Industry Update: Business Development
Latest business developments compiled from July 01 — August 31 2016
Latest business developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions from July 01 — August 31 2016, scheduled to be published in Volume 11 Issue 8 of Regenerative Medicine.
Collaborations, partnerships & alliances
Co-development agreement: Thrive and HSCI
Thrive Bioscience (MA, www.thrivebio.com), a manufacturer of automated cell culture management systems, and the Harvard Stem Cell Institute (HSCI; MA, http://hsci.harvard.edu) announced an agreement under which Thrive will develop instruments for the growth and maintenance of induced pluripotent stem cells (iPSCs). The agreement includes development of automated iPSC protocols critical to HSCI and its researchers. HSCI plans to utilize the Thrive systems within the Harvard Stem Cell Institute iPSC Core Facility and affiliated research sites.
Collaboration agreement: Editas and SR-TIGET
Editas Medicine (MA, USA; www.editasmedicine.com) and Fondazione Telethon and Ospedale San Raffaele, which operate a joint research collaboration known as the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET; Italy: www.hsr.it/research/organization/institutes/sr-tiget), have entered into a scientific collaboration to research and develop genome edited hematopoietic stem cell (HSC) and T cell therapies.
Collaboration agreement: Plasticell and NUI
Plasticell (UK; www.plasticell.com) signed a collaboration agreement with the National University of Ireland, Galway (NUI Galway) focused on methods of eradicating cancer stem cells. Plasticell’s proprietary Combinatorial Cell Cultureâ„¢ (CombiCult®) platform will be used to discover combinations of drugs, growth factors and Plasticell and National University of Ireland initiate cancer stem cell collaboration chemotherapeutic agents that are capable of eradicating CSCs in leukaemia.
Collaboration and licensing agreement: Regeneron and Adicet
Regeneron Pharmaceuticals (NY, USA; www.regeneron.com) and Adicet Bio (CA, USA; www.adicetbio.com) announced a collaboration and licensing agreement to develop next-generation engineered immune cell therapeutics. The companies plan to engineer immune cells with fully human chimeric antigen receptors (CARs) and T-cell receptors (TCRs) directed to disease-specific cell surface antigens in order to enable the precise engagement and killing of tumor cells. The collaboration is intended to generate multiple clinical product candidates for various hematological and solid tumor cancers. Under the terms of the agreement, Regeneron and Adicet will collaborate to identify and validate appropriate targets and work together to develop a pipeline of engineered immune cell therapeutics for the selected targets. Adicet will receive a USD 25 million upfront payment, as well as research funding over the course of a five-year research term. Regeneron has the option to obtain development and commercial rights for a certain number of the product candidates, and Adicet has an option to participate in the development and commercialization on these potential products or is entitled to royalty payments by Regeneron.
Joint venture agreement: Sorrento and CHA
Sorrento Therapeutics (CA, USA; www.sorrentotherapeutics.com) has entered into a binding term sheet to create a joint venture (“JV”) with CHA Biotech (Korea; www.chabio.com) to develop and commercialize proprietary Chimeric Antigen Receptor (CAR) modified cellular therapies based on CBT’s Activated Killer Cell (“AKC”) technology and five of Sorrento’s CARs for all disease conditions, including oncology and infectious diseases. The JV covers products on a global basis with the exception of the Greater Chinese market. In addition, Sorrento will obtain an exclusive license to develop and commercialize CBT’s novel investigator-initiated trial stage AKC technology in major territories, including the United States, Europe, and with a co-exclusive license in China. The AKC technology utilizes proprietary methodologies for the generation of highly activated, patient-derived Natural Killer (NK) Cells from 60 ml of peripheral blood. The expansion and activation process takes less than 3 weeks and yields sufficient cells for several treatment cycles.
Licensing agreement: Cell Medica and UCL
Cell Medica (UK; www.cellmedica.co.uk) has signed a research deal with University College London (UK; www.ucl.ac.uk) for access to its novel T cell receptor technology, in a bid to generate leading-edge modified TCR products for the treatment of cancer. The deal gives the company an exclusive worldwide option and license agreement for these technologies, as well as TCR gene sequences for the development and commercialization of specific products. Under the collaboration, UCL will conduct the preclinical and early clinical research under the guidance of a Joint Steering Committee, and Cell Medica will support the product development work with its experience in manufacturing clinical-grade cell therapies and establishing robust production processes suitable for industrial scale-up. When successful first-in-man studies are complete, the products will transfer to Cell Medica for later-stage clinical development and commercialization. Cell Medica has paid an undisclosed up-front fee and will make additional payments to exercise its exclusive option to license future products, and UCL is eligible to receive further payments related to clinical, regulatory and sales milestones, as well as single digit royalties.
Licensing agreement: Celyad and ONO
Celyad (Belgium, www.celyad.com) announced an exclusive license agreement with ONO Pharmaceutical (Japan, www.ono.co.jp/eng/index.html), for the development and commercialization of Celyad’s allogeneic NKR-2 T-cell immunotherapy in Japan, Korea and Taiwan. Celyad will receive an upfront payment of USD 12.5 million (JPY 1.25 B) and is eligible of up to USD 299 million (JPY 30.075 B) in development and commercial milestones. Celyad will also receive double digit royalties based on net sales of the licensed product in ONO’s territories. Under the terms of the agreement, Celyad will continue developing its allogeneic NKR-2 T-cell immunotherapy in the EU and US territories, and ONO will be exclusive licensee for development and commercialization of NKR-2 T cell product in Japan, Korea and Taiwan.
