On July 11, 2022 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), reported that its long-standing partner, Roche, will license and advance IONIS-FB-LRx, an investigational antisense medicine, into a Phase 3 clinical study in patients with immunoglobulin A nephropathy (IgAN) (Press release, Ionis Pharmaceuticals, JUL 11, 2022, View Source [SID1234616596]). IgAN is a rare and serious condition that often leads to chronic kidney disease and renal failure. Roche’s decision to advance the program comes after positive data from a Phase 2 clinical study in which IONIS-FB-LRx met its primary endpoint of change in 24-hour urinary protein at 29 weeks compared to baseline.

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In the Phase 2 study (NCT04014335), IONIS-FB-LRx demonstrated a favorable safety and tolerability profile. The study data are consistent with the clinical profile seen across Ionis’ other LICA programs, further validating how advancements in the company’s LIgand-Conjugated Antisense technology platform position Ionis to deliver potentially transformative treatments for a range of unmet medical needs. Data from the Phase 2 study of IONIS-FB-LRx in patients with IgAN has been submitted for presentation at an upcoming medical meeting.

IgAN occurs when too much IgA protein accumulates in the kidneys, causing inflammation and tissue damage, which is the root cause of the disease. IONIS-FB-LRx was designed by Ionis to reduce the production of complement factor B (FB), which is associated with the development of several complement-mediated diseases, including IgAN.

"Roche’s decision to advance the program reaffirms our shared confidence in the ability of Ionis’ antisense medicines to effectively target the root cause of difficult to treat diseases like immunoglobulin A nephropathy," said Michael McCaleb, Ph.D., vice president, clinical development at Ionis. "The results of the Phase 2 study provide initial clinical evidence that IONIS-FB-LRx reduces complement and protein levels in the urine of patients with IgAN."

Roche will lead and be responsible for the Phase 3 study of IONIS-FB-LRx in patients with IgAN and for future global development, regulatory and commercialization activities.

IONIS-FB-LRx is also being evaluated in GOLDEN (NCT03815825), a Phase 2 clinical study to determine whether the medicine can slow or halt the progression of geographic atrophy due to age-related macular degeneration, or AMD. Ionis will receive $55 million from Roche for licensing IONIS-FB-LRx for IgAN and achieving a development milestone in the GOLDEN study.

About IgA Nephropathy (IgAN)
Immunoglobulin A nephropathy (IgAN) is an important cause of chronic kidney disease and renal failure. Also known as Berger’s disease, IgAN is characterized by deposition of IgA and Complement 3 (C3) activation products in the glomerular mesangium of the kidneys, resulting in inflammation and tissue damage. Although IgAN may occur at any age, it generally presents in the second or third decade of life. The clinical presentation, disease progression and histologic findings are highly variable among affected individuals. Current therapies are aimed at reduction of protein levels in the urine with administration of angiotensin inhibitors and control of blood pressure. Sometimes immunosuppressive therapies are given; however, this practice is not universally accepted.

On July 11, 2022 Athenex, Inc., (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, reported that Athenex and certain affiliates have entered into an agreement to sell all of its equity interests in its China subsidiaries, which are primarily engaged in Active Pharmaceutical Ingredient (API) manufacturing operations, to TiHe Capital (Beijing) Co., Ltd. for RMB 124.4 million, or approximately $19.0 million (Press release, Athenex, JUL 11, 2022, View Source [SID1234616595]). Athenex will receive at least 70% of the proceeds at Closing, followed by 20% within three months after the Closing Date, and the remaining balance within six months after the Closing Date. Proceeds from the transaction will be used in part toward repaying existing debt and operating the business. The deal is subject to customary closing conditions, including obtaining certain regulatory approvals in China. Athenex and TiHe also plan to enter into a long-term supply agreement for the manufacture and supply of certain API products.

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"Following the sale of our Dunkirk facility, as well as the sale of our U.S. and European tirbanibulin royalty and milestone interests, the Athenex team continues to execute on our strategy to monetize our non-core assets, bolster our balance sheet, extend our cash runway, and focus on our potential best-in-class NKT cell therapy program," said Dr. Johnson Lau, Chief Executive Officer of Athenex. "We continue to look to identify additional opportunities to deliver on those plans."

