On December 18, 2023 Notable Labs, Ltd. ("Notable") (Nasdaq: NTBL), a clinical-stage therapeutic platform company developing predictive precision medicines for cancer patients, and CicloMed, LLC ("CicloMed"), a developmental-stage pharmaceutical company focusing on unmet medical needs in oncology, reported initial safety and efficacy data of fosciclopirox and the performance of PPMP, Notable’s Predictive Precision Medicines Platform, from the Phase 2a trial in adult patients with relapsed/refractory acute myeloid leukemia, R/R AML (NCT04956042) (Press release, Notable Labs, DEC 18, 2023, View Source [SID1234638654]). Fosciclopirox is sponsored by CicloMed and co-developed by both companies. In the Phase 2a trial, PPMP-predicted patient responses were confirmed by the actual responses to treatment.

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"The successful outcome prediction means that Notable’s PPMP platform accomplished its goal in this trial," stated Thomas Bock, M.D., Chief Executive Officer of Notable. "These data further corroborate and extend our four independent PPMP validation trials in collaboration with leading medical centers*."

Fosciclopirox, a patented, parentally administered prodrug of a commercially available topical antifungal agent, ciclopirox, is being developed for the treatment of bladder cancer and AML.

This Phase 2a study was designed to characterize the safety and efficacy of fosciclopirox in R/R AML and, in parallel, to test PPMP’s ability to predict patient responses to treatment. Patients were not selected for enrollment based on PPMP results and, to avoid potential bias, PPMP analysis was blinded to the actual patient outcomes during the trial. Clinical efficacy in this heavily pretreated group of patients was defined by standard criteria in AML medical research and practice.

Eighteen heavily pre-treated patients were enrolled in the trial with nine patients evaluable for response assessment per-protocol. Fosciclopirox, administered at the Recommended Phase 2 Dose, was well-tolerated. However, none of the nine evaluable patients achieved a complete response. Stable disease, over four months, was observed in two evaluable patients.

Importantly, PPMP predicted that all patients enrolled into this trial would be non-responsive to fosciclopirox. The accuracy of this prediction was confirmed by the actual patient responses to treatment. These PPMP results indicate that the enrolled patient population was biased towards non-responsiveness to fosciclopirox and suggest that the negative clinical outcome of this Phase 2a trial may have been avoided if PPMP had initially been used to selectively enroll only predicted responders. Further analyses are ongoing.

"Obviously, we would have preferred observing therapeutic responses in this Phase 2a trial in heavily pretreated AML patients" shared Tammy Ham, Chief Executive Officer of CicloMed. "But based on the PPMP results, we will assess the merits of conducting a prospective trial in which we use PPMP to identify and selectively enroll patients predicted to respond to fosciclopirox treatment."

"Once our PPMP team merged the blinded PPMP and clinical data, we observed that the patients’ PPMP-predicted non-responsiveness to fosciclopirox was accurate and confirmed by the actual patient responses," said Joseph Wagner, PhD, Chief Scientific Officer of Notable. "This additional validation gives us further confidence that PPMP will play a significant role in drug development and suggests PPMP should be advanced to the front-end of clinical development programs to maximize success and avoid from the start, the risk and cost of enrolling treatment non-responsive patients."

On December 18, 2023 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that it will webcast its presentation at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 (Press release, Regeneron, DEC 18, 2023, View Source [SID1234638652]). The presentation is scheduled for 2:15 p.m. Pacific Time (5:15 p.m. Eastern Time) and may be accessed from the "Investors & Media" page of Regeneron’s website at View Source A replay and transcript of the webcast will be archived on the Company’s website for at least 30 days.

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On December 18, 2023 Propanc Biopharma, Inc. (OTC Pink: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that a boutique advisory firm has been engaged to identify strategic investment opportunities primarily to fund the advancement of the Company’s lead product candidate, PRP, to the completion of the Company’s planned Phase I, First-In-Human study in advanced cancer patients suffering from solid tumors (Press release, Propanc, DEC 18, 2023, View Source [SID1234638651]). Funds will also be utilized to advance the Company’s back up clinical candidate, Rec-PRP, a synthetic recombinant version of PRP, which will further enhance the potency and stability of the naturally derived proenzyme treatment. As part of the process, the opportunity to consider up-listing to a national US stock exchange will also be considered. The emerging boutique advisory firm currently has a $3B+ transaction history and access to a global network of investors the Company intends to fully utilize.

