On August 10, 2015 Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a leading biotechnology company in the emerging field of regenerative medicine, reported financial and operating results for the second quarter ended June 30, 2015 (Press release, BioTime, AUG 10, 2015, View Source;p=RssLanding&cat=news&id=2078493 [SID:1234507130]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In the second quarter, we continued to make progress on multiple fronts toward advancing the clinical development of our key therapeutic programs," stated Pedro Lichtinger, President and CEO of Asterias. "We presented new, long-term follow-up Phase 2 clinical data of AST-VAC1 in acute myelogenous leukemia, the most common form of acute leukemia in adults, which showed potential for prolonged relapse-free survival in the overall study population, as well as in the sub-population of adults 60 years and older for which there remains a major unmet need. Based on the promising clinical data observed with AST-VAC1, we are pursuing opportunities to fund a focused clinical development plan for potential commercialization of AST-VAC1 through partnerships or non-dilutive funding."

Mr. Lichtinger continued, "In addition, we dosed the first patient in our Phase 1/2a clinical trial of AST-OPC1 for complete cervical spinal cord injury, a trial which is designed to evaluate the product at the doses and in the population where it has the maximum potential to bring benefit to patients. Importantly, we were successful in our efforts to further strengthen our balance sheet during the quarter by increasing our cash position by approximately $8 million, which has enhanced our financial flexibility to continue to execute our strategic plan through mid-2016. At the same time, during the quarter we completed some administrative tasks, namely the registration of all unregistered Asterias outstanding shares, including those shares held by our parent company BioTime. BioTime remains our largest shareholder and it is important to note that BioTime has not sold any of its position in Asterias since the June 10, 2015 effective date of the registration statement."

Recent Research and Development Highlights:

AST-VAC1 (antigen-presenting autologous dendritic cells)

Positive, new, long-term follow-up data from a Phase 2 clinical trial of AST-VAC1, the Company’s autologous telomerase-based dendritic cell cancer vaccine, in patients with intermediate and high risk acute myelogenous leukemia (AML) was presented at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June. The long-term follow-up showed that 57% of patients who received AST-VAC1 had prolonged relapse-free survival, even patients with high-risk AML including those over 60 years old and patients in second remission.

Asterias is in the process of establishing its clinical development plan for potential commercialization of AST-VAC1. The next step is planned to be a larger Phase 2/3 clinical trial designed to validate the positive findings from the completed Phase 2 study. Concurrently, the Company has commenced evaluation of strategic alternatives to further advance clinical development either independently or with a partner, and maximize the value of AST-VAC1.

AST-OPC1 (oligodendrocyte progenitor cells)

In June, the first patient was successfully dosed at the Atlanta-based Shepherd Center in a Phase 1/2a clinical trial evaluating activity of escalating doses of AST-OPC1 in newly injured patients with sensory and motor complete cervical spinal cord injury (SCI). The Phase 1/2a trial, to be conducted in eight to ten centers in the United States, is part of the planned registration program for AST-OPC1, with neurologically complete cervical SCI as the first targeted indication. The open-label, single-arm study will test three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in 13 patients with sub-acute, C-5 to C-7, neurologically complete cervical SCI. These individuals have essentially lost all sensation and movement below their injury site with severe paralysis of the upper and lower limbs. AST-OPC1 will be administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging methods to assess the safety and activity of the product. The Company expects availability of safety data from the first patient cohort in the Phase 1/2a trial during the second half of 2015, and initial efficacy data readouts from the trial in the second half of 2016.

If initial safety data from the 10 million cell cohort is positive, Asterias plans to file an Investigational New Drug Application (IND) amendment with the U.S. Food and Drug Administration (FDA) to expand enrollment in the study to include up to 40 patients with an adaptive design. An adaptive design clinical study is a study that includes a prospectively planned opportunity for modification of one or more specified aspects of the study design and hypotheses based on analysis of data from subjects in the study. The Company believes this flexible methodology can increase the chance to demonstrate the superiority of AST-OPC1 by increasing the statistical confidence of the safety and efficacy readouts, and position the product for potential accelerated regulatory approvals.

In the second quarter, Asterias received $1.1 million from the California Institute of Regenerative Medicine (CIRM) under the previously announced $14.3 million CIRM grant award for clinical development of AST-OPC1. CIRM disburses the grant funds in accordance with a quarterly disbursement schedule, subject to Asterias’ achievement of certain progress and safety milestones.

