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Aduro Biotech Reports Second Quarter 2017 Financial Results

On August 2, 2017 Aduro Biotech, Inc. (NASDAQ:ADRO) reported financial results for the second quarter of 2017 (Press release, Aduro Biotech, AUG 2, 2017, View Source [SID1234519990]). Net loss for the second quarter 2017 was $19.4 million, or $0.27 per share, and for the six months ended June 30, 2017 net loss was $41.2 million, or $0.59 per share, compared to net income of $2.3 million, or $0.04 per share, and net loss of $26.5 million, or $0.41 per share, respectively, for the same periods in 2016.

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Cash, cash equivalents and marketable securities totaled $377.2 million at June 30, 2017, compared to $361.9 million at December 31, 2016.

"We are making great progress as we approach a number of near-term milestones across all three of our distinct immunotherapy platforms," said Stephen T. Isaacs, chairman, president and chief executive officer of Aduro. "In the remaining period of 2017, we expect to initiate a number of new clinical trials, including a combination trial with ADU-S100 and anti-PD-1; a first-in-human Phase 1 trial with pLADD, a personalized second-generation LADD targeting neoantigens, in patients with certain colorectal cancers; and a first-in-human clinical trial with our lead B-select candidate, an anti-APRIL monoclonal antibody, in multiple myeloma. With these anticipated new trials, all of our technology platforms will be in the clinic. In addition, we expect to share preliminary clinical data from the ongoing Phase 1 dose escalation monotherapy trial of ADU-S100, as well as preliminary data from the ongoing Phase 2 trial of CRS-207 and anti-PD-1 in mesothelioma. With a comprehensive portfolio of investigational immunotherapies, we are poised with multiple opportunities to deliver on our goal of building a successful biotech company by bringing innovative medicines to patients."

Key Recent Accomplishments

Established a clinical collaboration with Merck to evaluate the combination of Aduro’s LADD agent CRS-207 with Merck’s anti-PD-1 KEYTRUDA (pembrolizumab) in a Phase 2 trial in mesothelioma cancer and subsequently initiated this trial
Received FDA clearance of an Investigational New Drug Application (IND) for the Phase 1b study of Aduro’s ADU-S100 and PDR001, Novartis’ anti-PD-1 checkpoint inhibitor
Initiated a Phase 2 clinical trial of CRS-207 in combination with pembrolizumab for patients with previously-treated gastric cancer
Earned a $2 million milestone under a worldwide licensing agreement with Merck for work supporting the preparation of an IND for the B-select anti-CD27 monoclonal antibody
Remaining Anticipated 2017 Milestones

Initiate Phase 1 pLADD (personalized LADD) trial in certain colorectal cancers
Janssen expected to initiate Phase 1b/2 trial of ADU-214 in lung cancer and determine next steps for ADU-741 in prostate cancer
Initiate Phase 1b trial of ADU-S100 in combination with anti-PD-1 in collaboration with Novartis
Report early results from the Phase 2 mesothelioma study evaluating CRS-207 in combination with pembrolizumab
Report preliminary top-line findings from Phase 1 monotherapy trial of ADU-S100
File an IND for BION-1301, an anti-APRIL antibody
Initiate Phase 1 multiple myeloma trial with BION-1301, an anti-APRIL antibody
Second Quarter 2017 Financial Results

Revenue was $5.9 million for the second quarter of 2017 and $9.7 million for the six months ended June 30, 2017, compared to $39.0 million and $43.0 million, respectively, for the same periods in 2016. The decrease in revenue in both periods is due to the recognition of a $35.0 million milestone payment in the second quarter of 2016 in connection with the clinical advancement of ADU-S100 under our agreement with Novartis. For the second quarter of 2017, the decrease was partially offset by the recognition of $2.0 million in connection with the achievement of a milestone under our anti-CD27 antibody agreement with Merck.

