Novel clinical programs,
driven by a focused vision.

Practical Innovation

Saol focuses on developing solutions that directly address current unmet needs, building upon established knowledge.

Changing the Standard of Care

Incorporating new clinical and scientific innovations, our programs aim to deliver an enhanced product profile.

Novel, Proprietary, Neurolytic Agent

  • Unique, patent-protected technology
  • Two Phase 2 trials underway in pain and spasticity

First-of-Kind Potential Treatment for a Rare Mitochondrial Disorder

  • Potential to be first approved FDA treatment for extremely rare disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD)
  • Individualized genetically driven dosing

Saol Therapeutics Pipeline

Targeted Area of Research

Pain Related to Osteoarthritis (OA) of the Knee

Estimated US Incidence

27M Patients 28


Chemoneurolytic Injection

Clinical Trial


Principal Investigator
Zachary L. McCormick, MD_Saol_Principal_Investigator
Zachary L. McCormick, MD - Salt Lake City, UT
  • Vice Chair and Associate Professor
  • Chief of Spine and Musculoskeletal Medicine
  • University of Utah

Program Highlights

  • Phase II Enrollment Complete
  • Proprietary, optimized formulation
  • Well-characterized molecule
  • Potential blockbuster, multi-indication franchise


Phase II Clinical Trial Summary

This is a multi-center, randomized, double-blind, placebo-controlled single-ascending dose escalation study intended to assess the safety, and efficacy of SL-1002 for patients with knee pain associated with osteoarthritis. The study enrolled 4 cohorts for a total of 132 patients. Patients were randomized to receive either SL-1002 or placebo in a 3:1 ratio. The study period will be up to 26 weeks, with an additional screening period of up to 2 weeks.


Disease Background

Osteoarthritis of the knee results from an imbalance between breakdown and repair of the tissues in the synovial joint organ and occurs as a result of multiple risk factors including trauma, overuse, and genetic predisposition.1  This condition is characterized by pain, stiffness, and loss of function, which negatively impact on health-related quality of life.2,3  It is one of the leading causes of disability4, with a lifetime risk of painful knee OA approaching 45%5 and expected to rise significantly in the coming decades as the population continues to age.6

Today, symptomatic knee OA is estimated to affect approximately 27 million people in the US alone. It carries a significant economic burden, with estimates of related annual healthcare costs approaching $15 billion dollars.7, 8, 27

Nonsurgical management of knee osteoarthritis includes weight loss, activity modification, and physical therapy. Other treatments include nonsteroidal anti-inflammatory drugs (NSAIDs), oral opioids, and intra-articular corticosteroid injections.  However, each of these comes with potential risks and limitations.9,10

If conservative and injection-based therapies fail to provide relief, this condition is frequently treated by total knee arthroplasty (TKA).11  However, many patients – with some estimates as high as 25% of patients suffering from pain related to knee osteoarthritis - are poor candidates for TKA due to a variety of health and non-health related reasons, and they may elect to continue with nonsurgical management.12,13

For patients who do elect to undergo a TKA, chronic post-surgical pain has been reported in 8-34% of patients,14 with an estimated 3.8% eventually requiring revision surgery.15

These statistics, combined with the ongoing opioid crisis, have recently led to a call for the “development of non-opioid-focused multimodal pain management formulations” by global thought leaders16, signaling a significant need for additional non-surgical, therapeutic alternatives.

Targeted Area of Research


Estimated US Prevalence

4M Patients


Chemoneurolytic Injection

Clinical Trial
Principal Investigators
Gerard Francisco, MD - Houston, TX
  • The Wulfe Family Chair of Physical Medicine and Rehabilitation, McGovern Medical School - UTHealth
  • Chairman and Professor (with tenure), Distinguished Teaching Professor, UT Systems
  • Chief Medical Officer and Clinical Scientist, TIRR Memorial Hermann
  • Director, UTHealth NeuroRecovery Research Center at TIRR Memorial Hermann
Sheng Li, MD_Saol_Principal_Investigator
Sheng Li, MD - Houston, TX
  • Professor, Department of Physical Medicine and Rehabilitation (PM&R) at McGovern Medical School at UTHealth Attending Physician, TIRR Memorial Herman
  • Director, Stroke Rehabilitation and Recovery Research, TIRR Memorial Hermann Hospital
  • Director, Neurorehabilitation Research Laboratory, UTHealth NeuroRecovery Research Center at TIRR Memorial Hermann
John McGuire, MD_Saol_Principal_Investigator
John R. McGuire, MD – Madison, WI
  • Professor, Department of Physical Medicine and Rehabilitation - Medical College of Wisconsin
  • Director, Stroke Rehabilitation & Spasticity Management – Medical College of Wisconsin and Froedtert Hospital

Program Highlights

  • Phase II Enrollment Complete
  • Proprietary, optimized formulation
  • Well-characterized molecule
  • Potential blockbuster, multi-indication franchise


Phase II Clinical Trial Summary

A multi-center, randomized, double-blind, placebo-controlled single ascending dose escalation study intended to assess the safety, pharmacokinetics and efficacy of a single treatment of SL-1002 in patients with mild to severe limb spasticity. The study enrolled 4 cohorts of 8 patients per cohort for a total of up to 32 patients. Patients were randomized to receive either SL-1002 or placebo in a 3:1 ratio. The study period will be up to 26 weeks, with an additional screening period of up to 2 weeks.


