Apogenix Reports Significant Progress in Collaboration with CANbridge

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.

Amethyst NSCLC Trial: Phase 2 Trial of MGCD265 in Patients (pts) with Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) with Activating Genetic Alterations in Mesenchymal-Epithelial Transition Factor (MET)

Amethyst NSCLC Trial: Phase 2 Trial of MGCD265 in Patients (pts) with Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) with Activating Genetic Alterations in Mesenchymal-Epithelial Transition Factor (MET)

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Data from Three GlycoMimetics Drug Candidates to Be Highlighted at 58th Annual American Society of Hematology (ASH) Meeting

On November 4, 2016 GlycoMimetics, Inc. (NASDAQ: GLYC) reported that data from three drug candidates in its development pipeline will be presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Expo (Press release, GlycoMimetics, NOV 4, 2016, View Source [SID1234516348]). The ASH (Free ASH Whitepaper) meeting will take place December 3 to 6, 2016, at the San Diego Convention Center.

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The oral presentation and six posters to be shared at the ASH (Free ASH Whitepaper) meeting will review the results of pre-clinical and clinical research on the pan-selectin antagonist, rivipansel, that GlycoMimetics is developing in partnership with Pfizer, Inc.; the company’s E-selectin antagonist, GMI-1271; and the company’s dual E-selectin/CXCR4 antagonist, GMI-1359. The data presented will show significant progress in a clinical trial for acute myeloid leukemia (AML), pre-clinical data further expanding on the mechanism and potential for therapeutic benefit in sickle cell anemia, and pre-clinical data on effects of GMI-1359 in models of hematologic malignancies. The company will present updates with interim data from an on-going Phase 1/2 trial of GMI-1271 in patients with AML. This will include an update on patients enrolled in the Phase 1 portion of the trial as well as data on evaluable patients enrolled to date in the two Phase 2 arms of the study. The Phase 2 portion includes one arm enrolling patients with relapsed or refractory disease and one arm enrolling patients over the age of 60 with newly diagnosed disease.

"We are delighted to have multiple abstracts accepted at ASH (Free ASH Whitepaper), highlighting the success and breadth of our growing pipeline," said Rachel King, Chief Executive Officer of GlycoMimetics. "Our new clinical and preclinical data show the scientific rationale for continuing development of all three drug candidates, as well as significant momentum in our clinical program for GMI-1271 in AML."

Details of the data to be presented at ASH (Free ASH Whitepaper), including session times and locations, include:

Oral Presentation (submitted by Pfizer, Inc.)

Abstract #270-Rivipansel: A Small Pan-Selecting Antagonist Improves Cerebral Perfusion and Inhibits Leukocyte Adhesion and in Murine Sickle Cell Disease Model. Sunday, Dec. 4, 7:30-9:00 a.m. PT, Room 7AB.

Posters (all poster sessions are in the San Diego Convention Center, Hall G)

Abstract #4049–A Phase I/II Study of GMI¬1271, a Novel E¬Selectin Antagonist, in Combination with Induction Chemotherapy in Relapsed/Refractory and Elderly Previously Untreated Acute Myeloid Leukemia; Results to Date. Monday, Dec. 5, 6:00-8:00 p.m. PT. Poster Session

Abstract #3826–E¬Selectin Antagonist GMI¬1271 Shows a Favorable Safety, PK and Bleeding Profile in Phase I Studies of Healthy Volunteers. Monday, Dec. 5, 6:00-8:00 p.m. PT. Poster Session

Abstract #2823–Vascular E-Selectin Protects Leukemia Cells from Chemotherapy By Directly Activating Pro-Survival NF-Kb Signaling – Therapeutic Blockade of E-Selectin Dampens NF-Kb Activation. Sunday Dec. 4, 6:00-8:00 p.m. PT. Poster Session

Abstract #3519—Dual E-Selectin/CXCR4 Antagonist GMI-1359 Exerts Efficient Anti-Leukemia Effects in a FLT3 ITD Mutated Acute Myeloid Leukemia Patient-Derived Xenograph Murine Model. Sunday Dec. 4, 6:00-8:00 p.m. PT. Poster Session

Abstract #2826—Administration of the Dual E-Selectin/CXCR4 Antagonist, GMI-1359, Results in a Unique Profile of Tumor Mobilization from the Bone Marrow and Facilitation of Chemotherapy in a Murine Model of FLT3 ITD AML. Sunday Dec. 4, 6:00-8:00 p.m. PT. Poster Session

Abstract #2509–Rivipansel (GMI-1070) Inhibits E-Selectin Recognition of Sialyl LewisX Activation and Arrest of Human Neutrophils Expressed on CD62L (L-selectin) and Blocks Integrin. Sunday Dec. 4, 6:00-8:00 p.m. PT. Poster Session

The meeting abstracts are available at ASH (Free ASH Whitepaper)’s website.

