What Saol Therapeutics does

Saol Therapeutics is a privately held, clinical-stage pharmaceutical company founded in 2015 and headquartered in Roswell, Georgia, with additional operating entities in Dublin, Ireland (Saol Therapeutics Research Limited) and Hamilton, Bermuda (Saol International Development LTD). The company focuses on late-stage clinical development of therapeutics for ultra-rare pediatric mitochondrial diseases and central nervous system (CNS) disorders, specifically pain and spasticity. Its pipeline comprises two main drug candidates: SL1009 (Sodium Dichloroacetate, an oral solution for Pyruvate Dehydrogenase Complex Deficiency) and SL1002 (a chemoneurolytic injection built on the proprietary CYCLOPHLEX platform). SL1009 is paired with a patented GSTZ1/MAAI genotype test (licensed from Medosome Biotec) that enables individualized dosing based on patient metabolizer status.

The company's core technology includes the proprietary CYCLOPHLEX chemoneurolytic injection platform for SL1002 and the companion diagnostic GSTZ1/MAAI genotype test for SL1009. Saol has secured multiple FDA regulatory designations for SL1009 — Orphan Drug, Fast Track, Rare Pediatric Disease, Priority Review, and Priority Review Voucher eligibility — positioning it as a potential first FDA-approved treatment for PDCD, an ultra-rare disease with ~90 US births per year and no approved therapeutic alternatives. SL1009 received a Complete Response Letter from FDA in September 2025; a Type A meeting in December 2025 is intended to establish a path forward without requiring an additional clinical trial. SL1002's Phase II RAISE spasticity trial produced statistically significant results (p=0.0060) in October 2024.

Saol's business model is currently pre-revenue. The company previously marketed five mature commercial products (including the Baclofen franchise generating ~$25M annual net revenue from Lioresal), but divested all commercialized assets in January 2022 — selling the Baclofen franchise to Amneal Pharmaceuticals for approximately $83.5 million cash plus low double-digit royalties, and a four-product portfolio to Kamada — to focus exclusively on late-stage clinical development. The divestiture proceeds, combined with over $100 million in cumulative institutional financing under CEO Dave Penake, have funded current R&D operations. Customers (in a clinical-trial context) include principal investigators at University of Florida, UTHealth Houston, University of Utah, and Medical College of Wisconsin, and the company operates an Expanded Access Program for PDCD patients not enrolled in trials. Future commercialization will likely follow a specialty pharmacy/distributor model targeting specialist physicians at academic medical centers.

Name

Saol Therapeutics

Legal name

Saol Therapeutics Inc.

Website

https://saolrx.com

Company type

Private

Founded year

2015

Operating status

Operating

Headcount range

11–50 employees

Short description

Saol Therapeutics is a privately held, clinical-stage pharmaceutical company developing SL1009 (DCA oral solution for ultra-rare PDCD) and SL1002 (a chemoneurolytic injection for spasticity and osteoarthritis knee pain), with FDA designations including Orphan Drug, Fast Track, and Priority Review on its lead program.

Ownership category

akta.pro rank

Product category

Rare Disease Pharmaceutical Development

akta.pro primary industry

Prescription Digital Therapeutics (PDT) (HLACANAB)

Keywords

• Rare disease therapeutics
• Mitochondrial disease treatments
• CNS disorder drug development
• Chemoneurolytic injections
• Orphan drug development

Headquarters

HQ city

Roswell

HQ country

United States

HQ region

North America

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Pipeline Development (Pre-revenue): Saol Therapeutics is a clinical-stage pharmaceutical company that has not yet commercialized any products. The company was previously commercial (having marketed five mature commercial products) but divested all commercialized assets to focus on late-stage clinical development. No revenue is currently generated from product sales.
2. Divested Commercial Assets: Saol divested its Baclofen franchise (Lioresal and LYVISPAH) to Amneal Pharmaceuticals for approximately $83.5 million cash plus low double-digit royalties. Also divested a portfolio of four FDA-approved commercial products to Kamada. These transactions provided capital to fund current R&D activities.

