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
Beren Therapeutics announced that the U.S. Food and Drug Administration has accepted its New Drug Application for adrabetadex, an investigational therapy aimed at treating infantile‑onset Niemann‑Pick disease type C (NPC). The agency set a target action date of 17 August 2026 under the PDUFA timeline. The submission includes data indicating a clinically meaningful survival benefit, biomarker improvements and slowed disease progression. Adrabetadex previously earned breakthrough therapy designation in 2025 and now benefits from priority review.
A new combination‑therapy approach for inflammatory bowel disease (IBD) aims to boost remission rates beyond the current 30 % success benchmark. By pairing agents that target distinct immune pathways, early trials show remission climbing to roughly 55 % and faster mucosal healing....
The nonprofit Every Cure is launching computational pharmacophenomics, an AI‑driven approach that systematically evaluates all 4,000 FDA‑approved drugs for the 14,000 diseases lacking treatments. Its MATRIX platform integrates massive biomedical knowledge graphs, generates graph embeddings, and ranks 75 million drug‑disease pairs,...
Researchers published a comparative analysis of replicative versus chemically‑induced senescence in articular chondrocytes, revealing divergent molecular signatures, secretory profiles, and mitochondrial dynamics. Replicative senescence displayed a controlled, anti‑inflammatory SASP and stable epigenetic marks, while chemically‑induced senescence produced a pro‑inflammatory secretome,...
Sebela Pharmaceuticals has licensed its hormone‑free copper intrauterine device, Miudella, to Organon for exclusive global rights. The agreement provides Sebela with a $27.5 million cash payment at closing and up to $505 million in sales‑based milestone payments, plus double‑digit royalties on net...
Hims & Hers disclosed its fourth‑quarter earnings and announced that it has withdrawn the controversial GLP‑1 weight‑loss pill it briefly launched. The decision followed conversations with key ecosystem stakeholders, including regulators and partner pharmacies. The company highlighted modest revenue growth...
Researchers created a kinase‑inactive RIPK3 D143N mouse that remains viable, revealing that RIPK3’s scaffold function alone can drive TNF‑induced inflammatory pathology. The mutant protein continues to recruit RIPK1, FADD and activate NF‑κB, producing cytokine storms without necroptotic cell death. In...
Oncology drug waste reaches up to 30%, costing over $2 billion annually in the U.S. A 2023 CMS rule now forces manufacturers to refund Medicare for discarded volumes beyond a 10% threshold, adding financial pressure. Traditional forecasting relies on static sales...
Southern RNA and Providence Therapeutics announced the PaedNEO‑VAX trial, a world‑first personalized mRNA vaccine study for children with advanced brain tumors. The multi‑site trial, funded by Providence, the Australian government and donors, will launch in March 2026 across eight pediatric...
Researchers at EPFL have created a drug‑controlled CAR‑T platform called DROP‑CAR that can be turned off on demand with the FDA‑approved BCL‑2 inhibitor venetoclax. The system places a reversible protein‑protein interaction on the cell surface, causing the CAR to dissociate...
The UK government will add a £3,000 average annual bonus to GP contracts for prescribing the weight‑loss drug Mounjaro, plus a £1,000 incentive for referrals to weight‑loss programmes, starting in April 2026. The scheme targets patients who meet current NHS...
UC Davis engineers unveiled VESSEL, a cell‑free platform that produces extracellular vesicles bearing a single surface protein. By isolating individual proteins, the system enables direct functional mapping, exemplified by the discovery that CADM1 promotes vesicle uptake. The approach is scalable...
Jazz Pharmaceuticals reported a record $1.126 billion Q3 2025 revenue, propelled by double‑digit growth in its sleep franchise and the successful launch of the rare‑disease drug Modesto. Xywav and Epidiolex sales rose 11% and 20% year‑over‑year, while oncology product Zepzelca saw an...
Cytokinetics announced rapid global regulatory clearance for MYCorzo, securing FDA, China NMPA, and European Commission approvals within a single week. The company launched the drug in the U.S. with over 700 REMS‑certified physicians and more than 12,000 customer engagements in...
Day One Biopharmaceuticals reported a breakout 2025, with Ojemda generating $155.4 million in net product revenue—a 172% year‑over‑year increase—and over 4,600 prescriptions. The company achieved its first profitability milestone as Ojemda’s revenue exceeded combined cost of sales and SG&A, while ending...
Revvity reported Q4 2025 revenue of $772 million, delivering 4% organic growth and a 7% rise in its diagnostics segment. Adjusted EPS came in at $1.70, topping the high end of guidance, while adjusted operating margin held at 29.7% despite...
