On September 23, 2019 CARsgen Therapeutics Inc., a clinical-stage biopharmaceutical company reported the European Medicines Agency (EMA) has granted PRIority MEdicines (PRIME) eligibility to its investigational CAR-T cell therapy fully human anti-BCMA (B Cell Maturation Antigen) autologous chimeric antigen receptor (CAR) T Cells (ct053) for the treatment of relapsed or refractory multiple myeloma (Press release, Carsgen Therapeutics, SEP 23, 2019, View Source [SID1234539727]).

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PRIME eligibility was based on clinical data from an ongoing CT053 BCMA CAR-T phase 1 study in China. The results from the trial were presented at an oral presentation on September 14, 2019 in Boston at the 17th International Myeloma Workshop. As of June 30, 2019, 21 out of 24 myeloma patients (87.5%) who received a median of 4.5 prior lines of myeloma therapy showed objective response. 19 out of 24 patients (79.2%) achieved complete response. There was no grade 3 or higher cytokine release syndrome. The duration of response data will be reported at a future date.

"PRIME eligibility is an important regulatory milestone in the continued development and commercialization of CT053 anti-BCMA CAR T cells," said Zonghai Li, M.D., Ph.D., chief executive officer of CARsgen. "CT053 has demonstrated the potential to become the best-in-class BCMA CAR-T therapy. PRIME designation is invaluable to the advancement of this cutting-edge therapeutic to potential market approval and being available to patients as quickly as possible." The CT053 anti-BCMA CAR-T program has received Investigational New Drug (IND) clearance and Orphan Drug designation from the U.S. Food and Drug Administration and authorization of its Clinical Trial Application (CTA) from Health Canada.

EMA’s voluntary PRIME scheme supports the development of promising medicines that show a high potential to benefit patients and target a significant unmet medical need. The status is granted to medicines that may offer a major therapeutic advantage over existing treatments. The program provides developers with enhanced interaction and early dialogue with the EMA to expedite drug development. Between the launch of PRIME in March 2016 through July 25, 2019, the EMA has received and assessed a total of 246 requests for eligibility. Of these, only 24 out of a total of 76 requests in oncology and hematology have been accepted into the scheme.

On September 23, 2019 I-Mab Biopharma ("I-Mab"), a China and U.S.-based clinical stage biopharmaceutical company exclusively focusing on the discovery and development of potential first-in-class and best-in-class biologics in immuno-oncology and autoimmune diseases, reported that it has entered into a clinical research collaboration agreement with MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ., USA, to evaluate the combination of I-Mab’s TJC4 and MSD’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in patients with multiple cancer types (Press release, I-Mab Biopharma, SEP 23, 2019, View Source [SID1234539726]).

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Under this collaboration agreement, MSD will supply KEYTRUDA (pembrolizumab) to I-Mab for use in clinical studies in combination with TJC4, a fully human anti-CD47 monoclonal antibody. Both parties will collaborate on a Phase 1 clinical trial, based on an agreed and finalized protocol, to evaluate the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of TJC4 and KEYTRUDA in patients with multiple cancer types.

"TJC4 is a highly differentiated anti-CD47 antibody currently under clinical development. This collaboration will provide an excellent opportunity to further explore therapeutic potential of combining TJC4 with KEYTRUDA across different tumor types." said Dr. Jingwu Zang, Founder and Chairman of I-Mab.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

About TJC4

CD47 represents one of the most promising immuno-oncology targets. The current investigational drugs targeting the CD47-SIRPa pathway have undesirable properties of binding to human red blood cells (RBC) or platelets. Such unwanted binding properties have been shown in pre-clinical and clinical settings to cause hematologic side-effects, e.g., anemia and thrombocytopenia, and the so-called "antigen sink effect" that affects pharmacokinetics of the investigational drugs. TJC4 is a highly differentiated monoclonal antibody made, by design, to recognize a unique epitope on CD47 and avoid strong binding to red blood cells. As such, TJC4 did not cause anemia in non-human primate studies，while it was shown to maintain anti-tumor activities. TJC4 is currently under clinical development in the U.S. and has been granted an IND approval by the National Medical Products Administration (NMPA) to start clinical trials in China.

On September 23, 2019 Menarini reported that results from the preclinical study conducted by Menarini Ricerche on MEN1611, a potent and selective orally available phosphatidylinositol 3-kinase (PI3K) inhibitor currently in development for the treatment of breast cancer (Press release, Menarini, SEP 23, 2019, View Source [SID1234539725]). The poster entitled "Characterization of the mechanism of action and efficacy of MEN1611, a novel PI3K inhibitor, in breast cancer preclinical models" will be presented at the ESMO (Free ESMO Whitepaper) Annual Meeting 2019, which will take place in Barcelona on September 27th, during the poster display session scheduled on September 30th (time 12-13).

