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CytRx Comments on Quarterly Results and Recent Strategic Initiatives

On August 12, 2021 CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, reported on its results for the second quarter ended June 30, 2021 (Press release, CytRx, AUG 12, 2021, View Source [SID1234586480]). In addition, CytRx recapped corporate developments as well as matters pertaining to its agreements with ImmunityBio, Inc. (NASDAQ:IBRX) ("ImmunityBio") and Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme"). The Company’s 10-Q was filed today.

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Steven A. Kriegsman, Chairman and Chief Executive Officer of CytRx, commented:

"We took important steps to enhance our capital position and further strengthen our corporate governance during the second quarter. By raising gross proceeds of $10 million from our recent financing, we have obtained valuable working capital that can help us maintain stability as we efficiently manage our portfolio of licensing agreements and high-potential assets. We continue to believe in the long-term promise of our licensed drugs and Centurion Biopharma. We look forward to monitoring Orphazyme’s pursuit of European regulatory approval for arimoclomol in Q4 2021 and ImmunityBio’s Q1 2022 release of Cohort C survival data from its QUILT 88 study, which is a Phase 2 pancreatic cancer trial that includes aldoxorubicin."

First Quarter Financial Overview

CytRx concluded the quarter ended June 30, 2021 with cash on hand of approximately $8.4 million.
The Company recorded a net loss of $1.2 million for the quarter ended June 30, 2021, compared to a net loss of $1.3 million for the same period in 2020.
General and administrative expenses were $1.2 million for the quarter, compared with $1.4 million for the same period in 2020.
Recent Developments

Corporate Highlights

Last month, CytRx entered into a securities purchase agreement with a healthcare-focused institutional investor, resulting in aggregate gross proceeds of approximately $10 million. The investor is independent of the Company’s Board of Directors and management team. The Company intends to use the net proceeds for working capital purposes.
Last month, Jennifer K. Simpson, Ph.D joined the Company’s Board of Directors. Dr. Simpson is the Chief Executive Officer and a Director of Panbela Therapeutics Inc. (NASDAQ: PBLA), a clinical stage drug development company. Shortly after joining Panbela Therapeutics, Dr. Simpson led a public financing with an uplist to the NASDAQ exchange. She has more than 13 years’ experience in pharmaceutical executive leadership, global marketing and product commercialization.
With respect to Centurion Biopharma, Mr. Kriegsman and Lead Director Louis Ignarro, PhD continued pursuing strategic partnership opportunities to advance clinical testing for the platform’s assets. Discussions with prospective partners under confidentiality agreements are ongoing. There are no formal updates to report at this time.
CytRx maintains federal and state net operating loss ("NOL") carryforwards of $327.6 million and $252.6 million, respectively, available to offset against future taxable income. Of this amount, $258.3 million of federal NOLs and $252.6 million of state NOLs are unrestricted.
ImmunityBio Highlights

ImmunityBio announced during the quarter that Cohort C of its QUILT 88 study in pancreatic cancer, which includes aldoxorubicin and patients who have previously failed two lines of standard-of-care therapy, is expected to be completed in the third quarter of 2021. An early readout of survival data is expected in the first quarter of 2022.
Orphazyme Highlights

Orphazyme announced during the quarter that it received a Complete Response Letter ("CRL") from the Food and Drug Administration ("FDA") following its review of the new drug application for arimoclomol. Orphazyme disclosed that the FDA issued the CRL based on needing additional evidence to further substantiate the validity and interpretation of the 5-domain NPC Clinical Severity Scale and, in particular, the swallow domain. Further, the FDA noted in the CRL that additional data is needed to bolster confirmatory evidence beyond the single phase 2/3 clinical trial to support the benefit-risk assessment of the NDA.
Subsequently, Orphazyme announced 24-month interim results of an open-label extension ("OLE") trial of arimoclomol for the treatment of NPC. Orphazyme provided efficacy and safety data for its investigational treatment arimoclomol in NPC for up to 36 months. The results demonstrate that arimoclomol provided a sustained benefit to study participants by reducing NPC progression as measured by the 5-domain NPC Clinical Severity Scale (5D-NPCCSS).
Orphazyme is expecting prospective European regulatory approval for arimoclomol in the treatment of NPC by the end of 2021.

Humanigen Reports Second Quarter 2021 Financial Results

On August 12, 2021 Humanigen, Inc. (Nasdaq: HGEN) ("Humanigen"), a clinical stage biopharmaceutical company focused on preventing and treating an immune hyper-response called "cytokine storm’" with its lead drug candidate, lenzilumab, reported financial results for the second quarter and six months ended June 30, 2021 (Press release, Humanigen, AUG 12, 2021, View Source [SID1234586479]).

