On January 24, 2022 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported the presentation of results of two trials evaluating eryaspase in advanced pancreatic cancer at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI) on January 21, 2022 (Press release, ERYtech Pharma, JAN 24, 2022, View Source [SID1234606729]).

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Abstract # 581 – rESPECT: A Phase I dose-escalation study of eryaspase in combination with modified FOLFIRINOX in locally advanced and metastatic pancreatic ductal adenocarcinoma: Interim update (NCT04292743).

Dr Marcus Noel, MD, medical oncologist at Georgetown University Lombardi Comprehensive Cancer Center, Washington, DC, USA, and principal investigator of the rESPECT trial, presented an interim update of this Phase 1 trial evaluating eryaspase in combination with mFOLFIRINOX, a fluoropyrimidine- and irinotecan-based chemotherapy regimen, as first-line treatment for advanced pancreatic cancer.

To date, eleven patients have been enrolled with a mean age of 68. Three patients were enrolled at a dose level of 75 U/kg and eight at a dose level of 100 U/kg. The novel combination of mFOLFIRINOX plus eryaspase was well tolerated and no dose limiting toxicity (DLT) was observed. The maximum tolerated dose (MTD) has been declared with 5-FU 2400 mg/m2, Oxaliplatin 85 mg/m2, Irinotecan 150 mg/m2 and eryaspase 100 units/kg.

Among ten patients with imaging available to evaluate response, 5 (50%) had partial response (PR) and 5 (50%) had stable disease (SD), corresponding to a disease control rate (PR + SD) of 100%. One patient with locally advanced disease became eligible for resection after receiving therapy.

Dr Marcus Noel, MD, Principal Investigator of the rESPECT IST, commented: "The early results from the rESPECT study are highly encouraging for the combination of eryaspase and modified FOLFIRINOX in first-line pancreatic cancer, with the MTD now established and initial signs of clinical activity demonstrated. We plan to expand enrolment on rESPECT to further evaluate efficacy. Alongside a group of international experts, we are now in the process of potentially considering a larger study in light of both our experience with rESPECT to date and the results observed in patients treated with the combination of eryaspase and FOLFIRI in TRYbeCA-1."

Abstract # 518 – TRYbeCA-1: A randomized, Phase 3 study of eryaspase in combination with chemotherapy versus chemotherapy alone as second-line treatment in patients with advanced pancreatic adenocarcinoma (NCT03665441).

Prof. Pascal Hammel, MD, PhD, medical oncologist at University Paris-Saclay, Hôpital Paul Brousse (APHP), France, and co-principal investigator of the TRYbeCA-1 trial, presented the final results of this Phase 3 trial. The top-line results had been reported in October 2021.

As reported before, the trial did not meet primary efficacy endpoint of overall survival (OS). The median OS for patients treated with eryaspase plus chemotherapy was 7.5 months (95% CI, 6.5-8.3), compared to 6.7 months (95% CI, 5.4-7.5) for chemotherapy alone.

The prespecified subgroup of patients treated with eryaspase and FOLFIRI, a fluoropyrimidine- and irinotecan-based chemotherapy regimen, demonstrated a nominal increase in median OS of 2.3 months versus FOLFIRI alone, from 5.7 to 8 months (HR = 0.81; 95% CI, 0.6-1.1). The FOLFIRI treated patients represented approximately 42% of the patients in this trial.

The treatment was well tolerated, and the addition of eryaspase did not enhance the cytotoxicity of chemotherapy.

Following Prof. Hammel’s presentation, the discussant, Dr Nilofer Saba Azad, MD, Department of Oncology, Johns Hopkins Sidney Kimmel Cancer Center, concluded that TRYbeCA-1 was well designed with appropriate power and stratification factors. Furthermore, the study findings open the possibility for additional study of eryaspase in combination with FOLFIRI in neo-adjuvant, adjuvant and first-line settings.

