FDA Calendar

Jaguar Health, Inc. and Napo Therapeutics announced that preliminary results from the ongoing pediatric investigator-initiated trial (IIT) of a novel liquid formulation of crofelemer, Jaguar's novel plant-based anti-secretory prescription drug, for various congenital diarrheal disorders (CDD), including MVID and SBS with intestinal failure (SBS-IF), will be presented by Dr. Mohamad Miqdady at the April 24-26, 2025 Annual ELITE PED-GI Congress in Abu Dhabi in the United Arab Emirates.

NANO-X IMAGING announced that it has received 510(k) clearance from the U.S. Food and Drug Administration (FDA) for the Nanox.ARC X, its new multi-source digital tomosynthesis system.

Alector, Inc. announced the completion of enrollment in PROGRESS-AD, a 76-week Phase 2 clinical trial evaluating the safety and efficacy of AL101/GSK4527226 in slowing disease progression in individuals with early Alzheimer's disease (AD).

Actuate Therapeutics, Inc announced that data on elraglusib in advanced salivary gland carcinoma will be presented in a poster session at the American Association for Cancer Research (AACR) Annual Meeting 2025 taking place from April 25th – 30th at McCormick Place Convention Center, Chicago, IL.

Calidi Biotherapeutics Inc. announced that that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for CLD-201.

Palatin Technologies, Inc. announced positive appetite suppression results from its BMT-801 Phase 2 obesity study.

uniQure N.V announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington's disease, a rare, inherited neurodegenerative disorder for which there are currently no disease-modifying therapies available.

Nurix Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the IND for the IRAK4 degrader GS-6791/NX-0479

Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for EYLEA HD® (aflibercept) Injection 8 mg.

Addex Therapeutics announced today that following the previously announced termination of development of ADX71149 (JNJ-40411813) in epilepsy, its partner Janssen Pharmaceuticals, Inc. (now known as J&J Innovative Medicine) has return all development and commercialization rights to ADX71149 (JNJ-40411813) and the partnership between the two companies has been terminated.

Pfizer Inc. that the U.S. Centers for Disease Control and Prevention's (CDC) Advisory Committee on Immunization Practices (ACIP) voted to expand its recommendation for the use of respiratory syncytial virus (RSV) vaccines approved for adults 50-59 years of age at increased risk of RSV-associated lower respiratory tract disease (LRTD).

Clearmind Medicine Inc. announced it has initiated its Phase I/IIa clinical trial at its first U.S. clinical site, the Johns Hopkins University School of Medicine. The first in human clinical trial will investigate the safety, tolerability and full pharmacokinetic profile of Clearmind's innovative treatment, CMND-100, in Alcohol Use Disorder (AUD) patients

RenovoRx, Inc announced that it is increasing production of its FDA-cleared RenovoCath catheter-based device in order to meet increased demand for the targeted delivery of diagnostic and/or therapeutic agents from oncologists and interventional radiologists.

Aditxt, Inc. announced that its subsidiary, Pearsanta, Inc. ("Pearsanta"), has received Institutional Review Board (IRB) approval from WCG Clinical to initiate a prospective clinical study evaluating the Mitomic® Endometriosis Test (MET), a novel blood-based diagnostic designed to aid in the early detection of endometriosis.

AptarGroup, Inc. announces the commencement of a clinical study to validate its proprietary SmartTrack™ platform.

PDS Biotechnology nnounced that preclinical immune response data with a novel Infectimune® based flu vaccine will be featured in a symposium on universal influenza vaccines at the American Association of Immunologists' IMMUNOLOGY2025™ Annual Meeting, taking place May 3-7, 2025, in Honolulu, Hawaii.

MIRA Pharmaceuticals announced compelling data demonstrating the efficacy of the oral ketamine analog, Ketamir-2, in a validated animal model of diabetic neuropathy.

BioVie Inc announced that patient enrollment is now open for the Phase 2 SUNRISE-PD clinical trial evaluating the safety and efficacy of bezisterim (NE3107) on motor and non-motor symptoms in patients with Parkinson's disease (PD) who haven't been treated with carbidopa/levodopa.

Cardiol Therapeutics Inc. announced that Northwestern University has enrolled the first patient in the pivotal Phase III MAVERIC trial ("MAVERIC") evaluating Cardiol's lead drug candidate CardiolRx™ for the prevention of recurrent pericarditis.

BriaCell Therapeutics Corp announces new positive survival data in its Phase 2 study of Bria-IMT plus check point inhibitors (CPI), outperforming ADC drugs in hormone receptor positive (HR+) metastatic breast cancer (MBC) patients.

INmune Bio Inc announced a major intellectual property milestone with respect to its next-generation mesenchymal stromal cell (MSC) product, CORDStrom™.

Purple Biotech Ltd. announced the publication of an independent study titled "IRS2 as a driver of brain metastasis in colorectal cancer: a potential target for novel therapeutic strategies" in the peer reviewed journal, Neuro Oncology. NT219 is a first-in-class small molecule drug designed to target key cancer resistance mechanisms by degrading IRS1/2 and blocking downstream signaling towards AKT and b-catenin, as well as STAT3 survival pathways.

Q32 Bio Inc. announced that the Company has dosed the first patients in both the Part A open-label extension (OLE) and Part B of the SIGNAL-AA Phase 2a clinical trial evaluating bempikibart in patients with alopecia areata (AA).

Cullinan Therapeutics, announced that the European Medicines Agency (EMA) approved the Company's Clinical Trial Application (CTA) for CLN-978.

