On May 5, 2021 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported financial results for the first quarter ended March 31, 2021 (Press release, Tracon Pharmaceuticals, MAY 5, 2021, View Source [SID1234579391]). The Company will host a conference call and webcast today at 4:30 PM Eastern Time / 1:30 PM Pacific Time.

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"We continue to be pleased with the pace of enrollment in the pivotal ENVASARC trial and remain on track to deliver interim data in the 2nd half of this year and final data in 2022," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "We have now initiated 22 sites and have enrolled more than 20 patients which has triggered the initial Data Monitoring Committee review of safety data from each cohort, which we expect later this quarter."

Recent Corporate Highlights

Envafolimab

In April, we resubmitted our Orphan Drug Designation application to the FDA in response to a request for preclinical or clinical evidence of activity for envafolimab in sarcoma. We expect correspondence from the FDA this quarter based on the amended application.

As of May 5, we have initiated 22 U.S. clinical sites and enrolled more than 20 patients in the pivotal ENVASARC trial of single agent envafolimab and envafolimab combined with Yervoy, which has triggered the initial Data Monitoring Committee review of safety data from each cohort.
Expected Key Upcoming Milestones

Orphan Drug Designation for envafolimab in soft tissue sarcoma from FDA in 1H 2021.

Independent Data Monitoring Committee review of ENVASARC safety data in 1H 2021.

American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) presentation of ENVASARC pivotal trial design in 1H 2021.

ASCO presentation of TJ004309 Phase 1 data in 1H 2021.

Interim ENVASARC efficacy and safety data in 2H 2021.

Request FDA breakthrough therapy designation or Fast Track designation for envafolimab in 2H 2021.

Decision on the envafolimab New Drug Application (NDA) in MSI-H/dMMR cancer that is under priority review by the Chinese National Medical Products Administration (NMPA).
First Quarter 2021 Financial Results

Cash, cash equivalents and short-term investments were $30.4 million at March 31, 2021, compared to $36.1 million at December 31, 2020. The Company expects that its current cash, cash equivalents and short-term investments will fund operations into the second half of 2022.

Research and development expenses for the first quarter of 2021 were $2.3 million, compared to $2.0 million for the first quarter of 2020.

General and administrative expenses for the first quarter of 2021 were $2.7 million, compared to $1.9 million for the first quarter of 2020.

Net loss for the first quarter of 2021 was $5.1 million, compared to $4.0 million for the first quarter of 2020.
Conference Call Details

Wednesday, May 5, at 4:30 PM Eastern Time / 1:30 PM Pacific Time
Domestic: 855-779-9066
International: 631-485-4859
Conference ID: 8852857
A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About Envafolimab

Envafolimab (KN035), a novel, single-domain antibody against PD-L1, is the first subcutaneously injected PD-(L)1 inhibitor to be studied in pivotal trials. Envafolimab is currently being studied in the ENVASARC Phase 2 pivotal trial in the U.S. sponsored by TRACON, has been studied in a completed Phase 2 pivotal trial as a single agent in MSI-H/dMMR advanced solid tumor patients in China and is being studied in an ongoing Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China, with both Chinese trials sponsored by 3D Medicines. TRACON’s partners Alphamab Oncology and 3D Medicines submitted an NDA to the NMPA in China for envafolimab in MSI-H/dMMR cancer that was accepted for review in December 2020 and granted priority review in January 2021. In the Phase 2 MSI-H/dMMR advanced solid tumor trial, the confirmed objective response rate (ORR) by blinded independent central review in MSI-H/dMMR colorectal cancer (CRC) patients treated with envafolimab who failed a fluoropyrimidine, oxaliplatin and irinotecan was 32%, which was similar to the 28% confirmed ORR reported in the Opdivo package insert in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin, and irinotecan and the 33% confirmed ORR reported for Keytruda in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin and irinotecan in cohort A of the KEYNOTE-164 clinical trial.

About ENVASARC (NCT04480502)

The ENVASARC pivotal trial is a multi-center, open label, randomized, non-comparative, parallel cohort study at approximately 25 top cancer centers in the United States that began dosing in December 2020. TRACON expects the trial to enroll 160 patients with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor, with 80 patients enrolled into cohort A of treatment with single agent envafolimab and 80 patients enrolled in cohort B of treatment with envafolimab and Yervoy. The primary endpoint is ORR by blinded independent central review with duration of response a key secondary endpoint.

About TRC102

TRC102 (methoxyamine) is a novel, small molecule inhibitor of the DNA base excision repair pathway, which is a pathway that causes resistance to alkylating and antimetabolite chemotherapeutics. TRC102 is currently being studied in multiple Phase 1 and Phase 2 clinical trials sponsored by the National Cancer Institute through a Cooperative Research and Development Agreement (CRADA) and has orphan drug designation from the U.S. FDA in malignant glioma, including glioblastoma.

About TJ004309

TJ004309 is a novel, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine, which is highly immunosuppressive. TJ004309 is currently being studied in an ongoing Phase 1 trial to assess safety and preliminary efficacy as a single agent and when combined with the PD-L1 checkpoint inhibitor Tecentriq in patients with advanced solid tumors.

