VEDANTA

(VE303, VE202, VE416, VE800)

TRIAL PHASE
Product Candidate
PureTech Ownership
Initial Indications
Preclinical
Phase 1
Phase 2
Phase 3
VE303
53.3% (Vedanta)
rCDI

Initial Indication(s):

rCDI

Patient Population:

100-120K per year

Collaborators:

keioU

Key Differentiation:

Developing a new category for immune-mediated diseases based on a rationally-defined consortia of human microbiome-derived bacteria

VE202
53.3% (Vedanta)
IBD

Initial Indication(s):

IBD

Patient Population:

~3M

Collaborators:

keioUjanssen

Key Differentiation:

Developing a new category for immune-mediated diseases based on a rationally-defined consortia of human microbiome-derived bacteria

VE416
53.3% (Vedanta)
Food Allergy

Initial Indication(s):

Food allergy

Patient Population:

~2.5M

Collaborators:

keioU

Key Differentiation:

Developing a new category for immune-mediated diseases based on a rationally-defined consortia of human microbiome-derived bacteria

VE800
53.3% (Vedanta)
Solid Tumors

Initial Indication(s):

Solid tumors
including MSS CRC,
gastric, and melanoma

Patient Population:

~46K (MSS CRC)
~11K (Gastric)
~9K (Melanoma)

Collaborators:

keioUbristol meyers

Key Differentiation:

Developing a new category for immune-mediated diseases based on a rationally-defined consortia of human microbiome-derived bacteria



VE303
rCDI
53.3% (Vedanta)
2

Initial Indication(s):

rCDI

Patient Population:

100-120K per year

Collaborators:

keioU

Key Differentiation:

Developing a new category for immune-mediated diseases based on a rationally-defined consortia of human microbiome-derived bacteria

VE202
IBD
53.3% (Vedanta)
1

Initial Indication(s):

IBD

Patient Population:

~3M

Collaborators:

keioUjanssen

Key Differentiation:

Developing a new category for immune-mediated diseases based on a rationally-defined consortia of human microbiome-derived bacteria

VE416
Food Allergy
53.3% (Vedanta)
1

Initial Indication(s):

Food allergy

Patient Population:

~2.5M

Collaborators:

keioU

Key Differentiation:

Developing a new category for immune-mediated diseases based on a rationally-defined consortia of human microbiome-derived bacteria

VE800
Solid Tumors
53.3% (Vedanta)
1

Initial Indication(s):

Solid tumors
including MSS CRC,
gastric, and melanoma

Patient Population:

~46K (MSS CRC)
~11K (Gastric)
~9K (Melanoma)

Collaborators:

keioUbristol meyers

Key Differentiation:

Developing a new category for immune-mediated diseases based on a rationally-defined consortia of human microbiome-derived bacteria



Modulating the immune system via the gut microbiome

Founded by PureTech as part of its microbiome initiative, PureTech’s Vedanta Biosciences is developing a new category of therapies for immune-mediated diseases based on a rationally-defined consortia of human microbiome-derived bacteria. Vedanta Biosciences is a leader in the microbiome field with capabilities and deep expertise to discover, develop, and manufacture live bacteria drugs. These include what is believed to be a leading IP position with the largest collection of human microbiome-associated bacterial strains, a suite of proprietary assays to select pharmacologically potent strains, vast proprietary datasets from human interventional studies, and facilities for current good manufacturing practice, or cGMP-compliant manufacturing of rationally-defined bacterial consortia in powder form.

Vedanta Biosciences’ pioneering work, in collaboration with its scientific co-founders, has led to the identification of human commensal bacteria that induce a range of immune responses – including induction of regulatory T cells, CD8+ T cells, and Th17 cells, among others. These advances have been published in leading peer-reviewed journals, including Science (multiple), Nature (multiple), Cell, and Nature Immunology. Vedanta Biosciences has harnessed these biological insights and its capabilities to generate a clinical pipeline of programs in infectious disease, autoimmune disease, allergy, and immuno-oncology.

In 2017, we initiated a Phase 1a/1b clinical trial of VE303, our lead product candidate for the treatment of recurrent C. difficile infection. We also continued to expand our internal, state-of-the-art cGMP-compliant manufacturing capabilities, giving us a distinct competitive advantage in the microbiome field. A Phase 2 study of VE303 is on track to begin in the second half of 2018. Three additional product candidates are also expected to enter clinical development in 2018, with Phase 1 trial initiations planned in inflammatory bowel disease (IBD) and food allergy, and the submission of an investigational new drug application (IND) for an immuno-oncology product candidate.


