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Five Prime Therapeutics Reports Second Quarter 2015 Results and Provides Business Update

On August 6, 2015 Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a clinical-stage biotechnology company focused on discovering and developing novel protein therapeutics for cancer and inflammatory diseases, provided a corporate update and reported financial results for the second quarter ending June 30, 2015 (Press release, Five Prime Therapeutics, AUG 6, 2015, View Source [SID:1234507088]).

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"Five Prime is actively building a comprehensive and complementary portfolio of immuno-oncology candidates and programs that target macrophages, immune check points, T cell agonist pathways and regulatory T cells in the tumor microenvironment," said Lewis T. "Rusty" Williams, M.D., Ph.D., president and chief executive officer of Five Prime. "Regarding our clinical programs, we recently received IND clearance for our Phase 1a/1b clinical trial to evaluate the combination of our CSF1 receptor antibody, FPA008, and Bristol-Myers Squibb’s nivolumab, and plan to initiate dosing this month. In July, we began dosing patients in our Phase 1/2 clinical trial of FPA008 in PVNS, a tumor driven by the CSF1 pathway. On the research side, we added a T cell agonist to our early pipeline with the in-licensing of Inhibrx’s antibodies to GITR, a pathway we identified in our protein library and proprietary in vivo screens as one of the most potent inhibitors of tumor growth. We also licensed to bluebird bio our novel human antibodies to an undisclosed target to develop CAR T cell therapies for hematologic malignancies and solid tumors."

"Looking ahead, we expect data from two of our clinical programs before year end. GSK intends to present initial data from the Phase 1b clinical trial of FP-1039 in squamous non-small cell lung cancer and mesothelioma patients at the World Conference on Lung Cancer in September, and we plan to report preliminary data from the open label portion of our Phase 1 study of FPA008 in rheumatoid arthritis patients by year end."

Business Highlights and Recent Developments

Pipeline:

FPA008: FPA008 targets macrophages and monocytes, which are activated or elevated in multiple disease settings. In cancer, tumor-associated macrophages suppress the immune system’s ability to kill cancer cells. In joint diseases, such as PVNS and RA, synovial macrophages play a central role in the disease process.

Initiated Phase 1/2 Clinical Trial of FPA008 in PVNS. In July, Five Prime initiated patient dosing in its Phase 1/2 clinical trial of FPA008 in pigmented villonodular synovitis (PVNS), an orphan indication. During the Phase 1 dose escalation portion of the trial, Five Prime will assess safety and pharmacodynamics of multiple ascending doses of FPA008 to determine the dose for expansion. During the Phase 2 expansion, the company will evaluate tumor response rate and duration, as well as measures of pain and joint function, in approximately 30 patients. Five Prime plans to complete dose escalation and move into dose expansion by the end of 2015 or early 2016.
Prepared for Initiation of Phase 1a/1b FPA008/Nivolumab Combination Trial in Collaboration with Bristol-Myers Squibb (BMS). In July, Five Prime received IND clearance for the Phase 1a/1b clinical trial to explore the combination of FPA008 and nivolumab, BMS’s PD-1 immune checkpoint antibody, in multiple tumor types. The trial will evaluate the safety, tolerability and preliminary efficacy of the combination in patients with non-small cell lung cancer, melanoma, head and neck cancer, pancreatic cancer, colorectal cancer and malignant glioma. Five Prime plans to initiate patient dosing during August and to present the trial design at the International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) in September. The company expects to complete Phase 1a dose escalation and expand into Phase 1b with the selected dose of FPA008 in late 2015 or early 2016.

Continued Dosing Rheumatoid Arthritis (RA) Patients in Open-Label Portion of Phase 1 Clinical Trial of FPA008. Five Prime continued to dose FPA008 in RA patients with active disease who are on methotrexate in its Phase 1 clinical trial. The company plans to report preliminary open-label data from RA patients by the end of 2015.

Continued Enrollment in the Phase 1 Clinical Trial of FPA144. Five Prime continued to enroll its Phase 1a/1b clinical trial of FPA144, an FGF receptor 2b-selective antibody. By the end of 2015, Five Prime expects to complete Phase 1a dose escalation in patients with solid tumors, including gastric cancer, and to begin the Phase 1b expansion at a selected dose in gastric cancer patients whose tumors have evidence of FGFR2b protein overexpression or FGFR2 gene amplification. The company anticipates preliminary Phase 1a data will be available by the end of 2015 or early 2016.

