On January 25, 2024 QIAGEN (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported a first-in-kind collaboration with Penn State University in the United States to create a shared research and education facility for the fast-developing microbiome sciences (Press release, Qiagen, JAN 25, 2024, View Source [SID1234639480]).

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The university-industry partnership will serve as a beacon for this field by investigating research opportunities that address challenges and research gaps facing the microbiome, which involves the research into a community of microorganisms that can be found living together in any given environment, including the human body.

In doing so, this new partnership will provide QIAGEN with a site to support the development of new products as a testing center. It is also designed to provide vital industry research and training opportunities for next-generation scientists. This includes an internship program for graduate students from Penn State at QIAGEN laboratories at the European operational headquarters in Hilden, Germany, and helps them prepare for careers in the biotechnology industry.

"This new partnership with Penn State, one of the leading academic research institutions in the field of microbiome research, is anchored by a shared vision for an interconnection between the health of humans, animals and ecosystems. It will help shape research, education and outreach in the young field of the microbiome sciences, and hopefully accelerate the careers of a new generation of scientists interested in this field," said Nitin Sood, Senior Vice President and Head of the Life Sciences Business Area at QIAGEN. "Additionally, it will foster relationships with the microbiome research community and enable us to better develop new products for microbiome research based on direct customer feedback."

Seth Bordenstein, Director of the One Health Microbiome Center, Professor of Biology and Entomology, and Huck Endowed Chair in the Microbiome Sciences, at Penn State said: "The ‘One Health’ vision shared by QIAGEN and Penn State University is critical for microbiome research. We are pleased to partner with the leading provider of microbiome solutions to equip researchers with the tools to explore how microorganisms flow through humans, animals, plants and the environment, impacting the health of all these ecosystems. With this multi-year partnership, we will bridge the gap between industry and academia and shape the future of microbiome research."

Among the various projects in this partnership, the team will support the worldwide science education program "Discover the Microbes Within! The Wolbachia Project". This program enables students at the middle and high school levels, as well as those in college, to learn about arthropods (animals without backbones that have an outer skeleton made of chitin, segmented bodies and legs with joints, including insects, spiders, mites and crustaceans) and collect scientific data about the bacterial endosymbiont (an organism living inside another one for the benefit of both) Wolbachia pipientis.

This bacterium is estimated to be found in approximately 50% of the world’s arthropods and has been shown, for example, to block the reproduction of potentially fatal RNA viruses such as Dengue, West Nile and Zika virus[1]. Additionally, it is used to reduce the transmission of these and other viruses spread by the bite of infected arthropods (arboviruses)[2]. Due to these real-world impacts, Wolbachia is often used as a model organism to investigate animal-microbe interactions, genetics, evolution, ecology and human health.

Microbiome research aims to explore the relationships between microorganisms such as bacteria, fungi and viruses, and their hosts. It can help to better understand the microbiome’s impact on health, diseases and ecological processes in order to develop novel diagnostic solutions and therapeutic strategies.

The flagship project at the One Health Microbiome Center (OHMC) at Penn State’s Huck Institutes of the Life Sciences will see QIAGEN provide instruments and kits for preparing and processing microbial samples.

Penn State is among the top 30 public research universities in the U.S., with more than $1.2 billion in annual research expenditures. With over 500 members, including 125 faculty from various Penn State campuses and more than 42 departments, OHMC is one of the largest organizations of its type in microbiome research.

QIAGEN’s comprehensive microbiome portfolio encompasses tools for every aspect of the scientific workflow, including reliable sample preparation kits optimized for investigating challenging samples from environmental and human microbiomes. To ensure reproducibility, QIAGEN offers sample preparation automation for standardization and reliability. The extensive range of microbiome solutions also includes downstream processing technologies such as NGS, digital PCR (dPCR), or quantitative PCR (qPCR), all complemented by robust bioinformatics tools for seamless digital analysis.

