Company On-track to Submit First IND for BEAM-101 in the Second Half of 2021
Continued Progress Across Base Editing Portfolio, Including Initiation of IND-Enabling Studies for BEAM-201
CAMBRIDGE, Mass., Aug. 10, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported recent business and pipeline updates, as well as second quarter 2021 financial results.
We have made meaningful progress in advancing our base editing programs in the first half of the year, and importantly, remain on track to submit our first investigational new drug (IND) for BEAM-101 in the second half of this year, said John Evans, chief executive officer of Beam. With the initiation of IND-enabling studies for BEAM-201, we are now bringing the versatility and precision of base editing to a second therapeutic area, targeting the high unmet need in T-cell cancers with the first quad-edited cell therapy. Weve also continued to expand our innovative collaborator network, most recently through our partnership with Apellis to apply base editing to the more biologically complex disease area of the complement pathway. With a strong balance sheet, we are well positioned to advance our robust pipeline of novel base editing programs through IND filings and into the clinic as we strive to provide potentially life-long cures for patients suffering from serious diseases.
Base Editing Pipeline Progress
IND-Enabling Studies for BEAM-201 Initiated: Beam has initiated IND-enabling studies for BEAM-201 for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL). BEAM-201 is a potent and specific allogeneic CAR-T targeting CD7+ malignancies, which uses multiplex base editing to simultaneously make four knockout edits at over 96% efficiency for each gene. Relapsed/refractory T-ALL is a severe disease affecting children and adults with a five-year overall survival rate of less than 25%.
Additional Upcoming Milestones in Ex Vivo and In Vivo Base Editing Pipeline On Track: Beam anticipates filing an IND for BEAM-101, which reproduces single base changes seen in individuals with Hereditary Persistence of Fetal Hemoglobin, or HPFH, to potentially protect them from the effects of mutations causing sickle cell disease (SCD), and initiating IND-enabling studies for BEAM-102, which directly corrects the causative mutation in SCD by recreating a naturally-occurring normal human hemoglobin variant, both in the second half of 2021. Beam is also on track to nominate its first Development Candidate for in vivo base editing of the liver using LNP delivery by the end of 2021.
Data Highlighting Base Editing Approach for the Treatment of Alpha-1 Antitrypsin Deficiency Published in Molecular Therapy: In July 2021, data from a preclinical study led by the Center for Regenerative Medicine at Boston Medical Center and Boston University evaluating Beams base editors in induced pluripotent stem cells (iPS cells) from patients with alpha-1 antitrypsin deficiency (AATD) were published in Molecular Therapy. As part of the study, the researchers utilized patient-derived liver cells that mimic the biology of liver hepatocytes, the main producers of alpha-1 antitrypsin protein in the body, that likewise perform important metabolic, endocrine and secretory functions. The data showed that Beams base editors corrected the mutation in the gene responsible for AATD and reduced the effects of the disease in the hepatocytes, demonstrating successful base editing in human cells.
Upcoming Base Editing Data Presentations
Story continues
Data from Sickle Cell Disease Program to be Presented at Cold Spring Harbor Laboratorys Genome Engineering: CRISPR Frontiers: Beam plans to present preclinical data highlighting its approach to using adenine base editing (ABE) for the treatment of sickle cell disease, including updated safety data during an oral session at the Cold Spring Harbor Laboratorys Genome Engineering: CRISPR Frontiers Conference. Details of the presentation are as follows:
Title: Adenine base editing strategy for the treatment of sickle cell disease by elimination of the pathogenic globin proteinSession: Disease/TherapeuticDate: Friday, August 20, 2021, 2:30 -5:30 p.m. ET
Updated Data on Optimization of LNP Delivery Approach to be Presented at TIDES 2021: Beam plans to present preclinical data highlighting work to optimize its lipid nanoparticles (LNPs) for the delivery of mRNA encoding a base editor and guide RNA to hepatocytes in the liver during an oral session at the TIDES USA Oligonucleotide & Peptide Therapeutics Conference. Details of the presentation are as follows:
Title: Optimization of LNP for in vivo Base Editing in LiverTrack: mRNA and Genome Editing TRACK: Emerging Genome Editing TechnologiesDate/Time: Thursday, September 23, 2021, 11:15 a.m. - 11:45 a.m. ET
Business Highlights
Groundbreaking on Internal Manufacturing Facility to Support Future Product Development: In July 2021, Beam broke ground on its 100,000 square foot current Good Manufacturing Practice (cGMP) compliant manufacturing facility in Research Triangle Park, North Carolina. The facility will be designed to support manufacturing for Beams ex vivo cell therapy programs in hematology and oncology and in vivo non-viral delivery programs for liver diseases, with flexibility to support manufacturing of its viral delivery programs, and ultimately, scale-up to support commercial supply. Beam expects the facility to be operational in 2023.