On July 11, 2022 Anthos Therapeutics, a clinical-stage biotechnology company developing innovative therapies for cardiovascular and metabolic diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its investigational Factor XI inhibitor, abelacimab, for the treatment of thrombosis associated with cancer (Press release, Anthos Therapeutics, JUL 11, 2022, View Source [SID1234616593]). The company will also be announcing this important milestone today at a session of the ongoing 2022 Congress of the International Society on Thrombosis and Haemostasis (ISTH) Congress in London, UK.

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The Fast Track Designation process is designed to facilitate the development and expedite the review of treatments for serious medical conditions, thereby, addressing unmet medical needs. Drugs that are included in this program may be eligible for more frequent interactions with the FDA to discuss the development path, and if the program criteria are met, eligibility for a potential Rolling Review, Accelerated Approval, and Priority Review.

Venous Thromboembolism (VTE), including both deep vein thrombosis and pulmonary embolism, is the second most prevalent cause of death in patients with cancer, second only to the disease itself.1 However, treatment of Cancer Associated Thrombosis (CAT) can be challenging because the currently available anticoagulants used to treat VTE can have an increased risk of bleeding.2,3

"We believe that abelacimab has the potential to provide patients with cancer associated thrombosis an enhanced safety profile and overall low risk of bleeding, without sacrificing any efficacy of currently available agents. This unmet need is particularly true in patients with gastrointestinal / genitourinary (GI/GU) cancers who are at an even higher risk of bleeding and can be further burdened by the inconvenience of daily injections," said Dan Bloomfield, Chief Medical Officer at Anthos Therapeutics. "Fast track designation by the FDA is a significant milestone for abelacimab and Anthos Therapeutics, but more importantly represents another hopeful step forward for patients. We look forward to working closely with the FDA on our clinical trial program to bring once-monthly abelacimab to patients in need."

"Caring for cancer patients is a delicate and complex process, requiring a fine balance between the risks and benefits of their anticoagulant treatments. Managing thrombosis episodes is of the utmost importance for physicians, patients, and their caregivers, as untreated blood clots or bleeding episodes associated with currently available anticoagulants, can have dire consequences," said Jean Marie Connors, M.D., Associate Professor of Hematology at Harvard Medical School. "The hemostasis sparing potential of FXI inhibitors, such as abelacimab, may represent an important treatment advance in how we manage patients moving forward."

About the Abelacimab Phase 3 Program in Cancer Associated Thrombosis (CAT)
The abelacimab phase 3 CAT program comprises two complementary studies targeting to enroll approximately 2700 patients across 220 sites in more than 20 countries — the largest program of any anticoagulant performed in Cancer-Associated Thrombosis.

ASTER is an international multicenter, randomized, open-label, blinded endpoint evaluation, phase 3 study comparing the effect of abelacimab relative to apixaban on venous thromboembolism (VTE) recurrence and bleeding in patients with cancer associated VTE in whom DOAC treatment is recommended. Abelacimab 150 mg will be administered intravenously (IV) on Day 1 and subcutaneously (SC) monthly thereafter for up to 6 months; Apixaban 10 mg will be administered orally, twice daily (bid) for the first 7 days, followed by 5 mg bid up to 6 months. Enrollment in this trial began in May 2022.

MAGNOLIA is an international multicenter, randomized, open-label, blinded endpoint evaluation, phase 3 study in patients with gastrointestinal (GI) / genitourinary (GU) cancer in whom DOAC treatment is not recommended. The study will compare the effect of abelacimab relative to dalteparin on VTE recurrence and bleeding in patients with cancer associated VTE who are at a high bleeding risk with non-resectable, locally or regionally invasive GI / GU tumors. Abelacimab 150 mg will be administered intravenously (IV) on Day 1 and subcutaneously (SC) monthly thereafter for up to 6 months; dalteparin administered subcutaneously will be given daily, 200 IU/kg/day for the first month, and then 150 IU/kg/day up to 6 months.

About the AZALEA-TIMI 71 Phase 2 Trial
The AZALEA-TIMI 71 trial is an event-driven, randomized, active-controlled, blinded endpoint, parallel-group study to evaluate the effect of two blinded doses of abelacimab relative to open label rivaroxaban on the rate of major or clinically relevant non-major (CRNM) bleeding events in patients with atrial fibrillation (AF) who are at moderate-to-high risk of stroke. The trial completed enrollment in December 2021, with 1287 patients across 95 global study sites including the U.S., Canada, as well as from parts of Europe, and Asia.