"While it was initially disappointing to be unable to demonstrate an improvement in survival across the entire population in one of the largest prospective second-line pancreatic cancer studies conducted to date, TRYbeCA-1 has improved our understanding of pancreatic cancer in this setting, and importantly, has also identified a potential utility of the combination of eryaspase with fluoropyrimide- and irinotecan-containing regimens in this difficult to treat cancer. I look forward to furthering discussions with the pancreatic cancer community on future potential clinical trials with eryaspase," said Prof. Pascal Hammel, MD, PhD, Co-principal Investigator of the TRYbeCA-1 trial.

Dr Iman El-Hariry, MD, PhD, Chief Medical Officer at ERYTECH, added: "I would like to thank all investigators and patients who are participating and have participated in the rESPECT and the TRYbeCA-1 studies. Pancreatic cancer remains very difficult to treat with few treatment options, but we have been encouraged by the clinical activity observed with eryaspase in both trials. We continue to evaluate the path forward for potential further clinical development focused on evaluating eryaspase in combination with fluoropyrimidine-and irinotecan-based chemotherapy. Meanwhile, the company is currently preparing a BLA to seek approval for eryaspase for the treatment of ALL patients who developed hypersensitivity to E. coli-derived asparaginase, based on the results of a Phase 2 clinical trial sponsored by the NOPHO group1. The Company intends to submit the BLA in the first quarter of 2022, subject to completion of remaining data requested by the FDA."

On January 24, 2022 RhoVac AB ("RhoVac") reported that the Canadian Intellectual Property Office ("CIPO") has issued a "Notice of Allowance", which means that it intends to grant RhoVac’s patent application for RV001 (onilcamotide) cancer vaccine (Press release, RhoVac, JAN 24, 2022, View Source [SID1234606747]). The company has previously been granted patents relating to onilcamotide in USA, Europe and Japan. The Canadian patent will provide RhoVac’s onilcamotide vaccine with broad protection in a key market and significantly strengthens the company’s patent portfolio.

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The Canadian Intellectual Property Office ("CIPO") has communicated its intention to grant RhoVac’s Patent Application No. 2,710,061. The patent covers RhoVac’s onilcamotide cancer vaccine, possible variants of the vaccine and the use of such vaccines in the treatment or prevention of metastatic cancer where RhoC is expressed. Once the issue fee is paid by RhoVac and necessary formalities have been completed by CIPO, the patent will be granted. The patent term will then extend to December 2028, potentially longer if an application for a Certificate of Supplementary Protection is filed and granted in Canada.

Patents from this first patent family have previously been granted in USA, Europe and Japan for treatment with onilcamotide of metastatic cancer expressing the RhoC protein. The Canadian patent, when granted, will complement these granted patents and another pending US application in this family

CEO Anders Månsson comments:"This patent provides very important protection for our cancer vaccine in Canada, which is an important territory for innovative therapies such as onilcamotide. This patent, when granted, will give product protection for our vaccine, along with additional protection for its use in the treatment and prevention of metastatic cancer [where RhoC is expressed], and in combination therapies".

On January 23, 2022 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with cancer reported the presentation of interim clinical data from its Phase 1b combination therapy study of SBP-101, a proprietary polyamine analogue, with gemcitabine and nab-paclitaxel in patients with metastatic pancreatic ductal adenocarcinoma (PDA), at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal (GI) Meeting that took place January 20-22, 2022 (Press release, Panbela Therapeutics, JAN 23, 2022, View Source [SID1234606716]).

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Jennifer K. Simpson, PhD, MSN, CRNP President & Chief Executive Officer of Panbela Therapeutics, commented, "We are excited to share interim data from cohort 4 and the expansion. A median overall survival (OS) of 12.0 months which is not yet final, and an objective response rate (ORR) of 48%, both exceeded historical rates reported for gemcitabine + nab paclitaxel and supports the continued development of SBP-101 as an addition to first-line treatment for advanced PDA and as neo-adjuvant treatment for patients with potentially resectable disease."

