Know What's Happening in Pharma
Know What's Happening in Pharma
Eli Lilly’s oral GLP‑1 pill and triple‑agonist trial reshape obesity drug race
The FDA approved Lilly’s oral GLP‑1 weight‑loss tablet Foundayo (orforglipron), which can be taken without food or water restrictions. In a Phase 3 trial, Lilly’s experimental injectable triple‑hormone agonist retatrutide achieved a 28.7% average body‑weight reduction, outpacing Novo Nordisk’s next‑generation candidates.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma
The pharmaceutical industry faces commercial risk as it prepares for the 2033 transition to a uniform 12‑digit NDC format, with experts warning that thousands of zero‑prefix collisions could trigger PBM claim rejections, disrupt patient‑hub enrollments, and corrupt market‑intelligence data. Simultaneously, the FDA granted accelerated approval to Avlayah, the first therapy designed to cross the blood‑brain barrier for Hunter syndrome, citing a 91% reduction in a key disease biomarker. The approval is conditional, pending results from a confirmatory trial, and represents the first new treatment for the rare disorder in nearly two decades. Companies that fail to update their systems early risk significant revenue leakage, while Avlayah opens a new therapeutic frontier for rare‑disease biotech firms.
MIT researchers discovered that three widely used anesthetics—propofol, ketamine and dexmedetomidine—produce an identical destabilization of brain dynamics, measurable as a loss of dynamic stability. Using EEG‑based perturbation analysis, they showed that despite distinct molecular targets, each drug pushes the brain...
HaemaLogiX, an Australian clinical‑stage biotech, is developing precision immunotherapies for multiple myeloma by targeting novel antigens KMA and LMA that appear only on malignant plasma cells. Peer‑reviewed research validates these targets, allowing the company to spare healthy plasma cells and...
Novartis announced a definitive agreement to acquire Excellergy, a private biotech developing next‑generation anti‑IgE therapies. The deal values Excellergy at up to $2 billion in upfront and milestone payments, with closing targeted for the second half of 2026. Exl‑111, Excellergy’s lead...
Insilico Medicine and Tenacia Biotechnology have expanded their AI‑driven partnership to create a second central‑nervous‑system (CNS) candidate, now advancing to the preclinical stage. The original March 2025 collaboration combined Insilico’s Pharma.AI platform with Tenacia’s expertise in blood‑brain‑barrier‑permeable small‑molecule inhibitors. Under...
Abcuro presented Phase II/III MUSCLE trial data for ulviprubart (ABC008) in inclusion body myositis (IBM) at the GCOM 2026 meeting. The study enrolled 272 patients who received either 0.5 mg/kg, 2 mg/kg, or placebo. Across the entire cohort the drug showed only...
AstraZeneca’s experimental antibody tozorakimab cut moderate‑to‑severe COPD flare‑ups in two phase‑III trials, meeting its primary endpoint. The drug showed a statistically meaningful reduction across both current and former smokers, including those with varying lung damage. The positive data lifted AstraZeneca...
The coolest meeting I had this week with was Paul, who used ChatGPT and other LLMs to create an mRNA vaccine protocol to save his dog Rosie. It is amazing story. "The chat bots empowered me as an individual to act...
Senior HHS official and Medicare administrator Chris Klomp warned that competition with China’s rapidly advancing biotech sector is “a war,” signaling heightened urgency in U.S. policy circles. He highlighted gaps in domestic research funding, manufacturing capacity, and talent pipelines that...
Now imagine every cancer patient had access to this technology (they should) "A specifically designed mRNA vaccine - a precision-guided weapon to strike the cancer, developed with a pipeline designed by ChatGPT o1, implemented for candidate development by Gemini Pro 2,...
Efficacy of Alpha-Lipoic Acid in Patients With Ischemic Heart Failure: A Double-Blind, Randomized, Placebo-Controlled Study | @JACCJournals https://t.co/nW9SbwEDfy https://t.co/qsemjmMb9k
Innovent Biologics reported its first annual profit, propelled by strong demand for its weight‑loss medication. The Hong Kong‑listed company’s shares surged as much as 7.75%, the biggest jump since late October. The profit milestone underscores the rapid growth of China’s...
GLP-1 and the cardiovascular system "This Review summarizes the effects of GLP-1 and GLP-1RAs in the CV system..." https://t.co/Sy7Jjb96WD https://t.co/v3T01fIcOs
Vytrus Biotech SA announced full‑year earnings of €2.89 million, up 183% from the prior year, while revenue surged 66% to €8.37 million. The results highlight robust market demand for its pipeline and signal momentum for European biotech firms.