Continued Enrollment in the Phase 1b Clinical Trial of FP-1039/GSK3052230 in Squamous Non-Small Cell Lung Cancer (NSCLC) and Mesothelioma. GlaxoSmithKline (GSK) continued to enroll patients in the Phase 1b clinical trial of FP-1039, an FGF ligand trap, combined with standard doses of chemotherapy. Patients with newly-diagnosed or recurrent squamous non-small cell lung cancer whose tumors have amplification of the FGF receptor 1 gene are being studied in Arms A and B, respectively. In Arm C, GSK is studying patients with malignant pleural mesothelioma, a tumor in which the FGF2 ligand is overexpressed. GSK intends to present preliminary safety and efficacy data from the trial at the World Conference on Lung Cancer in September.

Research Programs:

Established Strategic Research and License Agreement With Inhibrx for Novel GITR Antibodies. In July, Five Prime established a research collaboration and license agreement for Inhibrx’s novel glucocorticoid-induced tumor necrosis factor receptor (GITR) antibodies. The program is currently at lead selection stage and could potentially reach IND in 2017. Using its comprehensive protein library and proprietary in vivo screening technologies, Five Prime identified the GITR pathway as one of the most potent inhibitors of tumor growth. Additionally, agonist antibodies have demonstrated the ability to induce tumor regressions in preclinical models, particularly when administered with other immuno-oncology therapies. The Inhibrx technology represents a potentially best-in-class approach for engineering a multivalent GITR agonistic antibody. Five Prime paid an upfront license fee of $10 million to Inhibrx in July 2015.

Entered into License Agreement with bluebird bio for Novel Antibodies to Develop CAR T Cell Therapy. In May, Five Prime granted an exclusive license to bluebird bio to research, develop and commercialize chimeric antigen receptor (CAR) T cell therapies using Five Prime’s proprietary human antibodies to an undisclosed target for hematologic malignancies and solid tumors. Five Prime received a $1.5 million upfront payment, and is eligible for development, regulatory, and commercial milestones payments of up to $131 million per licensed product as well as tiered royalties on future product sales.

Continued to Advance Immuno-Oncology Research Programs. Five Prime continues to progress its immuno-oncology product candidates toward preclinical development, with fully human antibody campaigns underway to multiple targets. The company provided background on its immuno-oncology discovery methods and preclinical data on novel immune checkpoint candidates during its Research and Development Day in May. Five Prime plans to present further updates at scientific conferences and remains on track for one new IND per year from its research programs beginning in 2017.

Summary of Financial Results and Guidance:

Cash Position. Cash, cash equivalents and marketable securities totaled $207.4 million on June 30, 2015 compared with $149.1 million on December 31, 2014. The increase was primarily attributable to Five Prime’s January 2015 public offering of common stock, offset by cash used in operations.

Revenue. Collaboration and license revenue for the second quarter of 2015 increased by $1.3 million, or 26%, to $6.3 million from $5.0 million in the second quarter of 2014, primarily due to the $1.5 million upfront license payment from bluebird bio.
R&D Expenses. Research and development expenses for the second quarter of 2015 increased by $1.4 million, or 12%, to $13.3 million from $11.9 million in the second quarter of 2014. This increase was primarily related to advancing the FPA008 development program into additional indications and expanding the company’s internal immuno-oncology research and preclinical activities.
G&A Expenses. General and administrative expenses for the second quarter of 2015 increased by $1.6 million, or 53%, to $4.6 million from $3.0 million in the second quarter of 2014. This increase was primarily due to increases in personnel related expenses, including stock-based compensation and facility costs.

Net Loss. Net loss for the second quarter of 2015 was $11.5 million, or $0.45 per basic and diluted share, compared with a net loss of $9.9 million, or $0.46 per basic and diluted share, for the second quarter of 2014. This increase in net loss was primarily related to advancing the FPA008 development program into additional indications and expanding internal immuno-oncology research and preclinical activities.

Updated 2015 Cash Guidance. Five Prime expects full-year 2015 net cash used in operating activities to be between $65 and $70 million and estimates ending 2015 with between $158 and $163 million in cash, cash equivalents and marketable securities. With the addition of the GITR program to the pipeline in July 2015, Five Prime expects to have cash to fund operations through 2017, without entering into any additional collaboration or license agreements or receiving any future milestone payments. This provides sufficient runway to move Five Prime’s three clinical programs beyond efficacy data readouts and to move one or more new immuno-oncology candidates into clinical trials.