About Abelacimab
Abelacimab is a novel, highly selective, fully human monoclonal antibody designed to induce effective hemostasis-sparing anticoagulation through Factor XI inhibition. Abelacimab targets the active domain of Factor XI, demonstrating dual inhibitory activity against both Factor XI and its activated form, Factor XIa. Abelacimab can be administered intravenously (IV) to achieve rapid inhibition of Factor XI activity and then used subcutaneously (SC) monthly to maintain nearly complete inhibition in a chronic setting. In a PK/PD study, abelacimab administered IV provided profound suppression of Factor XI within one hour after the start of therapy and maintained near maximal inhibition for up to 30 days. 4,5 In a Phase 2 study whose results were published in the New England Journal of Medicine in 2021, a single intravenous dose of abelacimab after knee surgery reduced the rate of venous thromboembolism by 80%, measured 10 days after surgery, compared to enoxaparin.4 Factor XI inhibition offers the promise of hemostasis-sparing anticoagulation for the prevention and treatment of arterial and venous thromboembolic events.6 Abelacimab is an investigational agent and has not been approved for any indication.

On July 11, 2022 Singapore-based SCG Cell Therapy Pte Ltd ("SCG") reported that U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for SCG101, an investigational T-cell receptor (TCR) T cell therapy for patients with hepatitis B virus (HBV) related liver cancer (Press release, SCG Cell Therapy, JUL 11, 2022, View Source [SID1234616592]). This clearance initiates the advancement of SCG101 Phase 1/2 clinical trials globally.

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This announcement follows SCG’s previous IND approvals from China National Medical Products Administration (NMPA) in March 2022 and Singapore Health Sciences Authority (HSA) in May 2022. This further establishes SCG101 as the first TCR-T cell therapy product approved for clinical trial across the U.S, China and Singapore.

"Clearance of the IND for SCG’s leading cell therapy product SCG101 across the United States and Asia is an important milestone for the company," said Frank Wang, Chief Executive Officer of SCG Cell Therapy. "We are eager to start international multicenter clinical trial that utilizes our TCR-T technologies to offer innovative treatment option for patients."

SCG101 is an autologous TCR T cell therapy that can recognize HBV-derived T cell epitope presented on the cell surface by specific major histocompatibility complex (MHC) class I molecules. With the specific HLA typing, SCG101 can redirect T cells specifically against the HBV antigen not only to target and eliminate HBsAg-positive HCC cells but also to eradicate HBV cccDNA (covalently closed circular DNA).

"Our previous preclinical and clinical studies demonstrated that SCG101 has dual antiviral and antitumor activities, which is a profoundly important and exciting clinical proof-of-concept for SCG in cancer drug development," stated Ke Zhang, PhD, Chief Scientific Officer of SCG Cell Therapy. "We believe tumour microenvironment is a major barrier to successful cell therapy in solid tumours. Redirecting T cells specifically against HBsAg-positive HCC cells as well as HBV-infected cells could potentially induce immunological cascades altering the tumour microenvironment and offer a path forward to better outcomes for patients."

In June 2022, SCG Cell Therapy presented interim data from an investigator-initiated clinical trial of SCG101 at the International Liver Congress (ILC) 2022, the 57th EASL Annual Meeting. The trial evaluated single-dose SCG101 in patients with advanced HBV-related hepatocellular carcinoma (HCC) who had received at least two prior cancer therapies. The data showed significant antiviral activity and tumour control. Two out of three patients observed lesion shrinkage as well as rapid and significant serum HBsAg reduction from a baseline of 1,004.3 IU/mL and 521.6 IU/ML to 23.8 IU/mL and 9.1 IU/mL in just 28 days from SCG101 treatment and further reduced to 14.0 IU/m and 0.3 IU/mL within 60 days from treatment.

