Sangamo plans to initiate enrollment of patients in the Phase 1/2 study for ST-503 in mid-2025 RICHMOND, Calif. / Nov 19, 2024 / Business Wire / Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for its ST-503 program, an investigational epigenetic regulator for the treatment of intractable pain due to idiopathic small fiber... Read more
Phase 1b study for the potential treatment of osteoarthritis and low back pain expected to commence in the first quarter of 2025
Initial data readout planned for early 2026
Neuropathic component of chronic pain linked to LPA1 activation SAN DIEGO / Nov 18, 2024 / Business Wire / Contineum Therapeutics, Inc. (NASDAQ: CTNM) (Contineum or the Company), a clinical stage biopharmaceutical company focused on discovering and developing novel, oral small molecule therapies... Read more
The New Drug Application is based on positive results from the Phase 3 PALISADE study
People living with familial chylomicronemia syndrome have extremely high triglyceride levels and a substantially higher risk of developing acute pancreatitis and associated long-term complications, including poor quality of life
There are currently no FDA approved therapies to treat familial chylomicronemia syndrome PASADENA, Calif. / Nov 18, 2024 / Business Wire / Arrowhead Phar... Read more
PDUFA Date is April 2, 2025 LEXINGTON, Mass. / Nov 18, 2024 / 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 assigned a Prescription Drug User Fee Act (PDUFA) date... Read more
Northvale, New Jersey--(Newsfile Corp. - November 18, 2024) - Elite Pharmaceuticals, Inc. (OTCQB: ELTP) ("Elite" or the "Company"), a specialty pharmaceutical company engaged in the development, manufacture, and distribution of niche generic products, today announced that it received approval from the US Food and Drug Administration (FDA) for an Abbreviated New Drug Application (ANDA) for a generic version of Vyvanse® (Lisdexamfetamine Dimesylate) with strengths of 10... Read more
VAX-31 Infant Indication: Investigational New Drug Application Cleared by FDA; Company Expects to Initiate VAX-31 Infant Phase 2 Study by the End of January 2025
VAX-31 Adult Indication: Breakthrough Therapy Designation Granted by FDA; Company Plans to Initiate Adult Phase 3 Pivotal, Non-Inferiority Study by Mid-2025
VAX-31, Designed to Cover Currently Circulating and Historically Prevalent Strains, is Being Studied for the Prevention of Invasive Pneumococcal... Read more
FDA sets PDUFA Action Date of June 28, 2025
Company Preparing for 2025 Commercial Launch LOS ALTOS, Calif., Nov. 11, 2024 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company” or “Unicycive”), today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for Oxylanthanum Carbonate (OLC)... Read more
Donidalorsen will be a first-in-class RNA-targeted medicine for hereditary angioedema, assuming approval
Donidalorsen PDUFA date set for August 21, 2025
Donidalorsen has the potential to be Ionis' second independent commercial launch CARLSBAD, Calif., Nov. 4, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for donidalorsen,... Read more
Recurrent low-grade serous ovarian cancer is a rare cancer with no FDA-approved treatments
Company seeking accelerated approval and priority review of its NDA submission in patients with KRAS mutant low-grade serous ovarian cancer; FDA filing decision expected before the end of 2024 with potential for FDA approval decision by mid-2025 BOSTON / Oct 31, 2024 / Business Wire / Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines... Read more
WARREN, N.J., Oct. 30, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the New Drug Application (NDA) for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
"The NDA acceptance for review is a significant milestone that brings us one step closer to providing this important treatment to boys and young men living... Read more
Encinitas, California--(Newsfile Corp. - October 29, 2024) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) ("Kiora" or the "Company") today announced it received regulatory approval to initiate a Phase 2 clinical trial to investigate KIO-301 for vision restoration in patients with retinitis pigmentosa. The ABACUS-2 trial will be a 36 patient, multi-center, double-masked, randomized, controlled, multiple dose study enrolling patients with ultra-low vision or no light perception... Read more
TYRA-300 is the first oral FGFR-3 selective inhibitor to be well-tolerated in clinical studies
First child with achondroplasia expected to be dosed in Q1 2025 CARLSBAD, Calif., Oct. 28, 2024 /PRNewswire/ -- Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, announced today that the U.S.... Read more
IDE275 (GSK959) development is progressing into First-in-Human Phase 1 clinical trial(s) for the treatment of MSI-High solid tumors, representing IDEAYA's 5th potential first-in-class clinical program
MSI-High prevalence in endometrial, colorectal, and gastric cancers is ~31%, 20%, and 19%, respectively, highlighting the market potential of IDE275 (GSK959)
IDEAYA to receive a $7 million payment for IND acceptance, and potential future aggregate milestones of up to $950... Read more
Company to conduct a Phase 1 Drug-Drug Interaction Study
Phase 2 filing on track for Q1 2025 SOUTH SAN FRANCISCO, Calif., Oct. 22, 2024 (GLOBE NEWSWIRE) -- Aligos Therapeutics, Inc. (Nasdaq: ALGS, “Aligos”), a clinical stage biopharmaceutical company focused on improving patient outcomes through best-in-class therapies for liver and viral diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New... Read more
