Orphan Drug Designation Granted to KB408 PITTSBURGH, Sept. 21, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development, and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced today that the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug Application (IND) for...Read more
SOUTH SAN FRANCISCO, Calif., Sept. 21, 2023 (GLOBE NEWSWIRE) -- Aligos Therapeutics, Inc. (Nasdaq: ALGS, “Aligos”), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in liver and viral diseases, today announced it has received clearance of its Investigational New Drug (IND) application from the US Food and Drug Administration (FDA) for ALG-055009, it’s thyroid hormone...Read more
OSAKA, Japan & CAMBRIDGE, Mass. / Sep 20, 2023 / Business Wire / Takeda (TSE:4502/NYSE:TAK) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review its New Drug Application (NDA) resubmission for TAK-721 (budesonide oral suspension) which is being investigated for the short-term treatment of eosinophilic esophagitis (EoE), a chronic inflammatory disease that can cause damage to the esophagus.1 The...Read more
Acceptance based on results from the Phase 3 LITESPARK-005 trial, which showed a statistically significant and clinically meaningful improvement in progression-free survival compared to everolimus in patients with advanced RCC that progressed following PD-1/L1 and VEGF-TKI therapies If approved, WELIREG would provide a new, novel mechanism of action for patients with advanced RCC in need of new options RAHWAY, N.J. / Sep 19, 2023 /...Read more
Pulmatrix received a study may proceed letter for a Phase 2 Study to Evaluate PUR3100, an orally inhaled dry powder formulation of dihydroergotamine ("DHE") in the treatment of acute migraine BEDFORD, Mass., Sept. 19, 2023 /PRNewswire/ -- Pulmatrix, Inc. ("Pulmatrix" or the "Company") (NASDAQ: PULM), a clinical-stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary and central nervous...Read more
Prompted by advice from FDA regarding study design, 60P Australia Pty Ltd, a majority-owned subsidiary of 60 Degrees Pharmaceuticals, has withdrawn its IND for ACLR8-LR, a Phase IIB study of the use of tafenoquine in treating COVID-19; Company plans to resubmit a revised IND later in 2023, pending a feasibility assessment 60P has paused further study start-up preparations related to ACLR8-LR, decreasing the Company’s short-term burn...Read more
This is the second IND Clearance over the last 100 days for drug candidates that are being developed using guidance from Lantern’s AI Platform, RADR®. LP-284 is the second of Lantern’s synthetic-lethal franchise of molecules that have shown to be synthetically lethal in DNA damage repair deficient cancers and are entering into first-in-human clinical trials. Lantern estimates that LP-284 can have the potential to improve outcomes for...Read more
FDA approves supplemental new drug application (sNDA) for Talicia, changing dosing to a simplified three times daily (TID) regimen, which may support increased patient adherence and treatment outcomes Talicia is the leading branded first-line therapy prescribed by U.S. gastroenterologists[1] for eradication of H. pylori - a bacterial infection that affects approximately 35% of the U.S. adult population[2] RALEIGH, N.C. and TEL-AVIV,...Read more
YONKERS, N.Y., Sept. 18, 2023 (GLOBE NEWSWIRE) -- ContraFect Corporation (Nasdaq: CFRX), a clinical-stage biotechnology company focused on the discovery and development of direct lytic agents (DLAs), including lysins and amurin peptides, today announces that it submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) on September 15, 2023. The IND application supports a proposed indication for...Read more
Prescription Drug User Fee Act (PDUFA) Target Action Date Set for April 30, 2024 SAN DIEGO, Sept. 14, 2023 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for INGREZZA® (valbenazine) oral granules, a new sprinkle formulation of INGREZZA® (valbenazine) capsules for oral administration. The agency set a Prescription...Read more
Cardiovascular and Renal Drugs Advisory Committee Voted 9:3 That the Benefits of Patisiran Outweigh its Risks for the Treatment of the Cardiomyopathy of ATTR Amyloidosis Prescription Drug User Fee Act Target Action Date is October 8, 2023 CAMBRIDGE, Mass. / Sep 13, 2023 / Business Wire / Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the positive outcome of the U.S....Read more