Learn more about QIAGEN’s solutions for microbiome research at View Source

On January 25, 2024 Processa Pharmaceuticals, Inc. (Nasdaq: PCSA) ("Processa" or the "Company"), a clinical-stage pharmaceutical company focused on developing the next generation of chemotherapeutic drugs to improve the efficacy and safety for more patients suffering from cancer, reported the successful completion of the safety tolerability evaluation in its Phase 1b trial of Next Generation Capecitabine ("NGC-Cap") (Press release, Processa Pharmaceuticals, JAN 25, 2024, View Source [SID1234639479]). From the Phase 1b data, two dosage regimens have been selected for the Phase 2 trial. The Phase 2 trial will be in advanced or metastatic breast cancer given FDA’s agreement that the Phase 1b data can be used to support the design of the Phase 2 trial in breast cancer.

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NGC-Cap is PCS6422 administered in combination with capecitabine, a precursor of the cancer drug 5-FU. PCS6422 is administered as a single dose 12-24 hours prior to receiving seven days of capecitabine followed by seven drug-free days. The NGC-Cap Phase 1b trial evaluated capecitabine doses from 75 mg once a day (QD) to 225 mg twice a day (BID).

The 5-FU drug exposure for the 18 total patients that received NGC-Cap treatment across four different dosing regimens was 2-10 times that of FDA-approved capecitabine. Sixteen of these patients received at least two cycles of NGC-Cap with the other two patients discontinuing treatment before completing two cycles because of progressing disease. Four of the 16 patients are still receiving treatment in the study. At this time, only one of the 16 patients (6%) has experienced a mild case of hand-foot-syndrome (HFS), a side effect associated with the 5-FU metabolite fluoro-beta-alanine (FBAL). This lower incidence of HFS was expected given PCS6422 inhibits the metabolism of 5-FU to FBAL. The 6% incidence of HFS differs from the incidence reported for FDA-approved capecitabine, where greater than 50% of patients on capecitabine developed HFS and greater than 10% of the patients developed severe HFS.

The incidence of myelosuppression in patients on the high dose of NGC-Cap (225 mg BID) is currently approximately 71%, with more severe myelosuppression occurring in approximately 57% of the patients. The overall myelosuppression incidence rate after patients received the high dose of NGC-Cap is comparable to the 80% rate reported in the capecitabine label. As expected, given the greater 5-FU exposure, the incidence rate of more severe myelosuppression after the high dose of NGC-Cap was greater than the 3% rate reported for capecitabine. For the lower NGC-Cap dose of 150 mg BID, which also has a greater 5-FU exposure than capecitabine, the incidence of myelosuppression (33%) and more severe myelosuppression (0%) was lower for NGC-Cap compared to capecitabine.

Although the primary objective of the Phase 1b trial in patients with advanced gastrointestinal (GI) cancer was not to evaluate efficacy, the Phase 1b trial was designed to provide some preliminary data on efficacy. At this time, of the 11 cancer patients receiving one of the two highest doses of NGC-Cap, five have completed an efficacy evaluation at this time, and four of these five patients (or 80%) showed a positive response. One of these four patients had a partial response, and three patients demonstrated stable disease. Two patients receiving the highest dose will potentially become eligible for an efficacy evaluation by the end of the first quarter of 2024. Of the remaining four patients in the two highest doses, two dropped out prior to their evaluation because of disease progression and two dropped out because of side effects.

"Processa is very grateful to the patients and their physicians who participated in this trial and have been instrumental in our reaching these impactful findings. We are encouraged that NGC-Cap in the Phase 1b trial was tolerated better than or similar to the existing FDA-approved capecitabine even though the exposure to NGC-Cap’s 5-FU cancer-treating metabolite was 2-10 times that of capecitabine," said David Young, PharmD, Ph.D., President of Research and Development at Processa. "This greater exposure suggests that NGC-Cap can distribute more 5-FU to the cancer cells, potentially forming more cancer-killing metabolites that, in our small number of patients, has shown to improve the cancer-killing effect of NGC-Cap over capecitabine."

