On January 18, 2022 Celularity Inc. (Nasdaq: CELU) ("Celularity"), a clinical-stage biotechnology company developing placental-derived off-the-shelf allogeneic cell therapies, reported the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for its genetically modified cryopreserved human placental hematopoietic stem cell-derived natural killer (NK) cell therapy, CYNK-101, which is being developed in combination with standard chemotherapy, trastuzumab and pembrolizumab in patients in first-line locally advanced unresectable or metastatic HER2/neu positive gastric or gastroesophageal junction (G/GEJ) adenocarcinoma (Press release, Celularity, JAN 18, 2022, View Source [SID1234605527]). CYNK-101 is an investigational genetically modified NK cell therapy designed to synergize with approved antibody therapeutics through enhanced antibody-dependent cellular cytotoxicity (ADCC).

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Robert Hariri, M.D., Ph.D., Founder, Chairperson and Chief Executive Officer of Celularity, said, "We are extremely excited to receive this fast track designation and the support from the FDA for our investigational genetically modified NK cell therapy in the first-line setting of G/GEJ cancers. CYNK-101 is built on the foundation of our unique placental-derived source material, which as compared to other cell sources, has naturally enhanced proliferative potential (or "stemness"), that has been shown to be a determinant of persistence and efficacy potential. Using novel genetic engineering, we have enhanced the ability of CYNK-101 cells to synergize with approved antibodies and provide a novel and potentially non-cross resistant therapy to improve the lives of patients with G/GEJ cancers as well as a broad range of other indications."

"The addition of immune-based therapy of blocking PD-1 with a checkpoint inhibitor (pembrolizumab) to the prior standard of care (chemotherapy and traztuzumab) has recently been shown to be of benefit in patients with first-line HER2/neu positive unresectable G/GEJ cancer," added Andrew Pecora, M.D., President of Celularity. While overall response rates in first-line G/GEJ treated with the triple combination of chemotherapy, traztuzumab and pembrolizumab were significantly greater with the addition of pembrolizumab (74.4% vs 51.9%; p=0.000006; Keynote-811 trial of dual PD-1 and HER2 blockade in HER2-positive gastric cancer; Janjigian Y et al., Nature 600, 727-730 (2021), complete response rates remained modest, however (11.3%). "Our recently accepted IND enables the assessment to possibly further improve outcomes in G/GEJ treated with triple combination therapy by adding CYNK-101 cells, a potentially non-cross resistant therapy (enhanced ADCC, direct NK cell tumor killing and help of T cell function and memory) after initially cytoreducing the tumor mass and potentially diminishing resistance in the tumor microenvironment with combined chemotherapy, traztuzumab and pembrolizumab "induction" followed by reinduction and maintenance with CYNK-101 cells in combination with traztuzumab and pembrolizumab."

About Fast Track Designation

Fast Track Designation is an FDA process designed to facilitate the development and expedite the review of new drugs that are intended to treat a serious condition and have the potential to address unmet medical needs. The purpose of Fast Track designation is to expedite the process of getting important new drugs to patients. The designation may offer frequent interactions with the FDA review team on the product’s development and the product may be eligible for rolling review and priority review if certain criteria are met.

About Gastric Cancer

Gastric cancer is the fifth most common cancer worldwide(1). Despite recent improvements in treatment quality and options, advanced gastric cancer remains one of the hardest to cure cancers, with a median overall survival (OS) of 10–12 months and a five-year OS of approximately 5–20%. In May 2021, the U.S. FDA granted accelerated approval to pembrolizumab in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic HER2+ G/GEJ cancers (2)

REFERENCES

Bray F, Ferlay J, Soerjomataram I, et al. 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. doi:10.3322/caac.21492.
FDA grants accelerated approval to pembrolizumab for HER2-positive gastric cancer. Access here: View Source Accessed January 17, 2022.
About CYNK-001

Celularity’s lead therapeutic program based on its placental-derived unmodified NK cell type is CYNK-001, an allogeneic unmodified NK cell being developed as a treatment for hematologic malignancies, solid tumors, and infectious diseases. CYNK-001 is a cryopreserved allogeneic off-the-shelf cell therapy enriched for CD56+/CD3- NK cells expanded from human placental CD34+ cells. The safety and efficacy of CYNK-001 have not been established, and CYNK-001 has not been approved for any use by the U.S. Food and Drug Administration or any other analogous regulatory authority.

