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10-Q – Quarterly report [Sections 13 or 15(d)]

(Filing, 10-Q, Heat Biologics, AUG 13, 2015, View Source [SID:1234507240])

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OncoGenex Pharmaceuticals, Inc. Reports Financial Results for Second Quarter 2015

On August 13, 2015 OncoGenex Pharmaceuticals, Inc. (NASDAQ: OGXI) reported second quarter 2015 financial results (Press release, OncoGenex Pharmaceuticals, AUG 13, 2015, View Source [SID:1234507254]).

Recent Developments and Anticipated Near-term Milestones

Custirsen – Phase 3 Lung and Prostate Cancer Trials

On July 13, 2015, the company announced that its Phase 3 ENSPIRIT trial evaluating custirsen for the treatment of advanced or metastatic non-small cell lung cancer (NSCLC) is continuing as planned per the recommendation of an Independent Data Monitoring Committee (IDMC). This decision was based upon completion of the second and final planned interim futility analysis that included a more rigorous evaluation for determining futility in achieving a survival benefit associated with custirsen in NSCLC. Based on current enrollment projections, the company believes final survival results could be available in the second half of 2016.

On June 10, 2015, OncoGenex announced that the U.S. Food and Drug Administration (FDA) has agreed to the company’s proposed amendment to the Phase 3 AFFINITY protocol and statistical analysis plan. The proposed amendment includes the addition of a co-primary survival objective designed to prospectively evaluate the survival benefit of custirsen in men who are at increased risk for poor outcomes when treated with cabazitaxel for metastatic castrate-resistant prostate cancer (CRPC). Patients at risk for poor outcomes will be identified as having two or more of five common risk factors. In addition, OncoGenex and the FDA agreed that an interim analysis will occur for the entire study population when the final analysis for the poor prognosis subpopulation occurs. Advice from the European Medicines Agency through its Scientific Review process will be completed prior to finalizing the protocol amendment. Subject to finalizing the pending protocol amendment, timing for the final analysis of the poor prognosis subpopulation is projected to occur by the end of 2015, while the final analysis for the entire study population is projected to occur in the second half of 2016.

On May 30, 2015, the company announced that results from a retrospective analysis of the Phase 3 SYNERGY trial showed a benefit with custirsen therapy in men with metastatic CRPC who were at risk for poor outcomes. The analysis, exploring the effect of clusterin inhibition in men at risk for poor outcomes, showed that over 40% of men in the trial had at least two of five common risk factors for poor prognosis. In these men, the analysis found a 27% lower risk of death when custirsen was used in combination with first-line docetaxel compared to docetaxel alone. These results were presented at the 51st Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago.

Apatorsen – Phase 2 Bladder, Lung, Pancreatic and Prostate Cancer Trials

Investigators from the Phase 2 Borealis-1 trial presented results from an exploratory analysis that showed metastatic bladder cancer patients with poor prognostic features (KPS, liver involvement, low hemoglobin and high alkaline phosphatase) benefited from apatorsen 600mg added to first-line chemotherapy (OS HR = 0.72) compared to chemotherapy alone. Patients in the trial with a Karnofsky Performance Status (KPS) of 80% or less, a common indicator of poor prognosis, experienced a 50% reduction in risk of death with the addition of apatorsen therapy (OS HR = 0.50). These results were presented in an oral session on June 1, 2015 at the 51st Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago.

"This is an exciting time for the company with multiple anticipated upcoming clinical milestones through 2015 and into 2016, including a Phase 3 data readout expected by the end of the year," said Scott Cormack, President and CEO of OncoGenex. "Our two priority assets – custirsen and apatorsen – continue to demonstrate their potential value to provide clinical benefit in the most vulnerable patients – those at increased risk for poor outcomes and/or more resistant disease."

Financial Update and Results

Revenue for the three and six months ended June 30, 2015 decreased to $4.0 million and $5.4 million, respectively, from $4.9 million and $16.7 million for the three and six months ended June 30, 2014, respectively.

Total operating expenses for the three and six months ended June 30, 2015 were $9.6 million and $16.0 million, respectively, compared to $12.6 million and $32.2 million for the three and six months ended June 30, 2014, respectively.