CTI BioPharma Reports Second Quarter 2015 Financial Results

On August 6, 2015 CTI BioPharma Corp. (NASDAQ and MTA: CTIC) reported financial results for the second quarter ended June 30, 2015 (Press release, CTI BioPharma, AUG 6, 2015, View Source;p=RssLanding&cat=news&id=2076830 [SID:1234507086]).

"The significant interest from the oncology community generated by the Phase 3 PERSIST-1 clinical data, presented at the ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) conferences, supports our belief that there remains a significant unmet medical need for patients with myelofibrosis and that pacritinib may play an important role in addressing the current treatment gaps for this disease," said James A. Bianco, M.D., CTI BioPharma’s President and CEO. "Armed with these positive data from the PERSIST-1 trial, our efforts are now directed toward exploring potential regulatory pathways in the U.S., while our partner Baxalta expects to submit a marketing application in Europe before the end of the year. Concurrently, we remain committed to completing the second pacritinib Phase 3 trial, PERSIST-2, and to continuing investigation into the potential for pacritinib in other blood-related cancers outside of myelofibrosis."

Second Quarter 2015 and Recent Highlights

Clinical:

In May, data from the PERSIST-1 Phase 3 clinical trial of pacritinib for the treatment of patients with myelofibrosis showed that, compared to best available therapy (exclusive of a JAK inhibitor), or BAT, pacritinib therapy resulted in a significantly higher proportion of patients with spleen volume reduction and control of disease-related symptoms. Treatment with pacritinib resulted in improvements in severe thrombocytopenia and severe anemia, eliminating the need for blood transfusions in a quarter of patients who were transfusion dependent at the time of enrollment. Gastrointestinal symptoms were the most common adverse events and typically lasted for approximately one week. A limited number of patients discontinued treatment due to side effects. There were no Grade 4 gastrointestinal events reported. These results were presented in a late-breaking oral session at the 51st Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper).

In June, results from PERSIST-1 patient-reported outcome (PRO) and other quality of life measures presented at a late-breaking oral session at the 20th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) showed significant improvements in symptom score with pacritinib therapy compared to BAT across the symptoms reported in the presentation.

In June, data from an investigator-sponsored Phase 2 trial of tosedostat in elderly patients with either primary acute myeloid leukemia (AML), or AML that has evolved from myelodysplastic syndrome (MDS) showed that the combination of tosedostat with low-dose cytarabine/Ara-C (LDAC) resulted in an overall response rate of 54 percent in elderly patients with AML, with 45 percent of patients achieving durable complete responses. These findings were also presented at the EHA (Free EHA Whitepaper) congress.

Corporate:

In June, the following two potential milestone payments from Baxalta Incorporated, or Baxalta, to CTI BioPharma were accelerated in the amount of $32 million: a $12 million development milestone advance payable in connection with the potential regulatory submission to the European Medicines Agency with respect to pacritinib, which Baxalta anticipates submitting as early as late in 2015, and a $20 million development milestone advance payable for the first treatment dosing of the last patient enrolled in PERSIST-2 (the ongoing randomized Phase 3 trial evaluating pacritinib for patients with myelofibrosis whose platelet counts are less than or equal to 100,000 per microliter).

In June, entered into an amendment to the loan agreement with Hercules Technology Growth Capital, Inc. under which we received $6.2 million in additional funding with the potential to borrow an additional $5 million subject to certain conditions.
In July, Bruce J. Seeley was appointed as Executive Vice President and Chief Commercial Officer to lead all aspects of commercial operations.

Second Quarter 2015 Financial Results

Total revenues for the second quarter and the six months ended June 30, 2015 were $1.1 million and $3.8 million, respectively, compared to $1.3 million and $2.8 million for the same periods in 2014. Net product revenues of PIXUVRI for the second quarter 2015 were $0.8 million and $1.7 million, respectively, compared to $1.1 million and $2.4 million for the same periods in 2014.

