This presentation discusses RES-529, a first-in-class PI3K/Akt/mTOR pathway inhibitor being developed for ophthalmology and oncology indications. Preclinical studies show RES-529 inhibits VEGF-induced angiogenesis and permeability. Phase I clinical trials in wet age-related macular degeneration (AMD) show a favorable safety profile and preliminary signs of efficacy. Upcoming studies will evaluate subconjunctival administration in AMD patients. RES-529 also shows preclinical efficacy in glioblastoma models and has received FDA orphan drug designation for this indication. Planned studies will initiate a Phase I/II trial in glioblastoma patients in 2016.
Public Device & Biopharma Ophthalmology Company Showcase - Adverum Biotechnol...Healthegy
Public Device & Biopharma Ophthalmology Company Showcase - Adverum Biotechnologies at OIS@AAO 2016.
Presenter:
Samuel B. Barone, MD SVP, Clinical Development
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Public Device & Biopharma Ophthalmology Company Showcase - QLTHealthegy
Public Device & Biopharma Ophthalmology Company Showcase - QLT at OIS@AAO 2016.
Presenter:
David Saperstein, MD, Chief Medical Advisor
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Posterior Segment Company Showcase - OptheaHealthegy
Posterior Segment Company Showcase - Opthea at OIS@AAO 2016.
Presenter:
Megan Baldwin, PhD, CEO & Managing Director
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RETINA COMPANY SHOWCASE- Ocular TherapeutixHealthegy
Presentation by Ocular Therapeutix at OIS@ASRS 2016.
Participant:
Jonathan Talamo, MD, Chief Medical Officer- Ocular Therapeutix
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Posterior Segment Company Showcase - Clearside BiomedicalHealthegy
Posterior Segment Company Showcase - Clearside Biomedical at OIS@AAO 2016.
Presenter:
Daniel White, President & CEO
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Public Device & Biopharma Ophthalmology Company Showcase - Inotek Pharmaceuti...Healthegy
Public Device & Biopharma Ophthalmology Company Showcase - Inotek Pharmaceuticals at OIS@AAO 2016.
Presenter:
David Southwell, President & CEO
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Public Device & Biopharma Ophthalmology Company Showcase - Adverum Biotechnol...Healthegy
Public Device & Biopharma Ophthalmology Company Showcase - Adverum Biotechnologies at OIS@AAO 2016.
Presenter:
Samuel B. Barone, MD SVP, Clinical Development
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Public Device & Biopharma Ophthalmology Company Showcase - QLTHealthegy
Public Device & Biopharma Ophthalmology Company Showcase - QLT at OIS@AAO 2016.
Presenter:
David Saperstein, MD, Chief Medical Advisor
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Posterior Segment Company Showcase - OptheaHealthegy
Posterior Segment Company Showcase - Opthea at OIS@AAO 2016.
Presenter:
Megan Baldwin, PhD, CEO & Managing Director
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RETINA COMPANY SHOWCASE- Ocular TherapeutixHealthegy
Presentation by Ocular Therapeutix at OIS@ASRS 2016.
Participant:
Jonathan Talamo, MD, Chief Medical Officer- Ocular Therapeutix
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Posterior Segment Company Showcase - Clearside BiomedicalHealthegy
Posterior Segment Company Showcase - Clearside Biomedical at OIS@AAO 2016.
Presenter:
Daniel White, President & CEO
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Public Device & Biopharma Ophthalmology Company Showcase - Inotek Pharmaceuti...Healthegy
Public Device & Biopharma Ophthalmology Company Showcase - Inotek Pharmaceuticals at OIS@AAO 2016.
Presenter:
David Southwell, President & CEO
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Posterior Segment Company Showcase - Ohr pharmaceuticalHealthegy
Posterior Segment Company Showcase - Ohr pharmaceutical at OIS@AAO 2016.
Presenter:
Jason Slakter, CEO
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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. The Company launched two new clinical trials in 2021. 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.
Adrian W. Orr has over 25 years of experience in clinical research and R&D for medical devices. He specialized in developing and restructuring clinical research departments for mid-sized global corporations. Currently he is the Global Clinical Research Director at Haemonetics, where he is responsible for global regulatory and post-marketing clinical research on blood management devices. Previously he held clinical research leadership roles at several medical device companies.
Posterior Segment Company Showcase - Aura BiosciencesHealthegy
Posterior Segment Company Showcase - Aura Biosciences at OIS@AAO 2016.
Presenter:
Elisabet de los Pinos, CEO
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1) The presentation discusses clinical trial results from Ocata Therapeutics' RPE transplantation therapy for dry age-related macular degeneration (AMD) and Stargardt's macular dystrophy (SMD). Safety has been demonstrated over 1-4 years with no adverse events, and efficacy signals include vision improvement or stability.
2) Ocata is initiating a pivotal trial for SMD in Europe and a Phase 2 trial for dry AMD, with potential approval and commercial launch for SMD in 2019. Additional clinical programs are outlined for other ocular diseases.
3) Preclinical research demonstrates the RPE layer is vital for photoreceptor health, and Ocata aims to transplant new R
Posterior Segment Company Showcase - AerpioHealthegy
Posterior Segment Company Showcase - Aerpio at OIS@AAO 2016.
Presenter:
Joseph Gardner, President & CEO
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Ocata Therapeutics is developing regenerative ophthalmology therapies using retinal pigment epithelium (RPE) cells derived from pluripotent stem cells. Data from completed Phase 1/2 studies in Stargardt's macular degeneration and dry age-related macular degeneration showed long-term safety and evidence that transplanted RPE cells engrafted and persisted. Ocata is initiating pivotal and Phase 2 studies to further evaluate these RPE therapies with the goal of commercial launches beginning in 2019.
