On November 9, 2017 OncoSec Medical Incorporated ("OncoSec") (NASDAQ:ONCS), a company developing DNA-based cancer immunotherapies, reported that it will present emerging data from its novel, multi-gene expression platform termed Polycistronic Interleukin-12 Immune Modulator (PIIM) (Press release, OncoSec Medical, NOV 9, 2017, View Source [SID1234521893]). The poster presentation entitled: "Intratumoral administration of a multigene construct by electroporation can effectively modulate anti-tumor response in a murine B16.F10 model" (P403) will be presented during the "Oncolytic Viruses and Intratumoral Therapies" session on Friday, November 10th at 12:30-2:00 p.m and 6:30-8:00 p.m. ET at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting 2017, in National Harbor, MD.

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The PIIM platform builds upon OncoSec’s existing plasmid-based cancer immunotherapy platform and may offer both enhanced therapeutic activity as well as manufacturing efficiencies. Preclinical data demonstrate the novel platform’s flexibility to add multiple immune modulating genes to alter the tumor microenvironment which, when combined with electroporation in a preclinical tumor animal model, resulted in regression of tumor growth in both the treated primary lesion and a distant untreated lesion.

"Immunotherapy has transformed the treatment of multiple cancers, including melanoma; however, current agents do not help all patients or have toxic side effects preventing broader use or in combination with other agents," said Dan O’Connor, CEO of OncoSec. "The ability to enhance response with a simultaneous and potentially safe combination therapy, including cytokines, checkpoint modulators, and antibodies, allows the immune system to better recognize and attack the tumor, an advantage of the PIIM platform versus other products currently under development."

The full abstract is available and can be viewed on the STIC website at www.sitcancer.org. The presentation is available in the Publications section of OncoSec’s website.

About the SITC (Free SITC Whitepaper) Annual Meeting
The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) is a non-profit medical professional society of influential scientists, academicians, researchers, clinicians, government representatives, and industry leaders from around the world dedicated to improving cancer patient outcomes by advancing the science and application of cancer immunotherapy. Currently, SITC (Free SITC Whitepaper) has nearly 1,600 members representing 17 medical specialties and are engaged in research and treatment of at least a dozen types of cancer. The 32nd SITC (Free SITC Whitepaper) Annual Meeting & Associated Programs will take place November 8-12, 2017 at the Gaylord National Hotel & Convention Center in National Harbor, MD. For more information, please go to View Source

About Polycistronic Interleukin-12 Immune Modulator (PIIM)
PIIM represents an advancement for OncoSec’s plasmid-based cancer immunotherapy platform, coupling improved in vivo electroporation with the ability to combine multiple therapeutic molecules in a single DNA plasmid. The PIIM technology platform offers expedited design and molecular cloning of these molecules, individually or fused, with tailored effector functions and relative expression levels. Delivery of multifunctional combinations from a single plasmid backbone may also simplify the manufacturing process.

On November 9, 2017 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported the highlights of numerous advances in understanding immune response and cancer immunotherapy using the nCounter platform that will be presented at the 32nd Annual Meeting of the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (Press release, Ligand, NOV 9, 2017, View Source [SID1234521879]).

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"The scope of nCounter-based research being presented at this year’s SITC (Free SITC Whitepaper) conference demonstrates our scientific and commercial momentum in immuno-oncology," said Alessandra Cesano, chief medical officer of NanoString. "In addition, several abstracts outline the unique capabilities of our Digital Spatial Profiling technology to characterize the tumor and its microenvironment to inform cancer research and drug development."

At least 45 abstracts using NanoString’s nCounter platform will be presented at the SITC (Free SITC Whitepaper) Annual Meeting, being held in National Harbor, Maryland, Nov. 8-12, 2017. The research being presented spans a wide breadth of applications including biomarker development, assessing the biology of immune responsiveness and resistance, and digital pathology. They include biomarker studies covering 21therapeutic agents, as single agents or in combination.

Nineteen studies used NanoString’s PanCancer Series of panels to explore biomarkers associated with response to immunotherapy. Fourteen of these studies incorporate NanoString’s best-selling PanCancer Immune Profiling Panel. An additional five studies incorporating both early access and commercial versions of NanoString’s new PanCancer IO 360 Panel. The PanCancer IO 360 Panel assays key pathways from the tumor, the microenvironment and the immune system and includes more than 20 signatures that are potentially associated with therapeutic response to novel therapeutic agents with "matched" mechanisms of action. Three additional studies incorporate the nCounter Hallmarks of Cancer suite of gene expression panels, which includes three panels covering Cancer Immune Profiling, Cancer Pathways, and Cancer Progression.

