Cyclo Therapeutics: (OTCQB: CTDH) is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The Company’s Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, is used to treat Niemann-Pick Disease Type C (NPC), a rare and fatal genetic disease, on a compassionate use basis as well as in three ongoing formal clinical trials. The Company’s Scientific Advisory Board endorsed Alzheimer’s Disease (AD) as the company’s next drug development indication. The Company was authorized by the FDA to use Trappsol® Cyclo™ for a late-onset AD patient under a Compassionate Use Program which began intravenous dosing in May 2018. Learn more at CTDHinfo.com.
Annovis Bio is a clinical-stage, drug platform company addressing neurodegeneration, such as Alzheimer’s disease (AD), Parkinson’s disease (PD) and Alzheimer’s in Down Syndrome (AD-DS). Annovis is believed to be the only company developing a drug for AD, PD and AD-DS that inhibits
more than one neurotoxic protein and improves the information highway of the nerve cell, known as axonal transport. When this information flow is impaired, the nerve cell gets sick and dies. The company expects its treatment to improve memory loss and dementia associated with AD and AD-DS, as well as body and brain function in PD. Annovis has an ongoing
Phase 2a study in AD patients and a second Phase 2a study in early PD and early AD patients.
Treatment landscape of alk+ nsclc 12 novemberssuser4c22ca
This document provides an overview of treatments for ALK+ non-small cell lung cancer (NSCLC). It discusses the epidemiology of ALK+ NSCLC, occurring in 3-5% of NSCLC cases worldwide. First generation ALK inhibitors like crizotinib provided significant benefits for patients. Current NCCN guidelines recommend first-line treatment with newer ALK inhibitors such as alectinib, brigatinib, or lorlatinib which have led to median overall survival rates of over 5 years for some patients with metastatic ALK+ NSCLC. The document reviews the classes of ALK inhibitors including first, second, and third generation treatments and their effectiveness against ALK+ NSCLC.
Annovis Bio is a clinical-stage, drug platform company addressing
neurodegeneration, such as Alzheimer’s disease (AD), Parkinson’s disease
(PD) and Alzheimer’s in Down Syndrome (AD-DS). Annovis is believed to
be the only company developing a drug for AD, PD and AD-DS that inhibits
more than one neurotoxic protein and improves the information highway of
the nerve cell, known as axonal transport. When this information flow is
impaired, the nerve cell gets sick and dies. The company expects its
treatment to improve memory loss and dementia associated with AD and
AD-DS, as well as body and brain function in PD. Annovis has an ongoing
Phase 2a study in AD patients and a second Phase 2a study in early PD
and early AD patients.
Cellgen Diagnostics is an early stage venture that is developing a break through Companion Diagnostic platform that will enable Precision Medicine by determining whether a patients genetic profile is a match for the prescribed cancer therapeutic.
This corporate presentation provides an overview of Oncolytics Biotech and its lead product REOLYSIN. Key points include:
- REOLYSIN is a proprietary reovirus being tested in combination with chemotherapy in multiple randomized phase 2 clinical trials for various cancers.
- Completed phase 2 trial REO 018 in head and neck cancer showed increased progression-free and overall survival when combined with chemotherapy.
- Ongoing trials include studies sponsored by the NCI in pancreatic, ovarian, lung, and breast cancers.
- REOLYSIN works by selectively replicating in Ras-activated cancer cells and is believed to have a favorable safety profile.
CLBS Corporate Slide Presentation March 2018Steve Sizer
This corporate presentation discusses Caladrius Biosciences' business model evolution and future plans. It summarizes that Caladrius has transitioned to focus solely on clinical-stage therapeutics development using two technology platforms: autologous T-regulatory cells for immune modulation and CD34 cells for ischemic repair. Key programs outlined include an ongoing Phase 2 trial of T-regulatory cells for recent-onset type 1 diabetes and planned Phase 2 trials of CD34 cells for critical limb ischemia and coronary microvascular dysfunction. The presentation positions Caladrius for continued growth with a well-funded pipeline and strategic partnerships.
The corporate presentation summarizes Oncolytics Biotech's lead product REOLYSIN, a therapeutic reovirus being studied in multiple clinical trials. Key points include:
- REOLYSIN has been administered to over 1,000 patients safely and shows efficacy against Ras-activated cancers.
- A randomized Phase II trial of REOLYSIN in head and neck cancer, REO 018, showed improved tumor stabilization and shrinkage for those receiving REOLYSIN compared to the control arm.
- Oncolytics intends for REO 018 to support a planned Phase III registration trial of REOLYSIN in head and neck cancer.
IntelGenx is an innovative pharmaceutical film company presenting its product pipeline and technology platforms to investors. The presentation discusses IntelGenx's oral thin film and buccal film technologies, product development strategies, business model, pipeline of products including treatments for migraines, erectile dysfunction, and brain degenerative diseases, and clinical trial results. It highlights a de-risked product candidate using montelukast to treat brain degenerative diseases and significant market opportunities for its pipeline products.
Annovis Bio is a clinical-stage, drug platform company addressing neurodegeneration, such as Alzheimer’s disease (AD), Parkinson’s disease (PD) and Alzheimer’s in Down Syndrome (AD-DS). Annovis is believed to be the only company developing a drug for AD, PD and AD-DS that inhibits
more than one neurotoxic protein and improves the information highway of the nerve cell, known as axonal transport. When this information flow is impaired, the nerve cell gets sick and dies. The company expects its treatment to improve memory loss and dementia associated with AD and AD-DS, as well as body and brain function in PD. Annovis has an ongoing
Phase 2a study in AD patients and a second Phase 2a study in early PD and early AD patients.
Treatment landscape of alk+ nsclc 12 novemberssuser4c22ca
This document provides an overview of treatments for ALK+ non-small cell lung cancer (NSCLC). It discusses the epidemiology of ALK+ NSCLC, occurring in 3-5% of NSCLC cases worldwide. First generation ALK inhibitors like crizotinib provided significant benefits for patients. Current NCCN guidelines recommend first-line treatment with newer ALK inhibitors such as alectinib, brigatinib, or lorlatinib which have led to median overall survival rates of over 5 years for some patients with metastatic ALK+ NSCLC. The document reviews the classes of ALK inhibitors including first, second, and third generation treatments and their effectiveness against ALK+ NSCLC.
