This corporate presentation from Caladrius Biosciences outlines their pipeline of late-stage development programs focused on cell and immunotherapies. They have four active programs targeting ischemic repair using CD34+ cells and immune modulation using T regulatory cells. Their lead programs include CLBS12 for critical limb ischemia in Japan, which has received a breakthrough designation, and CLBS14-CMD for coronary microvascular dysfunction in the US. The presentation highlights the large market opportunities and compelling clinical data from prior studies supporting further development of these programs. Caladrius has an experienced management team and a strong cash position to advance multiple value-creating milestones over the next 18 months.
This corporate presentation provides an overview of Caladrius Biosciences and its pipeline. It discusses the company's two technology platforms focusing on CD34+ cells for ischemic repair and T regulatory cells for immune modulation. Key programs discussed include CLBS12 in critical limb ischemia, CLBS14-CMD in coronary microvascular dysfunction, CLBS14-RfA in refractory angina, and CLBS03 in type 1 diabetes. It highlights positive clinical data from previous studies and outlines upcoming milestones and clinical trials. The presentation positions Caladrius as financially stable with multiple value creating events expected in the next 18 months across its late-stage cell therapy programs.
Caladrius Corporate Presentation - August 2018Steve Sizer
This corporate presentation by Caladrius Biosciences outlines their late-stage therapeutics development focusing on two technology platforms: CD34+ cells for ischemic repair and T regulatory cells for immune modulation. They have three CD34+ cell therapy candidates in development for critical limb ischemia, coronary microvascular dysfunction, and refractory angina. The presentation provides details on the clinical development programs and positive results to date for these candidates. It also introduces the experienced executive team leading the company and their financial position with $50 million in cash.
Caladrius Corporate Presentation - October 2018Steve Sizer
This corporate presentation by Caladrius Biosciences provides an overview of the company and its pipeline. Key points include:
- Caladrius has two technology platforms using CD34+ cells for ischemic repair and T regulatory cells for immune modulation.
- They have a late-stage development pipeline including programs in critical limb ischemia (CLBS12), coronary microvascular dysfunction (CLBS14-CMD), and refractory angina (CLBS14-RfA).
- The presentation highlights clinical data from prior studies of CD34+ cell therapy and outlines ongoing and planned trials across the pipeline.
This corporate presentation from Caladrius Biosciences provides an overview of the company and its pipeline. Key points include:
- Caladrius is a late-stage therapeutics development company focused on four development programs using CD34+ cells for ischemic repair and T regulatory cells for immune modulation.
- The company has a strong balance sheet with $50 million in cash as of June 2018 and low operating costs, positioning it for near-term success.
- Multiple value-creating events are expected in the next 18 months, including regulatory and clinical trial milestones across the pipeline.
Big Data and Analytic Strategy for Clinical ResearchBBCR Consulting
This document discusses how big data and analytics can help simplify clinical research and make trials more cost-effective. It begins by providing context on how Henry Ford revolutionized car manufacturing using specialized machinery and standardized processes. Similarly, big data is creating a radical shift in how research is conducted by enabling the analysis of large and complex datasets. The rest of the document outlines opportunities in areas like personalized medicine, challenges like dealing with diverse and fast-changing data, and how innovation in clinical research design can help address these challenges to develop more targeted treatments.
Cytori Therapeutics is developing novel cell therapies including ECCS-50 for the treatment of hand dysfunction in scleroderma patients. Clinical trials show ECCS-50 improves patient reported outcomes like hand function and reduces symptoms over 24 months. Cytori is currently enrolling two phase 3 trials and plans to submit for FDA and EMA approval in 2018-2019. In 2016, Cytori will launch a managed access program in Europe to provide early access to ECCS-50 for patients while the company seeks full marketing authorization.
Investor cytori presentation public website 9 9 15_finalcytoriIR
Cytori Therapeutics is developing Cytori Cell Therapy, which uses a patient's own adipose-derived regenerative cells, for several indications including scleroderma. Scleroderma causes fibrosis and impaired hand function, which is a major cause of disability. Cytori has completed early clinical trials for scleroderma showing good safety and sustained improvements in hand function, pain, and quality of life. Cytori is currently enrolling patients in a Phase 3 pivotal trial in the US and plans to initiate a Phase 2/3 trial in Europe. Preclinical studies demonstrate Cytori Cell Therapy's pleiotropic mechanisms of action in reducing fibrosis.
Cytori Therapeutics provides an overview of their cell therapy technology and clinical pipeline. Their lead indication is treating hand dysfunction associated with scleroderma using their Cytori Cell Therapy, which involves harvesting a patient's own adipose tissue, processing it to isolate regenerative cells, and delivering the cells back to affected areas. They have an ongoing phase III trial in Europe for scleroderma and anticipate European introduction in 2016 and potential US approval in 2018. Their clinical pipeline also includes trials for knee osteoarthritis, urinary incontinence, and burns. A pilot study of 12 patients with scleroderma found improvements in hand function, Raynaud's scores, and other measures out to 24 months follow up
This corporate presentation provides an overview of Caladrius Biosciences and its pipeline. It discusses the company's two technology platforms focusing on CD34+ cells for ischemic repair and T regulatory cells for immune modulation. Key programs discussed include CLBS12 in critical limb ischemia, CLBS14-CMD in coronary microvascular dysfunction, CLBS14-RfA in refractory angina, and CLBS03 in type 1 diabetes. It highlights positive clinical data from previous studies and outlines upcoming milestones and clinical trials. The presentation positions Caladrius as financially stable with multiple value creating events expected in the next 18 months across its late-stage cell therapy programs.
Caladrius Corporate Presentation - August 2018Steve Sizer
This corporate presentation by Caladrius Biosciences outlines their late-stage therapeutics development focusing on two technology platforms: CD34+ cells for ischemic repair and T regulatory cells for immune modulation. They have three CD34+ cell therapy candidates in development for critical limb ischemia, coronary microvascular dysfunction, and refractory angina. The presentation provides details on the clinical development programs and positive results to date for these candidates. It also introduces the experienced executive team leading the company and their financial position with $50 million in cash.
Caladrius Corporate Presentation - October 2018Steve Sizer
This corporate presentation by Caladrius Biosciences provides an overview of the company and its pipeline. Key points include:
- Caladrius has two technology platforms using CD34+ cells for ischemic repair and T regulatory cells for immune modulation.
- They have a late-stage development pipeline including programs in critical limb ischemia (CLBS12), coronary microvascular dysfunction (CLBS14-CMD), and refractory angina (CLBS14-RfA).
- The presentation highlights clinical data from prior studies of CD34+ cell therapy and outlines ongoing and planned trials across the pipeline.
This corporate presentation from Caladrius Biosciences provides an overview of the company and its pipeline. Key points include:
- Caladrius is a late-stage therapeutics development company focused on four development programs using CD34+ cells for ischemic repair and T regulatory cells for immune modulation.
- The company has a strong balance sheet with $50 million in cash as of June 2018 and low operating costs, positioning it for near-term success.
- Multiple value-creating events are expected in the next 18 months, including regulatory and clinical trial milestones across the pipeline.
Big Data and Analytic Strategy for Clinical ResearchBBCR Consulting
This document discusses how big data and analytics can help simplify clinical research and make trials more cost-effective. It begins by providing context on how Henry Ford revolutionized car manufacturing using specialized machinery and standardized processes. Similarly, big data is creating a radical shift in how research is conducted by enabling the analysis of large and complex datasets. The rest of the document outlines opportunities in areas like personalized medicine, challenges like dealing with diverse and fast-changing data, and how innovation in clinical research design can help address these challenges to develop more targeted treatments.
