Press release d. 3. januar 2022
Hansa Biopharma enters into agreement with AskBio to evaluate feasibility of imlifidase as pre-treatment ahead of gene therapy in Pompe disease
Collaboration to evaluate imlifidase as potential pre-treatment prior to the administration of gene therapy in Pompe disease for patients with neutralizing antibodies (NAbs) to adeno-associated virus (AAV)
Lund, Sweden January 3, 2022. Hansa Biopharma AB, "Hansa", (Nasdaq Stockholm: HNSA), a pioneer in enzyme technology for rare immunological conditions, today announced that it has entered into an agreement with Asklepios BioPharmaceutical, Inc. ("AskBio"), a fully integrated AAV gene therapy company and a fully owned subsidiary of Bayer AG dedicated to developing medicines that improve the quality of life for patients with genetic diseases.
The collaboration will evaluate the potential use of imlifidase as a pre-treatment prior to the administration of AskBio's gene therapy in Pompe disease in a pre-clinical and clinical feasibility program for patients with pre-existing neutralizing antibodies (NAbs) to adeno-associated virus (AAV).
Pompe disease is a rare genetic and often fatal metabolic disorder that occurs in 1 in every 40,000 births. The disease is caused by the deficiency of an enzyme called acid alpha-glucosidase (GAA) and results in the accumulation of glycogen in organs and tissues, especially muscles, which impairs their ability to function normally.
Under the terms of the agreement, Hansa will receive a USD 5 million payment upon execution of the agreement and AskBio will have an exclusive option to enter into a full development and commercialization agreement following evaluation of the results from an initial Phase I/II study. AskBio will be responsible for conducting the pre-clinical and clinical studies with imlifidase under the feasibility program and Hansa will supply its materials and additional support set forth in the feasibility program.
"Neutralizing antibodies against AAV vectors used in a broad range of investigational gene therapies remain a major challenge and we see significant potential for our antibody-cleaving enzyme technology to help overcome this barrier. This collaboration with AskBio marks another important step in the implementation of our partnership strategy in the gene therapy space," said Søren Tulstrup, President & CEO of Hansa Biopharma.
This is information that Hansa Biopharma AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the contact persons set out below, at 18:35 CET on January 3 2022.
About Hansa Biopharma
Hansa Biopharma is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative, lifesaving and life altering treatments for patients with rare immunological conditions. Hansa has developed a first-in-class immunoglobulin G (IgG) antibody cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients. Hansa has a rich and expanding research and development program, based on the Company's proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in transplantation, autoimmune diseases, gene therapy and cancer. Hansa Biopharma is based in Lund, Sweden and has operations in Europe and the U.S. The Company is listed on Nasdaq Stockholm under the ticker HNSA. Find out more at https://hansabiopharma.com.
About AskBio
Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated AAV gene therapy company dedicated to developing medicines that improve the quality of life for patients with genetic diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson's disease and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. AskBio's gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, U.K., the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 800 patents in areas such as AAV production and chimeric and self-complementary capsids.
About Pompe disease
Pompe disease is a rare genetic, often fatal, disorder affecting 1 in 40,000 births in the United States. It is caused by a defect in a gene making an enzyme called acid alpha-glucosidase (GAA). GAA is used to break down glycogen (a sugar used to store energy in cells) and a defect GAA enzyme leads to accomulation of glycogen in the body's cells. The glycogen accumulation in certain organs and tissues, especially muscles, liver and heart, result in severe impact on the normal organ function. Up to 300 different mutations resulting in Pompe disease are identified and the severity and age of onset is related to the specific mutations. The most severe forms (early onset or infantile form) result in for example enlarged heart, muscle weakness, respiratory difficulties and most patients with the infantile form dies from cardiac or respiratory failure before their first birthday. The late onset or juvenile/adult form initially shows muscle weakness progressing to respiratory weakness and death from respiratory failure after several years. While enzyme replacement therapy (ERT) has shown promise in patients with infantile-onset Pompe disease, no curative therapy is available.
