rna eye technologies commercialization report

8/4/2019 RNA EYE Technologies Commercialization Report

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RNA-EYE TECHNOLOGIES 1

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RNA-EYE TECHNOLOGIES

BUSINESS PLAN 2009

Rohit Dayal ShahResearch and Development Area

PIGDONS ROAD,

GEELONG-3217

(AUSTRALIA)

PHONE: 0434129518


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TABLE OF CONTENT

Pg.

1. Introduction 112. Scientific Report 12

2.1 Research and Production 14

3. Marketing Report 163.1Business Potential 163.2SWOT Analysis 183.3Marketing Strategies-7 Ps 193.4Special Program 203.5Action Plan 21

4. Legal Report 225. Financial Report 23

5.1Funds for discovery phase 235.2Action Plan- Maturity phase 245.3Finances 25

6. Management Report 287. References 30


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MISSION

To provide excellence in eye and vision research, through investment in

people, systems and innovation. Our mission is to lead efforts to preserve sight

and provide resources to prevent avoidable blindness To achieve this, we work

in partnership with leading eye specialists, medical professionals, industry

representatives, the ophthalmic and allied health community, the public,

business communities and government.

VISION

Our vision is for every person to be given every opportunity to see.


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1.INTRODUCTIONRNA-EYE Technologies is a Geelong, Australia based company that explores the use of

RNA interference in human eye offering competitive prices and expert advice. With this newinnovative RNAi technology, the company is having an opportunity to treat the disease and

impacting the lives of millions of people around the world by silencing the disease causing

genes.

Since past many years, the company through its rigorous research has made remarkable

advancement and considerable understanding of eyes genetic diseases and hence large

number of molecular targets has been obtained. Our company has been well supported by

various grants at different stages of product formulation and phase of company. Apart from

grants, company had also raised funds from venture capitalist Innovation Capital.

Presently there are only few known treatments available for the diseases with therapeutic

modalities such as monoclonal antibodies and small molecules which are unable to treat the

disease and can only temporarily stabilize the condition. With the RNAi technology, any

gene in the genome involved in disease can be targeted. RNA-EYE Technologies has

developed novel, proprietary short interefering RNAs which has number of advantages over

conventional siRNAs as they have good stability against nuclease degradation. Presently, the

company is involved in extensive research in treatment of genetic eyes disorder such as

macular degeneration, Retinitis pigmentosa and Leber's hereditary optic neuropathy. Some of

the products are in the clinical developmental phase.


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2.SCIENITIFIC REPORTThrough exhaustive research, RNA-EYE Technologies has successfully developed its first

product i-FIT since its inception, which is certainly better than any other product associatedwith this disease which will fully maximise its selling potential. Through our gene silencing

technology, the company has found 17 disease targets in 3 tissues and in 3 species including

humans. Other products i-CONES and i-NERVETIS are also in developing phase.

Table 1: List of Products

THERAPEUTIC

AREA COMPOUND/VACCINE TYPE INDICATION PHASE

EYES i-FIT shRNA

Age related macular

degeneration Approved

i-CONES shRNA Retinitis pigmentosa phase-1

i-NERVETIS shRNA

Leber's hereditary

optic neuropathy Phase-11

i-FIT

The product i-FIT (figure 1) is RNAi based treatment for genetic disease Age RelatedMacular Degeneration. As compared to treatments, it does not have RNAi delivery issue as

naked siRNAi can be directly injected in the eye which acts as drug, short strand of RNA that

are not packaged or protected in membranes and quickly break down in blood stream and so

it can reach the target intact.

Figure 1: siRNA Design (i-FIT)

AGE RELATED MACULAR DEGENERATION- DISEASE CAUSE

Wet age related macular degeneration is the main cause of blindness in the elderly. It is dueto the abnormal growth of blood vessels below the macula region that is responsible for the
http://en.wikipedia.org/wiki/Retinitis_pigmentosahttp://en.wikipedia.org/wiki/Leber%27s_hereditary_optic_neuropathyhttp://en.wikipedia.org/wiki/Leber%27s_hereditary_optic_neuropathyhttp://en.wikipedia.org/wiki/Leber%27s_hereditary_optic_neuropathyhttp://en.wikipedia.org/wiki/Leber%27s_hereditary_optic_neuropathyhttp://en.wikipedia.org/wiki/Leber%27s_hereditary_optic_neuropathyhttp://en.wikipedia.org/wiki/Retinitis_pigmentosa


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central vision which is required for fine detail visual activities such as driving, reading,

recognising faces and other day to day activies.

i-FIT has designed and developed by following the procedure described briefly in diagram 2.