RedHill Biopharma Ltd. announced the new publication[2] of positive in vivo data, in the journal Diabetes, Metabolic Syndrome and Obesity, in an article entitled "Opaganib Promotes Weight Loss and Suppresses High-Fat Diet (HFD)-Induced Obesity and Glucose Intolerance".

EXCLUSIVE: MIRA Pharmaceuticals Ketamir-2 Shows Efficacy In Diabetes-Associated Nerve Damage In Animal Study

Cardiff Oncology, Inc. announced completion of patient enrollment in the ongoing Phase 2 CRDF-004 trial evaluating onvansertib in combination with standard of care (SoC) for the treatment of first-line RAS-mutated metastatic colorectal cancer (mCRC).

Hyperfine, Inc announced the enrollment of initial patients in the NEURO PMR (Neurological Evaluation in the Office with Portable MRI) study.

Scilex Holding Company announced that it has received approval of a New Drug Submission (NDS) from the Health Canada's Pharmaceutical Drugs Directorate, Bureau of Cardiology, Allergy and Neurological Sciences for ELYXYB®'s acute treatment of migraine with or without aura in Canada.

BioCardia, announced that the study's independent DSMB, which conducted a planned review of the 30-day safety data from the roll-in 20 million cell dosing cohort in the CardiALLO-HF trial, recommended that the study continue as designed.

Hoth Therapeutics, Inc. announced positive interim data from the open-label portion of its Phase 2a clinical trial, CLEER-001, evaluating HT-001 for the treatment of pruritus associated with skin toxicities caused by Epidermal Growth Factor Receptor (EGFR) inhibitors.

Mural Oncology plc announced that following review of data from its phase 2 ARTISTRY-6 trial in melanoma and previously announced results from the phase 3 ARTISTRY-7 trial in platinum-resistant ovarian cancer, the company is discontinuing all clinical development of nemvaleukin alfa and plans to immediately commence the exploration of strategic alternatives focused on maximizing shareholder value. Mural has engaged Lucid Capital Markets, LLC to act as its financial advisor in connection with the exploration of strategic alternatives.

Ensysce Biosciences, Inc. announced completion of Part 1 of its second clinical trial to evaluate PF614-MPAR for overdose protection.

Tectonic Therapeutic, announced that it will make a late-breaking oral presentation at the European Society of Cardiology (ESC) Heart Failure 2025 Congress.

ImmunityBio, Inc announced that it has completed multiple submissions to the FDA including an sBLA for BCG-unresponsive NMIBC in papillary disease and an EAP for ANKTIVA® (nogapendekin alfa inbakicept-pmln) for the treatment of lymphopenia.

Plus Therapeutics, Inc. announces the online availability of new data on its lead compound REYOBIQ™ (rhenium Re186 obisbemeda) in an abstract for both an oral presentation and a poster to be presented at the Nuclear Medicine and Neurooncology conference to be held May 9-10, 2025 in Vienna, Austria.

MetaVia Inc. announced positive results from the 4-week multiple ascending dose (MAD) Part 2 of its Phase 1 clinical trial of DA-1726, a novel, dual oxyntomodulin (OXM) analog agonist that functions as a glucagon-like peptide-1 receptor (GLP1R) and glucagon receptor (GCGR), for the treatment of obesity.

Precision BioSciences, Inc announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for PBGENE-HBV, the Company's lead wholly owned in vivo gene editing program designed to cure chronic hepatitis B by eliminating cccDNA, the key source of replicating hepatitis B virus (HBV), and inactivating integrated HBV DNA in hepatocytes.

Rein Therapeutics announced a publication highlighting the therapeutic potential of Caveolin-1-related peptide LTI-2355 in Idiopathic Pulmonary Fibrosis (IPF) and Post-Acute Sequelae of COVID Fibrosis (PASC-F) in the peer-reviewed journal, Biomedicines.

Bluejay Diagnostics, Inc. announced acceptance of an abstract related to the Symphony IL-6 Test, the company's lead product candidate, for presentation at the Society of Academic Emergency Medicine (SAEM) Annual Meeting, taking place in Philadelphia May 13-16, 2025.

Mind Medicine announced that the first patient has been dosed in its Phase 3 Emerge study evaluating MM120 ODT, a proprietary, pharmaceutically optimized form of LSD for the treatment of MDD.

Skye Bioscience, Inc announced new preclinical data for its novel CB1 antibody, nimacimab.

Sanofi today shared new progress from its mid- to late-stage respiratory pipeline, including preliminary phase 2 results for amlitelimab in adults with moderate-to-severe asthma.

Exicure, Inc announced that GPCR Therapeutics USA, a subsidiary of Exicure Inc., has dosed the 19th patient in its ongoing Phase 2 clinical trial evaluating GPC-100 (burixafor), a small molecule CXCR4 inhibitor, for the mobilization of stem cells in multiple myeloma (MM) patients undergoing autologous stem cell transplant (ASCT) (NCT05561751).

Bristol Myers Squibb announced the Phase 3 ODYSSEY-HCM trial evaluating Camzyos (mavacamten) for the treatment of adult patients with symptomatic New York Heart Association (NYHA) class II-III non-obstructive hypertrophic cardiomyopathy (nHCM) did not meet its dual primary endpoints of changes from baseline to Week 48 compared to placebo in the Kansas City Cardiomyopathy Questionnaire – Clinical Summary Score (KCCQ-23 CSS) and peak oxygen consumption (pVO2).

Mirum Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of LIVMARLI® (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC).

