Big Sangamo Deal for California Stem Cell Agency with an Asterisk

It was a $20 million deal -- possibly as much as $300 million --for Sangamo BioSciences, and its president told the California stem cell agency,

“We wouldn't be where we are today without you.”

The $3 billion agency has pumped $5.4 million into the publicly traded Richmond, Ca., firm for HIV/AIDS research and another $6.4 million last May for a phase one clinical trial dealing with beta-thalassemia, a genetic blood disease.

Last week Sangamo announced that Biogen Idec of Cambridge, Mass., would buy into its research related to beta-thalassemia as well as sickle cell anemia. The announcement said Biogen Idec will also reimburse Sangamo for “its internal and external research and development program-related costs.” Payments of up to another $300 million could come Sangamo's way if it meets certain goals.

Kevin McCormack, CIRM's senior director of public communications,wrote on the agency's blog that it has been “a very good start” in 2014 for CIRM. He noted that the Sangamo announcement followed similar good news from Capricor, another CIRM grant recipient that has announced funding from Big Pharma.

He quoted Ellen Feigal, senior vice president at the agency, as showing how “important the funding we provide is in helping companies like Sangamo get their research to a point where big pharmaceutical companies stand up and take notice, and invest.” 

McCormack wrote that Edward Lanphier, president of Sangamo, sent an email to CIRM in connection with the announcement that said,

"Thank you for ALL of your support over the past several years. We wouldn't be where we are today without you."

The Sangamo deal, however, has an asterisk. In May 2012, a “special advisor” to the stem cell agency, Saira Ramasastry, was nominated to the seven-member Sangamo board of directors. Following an item in the California StemCell Report, the agency announced that her contract was not being renewed. CIRM said she was not involved in a decision-making role and was not required to file a statement of economic interests with the agency. Ramasastry subsequently was elected to the Sangamo board where she still holds a seat.

Sangamo's stock closed at $19.84 today, up 46 cents. Its 52 week high is $20.33 and its low $6.86.

Our take: The investment in Sangamo is a healthy sign of expanding interest from larger firms in stem cell therapies. However, it is darkened by the "revolving door" situation involving Ramasastry. CIRM does not require public disclosures of the financial interests of its consultants. Nor does it restrict revolving door arrangements with them that permit later employment at CIRM-connected firms. At the same time, the agency is moving aggressively to engage industry ever more closely and to generate results that will resonate with the public. The agency would do well to heed the conflict-of-interest study from the prestigious Institute of Medicine in 2009. Bernie Lo of UCSF was one of the editors of the study. He wrote that medical research and the private sector have sharply divergent priorities and sometimes irreconcilable differences. The stem cell agency should act to ensure that both the appearance and reality of cronyism, insider dealings and conflicts-of-interest do not blemish its scientific track record.

More on CIRM's Restriction of Info on Biotech Applicants

The California stem cell agency says its brief statement this week on restriction of information involving biotech companies was generated in an effort to offer a public explanation of its policy.

In response to a query about what led to the statement, Maria Bonneville, executive director of the CIRM board, said,

"In light of CIRM's efforts to work with companies to develop new therapies, we felt it was important to offer a public explanation regarding CIRM's efforts to balance its obligation to provide information to the public with the need to protect the proprietary information of applicants for therapy development projects."

The California Stem Cell Report will have more on this subject in the next week, including a discussion of how it relates to the state's constitution, which states that the public has as "broadly construed" right to information about state government activities.

California Stem Cell Agency on its Need to Advance Biotech Industry and Restrict Information

The California stem cell agency yesterday posted a 245-word statement justifying its efforts to protect what it calls the "confidential" information of business applicants seeking some of the state's $3 billion in research funds.

The statement appeared on the agenda for next Wednesday's CIRM board meeting under a section dealing with material to be presented by President Alan Trounson. The agency posted no explanation about what led to creation of the document or why it was needed. We have queried CIRM concerning the circumstances surrounding the statement.

In the document, CIRM said its aggressive push into therapy development and closer ties to the biotech industry presents "special challenges." The agency said it must "protect the companies’ proprietary information and their ability to obtain follow-on financing." CIRM also justified its position based on CIRM's need to "advance the biotech industry in California to world leadership."

Earlier this year, CIRM embarked on its first-ever involvement in a clinical trial – Geron's historic trial of an hESC therapy. The $25 million loan was handled in a unique and unusual manner that deviated sharply from other grant rounds. CIRM failed to provide a scientific score on Geron's application, which has been standard practice on more than 1,000 other applications, including other businesses. CIRM failed to provide the usual summary of grant reviewer comments that were also provided on all previous business and academic applicants. The three other applicants in the $50 million round all withdrew prior to presentation to the CIRM board – another first in CIRM's grant program. And no public explanation was provided at the time for the departures from long-established procedures used for more than 400 other approved grants and loans.

The California Stem Cell Report subsequently reported the score – 66 out of a possible 100 – which is a public record. The CSCR also wrote in August about the unusual procedures and the 16-1 vote for Geron funding.

CIRM director Joan Samuelson, a patient advocate who has pressed hard for development of therapies, cast the lone vote in May against the Geron application. She said the trial was not ready, and CIRM was not ready. According to the transcript of the meeting, Samuelson said,

"There were lots of -- this is based on the peer review and the comments by the scientist members of the Grants Working Group. There were many concerns that many of the scientist members felt should be satisfied before embarking on a clinical trial and they weren't."

