FDA assigns a Prescription Drug User Fee Act (PDUFA) date of July 11, 2026 REDWOOD CITY, Calif. / Sep 10, 2025 / Business Wire / Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, today announced that the U.S. Food and Drug...Read more
There are currently no approved vaccines or treatments for norovirus infection Cocrystal’s CDI-988 is the first antiviral for the potential prevention and treatment of viral gastroenteritis caused by norovirus infections Phase 1b study is expected to start by year-end 2025 BOTHELL, Wash., Sept. 08, 2025 (GLOBE NEWSWIRE) -- Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) announces that the Company received a...Read more
If approved, ZORYVE cream would be the first and only topical PDE4 inhibitor indicated for plaque psoriasis in children as young as 2, offering patients and caregivers an important alternative to topical steroids and vitamin-D analogs ZORYVE cream is uniquely formulated to be effective, safe, and well tolerated for all areas of the body, including sensitive areas such as intertriginous skin, where plaque psoriasis often presents in...Read more
Filing based on pivotal Phase 3 ORCA-2 and ORCA-3 clinical trials demonstrating statistically significant and clinically meaningful smoking cessation FDA acceptance starts the review process and sets timing for potentially the first new FDA-approved pharmacotherapy in two decades PDUFA targeted date set for June 20, 2026 SEATTLE and VANCOUVER, British Columbia, Sept. 03, 2025 (GLOBE NEWSWIRE) -- Achieve Life Sciences, Inc....Read more
Dosing of first patient in Phase 1 dose escalation trial of IDE892 targeted in 4Q 2025 Preclinical profile of IDE892 and mechanistic combination rationale with IDE397, IDEAYA's proprietary MAT2A inhibitor, will be presented at the 10-Year Anniversary R&D Day on September 8th SOUTH SAN FRANCISCO, Calif., Sept. 3, 2025 /PRNewswire/ -- IDEAYA Biosciences, Inc. (Nasdaq: IDYA), a leading precision medicine oncology company, announced...Read more
FDA Establishes April 23, 2026 as PDUFA Target Date for Review of Submission Seeking Approval for GTx-104 in the Treatment of Patients with aneurysmal Subarachnoid Hemorrhage (aSAH) Comprehensive Data Package Supported by Positive Results from Phase 3 STRIVE-ON Safety Trial of GTx-104 PRINCETON, N.J., Aug. 27, 2025 (GLOBE NEWSWIRE) -- Grace Therapeutics, Inc. (Nasdaq: GRCE) (Grace Therapeutics or the Company), a late-stage,...Read more
New Drug Application (NDA) for low-dose contrast agent gadoquatrane seeks approval for contrast-enhanced magnetic resonance imaging (MRI) of the central nervous system (CNS) and other body regions for adults and pediatric patients including neonates Submitted dose corresponds to a 60 percent reduction in gadolinium compared to macrocyclic gadolinium-based contrast agents (GBCAs) dosed at 0.1 mmol gadolinium per kilogram of body...Read more
FDA acceptance enables initiation of a Phase 2 multicenter, double-blind, placebo-controlled clinical study to evaluate the efficacy and safety of COYA 302 in patients with ALS HOUSTON, Aug. 25, 2025 /PRNewswire/ -- Coya Therapeutics, Inc. (NASDAQ: COYA) ("Coya" or the "Company"), a clinical-stage biotechnology company focused on developing biologics that enhance regulatory T cell (Treg) function in patients with...Read more
SL-325 is a potentially first-in-class DR3 antagonist antibody being developed for the treatment of Inflammatory Bowel Disease (IBD) and other inflammatory and immune-mediated diseases The Company is on track to dose the first participant in its Phase 1 SL-325 clinical trial in healthy volunteers in Q3 2025 Current cash and cash equivalents, plus the anticipated aggregate proceeds of up to $103 million from recent private placement,...Read more
U.S. Food and Drug Administration accepts sNDA for filing with priority review; sets PDUFA goal date of December 20, 2025 European Medicines Agency accepts and validates submission for Type II variation MAA for setmelanotide to treat acquired hypothalamic obesity Company to host “Commercial Readiness for Acquired Hypothalamic Obesity,” event for investors and analysts on Wednesday, September 24, in Boston BOSTON,...Read more
INDs Transferred from Statera Biopharma Advance Entolimod for ARS Along Regulatory Pathway and Enable Pursuit of Clinical Trial Program in Neutropenia and Lymphocyte Exhaustion FREMONT, Calif. / Aug 20, 2025 / Business Wire / Tivic Health® Systems, Inc. (Nasdaq: TIVC), a diversified immunotherapeutics company, today announced it has received, from Statera Biopharma, two investigational new drug applications (INDs) for its lead...Read more
