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Jul 11, 2023
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Astellas Pharma Inc. announced that the FDA has accepted and granted Priority Review for the company’s Biologics Licence Application (BLA) for zolbetuximab, a first-in-class investigational Claudin 18.2 (CLDN18.2)-targeted monoclonal antibody, for first-line treatment of patients with CLDN18.2-positive HER2-negative gastric or GEJ adenocarcinoma. If approved, zolbetuximab would be the first CLDN18.2-targeted treatment for these patients in the United States.
The FDA has set a January 12, 2024 action date under the Prescription Drug User Fee Act (PDUFA). The FDA assessed the application as part of its Real-Time Oncology Review (RTOR) program, which aims to investigate a more efficient review procedure in order to guarantee that safe and effective therapies are provided to patients as soon as feasible.
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In the United States, it is anticipated that 26,500 people will be diagnosed with stomach cancer in 2023, with 11,130 dying from the condition. Early-stage gastric cancer symptoms typically overlap with more common stomach-related illnesses, frequently resulting in a gastric cancer diagnosis at the advanced or metastatic stage, or after the tumor has spread to other body tissues or organs. Patients with metastatic disease have a five-year relative survival rate of 6.6%.
“Astellas is dedicated to bringing innovative therapies to patients suffering from difficult-to-treat cancers, such as gastric cancer.” While rare in the United States, stomach cancer can be fatal if detected late,” said Moitreyee Chatterjee-Kishore, PhD, MBA, Senior Vice President and Head of Immuno-Oncology Development at Astellas. “The FDA’s acceptance of the Biologics Licence Application filing and Priority Review designation for zolbetuximab confirms the urgent therapeutic need and brings us one step closer to delivering on this commitment to patients, families, and carers.”
The BLA is based on the findings of the Phase III clinical studies SPOTLIGHT and GLOW. The SPOTLIGHT study compared zolbetuximab plus mFOLFOX6 (an oxaliplatin, leucovorin, and fluorouracil combination regimen) against placebo plus mFOLFOX6. The GLOW trial compared zolbetuximab plus CAPOX (a capecitabine and oxaliplatin-based chemotherapy treatment) versus placebo plus CAPOX. According to a validated immunohistochemical assay, roughly 38% of patients screened for SPOTLIGHT and GLOW had CLDN18.2-positive tumours (75% of tumour cells with moderate-to-strong membranous CLDN18 staining intensity). Astellas has already accounted for the impact of this acceptance in its financial forecast for the current fiscal year, which ends on March 31, 2024.
Eisai Co., Ltd. and Biogen Inc. announced that the FDA had approved the supplemental Biologics Licence Application (sBLA) supporting the traditional approval of LEQEMBI® (lecanemab-irmb) 100 mg/mL injection for intravenous use, making LEQEMBI the first and only approved Alzheimer’s disease treatment shown to slow cognitive and functional decline in adults with Alzheimer’s disease (AD). LEQEMBI demonstrated clinically meaningful slowing of cognitive and functional decline in a patient group that was generalizable to Medicare beneficiaries in the United States. This patient group included a mix of racial and ethnic groups, patients with common comorbid conditions, concomitant medications, and patients with mild cognitive impairment (MCI) due to AD or mild AD. Treatment with LEQEMBI should begin in patients with MCI or mild dementia stages of disease (together referred to as early Alzheimer’s disease), the population in which treatment was started in clinical trials.
“Today, the FDA approved LEQEMBI through the traditional approval pathway, making it the first and only approved anti-amyloid Alzheimer’s disease treatment shown to slow cognitive impairment in the early and mild dementia stages of the disease.” We are proud that the results of Eisai’s AD research over the past 40 years have been recognized and delivered to people living with this disease in the United States as a research and development-focused company based on our hhc (human health care) concept,” said Haruo Naito, Eisai’s Chief Executive Officer. “Alzheimer’s disease is a progressive, fatal disease that has a significant impact not only on the people who have it, but also on their loved ones, carers, and society.” We will continue to try to provide patients with easy access to LEQEMBI and to encourage early disease detection and treatment. Eisai will strive tirelessly to educate physicians on the safe and appropriate use of LEQEMBI in order to maximise its usefulness to persons living with early Alzheimer’s disease and their families.”
The conventional approval of LEQEMBI is based on Phase III data from Eisai’s large, global Clarity AD clinical trial, in which LEQEMBI met its primary endpoint and all critical secondary endpoints with statistically significant findings, confirming LEQEMBI’s clinical benefit. The primary outcome was the Clinical Dementia Rating Sum of Boxes (CDR-SB), a global cognitive and functional scale. When compared to placebo, LEQEMBI therapy reduced clinical decline on CDR-SB by 27% after 18 months. Furthermore, the secondary objective of the AD Cooperative Study-Activities of Daily Living Scale for Mild Cognitive Impairment (ADCS MCI-ADL), as judged by carers for patients with AD, revealed a 37% statistically significant benefit. This assesses patients’ ability to function independently, such as dressing, feeding themselves, and participating in community activities. On November 29, 2022, the full findings of the Clarity AD trial were presented at the Clinical Trials on Alzheimer’s Disease (CTAD) 2022 conference and simultaneously published in the peer-reviewed medical publication The New England Publication of Medicine.