The PanCancer IO 360 Panel studies provide initial evidence of positive association between the Tumor Inflammation Signature scores and clinical response to different immuno-oncology agents including nivolumab, ipilimumab, pembrolizumab and entinostat. NanoString’s Tumor Inflammation Signature (TIS) was recently described by Ayers, et al. (View Source) and is included in the PanCancer IO360 panel. The Tumor Inflammation Signature measures the presence or absence of a peripherally suppressed adaptive immune response within the tumor.

Five studies cover the use of NanoString’s Digital Spatial Profiling (DSP) platform in immuno-oncology research. DSP allows for digital quantification of protein from discrete regions of FFPE tissue in an automated and multiplex format. DSP will become widely available with the launch of a new instrument planned for late 2018, and in the meantime is available under a Technology Access Program.

Two studies cover the use of 3D Biology panels and demonstrate the utility of the nCounter platform. NanoString’s 3-D Flow technology provides detailed molecular profiles of T cell populations, and enables unique, simultaneous analysis of high-plex protein and RNA data. 3D Biology and 3D Flow approaches can be used to characterize baseline immunological state and response to stimulation, which may be useful for profiling mechanisms of action or therapeutic response.

At the 2017 SITC (Free SITC Whitepaper) Annual Meeting, NanoString will showcase its nCounter platform, IO360 Data Analysis, Digital Spatial Profiling and 3D Biology capabilities at booth #605.

NanoString will host a Digital Spatial Profiling Educational Session on Nov. 11, 2017, 12:45 – 1:45 p.m.

Below is a summary of abstracts co-authored by NanoString employees:

Abstract # Title Hyperlink
05 A dendritic cell targeting NY-ESO-1 vaccine significantly augments early and durable immune responses in melanoma patients pretreated with human Flt-3 Ligand View Source

019 ENCORE-601: Phase 1b/2 study of entinostat (ENT) in combination with pembrolizumab (PEMBRO) in patients with non-small cell lung cancer (NSCLC) View Source

P40 Deep proteomic and transcriptomic analysis of sorted T cells with a simple, integrated workflow View Source

P43 Assessment of Pharmacodynamic Effects of Immuno-Oncology Agents in Cynomolgus Monkeys using High-Content Gene Expression Profiling View Source

P64 Analytical Validation of Digital Spatial Profiling – a novel approach for multiplexed characterization of protein distribution and abundance in FFPE tissue sections View Source

P65 Spatially-resolved, multiplexed digital characterization of protein abundance in FFPE tissue sections: application in preclinical mouse models View Source

P66 Digital spatial profiling platform allows both spatially-resolved, multiplexed measurement of solid tumor and immune-associated protein distribution and abundance using a single FFPE tissue section View Source

P72 Analysis of biomarkers from a cohort of advanced melanoma patients previously exposed to immune checkpoint inhibition treated with entinostat (ENT) and pembrolizumab (PEMBRO).

View Source
P73 First-in-human neoadjuvant study of the immunogenomic impact of the oral IDO inhibitor epacadostat (INCB024360) on the tumor microenvironment of advanced ovarian cancer View Source

P98 Immunological profiling of baseline and resected biopsies from locally/regionally advanced/recurrent melanoma treated with neoadjuvant combination ipilimumab (3mg/kg or 10mg/kg) and high dose IFN-α2B View Source

P99 Biomarker analysis from the OpACIN trial (Neo-/adjuvant ipilimumab + nivolumab (IPI+NIVO) in palpable stage 3 melanoma) View Source

P100 Pretreatment gene expression correlation with clinical response to pembrolizumab or nivolumab in metastatic melanoma View Source

P383 Molecular and immune characterization of melanoma metastases with heterogeneous PTEN expression View Source

P485 Use of the NanoString Gene Expression Profiling Platform to Capture the Immunological Status of the Leukemia Microenvironment View Source

P512 Deep immunoprofiling of rare T-cell populations from clinical samples View Source

P524 Clinical and biomarker analyses of a phase II study of intratumoral tavokinogene telseplasmid (pIL-12) plus pembrolizumab in stage III/IV melanoma patients predicted to not respond to anti-PD-1 View Source

Ophthotech has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Ophthotech, 2017, NOV 8, 2017, View Source [SID1234521770]).

