Assembly Biosciences(US:ASMB)2025-11-10 21:15Clinical Development - The company is developing multiple clinical-stage investigational therapies targeting serious viral diseases, including two helicase-primase inhibitors (HPIs) for recurrent genital herpes and an orally bioavailable hepatitis delta virus (HDV) entry inhibitor[74]. - Interim results from the Phase 1b study of the HPI ABI-5366 showed a 94% reduction in HSV-2 shedding rate compared to placebo over a 29-day evaluation period with a 350 mg weekly dose[86]. - The Phase 1a study of ABI-5366 demonstrated a mean half-life of approximately 20 days, supporting potential once-weekly or once-monthly oral dosing regimens[84]. - The company has initiated both Phase 1a and Phase 1b studies for ABI-1179, another long-acting HPI, with positive interim data reported in February 2025[92]. - Enrollment for the Phase 1b portion of the ABI-5366 study is complete, with plans to move directly into Phase 2 clinical study preparation[89]. - The company plans to report interim data on two cohorts of weekly dosing of ABI-1179 and one cohort of monthly dosing of ABI-5366 by the end of 2025[94]. - The company is preparing for Phase 2 clinical studies for 6250 following the completion of the Phase 1a study[110]. Hepatitis B and D Virus Programs - The World Health Organization estimates that 254 million people are chronically infected with HBV, with only 3% of diagnosed patients receiving treatment[95]. - Current standard care for chronic HBV infection has not seen new mechanisms of action approved in over 25 years, highlighting the need for innovative therapies[96]. - The company is advancing its HBV program to improve patient outcomes and increase the number of patients diagnosed and treated through finite and curative therapies[97]. - HDV affects approximately 12 to 72 million HBV-infected individuals, accounting for 18% of cirrhosis and 20% of hepatocellular carcinoma associated with HBV[98]. - The novel small molecule entry inhibitor 6250 demonstrated low nanomolar potency against all tested HBV/HDV genotypes and good oral bioavailability, supporting once-daily dosing[103]. - In Phase 1a studies, 6250 showed a mean half-life of approximately four days, with exposures on the last day of dosing reaching six- to seven-fold higher than after the first dose[105]. - The 150 mg and 400 mg cohorts of the next-generation capsid assembly modulator 4334 observed mean declines in HBV DNA of 2.9 log10 IU/mL and 3.2 log10 IU/mL over 28 days, respectively[115]. - The first therapy approved for HDV, bulevirtide, is currently only available in Europe, highlighting the need for new treatments[99]. - The company has identified a development candidate, ABI-7423, in its broad-spectrum NNPI program targeting transplant-associated herpesviruses, transitioning to its parent molecule ABI-7272 for further studies[77]. Financial Performance - Collaboration revenue for the three months ended September 30, 2025, was $10.8 million, a 58% increase from $6.8 million in the same period in 2024, primarily due to higher costs incurred under the Gilead Collaboration Agreement[129]. - Research and development expenses for the three months ended September 30, 2025, totaled $16.6 million, up 23% from $13.5 million in 2024, driven by increased external program expenses and higher employee-related costs[133]. - General and administrative expenses increased by 19% to $5.1 million for the three months ended September 30, 2025, compared to $4.3 million in 2024, mainly due to higher professional fees and stock-based compensation[136]. - Interest and other income, net, was $1.7 million for the three months ended September 30, 2025, reflecting a 26% increase from $1.3 million in 2024, attributed to higher interest earned on marketable securities[137]. - For the nine months ended September 30, 2025, collaboration revenue reached $29.8 million, a 41% increase from $21.2 million in 2024, due to increased costs under the Gilead Collaboration Agreement[138]. - Total research and development expenses for the nine months ended September 30, 2025, were $47.6 million, a 14% increase from $41.7 million in 2024, driven by higher external program expenses related to ongoing clinical studies[141]. - External program expenses for the 5366 program increased by 82% to $7.2 million for the nine months ended September 30, 2025, compared to $3.9 million in 2024[139]. - General and administrative expenses increased to $14.2 million for the nine months ended September 30, 2025, up from $13.4 million in 2024, representing a 6% increase[142]. - Interest and other income, net decreased to $3.7 million for the nine months ended September 30, 2025, down from $4.5 million in 2024, a decline of 17%[144]. - Net cash used in operating activities was $55.3 million for the nine months ended September 30, 2025, compared to $50.7 million in 2024, indicating increased operational expenses[148]. - Net cash used in investing activities was $134.7 million for the nine months ended September 30, 2025, compared to net cash provided of $46.9 million in 2024, primarily due to purchases of marketable securities[149]. - Net cash provided by financing activities was $174.2 million for the nine months ended September 30, 2025, significantly higher than $12.5 million in 2024, due to larger proceeds from financing transactions[150]. - As of September 30, 2025, the company had an accumulated deficit of $854.1 million, with expectations of continued losses as product candidates are developed[151]. - The company held cash, cash equivalents, and marketable securities of $232.6 million as of September 30, 2025, with sufficient funds projected to meet operating requirements into late 2027[152]. - The company raised an aggregate of $821.8 million in net proceeds from equity financings and $200.9 million from strategic collaborations since incorporation[145]. - In August 2025, the company raised approximately $166.4 million from a financing transaction involving the sale of 5,591,840 shares of common stock and warrants[146]. - Future capital requirements will depend on various factors, including the progress of drug development and regulatory reviews[154]. Collaboration with Gilead - Gilead made an upfront cash payment of $84.8 million as part of the collaboration agreement, with potential regulatory and commercial milestones up to $330 million per program[121][122]. - The opt-in fee for Gilead to exercise its rights ranges from $45 million to $125 million per program, depending on the clinical stage[121]. - Gilead made an upfront equity investment of $15.2 million by purchasing 1,089,472 shares at $13.92 per share, with an option to purchase additional shares to reach 29.9% ownership[125]. - Gilead's participation in the August 2025 financing transaction included the purchase of 2,295,920 shares at $19.60 per share, along with warrants for additional shares[127]. - The company filed a Registration Statement on Form S-3 with the SEC to register shares issued to Gilead, as required under the Investor Rights Agreement[128].