On February 9, 2023 Nexcella, Inc. ("Nexcella", "Company", "We" or "Us"), a biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications and a subsidiary of Immix Biopharma, Inc. (NASDAQ: IMMX), reported updated clinical data from its ongoing Phase 1 NEXICART-1 (NCT04720313) study of its novel, autologous, BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy NXC-201 for the treatment of patients with relapsed or refractory multiple myeloma and light chain amyloidosis (AL) (Press release, Immix Biopharma, FEB 9, 2023, View Source [SID1234626994]). The dataset represents 22 new evaluable patients in relapsed or refractory multiple myeloma and one new evaluable patient in AL (previous clinical data published in Haematologica 2022 and Clinical Cancer Research 2022). The new data are being presented during a poster presentation at the European Society for Blood and Marrow Transplantation and European Hematology Association (EHA) (Free EHA Whitepaper) 5th European CAR T-cell Meeting to be held in Rotterdam, Netherlands February 9-11, 2023.

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"I am pleased to present promising NXC-201 efficacy data from our interim Phase 1 dataset, which brings us one step closer to meeting an urgent need for greater access to CAR-T therapies that can considerably shorten treatment waiting time for patients with relapsed or refractory multiple myeloma," said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children and study investigator. "Also encouraging is that we have not yet reached median progression free survival or overall survival, which means that not only could we improve patient outcomes with efficacy, but we may be able to extend lives as well. I look forward to continuing to enroll patients in our ongoing NXC-201 clinical trial."

As of the data cutoff of October 23, 2022, 42 multiple myeloma patients were evaluable for efficacy and safety. These patients comprised the dose escalation cohorts for the first dose level (150 million CAR+ T cells, n=6), the second dose level (450 million CAR+T cells, n=7), and a dose expansion cohort at the recommended Phase 2 dose (RP2D) of 800 million CAR+T cells (n=29). This dataset represents 22 new evaluable patients in relapsed or refractory multiple myeloma at the NXC-201 RP2D.

The interim NXC-201 data demonstrate potentially meaningful efficacy and durable responses in relapsed or refractory patients who have a poor prognosis.

Of the 42 evaluable patients across all dose levels with median follow-up of 146 days (range, 18-314):
35 of 42 (83%) overall response rate (ORR) was achieved per International Myeloma Working Group criteria
21 of 42 (50%) patients achieved complete response (CR) or a stringent complete response (sCR)
34 of 42 (81%) patients achieved > very good partial response (VGPR)
1 of 42 (2%) patients achieved a partial response (PR)
Improved outcomes were observed in the 29 relapsed or refractory multiple myeloma patients receiving the therapeutic dose of NXC-201 (800 million CAR+T cells):
90% ORR was achieved per International Myeloma Working Group criteria
17 of 29 (59%) patients achieved CR or sCR
25 of 29 (86%) patients achieved > VGPR
1 of 29 (4%) patients achieved a PR
NXC-201 continues to be well-tolerated. Of the 42 evaluable patients:
No cases of immune effector cell-associated neurotoxicity syndrome (ICANs)
Low-grade CRS duration of median 2 days (range, 1-7 days) was observed, with the vast majority of CRS events starting on the day of infusion
"We are excited to present 42 patients of NXC-201 clinical trial data in Rotterdam. As of the data cutoff, 29 patients have been treated at our identified recommended phase 2 dose. We plan to continue to enroll additional NXC-201 patients at RP2D in Israel and expand to United States clinical trial sites," said Gabriel Morris, President of Nexcella.

Ilya Rachman, M.D., Executive Chairman of Nexcella added: "These data continue to highlight meaningful, durable responses with a favorable tolerability profile. We believe NXC-201 could be the world’s first out-patient CAR-T. These results reinforce our plan to advance NXC-201 to BLA submission in 1H 2025."

