On May 05, 2016 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, today reported financial results for the first quarter ended March 31, 2016 (Press release, NanoString Technologies, MAY 5, 2016, View Source [SID:1234512029]).

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First Quarter Financial Highlights

Total revenue of $14.7 million, 27% year-over-year growth
Total product and service revenue of $12.1 million, 12% year-over-year growth
Consumables revenue of $8.0 million, including $0.8 million of Prosigna IVD kits, 35% year-over-year growth
Instrument revenue of $3.4 million, 22% year-over-year decline
Collaboration revenue of $2.6 million
"We continue to strengthen our leadership in precision oncology on all fronts, while our new biopharma partnerships helped deliver positive operating cash flow during the first quarter," said president and chief executive officer, Brad Gray. "We generated 27% year-on-year revenue growth despite lower than expected instrument sales, as some orders slipped into the second quarter. We have seen a strong recovery in instrument sales during April, expect instrument revenue growth to normalize over the balance of the year, and reiterate our guidance for total revenue of $86 to $90 million for fiscal year 2016."

Recent Business Highlights

Grew installed base to over 370 nCounter Analysis Systems at March 31, 2016
Introduced the nCounter Vantage portfolio of assays that power 3D Biology experiments in cancer research, including immuno-oncology
Presented first proof-of-concept data for digital immunohistochemistry (IHC), a novel technology to simultaneously count multiple protein targets in the spatial context of tumor tissue biopsies
Entered into a collaboration with HalioDx SAS to develop gene expression assays for assessing response to cancer immunotherapies using the nCounter system
Received favorable final Medicare local coverage determination for Prosigna by Noridian Healthcare Solutions and Novitas Solutions, Inc. covering 22 states
Entered into two collaborations with Merck and with Medivation and Astellas to develop and commercialize novel diagnostic tests to predict drug response
First Quarter Financial Results

Revenue for the three months ended March 31, 2016 increased by 27% to $14.7 million, as compared to $11.6 million for the first quarter of 2015. Instrument revenue was $3.4 million, down 22% versus the prior year period, resulting from seasonal trends compounded by slower than expected conversion of sales opportunities into orders, as well as a lower overall average selling price resulting from the mid-2015 launch of the nCounter SPRINT Profiler. Consumables revenue, excluding Prosigna, was $7.2 million for the first quarter of 2016, 31% higher than in the comparable 2015 quarter. Prosigna IVD kit revenue was $0.8 million for the quarter, and collaboration revenue totaled $2.6 million. Gross margin on product and service revenue was 52% for the first quarter of 2016, up from 51% for the first quarter of 2015.

Research and development expense increased by 22% to $7.2 million for the first quarter of 2016 versus $5.9 million for the first quarter of 2015, reflecting increased costs associated with collaboration activities and new products and technologies under development for the life science research market. Selling, general and administrative expense was $14.9 million for the first quarter of 2016 compared to $14.1 million for the prior year period.

Net loss for the three months ended March 31, 2016 declined to $14.6 million, or a loss of $0.74 per diluted share, compared with $14.9 million, or a loss of $0.81 per diluted share, for the first quarter of 2015.

Outlook for 2016

The company’s financial outlook for 2016 is unchanged, and includes:

Total revenue in the range of $86 million to $90 million
Gross margin on product and service revenues in the range of 54% to 55%
Operating expenses in the range of $94 million to $99 million
Operating loss in the range of $40 million to $43 million
Net loss per share in the range of $2.30 to $2.45
Cash from collaborations in 2016 in the range of $40 million to $45 million

On May 05, 2016 Threshold Pharmaceuticals, Inc. (Nasdaq:THLD), a clinical-stage biopharmaceutical company developing novel therapies for cancer, reported financial results for the first quarter ended March 31, 2016 and provided an update on the Company’s corporate and clinical development activities (Press release, Threshold Pharmaceuticals, MAY 5, 2016, View Source [SID:1234512061]).

