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Interview with Steve Perrin – CEO of ALS-TDI – an innovative non-profit biotech

I got the opportunity to chat this week with Steve Perrin, CEO of the ALS Therapy Development (TDI) Institute in Cambridge, MA. This was started in 1999 by Jamie Heyward after his brother developed the disease. Jamie gave an inspiring talk at TEDMED last year about how he set that up (and another site that is the poster child for the next wave of patient-centric healthcare,PatientsLikeMe).

A new organizational model - non-profit biotech with an innovative research and funding model

ALS-TDI is a 'non-profit biotech' and this they say 'combines the passion and dedication of a nonprofit organization with the entrepreneurial and scientific spirit of a biotechnology company'. It's a 45 person company with 25 full time scientists and 8 fund raisers. Steve Perrin joined as CEO in 2006.  Their annual expenditures are $10-$11m/year raising money from the government, grant foundations and philanthropists. Over the next few years, they aim to add models of two other diseases areas FTD & tau pathologies that share similar molecular signature to ALS in order to spread the target identification and early stage drug development costs. ALS-TDI don't fund any academic researchers - the money they raise goes to internal scientists or external contract research organizations for focused development work. They have low overhead costs (appx 10%) through being lean and efficient, and having minimal staff (e.g. outsourcing roles for CFO and HR). [Interesting side note: fund raising can be disease specific -other diseases e.g. cystic fibrosis that affect people at a younger age often have more of an emotional impact, and it's not as fatal so the benefactors are around longer.]

Broad spectrum of capabilities, with a focus on drug development

They work along the drug development chain from target discovery to drug development. Unlike academic labs ALS-TDI is focused on drug development (the what) and less interested in the pure science (the why). They test about 30 drugs per year using the standard ALS mouse model (SOD1 gene). They've tested around 180 so far and only found 1 viable compound (this is moving forward). They have an 'industrial scale' testing platform and make extensive use of offshoring critical testing (assay testing and small molecule creation) to increasingly sophisticated, yet economical offshore locations such as China, India and Russia.

Two key research areas around FTD/Tau:

i) Testing viability of the TDP43 mutant in mice (is there unacceptable variability in disease onset) and looking at other factors that may explain differences in onset, such as gender, how many copies of the gene do they have, how much exercise do the mice get?

ii) Understanding of the disease progression at the the molecular level using e.g. tissue analysis.

Fairly conservative around IP

Steve comes from a pharma background that is generally not that interested in intellectual property (IP) unless it is for compounds. ALS-TDI has generated a new model (currently concept only) of a for-profit company they can use to spin out promising drug compounds that aren't developed enough for pharma.  The ultimate goal of such a concept would be to advance molecules through phase I (or even through phase II) before partnering with a large pharma for final clinical efficacy testing and in order to scale up to meet market and commercialization standards and demand.

It's expensive to collaborate with pharma

ALS TDI helps make the business case for drugs using e.g. market opportunity map for disease areas, competition in the market place, current opportunity cost to the healthcare system.

Before getting interested in deals, pharma companies tend to need: pre clinical work, results showing disease amelioration in animal models, knowledge of how the drug works, optimized dosing and safety trials phase 1). It costs appx. $3m to get this info for each compound plus the $6-$7m for a 20 patients test group for FDA IND submission. Steve estimates the cost for phase 2 at about $50m and for phase 3 at about 300. Across ALS research, there approximately three in phase 3, six in phase 2 and twelve in phase 1.

Potential collaboration areas

Makes sense to collaborate with other groups (e.g. Fordcastle-client) if there are similar molecular signatures (e.g. extent, location and timing of protein misfolding).

Advisory Boards

Delicate balance because it can be the case that academic professors on the board end up competing for funding or repurposing the ideas they've been exposed to while on the board. The ALS advisory board now constitutes i) people who are focused on the best interests of the ALS-TDI or ii) from biotech and pharma industry (appx 50%).