On August 2, 2017 Eisai Co., Ltd. reported financial results for the first quarter ended June 30, 2017 (Report, Eisai, AUG 2, 2017, View Source [SID1234520053]).

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Gross profit for the first quarter of 2017 was 92.5 billions of Yen in comparison to that of 87.1 billions of Yen in the first quarter of 20156. Sales of Oncology products amounted to 31.1 billions of Yen

For Eisai’s detailed sales figures, View Source

On August 2, 2017 bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, reported business highlights and financial results for the second quarter ended June 30, 2017 (Press release, bluebird bio, AUG 2, 2017, View Source [SID1234520007]).

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"In the first half of 2017, we’ve made tremendous progress against our stated goals and continue to build momentum to the end of the year. New data from our bb2121 anti-BCMA CAR T program reinforced our confidence in this program, and we and Celgene are moving full speed ahead to continue to the next stage of development. We presented early data demonstrating the impact of manufacturing improvements for LentiGlobin, with our first patients with TDT in Northstar-2 showing significantly higher drug product VCNs than we saw in previous studies. We also announced interim data from the first 17 patients in the Starbeam study of Lenti-D that showed 88% of those patients met the primary endpoint," said Nick Leschly, chief bluebird. "We look forward to sharing more of our progress later this year at ASH (Free ASH Whitepaper), where we will provide updated data on our TDT and multiple myeloma programs, as well as a first look at the progress made in the HGB-206 study in severe SCD with changes in the study protocol. For the rest of the year, we’re remaining laser-focused on the execution of our clinical development goals across our programs to bring our transformative therapies to patients and on preparing our organization to bring our gene therapy products to many more patients in a commercial setting."

