Secretive biotech unicorn Moderna unveils pipeline, financials

Francisco - Moderna Therapeutics, one of the best-funded private biotechnology companies in the US, unveiled its long-secret research and development pipeline on Monday, including experimental vaccines, cancer treatments and a cardiovascular therapy.

The Cambridge, Massachusetts-based start-up has about $1.3bn in cash on hand from investors, pharmaceutical partners and government grants, according to a presentation on Monday at the JP Morgan Chase & Company.

Healthcare Conference in San Francisco. It’s privately valued at about $5bn, according to a person familiar with the matter who asked not to be identified because the matter wasn’t public.

Moderna’s treatments are based around the idea of using genetic instructions to turn a patient’s own cells into miniature drug making factories.

Given by injection or intravenously, the therapies encode fragments of genetic instruction using what’s known as messenger RNA. That code then instructs cells to make proteins to fight a disease or infection.

Part of investors’ interest is the potential at Moderna and companies like it to create a whole new industry of drugs using the techniques.

The company’s unveiling comes at a time of great excitement - and caution - in the sector.

Venture investors have put more than $1.5bn every quarter into biotech start-ups since the end of 2014, according to data compiled by PricewaterhouseCoopers. Yet the industry has faced criticism over how new treatments are priced, and skepticism of breakthrough claims has risen following the discrediting of medical diagnostics company Theranos.

Easier, cheaper

Messenger RNA is relatively quick to create and can be easily mass-produced, and the hope is that mRNA therapies will be faster, cheaper alternatives to traditional biotechnology drugs, which are typically grown in labs or harvested from living organisms.

"It’s almost plug-and-play," said Rick Bright, director of the US government’s Biomedical Advanced Research and Development Authority, also known as BARDA.

The agency, which develops countermeasures to bioterrorism, gave Moderna funding because it sees the platform as potentially being a fast responder in an emergency situation.

“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,” Bright said.

High promise

It sounds simple, but to succeed Moderna will have to demonstrate that it can accurately target specific cells in different organs in the body. Moderna is working on the eye, lungs, joints and even the brain, said chief executive officer Stephane Bancel, though those programs are still in early research stages.
 
The company, which was was created in part by venture capital firm Flagship Pioneering, has financing to cover "three to four years" of operations and won’t seek an initial public offering this year, Bancel said.

Its most advanced assets are for two strains of flu, H10N8 and H7N9, and the treatments are in early-stage human studies, the company said Monday. Both strains have barely affected humans at this point, but they have pandemic potential.

"It’d be nice to have one ready to go," said Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases. Normally, however, pharmaceutical companies don’t prepare vaccines ahead of time because there isn’t a market for them. "I’m not sure what their business plan would be," he said.

Bancel said they picked the two influenzas because they’re good proof-of-concept cases with clear paths to approval by drug regulators.

The company says it’s also working on vaccines for Zika, Chikungunya, human metapneumovirus and cytomegalovirus.

Zika effort

BARDA is backing a Zika vaccine the company is developing and Bright said he’s enthusiastic, but cautious about the potential.

"There is no major safety concern," he said, "but because we have limited experience, we have to tread cautiously with the unknown."

In cancer, Moderna has a previously disclosed collaboration with Merck & Company to develop personalized cancer vaccines.

On Monday, it revealed two additional targets that are meant to help a patient’s immune system attack tumors, which often have developed ways of evading the body’s defences. The two targets are known as OX40L and IL-12.

"The IL-12 can turn a cold tumour hot, and the OX40L decorates the tumour like a Christmas tree," said Bancel, making them vulnerable to attack. None of the cancer treatments have started human studies.

Finally, Moderna is collaborating with London pharma giant AstraZeneca to develop an injection intended to stimulate blood vessel creation and improve blood supply in patients who have suffered heart failure or a heart attack. That program has started its first study in humans.