General Investor and partnership presentation for the launch of a new pharmaceutical product. Specifically funds the initial clinical trial and market tests.
- The document discusses Ophthotech's development of Fovista, a potential new treatment for wet age-related macular degeneration (AMD).
- Fovista is currently in Phase 3 clinical trials and aims to show superiority over anti-VEGF monotherapy by inhibiting platelet-derived growth factor (PDGF) in addition to VEGF. Topline Phase 3 data is expected in 2016.
- Ophthotech has an ex-US licensing agreement with Novartis for Fovista that could provide over $1 billion in payments. Ophthotech will independently commercialize Fovista in the US if approved.
This document discusses ophthalmology IPOs from 2000 to the present. It notes that early ophthalmology companies like Bausch & Lomb and Allergan went public in the 1950s-1980s. The first ophthalmic biotech IPOs were in 2000. Since then, ophthalmology companies going public have had a median market capitalization of $293 million. Most ophthalmology companies that went public from 2000-2012 were later acquired. Recent ophthalmology IPOs from 2013-2014 raised between $58-192 million and have market caps ranging from $178 million to $1.3 billion. Ophthalmology has been one of the most attractive areas for biotech investors. A panel then
1) The document discusses Ophthotech's development of new therapies for age-related macular degeneration (AMD), including their lead drug Fovista.
2) Fovista is currently in Phase 3 clinical trials in combination with anti-VEGF drugs to treat wet AMD, with initial data expected in 4Q 2016.
3) Previous Phase 2b results showed Fovista in combination with Lucentis was statistically superior to Lucentis alone and had a favorable safety profile.
Spark Therapeutics is developing gene therapies for genetic disorders including SPK-RPE65 for retinal diseases. SPK-RPE65 achieved highly statistically significant results in a pivotal Phase 3 trial, demonstrating long-lasting benefits on vision tests over 3 years. If approved, it could be the first gene therapy approved in the US for a genetic disease. Spark is applying its validated AAV gene therapy platform to expand its pipeline to additional retinal diseases and indications.
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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For more ophthalmology innovation
Visit us at www.ois.net
The speaker summarizes recent work analyzing trends in antibody-drug conjugate (ADC) clinical trials using a proprietary database. Some key findings include: (1) There are currently 59 ADCs in active clinical development. (2) In the last year alone, 15 new ADCs entered clinical trials with most targeting solid tumors. (3) There is increasing diversity in the payloads being tested, now over 11 distinct payloads in clinical trials. Continued analysis of drug performance and dosing strategies may help improve the therapeutic window for ADCs.
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing what it believes will be transformative therapies to overcome unmet medical needs in neurodegeneration and liver disease. The Company is developing NE3107 for Alzheimer’s (AD) and Parkinson’s (PD) and BIV201 for refractory ascites and HRS-AKI.
BioVie Inc. (OTCQB: BIVI) is a clinical-stage company developing innovative drug therapies for liver disease. The Company’s drug candidate, BIV201 (continuous infusion terlipressin), has an Orphan Drug designation for the treatment of refractory ascites, FDA Fast Track status, and US patent pending. BIV201 also has an Orphan Drug designation for the treatment of hepatorenal syndrome (HRS). The active agent in BIV201, terlipressin, is approved for use in about 40 countries for the treatment of related complications of advanced liver cirrhosis but is not available in the US or Japan. The FDA has never approved terlipressin. BioVie is targeting this landmark achievement.
Visit BIVIinfo.com to learn more.
- The document discusses Ophthotech's development of Fovista, a potential new treatment for wet age-related macular degeneration (AMD).
- Fovista is currently in Phase 3 clinical trials and aims to show superiority over anti-VEGF monotherapy by inhibiting platelet-derived growth factor (PDGF) in addition to VEGF. Topline Phase 3 data is expected in 2016.
- Ophthotech has an ex-US licensing agreement with Novartis for Fovista that could provide over $1 billion in payments. Ophthotech will independently commercialize Fovista in the US if approved.
This document discusses ophthalmology IPOs from 2000 to the present. It notes that early ophthalmology companies like Bausch & Lomb and Allergan went public in the 1950s-1980s. The first ophthalmic biotech IPOs were in 2000. Since then, ophthalmology companies going public have had a median market capitalization of $293 million. Most ophthalmology companies that went public from 2000-2012 were later acquired. Recent ophthalmology IPOs from 2013-2014 raised between $58-192 million and have market caps ranging from $178 million to $1.3 billion. Ophthalmology has been one of the most attractive areas for biotech investors. A panel then
1) The document discusses Ophthotech's development of new therapies for age-related macular degeneration (AMD), including their lead drug Fovista.
