Core Insights - Idorsia Ltd announces the presentation of long-term results from the lucerastat treatment at the 22nd Annual WORLDSymposium™, highlighting its potential as a first-in-class oral substrate reduction therapy for Fabry disease [1][7] Study Overview - The MODIFY study was a multicenter, double-blind, randomized, placebo-controlled trial involving 118 patients to assess the efficacy and safety of lucerastat as an oral monotherapy for Fabry disease [3] - The study did not meet its primary endpoint of reducing neuropathic pain but showed significant reductions in plasma and urinary Gb3 levels compared to placebo, sustained over time in the open-label extension [4][14] Long-term Efficacy - An interim analysis of the open-label extension revealed a notable reduction in the rate of eGFR decline among patients treated with lucerastat, suggesting a potential disease-modifying effect [5] - Patients with impaired renal function at baseline experienced a marked attenuation of kidney function loss, indicating lucerastat's promise in long-term organ protection [5][14] Kidney Biopsy Sub-study - A kidney biopsy sub-study evaluated Gb3 accumulation in key kidney cell types in male patients with classic Fabry disease who received lucerastat for at least two years, providing insights into its impact on renal disease biology [2] Patient Population and Disease Background - Fabry disease is a rare, X-linked lysosomal storage disorder caused by mutations in the GLA gene, leading to the accumulation of Gb3 and progressive damage across multiple organ systems [6][8] - Recent studies suggest a higher prevalence of Fabry disease than previously estimated, with over 21,000 diagnosed patients expected across the US, EU5, and Japan by 2034 [9] Current Treatment Landscape - Current treatment options for Fabry disease include enzyme replacement therapies and oral chaperone therapy, which have limitations, highlighting the unmet need for a well-tolerated, oral, disease-modifying therapy [10][11]

Nuvation Bio Inc. (NYSE:NUVB) is one of the best high short interest stocks with biggest upside potential. On January 27, Truist raised its price target on Nuvation Bio to $13 from $11 and maintained a Buy rating, citing the Ibtrozi franchise’s robust initial performance as part of a Q4 2025 earnings preview for the biotech sector. Despite seasonal challenges, the firm observed stronger-than-expected market uptake, which positions Ibtrozi as the preferred ROS1 agent over its competitors. Earlier on Januar ...

Mineralys Therapeutics Inc. (NASDAQ:MLYS) is one of the best high short interest stocks with biggest upside potential. On January 6, Mineralys Therapeutics announced the late 2025 filing of its NDA for lorundrostat. The company highlighted several clinical milestones, including the completion of three positive trials (Explore-CKD, Launch-HTN, and Advance-HTN), which demonstrated the drug’s 24-hour blood pressure control and safety across diverse patient populations. Additionally, Mineralys remains on track ...

Apellis Pharmaceuticals Inc. (NASDAQ:APLS) is one of the best high short interest stocks with biggest upside potential. On January 21, Bank of America analyst Tazeen Ahmad upgraded Apellis to Buy from Neutral with an unchanged price target of $28. BofA maintains that the market is currently undervaluing Empaveli’s commercial prospects in treating rare kidney diseases, citing a robust launch trajectory that has already secured 267 new patient start forms since its late July approval. This initial 5% penetra ...

As filed with the Securities and Exchange Commission on January 29, 2026. Registration No. 333-292059 ________________________________________________ AMENDMENT NO. 1 TO FORM S-1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 ________________________________________________ STARTON HOLDINGS, INC. UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 (Exact name of registrant as specified in its charter) DELAWARE (Jurisdiction of incorporation or organization) _____________________ ...

LA JOLLA, Calif., Jan. 29, 2026 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875), today announced that as of the end of January 2026, 12 sites in the US are activated and 100 patients have been enrolled in the SEANOBI study (Scalable Expanded Access with Analysis of Neurofilament and Other Biomarkers in ALS; NCT 06743776) representing 50% of the planned 200-enrollment, ev ...

Group 1 - Altimmune, Inc. has closed a registered direct offering of 17,045,454 shares, generating approximately $75 million in gross proceeds before fees and expenses [1][2] - The funds will be used for the upcoming Phase 3 trial in metabolic dysfunction-associated steatohepatitis (MASH) and for general corporate purposes [2] - The offering was facilitated by Titan Partners, acting as the sole placement agent [3] Group 2 - Pemvidutide, Altimmune's lead candidate, is a dual-action therapy targeting glucagon and GLP-1 receptors, showing potential as a differentiated treatment for MASH [2][6] - The company has received Breakthrough Therapy Designation from the U.S. FDA for pemvidutide, indicating its significance in addressing serious liver diseases [2] - Altimmune is focused on delivering long-term value for shareholders while enhancing its operational flexibility through this investment [2]

THE WOODLANDS, Texas, Jan. 29, 2026 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) (“Lexicon”) today announced that it has commenced an underwritten public offering to offer and sell, subject to market and other conditions, shares of its common stock, par value $0.001. In addition, Lexicon intends to grant the underwriters a 30-day option to purchase additional shares of common stock. There can be no assurance as to whether or when the proposed offering may be completed, or as to the actua ...

RAMAT GAN, ISRAEL, Jan. 29, 2026 (GLOBE NEWSWIRE) -- XTL Biopharmaceuticals Ltd. (Nasdaq:XTLB) (TASE:XTLB.TA) (the “Company” or “XTL”), announced that it is working diligently to close the acquisition of 85% of the shares of NeuroNOS Ltd. from Beyond Air Inc, following the execution of the Binding Letter of Intent on January 13 , 2026 (as disclosed in the Company’s Report of Foreign Private Issuer on Form 6-K filed on January 13, 2026), and has already scheduled a shareholders meeting for February 17, 2026 ...

Argenx SE (NASDAQ:ARGX) is one of the 11 Best Stocks to Buy for Investment. On January 20, TheFly reported that Wells Fargo increased the price target on Argenx SE (NASDAQ:ARGX) from $1,264 to $1,317, keeping an Overweight rating on the shares. Wells Fargo Analysts Sees Potential in Argenx SE's Vyvgart, Increases PT To $1,317 Pixabay/Public Domain Derek Archila from Wells Fargo raised the price target on ARGX following its strong outlook on Argenx’s Vyvgart, according to TheFly. Archila has raised the ...