BOSTON / Dec 23, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Dr. Reshma Kewalramani, Chief Executive Officer and President, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025 at 10:30 a.m. ET/7:30 a.m. PT. A live webcast of management's remarks will be available through the Vertex website, www.vrtx.com in the "Investors" section under the "News...Read more
SOUTH SAN FRANCISCO, Calif., Dec. 23, 2024 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for aficamten, a next-in-class cardiac myosin inhibitor, for the treatment of obstructive hypertrophic cardiomyopathy (HCM). The MAA will now be reviewed by the Committee for Medicinal Products for Human Use...Read more
ARO-INHBE targets a known pathway that signals the body to store fat in adipose tissue In preclinical studies ARO-INHBE reduced body weight and fat mass with a novel mechanism of action that may better preserve lean muscle mass compared to currently approved obesity therapies PASADENA, Calif. / Dec 23, 2024 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has dosed the first subjects in a Phase...Read more
WVE-007 is a novel approach for treating obesity and is designed to silence INHBE to achieve healthy, sustainable weight loss through fat burning, muscle maintenance and the potential for once or twice-annual administration WVE-007 is Wave’s first siRNA program to enter clinical development and uses best-in-class oligonucleotide chemistry and GalNAc delivery Wave expects to initiate the first-in-human study of WVE-007 in 1Q...Read more
IMC-P115C (PRAME-HLE-A02) targets the same peptide and has the same CD3 effector and TCR specificity as brenetafusp, and is designed with extended half-life The Phase 1 trial will assess the safety and clinical activity of IMC-P115C in HLA-A*02:01-positive patients with advanced solid tumors that express PRAME (OXFORDSHIRE, England & CONSHOHOCKEN, Penn. & GAITHERSBURG, Md., US, 23 December 2024) Immunocore...Read more
WOBURN, Mass., Dec. 23, 2024 (GLOBE NEWSWIRE) -- Replimune Group, Inc. (Nasdaq: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, today announced that members of the Replimune management team will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025 at 2:15 PM PT. A simultaneous webcast will be available in the Investors section of Replimune’s...Read more
Blarcamesine: Potential novel oral treatment to target upstream Alzheimer’s disease pathology through autophagy enhancement Submission based on favorable ANAVEX®2-73-AD-004 trial results in patients with early Alzheimer’s disease Once daily oral administration of blarcamesine meaningfully slowed clinical decline in early Alzheimer's disease patients, demonstrating a favorable safety profile with no associated neuroimaging adverse...Read more
Company to host a webcast today at 8:30 a.m. Eastern Time NEW YORK, Dec. 23, 2024 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer's disease, Parkinson's disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system...Read more
New Drug Application (NDA) is based on pooled data from the pivotal Phase 2 TRUST-I and TRUST-II studies of taletrectinib that demonstrated durable responses and prolonged progression-free survival in patients with advanced ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC) If approved, taletrectinib represents a potential best-in-class treatment option for patients with advanced ROS1+ NSCLC U.S. Food and Drug Administration (FDA)...Read more
PARIS, France, December 23, 2024, 10:05 PM CEST – Abivax SA (Euronext Paris & Nasdaq: ABVX) (“Abivax” or the “Company”), a clinical-stage biotechnology company focused on developing therapeutics that harness the body’s natural regulatory mechanisms to modulate the inflammatory response in patients with chronic inflammatory diseases, today announced that Dr. Philippe Pouletty, representative of Truffle Capital, tendered his resignation...Read more
NEW YORK, Dec. 23, 2024 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel radioimmunotherapy and antibody-based therapeutic products for the treatment of cancer, today announced that Michael Rossi, President and Chief Executive Officer, will present at the 43rd Annual J.P. Morgan Healthcare...Read more
TibuSURE is the first clinical trial designed to investigate anti-BAFF and IL-17 pathways in systemic sclerosis. TibuSURE launches with the initiation of its first clinical site. HENDERSON, Nev. / Dec 23, 2024 / Business Wire / Zura Bio Limited (Nasdaq: ZURA) (“Zura Bio”), a clinical stage, multi-asset immunology company developing novel dual-pathway antibodies for autoimmune and inflammatory diseases, today announced the launch of...Read more
