On May 12, 2020 Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening bacterial infections, reported financial and corporate results for the first quarter ended March 31, 2020 (Press release, Aridis Pharmaceuticals, MAY 12, 2020, View Source [SID1234557624]).

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First Quarter Highlights and Recent Developments

Enrolled the first patient with COVID-19 in AR-301 Phase 3 trial for patients with ventilator associated pneumonia (VAP)
Initiated dosing of the last dose cohort in the healthy volunteer portion of the AR-501 Phase 1/2a clinical trial with top-line data provisionally expected in 1H 2020 (healthy subjects), and in 2H 2021 (cystic fibrosis subjects)
Commenced COVID-19 monoclonal antibody and vaccine discovery activities utilizing the APEX platform technology
"While the COVID-19 pandemic has provided significant headwinds to patient trial enrollment across therapeutic indications including our own ongoing studies, we were still able to advance our lead programs and in fact, enrolled our first patient diagnosed with COVID-19 and on a ventilator into the AR-301 Phase 3 VAP study," commented Vu Truong, Ph.D., Chief Executive Officer of Aridis Pharmaceuticals. "Furthermore, the pandemic provided a unique opportunity to leverage our APEX platform technology for the rapid discovery and development of highly potent monoclonal antibodies from convalescent COVID-19 patients."

During the quarter, Aridis initiated COVID-19 monoclonal antibody and vaccine discovery activities utilizing its APEX technology platform for the unbiased discovery of new and highly potent antibodies against pathogens. The APEX platform is comprised of a silicon wafer-based array of nanoliter sized tissue micro-culture wells that enable rapid screening of antibody secreting cells, enabling discovery of potent antibodies against targets such as the virus that causes COVID-19 disease (called ‘SARS-CoV-2’) within a few days of patient sample availability. It also features CRISPR enabled activation of endogenous genetic control elements that dramatically increase the yield of bio-therapeutic drugs from manufacturing production cell lines. The technology also features a proprietary production cell line that is designed to rapidly manufacture multiple monoclonal antibody therapeutics at approximately half the manufacturing cycle time than currently available manufacturing technologies.

Clinical Program Update

AR-301: In April, Aridis enrolled its first COVID-19 patient in the Company’s ongoing Phase 3 clinical trial of AR-301. COVID-19 patients on prolonged mechanical ventilation in the intensive care unit (ICU) are prone to secondary infections (also called ‘superinfections’) by opportunistic pathogens such as bacteria. Superinfection is a reported complication in COVID-19 patients, which exacerbates morbidity and rate of mortality. The Company’s ongoing AR-301 Phase 3 study allows for the enrollment of patients with baseline characteristics which are inclusive of certain COVID-19 patients. While AR-301 is not an agent to treat SARS-CoV-2 virus itself, it can potentially reduce the morbidity associated with secondary S. aureus pneumonia, which is a coronavirus complication and a contributing cause of death in such patients.

The trial, which was initiated in the first quarter of 2019, is expected to enroll 240 patients at approximately 160 clinical centers in 22 countries. However, the advent of coronavirus infections which became apparent during the fourth quarter of 2019, impacted the global patient enrollment rate, and delayed further clinical site activations in regions with large number of clinical sites, such as in China and India. Contingent upon the evolution of the coronavirus pandemic, Aridis is provisionally expecting interim data to be reported in 2H 2020, and top line data in 2H 2021. Participating clinical centers that are activated continue to follow the same stringent clinical protocols and procedures for critically ill VAP patients, as is standard in the U.S. and Europe. The trial represents the first ever Phase 3 superiority clinical study evaluating immunotherapy with a fully human monoclonal antibody to treat acute pneumonia in the intensive care unit setting. Details of the study can be viewed on www.clinicaltrials.gov using identifier NCT03816956.