Aditxt, Inc announced that a recently completed study conducted by the Mayo Clinic further validates the preclinical findings of ADI-100, the lead therapeutic candidate developed by Aditxt's wholly owned subsidiary, Adimune™, Inc. ("Adimune").

NeOnc Technologies announced that Trial Read-Out Data Expected in Early 2026

NeOnc Technologies announced that it expects full enrollment in its Phase 2a clinical trial for its lead therapeutic candidate, NEO100-01 in September. Only six patients remain to complete the trial's 25-patient enrollment target.

UroGen Pharma Ltd announced that data on investigational drug UGN-102 (mitomycin) for intravesical solution, JELMYTO (mitomycin) for pyelocalyceal solution and UGN-301 (zalifrelimab) will be presentedat the American Urological Association (AUA) 2025 Annual Meeting being held in Las Vegas, Nevada from April 26-29.

Ainos, Inc. announced significant progress in advancing its oral interferon drug platform, VELDONA® in the rare disease space.

Fate Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to FT819, an investigational, off-the-shelf, iPSC-derived CAR T-cell therapy in Phase 1 clinical development for the treatment of active moderate to severe systemic lupus erythematosus (SLE), including lupus nephritis (LN).

Fate Therapeutics, Inc Additional Phase 1 clinical data of FT819 to be presented at medical conferences in 2025

INmune Bio Inc. a clinical-stage biotechnology company targeting inflammation and immunology through the innate immune system has partnered with the Cell and Gene Therapy Catapult (CGT Catapult) to establish large-scale, commercial-ready manufacturing for its cell therapy platforms.

Soligenix, Inc announced interim results from the ongoing open-label, investigator-initiated study (IIS) evaluating extended HyBryte™ (synthetic hypericin) treatment for up to 54 weeks in patients with early-stage cutaneous T-cell lymphoma (CTCL).

Relmada Therapeutics, announced the presentation of an abstract at the American Urology Association (AUA2025), taking place from April 26-29th in Las Vegas.

Soleno Therapeutics, Inc announced the U.S. commercial availability of VYKAT™ XR (diazoxide choline) extended-release tablets, the company's treatment for hyperphagia in patients four years of age and older with Prader-Willi syndrome (PWS), which was approved by the U.S. Food and Drug Administration (FDA) on March 26, 2025.

NanoViricides, Inc. announced that it has begun evaluation of its clinical drug NV-387 for the treatment of the Measles virus.

Verve Therapeutics announced positive initial data from the Heart-2 Phase 1b clinical trial of VERVE-102. The Heart-2 Phase 1b clinical trial is evaluating patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD), two populations that require deep and durable reductions of low-density lipoprotein cholesterol (LDL-C) levels in the blood.

Ironwood Pharmaceuticals, Inc announced that, based on a recent discussion with the U.S. Food and Drug Administration (FDA), a confirmatory Phase 3 trial is needed to seek approval of apraglutide for patients with short bowel syndrome (SBS) with intestinal failure (IF) who are dependent on parenteral support.

Exact Sciences Corp. announced the publication of a comprehensive review in JAMA Oncology that strengthens the evidence supporting the Oncotype DX Breast Recurrence Score test.

IDEAYA Biosciences, Inc. announced a successful FDA Type D meeting on the Phase 3 registrational trial design that will assess the safety and efficacy of darovasertib for potential regulatory approval as neoadjuvant therapy for primary uveal melanoma (UM).

Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) approved Opdivo®(nivolumab) plus Yervoy® (ipilimumab) as a first-line treatment for adult patients with unresectable or metastatic hepatocellular carcinoma (HCC), the most common primary liver cancer.

Amgen announced that the global Phase 3 DeLLphi-304 clinical trial evaluating IMDELLTRA® (tarlatamab-dlle) as a treatment for patients with small cell lung cancer (SCLC) who progressed on or after a single line of platinum-based chemotherapy met its primary endpoint at a planned interim analysis.

Biodexa Pharmaceuticals Expects Initiation Of Phase 3 Registrational Study Of eRapa In FAP, Initiation Of IIT Of Tolimidone In T1D At University Of Alberta As 2025 Clinical Milestones

Palvella Therapeutics, Inc. announced QTORIN™ rapamycin 3.9% anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs) was featured by Dr. Amy Paller in an oral presentation at the 15th World Congress of Pediatric Dermatology in Buenos Aires, Argentina. Dr. Amy Paller is the Walter J. Hamlin Professor and Chair of Dermatology, Professor of Pediatrics, and Principal Investigator of the NIH-funded Skin Biology and Diseases Resource-based Center at Northwestern University's Feinberg School of Medicine and has served as President of the Society for Investigative Dermatology (SID), the Society for Pediatric Dermatology (SPD), the International Eczema Council (IEC), the Pediatric Dermatology Research Alliance (PeDRA), and the Women's Dermatological Society (WDS).

Verve Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for VERVE-102 for the treatment of patient groups with hyperlipidemia and high lifetime cardiovascular risk to reduce low-density lipoprotein cholesterol (LDL-C).

Perspective, Therapeutics, Inc. announced that the first patient was dosed in a new cohort of a Phase 1/2a trial evaluating the safety of [212Pb]VMT01, a targeted alpha-particle therapy (TAT), as monotherapy in patients with histologically confirmed melanoma and positive melanocortin 1 receptor (MC1R) imaging scans.

argenx SE announced that the U.S. Food and Drug Administration (FDA) approved a new option for patients to self-inject VYVGART® Hytrulo with a prefilled syringe (efgartigimod alfa and hyaluronidase-qvfc) for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive and adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP).