Annovis Bio is a clinical-stage, drug platform company addressing
neurodegeneration, such as Alzheimer’s disease (AD), Parkinson’s disease
(PD) and Alzheimer’s in Down Syndrome (AD-DS). Annovis is believed to
be the only company developing a drug for AD, PD and AD-DS that inhibits
more than one neurotoxic protein and improves the information highway of
the nerve cell, known as axonal transport. When this information flow is
impaired, the nerve cell gets sick and dies. The company expects its
treatment to improve memory loss and dementia associated with AD and
AD-DS, as well as body and brain function in PD. Annovis has an ongoing
Phase 2a study in AD patients and a second Phase 2a study in early PD
and early AD patients.
Cellgen Diagnostics is an early stage venture that is developing a break through Companion Diagnostic platform that will enable Precision Medicine by determining whether a patients genetic profile is a match for the prescribed cancer therapeutic.
This corporate presentation provides an overview of Oncolytics Biotech and its lead product REOLYSIN. Key points include:
- REOLYSIN is a proprietary reovirus being tested in combination with chemotherapy in multiple randomized phase 2 clinical trials for various cancers.
- Completed phase 2 trial REO 018 in head and neck cancer showed increased progression-free and overall survival when combined with chemotherapy.
- Ongoing trials include studies sponsored by the NCI in pancreatic, ovarian, lung, and breast cancers.
- REOLYSIN works by selectively replicating in Ras-activated cancer cells and is believed to have a favorable safety profile.
CLBS Corporate Slide Presentation March 2018Steve Sizer
This corporate presentation discusses Caladrius Biosciences' business model evolution and future plans. It summarizes that Caladrius has transitioned to focus solely on clinical-stage therapeutics development using two technology platforms: autologous T-regulatory cells for immune modulation and CD34 cells for ischemic repair. Key programs outlined include an ongoing Phase 2 trial of T-regulatory cells for recent-onset type 1 diabetes and planned Phase 2 trials of CD34 cells for critical limb ischemia and coronary microvascular dysfunction. The presentation positions Caladrius for continued growth with a well-funded pipeline and strategic partnerships.
The corporate presentation summarizes Oncolytics Biotech's lead product REOLYSIN, a therapeutic reovirus being studied in multiple clinical trials. Key points include:
- REOLYSIN has been administered to over 1,000 patients safely and shows efficacy against Ras-activated cancers.
- A randomized Phase II trial of REOLYSIN in head and neck cancer, REO 018, showed improved tumor stabilization and shrinkage for those receiving REOLYSIN compared to the control arm.
- Oncolytics intends for REO 018 to support a planned Phase III registration trial of REOLYSIN in head and neck cancer.
IntelGenx is an innovative pharmaceutical film company presenting its product pipeline and technology platforms to investors. The presentation discusses IntelGenx's oral thin film and buccal film technologies, product development strategies, business model, pipeline of products including treatments for migraines, erectile dysfunction, and brain degenerative diseases, and clinical trial results. It highlights a de-risked product candidate using montelukast to treat brain degenerative diseases and significant market opportunities for its pipeline products.
This document summarizes research on CLN3-Batten disease, a rare neurodegenerative lysosomal storage disorder. It describes a natural history study and biorepository at the National Institutes of Health to gather clinical data and biosamples from well-characterized patients over time and across institutions. The study aims to identify blood-based biomarkers for diagnosis and disease monitoring, clinically meaningful outcomes measures, and advance collaborative research efforts between patients, clinicians, and researchers to develop therapies for this currently untreatable disease.
ACT is conducting three clinical trials for dry age-related macular degeneration (AMD) and Stargardt's disease (SMD) using retinal pigment epithelium (RPE) cells derived from human embryonic stem cells. The trials have shown no adverse events and persistence of the transplanted cells, with functional vision improvements in most patients. ACT has additional clinical programs planned or underway for myopic macular degeneration and has a pipeline of other ophthalmology and regenerative medicine programs. It has strong intellectual property around RPE cell production and therapy. Upcoming milestones include further patient follow-up data and the potential start of Phase II trials. ACT is led by an experienced management team and board of directors.
The document provides information on CEL-SCI Corporation and their investigational cancer immunotherapy drug Multikine, including details on Multikine's mechanism of action, Phase I-II safety profile, "proof of concept" Phase II results, the ongoing pivotal Phase III clinical trial design in head and neck cancer patients, and the potential initial target market if approved. It summarizes key findings from early clinical studies that support Multikine's potential as an effective immunotherapy treatment when administered prior to standard therapies.
- The document is an investor presentation for IntelGenx Corp from April 2018.
- It provides an overview of IntelGenx, including its business strategies, product pipeline, clinical trial results, manufacturing facilities, and leadership team.
- The presentation highlights IntelGenx's drug delivery technology platforms, focus on developing generic and repurposed products, and partnerships with pharmaceutical companies.
This document discusses post-intensive care syndrome (PICS), a condition experienced by many survivors of critical illness involving new or worsening impairments in physical, cognitive, or mental health status after leaving the ICU. It provides an overview of PICS, noting that evidence in recent decades has shown PICS to be a significant problem. The document discusses how certain evidence-based ICU interventions like maintaining light sedation levels and early mobilization may help reduce risks for PICS. It promotes upcoming issues of the publication that will provide more information on PICS and the roles of various professionals in addressing it.
Bellus corporate presentation march 23BellusHealth
This corporate presentation by Roberto Bellini, President and CEO of BELLUS Health Inc (TSX: BLU), provides an overview of the company and its rare disease drug pipeline. The lead product candidate KIACTA has shown positive results in a Phase II/III trial for AA amyloidosis, a rare and deadly kidney disease with no approved treatments. BELLUS plans to complete the ongoing Phase III confirmatory trial in 2016 to support regulatory approval. The company is also developing KIACTA for sarcoidosis and has a second rare disease candidate, Shigamab, in preclinical testing for STEC related hemolytic uremic syndrome.
