On March 30, 2020 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported recent business highlights and full year 2019 financial results (Press release, Beam Therapeutics, MAR 30, 2020, View Source [SID1234555977]).
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"So far in 2020, we have made strides across all aspects of our business, and despite the many uncertainties we face today amid the COVID-19 pandemic, we remain committed to moving our programs forward for patients," said John Evans, chief executive officer of Beam. "Following the successful completion of our initial public offering and the recent additions to our team, we have the resources needed to steadily advance a broad and deep portfolio of base editing programs addressing a wide range of disease areas and to invest in the science of base editing, illustrated by the recent platform technology publications. As we look ahead, we are planning for additional scientific publications throughout the year and remain on track to file an initial wave of IND applications beginning in 2021, in an effort to bring a new class of precision genetic medicines to the clinic and, ultimately, to patients."
Recent Business Highlights
Leadership Team Strengthened with Key Appointments: Since the beginning of 2020, Beam has named several appointments and promotions aimed at further supporting advancement of its pipeline and expanding its business operations. These include the promotion of Giuseppe "Pino" Ciaramella, Ph.D., to president and chief scientific officer, the promotion of Manmohan Singh, Ph.D., to senior vice president of pharmaceutical sciences and delivery technologies, and the appointment of Christine Swenson as senior vice president of regulatory affairs.
Advancements in Base Editing Technology Platforms Highlighted in Scientific Publications: New research on Beam’s novel adenine base editor (ABE) and cytosine base editor (CBE) technologies were reported in multiple scientific publications. Data from these internal and collaborator publications further support that Beam’s continued evolution of ABEs and CBEs can enhance the precision of targeted A-to-G and C-to-T DNA base changes, potentially improving the range of therapeutic applications.
In a paper entitled, "ABE8: Superior adenine base editors with expanded targeting range, higher activity, and therapeutic application", accepted for publication in Nature Biotechnology, Gaudelli, et al., highlight continued evolution of Beam’s adenine base editors. These proprietary, next-generation "ABE8 variants" demonstrated improved therapeutic potential compared to previously published versions, including increased on-target activity while maintaining low-levels of off-target editing across a number of cell types, including transformed and human primary cells.
In a paper entitled, "Next-generation cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity," accepted for publication in Nature Communications by Yu, et al., highlighted proprietary next-generation versions of CBEs. Off-target editing rates with the first published version of CBEs, known as BE3, are low and within mutation rates that naturally occur with no consequence in somatic cells. These next-generation CBEs display comparable on-target editing to BE3 while further reducing the low levels of off-target editing seen with BE3.
Two papers published in Nature Biotechnology by Beam co-founder Dr. David Liu, highlight novel CBEs that diminish or eliminate the low levels of off-target editing observed with BE3s, and novel variants of base editors that are able to bind a wider range of genetic targets, expanding their therapeutic potential.
Oversubscribed $207M Initial Public Offering Completed: On February 11, 2020, Beam announced the closing of its initial public offering of 12,176,471 shares of common stock, including the exercise in full by the underwriters of their option to purchase up to 1,588,235 additional shares of common stock, at a public offering price of $17.00 per share. The net proceeds to Beam Therapeutics from the offering were approximately $188.3 million, after deducting underwriting discounts and commissions and estimated offering expenses.
Business Continuity: Beam is executing its business objectives for 2020 and beyond, while closely monitoring the impact of the COVID-19 situation. The company has established a remote operating model while prioritizing the most critical research and development activities needed to advance its portfolio of novel base editing programs.
Full Year 2019 Financial Results
Cash Position: Cash, cash equivalents and marketable securities were $91.8 million as of December 31, 2019, which does not include the $188.3 million in net proceeds from the company’s IPO completed in February 2020. In addition, cash used to fund our operations was $72.0 million for the year ended December 31, 2019.
R&D Expenses: Research and development (R&D) expenses were $54.6 million for the full year ended December 31, 2019.
G&A Expenses: General and administrative (G&A) expenses were $20.6 million for the full year ended December 31, 2019.
Net Loss: Net loss was $78.3 million for the full year ended December 31, 2019.