AR-301 is a fully human monoclonal IgG1 antibody specifically targeting gram-positive S. aureus alpha-toxin. It has been shown in vitro to protect against alpha-toxin mediated destruction of host cells, thereby potentially preserving the human immune response. AR-301’s mode of action is independent of the antibiotic resistance profile of S. aureus and it is active against infections caused by both MRSA (methicillin resistant S. aureus) and MSSA (methicillin sensitive S. aureus).

AR-501: During the first quarter, Aridis initiated dosing of the last dose cohort in healthy volunteer portion of the AR-501 Phase 1/2a clinical trial of this inhalable formulation of gallium citrate being evaluated for the treatment of chronic lung infections associated with cystic fibrosis. The single ascending dose cohorts of healthy subjects have completed dosing and the safety monitoring committee has recommended proceeding into the multiple ascending dose cohorts. The Company expects to report data from the Phase 1 portion of the trial which consists of healthy subjects provisionally in 1H 2020 and the Phase 2a segment with cystic fibrosis subjects in 2H 2021.

AR-501, which is being developed in collaboration with the Cystic Fibrosis Foundation (CFF), has been granted Orphan Drug Designation (ODD), Fast Track and Qualified Infectious Disease Product (QIDP) designations by the U.S. Food and Drug Administration (FDA). In addition, the European Medicines Agency (EMA) granted ODD to AR-501. Details of the Phase 1/2a clinical trial, which is a randomized, double-blinded, placebo controlled single and multiple dose-ascending trial investigating the safety and pharmacokinetics of inhaled AR-501 in healthy volunteers and cystic fibrosis patients with chronic bacterial lung infections, can be viewed on www.clinicaltrials.gov using identifier NCT03669614. The study is expected to accrue 48 healthy adult volunteers and 48 cystic fibrosis patients from approximately 15 sites in the U.S.

Corporate Update

During the first quarter, Aridis continued to present at leading investor and medical conferences, which due to the COVID-19 pandemic have been transitioned to virtual forums. The Company participated in Maxim Group’s Infectious Disease Virtual Conference held on May 5, 2020. The event consisted of four panels of companies in various stages of development, from early stage to near commercialization, that represent the next wave of innovation in the infectious disease sector. Vu Truong, Ph.D., Aridis’ Chief Executive Officer, was a speaker on two panels entitled "Non-antibiotic Anti-infectives" and "COVID-19 (Therapeutics)." The presentation slides can be found at View Source

Fiscal 2020 First Quarter Results:

Cash: Total cash and cash equivalents as of March 31, 2020 was $16.3 million.
Revenues: Grant revenue decreased from approximately $1.0 million for the quarter ended March 31, 2019 to zero for the quarter ended March 31, 2020 primarily due to the recognition of revenue related to a milestone under the grant award from the CFF during the first quarter of 2019 and none during the first quarter of 2020.
Research and Development Expenses: Research and development expenses incurred in the quarter ended March 31, 2020 were $4.9 million, a decrease of approximately $2.2 million over the same period in 2019 due primarily to a decrease in spending on clinical trial activities for the AR-105 Phase 2 program, which was completed in 2019, and a decrease in drug manufacturing expenses related to the AR-301 Phase 3 program. These decreases were partially offset by an increase in spending on clinical trial activities for both the AR-301 Phase 3 and the AR-501 Phase 1/2a programs.
General and Administrative Expenses: There was no material difference in general and administrative expenses for the quarter ended March 31, 2020 when compared to the same period in 2019.
Interest and Other Income, net: Interest and other income, net was $61,000 for the quarter ended March 31, 2020, a decrease of approximately $55,000 over the same period in 2019. These decreases were due primarily to a lower average cash balance and lower interest rates.
Share of Loss from Equity Method Investment: Loss from equity method investment decreased by $433,000 to $9,000 for the quarter ended March 31, 2020 over the same period in 2019 due to our share of loss from our minority interest under the equity method was limited to the book value of the investment.
Net Loss: The net loss for the quarter ended March 31, 2020 was $6.5 million, or ($0.73) per share, compared to a net loss of approximately $8.1 million, or ($0.99) per share, for the quarter ended March 31, 2019. The weighted average common shares outstanding was approximately 8.9 million and approximately 8.1 million for the first quarter of 2020 and 2019, respectively.

