SaluTech is developing a Conductive Biomaterial (CBM) to treat Atrial Fibrillation by resynchronizing the electrical propagation in the heart. CBM is a non-toxic, long-lasting, and biocompatible nano-membrane that mimics the conductive velocity of healthy heart tissue – addressing the underlying conduction issues to improve the synchronization of heart cell electrical and mechanical activity. The technology is patent protected and supported by 11 published papers. Extensive pre-clinical studies show significant reduction in cardiac arrhythmia, improved heart function, and safety for up to 12 months. SaluTech is currently completing animal studies, and looking to move forward to start clinical trials.

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‍UNMET NEED/COMPETITIVE ADVANTAGE

Atrial Fibrillation (AF) is irregular atrial tissue contraction, in which patients have a 5-fold increased risk for stroke, 3-fold increased risk for heart failure, and 2-fold increased risk for dementia. Over 33 million people globally suffer from AF, and this number is growing by over 5% annually due to an aging population. In Canada, a study from 2007-2008 found that AF was already creating a substantial economic burden to the healthcare system, costing over $800 million in just hospital costs. The death rate associated with AF as the primary contributing factor has continued to rise for over two decades.

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‍INNOVATION

‍SaluTech's conductive biomaterial is the only AF treatment focused on addressing the underlying conduction issues within the heart and restoring a healthy heart beat. This provides a highly effective long-term solution, without further damaging the heart or causing side-effects.

 For more information: https://www.salutechlimited.com/

SaluTech has developed a novel conductive biomaterial (CBM) to treat patients living with AF by addressing the underlying electrophysiological cause. The CBM consists of a conductive polymer, combined with a biomaterial to allow for a biocompatible and long-lasting membrane that can be easily used in the human body.  

 THE CBM is a platform technology that can also be used for treating other key cardiovascular issues including ventricular tachycardia led heart failure, improving implantable devices, and replacing synthetic surgical materials.

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‍APPLICATION/UTILITIES

SaluTech has developed a novel conductive biomaterial (CBM) to treat patients living with AF by addressing the underlying electrophysiological cause. The CBM consists of a conductive polymer, combined with a biomaterial to allow for a biocompatible and long-lasting membrane that can be easily used in the human body.  

 THE CBM is a platform technology that can also be used for treating other key cardiovascular issues including ventricular tachycardia led heart failure, improving implantable devices, and replacing synthetic surgical materials.

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INVESTMENT OBJECTIVES: 

US$5M Seed round to:

(1) Complete biocompatibility testing and GLP large animal testing

(2) Develop prototypes for minimally-invasive delivery device

(3) Hire experienced team and grow clinical advisory group

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COMPETITIVE ADVANTAGE:

-         Bio-Conductivity– capable of synchronizing myocardial conduction by matching the conductive velocity of myocardial tissue

-         Bio-Compatibility- patented technique to conjugate conductive polymers onto biomaterial creates a conductive, yet biocompatible material

-         Flexibility in shape - flexible membrane that allow for proper attachment onto atrial tissue. The flexibility supports future commercial use through a minimally-invasive delivery procedure

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MARKET SIZE:

The global AF device market is $4.1 billion  (2018); it is growing rapidly at a CAGR of 13.6%, and is estimated to be $7.8  billion in 2023

PATENT PORTFOLIO:

Two patents for matter composition and application of use:

 Patent 1: Conductive Biomaterial for Enhancement of Conduction in vitro and in vivo

 Patent 2: Conductive Benzoic Acid-based Polymer Containing Biomaterial for Enhancement of Tissue Conduction invitro and in vivo

 Additional patents are planned for the next generation of the technology, as well as patents related to development of a minimally-invasive delivery device.

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READINESS LEVEL:

After completing a Pre-Submission meeting with the FDA, we have confirmed all the remaining testing that we need to complete before starting clinical trials and ensure completion is in line with regulatory expectations.

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MANAGEMENT TEAM:

Shuhe Li, JD/MBA (CEO)

Shuhe Li previously worked at the Bank of Montreal Capital Markets as an Investment Banking Associate in the North American Financial Institutions Group. She covered international companies, and worked on buy- and sell-side mandates, debt and equity issuances, and shareholder activism defense.