Recent Highlights

COMPLETED ENROLLMENT IN NORTHSTAR-2 – In June, bluebird bio completed the enrollment of the adult and adolescent patient cohort in the Northstar-2 study of LentiGlobin drug product in patients with TDT and non-β0/β0 genotypes.
UPDATED DATA FROM BB2121 ANTI-BCMA CAR T PROGRAM PRESENTED – At ASCO (Free ASCO Whitepaper) in June, bluebird bio presented updated results from the ongoing CRB-401 Phase 1 clinical study of bb2121, an investigational anti-BCMA CAR T cell therapy, in 18 patients with relapsed/refractory multiple myeloma. 100% of the 15 evaluable patients in active dose cohorts (doses above 50 x 106) achieved an objective response; overall response rate (ORR) across all cohorts (n=18) was 89%. 73% of evaluable patients in active dose cohorts achieved a very good partial response (VGPR) or better; 27% complete response (CR) rate across active dose cohorts. All patients tested for minimal residual disease (MRD) status (n=4) were found to be MRD-negative. No disease progression had been observed in active dose cohorts as of the May 4, 2017 data cut-off; range of follow-up was 8 to 54 weeks. No dose-limiting toxicities had been observed. The objective of this Phase 1 dose-escalation study is to evaluate the safety and efficacy of bb2121 and determine a recommended Phase 2 dose. bluebird bio and Celgene are jointly developing bb2121.
EARLY DATA FROM NORTHSTAR-2 PRESENTED – At EHA (Free EHA Whitepaper) in June, bluebird bio presented early data from its Phase 3 Northstar-2 (HGB-207) study of LentiGlobin drug product in patients with transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes. Drug product vector copy number (DP VCN) and percentage of lentiviral vector positive cells (LVV+) for the initial 7 drug product lots manufactured in Northstar-2 were consistently higher than in Northstar (HGB-204), with a median DP VCN of 3.0. Initial results show that the three patients treated to date had achieved in vivo VCN and HbAT87Q production as good as or better than patients achieving transfusion independence in Northstar. The first patient treated in Northstar-2 with 6 months of follow-up achieved normal levels of total hemoglobin (13.3 g/dL) after discontinuing transfusions, producing 9.5 g/dl of HbAT87Q at last follow-up. The safety profile was consistent with autologous transplantation.
NEW DATA FROM HGB-205 PRESENTED – At EHA (Free EHA Whitepaper) in June, bluebird bio presented new data from the HGB-205 study of LentiGlobin drug product in patients with TDT and severe sickle cell disease (SCD). Ongoing transfusion independence up to 3.5 years was observed in patients with TDT; three patients have discontinued iron chelation. The first patient with SCD treated with gene therapy (Patient 1204) continues to show clinically meaningful improvement in symptoms of SCD and stable vector copy number and HbAT87Q in peripheral blood. Two recently treated patients with severe SCD show increasing levels of HbAT87Q and stable in vivo VCN. As with Patient 1204, the first patient with SCD treated in HGB-205, these two patients received a more stringent busulfan conditioning regimen and regular blood transfusions prior to stem cell harvest.
TOPLINE INTERIM LENTI-D DATA ANNOUNCED – In June, bluebird bio announced topline interim clinical data on the initial 17 patients treated in the Starbeam study of Lenti-D drug product in CALD. As of June 13, 15/17 patients (88%) in initial study cohort remain free of major functional disabilities (MFDs) at 24 months, the primary endpoint of the trial. This exceeds bluebird’s pre-defined interim efficacy benchmark for the study of MFD-free survival of 76%, derived from the literature and based on clinical data from an earlier observational study describing that natural history of CALD and outcomes from allogeneic hematopoietic stem cell transplant. The safety profile of Lenti-D was consistent with myeloablative conditioning. No patients treated with Lenti-D had graft versus host disease, and there was no graft rejection or clonal dominance. An expansion cohort is enrolling additional patients to gain European manufacturing experience.
NEW BOARD APPOINTMENTS – In June, bluebird bio appointed John O. Agwunobi, M.D. and Douglas A. Melton, Ph.D. to its Board of Directors.
DUKE COLLABORATION – In May, bluebird bio announced that it has entered into a collaboration with Duke University’s Robert J. Margolis, MD, Center for Health Policy to develop a broadly-supported path for value-based payment reform models for gene therapies and other innovative treatments.
STRENGTHENED BALANCE SHEET – In June, bluebird raised $436.8 million in net proceeds in an equity financing. The company’s cash, cash equivalents and marketable securities are sufficient to fund operations into 2020 based on the company’s current business plan. Proceeds from the equity financing will fund the potential exercise of the option to co-develop and co-promote bb2121; planned clinical studies in oncology and severe genetic diseases; and to further expand the company’s manufacturing platform and capabilities to support ongoing and anticipated product development efforts and in anticipation of a potential commercial launch; and general and administrative expenses.
Second Quarter 2017 Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2017 were $1.2 billion, compared to $884.8 million as of December 31, 2016, an increase of $312.2 million.
Revenues: Total revenue was $16.7 million for the second quarter of 2017 compared to $1.6 million for second quarter of 2016. The increase is primarily attributable to revenue recognized under bluebird bio’s out-licensing agreements with Novartis Pharma AG and GlaxoSmithKline Intellectual Property Development Limited (GSK) and the commencement of revenue recognition for the bb2121 license and manufacturing services under the company’s agreement with Celgene.
R&D Expenses: Research and development expenses were $64.3 million for the second quarter of 2017 compared to $41.8 million for the second quarter of 2016. The increase in research and development expenses was primarily attributable to increased manufacturing expenses, clinical trial expenses, and employee-related costs due to increased headcount to support overall growth.
G&A Expenses: General and administrative expenses were $21.2 million for the second quarter of 2017 compared to $18.4 million for the second quarter of 2016. The increase in general and administrative expenses was primarily attributable to increased employee-related costs due to increased headcount, and increased facility-related expenses to support overall growth.
Net Loss: Net loss was $70.9 million for the second quarter of 2017 compared to $58.8 million for the second quarter of 2016.
Financial Guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $1.2 billion as of June 30, 2017 will be sufficient to fund its current operations into 2020.

On August 2, 2017 BioTime, Inc. (NYSE MKT: BTX), a clinical-stage biotechnology company developing and commercializing products addressing degenerative diseases, reported financial results for the second quarter ended June 30, 2017 (Press release, BioTime, AUG 2, 2017, View Source [SID1234520006]).

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"The second quarter of 2017 was very productive for BioTime, with numerous significant clinical, financial and operational accomplishments. BioTime and its subsidiaries and affiliates now have six products in clinical trials. The data from those trials continue to be positive and encouraging," said Adi Mohanty, Co-Chief Executive Officer. "On the strength of the positive results from our pivotal study of Renevia, we are preparing to file for a CE mark for commercial approval in Europe. Our goal is to commercialize Renevia in its first indication in 2018. For OpRegen, our therapeutic product candidate for the treatment of dry AMD, we were pleased to receive DSMB approval to advance the ongoing Phase I/IIa trial to the third cohort, which will include clinical sites in the U.S."