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PRP, a combination of two proenzymes trypsinogen and chymotrypsinogen, is a novel, targeted therapy for the treatment and prevention of metastatic cancer. Scientific evidence has shown potent effects against cancer stem cells, inducing these cells to differentiate (transform) into a less malignant, or dormant state, thus rendering them benign. Cancer stems cells represent the final frontier, as they are responsible for metastasis and invasion, causing the cancer to return and spread. They also eventually become resistant to standard treatment options like chemotherapy, where PRP has shown to be effective against chemoresistant pancreatic cancer cells, where patients suffer from a high mortality rate.

"PRP represents an exciting and novel method to control the threat of resistant tumors, which, if left untreated, can ultimately lead to patient death," said James Nathanielsz, Propanc’s Chief Executive Officer. "Importantly, PRP targets cancer stem cells, but leaves healthy stem cells alone. This means it is less toxic than standard treatments. We believe PRP could become a welcome addition to the treatment process for many aggressive cancer types, like pancreatic and ovarian cancers, where patient prognosis is often poor when left diagnosed. Therefore, we are offering an attractive investment opportunity to become involved in the development of a novel, first-in-class cancer therapy that as we progress further down the clinical development pathway, we believe has the potential to become a highly sought after strategic asset to the oncology portfolio of a global pharmaceutical, biotech, or mid-tier specialty company, serious about treating this killer disease."

On December 18, 2023 Philogen reported that Prof. Dr. Giuseppe Curigliano, is giving a presentation about the Phase I DODEKA study on February 26th, entitled "Phase I dose-escalation trial with tumor-targeted interleukin-12 (IL12-L19L19) in patients with solid tumors" (Press release, Philogen, DEC 18, 2023, https://www.philogen.com/2023/12/18/philogen-to-attend-the-esmo-tat-congress-in-paris-on-february-26-28-2024/ [SID1234638650]).

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Dr. Jacqueline Mock is presenting a poster about Philochem’s new small molecule drug conjugate on February 26th, entitled "Optimisation of FAP-targeted small molecule drug conjugates for the delivery and release of cytotoxic payloads in solid tumors"

On December 18, 2023 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported that US WorldMeds1 (USWM), a Kentucky-based specialty pharmaceutical company to whom it divested certain assets in its eflornithine pediatric neuroblastoma program, received FDA approval of its New Drug Application (NDA) for the use of eflornithine as a maintenance therapy for high-risk neuroblastoma patients who have achieved at least a partial response to certain prior therapies (Press release, Panbela Therapeutics, DEC 18, 2023, View Source;utm_medium=rss&utm_campaign=panbela-therapeutics-announces-us-worldmeds-nda-approval-for-eflornithine-dfmo-in-pediatric-neuroblastoma [SID1234638649]).

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The approval of USWM’s NDA for the use of eflornithine for the treatment of patients with high-risk neuroblastoma marks the first FDA approval of an NDA for any polyamine targeted therapy in a cancer indication. "This approval highlights the role polyamines can play in cancer therapy as we look forward to data from our ongoing programs in metastatic pancreatic cancer, colorectal cancer, non-small cell lung cancer, and prostate cancer and the advancement of preclinical programs in ovarian and multiple myeloma," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela.

In July 2023, Panbela divested its pediatric neuroblastoma program to USWM in an arrangement entitling Panbela to up to approximately $9.5 million of non-dilutive funding, including payments upon USWM’s successful completion of milestones related to eflornithine’s clinical development, regulatory approval, and commercial sales. "The FDA’s approval of USWM’s eflornithine NDA for high-risk neuroblastoma is an exciting milestone in our partnership. This demonstrates the potential for polyamine targeted therapies in cancer," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela.

"This approval is a prerequisite for considerable development milestone payments for Panbela as US WorldMeds continues its efforts to bring eflornithine to the market." "After many years investigating the role of polyamines and the therapeutic potential of eflornithine in neuroblastoma, it is rewarding to see USWM’s success with the FDA’s positive review of its NDA for eflornithine," said Elizabeth Bruckheimer, Ph.D., Vice President & Chief Scientific Officer of Panbela, "We are excited about how the USWM NDA approval of eflornithine should help address this high unmet need in patients with neuroblastoma."

Neuroblastoma, a rare cancer originating from immature nerve cells, contributes to nearly 15% of pediatric cancer deaths.[1] Panbela’s subsidiary, Cancer Prevention Pharmaceuticals, extensively collaborated with leading neuroblastoma research groups such as the Neuroblastoma Medulloblastoma Translational Research Consortium (NMTRC) (now Beat Childhood Cancer), New Advances in Neuroblastoma Therapy (NANT), the Children’s Oncology Group (COG), and the National Cancer Institute (NCI) in the clinical development of eflornithine as a treatment for neuroblastoma before the program was divested to USWM.