"Given the beneficial 5-FU exposure findings, the safety results, and the preliminary efficacy evaluation, we believe that NGC-Cap may be able to provide patients with a better efficacy profile along with fewer side effects than the presently prescribed capecitabine. Assuming this is confirmed in subsequent clinical studies, this would allow us to treat more patients with an NGC-Cap optimal dose rather than having to decrease the dose or discontinue therapy because of the tolerability with approved capecitabine. The data obtained from the Phase 1b trial — together with the feedback from the FDA — have allowed us to develop a Phase 2 and 3 strategy that will likely be more efficient in terms of time and cost as well as lead to a greater likelihood of FDA approval as we expand into advanced or metastatic breast cancer in Phase 2," concluded Dr. Young.

About the Study

This Phase 1b dose-escalation study assessed overall safety, pharmacokinetics, and anti-tumor activity of capecitabine administered in combination with PCS6422 (NGC-Cap) in 18 patients after a single dose of PCS6422 followed 12-24 hours with seven days of capecitabine and seven drug-free days. Patients had advanced GI cancer even after multiple types of treatment. The primary objective was to estimate the recommended Phase 2 dose and the maximum tolerated dose. Patients received a single dose of PCS6422 (40 mg) to inhibit the metabolism of 5-FU followed by seven days of capecitabine (75 mg QD to 225 mg BID) and seven days drug-free for each cycle.

The most common treatment related to adverse events were myelosuppression (e.g., anemia, neutropenia), GI-related adverse events (e.g., vomiting, diarrhea), and mucositis. The maximum number of cycles received across all doses has been 26 cycles with four patients presently still on treatment. At the two highest dosing cohorts, there were four advanced GI cancer patients with disease control responses out of the five patients who 1) received at least two cycles of one of the two highest doses of NGC-cap and 2) had a cancer response evaluation. This includes three patients with stable disease and one patient who had a partial response.

About Capecitabine Administered with PCS6422 (NGC-Cap)

NGC-Cap combines the administration of PCS6422, the Company’s irreversible dihydropyrimidine dehydrogenase (DPD) enzyme inhibitor, with the administration of low doses of the commonly used chemotherapy Capecitabine.

Capecitabine is the oral form of 5-FU and, along with 5-FU, is among the most widely used chemotherapy drugs available, particularly for solid tumors. When metabolized (after oral ingestion), it becomes 5-FU in the body, which, in turn, metabolizes to molecules called anabolites that actively kill duplicating cells, such as cancer cells, and to molecules called catabolites that only cause side effects. The presence of the DPD enzyme plays an integral role in the undesirable conversion of 5-FU to catabolites.

PCS6422 is an analog of uracil that irreversibly inhibits DPD. PCS6422 is neither toxic nor active as a single agent in animals at comparable dose levels. However, when administered in combination with Capecitabine or 5-FU, PCS6422 decreases the metabolism of 5-FU to the catabolites that only cause side effects, allowing more of the 5-FU to distribute to cancer cells.

On January 25, 2024 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported that it has reached 50% enrollment for its ASPIRE global clinical trial in the first-line treatment of metastatic pancreatic cancer (Press release, Panbela Therapeutics, JAN 25, 2024, View Source;utm_medium=rss&utm_campaign=panbela-exceeds-50-enrollment-for-aspire-trial-in-pancreatic-cancer [SID1234639478]). ASPIRE is a global randomized, double-blind placebo-controlled clinical trial to evaluate ivospemin in combination with gemcitabine and nab-Paclitaxel in patients with metastatic pancreatic ductal adenocarcinoma. Detailed information on the trial can be located at View Source

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"We’re thrilled to have surpassed 50% enrollment for the ASPIRE trial and to be moving faster than originally projected," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela. "As we have reached the near full complement of sites open and enrolling, we have seen a steady cadence of enrollment, expecting full enrollment to be completed by Q1 2025. We are looking forward to the interim data analysis based on overall survival in mid-2024."

