On February 17, 2022 Bayer reported that Results from the investigational Phase III ARASENS trial have shown that the use of oral androgen receptor inhibitor (ARi) NUBEQA (darolutamide) plus androgen deprivation therapy (ADT) and docetaxel significantly increased overall survival (OS) in patients with metastatic hormone-sensitive prostate cancer (mHSPC) compared to ADT plus docetaxel (Press release, Bayer, FEB 17, 2022, View Source [SID1234608245]). NUBEQA plus ADT and docetaxel significantly reduced the risk of death by 32.5% compared to ADT plus docetaxel (HR=0.68, 95% CI 0.57-0.80; P<0.001). At the data cutoff date for the primary analysis (October 25, 2021), the median treatment duration was longer for NUBEQA plus ADT and docetaxel (41.0 months) versus ADT plus docetaxel (16.7 months).1 NUBEQA is currently indicated for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC).

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These results were presented at the 2022 ASCO (Free ASCO Whitepaper) GU Cancers Symposium and simultaneously published in The New England Journal of Medicine.1

"Metastatic prostate cancer is a uniformly fatal disease and despite progress in recent years, only 30% of these men will survive beyond five years. ARASENS demonstrated that the addition of NUBEQA, an androgen receptor inhibitor, significantly increased overall survival for patients receiving standard androgen deprivation therapy and docetaxel as initial treatment for metastatic hormone-sensitive prostate cancer. NUBEQA also improved time to castration-resistant prostate cancer and other key secondary endpoints," said Matthew Smith, M.D., Ph.D., Director of the Genitourinary Malignancies Program, Massachusetts General Hospital Cancer Center. "These results are an important step forward for the treatment of patients with metastatic hormone-sensitive prostate cancer."

"With the ARASENS results in mHSPC adding to the existing Phase III ARAMIS data in nmCRPC, NUBEQA has now shown positive data in two pivotal clinical trials for patients across these key disease stages in prostate cancer," said Robert LaCaze, Member of the Executive Committee of Bayer’s Pharmaceutical Division and Head of the Oncology SBU at Bayer. "We believe NUBEQA has the potential to become a foundational drug for appropriate prostate cancer patients. We are committed to making this potential new treatment option in mHSPC available to patients and their treating physicians, and therefore are pursuing the fastest possible regulatory timelines."

ARASENS is the only randomized, double-blind pivotal study prospectively designed to compare the use of a second-generation ARi plus ADT and docetaxel to ADT plus docetaxel (a guideline recommended standard-of-care) in mHSPC.2

Detailed results from ARASENS

The significant improvement in OS was observed despite substantially higher use of subsequent systemic antineoplastic therapies (such as abiraterone, enzalutamide, cabazitaxel, docetaxel, radium-223 dichloride, sipuleucel-T, lutetium-177 PSMA, or apalutamide) among patients receiving ADT plus docetaxel who entered follow-up (75.6%) compared with the group who received NUBEQA plus ADT and docetaxel (56.8%).1

NUBEQA plus ADT and docetaxel demonstrated statistically significant benefits across multiple secondary endpoints compared to ADT plus docetaxel, including delaying the time to castration-resistant prostate cancer (CRPC) (HR=0.36, 95% CI 0.30-0.42; P<0.001), time to pain progression (HR=0.79, 95% CI 0.66-0.95; P=0.01), time to first symptomatic skeletal event (SSE) (HR=0.71, 95% CI 0.54-0.94; P=0.02) and time to initiation of subsequent systemic antineoplastic therapy (HR=0.39, 95% CI 0.33-0.46; P<0.001).1

Treatment-emergent adverse events (TEAEs) were similar between treatment arms. The most common TEAEs (≥10%) were highest during the overlapping docetaxel treatment period for both arms and decreased progressively thereafter. The most frequently reported AEs in the treatment arms (NUBEQA plus ADT and docetaxel versus ADT plus docetaxel) were alopecia (40.5% and 40.6%, respectively), neutropenia (39.3% and 38.8%, respectively), fatigue (33.1% and 32.9%, respectively) and anemia (27.8% and 25.1%, respectively). Grade 3 or 4 AEs reported in 66.1% versus 63.5% of patients were mainly due to neutropenia (33.7% versus 34.2%, respectively). Serious AEs occurred in 44.8% versus 42.3% of patients, and TEAEs leading to treatment discontinuation occurred in 13.5% versus 10.6% of patients.1

