Shots: The US FDA has granted orphan drug designation to the company’s NM5072, first-in-class monoclonal antibody, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) The drug concluded P-I study in healthy participants (n=48) showing a well-tolerated & favorable safety profile at all dosing up to 20mg/kg and AP inhibition demonstrating a dose-dependent response The company anticipates the P-II clinical evaluation of NM5072 in treatment-naïve patients with paroxysmal nocturnal hemoglobinuria and is being reviewed for other indications such as C3G & aHUS. The SC formulation i ..read more

Shots: The P-III (TOPAZ-1) study investigates Imfinzi + CT (gemcitabine + cisplatin) vs CT + PBO as a 1L treatment of unresectable, locally advanced/metastatic biliary tract cancer patients (n=685) with intrahepatic & extrahepatic cholangiocarcinoma as well as gallbladder cancer The updated exploratory data of the study demonstrated a reduction in the death risk by 26%, mOS of 12.9mos. vs 11.3mos. at a median follow-up of 41.3mos. (>3yrs.) and OS rate of 14.6% vs 6.9% at 36mos. Additionally, the trial reached its 1EP of OS at a planned interim analysis depicting a reduction in the death ..read more

Becoming a healthcare professional is both rewarding and difficult. Success requires hard work, resilience, and an effective plan of attack. For students hoping to enter this industry, following their goals efficiently on this path could make all of the difference in achieving them. In this article, we'll share some essential advice that can assist students on their path toward becoming healthcare professionals. Stay Updated with the Latest News Staying abreast of current healthcare news, trends, and developments is essential in remaining up-to-date and helping shape goals and projects for bot ..read more

Shots: The US FDA has accepted the BLA of 5-in-1 meningococcal ABCWY (MenABCWY) vaccine based on the P-III study assessing the safety, tolerability & immunogenicity among individuals (n=~3650, 10-25yrs.) for IMD. The decision is anticipated on Feb 14, 2025 The study reached all the 1EPs showing immunological non-inferiority to 1 dose of Meningococcal Groups A, C, Y & W vaccine & non-inferior immune responses against 110 diverse MenB invasive strains The vaccine combines the antigenic parts from Bexsero (Meningococcal Group B Vaccine) & Menveo (Meningococcal [Groups A, C, Y & ..read more

Shots: The company has achieved the target patient recruitment (n=249) across the EU, the US & Canada in the P-II (DAZALS) study investigating dazucorilant (150mg & 300mg, daily) vs PBO for 24wks. The 1EP of the study includes the change in ALSFRS-R to assess the motor impairment & functional decline in ALS patients from baseline and 2EPs are the OS as well as QoL. The company anticipates the clinical data by the end of 2024 Dazucorilant, a cortisol modulator, selectively binds to the glucocorticoid receptor (GR) that is currently under evaluation for ALS and has potential for vari ..read more

Shots: The P-Ib dose escalation part of the study assessed the safety, tolerability, PK profile, provisional RP2D & preliminary antitumor activity of ORIC-114 for treating solid tumors associated with EGFR & HER2 exon 20 mutations as well as HER2 amplifications ORIC concluded the dose escalation part of the study and selected 80mg & 120mg QD as provisional RP2D to be further evaluated for final RP2D in the P-Ib dose expansion part assessing ORIC-114’s safety & efficacy in mutated NSCLC patients incl. EGFR exon 20, HER2 exon 20 & EGFR atypical mutations  Furthermore, th ..read more

Shots: Novartis has released pre-specified interim analysis data from the P-III (APPLAUSE-IgAN) trial to investigate Fabhalta's (200mg, oral, BID) safety & efficacy vs PBO to treat primary IgAN adults (n=518). The analysis evaluated 250 participants for efficacy & 443 for safety The analysis demonstrated a 38.3% reduction in proteinuria at 9mos. vs PBO alongside supportive care. The 1EP of slowing IgAN progression, estimated by annualized total eGFR slope at 24mos. is anticipated during trial completion in 2025 The safety of the drug was well-tolerated and favorable, aligning with the ..read more

Shots:  PharmaShots has compiled a list of US FDA-approved drugs in the month of March 2024  The US FDA approved a total of 14 drugs including 12 new molecular entities and 2 biologics leading to the treatments for patients and advances in the healthcare industry    The major highlighted drug was BeiGene’s Tevimbra for treating Advanced or Metastatic Esophageal Squamous Cell Carcinoma    1.Formosa Pharmaceuticals Receives the US FDA’s Approval for Clobetasol Propionate to Treat Post-Operative Inflammation and Pain Following Ocular Surgery    &n ..read more

Shots: TELA Bio has introduced OviTex IHR (Inguinal Hernia Repair) Reinforced Tissue Matrix for laparoscopic & robotic-assisted inguinal hernia repair across the US The device depicted a decreased 1.2% recurrence rate with an avg. follow up of 1.5yrs. in a study titled, “Minimizing Retained Foreign Body in Hernia Repair Using a Novel Technique: Reinforced Biologic Augmented Repair (ReBAR)” evaluating OviTex Core Permanent in patients (n=259) who had a robotic inguinal hernia TAPP repair by the ReBAR technique OviTex IHR has 3 configurations, 3- or 4-layer anatomically shaped devices or a 3 ..read more

Shots: The EAP study in patients (n=140) with SR/SD GI-aGvHD showed a GI-ORR of 52% at D28, OS of 54%, 47% & 42% at 6mos., 12mos. & 18mos., respectively, with 28% achieving CR. OS was greater with 68% vs 24% at 12mos. & 58 vs 24% at 18mos. in MaaT013 responders vs non-responders Among a subset of patients (n=49) refractory to steroids & ruxolitinib, GI-ORR was 63% at D28 with 49% achieving CR, OS rates were 52%, 49% & 42% at 6mos., 12mos. & 18mos., respectively, & overall ORR was 61% with 43% CR. OS was greater with 76% vs 6% at 12mos. & 64% vs 6% at 18mos. in M ..read more