On June 12, 2024 Sibylla Biotech and The University of Texas MD Anderson Cancer Center reported a strategic collaboration agreement to discover and develop novel small-molecule cancer therapies known as Folding Interfering Degraders (FIDs), which disrupt the proper folding of target proteins and lead to their degradation (Press release, Sibylla Biotech, JUN 12, 2024, View Source [SID1234644287]).

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Under the agreement, Sibylla and MD Anderson will jointly conduct discovery and development work from target identification through drug candidate nomination on selected proteins, with the potential to continue further pre-clinical and clinical development. The collaboration brings together Sibylla’s Pharmacological Protein Inactivation by Folding Intermediates Targeting (PPI-FIT) technology with the drug development expertise and capabilities of MD Anderson’s Therapeutics Discovery division.

"The Sibylla team is committed to expanding the applications of our PPI-FIT technology and FIDs to provide new treatment options for hard-to-treat indications. This collaboration builds upon the progress we have achieved to date and the applicability of our technology and know-how," said Lidia Pieri, PhD, MBA, Co-Founder and Chief Executive Officer of Sibylla Biotech. "We value the opportunity to work with MD Anderson’s team of drug development experts in order to foster our vision of treating patients with high unmet medical need."

Sibylla’s proprietary PPI-FIT technology is used to discover and develop FIDs, small molecules that induce the degradation of target proteins by interfering with the folding pathway. Notably, PPI-FIT can be applied to target proteins currently considered "undruggable" due to the absence of suitable pockets in their native state.

"Folding interfering degraders represent an exciting new modality to target key cancer drivers, and we look forward to opportunities to advance compelling candidates forward as novel therapeutic options," said Tim Heffernan, Ph.D., vice president and head of Therapeutics Discovery at MD Anderson. "By aligning Sibylla’s innovative PPI-FIT technology with our drug development engine, we hope to create impactful new medicines that will expand options for patients in need of more effective therapies."

By uniting scientists, clinicians and drug development experts together within MD Anderson, the institution’s Therapeutics Discovery division is designed to eliminate the bottlenecks slowing traditional drug discovery. Seamless integration with MD Anderson physicians allows Therapeutics Discovery to develop impactful cancer therapies inspired directly by patient needs and clinical insights.

Once a drug candidate is nominated, Sibylla and MD Anderson may consider further drug development, translational, and clinical activities to advance the candidate to patients in need.

On June 12, 2024 Race Oncology reported that the United States Food and Drug Administration (FDA) has extended Orphan Drug Designation (ODD) to our proprietary formulation of bisantrene, RC220, for acute myeloid leukemia (Press release, Race Oncology, JUN 12, 2024, View Source [SID1234644285]).

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Race CEO Dr Daniel Tillett spoke with Proactive about this significant announcement and the commercial advantage that the ODD will bring to bisantrene.

On Prelude therapeutics presented its corporate presentation (Presentation, Prelude Therapeutics, JUN 12, 2024, View Source [SID1234644283]).

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On June 12, 2024 PDS Biotechnology Corporation (Nasdaq: PDSB) ("PDS Biotech" or the "Company"), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines, reported a data update from its ongoing VERSATILE-002 Phase 2 clinical trial (Press release, PDS Biotechnology, JUN 12, 2024, View Source [SID1234644282]). VERSATILE-002 is evaluating Versamune HPV + KEYTRUDA (pembrolizumab) in patients with HPV16-positive head and neck squamous cell cancer ("HNSCC").

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The Kaplan-Meier analysis described below captures the survival data for immune checkpoint inhibitor ("ICI") naïve patients from the ongoing VERSATILE-002 Phase 2 clinical trial. All patients whose data are reported in the Kaplan-Meier analysis are properly censored to confirm their follow-up and survival status.

Based on a data cut as of May 17, 2024, the updated survival data for the cohort of ICI naïve patients after an additional follow-up of approximately 6 months in the VERSATILE-02 Phase 2 clinical trial with a total of 53 enrolled patients was as follows:

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mOS is 30 months, consistent with data presented at the Company’s Key Opinion Leader event on May 9, 2024, which was based on a data cut as of November 30, 2023.

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27 of the censored patients remained alive and were awaiting their next clinical assessment, 6 censored patients had withdrawn consent for further follow-up, and 2 patients had been lost to follow-up, and 18 patients had died.

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The lower limit of the 95% confidence interval is 19.7 months, and the upper limit is not yet estimable, as the majority of the patients continue to be followed for survival.

Full data from the May 17, 2024 data cut are expected to be announced in Q3 2024.

Dr. Kirk Shepard, M.D., Chief Medical Officer of PDS Biotech stated, "In recurrent and/or metastatic HNSCC objective response rate and progression-free survival have generally not translated into increased survival, and under current standards of care survival rates are well established to be less than 18 months. We believe that our VERSATILE-002 clinical trial and triple combination trial provide us with the critical survival information needed to effectively design the statistical primary endpoint in our planned Phase 3 trial".

