Today's Pharma Pulse
Neolaia unveils new CD38 inhibitor series in preclinical data
Neolaia announced the synthesis of a novel series of CD38 inhibitors, marking a significant step in its preclinical development pipeline. In parallel, Incyte disclosed its own next‑generation TYK2 and JAK1 inhibitor candidates, highlighting broader activity in kinase‑targeted drug discovery.
Also developing:
By the numbers: Asahi Kasei acquires German biotech Aicuris for $920.7M
FDA Accepts BLA for Gazyva/Gazyvaro for Systemic Lupus Erythematosus
Roche has received FDA acceptance of its supplemental Biologics License Application for obinutuzumab (Gazyva/Gazyvaro) as a treatment for systemic lupus erythematosus, with a regulatory decision expected by December 2026. The approval request is backed by the Phase III ALLEGORY trial, which demonstrated a 76.7% SRI‑4 response rate at week 52 versus 53.5% for placebo, along with reduced glucocorticoid use and fewer disease flares. Safety data were consistent with the drug’s known profile, showing no new signals. If approved, obinutuzumab would become the first anti‑CD20 therapy specifically indicated for SLE, potentially reshaping the standard of care.
Apeloa Pharmaceutical Q1 Profit Rises to RMB249 Million as Revenue Falls 10%
Apeloa Pharmaceutical reported a slight rise in first‑quarter profit to RMB249 million ($35 m) versus a year earlier, even as revenue dropped 10.4% to RMB2.45 billion ($343 m). The results underscore both growth in earnings per share and pressure on top‑line sales in China’s...
Redesigned Pre-Submission Meetings in GDUFA III: Benefits for ANDA Submission and Approval - 05/09/2024
On May 9, 2024, the FDA’s Office of Generic Drugs hosted a webinar on the redesigned pre‑submission meetings under GDUFA III. The session outlined new scope, procedural updates, and illustrated a hypothetical case to help sponsors prepare effective meeting requests. Speakers...
AI Can Streamline Drug Approval, Boost Biotech Valuations
That is an interesting idea. This doesn't seem catered to investors per se, but with two partners in the summer of 25, we explored building an “AI-powered AlphaSense for biopharma” ultimately concluding it was too much work to do as...
Complex Generics News
The FDA’s Center for Drug Evaluation and Research is intensifying its focus on complex generic drugs, which feature intricate active ingredients, formulations, or delivery systems. Recent milestones include the July 2023 approval of the first generic Vivitrol injectable and the March 2026...
Genflow and Acuitas Forge Fully Funded LNP Collaboration to Accelerate SIRT6 Gene Therapies
Genflow Biosciences announced a strategic, fully funded collaboration with Acuitas Therapeutics to apply Acuitas' lipid‑nanoparticle (LNP) delivery platform to Genflow's SIRT6 mRNA payload. The non‑dilutive agreement lets Genflow generate preclinical data without cash outlay, signaling strong external validation of its...
Xenon’s Azetukalner Clears Phase III Hurdle in Focal Epilepsy, Eyes FDA Filing
Xenon Pharmaceuticals reported on 19 April at the American Academy of Neurology that its KV7 potassium‑channel opener azetukalner met primary endpoints in the Phase III X‑TOLE2 trial for focal onset seizures. The study enrolled 380 adults, with 374 completing safety and...
The $500,000 Drug and the Cost of Modern Medicine
A 70‑year‑old man with no cardiac symptoms was diagnosed with wild‑type transthyretin amyloid cardiomyopathy after a routine coronary calcium scan revealed a score over 600. The diagnosis triggered a cascade of advanced imaging and a biopsy, leading to approval of...
Rapamycin Might Blunt Exercise Response in Humans
A double‑blind, placebo‑controlled trial gave 40 sedentary adults aged 65‑85 a weekly 6 mg dose of rapamycin alongside a 13‑week home‑based exercise program. Participants receiving rapamycin showed smaller gains in chair‑stand performance and trended worse on six‑minute walk and grip strength,...
Top 15 Specialty Pharmacies of 2025: Report
The Drug Channels report shows the specialty pharmacy market remains tightly concentrated, with PBM‑affiliated chains capturing roughly two‑thirds of dispensing revenue in 2025. Specialty drug spending rose 9.6% to $293.4 billion, while accredited locations topped 1,900, a 3% year‑over‑year increase. Hospital‑owned...
SMMT Spikes as Akeso OS Data Lands Plenary
Summit Thera. $SMMT Well… that answers that. SMMT shares initially popped (as much as ~15%) on today’s release of ASCO abstract titles with the unexpected news that the much-anticipated OS data from Akeso’s HARMONi-6 will be released in a Plenary slot...