Atara Bio Announces Third Quarter 2016 Financial Results and Recent Highlights

On November 4, 2016 Atara Biotherapeutics, Inc. (Nasdaq:ATRA), a biopharmaceutical company developing meaningful therapies for patients with severe and life-threatening diseases that have been underserved by scientific innovation, reported financial results for the third quarter ended September 30, 2016 and recent operational highlights (Press release, Atara Biotherapeutics, NOV 4, 2016, View Source [SID1234516329]).

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"We are pleased to report that for our lead product candidate, EBV-CTL, we have received feedback from FDA on our approach to comparing material manufactured by our CMO with material previously produced at MSK and have commenced manufacturing to support our Phase 3 trials," said Isaac Ciechanover, Chief Executive Officer and President of Atara Bio. "We have also submitted our Phase 3 trial protocols to FDA, and we look forward to the initiation of these trials by year end."

Recent Highlights and Anticipated Upcoming Milestones

EBV-CTL

Submitted Phase 3 protocols to the U.S. Food and Drug Administration (FDA) incorporating its feedback on two trials of allogeneic Epstein-Barr virus (EBV)-specific cytotoxic T lymphocytes (EBV-CTL) in patients with rituximab refractory EBV-post transplant lymphoproliferative disorder (PTLD) following hematopoietic cell transplant (HCT) and solid organ transplant (SOT).
Phase 3 trials are expected to initiate by year end.

Completed manufacturing process transfer for EBV-CTL to our contract manufacturing organization (CMO).
Developed end-to-end supply chain and logistics to support manufacturing and distribution of EBV-CTL.

Received feedback from FDA regarding our comparability protocol designed to compare EBV-CTL material manufactured by our CMO with material previously produced at Memorial Sloan Kettering (MSK).
Commenced production of full scale EBV-CTL lots for the expanded access protocol (EAP) and the Phase 3 trials.

Granted access to priority medicines (PRIME) regulatory support by the European Medicines Agency (EMA) for allogeneic EBV-CTL for the treatment of EBV-PTLD after HCT.

Scientific Advice meeting with the EMA and health technology assessment groups (HTAs) scheduled in the 4th quarter of 2016 to discuss potential pathways for submission of a marketing authorization application (MAA) for EBV-CTL in the treatment of rituximab refractory EBV-PTLD after HCT.

First patient dosed in multi-center EAP trial of EBV-CTL in patients with rituximab refractory EBV-PTLD.
We plan to broaden our EAP trial to include enrollment of patients with other EBV-associated hematologic malignancies and solid tumors.
CMV-CTL

Conducted an end of Phase 2 meeting with the FDA to discuss late-stage development of allogeneic cytomegalovirus (CMV)-specific CTL (CMV-CTL) for the treatment of anti-viral refractory or resistant CMV infection following either HCT or SOT.
We expect to initiate a Phase 3 trial in 2017, once we have completed discussions with the FDA on trial design.

Received positive opinion from the EMA on orphan drug designation for the treatment of CMV infection in patients with impaired immune systems.
CTL Platform Expansion

Expanded our relationship with the Queensland Institute of Medical Research (QIMR Berghofer) including the development of allogeneic CTLs targeting human papilloma virus (HPV) and BK virus (BKV).
HPV is associated with a number of solid tumors including head and neck cancer, cervical cancer, and anal cancer.
BKV is a challenging clinical problem in kidney transplant patients and is a potential cause of organ loss.
Third Quarter 2016 Financial Results

Cash and investments as of September 30, 2016 totaled $278.1 million, which the Company believes will be sufficient to fund its planned operations through 2018.
The Company reported a net loss of $25.4 million, or $0.88 per share, for the third quarter of 2016, as compared to a net loss of $11.9 million, or $0.43 per share, for the third quarter of 2015.
Total research and development expenses were $18.8 million for the third quarter of 2016, compared to $8.1 million for the third quarter of 2015, including $2.6 million and $0.9 million of non-cash stock-based compensation expenses, respectively. The increase was primarily due to an increase in our clinical trial, research, regulatory and manufacturing expenses associated with our T-cell programs of $10.9 million and increased headcount related costs to support these activities of $4.4 million, offset by a decrease in costs associated with our other molecular programs of $4.6 million.
General and administrative expenses were $7.1 million for the third quarter of 2016, compared to $4.1 million for the third quarter of 2015, including $2.7 million and $1.3 million of non-cash stock-based compensation expenses, respectively. The increase was primarily due to a $1.4 million increase in non-cash stock-based compensation driven by new award grants, a $0.9 million increase in compensation-related expenses driven by increased headcount, and to a lesser extent, higher consulting and outside services costs.

