On November 16, 2021 ORPHELIA Pharma and K.S. KIM INTERNATIONAL (SK-PHARMA) LTD reported the execution of an exclusive distribution and marketing agreement for Kigabeq (vigabatrin) in the territory of the Russian Federation (Press release, ORPHELIA Pharma, NOV 16, 2021, View Source;utm_medium=rss&utm_campaign=orphelia-pharma-and-k-s-kim-sk-pharma-sign-an-agreement-to-supply-kigabeq [SID1234595693]).

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!

Kigabeq, the first pediatric form of vigabatrin developed by ORPHELIA Pharma, is notably indicated in the treatment of infantile spasms (West syndrome). Kigabeq is approved in the European Union, where this medicine, intended exclusively for children, benefits from a centralized marketing authorization (Pediatric Use Marketing authorization, PUMA).

"We are delighted to sign this agreement with K.S. KIM, which is based on the value of Kigabeq in paediatrics", says Hugues BIENAYME, Founder and CEO of ORPHELIA Pharma, "With their strong footstep in the distribution of orphan medicines in the Russian territory, K.S. KIM is an excellent partner for Kigabeq."

"With Kigabeq, the only pediatric presentation of vigabatrin, K.S. KIM is expanding its portfolio of drugs intended for rare and serious pathologies in children" adds Dr. Shlomo Sadoun, CEO of K.S. KIM, "Kigabeq is a drug which address unmet medical needs for young patients affected by severe and resistant epilepsies. Our ultimate objective is to register Kigabeq as an orphan drug in Russia, nevertheless we will start distributing it immediately as an unlicenced medicine augmenting unmet needs ".

"Our objective is to make Kigabeq available as widely as possible, so that all children affected by infantile spasms can benefit." concludes Gilles ALBERICI, President of ORPHELIA Pharma, "Thanks to this agreement with K.S. KIM, we are very confident that Russian children suffering from infantile spasms will have access to Kigabeq as quickly as possible."

On November 16, 2021 CytoDyn Inc. (OTCQB: CYDY) ("CytoDyn" or the "Company"), a late-stage biotechnology company developing leronlimab, a CCR5 antagonist with the potential for multiple therapeutic indications, reported that Health Canada authorized the emergency use of leronlimab for the treatment of a patient with metastatic triple-negative breast cancer (mTNBC) (Press release, CytoDyn, NOV 16, 2021, View Source [SID1234595694]).

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!

Nader Pourhassan, Ph.D., CytoDyn’s President and Chief Executive Officer, said, "We are encouraged by Health Canada’s emergency use approval of leronlimab, which we believe could have potential benefit as a therapeutic option for treating mTNBC. We now plan to ask Health Canada to allow expanded access use for all mTNBC patients who might have no other treatment options. We are also seeking similar approvals in other countries and look forward to providing leronlimab for mTNBC patients, as well as for treatment of other solid tumor cancers, in the future. In addition, we will be filing for expanded access use for mTNBC patients in the U.S. shortly."

On November 16, 2021 NOXXON Pharma N.V. (Euronext Growth Paris: ALNOX) (Paris:ALNOX), a biotechnology company focused on improving cancer treatments by targeting the tumor microenvironment (TME), reported that its key opinion leader (KOL) webinar on NOX-A12 and radiotherapy combination, which is a promising new approach to treating brain cancer, will be hosted on Tuesday, November 23, 2021 at 02:00 p.m. CET (08:00 a.m. EST) (Press release, NOXXON, NOV 16, 2021, View Source [SID1234595710]).

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 webinar will feature a presentation from Frank A. Giordano, M.D., Director and Chair of the Department of Radiation Oncology, University Hospital Bonn, Germany, who will discuss the current treatment landscape and high unmet medical need in treating patients with brain cancer, particularly glioblastoma multiforme, GBM. Following his presentation at the Society for Neuro-Oncology (SNO) Annual Meeting on November 19, Dr. Giordano will further discuss the latest promising patient results from the GLORIA study, evaluating NOX-A12 in combination with radiotherapy in first-line brain cancer patients with unmethylated MGMT promoter.

NOXXON’s management team will also give a corporate overview and an update on clinical programs. Dr. Giordano will be available for questions following the formal presentation.

To register for the event, please click here.

NOX-A12 is an inhibitor of the chemokine CXCL12 and has been granted orphan drug status in the US and EU for the treatment of certain brain cancers and produced encouraging clinical data thus far. Dr. Giordano will also compare NOXXON’s trial data to historical data evaluating how a matched patient cohort responded to the treatment according to current standard of care, which has remained unchanged since 2006.

