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.
FDA Approves Otarmeni, First Gene Therapy for Genetic Hearing Loss
The U.S. Food and Drug Administration authorized Otarmeni, the first dual‑AAV gene therapy for OTOF‑associated severe-to-profound hearing loss, after a 61‑day accelerated review. The approval opens a new therapeutic pathway for patients whose condition has previously been managed only with devices such as cochlear implants.
Biogen Inc (BIIB) Q1 2026 Earnings Call Transcript
Biogen reported $2.8 billion revenue for Q1 2026, a 7% increase driven by four new launch products and resilient U.S. multiple‑sclerosis sales. Non‑GAAP diluted EPS rose 4% to $5.25, and the company lifted its 2025 EPS guidance to $15.50‑$16 while keeping revenue...
Agios Pharmaceuticals Inc (AGIO) Q1 2026 Earnings Call Transcript
Agios Pharmaceuticals reported a 45% year‑over‑year jump in PYRUKYND net revenue to $12.5 million, driven by stronger PKD sales, an extra ordering week, and higher specialty‑pharmacy processing. The company ends the quarter with roughly $1.3 billion in cash, supporting both commercial rollout...
Adeno-Associated Virus-Based Approaches for Mitochondrial Diseases: Advances and Challenges
Adeno‑associated virus (AAV) vectors are emerging as a versatile platform for treating mitochondrial diseases, especially those caused by nuclear‑encoded gene defects. Pre‑clinical studies have shown that AAV‑mediated delivery of nuclear genes can restore oxidative phosphorylation, extend survival, and improve organ...
Safety and Efficacy of Intratumoural Anti-CTLA4 with Intravenous Anti-PD1
The phase 1b NIVIPIT trial compared intratumoural (IT) ipilimumab at 0.3 mg kg⁻¹ plus intravenous nivolumab with the standard intravenous (IV) ipilimumab‑nivolumab regimen in untreated advanced melanoma. The IT arm achieved a markedly lower rate of grade 3‑4 treatment‑related adverse events (24 % vs 67 %...
Ionis Pharmaceuticals Inc (IONS) Q1 2026 Earnings Call Transcript
Ionis Pharmaceuticals reported record 2025 revenue of $944 million, a 34% year‑over‑year increase driven by strong sales of TRYNGOLZA, the launch of DAWNZERA, and robust R&D collaboration fees. Phase III data for olezarsen demonstrated up to a 72% reduction in triglycerides and...
Urban Edge Properties (UE) Q1 2026 Earnings Call Transcript
Urban Edge Properties reported Q1 2026 net revenue of $12.5 million, a 45% year‑over‑year increase driven by stronger PKD sales, an extra ordering week, and expanded specialty pharmacy distribution. Patient enrollment rose to 248 completed forms with 142 patients now on...
Regeneron Pharmaceuticals Inc (REGN) Q1 2026 Earnings Call Transcript
Regeneron reported Q1 2025 revenue of $3 billion, with Dupixent and Libtayo delivering double‑digit growth while U.S. EYLEA sales fell 39% to $736 million amid co‑pay assistance gaps and rising Avastin use. Gross margin guidance was revised to 86‑87% after larger‑than‑expected inventory...
Anika Therapeutics Inc (ANIK) Q1 2026 Earnings Call Transcript
Anika Therapeutics posted total revenue of $28.2 million for the quarter, an 8% decline driven by manufacturing yield issues and a $900,000 shortfall in international osteoarthritis‑pain sales. Gross margin fell to 51%—down 16 percentage points—after a one‑time $3 million non‑cash charge, though...
Boosting T Helper Cells Could Curb Lifelong Viral Infections
The immune system actively combats lifelong viral infections acquired at birth, but its response is limited by a reduced pool of T helper cells; enhancing these cells may offer new therapeutic strategies. immunology
Psychedelics for Mental Illness: Dr. Joseph Varon on Trump’s Executive Order
In this episode, Fox News discusses President Trump's new executive order aimed at loosening restrictions and expanding access to psychedelic therapies for severe mental health conditions such as depression, PTSD, and addiction, especially among veterans. Dr. Joseph Varon, president and...
NVO’s 4.4% Yield Boosted by Oral Semaglutide Surge
$NVO pays a decent 4.4% dividend and oral semaglutide is surging: "First-time anti-obesity medication (AOM) prescribing increased 21.7% from December 2025 to March 2026, with first-time AOM semaglutide prescribing increasing by more than 50%. AOM semaglutide also showed its largest quarter-over-quarter percentage...
