Saol Therapeutics News

Saol Therapeutics Announces First Patient Enrolled in Phase 2 RAISE Spasticity Trial Evaluating the Safety Profile of SL-1002 for Limb Spasticity

ROSWELL, Ga. and DUBLIN and HAMILTON, BermudaMay 3, 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 RAISE Spasticity Trial.

SL-1002 is a novel, proprietary chemoneurolytic injection currently under development at Saol Therapeutics and is being evaluated in the RAISE Spasticity Trial.  The trial is a 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.

"We are extremely excited to be working with Saol Therapeutics to investigate SL-1002 in the RAISE Spasticity Trial," said Principal Investigator John McGuire, a professor of Physical Medicine and Rehabilitation at the Medical College of Wisconsin.  "Despite the number of current treatments for spasticity, there remains a need for additional effective and reliable options."

"I am very proud of the work the team at Saol Therapeutics has done to achieve this important milestone for our company," said Saol Therapeutics CEO, David Penake.  "Saol has heard from countless physicians about the need for efficacious, long-acting therapeutic agents to help manage spasticity, and we are grateful for the partnership of our trial sites to help advance the development of this therapy."

In addition to the RAISE Spasticity Trial for the treatment of spasticity, Saol Therapeutics also announced a second open IND for SL-1002 for the treatment of pain related to osteoarthritis of the knee.  The first patient in that trial – the COMPASS Osteoarthritis Trial – is expected to be enrolled in Q2, 2022.

"Our recent strategic divestments have allowed Saol to expand investment in high-potential, clinical programs, including an additional study to evaluate the safety and efficacy of SL-1002 in patients with pain related to osteoarthritis of the knee. After successfully opening a second IND, we are ramping towards enrolling the first patient into the COMPASS Osteoarthritis Trial." added Penake.  "Additionally, beyond the treatment of spasticity and pain related to osteoarthritis of the knee, we believe SL-1002 could have applicability in multiple potential conditions that we are currently exploring."

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

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

Saol expects to begin enrolling patients in the COMPASS Osteoarthritis Trial in the 2nd Quarter of 2022.

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 clinical 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.

SOURCE: Saol Therapeutics

Saol Therapeutics News

Saol Therapeutics and InformedDNA Partner to Offer Genetic Counseling to Patients with Rare Mitochondrial Disease

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

ROSWELL, Ga. and ST. PETERSBURG, Fla.April 26, 2022 /PRNewswire/ -- Saol Therapeutics, a company researching new treatments for rare diseases, is pleased to announce a partnership with InformedDNA®, the nation's leading applied genomics solutions company, to offer genetic counseling for the families of individuals with a rare mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD).  The counseling will also review potential participation in a Phase 3 clinical trial. PDCD affects less than 300 children in the United States annually and lacks any FDA-approved treatment.

InformedDNA's board-certified genetic counselors are highly knowledgeable about inherited metabolic disorders and can answer questions and review eligibility to enable individuals to make more informed decisions about clinical trial participation. After speaking with a genetic counselor, families interested in learning more about the clinical research study will be connected with a trial site.

This pivotal phase 3 trial administers the investigational drug dichloroacetate (DCA) to 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 fatal.  DCA has Orphan Product designation from the FDA for congenital lactic acidosis (CLA), including patients with PDCD. (ClinicalTrials.gov link: Trial of DCA in Pyruvate Dehydrogenase Complex Deficiency)

Some of the questions and topics addressed during the genetic counseling appointment include:

  • Discussion of the clinical features, progression, and inheritance of Pyruvate Dehydrogenase Complex Deficiency (PDCD)
  • High-level overview of the clinical trial and participation

Dr. Peter Stacpoole, Principal Investigator of the DCA/PDCD trial and Prof. of Medicine at the University of Florida, notes that "There are currently no FDA-approved treatments for patients with PDCD. This service allows them to make an informed decision about clinical trial participation, which is critical to advancing treatment options for rare diseases."

Dave Penake, CEO of Saol Therapeutics, is pleased to partner with InformedDNA to provide this valuable service. "Individualized genetic counseling offers families the insights needed to better understand PDCD and to determine if participation in this clinical trial makes sense.  Completing the trial and having a potential FDA-approved treatment for PDCD will be a huge milestone for Saol and the families that have participated in the study."

"Like many rare genetic diseases, PDCD is a severe disease with no proven therapies. Many affected patients do not survive childhood. We are thrilled that Saol Therapeutics is investing in this rare disease, and we are passionate about leveraging InformedDNA's highly effective virtual screening process to engage and connect appropriate patients to the clinical trial and advance therapeutic progress in this devastating disease," said Karmen Trzupek, Director of Clinical Trial Services at InformedDNA.  Healthcare providers and caregivers for children with Pyruvate Dehydrogenase Complex Deficiency (PDCD) may request a genetic counseling appointment by visiting www.InformedDNA/Saol.

