Mission Therapeutics starts a clinical trial on its Parkinson’s Disease lead, MTX325
Mission Therapeutics recently announced the commencement of Phase I first-in-human clinical trial of MTX325 as a potential disease-modifying treatment for Parkinson’s Disease (PD). The potent selective central nervous system-penetrant compound is designed to improve mitochondrial quality and function by enhancing mitophagy. It targets a deubiquitylating enzyme, USP30 known to inhibit mitophagy. While normal cells can […]
Accelerated Approval Granted to Enhertu for HER2-Positive Solid Tumors
The Food and Drug Administration (FDA) has recently granted accelerated approval to fam-trastuzumab deruxtecan-nxki (Enhertu). This approval is significant for adult patients suffering from unresectable or metastatic HER2-positive (IHC3+) solid tumors who have exhausted prior systemic treatment options. Enhertu is a specifically engineered HER2-directed antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly […]
Wegovy is the New Weight Loss Superstar to Win FDA Approval
Novo Nordisk, already popular weight loss med Wegovy, added another feather in its cap. NOVO’s SELECT cardiovascular outcome showed a 20% reduction in CV events. The study was conducted for five years on 17,604 patients who were obese or overweight. The trial was initiated in 2018. The FDA nod was based on the results of […]
Supplemental Biologics License Application for intravenous maintenance dosing of Alzheimer’s drug, LEQEMBI
A Supplemental Biologics License Application (sBLA) was filed to the U.S. Food and Drug Administration (FDA) by Eisai for Lecanemab-irmb (U.S. brand name: LEQEMBI®), the approved drug for treating mild cognitive impairment or early Alzheimer’s Disease (AD). Alzheimer’s Disease (AD) is the most common cause of dementia where aggregates of amyloid beta (Aβ) plaques and […]
C For Crigler-Najjar Syndrome
Crigler-Najjar Syndrome (CNS) is a rare, autosomal inherited disorder characterized by a loss or deficiency of the liver enzyme Uridine Diphosphate glucuronosyltransferase (UGT), resulting in the inability to convert and clear bilirubin from the liver. Bilirubin is an orange-yellow pigment produced by the normal breakdown of worn-out RBCs, by a process called hemolysis. To be […]
B for Beta Thalassemia – Part II
Treatment options for Beta Thalassemia involve alleviating symptoms, managing complications, and improving quality of life. The specific treatment approach depends on the type and severity of Thalassemia. Standard therapy for this disease includes repeated lifelong blood transfusions to compensate for the lack of enough functional red blood cells. Patients with transfusion-dependent Thalassemia (TDT)require transfusions every […]
Advancing Cancer Treatment: FDA Approval of Mirvetuximab Soravtansine-Gynx for Platinum-Resistant Epithelial Ovarian Cancer
Image Source: Vinoth Khandelwal In a recent development for the treatment of platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, the Food and Drug Administration (FDA) has recently approved mirvetuximab soravtansine-gynx (Elahere, ImmunoGen, Inc. [now a part of AbbVie]). This approval marks a milestone in addressing the medical needs of adult patients with FRα […]
B for Beta Thalassemia
Beta Thalassemia (BT) is an inherited autosomal rare recessive blood disorder caused by reduced levels of functional hemoglobin (Hb), the red iron heavy oxygen-carrying pigment of the blood. Hemoglobin is a tetramer of globin chains each bound to a heme group which is bound to iron which accommodates oxygen. The four types of globin chains […]
Novo Weight Loss Drug Semaglutide (Ozempic’s) Reduces Kidney Disease Progression
Novo Semaglutide, aka Ozempic, has added another feather to the cap besides reducing body weight. Ozempic has been shown to reduce the progression of chronic kidney disease (CKD). The FLOW trial has shown that administration of Semaglutide (1.0 mg) reduces the risk of kidney disease-related problems in patients suffering from type 2 diabetes. This reduction is 24 […]
A for Acromegaly – Part II
On March 5th, 2024, Camurus AB, a Swedish biopharmaceutical company, announced FDA approval for the review of their New Drug Application (NDA) for OclaizTM (CAM2029) for the treatment of Acromegaly. The FDA has also set a Prescription Drug User Fee Act (PDUFA) target action date for October 21, 2024. The NDA submission is supported by […]
Takeda’s ICLUSIG Receives FDA Approval for Frontline Treatment of Ph+ Acute Lymphoblastic Leukemia