Research and development expenses were $21.4 million for the second quarter of 2017 and $42.0 million for the six months ended June 30, 2017, compared to $26.9 million and $47.8 million, respectively, for the same periods in 2016. The decrease in research and development expenses in both periods was primarily related to reduced GVAX Pancreas manufacturing and pancreatic cancer clinical trial expenses, partially offset by increased costs to manufacture our B-select antibodies as well as higher personnel and facility related costs in 2017.

General and administrative expenses were $8.3 million for the second quarter of 2017 and $16.5 million for the six months ended June 30, 2017, compared to $8.7 million and $17.7 million, respectively, for the same periods in 2016. The decrease in general and administrative expenses in both periods was primarily related to lower professional services and consulting expenses in 2017, partially offset by higher facility costs in 2017.

Income tax benefit was $3.8 million for the second quarter of 2017 and $6.5 million for the six months ended June 30, 2017, compared to a provision for income taxes of $1.5 million and $4.7 million, respectively, for the same periods in 2016. The income tax benefit recorded in 2017 was due to the current benefit of federal income taxes paid in 2016.

BioTime Reports Second Quarter Results and Recent Corporate Accomplishments

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 [SID1234519988]).

"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.

Advaxis to Present at Third CRI-CIMT-EATI-AACR International Cancer Immunotherapy Conference: Translating Science into Survival

On August 2, 2017 Advaxis, Inc. (NASDAQ:ADXS), a late-stage biotechnology company focused on the discovery, development and commercialization of immunotherapy products, reported three abstracts were selected for presentation at the third annual CRI-CIMT-EATI-AACR International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper): Translating Science into Survival (Press release, Advaxis, AUG 2, 2017, View Source [SID1234519986]). The prestigious program brings together leading stakeholders in the field to teach, learn and disseminate the latest cutting-edge information and research in cancer immunology and immunotherapy.

Two poster presentations will feature preliminary immune correlative data from the ADXS-PSA monotherapy treatment arm of Advaxis’ Phase 1/2 study in combination with KEYTRUDA (pembrolizumab) in metastatic castration-resistant prostate cancer (mCRPC) patients. The third presentation will feature preclinical data with the company’s HPV-targeted immunotherapy, axalimogene filolisbac.

The poster presentation, "Gene expression profiles associated with stable disease in metastatic castration-resistant prostate cancer patients treated with ADXS-PSA immunotherapy," will feature preliminary data identifying potential pharmacodynamic biomarkers of clinical response in mCRPC patients treated with ADXS-PSA monotherapy.

The second poster presentation, "Persistence of expanded TCRβ clonotypes is associated with clinical activity of ADXS-PSA immunotherapy in metastatic castration-resistant prostate cancer," will focus on the stability of expanded T cell clones in mCRPC patients following treatment with ADXS-PSA monotherapy. The preliminary immune correlative data will be presented by Sandy Hayes, Ph.D., Senior Director of Research and Biomarker Lead at Advaxis.

The third poster presentation, "Identification of an intratumoral immune gene signature associated with tumor regression in an axalimogene filolisbac-treated murine HPV+ tumor model," will include preclinical data regarding molecular biomarkers associated with tumor regression in a murine HPV+ tumor model. It will be presented by Rachelle Kosoff, Ph.D., Senior Scientist in Research at Advaxis.

"Our findings provide important insights into identifying those patients who are benefiting from these treatments," said Dr. Hayes. "We’re pleased to share our findings with the rest of the community at the International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) as we continue our work to bring new treatment options to patients."

The third annual International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) takes place in the city of Mainz near Frankfurt, Germany from Sept. 6 – 9, 2017, and is jointly sponsored by the Cancer Research Institute (CRI), the Association for Cancer Immunotherapy (CIMT) (Free CIMT Whitepaper), the European Academy of Tumor Immunology (EATI) and the American Association for Cancer Research (AACR) (Free AACR Whitepaper). Presentations will cover all areas of inquiry in cancer immunology and immunotherapy, including: neoantigens and vaccines, novel mechanisms of immunosuppression and immune evasion, biomarkers, new checkpoint immunotherapies and the tumor microenvironment.

