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 the continued utility, clarity, organization, and potential new topics of these five guidances, with a comment deadline of June 1, 2026. Updates could align SUPAC with modern risk‑assessment tools and recent CMC regulations, potentially reducing regulatory burden for manufacturers. Stakeholders have an opportunity to influence how future CMC change submissions are evaluated.
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...
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...
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...
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 %...
Aplantex and Investissement Québec Team up to Produce Molecules for the Cosmetic and Food Industries
Aplantex has partnered with Investissement Québec to use its state‑of‑the‑art demonstration plant for green biotechnology extraction. The collaboration gives Aplantex access to advanced equipment and technical experts, enabling faster refinement of plant‑based molecules with antioxidant, anti‑inflammatory, antimicrobial and anti‑aging properties....
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...
One-Time Pest Turned Eco-Warrior Degrades Polystyrene
Researchers from Harbin Institute of Technology and Stanford University found that the tropical cockroach Blaptica dubia can rapidly degrade polystyrene. In a 42‑day experiment, 50 roaches consumed about 6 mg of plastic per day, removing 54.9% of the material and achieving...
Eurofins Biomnis Launches New Clinical LC‑MS/MS Method for the Detection of Cereulide Toxin in Stool Samples
Eurofins Biomnis has developed and validated a new LC‑MS/MS method to detect and quantify cereulide toxin in human stool samples. The assay meets ISO 15189 requirements, accounting for matrix effects, and delivers turnaround times suitable for routine clinical labs. Cereulide, linked...
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...
Arguing for PPARα Agonist Fenofibrate to Be Geroprotective
Researchers report that fenofibrate, a PPARα agonist approved for hyperlipidemia, extends lifespan and reverses cellular senescence in several mouse models, including D‑galactose‑induced, 18‑month‑old, and SAMP8 strains. The drug reduces age‑related lipid accumulation and restores mitochondrial function by up‑regulating CPT1C, a...
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,...
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...
![Understanding the Science Behind Embryo Grading Improves IVF Decision Making [PODCAST]](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://kevinmd.com/wp-content/uploads/Design-1-scaled.jpg)
Understanding the Science Behind Embryo Grading Improves IVF Decision Making [PODCAST]
In a KevinMD podcast, reproductive endocrinologist Dr. Erica Bove breaks down embryo grading, contrasting day‑three cell counts with day‑five blastocyst morphology. She explains how labs assess cell number, fragmentation, trophectoderm and inner cell mass to assign grades such as 8A...
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...
Scientists Successfully Freeze and Rewarm Mouse Brain Slices
Researchers at Friedrich‑Alexander‑Universität Erlangen‑Nürnberg successfully vitrified mouse brain slices and, in a limited trial, an entire mouse brain, preserving neuronal structure and function after rewarming. By using a high‑concentration cryoprotective agent cocktail, they avoided ice crystal formation, maintained synaptic architecture,...
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...
3D Printing Meets Embryo Screening: Additive Manufacturing in IVF and Reproductive Medicine
Additive manufacturing is reshaping IVF and embryo screening by enabling high‑resolution microfluidic chips, biocompatible scaffolds, and precision tools. Two‑photon polymerization printers can produce sub‑50 µm channels in hours, cutting device lead times from weeks to days and improving embryo handling consistency....
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...
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...
Pulse Biosciences Announces Clinical Data From nPulse™ Vybrance™ System First-In-Human Clinical Durability Study of Benign Thyroid Nodule Ablation
Pulse Biosciences reported long‑term results from its first‑in‑human nPulse Vybrance study, showing an average 74% reduction in benign thyroid nodule volume after 15‑22 months. The data, presented at the 2026 North American Society for Interventional Thyroidology meeting, revealed no nodule...
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...
Improved Photosynthesis
Researchers identified a simple protein tail in hornworts, termed RbcS‑STAR, that causes Rubisco enzymes to cluster together, enhancing carbon‑concentrating potential. The STAR region was successfully transferred to Arabidopsis, demonstrating functional clumping in an unrelated model plant. This discovery offers a...
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...
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....
Electric Fields Allow Bioprinting of Aligned Muscle Fibers
Researchers have integrated an electric field into electrohydrodynamic (EHD) bioprinting to orient fibrin‑alginate hydrogels, producing nanofiber alignment that directs myocyte organization. The conductive polymer‑enhanced constructs exhibit improved myotube differentiation and mimic native muscle conductivity. In vivo tests on rats demonstrated...
A SEMA7A Feedback Loop in Macrophages Accelerates Atherosclerosis
Researchers identified macrophage‑derived Semaphorin 7A (SEMA7A) as a potent driver of atherosclerosis. Gene‑expression analysis showed high SEMA7A and its receptor integrin β1 in human mononuclear cells, and mouse models with macrophage‑specific Sema7a deletion exhibited a 57.2% reduction in lesion size and improved...
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...
ENHERTU® Granted Priority Review in the U.S. as Post-Neoadjuvant Treatment for Patients with HER2 Positive Early Breast Cancer
Daiichi Sankyo and AstraZeneca have received FDA priority review for ENHERTU® as a post‑neoadjuvant therapy in HER2‑positive early breast cancer. The decision follows the DESTINY‑Breast05 phase 3 trial, which showed a 53% reduction in invasive disease‑free survival events versus trastuzumab‑emtansine (T‑DM1). Three‑year...
