On May 26, 2022 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported that it will present a Trials in Progress poster related to its ADVANCED-1 Phase 1 trial at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held in Chicago, Illinois and virtually from June 3 through June 7, 2022 (Press release, Protara Therapeutics, MAY 26, 2022, View Source [SID1234615097]). The ADVANCED-1 study is evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of non-muscle invasive bladder cancer (NMIBC).

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"There is a significant need for new treatment options for patients with NMIBC, one of the most recurrent and difficult to treat cancers," said Jathin Bandari, M.D., Chief Medical Officer of Protara Therapeutics. "Based on its mechanism of action, and promising clinical data from its predecessor therapeutic OK-432, we believe that TARA-002 may address this pressing area of high unmet need. We look forward to continuing to advance this trial and exploring TARA-002’s full potential in NMIBC."

Details of the poster presentation are as follows:

Title: A Phase 1a/b safety study of intravesical instillation of TARA-002 in adults with high-grade non-muscle invasive bladder cancer (ADVANCED-1)
Abstract Number: TPS4620
Session Title: Genitourinary Cancer—Kidney and Bladder
Session Date and Time: Saturday, June 4, 2022, from 2:15 PM – 5:15 PM EDT
Location: In-Person & Online | McCormick Place, Hall A

ADVANCED-1 is a Phase 1 dose-finding, open-label trial (NCT05085977 and NCT05085990) evaluating TARA-002 in treatment-naïve and treatment-experienced NMIBC patients with high-grade carcinoma in situ (CIS) and high-grade papillary tumors (Ta). In the initial dose escalation phase of the trial, patients will receive six weekly intravesical doses of TARA-002. The primary objective of the trial is to evaluate the safety, tolerability and preliminary signs of anti-tumor activity of TARA-002, with the goal of establishing a recommended dose for a planned Phase 2 clinical trial.

A copy of the abstract is available at View Source

About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and LMs for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and Taiwan by Chugai Pharmaceutical Co., Ltd.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins (IL)-2, IL-6, IL-8, IL-10, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, granulocyte colony-stimulating factor, and granulocyte-macrophage colony-stimulating factor, are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.

About Non-Muscle Invasive Bladder Cancer

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

On May 26, 2022 FibroGen, Inc. (NASDAQ: FGEN) reported that Enrique Conterno, Chief Executive Officer, will participate in a fireside chat at the Jefferies Healthcare Conference on Thursday, June 9 at 1:30pm EDT (Press release, FibroGen, MAY 26, 2022, View Source [SID1234615113]).

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A live audio webcast of the event will be available on the "Events & Presentations" section of the FibroGen Investors webpage at www.fibrogen.com. A replay will be available for approximately 30 days.

On May 26, 2022 Flatiron Health reported three Flatiron-authored abstracts have been accepted for poster discussion and presentations at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to be held June 3-7 (Press release, Flatiron Health, MAY 26, 2022, View Source [SID1234615129]).

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"As demonstrated by our research at ASCO (Free ASCO Whitepaper) this year, we are at the forefront of our industry to improve patient lives through the power of integrated evidence, and are committed to advance equitable care for all patients," said Carolyn Starrett, Flatiron CEO. "At Flatiron, we are reimagining the infrastructure of cancer care. We bring together science, technology, and medicine to transform possibilities into results and solve critical challenges with our partners."

Highlights include:

an investigation that uses one of the largest real-world datasets with a telemedicine variable available to assess patterns of use among patients with cancer during the COVID pandemic, illuminating health inequity
a study that uses integrated evidence to quantify the clinical value of multi-gene testing in early-stage lung adenocarcinoma, showcasing potential benefit in treatment decision-making
a study that evaluates and understands the value of ctDNA as a non-invasive tool to treat patients with advanced NSCLC by leveraging the Flatiron Health-Foundation Medicine Clinico-Genomic Database
Read more about the research on the Evidence Desk.

Poster Discussions and Presentations

Racial and Socioeconomic Disparities in Telemedicine Use Among US Patients Initiating Cancer Treatment During the COVID-19 Pandemic
First author: Jenny Guadamuz, Xiaoliang Wang, Trevor J. Royce, Gregory S. Calip
Abstract: 6511

A real-world evidence study quantifying the clinical value of multi-gene testing in early-stage lung adenocarcinoma (LUAD)
Partner: Foundation Medicine
First author: Nathan Pennell, Lianliang Zhang, Katherine T. Lofgren, Bharathi Muthusamy, Emily Castellanos, Karen Schwed, Oliver Humblet, Alexa B. Schrock, Geoffrey R. Oxnard
Abstract: 8525

ctDNA Shed and Outcomes for Patients (pts) with Advanced Non-small Cell Lung Cancer (aNSCLC) Treated with Immune Checkpoint Inhibitors (ICPI)
Partner: Foundation Medicine
First author: Benjamin Besse, Russell W. Madison, Cheryl Cho-Phan, Jermey Snider, Tamara Snow, Filippo G Dall’Olio, Khaled Tolba, Alexa B. Schrock, Geoffrey R. Oxnard
Abstract: 9045

On May 26, 2022 Protagonist Therapeutics (Nasdaq: PTGX) reported new data from its ongoing Phase 2 REVIVE study evaluating rusfertide in patients with polycythemia vera (PV) (Press release, Protagonist, MAY 26, 2022, View Source [SID1234615145]). These results will be shared as an oral presentation at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, being held in Chicago from June 3-7, 2022.

