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
Six biotech firms are pushing next‑generation prostate‑cancer therapies through late‑stage trials and financing milestones. Candel Therapeutics secured a $100 million royalty deal to fund aglatimagene’s launch, while Coherus Oncology closed a $50.1 million offering and partnered with J&J for a CCR8 antibody study. Monte Rosa Therapeutics reported 100 % PSA response in an AR‑mutant cohort, Telix’s TLX591‑Tx met primary safety goals in phase 3 part 1, and Vir Biotechnology raised $150 million and teamed with Astellas on a dual‑masked T‑cell engager. The global prostate‑cancer therapeutics market is projected to reach $21.2 billion by 2030, underscoring the commercial potential of these advances.
Almirall reported that lebrikizumab met both co‑primary endpoints in the Phase 3 ADorable‑1 trial, with 63% of pediatric participants achieving EASI‑75 and 44% attaining clear or almost clear skin (IGA 0/1) after 16 weeks. Key secondary outcomes showed significant itch reduction, higher...
Recent FDA approvals of resmetirom and semaglutide have shifted MASH care from a treatment‑void to a therapeutic reality, prompting sponsors to redesign trial endpoints and enrollment strategies. Non‑invasive diagnostic tools are emerging as potential primary endpoints, reducing reliance on liver...
Researchers at the Cleveland Clinic pursued fragment‑based inhibitors of SARS‑CoV‑2 NSP14, a viral exonuclease essential for replication and immune evasion. Initial crystal‑guided merges appeared active in a biochemical assay, prompting optimism about fragment linking. Subsequent resynthesis and rigorous purification revealed...
Drug Hunter’s online curriculum includes a Module 2 Quiz that tests learners on target identification and validation concepts. The quiz is part of a broader course designed for drug discovery professionals and requires full platform access via subscription or sign‑in. It...
Elevara Medicines has dosed the first patient in its phase 2b START‑SYNERGY trial, testing the oral CDK4/6 inhibitor ELV001 in rheumatoid arthritis patients who have failed methotrexate and TNF inhibitors. The randomized, placebo‑controlled study will enroll about 180 participants across nine...
1/6 Excited to join @START_Global this week in Switzerland for a fireside chat on the psychedelic medicine opportunity. As co-founder and former CEO of one of the first psychedelics-focused biotechs, I’ve had a front-row seat to this field’s move from stigma...
Cir‑Code Bio‑med, a Chinese biotech focused on circular RNA medicines, has secured an Investigational New Drug (IND) approval from the U.S. Food and Drug Administration to commence its first human trial. The therapy targets a rare genetic disorder using a...
Structure Therapeutics announced that its daily oral GLP‑1 obesity pill produced an average 16% body‑weight reduction versus placebo after 44 weeks in a Phase 2 trial. The result outperforms Eli Lilly’s orforglipron, which showed about 11% loss over 72 weeks, and rivals...
Structure Therapeutics released Phase 2 data for its oral GLP‑1 agonist, positioning the candidate as a next‑generation alternative to injectable therapies from Eli Lilly and Novo Nordisk. The trial demonstrated a mean 30% reduction in HbA1c and 70% of participants achieving target...
The Atlantic outlines a surge in off‑label and experimental peptide use, noting that Vyleesi—approved for women’s hypoactive sexual desire disorder—is being bought by men through “research use only” listings and online pharmacies. Compounding pharmacies and telehealth firms now market customized...
The FDA is accelerating the shift of prescription medicines to over‑the‑counter status, highlighted by Commissioner Marty Makary’s call for broader OTC availability and the introduction of the Additional Condition for Non‑Prescription Use (ACNU) framework. Former FDA counsel Heidi Gertner stresses...
The blog recaps the 2026 New Antibacterial Discovery and Development conference in Tuscany, where researchers presented emerging strategies against antimicrobial resistance (AMR). Dr. Quave highlighted her lab’s plant‑derived natural products targeting Gardnerella vaginalis, a key cause of bacterial vaginosis. The...
Pete Pharma has entered a strategic partnership with Atrium24 Technologies, designating Pete Pharma as the primary 3D‑printing provider within Atrium24’s group‑ purchasing organization. The deal gives Atrium24‑member independent pharmacies preferred pricing on Pete Pharma’s FABRx 3D‑printing platform, enabling on‑site production...
RevnaBio received triple accreditation from the American Association for Laboratory Accreditation (A2LA) for ISO 15189, ISO 20387 and ISO/IEC 17043, validating its medical laboratory, biobanking and proficiency‑testing operations. The certifications boost diagnostic quality, enable local molecular testing, and provide a trusted platform for...
