On November 13 2018 FUJIFILM Corporation (President: Kenji Sukeno) reported that it has succeeded in stably encapsulating the anti-cancer agent topotecan*, approved for the treatment of several solid cancers, in newly invented liposome (Press release, Fujifilm, NOV 13, 2018, View Source [SID1234538525]). When preclinical studies in mice were conducted using a drug where topotecan was encapsulated in liposomes (development number: FF-10850), in addition to confirming high tumor shrinking effects, improved pharmacological efficacy was demonstrated when administered in combination with an immune checkpoint inhibitor**, with prolonged survival compared to monotherapy. Going forward, based on the results of these nonclinical studies, the company will prepare for the early initiation of clinical studies of the drug candidate.

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Fujifilm conducts research and development on the potential use of encapsulating drugs in liposomes, artificially constructed vesicles made from organic phospholipids that make up cellular membranes, by harnessing its advanced nano-dispersion technology, analysis technology, and process technology cultivated through its wide range of product development. In another study the liposome drug candidate FF-10832, which encapsulates the anti-cancer agent gemcitabine*** , has been observed to protect gemcitabine from elimination and demonstrate an enhanced permeability and retention (EPR) effect*4 by which the FF-10850 accumulates within tumor and releases gemcitabine. A Phase I clinical trial of FF-10832 is underway in the U.S.
Topotecan has an extremely short half-life*5 in the blood, and also has the issue of causing serious bone marrow suppression*6 as a side effect in more than 80% of patients. A liposome drug where topotecan is encapsulated can be considered as a way to resolve these issues; however, topotecan has a tendency to pass through the liposome membrane, resulting in a problem where topotecan would leak into the blood before reaching the tumor. By enhancing the strength of the liposome membrane with the addition of new materials to the liposome ingredients, Fujifilm has succeeded in stably encupsulating topotecan. Fujifilm is conducting research on the drug as a candidate anti-cancer agent (development number: FF-10850), and has obtained the following results in studies in mice.

[Research Result 1] High tumor shrinking effects confirmed with monotherapy

1) Experiment:
Mice with transplanted human-derived ovarian cancer cells (ES-2) were administered with topotecan and FF-10850, respectively, and the efficacy and tolerability were confirmed for each dosage. The period of administration was five consecutive days for topotecan and two cycles of one administration per week for FF-10850.
2) Results:
With topotecan, efficacy was observed with 30 mg/m2 (6 mg/m2 x five times), while with FF-10850, efficacy was confirmed with 3 mg/m2 (1.5 mg/m2 x twice). When the two drugs were compared, FF-10850 demonstrated efficacy that was greater than or equal to topotecan with 1/10 of the total dose of topotecan.
With FF-10850, tumor shrinking effects were seen with 8 mg/m2 (4 mg/m2 x twice). Furthermore, the relative body weight change which is an indicator of tolerability, was less than that of topotecan.
[Chart] Tumor volume and relative body weight change upon administration of FF-10850 and topotecan
Based on the above, FF-10850 is expected to have high pharmacological efficacy that exceeds that of topotecan while maintaining tolerability even when dosage is increased.

[Research Result 2] Improved pharmacological efficacy observed when administered in combination with an immune checkpoint inhibitor

1) Experiment:
Monotherapies and combination therapy with an immune checkpoint inhibitor** and FF-10850 was conducted on mice transplanted with mouse-derived colorectal cancer cells (CT26), and efficacy and tolerability were confirmed. The period of administration was three cycles of two administrations per week for the immune checkpoint inhibitor (30 mg/m2) and three cycles of one administration per week for FF-10850 (6 mg/m2).
2) Results:
When administered as a monotherapy, the median value for mouse survival time was 19 days for the immune checkpoint inhibitor and 27.5 days for FF-10850. Meanwhile, when administered as a combination therapy, the median value for mouse survival time exceeded 40 days, demonstrating a statistically significant difference when compared with the monotherapies. Mouse survival rates for the combination therapy were 75% at 40 days after administration, which was higher than the monotherapies.
Even when administered as a combination therapy, noticeable side effects such as weight loss were not observed, and there were no issues with tolerability.
[Chart] Survival rates and median survival time when administered in combination with an immune checkpoint inhibitor
Based on the above, FF-10850 is expected to further suppress tumor proliferation through administration with an immune checkpoint inhibitor and prolong survival time.

Fujifilm will present its research results on FF-10850 at the "30th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) SYMPOSIUM", one of the world’s most prominent cancer related conferences, to be held in Dublin, Ireland from November 13th to 16th, 2018.

