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
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.
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.
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.
Caladrius Biosciences Corporate Presentation, January 2019Steve Sizer
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.
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
Tcelna is a precision immunotherapy under development by Opexa Therapeutics for the treatment of multiple sclerosis (MS). Opexa is currently conducting a Phase IIb clinical trial of Tcelna in patients with secondary progressive MS (SPMS). Previous clinical trials of Tcelna in both relapsing-remitting and SPMS patients showed promising results, including a 37% reduction in annualized relapse rate in a Phase IIb RRMS trial. Opexa has an option agreement with Merck Serono for the development and commercialization of Tcelna in MS that could provide over $220 million in payments if milestones are achieved.
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 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 $
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.
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.
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.
Caladrius Biosciences Corporate Presentation, January 2019Steve Sizer
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.
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
Tcelna is a precision immunotherapy under development by Opexa Therapeutics for the treatment of multiple sclerosis (MS). Opexa is currently conducting a Phase IIb clinical trial of Tcelna in patients with secondary progressive MS (SPMS). Previous clinical trials of Tcelna in both relapsing-remitting and SPMS patients showed promising results, including a 37% reduction in annualized relapse rate in a Phase IIb RRMS trial. Opexa has an option agreement with Merck Serono for the development and commercialization of Tcelna in MS that could provide over $220 million in payments if milestones are achieved.
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 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 (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
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.
Opexa therapeutics corporate presentation december 16OpexaTherapeutics
Tcelna is a precision immunotherapy under development by Opexa Therapeutics for the treatment of secondary progressive multiple sclerosis (SPMS). It is currently being tested in a Phase IIb clinical trial called Abili-T that aims to enroll 180 patients to receive two annual courses of treatment. The primary endpoint is reduction in whole brain atrophy, with secondary endpoints including measures of disability progression, relapse rate, and lesion activity. Positive results from previous trials support Tcelna's mechanism of reducing myelin-reactive T-cells and stabilizing disease progression in SPMS patients. An option agreement with Merck Serono provides the potential for commercialization if successful.
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin-reactive T-cell clones that are designed to program the immune system to target pathogenic myelin-reactive T-cells. Opexa has an ongoing Phase IIb clinical trial of Tcelna in SPMS and expects to complete enrollment in mid-2014, with top-line data expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Opexa therapeutics corporate presentation march 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, a precision immunotherapy, for the treatment of secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin reactive T-cell clones that are believed to trigger an immune response targeting pathogenic myelin reactive T-cells. Opexa has an ongoing Phase IIb clinical trial of Tcelna in SPMS and expects to complete enrollment in mid-2014, with top-line data expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
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.
Scientia Med Devices Cardiovascular Fall 08glorikian
The document provides an overview of the cardiovascular medical device market. It discusses definitions and classifications of medical devices, the worldwide medical device market analysis including sizing and segmentation, and focuses on key cardiovascular device segments - stents, cardiac rhythm management, valves, and catheters. The cardiovascular device market is large and growing, driven by an aging population and the increasing prevalence of cardiovascular disease globally. Major players operate in traditional segments like stents and cardiac rhythm management, while emerging markets provide new opportunities for growth.
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
MST-188 is a drug being developed by Mast Therapeutics to treat microcirculatory insufficiency. It is currently in phase 3 clinical trials for sickle cell disease and phase 2 trials are planned for acute limb ischemia. If successful, the company hopes to expand MST-188 to larger markets such as heart failure and trauma resuscitation. Preclinical studies show MST-188 improves blood flow and survival in animal models of various diseases.
Culprit vessel only PCI may reduce costs and improve outcomes for patients with acute myocardial infarction (AMI) complicated by cardiogenic shock, compared to immediate multivessel PCI. However, the optimal revascularization strategy is unknown. The CULPRIT-SHOCK trial will evaluate whether culprit vessel only PCI with potential staged revascularization reduces mortality and renal failure at 30 days compared to immediate multivessel PCI. An economic evaluation will also assess the costs and quality-adjusted life years of both strategies from the perspective of national health services over the lifetime of patients.
GALE-401 is a controlled release formulation of anagrelide being developed for the treatment of essential thrombocythemia. Phase 1 and 2 trials show it has an improved tolerability and safety profile compared to immediate release anagrelide, with a faster onset of platelet lowering effect. The company plans to initiate a pivotal Phase 3 trial in 2017 to evaluate GALE-401's ability to reduce platelet counts in essential thrombocythemia patients. NeuVax is an immunotherapy targeting HER2-positive breast cancer that elicits a strong CD8+ T-cell immune response. It is being evaluated in multiple clinical trials in various breast and gastric cancer settings both as a monotherapy and in combination with other agents like tra
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.
