Linsitinib

- Linsitinib is the first and only oral small molecule therapy to establish statistical and clinical significance in Thyroid Eye Disease (TED) - Phase 2b/3 LIDS trial met primary endpoint of proptosis reduction with statistical significance at 150mg BID dose - Linsitinib was well-tolerated particularly in IGF-1R areas of interest such as hearing impairment, glycemic changes, and menstrual cycle changes - Confirmatory Phase 3 trial on track to commence in 2025 ANN ARBOR, Mich., Jan. 14, 2025 /PRNewswire/ -- Sling Therapeutics, Inc., a late-stage biopharmaceutical company focused on the development of an oral small molecule for the treatment of thyroid eye disease (TED), today announced topline efficacy and safety data from the Phase 2b/3 LIDS trial of linsitinib in patients with active, moderate to severe TED. Linsitinib, Sling's lead product candidate, is a convenient oral small molecule, taken twice-daily, in clinical development for TED. Linsitinib works by inhibiting the validated IGF-1R target and has an established safety profile through treatment of more than 900 patients across fifteen clinical trials in multiple disease areas. "The positive data from this trial establish the clinical significance of linsitinib and represent the first ever successful clinical trial of an oral small molecule for the treatment of TED," said Ryan Zeidan, Ph.D., President and Chief Executive Officer of Sling Therapeutics. "We believe linsitinib can be a potential new treatment option that could enable a broader number of physicians across multiple therapeutic disciplines to treat patients diagnosed with TED. We are excited to continue our clinical program and are on track to initiate our confirmatory Phase 3 registrational trial later this year." Trial Overview and Topline Results The LIDS Phase 2b/3, randomized, double-masked, placebo-controlled study was designed to evaluate the safety, pharmacokinetics, and efficacy of linsitinib in patients with active, moderate to severe TED. The primary endpoint of the study was the percentage of subjects who were proptosis responders at week 24, defined as at least a two-millimeter reduction in proptosis from baseline. The LIDS trial enrolled 90 patients who were randomized 1:1:1 and received either linsitinib 150mg BID, linsitinib 75mg BID, or placebo for 24 weeks. The trial was conducted globally at 35 sites in five countries. The LIDS trial met its primary endpoint with statistical significance for the 150mg BID dose. Linsitinib in this trial validated the safety profile seen in the prior oncology studies and importantly demonstrated a favorable safety profile on key adverse events (AEs) of interest for the IGF-1R target such as hearing impairment, hyperglycemia, and menstrual cycle changes. Additionally, no QTc prolongation was observed in any patient with rigorous ECG monitoring throughout the study. Key Topline Data: Statistically Significant Proptosis Responder Rate (PRR): PRR was statistically significant and clinically meaningful at the 150mg BID dose, with a PRR of 52% (p = 0.01) at Week 24. Consistent Safety Data, No Drug-Related Hearing Impairments: Linsitinib was well-tolerated and consistent with the safety profile of the previous 15 linsitinib clinical trials, which now includes more than 900 patients in multiple disease areas. Key AE data from the 150mg BID arm include: No drug-related hearing impairment reported (1 unrelated report out of 29 patients) 0% tinnitus placebo-adjusted rate 3% rate of hyperglycemia (1 report out of 29 patients with no intervention required) 0% menstrual cycle changes reported Treatment emergent AEs greater than or equal to 10%: diarrhea (20.7%), headache (20.7%), nausea (20.7%), fatigue (17.2%), ALT increase (17.2%), hyperhidrosis (13.8%), AST increase (10.3%), and muscle spasms (10.3%) Hepatic transaminases were all quickly resolved and not associated with any elevations of total bilirubin, alkaline phosphatase, hepatic dysfunction, or other signs or symptoms of drug-induced liver injury The majority of AEs were either mild or moderate, reversible, and quickly resolved upon treatment pause or discontinuation consistent with a shorter half-life treatment No QTc prolongation was observed in any patient with rigorous monitoring throughout the study "TED is a debilitating auto-immune disorder with limited options available for patients today. Patients must currently choose between an invasive orbital surgery or eight infusions over 24 weeks, which can be inconvenient and time-consuming and pose serious potential risks," said Raymond Douglas, M.D., Ph.D., Professor at Cedars-Sinai Medical Center and KOL and lead investigator in previous TED trials, as well as Chief Scientific Officer at Sling Therapeutics. "In this trial, patients demonstrated significant improvement in disease with no drug-related hearing impairments or significant hyperglycemia. These side effects are the largest barriers for current medical treatments, making linsitinib an important potential new therapy for patients with TED. As a practicing physician, it makes sense to start a new patient's treatment journey with an oral therapy that shows an early response that increases over time." The Company is engaging with regulatory authorities to discuss the confirmatory Phase 3 trial design, which is on track to commence in 2025. Full results of this Phase 2b/3 trial will be presented at an upcoming medical conference. About Linsitinib Linsitinib is a convenient oral small molecule, taken twice-daily and with a short half-life, in clinical development for thyroid eye disease (TED). Linsitinib works by inhibiting the IGF-1R target, which is the only validated clinical target in TED and the only target for current FDA-approved therapies. Activation of the IGF-1R target leads to inflammation and proptosis seen in TED. Linsitinib has an established safety profile through treatment of more than 900 patients in fifteen clinical trials in multiple diseases. About Thyroid Eye Disease (TED) Thyroid Eye Disease (TED) is a serious, progressive, and vision-threatening rare autoimmune disease that affects approximately 70,000 people in the U.S. and has a similar prevalence in the EU. TED often occurs in people living with Graves' disease and hyperthyroidism and is caused by dysfunction in the IGF-1R signaling pathway which results in fibrous tissue growth behind the eyes. This leads to several negative symptoms that may have long-term, irreversible damage as the tissue growth pushes the eyes forward or causes the eyes and eyelids to become red and swollen. As the disease progresses it can lead to pain, eye bulging (proptosis), and double vision (diplopia), thus dramatically impacting a patient's quality of life. TED predominantly affects women, and most frequently affects people with hyperthyroidism due to Graves' disease. Current standard of care typically involves either invasive orbital surgery or a lengthy series of infusions with potential adverse events like loss of hearing, hyperglycemia or menstrual cycle changes. About Sling Therapeutics Sling Therapeutics, Inc., is a late-stage biopharmaceutical company focused on the development of an oral small molecule for the treatment of thyroid eye disease (TED). The company's lead product candidate, linsitinib, works by inhibiting the validated IGF-1R target. It recently completed a Phase 2b/3 clinical trial for the treatment of TED and is on target for initiating its confirmatory Phase 3 trial in 2025. Based on extensive preclinical and clinical data from more than 900 patients, linsitinib offers the potential of a convenient oral small molecule that could significantly reduce the treatment burden for people living with TED while improving and simplifying patient care by physicians and hospitals. For more information, please visit or follow us on LinkedIn or X (formerly known as Twitter). Investor and Media Contact: Argot Partners (212) 600-1902 [email protected] SOURCE Sling Therapeutics, Inc. 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