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
Scientists at Gladstone Institutes identified two host genes, DDIT4 and ZNF254, that act as molecular locks keeping HIV dormant after antiretroviral therapy (ART) cessation. Multi‑omic analysis of 75 participants from analytical treatment interruption trials linked higher expression of these genes, as well as stem‑cell memory CD8+ T cells and atypical NK cells, to delayed viral rebound. The common diabetes drug metformin was shown to induce DDIT4, blocking HIV reactivation in vitro, suggesting a low‑cost, repurposable pathway toward ART‑free remission. The team plans preclinical and clinical studies to test metformin’s therapeutic potential.
The FDA has added explicit cardiovascular risk information, including heart‑attack warnings, to the labeling and Medication Guide for rosiglitazone (Avandia) and its combination products. The revised label restricts use to patients already on the drug or those whose blood sugar...
The sterile injectable contract manufacturing market is rapidly evolving as demand surges for both large‑scale biologics and niche, small‑batch therapies. Leading CDMOs are responding by expanding production capacity, building global redundancy, and investing heavily in specialized talent. Development and pre‑commercial...
One of the most remarkable things about 5-meo-DMT as an antidepressant is that the psychoactive experience is so brief. 10 minute peak, perhaps. 20 min total. And people seldom remember it well. Yet it has possibly the largest anti-depressant effect...
A Financial Times analysis of Statista data identifies Europe’s five fastest‑growing pharma and biotech firms between 2021 and 2024. Italy’s Itaste Medical surged to €18.4 billion in sales, a 2,035% absolute growth, while the UK‑based Grow Group expanded cannabis‑based medicines to...
Dr. Michael Yeadon highlights testimony from former Pfizer Europe chief toxicologist Dr. Helmut Sterz, who claimed essential toxicology studies were omitted to accelerate approval of Pfizer‑BioNTech’s mRNA COVID‑19 vaccine. Sterz alleges the product lacked carcinogenicity and reproductive toxicity data, was...
Researchers at the University of Edinburgh have engineered bacteria to transform PET plastic waste into levodopa, a primary treatment for Parkinson’s disease. By inserting a seven‑gene, four‑step biosynthetic pathway into Escherichia coli, the team converted both industrial PET feedstock and...
Since 1995 the FDA has moved from approving standard opioid products to implementing a comprehensive regulatory regime aimed at curbing misuse and overdose. Key milestones include the 2007 authority to require Risk Evaluation and Mitigation Strategies (REMS), a series of...
The FDA’s Center for Drug Evaluation and Research (CDER) launched the Framework for Regulatory Advanced Manufacturing Evaluation (FRAME) to create a regulatory pathway for emerging manufacturing technologies. FRAME focuses on four priority technologies—end‑to‑end continuous manufacturing, distributed manufacturing (including non‑traditional sites),...
Phase 3 data presented at the 2026 Conference on Retroviruses and Opportunistic Infections show that the doravirine‑islatravir (DOR/ISL) two‑drug regimen met non‑inferiority criteria versus the standard bictegravir/emtricitabine/tenofovir alafenamide (BIC/FTC/TAF) triple‑pill. At week 48, 91.8% of participants on DOR/ISL achieved viral suppression compared...
BTK inhibitors have become the cornerstone of chronic lymphocytic leukemia therapy, replacing traditional chemotherapy. Brukinsa (zanubrutinib) is a second‑generation irreversible inhibitor that offers high selectivity and strong efficacy in treatment‑naïve or early‑relapse patients, with reduced cardiovascular risk. Jaypirca (pirtobrutinib) is...
The FDA updated its safety review of recombinant human growth hormone (somatropin) after the French SAGhE study raised concerns about a possible increased risk of death. The agency identified significant design flaws in the study and found no supporting evidence...
Kyorin Pharmaceutical Co. has signed a worldwide licensing agreement with Japan’s UBE Corp., securing exclusive rights to develop, manufacture and commercialize UBE’s novel drug candidates. The deal includes an undisclosed upfront payment and milestone‑based royalties, and it nudged Kyorin’s shares...
Europa Biosite has formed a strategic distribution partnership with Quantoom Biosciences to bring Quantoom’s Ntensify® mano and micro RNA production technologies to European researchers. The deal also anticipates future distribution of Quantoom’s Ncapsulate® LNP formulation kits. By adding rapid, high‑quality...
Rhythm Pharmaceuticals' Imcivree (setmelanotide) received FDA approval as the first treatment for acquired hypothalamic obesity, expanding its indication to adults and children aged four and older. The Phase III TRANSCEND trial demonstrated a 15.8% mean BMI reduction at 52 weeks, an...
