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8-K – Current report

On November 9, 2015 Galena Biopharma, Inc. (NASDAQ: GALE), a biopharmaceutical company committed to the development and commercialization of targeted oncology therapeutics that address major unmet medical needs, reported its financial results for the quarter ended September 30, 2015 (Filing, 8-K, Galena Biopharma, NOV 9, 2015, View Source [SID:1234508156]). The Company also announced it has completed a strategic review of the organization and has elected to focus its efforts and financial resources exclusively on the continued development of its high value oncology pipeline led by NeuVax (nelipepimut-S), and divest its commercial business which consists of Abstral (fentanyl) Sublingual Tablets and Zuplenz (ondansetron) Oral Soluble Film.

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For financial and accounting purposes, Galena has classified its commercial business activities as discontinued operations effective as of the third quarter, and the Company removes all revenue and expense guidance as it relates to its commercial business. Galena has engaged a financial advisor to provide strategic advice and a process to divest the commercial business, and the Company anticipates exiting the commercial business by the end of the first quarter of next year. Providers and patients will have ongoing access to both drugs until we have transitioned out of the business.

"Led by NeuVax, Galena has an extremely robust clinical development pipeline targeting areas of high unmet medical need that represent significant high-value market opportunities for the company," said Mark W. Schwartz, Ph.D., President & CEO. "Over the past year, we have met several key development milestones while also expanding our clinical pipeline to four assets in eight ongoing or planned clinical trials. Our strategy going forward is to advance these programs while exploring additional indications in the immuno-oncology field where our assets can potentially make a difference in the treatment of cancer or in addressing the rapidly growing patient population of cancer survivors by harnessing the power of the immune system to prevent their cancer recurrence."

Dr. Schwartz continued, "When I assumed the position of President and CEO of Galena, I, along with our executive team, began a careful examination of our operations and assets to determine the optimal strategy for Galena that would enable the greatest opportunity for growth, while maximizing shareholder value. As a result of this analysis and review by our Board of Directors, we have concluded that it is in the best interest of our patients, our shareholders, and the long-term success of our company to focus our energy and resources exclusively on our clinical development programs. Since acquiring the products we have significantly grown the sales of Abstral and successfully launched Zuplenz, and I believe that each has strong commercial potential and offers significant benefits to their respective patient populations. However, the foundation of Galena has always been our cancer immunotherapy programs, which are now rapidly advancing towards several key inflection points. Therefore, we believe it is important for Galena to focus on our core expertise and the successful advancement of our late and mid stage clinical pipeline. We appreciate the dedication and hard work of the commercial team as we transition out of the commercial business and are extremely grateful for all of their efforts."

Dr. Schwartz concluded, "For both patients and shareholders of Galena, there is a much greater opportunity to generate value if we dedicate all of our resources to our clinical programs, and we are eager to move the company in this new direction. As part of this renewed focus, we have officially consolidated at our new headquarters in San Ramon, California. We look forward to discussing these advances in more detail during our third quarter earnings webcast this afternoon."

Galena will host a webcast and conference call today at 2:00 p.m. P.T./5:00 p.m. E.T. to discuss financial and business results. The live webcast will include slides that can be accessed on the Company’s website under the Investors section/Events and Presentations: View Source The conference call can be accessed by dialing (844) 825-4413 toll-free in the U.S., or (973) 638-3403 for participants outside the U.S. The Conference ID number is: 54883466. The archived webcast replay will be available on the Company’s website for 90 days.

FINANCIAL HIGHLIGHTS AND GUIDANCE

As a result of our strategic decision to divest our commercial business, our commercial activities are classified as discontinued operations in our third quarter financial statements.

Operating loss from continuing operations for the third quarter of 2015 was $8.6 million, including $0.6 million in stock based compensation, compared to an operating loss from continuing operations of $10.6 million, including $1.1 million in stock-based compensation for the same period in 2014. Operating loss from continuing operations through the third quarter of 2015 was $26.6 million, including $1.3 million in stock based compensation, compared to an operating loss from continuing operations of $34.2 million, including $3.9 million in stock-based compensation for the same period in 2014. The decrease in net operating loss year-over-year is primarily the result of the completion of enrollment in our Phase 3 PRESENT trial for NeuVax, as well as the decrease in stock based compensation, and a reduction in legal expenses associated with ongoing litigation and legal proceedings.