The document discusses drug repurposing for rare diseases. Drug repurposing involves finding new uses for existing drugs to treat different patient populations. The document provides examples of repurposing drugs to treat rare conditions like Wolfram syndrome and CDKL5. It also discusses using 'omics data to identify repurposing opportunities and describes a potential social impact bond model to fund repurposing clinical trials through healthcare savings.
Nature medicine top ten advancements in biomedicine in 2020DoriaFang
In the field of biomedicine, which is closely related to human health, many innovative therapies have matured this year and promoted medical progress. Recently, the authoritative academic journal "Nature Medicine" published a series of articles reviewing ten remarkable advancements in 2020.
Correlation between Demographic, Socio-economic, and Cancer-Specific Factors with Quality of Life Scores among Newly-Diagnosed Cancer Patients of the Medical Oncology Clinics of the Philippine General Hospital Cancer Institute
https://www.actamedicaphilippina.org/issue/1102
This corporate presentation discusses BELLUS Health's focus on developing drugs for rare diseases. Their lead product candidate, KIACTA, is in Phase 3 trials for AA amyloidosis, a rare and deadly kidney disease with no existing treatment. Positive results from a previous Phase 3 trial showed KIACTA slowed kidney function decline. If successful, KIACTA could achieve premium pricing compared to other orphan drugs. BELLUS is also developing treatments for other rare diseases including sHUS and AL amyloidosis through clinical trials and partnerships.
- Neogenix Oncology announced that members of its scientific staff will present preclinical studies of NEO-101, a monoclonal antibody being developed to treat pancreatic and colorectal cancers, at the AACR annual meeting in April 2011.
- Radient Pharmaceuticals announced preliminary results from a clinical study showing its cancer diagnostic test, Onko-Sure, had a statistical advantage over CEA in detecting early-stage colorectal cancer.
- Cephalon Inc made an offer to acquire the remaining 72.48% stake in ChemGenex Pharmaceuticals it does not already own for approximately $140 million Australian dollars in cash.
Rare Diseases: A Report on Orphan Drugs in the PipelinePhRMA
Rare diseases, when taken together, are not that rare at all. In fact, according to the National Institutes of Health (NIH), 30 million Americans have one of the nearly 7,000 diseases that are officially deemed “rare” because alone they each affect fewer than 200,000 people in the United States.
This document summarizes a webinar on building Canada's strategy for access to rare drugs. Day 1 focuses on challenges to access. Dr. Cheryl Rockman-Greenberg discusses her work on hypophosphatasia (HPP), a rare bone disease. She outlines the different forms of HPP and clinical studies of asfotase alfa treatment. Asfotase alfa was approved in Canada for pediatric HPP but access challenges remain for adults. Real-world evidence is needed to understand natural history and inform treatment criteria for adults. The HPP patient registry collects global data on treatment effects, safety and outcomes to address knowledge gaps.
Oncoceutics leerink global healthcare 2015oncoceutics
- Oncoceutics has discovered a novel class of compounds called ONC201 that shows compelling efficacy against aggressive and refractory tumors in preclinical studies.
- Phase I/II clinical trials are underway at leading cancer centers to evaluate ONC201's safety and efficacy in hematological malignancies and solid tumors.
- ONC201 engages multiple critical cancer pathways without toxicity, demonstrating potential as a first-in-class therapeutic for treatment-resistant cancers.
Inotuzumab ozogamicin is an antibody-drug conjugate being studied for the treatment of acute lymphoblastic leukemia (ALL) in adults. The INO-VATE ALL study compared inotuzumab ozogamicin to standard chemotherapy for adults with relapsed or refractory ALL. The primary objectives were to demonstrate higher rates of complete remission and longer overall survival with inotuzumab ozogamicin. Complete remission, safety, and overall survival were evaluated. If shown to be effective, inotuzumab ozogamicin could provide a new treatment option for adults with relapsed/refractory ALL.
After the intravenous transplantation of MSCs, a significant population of cells accumulates in the lung, which they alongside immunomodulatory effect could protect alveolar epithelial cells, reclaim the pulmonary microenvironment, prevent pulmonary fibrosis, and cure lung dysfunction. The fact that the transplantation of MSCs improved the outcome of COVID-2019 patients may be due to regulating inflammatory response and promoting tissue repair and regeneration. This is a preliminary report of our study in Iran.
This document provides an overview of a proposed new drug called Regenozene to treat multiple sclerosis (MS) by inhibiting Death Receptor 6 (DR-6). The drug aims to promote remyelination of neurons in MS patients. If successful, Regenozene could potentially stop relapses and reverse disability from MS, addressing major unmet needs. The document outlines the pathogenesis of MS, Regenozene's proposed mechanism of action, target product profile, development plan through clinical trials and regulatory approval, manufacturing and intellectual property considerations, market analysis and financial projections. The virtual business model aims to minimize risk through partnerships while maintaining control over development.
Based in Ann Arbor, Michigan, Zomedica is a veterinary health company creating diagnostic and therapeutic products for horses, dogs, and cats by focusing on the unmet needs of clinical veterinarians. With modest cash burn and a strong balance sheet, including $142.4 million cash and cash equivalents as of June 30, 2023, Zomedica is well-positioned to fund both organic growth and acquisitions.
Docola has developed a healthcare communication platform that utilizes asynchronous telehealth to deliver patient education and support. Their proprietary platform currently has over 55,000 patient users and over 1,100 clinician users. Docola seeks to raise up to $500,000 through a convertible note to fund working capital, research and development, and costs associated with an upcoming IPO.
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This document summarizes research on CLN3-Batten disease, a rare neurodegenerative lysosomal storage disorder. It describes a natural history study and biorepository at the National Institutes of Health to gather clinical data and biosamples from well-characterized patients over time and across institutions. The study aims to identify blood-based biomarkers for diagnosis and disease monitoring, clinically meaningful outcomes measures, and advance collaborative research efforts between patients, clinicians, and researchers to develop therapies for this currently untreatable disease.
