At PureTech, we are creating new categories of medicine by leveraging the science of the Brain-Immune-Gut (BIG) axis

Our Publications

PureTech is discovering and developing highly differentiated medicines for dysfunctions of the Brain-Immune-Gut (BIG) axis.

The goal at PureTech is to identify, invent, develop, and commercialize innovative new categories of therapeutics that are derived from an advanced understanding of the BIG axis to address significant unmet medical needs.

 

CASE STUDIES

DISCOVERY REINVENTED

New mechanism for treating psychosis and cognitive impairments (KarXT)

Open Case Study

The Challenge

Psychosis, negative symptoms, and cognitive impairments in schizophrenia, Alzheimer’s disease and other mental illnesses remain poorly treated

There are approximately 2.7M living with schizophrenia and 5.7M living with Alzheimer's in the US

Existing Approaches

Antipsychotics are the mainstay therapy targeting dopamine pathways, however with no new treatment mechanism in 60 years, the prognosis for patients remain poor

Cuurent antipsychotics only address psychosis (positive symptoms) and are associated with serious side effects such as sedation, extrapyramidal side effects such as motor rigidity, tremors and slurred speech, and weight gain 

The Big Idea

A class of medicines (M1/M4 agonists) that showed enormous potential in clinical studies was never developed due to tolerability issues.

We asked “What if you you could selectively target M1/M4 receptors in the central nervous system without affecting the peripheral tissues where most side effects occur?”

The Result

Karuna is developing a potentially first-in-class oral modulator of muscarinic receptors

KarXT combines xanomeline, a novel muscarinic receptor agonist, with trospium chloride, a muscarinic receptor antagonist that acts peripherally and does not measurably cross the blood-brain barrier

Selective mTORC1 Inhibitors (resTORbio)

Open Case Study

The Challenge

Many of the afflictions of a rapidly aging population – from declines in cognition, to immunosenescence and cancer – remain poorly addressed. One such indication is respiratory tract infection, or RTI, a leading cause of mortality in elderly people

Existing Approaches

Limited therapeutic options exist today to prevent and treat viral respiratory tract infections.

Some vaccines protect against specific viruses; however there is no available treatment for most RTIs, a leading cause of mortality in high risk, elderly populations.

The Big Idea

Can we develop an anti-viral that works by boosting the immune system?

The Result

A new approach in targeting the mTOR pathway by selectively targeting TORC1, linked to increased lifespan and other beneficial effects. We are focusing initially on the reduction of RTIs in elderly individuals at increased risk of RTI related morbidity and mortality. Our selective mTORC1 inhibition program has potential in other aging related indications including cognition, cardiovascular and cancer.

Rationally defined, immune modulating non-pathogenic human microbes (VE202)

Open Case Study

The Challenge

The human microbiome is increasingly implicated in various immune-mediated disease states

Vedanta has discovered specific bacteria that induce T regs (which form the basis for Vedanta's IBD and food allergy candidates) and CD8+ cells (which form the basis for Vedanta's IO candidate)

Existing Approaches

Many existing IBD interventions are limited by toxicities and systemic immune suppression

Food Allergy treatment today primarily centers around allergen avoidance, and new immunotherapies focused on desensitization may note prove cost-effective relative to this approach

Checkpoint Inhibitors are only effective in 20-30% of patients

C. difficile is typically treated using antibiotics (damage the microbiome and leave patients vulnerable to re-infection) or FMT (uncharacterized safety issues)

The Big Idea

What if we could treat immune and infectious disease by mimicking the ways in which the gut microbiota maintains a healthy immune system in humans?

The Result

Vedanta is developing a potentially new category of therapies based on a rationally-defined consortia of human microbiome-derived bacteria

Defined consortia have potential to shift microbiota, stimulate immune responses, and provide colonization resistance against infectious pathogens

Clinical results for VE303, VE800, and VE416 are anticipated in 2020

Foundational patents issued in key territories 

Targeting immunologically silent tumors with first-in-class mAbs (LYT-200)

Open Case Study

The Challenge

Low five year survival rate for many aggressive solid tumors, including metastatic pancreatic and colorectal cancer, and metastatic cholangiocarcinoma

Many aggressive solid tumors do not benefit from approved immuno-oncology agents

Low response rate to immunotherapy

Existing Approaches

"Cold" tumors lack cancer-killing immune cells

Finding targets that suppress multiple immunosuppressive pathways simultaneously has proven challenging, and combination therapies are limited by toxicity

The Big Idea

Can we target the immunosuppressive cells that solid, malignant tumors establish to ward off the body’s natural defenses?

The Result

Developing first-in-class fully human mAbs targeting immunologically silent tumors with galectin-9 and immunosuppressive γδ T cells that mediate immunosuppression through multiple mechanisms and has the potential for single efficacy as well as combinations.

 

Relationships with 8 major pharma companies or their investment arms

 

 

Amgen Johnson & Johnson Lilly Merck Boehringer Ingelheim Bristol-Myers Squibb Teva Roche