Shuhe co-founded the company, and has led business development through the license negotiation, fundraising of pre-seed round, and management of the patent and

regulatory process.

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INVENTOR(s) /SCIENTIFIC FOUNDER(s):

Ren-Ke Li, MD/PhD/FCAHS (CSO)

Dr. Ren-Ke Li is the inventor of SaluTech’s conductive biomaterial. Dr. Li is a Senior Scientist at the Toronto General Hospital Research Institute, University Health Network and a Professor of Medicine in the Department of Surgery, Division of Cardiac Surgery at the University of Toronto. He is the Canada Research Chair in Cardiac Regeneration(Tier 1), was a Career Investigator of the Heart and Stroke Foundation of Canada, and has published 276 peer-reviewed papers in reputable journals. He is Editor-in-Chief of Stem Cells International Journal and Fellow of Canadian Academy of Health Science since 2011.

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Medly Therapeutics (Medly) is a digital health company transforming care for cardiac and chronic disease patients. Developed based on technology invented at the University Health Network in concert with its Peter Munk Cardiac Centre (PMCC) and Centre for Digital Therapeutics as well as the Ted Rogers Centre for Heart Research, the Medly program is a first of its kind in Canada. The program uses the Medly management algorithm developed by heart failure clinicians to rapidly assess and triage patients, and includes access to a digital health team for patients. Medly demonstrates unrivaled clinical validation with a 50% reduction in heart failure related hospitalizations and a 6-fold increase in patient capacity, as well as validation for its platform technology via 27 peer-reviewed publications.

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UNMET NEED

50% of  heart failure patients are re-hospitalized within 6 months of admission, leading to significant health system costs. There is a current shortage of health care staff specializing in cardiac care, e.g.(nurses, cardiologists and primary care physicians who are trained in heart failure management. Consequently, clinicians are forced to manage care by events instead of outcomes, a costly approach.

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INNOVATION
Medly Therapeutics (Medly) combines gold standard protocols integrated with a clinician developed expert system, directing the patient through an automated front (e.g. smart phone app or digital portal) that transforms event-based care to proactively managing patients to their baseline. Medly demonstrates unrivaled clinical validation with a 50% reduction in heart failure related hospitalizations and a 6-fold increase in patient capacity. Medly's platform has gained validation in over 27 peer-reviewed publications.

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‍APPLICATION/UTILITIES

The Medly platform is being prescribed by Canadian clinicians to manage their heart failure patients on a daily basis and boasts an 80% adherence rate in patients over 70 years of age. Medly is changing the face of chronic disease management with additional applications including co-morbidities such as diabetes, COPD and mental health/depression.

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‍INVESTMENT & OBJECTIVES

$3M USD Series Seed; $10M pre-money valuation with $2.4M committed.

Investment would be targeted to:

·   Port the platform to the cloud for commercial scale

·  Continue with FDA application (breakthrough designation received 11/22)

·  Execute2-3 US pilots (pilot sites in discussion

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‍COMPETITIVE ADVANTAGE

1)   Evidence-based (27 peer reviewed publications; competition has only 1)

2)  (Validated) trade-secret data and algorithms - usually takes years take years to validate

3)  Successful early adoption in Canada

4) Breakthrough designation from FDA

5) Health Canada approval for medical device

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‍MARKET SIZE

US and Canada TAM $22B

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‍IP PORTFOLIO

Extensive trade secret portfolio; additional patents being filed on remote medication titration and multiple chronic condition management

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‍CURRENT MANAGEMENT TEAM

Tracey Dodenhoff, CEO

With a personal mission to “Help Good People do Great Things with Innovation”, Tracey is an impact-focused entrepreneur dedicated to high value solutions. Tracey’s background encompasses technology entrepreneurship, innovation leadership and venture investing. Over her career, Tracey has launched and invested in over two dozen startups, as well as partnering with ventures to accelerate the growth of new product initiatives across medical, consumer, software and industrial verticals. Tracey specializes in integrating innovative programming with technology, resulting in game-changing collaborations across geographies and disciplines. She holds an MBA from Babson College, an MID from Pratt Institute and a BSc from Rhode Island College. She is also the holder of several patents in medical device and software. She advises and mentors early stage ventures across medtech, advanced materials and data science.