Peptides / Bioregulators
The AGI House in San Francisco hosted the inaugural California Peptide Club, drawing over 100 invite‑only attendees to discuss self‑optimization peptides. Organiser Julius Ritter highlighted the fragmented information landscape and showcased stacks ranging from growth‑hormone releasers to cognitive enhancers. Days later, HHS...
FOXO4-DRI Is Fascinating, but Was Never Intended for Human Use, What Are the Takeaways?
FOXO4‑DRI is an experimental senolytic peptide that selectively eliminates senescent cells by disrupting the FOXO4‑p53 interaction, prompting p53‑mediated apoptosis. Pre‑clinical studies across vascular, reproductive, musculoskeletal and renal models report improved endothelial function, restored testosterone production, chondrocyte rejuvenation, and reduced frailty....
Peptides / Bioregulators
A new study examined 6,441 gray‑market peptide samples covering 14 compounds, measuring purity, dose accuracy, and endotoxin levels. Between 41.6% and 71.1% of the products failed basic pharmaceutical standards, and 2.4% contained no active peptide at all. Endotoxin contamination appeared...
The BioPharm Brief: Expanding Access and Redefining Cancer Treatment
Henlius and Organon received European Commission approval for a pertuzumab biosimilar, expanding HER2‑targeted therapy access across the EU. Pfizer announced Phase 3 data showing its bispecific antibody Elrexfio improves progression‑free survival in relapsed or refractory multiple myeloma. Genexine disclosed pre‑clinical activity...
Living Vessel ATEV Offers New Hope for Dialysis Access
Humacyte’s ATEV could change the game for dialysis patients who run out of fistula options. It’s a living vessel that resists infection and needs fewer repairs. Major unmet need, big potential. Biotech
Nicotinamide Boosts NK Cells, Induces NHL Remissions
Nicotinamide enhances natural killer cell function and yields remissions in patients with non-Hodgkin lymphoma https://t.co/VrC2ertsVd
Semiglutide Regenerates Cartilage Loss Through Weight Loss Independent Metabolic Restoration Mechanism
Semaglutide, a GLP‑1 receptor agonist originally developed for diabetes, is showing promise as a cartilage‑regenerating therapy. Preclinical pair‑fed mouse studies demonstrate cartilage protection independent of weight loss, while a 24‑week pilot in humans reported a 17% increase in cartilage thickness....
Scientists, Not Regulators, Block Revolutionary Medicines
Finally, a show exposing the main obstacle to new medicines. Hint, it’s not the FDA: “Throughout history, society hasn’t always appreciated revolutionary scientific findings. For hundreds of years & still today, some of the biggest attackers are fellow scientists.” 🧑🔬🔥👩🔬 https://t.co/h2BpILNV4D
Alamar Biosciences IPO Raises $219.9M for Blood-Based Biomarker Tools
Alamar Biosciences closed an upsized IPO, raising $219.9 million by pricing 12.9 million shares at $17 each, and began trading on Nasdaq under the ticker ALMR. The California‑based firm offers the NULISA platform, which detects low‑level protein biomarkers in blood, and its...
STAT+: AIDS Group Sues Trump Administration over Undisclosed Agreement with Gilead
An AIDS activist group has sued the Trump administration for failing to disclose a research and development agreement that underpinned a 2025 settlement with Gilead Sciences over patents on HIV‑prevention drugs Truvada and Descovy. The settlement ended a lawsuit dating...
Hospitals, Insurers Clash on 340B Rebate Costs
The Health Resources and Services Administration (HRSA) issued a request for information on reviving a 340B drug‑rebate model after a court‑blocked rollout. Hospital groups, led by the American Hospital Association, warn the model could impose over $1 billion in annual costs,...
FDA Accepts NDA for Zipalertinib, Targeting EGFR Exon‑20 Insertion NSCLC
Taiho Oncology, Taiho Pharmaceutical and Cullinan Therapeutics said the FDA has accepted a New Drug Application for zipalertinib, an oral EGFR inhibitor for patients with EGFR exon‑20 insertion NSCLC who have progressed after platinum chemotherapy. The filing is backed by...
Eli Lilly Signs $2.25 B AI‑driven DNA‑editing Pact with Profluent Bio
Eli Lilly has entered a research agreement with AI‑focused Profluent Bio that could deliver up to $2.25 billion in development and commercial milestones. The deal targets kilobase‑scale DNA editing, a capability the partners say could unlock therapies for complex genetic disorders.
