On May 13, 2021 Seattle Cancer Care Alliance (SCCA), the only National Comprehensive Cancer Network designated cancer center in Washington State, reported that it is an authorized treatment center for the new B-cell maturation antigen (BCMA) targeted chimeric antigen receptor (CAR) T-cell therapy, idecabtagene vicleucel, also known as ide-ce (Press release, Seattle Cancer Care Alliance, MAY 13, 2021, View Source [SID1234579962]).

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!

Ide-cel was approved by the U.S. Food and Drug Administration (FDA) on March 26, 2021, and is indicated for the treatment of adult patients with relapsed refractory multiple myeloma after four or more prior lines of therapy including a proteasome inhibitor, an immunomodulatory therapy and an anti-CD38 antibody. It is the first cell-based gene therapy approved by the FDA for the treatment of multiple myeloma and is being marketed under the brand name Abecma.

"We are pleased to offer this new advanced therapy to patients who are suffering from relapsed or refractory multiple myeloma," said Nancy Davidson, MD, president and executive director of Seattle Cancer Care Alliance. "We are committed to delivering personalized care to our patients and improving patient outcomes and excited to be among the first cancer centers in the nation to offer this treatment to adult patients with multiple myeloma."

Multiple myeloma is a cancer of plasma cells in which abnormal plasma cells build up in bone marrow and limit the body’s ability to make enough healthy blood cells, thus resulting in low blood counts. Multiple myeloma is also associated with bone and kidney damage as well as a weakened immune system. There are over 140,000 people in the United States living with this cancer and according the American Cancer Society approximately 34,920 new cases will be diagnosed in 2021, and 12,410 deaths among those with multiple myeloma will occur.

Ide-cel is a one-time therapy that is created from a patient’s own white blood cells, which have been modified to recognize and attack myeloma cells. As an anti-BCMA CAR T-cell therapy, ide-cel recognizes and binds to BCMA, a protein that is nearly universally expressed on cancer cells in multiple myeloma, leading to the death of BCMA-expressing cells.

In the clinical study that supported its approval, ide-cel was shown to be safe and effective. Approximately 72% of patients partially or completely responded to the treatment with 28% of patients showing complete response. An estimated 65% of this group remained in complete response to ide-cel for at least 12 months.

"The FDA approval of this novel therapy is a significant milestone in the advancement of new, innovative therapies for multiple myeloma," said David Maloney, MD, PhD, medical director for cellular immunotherapy at the Bezos Family Immunotherapy Clinic at Seattle Cancer Care Alliance. "We are excited about the continued expansion of CAR T-cell treatment options available to our patients, and the potential ide-cel offers to extend the lives of those who have multiple myeloma."

"Our clinical trials at the SCCA have provided us with extensive experience using BCMA CAR T-cells for multiple myeloma. The new FDA approval allows our to leverage this knowledge and safely bring a promising therapy to a wider population of adult patients with multiple myeloma," said Damian Green, MD, Seattle Cancer Care Alliance and Associate Professor, and who leads translational myeloma research programs at Seattle Cancer Care Alliance and the Fred Hutchinson Cancer Research Center.

SCCA is home to several of the world’s leading immunotherapy experts whose research has contributed to the foundation of many immunotherapies currently used to treat cancer. SCCA’s Bezos Family Immunotherapy Clinic, which opened in 2016, is a state-of-the-art center dedicated to offering the newest cellular immunotherapy clinical trials and FDA approved treatments.

On May 13, 2021 Osmol Therapeutics reported that it has initiated Investigational New Drug (IND) enabling studies to develop a therapy to prevent chemotherapy-induced peripheral neuropathy (CIPN) (Press release, Osmol Therapeutics, MAY 13, 2021, View Source [SID1234579961]). A phase 1 clinical study is projected to begin in 2022 . There are currently no Food and Drug Administration (FDA) approved therapies for the prevention or treatment of CIPN, a debilitating condition resulting from the off-target toxicity of many chemotherapy treatments.

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!

Osmol was founded by Dr. Barbara Ehrlich, Professor of Pharmacology and of Cellular and Molecular Physiology, Yale School of Medicine. Her research on neuronal calcium sensor-1 (NCS1), a critical calcium binding protein that regulates intracellular calcium levels, forms the basis of Osmol’s CIPN treatment, OSM-0205 and future potential NCS1 therapeutics. OSM-0205’s mechanism addresses the off-target toxicity of microtubule-based chemotherapy agents that results in a calcium surge leading to CIPN. OSM-0205 modulates NCS1 to prevent the calcium surge and maintain neuronal integrity.

