On November 4, 2016 AbbVie (NYSE: ABBV), a global biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to veliparib, an oral poly (adenosine diphosphate [ADP]-ribose) polymerase (PARP) inhibitor, being investigated in combination with chemotherapies, such as carboplatin and paclitaxel, or radiation for the treatment of advanced squamous non-small cell lung cancer (NSCLC) (Press release, AbbVie, NOV 4, 2016, View Source [SID1234516249]).

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PARP is a naturally-occurring enzyme in the body that repairs damage to DNA in cells. While this repair is a useful process to maintain the integrity of healthy cells, the same process may also help repair DNA in cancer cells, causing them to survive.3 Researchers are investigating whether veliparib, a PARP inhibitor, in combination with DNA damaging therapies, such as chemotherapy or radiation, may lessen the repair of DNA damage in cancer cells, eventually causing some cells to die.3

NSCLC is the most common type of lung cancer in the U.S., accounting for approximately 80 to 85 percent of diagnosed cases.2 Survival in people with lung cancer can vary depending on the stage, or extent, of the cancer when it is diagnosed.2 Squamous cell carcinoma accounts for about 25 to 30 percent of NSCLC cases.2 It is usually found in the middle airways of the lungs and is often linked to a history of smoking.2

"Lung cancer is the leading cause of cancer-related deaths in the U.S. and can be difficult to treat, particularly when diagnosed in later stages. This Orphan Drug Designation for veliparib recognizes the significant unmet need in patients with advanced squamous non-small cell lung cancer," said Michael Severino, M.D., executive vice president of research and development and chief scientific officer, AbbVie. "AbbVie is committed to the ongoing development of veliparib in solid tumors to help advance the care of people living with cancer."

AbbVie is currently investigating the efficacy and safety of veliparib in combination with chemotherapy or radiation for the treatment of advanced squamous NSCLC, including in Phase 3 studies. Veliparib is not currently approved to treat any form of NSCLC.1

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.4

About Non-Small Cell Lung Cancer
Non-small cell lung cancer (NSCLC) is the most common type of lung cancer in the U.S., accounting for approximately 80 to 85 percent of diagnosed cases.2 There are three main subtypes of NSCLC: adenocarcinoma, squamous cell (epidermoid) carcinoma and large cell (undifferentiated) carcinoma.2 Currently, about 25 to 30 percent of NSCLC cases worldwide are squamous tumors.2 Survival in people with lung cancer can vary depending on the stage, or extent, of the cancer when it is diagnosed.2

About Veliparib
Veliparib is an investigational oral poly (adenosine diphosphate [ADP]–ribose) polymerase (PARP) inhibitor being evaluated in multiple tumor types.5,6 PARP is a naturally-occurring enzyme in the body that repairs damage to DNA in cells. While this repair is a useful process to maintain the integrity of healthy cells, the same process may also help repair DNA in cancer cells, causing them to survive.3

Discovered and developed by AbbVie researchers, veliparib is being studied in combination with chemotherapy or radiation to help determine whether it can prevent DNA repair in cancer cells to possibly increase the effectiveness of common DNA-damaging therapies, such as chemotherapy or radiation.5 Veliparib is currently being studied in more than a dozen cancers, including in Phase 3 studies in advanced squamous and non-squamous non-small cell lung cancer (NSCLC), ovarian cancer and breast cancer.7 Veliparib is an investigational medicine and its efficacy and safety have not been established by the U.S. Food and Drug Administration (FDA) or any other health authority.

On November 4, 2016 AVEO Oncology (NASDAQ:AVEO) reported financial results for the third quarter ended September 30, 2016 (Press release, AVEO, NOV 4, 2016, View Source;p=RssLanding&cat=news&id=2219710 [SID1234516250]).

