Some had hoped that the mRNA specialist Moderna Therapeutics would use its JP Morgan slot to announce plans to go public. In the event, the secretive group did the next best thing by revealing more tidbits about its pipeline, which it says now includes five projects already in the clinic.
There is much enthusiasm for Moderna, but there are major risks, the greatest of which is perhaps the problem of getting mRNA into a cell. At the JP Morgan presentation the group's chief executive, Stéphane Bancel, shed no further light on the delivery problem, and admitted that the approach was “either going to be a huge success or not work”, adding that there was “nothing in between”.
Intracellular mRNA delivery is of course a problem for all groups looking at this approach, including Curevac and Biontech, in addition to Moderna (Vantage Point – The messenger comes calling, July 23, 2015). While doubts will continue, the fact that Moderna now has several clinical assets will be perceived as a positive sign, pending actual readout of trials.
Until now it has only disclosed two clinical-stage assets, and had given few details about them. And at JP Morgan he did not mention any plans to float – but with $1.3bn in the bank, including last year’s huge financing, Moderna does not need cash and might want to wait for more favourable market conditions.
In the meantime, the company has its hands full with a 12-strong product pipeline, including the five clinical assets. Four of those projects are being tested in infectious diseases while the other, partnered with Astrazeneca, is in cardiovascular disease.
The company had already disclosed that mRNA-1440 and mRNA-1851 were in human trials, but had not given details of their indications or targets. Now it has revealed that the assets are being evaluated in potential pandemic influenza strains, H10 and H7 respectively. Topline results should be available this year, and could go some way towards validating Moderna’s platform.
The influenza strategy is unusual for a biotech start-up, as this kind of business would naturally depend on selling to government stockpiles. Meanwhile, the Merck & Co-partnered mRNA MRK-1777 went into a phase I study in an undisclosed infectious disease in November 2016. Rounding out the clinical infectious disease contenders is the Zika vaccine candidate mRNA-1325.
Moderna also has a Chikungunya vaccine, called mRNA-1388, which could start human trials soon, and next to enter the clinic could be the cytomegalovirus vaccine mRNA-1647. There is currently no vaccine available for the virus, the most common cause of newborn disability, because “no one has been able to do the right biology”, according to Mr Bancel.
Cardiovascular and oncology
The final clinical-stage asset, the Astrazeneca-partnered mRNA AZD-8601, encodes VEGF-A, and is designed to create more blood vessels to regenerate heart tissue after a heart attack or in heart failure. The project is injected directly into the heart, so it might avoid any problems that might result from high systemic levels of VEGF-A.
Moderna also has preclinical projects in oncology, and at JP Morgan Mr Bancel highlighted two of these: mRNA-2416, which encodes the Ox40 ligand and is designed to recruit T cells; and mRNA-2905, which encodes the cytokine IL-12 – the subject of another partnership with Astrazeneca.
Both are injected directly into tumours, so again could avoid systemic side effects – and both could be combined with checkpoint inhibitors, Mr Bancel said. In addition, Moderna has a second collaboration with Merck investigating a personalised cancer vaccine, an especially risky approach.
Moderna is not afraid to splash the cash to find out whether this and other projects will work. The chief executive said the group planned to burn through $300m in 2017 – “more than the market cap of most biotech companies”. Its pharma partners and investors will hope the gamble pays off.
To contact the writer of this story email Madeleine Armstrong in San Francisco at email@example.com. For live updates from the JP Morgan healthcare conference in San Francisco on January 9-12 follow @ByMadeleineA on Twitter.