On August 16, 2017 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer, wet age-related macular degeneration and fibrotic diseases, reported that positive results from the National Cancer Institute (NCI) Phase 1/2 trial of TRC105 and Nexavar (sorafenib) in hepatocellular cancer (HCC) were published in the August 15 issue of Clinical Cancer Research (Volume 23, Issue 16, pages 4633-4641) (Press release, Tracon Pharmaceuticals, AUG 16, 2017, View Source [SID1234520278]).

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The Phase 1b/2 trial enrolled a total of 26 patients with advanced HCC. Patients were dosed at one of four levels of TRC105 (3, 6, 10 and 15 mg/kg every two weeks), and with the standard dose of Nexavar of 400 mg twice daily. The overall response rate (ORR) in the 20 evaluable patients with measurable disease over all four dose levels was 25% (95% CI: 8.7-49.1%) by the Response Evaluation Criteria in Solid Tumors (RECIST), with all responses occurring at the highest two dose levels of TRC105. The ORR in the two highest dose levels of TRC105 was 33%. Four additional patients had confirmed stable disease, one of whom was treated for 22 months. Median progression free survival (PFS) was 3.8 months (95% CI: 3.2-5.6 months) and median overall survival (OS) was 15.5 months (95% CI: 8.5-26.3 months). Nexavar was approved for the treatment of patients with advanced HCC based on median OS of 10.7 months (95% CI: 9.4-13.3 months) versus 7.9 months (95% CI: 6.8-9.1 months) with placebo in the multicenter SHARP trial. The ORR for Nexavar treatment by RECIST in the SHARP trial was 2%.

NCI study researchers concluded that the combination of TRC105 and Nexavar was well-tolerated at the recommended single agent doses of both drugs, and that encouraging evidence of activity was observed. TRACON is currently sponsoring a separate Phase 1/2 multicenter study of TRC105 and Nexavar (ClinicalTrials.gov identifier NCT02560779) to confirm the activity reported by the NCI. The NCI will recruit patients into this multicenter study.

“The final data from the NCI study of TRC105 and Nexavar in HCC published today reinforce the encouraging preliminary data presented previously at ASCO (Free ASCO Whitepaper). Collectively, these data suggest that the combination of TRC105 and Nexavar is active in patients with HCC and support the advancement of this combination into further clinical studies,” said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. “We expect to report data from the TRACON-sponsored multicenter Phase 1/2 HCC trial in early 2018.”

About Carotuximab (TRC105) and other Endoglin Antibodies

TRC105 is a novel, clinical stage antibody to endoglin, a protein overexpressed on proliferating endothelial cells that is essential for angiogenesis, the process of new blood vessel formation. TRC105 is currently being studied in one Phase 3 and multiple Phase 2 clinical trials sponsored by TRACON or the National Cancer Institute for the treatment of solid tumors in combination with VEGF inhibitors. TRC105 has received orphan designation for the treatment of soft tissue sarcoma in both the U.S. and EU. The ophthalmic formulation of TRC105, DE-122, is currently in a Phase 2 clinical trial for patients with wet AMD. TRC205, a second generation antibody to endoglin, is undergoing preclinical testing in models of fibrosis. For more information about the clinical trials, please visit TRACON’s website at www.traconpharma.com/clinical_trials.php.

On August 16, 2017 Medigene AG (FSE: MDG1, Prime Standard, TecDAX) reported that scientists of the Company were invited to present two posters on its T cell receptor (TCR)-modified T cell therapies during the upcoming CRI-CIMT-EATI-AACR Conference (Press release, MediGene, AUG 16, 2017, View Source [SID1234520275]). The Cancer Research Institute (CRI), the Association for Cancer lmmunotherapy (CIMT) (Free CIMT Whitepaper), the European Academy of Tumor Immunology (EATI), and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) sponsor the Third International Cancer lmmunotherapy Conference that takes place in Mainz, Germany, from September 6-9, 2017.

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During the "Neoantigens and Cancer Mutations" session, Dr. Christiane Mummert, scientist at Medigene Immunotherapies, will present her poster describing a semi-automated method for the isolation and initial characterization of neoantigen-specific T cell receptors. In summary, the experiments demonstrated that neoantigen-specific TCRs can be isolated from peripheral blood samples of healthy donors and initially characterized in eight to ten weeks using an innovative high-throughput robotics platform.

On the second poster, presented during the "Adoptive Cell Therapy" session, shared co-authors Dr. Christian Ellinger and Dr. Manon Weis describe properties of Medigene’s PRAME-specific TCR for adoptive T cell immunotherapy of cancer. Using an innovative TCR isolation and characterization platform, a TCR with natural high avidity for a PRAME-derived epitope was isolated showing potent efficacy and a favorable safety profile that will be further evaluated in clinical trials.

Both posters will be presented during "Poster Session A" that will take place on September 6 between 6:00 pm – 8:00 pm.