On May 12, 2020 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported that it is presenting three oral presentations and two poster presentations at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper), taking place virtually from May 12-15, 2020 (Press release, Intellia Therapeutics, MAY 12, 2020, View Source [SID1234557567]). Intellia researchers are presenting new data in support of NTLA-5001, the company’s engineered cell therapy candidate for the treatment of acute myeloid leukemia (AML). Intellia is also providing an update on NTLA-2002, its newest development candidate for the treatment of hereditary angioedema (HAE).

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"At Intellia, we are applying our CRISPR/Cas9 technology to develop new processes that can produce enhanced engineered cell therapies to treat severe cancers, such as AML, that traditional approaches cannot address. Our proprietary platform provides a powerful tool to generate more potent TCR-directed cells, that can treat blood cancers initially and potentially solid tumors. The data being presented today validate Intellia’s approach of reducing AML tumor cell blasts, and our plans to enter the clinic with NTLA-5001 next year," said Intellia President and CEO John Leonard, M.D. "We are also pleased to present data that support our recently announced HAE development candidate, NTLA-2002, Intellia’s second systemic therapy employing our in vivo knockout approach and modular delivery platform."

Data Presentations on Intellia’s First Engineered Cell Therapy Development Candidate, NTLA-5001 for the Treatment of AML, and Proprietary Cell Engineering Process

NTLA-5001 is Intellia’s first engineered T cell receptor (TCR) T cell therapy development candidate, which targets the Wilms’ Tumor 1 (WT1) intracellular antigen for the treatment of AML. NTLA-5001 is being developed in collaboration with Chiara Bonini’s team at IRCCS Ospedale San Raffaele to treat AML patients regardless of the genetic subtype of a patient’s leukemia. AML is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment and is the most common type of acute leukemia in adults (Source: NIH SEER Cancer Stat Facts: Leukemia – AML).

Intellia’s proprietary process is a significant improvement over standard engineering processes commonly used to introduce nucleic acids into cells. Intellia’s process enabled multiple gene edits using CRISPR/Cas9, while maintaining cell products with high expansion potential and minimal undesirable chromosomal translocations. CRISPR/Cas9 was used to insert a WT1-directed TCR in locus, while eliminating the expression of the endogenous TCRs, with the goal of producing homogeneous T cell therapies like NTLA-5001.

Intellia’s novel approach with NTLA-5001 can overcome the challenges of standard T cell therapy, including risks of reduced specificity associated with mixed expression and mispairing of endogenous and transgenic TCRs (tgTCRs); graph-versus-host disease (GvHD) risks, which could lead to an attack on the patient’s healthy cells; and reduced efficacy tied to lower tgTCR expression per T cell. Intellia’s unprecedented process is expected to streamline cell engineering and manufacturing, yielding a homogenous product comprising WT1-targeted T cells with high anti-tumor activity. Data highlights from today’s presentation include the following:

Developed a sequential genome editing process in primary human T cells that lead to the knockout of three genes with up to >98% efficiency and no detectable target-to-target translocations. Applied sequential editing approach to achieve knockout of the endogenous TCR with up to >99% efficiency, along with insertion of the tgTCR targeting WT1 into 50-70% of the cells.
Improved T cell viability post-editing that resulted in a significant increase in T cell expansion relative to cells engineered by standard methods, and which is expected to shorten the time required for T cell manufacturing and to increase the yield of therapeutic cells.
Increased proportion of cells having a desirable early stem cell memory phenotype with improved reactivity against WT1-expressing tumor cell lines and higher long-term proliferative capacity, which may be associated with better persistence in patients.
Intellia’s cell engineering efforts are focused on its initial clinical investigation of NLTA-5001 on AML, while continuing preclinical studies exploring the potential for targeting WT1 in solid tumors. The company confirmed plans last week to submit an IND or IND-equivalent for NTLA-5001 for the treatment of AML in the first half of 2021.

