Rejects DEA’s Longstanding Interpretation of a Pharmacist’s “Corresponding Responsibility” And “Usual Course of Professional Practice” Regulations
The Fifth Circuit rejected the DEA’s long‑standing reading of the pharmacist’s “corresponding responsibility” and “usual course of professional practice” regulations, ruling that liability requires proof a prescription was invalid when issued and that the pharmacist actually knew it was invalid....
Pacira Biosciences Inc (PCRX) Q4 2025 Earnings Call Transcript
Pacira BioSciences reported a record $726 million in total 2025 revenue and an all‑time high non‑GAAP gross margin of 80%, driven by growth in its flagship product EXPAREL. EXPAREL sales rose 7% year‑over‑year to $155.8 million, though the increase was partially offset...
OPKO Health Inc (OPK) Q4 2025 Earnings Call Transcript
OPKO Health reported a $369 million cash position for Q4 2025, driven by asset sales, BARDA funding and partnership payments. The company repurchased 34.6 million shares for $47 million and allocated $109 million to stock and convertible note buybacks. Diagnostics revenue dropped to $71.1 million, but...
OrthoPediatrics Corp (KIDS) Q4 2025 Earnings Call Transcript
PolyPid announced successful completion of the Phase III SHIELD II trial, with D‑PLEX100 achieving its primary and key secondary endpoints and demonstrating a meaningful reduction in surgical site infections. The FDA provided written support for a rolling NDA review, and the company...
MannKind Corp (MNKD) Q4 2025 Earnings Call Transcript
MannKind reported a record Q4 2024 with $77 million in revenue and a full‑year total of $286 million, a 43% increase year‑over‑year. Afrezza sales grew 17% to $64 million, and the company announced a pediatric filing for the inhaled insulin in the first...
Rhythm Pharmaceuticals Inc (RYTM) Q4 2025 Earnings Call Transcript
Rhythm Pharmaceuticals reported a strong Q4 2025, with product revenue rising to $57.3 million—a 12% sequential increase and 37% year‑over‑year growth. Full‑year revenue jumped 50% to $194.8 million, driven by expanding U.S. sales and broader international access for IMCIVREE. The company highlighted...
Endometrium-Targeted mRNA-Lipid Nanoparticles for Treating Reproductive Conditions
Researchers have engineered ligand‑conjugated mRNA‑lipid nanoparticles that home specifically to the endometrium, delivering therapeutic mRNA directly to uterine tissue. In a murine model of endometrial injury, the targeted formulation restored embryo implantation rates to near‑normal levels. Safety profiling showed reduced...
Definium Therapeutics Inc (DFTX) Q4 2025 Earnings Call Transcript
Definium Therapeutics reported that its lead psychedelic candidate DT120 ODT has fully enrolled the EMERGE Phase III trial for major depressive disorder, with top‑line results expected in late Q2 2026. The VOYAGE Phase III study for generalized anxiety disorder is...
BioCryst Pharmaceuticals Inc (BCRX) Q4 2025 Earnings Call Transcript
BioCryst Pharmaceuticals reported a 37% year‑over‑year rise in Orladeyo revenue to $159.1 million in Q3 and lifted its 2025 sales guidance to $590‑$600 million. The company completed the divestiture of its European business, repaid a $200 million term loan and entered Q3 with...
Puma Biotechnology Inc (PBYI) Q4 2025 Earnings Call Transcript
Puma Biotechnology reported total 2025 revenue of $75.5 million, driven by $59.9 million net product revenue from its HER2‑positive breast cancer drug NERLYNX and a surge in royalty income to $15.6 million after a large shipment to a Chinese partner. Ex‑U.S. inventory rose...
Kymera Therapeutics Inc (KYMR) Q4 2025 Earnings Call Transcript
Kymera Therapeutics reported Q4 2025 revenue of $2.8 million, entirely from its Gilead collaboration, while maintaining a cash balance of $978.7 million that extends runway into the second half of 2028. Adjusted R&D and G&A expenses fell 7% and 3% quarter‑over‑quarter, respectively....
Intellia Therapeutics Inc (NTLA) Q4 2025 Earnings Call Transcript
Intellia Therapeutics announced that the FDA has placed a clinical hold on its nex‑z MAGNITUDE and MAGNITUDE‑2 Phase III trials after a patient death and liver enzyme abnormalities. The company is implementing intensified safety monitoring and data reviews while awaiting regulatory...