This blog is a summary of Chapter 5 of the Advanced Therapies Investment Report 2017 produced by Phacilitate & Biotech and Money. The report aims to equip investors with the basic knowledge required to understand the risks of investing in cell and gene-based technologies through contextualised and empirical experience, valuable insight from a series of leading industry stakeholders, and market research and analysis. You can get the full detailed report here.

Having shown reservation during the earliest development stage of the advanced therapy sector, major pharmaceutical organisations now play a defining role in characterising the commercial ecosystem. Some of the first applications of modern cell and gene engineering technologies was in developing healthy and disease phenotype tissue models, initially developed for basic research but now heavily integrated into medium to high-throughput drug screening pipelines. Today, GSK are responsible for the authorisation of one of the only two market authorised gene therapies, Novartis are widely anticipated to achieve the first CAR-T MA later this year, and Pfizer have bought up a number of gene therapy assets. Sanofi, AstraZeneca, Roche, Bayer, Shire, Johnson & Johnson (Janssen), and Bristol MyersSquibb have all developed links to cell or gene therapy assets. Most deals are occurring in early-mid clinical development but becoming increasingly early-stage as the precedence expands and stakeholders generate an increasingly clear understanding of the risk landscape. Some deals are at unprecedently early stages of development (Roche signed a $500 million plus deal with SQZ Biotech, a proof-of-concept stage MIT spinout), testament to the importance of understanding the science behind a technology. Deals have focused on gene therapies (particularly haemophilia and cardiology), CAR-Ts, and manufacturing infrastructure. Although deals are to date relatively low in number, pharmaceutical companies may be considered for exit opportunities and should be consulted as part of investment due diligence, particularly where there is evidence of exceptional scientific value.

Big Pharma strategy in the advanced therapy sector

The expansive product portfolio and robust financial infrastructure of larger pharmaceutical companies places them well to handle the volatility associated with early stage advanced therapy development as they can support ongoing R&D costs through diversified revenue streams. Pharmaceutical companies are key stakeholders in both directly developing, acquiring, and sponsoring and supporting smaller biotech companies.

'Big Pharma' strategy in the cell and gene therapy space

Large pharmaceutical companies are traditionally averse to high-risk investments and have to date been slow to adopt cell and gene therapies, although this is rapidly changing, and multiple organisations are explicitly shifting the focus of their pipelines away from small molecules and towards advanced biologics. Sanofi is to demolish a $118 million small molecule manufacturing plant it completed in 2011 but never used, stating that “in the meantime, the company’s product portfolio had evolved towards a majority of biologics products rather than chemical products... Thus, the production requirements for chemical clinical batches had greatly decreased”. In February, Sanofi announced the construction of a €270 million ($286.3 million) biologics manufacturing plant with Lonza, at the CDMO’s Swiss site. Further, Roche announced in November 2015 a major strategic shift towards more specialised small molecules and biologics, stopping short of specifically announcing an advanced therapeutics focus but demonstrating discomfort with traditional small molecules, cutting 1,200 jobs.

Case study: Novartis and CTL019

The University of Pennsylvania (UPenn) published their first CAR-T data in August 2011 in the New England Journal of Medicine. The paper described first-in-man data of their novel CAR T-cell product, achieving ongoing remission (at 10-month follow-up) in a 65 year old male with refractory chronic lymphocytic leukaemia (CLL). Further clinical data was generated through a series of later publications, culminating in a 2014 paper describing complete remission in 27 of 30 patients with relapsed or refractory acute lymphoblastic leukaemia (r/r ALL). 67% of patients achieved sustained remission, with an overall survival rate of 78%. This stream of publications generated data unlike anything ever achieved in cancer treatment, let alone in terminal patients.

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