The non-GAAP operating loss, which excludes non-cash share-based compensation expense, for the second quarter ended June 30, 2015 was $28.3 million, compared to non-GAAP operating loss of $21.3 million for the same period in 2014. The GAAP operating loss for the second quarter ended June 30, 2015 was $31.0 million, compared to a GAAP operating loss of $26.7 million for the same period in 2014. The increase in operating loss is predominantly associated with the Phase 3 development program for pacritinib and the PIX306 post-authorization Phase 3 trial for PIXUVRI. For the six months ended June 30, 2015, the non-GAAP operating loss was $51.4 million, compared to $41.1 million for the same period in 2014. For the six months ended June 30, 2015, the GAAP operating loss was $58.5 million, compared to $54.3 million for the same period in 2014. Non-cash share-based compensation expense for the second quarter and six months ended June 30, 2015 was $2.8 million and $7.1 million, respectively, compared to $5.4 million and $13.2 million for the same periods in 2014. For information on CTI BioPharma’s use of the aforementioned non-GAAP measure and a reconciliation of such measure to GAAP operating loss, see the section below entitled "Non-GAAP Financial Measures."

Net loss for the second quarter ended June 30, 2015 was $32.6 million, or $0.19 per share, compared to a net loss of $27.4 million, or $0.19 per share, for the same period in 2014. Net loss for the six months ended June 30, 2015 was $61.2 million, or $0.35 per share, compared to $56.4 million, or $0.39 per share, for the same period in 2014.

As of June 30, 2015, cash and cash equivalents totaled $54.9 million, compared to $70.9 million as of December 31, 2014.

2015 Financial Outlook

CTI BioPharma reaffirms prior financial guidance that it expects total revenues for 2015 will be approximately $50 million to $55 million, and it expects that non-GAAP operating loss for 2015 will be approximately $75 million to $85 million, which excludes non-cash share-based compensation expense. These financial projections are primarily based on factors previously outlined in the Company’s fourth quarter and full year 2014 financial results press release.

Clovis Oncology Announces Second Quarter 2015 Operating Results

On August 6, 2015 Clovis Oncology, Inc. (NASDAQ:CLVS) reported financial results for its quarter ended June 30, 2015, and provided an update on the Company’s clinical development programs for 2015 (Press release, Clovis Oncology, AUG 6, 2015, View Source;p=RssLanding&cat=news&id=2076802 [SID:1234507085]).

"This is a very exciting time for our company," said Patrick J. Mahaffy, President and CEO of Clovis Oncology. "We have completed our first rociletinib NDA and MAA submissions, we are building out our commercial organizations in both the U.S. and E.U., and we are preparing for a potential U.S. launch as early as the end of this year. We are quickly accelerating toward becoming a global commercial biopharmaceutical organization."

Second Quarter 2015 Financial Results

The Company reported no revenues for the second quarter and first half of 2015, compared to $13.6 million for the first quarter and first half of 2014 which consisted of a milestone payment pursuant to its collaboration and license agreement for lucitanib with Les Laboratoires Servier (Servier).

Research and development expenses totaled $60.4 million for the second quarter of 2015 and $117.1 million for the first half of 2015, compared to $28.4 million and $52.6 million for the comparable periods in 2014. The increase in expenses for both the three- and six-month periods is due to the significantly expanded clinical development activities for rociletinib and rucaparib, increased launch planning activities for rociletinib and increased personnel-related expenses associated with the hiring of additional staff to support the Company’s expanded activities.

General and administrative expenses totaled $7.2 million for the second quarter of 2015 and $14.0 million for the first half of 2015, compared to $5.3 million and $10.6 million for the comparable periods in 2014. The year over year increase is primarily due to higher share-based compensation and personnel expense for employees engaged in general and administrative activities, increased facility costs and higher professional service fees.

Net loss attributable to common shareholders was $71.5 million ($2.10 per share) for the second quarter of 2015 and $134.7 million ($3.96 per share) for the first half of 2015, compared to a net loss of $34.8 million ($1.03 per share) and $65.5 million ($1.93 per share) for the comparable periods of 2014. Share-based compensation expense totaled $8.4 million for the second quarter of 2015 and $17.1 million for the first half of 2015.

Clovis had $377.6 million in cash, cash equivalents and available-for-sale securities and approximately 34.1 million outstanding shares of common stock as of June 30, 2015. In July 2015, the Company raised net proceeds of $298.0 million through an offering of 4.1 million shares of common stock.

