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."

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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.

On August 13, 2015 Hemispherx Biopharma (NYSE MKT: HEB) reported its financial results for the six months ended June 30, 2015 (Press release, Hemispherx Biopharma, AUG 13, 2015, http://www.hemispherx.net/content/investor/default.asp?goto=836 [SID:1234507244]). The net loss (including non-cash effects) was approximately $8,290,000 or $(0.04) per share as compared to a net loss of $8,792,000 or ($0.05) per share for the same six month period in 2014.

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Cash, cash equivalents and marketable securities were approximately $15,714,000 at June 30, 2015 as compared to $16,108,000 as of December 31, 2014.

We have recently expanded our international collaborative programs with both major components of our treatment platform (Ampligen, an experimental therapeutic, and Alferon N, and FDA approved commercial product, more fully described below) to create an expanded patient access path while seeking formal approval in the European Union, Australia, and New Zealand.

Also, we have recently initiated new programs in potential cancer therapy, based on a novel concept of synergy in cancer immunotherapy. The medical concept of therapeutic synergy is to use the highly selective Toll-like Receptors (TLR3) agonist, such as Ampligen, in combination with other immune therapeutic treatment to modify the immune microenvironment and potentially increase the number of patients anticipated to respond to such treatment. This concept is supported by preclinical studies in tumor models demonstrating certain interactions as well as results from early clinical studies.

Our overall objectives also include plans to continue seeking approval for commercialization of Ampligen in the United States and abroad as well as to widen existing commercial therapeutic indications of Alferon N Injection presently approved for sales in the United States and Argentina. In addition, we have formed collaborations with multiple research laboratories around the world to examine Ampligen, an experimental therapeutic, and Alferon N, an FDA-approved commercial product (for refractory venereal warts (HPV)) as potential preventatives for, and treatments of, Ebola Virus Disease (EVD) and other hazardous viruses including pandemic influenza and Middle East Respiratory Syndrome (MERS). Our ability to commercialize our products, widen commercial therapeutic indications of Alferon N Injection and/or capitalize on our collaborations with research laboratories to examine our products as potential preventatives for, and treatments of newly emerging viral diseases are subject to a number of significant risks and uncertainties including, but not limited to, our ability to enter into more definitive agreements with some of the research laboratories and others that we are collaborating with, to fund and conduct additional testing and studies, whether or not such testing is successful or requires additional testing and meets the requirements of the FDA and comparable foreign regulatory agencies. We do not know when, if ever, our products will be generally available for commercial sale for any new indications.

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.

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.