The company also includes a VP of Engineering, Clinical Director, CTO, CFO, and VP of Operations.

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‍CURRENT READINESS LEVEL

TRL 8 – Porting to the commercial cloud platform (completion by Q3 2023 this year).

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‍‍SCIENTIFIC FOUNDER(S)

Dr. Heather Ross - Head Division of Cardiology at the Peter Munk Cardiac Centre, Professor of Medicine, University of Toronto, Site Lead Ted Rogers Centre for Heart Research

Dr. Joseph Cafazzo - ‍Executive Director, Biomedical Engineering, the Centre for Digital Therapeutics, and Healthcare Human Factors (HHF) at UHN, and Wolfond Chair in Digital Health

Nearly half of all patients receiving treatment for cancer will receive radiation treatment. The radiation treatment process takes clinicians hours, sometimes days, to complete. Curait Medical has a patent-protected platform providing automated, quantitative quality review enabling the radiation team to make more informed clinical decisions for each radiation treatment with reduced costs, increased quality, higher patient throughput, and fewer treatment errors. Curait's medical platform is Software as a Service (SaaS) based, enabling users access to machine learning of clinical practice and the ability to build models based on their clinical expertise. In addition, the platform facilitates more objective and consistent clinical practices across cancer centers, making it a valuable tool for healthcare managers.

‍UNMET NEED

There is a significant shortage of clinically trained experts in radiation oncology able to deliver radiation treatment to cancer  patients. In short, we will not be able to train enough professionals to meet  the increasing clinical demands associated with treatment delivery.

The radiation treatment process requires tremendous clinical expertise, is highly time consuming and expensive. Radiation treatments are planned (i.e. simulated) using specialized software and are subject to rigorous review by the entire radiation treatment team, before any radiation is approved and delivered to the patient. The radiation treatment plan is customized for each patient and specifies how the radiation treatment machine will deliver the radiation, and the radiation expected to reach the targeted tumour and any healthy organs.

RT treatment planning involves collaborating with a multidisciplinary team through repeated touchpoints. Curait’s patented SaaS uses machine learning to aid the team in evaluating and reviewing thousands of characteristics for each treatment plan in minutes. Whereas the total treatment planning process can take many hours to days to complete, with Curait, that time can be reduced by 30%, typically saving 3-6 hours per patient. This is because the Curait platform is designed to perform on-demand consultation, helping technicians complete treatment plans quickly, as well as expedite the mandatory treatment plan reviews for medical physicists and radiation oncologists. Curait’s SaaS improves workflow and eliminates expensive process bottlenecks resulting from handoffs within the radiation treatment team, thereby helping patients get their treatment faster.

The platform is vendor-agnostic, scalable, and can readily be integrated into the clinical workflow of any cancer center. It increases both the quality of and access to radiation treatment, while  providing immediate savings. The increased capacity realized using the Curait platform will positively impact revenues.

‍INNOVATION

Curait’s SaaS learns the characteristics of high quality treatment plans to build machine learning “models”.  It has been developed based on a comprehensive database from one of the world’s top 5 cancer centres,the Princess Margaret Cancer Centre. Curait’s SaaS also connects the radiation treatment team to a constantly expanding database of models.

Curait’s SaaS creates a personalized approach for reviewing each patient’s care by drawing on thousands of relevant historical cases and it can automatically access the most applicable patient data without relying on hard-coded metrics being entered by the user, as is common practice.

Curait also provides the team with understandable reasoning behind the evaluation of each component of the radiation treatment plan to aid in both identifying specific issues/errors, saving the radiation oncologist from having to complete multiple intermediary reviews, and in providing direct insights for making necessary adjustments to improve the radiation treatment plan quality.

For more information: https://curaitmedical.com/

‍APPLICATION/UTILITIES

‍Curait’s QA solution is applicable to all cancer patients requiring radiation treatment.  The technology can be easily integrated into any healthcare application that leverages a treatment plan.

