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
Nurix Therapeutics has launched NX-1607, the first orally bioavailable small‑molecule inhibitor of the immune regulator CBL‑B, into a Phase 1a/1b trial for advanced cancers. CBL‑B modulates activation of T, B and NK cells, and NX-1607 locks the protein in an inactive state, boosting T‑cell activity. Pre‑clinical models showed robust anti‑tumor effects and extended survival, and early clinical data indicate encouraging efficacy signals. The program was highlighted at the ACS Spring 2024 meeting.
Researchers at Johns Hopkins found a single dose of psilocybin dramatically increased smoking cessation rates compared with nicotine patches. In a randomized trial of 82 smokers, 17 of 41 participants who received psilocybin remained abstinent after six months versus four...
Scientists announced ETVAX, the first oral vaccine that targets enterotoxigenic *E. coli* (ETEC) in children, after a large‑scale trial in The Gambia. The study involved 4,936 infants aged six to 18 months and demonstrated a 48% reduction in moderate‑to‑severe ETEC...
Novo Nordisk received an FDA warning letter on March 5, 2026 for failing to report suspected side effects of its GLP‑1 medicines. The violations were uncovered during a 2025 inspection of the company’s facilities and were described as “serious.” The agency warned...
The Trump administration, led by Health Secretary Robert F. Kennedy Jr., plans to eliminate over a third of the CDC childhood vaccine schedule, targeting seven of eleven vaccines. The United States, which produces roughly 36% of global vaccine output, could...
Dubai is positioning itself as a long‑term hub for healthcare and life sciences through two complementary free‑zone clusters – Dubai Healthcare City (DHCC) and Dubai Science Park (DSP). DHCC is undergoing a $1.3 bn expansion that will create six tightly linked...
The FDA has defined the scope of pre‑submission facility meetings to curb manufacturing‑related complete response letters that delay drug approvals. It agreed to cover prior production‑site inspections, novel process elements and supply‑chain node strategies, while rejecting topics such as alternative...
Arizona's biotech sector is booming, with $3.7 billion invested over the past seven years and a growing pipeline of innovative companies. Nectero Therapeutics secured a $96 million Series D to develop a fast‑track, breakthrough‑designated endovascular treatment for abdominal aortic aneurysms, while Humabiologics obtained...
BioNTech co‑founders Ugur Sahin and Özlem Türeci will leave the company by year‑end to launch an unnamed mRNA‑focused venture, with BioNTech retaining a minority stake and licensing key technology. The split allows BioNTech to concentrate on its late‑stage cancer pipeline...
Deaminet 2026 showcased accelerating advances in base and prime editing, highlighted by rapid Addgene distribution growth and new mechanistic insights such as PE6d’s heightened processivity and ABE8e’s dimer‑driven efficiency. Novel off‑target detection platforms like beCasKAS and Inrich‑seq revealed far more unintended...
Sticking with the science. I find that really impressive. BioNTech founders Uğur Şahin and Özlem Türeci are leaving the biotech they founded to start a new one. Maybe by the time they have something for the clinic, the FDA will...
DNA Repair gene alterations and efficacy from gemcitabine and nab-paclitaxel with/without durvalumab and tremelimumab in metastatic pancreatic ductal adenocarcinoma https://t.co/v39bOreJwu
China is actively targeting ultra‑expensive gene therapies, challenging the notion that these treatments are immune to price competition. Recent regulatory approvals and centralized procurement initiatives have forced manufacturers to negotiate steep discounts on therapies that once commanded multi‑million‑dollar price tags....
Correction: I took down a post about Thursday's #VRBPAC meeting because it contained an error. #FDA has named 6 temporary voting members (not 2), which will bring the number for the meeting to 9. The temps are all former members....
$CAPR resubmission of the deramiocel application accepted by the FDA. New decision date: Aug. 22. https://t.co/mWSyyXg8di
The U.S. Food and Drug Administration has approved the generic form of leucovorin, also known as folinic acid, for treating cerebral folate deficiency (CFD). The decision widens the drug’s label despite the absence of new clinical trial data, relying on...
How CRISPR works (in nature) and how it’s being leveraged as a tool for gene editing to cure and prevent diseases. Dr Alex Marson MD PhD of UCSF on the Huberman Lab podcast out now. https://t.co/fiL48JHxSL
$GSK is ostensibly the "sponsor" that won the approval but is once again saying it won't produce the drug.