Public Device & Biopharma Ophthalmology Company Showcase - Aerie PharmaceuticalsHealthegy
This document summarizes information from Aerie Pharmaceuticals regarding their late-stage IOP-lowering products RhopressaTM and RoclatanTM. Key points include:
- RhopressaTM (netarsudil ophthalmic solution) NDA was filed in Q3 2016 based on positive results from two Phase 3 trials, Rocket 1 and Rocket 2.
- RoclatanTM (netarsudil/latanoprost ophthalmic solution) achieved statistical superiority over individual components in its Phase 3 trial Mercury 1 at all timepoints.
- Mercury 2 and Mercury 3 trials are ongoing to support the RoclatanTM NDA filing expected near year-end 2017
Posterior Segment Company Showcase - AGTCHealthegy
Posterior Segment Company Showcase - AGTC at OIS@AAO 2016.
Presenter:
Sue Washer, President & CEO
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1) Ocata Therapeutics presented a corporate presentation on their RPE cell therapy for macular degeneration.
2) Their phase 1/2 clinical trials showed signs of safety and efficacy, with anatomical and functional evidence of repair. Some patients showed visual acuity gains after over 3 years.
3) They are initiating pivotal trials for Stargardt's Macular Degeneration and a phase 2 trial for dry AMD to further evaluate the safety and efficacy of their RPE transplantation therapy.
This document discusses the importance of data quality in clinical trials and the use of MedDRA for coding clinical trial data. It provides an overview of MedDRA and outlines best practices for coding clinical data to ensure accurate analysis and safety signal detection. High quality input data is emphasized as it directly impacts the quality of outputs and analysis. Challenges in coding ambiguous or vague data are also explored.
The document describes Zepto, an innovative disposable device for cataract surgery that uses precision-pulse technology to create an accurate and reproducible capsulotomy quickly and safely. It discusses studies showing Zepto results in minimal temperature change in the eye and stronger capsulotomy edges compared to other methods. The document also outlines Dymedex's market analysis projecting rapid adoption of Zepto as it addresses the needs of high-risk patient populations and its ease of use could make it the standard of care.
FDA 2013 Clinical Investigator Training Course: Issues in Clinical Trials Des...MedicReS
FDA 2013 Clinical Investigator Training Course: Issues in Clinical Trials Designs for Devices
Owen Faris, Ph.D.,Deputy Director, Division of Cardiovascular Devices, Office of Device Evaluation, CDRH, FDA
The document provides an overview of MedDRA, including:
- MedDRA was developed under ICH to facilitate exchange of safety information. Its maintenance is overseen by an ICH committee.
- It defines MedDRA as a medical terminology used for regulatory processes from pre- to post-marketing.
- MedDRA has a hierarchical structure of System Organ Classes, High Level Groups, High Level and Preferred Terms to classify adverse event information.
The document summarizes the development of an anti-VEGF eye drop called PAN-90806 being developed by PanOptica for the treatment of wet age-related macular degeneration and other retinal diseases. It describes positive preliminary results from a Phase 1/2 clinical trial in wet AMD patients showing biological activity with no systemic side effects. It also outlines plans to further study PAN-90806 using an advanced formulation in additional clinical trials for wet AMD and proliferative diabetic retinopathy. Developing an effective anti-VEGF eye drop could provide significant benefits over frequent eye injections.
Statistical Issues In Medical Device TrialsJacobe2008
Authors:
George Koustenis,
FDA-CDRH
David Breiter,
Boston Scientific
Roseann White,
Abbott Vascular
George Woodworth, Univ. of Iowa
FDA/INDUSTRY STATISTICS WORKSHOP: Washington, D.C. Sept. 29, 2006
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.
Verisome (TM) a New Injectable Sustained Release and Biodegradable Intraocula...Randall Wong, M.D.
- Verisome is a new biodegradable and injectable intraocular drug delivery system that is designed to provide sustained release of medications over extended periods of time from 1 week to over 1 year.
- An ongoing Phase I clinical trial is evaluating the safety and tolerability of Verisome (IBI 20089) containing triamcinolone acetonide for the treatment of cystoid macular edema. No significant safety issues have been reported to date.
- Preliminary results from the first 10 patients in the trial suggest the Verisome system is able to control drug release as intended and reduce macular edema without serious adverse events. Further Phase II testing is planned to begin in the fourth quarter
There are several key challenges to commercializing new medical devices:
1) New devices must prove they offer real benefits over existing options. 2) Securing funding from investors is difficult due to the high risks and costs involved. 3) Extensive testing is required, including clinical trials, to obtain regulatory approval, which is also an expensive process. 4) There is often a disconnect between academic researchers developing new technologies and the business communities needed to commercialize them. Initiatives like IDEA Labs aim to help bridge this gap.
Introduction to MedDRA Coding in Drug Safety & Pharmacovigilance Process for Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
Contact:
"Katalyst Healthcares & Life Sciences"
South Plainfield, NJ, USA
info@KatalystHLS.com
This presentation discusses Eleven Biotherapeutics' development pipeline and upcoming milestones. Eleven is developing two protein therapeutics: Isunakinra, a topical IL-1 receptor blocker in Phase 3 for allergic conjunctivitis, with topline data expected in 1Q16; and EBI-031, a long-acting intravitreal IL-6 antibody planned to enter IND in 1H16 for diabetic macular edema and uveitis. The company highlighted opportunities for these programs to address unmet needs in large ophthalmic markets and provided an overview of upcoming clinical trial timelines and goals.
