The FDA’s decision is based on data from the key Phase III clinical trial RENOIR (RSV vaccine Efficacy study in Older Adults Immunised against RSV illness) (NCT05035212). RENOIR is a global, randomized, double-blind, placebo-controlled trial that aims to evaluate the efficacy, immunogenicity, and safety of a single dose of the vaccine in persons 60 and older. RENOIR included approximately 37,000 patients who were randomly assigned to either RSVpreF 120 g or placebo in a 1:1 ratio. The findings were published recently in The New England Journal of Medicine. RENOIR is still active, with efficacy data being obtained in the study’s second RSV season.
The Advisory Committee on Immunisation Practises (ACIP) of the Centres for Disease Control and Prevention (CDC) will convene on June 21, 2023, to examine recommendations for the proper use of RSV vaccines in older persons. Pfizer estimates supply availability in Q3 2023, ahead of the expected RSV season this autumn, depending on the outcome of this conference.
FDA Approves First Intravenous Iron Replacement Therapy for Adults With Heart Failure, Iron Deficiency
The FDA has approved ferric carboxymaltose injection to increase exercise capacity in adult patients with heart failure classified as New York Heart Association (NYHA) class 2/3. The New York Heart Association Functional Classification assesses patients’ heart failure depending on the severity of their symptoms. Patients with class 2 or class 3 heart failure have a slight or significant limitation of physical activity due to weariness, palpitation, or dyspnea. The FDA has approved the first and only intravenous (IV) iron replacement medication for this indication.
“This new indication for Injectafer marks the first and only FDA approval of a [IV] iron replacement therapy for adult patients with heart failure, a progressive and chronic disease,” stated Ravi Tayi, MD, MPH, chief medical officer of American Regent, in a press release. “Approximately 2.8 million US adults are impacted by heart failure with iron deficiency or iron deficiency anaemia, which can affect their daily lives and activities.”
Iron deficiency in adults with heart failure; iron deficiency anemia (IDA) in adult and pediatric patients 1 year of age and older who are intolerant to oral iron or have an unsatisfactory response; and adults with IDA who have non-dialysis dependent chronic kidney disease are now among the indications for ferric carboxymaltose injection. It is delivered through IV by a health care practitioner in two doses of 750 mg each, at least seven days apart, to individuals weighing 50 kg or more. Each of the two doses is taken as 15 mg/kg body weight spaced by at least 7 days for individuals weighing less than 50 kg.
Data from the CONFIRM-HF trial, which evaluated the efficacy and safety of ferric carboxymaltose injection in adult patients with chronic heart failure and iron deficiency, were used to support the approval. In iron-deficient individuals with heart failure, treatment with Injectafer dramatically enhanced exercise capacity compared to placebo.
ANeuroTech receives IND approval from the FDA for pivotal Phase IIIB trial of adjunctive anti-depression drug, ANT-01
ANeuroTech, a leader in the development of innovative mental health treatments with minimal or no side effects, today announces that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application to initiate a pivotal Phase IIIB trial of ANT-01 as an adjunctive antidepressant drug for Major Depressive Disorder (MDD). Phase IIIB will include secondary endpoints of improvements in cognitive function and the ability to feel pleasure, the first time these have been included in an anti-depression drug clinical trial. ANeuroTech expects to begin the trial later this year.
Over 60% of individuals suffering from depression and related cognitive difficulties do not benefit from current primary treatment options or encounter difficult side effects. ANeuroTech is working on the development of ANT-01, an additional medication for depression that aims to have minimal or no adverse effects. ANT-01 is a low dosage of an already existing drug called pipamperone dihydrochloride, which is a typical antipsychotic that has been used at higher doses for over four decades in Europe to treat psychotic disorders.
During his clinical practice, Dr. Erik Buntinx, the CEO and Founder of ANeuroTech, made an intriguing observation that ANT-01 possesses a distinctive characteristic of having a strong preference and attachment to the serotonin 5-HT2A and dopamine D4DR receptors in the brain. This finding suggests that ANT-01 holds promise as an additional treatment option with reduced side effects. As part of its development, ANT-01 is being formulated as an orally administered medication that needs to be taken once daily alongside a primary antidepressant.
The pivotal Phase IIIB trial, conducted in a double-blinded manner, will evaluate the effectiveness and safety of a single low dose (15mg) of ANT-01 in combination with a primary antidepressant, compared to a placebo. This trial specifically targets patients with Major Depressive Disorder (MDD) who have not experienced sufficient improvement with selective serotonin reuptake inhibitor (SSRI) or selective noradrenaline serotonin reuptake inhibitor (SNRI) treatments. Building upon positive results obtained from earlier trials, which showcased ANT-01’s antidepressant properties and favorable safety record, the Phase IIIB trial aims to gather further clinical data. Notably, these previous trials demonstrated that ANT-01 enhances the ability to experience pleasure and enhances cognitive function, a unique aspect not observed with any other adjunctive antidepressant compound. The trial aims to enroll over 500 patients across multiple sites in the United States, Europe, and South America, and is scheduled to commence in 2023.
As part of its Series A funding round, ANeuroTech has successfully obtained financial support from KOIS, a global impact investing firm. KOIS specializes in investments related to healthcare and life sciences and is committed to transforming initiatives that have significant societal and environmental impact into viable investment opportunities.