MIAMI, FL / ACCESSWIRE / December 23, 2024 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), or the "Company", an emerging leader in age-reversal science, today announced significant findings from its copper binding studies with Telomir-1. This is a promising development as the company explores Telomir-1 for the treatment of Wilson's disease and other copper related disorders. The announcement marks an important step forward in Telomir's...Read more
LOS ANGELES / Dec 23, 2024 / Business Wire / Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, announced that the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Cervical Cancer were updated to include an addition involving neratinib (NERLYNX®). The updated NCCN Practice Guidelines for Cervical Cancer include neratinib monotherapy for use as second-line or...Read more
Inmagene Biopharmaceuticals is a clinical stage company focused on developing IMG-007, a non-depleting anti-OX40 monoclonal antibody with an extended half-life and a silenced ADCC function The transaction is expected to result in approximately $175 million to support further development of IMG-007, including $75 million from an oversubscribed Private Placement that will close immediately following the merger The Financing includes...Read more
Tonix received FDA’s Day 74 Letter granting TNX-102 SL a Prescription Drug User Fee Act (PDUFA) goal date of August 15, 2025 TNX-102 SL is a non-opioid, centrally acting analgesic, granted Fast Track designation by FDA Fibromyalgia affects more than 10 million adults in the U.S., who are mostly women TNX-102 SL has the potential to be the first member of a new class of analgesic drugs for fibromyalgia and first new drug for its...Read more
HOUSTON, Dec. 23, 2024 (GLOBE NEWSWIRE) -- FibroBiologics, Inc. (Nasdaq: FBLG) ("FibroBiologics”), a clinical-stage biotechnology company with 160+ patents issued and pending with a focus on the development of therapeutics and potential cures for chronic diseases using fibroblasts and fibroblast-derived materials, announced that it has entered into a Standby Equity Purchase Agreement (the “SEPA”) with YA II PN LTD. (“Yorkville”), an...Read more
SOUTH SAN FRANCISCO, Calif., Dec. 23, 2024 (GLOBE NEWSWIRE) -- RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based therapeutics company focused on discovering, developing and commercializing novel therapies for patients with significant unmet needs in inflammatory diseases, today announced that it has entered into a securities purchase agreement with a group of accredited investors for the private placement of (i)...Read more
RAPT obtains worldwide rights excluding China Jemincare receives $35 million upfront payment, up to $672.5 million in milestone payments and high single-digit to low-double digit royalties on future sales Jemincare is currently conducting Phase 2 trials in asthma and chronic spontaneous urticaria in China RAPT plans to initiate Phase 2b trial in food allergy next year RAPT to host a conference call at 8:30...Read more
The Company has received approval to begin enrollment of the Phase 1b clinical trial in Australia Phase 1b clinical trial will assess safety and tolerability along with biomarkers during three months of dosing with GT-02287 in people diagnosed with Parkinson’s disease Phase 1b clinical trial follows successful Phase 1 study in which GT-02287 was safe and well tolerated while demonstrating GCase target engagement BETHESDA, Md.,...Read more
Promising results support VYN202’s potential as a novel, once-daily oral treatment for a broad range of immune-mediated disorders Consistent with Phase 1a SAD results, VYN202 demonstrated a favorable safety and tolerability profile with no drug-related adverse events historically associated with earlier generation, less selective BET inhibitors VYN202 demonstrated robust pharmacodynamic activity including evidence of target engagement...Read more
AUSTIN, Texas / Dec 23, 2024 / Business Wire / Anebulo Pharmaceuticals, Inc. (Nasdaq: ANEB), a clinical-stage biopharmaceutical company developing novel solutions for people suffering from acute cannabinoid-induced toxicities (the “Company” or “Anebulo”), today announced a positive regulatory update and the close of a capital raise. In a Phase 2 proof-of-concept study, Anebulo enrolled 134 adult subjects challenged with oral...Read more
Binding Term Sheet with a leading global pharmaceutical company includes a substantial upfront payment and funding for the Phase 3 study Additionally, milestone payments and double-digit royalties on annual net sales The transaction is subject to finalization of a definitive agreement, anticipated in Q1 2025 CAMBRIDGE, Mass., Dec. 23, 2024 /PRNewswire/ -- NeuroSense Therapeutics Ltd. (Nasdaq: NRSN) ("NeuroSense"), a...Read more
