JP Morgan 2017 – Moderna finally unveils its R&D pipeline

In a move that many view as a precursor to going public, mRNA specialist Moderna revealed more of its previously undisclosed R&D pipeline at the JP Morgan Healthcare conference on Monday. The company confirmed it currently has five products in the clinic, all of which are in Phase I studies.

Four of these are mRNA vaccines (two for influenza, one for Zika virus and one for an undisclosed infectious disease) and the fifth an mRNA therapy which encodes VEGF-F being developed in conjunction with AstraZeneca as a treatment for cardiovascular disease. Injected into the heart directly, mRNA AZD-8601 is designed to proliferate blood vessels and improve blood supply in patients with heart failure or those who have suffered a heart attack. It is the most recent of Moderna's assets to progress into clinical testing, having done so earlier this month. An additional vaccine – for the prevention of Chikungunya virus – is reportedly ready to enter Phase I studies.

CEO Stéphane Bancel suggested that progression of mRNA-1440 and mRNA-1851 – for influenza types H10 and H7, respectively – has been prioritised as they provide a good opportunity for Moderna's mRNA delivery technology to be assessed in a clinical setting. Nevertheless, the predominance of vaccines within Moderna's newly presented clinical pipeline has drawn some scepticism, raising questions about both the commercial return on these products and whether the company is facing difficulties in delivering mRNA into cells without unacceptable safety risks – as others in this field have in the past.

Thus the cardiovascular collaboration with AstraZeneca is likely to draw focus, as will the preclinical assets mRNA-2905 – an injection of mRNA encoding the IL-12 protein for the treatment of cancer (also being developed with AstraZeneca) and mRNA-4157 – a personalised cancer vaccine being developed with Merck & Co. designed to prime the immune system to recognise cancer cells. Moderna also confirmed that it has an mRNA treatment encoding for the OX-40L protein, which will be injected directly into tumours (theoretically reducing exposure to side effects), that is ready to enter Phase I testing. The company hopes that each of these assets could be synergistic with checkpoint inhibitor therapy.

R&D spend is likely to exceed $300 million this year, said Bancel, but financing is not a problem for Moderna. Valued at around $5 billion, it is considered to be the private-sector's most valuable biotech company. The biggest challenge for Bancel is validating the fruits of Moderna's labour.

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