On January 19, 2023 PDS Biotechnology presenting its Oncology platfarms and clinical data summary (Presentation, PDS Biotechnology, JAN 19, 2023, View Source [SID1234626391]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On January 19, 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 the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has issued the Adoption of Commission Implementing Decision relating to the designation of ivospemin (SBP-101) as an orphan medicinal product in combination with gemcitabine and nab-Paclitaxel in patients with metastatic pancreatic ductal adenocarcinoma (PDA) (Press release, Panbela Therapeutics, JAN 19, 2023, View Source;utm_medium=rss&utm_campaign=panbela-announces-adoption-of-commission-implementing-decision-from-the-ema-for-the-orphan-designation-of-ivospemin-sbp-101 [SID1234626390]). The Commission adopted the decision on January 13, 2023 and it will be published for information in all official languages of the EU in the Community Register of Orphan Medicinal Products

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Previously, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to SBP-101.

"We are pleased to receive notice that the European Commission has adopted the positive decision for orphan drug designation from EMA’s Committee for Orphan Medicinal Products," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela. "The designation of orphan drug status within the EU is an important achievement as we continue to advance the global ASPIRE trial with the potential that this may be an option for patients with first line metastatic pancreatic cancer in the future."

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. The EMA’s orphan designation is available to companies’ developing treatments for life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the EU. Medicines that meet the EMA’s orphan designation criteria may qualify for financial and regulatory incentives, including a 10-year period of marketing exclusivity in the EU after product approval, protocol assistance from the EMA at reduced fees during the product development phase and access to centralized marketing authorization.

Panbela is continuing to focus on site initiation and enrollment in the ASPIRE trial to ultimately deliver a more effective treatment for pancreatic cancer, a deadly disease with few treatment options. The Company expects that the full complement of sites will be open by mid 2023.

About our Pipeline

The pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention and ovarian cancer. The combined development programs have a steady cadence of catalysts with programs ranging from pre-clinical to registration studies.

SBP-101 Ivospemin

Ivospemin 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. It has shown signals of tumor growth inhibition in clinical studies of metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months and an objective response rate (ORR) of 48%, both exceeding what is typical for the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, ivospemin has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events 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 previous Panbela-sponsored clinical trials provide support for continued evaluation of ivospemin in the ASPIRE trial. For more information, please visit View Source

Flynpovi

Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increase polyamine export and catabolism. In a Phase 3 clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase 3 trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), Flynpovi showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety profile for Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP.

CPP-1X

CPP-1X (eflornithine) is being developed as a single agent tablet or high dose power sachet for several indications including prevention of gastric cancer, treatment of neuroblastoma and recent onset Type 1 diabetes. Preclinical studies as well as Phase 1 or Phase 2 investigatorinitiated trials suggest that CPP-1X treatment may be well-tolerated and has potential activity.

On January 19, 2023 ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, reported positive results in its fully-enrolled metastatic pancreatic cancer study in third-line or greater subjects (QUILT 88) showing that the overall survival rate for patients continues to be double compared to historical survival rates after two or more prior lines of therapy (Press release, ImmunityBio, JAN 19, 2023, View Source [SID1234626388]). The results were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal (ASCO GI) conference in San Francisco January 19-21. See link to poster here.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The median OS in this highly advanced group of patients, up to seven lines (N=83) of treatment, was 5.8 months (95% CI: 4.9, 6.4 months), exceeding the approximately 2- to 3-month historical median OS. In the third-line setting (N=41), the median OS in this group was 6.3 months (95% CI: 5.0, 7.2 months), more than doubling the historical OS.

The baseline median CA 19-9 level (a marker of metastatic pancreatic disease) of the enrolled subjects (N=83) was very high at 4120 IU/ml, a significant increase from normal levels of 40 IU/ml. In subjects with CA 19-9 levels less than 4120 IU/ml (N=40), the median OS was 6.9 months (95% CI: 5.7,10.9).

"We are encouraged by the positive results in these patients with 3rd, 4th, 5th and even 7th line advanced pancreatic cancer and the considered and helpful feedback from the FDA," said Patrick Soon-Shiong, M.D., Executive Chairman and Global Chief Scientific and Medical Officer at ImmunityBio. "Treatments for pancreatic cancer in the advanced setting remain an unmet need and we are committed to confirming our hypothesis that orchestrating the innate and adaptive immune system will advance the care of these patients."

ImmunityBio also announced that it held two productive Type B meetings with the FDA in December. The first was to present the recent data and obtain guidance toward a registration pathway in metastatic pancreatic cancer with combination immunotherapy and NK cell therapy. The second meeting concerned the papillary cohort of the company’s BCG-unresponsive non-muscle-invasive bladder cancer study (QUILT 3.032; Cohort B). Cohorts A and C from this study were submitted in the BLA application for BCG-unresponsive NMIBC CIS, which has a May 2023 PDUFA date. The Agency advised the company to conduct randomized trials in localized BCG-unresponsive NMIBC papillary disease and in late-stage metastatic pancreatic cancer.

QUILT-88 Study Details

This Phase 2, randomized, three-cohort, open-label study will evaluate the comparative efficacy and overall safety of standard-of-care chemotherapy versus low-dose chemotherapy in combination with PD-L1 t-haNK, Anktiva (N-803), and aldoxorubicin in subjects with locally advanced or metastatic pancreatic cancer (NCT04390399). Each treatment setting, as well as each first- and second-line or later maintenance treatment, will be evaluated independently as Cohorts A, B, and C, respectively, with Cohorts A and B having independent experimental and control arms. The primary objective of Cohorts A and B is progression-free survival (PFS) per RECIST V1.1, and the objective of Cohort C is overall survival (OS). Secondary objectives include initial safety and additional efficacy measures, including overall response rate (ORR), complete response (CR) rate, durability of response (DoR), disease control rate (DCR), and overall survival (OS).

