On May 9, 2016 Juno Therapeutics, Inc. (NASDAQ:JUNO), a biopharmaceutical company focused on re-engaging the body’s immune system to revolutionize the treatment of cancer, reported financial results and business highlights for the first quarter 2016 (Press release, Juno, MAY 9, 2016, View Source;p=RssLanding&cat=news&id=2166580 [SID:1234512138]).
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"In the first quarter, we continued to advance our CD19-directed portfolio – enrolling multiple trials, commencing manufacturing of clinical trial material from our Juno-operated facility, and securing Celgene’s opt-in to product candidates in our CD19 program, which will accelerate our pace outside of North America. Also, we recently reported encouraging early data for product candidates against two other targets, CD22 and WT-1, as our pipeline and research continue to progress beyond CD19," said Hans Bishop, Juno’s President and Chief Executive Officer. "Juno’s capabilities are growing, and we look forward to sharing more data with you later this quarter at ASCO (Free ASCO Whitepaper) and throughout 2016."
First Quarter 2016 and Recent Corporate Highlights
Clinical Progress:
CD19 Portfolio – An investigational new drug (IND) amendment cleared the FDA, allowing Juno to begin clinical manufacturing of JCAR015 for the Phase II ROCKET trial out of a Juno-operated manufacturing facility in Bothell, Washington. Juno plans to use this facility to manufacture product for Juno’s first commercial launches. Juno also announced the initiation of enrollment for its trial combining JCAR014 and MedImmune’s anti-PDL-1 immune checkpoint inhibitor, durvalumab.
JCAR018 – This CD22-directed, fully-human chimeric antigen receptor (CAR) T cell product candidate has the potential to treat or prevent CD19-negative relapses. JCAR018 reached two clinical milestones in a Phase I trial conducted at the National Cancer Institute (NCI) in pediatric and young adult relapsed or refractory (r/r) acute lymphoblastic leukemia (ALL) patients, which triggered payments to Opus Bio in the first quarter of 2016 totaling 603,364 shares of Juno common stock. Investigators presented data from the ongoing Phase I study in pediatric and young adult r/r ALL patients at the American Association for Clinical Research (AACR) (Free AACR Whitepaper) 2016, showing all three patients at dose level 2 (1 x 106 cells/kg) achieved a complete remission and complete molecular remission as measured by flow cytometry. These patients remain in complete remission with follow-up ranging from 3 to 6 months. Limited cytokine release syndrome (CRS) and no severe neurotoxicity was seen at dose level 2. Dose limiting toxicity was observed at higher doses, so dosing will continue at dose level 2 (1 x 106 cells/kg). CD22-directed CAR T treatment has the potential, when combined with CD19-directed CARs, to meaningfully increase the percentage of ALL patients that experience long-term remissions.
JTCR016 – This WT-1-directed, T cell receptor (TCR) cell product candidate is currently being studied in acute myeloid leukemia (AML), refractory mesothelioma, and non-small cell lung cancer. In the first three solid organ tumor patients treated to date, all with mesothelioma, preliminary data presented at AACR (Free AACR Whitepaper) 2016 showed one patient with an ongoing partial response to the WT-1 TCR and one with stable disease. The clinical activity appeared to correlate with the pharmacokinetics of the engineered T cells, as the patient with the partial response had the best T cell expansion and persistence. JTCR016 was generally well-tolerated in these three refractory mesothelioma patients, with no evidence of severe CRS or severe neurotoxicity.
Corporate News:
Juno announced that Celgene exercised its option to develop and commercialize product candidates from Juno’s CD19 program outside North America and China. With the exercise of this option, Celgene paid Juno a fee of $50.0 million and the companies will now share global development expenses for product candidates in the CD19 program. Celgene has commercial rights outside of North America and China, and will pay Juno a royalty at a percentage in the mid-teens on any future net sales in Celgene’s territories of therapeutic products developed through the CD19 program. Juno retains commercialization rights in North America and China.
Juno acquired AbVitro, a leading next-generation single cell sequencing platform company that will augment Juno’s capabilities to create best-in-class engineered T cells against a broad array of cancer targets, including significantly improving the speed of generating TCR binders, while also enabling comprehensive profiling of functional immune repertoires with cancer tissues. Juno and Celgene have agreed in principle to enter an agreement to license Celgene a subset of the acquired technology and grant Celgene options to certain related potential product rights emanating from the acquired technology.
Juno announced, along with WuXi AppTec, the formation of a new company, JW Biotechnology (Shanghai) Co., Ltd., with a mission to develop novel cell-based immunotherapies for patients with hematologic and solid organ cancers in China. The new company will leverage Juno’s world-class CAR and TCR technologies and WuXi AppTec’s research and development and manufacturing platform and local expertise.
Juno announced the creation of a new, best-in-class clinical trials unit dedicated to immuno-oncology, in collaboration with the University of Washington, the Seattle Cancer Care Alliance, and the Fred Hutchinson Cancer Research Center (FHCRC). The clinical trials unit has been established to accelerate the clinical care of patients and the generation of translational medicine insights with cutting-edge immuno-oncology therapeutic candidates.