"We continue to make progress on simplifying our corporate structure to allow us to execute our objectives more efficiently, as well as to make it easier for investors and other external stakeholders to better understand BioTime," continued Mr. Mohanty. "We achieved an important milestone toward accomplishing these goals with the launch of AgeX Therapeutics, a subsidiary formed to consolidate our early-stage research and development programs related to the biology of aging and age-related disease. AgeX recently commenced operations following a $10 million equity financing."

Highlights

Clinical Progress

Renevia (adipose cells + cell delivery matrix)

Renevia successfully met its primary endpoint in a pivotal trial in patients with HIV-associated lipoatrophy (facial fat loss) conducted in Europe. Treated patients retained approximately 100% of transplanted volume at 6 months compared to no incremental hemifacial volume in the untreated patients (p<0.001). All Renevia transplants were shown to be safe and well tolerated and there were no serious adverse events during the trial.
BioTime is on track to file for CE Mark for commercial approval for Renevia in Europe by the end of 2017.
Additional trials in the U.S. are planned that target a broader $7 billion aesthetics market opportunity, which is consistent with the previously stated goal of indication and geographic expansion for Renevia.
OpRegen (retinal pigment epithelial cells)

In April, new positive clinical data on OpRegen were presented at the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO). The data, from the first and second cohorts of the ongoing Phase I/IIa clinical trial in the advanced form of dry-AMD, showed that OpRegen cells engraft and that there was evidence of a biological response.
The Data Safety Monitoring Board (DSMB) monitoring the Phase I/IIa OpRegen trial has authorized BioTime to move forward with enrollment for cohort 3 which will include two US sites with leading ophthalmologists.
An abstract related to the Phase I/IIa OpRegen trial has been accepted for presentation at the American Academy of Ophthalmology (AAO) annual meeting being held in New Orleans, November 11-14, 2017.
BioTime expanded its ophthalmology program with the signing of a revised and expanded licensing agreement with Hadassah Medical Organization of Jerusalem, Israel. The revised and expand license agreement increases BioTime’s field-of-use for RPE cells to all eye disorders, and also adds photoreceptor cells for all eye disorders.
AST-OPC1 (oligodendrocyte progenitor cells)

In June, BioTime’s affiliate, Asterias Bio-Therapeutics (NYSE MKT: AST) announced new 9-month follow-up data from the company’s ongoing SCiStar Phase I/IIa clinical trial. The results showed that previously reported meaningful improvements in arm, hand and finger function in the 10 million cell cohort treated with AST-OPC1 cells have been maintained and in some patients have been further enhanced 9 months following dosing.
The FDA has accepted Asterias’ amendment to the clinical research protocol for the SCiStar trial to include patients with a C-4 spinal cord injury, the second most common form of cervical spinal cord injury.
Liquid Biopsy (lung cancer confirmatory blood test)

In May, BioTime’s affiliate, OncoCyte (NYSE MKT: OCX) presented positive results from its 300-patient multi-site R&D validation study for its lung cancer diagnostic test at the American Thoracic Society 2017 International Conference (ATS) in Washington, D.C. Results from this study of the optimized final predictive algorithm confirmed the data from a previous study completed in 2016 and further validate the test’s commercial potential.
OncoCyte is on track to launch its lung cancer confirmatory liquid biopsy diagnostic test in 2017. The test could eventually replace a high percentage of invasive, risky, and expensive lung biopsies with simple blood tests, improving outcomes for patients while also capturing significant cost savings for the U.S. healthcare system. The test targets a market opportunity believed to exceed $4 billion annually.
Simplification and Unlocking Value

New Subsidiary AgeX Therapeutics, Inc.