Net loss for the three and six months ended June 30, 2015 was $6.0 million, or $0.26 per dilluted common share, and $10.5 million, or $0.46 per dilluted common share, respectively, compared with $7.0 million, or $0.47 per diluted common share, and $15.7 million, or $1.05 per dilluted common share, respectively, for the three and six months ended June 30, 2014.

As of June 30, 2015, cash, cash equivalents and short-term investments increased to $60.2 million from $47.1 million as of December 31, 2014.

Subsequent to June 30, 2015, the company raised $14.7 million from the sale of common stock to Lincoln Park Capital, LLC under the terms of the share purchase agreement. As of August 13, 2015, no further amounts remained available for sale under this offering program.

Based on current expectations, the company believes that these resources, in addition to the amounts received from the sale of common stock to Lincoln Park Capital, LLC in the third quarter of 2015 will be sufficient to fund its currently planned operations late into the fourth quarter of 2016, which may include:

announcement of final results of the poor prognosis subpopulation in the Phase 3 AFFINITY prostate cancer trial by the end of 2015 and final analysis for the entire study population in the second half of 2016, depending on timing of the event-driven final analysis and subject to completion and submission of the proposed protocol amendment;

announcement of final survival results in the Phase 3 ENSPIRIT lung cancer trial expected in the second half of 2016;

completion of enrollment in the Phase 2 Borealis-2 bladder cancer trial expected to occur in the third quarter of 2015;

announcement of the Phase 2 Rainier pancreatic cancer trial results expected by the end of 2015;

announcement of the Phase 2 Spruce lung cancer trial results expected in the first half of 2016;

announcement of the Phase 2 Pacific prostate cancer trial preliminary results expected in 2016; and
completion of enrollment in the Phase 2 Cedar lung cancer trial expected in 2016.
As of August 13, 2015, OncoGenex had 29,791,776 shares outstanding.

NanoString Technologies Receives Favorable Final Local Coverage Determination by Palmetto GBA for Its Prosigna Breast Cancer Assay

On August 13, 2015 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported that Palmetto GBA, a Medicare Administrative Contractor (MAC) that assesses molecular diagnostic technologies through its MolDx program, has issued a favorable final local coverage determination (LCD) for the Prosigna Breast Cancer Assay (Press release, NanoString Technologies, AUG 13, 2015, View Source [SID:1234507253]).

"We are delighted by the favorable LCD published today by Palmetto’s MolDx team. The policy decision is expected to increase patient access across Prosigna’s entire intended use population, including patients with both node-negative and node-positive breast cancer," said Brad Gray, President and Chief Executive Officer of NanoString Technologies. "The MolDx process is viewed as one of the most sophisticated and influential technology assessments in the US, and the positive MolDx policy demonstrates the effectiveness and impact of our reimbursement team."

The final LCD, which confirms the coverage policy for Medicare beneficiaries, includes reimbursement coverage for postmenopausal patients with ER+, lymph node-negative, stage I or II breast cancer; and ER+, lymph node-positive (1-3 positive nodes), stage II breast cancer. The draft LCD is posted to the Medicare Coverage Database on the Centers for Medicare & Medicaid Services (CMS) website at: View Source;ContrId=374&ver=5&ContrVer=1&Date=11%2f01%2f2015&DocID=L36125&bc=iAAAAAgAAAAAAA%3d%3d&

This decision is consistent with the draft guidance issued by Palmetto GBA on May 14. Following a public comment period that ended on July 24, Palmetto GBA finalized and published the guidance, which becomes effective for services on or after October 1, 2015. The final LCD applies to the J11 jurisdiction, comprising North Carolina, South Carolina, Virginia and West Virginia. Other Medicare jurisdictions participating in the MolDx program may choose to adopt the same coverage policy in the future.

About the Prosigna Breast Cancer Prognostic Gene Signature Assay and nCounter Dx Analysis System

The Prosigna Assay provides a risk category and numerical score for assessment of the risk of distant recurrence of disease at 10 years in postmenopausal women with node-negative (Stage I or II) or node-positive (Stage II), hormone receptor-positive (HR+) breast cancer. Based on the PAM50 gene signature initially discovered by Charles Perou, Ph.D. and colleagues, the Prosigna Assay is an in vitro diagnostic tool that utilizes gene expression data weighted together with clinical variables to generate a risk category and numerical score to assess a patient’s risk of distant recurrence of disease. The Prosigna Assay measures gene expression levels of RNA extracted from formalin-fixed paraffin embedded (FFPE) breast tumor tissue previously diagnosed as invasive breast carcinoma.

