XTalks

SEPTEMBER 14, 2022

In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.

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Life Science FDA Approval Insulin Drugs 90

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. These companies are at various stages of research and clinical trials.

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XTalks

APRIL 21, 2023

With just under a week left before Theranos founder Elizabeth Holmes is set to begin her 11-year prison sentence, the former CEO has appealed her conviction, calling it ‘unjust,’ asking for a new trial or a reduced prison term. Holmes founded healthcare startup Theranos in 2003.

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XTalks

AUGUST 23, 2023

BriaCell is developing cell therapies for the treatment of advanced breast cancer and has promising compounds, including Bria-IMT, that are currently being evaluated in clinical trials. BriaCell recently received FDA approval for its Bria-IMT Combination pivotal study design in advanced metastatic breast cancer.

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Life Science Clinical Trials Clinical Trials FDA Approval 98

XTalks

APRIL 9, 2025

The FDAs accelerated approval of Vanrafia was based on an interim analysis of the global Phase III ALIGN trial, in which atrasentan added to optimized reninangiotensin system inhibitor therapy achieved a 36.1% reduction in urine proteintocreatinine ratio versus placebo (p<0.0001).

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FDA Approval Clinical Trials Clinical Trials Trials 59

XTalks

FEBRUARY 4, 2025

Current Share Price: The announcement follows a $115 million Series D financing in December 2024, co-led by Frazier Life Sciences and Deep Track Capital, with participation from Janus Henderson Investors and Logos Capital. The Phase I trial showed promising safety and pharmacokinetics. The offering, which includes 8.75

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Clinical Trials Clinical Trials Trials Genetics 66

XTalks

FEBRUARY 19, 2025

In the MOTION study a double-blind , multicenter, randomized (2:1) trial patients with measurable TGCT received either vimseltinib at a recommended dose of 30 mg orally twice weekly (with a minimum 72-hour interval between doses) or placebo for 24 weeks. Patients also reported improvements in pain, physical functioning and range of motion.

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FDA Approval Drugs Trials Clinical Trials 59

XTalks

JANUARY 21, 2025

The approval makes Datroway AstraZenecas eighth new medicine since 2020, the first US approval for Daiichi Sankyo and AstraZenecas TROP2-directed ADC and the second based on their proprietary DXd technology. The FDA approval sent Daiichi Sankyos shares soaring by 8.7% to 4,437 yen, reflecting investor confidence.

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Antibody FDA Approval HR Trials 105

XTalks

JANUARY 6, 2025

The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.

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XTalks

APRIL 15, 2025

Soleno Therapeutics has announced the FDA approval of Vykat XR (diazoxide choline extended-release tablets) for the treatment of hyperphagia an intense, persistent hunger in patients with Prader-Willi syndrome (PWS). The approval marks the first FDA-approved therapy specifically targeting hyperphagia in this rare genetic disorder.

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FDA Approval Clinical Trials Clinical Trials Trials 59

XTalks

FEBRUARY 6, 2023

GlaxoSmithKline LLC (GSK) recently announced that the US Food and Drug Administration (FDA) approved Jesduvroq (daprodustat), a new once-a-day oral treatment for anemia due to chronic kidney disease (CKD). The approval is currently only for adult patients who have been undergoing dialysis for at least four months.

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FDA Approval Clinical Trials Clinical Trials Trials 97

XTalks

JANUARY 16, 2025

Sling Therapeutics has positive topline results from its Phase IIb/III LIDS clinical trial evaluating its oral small-molecule therapy linsitinib for the treatment of thyroid eye disease (TED). Ninety patients in the trial were randomized to receive one of two doses of linsitinib or placebo.

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Trials Clinical Trials Clinical Trials Insulin 59

XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. Safety and Efficacy of Filsuvez The FDA’s approval of Filsuvez is supported by compelling clinical evidence. Specifically, 41.3 percent observed in the control gel arm. percent of cases.

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FDA Approval Gene Therapy Gene Trials 111

XTalks

DECEMBER 9, 2024

Despite its potential to slow cognitive decline, the delays highlight the challenges of timely drug approval for life-threatening conditions. The draft builds on prior FDA policies but introduces new provisions from the Consolidated Appropriations Act, 2023.

