On March 3, 2022 Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator drug, mocravimod, reported that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted orphan drug designation (ODD) to mocravimod for the treatment of Acute Myeloid Leukemia (AML) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT) (Press release, Priothera, MAR 3, 2022, View Source;fda-and-ema-grant-orphan-drug-designation-to-mocravimod-for-the-treatment-of-acute-myeloid-leukemia-aml-in-patients-undergoing-allogeneic-hematopoietic-stem-cell-transplantation-hsct-301494655.html [SID1234609500]). EMA’s ODD follows a recommendation from the Committee for Orphan Medicinal Products (COMP).

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Florent Gros, Co-Founder and CEO of Priothera, commented: "The orphan drug designations we received for mocravimod from both the FDA and EMA are important milestones towards addressing the urgent, unmet needs of AML patients. Allogenic stem cell transplantation is the only potentially curative approach for AML patients but has unacceptably high mortality rates with current treatments. We are looking forward to initiating the global Phase 2b clinical trial with mocravimod in multiple centers in the US, Europe and Asia in the coming months."

Mocravimod, a sphingosine 1 phosphate (S1P) receptor modulator which has been previously tested in multiple autoimmune indications, is being developed to enhance the curative potential of HSCT. Moreover, it has shown a clinically relevant benefit in an early clinical study in patients with hematologic malignancies undergoing HSCT.

A multicenter Phase 2b study evaluating the efficacy and safety of mocravimod as an adjunctive and maintenance therapy to HSCT in adult AML patients is planned for the second half of 2022. The study will include approximately 250 patients in several countries in Europe, the US and Asia, upon approvals from respective health authorities.

Orphan drug designation is reserved for medicines treating rare, life-threatening or chronically debilitating diseases.

About mocravimod

Mocravimod (also known as KRP203), is a novel, synthetic, sphingosine 1-phosphate receptor (S1PR) modulator with a long duration in the body. Phase 1 and Phase 2 trials successfully assessed mocravimod for safety and tolerability in several autoimmune indications. Promising data from a Phase 1b/2a clinical study in patients with hematological malignancies led Priothera to further develop mocravimod for the treatment of blood cancers.

Mocravimod will be investigated in a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic stem cell transplantation (HSCT). Allogenic HSCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages S1PR’s unique mode of action to maintain anti-leukemia activity – graft-versus leukemia (GVL) – while reducing tissue damage resulting from graft-versus-host disease (GVHD), a consequence of allogenic HSCT. This novel treatment approach – the only S1PR modulator treating blood cancers – tackles a high unmet medical need and intends to add quality life to patients.

On March 3, 2022 Bausch Health Companies Inc. (NYSE/TSX: BHC) ("Bausch Health" or the "Company") reported that the Company will participate at two upcoming investor conferences (Press release, Bausch Health, MAR 3, 2022, View Source [SID1234609499]).

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Joseph C. Papa, chairman and chief executive officer; Sam Eldessouky, executive vice president and chief financial officer; and Arthur J. Shannon, senior vice president and head of Investor Relations and Communications, are scheduled to participate at the virtual Cowen & Co. 42nd Annual Health Care Conference on March 8, 2022 at 12:50 p.m. ET and at the Barclays Global Healthcare Conference 2022 in Miami on March 15, 2022 at 11:15 a.m. ET.

A live webcast and audio archive of the conferences will be available on the Investor Relations page of the Bausch Health Companies Inc. website at: View Source

On March 3, 2022 Evogene Ltd. (NASDAQ: EVGN) (TASE: EVGN), a leading computational biology company focused on revolutionizing product discovery and development in multiple life-science based industries, reported that it will present at the 34th Annual ROTH Conference being held both virtually and in-person in Dana Point, California on March 13-15, 2022 (Press release, Evogene, MAR 3, 2022, View Source [SID1234609498]).

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The conference will feature on-demand online and live presentations from companies across a variety of sectors, one-on-one and small group meetings, industry panels, and fireside chats. Past conferences have drawn thousands of attendees, including institutional investors, analysts, family offices and high net-worth investors.