Non-operating income or expenses include non-cash charges related to changes in the fair value estimates of the company’s warrant liabilities, contingent purchase price liability, and interest expense. The non-cash income related to the changes in the value of our warrant liability for the third quarter of 2015 was $2.1 million versus $6.7 million for the same period in 2014, respectively. The non-cash expense related to the changes in the value of our warrant liability through the third quarter of 2015 was $1.0 million versus a non-cash income of $13.2 million for the same period in 2014, respectively.

Loss from continuing operations for the third quarter of 2015 was $6.4 million, including $2.1 million in non-cash income described above, or $0.04 per basic and diluted share. Loss from continuing operations for the third quarter of 2014 was $3.5 million, including a $6.7 million in non-cash income described above, or $0.03 per basic and diluted share. Loss from continuing operations through the third quarter of 2015 was $28.2 million, including $1.0 million in non-cash expense described above, or $0.18 per basic and diluted share. Loss from continuing operations through the third quarter of 2014 was $22.0 million, including $13.2 million in non-cash income described above, or $0.19 per basic and diluted share.

Loss from discontinued operations for the third quarter of 2015 was $11.7 million, or $0.07 per basic and diluted share, compared to $2.6 million, or $0.02 per basic and diluted share, for the same period of 2014. Loss from discontinued operations through the third quarter of 2015 was $16.1 million, or $0.11 per basic and diluted share, compared to $6.6 million, or $0.06 per basic and diluted share, for the same period of 2014. Loss from discontinued operations include a $8.1 million non-cash impairment charge from classification of assets held for sale for the three and nine months ended September 30, 2015.

As of September 30, 2015, Galena had cash and cash equivalents of $34.8 million, compared with $23.7 million as of December 31, 2014. The $11.1 million increase in cash through the third quarter of 2015 represents $47.4 raised from issuance of common stock, partially offset by $27.8 million used in continuing operating activities, $5.0 million used in discontinued operating activities, $0.5 million milestone payment for Zuplenz, and $3.0 million in debt service payments.

THIRD QUARTER AND RECENT HIGHLIGHTS

Presented GALE-302 Preliminary Immunological Data Optimizing GALE-301 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 30th Anniversary Annual Meeting. The poster, entitled, "Preliminary report of a clinical trial supporting the sequential use of an attenuated E39 peptide (E39’) to optimize the immunologic response to the FBP (E39+GM-CSF) vaccine," compared three primary vaccine series (PVS) sequences of GALE-301 (E39) and GALE-302 (E39’) in ovarian and breast cancer patients to optimize the ex vivo immune responses, local reactions (LR), and delayed type hypersensitivity (DTH) reactions. The data demonstrated that the in vivo immune response is enhanced with the use of the attenuated E39’ (GALE-302) after E39 (GALE-301). The optimal vaccination sequence utilizing three inoculations of GALE-301 followed by three inoculations of GALE-302 produced the most prominent and statistically significant LR and DTH responses.

Announced the collaboration with the National Cancer Institute (NCI) to initiate a new, Phase 2 Clinical Trial With NeuVax in Ductal Carcinoma in Situ (DCIS) Patients. The trial will be entitled, VADIS: Phase 2 trial of the Nelipepimut-S Peptide VAccine in Women with DCIS of the Breast, and The University of Texas M.D. Anderson Cancer Center (MDACC) Phase I and II Chemoprevention Consortium is the lead for this multi-center trial. The Consortium is funded through the Division of Cancer Prevention at NCI, which will provide financial and administrative support for the trial. Galena will provide NeuVax, as well as additional financial and administrative support. The trial is expected to initiate in the fourth quarter of 2015.

Presented Positive GALE-301 Phase 2a Clinical Trial Data at the European Cancer Congress 2015. Poster #P427 (abstract #2764), entitled "Preliminary results of the phase I/IIa dose finding trial of a folate binding protein vaccine GALE-301 (E39) + GM-CSF in ovarian and endometrial cancer patients to prevent recurrence," provided updated data for all patients who had received at least twelve months of treatment. As presented, the clinical recurrence rate based on all treatment cohorts was 41% in the Vaccine Group (VG) (n=29) versus 55% in the Control Group (CG) (n=22), p=0.41. However, in the 1000 mcg VG cohort (n=15), there have only been two clinical recurrences (13.3% versus 55% CG, p=0.02), and the two-year Disease Free Survival (DFS) estimate is 85.7% versus 33.6% in the CG, p < 0.02, as compared by Kaplan-Meir and Log rank tests. Demographic, safety, immunologic, and clinical recurrence data are continuing to be collected.