About Medigene’s TCR technology: The TCR technology aims at arming the patient’s own T cells with tumor-specific T cell receptors (TCRs). The receptor-modified T cells are then able to detect and efficiently kill tumor cells. This immunotherapy approach attempts to overcome the patient’s tolerance towards cancer cells and tumor-induced immunosuppression by activating and modifying the patient’s T cells outside the body (ex vivo).
TCR therapy is developed to utilize a higher number of potential tumor antigens than other T cell-based immunotherapies, such as chimeric antigen receptor T cell (CAR T) therapy. Medigene is preparing the clinical development of its first TCR candidates and is establishing a pipeline of recombinant T cell receptors, and has established Good Manufacturing Practice (GMP)-compliant processes for their combination with patient-derived T cells.

Medigene’s first TCR immunotherapy "MDG1011" will be tested in a clinical Phase I/II trial intended to be started by the end of 2017.

Besides the planned company-sponsored TCR trial, Medigene is involved in an investigator-initiated trial (IIT) with TCRs in multiple myeloma (MM) to be conducted by the Charité – Universitätsmedizin Berlin in cooperation with the Max Delbrück Centre (MDC), Berlin.

On August 16, 2017 Kitov Pharmaceuticals Holdings Ltd. (NASDAQ: KTOV) (TASE: KTOV), an innovative biopharmaceutical company, reported a corporate update and reported financial results for the first half of 2017, ending June 30, 2017 (Press release, Kitov Pharmaceuticals , AUG 16, 2017, View Source [SID1234520266]).

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"We are pleased with the significant progress we have achieved during the recent months. Thanks to the intensive, dedicated and professional work of our staff, investigators and CROs, we recently submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for KIT-302, our lead drug candidate developed to simultaneously treat pain caused by osteoarthritis and to treat hypertension," said Kitov’s CEO, Isaac Israel. "We are confident in the quality of the submitted package, and look forward to a response from the FDA shortly regarding whether the NDA is complete and acceptable for filing.

"In addition, we have expanded our pipeline with NT-219, a promising cancer therapy candidate, through the acquisition of a controlling interest in TyrNovo. Results from pre-clinical studies of NT-219, a small molecule, demonstrated its efficacy in overcoming drug resistance in a variety of cancers and in combination with multiple pharmacologic cancer therapies. We are focused on advancing this highly promising therapeutic candidate into clinical trials in 2018 in order to provide enhanced treatment options to cancer patients.

"With the completion of the recent equity offering, we are well positioned to accomplish our strategic initiatives in 2018. Our plan is to continue growing Kitov as an innovative, emerging pharmaceutical company. Our mission is to leverage the significant potential of our product development pipeline to create long-term shareholder value."

Recent Corporate Highlights:

Submitted an NDA to the FDA for KIT-302
Signed a definitive License Agreement for KIT-302 for the territory of South Korea with Kuhnil Pharmaceutical Co. Ltd., a leading South Korea-based pharmaceutical company
Received waiver from FDA for the $2 Million New Drug Application fee for KIT-302
Completed recruitment of our renal function clinical trial to demonstrate the beneficial effects of KIT-302 on kidney function
Acquired a controlling interest in TyrNovo
Completed an equity offering for gross proceeds of $3.5 million

Expected Upcoming Milestones:

FDA filing of our NDA for KIT-302
Top-line results of our renal function clinical trial for KIT-302
Additional marketing agreements for the distribution of KIT-302
Pre-IND meeting with the FDA for NT-219

The information contained below should be read in conjunction with (1) our Unaudited Condensed Consolidated Interim Financial Statements as of June 30, 2017, and for the six months then ended; and, (2) our audited consolidated financial statements for the year ended December 31, 2016, which appears in the Company’s Annual Report on Form 20-F filed with the Securities and Exchange Commission on May 1, 2017, as well as the other information contained in such Annual Report on Form 20-F and in our Registration Statement on Form F-1 filed with the SEC (file number 333-211477).

Financial Results for Six Months Ended June 30, 2017

Research and development expenses for the six months ended June 30, 2017 were $2,516 thousand, an increase of $552 thousand, or 28%, compared to $1,964 thousand for the six months ended June 30, 2016. The increase resulted primarily from expenses deriving from preparation of our NDA submission to the FDA for KIT-302, our renal function clinical trial for KIT-302, and pre-clinical trials for NT-219.

General and administrative expenses for the six months ended June 30, 2017 were $2,524 thousand, an increase of $1,330 thousand or 111%, compared to $1,194 thousand for the six months ended June 30, 2016. The increase resulted from increases in salaries and benefits and directors’ fees, legal fees in connection with the Taoz settlement and the class action lawsuits, and officers’ and directors’ insurance.

Other expenses for the six months ended June 30, 2017 were $1,029 thousand, consisting of the fair value of rights granted to Taoz, a minority shareholder in TyrNovo, following the acquisition of TyrNovo.

