On July 6, 2022 EXUMA Biotech, Corp., a clinical-stage biotechnology company discovering and developing cell and gene therapies for liquid and solid tumors, reported that Dr. Sid Kerkar, the company’s Vice President of Oncology, Research and Development, will participate in the In Vivo Engineering of Therapeutic Cells Summit organized by Hanson Wade on July 12-14, 2022 in Boston (Press release, EXUMA Biotechnology, JUL 6, 2022, View Source [SID1234616505]).

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!

"We are encouraged by the preclinical findings with EXUMA’s novel in vivo GCAR technology, which has the potential to overcome many of the barriers surrounding current ex vivo CAR therapies.

Our mission is centered on creating highly effective, safe, and durable treatments while reducing manufacturing complexity and costs, and increasing the speed and quality of delivery for the next generation of cell and gene therapies for cancer patients," said Sid Kerkar, M.D., VP Oncology, R&D.

EXUMA Biotech-Tuesday, July 12th, 2022

Participation: Pre-Conference Workshop

Title: Moving from Ex Vivo to In Vivo CAR T Engineering to Improve Clinical Performance & Accessibility

Details: In vivo CAR T has the potential to overcome challenges including production time, cost, and manufacturing delays, which could make this therapy more broadly available to patients.

This workshop will offer a deep-dive view into everything you need to know about CAR T and how to make the switch to the in vivo approach.

EXUMA Biotech Speaker: Dr. Sid Kerkar, EXUMA Biotech VP Oncology, R&D

Time: 10:00 AM ET

EXUMA Biotech-Wednesday, July 13th, 2022

Participation: Fireside Chat

Title: Industry Leader’s Fireside Chat: In Vivo Engineering of Therapeutic Cells as the Future of Cell & Gene Therapy

Details: This panel will explore what has inspired the move from ex vivo to in vivo therapies and will outline the advantages that in vivo cell and gene therapies offer.

Speakers will discuss next steps to streamline pre-clinical development to fast-track in vivo therapies to the clinic.

EXUMA Biotech Speaker: Dr. Sid Kerkar, EXUMA Biotech VP Oncology, R&D

Time: 9:30 AM ET

EXUMA Biotech- Thursday, July 14th, 2022

Participation: Roundtable Session

Title: Optimizing the In Vivo Approach: Considerations for Safety & Tolerability

Details: Dr. Kerkar will offer details on improving the safety and tolerability of in vivo therapies.

He’ll explore off target effects and will address issues of immunogenicity, integration, insertional mutagenesis, oncogenicity and pre-clinical models for toxicology.

EXUMA Biotech Speaker: Dr. Sid Kerkar, EXUMA Biotech VP Oncology, R&D

Time: 1:30 PM ET

On July 6, 2022 Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the "Company"), a a clinical-stage macrophage reprogramming immunotherapy company targeting diseased macrophages in patients with sepsis and solid tumors, reported that the first patient has been dosed in a Phase I/II clinical trial evaluating Allocetra combined with chemotherapy in patients with peritoneal metastases arising from solid cancer (Press release, Enlivex Therapeutics, JUL 6, 2022, View Source [SID1234616504]).

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!

Peritoneal cancer, whether originating from a primary tumor within the peritoneum or from a metastatic tumor elsewhere in the body, is a terminal disease with a poor prognosis. Patients with peritoneal metastases are in urgent need of novel treatment options, as standard-of- care (SOC) chemotherapy currently provides only modest survival benefits. The median survival of patients with peritoneal metastases differs based on the location of the primary tumor but is frequently poor, with survival rates of 2.9 months, 6.5 months, and 6.9 months reported for cancers of pancreatic, gastric, and colorectal origins, respectively.

Prof. Aviram Nissan, M.D., Head of the Department of General and Oncological Surgery at Sheba Medical Center and Principal Investigator of the trial commented: "The patients are operated using the Pressurized Intra-Peritoneal Aerosol Chemotherapy (PIPAC) technology, which allows for efficient insertion of therapeutics directly to the peritoneum. We hope that the combination of chemotherapy and Allocetra, a next-generation cell therapy in development for oncological indications, will generate a breakthrough in the treatment of peritoneal metastases, which are not treatable in most patients with anti-cancer drugs available today. We are eager to test this new combination with the hope of changing the lives of patients with peritoneal metastases."