Protara Therapeutics, Inc. announced that two presentations and a panel discussion highlighting the ongoing Phase 2 open-label ADVANCED-2 trial of TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC) will be featured at the upcoming American Urological Association (AUA) 2025 Annual Meeting taking place from April 26, 2025 to April 29, 2025 in Las Vegas.

DexCom, Inc announced today the FDA has cleared the Dexcom G7 15 Day Continuous Glucose Monitoring System for people over the age of 18 with diabetes in the United States.

Clearmind Medicine Inc announced . that it has initiated its Phase I/IIa clinical trial at its first U.S. clinical site, the Johns Hopkins University School of Medicine.

Theriva Biologics announced the presentation of the previously disclosed blinded safety and pharmacokinetic (PK) data from the ongoing Phase 1b/2a randomized, double-blinded, placebo-controlled clinical trial of SYN-004 (ribaxamase) in allogeneic hematopoietic cell transplant (HCT) recipients for the prevention of acute graft-versus-host-disease (aGVHD).

Oculis Holding AG announced that it has completed enrollment in both Phase 3 DIAMOND-1 and DIAMOND-2 trials of OCS-01 eye drops in DME, designed as pivotal registration studies to support global marketing applications including NDA submission and approval by the U.S. Food and Drug Administration (FDA).

Arcturus Therapeutics Holdings Inc announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the self-amplifying mRNA (sa-mRNA) vaccine candidate, ARCT-2304, designed for active immunization to protect against disease caused by influenza A H5N1 subtype contained in the vaccine.

OS Therapies announced positive data in the prevention or delay of amputation during the treatment of primary osteosarcoma for OST-HER2 combined palliative radiation in dogs with unresected appendicular osteosarcoma.

Alterity Therapeutics announced that new presentations related to its Multiple System Atrophy (MSA) program were delivered at the American Academy of Neurology (AAN) 2025 Annual Meeting, one of the premier global neurology meetings.

Palatin Technologies, Inc. announced that data from the Phase 2b BREAKOUT study will be presented today at the National Kidney Foundation Spring Meeting in Boston, MA.

BrainStorm Cell Therapeutics Inc. announced the submission of an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration (FDA) for NurOwn®, the company's autologous mesenchymal stem cell therapy for amyotrophic lateral sclerosis (ALS).

Harrow announced an expansion of its VEVYE® Access for All("VAFA") program to include patients currently prescribed Klarity-C Drops®, a compounded cyclosporine 0.1% product manufactured and distributed by ImprimisRx®, Harrow's compounding subsidiary.

Pasithea Therapeutics Corp. announced that the external Safety Review Committee recommended that the Company's Phase 1 clinical trial of PAS-004 in advanced cancer should proceed to Cohort 6, 30mg capsule, without modification.

Gain Therapeutics, Inc. announced that an oral presentation as well as a poster were presented at the AD/PD™ 2025 International Conference on Alzheimer's and Parkinson's Diseases and related neurological disorders held April 1-5, 2025, in Vienna, Austria.

IDEAYA Biosciences, Inc announced the initiation of a Phase 1/2 expansion in the clinical trial evaluating IDE397, its investigational, potential first-in-class, small molecule methionine adenosyltransferase 2a (MAT2A) inhibitor, in combination with Gilead's Trodelvy® (sacituzumab govitecan-hziy), a Trop-2 directed antibody-drug conjugate (ADC), in methylthioadenosine phosphorylase (MTAP)-deletion urothelial cancer (UC) based on preliminary safety and clinical efficacy data.

Halozyme Therapeutics, Inc. announced that Janssen-Cilag International NV, a Johnson & Johnson company, received European Commission (EC) approval for an indication extension of DARZALEX® (daratumumab) subcutaneous (SC) co-formulated with ENHANZE® in the frontline setting.

Palisade Bio, Inc. announced the completion of enrollment and dosing in all five Phase 1a SAD cohorts, all four MAD cohorts, and the food effects crossover in the Phase 1a portion of its ongoing Phase 1a/b study of PALI-2108 for the treatment of Ulcerative Colitis (UC).

Microbot Medical Inc presented for the first time the data from its ACCESS-PVI pivotal trial at the Society of Interventional Radiology (SIR) annual meeting.

NeuroSense Therapeutics, Ltd. announced promising new findings from its Phase 2b PARADIGM clinical trial.

CERo Therapeutics Holdings, Inc. announces its first clinical trial site for the Company's Phase 1 clinical trial of CER-1236.

Quest Diagnostics announced the launch of a new laboratory blood test designed to help physicians confirm amyloid brain pathology due to Alzheimer's disease (AD) in patients with mild cognitive impairment (MCI) or dementia.

Phio announced today that the Safety Monitoring Committee (SMC) recommended dose escalation in Phio's Phase 1b clinical trial (NCT 06014086) for Phio's lead product candidate, PH-762.

Grace Therapeutics, Inc. announced details of the Company's Type C meeting with the U.S. Food and Drug Administration (FDA).

Context Therapeutics Inc. announced that the first patient has been dosed in the Phase 1 clinical trial of CT-95, a mesothelin ("MSLN") x CD3 T cell engaging ("TCE") bispecific antibody designed to target mesothelin-expressing cancers. The Company anticipates sharing initial data for the CT-95 Phase 1 trial in mid-2026.

Tonix Pharmaceuticals Holding Corp. announced a collaborative research agreement under which Tonix and Makana will study Tonix's anti-CD40L (CD40 ligand, also called CD154) monoclonal antibody candidate, TNX-1500, in combination with Makana's human-compatible organs and cells for the treatment of organ failure.