CONSHOHOCKEN, Pa., Sept. 13, 2023 (GLOBE NEWSWIRE) -- Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), today announced that the U.S. Food and Drug Administration (FDA) has accepted for review its New Drug Application (NDA) for resmetirom for the treatment of adult patients with NASH with liver fibrosis. The FDA has granted Priority...Read more
BAUDETTE, Minn., Sept. 12, 2023 (GLOBE NEWSWIRE) -- ANI Pharmaceuticals, Inc. (ANI or the Company) (Nasdaq: ANIP) today announced that it received U.S. Food and Drug Administration (FDA) approval for the Abbreviated New Drug Application (ANDA) for Estradiol Tablets USP. ANI’s Estradiol Tablets USP are the generic version of the Reference Listed Drug (RLD) Estrace®. The According to IQVIA, a leading healthcare data and analytics provider,...Read more
Atopic dermatitis is the most common type of eczema, affecting approximately 9.6 million children and 16.5 million adults in the United States In pivotal Phase 3 trials, roflumilast cream demonstrated statistically significant improvements over vehicle on the primary endpoint of Investigator Global Assessment (IGA) Success at Week 4, as well as on key secondary endpoints including rapid improvement in itch Roflumilast cream was well...Read more
PDUFA Target Action Date of June 26, 2024 Ensifentrine, if approved, is expected to be the first novel mechanism available for the maintenance treatment of COPD in more than a decade LONDON and RALEIGH, N.C., Sept. 11, 2023 (GLOBE NEWSWIRE) -- Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), announces the US Food and Drug Administration (“FDA”) has accepted for review the Company’s New Drug Application (“NDA”)...Read more
Overall response rate (ORR) greater than 50% across three assessment criteria Median duration of tovorafenib treatment of 15.8 months as of June 5, 2023, with 66% of patients remaining on treatment FDA filing decision expected by mid-November BRISBANE, Calif., Sept. 11, 2023 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals (Nasdaq: DAWN) (“Day One” or the “Company”), a clinical-stage biopharmaceutical company dedicated to...Read more
HT-KIT received Orphan Drug Designation from FDA for its mRNA Frame Shifting Cancer Therapeutic Hoth intends to initially target mast cell neoplasms for development of HT-KIT, which is a rare, aggressive cancer with poor prognosis NEW YORK, Sept. 11, 2023 /PRNewswire/ -- Hoth Therapeutics, Inc. (NASDAQ: HOTH), a patient-focused biopharmaceutical company, today announced it has submitted a request for a...Read more
FDA Acceptance of Libervant™ (diazepam) Buccal Film NDA for treatment of seizure clusters in patients between two and five years of age Prescription Drug User Fee Act (PDUFA) target goal date set for April 28, 2024 WARREN, N.J., Sept. 11, 2023 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ: AQST) (the “Company” or “Aquestive”), a pharmaceutical company advancing medicines to solve patients’ problems with current standards...Read more
Announced VG-3927 as lead candidate to enter clinical development for potential treatment of Alzheimer’s Disease IND for VG-3927 now open; Phase 1 clinical trial in healthy volunteers allowed to proceed with partial clinical hold related to maximum exposure limit Phase 1 dosing expected to commence in October 2023; No anticipated delays in current clinical development plans Company to host virtual R&D event highlighting its small...Read more
Investigational New Drug (IND) application submitted to U.S. Food and Drug Administration (FDA) for ELX-02 for the treatment of Alport syndrome with nonsense mutations Rebound in average UPCR 3-months post treatment provides further evidence that proteinuria remission in one out of three patients was drug related All 3 Alport patients (100% response rate) treated with ELX-02 had biopsy confirmed disease regression, suggesting clinical...Read more
Key injectable currently on the U.S. FDA shortage list Received Competitive Generic Therapy approval designation BRIDGEWATER, N.J. / Sep 06, 2023 / Business Wire / Amneal Pharmaceuticals, Inc. (NYSE: AMRX) (“Amneal” or the “Company”) today announced it has received Abbreviated New Drug Application (“ANDA”) approval from the U.S. Food and Drug Administration (“FDA”) for calcium gluconate in sodium chloride injection, 1000 mg/50...Read more
BOSTON, Sept. 06, 2023 (GLOBE NEWSWIRE) -- Tango Therapeutics, Inc. (NASDAQ: TNGX), a clinical-stage biotechnology company committed to discovering and delivering the next generation of precision cancer medicines, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for TNG348, a novel inhibitor of USP1 (ubiquitin-specific protease 1), for the treatment of...Read more