2015 Key Milestones and Objectives

Highlights of planned or completed objectives for each product follows:

Rociletinib

Rociletinib is an oral, potent, mutant-selective inhibitor of epidermal growth factor receptor (EGFR) under investigation for the treatment of EGFR-mutated non-small cell lung cancer (NSCLC). Rociletinib targets the activating mutations of EGFR (L858R and Del19), while also inhibiting the dominant acquired resistance mutation, T790M. The T790M mutation develops in approximately 60 percent of patients treated with first- and second-generation EGFR inhibitors.

On July 30, 2015, the Company submitted its New Drug Application (NDA) regulatory filing to the U.S. Food and Drug Administration (FDA) for rociletinib for the treatment of patients with mutant EGFR NSCLC who have been previously treated with an EGFR-targeted therapy and have the EGFR T790M mutation as detected by an FDA approved test. Rociletinib was granted Breakthrough Therapy designation by the U.S. FDA in May 2014. Clovis also submitted its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) through the centralized procedure for rociletinib for the same indication. There is a validation period before both applications are formally accepted, after which the review commences.

The U.S. and E.U. regulatory submissions include data from two single-arm studies, TIGER-X and TIGER-2. During the second quarter, updated findings from the TIGER-X study, evaluating the safety and activity of rociletinib in a very advanced EGFR-mutant NSCLC patient population were presented at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting. Highlights of the data presented included the following:

60% overall response rate (ORR) and 90% disease control rate (DCR) in heavily pretreated centrally confirmed tissue T790M-positive patients at the 500mg BID dose

Median progression free survival (PFS) of 10.3 months observed in patients without a history of CNS metastases; median PFS of 8 months observed in an overall population of 270 heavily pretreated centrally confirmed tissue T790M-positive patients, including 40% of patients with a history of CNS metastases

37% ORR in centrally confirmed tissue T790M-negative patients

57% ORR and 80% DCR in centrally confirmed plasma-genotyped T790M-positive patients – may allow for broader testing for mutations in patients ineligible for tissue biopsy

Well-tolerated; the most frequent adverse reactions or lab abnormalities reported were diarrhea, nausea, fatigue, QTc prolongation and hyperglycemia; the only Grade 3 adverse reaction or lab abnormality reported in greater than 5% of patients was hyperglycemia

Rucaparib

Rucaparib is an oral, potent small molecule inhibitor of PARP1 and PARP2 being developed for the treatment of ovarian cancer, specifically in patients with tumors with BRCA mutations and other DNA repair deficiencies beyond BRCA, commonly referred to as "BRCA-like."

In April 2015, Clovis was granted its second Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA), in this case for rucaparib as monotherapy treatment of advanced ovarian cancer in patients who have received at least two lines of prior platinum-containing therapy, with BRCA-mutated tumors, inclusive of both germline BRCA (gBRCA) and somatic BRCA (sBRCA) mutations.

Also during the second quarter, updated findings from ARIEL2 and Study 10, two ongoing studies evaluating the safety and activity of rucaparib in advanced ovarian cancer patients, were presented at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting. Data from these studies demonstrated encouraging activity and safety in women with advanced, platinum-sensitive ovarian cancer with gBRCA and sBRCA mutations. In addition, these data demonstrated that the application of Clovis’ proprietary BRCA-like tumor DNA signature to Foundation Medicine’s companion diagnostic assay successfully predicts the population of BRCA-like patients that are not gBRCA or sBRCA that respond to rucaparib therapy. Highlights of the data presented included the following (all RECIST response rates):

Data from ARIEL2 in BRCA-mutant patients demonstrated an ORR of 69%, a DCR of 94% and a median PFS of 9.4 months
Complete responses (CRs) observed in 10% of patients
Data from ARIEL2 in patients with the BRCA-like signature demonstrated an ORR of 30%, a DCR of 73% and a median PFS of 7.1 months

Approximately 60% of the 204 patients treated in the initial ARIEL2 study had either BRCA-mutant or BRCA-like tumors
61% ORR and median duration of response greater than 11 months observed in 23 BRCA-mutant patients treated with at least three prior lines of chemotherapy from ARIEL2 and Study 10 combined
CRs observed in 13% of patients in this group

Rucaparib is well-tolerated with a manageable safety profile. The grade 3/4 treatment-related adverse events (AEs) observed in >15% of patients treated with the recommended 600mg BID dose were anemia/decreased hemoglobin (16%) in ARIEL2, and fatigue/asthenia (18%), and anemia (25%) in Study 10

