Sangamo Therapeutics(US:SGMO)2021-11-03 16:00Financial Position - The company has received approximately $815.0 million in upfront licensing fees, milestone payments, and proceeds from the sale of common stock to collaborators, with potential future milestone payments of up to $6.9 billion[154]. - As of September 30, 2021, the company had $519.0 million in cash, cash equivalents, and marketable securities, indicating a strong financial position[173]. - As of September 30, 2021, the company had cash, cash equivalents, and marketable securities totaling $519.0 million, down from $692.0 million as of December 31, 2020[199]. - Net cash used in operating activities for the nine months ended September 30, 2021, was $180.5 million, reflecting a net loss of $140.8 million[202]. - Net cash provided by investing activities for the nine months ended September 30, 2021, was $195.6 million, primarily from net maturities and sales of marketable securities[205]. - Available cash, cash equivalents, and marketable securities, along with expected revenues from collaborations and research grants, are believed to be adequate to fund operations for at least the next 12 months[207]. - Future capital requirements will be substantial, necessitating additional capital through equity or debt financing to fund product development and potential commercialization[207]. Research and Development - The Phase 1/2 STAAR study of isaralgagene civaparvovec for Fabry disease, four patients showed above normal α-Gal A activity, with a 40% decrease in plasma Lyso-Gb3 levels in one patient[157]. - The Phase 1/2 PRECIZN-1 study of SAR445136 for sickle cell disease reported that none of the four treated patients required blood transfusions post-engraftment, with increases in total hemoglobin and fetal hemoglobin[157]. - In the Phase 1/2 study of giroctocogene fitelparvovec for severe hemophilia A, the mean Factor VIII activity was 30.9% at week 104, with an annualized bleeding rate of zero for the first year[157]. - The ongoing PRECIZN-1 study involves 8 subjects with severe Sickle Cell Disease (SCD) across six U.S. sites, with 5 out of 7 subjects achieving successful target yields of hematopoietic stem and progenitor cells (HSPCs) by the cutoff date of June 25, 2021[34]. - Total hemoglobin levels stabilized at 9-10 g/dL by week 26 post SAR445136 infusion, with fetal hemoglobin levels increasing from 1-11% at screening to 15-29% by week 13 and persisting at 35% in one subject with 65 weeks of follow-up[34]. - The adverse events reported were consistent with plerixafor mobilization and busulfan myeloablation therapy, with no infusion-related reactions reported for SAR445136[34]. - The company expects to incur net losses for at least the next several years as it continues its research and development activities[176]. - Research and development expenses are expected to increase in the coming years as the company advances its product candidates through clinical trials[177]. - The company expects to continue to increase research and development expenses in the coming years to support clinical programs and product candidates[189]. Clinical Trials and Studies - The Phase 3 AFFINE trial of giroctocogene fitelparvovec is currently on clinical hold by the FDA, with over 50% of patients enrolled[157]. - The company plans to present updated clinical data from the STAAR study and the PRECIZN-1 study at the 63rd Annual Meeting of the American Society of Hematology on December 12, 2021[157]. - The highest dose cohort in the Giroctocogene Fitelparvovec study demonstrated an annualized bleeding rate of zero for the first year post-infusion[167]. - Treatment-related serious adverse events were reported in one patient in the highest dose cohort, but these events fully resolved without delaying post-infusion discharge[167]. - The ongoing COVID-19 pandemic has caused delays in clinical study timelines, with the STAAR study experiencing setbacks of approximately three to six months[169]. - The company continues to monitor the impact of COVID-19 on its operations and clinical trials, with potential for future disruptions[172]. Revenue and Expenses - Revenues for the three months ended September 30, 2021, were $28.6 million, a decrease of $29.2 million (51%) compared to $57.8 million in the same period of 2020[181]. - Revenues for the nine months ended September 30, 2021, totaled $82.7 million, down $9.7 million (10%) from $92.4 million in the same period of 2020[183]. - The decrease in revenues was primarily due to a $39.3 million decline in milestone fees and upfront license fees related to collaborations with Pfizer, attributed to the completion of activities in Q4 2020[182]. - Research and development expenses for the three months ended September 30, 2021, increased by $17.2 million (38%) to $62.5 million, driven by increased clinical and lab supply expenses[187]. - General and administrative expenses for the three months ended September 30, 2021, decreased by $1.7 million (10%) to $14.5 million, primarily due to reduced legal and professional fees[193]. Leadership and Operations - The company has made the decision to discontinue the development of ST-400 to prioritize the development of SAR445136[157]. - The company has appointed D. Mark McClung as Chief Operating Officer, previously serving as Chief Business Officer[157]. - The company is collaborating with Biogen on a neuromuscular pre-clinical target, specifically type 1 myotonic dystrophy (DM1)[157]. - The company entered into a new lease for approximately 5,800 square feet of research and office space in Valbonne, France, with contractual obligations of approximately $0.8 million[211]. - The investment portfolio consists of U.S. government-sponsored entity debt securities, commercial paper, corporate debt securities, asset-backed securities, and certificates of deposit, with a focus on capital preservation and liquidity[212]. - The majority of available-for-sale securities are short-term in nature and subject to minimal interest rate risk[213]. - Volatile market conditions due to the COVID-19 pandemic may significantly affect exchange rates relative to the U.S. dollar and impact operating results[214].