Atea Pharmaceuticals, Inc. announced that the first patient has been dosed in C-BEYOND, Atea's Phase 3 trial evaluating the regimen of bemnifosbuvir and ruzasvir for the treatment of adults with chronic hepatitis C virus (HCV).

Assembly Biosciences, Inc. announced data from its herpes simplex virus (HSV) program featured in three poster presentations at the 2025 Congress of the European Society of Clinical Microbiology and Infectious Diseases (ESCMID) taking place in Vienna, Austria, on April 11-15, 2025.

MediciNova, Inc announced enrollment of the first patient in the NIH-funded Expanded Access Program (EAP) trial to evaluate MN-166 (ibudilast) in patients with Amyotrophic Lateral Sclerosis (ALS).

Annexon, Inc. today presented data for its late-stage targeted therapy for GBS and showcased new disease education activities at the AAN Annual Meeting taking place April 5–9, 2025, in San Diego, California.

NeOnc Technologies Holdings, Inc announced that its Phase I clinical trial of NEO212, a development-stage bio-conjugated therapeutic for brain cancer, is nearing full enrollment. The final cohort (Cohort 5) is expected to complete the study's dosing protocol, marking a major milestone in the drug's development timeline.

Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) approved Opdivo®(nivolumab) plus Yervoy® (ipilimumab) as a first-line treatment of adult and pediatric patients (12 years and older) with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC).

Avidity Biosciences, Inc. announced that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation (ODD) to delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1), an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.

SELLAS Life Sciences Group, announced Cohort 3 data from the ongoing Phase 2 trial of SLS009 (tambiciclib), a highly selective CDK9 inhibitor, in relapsed/refractory acute myeloid leukemia (r/r AML).

Outlook Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has acknowledged receipt of the resubmission of the Biologics License Application (BLA) for ONS-5010 (bevacizumab-vikg), an investigational ophthalmic formulation of bevacizumab for the treatment of wet AMD.

Outlook Therapeutics, Inc announced that he FDA set a Prescription Drug User Fee Act (PDUFA) goal date of August 27, 2025.

Catalyst Pharmaceuticals, Inc today reported that its sub-licensee in Canada, Kye Pharmaceuticals, Inc. (Kye), has announced that Health Canada has accepted the New Drug Submission (NDS) for AGAMREE®, a novel corticosteroid treatment for Duchenne muscular dystrophy (DMD), for review.

Harmony Biosciences Holdings, Inc announced the presentation of updated data from its Open-Label Extension (OLE) study (ZYN2-CL-017) evaluating the safety and effectiveness of ZYN002 in children, adolescents, and adults with Fragile X syndrome (FXS).

Rallybio Corporation announced the discontinuation of the RLYB212 program for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT).

SCYNEXIS, Inc. announced the presentation of preclinical efficacy data on its second-generation fungerp candidate SCY-247 at the European Society of Clinical Microbiology and Infectious Diseases (ESCMID Global) in Vienna, Austria being held from April 11-15, 2025.

Recursion announced that the first patient has been dosed in the Phase 1 EXCELERIZE clinical study evaluating REC-3565 for the treatment of relapsed or refractory B-cell lymphomas.

Clene, Inc. announced new evidence of remyelination and neuronal repair in MS participants following treatment with CNM-Au8® 30 mg from analyses of the VISIONARY-MS Trial long-term open-label extension study.

Opus Genetics, Inc. announced one-month clinical data from the first pediatric patient treated with its investigational gene therapy, OPGx-LCA5, in a Phase 1/2 open-label trial for LCA5-related inherited retinal disease (IRD).

NeuroPace, Inc. announced three-year effectiveness data from the Post-Approval Study (PAS) of the RNS® System, which showed an 82% median reduction in seizures in adults treated with brain-responsive stimulation for drug-resistant focal epilepsy (DRE).1

TG Therapeutics, Inc. announced the presentation of data highlighting BRIUMVI® (ublituximab-xiiy) in patients with relapsing forms of multiple sclerosis (RMS), at the American Academy of Neurology 2025 annual meeting. Links to each presentation are included below.

Kura Oncology, Inc announced Kura submitted a New Drug Application (NDA) for ziftomenib, a highly selective, once-daily, oral, investigational menin inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM 1) mutation to the U.S. Food and Drug Administration (FDA) on March 31, 2025.

Cartesian Therapeutics announced 12-month efficacy and safety data from the Phase 2b trial of Descartes-08 in participants with generalized myasthenia gravis (MG).

Enanta Pharmaceuticals, Inc announced that data from the Company's Phase 2a human challenge study of EDP-323 in healthy adults infected with respiratory syncytial virus (RSV) has been accepted for an oral presentation as an ePoster at the European Society of Clinical Microbiology & Infectious Diseases Global 2025 (ESCMID, formerly ECCMID) being held April 11-15, 2025 in Vienna, Austria.

Theratechnologies Inc. announced that the U.S. Food and Drug Administration (FDA) has approved the Company's Prior Approval Supplement (PAS) to the supplemental biologics license application (sBLA) for EGRIFTA SV® (tesamorelin for injection).

Lipella Pharmaceuticals Inc. announced the completion of enrollment in its Phase 2a multicenter, dose-ranging trial evaluating LP-310, a liposomal tacrolimus oral rinse for the treatment of oral lichen planus (OLP).

AbbVie announced that the European Commission (EC) granted marketing authorization to RINVOQ® (upadacitinib; 15 mg, once daily) for the treatment of giant cell arteritis (GCA) in adult patients.

NeuroSense Therapeutics Ltd. provides a business update.