Biophytis has broadened its alliance with AI specialist LynxKite to fast‑track longevity drug discovery, launching the MASSIVE project that seeks Mas receptor activators for sarcopenia. Backed by Enterprise Singapore, the partnership will augment Biophytis’ computational platform with LynxKite’s chemoinformatics, advanced...
Bayer disclosed results from the pivotal Phase III FIND‑CKD trial evaluating Kerendia (finerenone) in more than 1,500 adults with non‑diabetic chronic kidney disease. Patients received 10 mg or 20 mg of Kerendia alongside standard of care and were compared with placebo. The study...
Bayer announced that Kerendia® (finerenone) met its primary endpoint in the Phase III FIND‑CKD trial, showing a statistically significant slowing of eGFR decline versus placebo in non‑diabetic chronic kidney disease patients. The study enrolled over 1,500 participants and represents the fifth...
Johnson & Johnson announced encouraging Phase I data for its intravesical drug‑releasing system Erda‑iDRS in non‑muscle‑invasive bladder cancer (NMIBC) with FGFR alterations. The trial met its primary safety endpoint and delivered an 89% complete response rate in intermediate‑risk patients, with responses...
Roche has secured CE Mark approval for its Elecsys ApoE4 in‑vitro diagnostic immunoassay, a blood‑based test that detects the ApoE4 gene variant linked to Alzheimer’s disease. In a validation study of 607 patients with cognitive complaints, the assay achieved 100%...
A model that predicts cellular age from Cell Painting microscopy images alone. No DNA methylation needed. The model captures morphological aging hallmarks across nuclear and cytoplasmic compartments, correlates with chronological age AND epigenetic clocks. The promise: screen for rejuvenation compounds...
Sarepta tests new Elevidys safeguards; Sana advances diabetes cell therapy https://t.co/hkixuANCCV $SRPT $SANA $INO $BAYRY #biotech
Viatris agreed to dismiss the Henrietta Lacks estate lawsuit with prejudice, ending claims that the company used HeLa cells without permission in its herpes drug Denavir and depression treatment Mylan‑Mirtazapine. The settlement details were kept confidential. This resolution follows similar...
Pretty impressive data from $gpcr, which remains my favorite ticker. Structure's obesity pill shows weight loss promise in Phase 2 trial https://t.co/T0JU2UuftY
#AZN Imfinzi (durvalumab) combined with 'standard-of-care' Chemotherapy has been approved in the EU for treating Adults with forms of Gastric and Gastroesophageal Junction Cancers, following the MATTERHORN Phase III trial.
Obicetrapib, a next‑generation CETP inhibitor, has demonstrated potent LDL‑C, apoB, and Lp(a) reductions in a large phase III lipid trial. A pre‑specified biomarker sub‑study reported a marked attenuation of p‑tau217 progression, especially among APOE4/4 carriers, hinting at a potential Alzheimer’s‑related benefit....
In this special episode, host Peter Atiyah dives deep into obicetrapib, a CETP inhibitor that lowers LDL‑C and ApoB while raising HDL‑C, and examines its renewed promise in cardiovascular disease and potential Alzheimer’s prevention, especially for APOE‑E4 carriers. He outlines...
REGENXBIO's gene therapy RGX‑121 for Hunter syndrome received an FDA Complete Response Letter, with the agency flagging patient‑eligibility definitions, natural‑history control comparability, and the surrogate endpoint as problematic. The rejection redirects focus to Denali Therapeutics, whose enzyme‑replacement candidate tividenofusp alfa...
The dog *also* got standard-of-care treatment. Who knows what worked? Randomize some getting one-off custom vaccines but whole group gets standard of care. How ALL modern clinical trials work. If true, put big money to improve. Otherwise don’t push snake oil on...
Samsung Bioepis and its sister firm Epis NexLab have signed a research collaboration and exclusive license agreement with G2GBIO to develop a long‑acting semaglutide formulation using G2GBIO’s proprietary microsphere technology. The deal grants Samsung Bioepis full rights to the semaglutide...
A CRISPR‑Cas9 screen revealed that riboflavin (vitamin B2) sustains the ferroptosis suppressor protein FSP1, shielding cancer cells from iron‑driven lipid peroxidation. Depleting vitamin B2 destabilizes FSP1 and renders tumor cells highly susceptible to ferroptosis. The researchers demonstrated that roseoflavin, a bacterial analog...