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.
Posterior Segment Company Showcase - Ohr pharmaceuticalHealthegy
Posterior Segment Company Showcase - Ohr pharmaceutical at OIS@AAO 2016.
Presenter:
Jason Slakter, CEO
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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. The Company launched two new clinical trials in 2021. 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.
Adrian W. Orr has over 25 years of experience in clinical research and R&D for medical devices. He specialized in developing and restructuring clinical research departments for mid-sized global corporations. Currently he is the Global Clinical Research Director at Haemonetics, where he is responsible for global regulatory and post-marketing clinical research on blood management devices. Previously he held clinical research leadership roles at several medical device companies.
Posterior Segment Company Showcase - Aura BiosciencesHealthegy
Posterior Segment Company Showcase - Aura Biosciences at OIS@AAO 2016.
Presenter:
Elisabet de los Pinos, CEO
Powered by:
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For more ophthalmology innovation
Visit us at www.ois.net
1) The presentation discusses clinical trial results from Ocata Therapeutics' RPE transplantation therapy for dry age-related macular degeneration (AMD) and Stargardt's macular dystrophy (SMD). Safety has been demonstrated over 1-4 years with no adverse events, and efficacy signals include vision improvement or stability.
2) Ocata is initiating a pivotal trial for SMD in Europe and a Phase 2 trial for dry AMD, with potential approval and commercial launch for SMD in 2019. Additional clinical programs are outlined for other ocular diseases.
3) Preclinical research demonstrates the RPE layer is vital for photoreceptor health, and Ocata aims to transplant new R
Posterior Segment Company Showcase - AerpioHealthegy
Posterior Segment Company Showcase - Aerpio at OIS@AAO 2016.
Presenter:
Joseph Gardner, President & CEO
Powered by:
Healthegy
For more ophthalmology innovation
Visit us at www.ois.net
Ocata Therapeutics is developing regenerative ophthalmology therapies using retinal pigment epithelium (RPE) cells derived from pluripotent stem cells. Data from completed Phase 1/2 studies in Stargardt's macular degeneration and dry age-related macular degeneration showed long-term safety and evidence that transplanted RPE cells engrafted and persisted. Ocata is initiating pivotal and Phase 2 studies to further evaluate these RPE therapies with the goal of commercial launches beginning in 2019.
Public Device & Biopharma Ophthalmology Company Showcase - Aerie PharmaceuticalsHealthegy
This document summarizes information from Aerie Pharmaceuticals regarding their late-stage IOP-lowering products RhopressaTM and RoclatanTM. Key points include:
- RhopressaTM (netarsudil ophthalmic solution) NDA was filed in Q3 2016 based on positive results from two Phase 3 trials, Rocket 1 and Rocket 2.
- RoclatanTM (netarsudil/latanoprost ophthalmic solution) achieved statistical superiority over individual components in its Phase 3 trial Mercury 1 at all timepoints.
- Mercury 2 and Mercury 3 trials are ongoing to support the RoclatanTM NDA filing expected near year-end 2017
Posterior Segment Company Showcase - AGTCHealthegy
Posterior Segment Company Showcase - AGTC at OIS@AAO 2016.
Presenter:
Sue Washer, President & CEO
Powered by:
Healthegy
For more ophthalmology innovation
Visit us at www.ois.net
1) Ocata Therapeutics presented a corporate presentation on their RPE cell therapy for macular degeneration.
2) Their phase 1/2 clinical trials showed signs of safety and efficacy, with anatomical and functional evidence of repair. Some patients showed visual acuity gains after over 3 years.
3) They are initiating pivotal trials for Stargardt's Macular Degeneration and a phase 2 trial for dry AMD to further evaluate the safety and efficacy of their RPE transplantation therapy.
This document discusses the importance of data quality in clinical trials and the use of MedDRA for coding clinical trial data. It provides an overview of MedDRA and outlines best practices for coding clinical data to ensure accurate analysis and safety signal detection. High quality input data is emphasized as it directly impacts the quality of outputs and analysis. Challenges in coding ambiguous or vague data are also explored.
The document describes Zepto, an innovative disposable device for cataract surgery that uses precision-pulse technology to create an accurate and reproducible capsulotomy quickly and safely. It discusses studies showing Zepto results in minimal temperature change in the eye and stronger capsulotomy edges compared to other methods. The document also outlines Dymedex's market analysis projecting rapid adoption of Zepto as it addresses the needs of high-risk patient populations and its ease of use could make it the standard of care.
FDA 2013 Clinical Investigator Training Course: Issues in Clinical Trials Des...MedicReS
FDA 2013 Clinical Investigator Training Course: Issues in Clinical Trials Designs for Devices
Owen Faris, Ph.D.,Deputy Director, Division of Cardiovascular Devices, Office of Device Evaluation, CDRH, FDA
The document provides an overview of MedDRA, including:
- MedDRA was developed under ICH to facilitate exchange of safety information. Its maintenance is overseen by an ICH committee.
- It defines MedDRA as a medical terminology used for regulatory processes from pre- to post-marketing.
- MedDRA has a hierarchical structure of System Organ Classes, High Level Groups, High Level and Preferred Terms to classify adverse event information.