Cytori Therapeutics is developing novel cell therapies including ECCS-50 for the treatment of hand dysfunction in scleroderma patients. Clinical trials show ECCS-50 improves patient reported outcomes like hand function and reduces symptoms over 24 months. Cytori is currently enrolling two phase 3 trials and plans to submit for FDA and EMA approval in 2018-2019. In 2016, Cytori will launch a managed access program in Europe to provide early access to ECCS-50 for patients while the company seeks full marketing authorization.
Investor cytori presentation public website 9 9 15_finalcytoriIR
Cytori Therapeutics is developing Cytori Cell Therapy, which uses a patient's own adipose-derived regenerative cells, for several indications including scleroderma. Scleroderma causes fibrosis and impaired hand function, which is a major cause of disability. Cytori has completed early clinical trials for scleroderma showing good safety and sustained improvements in hand function, pain, and quality of life. Cytori is currently enrolling patients in a Phase 3 pivotal trial in the US and plans to initiate a Phase 2/3 trial in Europe. Preclinical studies demonstrate Cytori Cell Therapy's pleiotropic mechanisms of action in reducing fibrosis.
Cytori Therapeutics provides an overview of their cell therapy technology and clinical pipeline. Their lead indication is treating hand dysfunction associated with scleroderma using their Cytori Cell Therapy, which involves harvesting a patient's own adipose tissue, processing it to isolate regenerative cells, and delivering the cells back to affected areas. They have an ongoing phase III trial in Europe for scleroderma and anticipate European introduction in 2016 and potential US approval in 2018. Their clinical pipeline also includes trials for knee osteoarthritis, urinary incontinence, and burns. A pilot study of 12 patients with scleroderma found improvements in hand function, Raynaud's scores, and other measures out to 24 months follow up
The presentation discussed Protalix's plant cell-expressed, chemically modified human alpha-galactosidase for the treatment of Fabry disease, called PRX-102. Fabry disease is caused by a deficiency of the enzyme alpha-galactosidase A, leading to accumulation of Gb3 substrate and increased risks of stroke, cardiomyopathy, and renal issues. Currently available treatments have short half-lives. PRX-102 aims to be a "bio-better" enzyme by using chemical modification via covalent cross-linking of the subunits to create a stable dimer, which may provide advantages like improved stability, longer half-life, and enhanced uptake in target organs, potentially leading to better clinical efficacy than
Opexa therapeutics corporate presentation october 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, an autologous T cell immunotherapy, for the treatment of multiple sclerosis (MS). A Phase 2b clinical trial of Tcelna in secondary progressive MS (SPMS) is ongoing, with top-line results expected in late 2016. Previous clinical trials of Tcelna in relapsing-remitting MS showed a 37% reduction in relapse rate compared to placebo and reversal of disability progression in more active patients. Opexa signed an agreement with Merck Serono for development and commercialization of Tcelna in MS worldwide, excluding Japan, which could provide over $220 million in milestones to Opexa.
biOasis Technologies, Inc. Presentation - October 2014graemedick
This document discusses biOasis Technologies Inc., a company developing a drug delivery platform to transport therapeutics across the blood-brain barrier (BBB) for treating central nervous system diseases. The platform utilizes melanotransferrin (MTf), a human protein that naturally transports iron into the brain. BiOasis has discovered a peptide within MTf, called MTfp, that offers improved brain penetration. Studies show MTf and MTfp conjugated to various compounds, including antibodies, enzymes and chemotherapy drugs, significantly increase transport into the brain compared to the compounds alone. BiOasis is working to validate and expand this technology with the goal of developing treatments for diseases like Alzheimer's, lysosomal storage disorders and brain cancer.
Cardium Therapeutics' (NYSE MKT: CXM) Generx® product candidate (alferminogene tadenovec, Ad5FGF-4) is a DNA-based angiogenic growth factor therapeutic being developed for the potential treatment of patients with advanced coronary artery disease. Generx is designed to stimulate the growth of supplemental collateral blood vessels in the heart in order to enhance myocardial blood flow in patients who have insufficient blood flow due to atherosclerotic plaque restricting flow in the coronary arteries that supply the heart. Generx has progressed through four randomized, placebo-controlled clinical studies at over 100 medical centers in the United States and Western Europe that have enrolled over 650 patients.
Generx is designed as a disease-modifying regenerative medicine therapeutic that can elicit structural and physiologic changes in the heart (the growth of new collateral blood vessels) following a one-time intracoronary administration from a standard cardiac infusion catheter. In contrast, traditional drug therapies such as nitrates and beta blockers provide transient symptomatic relief of anginal chest pain without changing the underlying disease. In the United States, anti-anginal drugs have traditionally been registered by the FDA based on improvements in exercise treadmill tolerance testing (ETT), which can confirm short-term symptomatic relief of angina. Based on the disease-modifying nature of Generx, Cardium believes that a more objective and appropriate measure to assess the effectiveness of angiogenic therapy is cardiac perfusion, the actual blood flow in the heart under stress using SPECT imaging (single photon emission computed tomography) or another advanced imaging technique. Cardiac perfusion directly shows and quantifies improvements in the underlying physiologic condition, i.e. insufficient blood flow under stress. Generx is currently being developed for international markets outside United States as a treatment alternative for patients who may not have access to or may not be candidates for costly and invasive surgical revascularization procedures, such as coronary artery bypass surgery and angioplasty/stents.
Learn more at: http://cardiumthx.com/generx.html
Cytori Therapeutics provides a cell therapy for the treatment of scleroderma using cells derived from a patient's own adipose tissue. Their lead indication is for the treatment of hand dysfunction in scleroderma patients. A pilot clinical trial in France showed improvements in hand function, Raynaud's symptoms, and pain out to 24 months with a single administration of the therapy. Cytori is preparing for commercial launch in the EU in 2016 and anticipates FDA approval in the US in 2018.
Art 923 rev-c-updating investor presentation on valeritas website_final_12.05.16valeritasir
V-Go is a single-use, fully disposable insulin delivery device that provides basal and bolus insulin. It addresses the needs of the 4.6 million Type 2 diabetes patients in the US who require insulin but are not achieving treatment goals. Extensive clinical data shows V-Go lowers A1C levels and total daily insulin dose. It has established reimbursement through pharmacy benefits, making it cost-neutral for payors and patients compared to insulin pens. Valeritas sees significant growth opportunities by expanding its sales force to reach more prescribers.
Cardiac resynchronization therapy (crt) devices global trends, estimates an...Research Hub
Get Full Report With Table Of Contents at
http://www.yourresearchhub.com/products/regenerative-medicine-global-trends-estimates-and-forecasts-2013-2019
Opexa Therapeutics is developing Precision Immunotherapy using their T-cell platform to treat autoimmune diseases like multiple sclerosis (MS). Their lead product, Tcelna, is in Phase II clinical trials for secondary progressive MS (SPMS), which affects around 30-45% of MS patients. Tcelna works by programming the immune system to specifically recognize and inhibit myelin reactive T-cells that cause damage to the protective myelin sheath surrounding nerves. If successful, Tcelna could be the first approved treatment for SPMS, representing a potential $7 billion market in North America alone. Opexa has an option agreement with Merck for the development of Tcelna in MS.
