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, nerve-blocking agent 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 nerve blocking agent 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

Amneal Acquires Saol Therapeutics’ Baclofen Franchise

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 LYVISPAHTM 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. LYVISPAHTM 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 LYVISPAHTM 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 LYVISPAHTM, 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

LYVISPAHTM: 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 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

Ipsen announces Dysport® (abobotulinumtoxinA) co-promotion agreement with Saol Therapeutics to expand commercial reach in the United States

Saol sales team to promote Dysport® for select approved therapeutic indications in U.S. hospitals

BASKING RIDGE, N.J., June 30, 2017 – Ipsen Biopharmaceuticals, Inc., an affiliate of Ipsen SA (Euronext: IPN; ADR: IPSEY) (Ipsen), today announced that it has entered into an exclusive, three-year agreement with Saol Therapeutics Inc. to promote Dysport® (abobotulinumtoxinA) for injection for approved therapeutic indications in adult spasticity and pediatric lower limb spasticity in the United States.

“By adding the Saol team's extensive experience with physicians in the hospital setting to our existing efforts, we are able to educate more U.S. healthcare professionals on Dysport® ,” said Cynthia Schwalm, President, North American Commercial Operations, Ipsen. “As the only botulinum toxin approved by the FDA for the treatment of spasticity in adults in upper and lower limbs, and also for the treatment of lower limb spasticity in children ages two and older, it is critical to raise awareness of Dysport® as a potential option for appropriate patients.”

Dysport® and all botulinum toxin products have a Boxed Warning which states that the effects of the botulinum toxin may spread from the area of injection to other areas of the body, causing symptoms similar to those of botulism. Those symptoms include swallowing and breathing difficulties that can be life-threatening. Dysport® is contraindicated in patients with known hypersensitivity to any botulinum toxin preparation or to any of the components; or in the presence of infection at the proposed injection site(s); or in patients known to be allergic to cow's milk protein. The potency Units of Dysport® are specific to the preparation and assay method utilized. They are not interchangeable with other preparations of botulinum toxin products. Please see below for additional Important Safety Information.

Under the terms of the agreement, Saol’s sales force will promote Dysport® to healthcare professionals largely in the hospital setting beginning August 2017. Ipsen will maintain its current number of sales representatives fully dedicated to Dysport® including all its therapeutic indications. Additional details of the agreement are not disclosed.

Based in Roswell, GA, Saol Therapeutics is a privately held specialty pharmaceutical company focused on providing therapies to patients with unmet medical needs. The company has a strategic emphasis on spasticity and neurologic areas. Saol currently markets Lioresal® Intrathecal (baclofen injection), the first FDA-approved intrathecal baclofen for the treatment of severe spasticity. By detailing both products, Saol believes it can further support healthcare professionals and the patients they serve with forms of spasticity that each product is FDA approved to treat.

“At Saol, we are committed to the treatment of patients with spasticity. As a cornerstone to that, we believe it is important that physicians are made aware of available treatment options,” said Saol Therapeutics Chief Executive Officer, David Penake. “Our agreement with Ipsen helps us in PRESS RELEASE 2 that mission. It also allows us to align with a company that matches our passion for doing everything we can to support and educate physicians. We look forward to working with Ipsen and growing our organization, with the goal of helping patients in the United States.”

Please find included Important Safety Information – including BOX WARNINGS – for Dysport® and Lioresal® Intrathecal (baclofen injection).

About Dysport® (abobotulinumtoxinA) for Injection

Dysport® is an injectable form of botulinum toxin type A (BoNT-A), which is isolated and purified from Clostridium bacteria producing BoNT-A. It is supplied as a lyophilized powder. Dysport® has approved indications in the United States for the treatment of adults with Cervical Dystonia (CD) and for the treatment of spasticity in adult patients. Dysport® is also the first and only FDAapproved botulinum toxin for the treatment of lower limb spasticity in pediatric patients two years of age and older.

About IPSEN CARES ®

IPSEN CARES® (Coverage, Access, Reimbursement, & Education Support) is dedicated to ensuring patients, providers and caregivers have the resources needed to help access the Ipsen medications that are critical to managing their conditions. IPSEN CARES® is staffed Monday to Friday by experts who can assist with a broad range of medical, educational, logistical and coverage information regarding Ipsen medicines. Involving the entire treatment team that surrounds patients on a daily basis, IPSEN CARES® can provide benefits verification (research of a patient’s medical or pharmacy benefit insurance coverage); prior authorization information; a patient assistance program (free medications for uninsured patients); co-pay assistance programs for eligible patients; billing and coding support; coordination with specialty pharmacies. Additional information is also available by visiting (http://www.ipsencares.com).

