JPM17: Secretive Moderna Finally Unveils Pipeline, Financials

JPM17: Secretive Moderna Finally Unveils Pipeline, Financial January 10, 2017
By Mark Terry, Breaking News Staff

Notoriously secretive Moderna Therapeutics, based in Cambridge, Mass., gave investors a peak at the company’s research-and-development pipeline yesterday at the JP Morgan Chase & Co. Healthcare Conference in San Francisco.

Moderna is focused on engineering messenger RNA (mRNA), which when injected, turns the patient’s cells into drugmaking factories. A private company, Moderna reportedly has about $1 billion in cash, mostly from payments related to its collaborations with Merck & Co. and Vertex . Both deals were made in mid-year 2016. It also has a Global Health Partnership with the Bill & Melinda Gates Foundation to develop an mRNA-based antibody against HIV.

At the JP Morgan conference, Moderna focused on 12 mRNA development candidates (DCs), which includes vaccines and therapeutics in infectious diseases, immuno-oncology and cardiovascular diseases. Five of the DCs have clinical trials underway in the U.S., Europe and Australia. One includes its Zika mRNA vaccine, mRNA-1325.

“With clinical studies underway for five medicines, 332 healthy subjects dosed thus far, and seven additional development candidates advancing to the clinic, we have rapidly pivoted from a discovery company to a development company with a pipeline of unusual breadth and depth,” said Stephane Bancel, Moderna’s chief executive officer, in a statement. “Moderna is at an inflection point. We’re invested heavily in our mRNA platform, research engine and early development engine to build the world’s leading mRNA company. With this infrastructure in place, we are now able to advance high-quality mRNA medicines with a breadth, speed and scale not common in our industry.”

The company also has funding from the U.S. government’s Biomedical Advanced Research and Development Authority (BARDA). Of the technology, Rick Bright, director of BARDA, told Bloomberg, “It’s almost plug-and-play. All you need is a sequence from a part of the virus you want to make, drop it into the basic machinery, and you could rapidly have dozens of vaccines.”

The company’s highlighted programs include:

AZD-8601 — Partnered with AstraZeneca , this just started clinical trials last week, and is developed to increase levels of vascular endothelial growth factor-A, for patients with heart failure or post-heart attack.

mRTNA-1440 — A vaccine for influenza A virus subtype H10N8.

mRNA-1851 — A vaccine for influenza A subtype H7N9.

mRNA MRK-1777 — it’s a viral vaccine, but it’s target has not been announced. Moderna is partnered with Merck for this program.

mRNA-1388 — A vaccine for Chikungunya virus. This is funded through the Defense Advanced Research Projects Agency (DARPA), part of the U.S. Department of Defense (DoD).

mRNA-1325 — a vaccine for the Zika virus. Funded through BARDA for up to $125 million.

mRNA-1706 — A proprietary formulation of the Zika virus vaccine, a second version that uses the same active pharmaceutical ingredient (API) as mRNA-1325, but uses a next generation formulation, V1GL.

mRNA-1647 — Vaccine for Cytomegalovirus (CMV), one of the most common causes of newborn disability.

mRNA-1653 — Vaccine for Human Metapneumovirus (HMPV) and Parainfluenza virus (PIV3). These typically infect young children by the age of five, and cause mild respiratory illness, which can become severe in young children, the elderly and people who are immunocompromised.

mRNA-4157 — In partnership with Merck, this is an mRNA-based personalized cancer vaccine.

mRNA-2416 — OX40L Immunotherapy. OX40 Ligand is a co-stimulatory protein that boosts T cells in their effects against cancer cells.

mRNA-2905 — Interleukin 12 (IL-12) Immunotherapy. IL-12 actives the immune system. It is in development in partnership with AstraZeneca.

mRNA AD-8601 — VEGF-A, in conjunction with AstraZeneca, this encodes for vascular endothelial growth factor-A (VEGF-A), which may lead to the creation of more blood vessels to improve blood supply.

 JPM17: Secretive Moderna Finally Unveils Pipeline, Financial
“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.,” Bancel said in a statement. “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 work diligently to ensure that our employees continue to feel inspired and empowered every day to innovate and drive impact for patients.”

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