Immix Biopharma, Inc. ("ImmixBio", "Company", "We" or "Us", "IMMX") (Nasdaq: IMMX), a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and autoimmune disease, reported that the 1st patient has been dosed at MSKCC in its U.S. NEXICART-2 trial with NXC-201, a sterically-optimized BCMA-targeted CAR-T cell therapy (Press release, Immix Biopharma, JUL 8, 2024, View Source [SID1234644712]).

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The NEXICART-2 study is intended to evaluate the safety and efficacy of NXC-201 in relapsed/refractory AL Amyloidosis patients with adequate cardiac function who have not been exposed to prior BCMA-targeted therapy. The study builds on positive data from the initial ex-U.S. study, NEXICART-1, presented at the 27th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT 2024) which showed a 92% overall response rate in relapsed/refractory AL Amyloidosis patients (12/13). The best responder experienced a 28.0 month duration of response (as reported May 10, 2024, with ongoing follow-up).

"I am excited to initiate the only CAR-T clinical trial currently recruiting for AL Amyloidosis patients that have progressed on front-line daratumumab (DARZALEX)-combination therapy," said Heather Landau, MD, Memorial Sloan Kettering Cancer Center Amyloidosis Program Director and NEXICART-2 principal study investigator. "A one-time therapy such as NXC-201 would provide an attractive option for AL Amyloidosis patients and clinicians. There are no approved drugs for relapsed/refractory AL Amyloidosis today."

"We believe initiation of the U.S. NEXICART-2 study is an important advancement in AL Amyloidosis. NXC-201 has the potential to become a first-in-class safe and durably-effective therapy for patients with relapsed/refractory AL Amyloidosis. We are humbled by the enthusiasm across the U.S. AL Amyloidosis community and look forward to robust enrollment for NEXICART-2," said Ilya Rachman, M.D., Ph.D., Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added, "Dosing of the first patient in line with our mid-2024 guidance is a testament to the stellar execution of our extraordinary team and partners."

NXC-201 is the only CAR-T therapy currently in development in AL Amyloidosis, mentioned in a review article entitled "Systemic Light Chain Amyloidosis" published in June, 2024 New England Journal of Medicine.

About NEXICART-2
NEXICART-2 (NCT06097832) is an open-label, single-arm, multi-site U.S. Phase 1b/2 dose expansion clinical trial of CAR-T NXC-201 in relapsed/refractory AL Amyloidosis. NEXICART-2 is expected to enroll 40 patients with adequate cardiac function who have not been exposed to prior BCMA-targeted therapy. The study is designed with a standard 6 patient safety-run in to evaluate two doses (three patients each at 150 million CAR+T cells and 450 million CAR+T cells), with the potential for further escalation to 800 million CAR+T cells (all 3 dose levels were evaluated in the NEXICART-1 study and have produced complete responses in relapsed/refractory AL Amyloidosis patients). The study aims to evaluate the safety and efficacy of NXC-201 in this patient population. Primary endpoints are complete response rate and overall response rate, according to consensus recommendations (Palladini et al. 2012).

About NEXICART-1
NEXICART-1 (NCT04720313) is an open-label, ex-U.S. Phase 1b/2a clinical trial of NXC-201 (formerly HBI0101) in patients with relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis (including AL Amyloidosis patients with impaired cardiac function and including AL Amyloidosis patients exposed to prior BCMA-targeted therapy). The primary objective of the study is to characterize the safety and efficacy, as well as confirm the recommended Phase 2 dose (RP2D) of NXC-201 (which has already been confirmed). NEXICART-1 clinical results, most recently from ASGCT (Free ASGCT Whitepaper) 2024, are available at View Source

About NXC-201
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy. Initial data from Phase 1b/2a ex-U.S. study NEXICART-1 has demonstrated short duration of cytokine release syndrome (CRS) and no grade 3 or delayed neurotoxicity in high-volume disease, as well as short duration CRS and no neurotoxicity of any kind in AL Amyloidosis.

NXC-201 is being studied in a comprehensive clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, with the potential to expand into autoimmune indications. The NXC-201 NEXICART-2 U.S. clinical trial builds on a robust clinical dataset. NXC-201 has been awarded Orphan Drug Designation (ODD) in the US by the FDA and in the EU by the EMA in AL Amyloidosis.

About AL Amyloidosis
AL amyloidosis is caused by abnormal plasma cells in the bone marrow, which produce misfolded amyloid proteins that build-up in the heart, kidney, liver, and other organs. This build-up causes progressive and widespread damage to multiple organs, including heart failure, and leads to high mortality rates.