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 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
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.
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 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.
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.
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.
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
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.
Opexa therapeutics corporate presentation december 16OpexaTherapeutics
Tcelna is a precision immunotherapy under development by Opexa Therapeutics for the treatment of secondary progressive multiple sclerosis (SPMS). It is currently being tested in a Phase IIb clinical trial called Abili-T that aims to enroll 180 patients to receive two annual courses of treatment. The primary endpoint is reduction in whole brain atrophy, with secondary endpoints including measures of disability progression, relapse rate, and lesion activity. Positive results from previous trials support Tcelna's mechanism of reducing myelin-reactive T-cells and stabilizing disease progression in SPMS patients. An option agreement with Merck Serono provides the potential for commercialization if successful.
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin-reactive T-cell clones that are designed to program the immune system to target pathogenic myelin-reactive T-cells. Opexa has an ongoing Phase IIb clinical trial of Tcelna in SPMS and expects to complete enrollment in mid-2014, with top-line data expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Opexa therapeutics corporate presentation march 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, a precision immunotherapy, for the treatment of secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin reactive T-cell clones that are believed to trigger an immune response targeting pathogenic myelin reactive T-cells. Opexa has an ongoing Phase IIb clinical trial of Tcelna in SPMS and expects to complete enrollment in mid-2014, with top-line data expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
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.
Scientia Med Devices Cardiovascular Fall 08glorikian
The document provides an overview of the cardiovascular medical device market. It discusses definitions and classifications of medical devices, the worldwide medical device market analysis including sizing and segmentation, and focuses on key cardiovascular device segments - stents, cardiac rhythm management, valves, and catheters. The cardiovascular device market is large and growing, driven by an aging population and the increasing prevalence of cardiovascular disease globally. Major players operate in traditional segments like stents and cardiac rhythm management, while emerging markets provide new opportunities for growth.
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
MST-188 is a drug being developed by Mast Therapeutics to treat microcirculatory insufficiency. It is currently in phase 3 clinical trials for sickle cell disease and phase 2 trials are planned for acute limb ischemia. If successful, the company hopes to expand MST-188 to larger markets such as heart failure and trauma resuscitation. Preclinical studies show MST-188 improves blood flow and survival in animal models of various diseases.
Culprit vessel only PCI may reduce costs and improve outcomes for patients with acute myocardial infarction (AMI) complicated by cardiogenic shock, compared to immediate multivessel PCI. However, the optimal revascularization strategy is unknown. The CULPRIT-SHOCK trial will evaluate whether culprit vessel only PCI with potential staged revascularization reduces mortality and renal failure at 30 days compared to immediate multivessel PCI. An economic evaluation will also assess the costs and quality-adjusted life years of both strategies from the perspective of national health services over the lifetime of patients.
GALE-401 is a controlled release formulation of anagrelide being developed for the treatment of essential thrombocythemia. Phase 1 and 2 trials show it has an improved tolerability and safety profile compared to immediate release anagrelide, with a faster onset of platelet lowering effect. The company plans to initiate a pivotal Phase 3 trial in 2017 to evaluate GALE-401's ability to reduce platelet counts in essential thrombocythemia patients. NeuVax is an immunotherapy targeting HER2-positive breast cancer that elicits a strong CD8+ T-cell immune response. It is being evaluated in multiple clinical trials in various breast and gastric cancer settings both as a monotherapy and in combination with other agents like tra
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.
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 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
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.
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 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.
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.
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.
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.
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.
Prescient is preparing to commence a Phase 1b/2 clinical trial of PTX-200 in acute myeloid leukemia (AML) at prominent cancer research centers in the United States. PTX-200 is a novel inhibitor of the Akt signaling pathway that has shown promise in overcoming chemotherapy resistance and inducing cancer cell death with fewer toxic side effects than other Akt inhibitors. If successful, the upcoming AML trial could increase interest in Prescient and validate PTX-200 as an improved treatment for this disease with high unmet medical need. Prescient has additional clinical-stage oncology programs in breast cancer, ovarian cancer, and multiple myeloma across its pipeline.
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.