‍INVESTMENT & OBJECTIVES

‍Investment Raise: $2 Million

Investment would be targeted to:

·  Product Development (45%) 

·  Sales-Marketing Commercialization (30%)

·  General Administration (25%)

‍COMPETITIVE ADVANTAGE
Patent pending with priority date in the U.S.; AI training using patient datasets from one of the world’s top 5 cancer centres; automatically learned clinical practice metrics.

‍MARKET SIZE
RT addressable market:  >US$500M

‍IP PORTFOLIO
The company's IP portfolio includes a patent pending for  automated quality assurance in radiation therapy.

US patent 201180211725A1: https://patents.google.com/patent/US20180211725A1/  

‍CURRENT MANAGEMENT TEAM
Curait's management team is experienced and has a track record of successfully navigating early-stage companies to commercial success.

‍CURRENT READINESS LEVEL

‍Curait’s platform (“Thor”) contains the following products:

Thor Data; completed – ready for sale.

Thor Audit; completion and sale readiness Q4 2023 (assuming financing)

Thor On-Demand; requires 510K for regulatory approval – completion  and sale readiness in 2024

Note: a NON-AI version of Thor Audit and On-Demand is  being used clinically (>9,000 patients) – the products are not fully  commercial-ready, but functional.

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‍‍SCIENTIFIC FOUNDER(S)
Dr. Thomas Purdie, Medical Physicist, Princess Margaret Cancer Centre; Associate Professor, University of Toronto.

Dr. Purdie is a staff medical physicist/clinician scientist and is the recipient of academic awards from NSERC and CIHR. Dr. Purdie’s research lab focuses on developing and deploying machine learning algorithms and methods for automating clinical radiation oncology workflow processes, including radiation treatment planning, quality assurance, and decision support.

‍Dr. Chris McIntosh, Toronto General Hospital Research Institute

Dr. McIntosh is the recipient of academic awards from NSERC, CIHR, and the Michael Smith Foundation for Health Research. His lab is focused on the theory and clinical application of AI in medicine for improving patient care including transfer learning, meta learning, computer vision, and explainable AI. Applications include deep learning for automated diagnosis, segmentation, quality assurance, and treatment planning.

Our novel drug candidate blocks the cis-interaction between Repulsive Guidance Molecule A (RGMa) and Neogenin and disrupts Neogenin incorporation in lipid rafts. This candidate’s therapeutic interaction promotes cell survival and axonal regeneration in severe medical conditions including retinitis pigmentosa, multiple sclerosis, ischemia (e.g., stroke), and spinal cord injury. The ideal strategy to ameliorate CNS damage is through promoting both survival and axonal regeneration.

UNMET NEED
4IG targets the neuronal regeneration pathway that has not yet been drugged. Multiple disease indications are implicated including Retinitis Pigmentosa, Stroke, Spinal Cord Injury, and Multiple Sclerosis.

INNOVATION
Biologic therapeutic comprised of ~400 amino acid, soluble Ig extracellular domain of Neogenin

Dual mechanism of action by:
1.binding (Repulsive Guidance Molecule a (RGMa) and
2. inhibiting Neogenin incorporation in lipid rafts

Potential therapeutic for preventing neuronal death in multiple human indication through direct effect at neurons and indirect effect through reducing permeability of Blood Brain Barrier

APPLICATION/UTILITIES
Biologic therapeutic targeting diseases involving neurodegeneration; multi-disease potential (one product).

INVESTMENT & OBJECTIVES
 $5-7M for IND enabling studies.

COMPETITIVE ADVANTAGE
Closest competitors are elezanumab and unasnemab, which are under development from AbbVie Inc and Mitsubushi Tanabe Pharma Corp, respectively.   Both elezanumab and unasnemab  are intravenously administered, α-RGMa monoclonal antibodies. Elezanumab is under Phase II development for the treatment of relapsing-remitting multiple sclerosis, acute ischemic stroke, primary and secondary progressive multiple sclerosis and spinal cord injury. Unasnemab is under Phase II development for the treatment of spinal cord injury and Phase I development for the treatment of Tropical Spastic Paraparesis.

