On November 4, 2021 Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and biologic cancer therapeutics, reported a collaboration with Bristol Myers Squibb on a clinical trial of a combination therapy using RGX-104 (abequolixron), the Company’s small molecule agonist of the Liver X Receptor/Apolipoprotein E ("LXR/APOE") pathway, and Yervoy (ipilimumab), Bristol Myers Squibb’s Cytotoxic T-Lymphocyte Associated protein 4 (CTLA-4) inhibitor (Press release, Inspirna, NOV 4, 2021, View Source [SID1234594520]). Under the terms of the agreement, Bristol Myers Squibb will provide ipilimumab for a Phase 1b/2 expansion study investigating the combination therapy for the 2nd and 3rd line treatment of patients with metastatic endometrial cancer whose genomes possess the E2 or E4 APOE genetic biomarker, including those patients who have progressed on prior checkpoint inhibitor therapy. Inspirna will sponsor the study and be responsible for study costs.

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RGX-104 is an orally administered LXR agonist that potently activates APOE protein expression in tumors to inhibit cancer progression. Approximately 40% of the human population harbors either the E2 or E4 variants of the APOE gene in their genomes. These APOE genetic variants can be readily identified by analyzing DNA from blood samples. The presence of either of the E2 or E4 variants has been shown to increase the likelihood of a favorable clinical response to RGX-104 treatment in some cancers, including endometrial cancer, in a Phase 1 clinical trial, thus providing a potential biomarker for RGX-104 therapy. RGX-104 is currently being investigated in a Phase 1b/2 clinical trial in combination with standard-of-care regimens in several lung cancer indications that are enriched for dysregulation of the APOE gene, including small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC).

"We are proud to embark on this collaboration with Bristol Myers Squibb to uncover an additional possible application of RGX-104 beyond our current areas of study," said Masoud Tavazoie, M.D., Ph.D., and Chief Executive Officer of Inspirna. "This collaboration validates the work we are doing with RGX-104, and follows our recent phase 1 combination dose escalation trial results, in which treatment with RGX-104 combination regimens resulted in a 60% response rate in patients with relapsed or refractory cancers that had progressed on prior checkpoint inhibitor therapy and possessed the E2 or E4 APOE genetic biomarkers. We look forward to working with Bristol Myers Squibb and unlocking the potential of RGX-104 as we aim to improve the lives of people suffering with cancer."

Yervoy is a trademark of Bristol Myers Squibb Company.

About RGX-104 (abequolixron)

RGX-104 (abequolixron) is an orally administered small molecule agonist of the Liver X Receptor (LXR) which activates expression of the APOE tumor suppressor protein. APOE expression becomes dysregulated (silenced) in the tumors of select patients with solid cancers. APOE dysregulation results in increased tumor angiogenesis (tumor blood vessel growth) as well as a shifting of the tumor myeloid cell population from immune-stimulatory to immune-suppressive, which are both counteracted by RGX-104. RGX-104 is currently being tested in a Phase 1b/2 clinical trial in combination with standard-of-care regimens in several lung cancer indications that are enriched for APOE dysregulation, including SCLC and NSCLC. Inspirna expects to present data from this clinical study in 2H 2022.

On November 4, 2021 Ambrx Biopharma Inc., or Ambrx, (NYSE: AMAM), a clinical stage biopharmaceutical company using an expanded genetic code technology platform to create Engineered Precision Biologics, reported the first patient has been dosed in its global ACE-Breast-03 Phase 2 clinical study of ARX788 in patients with HER2-positive metastatic breast cancer (Press release, Ambrx, NOV 4, 2021, View Source [SID1234594519]).

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ARX788 is an anti-HER2 antibody drug conjugate (ADC) that is being studied broadly in breast cancer, gastric/GEJ cancer and other solid tumors. The FDA has granted ARX788 Fast-Track Designation for the treatment of HER2-positive metastatic breast cancer in December 2020.

