On November 14, 2018 The Janssen Pharmaceutical Companies of Johnson & Johnson reported the submission of two Type II variation applications to the European Medicines Agency (EMA) seeking approval for the expanded use of IMBRUVICA (ibrutinib) (Press release, Johnson & Johnson, NOV 14, 2018, View Source [SID1234531316]). One application seeks to include use of ibrutinib in combination with obinutuzumab in previously untreated adults with chronic lymphocytic leukaemia (CLL) and to add long-term follow-up data from the existing label studies RESONATETM (PCYC-1112) and RESONATETM-2 (PCYC-1115). The second is for use of ibrutinib plus rituximab for the treatment of previously untreated and relapsed/refractory adults with Waldenström’s macroglobulinemia (WM).

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"Today’s news brings us one step closer to potentially offering ibrutinib in new combinations for patients where unmet needs still persist," said Dr. Catherine Taylor, Haematology Therapy Area Lead, Europe, Middle East and Africa (EMEA), Janssen-Cilag Limited. "Ibrutinib continues to demonstrate clinical benefit over the long term for a broad group of patients living with blood cancer, and we look forward to working with relevant authorities to secure approval of these new combinations."

Ibrutinib, a first-in-class Bruton’s tyrosine kinase (BTK) inhibitor, is jointly developed and commercialised by Janssen Biotech, Inc., and Pharmacyclics LLC, an AbbVie company.

The CLL submission is supported by positive results from the Phase 3 iLLUMINATE (PCYC-1130) study which investigated ibrutinib in combination with obinutuzumab versus chlorambucil plus obinutuzumab in patients with newly diagnosed CLL.1 Study findings from iLLUMINATE will also be featured as an oral presentation (abstract #691), whilst further analysis of RESONATETM and RESONATETM-2 results in comparison with real-world evidence databases (abstract #4427) will be included at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place in San Diego next month.1,2 A supplemental New Drug Application (sNDA) which was also recently submitted to the U.S. Food and Drug Administration (FDA) received Priority Review.

In WM, the submission is supported by data from the Phase 3 iNNOVATE (PCYC-1127) study evaluating ibrutinib in combination with rituximab, versus rituximab with placebo, in patients with previously untreated and relapsed/refractory WM.3 Follow-up efficacy and safety findings from the iNNOVATE study will also be presented at ASH (Free ASH Whitepaper) 2018 (abstract #149).4 In August 2018, the FDA approved ibrutinib in combination with rituximab for the treatment of WM based on the data from iNNOVATE.5

Additional information about both studies can be found at www.ClinicalTrials.gov (NCT02264574 and NCT02165397).6,7

#ENDS#

About ibrutinib

Ibrutinib is a first-in-class Bruton’s tyrosine kinase (BTK) inhibitor, which works by forming a strong covalent bond with BTK to block the transmission of cell survival signals within the malignant B-cells.8 By blocking this BTK protein, ibrutinib helps kill and reduce the number of cancer cells, thereby delaying progression of the cancer.9

Ibrutinib is currently approved in Europe for the following uses:10

Chronic lymphocytic leukaemia (CLL): As a single agent for the treatment of adult patients with previously untreated CLL, and as a single agent or in combination with bendamustine and rituximab (BR) for the treatment of adult patients with CLL who have received at least one prior therapy.
Mantle cell lymphoma (MCL): Adult patients with relapsed or refractory mantle cell MCL.
Waldenström’s macroglobulinemia (WM): Adult patients who have received at least one prior therapy or in first-line treatment for patients unsuitable for chemo-immunotherapy.
The most common adverse reactions seen with ibrutinib include diarrhoea, neutropenia, haemorrhage (e.g., bruising), musculoskeletal pain, nausea, rash, and pyrexia.10

For a full list of side effects and information on dosage and administration, contraindications and other precautions when using ibrutinib please refer to the Summary of Product Characteristics for further information.