In August, James Harrison, outside counsel to the CIRM board, also invoked the need to protect business information in the case of the Geron application. In many ways, the most recent statement appears to be a summary of Harrison's earlier remarks to the California Stem Cell Report.

Here is the full text of yesterday's statement from CIRM.

"CIRM has historically balanced its obligation to provide information to the public with its responsibility to protect the proprietary information of applicants. With applications for basic research, for example, CIRM has provided detailed information regarding the applications and the recommendation of the Grants Working Group. Applications for awards involving therapy development, however, present special challenges. In order to succeed in its mission to provide therapies and cures for the millions of patients who suffer from chronic disease and injury and to 'advance the biotech industry in California to world leadership, as an economic engine for California’s future,' it is critical that CIRM work closely with the biotech and pharmaceutical sectors.

"Engaging industry requires that CIRM assure the companies with which it works of CIRM’s capacity to protect the companies’ proprietary information and their ability to obtain follow-on financing. This is particularly true for companies involved in clinical research. At this stage of commercial product development, many things are proprietary (e.g., FDA communications, data, clinical plans, etc.); therefore, CIRM has a significant challenge and responsibility to protect the confidentiality of the companies’ submissions as any violation could have adverse consequences for the companies, including a material disclosure, particularly for those companies that are publicly traded. Consistent with this responsibility and its over-arching mission, CIRM has taken what we think are reasonable and rational steps to protect confidential information while providing the public with critical information regarding therapy development projects and the Grant Working Group’s recommendation."

$250,000, Six-Month Contract for CIRM's Lewis

The new interim vice president for research and development at the California stem cell agency holds a $250,000, six-month contract that calls for him to push hard to develop clinical applications that CIRM hopes will demonstrate the success of its $3 billion effort.

The hiring -- as a consultant -- of Alan Lewis (at right), onetime head of ViaCyte, Inc. of San Diego (formerly Novocell) and the Juvenile Diabetes Research Foundation,  brings at least a temporary halt to the search to fill the new VP post that was created after Marie Csete resigned suddenly as chief scientific officer. The CIRM Web site no longer lists the VP position as available.

CIRM President Alan Trounson sought unsuccessfully for a nearly a year to fill the job. This spring he appeared to have run afoul of resistance to his plans for compensation for the post, which would normally top out at $332,000 annually if the position were filled by a regular CIRM employee.

Under the terms of Lewis' contract, he is expected to work about 24 hours a week and meet with CIRM officials at least once a week at the agency's San Francisco headquarters. (CIRM provided an unsigned copy of the contract, which is a public document, at the request of the California Stem Cell Report.)

Lewis is the third top executive of CIRM to be working on a part-time, paid basis. The others are CIRM Chairman Robert Klein, who receives $150,000 for half-time work, and co-Vice Chairman Art Torres, who is paid $225,000 for four days a week. By way of contrast, CIRM President Alan Trounson receives $490,008 annually for fulltime work.

Lewis' agreement runs from June 21 through the end of this year at a rate of $2,500 a day, “with pro-ration (sic) for less than four hours.” The contract is capped at $250,000 not including travel expenses.

On June 23, Trounson told the CIRM governing board that Lewis would be serving as a consultant two or three days a week "to help us with the clinical, preclinical programs." Trounson did not mention that Lewis would carry the title of interim vice president for research and development nor did he mention the size of the contract. If it had been above $250,000, it would have required director approval.   

Lewis is expected to travel widely and possibly internationally. Trips from his home in Solana Beach near San Diego in Southern California to CIRM's headquarters will be paid for by CIRM.

Lewis has had a long business career, which raises questions about possible conflicts of interest. In addition to Novocell, he worked for Signal Pharmaceuticals and Celgene in the San Diego area. He left Novocell in 2008 to work for the diabetes group. The CIRM contract said Lewis affirmed that “there exists no actual or potential conflict between the consultant's family, business or financial interest and the services provided under this agreement.”

ViaCyte is the recipient of more than $26 million in awards from CIRM.

Lewis must file a statement of economic interests that is required of most state officials. We have asked CIRM for a copy of that document.

Here is how the contract describes Lewis' responsibilities:

“• Consult Senior Management on biotechnology, pharmaceutical and investment sectors to enable and enhance the development of clinical applications in CIRM’s scientific portfolio.
“• Consult on the preclinical and clinical development phases of CIRM’s programs and projects involving not-for-profit and for-profit teams, including assembling and working closely with CIRM advisory committees to provide oversight of these programs and make go/no go recommendations to the President for continuation of CIRM support.
“• Work in close collaboration with the Executive Director of Scientific Activities.
“• Integrate basic discoveries where possible into the translational and preclinical pipeline and identifies gaps in CIRM’s scientific program for delivery of cell therapies and related products.
“• Develop and implements strategies to aid clinical applications such as in the critical areas of manufacturing, drug and cell product safety and efficacy.
“• Consult Senior Management regarding interface approaches with national and international regulatory organizations”

The contract differs somewhat from the CIRM news release on Lewis. It said that he would “take direction” from the executive director of scientific affairs – not work in collaboration. This is of significance because their duties overlap and could be a source of friction if not carefully managed.

(Photo from San Diego Union-Tribune)

(Editor's note: An earlier version of this item incorrectly said that Lewis' contract was not mentioned by Trounson at the June CIRM board meeting.)