CSL Behring to Acquire Biotech Company Calimmune and its Proprietary Stem Cell Gene Therapy Platform

KING OF PRUSSIA, Pa., Aug. 28, 2017 /PRNewswire/ -- Global biotherapeutics leader CSL Behring announced today that it has agreed to acquire Calimmune, Inc., a biotechnology company focused on the development of ex vivo hematopoietic stem cell (HSC) gene therapy with R&D facilities in Pasadena, California and Sydney, Australia for an upfront payment of $91 million. 

The acquisition will provide CSL Behring with Calimmune's pre-clinical asset, CAL-H, an HSC gene therapy for the treatment of sickle cell disease and β-thalassemia, which complements CSL Behring's current product portfolio and deep expertise in hematology.

Additionally, CSL Behring will acquire two unique proprietary platform technologies, Select+™ and Cytegrity™. These technologies are designed to address some of the major challenges currently associated with the commercialization of stem cell therapy, including the ability to manufacture consistent, high-quality products, and to improve engraftment, efficacy and tolerability. Both technologies have broad applications in ex vivo stem cell gene therapy.

"Calimmune shares in our promise and focus to improve the lives of patients with rare and serious medical conditions," said CSL Limited Chief Executive Officer and Managing Director, Paul Perreault. "The acquisition represents another important step in the execution of our strategy for sustainable growth."

"Calimmune's scientific accomplishments are impressive," Perreault added. "The team has built a robust technology platform, and designed a promising HSC gene therapy candidate - CAL-H, which strongly aligns with our longer-term strategic goals, and complements our core competencies and areas of therapeutic focus.  While Calimmune is still in the early stages, we believe that our combined strengths have tremendous potential to change treatment paradigms, and most importantly, significantly improve the lives of our patients."

Calimmune Chief Executive Officer Louis Breton said, "We are excited to become part of CSL Behring. They are an established global industry leader in protein-replacement therapies and have a proven track record of driving innovations through the development pipeline and delivering differentiated products to the global marketplace. Together, we are well positioned to take our achievements to the next level."

CAL-H, Calimmune's HSC gene therapy for sickle cell and β-thalassemia, employs both the Select+™ system, and the Cytegrity™ virus production platform.  CAL-H has yielded early positive preclinical results and demonstrates the potential to offer a significant advantage to patients suffering from these currently incurable genetic diseases. 

Both proprietary technologies have the potential to be used in treatments for a wide range of other rare diseases that would complement CSL Behring's business, including those within the company's current product portfolio.

About Sickle Cell Disease and β thalassemia
Sickle cell disease and β-thalassemia are inherited disorders that affect hemoglobin, the protein in red blood cells that carries oxygen to different parts of the body. They are chronic diseases that dramatically impair the function of many organs and are associated with substantial morbidity, poor quality of life and a shortened life expectancy. The severe forms of both these diseases remain areas of high unmet need with sickle cell disease affecting approximately 150,000 Americans and Europeans and β-thalassemia approximately 16,000.  Although there are effective treatments available to relieve the symptoms of these diseases, there are no disease modifying treatments and in many cases regular blood transfusions are also required. Bone marrow transplant has been shown to be an effective cure in children, however, is rarely done due to the lack of closely matched donors.

About Select+™
Calimmune's Select+^TM is a proprietary technology aimed at driving selection of the genetically modified stem cells once they are given back to patients, to decrease toxicity and improve efficacy. One of the historical challenges for gene therapy is achieving a high enough engraftment of stem cells in the bone marrow to reach the relevant therapeutic window. Toxic conditioning regimens used to drive engraftment of gene modified cells can cause a range of adverse events that often require hospitalization and have additional long-term risks. Calimmune has focused on and made significant investments in solving this issue with Select+^TM. The combination of Select+^TM and lentiviral therapeutic applications aims to reduce the conditioning regimens, increase engraftment and overall efficacy, and improve the patient experience, ultimately making stem cell gene therapy an out-patient modality.

About Cytegrity™
Calimmune's Cytegrity^TM is a scalable manufacturing technology for the production of lentiviral vectors, which are used as a delivery mechanism for gene therapy. Lentiviral vectors are traditionally manufactured in small batches through a convoluted process; Cytegrity represents a new system that increases consistency and quality, and significantly lowers costs.

Transaction & Closing
CSL Behring will have operational control of Calimmune following closing of the transaction.  In addition to the upfront payment, the agreement between the parties includes the potential for performance based milestone payments of up to $325 million over a period currently anticipated to be around eight years or more following the closing of the transaction. The transaction is expected to close within the next two weeks, subject to the satisfaction of various closing conditions.

Weil, Gotshal & Manges LLP acted as legal advisor to CSL Behring.  Piper Jaffray & Co. acted as exclusive financial advisor and Cooley LLP acted as legal advisor to Calimmune.

About Calimmune
Calimmune is a privately owned company committed to accelerating the promise of gene therapy to liberate patients from chronic and currently incurable diseases. To achieve this ambitious goal, Calimmune has built a suite of technologies to advance the delivery, manufacturing, and overall efficiency of these life-changing medicines. Calimmune's lead development programs are novel ex vivo gene therapies for hematologic diseases. 

About CSL Behring
CSL Behring is a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients' needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, inherited respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn. 

CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX:CSL; USOTC:CSLLY), headquartered in Melbourne, Australia, employs nearly 20,000 people, and delivers its life-saving therapies to people in more than 60 countries. For more information visit www.cslbehring.com and follow us on www.Twitter.com/CSLBehring.

SOURCE CSL Behring

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