ABI-6250

Plus, news about Assembly Biosciences, Vaxcyte, CorMedix, Melinta Therapeutics, Innogen, Sana Biotechnology, SNIPR Biome, Voyager Therapeutics, Gilead, Arcus, Travere and Alltrna: XtalPi to work with DoveTree Medicines: The Chinese AI drug discovery company got $51 million upfront, so that DoveTree can get global R&D and commercial rights for drugs made from the partnership. There’s an additional $49 million of near-term payments on the table, as well as milestones and royalties that could balloon up to $5.9 billion. The disease targets include immunology, inflammation, neurology, oncology and metabolic disease. DoveTree was founded by biotech veteran Greg Verdine. — Reynald Castaneda Regenxbio amends eye deal with AbbVie: The companies first teamed up in 2021 to examine surabgene lomparvovec as a treatment for several eye diseases. The biotech now plans to initiate a Phase 2b/3 study in diabetic retinopathy, noting that it will receive $100 million when the first patient is dosed in that trial and another $100 million if a patient is dosed in the second Phase 3 study. Regenxbio is responsible for paying for the Phase 2b portion of the trial, while AbbVie will cover the costs of a new Phase 3 trial for sura-vec in wet AMD. — Jaimy Lee Assembly Biosciences sees safety signal in hepatitis delta study: The company said a grade 2 liver enzyme elevation was recorded in a healthy volunteer who received the highest single dose level of ABI-6250, its experimental hepatitis delta virus entry inhibitor. Assembly said that “further pharmacological assessment” would be done as it prepares for Phase 2 studies. The company noted the studies would “explore potential factors associated with these elevations given the role of ABI-6250’s target as a bile acid transporter.” — Lei Lei Wu Vaxcyte prunes early-stage pipeline: The company is pausing the advancement of two prophylactic vaccine candidates into the clinic — VAX-A1 for strep infections and VAX-GI for shigella. It is also discontinuing a therapeutic vaccine called VAX-PG for periodontal disease. Vaxcyte said the changes should allow it to focus on its pneumococcal conjugate vaccine. — Ayisha Sharma CorMedix to buy Melinta Therapeutics: The New Jersey biotech companies will come together in a deal that sees CorMedix pay $260 million in cash and $40 million in equity to “Melinta shareholders of Deerfield Management Company.” Deerfield acquired Melinta pursuant to a Chapter 11 reorganization about five years ago. CorMedix said the deal will expand its “reach in infectious disease.” — Kyle LaHucik Innogen’s Hong Kong IPO: The biotech expects proceeds of HK$610 million (about $77 million) from its initial public offering, according to an HKEX filing . The 11-year-old biotech markets a type 2 diabetes drug in China and is also working in MASH, obesity and other areas. DualityBio , Hengrui Pharma and other biotechs have gone public on the Hong Kong exchange this year. — Kyle LaHucik Sana Biotechnology’s $75M offering: The biotech is selling about 21 million shares at $3.35 apiece. — Jaimy Lee SNIPR Biome raises $40.7M Series B: The Copenhagen-based biotech will use the funds to support the development of a CRISPR/Cas therapy for airway infections caused by Pseudomonas aeruginosa bacteria in cystic fibrosis patients, among other programs. The fundraise was supported by both new and existing investors. — Ayisha Sharma Voyager Therapeutics restructures: The company executed “cost-savings measures” in the first half of the year that included changes in trial designs, outsourcing and an undisclosed number of workforce cuts, according to a SEC filing . The move extends its cash runway into 2028, as per a press release . Voyager had $262 million in cash, cash equivalents and marketable securities as of June 30. It had 172 staffers at the end of 2024. — Reynald Castaneda Gilead drops Arcus cancer drug: The partners have decided not to advance etrumadenant into registrational development for colorectal cancer, despite the FDA confirming a potential path forward. Gilead returned its license for the A2a/A2b receptor antagonist in June. It paid $725 million to exercise options for etrumadenant and several other Arcus drugs back in 2021. — Ayisha Sharma Travere gets $17.5M from CSL Vifor: The milestone payment stems from Filspari’s conversion from a conditional to standard approval in the EU and UK. Filspari is Travere’s treatment for IgA nephropathy, which received conditional approvals in Europe last year. The drug also won full FDA approval in 2024, following an initial accelerated approval in 2023. — Lei Lei Wu Flagship’s Alltrna lays off 12% of staff: The biotech let go of eight employees as it aims to get its engineered tRNA drug candidate into the clinic. “Our strategy and vision remain unchanged,” an Alltrna spokesperson said in a statement. — Jaimy Lee

– Data to date in single- and multiple-ascending dose cohorts of healthy participants demonstrate a four-day half-life for ABI-6250, supporting target daily oral dosing profile and dose-dependent elevations in biomarker of target engagement – – Further pharmacological assessment to be conducted in parallel with Phase 2 preparation – SOUTH SAN FRANCISCO, CA, USA I August 06, 2025 I Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative therapeutics targeting serious viral diseases, today announced interim data from several cohorts from the ongoing Phase 1a clinical trial of ABI-6250, the company’s orally bioavailable, small molecule hepatitis delta virus (HDV) entry inhibitor candidate. In the cohorts evaluated to date, a mean half-life of four days was observed for ABI-6250 when dosed orally, supporting the target daily oral dosing profile. Additionally, dose-dependent elevations of total serum bile acids (TBA) were seen over both