SL-1002 is currently being developed for the treatment of chronic knee pain associated with osteoarthritis (COMPASS Trial) and mild to severe limb spasticity (RAISE Trial). Topline data for the COMPASS Osteoarthritis Knee Pain Trial will be available in the first quarter of 2024, with topline data from the RAISE Limb Spasticity Trial available shortly thereafter. 

ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, September 19, 2023 / PRNewswire/ – Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, announced today that enrollment has been completed in the company’s two Phase II trials for SL-1002.

Both the COMPASS Osteoarthritis Knee Pain Trial (NCT05470608) and the RAISE Limb Spasticity Trial (NCT05311215) are each fully enrolled, at 132 patients and 32 patients, respectively.

SL-1002 is a novel, chemoneurolytic injection, that utilizes Saol’s proprietary CYCLOPHLEX^TM technology.

“Running parallel, multi-indication Phase II studies for SL-1002 has only been possible due to our committed investigator partners, their research teams, and the tireless work of the employees at Saol Therapeutics,” said David Penake, CEO of Saol Therapeutics.   “This is a tremendous milestone for our company, and we look forward to sharing the results with the medical community.  While celebrating this accomplishment, our team has already shifted into preparing for what will be necessary to initiate the Phase III programs and expansion to other important indications.”

The COMPASS Osteoarthritis Knee Pain Trial is a multicenter, randomized (3:1), double-blind, placebo-controlled, single ascending-dose escalation study to assess the safety and efficacy of SL-1002 for the treatment of chronic knee pain associated with osteoarthritis in adult patients.  The efficacy of SL-1002 will be assessed in comparison to placebo in its ability to reduce the average pain intensity at 3 months (12 weeks). The safety of SL-1002 will be assessed throughout the study compared to placebo when used for treating chronic knee pain associated with osteoarthritis.  Additional secondary measures include improvements in function, concomitant medication, and healthcare utilization.

“The completion of enrollment in the COMPASS Osteoarthritis Knee Pain Trial brings this therapy one step closer to ultimately helping patients,” said Principal Investigator Zachary McCormick, MD, Vice Chair and Associate Professor of Physical Medicine and Rehabilitation at the University of Utah.  “Recent developments in our understanding of the neuroanatomy of the knee indicate that this treatment could be incredibly beneficial to the millions of patients who deal with chronic pain related to osteoarthritis of the knee.  We have enjoyed collaborating with the Saol Therapeutics team and the 14 additional sites in the COMPASS trial. I look forward to seeing the results soon.”

The RAISE Limb Spasticity Trial is a randomized (3:1), 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. Following randomization, the study period will be up to 26 weeks.  The primary endpoint is the overall safety profile of a single treatment exposure of SL-1002 in comparison to a placebo.

Saol Therapeutics currently expects topline results for the COMPASS Osteoarthritis Knee Pain Trial in the first quarter of 2024, and the RAISE Limb Spasticity Trial in the second quarter of 2024.

About COMPASS Osteoarthritis Knee Pain Trial

The COMPASS Osteoarthritis Knee Pain Trial is a multicenter, randomized, double-blind, placebo-COntrolled, single-ascending dose escalation study to assess the safety and efficacy of SL-1002 injectable for treatMent of PAin aSSociated with OsteoArthritis of the knee.  (NCT05470608).

About RAISE Limb Spasticity Trial

The RAISE Limb Spasticity trial is a Randomized double-blind, placebo-controlled, single AscendIng dose escalation study to assess the Safety, Pharmacokinetics and Efficacy of SL-1002 in adult patients with limb spasticity.  (NCT05311215).

About SL-1002

SL-1002 is a novel, chemoneurolytic injection, that utilizes Saol’s proprietary CYCLOPHLEX^TM technology. It is currently being developed for the treatment of chronic knee pain related to osteoarthritis and limb spasticity, both in the adult population (18+) in the United States.

About Saol Therapeutics

Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland, and Hamilton, Bermuda.  Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases.  Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations.  For more information, visit www.saolrx.com.

Saol Therapeutics Contact

Senior Vice President, Strategy
Brian Nappi
bnappi@saolrx.com

The third cohort of the COMPASS trial confirmed the highest planned dose target for the study is well tolerated. 

ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, April 12, 2023 / – Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, announced today the company’s Phase II COMPASS Trial is moving into the fourth and final cohort, where it will enroll approximately 100 patients. 

