Ketamine Therapy for Chronic Pain and Substance Misuse
Recent peer‑reviewed study of 20 adults with chronic pain and substance misuse found ketamine therapy improved pain, mood, and dependence scores. The integrated treatment was delivered within a coordinated pain program, highlighting benefits of interdisciplinary care. Findings suggest ketamine can break the pain‑substance use cycle and support value‑based outcomes. Larger trials are needed to confirm efficacy.
Renal Cell Carcinoma Strategic Intelligence Report
The latest ASCO GU strategic intelligence report spotlights renal cell carcinoma (RCC) as a field entering a transformative phase. Analysts highlight emerging biomarkers, novel HIF‑2α inhibitors, and evolving immunotherapy combinations as potential high‑impact developments. While these advances promise to reshape...
Pharma Pulse: SteinCares Partners to Expand Biosimilar Access in Latin America and Eli Lilly Issues Warning Over Compounded Tirzepatide Safety
SteinCares and Shilpa Biologicals have signed a licensing deal to commercialize biosimilars across Latin America, with SteinCares handling regional registration and distribution while Shilpa oversees product development and long‑term manufacturing. The partnership targets broader patient access to cost‑effective biologics in...
Kvantify Advances Hybrid Quantum-Classical Software for Drug Discovery
Kvantify closed a €7 million second‑stage funding round to accelerate its hybrid quantum‑classical software stack for drug discovery, building on a $10.8 million seed round and earlier EU Innovation Council support. The company’s Qrunch platform integrates conventional chemistry methods with proprietary FAST‑VQE...
Functionalized Nanoparticles Could Open the Door to Swallowable Insulin Pills
Researchers have grafted the permeation enhancer 1‑phenylpiperazine onto safe silica nanoparticles, creating a hybrid that boosts intestinal insulin absorption while eliminating toxicity. In obese, insulin‑resistant mice, oral insulin delivered with these functionalized particles lowered blood glucose for 8‑10 hours, outperforming...
Closing the 9-Year Gap: A New Biomarker Targets the Diagnostic Delay in Axial Spondyloarthritis
Axial spondyloarthritis affects about 1.4% of adults but patients wait an average of nine years for diagnosis in North America, far longer than rheumatoid arthritis. Augurex has introduced SPINEstat, an anti‑14‑3‑η multiplex blood test that can distinguish inflammatory back pain...
Pharmaceutical Executive Daily: Senate Democrats Seeking Information on MFN Pharma Deals
Senate Democrats, led by Sen. Ron Wyden, have sent letters to eleven pharmaceutical companies demanding details on their most‑favored‑nation (MFN) agreements and whether those prices generate real savings for Medicaid, with a March 23 response deadline. The inquiry follows earlier requests...
‘Magic Mushroom’ Derivative Could Heal without Hallucinations, Sparking Hope for New Therapies
Scientists at the University of Padova synthesized fluorinated psilocin derivatives, identifying compound 4e as a lead that retains serotonergic activity while markedly reducing hallucinogenic effects in mice. In vitro assays showed 4e is a selective partial agonist at 5‑HT2A and...
Navigating Complexity in Emerging Biotech: Innovations, Integrations, and Initial Hurdles
Industry experts highlight three intersecting forces reshaping emerging biotech: the persistent funding and regulatory hurdles faced by early‑stage startups, the rapid migration of AI from a supportive tool to an operational backbone, and breakthrough computational methods—including quantum chemistry—that are redefining...
Baseimmune Announces Strategic Expansion Into Fibrosis with Lead Program Targeting Idiopathic Pulmonary Fibrosis (IPF)
Baseimmune announced a new fibrosis‑focused pipeline leveraging its computational protein design platform to create multi‑pathway immunotherapies, starting with idiopathic pulmonary fibrosis (IPF). The company aims to deliver proof‑of‑concept efficacy data for its lead IPF program in 2026‑2027, addressing the limitations...
How Does Cocaine Rewire the Brain?
Researchers at Michigan State University used mouse models and CRISPR technology to map how cocaine rewires the ventral hippocampus‑nucleus accumbens (vHPC‑NAc) circuit. They discovered that the transcription factor ΔFosB acts as a molecular switch, accumulating with repeated cocaine exposure and...
Generic GLP-1s Are Coming, but Americans Don’t Want to Wait
GLP‑1 drugs such as semaglutide have surged from diabetes treatment to a mass‑market weight‑loss solution, with roughly 12.4% of Americans now using them. Global sales are projected to climb from $50‑60 billion today to over $135 billion within a decade, driven largely...