Update on Status of Emergency Use Authorization ("EUA") Application

On May 28, 2021, Humanigen submitted an EUA application for lenzilumab in patients hospitalized with COVID-19. Since that time, the company has responded to several requests from the U.S. Food and Drug Administration ("FDA") regarding the application. No formal timelines exist for the FDA to complete their review of our EUA application and as a result the company is unable to give guidance on the timing of a decision by the FDA.

"We remain firm in our belief the results of our LIVE-AIR Phase 3 study warrant lenzilumab being granted emergency use authorization. The achievement of the primary endpoint for the overall patient population, and the recent supplemental subset analysis which showed significant response to treatment by Black and African-American patients in the study, support our view of the potential benefit lenzilumab could bring to patient care if authorization were to be granted," said Cameron Durrant, MD, Chief Executive Officer, Humanigen.

Timothy Morris, CFO and COO, Humanigen, noted, "While the review of the EUA application continues, we are preparing for potential launch in the U.S., and simultaneously working to complete, before the end of the third quarter 2021, the submission of the Marketing Authorization Application ("MAA") to the Medicines Healthcare products Regulatory Agency ("MHRA") in the U.K. We have initiated the MAA process to the European Medicines Agency ("EMA") and anticipate the appointment of rapporteurs in the near term."

Second Quarter and Recent Highlights:

Lenzilumab in hospitalized COVID-19

Submitted an application to the FDA for an EUA for lenzilumab for the treatment of patients hospitalized with COVID-19.
Initiated filing and received acknowledgement the UK submission for Marketing Authorization for lenzilumab in COVID-19 has been accepted for expedited COVID-related rolling review by the MHRA. The submission is expected to be completed by September 30, 2021.
Initiated the process to make a submission to the European Medicines Agency for Marketing Authorization of lenzilumab in COVID-19.
The company’s commercial partners in South Korea and the Philippines, KPM Tech and Telcon RF Pharmaceutical, received approval from South Korea’s Ministry of Food and Drug Safety, the South Korean equivalent of FDA, to conduct a Phase 1 clinical study of lenzilumab, to enable submission and potential approval in South Korea.
Announced analysis of results from our Phase 3 LIVE-AIR study of lenzilumab in hospitalized patients with COVID-19 suggesting Black and African-American patients having a CRP<150 mg/L may be the highest responders to treatment, with a nearly 9-fold increase in likelihood of survival without ventilation ("SWOV") [n=51, p-value=0.0418]. In the overall population with CRP<150 mg/L, LIVE-AIR Phase 3 results show patients treated with lenzilumab demonstrated a 2.5-fold increased likelihood of SWOV [mITT, n=351, p-value=0.0009].
Entered into manufacturing agreement with Chime Biologics to produce lenzilumab bulk drug substance and drug product to be sold, with requisite regulatory authorization, in regions outside the United States.
Corporate

Completed underwritten public offering of common stock, raising net proceeds of $94.2 million.
The company was added to the Russell 3000 Index in June 2021.
Ken Trbovich was appointed to the newly-created role of SVP Investor Relations.
ACTIV-5 Update

In August 2021, the National Institutes of Health ("NIH") announced the expansion of the Accelerating COVID-19 Therapeutic Interventions and Vaccines ("ACTIV-5") and Big Effect Trial, in the "B" arm of the trial ("BET-B"), referred to as ACTIV-5/BET-B. Following feedback from and consultation with the company, the NIH advanced the study to a Phase 2/3 study with target enrollment of at least 400 patients and amended the protocol for ACTIV-5/BET-B in a manner that aligns with the design of the company’s LIVE-AIR trial. As a result of the advancement to a Phase 2/3 and amended protocol, the company anticipates that ACTIV-5/BET-B may serve as a second confirmatory study required for submission to FDA as part of a Biologics License Application ("BLA") that the company would submit if the ACTIV-5/BET-B data further validate the benefits of lenzilumab in COVID-19 patients.