About imlifidase
Imlifidase is a unique antibody-cleaving enzyme originating from Streptococcus pyogenes that specifically targets IgG and inhibits IgG-mediated immune response. It has a rapid onset of action, cleaving IgG-antibodies and inhibiting their activity within hours after administration. Imlifidase has conditional marketing approval in Europe and is marketed under the trade name Idefirix® for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor.
For further information, please contact:
Klaus Sindahl, Head of Investor Relations
Hansa Biopharma
Mobile: +46 (0) 709-298 269
E-mail: [email protected]
Katja Margell
Head of Corporate Communications
Hansa Biopharma
Mobile: +46 (0) 768-198 326
Hansa Biopharma har en betalt digital IR-aftale med ProInvestor.com
Hansa Biopharma enters into agreement with AskBio to evaluate feasibility of imlifidase as pre-treatment ahead of gene therapy in Pompe disease
Collaboration to evaluate imlifidase as potential pre-treatment prior to the administration of gene therapy in Pompe disease for patients with neutralizing antibodies (NAbs) to adeno-associated virus (AAV)
Lund, Sweden January 3, 2022. Hansa Biopharma AB, "Hansa", (Nasdaq Stockholm: HNSA), a pioneer in enzyme technology for rare immunological conditions, today announced that it has entered into an agreement with Asklepios BioPharmaceutical, Inc. ("AskBio"), a fully integrated AAV gene therapy company and a fully owned subsidiary of Bayer AG dedicated to developing medicines that improve the quality of life for patients with genetic diseases.
The collaboration will evaluate the potential use of imlifidase as a pre-treatment prior to the administration of AskBio's gene therapy in Pompe disease in a pre-clinical and clinical feasibility program for patients with pre-existing neutralizing antibodies (NAbs) to adeno-associated virus (AAV).
Pompe disease is a rare genetic and often fatal metabolic disorder that occurs in 1 in every 40,000 births. The disease is caused by the deficiency of an enzyme called acid alpha-glucosidase (GAA) and results in the accumulation of glycogen in organs and tissues, especially muscles, which impairs their ability to function normally.
Under the terms of the agreement, Hansa will receive a USD 5 million payment upon execution of the agreement and AskBio will have an exclusive option to enter into a full development and commercialization agreement following evaluation of the results from an initial Phase I/II study. AskBio will be responsible for conducting the pre-clinical and clinical studies with imlifidase under the feasibility program and Hansa will supply its materials and additional support set forth in the feasibility program.
"Neutralizing antibodies against AAV vectors used in a broad range of investigational gene therapies remain a major challenge and we see significant potential for our antibody-cleaving enzyme technology to help overcome this barrier. This collaboration with AskBio marks another important step in the implementation of our partnership strategy in the gene therapy space," said Søren Tulstrup, President & CEO of Hansa Biopharma.
This is information that Hansa Biopharma AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the contact persons set out below, at 18:35 CET on January 3 2022.
About Hansa Biopharma
Hansa Biopharma is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative, lifesaving and life altering treatments for patients with rare immunological conditions. Hansa has developed a first-in-class immunoglobulin G (IgG) antibody cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients. Hansa has a rich and expanding research and development program, based on the Company's proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in transplantation, autoimmune diseases, gene therapy and cancer. Hansa Biopharma is based in Lund, Sweden and has operations in Europe and the U.S. The Company is listed on Nasdaq Stockholm under the ticker HNSA. Find out more at https://hansabiopharma.com.
About AskBio
Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated AAV gene therapy company dedicated to developing medicines that improve the quality of life for patients with genetic diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson's disease and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. AskBio's gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, U.K., the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 800 patents in areas such as AAV production and chimeric and self-complementary capsids.
