AAK1

Top-line Data Now Anticipated in Q1 2025 LX9211 has previously received Fast Track designation from the U.S. Food and Drug Administration (FDA) for DPNP THE WOODLANDS, Texas, Oct. 22, 2024 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced that it has closed screening and expects to complete enrollment ahead of schedule in PROGRESS (A Phase 2b, Dose-ranging, Randomized, Double-blind, PlacebO-controlled, Parallel-GRoup, MulticEnter Study in PatientS with Diabetic Peripheral Neuropathic Pain) studying LX9211, an orally-delivered, potent, selective, investigational small molecule inhibitor of adaptor-associated kinase 1 (AAK1). The Company also updated its expectations for the timing of anticipated top-line data, from the second quarter of 2025 to the first quarter of 2025. “We are excited to be ahead of schedule for a near-term completion of PROGRESS for LX9211, with top-line data now anticipated in Q1 of next year,” said Craig Granowitz, M.D., Ph.D., Lexicon’s senior vice president and chief medical officer. “There have been no new, non-opioid medications approved for neuropathic pain in more than two decades, and there is an urgent need for novel treatment options. We believe that LX9211 has the potential to positively impact the lives of people suffering with neuropathic pain and we thank the investigators, study sites, Lexicon clinical team members and especially the patients for their participation in this important study.” PROGRESS commenced in December of 2023 with the goal of enrolling adult patients with a diagnosis of diabetes (type 1 or type 2) with moderate to severe DPNP, with patients allowed to continue their current pain therapy. The primary endpoint of PROGRESS is a reduction of Average Daily Pain Score (ADPS) at 8 weeks. About Lexicon Pharmaceuticals Lexicon is a biopharmaceutical company with a mission of pioneering medicines that transform patients’ lives. Through the Genome5000™ program, Lexicon’s unique genomics target discovery platform, Lexicon scientists studied the role and function of nearly 5,000 genes and identified more than 100 protein targets with significant therapeutic potential in a range of diseases. Through the precise targeting of these proteins, Lexicon is pioneering the discovery and development of innovative medicines to treat disease safely and effectively. Lexicon has commercially launched one of these medicines, INPEFA® (sotagliflozin) in the United States, and has a pipeline of other promising drug candidates in discovery and clinical and preclinical development in neuropathic pain, diabetes and metabolism and other indications. For additional information, please visit www.lexpharma.com. About LX9211 Discovered using Lexicon’s unique approach to gene science, LX9211 is a potent, orally delivered, selective, investigational small molecule inhibitor of AAK1. Lexicon identified AAK1 in its target discovery efforts as a promising approach for the treatment of neuropathic pain. Preclinical studies of LX9211 demonstrated central nervous system penetration and reduction in pain behavior in models of neuropathic pain without affecting opiate pathways. LX9211 received Fast Track designation from the U.S. Food and Drug Administration for development in diabetic peripheral neuropathic pain. Safe Harbor Statement This press release contains “forward-looking statements,” including statements relating to Lexicon’s financial position and long-term outlook on its business, including the commercialization of its approved products and the clinical development of, regulatory filings for, and potential therapeutic and commercial potential of LX9211 and its other drug candidates. In addition, this press release also contains forward looking statements relating to Lexicon’s growth and future operating results, discovery, development and commercialization of products, strategic alliances and intellectual property, as well as other matters that are not historical facts or information. All forward-looking statements are based on management’s current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including Lexicon’s ability to meet its capital requirements, successfully commercialize its approved products, successfully conduct preclinical and clinical development and obtain necessary regulatory approvals of its other drug candidates on its anticipated timelines, achieve its operational objectives, obtain patent protection for its discoveries and establish strategic alliances, as well as additional factors relating to manufacturing, intellectual property rights, and the therapeutic or commercial value of its approved products and other drug candidates. Any of these risks, uncertainties and other factors may cause Lexicon’s actual results to be materially different from any future results expressed or implied by such forward-looking statements. Information identifying such important factors is contained under “Risk Factors” in Lexicon’s annual report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission. Lexicon undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise. For Investor and Media Inquiries:Lisa DeFrancesco Lexicon Pharmaceuticals, Inc.lexinvest@lexpharma.com