NEW YORK, Sept. 06, 2023 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced successful completion of a recent pre-New Drug Application (“NDA”) meeting with the Food and Drug Administration (FDA) regarding its govorestat (AT-007) Galactosemia...Read more
TORONTO, Sept. 06, 2023 (GLOBE NEWSWIRE) -- PharmaTher Holdings Ltd. (the “Company” or “PharmaTher”) (OTCQB: PHRRF) (CSE: PHRM), a commercial-focused specialty pharmaceutical company, is pleased to announce it has submitted a priority original Abbreviated New Drug Application (“ANDA”) for KETARX™ (racemic ketamine) to the Food and Drug Administration (“FDA”) for expedited review of its ANDA. The Company anticipates KETARX™ approval and...Read more
LEXINGTON, Mass. and AMSTERDAM, Sept. 05, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AMT-260, the Company’s gene therapy candidate for refractory mesial temporal lobe epilepsy (MTLE). AMT-260...Read more
BOSTON, Sept. 05, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced the submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for the approval of once-daily, oral mavorixafor to treat individuals aged 12 and older with WHIM (Warts, Hypogammaglobulinemia, Infections, and...Read more
IND builds on previously-published, preliminary evidence of efficacy Filing is supported by robust manufacturing, pre-clinical and clinical data already reviewed by FDA for NRX-101 associated with treatment of bipolar depression RADNOR, Pa., Aug. 30, 2023 /PRNewswire/ - NRx Pharmaceuticals, Inc. (Nasdaq: NRXP) ("NRx Pharmaceuticals", the "Company"), a clinical-stage biopharmaceutical company, today announced submission an...Read more
Safety aspects of AL001 development may qualify for (505)(b)(2) pathway for FDA approval Alzamend’s recently completed a Phase IIA Study of AL001 in Alzheimer’s and healthy patients that identified a candidate dose that is unlikely to require therapeutic drug monitoring ATLANTA / Aug 30, 2023 / Business Wire / Alzamend Neuro, Inc. (Nasdaq: ALZN) (“Alzamend”), a clinical-stage biopharmaceutical company focused on developing novel...Read more
WALTHAM, Mass., Aug. 29, 2023 (GLOBE NEWSWIRE) -- TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies (TCR-T) for the treatment of patients with cancer, today announced that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for TSC-203-A0201, a TCR-T targeting PReferentially...Read more
BAUDETTE, Minn., Aug. 28, 2023 (GLOBE NEWSWIRE) -- ANI Pharmaceuticals, Inc. (ANI or the Company) (Nasdaq: ANIP) today announced that it received U.S. Food and Drug Administration (FDA) approval for the Abbreviated New Drug Application (ANDA) for Estradiol Gel, 0.1%. ANI’s Estradiol Gel, 0.1%, is the generic version of the Reference Listed Drug (RLD) Divigel®. The current annual U.S. market for Estradiol Gel, 0.1%, is approximately $42.3...Read more
This Approval Supports Veklury’s Strong Safety Profile and Makes Veklury the Only Approved COVID-19 Antiviral Treatment Across all Stages of Liver Disease FOSTER CITY, Calif. / Aug 24, 2023 / Business Wire / Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) approved a supplemental new drug application (sNDA) for the use of Veklury® (remdesivir) with no dose adjustments to treat...Read more
If approved, XTANDI would become the first and only novel hormone therapy approved in this earlier type of prostate cancer NEW YORK and TOKYO, Aug. 23, 2023 /PRNewswire/ -- Pfizer Inc. (NYSE: PFE) and Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for the companies' supplemental New Drug...Read more
Received FDA clearance of IND for gedatolisib in combination with darolutamide Virtual Science Day to be held on September 21, 2023 from 10:00 a.m. – 12:00 p.m. ET MINNEAPOLIS, Aug. 22, 2023 (GLOBE NEWSWIRE) -- Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, has been notified by the U.S. Food and Drug Administration (FDA) that its Investigational New Drug...Read more
Phase 1 trial expected to begin in early Q4 2023 VIP943 is Vincerx’ first candidate from next-generation ADC platform and its second IND clearance within 8 months, highlighting Vincerx’s development and regulatory expertise PALO ALTO, Calif., Aug. 22, 2023 (GLOBE NEWSWIRE) -- Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through...Read more