About CLL

CLL is typically a slow-growing blood cancer of the white blood cells.11 The overall incidence of CLL in Europe is approximately 4.92 cases per 100,000 persons per year with rates amongst men and women approximately 5.87 and 4.01 cases per 100,000 persons per year, respectively.12 CLL is predominantly a disease of the elderly, with a median age of 72 years at diagnosis.13

CLL is a chronic disease; median overall survival ranges between 18 months and more than 10 years, according to the stage of disease.14 The disease eventually progresses in the majority of patients, and patients are faced with fewer treatment options with each relapse. Patients are often prescribed multiple lines of therapy as they relapse or become resistant to treatments.

About Waldenström’s macroglobulinemia

Waldenström’s macroglobulinemia (WM) is a rare form of non-Hodgkin’s lymphoma (NHL).15 It causes overproduction of a protein called monoclonal immunoglobulin M (IgM) antibody, which causes a thickening of the blood.16 Incidence rates among men and women in Europe are approximately 7.3 and 4.2 per million persons, respectively.17 The causes of WM are unknown, with it typically affecting older adults and being slightly more common in men than women.15,17

On November 14, 2018 Personalis, Inc., a leader in advanced genomics for precision oncology, reported that they are scheduled to present at the upcoming NeoAG Summit in Boston, MA on Thursday, November 15, 2018 at 4 PM ET (Press release, Personalis, NOV 14, 2018, View Source [SID1234531317]).

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The presentation, entitled "ImmunoID NeXT Platform: Improving Neoantigen Prediction, TME Assessment, and ctDNA Detection for Vaccine Development," will introduce Personalis’ new ImmunoID NeXT Platform and discuss how it facilitates highly accurate neoantigen identification, optimal MHC-binding prediction and ranking, and the assessment of the tumor microenvironment and tumor escape mechanisms that may impact response to personalized therapeutics.

ImmunoID NeXT is the first and only platform to provide comprehensive analysis of both a tumor and its microenvironment from a single sample. The platform can be used to investigate the key tumor- and immune-related areas of cancer biology; consolidating multiple oncology biomarker assays into one. This maximizes the biological information that can be generated from a precious tumor specimen.

The presentation will be delivered by Sean Boyle, PhD; Director, Bioinformatics Applications.

On November 13, 2018 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a biotechnology company developing novel cancer immunotherapies based on tumor-infiltrating lymphocyte (TIL) technology, reported that company management will present and participate in the following conferences in November (Press release, Iovance Biotherapeutics, NOV 13, 2018, View Source;p=irol-newsArticle&ID=2376890 [SID1234531249]):

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Credit Suisse 27th Annual Healthcare Conference in Scottsdale, AZ on Tuesday, November 13, 2018.

Jefferies Global Healthcare Conference in London, United Kingdom on Thursday, November 15, 2018 at 4:00 p.m. GMT.

Evercore ISI HealthCONx Conference in Boston, MA on Tuesday, November 27, 2018 at 11:20 a.m. ET.
A live audio webcast of the Jefferies and Evercore ISI conference presentations will be available by visiting the Investors section of Iovance Biotherapeutics’ website at View Source A replay of the webcasts will be archived on Iovance Biotherapeutics’ website for 30 days following the presentations.

On November 13, 2018 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a company focused on the
development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell ("CAR T")
technology and gene therapies for rare diseases, reported financial results and recent corporate highlights for the third quarter ended September 30, 2018 (Press release, Mustang Bio, NOV 13, 2018, View Source [SID1234531346]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "The third quarter of 2018 and recent months have been marked by clinical progress and a key addition to our management team with the appointment of Martina A. Sersch, M.D., Ph.D., as Chief Medical Officer. Notably, we expanded our pipeline into gene therapy by securing an exclusive worldwide license agreement with St. Jude Children’s Research Hospital ("St. Jude") for the development of a first-in-class, ex vivo, clinical-stage lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("X-SCID"). Data from a multicenter Phase 1/2 trial led by St. Jude in infants under the age of two years old are extremely encouraging. Eight patients under the age of two with X-SCID have been treated to date, with results presented at the 21st Annual Meeting of the American Society of Gene & Cell Therapy in May 2018. The therapy was well tolerated. In addition, six patients achieved reconstituted immune systems within three to four months following treatment, with the remaining two patients continuing to progress favorably in earlier stages of recovery. Four of these six patients have discontinued monthly infusions of intravenous immunoglobulin, and the remaining patients, at earlier stages of recovery, continue to progress favorably. In three patients who had disseminated infections prior to therapy, all infections resolved completely. In addition, the therapy is being investigated in patients over the age of two in a second Phase 1/2 trial at the National Institutes of Health ("NIH"), with equally encouraging data and an excellent safety profile to date. The two patients with the longest follow-up have seen sustained restoration of antibody production after immunization, and all five patients treated experienced a decrease in viral infections and overall clinical improvement."

