conference date: February 29, 2016 @ 2:00 PM Pacific Time
for quarter ending: December 31, 2015 (Q4, fourth quarter 2015)
Overview: Clinical stage development company continued to make pipeline progress in the quarter. Believes could get first approval of JCAR015 for ALL in 2017.
Basic data (GAAP):
Revenue in the quarter was $4.2 million, up sequentially from $1.6 million. There was no revenue in the year-earlier quarter.
Net loss was $85.2 million, down sequentially from a loss of $23.2 million, and up from a loss of $191.9 million year-earlier.
EPS (diluted) was negative $0.89, down sequentially from negative $0.26, but improved from negative $13.68 year-earlier.
Cash burn in 2016 is expected between $220 and $250 million, including capital expenditures of $40 to $55 million. This assumes Celgene will exercise its CD19 opt-in rights.
Juno Therapeutics is aiming for remissions and even cures for cancers, with some data already available supporting that this is possible, at least for blood cancers.
$3.8 million of revenue in Q4 was from Celgene collaboration. "Celgene is already contributing to our clinical development plan."
JCAR015 for the treatment of adult patients with relapsed/refractory ALL (acute lymphoblastic leukemia) continued a Phase 2 study (rocket) which could serve as Juno’s U.S. registration trial. Planning for an FDA submission for commercialization in 2017.
The JCAR017 Phase 1 study in relapsed/refractory B cell NHL (non-Hodgkin lymphoma) continues to enroll. Also getting a very high remission rate.
JCAR014 Phase 1 trial data for ALL released in December should 100% CR (complete response) and molecular remission. In NHL preliminary Phase 1 data released in December showed 64% of patients with CR and no progression. Phase 1 CLL data showed a 57% complete response rate, and all patients showed some response. A new trial in combination with MedImmune's durvalumab PD-L1 CPM is expected to begin enrollment in 2016.
JCAR018 in a Phase 1 trial for ALL presented interim data at ASH. It targets CD22.
"Juno began trials for solid tumor product candidates against four different targets in 2015 and early 2016 – JCAR024 (ROR-1-directed CAR T), JCAR020 (MUC-16-directed armored CAR T engineered to secrete IL-12), JCAR023 (L1-CAM-directed CAR T), and JTCR016 (WT-1- directed TCR). These trials explore treatment across a variety of solid organ tumor settings with data expected over the next 6-18 months."
Patients that relapse after remission is due to the cancer becoming CD19 negative. That can be about one-quarter of patients. Believes adding JCAR018, which targets CD22, can prevent this problem.
Side effects are still a problem, but believes progress can be made on that issue.
Believes manufacturing capabilities are critical to success. Juno continues to invest in process development and manufacturing. The Bothel facility is now ready for production. Juno is still working on creating a completely automated, 2-day process for CART production.
Other trials are ongoing. See Juno Therapeutics pipeline. Plans to have 9 product candidates against 6 targets by mid 2016. A 10th product candidate should be in the clinic in later 2016.
Cash and equivalents balance ended at $1.22 billion, down sequentially from $1.27 billion.Cash burn for the quarter was $51.4 million, including $4.8 million for capital expenditures. No debt.
Operating expense of $91.6 million consisted of: $75.6 million for R&D and $16.0 million for general and administrative. Operating loss was $87.4 million. Interest income $1.0 million. Income tax benefit $1.2 million.
In Q1 paid made a milestone payment in stock (about 400,000 shares) to Opus Bio.
Juno has adequate cash "to last us into the next decade."
Importance of first to market with a CART product? We don't know when we will be presenting the data. Our policy is to publish at clinical meetings. There are plusses and minuses to being first to market. We believe we will be the first to be approved in adult ALL, but one of our competitors may be first in pediatric ALL. The winning company will be the one that provides the best clinical benefits. As we get more data, so far it seems to get better and better.
Duration and durability in JCAR014 in small number of patients, how much more data do you need for confidence? 40% response and 9 months of durability has been told to us by specialist physicians as significant. We believe we can do better than that. We would like to see at least 10 patients with more than 6 months of follow-up.
Solid tumor products, can you give some details on dose escalation? We are starting at very low doses and no conditioning. For first-in human studies we treat one patient at a time at first to see the response.
Armored CAR, any increased side effects expected? In ovarian cancer the protocol is enrolling now. The second phase would start in the next few months. IL12 has a history of side effects at high levels, so we are being conservative in dose escalation and conditioning. Next generation CARS preclinical data suggests we should also go fairly slowly in dose escalation. The risk of toxicity is higher with the IL12 products, so we are watching for that.
There is still a need to educate physicians about CART. We are also going to be meeting with payers, as they need to understand short term vs. long term costs of this therapy.
Memory T cells role and degree needed? The states of differentiation of the cells in the product is important. We are still learning how to control the percentages of the cell types. If they are all early enough they could convert in vitro. We are testing all this in both preclinical and clinical studies. We know that it will matter, we don't know what the optimum ratio is yet.
Could we see the ALL data at ASCO? We can't predict which data will be available and accepted at ASCO, but we have several products, so some data should come out at ASCO. But ASCO is too early for the most advanced study, JCAR015.
We should see preliminary combo data with PD-L1 this year.
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