FOSTER CITY, Calif. / Aug 22, 2023 / Business Wire / Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has assigned a standard review and a Prescription Drug User Fee Act (PDUFA) action date of June 16, 2024 for Geron’s New Drug Application (NDA) for imetelstat for the treatment of transfusion-dependent anemia in patients with lower...Read more
MELBOURNE, Australia, Aug. 22, 2023 (GLOBE NEWSWIRE) -- Incannex Healthcare Limited (Nasdaq: IXHL) (ASX: IHL), (‘Incannex’ or the ‘Company’) a clinical-stage pharmaceutical company developing proprietary medicinal cannabinoid products and psychedelic assisted psychotherapies for unmet needs, is pleased to announce that it has received approval from the US Food and Drug Administration (‘FDA’) to conduct the Company’s Investigational New...Read more
THOUSAND OAKS, Calif., Aug. 21, 2023 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced the U.S. Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) will review data supporting the supplemental New Drug Application (sNDA) for the full approval of LUMAKRAS® (sotorasib) for adults with previously treated locally advanced or metastatic KRAS G12C-mutated non-small cell lung cancer (NSCLC) at a meeting on Oct. 5,...Read more
GSK101 (IDE705) is being developed as a potential first-in-class Pol Theta Helicase Inhibitor in combination with niraparib in GSK-sponsored Phase 1/2 clinical trial GSK101 and niraparib combination development will focus on advanced solid tumors with HR mutations or HRD, pursuant to the clinical protocol IDEAYA to receive a $7 million milestone payment upon IND acceptance, and potential future aggregate milestones of up to $950...Read more
FOSTER CITY, Calif. / Aug 21, 2023 / Business Wire / Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has accepted the filing of Geron’s New Drug Application (NDA) for imetelstat, its first-in-class telomerase inhibitor, for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes...Read more
SHANGHAI, Aug. 20, 2023 /PRNewswire/ -- Everest Medicines (HKEX 1952.HK, "Everest", or the "Company") announced today that U.S. Food and Drug Administration (FDA) has accepted the submission for the supplemental New Drug Application (sNDA) for Nefecon® from its partner Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) ("Calliditas") and granted Priority Review. The Prescription Drug User Fee Act (PDUFA) goal date is Dec....Read more
STOCKHOLM, Aug. 18, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT), (Nasdaq Stockholm: CALTX) ("Calliditas"), today announced that the U.S. Food and Drug Administration (FDA) has accepted the submission for the supplemental New Drug Application (sNDA) for TARPEYO® (budesonide) delayed release capsules and granted Priority Review. The Prescription Drug User Fee Act (PDUFA) goal date is 20 December 2023. TARPEYO is...Read more
An IND was required to conduct the SB221 study of platinum-resistant ovarian cancer (PROC) with the combination of Sonnet's SON-1010 and Roche's atezolizumab (Tecentriq®) in the US As part of the overall design of the program, the clinical trial was initiated at several sites in Australia after HREC regulatory approval and TGA notification The IND acceptance by FDA allows for a global clinical effort, with the possibility for more...Read more
Foralumab to advance into Phase 2 human clinical trials using the world’s only fully human intranasal anti-CD3 monoclonal antibody Trial to be overseen by Brigham and Women’s Hospital, a founding member of Mass General Brigham Healthcare System NEW YORK, Aug. 15, 2023 (GLOBE NEWSWIRE) -- Tiziana Life Sciences Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation...Read more
Next-generation and first oral TNF-α inhibitor, which completed Phase 2 study for age-related condition sarcopenia, represents a potentially groundbreaking advance in the treatment of RA and disruption of a $41 billion industry Acceptance follows statistically significant Phase 2 data in sarcopenia showing MYMD-1 reduced TNF-α and other inflammatory markers common to RA, while demonstrating safety and tolerability MyMD now advancing 3...Read more
The Company remains committed to its goal of bringing avasopasem to patients and intends to meet with FDA as soon as possible to discuss potential next steps Galera will take actions to extend its cash runway and continue enrolling its rucosopasem clinical trials Conference call tomorrow, August 10, 2023 at 8:30 a.m. ET MALVERN, Pa., Aug. 09, 2023 (GLOBE NEWSWIRE) -- Galera Therapeutics, Inc. (Nasdaq: GRTX), a clinical-stage...Read more