The company’s initial focus is CIPN in breast cancer patients resulting from taxane-based chemotherapy treatment. Taxanes are the most widely used chemotherapy treatment for breast cancer and can lead to CIPN in up to 80% of patients. Independent market research conducted with breast cancer oncologists at leading US cancer centers confirmed that CIPN is the most significant toxicity issue facing clinicians and patients when treating breast cancer.

"Chemotherapy-induced peripheral neuropathy is a devastating adverse event that can leave patients and their treating physicians with a very difficult choice – reduce taxane therapy with the possibility of negatively impacting patient outcomes or continue therapy at the recommended dose with the potential of causing increased and possibly permanent, disabling CIPN," said Dr. Ehrlich. "OSM-0205 is being developed with the goal of preventing CIPN before it occurs. By blocking the calcium surge that causes neuropathy, optimal treatment with taxanes can continue. This is particularly important in the treatment of breast cancer where taxanes remain the foundation of most therapeutic regimens."

"There are currently no FDA-approved therapies to avoid or reduce CIPN, creating an urgent need for patients being treated with chemotherapy," said Robert Berman, M.D., Executive Chairman of Osmol Therapeutics and co-founder and former Chief Medical Officer at Biohaven Pharmaceuticals. "Over 225,000 patients in the U.S. and the European Union with early stage or metastatic breast cancer are treated with taxanes each year. We have recruited an experienced and capable executive team, led by Bob Linke, Osmol’s Chief Executive Officer, to advance OSM-0205 to address this need as well as explore the potential use of neuronal calcium sensor-1 (NCS1) in other indications. We are excited by the potential of OSM-0205 and expect to begin clinical development as early as next year."

Bob Linke, MBA, is an experienced biopharma entrepreneur, who is also Executive Chairman of Embera NeuroTherapeutics and IonSense. He has an established track record developing strategies, building and leading emerging companies through all phases of growth – from research, product development and clinical studies to successful commercialization, partnership and acquisitions. Bob brings an open management style to create cohesive, high-functioning teams. He has raised over $70 million in private equity and non-dilutive financing to fund these companies’ development and commercialization efforts. His early career was spent at Baxter, developing and commercializing pharmaceuticals and drug delivery systems.

About OSM-0205 and CIPN

Osmol’s lead drug, OSM-0205, is based on Dr. Barbara Ehrlich’s research in neuronal calcium sensor-1 (NCS1) at Yale University and is designed to prevent the off-target calcium surge caused by taxanes and potentially other chemotherapy treatments associated with peripheral nerve damage. Data from preclinical studies conducted by Osmol show that pre-treatment with OSM-0205 prevents neuronal damage from taxanes in mice by preventing the off-target intracellular calcium surge caused by these chemotherapy agents. It is hypothesized that OSM-0205 modulates NCS1 in patients to protect neurons from damage leading to a reduction of CIPN. CIPN affects hundreds of thousands of cancer patients every year and can compromise optimum chemotherapy dosing. There are no effective treatments for CIPN, a condition which can diminish quality of life and lead to lifelong disability.

On May 13, 2021 Genetron Holdings Limited ("Genetron Health" or the "Company", NASDAQ:GTH), a leading precision oncology platform company in China that specializes in offering molecular profiling tests, early cancer screening products and companion diagnostics development, reported a strategic partnership with Siemens Healthineers at the China Medical Equipment Fair (Press release, Genetron Health Technologies, MAY 13, 2021, View Source [SID1234579960]).

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!

This partnership aims to promote large scale application of Genetron’s S5 platform and lung cancer 8-gene IVD assay in Chinese hospitals, in order to provide non-small cell lung cancer (NSCLC) patients with efficient and accurate personalized diagnosis and treatment guidance. The companies also plan to work together to standardize cancer molecular testing in hospitals.

Representing Genetron Health at the signing ceremony in Shanghai were Co-founder and CEO Sizhen Wang, and Vice President Zhaohui Yin. Elisabeth Staudinger, President of Siemens Healthineers Asia Pacific, and Guo Yiming, Vice President of Siemens Healthineers Laboratory Diagnostic System, Greater China Region, also attended the event.