ACT is conducting three clinical trials for dry age-related macular degeneration (AMD) and Stargardt's disease (SMD) using retinal pigment epithelium (RPE) cells derived from human embryonic stem cells. The trials have shown no adverse events and persistence of the transplanted cells, with functional vision improvements in most patients. ACT has additional clinical programs planned or underway for myopic macular degeneration and has a pipeline of other ophthalmology and regenerative medicine programs. It has strong intellectual property around RPE cell production and therapy. Upcoming milestones include further patient follow-up data and the potential start of Phase II trials. ACT is led by an experienced management team and board of directors.
The document provides information on CEL-SCI Corporation and their investigational cancer immunotherapy drug Multikine, including details on Multikine's mechanism of action, Phase I-II safety profile, "proof of concept" Phase II results, the ongoing pivotal Phase III clinical trial design in head and neck cancer patients, and the potential initial target market if approved. It summarizes key findings from early clinical studies that support Multikine's potential as an effective immunotherapy treatment when administered prior to standard therapies.
- The document is an investor presentation for IntelGenx Corp from April 2018.
- It provides an overview of IntelGenx, including its business strategies, product pipeline, clinical trial results, manufacturing facilities, and leadership team.
- The presentation highlights IntelGenx's drug delivery technology platforms, focus on developing generic and repurposed products, and partnerships with pharmaceutical companies.
This document discusses post-intensive care syndrome (PICS), a condition experienced by many survivors of critical illness involving new or worsening impairments in physical, cognitive, or mental health status after leaving the ICU. It provides an overview of PICS, noting that evidence in recent decades has shown PICS to be a significant problem. The document discusses how certain evidence-based ICU interventions like maintaining light sedation levels and early mobilization may help reduce risks for PICS. It promotes upcoming issues of the publication that will provide more information on PICS and the roles of various professionals in addressing it.
Bellus corporate presentation march 23BellusHealth
This corporate presentation by Roberto Bellini, President and CEO of BELLUS Health Inc (TSX: BLU), provides an overview of the company and its rare disease drug pipeline. The lead product candidate KIACTA has shown positive results in a Phase II/III trial for AA amyloidosis, a rare and deadly kidney disease with no approved treatments. BELLUS plans to complete the ongoing Phase III confirmatory trial in 2016 to support regulatory approval. The company is also developing KIACTA for sarcoidosis and has a second rare disease candidate, Shigamab, in preclinical testing for STEC related hemolytic uremic syndrome.
The document discusses drug repurposing for rare diseases. Drug repurposing involves finding new uses for existing drugs to treat different patient populations. The document provides examples of repurposing drugs to treat rare conditions like Wolfram syndrome and CDKL5. It also discusses using 'omics data to identify repurposing opportunities and describes a potential social impact bond model to fund repurposing clinical trials through healthcare savings.
Nature medicine top ten advancements in biomedicine in 2020DoriaFang
In the field of biomedicine, which is closely related to human health, many innovative therapies have matured this year and promoted medical progress. Recently, the authoritative academic journal "Nature Medicine" published a series of articles reviewing ten remarkable advancements in 2020.
Correlation between Demographic, Socio-economic, and Cancer-Specific Factors with Quality of Life Scores among Newly-Diagnosed Cancer Patients of the Medical Oncology Clinics of the Philippine General Hospital Cancer Institute
https://www.actamedicaphilippina.org/issue/1102
This corporate presentation discusses BELLUS Health's focus on developing drugs for rare diseases. Their lead product candidate, KIACTA, is in Phase 3 trials for AA amyloidosis, a rare and deadly kidney disease with no existing treatment. Positive results from a previous Phase 3 trial showed KIACTA slowed kidney function decline. If successful, KIACTA could achieve premium pricing compared to other orphan drugs. BELLUS is also developing treatments for other rare diseases including sHUS and AL amyloidosis through clinical trials and partnerships.
- Neogenix Oncology announced that members of its scientific staff will present preclinical studies of NEO-101, a monoclonal antibody being developed to treat pancreatic and colorectal cancers, at the AACR annual meeting in April 2011.
- Radient Pharmaceuticals announced preliminary results from a clinical study showing its cancer diagnostic test, Onko-Sure, had a statistical advantage over CEA in detecting early-stage colorectal cancer.
- Cephalon Inc made an offer to acquire the remaining 72.48% stake in ChemGenex Pharmaceuticals it does not already own for approximately $140 million Australian dollars in cash.
Rare Diseases: A Report on Orphan Drugs in the PipelinePhRMA
Rare diseases, when taken together, are not that rare at all. In fact, according to the National Institutes of Health (NIH), 30 million Americans have one of the nearly 7,000 diseases that are officially deemed “rare” because alone they each affect fewer than 200,000 people in the United States.
This document summarizes a webinar on building Canada's strategy for access to rare drugs. Day 1 focuses on challenges to access. Dr. Cheryl Rockman-Greenberg discusses her work on hypophosphatasia (HPP), a rare bone disease. She outlines the different forms of HPP and clinical studies of asfotase alfa treatment. Asfotase alfa was approved in Canada for pediatric HPP but access challenges remain for adults. Real-world evidence is needed to understand natural history and inform treatment criteria for adults. The HPP patient registry collects global data on treatment effects, safety and outcomes to address knowledge gaps.
Oncoceutics leerink global healthcare 2015oncoceutics
- Oncoceutics has discovered a novel class of compounds called ONC201 that shows compelling efficacy against aggressive and refractory tumors in preclinical studies.
- Phase I/II clinical trials are underway at leading cancer centers to evaluate ONC201's safety and efficacy in hematological malignancies and solid tumors.
- ONC201 engages multiple critical cancer pathways without toxicity, demonstrating potential as a first-in-class therapeutic for treatment-resistant cancers.
