REGULATORY AFFAIRS PROFESSIONALS SOCIETY WEST COAST CONFERENCE & EXHIBITION March 23, 2005 CALIFORNIA STEM CELL RESEARCH AND CURES ACT Michael A. Swit, Esq. Vice President, Life Sciences

2 Proposition Basics Established "California Institute For Regenerative Medicine" To Regulate Stem Cell Research And Provide Funding, Through Grants And Loans, For Such Research And Research Facilities. Created Constitutional Right To Conduct Stem Cell Research – Adds Article Xxxv To California Constitution – “Medical Research” Prohibited Institute's Funding Of Human Reproductive Cloning Research. Established Oversight Committee To Govern Institute.

3 Proposition 71 – Basics … Provides General Fund loan up to $3 million for Institute's initial administration/implementation costs. Authorizes issuance of general obligation bonds to finance Institute activities up to $3 billion subject to annual limit of $350 million. Appropriates monies from General Fund to pay for bonds.

4 Proposition Specifics The institute shall have the following purposes: –(a) To make grants and loans for stem cell research, for research facilities, and for other vital research opportunities to realize therapies, protocols, and/or medical procedures that will result in, as speedily as possible, the cure for, and/or substantial mitigation of, major diseases, injuries, and orphan diseases. –(b) To support all stages of the process of developing cures, from laboratory research through successful clinical trials. –(c) To establish the appropriate regulatory standards and oversight bodies for research and facilities development.

5 Stem Cell Research Stem cell research -- includes research involving adult stem cells, cord blood stem cells, pluripotent stem cells, and/or progenitor cells. –Pluripotent stem cells – cells capable of self-renewal have broad potential to differentiate into multiple adult cell types may be derived from somatic cell nuclear transfer or from surplus products of in vitro fertilization treatments when such products are donated under appropriate informed consent procedures. –Progenitor cells -- multipotent or precursor cells that are partially differentiated, but retain the ability to divide and give rise to differentiated cells.

6 Covered Research Per “Purpose & Intent” – scientists at California universities and other advanced medical research facilities Maximize the use of research funds by giving priority to stem cell research that has the greatest potential for therapies and cures, specifically focused on pluripotent stem cell and progenitor cell research among other vital research opportunities that cannot, or are unlikely to, receive timely or sufficient federal funding, unencumbered by limitations that would impede the research.

7 California Institute For Regenerative Medicine -- CIRM Established in early 2005 with the passage of Proposition 71 – to administer Prop. 71 The Independent Citizens Oversight Committee ("ICOC") – –29-member governing board for the Institute; represent: California's leading public universities Non-profit academic and research institutions Patient advocacy groups – specifically -- spinal cord injury and Alzheimer’s Disease, type II diabetes, multiple sclerosis or amyotrophic lateral sclerosis, type I diabetes and heart disease, : cancer and Parkinson’s disease, mental health disease, and HIV/AIDS Biotechnology industry

8 CIRM … ICOC Chairs –Chair -- Robert Klein, Klein Financial Group –Vice Chair -- Ed Penhoet, Ph.D. President, Gordon & Betty Moore Foundation Interim President – Zach Hall – ex-associate dean of medical research, USC Working Groups: –Scientific and Medical Research Funding Working Group. –Scientific and Medical Accountability Standards Working Group. –Scientific and Medical Research Facilities Working Group.

9 Key Issues Facing CIRM Sunshine on process – –Meetings Two public meetings per year required Subject to Bagley-Keene Open Meeting Act for all meetings – although some meetings can be closed – specific examples: –Patient confidentiality –Trade secret information –Prepublication scientific information –Personnel actions involving CIRM employees Grants and loans must be adopted in open meetings –Records – California Public Records Act applies Some restrictions – similar to those under public meeting –Must use competitive bidding for services

10 Key Issues Facing CIRM … Location of CIRM – candidates include: –San Francisco –San Diego –Los Angeles –Sacramento Can non-stem cell research be supported – see Slide 4 -- “for other vital research opportunities”

11 Key Issues Facing CIRM … What can be funded –Preference – ensure funding does not duplicate or supplant existing funding, high priority shall be placed on funding pluripotent stem cell and progenitor cell research that cannot, or is unlikely to, receive timely or sufficient federal funding, unencumbered by limitations that would impede the research –“Vital research opportunity” -- scientific and medical research and technologies and/or any stem cell research not actually funded by the institute under [first bullet point] which provides a substantially superior research opportunity vital to advance medical science as determined by at least a two-thirds vote of a quorum of the members of the Scientific and Medical Research Funding Working Group and recommended by WG to ICOC

12 Key Issues Facing CIRM … What Can Be Funded –Restrictions on what therapies can be covered Text says -- to realize therapies, protocols, and/or medical procedures that will result in, as speedily as possible, the cure for, and/or substantial mitigation of, major diseases, injuries, and orphan diseases. –“major” – appears to modify diseases and injuries? –Are “minor” diseases and injuries eligible for support? –Are improvements that are incremental, but not substantial, eligible even if for “major diseases, injuries, and orphan diseases”