2) Fovista is currently in Phase 3 clinical trials in combination with anti-VEGF drugs to treat wet AMD, with initial data expected in 4Q 2016.
3) Previous Phase 2b results showed Fovista in combination with Lucentis was statistically superior to Lucentis alone and had a favorable safety profile.
Spark Therapeutics is developing gene therapies for genetic disorders including SPK-RPE65 for retinal diseases. SPK-RPE65 achieved highly statistically significant results in a pivotal Phase 3 trial, demonstrating long-lasting benefits on vision tests over 3 years. If approved, it could be the first gene therapy approved in the US for a genetic disease. Spark is applying its validated AAV gene therapy platform to expand its pipeline to additional retinal diseases and indications.
Public Device & Biopharma Ophthalmology Company Showcase - pSividaHealthegy
Public Device & Biopharma Ophthalmology Company Showcase - pSivida at OIS@AAO 2016.
Presenter:
Nancy Lurker, President & CEO
Powered by:
Healthegy
For more ophthalmology innovation
Visit us at www.ois.net
The speaker summarizes recent work analyzing trends in antibody-drug conjugate (ADC) clinical trials using a proprietary database. Some key findings include: (1) There are currently 59 ADCs in active clinical development. (2) In the last year alone, 15 new ADCs entered clinical trials with most targeting solid tumors. (3) There is increasing diversity in the payloads being tested, now over 11 distinct payloads in clinical trials. Continued analysis of drug performance and dosing strategies may help improve the therapeutic window for ADCs.
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing what it believes will be transformative therapies to overcome unmet medical needs in neurodegeneration and liver disease. The Company is developing NE3107 for Alzheimer’s (AD) and Parkinson’s (PD) and BIV201 for refractory ascites and HRS-AKI.
BioVie Inc. (OTCQB: BIVI) is a clinical-stage company developing innovative drug therapies for liver disease. The Company’s drug candidate, BIV201 (continuous infusion terlipressin), has an Orphan Drug designation for the treatment of refractory ascites, FDA Fast Track status, and US patent pending. BIV201 also has an Orphan Drug designation for the treatment of hepatorenal syndrome (HRS). The active agent in BIV201, terlipressin, is approved for use in about 40 countries for the treatment of related complications of advanced liver cirrhosis but is not available in the US or Japan. The FDA has never approved terlipressin. BioVie is targeting this landmark achievement.
Visit BIVIinfo.com to learn more.
Aerie Pharmaceuticals is developing Rhopressa and Roclatan as once-daily eye drops to lower intraocular pressure for glaucoma. Rhopressa demonstrated efficacy in Phase 3 trials and Aerie expects to file an NDA in Q3 2016. Roclatan achieved all endpoints in a Phase 2b trial and is now in Phase 3 trials. Preclinical research also shows Rhopressa may modify diseased tissue and increase blood flow in the eye. Aerie is exploring AR-13154 for wet AMD which showed lesion size reduction exceeding the leading product in studies. The company collaborates with other organizations on delivery technologies and product candidates for the front and back of the eye.
GrayBug is developing an injectable, long-acting ocular drug delivery technology called GB-102 for treating wet age-related macular degeneration. GB-102 is a dual anti-VEGF/anti-PDGF therapeutic that enables injections every 4-6 months, compared to monthly injections currently required. GrayBug has worldwide rights to the technology from Johns Hopkins University and aims to file an IND for GB-102 in wet AMD in 2016. The company is also exploring applications of its technology for glaucoma and other ocular diseases, with the goal of developing long-acting drugs that improve compliance over daily eye drops.
This presentation discusses AGTC's visionary gene therapy programs to treat ophthalmic diseases. It highlights AGTC's lead product candidates for X-linked Retinoschisis (XLRS), Achromatopsia (ACHM-B3 and A3), and X-linked Retinitis Pigmentosa (XLRP). It also summarizes AGTC's competitive advantages in gene therapy manufacturing and its key upcoming milestones, including filing INDs for ACHM-A3 and XLRP and reporting initial clinical data for XLRS and ACHM-B3 programs in 2016. AGTC aims to become the world leader in ophthalmology gene therapy through its broad pipeline and partnerships.
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 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.
