Pipeline and Product Development - Catalyst Biosciences is focused on developing protease therapeutics targeting disorders of the complement system, with a pipeline including a preclinical C3 degrader program licensed to Biogen for geographic atrophy in dry AMD[77]. - The complement portfolio includes CB 4332, a first-in-class CFI molecule for subcutaneous administration in CFI deficiency, and CB 2782-PEG, a C3 degrader in preclinical development for dry AMD[79]. - Patient enrollment for the CFI-001 and CFI-002 studies commenced in July 2021 to assess CFI blood levels and identify candidates for CB 4332 treatment[80]. - The prevalence of rare CFI variants in the overall AMD population is estimated at approximately 6%, with around 40% expected to have low serum CFI levels that could benefit from targeted therapy[91]. - The ProTUNE™ C3b/C4b degrader platform is prioritizing IgA nephropathy, which affects over 100,000 individuals in the US and leads to significant economic burden due to chronic treatment costs[95]. - The ImmunoTUNE™ C3a/C5a degrader platform targets ANCA vasculitis, with an estimated addressable patient population of around 50,000 in the US[96]. - MarzAA, a next-generation Factor VIIa variant, has seen enrollment in a Phase 3 trial for hemophilia treatment but has been discontinued due to enrollment challenges and competition[97]. - The company is exploring opportunities to license or sell MarzAA for further development following the discontinuation of its trials[97]. - The protease candidates are designed to have improved properties such as longer half-life and higher potency, potentially allowing for better safety and efficacy in treatment[78]. - DalcA, a next-generation SQ Factor IX product candidate, completed a Phase 2b study in 2020, raising FIX plasma activity levels from severe to mild phenotype[98]. - The company is actively seeking a partner for the DalcA program and exploring opportunities for licensing or selling it for further development[98]. Financial Performance - In Q1 2021, the company issued 9,185,000 shares at $5.75 per share, netting approximately $49.3 million after underwriting discounts[101]. - The company reported net losses of $25.2 million for Q3 2021, compared to $16.0 million for Q3 2020, marking a 57% increase in losses[103]. - Total research and development expenses for the nine months ended September 30, 2021, were $52.8 million, with significant investments in hemophilia and complement programs[113]. - License and collaboration revenue for Q3 2021 was $2.3 million, a 157% increase from $893,000 in Q3 2020[123]. - The accumulated deficit as of September 30, 2021, was $382.4 million, with cash and cash equivalents totaling $64.5 million[103]. - The company expects to incur significant expenses and increasing operating losses for the next several years as it continues development and seeks regulatory approval[104]. - Research and development expenses for hemophilia were $6.0 million for Q3 2021, and $17.8 million for the nine months ended September 30, 2021[113]. - The company has not generated any revenue from drug product sales and does not expect to until regulatory approval is obtained[109]. - License and collaboration revenue decreased to $4.9 million for the nine months ended September 30, 2021, down 74% from $18.9 million in the same period of 2020[125]. - Research and development expenses increased to $52.8 million for the nine months ended September 30, 2021, a rise of 37% compared to $38.4 million in 2020[128]. - General and administrative expenses rose to $14.8 million for the nine months ended September 30, 2021, reflecting a 24% increase from $11.9 million in 2020[130]. - The net loss for the nine months ended September 30, 2021, was $67.6 million, an increase of 81% from a net loss of $37.3 million in 2020[134]. - Cash used in operating activities for the nine months ended September 30, 2021, was $66.1 million, compared to $32.9 million in the same period of 2020[139]. - Cash provided by investing activities was $45.3 million for the nine months ended September 30, 2021, primarily due to $46.2 million in proceeds from maturities of investments[141]. - Cash provided by financing activities was $49.6 million for the nine months ended September 30, 2021, mainly from $49.3 million in net proceeds from the issuance of common stock[142]. - As of September 30, 2021, the company had $64.5 million in cash, cash equivalents, and short-term investments[134]. - The accumulated deficit as of September 30, 2021, was $382.4 million[134]. - The company plans to fund future losses through equity and/or debt financings, as well as potential asset sales and collaborations[135]. Operational Challenges - Catalyst continues to face operational challenges due to the COVID-19 pandemic, which may impact development timelines[82].
Gyre Therapeutics(GYRE) - 2021 Q3 - Quarterly Report