The document summarizes the development of an anti-VEGF eye drop called PAN-90806 being developed by PanOptica for the treatment of wet age-related macular degeneration and other retinal diseases. It describes positive preliminary results from a Phase 1/2 clinical trial in wet AMD patients showing biological activity with no systemic side effects. It also outlines plans to further study PAN-90806 using an advanced formulation in additional clinical trials for wet AMD and proliferative diabetic retinopathy. Developing an effective anti-VEGF eye drop could provide significant benefits over frequent eye injections.
Statistical Issues In Medical Device TrialsJacobe2008
Authors:
George Koustenis,
FDA-CDRH
David Breiter,
Boston Scientific
Roseann White,
Abbott Vascular
George Woodworth, Univ. of Iowa
FDA/INDUSTRY STATISTICS WORKSHOP: Washington, D.C. Sept. 29, 2006
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.
Verisome (TM) a New Injectable Sustained Release and Biodegradable Intraocula...Randall Wong, M.D.
- Verisome is a new biodegradable and injectable intraocular drug delivery system that is designed to provide sustained release of medications over extended periods of time from 1 week to over 1 year.
- An ongoing Phase I clinical trial is evaluating the safety and tolerability of Verisome (IBI 20089) containing triamcinolone acetonide for the treatment of cystoid macular edema. No significant safety issues have been reported to date.
- Preliminary results from the first 10 patients in the trial suggest the Verisome system is able to control drug release as intended and reduce macular edema without serious adverse events. Further Phase II testing is planned to begin in the fourth quarter
There are several key challenges to commercializing new medical devices:
1) New devices must prove they offer real benefits over existing options. 2) Securing funding from investors is difficult due to the high risks and costs involved. 3) Extensive testing is required, including clinical trials, to obtain regulatory approval, which is also an expensive process. 4) There is often a disconnect between academic researchers developing new technologies and the business communities needed to commercialize them. Initiatives like IDEA Labs aim to help bridge this gap.
Introduction to MedDRA Coding in Drug Safety & Pharmacovigilance Process for Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
Contact:
"Katalyst Healthcares & Life Sciences"
South Plainfield, NJ, USA
info@KatalystHLS.com
This presentation discusses Eleven Biotherapeutics' development pipeline and upcoming milestones. Eleven is developing two protein therapeutics: Isunakinra, a topical IL-1 receptor blocker in Phase 3 for allergic conjunctivitis, with topline data expected in 1Q16; and EBI-031, a long-acting intravitreal IL-6 antibody planned to enter IND in 1H16 for diabetic macular edema and uveitis. The company highlighted opportunities for these programs to address unmet needs in large ophthalmic markets and provided an overview of upcoming clinical trial timelines and goals.
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.
Opexa Therapeutics provides a summary of its precision immunotherapy platform and clinical programs. Key points include:
- Tcelna in development for multiple sclerosis, with a Phase 2b trial ongoing in secondary progressive MS. Topline data expected in 2016.
- Pipeline expansion includes OPX-212 for neuromyelitis optica, with IND submission planned for mid-2015.
- Clinical trials show Tcelna reduces myelin reactive T-cells and improved outcomes for relapsing remitting MS patients versus placebo in Phase 2b trials.
- Agreement with Merck Serono provides potential for commercial partnership if Phase 3 is initiated for multiple sclerosis indications.
This document provides an overview of Advanced Cell Technology's annual meeting on regenerative medicine. The meeting will take place on October 22, 2013 in Palm Springs, CA.
The CEO update discusses ACT's progress in resolving past issues, developing clinical trials for retinal pigment epithelium transplants to treat dry AMD and Stargardt's disease, and advancing mesenchymal stem cell research.
The science update outlines ACT's research programs in developing stem cell treatments for various eye diseases from pluripotent stem cells, progress in clinical trials, and next steps to scale up production of retinal pigment epithelium cells and other ocular cell types for clinical applications.
The annual meeting presentation summarizes ACT's regenerative medicine programs and provides updates. Key points include:
1) ACT has concluded a successful meeting with its OAB and developed a 2014 goal-driven plan focusing on advancing its retinal pigment epithelium and ophthalmic programs as well as moving mesenchymal stem cell activities toward the clinic.
2) ACT has a robust development pipeline for ophthalmology and stem cell therapies providing multiple opportunities for commercialization and partnerships. Programs include treatments for dry AMD, Stargardt's disease, myopia, glaucoma, corneal disease, and mesenchymal stem cells.
3) Upcoming clinical milestones in the fourth quarter of 2013
OPT-302 is a soluble form of VEGFR-3 that inhibits VEGF-C and VEGF-D, blocking their binding to VEGFR-2 and VEGFR-3. It is being developed in combination with anti-VEGF-A therapies for the treatment of wet AMD. A Phase 1/2A clinical trial is actively recruiting wet AMD patients to evaluate the safety, pharmacokinetics, and efficacy of multiple doses of intravitreal OPT-302 in combination with ranibizumab. Preliminary data is expected in early 2016, with the goal of demonstrating OPT-302 can improve outcomes when added to anti-VEGF-A therapy by more completely suppressing the VEGF pathway.
Opexa Therapeutics presented information on their Precision Immunotherapy platform and lead programs. Their lead candidate, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has shown promise in reducing reactive T-cells and relapse rates in prior studies. Their platform also supports development of OPX-212 for neuromyelitis optica, a rare disease with no approved therapies. Opexa has an option agreement with Merck Serono for Tcelna in MS and recently secured $5 million in funding to advance OPX-212 into clinical trials for NMO.
