Memorandum
- The table below includes an overview of the competitive landscape for type 1 diabetes cure or prevention therapies. It includes all the companies we are aware of with projects in development for beta cell replacement, immune therapy, or other approaches with the aim of preventing, delaying, slowing, or curing type 1 diabetes. We will continuously update the table as timelines change. Our glucose-responsive insulin and automated insulin delivery competitive landscapes are hosted on separate pages.
Beta Cell Encapsulation
|
Company |
Product |
Type |
Status |
Timeline |
|
ViaCyte |
PEC-Encap/VC-01 |
Macroencapsulation, no direct vascularization
|
Phase 1/2 |
Data from new Gore device expected in coming months from JPM 2020; Phase 1/2 trial initiated in 2014; Clinical development halted due to issues with FBR, but expected to resume in 1H19 |
|
Sernova |
Cell Pouch System (CPS) |
Microencapsulation within vascularized tissue matrix |
Phase 1/2 |
Positive preliminary phase 1/2 results released July 2019; Phase 1/2 trial recruiting with expected primary completion July 2019; IND application cleared by FDA in December 2017; Positive interim phase 1/2 results reported at JPM 2016 from a Canadian trial, but the trial has since been terminated; Announced collaboration with CTI Clinical Trial and Consulting Services in November 2016 |
|
ViaCyte |
PEC-Direct/VC-02 |
Direct vascularization, requires immunosuppressive therapy |
Phase 1/2 |
“On track” to have a Regenerative Medicine Advanced Therapy (RMAT) designation from FDA as of JPM 2020; European clinical program initiated January 2019; Phase 1/2 trial initiated August 2017, expected to complete December 2020; Phase 3 was expected to initiate mid-2018 with approval projected for 2020 or beyond; Proof-of-efficacy now expected mid-2019 |
|
Defymed/Semma Therapeutics |
MAILPAN |
Macroencapsulation, no direct vascularization |
Preclinical |
Semma acquired by Vertex for $950 million in September 2019; Positive preclinical data released in July 2019; $114M Series B financing in December 2017; Received funding for preclinical trials from JDRF in July 2016; Partnered with Semma Therapeutics for an unlimited stem cell-derived beta cell source in December 2016 |
|
JDRF/UCSF/Stanford |
Undisclosed |
Cell Replacement Cures Without Immunosuppresion |
Preclinical |
Collaboration announced |
|
Lilly/Sigilon |
Undisclosed |
Macroencapsulation using FBR-resistant Afibromer encapsulation device |
Preclinical |
Collaboration announced April 2018 |
|
Novo Nordisk/UCSF/Cornell |
Undisclosed |
Undisclosed |
Preclinical |
Collaboration announced May 2018 |
|
Dr. Tejal Desai lab at UCSF |
nanotemplating |
Next-generation encapsulation approach |
Preclinical |
Presented at the 2016 JDRF Bay Area Annual Meeting and Research Update |
|
Goethe University/DRIVE Consortium |
LSFM4Life |
“Organoid” (insulin-producing cells derived from adult stem cells and embedded in a three-dimensional matrix)
|
Preclinical |
Project launched in April 2016 with goal of entering clinical trials by end of 2019 |
|
DRIVE Consortium/Boston Scientific |
Undisclosed |
Encapsulation device containing gel-embedded clusters of islet cells; capsule releases immunosuppressant |
Preclinical |
Announced in June 2015 |
|
Dr. Shuvo Roy lab at UCSF |
Bioartificial pancreas |
Islets contained in semi-permeable silicon ultrafiltration capsule with active blood flow; Graft rather than subcutaneous implant |
Preclinical |
Presented at the 2016 JDRF Bay Area Annual Meeting and Research Update |
