What Eloxx does

Eloxx Pharmaceuticals is a clinical-stage biopharmaceutical company developing small-molecule therapies that restore full-length protein production in patients with rare genetic diseases caused by nonsense mutations. Its lead compound, exaluren, is a novel eukaryotic ribosome-selective glycoside designed to selectively modulate the human ribosome at premature stop codons, enabling near-cognate aminoacyl-tRNA insertion and continued translation in place of the truncated peptides produced by nonsense alleles. Exaluren is administered subcutaneously and concentrated in kidney cells via megalin receptor-mediated uptake, supporting a precision nephrology positioning across two indications: nonsense mutation Alport syndrome (NMAS), currently in the EXACT Phase 2b study (NCT07523581), and nonsense mutation autosomal dominant polycystic kidney disease (NM-ADPKD), in Phase 2 planning. A second asset, ZKN-013, has been exclusively licensed to Almirall, S.A. for rare dermatological diseases (RDEB and JEB) and is in Phase 1 development.

The company's underlying technology is a gene-agnostic readthrough platform: by targeting the ribosome rather than any single gene, the same mechanism can be applied across COL4A3, COL4A4, and COL4A5 (Alport), PKD1 and PKD2 (ADPKD), and COL7A1, LAMA3, and COL17A1 (dermatology). The selectivity profile for human versus mitochondrial and bacterial ribosomes is intended to avoid the ototoxicity and nephrotoxicity associated with aminoglycoside-class antibiotics, addressing the historical safety ceiling of nonsense suppression approaches. Recent proof-of-concept data showed 100% (3/3) biopsy-confirmed disease regression in NMAS patients treated with ELX-02, with new protein analyses confirming production of both collagen α3 and α4/α5 post-treatment.

Eloxx is pre-revenue with no commercialized products and no public pricing. The business model is a hybrid of clinical development for retained indications (exaluren for NMAS and NM-ADPKD) and out-licensed development for non-core assets (ZKN-013 to Almirall, with $3 million upfront and up to $470 million in potential milestones plus tiered royalties). The company is publicly traded on the Nasdaq Capital Market under ticker ELOX following a $66.0 million offering in June 2026 and is headquartered in Waltham, Massachusetts. Customer-facing activity today is concentrated in clinical trial recruitment through patient advocacy partnerships (Alport Syndrome Foundation, PKD Foundation), scientific/medical engagement at nephrology conferences (ASN Kidney Week), and investor communications rather than commercial promotion.

Name

Eloxx

Legal name

Eloxx Pharmaceuticals, Inc.

Website

https://eloxxpharma.com

Company type

Public

Founded year

2013

Operating status

Operating

Headcount range

11–50 employees

Short description

Eloxx Pharmaceuticals is a clinical-stage biopharmaceutical company developing exaluren, a small-molecule ribosome modulator that induces premature stop codon readthrough to restore full-length proteins in patients with nonsense mutation Alport syndrome and ADPKD. The company is pre-revenue and Nasdaq-listed.

Ownership category

akta.pro rank

Product category

Clinical-stage biopharmaceuticals (rare disease therapeutics)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Enzyme Replacement & Metabolic Disorder Therapies (HLAIAIAB)

Keywords

• Rare kidney disease therapeutics
• Nonsense mutation readthrough
• Premature termination codon therapy
• Ribosome-selective glycoside platform
• Clinical-stage biopharmaceutical

Headquarters

HQ city

Waltham

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Pharmaceutical Product Sales: Pre-revenue clinical-stage company. Anticipated revenue from commercialization of investigational therapies (exaluren, ZKN-013) following regulatory approval. Future revenue model will likely follow specialty pharmaceutical distribution through pharmacy channels upon FDA approval.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels4 records

Core offering

Eloxx is developing exaluren, a novel eukaryotic ribosome-selective glycoside designed to induce premature termination codon readthrough and restore full-length functional proteins in patients with rare genetic diseases caused by nonsense mutations. The lead program is in the EXACT Phase 2b trial for nonsense mutation Alport syndrome, with a Phase 2 study planned for nonsense mutation ADPKD. A second asset, ZKN-013, has been exclusively licensed to Almirall for rare skin diseases including RDEB and JEB.

Product overview

Eloxx is a clinical-stage biopharmaceutical company developing a single core compound, Exaluren, across two genetically defined kidney indications: nonsense mutation Alport syndrome (NMAS) and nonsense mutation autosomal dominant polycystic kidney disease (NM-ADPKD). Exaluren is a novel eukaryotic ribosome-selective glycoside designed to induce premature termination codon readthrough, restoring full-length protein production. The company also has a second compound, ZKN-013, which has been exclusively licensed to Almirall, S.A. for rare dermatological diseases (RDEB and JEB). Both programs share the company's gene-agnostic nonsense mutation readthrough technology platform.