Go-to-market motion1 record

Distribution channels3 records

Marketing channels6 records

Core offering

Saol Therapeutics is a clinical-stage pharmaceutical company developing two late-stage drug candidates: SL1009, an oral sodium dichloroacetate (DCA) solution for the ultra-rare pediatric mitochondrial disease Pyruvate Dehydrogenase Complex Deficiency (PDCD), paired with a proprietary GSTZ1 genotype companion diagnostic for individualized dosing; and SL1002, a proprietary CYCLOPHLEX™ chemoneurolytic injection for limb spasticity and chronic osteoarthritis knee pain. The company has divested all former commercial assets and currently focuses exclusively on clinical development of these two programs.

Product overview

Saol Therapeutics is a privately held, clinical-stage pharmaceutical company focused on late-stage clinical development in CNS disorders (pain and spasticity) and orphan diseases. The company's pipeline consists of two main drug candidates: SL1009 (Sodium Dichloroacetate/DCA), an oral solution for Pyruvate Dehydrogenase Complex Deficiency (PDCD) that is used with a proprietary GSTZ1 genetic test for individualized dosing; and SL1002, a novel chemoneurolytic injection utilizing the proprietary CYCLOPHLEX™ technology platform for treatment of limb spasticity and knee pain associated with osteoarthritis. The company recently divested its commercial products to focus exclusively on development-stage programs.

Differentiator

Problem solved

Functional benefit

Products and services

• SL1009 (Sodium Dichloroacetate Oral Solution) Investigational oral sodium dichloroacetate (DCA) solution being developed for the treatment of Pyruvate Dehydrogenase Complex Deficiency (PDCD), an ultra-rare pediatric mitochondrial disease with no FDA-approved treatments. Used with a proprietary GSTZ1/MAAI genotype companion diagnostic for individualized dosing based on patient metabolizer status. Phase III trials showed statistically significant improvement in motor function (p=0.002), survival benefit (p=0.027), and reduced plasma lactate (p=0.006).
• SL1002 (CYCLOPHLEX Chemoneurolytic Injection) Investigational proprietary chemoneurolytic injection utilizing CYCLOPHLEX™ technology, being developed for the treatment of limb spasticity in adults (post-stroke, TBI, cerebral palsy, MS, spinal cord injury) and for chronic knee pain associated with osteoarthritis. Phase II RAISE trial showed statistically significant reduction in Modified Ashworth Scale at 6 months (-1.18 vs -0.33 vehicle, p=0.0060), favorable safety profile, and durable effect lasting at least 6 months.
• GSTZ1/MAAI Proprietary Genotype Test Proprietary patented companion diagnostic test identifying mutations at GSTZ1 (glutathione S-transferase zeta 1) to categorize individuals as fast or slow metabolizers of dichloroacetate. Licensed from Medosome Biotec, this enables individualized DCA dosing to reduce treatment-related adverse events such as peripheral neuropathy.

Quantifiable outcome

• SL1009 demonstrated statistically significant improvement in motor function in ITT population during open label extension (p=0.002)
• +4 more outcomes

Named customers6 records

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Nine partnerships are on record, tiered core and minor.