To learn more about Advaxis and its immunotherapy clinical programs, visit www.advaxis.com/clinical-trials.

Helsinn Group and MEI Pharma Announce First Patient Dosed in Pivotal Phase 3 Study of Pracinostat and Azacitidine in Acute Myeloid Leukemia

On August 2, 2017 Helsinn, a Swiss pharmaceutical group focused on building quality cancer care products, and MEI Pharma, Inc. (Nasdaq: MEIP), an oncology company focused on the clinical development of novel therapies for cancer, reported that the first patient has been dosed in the pivotal Phase 3 study of the investigational agent pracinostat in combination with azacitidine in adults with newly diagnosed acute myeloid leukemia (AML) who are unfit to receive intensive induction chemotherapy (Press release, MEI Pharma, AUG 2, 2017, View Source [SID1234519985]).

The randomized, double-blind, placebo-controlled study will enroll approximately 500 eligible patients worldwide. Patients will be randomized 1:1 to receive pracinostat or placebo with azacitidine as background therapy. The primary endpoint of the study is overall survival. Secondary endpoints include, among others, morphologic complete remission (CR) rate, event free survival (EFS) and duration of CR.

Riccardo Braglia, Helsinn Group Vice Chairman and CEO, said: "Helsinn was delighted to be able to announce our strategic partnership with MEI Pharma last year, leveraging on the potential of pracinostat, which was demonstrated in the Phase 2 study. We are very pleased that pracinostat is moving into Phase 3, showing the continued momentum of the clinical programme. As Helsinn broadens its focus beyond cancer supportive care and into cancer therapeutics, high quality partnerships such as our collaboration with MEI Pharma are key for Helsinn to create value and benefit more people with cancer."

"The initiation of this highly anticipated study is the culmination of diligent preparation in collaboration with our partners at Helsinn," said Daniel P. Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "AML is a rapidly progressing, often fatal disease, and patients who are unable to undergo intensive therapies are in urgent need of new treatment options. We believe that with the well-powered, rigorously designed Phase 3 study underway, pracinostat is now one pivotal step closer to serving this need. Meanwhile, enrollment in our Phase 2 dose-optimization study of pracinostat and azacitidine in myelodysplastic syndrome (MDS) continues as planned and we look forward to reporting interim data from the MDS study early next year."

The Phase 3 study of pracinostat in combination with azacitidine is open to adult patients with newly diagnosed AML who are unfit to receive standard induction chemotherapy due to age ≥ 75 years or predefined co-morbidities. Treatments will be administered based on 28-day cycles, with pracinostat or placebo administered orally once every other day, 3 times a week for 3 weeks, followed by one week of no treatment and azacitidine administered for 7 days of each cycle. Additional information regarding the study, including detailed inclusion and exclusion criteria, is available at ClinicalTrials.gov (identifier: NCT03151408).

Results from a Phase 2 open-label, single-arm, multicenter study of pracinostat and azacitidine in 50 patients aged ≥ 65 years with newly diagnosed AML not eligible for induction chemotherapy showed a median overall survival of 19.1 (95%CI: 10.0-26.5) months, one-year survival of 62% and a CR rate of 42%. Responses were durable (median CR+CRi 17.2 months). Blast clearance was rapid (median 8 weeks) while CR required prolonged therapy (>6 months) in some patients. The combination of pracinostat and azacitidine had no unexpected toxicities. The most common grade 3/4 treatment-emergent adverse events reported in >10% of all patients included thrombocytopenia, febrile neutropenia, neutropenia, fatigue and anemia. These results were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2016.