Opexa Therapeutics Corporate Presentation September 2015OpexaTherapeutics
Opexa Therapeutics presented information on their Precision Immunotherapy platform and lead programs. Their lead candidate, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has shown promise in reducing reactive T-cells and relapse rates in prior studies. Their platform also aims to develop OPX-212 as a potential first-in-class therapy for neuromyelitis optica, an orphan disease with no approved treatments. Opexa has an option agreement with Merck Serono for Tcelna in multiple sclerosis and recently secured $5 million in funding to advance OPX-212 into clinical studies.
IntelGenx is an innovative pharmaceutical film company presenting its product pipeline and technology platforms to investors. The presentation discusses IntelGenx's oral thin film and buccal film technologies, product development strategies, business model, pipeline of products including treatments for migraines, erectile dysfunction, and brain degenerative diseases, and clinical trial results. It highlights a de-risked product candidate using montelukast to treat brain degenerative diseases and significant market opportunities for its pipeline products.
- The document is an investor presentation for IntelGenx Corp from April 2018.
- It provides an overview of IntelGenx, including its business strategies, product pipeline, clinical trial results, manufacturing facilities, and leadership team.
- The presentation highlights IntelGenx's drug delivery technology platforms, focus on developing generic and repurposed products, and partnerships with pharmaceutical companies.
ACT is conducting three clinical trials for dry age-related macular degeneration (AMD) and Stargardt's disease (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells. The trials have shown no adverse events and persistence of the cells with measurable improvements in visual acuity for most patients. ACT has additional programs developing blood components like red blood cells and platelets from stem cells, as well as mesenchymal stem cells for treating autoimmune diseases. ACT has an experienced management team and scientific advisors to advance its pipeline of ophthalmology and regenerative medicine therapies.
ACT is conducting three clinical trials for dry age-related macular degeneration (AMD) and Stargardt's disease (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells. The trials have shown no adverse events and persistence of the cells with visual improvements in most patients. ACT has additional programs in development for other ophthalmologic conditions as well as mesenchymal stem cells, blood components, and proprietary processes for generating large quantities of functional red blood cells, platelets, and megakaryocytes from stem cells. ACT has an experienced management team and board of directors to advance its pipeline and monetize assets.
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.
This document summarizes Sanofi's Sustaining Innovation Analyst Day held on December 13, 2017 in Paris. It outlines Sanofi's strategic focus on sustaining innovation in R&D through developing high-value projects, improving launch pipeline and portfolio prioritization processes. Sanofi's R&D model focuses on key disease areas like immunology, oncology, rare diseases, diabetes and vaccines. The presentation highlights 9 potential product submissions in the next 18 months and over 10 new pivotal trials expected to start in the next 12 months. Sanofi aims to financially discipline its R&D investments based on rigorous project prioritization methodology.
Opexa Therapeutics Corporate Presentation October 2015OpexaTherapeutics
Opexa Therapeutics presented information on their precision immunotherapy programs, Tcelna and OPX-212. Tcelna is in a Phase 2b clinical trial for secondary progressive multiple sclerosis and has shown a 37% reduction in relapses in prior trials. OPX-212 is being developed for neuromyelitis optica, an orphan disease with no approved therapies. Opexa expects to file an IND for OPX-212 by the end of 2015 after completing preclinical studies. The company highlighted their personalized T-cell immunotherapy platform, clinical progress, pipeline expansion opportunities, and management team experience in drug development.
Public Device & Biopharma Ophthalmology Company Showcase - pSividaHealthegy
Public Device & Biopharma Ophthalmology Company Showcase - pSivida at OIS@AAO 2016.
Presenter:
Nancy Lurker, President & CEO
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Presentation from OIS@ASCRS 2016
Amar Sawhney, PhD, President, CEO & Chairman
Video Presentation:
https://www.youtube.com/watch?v=nGQzo6czij0&list=PL1dmdBNnPTZJBhQxPOp0vdNg3s3wtN2yw&index=31
Vivos Inc. presented a new brachytherapy tool called RadioGel for treating cancers in humans and animals. RadioGel consists of yttrium-90 phosphate particles delivered via a hydrogel vehicle. It is designed to deliver a high radiation dose directly to tumors while minimizing damage to healthy tissue. The company expects to generate near-term revenue from veterinary applications and international licensing as it pursues FDA approval for multiple human cancer indications starting with skin cancer. Vivos has strong leadership and advisors in place and an intellectual property protected platform technology to become an important new tool for cancer treatment.
Immuron Limited is a clinical stage biopharmaceutical company developing oral immunotherapies for inflammatory and infectious diseases. Their lead program, IMM-124E, is in Phase 2 trials for NASH, ASH, and pediatric NAFLD, with interim data expected in 3Q 2017 and full results by 4Q 2017. IMM-124E has shown positive preclinical data, demonstrating a reduction in liver fibrosis, inflammation, and metabolic markers. Immuron also has a drug candidate, IMM-529, in development for C. difficile infection, expected to begin Phase 1/2 trials in 2Q 2017.
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2. 2
This presentation includes "forward-looking statements" under the Private Securities Litigation Reform Act
of 1995. All statements other than statements of historical facts contained in this presentation, including
statements regarding our anticipated future clinical and regulatory events, future financial/position,
business strategy and plans and objectives of management for future operations, are forward-looking
statements. Forward-looking statements can be identified by words such as “potential,” "may," "will,"
"should," "forecast,“ "project," "could," "expect," "believe," "estimate," "anticipate," "intend," "plan,“
“continue”, other words of similar meaning, derivations of such words and the use of future dates.
