On December 30, 2019 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics targeting infectious diseases and cancers based on its proprietary arenavirus platform, reported that the first patient has been dosed in a Phase 1/2 clinical trial (NCT04180215) of HB-201, an immunotherapy for the treatment of Human Papillomavirus 16-positive (HPV16+) cancers. This trial is HOOKIPA’s first clinical trial in immuno-oncology (Press release, Hookipa Pharma, DEC 30, 2019, View Source [SID1234553433]).

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HB-201 is a TheraT platform-based vector (replication attenuated) from the arenavirus family expressing a non-oncogenic but highly antigenic E6/E7 fusion protein from HPV16. In preclinical studies, HB-201 was observed to be highly immunogenic, resulting in a robust CD8+ T cell response as compared to the levels induced by other approaches including adoptive cell therapies. In addition to strong immunogenicity, HOOKIPA observed robust anti-tumor activity in mouse models. HOOKIPA believes that HB-201 has the potential to provide therapeutic benefit to patients across the HPV16+ cancer setting.

Joern Aldag, Chief Executive Officer at HOOKIPA, commented: "We are excited to begin first-in-human testing with HB-201, our first clinical trial in immuno-oncology. Translating our promising preclinical data to cancer patients is an important milestone. We believe HOOKIPA’s approach can supercharge the natural defence mechanisms by inducing strong T cell responses to the benefit of patients affected by cancer and infectious diseases."

About the HB-201 Clinical Trial

The HB-201 Phase 1/2 trial is an open-label dose-escalation and dose-expansion trial in 100 patients with treatment-refractory HPV16+ cancers. It is designed to evaluate the safety and tolerability and preliminary efficacy of HB-201 as monotherapy and in combination with an immune checkpoint inhibitor.

For Phase 1 dose escalation, the patient population will be divided into two groups of 20 patients, each: the first group will include patients with progressing HPV16+ tumors who will receive monthly intravenous (IV) administration of HB-201; the second group will include patients with progressing HPV16+ tumors and an accessible tumor site who will receive one intratumoral (IT) administration of HB-201 followed by monthly IV administration of HB-201.

The primary endpoint of the Phase 1 portion of this trial will be to evaluate safety and tolerability to determine the recommended dose for the Phase 2 portion. Secondary endpoints will evaluate anti-tumor activity, as defined by RECIST (Response Evaluation Criteria In Solid Tumors) 1.1, and immunogenicity. The Phase 2 portion of the trial will also investigate the efficacy of HB-201 alone and in combination with a PD-1 inhibitor. HOOKIPA expects to provide interim safety, dose escalation, and efficacy data from HB-201 in late 2020 or early 2021. These data will be supplemented by a series of translational data sets designed to demonstrate the mechanism of action.

About Human Papillomavirus

Human Papillomavirus (HPV) is estimated to cause about 5% of the worldwide burden of cancer including approximately 99% of cervical cancers, 25% to 60% of head and neck cancers, 70% of vaginal cancers and 88% of anal cancers1. The majority of these cancers are caused by the HPV serotype 16. Most infections with HPV are cleared from the body with no lasting consequences. However, in some cases, HPV DNA becomes integrated into chromosomal DNA. When host cells take up this DNA, they express the HPV E6 and E7 proteins. This can potentially lead to cancer since expression of these proteins leads to alterations in cell cycle control, which in turn predisposes these cells to becoming cancerous.

On December 30, 2019 Bruker Corporation (Nasdaq: BRKR) reported it will participate in the 38th annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Bruker, DEC 30, 2019, View Source [SID1234552638]). Frank Laukien, Chairman, President & CEO and Gerald Herman, CFO will present on behalf of the company on Monday, January 13th, 2020 at 11:30 AM Pacific Time.

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A live audio webcast of the presentation will be available on the Investor Relations section of the Company’s website at View Source . A replay of the presentation will be posted in the "Events & Presentations" section of the Bruker Corporation Investor Relations website after the event and will be available for 30 days following the presentation.