The business plan outlines an opportunity in the US healthcare market for point-of-care diagnostic solutions. Specifically, it aims to develop a biosensor using cytochrome P450 enzymes to enable quick diagnosis and drug monitoring. This would help address delays in prognosis and high costs associated with traditional lab tests. The plan is to launch solutions first for clinical trials and blood analysis, followed by urine analysis. Financial projections estimate capturing 3-5% of relevant markets by 2017 and achieving profitability, with research and development comprising 20% of expenses as new solutions are developed to address pain points in geriatric care and diagnostics.
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 Corporate Overview provides an overview of Cytori Therapeutics, Inc., a company that develops cell therapy technology. The summary includes:
1) Cytori has a transformative cell therapy platform that is regulated as a device and uses a business model of single-use consumables.
2) The technology has clinical experience in thousands of patients and strong intellectual property protection.
3) Near-term value drivers include milestones from a U.S. government contract, upcoming cardiovascular trial data, and international approvals and revenue growth.
This presentation provides an overview of Interpace Diagnostics Group (IDXG), a commercial company that provides molecular diagnostic tests and pathology services. Key points:
- IDXG has proprietary molecular diagnostic tests for pancreatic cysts (PancraGEN), thyroid nodules (ThyGenX/ThyraMIR), and Barrett's esophagus (BarreGEN).
- Clinical studies show PancraGEN more accurately determines cancer risk of pancreatic cysts compared to current guidelines. ThyGenX/ThyraMIR combination testing can accurately rule in or rule out thyroid cancer risk.
- The tests have significant market opportunities and address unmet clinical needs to avoid unnecessary surgeries and
- Sanofi is building a leading rare blood disorder franchise through the acquisitions of Bioverativ and Ablynx which expand their portfolio of therapies for rare diseases like hemophilia and acquired thrombotic thrombocytopenic purpura (aTTP).
- Bioverativ strengthens Sanofi's position in hemophilia with therapies like Eloctate and Alprolix and the investigational fitusiran. Caplacizumab shows strong results for aTTP and was filed for approval in the EU and U.S.
- The global hemophilia market is approximately $10 billion and growing at 7% annually, driven by reliable extended half-life factor therapies and broader
Similar to Caladrius Corporate Deck October 2018 (20)
Promoting Wellbeing - Applied Social Psychology - Psychology SuperNotesPsychoTech Services
A proprietary approach developed by bringing together the best of learning theories from Psychology, design principles from the world of visualization, and pedagogical methods from over a decade of training experience, that enables you to: Learn better, faster!
Basavarajeeyam is a Sreshta Sangraha grantha (Compiled book ), written by Neelkanta kotturu Basavaraja Virachita. It contains 25 Prakaranas, First 24 Chapters related to Rogas& 25th to Rasadravyas.
Here is the updated list of Top Best Ayurvedic medicine for Gas and Indigestion and those are Gas-O-Go Syp for Dyspepsia | Lavizyme Syrup for Acidity | Yumzyme Hepatoprotective Capsules etc
TEST BANK For An Introduction to Brain and Behavior, 7th Edition by Bryan Kol...rightmanforbloodline
TEST BANK For An Introduction to Brain and Behavior, 7th Edition by Bryan Kolb, Ian Q. Whishaw, Verified Chapters 1 - 16, Complete Newest Versio
TEST BANK For An Introduction to Brain and Behavior, 7th Edition by Bryan Kolb, Ian Q. Whishaw, Verified Chapters 1 - 16, Complete Newest Version
TEST BANK For An Introduction to Brain and Behavior, 7th Edition by Bryan Kolb, Ian Q. Whishaw, Verified Chapters 1 - 16, Complete Newest Version
Does Over-Masturbation Contribute to Chronic Prostatitis.pptxwalterHu5
In some case, your chronic prostatitis may be related to over-masturbation. Generally, natural medicine Diuretic and Anti-inflammatory Pill can help mee get a cure.
Cell Therapy Expansion and Challenges in Autoimmune DiseaseHealth Advances
There is increasing confidence that cell therapies will soon play a role in the treatment of autoimmune disorders, but the extent of this impact remains to be seen. Early readouts on autologous CAR-Ts in lupus are encouraging, but manufacturing and cost limitations are likely to restrict access to highly refractory patients. Allogeneic CAR-Ts have the potential to broaden access to earlier lines of treatment due to their inherent cost benefits, however they will need to demonstrate comparable or improved efficacy to established modalities.