In the global ASPIRE trial, there are approximately 90 sites up and running throughout the United States, Europe, Australia, and South Korea. Panbela is committed to delivering a more effective treatment for pancreatic cancer, a deadly disease with few treatment options.

On January 25, 2024 GlycaNova AS, the Norwegian biotechnology company focused on structurally intact beta-glucans for human and animal health, reported that no serious or other adverse events have been reported to date in its pioneering placebo-controlled trial of LentinexHP, a proprietary immuno-modulatory beta-glucan, administered as a non-drug therapeutic supplement alongside chemotherapy in patients with late-stage colorectal cancer (Press release, GlycaNova, JAN 25, 2024, View Source;utm_medium=rss&utm_campaign=clinical-trial-update [SID1234639477]).

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LentinexHP is a novel beta-glucan derived from the shiitake mushroom and is produced by GlycaNova’s proprietary manufacturing process, which enables production of beta-glucans with high biological activity and high structural integrity, maintaining the triple helix structure found in nature.

The clinical trial of LentinexHP is approaching the halfway stage with 15 patients having now been treated out of a planned total of 36 patients. No serious, or other, adverse events have been reported to date, which was confirmed by an independent audit by a Clinical Research Associate (CRA). Also, there have been no deaths among the 15 patients, which include some on LentinexHP and some on placebo.

In addition, all patients who have completed the 12-week duration of the trial have elected to stay on the trial on LentinexHP during a 40-week follow-up period. Whilst data from the trial is still being collected, early indications are that patients on the trial have experienced an increase in energy, a reduction in fatigue and an overall improvement in the quality of life.

The trial is being conducted at the Specialised Hospital for Active Treatment in Oncology (SHATO) in Haskovo, Bulgaria, and is of a randomised, parallel group, placebo-controlled design. Recruitment of patients into the trial is ongoing, and the addition of a further clinical site in Bulgaria is being considered to increase the pace of enrolment.

The primary endpoint of the trial is quality of life of patients with stage 4 metastatic colorectal cancer and secondary endpoints include improved quality of life and overall survival after one year.

Patients on the trial are randomly assigned to receive either 2ml of LentinexHP, or placebo, taken orally, twice daily for 12 weeks. Quality of life questionnaires and other assessments are completed after six and 12 weeks. The primary endpoint of quality of life is assessed at week 12, and further secondary endpoints of quality of life are assessed at various times after 12 weeks. The secondary endpoint of overall survival will be measured after one year.

The rationale for conducting the trial is that the combination of a biologically active but non-toxic, non-drug therapeutic supplement, LentinexHP, brings the potential to reduce resistance to chemotherapy and to reduce toxicity to non-cancerous cells thereby improving patient outcomes. The Company has previously demonstrated1 that the mode of action of LentinexHP is to stimulate a number of immune system responses thereby augmenting the effects of chemotherapy.

Dr Bjørn Kristiansen, GlycaNova’s Founder and Chief Executive Officer, said:

"We are delighted to be approaching the halfway point in this pioneering clinical trial of LentinexHP, which is being given as a non-drug therapeutic supplement alongside chemotherapy in late-stage colorectal cancer. It is particularly encouraging that, with 15 patients having been dosed in the study, that there have been no serious or other adverse events and that there have been no patient deaths during the trial.

"Early indications are that patients are experiencing an increase in energy, a reduction in fatigue and an overall improvement in the quality of life. Enrolment into the trial is ongoing, and we look forward to the results of the trial in due course."

On January 25, 2024 FibroGen, Inc. (NASDAQ: FGEN) reported graduation and completion of the pamrevlumab experimental arm in the Pancreatic Cancer Action Network’s (PanCAN) Precision PromiseSM Phase 2/3 adaptive platform trial, which evaluates pamrevlumab in combination with the chemotherapy treatments gemcitabine and nab-paclitaxel for patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) (Press release, FibroGen, JAN 25, 2024, View Source [SID1234639476]). Topline data from this registration study is anticipated in the second quarter of 2024.