Finance income, net for the six months ended June 30, 2017 was $56 thousand and is primarily related to interest on bank deposits. Finance income, net for the six months ended June 30, 2016 was $121 thousand and is primarily related to derivative instruments that expired during the period.

The Company’s net loss for the six months ended June 30, 2017 amounted to $6,013 thousand, compared with a loss of $3,037 thousand for the corresponding period in 2016.

Balance Sheet Highlights

Cash, cash equivalents and short-term deposits totaled $8,132 thousand as of June 30, 2017, compared to $14,657 thousand on December 31, 2016. The decrease compared to December 31, 2016 reflects the cash used in operations and the cash used in the acquisition of TyrNovo during the first half of 2017. In July 2017 the Company raised approximately $3.1 million, net in a direct registered offering.
Shareholders’ equity totaled $12,471 thousand, including $1,827 in non-controlling interests as of June 30, 2017, compared to $13,385 thousand as of December 31, 2016.

On August 16, 2017 Biocon reported that it has requested for withdrawal of the dossiers for biosimilars, Trastuzumab and Pegfilgrastim, as part of the procedural requirements for re-inspection by the European regulator EMA (Article, Biocon, AUG 16, 2017, View Source [SID1234520269]).

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"While our drug substance facilities for Trastuzumab and Pegfilgrastim were approved, the European regulatory authorities had informed us of the need for a re-inspection of our drug product facility for these products," the bio-pharmaceutical firm said in a regulatory filing.

The request for withdrawal of the dossiers and re-submission is part of the European Medicines Agency’s (EMA) procedural requirements linked to this re-inspection and will be considered by the EMA’s Committee for Medicinal Products for Human Use (CHMP), the statement added.

"We are on track to complete our corrective and preventive actions (CAPAs) by the end of this quarter and it is our intent to seek re-inspection and re-submission thereafter," spokesperson said.

On Aug. 16, 2017 Mateon Therapeutics, Inc. (OTCQX:MATN), a biopharmaceutical company developing vascular disrupting agents (VDAs) for the treatment of orphan oncology indications, reported results from its second scheduled interim analysis of the ongoing phase 2/3 FOCUS study evaluating CA4P in combination with bevacizumab (Avastin) and physician’s choice chemotherapy in patients with platinum resistant ovarian cancer (prOC) (Press release, Mateon Therapeutics, AUG 16, 2017, View Source [SID1234520263]).

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FOCUS is designed to evaluate whether the addition of CA4P improves progression-free survival (PFS), the primary endpoint of the study, as well as objective response rate (ORR) and other measures. All patients enrolled in the FOCUS study are receiving either CA4P or placebo plus the current standard-of-care for platinum-resistant ovarian cancer, bevacizumab (Avastin) and chemotherapy. The current interim analysis is based on initial results from the first 40 patients (19 with CA4P, 21 with placebo) in the study who have been treated for at least two months or discontinued from the trial. A total of 91 patients have been enrolled in the phase 2 portion of FOCUS. The next (third) interim analysis will be conducted when approximately ¾ of the enrolled patients (originally targeted at 80) have been treated for at least two months or discontinued from the study.

"We are encouraged that early data on the primary endpoint of the study continue to favor CA4P and that our investigational drug remains well tolerated," said William D. Schwieterman, M.D., President and Chief Executive Officer. "There is a large unmet medical need in the ovarian cancer market as patients with prOC have low survival rates and few treatment options. We look forward to additional and more mature data from the 3rd interim analysis expected just over one month from now – in this upcoming analysis we will have more data on the current patients, who will have had additional time under treatment, as well as initial efficacy and safety information on approximately 25 additional patients."

Efficacy Results

PFS, the primary endpoint of the study, continues to favor the CA4P group, with a 1.68 month increase in median PFS for the patients receiving CA4P compared to control (6.64 months vs. 4.96 months; HR=0.68; p=0.456). Progression events are available from 16 of 40 (40%) patients: six patients (31.6%) in the CA4P arm and ten (47.6%) patients in the control arm progressed or died while in the study.

Partial responses were observed in 4 of 16 (25.0%) patients treated with CA4P and 6 of 19 (31.6%) patients treated with the control regimen. Stable disease was observed in 9 of 16 (56.3%) patients treated with CA4P compared to 11 of 19 (57.9%) patients treated in the control arm.

Safety Results

CA4P continues to show a favorable safety profile. Most patients receiving CA4P experienced transient increases in blood pressure (BP) compared to the control arm (57.9% vs. 9.5%, respectively). BP increases generally peaked two hours following treatment and normalized without clinical sequelae two to three hours later. Rates of grade 3 hypertension were similar between the treatment and control arms (21.1% vs. 23.8%). There was one case of grade 4 hypertensive crisis in the CA4P arm. Adverse events that occurred in > 25% of patients and more frequently in the treatment arm included nausea, fatigue, cough, and hypertension, most of which were mild to moderate in severity. Rates of neutropenia, anemia, and thrombocytopenia were low and similar between treatment arms.