"Genetron Health focuses on the innovation, clinical transformation and commercialization of cancer genomics technologies. Both our lab developed test (LDT) and in vitro diagnostic (IVD) products’ business models have been progressing well," said Sizhen Wang, Co-founder and CEO of Genetron Health. "We are pleased to collaborate with Siemens Healthineers for our S5 instrument and lung 8 IVD assay, which helps diagnose and treat cancer patients. We also expect this partnership to help standardize cancer molecular testing in China, and reach more patients in need," Wang said.

"As a global leader in healthcare technology, Siemens Healthineers stays committed to addressing the needs of patients. To help Chinese cancer patients access quality care, we are not only introducing cutting-edge testing analyzers and reagents to China, but are also working with local innovators to offer personalized diagnostics. We are excited to forge a strategic partnership with Genetron Health, an industry leading precision oncology platform company. Meanwhile, we expect to push forward precision care in the field of lung cancer, therefore contributing to the vision of a Healthy China," said Guo Yiming, Vice President of Laboratory Diagnostics, Siemens Healthineers Greater China.

At present, lung cancer is one of the most serious diseases in China, placing the highest amongst morbidity and mortality rankings of all cancer types. In China, 787,000 cases of lung cancer are diagnosed every year, with about 631,000 deaths [1] caused annually as well. NSCLC is the most common type of lung cancer. In recent years, with the development of more advanced diagnostics and the emergence of targeted drugs, more NSCLC patients have been able to receive quicker and more accurate treatment. As the "first step" of targeted therapy for lung cancer, cancer molecular gene detection is the key to guided clinical treatment and drug applications. The U.S. National Comprehensive Cancer Network (NCCN) Guidelines for NSCLC also recommend that molecular testing of lung cancer-related genes be used to guide targeted therapies for NSCLC patients. Accurate lung cancer gene detection for NSCLC patients can help clinicians opt for more precise treatment.

Based on Genetron Health’s One-Step Seq technology (Chinese invention patent ZL 201710218529.4), the lung cancer 8-gene IVD assay can detect many mutations and fusions across 8 different genes at once, for targeted therapy selection in NSCLC. The assay can offer patients rapid and accurate molecular typing, medication guidance and drug resistance monitoring. Combined with Genetron S5, it offers advantages in detection efficiency, ease of use, economic cost, sample size, and can achieve a two-day turnaround time. It is suitable for independent clinical molecular testing in Chinese hospitals at all levels.

[1] Zheng R., Sun K., Zhang S., et al. Report of cancer epidemiology in China, 2015, Chinese Journal of Oncology, 2019, 41(1):19-28.

On May 13, 2021 Compass Therapeutics, Inc. ("Compass"; OTCQB: CMPX), a clinical-stage biotechnology company developing proprietary antibody therapeutics to treat cancer, and TRIGR Therapeutics, Inc. ("TRIGR"), a private biotechnology oncology company, reported that the companies have entered into a definitive merger agreement, under which Compass, through a subsidiary, will acquire TRIGR, a private cancer drug development company founded by George Uy, an industry veteran and a former commercial executive at Roche, in a stock-for-stock transaction (Press release, Compass Therapeutics, MAY 13, 2021, View Source [SID1234579959]). Under the terms of the agreement, Compass’ subsidiary will acquire all of the outstanding shares of TRIGR. In addition, Miranda Toledano, TRIGR’s Chief Financial Officer and Chief Operating Officer, will be joining the Compass Board of Directors. The transaction was unanimously approved by the Boards of Directors of both companies.

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 the core of this transaction is a differentiated bispecific antibody targeting both DLL4 and VEGF-A, which inhibits both DLL4-mediated Notch signaling and VEGF signaling, which has been renamed CTX-009 (formerly designated TR009/ABL001/NOV1501). CTX-009 has completed a Phase 1 dose escalation study and is in a Phase 1b study in patients with solid tumors in S. Korea, where it has been observed to be well tolerated. In addition, there have been multiple confirmed partial responses (PRs) by RECIST criteria in several tumors in those studies, including PRs in colorectal cancer, cholangiocarcinoma, gastric cancer, and pancreatic cancer. The Phase 1b study in combination with chemotherapy is expected to be completed in the second half of this year. Compass plans to file an IND in the US in H2 2021 and initiate clinical studies in patients with cholangiocarcinoma, ovarian cancer and advanced colorectal cancer. Exclusive global rights to the program, except for S. Korea and China, are held by TRIGR through a license with ABL Bio Inc. (KOSDAQ: 298380). South Korean rights are held by Handok Inc. (KOSDAQ: 002390) and China rights were out-licensed to Elpiscience Biopharmaceuticals Co., Limited, under a license agreement executed on Jan 20th, 2021.

