On May 12, 2020 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a precision oncology medicine company pioneering the discovery and development of small molecule, tumor-agnostic therapies, reported financial results for the first quarter ended March 31, 2020, and provided a corporate update (Press release, Black Diamond Therapeutics, MAY 12, 2020, View Source [SID1234557641]).

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"We are pleased with our first quarter progress which included advancing the Phase 1/2 clinical trial of our lead product candidate BDTX-189 and completing our successful initial public offering," said David M. Epstein, Ph.D., President and CEO. "All companies are being impacted by the COVID-19 pandemic and Black Diamond is no exception. Our priority is the safety and well-being of our patients, as well as employees and their families, and we have adjusted our operations accordingly. We have also put in place contingency plans and additional resources from third-party service providers to minimize the impact on our business and strategy. We believe we are well positioned not only to execute on the clinical development of BDTX-189 as planned, but also to continue to invest in our proprietary MAP platform and to progress our early stage pipeline of small molecule, tumor-agnostic precision medicine programs. We will continue to closely monitor the global COVID-19 situation and its impact on our business as it evolves."

Recent Developments
•In February 2020, Black Diamond completed an initial public offering (IPO) pursuant to which it issued and sold 12,174,263 shares of common stock, including full exercise of the underwriters’ over-allotment option, resulting in gross proceeds of $231.3 million before deducting underwriting discounts and commissions and other offering expenses.
•Black Diamond continued to enroll and dose patients in the Phase 1 portion of a Phase 1/2 clinical trial of BTDX-189 (MasterKey-01; NCT04209465) and expects to complete the Phase 1 portion of the trial by the first half of 2021. BDTX-189 is Black Diamond’s mutation spectrum-selective, oral, irreversible small molecule inhibitor product candidate, which targets cancer-causing driver mutations in human epidermal growth factor receptor 2 (HER2) and epidermal growth factor receptor (EGFR) that have not yet been drugged.
•Black Diamond continued to advance its program in glioblastoma multiforme (GBM) toward nomination of a development candidate targeting a range of driver mutations in GBM, as well as its earlier-stage programs derived from the Company’s Mutation-Allostery-Pharmacology (MAP) platform.
•Black Diamond implemented risk mitigation measures and adjusted operations in response to the COVID-19 pandemic.
Financial Highlights
•Black Diamond ended the first quarter of 2020 with $357.2 million in cash and cash equivalents, which included net proceeds from the Company’s IPO of $212.1 million, compared to $44.7 million for the first quarter of 2019. Net cash used in operations was $11.3 million for the first quarter of 2020 compared to $6.9 million for the first quarter of 2019.
•Research and development (R&D) expenses were $7.4 million for the first quarter of 2020 compared to $3.0 million for the first quarter of 2019. The increase in R&D expenses was primarily related to an increase in headcount, preclinical development, and advancement of the BDTX-189 Phase 1/2 clinical trial.
•General and administrative (G&A) expenses were $5.5 million for the first quarter of 2020 compared to $0.8 million for the first quarter of 2019. The increase in G&A expenses was primarily due to an increase in personnel and costs associated with operations as a public company.

Upcoming Events
•The Company will present a poster about the MasterKey-01 Phase 1/2 clinical trial design at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program:
◦Abstract Title: Masterkey-01: Phase I/II, open-label multicenter study to assess safety, tolerability, pharmacokinetics, and antitumor activity of BDTX-189, an inhibitor of allosteric ErbB mutations, in patients with advanced solid malignancies (Abstract TPS3665; Poster 395)
◦Poster Session: Developmental Therapeutics – Molecularly Targeted Agents and Tumor Biology
•David M. Epstein, Ph.D., President and CEO, is scheduled to present at the following upcoming conferences:
◦Bank of America Global Research Health Care Conference 2020, on Tuesday, May 12, 2020, at 4:20 PM ET
◦Jefferies Global Healthcare Conference, on Wednesday, June 3, 2020, at 1:30 PM ET
◦BMO 2020 Prescriptions for Success Healthcare Virtual Conference, on Tuesday, June 23, 2020

On May 12, 2020 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported that advances from its "off-the-shelf" or allogeneic platform at the American Society for Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual meeting which kicked off in virtual format today (Press release, Adaptimmune, MAY 12, 2020, View Source [SID1234557587]).

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Data from a poster summarized recent advances in Adaptimmune’s allogeneic platform demonstrating the production of engineered T-cells, differentiated from human induced pluripotent stem cells (hiPSC) (iT cells). The poster was presented by Garth Hamilton, PhD, a Principal Scientist at Adaptimmune, and Joanna Brewer, PhD, Adaptimmune’s SVP Allogeneic Research. There is a video webcast of this presentation available on the Adaptimmune website: https://bit.ly/2L6jtx5.