Evogene’s CEO, Ofer Haviv will participate in a live, in-person fireside chat on Tuesday, March 15 at 12pm PT. Furthermore, Mr. Haviv, will participate in one-on-one meetings with institutional analysts and investors throughout the days of the conference.

Investors that wish to meet in-person with Mr. Haviv, are welcome to coordinate the meeting via their Roth representatives. Alternatively, investors may contact Evogene’s investor relations team which will coordinate either a face-to-face meeting with the CEO at the conference or alternatively a virtual meeting in the weeks following.

On March 3, 2022 I-Mab (the "Company") (Nasdaq: IMAB), a clinical-stage biopharmaceutical company committed to the discovery, development, and commercialization of novel biologics, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for TJ-CD4B, a novel Claudin 18.2 x 4-1BB bispecific antibody, for the treatment of gastric cancer including cancer of gastroesophageal junction (Press release, I-Mab Biopharma, MAR 3, 2022, View Source [SID1234609497]).

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"The Orphan Drug Designation underscores the FDA’s recognition of TJ-CD4B’s potential promise as a novel therapy for gastric cancer and other related cancers," said Dr. Andrew Zhu, President of I-Mab. "TJ-CD4B is designed to work through a novel mechanism of action and is being tested in cancer patients. With the Orphan Drug Designation, we expect to expedite its global clinical development and potentially offer a novel treatment option for such an aggressive group of cancers that have very poor prognosis."

TJ-CD4B is the first clinical-stage bispecific antibody that binds to Claudin 18.2 (CLDN18.2)-expressing cancer cells and co-stimulatory molecule 4-1BB on T cells to exert a tumor-killing effect. Unlike other therapies involving CLDN18.2, TJ-CD4B has a broader anti-tumor effect covering cancers expressing low levels of CLDN18.2. Preclinical studies have suggested that TJ-CD4B is superior to current CLDN18.2 antibodies and 4-1BB agonistic antibodies due to its stronger anti-tumor activity and a minimal 4-1BB related systemic toxicity.

TJ-CD4B is part of I-Mab’s highly innovative bispecific antibody pipeline. It is currently undergoing phase 1 clinical trials (NCT04900818) both in the U.S. and China in patients with advanced solid tumors, including gastric cancer, gastroesophageal junction carcinoma, esophageal adenocarcinoma, and pancreatic ductal carcinoma. I-Mab has made significant progress in the global clinical development of TJ-CD4B. In the ongoing dose-escalation study, TJ-CD4B was safe and well-tolerated at dose up to 3 mg/kg weekly. The Company plans to advance the study in biomarker-selected population subsequently.

Gastric cancer is the fifth most common cancer and third most common cause of death due to cancers globally. As the condition is asymptomatic in the early stages, it is generally diagnosed in advanced stages and carries a poor prognosis. Existing therapies for advanced refractory gastric cancer offer only modest clinical outcomes. In the U.S., gastric cancer is found in about 26,000 persons every year and accounts for about 1.5% of all new cancers diagnosed.[1] In China, gastric cancer is the second most frequently diagnosed cancer and the second leading cause of cancer–related deaths[2].

The FDA grants Orphan Drug Designation 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 Designation provides drug developers with various benefits designed to support the development of novel drugs and biologics, including market exclusivity for seven years upon FDA approval, tax credits for qualified clinical trials, and exemption from FDA application fees.

[1] American Cancer Society. Key Statistics about stomach cancer. Available at: View Source,6%2C740%20men%20and%204%2C440%20women)

[2] Bray F, Ferlay J, Soerjomataram I, Siegel RL, Torre LA, Jemal A. Global cancer statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2018;68(6):394–424.