Figure 2: Process diagram for designing the siRNA

The guidelines for building siRNA are:

1. Find 21 nt sequences in the target mRNA that begin with an AA dinucleotide.2. Select 2-4 target sequences which can be done at www.ncbi.nlm.nih.gov/BLAST3. Design appropriate controls.

CHEMICAL MODIFICATION

Chemical modification of siRNA duplexes is done to enhance its performance which helps in

increasing the siRNA stability, decrease off-target effect and reduction of cytokine activation.

Chemical modification is done to improve siRNA serum stability against nuclease

degradation which is greatly responsible for siRNA delivery.


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These are the properties of the i-FIT :

Low toxicity Biodegradability Efficient cellular uptake Efficient endosomal escape

2.1 RESEARCH AND PRODUCTION

The physical plant is located at: PIGDONS ROAD,GEELONG-3217 (AUSTRALIA)

Phone: 0434129518

Email:[email protected]

Website: rnaeyetechnologies.com.au

The layout and the production chamber is shown in the figure 3 below:

Figure 3: Laboratory and the production unit
mailto:[email protected]:[email protected]:[email protected]:[email protected]


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QUALITY CONTROL

RNA-EYE Technologies has established a well-deserved reputation by ensuring compliancewith laws and regulations.

A special officer takes care of the quality control of the cell lines that we produce at RNA-EYE Technologies. The Quality control officer works in closely with the Chief Productionmanager to maintain the quality, and the Brand Level of the RNA-EYE Technologies.

DISTRIBUTION

The production department has also a role to play in the distribution of the siRNA product.Clients give the order for making a specific cell lines they need, the specific cell line is

directly delivered to the clients in 12 hours once the product is ready if in Victoria.

If outside Victoria, it will take a min of 24-48 hours for the product to be delivered, oncethe product is ready. RNA-EYE Technologies has got the Distributors in the other parts ofthe Australia.


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3.MARKETING REPORTIn 2003, Wet age related Macular Degeneration market was US$ 357 million dollars to US$

2.0 billion dollars in 2009 as in figure 4. By 2013, it is estimated that the market value willrise in excess of US$ 3.2 billion dollars. As the success development of i-FIT is observed,

other drugs which are similar in nature such as i-CONES and i-NERVETIS for diseases

Retinitis pigmentosa and Leber's hereditary optic neuropathy have good potential thereby

expanding the market.

Figure 4: Growth of the wet age related Macular Degeneration Market

In the year one, as RNA-EYE Technologies has developed innovative breakthrough

technology, the profit from the drug will be maximised through premium pricing of the drug

and also market will have more sales as occurrence of the disease is increasing with the

increasing population.

3.1 THE BUSINESS POTENTIAL

Competitive advantages

RNA-EYE Technologies i-FIT targets VEGF m RNA is the first RNA based technology to

enter the human clinical trials system. It is certainly far better product than conventional

treatment such as with monoclonal antibodies and other small molecules. Product i-FIT

shows considerable improvement in visual acuity of patients which is very encouraging and

exciting. RNA based technology is advantageous over the conventional treatment in

following ways: better efficacy, requires less dosage, and is permanent in nature. It is safe

and effective product as well. Our competitors are listed in table 2.
http://en.wikipedia.org/wiki/Retinitis_pigmentosahttp://en.wikipedia.org/wiki/Leber%27s_hereditary_optic_neuropathyhttp://en.wikipedia.org/wiki/Leber%27s_hereditary_optic_neuropathyhttp://en.wikipedia.org/wiki/Retinitis_pigmentosa


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Table 2: Main competitors of our drug

Due to its above mentioned capabilities, it has great potential to dominate the market once it

is open to the public. RNA-EYE Technologies is aiming for annual sales of $800 million

dollars in the first year of operation. This will be well supported by extensive marketing

channels. Future growth will be driven by expansion of product lines, opening of

commercialization plant in India and exporting the products to developed nations such as US,

UK, Germany, etc.


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3.2 SWOT ANALYSIS

Figure 5: SWOT Analysis

The SWOT analysis is briefly described in figure 5.

STRENGTH

Promotion: In order to promote our product RNA-EYE Technologies organise scientific

seminars and conferences, in which customers are made aware about the benefits of our

product when compared to the other products in the markets.

Expertise: RNA-EYE Technologies have a good experienced and skilled staff, with their

valuable insights and thoughts that have strengthened the organisation with the organisation

and even in the market.

Cost Advantage: As compared to our competitors, our prices are high but overall treatmentcost is less.

Easy to modify: As compared to other treatments, in case of any side effects we can easilychange or modify siRNA.