Opexa therapeutics corporate presentation november 21OpexaTherapeutics
Opexa Therapeutics is developing Precision Immunotherapy using their T-cell platform to treat autoimmune diseases like multiple sclerosis. Their lead product Tcelna is in Phase 2 clinical trials for secondary progressive multiple sclerosis, a later stage form of MS with limited treatment options. Tcelna works by specifically targeting and reducing myelin reactive T-cells that cause damage to the protective myelin sheath surrounding nerves. If successful, Tcelna has the potential to be the first treatment approved for secondary progressive MS and generate over $7 billion in annual sales. Opexa has an option agreement with Merck for the development of Tcelna in MS.
Opexa Therapeutics is developing Tcelna, an autologous T-cell immunotherapy, for the treatment of secondary progressive multiple sclerosis (SPMS). They are currently conducting a Phase IIb clinical trial of Tcelna in SPMS patients. Preliminary data from earlier trials in both relapsing-remitting and SPMS patients showed signs of efficacy, including reduced disability progression, brain atrophy, and relapse rates. If successful, Tcelna has the potential to be the first approved treatment specifically for SPMS, an underserved market with no approved therapies. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $
Opexa Therapeutics is developing Precision Immunotherapy using Tcelna to treat multiple sclerosis (MS). Tcelna is an antigen-specific T-cell immunotherapy that targets myelin-reactive T-cells that cause damage in MS. It is currently in a Phase IIb clinical trial called Abili-T for secondary progressive MS (SPMS), which has limited treatment options. Previous clinical trials of Tcelna showed reductions in brain atrophy, disability progression, and relapse rates in SPMS patients. If successful, Abili-T could support Tcelna becoming the first approved treatment for SPMS.
Opexa Therapeutics is developing Precision Immunotherapy using Tcelna to treat multiple sclerosis (MS). Tcelna is an antigen specific T-cell immunotherapy that targets myelin reactive T-cells (MRTCs) that cause damage in MS. Opexa is currently conducting a Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) called Abili-T. Previous clinical trials of Tcelna showed promising results in reducing brain atrophy, disability progression, and relapse rates in MS patients. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS, which could provide up to $220 million in payments if milestones are
10 the importance of audit to monitor applications of procedures and improve ...NPSAIC
This document discusses the importance of auditing primary percutaneous coronary intervention (PPCI) programs in the UK to monitor quality and drive improvements. It outlines two national audit projects - MINAP and BCIS - that allow electronic collection of data on all PPCI patients. Regular feedback is provided to hospitals on process measures like door-to-balloon times and outcomes. This reporting has been associated with steady quality improvements and reduced variations in care across centers. The audits find that while PPCI activity and outcomes have improved in recent years, there remains room for further enhancements to ensure standards met in clinical trials can be achieved in routine practice.
Ascendis Pharma is a biopharmaceutical company developing its TransCon technology to create long-acting prodrug therapies. It has three drug candidates in clinical trials for growth hormone deficiency, hypoparathyroidism, and achondroplasia. Its lead candidate, TransCon hGH, is in Phase 3 trials for growth hormone deficiency and top-line data is expected in Q1 2019. Analysts have rated Ascendis Pharma stock a "Buy" based on the potential of its TransCon technology to improve treatment outcomes for multiple rare diseases.
Aptorum Group Limited (NASDAQ: APM) is a pharmaceutical company dedicated to
developing and commercializing novel therapeutics to tackle unmet medical needs.
Aptorum's current drug pipeline includes indications in orphan diseases, infectious
diseases, and metabolic diseases. In 2021, the company plans to bring two candidates to
clinical trials. Aptorum’s Smart-ACT™ platform is designed to bring an average of three
drug candidates for orphan diseases to clinical trials every 12-18 months. The company is
now preparing to launch a dietary supplement for women undergoing menopause and
experiencing related symptoms, including osteoporosis. Targeting a global woman’s health
supplement market that is expected to reach $17 billion in 2025, Aptorum is expected to
generate near-term revenue with significant long-term growth potential.
1) The study evaluated the use of wearable cardioverter defibrillators (WCDs) and subsequent cardiac procedures in 1,199 patients based on administrative claims data. 2) 38% of WCD users received an implantable cardioverter defibrillator (ICD) within 60 days, while 62% did not receive an ICD in that period. 3) Evaluation of patients without an ICD in the first 60 days found that approximately half (47%) did not undergo any additional diagnostic or intervention procedures in the following 240 days.
Moore Expedited Devel & Rev Of New Drugs 2010 (L)jmoore89
This document summarizes strategic regulatory options for expediting drug development and approval through the FDA. It discusses expedited review pathways including Subpart E for life-threatening diseases and Subpart H (accelerated approval) for serious or life-threatening conditions. Key elements covered are early consultation, use of surrogate endpoints in phase 2 trials, post-approval studies, and consideration of disease severity and treatment alternatives in the risk-benefit assessment. The goal is to provide patients sooner access to promising new therapies while maintaining adequate evidence of safety and efficacy.
This document summarizes a proposed gene therapy treatment for Hemophilia B called HemB. Key points:
- Hemophilia B is an X-linked bleeding disorder caused by a lack of coagulation Factor IX. Current treatments are invasive and costly.
- HemB uses an AAV8 vector to deliver a normal Factor IX gene via a single intravenous infusion, allowing long-term expression of Factor IX at therapeutic levels to reduce bleeding episodes.
- Phase I trials showed HemB increased Factor IX levels in patients from <1% to 1-6% of normal with no significant safety issues. A Phase II trial will enroll 60 patients to further evaluate efficacy.
- If approved, HemB could
This corporate presentation by Caladrius Biosciences provides an overview of the company and its pipeline. Key points include:
- Caladrius has two technology platforms using CD34+ cells for ischemic repair and T regulatory cells for immune modulation.
- They have a late-stage development pipeline including programs in critical limb ischemia (CLBS12), coronary microvascular dysfunction (CLBS14-CMD), and refractory angina (CLBS14-RfA).
- The presentation highlights clinical data from prior studies of CD34+ cell therapy and outlines ongoing and planned trials across the pipeline.
- Caladrius has an experienced management team and a strong financial position to support advancing its programs toward value
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.
Cytori Therapeutics is developing adipose-derived regenerative cell therapies using a point-of-care device platform. The platform allows for autologous cell therapies to be prepared at the bedside from a patient's own fat tissue. Clinical trials show the cells are safe and may provide benefits in cardiac and soft tissue applications. Near-term value drivers include government contract milestones and cardiovascular trial data. The business model involves selling single-use consumables for each procedure at price points around $2,000-$12,000, depending on the indication.
Cytori Therapeutics is presenting at the 26th Annual Roth Conference. They provide an overview of their adipose-derived cell therapy technology and development pipeline. Their focus is on cardiovascular disease and soft tissue injuries. Key highlights include their ongoing Phase II U.S. heart failure trial, a U.S. government contract for up to $106 million to develop treatment for thermal burns, and a pipeline supported by independent clinical studies of additional indications. They discuss their commercial model of selling proprietary cell therapy devices and single-use consumables, as well as progress on strategic partnerships.
