FDA Calendar

BridgeBio Pharma, Inc. announced it has surpassed its interim analysis enrollment target and expects topline interim data from its Phase 3 registrational study (FORTIFY) in individuals with LGMD2I/R9 in 2025.

Neurogene Inc announced that the first patient in Cohort 2 received high-dose NGN-401 gene therapy in the Phase 1/2 trial for female pediatric patients with Rett syndrome, and high-dose NGN-401 has been well-tolerated following dosing in May 2024.

Acadia Pharmaceuticals, Inc. announced that interim data from the open-label real-world LOTUS™ study will be presented at the 2024 International Rett Syndrome Foundation (IRSF) Annual Scientific Meeting, being held this week in Westminster, Colorado.

Lexaria Bioscience Corp. announces it has now hired a contract research organization ("CRO") to oversee execution of the Company's 12-week chronic study GLP-1-H24-4, (the "Study") which will evaluate various DehydraTECH-GLP-1 (glucagon-like peptide) formulations and other treatments with efficacy objectives of weight loss, blood sugar reduction, and more.

Relmada Therapeutics, Inc announced the publication of REL-1017 clinical data from the Reliance I Study in the peer-reviewed journal, The Journal of Clinical Psychiatry.

Day One Biopharmaceuticals announced it has entered into an exclusive licensing agreement (the Agreement) with MabCare Therapeutics (MabCare) for MTX-13, a novel ADC targeting protein-tyrosine kinase 7 (PTK7).

Aethlon Medical, Inc. announced that, on June, 13, 2024, the Human Research Ethics Committee (HREC) of the Central Adelaide Local Health Network (CALHN) granted full ethics approval for Aethlon's safety, feasibility and dose-finding clinical trial of the Hemopurifier® in cancer patients with solid tumors who have stable or progressive disease during anti-PD-1 monotherapy treatment, such as Keytruda® (pembrolizumab) or Opdivo® (nivolumab) (AEMD-2022-06 Hemopurifier Study).

Lexicon Pharmaceuticals, Inc. announced that the peer-reviewed Journal of the American College of Cardiology: Heart Failure has published a research paper concluding that INPEFA® (sotagliflozin) is cost-effective for people with diabetes and recent worsening heart failure using commonly accepted willingness-to-pay thresholds.

Annexon, Inc. announced the Company will have several presentations on the Company's ANX005 Guillain-Barré Syndrome (GBS) program at the 2024 Peripheral Nerve Society (PNS) Annual Meeting being held June 22-25, 2024 at the Palais des congrès de Montréal in Montréal, Canada.

Assembly Biosciences, Inc. announced that the first participant has been dosed in the Phase 1b trial of ABI-4334, a next-generation capsid assembly modulator (CAM) candidate in development for the treatment of chronic hepatitis B virus (HBV) infection.

4D Molecular Therapeutics announced that the Company will present the initial interim 24-week landmark analysis from the Population Extension cohort of the PRISM Phase 2 Clinical Trial evaluating 4D-150 in a broad wet AMD patient population at the ASRS Annual Scientific Meeting being held in Stockholm, Sweden from July 17-20, 2024.

FibroGen, Inc. announced it will host the second part of its virtual KOL investor event series on Wednesday, June 26, 2024 at 10:00 AM ET.

Takeda announced data from the Phase 3 ADVANCE-CIDP 3 clinical trial, a long-term extension study evaluating the safety and efficacy of HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] in patients with chronic inflammatory demyelinating polyneuropathy (CIDP).

Daxor Corporation announces today it will exhibit at the American Association of Heart Failure Nurses (AAHFN) 20th Annual Meeting from June 20-22nd, 2024 at the Rosen Shingle Creek Resort in Orlando, Florida.

Erasca, Inc announced the initiation of the global SEACRAFT-2 Phase 3 trial evaluating the pan-RAF inhibitor naporafenib in combination with the MEK inhibitor trametinib (MEKINIST®) in patients with NRAS-mutant (NRASm) melanoma.

Erasca, Inc announced that Randomized Stage 1 readout for naporafenib plus trametinib vs. trametinib monotherapy expected in 2025

ProQR Therapeutics NV announced presentation will detail AX-0810, the Company's program targeting NTCP for cholestatic diseases, including further assessment in in vivo models of biomarkers for NTCP.

ProQR Therapeutics NV announced it will participate in the RNA Editing Summit, June 18-20, 2024, in Boston, Massachusetts.

BridgeBio Pharma, Inc. announced it has surpassed its interim analysis enrollment target and expects topline interim data from its Phase 3 registrational study (FORTIFY) in individuals with LGMD2I/R9 in 2025.

Intra-Cellular Therapies, announced positive topline results from Study 502 evaluating lumateperone 42 mg as an adjunctive therapy to antidepressants for the treatment of MDD.

Spyre Therapeutics, Inc. announced that it has initiated dosing of healthy volunteers in its first clinical trial of SPY001, an investigational novel half-life extended anti-α4β7 monoclonal antibody.

Altimmune, Inc. announced oral presentations on pemvidutide, an investigational GLP-1/glucagon dual receptor agonist in development for the treatment of obesity and MASH, at the American Diabetes Association's (ADA) 84th Scientific Sessions, to be held in Orlando, FL from June 21-24, 2024.

Taysha Gene Therapies, Inc. announced positive longer-term clinical data from the ongoing REVEAL Phase 1/2 adolescent and adult trial and initial clinical data from the REVEAL Phase 1/2 pediatric trial evaluating TSHA-102 in Rett syndrome.

Orsini Specialty Pharmacy and Zevra Therapeutics, Inc announced that Orsini is now the pharmacy partner for OLPRUVA® (sodium phenylbutyrate) for oral suspension. OLPRUVA® is a prescription medicine used along with certain therapies, including changes in diet, for long-term management of certain adult and pediatric patients with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinic acid synthetase (AS)..

Zentalis® Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on the following studies of azenosertib: the Phase 1 ZN-c3-001 dose-escalation study in solid tumors, the Phase 2 ZN-c3-005 (DENALI) study in platinum-resistant ovarian cancer (PROC) and the Phase 2 ZN-c3-004 (TETON) study in uterine serous carcinoma (USC). This action follows two recent deaths due to presumed sepsis in the DENALI study.

REGENXBIO Inc. announced that Submission of a rolling BLA using the accelerated approval pathway expected to start in Q3 2024

REGENXBIO Inc announced it completed a successful Pre-Biologics License Application (BLA) meeting for RGX-121 for the treatment Mucopolysaccharidosis Type II (MPS II), where it finalized details of its BLA with the U.S. Food and Drug Administration (FDA).

Clene Inc , today presented new long-term CNM-Au8 treatment results for survival and neurofilament light (NfL) levels from the HEALEY ALS Platform Trial open label extension (OLE) at the European Network for the Cure of ALS (ENCALS) meeting in Stockholm, Sweden.

Immunocore Holdings plc announces randomization of the first patient in the PRISM-MEL-301 trial, assessing the efficacy and safety of brenetafusp (IMC-F106C; PRAME-A02), in combination with nivolumab, in first-line advanced or metastatic cutaneous melanoma.

Chemomab Therapeutics announced a new scientific publication that further confirms the important role of the soluble protein CCL24 in the pathologies underlying the rare fibrotic liver disease primary sclerosing cholangitis (PSC). The new study reinforces the extensive evidence showing the potential of Chemomab's CCL24-neutralizing antibody, CM-101, to interrupt the biological processes driving PSC disease progression and severity.