Know What's Happening in Pharma
Know What's Happening in Pharma
FDA approves KRESLADI, first CIRM‑funded gene‑editing therapy for LAD‑I
The FDA has granted approval to KRESLADI, a gene‑editing therapy from Rocket Pharmaceuticals that treats severe leukocyte adhesion deficiency‑I in children without a matching bone‑marrow donor. This marks the first FDA‑approved product directly funded by California’s Institute for Regenerative Medicine (CIRM), highlighting the agency’s impact on translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma from GHO Capital
The FDA’s Oncologic Drugs Advisory Committee will convene on April 30 to evaluate AstraZeneca’s oral SERD camizestrant for first‑line HR‑positive, HER2‑negative breast cancer and its AKT inhibitor Truqap for metastatic hormone‑sensitive prostate cancer. Camizestrant’s Phase 3 SERENA‑6 trial reported a 56% reduction in disease progression or death, while Truqap combined with abiraterone improved radiographic progression‑free survival in the CAPItello‑281 study. The meeting ends a nine‑month hiatus for the ODAC and occurs amid growing criticism of panel balance and nuance.
The FDA rejected Incyte’s supplemental application to add non‑small cell lung cancer to Zynyz’s label, citing compliance failures at Novo Nordisk’s Catalent‑owned Indiana manufacturing plant. The agency’s complete response letter pinpointed inspection findings at the site as the sole approvability...
Shares of Hims & Hers Health surged after the telehealth firm announced a partnership with Novo Nordisk to sell the Danish drugmaker’s GLP‑1 weight‑loss products, including Wegovy. The deal reverses a recent legal clash in which Novo threatened litigation over...
Amgen’s Lumakras earned accelerated FDA approval in 2021 as the first KRAS‑targeted therapy, but sales have been modest, reaching $92 million in Q4 2025 with only an 8% year‑over‑year increase. A second G12C inhibitor, Krazati, entered the market in 2022, narrowing Amgen’s...
Roche’s oral SERD giredestrant failed to meet its primary endpoint in the phase 3 persevERA trial, showing no significant improvement over letrozole when combined with palbociclib in first‑line HR‑positive, HER2‑negative advanced breast cancer. The company now limits U.S. filing to ESR1‑mutated...
Macro: drug-safety shock; Key factor: SYMPHONY‑1 found secondary hematologic malignancies; Risk: regulatory re‑rating and pipeline impairment; Insight: trim Ipsen (IPN) exposure; favor defensive pharma 📉 — Viktor Kopylov, PhD, CFA. More insights: t.me/si14Kopylov
We will solve aging or will die trying. Check out the TopTown SuperDNA project. It is no longer a concept - projected launch in Q3 2028. Fully-integrated residential-biotechnology-clinical infrastructure is key to longitudinal AI-powered drug discovery. https://t.co/LK5XaTci29
Researchers have engineered an iron‑doped mesoporous silica nanoplatform (FOBA) that, when exposed to 808 nm near‑infrared light, uses the Y6 photothermal converter to heat and trigger a PEG gate, creating a transient solvent that enables in‑situ synthesis of the cytotoxic agent...
The $RLMD bladder cancer data (phase 2) reported this morning look very good. Another competitive blow to $IBRX particularly at its bloated valuation. https://t.co/wYThfwIpG8
9March: What a roller-coaster ride with $QURE and @US_FDA ! I sum up a wild week--plus $MRNA's could've-been-worse settlement with $ABUS & $ROIV (w/ insights from CEO Matt Gline) and updates from $DAWN and $TNGX in my latest StockWatch for...
Aisa Pharma announced positive Phase II data for AISA‑021, a once‑daily calcium channel blocker, in systemic sclerosis‑associated Raynaud’s phenomenon (SSc RP). The double‑blind, placebo‑controlled RECONNOITER trial enrolled 64 patients and showed a 22.1% reduction in weekly Raynaud attacks and a 155% placebo‑adjusted...
British drugmaker GSK has licensed worldwide rights to its experimental liver‑disease drug linerixibat to Italy’s Alfasigma for up to $690 million. The agreement includes a $300 million upfront payment, a $100 million tranche upon U.S. approval, $20 million upon European and UK clearance, and...
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...
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...
Researchers identified more than 200 metabolic enzymes physically attached to chromatin, revealing a distinct "nuclear metabolic fingerprint" for each cell type and cancer. Approximately 7% of all chromatin‑bound proteins were metabolic, including unexpected oxidative‑phosphorylation components. The fingerprint varied by tumor...