SL-1002 is a novel, proprietary chemoneurolytic injection currently under development at Saol Therapeutics and is being evaluated in the COMPASS Osteoarthritis Knee Pain Trial. The trial is a multicenter, randomized, double-blind, placebo-controlled, single ascending-dose escalation study to assess the safety and efficacy of SL-1002 for the treatment of knee pain associated with osteoarthritis in adult patients (NCT05470608). 

“We are pleased that the Safety and Dose Escalation Committee (SDEC) continues to confirm that there have been no dose limiting adverse events in cohorts 1, 2 or 3. Saol will be advancing the COMPASS trial into the 4th and largest cohort to establish efficacy,” said Saol Therapeutics CEO David Penake. 

In addition to the COMPASS Osteoarthritis Knee Pain Trial, Saol Therapeutics is also sponsoring the RAISE Spasticity Trial (NCT05311215), evaluating the safety, pharmacokinetics and efficacy profile of SL-1002 in adult patients with limb spasticity. Enrollment has also advanced through completion of the third patient cohort. 

“SL-1002 continues to be an exciting program for our company and we are encouraged with the progress to date,” added Penake. “We want to thank the patients and clinicians for their partnership in our Phase II program.” 

Saol Therapeutics currently expects topline results of both the COMPASS Osteoarthritis Knee Pain Trial and the RAISE Spasticity Trial to readout by early 2024. 

About COMPASS Osteoarthritis Knee Pain Trial 

The COMPASS Osteoarthritis Knee Pain Trial is a multicenter, randomized, double-blind, placebo-COntrolled, single-ascending dose escalation study to assess the safety and efficacy of SL-1002 injectable for treatMent of PAin aSSociated with OsteoArthritis of the knee. 

Saol began enrolling patients in the COMPASS Osteoarthritis Pain Trial in the 3rd Quarter of 2022. (NCT05470608). 

About RAISE Spasticity Trial 

The RAISE Spasticity trial is a Randomized double-blind, placebo-controlled, single AscendIng dose escalation study to assess the Safety, Pharmacokinetics and Efficacy of SL-1002 in adult patients with limb spasticity. (NCT05311215). 

About SL-1002 

SL-1002 is a novel, proprietary chemoneurolytic injection currently under investigation for the treatment of limb spasticity and pain related to osteoarthritis of the knee in adults. 

About Saol Therapeutics 

Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda. Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases. Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations. For more information, visit www.saolrx.com. 

• By signing an agreement with Medosome Biotec
• By providing support for clinical trial treatment of GBM at the University of Florida
• By starting a collaborative research project in rare pediatric cancers with a leading cancer center

ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, March 15, 2023 / PRNewswire/ –

Saol Therapeutics (“Saol”), a privately held, clinical-stage pharmaceutical company, announced today the signing of an agreement with Medosome Biotec to provide Saol the rights to use their patented genotype test for all potential indications where SL-1009 might be used as a therapy.   The test is performed to identify potential mutations at GSTZ1 (glutathione S-transferase zeta 1 human enzyme) that categorize individuals as fast or slow metabolizers of DCA (SL-1009).   Utilization of the genotype testing enables individual dosing, potentially reducing the incidence of treatment related adverse events.  Prior to this agreement Saol only had rights to the patented genotype test for the indication of Pyruvate Dehydrogenase Complex Deficiency (PDCD).

As part of the agreement Saol will aid patient recruitment efforts for the Phase IIA Trial of DCA in Glioblastoma Multiforme (GBM) by providing additional funding and resource support.  Glioblastoma is aggressive and the most common type of primary brain cancer in adults.  GBM is difficult to treat and there is no known cure.  This study, sponsored by the University of Florida and the Food and Drug Administration (FDA) (NCT05120284), is further supported in partnership with Medosome Biotec and Saol.

Dr. Peter Stacpoole is Principal Investigator for the PDCD trial and the GBM trial and is Professor of Medicine at University of Florida. Dr. Stacpoole stated, “We are pleased to expand our partnership with Saol Therapeutics.  Our partnership has led to over-enrollment of our Phase III study in PDCD (NCT02616484) and we mutually see the broader potential application of DCA as the prototypic inhibitor of Pyruvate Dehydrogenase Kinases (PDKs) that, in turn, inhibit PDC enzymatic activity. We look forward to collaborating with Saol on potential additional indications for DCA.  Expansion of our collaboration on the GBM trial will result in the addition of more study sites, allowing for quicker recruitment of patients.  We are hopeful that DCA will be an important addition to the treatment of GBM.”

