Henlius’ Serplulimab Granted Orphan-Drug Designation in the United States for Small Cell Lung Cancer

On April 7, 2022 Shanghai Henlius Biotech, Inc. (2696.HK) reported that the United States Food and Drug Administration (FDA) has granted Orphan-Drug Designation (ODD) for HANSIZHUANG (serplulimab) for the treatment of small cell lung cancer (SCLC). This is the first such designation granted to HANSIZHUANG (Press release, Shanghai Henlius Biotech, APR 7, 2022, View Source [SID1234611618]). The ODD granted by the FDA is beneficial for the continuous development of HANSIZHUANG and the enjoyment of certain policy support in terms of registration and commercialization in the United States. The company plans to file its NDA in China and MAA in the EU in 2022, making HANSIZHUANG potentially to be the world’s first PD-1 inhibitor for the first-line treatment of SCLC.

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Orphan drugs refer to pharmaceutical products developed for the prevention, diagnosis, and treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. at the time of designation and to offer certain policy support, including but not limited to: 1) tax credits for clinical trial costs; 2) waiver of application fees for new drugs; and 3) seven years of market exclusivity without being affected by patent.

According to GLOBOCAN 2020, lung cancer (LC) is the second most common cancer around the world. There were 2.2 million new LC cases and 1.8 million new deaths in 2020 worldwide, and LC is the leading cause of cancer deaths[1]. SCLC accounts for 15%–20% of all cases and is the most aggressive type of LC. It is classified into two stages: limited stage (LS-SCLC) and extended stage (ES-SCLC), with both exhibiting high malignancy, strong invasiveness, early metastasis, fast disease progression, and a poor prognosis. The advent of immune checkpoint inhibitors has been proved to bring hope to patients with ES-SCLC but fraught with challenges. In recent years, a number of PD-1 mAbs have failed in the area. On the other hand, the standard treatment regimens for LS-SCLC are surgery, chemotherapy and concurrent radiotherapy. Traditional chemotherapeutic drugs did not exhibit significant progress in patients with LS-SCLC, and most patients tend to develop drug resistance or rapid relapse[2-4].

Based on the unmet clinical needs, Henlius promotes a series of immunotherapy studies in the field of SCLC, with the goal of providing more effective treatment approaches for patients. Henlius has undertaken multiple trials to investigate therapy options for both LS-SCLC and ES-SCLC, including the global multi-centre phase 3 study of HANSIZHUANG for the first-line treatment of ES-SCLC (ASTRUM-005) and the investigational new drug application (IND) of international multi-centre phase 3 studies of HANSIZHUANG (serplulimab) approval from the National Medical Products Administration (NMPA). In detail, ASTRUM-005 is a randomised, double-blind, international, multi-centre, phase 3 clinical study aimed to compare the efficacy and safety of HANSIZHUANG with placebo when combined with chemotherapy (carboplatin-etoposide) in previously untreated patients with SCLC. This study has set up about 128 sites in China, Turkey, Poland, Georgia, etc. and 585 subjects were enrolled, among whom 31.5% were Caucasian. In Dec 2021, ASTRUM-005 has met the primary study endpoint of the overall survival (OS), reducing the risk of death by 38% of the overall population (41% in the Asian subgroup). Based on the excellent readouts, the company intends to file NDA for the indication shortly.

In the future, Henlius will continue to emphasize unmet clinical needs and actively promote the combination immunotherapy of serplulimab and international regulatory registration to benefit more patients around the world.

About HANSIZHUANG

HANSIZHUANG (recombinant humanized anti-PD-1 monoclonal antibody injection, generic name: serplulimab injection) is the first innovative monoclonal antibody developed by Henlius. It is approved by the NMPA for the treatment of MSI-H solid tumors in March 2022. Henlius actively promotes HANSIZHUANG in conjunction with in-house products of the company and innovative therapies. It has successively obtained clinical trial licenses in China, the United States, the European Union and other countries and regions to initiate 9 clinical trials on immuno-oncology combination therapies of HANSIZHUANG worldwide in a wide variety of indications, such as lung cancer, esophageal carcinoma, head and neck squamous cell carcinoma and gastric cancer, etc., and covering the full range of first-line treatments of lung cancers. Up to date, the company has enrolled more than 2,800 subjects in China, Turkey, Poland, Georgia and other countries and regions, and the proportion of Caucasians is over 30% in two MRCTs, making HANSIZHUANG an anti-PD-1 mAb with one of the largest global clinical data pools. The NDA of the treatment for squamous non-small cell lung cancer (sqNSCLC) has been accepted by the NMPA and is expected to be approved in 2022. Furthermore, the global multi-center phase 3 clinical study of HANSIZHUANG in combination with chemotherapy in previously untreated extensive small-cell lung cancer (ES-SCLC) met the primary endpoint of overall survival (OS) with remarkable readouts and its NDA in China and MAA in the EU are expected to be filed in 2022, making this product potentially the world’s first anti-PD-1 mAb for the first-line treatment of SCLC.