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"We remain focused on establishing a potential regulatory path forward for evofosfamide as well as a possible strategic partnering initiative, and we are making progress on both of these fronts," said Barry Selick, Ph.D., Chief Executive Officer of Threshold. "I am also pleased with the appointment of Stew Kroll as Chief Operating Officer who most capably leads the strategy, design and execution of our clinical development programs."

Recent Highlights
Evofosfamide – The Company’s lead product candidate is an investigational hypoxia-activated prodrug that is designed to be activated under tumor hypoxic conditions, a hallmark of many cancers. Additional analysis of the MASTRO Phase 3 data combined with previous Phase 2 experience strongly suggests that evofosfamide plus gemcitabine is an active regimen in patients with pancreatic cancer, most notably in the Japanese patients.

Conducted additional analyses of evofosfamide data in pancreatic cancer; the Company intends to discuss potential registration pathways with health regulatory authorities; and
Continued ongoing clinical development collaborations investigating evofosfamide in patients with pancreatic neuroendocrine tumors (pNET), recurrent glioblastoma (GBM) and hepatocellular carcinoma (HCC).
Tarloxotinib – Beyond the Company’s evofosfamide program, the clinical development team is focused on tarloxotinib, a hypoxia-activated epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI), which is designed to selectively release an irreversible EGFR-TKI in hypoxic tumors.

Continued to enroll patients in two proof-of-concept Phase 2 clinical trials in patients with advanced non-small cell lung cancer (NSCLC) as well as metastatic head and neck squamous cell carcinoma; the Company plans to share preliminary results from both trials in mid-2016.
First Quarter 2016 Financial Results

Cash, cash equivalents and marketable securities totaled $38.0 million at March 31, 2016 compared to $48.7 million at December 31, 2015; the net decrease was a result of operating cash requirements for the quarter ended March 31, 2016, including the payment of $2.3 million of accrued severance benefits related to the previously announced workforce reduction in December of 2015. With the previously announced decision to cease joint development of evofosfamide under the Company’s former collaboration with Merck KGaA and the workforce reduction, the Company expects its quarterly operating cash requirements to decrease for the remainder of fiscal year 2016 compared to the first quarter ended March 31, 2016.

No revenue was recognized in the first quarter ended March 31, 2016 compared to $3.7 million for the same period of 2015. Revenue for the quarter ended March 31, 2015 related to the amortization of the aggregate of $110 million in upfront and milestone payments received from the Company’s former collaboration with Merck KGaA, Darmstadt, Germany. The revenue from the upfront payment and milestone payments received under the agreement were previously being amortized over the relevant performance period, rather than being immediately recognized when the upfront payment and milestones were earned or received. As a result of Merck KGaA, Darmstadt, Germany’s and the Company’s decision to cease further joint development of evofosfamide in December 2015, the Company immediately recognized all of the remaining deferred revenue into revenue during the quarter ending December 31, 2015. Also as a result of the termination of the agreement, the Company is no longer eligible to receive any further milestone payments from Merck KGaA, Darmstadt, Germany.

Research and development expenses were $6.0 million for the first quarter ended March 31, 2016, compared to $10.7 million for the same period in 2015. The decrease in research and development expenses, net of reimbursement for Merck KGaA, Darmstadt, Germany’s 70 percent share of total eligible collaboration expenses for evofosfamide, was due primarily to a $3.1 million decrease in employee related expenses, including a $0.5 million decrease in non-cash stock-based compensation expense and a $1.6 million decrease in clinical development expenses and consulting expenses. The Company expects research and development expenses to continue to decline in 2016 as result of the decision to cease further joint development of evofosfamide under the Company’s former collaboration with Merck KGaA and the workforce reduction.

General and administrative expenses were $2.2 million for the first quarter of 2016 compared to $2.6 million for the same period in 2015. The decrease in general and administrative expenses was due primarily to a $0.2 million decrease in consulting expenses and a $0.2 million decrease in employee related expenses.

