Abliva AB

Name

Abliva AB

Legal name

Abliva AB (publ)

Website

https://abliva.com

Company type

Private

Founded year

2000

Operating status

Acquired

Headcount range

1–10 employees

Short description

Abliva AB is a Swedish clinical-stage pharmaceutical company developing first-in-class therapies for primary mitochondrial diseases, with lead candidate KL1333 (NAD+/NADH modulator) in the Phase 2 FALCON study. As of July 2025 it operates as a wholly-owned subsidiary of Pharming Group N.V.

Ownership category

akta.pro rank

Product category

Mitochondrial disease therapeutics

NAICS

Pharmaceutical Preparation Manufacturing (325412)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

akta.pro secondary industry

Therapeutic Enzymes & Enzyme Replacement Therapies (ERT) (HLAAAAAK)

Keywords

• Mitochondrial disease therapeutics
• Orphan drug development
• Clinical stage pharmaceuticals
• Rare disease treatments
• NAD+ NADH modulators

Headquarters

HQ city

Lund

HQ country

Sweden

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Others

Revenue model

1. Pre-revenue clinical-stage pipeline: Abliva is a clinical-stage pharmaceutical company with no approved products and therefore no current product revenue. Revenue generation is expected post-regulatory approval of KL1333 (lead candidate) or other pipeline assets. As a wholly-owned subsidiary of Pharming Group N.V. since July 2025 (acquired during 2024), any future revenue will be consolidated within Pharming Group. Near-term revenue could include licensing/collaboration deals, milestone payments, or expanded access / compassionate use programs (although Abliva currently does not run any expanded access programs).

Go-to-market motion1 record

Distribution channels2 records

Marketing channels7 records

Core offering

Abliva AB is a clinical-stage pharmaceutical company developing first-in-class medicines for primary mitochondrial disease, an ultra-rare condition with no approved drug treatments (except Raxone for LHON in Europe/Israel). Its portfolio is led by KL1333, an oral NAD+/NADH modulator in the global Phase 2 FALCON study for fatigue and myopathy in adult patients with MELAS-MIDD, KSS-CPEO, and MERRF syndromes, supported by NV354, a brain-penetrable succinate prodrug for mitochondrial disease with neurologic complications (Leigh syndrome, MELAS, LHON), plus the earlier NeuroSTAT program and discovery-stage early programs. The company currently has no approved or marketed products and is pre-revenue, operating as a wholly-owned subsidiary of Pharming Group N.V. since July 2025.

Product overview

Abliva AB is a clinical-stage Swedish pharmaceutical company (since July 2025 a wholly-owned subsidiary of Pharming Group N.V.) that develops medicines for primary mitochondrial disease through an in-house R&D platform. Its offering is a drug-development pipeline (single unified R&D organization) rather than a multi-module software platform; the core assets are the late-stage clinical candidate KL1333 (NAD⁺/NADH modulator in the global Phase 2 FALCON study, with US/EU Orphan Drug Designation and US Fast Track designation), the preclinical succinate-prodrug candidate NV354 (Orphan Drug Designation in the US and EU; currently de-prioritized operationally in favor of KL1333), the earlier traumatic-brain-injury investigational product NeuroSTAT (cyclosporine-based cyclophilin inhibitor), and supporting Early Programs focused on regulation and stabilization of cellular energy production. KL1333 sits at the center of the portfolio as the registrational lead, NV354 and the Early Programs feed the pipeline, and NeuroSTAT represents an earlier-stage non-mitochondrial indication derived from Abliva's cyclophilin-inhibitor science.

Differentiator

Problem solved

Functional benefit

Products and services

• KL1333 Oral small-molecule drug candidate (NAD+/NADH modulator) being developed as a long-term treatment for consistent debilitating fatigue and myopathy in genetically confirmed adult patients with primary mitochondrial disease (MELAS-MIDD, KSS-CPEO, MERRF). Currently in the global Phase 2 FALCON study (180 patients, 3:2 randomized vs placebo, 48 weeks). Holds Orphan Drug Designation in the EU and US plus US FDA Fast Track designation.
• NV354 First-in-class, brain-penetrable succinate prodrug being developed for mitochondrial disease with neurologic complications caused by insufficient activity of mitochondrial respiratory complex I (e.g., Leigh syndrome, MELAS, LHON). Delivers the body's own energy substrate (succinate) into cells to bypass complex I deficiency. Preclinical stage with Orphan Drug Designation in the EU and US.
• NeuroSTAT Cyclophilin-inhibitor-based investigational medicinal product intended for administration in the acute phase (within hours) following a traumatic brain injury. Referenced in Abliva's expanded access policy as requiring product availability at hospital arrival, making individual expanded access requests not possible.

Quantifiable outcome

• Both primary endpoints of the FALCON Phase 2 study (PROMIS Fatigue Mitochondrial Disease Short Form and 30-second Sit-to-Stand test) passed futility in July 2024 interim analysis of Wave 1 patients.
• +3 more outcomes

Segments3 records

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Eight partnerships are on record, tiered flagship, core and minor.

