On November 29, 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 that its partners Alphamab Oncology (stock code: 9966.HK) and 3D Medicines (Beijing) Co., Ltd. announced that envafolimab (KN035), the world’s first single-domain PD-L1 antibody formulated for subcutaneous injection received marketing authorization from the Chinese National Medical Products Administration (NMPA) (Press release, Tracon Pharmaceuticals, NOV 29, 2021, View Source [SID1234596187]).

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Envafolimab was approved for adult patients with microsatellite instability-high (MSI-H) or deficient MisMatch Repair (dMMR) advanced solid tumors, including those patients with advanced colorectal cancer who have experienced disease progression following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan, as well as patients with other advanced solid tumors who have experienced disease progression following prior systemic treatment and have no satisfactory alternative treatment options.

Prior to this approval, all marketed PD-1 and PD-L1 antibody drugs required intravenous infusions. As a subcutaneously administered PD-L1 antibody, envafolimab can be administered within 30 seconds in the physician’s office—thereby increasing convenience, shortening treatment time and sparing patients from the risk of infusion reactions.

In a pivotal phase 2 clinical study in patients with advanced dMMR/MSI-H tumors who received one or more lines of treatment, envafolimab demonstrated an objective response rate (ORR) by blinded independent radiographic review (BIRR) of 44.7%, including 12 (11.7%) cases of complete response. Responses were durable, with duration of response at 12 months in responding patients with advanced colorectal cancer (CRC), advanced gastric cancer, other advanced solid tumors, and all responding patients of 89%, 100%, 100%, and 93%, respectively. Median progression-free survival was 11.1 months and the 12-month overall survival rate was 73.6%. The confirmed ORR by BIRR in MSI-H/dMMR 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 treatment and the 33% confirmed ORR reported for KEYTRUDA in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin and irinotecan treatment in cohort A of the phase 2 KEYNOTE-164 trial. Envafolimab was well tolerated in this study and no cases of immune-related pneumonitis, immune-related colitis, or immune-related nephritis were reported.

"We are pleased the dedication of our partners Alphamab Oncology and 3D Medicines has resulted in the initial approval of the first subcutaneously administered checkpoint inhibitor," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "Importantly, the TRACON sponsored pivotal ENVASARC trial of envafolimab for the treatment of undifferentiated pleomorphic sarcoma (UPS) and myxofibrosarcoma (MFS) in the United States continues to enroll well at 26 sites, and we look forward to the Independent Data Monitoring Committee review of interim efficacy and safety data before the end of the year."

About Envafolimab

Envafolimab (KN035), a single-domain antibody against PD-L1 invented by Alphamab Oncology, is the first subcutaneously injected PD-(L)1 inhibitor approved by the NMPA in November 2021 in adult patients with MSI-H/dMMR advanced solid tumors who failed systemic treatment and have no satisfactory alternative treatment options. In December 2019, Alphamab Oncology, 3D Medicines and TRACON entered into a collaboration whereby TRACON has the right to develop and commercialize envafolimab in soft tissue sarcoma in North America. Envafolimab is currently being studied in the pivotal ENVASARC Phase 2 trial in the U.S. sponsored by TRACON and a Phase 3 pivotal 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.

About ENVASARC (NCT04480502)

The ENVASARC pivotal trial is a multicenter, 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 into 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.

On November 29, 2021 Celularity Inc. (Nasdaq: CELU) ("Celularity"), a clinical-stage biotechnology company developing placental-derived allogeneic cell therapies, reported the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for the use of CYNK-101 in combination with standard chemotherapy, trastuzumab and pembrolizumab in patients with first-line locally advanced unresectable or metastatic HER2/neu positive gastric or gastroesophageal junction (G/GEJ) adenocarcinoma (Press release, Celularity, NOV 29, 2021, View Source [SID1234596186]). CYNK-101 is an investigational genetically engineered natural killer (NK) cell therapy designed to enhance antibody-dependent cellular cytotoxicity (ADCC) with approved and novel antibody therapeutics.