INDICATIONS AND IMPORTANT SAFETY INFORMATION

DYSPORT® INDICATIONS

Dysport® (abobotulinumtoxinA) for injection is indicated for the treatment of:

  • Spasticity in adult patients
  • Adults with cervical dystonia
  • Lower limb spasticity in pediatric patients 2 years of age and older.

The safety and effectiveness of Dysport® injected into upper limb muscles or proximal muscles of the lower limb for the treatment of spasticity in pediatric patients has not been established.

Safety and effectiveness in pediatric patients with lower limb spasticity below 2 years of age have not been evaluated. Safety and effectiveness in pediatric patients with cervical dystonia or upper limb spasticity have not been established.

IMPORTANT SAFETY INFORMATION

Warning: Distant Spread of Toxin Effect Postmarketing reports indicate that the effects of Dysport® and all botulinum toxin products may spread from the area of injection to produce symptoms consistent with botulinum toxin effects. These may include asthenia, generalized muscle weakness, diplopia, blurred vision, ptosis, dysphagia, dysphonia, dysarthria, urinary incontinence, and breathing difficulties. These symptoms have been reported hours to weeks after injection. Swallowing and breathing difficulties can be life threatening and there have 3 been reports of death. The risk of symptoms is probably greatest in children treated for spasticity, but symptoms can also occur in adults treated for spasticity and other conditions, particularly in those patients who have underlying conditions that would predispose them to these symptoms. In unapproved uses, including upper limb spasticity in children, and in approved indications, cases of spread of effect have been reported at doses comparable to lower than the maximum recommended total dose.

Contraindications

Dysport® is contraindicated in patients with known hypersensitivity to any botulinum toxin preparation or to any of the components; or in the presence of infection at the proposed injection site(s); or in patients known to be allergic to cow’s milk protein. Hypersensitivity reactions including anaphylaxis have been reported.

Warnings and Precautions

Lack of interchangeability between botulinum toxin products

The potency Units of Dysport® are specific to the preparation and assay method utilized. They are not interchangeable with other preparations of botulinum toxin products, and, therefore, units of biological activity of Dysport® cannot be compared to or converted into units of any other botulinum toxin products assessed with any other specific assay method.

Dysphagia and Breathing Difficulties

Treatment with Dysport® and other botulinum toxin products can result in swallowing or breathing difficulties. Patients with pre-existing swallowing or breathing difficulties may be more susceptible to these complications. In most cases, this is a consequence of weakening of muscles in the area of injection that are involved in breathing or swallowing. When distant side effects occur, additional respiratory muscles may be involved (see Boxed Warning). Deaths as a complication of severe dysphagia have been reported after treatment with botulinum toxin. Dysphagia may persist for several weeks, and require use of a feeding tube to maintain adequate nutrition and hydration. Aspiration may result from severe dysphagia and is a particular risk when treating patients in whom swallowing or respiratory function is already compromised. Patients treated with botulinum toxin may require immediate medical attention should they develop problems with swallowing, speech, or respiratory disorders. These reactions can occur within hours to weeks after injection with botulinum toxin.

Pre-existing Neuromuscular Disorders

Individuals with peripheral motor neuropathic diseases, amyotrophic lateral sclerosis, or neuromuscular junction disorders (eg, myasthenia gravis or Lambert-Eaton syndrome) should be monitored particularly closely when given botulinum toxin. Patients with neuromuscular disorders may be at increased risk of clinically significant effects including severe dysphagia and respiratory compromise from typical doses of Dysport® .

Human Albumin and Transmission of Viral Diseases

This product contains albumin, a derivative of human blood. Based on effective donor screening and product manufacturing processes, it carries an extremely remote risk for transmission of viral diseases and variant Creutzfeldt-Jakob disease (vCJD). There is a theoretical risk for transmission of Creutzfeldt-Jakob disease (CJD), but if that risk actually exists, the risk of transmission would also be considered extremely remote. No cases of transmission of viral diseases, CJD, or vCJD have ever been identified for licensed albumin or albumin contained in other licensed products.

Intradermal Immune reaction

The possibility of an immune reaction when injected intradermally is unknown. The safety of Dysport® for the treatment of hyperhidrosis has not been established. Dysport® is approved only for intramuscular injection.