– The first healthy volunteer has been dosed in the first component of the Phase 1 study – Initial safety and pharmacokinetic data expected in Q4 2024 – Initiation of the second component of the Phase 1 study, in adult DMD patients, expected in Q1 2025 TORONTO, Canada I September 18, 2024 I Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today that the first participant has been dosed in a Phase 1 clinical study of SAT-3247. SAT-3247 is a novel, oral small molecule drug targeting AAK1 that is designed to regenerate skeletal muscle in Duchenne muscular dystrophy (DMD or Duchenne) and other degenerative or injury conditions involving muscle tissue. “We are excited to announce the dosing of the first participants in our Phase 1 clinical trial of SAT-3247, transforming Satellos into a clinical stage company. This milestone marks a significant step in our commitment to developing innovative therapies to regenerate muscle for those living with Duchenne and other muscle degenerative or injury conditions,” said Frank Gleeson, Co-founder and CEO, Satellos. “We look forward to advancing this trial and gaining valuable insights into the safety and pharmacological profile of SAT-3247.” The Phase 1 clinical trial will comprise two components. In the first component, 72 healthy volunteers will be enrolled in a blinded, randomized, placebo-controlled, staggered, parallel design study to assess the safety and pharmacokinetic properties of SAT-3247. Participants will be randomized across five single-ascending dose (SAD) cohorts, four multiple-ascending dose (MAD) cohorts, and one food effect dose cohort. In the second component, expected to begin in late Q4 2024, 10 adult volunteers with genetically confirmed DMD will be enrolled in a 28-day, open-label, single dose cohort to assess safety and pharmacokinetic properties in patients and explore potential pharmacodynamic markers. About SAT-3247 SAT-3247 is a proprietary, oral small molecule drug being developed by Satellos as a novel treatment to regenerate skeletal muscle which is lost in Duchenne muscular dystrophy (DMD or Duchenne) and other degenerative or injury conditions. Satellos is advancing SAT-3247 as a potential treatment for DMD, independent of dystrophin and regardless of exon mutation status. About Duchenne Muscular Dystrophy Duchenne muscular dystrophy is an inherited disease caused by mutations in the dystrophin gene that no longer allow the dystrophin protein to function properly. Consequently, as discovered by Satellos, muscle repair and regeneration are impaired. Satellos designed SAT-3247 to restore the process of muscle repair and regeneration by regulating a dystrophin-independent pathway with the goal of increasing muscle function. SAT-3247 is intended to work as a standalone therapeutic without regard to a patient’s genetic mutation or ambulatory status. Our approach has the potential to complement approaches designed to restore dystrophin production. About Satellos Bioscience Inc. Satellos is a publicly traded biotechnology company dedicated to developing life-improving medicines to treat degenerative muscle diseases. Satellos has incorporated breakthrough research in muscle stem cell polarity into a proprietary discovery platform, called MyoReGenX TM , to identify degenerative muscle diseases where deficits in this process affect muscle regeneration and are amenable to therapeutic intervention. With this platform, Satellos is building a pipeline of novel therapeutics to correct muscle stem cell polarity and promote the body’s innate muscle repair and regeneration process. The Company’s lead program is an oral, small molecule drug candidate in clinical development as a potential disease-modifying treatment for Duchenne muscular dystrophy. Satellos is headquartered in Toronto, Ontario. For more information, visit www.satellos.com . SOURCE: Satellos Bioscience

Lexicon Pharmaceuticals said Tuesday it will reallocate resources towards the potential US launch of Zynquista for diabetes at the end of the year and “right-size” its promotional activities for heart failure medicine Inpefa. Both drugs contain the same active ingredient of sotagliflozin, an SGLT-1/2 dual inhibitor.The company will also lay off more than 75 employees, accounting for about half of its workforce, to realise about $40 million in cost-savings for 2025. According to CEO Mike Exton, the strategic shift reflects a “plan to allocate resources… and focus on where we have the potential to lead and succeed.” Lexicon is expecting an FDA decision by December 20 on Zynquista to improve glycaemic control in adults with type 1 diabetes and chronic kidney disease. The US regulator rejected Lexicon’s efforts to win approval for Zynquista in 2019, though the drug was cleared that year in the EU for patients with type 1 diabetes and a body mass index of at least 27 who failed to achieve adequate glycaemic control despite optimal insulin therapy.  With the cost-savings initiative, Lexicon plans to make a “proper investment” in Zynquista’s launch, pending a positive decision from the FDA.  The reduced expenses will also enable the company to ensure that all of its R&D programmes are fully funded, including a Phase III study of sotagliflozin in hypertrophic cardiomyopathy and an ongoing Phase IIb trial in diabetic neuropathic pain for LX9211, a small molecule inhibitor of adaptor-associated kinase 1.