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.
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.
Posterior Segment Company Showcase - Notal visionHealthegy
Posterior Segment Company Showcase - Notal vision at OIS@AAO 2016.
Presenter:
Quinton Oswald, CEO
Powered by:
Healthegy
For more ophthalmology innovation
Visit us at www.ois.net
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
Powered by:
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For more ophthalmology innovation
Visit us at www.ois.net
Acucela is a clinical-stage ophthalmology company that develops novel therapeutics to treat sight-threatening eye diseases. Their lead product candidate, Emixustat hydrochloride, is being studied in clinical trials as a potential treatment for geographic atrophy associated with dry age-related macular degeneration. Preclinical studies showed Emixustat reduced the accumulation of toxins in the retina and protected retinal cells from light damage in a dose-dependent manner. Early clinical trials found Emixustat to be generally safe and well-tolerated, with pharmacologic effects observed in the retina. An ongoing Phase 2b/3 clinical trial is evaluating Emixustat's ability to reduce the growth rate of geographic
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 discusses a clinical trial being conducted by Ocata Therapeutics to evaluate the safety and efficacy of retinal pigment epithelium (RPE) cell transplantation for dry age-related macular degeneration (AMD). Previous phase 1 trials showed anatomical engraftment and functional improvement in vision. The current phase 2 trial will evaluate three regimens of short-term immunosuppression to prevent rejection of the transplanted cells. Safety will be the primary outcome measured with the goal of establishing a protocol to maintain graft survival without long-term immunosuppression.
- 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.
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.
Globavir is presenting its corporate strategy to target tropical diseases through accelerated drug development. The presentation highlights GBV006, a late-stage drug candidate for the treatment of dengue fever derived from two FDA-approved drugs. GBV006 has shown potent antiviral activity and 100% survival in animal studies. Globavir also has a pipeline of diagnostic tests and is pursuing partnerships to develop and commercialize its products globally.
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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For more ophthalmology innovation
Visit us at www.ois.net
Posterior Segment Company Showcase - Graybug VisionHealthegy
Posterior Segment Company Showcase - Graybug Vision at OIS@AAO 2016.
Presenter:
Jeffrey Cleland, PhD, President & CEO
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For more ophthalmology innovation
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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
ForSight VISION5 is developing an ocular insert system to deliver sustained drug therapy to the eye over 3-6 months for conditions like glaucoma. Their lead product is in Phase 2 trials using a prostaglandin analog to lower intraocular pressure. The insert is designed to eliminate the need for daily eye drops and improve patient compliance. Clinical studies show the insert is well-tolerated and has high retention rates. ForSight VISION5 aims to address the large unmet need of non-compliant glaucoma patients through this novel, non-invasive drug delivery approach.
Posterior Segment Company Showcase - Allegro OphthalmicsHealthegy
The document summarizes results from a phase 2b clinical trial of Luminate, a novel anti-integrin product being developed by Allegro Ophthalmics for the treatment of retinal diseases. The trial met its primary endpoint, showing Luminate was non-inferior to Avastin for improving visual acuity in patients with diabetic macular edema, with half as many injections. Luminate also met secondary endpoints for anatomical improvements measured by OCT. The results demonstrate the potential for Luminate as a well-tolerated monotherapy option with 12 weeks of durability through a new mechanism of action.
The company founder effectively life-hacked a $billion dollar OTC product and was awarded 6 patents for it. The funds raised were to prove efficacy and will probably be the only funding required before licensing.
We took a highly technical breifing and created a story that is both understandable and informative. We developed the brands, strategy, copy, graphics, and put together the deck and arranged key meetings with investors.
www.wildoutwest.com
Virginia Llera - Cómo optimizar la investigación en Enfermedades RarasFundación Ramón Areces
La Doctora Virginia Llera, Virginia A. Llera ofreció una conferencia el 17/09/2014 en la Fundación Ramón Areces. Llera es la Fundadora de la primera organización de Enfermedades Raras y drogas huérfanas en Latino América y Caribe, GEISER, y Presidenta del Foro Internacional, ICORD (International Conference on Rare Diseases & Orphan Drugs). Su conferencia, titulada 'Optimizando los procesos de investigación en enfermedades raras y medicamentos huérfanos', tuvo lugar dentro del ciclo sobre patologías poco frecuentes organizado por Fundación Ramón Areces en colaboración con Vall d'Hebron Institute of Research, Barcelona.