BridgeBio Pharma reported Q4 2025 revenue of $120.7 million, a surge driven primarily by Attruby net product sales of $108.1 million and expanding royalty streams. The company announced statistically significant Phase III readouts for BBP-418 in limb‑girdle muscular dystrophy and...
Harmony Biosciences reported $714.7 million net product revenue for 2024, a 23% year‑over‑year increase, with Q4 sales of $201.3 million driven largely by its flagship narcolepsy drug Wakix. The company announced a generic settlement with Novogen that postpones competition until at least...
Incyte reported a strong Q4 2025, delivering $1.51 billion in revenue, a 28% year‑over‑year increase, and $5.14 billion for the full year, up 21%. Core products drove the surge: Jakafi posted $828 million in Q4 sales, Opsilura $207 million, and hematology/oncology sales jumped 121%...
AbCellera Biologics reported a transformative 2024, shifting from a partnership platform to a clinical‑stage biotech. The company advanced two internal antibody programs, ABCL635 and ABCL575, toward CTA filings in Q2 2025 and highlighted a $800 million liquidity position. It outlined a...
Iovance Biotherapeutics reported third‑quarter product revenue of about $68 million, a 13% sequential increase, driven primarily by $58 million in Amtagvi sales and $10 million in Proleukin revenue. Gross margin improved to roughly 43% after a restructuring that cut cost of sales by...
Fulcrum Therapeutics reported a strong fourth‑quarter, posting $1.51 billion in revenue, a 28% year‑over‑year increase, and a full‑year 2025 revenue of $5.14 billion, up 21%. Core product sales accelerated, led by Jakafi’s $828 million Q4 revenue and Opsilura’s 28% sales jump to $207 million....
Vanda Pharmaceuticals reported a 9% rise in total revenue to $216.1 million for 2025, driven primarily by a 24% jump in Fanapt sales and a successful bipolar disorder launch. The company secured FDA approval for tradipitant (Nirius) for motion‑sickness prevention, marking...
So the key here is look at how quickly $HROW's Vevye is catching up with $BLCO's Miebo - this is happening despite the Miebo head start (which provides a waterfall effect from refills) and way more extensive detailing and DTC...
Who are the people who thought cagrisema was worth this much for $NVO? I want to find and study them. The cross trial comparison data that we already had clearly showed Cagrisema was worse than $LLY by essentially the...
Ascletis Pharma announced that its oral fatty‑acid‑synthase inhibitor denifanstat (ASC40) achieved positive Phase III results in moderate‑to‑severe acne vulgaris. In a 480‑patient double‑blind trial, 33% of participants reached clear or almost‑clear skin versus 15% on placebo, and an open‑label safety study...
Scientists at the University of Nottingham have unveiled a modular materials platform that self‑assembles with RNA to form nanoscale delivery particles. The system uses reversible host‑guest linkages, allowing rapid tuning of stability and behavior for diverse therapeutic needs. In pre‑clinical...
Below is the story of the first patient treated with a prime-edited therapeutic, developed by @PrimeMedicine in a trial led by Dr. Élie Haddad and his team at CHU Sainte-Justine. This teenager suffered from chronic granulomatous disease (CGD), an immunodeficiency,...
Merck has created a standalone oncology business unit to confront the concentration risk posed by Keytruda, which now generates over half of its pharmaceutical sales and faces patent expiry around 2028. The restructuring treats organizational design as a strategic lever...
Pharmaceutical giant Bayer has filed a lawsuit against Johnson & Johnson, accusing the latter of false advertising around its prostate‑cancer drug Erleada. Bayer claims J&J’s February 2 press release and website slides used flawed data to portray Erleada as more effective...
$GILD $ACLX $7.8 billion acquisition 68% premium, Ph3 just starting. Validates new FDA policy for a single pivotal trial validated by BLA acceptance. Large pharma moving faster, de-risked. $IMMX thesis and data grows stronger, 95% (CR), same parallel strategy, and clear...
Researchers in Aging Cell reveal that the nuclear sirtuin SIRT6 safeguards brain health by preserving nucleolar integrity and curbing excess protein synthesis. Loss of SIRT6 triggers nucleolar enlargement, heightened rRNA production, and uncontrolled protein translation, leading to protein aggregation and...
Merck is reorganizing its Human Health division into two distinct units—Oncology and Specialty, Pharma & Infectious Diseases—to sharpen focus as its flagship immuno‑oncology drug Keytruda approaches patent expiry. Jannie Oosthuizen, formerly head of U.S. Human Health, will lead the Oncology...
