On November 6, 2017 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new immunotherapies, reported the release of four presentation abstracts highlighting data from the Company’s controlled human interleukin-12 (hIL-12) gene therapy candidate for brain cancer at the 22nd Annual Meeting and Education Day of the Society for Neuro-Oncology (SNO), November 16-19, 2017 in San Francisco (Press release, Ziopharm, NOV 6, 2017, View Source [SID1234521591]).

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With three oral and one poster presentations, the Company will update data on Ad-RTS-hIL-12 plus veledimex, a gene therapy designed to control the expression of hIL-12, a powerful cytokine that has demonstrated a targeted, anti-tumor immune response for the treatment of recurrent GBM (rGBM).

"In our Phase 1, multi-centric study of Ad-RTS-hIL-12 plus veledimex, we continue to show a marked improvement of survival in patients with recurrent glioblastoma over historical controls. We are excited about the long-lasting T-cell response that is being observed through brain biopsies as well as emerging radiologic and peripheral blood correlative studies," said Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM.

The Company previously presented data from a cohort of 15 patients receiving intra-tumoral Ad-RTS-hIL-12 with 20 mg of orally-administered veledimex following craniotomy with median overall survival (mOS) of 12.5 months at a mean follow up of 9.2 months, which compares favorably to historical controls. Emerging data revealed that an elevated ratio of circulating CD8+/FOXP3+ (effector/suppressor) T cells correlated with mOS which is consistent with IL-12-mediated activation of the immune system. The superior survival rate in the 20 mg cohort that had been observed will be updated in those patients that received low dose steroids.

"We look forward to sharing this important data with the scientific community at SNO," continued Dr. Lebel.

The Company plans to host a conference call and webcast soon after the presentations of data at SNO. Details of the call and webcast, including timing, will follow at a later date.

Details for the SNO presentations are as follows:

Poster Presentation Title: Phase 1 Study of Ad-RTS-hIL-12 plus Veledimex in Pediatric Brain Tumors
Author: Stuart Goldman, MD
Session: Friday Traditional Posters
Date and Time: Friday, November 17, 2017, 7:30 p.m. PT
Abstract code: 5571

E-Talk Title: A Phase 1 Study of Ad-RTS-hIL-12 + Veledimex in Adult Recurrent Glioblastoma
Presenter: E. Antonio Chiocca, M.D., Ph.D.
Session Title: Adult Therapeutics
Date and Time: Saturday, November 18, 2017, 5:32 — 5:36 p.m. PT
Abstract Code: ATIM-26

Title: Controlled Expression of IL-12 Improves Survival in Glioma by Activating the Immune Response in Mice and Humans
Presenter: John A. Barrett, Ph.D.
Session Title: Immunology — Preclinical and Clinical I
Date and Time: Sunday, November 19, 2017, 9:15-9:20 a.m. PT
Abstract Code: IMMU-34

Title: Controlled Local Expression of IL-12 as Gene Therapy Concomitant with Systemic Chemotherapy Improves Survival in Glioma
Presenter: John A. Barrett, Ph.D.
Session Title: Immunology — Preclinical and Clinical I
Date and Time: Sunday, November 19, 2017, 10:00-10:05 a.m. PT
Abstract Code: IMMU-33

All accepted abstracts will be accessible via SNO’s official journal.

About Ad-RTS-hIL-12 plus Veledimex:

ZIOPHARM is advancing Ad-RTS-hIL-12 plus veledimex as a gene therapy for glioblastoma. Ad-RTS-hIL-12 is an adenoviral vector administered via a single injection into the tumor and engineered to express hIL-12, a powerful cytokine that has demonstrated the potential to stimulate a targeted, anti-tumor immune response. The expression of hIL-12 is controlled and modulated with the RheoSwitch Therapeutic System (RTS) by the small molecule veledimex, an activator ligand which has been shown to cross the blood brain barrier. ZIOPHARM anticipates initiation of a pivotal registration trial for Ad-RTS-hIL-12 plus veledimex for the treatment of rGBM by the end of 2017. The Company also has initiated a Phase 1 study to evaluate the stereotactic administration of Ad-RTS-hIL-12 plus veledimex in adult patients with rGBM, as well as a trial to evaluate the gene therapy as a treatment for pediatric brain tumors. In addition, ZIOPHARM plans to initiate enrollment of adult patients with rGBM who will receive a single dose of Ad-RTS-hIL-12 plus veledimex in combination with a checkpoint inhibitor targeting programmed cell death protein 1 (PD-1) by the end of the year.

Heron Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Heron Therapeutics, 2017, NOV 6, 2017, View Source [SID1234521578]).