Mohammad Adileh, M.D., Attending Surgical Oncologist, Department of Surgery and Surgical Oncology – Surgery C, and a Co-Investigator in the trial stated: "Together with my team, I performed a ground breaking procedure in a patient with abdominal metastasis, combining a novel drug delivery system, PIPAC, with Allocetra, the development-stage macrophage-reprogramming cell therapy produced by Enlivex. I look forward to understanding the scope of the effect of Allocetra and its impact on the immune system in its efforts to recognize and kill malignant tumors in the abdomen. We are excited to test this combination as a new hope for patients with peritoneal cancers."

"The initiation of our first oncology trial is a crucial moment in Enlivex’s evolution," said Oren Hershkovitz, Ph.D., Chief Executive Officer of Enlivex. "We believe that it de-risks our pipeline with another avenue for value creation and positions us to substantially expand our addressable patient population. We look forward to the trial’s advancement and to expanding our oncology clinical program and would like to thank all those who helped achieve this important milestone."

Prof. Dror Mevorach, M.D., Chief Scientific Officer of Enlivex commented: "We believe that Allocetra has strong anti-cancer potential in a novel mechanism. Preclinical data suggests that Allocetra rebalances macrophage populations within the tumor microenvironment into a homeostatic state that favors anti-tumoral macrophages. This, in-turn, is expected to weaken the tumor defense mechanisms that limit the efficacy of currently available therapies. With this novel mechanism of action, Allocetra can potentially enhance the anti-cancer activity of a broad range of drug classes across a spectrum of highly prevalent solid cancers."

"Pro-tumor" tumor associated macrophages typically form a physical layer on top of solid cancers and induce an immunosuppressive tumor microenvironment. This promotes cancer growth and metastasis and limits the efficacy of anti-cancer agents, which ultimately contributes to poor clinical outcomes. Preclinical studies, including those that were recently featured at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the International Society for Cell and Gene Therapy Annual Meeting, suggest that Allocetra has the potential to synergistically combine with various anti-cancer agents to provide patients who do not respond well to existing FDA-approved therapies with an effective treatment option.

ABOUT THE PHASE I/II TRIAL

The Phase I/II trial is a company-sponsored, open-label, dose escalation and expansion trial that is expected to enroll a total of approximately 12 patients across four cohorts. It is designed to evaluate the safety and potential preliminary efficacy of Allocetra combined with SOC chemotherapy in patients with peritoneal metastases arising from solid cancer. The study will begin with two cohorts of intra-patient and intra-cohort dose escalation to determine the maximum feasible dose (MFD) of Allocetra in this population, followed by two additional cohorts comparing administration of Allocetra at the selected dose either before or after administration of SOC via a pressurized intraperitoneal aerosol chemotherapy procedure (PIPAC; a technique applied when patients are not eligible to receive the standard treatment due to a considerable tumor load, large quantities of persistent ascites, or other circumstances).

Intraperitoneally delivered Allocetra and SOC chemotherapy administered via PIPAC will be given to patients every six weeks. Systemic chemotherapy will also be administered per the treating oncologist’s plan. The primary endpoint is the number and severity of Allocetra-related adverse events and serious adverse events during the 16-week period, starting from the first administration of study treatment. Secondary endpoints include efficacy assessments, such as best overall response rate (ORR), progression-free survival, and overall survival. Changes from baseline in macrophage and immune cell characteristics in peritoneal fluid and tissues will also be assessed as an exploratory endpoint.