NEW YORK, Aug. 11, 2025 (GLOBE NEWSWIRE) -- Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies with its lead development candidate, intranasal foralumab, a fully human, anti-CD3 monoclonal antibody, is pleased to announce that the U.S. Food & Drug Administration (FDA) have approved the IND for its Phase 2a clinical trial of intranasal...Read more
Filing based on pivotal Phase 3 VERITAC-2 clinical trial demonstrating statistically significant and clinically meaningful improvement in median progression-free survival versus fulvestrant Vepdegestrant is the first PROteolysis TArgeting Chimera (PROTAC) to demonstrate clinical benefit in patients with breast cancer NEW HAVEN, Conn., Aug. 08, 2025 (GLOBE NEWSWIRE) -- Arvinas, Inc. (Nasdaq: ARVN), today with its partner...Read more
Regulatory filing marks significant milestone for medication designed to provide once-daily, entire active-day symptom control in ADHD patients KANSAS CITY, Kan., Aug. 06, 2025 (GLOBE NEWSWIRE) -- Cingulate Inc. (NASDAQ: CING), a biopharmaceutical company utilizing its proprietary Precision Timed Release™ (PTR™) drug delivery platform technology to build and advance a pipeline of next-generation pharmaceutical products, today...Read more
Novel combination therapy with STAR-001 targets the most aggressive and deadliest forms of brain cancer at first progression GBM represents an annual market opportunity of $3 to $5 billion USD with an accelerating number of cases globally Lantern Pharma has previously received both FDA Orphan and Fast Track Designations in GBM DALLAS / Aug 06, 2025 / Business Wire / Starlight Therapeutics, a wholly owned subsidiary of Lantern Pharma...Read more
Designed to expand PSMA PET imaging access for patients by increasing batch size by ~50% and enhancing supply resilience PDUFA date set for March 6, 2026 BEDFORD, Mass., Aug. 06, 2025 (GLOBE NEWSWIRE) -- Lantheus Holdings, Inc. (“Lantheus”) (NASDAQ: LNTH), the leading radiopharmaceutical-focused company committed to enabling clinicians to Find, Fight and Follow disease to deliver better patient outcomes, today announced that...Read more
CID-103 is a potential best-in-class, anti-CD38 monoclonal antibody; binds to unique CD38 epitope Phase 1 study in adults with active and chronic active renal allograft AMR planned to initiate AMR is a leading cause of kidney transplant loss, leading to dialysis and/or repeat renal transplant SAN FRANCISCO, CA / ACCESS Newswire / August 4, 2025 / CASI Pharmaceuticals, Inc. (NASDAQ:CASI), a clinical-stage...Read more
Progress Follows Positive Immune Response Observed in Phase 1 Study SAN JOSE, Calif., Aug. 4, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that, in collaboration with Cleveland Clinic, it has initiated the transfer of the Investigational New Drug (IND) application that supported the Phase 1...Read more
Combination therapy has potential to be an all-oral, fixed-duration regimen for previously untreated patients with CLL Application is supported by data from the Phase 3 AMPLIFY trial that showed statistically significant improvement in progression-free survival vs chemoimmunotherapy Regimen offers an opportunity for patients to take time off treatment, an important step toward improved disease management NORTH CHICAGO, Ill., July...Read more
Oral, non-opioid drug candidate advancing through Phase 1 and nearing completion of the single ascending dose (SAD) portion as the Company prepares to launch U.S. Phase 2a by year-end MIAMI, FLORIDA / ACCESS Newswire / July 29, 2025 / MIRA Pharmaceuticals, Inc. (NASDAQ:MIRA), a clinical-stage pharmaceutical company developing novel therapies for neurologic, neuropsychiatric, and metabolic disorders, today announced that the U.S. Food...Read more
Second antibody-drug conjugate (ADC) to progress into clinical development utilizing our proprietary payload and optimized antibody Preclinical results demonstrate strong anti-tumor activity and favorable tolerability profile Phase 1 clinical trial evaluating ZW251 in hepatocellular carcinoma (HCC) expected to be initiated in 2025 VANCOUVER, British Columbia, July 28, 2025 (GLOBE NEWSWIRE) -- Zymeworks Inc. (Nasdaq: ZYME), a...Read more