About Pompe disease
Pompe disease is a rare genetic, often fatal, disorder affecting 1 in 40,000 births in the United States. It is caused by a defect in a gene making an enzyme called acid alpha-glucosidase (GAA). GAA is used to break down glycogen (a sugar used to store energy in cells) and a defect GAA enzyme leads to accomulation of glycogen in the body's cells. The glycogen accumulation in certain organs and tissues, especially muscles, liver and heart, result in severe impact on the normal organ function. Up to 300 different mutations resulting in Pompe disease are identified and the severity and age of onset is related to the specific mutations. The most severe forms (early onset or infantile form) result in for example enlarged heart, muscle weakness, respiratory difficulties and most patients with the infantile form dies from cardiac or respiratory failure before their first birthday. The late onset or juvenile/adult form initially shows muscle weakness progressing to respiratory weakness and death from respiratory failure after several years. While enzyme replacement therapy (ERT) has shown promise in patients with infantile-onset Pompe disease, no curative therapy is available.
About imlifidase
Imlifidase is a unique antibody-cleaving enzyme originating from Streptococcus pyogenes that specifically targets IgG and inhibits IgG-mediated immune response. It has a rapid onset of action, cleaving IgG-antibodies and inhibiting their activity within hours after administration. Imlifidase has conditional marketing approval in Europe and is marketed under the trade name Idefirix® for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor.
For further information, please contact:
Klaus Sindahl, Head of Investor Relations
Hansa Biopharma
Mobile: +46 (0) 709-298 269
E-mail: [email protected]
Katja Margell
Head of Corporate Communications
Hansa Biopharma
Mobile: +46 (0) 768-198 326
Hansa Biopharma har en betalt digital IR-aftale med ProInvestor.com
3/1 2022 20:06 ProInvestorNEWS 2
99870 Uddybning og perspektivering af aftalen af Klaus Sindahl, Head of Investor Relations, Hansa Biopharma:
"Background
· The background for the collaboration is to evaluate imlifidase as potential pre-treatment prior to the administration of AskBio's gene therapy in Pompe disease for patients with neutralizing antibodies (NAbs) to adeno-associated virus (AAV)
· AskBio has a phase I/II clinical program for Pompe disease with their gene therapy candidate "AAV2/8-LSPhGAA". The current clinical program is an open-label trial designed to objectively assess the safety and bioactivity of AAV2/8-LSPhGAA in subjects diagnosed with Pompe disease, which is caused by a defect in acid α-glucosidase (GAA) gene. AAV2/8-LSPhGAA is intended to enable expression of a functional copy of the GAA gene in subject's hepatocytes. https://clinicaltrials.gov/ct2/show/NCT03533673
· Imlfidase has in preclinical setting proofed to inactivate Nabs in a mouse model and in NHPs (Nature Medicine, June 2020, Leborgne et al.) and is currently being investigated in DMD and LGMD preclinically in a similar Hansa-Sarepta Therapeutics collaboration
· Pompe disease is a rare genetic and often fatal metabolic disorder that occurs in 1 in every 40,000 births. The disease is caused by a defect in a gene making an enzyme called acid alpha-glucosidase (GAA). GAA is used to break down glycogen (a plysaccharide of glucose used to store energy in cells) and a defect GAA enzyme leads to accumulation of glycogen in the body's cells. The glycogen accumulation in certain organs and tissues, especially muscles, liver and heart, result in severe impact on the normal organ function. The most severe form of the mutations results in enlarged heart, muscle weakness, respiratory difficulties and most patients with the infantile form dies from cardiac or respiratory failure before their first birthday. While enzyme replacement therapy (ERT) has shown promise in patients with infantile-onset Pompe disease, no curative therapy is available.
· Under the terms of the agreement, Hansa will receive a USD 5 million payment upon execution of the agreement and AskBio will have an exclusive option to enter into a full development and commercialization agreement following evaluation of the results from an initial Phase I/II study.
