On December 10, 2020 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 dosing of the first patient in the ENVASARC registration trial (Press release, Tracon Pharmaceuticals, DEC 10, 2020, View Source [SID1234572606]).

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"We are pleased to initiate dosing in the ENVASARC registration trial of envafolimab in sarcoma," said Sant Chawla, M.D., Director of the Sarcoma Oncology Center, Santa Monica. "Immunotherapy has radically changed the treatment paradigm for a number of cancers, and we believe envafolimab has the potential to do the same for sarcoma patients who have few treatment options."

"Dosing the first patient in the ENVASARC registration trial within one year of executing the license to envafolimab fulfills our 2020 expectations for what has been a productive year of clinical development and regulatory interactions for our lead product candidate," said Charles Theuer, M.D., Ph.D., President and Chief Executive Officer of TRACON. "We look forward to the availability of interim top-line data from this important study, which we expect in mid-2021."

About ENVASARC (NCT 04480502)

Key elements of the ENVASARC registration trial include:

Multi-center, open-label, randomized, non-comparative, parallel cohort study at approximately 25 top cancer centers in the United States.
Eligible patients will have undifferentiated pleomorphic sarcoma (UPS) or myxofibrosarcoma (MFS) and received one or two prior cancer therapies, but no prior immune checkpoint inhibitor therapy.
Planned total enrollment of 160 patients, 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.
Primary endpoint of objective response rate (ORR) with duration of response a key secondary endpoint.
Open-label format with blinded independent central review of efficacy endpoint data.
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 registration trials. Envafolimab is currently being studied in the ENVASARC Phase 2 registration trial in the U.S. sponsored by TRACON, as well as in a Phase 2 registration trial as a single agent in MSI-H/dMMR advanced solid tumor patients and a Phase 3 registration trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China sponsored by TRACON’s corporate partners, Alphamab Oncology and 3D Medicines. Alphamab Oncology and 3D Medicines submitted an NDA to the NMPA in China for envafolimab in MSI-H/dMMR cancer in November 2020. In the Phase 2 registration 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 KEYNOTE-164.

On December 10, 2020 Agendia, Inc., a world leader in precision oncology for breast cancer, reported data from the APHINITY trial in a poster spotlight discussion at the 2020 San Antonio Breast Cancer Symposium (SABCS 2020) (Press release, Agendia, DEC 10, 2020, View Source [SID1234572622]).

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The poster, titled Prediction of benefit from adjuvant pertuzumab by BluePrint RNA sequencing in the APHINITY trial, outlines data from an exploratory analysis of the Phase 3 study that randomized 4,805 patients with histologically centrally confirmed HER2+ early breast cancer into two arms, one which received standard adjuvant chemotherapy and trastuzumab plus pertuzumab, and one which received standard adjuvant chemotherapy and trastuzumab plus placebo, for one year. The analysis was conducted in collaboration with Roche Pharmaceuticals.

At median follow up of 45 months, the primary analysis of the study showed a significant invasive disease-free survival benefit for patients treated with pertuzumab. In this analysis, BluePrint, Agendia’s 80-gene molecular subtyping test, was used to evaluate a subset of tumor samples to identify which patients in the APHINITY trial population would benefit from the addition of pertuzumab based on gene expression profiling of their tumors. The poster presented at SABCS showed that BluePrint may identify subgroups of patients within the study population with differing degrees of benefit from the addition of pertuzumab based on gene expression.

"What is exciting about these data is that we are going back to look deeper at the genomic makeup of tumors in the APHINITY cohort to better understand how these cancers respond to therapy," said Ian Krop, M.D., Ph.D., Associate Chief, Division of Breast Oncology at the Dana-Farber Cancer Institute, Associate Professor of Medicine at Harvard Medical School, and first author on the poster. "It reveals interesting trends that need to be studied further but could ultimately provide information to make more precise treatment decisions right at diagnosis."