In April, BioTime announced the formation of AgeX Therapeutics, Inc. a new subsidiary that will focus on applying technology relating to cell immortality and regenerative biology, to aging and age-related diseases. AgeX has three initial areas of product development: pluripotent stem cell-derived brown adipocytes (AGEX-BAT1); vascular progenitors (AGEX-VASC1); and induced Tissue Regeneration (iTR). Initial planned indications for these products are Type II diabetes, cardiac ischemia, and cancer, respectively.
In August, AgeX closed an equity financing to raise $10 million. The transaction values AgeX at approximately $68 million. BioTime retains approximately 87% ownership of AgeX.
Value of Holdings in Public Affiliates

At June 30, 2017, BioTime held common stock in publicly-traded affiliates valued at $153.5 million. This amount was the market value of BioTime’s 21.7 million shares in Asterias Bio-Therapeutics (NYSE MKT: AST) and 14.7 million shares in OncoCyte (NYSE MKT: OCX).

Second Quarter Financial Results

Cash Position and Marketable Securities: Cash, cash equivalents, restricted cash in escrow, and available for sale securities totaled $20.9 million as of June 30, 2017, compared to $24.7 million as of March 31, 2017.

Revenues: BioTime’s revenue is generated primarily from research grants, licensing fees and royalties, and subscription and advertising from the marketing of online database products. Total revenue was $381,000 for the second quarter of 2017, compared to $1.3 million in the second quarter of 2016.

Operating Expenses: Operating expenses for the second quarter of 2017 were $10.7 million. On an adjusted basis, operating expenses were $8.8 million, of which $7.5 million was mainly attributable to our clinical programs, $0.8 million in expenses is expected to be funded by AgeX investors going forward and $0.5 million was incurred by our subsidiary LifeMap Solutions, expenses, which are not expected to recur.

Our operating expenses for the six months ended June 30, 2017 were $22.3 million. Adjusted operating expenses were $18.2 million for this period, including $14.4 million spent on our clinical and early stage programs. The remaining $3.8 million in expenses were contributed by OncoCyte during the period in 2017 in which it was consolidated or were in areas to be funded by AgeX going forward; these expenses are not expected to recur.

Cash expenditures in the first half of 2017 were higher than normal due to annual bonuses, AgeX formation costs and some project-based, non-recurring legal expenses. Cash expenditures were further impacted in the second quarter of 2017 due to timing of the payments of certain expenses, including executive bonuses and an extra payroll period.

The reconciliation between GAAP and non-GAAP operating expenses by entity, is provided in the financial tables included with this press release.

R&D Expenses: Research and development expenses were $6.3 million for the second quarter of 2017, compared to $8.9 million for the comparable period in 2016, a decrease of $2.6 million. This decrease was primarily attributable to the deconsolidation of Asterias in May 2016 and OncoCyte in February 2017.

G&A Expenses: General and administrative expenses were $4.4 million for the second quarter of 2017 compared to $6.6 million for the comparable period in 2016. The $2.2 million decrease was primarily due to the deconsolidation of Asterias and OncoCyte.

Net Income or loss attributable to BioTime: Net loss attributable to BioTime was $11.7 million, or ($0.11) per basic and diluted common share for the three months ended June 30, 2017, compared to net income of $24.5 million, or $0.26 per basic and diluted common share for the three months ended June 30, 2016. For the six months ended June 30, 2017, net income attributable to BioTime was $37.6 million, or $0.34 per diluted common share, compared to $7.4 million, or $0.08 per share for the six months ended June 30, 2016. Results in each period were primarily driven by noncash deconsolidation gains and noncash gains and losses in the changes in share prices of our public affiliate investments in Asterias and OncoCyte common stock.

On August 2, 2017 OncoMed Pharmaceuticals, Inc. (NASDAQ:OMED), a clinical-stage biopharmaceutical company focused on discovering and developing novel anti-cancer therapeutics, reported second quarter financial results (Press release, OncoMed, AUG 2, 2017, View Source [SID1234520005]). As of June 30, 2017, cash and short-term investments totaled $129.8 million.

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"OncoMed continues to focus on discovering and developing novel therapeutics to improve outcomes for cancer patients. The company is advancing our navicixizumab and rosmantuzumab phase 1b clinical trials and our immuno-oncology pipeline, with anti-TIGIT, GITRL-Fc and ongoing immuno-oncology discovery and R&D efforts," said Paul J. Hastings, OncoMed’s Chairman and CEO. "In addition, OncoMed is well positioned, with more than two years cash on the balance sheet and $98 million in potential opt-in payments by 2019."