Lucitanib

Lucitanib is an oral, potent inhibitor of the tyrosine kinase activity of fibroblast growth factor receptors 1 through 3 (FGFR1-3), vascular endothelial growth factor receptors 1 through 3 (VEGFR1-3), and platelet-derived growth factor receptors alpha and beta (PDGFRα-β). Clovis, which holds exclusive U.S. and Japanese rights, is collaborating with its development partner Les Laboratoires Servier (Servier) on the global clinical development of lucitanib outside of China, initially targeting solid tumors with FGFR pathway activation, including breast and lung cancers.

A Phase 2 program is underway to explore lucitanib in multiple indications, including a U.S. study in patients with treatment-refractory FGF-aberrant breast cancer and a global study in patients with advanced lung cancer with FGFR1 amplification, both of which are currently enrolling patients. In parallel with these Clovis-sponsored studies, a Servier-sponsored Phase 2 study of lucitanib in patients with advanced breast cancer is underway to identify the population of patients most likely to benefit from lucitanib therapy. The Company anticipates initial data from its breast cancer study by year-end 2015.

ChemoCentryx Reports Second Quarter 2015 Financial Results and Provides Corporate Update

On August 6, 2015 ChemoCentryx, Inc., (Nasdaq:CCXI), a clinical-stage biopharmaceutical company focused on autoimmune diseases, inflammatory disorders and cancer, reported financial results for the second quarter ended June 30, 2015 and provided an update on the Company’s corporate and clinical development activities (Press release, ChemoCentryx, AUG 6, 2015, View Source [SID:1234507084]).

"We continued to execute on all of our planned initiatives during the quarter and remain on track to deliver on several key milestones during 2015. We are particularly excited about the progress in our orphan and rare diseases program with our lead drug candidate, CCX168," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "Both the CLEAR and CLASSIC Phase II trials of CCX168 in patients with ANCA-associated vasculitis reached significant enrollment milestones, and we also initiated an important proof-of-concept trial in patients with atypical Hemolytic Uremic Syndrome. Further, in the immuno-oncology area, we have completed the first part of a two-part clinical trial of CCX872 in patients with advanced pancreatic cancer and we look forward to reporting early results from this trial by year-end."

Recent Pipeline Developments Across Key Therapeutic Areas

Orphan and Rare Diseases: CCX168 is one of the Company’s lead drug candidates. It is an orally-administered inhibitor of the complement C5a receptor (C5aR), and is being developed for several rare disease indications, including ANCA-associated vasculitis (AAV) and atypical Hemolytic Uremic Syndrome (aHUS). These are severe and often fatal autoimmune diseases that are characterized by inflammation that can negatively affect many different areas of the body.

Patient enrollment was completed in the European Phase II CLEAR clinical trial of CCX168 in AAV;
Additionally, patient enrollment in the Phase II CLASSIC trial in North America of CCX168 in AAV reached the halfway mark, per plan;

A Phase II proof-of-concept clinical trial was initiated for the treatment of aHUS in patients with end stage renal disease with CCX168;

Data demonstrating the anti-thrombogenic effect of CCX168 in serum from patients with aHUS were presented at the 52nd European Renal Association – European Dialysis and Transplant Association (ERA-EDTA) Congress in London; and
ChemoCentryx collaborators presented data supporting the biological mechanism of C5a inhibition and the resultant anti-thrombotic effect in aHUS by showing that, in serum, CCX168 blocks aHUS induced up-regulation of Von Willebrand factor (VWF) on human microvascular endothelial cells at the 15th European Meeting on Complement in Human Disease in Uppsala, Sweden.
Chronic Kidney Disease: CCX140, an inhibitor of the chemokine receptor known as CCR2 and one of the Company’s lead drug candidates, is being developed as an orally administered therapy for the treatment of diabetic nephropathy, a form of chronic kidney disease.

Positive clinical results from the Phase II trial in patients with diabetic nephropathy treated with CCX140 were presented at the 52nd ERA-EDTA Congress in London in a late breaking oral presentation.

Immuno-Oncology: CCX872, a potent and selective inhibitor of the chemokine receptor known as CCR2, is being evaluated in patients with non-resectable pancreatic cancer.