Pharma Pulse: FDA Launches AEMS and the Rise of Direct-to-Employer Drug Purchasing
The FDA unveiled the Adverse Event Monitoring System (AEMS), a unified platform that shifts drug safety surveillance from quarterly updates to real‑time reporting and is expected to save roughly $120 million over five years. Simultaneously, a direct‑to‑employer drug‑purchasing model is gaining...
Rethinking Endocrine Therapy in ER-Positive Breast Cancer
Dr. Steven Quay, CEO of Atossa Therapeutics, highlighted a new focus on tolerability and prevention in estrogen‑receptor‑positive breast cancer, where five‑year survival now exceeds 90%. Atossa is developing a next‑generation SERM that aims to reduce side‑effects while maintaining efficacy and...
Injectable Mini-Livers as an Alternative to Liver Regeneration
Researchers have introduced INSITE, an injectable platform that combines primary human hepatocytes with hydrogel microspheres to form self‑assembling, vascularizable tissue ensembles in situ. Using ultrasound guidance, the scaffold is delivered to an ectopic site where it integrates with host vasculature...
GSK Backs £11m Centre to Build Digital Twins of Organs for Faster Drug Discovery
GSK has pledged £11 million to launch the Modelling‑Informed Medicine Centre (MiMeC), a joint venture with Imperial College London and the University of Oxford. The centre will develop digital twins—computer‑based replicas of lungs, liver and kidneys—to run in‑silico experiments and speed...
Moving CAR-T Beyond Oncology
Researchers are expanding CAR‑T cell therapy beyond cancer to treat autoimmune diseases such as lupus and rheumatoid arthritis. Sail Biomedicine’s chief products and operations officer highlighted on the Pharmaceutical Executive podcast that the company has pivoted to RNA‑based CAR‑T platforms,...
The Trump Drug Deal Threatens the Very Purpose of the NHS
The UK government has negotiated a UK‑US pharmaceutical trade deal that raises NICE’s cost‑effectiveness threshold from £20‑30k to £25‑35k per QALY. The higher threshold will deem more expensive drugs cost‑effective, increasing NHS drug spend while reducing rebate rates from 22.9%...
FDA Signals Potential Updates to SUPAC Guidances—Comments Due June 1, 2026
The FDA has opened a public docket to solicit comments on its long‑standing Scale‑Up and Post‑Approval Changes (SUPAC) guidances for immediate‑release solid oral, non‑sterile semisolid, modified‑release solid oral dosage forms and the manufacturing equipment addendum. The agency seeks feedback on...
Pharmaceutical Executive Daily: FDA's New Guidance for Biosimilar Development
The FDA issued its fourth revision of draft biosimilar development guidance, allowing scientifically justified streamlining of pharmacokinetic (PK) studies and estimating up to a 50% cost reduction—about $20 million per program. The new guidance also expands the use of clinical data...
A Better View of How Cells Take Up Mitochondria to Restore Function
Researchers have demonstrated that mesenchymal stromal cells (MSCs) can actively internalize isolated, functional mitochondria through endocytic pathways. The study shows that the internalized organelles retain structural integrity and boost cellular proliferation, stress tolerance, and oxygen consumption. Chemical inhibition of endocytosis...
Veradermics $294.8M IPO to Fund Phase III Hair Loss Trials
Veradermics closed a $294.8 million IPO, pricing 17 dollars per share and debuting on the NYSE under the ticker MANE. The capital will fund its late‑stage clinical program for VDPHL01, an extended‑release oral minoxidil aimed at androgenetic alopecia. The company has completed...
From Insights to Impact: Rare Disease Therapies with UCB’s Dr. Kim Moran — Episode 246
UCB’s Senior Vice President Dr. Kim Moran discusses the company’s rare disease pipeline, including therapies for mitochondrial TK2 deficiency, on Xtalks Life Science Podcast episode 246. Moran, who pioneered UCB’s Insights‑to‑Impact function, explains how patient‑derived data informs strategy, digital transformation,...
Seven Ways to Skin KRAS: Emerging Approaches to Watch Out For
The article surveys seven early‑stage programmes tackling KRAS, each proposing a distinct therapeutic angle. While many firms still chase the classic model of deeper, longer, or more selective pathway blockade, these initiatives span elegant biochemical tricks to counterintuitive concepts that...
Direct-to-Employer Drug Purchasing Emerges as Companies Navigate High Prescription Costs
Jay Bregman, CEO of Andel, unveiled a direct‑to‑employer medication platform that lets companies purchase drugs straight from manufacturers, starting with Eli Lilly’s Zepbound KwikPen. The model bypasses PBMs and insurers, aiming to lower out‑of‑pocket costs and curb rising spend on high‑priced...
