On August 16, 2016 Accurexa Inc. (the "Company" or "Accurexa") (ACXA), a biotechnology company focused on the development of novel neurological therapies to be directly delivered into the brain reported that it received a written response from the FDA (U.S. Food and Drug Administration) regarding a pre-IND (Investigational New Drug) meeting request for its ACX-31 program for the local delivery of temozolomide as adjunctive therapy to BCNU, both chemotherapeutics, in the treatment of brain tumors in conjunction with surgery and radiation (Press release, Accurexa, AUG 16, 2016, View Source [SID1234516522]). The FDA confirmed the acceptability of a 505(b)(2) application pathway with one first-in-human Phase 2 clinical trial followed by one Phase 3 clinical trial.

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Key Points of Written Response:

Acceptability of a 505(b)(2) application pathway confirmed
Pharmacology studies appear adequate
Non-clinical studies appear adequate with the exception of one GLP-compliant toxicology study in a single animal species using the route and schedule of administration proposed for the planned Phase 2 trial to be required
Phase 2 clinical trial strongly recommended to be conducted in patients with relapsed/recurrent glioblastoma
Proposed Phase 2 trial primary endpoints appear adequate
If an adequately designed Phase 2 trial demonstrates tolerability and suggests activity, FDA would encourage Accurexa to request an End-of-Phase 2 meeting to discuss the design of an adequate and well-controlled, randomized Phase 3 trial
"We are very pleased with the outcome of the FDA meeting. The FDA’s acceptance of a 505(b)(2) application pathway reduces the cost and time for the development of our ACX-31 Brain Cancer program than it would otherwise require. The FDA provided valuable guidance and we continue moving our ACX-31 program forward towards clinical development," said George Yu, MD, Accurexa’s President & CEO.

Under a 505(b)(2) application pathway the Company can rely on studies of previously approved drugs that were not conducted by the Company. The benefits of a 505(b)(2) application include lower risk due previous drug approvals, lower cost and accelerated development due to fewer studies, than a typical new drug application.

On August 16, 2016 Abeona Therapeutics Inc. (NASDAQ: ABEO) a clinical-stage biopharmaceutical company focused on delivering gene and plasma-based therapies for life-threatening rare diseases, reported financial results for the second quarter (Press release, Abeona Therapeutics, AUG 16, 2016, View Source;p=RssLanding&cat=news&id=2195628 [SID:1234514594]). The Company will provide investors an update on recent and ongoing business activities and an overview of its 2Q16 financials on, Wednesday, August 17th, at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-269-7756 (toll free domestic) or 201-689-7817 (international).

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"The second fiscal quarter of 2016 brought significant advancements in our goal of building a strong leadership position in the development of innovative therapies for rare diseases," stated Steven H. Rouhandeh, Executive Chairman. "We are excited about the initial biopotency signals seen in our Sanfilippo Type A clinical trial, and are looking forward to advancing multiple important new therapeutic candidates for the treatment of epidermolysis bullosa (EB), as announced last week. We thank our collaborators, shareholders and staff for their ongoing commitment and support as we move these therapies further into clinical development."

Timothy J. Miller, Ph.D, stated, "Abeona achieved many important regulatory and clinical milestones in the second quarter of 2016. The milestones include the dosing of the first patient in our Phase 1/2 clinical trial with ABO-102 for patients with Sanfilippo syndrome type A, the FDA allowance of our Phase 1/2 clinical study in Sanfilippo syndrome type B (MPS IIIB), and, most recently, the European regulatory approval for our MPS IIIA Phase 1/2 clinical study to be conducted at Cruces University Hospital in Bilbao, Spain. Additionally, we are excited about our new collaboration with the EB Research Partnership, EB Medical Research Foundation and Stanford University to accelerate up to three new promising EB product candidates toward commercialization."

