One disease.
One drug.
One decade.
One billion dollars.
Metabolism sits at the center of many diseases, from rare genetic disorders to obesity, neurodegeneration and aging. Many share the same broken pathways, yet medicines are still developed one disease at a time, each taking over a decade and $1B+ to reach patients. This approach doesn't scale.Most metabolic diseases still have no approved medicine.
Metabolic diseases aren't as different as they appear.
Metabolic diseases are genetically different, yet many disrupt the same metabolic hubs. A small set of naturally occurring metabolites can restore these hubs—and treat many diseases. We call them keystone metabolites: shaped by billions of years of evolution to work with human biology.
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One keystone medicine. Many diseases.
Metabolis is the first biotech discovering and developing keystone medicines across all diseases of metabolism.
Why now
01
Shared biology
The same metabolites restore cellular function across different metabolic diseases, revealing a new drug class.
02
AI-powered clinical studies
Clinical data can now be generated at a fraction of traditional trial cost, through AI-powered, at-home patient studies designed to capture real-world patient outcomes.
03
Scalable discovery
Our yeast-based platform can discover keystone metabolites across hundreds of metabolic diseases.
Our platform
Discovering keystones. Developing medicines.
Every experiment makes the next discovery faster. Our platform screens metabolites across hundreds of yeast disease models, tests rescue in yeast patient avatars and runs AI-powered at-home clinical studies — generating data faster and cheaper than traditional trials. Every result trains predictive models for the next keystone metabolite, the next disease cluster, and the patients most likely to respond.
Step 01
Discover
Screen metabolites across hundreds of yeast disease models to identify keystone candidates across metabolic diseases.
Step 02
Prove
Test rescue in yeast patient avatars and AI-powered, at-home clinical studies — generating regulatory-quality evidence at a fraction of traditional trial cost.
Step 03
Develop
Advance keystone metabolites into medicines across many metabolic diseases — stacking programs, approvals, and learnings.
3 years
From discovery to clinical trial
See how our lead program went from yeast screen to Phase 1 in three years.
DHDDS-CDG is our proof of concept: the same yeast screening platform that found NMN scales across hundreds of metabolic diseases, identifying the next keystone metabolites and disease clusters.
NMN (nicotinamide mononucleotide) is our first keystone metabolite in clinical development as a medicine for DHDDS-CDG — a rare and progressive pediatric neurological disease with no approved treatment.
In yeast models
NAD+‑boosting molecules restore growth in a yeast model of DHDDS-CDG — identifying NMN as a candidate keystone metabolite for DHDDS-CDG.
In patient organoids
NMN restores coordinated brain network activity in DHDDS-CDG patient brain organoids — validating the mechanism in human biology.
In patients
Functional movement improvement observed in patients following 6 months of daily NMN treatment. A Phase 1 clinical trial is now underway.
Muffels et al., 2026 (accepted, Brain). Preprint here.
DHDDS-CDG Patient Response to NMN
Before NMN
Rosie_0M_tandem_short.mov
6 months on NMN
Rosie_6M_tandem_short.mov
Path to approval
NDA submission by 2028
Q2 2026
ODD & RPDD Filed
Orphan Drug Designation and Rare Pediatric Disease Designation applications submitted.
Q3 2026
First Patient Dosed — Phase 1
Phase 1 safety and tolerability study underway in DHDDS-CDG patients. NCT#07572825
Q1 2027
Phase 1 Data Readout
6-month safety, tolerability, and functional data readout informs Phase 2/3 trial design.
Q2 2027
GMP Manufacturing & CMC Finalized
GMP drug product manufacturing, formulation and testing finalized for Phase 2/3 pivotal study.
Q3 2027
First Patient Dosed — Phase 2/3
12-month AI-powered, at-home pivotal study begins. Designed to capture real-world patient outcomes at a fraction of traditional trial cost.
Q3 2028
Data Readout & Analysis
Phase 2/3 data analyzed and pivotal data package assembled.study analyzed.
Q4 2028
NDA Submission
NDA package submitted. Rare Pediatric Disease voucher eligibility established at approval — a saleable asset that funds the next program.
Orphan drug market exclusivity, stackable across multiple disease indications
2 years & $5M
FDA approval for NMN in DHDDS-CDG
Beyond metabolic disease. The longevity thesis.
Metabolic disease is our proving ground — and metabolic dysfunction sits at the root of nearly every major driver of aging. When a keystone medicine rescues a severely broken metabolic pathway, it validates the biology under the most demanding conditions possible. The same metabolites that repair broken metabolism in disease are the ones that will matter for longevity and healthy aging in everyone.
Team
The team behind a new class of metabolic medicines.
We’re scientists, biotech founders, and clinical investigators who’ve taken metabolic disease programs from discovery to the clinic.
Co-Founder & CEO
Kristin Kantautas, PhD
Kristin believes metabolic disease drug development can be fundamentally rebuilt: that targeting the shared pathways across diseases will unlock a new generation of therapies, no longer developed one disease at a time. As Co-Founder and CEO of Metabolis, she is building a new model for metabolic disease medicines, one designed to scale beyond the one-disease-at-a-time approach that has left most patients behind. Her discovery of NMN as a therapeutic candidate for DHDDS-CDG is now in Phase 1 clinical development, the company’s first program.
She has spent her career developing treatments for metabolic diseases. At Perlara, she led drug discovery and clinical translation programs for congenital disorders of glycosylation. At Cure SRD5A3, she led the translational research program, where she identified and advanced atorvastatin for SRD5A3-CDG into a global multi-site clinical study.
She also created CDG Hub, the first global knowledge platform for the congenital disorders of glycosylation ecosystem.
She holds a PhD in Molecular Genetics from the University of Toronto.
Co-Founder & CSO
Ethan Perlstein, PhD
Ethan pioneered the use of yeast-based models to find drugs for metabolic diseases, the same approach behind Metabolis’s first keystone metabolite.
He trained as a chemical biologist at Harvard and Princeton, then founded Perlara PBC in 2014 — the world's first biotech public benefit corporation and a Y Combinator company (W16), built to partner directly with rare disease families. He was also co-founder and CEO of Maggie's Pearl, a clinical-stage biotech developing treatments for PMM2-CDG.
At Metabolis, Ethan brings the scientific approach and the operating experience to take a metabolic disease platform from discovery into patients.
Study PI
Eva Morava-Kozicz, MD, PhD
Eva Morava-Kozicz is one of the world's leading clinician-scientists in inherited metabolic diseases, with over three decades of experience in the diagnosis, treatment, and clinical investigation of inborn errors of metabolism.
Professor of Genetics and Genomic Sciences at the Icahn School of Medicine at Mount Sinai, she has dedicated her career to the development of therapies for congenital disorders of glycosylation. She has led some of the first successful treatments in previously untreatable CDG types and serves as Principal Investigator of the Frontiers in CDG Consortium.
At Metabolis, Dr. Morava-Kozicz serves as Principal Investigator for the clinical studies of NMN in DHDDS-CDG patients, bringing decades of clinical expertise to the program.
Contact
Let's talk.
Metabolis is currently raising a pre-seed round.
We're looking for investors and partners who believe metabolic disease is the proving ground for the next generation of longevity medicines.
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