LOS ANGELES / Aug 08, 2023 / Business Wire / Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, has been notified by the U.S. Food and Drug Administration (FDA) that its Investigational New Drug (IND) submission has been reviewed, and Puma can proceed with the clinical development of alisertib monotherapy for the treatment of patients with extensive stage small cell lung cancer (SCLC). Puma’s Phase II trial (Study...Read more
MVID is a life-threatening and ultra-rare autosomal recessive disease that affects newborns and children, leading to intestinal failure, significant morbidity and even death from severe secretory diarrhea SAN FRANCISCO, CA / ACCESSWIRE / August 8, 2023 / Napo Pharmaceuticals Inc. (Napo), a Jaguar Health, Inc. (NASDAQ:JAGX) company, today announced the activation by the U.S. Food and Drug Administration (FDA) of the Investigational...Read more
FDA assigns PDUFA target action date of March 8, 2024 New Drug Application supported by positive Phase III efficacy and safety clinical trial results Milestone builds upon Viatris' long-standing and continued commitment toward addressing the unmet needs of the MS community PITTSBURGH and NESS ZIONA, Israel, Aug. 7, 2023 /PRNewswire/ -- Viatris Inc. (NASDAQ: VTRS), a global healthcare company, and Mapi Pharma Ltd., a fully...Read more
Investigational New Drug (IND) clearance transitions Qualigen Therapeutics from preclinical to clinical-stage company and demonstrates leadership in G4-targeting therapies for areas of high unmet need in oncology CARLSBAD, Calif., Aug. 01, 2023 (GLOBE NEWSWIRE) -- Qualigen Therapeutics, Inc. (Nasdaq: QLGN), a clinical-stage therapeutics company focused on developing treatments for adult and pediatric cancers with potential for Orphan...Read more
Napo Pharmaceuticals, a Jaguar Health company and the manufacturer of crofelemer, is supporting this physician-generated IND SAN FRANCISCO, CA / ACCESSWIRE / August 1, 2023 / Jaguar Health (NASDAQ:JAGX) ("the Company") today announced the activation by the U.S. Food and Drug Administration (FDA) of a physician-requested Investigational New Drug (IND) application to evaluate crofelemer for treatment of uncontrolled diarrhea in a...Read more
No new clinical data is required to add PH-ILD indication as per FDA correspondence Recertified that YUTREPIA does not infringe any valid patents listed in the Orange Book for Tyvaso Preparing for potential launch of YUTREPIA between end of 2023 to mid-2024 MORRISVILLE, N.C., July 27, 2023 (GLOBE NEWSWIRE) -- Liquidia Corporation (NASDAQ: LQDA) announced today the submission of an amendment to the tentatively approved new drug...Read more
PITTSBURGH, July 26, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced today that it has expanded its R&D pipeline to oncology and that the US Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND)...Read more
Represents three high-value medicines across complex dosage forms BRIDGEWATER, N.J. / Jul 26, 2023 / Business Wire / Amneal Pharmaceuticals, Inc. (NYSE: AMRX) (“Amneal” or the “Company”) today announced it has submitted Abbreviated New Drug Applications (“ANDA”) to the U.S. Food and Drug Administration (FDA) for three key complex generic products, including: Product Dosage Category U.S. IQVIA...Read more
MELBOURNE, Australia, July 21, 2023 (GLOBE NEWSWIRE) -- Incannex Healthcare Limited (Nasdaq: IXHL) (ASX: IHL), (‘Incannex’ or the ‘Company’) a clinical-stage pharmaceutical company developing unique medicinal cannabinoid pharmaceutical products and psychedelic medicine therapies for unmet medical needs, is pleased to announce that it has successfully submitted an Investigational New Drug (‘IND’) application to the US Food and Drug...Read more
Study will evaluate drug's potential neuro-restorative mechanism(s) of action for improving synaptic health and cognitive function in MS patients NEW YORK, July 19, 2023 /PRNewswire/ -- Synaptogenix, Inc. (Nasdaq: SNPX) ("Synaptogenix" or the "Company"), an emerging biopharmaceutical company developing regenerative therapeutics for neurodegenerative disorders, today announced that it entered into an agreement with Cleveland...Read more
CONSHOHOCKEN, Pa., July 17, 2023 (GLOBE NEWSWIRE) -- Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), announced the completion of the rolling submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for resmetirom for the treatment of adults with NASH with liver fibrosis, a disease with no...Read more
