Know What's Happening in Pharma
Know What's Happening in Pharma
FDA greenlights KRESLADI, first CIRM‑funded gene‑editing therapy for LAD‑I
The FDA approved KRESLADI, a gene‑editing treatment from Rocket Pharmaceuticals for severe leukocyte adhesion deficiency‑I in children lacking a bone‑marrow donor. It is the first product directly funded by California’s Institute for Regenerative Medicine, highlighting CIRM’s role in translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma
Eurofins CDMO Alphora has launched an AI‑powered software that predicts salt and co‑crystal formation for APIs and intermediates, developed in partnership with a local university. The machine‑learning platform is now a core component of its solid‑state screening programs, delivering highly accurate predictions that reduce trial‑and‑error experimentation. By shortening development timelines and lowering screening costs, the tool enables faster, data‑driven decisions in drug substance development. The solution is backed by Alphora’s seasoned solid‑state R&D team, offering end‑to‑end support from data interpretation to experimental strategy.
Peptide therapeutics are powerful. But making them has always been difficult. Traditional chemical synthesis can involve long routes, poor selectivity, and significant environmental impact, while biological production is often limited by the pathways evolution happened to provide. What if we could design...
Q4 earnings highlighted a mixed biotech landscape, with regulatory turbulence easing for some firms while high‑stakes catalysts loom. Capricor Therapeutics is preparing to resubmit its Duchenne cardiomyopathy cell therapy after the departure of FDA CBER director Vinay Prasad, and reported...
Researchers identified a high‑altitude Retsat Q247R mutation that enhances myelin formation under hypoxic stress and accelerates remyelination in mouse models. The variant boosts neuronal production of the vitamin‑A‑derived metabolite ATDR, which activates the RXR‑γ pathway in oligodendrocyte progenitors. Administering ATDR...
Immutep’s LAG‑3 fusion protein eftilagimod‑alpha failed to improve overall survival in a Phase 3 randomized study in non‑small cell lung cancer. The trial, enrolling roughly 600 patients, did not meet its primary endpoint and showed no statistically significant benefit versus standard...
Ultragenyx announced that its investigational gene therapy for ornithine transcarbamylase (OTC) deficiency met a primary endpoint in a Phase 3 trial involving 37 patients. The study demonstrated a statistically significant reduction in blood ammonia levels, the key marker of disease...
Orphan‑drug sales are projected to exceed $400 billion by 2032, more than double the 2025 level. Small‑molecule therapies dominate the pipeline, accounting for 45% of the 20 most valuable orphan candidates, signaling a resurgence after years of biologic focus. The Inflation...
Parkinson’s research has entered a pivotal phase, driven by deeper disease insights and advanced models such as patient‑derived iPSCs. Despite a pipeline of potential disease‑modifying therapies, funding shortfalls and outdated trial endpoints continue to impede progress. Parkinson’s UK’s Virtual Biotech...
LabConnect announced the grand opening of a new central laboratory in Wuxi, China, expanding its global network to eight sites. The facility, built with Teddy Laboratory (now Frontage Laboratories), integrates LabConnect’s technology‑driven logistics and data oversight with local expertise. It...
I teach medical students that fatty liver disease (MASLD) affects 1 in 3 adults and has limited approved drug treatments. That may be changing. Hebrew University researchers found CBD and CBG -- two non-psychoactive cannabis compounds -- reversed fatty liver in mice...
Australian biotech Immutep saw its ASX shares tumble more than 88% after the independent data monitoring committee recommended halting its pivotal phase‑3 TACTI‑004 trial of the LAG‑3 inhibitor eftilagimod alfa (efti) in first‑line non‑small cell lung cancer. The trial, which...
Otsuka Pharmaceutical disclosed early open‑label extension (OLE) data for repinatrabit (JNT‑517) in adolescents with phenylketonuria (PKU). A 75 mg twice‑daily regimen achieved a 67% mean reduction in blood phenylalanine by day 56, with responses observed across prior sapropterin responders, non‑responders, and a...
A head‑to‑head trial published in the New England Journal of Medicine compared Eliquis (apixaban) and Xarelto (rivaroxaban) in patients with deep‑vein thrombosis. The study found Eliquis significantly reduced major bleeding risk while maintaining equal efficacy in preventing clot recurrence. This...
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South Africa’s Red Cross Children’s Hospital is using donated Trikafta, a $300,000‑a‑year cystic fibrosis therapy, to keep seven‑year‑old Jaylin Leitjies alive after his lungs were severely damaged in infancy. The drug, produced by Vertex Pharmaceuticals, is not registered with SAHPRA...