Recent Abeona Operating Highlights

On May 17, 2016, Abeona announced that the first patient in its Phase 1/2 trial for ABO-102 (AAV-SGSH), a single treatment gene therapy strategy for patients with Sanfilippo syndrome type A (MPS IIIA), has been enrolled at Nationwide Children’s Hospital in Columbus, Ohio.
On May 24, 2016, Abeona announced the FDA Allowance of its Investigational New Drug (IND) for a Phase 1/2 clinical study with ABO-101 (AAV-NAGLU) for patients with Sanfilippo syndrome type B (MPS IIIB).
On August 2, 2016, Abeona provided an update on the initial subjects enrolled in this trial, stating that ABO-102 had been well tolerated with no safety or tolerability concerns identified through 30-days post-injection, and that encouraging signs of early biopotency had been observed in urinary and CSF GAG (heparan sulfate) measurements as well as potential disease-modifying effects in the liver and spleen.
On August 4, 2016, the Company announced it had received European regulatory approval by the Agencia Espanola de Medicamentos y Productos Sanitarios for its Phase 1/2 trial for ABO-102 (AAV-SGSH) to be conducted at Cruces University Hospital (Bilbao, Spain).
On August 9, 2016, Abeona announced a collaboration with the EB Research Partnership, EB Medical Research Foundation and Stanford University for the development of treatments for recessive dystrophic epidermolysis bullosa (RDEB). Clinical results for the lead EB program (EB-101) were recently presented at the opening Plenary Session of the Society for Investigative Dermatology in May 2016, and Investigators at Stanford are recruiting patients for a Phase 2 clinical trial of EB-101 in adolescents age 13 and older to determine the effect of type VII collagen gene corrective grafts on wound healing efficacy.
Second Quarter Summary Financial Results

Cash Position : Cash, cash equivalents and marketable securities as of June 30, 2016 were $34.3 million, compared to $37.4 million as of March 31, 2016. Net cash used in operating activities in the Six Months Ended June 30, 2016 was $5.6 million as compared to $5.0 million in the same period in 2015, an increase of $0.6 million.
Revenues : Revenues were $214 thousand for the second quarter of 2016, compared to $282 thousand in in the second quarter of 2015. Revenues consisted of a combination of royalties from marketed products, primarily MuGard, and recognition of deferred revenues related to upfront payments from early license agreements.
Loss per share : Loss per share was $0.20 for the second quarter of 2016, compared to a loss per share of $0.16 in comparable period in 2015.

On August 15, 2016 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported a corporate update and reported financial results for the second quarter ended June 30, 2016 (Press release, Onconova, AUG 15, 2016, View Source [SID:1234514563]).

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"We are encouraged by the progress of our global Phase 3 trial of our lead product candidate, rigosertib, for patients with myelodysplastic syndromes (MDS). The INSPIRE trial is actively enrolling patients in the U.S., Europe and Japan, and we have recently opened trial sites in Israel and Australia, bringing us more than two-thirds of the way to our target of approximately 135 sites worldwide. The enrollment of patients in Japan by our Japan/Korea partner, SymBio Pharmaceuticals, is further accelerating this important pivotal trial," said Ramesh Kumar, Ph.D., President and CEO of Onconova. "As a result of the completion of our oversubscribed rights offering in July, we believe we are positioned to deliver multiple key milestones in 2016 and 2017, including opening additional INSPIRE trial sites, pre-planned interim analysis and enrollment of approximately 225 patients. Finally, we have initiated discussions with U.S. and European regulatory authorities towards formal End-of-Phase 2 meetings to define the pathway forward for further development of oral rigosertib. We intend to provide an update on these discussions later this year."

Recent Business Highlights:

Completion of $17.4 Million Oversubscribed Financing

On July 29, 2016, Onconova closed its oversubscribed rights offering. Although the number of units able to be sold was capped at a maximum of 4,256,186 units (or approximately $17.4 million in gross proceeds), there was a total demand for approximately 4.9 million units in the rights offering.
Overall, 4,256,186 units consisting of a total of 3,599,786 shares of common stock, pre-funded warrants to purchase an additional 656,400 shares of common stock, and 3,192,022 tradable warrants were issued in this offering.
Including the net proceeds from the rights offering of approximately $15.8 million, Onconova had cash and cash equivalents of approximately $27.6 million at July 31, 2016.
Progress in INSPIRE Pivotal Trial of Rigosertib in Higher-risk MDS (HR-MDS)

