On August 8, 2022 Pyramid Biosciences, Inc., a Boston-based, clinical-stage biotechnology company focused on developing new and highly differentiated precision therapies for cancer and other serious diseases, reported the expansion of its senior leadership team (Press release, Pyramid Biosciences, AUG 8, 2022, View Source [SID1234617814]). Nushmia Khokhar, MD has joined the company as Chief Medical Officer, where she will oversee the advancement and expansion of the Company’s clinical pipeline.

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!

"Dr. Khokhar has a proven track record of successfully bringing novel oncology compounds through clinical development to global approval," said CEO of Pyramid Biosciences, Brian Lestini, MD, PhD. "We are looking forward to Dr. Khokhar’s leadership of our clinical programs during this important growth stage of our company."

Prior to joining Pyramid Biosciences, Dr. Khokhar served as Chief Medical Officer at Umoja Biopharma, a multi-platform, immuno-oncology company based in Seattle, WA. In this capacity, Dr. Khokhar oversaw Umoja’s clinical development, operations, and regulatory functions. Prior to joining Umoja, Dr. Khokhar was the Senior Vice President & Head of Clinical Development at Autolus Therapeutics, where she played a critical role in advancing the company’s autologous CAR T-cell products and clinical programs. Prior to that, Dr. Khokhar held numerous roles at Janssen Oncology, a subsidiary of Johnson & Johnson (NYSE: JNJ) where she led several successful clinical trials, directed the Phase 3 registration trial of trabectedin (YONDELIS) in soft tissue sarcomas, and served as the Global Clinical Leader for the breakthrough therapy daratumumab (DARZALEX) for hematologic cancers.

"I look forward to working with the outstanding team at Pyramid Biosciences, progressing the Company’s promising therapies through clinical development and building a robust pipeline," said Dr. Khokhar.

"We are extremely pleased to have Nushmia join our senior leadership team as we continue to grow our clinical programs and develop new medicines for underserved patients," said Jordan Leef, Pyramid Biosciences’ Co-Founder & COO.

Dr. Khokhar is a graduate of Aga Khan University and completed her post-doctoral fellowship at Memorial Sloan-Kettering Cancer Center, where she focused on pharmacology and molecular therapeutics. Following her post-doctoral fellowship, Dr. Khokhar completed a residency in internal medicine at Washington Hospital Center and a fellowship in Hematology/Oncology at the Northwestern University Hospital. Dr. Khokhar is board certified in medical oncology.

On August 8, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, reported that the company will host a conference call and live audio webcast on Monday, August 15, 2022, at 8:00 a.m. ET to review its second quarter 2022 financial results and provide an update on the company (Press release, Gamida Cell, AUG 8, 2022, View Source [SID1234617813]).

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!

To access the conference call, please register here and be advised to do so at least 10 minutes prior to joining the call. A live webcast of the conference call can be accessed in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. A replay of the webcast will be available approximately two hours after the event, for approximately 30 days.

On August 8, 2022 PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TPEx:6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, reported that the clinical rationale and protocol for the SURPASS-ET trial evaluating ropeginterferon alfa-2b-njft as a second-line treatment option for adults with high-risk essential thrombocythemia (ET) has been published in the journal, Future Oncology (Press release, PharmaEssentia, AUG 8, 2022, View Source [SID1234617812]).

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!

ET is a myeloproliferative neoplasm (MPNs), a group of rare blood cancers caused by genetic mutations that trigger the overproduction of blood cells; ET is characterized by the overproduction of platelets. Without proper treatment, these disorders carry a higher risk of thrombosis or other cardiovascular complications, or may progress toward myelofibrosis or secondary acute myeloid leukemia (sAML). Yet many patients who receive conventional approved treatments for ET experience resistance or intolerance, or the efficacy wanes over time, so new therapeutic options are needed to help address these limitations and improve treatment outcomes.

Ropeginterferon alfa-2b-njft is a unique monopegylated, long-acting interferon that was recently approved to treat adults with polycythemia vera (PV), another type of MPN. Given the well-established safety and efficacy profile demonstrated in prior studies, the treatment may represent a useful alternative to approved options for ET.

"Physicians treating people with MPNs have lacked effective, durable therapeutics that are designed specifically for these cancers. To help improve the long-term outlook for these patients, we need to focus not only on normalizing symptoms and improving quality of life, but also targeting driver mutations to more completely control the disease," said Srdan Verstovsek, M.D., Ph.D., Director of the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms, Department of Leukemia at the University of Texas MD Anderson Cancer Center. "This trial will provide critical data on how use of a monopegylated interferon for the first time in this disease state could reduce the risk of progression for these patients over time."

The global phase 3, randomized, open-label, multicenter SURPASS-ET (NCT04285086) trial is evaluating the safety, efficacy, tolerability, and pharmacokinetics of ropeginterferon alfa-2b-njft compared to anagrelide as second-line therapy in high-risk ET. Approximately 160 patients are being enrolled from 61 study sites across the United States, Canada and multiple regions across Asia, and will be randomized to receive treatment with ropeginterferon alfa-2b-njft (via subcutaneous injection every two weeks starting at 250 mcg, with a target optimal dose of 500 mcg by week four and onwards) or a daily anagrelide capsule (0.5 mg).