Adverse reactions

Most common adverse reactions (≥2% and greater than placebo in either Dysport® group) in adults with upper limb spasticity for Dysport® 500 Units, Dysport® 1000 Units, and Placebo, respectively, were: nasopharyngitis (4%, 1%, 1%), urinary tract infection (3%, 1%, 2%), muscular weakness (2%, 4%, 1%), musculoskeletal pain (3%, 2%, 2%), dizziness (3%, 1%, 1%), fall (2%, 3%, 2%), and depression (2%, 3%, 1%).

Most common adverse reactions (≥ 5% and greater than placebo in either Dysport® group) in adults with lower limb spasticity for Dysport® 1000 Units, Dysport® 1500 Units, and Placebo, respectively, were: falls (9%, 6%, 3%), muscular weakness (2%,7%, 3%), pain in extremity(6%, 6%, 2%). Muscular weakness was reported more frequently in women (10%) treated with 1500 units of Dysport compared to men (5%). Most common adverse reactions (≥5% and greater than placebo) in adults with cervical dystonia for Dysport® 500 Units and Placebo, respectively, were: muscular weakness (16%, 4%), dysphagia (15%, 4%), dry mouth (13%, 7%), injection site discomfort (13%, 8%), fatigue (12%, 10%), headache (11%, 9%), musculoskeletal pain (7%, 3%), dysphonia (6%, 2%), injection site pain (5%, 4%), and eye disorders (7%, 2%).

Most common adverse reactions (≥10% in any group and greater than placebo) in pediatric patients with lower limb spasticity for Dysport® 10 Units/kg, 15 Units/kg, 20 Units/kg, or 30 Units/kg; and Placebo, respectively, were: upper respiratory tract infection (9%, 20%, 5%, 10%, 13%), nasopharyngitis (9%, 12%,16%, 10%, 5%), influenza (0%, 10%, 14%, 3%, 8%), pharyngitis (5%, 0%,11%, 3%, 8%), cough (7%, 6%, 14%, 10%, 6%), and pyrexia (7%, 12%, 8%, 7%, 5%).

Drug interactions

Co-administration of Dysport® and aminoglycosides or other agents interfering with neuromuscular transmission (e.g., curare-like agents), or muscle relaxants, should be observed closely because the effect of botulinum toxin may be potentiated. Use of anticholinergic drugs after administration of Dysport® may potentiate systemic anticholinergic effects such as blurred vision. The effect of administering different botulinum neurotoxins at the same time or within several months of each other is unknown. Excessive weakness may be exacerbated by another administration of botulinum toxin prior to the resolution of the effects of a previously administered botulinum toxin. Excessive weakness may also be exaggerated by administration of a muscle relaxant before or after administration of Dysport® .

Use in Pregnancy

Based on animal data Dysport® may cause fetal harm. There are no adequate and wellcontrolled studies in pregnant women. Dysport® should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus.

Pediatric Use

Based on animal data Dysport® may cause atrophy of injected and adjacent muscles; decreased bone growth, length, and mineral content; delayed sexual maturation; and decreased fertility.

Geriatric Use

In general, elderly patients should be observed to evaluate their tolerability of Dysport® , due to the greater frequency of concomitant disease and other drug therapy. Subjects aged 65 years and over who were treated with DYSPORT® for lower limb spasticity reported a greater percentage of fall and asthenia as compared to those younger (10% versus 6% and 4% versus 2%, respectively).

To report SUSPECTED ADVERSE REACTIONS or product complaints, contact Ipsen at 1-855- 463-5127. You may also report SUSPECTED ADVERSE REACTIONS to the FDA at 1-800- FDA-1088 or www.fda.gov/medwatch.

Please see Dysport® Full Prescribing Information including Boxed Warning and Medication Guide.

INDICATIONS AND IMPORTANT SAFETY INFORMATION

Lioresal® Intrathecal (baclofen injection)

Indications and Usage

  • Lioresal® Intrathecal (baclofen injection) is a muscle relaxant and antispastic that is indicated for use in the management of severe spasticity of cerebral or spinal origin.
  • Lioresal® Intrathecal is intended for use by the intrathecal route in single bolus test doses (via spinal catheter or lumbar puncture) and, for chronic use, only in implantable pumps approved by the FDA specifically for the administration of Lioresal® Intrathecal into the intrathecal space.
  • Lioresal® Intrathecal via an implantable pump should be reserved for patients unresponsive to oral baclofen therapy or those who experience intolerable CNS side effects at effective doses.
  • Patients with spasticity due to traumatic brain injury should wait at least one year after the injury before consideration of long term intrathecal baclofen therapy.
  • Prior to implantation of a device for chronic intrathecal infusion of Lioresal® Intrathecal, patients must show a response to Lioresal® Intrathecal in a screening trial. Please review the dosing and administration section of the Lioresal® Intrathecal prescribing information for further details.