|
ViaCyte/CRISPR Therapeutics |
PEC-QT |
Islets are derived from genetically engineered stem cells that are immune-evasive |
Preclinical |
Announced at JPM 2018; Received $1.4 million from CIRM for development of immune-evasive stem cells in December 2017; Subsequently partnered with CRISPR Therapeutics |
Beta Cell Replacement Sources
|
Company |
Product |
Status |
Timeline |
|
Semma Therapeutics |
Stem cell-derived insulin producing cells |
Preclinical |
Cell therapy to move into clinic by late 2020/early 2021, as of JPM 2020; Acquired by Vertex Pharmaceuticals for $950 million in September 2019; Partnership with AZ, Novartis, and Defymed; Published in Nature in 2019 detailing improved beta cell generation/purification process; Semma Therapeutics founded in April 2015 to translate Dr. Doug Melton’s beta cell generation procedure into therapy |
|
Sanofi/Evotec |
Functional stem cell-derived human beta cells for replacement therapies and for identification of active therapeutic targets |
Preclinical |
Possibly discontinued following Sanofi's decision to discontinue R&D in diabetes; Management expressed enthusiasm in January 2019 (interview); Partnership announced in August 2015 |
|
AZ MedImmune/Evotec |
Biologic beta cell regeneration |
Preclinical |
Partnership announced in December 2010 |
|
ViaCyte |
Stem cell-derived insulin producing cells |
Preclinical |
In 2006, ViaCyte published the first study demonstrating successful in vitro differentiation of human embroyonic stem cells into pancreatic tissue; In 2014 BetaLogics (now part of ViaCyte) published the first paper on using in vitro stem-cell derived beta cells to reverse diabetes in mice |
|
Frequency |
Progenitor Cell Activation (PCA) platform, which induces stem cell-like properties in somatic cells |
Preclinical |
Received JDRF funding in February 2017 to optimize the PCA platform for type 1 diabetes |
| UCSF Hebrock Lab | Stem cell-derived enriched beta clusters (more advanced insulin-producing cells) | Preclinical | Published methodology for growing enriched beta clusters (eBCs – more mature, insulin-producing cells) from stem cells |
Immune Therapies
|
Organization |
Product |
Type |
Status |
Timeline |
| Provention | teplizumab | Anti-CD3 | Phase 3 | FDA Breakthrough Therapy Designation in August 2019; First therapy shown to delay type 1 diagnosis (ADA 2019); Phase 3 PROTECT study underway as of April 2019, following aquisition of candidate from MacroGeneics |
|
Novo Nordisk |
NN9828 |
Anti-IL21/GLP-1 agonist (liraglutide) combination |
Phase 2 |
Positive phase 2 topline results shared in 2Q19; Phase 2 trial expected to complete March 2019; FDA orphan drug designation in January 2017 |
|
Immune Tolerance Network |
alefacept |
Anti-CD2 |
Phase 2 |
Two year T1DAL phase 2 trial results presented at ADA 2015 |
|
TrialNet |
ATG/GCSF |
Combination approach |
Phase 2 |
Phase 2 results presented at ADA 2018; ClinicalTrials.gov lists full phase 2 completion in October 2018 |
|
TrialNet |
CTLA4-lg (abatacept) |
Immunosuppressant |
Phase 2 |
Phase 2 trial expected to complete in November 2020, pushed back from February 2018; Results expected in 2020 |
|
Caladrius Biosciences |
CLBS03 |
Treg |
Phase 2 |
Topline phase 2a T-Rex (pediatric) results released; Phase 1 results presented at ADA 2014; Phase 2 clinical studies launched in March 2016, full completion is expected in March 2020 |
|
TrialNet |
teplizumab |
Anti-CD3 |
Phase 2 |