The primary efficacy endpoint is durable patient response, as defined by modified ELN response criteria (peripheral blood count remission; improvement or non-progression in disease-related signs; improvement or non-progression based on the MPN-SAF TSS; absence of hemorrhagic or thrombotic events; and durability at months 9 and 12). Other endpoints being evaluated include quality of life and change in allelic burden (the proportion of mutated cells in the blood). Data from the trial are expected by 2024.

"With nearly a decade of ongoing research with interferons in MPNs, we have strong evidence supporting the use of ropeginterferon alfa-2b-njft to treat individuals with ET," said Raymond Urbanski, M.D., Ph.D., U.S. Head of Clinical Development and Medical Affairs, PharmaEssentia. "The series of clinical trials currently underway are providing an increasingly clear picture of the profile of this treatment to potentially improve care standards and patient outcomes across the MPN category."

About Ropeginterferon alfa-2b

Ropeginteferon alfa-2b-njft is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, the product has a long duration of activity in the body.

Ropeginteferon alfa-2b-njft (marketed as BESREMi) has orphan drug designation for treatment of polycythemia vera (PV) in adults in the United States. The product was approved by the European Medicines Agency (EMA) in 2019, in the United States in 2021, and has recently received approval in Taiwan and South Korea. The product was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications.

BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders.

About Essential Thrombocythemia

Essential thrombocythemia (ET) is a myeloproliferative neoplasm (MPN) characterized by an overproduction of platelets in the blood that results from a genetic mutation; data indicates a JAK2 gene mutation is present in approximately half of diagnosed patients. ET is estimated to affect up to 57 per 100,000 people in the U.S. The disease is most commonly diagnosed through routine blood work, and is most common in people over the age of 50, with women 1.5 more times more likely to be diagnosed than men. As a chronic, progressive disease, ET requires regular monitoring and appropriate treatment. Over time, the disease may progress into more deadly conditions such as myelofibrosis or acute leukemia.1,2

On August 8, 2022 Daiichi Sankyo (TSE: 4568) reported that the first patient has been dosed in the global HERTHENA-Lung02 phase 3 trial evaluating the efficacy and safety of patritumab deruxtecan (HER3-DXd) versus platinum-based chemotherapy in patients with EGFR-mutated locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) with disease progression following treatment with one or more EGFR tyrosine kinase inhibitors (TKIs) including a third-generation EGFR TKI (Press release, Daiichi Sankyo, AUG 8, 2022, View Source [SID1234617811]).

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!

Patritumab deruxtecan is a specifically designed potential first-in-class HER3 directed antibody drug conjugate (ADC) discovered and being developed by Daiichi Sankyo.

Lung cancer is the second most common cancer and the leading cause of cancer-related deaths worldwide.1 Approximately 80% to 85% of lung cancer is classified as NSCLC with EGFR mutations occurring in up to 30% of tumors.2,3 While the efficacy and safety of targeted therapy with EGFR TKIs is well-established for the treatment of advanced EGFR-mutated NSCLC, the development of a broad range of resistance mechanisms is likely to lead to disease progression.4,5,6 After failure of one or more EGFR TKIs, platinum-based chemotherapy and subsequent salvage therapies have limited efficacy.7

"We are encouraged by the early results seen with patritumab deruxtecan and have initiated HERTHENA-Lung02 to further evaluate whether this HER3 directed antibody drug conjugate will be more effective than standard chemotherapy in treating patients with previously treated EGFR-mutated metastatic non-small cell lung cancer," said Gilles Gallant, BPharm, PhD, FOPQ, Senior Vice President, Global Head, Oncology Clinical Development, Oncology R&D, Daiichi Sankyo. "Initiating this phase 3 trial emphasizes our ongoing commitment to accelerate development of patritumab deruxtecan to potentially improve the standard of care for patients with this specific subtype of lung cancer."

About HERTHENA-Lung02

HERTHENA-Lung02 is a global, multicenter, open-label, phase 3 trial evaluating the efficacy and safety of patritumab deruxtecan (5.6 mg/kg) versus platinum-based chemotherapy (pemetrexed in combination with cisplatin or carboplatin) in patients with locally advanced or metastatic non-squamous NSCLC with an EGFR-activating mutation (exon 19 deletion or exon 21 L858R mutation) previously treated with an EGFR TKI with disease progression on or after treatment with a third-generation TKI. Patients will be randomized 1:1 to receive patritumab deruxtecan or platinum-based chemotherapy.

The primary endpoint of HERTHENA-Lung02 is progression-free survival (PFS) as assessed by blinded independent central review (BICR). Secondary endpoints include overall survival, investigator-assessed PFS as well as BICR and investigator-assessed objective response rate, clinical benefit rate, disease control rate, duration of response, time to response and safety. HERTHENA-Lung02 will enroll approximately 560 patients at multiple sites across Asia, Europe, North America and Oceania. For more information about the trial, visit ClinicalTrials.gov.