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XTalks

MARCH 3, 2022

Developed in partnership with Legend Biotech Corp of China, Johnson & Johnson’s cell therapy Carvykti (formerly known as cilta-cel) received approval from the US Food and Drug Administration (FDA) this week for the treatment of multiple myeloma. Live and On-Demand: March 16, 2022 | 9am EDT (NA) / 1pm GMT (UK).

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FDA Approval Life Science Production Trials 98

XTalks

NOVEMBER 27, 2024

Syndax Pharmaceuticals’ Revuforj (revumenib) has won US Food and Drug Administration (FDA) approval for the treatment of relapsed or refractory (R/R) acute leukemia in patients aged one year and older who have a lysine methyltransferase 2A ( KMT2A, formerly MLL ) gene translocation. times its initial investment.

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FDA Approval Drugs Gene Trials 99

XTalks

OCTOBER 16, 2024

Hympavzi’s approval, with its innovative approach to balancing clotting, could offer a modern solution that adds to the global effort to reduce the risk of thrombosis. This is Pfizer’s second FDA-approved treatment for a rare genetic blood disorder this year.

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FDA Approval Trials Clinical Trials Clinical Trials 105

XTalks

DECEMBER 10, 2021

Biohaven Pharmaceuticals revealed positive top line results from a late-stage trial which show that its migraine medication Zavegepant provides “ultra-rapid” pain relief in just 15 minutes. Biohaven’s Nurtec ODT is the first and only FDA-approved CGRP receptor antagonist available in a quick-dissolve ODT formulation.

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XTalks

JANUARY 3, 2024

Wainua is the only FDA-approved drug for the treatment of ATTRv-PN that can be self-administered via an auto-injector. Approval of Wainua represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease,” said Michael J.

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FDA Approval Protein RNA Clinical Trials 105

XTalks

MARCH 15, 2023

Read more here: Lenire Bimodal Neuromodulation Device by Neuromod Improves Tinnitus Symptoms in Clinical Trial The weekly podcast is available for streaming every Wednesday on Spotify , Apple Music and wherever you stream your podcasts. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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Life Science FDA Approval Engineer Clinical Trials 75

XTalks

NOVEMBER 13, 2024

Merck KGaA recently shared positive results from its Phase III MANEUVER trial on pimicotinib, an investigational oral therapy for tenosynovial giant cell tumor (TGCT). These findings align with early trial results, reinforcing pimicotinib’s promise for improving daily function in TGCT patients. percent of patients.

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Clinical Research FDA Approval Trials Research 105

XTalks

NOVEMBER 2, 2022

In this episode, Ayesha discussed the FDA approval of two new immunotherapies, including Janssen/Johnson & Johnson’s bispecific antibody Tecvayli for the treatment of relapsed or refractory multiple myeloma. The drug is the first bispecific T cell antibody to be approved in the US.

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FDA Approval Life Science Antibody Drugs 52

Pharmaceutical Technology

MARCH 14, 2023

Biopharmaceutical firm Cingulate and life sciences commercialisation firm Indegene have entered into a joint commercialisation deal for the former’s attention-deficit/hyperactivity disorder (ADHD) therapy CTx-1301 (dexmethylphenidate). The company expects to receive data from the trial in the third quarter of this year.

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XTalks

JANUARY 22, 2025

Valneva SE, who developed the worlds first FDA-approved chikungunya vaccine , has now unveiled promising Phase III data for its single-shot chikungunya vaccine, IXCHIQ, in adolescents aged 12 to 17. The VLA1553-321 trial, conducted in Brazil, was the first to assess the vaccine in an endemic region.

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Immune Response Vaccine Vaccination Trials 59

XTalks

MAY 2, 2022

This is the very first approval for a medication for the chronic version of VVC. The approval is a significant breakthrough for RVVC as it is a condition with significant unmet need. For Mycovia Pharmaceuticals , Vivjoa is the company’s first FDA-approved drug in its pipeline of novel treatments for fungal infections.

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FDA Approval Drugs Clinical Trials Clinical Trials 98

XTalks

AUGUST 7, 2023

has received US Food and Drug Administration (FDA) approval for treating molluscum contagiosum in adult and pediatric patients aged two years and older in the US. Formerly known as VP-102, Ycanth is the first cantharidin formulation approved for this purpose. Both trials demonstrated no severe adverse reactions.

