Sorting Through the Chaff: The Michael J. Fox Foundation's Tracey Mumford on the Quest to Cure Parkinson's Disease

Source: George S. Mack of The Life Sciences Report (1/9/14)
The business of drug development survives by saving lives, but sometimes nonprofit, private activism is needed to kick-start a new medicine into development. The Michael J. Fox Foundation for Parkinson’s Research is the world’s largest nonprofit funder of Parkinson’s disease research, having awarded more than $400 million in grants during its 13-year existence. Senior Associate Director of Research Partnerships Tracey Mumford is the foundation’s link to companies with good ideas. In this interview with The Life Sciences Report, Mumford discusses exciting companies that have warranted foundation support—companies that investors might find very interesting.
The Life Sciences Report: Tracey, you’re responsible for industry strategy at The Michael J. Fox Foundation for Parkinson’s Research (MJFF). You engage and deal with companies, large and small. Can you give me a very brief overview of what that means?
Tracey Mumford: We at MJFF have always engaged with industry groups because we feel they are a very important component of the drug development process. Over the years we have approached businesses by opening up discussions, whether strictly intellectual or targeted to specific programs. The company could be a startup biotech, a large pharma or something in between. We are open to working with all groups to advance our mission of accelerating the development of therapies and finding a cure for Parkinson’s disease.
TLSR: I’m guessing that you get hundreds of proposals each year from companies and academics. Is that reasonable?
TM: We receive between 900 and 1,000 applications each year, and they are all Parkinson’s disease-specific. We are in a very unique spot given that high volume.
TLSR: How do you sort through that number of ideas? A thousand proposals would work out to about four each workday. How do you manage that?
TM: That’s a very good question. I’ll start by saying that our research team includes more than 30 people, and 10 of those are scientists—nine Ph.D.s and one M.D. Those scientists are coupled with business strategists, myself included, to ensure that we fund the most exciting research scientifically and also apply sound business principles to the projects we select. The goal is to ensure the highest likelihood of success. The marriage of business and science also applies to our overall strategy—how to lay that strategy out in any given year and adjust accordingly.
In terms of specifics, we typically employ a two-step application process. We ask for a short letter of intent or pre-proposal, which is reviewed internally by our team of scientists and business strategists. Depending on the project, we may ask an external expert—perhaps a member of our scientific advisory board—to give his or her thoughts.
TLSR: You narrow the pre-proposals down, and then what?
TM: Once we have gone through all the pre-proposals, we come up with a list and invite those on the list to submit a full application. We then begin a second review and, again, ask external reviewers to give their thoughts and feedback. The MJFF team is very involved in this review. We consider the opinions and advice of the external reviewers vis-à-vis what we know about the field, the projects that we have funded in the past, the makeup of our current portfolio and what we’re focused on in a given year and/or funding program. All final funding decisions rest with The Michael J. Fox Foundation staff.
TLSR: You were an analyst at Deutsche Bank. Were you a sellside analyst?
TM: I wasn’t a sellside analyst; I was on the traditional investment banking side.
TLSR: I’m wondering if you apply the same type of diligence to a proposed product as you would when you were looking at drug development companies as an investment banker.
TM: There is a vein of that, but the process is certainly different. We see ourselves as a group funding within the valley of death, so we are willing to take on more risk than other groups might have the appetite for.
TLSR: Tracey, when you look at a proposed therapy—perhaps a preclinical idea—are you determining whether it has a certain percentage chance of being efficacious, getting approved and getting marketed?
TM: While those factors ultimately come into play, we’re really assessing the scientific rationale and the data generated so far, particularly if we are considering a preclinical project. When we’re funding a therapeutic project, particularly if it’s early on in the approval process, we do need to understand what the commercial landscape looks like.
TLSR: You could give a company a grant of $75,000 ($75K) or $1 million ($1M), and you have to be quite careful about how you allocate your funds. However, drug developers see you as a source of nondilutive financing, and cash is fungible. How do you make certain that progress is being made on your targets? How do you ensure that the funds are applied to your goals?
TM: We are not a group that just writes checks. More often than not, before funding a project, we call the principal investigator and say we’re very interested, but we have these suggestions, and this is how we would like to move forward with the proposal. We ultimately develop a project plan together with that investigator, and we are very clear in laying out the milestones that should be achieved over the course of the grant. We tie our payments to those scientific milestones.
We are not only worried about achieving the scientific steps, we also want to ensure that there is a sense of urgency in our awardees as they execute their projects. We’re very thoughtful about the timelines we set out and what must be done to achieve funding.