First quarter revenues increased by 360% year over year First quarter net loss for the quarter of $0.002 per share Grant of 1,535,715 stock options to independent directors QUEBEC CITY / Dec 23, 2024 / Business Wire / Devonian Health Group Inc. (“Devonian” or the “Company”) (TSXV: GSD; OTCQB: DVHGF), today announced financial results for its first quarter ended on October 31, 2024. Management Comments “Devonian continued to improve...Read more
630 Patients Enrolled To Date; Enrollment Completion Now Expected in Q1 2025 with Top-line Results Anticipated in Q2 2025; Upon Potential Positive Phase 3 Data the Company Expects to Submit a New Drug Application (“NDA”) for D-PLEX100 under Fast Track and Breakthrough Therapy Designations, Previously Granted to D-PLEX100 by the FDA; Proceeds from this Financing and Exercise of Data-Triggered Warrant Expected to Extend Cash...Read more
KANSAS CITY, Kan., Dec. 23, 2024 (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, has completed a financing transaction with an accredited investor which provided net proceeds to CING of $5 million. The transaction was structured as a...Read more
WASHINGTON, Dec. 23, 2024 (GLOBE NEWSWIRE) -- 60 Degrees Pharmaceuticals, Inc. (NASDAQ: SXTP; SXTPW) (“60 Degrees” or the “Company”), pharmaceutical company, and Tufts Medical Center (“Tufts MC”), a leading academic medical center, today announced the signing of a Patent License Agreement to jointly advance the development and commercialization of tafenoquine for the treatment and prevention of babesiosis. Tafenoquine is not currently...Read more
Marlborough, Massachusetts--(Newsfile Corp. - December 23, 2024) - Phio Pharmaceuticals Corp. (NASDAQ: PHIO), a clinical-stage biotechnology company that develops therapeutics using its INTASYL® siRNA gene silencing technology to make the body's immune cells more effective in killing cancer cells, today announced that it has entered into definitive agreements for the purchase and sale of an aggregate of 240,000 shares of its common stock...Read more
Investigational agent in development for treatment or prevention of H5N1 Bird Flu Phase 1 dosing completed in healthy volunteers Potent inhibition of drug-resistant and bird flu viruses in vitro In vivo study in mice, with H5N1 isolated from an infected dairy worker, showed potent protection and suppression of virus replication in lungs Phase 2 study expected to begin in H1 2025 Traws Pharma is expanding its...Read more
Company committed to building a robust global patent estate across its innovative pipeline of NKT cell modulators LA JOLLA, CA, Dec. 23, 2024 (GLOBE NEWSWIRE) -- GRI Bio, Inc. (NASDAQ: GRI) (“GRI Bio” or the “Company”), a biotechnology company advancing an innovative pipeline of Natural Killer T (“NKT”) cell modulators for the treatment of inflammatory, fibrotic and autoimmune diseases, today announced that European Patent Office...Read more
ALYFTREK™ is approved for patients 6 years and older with at least one responsive mutation, including 31 additional mutations not responsive to other CFTR modulator therapies In head-to-head clinical trials, ALYFTREK was non-inferior on ppFEV1 and further decreased sweat chloride compared to TRIKAFTA® BOSTON / Dec 20, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug...Read more
Approximately 300 more people with cystic fibrosis in the U.S. are now eligible for a medicine that treats the underlying cause of their disease for the first time BOSTON / Dec 20, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of people with...Read more
CRENESSITY, a First-in-Class Treatment for Classic Congenital Adrenal Hyperplasia (CAH), is now commercially available in the U.S. CAH-trained pharmacists are available 24/7 to support patients with prescriptions exclusively through PANTHERx Rare, a specialty pharmacy Neurocrine Access Support is available to provide free, comprehensive access and support information to patients, caregivers and healthcare providers SAN DIEGO, Dec....Read more
SOUTH SAN FRANCISCO, Calif., Dec. 20, 2024 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that Sanofi will acquire exclusive rights to develop and commercialize aficamten from Corxel Pharmaceuticals (CORXEL) for the treatment of patients with obstructive and non-obstructive hypertrophic cardiomyopathy (HCM) in Greater China. Aficamten is a next-in-class cardiac myosin inhibitor for the potential...Read more
BOSTON, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for IMCIVREE® (setmelanotide) to include children as young as 2 years old. IMCIVREE is indicated to reduce excess...Read more