On January 17, 2022 Midatech Pharma PLC (AIM: MTPH; Nasdaq: MTP), a drug delivery technology company focused on improving the bio-delivery and biodistribution of medicines, reported that it is pleased to announce the extension of its R&D collaboration with Janssen Pharmaceutica NV (Janssen) originally announced on 21 July 2020 (Press release, Midatech Pharma, JAN 18, 2022, View Source [SID1234605544]).

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On 17 June 2021, the Company announced that, using its Q-Sphera technology, it had successfully encapsulated a proprietary Janssen experimental large molecule medicine and importantly preserved its functional integrity. The Company believes no other commercial or academic organisation has been able to successfully deliver any such experimental medicine over extended periods using methods capable of commercial scaling.

Building on this important discovery, the Company has extended its R&D collaboration with Janssen. Under the extended collaboration the Company will focus on maximizing drug loading and optimizing in vitro duration of release for this undisclosed Janssen experimental molecule using the Company’s Q-Sphera technology.

Dmitry Zamoryakhin, Chief Scientific Officer of Midatech, said:

"We are excited about the potential of our Q-Sphera technology in the delivery of API via long acting injectables. We look forward to our continued collaboration with Janssen and the application of our technology specific to these APIs."

This announcement contains inside information for the purposes of Article 7 of Regulation (EU) 596/2014 (MAR).

On January 18, 2022 TC BioPharm reported that Executive Chairman and Founder, Dr. Michael Leek will be participating in Advanced Therapies Week in Miami, Florida January 25-28 (Press release, TC Biopharm, JAN 18, 2022, View Source [SID1234605603]).

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Michael will participate in a session titled, Treating Cancer and Covid – Use of Banked Allogeneic Gamma-Delta T Cells in Oncology and Severe Viral Infection. The presentation will take place on Friday January 28th at 9:50 am ET at the Miami Beach Convention Centre. For more information or to register for the event, please visit; View Source

Dr. Michael Leek has 30 years’ experience in regenerative medicine, during which he progressed 10 different cell-based products from the laboratory into clinic. In 2017, Michael received the ‘Scottish Life-Sciences Entrepreneurial Business Leadership’ award for 2016-2017. Michael is also a Fellow of the Royal Society of Medicine and Honorary Lecturer at the University of Aberdeen, School of Medical Sciences.

Advanced Therapies Week is a large and immersive expo for companies in the cell and gene therapy sector. It has a focus on knowledge sharing, relationship building and deal making to advance a major pillar of medicine.

On January 18, 2022 Abbisko Therapeutics of Shanghai reported a global collaboration worth up to $258 million with Lilly to continue discovery and development of novel molecules for cardiometabolic diseases (Press release, ChinaBio, JAN 18, 2022, View Source [SID1234605528]). Abbisko will be responsible for further discovery/development of molecules that modulate a novel target using its proprietary R&D platform. If Abbisko successfully advances the compounds to their endpoints, Lilly will have the right to take over commercialization. If Lilly elects not to advance the compounds, Abbisko will have global rights to the candidate and pay milestones and royalties to Lilly.

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On January 18, 2022 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, today reported that it has received an updated independent safety review of certain preliminary data for the first 30 patients in its three Phase 1 clinical trials with Annamycin targeting relapsed or refractory acute myeloid leukemia (AML) and the metastases of soft tissue sarcoma to the lungs (STS Lung), which concluded there was no evidence of cardiotoxicity (Press release, Moleculin, JAN 18, 2022, View Source [SID1234605545]). The review included analysis of ejection fraction, echo strain and certain troponin levels intended to assess potential for both acute and chronic heart damage. Additionally, the Company reported evidence that Annamycin may have a substantially lower incidence of alopecia (hair loss) than currently prescribed anthracyclines such as doxorubicin. Although 65%-92% of patients treated with doxorubicin typically experience hair loss, the incidence to date in patients treated with Annamycin is less than 10%1. Alopecia is considered an important factor in quality of life for many cancer patients.

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Two of the three clinical trials, as described more fully below, are ongoing and the data from those trials remain preliminary and are subject to change and future updates. The ongoing trials are both in the dose escalation phase (Phase 1) with the goal of determining the recommended Phase 2 dose (RP2D). In the course of this dose escalation, 19 of the 30 patients that have been reviewed to date have now been dosed above the lifetime maximum anthracycline limit currently set by the US Food and Drug Administration (FDA), further underscoring the potential for Annamycin to improve patient safety.