Second Quarter and Six Months Ended June 30, 2021 Financial Results

Net loss for the three months ended June 30, 2021 was $70.8 million or $1.20 per share as compared to $24.0 million or $0.79 per share for the three months ended June 30, 2020. The net loss for the six months ended June 30, 2021 was $136.4 million or $2.45 per share as compared to $26.5 million or $1.00 per share for the six months ended June 30, 2020. The increase in net loss for both periods was largely due to an increase in total expenses, mainly Research and Development ("R&D") expense which rose significantly as the company accelerated its efforts to manufacture lenzilumab for potential commercialization upon a regulatory authorization. R&D expense increased $41.9 million from $21.1 million for the three months ended June 30, 2020, to $63.0 million for the three months ended June 30, 2021, and increased $101.1 million from $21.8 million for the six months ended June 30, 2020, to $122.9 million for the six months ended June 30, 2021. The manufacturing expense included in R&D was $57.1 million for the second quarter of 2021 as compared to $17.1 million for the prior year quarter, and $107.1 million for the six months ended June 30, 2021, as compared to $17.4 million for the prior year period. The costs incurred to produce lenzilumab will continue to be included in R&D expense until lenzilumab is authorized or approved for commercial use, at which point the amounts expended for production would be reclassified as inventory. A meaningful portion of these expenses are associated with initiation of manufacturing processes on a site-by-site basis.

Cash and Cash Equivalents

Net cash used in operating activities, net of balance sheet changes, was $103.8 million for the six months ended June 30, 2021. During the same period, the company raised net proceeds of $36.1 million from the sale of shares of common stock under its At-the-Market offering program, drew the first tranche of $25.0 million under its credit facility with Hercules Capital, providing net proceeds of $24.4 million, and completed a public offering of common stock with net proceeds of $94.2 million. As of June 30, 2021, the company had cash and cash equivalents of $120.5 million. The company expects to continue to use its funds on the manufacturing of lenzilumab in anticipation of its potential commercialization under EUA in the US or conditional marketing authorization in the UK. For the third quarter of 2021 the company anticipates the R&D expense related to lenzilumab production will be same level as the second quarter of 2021. If an EUA or CMA for lenzilumab is not received in the third quarter of 2021, the company would seek to decrease its spending on lenzilumab production.

A summary of key financial highlights as of and for the three and six months ended June 30, 2021 and 2020 is as follows ($ in thousands):

bioAffinity Technologies Publishes Sputum Processing and Innovative High-Throughput Analysis Methods

On August 12, 2021 bioAffinity Technologies, a privately held company focused on early diagnostics and cancer therapeutics, announced publication in the peer-reviewed Journal of Visualized Experiments (JoVE) (Press release, BioAffinity Technologies, AUG 12, 2021, View Source [SID1234586478]). The paper, entitled Quality-Controlled Sputum Analysis by Flow Cytometry, presents an efficient and effective method to analyze sputum on a flow cytometric platform, allowing for development of high-throughput diagnostic tests for lung cancer and other lung diseases.

"Sputum provides a superior sample in which to analyze cellular content and other microenvironmental features. Analysis of sputum by the method we describe opens a new and exciting window of opportunity for determining the health of an individual’s lungs," bioAffinity President and CEO Maria Zannes said. "The processing method provided in JoVE describes an innovative and highly efficient method to process sputum for use with flow cytometry, an approach that can rapidly analyze a sample for the presence of diseases such as COPD, asthma and lung cancer."

bioAffinity Technologies has developed CyPath, a platform technology to diagnose cancer. The Company’s first product is CyPath Lung, a non-invasive and cost-effective test for the early detection of lung cancer that allows patients to collect their sputum sample at home.

bioAffinity Technologies recently completed a test validation trial of CyPath Lung evaluating sputum from people at high risk for lung cancer, including patients with the disease and others who were cancer-free. The trial resulted in 92% sensitivity and 87% specificity in the group of patients who had no or smaller than 2 cm nodules in the lung.

CyPath Lung uses flow cytometry, a method able to interrogate an individual cell in a fraction of seconds, and automated analysis to identify parameters indicative of cancer. Unlike genomic or other molecular markers used in many liquid biopsies, bioAffinity’s CyPath technology does not collect genetic material for evaluation. CyPath uses flow cytometry to investigate the lung micro-environment by identifying cell populations including those preferentially labeled by the porphyrin TCPP that are indices of cancer.

Immune-Onc Therapeutics Announces FDA Clearance of IND Application to Initiate First-In-Human Trial of IO-108, a Novel Antagonist Antibody Targeting LILRB2 (ILT4), in Patients with Advanced Solid Tumors

On August 12, 2021 Immune-Onc Therapeutics, Inc. ("Immune-Onc"), a clinical-stage cancer immunotherapy company developing novel biotherapeutics targeting immunosuppressive myeloid checkpoints, reported that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for IO-108, a novel antagonist antibody targeting Leukocyte Immunoglobulin-Like Receptor B2 (LILRB2, also known as ILT4) for the treatment of solid tumors (Press release, Immune-Onc Therapeutics, AUG 12, 2021, View Source [SID1234586477]). Preclinical data presented at the 2020 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s annual meeting demonstrate that IO-108 functions as a myeloid checkpoint inhibitor. IO-108 reprograms immune-suppressive myeloid cells toward a pro-inflammatory phenotype, leading to enhanced innate and adaptive anti-tumor immunity.

