July 8, 2026 -- Grünenthal announced today that its investigational compound tegacorat (GRM-01) received Orphan Drug and Rare Pediatric Disease Designations from the US Food and Drug Administration (FDA) for the treatment of Duchenne muscular dystrophy (DMD). Tegacorat, a non-steroidal Selective Glucocorticoid Receptor Agonist and Modulator (SEGRAM), is an orally available investigational compound being developed to provide an alternative to glucocorticoid-based treatments such as prednisone, t

PDUFA target action date set for April 28, 2027 Varegacestat could provide a meaningful new oral treatment option for adults with desmoid tumors if approved Immunome, Inc. (Nasdaq: IMNM), a biotechnology company committed to developing first-in-class and best-in-class targeted cancer therapies, today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted its New Drug Application (NDA) for varegacestat, an investigational, oral, once-daily gamma secretase inhibitor (GSI

– Comprehensive updates to be presented at Barth Syndrome Foundation conference – July 8, 2026 -- Mighty Therapeutics (the "Company" or "Mighty"), a commercial-stage biotechnology company pioneering a new class of medicines that directly target mitochondrial dysfunction in rare and age-related diseases, today announced that the first patient has been dosed in 4TAZPower, its global Phase 4 study to confirm the efficacy, safety, and pharmacokinetics of once-daily elamipretide in individuals at le

Enpatoran could address a significant unmet need in lupus, specifically targeting cutaneous manifestations of the disease, which currently have no approved therapies FDA's decision was supported by Phase 2 WILLOW study results, which demonstrated meaningful symptoms improvement, particularly among patients with active cutaneous manifestations Enpatoran's potential to go beyond current treatment standards in lupus prompted FDA's breakthrough designation, which is designed to expedite drug develop

FDA clearance enables initiation of a planned Phase 1 clinical study evaluating local intravesical administration of UGN-501, with patient enrollment expected to begin in Q4 2026 UGN-501 is a differentiated investigational next-generation oncolytic virus designed to combine direct tumor cell destruction with anti-tumor immune activation July 08, 2026 -- UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial an

MeiraGTx has secured an investment of up to $400 million from Oberland Capital to help get its late-stage gene therapies across the commercialization finish line. Earlier this year, MeiraGTx bought back botaretigene sparoparvovec (bota-vec) from Johnson & Johnson, despite the therapy failing to improve vision-guided mobility in patients with X-linked retinitis pigmentosa (XLRP) in a phase 3 trial . Despite the trial miss, the company touted “clinically meaningful benefit” seen in the data and s

Existing data supports direct path to resubmission following constructive FDA engagement July 7, 2026 -- Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, today announced the resubmission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for SL1009, sodium dichloroacetate (DCA), for the treatment of pyruvate dehydrogenase complex deficiency (PDCD), a rare, life-threatening mitochondrial disease with no FDA-approved therapies. The resubmi

Vertex Pharmaceuticals is making the largest acquisition bet in its history, announcing late Monday an agreement to purchase a biotechnology company, Crinetics Pharmaceuticals, that recently brought to market a new kind of medicine for a rare hormonal disorder. Vertex is paying $85 per share in cash, or $10 billion overall, for Crinetics. The deal ranks as the fourth largest, by total proceeds, in an already busy year for biotech company acquisitions, according to BioPharma Dive data. Additiona

July 7, 2026 -- Kenai Therapeutics, a clinical-stage biotechnology company pioneering next generation approaches to cure neurological conditions, today announced the completion of enrollment for the REPLACE™ Phase 1b/2a clinical trial of RNDP-001, an investigational cell therapy being developed for the treatment of moderate to moderate-severe idiopathic Parkinson's disease (iPD). RNDP-001 is an off the shelf, allogeneic neuron replacement cell therapy designed to restore dopaminergic neurons lo

TTI-109 delivered TTI-101-equivalent exposure with improved tolerability and meaningful reductions in disease-relevant STAT3-driven immune cell populations including Th17, T follicular helper (Tfh) and B cells Company plans to advance TTI-109 into STAT3-driven dermatologic and gastrointestinal (GI) diseases - subject to additional funding Company to host investor webcast today, July 7th, at 8:30am ET July 07, 2026 -- Tvardi Therapeutics, Inc. (“Tvardi” or the “Company”) (NASDAQ: TVRD), a clinic