Y-mAbs Announces Priority Review of BLA for DANYELZA® (naxitamab-gqgk) in China

On September 13, 2021 Y-mAbs Therapeutics, Inc. ("Y-mAbs" or the "Company"), 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 its partner SciClone Pharmaceuticals (Holdings) Limited ("SciClone Pharmaceuticals") has been granted priority review of the Biologics License Application ("BLA") for DANYELZA (naxitamab-gqgk) for the treatment of patients with relapsed/refractory high-risk neuroblastoma by the Center for Drug Evaluation ("CDE") of China’s National Medical Products Administration ("NMPA") (Press release, Y-mAbs Therapeutics, SEP 13, 2021, View Source [SID1234587598]).

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"We are pleased to see SciClone Pharmaceuticals receive priority review of the DANYELZA BLA in China. Priority review in China is intended to expedite review and approval, which means that we could potentially be facing approval and launch of DANYELZA in China as soon as the first quarter of 2022," commented Thomas Gad, founder, Chairman and President at Y-mAbs.

Dr. Claus Moller, Chief Executive Officer, continued, "DANYELZA was recently prescribed for the first time in China, and we were excited to learn that the first patient had received treatment at the Lecheng Branch of Hainan Women and Children’s Medical Center in Hainan Boao Lecheng International Medical Tourism Pilot Zone."

Researchers at 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 product.

About DANYELZA (naxitamab-gqgk)

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 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.

According to New Multinational Survey, Healthcare Providers Believe Immunotherapy Has Potential to Positively Impact Earlier-Stage Cancer Treatment Landscape Across Tumor Types

On September 13, 2021 Bristol-Myers Squibb reported that Results of a new multinational survey of healthcare providers revealed that the majority of participants expect immunotherapy to have a positive impact on the treatment landscape for patients with earlier-stage cancers in the adjuvant (after surgery), neo-adjuvant (before surgery) and peri-operative (both before and after surgery) settings, if approved by regulatory bodies (Press release, Bristol-Myers Squibb, SEP 13, 2021, View Source [SID1234587616]). The survey, commissioned by Bristol Myers Squibb (NYSE: BMY), included over 250 oncologists, surgeons and specialists in the U.S., Japan, Germany, Italy and France who currently treat patients with stage I-III disease across eight different types of cancer. While healthcare providers surveyed are more satisfied with current treatments in cancers where earlier options are well established, they do not always use treatment before or after surgery, and the vast majority of respondents express enthusiasm for the potential of immunotherapy in earlier-stage cancers.

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"Cancer recurrence often marks the transition from curable to incurable disease and can be life-altering for patients, which is why we continually investigate ways to improve upon the standard of care," said Michele Maio, M.D., Ph.D., Director of Medical Oncology and Immunotherapy, University Hospital of Siena. "Optimizing cancer treatment in its early phases, before the disease returns or spreads, represents a significant opportunity and unmet need. Research with immunotherapy in these settings is growing, and the survey results announced today reinforce that a majority of healthcare providers surveyed are enthusiastic about its future potential."

Current Treatment Approaches in Earlier Stages of Cancer

Today, treatment in the neoadjuvant, adjuvant or peri-operative settings may consist of chemotherapy, radiation, targeted therapy, chemoradiation therapy, and increasingly in a subset of tumors, immunotherapy. The survey identified a number of trends on the current use, satisfaction and drivers of treatment choices in earlier-stage cancers.

HCPs surveyed do not always use treatment beyond surgery: The majority of HCPs surveyed report that they "sometimes" use neoadjuvant (62%), adjuvant (55%) or peri-operative (54%) treatments for patients with earlier stages of cancer, highlighting the opportunity for earlier intervention.
HCPs surveyed are more satisfied with current treatments in cancers where earlier options are well established :Six out of 10 or more survey respondents are "very" or "fairly" satisfied with current neoadjuvant (67%), adjuvant (70%) and peri-operative (61%) treatment options as a whole. However, satisfaction varies by tumor* and is highest among cancers with well-established therapies, like breast cancer (87% satisfaction in the neoadjuvant/adjuvant settings) and melanoma (77% satisfaction in adjuvant). In kidney and liver cancers, satisfaction is noticeably lower (less than 35% satisfaction with neoadjuvant, adjuvant and peri-operative options), signaling the need for additional research.
HCPs surveyed use immunotherapy in earlier stages (either as approved therapies or in clinical trials), but not as often as other treatments : Currently, respondents report more experience using chemotherapy (85%, 86% and 73% for neoadjuvant, adjuvant and peri-operative, respectively) than immunotherapy (48%, 65% and 39%, respectively), likely reflecting that immunotherapy remains under investigation in a number of tumor types and only recently emerged as an approved option in others.
The Potential of Immunotherapy for the Future of Earlier-Stage Treatment