Credence Research forecasts the global cell and gene therapy manufacturing market to surge from $19.3 billion in 2024 to $146.2 billion by 2032. The compound annual growth rate is estimated at 28.8%, propelled by rising demand for advanced therapies, expanding commercialization, and...
BIOTECanada welcomes the launch of the National Research Council of Canada’s Biomedical Countermeasures Initiative, a key component of the federal Defence Industrial Strategy. The program is designed to strengthen Canada’s domestic capacity to develop and manufacture diagnostics, vaccines, and therapeutics...
Stylus Medicine entered the cell‑therapy arena in May 2025 with an in‑vivo CAR‑T platform that delivers a lipid nanoparticle‑encapsulated recombinase to engineer T cells inside patients. The move comes after major pharma acquisitions—BMS buying Orbital Therapeutics for $1.5 billion and Gilead...
Drug pricing is increasingly viewed as a reputation issue, not just a financial decision. Carreen Winters of MikeWorldWide argues that pricing choices shape public perception of a company's values, as illustrated by Novo Nordisk’s 50% cut to GLP‑1 drug prices....
French biotech Ipsen announced an immediate, global voluntary withdrawal of its oncology drug tazemetostat (Tazverik) after safety signals in the SYMPHONY‑1 confirmatory trial indicated a rise in secondary hematologic malignancies. The company is coordinating with the FDA to manage the...
India’s active pharmaceutical ingredient (API) market posted a landmark trade balance in FY25, with exports climbing to roughly Rs 41.5 billion while imports lagged at Rs 39.2 billion. The surplus marks the first time the sector has out‑exported its imports, signaling a maturing domestic...
Samsung Biologics and U.S. pharma leader Eli Lilly have signed an agreement to launch a biotech incubator in Incheon’s Songdo district. The facility will host Lilly’s Gateway Labs platform, offering lab space, equipment, funding and R&D collaboration to emerging biotech firms....
Dyne Therapeutics reported that its exon‑skipping candidate z‑rostudirsen sustained respiratory and cardiac benefits through 24 months in the Phase 1/2 DELIVER study for Duchenne muscular dystrophy. The therapy maintained forced vital capacity, circumferential strain and left‑ventricular ejection fraction improvements compared with...
Junshi Biosciences has received acceptance from China’s National Medical Products Administration for its subcutaneous toripalimab injection (JS001sc), covering 12 cancer indications. The filing marks the first domestic anti‑PD‑1 monoclonal antibody in a subcutaneous formulation to reach the marketing application stage....
Ascletis Pharma reported positive topline data from a U.S. Phase II, 24‑week study of its ultra‑long‑acting subcutaneous depot GLP‑1R agonist ASC30. The once‑monthly formulation A1 produced a placebo‑adjusted mean weight loss of 7.5% at week 16 and sustained a 5.8%...
Uğur Şahin and Özlem Türeci announced they will leave BioNTech by the end of 2026 to launch a new company focused on next‑generation mRNA technologies. BioNTech will concurrently narrow its portfolio, concentrating on late‑stage therapeutic candidates and its existing vaccine...
A new analysis shows the world’s largest pharmaceutical firms cut antimicrobial development by 35% over the past five years, dropping from 92 to 60 candidates. Only five of the 39 pipeline projects aimed at WHO priority pathogens include pediatric formulations...
The FDA has eliminated the longstanding requirement for two pivotal clinical trials, adopting a single‑trial default to speed drug approvals. The agency pairs this change with a mandate for more rigorous post‑market surveillance to catch safety signals after launch. Pharmaceutical...
NewcelX has entered a collaborative research agreement with Eledon Pharmaceuticals to advance its NCEL‑101 cell therapy for type‑1 diabetes. The partnership integrates NewcelX’s off‑the‑shelf islet replacement product with Eledon’s anti‑CD40L monoclonal antibody, tegoprubart, which has been used in over 100...
Researchers at Texas Children’s Duncan Neurological Research Institute and Baylor College of Medicine demonstrated that skipping exon e2 of the MECP2 gene boosts MeCP2 protein production by 50‑60%, rescuing neuronal function in Rett syndrome mouse models and patient‑derived cells. The study...