ABOUT ALLOCETRA

Allocetra is being developed as a universal, off-the-shelf cell therapy designed to reprogram macrophages into their homeostatic state. Diseases such as solid cancers, sepsis, and many others reprogram macrophages out of their homeostatic state. These non-homeostatic macrophages contribute significantly to the severity of the respective diseases. By restoring macrophage homeostasis, Allocetra has the potential to provide a novel immunotherapeutic mechanism of action for life-threatening clinical indications that are defined as "unmet medical needs", as a stand-alone therapy or in combination with leading therapeutic agents.

On July 6, 2022 Elicio Therapeutics, a clinical stage biotechnology company developing a pipeline of novel immunotherapies for the treatment of cancer and other diseases, reported that it presented preclinical data demonstrating its Amphiphile (AMP) platform adjuvant AMP-CpG combined with cell surface-associated viral protein EBV gp350 and EBVpoly protein, elicits a robust and durable immune response to Epstein-Barr virus (EBV) (Press release, Elicio Therapeutics, JUL 6, 2022, View Source [SID1234616503]). EBV gp350 is expressed on the outside of the virus and on the cells producing the virus, making it a major target for neutralizing antibodies and CD4+ T cells, while EBVpoly protein contains multiple epitopes for CD8+ T cells. The data was presented at the 2022 Keystone Symposia on Viral Immunity Basic Mechanisms and Therapeutic Applications virtually and in-person at the Keystone Resort in Keystone, CO from June 29-July 2, 2022. The electronic poster is accessible here.

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!

EBV is a herpesvirus responsible for the well-known mononucleosis infections ("mono") and has also been implicated in multiple lymphoid and epithelial cancers. EBV targets both B cells and epithelial cells and utilizes their molecular machinery to replicate the viral genome. The virus causes B cells to differentiate into memory B cells, which then can move into the circulatory system, or become latent until a trigger causes reactivation. Approximately 95%[1] of the adult population worldwide is infected with EBV which persists for the life of the individual, however, there is currently no approved vaccine.

"Vaccines against EBV have historically been difficult to develop because of the associated viral latency, persistence and immune modulation properties which enable it to evade effective antibody targeting," said Lisa McNeil, Ph.D., Head of Translational Medicine at Elicio Therapeutics. "These data reinforce what we’ve seen in previous studies with our AMP platform, demonstrating the potent and durable immune responses elicited by delivering a therapeutic payload direct to the lymph nodes, the control center of the immune system. By targeting the most abundant glycoprotein expressed on the virus with the AMP-CpG adjuvant, in a mouse model mimicking human-specific immune responses to viral infection, we were able to induce neutralizing antibodies and strong T cell responses. This holds great promise for prevention of EBV associated diseases and controlling the spread of latently infected B cells."

The EBV vaccine is based on the research of Dr. Rajiv Khanna, Professor, Senior Scientist and Coordinator of QIMR Berghofer’s Centre for Immunotherapy and Vaccine Development. Professor Khanna said, "I am encouraged by the data demonstrating the potential of the AMP-CpG adjuvant combined with the EBV proteins to not only activate but supercharge the immune system as shown by the high frequencies of polyfunctional gp350-specific CD4+ T cells and EBVpoly-specific CD8+ T cells induced in this model."

Poster Presentation Details

Title: A Lymph Node Targeted Engineered Subunit Antigen and Molecular Adjuvant Vaccine Promotes Potent Cellular and Humoral Immunity to Epstein Barr Virus in HLA-expressing Mice

Highlights from the Poster

EBVpoly is a polyepitope protein developed at QIMR Berghofer Medical Research Institute that includes 20 CD8+ T cell epitopes from EBV latent and lytic antigens with broad coverage against multiple HLA types.
AMP-CpG delivers adjuvant directly to lymph nodes, boosting the immune response to protein antigens.
Vaccination with AMP-CpG combined with EBV gp350 and EBVpoly proteins rapidly induced potent gp350-specific IgG and EBV neutralizing antibody responses in HLA transgenic mice.
AMP-CpG immunization induced high frequencies of polyfunctional gp350-specific CD4+ T cells and EBVpoly-specific CD8+ T cells.
The potent humoral and cellular immunity induced by AMP-CpG was durable, with responses maintained for >7 months.
The broad coverage against multiple viral determinants and the AMP-CpG adjuvant are likely to provide better protection against primary EBV infection while the strong T cell responses will be critical in controlling the spread of latently infected B cells and the development of EBV-associated diseases, such as malignancies and multiple sclerosis.
About the Amphiphile Platform