Contraindications

  • Hypersensitivity to baclofen
  • Lioresal® Intrathecal is not recommended for intravenous, intramuscular, subcutaneous or epidural administration.

Select Warnings and Precautions

  • It is mandatory that all patients, caregivers, and treating physicians receive adequate information regarding the risks of the mode of treatment. Instruction should be given on signs and symptoms of overdose, procedures to be followed in the event of an overdose, and proper home care of the pump and insertion site.
  • Due to the possibility of life-threatening CNS depression, cardiovascular collapse, and/or respiratory failure, physicians must be adequately trained and educated in chronic intrathecal infusion therapy.
  • Patients should be infection-free prior to both a screening trial and a pump implantation. The presence of infection may interfere with an assessment of the patient’s response to bolus Lioresal® Intrathecal, increase the risk of surgical complications and complicate dosing.
  • Reservoir refilling must be performed by fully trained and qualified personnel following the directions provided by the pump manufacturer. Extreme caution must be used when filling an FDA approved implantable pump, following strict aseptic technique and ensuring refill directly into the reservoir and not the catheter access port.
  • An attempt should be made to discontinue concomitant oral antispasticity medication to avoid possible overdose or adverse drug interactions, either prior to screening or following implant and initiation of chronic Lioresal® Intrathecal infusion.
  • Following pump implantation, and for each adjustment of the dosing rate of the pump and/or concentration of Lioresal® Intrathecal, the patient should be monitored closely until it is certain the patient’s response to the infusion is acceptable and reasonably stable.
  • Early symptoms of baclofen withdrawal may include return of baseline spasticity, pruritus, hypotension and paresthesias.
  • Priapism may develop or recur if treatment with intrathecal baclofen is interrupted.
  • Signs of overdose may appear suddenly or insidiously, and a massive overdose may present as coma. Less sudden and/or less severe forms of overdose may present with signs of drowsiness, lightheadedness, dizziness, somnolence, respiratory depression, seizures, rostral progression of hypotonia and loss of consciousness progressing to coma.
  • Should overdose appear likely, the patient should be taken immediately to a hospital for assessment and emptying of pump reservoir.
  • Except in overdose related emergencies, the dose of Lioresal® Intrathecal should ordinarily be reduced slowly if the drug is discontinued for any reason.

Adverse Reactions Common Adverse Reactions

  • The most frequent drug adverse events vary by indication but include: hypotonia (34.7%), somnolence (20.9%), headache (10.7%), convulsion (10.0%), dizziness (8.0%), urinary retention (8.0%), nausea (7.3%), and paresthesia (6.7%). Dosing and programming errors may result in clinically significant overdose or withdrawal. Acute massive overdose may result in coma and may be life threatening.
  • Drowsiness has been reported in patients on Lioresal® Intrathecal. Patients should be cautioned regarding the operation of automobiles or other dangerous machinery and activities made hazardous by decreased alertness. Patients should also be cautioned that the central nervous system depressant effects of Lioresal® Intrathecal may be additive to those of alcohol and other CNS depressants.

Serious Adverse Reactions

  • Seizures have been reported during overdose and with withdrawal from Lioresal® Intrathecal as well as in patients maintained on therapeutic doses of Lioresal® Intrathecal.
  • Fatalities have been reported with Lioresal® Intrathecal use.

Postmarketing Experience

  • The following adverse events have been reported during post-approval use of Lioresal® Intrathecal. o Musculoskeletal
    • The onset of scoliosis or worsening of a pre-existing scoliosis has been reported. o Urogenital
    • Sexual dysfunction in men and women including decreased libido and orgasm dysfunction have been reported.

Use in Specific Populations

  • There are no adequate and well controlled studies in pregnant women. Lioresal® Intrathecal 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. ▪ Safety and effectiveness in pediatric patients below the age of 4 have not been established.
  • Patients suffering from psychotic disorders, schizophrenia, or confusional states should be treated cautiously with Lioresal® Intrathecal and kept under careful surveillance.
  • Lioresal® Intrathecal should be given with caution in patients with impaired renal function. Dose reduction may be necessary.
  • Lioresal® Intrathecal should be used with caution in patients with a history of autonomic dysreflexia.

For more information, including BOX WARNING, refer to Lioresal® Intrathecal (baclofen injection) prescribing information, located here.