The Prosigna Assay requires minimal hands-on time and runs on NanoString’s proprietary nCounter Dx Analysis System, which offers a reproducible and cost-effective way to profile many genes simultaneously with high sensitivity and precision.

The nCounter Dx Analysis System is a highly automated and easy-to-use platform that utilizes a novel digital barcoding chemistry to deliver high precision multiplexed assays. The system is available in the multi-mode FLEX configuration, which is designed to meet the needs of high-complexity clinical laboratories seeking a single platform with the flexibility to run the Prosigna Breast Cancer Assay and, when operated in the "Life Sciences" mode, process translational research experiments and multiplexed assays developed by the laboratory.

In the United States, the Prosigna Assay is available for diagnostic use when ordered by a physician. The Prosigna Assay has been CE-marked and is available for use by healthcare professionals in the European Union and other countries that recognize the CE Mark, as well as Canada, Israel, Australia, New Zealand and Hong Kong.

In the U.S., the Prosigna Assay is indicated in female breast cancer patients who have undergone surgery in conjunction with locoregional treatment consistent with standard of care, either as:

(1) a prognostic indicator for distant recurrence-free survival at 10 years in postmenopausal women with Hormone Receptor-Positive (HR+), lymph node-negative, Stage I or II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors or (2) a prognostic indicator for distant recurrence-free survival at 10 years in postmenopausal women with Hormone Receptor-Positive (HR+), lymph node-positive (1-3 nodes), Stage II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors. The device is not intended for patients with four or more positive nodes.

Sophiris Bio Reports Second Quarter Financial Results

On August 13, 2015 Sophiris Bio Inc. (NASDAQ: SPHS) (the "Company" or "Sophiris"), a biopharmaceutical company developing PRX302 (topsalysin) for the treatment of urological diseases, reported financial results for the three and six months ended June 30, 2015 (Press release, Sophiris Bio, AUG 13, 2015, View Source [SID:1234507247]).

"In May we met our goal of dosing our first patients in a Phase 2a proof of concept trial of PRX302 in patients with localized low to intermediate risk prostate cancer," stated Randall Woods, president and CEO of Sophiris Bio. "With the capital available, we are positioned to achieve clinical milestones from our ongoing trials in BPH and localized prostate cancer and look forward to reporting on these programs over the next several quarters."

Financial Results

At June 30, 2015, we had cash, cash equivalents and securities available-for-sale of $13.2 million and net working capital of $11.6 million. We expect that our cash, cash equivalents and securities available-for-sale as of June 30, 2015 will be sufficient to fund our operations through the end of April 2016 assuming that we do not initiate any additional clinical development of PRX302. We will need to find additional capital to fund a second Phase 3 clinical trial of PRX302 for the treatment of the symptoms of BPH and for any future clinical development of PRX302 for the treatment of localized prostate cancer beyond our ongoing Phase 2a proof of concept clinical trial.

For the three months ended June 30, 2015

The Company reported a net loss of $3.7 million ($0.22 per share) for the three months ended June 30, 2015 compared to a net loss of $8.8 million ($0.53 per share) for the three months ended June 30, 2014.

Research and development expenses

Research and development expenses were $2.6 million for the three months ended June 30, 2015 compared to $7.1 million for the three months ended June 30, 2014. The decrease in research and development costs are attributable to a decrease in the costs associated with the Company’s Phase 3 PLUS-1 clinical trial of PRX302 and costs associated with the manufacturing activities for PRX302. This decrease is offset by an increase in costs associated with the Company’s Phase 2a proof of concept trial for localized low to intermediate risk prostate cancer.

General and administrative expenses

General and administrative expenses were $1.0 million for the three months ended June 30, 2015 compared to $1.5 million for the three months ended June 30, 2014. The decrease is primarily due to a decrease in non-cash stock-based compensation expense and, to a lesser extent, a decrease in legal, consulting and personnel related costs.

For the six months ended June 30, 2015

The Company reported a net loss of $8.0 million ($0.48 per share) for the six months ended June 30, 2015 compared to a net loss of $17.2 million ($1.05 per share) for the six months ended June 30, 2014.