The presentation titled, "Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering," will be made today by Aaron Prodeus, Ph.D., senior scientist, Cell Therapy, and can be found here, on the Scientific Publications & Presentations page of Intellia’s website. These data were a follow-on to the study presented at Keystone Symposia’s Engineering the Genome Conference from this past February.

In Vivo Data Supports Intellia’s Novel TCR Candidate

A second presentation on engineered cell therapy progress, in collaboration with IRCCS Ospedale San Raffaele, showed in vivo data demonstrating the potential of TCR-edited T cells to effectively target WT1 tumor cells in AML. In addition to the previously disclosed results of effective in vitro recognition of primary AML tumor cells by edited WT1-specific cytotoxic T cells (CD8 T cells), new data indicate that the selected TCR also enables T helper cells (CD4 T cells) to react to WT1-expressing tumor cells, providing cytokine support. This distinguishes Intellia’s TCR from other therapeutic TCR candidates, which either exclusively activate toxic CD8 T cells or require the co-transfection of CD8 into CD4 T cells to render them functional.

Using a mouse model carrying disseminated human primary AML, researchers observed a significant therapeutic effect, including decreased AML tumor burden. In addition, no signs of GvHD were observed in mice treated with the WT1-specific T cells. The data show that tgTCR-engineered cells have targeted anti-cancer activity in a challenging model of systemic AML, demonstrating the therapeutic potential of Intellia’s engineered TCR T cell approach.

The presentation titled, "Exploiting CRISPR-Genome Editing and WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid Leukemia," will be given today by Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy. Notably, ASGCT (Free ASGCT Whitepaper) meeting organizers selected this presentation as one of six to receive the ASGCT (Free ASGCT Whitepaper) Excellence in Research Award this year.

Continued Progress on Intellia’s Second In Vivo Development Candidate, NTLA-2002 for the Treatment of HAE

Intellia is presenting development data updates on its potential HAE therapy, NTLA-2002, which utilizes the company’s systemic in vivo knockout approach, including its proprietary lipid nanoparticle (LNP) system. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. NTLA-2002 aims to prevent unregulated production of bradykinin by knocking out the prekallikrein B1 (KLKB1) gene through a single course of treatment to ameliorate the frequency and intensity of these swelling attacks.

The KLKB1 gene knockout in an ongoing non-human primate (NHP) study resulted in a sustained 90% reduction in kallikrein activity, a level that translates to a therapeutically meaningful impact on HAE attack rates (Source: Banerji et al., NEJM, 2017). This kallikrein activity reduction was sustained for at least six months, demonstrating the same high level of efficacy and durability seen in earlier rodent studies.

The short talk titled, "CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat Hereditary Angioedema," will be given by Jessica Seitzer, director, Genomics, Intellia on Fri., May 15, 2020, when it will be made available here, on the Scientific Publications & Presentations page of Intellia’s website. The presented data include results from ongoing collaborations with researchers at Regeneron, and the program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party. Intellia expects to submit an IND or IND-equivalent to initiate a Phase 1 trial for NTLA-2002 in the second half of 2021.

On May 12, 2020, Genocea Biosciences Presented the Corporate Presentation (Presentation, Genocea Biosciences, MAY 12, 2020, View Source [SID1234557593]).

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On May 12, 2020 NuVasive, Inc. (NASDAQ: NUVA), the leader in spine technology innovation, focused on transforming spine surgery with minimally disruptive, procedurally integrated solutions, reported that management will present at the UBS Virtual Global Healthcare Conference on Tuesday, May 19 at 3:00 p.m. ET/12:00 p.m. PT (Press release, NuVasive, MAY 12, 2020, View Source [SID1234557625]).

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A live webcast of the presentation will be available online from the Investor Relations page of the Company’s website at www.nuvasive.com. A replay of the presentation will remain available on the website for 30 days after the live webcast.