Whereas elezanumab and unasnemab have a single mechanism action, 4Ig both binds RGMa and inhibits the incorporation of Neogenin in lipid rafts.

‍MARKET SIZE
·   Global stroke therapy market expected to grow to over $US10B by 2027

·   Global Multiple Sclerosis therapy market expected to grow to over US$29B by 2030

·   Global Retinitis Pigmentosa therapy market expected to exceed US$2B by 2030

·   Global spinal cord injury treatment market expected to reach almost US$3B by 2027

IP PORTFOLIO
Two patent families describing methods, with 6 issued patents and 1 issued patent.

CURRENT MANAGEMENT TEAM
‍n/a

CURRENT READINESS LEVEL
Proof of concept/pre-clinical.‍

EIR STATUS
‍Recruitment in progress.

‍SCIENTIFIC FOUNDER(S)
Dr. Philippe Monnier, Senior Scientist, Krembil Research Institute, an accomplished researcher focused on uncovering the role of extracellular proteins in the developing and regenerating CNS.

Porphysomes are a platform technology based on lipid molecules that form porphysome nanoparticles, with versatile applications including cancer imaging, phototherapy, and drug delivery. Porphysome nanoparticles have the unique ability to be loaded with drugs, such as chemotherapeutics or immunotherapy agents, and have properties that make them ideal for both targeted photothermal therapy (PTT) and photodynamic therapy (PDT) and photo-immune stimulation. Porphysomes are also capable of fluorescence, which enables more precise, targetted tumor treatment.They also selectively accumulate in cancer tissue and release photoenergy when a specific laser light is directed at the tumor, which means they can selectively ablate malignant tissue. Additionally, porphysomes can be modified with heavy metals to facilitate PET imaging.

‍UNMET NEED/COMPETITIVE ADVANTAGE
‍The FDA approved the only photosensitizer agent available in the US for internal solid tumors in 1995.  While this agent has been proven to be effective in both early and late-stage cancers, the major limitations are severe skin photosensitivity that can last >30 days and the inability to fluoresce cancer cells. Porphysomes represent an advanced generation of photosensitizers with minimal expected side effects and additional capabilities, such as enhanced tumor detection and visualization via fluorescence.  

‍INNOVATION
Porphysomes are a patented porphyrin-lipid technology. The porphyrin component of a porphysome delivers the photosensitizing property while the lipid component provides additional functional capabilities. These multi-functional characteristics provide the opportunity to design agents for a variety of applications.

‍APPLICATION/UTILITIES
The initial target application for porphysomes is peripheral lung cancer, which is defined as tumors that reside in the outer 1/3 of the lung. The development pipeline also includes oropharyngeal cancer, pancreatic cancer and skin cancer.

‍INVESTMENT & OBJECTIVES
$10-15M USD, for the purpose of: 

-        GMP manufacturing of clinical drug product       

-        Initiation of first-in-human phase-I studies in peripheral lung cancer

‍MARKET SIZE
Estimated market value of porphysomes in peripheral lung cancer is $1 Billion USD. CAGRis estimated at 3-5%.

IP PORTFOLIO
Three patents cover the composition of porphysomes, usage in photothermal therapies and photoacoustic imaging as well as different porphysomes with alternative lipid conjugates. Several patent filings are in progress covering porphysome modifications that enhance uptake into cancer cells and enhance ability to carry chemotherapeutic agents (e.g.paclitaxel), as well as patents focused on proprietary potency testing device sand methods.

‍CURRENT READINESS LEVEL
‍Regulatory IND/CTA filings followed by the initiation of first in human trials

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MANAGEMENT TEAM
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Glenn Kutschera, CEO 

Glenn is an experienced industry executive with recent experience as president of a U.S. photodynamic therapy company.

Paul Sieroslawski, CFO/COO

Paul has 12 years of experience working as a finance and operations executive in the pharmaceutical and medical device industry, most recently at a photodynamic focused company.