AEs of special interest in patients treated with AR pathway inhibitors for prostate cancer such as fatigue, falls, fractures, mental impairment and cardiovascular events were similar between study arms.1

About the ARASENS Trial2

The ARASENS trial (NCT02799602) is a randomized, Phase III, multi-center, double-blind, placebo-controlled trial which was prospectively designed to investigate the efficacy and safety of oral NUBEQA, an androgen receptor inhibitor (ARi), plus androgen deprivation therapy (ADT) and the chemotherapy docetaxel in patients with metastatic hormone-sensitive prostate cancer (mHSPC). A total of 1,306 newly diagnosed patients were randomized in a 1:1 ratio to receive 600 mg of NUBEQA twice a day or matching placebo, plus ADT and docetaxel.

The primary endpoint of this trial was overall survival (OS). Secondary endpoints included time to castration-resistant prostate cancer (CRPC), time to pain progression, time to first symptomatic skeletal event (SSE), time to initiation of subsequent anticancer therapy, all measured at 12‐week intervals, as well as adverse events (AEs) as a measure of safety and tolerability.

About NUBEQA (darolutamide)3

NUBEQA is an androgen receptor inhibitor (ARi) with a distinct chemical structure that competitively inhibits androgen binding, AR nuclear translocation, and AR-mediated transcription.3

On July 30, 2019, the FDA approved NUBEQA (darolutamide) based on the ARAMIS trial, a randomized, double-blind, placebo-controlled, multi-center Phase III study, which evaluated the safety and efficacy of oral NUBEQA in patients with non-metastatic castration-resistant prostate cancer (nmCRPC) who were receiving a concomitant gonadotropin-releasing hormone (GnRH) analog or had a bilateral orchiectomy. In the clinical study, 1,509 patients were randomized in a 2:1 ratio to receive 600 mg of NUBEQA orally twice daily or androgen deprivation therapy (ADT) alone. The primary efficacy endpoint was metastasis-free survival (MFS). NUBEQA is also being investigated in further studies across various stages of prostate cancer, including another Phase III trial in metastatic hormone-sensitive prostate cancer (mHSPC) (ARANOTE) as well as a Phase III trial evaluating NUBEQA as an adjuvant treatment for localized prostate cancer with very high risk of recurrence (DASL-HiCaP). Information about these trials can be found at www.clinicaltrials.gov.

Developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company, NUBEQA is currently indicated for the treatment of men with nmCRPC.3 The approvals of NUBEQA in the U.S., European Union (EU), and other global markets have been based on the pivotal Phase III ARAMIS trial data evaluating the efficacy and safety of NUBEQA plus ADT compared to ADT alone.3 Filings in other regions are underway or planned.

INDICATION FOR NUBEQA (darolutamide)

NUBEQA (darolutamide) is an androgen receptor inhibitor indicated for the treatment of patients with non-metastatic castration-resistant prostate cancer.

IMPORTANT SAFETY INFORMATION FOR NUBEQA (darolutamide)

Embryo-Fetal Toxicity: Safety and efficacy of NUBEQA have not been established in females. NUBEQA can cause fetal harm and loss of pregnancy. Advise males with female partners of reproductive potential to use effective contraception during treatment with NUBEQA and for 1 week after the last dose.

Adverse Reactions

Serious adverse reactions occurred in 25% of patients receiving NUBEQA and in 20% of patients receiving placebo. Serious adverse reactions in ≥1% of patients who received NUBEQA were urinary retention, pneumonia, and hematuria. Overall, 3.9% of patients receiving NUBEQA and 3.2% of patients receiving placebo died from adverse reactions, which included death (0.4%), cardiac failure (0.3%), cardiac arrest (0.2%), general physical health deterioration (0.2%), and pulmonary embolism (0.2%) for NUBEQA.

Adverse reactions occurring more frequently in the NUBEQA arm (≥2% over placebo) were fatigue (16% vs 11%), pain in extremity (6% vs 3%) and rash (3% vs 1%).

Clinically significant adverse reactions occurring in ≥2% of patients treated with NUBEQA included ischemic heart disease (4.0% vs 3.4% on placebo) and heart failure (2.1% vs 0.9% on placebo).

Drug Interactions

Effect of Other Drugs on NUBEQA – Combined P-gp and strong or moderate CYP3A4 inducers decrease NUBEQA exposure, which may decrease NUBEQA activity. Avoid concomitant use.

Combined P-gp and strong CYP3A4 inhibitors increase NUBEQA exposure, which may increase the risk of NUBEQA adverse reactions. Monitor more frequently and modify NUBEQA dose as needed.