The Company continues to advance its clinical strategy, which consists of a three arm registrational trial in first line treatment of HPV16-positive recurrent/metastatic HNSCC. The planned trial has two active arms: the double combination of Versamune HPV + pembrolizumab, and the triple combination of Versamune HPV + PDS01ADC + pembrolizumab. PDS01ADC is the Company’s tumor-targeted IL-12-fused antibody-drug conjugate ("ADC"), which has shown promise in ongoing Phase 2 clinical trials including a Phase 2 clinical trial of Versamune HPV + PDS01ADC + an investigational ICI conducted by the National Cancer Institute.

On June 12, 2024 NKGen Biotech, Inc. (Nasdaq: NKGN) ("NKGen" or the "Company"), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous, allogeneic and CAR-NK natural killer ("NK") cell therapeutics, reported details on its novel allogeneic blood-derived NK cell therapy ("SNK02") commercial manufacturing and cryopreservation process by Paul Y. Song, MD, Chairman and Chief Executive Officer of NKGen, entitled, "Protecting Patients by Removing Need for Lymphodepletion to Better Preserve Immune Function" (Press release, NKGEN Biotech, JUN 12, 2024, View Source [SID1234644281]). Dr. Song also provided an update on the Company’s initial Phase 1 results using SNK02 to treat patients with advanced refractory solid tumors.

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Dr. Song’s presentation explored the potential benefits of eliminating pre-treatment lymphodepletion in patients undergoing SNK02 therapy, aiming to safeguard immune function and aid in recovery. Avoiding lymphodepletion before administering cancer treatment can provide many benefits including reduced toxicity, preservation of immune function and potentially enhancing treatment efficacy. The presentation also included a discussion on the results from the Company’s Phase 1 SNK02 clinical study in solid tumors previously disclosed in a publication at the 2024 American Society for Clinical Oncology annual meeting. Moreover, unpublished Phase 1 SNK02 data were also presented.

The Phase 1 clinical trial administered SNK02 intravenously ("IV") weekly for a total of 8 weeks with a starting dose of 6 x 109 SNK02 cells in patients with advanced refractory solid tumors, without lymphodepletion. The primary endpoint was safety based on adverse events, vitals, laboratory tests and physical exams. Tolerability and maximum tolerated dose were also evaluated. SNK02 was found to be well tolerated as a monotherapy and appears to have some clinical activity against pretreated solid tumors despite the lack of lymphodepletion.

Dr. Song commented during the presentation, "As most of the focus of NK cell therapy has been for liquid tumors with lymphodepletion, we have always believed that lymphodepletion could be detrimental to patients with solid tumors especially those being treated with immune checkpoint inhibitors, monoclonal antibodies or bispecific therapies where a robust immune response is essential. We therefore set out to develop a commercial scale manufacturing and cryopreservation process which could yield greater than 100,00 doses of SNK02 (cryopreserved enhanced activated allogeneic NK cells) with the idea that large doses could be delivered without lymphodepletion to potentially overcome any host versus graft reaction. We are pleased to show that, despite developing autoantibodies to sustained repeated dosing of our allogeneic product, SNK02 was safe and treatment appeared to stop the progression of several heavily pretreated solid tumors as a monotherapy. We are excited to further explore the efficacy of SNK02 in combination with immune checkpoint inhibitors and antibodies against solid tumors."

Presentation Highlights:

In the Phase 1 SNK02 clinical trial, 6 patients, with advanced refractory solid tumors and an average of 4 prior lines of therapy, were enrolled. The median age was 64 years old (range, 44–71), and 4 were male.
The cancer subtypes included 2 colorectal cancers, 1 leiomyosarcoma, 1 angiosarcoma, 1 endometrial adenocarcinoma, and 1 undifferentiated pleomorphic sarcoma
Four of six patients completed 8 cycles of SNK02. The best objective response of stable disease (tumor stopped growing) was demonstrated in 100% of patients that completed the 8 cycles.
One patient received 18 consecutive weekly doses and another patient received 12 consecutive weekly doses.
Out of the 36 doses administered through Cycle 8, there were 17 Grade 1, 3 Grade 2, and 1 Grade 3 adverse events ("AEs") related to investigational product ("IP"). The Grade 3 AE of increased fatigue resolved after 1 day with no intervention required.
There was 1 death on study, which was deemed unrelated to the IP.
Auto-antibodies appeared to develop around Cycle 5 and appeared to correlate with AEs.
There did not appear to be any correlation with KIR mismatch or HLA subtyping with AEs or tumor response.
SNK02 was well-tolerated as a monotherapy and appears to have some clinical activity against pretreated solid tumors despite the lack of lymphodepletion. SNK02 will continue to be studied as a monotherapy and in potential combination treatment regimens with monoclonal antibodies and immune checkpoint inhibitors.
A copy of the presentation is available on the Scientific Publications page of the Company’s website at View Source Previously disclosed Phase 1 data on the positive effects of SNK02 on advanced solid tumors, which may not be included in this conference presentation, can also be found on the Scientific Publications page.

About SNK02

SNK02 is a novel cell-based, donor-derived ex vivo expanded allogeneic NK cell immunotherapeutic drug candidate. NKGen Biotech, Inc. is developing SNK02 for the treatment of a broad range of cancers.