Healio’s latest editorial highlights the expanding role of GLP‑1 receptor agonists in immune‑mediated inflammatory diseases, emphasizing weight‑independent anti‑inflammatory mechanisms. Emerging data suggest central nervous system GLP‑1 activation can modulate peripheral immunity, linking the gut‑brain‑immune axis. The FDA’s recent approval of...
Xeruborbactam (QPX‑7728) is a broad‑spectrum β‑lactamase inhibitor designed to revive the activity of β‑lactam antibiotics against multidrug‑resistant Gram‑negative bacteria. Developed by Qpex Biopharma and Shionogi, the molecule targets both serine‑ and metallo‑β‑lactamases, addressing a key resistance mechanism. Preclinical data show...
The U.S. Supreme Court denied Takeda Pharmaceutical and Eli Lilly’s request to block a racketeering lawsuit, allowing a class action alleging the companies concealed Actos’s link to bladder cancer to proceed. The suit targets tens of thousands of insurers and other...
The article argues that drug pricing debates must focus on aligning prices with the full value medicines deliver to patients and society. It critiques current health technology assessment (HTA) practices for using narrow benefit definitions, which can depress prices and...
Portugal’s biotech sector is gaining momentum, highlighted by BIO‑Europe Spring in Lisbon. Six home‑grown companies—Beat Therapeutics, BSIM Therapeutics, Exogenus, Immunethep, Mondego Bio and TechnoPhage—are advancing novel therapies ranging from DDR inhibitors for pancreatic cancer to exosome‑based wound treatments and bacteriophage...
Theranica’s Nerivio, a remote electrical neuromodulation (REN) device, has secured nationwide coverage for roughly 130 million lives through Blue Cross Blue Shield plans. The therapy’s dual acute and preventive indications offer a drug‑free alternative that expands access to underserved groups such...
The ongoing Iran‑Israel conflict has not yet crippled global pharmaceutical supply chains, but it threatens the modest 0.3% of worldwide drug output and 0.6% of active‑pharmaceutical‑ingredient (API) production that originates in the region. Disruptions to key shipping lanes, especially the...
The $PFE/ $VALN Lyme vaccine was a product for which there was a lot of hope. Results today show OK efficacy. Vaccine efficacy landed at 70%; investors hoped for 80% but thought as low as 60% would be relevant. But the...
Another win for Dizal, but how do US patients get hold of Zegfrovy? Via @ApexOnco -> https://t.co/4GVjyHS9vz https://t.co/bjjrnZkNL6
Pharmaceutical manufacturers are re‑examining traditional PBM‑centric distribution by offering direct‑to‑employer drug purchasing models. Companies like Andel charge a per‑prescription transaction fee and eliminate administrative or per‑member fees, aiming to lower drug spend for employers. Their platform centralizes prescription intake, routes...
FDA seeks feedback on voucher program; Study data lift ‘overhang’ on Insmed shares https://t.co/BCRJAUwGIJ $INSM + 7% $SRPT $SNY $GSK #biotech
As someone who lived in Vermont and still spends the summer there, it's disappointing to see the ambiguity in Pfizer's data on a new Lyme disease vaccine. It's a serious threat. Now we can move on to speculating on the...
The FDA’s recent safety alert warns that high‑dose levodopa/carbidopa regimens can provoke seizures when patients develop vitamin B6 deficiency. Fourteen post‑marketing cases, including two fatalities, involved doses exceeding 1,000 mg daily and featured focal‑onset seizures that generalized. Vitamin B6 supplementation halted...
Paradox: The FDA approved some fecal transplants for C. difficile. But accessing treatment got harder. https://t.co/AFFyCGPeKW
The news is mixed in the preliminary results from a Phase 3 trial of a needed #Lyme disease vaccine, @matthewherper reports. https://t.co/bcbfeXchk1
Kali Therapeutics has signed a license agreement with Sanofi granting the French giant exclusive worldwide rights to KT501, a next‑generation tri‑specific T‑cell engager aimed at B‑cell‑mediated autoimmune diseases. The deal provides Kali with $180 million in upfront and near‑term payments and...
From Prognosis to Action: Circulating Tumor DNA and the Next Phase of Risk Stratification in Triple-Negative Breast Cancer [Mar 18, 2026] Schneider & @StoverLab @JCO_ASCO https://t.co/6jPBxQe9QP #bcsm #PrecisionMedicine #LiquidBiopsy https://t.co/MOG73iTcNl
Guideline Update Provides New Testing & Tx Recx for Pts w/ ER-Positive, HER2-Negative Met Breast Cancer w/ ESR1 Mutations [May 17-18, 2023] @ASCO Daily News - https://t.co/3ZVrEkQEN5 @DrHBurstein et al. @JCO_ASCO https://t.co/68e7D0CSgU #bcsm #PrecisionMedicine #LiquidBiopsy https://t.co/KBx2hq2BDW
Oryon Cell Therapies presented interim Phase 1b/2a data showing that its autologous dopaminergic neuron‑replacement therapy produced sustained motor improvements in Parkinson’s patients. Five participants experienced 29‑62% reductions in OFF‑state MDS‑UPDRS Part III scores over 6‑18 months, with continued gains beyond six months....