Non-cash stock-based compensation expense included in total operating expenses was $0.8 million for the first quarter of 2016 compared to $1.4 million for the same period in 2015. The decrease in stock-based compensation expense was due to the amortization of a smaller number of options with lower fair values.

Net loss for the first quarter of 2016 was $7.9 million compared to $11.2 million for the same period in 2015. Included in the net loss for the first quarter of 2016 was an operating loss of $8.3 million and non-cash income of $0.4 million compared to an operating loss of $9.6 million and non-cash expense of $1.5 million for the first quarter of 2015. The non-cash income or expense is related to changes in the fair value of the Company’s outstanding and exercised warrants that was classified as other income (expense).
About Evofosfamide
Evofosfamide (previously known as TH-302) is an investigational hypoxia-activated prodrug of a bis-alkylating agent that is preferentially activated under severe hypoxic tumor conditions, a feature of many solid tumors. Areas of low oxygen levels (hypoxia) in solid tumors are due to insufficient blood vessel supply. Similarly, the bone marrow of patients with hematological malignancies has also been shown, in some cases, to be severely hypoxic. On December 6, 2015, the Company announced the outcomes of two Phase 3 studies (MAESTRO and TH-CR-406/SARC021) of evofosfamide stating that neither study met its primary endpoint.

About Tarloxotinib Bromide
Tarloxotinib bromide (the proposed International Nonproprietary Name, previously known as TH-4000), or "tarloxotinib", is a prodrug designed to selectively release a covalent (irreversible) EGFR tyrosine kinase inhibitor under severe hypoxia, a feature of many solid tumors. Accordingly, tarloxotinib has the potential to effectively shut down aberrant EGFR signaling in a tumor-selective manner, thus potentially avoiding or reducing the systemic side effects associated with currently available EGFR tyrosine kinase inhibitors. Tarloxotinib is currently being evaluated in two Phase 2 proof-of-concept trials: one for the treatment of patients with mutant EGFR-positive, T790M-negative advanced non-small cell lung cancer progressing on an EGFR tyrosine kinase inhibitor, and the other for patients with recurrent or metastatic squamous cell carcinomas of the head and neck or skin. Threshold licensed exclusive worldwide rights to tarloxotinib from the University of Auckland, New Zealand, in September 2014.

On May 05, 2016 Genocea Biosciences, Inc. (NASDAQ:GNCA), a biopharmaceutical company developing T cell-directed vaccines and immunotherapies, reported corporate highlights and financial results for the first quarter ended March 31, 2016 (Press release, Genocea Biosciences, MAY 5, 2016, View Source [SID:1234511999]).

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"Our positive GEN-003 Phase 2 12-month efficacy data suggests that annual treatment with GEN-003 may offer genital herpes patients similar benefits to a full year of daily administration of oral antivirals. These data give us confidence in the potential of GEN-003 to become the cornerstone therapy for patients suffering from genital herpes," said Chip Clark, president and chief executive officer of Genocea. "For our recently initiated Phase 2b efficacy trial, we anticipate reporting virologic efficacy data in the third quarter of 2016, which we anticipate will confirm the efficacy of GEN-003 manufactured using improved processes at an increased scale. We expect clinical efficacy data versus placebo against potential Phase 3 endpoints at six months post dosing around the end of 2016 and we expect to conduct our end-of-Phase 2 meeting with the FDA in the first quarter of 2017. We also continue to advance our academic collaborations in the immuno-oncology field and expect to provide further updates in the coming months on our potential path to the clinic in 2017 for a neoantigen-based cancer vaccine."

Recent Business Highlights and Anticipated Milestones

GEN-003 – Immunotherapy for treatment of genital herpes in Phase 2 development. Greater than $1 billion potential revenue opportunity in U.S. alone

• Reported positive 12-month efficacy data from Phase 2 dose optimization trial
• Full data to be presented at an upcoming scientific meeting

In March 2016, Genocea announced positive 12-month efficacy data from its Phase 2 dose optimization trial evaluating GEN-003 for the treatment of genital herpes. GEN-003 demonstrated statistically significant reductions compared to baseline in the rate of viral shedding 12 months after dosing as well as sustained efficacy across secondary clinical endpoints, in each case across multiple dose groups. GEN-003 was safe and well tolerated by patients, with no serious adverse events reported related to the vaccine in the trial.