Our proprietary Amphiphile, or AMP, platform delivers investigational immunotherapeutics directly to the "brain center" of the immune system – the lymph nodes. We believe this site-specific delivery of disease-specific antigens, adjuvants and other immunomodulators may efficiently educate, activate and amplify critical immune cells, potentially resulting in induction and persistence of potent adaptive immunity required to treat many diseases. In preclinical models, we have observed lymph node-specific engagement driving therapeutic immune responses of increased magnitude, function and durability. We believe our AMP lymph node-targeted approach will produce superior clinical benefits compared to immunotherapies that do not engage the lymph nodes.

Our AMP platform, originally developed at the Massachusetts Institute of Technology, or MIT, has broad potential across cancers, infectious diseases and other disease indications to advance a number of development initiatives through internal activities, in-licensing arrangements or development collaborations and partnerships.

The Amphiphile platform is thought to deliver immunotherapeutics directly to the lymph nodes by latching on to the protein albumin, found in the bloodstream, as it travels to lymphatic tissue. In preclinical models, we have observed lymph node-specific engagement driving therapeutic immune responses of increased magnitude, function and durability.

On July 6, 2022 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a leader in the field of conditionally activated oncology therapeutics, reported that the Phase 2 study of praluzatamab ravtansine in patients with hormone receptor-positive (HR+)/human epidermal growth factor receptor 2 (HER2)-non-amplified breast cancer (Arm A) met its primary efficacy endpoint of confirmed objective response rate (ORR) of greater than 10 percent by central radiology review (Press release, CytomX Therapeutics, JUL 6, 2022, View Source [SID1234616502]). Praluzatamab ravtansine is a DM4-conjugated, conditionally activated antibody-drug conjugate (ADC) targeting CD166 and is wholly owned by CytomX.

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!

As of the data cutoff on May 13, 2022, 47 patients unselected for CD166 expression with advanced HR+/HER2-non-amplified breast cancer were evaluable for the primary efficacy endpoint. The ORR by central radiology review was 15 percent. Clinical benefit rate at 24 weeks by investigator (CBR24), as defined in the protocol as any response (confirmed or unconfirmed) or stable disease for 24 weeks, was 40 percent; median progression-free survival was 2.6 months. All patients in Arm A were treated at the initial Phase 2 starting dose of 7 mg/kg administered every three weeks. Arm B did not pass protocol-defined futility boundary (ORR was less than 10%) in patients with advanced triple-negative breast cancer (TNBC) and enrollment into Arms B and C will be discontinued.

As of this data cut, the safety profile of praluzatamab ravtansine in Arm A was generally consistent with toxicities observed in Phase 1 and with the DM4 payload; namely, high-grade toxicities or toxicities resulting in dose modifications were predominantly ocular or neuropathic in nature. Thirty percent of patients discontinued treatment for an adverse event (AE). Grade 3+ ocular and neuropathic toxicities were 15 and 10 percent, respectively. Arm B evaluated both 7 mg/kg and 6 mg/kg in patients with TNBC. The toxicity profile of 7 mg/kg starting dose was consistent with Arm A. In the 6 mg/kg cohort, no patients discontinued treatment for an AE and Grade 3+ ocular or neuropathic related events were lower at 3, and 0 percent, respectively. Biomarker analysis is ongoing. CytomX intends to submit data from this study for presentation at a medical conference in the second half of 2022.

"These results from our Phase 2 evaluation of praluzatamab ravtansine support single-agent activity of this novel drug candidate in hormone receptor-positive breast cancer where significant unmet need remains," said Sean McCarthy, D.Phil., chief executive officer and chairman at CytomX Therapeutics. "However, we do not believe the median progression-free survival at 7 mg/kg supports further evaluation at this dose. While we are encouraged by the emerging safety profile of 6 mg/kg, we do not plan to further advance this program alone given current financial market conditions and will be seeking a partnership."