"The clearance of the IO-108 IND represents another major milestone for Immune-Onc as we progress our pipeline of novel myeloid checkpoint inhibitors targeting the LILRB family of immune inhibitory receptors," said Charlene Liao, Ph.D., chief executive officer of Immune-Onc. "We are highly encouraged by the strength of the preclinical data of IO-108 and are pleased that our expertise in LILRB biology and translational sciences enables us to advance this asset into the clinic. We look forward to initiating the trial to further understand the role of LILRBs in cancer, and to test the potential of IO-108 in treating patients with advanced solid tumors."

The Phase 1, multicenter, dose-escalation study will consist of a monotherapy cohort and a combination therapy cohort to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of IO-108 alone and in combination with pembrolizumab, an anti-PD-1 antibody. Biomarkers will be assessed to enable a mechanistic understanding of clinical data and inform future trials. This study may also provide an opportunity to identify preliminary efficacy signals. After determination of the recommended Phase 2 dose, Immune-Onc plans to evaluate the efficacy, safety, and tolerability of IO-108 in combination with pembrolizumab and as monotherapy in indication-specific expansion cohorts.

IO-108 binds to LILRB2 with high affinity and specificity and blocks the interaction of LILRB2 with multiple ligands that are involved in cancer-associated immune suppression including HLA-G, ANGPTLs, SEMA4A, and CD1d. In preclinical studies, treatment of various primary human immune cell systems containing myeloid cells with IO-108 results in enhanced pro-inflammatory responses to multiple stimuli that are relevant to anti-tumor immunity. As a single agent, IO-108 reverses the anti-inflammatory myeloid cell phenotype that results from "tumor conditioning" and promotes the differentiation of monocytes into pro-inflammatory dendritic cells. Moreover, IO-108 potentiates the effect of PD-1 blocking antibodies on CD4+ T cell activation in co-cultures with allogeneic macrophages. In mouse models IO-108 inhibits the growth of solid tumors, which is associated with enhanced T cell responses. Together these data demonstrate that IO-108 has the potential to provide additive or synergistic benefit in combination with standard-of-care immunotherapies and/or immunogenic therapies for solid tumors that are both resistant and sensitive to T-cell checkpoint inhibitors.

ABOUT LILRB2 (ILT4)

LILRB2, also known as ILT4, is expressed mostly on myeloid cells, including monocytes, dendritic cells, macrophages, and neutrophils. In solid tumors, interaction of LILRB2 with tumor microenvironment (TME) relevant ligands, including HLA-G, ANGPTLs, SEMA4A, and CD1d, makes myeloid cells pro-tumorigenic (tolerating or promoting tumor growth) and promotes tumor immune evasion.

Noxopharm Pre-clinical Study Confirms Survival Advantage of Combination LuPSMA Therapy in Prostate Cancer

On August 12, 2021 Australian clinical-stage drug development company Noxopharm Limited (ASX:NOX) has reported pre-clinical data confirming a survival benefit of adding Veyonda to 177lutetium-PSMA-617 (LuPSMA) treatment in prostate cancer (Press release, Noxopharm, AUG 12, 2021, View Source [SID1234586476]). This result validates the survival benefit of the same combination seen in a recently completed Phase I/II trial of Veyonda in men with end-stage metastatic castrate-resistant prostate cancer (mCRPC).

Pre-Clinical Data Confirm Survival Benefit of Combination Treatment

A study in mice bearing human prostate cancer xenografts, and led by Professor Kristofer Thurecht, Ph.D., at The University of Queensland, confirmed a potent ability of Veyonda to enhance the cancer-killing effect of LuPSMA treatment.

"The combination of Veyonda with LuPSMA exhibited an impressive synergistic therapeutic response, with sustained and almost complete regression of the tumor and minimally observed systemic toxicity," said Dr. Thurecht. "This combined response was not observed in any of the animals treated with monotherapy."

Results Support Survival Benefit Found in Phase I/II Clinical Trial

The results of Noxopharm’s LuPIN Phase I/II clinical trial were published recently in The Journal of Nuclear Medicine and showed a median overall survival of 19.7 months with combination therapy in men with mCRPC with no remaining treatment options.

"The LuPIN study was a non-randomized study, so the question remained of how much the remarkable outcome of 19.7 months was due to the combination effect versus LuPSMA monotherapy alone," said Noxopharm CEO, Graham Kelly. "The pre-clinical study results confirmed that LuPSMA monotherapy had an impressive anti-cancer effect on tumor growth — but when Veyonda was added, the tumors mostly disappeared."