Forward-looking statements in this presentation include, without limitation, statements regarding our
current business strategies, the potential future commercialization of our product candidates, potential
estimated market sizes for our product candidates, anticipated start dates, durations and completion
dates, as well as potential future results, of our future clinical trials, anticipated designs of our future
clinical trials, and anticipated future regulatory submissions and events. Uncertainties and risks may cause
actual results to be materially different than those expressed in or implied by our forward-looking
statements. Particular uncertainties and risks include, among others, uncertainties regarding our ability to
license out our existing and license in additional products and technologies and the terms of such licenses;
uncertainties involved in clinical testing, the difficulty of developing pharmaceutical products, obtaining
regulatory and other approvals and achieving market acceptance, and other risks and uncertainties
described in our filings with the Securities and Exchange Commission, including our most recent annual
report on Form 10-K/A, subsequent quarterly reports on Form 10-Q and final prospectus dated July 31,
2014. All forward-looking statements in this presentation speak only as of the date of this presentation
and we undertake no obligation to update or revise any forward-looking statement, whether as a result of
new information, future events or otherwise.
3. 3
Company Highlights
• Specialty biopharmaceutical company focused on developing
products for ophthalmology, oncology and dermatology
• Two core proprietary technologies applicable in multiple
indications
– RES-529: PI3K/Akt/mTOR Pathway Inhibitor (AMD, Glioblastoma)
– RES-440: “Soft” Anti-Androgen (Acne)
– Strong preclinical in-vitro and in-vivo data
• Two Phase I clinical trials completed for RES-529 in AMD
• Three Phase I/II clinical trials to be initiated in mid-2016
– Age Related Macular Degeneration (AMD)
– Glioblastoma (Orphan Drug designation received)
– Acne
• Solid balance sheet with no debt
• Experienced management team supported by strong board of
directors
4. 4
• Stephen M. Simes - Chief Executive Officer
– BioSante, Unimed, Bio-Technology General, Gynex, Searle
• Phillip B. Donenberg, CPA – Chief Financial Officer
• BioSante, Unimed, Gynex
• Mark Weinberg, MD, MBA – Senior VP, Clinical Development
– Astellas, Lundbeck, Ovation, Takeda, Abbott
• David Sherris, Ph.D. - Chief Scientific Officer
– Paloma/Vasculomedics, OXiGENE, Serono, Unilever, Centocor
– Ph.D. in Biochemistry and Molecular Genetics
Experienced Management Team
5. 5
Strong Board of Directors
• Sol Barer, PhD - Chairman
– Former CEO & Executive Chairman, and Chairman, Celgene Corporation
– Board member Teva Pharmaceuticals, Aegerion Pharmaceuticals, Amicus Therapeutics
– Chairman of Medgenics, InspireMD and Contrafect Corporation
• Isaac Blech - Vice-Chairman
– Leading biotechnology entrepreneur and investor
– Genetic Systems, Nova, Celgene, ICOS, Texas BioTechnology, Pathogenesis
• Stephen M. Simes – CEO
– BioSante, Unimed, Bio-Technology General, Gynex, Searle
• Rex Bright
– SkinMedica, J&J, GlaxoSmithKline, Allergan
• Nelson Stacks
– Waveguide Corporation
• David Sherris, PhD – Chief Scientific Officer
– Paloma/Vasculomedics, OXiGENE, Serono, Unilever, Centocor
6. 6
Program / Therapeutic Area 2014 2015 2016
RES-529
OPHTHALMOLOGY
Age-Related Macular Degeneration
(AMD)
ONCOLOGY
Glioblastoma Multiforme (GBM)
RES-440
DERMATOLOGY
Acne
Phase I IVR
Formulation / Nonclinical
Phase I/II
SubConj
Formulation / Nonclinical Phase I/II
Formulation / Nonclinical Phase I/II
Phase I
SubConj (NEI)
Summary Product Portfolio & Timelines
Data expected from three clinical trials in 2017
8. 8
RES-529 & the PI3K Pathway
• The PI3K/Akt/mTOR pathway is an important signaling pathway for
many cellular regulation functions such as cell proliferation,
angiogenesis and vascular permeability
– Significant therapeutic opportunities for pathway signaling
– Agents in development affect different targets
• PI3K inhibitors
• Akt inhibitors
• mTOR inhibitors
• RES-529 is a novel approach to inhibition of the PI3K pathway
– Rather than interfering with the pathway directly via specific signaling proteins
(e.g. PI3K, Akt, mTOR), RES-529’s action results in the loss of the TORC1 and
TORC2 protein complexes thus preventing these complexes from generating
and potentiating signaling within the pathway.