single- and multiple-dose cohorts. These biomarker data indicate ABI-6250’s potent engagement of the sodium taurocholate cotransporting polypeptide (NTCP) target, the receptor used by HDV to infect hepatocytes, and inform the potential dose range for evaluation of antiviral activity in further studies in participants with chronic HDV (cHDV) infection. As the study remains blinded, combined safety data for ABI-6250 and placebo recipients are reported. A grade 2 alanine transaminase (ALT) elevation was observed in the cohort evaluating the highest single-dose level, and grade 1 elevations were seen at a low frequency in the other cohorts. All were self-limited and none were accompanied by bilirubin elevations or other markers of liver injury. Additional studies are planned to explore potential factors associated with these elevations given the role of ABI-6250’s target as a bile acid transporter. This assessment is expected to include further pharmacological characterization of ABI-6250 and may incorporate additional cohorts in this Phase 1a study. Assembly Bio expects to conduct these activities in parallel with preparations for Phase 2 clinical studies and the completion of ongoing chronic toxicology studies. “In this first clinical study of ABI-6250, we are encouraged by the observed half-life supportive of once-daily oral dosing and bile acid elevations indicative of NTCP target engagement,” said Anuj Gaggar, MD, PhD, chief medical officer of Assembly Bio. “As we continue to evaluate these Phase 1a clinical data, we plan to further explore ABI-6250’s pharmacologic and biologic profile in preparation for proceeding to further clinical studies in participants with cHDV infection. With just one approved therapy for cHDV in the European Union and none in the U.S., the need for new treatment options for this underserved patient population remains critical – a need we believe ABI-6250 could have an important role in addressing given its potential to be the first oral therapy.” ABI-6250 is an investigational product candidate that has not been approved anywhere globally, and its safety and efficacy have not been established. Study ABI-6250-101 – Phase 1a Interim Results Study Overview ABI-6250-101 is an ongoing randomized, blinded and placebo-controlled Phase 1a clinical study evaluating the safety, tolerability and pharmacokinetics (PK) of ABI-6250 following single- and multiple-ascending oral dose administration in healthy participants. Changes in TBAs are also evaluated as a biomarker for NTCP engagement. Dosing is complete in two single-dose cohorts evaluating doses of 5 mg and 25 mg and three multiple-dose cohorts evaluating doses of 0.05 mg, 0.2 mg and 1 mg, each randomized 6:2 between ABI-6250 and placebo. Multiple-dose cohorts evaluated repeat daily dosing over 10 days. Data are reported here for all cohorts dosed to date through completion of the study follow-up period of 10 days after dosing. As the study remains blinded, combined safety data for ABI-6250 and placebo recipients are reported. Results PK: Across the cohorts evaluated to date, a mean half-life of approximately four days was observed for ABI-6250 when dosed orally, supporting the once-daily oral dosing profile target. Given this half-life, accumulation was observed in the multiple-dose cohorts with exposures on the last day of dosing generally reaching 6- to 7-fold higher than the exposure seen after the first dose. Biomarkers : Dose-dependent elevations of TBAs were observed for both the 5 mg and 25 mg single-dose cohorts, indicative of NTCP target engagement. In the highest single-dose cohort of 25 mg, coproporphyrin I (CP-1), a biomarker for off-target engagement of the organic anion transporters, OATP1B1 and/or OATP1B3, was also elevated. Given the predicted ABI-6250 accumulation driven by the long half-life and the observed elevations in TBAs for the single-dose cohorts, doses at and below 1 mg daily were selected for the multiple-dose cohorts to characterize the lower end of the dose-response curve. Elevation of TBAs was observed for both the 0.2 mg and 1 mg daily multiple-dose cohorts, consistent with the respective ABI-6250 exposures. Minimal TBA elevation was observed in the 0.05 mg daily multiple-dose cohort. Safety: Treatment-emergent adverse events (AEs) and laboratory abnormalities were all grade 1 or 2 in severity with the majority being grade 1. There were no serious AEs in any dose cohort. No protocol defined stopping criteria were met. There were no clinically significant electrocardiogram abnormalities or patterns of AEs noted. One grade 2 ALT elevation was observed in the cohort evaluating the highest single-dose level of 25 mg. In this cohort, off-target engagement of other liver transporters was also seen as indicated by elevated CP-1 levels. Grade 1 ALT elevations were observed at a low frequency across the other cohorts. All ALT elevations were self-limited, and none were accompanied with elevations in bilirubin or other markers of liver injury. Additional information about the Phase 1a trial is available at clinicaltrials.gov using the identifier NCT06740474. Assembly Bio expects to submit complete data from the trial for presentation at future scientific meetings. About Assembly Biosciences Assembly Biosciences is a biotechnology company dedicated to the development of innovative small-molecule therapeutics designed to change the path of serious viral diseases and improve the lives of patients worldwide. Led by an accomplished team of leaders in virologic drug development, Assembly Bio is committed to improving outcomes for patients struggling with the serious, chronic impacts of herpesvirus, hepatitis B virus (HBV) and hepatitis delta virus (HDV) infections. For more information, visit assemblybio.com. SOURCE: Assembly Biosciences