Regenerative medicine company Mesoblast Ltd. received an early Christmas present from the FDA for approval of its allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy, Ryoncil (remestemcel-L), for steroid-refractory acute graft-vs.-host disease (SR-aGvHD) in children 2 months and older, including adolescents.
Researchers from EMD Serono Research and Development Institute Inc. hypothesized that modulation of two T-cell costimulatory pathways, such as CD28 and OX40, in one single molecule would be more efficient at controlling T-cell activation than modulating each pathway separately.
The signaling of TNF receptor (TNFR) superfamily member 4 (OX40) and its ligand (OX40L) plays a crucial role in the development of immunological and inflammatory disorders due to triggering a subset of T-cell responses.
Inmagene LLC presented preclinical data for the novel noncovalent reversible Bruton tyrosine kinase (BTK) inhibitor IMG-004, which is in phase I development for autoimmune diseases.
Wall Street promptly started speculating about the product’s odds in the graft-vs.-host disease (GVHD) marketplace shortly after Syndax Pharmaceuticals Inc. and Incyte Corp. scored U.S. FDA approval – well ahead of the Aug. 29 PDUFA date – of Niktimvo (axatilimab), an anti-CSF-1R antibody for the treatment of chronic disease after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs.).
Researchers in Japan have discovered that a phage-derived enzyme called endolysin, which targets highly resistant biofilm-forming bacteria, could help restore the gut microbiota to mitigate acute graft-vs.-host disease. Acute graft-vs.-host disease (aGVHD) is a common complication for patients who undergo allogeneic hematopoietic cell transplantation (allo-HCT). Recent studies have highlighted the significant role of the microbiome in aGVHD, with dysbiosis contributing to its pathogenesis.
MTX-101 (Mozart Therapeutics Inc.) is a bispecific CD8 Treg modulator that targets CD8 and the KIR2DL family, including KIR2DL1, KIR2DL2 and KIR2DL3, and is being developed for the treatment of CD4-driven autoimmunity and restoring immune balance.
Altrubio Inc. secured up to $225 million in an oversubscribed series B financing round, led by BVF Partners LP. The full amount is dependent on Altrubio meeting certain undisclosed milestones. New investors RA Capital Management, Cormorant Asset Management and Soleus Capital, and existing investors Amoon Fund and Blackstone Multi-Asset Investing, as well as other new and existing investors, also participated in the financing round.
The U.S. FDA has cleared Tr1x Inc.’s IND application for TRX-103 for the prevention of graft-vs.-host disease (GVHD) in patients undergoing HLA-mismatched hematopoietic stem cell transplantation (HSCT).
Tr1x Inc. announced a $75 million series A financing to advance universal allogeneic regulatory T (Treg) and chimeric antigen receptor (CAR)-Treg cell therapies into the clinic to treat autoimmune and inflammatory diseases.
