Y-mAbs Announces Regulatory Filing for DANYELZA® (naxitamab-gqgk) in Brazil

On September 26, 2022 Y-mAbs Therapeutics, Inc. ("Y-mAbs", NASDAQ: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that Adium Pharma S.A. ("Adium") has submitted a regulatory filing for DANYELZA (naxitamab-gqgk) for the treatment of patients with relapsed/refractory high-risk neuroblastoma to the Brazilian Health Regulatory Agency, Agência Nacional de Vigilância Sanitária ("Anvisa") (Press release, Y-mAbs Therapeutics, SEP 26, 2022, View Source [SID1234621434]).

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"We are very pleased with the regulatory capabilities of our partner Adium resulting in submission of DANYELZA for consideration of potential approval in Brazil. We believe this submission marks another important milestone in our aim to make DANYELZA globally available and address an unmet medical need for families in Brazil," said Thomas Gad, President, and Interim Chief Executive Officer. "If approved by Anvisa, we look forward to working closely with Adium on the production and logistic planning for DANYELZA to help ensure timely availability to patients in Brazil."

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

About DANYELZA (naxitamab-gqgk)

In the United States, DANYELZA (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication was approved under accelerated approval by the U.S. Federal Drug Administration based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA includes a Boxed Warning for serious infusion-related reactions, such as cardiac arrest and anaphylaxis, and neurotoxicity, such as severe neuropathic pain and transverse myelitis. See full Prescribing Information for complete Boxed Warning and other important safety information.

Innocoll and DURECT Announce U.S. launch of POSIMIR®

On September 26, 2022 Innocoll Pharmaceuticals Limited, a commercial-stage biotechnology company and portfolio business of Gurnet Point Capital, and DURECT Corporation ( Nasdaq: DRRX) reported the recent commercial launch of POSIMIR (bupivacaine solution) in the United States (Press release, DURECT, SEP 26, 2022, https://www.durect.com/news/innocoll-and-durect-announce-u-s-launch-of-posimir/ [SID1234621433]). POSIMIR is an FDA-approved non-opioid, sustained-release local analgesic for the treatment of post-surgical pain in adults following arthroscopic subacromial decompression surgery.

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"We are excited to bring a potential cornerstone of multi-modal post-operative pain management to surgeons with the launch of POSIMIR," said Louis Pascarella, Chief Executive Officer of Innocoll. "The launch of POSIMIR establishes Innocoll as a leader in non-opioid pain management with two commercialized bupivacaine-based, sustained-release, non-opioid products and we look forward to bringing this new option to patients."

"We are pleased that Innocoll, with its dedicated hospital sales and marketing organization, has launched POSIMIR in the United States and recorded its first sales," added James E. Brown, President and Chief Executive Officer of DURECT Corporation. "Bringing a new non-opioid analgesic option with POSIMIR is a welcome next step in the evolution of pain management for patients."

Per the terms of the collaboration between Innocoll and DURECT, Innocoll will make a $2 million payment to DURECT triggered by the first commercial sale of POSIMIR. Previously, in August 2022, DURECT was issued a new patent by the US Patent Office, extending US patent coverage of POSIMIR to at least 2041, resulting in an $8 million milestone payment by Innocoll to DURECT. These payments are in addition to the $4 million upfront license fee received in January 2022. As the launch progresses, DURECT will receive tiered, low double-digit to mid-teen royalties on net product sales in the United States, as well as additional milestone payments up to $122 million in the aggregate, depending on the achievement of certain commercial, regulatory and intellectual property milestone payments with respect to POSIMIR.

In addition to the exclusive right to develop and commercialize POSIMIR in the United States, Innocoll also has been granted the right to conduct additional development activities to expand the approved indications for POSIMIR, and DURECT’s contract manufacturing supply agreement for POSIMIR has been assigned to Innocoll. DURECT retains all commercial rights to POSIMIR throughout the rest of the world.

About POSIMIR
POSIMIR (bupivacaine solution) for infiltration use is a novel and proprietary product that combines the strength of 660 mg of bupivacaine base with the innovative SABER platform technology, enabling continuous sustained delivery of a non-opioid local analgesic over 3 days in adults. POSIMIR contains more bupivacaine than any other approved single-dose sustained-release bupivacaine product. At the end of surgery, POSIMIR is administered into the subacromial space under direct arthroscopic visualization, where it continuously releases bupivacaine for 72 hours or more.

Indications and Usage
POSIMIR (bupivacaine solution) for infiltration use is indicated in adults for administration into the subacromial space under direct arthroscopic visualization to produce post-surgical analgesia for up to 72 hours following arthroscopic subacromial decompression.

Limitations of Use
Safety and effectiveness have not been established in other surgical procedures, including soft tissue surgical procedures, other orthopedic procedures, including for intra-articular administration, and boney procedures, or when used for neuraxial or peripheral nerve blockade.