The poster can be accessed on the Nexcella corporate website at this link: View Source

Poster Presented:

Title: "Point-of-care CART manufacture and delivery: Expanding access to CART therapy via local institutions, Hadassah Medical Center experience"
Event: European Society for Blood and Marrow Transplantation and European Hematology Association (EHA) (Free EHA Whitepaper) 5th European CAR T-cell Meeting
Dates: February 9-11, 2023
Location: Postillion Hotel & Convention Centre WTC Rotterdam, Beursplein 37, 3011 AA Rotterdam, The Netherlands
Times: Thursday, February 9 12:00 – 20:30 CEST / Friday, February 10 08:00 – 18:30 CEST / Saturday, February 11 07:30 – 15:00 CEST

About NEXICART-1
NEXICART-1 (NCT04720313) is an ongoing Phase 1b/2, open-label study evaluating the safety and efficacy of NXC-201 (formerly HBI0101), in adults with relapsed or refractory multiple myeloma, all of which as of October 23, 2022 were triple-class refractory (to at least 1 immunomodulatory drug, 1 proteasome inhibitor and 1 anti-CD38 antibody).
The primary objective of the Phase 1b portion of the study, is to characterize the safety and confirm the Maximally Tolerated Dose (MTD) and Phase 2 dose of NXC-201. The Phase 2 portion of the study will evaluate the efficacy and safety of NXC-201 with endpoints of overall survival, progression-free survival and response rates according to International Myeloma Working Group (IMWG) Uniform Response Criteria.

About NXC-201
NXC-201 (formerly HBI0101) is a BCMA-targeted investigational chimeric antigen receptor T (CAR-T) cell therapy that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and AL amyloidosis. The design consists of a structurally differentiated CAR-T, with our proprietary BCMA-targeting CAR, which has demonstrated reduced toxicity in NEXICART-1, supporting investigating NXC-201 as an outpatient therapy.

About Multiple Myeloma
Multiple myeloma ("MM") is an incurable blood cancer of plasma cells that starts in the bone marrow and is characterized by an excessive proliferation of these cells. Despite initial remission, unfortunately, most patients are likely to relapse. There are 34,470 patients in the United States diagnosed with MM each year. Prognosis for patients who do not respond to or relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents remains poor.

On February 9, 2023 GSK plc (LSE/NYSE: GSK) reported that the US Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) voted 8 to 5 in support of the question posed to the committee regarding whether data from two proposed single-arm trials will be "sufficient to characterize the benefits and risks" of Jemperli (dostarlimab-gxly) in the curative-intent setting for patients with mismatch repair-deficient/microsatellite instability-high (dMMR/MSI-H) locally advanced rectal cancer (Press release, GlaxoSmithKline, FEB 9, 2023, View Source [SID1234626993]).

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Hesham Abdullah, Senior Vice President, Global Head of Oncology Development, GSK, said: "The Committee’s positive vote in favour of our proposed clinical trial programme for dostarlimab reinforces our plans to generate data in support of a future US regulatory submission for the potential treatment of patients with dMMR/MSI-H locally advanced rectal cancer, a patient population with significant unmet medical needs and a standard of care that results in serious quality of life concerns. We thank the committee for the constructive dialogue and we look forward to continued interactions with FDA as we progress our development programme."

The current standard of care (SoC) for patients with dMMR/MSI-H locally advanced rectal cancer is neoadjuvant chemoradiotherapy (CRT) followed by surgery and adjuvant chemotherapy.[i] Neoadjuvant CRT provides local tumour control in most patients, but nearly one-third ultimately die from distant metastasis[ii]. Additionally, SoC is associated with long-term adverse effects, including bowel, urinary and sexual dysfunction, secondary malignancy and infertility i.