AskBio
AskBio is a US based wholly owned and independently operated subsidiary of Bayer AG (acquired in 2020 at a purchase price of USD 2bn) and a fully integrated AAV gene therapy company dedicated to developing medicines that improve the quality of life for patients with genetic diseases. Portfolio include clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications. AskBio was founded in 2001 and is characterized as an early innovator in the gene therapy field. The Company is headquartered in Research Triangle Park, North Carolina
Pompe disease prevalence
It is estimated that approximately 10,000* patients live with Pompe disease today in the EU and the US (*corrected - not 20,000 as previously shown in our chart). Approximately 200-250 children are born with Pompe in the EU and the US annually and the average life expectancy is 39 years. The specific prevalence of Nabs to the AskBio-GTx product remains to be seen but AskBio apparently assumes that the AAV8 is the main factor since they are excluding patients who are ELISA positive for anti-AAV8 capsid IgG antibodies in their currently ongoing study. It is estimated that between 40% and 60% of the prevalent and incident patients have anti-AAV8 antibodies
Financials
The USD 5m will be received as cash upon execution of the deal but will be recognized as deferred revenue over the time of the feasibility program (probably 24 months) as Hansa supply materials and support AskBio according to the terms of the agreement. There are no additional monetary milestone payments agreed in the current agreement - it is only scoped around evaluating feasibility of imlifidase ahead of AskBios gene therapy in Pompe disease during preclinical and initial clinical phase I/II.
"Following the results from the preclinical program and the initial Phase I/II study AskBio has an exclusive option to enter into a full development and commercialization agreement. If AskBio decides to enter into a full development and commercialization agreement it is likely that such an agreement will include the relevant milestone payments for the continued development until potential launch as well as royalties based on the incremental gene therapy sales enabled by imlifidase and sales from imlifidase to Hansa. Important to stress that such an agreement has not been agreed yet and is depending on successful outcome of the feasibility program but fair to assume that Hansa aims for a similar structure deal as Sarepta collaboration. Prevalence and incidences are however different in Pompe disease vs. DMD and Limb-Girdle
Agreement scope and structure
The scope of the agreement is currently focused on evaluation of the potential use of imlifidase in Pompe disease in a pre-clinical and clinical feasibility program for patients with preexisting Nabs to AAV. AskBio will be responsible for conducting the pre-clinical and clinical studies with imlifidase under the feasibility program and Hansa will supply its materials and additional support set forth in the feasibility program.
The structure of the agreement with AskBio is quite similar Sarepta. The major difference is that the Sarepta agreement includes late development work plus regulatory and commercialization activities and associated milestones. With AskBio we accommodate flexibility in two steps, starting with the establishment of clinical Proof-of-Concept for both parties to increase the visibility of the value imlifidase potentially will bring to AskBios gene therapy in Pompe, and then, following clinical PoC, we will define late development and commercialization activities and milestones".
Klaus Sindahl
Head of Investor Relations
Hansa Biopharma har en betalt digital IR-aftale med ProInvestor.com
"Background
· The background for the collaboration is to evaluate imlifidase as potential pre-treatment prior to the administration of AskBio's gene therapy in Pompe disease for patients with neutralizing antibodies (NAbs) to adeno-associated virus (AAV)
· AskBio has a phase I/II clinical program for Pompe disease with their gene therapy candidate "AAV2/8-LSPhGAA". The current clinical program is an open-label trial designed to objectively assess the safety and bioactivity of AAV2/8-LSPhGAA in subjects diagnosed with Pompe disease, which is caused by a defect in acid α-glucosidase (GAA) gene. AAV2/8-LSPhGAA is intended to enable expression of a functional copy of the GAA gene in subject's hepatocytes. https://clinicaltrials.gov/ct2/show/NCT03533673
· Imlfidase has in preclinical setting proofed to inactivate Nabs in a mouse model and in NHPs (Nature Medicine, June 2020, Leborgne et al.) and is currently being investigated in DMD and LGMD preclinically in a similar Hansa-Sarepta Therapeutics collaboration
· Pompe disease is a rare genetic and often fatal metabolic disorder that occurs in 1 in every 40,000 births. The disease is caused by a defect in a gene making an enzyme called acid alpha-glucosidase (GAA). GAA is used to break down glycogen (a plysaccharide of glucose used to store energy in cells) and a defect GAA enzyme leads to accumulation of glycogen in the body's cells. The glycogen accumulation in certain organs and tissues, especially muscles, liver and heart, result in severe impact on the normal organ function. The most severe form of the mutations results in enlarged heart, muscle weakness, respiratory difficulties and most patients with the infantile form dies from cardiac or respiratory failure before their first birthday. While enzyme replacement therapy (ERT) has shown promise in patients with infantile-onset Pompe disease, no curative therapy is available.
