Brian Finrow Founder, President & CEO

Transcription

1 Brian Finrow Founder, President & CEO Academia to industrial practice - barriers to commercialization: Navigating US regulatory and market adoption challenges

2 Who is Lumen Bioscience?

3 Lumen Bioscience Overview Lumen s core technology is a new engineerable biotechnology platform for producing complex products for food, cosmetic and industrial uses. Lumen is the first company to successfully engineer Arthrospira platensis (aka spirulina). We use this platform to make things that are impossible or unduly expensive in traditional platforms like yeast or E. coli. Proudly based in Seattle, Washington Founded and Funded March 2017; 16 employees (including 10 PhD s) Technology platform built over 10 years with ~$25M in funding 3

4 Founders BRIAN FINROW CEO Adaptive Biotechnologies AltAir Fuels Cooley, LLP; Venture Law Group Harvard Law School JIM ROBERTS, MD, PHD CSO Director of the Fred Hutch Cancer Research Center Basic Sciences Division Former HHMI Investigator Co-inventor of mammalian cell transformation (Columbia University) 4

5 Examples Pigments and nutritional supplements o Natural pigments require pathways missing in yeast and E. coli, but naturally present in photosynthetic microbes like Spirulina. Antioxidant production is facilitated by unique redox environment of photosynthetic cells. Oral therapeutic delivery o Requires a delivery organism with GRAS status under FDA (human and animal consumption) 5

6 Natural Blue Pigment (Phycocyanin) Used for food production, cosmetics and analytical chemistry A natural replacement for food color Blue #1: Brilliant Blue artificial derived from coal tar or oil banned in the EU in the U.S., food makers are searching for natural replacements due to consumer demand Blue food color derived from natural spirulina is approved for use in the U.S. $110M market (2015), growing 11% annually; supplies are insufficient to meet demand quality is highly variable when derived from natural strains and grown outdoors Lumen s proprietary strain and production process is optimized for: pigment production rate; purity and consistency; cheap extraction Higher grade materials sell for more for cosmetics and research $ /kg (food grade, purity dependent); ~$2,000/kg (cosmetic grade); up to $20,000/kg or more (highly refined analytical grade) 6 Notes: See Brand New Hue: The Quest to Make a True Blue M&M, Malia Wollan, The New York Times, Oct 5, 2016

7 Empirically Verifiable Improvement Spectral measurement of phycocyanin purity Standard Improved 7

8 Index 1. Regulatory Alphabet Soup 2. Market Adoption 8

9 Governing Agencies and Laws in the USA EPA TSCA (Toxic Substances Control Act) FDA FDC&A (Food Drug & Cosmetics Act) National Bioengineered Food Disclosure Standard (Pub. L ) USDA APHIS program (organisms that are possible plant pests) under the Animal Health Protection Act and the Plant Protection Act Virus-Serum-Toxin Act (veterinary drugs) (with FDA s Center for Veterinary Medicine) FMIA, PPIA, EPIA (meat, poultry, eggs) State Laws Vermont GMO labeling law (rendered moot by new federal GMO labeling statute) 9

10 Governing Agencies and Laws in the USA EPA TSCA (Toxic Substances Control Act) FDA FDC&A (Food Drug & Cosmetics Act) National Bioengineered Food Disclosure Standard (Pub. L ) USDA APHIS program (organisms that are possible plant pests) under the Animal Health Protection Act and the Plant Protection Act Virus-Serum-Toxin Act (veterinary drugs) (with FDA s Center for Veterinary Medicine) FMIA, PPIA, EPIA (meat, poultry, eggs) 10

11 Governing Agencies and Laws in the USA EPA TSCA (Toxic Substances Control Act) FDA FDC&A (Food Drug & Cosmetics Act) National Bioengineered Food Disclosure Standard (Pub. L ) USDA APHIS program (organisms that are possible plant pests) under the Animal Health Protection Act and the Plant Protection Act Virus-Serum-Toxin Act (veterinary drugs) (with FDA s Center for Veterinary Medicine) FMIA, PPIA, EPIA (meat, poultry, eggs) 11

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13 Common Sense Does not Enter into It Excluded: enzymes (extracted) other proteins (extracted) oils (extracted) gene knock-outs organisms modified to over-express or include additional copies of endogenous genes? Final rules are still pending 13

14 Compare That to the U.S. Organic Foods Protection Act 14 Source: USDA Policy Memorandum entitled Consistency between Bioengineered Disclosure and the National Organic Program dtd Sept 19, 2016

15 and the similarly broad language under TSCA 15

16 So Here s the Outcome EPA TSCA (Toxic Substances Control Act) FDA FDC&A (Food Drug & Cosmetics Act) National Bioengineered Food Disclosure Standard (Pub. L ) USDA APHIS program (organisms that are possible plant pests) State Laws Vermont GMO labeling law (superseded by new federal statute 16

17 TSCA covers all activity with bioengineered microorganisms including R&D if the organism falls within the definition above. However! There are some exemptions

18 TSCA Exemptions 40 CFR (b): R&D carried out by non-commercial entities E.g., universities, research institutes, governmental agencies Even remote or far-off commercial plans are sufficient if the entity is for-profit 40 CFR : a complete exemption from TSCA 5 for certain R&D activities which are conducted in contained structures, and are subject to regulation by another federal agency E.g., biotechnology research governed by NIH guidelines Not a full exemption: recordkeeping requirements still apply 40 CFR : Experimental Release Application(TERA) exemption process for R&D activities Essentially an abbreviated MCAN submission which is submitted for an individual field test 40 CFR Subpart G: certain microorganisms in certain kinds of closed systems 18