Inotuzumab ozogamicin is an antibody-drug conjugate being studied for the treatment of acute lymphoblastic leukemia (ALL) in adults. The INO-VATE ALL study compared inotuzumab ozogamicin to standard chemotherapy for adults with relapsed or refractory ALL. The primary objectives were to demonstrate higher rates of complete remission and longer overall survival with inotuzumab ozogamicin. Complete remission, safety, and overall survival were evaluated. If shown to be effective, inotuzumab ozogamicin could provide a new treatment option for adults with relapsed/refractory ALL.
After the intravenous transplantation of MSCs, a significant population of cells accumulates in the lung, which they alongside immunomodulatory effect could protect alveolar epithelial cells, reclaim the pulmonary microenvironment, prevent pulmonary fibrosis, and cure lung dysfunction. The fact that the transplantation of MSCs improved the outcome of COVID-2019 patients may be due to regulating inflammatory response and promoting tissue repair and regeneration. This is a preliminary report of our study in Iran.
This document provides an overview of a proposed new drug called Regenozene to treat multiple sclerosis (MS) by inhibiting Death Receptor 6 (DR-6). The drug aims to promote remyelination of neurons in MS patients. If successful, Regenozene could potentially stop relapses and reverse disability from MS, addressing major unmet needs. The document outlines the pathogenesis of MS, Regenozene's proposed mechanism of action, target product profile, development plan through clinical trials and regulatory approval, manufacturing and intellectual property considerations, market analysis and financial projections. The virtual business model aims to minimize risk through partnerships while maintaining control over development.
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Based in Ann Arbor, Michigan, Zomedica is a veterinary health company creating diagnostic and therapeutic products for horses, dogs, and cats by focusing on the unmet needs of clinical veterinarians. With modest cash burn and a strong balance sheet, including $142.4 million cash and cash equivalents as of June 30, 2023, Zomedica is well-positioned to fund both organic growth and acquisitions.
Docola has developed a healthcare communication platform that utilizes asynchronous telehealth to deliver patient education and support. Their proprietary platform currently has over 55,000 patient users and over 1,100 clinician users. Docola seeks to raise up to $500,000 through a convertible note to fund working capital, research and development, and costs associated with an upcoming IPO.
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Everything Blockchain builds platforms of trust for the modern enterprise and is on a mission to ensure every organization has access to the tools and platforms that enable them to manage, store, and protect data without the cost and complexity that holds them back today. The Company’s patented advances in engineering deliver the essential elements needed for real-world business use: speed, security, and efficiency. Everything Blockchain’s current business lines include: EB Advise, Build DB and EB Control.
ASP Isotope is an isotope enrichment company utilizing technology developed in South Africa over the past 20 years to enrich isotopes of elements or molecules with low atomic masses. Many of these elements are unsuitable for enrichment using traditional methods such as centrifuges. The Company’s initial focus is on producing and commercializing highly enriched isotopes for the healthcare and technology industries.
MDNA Life Sciences is a pioneer in the science of mitochondrial DNA. It’s our mission to create an extensive portfolio of proprietary tests that dramatically improve diagnosis, treatment, prognosis and monitoring. Putting an end to the unnecessary surgical procedures, pain and uncertainty that affect patients across the world.
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Lantern Pharma is an AI company transforming the cost, pace, and timeline of oncology drug discovery and development. Our proprietary AI and machine learning (ML) platform, RADR®, leverages over 25 billion oncology-focused data points and a library of 200+ advanced ML algorithms to help solve billion-dollar, real-world problems in oncology drug development. By harnessing the power of AI and with input from world-class scientific advisors and collaborators, we have accelerated the development of our growing pipeline of therapies including eleven cancer indications and an antibody-drug conjugate (ADC) program. On average, our newly developed drug programs have been advanced from initial AI insights to first-in-human clinical trials in 2-3 years and at approximately $1.0-2.0 million per program.
Sharps Technology is a medical device and pharmaceutical packaging company specializing in the development and manufacturing of innovative drug delivery systems. The Company’s product lines focus on low waste and ultra-low waste syringe technologies that incorporate both passive and active safety features. These features protect front line healthcare workers from life-threatening needle stick injuries and protect the public from needle re-use. Sharps Technology has extensive expertise in specialized prefilled syringe systems and is on track to launch this new product line in Q4 2023. The Company has a manufacturing facility in Hungary and has partnered with Nephron Pharmaceuticals to expand its manufacturing capacity in the US.
Aditxt is a biotech company developing immune monitoring and immune modulation platforms. Its AditxtScore platform can provide comprehensive immune profiles to monitor responses to pathogens, vaccines, drugs and transplants. Its Adimune platform aims to modulate the immune system to treat conditions like psoriasis, type 1 diabetes, and increase skin allograft survival. The company is working to develop, operate and commercialize these platforms. It currently generates revenue from immune monitoring tests and expects revenue from licensing deals for immune modulation programs as they advance in clinical trials towards commercialization.
1847 Holdings LLC, a publicly traded diversified acquisition holding company, was founded by Ellery W. Roberts, a former partner of Parallel Investment Partners, Saunders Karp & Megrue and Principal of Lazard Freres Strategic Realty Investors. EFSH's investment thesis is that capital market inefficiencies have left the founders and/or stakeholders of many small business enterprises and lower-middle market businesses with limited exit options, despite the intrinsic value of their business. Given this dynamic, EFSH can consistently acquire "solid" businesses for reasonable multiples of cash flow and then deploy resources to strengthen the infrastructure and systems to improve operations. These improvements may lead to a sale or IPO of an operating subsidiary at considerably higher valuations than the purchase price (as successfully demonstrated with the mid-2020 IPO of 1847 Goedeker on the NYSE American) and/or alternatively, an operating subsidiary may be held in perpetuity and contribute to EFSH's ability to pay regular and special dividends to shareholders.
Sharps Technology is a medical device and pharmaceutical packaging company specializing in the development and manufacturing of innovative drug delivery systems. The Company’s product lines focus on low waste and ultra-low waste syringe technologies that incorporate both passive and active safety features. These features protect front line healthcare workers from life-threatening needle stick injuries and protect the public from needle re-use. Sharps Technology has extensive expertise in specialized prefilled syringe systems and is on track to launch this new product line in Q4 2023. The Company has a manufacturing facility in Hungary and has partnered with Nephron Pharmaceuticals to expand its manufacturing capacity in the US.