SAN FRANCISCO and SUZHOU, China, Dec. 20, 2024 /PRNewswire/ -- Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of oncology, cardiovascular and metabolic, autoimmune, ophthalmology and other major diseases, today announced that China's National Medical Products Administration (NMPA) has approved a New Drug...Read more
Acquisition bolsters Eton’s commercial pediatric endocrinology portfolio Product is now available through AnovoRx, a specialty pharmacy dedicated to serving patients with rare and chronic conditions DEER PARK, Ill., Dec. 20, 2024 (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...Read more
THE WOODLANDS, Texas, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced it has received a complete response letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) for Zynquista™ (sotagliflozin) as an adjunct to insulin therapy for glycemic control in adults with type 1 diabetes and chronic kidney disease (CKD). This expected communication...Read more
WASHINGTON, Dec. 20, 2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor for the treatment of polycythemia vera (PV), a form of a rare hematologic malignancy that is estimated to affect 1 in 2000 Americans.1 PV is a chronic myeloproliferative...Read more
Renovaro will Establish the GEDi CUBE Next Generation Nvidia Blackwell Cluster with Nebul's certified AI Healthcare Cloud solutions LOS ANGELES, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Renovaro Inc. (NASDAQ: RENB), a pioneer in cancer diagnostics and therapeutics powered by artificial intelligence, today announced its subsidiary Renovaro Cube (the “Cube”), a leader in specific AI-driven healthcare solutions, has entered into a strategic...Read more
PEDMARQSI® (anhydrous sodium thiosulfate) is the first and only treatment available within NHS England and Wales for the prevention of cisplatin-induced ototoxicity (hearing loss) in children and young people (1 month-17 years of age) Data from two open-label, randomized Phase 3 trials, SIOPEL 6 (pivotal) and the Clinical Oncology Group (COG) Protocol ACCL0431, demonstrated an approximate 50% reduction in the occurrence of...Read more
JENA, Germany, Dec. 20, 2024 (GLOBE NEWSWIRE) -- InflaRx N.V. (Nasdaq: IFRX), a biopharmaceutical company pioneering anti-inflammatory therapeutics targeting the complement system, today announced that the first patient has been dosed in its Phase 2a basket study in chronic spontaneous urticaria (CSU) and hidradenitis suppurativa (HS), investigating the Company’s oral C5aR inhibitor, INF904. Camilla Chong, MD, Chief...Read more
TORONTO / Dec 20, 2024 / Business Wire / Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, today announced that it has closed an equity offering, issuing a total of 63,285,000 equity securities for gross proceeds of approximately $57 million (USD$40 million) (the...Read more
Shoshana Shendelman Steps Down as CEO; Les Funtleyder Appointed Interim Chief Executive Officer Announces Business Updates NEW YORK, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a biopharmaceutical company dedicated to creating transformative treatments for rare disease, today announced the following leadership changes, effective immediately: John H. Johnson, a recognized leader in the...Read more
Dispensed applicator units in the fourth quarter of 2024 have exceeded the complete prior quarter even with significant cost reductions in sales and operational infrastructure Observed significant reduction in YCANTH distributor inventory levels New single applicator configuration for YCANTH® expected to be available in the first quarter of 2025 to help meet growing product demand, reduce acquisition costs for physician practices and...Read more
Funds expected to be used to advance IO Biotech’s immune-modulating therapeutic cancer vaccines, including potential BLA submission for IO102-IO103 in 2025 Commitment is part of the European Investment Bank’s strategy to support biotech companies with cutting-edge expertise in therapeutic areas such as immuno-oncology Debt facility expected to extend company’s cash runway into the second quarter of 2026 COPENHAGEN, Denmark and NEW...Read more
In the pre-specified per-protocol population, belapectin showed a statistically significant reduction (p-value < 0.05) in development of esophageal varices in 2mg/kg cohort compared to placebo While there was a favorable trend for incidence of varices in the primary end point intent-to-treat population, belapectin did not achieve statistical significance Belapectin was overall well tolerated with no safety signals; incidence of...Read more