Disease Background

Spasticity is a motor disorder characterized by velocity-dependent increase in tonic stretch reflexes resulting from an upper motor neuron lesion, presenting as intermittent or sustained involuntary activation of muscles. Clinically, this results in increased muscle tone, exaggerated tendon reflexes, clonus and re-emergence of primitive reflexes.17

Disorders of the central nervous system, such as strokes, traumatic brain injuries, cerebral palsy, multiple sclerosis and spinal cord injuries result in neural reorganization causing abnormal neural and muscle control. Spasticity develops because of an imbalance between excitatory and inhibitory input to motor neurons resulting in disinhibition of the stretch reflex and increased muscle excitability. The pattern of spasticity depends on the location of the injury in the CNS. Alterations in neural pathways lead to changes in mechanical properties of muscles and joints that account for some features of spasticity.18

It is estimated that spasticity effects over 12 million people worldwide19 including over 4 million in the United States alone.20 The epidemiology of spasticity is specific to the type and severity of CNS injury. For example, it is estimated that spasticity affects 65-78% of chronic spinal cord injury (SCI) patients21, 25% of stroke patients22, 80% of patients with MS at some point during their clinical course23 and more than 80% in patients with Cerebral Palsy.24

Targeted Area of Research

Pyruvate Dehydrogenase Complex Deficiency (PDCD)

Estimated US Incidence

Approximately 90 Births per Year 29


Oral Solution

Clinical Trial
Principal Investigator
Peter W. Stacpoole, PhD, MD__Saol_Principal_Investigator
Peter W. Stacpoole, PhD, MD - Gainesville, FL
  • Professor of Medicine, Biochemistry and Molecular Biology
  • College of Medicine
  • University of Florida

Program Highlights

  • Phase III Enrollment Complete
  • First potential approved treatment for PDCD
  • Individualized genetically driven dosing
  • Supported by grants from the NIH and FDA
  • Rare Pediatric Disease Designation, US Orphan Drug Designation


Phase III Clinical Trial Summary

This Phase III randomized, placebo-controlled, double-blind trial of 34 children, aged 6 months through 17 years, with confirmed diagnosis of PDC Deficiency.  A novel Observer reported outcome (ObsRO) validated survey was completed by study participant's parent/caregiver is the primary efficacy outcome measure.


Expanded Access for SL-1009

Saol does not currently offer an expanded access program for the ongoing phase III study: “Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency”: (DCA/PDCD), NCT02616484. We recognize the importance of expanded access and are actively exploring the possibility of implementing such a program in the near future. Our primary focus is on completing the ongoing clinical trials and advancing the regulatory submissions required to obtain regulatory approval.

While we work to achieve these milestones, we will also be evaluating the feasibility of establishing an expanded access program to accommodate individuals who were unable to participate in the clinical trial. We understand the urgency and the critical needs of individuals, we appreciate your patience and encourage you to stay updated on our progress through our official channels and announcements. Together, we aim to advance patient care and improve outcomes for those with Pyruvate Dehydrogenase Complex Deficiency.

If you would like to receive a notification should a program become available in the future, please provide your contact information with the subject line EAP Inquiry here.


Disease Background25-27

Pyruvate dehydrogenase complex deficiency (PDCD) is a mitochondrial disorder of carbohydrate oxidation that mostly affects the brain and leads to decreased ATP production and energy deficit. Symptoms of PDC deficiency include signs of metabolic dysfunction such as extreme tiredness (lethargy), poor feeding, and rapid breathing (tachypnea). Other symptoms may include signs of neurological dysfunction such as developmental delay, periods of uncontrolled movements (ataxia), low muscle tone (hypotonia), abnormal eye movements, and seizures. Symptoms usually begin in infancy, but signs can first appear at birth or later in childhood. Symptoms may be especially apparent during times of illness, stress, or after meals with high amounts of carbohydrates. Several hundred children with PDCD have been reported, but the overall frequency is unknown. More males than females are affected by X-linked PDCD. Female carriers of X-linked PDCD may be less severely affected and more difficult to diagnose.

PDCD is caused by abnormalities in the genes that encode the components of the pyruvate dehydrogenase complex. The pyruvate dehydrogenase complex contains three enzymes, E1, E2, and E3, and multiple coenzymes. The E1 enzyme is comprised of an alpha and a beta subunit. PDCD is most commonly caused by abnormalities in the gene that encodes the E1 alpha subunit, E1-alpha subunit pyruvate dehydrogenase gene or PDHA1. The most common form of PDC deficiency is caused by genetic changes (genetic changes or pathogenic variants) in the PDHA1 gene. These pathogenic variants are inherited in an X-linked manner. Pathogenic variants in other genes including PDHB, DLAT, PDHX, DLD, and PDP1 can also cause PDC deficiency. These pathogenic variants are inherited in an autosomal recessive manner.

There are no FDA-approved therapies available for PDCD. Treatment recommendations are based on open label case reports and small trials. Ketogenic diet is the gold-standard therapy for individuals with PDCD because they do not metabolize carbohydrates effectively. Some affected individuals respond to treatment with thiamine, a cofactor for the E1 subunit of the pyruvate dehydrogenase complex.