On June 3, 2021 Protagonist Therapeutics ("Protagonist" or "the Company") (Nasdaq: PTGX), reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its lead investigational new drug candidate, rusfertide, for the treatment of patients with polycythemia vera (PV) for the reduction of erythrocytosis in those patients who do not require further treatment for thrombocytosis and/or leukocytosis (Press release, Protagonist, JUN 3, 2021, View Source [SID1234583611]). Breakthrough Therapy Designation requires that the drug candidate treat a serious or life-threatening disease or condition. It also requires preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The designation has the potential to expedite the development and regulatory review process.

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!

"We are thrilled to receive the Breakthrough Therapy Designation for rusfertide in PV, a serious disease where the need for different and better treatment options is clear and pressing," said Suneel Gupta, PhD, Chief Development Officer at Protagonist. "Rusfertide is a natural hormone mimetic and may stand out as the first non-cytoreductive therapeutic drug for PV. We look forward to working closely with FDA regulators to advance and complete all relevant clinical studies, both ongoing and planned, as quickly as possible."

The designation for rusfertide was supported in part by promising data from the ongoing Phase 2 clinical trial in patients with PV, presented at the Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in 2020. The data showed that when treated with rusfertide, a majority of patients were able to eliminate therapeutic phlebotomies, maintain a target hematocrit level of less than 45 percent, reverse iron deficiency, and experience symptom improvements. The FDA previously granted orphan drug status and Fast Track Designation to rusfertide in PV. Breakthrough Therapy Designation offers additional advantages over Fast Track Designation, including FDA actions to expedite both planned clinical trials and plans for expediting the manufacturing development strategy.

Updated data from the ongoing Phase 2 study has been selected for oral presentation at the upcoming annual meeting of the European Hematology Association (EHA) (Free EHA Whitepaper). This meeting will take place June 9 through 17 and will remain accessible until August 15, 2021.

About FDA Breakthrough Therapy Designation

Breakthrough Therapy Designation is an FDA program intended to expedite the development and regulatory review of investigational therapies that are designed to address serious or life-threatening conditions. The criteria for Breakthrough Therapy Designation requires preliminary clinical evidence that indicates that the candidate may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. This designation provides the Company with more intensive FDA guidance on an efficient drug development program, and eligibility for other actions to expedite the FDA review, such as a rolling review of a New Drug Application (NDA), where the FDA may review sections of the NDA before the complete application is submitted. An NDA for a product candidate receiving Breakthrough Therapy Designation may also be eligible for priority review if the relevant criteria are met. Breakthrough Therapy Designation does not change the standards for approval. For more information, please visit the FDA website at www.fda.gov.

On June 3, 2021 Histogen Inc. (NASDAQ: HSTO), a clinical-stage therapeutics company focused on developing potential first-in-class restorative therapeutics that ignite the body’s natural process to repair and maintain healthy biological function, reported an update on its pipeline focus following a strategic evaluation of its regenerative medicine platform technology development programs with the goal of focusing on high value orthopedic indications, creating pipeline synergies and maximizing resources in an effort to further drive long-term shareholder value (Press release, Conatus Pharmaceuticals, JUN 3, 2021, View Source [SID1234583445]).

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!

Development Program Updates

HST 001 – we completed our strategic evaluation of the HST 001 program taking into consideration the results from our Phase 1b/2a clinical trial of HST 001 as announced earlier this year, and as a result, we will suspend development of this program. While HST 001 has demonstrated a favorable safety and tolerability profile in androgenic alopecia in men, the development resources required to potentially achieve an acceptable efficacy threshold are substantial in terms of cost and time. Therefore, we believe the best business decision at this time, is to redirect these resources towards our high value orthopedic programs.
HST 003 – we are on track to initiate our Phase 1/2 clinical study of HST 003 in June 2021. The upcoming study is designed to evaluate the safety and efficacy of human extracellular matrix (hECM) implanted within microfracture interstices and the cartilage defect in the knee to regenerate hyaline cartilage in combination with a microfracture procedure. Patients will be enrolled at three sites: Oasis MD in San Diego, CA, The Steadman Clinic in Vail, CO, and Walter Reed Medical Center in Bethesda, MD.
HST 004 – We recently initiated an investigational new drug application (IND) enabling activities for HST 004, a CCM solution intended to be administered through an intradiscal injection for spinal disc repair. Our initial preclinical research has shown that HST 004 stimulates stem cells from the spinal disc to proliferate and secrete aggrecan and collagen II, regenerate normal matrix and cell tissue structure, and restore disc height. HST 004 was also shown to both reduce inflammation and protease activity and upregulate aggrecan production in an ex vivo spinal disc model. We anticipate filing an IND in the second half of 2022.

