Alzheimers Association Small Company Consortia AASCC Meeting 2015

Alzheimer’s Association Small Company Consortia (AASCC) Meeting @ 2015 AAIC Questionnaire Results & Poster Jim Hendrix, Alzheimer’s Association Richard Margolin, Cere. Spir Joseph A. Araujo, Inter. Vivo Solutions Kira Sheinerman, Diami. R Dennis Van Epps, Nano. Somi. X July 21, 2015

Mission 12 10 8 6 4 2 0 r he Ot t or pp Su no ag Diagnostics 28% Other 6% Therapeutics 55% Di ap e er Th st ic ics s Support 11% ut Number of Companies Company Profiles q Currently, 20 member companies in the consortium. q 15 companies completed questionnaires. q The majority of the companies in the consortium are therapeutic companies.

Funding: Current, Desired 3, 5 Rank Sum/ # of Companies 3 2, 5 2 Current Funding 1, 5 12 -18 mo. Strategy 1 0, 5 0 Gov. Grants Found. Grants Angel VC Pharma Partner q A significant number of member companies indicated that currently they have only one main source of funding. q Angel investors and pharma partners represented the greatest ranked current funding sources followed closely by VCs. q Government grants and pharma partners are most highly ranked targets by member companies as their primary funding strategy over the next 12 to 18 months, consistent with a preference for a non-dilutive funding.

Funding: How much do funding difficulties impede your company’s business goals? 5 = greatest difficulty; 1 = no difficulty Funding Challenges 4, 5 4 3, 5 3 2, 5 2 1, 5 1 0, 5 0 5 4 3 2 1

Collaborations & Partnerships Type of Business /Partnership Frequency Sought (%) Pharma Partner 29 Other Biotech 21 Government Grants 17 CRO 12 Foundation Grants 9 Angel 7 VC 5 q Three companies currently participate in precompetitive collaborations/consortia other than the ASCCC q Four currently collaborate with academic groups

Collaborations & Partnerships within AASCC q. Would you consider collaborations with one or more of the AASCC member companies? – Unanimous “YES”

Recognition & Visibility: how important is visibility to funders or strategic partners to your company’s business goals in the next 2 years? Visibility Very Important (12) Moderate Importance (3) Not Very Important (2) q A higher percentage of member companies indicated that visibility is very important.

Recognition & Visibility: publications by the AASCC Members Publications 50 45 40 35 30 25 20 15 10 5 0 2013 2014 2015 q Large # of publications demonstrate that member companies are driven by science, fundamentals q 13/15 companies indicated they are willing to collaborate on joint publications with other consortium members

Recognition & Visibility: does your company present or attend industry and investor conferences? Issue press releases? Employ a PR firm? 12 10 8 6 Yes 4 No 2 0 Conferences Press Releases PR Firm q Conferences Mentioned: AAIC, AD/PD, AAN, BIO, ACS, Sf. N, Brown University Day of Biology, The Early Stage Life Sciences Conference, NEALS, Investment Banking Conferences, JP Morgan, NY Life Sciences Summit, NYC Med Tech, ISSX, ESP, CHI Drug Discovery, SLAS, World Health, Goldman Sachs, NYAS, Angel Venture Fair in New York, Bio. NJ q Other strategies included: Website, Linked. In messages, Networking Events, Membership in major consortia with Pharma and Academia, Invited interviews with industry publications (e. g. genomeweb) q Analysis: Member companies are less likely to use PR firms for achieving visibility

Information and Knowledge Type of Information Ranked Most Important Reg. Biomark Information Most Difficult to Obtain 8% Regs. DX Ind. News 5% 9% Regs. Ther. 8% Reg. Biomark 14% Sci. Journ. 17% Regs. Ther. 14% Comp. Intel. 10% Sci. Journ. 48% Comp. Intel. 67% q ~50% of member companies ranked scientific journals as the most important type of information for success of their business. q ~70% of member companies ranked competitive intelligence as the most difficult type of information to obtain for success of their business.

Information & Knowledge: are you interested in developing a network of the member companies to share information about products, vendors, services and/or consultants commonly utilized by small biotech -related companies? 16 14 12 10 Yes 8 No 6 4 2 0 Products Services Consultants

Challenges Beyond Funding CHOOSING PRE-CLINICAL MODEL – Finding the most convincing collection of data points/assays for successful out-licensing of lead – Conducting preclinical studies that will predict and inform success in the clinic – Model validation – Access to AD mouse models, access to reagents • INFORMATION ACCESS, EXPERT ADVICE – Access to up-to-date information related to studies and clinical trials in the planning stages – Information on early stage in vitro / ex vivo models developed at other companies – Identification of “trusted” experts on specific issues relevant to drug development in AD space • CLINICAL TESTING – Clinical trial enrollment; keeping patients in the study – Clinical validation – Getting into clinic as soon as possible • NETWORKING, EFFECTIVE COMMUNICATION – Create exposure / Visibility for expanding operations – Getting a seat at the table with larger players for a meaningful dialogue – Networking with other companies in AD space

Other Topics Indicated as Potentially Relevant and Helpful to the Mission & Development of the Consortium q An event bringing small companies and potential funding groups together. q Shared resource model – e. g. sharing control data to reduce study costs, sharing industry reports, etc. q Means of enhancing visibility for small companies with new technologies and advancing the field based on scientific publications relevant to each company and of interest to the general AD community and pharmaceutical companies working in the field: sponsored presentations, webinars for new technology etc. q General framework for “pre-competitive” collaboration (to be used as a starting point in drafting specific agreements) – to streamline, expedite, provide a starting point for negotiations. q Databank of “trusted” experts on specific issues for drug development in AD space: preclinical; specific IND enabling studies currently required by FDA; appropriate clinical study design; regulatory requirements in other countries: Europe, Japan, Canada, Australia, China. q Member access to competitive intelligence resources (e. g. limited access per member company; access fees paid jointly by companies wishing to have access to the resource). q AASCC Blog with contributions from member companies: relevant news, announcements, discussions of recent scientific papers, requests for help, success stories.

Do you have a success story that you would be willing to share with the AASCC? q “A couple fundraising stories as well as stories about how we have designed our studies and thought about translatability. ” q Stories about setting up collaborations and meeting with pharma. q Non-dilutive funding experiences. q The value of dog model in helping companies raise subsequent funding and licensing to the veterinary industry.

Consortium Name: are you happy with “Alzheimer’s Association Small Company Consortium”? q 7 / 15 indicated “Yes” q Proposed Alternatives: – SMEs- AA SMEC (to include medium size companies) – Alzheimer’s Association Growth Company Consortium – Alzheimer's Disease Biotech Consortium

Poster Information q Session: Developing Topics, Poster #6209 q Talk Title: P 4 -280: Maximizing the Potential of Small Companies in Alzheimer's Disease Research: Challenges and Opportunities q Date: Wednesday, July 22 q Time: 9: 30 AM – 4: 15 PM q Location: Walter E. Washington Convention Center, Exhibit Hall D