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"In the last 18 months, we have made important progress in moving forward both elements of our strategy, including our oncology pipeline, for which we have retained significant North American rights, and our non-oncology pipeline, which is being advanced through partnerships with disease-area experts," said Michael Bailey, president and chief executive officer. "We anticipate a milestone rich calendar in the months ahead, spanning both our proprietary and partnered programs, that we expect will help define AVEO’s long-term potential in multiple areas of unmet medical need."

Mr. Bailey added: "For our lead oncology asset, tivozanib, we are well underway in three simultaneous paths. We expect an approval decision in first-line renal cell cancer (RCC) in Europe and initial results from the Opdivo combination TiNivo study in the first half of 2017, followed by, in the first quarter of 2018, pivotal top-line data from our U.S.-registration-directed Phase 3 TIVO-3 study in RCC. During 2017, we also expect to see progress in our partnered pipeline, serving to highlight the value-potential of these programs which are being developed at little or no cost to AVEO."

Potential Corporate Milestones through the First Half of 2017

Regulatory decision for tivozanib in the European Union and associated milestone payment by EUSA Pharma;
IND and proof-of-concept milestone payments for tivozanib in acute macular degeneration by Ophthotech;
Partnership for AV-353, a first-in-class opportunity to address a major unmet need in pulmonary arterial hypertension;
Initial safety results from the Phase 1 portion of the Phase 1/2 AVEO-sponsored TiNivo study of tivozanib in combination with Bristol-Myers Squibb’s Opdivo (nivolumab);
Development progress and milestone payments for AV-380 in Cachexia by Novartis;
Manufacturing tech transfer milestone payment for AV-203 by CANbridge.
Third Quarter and Recent Highlights

Initiation of Phase 1/2 TiNivo Trial Evaluating Tivozanib in Combination with Bristol-Myers Squibb’s Opdivo (nivolumab) in Advanced RCC. In August 2016, AVEO announced the initiation of a Phase 1/2 clinical trial of AVEO’s oral, once-daily, vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI), tivozanib, in combination with Bristol-Myers Squibb’s anti-PD-1 therapy, Opdivo (nivolumab), in advanced RCC, named the TiNivo Trial. The trial is being led by the Institut Gustave Roussy in Paris under the direction of Professor Bernard Escudier, MD, Chairman of the Genitourinary Oncology Committee. The Phase 1 trial is designed to evaluate whether tivozanib’s unique specificity and associated safety profile can overcome the tolerability issues that have challenged TKI-PD1 combinations to date. Initial safety results from the Phase 1 portion of the Phase 1/2 are expected to be available to the companies in the first half of 2017.
Ongoing Review of the Marketing Authorization Application (MAA) in Europe for Approval of Tivozanib as a First-Line RCC Treatment Option. EUSA Pharma, to which the Company licensed European and additional rights outside North America, is working to submit responses to the European Medicines Agency (EMA) Day 120 List of Questions before year-end. The MAA, which is based on tivozanib’s existing dataset, including the Phase 3 TIVO-1 study of tivozanib in first-line RCC, seeks approval for tivozanib as a first-line treatment for advanced RCC under the EMA’s centralized review process. Following the response, EUSA Pharma expects to receive the EMA Day 180 List of Outstanding Issues, the last review stopping period prior to a recommendation from the Committee for Medicinal Products for Human Use (CHMP) and the final approval decision from EMA. If approved, tivozanib’s distinct safety profile has the potential to offer a new, well tolerated alternative to currently approved VEGF TKIs, which, in clinical studies, have been associated with challenging tolerability necessitating significant dose reductions and interruptions.
Continued Execution of the Pivotal Phase 3 TIVO-3 Study of Tivozanib in RCC. In May 2016, AVEO announced the commencement of enrollment and patient treatment for the Company’s pivotal TIVO-3 trial, a randomized, controlled, multi-center, open-label study to compare tivozanib to sorafenib in subjects with refractory advanced RCC. The study continues to be on track to reach a top line readout in the first quarter of 2018. The Phase 3 trial is expected to enroll approximately 322 patients with recurrent or metastatic RCC who have failed at least two prior regimens, including VEGF-TKI therapy (other than sorafenib). Eligible patients may also have received checkpoint inhibitor therapy in earlier lines of treatment. Patients will be randomized 1:1 to receive either tivozanib or sorafenib, with no crossover between arms.