When Erum Khan asked me why 80% of biotech companies fail at execution, I replied: "The majority of synthetic biology founders have become enamored with the tool, the technology that they developed and then they went out there with that hammer...
Evotec, Vistagen lay off staff; Immutep shares collapse on study failure https://t.co/OikmCdJ6sg $EVO $VTGN $IMMP - 81% $RARE $ATRA
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...
"Ex-US Clinical Trials: Tribulations, Preparations, and Expectations" - new From The Trenches blog from @ArthurTzianabos, CEO of Lifordi and seasoned drug developer, on the considerations for ex-US clinical trials... https://t.co/IZ6RUVKrZj
Lung (not head & neck) was the right choice for $IMMP to pursue in phase 3, but it still failed. Stock -89%. Sory via @APEXONCO -> https://t.co/Y1ilGP5Aqq
PsiQuantum has signed a research agreement with Japan's National Cancer Center to explore utility‑scale quantum computing for oncology drug discovery. The partnership will focus on developing fault‑tolerant quantum algorithms and clinically relevant applications using PsiQuantum's Construct platform. It also brings...
#GSK Arexvy RSV Vaccine approved by US FDA with a wider indication for Adults aged 18 to 49 who are at increased risk of Lower Respiratory Tract Disease caused by RSV.
Pilatus Biosciences has begun dosing the first patient in a Phase I, open‑label trial of PLT012, its first‑in‑class anti‑CD36 monoclonal antibody, at Next Oncology in Houston. The FDA recently issued IND clearance along with orphan‑drug status for hepatocellular carcinoma and fast‑track...
Artificial intelligence is reshaping large‑scale pharmaceutical manufacturing by automating process design, real‑time control, and supply‑chain management. Machine‑learning models can simulate optimal temperature, pressure and reactant levels, accelerating scale‑up and reducing waste. AI‑driven advanced process control continuously adjusts bioreactor conditions, while...
A recent KFF poll shows 41% of Americans believe the Trump administration’s policies will lower prescription drug costs, outpacing awareness of the Biden‑era Medicare price‑negotiation law. Support is sharply partisan—79% of Republicans versus 11% of Democrats share this view. Only...
Janita Good, a Fieldfisher partner with a D.Phil. in biochemistry, offers biotech leaders a roadmap for maximizing partnership and licensing value. She emphasizes initiating pharma discussions early, embedding commercialization plans into R&D, and aligning fundraising expectations with realistic exit timelines....
PRISM BioLab has teamed with Receptor.AI to build an AI‑driven, physics‑guided platform for discovering orally available small molecules that target intracellular protein‑protein interactions, membrane proteins, and complex receptor systems. The collaboration fuses PRISM’s PepMetics technology—3‑dimensional scaffolds that mimic α‑helix and...
On 11 December 2025 the EMA’s CHMP issued a positive opinion to extend Uplizna’s (inebilizumab) marketing authorisation to three additional autoimmune conditions: neuromyelitis optica spectrum disorder (NMOSD), immunoglobulin G4‑related disease (IgG4‑RD) and generalized myasthenia gravis (gMG). The recommendation designates Uplizna as monotherapy for...
Ultragenyx announced Phase 3 Enh3ance data for its DTX301 AAV8 gene therapy targeting ornithine transcarbamylase (OTC) deficiency. At week 36, the therapy lowered 24‑hour plasma ammonia by 18% versus placebo and kept levels in the normal range, even as scavenger drug use...
Natera announced the commercial launch of Zenith Genomics, a next‑generation whole‑genome sequencing (WGS) assay aimed at diagnosing rare and ultra‑rare diseases in the United States. The platform pairs standard WGS with long‑read sequencing confirmation to capture complex genomic features such...
At the inaugural BIO BIG Summit, a Washington Policy Brief panel dissected the shifting U.S. healthcare landscape for biotech. The discussion highlighted progress on reauthorizing the Prescription Drug User Fee Act (PDUFA) and emerging pharmacy benefit manager (PBM) reforms, while...
The NRG‑LU005 phase III trial evaluated atezolizumab combined with concurrent chemoradiation in patients with limited‑stage small‑cell lung cancer (SCLC). Adding the immunotherapy did not improve overall or progression‑free survival, with median overall survival of 31.1 months versus 36.1 months for...
Solid Biosciences reported Phase I/II data for its DMD gene therapy SGT‑003, showing higher microdystrophin expression and a greater proportion of positive fibers than Sarepta’s Elevidys. The results strengthen SGT‑003’s case for accelerated FDA approval, but the pathway is clouded by...