The presentation discussed Protalix's plant cell-expressed, chemically modified human alpha-galactosidase for the treatment of Fabry disease, called PRX-102. Fabry disease is caused by a deficiency of the enzyme alpha-galactosidase A, leading to accumulation of Gb3 substrate and increased risks of stroke, cardiomyopathy, and renal issues. Currently available treatments have short half-lives. PRX-102 aims to be a "bio-better" enzyme by using chemical modification via covalent cross-linking of the subunits to create a stable dimer, which may provide advantages like improved stability, longer half-life, and enhanced uptake in target organs, potentially leading to better clinical efficacy than
Opexa therapeutics corporate presentation october 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, an autologous T cell immunotherapy, for the treatment of multiple sclerosis (MS). A Phase 2b clinical trial of Tcelna in secondary progressive MS (SPMS) is ongoing, with top-line results expected in late 2016. Previous clinical trials of Tcelna in relapsing-remitting MS showed a 37% reduction in relapse rate compared to placebo and reversal of disability progression in more active patients. Opexa signed an agreement with Merck Serono for development and commercialization of Tcelna in MS worldwide, excluding Japan, which could provide over $220 million in milestones to Opexa.
biOasis Technologies, Inc. Presentation - October 2014graemedick
This document discusses biOasis Technologies Inc., a company developing a drug delivery platform to transport therapeutics across the blood-brain barrier (BBB) for treating central nervous system diseases. The platform utilizes melanotransferrin (MTf), a human protein that naturally transports iron into the brain. BiOasis has discovered a peptide within MTf, called MTfp, that offers improved brain penetration. Studies show MTf and MTfp conjugated to various compounds, including antibodies, enzymes and chemotherapy drugs, significantly increase transport into the brain compared to the compounds alone. BiOasis is working to validate and expand this technology with the goal of developing treatments for diseases like Alzheimer's, lysosomal storage disorders and brain cancer.
Cardium Therapeutics' (NYSE MKT: CXM) Generx® product candidate (alferminogene tadenovec, Ad5FGF-4) is a DNA-based angiogenic growth factor therapeutic being developed for the potential treatment of patients with advanced coronary artery disease. Generx is designed to stimulate the growth of supplemental collateral blood vessels in the heart in order to enhance myocardial blood flow in patients who have insufficient blood flow due to atherosclerotic plaque restricting flow in the coronary arteries that supply the heart. Generx has progressed through four randomized, placebo-controlled clinical studies at over 100 medical centers in the United States and Western Europe that have enrolled over 650 patients.
Generx is designed as a disease-modifying regenerative medicine therapeutic that can elicit structural and physiologic changes in the heart (the growth of new collateral blood vessels) following a one-time intracoronary administration from a standard cardiac infusion catheter. In contrast, traditional drug therapies such as nitrates and beta blockers provide transient symptomatic relief of anginal chest pain without changing the underlying disease. In the United States, anti-anginal drugs have traditionally been registered by the FDA based on improvements in exercise treadmill tolerance testing (ETT), which can confirm short-term symptomatic relief of angina. Based on the disease-modifying nature of Generx, Cardium believes that a more objective and appropriate measure to assess the effectiveness of angiogenic therapy is cardiac perfusion, the actual blood flow in the heart under stress using SPECT imaging (single photon emission computed tomography) or another advanced imaging technique. Cardiac perfusion directly shows and quantifies improvements in the underlying physiologic condition, i.e. insufficient blood flow under stress. Generx is currently being developed for international markets outside United States as a treatment alternative for patients who may not have access to or may not be candidates for costly and invasive surgical revascularization procedures, such as coronary artery bypass surgery and angioplasty/stents.
Learn more at: http://cardiumthx.com/generx.html
Cytori Therapeutics provides a cell therapy for the treatment of scleroderma using cells derived from a patient's own adipose tissue. Their lead indication is for the treatment of hand dysfunction in scleroderma patients. A pilot clinical trial in France showed improvements in hand function, Raynaud's symptoms, and pain out to 24 months with a single administration of the therapy. Cytori is preparing for commercial launch in the EU in 2016 and anticipates FDA approval in the US in 2018.
Art 923 rev-c-updating investor presentation on valeritas website_final_12.05.16valeritasir
V-Go is a single-use, fully disposable insulin delivery device that provides basal and bolus insulin. It addresses the needs of the 4.6 million Type 2 diabetes patients in the US who require insulin but are not achieving treatment goals. Extensive clinical data shows V-Go lowers A1C levels and total daily insulin dose. It has established reimbursement through pharmacy benefits, making it cost-neutral for payors and patients compared to insulin pens. Valeritas sees significant growth opportunities by expanding its sales force to reach more prescribers.
Cardiac resynchronization therapy (crt) devices global trends, estimates an...Research Hub
Get Full Report With Table Of Contents at
http://www.yourresearchhub.com/products/regenerative-medicine-global-trends-estimates-and-forecasts-2013-2019
Opexa Therapeutics is developing Precision Immunotherapy using their T-cell platform to treat autoimmune diseases like multiple sclerosis (MS). Their lead product, Tcelna, is in Phase II clinical trials for secondary progressive MS (SPMS), which affects around 30-45% of MS patients. Tcelna works by programming the immune system to specifically recognize and inhibit myelin reactive T-cells that cause damage to the protective myelin sheath surrounding nerves. If successful, Tcelna could be the first approved treatment for SPMS, representing a potential $7 billion market in North America alone. Opexa has an option agreement with Merck for the development of Tcelna in MS.
Opexa therapeutics corporate presentation november 21OpexaTherapeutics
Opexa Therapeutics is developing Precision Immunotherapy using their T-cell platform to treat autoimmune diseases like multiple sclerosis. Their lead product Tcelna is in Phase 2 clinical trials for secondary progressive multiple sclerosis, a later stage form of MS with limited treatment options. Tcelna works by specifically targeting and reducing myelin reactive T-cells that cause damage to the protective myelin sheath surrounding nerves. If successful, Tcelna has the potential to be the first treatment approved for secondary progressive MS and generate over $7 billion in annual sales. Opexa has an option agreement with Merck for the development of Tcelna in MS.
Opexa Therapeutics is developing Tcelna, an autologous T-cell immunotherapy, for the treatment of secondary progressive multiple sclerosis (SPMS). They are currently conducting a Phase IIb clinical trial of Tcelna in SPMS patients. Preliminary data from earlier trials in both relapsing-remitting and SPMS patients showed signs of efficacy, including reduced disability progression, brain atrophy, and relapse rates. If successful, Tcelna has the potential to be the first approved treatment specifically for SPMS, an underserved market with no approved therapies. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $
Opexa Therapeutics is developing Precision Immunotherapy using Tcelna to treat multiple sclerosis (MS). Tcelna is an antigen-specific T-cell immunotherapy that targets myelin-reactive T-cells that cause damage in MS. It is currently in a Phase IIb clinical trial called Abili-T for secondary progressive MS (SPMS), which has limited treatment options. Previous clinical trials of Tcelna showed reductions in brain atrophy, disability progression, and relapse rates in SPMS patients. If successful, Abili-T could support Tcelna becoming the first approved treatment for SPMS.
Opexa Therapeutics is developing Precision Immunotherapy using Tcelna to treat multiple sclerosis (MS). Tcelna is an antigen specific T-cell immunotherapy that targets myelin reactive T-cells (MRTCs) that cause damage in MS. Opexa is currently conducting a Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) called Abili-T. Previous clinical trials of Tcelna showed promising results in reducing brain atrophy, disability progression, and relapse rates in MS patients. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS, which could provide up to $220 million in payments if milestones are
10 the importance of audit to monitor applications of procedures and improve ...NPSAIC
This document discusses the importance of auditing primary percutaneous coronary intervention (PPCI) programs in the UK to monitor quality and drive improvements. It outlines two national audit projects - MINAP and BCIS - that allow electronic collection of data on all PPCI patients. Regular feedback is provided to hospitals on process measures like door-to-balloon times and outcomes. This reporting has been associated with steady quality improvements and reduced variations in care across centers. The audits find that while PPCI activity and outcomes have improved in recent years, there remains room for further enhancements to ensure standards met in clinical trials can be achieved in routine practice.