As part of its proposed clinical trial programme, GSK is initiating a global, open-label, phase II clinical trial to investigate the efficacy and safety of dostarlimab-gxly as monotherapy – as a replacement for chemotherapy, radiation and/or surgery – for treatment-naïve patients with dMMR/MSI-H locally advanced rectal cancer. The primary endpoint of GSK’s proposed trial is clinical complete response for 12 months (cCR12) as assessed by Independent Central Review. Key secondary endpoints will include cCR for 36 months and event-free survival for three years by investigator assessment. In addition, the trial aims to confirm results generated in a separate ongoing investigator-initiated trial by researchers at Memorial Sloan Kettering Cancer Center (MSK). Researchers at MSK shared these findings in a late-breaking presentation at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting with simultaneous publication in The New England Journal of Medicine.i GSK intends to use data from the Company’s proposed trial, alongside data from MSK’s ongoing trial of 30 patients, to support a supplemental Biologics License Application (sBLA) for accelerated regulatory approval in this indication.

In January 2023, the US FDA granted dostarlimab-gxly Fast Track designation for the treatment of dMMR/MSI-H locally advanced rectal cancer. Fast Track designation is designed to accelerate the development and expedite the review of potential new medicines to treat serious conditions with unmet medical needs. In addition, the application could be eligible for priority review if supported by clinical data at the time of the submission to the FDA.

About dMMR/MSI-H rectal cancer

Rectal cancer is a form of cancer that starts in the rectum, the final section of the large intestine, and is often categorised as part of a group of cancers called colorectal cancer. Colorectal cancer is the third most commonly diagnosed cancer in the world.[iii] In the US, it is estimated that approximately 20,220 individuals are diagnosed annually with rectal cancer[iv]. Approximately 5-10% of all rectal cancers are dMMR/MSI-H, meaning that they contain abnormalities that affect the proper repair of DNA when copied in a cell.[v] Mismatch repair-deficient status is a biomarker that has been shown to predict response to immune checkpoint blockade with PD-1 therapy.[vi] [vii] Tumours with this biomarker are most commonly found in endometrial, colorectal and other gastrointestinal cancers but may also be found in other solid tumours.[viii] [ix] [x]

About Jemperli (dostarlimab-gxly)

Jemperli is a programmed death receptor-1 (PD-1)-blocking antibody that binds to the PD-1 receptor and blocks its interaction with the PD-1 ligands PD-L1 and PD-L2.[xi] GSK’s ambition is for dostarlimab to become the backbone of the Company’s ongoing immuno-oncology-based research and development programme when used alone and in combination with standard of care and future novel cancer therapies, particularly in patients with currently limited treatment options. Dostarlimab is being investigated in registrational enabling trials as monotherapy and as part of combination regimens, including in women with recurrent or primary advanced endometrial cancer, women with Stage III or IV non-mucinous epithelial ovarian cancer, and patients with other advanced solid tumours or metastatic cancers. Dostarlimab has not been approved anywhere in the world as monotherapy for treatment-naïve patients with dMMR/MSI-H locally advanced rectal cancer. The US FDA has granted dostarlimab Fast Track designation for the treatment of dMMR/MSI-H locally advanced rectal cancer.

Dostarlimab was discovered by AnaptysBio, Inc. and licensed to TESARO, Inc., under a collaboration and exclusive license agreement signed in March 2014. The collaboration has resulted in three monospecific antibody therapies that have progressed into the clinic. These are: dostarlimab (GSK4057190), a PD-1 antagonist; cobolimab, (GSK4069889), a TIM-3 antagonist; and GSK4074386, a LAG-3 antagonist. GSK is responsible for the ongoing research, development, commercialisation, and manufacturing of each of these medicines under the agreement.

Important Information for Jemperli in the EU

Indication

Jemperli is indicated as monotherapy for the treatment of adult patients with mismatch repair deficient (dMMR)/microsatellite instability high (MSI H) recurrent or advanced endometrial cancer that has progressed on or following prior treatment with a platinum containing regimen.

Refer to the Jemperli Reference Information for a full list of adverse events and the complete important safety information in the EU.