Icotrokinra is a first-in-class investigational targeted oral peptide that selectively blocks the IL-23 receptor Filing based on unprecedented data package that met all primary endpoints across four Phase 3 studies, including head-to-head superiority comparisons versus deucravacitinib and evaluation of difficult to treat skin sites Submission underscores potential to offer moderate-to-severe plaque psoriasis patients the standout...Read more
Icotrokinra is a first-in-class investigational targeted oral peptide that selectively blocks the IL-23 receptor Filing based on unprecedented data package that met all primary endpoints across four Phase 3 studies, including head-to-head superiority comparisons versus deucravacitinib and evaluation of difficult to treat skin sites Submission underscores potential to shift the treatment paradigm for moderate-to-severe plaque...Read more
WATERTOWN, Mass., July 21, 2025 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced positive feedback from its pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) to discuss the planned NDA submission for...Read more
Company focused on obtaining approval for outpatient (at-home) use of IGALMI® for the acute treatment of agitation associated with bipolar disorders or schizophrenia Pre-Supplemental New Drug Application meeting with FDA scheduled for August 20, 2025 NEW HAVEN, Conn., July 21, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative...Read more
Applications supported by positive results from the pivotal Phase 3 POETYK PsA-1 and POETYK PsA-2 clinical trials, in which significantly more patients treated with Sotyktu achieved ACR20 response compared with placebo at Week 16 U.S. Food and Drug Administration assigned a target action date of March 6, 2026 PRINCETON, N.J. / Jul 21, 2025 / Business Wire / Bristol Myers Squibb (NYSE:BMY) today announced that the U.S. Food and Drug...Read more
New Drug Application supported by results from the Phase 2b SunRISe-1 study RARITAN, N.J., July 17, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced that the U.S. Food and Drug Administration (FDA) granted Priority Review to the New Drug Application (NDA) filed for TAR-200, an intravesical gemcitabine releasing system, for the treatment of patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk...Read more
PDUFA Target Action Date December 16, 2025 LEXINGTON, Mass. / Jul 17, 2025 / Business Wire / Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmitted New Drug Application (NDA) for topical ocular reproxalap, a first-in-class investigational new drug candidate, for the treatment of the signs and symptoms of dry eye disease. The FDA...Read more
REDWOOD CITY, Calif. / Jul 14, 2025 / Business Wire / Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for its proprietary, selective...Read more
Doravirine/islatravir would be the first FDA-approved two-drug regimen without an integrase inhibitor that demonstrated non-inferior efficacy and a generally comparable safety profile to the three-drug InSTI-based regimen, BIC/FTC/TAF, in a Phase 3 pivotal trial RAHWAY, N.J. / Jul 10, 2025 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that the U.S. Food and Drug...Read more
SGT-501 is a novel gene therapy for rare, life-threatening, genetic arrhythmogenic disease with no approved therapies SGT-501 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA Solid expects to initiate Phase 1b clinical trial of SGT-501 in the fourth quarter of 2025 Expands Solid’s clinical pipeline to include first cardiac indication with urgent unmet medical...Read more
Accepted IND application allows for development program to move directly to a Phase 2 pivotal registration clinical trial Phase 2 clinical trial initiation anticipated in first half of 2026 Pediatric cardiomyopathies affect at least 100,000 children worldwide Effective treatment options are limited, with nearly 40% of children with DCM requiring a heart transplant or dying within two years of diagnosis MIAMI, July 08, 2025 (GLOBE...Read more
Submission is based on long-term Phase 3 data demonstrating 63 percent reduction in risk of relapse in adults with schizophrenia compared to placebo CAPLYTA® is FDA approved to treat schizophrenia and is the first and only approved treatment for bipolar I and II depression as an adjunctive and monotherapy With the addition of CAPLYTA® to Johnson & Johnson's robust portfolio of therapies, the Company now offers the broadest...Read more
NDA assigned a Target Action Date of February 25, 2026 Product has patent protection through 2044 DEER PARK, Ill., July 08, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the Company’s New Drug Application (NDA) for ET-600, a proprietary patented formulation...Read more