"Dosing the first patient in this Phase 2 study of ARX788 in patients with HER2-positive metastatic breast cancer marks an important milestone for Ambrx," said Feng Tian, Ph.D., Chairman of the Board, President, and CEO of Ambrx. "We have made excellent progress with our clinical development pipeline over the last few months, highlighted by our positive data of ARX788 for HER2-positive gastric cancer, as well as the dosing of the first patient in a Phase 1 trial of ARX517 for PSMA expressing tumors. Our growing clinical programs, coupled with an influx of capital from our IPO in June 2021, leaves Ambrx well-positioned to potentially attain several near-term clinical and corporate milestones."

The global ACE-Breast-03 Phase 2 clinical study is a multicenter study to evaluate the efficacy and safety of ARX788 in HER2-positive, metastatic breast cancer patients whose disease is resistant or refractory to T-DM1, and/or T-DXd, and/or tucatinib-containing regimens. The primary outcome measure of the study will be the objective response rate.

On November 4, 2021 Primmune Therapeutics reported that it has received $8.4 million in a second tranche of the Company’s Series A financing. The total proceeds for the equity raised in the Series A was $31.4 million (Press release, Primmune Therapeutics, NOV 4, 2021, View Source [SID1234594518]). These funds will be used to support the further clinical development of PRTX007 as a TherAjuvant for acute viral diseases, pre-cancerous lesions, and advanced cancer. PRTX007 is a novel orally-administered, small molecule toll-like receptor 7 (TLR7) agonist that has both therapeutic and adjuvant properties.

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"Given the initial results from our Phase 1 study in healthy volunteers, we achieved our target clinical milestone that triggered a second tranche of $8.4 million from our existing investors. These funds will be used to set the stage for Primmune’s expansion into multiple definitive efficacy studies," said Charlie McDermott, Chairman and Chief Executive Officer of Primmune Therapeutics. "In 2022, we intend to study PRTX007 in ambulatory respiratory syncytial virus (RSV), outpatient SARS-CoV-2, human papilloma virus (HPV) driven high-grade squamous intraepithelial lesions (HSIL) of the cervix, and in the neo-adjuvant setting in combination with checkpoint inhibitors in advanced cancer."

About TherAjuvants

Primmune Therapeutics coined the term TherAjuvants to reference its lead candidate PRTX007, a toll-like receptor 7 (TLR7) agonist with a combination of therapeutic and adjuvant mechanisms of action. PRTX007 is designed to provide immediate benefit to patients through controlled stimulation of the innate immune response while also potentiating long-term effective innate and adaptive immune responses. PRTX007 uniquely engages TLR7 and targeted immune cells without exacerbating inflammation, a critical feature in treating respiratory viral infections. TherAjuvants differ from therapeutic vaccines in that the source of the antigens presented to the patient’s immune system come from the treated pathology. Additionally, TherAjuvants differ from most small molecule approaches in that they target the patient’s immune system and not tumor cells or virally encoded targets.

On November 4, 2021 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, reported that Aviad Pato, Ph.D., Head of Immunology Research, presented data on two nicotinamide (NAM)-enabled NK cell therapies, GDA-501 and GDA-301, at the Protein & Antibody Engineering Summit (PEGS) Europe taking place in Barcelona, Spain, and virtually November 2-4, 2021 (Press release, Gamida Cell, NOV 4, 2021, View Source [SID1234594517]).

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The presentation included data from early-stage studies of GDA-501, Gamida Cell’s investigational cell therapy comprised of CAR-engineered NK cells designed to enhance homing and activation against cancers with HER2 overexpression such as breast, ovarian, lung, bladder, and gastric cancers. Data were also presented on GDA-301, which combines a CRISPR/Cas9 knockout of the CISH (cytokine inducible SH2 containing protein) gene in NK cells with a membrane-bound IL-15/IL-15Ra CAR, which is designed to improve tumor killing by promoting activation and inhibiting negative feedback signals and has potential application in a range of solid tumors and hematologic malignancies.

Data presented by Gamida Cell demonstrated that the engineered GDA-501 NK enhances potency and cytotoxicity against a HER2-expressing tumor cell line. Data also showed that GDA-301 has cytotoxic activity against a chronic myelogenous leukemia cell line (K562) and a multiple myeloma cell line (RPMI).

"The field of NK cell immunotherapy is advancing beyond what has previously been understood from T cell gene editing," said Yona Geffen, Ph.D., Vice President, Research and Development at Gamida Cell. "We are pleased to share this important update on two key potential therapies in Gamida Cell’s robust NK pipeline that show their potential as clinical immunotherapy agents."

