On May 9, 2022 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported pipeline and business highlights and financial results for the first quarter ended March 31, 2022 (Press release, Beam Therapeutics, MAY 9, 2022, View Source [SID1234613941]). 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.

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"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 Milestones
Ex 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) (Free ASGCT Whitepaper) 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 (Free ASGCT Whitepaper), 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 Study
SCD 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 <40% of total, which is similar to levels seen in individuals with sickle cell trait. BEAM-102 is designed to replace the sickle mutation with a normal human variant of hemoglobin, HbG-Makassar, potentially reducing even further the abnormal sickle hemoglobin in patient cells. A better understanding of sickle cell trait, along with an in-depth understanding of SCD, will help better establish the relationship between levels of the abnormal sickle hemoglobin and long-term clinical outcomes.

The AUNT Study will create a first of its kind multi-center, prospective, longitudinal cohort of individuals with sickle trait, targeting a large enrollment of approximately 1,000 participants. This research is designed to establish an understanding of the hematologic and clinical phenotype of people with sickle cell trait, including blood rheology, potential complications, and genetic modifiers, in an effort to better understand the hematologic phenotype that is associated with good health and lack of organ dysfunction, as well as provide increased counseling to people with sickle cell trait.

Business Updates

Manmohan Singh, Ph.D., senior vice president, pharmaceutical sciences & delivery technologies, was appointed to the company’s executive leadership team. Dr. Singh joined Beam in 2018, bringing more than 24 years of drug discovery and development experience from Takeda Pharmaceuticals, Novartis and Chiron Corporation.
Anne Marie Woodland joined Beam as senior vice president, regulatory affairs, bringing more than 20 years of experience to Beam, previously serving in regulatory focused roles at Replimune, UniQure and BioVex.
Upcoming ASGCT (Free ASGCT Whitepaper) Presentation Details
Title: Single, systemic administration of BEAM-301 mitigates fasting hypoglycemia and restores metabolic function in a transgenic mouse model of glycogen storage disease type Ia
Date & Time: Monday, May 16, 2022, from 4:15-4:30 p.m. ET

Title: Optimized base editing reagents yield more potent genetic correction in a mouse model of alpha-1 antitrypsin deficiency (poster M-123)
Date & Time: Monday May 16, 2022, from 5:30-6:30 p.m. ET

Title: Efficient LNP delivery of mRNA in vivo and in vitro to T and NK cells (poster Tu-107)
Date & Time: Tuesday May 17, 2022, from 5:30-6:30 p.m. ET

First Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $1.2 billion as of March 31, 2022, as compared to $965.6 million as of December 31, 2021.
Research & Development (R&D) Expenses: R&D expenses were $65.4 million for the first quarter of 2022, compared to $190.1 million for the first quarter of 2021. R&D expenses for the first quarter of 2021 includes $155.0 million of expense related to in-process research and development acquired from Guide Therapeutics, Inc.
General & Administrative (G&A) Expenses: G&A expenses were $19.2 million for the first quarter of 2022, compared to $10.3 million for the first quarter of 2021.
Net Loss: Net loss was $69.2 million for the first quarter of 2022, or $1.01 per share, compared to $201.6 million for the first quarter of 2021, or $3.35 per share.