The Independent Data Safety Monitoring Committee (IDMC) Recommended Reduction of Cardiac Toxicity Monitoring for the NeuVax PRESENT (Prevention of Recurrence in Early-Stage, Node-Positive Breast Cancer with Low to Intermediate HER2 Expression with NeuVax Treatment) Clinical Trial. Following its most recent IDMC meeting in June 2015, the IDMC recommended routine cardiac monitoring could be reduced in the PRESENT trial and that such a reduction is justified and consistent with the pre-specified Cardiac Toxicity Monitoring Stopping Rules defined in the study protocol. The IDMC concluded that cardiac toxicity monitoring by echocardiogram (ECHO) or multiple-gated acquisition (MUGA) scans could be reduced. The IDMC had no other suggestions and recommended the trial continue as planned.

CORPORATE HIGHLIGHTS

Appointed Bijan Nejadnik, M.D., as Executive Vice President and Chief Medical Officer. Dr. Nejadnik will be responsible for managing all of Galena’s clinical development programs and joins Galena from Jazz Pharmaceuticals where he was the Executive Director, Hematology-Oncology and led the clinical team towards a recently filed new drug application. Prior to Jazz, Dr. Nejadnik was at Johnson & Johnson and spent more than 13 years in teaching, research and caring for patients at world-renowned academic institutions. Dr. Nejadnik graduated from the University of Louvain in Belgium for both his undergraduate degree in premedical studies and his medical degree, and completed his internship and residency programs specializing in internal medicine focused on hematology-oncology at the University of Louvain and Oregon Health Sciences University. He completed his fellowships at Cornell University’s Weill Medical College and Johns Hopkins University School of Medicine. Dr. Nejadnik has led or participated in more than 20 peer-reviewed publications.

DelMar Pharmaceuticals to Present Updated Phase II Safety and Efficacy Data on VAL-083 in Refractory Glioblastoma Multiforme

On November 9, 2015 DelMar Pharmaceuticals, Inc. (OTCQX: DMPI) ("DelMar" and the "Company"), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, reported that its abstract entitled, "Phase I/II study of Dianhydrogalactitol (VAL-083) In Patients With Recurrent Malignant Glioma," was accepted for poster presentation at the 20th Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology being held November 19 – 22, 2015, in San Antonio, Texas (Press release, DelMar Pharmaceuticals, NOV 9, 2015, View Source [SID:1234508154]).

The poster presentation will include an update on the Company’s fully enrolled 14-patient expansion cohort from its Phase II clinical study of VAL-083 (dianhydrogalactitol) in refractory glioblastoma multiforme (GBM). Patients enrolled in the GBM trial have failed both front-line therapy with temozolomide and second-line Avastin and, in most cases, one or more salvage therapies.

DelMar recently completed enrollment in the Phase II study and confirmed 40mg/m2 as the maximum tolerated dose (MTD) for advancement into registration-directed Phase II/III clinical trials in GBM.

The Company previously presented data from the Phase I dose-escalation portion of its multicenter Phase I/II clinical study with VAL-083 in patients with recurrent GBM in which dose limiting toxicity was observed at 50mg/m2/day, no drug-related severe adverse events were reported, and myelosuppression was mild at doses <40mg/m2/day.

Additionally, data on a sub-group analysis for patients receiving up to 5 mg/m2 daily x 3 every 21 days (low dose) versus those patients receiving 30mg/m2 or 40mg/m2 (therapeutic dose) of VAL-083 from the clinical trial were also previously presented. The sub-group analysis supports a dose-dependent and clinically meaningful survival benefit in GBM patients whose tumors had progressed following standard treatment with temozolomide, radiotherapy, bevacizumab and a range of salvage therapies.

DelMar’s abstract has been published online in the 2015 abstract supplement to the Society for Neuro-Oncology’s official journal, Neuro-Oncology, at View Source

About VAL-083

VAL-083 is a "first-in-class," small-molecule chemotherapeutic. In more than 40 Phase I and II clinical studies sponsored by the U.S. National Cancer Institute, VAL-083 demonstrated clinical activity against a range of cancers including lung, brain, cervical, ovarian tumors and leukemia both as a single-agent and in combination with other treatments. VAL-083 is approved in China for the treatment of chronic myelogenous leukemia (CML) and lung cancer, and has received orphan drug designation in Europe and the U.S. for the treatment of malignant gliomas.

DelMar has demonstrated that VAL-083’s anti-tumor activity is unaffected by the expression of MGMT, a DNA repair enzyme that is implicated in chemotherapy resistance and poor outcomes in GBM patients following standard front-line treatment with Temodar (temozolomide).