Saol also recently signed a sponsored research agreement with a leading cancer center to evaluate DCA in rare pediatric solid tumors.  The preclinical research will investigate DCA alone and in combination with chemotherapy in several different pediatric tumor models, such as Wilms, Neuroblastoma, Rhabdoid Tumor, Osteosarcoma, Ewing Sarcoma, and Rhabdomyosarcoma.

Literature suggests that DCA could be effective in several rare, pediatric, solid tumors.  The rationale for the pediatric tumor screening includes the fact that DCA has been widely studied in animals and humans and has a mechanism of action that might override the Warburg effect seen in cancer cells^1,2,3,4.  Saol is currently studying this medicine in children using a proprietary formulation developed for pediatric use (NCT02616484).  “If the pre-clinical assessment provides encouraging data, the work already in pediatric patients may allow us to quickly transition toward clinical investigation”, commented Dr. Virinder Nohria, Chairman and Chief Medical Officer at Saol.

These agreements, and the additional investments behind them, represent an important next step in the evolution of Saol Therapeutics as an emerging pharmaceutical company.  “Now that we have completed enrollment in the PDCD trial we are rapidly transitioning to other important therapeutic areas, and are evaluating the utility of DCA (SL-1009) in adult and pediatric oncology indications with limited treatment options”, said Saol CEO David Penake. “We are pleased to have executed these agreements to support our pursuit of expanded opportunities beyond PDCD”.

Saol expects to have topline data in the PDCD trial in the 3^rd quarter of 2023. Should the results of the trial support a subsequent FDA approval, it will be the first and only medicine approved for this rare, pediatric indication.

References

1. 1. Aminzadeh S, Vidali S, Sperl W, Kofler B, Feichtinger RG. Energy metabolism in neuroblastoma and Wilms tumor. Transl Pediatr. 2015 Jan;4(1):20-32. doi: 10.3978/j.issn.2224-4336.2015.01.04.
2. 2. Guo JQ, Tang HY, Wang CD, Sang BT, Liu X, Yi FP, Liu GL, Wu XM. Influence of Dichloroacetate on Wilms' Tumor in vitro. Ann Clin Lab Sci. 2022 Jan;52(1):101-108. PMID: 35181623.
3. 3. Kankotia S, Stacpoole PW. Dichloroacetate and cancer: new home for an orphan drug? Biochim Biophys Acta. 2014 Dec;1846(2):617-29. doi: 10.1016/j.bbcan.2014.08.005.
4. 4. Tataranni T, Piccoli C. Dichloroacetate (DCA) and Cancer: An Overview towards Clinical Applications. Oxid Med Cell Longev. 2019 Nov 14; 2019:8201079. doi: 10.1155/2019/8201079. PMID: 31827705; PMCID: PMC6885244.

About Dichloroacetate (DCA; SL-1009) 

DCA, a pan- PDK inhibitor, has the potential to be the 1^st approved medication for the mitochondrial disease PDCD and will be available as an oral solution.  Gene mutations in the mitochondrial Pyruvate Dehydrogenase Complex (PDC) lead to congenital PDCD. However, PDC is also inhibited by PDKs, that may be over-expressed in PDCD. DCA inhibits PDKs to stimulate residual PDC activity and increase energy (ATP) production by mitochondria. DCA dosing is based on a proprietary genetic test that dichotomizes subjects into “fast” and “slow” drug metabolizers, providing individualized dosing.

About Saol Therapeutics

Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda.  Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases.  Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations.  For more information, visit www.saolrx.com.

About Medosome Biotec

Medosome Biotec, LLC is a privately held, preclinical and early-stage clinical pharmaceutical company with operations in Alachua, FL and Bloomington, IN.  The Company focuses on pediatric diseases with an emphasis on developing and offering genetic tests for diagnosing rare diseases and providing personalized dosing of pharmaceutical drugs.  For more information, visit www.mdbiotec.com

Media contact:  Brian Nappi, Senior Vice President Strategy, bnappi@saolrx.com

ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, February 13, 2023 – Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, is proud to announce it has received the 2023 Innovation Award from the Center for Global Health Innovation’s (CGHI) Office of Life Sciences and Digital Health. CGHI - formerly known as Georgia Bio - is the state’s life science trade association.

The Innovation Award is presented to the “department, institution, company or individuals who are forging new ground by thinking outside traditional paradigms to create some unique technology.”