• Pharming Group N.V.flagshipStrategic or Co-development Partner · 1 January 2024Pharming Group N.V. (Dutch specialty pharma) acquired Abliva in 2024. As of July 2025, Abliva is a wholly-owned subsidiary of Pharming Group. Abliva is listed on its website as 'Abliva AB, member of Pharming Group'. Pharming provides corporate backing and access to its commercial infrastructure; general inquiries redirect to [email protected]. Abliva's mitochondrial disease pipeline (KL1333, NV354) is integrated within Pharming's broader rare-disease portfolio alongside RUCONEST and Joenja (leniolisib).
• United Mitochondrial Disease Foundation (UMDF)coreGTM or Marketing PartnerUS patient organization promoting research, education, and support for mitochondrial disorders. Abliva contributed to the making and funding of UMDF's Voice of the Patient Report 2019, and engages with UMDF's mitoSHARE patient registry initiative. The two organizations collaborate on KL1333 patient registry and interview studies in preparation for the efficacy study.
• International Mito Patients (IMP)coreGTM or Marketing PartnerInternational patient organization that started in the Netherlands and facilitates cross-border cooperation among national mitochondrial patient organizations. IMP started the World Mitochondrial Disease Week (WMDW), which Abliva actively supports each year in the third week of September.
• Cure MitocoreGTM or Marketing PartnerGlobal Leigh syndrome patient organization. Abliva is a proud member of Cure Mito's Corporate Advisory Council (CAC), helping unite the global Leigh syndrome community to accelerate patient-centered research, treatments, and cures — directly relevant to NV354's target indication.
• The Lily FoundationminorGTM or Marketing PartnerUK's leading mitochondrial disease charity, working to improve the lives of people affected by mitochondrial diseases and toward effective treatments and cures. Abliva engages as a partner in the UK mitochondrial disease patient community.
• MitoconminorGTM or Marketing PartnerItalian mitochondrial patient organization working for the best quality of life for mitochondrial disease patients and their families. Abliva engages with Mitocon as part of its patient-organization outreach in Italy (also an active FALCON study country).
• LHON Eye SocietyminorGTM or Marketing PartnerSwedish patient organization for patients and families affected by Leber's Hereditary Optic Neuropathy (LHON). Organizes the 'Mito Race' (5 km run) during World Mitochondrial Disease Week. Relevant to Abliva's LHON-related research interests.
• Frank J. Sasinowski (US regulatory consultant)minorOthersAbliva's US regulatory consultant who has presented on the Orphan Drug Act and what it means to the development of drugs for rare diseases (interview from Abliva's Mitochondria Day, 16 Sep 2020). Sasinowski is a noted US orphan-drug regulatory expert.

Scale indicators9 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Stealth BioTherapeutics: Clinical-stage biotech developing elamipretide and other mitochondria-targeted therapeutics for primary mitochondrial disease and Barth syndrome. Most direct competitor to Abliva given overlapping mitochondrial myopathy indications and similar orphan-drug regulatory strategy.
• Pretzel Therapeutics: Emerging biotech developing therapies for mitochondrial disease, including sirtuin modulators and mitochondrial DNA-related approaches. Directly comparable as a focused mitochondrial-disease drug developer targeting rare genetic indications.
• Khondrion: Dutch clinical-stage company developing sonlicromanol for mitochondrial disease, including MELAS and other primary mitochondrial disorders. Highly comparable given overlapping target indications, similar clinical-stage status, and European base.
• Reneo Pharmaceuticals: Clinical-stage rare-disease company developing therapies for inherited mitochondrial diseases (mitochondrial myopathies) using PPAR delta agonists. Directly comparable pipeline focus on primary mitochondrial myopathies with orphan drug strategy.
• GenSight Biologics: French clinical-stage gene therapy company developing LUMEVOQ for Leber's Hereditary Optic Neuropathy (LHON), one of NV354's named target indications. Directly comparable as a focused developer of treatments for a specific mitochondrial disorder (LHON).
• Modis Therapeutics (acquired by Zogenix/UCB): Was a clinical-stage biotech focused on mocetinostat and other therapies for mitochondrial disease including TK2 deficiency; acquired by Zogenix in 2020. Provides a useful comp for valuation/exit dynamics of late-stage mitochondrial disease biotechs.
• Santhera Pharmaceuticals: Swiss specialty pharma developing treatments for rare mitochondrial and neuromuscular diseases, including vamorolone for Duchenne muscular dystrophy and earlier-stage mitochondrial programs. Comparable as a European rare-disease and mitochondrial-focused commercial-stage player.

Broad incumbents

• Reata Pharmaceuticals: Acquired by Biogen in 2023, previously developed Skyclarys (omaveloxolone) for Friedreich's ataxia, a mitochondrial/neuromuscular rare disease. Provides a useful comp on regulatory pathways and commercialization for mitochondrial rare diseases with no prior approved therapies.
• Horizon Therapeutics: Large rare-disease specialty pharma (acquired by Amgen in 2023) with rare-disease commercial infrastructure and several orphan drugs across metabolic, endocrine, and inflammatory conditions. Comparable as a broad incumbent illustrating scale rare-disease commercial economics relevant to Abliva's eventual launch.
• Recordati Rare Diseases: Specialty division of Recordati focused on rare and ultra-rare diseases, including metabolic disorders and treatments for LHON (Raxone/idebenone — currently the only approved mitochondrial disease therapy besides Abliva's targets). Provides a comp for European ultra-rare-disease commercialization and pricing.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights6 records

Customer concentration

• YouTubeyoutube.com/channel/UChqP7Ky5caXtp72CELhD6Mg
• LinkedInlinkedin.com/company/abliva

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds7 records

Investors6 records

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M&A

Investments1 record

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

Frequently asked questions about Abliva AB