Fujifilm is harnessing its advanced technologies such as the nano-dispersion technology and ability to synthesize and design compounds to undertake the development of new drugs in the key areas of cancer, central nervous system diseases, and infectious diseases. The company is also focusing on developing drug delivery system (DDS) technologies including liposome drugs, undertaking research and development to apply DDS technologies not only to low-molecular-weight drugs but also to next-generation drugs such as nucleic acid drugs and gene therapy drugs. Going forward, the company will contribute to the resolution of social issues by developing and delivering innovative, high value-added pharmaceutical products.

* An anti-cancer agent (generic name: topotecan, product name: Hycamtin) developed by GlaxoSmithKline plc. Currently, the drug is distributed by Novartis. It is used as a treatment for ovarian cancer, small-cell lung cancer, and cervical cancer.
** The general term for drugs that have an effect by enabling activated immune cells to attack cancer cells by inhibiting the mechanism (immune checkpoints) that weakens the action of immune cells. Widely used in the treatment of malignant melanomas, lung cancer, stomach cancer, and kidney cancer. The immune checkpoint inhibitor used in the combination therapy with FF-10850 was an anti-PD-1 antibody.
*** An anti-cancer drug (generic name: gemcitabine, product name: Gemzar) developed by the U.S. company Eli Lilly and Company. It is used as a drug of first choice for the treatment of pancreatic cancer, and is also indicated for the treatment of a wide range of other cancers (such as lung cancer and ovarian cancer).
*4 As they grow, tumors generate surrounding blood vessels, but these newly generated blood vessels are not fully developed and have large gaps that are much smaller in normal blood vessels. When liposomes and polymers are retained within the blood, they do not permeate the walls of normal blood vessels, which have small gaps, permeating only the vascular walls around the tumor. In addition, as lymphatic vessels are not fully developed in tumors, the liposomes and polymers that have permeated are not easily eliminated, resulting in the accumulation of these liposomes and polymers in the tumor. This is called the EPR (enhanced permeability and retention) effect.
*5 The time required for the concentration of a drug in the blood to be reduced to half.
*6 The state where production of white blood cells, platelets, and red blood cells in the bone marrow is reduced, leading to increased risk of infection and bleeding and symptoms such as anemia.

On November 13, 2018 SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company delivering innovative therapies for difficult-to-treat and often life-threatening infections, reported financial results for the quarter ended September 30, 2018, and provided an update on recent clinical and operational developments (Press release, Scynexis, NOV 13, 2018, View Source [SID1234531250]).

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Key Messages:

Women suffering from vaginal yeast infections (vulvovaginal candidiasis or VVC) have only one approved oral treatment option. With SCYNEXIS’s U.S. Food and Drug Administration (FDA)-endorsed Phase 3 registration program for VVC on track to begin by year-end, and with a potential New Drug Application (NDA) filing planned for 2020, ibrexafungerp may become the much-needed oral alternative for the millions of women who are not well-served by fluconazole.

Despite existing therapies, mortality associated with invasive aspergillosis remains as high as 50%. Based on positive pre-clinical data, an ibrexafungerp combination regimen has the potential to show superiority to the current standard of care. SCYNEXIS has initiated a Phase 2 study as a proof of concept in this indication.

Patients with no therapeutic options for invasive fungal infections continue to enroll in SCYNEXIS’s ibrexafungerp programs designed for such refractory infections, including infections caused by Candida auris, a multidrug-resistant pathogen that is the subject of recent warnings by the Centers for Disease Control and Prevention (CDC) and other health authorities.

As of September 30, 2018, SCYNEXIS has $49.5 million in cash and cash equivalents and short-term investments, adequate to fund activities into 2020.
"With our successful End-of-Phase 2 Meeting with the FDA, we continue to advance toward our goal of bringing ibrexafungerp to patients in need," said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. "Following FDA’s agreement with our proposed overall design of the Phase 3 registration program for VVC, we anticipate that results showing superiority of ibrexafungerp over placebo would lead to filing an NDA in 2020 for the treatment of VVC, followed by a supplemental NDA in 2021 for the prevention of recurrent VVC."

Dr. Taglietti continued: "In parallel, we are advancing the development of ibrexafungerp for severe, invasive indications. We continue to enroll patients in our FURI study for the treatment of refractory infections; we recently dosed the first patient in our CARES study for the treatment of high-mortality Candida auris infections and we initiated our Phase 2 combination study for the treatment of invasive aspergillosis. We remain committed to maximizing the clinical utility of ibrexafungerp, a first-in-class therapy that combines the broad-spectrum antifungal activity and safety profile of the echinocandin class with the oral convenience of the azole class. With a cash runway into 2020, we are well-funded to progress all ongoing programs."