Full Prescribing Information, including the Boxed Warning, is available at www.POSIMIR.com

Important Safety Information
BOXED WARNING: Risk of Potential Adverse Embolic Effects Resulting From Inadvertent Intravascaular Injection. Inadvertent intravascular injection could cause POSIMIR droplets to be deposited in the pulmonary and other capillary beds. Administer POSIMIR into the subacromial space at the end of arthroscopic shoulder surgery. Direct arthroscopic visualization must be used to confirm proper placement of the needle tip before injecting POSIMIR.

In POSIMIR clinical studies, no inadvertent intravascular injections were observed. Do not inject POSIMIR intravascularly.

POSIMIR is contraindicated in patients with a known hypersensitivity to any amide local anesthetic, or other components of POSIMIR, as well as in patients undergoing obstetrical paracervical block anesthesia. There is a risk of joint cartilage necrosis with unapproved intra-articular use of POSIMIR. Unintended intravascular injection of POSIMIR may be associated with systemic toxicities, including CNS or cardiorespiratory depression and coma, progressing ultimately to respiratory arrest. As with other local anesthetics, patients should be monitored for central nervous system, cardiovascular, and allergic reactions. Avoid additional use of local anesthetics within 168 hours following administration of POSIMIR. Cases of methemoglobinemia have been reported in association with use of local anesthetics. There have been reports of chondrolysis (mostly in the shoulder joint) following intra-articular infusion of local anesthetics, which is an unapproved use. POSIMIR should be used cautiously in patients with impaired hepatic and cardiovascular function. Adverse events reported with an incidence greater than or equal to 10% and greater than control following POSIMIR administration in shoulder surgery were dizziness, dysgeusia, dysuria, headache, hypoesthesia, paresthesia, tinnitus, and vomiting. Adverse events reported with an incidence greater than or equal to 10% and greater than control following POSIMIR administration in soft tissue surgical procedures were anemia, bradycardia, constipation, C-reactive protein increased, diarrhea, dizziness, dysgeusia, headache, nausea, post-procedural contusion (bruising), procedural pain, pruritus, pyrexia, somnolence, surgical site bleeding, visible bruising and vomiting.

Bioheng Biotech Announces Publication of Impressive Results with CD7-targeted allogeneic CAR-T cell therapy for Relapsed or Refractory T Cell Malignancies

On September 26, 2022 Nanjing Bioheng Biotech Co., Ltd, a clinical-stage biotechnology company focused on developing novel cellular immunotherapy, reported that a phase I clinical study results of RD13-01, an anti-CD7 universal CAR-T therapy product, have been published in the reputable academic journal, Cell Research. (View Source) (Press release, Bioheng Biotech, SEP 26, 2022, View Source [SID1234621432]).

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The results published with the title of "CD7-targeted allogeneic CAR-T cell therapy with enhanced efficacy for relapsed/refractory CD7-positive hematological malignancies: A phase I clinical study" was from a first-in-human, single-arm, dose-escalation phase I clinical study. The primary objective of the study was to assess the safety and tolerability of RD13-01, while the secondary objectives were to assess the preliminary anti-tumor activity and characterize the pharmacokinetics of RD13-01. Patients were given a lymphodepletion chemotherapy with fludarabine, cyclophosphamide, and etoposide before RD13-01 infusion. RD13-01 demonstrated a manageable safety and tolerability profile, no DLT, GvHD or ICANS was observed, and no patients experienced ≥ grade 3 CRS. Of the 11 patients included in efficacy analysis, 9 (82%) achieved an objective response. Of the 8 patients with leukemia, 6 (75%) achieved MRD-CR/CRi.

"T cell malignancies are highly aggressive hematological tumors which are generally associated with poor prognosis. In particular relapsed or refractory (r/r) disease has dismal outcomes with a 5-year overall survival (OS) rate lower than 20%, there is an urgent medical need to develop novel therapies for this type of disease," said the PI of this study, He Huang, MD, PhD, Professor of hematology, President of The First Affiliated Hospital, Zhejiang University School of Medicine. "Besides the marked attenuated cytokine release syndrome (CRS) safety profile and impressive efficacy enhancement, I am also impressed with the availability and accessibility of RD13-01 for these patients with uncommon aggressive malignancies. The interval between enrollment and infusion was no more than 7 days, and no patients enrolled failed to receive RD13-01 infusion due to disease progression, leukapheresis or manufacture failure. It is expected that allogeneic CAR-T cell products provide more choices to address the unmet medical needs."

"We are proud to have our innovative allogeneic CAR-T studies published in a reputable, peer-reviewed scientific journal," said Jiangtao Ren, PhD, Co-founder and Chief Scientific Officer of Bioheng, "We developed healthy donor-derived, CD7-targeted CAR-T cells (RD13-01) with genetic modifications to resist fratricide, GvHD and allogeneic rejection, as well as to potentiate antitumor function. It is easy to use with excellent safety and efficacy. Collectively, these promising results demonstrate our next generation allogeneic CAR-T platform’s potential to maximize CAR-T’s functionality to eradicate malignant tumors. Further phase II investigations are warranted to evaluate the long-term safety and efficacy in larger scales."