Emricasan – In May, we, along with our partner Amerimmune, completed enrollment of the Phase 1 study of emricasan for the treatment of mild-symptomatic COVID-19 patients. A total of 13 patients have been enrolled at our single site in New York City versus the initially targeted 40 patients. The decision to stop enrollment with a lesser number of patients was based solely upon the overall decline in COVID-19 cases in New York City and its negative impact on patient recruitment. To date, there have been no reports of serious adverse events, and we anticipate top-line safety, biomarker and patient reported outcomes data to be available in June 2021.
"Following the completion of our HST 001 Phase 1a/2b study in androgenic alopecia in men in the first quarter of this year, we embarked upon a strategic pipeline evaluation with the goal of determining the optimal value-creating opportunities for our regenerative medicine technology platform," said Richard W. Pascoe, President and Chief Executive Officer. "As a result of our evaluation, we have charted a new course for Histogen with a focus on orthopedic indications that we believe sit at the crossroads of pre-clinical and clinical proof of concept, significant commercial opportunity, and unmet medical needs. Moreover, we believe that by developing products that are therapeutically synergistic, we can be more efficient with our resources and create a strategic pipeline of novel therapeutics that has the potential to create long-term value for the benefit of our shareholders."

On June 3, 2021 VBL Therapeutics (Nasdaq: VBLT) reported a primary endpoint amendment in the OVAL Phase 3 registration-enabling study of VB-111 (Press release, VBL Therapeutics, JUN 3, 2021, View Source [SID1234583462]). The clinical trial amendment included a second, separate primary endpoint, of progression free survival (PFS), in addition to the original primary endpoint of the trial, overall survival (OS). Based upon the changes that were reviewed by the U.S. Food and Drug Administration (FDA), successfully meeting either primary endpoint is expected to be sufficient to support BLA submission. Successful meeting of the PFS endpoint, with a readout anticipated in 2022, could accelerate BLA submission by approximately one year compared to original projections based on the readout of the OS primary endpoint that remains anticipated in 2023. The OVAL study amendment, along with an update on the number of patients enrolled, which as of April 30, 2021, exceeded 260 patients, will be presented tomorrow as part of a virtual Clinical Trial in Progress poster presentation at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting.

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 addition of PFS as a second independent primary endpoint has several very important implications on the OVAL study," said Bradley Monk, M.D., FACS, FACOG, Arizona Oncology (U.S. Oncology Network), and Chair of the OVAL Study Steering Committee. "First, it de-risks the study, as it provides two options for study success. Second, it should accelerate the time to clinical readout and to potential approval, as PFS data are expected during 2022. Third, keeping OS as a primary endpoint preserves the opportunity of differentiating VB-111 from current ovarian cancer treatments, which were approved based on PFS data and have not as yet shown an OS benefit."

Title: Clinical Trial in Progress: Pivotal Study of VB-111 Combined with Paclitaxel vs. Paclitaxel for Treatment of Platinum-Resistant Ovarian Cancer (OVAL, VB-111-701/GOG-3018)
Authors: Arend, R.C., et al.
Session: Gynecologic Cancer
Session type: Poster Session
Abstract: 5599

About the OVAL study (NCT03398655)
OVAL is an international Phase 3 randomized pivotal registration enabling clinical trial that compares a combination of VB-111 and paclitaxel to placebo plus paclitaxel, in patients with platinum resistant ovarian cancer. The study is planned to enroll approximately 400 patients. OVAL is conducted in collaboration with the GOG Foundation, Inc., an independent international non-profit organization with the purpose of promoting excellence in the field of gynecologic malignancies.

