On September 21, 2022 PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, reported that checkpoint inhibitor (CPI) refractory patients have been selected as the optimal treatment group in the ongoing development of the PDS0101-based triple combination therapy in advanced HPV-positive cancers (Press release, PDS Biotechnology, SEP 21, 2022, View Source [SID1234621304]). After consulting with PDS Biotech, the study, conducted at the Center for Cancer Research (CCR) at the National Cancer Institute (NCI), one of the Institutes of the National Institutes of Health, has been closed to further enrollment given that the CPI refractory arm has been fully recruited. PDS Biotech and the NCI CCR plan to meet with the U.S. Food and Drug Administration (FDA) in the coming months to discuss the registrational path forward for the PDS0101-based triple combination therapy as a potential third-line treatment for CPI refractory, HPV-positive cancers.

The NCI-led Phase 2 clinical trial (NCT04287868) is investigating PDS0101 in combination with two investigational immune-modulating agents – M9241, a tumor-targeting IL-12 (immunocytokine), and bintrafusp alfa, a bifunctional checkpoint inhibitor (PD-L1/ TGF-β) – in recurrent or metastatic HPV-positive cancers in patients who have failed at least two standard of care therapies including CPI treatment. Both M9241 and bintrafusp alfa are owned by Merck KGaA. Data from this study, as presented at ASCO (Free ASCO Whitepaper) 2022, demonstrated that the triple combination therapy resulted in a 77% survival at a median of 12-months of follow-up in CPI refractory patients. Historical median survival rates for CPI refractory patients are 3-4 months.1 The triple combination therapy also appeared to be safe and well-tolerated with Grade 3 (43%) and Grade 4 (7%) treatment-related adverse events (AEs) reported.

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"The data generated in the CPI refractory arm of the Phase 2 trial investigating the PDS0101-based triple combination in advanced HPV-positive cancers seems compelling and potentially provides a foundation for advancing this program into late-stage clinical development," stated Dr. Frank Bedu-Addo, President and Chief Executive Officer of PDS Biotech. "For patients with CPI refractory HPV-associated disease, there is no clear effective standard of care therapy despite the severity of the disease. We intend to focus on the development of the triple combination therapy specifically for this patient group given the extreme unmet need coupled with the signals of efficacy observed in the CPI refractory Phase 2 trial arm. We believe this represents a significant value-building opportunity for PDS Biotech and our shareholders."

"The PDS Biotech team looks forward to our continued partnership with the NCI as we prepare to discuss our results in the CPI refractory population with the FDA to determine the most expeditious path forward," said Dr. Lauren V. Wood, Chief Medical Officer of PDS Biotech. "Accordingly, in collaboration with the NCI, PDS Biotech has decided to halt enrollment of the CPI naïve patient arm to allow both parties to direct resources on progressing the trial in CPI refractory patients."

On September 21, 2022 NKGen Biotech, a biotechnology company harnessing the power of the body’s immune system through the development of natural killer cell therapies, reported a publication in the peer-reviewed Journal of Cancer Research and Cellular Therapeutics entitled "Durable responses using SNK01 autologous enhanced natural killer cells and pembrolizumab for chemotherapy-resistant advanced sarcoma: Case reports, review of literature, and future perspectives (Press release, NKMax America, SEP 21, 2022, View Source [SID1234621303])."

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The paper by Gordon et al. discloses enduring response data in chemotherapy resistant metastatic sarcoma patients who were treated with SNK01, autologous enhanced natural killer cell therapy, and pembrolizumab, an immune checkpoint inhibitor, along with a review of literature and future perspectives on the use of SNK01 in advanced sarcoma.

"We are pleased to be able to share the promising results of our SNK01 natural killer cell therapy in combination with pembrolizumab, in these two important case studies since metastatic sarcoma is typically associated with poor outcomes when treated with standard regimens," commented Paul Song, M.D., Vice Chairman of NKGen Biotech. "It is encouraging to see that Patient #1, despite being PD-L1 negative and having failed prior pembrolizumab, eventually showed a gradual tumor reduction with a 47% partial response, after which the patient underwent two surgical resections and achieved a sustained remission. Of note is that the patient has safely been on this regimen for almost 35 months without any toxicity at all and remains no evidence of disease."

"Patient #2 has low PD-L1 expression and had failed prior nivolumab. The patient showed a gradual reduction in tumor size, achieving a 38% partial response," added Dr. Song, "This response data is very promising especially since both patients had failed prior immune checkpoint inhibitor therapy. It appears that SNK01 might be able to change the tumor microenvironment and suggests that SNK01 plus pembrolizumab has the potential to be a viable therapeutic option for patients with metastatic sarcoma, and especially in PD-L1 negative and low expressing tumors. Further studies are warranted, and we look forward to confirming these findings in a Phase II clinical trial."

The encouraging data from these two case reports suggests SNK01 combined with pembrolizumab may be a viable salvage therapy regimen for chemotherapy-resistant advanced sarcoma with manageable toxicity. The data also supports existing evidence that natural killer cells mediate a favorable response to immune checkpoint inhibitor therapy while reducing immune-related adverse events.