Early Myocarditis Onset After Immunotherapy May Predict Treatment-Related Fatality
A new analysis of WHO VigiBase data presented at the AACR 2026 meeting shows that immune checkpoint inhibitor (ICI)‑induced myocarditis occurring within the first month of therapy dramatically increases the risk of death. Patients who develop myocarditis early are 59%...
Intralesional Nivolumab May Be Effective Against Precancerous Oral Lesions, Phase I Trial Results Indicate
A Phase I trial presented at AACR 2026 showed that injecting low‑dose nivolumab directly into precancerous oral lesions produced an 85% clinical response rate, with lesions shrinking an average of 60% and 41% achieving histologic downgrading. Patients received 10 mg or 20 mg...
CellCarta Eliminates 9-Hours-Per-Week Regulatory Bottleneck with RegASK’s AI-Driven Intelligence Platform
CellCarta, a global contract research organization, partnered with RegASK to overhaul its regulatory intelligence function. By deploying RegASK’s agentic AI platform, the CRO replaced a manual nine‑hour‑per‑week monitoring process with near‑real‑time updates. The new centralized hub automatically captures, validates and...
Agenus Names BAP Pharma as Exclusive Global Partner for BOT+BAL Access Programs
Agenus appointed BAP Pharma as its exclusive global partner to manage early‑access programs for the botensilimab‑balstilimab (BOT+BAL) immunotherapy combo. The collaboration will handle France’s government‑reimbursed Autorisation d’Accès Compassionnel (AAC) pathway and paid named‑patient programs in several other markets. Agenus has...
Charles River Highlights Effectiveness of VCGs in Toxicology
Charles River Laboratories published a retrospective analysis of 20 nonclinical toxicology studies that replaced traditional concurrent control groups with virtual control groups (VCGs). The review found 100% concordance in No Observed Adverse Effect Level (NOAEL) determinations and demonstrated up to...
Star Therapeutics Receives FDA Rare Pediatric Disease and Breakthrough Therapy Designations for VGA039 in Von Willebrand Disease Prophylaxis
Star Therapeutics announced that the FDA has granted both rare pediatric disease and Breakthrough Therapy designations to its lead candidate VGA039, a monoclonal antibody aimed at preventing bleeding in von Willebrand disease (VWD). The designations support the ongoing Phase 3 VIVID‑6 study,...
Simulations Plus Announces Collaboration with Lonza and U.S. FDA to Advance Predictive Frameworks for Complex Oral Drug Products
Simulations Plus announced a funded collaboration with CDMO Lonza and the U.S. FDA to create a mechanistic, predictive framework for amorphous solid dispersion (ASD) oral drugs. The partnership will combine Lonza's advanced in‑vitro dissolution testing with Simulations Plus' GastroPlus and...
Combining Cannabis with Opioids Offers No Added Pain Relief for Knee Arthritis Patients, Study Concludes
Researchers conducted a double‑blind, placebo‑controlled trial with 21 knee‑osteoarthritis patients to evaluate whether dronabinol, a synthetic THC, enhances the analgesic effect of hydromorphone. The study found that neither drug alone, nor their combination, produced meaningful acute pain relief during laboratory...
Tortugas Takes Neuro Deep Dive with $106M to Develop Eisai, Hansoh Programs
Tortugas, a Massachusetts‑based biotech, launched with $106 million in seed and Series A funding, securing clinical assets from Japan’s Eisai and China’s Hansoh Pharmaceutical. The capital will support two mid‑stage, oral small‑molecule trials targeting CNS disorders such as schizophrenia, tinnitus, and focal...
NIMBLE Trial Shows Efficacy of Cemdisiran in gMG: Tuan Vu, MD
The phase 3 NIMBLE trial evaluated cemdisiran, an RNA‑interference therapy, in patients with generalized myasthenia gravis (gMG). Over 26 weeks, cemdisiran monotherapy and its combination with pozelimab both achieved statistically significant improvements on the MG‑ADL scale versus placebo, and met the key...
Boehringer Targets AI-Driven Advances in Disease Research
Boehringer Ingelheim announced a £150 million (US$203 million) AI and machine‑learning accelerator in King’s Cross, London, expanding its global computational R&D network. The new hub, part of the UK Knowledge Quarter, will focus on disease biology, target identification and predictive modeling to...
BioAge Reports Positive Phase 1 Data for BGE-102
BioAge Labs announced Phase 1 results for BGE‑102, an oral, brain‑penetrant NLRP3 inhibitor, showing up to 86% reductions in high‑sensitivity C‑reactive protein (hsCRP) in obese participants. A 60 mg once‑daily regimen over 21 days achieved biomarker improvements comparable to the previously tested...