About VB-111 (ofranergene obadenovec)
VB-111 is an investigational anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. VB-111 is a unique biologic agent that is designed to use a dual mechanism to target solid tumors. Its mechanism combines blockade of tumor vasculature with an anti-tumor immune response. VB-111 is administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in an "all comers" Phase 1 trial as well as in three tumor-specific Phase 2 studies. VB-111 has received an Orphan Designation for the treatment of ovarian cancer from the European Commission. VB-111 has also received orphan drug designation in both the US and Europe, and fast track designation in the US, for prolongation of survival in patients with recurrent glioblastoma. VB-111 demonstrated proof-of-concept and survival benefit in Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum-resistant ovarian cancer (NCT01711970)

On June 3, 2021 IASO Biotherapeutics (IASO Bio), a clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and autoimmune diseases, reported that its investigational new drug (IND) application for CT120, a fully human CD19/CD22 dual-targeting chimeric antigen receptor (CAR)-T cell therapy, has been accepted by the China National Medical Products Administration (NMPA) (acceptance number CXSL2101070) for treatment of relapsed/refractory B-acute lymphoblastic leukemia (B-ALL) (Press release, IASO BioMed, JUN 3, 2021, View Source [SID1234583478]). CT120 is the second clinical stage CAR-T therapies developed by IASO Bio, which signifies the company’s solid step in the development of next-generation CAR-T therapies.

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!

B-ALL is the most common of all Acute lymphoblastic leukemia (ALL). The emergence of CAR-T therapy and its application in ALL diseases in recent years have brought revolutionary changes to B-ALL treatment. However, the risk for poor prognosis and loss of lives remains high with a significant number of relapsed/refractory patients. Tumor recurrence and drug resistance are often attributed to the loss of target antigen expression. Therefore, next-generation CAR-T therapy such as dual targeting with multiple antigens is important to overcome the relapse challenge.

About CT120

CT120 is an autologous dual-antigen specific CAR-T therapy. Its extracellular domain contains two fully-human scFv sequences that can specifically recognize CD19 and CD22. Dual-antigen specific CAR-T cells have the potential to persistent in vivo longer than mono-specific CAR-T cells, and also enhance therapeutic effects by reducing relapse resulted from antigen escape.

CT120 proves to be significantly effective in an ongoing investigator-initiated trial (IIT) in China. The results show that CT120 not only has a durable response on CAR-T treatment-naive relapsed/refractory B-ALL patients, but also has a curative effect on relapsed patients who have previously received mono-specific CAR-T treatment. CT120 can reduce the risk of antigen escape and tumor relapse as a result of lower/loss of CD19 or CD22 expression following mono-specific CAR-T treatment, which will bring better therapeutic outcome and longer survival benefit for patients.

About B-ALL

Acute B-Lymphoblastic Leukemia (B-ALL) is a rapidly progressing cancer of the blood and bone marrow that occurs in both adults and children. About 75% of cases in adult patients occur in B cell lineage. Based on 2014-2018 cases and deaths, new cases of ALL were 1.8 per 100,000 men and women per year with the death rate at 0.4 per 100,000 men and women per year in the United States. In 2016 alone, there were 6,590 new ALL cases, and 1,400 deaths. Globally, ALL affected around 837,000 people and resulted in 110,000 deaths in 2015. The relapse rate for children afflicted by ALL is nearly 10% and for adults is as high as 50%. B-ALL is one of the most common forms of cancer in children between ages two and five and adults over age 50. (National Cancer Institute)

On June 3, 2021 US Oncology Research, The US Oncology Network (The Network), and OntadaTM reported that they contributed to more than 75 studies covering topics including gynecologic cancer, lung cancer and immunotherapy which will be presented at the upcoming 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to be held virtually from June 4 – 8 (Press release, US Oncology, JUN 3, 2021, View Source [SID1234583494]).

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!

"At this year’s ASCO (Free ASCO Whitepaper) Annual Meeting, we are looking forward to sharing our latest insights and knowledge across the global oncology community," said Robert L. Coleman, MD, chief scientific officer, US Oncology Research. "As we emerge from a year that challenged how we approach the fight against cancer, it is more important than ever to discuss how we can work together to ensure this next chapter of commitment to equitable care and innovation."