  • Patient Need & Market Potential
    • Despite profound survival improvements in some patients, checkpoint inhibitors (PD-1/ PDL-1, CTLA-4) are only effective in 20-30 percent of patients. Common tumor types where checkpoint inhibitors are utilized include lung, bladder, skin, and renal cancers. Vedanta Biosciences’ immuno-oncology product candidate, VE800, is designed to act in combination with approved checkpoint inhibitors and potentially other immunotherapies to safely improve their efficacy.
    • Food allergies are a growing U.S. public health concern – they affect eight percent of children and have an estimated annual economic cost near $25 billion. Current treatment options primarily center around allergen avoidance. Desensitization regimens in development have limited efficacy, are risky, and require treatment for life and may not be cost effective. Vedanta Biosciences’ product candidate, VE416, is being developed to safely induce permanent tolerance to food allergens including peanut allergy.
    • Inflammatory bowel disease (IBD) is estimated to affect approximately three million people in the U.S., and other autoimmune diseases affect over 20 million people in the U.S. Many of the existing interventions are limited by toxicities and systemic immune suppression. Vedanta Biosciences’ collaborator, Janssen Biotech Inc., is advancing a product candidate, VE202, designed to modulate the activity of regulatory T cells and thereby potentially treat IBD.
    • The Center for Disease Control and Prevention (CDC) considers C. difficile infections one of the most urgent bacterial threats. C. difficile infections account for nearly 15,000 deaths each year in the U.S. alone and there are approximately 500,000 cases annually, of which 100,000 to 120,000 patients experience recurrence. Existing interventions include antibiotics such as vancomycin or metronidazole, which have the undesirable side effect of damaging the gut microbiome and leaving patients vulnerable to re-infection. An alternative intervention, fecal transplantation, is an experimental procedure which is exceedingly difficult to standardize and scale and is fraught with potential safety issues. Vedanta Biosciences’ lead, orally-administered live biotherapeutic product candidate, VE303, is designed to restore colonization resistance against gut pathogens, including C. difficile, following recurrence.
  • Our Approach to Solving the Problem
    • Unlike fecal transplants, which require use of donors and are untargeted, inherently variable procedures, Vedanta Biosciences’ approach is based on bacterial consortia therapeutics, which are defined drug compositions produced from clonally isolated bacteria that can trigger targeted immune responses. Unlike reductionistic approaches such as single strain probiotics, defined consortia can robustly shift the composition of the gut microbiota and provide colonization resistance against a range of intestinal infectious pathogens. These therapeutics can also stimulate a range of immune responses ranging from immunoregulatory responses, which hold potential in the treatment of autoimmune and allergic diseases, to immunopotentiating responses, which hold potential in cancer and vaccination.
    • Vedanta Biosciences’ collaborators have pioneered the fields of innate immunity, Th17, and regulatory T cell biology. These discoveries, which have formed the leading scientific foundation for Vedanta Biosciences, have been reported in seminal scientific papers and published in leading journals such as Science, Nature and Cell, demonstrating that the gut microbiome influences important processes related to the proper functioning of the immune system and resistance to infection.
    • Vedanta Biosciences’ novel product candidates are administered in a lyophilized powder in a capsule dosage form, designed to have specific effects on the immune system,including restoring the balance of the microbiome in the gut to treat immune and infectious diseases and immunopotentiating responses to treat cancer.
  • Intellectual Property
    • Vedanta Biosciences has broad intellectual property coverage worldwide, currently owning or having exclusive rights to one hundred and eight (108) patent applications and issued patents in sixteen (16) families of patent filings, including seven (7) patents that issued in 2018.
    • Vedanta Biosciences’ IP estate positions the company as a leader in the microbiome field.
    • Vedanta Biosciences’ IP portfolio includes foundational patents covering compositions and therapeutic uses of products containing microbiome bacteria belonging to Clostridium clusters IV and XIVa, which are among the most abundant colonizers of the human intestine and play an important role in human health, including regulating inflammatory responses and other immune responses.
    • The IP estate includes issued patents in the major pharmaceutical markets (U.S., Europe, and Japan). These patents provide coverage through at least 2031, with priority filing dates as early as 2010.
  • Team
    • Dr. Bernat Olle (previously PureTech) serves as Chief Executive Officer, Dr. Bruce Roberts (previously Sanofi-Genzyme) serves as Chief Scientific Officer, and Mr. Dan Couto (previously ContraFect Corp) serves as Chief Technical Officer and head of manufacturing.
    • Scientific co-founders and advisory board members include some of the world’s leading immunologists, including Dr. Ruslan Medzhitov (Yale and Howard Hughes Medical Institute (HHMI)), Dr. B Brett Finlay (University of British Columbia and HHMI), Dr. Kenya Honda (inventor of Vedanta Biosciences’ IBD product candidate; Keio University and RIKEN), Dr. Dan Littman (NYU School of Medicine, Howard Hughes Medical Institute; member of the Board of Pfizer), Dr. Alexander Rudensky (Sloan Kettering and HHMI), and Dr. Jeremiah Faith (Mount Sinai School of Medicine).
  • Milestones Achieved
    • VE303, Vedanta’s product candidate for the treatment of rCDI, is being studied in a Phase 2 clinical trial in patients at high risk of rCDI. The trial was initiated in December 2018, and dose selection was based on the results from the Phase 1a/1b clinical trial in healthy volunteers, which showed that VE303 treatment resulted in rapid, durable, dose-dependent colonization and accelerated gut microbiota restoration after antibiotics.
    • VE202, Vedanta’s product candidate in IBD, is being advanced by Vedanta’s collaborator, Janssen. Janssen initiated a Phase 1 clinical trial in healthy volunteers of VE202 in November 2018, with PK/PD results anticipated in the second half of 2019. In conjunction with the initiation of this study, Vedanta has received $12 million from Janssen in milestone payments as part of its ongoing collaboration.
    • VE416, Vedanta’s product candidate in food allergy, is being evaluated in a Phase 1b/2 investigator sponsors trial at MassGeneral Hospital for Children for patients 12 years of age or older with a history of peanut allergy. The first patient was enrolled in July 2019 and will explore VE416 both as a monotherapy and in combination with an oral peanut immunotherapy over the course of several months. 