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"We are pleased to observe that administration of rusfertide continues to provide PV patients with an effective therapy that leads to rapid and sustained hematocrit control, and potentially offers patients a better quality of life by keeping them essentially phlebotomy-free for up to 18 months," said Ronald Hoffman, M.D., Director of the Myeloproliferative Disorders Research Program at the Icahn School of Medicine at Mount Sinai and principal investigator of the REVIVE study. "Importantly, the new results show that rusfertide administration suspension, due to the brief clinical hold, directly led to increases in hematocrit levels, red blood cell counts, and phlebotomy rates. In contrast, resumption of rusfertide quickly restored the therapeutic benefits for patients, confirming the direct and rapid effect of rusfertide and its potential utility in treating this serious disease."

"These highly promising new results continue to demonstrate the rapid therapeutic effect of rusfertide and its utility as an effective potential treatment across all categories of PV patients, independent of patient risk category, or concurrent therapy with other cytoreductive treatments including hydroxyurea, interferons or JAK inhibitors," said Dinesh V. Patel, Ph.D., President and Chief Executive Officer of Protagonist. "Taken together, these data reaffirm our belief in the potential of rusfertide to provide a highly effective treatment option for patients with PV, providing an opportunity to fundamentally transform the management of this disease. Rusfertide continues to be the primary focus of our corporate resources and efforts, and we continue to explore the full therapeutic potential of rusfertide with a sharp focus on the execution of the recently initiated Phase 3 VERIFY study."

Summary of Key Results

Updated Results from Phase 2 Studies Evaluating Rusfertide in Patients with PV

REVIVE Study

The ongoing Phase 2 REVIVE study was designed to evaluate rusfertide in patients with phlebotomy-dependent PV for up to 18 months. Results from the 70 phlebotomy-dependent PV patients continued to demonstrate that rusfertide treatment essentially eliminated the need for therapeutic phlebotomy (TP), and led to rapid, sustained, and durable control of hematocrit (HCT) levels below 45% without a clinically meaningful increase in white blood cell numbers of PV-related thromboses. Rusfertide treatment also led to normalization of iron stores and improved symptoms including concentration.

Furthermore, the new data showed that treatment suspension in PV patients led to increases in hematocrit levels, RBC count, and phlebotomy rates. In contrast, resumption of rusfertide treatment in those patients led to significant improvement in those parameters, providing further evidence of the rapid and beneficial therapeutic effect of rusfertide in PV. Upon the lifting of the clinical hold placed on rusfertide in PV, about 85% of patients resumed treatment with rusfertide.

PACIFIC Study

The ongoing Phase 2 PACIFIC study enrolled 20 patients with confirmed high HCT levels above 48% to evaluate rusfertide as an induction therapy. Results demonstrated that all erythrocytotic PV patients on rusfertide induction therapy with twice weekly dosing achieved rapid, sustained and durable HCT control below 45%, and without the need for TP.

Details for the ASCO (Free ASCO Whitepaper) 2022 oral presentation are as follows:

Title: Rusfertide (PTG-300) treatment in phlebotomy-dependent polycythemia vera patients.
Authors: Ronald Hoffman, M.D., The Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, Protagonist Therapeutics
Abstract Number: #7003
Session: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant
Presentation Date and Time: June 7, 2022 at 10:45 a.m. CT

About Rusfertide

Rusfertide (PTG-300) is an investigational, injectable hepcidin mimetic that is currently being developed for various disorders associated with iron overload and/or excessive erythrocytosis (red blood cell production). Rusfertide regulates iron homeostasis and controls the absorption, storage, and distribution of iron in the body. Discovered through Protagonist’s peptide technology platform, rusfertide is currently being investigated in the REVIVE Phase 2 proof-of-concept clinical trial for polycythemia vera (PV), a rare chronic blood disorder that affects about 160,000 patients in the U.S., the PACIFIC Phase 2 study in PV subjects with high hematocrit levels, and a recently completed Phase 2a study for hereditary hemochromatosis. The VERIFY Phase 3 study is currently underway.

On May 26, 2022 PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of molecularly targeted cancer immunotherapies and infectious disease vaccines, reported an upcoming poster presentation of preliminary data from its ongoing Phase 2 VERSATILE-002 clinical trial at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place June 3-7, 2022 in Chicago and online (Press release, PDS Biotechnology, MAY 26, 2022, View Source [SID1234615162]).

