Parkinson s Disease: Gene Therapy as a Novel Therapeutic Strategy. Forecasting Analysis Excerpt

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1 Parkinson s Disease: Gene Therapy as a Novel Therapeutic Strategy Forecasting Analysis Excerpt

2 Gene Therapy is a Lucrative Market

3 Market Analysis: Developmental PD Gene Therapies 5 Major Gene Therapies In Clinical Development AAV2 GDNF (Phase I) AAV-hAADC (Phase I) ProSavin (Phase 2b) Collategene (Phase III) NLX-P101 (Phase II)

4 Market Potential: Nat ional & International Likely Case: 5% Adoption US Cases: 1,000,000 Patients Worldwide Cases: 8,500,000 Patients Patients in Stage 4,5 50,000 Patients in Stage 4,5 425,000 US Market Opportunity: $5.26B World Market Opportunity: $44.7B

5 Market Potential: Nat ional & International Ideal Case: 15% Adoption US Cases: 1,000,000 Patients Worldwide Cases: 8,500,000 Patients Patients in Stage 4,5 150,000 Patients in Stage 4,5 1,275,000 US Market Opportunity: $15.7B World Market Opportunity: $134B

6 Est im at ed Parkinson s Gene Therapy Price Gene Therapy currently manages disease but is expected to cure in future Prosavin: an additional five years before disease progresses further Eliminates recurring medication costs Value-based pricing (discussed later) $105,238 Estimated Cost for Parkinson s gene therapy Eventually, gene therapy will become a staple of 21st century medicine. - Jack McCain, Biotechnology Healthcare 2005

7 Market Potential: Nat ional & International Likely Case: 5% Adoption US Cases: 1,000,000 Patients Worldwide Cases: 8,500,000 Patients Patients in Stage 4,5 50,000 Patients in Stage 4,5 425,000 US Market Opportunity: $5.26B World Market Opportunity: $44.7B

8 Market Potential: Nat ional & International Ideal Case: 15% Adoption US Cases: 1,000,000 Patients Worldwide Cases: 8,500,000 Patients Patients in Stage 4,5 150,000 Patients in Stage 4,5 1,275,000 US Market Opportunity: $15.7B World Market Opportunity: $134B

9 Future trends indicate strong market potential. PD is a unique untapped patient pool Strong sector growth Large pool of investors willing to fund R&D and trial process Industry largely unaffected by economies of scale Unsaturated market Ten Year Sector Growth Comparison Biotech Index Pharmaceutical Margins ~35% ~45% Biotechnology Margins Pharma Index and DOW

10 Pricing is not based on cost of development but on value of drug High ticket drug prices face opposition from insurers Alternative annuity based payment Pricing strategies will focus on value based compensat ion.

Patients enroll in therapy in a 3 year period Assuming that only.

11 Total Revenue will compensate the company over time. Few Things to Consider Assuming that 5% of current Parkinson s Disease Patients enroll in therapy in a 3 year period Assuming that only.5% of new Parkinson s patients undergo therapy Yearly annuity not adjusted for inflation Very conservative model

12 Nearly 3,000 patents have been awarded for genes in the US A patent ensures exclusivity for 20 years during which a drug would be fully exclusive Annuity pricing plan extends the profitability of therapy Competition and generics are not immediate concerns.

13 Sales and marketing will guide profit s. Capturing consumer loyalty for drug is necessary. Annuity pricing is appealing to insurers because of smaller upfront costs and minimal risk. Total marketing costs minimal due to modified oligopoliy. Marketing to Insurers Profits Marketing to Consumers Nearly 90% of physicians were very or somewhat satisfied with the information received from sales representatives.

14 Market Analysis: Developmental PD Gene Therapies 5 Major Gene Therapies In Clinical Development AAV2 GDNF (Phase I) AAV-hAADC (Phase I) ProSavin (Phase 2b) Collategene (Phase III) NLX-P101 (Phase II) Heavy em phasi s on R&D High Investment - with promising drugs and drugs in the pipeline

15 Conclusion The br eakdow n of Par kinson s into subtypes pr esents the oppor tunity for tar geted ther apies. The biggest issue for Par kinson s gene ther apy is the selective Blood Brain Barrier. Gene ther apy is a pr ofitable mar ket w ith high financial potential and VC interest. Ther e ar e new and competitive pr icing mechanisms w hich can offer higher returns and better pricing protection. Pr eliminar y models suggest that even w ith competetor s profits offer fair compensation for drug development The five major gene ther apies have a good potential for market transition.

16 Citations s-disease-medications "Global, Regional, and National Age sex Specific All-cause and Cause-specific Mortality for 240 Causes of Death, : A Systematic Analysis for the Global Burden of Disease Study 2013." The Lancet (2015): Web Mayo Clinic Staff. "Parkinson's Disease." Symptoms. Mayo Clinic, n.d. Web. Rana, Abdul Qayyum, Bilun Naz Böke, Abdul-Rehman M. Qureshi, Mohammad A. Rana, and Maniza Rahman. "Prevalence of Essential Tremor in an Idiopathic Parkinson's Disease Patient Population." Int J Neurosci International Journal of Neuroscience (2015): Web. Hout, A. Van Den, and F. E. Matthews. "Estimating Dementia-free Life Expectancy for Parkinson's Patients Using Bayesian Inference and Microsimulation." Biostatistics 10.4 (2009): Web. Stefanis, L. " -Synuclein in Parkinson's Disease." Cold Spring Harbor Perspectives in Medicine 2.2 (2011): n. pag. Web. Brooks, D. J. "Dopamine Agonists: Their Role in the Treatment of Parkinson's Disease." Journal of Neurology, Neurosurgery & Psychiatry 68.6 (2000): Web. "PARKINSON DISEASE 8, AUTOSOMAL DOMINANT; PARK8." Online Mendelian Inheritance in Man. National Center for Biotechnology Information, n.d. Web. "Summary: Parkinson's Disease 8, Autosomal Dominant." National Center for Biotechnology Information. U.S. National Library of Medicine, n.d. Web..

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