Patient enrollment advanced in the clinical trial of non-resectable pancreatic cancer treated with CCX872; Part A of the trial has completed and enrollment in Part B has commenced. The two-part trial is evaluating if the combination of CCX872 and FOLFIRINOX, one of the current standard of care treatments for advanced pancreatic cancer, can promote a more effective anti-tumor response in these patients than standard care treatment alone.

Anticipated Milestones

Orphan and Rare Diseases:

Report top-line data by the end of 2015 from the Phase II CLEAR trial in patients with AAV treated with CCX168, which is being conducted in Europe;
Continue enrollment in the Phase II CLASSIC trial in AAV in North America, and report top-line data in the first half of 2016;
Plan for an End of Phase II meeting in 2016 and, with positive CLEAR and CLASSIC Phase II results, initiate Phase III trial in patients with AAV by the end of 2016; and
Report initial results from the CCX168 Phase II proof-of-concept trial in patients with aHUS by the end of 2015.

Chronic Kidney Disease:

Present additional study results from the CCX140 Phase II trial in diabetic nephropathy at subsequent medical meetings and in peer-reviewed publications during the year;
Continue design of Phase III trials and preparation for Phase III development; and
Prepare for an End of Phase II meeting with the FDA.
Immuno-Oncology:

Continue to advance the pancreatic cancer trial of CCX872 in combination with FOLFIRINOX and report early results by the end of 2015; and
Present preclinical results from the combination therapy of chemokine receptor and check point inhibitors in the fourth quarter of 2015.

Second Quarter 2015 Financial Results

Research and development expenses were $8.6 million for the three months ended June 30, 2015 compared to $9.0 million reported for the same period in 2014. The decrease in research and development expenses from 2014 to 2015 were primarily attributable to lower expenses associated with CCX140, the Company’s CCR2 inhibitor, due to the completion of the Phase II clinical trial in patients with diabetic nephropathy in the fourth quarter of 2014 and CCX507, the Company’s second generation CCR9 inhibitor, due to the completion of Phase I clinical development in the third quarter of 2014. These decreases were partially offset by higher expenses associated with CCX168, the Company’s C5aR inhibitor, due to continuing patient enrollment in the third and final step of the CLEAR Phase II clinical trial in Europe and the CLASSIC Phase II clinical trial in North America for the treatment of AAV. Further, the commencement of enrollment in Phase II proof of concept clinical trials in patients with Immunoglobulin A nephropathy, or IgAN and aHUS and costs associated with initiating a Phase Ib clinical trial with CCX872, the Company’s second generation CCR2 inhibitor, in patients with pancreatic cancer in the second quarter of 2015 contributed to the increase.

General and administrative expenses were $3.6 million for the three months ended June 30, 2015 compared to $3.4 million for the comparable period in 2014. The increase in general and administrative expenses from 2014 to 2015 was primarily due to increases in stock based compensation expense for stock option grants and restricted stock unit awards and professional service expenses.

Net loss was $12.1 million for the second quarter ended June 30, 2015 compared to $12.3 million in the same period in 2014.

Total shares outstanding at June 30, 2015 were approximately 44.1 million shares.

Cash, cash equivalents and investments totaled $94.2 million at June 30, 2015.

bluebird bio Reports Second Quarter 2015 Financial Results and Recent Operational Progress

On August 6, 2015 bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies, reported business highlights and financial results for the second quarter ended June 30, 2015 (Press release, bluebird bio, AUG 6, 2015, View Source;p=RssLanding&cat=news&id=2076874 [SID:1234507082]).

"The first half of this year was a period of exceptional progress for bluebird bio across all fronts, and we made significant strides toward our goal of delivering transformative gene therapy products to change the lives of patients," said Nick Leschly, chief bluebird. "We gained general agreement from U.S. and E.U. regulators on the path forward for LentiGlobin in beta-thalassemia major, and presented early data in severe sickle cell disease that supports the potential for LentiGlobin to make a meaningful difference for these patients. We also spoke for the first time in greater detail about our strategy to build an independent, differentiated immuno-oncology business."