About RD13-01

RD13-01 is allogeneic CAR-T cell therapy targeting CD7 with genetic modifications to resist fratricide, graft-versus-host-disease (GvHD) and allogeneic rejection, as well as to potentiate antitumor function. It is the first proprietary product based on Bioheng’s next-generation generic CAR-T technology platform. It has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of T-cell acute lymphoblastic leukemia (T-ALL) on June 20, 2021.

Icon Group and Varian Collaborate on an International HyperArc Registry to Advance Treatment of Brain Tumors

On September 26, 2022 This World Cancer Research Day, Icon Group (Icon) reported that becomes the first international participant in the HyperArc registry, which is sponsored by Varian, a Siemens Healthineers company (Varian) (Press release, Icon Group, SEP 26, 2022, View Source [SID1234621430]). HyperArc technology allows clinicians to treat tumors with highly conformal doses of radiation therapy. The automated system can even target several tumors in different locations in the brain. The Hyperarc registry will collect patient data for clinical research that will be used to inform and expand treatment options for patients with brain tumors.

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Icon was the first care provider in Australia to offer HyperArc technology. The collaboration marks the first research project for Varian’s and Icon’s global five-year strategic partnership, which includes the development of ongoing clinical and technical research programs to advance cancer care.

The HyperArc registry will contain data collected during and after the course of radiation treatment using HyperArc on a Varian linear accelerator. It helps clinicians understand real-world use cases of HyperArc and evaluate caseloads, processes, and outcomes from treatment techniques. An automated process uploads treatment data to the registry to facilitate clinical research.

Radiation Oncologist and Principal Investigator, Associate Professor Matthew Foote says this registry could help physicians improve patient outcomes.

"We have already started to see the potential of HyperArc to greatly improve quality of life for patients with multiple brain tumors. Previously many of these patients would have had to receive radiation to the whole brain, resulting in undesirable side effects and suboptimal quality of life. Now we can treat up to 20 metastases in one treatment session, with promising results. The registry is another step forward in our ability to analyze real world data to inform treatment pathway decisions and ultimately push the boundaries of this technology to improve patient outcomes and evolve how we treat cancer," said Associate Professor Foote.

The registry will be open across multiple Icon Cancer Centre locations in Australia, with the potential to expand across the group’s international network. This multicentre approach will collect anonymized data to evaluate the comparative effectiveness, relative toxicity, and quality of care in a large cohort of patients.

Icon Group CEO, Mark Middleton said, "Icon proudly invests in clinical trials and research to bring tomorrow’s treatments closer to today’s reality. With global partnerships and clinicians passionate about research, we can activate trials and research programs efficiently and effectively to advance cancer care and ultimately benefit even more cancer patients."

Kevin O’Reilly, President of Radiation Oncology Solutions at Varian, said, "Varian has a broad commitment to supporting clinical research to advance cancer treatment. Collaborating with Icon and their network of clinicians represents an important opportunity to develop clinical evidence, so that care providers can offer optimal treatment to their patients. We value our strategic partnership with Icon, and we look forward to continuing to work together to improve the overall patient experience."

The HyperArc registry will enable Icon to collaborate with other institutions, such as the University of Alabama at Birmingham, which is another key participant in the HyperArc registry. The registry is currently enrolling at Icon cancer centers located in Gold Coast University Hospital and Gold Coast Private. The registry will also open at Icon’s locations in Canberra, ACT and Queensland centers at Greenslopes and Maroochydore.

Prosoma Digital Therapeutics and EVERSANA Announce Commercialisation Partnership for New Therapeutic Supporting Mental Health and Behavioural Needs of Cancer Patients

On September 26, 2022 Prosoma Digital Therapeutics, innovators in reinventing healthcare for oncology patients, reported that it has partnered with EVERSANA, a pioneer of next-generation commercial services to the global life sciences industry, to support the expected launch and commercialisation for Prosoma’s digital therapy to support the mental health of cancer patients (Press release, EVERSANA, SEP 26, 2022, View Source [SID1234621429]).

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The digital therapy, Living Well, is the company’s proprietary application designed to improve the quality of life and reduce stress, anxiety, and depression symptoms related to cancer diagnosis and treatment.EVERSANA’s leading experts in launching and commercialising digital therapeutic solutions will help Prosoma navigate regulatory pathways and plan for commercialisation.

"As we looked to navigate the complexity of commercialising our digital solutions in the United States, it became clear we need a partner with the infrastructure and experience to help us move quickly," said Andrzej Jonczyk, Chief Executive Officer, Prosoma U.S. "As we spoke with partners across Europe and the world, one name continued to rise to the top and that was EVERSANA. Their integrated platform and deep experience in digital therapeutics make them the perfect partner to help bring our important therapies to patients."

"The future of healthcare continues to evolve as innovative solutions improve the patient journey," said Jim Lang, CEO, EVERSANA. "EVERSANA was built with this in mind. By helping Prosoma, we will provide critical mental health support for cancer patients and caregivers through this novel approach to digital therapy."

EVERSANA and Prosoma Digital Therapeutics will attend and be available to discuss the partnership at the 5th annual DTx East Conference, September 27-29 in Boston, Massachusetts.