Ibrexafungerp (formerly SCY-078) Update

VVC Phase 3 Registration Program on Track for Initiation by End of 2018, with Potential NDA Filing in 2020.
In October 2018, SCYNEXIS announced the successful completion of an End-of-Phase 2 Meeting with the FDA for VVC. The FDA has agreed with the SCYNEXIS’s proposed design of the Phase 3 registration program to support approval of oral ibrexafungerp for the treatment of VVC and the prevention of recurrent VVC. SCYNEXIS anticipates initiating the program by the end of 2018.
If approved, ibrexafungerp would provide a much-needed oral option for the millions of women who are currently not well-served by existing VVC therapies. Fluconazole, approved over 25 years ago, is the only oral treatment available and fails to adequately address several patient segments: fluconazole-failure patients, management of VVC during pregnancy, moderate-to-severe VVC, recurrent VVC and VVC caused by fluconazole-resistant Candida spp.

Continued Progress on SCYNEXIS’s Strategy to Expand the Use of Ibrexafungerp in Severe Invasive Fungal Infections.
In October 2018, SCYNEXIS dosed the first patient in the CARES study, a Phase 3, multi-center (U.S. and India), open-label, single-arm study evaluating the efficacy, safety and tolerability of oral ibrexafungerp for the treatment of Candida auris infections. C. auris is an emerging life-threatening and multidrug-resistant fungal pathogen, with a mortality rate of up to 60%.
The FURI study, evaluating oral ibrexafungerp for the treatment of fungal infections refractory or resistant to standard of care, is ongoing with a preliminary data review planned by the end of the year.
SCYNEXIS recently initiated a Phase 2 combination study of oral ibrexafungerp in invasive aspergillosis. An animal model of pulmonary aspergillosis showed improved outcomes and survival rates, supporting the potential superiority of ibrexafungerp in combination with azole therapy versus the standard of care.
Ibrexafungerp is well-positioned to address significant unmet needs in patients suffering from severe invasive fungal infections. It is a first-in-class therapy, with broad-spectrum activity (including against multidrug-resistant strains), fungicidal activity versus Candida, high tissue penetration and a favorable safety profile. These features, available in the convenience of an oral formulation, differentiate ibrexafungerp from available alternatives, and, if approved, would make it an attractive option for the treatment of severe invasive fungal infections.

Presentation at the 2018 ESCMID/ASM Conference on Drug Development. In September 2018, SCYNEXIS presented a poster at the 2018 European Society of Clinical Microbiology and Infectious Diseases (ESCMID)/American Society for Microbiology (ASM) Conference on Drug Development to Meet the Challenge of Antimicrobial Resistance. The poster, titled "Ibrexafungerp (formerly SCY-078) Displays Potent In Vitro Activity Against C. Glabrata Isolates with Mutations in fks Genes," describes the results of several pre-clinical studies designed to evaluate the in vitro activity of ibrexafungerp in Candida glabrata strains with fks mutations, which are often contributory in the development of echinocandin resistance. C. glabrata is the second-most common fungal species isolated from blood in the US and one of the most common fungal pathogens worldwide.
Third Quarter 2018 Financial Results
Cash, cash equivalents and short-term investments totaled $49.5 million as of September 30, 2018, with net working capital of $39.1 million.

Research and development expenses decreased to $3.9 million in the third quarter of 2018, compared to $4.5 million in the third quarter of 2017. The decrease of $0.5 million, or 12%, for the three months ended September 30, 2018, was primarily driven by a decrease of $0.8 million in preclinical development expense and a decrease of $0.2 million in consulting expense and was offset in part by an increase of $0.5 million in chemistry, manufacturing, and controls.

Selling, general and administrative expenses increased to $2.4 million in the third quarter of 2018, compared with $2.0 million in the third quarter of 2017. The increase of $0.4 million, or 21%, for the three months ended September 30, 2018, was primarily driven by a $0.2 million charge for deferred offering costs recognized during the three months ended September 30, 2018.

Total other income increased to $6.7 million in the third quarter of 2018 due to a $6.9 million non-cash gain recorded on the fair value adjustment of the warrant liabilities.

Net income for the third quarter of 2018 was $0.4 million, or $0.01 per share. This compares with a net loss for the third quarter of 2017 of $8.4 million, or $0.31 per share.

