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 2 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 2 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 2 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 2 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 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).  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.

Saol Therapeutics announces the approval of LYVISPAH^TM (baclofen) oral granules and the strategic transaction to divest the plasma-derived hyperimmune portfolio to Kamada Ltd. for a total value of up to $160M 

ROSWELL, GA US/Dublin, IE/Hamilton, BM Dec. 6, 2021 – Saol Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has approved Saol’s LYVISPAH™ (baclofen) oral granules.

LYVISPAH^TM (lye-vis’-pah) is indicated for the treatment of spasticity resulting from multiple sclerosis, particularly for the relief of flexor spasms and concomitant pain, clonus, and muscular rigidity. LYVISPAH^TM may also be of some value in patients with spinal cord injuries and other spinal cord diseases. LYVISPAH^TM is a strawberry-flavored, dissolvable granular formulation of baclofen and will be available for patients 12 years and above in 5mg, 10mg, and 20mg packets. Unlike other formulations of baclofen, it is approved for administration with or without water, with soft foods and with enteral feeding tubes.

Patients suffering from spasticity may concurrently develop swallowing difficulties. Nearly one million people in the United States are living with multiple sclerosis1 , and the prevalence of spasticity within this patient population has been estimated to be as high as 67%2 . Additionally, the prevalence of dysphagia in the multiple sclerosis population has been reported to be between 34-43%3,4, with aspiration pneumonia frequently cited as a contributing factor in deaths of these patients5 .

Dr Michael Saulino, Chair of Physical Medicine and Rehabilitation at Cooper University Hospital commented, "LYVISPAH^TM represents an important treatment option for individuals with spasticity who have dysphagia. The bioequivalence between LYVISPAH^TM and traditional oral baclofen products should allow for straightforward prescribing by clinicians who manage patients with both clinical problems.”

David Penake, CEO of Saol Therapeutics stated, “We are tremendously excited by the approval of LYVISPAH^TM. Spasticity is a challenging condition to treat, and we have commonly heard that no two patients are alike. Because of this, clinicians stressed to us that there is a need for new formulations designed to benefit their patients who have difficulty swallowing. I’m incredibly proud of the work our team has done to get this approved, and our hope is that this is the first in the line of many new therapies we can bring to market to support health care providers and the patients they treat.”

Following this approval, Saol Therapeutics is preparing for a full commercial launch of LYVISPAH^TM in 2022.

Additionally, Kamada LTD announced on November 22nd , 2021, the acquisition of our four plasmaderived hyperimmune products for:

• CYTOGAM® (Cytomegalovirus Immune Globulin Intravenous [Human]) (CMV-IGIV) is indicated for the prophylaxis of cytomegalovirus disease associated with the transplantation of the kidney, lung, liver, pancreas, and heart.
• WINRHO® SDF is a Rho(D) Immune Globulin Intravenous (Human) is indicated for use in clinical situations requiring an increase in platelet count to prevent excessive hemorrhage in the treatment of non-splenectomies, for Rho(D)-positive children with chronic or acute immune thrombocytopenia (ITP), adults with chronic ITP, and children and adults with ITP secondary to HIV infection. WinRho SDF is also used for suppression of Rhesus (Rh) Isoimmunization during pregnancy and other obstetric conditions in non-sensitized, Rho(D)-negative women.
• HEPAGAM B® is a hepatitis B Immune Globulin (Human) (HBIg) product indicated to both prevent hepatitis B virus (HBV) recurrence following liver transplantation in hepatitis B surface antigen positive (HBsAg- positive) patients and provide post-exposure prophylaxis.
• VARIZIG® [Varicella Zoster Immune Globulin (Human)] is indicated for post-exposure prophylaxis of varicella (chickenpox) in high-risk patient groups, including immunocompromised children, newborns, and pregnant women. VARIZIG is intended to reduce the severity of chickenpox infections in these patients.