DelMar recently announced the completion of enrollment in a Phase II clinical trial of VAL-083 in refractory GBM. Patients have been enrolled at five clinical centers in the United States: Mayo Clinic (Rochester, MN); UCSF (San Francisco, CA) and three centers associated with the Sarah Cannon Cancer Research Institute (Nashville, TN, Sarasota, FL and Denver, CO).

In the Phase I dose-escalation portion of the study, VAL-083 was well tolerated at doses up to 40mg/m2 using a regimen of daily x 3 every 21 days. Adverse events were typically mild to moderate; no treatment-related serious adverse events reported at doses up to 40 mg/m2. Dose limiting toxicity (DLT) defined by thrombocytopenia (low platelet counts) was observed in two of six (33%) of patients at 50 mg/m2. Generally, DLT-related symptoms resolved rapidly and spontaneously without concomitant treatment, although one patient who presented with hemorrhoids received a platelet transfusion as a precautionary measure.

Sub-group analysis of data from the Phase I dose-escalation portion of the study suggested a dose-dependent and clinically meaningful survival benefit following treatment with VAL-083 in GBM patients whose tumors had progressed following standard treatment with temozolomide, radiotherapy, bevacizumab and a range of salvage therapies.

Patients in a low dose (≤5mg/m2) sub-group had a median survival of approximately five (5) months versus median survival of approximately nine (9) months for patients in the therapeutic dose (30mg/m2 & 40mg/m2) sub-group following initiation of VAL-083 treatment. DelMar reported increased survival at 6, 9 and 12 months following initiation of treatment with VAL-083 in the therapeutic dose sub-group compared to the low dose sub-group.

ChemoCentryx Reports Third Quarter 2015 Financial Results and Provides Corporate Update

On November 9, 2015 ChemoCentryx, Inc., (Nasdaq:CCXI), a clinical-stage biopharmaceutical company developing orally-administered therapeutics to treat autoimmune diseases, inflammatory disorders and cancer, reported financial results for the third quarter ended September 30, 2015 and provided an update on the Company’s corporate and clinical development activities (Press release, ChemoCentryx, NOV 9, 2015, View Source [SID:1234508151]).

"We continue to achieve important milestones according to plan in the development of the CCXI pipeline," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "Our novel drug candidates for orphan diseases, cancer, and renal pathologies are gaining increased visibility within the global medical community following presentations at high-profile medical meetings. We have several key milestones in the near-term, especially in our complement inhibitor CCX168 program, from which we expect to report top-line data from the ANCA Vasculitis Phase II CLEAR trial by the end of this year or in early January. We look forward to the CLEAR trial results which may provide promise for patients suffering from this devastating disease."

Recent Pipeline Developments Across Key Therapeutic Areas

Orphan and Rare Diseases: CCX168 is an orally-administered complement inhibitor targeting the 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 destroy different organ systems. AAV is the lead indication in the Company’s orphan and rare disease program.

By damaging the body’s small blood vessels, AAV affects many organ systems, mostly the kidneys, eyes, lungs, sinuses and nerves. This damage is caused by the destructive activity of inflammatory leukocytes in the body, with neutrophils considered to be the terminal effector cell. In AAV, neutrophils are attracted to sites of vascular destruction as well as activated at those sites by the activity of the complement system product known as C5a and its receptor, C5aR, which is the target of CCX168.

AAV affects approximately 40,000 people in the US (with approximately 4,000 new cases each year) and greater than 75,000 people in Europe, with at least 7,500 new cases each year, and is currently treated with courses of immuno-suppressants (cyclophosphamide or rituximab) combined with high dose steroid administration. Following initial treatment, up to 30 percent of patients relapse within 6 to 18 months, and approximately 50% of all patients will relapse within 3 to 5 years.

Current standard of care for AAV is associated with significant safety issues. First year mortality is approximately 11 to 18 percent. The single major cause of premature mortality is not disease related adverse events, but rather infection that is thought largely to be a consequence of steroid administration. Indeed, the multiple adverse effects of courses of steroid treatment (both initial courses and those that are repeated as a consequence of relapse) are major causes of both short-term and long-term disease and death. Such therapy related adverse events contribute significantly to patient care costs, as well as to the diminution of quality of life for patients.