Aerie Pharmaceuticals is developing Rhopressa and Roclatan as once-daily eye drops to lower intraocular pressure for glaucoma. Rhopressa demonstrated efficacy in Phase 3 trials and Aerie expects to file an NDA in Q3 2016. Roclatan achieved all endpoints in a Phase 2b trial and is now in Phase 3 trials. Preclinical research also shows Rhopressa may modify diseased tissue and increase blood flow in the eye. Aerie is exploring AR-13154 for wet AMD which showed lesion size reduction exceeding the leading product in studies. The company collaborates with other organizations on delivery technologies and product candidates for the front and back of the eye.
GrayBug is developing an injectable, long-acting ocular drug delivery technology called GB-102 for treating wet age-related macular degeneration. GB-102 is a dual anti-VEGF/anti-PDGF therapeutic that enables injections every 4-6 months, compared to monthly injections currently required. GrayBug has worldwide rights to the technology from Johns Hopkins University and aims to file an IND for GB-102 in wet AMD in 2016. The company is also exploring applications of its technology for glaucoma and other ocular diseases, with the goal of developing long-acting drugs that improve compliance over daily eye drops.
This presentation discusses AGTC's visionary gene therapy programs to treat ophthalmic diseases. It highlights AGTC's lead product candidates for X-linked Retinoschisis (XLRS), Achromatopsia (ACHM-B3 and A3), and X-linked Retinitis Pigmentosa (XLRP). It also summarizes AGTC's competitive advantages in gene therapy manufacturing and its key upcoming milestones, including filing INDs for ACHM-A3 and XLRP and reporting initial clinical data for XLRS and ACHM-B3 programs in 2016. AGTC aims to become the world leader in ophthalmology gene therapy through its broad pipeline and partnerships.
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 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.
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.
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.
Posterior Segment Company Showcase - Notal visionHealthegy
Posterior Segment Company Showcase - Notal vision at OIS@AAO 2016.
Presenter:
Quinton Oswald, CEO
Powered by:
Healthegy
For more ophthalmology innovation
Visit us at www.ois.net
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
Powered by:
Healthegy
For more ophthalmology innovation
Visit us at www.ois.net
Acucela is a clinical-stage ophthalmology company that develops novel therapeutics to treat sight-threatening eye diseases. Their lead product candidate, Emixustat hydrochloride, is being studied in clinical trials as a potential treatment for geographic atrophy associated with dry age-related macular degeneration. Preclinical studies showed Emixustat reduced the accumulation of toxins in the retina and protected retinal cells from light damage in a dose-dependent manner. Early clinical trials found Emixustat to be generally safe and well-tolerated, with pharmacologic effects observed in the retina. An ongoing Phase 2b/3 clinical trial is evaluating Emixustat's ability to reduce the growth rate of geographic
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 discusses a clinical trial being conducted by Ocata Therapeutics to evaluate the safety and efficacy of retinal pigment epithelium (RPE) cell transplantation for dry age-related macular degeneration (AMD). Previous phase 1 trials showed anatomical engraftment and functional improvement in vision. The current phase 2 trial will evaluate three regimens of short-term immunosuppression to prevent rejection of the transplanted cells. Safety will be the primary outcome measured with the goal of establishing a protocol to maintain graft survival without long-term immunosuppression.
- 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.
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.
Globavir is presenting its corporate strategy to target tropical diseases through accelerated drug development. The presentation highlights GBV006, a late-stage drug candidate for the treatment of dengue fever derived from two FDA-approved drugs. GBV006 has shown potent antiviral activity and 100% survival in animal studies. Globavir also has a pipeline of diagnostic tests and is pursuing partnerships to develop and commercialize its products globally.
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
Powered by:
Healthegy
For more ophthalmology innovation
Visit us at www.ois.net
Posterior Segment Company Showcase - Graybug VisionHealthegy
Posterior Segment Company Showcase - Graybug Vision at OIS@AAO 2016.
Presenter:
Jeffrey Cleland, PhD, President & CEO
Powered by:
Healthegy
For more ophthalmology innovation
Visit us at www.ois.net
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
ForSight VISION5 is developing an ocular insert system to deliver sustained drug therapy to the eye over 3-6 months for conditions like glaucoma. Their lead product is in Phase 2 trials using a prostaglandin analog to lower intraocular pressure. The insert is designed to eliminate the need for daily eye drops and improve patient compliance. Clinical studies show the insert is well-tolerated and has high retention rates. ForSight VISION5 aims to address the large unmet need of non-compliant glaucoma patients through this novel, non-invasive drug delivery approach.