Merck & Co has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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On May 11, 2021Appia Bio, Inc., an early stage biotechnology company developing engineered allogeneic cell therapies from hematopoietic stem cells (HSCs) for cancer patients, reported its launch from stealth backed by $52 million Series A financing led by 8VC (Press release, Appia Bio, MAY 5, 2021, View Source [SID1234583854]). Other investors included Two Sigma Ventures, among others, and participation from seed investors Sherpa Healthcare Partners and Freeflow Ventures.

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Appia Bio is focused on discovering and developing off-the-shelf allogeneic cell therapies across a broad array of cancer indications, utilizing a scalable technology platform with the goal to increase access for patients. With its ACUA technology platform, Appia Bio leverages the biology of lymphocyte development with chimeric antigen receptor (CAR) and T-cell receptor (TCR) gene engineering to generate CAR-engineered invariant natural killer T (CAR-iNKT) cells from HSCs. The ACUA platform originated from groundbreaking research in the laboratory of Lili Yang, PhD, associate professor at University of California, Los Angeles (UCLA), and her collaborations with the Company’s other scientific founders.

Proceeds from the financing will support the advancement of Appia Bio’s pipeline of allogeneic CAR-iNKT cell therapy candidates into the clinic. As part of the Series A financing, Francisco Gimenez, PhD, partner, and David Moskowitz, PhD, principal, at 8VC, join the Company’s board of directors, and David Baltimore, PhD, of the California Institute of Technology (Caltech) was elected chairman of the Board.

"There is tremendous potential to be explored in allogeneic cell therapies. The Board and I look forward to working with this stellar team to create transformative cell therapies that will help patients and the physicians who treat them," said Dr. Baltimore.

"We are grateful to our new and existing investors for their commitment to bring off-the-shelf allogeneic cell therapies to more patients," said JJ Kang, PhD, co-founder and chief executive officer (CEO) of Appia Bio. "This financing will position us to rapidly build out our CAR-iNKT pipeline and establish the clinical potential of our ACUA platform. We are excited to work with an outstanding Scientific Advisory Board that brings together world-class expertise and experience across the many fields necessary to develop cell therapies."

Appia Bio announced the establishment of a Scientific Advisory Board with leaders in immunology, immuno-oncology, and cell therapy engineering. These experts join Appia Bio’s scientific founders in advising the Company’s research and development.

Mitchell Kronenberg, PhD, president and CSO, La Jolla Institute for Immunology

Antoni Ribas, MD, PhD, professor of medicine, UCLA, and director of the Tumor Immunology Program at the Jonsson Comprehensive Cancer Center

Margo Roberts, PhD, former CSO, Kite Pharma and Lyell Immunopharma

"We are thrilled to partner with Appia Bio as it typifies the innovative, founder-driven start-ups we love to empower," said Dr. Gimenez. "Appia unifies compelling biology of iNKT cells with the power of CARs. This is only possible because of their fundamental innovations in manufacturing and engineering these cells."

The Company was founded in 2020 in Los Angeles, California, by a team of experts and industry leaders with a proven track record of cell therapy development and company building.

David Baltimore, PhD, Nobel Laureate, president emeritus and distinguished professor of biology, Caltech

JJ Kang, PhD, CEO, Appia Bio; former partner, The Column Group

Edmund Kim, PhD, chief operating officer of Appia Bio; former vice president of corporate development, Kite Pharma

Pin Wang, PhD, professor of chemical engineering and materials science and biomedical engineering, University of Southern California (USC)

Jeff Wiezorek, MD, chief medical officer, Appia Bio; former head of cell therapy development, Kite Pharma

Lili Yang, PhD, associate professor of microbiology, immunology, and molecular genetics, UCLA

On May 5, 2021 Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported financial results and corporate highlights for the first quarter 2021, as well as anticipated corporate milestones for the second quarter 2021 (Press release, Surface Oncology, MAY 5, 2021, View Source [SID1234579210]).

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"The first quarter of 2021 marked a significant transition for Surface, and I am honored to become CEO during this very exciting time," said Rob Ross, M.D., chief executive officer. "In June, we will be sharing data with the medical community and investors regarding the clinical progress we have made with our Phase 1 trials of both SRF617 and SRF388. We believe that both of these novel agents have the potential to improve outcomes for patients with cancer, and the data we share in June will be the first step in illustrating this potential."

Recent Corporate Highlights:

On February 11, 2021, Surface announced that Rob Ross, chief medical officer, would succeed Jeff Goater as CEO, effective April 1, 2021.

On March 8, 2021, Surface entered into a clinical trial collaboration with Merck, known as MSD outside the United States and Canada, through a subsidiary, to evaluate the safety and efficacy of combining Surface’s SRF388, an investigational antibody therapy targeting IL-27, with Merck’s KEYTRUDA (pembrolizumab), the first anti-PD-1 therapy approved in the United States. This combination will be studied as a component of the first-in-human Phase 1 study of SRF388 and will be evaluated in patients with solid tumors, with a focus on patients with liver cancer and kidney cancer.

