BEAM-101 Patient Enrollment, BEAM-102 and BEAM-201 IND Submissions and BEAM-301 IND-Enabling Studies All On-track for Second Half of 2022
Natural History Study Initiated in People with Sickle Trait to Provide Insights into Key Characteristics of Sickle Cell Trait and Sickle Cell Disease
Manmohan Singh, Ph.D., Appointed to Executive Leadership Team, and Anne Marie Woodland Appointed as Head of Regulatory
Ended First Quarter 2022 with $1.2 Billion in Cash, Cash Equivalents and Marketable Securities to Support Advancement of Precision Genetic Medicines Portfolio
CAMBRIDGE, Mass., May 09, 2022 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced pipeline and business highlights and financial results for the first quarter ended March 31, 2022. In addition, as part of a long-term effort to better understand sickle cell disease (SCD), a genetic disease in which individuals carry two copies of the sickle cell mutation, and sickle cell trait, in which individuals carry only one copy of the mutation, Beam will fund and collaborate with the National Alliance of Sickle Cell Centers (NASCC) to initiate the AUNT (Achieving Understanding of the Natural History of Sickle Trait) Study, a natural history study of sickle cell trait.“Base editing has the potential to offer life-changing medicines for a broad range of diseases, and we are committed to better understanding the pathophysiology of the diseases in our pipeline, and their impacts on the lives of patients and their families,” said John Evans, chief executive officer of Beam. “We are excited to be collaborating with the Globin Research Network for Data and Discovery (GRNDad) on the AUNT natural history study in people with sickle cell trait. We are on track and expect to commence SCD patient enrollment in our Phase 1/2 BEACON-101 clinical trial to evaluate the safety and efficacy of BEAM-101 in patients with SCD, as well as make our planned IND submission for BEAM-102, which is also in development for the treatment of SCD. Our immunology and liver-directed pipelines are also progressing, with plans for additional regulatory submissions, research studies and program nominations throughout the year, and we continue to build upon our delivery capabilities, including our novel LNP technology platform, potentially allowing us to expand the future reach of our programs. We believe the parallel advancement of these diverse programs creates a broad foundation for our future growth and is evidence of our commitment to developing new and better treatments for multiple patient populations and to unlocking the full potential of precision genetic medicine.”
Pipeline Highlights & Anticipated MilestonesEx Vivo HSC Programs
- Beam continues to advance its BEAM-101 program for the treatment of SCD and expects to enroll the first patient in its Phase 1/2 clinical trial evaluating the safety and efficacy of BEAM-101 for the treatment of SCD, referred to as the BEACON-101 trial, in the second half of 2022.
- Beam’s second SCD-focused program, BEAM-102, continues to progress and the company plans to submit an investigational new drug (IND) application for BEAM-102 for the treatment of SCD in the second half of 2022.
Ex Vivo T Cell Programs
- Beam’s BEAM-201 program for the treatment of relapsed/refractory T cell acute lymphoblastic leukemia/T cell lymphoblastic lymphoma is progressing with submission of an IND application anticipated in the second half of 2022.
- Beam continues to make progress towards its planned nomination of a second CAR-T development candidate in 2022.
In Vivo LNP Liver-targeting Programs
- Beam plans to present updated preclinical data from its BEAM-301 program at the American Society of Cell and Gene Therapy (ASGCT) meeting, demonstrating high and durable editing efficiency in a mouse model of glycogen storage disease 1a (GSDIa) out to 35 weeks. Beam plans to initiate IND-enabling studies in 2022 for BEAM-301, a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation, the most common disease-causing mutation of GSDIa.
- Also at ASGCT, Beam will present new preclinical data from its base editing program targeting the treatment of alpha-1 antitrypsin deficiency, highlighting optimizations made to the editor and the guide RNA that have led to two-fold increases in observed editing potency in mice, leading to potentially clinically relevant increases in circulating alpha-1 antitrypsin at doses below 1 mg/kg.
- Beam continues to anticipate the nomination of a second liver-targeted development candidate in 2022.
Initiation of AUNT Natural History StudySCD is a severe, inherited blood disorder that alters the structure and function of oxygen-carrying hemoglobin in red blood cells and is caused by a single point mutation in the beta globin gene. Carriers of the disease, or individuals with sickle cell trait, have only one copy of the sickle mutation and produce variable amounts of the abnormal sickle hemoglobin (25-45% of total hemoglobin). Despite the high prevalence of sickle cell trait (estimated to affect 300 million individuals worldwide), research to understand its full biology and clinical features has been limited.
Beam is developing two programs for SCD, BEAM-101 and BEAM-102. BEAM-101 is designed to raise fetal hemoglobin while lowering abnormal sickle hemoglobin to