Kathy D. Miller, MD, Ballvé Lantero Professor of Oncology, Indiana University Simon Comprehensive Cancer Center, Indianapolis, Indiana, and lead investigator of the Phase 2 study stated, "In this Phase 2 study, praluzatamab ravtansine showed single-agent activity in an unselected population of patients with advanced HR+/HER2-non-amplified breast cancer; additional clinical studies at 6 mg/kg are warranted."

Conference Call & Webcast
CytomX management will host a conference call and a simultaneous webcast today at 5:00 pm ET (2:00 pm PT) to discuss these results. Participants may register for the conference call here and are advised to do so at least 10 minutes prior to joining the call. A live webcast of the call can be accessed via the Events and Presentations page of CytomX’s website at View Source

About Praluzatamab Ravtansine
Praluzatamab ravtansine is a conditionally activated antibody-drug conjugate (ADC) comprised of a CD166-directed humanized monoclonal antibody conjugated to the maytansinoid DM4, a tubulin inhibitor. Praluzatamab ravtansine utilizes CytomX Probody platform technology, which incorporates a masking peptide to cover and block the cellular binding region of the antibody. Tethered to the antibody via a protease-cleavable linker, the masking peptide is designed to be removed in a protease-rich tumor microenvironment, enabling the ADC to be unmasked and engage its target to deliver the toxic DM4 payload inside tumor cells. The goal is to have praluzatamab ravtansine remain inert while in circulation to limit binding in healthy tissues until it is activated by tumor-associated proteases. Praluzatamab ravtansine was evaluated in a three-arm Phase 2 study (NCT04596150) in patients with hormone receptor-positive/HER2-non-amplified breast cancer and patients with triple-negative breast cancer.

About the Phase 2 Study of Praluzatamab Ravtansine in Breast Cancer (NCT04596150)
Arm A of the study evaluated praluzatamab ravtansine as monotherapy (7 mg/kg, Q3W) in patients with inoperable, locally advanced or metastatic hormone receptor-positive/human epidermal growth factor receptor 2 (HER2)-non-amplified breast cancer. Patients received 0 to 2 prior cytotoxic chemotherapies in the inoperable, locally advanced, or metastatic setting, regardless of the level of CD166 expression.

Arm B assessed praluzatamab ravtansine as a single agent (6 or 7 mg/kg, Q3W) in patients with inoperable, locally advanced or metastatic triple-negative breast cancer (TNBC). Patients received 1 to 3 prior lines of chemotherapy for inoperable, locally advanced, or metastatic disease and had CD166 expression.

Arm C studied praluzatamab ravtansine (6 mg/kg, Q3W) in combination with pacmilimab (1200 mg, Q3W), CytomX’s proprietary conditionally activated anti-PD-L1 antibody, in patients with TNBC. Eligibility was the same as Arm B with the additional requirement that patients’ tumors were programmed death-ligand 1 (PD-L1)-positive by an FDA-approved test.

On July 6, 2022 Shanghai Junshi Biosciences Co., Ltd. ("Junshi Biosciences", HKEX: 1877; SSE: 688180) and Coherus BioSciences, Inc. ("Coherus") reported that the United States Food and Drug Administration ("FDA") has accepted for review the Biologics License Application ("BLA") resubmission for toripalimab in combination with gemcitabine and cisplatin as first-line treatment for patients with advanced recurrent or metastatic nasopharyngeal carcinoma ("NPC") and for toripalimab monotherapy for the second-line or later treatment of recurrent or metastatic NPC after platinum-containing chemotherapy (Press release, Shanghai Junshi Bioscience, JUL 6, 2022, View Source [SID1234616501]).

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 has set a Prescription Drug User Fee Act ("PDUFA") action date for December 23, 2022. The Agency earlier communicated that the review timeline for the BLA resubmission would be six months, as onsite inspections in China would be required. Travel restrictions related to the COVID-19 pandemic previously hindered the FDA’s ability to complete required inspections. Coherus plans to launch toripalimab in the United States in the first quarter of 2023, if approved.

