Jaguar Health, Inc. (NASDAQ: JAGX) What's Different????? 3 NASDAQ:JAGX • The classic definition of insanity: Doing the same thing over and over again and expecting a different result • Equally crazy — changing the approach and expecting to get the same results Overview – May 2025 Forward-Looking Statements This presentation contains "forward-looking statements" within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts conta ...

SAN DIEGO, June 12, 2025 /PRNewswire/ -- Robbins Geller Rudman & Dowd LLP announces that purchasers or acquirers of Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) securities between February 27, 2025 and May 26, 2025, inclusive (the "Class Period"), have until August 11, 2025 to seek appointment as lead plaintiff of the Rocket Pharmaceuticals class action lawsuit. Captioned Ho v. Rocket Pharmaceuticals, Inc., No. 25-cv-10049 (D.N.J.), the Rocket Pharmaceuticals class action lawsuit charges Rocket Pharmaceutica ...

WASHINGTON, June 10, 2025 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the first dose in the first-in-human clinical trial to evaluate the safety and tolerability of VCA-894A, an antisense oligonucleotide (ASO) therapeutic, for a patient with a rare variant in the IGHMBP2 gene causing Charcot-Marie-Tooth disease Type 2S (CMT2S). VCA-894A is being developed for a patient who was first diagnosed at the age of 5 with a rare subtype of CMT disease known as CMT2S.1 CMT2S is a ...

Gary Ingenito, MD, PhD is retiring from Catalyst after a successful careerCORAL GABLES, Fla., June 02, 2025 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX) today announced the appointment of William (Will) T. Andrews, MD, FACP as Chief Medical Officer. Dr. Andrews will report directly to the Company’s President and Chief Executive Officer, Richard J. Daly, and will serve as a member of the Company’s executive leadership team. He succeeds Dr. Gary Ingenito, who is ...

PRINCETON, N.J., May 30, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that management will participate in upcoming presentations at the following investor conferences in June. Jefferies Global Healthcare Conference 2025 in New York, NY, on Wednesday, June 4, 2025, at 11:40 a.m. ETGoldman Sachs 46th Annual Global Healthcare Conference 2025 in Miami, FL, on Tuesday, June 10, 2025, at 2:00 p.m. ET A live audio webcast of each presentation can also be accessed via the investors se ...

- Reports continued progress in the evaluation of assets for potential in-licensing and development in the areas of oncology and rare disease - - New data from pilot phase of ongoing CheMo4METPANC Phase 2b combination trial of motixafortide in PDAC, sponsored by Columbia University, to be presented at upcoming 2025 ASCO Annual Meeting – - APHEXDA performing well under Ayrmid stewardship - - Management to host conference call today, May 27th, at 8:30 am EDT - TEL AVIV, Israel, May 27, 2025 /PRNewswire/ -- Bi ...

REDWOOD CITY, Calif., May 15, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced poster presentations from its VYKAT XR (diazoxide choline) extended-release tablets, previously referred to as DCCR, clinical development program for the treatment of hyperphagia associated PWS at the Pediatric Endocrine Society (PES) Meeting 2025, which is being held May 15-18, in National Harb ...

Scholar Rock Holding (SRRK) Q1 2025 Earnings Call May 14, 2025 08:15 AM ET Company Participants Rushmie Nofsinger - Vice President of Corporate Affairs & Investor RelationsDavid Hallal - CEO & ChairmanAkshay Vaishnaw - President, R&DKeith Woods - COOVikas Sinha - CFOMichael Yee - Managing DirectorDavid Nierengarten - Managing Director - Equity ResearchGary Nachman - Managing Director - Equity ResearchMalcolm Hoffman - Senior BioPharma Equity Research AssociateAndres Maldonado - Vice President Conference Cal ...

Core Viewpoint - Applied Therapeutics, Inc. is focused on advancing its lead drug candidate, govorestat, for the treatment of rare diseases, particularly CMT-SORD and Classic Galactosemia, while preparing for upcoming regulatory interactions and clinical data presentations [2][6]. Recent Highlights - The company will present full 12-month clinical data and new topline 18-month and 24-month data from the INSPIRE Phase 3 trial of govorestat at the Peripheral Nerve Society 2025 Annual Meeting [5][6]. - Ongoing reviews of govorestat development programs for Classic Galactosemia and CMT-SORD Deficiency are progressing as planned [5][6]. Financial Results - Cash and cash equivalents and short-term investments totaled $50.8 million as of March 31, 2025, down from $79.4 million at December 31, 2024 [6]. - Research and development expenses for Q1 2025 were $7.8 million, a decrease from $12.2 million in Q1 2024, primarily due to reduced clinical and regulatory expenses [6]. - General and administrative expenses increased to $17.7 million in Q1 2025 from $9.1 million in Q1 2024, mainly due to higher legal and professional costs [6]. - The net loss for Q1 2025 was $21.8 million, or $0.15 per share, compared to a net loss of $83.9 million, or $0.67 per share, in Q1 2024 [6][13].

Pre-specified interim analysis of the ongoing Phase 3 PIVOT-PO clinical trial of tebipenem HBr in complicated urinary tract infection (cUTI), including acute pyelonephritis (AP), on track to be completed in Q2 2025Existing cash, together with earned development milestones from GSK, provide runway to fund operating expenses and capital expenditures into Q2 2026 CAMBRIDGE, Mass., May 13, 2025 (GLOBE NEWSWIRE) -- Spero Therapeutics, Inc. (Nasdaq: SPRO), a clinical-stage biopharmaceutical company, focused on i ...