"The time now has come to maximize the therapeutic potential of this promising bispecific antibody, and I am confident that Compass has the management and development capabilities supporting the goal to advance CTX-009 to Phase 2 and Phase 3 studies in multiple indications" said George Uy, TRIGR Founder and Chief Executive Officer. "I am honored to join the Compass board and look forward to CTX-009’s success as part of Compass’ stellar immunotherapy pipeline" said Miranda Toledano, TRIGR’s Chief Operating Officer and Chief Financial Officer.

"CTX-009 presents an important addition to our pipeline of novel therapeutic antibodies and bispecific antibodies. It is uncommon to see multiple confirmed partial responses in a Phase 1 study in such an advanced patient population, and we are excited to begin to develop a robust Phase 2 program to evaluate the therapeutic potential of CTX-009 across all of the indications where this bispecific has shown substantial promise to-date" said Thomas Schuetz, MD, Ph.D., Co-Founder and Chief Executive Officer of Compass Therapeutics. "When we reviewed the data for CTX-009, we immediately saw the fit with our corporate strategy of advancing the best therapeutic candidates forward, regardless of origin" said Vered Bisker-Leib, Compass President and Chief Operating Officer. "We then moved quickly to conduct due diligence and reach agreement on a merger structure designed to deliver enhanced value for the shareholders of both companies".

Under the terms of the agreement, TRIGR will be acquired by Compass’ wholly owned subsidiary, Compass Acquisition Company, LLC, subject to certain customary conditions including the consent of the holders of a majority of TRIGR’s common shares. Consideration payable to TRIGR shareholders at closing totals an aggregate of 10,265,154 shares of Compass’ common stock, issued as unregistered shares in a private placement. In addition, TRIGR shareholders are eligible to receive up to $9 million, representing earnout payments that depend on certain events, including $5 million upon BLA approval of CTX-009 in the United States. Following the issuance of the share consideration, Compass has agreed to register such shares for resale by the recipients thereof. The transaction is expected to close in the second quarter of 2021.

About CTX-009

CTX-009 (formerly designated TR009/ABL001/NOV1501) is an anti-DLL4 X VEGF-A bispecific antibody. In November 2018, TRIGR licensed the exclusive, global rights to CTX-009, outside of South Korea, from ABL Bio, Inc. (KOSDAQ: 298380), a South Korea-based clinical-stage company focused on developing antibody therapeutics. A Phase 1 dose escalation study and a Phase 1b dose expansion monotherapy study have been completed and a Phase 1b combination study is ongoing in S. Korea. Data from CTX-009’s Phase 1 dose escalation and dose expansion monotherapy study demonstrates an approximately 20% Overall Response Rate (ORR) at the targeted therapeutic doses, with confirmed partial responses per RECIST criteria in heavily pre-treated colorectal and gastric cancer patients in whom multiple therapies have failed, including VEGF-targeted therapeutics, anti-PD-1/PD-L1 regimens and chemotherapies. Interim results from the ongoing Phase 1b combination study testing the tolerability and activity of CTX-009 in combination with irinotecan or paclitaxel, have also shown deep and sustained partial responses in difficult to treat intrahepatic cholangiocarcinoma (biliary tract cancer) patients in whom multiple lines of therapy have failed. In contrast to historical anti-DLL4 antibodies and other Notch targeted therapies, the administration of CTX-009 has not been associated with severe pulmonary hypertension. Full data from the ongoing Phase 1b studies is expected to be provided later in 2021.

On May 13, 2021 CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, reported on its results for the first quarter ended March 31, 2021 (Press release, CytRx, MAY 13, 2021, View Source [SID1234579957]). In addition, CytRx highlighted developments pertaining to its agreements with Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") and ImmunityBio, Inc. (NASDAQ:IBRX) ("ImmunityBio") as well as Centurion Biopharma. The Company’s 10-Q was filed on May 13, 2021.

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!