Multiple anticipated 2016 clinical milestones for GEN-003

• Phase 2b virologic efficacy data expected in third quarter of 2016
• Phase 2b 6-month clinical efficacy data expected in late 2016
• End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) expected in Q1 2017

In the third quarter of 2016, Genocea expects to report virologic efficacy data from a Phase 2b trial to confirm the efficacy of GEN-003 manufactured with Phase 3 processes at increased scale. Around the end of 2016, Genocea expects to report 6-month clinical efficacy versus placebo against the potential Phase 3 endpoint of the percentage of patients that are lesion free at 6-months after dosing. Positive data would provide an important step toward the end-of-Phase 2 meeting with the FDA, which is expected to occur in the first quarter of 2017.

This ongoing trial has enrolled approximately 135 subjects across the U.S. who have a history of recurrent genital herpes. Subjects will be randomized to one of three dose groups – placebo, 60µg per protein / 50µg of adjuvant, and 60µg per protein / 75µg of adjuvant – and will be monitored for 12 months.

Genocea also expects to commence a planned Phase 2b antiviral combination study in the second half of 2016. Clinical efficacy data from this trial is expected in the first half of 2017. If GEN-003 is additive to the effect of chronic suppressive oral anti-viral therapy, this would further strengthen GEN-003’s value proposition to patients and physicians.

First Quarter 2016 Financial Results

Cash Position: Cash, cash equivalents and investments as of March 31, 2016 were $95.7 million compared to $106.4 million as of December 31, 2015. Genocea expects that these funds will be sufficient to fund its operating expenses and capital expenditure requirements into the second half of 2017.
Research and Development (R&D) Expenses: R&D expenses for the quarter ended March 31, 2016 decreased $1.2 million, to $7.3 million, from the same period in 2015. The decrease was driven by lower clinical costs due to the completion of the GEN-004 Phase 2a trial, which was ongoing in the first quarter of 2015, and the conduct of a smaller Phase 2 trial for GEN-003 in the first quarter of 2016 compared to the same period in 2015. GEN-003 manufacturing costs also decreased due to the timing of activities in support of clinical trial supply. These lower costs were partially offset by higher personnel and lab-related costs to advance Genocea’s pre-clinical product candidates and develop the ATLAS platform for immuno-oncology.
General and Administrative (G&A) Expenses: G&A expenses for the first quarter of 2016 were $3.9 million, compared to $3.4 million for the same period in 2015. The increase reflects higher personnel costs, consulting and professional fees, and depreciation expense, all of which support Genocea’s expanding R&D operations.
Refund of Research and Development Expense: A gain of $1.6 million for the quarter ended March 31, 2016 resulted from cash received pursuant to contractual obligations under a collaboration agreement with Isconova AB ("Isconova") (since acquired by Novavax, Inc.) to refund R&D expenses paid by Genocea to Isconova between 2009 and 2011 relating to the development of the Matrix-M2TM adjuvant technology.
Net Loss: Net loss was $9.8 million for the quarter ended March 31, 2016, compared to a net loss of $12.1 million for the same period in 2015.

On May 5, 2016 Neurocrine Biosciences, Inc. (NASDAQ:NBIX) reported its financial results for the quarter ended March 31, 2016 (Press release, Neurocrine Biosciences, MAY 5, 2016, View Source;p=RssLanding&cat=news&id=2165670 [SID:1234512030]). For the first quarter of 2016, the Company reported a net loss of $19.3 million, or $0.22 loss per share, compared to a net loss of $1.2 million, or $0.01 loss per share, for the same period in 2015.

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The Company’s balance sheet at March 31, 2016 reflected cash, cash equivalents, investments and receivables of $448.6 million compared to $464.3 million at December 31, 2015.

