On November 5, 2020 Ardelyx, Inc. (Nasdaq: ARDX), a specialized biopharmaceutical company focused on developing innovative first-in-class medicines to improve treatment for people with kidney and cardiovascular diseases, reported business highlights and financial results for the third quarter ended September 30, 2020 (Press release, Ardelyx, NOV 5, 2020, View Source [SID1234570235]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"The FDA’s acceptance of our New Drug Application for tenapanor is a major milestone that continues our progress toward the potential launch of this novel therapeutic for the many dialysis patients who struggle with controlling hyperphosphatemia," said Mike Raab, president and chief executive officer of Ardelyx. "Our commitment to this field was further highlighted in clinical data presented at ASN Kidney Week 2020 generated by Ardelyx and our Japanese partner KKC, supporting the clinical safety and efficacy of tenapanor and reinforcing its potential to transform the treatment landscape for patients."

Recent Business and Pipeline Updates

The United States Food and Drug Administration (FDA) accepted the New Drug Application (NDA) for tenapanor to control serum phosphorus in adult patients with chronic kidney disease (CKD) on dialysis with a Prescription Drug User Fee Act ("PDUFA") goal date of April 29, 2021. The filing was supported by three successful Phase 3 studies demonstrating tenapanor’s ability to reduce phosphate levels, with two trials evaluating tenapanor as a monotherapy and the third evaluating tenapanor as part of a dual mechanism approach with phosphate binders.

Presented new clinical data supporting the clinical safety and efficacy of tenapanor at ASN Kidney Week 2020. Three poster presentations highlighted data from Phase 3 trials conducted by Ardelyx, including the BLOCK, AMPLIFY and PHREEDOM studies. Additionally, the company’s partner for tenapanor in Japan, Kyowa Kirin Co., Ltd., presented the results from two Phase 2 studies evaluating the efficacy and safety of tenapanor in Japanese patients on hemodialysis.
Third Quarter 2020 Financial Results

Cash Position: As of September 30, 2020, Ardelyx had total cash, cash equivalents and short-term investments of $185.5 million, as compared to total cash, cash equivalents and short-term investments of $247.5 million as of December 31, 2019.

Revenue: The company generated $2.7 million in revenue during the three months ended September 30, 2020, which primarily represents collaborative development revenue and sales of tenapanor for clinical supply to KKC.

R&D Expenses: Research and development expenses were $12.2 million for the three months ended September 30, 2020, a decrease of approximately $5.4 million, or 30 percent, compared to $17.6 million for the three months ended September 30, 2019. The decrease was primarily due to the completion of the Phase 3 PHREEDOM and AMPLIFY clinical trials evaluating tenapanor for the control of hyperphosphatemia.

G&A Expenses: General and administrative expenses were $7.6 million for the three months ended September 30, 2020, an increase of $0.7 million, or approximately 10 percent, compared to $6.9 million for the three months September 30, 2019. The increase was primarily due to an increase in costs associated with building and staffing our commercial infrastructure and teams as we prepare for the anticipated U.S. launch of tenapanor for the control of serum phosphorus in CKD patients on dialysis.

Net Loss: Net loss for the quarter ended September 30, 2020 was $18.1 million, or ($0.20) per common share, as compared to $23.5 million, or ($0.37) per common share, for the quarter ended September 30, 2019.

On November 5, 2020 ThermoGenesis Holdings, Inc. (Nasdaq: THMO), a market leader in automated cell processing tools and services in the cell and gene therapy field, reported that the Company will release its financial results for the third quarter ended September 30, 2020 and provide a corporate strategic update on Thursday, November 12, 2020 after the close of trading (Press release, Thermogenesis, NOV 5, 2020, View Source [SID1234570234]). A conference call and webcast will follow at 1:30 p.m. PT/ 4:30 p.m. ET.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

A webcast replay will be available on ThermoGenesis’ website for three months by visiting the Investor page of the Company’s website at www.thermogenesis.com.