"We have clearly demonstrated that we can make T-cells from stem cells. These cells express our engineered SPEAR TCR targeting MAGE-A4 and can kill tumor cells in vitro as effectively as control cells," said Jo Brewer, Adaptimmune’s SVP of Allogeneic Research. "We have also reached a milestone in editing our engineered TCRs into the hiPSC genome to enable successful differentiation. This is one critical element as we plan to build large banks of pluripotent cells for our proprietary differentiation process, to generate functional T-cells expressing our engineered receptors. These are great advances as we prepare for clinical use."

Summary

·Poster Title: Driving ADP-A2M4 SPEAR Expression from an Endogenous Hematopoietic Lineage Promotor for "Off-the-Shelf" T-Cell Therapy for MAGE-A4+ Solid Tumors
·Edited hiPSCs (iT-cells) expressing the ADP-A2M4 TCR can kill cancer targets in vitro
·Data suggests that, like autologous SPEAR T-cells currently in clinical trials, ADP-A2M4 iT-cells have potential to be an efficacious cell therapy
·Ability to promote T-cell receptor expression in iT-cells via genetic knock-in at a defined locus offers an opportunity to produce multiple clonal iPSC banks encoding specific SPEAR TCRs against a range of tumor antigens

On May 12, 2020 INmune Bio, Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, reported presentation of a study demonstrating the combination of INB03 with lapatinib reverses resistance to therapy in trastuzumab resistant HER2+ breast cancer (Press release, INmune Bio, MAY 12, 2020, View Source [SID1234557603]). The work lays the groundwork for INmune Bio’s planned Phase II trial and, was chosen for an oral presentation at the New York Academy of Science Frontiers in Cancer Immunotherapy 2020 publication on Monday, 11 May. Sophi Bruni, a doctoral student in the laboratory of Dr. Roxana Schillaci, in the Lab of Molecular Mechanisms, Instituto de Biología y Medicina Experimental-CONICET, Argentina will be presenting the work.

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This presentation is part of the evolving body of work by Dr. Schillaci and her team in the role of soluble TNF in resistance to immunotherapies in cancer. Dr. Schillaci previously reported that women with MUC4 expressing HER2+ breast cancers are resistant to trastuzumab and downregulation of MUC4 with INB03 reversed trastuzumab resistance. This work is part of a larger body of work looking at reversal of resistance to tyrosine kinase inhibitors in HER2+ breast cancer.

"Previous work by Dr. Schillaci suggests soluble TNF plays an important role in causing trastuzumab resistance in women with HER2+ breast cancer," said RJ Tesi MD, Chief Executive Officer of INmune Bio. "This work provides the therapeutic combination that fits neatly into the standard-of-care and may benefit women with brain metastasis caused by HER2 positive breast cancer."

"For the last two decades, trastuzumab administration as first line in HER2+ breast cancer has positively changed the prognosis of patients. However, in the metastatic setting, this disease is still life-threatening." said Dr. Schillaci. "Our previous work suggested MUC4 inhibited trastuzumab by steric hinderance. This work shows the mechanism is more interesting and involves intracellular mechanisms."

Dr. Schillaci’s work in defining the role of soluble TNF in trastuzumab resistance in women with HER2+ breast cancer is the basis for INMB’s planned Phase II trial in women with metastatic HER2+ breast cancer.

Today at 1:00PM EST a video of the presentation will be available which can be found on the Company’s YouTube channel by clicking here.

On May 12, 2020 Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) ("Eagle" or the "Company") reported that Scott Tarriff, Chief Executive Officer, and Pete Meyers, Chief Financial Officer, will host a fireside chat at the 2020 RBC Capital Markets Global Healthcare Virtual Conference as follows (Press release, Eagle Pharmaceuticals, MAY 12, 2020, View Source [SID1234557619]):

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Date: Tuesday, May 19, 2020
Time: 3:05 p.m. Eastern Time
Webcast: https://www.veracast.com/webcasts/rbc/healthcare2020/01112239705.cfm
The presentation will be webcast live at the aforementioned time, and archived for 30 days thereafter, via the Company’s website at www.eagleus.com, under the Investors section.

On May 12, 2020 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 first quarter ended March 31, 2020 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, MAY 12, 2020, View Source [SID1234557588]).