Iovance Biotherapeutics, a biotechnology company dedicated to pioneering and advancing new therapies using polyclonal tumor infiltrating lymphocytes (TILs) for cancer patients, has shared encouraging updates regarding its Phase 2 IOV-LUN-202 trial. This trial aims to evaluate the efficacy of TIL therapy in patients with non-small cell lung cancer (NSCLC) who have previously received anti-PD-1 treatment. The company announced positive regulatory and clinical advancements concerning this pivotal Phase 2 trial.
NSCLC Regulatory and Clinical Update
Iovance Biotherapeutics recently had a productive meeting with the U.S. Food and Drug Administration (FDA), referred to as a Type B Pre-Phase 3 meeting. During this meeting, the FDA provided positive feedback on the regulatory aspects of the IOV-LUN-202 trial. The FDA indicated that the trial’s design may be acceptable for accelerated approval of LN-145 TIL therapy. This therapy is intended for patients with advanced non-small cell lung cancer (NSCLC) who have experienced disease progression after receiving chemotherapy and anti-PD-1 therapy, and who do not have certain genetic mutations (EGFR, ROS, or ALK). Additionally, the patients should have received targeted therapy if there were other actionable mutations detected. Building on this feedback, Iovance conducted a preliminary analysis of the IOV-LUN-202 trial, which included 23 NSCLC patients treated with LN-145.
The analysis revealed an objective response rate (ORR) of 26.1% according to the RECIST v1.1 criteria, with six patients showing a response (one complete response and five partial responses). The disease control rate was reported as 82.6%. Although it is still early in the study, the median duration of response (DOR) has not been reached, and the range of DOR observed so far varied from 1.4+ months to 9.7+ months. The adverse events observed during treatment were consistent with the underlying disease and the known adverse event profiles associated with non-myeloablative lymphodepletion and interleukin-2.
Following the regulatory discussions, Iovance plans to enroll approximately 120 patients in the registrational IOV-LUN-202 trial, with enrollment expected to be completed in the second half of 2024. Additionally, Iovance is preparing for a meeting with the FDA this year to discuss a randomized confirmatory trial involving LN-145 in frontline advanced NSCLC patients. This confirmatory trial in frontline advanced NSCLC is expected to be underway by the time potential approval is granted for advanced post-anti-PD-1 NSCLC, as previously announced.
Iovance’s submission of the Biologics License Application (BLA) for lifileucel, intended for the treatment of advanced melanoma, is proceeding smoothly with the FDA’s Priority Review. The review process is on track, and there have been no significant delays. The target action date for the BLA, as per the Prescription Drug User Fee Act, is set for November 25, 2023.
Biophytis, a biotechnology company focused on developing therapies to slow down age-related degeneration and improve functional outcomes in patients with age-related diseases, including respiratory failure in COVID-19 patients, has announced that it has submitted an application to the Food and Drug Administration (FDA) portal for the approval of its SARA-31 program in the United States. This marks the initiation of the first-ever Phase 3 study in sarcopenia, a condition characterized by muscle loss associated with aging.
The decision to launch the Phase 3 program follows promising results from the SARA-INT Phase 2b study and discussions with health authorities in 2022. Based on the positive outcomes of the previous study and feedback received from the FDA, Biophytis is commencing its Phase 3 program by submitting the first-ever Phase 3 application (SARA-31) for sarcopenia to the FDA. Furthermore, the company has also recently submitted the program to the European Medicines Agency.
The objective of the Phase 3 study, SARA-31, is to evaluate the efficacy and safety of Sarconeos (BIO101) in treating sarcopenic patients at risk of mobility disability. The study will include approximately 900 patients aged over 65 years with severe sarcopenia and will assess their ability to walk 400 meters in less than 15 minutes, among other secondary endpoints such as walking speed, handgrip strength, and patient-reported quality of life. The patients will receive either a placebo or a dosage of 350mg of Sarconeos (BIO101) twice daily for a duration of 12 to 36 months.
Biophytis expects to receive a response from regulatory authorities in the third quarter of 2023, which would allow them to initiate the study in the United States. The principal investigator for the study will be Roger A. Fielding, PhD, who heads the Nutrition, Physiology, Exercise, and Sarcopenia (NEPS) Laboratory at Tufts University in Boston.
Stanislas Veillet, Chairman and CEO of Biophytis, expressed the significance of this milestone for the company, highlighting their pioneering role in the field of sarcopenia. They aim to be the first company, in collaboration with global or regional pharmaceutical companies, to launch a Phase 3 clinical development program for sarcopenia.