Submission seeks approval for BRUKINSA in combination with obinutuzumab as a treatment for relapsed or refractory follicular lymphoma BASEL, Switzerland & BEIJING & CAMBRIDGE, Mass. / Jul 12, 2023 / Business Wire / BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company, today announced the U.S. Food and Drug Administration (FDA) has accepted for review the Company’s supplemental new drug application...Read more
The trial will assess the safety and preliminary efficacy of Salarius’ novel targeted protein degrader SP-3164; Patient dosing expected to begin in 2H 2023 The trial will also assess the utility of a gene signature to identify patients that are potentially sensitized to SP-3164 treatment and may be more likely to respond HOUSTON, July 11, 2023 (GLOBE NEWSWIRE) -- Salarius Pharmaceuticals, Inc. (NASDAQ: SLRX), a clinical-stage...Read more
LEXINGTON, Mass., July 11, 2023 /PRNewswire/ -- Pulmatrix, Inc. ("Pulmatrix" or the "Company") (NASDAQ: PULM), a clinical-stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary and central nervous system disease, today announced the submission of an investigational new drug ("IND") application to the United States Food and Drug Administration ("FDA") for PUR3100, an orally inhaled dry powder...Read more
WATERTOWN, Mass., July 05, 2023 (GLOBE NEWSWIRE) -- C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation (TPD) science to develop a new generation of small-molecule medicines and transform how disease is treated, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s investigational new drug (IND) application for...Read more
Believed to be the first FDA IND clearance of an allogeneic dual CAR-T therapy targeting CD19 and CD20 SAN DIEGO, July 5, 2023 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND)...Read more
Letter requests additional pharmacokinetic data No clinical efficacy or manufacturing issues identified BRIDGEWATER, N.J. / Jul 03, 2023 / Business Wire / Amneal Pharmaceuticals, Inc. (NYSE: AMRX) (“Amneal” or the “Company”) today announced it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the Company’s New Drug Application (NDA) for IPX203 for the treatment of Parkinson’s...Read more
CAMBRIDGE, Mass. / Jun 30, 2023 / Business Wire / Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has set a date of September 13, 2023 for the meeting of the Cardiovascular and Renal Drugs Advisory Committee to review the supplemental New Drug Application for patisiran, an investigational treatment for the cardiomyopathy of...Read more
Madrigal expects to complete full submission of the New Drug Application in July 2023 CONSHOHOCKEN, Pa., June 30, 2023 (GLOBE NEWSWIRE) -- Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), initiated a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval...Read more
LOS ALTOS, Calif., June 29, 2023 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company” or “Unicycive”), today provided an update based on recent interactions with the U.S. Food and Drug Administration (FDA or Agency) concerning the Company’s New Drug Application (NDA) for lanthanum dioxycarbonate (LDC), previously...Read more
Submitted NDA for Libervant™ (diazepam) Buccal Film for treatment of seizure clusters in patients between two and five years of age Appointed Carl N. Kraus, M.D. as Chief Medical Officer WARREN, N.J., June 29, 2023 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ: AQST) (the “Company” or “Aquestive”), a pharmaceutical company advancing medicines to solve patients’ problems with current standards of care and provide...Read more
MVID is a life-threatening and ultra-rare autosomal recessive disease that affects newborns and children, leading to intestinal failure, significant morbidity and even death from severe secretory diarrhea SAN FRANCISCO, CA / ACCESSWIRE / June 28, 2023 / Napo Pharmaceuticals Inc., a Jaguar Health, Inc. (NASDAQ:JAGX) company, today announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug...Read more
DEER PARK, Ill., June 28, 2023 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, announced today that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) in response to its New Drug Application (NDA) for dehydrated alcohol injection for the treatment of methanol poisoning. The issues...Read more