Frank Giordano, M.D., is Director and Chair of the Department of Radiation Oncology at the University Hospital Bonn, Germany. He is an expert in precision radiation therapy and intraoperative irradiation of malignant tumors and has received international recognition for his brain tumor research, including an award from the American Society of Radiation Oncology (ASTRO) and an honorary membership of the Spanish Society of Radiation Oncology (SEOR). Dr. Giordano received his medical degree from the University of Heidelberg, Germany, and did his post-doctoral training as a Peter Engelhorn fellow at the German Cancer Research Center (DKFZ). He received clinical training at the National Center for Tumor Diseases (NCT) Heidelberg and the University Medical Center Mannheim, where he served as acting chairman and director of the Department of Radiation Oncology before moving to Bonn. For many years, his research has focused on optimized radiation therapy of brain cancers to offer cancer patients personalized and even more effective treatment. As one of the few Else-Kröner-Fresenius Excellence Fellows, Dr. Giordano developed innovative therapy options that even found their way in clinical practice. He sees great potential in the combination of radiotherapy and immunomodulatory therapy.

On November 16, 2021 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of targeted drugs for the treatment of cancer, reported that patients in a Phase 1 study of iopofosine I-131 ("iopofosine") in children and adolescents with relapsed and refractory high grade gliomas (HGGs) and soft tissue sarcomas, exhibited positive changes in various tumor parameters (Press release, Cellectar Biosciences, NOV 16, 2021, View Source [SID1234595695]). The Phase 1 study is an international, open-label, dose escalation, safety study of iopofosine in children and adolescents with relapsed or refractory cancers, specifically HGGs, high risk neuroblastoma and select soft tissue sarcomas.

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 independent data monitoring committee (DMC) had previously determined that doses up to 60 mCi/m2 were safe and tolerable and to initiate the 75 mCi/m2 dosing cohort. The DMC advised, based upon the initial data, to enrich the 60 mCi/m2 dose level for patients over the age of 10 with HGG and Ewing sarcoma. The initial response and tumor uptake were confirmed by further therapeutic responses, evidenced by changes in tumor parameters. This includes patients with relapsed HGGs experiencing over 5 months of progression free survival (PFS).

"Initial responses to iopofosine I-131 in relapsed pediatric brain tumors are most encouraging. Current treatment paradigms typically result in only 2-3 months1 of PFS and while there is no comparator in this study, iopofosine data to date have demonstrated nearly double the PFS versus historical data," said Laurence Reilly interim chief medical officer of Cellectar. "Based upon these data we will continue to enroll patients with high grade gliomas and soft tissue sarcomas, and we look forward to engaging with the FDA in order to outline a potential registrational pathway."

Pediatric HGGs are a collection of aggressive brain and central nervous system tumor subtypes including diffuse intrinsic pontine gliomas, glioblastomas, astrocytomas and ependymomas. Children with these tumors have a poor prognosis and 5-year survival of less than 30%.

About iopofosine (also known as CLR 131)

Iopofosine is a small-molecule Phospholipid Drug Conjugate designed to provide targeted delivery of iodine-131 (radioisotope) directly to cancer cells, while limiting exposure to healthy cells. We believe this profile differentiates iopofosine from many traditional on-market treatments. Iopofosine is currently being evaluated in the CLOVER-WaM Phase 2 pivotal study in patients with relapsed/refractory (r/r) Waldenstrom’s macroglobulinemia (WM), a Phase 2b study in r/r multiple myeloma (MM) patients and the CLOVER-2 Phase 1 study for a variety of pediatric cancers. The U.S. Food and Drug Administration granted iopofosine Fast Track Designation for WM patients having received two or more prior treatment regimens, as well as r/r MM and r/r diffuse large B-cell lymphoma (DLBCL). Orphan Drug Designations (ODDs) have been granted for WM, MM, neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. Iopofosine was also granted Rare Pediatric Disease Designation (RPDD) for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. The European Commission granted an ODDs for r/r MM and WM.

On November 16, 2021 NeoImmuneTech, Inc. (KOSDAQ: 950220), a clinical-stage T cell-focused biopharmaceutical company, reported that new data from two clinical trials evaluating the company’s lead asset NT-I7 (efineptakin alfa), a novel T cell amplifier, in three posters at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting (Press release, NeoImmuneTech, NOV 16, 2021, View Source [SID1234595711]). The data come from two cohorts of the Phase 2a portion of a Phase 1b/2a clinical trial evaluating NT-I7 in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) for the treatment of patients with relapsed/refractory advanced solid tumors, as well as a Phase 1 investigator-initiated trial evaluating NT-I7 following adjuvant chemotherapy (temozolomide [TMZ]) and radiation in patients with high-grade gliomas (HGG).