"2015 was a very successful year for Neurocrine and we have carried this momentum into the first quarter of 2016 beginning with positive top-line data from the second Phase III study of elagolix in endometriosis, the initiation of two Phase III studies of elagolix in uterine fibroids, the start of our Phase II T-Force GREEN study of valbenazine in children and adolescents with Tourette syndrome, as well as the acceptance of seven valbenazine abstracts at the American Academy of Neurology and American Psychiatric Association Annual Meetings" said Kevin Gorman, Ph.D., President and Chief Executive Officer of Neurocrine Biosciences. "Our focus for the coming months is to continue to execute across all aspects of our business; advancing compounds from research into the clinic, executing on clinical trials, filing the valbenazine NDA, and interacting with providers and payers as we prepare for the commercial launch of valbenazine for tardive dyskinesia upon FDA approval."

The $15.0 million of revenue for the first quarter of 2016 represents a milestone payment from AbbVie related to the commencement of Phase III studies of elagolix in uterine fibroids. The $19.8 million of revenue for the first quarter of 2015 represents recognized revenue in the form of license fees from the NBI-98854 (valbenazine) collaboration and license agreement with Mitsubishi Tanabe.

Research and development expenses increased to $23.9 million during the first quarter of 2016 from $16.6 million during the same period in 2015. This increase was primarily due to higher external clinical development expenses and associated internal costs related to the Company’s VMAT2 inhibitor, valbenazine, which is being evaluated in both tardive dyskinesia and Tourette syndrome. Additionally, expenses related to the Company’s preparation of the New Drug Application for valbenazine in tardive dyskinesia accounted for a portion of the increase in expenses quarter over quarter.

General and administrative expenses increased from $5.5 million in the first quarter of 2015 to $12.0 million for the first quarter of 2016, primarily due to pre-commercialization activities for valbenazine. Personnel related costs increased by $3.9 million quarter over quarter primarily due to the expansion of sales and marketing and medical affairs personnel. This increase in personnel related costs includes a $2.4 million increase in share-based compensation expense. Additionally, a significant increase in other pre-commercialization activities contributed to the overall growth in general and administrative expenses.

Pipeline Highlights

Valbenazine Update

During the fourth quarter of 2015, the Company announced positive efficacy results from the Kinect 3 study, a Phase III trial that included moderate to severe tardive dyskinesia in patients with underlying schizophrenia, schizoaffective disorder, bipolar or major depressive disorder who underwent six weeks of placebo controlled assessment. Subsequent to the initial six weeks of treatment, subjects were eligible to continue in the Kinect 3 study for an additional 42 week open-label safety assessment. The open-label safety evaluation is anticipated to complete dosing in mid-2016.

In addition to the ongoing safety assessment of Kinect 3, during the first quarter of 2016 the Company completed enrollment in a separate one-year open-label safety study of valbenazine, Kinect 4, to support the anticipated 2016 filing of a New Drug Application of valbenazine in tardive dyskinesia.

The Company also recently initiated a valbenazine roll-over study for those patients who complete the one year of dosing in either the Kinect 3 or Kinect 4 studies. This roll-over study is designed to permit open-label access to valbenazine for up to an additional 72 weeks of treatment.

As announced previously, Neurocrine has received Breakthrough Therapy Designation from the FDA for valbenazine in the treatment of tardive dyskinesia.

The Company is also exploring valbenazine in Tourette syndrome. The Company recently announced the initiation of two Phase II Tourette syndrome studies evaluating valbenazine in adults and pediatrics, the T-Forward study and T-Force GREEN study, respectively.

The T-Forward study is a randomized, double-blind, placebo-controlled, multi-dose, parallel group, study of up to 90 adults. Subjects will receive once-daily dosing of valbenazine during an eight-week treatment period to assess the safety, tolerability and efficacy of valbenazine in adult Tourette patients. The primary endpoint of this study is a change from baseline of placebo vs. active scores utilizing the Yale Global Tic Severity Scale at the end of Week 8.