On November 5, 2020 Aeglea BioTherapeutics, Inc. (NASDAQ: AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, reported its third quarter 2020 financial results, and provided recent corporate and program highlights (Press release, Aeglea BioTherapeutics, NOV 5, 2020, View Source [SID1234570233]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"Although it’s been a challenging environment for biotech with the global pandemic, we have continued to be productive in our Arginase 1 Deficiency patient identification and engagement efforts. I am pleased with the progress we are making overall as well as specifically at our PEACE trial sites," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "I am also excited by the momentum we are building in our Homocystinuria program with the recent granting of U.S. Orphan Drug Designation and a positive opinion for EU Orphan Drug Designation for ACN00177."

Recent Highlights and Updates

Pegzilarginase in Arginase 1 Deficiency

In September, Aeglea announced the PEACE Phase 3 pivotal trial was more than 50% enrolled with nearly twice the number of patients needed to complete enrollment identified at active trial sites. Enrollment for the trial is anticipated to be completed in January 2021.
As of September, Aeglea has identified over 250 Arginase 1 Deficiency patients in addressable markets, a 25% increase relative to the prior year. The number of currently identified patients represents more than 50% and 30% of the estimated genetic prevalence populations in the U.S. and EU5, respectively.
ACN00177 in Homocystinuria

In October, Aeglea announced the U.S. Food and Drug Administration granted Orphan Drug Designation to ACN00177 for the treatment of Homocystinuria.
Additionally, the European Medicines Agency Committee for Orphan Medicinal Products issued a positive opinion recommending Orphan Drug Designation for ACN00177 for the treatment of Homocystinuria in the European Union.
Corporate

In October, the Company strengthened its financial position with proceeds from shares of common stock sold under its ATM program, resulting in gross proceeds of $25 million, extending its cash runway into 2023.
Upcoming Events

Aeglea will be attending the following virtual investor conferences in the fourth quarter:

Piper Sandler 32nd Annual Virtual Healthcare Conference, December 1-3
3rd Annual Evercore ISI HealthCONx Conference, December 1-3
Third Quarter 2020 Financial Results

As of September 30, 2020, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $141.5 million. In addition, in October 2020 the Company raised approximately $24.6 million in net proceeds from shares of common stock sold under its ATM program. Based on Aeglea’s current operating plan, and taking into account the net offering proceeds, management believes it has sufficient capital resources to fund anticipated operations into 2023.

Research and development expenses totaled $12.5 million for the third quarter of 2020 and $17.8 million for the third quarter of 2019. The decrease was primarily associated with completing certain manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase, completing a Phase 1/2 clinical trial in patients with Arginase 1 Deficiency and closing cancer trials; offset by a ramp-up in manufacturing for ACN00177 in Homocystinuria and higher personnel-related expenses.

General and administrative expenses totaled $5.7 million for the third quarter of 2020 and $4.3 million for the third quarter of 2019. This increase was primarily due to ramping-up commercial capabilities and additional facilities to support company growth.

Net loss totaled $18.0 million and $21.6 million for the third quarter of 2020 and 2019, respectively, with non-cash stock compensation expense of $1.7 million and $1.4 million for the third quarter of 2020 and 2019, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically lowers levels of the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating, progressive disease presenting in childhood with persistent hyperargininemia, spasticity, developmental delay, intellectual disability, seizures and early mortality. Pegzilarginase is intended for use as an enzyme therapy to reduce elevated blood arginine levels in patients with ARG1-D. Aeglea’s Phase 1/2 and Phase 2 open-label extension data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company’s single, global pivotal Phase 3 PEACE trial is designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.

About ACN00177 in Homocystinuria

Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities including severe osteoporosis, developmental delay, intellectual disability, lens dislocation and severe near-sightedness. ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. With this mechanism, ACN00177 is intended to lower the abnormally high blood levels of homocysteine in patients with Homocystinuria. Preclinical data demonstrated that ACN00177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. The Company initiated a Phase 1/2 trial in the second quarter of 2020 and continues patient identification and administrative activities. The timing of first patient dosing in this Phase 1/2 trial will depend on determinations by individual sites as they adjust to impacts from COVID-19. ACN00177 has been granted Orphan Drug Designation by the U.S. FDA and received a positive opinion on Orphan Drug Designation from the European Medicines Agency.

