Advanced Chemistry for Pharmaceutical Progress

Team: Kiel Neumann (EL)

Stephen DiMagno (PI)

Allan Green (Mentor)

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PET is a non-invasive medical diagnostic technique for cardiac, brain, and tumor imaging

GFP technology makes new (unknown) and known (but clinically inaccessible) [18F]-labeled radiotracers readily available

Fast, multiplatform, high efficiency synthesis of these fleeting, precious agents.

Initial target indications: pediatric neuroblastoma, Parkinson’s disease.

General methodology for

adding fluorine to lead compounds of

interest

The Business Model Canvas

Accessibility (RCY)PuritySpeedPET/SPECTMultiplatformSensitivity (nca)Specific compounds

IPPoP dataRegulatory planUnderstanding of the regulatory process

Contract cGMP precursor manufactureSalary, RentsClinical trials

SOPs for precursors and drugsRecruit clinical sitesIn vivo animal studiesDevelop regulatory plan for pre IND meetingID cGMP CROFund-raising

cGMP manufacturerRadiopharmaciesNuclear Medicine and Radiology departments

Technical Assistance (Image Atlas)FDA regulatory support

Radiopharmacies

Equipment producers

Prescribing physicians

Radiologist who perform studies

Sales of intermediates

Technology license

Product license (royalty)

Direct sales of precursor

R&D and clinical studies presented in journals and meetings

Drug developers

Pharmaceutical development companies

IPPoP data

Radiologists

Technical assistance

I-Corps 10/11/11 4

1) Radiologists and Nuclear Medicine Physicians2) Radiopharmacy companies (Cardinal Health, Siemens, GE

Healthcare, IBA, AAA)3) Equipment manufacturers (GE, Philips, IBA, Advion)4) cGMP manufacturers

1) Pharmaceutical companies2) Radiologists and Nuclear Medicine Physicians

- Face to face with attending Radiologist at Stanford University

- Face to face with radiopharmacist at UCSF

- Conference call with Nuclear Radiologist at Memorial Sloan Kettering

- Conference call with president of medium size drug company with PET product at the FDA

- Telephone conference with cGMP facility

I-Corps 10/12/11 5

I-Corps 10/12/11 6

- Immediate need for our product

- Currently used SPECT product for neuroblastoma is limited by absence of correlative CT data

- Our lead PET agent would provide more information on existing imaging equipment base

- Two customers offered to participate in clinical trials

- Potential for further development of other tracers identified in interviews

- Actual need for the general procedure

- Allow access to previously unknown tracers

Face to Face meeting with Product Portfolio Manager for PET Radiopharmacy products

One of three largest U.S./E.U. PET radiopharmacy chains and instrument producers

Immediate interest in one of three PoP products we studied

Prepared to sign NDA for potential arrangements providing use of our technology in their proprietary modular synthesizers

Face to face meeting with head of radiochemistry and R&D of world’s largest PET chemicals supplier

Supply precursors in cGMP format to as broad a market as possible

Interested in new technology and willing to sign NDA

Need to assess value of this potential customer

I-Corps 10/18/11 7

Face to Face meeting with head of R&D of Synthra

German company producing proprietary PET modular synthesizers – meet European GMP standards

Would like to develop GMP-compliant modules for two of our three PoP compounds studied

Face to face meeting with Executive Director of Scott Tech Center in Omaha, NE

Develop and facilitate commercialization of technology

Would provide financial support for startup

Meeting set today at 4:30

I-Corps 10/18/11 8

Conference call with top 40 Fortune 500 chemical distribution company Open to cGMP production of our potentially proprietary precursors Interested in developing a general “plug-and-play” cassette

Would allow implementation of our methodology and precursors for any radiochemistry module

Important for FDA compliant production of any drug used in patient diagnostics

Face to face meeting with Director of Business Development of a leading drug discovery outsourcing company Discussed preclinical studies and contract manufacturing of

proprietary intermediates

- Face to face with former Director of Chemistry of major pharmaceutical company- Significant interest in general methodology application to proprietary

compound syntheses

I-Corps 10/18/11 9

- Initial value proposition rendered pivot focusing on product-driven rather than methodology-driven opportunities- Key learnings from new potential customers

- Significant interest in general technology for radiofluorinating broad classes of drugs

- Process is scalable but would require more resources (synthetic chemists)

- Need to identify targets

- Re-Pivot?

I-Corps 10/18/11 10

-Initially seeking to market method technology

-too diffuse, but many opportunities (i.e. product-driven opportunities more than general technology-driven)

-Need to identify specific imaging product opportunities

-Validated hypothesis for immediate need of tracers

-Raised question on identity of lead compound pipeline for Parkinson’s disease

-Recruited two potential partners for clinical trials

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I-Corps 10/11/11 12

Approximately 2.2 million procedures in the US.

Drug costs range from $700 (on-patent) to ~$150 (generic FDG)

US sales of radiopharmaceuticals for PET and SPECT $1.2 billion

US sales expected to grow to $6 billion by 2018

Global numbers approximately 2x

Source: Bio-Tech Systems Report #330; data for 2010.

I-Corps 10/11/11 13

2500 installed PET scannersPET radiopharmacies cover the entire US marketRadiopharmacies have an interest in proprietary agents as a basis of competition in their market.

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Neuroblastoma

Prevalence: about 6000 US cases about 1000 new cases per year

Subjects receive 3-6 images/yearto follow response to therapeutic protocols

World market at U.S. x 2 gives potential of 40,000-70,000 scans/year

Drug costs $500/per gives ~$20 - $35 M

Parkinson’s Disease

DatSCAN sales in Europe ~$100 M

The world's highest recorded prevalence of Parkinson's Disease of any region is in Nebraska, with 329.3 people per 100,000 population

US – 600,000 patients 1 scan per year @ $500 = $300 M