SPI Energy is a global renewable energy company and provider of solar storage and electric vehicle (EV) solutions that was founded in 2006 in Roseville, California and is headquartered in McClellan Park, California. The Company has three core divisions: SolarJuice which has solar wholesale distribution, as well as residential solar and roofing installation and solar module manufacturing (Solar4America & SEM Wafertech), SPI Solar and Orange Power which operates a commercial & utility solar division, and the EdisonFuture/Phoenix Motor EV division. SolarJuice is the leader in renewable energy system solutions for residential and small commercial markets and has extensive operations in the Asia Pacific and North America markets. The SPI Solar commercial & utility solar division provides a full spectrum of EPC services to third party project developers, and develops, owns and operates solar projects that sell electricity to the grid in multiple regions, including the U.S., U.K., and Europe. Phoenix Motor is a leader in medium-duty commercial electric vehicles, and is developing EV charger solutions, electric pickup trucks, electric forklifts, and other EV products. SPI maintains global operations in North America, Australia, Asia and Europe and is also targeting strategic investment opportunities in fast growing green energy industries such as battery storage, charging stations, and other EVs which leverage the Company's expertise and substantial solar cash flow.
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2. Safe Harbor Statement
Some of the information in this report relates to future events or future business and financial performance. Such statements
constitute forward-looking information within the meaning of the Private Securities Litigation Act of 1995. Such statements can
be only predictions and the actual events or results may differ from those discussed due to, among other things, the risks
described in the public filings and other publications of Cyclo Therapeutics, Inc. Forward-looking statements are identified by
words such as “anticipates”, “projects”, “expects”, “plans”, “intends”, “believes”, “estimates”, “target”, and other similar
expressions that indicate trends and future events.
The content of this report with respect to Cyclo Therapeutics, Inc. has been completed primarily from information available in
the public released by Cyclo Therapeutics, Inc. through news releases and SEC filings. Cyclo Therapeutics, Inc. uses data
from publicly available information and its accuracy has not been independently verified by Cyclo Therapeutics, Inc. Certain
summaries of scientific activities and outcomes have been condensed to aid the reader in gaining general understanding.
The information about Cyclo Therapeutics, Inc. and its subsidiaries is solely for information purposes and is not to be
construed as an offer to sell or the solicitation of an offer to buy any security in any state.
Factors that could cause the Company’s results to differ materially from those expressed in forward looking statements include
without limitation, variation in demand and the acceptance of the Company’s products and services, the frequency, magnitude
and timing of raw material price changes, general business and economic conditions beyond the Company’s control, the
consequences of competitive factors in the market place including the ability to attract and retain customers, and the
Company’s success in attracting and retaining key personnel.
Past performance does not guarantee future performance. This report is not to be copied, transmitted, displayed, distributed
(for compensation or otherwise), or altered in any way without the prior written consent of Cyclo Therapeutics, Inc.
2
3. Company Overview
Cyclo Therapeutics, a clinical-stage biotechnology company, is developing
cyclodextrin-based drugs for the treatment of disease.
• Corporate headquarters in Gainesville, FL
• Founded in 1990 as a cyclodextrin-distribution company
• Game changer: Dr. Benny Liu showed in 2006 that one version of cyclodextrins, hydroxypropyl beta
cyclodextrins, can release cholesterol from cells in animal models of Niemann-Pick Disease Type C (NPC).
NPC is a rare inherited neurodegenerative disease that affects infants, children and adults. It is caused by
an accumulation of lipids in the liver, brain and spleen.
• This discovery led directly to Cyclo providing cyclodextrins to NPC families for use in compassionate
programs. Dr. Hastings was first to administer our product to NPC patients, route of administration was
Intravenous.
• This led directly to ground-breaking work of other physicians in the US, Brazil and other countries, then
ultimately to our decision to develop our version of hydroxypropyl beta cyclodextrin, Trappsol® Cyclo™, as
a treatment for Niemann-Pick Disease Type C.
3
4. • Phase II in Europe/Israel and Phase I in U.S. - recruiting, dosing in Niemann
Pick Disease Type C (NPC)
• NPC and AD, Compassionate Use Program ongoing in multiple countries
• Granted Orphan Drug Designation (ODD) by the U.S. Food and Drug
Administration and European Medicines Agency; Rare Pediatric Disease
Designation (U.S.) and Fast Track designation (U.S.)
• Alzheimer's Disease moving towards Phase I/II
• NPC represents significant $500mm+ annual addressable market
• Alzheimer’s Disease $1 Billion+ Opportunity
Approximately $10 million invested by insiders
• Recently closed $7.4M institutional equity financing.
• Cyclodextrins are modifiable for multiple therapeutic areas
• Company is leader in Intravenous use of cyclodextrins
• ODD gives market exclusivity for 7- 12 years
• The company holds trademark, copyright and trade secret intellectual
property around the Trappsol® Cyclo™ product.
Investment Highlights
Trappsol®
Cyclo™
Active clinical programs in
Niemann Pick Disease and
Alzheimer's Disease
Large Market
Opportunity
Strong Management
Team & Board
Robust
Cyclodextrin
Portfolio & IP
4
Strong Balance
Sheet
5. Trappsol® Cyclo™
• Trappsol ® Cyclo ™ is Cyclo’s proprietary formulation of hydroxypropyl betacyclodextrin.
• HPβCD = 7 glucose molecules in a ring, modified by adding propyl groups, to enhance solubility. The inner
core of HPBCDs can make complexes with cholesterol and other molecules.
• HPβCDs widely used as excipient in products including Sporanox (broad-spectrum anti-fungal), eye drops,
and mouthwash.
5
Figure is courtesy of David Begley, Kings College
This schematic represents interaction of cylinder shaped
cyclodextrins and cholesterol in a 1:1 or 1:2 ratio
β-Cyclodextrin, R=H
HPβCD, R=OCH2CH(CH3)OH or H
6. Niemann-Pick Disease Type C:
Overview
An autosomal recessive lysosomal
storage disease
1/80,000 - 1/120,000
Live Birth Incidences
The disease is associated
with accumulation of
cholesterol in late
endosomes and
lysosomes due to loss of
normal function of the
NPC protein (NPC1 in
95% of cases, NPC2 in
5% of cases).