AST-VAC2 (antigen-presenting allogeneic dendritic cells)

Asterias is nearing completion of transfer of the cGMP-compatible AST-VAC2 process to development partner Cancer Research UK (CRUK). Confirmatory runs are in progress at both Asterias and CRUK, with completion of the full transfer expected in the fourth quarter of 2015. Following completion of the technology transfer, CRUK will, at its own cost, manufacture clinical grade AST-VAC2 and conduct the Phase 1/2a clinical trial in patients with non-small cell lung cancer in the UK, subject to regulatory approval. Asterias continues to expect potential regulatory clearance to begin treating patients as part of the Phase 1/2a trial in the second half of 2016.

Other Corporate Developments:

In May, Edward D. Wirth, III, M.D., Ph.D., was promoted to the newly created role of Chief Medical Officer. Dr. Wirth was Chief Translational Officer since joining Asterias in March 2013. In his new role, Dr. Wirth serves as an executive officer of the Company and provides strategic leadership for Asterias’ clinical development activities including its therapeutic programs, AST-OPC1 for spinal cord injuries, AST-VAC1 for AML and AST-VAC2 for lung cancer.

At the same time, Jane Lebkowski, President Research and Development was named as President of R&D and Chief Scientific Officer. Dr Lebkowski has been in the field and cell and gene therapy for 29 years and an employee of Asterias since March 2013. Dr Lebkowski is responsible for all research, product and regulatory development of Asterias products.

In May, Asterias received total proceeds of $11.7 million resulting from the exercise of all outstanding common share purchase warrants originally issued in June 2014. The proceeds will be used to further advance the Company’s development programs.
In June, Asterias was added to the Russell 2000, Russell 3000, Russell Global and Russell Microcap indexes following Russell Investments’ (Russell) reconstitution of its comprehensive set of U.S. and global equity indexes. Each June, Russell completely rebalances its indexes, known as a reconstitution, to reflect market changes in the past year. The Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for passive and active investment strategies.
Second Quarter [Unaudited] Financial Results

Total revenues in the second quarter 2015 were $772,000, which were primarily comprised of grant income and royalty revenues on product sales by licensees. Total revenues in the year ago quarter were $21,000. Operating expenses in the second quarter were $5.5 million, compared to $4.3 million in the prior year period. Research and development (R&D) expenses in the second quarter were $3.7 million, compared to $2.7 million in the year ago quarter. General and administrative (G&A) expenses in the second quarter were $1.8 million, compared to $1.5 million in the year ago quarter.

Net loss for the second quarter 2015 was $3.6 million, including a deferred income tax benefit of approximately $1.2 million. Net loss in the second quarter 2014 was $2.8 million, including a deferred income tax benefit of approximately $1.5 million. On a per share basis, net loss for the second quarter was $0.10 per share, compared to a loss of $0.09 per share for the year ago quarter.

Cash and cash equivalents were $15.6 million as of June 30, 2015, compared to $3.1 million as of December 31, 2014. In May 2015, Asterias received total proceeds of $11.7 million resulting from the exercise of all outstanding common share purchase warrants originally issued in June 2014. In addition, Asterias raised approximately $2.8 million in gross proceeds through the Company’s at-the-market (ATM) equity offering program during the second quarter of 2015. At June 30, 2015, the Company held 3.9 million BioTime common shares, with a market value of approximately $14.0 million on that date. For the second quarter, net cash used in operating activities was $4.7 million. The Company continues to expect net cash burn in 2015 to be in the range of $15 million to $17 million.

On August 10, 2015 AVEO Oncology (NASDAQ:AVEO) reported financial results for the second quarter ended June 30, 2015 (Press release, AVEO, AUG 10, 2015, View Source;p=RssLanding&cat=news&id=2078276 [SID:1234507127]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The second quarter was productive on multiple fronts for AVEO, including clinical updates, regulatory guidance and another partnership for tivozanib, in addition to completion of our corporate streamlining efforts," said Michael Bailey, president and chief executive officer. "These accomplishments have positioned us well to continue pursuing several additional value creating initiatives for tivozanib, including a potential confirmatory clinical and regulatory path forward for renal cell cancer in the US, as well as a potential marketing authorization application for renal cell cancer in Europe. We remain focused on executing against these goals as we continue to evaluate additional portfolio partnerships and further tivozanib development in colorectal cancer, throughout the balance of the year."