“We are honored to receive the 2023 Innovation Award from the Center for Global Health Innovation,” stated Saol Therapeutics CEO David Penake. “2022 was a transformational year for our company which has set Saol up for a tremendous future. It’s a wonderful honor for each employee at Saol to know that their hard work and progress in bringing new therapies to patients is being recognized.”

“The life science industry in Georgia is booming and a significant amount of that growth is due to the team at CGHI,” added Penake. “They are doing an unbelievable job driving new investment and growth in this space and to be recognized by a group that we align with and admire is incredibly meaningful to all of us at Saol.”

In 2022, Saol Therapeutics transformed from a global commercial organization to a late-stage development company. In the last 18 months, Saol divested all commercialized assets as well as two development programs, including one of which that was recently approved by the FDA. The proceeds from the transactions have fully funded current research activities for development programs SL-1002 and SL-1009. SIL-1002 is in two separate phase II studies in adult patients, one studying its effect for knee pain associated with osteoarthritis and the other for the treatment of spasticity. SIL-1009 is in a Phase III trial for a rare pediatric mitochondrial disease that recently completed enrollment, and a Phase II trial in glioblastoma. The topline results for all these studies are expected later in 2023.

The award will be presented when CGHI celebrates its 25th annual Golden Helix Awards on Wednesday, March 29th at the Fox Theatre in Atlanta, GA.

To view the press release from CGHI, including a list of all the 2023 honorees, please click here.

About Saol Therapeutics

Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda. Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases. Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations. For more information, visit www.saolrx.com.

ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, October 4, 2022 / PRNewswire/ -- Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, announced today that the pivotal Phase 3 clinical trial evaluating Sodium Dichloroacetate (DCA) for the treatment of a rare, pediatric, ultra-orphan mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD) has achieved its targeted enrollment. Saol is one of the trial collaborators, with topline results anticipated early in the second half of 2023.

“There are many challenges to overcome when enrolling patients in clinical trials for rare diseases”, said Saol Therapeutics CEO David Penake.  “The hard work of the individual study sites, patient advocacy groups, genetic testing companies, and genetic counseling partners allowed us to exceed our enrollment target of 30 patients.  Our team will now prepare for a rapid NDA submission should the treatment prove to be successful.”

This investigator-initiated, NIH and FDA funded trial, conducted in close partnership with University of Florida, Saol Therapeutics and Medosome Biotec, represents the culmination of years of work for Dr. Peter Stacpoole, Principal Investigator and Professor of Medicine at University of Florida.

“I began working on a treatment for PDCD in the 1980s after a family in Connecticut contacted me to see if I could help their child, so it is extremely rewarding to finally reach this milestone”, said Stacpoole.

Philip E. Yeske, Ph.D., Science & Alliance Officer at United Mitochondrial Disease Foundation, commented “The mitochondrial disease patient community is deeply appreciative of the time, effort, and capital that Saol and its collaborators are investing into this project. An over-enrolled clinical trial is a testament to the desire of the PDCD patient families to play an active role in therapeutic development.”

Study Design

This trial evaluates daily administration of DCA in children with a confirmed pathological mutation in the Pyruvate Dehydrogenase Complex or in a pyruvate dehydrogenase phosphatase gene. The primary efficacy outcome measure is based on a novel Observer Reported Outcome (ObsRO) survey developed specifically for this trial.

The study design is a 9-month double-blind crossover comparison between DCA and placebo, during which the ObsRO is completed daily by a parent/caregiver, followed by an open label phase of DCA administration.

More information on the clinical trial and participating institutions can be found at Phase 3 PDCD Trial.

About DCA (SL-1009) 

DCA has the potential to be the 1^st approved medication for the mitochondrial disease PDCD and will be available as an oral solution.  Gene mutations in the mitochondrial Pyruvate Dehydrogenase Complex (PDC) lead to congenital PDCD. However, PDC is also inhibited by pyruvate dehydrogenase kinases (PDK), that may be over-expressed in PDCD. DCA inhibits PDKs to stimulate residual PDC activity and increase energy (ATP) production by mitochondria. DCA dosing is based on a proprietary genetic test that dichotomizes subjects into “fast” and “slow” drug metabolizers, providing individualized dosing.  For more information on DCA (SL-1009), visit https://saolrx.com/pipeline/.

