Heightening prospects for an oral neutropenia therapy, X4 Pharmaceuticals Inc.’s lead CXCR4 antagonist mavorixafor slashed yearly infection rates in patients with WHIM syndrome by 58% in a pivotal phase III trial, an effect that was statistically significant.
Regulatory T-cell specialist Dualyx NV has closed a €40 million (US$43.5 million) series A to progress the lead autoimmune disease program to the clinic and to take forward two other Treg-based therapies. The company brings together expertise in antibody design with understanding of the role Tregs play in supressing the immune response to maintain homeostasis and self-tolerance, preventing autoimmunity.
Watchers of the Bruton’s kinase (BTK) inhibitor space may be casting renewed skepticism in that direction after Merck KGaA disclosed April 12 that the U.S. FDA placed a partial clinical hold on the sign-up of more patients in work testing evobrutinib in relapsing multiple sclerosis (MS) – but BTK efforts in MS continue in various quarters.
By analyzing a cohort of adolescents that developed myocarditis or pericarditis after vaccination against SARS-CoV-2 vaccination, researchers from Yale University School of Medicine were able to pinpoint the underlying mechanism as an overly active innate immune response to the vaccine that led to broad activation of T cells and natural killer (NK) cells. Myocarditis “has been seen in other vaccine contexts, though is most common after viral infection,” Carrie Lucas told reporters at a press conference announcing the findings.
Daewoong Pharmaceutical Co. Ltd. has out-licensed its phase I-ready autoimmune disease candidate, DWP-213388, to new spinout company Vitalli Bio in a deal worth up to $477 million.
Orbital Therapeutics Inc. raised $270 million in a series A round to fund a big push into the next generation of mRNA-based therapies. The Cambridge, Mass.-based firm is building out a comprehensive RNA platform from which it will launch programs in oncology, autoimmune disease and indications involving protein replacement approaches.
Pipeline Therapeutics Inc., which received U.S. FDA approval to move into a phase Ib/IIa study of PIPE-307 in relapsing-remitting multiple sclerosis (RRMS) last year, will be advancing the oral, small-molecule muscarinic M1 receptor antagonist in collaboration with Janssen Pharmaceutica NV in an agreement that could be worth more than $1 billion.
Pharming Group NV’s stock skyrocketed March 24 on news that the U.S. FDA approved Joenja (leniolisib) to treat activated phosphoinositide 3-kinase delta syndrome (APDS) for those ages 12 and older. The nod, which came a few days ahead of the March 29 PDUFA date, sent shares (NASDAQ:PHAR) up 33%, or $3.69, to end the day at $14.96.
Phase II data that rolled out from Merck KGaA with its Bruton's tyrosine kinase (BTK) inhibitor evobrutinib in relapsed multiple sclerosis (MS) put more eyeballs on the mechanism. It’s already well validated in oncology, but resistance has arisen there, and at least two firms – Beigene Ltd. and Nurix Therapeutics Inc. – are striving for solutions with degrader candidates.
Incyte Corp.’s bid for a once-daily vs. twice-daily version of the Janus kinase inhibitor Jakafi (ruxolitinib) was foiled, at least temporarily, by a complete response letter (CRL) from the U.S. FDA.
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