About SCG101

SCG101, an autologous T-cell receptor (TCR) T cell therapy, is an investigational cell therapy product that targets specific epitopes of hepatitis B surface antigen (HBsAg). SCG101 incorporates SCG’s proprietary technology, which allows for redirecting and engineering endogenous T cells using virus-specific TCRs with high sensitivity and avidity selectively against dysfunctional infected and tumour cells. Preclinical studies of SCG101 demonstrated tumour inhibition and HBV cccDNA eradication. In 2022, SCG101 was granted clinical trial approvals by the U.S Food and Drug Administration (FDA), China National Medical Products Administration (NMPA) and Singapore Health Science Authority (HSA) for the potential treatment of HBV-related HCC. The Phase 1/2 clinical trial evaluating SCG101 is underway.

About Liver Cancer

Hepatocellular carcinoma (HCC) is the most common type of liver cancer. It is estimated that more than 905,000 new cases of liver cancer and more than 830,100 deaths from the disease globally in 2020, making it one of the leading causes of cancer deaths around the world [1]. Chronic hepatitis B virus (HBV) infection accounts for at least 50% of cases of HCC worldwide [2]. HCC is typically diagnosed at an advanced stage and is associated with a poor prognosis. The five-year survival rate of less than 15%.

On July 11, 2022 RefleXion Medical, a therapeutic oncology company pioneering the use of biology-guided radiotherapy (BgRT)* for all stages of cancer, reported that researchers will present new data from 14 medical physics investigations validating the efficacy and feasibility of the RefleXion X1 radiotherapy platform with and without BgRT at the 2022 American Association of Physicists in Medicine (AAPM) Annual Meeting in Washington, D.C., July 10-14 (Press release, RefleXion Medical, JUL 11, 2022, View Source [SID1234616590]).

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Two of the 14 abstracts are oral presentations, one of which was selected as "Best in Physics," a distinction granted to only 15 abstracts by the AAPM. The company is showcasing the X1 radiotherapy platform and future BgRT capabilities in booth #5093.

RefleXion is also hosting an industry lunch symposium during the AAPM annual meeting at 12:45 pm EDT today. Titled "Preparing for Biology-guided Radiotherapy in the Clinic," the symposium features a presentation on BgRT and advantages for eliminating challenges with motion management by Murat Surucu, Ph.D., as well as a presentation on BgRT commissioning and workflow by Nataliya Kovalchuk, Ph.D., based on their facility’s experience participating in RefleXion’s Investigational Device Exemption (IDE) clinical study.

"We are extremely encouraged by the growing body of scientific evidence validating the RefleXion X1 platform, the future potential of BgRT, and its impact on workflow and treatment planning," said Sean Shirvani, M.D., chief medical officer of RefleXion. "To have 14 medical physics investigations presented at AAPM, including an oral presentation that earned the distinction ‘Best in Physics,’ further underscores our commitment to achieving and surpassing industry standards of quality radiotherapy treatments for patients."

The following two oral presentations and two poster presentations highlight some of the new research being shared regarding the use of BgRT for treatment planning for various anatomic sites, time savings, and simplification in workflow:

Oral Presentations:

"Investigation of computation time and storage savings using Generative Adversarial Network (GAN) source models for dose simulation of a binary MLC Lina," presented by Bin Han, Ph.D., Sunday, July 10 from 4:00 pm – 5:00 pm EDT. This study investigated the feasibility of Generative Adversarial Network (GAN) in modeling the RefleXion X1 Linac by replacing each collimated beam with a GAN generator for Monte Carlo simulation.
"Evaluation of 89Zr immune-PET for Biology Guided Radiation Therapy," selected "Best in Physics," and presented by Guillem Pratx, Ph.D., Thursday, July 14, from 7:30 am – 8:30 am EDT. This study investigated whether a long-lived PET isotope tagged with antibody (immune-PET) could simplify the BgRT workflow by reducing the multiple tracer injections while improving tumor-specific uptake and guidance.
Poster Presentations:

"One-year quality assurance experience of the first RefleXion system," presented by Murat Surucu, Ph.D., Tuesday, July 12, from 1:15 pm – 1:45 pm EDT. This study reports on the daily, monthly, and annual quality assurance (QA) measurement results of the first clinical BgRT capable machine following the AAPM TG-148 protocol.
"Biology Guided Radiotherapy (BgRT) treatment planning feasibility study for head-and-neck, abdomen, and pelvis," presented by Nataliya Kovalchuk, Ph.D., Tuesday, July 12 from 3:45 pm – 4:15 pm EDT. The purpose of this study was to evaluate the feasibility of BgRT treatment planning for various treatment sites in five cancer patients.