TLSR: How do you prioritize your projects? Do you go to the areas of greatest need, or do you prioritize in favor of projects that can be developed expediently?
TM: It is a bit of a mix. At the very highest level, the needs of Parkinson’s disease patients today drive our strategy. We look at the entire continuum of disease, and there are symptoms and targets that we particularly focus on.
TLSR: Please break that down for me.
TM: In the disease-modifying space, we have spent the most time and effort on alpha-synuclein and leucine-rich repeat kinase 2 (LRRK2). In the symptomatic realm, we’ve been very focused on improving motor function and alleviating dyskinesia, which is a very common side effect of the gold-standard symptomatic treatment for Parkinson’s disease, L-dopa (levodopa).
Another priority area for MJFF is the alleviation of cognitive impairment in Parkinson’s disease. We feel that understanding and further verifying biomarkers as research tools and diagnostics for Parkinson’s disease is very important, and so we have prioritized that. Other challenges that we are addressing will accelerate the field widely.
TLSR: Do you have preferred mechanisms of action?
TM: We don’t. We are generally agnostic to the approaches that groups take. We are open to exploring any interesting idea that seems to have legs and potential efficacy. Looking at our portfolio, you’ll see we support projects across all different types of mechanisms.
TLSR: You mentioned biomarkers as a priority goal. Are biomarkers currently in use in the Parkinson’s space?
TM: There are currently no fully validated biomarkers for Parkinson’s disease, but we are working hard on that. We are sponsoring a large study called the Parkinson’s Progression Markers Initiative (PPMI), an observational clinical study in which we use advanced, standardized imaging, biological sampling and clinical and behavioral assessments to see if we can come up with a constellation of progression biomarkers for Parkinson’s disease.
TLSR: It sounds like we’re a long way from having biomarkers that could be used as endpoints in clinical trials. Would that be fair to say?
TM: We hope that, via PPMI, we will have validated biomarkers in a few years’ time. Whether or not we can utilize those biomarkers as endpoints is difficult to say. Perhaps they could be utilized as secondary endpoints.
The goal is, of course, to have these serve as primary endpoints one day. But we need to generate more data around the biomarkers before they can be adequately utilized in that way.
TLSR: Can we talk about some companies? I’d love to hear about what particular companies are working on with MJFF.
TM: I’m happy to talk about more than one. Civitas Therapeutics Inc. (private) is a biotech based in Chelsea, Mass., right outside of Boston. We have been working with Civitas for a few years on its inhaled L-dopa project. The proposed product is CVT-301 (dry powder levodopa for inhalation), which is currently in a phase 2b clinical trial. It comes in a small inhaler and is meant to be a rescue therapy for Parkinson’s patients with motor response fluctuations, which are known as “off” phenomena. If you feel that you are going “off” from your L-dopa, and you are going into a meeting or have something you need to do, you can take a puff from the inhaler, after which your L-dopa plasma levels go back up and you regain motor functionality.
TLSR: How much support have you granted Civitas?
TM: We’ve supported it through phase 1 and into phase 2, to the tune of more than $1.3M. We’ve worked with the company very closely on its approach to the Parkinson’s patient population, and see ourselves as a true partner in how Civitas has designed its studies. Earlier this year, following our investment in its phase 2 study, the company was able to raise $38M in venture capital funding. This is a wonderful example of how the MJFF works with industry to help companies advance their programs and get more data, so that other investors get interested.
TLSR: With that level of investment from venture capital, I would imagine Civitas is close to proof of concept.
TM: This phase 2b trial of CVT-301, with a proposed enrollment of 80 patients, started in April 2013 and is scheduled for completion in February 2014.
TLSR: Another company you might speak to?
TM: Amicus Therapeutics Inc. (FOLD:NASDAQ) is a biotech working in the preclinical realm on a compound that increases the activity of the lysosomal enzyme glucocerebrosidase (GCase), which would lower alpha-synuclein levels within a patient’s system. We have funded Amicus to the tune of about $700K over the years. In September the company announced a partnership with Biogen Idec Inc. (BIIB:NASDAQ) around GCase and the alpha-synuclein pathology associated with Parkinson’s. This was very exciting for us, particularly for a product that is still in the preclinical realm.
TLSR: Biogen Idec has made tremendous inroads into neurodegenerative disease, and has a global network of connections with clinicians. I’m thinking that its interest in Amicus and the GCase/alpha-synuclein pathway must be a very hopeful sign.