LION BIOTECHNOLOGIES REPORTS THIRD QUARTER 2016 FINANCIAL RESULTS AND PROVIDES CORPORATE UPDATE

On November 4, 2016 Lion Biotechnologies, Inc. (NASDAQ: LBIO), a biotechnology company developing novel cancer immunotherapies based on tumor-infiltrating lymphocyte technology (TIL), reported its third quarter 2016 financial results and provided a corporate update (Press release, Lion Biotechnologies, NOV 4, 2016, View Source [SID1234516328]).

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"Lion continues building momentum in broadening the utility of our TIL technology in new indications through internal R&D as well as with our collaborators. We recently announced our collaboration with the Karolinska Institute which expands the utility of TIL into two new indications, glioblastoma and pancreatic cancer. We also extended our Cooperative Research and Development Agreement (CRADA) with Professor Rosenberg at the National Cancer Institute (NCI) for an additional 5 years. We continue our process development work in optimizing the process of manufacturing TIL. Some of our preliminary work on developing a more robust and lower cost processes for growth of TIL will be presented at the upcoming 2016 SITC (Free SITC Whitepaper) meeting. We are building our team to become a fully integrated immuno-oncology company with new members with extensive expertise in cell-based therapy as evident by our recent hire of our CFO, Greg Schiffman. We have now doubled the number of our employees since June 2016 and moved to our San Carlos headquarters," said Dr. Maria Fardis, Chief Executive Officer of Lion Biotechnologies.

Recent Business Highlights and Anticipated Milestones

Appointed New Chief Financial Officer: In October 2016, Greg Schiffman, was appointed Chief Financial Officer (CFO) of Lion. Mr. Schiffman has extensive experience in drug development and therapeutics using cellular technologies. Prior to joining Lion Biotechnologies, Mr. Schiffman was Executive Vice President and CFO of StemCells, Inc, a publicly traded company engaged in the research, development, and commercialization of stem cell therapeutics. Prior to that he served as Executive Vice President and CFO of Dendreon Corporation, a publicly traded biotechnology company engaged in the discovery, development and commercialization of novel therapeutics using a proprietary cellular immunotherapy technology.
Entered into License with PolyBioCept AB and Related Clinical Trials Agreement with Karolinska University Hospital: In September 2016, the Company entered into an Exclusive License Agreement with PolyBioCept AB, a Swedish corporation. PolyBioCept has filed two patent applications with claims related to a cytokine cocktail for use in expansion of lymphocytes. Under the License Agreement, the Company received the exclusive right and license to PolyBioCept’s intellectual property to develop, manufacture, market and genetically engineer TIL produced by expansion, selection and enrichment using a cytokine cocktail. The Company also received a co-exclusive license (with PolyBioCept) to develop, manufacture and market genetically engineered TIL under the same intellectual property. The licenses are for the use in all cancers and are worldwide in scope, with the exception that the uses in melanoma are not included for certain countries of the former Soviet Union. The agreement has an initial term of 30 years. Under the terms of the clinical trials agreement, Lion will fund two clinical studies in glioblastoma and pancreatic cancer to be conducted at the Karolinska University Hospital in which TIL is manufactured using the licensed combination of cytokines. Both Phase 1 trials are expected to begin in 2017.
Cooperative Research and Development Agreement with NCI Extended: In August 2016, the Company entered into the second amendment of the CRADA with the NCI, for research and development related to an adoptive cell therapy utilizing TIL in the treatment of metastatic melanoma. The amendment extended the term of the CRADA by five years to August 2021 and modified the focus on the development of TIL as a stand-alone therapy or in combination with FDA-licensed products and commercially available reagents routinely used for adoptive cell therapy.
Two Additional Programs to Enter Phase 2 in 2017: The Company plans to initiate Phase 2 trials for LN-145 for the potential treatment of head and neck and cervical cancers in 2017.
Enrollment in LN-144 Phase 2 Melanoma Study Continues: Lion continues enrollment of patients in the LN-144 Phase 2 melanoma study and intends to present initial data at an upcoming medical conference in 2017.
Headcount: During the three months ended September 30, 2016, the Company increased its headcount from 23 employees to 45 employees to support the expansion of the Company’s clinical product pipeline and development activities including next generation TIL technologies.
Opened New Corporate Headquarters: Lion’s corporate headquarters has now moved to the San Carlos, California location. Lion will, however, retain its existing New York City and Tampa offices.
Third Quarter and Year-to-Date 2016 Financial Results

As of September 30, 2016 the Company held $179.3 million in cash and cash equivalents and short-term investments, compared to $103.7 million as of December 31, 2015.

