BE-101 / Be Biopharma - LARVOL DELTA

Become–9: A Phase 1/2 Dose Escalation and Expansion Study of be-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B (ASH 2024) - "The total duration of study participation is approximately 52 weeks (1 year) post IV administration of BE-101. The study is expected to initiate enrollment in the first dose escalation cohort in the second half of 2024 (NCT : Pending)."

Clinical • P1/2 data • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

A Versatile B Cell Engineering Platform Enables Development of B Cell Medicines for Sustained Delivery of Therapeutic Biologics (ASH 2024) - "A robust nonclinical data package including genome safety assessments as well as short and long-term in vivo studies in NOG-IL6 mice was submitted to the US FDA, who cleared the BeCoMe-9 first-in-human (FIH) Phase 1/2 clinical trial to begin later this year and granted orphan drug designation for BE-101. In summary, our B cell engineering platform supports the development of novel BCMs which express therapeutically relevant transgenes and have the potential for broad and meaningful therapeutic utility in genetic diseases, cancer, and beyond."

IO biomarker • Genetic Disorders • Hemophilia • Hemophilia B • Rare Diseases • CD19 • IL6

Development of precision gene engineered B cells as a treatment for hemophilia B (ESGCT 2024) - "US FDA cleared IND Application for BE-101. We are on track to initiate the Phase 1/2 first-in-human clinical trial in 2024."

Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

A Precision Gene Engineered B Cell Medicine Producing Sustained Levels of Active Factor IX for Hemophilia B Therapy. (PubMed, Mol Ther) - "No safety signals were observed in animal tolerability and GLP toxicology studies. In conclusion, BE-101 produces sustained levels of active FIX-Padua with the ability to engraft without host preconditioning and with the potential for redosing and titratability."

Journal • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Be Bio Announces Closing of $92M Series C Financing and Completes Transition to a Multi-Program, Clinical Stage Company (Businesswire) - "Be Biopharma, Inc...today announced the closing of its $92M Series C financing...The proceeds from the Series C financing will be used to generate clinical proof-of-concept for BE-101 in the ongoing BeCoMe-9 Phase 1/2 clinical trial for people with hemophilia B and to advance BE-102 for the treatment of hypophosphatasia to the clinic. Both programs are built on Be Bio’s BCM product platform, creating durable, titratable, and redosable therapeutics that require no preconditioning and produce sustained and constant levels of therapeutic proteins."

Financing • Genetic Disorders • Hemophilia B

BeCoMe-9: a Clinical Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: Be Biopharma | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

BeCoMe-9: a Clinical Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B (clinicaltrials.gov) - P1/2 | N=24 | Not yet recruiting | Sponsor: Be Biopharma

New P1/2 trial • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Development of BE-101, an autologous ex vivo precision gene engineered B cell medicine that produces active and sustained levels of factor IX for the treatment of hemophilia B (ISTH 2024) - "Engineered BCMs secreted up to 60 ng/1e6 cells/hour of FIX protein. BCM-produced FIX analyzed by LC-MS demonstrated gamma carboxylation of FIX protein Gal-domain. Vitamin K-dependent activated partial thromboplastin time (aPTT) using the one stage clotting and immunocapture chromogenic assays (left Figure), were employed to verify biological activity of BCM-produced FIX."

Preclinical • Hematological Disorders • Hemophilia • Rare Diseases

FDA Grants Orphan Drug Designation for BE-101, a Novel Engineered B Cell Medicine, for the Treatment of Hemophilia B (Businesswire) - "Be Biopharma, Inc...announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to BE-101, a novel engineered B Cell medicine (BCM) being developed for the treatment of Hemophilia B. Be Bio is on track to initiate its Phase 1/2 study, BeCoMe-9, evaluating BE-101 in adults with severe or moderately severe Hemophilia B in the second half of 2024."

New P1/2 trial • Orphan drug • Genetic Disorders • Hemophilia

Be Biopharma Announces FDA Clearance of IND Application for BE-101 in Hemophilia B (Businesswire) - "Be Biopharma, Inc...announced the clearance of its Investigational New Drug application (IND) from the U.S. Food and Drug Administration (FDA) for BE-101, a first-in-class BCM in development for the potential treatment of hemophilia B. The Phase 1/2 clinical trial, BeCoMe-9, is a multi-center, first-in-human dose escalation study designed to assess the safety and preliminary efficacy of BE-101 in adult participants with moderately severe to severe hemophilia B. Be Bio expects to begin dosing participants in the second half of 2024."

IND • New P1/2 trial • Hemophilia