Research and development expenses

Research and development expenses were $5.6 million for the six months ended June 30, 2015 compared to $13.9 million for the six months ended June 30, 2014. The decrease in research and development costs are attributable to a decrease in the costs associated with the Company’s Phase 3 PLUS-1 clinical trial of PRX302 and costs associated with the manufacturing activities for PRX302. This decrease is offset by an increase in costs associated with the Company’s Phase 2a proof of concept trial for localized low to intermediate risk prostate cancer.

General and administrative expenses

General and administrative expenses were $2.0 million for the six months ended June 30, 2015 compared to $2.9 million for the six months ended June 30, 2014. The decrease is primarily due to a decrease in non-cash stock-based compensation expense and, to a lesser extent, a decrease in legal, consulting and personnel related costs.

8-K – Current report

On August 13, 2015 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies, reported financial results for the second quarter of 2015, and provided an update on the Company’s recent developments (Filing, 8-K, Bellicum Pharmaceuticals, AUG 13, 2015, View Source [SID:1234507245]).

"Over the last six months, we made substantial progress across all of our controlled immunotherapy programs," said Tom Farrell, President and CEO of Bellicum Pharmaceuticals. "In our lead BPX-501 clinical program, we have been pleased with the strong pace of patient recruitment into our BP-004 study. This study is evaluating pediatric patients with orphan genetic diseases or hematological cancers who undergo a haploidentical allogeneic hematopoietic stem cell transplant to attain a disease cure. We are assessing safety and the recovery of the immune system, and remain on track to present initial top-line results from approximately 40 patients in December of this year. BPX-501 is designed to address the clear medical need for a safer, more effective transplant option for patients who do not have a matched donor."

Continued Mr. Farrell, "We also advanced our pipeline of CAR-T and TCR products and expect to file two INDs by the end of 2015, for BPX-701, our TCR product candidate for solid tumors, and BPX-601, our CAR-T product candidate for solid tumors overexpressing prostate stem cell antigen, or PSCA. We believe the broad utility of our proprietary cellular control technologies and their potential to provide greater levels of efficacy and safety will become increasingly important as the field evolves toward the treatment of solid tumors and additional antigen targets. We also continued to strengthen our leadership team and have identified and leased space for the build-out of facilities to enable in-house cell therapy manufacturing to supply clinical trials."
Program Updates:

• BPX-501 on track for year-end read-out of initial data: The Company continued to enroll its Phase 1/2 clinical trials with BPX-501 in the allogeneic hematopoietic stem cell transplant (HSCT) setting. In the BP-004 trial, additional leading U.S. and European pediatric transplant centers have been initiated and the protocol has been amended to increase the study size to allow up to 90 patients. The open label dose escalation trial is evaluating whether BPX-501 T cells from a haploidentical donor, typically the child’s mother or father, administered following a T-depleted HSCT, are safe and can enhance immune reconstitution. To date, about half of the patients randomized into the trial have non-malignant inherited diseases, such as severe combined immunodeficiency (SCID), Wiskott-Aldrich Syndrome and beta thalassemia, all chronic life-long disorders for which HSCT is curative. The BPX-501 T cells contain the CaspaCIDe safety switch for use in the event of GvHD, allowing the add-back of T cells in order to promote faster immune recovery and anti-viral activity after a T-depleted transplant procedure. The Company expects to report initial top-line data from ongoing clinical trials in the HCST setting in December 2015.

In July 2015, the intellectual property for BPX-501 was strengthened with a U.S. method of use patent issued to Baylor College of Medicine. The patent, licensed exclusively to Bellicum, is scheduled to expire in 2031.

• DOTTI trial data published in BLOOD highlight safety, effectiveness of CaspaCIDe-enabled T cell add-back: Clinical results from an investigator-initiated 12-patient trial demonstrated that infusing haploidentical donor T cells containing the CaspaCIDe safety switch, following a T-depleted haplo-HSCT, led to improved immune reconstitution and infection control. The trial data also showed that when GvHD occurs, it can be rapidly controlled and eliminated by removing alloreactive cells in vivo, and that the productive, anti-viral and anti-cancer cells remain, repopulate and maintain immunity. The trial results were simultaneously published in BLOOD and featured as a late-breaking oral presentation at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) annual meeting in May 2015.