Trial sites include: Hoag Memorial Hospital Presbyterian in Orange County, Calif.; The Chan Soon-Shiong Institute for Medicine in Los Angeles County, Calif.; Astera Cancer Care in East Brunswick, NJ; and Avera McKennan Hospital and University Health Center in Sioux Falls, South Dakota, which serves patients in the tri-state area (Iowa, Nebraska and South Dakota).

Pancreatic cancer is the fourth leading cause of cancer-related death in the United States and has one of the highest mortality rates of all major cancers, taking nearly 50,000 lives in the U.S. every year. Today, surgery and subsequent adjuvant chemotherapy are the preferred treatment options for pancreatic cancer, but the five-year survival rate for late-stage cases is just 3%. For the majority of patients who present with more advanced disease, treatment typically consists of chemotherapy alone or supportive care for metastatic patients, and chemotherapy with or without radiation for those with locally advanced disease, leaving patients seeking new options.

On January 19, 2023 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM: HCM; HKEX:13) reported, following negotiations with the China National Healthcare Security Administration ("NHSA"), ORPATHYS (savolitinib) has been included in the updated National Reimbursement Drug List ("NRDL") for the treatment of locally advanced or metastatic non-small cell lung cancer ("NSCLC") adult patients with MET exon 14-skipping alterations who have progressed after or unable to tolerate platinum-based chemotherapy (Filing, 6-K, HUTCHMED, JAN 19, 2023, View Source [SID1234626387]). The updated NRDL will take effect from March 1, 2023.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Savolitinib, marketed in China under the brand name ORPATHYS, is an oral, potent and highly selective MET tyrosine kinase inhibitor ("TKI") jointly developed by AstraZeneca and HUTCHMED with HUTCHMED taking the lead in China, and commercialized by AstraZeneca worldwide.

Dr Weiguo Su, Chief Executive Officer and Chief Scientific Officer of HUTCHMED, said: "The NRDL has significantly broadened access to novel medicines for Chinese patients. We are gratified to see that our third novel oncology medicine, ORPATHYS, will be included in this year’s NRDL update. As the first and only selective MET inhibitor in the market, the inclusion of ORPATHYS will increase the affordability and access to this novel treatment."

Leon Wang, Executive Vice President, International and China President of AstraZeneca, said: "The inclusion of ORPATHYS on the NRDL is exciting news for NSCLC patients in China with MET exon 14 skipping alterations who will now have improved access to the only targeted medicine approved in this setting and who often do not respond well to chemotherapy. Since its launch in mid-2021, ORPATHYS has helped patients in need achieve better outcomes, and we are excited about the potential to reach even more patients in China with this transformational medicine."

ORPATHYS received conditional approval in China in June 2021 for the treatment of certain patients with NSCLC with MET exon 14 skipping alterations

On January 19, 2023 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company,reported that it is partnering with The Royal Marsden NHS Foundation Trust on Part C of its "Tracking mutations in cell free DNA to predict Relapse in eArly Colorectal Cancer" (TRACC) study, which will evaluate the use of circulating tumor DNA (ctDNA) to guide chemotherapy treatment decisions after curative-intent surgery in patients with early-stage colorectal cancer (CRC) (Press release, Guardant Health, JAN 19, 2023, View Source [SID1234626385]). The trial is intended to determine whether patients can be spared unnecessary chemotherapy and the associated side effects if they test negative for ctDNA using the Guardant Reveal blood test following surgery.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Currently, many patients with high-risk stage II and stage III CRC are routinely offered chemotherapy after surgery to help reduce the risk of relapse from microscopic minimal residual disease (MRD). However, approximately 50-80% of these patients are cured with surgery alone, and many may be over-treated because doctors have not been able to clearly advise patients on whether they are likely to benefit from the treatment.1,2,3 Chemotherapy can cause debilitating and sometimes life-long side-effects, such as damage to the nerves in the hands and feet, life-threatening infections and blood clots.

The study will use the Guardant Reveal blood test to detect MRD by measuring the DNA shed from tumor cells into the bloodstream. Global studies have shown that a negative ctDNA result after surgery is associated with a significantly lower likelihood of the cancer returning.4 The TRACC Part C trial aims to evaluate the use of ctDNA results from the Guardant Reveal test to guide chemotherapy treatment decisions in a multi-center, prospective, randomized study over the next four years.

"Patients with high-risk colorectal cancer are often over-treated and can suffer long-term neurotoxicity from chemotherapy," said Professor David Cunningham, director of clinical research at The Royal Marsden NHS Foundation Trust, chief investigator for the TRACC Part C study. "This study will generate evidence and insights to help us understand when we can avoid unnecessary chemotherapy for our patients who have no disease detected following surgery. This information can help us tailor treatment decisions to benefit patients and potentially realize significant cost savings for the healthcare system."

"With the Guardant Reveal test, a simple blood draw can be used to identify colorectal cancer patients who have residual disease and are most likely to benefit from adjuvant therapy," said Helmy Eltoukhy, Guardant Health co-CEO. "We’re pleased to partner with The Royal Marsden in the TRACC Part C study to enable the investigators to more accurately predict when cancer is unlikely to return, help guide chemotherapy treatment decisions and provide patients with a better quality of life."

TRACC Part C began opening sites in August 2022 and will involve approximately 40 sites across the United Kingdom with a planned recruitment of 1,621 patients over four years. The Royal Marsden NHS Foundation Trust announced that the first patient was enrolled in TRACC Part C in September 2022. The trial is funded by the Efficacy and Mechanism Evaluation (EME) Programme, a Medical Research Council (MRC) and National Institute of Health Research (NIHR) partnership.