Rare neurodegenerative disease

Resetting the immune response that drives neurodegeneration.

Omexia Therapeutics is developing OMX-002, an oral small-molecule prodrug of an autacoide that recalibrates microglial signalling — the shared inflammatory driver behind a group of rare disorders of the central nervous system.

3  orphan indications 1  shared mechanism Oral small molecule Italian innovative startup
OMX-002
Activated microglia · neuroinflammation Sentinel · homeostatic surveillance

Why rare disease

Focus on rare neurodegenerative disease.

There are roughly 300 rare neurodegenerative disorders, and very few have any approved treatment. Most therapies in development chase one disease-specific misfolded protein at a time. Omexia Therapeutics takes the opposite route: we target the neuroinflammatory, microglia-driven mechanism these diseases share — a single approach with the potential to reach several orphan indications, each with the development advantages of orphan drug status.

~300
rare neurodegenerative disorders, most without a disease-modifying therapy.
~3M
people in the EU and US live with a rare neurodegenerative disease.
0
approved disease-modifying therapies for our lead indications — today's care is symptomatic at best.
1
shared microglial mechanism across our three lead programmes.

Scientific rationale

Microglia: an increasingly central target in neurodegeneration.

Once regarded as a late bystander, chronic microglial activation is now recognised as a core driver of neurodegenerative disease — and a leading focus for therapeutic intervention. That shift in the literature is the foundation for our approach.

Human genetics and disease pathology point to the immune system — and microglia in particular — as a central influence on neurodegenerative disease progression, and the primary focus of neuroimmune therapeutic targeting.

Reviewed in Nature Reviews Drug Discovery 25:390–405 (2026) · 10.1038/s41573-025-01370-7

Approach

OMX-002 resets activated microglia.

Microglia are the brain's resident immune cells. In their resting state they are ramified — finely branched sentinels that continuously survey the brain. In neurodegenerative disease they retract into an amoeboid, chronically activated form that sustains inflammation and damages neurons. OMX-002 delivers a regulatory autacoide into the central nervous system, recalibrating the intracellular signalling that governs this switch and helping microglia return toward their homeostatic, surveying state — without broad immunosuppression.

  • 1Trigger. Disease-associated signals drive microglia from ramified surveillance into an amoeboid, activated state.
  • 2Amplification. Sustained inflammatory signalling releases mediators that stress and damage neurons.
  • 3Reset. OMX-002's active moiety recalibrates the signalling that maintains activation, favouring a return to ramified, homeostatic surveillance.
  • 4Delivery. As an oral small-molecule prodrug, OMX-002 is designed for good CNS exposure with a simple, non-invasive route of administration.

Safe and sustainable by design. OMX-002 is a small-molecule prodrug made through biotechnological processes, and its path toward the clinic is accelerated by AI-guided development.

Investigational · Preclinical stage · Mechanism illustrative

OMX-002
Activated · amoeboidSentinel · ramified

Pipeline

One mechanism across three genetically defined rare diseases.

Frontotemporal dementia
GRN mutation
Lead
Preclinical
Orphan · onset 50–65 yrs · no approved therapy
ResearchPreclinicalClinical
Amyotrophic lateral sclerosis
TDP-43
Co-development
Preclinical
Orphan · preclinical proof-of-mechanism confirmed
ResearchPreclinicalClinical
Parkinson's disease
GBA1 / LRRK2
Track 2
Preclinical
Genetically defined · preclinical proof-of-mechanism (GBA1-PD)
ResearchPreclinicalClinical

Preclinical proof-of-mechanism generated across three independent genetic models · Stages: Research → Optimization → Preclinical → IND-enabling → Clinical

Collaborations

Built with Italy's rare-disease and neuroscience research network.

Team

Founders.Decades of biotech and CNS drug development.One commitment: taking OMX-002 into the clinic.

Founder- and woman-led — a rare profile in deep-tech biotech
Elena Sgaravatti

Co-Founder · CEO

Elena Sgaravatti

LinkedIn ↗

Biotech entrepreneur with 25+ years across pharma and biotech and a strong focus on neuroscience partnering and translational strategy. She began at Fidia Farmaceutici and joined GlaxoWellcome/GSK in Licensing & Business Development, working with the UK Neuroscience Centre of Excellence and leading partnerships across neuroscience and vaccines.

She later served as CEO of IRB, driving a major turnaround through its acquisition by Croda International. She is Vice President of Assobiotec and a Contract Professor at the University of Padova, Department of Molecular Medicine. At Omexia Therapeutics she leads execution and capital strategy toward IND/CTA readiness.

Emilio Merlo Pich

Co-Founder · Chief Scientific Officer

Emilio Merlo Pich, PhD

LinkedIn ↗

Neurologist and neuropharmacologist with an academic background and 25+ years of pharma R&D across GSK, Hoffmann-La Roche, Takeda and Alfasigma, where he served as Global Head of R&D. His expertise spans target-to-clinic execution, translational models, biomarkers, and IP and regulatory strategy.

Author of 200+ scientific publications and an ACNP Fellow, he is the founder of Gelf Health and works at the intersection of AI and neurodegeneration prevention. At Omexia Therapeutics he drives scientific strategy and translational decisions toward early clinical proof-of-concept.

Advisory & scientific network — Riccardo Palmisano (Industry Advisor; former CEO of MolMed, President of Assobiotec 2016–2022) · Prof. Laura Calzà (Fondazione IRET).

Where we are

Two sites, one team.

Omexia Therapeutics

Neurotherapeutics for rare disease, developed from Italy.

Incorporated in July 2026 as an Italian innovative startup, we are advancing OMX-002 toward first-in-human studies. We welcome conversations with investors, pharmaceutical partners and patient organisations.

Get in touch