MDL-001 has demonstrated in vivo proof of concept (PoC) against three viral families with in vitro efficacy against six viral families in total.
San Diego, California - August 25, 2025 — Model Medicines today announced that it has been selected for an oral presentation at IDWeek 2025, where, it will unveil the complete preclinical Proof-of-Concept data package for MDL‑001, its oral broad-spectrum antiviral clinical candidate.
● Preclinical Milestones: MDL-001 has demonstrated in vivo proof of concept (PoC) against three viral families and in vitro efficacy against six, making it the most expansive oral broad-spectrum antiviral candidate to date.
● Advancing Toward Clinical Trials: Model Medicines is completing IND-enabling studies, has initiated a GMP manufacturing program, and is planning regulatory filings in 2026, with clinical trial initiation immediately following IND clearance.
● New Era of Virology: MDL-001 is a first-in-category, non-nucleoside antiviral that challenges traditional virus-specific treatment approaches. The drug is part of Model Medicines’ virology program that has demonstrated broad antiviral activity, from viruses causing acute respiratory disease to viruses causing chronic liver diseases, as well as emerging viral threats.
MDL‑001 is a non-nucleoside inhibitor of
RNA-dependent RNA polymerase (RdRp) that demonstrates consistent multi-log
reductions in viral load across multiple viral families in vivo.
New in vivo results build upon
preliminary in vitro studies in corona, influenza, and hepatitis
viruses and demonstrate broad-spectrum therapeutic potential applicable to
future pandemic response, stockpiling, and first-line monotherapy or
combination therapy for seasonal and chronic viruses. The data package
reflects reproducible efficacy in preclinical models generated at multiple
globally recognized research institutes, offering compelling support for the
compound’s advancement into clinical trials.
"As a clinician, we need an antiviral
that can be deployed the moment a patient presents. Like we do with
antibiotics, we should have something ready to start before we know exactly
what bug is causing the problem," said Davey Smith, MD, MAS, clinician and
virologist at UCSD. "MDL‑001 is likely that, an antiviral with
broad-spectrum activity that can be used as monotherapy or in combination with
current standards of care at patient presentation. The data we are presenting
at ID Week will, for the first time, publicly demonstrate its potential to
reshape frontline clinical decision-making.”
“This is the definitive preclinical dataset
we’ve been building toward,” said Daniel Haders II, Ph.D., Founder and CEO of
Model Medicines. “Clear, reproducible, in vivo antiviral activity across
diverse viral families, strong differentiation in mechanism, compelling pharmacokinetics
and safety data and a direct path to clinical development. As we look ahead to
regulatory filings in 2026 and human trials immediately following IND
clearance, we’re confident MDL‑001 has the potential to reshape how
broad-spectrum antivirals are designed and deployed.”
The presentation includes collaborations with
researchers at the Icahn School of Medicine at Mount Sinai, UC San Diego,
and Scripps Research, including virologists Dr. Adolfo García-Sastre,
Dr. Kris White, Dr. Davey Smith, and Dr. Philippe Gallay, who were
instrumental in the validation and characterization of MDL‑001’s antiviral
activity across diverse viral families.
“Most antiviral drug development remains
concentrated around a small set of viral targets, primarily proteases and
nucleoside analogs,” said Dr. Philippe Gallay, Professor of Immunology at
Scripps Research. “What differentiates MDL‑001 is the depth and breadth of the
preclinical data. There has never been an orally available antiviral that has
demonstrated multi-log viral load reductions across the breadth of viral
families that MDL-001 inhibits. That’s what makes this dataset unprecedented,
and what makes MDL‑001 a potential cornerstone of viral therapy.”
MDL‑001 has successfully achieved preclinical
proof-of-concept in multiple viruses and is completing IND-enabling studies.
Model Medicines has initiated a GMP manufacturing program, and is planning
regulatory filings in 2026, with clinical trial initiation immediately
following IND clearance. MDL-001 is being advanced in multiple indications due
to data that demonstrates the drug’s utility in multiple viral diseases.
Current Virology Standards of Care and
Clinical Need for Broad-Spectrum Therapeutics Current standards of care for major viral
infections remain narrowly targeted, with most therapies developed for
virus-specific indications and acting through a limited number of mechanisms.
For respiratory viruses, treatment paradigms vary but remain reactive and
indication-specific. COVID-19 management relies on Veklury® (remdesivir),
Paxlovid® (nirmatrelvir/ritonavir), and Lagevrio® (molnupiravir). Influenza
treatment options include neuraminidase inhibitors such as Tamiflu®
(oseltamivir) and endonuclease inhibitors like Xofluza® (baloxavir marboxil).
Respiratory syncytial virus (RSV) is typically managed through monoclonal
antibody prophylaxis and select fusion inhibitors, though therapeutic options
for active treatment remain limited. Chronic viral liver infections are
similarly segmented. Hepatitis B virus (HBV) is managed using nucleos(t)ide
analogs like Viread® (tenofovir disoproxil fumarate) and Baraclude®
(entecavir), while hepatitis C virus (HCV) is treated with direct-acting
antivirals such as Harvoni® (sofosbuvir/ledipasvir). For hepatitis D virus
(HDV), therapeutic choices are minimal, primarily pegylated interferons, which
have suboptimal efficacy and significant tolerability issues. Despite
therapeutic success in isolated indications, these treatments do not address
the broader threat landscape: co-infections, zoonotic spillover, rapid viral
mutation, and limited coverage in outbreak response. A first-line, orally
available, broad-spectrum antiviral that can be deployed at the point of care
remains an unmet medical and strategic imperative.
About Model Medicines Model Medicines is an AI-native pharmaceutical
company developing novel small molecules for infectious disease, oncology, and
inflammation. Its proprietary GALILEO™ platform combines generative AI,
phenotypic profiling, and predictive toxicology to rapidly design and
prioritize therapeutic candidates. The pipeline includes 67 validated assets in
cellular models of disease across 12 therapeutic targets for multiple areas of
biology. Learn more at www.modelmedicines.com
