NM-96: A Small Molecule For The! Treatment of Acute Sickle Crisis!! Life Science Summit 10/29/14! Steven Isaacman, Ph.D.!
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1 NM-96: A Small Molecule For The! Treatment of Acute Sickle Crisis!! Life Science Summit 10/29/14! Steven Isaacman, Ph.D.!! Founder & CEO!! Nanometics LLC!
2 About Nanometics! Founded in 2008 with focus on dermatology Divested this business into PHD Skin Care!
3 About Nanometics! Founded in 2008 with focus on dermatology Divested this business into PHD Skin Care!
4 About Nanometics! Oncology Hematology Mission Statement: develop therapeutics for diseases with high unmet medical need.!
5 About Nanometics! Oncology Hematology Mission Statement: develop therapeutics for diseases with high unmet medical need.! Specialize in the development of early stage technologies.!
6 About Nanometics! Oncology Hematology Mission Statement: develop therapeutics for diseases with high unmet medical need.! Specialize in the development of early stage technologies.!! Funded by ~$5 million since 2010 from the National Cancer Institute, National Heart Lung and Blood Institute, the Avon Foundation for Women and NYS CAT.!
7 About Nanometics! Oncology Hematology Mission Statement: develop therapeutics for diseases with high unmet medical need.! Specialize in the development of early stage technologies.!! Funded by ~$5 million since 2010 from the National Cancer Institute, National Heart Lung and Blood Institute, the Avon Foundation for Women and NYS CAT.! NM-96 project is funded by $3 million from NHLBI.!
8 About Nanometics! Oncology Hematology Mission Statement: develop therapeutics for diseases with high unmet medical need.! Specialize in the development of early stage technologies.!! Funded by ~$5 million since 2010 from the National Cancer Institute, National Heart Lung and Blood Institute, the Avon Foundation for Women and NYS CAT.! NM-96 project is funded by $3 million from NHLBI! TRACK RECORD: Nanometics sells polymers, OTC drugs, and skin care products globally and has an extensive network of collaborators and distributors in pharmaceuticals and personal care. The NM-96 molecule has demonstrated safety in 450 patients under the sponsorship of another pharmaceutical company. Nanometics has methods of use IP for SCD.!
9 NM-96 Program! Value Proposition: There is no approved treatment for sickle crisis, and 80,000 hospital admittances per year in the USA alone.!
10 NM-96 Program! Value Proposition: There is no approved treatment for sickle crisis, and 80,000 hospital admittances per year in the USA alone.! NM-96 is being developed as an acute therapy to resolve crisis and decrease the duration of hospital stay.!
11 NM-96 Program! Value Proposition: There is no approved treatment for sickle crisis, and 80,000 hospital admittances per year in the USA alone.! NM-96 is being developed as an acute therapy to resolve crisis and decrease the duration of hospital stay.! NM-96 is an orally available small molecule with demonstrated safety in 450 patients under the sponsorship of a different pharmaceutical company.! Developing IV form mitigates concerns regarding pricing strategy conflicts and eliminates off-label use concerns.! Developing Oral form will allow us to utilize more of the existing data from the other pharmaceutical company.!
12 NM-96 Program! Value Proposition: There is no approved treatment for sickle crisis, and 80,000 hospital admittances per year in the USA alone.! NM-96 is being developed as an acute therapy to resolve crisis and decrease the duration of hospital stay.! NM-96 is an orally available small molecule with demonstrated safety in 450 patients under the sponsorship of a different pharmaceutical company.! Developing IV form mitigates concerns regarding pricing strategy conflicts and eliminates off-label use concerns.! Developing Oral form will allow us to utilize more of the existing data from the other pharmaceutical company.! Recently raised $3,000,000 from the National Heart Lung and Blood Institute.!
13 NM-96 Program! Value Proposition: There is no approved treatment for sickle crisis, and 80,000 hospital admittances per year in the USA alone.! NM-96 is being developed as an acute therapy to resolve crisis and decrease the duration of hospital stay.! NM-96 is an orally available small molecule with demonstrated safety in 450 patients under the sponsorship of a different pharmaceutical company.! Developing IV form mitigates concerns regarding pricing strategy conflicts and eliminates off-label use concerns.! Developing Oral form will allow us to utilize more of the existing data from the other pharmaceutical company.! Recently raised $3,000,000 from the National Heart Lung and Blood Institute.! Methods of use IP filed and co-assigned to Nanometics and AECOM!
14 NM-96 Program! Value Proposition: There is no approved treatment for sickle crisis, and 80,000 hospital admittances per year in the USA alone.! NM-96 is being developed as an acute therapy to resolve crisis and decrease the duration of hospital stay.! NM-96 is an orally available small molecule with demonstrated safety in 450 patients under the sponsorship of a different pharmaceutical company.! Developing IV form mitigates concerns regarding pricing strategy conflicts and eliminates off-label use concerns.! Developing Oral form will allow us to utilize more of the existing data from the other pharmaceutical company.! Recently raised $3,000,000 from the National Heart Lung and Blood Institute.! Methods of use IP filed and co-assigned to Nanometics and AECOM! Currently working with the NHLBI SMARTT program to clear an IND and our goal is to find a partner for clinical trials.!