With further research, this trend could offer HER2+ breast cancer patients and their doctors early information at the beginning of their journeys that could help them define the path ahead.

"The findings from the APHINITY trial are encouraging and warrant further investigation to see if offering genomic profiling to clinically HER2+ patients routinely could have a meaningful impact on their outcomes," said Adam Brufsky, M.D., Ph.D., Medical Director of the Magee-Womens Cancer Program, part of the UPMC Hillman Cancer Center, and Professor of Medicine at the University of Pittsburgh School of Medicine.

These data are part of a large suite of 13 posters, spotlight sessions and an oral presentation on MammaPrint and BluePrint that were accepted to SABCS 2020, and underscore Agendia’s mission to help guide the diagnosis and personalized treatment of breast cancer for all patients throughout their treatment journey.

On December 10, 2020 Protagonist Therapeutics, Inc. (Nasdaq: PTGX), a clinical stage biopharmaceutical company, reported that it has commenced an underwritten public offering of $100,000,000 of shares of its common stock (Press release, Protagonist, DEC 10, 2020, View Source [SID1234572638]). All of the shares of common stock are being offered by Protagonist. In connection with this offering, Protagonist expects to grant the underwriters a 30-day option to purchase up to an additional 15% of the number of shares of its common stock offered in the public offering.

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J.P. Morgan Securities LLC, SVB Leerink LLC, Piper Sandler & Co. and BMO Capital Markets Corp. are acting as joint book-running managers for the offering. The offering is subject to market conditions and there can be no assurance as to whether or when the offering may be completed or the actual size or terms of the offering.

A shelf registration statement relating to the offered shares of common stock was filed with the Securities and Exchange Commission (SEC) on December 10, 2020. A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website, located at www.sec.gov. Prospective investors should read the preliminary prospectus supplement, when available, and the accompanying prospectus and other documents Protagonist has filed with the SEC for more complete information about Protagonist and the offering. Copies of the prospectus supplement and the accompanying prospectus related to the offering may be obtained, when available, from J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204 or by email at [email protected]; from SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132 or by email at [email protected]; from Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924 or by email at [email protected]; or from BMO Capital Markets Corp., Attn: Equity Syndicate Department, 3 Times Square, 25th Floor, New York, NY 10036, tel: (800) 414-3627, email: [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On December 10, 2020 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, reported achievement of fifty percent enrollment in the IMerge Phase 3 clinical trial of imetelstat in lower risk myelodysplastic syndromes (MDS) (Press release, Geron, DEC 10, 2020, View Source [SID1234572607]). Data from the IMerge Phase 2 were recently presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and support the ongoing Phase 3.

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"Reaching fifty percent enrollment is a key milestone towards the completion of this registration-enabling Phase 3 clinical trial, and we appreciate all of the support from our investigators and the patients who are participating in this study," said Aleksandra Rizo, M.D., Ph.D., Geron’s Chief Medical Officer. "We believe that the IMerge Phase 3 will confirm the meaningful and durable transfusion independence and the disease-modifying activity of imetelstat observed from the Phase 2, and that imetelstat could become a much-needed treatment alternative for patients with lower risk MDS."

The IMerge Phase 3 is a double-blind, randomized, placebo-controlled clinical trial with registration intent. The trial is designed to enroll approximately 170 transfusion dependent patients with Low or Intermediate-1 risk MDS, or lower risk MDS, who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). The primary endpoint is the rate of red blood cell (RBC) transfusion independence (TI) for a consecutive period of eight weeks or longer, or 8-week RBC-TI rate. Key secondary endpoints include the rate of RBC-TI of at least 24 weeks, or 24-week RBC-TI rate, rate of hematologic improvement-erythroid (HI-E), defined as a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden, and duration of transfusion independence.

The Company continues to expect full enrollment in the IMerge Phase 3 in the second quarter of 2021. As long as enrollment is completed by the end of the first half of 2021, Geron maintains its projection of top-line results from IMerge to be available in the second half of 2022.