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"BioXcel continued to advance on its key milestones for 2020," stated Vimal Mehta, Chief Executive Officer of BTAI. "Beginning with our neuroscience program, we have made significant progress advancing BXCL501, as evidenced by our three ongoing clinical trials, SERENITY I & II and TRANQUILITY, and our Phase 1b/2 RELEASE trial initiating shortly. In parallel, we are investigating biomarkers associated with agitation in hopes of expanding the potential market for BXCL501 to additional indications. These significant achievements showcase the versatility of this candidate and we believe provides the foundation for creating a highly valuable neuroscience franchise. In addition, we have made great strides with our immuno-oncology program, identifying the recommended dose of BXCL701 when used in combination with KEYTRUDA for our Phase 2 efficacy trial for treatment emergent Neuroendocrine Prostate Cancer. We believe this candidate has the potential to provide a treatment for this advanced prostate cancer that currently does not have an effective standard of care."

Dr. Mehta added, "In light of the COVID-19 pandemic, we are continuously monitoring the safety of our team, as well as its potential impact on our clinical and corporate plans. To date, we have not experienced any significant delays with our ongoing clinical trials and have developed a risk mitigation strategy to manage business operations."

First Quarter 2020 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.

·The SERENITY program, two Phase 3 studies of BXCL501 for the acute treatment of agitation in patients with schizophrenia and bipolar disorder, is ongoing, with more than one-third of the patients enrolled and treated as of March 19, 2020. Enrollment is progressing as planned, and the Company is on track to report topline data from both Phase 3 trials in mid-2020.
In January 2020, the first patient was enrolled in the TRANQUILITY study, a Phase 1b/2 trial of BXCL501 for the acute treatment of agitation associated with geriatric dementia. BTI is currently assessing safety and tolerability data in order to choose the next tested dose, and the Company expects to report topline results in mid-2020.

·Our Investigational New Drug application for the treatment of opioid withdrawal symptoms, a fourth indication for BXCL501, received clearance from the U.S. Food and Drug Administration in February 2020. The Company is planning to initiate the Phase 1b/2 RELEASE trial for the treatment of opioid withdrawal symptoms shortly.
·In February 2020, researchers at Yale University initiated a Phase 2 study designed to measure biomarkers associated with agitation in patients with schizophrenia and their response to treatment with BXCL501.
·The Company is currently completing the clinical planning stage for its fifth indication, agitation associated with hyperactive delirium, and is preparing to initiate a Phase 1b/2 trial of BXCL501 in the second half of 2020.

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.

·After completing the Phase 1b safety lead-in, the Company has initiated the Phase 2 portion of the Phase 1b/2 trial of BXCL701 in combination with pembrolizumab (KEYTRUDA) for treatment emergent Neuroendocrine Prostate Cancer (tNEPC). 0.3 mg of BXCL701 twice daily (BID) was found to be the recommended dose when used in combination with KEYTRUDA and this dose regime will be used for the efficacy assessment of the clinical program. The Company expects to report initial data from this trial in the fourth quarter of 2020.
·The open label Phase 2 basket trial evaluating the combination of BXCL701 and KEYTRUDA in patients with advanced solid cancers has been initiated. This study, which is being conducted at the MD Anderson Cancer Center, is following the dosing schedule used in the Phase 1b/2 study for tNEPC.
·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 begin following Nektar and Pfizer’s Phase 1b safety trial of a double combination of bempegaldesleukin and avelumab and the outcome of that trial.

Strengthened Balance Sheet

·In February 2020, the Company raised net proceeds of approximately $60 million in connection with its common stock offering. BTI believes that proceeds from this offering, together with current reserves, provide cash runway to fund key clinical, regulatory and operational milestones into 2021.

COVID-19

During the first quarter of 2020, the Company took steps in line with guidance from the U.S. Centers for Disease Control and Prevention (CDC) and the State of Connecticut to protect the health and safety of its employees and the community. In particular, the Company implemented a work-from-home policy for all employees and has restricted on-site activities to certain chemical, manufacturing and control ("CMC") and clinical trial activities. To date, the Company has not experienced any significant delays to its ongoing or planned clinical trials; however, this could rapidly change.

First Quarter 2020 Financial Results

BTI reported a net loss of $14.9 million for the first quarter of 2020, compared to a net loss of $7.2 million for the same period in 2019. The first quarter 2020 results include approximately $0.8 million in non-cash stock-based compensation.

Research and development expenses were $12.4 million for the first quarter of 2020, as compared to $5.7 million for the same period in 2019. The increase was primarily due to an increase in clinical trial expenses, salaries, bonus and related costs, professional research and project-related costs and chemical, manufacturing and controls costs related to our BXCL501 and BXCL701 product candidates.

General and administrative expenses were $2.6 million for the first quarter of 2020, as compared to $1.7 million for the same period in 2019. The increase was primarily due to professional fees for additional legal and patent services.

Total operating expenses for the first quarter of 2020 were approximately $15.0 million, as compared to total operating expenses of approximately $7.4 million for the same period in 2019.

As of March 31, 2020, cash and cash equivalents totaled approximately $80.1 million.

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 May 26, 2020.