About Non-Small Cell Lung Cancer

Lung cancer is the second most common cancer and the leading cause of cancer-related deaths worldwide.1 Approximately 80% to 85% of lung cancer is classified as NSCLC with EGFR mutations occurring in up to 30% of tumors.2,3 NSCLC is diagnosed at an advanced stage in more than 50% of patients and often has a poor prognosis with worsening outcomes after each line of subsequent therapy.8,9,10

The introduction of targeted therapies and checkpoint inhibitors in the past decade has improved the treatment landscape for patients with advanced or metastatic NSCLC, and for patients with advanced EGFR-mutated NSCLC, targeted therapy with EGFR TKIs offer higher response rates and PFS compared to chemotherapy.11 However, most patients eventually develop resistance to these therapies and subsequent therapies after EGFR TKI with platinum-based chemotherapy have limited efficacy with PFS of approximately 6.4 months.7,12

HER3 is a member of the EGFR family of receptor tyrosine kinases, which are associated with aberrant cell proliferation and survival.13 It is estimated that about 83% of all NSCLC tumors express the HER3 protein. Overexpression is associated with metastatic progression and decreased relapse-free survival.14

New treatment approaches are needed to overcome resistance and improve survival in EGFR-mutated NSCLC. Currently, there is no HER3 directed medicine approved for the treatment of any cancer.

About Patritumab Deruxtecan

Patritumab deruxtecan (HER3-DXd) is one of three lead DXd ADCs in the oncology pipeline of Daiichi Sankyo. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, patritumab deruxtecan is composed of a fully human anti-HER3 IgG1 monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

Patritumab deruxtecan is currently being evaluated as both a monotherapy and in combination with other anticancer therapies. In addition to the HERTHENA-Lung02 trial, patritumab deruxtecan is being evaluated in HERTHENA-Lung01, a pivotal phase 2 trial in patients with EGFR-mutated locally advanced or metastatic NSCLC previously treated with a EGFR TKI and platinum-based chemotherapy; a phase 1 trial in combination with osimertinib in EGFR-mutated locally advanced or metastatic NSCLC; and a phase 1 trial in previously treated patients with unresectable or metastatic NSCLC. A phase 1/2 trial in HER3 expressing metastatic breast cancer also has been recently completed.

In December 2021, patritumab deruxtecan was granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of patients with metastatic or locally advanced EGFR-mutated NSCLC with disease progression on or after treatment with a third-generation TKI and platinum-based therapies.

Patritumab deruxtecan is an investigational medicine that has not been approved for any indication in any country. Safety and efficacy have not been established.

On August 8, 2022 POINT Biopharma Global Inc. (NASDAQ: PNT) (the "Company" or "POINT"), a company accelerating the discovery, development and global access to life-changing radiopharmaceuticals, reported that next week, on Thursday, August 18, 2022 at 12:00 PM ET, the Company will be presenting a 45-minute educational webinar entitled "Understanding the PNT2002 Phase 3 SPLASH Trial Control Arm" (Press release, Point Biopharma, AUG 8, 2022, View Source [SID1234617810]).

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!

Topics to be presented at the webinar include:

Overview of SPLASH trial design (including lead-in phase) and rationale for hormone switches
What is a control arm benchmark, why PROfound1 & IMbassador2502 were selected as SPLASH benchmarks
Review current treatment patterns, including sequencing considerations, in real-world clinical settings for mCRPC patients
The webinar will feature presentations from two Thought Leaders: Dr. Oliver Sartor & Dr. Kim Chi. They will be joined by members of the Company’s executive leadership team including Dr. Neil Fleshner, Chief Medical Officer, and Dr. Sherin Al-Safadi, VP Medical Affairs.

About Dr. Oliver Sartor
Dr. Oliver Sartor is an internationally recognized expert in prostate cancer. His medical practice and research have focused on prostate cancer since 1990 when he finished a medical oncology fellowship at the National Cancer Institute (NCI). He has published over 400 peer-reviewed articles, led or co-led multiple national and international clinical studies, including three radiopharmaceutical phase III studies pivotal for FDA approval in prostate cancer. He has lectured widely, and at last count has given invited lectures in 33 countries. He is currently the Associate Dean for Oncology, Medical Director of the Tulane Cancer Center, and serves as the Laborde Professor for Cancer Research at Tulane Medical School with appointments in both the Medicine and Urology Departments. He is the Medical Oncology Chair of the GU committee of NRG, a national cancer research group. He is also a past member of the National Cancer Institute Board of Scientific Counselors (Clinical Sciences and Epidemiology).

About Dr. Kim Chi
Dr. Kim Chi, MD is the Vice President and Chief Medical Officer of BC Cancer. His research is focused in the area of genitourinary cancers with a special interest in prostate cancer and investigational new drugs. This includes phase I, II and III clinical trials, therapeutic use of antisense oligonucleotides and mechanisms of treatment resistance. Nationally and internationally he has led a number of multi-center Phase I, II and III clinical trials, and has also contributed to clinical trials that established new standard of care for patients with advanced prostate cancer (docetaxel, abiraterone acetate, enzalutamide).