Juno reported that Celgene exercised its annual "top-up" right, purchasing 1,137,593 shares of Juno common stock at a price of $41.32 per share for an aggregate cash purchase price of $47.0 million.
First Quarter 2016 Financial Results
Cash Position: Cash, cash equivalents, and marketable securities as of March 31, 2016 were $1.13 billion compared to $1.22 billion as of December 31, 2015. The decrease of $91.0 million is due to cash used in connection with the acquisition of AbVitro and cash used to fund operations, offset by cash proceeds of $47.0 million from Celgene for the purchase of 1,137,593 shares of Juno’s common stock. Celgene’s CD19 opt-in payment of $50.0 million occurred after the end of the first quarter.
Cash Burn: Excluding cash inflows and outflows from business development activities, cash burn in the first quarter of 2016 was $61.0 million including $4.0 million of capital expenditures, compared to $26.4 million in the first quarter of 2015. The increase of $34.6 million was primarily driven by cash outflows in connection with the overall growth of the business.
Revenue: Revenue for the three months ended March 31, 2016 was $9.8 million and included milestone revenue of $5.8 million received from Novartis and revenue recognized in connection with the Celgene collaboration agreement of $3.8 million.
R&D Expenses: Research and development expenses in the first quarter of 2016, inclusive of non-cash expenses and computed in accordance with GAAP, were $73.7 million compared to $57.8 million in the first quarter of 2015. The increase of $15.9 million was due to a $23.2 million non-cash expense associated with milestones achieved under its license agreement with Opus Bio related to Juno’s JCAR018 product candidate, which were paid through the issuance of 603,364 shares of Juno’s common stock, a $5.0 million payment to St. Jude in connection with the milestone achieved by Juno’s sublicensee Novartis, as well as increased costs incurred to execute Juno’s clinical development strategy, manufacture its product candidates, and expand its overall research and development capabilities.
For the three months ended March 31, 2016, Juno recorded a gain of $6.6 million related to the success payment liability and an expense of $38.9 million for the same period in 2015. The gain recorded in the first quarter of 2016 was primarily due to a decline in Juno’s stock price at March 31, 2016 compared to December 31, 2015.
Non-GAAP R&D Expenses: Non-GAAP research and development expenses for the three months ended March 31, 2016 and 2015 were $80.1 million and $17.0 million, respectively. Non-GAAP research and development expenses for the first quarter of 2016 include $9.1 million of stock-based compensation expense, $2.2 million of which was paid in connection with the acquisition of AbVitro, as well as the milestone payments described above. Non-GAAP research and development expenses for the first quarter of 2015 include $1.7 million of non-cash stock-based compensation expense. Non-GAAP research and development expenses for the first quarter of 2016 exclude the following:
A gain of $6.6 million associated with the change in the estimated value and elapsed service period for Juno’s potential success payment liabilities to FHCRC and Memorial Sloan Kettering Cancer Center (MSK).
Non-cash stock-based compensation expense of $1.2 million related to a 2013 restricted stock award to a co-founding director that became a consultant upon his departure from Juno’s board of directors in 2014.
A gain of $1.0 million associated with the change in estimated fair value of the contingent consideration recorded in connection with the Stage and X-Body acquisitions.
Non-GAAP research and development expenses for the first quarter of 2015 exclude the following:
An expense of $38.9 million associated with the change in the estimated value and elapsed service period for Juno’s potential success payment liabilities to FHCRC and MSK.
Non-cash stock-based compensation expense of $1.9 million related to a 2013 restricted stock award to a co-founding director that became a consultant upon his departure from Juno’s board of directors in 2014.
G&A Expenses: General and administrative expenses on a GAAP basis for the first quarter of 2016 were $16.0 million compared to $7.4 million for the first quarter of 2015. The increase of $8.6 million was primarily due to increased personnel expenses, including non-cash stock-based compensation expense, an increase in consulting fees including costs related to commercial readiness, and other expenses related to the growth of the business. General and administrative expenses include $4.9 million and $1.8 million of non-cash stock-based compensation expense for the three months ended March 31, 2016 and 2015, respectively.
GAAP Net Loss: Net loss for the three months ended March 31, 2016 was $71.1 million, or $0.72 per share, compared to $65.0 million, or $0.79 per share, for the same period in 2015.
Non-GAAP Net Loss: Non-GAAP net loss, which incorporates the non-GAAP R&D expense, for the three months ended March 31, 2016 was $77.5 million, or $0.78 per share, compared to $24.2 million, or $0.30 per share, for the same period in 2015.
A reconciliation of GAAP net loss to non-GAAP net loss is presented below under "Non-GAAP Financial Measures."
2016 Financial Guidance
Juno expects 2016 cash burn, excluding cash inflows or outflows from business development activities, to be between $220 million and $250 million.
Operating burn estimated to be between $170 million and $195 million.
Capital expenditures estimated to be between $40 million and $55 million, the vast majority of which are related to one-time infrastructure build-outs.