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Dr. Gang Zheng - Senior Scientist and Associate Research Director, Princess Margaret Cancer Centre & Scientific Co-Founder

Porphysomes are based on ground-breaking research by Dr. Gang Zheng, a world-renowned expert in nanomedicine and Associate Research Director at the Princess Margaret Cancer Centre. Named contributor to the “top 10 cancer breakthroughs of 2011” by the Canadian Cancer Society for his work on harnessing porphysome nanotechnology in the fight against cancer, Dr. Zheng’s nanotechnology platform is the foundation for this new venture. For more information: http://zhenglab.utoronto.ca/

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Dr. Brian Wilson - Senior Scientist, Princess Margaret Cancer Center; Professor, University of Toronto

Dr. Wilson's work lies in the development and commercialization of novel technologies and methods that can be translated to cancer patients and that are based on light and nanoparticles. The applications include cancer detection/diagnosis (using different forms of optical spectroscopy and imaging), cancer treatment (using light-activated drugs), and image-guided cancer interventions (surgery guided by fluorescence imaging).

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Dr. Michael Valic, Scientist, Princess Margaret Cancer Centre

Doxorubicin is effectively used in various cancers despite a significant risk of cardiotoxicity associated with treatment usage.  In an attempt to manage cardiotoxicity risk, there is a lifetime dosage limit.  With each dose, the risk of cardiac dysfunction increases.  Despite the attempt to manage cardiotoxicity risk up to about one third of patients experience cardiac dysfunction and many go on to develop heart failure. Dantrolene is a muscle relaxant that was first approved by the US FDA in1974.  Its primary use is treatment of malignant hyperthermia, an anesthesia emergency.  Research at UHN has shown that dantrolene is protective of the myocardium when used as a pre-treatment for doxorubicin. Dantrolene administration thus has significant potential to increase or remove the lifetime dosage limit for patients and protect the heart, preventing cardiac dysfunction and eventual chemotherapy induced heart failure.

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‍UNMET NEED
Doxorubicin is commonly used to treat breast cancer, leukemia, lymphoma, and myeloma. It is also a second line drug in many additional cancers.  About one third of all doxorubicin is used in breast cancer, making it the greatest use of the drug.  Breast cancer is often first diagnosed at a relatively young age.  Surviving breast cancer only to go on to develop cardiac dysfunction adds tragedy to a difficult situation.  Reducing or eliminating the risk of cardiac dysfunction can add many years and quality of life for cancer survivors.

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INNOVATION
Dantrolene is traditionally used for the acute treatment of malignant hyperthermia.  It also finds some niche applications managing other symptoms that malignant hyperthermia patients can experience.  Research at UHN has demonstrated that dantrolene can prevent the damage to the heart caused by doxorubicin.  Literature demonstrates that use of dantrolene does not impact the efficacy of cancer treatments. Further, dantrolene has applications in ventricular arrhythmias.

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APPLICATION/UTILITIES
·        Prophylaxis of chemotherapy induced cardiotoxicity

·        Acute treatment of Ventricular Tachycardia (VT)

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‍INVESTMENT & OBJECTIVES
$5M - $15M required to conduct: 

·        Phase I pharmacokinetics of reformulated Dantrolene in a UHN Clinic. 

·        Phase I/II dose escalation study in the target disease (breast cancer) sample

·        Phase I/II study of dantrolene on patients suffering from idiopathic PVCs.

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COMPETITIVE ADVANTAGE
To date, one drug has been approved for the mitigation of cardiac dysfunction associated with doxorubicin.  It is marginally effective and as a result experiences minimal sales. Many other drugs including beta blockers, calcium channel blockers, and statins, have been tested to reduce cardiotoxicity of doxorubicin. None have shown sufficient activity to warrant further development.  

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‍MARKET SIZE
In the US, nearly 300,000 patients are diagnosed annually with breast cancer.  Two thirds of them are eligible for chemotherapy and but half choose to undergo the treatment.  This results in about 90,000 addressable patients annually in the US.  Doxorubicin currently sells US$1.3B annually and its use is increasing despite substantial risk of cardiotoxicity. The success of an abatement for cardiotoxicity as well as potentially selling as much as doxorubicin may increase the demand for doxorubicin-based chemotherapy.

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IP PORTFOLIO
Two patent families, including issued patents in EU and US, covering multiple applications of the technology.