Ipsen is voluntarily withdrawing its EZH2 inhibitor Tazverik worldwide after an interim safety analysis in the phase 1b/3 SYMPHONY‑1 trial linked the drug to secondary hematologic malignancies. The pull‑back includes terminating all ongoing Tazverik studies and recalling the product in markets...
#GSK to get $300m upfront and $100m upon US FDA approval plus $20m for EU approval and up to $270m sales based payments from Alfasigma who will acquire rights to Linerixibat, a bile acid transporter inhibitor, currently under development.
Process Performance Qualification (PPQ) is the final validation step before commercial manufacturing of cell and gene therapies, but its complexity often triggers delays and compliance risks. The article highlights three proven strategies—early master‑plan development, continuous quality improvement, and data‑driven analytics—to...
Roche’s oral breast‑cancer therapy, touted as a potential blockbuster, missed its primary endpoint in the pivotal Phase 3 persevERA trial. The study evaluated the drug as a first‑line treatment for hormone‑receptor‑positive, HER2‑negative metastatic breast cancer and enrolled more than 1,200 patients...
Kyron.bio announced a strategic partnership with Servier to glycoengineer a Servier‑selected antibody using its precision glycosylation platform. Servier will fund the research and retain an option to pursue further development based on the results. The collaboration builds on Kyron.bio’s 2024...
Under the Trump administration, the U.S. government slashed nearly $500 million in mRNA research funding, canceling 22 projects and a $766 million Moderna contract. The FDA’s initial refusal then reversal to review Moderna’s flu vaccine highlighted regulatory skepticism toward the platform. Private‑sector...
Comparative analysis of senolytic drugs reveals mitochondrial determinants of efficacy and resistance "findings suggest that mitochondrial quality control is a key determinant of resistance to ABT263-induced and ARV825-induced senolysis, providing a possible framework for rational combination senotherapies." https://t.co/xB0wFkzIW9
Arcutis Biotherapeutics reported record Q4 2025 results, with net product revenue of $127.5 million, an 84% year‑over‑year increase, and full‑year revenue up 123% to $372.1 million. Management raised its 2026 net product revenue guidance to $480‑$495 million, citing strong prescription growth, expanded Medicare...
Researchers at the University of Massachusetts Amherst have engineered a logic‑gated nanoparticle that releases a STING agonist only under acidic pH and hypoxic conditions typical of metastatic tumor sites. The dual‑stimuli‑responsive system triggers robust innate immune activation while sparing healthy...
Zevra Therapeutics reported Q3 2025 net revenue of $26.1 million, driven primarily by $22.4 million from its FDA‑approved NPC therapy MyPlayFa. The company narrowed its net loss to $0.5 million, a dramatic improvement from a $33.2 million loss a year earlier, while cash and...
Editas Medicine reported Q2 2024 progress on its gene‑edited cell therapy reni‑cel, presenting interim data from the RUBY sickle‑cell and EdiTHAL beta‑thalassemia trials. All 18 RUBY patients were free of vaso‑occlusive events, with hemoglobin levels above 14 g/dL and fetal hemoglobin exceeding...
Researchers have engineered a dual‑stimuli‑responsive nanoparticle that activates the STING pathway only when both acidic pH and hypoxic NQO1 activity are present, creating an AND‑logic release of the agonist MSA‑2. In preclinical models of lung carcinoma, triple‑negative breast cancer and...
Anixa Biosciences reported $131.7 million in cash, no debt, and a cash burn of $7.1 million in Q1 2026, extending its runway to over three years. Operating expenses fell sharply, with R&D down 55% YoY and G&A reduced by 32%, reflecting the end...
MacroGenics reported 2024 revenue of $150 million, up from $58.7 million, largely fueled by $85 million in milestone payments from its Incyte partnership. The company posted a net loss of $67 million versus $9.1 million a year earlier, while cash and marketable securities fell to...
ARS Pharmaceuticals reported a pivotal quarter, with U.S. Neffy net product revenue soaring to $31.3 million—a 2.5‑fold quarter‑over‑quarter increase that beat consensus. New prescriber market share reached 10.3% and provider adoption rose 85% since August, while the Get Neffy On...