Halozyme Therapeutics, Inc. announced that Janssen-Cilag International NV, a Johnson & Johnson company, has received European Commission (EC) marketing authorization of the subcutaneous (SC) formulation of RYBREVANT® (amivantamab), in combination with LAZCLUZE® (lazertinib), for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations.

Inspira™ Technologies announced the successful completion of the first human treatment using its U.S. Food and Drug Administration-cleared INSPIRA™ ART100 system in a critical life-support procedure.

Allogene Therapeutics, Inc announced that ALLO-329, an investigational dual-targeted CD19/CD70 allogeneic CAR T, has received three Fast Track Designations (FTD) from the U.S. Food and Drug Administration (FDA)

NKGen Biotech, Inc. announced the oral presentation of updated Phase 1 clinical data from the Phase 1/2a trial evaluating troculeucel, cryopreserved expanded autologous NK cell therapy, in patients with moderate AD at the International Conference on Alzheimer's and Parkinson's Diseases and Related Neurological Disorders (AD/PD™ 2025) in Vienna, Austria.

Tharimmune, Inc. announced preclinical data from its expanded pipeline with HS1940, a dual-target multispecific biologic engineered to bind to both Programmed Death-1 (PD-1) and Vascular Endothelial Growth Factor (VEGF), and HS3215, a dual-target biologic binding to Human Epidermal Growth Factors 2 (HER2) and 3 (HER3) receptors.

Amneal Pharmaceuticals, Inc announced a new analysis of the pivotal RISE-PD Phase 3 study showed that patients who successfully converted to CREXONT from immediate release (IR) carbidopa/levodopa (CD/LD) experienced statistically significant improvements in sleep quality.

errica Pharmaceuticals Inc provided a business update.

ALX Oncology Holdings Inc announced receipt of U.S. Food and Drug Administration (FDA) clearance for the Investigational New Drug (IND) application for ALX2004, the company's potential best- and first-in-class antibody-drug conjugate (ADC) for the treatment of epidermal growth factor receptor (EGFR)-expressing solid tumors.

Vanda Pharmaceuticals Inc announced participation at the American Academy of Neurology (AAN) Annual Meeting, to be held in San Diego, California from April 5 through April 9, 2025.

TG Therapeutics, Inc. announced the publication of two journal articles one describing the evolution of CD20 treatments for multiple sclerosis (MS) and the other detailing the experience of seven individuals with MS who switched to BRIUMVI® (ublituximab-xiiy) from a different anti-CD20 monoclonal antibody therapy due to efficacy or tolerability concerns.

Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental Biologics License Application (sBLA) for AJOVY® (fremanezumab-vfrm) to expand the indication to include the prevention of episodic migraine in children and adolescent patients aged 6-17 years who weigh 45 kilograms (99 pounds) or more.

CureVac N.V announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for a Phase 1 clinical study of CVHNLC in patients with squamous non-small cell lung cancer (sqNSCLC).

iBio, Inc announced data from a non-GLP non-human primate (NHP) pharmacokinetics (PK) study suggesting IBIO-600, the company's novel lead asset and a potentially best-in-class long-acting anti-myostatin antibody designed for subcutaneous administration, could provide a significantly extended half-life in humans and a weight loss treatment option while preserving and promoting muscle growth.

Rhythm Pharmaceuticals, Inc. announced positive topline results from the pivotal Phase 3 TRANSCEND trial evaluating setmelanotide, a melanocortin-4 receptor (MC4R) agonist, for the treatment of acquired hypothalamic obesity.

Pharvaris provided a business update.

Lexeo Therapeutics, Inc announced positive interim data across all dose cohorts of LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy. .

Rhythm Pharmaceuticals, Inc. announced the Company will hold a conference call and webcast on Monday, April 7, 2025 at 8:00 a.m. ET to disclose topline results from the Pivotal Phase 3 TRANSCEND trial evaluating setmelanotide, a melanocortin-4 receptor (MC4R) agonist, in patients with acquired hypothalamic obesity.

Beam Therapeutics Inc. presented additional data from the Phase 1/2 clinical trial of BEAM-302 in patients with alpha-1 antitrypsin deficiency (AATD) at the 2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress, taking place April 4-5, 2025, in Lisbon, Portugal.

Biohaven announced that it will present 13 abstracts at the 2025 American Academy of Neurology (AAN) Annual Meeting, taking place from April 5 to April 9, 2025 in San Diego, California.

Anavex Life Sciences Corp. announced that over three years of continuous treatment with blarcamesine (ANAVEX®2-73) demonstrated significantly amelioration on clinical decline showing continued clinically and meaningful benefit for early Alzheimer's disease patients.

Sonnet BioTherapeutics announced positive safety results of SON-1010 (IL12-FHAB) at the highest dose combined with atezolizumab in the Phase 1b/2a clinical trial in adult patients with advanced solid tumors or platinum-resistant ovarian cancer (PROC) (the SB221 study).

NeuroSense Therapeutics, Ltd. announced that two members of its Scientific Advisory Board will present new data from the Company's Phase 2b trial during the General Neurology and Late Breaker sessions at the 77th Annual American Academy of Neurology (AAN) Meeting.

Elutia Inc. announced that EluPro™, the first ever FDA-cleared antibiotic-eluting bioenvelope was honored with a 2025 Bronze Edison Award™ for its innovative approach to reducing post-surgical complications.

