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Phase 1 study to assess safety and early efficacy of KMA‑targeting car‑t in multiple myeloma
Original title: “KOALA trial opens to enrol patients for first‑in‑human KMCAR T‑cell therapy”
Phase 2 bridge data show consistent outcomes in patients with impaired kidney function
Original title: “Pepaxti study supports use in wider myeloma population”
UCB’s Bimzelx elicited significantly stronger joint relief at 16 weeks than AbbVie’s Skyrizi in a Phase 3 head-to-head study of psoriatic arthritis.
Original title: “AbbVie’s new immunology standard-bearer Skyrizi kneels to UCB’s Bimzelx in psoriatic arthritis”
London: French drugmaker Sanofi said on Monday a trial showed its rare disease therapy was better than standard care in raising levels of a key protein in patients with a genetic form of lung... London: French drugmaker Sanofi said on Monday a trial showed its rare disease therapy was better than standard care in raising levels of a key protein in patients with a genetic form of lung disease. Unlike plasma-derived treatments, efdoralprin alfa is produced using recombinant technology, potentially easing dependence on donated plasma, said Alaa Ahmed, Sanofi's head of medical affairs for rare diseases. Ahmed said the drug's less frequent dosing could also appeal to patients for whom weekly treatment is a burden. Also Read:Sanofi's Thyrotropin Alfa Label Update: CDSCO Panel Recommends New Prescription Warning, Limits Sale to Specialists Only · sanofilung diseaseefdoralprin alfaaat deficiencyclinical trialinhibrx dealdupixentSource : Reuters Sanofi said the new data, presented at the American Thoracic Society meeting in Orlando, showed that in the 97-patient trial, efdoralprin alfa administered every three weeks raised trough levels for AAT protein by 24.1 micromolar at week 32, versus 7.6 micromolar for weekly plasma-derived standard therapy. Adding Metformin with GLP-1RA: What are the Five Points Clinicians Need to Know?- Dr NK Singh
Original title: “Sanofi's Rare Lung Disease Drug Outperforms Standard Therapy in Head-to-Head Trial”
Jaguar Health, Inc. has announced a key development in its clinical trial for microvillus inclusion disease (MVID), a rare pediatric condition lacking approved therapies. The trial focuses on crofelemer, an oral solution aimed at reducing reliance on parenteral support (PS), crucial for MVID patients. The transition to the active treatment... Jaguar Health, Inc. has announced a key development in its clinical trial for microvillus inclusion disease (MVID), a rare pediatric condition lacking approved therapies. The trial focuses on crofelemer, an oral solution aimed at reducing reliance on parenteral support (PS), crucial for MVID patients. The transition to the active treatment-only phase underscores longer-term safety and efficacy as pivotal targets. First MVID patient enters active treatment extension phase with liquid crofelemer as adjunctive therapy. Jaguar Health focuses on rare pediatric disease treatment With orphan drug status already secured, Jaguar aligns its efforts toward pioneering treatments for these severe, life-long health concerns.R. P. Copyright © 2026 FinanzWire, all reproduction and representation rights reserved. Disclaimer: although drawn from the best sources, the information and analyzes disseminated by FinanzWire are provided for informational purposes only and in no way constitute an incentive to take a position on the financial markets. Crofelemer Clinical Trial MVID Treatment Parenteral Support Pediatric Health
Original title: “Jaguar Health, Inc. (NASDAQ:JAGX) | Jaguar Health Advances Clinical Trial for MVID Treatment”
Analysts are extremely encouraged by Phase 2 trial results for Relay Therapeutics’ PI3KA inhibitor in treating vascular malformations (VM), prompting the biotech to eye a potential path to accelerated approval.
Original title: “Relay doubles the bar, outpacing Novartis with a 60% response in rare disease”
BioMarin’s investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth.
Original title: “BioMarin suffers another blow to rare disease portfolio in Phase 3 flop”
Trial shows first head‑to‑head superiority in joint endpoints for an approved PsA biologic
Original title: “UCB reports positive outcomes for bimekizumab in psoriatic arthritis”
Baxdrostat delivers clinically meaningful blood pressure reductions in phase 3 BaxHTN trial
Original title: “AstraZeneca wins US approval for first‑in‑class Baxfendy in hypertension”
Merck and Kelun-Biotech’s antibody-drug conjugate significantly improved progression-free and overall survival in a pivotal endometrial cancer study, though the companies have yet to specify when they plan to file for approval.
Original title: “Merck’s billion-dollar Kelun bet pays off as TROP2 improves survival in endometrial cancer”
A month after reporting that its RAS inhibitor daraxonrasib doubled survival in advanced pancreatic cancer, Truist said Revolution Medicines “is evolving into a major revenue-generating oncology company,” and projects an approval in second-line disease by the end of the third quarter.
Original title: “Is Revolution ‘the next oncology titan’? Truist analysts make the case”
A two-drug regimen of Regeneron’s fianlimab and Libtayo didn’t significantly outperform Keytruda in a late-stage study, a finding that surprised Wall Street analysts and wiped out billions in market value.
Original title: “Regeneron immunotherapy combo comes up short in melanoma trial”
The late-stage miss shakes analyst confidence in Regeneron’s clinical execution, according to BMO Capital Markets, also noting last year’s Phase 3 failure in chronic obstructive pulmonary disease.
Original title: “Regeneron misses again as melanoma combo bows to Merck’s Keytruda in Phase 3”
Parsortix platform to support minimally invasive analysis across various cancer trials
Original title: “Cellbxhealth partners with AdventHealth on CTC‑based monitoring in two major cancer studies”
Data suggests microbiome co-therapy may help overcome resistance to anti‑PD‑1 treatment
Original title: “Microbiotica reports positive results for MB097 in advanced melanoma”
Last month, Revolution Medicines’ RAS inhibitor doubled survival in a Phase 3 pancreatic cancer trial. On the biotech’s heels are Immuneering, Actuate Therapeutics, Erasca and more, looking to improve on that result with increased tolerability—and more time for patients.
Original title: “Can Revolution’s 'miracle' pancreatic cancer drug be topped? Immuneering, Actuate say yes”
The company plans to unblind the late-stage study that was halted due to safety concerns, which could lead to an overhaul of its entire development program.
Original title: “FDA hold puts Aardvark Prader-Willi drug in limbo”