Y-mAbs Announces U.S. FDA Acceptance of Biologics License Application for Danyelza™ (naxitamab) for the Treatment of Neuroblastoma For Priority Review

On June 2, 2020 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that the Biologics License Application ("BLA") for Danyelza (naxitamab) for the treatment of patients with relapsed/refractory high-risk neuroblastoma has been accepted for priority review by the U.S. Food and Drug Administration ("FDA") (Press release, Y-mAbs Therapeutics, JUN 2, 2020, View Source [SID1234560753]). The FDA set an action date of November 30, 2020, under the Prescription Drug User Fee Act ("PDUFA"). The Agency also indicated in the BLA filing communication letter that it is not currently planning to hold an advisory committee meeting to discuss the application.

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 believe that the FDA’s acceptance of our BLA for priority review of our first leading antibody compound, Danyelza (naxitamab), is a significant achievement for Y-mAbs and a crucial step forward as we anticipate that Danyelza, if approved, can address a significant unmet medical need for children with relapsed/refractory high-risk neuroblastoma," stated Thomas Gad, Founder, Chairman, President and Head of Business Development and Strategy.

Dr. Claus Moller, Chief Executive Officer, continued, "We look forward to working with the Agency to bring Danyelza to appropriate patients. We are excited to move forward and plan for a seamless commercial launch of Danyelza (naxitamab), if approved."

Researchers at Memorial Sloan Kettering Cancer Center ("MSK") developed naxitamab, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests in the product.

Moleculin Announces Emory University Clinical Trial of WP1066 Begins Enrollment

On June 2, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates, reported that recruiting has begun and the first patient has been enrolled in the Emory University Phase 1 clinical trial of WP1066 for the treatment of brain tumors in children (Press release, Moleculin, JUN 2, 2020, View Source [SID1234560752]). The study is being conducted at the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta.

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!

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

WP1066 is the lead molecule in Moleculin’s portfolio of immune stimulators and modulators of transcription. WP1066 has been shown in animal models to both stimulate a natural immune response, while also inhibiting the activated form of STAT3 (p-STAT3), a gene transcription factor that is considered a master regulator of tumor-related activity.

"p-STAT3 has long been considered an ‘undruggable’ target," commented Walter Klemp, Chairman and CEO of Moleculin. "We’ve shown activity in patients with WP1220, one of the other molecules in this portfolio. This trial represents another opportunity to show that p-STAT3 is a viable target. We are honored to be a part of this effort to cure childhood brain tumors."

Idera Pharmaceuticals Announces Preliminary Data From and Planned
Continuation of the ILLUMINATE-206 Trial for the Treatment of Micro-Satellite
Stable Colorectal Cancer

On June 2, 2020 Idera Pharmaceuticals, Inc. (Nasdaq: IDRA; the "Company") reported preliminary data from the first 10 patients in the safety cohort of ILLUMINATE-206, a Phase 2, open-label, multi-center study to evaluate tilsotolimod in combination with Opdivo (nivolumab) and Yervoy* (ipilimumab) in immunotherapy-naive micro-satellite stable colorectal cancer (MSS-CRC) patients (Press release, Idera Pharmaceuticals, JUN 2, 2020, View Source [SID1234560751]). Based on data to date, the Company plans to expand the study to further evaluate this triplet combination in MSS-CRC.

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 investigate the safety profile of this triplet combination, ILLUMINATE-206 was designed with a stepwise approach to Yervoy dosage. Patients in this initial safety cohort of the study, many of whom were heavily pre-treated and rapidly progressing, received 8 mg of intratumoral tilsotolimod and 3 mg/kg of intravenous (IV) Opdivo every 2 weeks, along with 1 mg/kg of IV Yervoy every 8 weeks. This regimen was generally well tolerated; no patients discontinued treatment due to adverse events (AEs) and none experienced Grade 4 or 5 AEs. One patient experienced stable disease per RECIST v1.1 criteria, and 9 patients progressed as defined by RECIST v1.1. Investigators reported that 6 of the progressing patients had stability or reduction in size of injected lesions and 6 had stability or reduction in overall size of uninjected lesions.

Based on these results, the Company plans to enroll additional patients in this MSS-CRC cohort of ILLUMINATE-206. Planned changes in the study design intended to improve potential outcomes in this patient population include increasing the frequency of Yervoy dosing and limiting the number of allowed prior lines of treatment to two or fewer. Enrollment of the next 10 patients is targeted to begin in the fourth quarter of 2020, with data anticipated in the second quarter of 2021. Pending data from those patients, the trial may be expanded further.

"We are encouraged by the initial safety profile of this first-time triplet combination," stated Elizabeth A. Tarka, M.D., Idera’s Chief Medical Officer. "We look forward to continuing to explore the potential clinical benefit of tilsotolimod in combination with ipilimumab and nivolumab in MSS-CRC, possibly yielding a treatment alternative for these patients with few current options."