The TIVO-3 trial, together with the TIVO-1 trial, is designed to support a first- and third-line indication for tivozanib in the U.S. TIVO-3 would also provide a unique data set, in that it is expected to include the first randomized Phase 3 results showing treatment with a VEGF TKI following prior PD1 therapy, and is designed to support approval of the first VEGF TKI specifically labeled for third-line treatment.
Third Quarter 2016 Financial Highlights

AVEO ended Q3 2016 with $30.8 million in cash, cash equivalents and marketable securities as compared with $34.1 million at December 31, 2015.
Total collaboration revenue in Q3 2016 was approximately $1.0 million compared with $15.2 million Q3 2015.
Research and development expense was $4.4 million in Q3 2016 compared with $4.5 million for Q3 2015.
General and administrative expense was $2.1 million in Q3 2016 compared with $2.2 million for Q3 2015.
Net loss for Q3 2016 was $5.0 million, or a loss of $0.07 per basic and diluted share, compared with net income of $7.9 million, or income of $0.14 per basic and diluted share for Q3 2015.
Financial Guidance

We believe that our $30.8 million in cash resources could allow us to fund our planned operations into the fourth quarter of 2017. Furthermore, we expect that these cash resources, together with certain anticipated operational milestone payments from our collaboration partners, could allow us to fund our U.S. tivozanib development strategy through at least pivotal Phase 3 TIVO-3 top-line data as well as our tivozanib-PD-1 inhibitor combination trial.

On November 4, 2016 GlycoMimetics, Inc. (NASDAQ: GLYC) reported progress on its clinical development programs and its financial results for the quarter ended September 30, 2016 (Filing, Q3, GlycoMimetics, 2016, NOV 4, 2016, View Source [SID1234516276]).

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"For GlycoMimetics, the third quarter was highlighted by continued achievements in our clinical development programs, particularly with regard to GMI-1271, our clinical-stage E-selectin antagonist. We announced initiation of a Phase 1 clinical trial of GMI-1271 in multiple myeloma, expanding potential uses of the drug candidate. We continue to enroll the Phase 2 portion of the GMI-1271 AML trial in both newly diagnosed and relapsed/refractory patients. We also announced the initiation of a Phase 1 clinical trial of our next drug candidate, GMI-1359, in healthy volunteers. After the close of the quarter, we announced the acceptance of multiple abstracts for six posters and one oral presentation at ASH (Free ASH Whitepaper) in December 2016. With our new trial initiations and continued enrollment in our ongoing trials, we expect to be in a position to provide additional news on the clinical development of GMI-1271 and GMI-1359 in late 2016 and throughout 2017," said Rachel King, GlycoMimetics’ Chief Executive Officer.

Key Operational Highlights:
·

GlycoMimetics dosed the first patient in a Phase 1 clinical trial of GMI-1271 for multiple myeloma (MM) in September 2016. The multi-center, open-label dose escalation trial, which has begun in Ireland, is designed to measure the efficacy, safety and pharmacokinetics of GMI-1271 in combination with bortezomib-based chemotherapy among patients who have been diagnosed with MM and have not responded well to standard chemotherapy.

·

GlycoMimetics initiated dosing in a Phase 1 clinical trial of its next drug candidate, GMI-1359, in healthy volunteers. GMI-1359 is a small molecule drug candidate that simultaneously inhibits both E-selectin and CXCR4. In this first-in-humans trial, volunteer participants will receive a single injection of GMI-1359, after which they will be evaluated for safety, tolerability, pharmacokinetics

and pharmacodynamics over 16 days. The randomized, double-blind escalating dose study is being conducted at a single site in the United States.