19 Examples of Products Excluded from TSCA 19 Source: Modernizing the Regulatory System for Biotechnology Products: Final Version of the 2017 Update to the Coordinated Framework for the Regulation of Biotechnology (Executive Office of the President)

20 What If I Can t Find an Exemption Under TSCA? Submit to EPA a Microbial Commercial Activity Notice (MCAN) 90 days prior to initiating manufacture or import 20

21 but Just because the product is exempt under TSCA, doesn t mean the production process will be 21

22 U.S. Food and Drug Administration Food Uses Formal pathways: Food or color additive petition 2-4 years Expensive GRAS pathway GRAS: Generally Regarded as Safe Revised GRAS regulations finalized October 2016 Full GRAS review: 180 days Self-determination process: not published; ~4 mos. If preliminary assessment indicates that safety/tox studies are required, minimum ~4 mos. and $100, ,000 to fund the animal studies 22

23 U.S. Food and Drug Administration Cosmetic Uses Hardly regulated Manufacturer bears the risk of ensuring consumer safety Lack of formal regulation does not reduce the burden of ensuring that products are safe In the U.S. the key backstop comes in the form of state-level tort and consumer protection laws 23

24 Preliminary Conclusions: Phycocyanin as Cosmetic Antioxidant EPA TSCA (Toxic Substances Control Act) FDA FDC&A (Food Drug & Cosmetics Act) National Bioengineered Food Disclosure Standard (Pub. L ) Review production plan against TSCA if to be manufactured in US (open pond, greenhouse, PBR) Internal safety studies only (leverage prior published data on this protein) USDA APHIS program (organisms that are possible plant pests) under the Animal Health Protection Act and the Plant Protection Act Virus-Serum-Toxin Act (veterinary drugs) (with FDA s Center for Veterinary Medicine) FMIA, PPIA, EPIA (meat, poultry, eggs) 24

25 Preliminary Conclusions: Phycocyanin as Food Antioxidant EPA TSCA (Toxic Substances Control Act) FDA FDC&A (Food Drug & Cosmetics Act) National Bioengineered Food Disclosure Standard (Pub. L ) Review production plan against TSCA if to be manufactured in US (open pond, greenhouse, PBR) Likely GRAS self-certification leveraging predicate GRAS notification filing previously submitted to FDA USDA APHIS program (organisms that are possible plant pests) under the Animal Health Protection Act and the Plant Protection Act Virus-Serum-Toxin Act (veterinary drugs) (with FDA s Center for Veterinary Medicine) FMIA, PPIA, EPIA (meat, poultry, eggs) 25

26 Preliminary Conclusions: Phycocyanin as Food Colorant EPA TSCA (Toxic Substances Control Act) FDA FDC&A (Food Drug & Cosmetics Act) National Bioengineered Food Disclosure Standard (Pub. L ) Review production plan against TSCA if to be manufactured in US (open pond, greenhouse, PBR) Color Additive Petition: 2-4 years and $$$ USDA APHIS program (organisms that are possible plant pests) under the Animal Health Protection Act and the Plant Protection Act Virus-Serum-Toxin Act (veterinary drugs) (with FDA s Center for Veterinary Medicine) FMIA, PPIA, EPIA (meat, poultry, eggs) 26

27 Preliminary Conclusions: Oral Vaccine Program (Animals) EPA TSCA (Toxic Substances Control Act) TSCA pre-empted by FDA process FDA FDC&A (Food Drug & Cosmetics Act) National Bioengineered Food Disclosure Standard (Pub. L ) USDA APHIS program (organisms that are possible plant pests) under the Animal Health Protection Act and the Plant Protection Act Virus-Serum-Toxin Act (veterinary drugs) (with FDA s Center for Veterinary Medicine) FMIA, PPIA, EPIA (meat, poultry, eggs) Veterinary drug review process 27

28 Take-aways Be realistic: regarding the cost and time required for the right regulatory path build these assumptions into the business plan early Engage experienced and thoughtful advisors: this is not an area for amateurs and autodidacts; preferably advisors who have spent significant time working inside the agency Be prepared for change: the regulatory landscape in this area continues to change rapidly 28

29 Index 1. Regulatory Alphabet Soup 2. Driving Customer Adoption 29

30 Moving from Approved to Commercial Engage with customers early in the development process unlike in human therapeutics, locking down regulatory clearances does not automatically ensure a ready market many customers will want to run their own testing, so engage them early so that wasteful duplication is minimized Prioritize customer needs price is always an issue; people rarely win on quality alone superior price and quality will almost always prevail but large-scale operators will also require reliable supply and consistent quality, maybe even at the expense of higher cost and modestly lower quality 30

31 Many thanks to the special group that makes this possible! 114 th U.S. Congress

32 Brian Finrow Founder, President & CEO

33 Appendix

34 USDA Regulation of Food Items (Eggs, Poultry, Fish, Meat) 34

35 USDA Regulation of Potential Animal and Crop Pests 35

36 FDA Regulatory Landscape 36