On December 14, 2022 Immix Biopharma, Inc. (Nasdaq: IMMX) ("ImmixBio", "Company", "We" or "Us"), a biopharmaceutical company pioneering Tissue-Specific Therapeutics (TSTx)TM targeting oncology and immuno-dysregulated diseases, reported that it has in-licensed BCMA-targeted next-generation CAR-T therapy NXC-201 (formerly HBI0101) with 85% overall response rate (ORR) and 71% complete response/stringent complete response (CR/sCR) at the therapeutic dose from the first 20 patients in an ongoing phase 1b relapsed/refractory multiple myeloma ongoing clinical trial as of June 27, 2022 data cutoff with a median of 6 (range, 3-13) prior lines of therapy (Press release, Immix Biopharma, DEC 14, 2022, View Source [SID1234625263]). NXC-201 also produced 100% ORR and 100% organ response rate in 4 relapsed/refractory AL Amyloidosis patients. In addition, zero neurotoxicity of any grade was observed and zero events of immune effector cell-associated neurotoxicity syndrome (ICANs) were observed with NXC-201 treatment as of the June 27, 2022 data cutoff. These data were published in Haematologica View Source (multiple myeloma) and Clinical Cancer Research View Source (AL amyloidosis). Immix Biopharma has formed a wholly-owned subsidiary, Nexcella, Inc., to develop and potentially commercialize NXC-201.

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Low-grade (grade 1/2) CRS duration of median 2 days with median onset of 1-day post-dosing (range, 1-5 days) at therapeutic dose in relapsed/refractory multiple myeloma points to NXC-201 potentially becoming the first and only out-patient CAR-T for Multiple Myeloma, AL Amyloidosis and other BCMA-positive malignancies. The formation of Nexcella, Inc is expected to have a minimal impact on Immix Biopharma’s financial position, as Nexcella, Inc is expected to be an independently financed company.

ThinkEquity LLC acted as advisor to Immix Biopharma in this transaction.

"We are thrilled to share that we have secured exclusive rights to NXC-201, a next generation BCMA CAR-T therapy whose 85% ORR and 71% CR rate in heavily pretreated multiple myeloma patients in the ongoing Phase 1 trial are compelling," said Ilya Rachman, MD PhD, CEO of Immix Biopharma. "Additionally, encouraged by the 100% complete response rate and 100% organ response rate in AL Amyloidosis, we are planning to enroll additional patients to validate these findings."

"Considering its short 2-day median CRS duration, occurring within a median 1-day post-dosing, we look forward to exploring outpatient administration of NXC-201," added Gabriel Morris, CFO of Immix Biopharma.

About NEXICART-1

NEXICART-1 (NCT04720313) is an ongoing Phase 1b/2, open-label study evaluating the safety and efficacy of NXC-201 (formerly HBI10101), in adults with relapsed or refractory multiple myeloma, all of which as of June 27, 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 HBI10101) 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 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 December 14, 2022 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that the Safety Review Committee (SRC) has approved continuation to the third and final cohort in the dose escalation Phase 1 portion of the Acclaim-1 Phase 1/2 clinical trial of REQORSA in combination with Tagrisso (osimertinib) to treat late-stage non-small cell lung cancer (NSCLC) (Press release, Genprex, DEC 14, 2022, View Source [SID1234625259]). In 2020, the combination of REQORSA and osimertinib received U.S. Food and Drug Administration’s (FDA) Fast Track Designation for treatment of the Acclaim-1 patient population.

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Acclaim-1 is an open-label, multi-center Phase 1/2 clinical trial evaluating the Company’s lead drug candidate, REQORSA Immunogene Therapy, in combination with Tagrisso (osimertinib) in patients with late-stage non-small cell lung cancer (NSCLC) whose disease progressed after treatment with Tagrisso.

The SRC is comprised of three physicians who are principal investigators in the trial. The SRC may recommend that the trial continues at the same dose or at a lower dose, that it escalates to a higher dose, or that the study be terminated altogether due to safety concerns.

"The SRC’s recommendation to increase the dosing of REQORSA is further confirmation of its favorable safety profile and it enables us to advance Acclaim-1 into the final cohort of the Phase 1 dose escalation portion of the study," said Mark Berger, M.D., Chief Medical Officer of Genprex. "We look forward to completing enrollment of this final cohort in the first quarter of 2023."

The Accaim-1 trial includes up to three sequential dose escalation cohorts that will treat study participants with REQORSA intravenously on Day 1 in addition to osimertinib 80 mg fixed dose oral daily tablet during 21-day treatment cycles until disease progression or unacceptable toxicity. The first group received REQORSA IV infusion at 0.06 mg/kg, the second group received 0.09 mg/kg, and the third group will receive 0.12 mg/kg.

Following successful completion of the Phase 1 dose escalation portion of the Acclaim-1 study, the Company will advance into the dose expansion portion of the study, which will evaluate the toxicity profile of REQORSA in combination with Tagrisso in patients with different eligibility criteria, and will also evaluate efficacy and other endpoints.