CARsgen Appoints Dr. Raffaele Baffa as Chief Medical Officer

On April 7, 2022 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T cell therapies for the treatment of hematologic malignancies and solid tumors, reported that Dr. Raffaele Baffa ("Dr. Baffa") has been appointed as the Chief Medical Officer ("CMO") of the Company, responsible for overseeing the global clinical development strategies and operations for the Company’s innovative pipeline product candidates (Press release, Carsgen Therapeutics, APR 7, 2022, View Source [SID1234611617]). Dr. Baffa will report to Dr. Zonghai Li, Founder, Chairman of the Board, CEO, CSO of CARsgen Therapeutics Holdings Limited.

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Dr. Baffa has rich experiences in pharmaceutical industry and research institutes, taking various leadership positions in multi-national corporations and biotech companies. Prior to joining CARsgen, Dr. Baffa served as Chief Medical Officer and Executive Vice President of Research & Development at Ziopharm Oncology (NASDAQ: ZIOP), which was rebranded to Alaunos in January 2022. (NASDAQ: TCRT). Prior to Ziopharm Oncology, Dr. Baffa was Head of Research & Development and Chief Medical Officer in Medisix Therapeutics, a company focused on developing novel immune cell therapies. Dr. Baffa was the Vice President, Therapeutic Area Head of Oncology, Global Clinical Development of Shire Pharmaceuticals, and following the acquisition of the oncology division by Servier Pharmaceuticals, Dr. Baffa served as the Chief Medical Officier of Servier Pharmaceuticals. Dr. Baffa has also held leadership positions at well-known pharmaceutical companies, such as Pfizer and Sanofi.

Dr. Baffa earned an M.D. from University of Padova, School of Medicine and a Ph.D. in biology and molecular pathology from University of Parma in Italy.

Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics Holdings Limited, said, "We warmly welcome Dr. Baffa to join CARsgen. Dr. Baffa is a medical professional and industry veteran with extensive experiences in clinical development and translational sciences in the field of oncology and cellular immunotherapy. Dr. Baffa brings a wealth of clinical, medical, and leadership experiences from multinational corporations and biotechnology companies. Dr. Baffa joins us at an exciting time as the clinical trials for both CT053 and CT041 are progressing in North America and being explored in other territories. Dr. Baffa’s joining will further accelerate the global clinical development of our innovative product candidates."

Dr. Raffaele Baffa, Chief Medical Officer of CARsgen Therapeutics Holdings Limited, said, "I am very excited to join CARsgen. As one of the leading companies in cell therapies globally, CARsgen has developed a rich and differentiated product pipeline and a suite of exciting technology platforms such as THANK-uCAR for allogeneic CAR T and LADAR for precise targeting. CARsgen has also shown big ambitions with continuous investment for the global market, such as the establishment of the manufacturing facility in the United States. I look forward to working closely with colleagues in CARsgen and external partners to accelerate CARsgen’s global clinical development and bring more innovative products to cancer patients."

CytoImmune to Participate in Upcoming April Investor Conferences

On April 7, 2022 CytoImmune Therapeutics, a clinical-stage immunotherapy company that is developing a novel class of engineered natural killer (NK) cell-based cancer therapies, reported that Christina Coughlin, M.D., Ph.D., chief executive officer, will participate in the following April investor conferences (Press release, CytoImmune Therapeutics, APR 7, 2022, View Source [SID1234611613]):

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Locust Walk Stem Cell Tx Conference in Partnership with Harvard Stem Cell Institute (formal presentation) on Monday, April 11, 2022, at 11:00 a.m. ET;
Needham 21st Annual Virtual Healthcare Conference’s "NK Therapeutics Panel" on Thursday, April 14, 2022, at 11:45 a.m. ET; and,
Chardan Annual Genetic Medicines & Cell Therapy Manufacturing Summit (fireside chat) on Monday, April 25, 2022, at 9:00 a.m. ET.

Adicet Bio to Participate in Upcoming Investor Conferences

On April 7, 2022 Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing first-in-class allogeneic gamma delta chimeric antigen receptor (CAR) T cell therapies for cancer, reported that company management will participate in two upcoming investor conferences in April (Press release, Adicet Bio, APR 7, 2022, View Source [SID1234611612]).

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Details of the events are as follows:

Canaccord Genuity 2022 Horizons in Oncology Virtual Conference, April 14, 2022
Chen Schor, President and Chief Executive Officer, will participate in a panel discussion titled, "Gamma Delta T-cells: A differentiated approach" on Thursday, April 14, 2022, at 12:00 PM ET.

2022 Jefferies Biotech on the Bay Summit, April 26-27, 2022
Adicet’s management team will participate in 1×1 investor meetings at the St. Regis, Bal Harbour in Miami, Florida on April 27, 2022.