A Popular Senolytic Treatment Causes Brain Damage in Mice
A recent PNAS study shows that the widely used senolytic cocktail dasatinib plus quercetin (D+Q) impairs myelination in the mouse corpus callosum. The treatment altered oligodendrocyte morphology within minutes, reduced myelin thickness, and triggered endoplasmic reticulum stress, without killing the...
New Blood-Based Method Identifies Testicular Cancer Missed by Standard Tests
Mayo Clinic scientists unveiled a blood‑based assay, GCT‑iSIGN, that detects germ cell tumors with 93% sensitivity and 99% specificity, even when conventional tumor markers are negative. In a cohort of 427 samples, the test caught 23 of 24 cases missed...
Multiple Myeloma Sequencing Evolves With CAR T, MRD Insights: Sylvester Homsy, MD
At an Institute for Value‑Based Medicine event in Charlotte, Sylvester Homsy, MD highlighted how CAR‑T cell therapy and bispecific antibodies are reshaping multiple myeloma treatment sequencing. The emergence of B‑cell maturation antigen‑targeted agents is prompting clinicians to consider these high‑efficacy...
FDA Partners with AZN, AMGN to Accelerate AI-Driven Trials
FDA launches effort with $AZN $AMGN to speed up clinical trials, using AI https://t.co/k4LjxnDLmq via @LizzyLaw_
Cannabis Rescheduling: What Does It Mean for Your Intellectual Property
The U.S. Department of Justice has reclassified FDA‑approved and state‑licensed medical marijuana products to Schedule III under the Controlled Substances Act, following President Trump’s 2025 executive order. This shift removes the “unlawful use” barrier that previously blocked trademark protection for cannabis‑related...
Supreme Court Case Could Broaden Vascepa Generic Labeling
Vascepa "skinny label" generic case at the Supreme Court. The generic label is only for hypertriglyceridemia, while Vascepa label is much broader. But gets promoted as a generic for Vascepa. Impact might be substantial. https://t.co/xtGWtscXkc
US Pricing Reform Reshapes Drug Launch Strategies: Dee Chaudhary
U.S. drug‑pricing reforms, especially the Most Favored Nation (MFN) rule, are forcing manufacturers to align American prices with the lowest prices offered abroad. As a result, firms are pulling back from launches in France, Germany and the Nordic region to...
Scientists Uncover a Hidden Mechanism Cancer Cells Use to Rewrite Genetic Messages, Revealing a Promising New Target for Treatment
A team of molecular biologists has uncovered a previously unknown RNA‑binding protein that rewrites messenger‑RNA messages in cancer cells, effectively reprogramming gene expression. The discovery explains how tumors can rapidly adapt to hostile environments and develop resistance to standard chemotherapy....
WHO Designates Network of Regional Biomanufacturing Training Centers
The World Health Organization has officially designated a network of regional biomanufacturing training centers spanning all six WHO regions, marking a major step in building a skilled global workforce for vaccines, biotherapeutics and other biologics. The centers—located in Senegal, South...
With Cell and Gene Therapies Proliferation, Specialty Pharmacy Faces New Pricing, Distribution Pressures
Cell and gene therapies are moving from ultra‑rare indications to broader patient populations, prompting a fundamental redesign of specialty‑pharmacy distribution and pricing models. At the 2026 Asembia meeting, experts warned that current flash‑title and buy‑and‑bill channels may not scale to...
Pharmaceutical Executive Daily: FDA Approves Breztri
Sun Pharmaceutical Industries agreed to buy Organon for $11.75 billion, propelling the combined entity into the top‑25 global pharma ranks. IMG Pharmaceutical announced the acquisition of Japan’s Matsumoto Pharmaceutical, adding a portfolio with more than 50 OTC approvals. The FDA granted...
How Price Transparency Could Fix the Abandoned Rx Issue: Laura Jensen
GoodRx’s chief commercial officer Laura Jensen argued that greater drug‑price transparency could dramatically cut the roughly one billion annual abandoned prescriptions in the United States. Speaking at Asembia’s AXS26 conference, she highlighted how transparent, consumer‑facing platforms can deliver easier‑to‑access discounts for...