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VERSATILE-002 is a single-arm Phase 2 study evaluating the safety and efficacy of PDS0101, an HPV16-targeted investigational T cell-activating immunotherapy that leverages PDS Biotech’s proprietary Versamune technology, in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab). The combination is being evaluated in checkpoint inhibitor (CPI) -naïve and CPI-refractory patients with recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC). The data being presented at ASCO (Free ASCO Whitepaper) will detail preliminary safety and efficacy data for CPI-naïve patients at a prespecified interim analysis point.

In the VERSATILE-002 clinical trial, patients are being treated with KEYTRUDA 200 mg by IV infusion every three weeks, plus subcutaneous injection of PDS0101 for the first 4 treatment cycles (Cycles 1-4) and again on Cycle 12; KEYTRUDA treatment continues for up to 35 cycles, or until disease progression or demonstrated intolerance to therapy.

Highlights of the abstract from 19 patients (safety) with available imaging data for 17 of the 19 (efficacy) in the VERSATILE-002 clinical trial include:

Response rates thus far** (tumor shrinkage greater than 30%) seen in 7/17 (41.2%) patients in comparison to the published results of approximately 19% for approved checkpoint inhibitors used as monotherapy for recurrent or metastatic head and neck cancer, with 2 of the 7 having complete responses (CR)*
Stable disease (SD) was reported in 6/17 (35.3%) patients, with 4 of the 6 (67%) experiencing tumor shrinkage of less than 30%
Clinical efficacy (ORR** + SD) was seen in 13/17 (76.5%) patients
Progressive/ongoing disease was reported in 4/17 (23.5%) patients
Patients had received a median of 4/5 doses of PDS0101 (range 1-5) and 9/35 doses of KEYTRUDA (range 1-18)
There were no treatment-related adverse events greater than or equal to Grade 3 (N=19)
No patients required dose interruption or reduction on the combination treatment
No patients discontinued the combination treatment
At 9 months of follow up (median not yet achieved):
Progression free survival (PFS) rate was 55.2%
Overall survival (OS) rate was 87.2%
"While preliminary, we are excited to see the enhanced clinical responses and tolerability of PDS0101 in combination with KEYTRUDA in the CPI-naive arm of the VERSATILE-002 trial," said Dr. Lauren V. Wood, Chief Medical Officer of PDS Biotech. "We believe PDS0101’s ability to generate tumor-attacking killer T-cells without increasing toxicity based on these preliminary results speaks to the specificity of the Versamune-based immunotherapies and the potential of these drug candidates to work in combination with a broad range of anti-cancer therapies. These data continue to strengthen our confidence that PDS0101 in combination with KEYTRUDA could potentially improve patient outcomes."

"The clinical activity seen with PDS0101 in combination with KEYTRUDA thus far, in addition to the favorable safety profile continues to show promise in this difficult-to-treat patient population," stated Dr. Jared Weiss, MD, Lead Principal Investigator at the University of North Carolina at Chapel Hill School of Medicine and Lineberger Comprehensive Cancer Center. "While the patient pool is small, the responses we are seeing in these patients based on the preliminary results are significant, leading to shrinking of tumors, and extending overall survival."

The abstract for this poster is now available online on the ASCO (Free ASCO Whitepaper) conference website: View Source; data in this press release have been updated since the abstract submission.

Abstract/Poster Number: 6041
Poster Title: PDS0101 a novel type 1 interferon and CD8+ T-cell activating immunotherapy in combination with pembrolizumab in subjects with recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC).
Presenting Author: Jared Weiss, M.D., Section Chief of Thoracic and Head and Neck Oncology at the University of North Carolina at Chapel Hill School of Medicine and Lineberger Comprehensive Cancer Center, who serves as the Lead Principal Investigator for VERSATILE-002
Session Title: Head and Neck Cancer
Date: Monday, June 6, 2022
Time: 1:15 PM-4:15 PM CDT

PDS Biotech is presenting a second abstract #2518 which presents clinical results for a PDS0101-based novel triple combination. The press release describing these data can be seen on the company website here.

The company is hosting a conference call on Tuesday, June 7 at 8:00 AM EDT to discuss the data from the two trials presented at ASCO (Free ASCO Whitepaper). To participate on the live call, please dial 877-407-3088 (US) or +1 201-389-0927 (International) and provide the conference ID "13729901" five to ten minutes before the start of the call. A live webcast of the event will be available online in the investor relations section of the company’s website at View Source

KEYTRUDA is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

*No control or comparative studies have been conducted between checkpoint inhibitors and PDS0101; Ferris R.L., Nivolumab for Recurrent Squamous-Cell Carcinoma of the Head and Neck; N Engl J Med 2016; 375:1856-1867; Burtness B et al., Pembrplizumab alone or with chemotherapy versus cetuximab with chemotherapy for recurrent or metastatic squamous cell carcinoma of the head and neck (keynote 048): a randomized, open label phase 3 study; Lancet 2019;394(10212):1915-1928
View Source
View Source
**The study is on-going and includes confirmed and unconfirmed responses.