Johnson & Johnson announced that the TREMFYA® (guselkumab) Phase 3b APEX study achieved both its primary endpoint (ACR20a) of reducing signs and symptoms and its major secondary endpoint of reducing progression of structural damage as measured by radiographic progression at 24 weeks, in adults living with active psoriatic arthritis (PsA), compared to placebo.1

Arvinas, Inc. today presented data from the first-in-human clinical trial of ARV-102, the Company's investigational PROteolysis TArgeting Chimera (PROTAC) leucine-rich repeat kinase 2 (LRRK2) degrader.

Axsome Therapeutics, Inc announced multiple presentations spanning its innovative, industry-leading psychiatry and neurology portfolio at the 2025 American Academy of Neurology (AAN) Annual Meeting, being held April 5-9 in San Diego, California..

Annexon, Inc. today highlights the company's leadership in advancing clinical research and education for GBS at the AAN Annual Meeting taking place April 5–9, 2025, in San Diego, California.

Amgen announced that the U.S. Food and Drug Administration (FDA) has approved UPLIZNA as the first and only treatment for adults living with Immunoglobulin G4-related disease (IgG4-RD). IgG4-RD is a chronic and debilitating immune-mediated inflammatory condition that can affect multiple organs.

Aldeyra Therapeutics, Inc announced receipt of a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for the resubmission of the New Drug Application (NDA) of reproxalap, an investigational drug candidate, for the treatment of dry eye disease.

Greenwich Lifesciences Provides Update On Its Phase III Clinical Trial, Flamingo-01, Which Is Evaluating GLSI-100, An Immunotherapy To Prevent Breast Cancer Recurrences

Akebia Therapeutics®, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the European Commission (EC) to approve XOANACYL® (Ferric Citrate as Coordination Complex) for the treatment of concomitant elevated serum phosphorous and iron deficiency in adult patients with chronic kidney disease (CKD).

Cingulate announced that it held a Pre-NDA meeting with the U.S. Food and Drug Administration (FDA) yesterday to discuss the submission of a new drug application (NDA) for its lead Phase 3 asset CTx-1301 (dexmethylphenidate HCl) for the treatment of Attention Deficit/Hyperactivity Disorder (ADHD).

Amneal Pharmaceuticals, Inc. announced that three large insurance coverage accounts, the Veterans Administration (VA), UnitedHealthcare and CVS Health, have added CREXONT® (carbidopa and levodopa) extended-release capsules for the treatment of Parkinson's disease (PD) to their national prescription drug formularies.

Pacira BioSciences, Inc. announced the first patient has been dosed in the Phase 2 ASCEND study of PCRX-201 (enekinragene inzadenovec) for the treatment of osteoarthritis, or OA, of the knee.

Enlivex Therapeutics Ltd. announced that the first patient has been dosed in an investigator-initiated Phase I trial to evaluate the safety, tolerability and initial efficacy of Allocetra™ for injection into the temporomandibular joint (TMJ) in patients suffering from TMJ osteoarthritis.

Vera Therapeutics, Inc announced that it has completed full enrollment in the pivotal ORIGIN Phase 3 trial of atacicept in patients with IgA Nephropathy (IgAN).

Alterity Therapeutics announced that an oral presentation and a poster presentation related to Alterity's clinical programs in Multiple System Atrophy (MSA) will be delivered at the American Academy of Neurology (AAN) 2025 Annual Meeting taking place April 5 - 9, 2025 in San Diego, CA.

Intellia Therapeutics announced the first patient has been dosed in MAGNITUDE-2, a global, pivotal Phase 3 trial of nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).

Incannex Healthcare Inc announced that the Company has completed Phase 2 patient enrollment in the global Phase 2/3 RePOSA study of IHL-42X for the treatment of Obstructive Sleep Apnea (OSA).

Castle Biosciences, announced the recent publication of two papers in the World Journal of Surgical Oncology and Cancer Medicine sharing reports from the prospective, multicenter DECIDE study demonstrating the significant impact of the Company's DecisionDx-Melanoma test on SLNB decision-making for patients with melanoma.3,4

BeiGene, Ltd announced the discontinuation of its clinical development program for ociperlimab (BGB-A1217), an anti-TIGIT antibody, as a potential treatment for lung cancer.

Neurogene Inc announced a peer-reviewed publication on its EXACT™ transgene regulation technology.

Mesoblast today provided an update on its plans to meet with the United States Food and Drug Administration (FDA) to discuss the accelerated approval pathway for Revascor® (rexlemestrocel-L) in the treatment of patients with ischemic chronic heart failure with reduced ejection fraction (HFrEF) and inflammation.

Novartis announced the US Food and Drug Administration (FDA) has granted accelerated approval for Vanrafia® (atrasentan), a potent and selective endothelin A (ETA) receptor antagonist, for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression.

BioMarin Pharmaceutical Inc announced that the Phase 3 PEGASUS trial evaluating PALYNZIQ® (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone.

Alpha Tau Medical Ltd. announced that the FDA has approved an Investigational Device Exemption (IDE) application to initiate a pilot study for the treatment of patients with recurrent glioblastoma (GBM) using the Alpha DaRT technology.

Denali Therapeutics Inc announced that the company's initiation of a rolling submission of a biologics license application (BLA) for accelerated approval of tividenofusp alfa for the treatment of Hunter syndrome (MPS II) has been received by the Center for Drug Evaluation and Research (CDER) of the U.S. Food and Drug Administration (FDA).

Tiziana Life Sciences, Ltd. announced dosing has commenced at Johns Hopkins University (JHU) Autoimmunity Center of Excellence, the third clinical site in its Phase 2 trial evaluating intranasal foralumab for the treatment of non-active Secondary Progressive Multiple Sclerosis (na-SPMS).

Palvella Therapeutics, Inc announced a late-breaking oral presentation at the upcoming 15th World Congress of Pediatric Dermatology, taking place April 8-11, 2025, in Buenos Aires, Argentina..

Hoth Therapeutics, Inc. announced groundbreaking preclinical data supporting the therapeutic potential of its lead Alzheimer's candidate, HT-ALZ, in improving cognitive function and reducing neuroinflammation in Alzheimer's disease (AD).

Quoin Pharmaceuticals Ltd. announces additional highly positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome (NS) study.

Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BIIB080, an investigational antisense oligonucleotide (ASO) therapy targeting tau, for the treatment of Alzheimer's disease.

Acumen Pharmaceuticals, presented extended results from its validated research-use plasma pTau217 assay to screen potential participants in the ongoing Phase 2 ALTITUDE-AD clinical trial of sabirnetug, showing this strategy has performed as intended.

Vigil Neuroscience presented data highlighting its oral small molecule program, including its lead clinical candidate VG-3927, in two oral presentations at the AD/PD™ 2025 International Conference on Alzheimer's and Parkinson's Diseases being held April 1 – April 5 in Vienna, Austria.

AC Immune SA announced additional interim safety and positive immunogenicity data from the Phase 2 VacSYn clinical trial evaluating ACI-7104.056, its wholly owned anti-alpha-synuclein (a-syn) active immunotherapy candidate, for the treatment of patients with early Parkinson's disease (PD).

Greenwich LifeSciences announced the following update on FLAMINGO-01 open label immune response data.

Edgewise Therapeutics, Inc announced positive top-line data of EDG-7500 from the Phase 2 CIRRUS-HCM four-week trial in participants with obstructive or nonobstructive HCM.

Applied DNA Sciences, announced that Applied DNA Clinical Labs (ADCL), its wholly-owned clinical laboratory subsidiary, is repositioning its TR8™ PGx pharmacogenomics testing service to offer tailored subpanels for indication-specific use cases in addition to full panel testing.

Connect Biopharma Holdings announced positive feedback from its Type C meeting with the U.S. Food and Drug Administration (FDA), Division of Pulmonology, Allergy, and Critical Care, in the Office of Immunology and Inflammation.

Castle Biosciences will share data via two poster presentations at the 11th World Congress of Melanoma and 21st European Association of Dermato-Oncology (EADO) Congress, being held April 3-5, 2025, in Athens, Greece.

Inventiva announced the completion of patient enrollment in its NATiV3 Phase 3 clinical trial with the randomization of the last patient in the main cohort.

GRI Bio reported interim safety results from its ongoing Phase 2a study evaluating GRI-0621 for Idiopathic Pulmonary Fibrosis (IPF).

Aquestive Therapeutics, Inc released topline results from its pediatric study for Anaphylm (epinephrine) sublingual film in patients aged seven to seventeen and weighing over 30 kgs with a personal history of allergic reactions.

RenovoRx, Inc. provided business updates

Moleculin Biotech, Inc., announced the first patient has been dosed in its Phase 3 pivotal trial evaluating Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).

Clearmind Medicine Inc. today the launch of its Electronic Data Capture (EDC) system to support its Phase I/II clinical trial of its MEAI-based treatment for Alcohol Use Disorder (AUD).

Candel Therapeutics, Inc. announced the publication of a manuscript reporting the results of a phase 1b clinical trial exploring safety and tolerability of the combination of CAN-2409 plus prodrug (valacyclovir) and nivolumab, in addition to standard of care (neurosurgery, radiotherapy, and temozolomide), in patients with newly diagnosed high-grade glioma.

MIRA Pharmaceuticals,announced the enrollment of the first subjects in its Phase 1 clinical trial of Ketamir-2, a novel oral ketamine analog in development for the treatment of neuropathic pain.

Cognition Therapeutics, presented biomarker results from the Phase 2 SHINE (COG0201) study of zervimesine (CT1812) in mild-to-moderate Alzheimer's disease at the AD/PD™ 2025 Alzheimer's & Parkinson's Diseases Conference taking place April 1-5, 2025 in Vienna, Austria.

Cardiol Therapeutics Inc. announced today its year-end 2024 update on operations following the filing of its audited Financial Statements and Management's Discussion and Analysis for the year ended December 31, 2024.

Alkermes plc announced the initiation of Vibrance-3, a phase 2 clinical study evaluating the safety and efficacy of ALKS 2680 compared to placebo in adults with idiopathic hypersomnia (IH).

Fractyl Health, Inc. announced positive early data from the open-label REVEAL-1 cohort of its ongoing REMAIN-1 pivotal study.

Compass Therapeutics, Inc. announced statistically significant top-line data on the primary efficacy endpoint for COMPANION-002, the Company's ongoing Phase 2/3 randomized trial of tovecimig (formerly CTX-009) in combination with paclitaxel in patients with advanced BTC.

Axsome Therapeutics, Inc. announced results of the PARADIGM Phase 3 proof-of-concept trial of solriamfetol in major depressive disorder (MDD) with and without severe excessive daytime sleepiness (EDS).

Apellis Pharmaceuticals, announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation of the supplemental New Drug Application (sNDA) for EMPAVELI® (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are severe and rare kidney diseases.

Pfizer Inc announced that the European Commission (EC) has issued a decision amending the marketing authorization for ABRYSVO®, the company's bivalent respiratory syncytial virus (RSV) prefusion F (RSVpreF) vaccine, to extend the indication to include prevention of lower respiratory tract disease (LRTD) caused by RSV in individuals 18 through 59 years of age