Recent Highlights

SICKLE CELL DISEASE (SCD) AND BETA-THALASSEMIA DATA PRESENTATION AT THE 20TH CONGRESS OF THE EUROPEAN HEMATOLOGY ASSOCIATION – Presented positive data from two patients with beta-thalassemia major and the first patient with severe sickle cell disease ever treated with our LentiGlobin BB305 product candidate. As of May 2015, Subjects 1201 and 1202 with beta-thalassemia major remained transfusion-independent for 16 and 14 months, respectively. Subject 1204 with severe SCD demonstrated increasing HbAT87Q production at six months’ follow-up and was free of transfusions for more than three months. At the six-month visit post-drug product infusion, the proportion of anti-sickling hemoglobin (HbAT87Q + HbF) in the patient with SCD accounted for 45 percent of all hemoglobin production. As of May 2015, the patient with SCD had no hospitalizations for sickle cell complications post-transplant, despite weaning of transfusions. LentiGlobin BB305 was well-tolerated, with no drug product-related adverse events observed as of the May 2015 data cut-off.

LENTIGLOBIN BETA-THALASSEMIA GLOBAL REGULATORY STRATEGY – Announced plan to pursue conditional approval of our LentiGlobin BB305 product candidate for the treatment of beta-thalassemia major in the E.U. through the Adaptive Pathways Pilot Program based on data from the ongoing Northstar and HGB-205 studies and plan to pursue accelerated approval in the U.S. based on our planned HGB-207 and HGB-208 studies. Completed NIH RAC review of HGB-207 and HGB-208 study protocols in adult and adolescent patients with beta-thalassemia major and pediatric patients with beta-thalassemia major, respectively.

IMMUNO-ONCOLOGY STRATEGY – Announced strategy to build a broad T cell-based immuno-oncology portfolio based on our immuno-oncology, gene therapy clinical development and lentiviral vector manufacturing expertise and genome editing capabilities. Revised Celgene collaboration to focus exclusively on anti-BCMA product candidates in multiple myeloma, initiated a strategic collaboration with Kite Pharma focused on second-generation T cell receptor (TCR) therapies in HPV-associated cancers and entered into an exclusive license agreement with Five Prime Therapeutics around chimeric antigen receptor (CAR) T cell therapies against an undisclosed cancer target for hematologic malignancies and solid tumors.

CHIEF SCIENTIFIC OFFICER – Hired Philip Gregory, D. Phil., as Chief Scientific Officer. Formerly Chief Scientific Officer and Senior Vice President, Research at Sangamo BioSciences, Dr. Gregory brings extensive expertise in the fields of genome editing and gene therapy.

STRENGTHENED BALANCE SHEET – Raised $477.2 million in net proceeds in an equity financing in June 2015. Our cash, cash equivalents and marketable securities are sufficient to fund our operations through 2018, based on the company’s current business plan. Proceeds from the equity financing will fund advancement of our immuno-oncology programs, development of a commercial infrastructure to support a potential conditional commercial launch of LentiGlobin in Europe, expansion of manufacturing capabilities to support ongoing and anticipated development and commercial efforts, and initiation of clinical studies of LentiGlobin in adult, adolescent and pediatric subjects with beta-thalassemia major.

Second Quarter 2015 Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2015 were $936.4 million, compared to $492.0 million as of December 31, 2014, an increase of $444.4 million, which was primarily driven by the June 2015 equity financing.
Revenues: Collaboration revenue was $4.9 million for the second quarter of 2015 compared to $6.3 million for the second quarter of 2014. Collaboration revenue is primarily comprised of the amortization of deferred revenue related to our collaboration agreement with Celgene.

R&D Expenses: Research and development expenses were $44.3 million for the second quarter of 2015, compared to $13.9 million for the same period in 2014, an increase of $30.4 million. The increase in research and development expenses was primarily attributable to an $11.0 million increase in stock-based compensation expense, of which $8.5 million is non-recurring, a $10.7 million increase in one-time in-license milestones and fees, and an increase in expenses necessary to support the advancement of our clinical and pre-clinical programs.

G&A Expenses: General and administrative expenses were $10.7 million for the second quarter of 2015, compared to $5.7 million for the same period in 2014, an increase of $5.0 million. The increase in general and administrative expenses was primarily attributable to a $3.5 million increase in employee- and contractor-related costs to support our overall growth.
Net Loss: Net loss was $51.8 million for the second quarter of 2015, compared to net loss of $1.5 million for the second quarter of 2014.

Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $936.4 million as of June 30, 2015 will be sufficient to fund its operations through 2018, based on the company’s current business plan.