Life Sciences Report with Tracy Mumford, who is responsible for
Industry Strategy at The Michael J Fox Foundation. Very informative interview.

http://wallstreetsectorselector.com/...nsons-disease/

Snapshot Enclosure:

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 (NYSEARCA:IBT) 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.

Does MJFF get anything in return for its partial funding? Equity, promises for maximum price, commitment to bring to the market as soon as possible, publication of all results, etc..

John