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On November 6, 2017 Sino Biopharmaceutical presented Promising Pipeline Presentation (Presentation, Sino Biopharmaceutical, NOV 6, 2017, View Source [SID1234525137]).

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On November 6, 2017 CombiMatrix Corporation (NASDAQ:CBMX), a family health molecular diagnostics company specializing in DNA-based reproductive health and pediatric testing services, reported financial and operating results for the three and nine months ended September 30, 2017 (Press release, CombiMatrix, NOV 6, 2017, View Source [SID1234521572]).

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Three Months Ended September 30, 2017 and 2016

CombiMatrix reported total revenues for the third quarter of 2017 of $4.0 million, a 23.5% increase from $3.2 million for the third quarter of 2016. The increase in the third quarter of 2017 was driven by higher test volumes in the reproductive health segment and improved reimbursement with higher average revenue per test across nearly all segments. Reproductive health diagnostic test revenues for the third quarter of 2017, which include prenatal, miscarriage analysis and preimplantation genetic screening (PGS) testing, increased 30.2% to $3.0 million with testing volumes increasing 18.9% to 1,764. Also, total billable customers increased to 277 during the third quarter of 2017 compared to 257 during the third quarter of 2016.

Total operating expenses were $4.6 million for the third quarter of 2017 compared with $4.1 million for the prior-year comparable period. The increase was due primarily to increased general and administrative expenses, which included $391,000 of merger-related expenses not incurred during the comparable 2016 period. Operating expenses also increased from higher cost of services associated with increased testing volumes, and were partially offset by lower sales and marketing expenses related to optimized headcount in the field. Excluding merger-related expenses, non-GAAP general and administrative expenses for the third quarter of 2017 were $1.5 million. A reconciliation of GAAP to non-GAAP measures is provided below. Gross margin improved to 59.4% for the third quarter of 2017 from 54.0% for the third quarter of 2016, driven primarily by improved average reimbursement per test reflected above as well as from cost containment strategies undertaken in recent periods.

Net loss for the third quarter of 2017 was $610,000, or $0.21 per share, and non-GAAP net loss for the third quarter of 2017 was $219,000, or $0.08 per share. Net loss for the third quarter of 2016 was $856,000, or $0.38 per share. The improvement in net loss was due primarily to the 23.5% increase in total revenues described above, coupled with improved gross margins. See below for a reconciliation of GAAP to non-GAAP measures.

Nine Months Ended September 30, 2017 and 2016

CombiMatrix reported total revenues for the first nine months of 2017 of $12.0 million, a 29.1% increase from $9.3 million for the first nine months of 2016. The increase in total revenues for the first nine months of 2017 was driven by increased volumes for reproductive health and pediatric segments as well as improved reimbursement resulting in higher revenue per test across nearly all segments.

Operating expenses for the first nine months of 2017 were $13.5 million compared with $12.9 million from the prior-year comparable period, with the increase due primarily to increased general and administrative expenses, which included $601,000 of merger-related expenses not incurred during the comparable 2016 period. Operating expenses also increased from higher cost of services associated with increased testing volumes, and were partially offset by lower sales and marketing expenses related to optimized headcount in the field. Excluding merger-related expenses, non-GAAP general and administrative expenses for the first nine months of 2017 were $4.8 million. General and administrative expenses for the first nine months of 2016 were $4.6 million. Gross margin improved to 60.2% for the first nine months of 2017 from 52.9% for the first nine months of 2016. See below for a reconciliation of GAAP to non-GAAP measures.

Net loss attributable to common stockholders for the first nine months of 2017 was $1.5 million, or $0.52 per share and non-GAAP net loss for the first nine months of 2017 was $897,000, or $0.31 per share. Net loss attributable to common stockholders for the first nine months of 2016 was $5.2 million, or $3.48 per share. The higher net loss in the 2016 period reflected one-time, non-cash charges of $1.9 million related to deemed dividends from the issuance of Series F convertible preferred stock and warrants in the $8.0 million public offering that closed on March 24, 2016. This increase was partially offset by the reversal of the $890,000 Series E deemed dividend recognized in 2015 from the repurchase of those securities upon closing of the public offering, partially reduced by $656,000 of deemed dividends paid to the Series E investors in February of 2016. See below for a reconciliation of GAAP to non-GAAP measures.