Takeda recently announced a significant milestone in cancer treatment with the U.S. Food and Drug Administration (FDA) granting accelerated approval for the supplemental New Drug Application (sNDA) of ICLUSIG® (ponatinib). This approval marks a crucial advancement in the treatment landscape for adult patients newly diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). ICLUSIG targets […]
A for Acromegaly – Part I
Acromegaly is a rare, acquired and slowly progressive condition caused by excessive production of growth hormone (GH) by the pituitary gland, due to the presence of a “pituitary adenoma”, a benign tumor. The pituitary gland is a pea-sized endocrine gland located at the base of the skull, in charge of producing several essential hormones and […]
FDA Approves Inotuzumab Ozogamicin for Pediatric Acute Lymphoblastic Leukemia
The Food and Drug Administration (FDA) has granted approval for inotuzumab ozogamicin (Besponsa, Pfizer) for pediatric patients aged 1 year and older with relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL). This approval marks a significant advancement in the treatment landscape for this challenging condition. Inotuzumab ozogamicin functions by targeting CD22-positive B-cell precursor […]
Personal Genome Diagnostics
Personal Genome Diagnostics (PGDx) specializes in developing genomic products for cancer diagnosis. Combining next-generation sequencing, bioinformatics, and oncology expertise, PGDx pioneers advanced diagnostic approaches to identify genomic alterations that guide therapy selection and monitoring. Founded in 2010 by Luis Diaz and Victor Velculescu based on technology developed at the Johns Hopkins University, the company has […]
Care For Rare: Prologue
What are rare diseases? Being unique isn’t easy and Rare Diseases due to their low prevalence have not been an exception. In a law passed by the United States Congress in 1983 called the Orphan Drug Act, Rare Diseases are defined as conditions affecting less than 200,000 people in the United States. In contrast, the […]
FDA Approval of Amivantamab-vmjw: Advancing Treatment for EGFR Exon 20 Insertion-Mutated Non-Small Cell Lung Cancer
The recent approval by the Food and Drug Administration (FDA) of amivantamab-vmjw (Rybrevant, Janssen Biotech) marks a significant milestone in the treatment landscape for non-small cell lung cancer (NSCLC). This approval targets locally advanced or metastatic NSCLC with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, a subgroup historically challenging to treat effectively. The […]
Versatile “Zepbound” by Eli Lilly Became the First Weight Loss Drug Approved by the FDA
Food and Drug Administration (FDA) approved ZepboundTM (Tirzepatide) for weight management in 2023. This twincertin compound is approved for controlling blood sugar in patients suffering from T2DM. Trizepatide is a precise amalgamation of the glucose-dependent insulinotropic polypeptide (GIP) and Glucagon-like peptide (GLP-1) receptors and has added advantage over Semaglutide, Liraglutide, Dulaglutide (agonist or activators of […]
EpCAM CAR-T Therapy receives Dual IND from U.S. FDA and China CDE
Immunofoco, has achieved a significant milestone with the approval of Investigational New Drug (IND) applications by both the U.S. Food and Drug Administration (FDA) and the China Center for Drug Evaluation (CDE) for IMC001, an autologous CAR-T cell therapy targeting EpCAM. This groundbreaking approval marks a pivotal moment in the treatment landscape for advanced solid […]
STEMPeers’ Oncology digital news aggregator platform Onco-This-Week acquired by Rosenblatt Life Science Consultants
February 9, 2024: Erie, PA: US-based non-profit, PhD Career Support Group DBA STEMPeers, a 21,000+ member organization, announces the asset purchase agreement of their oncology news aggregator platform, Onco-This-Week, to Rosenblatt Life Science Consultants (RLS Consultants). Rosenblatt Life Science Consultants is a global consulting and training firm focusing exclusively on the healthcare and life sciences […]
LAVA Therapeutics Announces Collaboration with Merck to Evaluate LAVA-1207 in Combination with KEYTRUDA
“We are excited to work with Merck & Co., Inc., Rahway, NJ, USA as we continue to unlock the therapeutic potential of LAVA-1207 and explore its potential capabilities in combination with KEYTRUDA®,” said Stephen Hurly, President and Chief Executive Officer, LAVA. “To date, LAVA-1207 has demonstrated a favorable safety profile and shown preliminary signs of […]
Inhibrx Announces Sale of INBRX-101 to Sanofi for an aggregate value of up to $2.2B