For more information about ILLUMINATE-206, visit www.ClinicalTrials.gov.

About Tilsotolimod (IMO-2125)

Tilsotolimod is an investigational, synthetic Toll-like receptor 9 agonist. Intratumoral injection of tilsotolimod has been shown to promote both innate (Type-I IFN, antigen presentation) and adaptive (T cells) immune activation. Tumors with an active immune response appear to respond better to CPIs than those that exclude or inhibit anti-tumor immune cells. Tilsotolimod in combination with CPIs may cause regression of locally injected and distant tumor lesions and increase the number of patients who benefit from immunotherapy.

Tilsotolimod received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for the treatment of anti-PD-1 refractory melanoma, in combination with ipilimumab, as well as Orphan Drug Designation for the treatment of stage IIb-IV melanoma. It is being evaluated in multiple tumor types and in combination with multiple checkpoint inhibitors. For more information on tilsotolimod trials, please visit www.ClinicalTrials.gov.

About MSS-CRC

Colorectal cancer involves the abnormal growth of cells in the colon or rectum. This type of cancer is typically tested to determine its "MSI" status, which will inform treatment approach and prognosis. MSI stands for "microsatellite instable." MSI-High (MSI-H) means that there is a high amount of instability in a tumor, whereas MSS tumors are "microsatellite stable." According to the American Cancer Society and other references, annually in the United States, approximately 140,000 people are diagnosed with CRC, of which 85% are MSS, and approximately 50,000 deaths are attributed to CRC. MSS-CRC has been shown to be highly immunosuppressive; there are no approved immunotherapy options, and a prior trial of Yervoy plus Opdivo (Bristol Myer Squibb’s CheckMate 142) yielded overall response rates of 0-10%. Given tilsotolimod’s mechanism of action of activating dendritic cells, it may serve a complementary function to Yervoy and Opdivo within the immunosuppressive tumor microenvironment of MSS-CRC patients.

TG Therapeutics to Present at the Jefferies 2020 Healthcare Conference

On June 2, 2020 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, will participate in a fireside chat during the Jefferies 2020 Healthcare Conference, being held virtually (Press release, TG Therapeutics, JUN 2, 2020, View Source [SID1234560750]). The fireside chat is scheduled to take place on Wednesday, June 3, 2020 at 10:00 AM ET.

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!

A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source

FDA approves Roche’s Tecentriq in combination with Avastin for people with the most common form of liver cancer

On June 2, 2020 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the US Food and Drug Administration (FDA) has approved Tecentriq (atezolizumab) in combination with Avastin (bevacizumab) for the treatment of people with unresectable or metastatic hepatocellular carcinoma (HCC) who have not received prior systemic therapy (Press release, Hoffmann-La Roche, JUN 2, 2020, View Source [SID1234560747]).

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’re excited that today’s approval of Tecentriq in combination with Avastin for unresectable or metastatic hepatocellular carcinoma brings a cancer immunotherapy option to people with this aggressive form of liver cancer," said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development. "The application was reviewed under the FDA’s Real-Time Oncology Review pilot and Project Orbis initiative, helping to bring this new treatment option rapidly to patients in the United States and around the world."

"The results of the IMbrave150 study are really transformative for patients with advanced liver cancer, one of the few cancers with a rising death rate and limited options in the first-line setting," said Dr Richard Finn, Professor of Medicine at the David Geffen School of Medicine at UCLA and Director of the Signal Transduction and Therapeutics Program at the UCLA Jonsson Comprehensive Cancer Center. "For the first-time we have a regimen that markedly improves survival over sorafenib, the standard of care for first-line hepatocellular carcinoma since 2007, and offers patients the opportunity for improved disease control with a favourable tolerability profile."

The review of this application was conducted under the FDA’s Project Orbis initiative, which provides a framework for concurrent submission and review of oncology medicines among international partners. According to the FDA, collaboration among international regulators may allow patients with cancer to receive earlier access to products in other countries where there may be significant delays in regulatory submissions.1 Simultaneous applications were submitted to regulators in the United States, Australia, Canada and Singapore under Project Orbis. Additionally, the FDA rapidly reviewed and approved the application under its Real-Time Oncology Review (RTOR) pilot programme, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.

The approval was based on results from the Phase III IMbrave150 study, which demonstrated that Tecentriq in combination with Avastin reduced the risk of death (overall survival; OS) by 42% (hazard ratio [HR]=0.58; 95% CI: 0.42-0.79; p=0.0006) and reduced the risk of disease worsening or death (progression-free survival; PFS) by 41% (HR=0.59; 95% CI: 0.47-0.76; p<0.0001), compared with sorafenib. IMbrave150 is the first Phase III cancer immunotherapy study to show an improvement in OS and PFS in people with unresectable or metastatic HCC compared with sorafenib. Serious adverse reactions (Grade 3-4) occurred in 38% of people in the Tecentriq and Avastin arm. The most frequent serious adverse reactions (≥2%) were bleeding in the gastrointestinal tract, infections and fever. These results were published in the New England Journal of Medicine on 14 May 2020.

