On November 14, 2019 VBL Therapeutics (Nasdaq: VBLT) reported financial results for the third quarter ended September 30, 2019, and provided a corporate update (Press release, VBL Therapeutics, NOV 14, 2019, View Source [SID1234551274]).

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"Our OVAL Phase 3 potential-registration trial of VB-111 in ovarian cancer is progressing well and we look forward to an important interim analysis expected in the first quarter of 2020," said Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics. "The interim analysis will be based on CA-125 response, a disease marker which correlated with survival benefit in our Phase 2 study in platinum-resistant ovarian cancer patients. We also look forward to the launch of an investigator-sponsored trial of VB-111 in recurrent GBM and an NCI-sponsored trial in colon cancer, expected before the end of 2019."

Third Quarter and Recent Corporate Highlights:

An investigational new drug (IND) application for an investigator-sponsored Phase 2 trial of VB-111 in recurrent glioblastoma ("rGBM") went into effect with the U.S. Food and Drug Administration (FDA). The IND was submitted by Dana-Farber Cancer Institute, on behalf of a group of top neuro-oncology US medical centers. Further details on the trial will be presented at the upcoming Annual Meeting and Education Day of the Society for Neuro-Oncology, to take place November 20 – 24, 2019 at the Marriott Desert Ridge Hotel, Phoenix, Arizona.

An NCI-sponsored trial of VB-111 in colon cancer is expected to begin by year-end 2019. Based on the potential of VB-111 to turn immunologically "cold" tumors "hot", as seen in ovarian cancer biopsies, the new study will evaluate for the first time a combination of VB-111 and a checkpoint inhibitor in colon cancer, a cold tumor in which checkpoints inhibitor are ineffective for the vast majority of patients.

VBL’s new gene therapy pharmaceutical grade manufacturing facility in Modiin, Israel, that was established to support the commercial supply of VB-111 for the first indication, was certified by a European Union (EU) Qualified Person (QP) as being in compliance with EU Good Manufacturing Practices (GMP). This important step is expected to support future commercialization of VB-111, if approved.

VBL continues to advance the development of lead MOSPD2 antibodies towards IND applications in inflammation and in oncology, expected by year-end 2020. The Company has signed a service agreement with Thermo Fisher Scientific, one of the leading vendors in the antibody field, for production of lead candidate VB-601 for toxicology and clinical development.
Third Quarter ended September 30, 2019 Financial Results:

Cash Position: At September 30, 2019, the Company had cash, cash equivalents, short-term bank deposits and restricted bank deposits totaling $41.1 million and working capital of $34.5 million. The Company expects that its cash, cash equivalents and short-term bank deposits will enable it to fund operating expenses and capital expenditure requirements for approximately two years.

Revenues: Revenues related to VBL’s collaborations were $0.1 million in the third quarter of 2019.

R&D Expenses: Research and development expenses, net, after government grants, were approximately $3.8 million for the quarter ended September 30, 2019, compared to approximately $4.1 million in the same period in 2018.

G&A Expenses: General and administrative expenses for the quarter ended September 30, 2019 were $1.2 million, compared to approximately $1.4 million in the same period in 2018.

Comprehensive Loss: VBL reported a net loss for the quarter ended September 30, 2019 of $4.9 million, or ($0.14) per share, compared to a net loss of $5.4 million, or ($0.15) per share, in the quarter ended September 30, 2018.
For further details on VBL’s financials, please refer to Form 6-K filed with the SEC.

Conference Call: Thursday, November 14th @ 8:30am Eastern Time

From the US: 877-407-9208
International: 201-493-6784
Conference ID: 13696234
Webcast: View Source

On November 14, 2019 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported that an overview of the company’s business strategy and development-stage programs will be given at the Jefferies 2019 London Healthcare Conference being held in London (Press release, Spectrum Pharmaceuticals, NOV 14, 2019, http://investor.sppirx.com/news-releases/news-release-details/spectrum-pharmaceuticals-present-corporate-update-jefferies-3 [SID1234551273]). The company presentation is on Thursday, November 21, 2019, at 7:20 AM GMT.