Posterior Segment Company Showcase - Allegro OphthalmicsHealthegy
The document summarizes results from a phase 2b clinical trial of Luminate, a novel anti-integrin product being developed by Allegro Ophthalmics for the treatment of retinal diseases. The trial met its primary endpoint, showing Luminate was non-inferior to Avastin for improving visual acuity in patients with diabetic macular edema, with half as many injections. Luminate also met secondary endpoints for anatomical improvements measured by OCT. The results demonstrate the potential for Luminate as a well-tolerated monotherapy option with 12 weeks of durability through a new mechanism of action.
The company founder effectively life-hacked a $billion dollar OTC product and was awarded 6 patents for it. The funds raised were to prove efficacy and will probably be the only funding required before licensing.
We took a highly technical breifing and created a story that is both understandable and informative. We developed the brands, strategy, copy, graphics, and put together the deck and arranged key meetings with investors.
www.wildoutwest.com
Virginia Llera - Cómo optimizar la investigación en Enfermedades RarasFundación Ramón Areces
La Doctora Virginia Llera, Virginia A. Llera ofreció una conferencia el 17/09/2014 en la Fundación Ramón Areces. Llera es la Fundadora de la primera organización de Enfermedades Raras y drogas huérfanas en Latino América y Caribe, GEISER, y Presidenta del Foro Internacional, ICORD (International Conference on Rare Diseases & Orphan Drugs). Su conferencia, titulada 'Optimizando los procesos de investigación en enfermedades raras y medicamentos huérfanos', tuvo lugar dentro del ciclo sobre patologías poco frecuentes organizado por Fundación Ramón Areces en colaboración con Vall d'Hebron Institute of Research, Barcelona.
ARSTAT Pharmaceuticals is seeking investors and partners as it prepares for an IPO. It has a pipeline of four first-in-class women's health products targeting large markets, including potential blockbusters Nuvocept and Premring. Nuvocept is a novel oral contraceptive designed for overweight/obese women, a major unmet need. Premring is a medicated vaginal ring to treat uterine fibroids and endometriosis. Both are in late-stage development and have the potential for high returns within 3-3.5 years due to ARSTAT's experienced team and advanced, low-risk programs. The company aims to complete development and sell assets at a valuation of at least $180M
This document describes a new patented nanotechnology called Hybrid-NanoengineeringTM that can resolve solubility issues for over 60 drugs. Dr. Mewa Singh is offering a free proof-of-concept trial to collaborate with pharmaceutical companies. The technology involves using a new molecule that acts as both the active drug and part of the nanoparticle formulation. Experimental results show Hybrid-NanoengineeringTM increases drug potency and overcomes drug resistance compared to conventional formulations. The technology has potential to extend drug patents and beat generic competition.
This was a lecture in the course "Significant Medical Conditions in Seniors" presented at Peer Learning in Chapel Hill, NC, USA in 2016 by Michael C. Joseph, MD, MPH.
- IntelGenx Corp is a drug delivery company focused on oral film technologies. They have a pipeline of products using their VersaFilm and AdVersa drug delivery platforms.
- They have a state-of-the-art manufacturing facility in Montreal approved by Health Canada. Their pipeline includes products for migraine, erectile dysfunction, schizophrenia, and neurodegenerative diseases.
- They are conducting clinical trials for a Montelukast VersaFilm to treat cognitive impairment associated with neurodegenerative diseases like Alzheimer's, with promising preclinical and Phase I results shown.
This corporate presentation discusses BELLUS Health's focus on developing drugs for rare diseases. Their lead product candidate, KIACTA, is in Phase 3 trials for AA amyloidosis, a rare and deadly kidney disease with no existing treatment. Positive results from a previous Phase 3 trial showed KIACTA slowed kidney function decline. If successful, KIACTA could achieve premium pricing compared to other orphan drugs. BELLUS is also developing treatments for other rare diseases including sHUS and AL amyloidosis through clinical trials and partnerships.
Bach Pharma Inc. is developing GVT, a potential solution of neurodegenerative diseases for U.S. distribution. Find out about GVT and our current fundraising goals.
