The much-hyped biotech--which had by the start of 2016 raised more than $1.2 billion in VC and partnering cash--is focused on mRNA, a process that should allow human cells the ability to send out new proteins to reverse all kinds of diseases, including cancer and diabetes.
It’s all very early stage, with only two drugs recently entering into the clinic: mRNA 1440 started a small, predominately safety-focused Phase I in Europe last year with a second Phase I study, for mRNA 1851, now getting underway in the U.S.
Both are infectious disease vaccine candidates for undisclosed targets. Cambridge, MA-based Moderna says that more than 200 healthy volunteers have been dosed to date across the two ongoing studies.
But despite much of its work being preclinical with only limited lab time on those targets in the clinic, Moderna has turned the heads and emptied the wallets of major players, including AstraZeneca, which signed up to a research pact with the biotech back in 2013.
The promise of AZD8601 is that it may enable the delivery of a genetic manual to spur the production of vascular endothelial growth factor A (VEGF-A).
The AZD8601 program is built upon a decade of research on heart stem cells in cardiovascular development conducted by Dr. Kenneth Chien, a professor of cellular and molecular biology and medicine, and his team at the Integrated Cardio-Metabolic Centre at Karolinska Institutet in Stockholm, Sweden.
Dr. Chien, who is also involved in the collab, believes that VEGF-A can act as a cell fate switch for cardiac progenitors, with AZD8601 set to target heart failure, diabetic wound healing and other ischemic vascular diseases.
In a statement, Moderna said it has filed a clinical trial application with the Paul Ehrlich Institute and the German Federal Ministry of Health to start a Phase I clinical trial of their candidate. More details, including timelines, study design and any payment structure, were not forthcoming.
But, as ever with Moderna, there was much excitement, if not too much detail. Marcus Schindler, VP of innovative medicines and early development at AstraZeneca, said in a statement: “Developing regenerative therapies for the treatment of cardiometabolic disease is a hugely exciting and innovative area.
“We believe that using mRNA therapeutics to initiate a strong, local and transient surge of VEGF-A expression could help us overcome challenges associated with previous approaches to regulate this protein in tissues. AZD8601 could one day provide a unique regenerative treatment option for patients with heart failure, diabetic wound healing and other ischemic vascular diseases.”
Stéphane Bancel, CEO of Moderna, added: “This marks a significant milestone for both Moderna and AstraZeneca as our first partnered mRNA program reaches the clinic. It is a validation of our shared vision to harness the potential of mRNA Therapeutics to address serious unmet needs with the goal of improving patients’ lives.”
AstraZeneca has clearly seen something in Moderna as back in January, the two also announced a new collab--this time seeing both work on immuno-oncology mRNA therapeutics.
This dovetails with Astra’s own I/O work in the form of durvalumab, a PD-L1 checkpoint inhibitor, which recently gained an FDA breakthrough therapy designation in bladder cancer--although it is much behind its checkpoint inhibitor rivals Merck ($MRK), Bristol-Myers Squibb ($BMY) and Roche ($RHHBY).
Merck and Alexion ($ALXN) are also big fans of Moderna, having too signed up with the biotech for its mRNA tech. With Merck, the biotech is working on MRK 1777, a vaccine, as well as mRNA 1388, a DARPA-backed infectious disease treatment. And with Alexion, the two have teamed up on ALXN 1540, a therapy for the rare Crigler-Najjar syndrome.
In the past two months, Moderna and Merck also penned a new deal that sees them push on with mRNA-based personalized cancer vaccines, specifically based on its marketed PD-1 cancer med Keytruda (pembrolizumab) in multiple cancer types. Clinical study of this personalized cancer vaccine should start next year.
And in July, the biotech signed a 3-year research deal with Vertex ($VRTX) in cystic fibrosis, which will naturally focus on the use of mRNA therapies.
The biotech also released its half-year update this morning, saying that as of June 30 it had around $1 billion in cash--up from the $802 million in cash it announced back in January during its last major update.
The biotech is also expanding its current workforce of roughly 320 people from late 2015 to about 500 by year's end, having already grown to 440 staffers by the end of June.
Outside of personnel, Moderna has also expanded both its manufacturing capacity and its overall footprint in Cambridge, including creating a wholly owned GMP manufacturing facility, as well as increasing the square footage of office, lab and manufacturing space from 115,000 sq. ft. at the beginning of 2016 to 200,000 sq. ft. as of June 2016.