Research Shows Nicotine May Boost Cognition, Guard Against Parkinson's
The New York Times mentioned me in an article about nicotine. They're skeptical. Here's what they didn't include... Dr. Paul Newhouse at Vanderbilt ran controlled trials showing nicotine improved concentration and cognitive function in people with mild cognitive impairment. Peer-reviewed, published...
Labor Dept Demands PBM Payment Transparency Amid Legal Pushback
The Labor Department wants pharmacy benefit managers to disclose, for the first time, all the different ways they get paid. PBMs are warning this proposal is illegal. Supporters want the proposal to go further. The black box of Rx prices...
Re: Alzheimer’s Drugs Targeting Amyloid Do Not Produce Clinically Meaningful Effects, Concludes Cochrane Review
A recent Cochrane review concluded that amyloid‑targeting drugs for Alzheimer’s disease do not deliver clinically meaningful benefits, prompting disappointment among researchers, investors, and caregivers. In a BMJ rapid response, emeritus professors Elaine and Robert Perry argue that cholinergic therapy—available for...
Scientists Save Billions; CRISPR Cures Sickle Cell
You live in luxury. Without scientists, 6 billion of us would not be alive & the rest would live in hell. Remember that Great choices @brkthroughprize 🏆 Congrats to my colleague @Harvard, Stu Orkin, who won the prize for helping cure...
WGS Uncovers Clinically Relevant Findings Missed in MDS
Whole genome sequencing [WGS] to identify novel, clinically relevant findings missed by standard of care for Pts w/ myelodysplastic syndrome [MDS] - @AlexBataller etal @garciamanero @UTMDAnderson @TempusAI #ASCO26 abst 6570 Pst 363 https://t.co/t7aNW89eRL #MDSsm #leusm #cagenome
Merck, Eisai’s Keytruda Triplet Fails to Improve Survival in Kidney Cancer
Merck and Eisai announced that their Phase 3 LITESPARK‑012 trial of a three‑drug regimen—Keytruda, Welireg and Lenvima—failed to improve overall survival or progression‑free survival in first‑line renal cell carcinoma. The control arm received the approved Keytruda‑Lenvima doublet, which also did not...
First Trial Shows Best‑in‑Class NLRP3 Inhibitor Promise
Excited to complete our first clinical trial for BGE-102. Clinical data supporting best-in-class potential, built on new chemistry and a novel binding site targeting NLRP3 - a central driver of of chronic inflammation in aging. On to Phase 2!
AQUARIUS Registry Uncovers Biomarkers in Blood Cancers
AQUARIUS: A longitudinal multi-center molecular biomarker discovery registry for Pts w/ hematologic malignancies - @mtmdphd et al. @TempusAI & IFLI - Instit Follicular Lymphoma Innovation #ASCO26 abstr TPS7105, Poster 599a https://t.co/70G0v1P968 #NCT07154823 #lymsm #cagenome
SaNOtize - 727784 - 04/17/2026
The U.S. Food and Drug Administration issued a warning letter to SaNOtize, the maker of “NOWONDER™ Nasal Cleanser,” on April 17, 2026. The agency determined the product is an unapproved new drug because its labeling and website claim nitric‑oxide‑based immune benefits, violating sections 505(a)...
ASCO26 Unveils Pancreatic
#ASCO26 abstract titles were released today: $RVMD RASolute Ph3 study in pancreatic cancer will be presented during the plenary session. Also, Akeso $SMMT ivonescimab HARMONi-6 trial in lung cancer. China-only data.
BioAge's Inflammation Drug Shows Best‑in‑class Promise
BioAge says early data suggest ‘best-in-class’ potential for inflammation drug https://t.co/D2owlYxCRa by Kristin Jensen $BIOA $LLY $NMRA
Pro Numb Tattoo Numbing Spray, LLC - 722589 - 04/14/2026
The FDA issued a warning letter to Pro Numb Tattoo Numbing Spray, LLC for multiple Current Good Manufacturing Practice (CGMP) violations, including failure to test finished drug batches, inadequate raw‑material verification, and lack of stability data. The firm also marketed...