About Ibrexafungerp (formerly SCY-078)
Ibrexafungerp [pronounced eye-BREX-ah-FUN-jerp] is an investigational antifungal agent and the first representative of a novel class of structurally-distinct glucan synthase inhibitors, triterpenoids. This agent combines the well-established activity of glucan synthase inhibitors with the potential flexibility of having oral and IV formulations. Ibrexafungerp is currently in development for the treatment of fungal infections caused primarily by Candida (including C. auris) and Aspergillus species. It has demonstrated broad spectrum antifungal activity, in vitro and in vivo, against multidrug-resistant pathogens, including azole- and echinocandin-resistant strains. The FDA has granted Qualified Infectious Disease Product (QIDP) and Fast Track designations for the formulations of ibrexafungerp for the indications of IC (including candidemia), IA and VVC, and has granted Orphan Drug Designation for the IC and IA indications.

On November 13, 2018 IntelGenx Technologies Corp. (TSX-V:IGX) (OTCQX:IGXT) (the "Company" or "IntelGenx") reported the closing of Tilray, Inc.’s strategic investment in IntelGenx by way of private placement (the "Private Placement") (Press release, IntelGenx, NOV 13, 2018, View Source [SID1234531303]). Pursuant to the Private Placement, the Company issued 1,428,571 common shares ("Common Shares") at a subscription price of U.S.$0.70 per Common Share for gross proceeds of U.S.$1,000,000. The Corporation intends to use the proceeds of the Private Placement for cannabis-infused VersaFilm product development under its previously announced definitive license, development and supply agreement (the "Agreement") with Tilray Inc.

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The Common Shares issued to Tilray, Inc. are subject to a four-month statutory hold period which expires March 14, 2019. Additionally, under the Private Placement purchase agreement, Tilray, Inc. agreed that until May 14, 2019 it would not cause or permit any transfer of any Common Shares.

On November 13, 2018 RadioMedix Inc. (a Texas-based clinical stage biotechnology company) and Orano Med (a nuclear biotechnology company) reported that the United States Food & Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AlphaMedixTM for the treatment of neuroendocrine tumors (NETs) (Press release, RadioMedix, NOV 13, 2018, View Source [SID1234531347]). AlphaMedixTM is a 212Pb-labeled somatostatin analogue used for the targeted alpha therapy (TAT) of neuroendocrine tumors (NETs).

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"TAT has brought new hope to our patients with cancer, and the range of available therapies in this area will only increase in the near future. We are pleased to conduct the first TAT clinical trial in the U.S. for neuroendocrine cancers, with the aim of registration of the drug," said Dr. Ebrahim S. Delpassand, CEO of RadioMedix and Medical Director of the clinical studies. "We are hoping that AlphaMedixTM will provide a breakthrough therapy that will benefit our patients soon," added Dr. Delpassand. "We are excited about the progress of our dose escalation studies of AlphaMedixTM", echoed Dr. Izabela Tworowska, Ph.D., CSO of RadioMedix.

The mechanism of action of AlphaMedixTM relies on the delivery of a cytotoxic load of alpha-emitters to SSTR-overexpressing neuroendocrine tumors. Alpha- emitters are highly energetic radioactive particles that can cause irreversible damage to the cancer cells. RadioMedix and Orano Med initiated the Phase 1 clinical trial of the safety and preliminary efficacy of AlphaMedixTM in February of 2018 (NCT03466216); the study is currently recruiting patients at the Excel Diagnostics and Nuclear Oncology Center in Houston, TX U.S.

"FDA’s decision to grant Orphan drug Designation is an important regulatory milestone for AlphaMedixTM, a key program in our pipeline of 212Pb-labeled compounds," said Julien Dodet, CEO of Orano Med. "We believe TAT with 212Pb represents a promising new approach and that AlphaMedixTM has the potential to have a meaningful impact on patients".

Orphan designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. The designation provides certain benefits to drug developers such as seven-year marketing exclusivity upon FDA approval, tax credits for qualified clinical testing or prescription drug user fee exemption.

About Neuroendocrine Tumors

Neuroendocrine tumors (NETs) are a heterogeneous group of rare neoplasms that originate from neuroendocrine cells. These neoplasms occur mostly in the gastrointestinal tract and pancreas, but can also occur in other tissues including thymus, lung, and other uncommon sites such as ovaries, heart, and prostate. Most NETs strongly express somatostatin receptors (SSTRs).

