Memorandum

JDRF funds Sernova’s Cell Pouch and Defymed’s MAILPAN beta cell encapsulation technologies – July 14, 2016

Executive Highlights

  • Sernova receives ~$2.5 million for human clinical trials of Cell Pouch macroencapsulation beta cell replacement system.
  • Defymed receives $500,000 for preclinical trials of the MAILPAN “bioartificial pancreas” beta cell encapsulation system.
  • Funding commits JDRF to having multiple shots on goal in this encapsulation arena.

Beta cell encapsulation companies Sernova and Defymed both recently announced research funding partnerships with the JDRF to advance their respective encapsulation therapies through clinical and preclinical trials for type 1 diabetes. Sernova’s phase 1/2 Cell Pouch received $2.45 million from the JDRF to support an in-human safety and efficacy clinical trial at “a major transplantation center” in the US. Sernova’s Cell Pouch System involves a thin, implantable, biocompatible device – approximately the size of a business card – that provides an vascularized environment for enclosed therapeutic cells including donor islet and stem cell-derived technologies. Sernova is also currently developing microencapsulation technologies to locally protect cells from the immune system within the vascularized chambers. Sernova previously presented encouraging interim results from the phase 1/2 clinical trial primarily evaluating safety for the candidate at JPM 2016, demonstrating that the device was safe and the encapsulated cells were able to produce insulin, glucagon, and somatostatin with no evidence of an inflammatory response or an immune attack on the cells. Sernova, a member of the JDRF Encapsulation Consortium, confirmed to us that the JDRF funding will support a separate US-based phase 1/2 safety and efficacy trial with a new advanced design that is expected to initiate in the second half of 2016. We are trying to better understand what happened with the trial originally reported on at JPM.

Defymed’s preclinical MAILPAN “bioartificial pancreas” received up to $500,000 in funding to advance preclinical studies for the candidate. Few details are available for Defymed’s MAILPAN encapsulation system, beyond that it takes a macroencapsulation approach involving a semi-permeable membrane pouch containing many insulin-secreting cells. Defymed and MAILPAN were developed through a consortium of partners in academia, clinical/public health research, and industry from Belgium, France, and the UK, including Oxford’s Dr. Paul Johnson. The company announcement for the JDRF partnership noted that the funding will also help support its entry into the US target market and that Defymed would join JDRF’s Encapsulation Consortium.

  • MAILPAN appears most directly comparable to ViaCyte’s phase 1/2 VC-01 cell replacement therapy that similarly encapsulates many progenitor islet cells at once. ViaCyte is currently leading the pack in terms of beta cell replacement or encapsulation therapies – the company recently shared an update at Levine-Riggs 2016 that 14 patients have been implanted with the system thus far. It must be more by now but we’re not sure of the exact number. Our guess is 20.
  • In contrast to the ViaCyte and Defymed’s approaches, Sernova’s approach enables cells within the device chambers to be intimately associated with a vascularized tissue matrix and will locally protect cells within the device through microencapsulation within the vascularized tissue matrix. While participants in the upcoming JDRF-funded study will receive anti-rejection medications, Sernova is developing microencapsulation technologies to protect individual cells, including unlimited sources such as stem cell-derived insulin producing cells, from immune attack within the Cell Pouch vascularized matrix. Notably, the biocompatible Cell Pouch is fully scalable and following implantation of the porous chambered device, developed vascularized tissue chambers for cell survival and function. We expect that this could provide an advantage over ViaCyte’s approach – that said, we hadn’t realized this part was still in development. A Harvard/MIT team led by Harvard’s Dr. Doug Melton has also published preclinical data for a microencapsulated beta cell system that uses fully-differentiated, stem-cell derived cells. We’re excited to see so much interest and investment in this area and are curious to see how the nuances of each group’s approach might affect the efficacy and durability of the encapsulated cells. There’s been a great deal of work for a great deal of time on this technology.
    • One of the biggest challenges toward widespread use in type 1 diabetes facing Sernova is the need to secure an unlimited cell source for the Cell Pouch technology. Currently, the technology uses donor islets, which are in limited supply, and is aimed to improve the quality of life for patients with severe hypoglycemia unawareness. The company is addressing the future widespread use of its products in type 1 diabetes through recently securing a worldwide exclusive license to a glucose-responsive stem cell derived technology and has been approached by multiple companies with stem cell and xenogeneic options for combination with Sernova’s device technologies.
  • These funding announcement further underscore the JDRF’s vested interest in supporting beta cell encapsulation research. Encapsulation is one of the JDRF’s six major research areas. The organization previously funded ViaCyte and also provided support for Dr. Melton’s encapsulation approach. We’re moved to see the JDRF’s continued investment in this area, particularly at a time in which there is so much demand for funds; as usual, we applaud the high quality of its scientific minds, its commitment to helping small companies on a variety of fronts, and its commitment to bringing potential type 1 diabetes cure therapies to market.

 

-- by Helen Gao and Kelly Close