To better understand the future landscape of neoadjuvant, adjuvant and peri-operative treatment, the survey explored HCPs’ perceptions of immunotherapy and found:

Many HCPs surveyed see potential for a positive impact with immunotherapy in earlier stages of disease: Participants see the greatest potential for positive outcomes in melanoma (92% report positive potential impact in the adjuvant setting), lung cancer (89% in the neoadjuvant setting) and bladder or urothelial cancer (84% in the adjuvant setting).*
HCPs surveyed believe the potential benefits of immunotherapy align with what currently drives treatment preferences in earlier settings : Selecting from a list, HCPs surveyed state the most important potential benefits of immunotherapy as longer overall survival (64%), increased disease-free, event-free or recurrence-free survival (57%) and maintenance of quality of life (54%). These responses align with the factors HCPs surveyed report as most important in making treatment decisions in patients with operable tumors (long-term survival, prevention of relapse or recurrence and quality of life).
HCPs surveyed cite the need for more data as a leading barrier to adoption of immunotherapy in earlier stages of cancer : From a list, surveyed participants selected the need for long-term and overall survival data as leading barriers to adoption of immunotherapy in earlier stages of cancer (53% and 50%, respectively), reinforcing the importance of ongoing research and follow-up analyses.
"Over the past decade, immunotherapy research has evolved, starting with a focus on metastatic cancers, and more recently, expanding to explore the role of these treatments in earlier stages of the disease," said Jonathan Cheng, senior vice president, head of Oncology Development, Bristol Myers Squibb. "We hope that by addressing cancer in earlier stages, when the immune system may be more responsive and intact, immunotherapy may have the potential to prevent recurrence and ultimately lead to patients living longer. Oncologists, surgeons and specialists who responded to this survey are similarly optimistic about the potential of bringing immunotherapy into earlier stages of cancer."

*Tumor-specific results are based on responses from HCPs who currently treat these types of cancer, a subset of the full sample.

About the Survey

On behalf of Bristol Myers Squibb, Ipsos MORI carried out an online survey on treatment perceptions and practices in earlier stages of cancer. A total of 256 healthcare providers across five countries (France n=50, Germany n=50, Italy n=50, U.S. n=56 and Japan n=50) chose to take part in the online survey. Fieldwork took place between June 3 and July 2, 2021. Respondents included medical oncologists, surgeons (general, thoracic, breast, respiratory and gastroenterological surgeons) and specialists (urologists, dermatologists, pulmonologists, gastroenterologists and otolaryngologists) who treat patients with stage I-III disease across eight different cancer types (bladder/urothelial cancer, breast cancer, gastroesophageal cancers, head and neck cancer, kidney cancer, liver cancer, lung cancer and melanoma). A quota was set to obtain a minimum of 25 medical oncologists in France (n=28), Germany (n=29), Italy (n=29) and U.S. (n=25). The sample included a mix of hospital-, university- and community-based HCPs. The respondents were sampled from pre-existing panels of self-selecting HCPs, managed by M3 and SHC.

Bristol Myers Squibb: Creating a Better Future for People with Cancer

Bristol Myers Squibb is inspired by a single vision — transforming patients’ lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to look at cancer from every angle. Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.

Walking Fish Therapeutics Launches with $50 Million in Series A Financing to Accelerate Development of B Cell Therapies for Multiple Diseases

On September 13, 2021 Walking Fish Therapeutics, a leader in B cell engineering, reported the close of $50M Series A financing— led by investors including Emerson Collective, Illumina Ventures and Quan Capital— to develop B cell therapeutics for oncology, rare disease, regenerative medicine, autoimmune disease, and recombinant antibody production (Press release, Walking Fish Therapeutics, SEP 13, 2021, View Source [SID1234587631]).