13 Key Issuing Facing CIRM … What Can Be Funded –Restrictions on what therapies can be covered … But, see Slide #6 – from Purposes & Intents Section of Prop. 71 – makes clear CIRM should maximize its dollar; but does that also require outcomes be used in “triage” of proposals? –Won’t fund anything funded by NIH

14 Key Issues Facing CIRM … What Can Be Funded – –Research funding -- any distinctions based on: For profit vs. not-for-profit research? In California or not – can part benefit a non-California firm? –Facilities and Equipment Funding -- Grantee must be a not-for-profit Remember -- must be in California to get $ –Split between research and facilities – 90/10

15 Key Issues Facing CIRM … Revenues from products developed with CIRM funds –The Law – “ICOC shall establish standards that require that all grants and loan awards be subject to intellectual property agreements that balance the opportunity of the State of California to benefit from the patents, royalties, and licenses that result from basic research, therapy development, and clinical trials with the need to assure that essential medical research is not unreasonably hindered by the intellectual property agreements. “ –Open Issues How much goes to state? Form of payment? Will CIRM seek joint ownership of inventions? Can outsiders share in process?

16 Key Issues Facing CIRM … California – location, location, location –Preference for California Suppliers ICOC shall establish standards to ensure that grantees purchase goods and services from California suppliers to the extent reasonably possible, in a good faith effort to achieve a goal of more than 50 percent of such purchases from California suppliers.

17 Key Issues Facing CIRM … Standards to be set on variety of subjects –Informed Consent –Patient Privacy –Protection of Human Subjects –No “compensation” to donors; can reimburse expenses –Paying for cell lines to cover a number of expenses to obtain, but (implicitly) not to pay a “profit” –Time Limits for Obtaining Cells -- on time during which cells may be extracted from blastocysts initially -- 8 to 12 days after cell division begins, not counting any time during which the blastocysts and/or cells have been stored frozen.

18 Key Issues Facing CIRM … Conflicts of Interest – Sen. Deborah Ortiz and Sen. George Runner – last week proposed constitutional amendment would require CIRM, its board and any outside members of the board's working groups to adhere to strict conflict-of-interest guidelines based on those used in the state government and the federal National Institutes of Health. Open meetings -- It also would force the board, its working groups and the institute to hold all meetings in accordance with California's open-meeting laws.

19 Key Issues Facing CIRM … Was Proposition 71 Legal? – two lawsuits –Allegedly violates requirement that an initiative cover just a single subject –Allegedly can not exempt certain members from government conflicts of interest laws

20 How to Contact CIRM P.O. Box Emeryville, CA Phone: (510) Fax: (510) Web Address: Street address: 5858 Horton Street, Emeryville, CA 94608

21 Questions? Call, , fax or write: Michael A. Swit, Esq. Vice President, Life Sciences THE WEINBERG GROUP INC. 336 North Coast Hwy. 101 Suite C Encinitas, CA Phone Fax Cell D.C. Office

22 About the speaker … Michael A. Swit, Esq., who is Vice President, Life Sciences at THE WEINBERG GROUP INC., has extensive experience in all aspects of FDA regulation with a particular emphasis on drugs and medical device regulation. In addition to his private legal and consulting experience, Mr. Swit also served for three and a half years as vice president and general counsel of Pharmaceutical Resources, Inc. (PRI) a prominent generic drug company and, thus, brings an industry and commercial perspective to his representation of FDA-regulated companies. While at PRI from 1990 to late 1993, Mr. Swit spearheaded the company’s defense of multiple grand jury investigations, other federal and state proceedings, and securities litigation stemming from the acts of prior management. Mr. Swit then served from 1994 to 1998 as CEO of Washington Business Information, Inc. (WBII) a premier publisher of FDA regulatory newsletters and other specialty information products for the FDA publishing company. Before joining THE WEINBERG GROUP, he served in the FDA Regulatory Law Practices at both Heller Ehrman and McKenna & Cuneo, first in that firm’s D.C. office and then in its San Diego office. He first practiced FDA regulatory law with the D.C. office of Burditt & Radzius from 1984 to Mr. Swit has taught and written on a wide variety of subjects relating to FDA law including, since 1989, co-directing a three-day intensive course on the generic drug approval process, serving on the Editorial Board of the Food & Drug Law Journal, and editing a guide to the generic drug approval process, Getting Your Generic Drug Approved, published by WBII. Mr. Swit holds an A.B., magna cum laude, with high honors in history, in 1979, from Bowdoin College, and earned his law degree from Emory University in He is a member of the California, Virginia and District of Columbia bars.

23 For more than twenty years, leading companies have depended on THE WEINBERG GROUP when their products are at risk. Our technical, scientific and regulatory experts deliver the crucial results that get products to market and keep them there.