The T-Force GREEN study is a randomized, double-blind, placebo-controlled, multi-dose, parallel group study of up to 90 children and adolescents. Subjects will receive once-daily dosing of valbenazine during a six-week treatment period to assess the safety, tolerability and efficacy of valbenazine in pediatric Tourette patients. The primary endpoint of this study is the change from baseline of the Yale Global Tic Severity Scale between placebo and active treatment groups at the end of Week 6.

Data from both of these Tourette studies is expected around year-end 2016.

The Company has submitted and had accepted seven valbenazine abstracts at two major medical conferences during the second quarter. Valbenazine data from all three Kinect clinical trials were presented at podium and plenary sessions at the American Academy of Neurology Annual Meeting in April. In addition, four valbenazine scientific abstracts were submitted and accepted for the American Psychiatric Association Annual Meeting in May.

Elagolix Update

During the first quarter of 2016, AbbVie announced positive top-line results from the second of two Phase III clinical trials, the Solstice Study, a multinational study designed to evaluate the efficacy and safety of elagolix in 815 premenopausal women with endometriosis. The top-line results from this trial were consistent with those of the initial Phase III clinical trial, the Violet Petal Study, where after six months of treatment, both doses of elagolix (150 mg once-daily and 200 mg twice-daily) met the study’s co-primary endpoints of reducing scores of non-menstrual pelvic pain and menstrual pain (or dysmenorrhea) associated with endometriosis at month three, as well as month six, as measured by the Daily Assessment of Endometriosis Pain scale. The observed safety profile of elagolix in the Solstice study was consistent with observations from prior studies. Among the most common adverse events (AEs) were hot flush, headache, and nausea. While most AEs were similar across treatment groups some, such as hot flush and bone mineral density loss, were dose-dependent. AbbVie is targeting a 2017 New Drug Application filing with the FDA for elagolix in endometriosis.

In early 2016, AbbVie announced the initiation of the Phase III uterine fibroids program consisting of two replicate randomized, parallel, double-blind, placebo-controlled clinical trials evaluating elagolix alone or in combination with add-back therapy in women with heavy uterine bleeding associated with uterine fibroids. The studies are expected to enroll approximately 400 subjects each for an initial six-month placebo-controlled dosing period. At the end of the six-months of placebo-controlled evaluation, subjects are eligible to enter an additional six-month safety extension study. The primary efficacy endpoint of the study is an assessment of the change in menstrual blood loss utilizing the alkaline hematin method comparing baseline to month six. Additional secondary efficacy endpoints will be evaluated including assessing the change in fibroid volume and hemoglobin. Bone mineral density will be assessed via DXA scan at baseline, the conclusion of dosing, and six months post-dosing.

Essential Tremor Program (NBI-640756) Update

NBI-640756 for patients with essential tremor was discovered in the Neurocrine laboratories. The Company has completed dosing in a single site, randomized, double-blind, placebo-controlled, sequential dose-escalation, pharmacokinetic study assessing the safety and tolerability of a single dose of NBI-640756 in up to 32 healthy volunteers. The study was conducted in multiple sequential cohorts of eight subjects per cohort. Data from this initial Phase I study is expected in the second quarter of 2016.

On May 5, 2016 Akebia Therapeutics, Inc. (NASDAQ:AKBA), a biopharmaceutical company focused on delivering innovative therapies to patients through the biology of hypoxia-inducible factor (HIF), reported financial results for the first quarter ended March 31, 2016 (Press release, Akebia , MAY 5, 2016, View Source [SID:1234512062]). Akebia also provided an update on its Phase 3 INNO2VATE program for vadadustat in dialysis-dependent chronic kidney disease (DD-CKD), and reported data from an ethnobridging study for vadadustat.