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"Gastric cancer represents the fifth most common cancer worldwide, and in advanced stages of the disease, continues to be associated with less than desirable survival outcomes despite recent advances," said Robert Hariri, M.D., Ph.D., Founder, Chairperson and Chief Executive Officer of Celularity. "By enhancing the innate ADCC activity of our placental-derived NK cells, we have developed a cellular therapy platform that holds promise to complement and synergize with a range of antibody treatment strategies across a variety of tumor types. Our goal is to combine the potential advantages of placental-derived cellular therapies, including enhanced persistence, proliferation and resistance to cell exhaustion, with approved treatment strategies," Hariri said.

Andrew Pecora, M.D., President of Celularity, said, "Among patients with locally advanced unresectable or metastatic HER2/neu positive gastric or gastroesophageal junction adenocarcinoma, advances in patient outcomes have been achieved with the addition of trastuzumab, and more recently, pembrolizumab, to standard chemotherapy, leading to regulatory approval of this combination. We are now excited to begin assessing if our off-the-shelf allogeneic placental-derived NK cells that have been genetically modified to enhance ADCC and resist cleavage of CD16 can improve clinical outcomes when added to the current combination in this patient population."

The Phase 1/2a open-label, non-randomized clinical trial is designed to evaluate the safety and preliminary efficacy of the combination treatment strategy.

About CYNK-101

Celularity’s lead therapeutic candidate based on its placental-derived genetically modified NK cell type is CYNK-101, an allogeneic, off-the-shelf human placental CD34+-derived NK cell product genetically modified to express high-affinity and cleavage-resistant CD16 (FCGRIIIA) variant to drive antibody-dependent cell-mediated cytotoxicity. Currently CYNK-101 is being developed as a treatment in combination with standard chemotherapy, trastuzumab and pembrolizumab for HER2+ overexpressing gastric or gastroesophageal junction adenocarcinoma. The safety and efficacy of CYNK-101 have not been established, and CYNK-101 has not been approved for any use by the U.S. Food and Drug Administration or any other analogous regulatory authority.

On November 29, 2021 OPKO Health, Inc. (NASDAQ: OPK) reported that management will be participating in the Piper Sandler 33rd Annual Virtual Healthcare Conference taking place November 30 through December 2, 2021 (Press release, Opko Health, NOV 29, 2021, View Source [SID1234596185]). A pre-recorded company fireside chat is available for registered attendees via the Piper Sandler conference site and at this link.

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Management will be available for one-on-one virtual meetings with investors on Wednesday, December 1st, which can be requested through Piper Sandler.

On November 29, 2021 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a precision oncology company targeting virus-associated malignancies, reported that the U.S. Food and Drug Administration (FDA) has granted its all-oral combination product candidate, nanatinostat and valganciclovir (Nana-val), orphan drug designation (ODD) for the treatment of Epstein Barr virus-positive diffuse large B-cell lymphoma, not otherwise specified (EBV+ DLBCL, NOS) (Press release, Viracta Therapeutics, NOV 29, 2021, View Source [SID1234596184]). DLBCL is the most common subtype of non-Hodgkin lymphoma (NHL) in the U.S. and worldwide, accounting for approximately 25% of newly diagnosed cases of NHL in the U.S, of which a subset are EBV+. Viracta has previously received ODD from the FDA for the treatment of T-cell lymphoma, post-transplant lymphoproliferative disorder (PTLD) and plasmablastic lymphoma.

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"This latest orphan drug designation underscores the potential benefits of our all-oral kick and kill approach to targeting EBV-positive cancers," said Ivor Royston, M.D., President and Chief Executive Officer of Viracta. "Nana-val has shown promising preliminary efficacy across multiple subtypes of relapsed/refractory EBV-positive lymphoma, including DLBCL. We are dosing patients in the pivotal NAVAL-1 trial, which includes patients with EBV-positive DLBCL, and look forward to its continued momentum with sites now open for enrollment in the U.S., Europe, and Asia. Through NAVAL-1’s progress, we aim to further develop Nana-val as a new and actionable therapy in indications where many patients recur from the standard of care and have particularly poor prognoses."