Effects of NUBEQA on Other Drugs – NUBEQA inhibits breast cancer resistance protein (BCRP) transporter. Concomitant use increases exposure (AUC) and maximal concentration of BCRP substrates, which may increase the risk of BCRP substrate-related toxicities. Avoid concomitant use where possible. If used together, monitor more frequently for adverse reactions, and consider dose reduction of the BCRP substrate.

NUBEQA inhibits OATP1B1 and OATP1B3 transporters. Concomitant use may increase plasma concentrations of OATP1B1 or OATP1B3 substrates. Monitor more frequently for adverse reactions and consider dose reduction of these substrates.

Review the prescribing information of drugs that are BCRP, OATP1B1, and OATP1B3 substrates when used concomitantly with NUBEQA.

For important risk and use information about NUBEQA, please see the accompanying full Prescribing Information.

About Metastatic Hormone-Sensitive Prostate Cancer

Prostate cancer is the second most commonly diagnosed malignancy in men worldwide. In 2020, an estimated 1.4 million men were diagnosed with prostate cancer, and about 375,000 died from the disease worldwide.4

At the time of diagnosis, most men have localized prostate cancer, meaning their cancer is confined to the prostate gland and can be treated with curative surgery or radiotherapy.5,6 Upon relapse when the disease will metastasize or spread, androgen deprivation therapy (ADT) is the cornerstone of treatment for this hormone-sensitive disease. Approximately 5% of men will already suffer from prostate cancer with distant metastases when first diagnosed. Current treatment options for men with metastatic hormone-sensitive prostate cancer (mHSPC) include hormone therapy, such as ADT, androgen receptor pathway inhibitors plus ADT or a combination of the chemotherapy docetaxel and ADT. Despite these treatments, most men with mHSPC will eventually progress to metastatic castration-resistant prostate cancer (mCRPC), a condition with limited survival.7,8

About Oncology at Bayer

Bayer is committed to delivering science for a better life by advancing a portfolio of innovative treatments. The oncology franchise at Bayer includes six marketed products and several other assets in various stages of clinical development. Together, these products reflect the company’s approach to research, which prioritizes targets and pathways with the potential to impact the way that cancer is treated.

On February 17, 2022 Immune-Onc Therapeutics, Inc. ("Immune-Onc"), a clinical-stage cancer immunotherapy company developing novel biotherapeutics targeting immunosuppressive myeloid checkpoints, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for IO-202, a first-in-class myeloid checkpoint inhibitor targeting leukocyte immunoglobulin-like receptor B4 (LILRB4, also known as ILT3) for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) (Press release, Immune-Onc Therapeutics, FEB 17, 2022, View Source [SID1234608244]). The Company received Orphan Drug Designation for IO-202 for the treatment of AML in 2020.

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"We are pleased that the FDA has granted IO-202 Fast Track designation in recognition of its potential to improve outcomes for people with relapsed or refractory AML," said Paul Woodard, Ph.D., chief medical officer of Immune-Onc. "We look forward to working closely with the FDA to accelerate the clinical development of IO-202, which is currently being evaluated as a monotherapy and in combination with other agents in a Phase 1 dose escalation and expansion trial in patients with AML with monocytic differentiation and in chronic myelomonocytic leukemia (CMML)."

The FDA’s Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need to get important new medicines to patients earlier. Drugs that receive Fast Track designation may be eligible for more frequent interactions and written communications with the FDA to discuss the development plan and data collection to support an approval pathway. The designation also supports the eligibility for Accelerated Approval and Priority Review if relevant criteria are met.

ABOUT LILRB4 (also known as ILT3)

LILRB4, also known as ILT3, is an immune inhibitory transmembrane protein found on monocytic myeloid cells, including dendritic cells. LILRB4 inhibits antigen-presenting cell activation, resulting in immune tolerance. LILRB4 is also expressed on certain hematologic cancer cells and monocytic myeloid cells in the solid tumor microenvironment. Immune-Onc and The University of Texas published pioneering research in Nature illuminating the role of LILRB4 in immune suppression and tumor infiltration in AML and presented the rationale for targeting LILRB4 in solid tumors at the AACR (Free AACR Whitepaper) Annual Meeting 2021.