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FDA Approval Dermatology Trials Clinical Trials 98

XTalks

APRIL 3, 2024

Related: Novartis’ Fabhalta Gets FDA Approval for Rare Complement Blood Disorder The FDA’s green light for Voydeya comes three months after the Japanese Ministry of Health, Labour and Welfare (JHLW) became the first regulator in the world to back the therapy. AstraZeneca is also venturing into cell therapy and genetic medicine.

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XTalks

MARCH 9, 2023

Results for this endpoint are expected in the final quarter of 2023 and are expected to support the traditional approval of Filspari. The featured speakers will discuss challenges, risks and opportunities in digital front door strategies for traditional, decentralized and hybrid trials.

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FDA Approval Drugs Clinical Trials Clinical Trials 98

XTalks

MAY 5, 2022

There are four FDA-approved ADHD-specific nonstimulant drugs, namely Eli Lilly’s Strattera (atomoxetine), Shionogi’s Kapvay (clonidine), Takeda’s Intuniv (guanfacine) and now Supernus’ Qelbree (viloxazine). In 2002, Strattera became the first FDA-approved nonstimulant drug to treat ADHD in adults.

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FDA Approval Drugs Clinical Trials Clinical Trials 98

XTalks

OCTOBER 4, 2022

The life expectancy of HRS-affected patients is very low, and the only definitive treatment is liver transplantation. XTALKS WEBINAR: Time for Change in NASH: How Non-Invasive Biomarkers are Driving a Paradigm Shift in Clinical Trials and Clinical Care. Outcomes of Clinical Trials of Terlipressin. percent (vs. percent (vs.

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FDA Approval Clinical Trials Clinical Trials Trials 98

XTalks

SEPTEMBER 14, 2022

Daxxify was FDA-approved for similar cosmetic purposes as Botox and other neuromodulators like Dysport and Xeomin. Used for both cosmetic and therapeutic cases, Botox is a US Food and Drug Administration (FDA)-approved injection of botulinum toxin, a neurotoxic protein that can effectively paralyze the facial muscles.

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Botox FDA Approval Cosmetics Clinical Trials 98

XTalks

JULY 28, 2021

Merck has scored US Food and Drug Administration (FDA) approval for its next-generation pneumonia vaccine Vaxneuvance that covers 15 different strains of the pneumococcal bacteria that causes the infection.

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FDA Approval Vaccine Vaccination Immune Response 89

XTalks

DECEMBER 18, 2024

Related: Arrowhead Pharmas New Rare Disease Campaign for FCS Highlights Importance of Lowering Triglycerides The FDAs approval of Crenessity was based on two randomized, double-blind, placebo-controlled trials in 182 adults and 103 children with classic CAH. Crenessity marks the third approved drug for Neurocrine.

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XTalks

JANUARY 26, 2023

The US Food and Drug Administration (FDA) recently granted approval for the Bruton tyrosine kinase inhibitor (BTKi) Brukinsa (zanubrutinib) based on the results from two Phase III clinical trials. Brukinsa is now approved to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

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FDA Approval Life Science Clinical Trials Clinical Trials 97

XTalks

OCTOBER 18, 2022

Global medical device company Nevro Corp has received US Food and Drug Administration (FDA) approval for the Senza HFX iQ spinal cord stimulation (SCS) system for the treatment of long-term or chronic pain. HFX iQ is designed to improve the consistency of pain relief and is the only SCS system that truly personalizes care,” said D.

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FDA Approval Big Data Life Science Clinical Trials 98

XTalks

OCTOBER 4, 2022

XTALKS WEBINAR: Decentralization in Action: What Oncology Trials Look Like Now. Register for this free webinar to learn new ways to operationalize hybrid oncology trials and how virtual sites help reduce patient burden. The featured speakers will discuss where the industry is headed with decentralized clinical trials and oncology.

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FDA Approval Clinical Trials Clinical Trials Trials 98

XTalks

NOVEMBER 4, 2021

The FDA approval of Vuity exemplifies our continued pursuit of innovative new treatments that push the boundaries of what’s possible in eye care.”. In May, New York biotech Eyenovia announced positive trial results for its drug-device combo called MicroLine for the treatment of presbyopia.

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