Ascendis Pharma is a biopharmaceutical company developing its TransCon technology to create long-acting prodrug therapies. It has three drug candidates in clinical trials for growth hormone deficiency, hypoparathyroidism, and achondroplasia. Its lead candidate, TransCon hGH, is in Phase 3 trials for growth hormone deficiency and top-line data is expected in Q1 2019. Analysts have rated Ascendis Pharma stock a "Buy" based on the potential of its TransCon technology to improve treatment outcomes for multiple rare diseases.
Aptorum Group Limited (NASDAQ: APM) is a pharmaceutical company dedicated to
developing and commercializing novel therapeutics to tackle unmet medical needs.
Aptorum's current drug pipeline includes indications in orphan diseases, infectious
diseases, and metabolic diseases. In 2021, the company plans to bring two candidates to
clinical trials. Aptorum’s Smart-ACT™ platform is designed to bring an average of three
drug candidates for orphan diseases to clinical trials every 12-18 months. The company is
now preparing to launch a dietary supplement for women undergoing menopause and
experiencing related symptoms, including osteoporosis. Targeting a global woman’s health
supplement market that is expected to reach $17 billion in 2025, Aptorum is expected to
generate near-term revenue with significant long-term growth potential.
1) The study evaluated the use of wearable cardioverter defibrillators (WCDs) and subsequent cardiac procedures in 1,199 patients based on administrative claims data. 2) 38% of WCD users received an implantable cardioverter defibrillator (ICD) within 60 days, while 62% did not receive an ICD in that period. 3) Evaluation of patients without an ICD in the first 60 days found that approximately half (47%) did not undergo any additional diagnostic or intervention procedures in the following 240 days.
Moore Expedited Devel & Rev Of New Drugs 2010 (L)jmoore89
This document summarizes strategic regulatory options for expediting drug development and approval through the FDA. It discusses expedited review pathways including Subpart E for life-threatening diseases and Subpart H (accelerated approval) for serious or life-threatening conditions. Key elements covered are early consultation, use of surrogate endpoints in phase 2 trials, post-approval studies, and consideration of disease severity and treatment alternatives in the risk-benefit assessment. The goal is to provide patients sooner access to promising new therapies while maintaining adequate evidence of safety and efficacy.
This document summarizes a proposed gene therapy treatment for Hemophilia B called HemB. Key points:
- Hemophilia B is an X-linked bleeding disorder caused by a lack of coagulation Factor IX. Current treatments are invasive and costly.
- HemB uses an AAV8 vector to deliver a normal Factor IX gene via a single intravenous infusion, allowing long-term expression of Factor IX at therapeutic levels to reduce bleeding episodes.
- Phase I trials showed HemB increased Factor IX levels in patients from <1% to 1-6% of normal with no significant safety issues. A Phase II trial will enroll 60 patients to further evaluate efficacy.
- If approved, HemB could
This corporate presentation by Caladrius Biosciences provides an overview of the company and its pipeline. Key points include:
- Caladrius has two technology platforms using CD34+ cells for ischemic repair and T regulatory cells for immune modulation.
- They have a late-stage development pipeline including programs in critical limb ischemia (CLBS12), coronary microvascular dysfunction (CLBS14-CMD), and refractory angina (CLBS14-RfA).
- The presentation highlights clinical data from prior studies of CD34+ cell therapy and outlines ongoing and planned trials across the pipeline.
- Caladrius has an experienced management team and a strong financial position to support advancing its programs toward value
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.
Cytori Therapeutics is developing adipose-derived regenerative cell therapies using a point-of-care device platform. The platform allows for autologous cell therapies to be prepared at the bedside from a patient's own fat tissue. Clinical trials show the cells are safe and may provide benefits in cardiac and soft tissue applications. Near-term value drivers include government contract milestones and cardiovascular trial data. The business model involves selling single-use consumables for each procedure at price points around $2,000-$12,000, depending on the indication.
Cytori Therapeutics is presenting at the 26th Annual Roth Conference. They provide an overview of their adipose-derived cell therapy technology and development pipeline. Their focus is on cardiovascular disease and soft tissue injuries. Key highlights include their ongoing Phase II U.S. heart failure trial, a U.S. government contract for up to $106 million to develop treatment for thermal burns, and a pipeline supported by independent clinical studies of additional indications. They discuss their commercial model of selling proprietary cell therapy devices and single-use consumables, as well as progress on strategic partnerships.
IntelGenx is an innovative pharmaceutical company focused on oral thin film drug delivery technologies. They have developed a proprietary oral thin film technology platform called VersaFilm that can be used to improve existing drugs or develop new products. Their pipeline includes several product candidates targeting large markets like migraines, erectile dysfunction, and brain degenerative diseases. They have state-of-the-art manufacturing facilities and strategic partnerships to commercialize their products globally.
Cytori Therapeutics provided a corporate update in March 2015. The document discusses Cytori's recent clinical trial progress, including receiving funding from BARDA for a thermal burn injury trial and approval to initiate a Phase III pivotal trial for scleroderma hand dysfunction. It also summarizes recent clinical data from a pilot study of ECCS-50 for scleroderma, which showed improvements in hand function, pain, and quality of life. Cytori is focused on advancing its cell therapy pipeline, with ongoing trials for knee osteoarthritis and plans to initiate additional trials in 2015.
This corporate presentation provides an overview of Exact Sciences' colorectal cancer screening test Cologuard and highlights its fourth quarter 2017 performance. Key points include: Cologuard revenue grew significantly in 2017 due to expanded insurance coverage, improved reimbursement rates, and increased marketing efforts. The presentation also outlines Exact Sciences' strategy to continue growing market share and penetration by engaging key audiences like patients, providers, and payers. The goal is to make Cologuard the standard of care for colorectal cancer screening in the United States.
Cytori Therapeutics provided an investor update and overview of their clinical pipeline focusing on scleroderma. They are initiating a Phase 3 trial in the US for scleroderma-associated hand dysfunction in 2015 based on positive results from previous pilot studies. Pilot studies of ECCS-50 cell therapy demonstrated improvements in hand function, pain, vascular outcomes, and ulcer healing for patients with scleroderma. Additionally, a Phase 2/3 trial is pending approval in Europe and clinical trials are ongoing or planned in other indications such as heart failure and burns, some receiving external funding.
Cytori Therapeutics provides an investor update on their autologous cell therapy platform and clinical pipeline. They are initiating a Phase 3 trial in the US for scleroderma-associated hand dysfunction in 2015 based on promising pilot data showing improved hand function, pain, and quality of life. Additionally, a Phase 2/3 trial is planned in the EU. Cytori is also developing cell therapies for knee osteoarthritis and other indications.
The document discusses liquid biopsies and next generation cancer molecular diagnostics. It summarizes that OncoCyte Corporation is focused on developing diagnostic tests for early cancer detection using liquid biopsies, with an initial focus on tests for lung cancer. Key points include that lung cancer diagnostics represents a large market opportunity and that OncoCyte's preliminary lung cancer diagnostic test shows strong performance in clinical trials with high sensitivity and specificity. The test has the potential to reduce risky follow-up procedures for patients and provide significant healthcare cost savings.
Exas september 2017 corporate presentation finalExact Sciences
Exact Sciences presented their corporate strategy in September 2017. They discussed their mission to help eradicate colon cancer by partnering with various stakeholders. They highlighted that Cologuard addresses the challenges of colon cancer as a non-invasive screening test with high early-stage cancer detection rates. Exact Sciences also reviewed Cologuard's growing adoption driven by their commercial strategies like marketing, salesforce, and increasing insurance coverage.