SAN DIEGO, Dec. 20, 2024 (GLOBE NEWSWIRE) -- BioAtla, Inc. (Nasdaq: BCAB), a global clinical-stage biotechnology company (“BioAtla” or the “Company”) focused on the development of Conditionally Active Biologic (CAB) antibody therapeutics for the treatment of solid tumors, today announced that it has entered into definitive agreements with certain institutional investors for the issuance and sale of 9,679,158 shares of its common stock in...Read more
Vancouver, British Columbia--(Newsfile Corp. - December 20, 2024) - Defence Therapeutics Inc. (CSE: DTC) (OTCQB: DTCFF) (FSE: DTC) ("Defence" or the "Company"), a Canadian biopharmaceutical company developing radiopharmaceuticals and ADC products using its proprietary platform and drug delivery technologies in addition to novel immune-oncology vaccines, is pleased to announce that Dr. Elias Theodorou has joined the board of...Read more
FOSTER CITY, Calif., Dec. 20, 2024 (GLOBE NEWSWIRE) -- Apollomics Inc. (Nasdaq: APLM) (“Apollomics” or the “Company”), a late-stage clinical biopharmaceutical company developing multiple oncology drug candidates to address difficult-to-treat and treatment-resistant cancers, announced today the results from its Phase 3 bridging trial of uproleselan in China in patients with relapsed or refractory acute myeloid leukemia. The trial did not...Read more
EDINBURGH, Scotland, Dec. 20, 2024 /PRNewswire/ -- TC BioPharm (Holdings) PLC ("TC BioPharm" or the "Company") (NASDAQ: TCBP) a clinical-stage biotechnology company developing platform allogeneic gamma-delta T cell therapies for cancer and other indications, today announced 3 patients have now completed the full-dose regimen in the ACHIEVE Phase 2b trial in the UK with no drug-related Adverse Events seen in any of the...Read more
FREEHOLD, N.J., Dec. 20, 2024 (GLOBE NEWSWIRE) -- Channel Therapeutics Corporation, (“Channel” or the “Company”), (NYSE American: CHRO), a pioneer in the development of non-opioid pain treatment therapeutics, is providing a statement regarding Vertex Pharmaceutical’s recently announced Phase 2 data of Suzetrigine, an investigational, oral, highly selective NaV1.8 pain signal inhibitor in people with painful lumbosacral radiculopathy. “We...Read more
NEW YORK / Dec 20, 2024 / Business Wire / Aptorum Group Limited (NASDAQ: APM) (“Aptorum Group” or the “Company”), a clinical stage biopharmaceutical company dedicated to meeting unmet medical needs in oncology, autoimmune and infectious diseases, today provided a business update and announced financial results for the six months ended June 30, 2024. “Our team and Yoov have spent considerable time and effort on the due diligence process,...Read more
Treatment with the highly selective NaV1.8 pain signal inhibitor suzetrigine met the primary endpoint with a statistically significant and clinically meaningful 2.02 point within-group reduction from baseline in the Numeric Pain Rating Scale (NPRS) Placebo arm showed similar within-group reduction in NPRS Suzetrigine was generally well tolerated Advancement to Phase 3 in painful lumbosacral radiculopathy planned,...Read more
Investigational REGN7508 (catalytic domain) and REGN9933 (A2 domain) are being evaluated for their potential to control thrombosis while minimizing bleeding risk in a variety of patient populations and clinical settings Evaluated against current standards of care, single doses of REGN7508 and REGN9933 administered 12 to 24 hours after total knee replacement demonstrated robust antithrombotic effects Phase 3 program to be initiated in...Read more
COPENHAGEN, Denmark, Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that YORVIPATH® (palopegteriparatide; developed as TransCon PTH) is now commercially available by prescription in the United States. YORVIPATH is a prodrug of parathyroid hormone (PTH [1-34]), administered once-daily, designed to provide continuous exposure to active PTH over the 24-hour dosing period. It is the first and only...Read more
SOMERSET, N.J., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, today announced that Ying Huang, Ph.D., the company’s Chief Executive Officer, will present the latest company updates at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco, CA, on Tuesday, January 14, 2025, at 3:45 p.m. PT. Investors and other interested parties can access the...Read more
TRYNGOLZA shown to significantly reduce triglycerides and substantially reduce acute pancreatitis events in adults with FCS; a rare, highly debilitating and life-threatening disease Indicated for adults with FCS regardless of genetically or clinically confirmed diagnosis TRYNGOLZA is the first of four independent launches planned over the next three years, pending approvals Ionis to host webcast today at 6:45pm ET...Read more