TM: Just to dovetail, the Amicus collaboration is the first of two deals that Biogen Idec did in the alpha-synuclein space this year. On Dec. 9 the company announced a deal with Proteostasis Therapeutics Inc. (private), another company we have funded that’s working in the preclinical space on alpha-synuclein disaggregation.
TLSR: Another company?
TM: We are working with Canada-based MedGenesis Therapeutix Inc. (private), which we have supported since 2010 to the tune of about $2M. MedGenesis is currently running a phase 2 study on a surgical approach for glial cell line-derived neurotrophic factor (GDNF) delivery. This exciting project is potentially disease modifying. The trophic factor space has been interesting in Parkinson’s disease over the past few years. The results of MedGenesis’ study are due mid-2014, and we hope it is successful. A lot hinges on the results of the trial, given some failures in the trophic factor space recently—namely Ceregene Inc. (a unit of Sangamo BioSciences Inc. [SGMO:NASDAQ]), which announced in mid-April that its phase 2b trial of the CERE-120 (AAV2-vector expressing neurturin) gene therapy product did not meet the endpoint.
TLSR: Is there another name you might mention?
TM: Cynapsus Therapeutics Inc. (CYNAF:OTCQX; CTH:TSX.V) is new to MJFF. We started funding the company in 2012. Cynapsus is working on a dissolvable apomorphine sublingual film as a rescue therapy for patients, again, having “off” episodes. Apomorphine is available in the European Union via injection, but is not available here in the U.S. Cynapsus’ under-the-tongue administration is certainly preferable to an injection, which would be difficult for patients suffering an off episode since the episode may include muscle stiffness and/or the patient having trouble just starting movement. The Cynapsus product is in a phase 1b clinical trial in healthy volunteers to see if blood levels of apomorphine can be achieved via the film delivery system. We’ve funded the company for about $1M, and it has been able to raise some additional funding on the back of our investment.
TLSR: Tracey, MJFF also serves to connect drug developers, academics and others in the field. Can you tell me about that?
TM: Yes. We are working to enable groups to find larger funders and partners to work with. The foundation does this via two formal mechanisms. One is our Annual Parkinson’s Disease Therapeutics Conference, which was held in October 2013 in New York City. The event actually sold out—we had almost 280 attendees. It was a very healthy mix of industry groups and academic investigators. Many conversations and partnerships came out of that meeting, which was the purpose.
We also host the Partnering Program. At the request of investors and business development folks within the pharmaceutical and larger biotech industry, we decided to launch an e-mail version of the Annual Parkinson’s Disease Therapeutics Conference, with a bit of a different slant. We cull through our portfolio and handpick those projects we feel have the most promising results. We then ask those companies to fill out two pages of nonconfidential information, which we share with the investment and pharma communities. It’s a nice way to keep Parkinson’s at the top of people’s minds, and also to share with potential investors our thoughts on the companies we think they should be paying attention to.
TLSR: I’ve enjoyed learning a bit about MJFF. Thank you for this overview.
TM: Thank you so much, George.
Tracey Mumford joined The Michael J. Fox Foundation in 2011. In her current role, she is responsible for MJFF’s industry strategy and is focused on increasing engagement and developing partnerships with various industry groups, including pharma, biotechs and venture capital firms. Prior to joining MJFF, Mumford was an associate in the Capital Services Group within the Equities Division at Credit Suisse AG, where she was responsible for the relationship management of hedge fund clients in regard to capital raising activities. Prior to joining Credit Suisse, Mumford was an investment bank analyst at Deutsche Bank, in the Global Consumer Group. Mumford graduated from Bucknell University with a bachelor’s degree in economics.
DISCLOSURE:
1) George S. Mack conducted this interview for The Life Sciences Report and provides services to The Life Sciences Report as an independent contractor. He or his family own shares of the following companies mentioned in this interview: None.
2) The following companies mentioned in the interview are sponsors of Streetwise Reports: Cynapsus Therapeutics Inc. Streetwise Reports does not accept stock in exchange for its services.
3) Tracey Mumford: I own, or my family owns, shares of the following companies mentioned in this interview: None. I personally am, or my family is, paid by the following companies mentioned in this interview: None. My company has a financial relationship with the following companies mentioned in this interview: Civitas Therapeutics Inc., Amicus Therapeutics Inc., Biogen Idec Inc., Proteostasis Therapeutics Inc., MedGenesis Therapeutix Inc., Sangamo BioSciences Inc. and Cynapsus Therapeutics Inc. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.
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