‍CURRENT READINESS LEVEL
Pre-clinical technology, requires demonstration in clinical trial. 

EIR
Nigel deGruyther

Nigel deGruyther is a pharmacist and entrepreneur with experience in drug repositioning efforts and venture capital and the former Head of Business Development at Cynapsus Therapeutics.

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‍SCIENTIFIC FOUNDER(S)
Dr. Kumaraswamy Nanthakumar is a clinical cardiologist and cardiac electrophysiologist at UHN and a Senior Scientist at the Toronto General Hospital Research Institute, with a focus in cardiac arrhythmia modulation and stabilization of RyR2. He is also Professor of Medicine at the University of Toronto.

Gliachem is developing a small molecule therapeutic whose first clinical opportunity will be Rett Syndrome, a rare, pediatric disease with limited treatment options.

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‍UNMET NEED

Rett Syndrome is a rare, X-linked genetic neurological and developmental disorder that affects the way the brain develops. With approximately 350,000 patients worldwide, it manifests primarily in young girls, presenting with a wide range of disabilities and often misdiagnosed as general autism, cerebral palsy, or other conditions of pervasive developmental delay. Limited treatment options exist for these patients, most of which are off-label treatments for symptom management & occupational therapy. There is one approved drug (Trofinitide, Acadia Pharmaceuticals; approved 2023) which has a small effect size and tolerability issues, leaving significant room for improvement.

Rett Syndrome is caused by mutations of the MECP2 gene. In Rett patients, the brain fails to properly mature, remains under-developed throughout life, and functions inefficiently.  While the errant function of many systems contributes to these neural impairments, the hypoactivity of mTOR represents a key convergence point of pathogenic processes. Strategies to improve mTOR activity represent potential treatments for Rett Syndrome. The Transient Receptor Potential Cation Channel Subfamily M Member 2 (TRPM2) is one system that negatively regulates mTOR, and is over-expressed in the Rett Syndrome brain. We have developed new molecules that block TRPM2. These small molecules display high affinity and selectivity for TRPM2, and represent a first-in-class approach to engage TRPM2 for translational development in Rett Syndrome.  

‍APPLICATION/UTILITIES
The first clinical indication for our small molecule will be Rett Syndrome. However, TRPM2 has been implicated in other indications beyond Rett. We will look to validate additional clinical indications once we have the compounds to build the pipeline.

‍INVESTMENT & OBJECTIVES

‍We are currently looking to raise a $2.9M seed round, which will enable us to complete our hit to lead activities and select our lead candidate in ~12-18 months. This includes proof of concept in a mouse model and a comparison study of our lead vs. Trofinitide. A follow-on round of ~$5M will support Lead Optimization and a Series A of ~$5M will advance us through IND-enabling studies.

‍MARKET SIZE
350,000 patients worldwide, including major markets of EU (13k), USA(11k), Canada (4k) & Japan (1k). With only one approved drug (Trofinitide)which has a small effect size and tolerability issues, there is an opportunity to capture a significant segment of this market.

‍IP PORTFOLIO
Composition patent applications pending (NCEs).

CURRENT READINESS LEVEL
‍Hit to lead

‍MANAGEMENT TEAM

Vanessa Williamson – Entrepreneur-in-Residence/CEO

Vanessa’s career has focused on helping life sciences companies grow, including leadership roles in industry and innovation intermediary organizations. She began her career as a development scientist with NPS Pharmaceuticals, where she successfully scaled up and transferred clinical and commercial manufacturing processes for small molecule therapeutics. Most recently, she held VP roles with scaling molecular diagnostics companies, responsible for M&A, in-licensing, strategic partnerships and financing.

‍SCIENTIFIC FOUNDER(S)
This new initiative is a collaboration among distinguished researchers at the Krembil Brain  Institute:

- Dr. Mark Reed, expertise in medicinal chemistry, chemical biology & CNS drug discovery, and leads UHN’s Centre for Medicinal Chemistry and Drug Discovery

- Dr. James Eubanks, epigenetic and mitochrondrial diseases expert, and a Rett Syndrome KOL.