Bellus corporate presentation feb 2015 (r)BellusHealth
This corporate presentation by Roberto Bellini of BELLUS Health Inc. discusses the company's focus on developing drugs for rare diseases. The lead product candidate is KIACTA for AA amyloidosis, a rare kidney disease with no existing treatment. Key highlights include:
- KIACTA showed positive results in a Phase III trial, significantly reducing kidney function decline. A Phase III confirmatory study is ongoing.
- If approved, KIACTA could achieve premium pricing like other orphan drugs, with over $70M in funding from partner Auven Therapeutics.
- The company also has a preclinical antibody, Shigamab, targeting STEC-related HUS, a rare kidney disease
The document discusses Genentech's capacity planning needs to meet potential demand for their cancer drug Avastin. It provides background on Genentech's operations, manufacturing processes, and previous capacity expansions. It examines potential demand scenarios for Avastin and outlines options for expanding capacity, including building additional facilities or expanding existing plants. A decision is needed on building a new plant called CCP3 to ensure sufficient capacity through 2015 while balancing risks and costs.
The document discusses Genentech's capacity planning needs to meet potential demand for their cancer drug Avastin. It provides background on Genentech's operations, manufacturing processes, and previous capacity expansions. It examines potential demand scenarios for Avastin and outlines options for expanding capacity, including building additional facilities or expanding existing plants. A decision is needed on building a new plant called CCP3 to ensure sufficient capacity through 2015 while balancing risks and costs.
The document discusses Genentech's capacity planning needs to meet potential demand for their cancer drug Avastin. It provides background on Genentech's operations, manufacturing processes, and previous capacity expansions. It examines potential demand scenarios for Avastin and outlines options for expanding capacity, including building additional facilities or expanding existing plants. A decision is needed on building a new plant called CCP3 to ensure sufficient production capacity through 2015.
The document discusses Genentech's capacity planning needs to meet potential demand for their cancer drug Avastin. It provides background on Genentech's operations, manufacturing processes, and previous capacity expansions. It examines potential demand scenarios for Avastin and outlines options for expanding capacity, including building additional facilities or expanding existing plants. A decision is needed on building a new plant called CCP3 to ensure sufficient capacity through 2015 while balancing risks and costs.
Gino Martini, EIPG President
Presentation at Malta Qualified Persons Association-European Industrial Pharmacists Group-University of Malta joint seminar “The Successes And Challenges Of Today’s Pharmaceutical Industry”, Malta 2008.
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Launch of a new Pharmaceutical Product
1. 100M people in the US suffer from a
problem for which we have a strong
solution. The patents are issued, we
are raising funds for FDA trial...
NOCTUROL™ for NOCTURIA
07.21.2014
2. Misery associated with nocturia
Insomnia, fatigue, depression,
lower quality of life
US lost work time $60B/year
Incontinence care currently 25%
of the costs of nursing home care
Heart disease, stroke, brain
damage, hip fractures
NOCTURIA is the #1 urology complaint
of men and women 55-84
65%of people
ages 55-84
100Msufferers in US
(2.2B worldwide)
THIS IS A SIGNIFICANT UNMET MEDICAL NEED AS NO CURRENT
DRUG TREATS LOW NOCTURNAL BLADDER CAPACITY
2
3. Nocturnal is a unique 8 hour extended release formulation
that provides increased nocturnal bladder capacity (NBC)
through the night. (+50-100%)
HOW IT WORKS: For 76% of people suffering from nocturia,
low NBC is a key issue. The cause is excess Prostaglandins
(PGE2) that irritate the bladder.
For sufferers of Nocturia and Over Active Bladder, a
combination of APAP & ibuprofen will inhibit PGE2
production to increase comfortable bladder capacity.
People are informally using the combination of already
approved drugs. It is proving effective for 80%. Our next step
is to prove efficacy in trials. Our use of these drugs for this
purpose and the addition of the timed release aspect are
protected by numerous broad patents.
3
INTRODUCING
APAP
ibuprofen
+ time release
Noctural has three synergistic
mechanisms of action.
4. SCIENTIFIC ADVISORS
Frank Rauscher, III, Ph.D., Chief Scientist: Dep. Director, Wistar Inst.
Khurshid Iqbal, Ph.D., Pharma Chemist, J&J, Hoffmann-LaRoche
Tony DelConte, MD, led Novartis Enablex FDA trials
Thomas Garvey III, MD, NIH, FDA, drug safety expert
David Shantz, Marketing
LICENSING AND LEGAL
Joy Barton, Ph.D., Novartis, Licensing Agent
Ed Allera, Buchanan Ingersoll, FDA Focused Lawyer
Ping Wang MD IP Medical Expert Andrews Kurth, (named top 1000 patent Lawyer)
4
World Class Team
David Dill, CEO, IBM, CFO of 7 firms, 35 years as manager
5. 6 Patents issued in 2012-2014 (16+ year life)
• Method of use claims; broad spectrum combinations.