RES-529 is a first-in-class PI3K/Akt/mTOR pathway inhibitor
9. 9
RES-529 Mechanism of Action
RES-529 interferes with the molecular components that form TORC1 and TORC2 preventing these complexes
from generating and potentiating signaling and thereby interferes with the activities of the PI3K/Akt/mTOR
pathway: translation, cell growth, ribosome biogenesis, metabolism, proliferation, and authophagy
11. 11
20 million cases of Wet AMD in the US/EU
10% of patients 66 to 74 years of age will have findings of macular
degeneration
Prevalence increases to 30% in patients 75 to 85 years of age
Age-Related
Macular
Degeneration
Competitor
Cost
Treatment
Sales
Lucentis (Genentech/Novartis)
Cost of single injection approximately
$2,000.00
Treatments every four to six weeks
Approximately $4 billion for all indications
Eylea (Regeneron/Bayer/Sanofi)
Cost of single injection approximately
$2,000.00
Treatments every eight to twelve weeks
Approximately $2.8 billion for all indications
• RES-529 initial ophthalmologic indication is for Wet AMD
• Back of the eye diseases have large market size and financial potential
• Wet AMD has approximately $6 billion in WW sales
Ophthalmology Market
12. 12
RES-529 Clinical Advantage in Wet AMD
• RES-529 is being developed as a subconjunctival injection
• Clinical trials for RES-529 will be designed to demonstrate that patients
may transition from initial therapy (Lucentis, Avastin, Eylea) to RES-529
– Enabling patients to be maintained with subconjunctival (vs. intravitreal) administration
– Once every three months therapy compared with monthly or on demand therapy requiring
frequent follow up
Intravitreal – needle
penetrates into the
globe of the eye
Subconjunctival – fluid is
injected beneath the
conjunctiva
13. 13
VEGF Induced Angiogenesis
• Mice pretreated with RES-529 IP 24 hrs prior to intradermal injection of
adenovirus expressing mouse VEGF-A164 to mouse ear
VEGF Induced Permeability
• Mice pretreated with RES-529 IP 24 hrs prior to intradermal injection of 100 ng
VEGF to ear; 0.1 ml of 0.5% Evans Blue given IV 30 minutes after VEGF treatment
RES-529 Inhibits VEGF Induced Angiogenesis and
Permeability
VEGF VEGF + RES-529
Xue Q. et al. Palomid 529, a Novel Small-Molecule Drug, Is a TORC1/TORC2 Inhibitor That Reduces Tumor Growth, Tumor Angiogenesis, and Vascular Permeability. Cancer Res 2008;68(22):9551–7.
RES-529 = P529 = Palomid 529
14. 14
RES-529: Completed Phase I Trials in AMD
• Protocol P52901 Company sponsored and conducted by Dr. Jeffrey Heier
(Ophthalmic Consultants of Boston) and Dr. David Brown (Retinal
Consultants Houston)
– A Phase I Open-Label Study to Investigate the Safety, Tolerability and
Pharmacokinetic Profile of Single Intravitreal and Subconjunctival Doses of
RES-529 in Patients with Advanced Neovascular Age-Related Macular
Degeneration (AMD)
• 15 patients treated via intravitreal injection
• Doses between 0.004mg and 0.5mg
• Protocol 11-EI-0066 sponsored by National Eye Institute (NEI) and
conducted by Dr. Catherine Meyerle
– A Phase I Unmasked Study to Investigate the Safety and Tolerability of
Subconjunctival Injections of RES-529 in Patients with Neovascular Age-
Related Macular Degeneration*
• 5 patients treated with subconjunctival injections of 2mg qmo x 3
*Dalal M. et al. Subconjunctival Palomid 529 in the treatment of neovascular age-related macular degeneration. Graefes Arch Clin Exp Ophthalmol (2013) 251:2705–2709.
RES-529 = P529 = Palomid 529
15. 15
RES-529: Clinical Data in AMD
• Patients Enrolled:
– End stage patients who were refractory or not appropriate for VEGF
therapy
– Most had significant visual deficits and retinal fluid on OCT
• Safety/Tolerability:
– Intravitreal administration (Company sponsored study)
• 0.5 mg (highest dose studied) associated with temporary visual
disturbance from haze in vitreous attributed to drug particles in the
vitreous. This effect resolved.
– Subconjunctival administration (NEI study)
• 2mg monthly x 3 administered; generally well tolerated
• Depot formed at injection site
• Efficacy:
– Preliminary evidence of biologic activity based on:
• Fluid pocket reduction
• Retinal thinning
• Cyst reduction
16. 16
RES-529: Upcoming Studies in AMD
• Finalize CMC work for subconjunctival
administration
• Complete pre-clinical studies necessary
to allow for next clinical studies to be
initiated in 2016
2015
• Phase I/II clinical trial in AMD
• Safety and tolerability, as well as
clinical efficacy
• Determine MTD for subconjunctival
administration
2016
18. 18
RES-529 in Glioblastoma
• A True Unmet Medical Need – Glioblastoma
– PI3K/Akt/mTOR pathway significantly up-regulated in glioblastoma
– Orally available: allows for improved administration and reduction in
health care costs
– FDA Orphan Drug Designation granted January 2015
– Significant financial upside due to limited and modestly effective
therapies currently available
– Phase 2a studies expected to commence in other tumors once MTD is
determined
• Market size expected to grow dramatically:
– 2013 World Wide market ~ $1 billion
– 2020 Expected WW market ~ $4.5 billion (Growth Rate of approximately 28%.* Growth
will be driven primarily by new agents.)
• EvaluatePharma, Adis R&D Insight
19. 19
RES-529 in Glioblastoma
• Current Standards of Care:
– Temodar, Avastin, Gliadel Wafer
– Median survival with only supportive care, less than 6 months**
– Median survival with aggressive chemotherapy in combination with
radiotherapy, 12 to 15 months**
• RES-529 potential advantages:
– Activity shown in multiple in vitro and in vivo animal models for GBM
with evidence that it can pass the blood brain barrier
– First-in-class mechanism of action exerting PI3K/Akt/mTOR pathway
control above other drugs targeting this pathway (PI3K pathway
significantly up-regulated in GBM)
– Specifically targets TORC1/TORC2 implicated in GBM
** Treatment options and outcomes for glioblastoma in the elderly patient, N.D. Arvold and D. A. Reardon, lin Interv Aging. 2014; 9: 357–367
20. 20
RES-529 Inhibits C6V10 Glioma Tumor Growth
• Tumor growth inhibited in subcutaneous
xenograft models
• Mice pretreated with RES-529 at 200
mg/kg/2 day, intraperitoneal for one week
• At second week, rat glioma cells were
injected subcutaneous
• Treatment continued while tumors were
allowed to grow for 21 days
• At termination, tumor volume reduced by
70% percent
*Data from Xue Q. et al Cancer Res 2008;68(22):9551–7.