On December 30, 2019 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the first patient has been enrolled in the pivotal Phase 2/3 clinical study for JZP-458, a recombinant Erwinia asparaginase molecule that uses a novel Pseudomonas fluorescens expression platform (Press release, Jazz Pharmaceuticals, DEC 30, 2019, View Source [SID1234552637]). The study, conducted in collaboration with the Children’s Oncology Group (COG), is evaluating JZP-458 as a potential treatment for pediatric and adult patients with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) who are hypersensitive to E. coli-derived asparaginases. Hypersensitivity reactions affect up to 30 percent of patients with ALL and LBL who are treated with E. coli-derived asparaginase.1

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"We’re pleased to collaborate with Jazz on this important study," said Dr. Mignon Loh, professor of pediatrics at the University of California San Francisco (UCSF), Deborah and Arthur Ablin Endowed Chair in Pediatric Molecular Oncology and COG’s Acute Lymphoblastic Leukemia Disease Committee Chair.

"This clinical trial represents a tremendously important effort as it is investigating a novel asparaginase, JZP-458, which can be critically important for the treatment of some children with ALL, the most common type of childhood malignancy," stated Dr. Luke Maese, assistant professor at the University of Utah, Primary Children’s Hospital and Huntsman Cancer Institute.

The single-arm, open-label, multicenter, dose confirmation and confirmatory study of JZP-458 will evaluate pediatric and adult patients with ALL or LBL who have silent inactivation or an allergic reaction to E. coli-derived asparaginases and have not previously received asparaginase Erwinia chrysanthemi. This study is designed to assess the safety, tolerability and efficacy of JZP-458 and is expected to enroll patients in approximately 60 COG institutions in the U.S. and Canada. The primary objective of the study is to determine the efficacy of JZP-458 measured by asparaginase activity.

"When undergoing treatment for ALL with asparaginase, it is critically important for patients to receive all of the necessary doses to maintain therapeutic levels throughout their regimen, something not always possible for patients who have an allergy to E. coli-derived asparaginase," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development of Jazz Pharmaceuticals. "Our ongoing collaboration with COG for this JZP-458 study, and the receipt of Fast Track designation from the U.S. Food and Drug Administration in October, are significant because they could potentially allow us to more quickly address this need with a new asparaginase option. Jazz is committed to addressing unmet needs for patients with hematologic cancers and the continued expansion of our asparaginase franchise is an important component of our development programs."

Additional information about the trial, including eligibility criteria and a list of clinical trial sites, can be found at: View Source (ClinicalTrials.gov Identifier: NCT04145531).

About JZP-458
JZP-458 is a recombinant Erwinia asparaginase that uses a novel Pseudomonas fluorescens expression platform. It is being developed for use as a component of a multi-agent chemotherapeutic regimen in the treatment of pediatric and adult patients with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) who are hypersensitive to E. coli-derived asparaginase products. JZP-458 was granted Fast Track designation by the U.S. Food and Drug Administration in October 2019 for the treatment of this patient population.

About Acute Lymphoblastic Leukemia
Acute lymphoblastic leukemia (ALL) is a cancer of the blood and bone marrow that can progress quickly if not treated.2 Leukemia is the most common cancer in children, and about three out of four of these cases are ALL.3 Although it is one of the most common cancers in children, ALL is among the most curable of the pediatric malignancies due to recent advancements in treatment.4,5 Adults can also develop ALL, and about four of every 10 cases of ALL diagnosed are in adults.6 The American Cancer Society estimates that almost 6,000 new cases of ALL will be diagnosed in the United States in 2019.6 Asparaginase is a core component of multi-agent chemotherapeutic regimens in ALL.7 However, asparaginase treatments derived from E. coli are associated with the potential for development of hypersensitivity reactions.8

About the Children’s Oncology Group
The Children’s Oncology Group (www.childrensoncologygroup.org) is the world’s largest organization devoted exclusively to childhood and adolescent cancer research. The Children’s Oncology Group (COG) unites almost 10,000 experts in childhood cancer at more than 200 leading children’s hospitals, universities, and cancer centers across United States, Canada, Australia, New Zealand, and parts of world in the fight against childhood cancer. Today, more than 90% of the 14,000 children and adolescents diagnosed with cancer each year in the United States are cared for at COG member institutions. Research performed by the COG institutions over the past fifty years has transformed childhood cancer from a virtually incurable disease to one with a combined 5-year survival rate of 80%. COG’s mission is to improve the cure rate and outcome for all children with cancer.