Roche has an extensive development programme for Tecentriq, including multiple ongoing and planned Phase III studies, across several types of lung, genitourinary, skin, breast, gastrointestinal, gynaecological and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

About the IMbrave150 study
IMbrave150 is a global Phase III, multicentre, open-label study of 501 people with unresectable or metastatic HCC who had not received prior systemic therapy. People were randomised 2:1 to receive the combination of Tecentriq and Avastin or sorafenib. Tecentriq was administered intravenously (IV), 1200 mg on day 1 of each 21-day cycle, and Avastin was administered IV, 15 mg/kg on day 1 of each 21-day cycle. Sorafenib was administered by mouth, 400 mg twice per day, on days 1-21 of each 21-day cycle. People received the combination or the control arm treatment until disease progression or unacceptable toxicity. The two primary endpoints were OS and independent review facility (IRF)-assessed PFS per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1). Additional study endpoints were IRF-assessed overall response rate (ORR) per RECIST and mRECIST.

About hepatocellular carcinoma
HCC is an aggressive cancer with limited treatment options and is a major cause of cancer deaths worldwide.2 Every year, more than 750,000 people worldwide are diagnosed with HCC,2,3 with the majority of cases in Asia and almost half of all cases in China.3,4 In the US, the number of liver cancer cases have more than tripled since 1980 and HCC represents the fastest-rising cause of cancer-related death, while in Europe, liver cancer is also on the rise.5-7 HCC develops predominantly in people with cirrhosis due to chronic hepatitis (B or C) or alcohol consumption, and typically presents at an advanced stage.2 The prognosis for unresectable HCC remains poor, with few systemic therapeutic options and a 1-year survival rate of less than 50% following diagnosis.8

About the Tecentriq and Avastin combination
There is a strong scientific rationale to support the use of Tecentriq plus Avastin in combination. The Tecentriq and Avastin regimen may enhance the potential of the immune system to combat a broad range of cancers. Avastin, in addition to its established anti-angiogenic effects, may further enhance Tecentriq’s ability to restore anti-cancer immunity, by inhibiting vascular endothelial growth factor (VEGF)-related immunosuppression, promoting T-cell tumour infiltration and enabling priming and activation of T-cell responses against tumour antigens.

About Tecentriq
Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1, which is expressed on tumour cells and tumour-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the activation of T-cells. Tecentriq is a cancer immunotherapy that has the potential to be used as a foundational combination partner with other immunotherapies, targeted medicines and various chemotherapies across a broad range of cancers. The development of Tecentriq and its clinical programme is based on our greater understanding of how the immune system interacts with tumours and how harnessing a person’s immune system combats cancer more effectively.

Tecentriq is approved in the US, EU and countries around the world, either alone or in combination with targeted therapies and/or chemotherapies in various forms of non-small cell and small cell lung cancer, certain types of metastatic urothelial cancer and in PD-L1-positive metastatic triple-negative breast cancer. In the US, Tecentriq in combination with Avastin is approved for people with unresectable or metastatic HCC.

About Avastin
Avastin is a prescription-only medicine that is a solution for intravenous infusion. It is a biologic antibody designed to specifically bind to a protein called VEGF that plays an important role throughout the lifecycle of the tumour to develop and maintain blood vessels, a process known as angiogenesis. Avastin is designed to interfere with the tumour blood supply by directly binding to the VEGF protein to prevent interactions with receptors on blood vessel cells. The tumour blood supply is thought to be critical to a tumour’s ability to grow and spread in the body (metastasise).

About Roche in cancer immunotherapy
Roche’s rigorous pursuit of groundbreaking science has contributed to major therapeutic and diagnostic advances in oncology over the last 50 years, and today, realising the full potential of cancer immunotherapy is a major area of focus. With over 20 molecules in development, Roche is investigating the potential benefits of immunotherapy alone, and in combination with chemotherapy, targeted therapies or other immunotherapies with the goal of providing each person with a treatment tailored to harness their own unique immune system to attack their cancer. Our scientific expertise, coupled with innovative pipeline and extensive partnerships, gives us the confidence to continue pursuing the vision of finding a cure for cancer by ensuring the right treatment for the right patient at the right time.

In addition to Roche’s approved PD-L1 checkpoint inhibitor, Tecentriq (atezolizumab), Roche’s broad cancer immunotherapy pipeline includes other checkpoint inhibitors, such as tiragolumab, a novel cancer immunotherapy designed to bind to TIGIT, individualised neoantigen therapies and T-cell bispecific antibodies. To learn more about Roche’s scientific-led approach to cancer immunotherapy, please follow this link:
View Source