US phase 1 trial of ‘2510 is expected to be initiated before the end of 2025 Anticipate ImmuneOnco sharing initial safety and efficacy results from phase 2 trial of ‘2510 + chemo in 1L NSCLC in the second half of 2025 DALLAS, July 02, 2025 (GLOBE NEWSWIRE) -- Instil Bio, Inc. (“Instil”) (NASDAQ: TIL), a clinical-stage biopharmaceutical company focused on developing a pipeline of novel therapies, today announced the clearance of...Read more
RP-A701 is a first-in-class gene therapy for the treatment of BAG3-associated dilated cardiomyopathy BAG3-associated dilated cardiomyopathy is a rare, inherited heart condition caused by mutations in the BAG3 gene, leading to early-onset, progressive heart failure due to impaired cardiac function, high morbidity, and premature mortality Rocket plans to conduct a Phase 1 trial in the U.S. and is working towards the first patient...Read more
Smoking, the leading cause of preventable death and disease, continues to affect nearly 29 million adults in the U.S. alone If approved, cytisinicline will be the first new FDA-approved pharmacotherapy option for nicotine dependence in two decades More than 2,000 clinical trial participants contributed to the body of evidence submitted in the NDA demonstrating cytisinicline’s efficacy, safety, and tolerability profile SEATTLE and...Read more
Submission Seeks Approval of GTx-104 in the Treatment of Patients with aneurysmal Subarachnoid Hemorrhage (aSAH) Comprehensive Data Package Includes Positive Results from Phase 3 STRIVE-ON Safety Trial of GTx-104 Submission Has the Potential to Trigger Exercise of up to $7.6 million in Gross Proceeds from 2023 Financing Warrants Tied to FDA Acceptance of NDA for Review PRINCETON, N.J., June 25, 2025 (GLOBE NEWSWIRE) -- Grace...Read more
Trial will be conducted by Angel Pharma and will study 12-week dosing of soquelitinib in patients with moderate-to-severe atopic dermatitis SOUTH SAN FRANCISCO, Calif., June 25, 2025 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, today announced that the IND application submitted by its partner in China, Angel Pharmaceuticals Ltd. (Angel Pharma), has been...Read more
Phase 1/2a clinical trial currently supported by a $3.0 million grant from the U.S. Department of Defense Trial will enroll children with high grade glioma and ependymoma HOUSTON, June 25, 2025 (GLOBE NEWSWIRE) -- Plus Therapeutics, Inc. (Nasdaq: PSTV) (the “Company”), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system (CNS) cancers, today...Read more
CUE-401, a first-in-class bispecific molecule designed to induce and expand Tregs in vivo through the co-activity of transforming TGF-β and a modified variant of IL-2 BOSTON, June 24, 2025 (GLOBE NEWSWIRE) -- Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of therapeutic biologics to selectively engage and modulate disease-specific T cells for the treatment of autoimmune...Read more
PDUFA action date set for October 25, 2025 sNDA being reviewed under FDA’s RTOR program Revumenib has the potential to become the first and only menin inhibitor approved in both R/R mNPM1 AML and R/R KMT2Ar acute leukemia NEW YORK, June 24, 2025 (GLOBE NEWSWIRE) -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today announced that the...Read more
DUBLIN, June 20, 2025 (GLOBE NEWSWIRE) -- GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients by developing a practice-changing treatment in depression, today announced that, in June, it submitted its complete response to the previously announced clinical hold of its Investigational New Drug Application (IND) for GH001 to the U.S. Food and Drug Administration...Read more
LEXINGTON, Mass. / Jun 17, 2025 / Business Wire / Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to discovering and developing innovative therapies designed to treat immune‑mediated and metabolic diseases, today announced the resubmission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for topical ocular reproxalap, an investigational new drug candidate, for the treatment...Read more
BOSTON and LONDON, June 16, 2025 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has cleared the IND to initiate a Phase 1 clinical study of ORX142 in healthy volunteers. ORX142 is an investigational, novel, highly potent and selective OX2R agonist being developed for the treatment of select neurological and...Read more