NPC damage can be
found in the brain, liver,
and other body tissues.
Depending on severity of
the disease, cognitive
impairment, movement
disorders, swallowing,
lung, liver and other
normal functions are
affected.
NPC is highly variable,
presenting usually in
young children, who often
do not survive into
adulthood, and also has a
later onset presentation
which leads to longer
term disability. The
disease is difficult to
diagnose and, therefore,
under-diagnosed.
6
7. Niemann-Pick Disease Type C:
Market Potential
Existing NPC
Cases
Number of
live births
Incidence
New Cases /
Year
Annual
Diagnosis
Rate
Patient
Penetration
Treatable
Cases/Year
Annual IV
Orphan Drug
Price (Avg.)
3,000* 137,000,000 **1/100,000 1,370 25% 50% 171 $404,737***
* Extrapolation from BMC Neurol. Dec 15,15(1):257) and other assumptions **Wassif, C et al. Genet Med. 2016 January ; 18
(1):41-48***LifeSci Capital., “Analysis of Orphan Drug Market”. Feb 4, 2016,
7
• Addressable market approaching $500mm
• Diagnosis capacity is improving – numbers of patients seeking treatment will increase
8. Compassionate Use/Expanded Access with
Intravenous Trappsol ® Cyclo ™
• Cyclo’s expanded access program in NPC began in 2009 with US and Brazilian patients first to use Trappsol ® Cyclo
™ intravenously, later intrathecal added in some cases. Physicians in Europe later joined the program. Some patients
now nearing the 10 year mark.
• Cyclo gathered safety and efficacy data from 20 NPC patients in 2014 – 2015 and used these data to launch formal
clinical trials for Trappsol® Cyclo™.
• We learned from expanded access programs: Favorable safety profile allowed physicians to continue to use the drug
for many years; individual patients benefited by reduction in liver size; restoration of language skills; resolution of
interstitial lung disease; improvement in fine and gross motor skills, improvement in quality of life.
• Based on these findings, Cyclo published most extensive set of case studies on expanded access use of HPBCDs to
treat NPC children and young adults, October 2019, in peer-reviewed journal Orphanet J. Rare Diseases 2019,
14:228. Hastings, Vieira, Lui… and Hrynkow.
• No safety issues, drug was well-tolerated, easy to administer.
• Moderately affected NPC patients treated with HPBCD showed slowing of disease progression.
• “Neurologic and neurocognitive benefits were seen in most patients with IV alone, independent of the
administration of IT administration.”
In Sept. 2019, FDA approved a single IND program for a pediatric NPC patient with neurologic symptoms to receive IV
Trappsol ® Cyclo™
8
9. Trappsol® Cyclo™ Phase I Study to Evaluate
Safety and Impact On Biomarkers of NPC Disease
• Niemann-Pick Disease Type C
- Confirmed diagnosis of NPC – 1
- NIH NPC Severity Score <30 and with no
more then 4 individual domains with a score of
> 3
- Age range: 18 years upwards
• Total Sites: 2 in United States
- Emmes is supporting the study with site
management and monitoring
- UCSF Benioff Children’s Hospital Oakland, CA;
and, Morristown Medical Center, Morristown, NJ
• Trial Timeline
- First patient enrollment: Q’3 17
- First patient dosed Q’3 17
- Last patient enrolled Q’4 19
- Topline date expected Q’1 20
9
United
States
Randomization 6:6 Between Dose Groups
Trappsol® Cyclo™: Bi-weekly 8 hour intravenous
treatment for a period of 14 weeks
RANDOMIZE (N=12)
Dose Group 1
1500 mg/kg
Dose Group 2
2500 mg/kg
Primary
Endpoint
• Plasma levels of Trappsol® Cyclo™
Secondary
Endpoint
• Markers of Cholesterol
metabolism/synthesis
• CSF Levels of Trappsol® Cyclo™
• hepatic and splenic morphology
• global impression of disease
Exploratory
Endpoint
• CSF biomarkers of NPC Disease
10. Trappsol® Cyclo™
Phase I/II Study to Evaluate Safety and Efficacy
10
Europe
Randomization 4:4:4 Between Dose Groups
Trappsol® Cyclo™: Bi-weekly 8 hour intravenous
treatment for a period of 48 weeks
RANDOMIZE (N=12)
Dose Group 2
2000 mg/kg
Dose Group 1
1500 mg/kg
Dose Group 3
2500 mg/kg
Primary
Endpoint
• Plasma levels of Trappsol® Cyclo™
Secondary
Endpoint
• Markers of Cholesterol
metabolism/synthesis
• CSF Levels of Trappsol® Cyclo™
• Clinical Outcomes (motor Skills,
cognition, eye movements, liver
morphology et al)
• global impression of disease
Exploratory
Endpoint
• CSF biomarkers of NPC Disease
• Niemann-Pick Disease Type C
- Confirmed diagnosis of NPC – 1
- NIH NPC Severity Score <30 and with no
more then 4 individual domains with a score of
> 3
- Age range: 2 years upwards
• Total Sites: 5-6 in 4 Countries
- UK, Sweden, Italy, Israel
- Aptus/Synteract is supporting the trial with site
management and monitoring
• Trial Timeline
- First patient enrollment: Q’2 17
- First patient dosed Q’3 17
11. Initial Data from U.S. and E.U./Israel trials
• Safety data show positive profile.
• Trappsol® Cyclo™ releases cholesterol from cells of NPC patients, allowing for cells
to normalize.
• Trappsol® Cyclo™ crosses the blood-brain-barrier following IV administration.
• One marker for NPC disease severity, lysosphingomyelin-509, shows a downward
trend with successive administration of IV Trappsol® Cyclo™. Another biomarker of
neurodegeneration, tau, trends downward in the cerebrospinal fluid. This tells us
that as Trappsol ® Cyclo™ clears cholesterol from cells, there are downstream
effects on markers of NPC disease severity.