• Covers use with Over Active Bladder (OAB)
• Extended release critical: 2-3 hour half life standard
• Wide range dosing of APAP with antimuscarinics, diuretics, antidiuretics,
spasmolytics, and/or NSAIDs.
• PCT process for worldwide protection filed 12/2010
5
Intellectual Property
8,236,857 B2 – Extended-release formulation
8,236,856 B2 – Delayed release formulation
8,685,453 B2 – Low dose APAP and NSAIDs
8,445,015 B2 – Extended-release formulation
8,445,011 B2 – Delayed-release formulation
8,703,184 B2 – Delayed-release formulation
US PATENTS ISSUED:
6. 6
Vesicare, Enablex, Myrbetriq
• Effects often take months
• Large % do not respond
• Severe side effects
• 43–83% stop in < 30 days
• 10-15% fewer bathroom trips
• Treats annoying problem
• Significant competition
OVERACTIVE BLADDER DRUGS
2M
patients
(0.1%)
$2B
SALES
• Effects in as little as 30 mins.
• 80% positive (anecdotal)
• No side effects
• 20% drop out (anecdotal)
• 33-100% fewer nightly trips
• Treats dangerous problem
• No current competition
$2-8B
SALES ?
Proving effective for
OAB, but focused
on treatment of
bladder problems
related to sleep
7. 7
Actionable Market
$2-8B
SALES
2018
Paxerol is a new product that is effective in ways that current OAB
(over active bladder) products are not.
*source 2013 Report by Debra E. Irwin et al / Company estimates
NOT A
SIGNIFICANT
PROBLEM
1 x per night
1.2 Billion
423 Million
Unaffordable
600 Million
130 Million No efficacy
50 Million Medical Issues
$0.30 / dose/daily = $39B
annual sales potential (retail)
NOTE: Forecasting a new market is
highly speculative.
2.4 Billion sufferers
es'mated
by
2018
8. Almost all regulatory bodies outside US permit use of unlicensed drugs
(“Specials”). We have an agreement for such distribution of Nocturol.
Contract signed April 2014 (Q1 2015 launch) with Ethicor Pharma, Ltd. (UK). Plan
to launch Paxerol with support of key opinion leaders, education sessions, and
published articles. Partnered with UDG, UK’s largest prescription drug distributor.
Ethicor exits when big pharma enters markets.
Top end price target – $5/dose controlled by Ethicor
Gross profit estimate – 98%
Only ages 60+, EU, MENA, and SA, with no advertising
8
Ethicor Short-term Revenues
2015 2016 2017 2018 2019 TOTAL
$832 $6,611 $26,949 $50,538 $73,161 $151,091
$k Ethicor Projected Income
9. We have protocol feedback and indications of interest from key
global pharmaceutical leaders.
Licensee to complete trials, patents, and launch Nocturol. We are expecting an
upfront payment after Phase 2 trial (mid-2015), milestone payments, 15% royalty.
9
Big Pharma License
10. Safe Drug, low risk Huge unmet need.
Tight patent barrier through 2030
10
Strengths of This Investment
This is not a new chemical entity. Approval can be attained
with a simpler, relatively inexpensive trial: Avoids Phase 1 Trial
safety tests, and years of risk from unexpected side effects.
Strong Ethicor validation. International sales begin before trial
completion, mitigates risk. Mid-2015 worldwide big pharma license.
Strong ROI @ up to 94% net profit.
$3M to positive Cash flow 12-15 months
1
2
3
4
5
$787K raised so far primarily through pooled
due diligence of Keiretsu Forum.
11. 11
Use of Funds
Primary use of funds will be in the management of the trial
process and preparations for Ethicor launch.