The Company’s clinical development program in AAV aims to reduce or eliminate steroids in the standard of care regimen, essentially replacing high dose steroids with the complement inhibitor CCX168. The Phase II CLEAR clinical trial has been conducted in a three step fashion. The Company previously reported data from Steps 1 and 2 of the CLEAR Phase II trial, which provided evidence that steroids could be significantly reduced or even eliminated in AAV patients who were dosed with CCX168, with no diminished therapeutic efficacy compared to that seen with full dose steroid standard of care. Cumulative data from all three steps of the trial is expected in late December or early January.

In addition to AAV, the Company is also exploring the expansion of the use of the complement inhibitor CCX168 in other diseases, particularly in rare and orphan diseases.

Progress in the complement inhibitor CCX168 program includes:

All patients in the AAV CLEAR Phase II trial have completed treatment. Centralized Birmingham Vasculitis Activity Score (BVAS) assessments, which serves as the primary endpoint, and database finalizations are now underway;

Clinical study sites for the CLASSIC Phase II trial in AAV in North America continue to successfully enroll patients; and
Data showing a strong anti-thrombogenic effect of C5a receptor inhibition by CCX168 in aHUS patients’ sera was presented on November 6th at the American Society of Nephrology (ASN) Kidney Week 2015.

Immuno-Oncology: CCX872 is a potent and selective inhibitor of the chemokine receptor known as CCR2 which is being evaluated in patients with non-resectable pancreatic cancer. Pancreatic cancer is the fourth leading cause of cancer death in the US. Even with the most recent advances in the treatment of pancreatic cancer, median overall survival is 6-8 months. As such, more effective and better tolerated treatments are needed in both first-line and second-line therapies. In an ongoing, multi-center clinical trial with CCX872, up to 54 patients with non-resectable pancreatic cancer will be enrolled. The primary efficacy measurement of this study is progression-free survival after at least 24 weeks of treatment.

Enrollment in the Phase Ib clinical trial of CCX872 in patients with non-resectable pancreatic cancer continues to advance; patient enrollment has surpassed 20 percent of target;

At the 2015 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), human pharmacokinetic data from the oral dosing of CCX872 in non-resectable pancreatic patients shows very good CCR2 blockade in the first step of the trial, and supports expectation of greater than 90 percent receptor coverage will be achieved throughout the day in the ongoing trial; and

At the same meeting as noted above, as well as at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting, results from the combination therapy of chemokine receptor and check point inhibitors in triple negative breast cancer models were presented. These data suggest that a CCR1 inhibitor combined with an anti-PD-L1 antibody potentiates anti-tumor effects in triple negative breast cancer in vivo.

Chronic Kidney Disease: CCX140 is an inhibitor of the chemokine receptor known as CCR2 (distinct from CCX872 above) and is being developed as an orally-administered therapy for the treatment of diabetic nephropathy (DN), one of the most common forms of chronic kidney disease affecting greater than 10 million individuals in the US alone. The Company previously reported that a Phase II clinical trial in DN patients taking CCX140 achieved its primary endpoint of significantly lowering protein in the urine, an important marker of improved kidney function, over 52 weeks of therapy. CCR2 inhibition therapy represents a therapeutic approach entirely distinct from current standard of care treatment, which comprises common blood pressure medications that slow but do not halt the progression of disease.

Study results from the Phase II trial in patients with diabetic nephropathy treated with CCX140 were presented in an oral presentation at ASN Kidney Week 2015 in a session entitled, "Clinical Trials in CKD: Pursuing a New Horizon"and at the European Association for the Study of Diabetes (EASD) Annual Meeting.

Post treatment follow-up data indicate persistence of therapeutic effect after CCX140 was withdrawn, consistent with a disease modifying effect; and

Phase II clinical trial results of CCX140 in patients with diabetic nephropathy were published in the Lancet Diabetes & Endocrinology illustrating CCX140 to be safe and well tolerated while demonstrating statistically significant improvements in kidney function in patients with diabetic nephropathy.

Anticipated Milestones

Orphan and Rare Diseases:

Report top-line results from AAV CLEAR Phase II trial in Europe with CCX168 in late December or early January 2016;
Complete enrollment in the AAV CLASSIC Phase II trial in North America with CCX168 by the end of 2015, and report top-line data in the second quarter of 2016;
Prepare for End of Phase II meetings with regulatory agencies in 2016 to review CLEAR and CLASSIC Phase II clinical results; plan to initiate a Phase III clinical program with CCX168 in patients with AAV by the end of 2016; and
Report early results from the Phase II proof-of-concept clinical trial of CCX168 in aHUS patients who are on dialysis in the first half of 2016.