The global INSPIRE trial is now enrolling patients in the United States, Europe and Japan. As of July 31, 2016, 103 sites, including 27 in the U.S., were open and recruiting patients. The first patient in Japan was enrolled in the trial in July by our partner, SymBio Pharmaceuticals, Inc.
Progress in Oral Rigosertib Combination with Azacitidine

Updated results from the Phase 2 trial 09-08 were presented in June 2016 at the 21st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper). Notably, the interim overall response rate was 77% (23 of 30 patients) among evaluable first- or second-line HR-MDS patients treated with oral rigosertib in combination with azacitidine. This trial is now fully enrolled and End-of-Phase 2 meetings to discuss the next stage of development with regulatory authorities in the U.S. and Europe are expected to occur in the second half of 2016.
Upcoming Events

Enrollment of patients in Israel, Australia and Canada for INSPIRE trial: 3Q2016

Key Opinion Leader investor event to discuss the potential future applications of the RAS-directed Mechanism of Action in oncology and for rigosertib: 3Q2016

End-of-Phase 2 meeting with FDA and European authorities to discuss trial results and future development plan for oral rigosertib in combination with azacitidine: 2H2016
Second Quarter 2016 Financial Results

Cash, cash equivalents, and marketable securities as of June 30, 2016 totaled $12.8 million, compared to $19.8 million as of December 31, 2015.
Total net revenue was $2.2 million for the second quarter of 2016 and $3.7 million for the six months ended June 30, 2016, compared to $0.1 million and $0.2 million, respectively, for the comparable periods in 2015.
Research and development expenses were $5.6 million for the second quarter of 2016 and $11.4 million for the six months ended June 30, 2016, compared to $6.5 million and $16.0 million, respectively, for the comparable periods in 2015.
General and administrative expenses were $2.1 million for the second quarter of 2016 and $5.3 million for the six months ended June 30, 2016, compared to $2.6 million and $5.5 million, respectively, for the comparable periods in 2015.

On August 15, 2016 AmpliPhi Biosciences Corporation (NYSEMKT:APHB), a global leader in the development of bacteriophage-based antibacterial therapies to treat drug-resistant infections, reported its financial results for the second quarter ended June 30, 2016 (Press release, AmpliPhi Biosciences, AUG 15, 2016, View Source [SID:1234514598]).

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"We have made significant progress executing on our clinical programs and look forward to presenting top-line data for both clinical trials later this year," said M. Scott Salka, CEO of AmpliPhi Biosciences. "The threat posed by drug-resistant infections is universally acknowledged. World leaders from government, medical and scientific communities continue to stress the importance of addressing this threat, and we remain confident that bacteriophage-based therapies will not only provide a potential alternative to existing broad-spectrum antibiotics, but will also be used to resensitize antibiotic-resistant infections to increasingly ineffective drugs, thereby ensuring the continued effectiveness of the drugs we currently use to treat serious, life-threatening infections."

Recent Corporate Highlights

Announced AB-SA01, our investigational phage therapy targeting Staphylococcus aureus (S. aureus) infections, was well-tolerated and caused no drug-related adverse events in the first cohort of our Phase 1 trial in patients with chronic rhinosinusitis (CRS). AmpliPhi remains on track to report final data later in 2016
Initiated the first Phase 1 phage therapy trial under a United States IND to evaluate the safety of AB-SA01 administered topically to the intact skin of 12 healthy adult volunteers in May. The trial is now fully enrolled and top-line results are expected before the end of the third quarter with the complete study report following later this year
The outstanding Series B Preferred stock was converted into common stock, streamlining AmpliPhi’s capital structure
Presented data at the European Congress of Clinical Microbiology and Infectious Diseases demonstrating that in a lung infection model, AB-PA01, our investigational phage therapy targeting Pseudomonas aeruginosa (P. aeruginosa) infections, was capable of infecting and killing 87.2% of the 429 clinical isolates in vitro, including those with multi-drug resistant strains of P. aeruginosa isolated from patients with cystic fibrosis
Strengthened our portfolio of intellectual property related to our proprietary bacteriophage platform with two new patents; one granted by the European Patent Office covering the treatment of all species of antibiotic-resistant bacterial infections through the staged use of bacteriophage preparations followed by the antibiotic to which the bacteria were initially resistant, and the second granted by the Japanese Patent Office that covers the same staged treatment regimen for P. aeruginosa infections
Completed a registered direct public offering of common stock and warrants in June, which yielded aggregate net proceeds of $4.2 million after fees and expenses
Second Quarter 2016 Financial Results