Steven A. Kriegsman, Chairman and Chief Executive Officer of CytRx, stated:

"CytRx continues to execute on its strategic plan to cut costs, maintain a stable capital position, and nimbly manage a portfolio of high-potential licensing agreements and strategic assets. We are very excited about the quarters ahead now that Orphazyme is preparing for prospective regulatory approvals for arimoclomol in the treatment of Niemann-Pick disease Type C and ImmunityBio is expanding its scope of clinical trials involving aldoxorubicin to treat advanced pancreatic cancer. We also continue to pursue viable partnership opportunities that can bring Centurion Biopharma’s platform into the clinical testing phase."

First Quarter Financial Overview

CytRx concluded the quarter ended March 31, 2021 with cash on hand of approximately $9.3 million, which management believes is sufficient to fund ongoing operations for the foreseeable future.
The Company recorded a net loss of $1.3 million for the quarter ended March 31, 2021, compared to a net loss of $1.2 million for the same period in 2020.
General and administrative expenses were $1.3 million for the quarter, compared with $1.2 million for the same period in 2020.
Based on a current projection of expenditures, the Company’s monthly cash burn rate is estimated to be approximately $430,000 per month. This number is largely unchanged from the close of the fourth quarter of fiscal year 2020.
Year-to-Date Highlights

Orphazyme Highlights

CytRx’s agreement with Orphazyme can deliver up to approximately $100 million in potential milestone payments and future single digit royalties paid on sales of arimoclomol.
CytRx is positioned to receive up to $10 million in potential milestone payments in 2021 based on possible U.S. and European approvals for arimoclomol to treat Niemann-Pick disease Type C ("NPC").
During the past quarter, multiple analysts issued notes that conveyed optimism regarding Orphazyme’s ability to obtain potential regulatory approvals for arimoclomol in the treatment of NPC.
Recently, Orphazyme announced the appointment of Christophe Bourdon as its new Chief Executive Officer, effective as of April 1, 2021.
As reported by Orphazyme, Mr. Bourdon has successfully launched a variety of products in demanding environments, making him an ideal individual to lead Orphazyme as it prepares for a potential commercial launch of arimoclomol.
He joins from Amgen, Inc., where he held the role of Senior Vice President, General Manager for the U.S. Oncology Business. He was leading commercialization planning and execution for several products.
Previously, Mr. Bourdon was Senior Vice President of Europe, Middle East, Africa and Canada at Alexion Pharmaceuticals Inc. as the company launched two breakthrough ultra-orphan drugs and negotiated payor access across the United Kingdom, Germany, France, Italy and Canada. He holds an MBA from IMD business school (Switzerland) and a BA from ISG (France).
Recently, Orphazyme also announced MIPLYFFA as the global brand name for arimoclomol and expanded its NPC Early Access Program in the U.S. and opened similar programs in France and Germany.
ImmunityBio Highlights

In January 2021, ImmunityBio and NantKwest announced that their ongoing Phase 2 clinical trials of a novel combination immunotherapy – which includes aldoxorubicin – for locally advanced or metastatic pancreatic cancer had produced early indications of increased survival rates for patients with no other approved treatment options.
Interim results of the three-cohort trials, known as QUILT 88, showed median survival rates of more than double that of the historic rate in patients with advanced metastatic pancreatic cancer (for which no other FDA approved treatment exists).
Recently, ImmunityBio and NantKwest announced the closing of their merger, with ImmunityBio now trading on the NASDAQ under ticker symbol IBRX.
Additional Corporate Highlights

In February 2021, CytRx announced that it is now a part of the LD Micro Index (the "Index").
The Index is designed to give the most accurate representation of the intraday activity of microcap stocks in North America.
In March 2021, CytRx participated in the H.C. Wainwright Global Life Sciences Conference and the Virtual 33rd Annual ROTH Conference.
Mr. Kriegsman’s presentation may be accessed on the News and Events page of our website.
With respect to Centurion Biopharma, Mr. Kriegsman and Lead Director Louis Ignarro, PhD have continued pursuing third-party financing and strategic partnership opportunities to advance clinical testing for the platform’s high-potential assets. Discussions with prospective partners are ongoing. There are no formal partnership updates to report at this time.
CytRx maintains federal and state net operating loss ("NOL") carryforwards of $327.6 million and $252.6 million, respectively, available to offset against future taxable income. Of this amount, $258.3 million of federal NOLs and $252.6 million of state NOL’s are unrestricted.