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 combination data of NT-I7 and pembrolizumab were presented in two posters, titled "Initial Biomarker and Clinical Data of a Phase 2a Study of NT-I7, a Long-Acting Interleukin-7, plus Pembrolizumab: Cohort of Subjects with Checkpoint Inhibitor-naïve Advanced MSS-Colorectal Cancer" and "Preliminary Biomarker and Clinical Data of a Phase 2a Study of NT-I7, a Long-Acting Interleukin-7, plus Pembrolizumab: Cohort of Subjects with Checkpoint Inhibitor-naïve Advanced Pancreatic Cancer," by lead authors Richard D. Kim, M.D., Moffitt Cancer Center, and Aung Naing, M.D., The University of Texas MD Anderson Cancer Center, respectively.

The data shows that the combination of NT-I7 and pembrolizumab was well tolerated and showed early anti-tumor activity in patients with checkpoint inhibitor (CPI)-naive relapsed/refractory (r/r) pancreatic and microsatellite-stable colorectal cancers (MSS-CRC), two immune-cold tumor types. The interim analysis of the phase 2 met the primary endpoint of overall response rate (ORR) in these cohorts. The median follow-up of the efficacy data was ~5.8 months in the MSS-CRC Cohort and ~4.6 months in the Pancreatic Cancer Cohort. Three immune partial responses (iPR) by iRECIST were observed, including one partial response (PR) by RECIST 1.1 in 17 evaluable MSS-CRC patients, as well as one iPR/PR observed in 17 evaluable pancreatic cancer patients. The pancreatic cancer responder was confirmed to have a microsatellite-stable tumor. Responses were first observed at weeks 12, 18, and 24 post-first dose. Increase of T cell infiltration in the tumor microenvironment were observed in the responders, a key indicator that the tumor microenvironment is shifting from immune-cold to immune-hot. Importantly, an even greater magnitude of increase in stem-cell memory CD8+ T cells (Tscm) was observed in the blood.

The data for NT-I7 following chemoradiation were presented in a poster titled "NT-I7, a long-acting interleukin-7, promotes expansion of CD8 T cells and NK cells and immune activation in patients with newly diagnosed high-grade gliomas after chemoradiation," by lead author Jian Campian, M.D., Ph.D., Mayo Clinic.

The data shows that NT-I7 is well tolerated following chemoradiation therapy in HGG patients. Notable ALC increases were observed, with more prominent increases in patients dosed at the 540µg/kg or higher levels of NT-I7. Marked increase of Tscm was also observed, in this case in absence of a checkpoint inhibitor. Durable progression-free survival (PFS) was noted in MGMTp unmethylated HGG patients, a sub-group who often have a poorer prognosis. Six out of eight patients treated at the therapeutic dose of 720µg/kg or higher are continuing on in the study.

"We are excited to share that the combination of NT-I7 and pembrolizumab was found to be well tolerated and showed early signs of clinical activity in patients with difficult-to-treat cancers, like relapsed/refractory pancreatic and microsatellite-stable colorectal cancer," said Se Hwan Yang, Ph.D., President and Chief Executive Officer of NeoImmuneTech. "Immune-cold tumors such as MSS-CRC and pancreatic cancer have shown very limited clinical activity with current standard of care therapies. We are also encouraged by the preliminary clinical efficacy results in NT-I7 following chemoradiation. These clinical data support our continued efforts in these and other tumor types, and we look forward to future analyses of the data as we move forward."

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, N.J., USA.

About NT-I7
NT-I7 (efineptakin alfa) is the only clinical-stage long-acting human IL-7, and is being developed for oncologic and immunologic indications, in which T cell amplification and enhanced functionality may provide clinical benefit. IL-7 is a fundamental cytokine for naïve and memory T cell development and for sustaining immune response to chronic antigens (as in cancer) or foreign antigens (as in infectious diseases). In clinical trials to date, NT-I7 has exhibited favorable PK/PD and safety profiles, both as a monotherapy and in combination with other anticancer treatments. NT-I7 is being studied in multiple clinical trials in solid tumors and as a vaccine adjuvant. Studies are being planned for testing in hematologic malignancies, additional solid tumors and other immunology-focused indications.