CheapSubstitutes

Market ShareVariation.

NewTechnology

MarketDemand

Regulations

Brand Name

Reactive asproposed tobeingpredictive

Investors

Promotion

Expertise

Cost-advantage

Easy tomodify

STRENGTH WEAKNESS

THREATSOPPORTUNITIES


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WEAKNESS

Brand Name: RNA-EYE TECHNOLOGIES is a new player in the market. So, customers donot know us much, however by our marketing strategies, we would overcome this

shortcoming.

Reactive as proposed in being proactive: RNA-EYE Technologies will be dealing with problems when occurred or encountered rather than predicting upcoming or other possible problems which can pose a hindrance for the company growth. In doing so, we are notanalysing unpredictable situations. In contrast we will be looking at the possible threatswhich will be present for every company.

OPPORTUNITIES

New Technology: RNA-EYE Technologies utilize latest technologies based on the market

requirements which give us a cutting edge over our competitors.

Market Demand: As other treatments for AMD are not effective and are temporary innature, so we can prove our expertise in the market.

Regulations: Regulations against siRNA has been loosened for the human treatment, and soit is good opportunity for us.

THREAT

Cheap substitutes: Customers view other treatment such as of monoclonal based as cheap

treatment, as they think of short term. However RNA-EYE Technologies will be competing

against these products based on effectiveness.

Market Share Variation: Present market is seeing recession, as we also otherwise know that

market has its pros and cons.

3.3 MARKETING STRATEGY-THE 7 PS

Product: Through exhaustive research, RNA-EYE Technologies has successfully developedits first product i-FIT since its inception, which is certainly better than any other productassociated with this disease which will fully maximize its selling potential. Through our genesilencing technology, the company has found 17 disease targets in 3 tissues and in 3 speciesincluding humans. Other products i-CONES and i-NERVETIS are also in developing phase.

Price: The pricing of the three siRNA product has been calculated based on the amount spentby the Research team and the Production unit. Our three products have been priced as followsin table 3:


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Table 3: Price of products

i-FIT 450 AUD

i-CONES 325 AUD

i-NERVETIS 400 AUD

Place: The product specification being siRNA construct, which is being used in the treatmentof genetic disease Age Related Macular Degeneration. Firstly we would be targeting the

patients in the cities, who can afford the premium price of the treatment. We would contactprivate practitioners as well as hospitals.

Promotion: We have excellent promotional strategies and marketing staff, by which ourproducts have caught the attention of the customers.

Process: Our marketing department have prepared particular process to market our products,we be advertising, as well as informing the potential customers by various mode such as

journals, seminars, articles, etc.

People: We want to give best to customers with what we already have. This requires goodmarket analysis to analyse the needs of the customers and so we have also hired marketresearch specialist.

Physical evidence: Packaging of our product as attractive as the product itself, so thatdamage to the product is minimized. Our company maintains the product at optimumconditions and are also stored appropriately.

3.4 SPECIAL PROGRAMS:

PROMOTIONAL STRATEGIES:

Articles: Various articles (journal & research papers) containing the information of ourproduct have been published.

Brochures: Brochures list of our product have been distributed to patients receiving otherform of treatment by contacting doctors and hospitals.

Seminars & Conferences: Many seminars and conferences are organised at variouslocations to educate people about our product.

Advertisements: Our product is advertised through leading newspapers and news channels.Other forms of advertisements are used as listed above.


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Website: We have created website for company wherein the list of product and itsadvantages have explained in http://www.rnaeyetechnologies.com.au

3.5 ACTION PLAN:

We have established our company, with highly productive siRNA production unit.Tie-up with pharmaceutical giant NOVARTIS and BIOCON would be of great

advantage considering they already have established sales and marketing channels.

Our website contains information describing our product; we plan to elaborate itfurther so that prospective customers can get the product in 5 working days.

As we have Australian based production unit, we plan to set up more distributionchannels for faster and cheaper delivery of our product.

We also plan to do agreement and contracts with research labs and eye hospital suchas ROYAL VICTORIA EYE & EAR HOSPITAL.

Research and discover other drugs/products, for genetic eye diseases such as Lebershereditary optic neuropathy, Diabetic Retinopathy.

It can also start Clinical Trials for eye by collaborating with hospital as it has got allthe facilities for the setup.


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4. LEGAL REPORTIn 2005, provisional patent was obtained, after a year standard patent was attained for fully

commercializing the product. At the same time, international patent search was done to

ensure that the concept or idea does not exist any where. Spin-off Company was created

under the name of RNA-EYE TECHNLOGY after the discovering and conceptualising the

product through Australian Securities and Investment Commission (ASIC) under Section 117

of the corporations ACT 2001. The Corporate Law Reform Act 1998 made the process much

simpler. So that, business can be carried out anywhere in Australia.