Immuno-Oncology:

Advance the Phase Ib pancreatic cancer trial of CCX872 in combination with FOLFIRINOX, and report early overall response data in the first half of 2016 and initial progression free survival in the second half of 2016.

Chronic Kidney Disease:

Continue preparations for an End of Phase II meeting with the FDA including the design of the Phase III clinical development program for CCX140 in diabetic nephropathy, as well as continued business development efforts.

Third Quarter 2015 Financial Results

Research and development expenses were $7.9 million for the three months ended September 30, 2015 compared to $7.5 million reported for the same period in 2014. The increase from 2014 to 2015 was primarily attributable to higher expenses associated with CCX168, our C5aR inhibitor, due to ongoing Phase II clinical trials for the treatment of AAV in Europe, the CLEAR trial, and in North America, the CLASSIC trial, and our Phase II pilot clinical trials in patients with aHUS and IgAN. Further, costs associated with CCX140, our CCR2 inhibitor, in preparation to conduct an end-of-Phase II meeting with the FDA and the ongoing clinical trial with CCX872, our second CCR2 inhibitor, in patients with pancreatic cancer also contributed to the increases. These increases were partially offset by lower expenses associated with CCX507, our second generation CCR9 inhibitor, due to the completion of Phase I clinical development in the third quarter of 2014.

General and administrative expenses were $3.8 million for the three months ended September 30, 2015 compared to $3.5 million for the comparable period in 2014. The increase from 2014 to 2015 was primarily due to increases in intellectual property related expenses, travel expenses and professional service fees, partially offset by lower employment related expenses and legal fees.

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

Cash, cash equivalents and investments totaled $85.2 million at September 30, 2015.

Asterias Biotherapeutics Reports Third Quarter Results

On November 9, 2015 Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company focused on the emerging field of regenerative medicine, reported financial and operating results for the third quarter ended September 30, 2015 (Press release, BioTime, NOV 9, 2015, View Source;p=RssLanding&cat=news&id=2110632 [SID:1234508149]).

"Asterias continued to achieve solid progress in the third quarter toward advancing the clinical development of our key therapeutic programs," stated Pedro Lichtinger, President and CEO of Asterias. "We completed recruitment for the initial, low-dose safety cohort of the AST-OPC1 Phase 1/2a clinical trial for spinal cord injury. To date, no serious adverse events have been observed in any of the three treated patients. Following recommendation by the Data Monitoring Committee, we started patient enrollment for the second cohort in which patients will be treated with a higher dose of AST-OPC1 that is in the range that we believe to be efficacious and that has the potential to bring benefit to patients. Importantly, the first patient in the initial, low-dose safety cohort has shown steady neurological improvement at both 2-month and 3-month post-injection assessments. Also during the quarter, we announced a strategic collaboration to advance development of large scale manufacturing processes for AST-VAC2, our allogeneic dendritic cell immunotherapy. This manufacturing agreement will streamline and scale manufacturing processes for AST-VAC2 to support advanced clinical trials, including the planned Phase 1/2a clinical trial in lung cancer, and eventual commercialization of AST-VAC2. We anticipate achieving significant additional clinical milestones during the next 12 months."

Recent Corporate Developments

On November 2, 2015, Asterias announced the appointment of Georgia Erbez as Chief Financial Officer. Ms. Erbez brings to Asterias an impressive track record of achievement and a broad range of financial management and strategic planning experience, especially in the areas of capital resource development and business development.
Research and Development Highlights

AST-VAC1 (antigen-presenting autologous dendritic cells)

Following the presentation of promising Phase 2 results at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting during the second quarter, the Company has continued to advance its planning for further clinical development of AST-VAC1, including holding a clinical advisory panel to design a pivotal trial, and initiating the selection process for a contract manufacturing organization for that trial.

AST-OPC1 (oligodendrocyte progenitor cells)

In August, Asterias concluded recruitment of the initial safety cohort of the SCiStar Phase 1/2a dose-escalation clinical trial of AST-OPC1 (oligodendrocyte progenitor cells) for complete cervical spinal cord injury (SCI), in which three patients were administered a low dose of 2 million AST-OPC1 cells. The results of the study continue to support a robust safety profile for AST-OPC1, with no serious adverse events observed in any of the three treated patients to date.