The U.S. observed prevalence of relapsed/refractory AL Amyloidosis is estimated to be growing at 12% per year according to Staron, et al Blood Cancer Journal, to approximately 33,277 patients in 2024.

The Amyloidosis market was $3.6 billion in 2017, and is expected to reach $6 billion in 2025, according to Grand View Research.

On July 8, 2024, IDEAYA Biosciences, Inc. (the "Company") reported clinical data from the Company’s ongoing Phase 2 monotherapy expansion dose of IDE397 in patients with methylthioadenosine phosphorylase (MTAP)-deletion urothelial cancer and non-small cell lung cancer ("NSCLC") (Press release, Ideaya Biosciences, JUL 8, 2024, View Source [SID1234644711]). IDE397 is the Company’s potent and selective potential first-in-class methionine adenosyltransferase 2 alpha (MAT2A) inhibitor in Phase 2 clinical trials for the treatment of MTAP-deletion solid tumors.

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The interim Phase 2 monotherapy expansion data for IDE397 in MTAP-deletion urothelial cancer and NSCLC demonstrated preliminary clinical efficacy and a favorable safety profile. The reported Phase 2 clinical data are based on 18 evaluable patients enrolled in the IDE397 Phase 2 monotherapy study at the expansion dose of 30 mg once-a-day ("QD") of IDE397. The heavily pre-treated MTAP-deletion urothelial cancer and NSCLC patients, including seven urothelial cancer patients, four adenocarcinoma NSCLC patients and seven squamous NSCLC patients, had a median of two prior lines of therapy, ranging from one to seven prior lines of treatment. Reported clinical efficacy and tolerability data are preliminary and based on investigator review from an unlocked database as of the data analysis cutoff date of June 21, 2024.

The Company reported an overall response rate of approximately 39% (one complete response and six partial responses by RECIST 1.1 evaluation), which includes two unconfirmed partial responses (one urothelial cancer patient that had a 100% tumor reduction in the target lesion at the last CT-scan assessment and one adenocarcinoma NSCLC patient). The complete response and two of the partial responses observed were in urothelial cancer patients, and among the evaluable patients with lung cancer, three of the partial responses observed were in squamous NSCLC patients and one of the partial responses observed was in an adenocarcinoma NSCLC patient.

The Company also observed a disease control rate of 94%, including one complete response, six partial responses and ten stable disease by RECIST 1.1 evaluation. In addition, the Company observed tumor shrinkage in 14 of the 18 evaluable patients. Of the evaluable patients, 11 are still on treatment and five of the seven responses by RECIST 1.1 evaluation remain in response. The median duration of treatment, median duration of response and median progression free survival have not yet been reached.

The Company also reported a ctDNA molecular response ("MR") rate of 81%, representing 13 of 16 reportable patients with 50% or greater ctDNA reduction (several quality control failures of patient samples precluded the other patients from MR analysis). In addition, the 30 mg QD expansion dose achieved target drug coverage and plasma S-adenosyl-l-methionine pharmacodynamic reduction associated with preclinical tumor regressions.

The interim Phase 2 monotherapy expansion data for IDE397 in MTAP-deletion urothelial cancer and NSCLC demonstrated a favorable adverse event ("AE") profile at the 30 mg QD expansion dose. Approximately 5.6% of patients experienced a Grade 3 or higher drug-related AE at the 30 mg QD dose, represented by one instance of Grade 3 asthenia, and no drug-related serious adverse events were observed. The Company observed no drug-related AEs leading to discontinuations, and one non-evaluable patient discontinued treatment due to rapid clinical progression of cancer fatigue and drug-unrelated adverse events in the first cycle of treatment. The Company anticipates that the favorable AE profile and dosing convenience of a 30 mg QD tablet has the potential to enable long-term dosing and combination development.

There are currently no FDA-approved therapies for patients with MTAP-deletion solid tumors, highlighting the unmet medical need. The priority MTAP-deletion solid tumor types for the IDE397 Phase 2 monotherapy program are urothelial cancer and NSCLC. MTAP-deletion prevalence has been reported at over 15% in NSCLC and over 25% in urothelial cancer, based on The Cancer Genome Atlas ("TCGA") database. The Company estimates that the MTAP-deletion annual incidence in the U.S. in NSCLC and urothelial cancer is approximately 48,000 patients, based on the 2024 Surveillance, Epidemiology, and End Results database. In addition, there are several potential expansion MTAP-deletion solid tumor types that are also being considered for monotherapy and combination development, including pancreatic, head and neck, gastric, and squamous esophageal cancer, among others. Based on the TCGA database, MTAP-deletion prevalence in pancreatic, head and neck, gastric, and squamous esophageal cancer represents an aggregate U.S. annual incidence of approximately 27,000 patients.