Exclusive Research Collaboration Executed with Apellis to Apply Base Editing to Discover Novel Therapies for Complement-Driven Diseases: In June 2021, Beam and Apellis Pharmaceuticals, Inc. (Apellis) announced an exclusive five-year research collaboration focused on the use of Beams proprietary base editing technology to discover new treatments for complement-driven diseases. Under the terms of the collaboration agreement, Beam will apply its base editing technology and conduct preclinical research on up to six base editing programs that target specific genes within the complement system, including C3, in various organs, including the eye, liver and brain. Apellis will have exclusive rights to license each of the six programs and will assume responsibility for subsequent development. Apellis agreed to pay a total of $75 million in upfront and near-term milestones to Beam. Beam is also eligible to receive development, regulatory and sales milestones, as well as royalty payments on sales of licensed products. Beam may also elect to enter into a 50-50 U.S. co-development and co-commercialization agreement with Apellis with respect to one program licensed under the collaboration.
Second Quarter 2021 Financial Results
Cash Position: Cash, cash equivalents and marketable securities were $615.3 million as of June 30, 2021, compared to $299.7 million as of December 31, 2020.
Research & Development (R&D) Expenses: R&D expenses were $45.6 million for the second quarter of 2021, compared to $19.4 million for the second quarter of 2020.
General & Administrative (G&A) Expenses: G&A expenses were $13.4 million for the second quarter of 2021, compared to $6.9 million for the second quarter of 2020.
Net Loss: Net loss attributable to common stockholders was $76.3 million, or $1.23 per share, for the second quarter of 2021, compared to $34.2 million, or $0.69 per share, for the second quarter of 2020.
About Beam TherapeuticsBeam Therapeutics Inc. (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beams suite of gene editing technologies is anchored by base editing, a proprietary technology that enables precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.
Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: our plans to enter the clinic; our expected timing for filing an investigational new drug application for BEAM-101, for initiating IND-enabling studies for BEAM-102, for nominating a development candidate for in vivo base editing of the liver using LNP delivery, and for our manufacturing facility becoming operational; any future payments we may receive under our collaboration agreement with Apellis; our planned base editing data presentations at upcoming scientific conferences; and the therapeutic applications and potential of our technology, including our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the headings Risk Factors Summary and Risk Factors in our Annual Report on Form 10-K for the year ended December 31, 2020, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, our Quarterly Report on Form 10-Q that we will file for the quarter ended June 30, 2021, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.
Contacts: Investors:Chelcie ListerTHRUST Strategic Communicationschelcie@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
Condensed Consolidated Balance Sheet Data (unaudited)
(in thousands)
June 30,2021
December 31,2020
Cash, cash equivalents, and marketable securities
$
615,279
$
299,671
Total assets
893,078
451,677
Total liabilities
344,527
206,116
Total stockholders equity
548,551
245,561
Condensed Consolidated Statement of Operations (unaudited)
(in thousands, except share and per share data)
Three Months EndedJune 30,
Six Months EndedJune 30,
2021
2020
2021
2020
License revenue
$
6
$
6
$
12
$
12
Operating expenses:
Research and development
45,577
19,354
235,683
40,903
General and administrative
13,403
6,937
23,676
13,749
Total operating expenses
58,980
26,291
259,359
54,652
Loss from operations
(58,974
)
(26,285
)
(259,347
)
(54,640
)
Other income (expense):
Change in fair value of derivative liabilities
(42,300
)
(8,700
)
(44,200
)
(11,400
)
Change in fair value of long-term investments
25,814
517
26,852
Read the rest here:
- Detection of biomagnetic signals from induced pluripotent stem cell-derived cardiomyocytes using deep learning with ... - Nature.com - April 1st, 2024