About Pyruvate Dehydrogenase Complex Deficiency (PDCD)

PDCD is a mitochondrial disorder of carbohydrate oxidation that mostly affects the nervous system and skeletal muscle and leads to decreased ATP production and energy failure.  It affects only a few hundred individuals in the U.S.  It is a common cause of congenital lactic acidosis, a life-threatening condition that may occur as early as the neonatal period. Patients may also exhibit extreme tiredness (lethargy), poor feeding, and rapid breathing (tachypnea)and other signs of neurological and neuromuscular dysfunction, such as developmental delay, low muscle tone (hypotonia), abnormal eye movements and seizures. Signs and symptoms usually begin soon after birth, but may appear later in childhood.

There are currently no FDA-approved therapies for PDCD.

About Saol Therapeutics

Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda.  Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases.  Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations.  For more information, visit www.saolrx.com.

About Medosome Biotec

Medosome Biotec, LLC is a privately held, preclinical and early-stage clinical pharmaceutical company with operations in Alachua, FL.  The Company focuses on pediatric diseases with an emphasis on developing and offering genetic tests for diagnosing rare diseases and providing personalized dosing of pharmaceutical drugs.  For more information, visit www.mdbiotec.com

Media contact:  Brian Nappi, Senior Vice President Strategy, bnappi@saolrx.com

SL-1002 is also being concurrently studied for the treatment of limb spasticity in the Phase II RAISE Trial – topline data for both studies (pain and spasticity) is expected in 2023

ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, August 9, 2022 / PRNewswire/ -- Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, announced today that the first patient has been enrolled in the company’s Phase II COMPASS Osteoarthritis Pain Trial.

SL-1002 is a novel, proprietary chemoneurolytic injection currently under development at Saol Therapeutics and is being evaluated in the COMPASS Osteoarthritis Pain Trial.  The trial is a multi-center, randomized, double-blind, placebo-controlled, single ascending-dose escalation study to assess the safety and efficacy of SL-1002 for the treatment of knee pain associated with osteoarthritis in adult patients (NCT05470608).  The first patient in the trial was enrolled at the International Spine, Pain and Performance Center in Washington, D.C.

“Our center is proud to enroll the first patient in Saol Therapeutics’ COMPASS Osteoarthritis Pain Trial,” said Investigator Mehul J. Desai, MD, President of the International Spine, Pain & Performance Center in Washington, D.C.  “Pain related to osteoarthritis of the knee is a condition that impacts millions of Americans, and while we have current approved treatments, there is a significant opportunity to improve on how we treat this population.  We’re excited to partner with Saol Therapeutics and the rest of the investigators around the United States in evaluating the potential of SL-1002.”

Efficacy and safety are the two primary endpoints in the study.  The efficacy of SL-1002 will be assessed in comparison to placebo in its ability to reduce the average pain intensity at 3 months (12 weeks). The safety of SL-1002 will be assessed throughout the study in comparison to placebo when used for the treatment of knee pain associated with osteoarthritis.  Additional secondary measures include improvements in function, concomitant medication and healthcare utilization.

“We are very enthusiastic to be partnering with Saol Therapeutics to investigate the use of SL-1002 in the COMPASS Osteoarthritis Pain Trial,” said Principal Investigator Zachary McCormick, MD, Vice Chair and Associate Professor of Physical Medicine and Rehabilitation at the University of Utah.  “There have been significant recent advancements in our understanding of the neuroanatomy of the knee with implications for enhancing the non-operative treatment of chronic knee pain due to osteoarthritis.  While treatments have improved in the past decade, there remains a need for additional safe and more effective options that can be delivered in an efficient and patient-friendly way.”

“The COMPASS Osteoarthritis Pain Trial marks another step in our commitment to develop innovative therapies for patients and the clinicians that treat them,” said Saol Therapeutics CEO David Penake.

In addition to the COMPASS Osteoarthritis Pain Trial, Saol Therapeutics recently announced that the first patient was enrolled in the RAISE Spasticity Trial (NCT05311215), evaluating the safety, pharmacokinetic and efficacy profile of SL-1002 in adult patients with limb spasticity.  Enrollment has advanced through completion of the first patient cohort.

“SL-1002 is a very exciting program.  Along with our announcement today that we have commenced enrollment in the COMPASS Osteoarthritis Pain Trial, in parallel we are rapidly enrolling patients in the RAISE Spasticity Trial,” added Penake.  “Beyond these two indications under investigation, our physician collaborators continue to stress to us that the characteristics of this novel agent may have broad applicability in multiple potential use cases.  With the committed partnership of these thought leading collaborators, and the efforts of the Saol team, we plan to complete these studies and evaluate further indications as we approach our pivotal programs in spasticity and osteoarthritis knee pain.”