Scientists have identified sulthiame, an old epilepsy drug, as a promising treatment for moderate‑to‑severe sleep apnea. In a German trial of 298 patients, higher doses cut breathing pauses by nearly 50% and boosted overnight oxygen levels. The findings, published in...
ACADIA Pharmaceuticals reported adjusted 2025 revenue of $1.08 billion, up 14% year‑over‑year, driven by strong performance of NUPLAZID and DAYBUE. NUPLAZID net sales reached $692 million with 15% growth, while DAYBUE posted $391 million, a 12% increase, aided by the newly approved DAYBUE...
![[Therapeutics] Pyruvate Kinase Activators in Hereditary Haemolytic Anaemias: Current Evidence and Clinical Potential](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/8c90dd45d33f73eeff78aead96a79a6a.webp)
Hereditary hemolytic anemias affect millions worldwide and have few disease‑modifying options. Oral pyruvate kinase activators, especially mitapivat, increase glycolytic ATP production, correcting a common metabolic defect in red cells. Clinical trials have shown efficacy in pyruvate kinase deficiency, sickle cell...
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...
Tonix Pharmaceuticals announced FDA approval and U.S. launch of TONMYA, the first new fibromyalgia drug in over 15 years, in August 2025 with commercial availability beginning November 17, 2025. The company reported fourth‑quarter product revenue of $5.4 million and full‑year revenue...
The FDA has asked manufacturers to strip the suicidal ideation and behavior warning from the labeling of three GLP‑1 receptor agonists—Saxenda, Wegovy and Zepbound—used for weight loss. A comprehensive safety review, including a meta‑analysis of 91 placebo‑controlled trials involving 107,910...
The FDA is adding an earlier MRI requirement before the third Leqembi infusion to catch amyloid‑related imaging abnormalities with edema (ARIA‑E) sooner. An analysis of pharmacovigilance data revealed 101 serious ARIA‑E cases, including six deaths, many occurring before the fifth...
A head‑to‑head trial (COBRRA) of 2,700 acute VTE patients found apixaban significantly lowers bleeding risk compared with rivaroxaban, while maintaining similar efficacy in preventing recurrent clots. Patients received standard dosing regimens for three months, with apixaban showing a 3.3% composite...
Doing some reading ahead of the next wave of $WVE INHBE "body composition" 🙄 data, smirking at the Jefferies analyst set up, which basically frames 6-month data as a win-win-win for the stock price. His "bear case" "Bear case would still...
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...
Intech‑Hanbon has unveiled a new line of advanced tangential flow filtration (TFF) systems aimed at biopharmaceutical, biotech and life‑science manufacturers. The platforms deliver high filtration efficiency, low shear stress and precise pressure control, while supporting a broad spectrum of applications...
Another testimony of Eli Lilly’s increasing dominance in the BioTech and Pharma sector - compared to other Big Pharma companies - such as $PFE $JNJ $NVS $NVO $BMY $ABBV $MRK $SNY and others, can be seen in this excellent @NatRevDrugDisc...
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...
The FDA’s Electronic Registration and Listing Compliance Program (eDRLS) safeguards the accuracy of drug registration and listing data that underpin inspections, electronic prescribing, and reimbursement systems. While automated validation rules catch many errors, the program also employs manual surveillance to...
🆕 @ScienceMagazine An exciting new approach vs pancreatic cancer: "cancer interception" Considered ubiquitous, the pancreas in healthy adults has hundreds of PanINs of microscopic premalignant cancer that can be regressed in the experimental model https://t.co/erVtthSMjS https://t.co/2xHIYdIPrx
The FDA has released an online searchable table of 249 pharmaceutical quality‑related guidances, MAPPs, and compliance programs. Recent entries include the Active Pharmaceutical Ingredient (API) Process Inspection compliance program (Sept 2025) and guidance on outsourcing facility inspections (Jan 2025). Topics span drug...
Researchers at UCSF discovered the Src kinase, traditionally an intracellular signaling protein, displayed on the outer membrane of malignant cells. The finding, published in Science, showed surface Src was absent from healthy donor tissue, suggesting a tumor‑specific marker. This unexpected...
Phase I trials have evolved from simple safety checks into data‑rich, adaptive studies that integrate biomarker strategies, exposure‑response analyses, and formulation optimization. Regulators now expect sponsors to justify Phase II dosing decisions, making early pharmacodynamic and target‑engagement data essential. Emerging biotechs face...