Licensing agreement: Juno, Eureka and MSK
Juno Therapeutics (WA, USA; www.junotherapeutics.com) has entered into an exclusive license agreement with Memorial Sloan Kettering Cancer Center (MSKCC; NY, USA; www.mskcc.org) and Eureka Therapeutics (CA, USA; www.eurekinc.com) for a novel, fully-human binding domain targeting B-cell maturation antigen (BCMA), along with binding domains against two additional undisclosed multiple myeloma targets to be used for the potential development and commercialization of chimeric antigen receptor (CAR) cell therapies for patients with multiple myeloma. The binding domains were developed under a collaboration agreement between Eureka Therapeutics and MSKCC. The parties expect the BCMA CAR to enter human testing as early as 2017.
Licensing agreement: Kite and UCLA
Kite Pharma (CA, USA; http://kitepharma.com) has entered into an exclusive, worldwide license agreement with The Regents of the University of California, on behalf of the University of California, Los Angeles (UCLA; http://ucls.edu), for use of the technology artificial thymic organoid (ATO). ATO is a cell culture system replicating the human thymic environment to support efficient ex vivo differentiation of T cells from primary and reprogrammed pluripotent stem cells.
Licensing agreement: TiGenix and Takeda
TiGenix (Belgium; www.tigenix.com) has sold to Takeda (Japan, http://betterhealth.takeda.com/) ex-U.S. rights to its stem cell treatment Cx601 for complex perianal fistulas in patients with Crohn’s disease–an up-to-USD435 (€390) million-plus deal reflecting what the Belgian biotech called a shift of resources toward its allogeneic stem cell platforms. As part of that shift, TiGenix said, it was withdrawing its European marketing authorization application for ChondroCelect®, an autologous cell therapy product for cartilage repair of the knee. Instead, TiGenix will refocus on its upcoming Phase III U.S. trial for Cx601, as well as its other clinical stage assets. Cx601 is a suspension of allogeneic adipose-derived stem cells (eASC) injected intralesionally. In March, TiGenix submitted a marketing authorization application to the European Medicines Agency for Cx601, based on positive 24-week results of the ADMIRECD Phase III clinical trial. More recently, TiGenix announced top-line 52-week data that it said confirmed the efficacy and safety of a single injection of Cx601. Following marketing authorization in Europe, Takeda will become the marketing authorization holder and will oversee all commercialization and regulatory activities. Takeda will also be responsible for additional development activities of Cx601 for the indication of complex perianal fistulas in Crohn’s disease. TiGenix will retain the rights to develop Cx601 in new indications–and will continue to hold US rights to the allogenic stem cell therapy.
Launching new projects, products & services
Antibe
Antibe Therapeutics (ON, Canada; www.antibethera.com) announced that its subsidiary Citagenix (QC, Canada; www.citagenix.com) has launched Neomem® FlexPlus, a high-performance single layer collagen membrane derived from porcine peritoneum for regenerative procedures in oral surgery. Suture pull-out testing has shown this membrane to be biomechanically strong yet conformability evaluations demonstrate the ability to adapt to the contour of the surgical site.
Takara Bio
Takara Bio (Japan; www.takara-bio.com) has announced expansion of tools for drug discovery and disease modelling, and in particular the launch of fully differentiated, Cellartis® human induced pluripotent stem (hiPS) beta cells for research use. These cells could provide physiologically relevant responses beneficial for modeling diabetes and beta-cell function, advancing research in personalized and regenerative medicine. Cellartis hiPS beta cells are developed and manufactured by Takara Bio Europe AB, a wholly-owned subsidiary of Takara Bio Inc., and will be available worldwide through Takara Bio affiliates and distributors.
Achievements
Biostage
Biostage (MA, USA; www.biostage.com) has received the National Instruments (“NI”) Engineering Impact Award for Regenerating and Restoring Organ Function Damaged by Disease or Trauma (http://sine.ni.com/cs/app/doc/p/id/cs-17124) for its groundbreaking Cellframeâ„¢ Technology. The Company was also the recipient of the NI 2016 Community’s Choice Humanitarian Award. Proprietary Cellframe technology is designed to harness the full potential of the in vivo microenvironment to achieve tissue regeneration and restore organ function. It employs a multistep process in which the patient’s own stem cells are taken from a simple adipose/fat tissue biopsy, expanded and banked, and then seeded onto a proprietary scaffold that mimics the natural dimensions of the organ being regenerated. After several days in a rotating bioreactor, the biocompatible scaffold containing the stem cells is ready to be implanted. Preclinical studies suggest that the organ implant signals the stem cell niche in the surrounding native tissue to guide the regeneration of a biological structure. This technology is based on the concept of in situ tissue regeneration using the body’s own biologic resources and reparative capability in combination with tissue-specific biomaterials implanted at the sites of disease or injury.
Cytori
Cytori Therapeutics (CA, USA; www.cytori.com) has received the Frost & Sullivan 2016 Technology Innovation Award in recognition of advancements made in the field of regenerative medicine for over a decade.