The FDA grants orphan drug designations to investigational drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits toward qualified clinical trial costs, assistance with clinical study design and drug development, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory approval for the disease or condition for which the drug has the orphan drug designation. In 2020, 31 of the 53 novel drug approvals, or 58%, in the FDA’s Center for Drug Evaluation and Research, were orphan designated products.

About NAVAL-1

NAVAL-1 (Nanatinostat in Combination with Valganciclovir) is a global, multicenter, open-label Phase 2 basket trial. The trial, which will include patients with multiple subtypes of relapsed/refractory EBV-positive (EBV+) lymphoma, is designed to evaluate the anti-tumor activity of Nana-val and enroll approximately 140 patients. The primary endpoint of the trial is objective tumor response rate as assessed by an independent review committee. If successful, Viracta believes this trial could potentially support multiple new drug application filings across various EBV+ lymphoma subtypes. The study employs a Simon two-stage design where a limited number of patients are enrolled into each cohort in Stage 1 and, if a pre-specified activity threshold is reached, additional patients will be enrolled in Stage 2. During Stage 2, Viracta anticipates discussing the preliminary results with the FDA and may amend the protocol to include additional patients as necessary to enable registration.

About Nanatinostat

Nanatinostat (VRx-3996) is an orally available histone deacetylase (HDAC) inhibitor being developed by Viracta. Nanatinostat is selective for specific isoforms of Class I HDACs, which is key to inducing viral genes that are epigenetically silenced in EBV-associated malignancies. Nana-val (nanatinostat and valganciclovir) is being investigated in multiple subtypes of relapsed/refractory EBV+ lymphoma and in advanced EBV+ solid tumors in three ongoing trials, one of which is a registration-enabling global, multicenter, open-label Phase 2 basket trial in relapsed/refractory EBV+ lymphoma (NAVAL-1).

About EBV-Associated Cancers

Approximately 95% of the world’s adult population is infected with Epstein-Barr Virus (EBV). Infections are commonly asymptomatic or associated with mononucleosis. Following infection, the virus remains latent in a small subset of lymphatic cells for the duration of the patients’ life. Cells containing latent virus are increasingly susceptible to malignant transformation. Patients who are immunocompromised are at an increased risk of developing EBV+ lymphomas. In addition, EBV is also associated with a variety of solid tumors, including nasopharyngeal carcinoma and gastric cancer.

On November 29, 2021 Athenex, Inc., (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, reported that its Marketing Authorization Application (MAA) for oral paclitaxel and encequidar (Oral Paclitaxel) for the treatment of advanced breast cancer has been validated by the United Kingdom’s (UK) Medicines and Healthcare products Regulatory Agency (MHRA) for review (Press release, Athenex, NOV 29, 2021, View Source [SID1234596183]). The application qualifies for a 150 day assessment by which a decision on approvability of the product will be provided.

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"We have been working diligently on the MAA submission to bring Oral Paclitaxel to patients with advanced breast cancer," said Dr. Rudolf Kwan, Chief Medical Officer of Athenex. "This is a key milestone for Oral Paclitaxel, the most advanced program in our Orascovery platform. The MAA validation is an important step forward in the regulatory process and underscores our commitment to making Oral Paclitaxel available to patients."

A single pivotal Phase III study of Oral Paclitaxel (KX-ORAX-001) served as the basis of the MAA. The study was a randomized, controlled clinical trial designed to compare the safety and efficacy of Oral Paclitaxel monotherapy versus IV paclitaxel monotherapy in patients with metastatic breast cancer. As previously reported, the study successfully achieved its primary endpoint showing statistically significant improvement in overall response rate (ORR), along with a lower incidence of neuropathy, for Oral Paclitaxel compared to IV paclitaxel.