In addition to infrastructure and capacity constraints, CAR-Ts face a very different risk-benefit dynamic in autoimmune compared to oncology, highlighting the need for tolerable therapies with low adverse event risk. CAR-NK and Treg-based therapies are also being developed in certain autoimmune disorders and may demonstrate favorable safety profiles. Several novel non-cell therapies such as bispecific antibodies, nanobodies, and RNAi drugs, may also offer future alternative competitive solutions with variable value propositions.
Widespread adoption of cell therapies will not only require strong efficacy and safety data, but also adapted pricing and access strategies. At oncology-based price points, CAR-Ts are unlikely to achieve broad market access in autoimmune disorders, with eligible patient populations that are potentially orders of magnitude greater than the number of currently addressable cancer patients. Developers have made strides towards reducing cell therapy COGS while improving manufacturing efficiency, but payors will inevitably restrict access until more sustainable pricing is achieved.
Despite these headwinds, industry leaders and investors remain confident that cell therapies are poised to address significant unmet need in patients suffering from autoimmune disorders. However, the extent of this impact on the treatment landscape remains to be seen, as the industry rapidly approaches an inflection point.
Integrating Ayurveda into Parkinson’s Management: A Holistic ApproachAyurveda ForAll
Explore the benefits of combining Ayurveda with conventional Parkinson's treatments. Learn how a holistic approach can manage symptoms, enhance well-being, and balance body energies. Discover the steps to safely integrate Ayurvedic practices into your Parkinson’s care plan, including expert guidance on diet, herbal remedies, and lifestyle modifications.
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 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 with 2 technology platforms
CD34+ cells for ischemic repair (CLBS12, CLBS14-CMD, CLBS14-RfA)
T regulatory cells for immune modulation (CLBS03)
Financially stable and debt-free
Strong balance sheet (~$50 million cash as of June 30, 2018)
Low operating cash burn (~$5 million/quarter)
Multiple value creating events within the next 18 months
Key regulatory and data milestones across the pipeline
3
4. Experienced executive team with broad domain-specific expertise
David J. Mazzo, PhD
President and Chief Executive Officer
30+ years of experience in all aspects of large pharma (Merck, Baxter, RPR, HMR,
Schering-Plough) and emerging biopharma (Chugai USA, Regado) company
operations; successful international drug development across all therapeutic areas and
international capital raising and business transactions; 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, Bristol-Myers
Squibb)
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 1: Sample collection via apheresis; shipment to processing center
Day 2: CD34+ cells isolated, prepared for patient and shipment to clinic
Day 3-4: Cells returned to patient through intracoronary infusion or intramuscular
injection near impacted area, depending on indication
Isolation of
CD34+ Cells
Sample collection
ShipmentShipment
9. 9
Week 4 Post-treatment Week 12 Post-treatmentBeforeTreatment
Provided by Dr. Atsu Kawamoto
CD34+ cell therapy improves blood flow in ischemic limbs
CLI Patient Laser Doppler Scans:
10. 10
CLI Represents a Multi-billion Dollar Global Market Opportunity
Japan USA Europe*
No-option CLI patients eligible for CLBS12
(not eligible for revascularization)
11,000 1,496,000 1,330,000
CLI represents an expedited commercial opportunity in Japan
*Europe:
Source: The Sage Group
Source: National Institute of Health
Source: CIA World Factbook; Norgren et al. (2007) J Vasc Surg
Source: CIA World Factbook; Becker F, et al. (2011), Chapter I: definitions, epidemiology, clinical presentation and prognosis
Source: Norgren et al., (2007) J Vasc Surg
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 pivotal 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 (CAD) 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 (CLBS to contribute ~$0.7 million – fully funded)
16. RfA Patients Eligible for CLBS14-RfA
1Global Cardiology Science & Practice: April 30, 2015
2National Institutes of Health: 2009
3Heart and Metabolism: 2017
4European Heart Journal (Kaplan Meier Analysis)
5Extrapolated from percentage of chronic heart failure patient prevalence in US and Europe
CLBS14-RfA presents a multi-billion dollar global market opportunity
16
Europe:
USA123 Europe3 Japan5
~900K ~1.0M ~200K
Patients with RfA often have multiple costly comorbidities
17. US development program in Refractory Angina (CLBS14-RfA)
Statistically Positive Late Stage Clinical Program Data Exclusively Licensed from Shire
Phases 1, 2, & 3 clinical studies1,2,3 (combined n=304); patient-level pooled-analysis results show
improvement in total exercise time (TET), angina frequency and major cardiac events (MACE)
(European Heart Journal, 1/2018)
17
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
18. Total exercise time (TET) and angina frequency results
US development program in Refractory Angina (CLBS14-RfA)
Statistically Positive Late Stage Clinical Program Data Exclusively Licensed from Shire
Phases 1, 2, & 3 clinical studies1,2,3 (combined n=304); patient-level pooled-analysis results show
improvement in total exercise time (TET), angina frequency and major cardiac events (MACE)
(European Heart Journal, 1/2018)
18
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.
