Beam Therapeutics(US:BEAM)2024-11-05 14:06Clinical Trials and Programs - BEAM-101 Phase 1/2 clinical trial for sickle cell disease has enrolled 35 patients, with 8 dosed and 7 expected to present additional data at ASH[81][82] - BEAM-101 demonstrated rapid and robust HbF induction (>60%) and HbS reduction (≤36%) in non-transfused blood for patients with at least one month of follow-up[81] - ESCAPE program aims to reduce toxicity in HSCT with BEAM-103 and BEAM-104, with Phase 1-enabling preclinical studies anticipated by end of 2024[83] - Preclinical data for ESCAPE in non-human primates showed robust long-term engraftment and high HbF expression, with additional data expected at ASH[83] - BEAM-302 Phase 1/2 trial for AATD has completed dosing of the first cohort, with initial clinical data expected in 2025[86] - BEAM-201 Phase 1/2 trial for T-cell malignancies showed complete responses in 2 of 3 patients at CAR-T cell doses >200 million, with additional data expected at ASH[90] Collaborations and Partnerships - The company entered into a four-year research collaboration with Pfizer in December 2021, focusing on in vivo base editing programs for three targets related to rare genetic diseases of the liver, muscle, and central nervous system[93] - Under the Pfizer collaboration, the company has the right to opt into a global co-development and co-commercialization agreement for one program, sharing net profits and costs with Pfizer in a 35%/65% ratio[93] - In October 2023, the company received a $200.0 million payment from Eli Lilly and is eligible for up to $350.0 million in future development-stage payments under the Lilly Agreement[95] - The company sold 2,004,811 shares of common stock to Eli Lilly at $24.94 per share, totaling approximately $50 million, representing a 15% premium to the 30-day volume-weighted average price[96] Financial Performance and Expenses - The company's net losses for the nine months ended September 30, 2024, were $286.4 million, compared to $275.3 million for the same period in 2023[102] - As of September 30, 2024, the company had an accumulated deficit of $1.5 billion[102] - The company recognized $33.5 million in license and collaboration revenue for the nine months ended September 30, 2024, compared to $61.5 million for the same period in 2023[103] - The company expects research and development expenses to increase substantially as it advances programs through preclinical and clinical development[106] - The company anticipates increased general and administrative expenses to support expanded research and development activities and public company compliance costs[107] - License and collaboration revenue decreased by $2.9 million to $14.3 million in Q3 2024 compared to $17.2 million in Q3 2023, driven by lower research activities[109] - Research and development expenses decreased by $5.8 million to $94.3 million in Q3 2024, primarily due to a $7.0 million reduction in external R&D expenses and a $2.3 million decrease in employee-related costs[110] - General and administrative expenses increased by $1.1 million to $26.5 million in Q3 2024, mainly due to a $1.7 million rise in employee-related costs and a $0.6 million increase in stock-based compensation[112] - Net loss for Q3 2024 was $96.7 million, slightly higher than the $96.1 million loss in Q3 2023[109] - License and collaboration revenue for the nine months ended September 30, 2024, was $33.5 million, down $28.1 million from $61.5 million in the same period in 2023[113] - Research and development expenses for the nine months ended September 30, 2024, decreased by $31.2 million to $266.1 million, driven by a $35.8 million reduction in external R&D expenses[114] - General and administrative expenses for the nine months ended September 30, 2024, increased by $9.3 million to $82.9 million, primarily due to a $4.6 million rise in stock-based compensation and a $3.5 million increase in personnel-related expenses[115] - Net loss for the nine months ended September 30, 2024, was $286.4 million, compared to $275.3 million in the same period in 2023[113] - Interest and other income for the nine months ended September 30, 2024, increased by $3.6 million to $38.2 million, driven by higher market rates[116] - The fair value of non-controlling equity investments resulted in a $13.0 million expense for the nine months ended September 30, 2024, compared to a $17.9 million expense in the same period in 2023[116] Revenue and Funding - The company's revenue to date has been primarily derived from license and collaboration agreements, with no revenue from product sales expected in the near future[103] - Company has not generated any revenue from product sales since inception in January 2017 and expects significant operating losses for the foreseeable future[117] - Company sold 10,860,992 shares of common stock under amended Sales Agreement at an average price of $51.93 per share for aggregate gross proceeds of $564.0 million[117] - Company received $200.0 million upfront payment from Lilly Agreement and is eligible for up to $350.0 million in potential future development-stage payments[117] - Company had $925.8 million in cash, cash equivalents, and marketable securities as of September 30, 2024[117] - Net cash used in operating activities for the nine months ended September 30, 2024 was $270.9 million, including a net loss of $286.4 million[120] - Cash provided by investing activities for the nine months ended September 30, 2024 was $58.7 million, primarily from net maturities of marketable securities[120] - Net cash provided by financing activities for the nine months ended September 30, 2024 was $4.3 million, mainly from proceeds from the issuance of common stock[120] - Company expects operating expenses to increase over the next twelve months due to clinical-stage development and biologics license application readiness activities[121] - Company's cash, cash equivalents, and marketable securities as of September 30, 2024 are expected to fund operations for at least the next 12 months[122] - Aggregate future minimum commitments under office and laboratory leases as of September 30, 2024 are $231.5 million[124] Manufacturing and Facilities - Company established a 100,000 square foot manufacturing facility in Research Triangle Park, NC, supporting clinical programs and potential commercial supply[91]