- Researchers discover new microRNA-regulated pathway to boost iPS cell-derived platelet production - Phys.org - March 30th, 2024
- Standardized production of hPSC-derived cardiomyocyte aggregates in stirred spinner flasks - Nature.com - March 28th, 2024
- Synthetic Circuits Reveal the Key to Rewinding the Cellular Clock - The Scientist - March 13th, 2024
- synNotch-programmed iPSC-derived NK cells usurp TIGIT and CD73 activities for glioblastoma therapy - Nature.com - March 3rd, 2024
- Breaking through new research barriers with iPSC technology - News-Medical.Net - February 15th, 2024
- Abu Dhabi Stem Cells Centre partners with Rege Nephro and Kyoto University's Center for iPS Cell Research and ... - Abu Dhabi Media Office - January 12th, 2024
- Abu Dhabi Stem Cells Center partners with Japan-based Kyoto University and Rege Nephro - ZAWYA - January 12th, 2024
- Efficient and reproducible generation of human induced pluripotent stem cell-derived expandable liver organoids for ... - Nature.com - December 23rd, 2023
- Mechanisms, pathways and strategies for rejuvenation through epigenetic reprogramming - Nature.com - December 15th, 2023
- Shinobi Therapeutics Launches with Completion of $51M Series A to Advance Hypoimmune iPS-T Cell Therapy Platform - PR Newswire - December 13th, 2023
- CRISPRi gene modulation and all-optical electrophysiology in post-differentiated human iPSC-cardiomyocytes ... - Nature.com - December 7th, 2023
- World Hindu Congress 2023: Know all about the sessions and speakers - Organiser - November 23rd, 2023
- VCCT Inc., Leading the Field of Retinal Regeneration through iPS ... - Business Wire - November 21st, 2023
- Efficient protocol for the differentiation of kidney podocytes from ... - Nature.com - November 17th, 2023
- Vitamin B12 is a limiting factor for induced cellular plasticity and ... - Nature.com - November 17th, 2023
- Study reveals the critical role of microglia in human brain development - Medical Xpress - November 15th, 2023
- VCCT Inc., Leading the Field of Retinal Regeneration through iPS Cells, Raises 600 million Japanese yen (approximately $4 million USD) through... - November 15th, 2023
- Who are the leading innovators in iPSCs for the pharmaceutical ... - Pharmaceutical Technology - November 7th, 2023
- Using iPS Cells from I Peace, Heartseed Succeeds in Stable Production of High Purity Cardiomyocytes, A Major Step Forward in Advancing Autologous... - November 5th, 2023
- Systematic differences in discovery of genetic effects on gene ... - Nature.com - October 21st, 2023
- Pairwise biosynthesis of ion channels stabilizes excitability and ... - pnas.org - October 11th, 2023
- Scientists find multitude of genes associated with autism - The Statesman - October 9th, 2023
- Stem Cell Banking Market is Expected to Reach USD 16,474.30 ... - Digital Journal - October 7th, 2023
- Probing the individuality of cells and molecules - Open Access Government - October 5th, 2023
- Thymmune Therapeutics Receives $37M in Funding from the ... - Manchestertimes - October 3rd, 2023
- Thymmune Therapeutics Receives $37M in Funding from the ... - Business Wire - September 29th, 2023
- Less Funding to More Researchers Gets Nobel-level Results, Study ... - The Japan News - September 27th, 2023
- Brentuximab Vedotin Plus AVD Gets Positive CHMP Opinion for ... - OncLive - September 20th, 2023
- DKMS BMST Foundation India and Kokilaben Hospital unite to spread awareness about the Importance of Stem Cell Donation on World Marrow Donor Day - APN... - September 18th, 2023
- Vitamin B6 is governed by the local compartmentalization of ... - Science - September 14th, 2023
- Dolly the sheep creator Ian Wilmut dies aged 79 - BBC - September 14th, 2023
- Scientist who created Dolly the sheep dies aged 79: Professor Sir Ian Wilmut passes away five years after reve - Daily Mail - September 11th, 2023
- The global kits market is expected to grow at a CAGR of 7.4% from ... - Digital Journal - September 8th, 2023
- Eterna Therapeutics Announces Initiation of Development Activities ... - PipelineReview.com - September 8th, 2023
- Tokyo: 100 Years of Urban Regeneration / Tokyo's Nihonbashi Area ... - The Japan News - August 31st, 2023
- Transcriptome-based prediction of drugs, inhibiting ... - Nature.com - August 30th, 2023
- From the birds and the bees to IVG - The Gauntlet - August 26th, 2023
- Breakthrough creates stem cells without any memories - Freethink - August 26th, 2023
- Aging impairs the neurovascular interface in the heart - Science - August 26th, 2023