Dr. Litchman continued, "We also recently announced updates on two Phase 1 clinical trials at City of Hope using our
HER2-specific CAR T cell therapy, including a first-of-its-kind trial using intraventricular delivery of CAR T cells to brains of patients with HER2-positive breast cancer with brain metastases. As we look ahead to the fourth quarter of 2018, we look forward to filing our first Investigational New Drug (IND) application to support a Phase 1/2 trial of MB-102 in acute myeloid leukemia ("AML"), blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic syndrome."

Recent Corporate Highlights:
• In July 2018, Mustang completed a pre-IND meeting with the U.S. Food and Drug Administration ("FDA") for MB102 (CD123 CAR T). Based on the meeting, Mustang expects to file an IND in the fourth quarter of 2018 to support a Phase 1/2 trial of MB-102 in AML, blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic
syndrome.
• In August 2018, Mustang announced that it entered into an exclusive worldwide license agreement with St. Jude
for the development of a first-in-class ex vivo lentiviral gene therapy for the treatment of X-SCID, also known as
bubble boy disease. The therapy is currently being evaluated in a Phase 1/2 multicenter trial in infants under the
age of two. This study is the world’s first lentiviral gene therapy trial for infants with X-SCID. The therapy is also
being investigated in patients over the age of two in a second Phase 1/2 trial at the National Institutes of Health
("NIH").
• In October 2018, Mustang appointed Martina A. Sersch, M.D., Ph.D., as Chief Medical Officer.
• Also in October 2018, Mustang announced that a first-of-its-kind Phase 1 clinical trial evaluating the safety and
effectiveness of intraventricular delivery of CAR T cells to the brains of patients with HER2-positive breast cancer
with brain metastases has been initiated at City of Hope. In addition, Mustang announced that City of Hope dosed
the first patient in a Phase 1 clinical trial of HER2-specific CAR T cells in treating recurrent or refractory grade IIIIV
glioma. The trial will evaluate the side effects and best dose of HER2-specific CAR T cells in treating patients
with grade III-IV glioma that has come back or does not respond to treatment.
Financial Results:
• As of September 30, 2018, Mustang’s consolidated cash, cash equivalents, short-term investments (certificates of
deposit) and restricted cash totaled $41.3 million, compared to $47.2 million as of June 30, 2018, and $61.5 million
as of December 31, 2017, a decrease of $5.9 million for the quarter and a decrease of $20.2 million year-to-date.
• Research and development expenses were $5.3 million for the third quarter of 2018, compared to $2.2 million for
the third quarter of 2017. Non-cash, stock-based compensation expenses included in research and development
were $0.7 million for third quarter of 2018, compared to $0.3 million for the third quarter of 2017.
• Research and development expenses from license acquisitions were $1.0 million for the third quarter of 2018,
compared to $0.3 million for the third quarter of 2017.
• General and administrative expenses were $1.3 million for the third quarter of 2018, compared to $4.6 million for
the third quarter of 2017. Non-cash, stock-based compensation expenses included in general and administrative
expenses were $0.2 million for the third quarter of 2018, compared to $0.5 million for the third quarter of 2017.
• Net loss attributable to common stockholders was $7.5 million, or $0.28 per share, for the third quarter of 2018,
compared to $6.9 million, or $0.27 per share, for the third quarter of 2017. Net loss attributable to common
stockholders was $18.9 million, or $0.70 per share, for the first nine months of 2018, compared to $15.7 million,
or $0.63 per share, for the first nine months of 2017.

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.