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]).

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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.

(Filing, 10-Q, OncoGenex Pharmaceuticals, AUG 13, 2015, View Source [SID:1234507255])

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(Filing, 10-Q, Sophiris Bio, AUG 13, 2015, View Source [SID:1234507257])

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On August 13, 2015 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported a corporate update and reported financial results for the three and six months ended June 30, 2015 (Press release, Onconova, AUG 13, 2015, View Source [SID:1234507238]).

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"Following receipt of regulatory guidance, we have completed the design of a proposed Phase 3 pivotal trial for IV rigosertib in patients with higher-risk myelodysplastic syndrome (HR-MDS) after failure of hypomethylating agent (HMA) therapy. We are finalizing our IND submission for the trial and intend to initiate enrollment in this study in the second half of 2015," said Ramesh Kumar, Ph.D., President and CEO of Onconova. "In addition, a Phase 2 trial of oral rigosertib in combination with azacitidine in MDS and AML is approaching full enrollment. We expect to present data from this study later this year."

Recent Business Highlights:

Development of Rigosertib IV in Higher-Risk MDS (HR-MDS)

A randomized controlled Phase 3 trial, referred to as 04-30, has been designed following discussions with FDA and EMA, input from key opinion leaders and incorporating learnings from the ONTIME study. This trial would enroll patients under 80 years of age who had progressed on or failed to respond to previous treatment with HMAs within the first nine months of HMA treatment and had HMA therapy discontinued within six months prior to enrollment in the 04-30 trial. The primary endpoint of this study is overall survival, and an interim analysis is anticipated. An IND and CTA are in preparation and are expected to be submitted this quarter. We expect the Phase 3 clinical trial to be conducted at approximately 100 sites in more than ten countries. We expect to begin enrolling patients in this trial later this year, though our ability to conduct the trial as planned will require us to obtain additional financing.

Development of Oral Rigosertib in Combination with Azacitidine for MDS and AML Patients

Our clinical trial of oral rigosertib in combination with azacitidine for the treatment of front-line and second-line HR-MDS and AML, referred to as 09-08, is based on previously published preclinical data demonstrating synergistic activity of this combination. Updated Phase 1 results and translational studies supporting the therapeutic rationale for the rigosertib/azacitidine combination were the subject of two presentations at the 13th International Symposium on Myelodysplastic Syndromes in the second quarter of 2015. These data demonstrated the tolerability and activity of the combination therapy in MDS and AML patients, including patients who had previously been treated with an HMA.

The Phase 2 portion of the 09-08 trial is designed to assess whether treatment with rigosertib in combination with azacitidine reduces the number of bone marrow blasts, improves peripheral blood counts and delays signs of disease progression in patients with MDS and AML. Thus far, 33 patients, including 28 MDS patients, have received the recommended Phase 2 dose in this study. Phase 2 data are expected to be presented at a scientific conference later this year.
Development of Oral Rigosertib in Lower-Risk MDS (LR-MDS)

Enrollment in an extension of a Phase 2 trial of oral rigosertib, referred to as 09-05, to assess the utility of DNA methylation patterns for the identification of LR-MDS patients likely to respond to treatment, is now complete. Onconova is collaborating with a methylation genomics company to refine this test and expects to announce results this year.
Upcoming Events

Filing of IND and CTA relating to Phase 3 trial 04-30: 3Q2015

Initiation of Phase 3 trial of IV rigosertib in HR-MDS: 2H2015

Presentation of Phase 2 data from the 09-08 oral rigosertib combination trial in MDS and AML: 4Q2015

Publication or presentation of results from the HR-MDS ONTIME trial: 4Q2015

Second Quarter 2015 Financial Results

Cash, cash equivalents, and marketable securities as of June 30, 2015 totaled $25.4 million, compared to $33.7 million as of March 31, 2015.

Total net revenue was $0.1 million for the second quarter of 2015 and $0.2 million for the six months ended June 30, 2015, compared to $0.1 and $0.6 million for the comparable periods in 2014.

Research and development expenses were $6.5 million for the second quarter of 2015 and $16.0 million for the six months ended June 30, 2015, compared to $12.9 and $27.2 million for the comparable periods in 2014.

General and administrative expenses were $2.6 million for the second quarter of 2015 and $5.5 million for the six months ended June 30, 2015, compared to $4.0 and $8.9 million for the comparable periods in 2014.
About Onconova Therapeutics, Inc.

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer. Onconova’s clinical and pre-clinical stage drug development candidates are derived from its extensive chemical library and are designed to work against specific cellular pathways that are important in cancer cells, while causing minimal damage to normal cells. In addition to rigosertib, the Company’s most advanced product candidate, two other candidates are clinical stage, and several candidates are in pre-clinical stages. For more information, please visit View Source

About Rigosertib

Rigosertib is a small molecule that inhibits cellular signaling by acting as a Ras mimetic. This is believed to be mediated by direct binding of rigosertib to the Ras-binding domain (RBD) found in many Ras effector proteins, including the Raf kinases and PI3K. The initial therapeutic focus for rigosertib is myelodysplastic syndromes (MDS), a group of bone marrow disorders characterized by ineffective formation of blood cells that often converts into acute myeloid leukemia (AML). Clinical trials with intravenous (IV) and oral formulations of rigosertib are being conducted at leading institutions in the U.S. and Europe.