CAMBRIDGE, Mass., Dec. 19, 2024 /PRNewswire/ -- Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced that James Porter, Ph.D., Chief Executive Officer, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2025, at 9:00 a.m. PT in San Francisco. A live...Read more
NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the first patient has been dosed in the pivotal Phase 3 Aspire study (NCT06617429) evaluating the efficacy and safety of GTX-102, its investigational antisense oligonucleotide (ASO) for Angelman syndrome. "Initiation of patient dosing in our Phase 3 Aspire study represents an important step forward in the development of...Read more
NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). “The path to get a treatment to the point of a BLA filing has been long...Read more
SAN DIEGO / Dec 19, 2024 / Business Wire / Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that it will present at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco, CA. Catherine Owen Adams, Chief Executive Officer will represent the Company in a session scheduled on Tuesday, January 14, 2025, at 9:00 a.m. Pacific Time / 12:00 p.m. Eastern Time. A live webcast of Acadia’s presentation will be accessible on the...Read more
HAMPTON, N.J., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Celldex Therapeutics, Inc. (NASDAQ:CLDX) today announced that the Company has initiated a Phase 2 study of barzolvolimab in atopic dermatitis (AD) and that the study is actively enrolling patients. AD is one of the most common chronic inflammatory skin diseases, with a lifetime prevalence of up to 20% of the US population and a substantial impact on quality of life1. Mast cells are strongly...Read more
SAN FRANCISCO, Dec. 19, 2024 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with cancer and inflammatory diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for NX-5948, a highly selective degrader of Bruton’s tyrosine kinase (BTK), for the treatment of...Read more
Inclusion of neffy on Express Scripts commercial formularies was effective November 22, 2024 and expands access to patients and caregivers managing Type 1 Allergic Reactions SAN DIEGO, Dec. 19, 2024 (GLOBE NEWSWIRE) -- ARS Pharmaceuticals, Inc. (Nasdaq: SPRY), a biopharmaceutical company dedicated to empowering at-risk patients and caregivers to better protect themselves from allergic reactions that could lead to anaphylaxis,...Read more
SYMVESS is a first-in-class bioengineered human tissue designed to be a universally implantable vascular conduit for use in arterial replacement and repair In clinical testing SYMVESS was observed to have high rates of patency, or blood flow, and low rates of amputation and infection Highly experienced sales team already recruited and trained in preparation for commercial launch DURHAM, N.C., Dec. 19, 2024 (GLOBE NEWSWIRE) --...Read more
Primary statistical analysis of overall survival compared TA-TMA patients treated with narsoplimab in its pivotal trial to a cohort of over 100 TA-TMA patients not treated with narsoplimab in an external control stem cell transplant registry; FDA-agreed analysis was conducted by an independent external statistical group Narsoplimab-treated TA-TMA patients had an over 3-fold reduction in risk of mortality (hazard ratio = 0.32 [95%...Read more
Business update to include masked Phase 3 patient demographics and baseline characteristics Presentation on Wednesday, January 15, 2025, 5:15 PM PT in San Francisco, California MELBOURNE, Australia and PRINCETON, N.J., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Opthea Limited (ASX/NASDAQ:OPT, “Opthea”, the “Company”), a clinical-stage biopharmaceutical company developing novel therapies to treat highly prevalent and progressive retinal...Read more
Agreement grants Astellas rights to employ Sangamo’s novel proprietary capsid, STAC-BBB, for up to five potential neurological disease targets Sangamo to receive a $20 million upfront license fee and is eligible to earn up to $1.3 billion in additional licensed target fees and milestone payments across all five potential disease targets, as well as tiered royalties on potential net sales RICHMOND, Calif. & TOKYO / Dec 19, 2024 /...Read more
- Agreement grants Astellas rights to employ Sangamo's novel proprietary capsid, STAC-BBB, for up to five potential neurological disease targets - - Sangamo to receive a $20 million upfront license fee and is eligible to earn up to $1.3 billion in additional licensed target fees and milestone payments across all five potential disease targets, as well as tiered royalties on potential net sales - TOKYO and RICHMOND, Calif.,...Read more