GAAP and Non-GAAP net loss attributable to common stockholders

GAAP net loss attributable to common stockholders, which included a one-time deemed dividend charge of $49.5 million incurred as a result of the conversion feature of the Series B convertible preferred stock, for the quarter ended September 30, 2016 was $68.2 million, or ($1.15) per share, compared to GAAP net loss attributable to common stockholders of $7.6 million or ($0.16) per share for the quarter ended September 30, 2015. The deemed dividend did not have any monetary impact for the Company.

Non-GAAP net loss attributable to common stockholders, which excludes amounts related to stock-based compensation and the non-cash deemed dividend, for the quarter ended September 30, 2016 was $10.1 million, or ($0.17) per share, compared to non-GAAP net loss attributable to common stockholders of $5.2 million, or ($0.11) per share for the quarter ended September 30, 2015. The non-GAAP net loss attributable to common stockholders for the three months ended September 30, 2016 excludes $8.6 million of non-cash stock-based compensation and a non-cash deemed dividend of $49.5 million. The stock compensation increase year-over-year of $6.3 million is primarily driven by the departure of the Company’s former CFO. The deemed dividend will only impact the current quarter’s financial statements.

GAAP net loss attributable to common stockholders for the nine months ended September 30, 2016, which includes a one-time deemed dividend related to a charge of $49.5 million incurred as a result of the conversion feature of the Series B convertible preferred stock was $86.7 million, or ($1.64) per share, compared to GAAP net loss attributable to common stockholders of $19.3 million or ($0.44) per share for the nine months ended September 30, 2015. Non-GAAP net loss, which excludes amounts related to stock-based compensation and the non-cash deemed dividend for the nine months ended September 30, 2016 was $21.4 million, or ($0.40) per share, compared to non-GAAP net loss of $13.5 million or ($0.31) per share for the nine months ended September 30, 2015.

The Company believes that it is important for investors to understand these non-cash charges as they are materially impacting the quarterly loss and EPS calculations. See "Use of Non-GAAP Financial Measures" below for a description of the Company’s Non-GAAP Financial Measures. Reconciliation between certain GAAP and Non-GAAP measures is provided at the end of this press release.

GAAP and Non-GAAP expenses

GAAP research and development (R&D) expenses of $8.5 million for the quarter ended September 30, 2016 increased by $3.5 million compared to the quarter ended September 30, 2015. The increase in R&D expense is due to increased spending on clinical activities for LN-144. In addition, R&D-associated stock option expenses were $0.6 million for the three months ended September 30, 2016 and $1.8 million for the nine months ended September 30, 2016. Non-GAAP R&D expenses of $7.8 million for the quarter ended September 30, 2016 increased by $3.7 million, compared to $4.1 million for the quarter ended September 30, 2015.

GAAP general and administrative (G&A) expenses of $10.5 million increased by $7.8 million compared to the quarter ended September 30, 2015. Non-GAAP G&A expenses of $2.5 million for the quarter ended September 30, 2016 increased by $1.3 million, compared to $1.2 million for the quarter ended September 30, 2015.

Reconciliation between certain GAAP and Non-GAAP measures is provided at the end of this press release.

Use of Non-GAAP Financial Measures

This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses. These measures are not in accordance with, or an alternative to, generally accepted accounting principles, or GAAP, and may be different from non-GAAP financial measures used by other companies. The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release are: (i) the non-cash stock-based compensation expense which may fluctuate from period to period based on factors including the timing and accounting of grants for stock options and changes in the Company’s stock price which impacts the fair value of options granted, and (ii) the one-time non-cash deemed dividend related to the conversion feature of the Series B Preferred Stock. The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding various financial and business trends relating to our financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of our ongoing operating performance. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating operational performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. To the extent this release contains historical or future non-GAAP financial measures, the Company has also provided corresponding GAAP financial measures for comparative purposes. Reconciliation between certain GAAP and non-GAAP measures is provided at the end of this press release.

2016 Cash Expectations

Lion anticipates the ending cash, cash equivalents and short-term investments as of December 31, 2016, to be in excess of $164.0 million.

Upcoming Events & Presentations

Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 31st Annual Meeting & Associated Programs in National Harbor, Maryland, November 9-13, 2016
Piper Jaffray 28th Annual Health Care Conference, at the Lotte New York Palace hotel in New York City, November 30 at 2:30 p.m. ET