Recent Highlights

Announces Exclusive Licensing Agreement with Pharmstandard for Tivozanib in Russia, Ukraine and CIS. In August, AVEO announced that it has entered into an exclusive license agreement with a subsidiary of Pharmstandard Group for the development, manufacturing and commercialization of tivozanib in the territories of Russia, Ukraine and the Commonwealth of Independent States, for all indications excluding non-oncology ocular conditions. Under the terms of the agreement, Pharmstandard is obligated to pay AVEO an upfront payment of $1.5 million. AVEO is also eligible to receive up to $7.5 million in connection with the first marketing authorization of tivozanib in Russia, $3.0 million for each additional approved indication thereafter and a high single-digit royalty on net sales in the above mentioned territories. Pharmstandard will be responsible for all activities and costs associated with the further development, regulatory filings, health services and commercialization of tivozanib in the specified territories. A percentage of all upfront, milestone and royalty payments received by AVEO are due to Kyowa Hakko Kirin as a sublicensing fee.
Presented Additional Biomarker Analyses from BATON-CRC Tivozanib Study—At the ESMO (Free ESMO Whitepaper) 17th World Congress on Gastrointestinal Cancer in July, AVEO presented additional biomarker analyses from the BATON-CRC tivozanib study, the Company’s Phase 2 clinical trial of modified FOLFOX6 combined with tivozanib or bevacizumab in metastatic colorectal cancer (CRC). The data were presented in a poster discussion titled "Neuropilin 1 (NRP1) may be Prognostic and Identify a Subgroup of Patients with Metastatic Colorectal Cancer (mCRC) who Benefit from Tivozanib + mFOLFOX6 compared to Bevacizumab + mFOLFOX6."

Received European Regulatory Guidance Regarding Potential Marketing Authorization Application for Tivozanib in RCC—In June, AVEO announced that, following pre-submission advisory meetings to discuss the potential submission of a Marketing Authorization Application (MAA) for tivozanib as a treatment for Renal Cell Carcinoma (RCC) in Europe, it had received written guidance from the Rapporteur and co-Rapporteur appointed by the Committee for Medicinal Products for Human Use for the filing of such an application. The application would be based on the Company’s existing dataset, which includes results from the Phase 3 TIVO-1 study of tivozanib in the first-line treatment of RCC in which tivozanib demonstrated a significant improvement over sorafenib in the study’s primary endpoint of progression free survival. The Company is evaluating partnership opportunities to take tivozanib forward in Europe as it continues to prepare for an MAA filing.

Presented Final Results of Extension Study 902 of Tivozanib in RCC—At the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June, AVEO presented final results from its TIVO-1 extension study, known as Study 902, in which patients with advanced RCC received tivozanib as second-line treatment subsequent to disease progression on sorafenib in the Company’s Phase 3 TIVO-1 first-line RCC study. The results were presented in a poster presentation titled "Tivozanib vs sorafenib targeted therapy for advanced renal cell carcinoma: Final results of a phase III trial (901) and efficacy results of a 2nd line tivozanib extension study (902)."

Received FDA Regulatory Guidance Regarding Proposed Phase 3 RCC Study—In May, AVEO announced it had received a written response from the FDA stating that a Phase 3 study outlined by the Company, in patients with RCC who have failed at least two prior regimens, including VEGF therapy, "may support AVEO’s proposed indication for tivozanib in the 3rd line setting." In response to whether the study, together with the TIVO-1 study, would be sufficient to also support licensure of tivozanib as a treatment for advanced [first line] RCC, the FDA indicated: "whether the results from this [third line] study can support AVEO’s proposal for tivozanib in the first line setting is a review issue." The Company continues to evaluate all options, including partnerships, for the clinical and regulatory advancement for tivozanib in RCC.

Received FDA Feedback for Tivozanib in CRC—In June, AVEO announced that it had received feedback from the U.S. Food and Drug Administration (FDA) regarding a potential pivotal study for tivozanib in the treatment of NRP-1 low (below the median, representing 50% of the population) CRC. This feedback is consistent with the Company’s current clinical strategy and discussions with cancer research cooperative groups. As such, AVEO plans to identify a commercially viable assay which will enable a prospectively defined, randomized Phase 2 study.