Under the terms of the agreement, Kamada will pay Saol up to $160 million, with a $95 million upfront payment, and up to an additional $50 million in sales milestones during 2022-2034. In addition, Kamada will acquire from Saol existing inventory at an estimated value of approximately $15 million, which will be paid over 10 equal quarterly installments.

“The proceeds from the sale of the hyperimmune products will be invested to expand our commercial infrastructure to launch LYVISPAH^TM and further development of our pipeline assets SIL-1002 for Spasticity, SIL-1009 for Pyruvate Dehydrogenase Complex Disorder and SIL-1010 for Chronic Pain associated with Osteoarthritis. The approval of LYVISPAH^TM on the same day as the announcement of the transaction highlights our company’s ability to execute on multiple priorities. Moving forward, we have a clear path to growth and continued profitability” concluded Mr. Penake.

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 CNS disorders such as spasticity, 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 LYVISPAHTM (baclofen) oral granules

LYVISPAH^TM is indicated for the treatment of spasticity resulting from multiple sclerosis, particularly for the relief of flexor spasms and concomitant pain, clonus, and muscular rigidity. LYVISPAH^TM may also be of some value in patient with spinal cord injuries and other spinal cord diseases. LYVISPAH^TM is a strawberry-flavored, dissolvable granular formulation of baclofen and will be available for patients 12 years and above in 5mg, 10mg, and 20mg packets.

1. Wallin MT, Culpepper WJ, Campbell JD, Nelson LM, Langer-Gould A, Marrie RA, Cutter GR, Kaye WE, Wagner L, Tremlett H, Buka SL, Dilokthornsakul P, Topol B, Chen LH, LaRocca NG; US Multiple Sclerosis Prevalence Workgroup. The prevalence of MS in the United States: A population-based estimate using health claims data. Neurology. 2019 Mar 5;92(10):e1029-e1040. doi: 10.1212/WNL.0000000000007035. Epub 2019 Feb 15. Erratum in: Neurology. 2019 Oct 8;93(15):688. PMID: 30770430; PMCID: PMC6442006.
2. McGuire JR. epidemiology of Spasticity in the Adult and Child. In: Brashear A, Elovic, eds. Spasticity. 2 ed. New York: Demos Medical; 2016: 5-15.
3. Calcagno P, Ruoppolo G, Grasso MG, De Vincentiis M, Paolucci S. Dysphagia in multiple sclerosis - prevalence and prognostic factors. Acta Neurol Scand. 2002 Jan;105(1):40-3. doi: 10.1034/j.1600- 0404.2002.10062.x. PMID: 11903107.
4. Aghaz A, Alidad A, Hemmati E, Jadidi H, Ghelichi L. Prevalence of dysphagia in multiple sclerosis and its related factors: Systematic review and meta-analysis. Iran J Neurol. 2018 Oct 7;17(4):180-188. PMID: 31210903; PMCID: PMC6555886.
5. Harding K, Zhu F, Alotaibi M, Duggan T, Tremlett H, Kingwell E. Multiple cause of death analysis in multiple sclerosis: A population-based study. Neurology. 2020 Feb 25;94(8):e820-e829. doi: 10.1212/WNL.0000000000008907. Epub 2020 Jan 13. PMID: 31932517; PMCID: PMC7136054.

IMPORTANT SAFETY INFORMATION

LYVISPAH^TM (baclofen) oral granules

Indications and Usage

• LYVISPAH™ (baclofen) oral granules is a muscle relaxant and antispastic that is indicated for the
  treatment of spasticity resulting from multiple sclerosis, particularly for the relief of flexor
  spasms and concomitant pain, clonus, and muscular rigidity.
• LYVISPAH™ may also be of some value in patients with spinal cord injuries and other spinal cord
  diseases.

Limitations of Use

• LYVISPAH™ is not indicated in the treatment of skeletal muscle spasm resulting from rheumatic
  disorders.

Contraindications

• LYVISPAH™ is contraindicated in patients with hypersensitivity to baclofen.