On March 29, 2021, the U.S. Food and Drug Administration granted Orphan Drug Designation for SRF617 for the treatment of patients with pancreatic cancer.

Surface presented updated preclinical data on lead candidates SRF617 and SRF388 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2021 Annual Meeting, held virtually April 10-15. The poster presentations can be found in the Posters & Publications section of our website.

On April 27, 2021, Surface announced the appointment of Henry Rath as chief business officer and the promotions of Alison O’Neill, M.D. to chief medical officer, and Jessica Fees to chief financial officer.
Selected Anticipated Near-term Corporate Milestones:

SRF388 clinical data presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Annual Meeting, to be held virtually June 4-8, 2021.
Webcast to provide data from ongoing SRF388 and SRF617 Phase 1 clinical studies, to be held on Friday, June 4, 2021 at 8:00 a.m. ET.
Investigational new drug (IND) filing for SRF813, partnered with GlaxoSmithKline, anticipated in 2021.
Financial Results:

As of March 31, 2021, cash, cash equivalents and marketable securities were $171.0 million, compared to $175.1 million on December 31, 2020.

Research and development (R&D) expenses were $10.5 million for the first quarter ended March 31, 2021, compared to $11.3 million for the same period in 2020. This decrease was primarily driven by reduction in headcount as well as decreased facility and lab costs as a result of the strategic restructuring announced in January 2020, partially offset by increased clinical costs as a result of progression in both our SRF617 and SRF388 Phase 1 clinical trials. R&D expenses included $0.8 million in stock-based compensation expense for the first quarter ended March 31, 2021.

General and administrative (G&A) expenses were $5.6 million for the first quarter ended March 31, 2021, compared to $4.8 million for the same period in 2020. This increase was primarily due to increases in personnel- and facility-related costs, including $0.4 million of stock-based compensation resulting from modifications to previously issued stock option awards in connection with the transition of our chief executive officer to chairman of the board. G&A expenses included $1.6 million in stock-based compensation for the first quarter ended March 31, 2021.

For the first quarter ended March 31, 2021, net loss was $15.6 million, or basic and diluted net loss per share attributable to common stockholders of $0.37. Net income was $22.6 million for the same period in 2020, or basic net income per share attributable to common stockholders of $0.81 and diluted net income per share attributable to common stockholders of $0.74.

Financial Outlook:

Surface Oncology continues to project that current cash, cash equivalents and an anticipated near-term milestone from GSK are sufficient to fund the Company through 2023.

On May 5, 2021 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported data on its 15-gene expression profile (15-GEP) test, DecisionDx-UM, at the Association for Research in Vision and Ophthalmology (ARVO) 2021: Revolutionary Eye and Vision Research Meeting (Press release, Castle Biosciences, MAY 5, 2021, View Source [SID1234579228]).

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The virtual poster is entitled "Uveal Melanoma Patient Attitudes Towards Prognostic Testing Using Gene Expression Profiling."

DecisionDx-UM, the test highlighted in the poster, is Castle’s prognostic 15-GEP test for patients with uveal melanoma, a rare cancer of the eye that carries a high risk of spreading (metastasizing). The DecisionDx-UM test is designed to accurately identify patients who are at low risk (Class 1) or high risk (Class 2) of metastasis based on the unique biology of their primary tumor and is the current standard of care in the management of uveal melanoma at the majority of U.S. ocular oncology practices.

"Up to half of patients diagnosed with uveal melanoma will experience metastatic disease, and prior studies show that newly diagnosed patients have overwhelmingly been in favor of learning their prognoses," said first author Basil K. Williams, M.D., assistant professor and director of Ocular Oncology at the University of Cincinnati College of Medicine. "This study demonstrated that uveal melanoma patients were satisfied with their decisions to pursue prognostic information through GEP testing, and they found particular value in DecisionDx-UM’s ability to help them understand their individual metastatic risk."

Study methods and findings:

The objective of the patient-based study was to understand uveal melanoma patients’ experiences following testing with DecisionDx-UM compared to patients with alternative or no prognostic testing.
An online questionnaire was distributed by the Melanoma Research Foundation’s CURE OM (Ocular Melanoma) initiative that captured de-identified information regarding patient-reported experiences. Patients were asked questions regarding the decision to undergo prognostic testing and the extent to which they felt regret about their decisions.
Of the 177 survey participants, 159 (90%) reported wanting prognostic information at diagnosis.
Of patients tested with DecisionDx-UM, the vast majority (80/81 respondents, 99%) reported gaining value from their test result, including:
Increased knowledge and understanding
More personalized treatment options
Information relevant to life planning
A sense of relief from uncertainty about the future
Of the patients who received prognostic testing with DecisionDx-UM, decision regret levels did not differ depending on whether they received a low or high-risk test result (Kruskal-Wallis; n=28, 23, 30 for 1A, 1B, 2; p=0.13).
Patients who received prognostic testing experienced lower levels of decision regret than those who opted out of testing, independent of which prognostic tests were used (Wilcoxon Rank-Sum tests: DecisionDx-UM vs. alternative tests: p=0.89, DecisionDx-UM vs. opt-out: p=0.0002, alternative tests vs. opt-out: p=0.003).