Kyverna Therapeutics Granted FDA Fast Track Designation for KYV-101 in Lupus Nephritis
On June 1, 2023, Kyverna Therapeutics announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its therapy, KYV-101, for treating patients with refractory lupus nephritis (LN). KYV-101 is an autologous version of a novel, fully human clinical-stage anti-CD19 chimeric antigen receptor T-cell (CAR T) construct with properties well suited for use in B cell-driven autoimmune diseases such as lupus nephritis and other B-cell driven autoimmune diseases.
The FDA granting us Fast Track designation for KYV-101 means we can move more quickly toward bringing this potentially transformative and life-saving medicine to patients with lupus. We believe KYV-101 has the potential to drive greater and more rapid reduction of disease activity in patients with LN, and we look forward to sharing clinical data on patients in the second half of 2023.
Peter Maag, Ph.D., chief executive officer (CEO) of Kyverna
Kyverna is actively enrolling patients in the Phase 1 open-label, multi-center clinical trial of KYV-101 at multiple sites throughout the US. In addition, Kyverna submitted its initial Clinical Trial Application (CTA) for a Kyv-101 Phase 1/2 clinical trial in Lupus nephritis to the Paul Ehrlich Institut (PEI) in Germany.
Lupus nephritis (LN) is a severe consequence of systemic lupus erythematosus (SLE), commonly known as lupus. Approximately 40 percent of adults diagnosed with lupus eventually develop Lupus nephritis and 60 percent of Lupus nephritis patients will fail standard of care and approved treatments. Patients with Lupus nephritis may report other symptoms of active SLE (e.g., fatigue, fever, rash, arthritis, serositis, or CNS disease); these are more common with focal (proliferative) and diffuse (proliferative) LN.
As per DelveInsight, in the year 2021, the total diagnosed cases of Lupus Nephritis was 209,000+ in the 7MM which is expected to grow in the coming years. As per the DelveInsight estimates, in 2022, the total diagnosed cases of lupus nephritis were ~210,080 cases in the US. Along with Kyverna Therapeutics, other major players such as AstraZeneca, Novartis, MorphoSys, Roche, Kezar Life Sciences, and others, are eyeing the existing untapped market of Lupus nephritis and trying their best to grab a significant market share. Some of the emerging therapies had already demonstrated positive late-stage results among the LN-affected individuals and are in further clinical evaluation.
Servier Presents Transformational Data from Pivotal Phase 3 INDIGO Trial of Vorasidenib in Recurrent or Residual Grade 2 IDH-Mutant Diffuse Glioma
On June 4, 2023, Servier presented results from the pivotal Phase 3 INDIGO clinical trial investigating vorasidenib, an investigational, oral, selective, highly brain-penetrant dual inhibitor of mutant IDH1/2 enzymes in patients with residual or recurrent isocitrate dehydrogenase 1 or 2 (IDH1/2) mutant low-grade glioma who have been treated with surgery only. The data was released concurrently in the New England Journal of Medicine and presented as a late-breaking abstract during the plenary session of the American Society of Clinical Oncology’s (ASCO) 2023 Annual Meeting.
Grade 2 gliomas are progressive, malignant brain tumors with a poor prognosis, and the current treatment paradigm, which can be associated with short- and long-term toxicities, has not seen progress in more than two decades. For patients living with IDH mutant low-grade glioma, as determined by molecular testing, treatment with a targeted therapy such as vorasidenib has the potential to provide transformative benefits.
Ingo K. Mellinghoff, M.D., Chair, Department of Neurology, Memorial Sloan Kettering Cancer Center
The overwhelmingly positive INDIGO results convincingly demonstrate the impact of targeting IDH mutations early in cancer biology where a monotherapy approach can lead to a profoundly meaningful outcome for patients with recurrent or residual IDH-mutant grade 2 gliomas. IDH mutations are disease defining alterations in IDH-mutant diffuse gliomas and these pivotal data coupled with vorasidenib’s especially high penetration of the blood-brain barrier, offer opportunities to evolve the treatment landscape for patients living with this malignancy
Susan Pandya, M.D., Vice President Clinical Development and Head of Cancer Metabolism Global Development Oncology & Immuno-Oncology, Servier
INDIGO is a registration-enabling Phase 3 global, randomized, double-blinded placebo-controlled study of vorasidenib in patients with residual or recurrent grade 2 glioma with an isocitrate dehydrogenase 1/2 (IDH1/2) mutation who have undergone surgery as their only treatment. IDH1/2 mutations occur in approximately 80% and 4% of grade 2 gliomas, respectively. As per the updates, INDIGO succeeded in meeting its primary endpoint of progression-free survival (PFS) per blinded independent review committee (BIRC) and the key secondary endpoint of time to next intervention (TTNI) at the prespecified second interim analysis. Earlier in March 2023, the U.S. Food & Drug Administration (FDA) granted fast-track status to vorasidenib. Servier is working to determine timelines for submitting an NDA for vorasidenib to the FDA.
Gliomas are the most prevalent type of adult brain tumor, accounting for 78% of malignant brain tumors. According to DelveInsight estimates, the total incident cases of glioma in the 7MM comprised approximately 47,000 cases in 2022 and are projected to increase in the coming years. In 2022, the highest number of incident cases were observed in the United States among the 7MM. Apart from Servier, several other major pharma and biotech companies are developing new therapies for the treatment of Gliomas. The upcoming glioma treatment landscape is poised to see further expansion after the emergence of new classes such as cancer vaccines, protein kinase C beta inhibitors, dendritic cell immunotherapy, cell and gene therapy, and others.