Differentiator

Problem solved

Functional benefit

Brands

• Exaluren: A small molecule therapy designed to induce premature termination codon readthrough to restore full-length proteins, being developed for nonsense mutation Alport syndrome and ADPKD.

Products and services

• Exaluren Exaluren is a novel eukaryotic ribosome-selective glycoside (ERSG) product candidate designed to induce premature termination codon readthrough at nonsense mutations and promote the restoration of full-length functional proteins. Administered subcutaneously, it is transported and concentrated into kidney cells via megalin receptor-mediated uptake. It is being developed for nonsense mutation Alport syndrome (COL4A3/COL4A4/COL4A5) and nonsense mutation ADPKD (PKD1/PKD2).
• ZKN-013 ZKN-013 is a ribosome modulating agent exclusively licensed to Almirall, S.A. for the development and commercialization of treatments for rare skin diseases caused by nonsense mutations, including recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB), targeting COL7A1, LAMA3, and COL17A1 nonsense mutations. Almirall is responsible for all development and commercialization; Eloxx receives $3M upfront, up to $470M in milestones, and tiered royalties.

Quantifiable outcome

• 100% response rate (3/3 Alport patients treated with ELX-02 had biopsy-confirmed disease regression)
• +2 more outcomes

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

One partnership is on record.

• Almirall, S.A.coreOEM/ Whitelabel/ Licensing Partner · 13 March 2024Eloxx has exclusively licensed ZKN-013 to Almirall, S.A. for the development and commercialization of treatments for rare skin diseases caused by nonsense mutations, including RDEB (Recessive Dystrophic Epidermolysis Bullosa) and JEB (Junctional Epidermolysis Bullosa). Eloxx receives $3 million upfront payment and is eligible for up to $470 million in development and commercial milestones plus tiered royalties on global sales. Almirall is responsible for all development and commercialization activities.

Scale indicators6 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• Sarepta Therapeutics: Commercial-stage rare disease company developing RNA-targeted and gene therapies for Duchenne muscular dystrophy and other genetic diseases. Highly comparable to Eloxx as a rare genetic disease biotech pursuing novel RNA/protein-restoration mechanisms with specialty distribution.
• Krystal Biotech: Rare disease biotech with an approved topical gene therapy for dystrophic epidermolysis bullosa (EB). Directly comparable to Eloxx's ZKN-013 program targeting RDEB/JEB nonsense mutations and represents the competitive benchmark in EB.
• BridgeBio Pharma: Clinical-to-commercial rare genetic disease company with programs in nephrology (e.g., atrasentan in IgA nephropathy) and other inherited conditions. Closely comparable as a rare-genetic-disease platform with kidney-focused assets.
• Travere Therapeutics: Rare disease company with a commercial and late-stage pipeline in kidney diseases (FSGS, IgA nephropathy). Comparable to Eloxx's nephrology focus and rare-disease commercial pathway planning.
• Abeona Therapeutics: Clinical-stage rare disease biotech with programs in epidermolysis bullosa and other genetic conditions. Comparable as an EB-focused player competing with ZKN-013 (via Almirall) for the same RDEB/JEB patient populations.

Broad incumbents

• Vertex Pharmaceuticals: Established rare-disease leader with the dominant cystic fibrosis franchise (Trikafta). Historically funded Eloxx-adjacent nonsense mutation CF research and represents the broad incumbent comparator for specialty rare-disease commercialization.
• Ionis Pharmaceuticals: Broad RNA-targeted therapeutics platform with multiple approved rare-disease antisense drugs. Comparable to Eloxx as an RNA/protein-modulation platform addressing genetic diseases, though at much larger scale.
• Chinook Therapeutics (Novartis): Rare kidney disease biotech (acquired by Novartis) developing therapies for IgA nephropathy and other kidney conditions. Comparable to Eloxx as a kidney-focused rare-disease developer, now operating as part of a large incumbent.

Emerging players

• Disc Medicine: Clinical-stage rare genetic disease biotech developing therapies for hematologic disorders. Comparable as a similarly staged rare-genetic-disease company pursuing novel mechanism-based therapies for small patient populations.
• ProKidney: Clinical-stage cell therapy company focused on kidney disease (diabetic kidney disease, CKD). Comparable as an emerging nephrology-focused biotech addressing rare/serious kidney indications through novel mechanisms.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat3 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/eloxxpharma

Revenue estimate

Valuation estimate

Number of profiles

Profiles5 records

Funding overview

Funding rounds11 records

Investors13 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A2 records

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Eloxx