On November 5, 2020 Cellecta, Inc. reported the launch of its new RNA Spatial Profiling Service using NanoString’s GeoMx Cancer Transcriptome Atlas (CTA) on the GeoMx Digital Spatial Profiler (Press release, Cellecta, NOV 5, 2020, View Source [SID1234570231]). As one of the few designated GeoMx Premier Access Site Partners worldwide, Cellecta offers immediate access to spatial transcriptomic analysis using the NanoString CTA and, upon release, to the GeoMx Whole Transcriptome Atlas.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Researchers are now able to send fresh-frozen or formalin-fixed paraffin-embedded (FFPE) tissue slides to Cellecta for gene expression profiling and spatial analysis to readily obtain comprehensive gene expression profiles of microenvironments and localized regions within tumors and related fixed tissues, as well as corresponding pathway analysis in a variety of visualization formats.

"With the increasing number of researchers interested in assessing the utility of spatial transcriptomics for their tumor samples, we are pleased to welcome Cellecta into our global service network as a GeoMx Premier Access Partner," said Chad Brown, senior vice president of sales and marketing at NanoString. "Their proven skills with RNA expression profiling and functional genomic analysis will be of great benefit to those seeking a greater understanding of tumor biology."

"We are honored to be counted among the handful of GeoMx Premier Access Partners chosen to offer this novel spatial analysis technology to cancer researchers in academia and industry," said Alex Chenchik, Ph.D., president and chief scientific officer of Cellecta. "Given our experience working with RNA throughout the years and especially with our DriverMap Targeted RNA-Seq technology, Digital Spatial Profiling is the logical next step in providing our customers with more comprehensive transcriptomic data to enable valuable research insights that advance the understanding of disease progression and drug development."

For more information Cellecta’s RNA Spatial Profiling Service, visit View Source

On November 5, 2020 OncoSec Medical Incorporated (NASDAQ:ONCS) (the "Company" or "OncoSec") reported that it will host an Investor and Analyst webinar on Wednesday, November 11, 2020 from 8:30 am ET – 10:00 am ET showcasing interim data from its KEYNOTE-695 registration-enabled Phase 2b clinical trial investigating TAVO (tavokinogene telseplasmid), a DNA plasmid-based interleukin-12 (IL-12), in combination with KEYTRUDA (pembrolizumab) in patients with anti-PD-1 checkpoint refractory metastatic melanoma (Press release, OncoSec Medical, NOV 5, 2020, View Source [SID1234570230]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Additional topics will include an update on its KEYNOTE-890 Phase 2 clinical trial investigating TAVO in patients with metastatic triple negative breast cancer (mTNBC) as well as a discussion on CORVax12, the Company’s novel DNA-encodable vaccine for COVID-19. CORVax12 combines TAVO with the National Institute of Health’s DNA-encodable stabilized trimeric SARS-CoV-2 spike glycoprotein and is expected to enter a Phase 1 clinical trial in the immediate future given recent IND acceptance.

Experts will also share insights about OncoSec’s new neoadjuvant melanoma clinical trial and opinions on the visceral lesion applicator program, which will look to the future of gene electrotransfer in both lung and liver tumors. A live question and answer session will follow the formal presentations.

This webcast is intended for institutional investors, sell-side analysts, and business development professionals.

To register for the investor and analyst day, please click here. For those who cannot participate, the event will be recorded and available on the company website.

The event will feature presentations by the following Key Opinion Leaders (KOLs):