Saol Therapeutics currently expects topline results of both the COMPASS Osteoarthritis Pain Trial and the RAISE Spasticity Trial to readout in 2023.

About COMPASS Osteoarthritis Pain Trial

The COMPASS Osteoarthritis Pain Trial is a multicenter, randomized, double-blind, placebo-COntrolled, single-ascending dose escalation study to assess the safety and efficacy of SL-1002 injectable for treatMent of PAin aSSociated with OsteoArthritis of the knee.

Saol began enrolling patients in the COMPASS Osteoarthritis Pain Trial in the 3^rd Quarter of 2022.  (NCT05470608).

About RAISE Spasticity Trial

The RAISE Spasticity trial is a Randomized double-blind, placebo-controlled, single AscendIng dose escalation study to assess the Safety, Pharmacokinetics and Efficacy of SL-1002 in adult patients with limb spasticity.  (NCT05311215).

The primary endpoint is the overall safety profile of a single treatment exposure of SL-1002 in comparison to placebo.  Secondary endpoints include, but are not limited to, measures such as the Modified Ashworth Score (MAS), Clinical Global Impression of Change (CGI-C) and the Tardieu scale.  Additional secondary measures include the characterization of the human pharmacokinetics and pharmacodynamics relating to metabolism and clearance of SL-1002 and its metabolites.

About SL-1002

SL-1002 is a novel, proprietary chemoneurolytic injection currently under investigation for the treatment of limb spasticity and in pain related to osteoarthritis of the knee in adults (18+) in the United States.

About Saol Therapeutics

Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda.  Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases.  Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations.  For more information, visit www.saolrx.com.

Program Supports Access to A Pivotal Phase 3 Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency (DCA/PDCD trial; NCT02616484)

ROSWELL, Ga., March 1, 2022 /PRNewswire/ -- Saol Therapeutics, a company researching new treatments for rare diseases, is pleased to announce a collaboration with GeneDx, Inc. a leader in genomic analysis, a wholly owned subsidiary of BioReference Laboratories, Inc., an OPKO Health company (NASDAQ:OPK), to assist in identifying patients diagnosed with a rare mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD) who may be eligible to participate in a Phase 3 clinical trial. PDCD affects less than 300 children in the United States annually and lacks any FDA-approved treatment.

This pivotal phase 3 trial administers the investigational drug dichloroacetate (DCA) to young children who have a deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency is the most common cause of congenital lactic acidosis and is frequently a fatal metabolic disease of childhood. DCA has Orphan Product designation from the FDA for congenital lactic acidosis (CLA), including patients with PDCD.

GeneDx's advanced genetic testing provides diagnostic information on disease-causing genetic changes thanks to expert gene variant interpretation built on the combination of an unparalleled dataset and deep clinical knowledge. Through the program, GeneDx, in collaboration with Saol, will help make clinicians who treat PDCD aware of this pivotal trial in an effort to possibly accelerate patient recruitment among this highly targeted patient population.

Dr. Peter Stacpoole, Principal Investigator of the DCA/PDCD trial and Prof. of Medicine at the University of Florida, notes, "There are currently no FDA-approved treatments for patients with PDCD. Despite this, finding and recruiting children appropriate for participation in clinical trials is not easy. With the help of GeneDx, we hope to complete trial recruitment this year."

Dave Penake, CEO of Saol Therapeutics, is excited about the collaboration with GeneDx. "Individualized genetic screening offers physicians and families the insights needed to avoid years of misdiagnosis. With their help, we are better able to identify mitochondrial diseases early. Without this technology, recruitment for a disease like PDCD could take many years to complete."

About Saol Therapeutics
Saol Therapeutics (pronounced "Sail") is a privately held, biopharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda. Saol is focused on clinical development activity in rare diseases, with a focus on mitochondrial disorders, as well as central nervous system disorders such as spasticity and pain management. Saol is one of the collaborators on a Phase 3 trial studying the first potential treatment for pyruvate dehydrogenase complex deficiency (PDCD). More information on the clinical trial can be found at Phase 3 PDCD Trial. More information about Saol can be found at https://saolrx.com/.

