LX101 (Innostellar)

NEW YORK, June 05, 2023 (GLOBE NEWSWIRE) -- Lirum Therapeutics, Inc. (“Lirum”), an innovative clinical-stage biopharmaceutical company focused on the treatment of debilitating diseases, today announced the presentation of positive data at the 7th Cancer World Congress in Palermo, Italy. LX-101, a novel clinical-stage payload-bearing targeted therapy directed to the insulin-like growth factor 1 receptor (IGF-1R), demonstrated potent anti-tumor activity against cell lines of pediatric cancers with well-established IGF-1/IGF-1R pathway involvement. These include cancers with known genetic alterations affecting the IGF-1/IGF-1R pathway and/or with high IGF-1R expression such as Ewing’s and related sarcomas, rhabdomyosarcoma, osteosarcoma, and neuroblastoma. LX-101 was active, with IC50s ranging from 6 nM to 33 nM, against a variety of pediatric cancer cell lines including Ewing’s sarcoma (with EWSR1-FLI1 and EWSR1-ERG gene fusions), alveolar rhabdomyosarcoma (with the PAX3-FOXO1 gene fusion), osteosarcoma, and neuroblastoma. The audio poster presentation from the conference is available on the Lirum website ( ) under the News and Events tab. Given these promising results, and the existing clinical data, Lirum is planning new clinical trials with LX-101 in these indications that carry strong ties to the IGF-1/IGF-1R pathway. In parallel, Lirum is also planning to develop LX-101 in IGF-1/IGF-1R-involved adult cancers as well as certain autoimmune diseases, including thyroid eye disease (TED) where IGF-1R has been clinically and commercially validated. About LX-101 Lirum’s lead product candidate, LX-101, is a novel, clinical-stage, next generation, precision-engineered, payload-bearing targeted therapy directed to the insulin-like growth factor 1 receptor (IGF-1R). The IGF-1/IGF-1R pathway has been implicated in a host of malignancies and autoimmune diseases, including thyroid eye disease (TED), and which Lirum believes represents a scientifically and medically rational target in both oncology and autoimmune indications. LX-101 consists of a proprietary, engineered variant of the IGF-1 ligand, conjugated to a cytotoxic payload, methotrexate, and harbors a novel and differentiated mechanism of action versus current and past IGF-1R targeted approaches. LX-101 (formerly 765IGF-MTX) has been evaluated in Phase 1 clinical trials of patients with advanced, pretreated solid and hematologic cancers. It was found to be well-tolerated and demonstrated single agent activity. No dose limiting toxicity or maximum tolerated dose were reached, and Lirum intends to explore further dose escalation and schedule optimization as well as focus on those indications with strong ties to the IGF-1/IGF-1R pathway. About Lirum Therapeutics, Inc. Lirum is an innovative clinical-stage biopharmaceutical company focused on the treatment of debilitating diseases through the acquisition, development and commercialization of novel drug candidates with compelling mechanisms of action, regulatory pathways and commercial opportunities. Lirum’s lead candidate, LX-101, is a novel clinical-stage targeted therapy directed to the insulin-like growth factor 1 receptor (IGF-1R) with a differentiated mechanism of action. Lirum is developing LX-101 in oncology and autoimmune indications, including thyroid eye disease (TED). For more information on Lirum, please visit . Forward Looking Statements This press release contains certain “forward-looking statements” within the meaning of the “safe harbor” provisions of the US Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “target,” “believe,” “expect,” “will,” “may,” “anticipate,” “estimate,” “would,” “positioned,” “future,” and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on Lirum’s current beliefs, expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of our control. Actual results and outcomes may differ materially from those indicated in the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. Important factors that could cause actual results and outcomes to differ materially from those indicated in the forward-looking statements include, among others, the following: (1) the ability of Lirum to successfully develop its product candidates, including obtaining positive results from planned clinical trials, 2) expectations for the clinical development, manufacturing, regulatory approval and commercialization of our product candidates or other products we may acquire or in-license(3) expectations for incurring capital expenditures and generating revenue, 4) estimates of the sufficiency of our existing cash and cash equivalents and investments to finance operations, 5) changes in applicable laws or regulations; (6) the possibility that Lirum may be adversely affected by other economic, business, and/or competitive factors; (7) the impact of health epidemics, including the COVID-19 pandemic, on Lirum’s business and the actions Lirum may take in response thereto; and (8) other risks and uncertainties indicated from time to time.; . There may be additional risks that Lirum considers immaterial or which are unknown. Any forward-looking statement made by us in this press release is based only on information currently available to Lirum and speaks only as of the date on which it is made. Lirum undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise, except as required by law. Also, the information found on our website is not incorporated by reference into this press release and is included for reference purposes only. Company contact: Matt Hoberman Investor Relations IR@lirumtx.com Phone: (646) 389-6015 Lirum Therapeutics, Inc. 590 Madison Avenue 21st Floor New York, NY 10022

ATSN-201 leverages AAV capsid that spreads laterally beyond subretinal injection site to facilitate safe delivery of RS1 to photoreceptors in the central retina/fovea Initiation of Phase I/II clinical trial, known as The Lighthouse Study, anticipated in mid-2023 DURHAM, N.C., May 01, 2023 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for a Phase I/II clinical trial of ATSN-201 in patients with X-linked retinoschisis (XLRS). ATSN-201 leverages one of the company’s novel spreading capsids, AAV.SPR, to overcome the challenges associated with intravitreally delivered AAVs in the treatment of XLRS. “Intravitreally delivered AAVs have limitations, as they do not drive sufficient gene expression in photoreceptors to confer therapy and can lead to vision-compromising inflammation,” said Shannon Boye, PhD, Founder and Director of Atsena Therapeutics. “AAV.SPR is well-suited for use in XLRS as it can drive therapeutic levels of gene expression in photoreceptors while avoiding the surgical risks of foveal detachment, which is important because XLRS patients have fragile retinas due to the presence of schisis lesions. Building on decades of research, we’re excited to progress our novel gene therapy for patients with XLRS who currently lack an approved treatment option.” “With the FDA’s clearance of the IND application for ATSN-201, we’re preparing to advance our first program utilizing AAV.SPR into the clinic for the treatment of XLRS in mid-2023,” said Kenji Fujita, Chief Medical Officer of Atsena Therapeutics. “We look forward to evaluating ATSN-201 and addressing the unmet need for a treatment to improve or restore vision in patients with XLRS.” The Lighthouse Study, a Phase I/II, open-label, dose-escalation clinical trial, will evaluate subretinal injection of ATSN-201 in male patients ages 6-65 with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. About X-linked Retinoschisis (XLRS) XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of the layers of the retina, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments. About AAV.SPR AAV.SPR, one of Atsena’s novel spreading capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in stark contrast to benchmark vector AAV5, which remains confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and does not cause inflammation. For more information about the preclinical study and how AAV.SPR works, visit . About Atsena Therapeutics Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company’s ongoing Phase I/II clinical trial is evaluating ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1), one of the most common causes of blindness in children. Interim safety and efficacy data has demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 6 months post-treatment. The company’s additional pipeline of proprietary assets includes ATSN-201, an investigational gene therapy that leverages one of the company’s novel spreading capsids, AAV.SPR, for the treatment of X-linked retinoschisis (XLRS), and ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com. Media Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com Business Contact: info@atsenatx.com