In 2008, IP Lawyer from world renowned law firm known as Baker & McKenzie was hired

and International Patent was applied under Patent Co-operation Treaty (PCT) ip#354167 in

many countries including Australia, United States of America, Europe, Canada, India , Japan,

etc) .

Further in 2008, the company was also listed in Australian Stock Exchange (ASX) which is

primary stock exchange in Australia as well as International Stock Exchange (NASDAQ).

Company entry into national and international stock exchange benefits the company, as

increases the market value, increases the profile, capital growth and improved valuation are

observed.

RNA-EYE TECHNOLOGIES has a confidentially policy which prohibits the board of

directors and staff against unauthorised disclose or discussion of any inside information,

except in situations where duty calls for it.

COMMERCIALIZATION OF IP: Primarily there are two kinds of IP, franchising and

licensing, RNA-EYE Technologies prefers the licensing approach. We have entered an

exclusive license agreement with NOVARTIS Pharmaceuticals. The annual royalty is

obtained from the company as the deal. RNA-EYE Technologies has patents for its products

not only in Australia but also in various other countries through the Patent Cooperation

Treaty(PCT), they have been listed in table.

Our patents are listed in table 4:

Table 4: Patents of our products

PRODUCT

PATENT

NUMBER LODGED/STATUS TECHNOLOGY

i-FIT Aus2198332748 Granted siRNA

i-CONES Aus2198365544 Granted siRNA

i-NERVETIS Aus2198674328 Granted siRNA
http://en.wikipedia.org/wiki/Leber%27s_hereditary_optic_neuropathyhttp://en.wikipedia.org/wiki/Leber%27s_hereditary_optic_neuropathy


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5.FINANCIAL REPORT

5.1 FUNDS FOR DISCOVERY PHASE

In this phase, all the research relating to the discovery and development is done.

siRNA design process, target discovery, testing on cell lines, animal models to

test the efficacy, safety, toxicity, pharmacokinetics and metabolism are all done

in this phase. So funds were accumulated in the following manner as given

below briefly.

In 1999: Central Research Grant Scheme (CRGS) 20, 000 AUD.

In 2000: Australian Research Council Grant (ARC) in which Linkage, Infrastructure,

Equipment & Facilities worth 100,000 AUD.

In 2002: National Health & Medical Research Council Grant (NHMRC), in which Standard

Project Grant of 800,000 AUD for 3 years.

In 2005: Provisional Patent cost was 80 AUD and for International Patent Search was 14, 00

AUD.

GROWTH PHASE

Establishment of spin-off company in 2006 named RNA-EYE TECHNOLOGIES tocommercialise the invention. It was created through Australian Securities and

Investment Commission (ASIC) under the corporations ACT 2001.

- FEES: Registration Fees: 660 AUD and Annual Fees : $1600 AUD Moved to Geelong Technology Precinct, under BIODEAKIN initiative. Agreed to pay

GTP, 20 % revenue during the growth phase.

2006- AusIndustry Pharmaceutical Partnership Program(P3) grant of $3.2 million- Commercial Ready Scheme Grant of $648,314

2006- Commercialising Emerging Technologies , (COMET) grant of $64,000- 125% R&D tax concession

Both grants utilised for Clinical Trials Phase I which took 2 years.

2008- The company hired IP Lawyer from Bakers & McKenzie


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- Applied forInternational Patent

Initial Fee: 2000 AUD andAnnual Fee: $100 for 1st 4 years

For 25 countries: $2500 annually

2008- Listed in international Stock Exchange NASDAQ

2008 VENTURE CAPITAL- Short-listed and selected

It is leading Australian Venture Capitalist. INNOVATION CAPITAL

They have $100 million dollars under management. They have team comprising of

entrepreneurs and executives with exceptional personal track record. After obtaining $10.5

million, it was spent on Clinical Trial phase II & III which took 7 years in total.

5.2 ACTION PLAN

MATURITY PHASE

2015: Many Pharmaceutical Companies were approached, Pfizer, Novartis,

Genentech

2016: Alliance was formed with NOVARTI S , so that their marketing channels

could be used for selling purpose.

- $6 billion was paid to NOVARTI S to exit.

- Alliance Payout $15 billion dollars from

As a result, Novartis could commercialise this as well as other products of

RNA-EYE Technologies in the pipeline.