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"We continue to execute our strategy designed to position vadadustat as a best-in-class treatment for renal anemia, define a clear path to registration and establish strong commercial support in key markets," said John P. Butler, President and Chief Executive Officer of Akebia. "With the recent European Patent Office decision, we have preserved our access to an important region and are well-positioned to pursue a European collaboration that would provide funding for the balance of our Phase 3 program."

Mr. Butler continued, "On the clinical front, we have now reached alignment with regulators regarding our global Phase 3 program. We are advancing our ongoing global Phase 3 PRO2TECT program in non-dialysis dependent patients (NDD-CKD), and look forward to initiating the INNO2VATE program in DD-CKD patients in the third quarter of 2016. We are also expanding our experience with HIF-PH inhibitors, and plan to begin a Phase 1 trial with our second clinical candidate, AKB-6899, this year."

INNO2VATE Program

Akebia announced today that it has reached alignment with both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) regarding key elements of the Phase 3 INNO2VATE program and expects to launch the program in the third quarter of 2016. The INNO2VATE program includes two separate studies and will collectively enroll approximately 2,600 DD-CKD patients globally. The correction study will enroll approximately 400 patients not currently being treated with recombinant erythropoiesis stimulating agents (rESAs). The conversion study will enroll approximately 2,200 patients currently receiving rESA who will be converted to either vadadustat or the active control with the goal of maintaining their baseline hemoglobin levels. Both studies will include a 1:1 randomization and an open label, active-control, non-inferiority design. Primary endpoints include an efficacy assessment of the hemoglobin response and an assessment of cardiovascular safety measured by major adverse cardiovascular events.

Ethnobridging Study Results

The company announced today that data from its recent ethnobridging study demonstrated that the pharmacokinetics of vadadustat in Japanese volunteers is similar to that in Caucasians at all doses studied. The double-blind study was designed to assess the pharmacokinetics and pharmacodynamics of vadadustat following the administration of multiple ascending doses (150 mg, 300 mg and 600 mg) once daily for 10 days in Japanese and Caucasian healthy volunteers. At all doses studied, the pharmacokinetics and pharmacodynamics of vadadustat in the Japanese population were similar to that observed in Caucasians.

"As we anticipated, these results demonstrate that ethnicity has no effect on the clearance of vadadustat, an important finding that further supports our global development and commercialization strategy in Japan and other Asian markets," stated Brad Maroni, Chief Medical Officer of Akebia. "Together with our partner in Asia, Mitsubishi Tanabe, we look forward to incorporating these results into our plans for vadadustat in the region."

First Quarter 2016 Corporate Highlights

Preserved access to the European market for vadadustat by prevailing in a patent dispute in which the European Patent Office confirmed that none of FibroGen, Inc.’s patent claims met the requirements for patentability and, as a result, revoked the patent in its entirety; and,
Raised approximately $61.0 million, net, in a public offering of approximately 7.3 million shares of common stock in January 2016.
Financial Results

Akebia reported a net loss of ($25.8) million, or ($0.70) per share, for the first quarter of 2016. Net loss for the first quarter of 2015 was ($10.7) million or ($0.53) per share.

Research and development expenses were $20.2 million for the first quarter of 2016 compared to $6.7 million for the first quarter of 2015. The increase is primarily attributable to costs related to the initiation of the PRO2TECT Phase 3 program. Research and development expenses were further increased by personnel-related costs due to additional headcount.

General and administrative expenses were $5.8 million for the first quarter of 2016 compared to $4.2 million for the first quarter of 2015. The increase is primarily due to the following expense increases: $0.8 million due to increased headcount and compensation related costs and $0.8 million in commercial planning costs as well as legal costs.

The Company’s cash provided by operations during the first quarter of 2016 was $17.4 million, an increase of $25.8 million from $8.3 million used in operations for the same period of 2015. The increase is primarily related to the upfront payment of $40.0 million received in January 2016 from Mitsubishi Tanabe in connection with our collaboration agreement. The Company ended the first quarter of 2016 with cash, cash equivalents and available for sale securities of $217.0 million and expects its cash resources to fund its current operating plan through at least the second quarter of 2017.