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Walking Fish Therapeutics has made critical advances in developing a platform to harness B cells’ capability to activate the immune system in the treatment of cancer, and to serve as in vivo protein factories that produce replacement proteins for deficiency diseases, regenerative proteins, and engineered antibodies.

Walking Fish Therapeutics encompasses a powerful executive leadership team, including Co- founder and CEO, Dr. Lewis "Rusty" Williams, who has devoted his career to helping patients by discovering, developing, and commercializing first-in-class therapies for unmet medical needs. He is a former practicing cardiologist, current member of the National Academy of Sciences, co-founder of COR Therapeutics (now part of Takeda) and founder of Five Prime Therapeutics (now part of Amgen). Dr. Williams is joined by distinguished prior investors, board directors, and executives to set the foundation for B cell platform and drug development at Walking Fish Therapeutics.

"In my 35 years of academic and biotech research on protein drugs, the field of B cell therapeutics may be the biggest paradigm shift for human protein therapies," said Dr. Williams. "Walking Fish is harnessing the unique features of B cells to address unmet needs in the treatment for solid tumors, and in non-oncology indications in which B cells can uniquely and durably deliver important proteins with known therapeutic activity."

"B cells play an important role in the rejection of tumors by the immune system," said Dr. Mark Selby, co-founder and VP Immunology of Walking Fish Therapeutics and co-inventor of the immuno-oncology drug OPDIVO and multiple others. "We have learned to engineer B cells that can target certain cancers and provide a treatment modality that is different from that of other T cell or antibody therapies."

"Walking Fish is at the forefront of the burgeoning B cell therapeutics field, rooted in well understood biology, the evolutions of protein therapeutics, and recent progress in cell therapies," said Momo Wu, Ph.D., Investment Manager at Emerson Collective. "We believe these novel therapies will drive a paradigm shift in the treatment of a number of diseases.

"Walking Fish’s financial backing, combined with its high-caliber leadership team, positions it well for success in pioneering B cell engineering," said Alexis Ji, Ph.D., Partner at Illumina Ventures. "We believe the company has the potential to take the protein and cell therapeutics field to a new plane."

"We are encouraged by the tremendous progress Walking Fish has made during the seed funding phase, and we now look forward to the company developing its product candidate in B cell therapeutics, which can be used to treat diseases that are challenging to address with current methods," said Stella Xu, Ph.D., Managing Director at Quan Capital.

OncoHost Publishes Data Identifying Predictive Signature for Response to Immunotherapy in Lung Cancer Patients

On September 13, 2021 OncoHost, a global leader in next-generation precision oncology for improved personalized cancer therapy, reported that it has published a study identifying a predictive proteomic signature for patient response to immune checkpoint inhibitor (ICI) therapy in non-small-cell lung carcinoma (NSCLC) patients (Press release, OncoHost, SEP 13, 2021, View Source [SID1234587646]). The signature was discovered using OncoHost’s first-of-its-kind, AI-based platform, PROphet, and the study will be presented as a poster at the ESMO (Free ESMO Whitepaper) Virtual Congress 2021.

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Immune checkpoint inhibitor (ICI)-based treatment has revolutionized the cancer therapy landscape, displaying a significant response in patients with advanced stage disease. However, only a small fraction of patients actually responds to this treatment, making it critical to identify reliable biomarkers for response and understand the mechanisms underlying resistance. OncoHost’s PROphet platform utilizes advanced machine learning and bioinformatics to analyze the complex tumor-patient-treatment biologic interplay, providing clinicians and their patients with a clinically actionable "disease navigator."

"This study has revealed that OncoHost’s bioinformatic analysis and host response evaluation based on proteomic data can help predict patient outcomes with very high accuracy," said Professor Yuval Shaked, founder and Chief Scientific Advisor at OncoHost, and Professor of Cell Biology and Cancer Science at the Technion – Israel Institute of Technology. "We are proud to be taking such strides in overcoming the challenge of non-responsiveness to immunotherapy and advancing the development of novel therapeutic strategies."

Researchers examined host-mediated effects occurring in response to ICI treatment, and their contribution to therapy resistance in stage IV NSCLC patients. Overall, 108 subjects participated in the study, of whom 80 were responders and 28 were non-responders. The research revealed that plasma proteome changes occur following immunotherapy, suggesting host (patient) response to ICI treatment. An analysis of differentially expressed proteins in the patients indicated therapy-specific mechanisms of resistance.