"Annamycin was designed to produce little to no cardiotoxicity, so we are pleased to see that this updated report continues to support that objective," commented Walter Klemp, Chairman and CEO of Moleculin. "This is a critical safety improvement in the field of anthracyclines since the risk of cardiotoxicity is the primary limitation in the use of currently prescribed anthracyclines. The apparent reduction in the incidence of hair loss is an added benefit that has the potential to significantly improve the quality of life for patients needing anthracycline therapy. We are also encouraged by the pace of recruitment for the STS Lung clinical trial and look forward to hopefully continuing the current pace of cohort updates. While the European AML trial has been slow in recruiting, we expect to announce an update regarding the next cohort in the first quarter of 2022."

1 Gonzalez et al. 2018; DOXORUBICIN HYDROCHLORIDE [package insert]. New York, NJ: Pfizer Injectables; 2019

An expert in assessing cardiotoxicity associated with chemotherapy at the Cleveland Clinic, the author of the independent review of Annamycin’s cardiac safety data added, "Although anthracyclines continue to be a cornerstone of chemotherapy for many cancer indications, their use has been limited due to the threat of both acute and chronic cardiotoxicity. The availability of an anthracycline that eliminated this risk would be a major advancement in patient safety."

Dr. Sant Chawla, Director of the Sarcoma Oncology Center, Director of the Cancer Center of Southern California, and a Principal Investigator in Moleculin’s STS Lung clinical trial concluded, "Patients often face difficult choices in an effort to balance the objectives of cancer therapy with quality of life. Having the ability to offer my patients a treatment alternative that substantially reduces the risk of alopecia would make Annamycin a valuable new alternative for patients who until now have had limited options."

Summary of Annamycin Clinical Trials

STS Lung Clinical Trial

The Phase 1b/2 study is an ongoing U.S. multi-center, open-label, single-arm study, which in its Phase 1b stage, will determine the maximum tolerated dose (MTD) or the recommended Phase 2 dose (RP2D) and safety of Annamycin. The Phase 2 portion of the study will explore the efficacy of Annamycin as a single agent for the treatment of subjects with STS with lung metastases for whom prior chemotherapy has failed, and for whom new chemotherapy is considered appropriate. A minimum of three subjects will be enrolled in each cohort of the Phase 1b portion of the study until an MTD is identified, after which there will be a recommendation for the RP2D based on an assessment of both safety and efficacy. Up to 25 subjects will be enrolled at the RP2D in Phase 2 to further evaluate efficacy. The Company has now opened enrollment in the fourth cohort of the Phase 1b portion of the study with dosing increased to 390 mg/m2. Three subjects minimum (6 maximum) for this and each subsequent dosing cohort will be enrolled until a maximum tolerated dose is identified. Therefore, up to 36 subjects may be enrolled in the Phase 1b portion of the study.

AML Europe Phase 1/2 Clinical Trial

The Phase 1/2 AML trial in Poland remains ongoing and is currently dosing patients at 240 mg/m2. Under the previous protocol transient elevated liver enzymes (AST and ALT) observed in two patients were initially deemed to be a dose limiting toxicity (DLT), however investigators believed this would inappropriately limit the potential for continued dose escalation. An amendment to the Annamycin clinical trial protocol was therefore undertaken, which allows for a change in the DLT criteria as it relates to transient grade 3 elevations and allows for the dosing of three additional patients in the 240 mg/m2 cohort. If no DLT (as defined by the new criteria) is experienced with these next three patients, the Company plans to escalate dosing in new cohorts by 30 mg/m2 instead of the 60 mg/m2 previously planned, and with a de-escalation of 15 mg/m2 at the DLT dose if future patients experience a DLT.

AML US Phase 1/2 Clinical Trial

The results from the Phase 1 portion of the Company’s U.S. Phase 1/2 clinical trial of Annamycin for the treatment of AML met its primary endpoint and demonstrated a clean safety profile with no evidence of cardiotoxicity when delivered to patients at or below the lifetime maximum anthracycline dose established by the FDA. The Company is awaiting data from its European AML trial prior to continuing with the Phase 2 portion of this trial.

About Annamycin

Annamycin is the Company’s next-generation anthracycline that has been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin. Importantly, Annamycin has also demonstrated a lack of cardiotoxicity in multiple human clinical trials, including ongoing trials for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases, and the Company believes that the use of Annamycin may not face the same usage limitations imposed on doxorubicin, one of the most common currently prescribed anthracyclines. Annamycin is currently in development for the treatment of AML and STS lung metastases and the Company believes it may have the potential to treat a number of additional indications.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of STS lung metastases, in addition to Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia. For more information about the Phase 1b/2 study evaluating Annamycin for the treatment of STS lung metastases, please visit clinicaltrials.gov and reference identifier NCT04887298.