Exas july 2017 corporate presentation finalExact Sciences
This corporate presentation discusses Exact Sciences' mission to help eradicate colon cancer through early detection. It summarizes the company's Cologuard test, which detects colorectal cancer through a non-invasive stool DNA test. The presentation outlines Cologuard's clinical validation and success in increasing early cancer detection rates and patient compliance with screening. It also reviews Exact Sciences' commercial strategy, growth opportunities, and expanding pipeline of non-invasive liquid biopsy tests to detect additional cancers.
Exas august 2017 corporate presentation finalExact Sciences
This corporate presentation discusses Exact Sciences' mission to help eradicate colon cancer through early detection. It summarizes the company's Cologuard test, which detects colorectal cancer through a non-invasive stool DNA test. The presentation outlines Cologuard's clinical validation and notes it is included in major guidelines. It also reviews Exact Sciences' commercial strategy of increasing awareness, access, and compliance through a sales force, marketing campaigns, and focus on improving health outcomes. Financial results for the second quarter of 2017 show increased revenue and cash on hand compared to the prior quarter.
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date. Piclidenoson received approval for COVID-19 clinical trial in Israel in April 2020 and is expected to file its IND in the US in the near-term.
3.31.17 exas april 2017 corporate presentationExact Sciences
This corporate presentation discusses Exact Sciences' mission to help eradicate colon cancer by partnering with various stakeholders. It summarizes the company's Cologuard test, which non-invasively screens for colorectal cancer with high sensitivity. Cologuard saw strong growth in 2016 and the presentation outlines Exact Sciences' commercial strategy and pipeline expansion into liquid biopsy tests for additional cancer types in collaboration with Mayo Clinic.
3.31.17 exas april 2017 corporate presentation v2Exact Sciences
Exact Sciences is working to establish Cologuard as the new standard for colon cancer screening by:
1) Increasing adoption through a national TV campaign and primary care sales force focused on educating physicians.
2) Growing revenue and test volume rapidly, with 152% revenue growth and over 68,000 tests completed in Q4 2016.
3) Expanding coverage with 77% of the addressable population now covered, including Medicare, following inclusion in screening guidelines.
This presentation provides an overview of Interpace Diagnostics Group (IDXG), a commercial company that provides molecular diagnostic tests and pathology services for cancer evaluation. IDXG operates two CLIA-certified labs and has four proprietary molecular diagnostic tests for pancreatic cysts and thyroid nodules that assess cancer risk. The tests have high margins and barriers to entry due to reimbursement and complexity. Recent accomplishments include raising funds, improving financials, expanding insurance coverage and launching international distribution. The molecular diagnostic market is large and growing due to advantages over drug development. IDXG's tests establish new standards in cancer risk assessment for pancreatic cysts and thyroid nodules compared to current guidelines.
Exact Sciences is becoming the leader in advanced cancer diagnostics by extending its Cologuard platform to next-generation liquid biopsy cancer diagnostics. Cologuard addresses the challenge of colorectal cancer screening by providing a non-invasive, easy-to-use test with high early-stage cancer sensitivity. Cologuard's commercial success is driven by expanding insurance coverage, growing physician adoption, and direct-to-consumer marketing campaigns.
R3 Stem Cell Therapy: A New Hope for Women with Ovarian FailureR3 Stem Cell
Discover the groundbreaking advancements in stem cell therapy by R3 Stem Cell, offering new hope for women with ovarian failure. This innovative treatment aims to restore ovarian function, improve fertility, and enhance overall well-being, revolutionizing reproductive health for women worldwide.
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LGBTQ+ Adults: Unique Opportunities and Inclusive Approaches to CareVITASAuthor
This webinar helps clinicians understand the unique healthcare needs of the LGBTQ+ community, primarily in relation to end-of-life care. Topics include social and cultural background and challenges, healthcare disparities, advanced care planning, and strategies for reaching the community and improving quality of care.
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2. Forward-looking statements advisory
This Investor Presentation contains forward-looking statements within the meaning of Private
Securities Litigation Reform Act of 1995. Forward-looking statements reflect management’s
current expectations, as of the date of this presentation, and involve certain risks and
uncertainties. All statements other than statements of historical fact contained in this Investor
Presentation are forward-looking statements. The Company’s actual results could differ
materially from those anticipated in these forward-looking statements as a result of various
factors. Factors that could cause future results to differ materially from the recent results or
those projected in forward-looking statements include the “Risk Factors” described in the
Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission
(“SEC”) on March 22, 2018, as subsequently amended on April 2, 2018, and in the Company’s
other periodic filings with the SEC. The Company’s further development is highly dependent
on, among other things, future medical and research developments and market acceptance,
which are outside of its control. You are cautioned not to place undue reliance on forward-
looking statements, which speak only as of the date of this Investor Presentation. Caladrius
does not intend, and disclaims any obligation, to update or revise any forward-looking
information contained in this Investor Presentation or with respect to the matters described
herein.
2
3. Caladrius Biosciences: Uniquely positioned for near-term success
Late-stage therapeutics development company
Four active development programs; two with “breakthrough” designation*
CD34+ cells for ischemic repair (CLBS12*, CLBS14-CMD, CLBS14-NORDA1*)
T regulatory cells for immune modulation (CLBS03)
Financially stable and debt-free
Strong balance sheet (~$43 million cash projected for December 31, 2018)
Low operating cash burn (~18 months of operating cash on-hand2)
Multiple value creating events within the next 18 months
Key regulatory and data milestones across the pipeline
3
1Formerly known as CLBS14-RfA
2As of January 1, 2019
4. Experienced executive team with broad domain-specific expertise
David J. Mazzo, PhD
President and Chief Executive Officer
35+ years of experience in all aspects of large pharma (Merck, Baxter, RPR, HMR,
Schering-Plough) and emerging biopharma (Chugai USA, Regado); successful
international drug development across all therapeutic areas, international capital
raising and business transactions; Director and former Chairman of EyePoint Pharma
Douglas W. Losordo, MD
Executive VP, Global Head of R&D and
Chief Medical Officer
25+ years of experience as a leader in cell therapy research and development;
renowned clinician with noteworthy academic (Tufts, Northwestern, NYU) and industry
(Baxter) credentials; pioneer of CD34+ cell therapy
Joseph Talamo, CPA, MBA
Senior VP and Chief Financial Officer
25+ years of experience as a versatile finance executive with strong accounting/audit
background (KPMG) and leadership roles in publicly traded pharmaceutical
development and commercial-stage companies (OSI Pharmaceuticals, BMS)
Todd Girolamo, JD, MBA
Senior VP, General Counsel and
Corporate Secretary
25+ years of legal experience as a practicing attorney (Cahill, Gordon & Reindel; Reid
& Priest) as well as finance and biotechnology industry experience (Oppenheimer,
CIBC, Leerink Swann)
John D. Menditto
Executive Director,
Investor Relations and Corporate Communications
20+ years of experience as an investor relations and corporate communications
professional with a major focus on healthcare and life science (Novartis, Medco Health
Solutions, Argos Therapeutics)
4
6. CD34+ cells promote angiogenesis of the microvasculature
>700 subjects studied in randomized double-blind placebo-controlled trials
provide consistent evidence of therapeutic benefit and tolerance
Improved mortality, reduced chest pain and increased exercise tolerance in refractory angina1