"The conclusion of the abstract is that SBP-101 may enhance first-line treatment with gemcitabine and nab-paclitaxel patients with metastatic PDA. We are encouraged by this conclusion even under sub-optimal conditions, including dose interruptions, which confounded results. Cohorts 2 and 3 did not have the dose interruptions that cohort 4 had, and cohort 2 had an objective response rate of 71%," continued Dr. Simpson. "We intend to continue development of SBP-101 and look forward to executing our global randomized phase 2 study in metastatic PDA."

At the Phase 1b dose and schedule (N=30), CA19-9 levels decreased 60-99% in 70% of evaluable patients, with 1/29 (3%) achieving a complete remission, 13/29 evaluable patients achieving partial responses (45%) and 10/29 achieving stable disease at 8 weeks (34%). PFS was 6.0 months. While PFS may be confounded by SBP-101 dosing holds implemented to investigate potential toxicity, the rates for 6-month PFS was 52% and for 12 month PFS was 10%. Nine subjects are in survival follow up as of the date the poster was presented at the ASCO (Free ASCO Whitepaper) GI meeting. Median OS is 12.0 months and is not final.

The safety population includes all subjects who received at least one dose of SBP-101 (N=50). The most common Grade ≥3 adverse events (AEs) related to any study medication were neutropenia in 20 subjects (19 attributed to G+A and 1 attributed to all 3) and elevated liver function tests in 14 subjects (5 attributed to SBP-101 and 9 attributed to all 3). SBP-101-related increases in LFTs were asymptomatic in all but 2 subjects and reversed in all subjects when SBP-101 administration was interrupted and dose-reduced or discontinued. Additionally, seven subjects experienced serious vision adverse events (4 possibly related to SBP-101, 1 related to gemcitabine and 2 related to all 3 based on PI assessment). All were considered by the sponsor to be possibly related to SBP-101; 5 had findings consistent with retinopathy.

The company has just begun a randomized trial to study SBP-101, as an addition to first-line treatment for metastatic PDA, will begin a neoadjuvant pancreatic trial this quarter and will begin an Ovarian Cancer program mid-year.

Additional meeting information can be found on the ASCO (Free ASCO Whitepaper) website at View Source . After presenting at ASCO (Free ASCO Whitepaper) GI, the poster will be available on the company’s website on January 24, 2022.

About SBP-101
SBP-101 is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. The molecule has shown potential signals of tumor growth inhibition in clinical studies of US and Australian metastatic pancreatic cancer patients, suggesting potential complementary activity with an existing FDA-approved standard chemotherapy regimen, if SPB-101 receives approval in the US. In data evaluated from clinical studies to date, SBP-101 has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events observed in the Company’s recently completed Phase 1a/1b clinical trial have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the current Panbela sponsored clinical trial provides support for continued evaluation of SBP-101 in a randomized clinical trial. For more information, please visit View Source .

On January 23, 2022 Applied Pharmaceutical Science, Inc. ("APS" or "the Company"), reported the Investigational New Drug (IND) application for its self-developed breakthrough new drug APS03118, a next generation selective RET inhibitor, has been approved by the U.S. Food and Drug Administration (FDA) (Press release, Applied Pharmaceutical Science, JAN 23, 2022, View Source [SID1234606699]). The clinical application is also in the process of being submitted to the National Medical Products Administration (NMPA) in China, and a global multi-center clinical trial is in the pipeline for initiation in the second quarter of 2022 in the U.S., China and Australia etc.

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APS03118 is a novel innovative drug developed by APS with global independent intellectual property rights for unlimited cancer types, targeting patients with non-small cell lung cancer, thyroid cancer, pancreatic cancer, breast cancer, ovarian cancer, colorectal cancer and other advanced solid tumors caused by rearranged during transfection (RET) gene alterations, as well as patients with resistance to first-generation selective RET inhibitors. The currently marketed first-generation selective RET inhibitors have both been granted priority review, breakthrough therapy, orphan drug status, accelerated approval and other review incentives by the FDA for their impressive efficacy.