Relocated Corporate Headquarters—In May, AVEO announced the relocation of its corporate headquarters to One Broadway in Cambridge, Massachusetts. Consistent with the Company’s goal of streamlining operations to align with its strategic needs going forward, the new facility consists of approximately 5,000 square feet of office space under flexible lease terms, with no laboratory or vivarium space.

Presented AV-380 Preclinical Data in Cancer Associated Cachexia – At the 2015 Annual Meeting of the American Association of Cancer Research in April, AVEO presented results from a preclinical study of AV-380, the Company’s potent, humanized inhibitory antibody targeting growth differentiation factor 15 (GDF15), in a cachectic human tumor xenograft model with significantly increased plasma GDF15 levels. The data were presented in a poster titled "Effective treatment of cancer associated cachexia by AV-380, a GDF15 inhibitory antibody".

Second Quarter 2015 Financial Highlights

AVEO ended Q2 2015 with $26.8 million in cash and cash equivalents.
Total collaboration revenue was approximately $0.1 million compared with $1.8 million for Q2 2014. The decrease was primarily due to an additional $1.8 million in revenue recognized in connection with our agreement with Astellas, which concluded in August 2014.

Research and development (R&D) expense was $1.8 million compared with $9.3 million for Q2 2014. The decrease in R&D expense was primarily due to a reduction in personnel-related expenses following our January 2015 strategic restructuring, the reduction of our leased facilities, as well as a decrease in external clinical trial and consulting costs associated with the decreased tivozanib clinical development activity and AV-380 preclinical development activity.

General and administrative (G&A) expense was $2.9 million compared with $4.8 million for Q2 2014. The decrease in G&A expense was primarily due to a reduction in external legal costs associated with various ongoing legal matters and a decrease in employee compensation, facilities and IT costs following our January 2015 restructuring and the reduction of our leased facilities.
Restructuring and lease exit expense was $25,000 compared with $5.2 million for Q2 2014. The expense incurred during Q2 2015 related to accretion expense associated with the lease termination liability for the 650 E. Kendall Street facility, whereas the expense incurred during Q2 2014 related to the portion of the 650 E. Kendall Street facility that we ceased using during that quarter.

Net loss for Q2 2015 was $5.5 million, or a loss of $0.10 per basic and diluted net loss per share compared with net loss of $18.0 million or a loss of $0.35 per basic and diluted net loss per share for Q2 2014.

Financial Guidance

We believe that our cash resources will allow us to fund our current operations at least through the third quarter of 2016. This estimate does not include our payment of potential licensing milestones or the costs of conducting any contemplated clinical trials and assumes no milestone payments from our partners, additional funding from new partnership agreements, equity financings, debt financings or accelerated repayment thereof or further sales under our ATM. The timing and nature of activities contemplated for 2015 and 2016 will be conducted subject to the availability of sufficient financial resources.

On August 10, 2015 Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing new therapeutics and molecular diagnostics that target the underlying mechanisms of cancer, reported that William G. Rice, Ph.D., Chairman, President and Chief Executive Officer, will present at the upcoming Canaccord Genuity 35th Annual Growth Conference on Thursday, August 13th at 9:00 a.m. ET at the Intercontinental Hotel Boston, MA. Dr. Rice will provide a corporate overview of the Company’s recent activities and strategic direction including plans to develop Aptose’s lead clinical agent, APTO-253 for acute myeloid leukemia (AML), myelodysplastic syndromes (MDS) and other hematologic malignancies (Filing, 6-K, Aptose Biosciences, AUG 10, 2015, View Source [SID:1234507121]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live audio webcast of the Aptose presentation will be accessible by visiting:

View Source

The audio webcast will be archived shortly after the live event and will be available for 90 days through the Aptose website at www.aptose.com.

On August 10, 2015 Aduro Biotech, Inc. (NASDAQ: ADRO) reported financial results for the second quarter ended June 30, 2015 (Filing, 8-K, Aduro BioTech, AUG 10, 2015, View Source [SID:1234507119]). Net loss was $26.3 million for the second quarter of 2015, or $0.50 per share, and $42.9 million, or $1.61 per share, for the six months ended June 30, 2015, compared to net loss of $3.6 million, or $12.27 per share, and $11.4 million, or $38.61 per share respectively, for the same periods in 2014.