Auto-CD34+ Cells Placebo
19. Major cardiac events (MACE) results
US development program in Refractory Angina (CLBS14-RfA)
Statistically Positive Late Stage Clinical Program Data Exclusively Licensed from Shire
Phases 1, 2, & 3 clinical studies1,2,3 (combined n=304); patient-level pooled-analysis results show
improvement in total exercise time (TET), angina frequency and major cardiac events (MACE)
(European Heart Journal, 1/2018)
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.
20. US development program in Refractory Angina (CLBS14-RfA)
Statistically Positive Late Stage Clinical Program Data Exclusively Licensed from Shire
IND reactivated with CLBS as sponsor
RMAT (Regen Medicine Advanced Therapy) designation – awarded 2Q2018
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 meeting
opportunities and potential for accelerated approval
FDA meeting to finalize development plan to BLA planned for 4Q2018
Preliminary discussions indicate grant funding likely to contribute to
any remaining clinical studies
20
22. 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
22
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
23. Immune modulation critical to curtailing disease progression and to acceptance
of transplantation or regeneration therapies
23
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 inslulin
producing cells
Availability Currently available with
more in development
Currently in
Phase 2 trial
Many years of
development remaining
24. 24
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
25. 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
25
26. CLBS03: Recent onset Type 1 Diabetes program overview
International regulatory recognition
FDA Fast Track designation (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 expected in early 2019
26
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
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
• <$2 million in study costs remaining (fully funded)
28. Country: USA
Pre-Clinical Phase 1 Phase 2 Phase 3
Country: Japan
Multi-product pipeline based on proprietary technology platforms
28
Active trial
Development plan to
BLA pending FDA
meeting in 4Q18
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-RfA Refractory Angina Country: USA
*Eligible for early conditional approval
Country: USA
29. Program 2018 (Jul-Dec) 2019 2020
Key dates and upcoming potential value creating events
29
CLBS03-----------------------------------------------------------------------------------------------------------------------------------------
CLBS12-----------------------------------------------------------------------------------------------------------------------------------------
CLBS14-CMD---------------------------------------------------------------------------------------------------------------------------------
CLBS14-RfA----------------------------------------------------------------------------------------------------------------------------------
Topline Data Expected
Topline Data Expected
Topline Data Expected
FDA Meeting
30. CLBS key financial information1
30
Current Cash2: $50.3m
2018 Actual (Jan-Jun) Operating Cash Burn3: $12.2m
2018 Projected (Jul-Dec) Operating Cash Burn4: $10m
Cash Runway Based on Current Plan4: 2020
Long-Term Debt: $0
Common Shares Outstanding: 9.7m shares
Options Outstanding:
Options Outstanding with Exercise Price < $5.00 = 551,000 shares
Options Outstanding with Exercise Price < $25.00 = 200,000 shares
Options Outstanding with Exercise Price > $25.00 = 393,000 shares
1.1m shares
(average exercise price $33.50)
1 As of June 30, 2018
2 Cash, cash equivalents and marketable securities
3 Includes approximately $2m in final retention payments related to the PCT sales transaction of 2017
4 Excludes CLBS14-RfA development costs - to be determined
31. Caladrius Biosciences: Uniquely positioned for near-term success
Late-stage therapeutics development company with 2 technology platforms
CD34+ cells for ischemic repair (CLBS12, CLBS14-CMD, CLBS14-RfA)
T regulatory cells for immune modulation (CLBS03)
Financially stable and debt-free
Strong balance sheet (~$50 million cash as of June 30, 2018)
Low operating cash burn (~$5 million/quarter)
Multiple value creating events within the next 18 months
Key regulatory and data milestones across the pipeline
31
32. Investor Relations Contact
John D. Menditto
Phone: 908.842.0084
Email: jmenditto@caladrius.com
www.caladrius.com
32
NASDAQ: CLBS