FREMONT, Calif. / Dec 19, 2024 / Business Wire / Personalis, Inc. (NASDAQ: PSNL) announced today agreements with long time collaborators Merck and Moderna that accelerate the advancement of the company’s business strategy. The company has received an investment from Merck, known as MSD outside the United States and Canada, and separately has signed a multi-year extension with Moderna to utilize Personalis’ ImmunoID NeXT Platform® and...Read more
WARREN, N.J., Dec. 19, 2024 (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 the U.S. Food and Drug Administration (FDA) has granted seven years of orphan drug exclusivity (ODE) to Libervant® (diazepam) Buccal Film for the acute...Read more
OCU410 has a very favorable safety and tolerability profile No serious adverse events related to the study drug have been reported, such as exudation, infectious endophthalmitis, intraocular Inflammation, anterior ischemic optic neuropathy, or vasculitis MALVERN, Pa., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing...Read more
Helen Tayton-Martin, Ph.D., M.B.A, Adaptimmune Co-Founder and Chief Business & Strategy Officer, will step down from the Company March 31, 2025 Gavin Wood, Chief Financial Officer, will step down from the Company May 31, 2025 Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - December 19, 2024) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company working to redefine the treatment of solid tumor cancers...Read more
Assembly Bio to receive approximately $20.1 million equity investment and $10 million in accelerated funding under the collaboration, extending cash runway to mid-2026 Additional equity investment raises Gilead’s ownership stake in Assembly Bio to 29.9% Funding supports pipeline progress, including amendment to accelerate clinical development of Assembly Bio’s novel small molecule hepatitis D virus candidate ABI-6250 SOUTH SAN...Read more
SAN DIEGO, Dec. 19, 2024 (GLOBE NEWSWIRE) -- Skye Bioscience, Inc. (Nasdaq: SKYE) (“Skye”), a clinical-stage biopharmaceutical company focused on unlocking new therapeutic pathways for obesity and other metabolic health disorders, will present a corporate overview at the 43rd Annual J.P. Morgan Healthcare conference on Thursday, January 16, 2025, at 10:30 AM - 11:10 AM. A live and archived webcast will be accessible on Skye’s...Read more
Enrollment underway for patients with metastatic/advanced clear cell renal cell carcinoma (ccRCC) Company expects to share preliminary Phase 1 clinical data in the first half of 2025 REDWOOD CITY, Calif. & BOSTON / Dec 19, 2024 / Business Wire / Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today...Read more
Results demonstrate that inhaled AP-PA02 provides a durable reduction of Pseudomonas aeruginosa in the lung, with a favorable safety and tolerability profile LOS ANGELES, Dec. 19, 2024 /PRNewswire/ -- Armata Pharmaceuticals, Inc. (NYSE American: ARMP) ("Armata" or the "Company"), a biotechnology company focused on the development of high-purity pathogen-specific bacteriophage therapeutics for chronic pulmonary diseases and...Read more
Aadi Enters into Exclusive License for Three-Asset ADC Portfolio Developed through a Collaboration between WuXi Biologics and HANGZHOU DAC Aadi Enters Agreement to Sell FYARRO® and Associated Infrastructure to KAKEN Pharmaceutical for $100M; Announces PIPE Financing of $100M Cumulative Capital Expected to Fund Operations into Late 2028, Including Anticipated Clinical Data for the ADC Portfolio Co-Founder and Former...Read more
ALPHARETTA, Ga., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), announced today that the official journal of The Royal College of Ophthalmologists, Eye, published a summary of critical insights into drug development and regulatory processes based on a presentation at the...Read more
All four patients treated with NXC-201 normalized their disease markers within 30 days of dosing, of which, two are already classified as complete responders (CR), and the remaining two are bone marrow MRD negative (10-6); all patients remain in response as of the data cutoff of Nov 14, 2024 Bone marrow MRD negativity predicts future CR; company believes remaining two patients could be confirmed as CRs in the coming weeks and...Read more
Achieved total revenue of RMB 371.9 million, reflecting a growth of 36.2% YoY Achieved gross profit of RMB 307.3 million, a growth of 39.1% YoY Gross margin increased to 82.6% from 80.9% in the same period of FY2024 Total operating expenses decreased to RMB 276.4 million, down 31.6% YoY Recorded net income of RMB 20.6 million, and adjusted net income[1] (Non-GAAP) of RMB 30.4 million, compared to net loss and adjusted...Read more
