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 […]
Care For Rare! – Celebrating Rare Disease Day
MedNess is proudly celebrating the Rare Disease Day today. We also take this opportunity to announce a new column “*Care For Rare!*” on the MedNess platform by our newest author, Malini Gupta, PhD! We will come back tomorrow with more info on Malini, her educational background and her interest, but in the meantime, we hope […]
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 […]
MaxCyte Signs Strategic Platform License with Imugene to Advance their Cancer Immunotherapy Programs
“By leveraging our scalable cell-engineering process and optimized clinical manufacturing workflow, Imugene is rapidly moving towards a potential Phase 2 registrational trial for azer-cel in cancer,” said Maher Masoud, President and CEO of MaxCyte. “Our technology has been integral to the manufacturing of the allogeneic T-cell immunotherapies and was efficiently transferred from Precision Biosciences when […]
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 […]
Complete Remission Of The Cohort 1 First Patient Treated For R/R B-ALL In The Phase 1 Clinical Trial Of PMB-CT01 (BAFFR-CAR T Cells) announced
“We are excited about the treatment outcomes as this patient had a relapsed B-cell ALL after prior treatment with chemotherapy and blinatumomab. His relapsed disease was both CD19- and CD22-negative, implying very limited available therapeutic options for him,” said Ibrahim T. Aldoss, M.D., City of Hope associate professor, Department of Hematology & Hematopoietic Cell Transplantation […]
Opdivo + CABOMETYX Demonstrates Long-Term Survival Benefits After Four Years of Follow-Up in the CheckMate -9ER Trial in 1L Advanced RCC
“Renal cell carcinoma can be very challenging to treat and patients who are diagnosed with advanced disease or develop metastasis often face poor outcomes,” said Maria Teresa Bourlon, Urologic Oncology Clinic, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico. “These updated results from the CheckMate -9ER trial continue to support the role of […]
KEYTRUDA Significantly Improved Disease-Free Survival (DFS) as Adjuvant Therapy Versus Observation in High-Risk Patients With Localized Muscle-Invasive and Locally Advanced Urothelial Carcinoma After Surgery
“Even after undergoing radical surgery with the goal of removing all cancerous tumors, up to half of patients with muscle-invasive bladder cancer still experience high rates of cancer recurrence,” said Dr. Andrea Apolo, principal investigator and chief of the Bladder Cancer Section, Genitourinary Malignancies Branch, National Cancer Institute (NCI), part of the National Institutes of […]
KEYTRUDA Reduced the Risk of Death by 38% Versus Placebo as Adjuvant Therapy for Patients With RCC at an Increased Risk of Recurrence Following Nephrectomy
“For patients with renal cell carcinoma, up to 40% may experience recurrence following surgery, at which point there is a significantly lower chance of survival,” said Dr. Toni K. Choueiri, director, Lank Center for Genitourinary Oncology, Dana-Farber Cancer Institute and Jerome and Nancy Kohlberg professor of medicine, Harvard Medical School. “Results from KEYNOTE-564 show that […]
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 […]
BLA accepted for obecabtagene autoleucel (obe-cel) as a potential treatment for relapsed/refractory Adult B-cell Acute Lymphoblastic Leukemia (ALL)
“Acceptance of the BLA filing is an important milestone for Autolus and we look forward to continuing our collaboration with the FDA during the review cycle,” commented Dr. Christian Itin, Chief Executive Officer of Autolus. “With the PDUFA date set for November, we remain focused on preparing for the potential launch of obe-cel.”
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 […]
Accurus Biosciences and ImmPACT Bio Enter into an Exclusive Global License Agreement for a Potentially Best-in-Class CLDN18.2 Antibody
“We are excited to announce that ImmPACT Bio chose to utilize our potential best-in-class CLDN18.2 antibody to develop next generation CAR T therapies,” said Dr. Richard Zhang, chief executive officer of Accurus. “We believe ImmPACT’s programs have the potential to deliver differentiated and powerful therapies to benefit cancer patients.”
Ferring announces full availability of ADSTILADRIN® (nadofaragene firadenovec -vncg) in the U.S.
“From the day we received FDA approval, Ferring has been committed to making ADSTILADRIN available to every appropriate high-risk NMIBC patient quickly and responsibly, working collaboratively with the bladder cancer community to inform our approach,” said Bipin Dalmia, Senior Vice President, Global Head, Uro-Oncology Franchise at Ferring Pharmaceuticals. “With our significant manufacturing investments, we have […]
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 […]
First Patient Enrolled in Pivotal FIERCE-HN Clinical Trial to Evaluate Ficlatuzumab in Combination with ERBITUX® (cetuximab) in Patients with HPV-negative Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC)
“The start of enrollment for the pivotal FIERCE-HN trial is an important milestone in the advancement of the combination of ficlatuzumab and cetuximab, as it brings us a step closer to offering a new potential therapy to a patient population that has limited effective treatment options available to them today,” said Michael Bailey, president and […]
NGC-Cap Program expanded into Advanced or Metastatic Breast Cancer
“We believe the pursuit of an advanced or metastatic breast cancer indication for NGC-Cap is a logical progression for Processa as it represents a larger market than colorectal cancer with the potential to differentiate NGC-Cap from the presently approved capecitabine as well as other treatments for breast cancer. The FDA and Processa discussed the advantages […]
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 […]
Data from a Phase 1b Study of Quemliclustat-Based Regimens Showed Promising Overall Survival in Treatment-Naïve Metastatic Pancreatic Cancer
“A quemliclustat-based regimen appears to meaningfully prolong survival compared to what we typically observe in patients with mPDAC who receive chemotherapy alone, the standard of care for more than 30 years,” said Zev A. Wainberg, MD, MSc, Co-Director of the GI Oncology Program at University of California Los Angeles and a principal investigator of the […]
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 […]
Orphan Drug Designation Granted to PTX-252 by U.S. FDA for the Treatment of Acute Myeloid Leukaemia (AML)
Stijn Van Rompay, Chief Executive Officer of Hyloris, commented: “Securing orphan drug designation for a product candidate incorporating a novel molecular entity, not yet approved by any regulatory agency, underscores our unwavering commitment to advancing the frontiers of scientific discovery within the repurposing space.”
Submission of Complete Response to Clinical Hold for HEMO-CAR-T IND
Dr Vladislav Sandler, Chief Executive Officer, commented: “We are pleased to have filed a complete response to the FDA addressing their concerns that resulted in a CH of the HEMO-CAR-T IND. We look forward to becoming a clinical stage biopharmaceutical company once consent is received from the FDA.”