2014 Q1 Q2 Q3
Ethicor GP Rev Share $42 $125 $221
Expenses
Mfg. Testing $500 – – –
Clinical Trials $612 $425 – –
Business Dev. $108 $29 $33 $38
Patents/ Legal WW $396 $130 $130 $90
G&A / other $475 $55 $50 $50
TOTAL Expenses $2,095 $639 $213 $178
Net Income ($2,095) ($597) ($88) $43
Figures expressed as $k
2015
Profits in Q3 2015
12. 12
Projections
2014 2015 2016 2017 2018 2019 2020 2021
Ethicor GP Rev Share $1 $7 $27 $51 $73
Big Pharma Milestones – $13 $5 $5 $38
Big Pharma Royalties – – – – $15 $77 $307 $768
TOTAL Revenue $0 $13 $12 $32 $103 $150 $307 $767
Expenses $2 $2 $1 $2 $6 $8 $16 $39
Net Income ($2) $11 $11 $30 $97 $142 $291 $729
% – 84% 92% 94% 95% 95% 95% 95%
Figures $Millions
Key Assumptions
1. FDA approval 2018 (no accelerated approval designation)
2. $0.30 retail ($0.15 wholesale) in 2018
3. 15% License fee (15-18% tiered structure would add $120M in 2021)
4. Licensing Agent 5% fee
5. A $5B big pharma drug by 2021 (no OAB or bedwetting solution)
13. Patents filed (issued 2012, 2013, & 2014)
FDA Confirms no Phase 1 safety tests required
EU“Special”Ethicor contract (launch Q1/2015)
Series A complete and trial contract signed
1st patient starts trial:
13 week crossover design; patients with 3+ voids/night
60 patient trial completed.
Sign big pharma license (Series A cash back)
FDA approval H1- Paxerol launch 6 weeks later
13
Milestones
7/2010
4/2012
4/2014
Q3/2014
Q2/2015
Q3/2015
2018
Q1/2015
14. • 22 patients informally tested Nocturnal for a Johns Hopkins physician.
17 reported good results (fewer nightly bathroom trips). (77%)
• 18 patients have since tested Nocturol, with 15 reporting good results
(1-4 fewer nightly bathroom trips).
• Placebo effects are always possible, but much less likely when sleeping.
• 32 of 40 patients (80%) self-report success – without placebo controls.
• 3 articles report OAB suffers’urine averages 700% higher PGE2 levels -
Nocturnal addresses PGE2 levels.
14
Anecdotal Evidence
15. Independent clinical studies consistently report that ~70% of nocturia patients
experience improvement when taking appropriate doses of certain NSAIDs
80% of patients with neurogenic bladder and 74% of patients with BPH
experience improvement in nocturia when taking appropriate doses of NSAIDs
(consistent with animal studies)
Johns Hopkins proves NSAIDs reduce bladder spasms
Ohio State preclinical model of bladder function showed acetaminophen equal
to NSAIDs in reducing inflammation, achieved through independent
mechanisms of action
Together acetaminophen and NSAIDs impact multiple independent molecular
pathways to relieve nocturia symptoms
15
Supporting Research
16. No matter how nice
your bathroom is, nobody wants
to visit in the middle of the night
17. 17
OAB Market Segment
$2.1B
SALES
2011
*source 2013 Report by Debra E. Irwin et al / Company estimates
Astellas
VesiCare
3.8M
1.6 M Enablex
In the 12 months ending January 2012, more than 17.8 million
prescriptions were filled for drugs in the Over Active Bladder,
accounting for almost $2.1 billion in sales.
Oxybutynin
3.7M
Oxybutynin
ER 3.6M
Pfizer
Detrol LA
3.3 M
712 k Pfizer Toviaz
18. 18
A Serious Problem
* Results from the Third National Health and
Nutrition Examination Study
12 year survival odds for
women ages 65-90 based on
nightly voiding episodes:
0 voids 68%
1 void 60%
2 voids 50%
3+ voids 22%
Association of Nocturia and
Mortality: Results From the
Third National Health and
Nutrition Examination Study
19. 19
Clinical Trial Design
Inclusion Criteria
• Those who use bathroom 3+ times/night
• Those with elevated PGE2 levels
Exclusion Criteria
• Congestive heart failure, diabetes, etc.
• Those with major arthritis or muscle pains
13 week double blind crossover study
• Each patient tests all alternatives (to normalize variables)
• Nocturol, APAP, ibuprofen, placebo
• Show Nocturol’s superiority in reducing nocturia
• Delay time to first awakening
• Improve quality of life
20. 20
Series A vs Series B
Both share 50% of returns until made whole for initial investment
Series A thereafter gets returns based on $1.10/share
Series B gets 50% until 15X invested $ back (1-4 years)
Inflated returns come from Founders, not Series A
Series B thereafter get returns based on $2.20/share