Key highlights include a plenary session exploring the role of adjuvant chemotherapy following chemoradiation in women with cervical cancer, featuring results from OUTBACK, a randomized Phase III trial to determine if the addition of adjuvant chemotherapy to standard cisplatin-based chemoradiation improves rates of overall survival. The plenary session, which will be held Sunday, June 6 from 1 – 4 p.m. ET, will feature abstract LBA3, "Adjuvant chemotherapy following chemoradiation as primary treatment for locally advanced cervical cancer compared to chemoradiation alone: The randomized phase III OUTBACK Trial."

"Despite the recently developed cervical cancer vaccine, many women will continue to die from this disease if existing treatments are not improved," said Bradley Monk, MD, FACOG, FACS, co-author of the study and oncologist with Arizona Oncology, a practice in The Network. "We are eager to present the results from this featured late-breaking plenary and to draw attention to the problem of cervical cancer among women in developing nations."

Also of note this year are findings from the MYLUNG ConsortiumTM study, which will be featured in an oral abstract session. Given the importance of molecular testing and targeted therapy for metastatic non-small cell lung cancer, the findings provide an assessment of real-world biomarker testing rates and turnaround times of more than 1,000 providers across the U.S. The session titled, "Lung Cancer—Non-Small Cell Metastatic," will be held Friday, June 4 from 1 – 4 p.m. ET and will feature abstract 9004, "Biomarker tissue journey among patients (pts) with untreated metastatic non-small cell lung cancer (mNSCLC) in The US Oncology Network community practices."

"While treatment decisions based on molecular analyses can improve outcomes, many patients do not undergo timely comprehensive testing," said Makenzi Evangelist, MD, study co-author, principal investigator for Protocol 2 of the MYLUNG Consortium and oncologist with New York Oncology Hematology (NYOH), a practice in The Network. "Through this real-world analysis, we observed that less than half of patients with mNSCLC had five or more biomarkers tested before initiating first-line systemic therapy. These findings deepen our understanding of the barriers patients face and help us target new approaches for improving their care."

"Through the MYLUNG Consortium, we are able to bring together providers, life sciences companies and patient advocacy groups with a common goal of improving patient outcomes," added Dr. Coleman, who is the MYLUNG Consortium program principal investigator. "What we are presenting at this year’s ASCO (Free ASCO Whitepaper) Annual Meeting are findings from Protocol 1. This innovative study will last five years with about 20 participating sites recruiting up to 12,000 patients."

In a poster session, results will be presented from a retrospective study leveraging electronic health record data from the Ontada database of adult patients with extensive-stage small cell lung cancer (ES-SCLC). The study investigated patient characteristics and treatment patterns for patients with ES-SCLC receiving treatment in the real-world community setting. The session titled, "Lung Cancer—Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers," will be held Sunday, June 6 from 8 – 11 a.m. ET and will feature abstract 8561, "Real-world evidence of cancer immunotherapy (CIT) combination treatment in first line (1L) extensive-stage small cell lung cancer (ES-SCLC)."

"This study was the first real-world study that analyzed the incorporation of immunotherapy in the treatment of small cell lung cancer," said Eric Nadler, MD, MPP, study co-author and oncologist with Texas Oncology, a practice in The Network. "Being able to leverage real-world data into today’s clinical research is a huge step forward. We now have the ability to take a plethora of already available and extremely valuable data and study thousands of patient journeys for numerous indications."

Ontada President Susan Shiff, PhD, MBA, concludes, "We are excited to present the results of studies that have used a variety of retrospective and prospective methodologies whose findings can help to improve the lives of cancer patients, which of course is our ultimate goal."

You can find the full schedule of affiliated data presentations from US Oncology Research, The US Oncology Network and Ontada at ASCO (Free ASCO Whitepaper) this year, including author details, here. For more information or to interview a trial investigator, contact Claire Crye at 281.825.9927 or [email protected], or Edie DeVine at 209.814.9564 or [email protected].