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The PI3K/AKT signaling pathway is often dysregulated in patients with cancer: about 25% of HER2-positive breast cancers carry PIK3CA gene mutations, known to confer resistance to anti-HER2 therapy (i.e. trastuzumab). This study, conducted in several HER2-positive PIK3CA-mutated breast cancer cell lines and patient-derived xenograft models, shows that MEN1611 is able to down-modulate the PI3K/AKT pathway both in vitro and in vivo, and acts synergistically when combined with trastuzumab; moreover, the in vivo efficacy in trastuzumab-resistant models is demonstrated by a long lasting antitumor activity.

These promising preclinical data, provide a strong rationale for MEN 1611 progression in clinical development in breast cancer patients. In this regard, the B-Precise-01 clinical trial (NCT03767335), a multicentre phase Ib study, is currently ongoing in Europe with the aim to evaluate the preliminary clinical activity of MEN1611 in combination with trastuzumab +/- fulvestrant, in patients affected by advanced or metastatic HER2-positive breast cancer, and to select the recommended phase 2 dose

This year Menarini Ricerche will be present at ESMO (Free ESMO Whitepaper) congress also with a dedicated area at the Menarini Silicon Biosystems booth, where it will be possible to get more detailed information about the entire Menarini oncology pipeline and the ongoing clinical trials. Indeed, MEN1611 is part of an innovative pipeline of investigational new drugs focused on precision medicine approaches, that represents the strong and growing commitment of Menarini to oncology and to patients affected by difficult to treat cancers.

On September 23, 2019 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported that it will participate in the 2019 Cantor Global Healthcare Conference in New York, NY (Press release, Halozyme, SEP 23, 2019, View Source [SID1234539724]). Dr. Helen Torley, president and chief executive officer, will represent the company in a question and answer session on Thursday, October 3 at 3:35 p.m. ET / 12:35 p.m. PT.

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A live webcast of the presentation can be accessed through the "Investors" section of www.halozyme.com, and a recording will be made available for 90 days following each event. To access a live webcast, please visit Halozyme’s website approximately 15 minutes prior to the presentation to register and download any necessary audio software.

On September 23, 2019 Phoenix Molecular Designs (PhoenixMD), a clinical stage biotechnology company developing precise cancer therapeutics targeting essential kinases, reported clearance from the Food and Drug Administration (FDA) for PhoenixMD’s Investigational New Drug (IND) application for its proprietary PMD-026 (Press release, PhoenixMD, SEP 23, 2019, View Source [SID1234539723]. That clearance permits PhoenixMD to begin patient enrollment into its Phase 1/1b clinical trial, which is expected to start in the United States during the fourth quarter of 2019.

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"Our IND clearance marks an important developmental milestone for PhoenixMD," said Sandra Dunn, Ph.D., chief executive officer of PhoenixMD. "To our knowledge, PMD-026 is the only purpose-built agent designed specifically to treat triple-negative breast cancer (TNBC). Additionally, we will be assessing the potential of PMD-026 to extend beyond TNBC and into other forms of advanced breast cancer."

PMD-026 is a proprietary first-in-class orally-available RSK (kinase) inhibitor being developed to treat certain forms of breast cancer. The Phase 1/1b clinical trial will evaluate safety, tolerability, pharmacokinetics and anti-tumor activity of PMD-026 in patients with advanced disease and a sub-group of women with TNBC. Importantly, this trial will include a CAP/CLIA certified companion diagnostic designed to detect RSK2 activation in breast tumors and to then correlate it with response to PMD-026. This is a new approach for the treatment of cancer that hinges on precision medicine and functional kinomics.

"TNBC patients with metastatic disease have limited treatment options and new drugs have not generally been specifically developed based on the biology of this type of tumor," said Gerrit Los, Ph.D., chief scientific officer of PhoenixMD. "We have had substantial interest from some of the top clinical oncology centers eager to participate in our upcoming trial, underscoring the need for treatment options for women with advanced disease."

About PMD-026
PhoenixMD’s lead candidate, PMD-026, is the first RSK inhibitor being developed for the treatment of TNBC. PMD-026 was precisely designed for TNBC because RSK2 was specifically identified as the key kinase, out of 519 kinases, that drives the growth of this breast cancer subtype. Preclinical data shows the potential for PMD-026 to be effective alone or in combination with conventional chemotherapies. It has the potential to be a platform technology for chemotherapy, hormone therapy and/or immunotherapy sensitization for a wide range of refractory cancers in the future.