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On November 8, 2017 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) announced today that Genentech Inc., a member of the Roche Group, obtained approval from the U.S. Food and Drug Administration (FDA), for Alecensa in the treatment of "anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC)" (Press release, Chugai, NOV 8, 2017, View Source [SID1234521712]). In addition to this approval, the FDA also converted Alecensa’s initial accelerated approval (given in December 2015) to a full approval for the treatment of people with ALK-positive, metastatic NSCLC who have progressed on or are intolerant to crizotinib (second-line).

"In July 2014, Alecensa obtained its first approval globally in Japan. With the goal of contributing to many patients in the world, the development of Alecensa was progressed with a focus on speed in each country," said Dr. Yasushi Ito, Chugai’s Senior Vice President, Head of Project & Lifecycle Management Unit. "The results of Phase III studies both in Japan and overseas showed that Alecensa could improve current treatment of ALK-positive NSCLC."

Alecensa received Breakthrough Therapy Designation from the FDA in September 2016 for the treatment of adults with advanced ALK-positive NSCLC who have not received prior treatment with an ALK inhibitor. Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat serious or life-threatening diseases and to help ensure people have access to them through FDA approval as soon as possible. Breakthrough Therapy designation was granted on the basis of the Japanese phase III J-ALEX study which was conducted prior to ALEX study.

The new approval is based on results from the phase III ALEX study.
The ALEX study evaluates the efficacy and safety of Alecensa compared with crizotinib in people with ALK-positive NSCLC who had not received prior systemic therapy (first-line). In the study, Alecensa significantly reduced the risk of disease worsening or death by 47% (HR=0.53, 95%CI: 0.38-0.73, stratified log-rank test, p<0.0001) compared to crizotinib as assessed by independent review committee. Median progression-free survival (PFS) was 25.7 months (95%CI: 19.9-not estimable) for people who received Alecensa compared with 10.4 months (95%CI: 7.7-14.6) for people who received crizotinib. The safety profile of both drugs was consistent with that observed in previous studies, with no new findings.

In addition, Alecensa significantly reduced the risk of the cancer spreading to or growing in the brain or central nervous system (CNS) compared to crizotinib by 84% (HR=0.16, 95%CI: 0.10-0.28, stratified log-rank test, p<0.0001). This was based on a time to CNS progression analysis in which there was a lower risk of progression in the CNS as the first site of disease progression for people who received Alecensa (12%) compared to people who received crizotinib (45%).

About Alecensa
Alecensa is a highly selective oral ALK inhibitor discovered by Chugai. It has been reported that approximately five percent of patients with NSCLC express a chromosomal rearrangement which leads to fusion of the ALK gene with another gene.1) ALK kinase signalling is constantly active in cells with such fusion genes, resulting in uncontrolled growth of tumour cells and transforming the cells into tumour cells.2, 3) Alecensa exerts its anti-tumour effect by selectively inhibiting ALK kinase activity to inhibit tumour cell proliferation and induce cell death.4) In addition, Alecensa is not recognized by the active efflux system in the blood brain barrier which actively pumps molecules out of the brain. Alecensa is able to remain active in the central nervous system and has proven activity against brain metastases.

Alecensa is currently approved in the United States, Europe, Kuwait, Israel, Hong Kong, Canada, South Korea, Switzerland, India, Australia, Singapore, Taiwan, Liechtenstein, Thailand, Argentina and Turkey for the treatment of people with metastatic (advanced) ALK-positive NSCLC whose disease has worsened after, or who could not tolerate treatment with, crizotinib. In Japan, Alecensa is available to patients with "ALK fusion gene positive unresectable, recurrent/advanced NSCLC" and is marketed by Chugai. The approved dosage and administration in Japan is "300mg alectinib administered orally twice daily for adult patient."

1) Biomarker committee of The Japan Lung Cancer Society, Guidelines for ALK gene tests in lung cancer patients
2) Soda et al., Nature. 448: 561-566 (2007)
3) Takeuchi et al., Clin Cancer Res. 15: 3143-3149 (2009)
4) Sakamoto et al., Cancer Cell. 19: 679-690 (2011)

Note: The dosage and administration of the ALEX study is "600mg alectinib administered orally twice daily," which is different from the Japanese dosage and administration.

On November 8, 2017 Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx, reported its financial results for the nine months ended September 30, 2017 and provided a corporate update (Press release, Protalix, NOV 8, 2017, View Source;p=RssLanding&cat=news&id=2315313 [SID1234521760]).

