At PureTech, we are creating new categories of therapeutics derived from our deep understanding of the Brain-Immune-Gut (BIG) axis
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Wholly Owned Pipeline
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New mechanism for treating psychosis and cognitive impairments (KarXT)
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)
The Challenge
Existing Approaches
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)
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)
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.
1 The FDA and corresponding regulatory authorities will ultimately review our clinical results and determine whether our wholly-owned therapeutic candidates are safe and effective. No regulatory agency has made any such determination that our wholly-owned therapeutic candidates are safe or effective for use by the general public for any indication.
2 Long COVID is a term being used to describe the emerging and persistent complications following the resolution of COVID-19 infection, also known as post-acute COVID-19 syndrome (PACS).
3 Funding figure includes private equity financings, loans and promissory notes, public offerings or grant awards. Funding figure excludes future milestone considerations received in conjunction with partnerships and collaborations such as those with Boehringer Ingelheim, Imbrium Therapeutics L.P., Shionogi & Co., Ltd. or Eli Lilly. Funding figure does not include Vor’s gross proceeds of $203.4 million from its February 2021 post-period IPO or Karuna’s gross proceeds of $269.8 million from its February 2021 post-period follow-on offering. Calculated as of January 1, 2017 to December 31, 2020.
4 This represents a non-IFRS number. For a reconciliation of this number to IFRS, please see below:
Consolidated Cash Reserves is a non IFRS-measure which consists of cash, cash equivalents and short-term investments held at PureTech Health plc and consolidated subsidiaries. PureTech Level Cash Reserves is a non-IFRS measure which consists of cash, cash equivalents and short-term investments held at PureTech Health plc and only wholly-owned subsidiaries. PureTech Level Cash and Cash Equivalents is a non-IFRS measure which consists of cash and cash equivalents held at PureTech Health plc and only wholly-owned subsidiaries.