Reduced amputation in critical limb ischemia2
Improved function in claudication3
6
1. Losordo et al. Circ Res 2011.; Povsic et al. JACC Cardiovasc Interv. 2016.
2. Losordo et al. Circ Cardiovasc Interv 2012.
3. From US study (n=17); Not yet published
Normal microvasculature Augmented microvasculature
post-CD34+ cells introduction
Compromised microvasculature
7. Simple, scalable and economical autologous cell therapy process
7
Cells returned to
same patient
Maximum of 4 days from donation to injection
Day -3: Patient dosed with GCSF to mobilize CD34+ cells from bone marrow to peripheral blood
Day 1: Sample collection via apheresis; shipment to processing center
Day 2: CD34+ cells isolated, preparation for patient injection and shipment to clinic
Day 3-4: Cells returned to patient through intramuscular, intracoronary or intramyocardial injection, depending on indication
Isolation of
CD34+ cells
Sample collection
ShipmentShipment
9. 9
CLI Represents a Multi-billion Dollar Global Market Opportunity
Japan USA Europe*
No-option CLI patients eligible for CLBS12
(not eligible for revascularization)
~51,000 ~300,000 ~560,000
CLI represents an expedited commercial opportunity in Japan
*Europe:
Source: Independent Third-party analysis; Full third-party report available upon request
CLBS12 eligible for early conditional approval based on current phase 2 study
Estimated >$100M initial commercial opportunity with significant pharmaco-eco benefits
Successful outcome in Japan could lead to expedited development in other major markets
11. 11
Design • Prospective, open label, controlled, randomized trial (1:1 w/SOC) CLI patients
Primary Endpoint • Time to continuous CLI-free status (2 consecutive monthly visits, adjudicated independently)
Study Size • 30 patients with no-option CLI plus 5 patients with Buerger’s Disease; ~10 centers in Japan
Dose • Up to 106 autologous G-CSF-mobilized peripheral blood-derived CD34+ cells/kg per affected limb
Control/ comparator
• Standard of Care drugs approved in Japan (e.g., antiplatelets, anticoagulants and vasodilators)
• Choice of pharmacotherapy will be made by the investigators according to protocol
Mode of
administration • Intramuscular, 20 injections in affected lower limb in single administration
Timing/Cost
• First patient enrolled in December 2017 with final results expected early 2020
• <$7 million costs remain to study completion (fully funded)
CLBS12 phase 2 study in Japan
Awarded SAKIGAKE (“breakthrough”) designation with priority review
Eligible for early conditional approval
13. 13
CMD is an unmet medical need with significant market potential
1Cleveland Clinic/AHA (American Heart Association)
2Townsend, N, et al.: Cardiovascular disease in Europe: epidemiological update 2016, European Heart Journal, Volume 37, Issue 42, 7 November 2016, Pages 3232–3245
3Kita, T; Coronary heart disease risk in Japan – an East/West divide?, European Heart Journal Supplements, Volume 6, Issue suppl_A, 1 March 2004, Pages A8–A11
4Ueshima, H, et al.; Cardiovascular Disease and Risk Factors in Asia, AHA Journal, December 16/23, 2008, Volume 118, Issue 25
Nearly 50% of patients with Coronary Artery Disease have CMD
Multi-billion dollar global opportunity based on significant pharmaco-eco benefits
USA1 Europe2 Japan3,4
~8,300,000 ~6,000,000 ~1,000,000
CMD Patients Eligible for CLBS14-CMD
Europe:
14. CLBS14-CMD Phase 1b/2a proof-of-concept study (ESCaPE-CMD)
Currently enrolling patients in USA
14
Design • Interventional, open label, proof-of-concept trial
Primary Endpoint • Safety and the evaluation of adverse events
Secondary Endpoints
• Changes from baseline to 6 months for coronary flow reserve, endothelial-dependent
microvascular function, time to angina; other cardiovascular metrics
Study Size • 20 patients at 2 centers in the USA (Cedars Sinai, LA & Mayo Clinic)
Dose • Up to 300 x 106 CD34+ cells
Control • No control arm
Mode of administration • Single intracoronary infusion
Timing/Cost
• First patient enrolled April 2018 with final results expected by end of 2019
• NIH grant covers majority of costs
• <$0.5 million CLBS costs remain to study completion (fully funded)
16. Patients Eligible for CLBS14-NORDA
NORDA presents a multi-billion dollar global market opportunity
16
Europe:
USA Europe Japan
<100K ~50K ~30K
Patients with NORDA often have multiple costly comorbidities
with no current effective treatment options
Source: Independent Third-party analysis; Full third-party report available upon request
17. Compelling previous results support CLBS14-NORDA development
17
Data license obtained from Shire in 1Q18
CLBS owns IP for product
IND active with CLBS as sponsor
RMAT (Regen Medicine Advanced Therapy) designation – awarded 2Q18
Granted by the FDA for therapies intended to treat serious conditions
Therapy must show preliminary evidence of addressing unmet medical need
Similar to breakthrough therapy designation, it includes increased agency interaction
and potential for accelerated approval
Type B meeting with FDA completed and discussions to finalize
remaining development steps expected to conclude in 1Q2019
FDA collaborating and showing flexibility consistent with the RMAT designation
18. 18
Compelling previous results support CLBS14-NORDA development
CD34+ cell therapy improves exercise tolerance in NORDA patients after single
administration in phase 2 study (n=168)1
1Losordo, D.W., et al., Intramyocardial, autologous CD34+++ cell therapy for refractory angina. Circ Res, 2011.
19. Compelling previous results support CLBS14-NORDA development
Phases 1, 2, & partial 3 clinical studies1,2,3 completed (combined n=304)
Compelling results of patient-level pooled-analysis published in European Heart Journal, 1/2018
Enrollment criteria meet NORDA definition in all studies
19
1 Losordo, D.W., et al, Intramyocardial transplantation of autologous CD34+++ stem cells for intractable angina: a phase I/Iia double-blind, randomized controlled trial. Circluation, 2007. 115(25): p. 3165-3172
2 Losordo, D.W., et al., Intramyocardial, autologous CD34+++ cell therapy for refractory angina. Circ Res, 2011. 109(4): p. 428-36.
3 Povsic, T.J., et al., The RENEW Trial: Efficacy and Safety of Intramyocardial Autologous CD34++(+) Cell Administration in Patients With Refractory Angina. JACC Cardiovasc Interv, 2016. 9(15): p. 1576-85.
IMPROVED total exercise time throughout the 3–
12 month period on treadmill stress test
Significant DECREASE in all-cause mortality at
24 months
LOWER relative frequency of angina throughout
the 3–12 month period
20. Compelling previous results support CLBS14-NORDA development
Phases 1, 2, & partial 3 clinical studies1,2,3 completed (combined n=304)
Compelling results of patient-level pooled-analysis published in European Heart Journal, 1/2018
Enrollment criteria meet NORDA definition in all studies
20
1 Losordo, D.W., et al, Intramyocardial transplantation of autologous CD34+++ stem cells for intractable angina: a phase I/Iia double-blind, randomized controlled trial. Circluation, 2007. 115(25): p. 3165-3172
2 Losordo, D.W., et al., Intramyocardial, autologous CD34+++ cell therapy for refractory angina. Circ Res, 2011. 109(4): p. 428-36.
3 Povsic, T.J., et al., The RENEW Trial: Efficacy and Safety of Intramyocardial Autologous CD34++(+) Cell Administration in Patients With Refractory Angina. JACC Cardiovasc Interv, 2016. 9(15): p. 1576-85.
