At PureTech, we are developing new categories of therapeutics by building upon the validated biology of known therapeutics while applying unique innovative steps that vastly improve pharmacologic profiles with the potential for broad application
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Wholly Owned Pipeline
Immunosuppressant
Non-opioid
| Phase completed | Phase in progress | Registration-enabling studies to begin in 1H2022 |
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Founded Entities
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 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. Funding figure does not include Gelesis’ gross proceeds of approximately $105.0 million from its January 2022 SPAC merger.
3 This represents a non-IFRS measure used by management for planning and reporting purposes. Please see below for a reconciliation of this measure to consolidated cash and cash equivalents, which is the most directly comparable measure calculated in accordance with IFRS:
PureTech Level Cash and Cash Equivalents is defined as cash and cash equivalents held at PureTech Health plc and its wholly-owned subsidiaries only. PureTech Level Cash and Cash Equivalents is an alternative performance measure which is adjusted and constitutes a non-IFRS measure. We believe that these non-IFRS performance measures, when provided in combination with IFRS measures, will provide investors, analysts and other stakeholders with helpful complementary information to better understand our financial position from period to period. The measures are not substitutable for IFRS measures and should not be considered superior to measures presented in accordance with IFRS. These figures are unaudited and do not present all information necessary for an understanding of the Company’s financial condition as of December 31, 2021 or March 31, 2022.
*Presumes the exercise of all call options written by the Company covering 477,100 Karuna shares with expiration dates in August and September 2022.