On July 10, 2023, Marker Therapeutics, Inc. (Nasdaq: MRKR) announced that the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has granted Orphan Drug Designation to its therapy zedenoleucel, a multi-tumor-associated antigen (multiTAA)-specific T cell product candidate, MT-401, for the treatment of patients with Acute Myeloid Leukemia (AML). The orphan drug designation will aid Marker Therapeutics in its ongoing development of MT-401 to close a sizable gap in the management of Acute Myeloid Leukemia and offers Marker Therapeutics a variety of possible advantages.
The multi-tumor associated antigen (multiTAA)-specific T cell platform from Marker Therapeutics is a unique, non-genetically modified cell treatment strategy that expands tumor-specific T cells from the patient or donor blood that are capable of recognizing a variety of tumor antigens. More than 180 patients with diverse hematological malignancies and solid tumors participated in clinical trials that revealed autologous and allogenic multiTAA-specific T-cell products to be well tolerated, exhibit persistent therapeutic responses, and spread epitope consistently. The latter can potentially contribute to a long-lasting antitumor impact and is often not seen with other T-cell therapy. MultiTAA-specific T cells are intended to be provided in an outpatient context, in contrast to other cell treatments that demand hospitalization and intensive monitoring.
The orphan drug designation for MT-401 by the EMA is a significant regulatory milestone. It acknowledges not just the potential therapeutic impact of MT-401, but also the urgent need to deliver innovative treatment options to patients living with Acute Myeloid Leukemia. In 2020, MT-401 was also granted orphan designation by the U.S. Food and Drug Administration for the treatment of patients with Acute Myeloid Leukemia. We are deeply committed to working with regulatory authorities to expedite the drug development and approval process.
Nadia Agopyan, Ph.D., RAC, Senior Vice President, Regulatory Affairs of Marker Therapeutics
We are extremely proud to have been granted Orphan Drug Designation by the EMA for MT-401. In our Phase 2 clinical trial of patients with post-transplant Acute Myeloid Leukemia, we have observed promising results from patients with measurable residual disease, suggesting that the unique and differentiated targeting technology of MT-401 can be a potential treatment for patients with Acute Myeloid Leukemia before relapse.
Juan F. Vera, M.D., President and Chief Executive Officer of Marker Therapeutics
In order to reduce tumor escape, MT-401 employs a unique, non-genetically modified technique that recognizes a variety of antigens expressed by tumor cells. For the treatment of recurrent Acute Myeloid Leukemia after allogeneic HSCT, MT-401 is now being investigated in a Phase 2 clinical trial. It was created to selectively target four distinct antigens that are elevated in Acute Myeloid Leukemia but have minimal expression in normal cells.
Acute Myeloid Leukemia is a type of cancer that affects the bone marrow and blood cells. Acute Myeloid Leukemia is one of the most common types of leukemia in adults. It is characterized by the rapid growth and accumulation of abnormal myeloid cells, which are immature white blood cells that normally develop into various types of mature blood cells. Acute Myeloid Leukemia is fairly rare overall, accounting for only about 1% of all cancers. The ongoing intense clinical and commercial developments anticipated high hope for better Acute Myeloid Leukemia treatment outlook in the coming years.
On July 07, 2023, Axsome Therapeutics, Inc. (NASDAQ: AXSM) announced that it has dosed the first patient in the FOCUS Phase 3 trial of solriamfetol, an investigational treatment for Attention Deficit Hyperactivity Disorder (ADHD) in adults. Solriamfetol is a dopamine and norepinephrine reuptake inhibitor. In vitro studies have also shown that solriamfetol has agonist activity at trace amine-associated receptor 1 (TAAR1) and 5HT1a receptors. Solriamfetol is not approved by the FDA for the treatment of ADHD.
FOCUS (Forward Treatment of Attention Deficit and Hyperactivity Using Solriamfetol) is a Phase 3, randomized, double-blind, placebo-controlled, multicenter trial to assess the efficacy and safety of solriamfetol for the treatment of ADHD in adults. About 450 patients will be randomized in a 1:1:1 ratio to receive solriamfetol (150 mg or 300 mg) or placebo for 6 weeks. The primary endpoint will be a change in the Adult ADHD Investigator Symptom Report Scale (AISRS).
Attention Deficit Hyperactivity Disorder (ADHD) is a neurodevelopmental disorder that primarily affects children, but can persist into adulthood. It is characterized by a persistent pattern of inattention, hyperactivity, and impulsivity that interferes with daily functioning and development. ADHD is thought to impact 5% of children and adolescents in the United States and 11.4 million adults. At least two-thirds of children with ADHD continue to have symptoms and difficulties as adults.
As per Moffitt et al. (2007), the North American prevalence rate (6.2%) only slightly exceeded the European prevalence rate (4.6%) for ADHD. The economic burden of ADHD is substantial. Treating ADHD involves various costs, including diagnosis, therapy, medication, and educational support. Additionally, individuals with ADHD may face difficulties in obtaining and maintaining employment, leading to financial strain and dependence on social welfare systems. Several major pharma and biotech companies including Axsome Therapeutics, among others, are actively working in the ADHD therapeutics market. The anticipated approval and launch of emerging therapies are expected to immensely improve the treatment dynamics in the coming years.
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