MRNA Brand Stigmatized by COVID Vaccine Backlash, Says Bhattacharya
That is literally a lie, as Bhattacharya wrote an op-ed trying to explain their move. He essentially was arguing that the "brand" of mRNA is tainted by public backlash to the COVID vaccine. https://t.co/5KXQJtCCxM https://t.co/HDQRvQK0ki
Promising Mufemilast Results Unveiled at DDW
Strong mufemilast data being presented at DDW this week for anyone still following the $PALI story. https://t.co/Oi4WI6R0iW
Mabwell’s T‑mab Subsidiary Clears First‑Time GMP Inspection in Jordan, Boosting Middle‑East Push
Mabwell announced that its subsidiary T‑mab successfully passed a Jordan Food and Drug Administration on‑site GMP inspection, earning a “Compliance” rating for its denosumab biosimilars. The milestone, the company’s first approval from a PIC/S member country, is expected to accelerate...
AI Accelerates Small Molecule Synthesis, Says Martin Burke
Small molecule synthesis made faster for the AI era: Listen to Martin Burke on The Long Run. Sponsors: @AlphaSenseInc and Dash Bio. https://t.co/D990phgzg8
Eli Lilly to Acquire Kelonia Therapeutics in $7 B Cash Deal
Eli Lilly agreed to acquire Cambridge‑based Kelonia Therapeutics for up to $7 billion in cash, including an undisclosed upfront payment. The deal adds Kelonia’s in‑vivo gene‑delivery platform and its lead candidate KLN‑1010 to Lilly’s oncology portfolio, and sent Lilly’s shares up 2.5%...
Chinese Researchers Propose Saliva Test to Detect Stomach Cancer Early
Researchers from Shanghai Jiao Tong University School of Medicine have pinpointed 20 bacterial species that appear in both the mouths and stomachs of gastric‑cancer patients. Their study, published in Cell Reports Medicine, proposes a non‑invasive saliva test that could identify...
BBOT Secures FDA Fast Track for Pan-KRAS Inhibitor BBO-11818 in KRAS‑Mutant Pancreatic Cancer
BridgeBio Oncology Therapeutics (BBOT) announced that the FDA granted Fast Track designation to its pan‑KRAS inhibitor BBO-11818 for advanced KRAS‑mutant pancreatic ductal adenocarcinoma. The designation accelerates development of a drug that showed a confirmed partial response in early data and...
Neolaia Synthesizes New CD38 Inhibitors
Biohaven disclosed detailed preclinical data on BHV‑2100, a TRPM3 calcium‑channel modulator aimed at neuropathic pain. A new meta‑analysis concluded that anti‑amyloid therapies for Alzheimer’s have not demonstrated clinically meaningful benefits, intensifying debate over the approach. Jiangsu and Shanghai Hengrui patented...
Incyte Discloses New TYK2 And/Or JAK1 Inhibitors
Incyte announced the discovery of a new series of small‑molecule inhibitors that selectively target TYK2 and JAK1, two kinases central to cytokine signaling in autoimmune disorders. The compounds demonstrated sub‑nanomolar potency in cell‑based assays and favorable safety margins in early...
Regulatory Hurdles, Not Tech, Slow Biotech Breakthroughs
"We need 6 'AlphaFold' moments to compress the drug delivery timeline from 10yrs to a few months" -Demis Yes and the FDA will still take 10yrs to approve it. Safetyism has throttled biotech unnecessarily and the only way to accelerate is to...
Pharma Exporters Facing Issues with Online National Drug Licensing System
Indian pharmaceutical exporters are encountering technical problems with the Online National Drug Licensing System (ONDLS), which issues Certificates of Pharmaceutical Products (COPP) under the WHO Good Manufacturing Practices scheme. The Pharmaceuticals Export Promotion Council (Pharmexcil) has launched a feedback drive...
This Biotech Firm Has Room to Run Despite Surging Nearly 500% in Past Year, Morgan Stanley Says
Arrowhead Pharmaceuticals’ stock has rocketed 473% over the past year, prompting Morgan Stanley to upgrade the shares to overweight and lift its price target to $100 from $78, indicating roughly a 44% upside. The firm is set to release Phase‑3...
Rethinking When to Start Long-Acting HIV Therapy
Real‑world data from the OPERA cohort of over 5,000 patients show that starting long‑acting cabotegravir plus rilpivirine (CAB+RPV LA) in individuals with detectable viral loads—about 11% above 50 copies/mL—can still achieve virologic suppression comparable to patients who begin treatment already...
Tortugas Neuroscience Launches to Develop Brain Disorder Drugs
Tortugas Neuroscience launches with hopes to develop drugs for brain disorders, other conditions https://t.co/zoM69VEQtV via @ADeAngelis_bio
Trump Order Boosts Psychedelic Biotech Stocks
Trump executive order lifts psychedelics biotechs https://t.co/3H00cxuF4A by @realJacobBell $GHRS $HRELP $DFTX $ENVB $COMP $ATAI #biotech