The FDA has granted approval to Rocket Pharma's gene therapy Kresladi for severe leukocyte adhesion deficiency type 1 (LAD‑1), an ultra‑rare immune disorder. The therapy was previously rejected in 2024 due to manufacturing concerns, but the agency cleared it after the...
GARM, operating as Longevity Advanced, announced the addition of the anti‑aging protein Klotho to its gene‑therapy portfolio at its Roatán clinic. The move broadens a suite that already includes Follistatin and VEGF, positioning the center as a hub for high‑end...
Scientists at Shenyang Pharmaceutical University have engineered eye drops using exosomes derived from pig semen, loaded with a carbon‑dot nanozyme, to breach the retinal barrier in mice. The formulation halted retinoblastoma tumor growth and preserved normal vision over a 30‑day...
Agenus announced a March 31, 2026 stakeholder webcast to detail progress on its botensilimab and balstilimab (BOT + BAL) immunotherapy program. The company highlighted clinical durability across multiple tumor types, with roughly 1,200 patients treated with botensilimab and over 900 with balstilimab...
Mexico published the Rules of the Pharmaceutical Investment Promotion Committee on February 23, 2026, implementing a 2025 decree aimed at boosting pharmaceutical investment and domestic health‑supply production. The rules tie participation in certain public procurement procedures, such as direct awards...
Clarity Pharmaceuticals has signed a manufacturing supply agreement with Theragenics to scale up production of copper‑64, a radiometal used in its investigational prostate‑cancer tracer 64Cu‑SAR‑bisPSMA. The deal leverages Theragenics’ Atlanta‑area facility, which houses 14 cyclotrons capable of producing roughly 100...
Researchers at Helmholtz‑Zentrum Hereon and partners propose a payload‑driven approach to nucleic‑acid delivery, designing polymeric carriers that are chemically tuned to each DNA, RNA or mRNA payload. The strategy contrasts with the one‑size‑fits‑all lipid nanoparticles that dominate current vaccines and...
LeonaBio announced it has secured an exclusive global license for the Phase 3 lasofoxifene program, a novel selective estrogen receptor modulator aimed at ESR1‑mutated metastatic breast cancer, and raised $90 million in a private placement that could expand to $236 million with warrant...
Frank Harrell, a former FDA statistician, responded to recent JAMA commentary on the agency’s draft guidance promoting Bayesian methods for clinical trials. He highlighted that while the guidance is a step forward, FDA reviewers still rely on traditional frequentist approaches...
Jiangsu Hengrui Medicine announced 2025 results showing a 13% jump in revenue to RMB 31.63 billion ($4.4 billion) and a 21.8% rise in net profit to RMB 7.72 billion ($1.1 billion). The surge was powered by strong innovative‑drug sales and a landmark Hong Kong...
Neion Bio, fresh from stealth mode, signed its first co‑development and supply agreement with a major global pharmaceutical company to produce recombinant biologics using its egg‑based Raptor™ platform. The deal provides upfront and milestone payments plus profit‑sharing after commercialization, delivering...
The FDA issued guidance clarifying how compounding pharmacies may use bulk drug substances, also known as active pharmaceutical ingredients. Under section 503A, state‑licensed physicians and pharmacists can compound with bulk substances that meet USP/NF monographs, are components of FDA‑approved drugs, or...
@Ronalfa is speaking at @SynBioBeta in May and you probably already know who he is if you've been paying any attention to the AI x bio space. Ron spent years at @RecursionPharma as SVP of Research and acting CSO, helping build...
In this episode of Touching Base, the Gen editorial team discusses the latest advances in AI for life sciences, including NVIDIA’s GTC announcements on agentic AI, the deployment of 3,500 GPUs by Roche, and the emergence of open‑source autonomous agents...
The Trump administration failed to meet the 210‑day deadline to nominate a permanent CDC director, leaving the agency without confirmed leadership for most of the second term. Meanwhile, the FDA granted accelerated approval to Denali Therapeutics’ Avlayah, the first enzyme...
Pinnacle Medicines announced the closing of an oversubscribed $89 million Series B financing, co‑led by LAV and Foresite Capital. The biotech firm leverages a proprietary platform that blends physics‑based molecular simulations, AI‑driven design, and advanced peptide chemistry to create oral peptide therapeutics....
Most cancer drugs go after the same targets: EGFR. PD-L1. HER2. Not because they’re the best targets. Because they’re the only ones we’ve been able to see. RyboDyn Inc. is going after what’s been invisible. The San Diego team, led by Imad Ajjawi, PhD,...
