RLYB331 / Sanofi, Rallybio - LARVOL DELTA

A human anti-matriptase-2 antibody limits iron overload, α-globin aggregates and splenomegaly in β-thalassemic mice. (PubMed, Blood Adv) - "RAP-536L alone did not prevent iron overload but significantly reduced apoptosis in the erythroid populations of the bone marrow, normalized red blood cell counts and improved hemoglobin and hematocrit levels. Interestingly, the association of RLYB331 with RAP-536L entirely reversed the β-thalassemia phenotype in Hbbth3/+ mice and simultaneously corrected iron overload, ineffective erythropoiesis, splenomegaly and hematological parameters, suggesting that a multi-functional molecule consisting of the fusion of RLYB331 with Luspatercept (human version of RAP-536L) would allow administration of a single medication addressing simultaneously the different pathophysiological aspects of β-thalassemia."

Journal • Preclinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • ST14 • TMPRSS6

Rallybio Announces In-Licensing of Potential First-In-Class Preclinical Antibody Candidate from Sanofi (Businesswire) - "Rallybio Corporation...today announced that it has obtained worldwide exclusive rights to Sanofi’s KY1066, which will be referred to as RLYB331 going forward, a preclinical potentially first-in-class antibody. RLYB331 has the potential to address a significant unmet need for patients with severe anemia with ineffective erythropoiesis and iron overload, such as beta thalassemia (BT) and a subset of myelodysplastic syndromes (MDS), amongst others....Under the terms of the license agreement, Rallybio will make an upfront cash payment of $3 million to Sanofi, in addition to development and commercial milestones, and mid to high single digit royalties on net sales."

Licensing / partnership • Hematological Malignancies • Myelodysplastic Syndrome

Generation and Characterisation of KY1066, a Fully Human Antibody Targeting the Enzymatic Activity of Matriptase-2 for the Treatment of Iron Overload in Beta Thalassemia (ASH 2019) - "Furthermore, with repeat dosing, we were able to show consistent iron restriction in a Hbbth3/+ mice over multiple weeks. Together this provides evidence KY1066 has the potential to treat iron overloaded patients, such as in beta thalassemia, to improve the anemia and reduce the transfusion and iron chelation need."

ST14 • TMPRSS6

Kymab Presents Updates at the American Society of Hematology (ASH) 61st Annual Meeting and Exposition (GlobeNewswire, Kymab Group Ltd) - "Kymab...today announced that abstracts describing two lead hematology programs, KY1049 and KY1066, will be presented at the American Society of Hematology 61st Annual Meeting and Exposition, being held December 7-10, 2019 in Orlando, Florida. The presentations will include posters highlighting preclinical data from these portfolio programs: KY1049 (a fully-human FVIII-mimetic common light chain bispecific antibody) and KY1066 (a fully-human antibody against matripase-2 for the treatment of iron overload in anemia)."

Preclinical