About GeneDx
GeneDx, Inc. is a global leader in genomics, providing testing to patients and their families from more than 55 countries. Originally founded by scientists from the National Institutes of Health, GeneDx offers a world-renowned clinical genomics program with particular expertise in rare and ultra-rare genetic disorders. In addition to its market-leading exome sequencing service, GeneDx offers a suite of additional genetic testing services, including diagnostic testing for hereditary cancers, cardiac, mitochondrial, neurological disorders, prenatal diagnostics, and targeted variant testing. GeneDx is a subsidiary of BioReference Laboratories, Inc., a wholly owned subsidiary of OPKO Health, Inc. To learn more, please visit https://www.genedx.com/.

SOURCE Saol Therapeutics

BRIDGEWATER, N.J. & ROSWELL, Ga., January 5, 2022 --(BUSINESS WIRE)-- Amneal Pharmaceuticals, Inc. (NYSE: AMRX) (“Amneal”) and Saol Therapeutics, a private specialty pharmaceutical company (“Saol”), today announced a definitive agreement under which Amneal will acquire Saol’s Baclofen franchise, including Lioresal^® and LYVISPAH^TM as well as a pipeline product under development. The acquisition expands Amneal’s commercial institutional and specialty portfolio in neurology while adding commercial infrastructure in advance of its entry into the biosimilar institutional market. The transaction is expected to be accretive to Amneal’s adjusted EBITDA and adjusted earnings per share results for 2022.

Lioresal^® is an intrathecal baclofen product delivered through an implantable intrathecal pump for use in the management of severe spasticity of cerebral or spinal origin for the institutional market. It has approximately $25 million in annual net revenue. LYVISPAH^TM is a baclofen oral granules (5, 10 and 20 mg) specialty product recently approved by the U.S. Food and Drug Administration (FDA) for the treatment of spasticity. The product is expected to launch in 2022 leveraging Amneal’s neurology commercial team. Together, Amneal expects these two products to generate between $40 and $50 million in combined annual net revenues by 2025.

As part of the transaction, Amneal is adding Saol’s experienced institutional commercial team for Lioresal^® that can be utilized to support future product launches, including three oncology biosimilar products, filgrastim (biosimilar for Neupogen^®), pegfilgrastim (biosimilar for Neulasta^®) and bevacizumab (biosimilar for Avastin^®). Amneal expects to launch all three biosimilars in 2022, subject to approval by FDA.

“This acquisition is highly aligned with Amneal’s long-term growth strategy adding to our specialty and biosimilars businesses. In specialty, we see LYVISPAH^TM fitting well with our neurology portfolio and pipeline. In addition, Lioresal^® is a durable product with a long-established presence in the institutional market that we look to leverage as we prepare to commercialize our biosimilars in 2022 and beyond,” said Chirag and Chintu Patel, Co-Chief Executive Officers.

“For over 5 years, the Saol team has worked to reinvigorate the Lioresal^® brand and develop new treatment options, like LYVISPAH^TM, for patients struggling with spasticity. We are excited to see these products find their new home at Amneal along with many of our team members that have been critical to our success,” said David Penake, CEO of Saol Therapeutics.

Baclofen is a skeletal muscle relaxant used to treat muscle spasms caused by spinal cord injury, multiple sclerosis, and other conditions. It was first approved by the FDA in 1977. Important Safety Information includes a boxed warning on abrupt discontinuation, which can result in sequalae and in rare cases, has advanced to multiple organ-system failure and death. Reported adverse drug reaction includes convulsion, hypotension, hypotonia, somnolence, dizziness, nausea and headache. Animal data indicates it may cause fetal harm.

See Package Insert (PI) for full prescribing information including boxed warning and complete safety information:

Lioresal^®: https://lioresal.com/wp-content/uploads/2019/03/Lioresal-PI-01-2019.pdf

LYVISPAH^TM: https://lyvispah.com/content/uploads/2021/11/LYVISPAH-USPI-NOVEMBER-2021-FDA-approved.pdf

Terms of the Transaction

Under the terms of the transaction, Amneal will pay approximately $83.5 million of cash at close, and certain royalties (low double-digits) based on annual net sales for certain acquired products. The transaction will be financed with cash on hand and is expected to close in the first quarter of 2022, subject to the satisfaction of customary closing conditions, including clearance under the Hart-Scott Rodino Antitrust Improvements Act.

Advisors

Morgan Lewis & Bockius LLP served as legal counsel to Amneal. SVB Leerink served as exclusive financial advisor and Mayer Brown LLP served as legal counsel to Saol Therapeutics.

About Amneal Pharmaceuticals, Inc.