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A live webcast of Spectrum’s presentation will be available at View Source

On November 14, 2019 AstraZeneca and MSD Inc., Kenilworth, N.J., US (MSD: known as Merck & Co., Inc. inside the US and Canada) reported that the US Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) and granted Priority Review for selumetinib as a potential new medicine for paediatric patients aged three years and older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PNs) (Press release, AstraZeneca, NOV 14, 2019, View Source [SID1234551243]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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This is the first acceptance of a regulatory submission for an oral monotherapy for the treatment of NF1, a rare and incurable genetic condition. A Prescription Drug User Fee Act (PDUFA) date is set for the second quarter of 2020.

The regulatory submission was based on positive results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored SPRINT Phase II Stratum 1 trial. An Objective Response Rate (ORR) was achieved in 66% of paediatric patients with NF1 and symptomatic, inoperable PNs (n=33/50 patients) when treated with selumetinib as a twice-daily oral monotherapy. ORR was defined as the percentage of patients with a confirmed complete or partial response of ≥ 20% tumour volume reduction.

Selumetinib, a MEK 1/2 inhibitor was granted US FDA Breakthrough Therapy Designation in April 2019, Orphan Drug Designation in February 2018, EU Orphan Designation in August 2018 and Swissmedic Orphan Drug Status in December 2018. AstraZeneca and MSD are jointly developing and commercialising selumetinib globally under a license agreement.

About SPRINT

SPRINT is a US NCI CTEP-sponsored Phase I/II trial. The Phase I trial was designed to identify the optimal Phase II dosing regimen, and the results were published in The New England Journal of Medicine.1

About selumetinib
Selumetinib is a MEK 1/2 inhibitor. It is designed to inhibit the MEK enzyme in the RAS/MAPK pathway, a cell-signalling pathway, associated with cancer cell growth and proliferation in a number of different tumour types.

About NF1

NF1 is an incurable genetic condition that affects one in every 3,000 to 4,000 individuals.2,3 It is caused by a spontaneous or inherited mutation in the NF1 gene and is associated with many symptoms, including soft lumps on and under the skin (cutaneous neurofibromas), skin pigmentation (so-called ‘cafe au lait’ spots) and, in 30-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas).1 These plexiform neurofibromas can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bowel/bladder dysfunction and disfigurement as well as having the potential to transform into malignant peripheral nerve sheath tumours (MPNST).1

People with NF1 may experience a number of complications such as learning difficulties, visual impairment, twisting and curvature of the spine, high blood pressure, and epilepsy. NF1 also increases a person’s risk of developing other cancers, including malignant brain tumours, MPNST and leukaemia. Symptoms begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.4

About the AstraZeneca and MSD strategic oncology collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the United States and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and potential new medicine selumetinib, a MEK inhibitor, for multiple cancer types. Working together, the companies will develop Lynparza and selumetinib in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and selumetinib in combination with their respective PD-L1 and PD-1 medicines.

About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance Oncology as one of AstraZeneca’s four Growth Platforms focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

On November 14, 2019 BioXcel Therapeutics, Inc. ("BTI" or "Company") (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence to identify improved therapies in neuroscience and immuno-oncology, reported its quarterly results for the third quarter ended September 30, 2019 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, NOV 14, 2019, View Source [SID1234551242]).

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"We are pleased with the progress made on both of our clinical development programs during the quarter," stated Vimal Mehta, Chief Executive Officer of BTAI. "BXCL501, our candidate for the acute treatment of agitation in patients with schizophrenia and bipolar disease and delivered in a sublingual thin film, is highly differentiated from the current standards of care, which can produce unwanted side effects and be difficult for caregivers to administer. BXCL501 has the potential to significantly improve care for patients, while providing healthcare providers with an important new option for treating their patients. We’re looking forward to reporting pivotal data during the first half of 2020."

Dr. Mehta added, "We are also pleased with the progress of our Phase 1b/2 double combination study of BXCL701 and Keytruda for tNEPC and anticipate additional safety data readouts from both cohorts in the fourth quarter of this year, followed by expected initial efficacy data in the first half of 2020."

Third Quarter 2019 and Recent Highlights

BXCL501-Neuroscience Program-

BXCL501 is an investigational sublingual thin film of dexmedetomidine, a selective alpha-2A adrenergic receptor agonist, designed for the treatment of acute agitation. The Company believes BXCL501 may directly target a causal agitation mechanism.