ABOUT IO-202

IO-202 is a first-in-class LILRB4 antagonist antibody with broad potential as an immunotherapy in both blood cancers and solid tumors. In hematologic malignancies, preclinical studies showed that IO-202 converts a "don’t kill me" to a "kill me" signal by activating T cell killing and converts a "don’t find me" to a "find me" signal by inhibiting infiltration of blood cancer cells. In solid tumors, preclinical data showed that IO-202 enhances dendritic cell function and T cell activation in vitro and inhibits tumor growth in a solid tumor model in vivo.

IO-202 is currently in Phase 1 clinical development for the treatment of AML and CMML. The U.S. Food and Drug Administration granted IO-202 Orphan Drug Designations for treatment of AML in 2020. The company has received IND clearance to evaluate IO-202 in solid tumors in January 2022.

On February 17, 2022 BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines, reported that BeiGene’s BTK inhibitor BRUKINSA (zanubrutinib) received approval from Swissmedic for the treatment of adult patients with Waldenström’s macroglobulinemia (WM) who have received at least one prior line of therapy, or for treatment-naïve patients who are not suited for standard chemo-immunotherapy (Press release, BeiGene, FEB 17, 2022, View Source [SID1234608243]). BRUKINSA had previously been granted orphan drug status.

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"The authorization of BRUKINSA will bring a new option and an innovative medicine that has potential to offer deep and durable response for eligible patients with WM in Switzerland," said Pr. Davide Rossi, Deputy Head of the Division of Hematology of the Oncology Institute of Southern Switzerland IOSI. "BRUKINSA is a next-generation BTK inhibitor which has also provided meaningful improvements in tolerability for some patients with WM compared to ibrutinib, as treatment discontinuation remains a concern."

Reto Kessler, Country Manager, Switzerland at BeiGene added, "This approval is a significant development for people living with WM in Switzerland and for BeiGene’s expansion in Europe. Our teams are committed to collaborating with the Federal Office of Public Health and healthcare professionals to ensure access to BRUKINSA for patients in Switzerland."

The Marketing Authorization Application (MAA) is supported by data from the global Phase 3 ASPEN clinical trial, a Phase 3 randomized, open-label, multicenter trial (NCT03053440) that evaluated BRUKINSA compared to ibrutinib in patients with relapsed/refractory (R/R) or treatment-naïve (TN) WM who harbor a MYD88 mutation (MYD88MUT). In the ASPEN trial, BRUKINSA demonstrated a numerically higher very good partial response (VGPR) rate and a favorable safety profile over ibrutinib, although the primary endpoint of statistical superiority related to deep response (VGPR or better) was not met. As assessed by independent review committee (IRC) per adaptation of the response criteria updated at the Sixth International Workshop on Waldenström’s Macroglobulinemia (IWWM), the combined complete response (CR) + VGPR rate in the overall intention-to-treat (ITT) population was 29% with BRUKINSA (95% CI: 20, 40), compared to 19% with ibrutinib (95% CI: 12, 30).

In the ASPEN trial, of the 101 patients with WM randomized and treated with BRUKINSA, four percent of patients discontinued due to adverse events, including cardiomegaly, neutropenia, plasma cell myeloma, and subdural hemorrhage. Adverse events leading to dose reduction occurred in 14% of patients, with the most common being neutropenia (3%) and diarrhea (2%).

The recommended dose of BRUKINSA is either 160 mg twice daily or 320 mg once daily, taken orally with or without food. The dose may be adjusted for adverse reactions and reduced for patients with severe hepatic impairment and certain drug interactions.

About Waldenström’s Macroglobulinemia

WM is a rare B-cell lymphoma that occurs in less than two percent of patients with non-Hodgkin lymphomas.2 The disease usually affects older adults and is primarily found in bone marrow, although lymph nodes and the spleen may be involved.1 Throughout Europe, the estimated incidence rate of WM is approximately seven for every one million men and four for every one million women.2

About BRUKINSA

BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.

BRUKINSA is supported by a broad clinical program which includes more than 3,900 subjects in 35 trials across 28 markets. To date, BRUKINSA has received more than 20 approvals covering more than 40 countries and regions, including the U.S., European Union, China, Australia, Great Britain and Switzerland. Currently, more than 40 additional regulatory submissions are in review around the world.

BeiGene Oncology

BeiGene is committed to advancing best- and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines for patients across the globe. We have a growing R&D and medical affairs team of approximately 2,900 colleagues dedicated to advancing more than 100 clinical trials that have involved more than 14,500 subjects. Our expansive portfolio is directed predominantly by our internal colleagues supporting clinical trials in more than 45 countries and regions. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in our research and development. BeiGene currently has three approved medicines discovered and developed in our own labs: BTK inhibitor BRUKINSA in the United States, China, the EU and U.K., Canada, Australia and additional international markets; and the non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab as well as the PARP inhibitor pamiparib in China.

BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen, Bristol Myers Squibb, EUSA Pharma and Bio-Thera. We also plan to address greater areas of unmet need globally through our other collaborations including with Mirati Therapeutics, Seagen, and Zymeworks.

In January 2021 BeiGene and Novartis announced a collaboration granting Novartis rights to co-develop, manufacture, and commercialize BeiGene’s anti-PD1 antibody tislelizumab in North America, Europe, and Japan. Building upon this productive collaboration, including a biologics license application (BLA) under FDA review, BeiGene and Novartis announced an option, collaboration and license agreement in December 2021 for BeiGene’s TIGIT inhibitor ociperlimab that is in Phase 3 development. Novartis and BeiGene also entered into a strategic commercial agreement through which BeiGene will promote five approved Novartis Oncology products across designated regions of China.

On February 17, 2022 NEC OncoImmunity AS (NOI) reported a publication that describes a novel approach to type new HLA alleles and characterize the HLA of tumors to guide personalized cancer immunotherapy (Press release, NEC OncoImmunity, FEB 17, 2022, View Source [SID1234608242]).

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The computational approach performs HLA typing from next generation sequencing data by using the current strengths of conventional HLA typing while simultaneously allowing for the discovery of novel HLA alleles and tumor-specific HLA variants, through leveraging proprietary mutation calling bioinformatics at NOI.

NOI collaborated with Ultimovacs ASA in the study to validate the HLA typing approach from the blood of numerous donors using deep targeted HLA sequencing. The validation confirmed almost 100% of HLA typing success at the 1st and 2nd fields of resolution.

The approach was published in the peer review journal HLA where the first author Anzar and her colleagues also demonstrated how the approach led to the discovery of a new officially named HLA allele, from standard sequencing data routinely used in the design of personalized cancer vaccines.

"This approach is an important development in the field of precision immunotherapy. It will help the HLA typing community to fill gaps in the global human HLA libraries, and help identify novel HLA variants in disease association studies in addition to improving the accuracy of HLA-typing for organ transplantation and vaccine design. We will now use this unique addition to our bioinformatics toolbox to further characterize the relationship between variation in HLA alleles and response to cancer immunotherapy." Said, Dr. Trevor Clancy, Chief Scientific Officer at NEC OncoImmunity.

References

Title: Personalized HLA typing leads to the discovery of novel HLA alleles and tumor-specific HLA variants

Authors: Irantzu Anzar, Angelina Sverchkova, Pubudu Samarakoon, Espen Basmo Ellingsen, Gustav Gaudernack, Richard Stratford and Trevor Clancy

URL: View Source

On February 17, 2022 Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a specialty pharmaceutical company focused on rare and orphan diseases, reported that its wholly-owned subsidiary, Quoin Pharmaceuticals, Inc., has entered into an exclusive Distribution Agreement with Neopharm Medical Supplies, an exclusive distributor for leading medical and pharma manufacturers in Israel, for QRX003, the Company’s investigational treatment for Netherton Syndrome, a rare and devastating genetic disease for which there is currently no available treatment or cure (Press release, NeoPharm, FEB 17, 2022, View Source [SID1234608241]).

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Under the terms of the revenue sharing agreement, Neopharm gains exclusive rights to commercialize QRX003 in Israel. Quoin will be the exclusive supplier of QRX003 to Neopharm.

Dr. Michael Myers, Chief Executive Officer of Quoin, commented, "This is Quoin’s sixth distribution agreement in the last four months for QRX003 and we’re excited to add Neopharm, who is a leading distributor in Israel. We now have 54 countries covered by our partnership agreements for QRX003, encompassing many regions of the globe. Establishing a broad distribution network is a key part of our commitment to ensure that every patient, everywhere will have access to this product once it has been approved for this devastating disease."

About Netherton Syndrome

Netherton Syndrome, a form of Ichthyosis, is a rare, hereditary skin disorder caused by a mutation in the SPINK5 gene (serine protease inhibitor, Kazal Type 5) that leads to severe skin barrier defects and recurring infections, as well as a pronounced predisposition to allergies, asthma, and eczema. Patients also often suffer from severe dehydration, chronic skin inflammation and stunted growth.

Currently, there is no cure for Netherton Syndrome, nor are there any approved therapeutic treatments.