LONDON and RALEIGH, N.C., June 27, 2023 (GLOBE NEWSWIRE) -- Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”) announces the submission of a New Drug Application (“NDA”) to the US Food and Drug Administration (“FDA”) for approval of ensifentrine for the maintenance treatment of patients with chronic obstructive pulmonary disease (“COPD”). Ensifentrine is a selective dual inhibitor of the enzymes phosphodiesterase 3 and 4...Read more
TORONTO, June 27, 2023 (GLOBE NEWSWIRE) -- PharmaTher Holdings Ltd. (the “Company” or “PharmaTher”) (OTCQB: PHRRF) (CSE: PHRM), a commercial-focused specialty pharmaceutical company, is pleased to announce it has filed a Pre-Submission Facility Correspondence in advance of its Abbreviated New Drug Application (“ANDA”) for KETARX™ (racemic ketamine) to the Food and Drug Administration (“FDA”) to support expedited review of its priority...Read more
LOS ANGELES, June 26, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc., a subsidiary of Immix Biopharma, Inc. (“Nexcella”, “Company”, “We” or “Us”), a biopharmaceutical company pioneering cell therapies targeting oncology and other diseases, today announced it has completed a Pre-Investigational New Drug (“Pre-IND”) meeting with the FDA. The subject of the Pre-IND meeting was planned NXC-201 US manufacturing and US clinical trials in AL...Read more
HAYWARD, Calif., June 26, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for BB-301, its silence and replace...Read more
STOCKHOLM, June 21, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas") today announced the submission of a supplemental New Drug Application ("sNDA") to the U.S. Food and Drug Administration ("FDA") seeking full approval of TARPEYO® (budesonide) delayed release capsules for the entire study population from the Phase 3 NeflgArd study. TARPEYO is currently approved under...Read more
BERKELEY HEIGHTS, N.J., June 21, 2023 (GLOBE NEWSWIRE) -- CorMedix Inc. (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of life-threatening diseases and conditions, today announced that the resubmission of the New Drug Application (NDA) for DefenCath has been accepted for filing by the U.S. Food and Drug Administration (FDA). The Agency considers...Read more
Based on U.S. Food & Drug Administration (FDA) Determination of Lack of Adequate and Well Controlled Investigations in the Scientific Literature, Complete Response Letter Received for New Drug Application (NDA) of ADX-2191 (methotrexate injection, USP) for the Treatment of Primary Vitreoretinal Lymphoma (PVRL) Due to Shortage of Methotrexate, Lack of Approved Therapy for PVRL, and Inbound Requests for ADX-2191, Expanded Access...Read more
FOSTER CITY, Calif. / Jun 20, 2023 / Business Wire / Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced the submission to the United States Food and Drug Administration (FDA) of a New Drug Application (NDA) for imetelstat for the treatment of transfusion-dependent anemia in adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have failed to respond or have lost...Read more
HAMILTON, BERMUDA / ACCESSWIRE / June 15, 2023 / Altamira Therapeutics Ltd. ("Altamira" or the "Company") (Nasdaq:CYTO), a company dedicated to developing therapeutics that address important unmet medical needs, today announced that the U.S. Food and Drug Administration ("FDA") has completed its safety review of the Company's Investigational New Drug (IND) application for AM-125 (betahistine nasal spray) in acute vestibular syndrome...Read more
Adds first ready-to-use presentation of key injectable for treating non-squamous non-small cell lung cancer and malignant pleural mesothelioma Represents a high value, complex product launch for Amneal’s expanding injectable portfolio BRIDGEWATER, N.J. / Jun 14, 2023 / Business Wire / Amneal Pharmaceuticals, Inc. (NYSE: AMRX) (“Amneal” or the “Company”) today announced the 505(b)(2) New Drug Application (NDA) approval from the U.S....Read more
Namodenoson’s robust effect reached 90% inhibition of pancreatic cancer growth in pre-clinical studies PETACH TIKVA, Israel / Jun 13, 2023 / Business Wire / Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncology, inflammatory, and liver diseases, today announced that it is doing all the preparatory work for an FDA Investigational...Read more
LP-184 is a novel, synthetically-lethal, small molecule that has been developed using insights from Lantern’s AI platform, RADR®. The first-in-human Phase 1A clinical trial has been cleared to proceed by the FDA and is anticipated to launch this summer for multiple advanced solid tumors and brain and central nervous system (CNS) cancers. Lantern has been granted multiple Orphan Drug Designations by the FDA for LP-184 in pancreatic...Read more