RES-529 = P529 = Palomid 529
C6V10 model (fast growing tumor, constitutive VEGF secreting tumor)*
RES-529 C6V10
RES-529 C6V10
Control C6V10
Control C6V10
21. 21
RES-529 Inhibits U87 Glioma Tumor Growth
• Tumor growth inhibited in subcutaneous xenograft models
– U87 model (slow growing tumor)*
• Mice were injected subcutaneous with U87 glioma cells
• At day 3, mice began treatment with RES-529 at doses of 25 and 50 mg/kg/2 day
intraperitoneal
• U87 tumors were allowed to grow for 24 days
• At termination, tumor volume reduced by 29% (25mg) and 76% (50 mg)
*Data from Xue Q. et al Cancer Res 2008;68(22):9551–7.
RES-529 = P529 = Palomid 529
Control 25mg 50mg
22. 22
RES-529: Extensively Studied in Preclinical Oncology Models
• Efficacy in glioma subcutaneous xenograft model
– Similar efficacy regardless of route of administration (iv, po, or ip)1
• Penetration of the blood brain barrier with pharmacologically
active levels reached in murine brain2
• Potent effects in multiple tumor types
– In NCI60 cell line panel3
– Efficacy in breast cancer orthotopic xenografts4
– Synergy with radiotherapy and chemotherapy in prostate cancer
xenografts5,6
– Efficacy in lung cancer model
1Xue Q. et al. Palomid 529, a Novel Small-Molecule Drug, Is a TORC1/TORC2 Inhibitor That Reduces Tumor Growth, Tumor Angiogenesis, and Vascular Permeability. Cancer
Res 2008;68(22):9551–7 and Xue Q. unpublished data.
2Lin F. et al. Dual mTORC1 and mTORC2 inhibitor Palomid 529 penetrates the Blood–Brain Barrier without restriction by ABCB1 and ABCG2. Int J Cancer
2013Sep1;133(5):1222-33.
3Diaz R. et al. The novel Akt inhibitor Palomid 529 (P529) enhances the effect of radiotherapy in prostate cancer. Br J Cancer 2009Mar24;100(6):932-40.
4Xiang T. et al. Targeting the Akt/mTOR pathway in Brca1-deficient cancers. Oncogene 20011May26;30(21):2443-50.
5Gravinia GL. et al. Torc1/Torc2 Inhibitor, Palomid 529, Enhances Radiation Response Modulating CRM1-Mediated Survivin Function and Delaying DNA Repair in Prostate
Cancer Models. Prostate 2014JanJun74(8);852-68.
6Gravinia GL. et al. The TORC1/TORC2 inhibitor, Palomid 529, reduces tumor growth and sensitizes to docetaxel and cisplatin in aggressive and hormone-refractory prostate
cancer cells. Endocrine Related Cancer (2011);18:385-400
RES-529 = P529 = Palomid 529
23. 23
RES-529: Planned Studies in Glioblastoma/Oncology
• Finalize CMC work for oral administration
• Conduct pre-clinical studies necessary to allow
for IND clinical studies to be initiated in 2016
2015
• Phase I/II clinical trial in glioblastoma
- Efficacy assessment in recurrent glioblastoma
- Determine MTD in glioblastoma
• Plan Phase 2a studies in other tumor types
using MTD dose
- non-clinical data in breast, prostate, and lung
2016
24. 24
Summary of Clinical Timelines & Milestones
– 2015:
• Finalize CMC work for early clinical trials with RES-529 and RES-440
• Conduct pre-clinical studies necessary to enable submission of INDs and/or conduct
of next clinical studies
– 2016:
• AMD:
– Phase I/II clinical trial in AMD
» Safety and tolerability, as well as clinical efficacy
» Determine MTD for subconjunctival administration
• Glioblastoma:
– Phase I/II clinical trial in glioblastoma
» Efficacy assessment in recurrent glioblastoma
» Determine MTD in glioblastoma
– Plan Phase IIa studies in other oncology indications using MTD dose
» Current non-clinical data supports breast, prostate, and lung cancers
• Acne:
– Phase I/II clinical trial
» Assess safety and tolerability as well as efficacy
• Near-term goal: increased stockholder value through active
implementation of development programs
25. 25
RestorGenex Summary Financial Information
as of March 31, 2015
• Cash: $19,200,000
• Debt: $0
• Planned burn rate in 2015: ~ $1.0 million/month
• Shares outstanding: 18,614,968
• Options outstanding: 3,248,430
• Warrants outstanding: 4,815,266
• Fully diluted: 26,678,664
• Insider ownership: 30%
26. 26
Key Takeaways
• Specialty biopharmaceutical company focused on
developing products for ophthalmology, oncology and
dermatology
– Pipeline based on proprietary platforms
• Two Phase I clinical trials completed for RES-529 in AMD
• Three Phase I/II clinical trials to be initiated in 1H 2016
– Age Related Macular Degeneration (AMD)
– Glioblastoma (Orphan Drug designation received)
– Acne
• Solid balance sheet with no debt
• Experienced management team supported by strong
board of directors