• Clinical efficacy data are limited but encouraging. Disease specific features,
including fine motor, gait, and cognition improve in some subjects. Most patients for
which data are available either stabilized or improved in disease specific features.
11
12. Milestones – Niemann Pick trials
• Phase I trial – Completed enrollment
• Phase I/II trial – completing enrollment
• Interim Analysis: Unblind data from Europe/Israel Phase I/II trial to correlate dose
and efficacy. Under discussion with regulators.
• Design of global pivotal trial -- Underway
• Meetings with Regulators to discuss pathway to approval – Upcoming
12
13. NPC and Alzheimer’s Disease share common features:
• Neurofibrillary tangles, tau, amyloid beta plaques in brains of NPC and AD patients
• Hypercholesterolemia is a risk factor for AD
• Lysosomal dysfunction found in both NPC and AD
• Increased cellular cholesterol increases the association of an enzyme, secretase, with
the precursor protein to amyloid beta (APP), leading to increased levels of amyloid beta
• Cognitive decline
Cell and Animal models for AD studies using HPBCDs show:
• HPBCDs added to cells that over-express the precursor protein APP lowers amyloid beta
• HPBCDs given subcutaneously to a mouse that over-expresses APP:
• reduces amyloid beta by reducing cleavage of APP;
• improves memory as shown in a standard water maze test;
• reduces microgliosis;
• up-regulates genes involved in cholesterol transport and amyloid beta clearance
(as well as NPC1)
13
Links between NPC and Alzheimer’s Disease
Reference: J. Yao, et al. Neuroprotection by cyclodextrin in cell and mouse models of Alzheimer disease. J. Exp. Med. 2012, V. 209 (13):
2501-2513
14. Alzheimer’s Disease
Compassionate Use Program
• Individual IND authorized by FDA to use Trappsol® Cyclo™ in late-onset Alzheimer’s
patient, first geriatric patient to receive Trappsol® Cyclo™
• PI is award-winning, highly recognized Alzheimer’s expert, Diana Kerwin MD
• President of Kerwin Research Center, Dallas Texas
• Formerly oversaw clinical trials and research for the National Institute on Aging-funded
Cognitive Neurology and Alzheimer’s Disease Center at Northwestern University,
Chicago
• Program costs funded 100% by external organization with Cyclo providing clinical manuals
and other clinical materials, validation methodologies for biomarker analyses, and expert
input on protocol design
• Intravenous Dosing of Alzheimer’s patient on a Monthly basis began in 2018
• FDA accepted annual report which described favorable safety profile, stabilization of
disease and improvements in behavior (less volatility and improved word-finding ability).
14
18. IP & Patent Protection
• The Company has a validated, robust manufacturing process for Trappsol® Cyclo™ that includes
important trade secret steps and is proven to be scalable to commercial production.
• Trappsol® Cyclo™ has stability monitoring data to support a 48 month expiration date and will
soon be able to support 60 months.
• This includes in-use stability data for 48 hours following dispensing
• The Company holds valuable registered trademarks and copyrights including the Trappsol®
Cyclo™ brand name
• Type II Drug Master File (DMF)
• Cyclo filed a patent, “Methods for Treating Alzheimer’s Disease”, with US Patent and Trademark
Office (Oct 2018); amended application based on 1-year data (Aug 2019); and filed International
Application/Patent Cooperation Treaty (Oct 2019)
• The filing describes dosing regimens and routes of administration of hydroxypropyl beta
cyclodextrins for the treatment of Alzheimer’s Disease.
• Orphan Drug Designation for NPC allows market exclusivity for 7 and 12 years in the US and EU,
respectively.
18
19. Management Team &
Board of Directors
N. Scott Fine Chairman & CEO
Sharon H. Hrynkow, PhD Chief Scientific Officer
Jeffrey L. Tate, PhD COO, CQO, Director
Michael Lisjak
Global Head of
Regulatory Affairs
Joshua M. Fine Chief Financial Officer
C.E. “Rick” Strattan Director & Founder
Markus Sieger Lead Director
William S. Shanahan Director
F. Patrick Ostronic Director
Randall Toig, MD Director
19
20. Scientific Advisory Board
Rita Colwell, PhD Co-Chair
Distinguished Professor, U. of Maryland, former
Director National Science Foundation
Sharon Hrynkow, PhD Co-Chair Cyclo CSO & SVPMA, Neuroscientist
M. Flint Beal, MD
Professor of Neurology and Neuroscience, Weill
Cornell Medical College
Caroline Hastings, MD
Fellowship Director, Pediatric
Hematologist/Oncologist, UCSF Benioff Children’s
Hospital Oakland
Benny Liu, MD
Gastroenterologist, Alameda Health System and
UCSF Benioff Children’s Hospital Oakland
20
21. • Phase II in Europe/Israel and Phase I in U.S. - recruiting, dosing in Niemann
Pick Disease Type C (NPC)
• NPC and AD, Compassionate Use Program ongoing in multiple countries
• Granted Orphan Drug Designation (ODD) by the U.S. Food and Drug
Administration and European Medicines Agency; Rare Pediatric Disease
Designation (U.S.) and Fast Track designation (U.S.)
• Alzheimer's Disease moving towards Phase I/II
• NPC represents significant $500mm+ annual addressable market
• Alzheimer’s Disease $1 Billion+ Opportunity
Approximately $10 million invested by insiders
• Recently closed $7.4M institutional equity financing.
• Cyclodextrins are modifiable for multiple therapeutic areas
• Company is leader in Intravenous use of cyclodextrins
• ODD gives market exclusivity for 7- 12 years
• The company holds trademark, copyright and trade secret intellectual
property around the Trappsol® Cyclo™ product.
Investment Highlights
Trappsol®
Cyclo™
Active clinical programs in
Niemann Pick Disease and
Alzheimer's Disease
Large Market
Opportunity
Strong Management
Team & Board
Robust
Cyclodextrin
Portfolio & IP
21
Strong Balance
Sheet