- For This Venture Capitalist, Research on Aging Is Personal; 'Bob ... - The Wall Street Journal - August 22nd, 2023
- Unleashing Potential: Illuminating the Future of Healthcare through Revolutionary Induced Pluripotent Stem Cel - openPR - August 18th, 2023
- A 'memory wipe' for stem cells may be the key to better therapies - The Conversation - August 16th, 2023
- Erasing epigenetic 'memory' to improve the quality of stem cells - FierceBiotech - August 16th, 2023
- HebeCell and Logomix Announce Collaboration for the ... - Business Wire - August 16th, 2023
- HebeCell and Logomix Announce Collaboration for the ... - Valdosta Daily Times - August 11th, 2023
- Advancing Clinical Trials with iPSC-Derived Liver Organoids - Microbioz India - August 11th, 2023
- Activation of cAMP (EPAC2) signaling pathway promotes hepatocyte ... - Nature.com - August 1st, 2023
- IPS HEART Announces Peer-Reviewed Publication in Cells of Aging Damage Reversal Showing GIVI-MPC Stem Cell Therapys Ability to Create New Skeletal... - July 30th, 2023
- Femtech in Japan: An Industry on the Rise - Femtech Insider - July 28th, 2023
- Association between enhanced carbonyl stress and decreased ... - Nature.com - July 28th, 2023
- Defects in early synaptic formation and neuronal function in Prader ... - Nature.com - July 27th, 2023
- IPS HEART Announces Peer-Reviewed Publication in Cells of ... - Business Wire - July 25th, 2023
- Cure to Autism and Alzheimer's May Be in Space, Says 1st Brazilian Scientist who Will Be on The ISS - Folha de S.Paulo - July 25th, 2023
- Futurama's Resurrection Ruins The Perfect Ending (& Shows Must ... - Looper - July 25th, 2023
- University Of Massachusetts Amherst's Team Bags Honour Of 2023 Armstrong Fund For Science Award - India Education Diary - July 20th, 2023
- Scientists working on IVG, a new reproductive method that can make human babies in labs - IndiaTimes - July 19th, 2023
- Co-transplantation of autologous Treg cells in a cell therapy for ... - Nature.com - July 13th, 2023
- Magic Valley and Biocellion Join Forces to Improve Bioreactor Tech ... - vegconomist - the vegan business magazine - July 13th, 2023
- IPS HEART Receives U.S. FDA Rare Pediatric Drug Designation for ... - Tullahoma News and Guardian - July 10th, 2023
- Hitachi : forms strategic research collaboration with Mass General Brigham in the field of regenerative medicine and GCT - Marketscreener.com - July 6th, 2023
- Cell therapy limitations with iPS-based multi-cell therapeutics - Drug Target Review - July 6th, 2023
- Remembering Dolly- The Clone Sheep On Her Birth Anniversary - Free Press Journal - July 4th, 2023
- Labs and Translational Research - UMass Medical School - June 28th, 2023
- Cellusion raises 2.83B series C round to progress iPS cell-derived ... - BioWorld Online - June 14th, 2023
- Japan-based Fujifilm introduces first-of-its-kind human iPSC-derived ... - BSA bureau - June 6th, 2023
- ALS: Parkinson's drug ropinirole may be an effective treatment - Medical News Today - June 4th, 2023
- Scientists predict lab-grown babies in five years - Kidspot - June 4th, 2023
- Lab-grown babies could become a reality within five years - Earth.com - May 26th, 2023
- Leading healthcare experts from MENA and beyond to explore the future of precision medicine in the region at PMES 23 this month - ZAWYA - May 24th, 2023
- Long-Awaited Ax-2 Mission Launches, Heads to Space Station - AmericaSpace - May 22nd, 2023
- Eterna Therapeutics and Factor Bioscience Announce New Data on ... - GlobeNewswire - May 17th, 2023
- Research Assistant job with UNITED ARAB EMIRATES ... - Times Higher Education - May 10th, 2023
- Regulation of synaptic connectivity in schizophrenia spectrum by ... - Nature.com - April 29th, 2023
- Researchers Discover Key Role of Glucose in Brain Activity - Laboratory Equipment - April 25th, 2023
- Structure of Huntington's Protein Revealed - Technology Networks - April 21st, 2023
- Study of brain immune cell infection may explain long COVID ... - NHK WORLD - April 19th, 2023
- The Mechanism That Regulates Activity of Memory Gene - Neuroscience News - April 19th, 2023
- The IPO Buzz: CytoMed Therapeutics Prices its Micro-Cap IPO at ... - IPOScoop - April 19th, 2023
- Macomics Presents its ENIGMAC Discovery Platform for Gene to Function Target Validation of Novel Macrophage Therapies at AACR - Yahoo Finance - April 19th, 2023
Recent Comments