Cash and cash equivalents totaled $465.9 million at June 30, 2015, compared to $119.5 million at December 31, 2014. Total cash at June 30, 2015 included a $200.0 million upfront payment from Novartis Pharmaceuticals Corporation under the companies’ collaboration agreement, $124.2 million in net proceeds from Aduro’s initial public offering and an additional $25.0 million from a private placement to Novartis concurrent with the company’s IPO.

"We are making significant progress in our existing oncology programs driven by our in-house research and development teams and in collaboration with our academic and corporate partners, including Novartis and Janssen, and believe there is tremendous potential to explore new indications with our immunotherapy platform technologies," said Stephen T. Isaacs, chairman, president and chief executive officer of Aduro. "We look forward to completing enrollment in our Phase 2b ECLIPSE trial in pancreatic cancer and initiating trials in prostate and lung cancers, as well as multiple other cancers, with immunotherapeutic agents derived from our platforms. In addition, based on continued encouraging data from our Phase 1b trial and following recent meetings with U.S. and European regulatory authorities, we are now planning to advance our mesothelioma program into a randomized global Phase 3 clinical trial next year."

Recent Progress

· Follow up of the seven long-term survivors in Phase 2a pancreatic cancer trial continues, with two patients continuing to receive the combination regimen of CRS-207 and GVAX Pancreas for almost three years

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

· Completed patient enrollment in the Phase 1b mesothelioma trial evaluating the combination of CRS-207 and standard chemotherapy

· Presented updated data from Phase 1b mesothelioma trial at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Meeting demonstrating 94% disease control following treatment with CRS-207 and standard chemotherapy

· Conducted meetings with the U.S. FDA and Paul-Ehrlich-Institut to discuss Phase 3 plans for mesothelioma program

· Published notable preclinical results for the STING-targeted CDN immuno-oncology platform in Science and Translational Medicine and Cell Reports

Key Upcoming Milestones

· Complete enrollment in Phase 2b ECLIPSE trial in pancreatic cancer in the third quarter of 2015 and report top line results in the first half of 2016

· Report top line results from the Phase 1b trial in mesothelioma in the first half of 2016

· Complete enrollment in Phase 2 STELLAR trial in pancreatic cancer in the first quarter of 2016 and report interim results in the second half of 2016

· Initiate randomized Phase 3 trial in mesothelioma in the first half of 2016

· Initiate Phase 1 trials in lung and prostate cancer with novel LADD agents in collaboration with Janssen in the first quarter of 2016

· Initiate Phase 1 trial in cutaneously accessible tumors with novel CDNs in collaboration with Novartis in the first half of 2016

Revenues were $9.9 million for the second quarter of 2015 and $19.5 million for the six months ended June 30, 2015, compared to $1.0 million for each of the three and six months ended June 30, 2014. The increase was primarily due to recognition of a portion of the upfront fees and development-related milestones achieved under the Janssen agreements.

Research and development expenses were $13.5 million for the second quarter of 2015 and $24.2 million for the six months ended June 30, 2015, compared to $5.4 million and $10.1 million, respectively, for the same periods in 2014. This increase was primarily due to clinical and manufacturing expenses related to the Phase 2b ECLIPSE clinical trial of CRS-207/GVAX Pancreas immunotherapy in pancreatic cancer, licensing fees and compensation costs due to continued growth in the number of personnel.

General and administrative expenses were $5.9 million for the second quarter of 2015 and $12.1 million for the six months ended June 30, 2015, compared to $2.1 million and $3.5 million, respectively, for the same periods in 2014. This increase was primarily due to increased personnel expenses to support the company’s expanding operations.

Loss from remeasurement of fair value of warrants was $16.7 million for the second quarter of 2015 and $26.1 million for the six months ended June 30, 2015, due to changes in the fair value of liability-classified warrants to purchase Aduro’s preferred and common stock. In April 2015, all such warrants ceased being liability-classified as the contingency surrounding the number of shares issuable upon the warrant exercise expired. As of June 30, 2015, all outstanding warrants were equity-classified and not subject to remeasurement.