French pharma group Servier announced a definitive agreement to acquire Nasdaq‑listed Day One Biopharmaceuticals for $2.5 billion, paying $21.50 per share—a 68% premium to the prior close. The cash‑only transaction will be funded from Servier’s reserves and is slated to close...
stated as a critique but what does this mean as no drug has been demonstrated to have anti-aging effects in human clinical trials NR is active versus inflammaging and PAD, which is an age-related conditions affecting 10s of millions of americans you...
Too funny. @emilyakopp (below) outs Vinay Prasad as the host of Thursday’s HHS/FDA media call to discuss $QURE.
The FDA has issued a final guidance titled “Scientific Considerations in Demonstrating Biosimilarity to a Reference Product.” The document outlines the agency’s scientific framework for establishing biosimilarity of therapeutic proteins submitted via the abbreviated licensure pathway. It details expectations for...
Osteoarthritis & back pain affects millions of people. Instead of managing symptoms, imagine rebuilding joints by making them young New study shows the reprogramming gene combo OSK regrows joints in mice & effect depends on TET2 so it's epigenetic...🧵 https://t.co/AlOIwcEXEQ
Researchers at Uppsala University reported that a single 30 mg/kg injection of the psychedelic N,N‑dimethyltryptamine (DMT) reversed depression‑like behaviors in mice subjected to chronic stress. The treated animals recovered their preference for sweetened water, displayed improved working‑memory performance, and showed reduced...
On Prasad’s last few weeks as CBER chief: 1. It was absurdly inappropriate to bring a large group of reporters together and rail on a pending application “on background” 2. The walk back of the Moderna RTF was a bad...
GLP-1s already have market cap close to 1/3 of the top 10 biopharma companies combined. Consumer health biotech products will dwarf disease-focused biotech products.
Researchers led by Dr. James Murrough and Dr. Emma Guttman‑Yassky identified the Th2 immune pathway as a contributor to major depressive disorder. Using proteomic profiling and computer modeling, they repurposed dupilumab—an IL‑4 receptor antibody approved for eczema—to target this pathway....
Patent Fights, GLP-1 Turf Wars, and the FDA Tightens the Screws | Ep. 952 https://t.co/v0fulR3NfB https://t.co/9gGdkAVvlv
There's a lot you can learn about sleep. Like 83% hadn't heard of DORAs. https://t.co/2lV3Fw6cwg https://t.co/IWRTdPTMOL
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....
One of the most impressive advances we've ever seen for some autoimmune diseases: cures https://t.co/OZDuq4Iooy
Even with AlphaFold, protein structure is not a solved problem. And protein structure was never the bottleneck for drug development anyway. Let me explain.
Researchers have identified lysophosphatidylcholine acyltransferase 1 (LPCAT1) as a driver of head and neck squamous cell carcinoma (HNSCC) by amplifying mitochondrial oxidative phosphorylation through the COX17 chaperone. Overexpression of LPCAT1 reshapes mitochondrial membrane lipids, stabilizing COX17 and boosting cytochrome c oxidase activity,...
Eli Lilly used an AI‑driven digital twin to dramatically increase manufacturing capacity for its high‑demand GLP‑1 drugs Zepbound and Mounjaro. The virtual factory model allowed real‑time optimization of equipment, process parameters, and defect detection, delivering production volumes the company says would...
A recent preclinical study shows that inhibiting interleukin‑2‑inducible T‑cell kinase (ITK) markedly improves the efficacy of anti‑CD19 CAR‑T cells. Researchers combined an ITK‑selective inhibitor with a second‑generation CD19‑CAR construct, observing enhanced cytotoxicity, reduced exhaustion markers, and prolonged persistence in mouse...
Bitter Compounds as Multifunctional Agents against Diabetes-associated Cognitive Dysfunction: Bridging Metabolic Regulation and Neuroprotection https://t.co/Xv3xItlP20
Baylor did some new pics photos by my friend Agapito Sanchez Jr. Gosh I’m old, estoy viejo, but still making new vaccines, hope to have results of our hookworm anemia vaccine soon, hoping to reproduce our low cost Covid vaccine...
![[Comment] Aldosterone Synthase Inhibition in Resistant Hypertension: Promises and Unknowns](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/0a5065acfb3787a2e2e6999bae6248e9.webp)
Resistant hypertension affects up to 20 % of hypertensive patients and carries heightened cardiovascular risk. Recent phase‑3 studies of aldosterone synthase inhibitors such as baxdrostat and lorundrostat have demonstrated significant ambulatory blood‑pressure reductions, positioning them as potential fourth‑line agents beyond traditional...
