Business Context and Reporting Period
Company: Assembly Biosciences, Inc.
Filing Type: Form 8-K (Current Report)
Date: April 12, 2018
Subject: Announcement of positive interim data for ABI-H0731, the Company's lead product candidate for a hepatitis B virus (HBV) cure. The data covers Phase 1a (safety/pharmacokinetics) and Phase 1b (antiviral efficacy) clinical studies.
Key Financial Metrics
This filing is a Current Report (Form 8-K) regarding clinical trial results and does not contain financial statements. Consequently, data regarding revenue, profit, cash flow, margins, debt, and liquidity are not provided in this document.
Material Changes and Clinical Results
The filing reports significant clinical progress for ABI-H0731, a novel oral Core protein Allosteric Modifier (CpAM):
- Antiviral Efficacy: Potent, dose-dependent antiviral activity was observed. In the ongoing 300 mg cohort, the mean overall decline from baseline was ≥2.8 log 10 IU/mL. Maximal viral load declines of 3.6 to 4.0 log 10 IU/mL were observed in certain HBeAg negative patients across dose levels (100 mg to 400 mg).
- Safety Profile: The drug was generally safe and well-tolerated across 62 patients and volunteers. No serious adverse effects or dose-limiting toxicities were identified. All treatment-emergent adverse events were minor (Grade 1), except for one isolated Grade 3 rash at the 400 mg dose which resolved rapidly.
- Study Status: Two cohorts (100 mg and 200 mg) of HBV patients completed dosing in Phase 1b. A third cohort (300 mg) is ongoing. Two HBeAg negative patients were treated at 400 mg.
Guidance, Outlook, and Risks
Future Plans:
- The Company intends to advance ABI-H0731 into Phase 2a combination studies using a 300 mg dose, expected to begin in the summer of 2018.
- Two Phase 2a studies are planned: one enrolling HBeAg positive patients on standard of care therapy to demonstrate inhibition of cccDNA generation, and a second enrolling treatment-naïve HBeAg positive patients to compare standard of care alone versus combination therapy.
- Results from these Phase 2a studies are anticipated during the first half of 2019.
- Complete results from the Phase 1b study will be reported at a scientific conference later in 2018.
Risks and Contingencies:
- The filing contains forward-looking statements regarding clinical potential, trial progress, and timing. Actual results may differ materially from projections.
- Interim data reliability to predict completed study results is noted as a risk factor.
- Further details on risks are referenced in the Company's Annual Report on Form 10-K for the year ended December 31, 2017.
Investor Verification Checklist
- Verify the full text of the poster presentation at The International Liver Congress (EASL) for complete interim data details.
- Confirm the specific enrollment criteria and primary endpoints for the upcoming Phase 2a studies scheduled for summer 2018.
- Review the "Risk Factors" section of the 2017 Form 10-K for a comprehensive list of uncertainties regarding clinical development.
- Monitor future announcements for the complete Phase 1b results expected later in 2018.