Pooled angina frequency results
21. Compelling previous results support CLBS14-NORDA development
Phases 1, 2, & partial 3 clinical studies1,2,3 completed (combined n=304)
Compelling results of patient-level pooled-analysis published in European Heart Journal, 1/2018
Enrollment criteria meet NORDA definition in all studies
21
1 Losordo, D.W., et al, Intramyocardial transplantation of autologous CD34+++ stem cells for intractable angina: a phase I/Iia double-blind, randomized controlled trial. Circluation, 2007. 115(25): p. 3165-3172
2 Losordo, D.W., et al., Intramyocardial, autologous CD34+++ cell therapy for refractory angina. Circ Res, 2011. 109(4): p. 428-36.
3 Povsic, T.J., et al., The RENEW Trial: Efficacy and Safety of Intramyocardial Autologous CD34++(+) Cell Administration in Patients With Refractory Angina. JACC Cardiovasc Interv, 2016. 9(15): p. 1576-85.
Major cardiac events (MACE) results
23. T regulatory cells (Tregs) control immune balance and function
Deficiency in number or function of Tregs manifests as autoimmune disease
Augmentation of Tregs is intended to restore the immune system to its “native” state
and reduce/eliminate autoimmune disease symptoms and progression
23
1 Normal immune system:
immune balance
2 Autoimmunity:
immune imbalance
3 Infusion of Tregs:
Immune balance restored
T regulatory cells T effector cells Natural polyclonal T regulatory cells
24. Immune modulation critical to curtailing disease progression
24
Chronic blood glucose
management
Disease Modification
(CLBS03)
Function regeneration
Approach
Symptom management
Reduce or eliminate
disease progression;
potentially “curative”
Replace depleted cells/organs
producing insulin; does not
address underlying autoimmune
disease
Insulin
Impact
Improve therapeutic
effect and/or efficiency of
delivery of insulin/analogs
Avoid or reduce need
for insulin by preserving
active beta cells
Provides new source of insulin
producing cells
Availability Currently available with
more in development
Currently in
Phase 2 trial
Many years of
development remaining
25. 25
USA4 Europe5 Japan6
New onset T1D patients eligible for CLBS03 19,000 54,500 3,000
T1D is a >$1 billion worldwide market opportunity
5Europe:
Each year ~20,000 newly diagnosed patients <20 years of age in USA1
3% CAGR worldwide2
No curative treatments, only lifelong insulin therapy
Frequent serious, costly co-morbidities
Preserving remaining beta cell function should slow/stop disease progression
Leading to long-term pharmaco-economic benefits3
1. National Diabetes Statistic Report, 2014
2. Maahs DM, et al. Endocrinol Metab Clin North Am. 2010
3. Nathan DM, et al. Arch Intern Med. 2009
4. Thunander M et al, Diabetes Res Clin Pract. 2008:82:247-255
5. Haller MJ et al, Pediatr Clin North Am. 2005;52:1553-157
6. Kawasaki E., Matsuura N., Eguchi K., Diabetologia, 2006:49(5):828-36
26. Reliable, scalable & economically viable autologous cell therapy process
Proprietary and efficient clinical manufacturing process:
Simple, minimally intrusive cell collection process (whole blood or, eventually, apheresis)
Reliable and well-characterized cGMP process
High Phase 2 manufacturing success rate (>93%)
Discounted development and manufacturing services rates from HCATS through 2024
1 Day 1: Patient whole
blood donation
3 Day ~14: Infusion of Treg
therapy to same patient
Collection & Shipment Processing & Return Shipment Infusion
2 Days 2-13: Treg isolation,
activation & expansion
26
27. Phase 2 (T-rex) trial in adolescents with T1D initiated in March 2016
27
Design
• Double-blind, placebo-controlled, randomized (1:1:1) trial
• Adolescent patients ages 8 to <18 with recent-onset T1D (diagnosed within 100 days)
Standard Endpoints
• Preservation of C-peptide level, insulin use, severe hypoglycemic episodes, glucose
and hemoglobin A1c levels
Study Size • 110 patients enrolled across 15 study sites in the USA (enrollment completed in Dec. 2017)
Power • 80% power to detect a 0.2 pmol/mL difference in AUC mean C-peptide (active vs. placebo)
Dose • CLBS03 dose cohorts of 2.5 or 20 million cells/kg body weight
Control • Placebo (standard of care including insulin)
Mode of Administration • Single infusion
Timing/Cost
• Top-line data in early 2019
• ~$1 million in study costs remaining (fully funded)
28. CLBS03: Recent onset Type 1 Diabetes program summary
International regulatory recognition
FDA Fast Track (first time granted to a T1D program) and Orphan designation
EU ATMP (Advanced Therapeutic Medicinal Product) classification
Enrollment completed for landmark Phase 2 clinical study in T1D (T-Rex trial)
T-rex trial based on published clinical studies showing the T regulatory cell therapy is
well tolerated1,2, durable1 and preserving of beta cell function in children2
CIRM and JDRF grants of ~$10 million combine to offset study costs
Strategic collaboration with Sanford Research (Sioux Falls, SD) providing $5 million in
equity investment plus operating support for trial and clinical sites
Planned interim analysis completed: therapy is well tolerated and non-futile for
therapeutic effect
Primary endpoint analysis on track for 1Q2019
28
1. Bluestone, et al. Science Translational Medicine 2015
2. Marek-Trzonkowska, N et al. Clinical Immunology 2014
3. Remission Definition: Daily dose of insulin ≤ 0.5 UI/kg body weight & fasting c-peptide > 0.5 ng/ml at 12 months after recruitment
29. Country: USA
Pre-Clinical Phase 1 Phase 2 Phase 3
Country: USA
Country: Japan
Multi-product pipeline based on proprietary technology platforms
29
Active trial
Development plan to
BLA pending finalization
of discussions with FDA
CLBS14-CMD Coronary Microvascular Dysfunction
CD34+ Cell Therapy Platform
(Ischemic Repair)
T Regulatory Cell Therapy Platform
(Immune Modulation)
CLBS12 Critical Limb Ischemia*
CLBS03 Recent Onset Type 1 Diabetes
CLBS14-NORDA No Option Refractory Disabling Angina Country: USA
*Eligible for early conditional approval
30. Program 2019 2020
Phase 2 Topline Data
Expected
Phase 2 Topline Data
Expected
Phase 2 Topline
Data Expected
Finalize Clinical
Plan with FDA
Timeline of key development milestones by project
30
CLBS03 (Type 1 Diabetes)---------------------------------------------------------------------------------------------------------------------
CLBS12 (Critical Limb Ischemia)---------------------------------------------------------------------------------------------------------------
CLBS14-NORDA (No Option Refractory ----------------------------------------------------------------------------------------------------
Disabling Angina)
CLBS14-CMD (Coronary Microvascular Dysfunction)----------------------------------------------------------------------------------------
Pivotal Clinical
Trial Initiation
Target
Target Enrollment
Completion
Target Enrollment
Completion
32. Caladrius Biosciences: Uniquely positioned for near-term success
Late-stage therapeutics development company
Four active development programs; two with “breakthrough” designation*
CD34+ cells for ischemic repair (CLBS12*, CLBS14-CMD, CLBS14-NORDA1*)
T regulatory cells for immune modulation (CLBS03)
Financially stable and debt-free
Strong balance sheet (~$43 million cash projected for December 31, 2018)
Low operating cash burn (~18 months of operating cash on-hand2)
Multiple value creating events within the next 18 months
Key regulatory and data milestones across the pipeline
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1Formerly known as CLBS14-RfA
2As of January 1, 2019
33. Investor Relations Contact
John D. Menditto
Phone: 908.842.0084
Email: jmenditto@caladrius.com
www.caladrius.com
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NASDAQ: CLBS