·BXCL501 met its primary endpoint and demonstrated statistically significant mean reduction in PEC (PANSS, or the Positive and Negative Syndrome Scale, Excitatory Component) in the Phase 1b trial with agitated schizophrenia patients. Pivotal studies for the acute treatment of agitation in schizophrenia and bipolar patients are expected to initiate in Q4 2019, with data readouts expected 1H 2020;

The Phase 1b/2 study of BXCL501 for acute treatment of agitation in geriatric dementia/Alzheimer’s disease is expected to begin in Q4 2019, with data expected in 1H 2020;

· Initiated BXCL501 strategic initiative to investigate the feasibility of development of digital device technology, such as the Apple Watch, that can be used in conjunction with BXCL501 to enhance the prevention and treatment of agitation, specifically in geriatric dementia patients;

·Awarded grant by CDMRP to expand clinical development of BXCL501 program for the treatment of alcohol and substance abuse disorders related to PTSD in collaboration with Yale University.

BXCL701-Immuno-Oncology Program-

BXCL701 is an orally-delivered small molecule, innate immunity activator designed to inhibit dipeptidyl peptidase (DPP) 8/9 and block immune evasion by targeting Fibroblast Activation Protein (FAP). It has shown single agent activity in melanoma and safety has been evaluated in more than 700 healthy subjects and cancer patients.

· The Phase 1b/2 trial of BXCL701 and Keytruda for tNEPC is ongoing. The Company presented safety and tolerability data from the first patient cohort at the Annual Prostate Cancer Foundation Scientific Retreat and is currently enrolling a second patient cohort. BTI expects to report additional safety findings by year-end before advancing to the Phase 2 stage of the trial;

·BXCL701 received third orphan drug designation (ODD) from the FDA for the treatment of AML. Potential to expand clinical development of BXCL701 program into hematological malignancies;

· The BXCL701 phase of the triple combination study of BXCL701, bempegaldesleukin (NKTR-214, Nektar Therapeutics, Inc.) and BAVENCIO (avelumab, Merck KGaA, Darmstadt, Germany and Pfizer) in pancreatic cancer is expected to be initiated following Nektar and Pfizer’s safety run-in trial of a double combination of bempegaldesleukin and avelumab and the outcome of that trial.

Strengthened Balance Sheet

·BioXcel raised gross proceeds of $19.0 million in the quarter, the net of which, together with current reserves, provides sufficient capital to fund operations through key data readouts including Phase 3 and Phase 1b/2 studies with BXCL501.

Third Quarter 2019 Financial Results

BTI reported a net loss of $9.0 million for the third quarter of 2019, compared to a net loss of $4.9 million for the same period in 2018. The third quarter 2019 results include approximately $0.8 million in non-cash stock based compensation.

Research and development expenses were $7.1 million for the third quarter of 2019, as compared to $3.8 million for the same period in 2018. The increase was primarily due to an expansion of research and development activities, including increased personnel costs, clinical trials expenses, and professional fees, associated with BTI’s two lead product candidates.

General and administrative expenses were $2.0 million for the third quarter of 2019, as compared to $1.3 million for the same period in 2018. The increase was primarily due to additional payroll and payroll-related expenses, professional fees and costs associated with operating as a public company.

As of September 30, 2019, cash and cash equivalents totaled approximately $40.3 million which included proceeds from the Company’s follow-on-financing completed on September 30, 2019. BTI believes it is well positioned to execute on key milestones.

Conference Call:

BTI will host a conference call and webcast today at 8:30 a.m. ET. To access the call, please dial 877-407-2985 (domestic) and 201-378-4915 (international). A live webcast of the call will be available on the Investors sections of the BTI website at www.bioxceltherapeutics.com. The replay will be available through November 28, 2019.

On November 14, 2019 Incyte Corporation (Nasdaq:INCY) reported that it will present at the Piper Jaffray 31st Annual Healthcare Conference on Wednesday, December 4, 2019 at 8:30 am (EST) in New York (Press release, Incyte, NOV 14, 2019, View Source [SID1234551241]).

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The presentation will be webcast live and can be accessed at www.incyte.com under For Investors, Events and Presentations and will be available for replay for 30 days. Investors interested in listening to the live webcast should log on before the start time in order to download any software required.