About Saol Therapeutics

Saol Therapeutics (pronounced "Sail") is a privately held, biopharmaceutical company with operations in Roswell, GADublin, 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 InformedDNA

InformedDNA is the country's leading applied genomics solutions company, helping harness the full power of precision medicine. With the largest independent staff of board-certified genetics specialists in the U.S., InformedDNA ensures that health organizations have access to the highest quality, most current genomics insights to optimize clinical decisions. Our solutions, which cover evidence-based guideline development, patient experience management, and value management, have helped optimize the health benefits of more than 135 million covered lives and have navigated hundreds of thousands of people to the right treatments or clinical trials. For more information, visit www.InformedDNA.com.

Clinical Trial Contact:

Kathy Dorsey, Director of Clinical Operations, Saol Therapeutics, KDorsey@Saolrx.com

Media Contacts:

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

Megan Smith, MERGE for InformedDNA, msmith@mergeworld.com, 404-408-3379

SOURCE: Saol Therapeutics

Saol Therapeutic News

Saol Therapeutics and GeneDx, Inc. Collaborate to Detect Patients with Rare Mitochondrial Disease

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

Saol Therapeutics News

Saol Therapeutics Announces FDA Approval of LYVISPAHTM (baclofen) Oral Granules and the Divesture of its Plasma-derived Hyperimmune Portfolio

Saol Therapeutics announces the approval of LYVISPAHTM (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.

LYVISPAHTM (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. LYVISPAHTM may also be of some value in patients with spinal cord injuries and other spinal cord diseases. LYVISPAHTM 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, "LYVISPAHTM represents an important treatment option for individuals with spasticity who have dysphagia. The bioequivalence between LYVISPAHTM 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 LYVISPAHTM. 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 LYVISPAHTM 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 LYVISPAHTM 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 LYVISPAHTM 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

LYVISPAHTM 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. LYVISPAHTM may also be of some value in patient with spinal cord injuries and other spinal cord diseases. LYVISPAHTM 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

LYVISPAHTM (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.

Saol Therapeutics News

Saol Therapeutics Announces that CDC Website has Been Updated to Reflect Wide Availability of VARIZIG® (Varicella Zoster Immune Globulin [Human])

Saol Therapeutics announces that the CDC website has been updated to reflect the wide availability of VARIZIG. The update notes that “VARIZIG is commercially available from a broad network of specialty distributors in the United States (list available at www.varizig.com).”

“This update is important for effective distribution and use of VARIZIG for these at-risk patient populations,” said Saol Therapeutics Chief Executive Officer, David Penake. “VARIZIG has a strong clinical value in the patient populations it serves, and we are pleased that it is now commercially available from a broad network of US specialty distributors. It is very important that all treating physicians realize that VARIZIG can be easily obtained within 24 hours for high-risk patients following exposure to varicella zoster virus.”

VARIZIG is an FDA-approved immunoglobulin for post-exposure prophylaxis of varicella in high-risk individuals, replacing VZIG (discontinued in 2006). VARIZIG is a hyperimmune product that contains antibodies specific for the varicella zoster virus, which causes the viral infection known as chickenpox.

Clinical Management of Exposure to Varicella

Despite being considered “benign” in healthy individuals, chickenpox and shingles may be responsible for health complications and death in high-risk patients.1-4 These patients may be exposed to varicella through chickenpox, or shingles.4 Exposure to chickenpox is considered serious and can occur in as little as 5 minutes depending on type of exposure.4 The CDC recommends VARIZIG for people exposed to varicella or herpes zoster who cannot receive varicella vaccine; varicella-zoster immune globulin can prevent varicella from developing or lessen the severity of the disease. Varicella-zoster immune globulin is recommended for people who cannot receive the vaccine and 1) who lack evidence of immunity to varicella, 2) whose exposure is likely to result in infection, and 3) are at high risk for severe varicella.5

Please see contraindications below in the Important Safety Information.

Evidence of immunity to varicella includes vaccination with varicella vaccine (1 dose ≥12 months through 3 years of age, 2 doses ≥4 years of age, adolescents, and adults: 2 doses), birth in the U.S. before 1980 (except for healthcare personnel, pregnant women, and immunocompromised persons), laboratory evidence of immunity or laboratory confirmation of disease, or history of varicella or herpes
zoster.6

High-risk patients include newborns with mothers having varicella symptoms around delivery; pregnant women without evidence of immunity; hospitalized infants born at or before 28 weeks and weighing less than 2 lbs; and immunocompromised patients without evidence of immunity, such as cancer patients, transplant recipients, and patients with autoimmune or immune-mediated inflammatory disorders. 5,7

Post-exposure Prophylaxis with VARIZIG® (Varicella Zoster Immune Globulin [Human])

VARIZIG is a single weight based IM injection intended to reduce the severity of varicella in at-risk patients.7

In an open-label expanded access protocol of over 500 high-risk individuals that received VARIZIG after exposure to chickenpox or shingles, a low percentage (<10%) developed clinical varicella. VARIZIG should ideally be administered within 96 hours for greatest effectiveness. However, a comparison of the incidence of clinical varicella based on treatment window revealed that treatment between 5 and 10 days post-exposure was no different from treatment within 96 hours. VARIZIG is commercially available from a broad network of specialty distributors in the United States (list available at www.varizig.com).7

IMPORTANT SAFETY INFORMATION ABOUT 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.