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5.3 FINANCES

Details of the finances that were incurred during initial company setup:

Initial administration costs.Table 5: Initial Administration Cost

Building costs $ 3200000

Utilities (Electricity, cafeteria, toiletries, $ 60000etc)Office supplies $ 30000Wages (annual) $ 1130000Legal expenses $ 90000Insurance (annual) $ 24000

Tax/Super/GST (annual) $ 67000

Total $ 4601000

Research and production costsTable 6: Research and production cost

Cost incurred for research $ 4403610

Cost of machinery and materials $ 2963610required for productionsiRNA room and cold storage $ 2972800

roomCost of general lab equipment $ 83000

Total $ 10423020

PRODUCT COST PRICE & SELLING PRICE

COST PRICE

Table 7: Cost Price of product

i-FIT 350 AUD

i-CONES 255 AUD

i-NERVETIS 300 AUD

SELLING PRICE


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Table 8: Selling Price of product

i-FIT 450 AUD

i-CONES 325 AUD

i-NERVETIS 400 AUD

SALARY

Table 9: Salaries

POSITIONS SALARY PER ANNUM(AUD)

CEO $ 120000SECRETARY $ 100000

LEVEL 1CSO $ 85000CPM $ 82000CLO $ 77000

CMRO $ 75000CMO $ 75000CFO $ 75000

HRM $ 75000LEVEL 2

PRM $ 73000Sr. RESEARCH SPECIALIST $ 70000

LAWYERS $ 67000LEVEL 3

MARKETING EXECUTIVES $45,000LAB TECHNICIANS $ 43000PATENT ADVISOR $ 45000

ACCOUNTANT $ 45000

Break-even analysis

Table 10: Break-even Analysis

Fixed Costs (yearly)

Wages $ 1670000

Insurance $ 47000Maintenance $ 2800Property taxes $ 84000

Other $1765Total Fixed Costs $1805565


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Variable Costs (yearly)

Raw materials $ 3760000

Marketing $ 3578000Packaging and Transport $ 281340Electricity $ 22180Office Supplies $ 16510

Total Variable costs $ 7658030

The above table 5, 6, 7, 8, 9 and 10 shows the cost incurred to produce the product. Thefinancial department will then estimate the actual profit that the company incurs on a yearly

basis. The estimation will be made by comparing the expenses incurred and the profitsgenerated on a yearly basis. We will finally compare the section of the financial plan which

shows our assumed profits to the actual income generated.


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6.MANAGEMENT REPORTORGANISATION CHART

Figure 6: Board Members

The figure 6 shows our board members as listed below:

BOARD MEMBERS

Dr. Rohit Dayal Shah Chief Executive OfficerMiss Katrina Kaif Chief Secretary

Dr. Ashley Bates Chief Scientific Officer

Dr. Morley Muralitharan Chief Legal Officer

Dr Anthony Coulepis Chief Marketing OfficerMr. Dick Norman Chief Financial OfficerMr. James Wates Chief Production Manager


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RECRUITMENT PROCESS

Our recruitment process as in figure 7 is designed to find the right person for the role we needto feel in. Lots of opportunities are given to the candidates, where the individual may asks as

many questions and see whether he fits in our company. Our recruitment is explained brieflyvia the chart.

Figure 7: Recruitment Process

After selection of candidate, adequate training is given and aspects such as Monthly feedback

form, employee profile chart, management review on employee, peer review chart, absence

form, are also prepared and dealt with appropriately. Workshops & conferences are also held

to keep our employees motivated.


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7. REFERENCES

1. www.ambion.com accessed on 10 June, 20092. www.lion-nathan.com.au accessed on 10 June, 20093. www.chemind.org accessed on 10 June, 20094. www.ier.org.au accessed on 10 June, 20095. www.nature.org accessed on 10 June, 20096. www.sirna.com accessed on 10 June, 20097. www.hopkinsmedicine.org accessed on 10 June, 20098. www.pbs.org accessed on 10 June, 20099. www.imgenex.com accessed on 10 June, 200910.www.ausbiotech.org accessed on 10 June, 200911.www.ipaustralia.gov.au accessed on 10 June, 200912.www.nhmrc.gov.au accessed on 10 June, 200913.Gomase V S; Tagore S, 2008, RNAiA Tool for Target Finding in New Drug

Development, Current Drug Metabolism, vol. 9, pp. 241-244

14.RNAi Goes Genomic: Target Identification and Validation with siRNAs, accessedfrom www.ambion.com accessed on 10 June, 2009

15.www.biotechnology.gov.au accessed on 10 June, 200916.www.grantslink.gov.au accessed on 10 June, 200917.www.deakin.edu.au accessed on 10 June, 2009

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