·

We continue to recruit and dose patients in the Phase 2 portion of our clinical study evaluating GMI-1271 in AML in both newly diagnosed and relapsed/refractory patients at 8 active sites in the United States, Ireland and Australia. Having recently been granted fast track status by the FDA for GMI-1271 in this indication, GlycoMimetics plans to continue to engage with the FDA to discuss clinical and manufacturing planning as the program progresses.

·

GlycoMimetics also recently announced that six posters and one oral presentation on data from three of the company’s clinical programs will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s Annual Meeting in December 2016.

Third Quarter 2016 Financial Results:

·

Cash position: As of September 30, 2016, the Company had cash and cash equivalents of $45.3 million as compared to $46.8 million as of December 31, 2015.

·

Revenue: Revenue for the three-month periods ended September 30, 2016 and 2015 was not material. There were no milestone or royalty payments due from Pfizer during the three months ended September 30, 2016 or 2015.

·

R&D Expenses: The Company’s research and development expenses increased to $5.9 million for the quarter ended September 30, 2016 as compared to $5.0 million for the third quarter of 2015. The increase was due to higher costs associated with the clinical trials for GMI-1271 in AML and MM and for GMI-1359 in healthy volunteers, partially offset by a decrease in expenses related to manufacturing and process development for GMI-1271.

·

G&A Expenses: The Company’s general and administrative expenses decreased to $2.0 million for the quarter ended September 30, 2016 as compared to $2.1 million for the third quarter of 2015. The decrease was related to slightly lower legal expenses, patent fees and commercial research fees.

·

Shares Outstanding: Shares outstanding as of September 30, 2016 were 23,063,430.

About GMI-1271
GMI-1271 is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. Preclinical research points to the drug’s potential role in moving cancerous cells out of the protective environment of the bone marrow where they hide and escape the effects of chemotherapy. In preclinical studies using animal models of AML, the results of which were presented at meetings of the American Society of Hematology (ASH) (Free ASH Whitepaper), GMI-1271 was also associated with a reduction of chemotherapy-induced neutropenia and chemotherapy-induced mucositis.
About GMI-1359
GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. Since E-selectin and CXCR4 are both adhesion molecules that keep cancer cells in the bone marrow, we believe that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that affect the bone marrow such as AML and MM, as compared to targeting CXCR4 alone. In December 2015 at the annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), we presented preclinical data suggesting that GMI-1359 may enhance the ability of chemotherapy to target and improve survival rates in patients with a high-risk form of mutated AML.
About Rivipansel
GlycoMimetics’ most advanced drug candidate, rivipansel, a pan-selectin antagonist, is being developed for the treatment of vaso-occlusive crisis in sickle cell disease and is being evaluated in a Phase 3 clinical trial being conducted by its strategic collaborator, Pfizer.

On November 4, 2016 Apogenix, a biopharmaceutical company developing next-generation immuno-oncology therapeutics, reported the achievement of additional milestones under its licensing agreement with CANbridge Life Sciences for the development and commercialization of lead candidate APG101 (INN: asunercept) in China, Macao, Hong Kong and Taiwan, triggering further paymentsto Apogenix (Press release, Apogenix, NOV 4, 2016, View Source [SID1234524576]). The milestones are related to the successful implementation of the technology transfer necessary for the production of this CD95 ligand inhibitor at the Chinese production site.

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Over the course of the past few months, Apogenix and CANbridge have successfully transferred the necessary cell banks, assays, protocols, and know-how for the manufacturing of asunercept to China. The manufacturing process has proven to be very robust with product quality and process yields of the active substance comparable to the manufacturing process developed by Apogenix

CANbridge also reported that clinical development of asunercept is now underway in Taiwan. The recently initiated Phase I/II trial is evaluating asunercept plus temozolomide (TMZ) during and after radiation therapy in 55 patients with newly diagnosed glioblastoma. The study design consists of an open-label, dose-escalation Phase I trial, and a multi-center, double-blind, randomized, placebo-controlled Phase II trial. The Phase I trial will evaluate safety, tolerability, pharmacokinetics and preliminary efficacy. The Phase II part will evaluate efficacy and safety.

