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OPHTHALMOLOGY INNOVATION SUMMIT (OIS@AAO) OCTOBER 25, 2018 CHICAGO, IL

OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

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Page 1: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

OPHTHALMOLOGY INNOVATION SUMMIT (OIS@AAO)

OCTOBER 25, 2018

CHICAGO, IL

Page 2: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

2CONFIDENTIAL AND PROPRIETARY |

Neurotech Pharmaceuticals, Inc.

• Privately owned life science company§ Lowy Family Group (LFG) – 100% owners

• Focused on treating chronic eye disorders• Products based on a robust technology platform.

§ Encapsulated Cell Therapy (ECT)

• Two mid-to-late staged product opportunities in clinical development with promising human clinical data.

Page 3: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

3CONFIDENTIAL AND PROPRIETARY |

Neurotech Team/Collaborators

Management• Rich Small, CEO• Ken Mandell, MD, PhD, CMO• Konrad Kauper, VP of

Development• Cahil McGovern, PhD, VP of

Manufacturing

Board of Directors• Jim Mazzo, Chairman• David Fuchs, LFG• John Fanning, LFG• Rich Small, CEO

Key Collaborators• Lowy Medical Research

Institute (LMRI) Marty Friedlander, MD, President

• Jeff Goldberg, MD, PhD Chairman of Ophthalmology at the Byers Eye Institute at Stanford

MacTel Joint Steering Committee• Marty Friedlander, MD• Emily Chew, MD (NEI)• Alan Bird, MD (Moorfields)• Tom Hohman, PhD,

Neurotech advisor• Ken Mandell, MD, PhD• John Fanning• Rich Small

Page 4: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

4CONFIDENTIAL AND PROPRIETARY |

Platform TechnologyEncapsulated Cell Therapy (ECT)

• Safely implanted in ~ 300 patients.

• Viable in vivo for more than two years.

• Ability to deliver a variety of different therapeutic proteins.

Intraocular implant that contains genetically engineered cells designed to release therapeutic proteins in a controlled, continuous delivery to the back of the eye.

Page 5: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

5CONFIDENTIAL AND PROPRIETARY |

Renexus® (CNTF) – Clinical Programs

• Renexus® releases ciliary neurotrophic factor (CNTF), a well studied neuroprotective agent shown to slow photoreceptor cell death in numerous animal models. • Renexus® is currently being developed to treat:

§ Macular Telangiectasia (MacTel) § Currently in Phase 3§ Program partnered with and financed by LFG

§ Glaucoma§ Currently in Phase 2§ Large market partnering opportunity

Page 6: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

6CONFIDENTIAL AND PROPRIETARY |

RENEXUS® MACULAR TELANGIECTASIA (MacTel)

Page 7: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

7CONFIDENTIAL AND PROPRIETARY |

Macular Telangiectasia (MacTel)

• Bilateral retinal disease that leads to a gradual loss of central vision.§ Onset of disease between age 40

to 60.

• Loss of photoreceptors in a well defined area of the retina. (MacTel Ellipsoid Zone)• No current treatment available

for MacTel.

Page 8: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

8CONFIDENTIAL AND PROPRIETARY |

Renexus® MacTelPositive Phase 2 Clinical Results• 67 participants (99 study eyes) multi-center, sham-controlled study.

24 month endpoints.

• Primary endpoint - Change in the MacTel Ellipsoid Zone

§ Statistically significant reduction in the rate of change in the MacTel

Ellipsoid Zone in the Renexus® treated eyes as compared with the sham

eyes.

• Positive Secondary endpoint outcomes

§ Microperimetry

§ Reading Speed

§ Macular thickening in treated group vs thinning in sham group.

§ No difference in BCVA (safety).

• Renexus® was well tolerated.

Page 9: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

9CONFIDENTIAL AND PROPRIETARY |

Renexus® MacTel Phase 3 Program Initiated• Two 112-patient multicenter, multinational (US/EU/AU), sham-

controlled 24 month studies

• Primary endpoint

§ Change in the MacTel Ellipsoid Zone area.

• Secondary endpoints

§ Microperimetry

§ Reading Speed

§ BCVA (safety)

• Enrollment to be completed mid-2019

• Clinical results expected mid-2021

Page 10: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

10CONFIDENTIAL AND PROPRIETARY |

RENEXUS ® GLAUCOMA

Page 11: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

11CONFIDENTIAL AND PROPRIETARY |

Treated

• Loss of retinal ganglion cells (RGCs) is characteristic of the disease.

• Numerous animal studies demonstrate CNTF’s role in reducing the loss of RGCs.

• Can Renexus® treatment have a beneficial effect on glaucoma patients?

Glaucoma - Trophic Factor Rescue of Retinal Ganglion Cells (RGCs)

CNTF Attenuates the Loss of RGCs in Axotomized Adult Mice1

Sources: 1. Flachsbarth et.al. Invest. Ophthalmol Vis Sci. 2014 Sep 30; 55(11):7029-39;

Page 12: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

12CONFIDENTIAL AND PROPRIETARY |

Renexus® Glaucoma Clinical Program

• Renexus® is currently being tested in a 54 patient sham-controlled Phase 2 study § Primary study endpoint - Change in Visual Fields§ Key secondary study endpoints - Change in the ganglion cell and

nerve fiber layers§ Measuring effect over 12 and 24 month time points.

• Interim Phase 2 data (38 patients) promising. § Neuroprotective effect as measured by Visual Fields seen after 12

months. § Complete 12-month data (54 patients) expected by end of 2018

Page 13: OPHTHALMOLOGY INNOVATION SUMMIT CHICAGO, IL · •67 participants (99 study eyes) multi-center, sham-controlled study. 24 month endpoints. ... Versatile platform can be utilized to

13CONFIDENTIAL AND PROPRIETARY |

Neurotech - Summary

• Promising clinical data in two indications.

§ Late stage (Phase 3) clinical program in MacTel.

§ Large market partnering opportunity in Glaucoma.

• Novel platform technology (ECT).

§ Deliver proteins safely over an extended period of time.

§ Versatile platform can be utilized to treat other ophthalmic disorders.

• Outstanding research, clinical, management and financial support teams in place.