In October, following review of the 30-day post-injection safety data from the initial safety cohort, the Data Monitoring Committee recommended dose escalation to the second cohort in the SCiStar Phase 1/2a clinical trial. Recruitment for the second cohort has commenced, with a planned enrollment of five patients who will each receive 10 million cells of AST-OPC1. Asterias expects to complete enrollment of the 10 million cell cohort, and to receive safety data, in the first half of 2016. Initial efficacy data readouts from the trial are expected in the second half of 2016. The first patient in the safety cohort, at the 2-month post-injection assessment, had progressed from a complete ASIA Impairment Scale (AIS) A injury to an incomplete AIS B injury. At 3-months post-injection, this patient demonstrated additional neurological improvement to an AIS C injury.

Asterias announced the publication of preclinical data in Regenerative Medicine that supports the safety and use of AST-OPC1 as a treatment for SCI. The preclinical results showed that AST-OPC1 cells did not cause any adverse clinical observations, toxicities, allodynia or tumors. AST-OPC1 exhibited robust persistence and limited migration within the thoracic and cervical spinal cord. In addition, AST-OPC1 demonstrated nerve growth stimulating properties and remyelinating properties that supported restoration of function in animal models.

In the third 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 announced a collaboration with the UK-based Cell Therapy Catapult to advance development of large scale manufacturing processes for AST-VAC2, Asterias’ allogeneic dendritic cell immunotherapy. Under the agreement, the Cell Therapy Catapult will streamline and scale manufacturing processes for AST-VAC2 to support advanced clinical trials and eventual commercialization of AST-VAC2. Asterias has an ongoing partnership with Cancer Research UK to execute the first stage of AST-VAC2 clinical development, under which Cancer Research UK will sponsor and manage a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung carcinoma.

Third Quarter [Unaudited] Financial Results

Total revenues in the third quarter 2015 were $1.4 million, which were primarily comprised of grant income and royalty revenues on product sales by licensees. Total revenues in the comparable quarter in the prior year were $85,000. Operating expenses in the third quarter were $6.2 million, compared to $4.0 million in the prior year period. Research and development (R&D) expenses in the third quarter were $4.6 million, compared to $2.6 million in the comparable quarter of the prior year. General and administrative (G&A) expenses in the third quarter were $1.6 million, compared to $1.5 million in the comparable quarter of the prior year.

Net loss for the third quarter 2015 was $3.5 million, including a deferred income tax benefit of approximately $1.6 million. Net loss in the third quarter 2014 was $1.7 million, including a deferred income tax benefit of approximately $2.3 million. On a per share basis, net loss for the third quarter was $0.09 per share, compared to a loss of $0.05 per share for the comparable quarter of the prior year.

Cash and cash equivalents were $12.6 million as of September 30, 2015, compared to $3.1 million as of December 31, 2014. At September 30, 2015, the Company held 3.9 million BioTime common shares, with a market value of approximately $11.6 million on that date.

For the third quarter, net cash used in operating activities was $3.6 million. The Company continues to expect net cash burn in 2015 to be in the range of $15 million to $17 million.

Bellicum Pharmaceuticals Reports Third Quarter 2015 Financial Results and Recent Program Updates

On November 9, 2015 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the third quarter of 2015 and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, NOV 9, 2015, View Source;p=RssLanding&cat=news&id=2110660 [SID:1234508146]).

"During the third quarter we continued to make good progress in the advancement of our stem cell transplant, CAR-T and TCR programs," said Tom Farrell, President and Chief Executive Officer of Bellicum. "We are particularly excited about the interim data that will be presented at ASH (Free ASH Whitepaper) 2015 that underscore the potential of our lead product candidate BPX-501 to improve outcomes for patients with blood cancers and inherited blood diseases undergoing haplo transplantation. BPX-501 could be used in the treatment of over 60 rare diseases, enabling a potential curative transplant for many of those disorders, including sickle cell disease, where treatment-related mortality has severely limited the adoption of allogeneic transplantation."

Added Mr. Farrell, "We are also looking forward to multiple presentations at ASH (Free ASH Whitepaper) highlighting our CAR-T and TCR adoptive cell therapy programs and the power of our cellular control technologies. We continue to anticipate that three new product candidates, BPX-601, BPX-701, and BPX-401 will be initiating clinical development in the first half of 2016."

Program Updates:

BPX-501 at ASH (Free ASH Whitepaper) 2015: Company to present interim data from the BP-004 ongoing Phase 1/2 clinical trial. Bellicum will present initial data in malignant and non-malignant blood diseases at the upcoming 26th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in early December in Orlando, Florida. The open label dose escalation trial in pediatric patients is evaluating whether BPX-501 T cells from a haploidentical donor, administered following a T-depleted hematopoietic stem cell transplant (HSCT), are safe and can enhance immune reconstitution.