Amneal Pharmaceuticals, Inc. (NYSE: AMRX), headquartered in Bridgewater, NJ, is a fully-integrated essential medicines company. We make healthy possible through the development, manufacturing, and distribution of generic and specialty pharmaceuticals, primarily within the United States. The Company has a diverse portfolio of approximately 250 products in its Generics segment and is expanding across a broad range of complex products and therapeutic areas, including injectables and biosimilars. In its Specialty segment, Amneal has a growing portfolio of branded pharmaceutical products focused primarily on central nervous system and endocrine disorders, with a pipeline focused on unmet needs. Through its AvKARE segment, the Company is a distributor of pharmaceuticals and other products for the U.S. federal government, retail, and institutional markets. For more, please visit www.amneal.com.

About Saol Therapeutics

Saol Therapeutics (pronounced "Sail") is a privately held, biopharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda. Saol is focused on commercial and clinical development activity in central nervous system disorders such as spasticity, pain management, and orphan diseases. Saol has a robust pipeline of novel, mid-to-late stage development programs in osteoarthritis, focal spasticity and pyruvate dehydrogenase complex deficiency (PDCD). For more information, visit www.saolrx.com.

Cautionary Statement on Forward-Looking Statements

Certain statements contained herein, regarding matters that are not historical facts, may be forward-looking statements (as defined in the U.S. Private Securities Litigation Reform Act of 1995). Such forward-looking statements include statements regarding management’s intentions, plans, beliefs, expectations or forecasts for the future, including among other things: discussions of future operations; expected operating results and financial performance; impact of planned acquisitions and dispositions; whether and when the required regulatory approvals will be obtained; whether and when the other closing conditions will be satisfied and whether and when the transaction will close; whether and when the Company will be able to realize the expected financial results and accretive effect of the transaction; how customers, competitors, suppliers and employees will react to the acquisition; the Company’s strategy for growth; product development; regulatory approvals; market position and expenditures. Words such as “plans,” “expects,” “will,” “anticipates,” “estimates” and similar words are intended to identify estimates and forward-looking statements.

The reader is cautioned not to rely on these forward-looking statements. These forward-looking statements are based on current expectations of future events. If the underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of the Company.

Such risks and uncertainties include, but are not limited to: the impact of the COVID-19 pandemic; the impact of global economic conditions; our ability to successfully develop, license, acquire and commercialize new products on a timely basis; our ability to obtain exclusive marketing rights for our products; the competition we face in the pharmaceutical industry from brand and generic drug product companies, and the impact of that competition on our ability to set prices; our ability to manage our growth through acquisitions and otherwise; our dependence on the sales of a limited number of products for a substantial portion of our total revenues; the risk of product liability and other claims against us by consumers and other third parties; risks related to changes in the regulatory environment, including U.S. federal and state laws related to healthcare fraud abuse and health information privacy and security and changes in such laws; changes to FDA product approval requirements; risks related to federal regulation of arrangements between manufacturers of branded and generic products; the impact of healthcare reform and changes in coverage and reimbursement levels by governmental authorities and other third-party payers; the continuing trend of consolidation of certain customer groups; our reliance on certain licenses to proprietary technologies from time to time; our dependence on third-party suppliers and distributors for raw materials for our products and certain finished goods; our dependence on third-party agreements for a portion of our product offerings; our ability to identify, make and integrate acquisitions of or investments in complementary businesses and products on advantageous terms; legal, regulatory and legislative efforts by our brand competitors to deter competition from our generic alternatives; the significant amount of resources we expend on research and development; our substantial amount of indebtedness and our ability to generate sufficient cash to service our indebtedness in the future, and the impact of interest rate fluctuations on such indebtedness; the impact of severe weather; and the high concentration of ownership of our Class A Common Stock and the fact that we are controlled by the Amneal Group. The forward-looking statements contained herein are also subject generally to other risks and uncertainties that are described from time to time in the Company’s filings with the Securities and Exchange Commission, including under Item 1A, “Risk Factors” in the Company’s most recent Annual Report on Form 10-K and in its subsequent reports on Forms 10-Q and 8-K. Investors are cautioned not to place undue reliance on any such forward-looking statements, which speak only as of the date they are made. Forward-looking statements included herein speak only as of the date hereof and we undertake no obligation to revise or update such statements to reflect the occurrence of events or circumstances after the date hereof.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220104005935/en/

Anthony DiMeo
Senior Director, Investor Relations
anthony.dimeo@amneal.com

Source: Amneal Pharmaceuticals, Inc.