For further information about VARIZIG, contact Susan Clement, Senior Director, Marketing, Saol Therapeutics, at sclement@saolrx.com.

About Saol Therapeutics
Saol Therapeutics is a commercial specialty pharmaceutical company concentrated on addressing the medical needs of underserved or unserved patient populations and the physicians that treat them.

Further information:
www.VARIZIG.com
VARIZIG Full Prescribing Information
CDC. Updated Chickenpox (varicella): For Healthcare Professionals — 2019
CDC. Updated Recommendations for Use of VARIZIG — United States, 2013
Available VARIZIG Distributors

SOURCE:
Saol Therapeutics
www.saolrx.com

REFERENCES:
1. Gershon AA, Gershon MD. Pathogenesis and current approaches to control of varicella-zoster virus infections. Clin Microbiol Rev. 2013;26(4):728-43.
2. Gnann Jr. JW. Antiviral therapy of varicella-zoster virus infections. In: Arvin A, Campadelli-Fiume G, Mocarski E, et al., editors. Human Herpesviruses: Biology, Therapy, and Immunoprophylaxis. Cambridge: Cambridge University Press; 2007. Chapter 65. Available at: https://www.ncbi.nlm.nih.gov/books/NBK47401/. Accessed September 13, 2018.
3. Cohen JI. Herpes Zoster. N Engl J Med. 2013;369:255-63.
4. American Academy of Pediatrics. Varicella-Zoster Virus Infections. In: Kimberlin DW, Brady MT, Jackson MA, Long SS, eds. Red Book: 2018 Report of the Committee on Infectious Diseases. 31st ed. Itasca, IL: American Academy of Pediatrics; 2018:869-83.
5. Marin M, Bialek SR, Seward JF. Updated recommendations for use of VariZIG — United States, 2013. MMWR Morb Mortal Wkly Rep. 2013;62(28):574–576.
6. Centers for Disease Control and Prevention Yellow Book 2018: Health Information for International Travelers. Available at:
https://wwwnc.cdc.gov/travel/yellowbook/2018/infectious-diseases-related-to-travel/varicellachickenpox. Accessed September 13, 2018.
7. Saol Therapeutics Inc. VARIZIG Full Prescribing Information. Available from: https://varizig.com/VARIZIG_PI.pdf. Accessed September 13, 2018.

ST-203-1039-02

Saol Therapeutics News

Saol Therapeutics Acquires Three Hyperimmune Products from Aptevo Therapeutics

ROSWELL, GA - SEPTEMBER 1, 2017 –- Saol Therapeutics today announced it had reached an agreement with Aptevo Therapeutics Inc. (Nasdaq: APVO) to acquire three marketed hyperimmune products, WinRho® SDF (Rh0(D) Immune Globulin Intravenous (Human)), HepaGam B® (Hepatitis B Immune Globulin Intravenous (Human)) and VARIZIG® (Varicella Zoster Immune Globulin (Human)). The deal includes both upfront and milestone payments.

Under the terms of a purchase agreement executed by the companies, Saol Therapeutics will acquire the global rights to three hyperimmune products currently marketed by Aptevo: WinRho® SDF for autoimmune platelet disorder and hemolytic disease of the newborn; HepaGam B® for the prevention of Hepatitis B following liver transplantation and for treatment following hepatitis B exposure; and VARIZIG® for treatment following exposure to varicella zoster virus for individuals with compromised immune systems.

“This is a very important day for our company,” said Saol Therapeutics Chief Executive Officer David Penake. “These three products are tremendous strategic fits for us, as we look to build out our orphan business unit. They each have strong clinical value to the patient populations they serve, and we look forward to supporting each product and the clinicians that count on them around the world.”

The transaction, which is expected to be completed in 2017, is subject to certain customary closing conditions.

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Please see BOX WARNING for WinRho® SDF below, and find full prescribing information here.
For prescribing information of HepaGam B®, please click here.
For prescribing information of VARIZIG®, please click here.

WARNING for WinRho® SDF: INTRAVASCULAR HEMOLYSIS (IVH) IN IMMUNE THROMBOCYTOPENIC PURPURA (ITP)

See full prescribing information for complete boxed warning. This warning does not apply to Rh0 (D)-negative patients treated for the suppression of Rh isoimmunization.

  • Intravascular hemolysis (IVH) 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 (5.2.1). Perform 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 do not indicate IVH cannot occur subsequently. Perform post-treatment laboratory tests if signs and/or symptoms of IVH are present or suspected after WinRho SDF administration.