"We are very pleased with the progress of our collaboration with CANbridge and the achievement of additional milestones," said Thomas Hoeger, Ph.D., CEO of Apogenix. "With the successful technology transfer and the initiation of the Phase I/II trial in Taiwan,the clinical development of asunercept in Asia in now well underway. We look forward to CANbridge initiating further trials in China soon."

James Xue Ph.D., Chairman and CEO of CANbridge: "The complex transfer of asunercept production technology and know-how has been very efficient due to the excellent collaboration between the CANbridge and Apogenix teams. In particular, we were impressed by the exceptional level of enthusiasm and professional handling during the entire process."

About Asunercept (APG101)
Apogenix’s lead immuno-oncology candidate asunercept is a fully human fusion protein that consists of the extracellular domain of the CD95 receptor and the Fc domain of an IgG antibody. Asunercept is being developed for the treatment of solid tumors and malignant hematological diseases. By blocking the CD95 ligand, which negatively regulates erythrocyte production in myelodysplastic syndromes (MDS) patients, asunercept directly addresses the cause of the disorder and could thus potentially provide a cure for MDS. The World Health Organization (WHO) has recently assigned the international nonproprietary name (INN) "asunercept" for APG101.

On November 4, 2016 Celsion Corporation (NASDAQ:CLSN), a clinical stage oncology drug development company, reported that the United States Patent and Trademark Office (USPTO) has granted two patents: Patent No. 9,468,687 B2 – Immuno Gene Therapy for Treatment of Cancer and Hyperproliferative Diseases, which expands the use of GEN-1 into additional cancer treatment modalities in combination with other chemotherapeutics and Patent No. 9,144,546 – Nucleic Acid-Lipopolymer Compositions, which expands and extends previous patent claims on the making of and composition of formulations consisting of our PPC delivery polymer and nucleic acids (Press release, Celsion, NOV 4, 2016, View Source [SID1234516252]).

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These new patents further strengthen coverage of GEN-1, the Company’s DNA-based immunotherapy in development for the localized treatment of ovarian cancer and glioblastoma multiforme (GBM), which is already covered by a composition of matter patent in the United States.

"Issuance of these patents further strengthens Celsion’s growing position as a leader in the development of gene-based immunotherapies addressing some of the most difficult-to-treat cancers, such as ovarian cancer and GBM by covering the use of GEN-1 for treating solid tumors as a monotherapy and in combination with chemotherapy," said Michael H. Tardugno, chairman, president and CEO. "GEN-1 leverages our proprietary TheraPlas technology platform, harnessing the power of IL-12 immunotherapy with a targeted delivery system engineered to overcome the limitations associated with the development of other dosage forms of IL-12 therapies."

About GEN-1
GEN-1 is being evaluated in an ongoing Phase 1b dose-escalating clinical trial (the "OVATION Study") combining GEN-1 with the standard of care for the treatment of newly diagnosed patients with advanced ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debunking surgery. The OVATION Study is designed to enroll three to six patients per dose cohort at escalating doses of GEN-1 with the goal to identify a safe, tolerable and therapeutically active dose of GEN-1 by recruiting and maximizing an antitumor immune response.

As previously reported, all six patients in the first two cohorts of the OVATION Study experienced a clinically meaningful response, ranging from stable disease to one pathologically confirmed complete response. In addition, all patients sustained decreases of 90% or greater of the prospective indicator of the presence of ovarian cancer cells, CA-125 protein as well as highly impressive pathologically responses, which is associated with prolonged survival. The first three cohorts each enrolled three patients. Enrollment in the fourth and final cohort is underway, and Celsion expects to report full data from the OVATION Study by the first quarter of 2017. Future studies of GEN-1 will include a Phase I/II study combining GEN-1 with Avastin and Doxil for the treatment of recurrent ovarian cancer.