Cash and cash equivalents as of June 30, 2016 totaled $7.1 million. AmpliPhi anticipates that its current financial resources will provide sufficient cash to fund operations into the fourth quarter of 2016
Revenues related to sublicensing agreements from AmpliPhi’s former gene therapy program were $0.1 million for the quarter ended June 30, 2016 and for the same period in 2015
Research and development expenses for the quarter ended June 30, 2016 totaled $1.2 million compared to $1.1 in the same period of 2015. The increase was primarily attributable to an increase in personnel costs
General and administrative expenses for the quarter ended June 30, 2016 were $2.5 million compared to $1.6 million for the same period of 2015. The increase was primarily attributable to an increase in compensation costs and primarily for non-cash stock-based compensation expenses
Net cash used in operations was $6.0 million during the six month period ended June 30, 2016
There are currently 11.1 million shares of common stock outstanding
For more information, visit www.ampliphibio.com.

On August 15, 2016 Heat Biologics, Inc. ("Heat") (Nasdaq: HTBX), an immuno-oncology company developing novel therapies that activate a patient’s immune system against cancer, reported a general business update and reported its financial results for the second quarter and six months ended June 30, 2016 (Filing, Q2, Heat Biologics, 2016, AUG 15, 2016, View Source [SID:1234514608]).

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"We are pleased to report a number of important scientific, clinical and financial developments at the company," said Jeff Wolf, Heat’s Founder and CEO. "First, the preclinical findings demonstrating that our ComPACT technology secreting the co-stimulator OX40L significantly enhanced tumor rejection were published in the journal, ‘Cancer Immunology Research.’ Additionally, we reported encouraging interim study findings from our Phase 1b trial evaluating HS-110 in combination with nivolumab (Opdivo), a Bristol-Myers Squibb anti-PD-1 checkpoint inhibitor, for the treatment of non-small cell lung cancer."

"We remain focused on driving shareholder value and minimizing dilution. We have implemented a number of cost-saving measures to help ensure we achieve important data readouts expected in the fourth quarter with current cash on-hand. Furthermore, I am pleased to report we have generated approximately $2.0 million in additional cash from the exercise of warrants. Significantly, we have also regained compliance with NASDAQ’s minimum closing bid price requirement, which alleviates the immediacy of effecting a reverse stock split."

"Overall, we remain encouraged by the outlook for the business and the growing interest from within the industry to utilize our platform technology with checkpoint inhibitors and other immunotherapies to activate a patient’s immune system against cancer. Importantly, our allogeneic cell-based immunotherapy has the potential to offer a broader, off-the-shelf solution that addresses many of the past challenges that have plagued the immuno-oncology market."

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Recent Developments & Second Quarter 2016 Corporate Highlights

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Heat remains on track to report topline data in the fourth quarter from its Phase 2 trial evaluating HS-410 for the treatment of non-muscle invasive bladder cancer (NMIBC) and its Phase 1b trial evaluating HS-110 in combination with an anti-PD-1 checkpoint inhibitor for the treatment of non-small cell lung cancer (NSCLC).

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In July, Heat announced that preclinical findings from its ComPACT platform technology were published online in the journal "Cancer Immunology Research." Heat demonstrated that its ComPACT technology secreting the co-stimulator OX40L significantly enhanced tumor rejection in two cancer tumor types compared to OX40 agonist antibody treatment. Heat also reported that ComPACT-enhanced antigen-specific T cell infiltration into tumors improved memory T cell responses and demonstrated greater specificity than OX40 agonist antibody treatments. Furthermore, the findings also showed that the ComPACT platform can be adapted to secrete other costimulatory molecules, including TL1A, 4-1BBL and ICOSL.

