On November 10, 2020 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported pipeline updates, recent business highlights and third quarter 2020 financial results (Press release, Beam Therapeutics, NOV 10, 2020, View Source [SID1234570443]).

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"2020 has been a year of significant progress for Beam," said John Evans, chief executive officer of Beam. "Since the start of the year, we’ve named three development candidates from our portfolio, now including BEAM-201, our multiplex editing program for the treatment of T-cell acute lymphoblastic leukemia. We are also pleased to report that we’re on track to submit our first IND in the second half of 2021, with BEAM-101 for the treatment of sickle cell disease. The continued advancement of our pipeline is a testament to both the strength and breadth of our base editing platform and our exceptional team. Combined with the capital we’ve added to our balance sheet, we are well positioned to continue our strategy of advancing multiple programs to the clinic in parallel, in the hope of providing much-needed new treatment options for patients with serious diseases."

Base Editing Progress

First CAR-T Base Editing Development Candidate, BEAM-201, Named for Treatment of T-ALL; Data Presented at Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 35th Anniversary Annual Meeting & Pre-Conference Programs (SITC 2020): Beam is advancing BEAM-201, a potent and specific anti-CD7 CAR-T multiplex editing program for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL), a severe disease affecting children and adults with five-year overall survival of less than 25%. BEAM-201 is produced using a GMP-compliant, clinical-scale process in which T-cells derived from healthy donors are simultaneously base edited at four genomic loci then transduced with a lentivirus coding for an anti-CD7 CAR. The resulting cells are universally-compatible, allogeneic ("off the shelf") CD7-targeting CAR-T cells resistant to both fratricide and immunosuppression. Preclinical data from this approach were reported in a poster as part of SITC (Free SITC Whitepaper) 2020, which is being held virtually from November 9-14, 2020. Editing with BEAM-201 led to potent, dose-dependent tumor control in vitro and in an in vivo xenograft mouse model. Details of the presentation are as follows:
Title: Highly Efficient Multiplexed Base Editing Enables the Development of Investigational Universal CD7-targeting CAR-T Cells to Treat T-ALL
Publication Number: 111
Category: Cellular Therapies
Upcoming Base Editing Data Presentations

First Data Highlighting Base Editing Program for Glycogen Storage Disease Type Ia to be Presented at American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting Digital Experience: Beam will present the first data highlighting its novel base-editing strategy for correcting disease-causing mutations underlying Glycogen Storage Disease Type Ia (GSDIa) during a poster session at AASLD’s The Liver Meeting Digital Experience, which is being held virtually November 13-16, 2020. Details of the presentation are as follows:
Title: Base-Editing as a Therapeutic Approach for the Direct Correction of Disease-Causing Mutations Underlying Glycogen Storage Disease Type Ia
Publication Number: 0589
Session Title: Genomics and Precision Medicine
Beam will also report data during an oral presentation at AASLD from its Alpha-1 Antitrypsin Deficiency (Alpha-1) program. Details of the presentation are as follows:

Title: Evaluation of Adenine Base Editing as a Potential Treatment for Alpha-1 Antitrypsin Deficiency
Publication Number: 0032
Session Title: Parallel 3: Metabolic and Genetic Diseases
Session Broadcast Date and Time: Saturday, November 14, 2020, 2:00 p.m. ET
Updated Data from Novel Sickle Cell Disease Approaches to be Presented at 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH 2020): Beam is pursuing two differentiated base editing approaches to treat hemoglobinopathies, BEAM-101 and BEAM-102, and will present updated preclinical data from these two complementary editing programs during poster sessions at ASH (Free ASH Whitepaper) 2020, being held virtually December 5-8, 2020. Details of the presentations are as follows:
Title: Adenine Base Editing of the Sickle Allele in CD34+ Hematopoietic Stem and Progenitor Cells Eliminates Hemoglobin S
Session: 801. Gene Editing, Therapy and Transfer: Poster I
Date: Saturday, December 5, 2020
Publication Number: 1543
Title: Adenine Base Editing of Gamma Globin Gene Promoters Shows No Detectable Off-Target RNA or DNA Editing
Session: 801. Gene Editing, Therapy and Transfer: Poster I
Date: Saturday, December 5, 2020
Publication Number: 1545
Recent Business Highlights

Successful $135 Million Follow-on Offering Completed: In October 2020, Beam sold 5,000,000 shares of common stock at a public offering price of $23.50 per share. Underwriters exercised in full their option to purchase up to an additional 750,000 shares of common stock at the public offering price, less the underwriting discounts and commissions. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Beam, were approximately $135.1 million. The net proceeds of the offering were $126.6 million.
Leadership Update – Courtney Wallace Promoted to Chief Business Officer: In November 2020, Beam promoted Courtney Wallace to chief business officer. Ms. Wallace previously served as senior vice president, head of business development and strategy at Beam. Ms. Wallace has served as Beam’s head of corporate strategy and business development since 2018.
Upcoming Investor Conference Presentation

John Evans, chief executive officer, will participate in a fireside chat during the Jefferies Virtual London Healthcare Conference on Thursday, November 19, 2020 at 4:25 p.m. GMT/11:25 a.m. ET.

The live webcast will be available in the investor section of the company’s website at www.beamtx.com. The webcast will be archived for 60 days following the presentation.

Third Quarter 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $202.2 million as of September 30, 2020. This cash balance does not include the proceeds of the October 2020 offering.
Research & Development (R&D) Expenses: R&D expenses were $29.8 million for the quarter ended September 30, 2020, compared to $12.5 million for the quarter ended September 30, 2019.
General & Administrative (G&A) Expenses: G&A expenses were $7.5 million for the quarter ended September 30, 2020, compared to $5.5 million for the quarter ended September 30, 2019.
Net Loss: Net loss attributable to common stockholders was $34.5 million, or $0.69 per share, for the quarter ended September 30, 2020, compared to $22.3 million, or $3.31 per share, for the quarter ended September 30, 2019.