On August 13, 2015 Heat Biologics, Inc. (NASDAQ: HTBX), a clinical stage biopharmaceutical company focused on the development of cancer immunotherapies, reported its financial results for the second quarter ended June 30, 2015 (Filing, 8-K, Heat Biologics, AUG 13, 2015, View Source [SID:1234507239]).

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Second Quarter 2015 Highlights & Recent Developments

· Announced the development of ComPACT, a next-generation combination immunotherapy platform

o Combines two important immunotherapy pathways, checkpoint inhibition and T-cell costimulation, in a single construct

o First IND with the ComPACT platform is expected in 2H 2016

· Presented interim immune response data from ongoing Phase 2 trial with HS-110 in advanced non-small-cell lung cancer at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting

· Announced new Phase 1b trial of HS-110 in non-small cell lung cancer in combination with other immunotherapeutic agents
o Trial will explore combination of HS-110 with various immune modulators.

· Provided an update on immune responder and non-responder phenotypes from the Phase 1 clinical trial of HS-410 in non-muscle invasive bladder cancer at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting

· Announced key management promotions

o Taylor Schreiber, M.D., Ph.D. to Chief Scientific Officer, responsible for leading the Company’s research efforts including its ImPACT and ComPACT Therapy drug development platforms

o Melissa Price, Ph.D., to Vice President of Product Development, responsible for directing the Company’s clinical development, regulatory and manufacturing strategy

"We continued to make advances and generate new data from our novel immunotherapy programs this quarter. Of particular note, we unveiled ComPACT, a next generation combination immunotherapy platform that holds the promise of delivering T-Cell priming and co-stimulatory molecules in a single biological product," said Jeff Wolf, Heat’s CEO. "We also announced a new Phase 1b trial of HS-110 in non-small cell lung cancer which will explore the combination of HS-110 with various immune modulators."

"We expect a number of important value creating milestones in the second half of 2015," continued Mr. Wolf. "For HS-410, our immunotherapy candidate for non-muscle-invasive bladder cancer, we plan to release one-year safety and immune response data from our Phase 1 trial and complete enrollment in our Phase 2 trial. Our accomplishments this quarter and throughout the year position us well to achieve our goals in the second half of 2015 and beyond."

Quarter Ended June 30, 2015 vs. Quarter Ended June 30, 2014
Research and development expenses for the second quarter of 2015 decreased 31% to $0.6 million, compared to the $0.8 million in the second quarter of 2014. The decrease is attributable to a decrease in pre-manufacturing costs associated with preparing to produce vaccines for use in our clinical trials, offset by increases in lab supplies and other fees.

Clinical and regulatory expenses increased to $3.4 million, compared to $1.1 million for the second quarter of 2014. The increase was attributable to increases in clinical trial execution costs, increases in investigator payments, as well as an increase in costs related to the production of vaccines for our clinical trials.

General and administrative expenses for the quarter were $0.9 million, compared to $1.0 million for the second quarter of 2014. The decrease is attributable to a decrease in personnel expense, a decrease in facility expense due to allocation to other departments, and a decrease in travel expenses.

Net loss attributable to Heat Biologics, Inc. in the second quarter was $4.7 million, or ($0.56) per basic and diluted share. This compares to a net loss of $2.8 million, or ($0.44) per basic and diluted share in the second quarter of 2014.

As of June 30, 2015, the Company had cash, cash equivalents, and short-term investments of approximately $19.3 million. This compares to cash, cash equivalents, and short-term investments of approximately $14.4 million at December 31, 2014.

Six Months Ended June 30, 2015 vs. Six Months Ended June 30, 2014
Research and development expenses for the six months ended June 30, 2015 (2015 Period) decreased to $1.1 million, compared to $1.4 million for the six months ended June 30, 2014 (2014 Period). The decrease from the 2014 Period to the 2015 Period is attributable to a decrease in pre-manufacturing costs associated with preparing to produce vaccines for use in our clinical trials, as well as decreases in patent, license and other professional fees, offset by increases in compensation costs associated with new hires, lab supplies and other costs, and depreciation related to the build out of the lab facility and other associated costs.

Clinical and regulatory expenses increased to $5.5 million, from $2.0 million in the first six months of 2014. The increase year over year is attributable to increases in clinical trial execution costs, increased investigator payments, as well as an increase in costs related to the production of vaccines for our clinical trials. Additionally, personnel cost, including consultants, professional fees, facilities costs, travel, and license fees increased.

General and administrative costs increased to $2.2 million, from $2.0 million in the comparable period in 2014. The increase is attributable to increase in pay to certain key employees, an increase in professional services such as accountants, attorneys and investor relations, and travel. These increases are offset by a decrease in facility and related costs as well as a decrease in depreciation.