On September 21, 2022 NANOBIOTIX (Euronext: NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-clinical stage biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported determination of the recommended phase 2 dose (RP2D) of NBTXR3 in combination with pembrolizumab or nivolumab for the treatment of patients suffering from inoperable locoregional recurrent (LRR) or recurrent and metastatic (R/M) head and neck squamous cell carcinoma (HNSCC) that is resistant to prior immunotherapy (Press release, Nanobiotix, SEP 21, 2022, View Source [SID1234621302]). RP2Ds were determined for NBTXR3 plus pembrolizumab or nivolumab for patients with LRR or R/M HNSCC that has not received prior immunotherapy, lung metastases (mets) from any primary tumor, or liver mets from any primary tumor as well.

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The combined dose escalation and dose expansion parts of Study 1100 are expected to enroll up to 141 patients. The complete dose escalation part enrolled 29 patients in three cohorts: (i) head and neck lesions from LRR or R/M HNSCC eligible for anti-PD-1 therapy; (ii) lung mets from any primary cancer eligible for anti-PD-1 therapy; and (iii) liver mets from any primary cancer eligible for anti-PD-1 therapy. Study participants received a one-time intratumoral injection of NBTXR3 prior to their first radiotherapy session. They then received radiotherapy, followed by anti-PD-1. Based on the study’s results, the RP2D for all three cohorts was determined to be 33% of gross tumor volume.

Nanobiotix aims to deliver an industry-leading head and neck cancer treatment franchise powered by NBTXR3, and to replicate that approach across other solid tumor indications. Pursuant to this strategy, the Company has amended the ongoing expansion phase of Study 1100 to further strengthen the rationale behind a registrational protocol evaluating NBTXR3 plus anti-PD-1 for patients with R/M HNSCC that is resistant to prior immunotherapy.

The amended dose expansion part of study 1100 also has three cohorts, however the cohorts have been re-designed to further explore NBTXR3 plus anti-PD-1 in several immunotherapy-eligible indications, with a particular focus on the treatment of patients with LRR or R/M HNSCC primary lesions that are either naïve or resistant to prior immunotherapy. The expansion part cohorts are as follows: (i) LRR or R/M HNSCC that is resistant to prior immunotherapy; (ii) LRR or R/M HNSCC that has not received prior immunotherapy; (iii) lung, liver, or soft tissue mets from inoperable NSCLC, malignant melanoma, hepatocellular carcinoma, renal cell carcinoma (RCC), urothelial cancer, cervical cancer, or triple negative breast cancer (TNBC) primary tumors.

The Company expects to provide updated clinical data from Study 1100 in Q4 2022. The registrational phase 3 protocol submission is expected in Q1 2023, followed by modification of the study design.

About NBTXR3

NBTXR3 is a novel, potentially first-in-class oncology product composed of functionalized hafnium oxide nanoparticles administered via one-time intratumoral injection and activated by radiotherapy. The product candidate’s physical mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the physical MoA, Nanobiotix believes that NBTXR3 could be scalable across solid tumors that can be treated with radiotherapy and across different therapeutic combinations.

On September 21, 2022 MannKind Corporation (Nasdaq: MNKD), a company focused on the development and commercialization of innovative therapeutic products for patients with endocrine and orphan lung diseases, reported that its Chief Executive Officer, Michael Castagna, PharmD, will participate in the Lytham Partners Fall 2022 Investor Conference taking place virtually on September 28-29, 2022 (Press release, Mannkind, SEP 21, 2022, View Source [SID1234621301]).

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The Company’s webcast presentation will be available for viewing at 9:00am ET on Wednesday, September 28, 2022, on the Company’s website at Events & Presentations or View Source The webcast will also be archived and available for replay.

Management will be participating in virtual one-on-one meetings throughout the event. To arrange a meeting with management, please contact Lytham Partners at 1×[email protected].

On September 21, 2022 Kinnate Biopharma Inc. (Nasdaq: KNTE) ("Kinnate"), a clinical-stage precision oncology company, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for Kinnate’s investigational pan-RAF inhibitor, KIN-2787, for treatment of patients with BRAF Class II or III alteration-positive and/or NRAS mutation-positive stage IIb to IV malignant melanoma that is metastatic or unresectable (Press release, Kinnate Biopharma, SEP 21, 2022, View Source [SID1234621300]).

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According to the FDA, Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need. A therapeutic candidate that receives Fast Track designation is eligible for more frequent interactions with the FDA to discuss the candidate’s development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.

KIN-2787 is an orally available, potent and selective small molecule pan-RAF inhibitor being evaluated in KN-8701 (NCT04913285), an ongoing Phase 1 global clinical trial in patients with solid tumors harboring BRAF alterations or who have NRAS mutant melanoma. Unlike currently available treatments that target only Class I BRAF kinase mutations, Kinnate has designed KIN-2787 to target Class II and Class III BRAF alterations, where it has the potential to be a first-line targeted therapy, in addition to covering Class I BRAF alterations, and as a potential treatment for NRAS mutation-positive melanoma.

The company previously announced that the FDA granted Orphan Drug Designation (ODD) for KIN-2787 for the treatment of stage IIb-IV melanoma. An ODD is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the U.S.