GLP-1/GIP receptor co-agonist designed to be highly potent and for once-monthly administration based on MBX’s novel PEP™ platform technology Phase 1 trial of MBX 4291 is expected to initiate in Q3 2025 CARMEL, Ind., June 16, 2025 (GLOBE NEWSWIRE) -- MBX Biosciences, Inc. (Nasdaq: MBX), a clinical-stage biopharmaceutical company focused on the discovery and development of novel precision peptide therapies for the treatment of...Read more
MALVERN, Pa., June 16, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy candidate being developed for all Stargardt disease...Read more
FDA assigns PDUFA target action date of January 31, 2026 WARREN, N.J., June 16, 2025 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ: AQST) (“Aquestive” or the “Company”), a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug...Read more
FDA is expected to notify Innoviva Specialty Therapeutics regarding its decision to conduct an Advisory Committee Meeting in the Day 74 letter If approved, zoliflodacin would be the first new antibiotic for treating gonorrhea in decades WALTHAM, Mass. & GENEVA / Jun 12, 2025 / Business Wire / Innoviva Specialty Therapeutics, Inc., a subsidiary of Innoviva, Inc. (NASDAQ: INVA), in collaboration with the Global Antibiotic Research...Read more
San Diego, California--(Newsfile Corp. - June 11, 2025) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) (OTCQX: TTIPF) ('Thiogenesis' or the 'Company') a clinical-stage biotechnology company developing disulfides that drive the production of intracellular cysteine, an amino acid that is a critical precursor to the generation of the master antioxidant glutathione and other important therapeutic compounds, today announced that the U.S. Food...Read more
Target Action (PDUFA) Date set for October 7, 2025 Application based on data from IMforte, the first Phase 3 trial demonstrating statistically significant and clinically meaningful improvements in both progression-free and overall survival in the ES-SCLC first-line maintenance setting Jazz to host investor webcast on Tuesday, June 10 at 4:30 p.m. EDT / 9:30 p.m. IST to review Zepzelca data For U.S. media and investors only DUBLIN,...Read more
MRT-8102, a highly selective NEK7-directed molecular glue degrader (MGD) developed to treat inflammatory conditions linked to NLRP3, IL-1β, and IL-6 dysregulation, expands Monte Rosa’s clinical I&I portfolio Potency, selectivity, and long-lasting pharmacodynamics of MRT-8102 observed in preclinical studies create potential for clinical differentiation from competitive approaches for inflammatory diseases MRT-8102 Phase 1 clinical...Read more
In laboratory studies, zoliflodacin has been shown to be active against Neisseria gonorrhoeae including multidrug-resistant strains. If approved, zoliflodacin could become the first new antibiotic treatment for gonorrhea in decades. WALTHAM, Mass. & GENEVA, Switzerland / Jun 10, 2025 / Business Wire / Innoviva Specialty Therapeutics, Inc., a subsidiary of Innoviva, Inc. (NASDAQ: INVA), in collaboration with the Global Antibiotic...Read more
The U.S. Food and Drug Administration (FDA) identified deficiencies at a third-party manufacturing vendor FDA to provide final decision by PDUFA action date of June 28, 2025 LOS ALTOS, Calif., June 10, 2025 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (Nasdaq: UNCY or the “Company”), a clinical-stage biotechnology company developing therapies for patients with kidney disease, today announced an update on its New Drug...Read more
NEW YORK, June 09, 2025 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) disorders, today announced it has received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) for its New Drug Application (NDA) for AXS-14 (esreboxetine) for the management of fibromyalgia. The FDA states that upon preliminary...Read more
This submission is supported by the pivotal Phase 3 VERITAC-2 clinical trial, results of which were recently presented at the 2025 American Society for Clinical Oncology Annual Meeting and published in The New England Journal of Medicine VERITAC-2 data support vepdegestrant as a potential treatment option in patients with ESR1m ER+/HER2- advanced or metastatic breast cancer NEW HAVEN, Conn., June 06, 2025 (GLOBE NEWSWIRE) --...Read more