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"I am excited about the progress we made recently," said Moshe Manor, Protalix’s President and Chief Executive Officer. "We are no longer facing any debt issue or financing overhang. Moreover, with the recent strategic collaboration with Chiesi, not only did we secure a strong, experienced clinical and commercial partner, but we also meaningfully increased our capital resources, providing us with sufficient cash into 2020, irrespective of any milestone payments we are entitled to from Chiesi, increased revenues or from future partnerships. In addition, on the strategic front, we now have three novel and clinically differentiated product candidates in the clinic with potentially superior efficacy which provides us multiple shots at goal to realize significant value for our stockholders."

Third Quarter and Recent Clinical Highlights

Pegunigalsidase alfa (PRX-102) for Fabry Disease

Protalix entered into an EX-US collaboration agreement with Chiesi Farmaceutici S.p.A., or Chiesi, for pegunigalsidase alfa, or PRX-102. Under the terms of the agreement, Protalix is entitled to an upfront payment of $25 million from Chiesi, up to $25 million to cover development costs for pegunigalsidase alfa, subject to a maximum of $10 million per year, and up to an additional $320 million in regulatory and commercial milestone payments. Additionally, Protalix is entitled to tiered payments of 15% to 35% of Chiesi’s net sales.

Patient enrollment in the Company’s phase III clinical trials, referred to as the Balance, Bridge and Bright studies, is on-going.
Alidornase alfa (PRX-110) for Cystic Fibrosis

Protalix applied for a financial grant from the Cystic Fibrosis Foundation to support the clinical development of alidornase alfa.

A poster titled "Development of Novel Actin Inhibition Resistant DNase I Enzyme – alidornase alfa – for the Treatment of Cystic Fibrosis" was presented at the North American Cystic Fibrosis Conference held in November 2017.
Oral antiTNF (OPRX-106) for Ulcerative Colitis

The final patients needed to complete enrollment in the Company’s phase II clinical trial of OPRX-106 for the treatment of ulcerative colitis are in the screening process. The Company remains on track to complete enrollment during the fourth quarter and report top-line results in early 2018.
Alfataliglicerase for Gaucher Disease

Shipment of approximately $3.0 million of alfataliglicerase was completed this quarter for a total of $6.6 million for the nine months ended September 30, 2017.

According to the purchase order received by the Company, additional shipments are scheduled to be made during fourth quarter of 2017, and into 2018.
Financial Results for Nine Months ended September 30, 2017

The Company reported a net loss of $32.1 million, or $0.25 per share, basic and diluted, for the nine-month period ended September 30, 2017, excluding a one-time, non-cash net charge of $38.1 million in connection with the remeasurement of a derivative, compared to a net loss of $26.7 million, or $0.27 per share, basic and diluted, for the same period of 2016.

The Company recorded total revenues of $16.8 million for the nine-month period ended September 30, 2017, compared to $7.1 million during the same period of 2016. The increase is primarily the result of increased sales of drug product to Brazil of $6.6 million in the nine months ended September 30, 2017, compared to $2.7 million in the same period of 2016, and the sale of drug substance to Pfizer Inc.

Research and development expenses, net were $19.8 million for the nine-month period ended September 30, 2017, compared to $18.9 million for the same period of 2016. Selling, general and administrative expenses were $8.2 million for the nine-month period ended September 30, 2017, compared to $6.2 million incurred during the same period of 2016. The increases are primarily attributed to increased activities in three ongoing clinical trials and selling in Brazil.

During the nine-month period ended September 30, 2017 and during October, note holders converted the entire $8.55 million in aggregate principal amount of the Company’s 4.50% convertible notes due 2022.

As of today, the Company’s outstanding convertible notes include 4.50% convertible notes due September 2018 with an aggregate principal amount of $5.9 million and senior secured 7.50% convertible notes due November 2021 with an aggregate principal amount of $61.9 million.

On September 30, 2017, the Company had $33.4 million of cash and cash equivalents. With the Company’s current cash, plus the additional $25 million upfront payment due from Chiesi, and without giving effect to any milestone payment we are entitled to from Chiesi, anticipated increase in revenue run rate or any additional potential partnerships, the Company has sufficient resources to fund operations into 2020.
Conference Call and Webcast Information

The Company will host a conference call on Wednesday, November 8, 2017, at 8:30 am ET to review the clinical, corporate and financial highlights.

To participate in the conference call, please dial the following numbers prior to the start of the call: United States: (844) 358-6760; International: (478) 219-0004. Conference ID number 6767278.

The conference call will also be broadcast live and available for replay for two weeks on the Company’s website, www.protalix.com, in the Events Calendar of the Investors section. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.