Enrollment in the BP-004 trial continues at a strong pace at sites in Europe and in the U.S., with 53 pediatric patients enrolled as of October 31st, including one patient with sickle cell disease.

Among non-malignant patients in the trial treated to date are four children with beta thalassemia major (in its most severe form, the β0/β0 type) who have successfully undergone the HSCT transplant procedure with the add-back of BPX-501 gene-modified T cells. Within two weeks of the transplant procedure all patients became blood transfusion-independent. All four patients are alive, disease-free and remain transfusion-independent.

At a medical symposium in Parma, Italy in early September, principal investigator Dr. Franco Locatelli shared initial outcomes from this ongoing trial. His presentation included the first 15 children enrolled in the clinical study with non-malignant inherited disorders (four with severe combined immunodeficiency, or SCID; three with Wiskott-Aldrich Syndrome; four with Fanconi anemia; three with beta thalassemia, and one with hemophagocytic lymphohistiocytosis) who received the BPX-501 T cell add-back following HLA-partially matched family donor HSCT. In all cases, the BPX-501 T cells engrafted and expanded with no secondary graft failures. Grade II skin-only graft versus host disease (GvHD) was observed in one patient, and promptly resolved with topical steroids. None of the patients so far have developed chronic GvHD, and all are alive and disease-free.

BPX-601 preclinical data to be presented at ASH (Free ASH Whitepaper) 2015. Bellicum continues to advance its first GoCAR-T product candidate, containing our proprietary iMC (inducible MyD88/CD40) activation switch, designed to treat solid tumors expressing prostate stem cell antigen (PSCA). The Company expects to file an IND for the initial indication of pancreatic cancer by the end of 2015. In addition to pancreatic cancer, PSCA is also expressed in prostate, ovarian, bladder, esophageal and gastric cancers. BPX-601 is differentiated from traditional CAR-T therapies with an MC co-stimulatory domain that is activated by administration of rimiducid.
BPX-401 CIDeCAR preclinical data to be highlighted in an oral presentation at ASH (Free ASH Whitepaper) 2015. BPX-401, a CIDeCAR product candidate incorporating Bellicum’s proprietary MC co-stimulatory domain and the CaspaCIDe safety switch, is designed to target blood cancers expressing CD19. BPX-401 is expected to enter the clinic in the first half of 2016.

BPX-701 progressing toward the clinic. Bellicum continues to advance its proprietary T cell receptor (TCR) product candidate designed to target solid tumors expressing the preferentially-expressed antigen in melanoma, or PRAME. The Company has identified clinical sites for its BPX-701 CaspaCIDe-enabled TCR product candidate and expects to file an IND by the end of 2015, initially for the indications of PRAME-expressing sarcomas and uveal melanoma.

Third Quarter and Nine Months Ended September 30, 2015 Financial Results:

Bellicum reported a net loss of $13.4 million for the third quarter of 2015 and $31.7 million for the nine months ended September 30, 2015, compared to a net loss of $4.1 million and $9.7 million for the comparable periods in 2014. The results included non-cash, share-based compensation charges of $2.3 million and $5.9 million for the third quarter and nine months ended September 30, 2015, respectively, and $0.1 million and $0.2 million for the comparable periods in 2014. As of September 30, 2015, cash and investments totaled $163.2 million.

Grant revenues were $0.1 million and $0.2 million for the three and nine months ended September 30, 2015, respectively, and $0.7 million and $1.8 million during the comparable periods in 2014. The decrease in grant revenues was primarily due to the June 2014 expiration of the Company’s grant award from the Cancer Prevention and Research Institute of Texas.

Research and development expenses were $9.8 million and $23.5 million, respectively, for the three and nine months ended September 30, 2015, compared to $2.3 million and $7.9 million during the comparable periods in 2014. The higher expenses in the 2015 periods were primarily due to an increase in manufacturing and clinical expenses as a result of increased patient enrollment in our BPX-501 clinical trials, increased expenses for the IND-enabling activities on the Company’s CAR-T and TCR product candidates and increased personnel and infrastructure costs.

General and administrative expenses were $3.9 million and $8.9 million, respectively, for the three and nine months ended September 30, 2015, compared to $1.3 million and $2.3 million during the comparable periods in 2014. The increased G&A expenses in 2015 were due to the growth of the organization and the costs associated with operating as a public company.