Lenalidomide + high-dose dexamethasone (RD) vs lenalidomide + low-dose dexamethasone (Rd) as initial therapy for newly diagnosed multiple myeloma: an open-label RCT [10/22/2009] @VincentRK et al. @TheLancetOncol https://t.co/hXxlFINejC #NCT00098475 #EAonc E4A03 #mmsm #caxtx #ctsm https://t.co/3Pa2TRMntk
BRE12-158: A Postneoadjuvant, Randomized Phase II Trial of Personalized Therapy Versus Treatment of Physician's Choice for Patients With Residual Triple-Negative Breast Cancer [Dec 15, 2021] Schneider et al. Radovich @JCO_ASCO https://t.co/oJm6BJrMtA #bcsm #PrecisionMedicine
Gene therapies promise one‑time cures for diseases like sickle‑cell and inherited blindness, but their price tags—often $1 million to $3 million per patient—clash with the U.S. insurance model. More than half of new cell and gene therapies face coverage restrictions because insurers...
Genome-Wide Associations [GWAS] and Functional Genomic Studies of Musculoskeletal Adverse Events in Women Receiving Aromatase Inhibitors [Sep 20, 2010] Ingle et al. @DrWeinshilboum @JCO_ASCO https://t.co/1NJcXkWy8c #bcsm #Supponc
Assoc of Circulating Tumor DNA & Circulating Tumor Cells After Neoadjuvant Chemotherapy w/ Disease Recurrence in Pts w/ Triple-Negative Breast Cancer: BRE12-158 RCT [Jul 9, 2020] Radovich et al. @JAMAOnc https://t.co/XxH9kTT9d5 #bcsm #cactc #NCT02101385 https://t.co/kgV0yM2I1f
Henlius announced that China’s National Medical Products Administration (NMPA) has granted IND clearance for HLX18, a biosimilar of Opdivo (nivolumab), targeting resected solid tumours. The company also reported first‑patient dosing in Phase I trials for HLX17 (Keytruda biosimilar) and HLX13...
Pfizer's phase 3 for its Lyme trial was supposed to be 18k patients -- but issues with a contractor at the start of the trial cut the size by a third, and @ky_lahucik + I reported a long time ago...
P7C3 is an activator of an enzyme that makes NAD called NAMPT. Cool that it’s working in a 🐁 model of AD 👏
Xilio Therapeutics reported a strong cash position of $137.5 million, extending its runway to the end of 2027, and highlighted significant pipeline advances. The company plans to file an IND for its bispecific PD‑1/masked IL‑2 candidate XTX501 in mid‑2026 and start...
New @nberpubs: "From Free Rider to Innovator: The Rise of China's Drug Development" https://t.co/cQpEqRC1jy https://t.co/pSL5kCzlnO
Pfizer’s Lyme vaccine shows efficacy, but misses key statistical hurdle There is a set up for drama here. $Valn $pfe Valneva stock is down 14% in premarket trading. https://t.co/WN0KOl4Nqh via @statnews
Latent Labs unveiled Latent‑Y, an autonomous AI agent that designs therapeutic antibodies from natural‑language prompts. Powered by the Latent‑X2 generative model, the platform compresses weeks of expert work into hours and can run multiple design campaigns in parallel. In three...
Pyxis Oncology announced completion of target enrollment for its Phase 1 MICVO monotherapy study in recurrent/metastatic head and neck cancer, enrolling roughly 40 patients. Interim CEO Thomas Civik highlighted promising early efficacy, with a 46% objective response rate as monotherapy and a...
Insmed announced that a Phase 3 trial showed adding its inhaled antibiotic Arikayce to standard therapy significantly improved respiratory symptoms and boosted culture conversion rates in patients with newly diagnosed mycobacterium avium complex (MAC) lung infection. The study met its primary...
Avalo Therapeutics announced its 2025 financial results, reporting $98.3 million in cash and short‑term investments that should fund operations into 2028. Research and development expenses jumped to $50.1 million, driven by the Phase 2 LOTUS trial of abdakibart (AVTX‑009) for hidradenitis suppurativa. The...
Researchers have revived a dead bacterial cell by transplanting the complete genome of Mycoplasma capricolum into a chemically inactivated host, creating the first living synthetic bacterium assembled from non‑living parts. The experiment builds on the 2010 landmark where a synthetic...