The full presentation shared at PEGS Europe is available at www.gamida-cell.com.

On November 4, 2021 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer and population sequencing, reported financial results for the third quarter ended September 30, 2021 (Press release, Personalis, NOV 4, 2021, View Source [SID1234594516]).

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Third Quarter and Recent Highlights

Reported quarterly revenue of $22.3 million in the third quarter of 2021 compared with $19.8 million in the third quarter of 2020, a 12% increase
Record quarterly revenue of $8.6 million from biopharma and all other customers, excluding the U.S. Department of Veterans Affairs Million Veteran Program (VA MVP), in the third quarter of 2021 compared with $5.7 million in the third quarter of 2020, a 50% increase
Record new orders received for cancer genomic testing from biopharma and all other customers, excluding the VA MVP; order value of more than three-times (3x) the amount of quarterly revenue reported in the third quarter of 2021
Received new order from the VA MVP with an aggregate value of approximately $10 million over a six-month period of performance from September 17, 2021 to March 31, 2022
Announced a collaboration with Mayo Clinic to provide clinical-grade comprehensive cancer genomic sequencing for patients; creates framework for Personalis to sponsor future defined research studies and establishes Personalis as a preferred provider to Mayo Clinic for research and clinical sequencing and analysis services, particularly in the area of immuno-oncology
Announced the appointment of Robert Bruce to the newly created position of Vice President, Reimbursement; the new role is tasked with driving efforts with Medicare and private payers
Announced the signing of a new building lease agreement in Fremont, California for approximately 100K square feet for laboratory capacity and office space, which is planned to be the new company headquarters and support future growth
Ended the third quarter of 2021 with cash and cash equivalents and short-term investments of $305.2 million
"I’m proud to say that revenue from our oncology customers grew 50% over the same period of the prior year, and increased sequentially for the eighth consecutive quarter. In addition, our new-orders-to-revenue ratio during the third quarter was more than three-to-one, and gives us confidence in our future growth," said John West, Chief Executive Officer. "Looking ahead, and building on our recent oncology success, we are working to enhance our clinical, regulatory, and reimbursement capabilities as we prepare to launch NeXT Personal, our Minimal Residual Disease (MRD) offering in December as planned. We expect initial orders to come from our pharma customers and, later in 2022, we will pursue orders for patient diagnostic tests in clinical settings."

Third Quarter 2021 Financial Results

Revenue was $22.3 million in the three months ended September 30, 2021, up 12% from $19.8 million in the same period of the prior year.

Gross margin was 36.2% in the three months ended September 30, 2021, compared with 26.9% in the same period of the prior year.

Operating expenses were $25.8 million in the three months ended September 30, 2021, compared with $15.0 million in the same period of the prior year.

Net loss was $17.7 million in the three months ended September 30, 2021 and net loss per share was $0.40 based on a weighted-average basic and diluted share count of 44.5 million, compared with a net loss of $9.5 million and a net loss per share of $0.27 based on a weighted-average basic and diluted share count of 35.5 million in the same period of the prior year.

Business Outlook

Personalis expects the following for the fourth quarter of 2021:

Total revenue to be in the range of $20.2 million to $20.4 million
Revenue from biopharma and all other customers, excluding VA MVP, to be in the range of $12.5 million to $14.5 million
Net Loss to be in the range of $22 million to $23 million and estimated outstanding shares of approximately 45 million
Personalis expects the following for the full year of 2021:

Total revenue to be approximately $85 million
Revenue from biopharma and all other customers, excluding VA MVP, to be in the range of $37 million to $39 million
Net Loss to be in the range of $67 million to $68 million and estimated outstanding shares of approximately 45 million
Webcast and Conference Call Information

Personalis will host a conference call to discuss the third quarter 2021 financial results before market opens on Thursday, November 4, 2021 at 5:30 a.m. Pacific Time / 8:30 a.m. Eastern Time. The conference call can be accessed live over the phone by dialing (866) 220-8061 for U.S. callers or (470) 495-9168 for international callers, using the conference ID: 7896264. The live webinar can be accessed at View Source