"The principal advantage of adding the dose expansion portion to Acclaim-1 is to gain efficacy data earlier than we would otherwise have received it from the Phase 2 portion of the study. We also will receive this data in the two distinct patient populations represented by the two expansion cohorts, which we believe will further increase the likelihood of a successful Phase 2 trial," added Dr. Berger.

About Acclaim-1

The Acclaim-1 clinical trial is an open-label, multi-center Phase 1/2 clinical trial evaluating the Company’s lead drug candidate, REQORSA, in combination with Tagrisso in patients with late-stage NSCLC with activating epidermal growth factor receptor ("EGFR") mutations whose disease progressed after treatment with Tagrisso. Genprex expects the dose escalation Phase 1 portion of the Acclaim-1 trial to enroll up to 18 patients to determine the maximum tolerated dose of the combination. The dose expansion Phase 1 portion of the study will then enroll 66 patients, half of whom will be patients who had received only prior Tagrisso treatment and the other half patients who had received prior Tagrisso treatment and chemotherapy, to determine toxicity profiles of patients with different eligibility criteria, as well as efficacy and other endpoints. There will be an interim analysis following the treatment of 19 patients in each cohort. The Phase 2 portion of the study is expected to enroll approximately 74 patients to be randomized 1:1 to receive either REQORSA and Tagrisso combination therapy or Tagrisso monotherapy. The primary endpoint of the Phase 2 portion of the trial is progression-free survival, which is defined as time from randomization to progression or death. An interim analysis will be performed at 25 events.

About REQORSA Immunogene Therapy

REQORSA Immunogene Therapy (quaratusugene ozeplasmid) for non-small cell lung cancer (NSCLC) uses Genprex’s unique, proprietary ONCOPREX Nanoparticle Delivery System, which is the first systemic gene therapy delivery platform used for cancer in human clinical trials. The plasmid portion of REQORSA contains the TUSC2 gene, a tumor suppressor gene. REQORSA consists of the TUSC2 gene containing plasmid encapsulated in a nanoparticle made from lipid molecules with a net positive electrical charge. REQORSA is injected intravenously and is preferentially taken up by cancer cells. Once REQORSA is taken up into a cancer cell, the TUSC2 gene is expressed, and the TUSC2 protein is capable of restoring certain defective functions arising in the cancer cell. REQORSA has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for programmed cell death, or apoptosis, in cancer cells, and modulates the immune response against cancer cells.

Tagrisso is a registered trademark of AstraZeneca plc and its largest selling drug with 2021 sales of over $5 billion.

On December 14, 2022 Epigenomics Inc, a wholly-owned subsidiary of Epigenomics AG (Frankfurt Prime Standard: ECX, OTCQX: EPGNY; the "Company"), reported that it has licensed from The University of Texas MD Anderson Cancer Center certain patent and technology rights to biomarkers associated with colorectal cancer detection (Press release, Epigenomics, DEC 14, 2022, View Source [SID1234625256]).

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Epigenomics intends to utilize this technology to complement its "Next-Gen" blood-based colorectal cancer screening test, which includes protein biomarkers as well as the Company’s proprietary DNA methylation markers such as Septin9. The Company’s goal is to produce an annual screening test that outperforms the accuracy of FIT and can be performed by local, regional, and reference laboratories throughout the U.S.

"Epigenomics is committed to eliminating colorectal cancer and we believe we have the opportunity to create a novel cost-effective blood-based test that will play a significant role in reducing the mortality of the disease", said Greg Hamilton, CEO of Epigenomics.

On December 14, 2022 EpicentRx Inc. ("EpicentRx"), a leading-edge, clinical stage biopharmaceutical company that uses groundbreaking science to treat cancer and inflammatory-driven diseases, reported the publication of a randomized, Phase 2 trial which demonstrated a statistically significant and clinically meaningful progression-free survival (PFS) benefit for the small molecule NLRP3 inhibitor, RRx-001, plus irinotecan versus standard of care regorafenib in patients with advanced colorectal cancer (Press release, EpicentRx, DEC 14, 2022, View Source [SID1234625255]). The trial, ROCKET, included patients with third- or fourth-line colorectal cancer that previously received the irinotecan-containing chemotherapy regimen, FOLFIRI.

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The ROCKET data was published in the December 2022 issue of the journal Clinical Colorectal Cancer.

Regorafenib, a poorly tolerated multi-kinase inhibitor, was FDA-approved in third- or fourth-line colorectal cancer based on a Phase 3 study, which demonstrated a 6.4-month overall survival and a 1.9-month progression free survival.[1]

In the ROCKET trial, 34 patients were randomized 2:1 to receive a priming dose of RRx-001 4 mg IV weekly for two months followed by irinotecan, a chemotherapy agent, associated with a 40% rate of severe diarrhea, which they had previously received and failed, at a dose of 180 mg per meter squared given every two weeks until progression versus regorafenib 160 mg given by mouth, three weeks on/one week off until progression.