Weekly Reads: Support Brain Tumor Work, Prasad Is Out (Again), Genetic Conditions, Texas AG, Immunotherapy Paper, SCBEM
The newsletter urges donations to support a lab studying lethal childhood glioma, noting NIH grant success rates of only 4‑5%. It reports FDA biologics chief Vinay Prasad’s second departure, a rare leadership turnover that could affect approval stability. Additional highlights...
Bayesian Inferences and Frequentist Evaluations
Researchers Forster, Novelli, and Welch applied four frequentist and two Bayesian sequential designs to the COVID‑disrupted UK DISC clinical trial. All six approaches confirmed the trial’s original finding of treatment superiority but suggested different optimal points for restarting patient recruitment....
Complete Genomics Sale: Gruyère or Emmental?
Swiss Rockets, a Basel‑based firm, announced the acquisition of Complete Genomics, taking full control of its CoolMPS sequencing technology and key personnel. The deal aims to shield the business from U.S. legislative bans tied to perceived Chinese ownership, while retaining...
Reviewing What Is Known of the Virome in Aging
Recent open‑access review maps the human virome’s role in aging, highlighting how gut and circulating viruses influence immunity, inflammation, and metabolism. Age‑related virome changes include expansion of bacteriophage families, reactivation of latent herpesviruses, and altered viral diversity, with centenarians displaying...
Early Encouraging UC Davis Trial Data on Cell Therapy for Spina Bifida
A first‑in‑human phase 1 trial at UC Davis evaluated placental mesenchymal stem cells delivered intra‑uterinely to fetuses with myelomeningocele. Six pregnancies treated between June 2021 and December 2022 resulted in intact repair sites, no cerebrospinal fluid leaks, infections, or tumor formation, and MRI scans...
Servier to Acquire Day One Biopharmaceuticals in a $2.5 Billion Cash Deal
France‑based Servier announced a $2.5 billion all‑cash acquisition of U.S. biotech Day One Biopharmaceuticals. The tender offer prices Day One shares at $21.50, a 68.23% premium, equating to 8.34 times the company’s sales. Servier will fund the transaction with existing cash and...
FMO-2 Upregulation Is Common to Multiple Longevity Associated Mutations in Nematodes
Researchers have identified flavin‑containing monooxygenase‑2 (FMO‑2) as a shared downstream effector in several long‑lived mitochondrial mutants of Caenorhabditis elegans, including clk‑1, isp‑1 and nuo‑6. RNA interference or genetic loss of fmo‑2 shortens the extended lifespan of these mutants, confirming its...
Taming the AI Chaos in Drug Discovery
Biopharma R&D is witnessing a rapid influx of specialized AI models for tasks such as structure prediction, retrosynthesis, and image analysis. While each tool delivers measurable benefits, their isolated deployment creates fragmented data streams, hidden costs, and increased cognitive load...
What Is Driving the Recent Wave of Layoffs in Biotech?
Biotech firms are cutting staff as venture‑capital inflows dry up, clinical‑trial setbacks mount, and operating costs climb. While breakthroughs in gene therapy, RNA therapeutics and precision medicine continue, companies are trimming non‑core programs to preserve cash. Strategic restructurings aim to...
Strategic Intelligence Report on Bladder Cancer
At the recent ASCO‑GU meeting, industry leaders highlighted that the greatest threat to emerging bladder‑cancer programs is strategic, not clinical. Phase‑2 candidates are poised to enter Phase‑3 trials, but shifting control arms, evolving endpoints, and changing patient demographics risk rendering...
Pharma Funding Roundup: Cognito Therapeutics Closes Oversubscribed $105 Million Series C Financing, Nexcure Launches $19 Million Series A Financing
Cognito Therapeutics closed an oversubscribed $105 million Series C round to fund its at‑home Alzheimer’s stimulation device, Spectris, targeting a 2027 market launch after a pivotal readout. NexCure raised $19 million in a Series A to build an outpatient platform that standardizes and expands...
Pirtobrutinib
Late 2025 saw the FDA grant traditional approval to pirtobrutinib, an oral, reversible BTK inhibitor targeting multiple B‑cell malignancies. The drug demonstrated robust efficacy in BTK‑resistant chronic lymphocytic leukemia and small lymphocytic lymphoma, backed by positive Phase 3 data and early...
Cancer-Eating Bacteria Engineered to Consume Tumors From the Inside Out
University of Waterloo researchers have engineered the anaerobic bacterium Clostridium sporogenes to consume solid tumors from the inside out. The spores infiltrate the oxygen‑free tumor core, proliferate and break down cancerous tissue. By inserting an oxygen‑resistant gene and a quorum‑sensing...
Xtalks Featured Member: Giorgia Palano, Life Science Consultant, Knightec Group
Xtalks has spotlighted Giorgia Palano, PhD, a Life Science Consultant at Knightec Group, highlighting her expertise in validation strategies and regulatory compliance. Palano works with cross‑functional teams to ensure quality documentation and continuous improvement across complex life‑science operations. She emphasizes...