Veristat Expands Regulatory and Clinical Services to Chinese Drug and Device Companies Seeking Efficient Way to Enter European Markets
Veristat announced an expansion of its regulatory and clinical‑trial services aimed at Chinese pharmaceutical and medical‑device companies seeking faster entry into European markets, as well as the United Kingdom, Switzerland, the United States, Canada and Australia. The CRO highlighted recent...
Parabilis Medicines’ Zolucatetide, the First and Only Direct Inhibitor of the Elusive Β-Catenin:TCF Interaction, Receives FDA Orphan Drug Designation for...
Parabilis Medicines announced that the FDA has granted both Orphan Drug and Fast Track designations to zolucatetide, the first direct inhibitor of the β‑catenin:TCF interaction, for desmoid tumors. Early Phase 1/2 data showed a 100 % disease‑control rate in ten patients and...
Orphan Product Risk Contracting
Ira Studin warns that while current policies such as TrumpRx and Medicare negotiations exempt orphan drugs, emerging mechanisms like most‑favored‑nation (MFN) pricing and pharmacy‑benefit‑manager (PBM) net pricing could introduce downward pressure. He argues manufacturers should evaluate risk‑contracting to safeguard pricing,...
What Matters More in DNA-Encoded Libraries: Size, Quality or Chemical Diversity?
DNA‑encoded libraries (DELs) enable billions of compounds to be screened, but X‑Chem argues that size alone does not predict success. By limiting synthesis to two‑three cycles, the company creates lead‑like molecules (350‑450 Da) with high three‑dimensional diversity, and its analysis of...
Chiesi and Bespak Partner to Advance Carbon Minimal Inhaler Production With UK Manufacturing Site
Chiesi Group and inhalation CDMO Bespak have expanded their long‑standing partnership by increasing pressurized metered‑dose inhaler (pMDI) manufacturing capacity at Bespak’s Holmes Chapel, UK site. The move supports Chiesi’s Carbon Minimal Inhaler (CMI) program, which targets up to a 90 %...
Gene Therapy for Duchenne Muscular Dystrophy: Genethon Confirms Two-Year Efficacy in Patients Treated with Its Drug Candidate GNT0004 at Therapeutic...
Genethon presented two‑year data from its dose‑escalation phase showing that GNT0004, administered at 3 × 10¹³ vg/kg, produced sustained functional gains and biomarker improvements in ambulatory DMD boys. Motor scores rose 9 points on the NSAA, the 6‑minute walk distance improved by...
Pharma Pulse: FDA Approval of Leucovorin Calcium for CFD and Persistent Inequities in OTC Naloxone Access
The FDA granted its first approval for a therapy targeting cerebral folate transport deficiency, an ultra‑rare neurological disorder, with Wellcovorin (leucovorin calcium) showing meaningful neurological improvements in 89% of patients. Meanwhile, over‑the‑counter naloxone prices have slipped by roughly $0.49 each...
When Ambition Meets Ambiguity: The Trends and Sentiments Shaping Biotech in 2026
ICON Biotech’s 2025 survey of 163 global biotech leaders, plus 100 Chinese executives, reveals shifting dynamics as China overtakes the United States in innovative trial volume and is projected to contribute 35% of FDA approvals by 2040. Funding remains scarce,...
Curatis and Neupharma Announce Exclusive Licensing Agreement to Develop and Market Corticorelin (C-PTBE-01) for the Treatment of Peritumoral Brain Edema...
Curatis Holding and Japan’s Neupharma have signed an exclusive licensing deal granting Neupharma rights to develop and market corticorelin (C‑PTBE‑01) for peritumoral brain edema (PTBE) in Japan. The agreement includes up to CHF 83.5 million in upfront and milestone payments and royalties...
Out-Of-Pocket’s 2025 Predictions | Out-Of-Pocket
Out‑Of‑Pocket’s 2025 outlook forecasts a turning point for several health‑care segments. Obesity drugs such as GLP‑1s are expected to become cost‑effective as pricing pressure and outcomes‑based contracts expand access. AI models will split, with healthcare‑specific versions emphasizing explainability, security and...
Pharmaceutical Executive Daily: FDA Expands Approval of Leucovorin
The FDA has expanded approval of leucovorin calcium tablets as the first therapy for cerebral folate transport deficiency linked to FOLR1 gene variants. The decision relied on published case literature, showing 87‑89% of patients achieved clinical improvement. In oncology, AstraZeneca...
Agomab IPO Raises $200M to Fund Fibrosis Therapies
Agomab Therapeutics NV closed its Nasdaq IPO, raising roughly $200 million by selling 12.5 million American Depositary Shares at $16 each. The capital will fuel its fibrosis‑focused pipeline, beginning with ontunisertib, an oral TGFβ‑ALK5 inhibitor for fibrostenosing Crohn’s disease that achieved its...