The median overall survival was 8.6 months for RRx-001 plus irinotecan and 4.7 months for regorafenib. Median progression free survival was 6.1 months for RRx-001 plus irinotecan vs. 1.7 months for regorafenib, a statistically significant result (two-sided log-rank test, p = 0.0030). Overall response rate was 20.8% for RRx-001 plus irinotecan vs. no response for regorafenib. Moreover, the toxicity profile of RRx-001 plus irinotecan was substantially improved compared with regorafenib. Also, there were no observed cases of severe diarrhea with irinotecan, likely due to protection of the gastrointestinal (GI) tract from RRx-001. Reduction or prevention of side effects from chemotherapy, like severe diarrhea, leads to less dose delays or dose reductions and consequently improves antitumor outcomes.

The prognosis for patients with CRC has historically been poor in later lines of therapy with response rates of approximately 1-2% and median PFS of approximately two months. [2]

According to EpicentRx CEO and practicing gastrointestinal oncologist, Dr. Tony Reid MD, PhD, "Granted, ROCKET was a small trial; however, as a GI oncologist, treatment with regorafenib is commonly associated with significant skin irritation and fatigue, among other symptoms. I think oncologists would welcome a better-tolerated, more active alternative for colorectal cancer patients that have progressed on front-line therapy. Hence, these results from ROCKET, if confirmed in a Phase 3 trial, would likely lead to additional options for third line colorectal cancer and beyond treatment."

About RRx-001
RRx-001 is a highly selective NLRP3 inhibitor with vascular normalization and tumor associated macrophage polarization properties that resensitizes tumors to previously administered therapies. RRx-001 is under investigation in a Phase 3 trial for the treatment of small cell lung cancer (SCLC), and in a Phase 2 trial for protection against oral mucositis in first line head and neck cancer. It is also under development as a medical countermeasure for nuclear and radiological emergencies and as a treatment for neurodegenerative diseases like Parkinson’s and ALS/MND. For more information visit www.epicentrx.com.

As previously disclosed, on July 30, 2021, Exicure, Inc. (the "Company") entered into a Collaboration, Option and License Agreement with (the "Ipsen Collaboration Agreement") with Ipsen Biopharm Limited (the "Ipsen"), pursuant to which the Company granted to Ipsen exclusive access and options to license SNA-based therapeutics arising from two collaboration programs related to the treatment of Huntington’s disease and Angelman syndrome (Press release, Exicure, DEC 14, 2022, View Source [SID1234625257]).

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On December 12, 2022, the Company and Ipsen entered into a Mutual Termination Agreement (the "Ipsen Termination Agreement"), pursuant to which the parties mutually agreed to terminate the Ipsen Collaboration Agreement. Following such termination, the parties will jointly own R&D Term IP (as defined in the Ipsen Collaboration Agreement) and Patents Covering the R&D Term IP (as defined in the Ipsen Collaboration Agreement), with each party owning an equal, undivided interest in and to such R&D Term IP and patents.

Termination of AbbVie Collaboration Agreement

On November 13, 2019, the Company entered into a Collaboration, Option and License Agreement (the "AbbVie Collaboration Agreement"), with a wholly-owned subsidiary of Allergan plc, Allergan Pharmaceuticals International Limited ("Allergan"). On May 8, 2020, Allergan plc, including Allergan was acquired by AbbVie Inc. ("AbbVie"). Pursuant to the AbbVie Collaboration Agreement, the Company granted to AbbVie exclusive access and options to license SNA-based therapeutics arising from two collaboration programs related to the treatment of hair loss disorders.

On December 13, 2022, the Company and Allergan entered into a letter agreement (the "AbbVie Termination Agreement"), pursuant to which the parties mutually agreed to terminate the AbbVie Collaboration Agreement. Following such termination, the Company will transfer to Allergan all data, information, and reports made or generated by the Company in the course of performing activities under the Development Plan (as defined in the AbbVie Collaboration Agreement), and grant to Allergan all rights to transfer, publish, present, or otherwise publicly disclose any Collaboration Technology (as defined in the AbbVie Collaboration Agreement) and data made or generated by the Company in the course of performing activities under the Development Plan.

As a result of the respective terminations of the Ipsen Collaboration Agreement and the AbbVie Collaboration Agreement, the Company regains the ability to independently develop medicines targeting hair loss disorders, Angelman syndrome, and Huntington’s disease whilst Ipsen retains the right to re-enter into the collaboration with the Company in Huntington’s Disease and Angelman’s Syndrome.

The foregoing descriptions of the Ipsen Termination Agreement and the AbbVie Termination Agreement do not purport to be complete and are qualified in their entirety by reference to such agreements, copies of which are filed as Exhibits 10.1 and 10.2, respectively, hereto and incorporated by reference herein.