Moderna finally unveils pipeline at JPM, with 5 candidates in testing

The biotech now has five of its candidates underway in testing with $1.3 billion in cash.

Moderna, often accused of being secretive after failing to reveal, publicly at least, its pipeline of meds, finally did so on Monday with its candidates targeting a host of infectious diseases, I-O and CV diseases.

In one of the more anticipated sessions of this week’s J.P. Morgan Healthcare Conference in San Francisco, the private company, believed to be worth up to $5 billion, says it has 12 mRNA development candidates that include vaccines and therapeutics across the three therapeutic areas.

Tests are now underway for five of these across the globe, including Moderna’s Zika mRNA vaccine, mRNA-1325, which started phase 1 testing a month ago, and two others against influenza targets, which also started last year.

Moderna has partnered with Merck on a viral vaccine mRNA MRK-1777 for an undisclosed target, starting a phase 1 in Australia back in November.

It also has a development deal with Merck for a V1GL personalized cancer vax, mRNA 4157, but this is still some way off entering human testing.

Moderna says with this candidate, it will identify neoantigens present in each patient’s specific tumor and will create a “personalized vaccine encoding for approximately 20 unique neoantigens.”

Moderna says its mRNA-based vax “has the potential to be synergistic with checkpoint inhibitor therapies, including its partner Merck’s anti-PD-1 therapy, Keytruda (pembrolizumab),” and follows along similar lines as other startups, such as Neon Therapeutics and its cancer vax NEO-PV-01, which is also being tested alongside BMS’ PD-1 Opdivo (nivolumab).

Moderna says it’s hoping to use small-batch manufacturing technique and a “digital infrastructure” to “manufacture and supply its personalized cancer vaccines tailored to individual patients within weeks.”

The company has teamed up with BARDA and DARPA for both the ‘1325 Zika vaccine and for mRNA 1388, a drug that is targeting Chikungunya, a viral disease transmitted to humans by infected mosquitoes, which is expected to enter the clinic soon given that an IND application has already been filed with the FDA.

Its most recent trial, started just this month, is for the AstraZeneca-partnered mRNA AZD-8601, a VEGF-A drug that is designed to use mRNA to kick-start a strong, local and transient surge of VEGF-A expression.

The plan is that this will lead to the creation of more blood vessels and improved blood supply and is slated for tests in heart failure or after a heart attack, as well as for diabetic wound healing and other ischemic vascular diseases.

Its initial phase 1 safety study is currently enrolling patients in Europe and is focused on male patients with Type 2 diabetes.

Moderna’s delivery tech has also been something of a secret, but some light has been shone here too.

In a statement, the company said that both the viral vaccines and personalized cancer vaccines are delivered via intramuscular injection.

“The localized therapeutics modality is being applied to advance mRNA-based immuno-oncology therapeutics, delivered via intratumoral injection, as well as mRNA-based therapeutics for cardiovascular disease and other ischemic vascular diseases,” it said.

Other ways Moderna is pursuing in discovery include IV systemic therapeutics, as well as IV liver therapeutics and inhaled pulmonary therapeutics. It’s using a mixture of both in-licensed delivery technologies as well as “proprietary, next-gen delivery technologies.”

At the end of last year, Moderna had $1.3 billion in cash, up around $500 million from the end of 2015, which “affords Moderna several years of runway to support its continued growth and pipeline acceleration.”

Stéphane Bancel, Moderna’s CEO, who presented at JPM, said: “In 2017, we will remain focused on progressing our current development candidates to and through the clinic; discovering and bringing forth additional mRNA medicines as new development candidates; and continuing to invest heavily in our mRNA platform as well as the build-out of our GMP clinical manufacturing facility in Norwood, Mass. We also look forward to begin publishing data on our clinical programs as well as key insights related to our platform.”

“And we will continue to invest in building our team and working diligently to ensure that our employees continue to feel inspired and empowered every day to innovate and drive impact for patients.”