"Although the COVID-19 pandemic has created tremendous challenges for everyone, our dedication to bring better treatment options to patients around the world remains steadfast," said Dr. Patricia Keegan, Chief Medical Officer of Junshi Biosciences. "Through our concerted efforts with our partner, Coherus, we have made continual progress towards obtaining toripalimab’s first marketing authorization outside of China. Over the next several months, we will work closely with the FDA to facilitate the review of this novel drug."

"Toripalimab would address a critical unmet medical need for patients with nasopharyngeal carcinoma, an aggressive cancer for which there are currently no FDA-approved immunotherapy treatments. We collaborated closely with our partner, Junshi Biosciences, to complete the quality process changes requested by the FDA and facilitate the rapid resubmission of the toripalimab BLA," said Dr. Theresa LaVallee, Chief Development Officer of Coherus.

"For Coherus, the toripalimab resubmission is one of several key development and commercialization milestones we are sharply focusing on over the next twelve months, and we are pleased with the Company’s execution and progress on all of them," said Denny Lanfear, CEO of Coherus. "We now look forward to the August 2, 2022 target action date for our BLA for CIMERLI, our Lucentis biosimilar, followed by product launch which we are confident will be very successful. The toripalimab December 2022 PDUFA date follows directly, and the projected toripalimab launch in Q1 2023 will formally mark our entry into immuno-oncology, where Coherus will be one of just a handful of companies with a proprietary PD-1 as a foundation stone to build its oncology franchise upon. Lastly, twelve months from now, in July 2023, we expect to begin marketing our Humira biosimilar, YUSIMRY, which was approved by the FDA in December 2021. Preparations for that commercial launch are going very well. Biosimilar market execution is a demonstrated Coherus competency, and we believe that our commercialization strategy provides a robust framework against which we can successfully execute to meet our market expectations and share projections."

Following approval of toripalimab for NPC, Coherus’ strategy in the US includes evaluating toripalimab’s ability to deliver substantial clinical benefit in significant indications, in combination with other cancer drugs and immunotherapies, through co-development agreements.

About Toripalimab in NPC
NPC is a type of aggressive cancer that starts in the nasopharynx, the upper part of the throat behind the nose and near the base of skull. Due to the location of the primary tumor, surgery is rarely an option, and patients with localized disease are treated primarily with radiation and chemotherapy. In the United States, there are presently no immunotherapies approved for the treatment of NPC.

The toripalimab BLA is supported by the results from JUPITER-02, a randomized, double blind, placebo-controlled, international multi-center Phase 3 clinical trial, as well as POLARIS-02, a multi-center, open-label, pivotal Phase 2 clinical study. The JUPITER-02 results were first presented in June 2021 in a plenary session of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") annual meeting (#LBA2) and subsequently published in detail as the cover article of the September 2021 issue of Nature Medicine. The POLARIS-02 results were published online in January 2021 in the Journal of Clinical Oncology.

The FDA has granted Breakthrough Therapy designation ("BTD") for toripalimab in combination with chemotherapy (gemcitabine and cisplatin) for the 1st line treatment of recurrent, locally advanced or primary metastatic non-keratinizing NPC and for toripalimab monotherapy for patients with recurrent or metastatic non-keratinizing NPC with disease progression on or after platinum-containing chemotherapy. Additionally, the FDA has granted Orphan Drug designation for toripalimab for NPC.

In China, the National Medical Products Administration ("NMPA") in 2021 approved toripalimab for two NPC indications.

About Toripalimab
Toripalimab is an anti-PD-1 monoclonal antibody developed for its ability to block PD-1 interactions with its ligands, PD-L1 and PD-L2, and for enhanced receptor internalization (endocytosis function). Blocking PD-1 interactions with PD-L1 and PD-L2 promotes the immune system’s ability to attack and kill tumor cells. In China, toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing (approved in China as TUOYI). Currently, there are five approved indications for toripalimab in China.