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In June, Heat reported interim study findings from its Phase 1b trial evaluating HS-110 in combination with nivolumab for the treatment of NSCLC. The findings suggested that the addition of HS-110 to nivolumab does not alter the nivolumab safety profile to-date. In addition, case studies of three trial patients (one non-responder and two responders) were characterized. While all three patients showed a decrease in immune cell PD-1 expression, which is consistent with nivolumab’s mechanism of action, both responders also showed a decrease in immunosuppressor cells, as well as increases in activated effector T cells in the peripheral blood. Furthermore, the two responders showed an increase in CD8+ T cells in biopsy samples after treatment with HS-110 + nivolumab. These early data appear to suggest that HS-110 in combination with nivolumab may improve response rates for patients with "cold tumors," who have historically not responded to checkpoint inhibitors alone.

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In June, Heat presented a poster at the ASCO (Free ASCO Whitepaper) Annual Meeting reviewing the design and endpoints for the ongoing Phase 1b trial of HS-110 in combination with nivolumab.

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In April, Heat presented three posters at the AACR (Free AACR Whitepaper) Annual Meeting. In the poster entitled "Phase I/II Study of Patients with NMIBC Treated with Vesigenurtacel-L (HS-410) with or without BCG," Heat reported that no additional recurrences had been reported to-date, with all patients at least 18 months out from enrollment. In another poster, Heat reported initial preclinical results from its collaboration with OncoSec Medical Incorporated. In the third poster, Heat reported positive preclinical data on its next generation ComPACT platform technology.

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In April, Heat implemented cost-saving measures and a focused corporate strategy to achieve important data readouts in the fourth quarter with its current cash on-hand.

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In April, Heat appointed John Prendergast, Ph.D., to its Board of Directors.

Second Quarter 2016 Financial Highlights

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Research and development (R&D) expenses decreased to approximately $0.5 million in the second quarter of 2016 compared to approximately $0.6 million in the second quarter of 2015, a decrease of approximately $0.1 million. The decrease is primarily attributable to reductions in non-cash stock compensation expense related to equity grants awarded to one of our Scientific Advisory Board members in 2015.

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Clinical and regulatory expenses decreased to approximately $1.3 million in the second quarter of 2016 compared to approximately $3.4 million in the second quarter of 2015, a decrease of approximately $2.1 million. The decrease is primarily attributable to reductions in clinical trial execution costs.

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General and administrative (G&A) expenses increased to approximately $1.1 million in the second quarter of 2016 compared to approximately $0.9 million in the second quarter of 2015, an increase of approximately $0.2 million. The increase is primarily attributable to separation expenses related to the departure of two of our former executive officers, as well as other incremental operating expenses.

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Net loss for the second quarter of 2016 was $3.0 million compared to a net loss of $4.9 million for the second quarter of 2015.

Six Months Ended June 30, 2016 Financial Highlights

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R&D expenses decreased to approximately $1.0 million for the six months ended June 30, 2016 compared to approximately $1.1 million for the six months ended June 30, 2015, a decrease of approximately $0.1 million. The decrease is attributable to reductions in patent, license and other professional fees, as well as reductions in compensation costs attributable to deferral in salary as part of our cost-savings initiatives.

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Clinical and regulatory expenses decreased to approximately $4.5 million for the six months ended June 30, 2016 compared to approximately $5.5 million for the six months ended June 30, 2015, a decrease of approximately $1.0 million. The decrease is primarily attributable to reductions in clinical trial execution costs.

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G&A expenses decreased to approximately $2.1 million for the six months ended June 30, 2016 compared to approximately $2.2 million for the six months ended June 30, 2015, a decrease of approximately $0.1 million. The decrease is primarily attributable to reductions in professional services as we bring more services in-house.

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Net loss for the six months ended June 30, 2016 was $7.8 million compared to a net loss of $8.9 million for the six months ended June 30, 2015.

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Cash and cash equivalents totaled approximately $7.1 million at June 30, 2016 compared to cash, cash equivalents and short-term investments totaled approximately $11.6 million at December 31, 2015. This does not include approximately $2.0 million raised from the exercise of warrants subsequent to June 30, 2016.