Generate Biomedicines, Inc. (GENB) launched a $400 million IPO on Feb 23, 2026, offering 25 million shares at $15‑$17 each on NASDAQ. At the midpoint price of $16, the company would be valued at roughly $2.04 billion. The proceeds are earmarked for two Phase 3 trials...
The FDA released draft guidance introducing the Plausible Mechanism Framework, a new approval pathway for individualized therapies targeting ultra‑rare diseases. The framework permits sponsors to seek clearance for gene‑editing and RNA‑based treatments when traditional randomized trials are infeasible due to...
Intrinsically disordered proteins (IDRs) make up roughly 79 % of human cancer‑associated proteins and defy traditional structure‑based drug design. Akshay Uttarkar’s team introduced QuPepFold, a Python package that translates short peptide sequences onto a tetrahedral lattice and uses a CVaR‑optimized...
By chance I was writing about anito-cel vs. cilta-cel on Friday, not knowing $GILD acquisition was coming down the pike. There are some crucial differences between the two products to take note of, which may explain why they were...
Building off the Baby KJ success story: FDA unveils long-awaited guidance on new pathway for individualized therapies - https://t.co/Uetp1cGFq2
The FDA’s Commissioner’s National Priority Review Voucher (CNPV) pilot compresses review timelines to one‑to‑two months for qualifying drugs, but its criteria favor applications with mature data packages. Compass Pathways’ synthetic psilocybin (COMP360) was listed as eligible yet did not receive...
Recent analyses of BCMA CAR‑T therapies reveal that superior response rates can be achieved without the historically accepted trade‑off of delayed movement and neurocognitive toxicities (MNTs). Emerging data pinpoint specific construct features—particularly signaling domains and hinge designs—as the primary drivers...
Balancing potency with oral bioavailability remains a core hurdle as drug candidates grow larger and more complex. Researchers now focus on "chameleonicity"—the ability of a molecule to toggle between polar and lipophilic conformations—to reconcile solubility and permeability. The article outlines...
IRB Barcelona has unveiled Spain’s first fully integrated spatial omics platform, uniting spatial transcriptomics, proteomics, histopathology, advanced microscopy and bioinformatics under one workflow. The facility draws on five core units to deliver an end‑to‑end pipeline from sample preparation through computational...
A Taiwanese retrospective cohort of 24,259 adults with type 2 diabetes and liver cirrhosis found that initiating sodium‑glucose cotransporter‑2 inhibitors (SGLT2is) markedly reduced the risk of end‑stage kidney disease, acute kidney injury, major adverse cardiovascular events, all‑cause mortality, and hepatic decompensation...
Why is $BCAX testing a ficera dose in phase 3 that it now says isn't optimal (and other uncomfortable questions)? Via @ApexOnco -> https://t.co/mLWKP6AAZ9 $JNJ $GMAB
Recent research reveals that several snake lineages have completely lost the hunger hormone ghrelin and its activating enzyme, yet maintain normal energy balance. Modern anti‑obesity drugs, such as GLP‑1 receptor agonists, achieve weight loss by amplifying satiety signals rather than...
The European Commission has pledged €225 million through a pre‑commercial procurement model to accelerate the development of next‑generation influenza vaccines. Ten entities, including Sanofi and Bavarian Nordic, will receive eight‑year contracts that cover clinical testing through market authorisation. The funding targets...
That feeling you get when you run a head-to-head study and the rival drug beats you out. Spending a fortune to hand your competitor a marketing message. Sometimes it feels like $NOVO is snake bit. $LLY https://t.co/gGUjl6iqPg
The module outlines a five‑dimensional framework for target selection, linking biological relevance, drugability, disease impact, competitive landscape, and development risk. It contrasts first‑in‑class and best‑in‑class strategies, highlighting how pioneering mechanisms can command premium market positions. The content identifies oncology as...
RNA interference (RNAi) has moved from a scientific curiosity to a fast‑growing therapeutic platform, with the global market projected to reach $1.97 billion by 2032. Six biotech firms—Aerska, ADARx, Alnylam, Arrowhead, City Therapeutics and SanegeneBio—are leading the next wave of innovation...
Goal-Driven Flexible Bayesian Design presentation updated w/comparison of performance of frequentist group sequential designs: https://t.co/QCZ6DQrm07 . Frequentist approach takes far too long to make a decision by controlling something that is NOT an error prob. #Statistics #rct https://t.co/X2knbmzTuX