About Ipsen in North America

Ipsen Biopharmaceuticals, Inc. is the US affiliate of Ipsen, a global specialty-driven biopharmaceutical group. The US head office is located in Basking Ridge, New Jersey. Ipsen Biopharmaceuticals Canada, Inc. is an integrated business unit within North America and has its head office located in Mississauga, Ontario. Ipsen Bioscience, Inc., the Ipsen US research and development center focused on the discovery of highly differentiated and competitive products in oncology and rare diseases, is located in Cambridge, Massachusetts. At Ipsen Bioscience, we focus on creating a highly cooperative and passionate R&D organization through partnerships, innovation, and continuous learning to effectively deliver new treatments for patients. At Ipsen, we focus our resources, investments, and energy on discovering, developing, and commercializing new therapeutic options for oncologic, neurologic, and endocrine diseases. For more information on Ipsen in North America, please visit www.ipsenus.com or www.ipsen.ca.

About Ipsen

Ipsen is a global specialty-driven biopharmaceutical group focused on innovation and specialty care. The group develops and commercializes innovative medicines in three key therapeutic areas - Oncology, Neurosciences and Rare Diseases. Its commitment to oncology is exemplified through its growing portfolio of key therapies for prostate cancer, neuroendocrine tumors, renal cell carcinoma and pancreatic cancer. Ipsen also has a well-established Consumer Healthcare business. With total sales close to €1.6 billion in 2016, Ipsen sells more than 20 drugs in over 115 countries, with a direct commercial presence in more than 30 countries. Ipsen's R&D is focused on its innovative and differentiated technological platforms located in the heart of the leading biotechnological and life sciences hubs (Paris-Saclay, France; Oxford, UK; Cambridge, US). The Group has about 5,100 employees worldwide. Ipsen is listed in Paris (Euronext: IPN) and in the United States through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information on Ipsen, visit www.ipsen.com.

Forward Looking Statements

The forward-looking statements, objectives and targets contained herein are based on the Group's management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect the Group's future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words "believes," "anticipates" and "expects" and similar expressions are intended to identify forwardlooking statements, including the Group's expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by the Group. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising product in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. The Group must face or might face competition from generic products that might translate into a loss of market share. Furthermore, the Research and Development process involves several stages each of which involves the substantial risk that the Group may fail to achieve its objectives and be forced to abandon its efforts with regards to a product in which it has invested significant sums. Therefore, the Group cannot be certain that favorable results obtained during pre-clinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the product concerned. There can be no guarantees a product will receive the necessary regulatory approvals or that the product will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the Group's ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the Group's patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. The Group also depends on third parties to develop and market some of its products which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to the Group's activities and financial results. The Group cannot be certain that its partners will fulfill their obligations. It might be unable to obtain any benefit from those agreements. A default by any of the Group's partners could generate lower revenues than expected. Such situations could have a negative impact on the Group's business, financial position or performance. The Group expressly disclaims any obligation or undertaking to update or revise any forward looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. The Group's business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers.

 

For further information:

Media

Marisol Peron

Vice President, North American Internal & External Communications

Tel.: 212-257-6724

E-mail: marisol.peron@ipsen.com

 

Elliot Fox

W2O Group

Tel.: 908-275-6330

E-mail: efox@w2ogroup.com

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References

  1. Martin A, et al. Epidemiological, humanistic, and economic burden of illness of lower limb spasticity in adults: a systematic review. Neuropsychiatric Disease and Treatment. 2014; 10 (111-122)
  2. Sköld A, et al. Spasticity after traumatic spinal cord injury: nature, severity, and location. Archives of Physical Medicine and Rehabilitation. 1999; 80 (1548-57)
  3. National Institute of Neurological Disorders and Stroke. Spasticity Information Page. https://www.ninds.nih.gov/Disorders/All-Disorders/Spasticity-Information-Page Accessed May 16, 2017.
  4. Gray H. Anatomy of the Human Body. “The Muscles and Fasciæ of the Leg.” http://www.bartleby.com/107/129.html. Accessed June 23, 2016.
  5. Delgado M, et al. AbobotulinumtoxinA for equinus foot deformity in cerebral palsy: A randomized clinical trial. Pediatrics. 2016;137(2).

Dysport® (abobotulinumtoxinA) for injection, for intramuscular use 300- and 500-Unit vials.

DYSPORT is a registered trademark of Ipsen Biopharm Limited.

IPSEN CARES is a registered trademark of Ipsen S.A. Lioresal® is a registered trademark of Saol International Limited Corporation.

© 2017 Ipsen Biopharmaceuticals, Inc. June 2017 DYS-US-001875

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.