The FDA released draft guidance encouraging the use of Bayesian statistics in drug and biologic clinical trials, aiming to shorten development timelines and lower costs. By allowing external data—known as priors—to be incorporated, the approach promises more efficient, adaptive studies,...
Following the FDA’s recent shift to require only one pivotal trial for new drug applications, sponsors now face heightened pressure to generate robust efficacy and safety data. Regulators expect a single, bullet‑proof study rather than two less conclusive trials, mirroring...
Rapport Therapeutics has granted Tenacia Biotechnology exclusive rights to develop and commercialize its TARPγ8‑specific AMPA receptor negative allosteric modulator RAP‑219 in Greater China, covering indications such as focal onset seizures and bipolar mania. The agreement provides Rapport with a $20 million...
Researchers at Icahn School of Medicine created the first sex‑specific atlas of GLP‑1 expression in the mouse brain using RNAscope, mapping the peptide across 25 nuclei. The atlas shows pronounced differences between females and males, especially in hindbrain nuclei of...
A nonprofit, the API Innovation Center (APIIC), is tackling the capital barrier that prevents U.S. drug makers from adopting continuous manufacturing. By pooling state, federal, philanthropic and private funds, APIIC installs equipment at manufacturers at no upfront cost, de‑risking the...
Merck released phase III data from its Litespark‑011 trial, showing a potent HIF‑2α inhibitor that could reshape renal‑cell carcinoma therapy. Researchers at INSERM uncovered hypothalamic tanycytes as a primary mechanism for clearing pathological tau, opening new avenues for Alzheimer’s treatment....
PCSK9 in Vascular Aging and Age-Related Diseases Comprehensive summary of PCSK9's regulatory functions in vascular aging, highlighting potential therapeutic targets for combating age-related cardiovascular diseases. https://t.co/uo3PaSJLCb https://t.co/w8UZgIduPV
BioNTech’s Q4 2024 earnings call highlighted a strategic pivot toward oncology, emphasizing the advancement of its bispecific antibody BNT327 and the recent acquisition of Biotheus to secure Chinese manufacturing and development capabilities. The company reported positive Phase 2 data for...
ADC Therapeutics reported Q4 2025 product revenue of $15.8 million, down from $18 million a year earlier, while GAAP net loss narrowed to $41 million ($0.30 per share). Non‑GAAP operating expenses fell 12.1% to $45 million, driven by lower R&D spend. A $60 million private...
Biogen reported full‑year 2025 non‑GAAP diluted EPS of $15.28 and $9.9 billion in revenue, a modest 2% YoY increase. Growth‑product sales surged 19% to $3.3 billion, driven by VUMERITY, SKYCLARIS, ZERZUVE and CALSADI, while legacy MS revenues are projected to fall mid‑teens...
Indiana University researchers linked clonal hematopoiesis of indeterminate potential (CHIP) to heightened severity of inflammatory bowel disease. Analysis of UK Biobank and All of Us data showed women with DNMT3A mutations and younger individuals with TET2 mutations face higher Crohn’s...
An international team genetically deleted the ENA1 gene from Saccharomyces boulardii, a common probiotic yeast. In immunosuppressed mice, the ENA1‑deficient strain showed no mortality, raising survival from 30‑40% to 100% compared with wild‑type isolates. The edit also reduced osmotic stress...
Senate Democrats have pressed major pharmaceutical companies to clarify the secretive most‑favored‑nation (MFN) pricing agreements they struck with the Trump administration in December. Lawmakers are questioning how those deals align with the Centers for Medicare & Medicaid Services’ new GENEROUS...
Roche’s oral selective estrogen receptor degrader (SERD) giredestrant failed to meet its primary endpoint in a late‑stage Phase III trial, but the data revealed meaningful activity in specific patient subgroups, particularly those with ESR1 mutations. The miss prompted Roche to...
A new AI-driven workflow predicts the outcomes of asymmetric cross-coupling reactions, enabling chemists to efficiently identify optimal molecular structures while significantly reducing laboratory time and costs. drugdiscovery
Actually he has it a bit backwards, those vaccinated with IPV can still acquire poliovirus and shed virus, but it prevents the virus from entering the bloodstream + protects almost 100% paralytic polio, just like the Covid vaccine protects 80-90%...
Vertex $VRTX says its drug successfully reduced marker of kidney disease in late-stage trial https://t.co/TbEkcHVYBB via @Jasonmmast