Tara Mitchell, MD, Associate Professor of Medicine, will describe the challenges of treating patients with checkpoint refractory metastatic melanoma. Dr. Mitchell will also give her initial review of the KEYNOTE-695 data.
Alain Algazi, MD, Associate Professor, Department of Medicine and Adil Daud, MD, Professor of Clinical Medicine, UCSF Helen Diller Family Comprehensive Cancer Center, will present and evaluate the KEYNOTE-695 clinical data and how it compares to other current treatments.
Matteo Carlino, MD, Medical Oncologist and Clinical Senior Lecturer, Westmead and Blacktown Hospitals and University of Sydney, will provide his interpretation of the KEYNOTE-695 data and comment on how it will impact the way he treats patients.
Ahmad Tarhini, MD, PhD, Director, Cutaneous Clinical and Translational Research, Moffitt Cancer Center, will provide an update on the ongoing neoadjuvant study combining TAVO and OPDIVO.
Bernard Fox, PhD, Harder Family Chair for Cancer Research, Member and Chief of the Laboratory of Molecular and Tumor Immunology, Earle A. Chiles Research Institute, will provide an overview of the trial design for CORVax12, a COVID-19 vaccine. He will also comment on the strategy behind this vaccine.
Rohit Joshi, MBBS, MD, FRACP, FACP, Medical Oncologist, Adelaide Medical School and Calvary Central Districts Hospital, will provide an overview of the KEYNOTE-890 study in triple negative breast cancer (TNBC).
Dan Simon, MD, Interventional Radiologist Chief, Maryland Cardiology Associates, will provide an overview of the Visceral Lesion Applicator program. He will share his experience with the technology and provide insight as to what this means for the future of gene electrotransfer.
About KEYNOTE-695

KEYNOTE-695 is OncoSec’s registration-directed Phase 2b trial (NCT#03132675) evaluating TAVO (tavokinogene telseplasmid), a DNA plasmid-based interleukin-12 (IL-12) + KEYTRUDA (pembrolizumab) in patients with rigorously confirmed anti-PD-1 checkpoint resistant metastatic melanoma. The trial aims to enroll up to 100 patients with refractory, locally advanced or metastatic disease defined as unresectable Stage III/IV metastatic melanoma that had definitively progressed on a full-course of anti-PD-1 treatment with KEYTRUDA (pembrolizumab) or OPDIVO (nivolumab). TAVO has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for the treatment of metastatic melanoma following progression on KEYTRUDA or OPDIVO.

About TAVO

OncoSec’s gene therapy technology combines TAVOTM (tavokinogene telseplasmid), a DNA plasmid-based interleukin-12 (IL-12), with an intra-tumoral electroporation gene delivery platform to achieve endogenous IL-12 production in the tumor microenvironment that enables the immune system to target and attack tumors throughout the body. TAVO has demonstrated a local and systemic anti-tumor response in several clinical trials, including the pivotal Phase 2b trial KEYNOTE-695 for metastatic melanoma and the KEYNOTE-895 Phase 2 trial in triple negative breast cancer (TNBC). TAVO has received Orphan Drug and Fast-Track Designation by the U.S. Food & Drug Administration (FDA) for the treatment of metastatic melanoma following progression on KEYTRUDA or OPDIVO.

About Advanced Metastatic Melanoma

Metastatic melanoma refers to stage IV melanoma, which has typically spread through the lymph nodes to distant sites in the body such as the liver, lungs, bones and brain. Every year, approximately 100,000 adults in the United States are diagnosed with metastatic melanoma. Due to this metastatic tumor burden, stage IV melanoma is often very difficult to treat. Available treatment options frequently combine surgery with immunotherapy or targeted therapy. The 5-year survival rate for metastatic melanoma is approximately 25%.

About Metastatic Triple Negative Breast Cancer (TNBC)

Metastatic triple negative breast cancer (mTNBC) is an aggressive type of breast cancer with a high recurrence rate within the first five years following diagnosis, which accounts for 10-20% of all breast cancers. Unlike some other breast cancers, mTNBC does not express estrogen or progesterone receptors or human epidermal growth factor receptor 2 (HER2), and it does not respond to existing cancer drugs designed to target these markers. mTNBC is difficult to treat and there are very few FDA approved treatment options for these patients, which mostly rely on surgery, radiation, and chemotherapy. The 5-year survival rate for these patients is approximately 11%.

About CORVax12

CORVax12 is the only DNA vaccine that uses an immune stimulant to promote an immune response against the SARS-CoV-2 virus. The CORVax12 vaccine approach combines the co-administration of TAVO (plasmid IL-12) with a DNA-encodable version of the SARS-CoV-2 spike or "S" glycoprotein to enhance immunogenicity of the component developed by scientists at the National Institute of Allergy and Infectious Diseases (NIAID) Vaccine Research Center. CORVax12 is designed to drive a coordinated vaccine response, capable of drawing upon the innate and adaptive humoral and cellular arms. This multi-pronged innate, adaptive and cellular immune response has the potential to generate a robust anti-viral response.