• BPX-601 is progressing ahead of schedule: The Company’s first GoCAR-T product candidate, containing the proprietary inducible MC (MyD88/CD40) activation switch, designed to treat solid tumors overexpressing prostate stem cell antigen, or PSCA, is progressing ahead of schedule and we now expect to file an IND for the initial indication of pancreatic cancer by the end of 2015. Previous guidance was for initiation of clinical trials in the second half of 2016. In addition to pancreatic cancer, PSCA is also expressed in prostate, ovarian, bladder, esophageal and gastric cancers.

• BPX-701 moves toward clinic: Bellicum continued to advance development of its next-generation, proprietary T cell receptor (TCR) product candidate designed to target solid tumors expressing the preferentially-expressed antigen in melanoma, or PRAME. The Company licensed the technology to develop, manufacture and commercialize high-affinity TCR product candidates against PRAME and an additional target from Leiden University Medical Center in April. Bellicum has identified clinical sites for its BPX-701 CaspaCIDe-enabled TCR product candidate and expects to file an IND by the end of 2015. The Company is planning to initially develop this product candidate for the indications of PRAME-expressing sarcomas and uveal melanoma.

• BPX-401 progressing on track: BPX-401, a CIDeCAR product candidate constructed with Bellicum’s proprietary MC co-stimulatory domain and the CaspaCIDe safety switch, is designed to target blood cancers expressing CD19, including potential indications of acute lymphocytic leukemia, chronic lymphocytic leukemia, and certain non-Hodgkin’s lymphomas. BPX-401 is expected to enter the clinic in the first half of 2016.

• American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO) (Free ASCO Whitepaper) data highlights potent anti-tumor effects of MC co-stimulatory domain: Bellicum’s poster presentation at ASCO (Free ASCO Whitepaper) highlighted results from two preclinical studies that evaluated the in vivo tumor-killing abilities of its CIDeCAR cells. The studies showed that MC co-stimulation increased T cell proliferation and enhanced efficacy in lymphoma and solid tumor models in vivo compared to CAR T cells that included the more commonly utilized co-stimulatory molecule CD28. Bellicum’s CD19-targeted CIDeCAR (BPX-401) elicited dose-dependent elevation of cytokines, analogous to cytokine release syndrome, but cytokine levels were rapidly normalized upon administration of rimiducid, safely and without loss of tumor control.

• BPX-201 program to discontinue after current study concludes: Based on the prioritization of our other pipeline opportunities, the Company no longer intends to progress its BPX-201 vaccine into additional studies after the conclusion of the ongoing Phase 1 trial.

Corporate Updates:

• Expansion of facilities to bring manufacturing of U.S. clinical supply in-house: Bellicum leased an additional 27,000 square feet at its corporate headquarters for the manufacture of BPX-501 for clinical studies and to support the development of its expanding pipeline of TCR and CAR-T adoptive cell therapy product candidates.

• Strengthened board of directors and management team: In July 2015, Bellicum added Stephen R. Davis, currently the interim Chief Executive Officer of ACADIA Pharmaceuticals, to its Board of Directors. The Company has also recruited three experienced executives to its management team: Scott Cullison joined as Vice President, Commercial Planning and Program Management, Anne Frese as Vice President, Human Resources, and Steve Toler as Vice President, Pharmaceutical Development.

Second Quarter and Six Months Ended June 30, 2015 Financial Results:

Bellicum reported a net loss of $10.5 million for the second quarter of 2015 and $18.3 million for the six months ended June 30, 2015, compared to a net loss of $3.3 million and $5.6 million for the comparable periods in 2014. The results included non-cash, stock-based compensation charges of $2.1 million and $3.6 million for the second quarter and six months ended June 30, 2015 and $0.1 million and $0.2 million for the comparable periods in 2014. As of June 30, 2015, cash and investments totaled $172.6 million.
Grant revenues were $0.1 million and $0.2 million for the three and six months ended June 30, 2015, respectively, and $0.6 million and $1.1 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 in the second quarter of 2015 were $8.0 million and $13.7 million, respectively, for the three and six months ended June 30, 2015, compared to $3.2 million and $5.6 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 in the second quarter of 2015 were $2.8 million and $5.0 million, respectively, for the three and six months ended June 30, 2015, compared to $0.6 million and $1.0 million during the comparable periods in 2014. The increased G&A expenses were due to the growth of the organization and the costs associated with operating as a public company.