Ketamine faces a current US drug shortage not expected to abate in the near future1 Current ketamine market estimated at $750 million and projected to reach $3.35 billion globally in 2034.2 NRx anticipates marketing ketamine for all approved uses Company anticipates priority review based on current and anticipated drug shortage NRX-100 to provide innovative, preservative-free IV ketamine formulation to eliminate...Read more
Once-weekly TransCon CNP demonstrated significantly higher annualized growth velocity, the primary endpoint, compared to placebo Multiple benefits beyond linear growth were observed compared to placebo, with a safety and tolerability profile similar to placebo The FDA grants Priority Review designation for the evaluation of medicines that, if approved, would provide a significant improvement in the safety or effectiveness of the...Read more
Both ERAS-4001 and potential best-in-class pan-RAS molecular glue ERAS-0015 received IND clearance in May ahead of company guidance Initial Phase 1 monotherapy data for both RAS-targeting programs expected in 2026 SAN DIEGO, June 02, 2025 (GLOBE NEWSWIRE) -- Erasca, Inc. (Nasdaq: ERAS), a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK...Read more
New Drug Application based on positive results from the Phase 2 KOMET-001 trial FDA assigns a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025 Potential first approval of a menin inhibitor for the treatment of adult patients with relapsed or refractory AML with an NPM1 mutation SAN DIEGO and TOKYO, June 01, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”) and Kyowa Kirin Co., Ltd....Read more
PRINCETON, N.J. / May 21, 2025 / Business Wire / UroGen Pharma Ltd. (Nasdaq: URGN), a leading biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, announced the outcome of today’s meeting of the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA), which discussed the new drug application (NDA) for investigational drug UGN-102...Read more
PDUFA target action date of January 13, 2026 If approved, FILSPARI would be the first and only FDA-approved treatment for FSGS, a rare kidney condition and a leading cause of kidney failure SAN DIEGO / May 15, 2025 / Business Wire / Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for traditional approval of FILSPARI...Read more
FDA grants Priority Review of application, with PDUFA target action date of September 12, 2025 ORLADEYO would be the first targeted oral prophylactic therapy for patients with HAE under the age of 12, if approved RESEARCH TRIANGLE PARK, N.C., May 14, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application...Read more
PRINCETON, N.J. / May 07, 2025 / Business Wire / UroGen (NASDAQ: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced that the U.S. Food and Drug Administration (FDA) has scheduled an Oncologic Drugs Advisory Committee (ODAC) meeting on May 21, 2025 to review the new drug application (NDA) for UGN-102 (mitomycin) for intravesical solution, an...Read more
Targeting to evaluate IDE849 (SHR-4849) in SCLC, NETs, and other DLL3-upregulated solid tumors, and in combination with IDE161/PARG to potentially enhance durability Targeting to present clinical data of over 40 SCLC patients, including the dose escalation and expansion, from partner Hengrui at medical conference in Q3 2025 SOUTH SAN FRANCISCO, Calif., May 6, 2025 /PRNewswire/ -- IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision...Read more
The FDA has cleared the investigational new drug (IND) application of LP-184 in triple negative breast cancer (TNBC) treatment – a novel small molecule advanced and developed with Lantern’s AI platform, RADR® The phase 1b/2 clinical trial plans to evaluate LP-184 in recurrent TNBC patients as both monotherapy and in combination with olaparib. This achievement follows the FDA’s Orphan Drug Designation for LP-184 in TNBC in 2023 and FDA...Read more
WASHINGTON, May 5, 2025 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) informed Vanda that the New Drug Application (NDA) for Bysanti™ (milsaperidone) has been filed, and that at this time no potential review issues have been identified. The FDA has set February 21, 2026 as the target date for decision on this application. Bysanti™ is a new chemical...Read more
QUEENSBURY, N.Y. / Apr 28, 2025 / Business Wire / Delcath Systems, Inc. (NASDAQ: DCTH), an interventional oncology company focused on the treatment of primary and metastatic liver cancers, today announced that the U.S. Food and Drug Administration (FDA) has completed its 30-day review of the Company's Investigational New Drug (IND) application for a Phase 2 clinical trial evaluating HEPZATO™ in combination with standard of care (SOC) for...Read more
