Juno Therapeutics - 2014 Fierce 15

Juno Therapeutics
A well-funded biotech sets out to turn the immune system into a cancer-killing force

CEO: Hans Bishop
Based: Seattle
Founded: 2013
Clinical focus: Immuno-oncology
Website: JunoTherapeutics.com

The scoop: Juno Therapeutics tends to attract superlatives. Since its launch last year, the startup has raised more than $300 million from venture suspects usual and otherwise, by far the largest short-term haul of any biotech in recent memory. And then there's its technology: The Seattle-headquartered company is at work on a new approach to treating cancer that has notched stunning--albeit early--clinical results, employing a method that was profiled in a Forbes story bearing the title "Is This How We'll Cure Cancer?"

But Juno would prefer to think of itself more simply: as a group of world-class cancer researchers working on a way to even the odds between patients' immune systems and the tumors that afflict them. And all that money is designed to help the company keep its head down as it expands its core technology to cover more and more malignancies.

What makes Juno Fierce: The body's own T cells are powerful fighters of disease, ferreting out pathogens and adapting to better combat them in the future. In the case of cancer, however, the immune system's natural defenses often fail to differentiate between tumor cells and healthy ones, allowing malignant presences to pass and proliferate undeterred.

Juno's platform technology addresses that problem by removing T cells from a patient's blood and equipping them with targeting mechanisms called chimeric antigen receptors (CARs), which seek out and bind to proteins expressed by cancer cells. The resulting CAR-equipped T cells, or CAR-Ts, are reinjected into the patient, at which point they seek out cancers and attack them as they would any commonplace infection.

And, in early results, Juno's three clinical CAR-T candidates are doing just that.

In a Phase I/II study on adults with acute lymphoblastic leukemia (ALL), ongoing at the Fred Hutchinson Cancer Research Center, the company's JCAR015 notched an 88% complete response rate, meaning it left the majority of patients with no traces of their cancers. Behind that, Juno is working on JCAR017 for pediatric ALL at Seattle Children's Hospital and JCAR014 for non-Hodgkin lymphoma at the nearby Fred Hutchinson Cancer Research Center.

And while blood cancers were a natural starting point, Juno believes researchers are only scratching the surface of CAR-Ts' potential, CEO Hans Bishop said. The biotech plans to advance multiple candidates into the clinic over the next 18 months, pairing CAR-T with another, similar receptor technology that allows for intracellular targeting. And the company has already validated targets for some solid organ tumors, keeping the particulars under wraps for now, Bishop said.

"We believe that we're right at the beginning of a brand-new industry," he said.

The early returns on CAR-T monotherapies--including from programs helmed by rivals Novartis ($NVS), Kite Pharma ($KITE) and others--are plenty promising, and Bishop believes their efficacy could be further bolstered by matching them with the new generation of checkpoint inhibitors. The prospect of pairing CAR-Ts with new treatments from the likes of Merck ($MRK) and Bristol-Myers Squibb ($BMY) could create "a number of intriguing opportunities," he said.

But, thanks in part to that $300 million purse, Juno's not getting ahead of itself. The biotech is in the enviable position of working at its own pace, investing at once in process development and R&D without worrying about locking down a partnership or pulling off a fundraise to keep the bioreactors running.

For now, Juno is focused on the unmet needs at hand, Bishop said. Take ALL: Patients who come down with the blood cancer have just a 10% chance of responding to approved therapies.

"It's a dismal prognosis, and we just have to do better," he said.

Consider that 88% complete response rate for JCAR015 and, with the major caveat that it's early days yet, "it's easy to compare those two numbers and, whether you're a physician or a patient, get quite excited," Bishop said.

Investors: Arch Venture Partners, the Alaska Permanent Fund, Jeff Bezos, undisclosed public funds

For more:
Juno stuffs its CAR-T war chest with another $134M fundraise
Juno wraps up a $176M A round as it hits the gas on its CAR-T contender
FDA lifts its hold on Juno's cutting-edge cancer drug after deaths trigger delay

-- Damian Garde (email | Twitter

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Juno Therapeutics - 2014 Fierce 15