15 Sickle Cell Disease and Sickle Crisis! SCD results from a single point mutation that weakens the affinity of hemoglobin for oxygen and enables polymers of hemoglobin to form and distort the shape of the red blood cell (RBC).! Orphan disease most common in African, Mediterranean or Asian ancestry.! Over 300,000 babies born each year with SCD.! 3892 BLOOD, 5 DECEMBER 2013 x VOLUME 122, NUMBER 24!
16 Sickle Cell Disease and Sickle Crisis! SCD results from a single point mutation that weakens the affinity of hemoglobin for oxygen and enables polymers of hemoglobin to form and distort the shape of the red blood cell (RBC).! Orphan disease most common in African, Mediterranean or Asian ancestry.! Over 300,000 babies born each year with SCD.! A cascade ensues that damages the endothelium, and causes leukocytes and RBC to adhere in vasculature. Vaso-occlusion (VOC) and painful episodes of sickle crisis result.! 3892 BLOOD, 5 DECEMBER 2013 x VOLUME 122, NUMBER 24!
17 Sickle Cell Disease and Sickle Crisis! SCD results from a single point mutation that weakens the affinity of hemoglobin for oxygen and enables polymers of hemoglobin to form and distort the shape of the red blood cell (RBC).! Orphan disease most common in African, Mediterranean or Asian ancestry.! Over 300,000 babies born each year with SCD.! A cascade ensues that damages the endothelium, and causes leukocytes and RBC to adhere in vasculature. Vaso-occlusion (VOC) and painful episodes of sickle crisis result.! Less symptomatic events cause other debilitating acute and chronic effects.! 3892 BLOOD, 5 DECEMBER 2013 x VOLUME 122, NUMBER 24!
18 Sickle Cell Disease and Sickle Crisis! SCD results from a single point mutation that weakens the affinity of hemoglobin for oxygen and enables polymers of hemoglobin to form and distort the shape of the red blood cell (RBC).! Orphan disease most common in African, Mediterranean or Asian ancestry.! Over 300,000 babies born each year with SCD.! A cascade ensues that damages the endothelium, and causes leukocytes and RBC to adhere in vasculature. Vaso-occlusion (VOC) and painful episodes of sickle crisis result.! Less symptomatic events cause other debilitating acute and chronic effects.! Significant financial stress on the United States Healthcare system, with annual costs exceeding $1.1 billion.! 3892 BLOOD, 5 DECEMBER 2013 x VOLUME 122, NUMBER 24!
19 NM-96 in BERK Models of Disease! A single dose of NM-96 mitigates endothelial activation and improves hemodynamic properties to near wild type levels!
20 NM-96 in NY1DD Models of Acute Crisis! A single dose of NM-96 mitigates the effects of sickle crisis!
21 Summary! Summary and Conclusions! NM-96 is an orally available, water soluble, low MW small molecule.!
22 Summary! Summary and Conclusions! NM-96 is an orally available, water soluble, low MW small molecule.! Program is significantly de-risked by the extensive suite of data available in regards to the pharmacological, toxicokinetic and safety pharmacology of administered NM-96 in human clinical trials.!
23 Summary! Summary and Conclusions! NM-96 is an orally available, water soluble, low MW small molecule.! Program is significantly de-risked by the extensive suite of data available in regards to the pharmacological, toxicokinetic and safety pharmacology of administered NM-96 in human clinical trials.! A single oral, IP or IV dose of NM-96mitigates vascular endothelial activation in BERK or NY1DD mice and improves hemodynamic parameters to near wild-type (C57-BL) levels.!
24 Summary! Summary and Conclusions! NM-96 is an orally available, water soluble, low MW small molecule.! Program is significantly de-risked by the extensive suite of data available in regards to the pharmacological, toxicokinetic and safety pharmacology of administered NM-96 in human clinical trials.! A single oral, IP or IV dose of NM-96mitigates vascular endothelial activation in BERK or NY1DD mice and improves hemodynamic parameters to near wild-type (C57-BL) levels.! Broad community support from NHLBI significantly de-risks the program.!
25 Summary! Summary and Conclusions! NM-96 is an orally available, water soluble, low MW small molecule.! Program is significantly de-risked by the extensive suite of data available in regards to the pharmacological, toxicokinetic and safety pharmacology of administered NM-96 in human clinical trials.! A single oral, IP or IV dose of NM-96mitigates vascular endothelial activation in BERK or NY1DD mice and improves hemodynamic parameters to near wild-type (C57-BL) levels.! Broad community support from NHLBI significantly de-risks the program.! GMP material available on multi-kilogram scale.!
26 THANK YOU Steven Isaacman, Ph.D.! 1400 Old Country Road, Suite 309! Westbury, New York 11590! P ! steve@nanometicslab.com! 26
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