The Company has activated over 35 clinical trial sites globally in the U.S., Canada, Europe, and Asia Pacific to enable potential rapid enrollment for the IDE397 Phase 2 monotherapy expansion in MTAP-deletion lung and bladder cancer in its ongoing trial (NCT04794699). There is also an Amgen-sponsored Phase 1/2 trial of IDE397 and AMG 193 combination in MTAP-Deletion NSCLC (NCT05975073) for which the companies intend to develop a joint publication strategy in 2024. In addition, the Company has initiated enrollment in a Phase 1 clinical trial evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of IDE397 in combination with Trodelvy (NCT04794699). The Company is also advancing multiple preclinical stage MTAP-deletion programs to enable wholly-owned combinations with IDE397, including a program targeting a development candidate nomination in the second half of 2024.

On July 8, 2024 Defence Therapeutics Inc. ("Defence" or the "Company"), (CSE: DTC, OTCQB: DTCFF, FSE: DTC), a Canadian biopharmaceutical company developing novel immune-oncology vaccines and drug delivery technologies, reported that it received a No Objection Letter ("NOL") from Health Canada for its Clinical Trial Study number ACCUM-002-01 entitles "A Phase I trial of Intratumoral Administration of ACCUM-002TM as a Monotherapy and in Combination with Opdualag in Patients with Unresectables, Stage IIIB and IV, Melanoma" (Press release, Defence Therapeutics, JUL 8, 2024, View Source;utm_medium=rss&utm_campaign=defence-receives-no-objection-letter-from-health-canada-for-phase-i-trial-of-its-accum-002-accutox-as-an-anti-cancer-molecule-in-patients-with-melanoma [SID1234644710]).

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The primary objectives of this study are to determine the safety and tolerability of intratumoral administration of AccuTOX, as a monotherapy and in combination with Opdualag which combines the LAG-3 checkpoint inhibitor relatlimab and PD-1 inhibitor nivolumab. The secondary objectives are to obtain preliminary efficacy data and to determine the Maximum Tolerated Dose ("MTD") and recommended Phase 2 Dose ("RP2D") of AccuTOX exploits as immune booster and anti-cancer molecule.

According to Vision Research Reports, the global cancer immunotherapy market size was estimated at USD 125.73 billion in 2023 and it is projected to increase USD 280.11 billion by 2033 with a CAGR of 8.34% from 2024 to 2033.

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The Defence’s team is already preparing this Phase I in Canada, most likely to be conducted in both Quebec and Ontario from which the hospitals sites will be confirmed upon agreements finalization.

"AccuTOX is Defence’s flagship asset in the anti-cancer therapeutics field and this Phase I clinical trial shall demonstrate and confirm primarily its safety and secondly its potency. We are thrilled to getting ready for this Phase I in Canada. Defence remains committed to its mission of addressing unmet clinical needs and in pursuing its goals to become a global leader in the development of innovative anti-cancer therapies," said Sebastien Plouffe, Chief Executive Officer of Defence Therapeutics.

On July 8, 2024 Iktos, a company sepecialized in Artificial Intelligence (AI) and Robotics for new drug design, reported the acquisition of Synsight, a French biotech company applying AI and high-content cell imaging to the discovery of novel drug candidates (Press release, Iktos, JUL 8, 2024, View Source [SID1234644709]).

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This acquisition brings significant advancements to Iktos’ drug discovery platform, now incorporating Synsight’s MT Bench technology for screening protein-protein and RNA-protein interactions (PPI and RPI). The merger also integrates automated biological testing into Iktos’ existing capabilities, which include generative AI design, retrosynthesis, and automated synthesis. This addition strengthens Iktos’ value chain by internalizing biological testing, accelerating the design-make-test process, and expanding drug discovery programs targeting critical disease mechanisms.

"This acquisition is an important milestone for Iktos", stated Yann Gaston-Mathé, co-founder and CEO of Iktos. "Iktos, a leader in AI-driven Drug Discovery, unlocks bottlenecks in challenging programs and accelerates identification of drug candidates. The acquisition of Synsight enables Iktos to expand its platform to new modulators of PPI’s and RPI’s, historically highly challenging targets, thereby addressing major unmet medical needs in various therapeutic areas such as cancer and neurodegenerative diseases."

On July 8, 2024 Century Therapeutics presented its corporate overview (Presentation, Century Therapeutics, JUL 8, 2024, View Source [SID1234644708]).

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