The Company reported $2.4 million in cash, cash equivalents and short-term investments as of September 30, 2017, compared with $3.7 million as of December 31, 2016. The Company used $389,000 and $995,000 in cash to fund operating activities during the quarter and nine months ended September 30, 2017, respectively. On a non-GAAP basis excluding merger-related expenditures, the Company used $53,000 and $591,000 in cash to fund operating activities during the quarter and nine months ended September 30, 2017, respectively. The Company used $817,000 and $3.4 million in cash to fund operating activities during the quarter and nine months ended September 30, 2016, respectively. The significant decrease in cash used to fund operating activities in the 2017 periods resulted primarily from improved cash reimbursement of $3.7 million and $10.9 million for the quarter and nine months ended September 30, 2017, respectively, compared with $3.1 million and $8.5 million for the quarter and nine months ended September 30, 2016, respectively. Improved gross margins and lower sales and marketing expenditures also contributed to the overall improvement in cash used to fund operating activities for all periods presented. See below for a reconciliation of GAAP to non-GAAP measures.

Non-GAAP Financial Measures

Consolidated financial information has been presented in accordance with GAAP as well as on a non-GAAP basis. On a non-GAAP basis, financial measures excluded the effect of merger-related expenses in calculating the non-GAAP operating expenses, net loss measures and cash used in operations. CombiMatrix has incurred significant expenses in connection with its proposed merger with Invitae Corporation, which it generally would not have otherwise incurred in the periods presented as part of its continuing operations. Merger-related expenses incurred to-date consist primarily of legal and accounting costs incurred associated with execution of the merger and related agreements and filing of various merger-related proxy materials. CombiMatrix management believes it is useful to understand the effects of these items on the total operating expenses, net loss measures and cash used in operations.

CombiMatrix management uses these non-GAAP financial measures to monitor and evaluate its operating results and trends on an ongoing basis, and internally for operating, budgeting and financial planning purposes. CombiMatrix management believes the non-GAAP information is useful for investors by offering them the ability to identify trends in what management considers to be CombiMatrix’s core operating results and to better understand how management evaluates the business. These non-GAAP measures have limitations, however, because they do not include all items of expense that affect CombiMatrix. These non-GAAP financial measures are not prepared in accordance with, and should not be considered in isolation of, or as an alternative to, measurements required by GAAP, and therefore these non-GAAP results should only be used for evaluation in conjunction with the corresponding GAAP measures. A description of the non-GAAP calculations and reconciliation to comparable GAAP financial measures is provided in the accompanying table entitled “Reconciliation of GAAP to Non-GAAP Financial Measures.”

On November 6, 2017 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported that it will host a reception for investors and analysts on Friday, November 10, 2017, from 6:00 a.m. to 8:00 a.m. to discuss the clinical development of IPI-549, including a review of data from the ongoing Phase 1/1b clinical study. The reception will take place in conjunction with the 2017 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting at the Gaylord National Hotel and Convention Center in National Harbor, MD (Press release, Infinity Pharmaceuticals, NOV 6, 2017, View Source;p=RssLanding&cat=news&id=2314510 [SID1234521584]). IPI-549 is an orally administered immuno-oncology development candidate that selectively inhibits phosphoinositide-3-kinase gamma (PI3K-gamma) and is believed to be the only PI3K-gamma inhibitor in clinical development.

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Featured speakers at the reception will include:

David Hong, M.D., Deputy Chair, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX
Taha Merghoub, Ph.D., Co-Director, Ludwig Collaborative Laboratory and the Swim Across America Laboratory at Memorial Sloan Kettering
The presentation portion of the reception will be webcast beginning at 6:30 a.m. ET. The webcast and accompanying slides can be accessed in the "investors/media" section of the company’s website, www.infi.com. A replay of the event will also be available.

About the IPI-549 and the Ongoing Phase 1 Study
IPI-549 is an investigational, orally administered immuno-oncology development candidate that selectively inhibits PI3K-gamma. In preclinical studies, IPI-549 reprograms macrophages from a pro-tumor, M2, to an anti-tumor, M1, phenotype and is able to overcome resistance to checkpoint inhibition as well as to enhance the activity of checkpoint inhibitors.1,2 As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially complementary approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

A Phase 1 study of IPI-549 in patients with advanced solid tumors is ongoing to evaluate the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with Opdivo in approximately 200 patients with advanced solid tumors.3 The four-part study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. Patient enrollment is complete in monotherapy dose-escalation, and monotherapy expansion is ongoing. Combination dose-escalation is also ongoing, and combination expansion is expected to begin in the second half of 2017.

The combination expansion component includes multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer (NSCLC), melanoma, and head and neck squamous cell carcinoma (HNSCC) whose tumors show initial resistance or subsequently develop resistance to immune checkpoint blockade therapy. This combination expansion will also include a cohort of patients with triple negative breast cancer (TNBC) who have not been previously exposed to immune checkpoint blockade therapy. Although there has been great progress in the treatment of cancer, there remains a need for additional treatment options. NSCLC, melanoma, HNSCC and TNBC account for more than 22 percent of all new cancer cases in the U.S.4,5

IPI-549 is an investigational compound and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.