Synthekine Announces Multiple Poster Presentations Showcasing Its Three Distinct Cytokine Engineering Platforms at American Association for Cancer Research (AACR) 2022 Annual Meeting

On April 7, 2022 Synthekine Inc., an engineered cytokine therapeutics company, reported five poster presentations based on research spanning each of its three distinct cytokine engineering platforms at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 taking place in New Orleans, LA from April 8-13, 2022 (Press release, Synthekine, APR 7, 2022, View Source [SID1234611610]).

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"Over the past year, we have made advances with all three of our platforms, including moving our first cytokine partial agonist into the clinic, advancing a novel solid tumor cell therapy program using our orthogonal IL-2 receptor/ligand system into preclinical studies, and establishing our first partnership for surrogate cytokine agonists," said Debanjan Ray, Chief Executive Officer of Synthekine. "We are excited to present updates across all of our platforms at AACR (Free AACR Whitepaper) 2022 to showcase the progress we are making to develop biased cytokine therapeutics and advance cytokine science."

Details are as follows and available on the AACR (Free AACR Whitepaper) online itinerary planner:

Title: Trial in Progress: A Phase 1a/1b study of STK-012, an α/β IL-2 receptor selective partial agonist as monotherapy and in combination with pembrolizumab in advanced solid tumors (NCT05098132)
Session Title: Phase I Trials in Progress 2
Session Date & Time: April 13, 2022, 9:00 AM – 12:30 PM CT
Location: First floor exhibit hall D-H, poster section 35 (poster board: 5)
Abstract Number: CT244
Summary: A first-in-human, open-label, dose escalation and expansion study in adults with advanced solid tumors (NCT05098132). The objectives of this study are to evaluate the safety, pharmacokinetics, immunogenicity, preliminary efficacy, and pharmacodynamics of STK-012 as monotherapy and in combination with pembrolizumab. We announced dosing of the first patient earlier this year.

Title: Engineered human IL-2/IL-2Rβ orthogonal pairs selectively enhance anti-GPC3 CAR T cells to drive complete responses in solid epithelial tumor models
Session Title: Adoptive Cell Therapy 3
Session Date & Time: April 12, 2022, 9:00 AM – 12:30 PM CT
Location: First floor exhibit hall D-H, poster section 35 (poster board: 15)
Abstract Number: 2824
Summary: Here we demonstrate the ability of our orthogonal IL-2 system to enhance the anti-tumor activity and persistence of anti-glypican 3 (GPC3) CAR T cells in human hepatocellular cancer models leading to tumor rejection in the majority of mice. These findings demonstrate that the orthogonal IL-2 system has the potential to improve the efficacy and durability of CAR T therapy for solid tumor targets such as GPC3 by selectively expanding CAR-T cells in vivo, driving CAR-T cells into the tumor, and activating CAR-T cells in the tumor microenvironment.

Title: Orthogonal IL-2/IL-2Rβ signaling in adoptively transferred T cells controls tumor growth without the need for lymphodepletion in a B16 tumor model
Session Title: Adoptive Cell Therapy 2
Session Date & Time: April 10, 2022, 1:30 PM – 5:00 PM CT
Location: First floor exhibit hall D-H, poster section 37 (poster board: 21)
Abstract Number: 586
Summary: We have previously shown a human orthogonal IL-2 receptor/ligand system can selectively proliferate and activate adoptively transferred T-cells (ACTs) without peripheral expansion of native lymphocytes. We have also developed a mouse orthogonal IL-2 receptor/ligand system to show in immune competent mice that the approach can additionally obviate the requirement of lymphodepletion in adoptive cell therapies to improve cell engraftment, persistence and efficacy of ACTs.

Title: IL-2Rβ/IL-2Rγ synthetic cytokines induce activation of human T and NK cells
Session Title: Immunomodulatory Agents and Interventions 3
Session Date & Time: April 13, 2022, 9:00 AM – 12:30 PM CT
Location: First floor exhibit hall D-H, poster section 38 (poster board: 5)
Abstract Number: 4225
Summary: We have generated a series of functional IL-2Rβ/IL-2Rγ surrogate cytokine agonists comprising dimers of heavy chain single domain antibodies (VHH) specific to IL-2Rβ and IL-2Rγ. The surrogate cytokine agonists show a variety of signaling strengths and bias, demonstrating the diversity of molecules that can be generated with this platform.

Title: IL10/IL2 surrogate cytokine agonists
Session Title: Preclinical Immunotherapy
Session Date & Time: April 8, 2022, 12:00 PM – 1:00 PM CT
Location: E-poster website
Abstract Number: 5544
Summary: Designing surrogate cytokine agonists that pair non-natural cytokine receptors allows for generating molecules that can decouple the pleiotropy of cytokines by preferentially stimulating the desired cell population. Here, we have generated IL10Rα/IL2Rγ surrogate cytokine agonists that are biologically active and signal with varying strengths in T cells with little to no activity on monocytes, thus providing an opportunity to decouple the pleiotropy of IL10 and bias its activity toward cell populations associated with anti-tumor efficacy.

E-posters will be released on April 8, 2022 at 12:00 PM CT, and will be available to registered attendees through July 13, 2022.