About TJ-CD4B/ABL111

TJ-CD4B, also known as ABL111, is a Claudin 18.2 and 4-1BB bispecific antibody capable of binding to tumor cells expressing Claudin 18.2, i.e., gastric cancer and pancreatic cancer cells, and stimulating intra-tumoral T cells by the 4-1BB arm designed to be activated only upon tumor engagement while silent elsewhere. TJ-CD4B/ABL111 effectively maintains a strong tumor binding property and anti-tumor activity attributable to a synergistic effect of both Claudin 18.2 antibody and 4-1BB antibody while avoiding or minimizing liver toxicity and systemic immunotoxicity commonly seen with 4-1BB antibodies as a drug class. Being developed under collaboration between I-Mab and ABL Bio, a clinical-stage biotechnology company in South Korea, TJ-CD4B/ABL111 is currently being investigated in a phase 1 clinical study in the U.S. and China.

On March 3, 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 the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China has approved the initiation of the CT041 confirmatory Phase II clinical trial for the treatment of patients with Claudin18.2 (CLDN18.2) positive advanced gastric /gastroesophageal junction cancer (GC/GEJ) who have failed at least 2 prior lines of systemic therapies (Press release, Carsgen Therapeutics, MAR 3, 2022, View Source [SID1234609496]). The trial aims to evaluate the efficacy and safety of CT041 for the treatment of advanced GC/GEJ.

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As of this announcement, CT041 is the world’s first and only CAR T-cell product candidate entering a confirmatory Phase II clinical trial for the treatment of solid tumors.

Professor Lin Shen M.D., Vice President of Peking University Cancer Hospital, Director of Department of Gastrointestinal Oncology, Director of Phase I Clinical Study Ward and Deputy Director of Beijing Institute for Cancer Research, the principal investigator, commented that, "Gastric cancer is one of the most common and deadly cancers in China, with an annual incidence about 500,000. However, the treatment options for gastric cancer patients who have failed at least two prior lines are very limited. The median progression-free survival (mPFS) of available therapies, including anti-PD-1 monoclonal antibody, is merely about two months[1], indicating an urgent unmet medical need for more innovative and effective drugs. In the early study of CT041 led by our center, as presented in the ESMO (Free ESMO Whitepaper) 2021, the mPFS of CT041 was over 5 months, which is very promising data. We are eager to start the study and will try our best to develop effective cellular therapies for cancer patients."

Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics Holdings Limited, commented that, "I would like to thank all the investigators and patients for their efforts in the clinical trials of CT041. With the approval of CT041 to initiate the confirmatory Phase II clinical trial of gastric cancer, we are one step further to successfully obtain the CT041 marketing authorization. With our continuous research and development, we believe that CT041 has a great potential to change the treatment paradigm for advanced gastric cancer worldwide."

About CT041

CT041 is an autologous CAR T-cell product candidate against the protein CLDN18.2 that has the potential to be the first-in-class globally. CT041 targets the treatment of CLDN18.2 positive solid tumors with a primary focus on GC/GEJ and pancreatic cancer (PC). CT041 has demonstrated promising therapeutic efficacy and favorable safety in ongoing clinical trials. CARsgen believes that CT041 has the potential to become a backbone therapy for GC/GEJ and PC and benefit a large population of patients worldwide.

As of the date of this announcement, CT041 is the only CLDN18.2-targeted CAR T-cell product candidate that is being studied in clinical trials with IND approvals from the FDA and the NMPA and CTA approval from Health Canada. In addition to the investigator-initiated trials in China, CARsgen has initiated a Phase Ib clinical trial for advanced GC/GEJ and PC in China and a Phase 1b clinical trial for advanced gastric or pancreatic adenocarcinoma in North America. In 2020 and 2021, CT041 received Orphan Drug designation from the FDA for the treatment of GC/GEJ and Orphan Medicinal Product designation from the EMA for the treatment of advanced GC. In November 2021, CT041 was granted PRIME Eligibility by the EMA for the treatment of advanced GC. In January 2022, CT041 was granted Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of advanced GC/GEJ with CLDN18.2 positive tumor. CARsgen intends to conduct a pivotal Phase 2 clinical trial in North America in 2022.