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Pamrevlumab, in Stage 1 of the trial, achieved a protocol pre-specified ≥ 35% predictive probability of success for the primary endpoint of overall survival at the completion of the trial, and thus graduated to the second and final Stage of the trial in the third quarter of 2022. Pamrevlumab is the first experimental arm to meet its required threshold for graduation to Stage 2 in the adaptive platform trial. The pamrevlumab arm of the study completed Stage 2 of the trial in January 2024.

"We are excited to announce the achievement of this significant milestone in Precision PromiseSM. The graduation and completion of the pamrevlumab arm of this study brings a potential new therapy one step closer for patients with metastatic pancreatic cancer, an underserved population with limited treatment options," said Thane Wettig, Chief Executive Officer, FibroGen. "We look forward to continuing to work with PanCAN in sharing the topline data in the coming months."

"We are enthusiastic about the graduation and completion of the pamrevlumab investigational arm in Precision PromiseSM and look forward to seeing the final study results, which could potentially lead to a new treatment option for pancreatic cancer patients," said Julie Fleshman, JD, MBA, President and CEO of PanCAN.

PanCAN’s Precision PromiseSM adaptive platform trial (NCT04229004) is a U.S.-based, seamless Phase 2/3 study that enrolled patients in approximately 24 sites around the country. The multi-arm study consists of experimental treatment arms and two comparator arms: gemcitabine + nab-paclitaxel and mFOLFIRINOX. The pamrevlumab experimental arm was offered to patients with mPDAC as either a first-line (1L) or second-line (2L) treatment option. In the initial stage of the study (Stage 1), at least 100 patients with mPDAC received pamrevlumab in combination with gemcitabine and nab-paclitaxel. Guided by Bayesian principles, the graduation threshold for pamrevlumab was a protocol pre-specified ≥ 35% predictive probability of success for the primary endpoint of overall survival at the completion of the trial. Upon graduation, an additional 75 patients with mPDAC were enrolled (Stage 2), receiving the same pamrevlumab treatment regimen as in Stage 1. All patients are dosed until disease progression and the final analysis is based upon the data collected up to 12 months after the last patient initiates treatment in Stage 2. The combined Stage 1 and Stage 2 data will form the basis for analysis of the primary and secondary endpoints for the pamrevlumab arm. Secondary endpoints include progression-free survival (PFS), objective response rate (ORR), and duration of response (DOR), offering a comprehensive understanding of the treatment’s impact. Biomarker-related data, including genomics data, have been collected to provide additional insights into the trial’s outcomes. Precision PromiseSM is a registration study and the statistical design has been discussed by PanCAN with the FDA. The statistical design of Precision PromiseSM is led by renowned statistician Dr. Donald Berry of Berry Consulting.

About Metastatic Pancreatic Cancer
Over 30,000 patients are diagnosed with metastatic pancreatic cancer each year in the U.S. alone1 for which the five-year survival rate is 3 percent1.
In metastatic, or Stage IV, pancreatic cancer, the patient’s cancer has spread to another part of the body and therefore the cancer cannot be removed by surgery. Treatment at this stage includes systemic chemotherapy that travels through the bloodstream to reach cancer cells.

About Pamrevlumab
Pamrevlumab is a potential first-in-class antibody being developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF). Pamrevlumab is in clinical development for the treatment of metastatic pancreatic cancer and locally advanced unresectable pancreatic cancer (LAPC). The U.S. Food and Drug Administration has granted Orphan Drug Designation for the treatment of patients with pancreatic ductal adenocarcinoma (PDAC), and Fast Track designation to pamrevlumab for the treatment of patients with LAPC. Pamrevlumab has demonstrated a safety and tolerability profile that has supported ongoing clinical investigation in LAPC and metastatic pancreatic cancer. Pamrevlumab is an investigational drug and not approved for marketing by any regulatory authority. For information about our pamrevlumab studies please visit www.clinicaltrials.gov.