New AI Models Quickly Find Compounds that Target Lyme Bacteria
Tufts University researchers have leveraged AI and machine‑learning to rapidly pinpoint narrow‑spectrum antibiotics that kill the Lyme disease bacterium Borrelia burgdorferi. Screening 60,000 existing compounds yielded several hundred hits, and generative models now explore an estimated 10^60 drug‑like molecules to...
Advanced Gene Editing ‘Promising’ for Sickle Cell Disease
Two recent New England Journal of Medicine studies demonstrate that CRISPR‑Cas12a (reni‑cel) and base‑editing (risto‑cel) autologous stem‑cell therapies can dramatically raise fetal hemoglobin and normalize total hemoglobin in sickle cell patients. The RUBY trial reported a rise from 2.5% to...
Asembia AXS26 Summit: The Future of High-Cost Specialty Drugs
At the Asembia AXS26 Summit, Clarivate’s Dee Chaudhary warned that U.S. drug‑pricing policy is moving from theory to real‑time execution, reshaping payer behavior and market volatility. The Inflation Reduction Act’s Medicare price negotiations, the new One Big, Beautiful Bill Act, and renewed Most Favored...
FDA Debuts Plan to Collect Real-Time Clinical Trial Data
The U.S. Food and Drug Administration announced a pilot program that will collect clinical‑trial data in real time, allowing scientific reviewers to see information as it is generated. The initiative, launched on Tuesday, includes participation from major drugmakers AstraZeneca and...
GLP-1 Receptor Agonist Add-On at Immune Checkpoint Inhibitor Initiation and Fewer Wasting-Related Diagnoses and Acute-Care Episodes in People with Cancer...
A target‑trial emulation of US TriNetX data examined cancer patients with obesity who started immune checkpoint inhibitors (ICIs) and received a GLP‑1 receptor agonist (GLP‑1 RA) at initiation. After 1:1 propensity matching, 988 patients per arm were followed for up to...
Pfizer Secures Patent Extension for Leading Rare‑Disease Drug
Pfizer deals extend patent life for a top-selling rare disease drug https://t.co/uXXbtLaIOL $PFE $BBIO $ALNY $IONS
Pfizer Delays Patent Cliff for Blockbuster Vyndamax
Pfizer announced a two‑year extension of market exclusivity for Vyndamax (tafamidis), pushing the expected generic launch to 2026. The extension stems from a new formulation patent and a pediatric study that grant additional protection. Vyndamax, approved for transthyretin amyloid cardiomyopathy,...
Dual-Agonist Survodutide Shows Significant Weight Loss in Phase III Obesity Trial
Boehringer Ingelheim reported that its dual glucagon/GLP‑1 agonist survodutide produced up to 16.6% average weight loss after 76 weeks in the Phase III SYNCHRONIZE‑1 trial. The study also showed that 85.1% of treated participants achieved at least a 5% reduction, with...
Psychedelics May Reverse Epigenetic Roots of Addiction
Epigenetic Mechanisms of Psychedelics in Addiction: Emerging Evidence and Therapeutic Potential these compounds may directly counteract the epigenetic and transcriptional imprints that sustain compulsive substance use https://t.co/GJyHoLPL83
Hikma and Amarin's Generic Drug Case Heads to Supreme Court Arguments
The U.S. Supreme Court will hear arguments this week in the dispute between Hikma Pharmaceuticals and Amarin over a prescription‑strength fish‑oil pill, the first generic challenge to Amarin’s Vascepa. The case centers on whether the FDA’s bioequivalence standards and the...
BIOTECanada Responds to Health Canada’s Gazette on a Draft Order Providing for Reliance on International Regulatory Authorities
Health Canada’s Gazette released a draft Order that would allow Canadian regulators to rely on approvals from trusted foreign agencies, a key component of the Red Tape Review initiative. BIOTECanada praised the proposal, calling it a meaningful step toward faster...
Asembia AXS26: Jessica Lovett on the RWE That Matters Most
Jessica Lovett, VP of Commercial Strategy and Innovation at Innomar Strategies, discussed the most valuable real‑world evidence (RWE) for specialty medicines at Asembia AXS26. She highlighted two primary data sources: patient support programs and patient registries, both of which can...
Profluent and Lilly: The Next Gene Editor Will Be Designed by AI
Profluent, an Air Street Capital portfolio company, announced a multi‑program partnership with Eli Lilly to create AI‑designed recombinases for kilobase‑scale gene editing. The deal includes an upfront cash payment, committed R&D funding, and up to $2.25 billion in development and commercial milestones...