On February 9, 2023, F-star Therapeutics, Inc., a Delaware corporation (the "Company"), invoX Pharma Limited, a private limited company organized under the laws of England and Wales ("Parent") and Fennec Acquisition Incorporated, a Delaware corporation and a wholly owned subsidiary of Parent ("Purchaser" and together with the Company and Parent, the "Parties"), entered into Amendment No. 6 ("Amendment No. 6") to the Agreement and Plan of Merger, dated as of June 22, 2022, and as amended, by and among the Parties and Sino Biopharmaceutical Limited, a company organized under the laws of the Cayman Islands, as "Guarantor" (the "Merger Agreement"). Capitalized terms used in this Current Report on Form 8-K without being defined herein shall have the same meanings ascribed to them in the Merger Agreement (Filing, 8-K, F-star, FEB 9, 2023, View Source [SID1234626992]).

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The purpose of Amendment No. 6 is to extend the End Date of the Merger Agreement in order to provide additional time for the Parties to complete negotiations with the Committee on Foreign Investment in the United States ("CFIUS") on the definitive terms of a mitigation agreement and to complete the ongoing tender offer (the "Offer") whose expiration date has been extended to February 22, 2023, unless further extended, as described below.

The Parties believe they are in the late stages of negotiating definitive terms of such mitigation agreement in order to permit the removal of CFIUS’s Interim Order. However, there can be no assurances that the Parties will reach agreement with CFIUS on a mitigation agreement.

Other than as expressly modified pursuant to Amendment No. 6, the Merger Agreement, which was previously filed as Exhibit 2.1 to the Current Report on Form 8-K filed with the Securities and Exchange Commission (the "SEC") by the Company on June 23, 2022, remains in full force and effect as originally executed on June 22, 2022, as amended. The foregoing description of Amendment No. 6 does not purport to be complete and is subject to, and qualified in its entirety by, the full text of Amendment No. 6 attached hereto as Exhibit 2.1 to this Current Report on Form 8-K, which is incorporated herein by reference.

On February 9, 2023 Erasca presented its key milestones presentation (Presentation, Erasca, FEB 9, 2023, View Source [SID1234626990]).

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On February 9, 2023 Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, reported that it will present findings from its drug discovery and development programs at the 46th Annual MidWinter Meeting of the Association for Research in Otolaryngology (ARO), being held in Orlando, Florida February 11–15, 2023 (Press release, Decibel Therapeutics, FEB 9, 2023, View Source [SID1234626989]).

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The Company will present new data on its lead investigational gene therapy product candidate, DB-OTO, as well as new data across its gene therapy pipeline. Decibel will also present additional data from the interim analysis of the Phase 1b clinical trial of DB-020 in patients receiving cisplatin chemotherapy.

Podium Presentations

Podium 1 | Precise Targeting of GJB2 Cells Results in Safe and Efficacious Gene Therapy in a Rodent Model of Hearing Loss Due to GJB2 Deficiency
Presenter: Kathryn Ellis, Ph.D.
Session Title: Gene Therapy
Date & Time: Saturday, February 11, 2:00 – 4:00 p.m. ET

Podium 26 | Development of DB-020, a Locally Administered Product for Protection Against Cisplatin-Induced Ototoxicity
Presenter: John Lee
Session Title: Cisplatin Ototoxicity
Date & Time: Tuesday, February 14, 10:30 a.m. – 12:30 p.m. ET

Poster Presentations

SU83 | Development of a Platform for Parallel Functional Evaluation of Cell Type Specific Synthetic Promoters for Gene Therapy

SU84 | Dual Vector Gene Therapy Approaches for STRC-Related Hearing Loss

MO83 | Nonclinical Pharmacology, Biodistribution, and Safety Studies Supporting the Clinical Development of DB-OTO (AAV1-Myo15-hOTOFv5) for Hearing Loss Due to Genetic Otoferlin Protein Deficiency

TU83 | Development of a Utricle Culture System for Exploring Dual AAV-Mediated Expression Kinetics of Human Otoferlin

TU232 | Advancing Bioinformatically Informed Targets to Achieve In Vivo Vestibular Regeneration