On December 30, 2019 Gracell Biotechnologies Co. Ltd., a clinical-stage immune cell therapy company, reported it will present at leading healthcare conferences in San Francisco, California at the beginning of January 2020 (Press release, Gracell Biotechnologies, DEC 30, 2019, View Source [SID1234552636]). Gracell will present at the 38th Annual J.P. Morgan Healthcare Conference, held between January 13-16, 2020 at The Westin St. Francis Hotel on Union Square.

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Focused exclusively on companies defining the healthcare industry, the J.P. Morgan Healthcare Conference is the largest of its kind and will welcome over 400 public and private companies to deliver presentations to over 8,000 attendees, including investors and industry leaders.

"We are delighted to be invited to present at some of the most recognized and regarded events in the healthcare industry," said Dr. William Cao, CEO of Gracell. "The invaluable data from Gracell’s clinical trials demonstrates our strong capabilities to bring multiple novel technologies to the forefront of immune cell therapy field. We are eager to share these findings with the global healthcare community."

Gracell will also present at the following conference in January.

China Showcase
Presentation time: 4:00pm, Sunday, January 12
Location: Parc 55 San Francisco – A Hilton Hotel
Track: Cyrill Magnin III (4th floor)

Biotech Showcase
Presentation time: 10:00am, Monday, January 13
Location: Hilton San Francisco Union Square Hotel
Track: Franciscan B (Ballroom Level)

On December 30, 2019 OncoImmune, Inc. reported that its Investigational New Drug ("IND") application for ONC-392, its novel, next generation anti-CTLA-4 antibody, has been approved by the U.S. Food and Drug Administration ("FDA") (Press release, ONCOIMMUNE, DEC 30, 2019, View Source [SID1234552635]). The IND approval enables OncoImmune to begin a Phase 1A/1B clinical trial of ONC-392 that is designed to assess the safety, pharmacokinetics, and efficacy of ONC-392 as a single agent in advanced solid tumors and in combination with anti-PD(L)1 standard of care in Non- Small Cell Lung Cancer. This open label trial is expected to begin in early 2020.

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ONC-392 was developed based on the research of OncoImmune’s Founders, Drs. Yang Liu and Pan Zheng, who proposed a new theory to improve both the efficacy and safety of immunotherapy drugs. The theory calls for preservation of the CTLA-4 immune checkpoint for safer and more effective immunotherapy. (https://www.sciencedirect.com/science/article/pii/S0165614719302639). This groundbreaking research was published in three papers in Cell Research in 2018 and 2019. The two 2018 papers were recognized with the Sanofi-Cell Research Outstanding Paper Award of 2018 (View Source).

"ONC-392 is OncoImmune’s second drug product candidate and the approval of this IND is an important milestone for OncoImmune," said Yang Liu, President and CEO of OncoImmune. "Unlike other anti-CTLA-4 antibodies that cause lysosomal degradation of CTLA-4, ONC-392 preserves CTLA-4 recycling and thus maintains CTLA-4 function outside of the tumor microenvironment while allowing more effective CTLA-4-targeted depletion of regulatory T cells within the tumor. The truly novel and differentiated mechanism of action of this drug has the potential to improve therapeutic outcomes while significantly reducing toxicity."

"We are very excited to test the potential of this novel antibody in cancer patients," said Pan Zheng, Chief Medical Officer of OncoImmune, Inc.

The CMC development and GMP manufacturing of the drug substance and drug product were performed by WuXi Biologics, a leading global open-access biologics technology platform for the ONC-392 program. "Throughout the development program from DNA to IND, we were very impressed by WuXi Biologics’ expertise and professionalism, and we could not have picked a better partner for this project," said Martin Devenport, OncoImmune’s Chief Operating Officer.