Select Warnings and Precautions

• Abrupt discontinuation of baclofen has resulted in serious adverse reactions including death;
  therefore, reduce the dosage slowly when LYVISPAH™ is discontinued.
• Neonatal withdrawal can occur; gradually reduce the dosage and discontinue LYVISPAH™ before
  delivery.
• LYVISPAH™ can cause drowsiness and sedation. Patients should avoid the operation of
  automobiles or other dangerous machinery until they know how the drug affects them. Advise
  patients that the central nervous system effects of LYVISPAH™ may be additive to those of
  alcohol and other CNS depressants.
• LYVISPAH™ can cause exacerbation of the following: psychotic disorders, schizophrenia, or
  confusional states; autonomic dysreflexia; epilepsy. Use with caution in patients with these
  conditions.
• LYVISPAH™ should be used with caution in patients who have had a stroke.

Adverse Reactions

Serious Adverse Reactions

• Advise patients and caregivers not to discontinue use of LYVISPAH™ without consulting with their healthcare provider because sudden withdrawal of LYVISPAH™ can result in serious complications that include hallucinations, seizures, high fever, confusion, muscle stiffness, multiple organ-system failure, and death. Inform patients that early symptoms of LYVISPAH™ withdrawal may include increased spasticity, itching, and tingling of extremities. Abrupt discontinuation of baclofen, regardless of the cause, has resulted in adverse reactions that include hallucinations, seizures, high fever, altered mental status, exaggerated rebound spasticity, and muscle rigidity, that in rare cases has advanced to rhabdomyolysis, multiple organ-system failure, and death. Therefore, reduce the dosage slowly when LYVISPAH™ is discontinued, unless the clinical situation justifies a rapid withdrawal.

Common Adverse Reactions

• The most common adverse reactions (>1%) in patients treated with baclofen for spasticity are drowsiness, dizziness, weakness, nausea, confusion, hypotension, headache, insomnia, constipation, urinary frequency, and fatigue.

Drug Interactions

• LYVISPAH™ (baclofen) oral granules can cause CNS depression when used concomitantly with other CNS depressants and alcohol.

Use in Specific Populations

• There are no adequate data on the developmental risks associated with the use of LYVISPAH™ in pregnant women. LYVISPAH™ should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus.
• Nursing mothers should exercise caution, as oral baclofen has been shown to pass into milk at therapeutic doses.
• Withdrawal symptoms can occur in breastfed infants when maternal administration of LYVISPAH™ is stopped, or when breastfeeding is stopped.
• Safety and effectiveness in pediatric patients below the age of 12 have not been established. • In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant disease of other drug therapy.
• Because baclofen is primarily excreted unchanged through the kidneys, LYVISPAH™ should be given with caution to patients with renal impairment, and it may be necessary to reduce the dosage.

For more information, refer to LYVISPAH™ (baclofen) oral granules prescribing information, located at www.LYVISPAH.com

To report SUSPECTED ADVERSE REACTIONS, contact Saol Therapeutics at toll-free phone 1-833 644- 4216 or FDA at 1-800- FDA-1088 or www.fda.gov/medwatch.

CYTOGAM® (Cytomegalovirus Immune Globulin Intravenous [Human]) (CMV-IGIV) Cytogam is contraindicated in individuals with a history of a prior severe reaction associated with the administration of this or other human immunoglobulin preparations. Persons with selective immunoglobulin A deficiency have the potential for developing antibodies to immunoglobulin A and could have anaphylactic reactions to subsequent administration of blood products that contain immunoglobulin A, including Cytogam. Immune Globulin Intravenous (Human) products have been reported to be associated with renal dysfunction, acute renal failure, osmotic nephrosis, and death. Patients predisposed to acute renal failure include patients with any degree of preexisting renal insufficiency, diabetes mellitus, age greater than 65, volume depletion, sepsis, paraproteinemia, or patients receiving known nephrotoxic drugs. Especially in such patients, IGIV products should be administered at the minimum concentrations available and the minimum rate of infusion practicable. Agents containing sucrose as a stabilizer (Cytogam contains sucrose) have been associated with reports of renal dysfunction given at daily doses of 350 mg/kg or greater.