MISSISSAUGA, Canada, Dec. 19, 2024 (GLOBE NEWSWIRE) -- Microbix Biosystems Inc. (TSX: MBX, OTCQX: MBXBF, Microbix®), a life sciences innovator, manufacturer, and exporter, reports results for its fiscal year and fourth quarter ended September 30, 2024 (“2024” and “Q4”) with record revenues, reflective of ongoing progress to increase sales from its diagnostic-test related ingredients and devices businesses and that resulted in material net...Read more
Announces preliminary Phase 1 data for XTX301, a tumor-activated IL-12, demonstrating an improved tolerability profile over historical data for rhIL-12, with no dose-limiting toxicities Completed additional private placement with Gilead for purchase of remaining equity investment in connection with XTX301 partnership WALTHAM, Mass., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Xilio Therapeutics, Inc. (Nasdaq: XLO), a clinical-stage...Read more
Registered Phase-1b 12-week study will investigate safety, diabetes control, and weight loss KELOWNA, BC / ACCESSWIRE / December 19, 2024 / Lexaria Bioscience Corp. (Nasdaq:LEXX) (Nasdaq:LEXXW) (the "Company" or "Lexaria"), a global innovator in drug delivery platforms, announces it has begun dosing in the Company's Phase 1b, 12-week chronic study GLP-1-H24-4, (the "Study"). The Study is designed mainly to determine whether Lexaria's...Read more
HOUSTON, Dec. 19, 2024 (GLOBE NEWSWIRE) -- Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage immuno-oncology company focusing on developing next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, today announced it has entered into a securities purchase agreement for a private placement resulting in gross proceeds of $16.1 million, before deducting placement agent...Read more
APOLLO study investigating MT-601 in patients with relapsed lymphoma: 78% of patients achieved objective response rates, with 44.4% demonstrating complete response (CR) MT-601 was observed to be well tolerated in all study participants with no observation of immune-effector cell associated neurotoxicity syndrome (ICANS) HOUSTON, Dec. 19, 2024 (GLOBE NEWSWIRE) -- Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage...Read more
Open label study designed to evaluate the safety, tolerability, and efficacy of bremelanotide in patients with Type 2 diabetic nephropathy Demonstrated efficacy at 6 months 71% percent of patients achieved a >30% reduction in the urine protein to creatinine ratio (UP/Cr) 71% of patients achieved improved or stabilized estimated glomerular filtration rate (eGFR) Bremelanotide therapy increased urinary VEGF levels...Read more
VICTORIA, British Columbia / Dec 19, 2024 / Business Wire / IPA (IMMUNOPRECISE ANTIBODIES LTD.) (the “Company” or “IPA”) (NASDAQ: IPA), an AI-driven biotherapeutic research and technology company, today announced is pleased to announce its participation in The Microcap Conference 2025, the premier event for growth-focused companies and investors. The conference will take place January 28-30, 2025, in Atlantic City, NJ. IPA’s management...Read more
Magdalena to explore coca leaf's possible therapeutic benefit for treatment of potentially ADHD or other neuropsychiatric indications The import was conducted in collaboration with the government of Peru Steven King, Ph.D. of Jaguar to speak about the potential promise of coca-based Botanical Drugs at the February 2025 Wisdom of the Leaf Coca Summit in Peru Magdalena is focused on developing novel, natural prescription medicines...Read more
| Company | Change | Last Trade |
|---|---|---|
| ZIVO Bioscience | 21.00 1,909.09 | $22.10 |
| Regeneron Pharmaceuticals | 13.28 1.89 | $715.13 |
| Finch Therapeutics | 9.00 500.00 | $10.80 |
| Vertex Pharmaceuticals | 8.00 2.01 | $405.27 |
| argenx | 4.16 0.67 | $627.98 |
| United Therapeutics | 4.07 1.13 | $363.65 |
| Ascendis Pharma | 3.32 2.43 | $139.70 |
| Verona Pharma | 3.26 7.76 | $45.28 |
| Mesoblast | 2.86 19.55 | $17.49 |
| Anavex Life Sciences | 2.55 29.55 | $11.18 |
| CAMP4 Therapeutics | 2.38 45.77 | $7.58 |
| Vaxcyte | 2.36 2.73 | $88.89 |
| Madrigal Pharmaceuticals | 2.12 0.69 | $310.57 |
| SpringWorks Therapeutics | 1.78 5.00 | $37.37 |
| Sarepta Therapeutics | 1.69 1.42 | $120.66 |
Immix Biopharma is a clinical-stage biopharmaceutical company pioneering a novel class of CAR-T cell therapies and Tissue-Specific Therapeutics targeting oncology and immuno-dysregulated diseases with >75 patients treated to-date. The company's lead cell therapy asset is NXC-201 in multiple myeloma...
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Astria Therapeutics is a biopharmaceutical company, and our mission is to bring life-changing therapies to patients and families affected by rare and niche allergic and immunological diseases. Our lead program, STAR-0215, is a monoclonal antibody inhibitor of plasma kallikrein in clinical development...
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