· Under the terms of the agreement, Hansa will receive a USD 5 million payment upon execution of the agreement and AskBio will have an exclusive option to enter into a full development and commercialization agreement following evaluation of the results from an initial Phase I/II study.
AskBio
AskBio is a US based wholly owned and independently operated subsidiary of Bayer AG (acquired in 2020 at a purchase price of USD 2bn) and a fully integrated AAV gene therapy company dedicated to developing medicines that improve the quality of life for patients with genetic diseases. Portfolio include clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications. AskBio was founded in 2001 and is characterized as an early innovator in the gene therapy field. The Company is headquartered in Research Triangle Park, North Carolina
Pompe disease prevalence
It is estimated that approximately 10,000* patients live with Pompe disease today in the EU and the US (*corrected - not 20,000 as previously shown in our chart). Approximately 200-250 children are born with Pompe in the EU and the US annually and the average life expectancy is 39 years. The specific prevalence of Nabs to the AskBio-GTx product remains to be seen but AskBio apparently assumes that the AAV8 is the main factor since they are excluding patients who are ELISA positive for anti-AAV8 capsid IgG antibodies in their currently ongoing study. It is estimated that between 40% and 60% of the prevalent and incident patients have anti-AAV8 antibodies
Financials
The USD 5m will be received as cash upon execution of the deal but will be recognized as deferred revenue over the time of the feasibility program (probably 24 months) as Hansa supply materials and support AskBio according to the terms of the agreement. There are no additional monetary milestone payments agreed in the current agreement - it is only scoped around evaluating feasibility of imlifidase ahead of AskBios gene therapy in Pompe disease during preclinical and initial clinical phase I/II.
"Following the results from the preclinical program and the initial Phase I/II study AskBio has an exclusive option to enter into a full development and commercialization agreement. If AskBio decides to enter into a full development and commercialization agreement it is likely that such an agreement will include the relevant milestone payments for the continued development until potential launch as well as royalties based on the incremental gene therapy sales enabled by imlifidase and sales from imlifidase to Hansa. Important to stress that such an agreement has not been agreed yet and is depending on successful outcome of the feasibility program but fair to assume that Hansa aims for a similar structure deal as Sarepta collaboration. Prevalence and incidences are however different in Pompe disease vs. DMD and Limb-Girdle
Agreement scope and structure
The scope of the agreement is currently focused on evaluation of the potential use of imlifidase in Pompe disease in a pre-clinical and clinical feasibility program for patients with preexisting Nabs to AAV. AskBio will be responsible for conducting the pre-clinical and clinical studies with imlifidase under the feasibility program and Hansa will supply its materials and additional support set forth in the feasibility program.
The structure of the agreement with AskBio is quite similar Sarepta. The major difference is that the Sarepta agreement includes late development work plus regulatory and commercialization activities and associated milestones. With AskBio we accommodate flexibility in two steps, starting with the establishment of clinical Proof-of-Concept for both parties to increase the visibility of the value imlifidase potentially will bring to AskBios gene therapy in Pompe, and then, following clinical PoC, we will define late development and commercialization activities and milestones".
Klaus Sindahl
Head of Investor Relations
Hansa Biopharma har en betalt digital IR-aftale med ProInvestor.com