About AlphaMedixTM

AlphaMedixTM is a radiolabeled SSTR-targeting therapeutic investigational drug for the treatment of NETs patients. The product consists of SSTR-targeting peptide complex radiolabeled with 212Pb and serve as an in vivo generator of alpha-emitting particles. 212Pb isotope is particularly suitable for SSTR therapy applications based upon its half-life, energy and decay properties.

On November 13, 2018 BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for multiple cancer indications, reported its results for the third quarter 2018 (Press release, BerGenBio, NOV 13, 2018, View Source [SID1234531234]). A presentation of the results by the Company’s management will take place today at 10.00 am CET in Oslo – details below.

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Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "We are pleased with the progress of our phase II clinical development programme with our selective AXL inhibitor bemcentinib, particularly in NSCLC. The observed correlation between patient response and positive AXL status in our combination trial with KEYTRUDA gives us confidence in bemcentinib’s proposed mode of action and its broad appeal as a promising new agent treating aggressive cancer. The next six to nine months will be an exciting time for the company as we anticipate further clinical data from our phase II trials, particularly in NSCLC and acute myeloid leukaemia."

Highlights – Third Quarter & 2018
Bemcentinib/KEYTRUDA combination in advanced non-small cell lung cancer (NSCLC) delivers highly promising phase II clinical results

40% overall response rate & 70% clinical benefit rate observed in AXL-positive, previously treated NSCLC patients, including PD-L1 negative patients (data presented at World Conference on Lung Cancer (WCLC))
5.9 months median progression-free-survival (PFS) in AXL positive vs. 3.3 months in AXL negative patients presented as late breaking abstract at Society Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) congress (SITC, post-period)
Efficacy endpoint met and stage two of the trial actively enrolling
Additional lung cancer phase II trials show promise for bemcentinib in combination with chemo- and targeted agents

Median PFS of NSCLC patients receiving the bemcentinib/TARCEVA combination in first line has surpassed that of TARCEVA monotherapy (data presented at WCLC)
Encouraging efficacy reported for bemcentinib in combination with docetaxel chemotherapy in patients who had exhausted all available therapy options (data presented at WCLC)
Tissue- and blood-based biomarkers with potential for development as companion diagnostics

Novel biomarkers identified and qualified across multiple clinical trials with bemcentinib, presented as poster discussion at European
Society for Medical Oncology meeting (ESMO) (Free ESMO Whitepaper) and SITC (Free SITC Whitepaper) 2018 (post period)
Arbitration process with Rigel Pharma Inc.

The Company is seeking clarification of interpretation and application of certain provisions of the 2011 bemcentinib license agreement
Anticipated data and news flow in the coming months

AML and MDS mono- and combination therapy at ASH (Free ASH Whitepaper) 2018
Stage 2 of bemcentinib/KEYTRUDA combination trial in H1 2019
IND filed for BGB149, first in class AXL function blocking antibody (post period)
Financial Highlights (Figures in brackets = same period 2017 unless otherwise stated)

Total operating expenses for the third quarter were NOK 38.1 million (NOK 36.6 million). Total operating expenses for the first nine months of 2018 amounted to NOK 143.6 million (NOK 136.2 million)
Research and development expenses accounted for 75.3 % of total operating expenses in Q3, and 72.3 % for the first nine months of 2018
Comprehensive loss for the third quarter amounted to NOK 37.7 million (loss of NOK 35.4 million). Comprehensive loss for the first nine months of 2018 was NOK 140.7 million (loss of NOK 134.6 million)
Cash and cash equivalents amounted to NOK 398.2 million at the end of September 2018 (NOK 440.3 million at 30 June 2018 and NOK 370.3 million at 31 December 2017)
Presentation and Webcast Details

A presentation by BerGenBio’s senior management team will take place at 10.00 am CET at:

Hotel Continental, Stortingsgaten 24/26, 0117 Oslo

The presentation will webcast live and the link will be available at www.bergenbio.com in the section Investors/ Financial Reports. A recording will be available shortly after the webcast has finished.

The results report and the presentation will be available at www.bergenbio.com in the section: Investors/ Financial Reports from 7:00 am CET the same day.

END

About AXL
AXL kinase is a cell membrane receptor and an essential mediator of the biological mechanisms that drive aggressive and life-threatening diseases. In cancer, AXL drives tumour survival, treatment resistance and spread, as well as suppressing the body’s immune response to tumours. AXL expression has been established as a negative prognostic factor in many cancers. AXL inhibitors, therefore, have potential value at the centre of cancer combination therapy, addressing significant unmet medical needs and multiple high-value market opportunities