Preclinical studies have shown that APS03118 is highly selective for RET kinases, and compared to the currently marketed first-generation selective RET inhibitors APS03118 showed significant nanomolar level potent antitumor activity in inhibition to various RET fusion and mutations including RET gatekeeper V804M/L/E and solvent frontier G810R/S/C mutations which lead to resistance to selective RET inhibitors. APS03118 also exhibited potent antitumor activity with a good safety profile in mouse models, and more significantly, in a brain tumor model, APS03118 completely eliminated brain tumors and all animals survived after dosing, demonstrating the therapeutic advantages of APS03118 for patients with brain metastases. It is potentially the best selective RET inhibitor of its kind globally.

Dr. Jun Zhong, Vice President of R&D at APS, said, "We are delighted that APS03118 has been clinically approved in the U.S. for a global unmet clinical need and that our self-developed innovative drug has been recognized by the FDA. APS has always adhered to its international development strategy to provide a new generation of precision therapeutic solutions for cancer patients worldwide."

On January 23, 2022 Ascletis Pharma Inc. (HKEX code: 1672) reported the dosing of the first patient in the Phase III registration clinical trial of ASC40 combined with bevacizumab for treatment of recurrent glioblastoma (rGBM) (Press release, Ascletis, JAN 23, 2022, View Source [SID1234606700]). ASC40 is an oral, selective inhibitor of fatty acid synthase (FASN), a key enzyme which regulates de novo lipogenesis (DNL) . ASC40 inhibits energy supply and disturbs membrane phospholipid composition of tumor cells by blocking de novo lipogenesis.

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The Phase III registration study (ClinicalTrials.gov Identifier: NCT05118776) is a randomized, double-blind, placebo-controlled, multi-center clinical trial in China to evaluate progression-free survival (PFS), overall survival (OS) and safety of patients with rGBM. Approximately 180 patients will be 1:1 randomized to Cohort 1 (oral ASC40 tablet once daily + Bevacizumab) and Cohort 2 (matching placebo tablet once daily + Bevacizumab). Approximately 80% of such 180 patients with rGBM in the Phase III clinical trial are expected to be randomized and enrolled by the end of December 2022.

The Phase II study, completed in the U.S., in patients with rGBM has shown that the objective response rate (ORR) for ASC40 plus Bevacizumab treatment was 65% including a complete response (CR) of 20% and a partial response (PR) of 45%.

Based on published data, in China, glioblastoma (GBM) represents 57% of gliomas and has an incidence rate of approximately 2.85 to 4.56 per 100,000 population per year, suggesting approximately 40,000 to 64,000 new cases of GBM per year. More than 90% GBM patients will relapse after surgery, radiation and chemotherapies. In the U.S., GBM represents 56.6% of gliomas and has an incidence rate of approximately 3.21 per 100,000 population per year.

"I am pleased that the first patient has been successfully dosed in the Phase III clinical trial of ASC40 combined with bevacizumab for treatment of recurrent glioblastoma. As the first clinical trial targeting tumor lipid metabolism in China, we are looking forward to the results of the trial," said Dr. Wenbin Li, Vice Chairman and Secretary General of Glioma Committee of Chinese Cancer Association, Director of the Comprehensive Tumor Treatment Center, Beijing Tiantan Hospital, Capital Medical University.

"Dosing the first patient in ASC40 Phase III registration study is a significant milestone for our oncology pipeline. We are looking forward to the data from this Phase III study," said Dr. Jinzi J. Wu, Founder, Chairman and CEO of Ascletis.

In Ascletis’ oncology pipeline, in addition to FASN inhibitors, there are two oral PD-L1 small molecule inhibitors developed in-house, namely ASC61 and ASC63. Ascletis has filed a U.S. Investigational New Drug (IND) application of ASC61 for the treatment of advanced solid tumors.