"Traditional precision medicine searches for biomarkers at the therapy-tumor axis. By adding the patient to the equation, OncoHost is fighting cancer through a distinctive, deep profiling of the patient-tumor interface," said Dr. Ofer Sharon, CEO of OncoHost. "Our research demonstrates the potential clinical utility of PROphet, offering physicians targeted treatment options for their patients and identifying previously unanticipated targets for future interventions and clinical trials."

The clinical study was conducted in collaboration with the Chaim Sheba Medical Center Institute of Oncology, the Technion Institute of Technology, Thomas Jefferson University and the University of Connecticut.

The virtual ESMO (Free ESMO Whitepaper) Congress 2021 will take place September 16-21.

Poster Title: ­A predictive signature for response to immunotherapy in non-small cell lung cancer based on plasma proteomics and clinical parameters.
Abstract #: 300
E-poster #: 74P

The abstract is available on the ESMO (Free ESMO Whitepaper) website here.

Kinnate Biopharma Inc. Presents Preclinical Data on its Lead FGFR Inhibitor Candidate at the JCA-AACR Precision Cancer Medicine International Conference

On September 13, 2021 Kinnate Biopharma Inc. (Nasdaq: KNTE) ("Kinnate"), a biopharmaceutical company focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically defined cancers, reported results from preclinical studies evaluating its lead Fibroblast Growth Factor Receptor (FGFR) inhibitor candidate, KIN-3248 (Press release, Kinnate Biopharma, SEP 13, 2021, View Source [SID1234587665]). These findings were presented during a virtual poster session at the joint JCA-AACR Precision Cancer Medicine International Conference that took place September 10-12, 2021.

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KIN-3248 is a next-generation, irreversible, small molecule pan-FGFR inhibitor designed to target cancer-associated FGFR2 and FGFR3 gene alterations, which are common oncogenic drivers seen in human cancers. KIN-3248 was developed to address both primary FGFR2 and FGFR3 oncogenic alterations and those predicted to drive acquired resistance to current FGFR-targeted therapies, including gatekeeper, molecular brake, and activation loop mutations observed in cancers such as intrahepatic cholangiocarcinoma (ICC) and urothelial carcinoma (UC). Kinnate anticipates filling an Investigational New Drug (IND) application for KIN-3248 with the U.S. Food and Drug Administration (FDA) in the first half of 2022.

"We are very pleased with the progress of our FGFR program, and these positive preclinical data are an important indicator of the potential anti-tumor activity of KIN-3248," said Eric Martin, Ph.D., SVP, Translational Research and Medicine at Kinnate. "In preclinical studies, we have demonstrated inhibitory activity across a wide range of clinically relevant mutations that drive primary disease and acquired resistance. We believe that by addressing these mutations and broadly covering multiple FGFR isoforms, KIN-3248 may be able to overcome challenges associated with currently approved FGFR inhibitors and provide a meaningful increase in the duration of response."

The poster presentation, delivered by Aleksandra Franovic, Ph.D., Senior Director of Translational Medicine at Kinnate, highlights data which show that in biochemical and cellular assays, KIN-3248 exhibited nanomolar potency against all four wild-type FGFR family members but not against other non-FGFR kinases. Importantly, KIN-3248 was active against mutations associated with resistance to FGFR inhibitors both in the clinic and in experimental models, including the FGFR2 and FGFR3 gatekeeper (V565X and V555M, respectively), molecular brake (N550X and N540X, respectively), and activation loop (L618V and K650M, respectively) mutations with less than a five-fold difference in IC50 values relative to corresponding wild-type receptors. In addition, dose-dependent inhibition of FGFR2- and FGFR3-driven human in vivo xenografts, including one with an acquired gatekeeper mutation, was attained with once-daily KIN-3248 treatment and was well tolerated. This efficacy was accompanied by both pharmacodynamic biomarker modulation and downstream pathway inhibition.

Kinnate’s poster presentation, titled "The next-generation FGFR inhibitor, KIN-3248, is active against acquired FGFR2 and FGFR3 gatekeeper and molecular brake drug resistance mutations," is available for on-demand viewing and can be accessed via: View Source