Pipeline Highlights

Navicixizumab (anti-DLL4/VEGF bispecific; OMP-305B83)

Enrollment continues in two Phase 1b multi-center, open-label, dose escalation and expansion studies of OncoMed’s anti-DLL4/VEGF bispecific antibody in combination with standard of care chemotherapies: one in patients with 2nd line metastatic colorectal cancer and a second in patients with platinum-resistant ovarian cancer who have failed more than 2 prior therapies or prior bevacizumab.
Celgene Partnered — potential $25 million end of Phase 1 opt-in, $505 million in remaining milestones
Rosmantuzumab (anti-RSPO3; OMP-131R10)

Enrollment continues in a Phase 1a/b multi-center, open-label, dose escalation and expansion study of OncoMed’s anti-RSPO3 antibody in patients with advanced solid tumors (Phase 1a) and in patients with previously treated metastatic colorectal or gastric cancer (Phase 1b; in combination with FOLFIRI). As previously announced, the trial is now enrolling only patients that harbor an RSPO3 gene fusion.
Interim Phase 1a results demonstrated the drug was safe and well tolerated.
Celgene Partnered — potential $38 million end of Phase 1 opt-in, $440 million in remaining milestones
Anti-TIGIT (OMP-313M32)

Enrollment continues in a single-agent Phase 1a multi-center, open-label, dose escalation study of OncoMed’s anti-TIGIT antibody in patients with advanced or metastatic solid tumors.
Presented data in the 2nd Quarter from multiple preclinical studies detailing the mechanism of action and anti-tumor activity of anti-TIGIT alone and in combination with checkpoint inhibitors at the AACR (Free AACR Whitepaper) Annual Meeting 2017.
Celgene Partnered – potential $35 million end of Phase 1 opt-in, $440 million in remaining milestones
GITRL-Fc (OMP-336B11)

OncoMed expects to enroll the first-patient in a Phase 1a single agent study of OncoMed’s GITRL-Fc in 2H17
Wholly-owned
Vantictumab (anti-Fzd, OMP-18R5) and Ipafricept (Fzd8-Fc, OMP-54F28)

OncoMed continues to evaluate potential partnering opportunities for Wnt/IO combinations, utilizing different dosing regimens than those used in the Phase 1b studies.
Second Quarter 2017 Financial Results

Cash and short-term investments totaled $129.8 million as of June 30, 2017, compared to $184.6 million as of December 31, 2016.

Revenues were $6.2 million for the second quarter of 2017, a decrease of $0.5 million, compared to $6.7 million for the same period in 2016. The decrease in revenue was primarily due to slightly lower revenue recognized from reimbursement of research and development costs for services performed in the second quarter of 2017.

Research and development (R&D) expenses were $15.1 million for the second quarter 2017, a decrease of $14.6 million, compared to $29.7 million for the same period in 2016. The decrease was primarily due to lower external research and development costs attributable to the decrease in Phase 2 clinical trial costs of demcizumab and tarextumab programs and decrease in internal program costs due to reduced headcount as a result of the restructuring actions in April 2017.

General and administrative (G&A) expenses were $4.1 million for the second quarter of 2017, a decrease of $0.7 million, compared to $4.8 million for the same period in 2016. The decrease was mainly due to a decrease in employee-related costs including stock-based compensation expenses as a result of the restructuring actions in April 2017.

Restructuring charges were $2.4 million for the second quarter of 2017 as a result of the restructuring plan that was implemented in April 2017. The restructuring charges were primarily related to severance and other one-time benefits.

Net loss for the second quarter of 2017 was $15.2 million ($0.40 per share), compared to $27.7 million ($0.91 per share) for the same period of 2016. The change in net loss from the prior year quarter was due to lower R&D and G&A expenses and restructuring charges.

2017 Financial Guidance

OncoMed anticipates 2017 full-year cash expenses will be approximately $90 million. Based on the current plan, OncoMed anticipates that its current cash balance is sufficient to fund pipeline development and company operations through the third quarter of 2019, before considering potential opt-in milestones under our Celgene collaboration.

Following potential opt-in, on a per program basis, OncoMed would be eligible to co-develop and co-commercialize rosmantuzumab and/or navicixizumab with Celgene, while Celgene would assume all downstream costs and development activities for anti-TIGIT. In addition to the $98 million in potential opt-in payments related to these three programs, the company could be eligible to receive approximately $1.5 billion in downstream milestones, plus potential royalties and/or profit-sharing.