NX-1607
Nurix Therapeutics has launched NX-1607, the first orally bioavailable small‑molecule inhibitor of the immune regulator CBL‑B, into a Phase 1a/1b trial for advanced cancers. CBL‑B modulates activation of T, B and NK cells, and NX-1607 locks the protein in an inactive...
Deaminet 2026: Breakthroughs in Base Editing, Deaminase Biology, and Therapeutic Translation
Deaminet 2026 showcased accelerating advances in base and prime editing, highlighted by rapid Addgene distribution growth and new mechanistic insights such as PE6d’s heightened processivity and ABE8e’s dimer‑driven efficiency. Novel off‑target detection platforms like beCasKAS and Inrich‑seq revealed far more unintended...
Pharma Pulse: Ipsen’s Global Tazemetostat Withdrawal and FDA Approval of Deucravacitinib for Psoriatic Arthritis
French biotech Ipsen announced an immediate, global voluntary withdrawal of its oncology drug tazemetostat (Tazverik) after safety signals in the SYMPHONY‑1 confirmatory trial indicated a rise in secondary hematologic malignancies. The company is coordinating with the FDA to manage the...
How FDA's Removal of the Two-Trial Requirement Affects Development Programs
The FDA has eliminated the longstanding requirement for two pivotal clinical trials, adopting a single‑trial default to speed drug approvals. The agency pairs this change with a mandate for more rigorous post‑market surveillance to catch safety signals after launch. Pharmaceutical...
Researchers Take a Step Closer to Finding a Treatment for a Rare Genetic Neurodevelopmental Condition
Researchers at Texas Children’s Duncan Neurological Research Institute and Baylor College of Medicine demonstrated that skipping exon e2 of the MECP2 gene boosts MeCP2 protein production by 50‑60%, rescuing neuronal function in Rett syndrome mouse models and patient‑derived cells. The study...
Accrufer Becomes First FDA-Approved Prescription Iron Therapy for Children 10+
Shield Therapeutics announced that the FDA has approved Accrufer, its ferric maltol oral iron formulation, for children ages 10 and older, making it the first prescription oral iron therapy for this age group. The approval expands the drug’s label beyond...
Pharmaceutical Executive Daily: Vinay Prasad Set to Leave CBER for Second Time
Vinay Prasad has left the FDA’s Center for Biologics Evaluation and Research (CBER) for the second time in under a year, signaling a notable leadership turnover at a key regulatory hub for biologics, vaccines and gene therapies. In parallel, GlaxoSmithKline...
ORN0829
Taisho Pharmaceutical’s vornorexant (TS‑142), marketed as Vorzzz®, received Japanese regulatory approval in August 2025 as a dual orexin‑1/2 receptor antagonist for insomnia. The drug distinguishes itself from existing DORAs through rapid absorption and a short elimination half‑life, aiming to minimize...
Bristol Myers Squibb Announces Positive Phase 3 Results From the SUCCESSOR-2 Study of Oral Mezigdomide in Relapsed or Refractory Multiple...
Bristol Myers Squibb reported positive interim Phase 3 results from the SUCCESSOR‑2 study, showing that oral mezigdomide combined with carfilzomib and dexamethasone (MeziKd) significantly extended progression‑free survival versus carfilzomib‑dexamethasone alone in relapsed or refractory multiple myeloma. The trial marks the first...
Selectivity in Cells May Vary
The Chemical Probes Portal’s >30‑fold selectivity rule, originally based on cell‑free assays, is challenged by a new open‑access J. Med. Chem. study comparing DiscoverX kinase panels with NanoBRET cellular profiling. Researchers found that most inhibitors appear less potent in living...
Associated Press Cannot Explain Bewildering Reporting on FDA’s Tracy Hoeg and Antidepressant Risks
The Associated Press published a story alleging that FDA senior regulator Dr. Tracy Hoeg was pushing a petition from Dr. Adam Urato to add pregnancy‑risk warnings to antidepressants and that their personal relationship constituted a conflict of interest. The author,...
Module 2, Section 3: Target Validation
The module on target validation walks through how phenotypic and target‑based strategies intersect in immune‑focused drug discovery. It highlights recent literature on TYK2 pseudokinase stabilization as a mechanism to block T‑cell signaling, and cites Icotrokinra and Deucravacitinib as successful examples....
Key Factors Contributing to Uncertainty in Moderna's mRNA Vaccine Review
The FDA initially refused to review Moderna’s mRNA‑based flu vaccine, prompting surprise among industry observers. After a White House meeting, the agency reversed course and granted Moderna a Type A meeting, effectively resetting the review process. Lanton notes this regulatory flip‑flop...