“Inhibrx and Sanofi announced that the companies have entered into a definitive agreement under which Aventis Inc., a Pennsylvania corporation (a subsidiary of Sanofi) will acquire all the assets and liabilities associated with INBRX-101, an optimized, recombinant alpha-1 antitrypsin (“AAT”) augmentation therapy currently in a registrational trial for the treatment of patients with alpha-1 antitrypsin […]
2023 Year-End Annamycin Clinical Trials Preliminary Data and 2024 Expectations for Multiple Data Readouts and Transition to Pivotal Phase 2B/3 announced
“Over the course of 2023, we delivered on our promise for a year of important data from our Annamycin clinical development programs. We are well-positioned to continue building upon our encouraging growing body of preliminary clinical data and transition to pivotal Phase 2B/3 clinical trials by year-end 2024. We believe that Annamycin should be positioned […]
FAILED TRIAL: Phase 3 EVOKE-01 study did not meet its primary endpoint of OS in previously treated metastatic NSCLC
“The totality of our data gives us continued confidence in Trodelvy’s potential in metastatic NSCLC, and in our broader lung cancer clinical development program,” said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences. “Treating metastatic NSCLC that has progressed on or after platinum-based chemotherapy presents significant challenges and the need for safe and effective […]
Submission of a Rolling NDA to the FDA for UGN-102 initiated
“The submission of the CMC portion of the NDA for UGN-102 marks a significant milestone for UroGen and underscores our dedication to advancing innovative therapies for the benefit of individuals grappling with low-grade, intermediate-risk non-muscle invasive bladder cancer,” said Liz Barrett, President and CEO, UroGen. “We look forward to working closely with the FDA throughout […]
Positive CHMP Opinion for CAR T Cell Therapy Abecma (idecabtagene vicleucel) in Earlier Lines of Therapy for Triple-Class Exposed R/R Multiple Myeloma
“This positive CHMP opinion represents an important step toward bringing our potentially transformative first-in-class anti-BCMA CAR T cell therapy, Abecma, to more patients earlier in the multiple myeloma treatment paradigm to improve outcomes,” said Anne Kerber, M.D., senior vice president and head, Late Clinical Development, Hematology, Oncology, Cell Therapy (HOCT), Bristol Myers Squibb. “We look […]
UroGen Secures Exclusive License from medac GmbH to Develop a Next-Generation Novel Mitomycin-Based Formulation for Urothelial Cancers
“The strategic alliance with medac fortifies our commitment to continuously innovate novel, non-surgical treatments for patients with urothelial cancers, including LG-IR-NMIBC,” said Liz Barrett, President and CEO of UroGen. “The 80 mg formulation of mitomycin from medac is specifically designed to be mixed with our RTGel® technology, which may provide advantages for patients looking for […]
First Patient Treated in the Phase 2a Trial of LSTA1 in Patients with Glioblastoma Multiforme
“We are very pleased to announce the first patient treated in this Phase 2a study evaluating LSTA1 in patients with newly diagnosed GBM, a very aggressive brain tumor that is often fatal. We hold great hopes for the benefits of LSTA1 in this indication based on preclinical evidence that demonstrates LSTA1 enhances penetration through the […]
IK-595 first cohort treated and cleared safety evaluation window
“We are laser focused on driving IK-930 and IK-595 forward in the next year to interpretable and clear data reads as we continue to build value for investors,” commented Mark Manfredi, Ph.D., Chief Executive Officer of Ikena. “Our extended team has achieved many significant milestones together, and IK-930 and IK-595’s clinical progress is evidence of […]
Imfinzi plus transarterial chemoembolisation (TACE) and bevacizumab reduced the risk of disease progression or death by 23% vs. TACE in liver cancer eligible for embolisation
Bruno Sangro, MD, PhD, Director of the Liver Unit and Professor of Medicine at Clínica Universidad de Navarra, Pamplona, Spain and a lead investigator in the EMERALD-1 trial, said: “In this earlier liver cancer setting, embolisation alone has been the standard of care for more than 20 years, and rates of disease progression have remained […]
Fast Track Designation for Combination of Avutometinib and Sotorasib for the Treatment of KRAS G12C-Mutant NSCLC
“Receiving Fast Track Designation for the combination of avutometinib and sotorasib reinforces the importance of improving the depth of MAPK pathway inhibition to enhance tumor regression relative to KRAS G12C inhibition alone and the potential of the combination of avutometinib and sotorasib in KRAS G12C mutant locally advanced or metastatic NSCLC,” said Dan Paterson, President […]