       

  • Collaborations
    • In January 2019, important research that underlies Vedanta’s proprietary oral immuno-oncology product candidate, VE800, was published in one of the top scientific journals, Nature. The research revealed a new mechanism by which human microbiota induce an important immune cell that is key to the body’s ability to generate anti-tumor immunity.
    • Additional data on Vedanta Biosciences’ microbiome technologies has been featured in high impact academic journals such as Nature, Science, and Cell.
    • Vedanta Biosciences announced a clinical collaboration with Bristol-Myers Squibb to evaluate VE800 in combination with Bristol-Myers Squibb’s programmed death-1 (PD-1) immune checkpoint inhibitor Opdivo (nivolumab) in patients with advanced or metastatic cancers. Bristol-Myers Squibb is a strategic equity investor in Vedanta Biosciences. Under the terms of the agreement, Vedanta Biosciences will maintain control of its VE800 program, including global R&D and commercial rights.
    • As part of the collaboration with Janssen Biotech, Vedanta Biosciences has received $12 million from Janssen in milestone payments as part of its ongoing collaboration.
    • Vedanta Biosciences received funding from the Crohn’s & Colitis Foundation to advance its new microbiome-derived therapeutic program for the treatment and potential interception of IBD.
    • Vedanta Biosciences has exclusively licensed key intellectual property from Keio University to develop and commercialize microbiome-derived cancer immunotherapies based on live biotherapeutics.
  • Expected Milestones and Timing
    • PK/PD results are anticipated in the second half of 2019 from the Phase 1 healthy subject study of VE202.
    • Topline data from the Phase 1b/2 clinical trial of VE416 in food allergy are expected in 2020.
    • VE800, Vedanta’s product immuno-oncology product candidate, will be evaluated in Phase 1b/2 trial with Bristol-Myers Squibb’s checkpoint inhibitor OPDIVO (nivolumab) in patients with selected types of advanced or metastatic cancer. The trial is expected to begin in 2019, with topline results in 2021.

Unlike fecal transplants or single strain approaches to microbiome modulation, Vedanta Biosciences uses pure, clonal cell banks to produced defined collections, or consortia, of bacterial strains designed to effect durable therapeutic changes in a patient’s gut microbiota. This bypasses the need to rely on direct sourcing of fecal donor material of inconsistent composition.


Our In-House, State-of-the-Art GMP Manufacturing Plant



Pipeline