At SXSW, Mark Cuban and eMed CEO Linda Yaccarino announced a partnership that will route employer‑sponsored GLP‑1 weight‑loss prescriptions through Cuban’s Cost Plus Drugs. Yaccarino highlighted eMed’s 90 % one‑year retention rate, contrasting it with the 50‑60 % churn seen at direct‑to‑consumer players...
Solve Aging: Biophytis and LynxKite are expanding their alliance to use graph-based AI to speed discovery of new longevity drugs, aiming to map complex aging biology and surface novel targets for age-related disease. The “AI x longevity” stack is quietly...
Summit Therapeutics reported a cash balance of $713 million and zero debt at year‑end, while GAAP operating expenses fell to $225 million. The FDA accepted its Biologics License Application for ivonesumab in EGFR‑mutant non‑small cell lung cancer, setting a PDUFA action date...
Researchers published a longitudinal study showing that people living with HIV who switch from daily oral antiretrovirals to the long‑acting injectable combo cabotegravir‑rilpivirine experience an early, transient rise in monocyte activation followed by a sustained decline below baseline levels. Flow...
The only sustainable path to development of longevity therapeutics is to get the potential longevity drug approved for a disease, then go for indication expansion and study it in the context of aging at different doses and regimens. To be...
Has anyone built full open source tooling for Hermes Agent @NousResearch for Bioinformatics (mRNA vaccine design is the target)? It'll need new agent skills too. Would you use it if I built it? I have a few hours left to...
The documentary "The Pink Pill" chronicles the decade‑long fight to bring Addyi, the first FDA‑approved drug for women’s low libido, to market. Founder Cindy Eckert bought the rights after a major pharma abandoned the project, launched a 13,000‑patient trial, and...
AI's capabilities have outpaced the FDA's capabilities to regulate it There are literal cures that can be designed and synthesized but have no way to reach the market due to regulatory/ethics blocks People will, like with Peptides, start dosing themselves & their...
Please @X I make low cost vaccines to help humanity and debunk pseudoscience. This is unacceptable
During a congressional hearing, neurologists warned that rare disease patients are dying while therapies linger in FDA review. The FDA recently rejected the SCA drug troriluzole, demanding more statistical certainty despite trial data showing over 50% fall‑risk reduction. Congress has...
GLP-1 drugs for extending healthspan? Intriguing but we're a long way off from evidence Discussed at length in Super Agers as a candidate drug beyond lifestyle + factors @TheEconomist gift link https://t.co/A2dpfcnApF https://t.co/3gY8iACeGs
From a tiny idea in our Lux office to deal after deal... ...@VariantBio finding outlier people, with outlier traits, in outlier parts of the world to make outlier drugs. organ by organ ... https://t.co/I5fK8nzZb4
Clinical trials have long relied on site‑based recruitment, leaving under‑ and misdiagnosed patients underrepresented. Recent studies show digital outreach can identify symptomatic individuals who never enter traditional healthcare pathways, dramatically expanding the eligible pool. Digital campaigns have cut cost per...
How do you break psychedelic molecules into parts, like cars in a chop shop, and build new ones? A look into non-psychedelic psychedelic medicine by Clayton Dalton, an ER doc and Ferriss-UC Berkeley Psychedelic Journalism Fellow @ucbsoj & @SciPsychedelics https://t.co/b7R4aTNdyf
The Enemy isn't Big Pharma. It's the FDA and Regulators who won't allow you to test experimental therapies on yourself (or your dog). Even if it's life or death.
Lupin is pivoting toward proprietary new chemical entities (NCEs) in India, aiming to launch multiple innovative products by next year. The company plans to focus on cardiac, diabetes, respiratory and oncology molecules, leveraging lower development costs and faster time‑to‑market. AI...
The man engineering smarter, healthier babies before they're born: Jonathan Anomaly. This philosopher-turned-biotech founder says the future of your child's health starts at the embryo. Here are 9 things you need to know about embryo selection: https://t.co/XVQF5le4bE
Eli Lilly has launched Employer Connect, a platform that lets large employers purchase its weight‑loss injection Zepbound at a discounted $449 price, aiming to close the coverage gap that leaves over half of workers without obesity drug benefits. The program bypasses...
Researchers analyzing over 30 million adverse‑event reports found that patients using Wegovy, the high‑dose semaglutide injection for obesity, have about five times the odds of developing ischemic optic neuropathy (ION) compared with those on Ozempic, the lower‑dose diabetes formulation. The association...