HEPAGAM B® Hepatitis B Immune Globulin (Human) (HBIg) Individuals known to have severe, potentially life-threatening reaction to human globulin preparations should not receive HepaGam B® or any other immune globulin (human). Individuals who are deficient in IgA may have the potential to develop IgA antibodies and have severe, potentially life-threatening allergic reactions. For post-exposure prophylaxis indications, HepaGam B® must be administered intramuscularly only. In patients who have severe thrombocytopenia or any coagulation disorder that would contraindicate intramuscular injections, HepaGam B® should be given only if the expected benefits outweigh the potential risks. HepaGam B® [Hepatitis B Immune Globulin Intravenous (Human)] is a sterile solution of gamma globulin (IgG) made from human plasma. Products made from human plasma may carry a risk of transmitting infectious agents, eg, viruses and, theoretically, the Creutzfeldt-Jacob disease (CJD) agent.

VARIZIG® [Varicella Zoster Immune Globulin (Human)] In patients who have severe thrombocytopenia or any coagulation disorder that would contraindicate intramuscular injections, only administer VARIZIG if the expected benefits outweigh the potential risks. Thrombotic events may occur following treatment with VARIZIG and other immune globulin products. Individuals known to have severe, potentially life-threatening reactions to human globulin should not receive VARIZIG or any other immune globulin (Human). Individuals who are deficient in IgA may have the potential for developing IgA antibodies and have severe, potentially life-threatening allergic reactions. Products made from human plasma may carry a risk of transmitting infectious agents, e.g. viruses and, theoretically, the Creutzfeldt-Jakob disease agent. The most serious adverse drug reactions observed in clinical trials for all subjects and patients include pyrexia, nausea, chills and vomiting. The most common adverse drug reactions observed in clinical trials for all subjects and patients were injection site pain, headache, chills, fatigue, rash and nausea.

WINRHO® SDF Rho(D) Immune Globulin Intravenous (Human)

WARNING: INTRAVASCULAR HEMOLYSIS (IVH) Intravascular hemolysis leading to death has been reported in patients treated for ITP with WinRho® SDF.

IVH can lead to clinically compromising anemia and multi-system organ failure including acute respiratory distress syndrome (ARDS).

Serious complications including severe anemia, acute renal insufficiency, renal failure, and disseminated intravascular coagulation (DIC) have also been reported.

Closely monitor patients treated with WinRho® SDF for ITP in a healthcare setting for at least eight hours after administration. Perform a dipstick urinalysis to monitor for hematuria and hemoglobinuria at baseline and 2 hours, 4 hours, and prior to the end of the monitoring period. Alert patients and monitor the signs and symptoms of IVH including back pain, shaking chills, fever, and discolored urine or hematuria. Absence of these signs and/or symptoms of IVH within eight hours does not indicate IVH cannot occur subsequently. If signs and/or symptoms of IVH are present or suspected after WinRho® SDF administration, post-treatment laboratory tests should be performed including plasma hemoglobin, haptoglobin, LDH, and plasma bilirubin (direct and indirect).

For use in the treatment of ITP, do not use WinRho® SDF in:

• Patients who have had known anaphylactic or severe systemic reaction to the administration of human immune globulin products
• A deficient patients with antibodies to IgA and a history of hypersensitivity
• Patients with autoimmune hemolytic anemia, with pre-existing hemolysis or at high risk for hemolysis

The liquid formulation of WinRho® SDF contains maltose. Maltose in IGIV products has been shown to give falsely high blood glucose levels in certain types of blood glucose testing systems. Due to the potential for falsely elevated glucose readings, only testing systems that are glucose-specific should be used to test or monitor blood glucose levels in patients receiving WinRho® SDF Liquid. WinRho® SDF is made from human plasma. It may carry a risk of transmitting infectious agents, e.g. viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent.

The safety and efficacy of WinRho® SDF have not been evaluated in clinical trials for patients with nonITP causes of thrombocytopenia or in previously splenectomized patients or in patients who are Rho(D)- negative.