NDA submitted for the treatment of central diabetes insipidus Expected 10-month review; commercial preparations underway for a potential Q1 2026 launch DEER PARK, Ill., April 28, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the submission of a New Drug...Read more
Expect to initiate a Phase 1/2a trial in the U.S. in advanced KRAS G12D mutant solid tumors in mid-2025 Initial safety and efficacy results from the trial of VS-7375 by partner GenFleet Therapeutics to be presented at the 2025 ASCO Annual Meeting BOSTON / Apr 23, 2025 / Business Wire / Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers,...Read more
SHANGHAI & CAMBRIDGE, Mass. / Apr 21, 2025 / Business Wire / Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced that China’s National Medical Products Administration (NMPA) has accepted the supplemental New Drug Application (sNDA) for repotrectinib for the treatment of adult patients with solid tumors that harbor a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. The application is intended for patients whose disease is...Read more
SAN DIEGO, April 17, 2025 (GLOBE NEWSWIRE) -- Calidi Biotherapeutics Inc. (NYSE American: CLDI) (“Calidi”), a clinical-stage biotechnology company pioneering targeted antitumor virotherapies, announced today that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for CLD-201. This investigational, allogeneic stem cell-based immunotherapy is set to advance into clinical development for...Read more
Collaboration partner, Gilead Sciences, to begin Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) study in healthy volunteers in Q2 2025 GS-6791/NX-0479 has potential clinical applications across multiple blockbuster markets in inflammation, in both rheumatology and dermatology, including rheumatoid arthritis and atopic dermatitis Nurix to receive a $5 million milestone payment from Gilead for FDA clearance of the...Read more
SAN DIEGO and TOKYO, April 08, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”), and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”), today 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...Read more
ALX2004 is a potential best- and first-in-class antibody-drug conjugate (ADC) for the treatment of EGFR-expressing solid tumors that was created from ALX Oncology’s proprietary linker-payload platform ALX2004, the company’s first ADC, was fully designed and developed in-house by ALX Oncology scientists Company expects to initiate Phase 1 clinical trials of ALX2004 in mid-2025, with initial safety data available in 1H 2026 SOUTH SAN...Read more
Significant Regulatory Milestone: U.S. FDA cleared IND application for CVHNLC, CureVac's investigational therapy targeting squamous non-small cell lung cancer Proprietary Epitopes: CVHNLC encodes novel tumor epitopes identified through proprietary whole genome-based discovery platform Enhanced Combination Therapy: CVHNLC to be tested in combination with pembrolizumab, aiming to amplify targeted anti-tumor immune...Read more
PDUFA target action date is July 28, 2025 WALTHAM, Mass., April 01, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today 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...Read more
Data demonstrated multiple clinical benefits beyond linear growth NDA supported by data from three randomized, double-blind, placebo-controlled clinical trials in children with achondroplasia, with up to three years of open-label extension data MAA in EU on track for submission during Q3 2025 COPENHAGEN, Denmark, March 31, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that it has submitted its New...Read more
WASHINGTON, March 31, 2025 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (NASDAQ: VNDA) today announced that a New Drug Application (NDA) was submitted to the U.S. Food and Drug Administration (FDA) requesting marketing approval of Bysanti™ (milsaperidone) for the treatments of acute bipolar I disorder and schizophrenia. The NDA is supported by several clinical studies assessing the efficacy and safety of Bysanti™. Bysanti™ is a new...Read more
MORRISVILLE, N.C., March 28, 2025 (GLOBE NEWSWIRE) -- Liquidia Corporation (NASDAQ: LQDA), a biopharmaceutical company developing innovative therapies for patients with rare cardiopulmonary disease, today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) resubmission for YUTREPIA™ (treprostinil) inhalation powder to treat pulmonary arterial hypertension (PAH) and pulmonary hypertension...Read more