Positive phase 2 results presented at ADA 2019; Phase 2 trial expected to complete in January 2019, moved up from January 2022 |
|
GeNeuro |
GNbAC1 |
Monoclonal antibody that neutralizes pathogenic protein MSRV-Env |
Phase 2a |
Phase 2a trial initiated April 2017 and expected to complete March 2019 |
|
ActoBio |
AG019 |
Engineered Lactococcus Lactis bacteria that releases proinsulin + IL-10 into gut |
Phase 1b/2a |
Phase 1b/2a in combination with teplizumab expected to complete in June 2020 |
|
Imcyse |
Imotopes |
Auto-antigen derived epitopes + thioreductase motif to reprogram autoimmune T-cells |
Phase 1b |
Positive poster presented at EASD 2019; Results expected 3Q19; Moving into phase 2 on back of Series B financing |
|
UCSF (Dr. Jeffrey Bluestone) |
Tregs + IL2 |
Combination of CD4, CD127, and CD25 Tregs with IL2 |
Phase 1 |
Phase 1 TILT study expected to complete July 2021 |
| Janssen | golimumab (Simponi) | TNF-alpha | Phase 1 | Phase 1 study in youth (n=30) with ≥2 antibodies expected to complete July 2021 |
| Pfizer | nanoformulations (tolerance molecule + antigen) | Immune tolerance therapy | Preclinical | Licensed from AnTolRx in March 2019 |
|
Provention Bio |
Enteroviral vaccine |
Vaccine targeting Coxsackievirus B infection |
Preclinical |
Announced July 2017; Funding from J&J Innovation and JDRF T1D Fund |
|
REGiMMUNE/JDRF/Pfizer |
RGI-3100 |
Type 1 diabetes-specific antigen, to be used with reVax immunomodulatory platform to promote Tregs |
Preclinical |
Collaboration with JDRF and Pfizer announced in May 2015 |
|
Mesoblast |
Undisclosed |
Mesenchymal precursor cell (MPC) treatment |
Preclinical |
As of January 2016, Mesoblast was investigating a type 1 diabetes application for its MPC treatment |
|
Calibr |
Undisclosed |
Voltage-gated potassium channel (Kv1.3) inhibitor |
Preclinical |
Discussed at 2016 JDRF Mission Summit |
|
Selecta |
SVP-Rapamycin |
Synthetic Vaccine Particle (SVP) nanotechnology for immunosuppression |
Preclinical |
Series E financing in September 2015 |
|
Selecta |
SVP-insulin |
Synthetic Vaccine Particle (SVP) nanotechnology for immunosuppression |
Preclinical |
Series E financing in September 2015 |
|
Kanyos Bio |
Undisclosed |
Immune tolerance therapy |
Preclinical |
Kanyos Bio created by partnership between Astellas Pharma and Anokion in July 2015 |
|
GSK |
otelixizumab |
Anti-CD3 |
Discontinued |
No longer listed in GSK pipeline; Negative phase 3 results in 2011; Revived phase 2 program completed September 2018 ahead of schedule |
Other
|
Organization |
Name |
Type |
Status |
Timeline |
|
TrialNet |
Oral insulin |
Oral insulin |
Phase 3 |
Phase 3 trial fully enrolled; Results presented at ADA 2017; Phase 2 dose-ranging trial expected to complete in December 2017 |
|
Global Platform for the Prevention of Autoimmune Diabetes |
Oral insulin |
Oral insulin (high-dose in infants) |
Phase 2b |
Primary Oral Insulin Trial (POInT) currently screening in Europe |
|
Kamada |
Alpha-1 Antitrypsin (AAT)
|
AAT protein |
Phase 2 |
Topline phase 2 data released November 2017; Early signs of efficacy in people age 12-18 with new-onset type 1 diabetes |
|
Calibr |
MST1 |
Beta cell proliferator |
Preclinical
|
Discussed at 2016 JDRF Mission Summit |
|
Massachusetts General Hospital (Dr. Denise Faustman) |
Bacillus Calmette- Guérin (BCG)
|
Repeat Vaccination |
Phase 2 |
Phase 1 results released in July 2018 controversial; primary completion expected in July 2020 with full completion expected in July 2023 |
-- by Abigail Dove, Helen Gao, Zhazira Irgebay, Sahaj Shah, Rhea Teng, and Kelly Close