About AML
AML is the most common acute leukemia affecting adults, and its incidence is expected to continue to increase as the population ages. The American Cancer Society estimates about 21,380 new cases and 10,590 deaths from AML in the U.S. for 2017; the average age of a patient with AML is about 67 years. According to the Surveillance of Rare Cancers in Europe project, the incidence of AML in Europe is 3.7 per 100,000. There are currently no drugs approved in the U.S. to treat AML in patients who are unfit for intensive induction chemotherapy, though hypomethylating agents are recommended by the National Comprehensive Cancer Network (NCCN) guidelines. In the EU, azacitidine is approved for the treatment of adult patients aged 65 years or older who are not eligible for hematopoietic stem cell transplant (HSCT) with AML with >30% marrow blasts according to the World Health Organization (WHO) classification.

About Pracinostat
Pracinostat is an oral histone deacetylase (HDAC) inhibitor that is in late-stage clinical development. The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation for pracinostat in combination with azacitidine for the treatment of patients with newly diagnosed AML who are ≥75 years of age or unfit for intensive chemotherapy. In August 2016, Helsinn and MEI Pharma entered into an exclusive license, development and commercialization agreement for pracinostat in AML and other potential indications. Under the terms of the agreement, Helsinn is granted a worldwide exclusive license to develop, manufacture and commercialize pracinostat, and is primarily responsible for funding its global development and commercialization. Pracinostat is an investigational agent and is not approved for commercial use in the U.S.

Kite Doses First Patient in the Phase 2 Trial of Axicabtagene Ciloleucel in Indolent B-Cell Non-Hodgkin Lymphoma (ZUMA-5)

On August 2, 2017 Kite Pharma, Inc., (Nasdaq:KITE), a leading cell therapy company, reported that patients with relapsed/refractory indolent B-cell non-Hodgkin lymphoma (iNHL) are now being treated in its Phase 2 ZUMA-5 trial with its lead investigational candidate, axicabtagene ciloleucel (axi-cel) (Press release, Kite Pharma, AUG 2, 2017, View Source [SID1234519984]). This study builds upon early clinical results using the same axi-cel anti-CD19 construct previously published in the March 2012 issue of Blood, "B-cell Depletion and Remissions of malignancy along with cytokine-associated toxicity in a clinical trial of anti-CD19 chimeric-antigen-receptor-transduced T cells,"1 in which patients with relapsed/refractory iNHL experienced a high response rate and durable disease remissions in a clinical trial at the National Cancer Institute (NCI).

"Despite recent advances in the treatment of NHL, iNHL generally remains an incurable disease. As a result, there is a great unmet need in patients with high-risk disease which is why the focus of this study has the opportunity to make a difference," said David Chang, M.D., Ph.D., Executive Vice President of Research and Development and Chief Medical Officer of Kite. "The dosing of the first patient in ZUMA-5 marks an important milestone in our commitment to serving patients. We will continue to explore the potential for axi-cel to treat hematologic malignancies beyond aggressive NHL, the foundation of our CAR-T program."

B-cell iNHL is expected to account for approximately 25 percent of 2017 newly diagnosed NHL cases in the United States (U.S.). Follicular lymphoma is the most common type of iNHL and one of the most common subtypes of NHL. The company estimates that there will be approximately 4,700 patients in 2017 with follicular lymphoma in the U.S. who could potentially benefit from CAR-T therapy.

ZUMA-5 is a single-arm, open-label, multi-center study in patients with iNHL whose disease has relapsed within two years of first line treatment, is refractory to second line or greater therapy or has relapsed at any point after transplant. The study will enroll approximately 50 patients. Additional details about this study can be found on ClinicalTrials.gov, using identifier NCT: 03105336.

About axicabtagene ciloleucel

Kite’s lead product candidate, axicabtagene ciloleucel is an investigational therapy in which a patient’s T cells are engineered to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells. Axicabtagene ciloleucel is currently under review by the U.S. Food and Drug Administration (FDA) for aggressive non-Hodgkin’s lymphoma and was granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL).