FDA

Quoin Pharmaceuticals Ltd. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the Company’s lead asset, QRX003, for the treatment of Peeling Skin Syndrome (PSS).

NanoViricides, Inc. announces that its Phase II Clinical Trial of NV-387 Oral Gummies as a Treatment for the Current Bundibugyo Ebolavirus and other Ebola viruses in the Democratic Republic of Congo ("DRC") was successfully registered in the Pan African Clinical Trials Registry (pactr.samrc.ac.za) database.

Coya Therapeutics, Inc. announced the publication of preclinical results for COYA 303 in the International Journal of Molecular Sciences.

Intellia Therapeutics, today announced the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lonvo-z and granted the BLA Priority Review with a PDUFA target action date of March 10, 2027.

Structure Therapeutics today reported positive clinical trial results from its two lead product candidates: ACCG-2671, an oral, non-peptide, small molecule dual amylin and calcitonin receptor agonist (DACRA), and aleniglipron, an oral, non-peptide, small molecule glucagon-like peptide-1 (GLP-1) receptor agonist.

BioAge Labs, Inc. announced that the first participant has been dosed in QUELL-DME, a randomized, controlled Phase 2 trial evaluating once-daily oral BGE-102 in adults with diabetic macular edema (DME).

Avalyn Pharma Inc. announced new data demonstrating predicted aerosol distribution of AP01 and survey data highlighting patient perspectives on participation in clinical studies for idiopathic pulmonary fibrosis (IPF).

Dyne Therapeutics, Inc. announced that it will present additional one-year clinical data from the Phase 1/2 ACHIEVE trial of zeleciment basivarsen (z-basivarsen, also known as DYNE-101) for DM1 at the 31st Annual International Congress of the World Muscle Society (WMS) being held September 29, 2026, through October 3, 2026, virtually and in Hiroshima, Japan, as well as the 2026 Annual Meeting of the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) being held September 29, 2026, through October 2, 2026, virtually and in Orlando, Florida.

AstraZeneca Announces Full results from the positive Phase III OBERON and TITANIA trials showed that tozorakimab is the first biologic to demonstrate statistically significant and highly clinically meaningful efficacy in reducing moderate and severe exacerbations in a broad population of patients with chronic obstructive pulmonary disease (COPD) still experiencing exacerbations while on inhaled standard of care.1,2

Clearmind Medicine Inc. announced that the Yale University’s IRB has approved the initiation of patient recruitment for Parts B and C of its Phase I/II clinical trial evaluating CMND‑100.

BriaCell Therapeutics Corp announced that Memorial Sloan Kettering Cancer Center (MSK) has joined BriaCell’s ongoing pivotal Phase 3 study of Bria-IMT™ plus an immune checkpoint inhibitor in metastatic breast cancer (ClinicalTrials.gov identifier: NCT06072612).

BioVie Inc. announced next stage of engagement with the Food and Drug Administration (FDA) to advance the Company’s Phase 3 development program evaluating bezisterim in Parkinson’s disease.

Artiva Biotherapeutics, Inc. today provided an update on anticipated data disclosures and development milestones for its lead program, AlloNK® (also known as AB-101), through year-end 2026.

Pharvaris announced statistically significant and clinically meaningful topline results of the CHAPTER-3 pivotal Phase 3 study evaluating deucrictibant extended-release (XR) tablet for the prevention of HAE attacks.

X4 Pharmaceuticals announced the positive outcome of its Type C Meeting with the U.S. Food and Drug Administration (FDA) related to the global 4WARD Phase 3 clinical trial.

Nkarta, Inc. announced its selection for three scientific presentations at ACR Convergence 2026 being held November 6-11 in Orlando.

Adlai Nortye Group Ltd. announced that the first patient has been dosed with AN4035 in Australia, initiating the global Phase I trial in patients with CEACAM5-enriched RAS-addicted solid tumors.

Definium Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for DT120 (lysergide) ODT.

Monte Rosa Therapeutics, Inc. announced that its collaborator Novartis has initiated a Phase 2 clinical study for the VAV1-directed MGD MRT-6160 (DDY391) in people living with Sjögren’s disease (SjD), the second most prevalent rheumatic autoimmune disease1.

Vor Bio announced the completion of enrollment in UPSTREAM MG, its global Phase 3 registrational trial evaluating telitacicept for the treatment of generalized myasthenia gravis (gMG).

Bristol Myers Squibb announced positive Phase 2 results from the registrational QUINTESSENTIAL trial (NCT06297226) of arlocabtagene autoleucel (arlo-cel; BMS-986393) in adult patients with quadruple-class exposed relapsed and refractory multiple myeloma (RRMM).

Belite Bio, Inc announced the submission of a New Drug Application (NDA) to the Ministry of Health, Labour, and Welfare (MHLW) in Japan for tinlarebant in Stargardt disease type 1 (STGD1).

OS Therapies, Inc. announced a statistically significant benefit for OST-HER2-treated patients in an interim 3-year overall survival data analysis when compared with a combined published historical control arm being used for regulatory interactions.

Roivant announced positive results from its Phase 2 PHocus clinical trial evaluating mosliciguat for the treatment of pulmonary hypertension associated with interstitial lung disease (PH-ILD), a progressive and life-threatening condition with significant unmet medical needs for patients.

Beam Therapeutics Inc. announced updated clinical data from the Phase 1/2 trial evaluating BEAM-302 for alpha-1 antitrypsin deficiency (AATD).

Upstream Bio, Inc. today presented results from the Phase 2 VALIANT clinical trial evaluating the safety and efficacy of verekitug in adults with severe asthma in a late-breaking oral presentation at the European Respiratory Society (ERS) Congress 2026 in Barcelona.

Nyxoah SA announced that the first patient has been implanted in BREATHE, the Company’s U.S. post-approval study of the Genio® system. The procedure was performed by Rolando Molina, MD, otolaryngologist at South Florida ENT Associates.

Ascendis Pharma A/S announced it will share the latest data from its Endocrinology Rare Disease programs during ESPE 2026, the annual congress of the European Society for Paediatric Endocrinology (ESPE) being held September 8-10, 2026, in Marseille, France.

Pulmovant announced that results from the Phase 2 PHocus study of mosliciguat in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) will be presented at the European Respiratory Society (ERS) International Congress 2026 at 12:15 CEST (6:15 a.m. ET) on Tuesday, September 8, 2026, by Marc Humbert, MD, PhD, Professor of Respiratory Medicine at Université Paris-Saclay and Director of the French National Reference Center for Pulmonary Hypertension.

Inhibrx Biosciences, Inc. announced that it will host a live webcast presentation on Tuesday, September 8, 2026 at 5:00 a.m. Pacific Time, to provide primary endpoint results from the randomized and controlled Phase 2 portion of the HexAgon study, which is evaluating the safety and efficacy of INBRX-106, a hexavalent OX40 agonist, in combination with pembrolizumab (the combination arm) versus pembrolizumab monotherapy (the control arm) in first-line patients with treatment-naïve, PD-L1 positive (CPS ≥ 20) metastatic or unresectable recurrent Head and Neck Squamous Cell Carcinoma (HNSCC).

Guardant Health, Inc announced that the U.S. Food and Drug Administration (FDA) has approved Guardant360® CDx as a companion diagnostic (CDx) to identify patients with hormone receptor (HR)-positive, HER2-negative advanced or metastatic breast cancer harboring tumors with ESR1 mutations before radiographic progression who may benefit from treatment with ETCAMAH™ (camizestrant), AstraZeneca’s next-generation oral selective estrogen receptor degrader (SERD).

AstraZeneca AstraZeneca’s ETCAMAH® (camizestrant) in combination with a cyclin-dependent kinase (CDK) 4/6 inhibitor (abemaciclib, palbociclib or ribociclib) has been approved in the US for the treatment of adult patients with hormone receptor (HR)-positive, HER2-negative, locally advanced or metastatic breast cancer upon detection of ESR1 mutation during aromatase inhibitor (AI) and CDK4/6 inhibitor therapy, based on a US Food and Drug Administration (FDA)-authorized test.

Royalty Pharma plc announced an update on Novartis’ Phase 3 results from the HORIZON outcomes trial for pelacarsen, an antisense oligonucleotide studied in patients with atherosclerotic cardiovascular (CV) disease and elevated lipoprotein(a), or Lp(a).

Rein Therapeutics announced that it will present a late-breaking poster at the European Respiratory Society (ERS) 2026 Congress taking place September 5-9, 2026, in Barcelona, Spain.