The FDA’s upcoming user‑fee framework, dubbed "America First," seeks to tighten eligibility for the small‑business waiver, limiting it to U.S.‑based applicants. Industry groups argue the change politicizes fee policy and could disadvantage foreign‑owned biotech firms that rely on the waiver...
Researchers applied a genome‑wide variance decomposition across TCGA’s 60,656 genes and 33 cancer types to prioritize therapeutic antigens, moving beyond traditional mean‑expression screens. The analysis yielded 17 candidates that met functional dependency, safety, and immune‑cold criteria, with three highlighted: CRIPTO/TDGF1,...
A new Biotechnology Innovation Organization poll of 1,000 U.S. voters shows eight in ten would back leaders who lower drug costs by reforming pharmacy‑benefit managers, insurers, and the 340B program. Across party lines, 88% support passing PBM rebates directly to...
Valneva SE announced its participation in the 26th World Vaccine Congress in Washington, D.C., from March 31 to April 2, 2026. The company’s CEO, Thomas Lingelbach, and senior executives will present data on the chikungunya vaccine IXCHIQ® and join a...
Transgene (Euronext: TNG) will deliver a 30‑minute oral presentation on its individualized neoantigen therapeutic vaccine TG4050 at the World Vaccine Congress in Washington, D.C. on April 1, 2026. TG4050, built on the AI‑driven myvac® platform, targets patient‑specific tumor mutations. Phase 1 data in...
The GLP‑1 drug semaglutide has entered the Indian market as generic versions after its patent expired last week, driving monthly prices down from roughly $144 to $36. The steep discount makes the medication affordable for a broader segment of diabetics...
Scientists at Baylor College of Medicine identified a brain pathway that enables metformin to lower blood sugar, a discovery that could expand the drug’s use beyond diabetes and fuel new longevity protocols among biohackers.
Cellular senescence has become a focal point for longevity medicine, prompting a surge of senolytic and senomorphic drug development. Pioneering studies showed that clearing senescent cells can extend healthspan, leading biotech firms like Rubedo, SENISCA, Deciduous Therapeutics, and Arda Therapeutics...
A research team has created magnetic silk‑iron nanoparticles that can be steered with external magnetic fields to deliver therapeutics to otherwise inaccessible disease locations. The nanoplatform merges biocompatible silk fibroin with iron oxide, enabling magnetic control while maintaining safety, a...
The World Health Organization announced new policy guidance aimed at simplifying tuberculosis detection tests, signaling a major push for more accessible diagnostics. While the guidance outlines a framework for low‑cost, point‑of‑care testing, specific technical details were not disclosed in the...
One large psilocybin dose beat nicotine patches by 6x odds for smoking cessation. 82 otherwise-healthy cigarette smokers, 42 received a single high-dose 30mg/70kg psilocybin session, and 40 initiated an 8- to 10-week course of nicotine patch treatment. At 6 months; participants were 6x...
This week's @TheLancet cover and editorial about getting GLP-1 drugs available for the people who need them the most https://t.co/BU9NZdmdGo https://t.co/TWOIKvfuth
Kodiak Sciences announced that its experimental eye drug Zenkuda (tarcocimab tedromer) achieved positive topline results in the GLow2 Phase 3 trial for diabetic retinopathy, outperforming sham treatment. The study met its primary endpoint, delivering a statistically significant gain in visual acuity...
Discontinuation of maintenance therapy in multiple myeloma guided by multimodal measurable residual disease negativity (MRD2STOP) - @bdermanmd et al. @ajjakubowiak #ASCO24 Abstract 106 https://t.co/FBTY7SnCxK #NCT04108624 #mmsm #mmMRD
A Historic Turning Point: ODAC Unanimously Votes [4/12/24] in Favor of MRD Testing as an Early Endpoint in Myeloma Clinical Trials to Support Accelerated Approvals of New Treatments [Apr 18, 2024] @IMFmyeloma https://t.co/eDOgIrpVeR #mmMRD #mmsm #ctsm @FDAOncology https://t.co/ejA8KSoxOL
A “kazarian?” Is that like a Kardashian? What planet are these X trolls from? I develop low-cost often patent-free vaccines for global health and to help humanity. So far 100 million have benefited from access to our vaccine technologies developed...
Wave crashes on obesity drug update; Kodiak’s reboot pays dividends https://t.co/20H9OXVtIB $WVE - 54% $KOD + 58%
My article in today's issue of the Journal of the American Medical Association Health Forum, on FDA's new rare disease guidance, and how the agency can build on these policy steps to promote innovation for inherited disorders, authored with Maarika...
FDA clears Denali drug in ‘clear step’ for rare disease biotechs https://t.co/x9yL4nyPaj by Kristin Jensen $DNLI #biotech