Prescription Drug User Fee Act (PDUFA) goal date of November 17, 2023 Combined launch of vonoprazan for Erosive GERD and H. pylori indications planned for Q4 2023, if approved FLORHAM PARK, N.J., June 12, 2023 (GLOBE NEWSWIRE) -- Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal diseases, today announced that the U.S. Food and...Read more
WALTHAM, Mass., June 06, 2023 (GLOBE NEWSWIRE) -- TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies (TCR-Ts) for the treatment of patients with cancer, today announced that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for TSC-200-A0201, a TCR-T targeting human...Read more
STAMFORD, Conn., June 05, 2023 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today that the U.S. Food and Drug Administration (FDA) has updated the Prescription Drug User Fee Act (PDUFA) action date for the New Drug Application (NDA) for nirogacestat for the treatment of...Read more
Submitted four efficacy supplements seeking inclusion of CV risk reduction and expansion of LDL-C lowering Anticipated approvals in U.S. in the first half of 2024 ANN ARBOR, Mich., June 01, 2023 (GLOBE NEWSWIRE) -- Esperion (NASDAQ: ESPR) today announced that it has submitted Supplemental New Drug Applications (sNDA) to the U.S. Food and Drug Administration (FDA) for the Company’s oral non-statin products NEXLETOL and...Read more
HPV is responsible for more than 90 percent of cervical cancer cases globally with approximately 300,000 women living with cervical cancer in the US Current response rates to second line treatments in recurrent or metastatic cervical cancer are very low and associated with a high rate of toxicity PRGN-2009 is designed to generate de novo T-cell immune response against HPV 16/18-positive solid tumors cells and is...Read more
Submitted a new drug application (NDA) for troriluzole in Spinocerebellar Ataxia Type 3 (SCA3) to U.S. FDA in 2Q2023, marking the team's fourth NDA in approximately 3 years Released additional data from Kv7 platform, including Phase 1 safety data by dose groups for BHV-7000 that further validates differentiated profile Projected Phase 3 Spinal Muscular Atrophy trial to complete enrollment in 2023 Initiated Phase 1 study of brain...Read more
MOUNTAIN VIEW, Calif., May 31, 2023 (GLOBE NEWSWIRE) -- IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing IgM antibodies, today announced that the U.S. Food and Drug Administration (FDA) has cleared two Investigational New Drug (IND) applications for imvotamab, an IgM-based CD20 X CD3 bispecific antibody T cell engager, enabling the initiation of Phase 1b studies in both severe...Read more
Application based on results from the registrational TRIDENT-1 trial, in which repotrectinib demonstrated high response rates and durable responses in patients with ROS1-positive locally advanced or metastatic non-small cell lung cancer If approved, repotrectinib will provide a potential best-in-class option for patients with ROS1-positive NSCLC who are TKI-naïve and a potential first-in-class treatment for patients who have been treated...Read more
Prescription Drug User Fee Act (PDUFA) Target Action Date Set for November 30, 2023 NDA Includes Results From the Phase 3 FRESCO-2 and FRESCO Clinical Trials OSAKA, Japan & CAMBRIDGE, Mass. & HONG KONG & SHANGHAI & FLORHAM PARK, N.J. / May 25, 2023 / Business Wire / Takeda (TSE:4502/NYSE:TAK) and HUTCHMED (China) Limited (Nasdaq/AIM:HCM, HKEX:13) (HUTCHMED) today announced that the U.S. Food and Drug Administration...Read more
KELOWNA, BC / ACCESSWIRE / May 24, 2023 / Tryp Therapeutics, Inc. (CSE:TRYP)(OTCQB:TRYPF) ("Tryp" or the "Company"), a clinical-stage biotechnology company focused on developing intravenous-infused psilocin (the active metabolite of psilocybin) for diseases with high unmet medical needs, today announced it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for its planned Phase 2a...Read more
Surmodics is the global leader in surface modification technologies for intravascular medical devices and a leading provider of chemical components for in vitro diagnostic immunoassay tests and microarrays. Surmodics is pursuing highly differentiated whole-product solutions that are designed to address...
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