U.S. IND Represents Regulatory Clearance For BEAM-302 Spanning Six Countries and Second Open IND for a Beam In Vivo Base Editing Program in the U.S. CAMBRIDGE, Mass., March 27, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has cleared the...Read more
BOSTON, March 24, 2025 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage company developing a new class of genetic medicines for cardiovascular disease, today announced the clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for VERVE-102 for the treatment of patients living with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD)....Read more
MELBOURNE, Australia and INDIANAPOLIS, March 21, 2025 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Nasdaq: TLX, Telix, the Company) today announces that the United States (U.S.) Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for Gozellix® (TLX007-CDx, kit for the preparation of gallium-68 (68Ga) gozetotide injection), Telix’s next-generation PSMA-PET imaging1 agent for prostate...Read more
TAMPA, FL, March 18, 2025 (GLOBE NEWSWIRE) -- – SCIENTURE HOLDINGS, INC. NASDAQ: SCNX), a holding company for existing and planned pharmaceutical operating companies focused on providing enhanced value to patients, physicians and caregivers through developing, bringing to market, and distributing novel specialty products to satisfy unmet market needs, is pleased to announce the U.S. Food and Drug Administration (FDA) has approved...Read more
First concentrated, ready-to-dilute liquid glucagon available for growing procedural gastroenterology market American Regent to commercialize GVOKE VialDx™ Availability is expected in the third quarter of 2025 CHICAGO / Mar 17, 2025 / Business Wire / Xeris Biopharma Holdings, Inc. (Nasdaq: XERS), a growth-oriented biopharmaceutical company committed to improving patient lives by developing and commercializing innovative products...Read more
PBGENE-HBV is the first-ever investigational in vivo gene editing therapy cleared to enter clinical trials for the treatment of chronic hepatitis B in the United States (U.S.) IND clearance represents a significant regulatory milestone for PBGENE-HBV Company to expand its Phase 1 ELIMINATE-B study to the U.S., joining world-class clinical sites in Moldova, Hong Kong, and New Zealand where strong clinical execution is...Read more
sNDA submission based on results from Phase 3 DUPLEX and Phase 2 DUET studies of FILSPARI in FSGS If approved, FILSPARI could become the first and only FDA-approved treatment for FSGS, a rare kidney condition and a leading cause of kidney failure Additionally, the FDA notified the Company that REMS monitoring for embryo-fetal toxicity is no longer necessary; the Company plans to submit an amendment to the REMS sNDA currently under...Read more
Phase 1/2 trial evaluating the proprietary combination to be run under CRADA with the National Cancer Institute Second Versamune® platform candidate targets MUC1-positive solid tumors U.S. Patent #12,201,685 covering methods of using combinations of the Versamune® platform and various immunocytokines, including PDS01ADC recently issued PRINCETON, N.J., March 13, 2025 (GLOBE NEWSWIRE) -- PDS Biotechnology Corporation (Nasdaq: PDSB)...Read more
TVB-3567 is the Company’s second fatty acid synthase (FASN) inhibitor First-in-human Phase 1 trial initiation planned in 2025 SAN MATEO, Calif., March 11, 2025 (GLOBE NEWSWIRE) -- Sagimet Biosciences Inc. (Sagimet, Nasdaq: SGMT), a clinical-stage biopharmaceutical company developing novel therapeutics targeting dysfunctional metabolic and fibrotic pathways, today announced the clearance of its Investigational New Drug (IND)...Read more
FDA approves scPharmaceuticals’ supplemental New Drug Application to expand the FUROSCIX indication to include the treatment of edema in adult patients with chronic kidney disease, including nephrotic syndrome FUROSCIX is expected to be available for chronic kidney disease patients in April 2025 BURLINGTON, Mass., March 06, 2025 (GLOBE NEWSWIRE) -- scPharmaceuticals Inc. (Nasdaq: SCPH) (the “Company”), a pharmaceutical company...Read more
Chimerix is on a mission to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. The company is devoted to filling gaps in the treatment paradigm. Chimerix’s most advanced clinical-stage program is in development for H3 K27M-mutant glioma....
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