• Medosome BioteccoreTechnology or Integration · 15 March 2023Saol signed an agreement with Medosome Biotec to obtain rights to use their patented genotype test (GSTZ1/MAAI) for all potential indications of SL-1009 (DCA), expanding beyond the original PDCD indication. The test enables individualized dosing by categorizing patients as fast or slow drug metabolizers. Saol also receives support for patient recruitment in the Phase IIA DCA in Glioblastoma trial at University of Florida.
• Leading Cancer Center (Pediatric Oncology Research)minorStrategic or Co-development Partner · 15 March 2023Saol signed a sponsored research agreement with a leading cancer center to evaluate DCA in rare pediatric solid tumors including Wilms, Neuroblastoma, Rhabdoid Tumor, Osteosarcoma, Ewing Sarcoma, and Rhabdomyosarcoma. Preclinical research investigates DCA alone and in combination with chemotherapy.
• University of FloridacoreStrategic or Co-development Partner · 4 October 2022Saol collaborates with the University of Florida as a key trial site and research partner for the Phase III PDCD trial and Phase IIA glioblastoma trial. Dr. Peter Stacpoole serves as Principal Investigator for both trials. The partnership has led to over-enrollment of the Phase III PDCD study and is expanding to additional DCA indications.
• InformedDNAcoreStrategic or Co-development Partner · 26 April 2022Saol partnered with InformedDNA, the nation's leading applied genomics solutions company, to offer genetic counseling for families of individuals with PDCD. InformedDNA's board-certified genetic counselors provide discussion of clinical features, progression, and inheritance of PDCD, and review potential participation in the Phase 3 clinical trial. This partnership addresses the challenge of identifying and engaging ultra-rare disease patients.
• GeneDx, Inc.coreStrategic or Co-development Partner · 1 March 2022Saol collaborated with GeneDx, a global leader in genomics and genomic analysis, to assist in identifying patients diagnosed with PDCD who may be eligible for the Phase 3 clinical trial. GeneDx's advanced genetic testing provides diagnostic information on disease-causing genetic changes, helping accelerate patient recruitment among this highly targeted ultra-rare patient population.
• Amneal PharmaceuticalsminorChannel Partner/ Reseller/ Distributor · 5 January 2022Amneal acquired Saol's Baclofen franchise including Lioresal and LYVISPAH for approximately $83.5 million cash plus low double-digit royalties. The transaction included Saol's experienced institutional commercial team. This divestment enabled Saol to focus entirely on its development pipeline.
• Hope for PDCD FoundationcoreStrategic or Co-development PartnerThe Hope for PDCD Foundation delivered a petition to the FDA urging approval of DCA as the first targeted treatment for PDCD. The foundation represents patient families advocating for urgent FDA approval, reflecting strong alignment with Saol's mission for this ultra-rare disease.
• United Mitochondrial Disease Foundation (UMDF)minorStrategic or Co-development PartnerUMDF represents the mitochondrial disease patient community and has expressed deep appreciation for Saol's investment in the PDCD project. UMDF's Science & Alliance Officer Philip E. Yeske, Ph.D. commented on the over-enrolled trial as a testament to patient families' desire to participate in therapeutic development.
• Kamada PharmaceuticalsminorChannel Partner/ Reseller/ DistributorKamada acquired a portfolio of four FDA-approved commercial products from Saol as part of Saol's strategic shift from commercial operations to a focused development company.

Scale indicators7 records

Recent moves8 records

Expansion highlights6 records

Direct peers

• Stealth BioTherapeutics: Clinical-stage biotech developing therapeutics for mitochondrial diseases, directly overlapping with Saol's PDCD program and broader mitochondrial disease pipeline. Highly comparable in therapeutic area focus and development stage.
• Soleno Therapeutics: Clinical-stage rare disease biotech with a similar lean operational footprint and a single late-stage asset (diazoxide choline for Prader-Willi syndrome). Closely comparable in company size, focus on ultra-rare pediatric indications, and regulatory pathway strategy.

Broad incumbents

• BridgeBio Pharma: Clinical and commercial-stage rare disease biotech with a broad pipeline across genetic diseases. Comparable in pursuing accelerated regulatory pathways and targeting ultra-rare indications with novel therapeutic approaches.
• BioMarin Pharmaceutical: Large rare disease biotech with multiple approved products for ultra-rare conditions. Comparable in regulatory strategy (orphan, rare pediatric designations) and commercialization of high-priced specialty therapeutics for small patient populations.
• PTC Therapeutics: Rare disease biotech with approved products and pipeline focused on CNS, metabolic, and oncology indications. Comparable in targeting ultra-rare genetic disorders and leveraging orphan drug designations.
• Ultragenyx Pharmaceutical: Established commercial-stage rare disease biotech with multiple approved and late-stage assets across metabolic, neuromuscular, and CNS indications. Comparable in rare disease focus and development of novel therapeutics for ultra-rare populations.
• Ipsen: Specialty pharma with a strong spasticity franchise (Dysport) competing in the same limb spasticity market where Saol's SL1002 is being developed. Directly comparable as a competitor in Saol's spasticity indication.

Emerging players

• Travere Therapeutics: Mid-stage rare disease company with late-stage assets in kidney and CNS indications. Comparable in leveraging FDA accelerated pathways and targeting conditions with high unmet need.
• Catalyst Pharmaceuticals: Specialty pharma focused on rare neurological and metabolic diseases with approved products. Comparable in commercializing therapies for small patient populations using orphan-drug pricing.
• Reneo Pharmaceuticals: Clinical-stage biotech focused on mitochondrial diseases and rare metabolic disorders. Directly comparable therapeutic area overlap with Saol's PDCD and broader mitochondrial disease programs.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks7 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/saol-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles5 records

Funding overview

Funding rounds2 records

Investors1 record

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

M&A

Investments

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

Frequently asked questions about Saol Therapeutics