On May 12, 2020 Sun BioPharma, Inc. (OTCQB: SNBP), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with pancreatic cancer, reported financial results for the quarter ended March 31, 2020 (Press release, Sun BioPharma, MAY 12, 2020, View Source [SID1234557900])

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First-Line Combination Pancreatic Cancer moves into expansion phase prior to COVID-19 pause During the first quarter of 2020, the Company completed enrollment in an added fourth cohort that was intended to study a dose schedule more conveniently aligned with the standard gemcitabine and nab-paclitaxel regimen, and immediately began enrollment of subjects in the expansion phase of this clinical trial. This trial, a Phase 1a/1b combination of SBP-101 with gemcitabine and nab-paclitaxel in patients previously untreated for metastatic pancreatic ductal adenocarcinoma ("PDA"), is being conducted at sites in the United States and Australia.

In early April 2020, as a result of the impact of the COVID-19 pandemic on the conduct of clinical trials around the world, the Company announced a pause in enrollment of new patients. Patients already enrolled in the clinical trial continue to be treated, and the Company intends to resume recruitment in the near future.

"We continue to monitor the situation in communities where our trial is in progress," noted Michael Cullen, MD, Executive Chairman, President and CEO. "We expect to resume enrollment in the second quarter; that timeline is dependent on the course of the pandemic.

" Financial Results for the Three months ending March 31, 2020

Operating Results

General and administrative ("G&A") expenses increased 54.5% to $468,000 in the first quarter of 2020, up from $303,000 in the first quarter of 2019. The increase is due to resumption of normal salary levels following a voluntary reduction of salaries for a part of the first quarter of 2019, in addition to higher stock compensation and legal expenses. Research and development ("R&D") expenses increased 70.9% to $598,000 in the first quarter of 2020, up from $350,000 in the first quarter of 2019, primarily due to an increase in clinical trial costs.

Other net expenses were $824,000 and $999,000 for the three months ended March 31, 2020 and 2019, respectively. The net expense in the quarter ended March 31, 2020 is composed primarily of a foreign currency exchange loss on the intercompany receivable balance. The net expense in the quarter ended March 31, 2019 is primarily the amortization of debt discount on convertible notes sold in December 2018 and January 2019, all of which converted into equity securities in 2019.

Net loss in the first quarter of 2020 was $1.8 million, or $0.27 per diluted share, compared to a net loss of $1.6 million, or $0.31 per diluted share, in the first quarter of 2019.

Balance Sheet and Cash Flow Total cash was $1.3 million and $2.4 million as of March 31,2020 and December 31, 2019, respectively. Total current assets were $2.1 million and $3.1 million as of March 31, 2020, and December 31, 2019, respectively. Current liabilities decreased to $1.4 million as of March 31, 2020, compared to $1.8 million as of December 31, 2019, primarily as a result of payments made on vendor balances.

Net cash used in operating activities was $1.1 million in the three months ended March 31, 2020, compared to $0.8 million in the same period of the prior year. The net cash used in each of these periods primarily reflected the net loss for these periods and was partially offset by the effects of changes in operating assets and liabilities.

About SBP-101
SBP-101 is a first-in-class, proprietary, polyamine analogue designed to induce polyamine metabolic inhibition (PMI), exploiting a high affinity for the compound specific to the exocrine pancreas and pancreatic ductal adenocarcinoma. Sun BioPharma originally licensed SBP-101 from the University of Florida Research Foundation in 2011. The molecule has shown signals of efficacy in US and Australian metastatic pancreatic cancer patients, demonstrating complementary activity with an existing FDA-approved chemotherapy regimen. SBP-101 is expected to differ from current pancreatic cancer therapies in that it does not appear to exacerbate the typical adverse events of bone marrow suppression and peripheral neuropathy. Management believes that SBP-101 may effectively treat patients with primary and metastatic pancreatic cancer, and may be effective in combination with other agents, and in other types of cancer. The safety and PMI profile demonstrated in Sun BioPharma’s current clinical trialsupport evaluation of the compound in a randomized clinical trial.