To learn more about IMerge and whether the study is enrolling patients in your area, please visit www.clinicaltrials.gov.

About Myelodysplastic Syndromes (MDS)

Myelodysplastic syndromes is a group of blood disorders in which the proliferation of malignant progenitor cells produces multiple malignant cell clones in the bone marrow resulting in disordered and ineffective blood production. There are approximately 60,000 people in the United States living with the disease and approximately 16,000 reported new cases of MDS in the United States every year. The majority of patients, approximately 70%, fall into the lower risk groups at diagnosis, according to the International Prognostic Scoring System that takes into account the presence of a number of disease factors, such as cytopenias and cytogenetics, to assign relative risk of progression to acute myeloid leukemia (AML) and overall survival.

Chronic anemia is the predominant clinical problem in patients who have lower risk MDS. Due to low hemoglobin, many of these patients become dependent on red blood cell transfusions which can lead to iron overload, higher risk of development AML and poorer overall survival. Currently, no drug therapy has been shown prospectively to alter or delay disease progression.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data strongly suggest that imetelstat has disease-modifying activity through the apoptosis of malignant stem and progenitor cells, which allows potential recovery of normal hematopoiesis. Current clinical studies of imetelstat include IMerge, an ongoing Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS), and IMpactMF, an upcoming Phase 3 clinical trial in refractory myelofibrosis (MF). Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.

On December 10, 2020 Molecular diagnostics company Lucence reported that the company’s Palo Alto laboratory received certification from the U.S. Department of Health and Human Services’ Centers for Medicare & Medicaid Services (CMS) under the Clinical Laboratory Improvement Amendments (CLIA) of 1988 (Press release, Lucence, DEC 10, 2020, View Source [SID1234572623]). With this accreditation, Lucence will rapidly scale United States-based testing capacity for its flagship liquid biopsy blood test, LiquidHALLMARK, furthering the Company’s mission of advancing precision cancer care for the benefit of patients everywhere.

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Dr. Tan Min-Han, Founding CEO of Lucence, said, "We are excited to expand our operations to offer the benefits of highly accurate amplicon-based liquid biopsy — quicker, more precise biomarker detection and less invasive testing — to even more patients. Our goal is to make non-invasive, ultrasensitive detection of clinically relevant biomarkers more accessible to oncologists across the country."

This milestone marks accelerating momentum in the company’s US operations. Early next year, Lucence will launch its first major prospective, observational study. LIQUIK, Liquid Biopsy for Detection of Actionable Genomic Biomarkers in Patients with Advanced Non-small Cell Lung Cancer, will compare different liquid biopsy technologies with conventional tissue-based profiling in patients with newly diagnosed metastatic non-squamous non-small cell lung cancer to evaluate the clinical utility of LiquidHALLMARK.

LiquidHALLMARK covers a wide range of clinically relevant biomarkers, including mutations in 80 genes, fusions in 10 genes, and somatic variants in 15 cancer types. LiquidHALLMARK is powered by AmpliMARK, the Company’s proprietary amplicon-based sequencing technology, which uses a unique molecular barcode and error-correction technology that improves test sensitivity across multiple mutation types for single nucleotide variants and fusion genes. AmpliMARK is the foundational technological innovation in Lucence’s liquid biopsy tests.

Earlier this year, the Company presented data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting demonstrating high sensitivity of AmpliMARK at a limit of detection (LOD) of 0.1% variant allele frequency, detecting clinically relevant biomarkers in lung, breast and blood cancers. AmpliMARK demonstrated 97.4% concordance for recommended biomarkers in lung cancer when compared orthogonally with another liquid biopsy platform. CLIA-certification of the Company’s Palo Alto laboratory now ensures accelerated delivery of LiquidHALLMARK’s high resolution, target-rich insights to US clinicians and patients.