Retinoblastoma

Rein advanced the clinical development of its lead asset, LTI-03, and aims to initiate a Phase 2 trial for the treatment of idiopathic pulmonary fibrosis (IPF) in the first half of 2025 Positive topline results from Cohort 2 (5 mg BID) of the Phase 1b clinical trial of LTI-03 in IPF patients demonstrated dose dependent effects in five biomarkers evaluated, with four biomarkers achieving statistical significance in the combined Cohort 1 and Cohort 2 data set AUSTIN, Texas, April 7, 2025 /PRNewswire/ -- Rein Therapeutics, Inc. (NASDAQ: RNTX), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, today reported financial results for the fourth quarter and full year ended December 31, 2024 and provided a business update. "We've made significant clinical progress during 2024, culminating in promising safety and positive topline data from our Phase 1b trial of LTI-03 in IPF patients, which demonstrated early signs of therapeutic effect. LTI-03 has shown potential to improve lung function and potentially reverse the course of IPF, with its dual mechanism of action on both epithelial cells and fibroblasts gaining increasing support within the medical community," said Brian Windsor, Ph.D., President and Chief Executive Officer of Rein Therapeutics. "With our recent rebranding and renewed mission to combat fibrosis, we are excited to advance our pipeline. We are planning to initiate a Phase 2 study for LTI-03 in IPF in the first half of this year, where we will continue to evaluate the safety, tolerability and efficacy of this promising asset." Recent Clinical and Corporate Highlights and Upcoming Milestones Clinical Updates In November 2024, Rein announced positive topline data from Cohort 2 of the Phase 1b clinical trial evaluating the safety and tolerability of high dose LTI-03 (5 mg BID) and a set of exploratory biomarkers in patients diagnosed with IPF. Four biomarkers showed statistical significance in the combined Cohort 1 and Cohort 2 data set, and five demonstrated dose dependence, indicative of active pharmacodynamics. High dose LTI-03 continued to exhibit a favorable safety profile. A Phase 2 trial of LTI-03 for the treatment of IPF is anticipated to be initiated in the first half of this year, subject to funding. In October 2024, at the 22nd International Colloquium on Lung and Airway Fibrosis (ICLAF), Rein presented two abstracts highlighting preclinical and Phase 1b data for low dose LTI-03 (2.5 mg BID), reinforcing the potential of LTI-03 to improve lung function and reverse the course of IPF. Following inhaled administration of low dose LTI-03 in 12 patients over the course of 14 days, a positive trend was observed in biomarkers with evidence of reduced expression among multiple profibrotic proteins produced by basal-like cells and fibroblasts that contribute to the progression of IPF, including data from three biomarkers (collagen synthesis, inflammation, and fibrogenesis) that were statistically significant. Pre-clinical data further supported the potential therapeutic effectiveness of LTI-03 for IPF through precision cut lung slices (PCLS) performed ex-vivo. The studies demonstrated molecular activity in IPF PCLS explants indicative of fibrosis during five days in culture and LTI-03 broadly attenuated profibrotic proteins and pathways. Also in October 2024, the Company announced entry into an exclusive option agreement with Advancium Health Network for the acquisition of ALRN-6924, a clinical-stage oncology agent for retinoblastoma developed by the Company prior to its 2023 merger with Lung Therapeutics, Inc. Under the terms of the agreement, Rein received an upfront payment from Advancium for the exclusive option to acquire ALRN-6924 and related assets and could receive additional payments for development, regulatory and commercial milestones as well as sales royalties. Corporate Updates In January 2025, the Company rebranded to Rein Therapeutics, Inc. from Aileron Therapeutics, Inc. The new name, logo, website, and branding elements strategically aligns with the Company's sole focus to develop therapies in orphan pulmonary and fibrosis indications, including two Phase 2-ready clinical assets. The Company's common stock began trading under the Nasdaq ticker symbol "RNTX" effective January 13, 2025. Fourth Quarter and Full Year 2024 Financial Results Cash Position: Cash, cash equivalents, and investments on December 31, 2024, were $12.9 million, compared to $17.3 million on December 31, 2023. Research and Development (R&D) Expenses: R&D expenses for the quarter ended December 31, 2024, were $3.3 million, compared to $2.0 million for the quarter ended December 31, 2023. R&D expenses for the full-year 2024 were $14.2 million, compared to $4.0 million for the prior year. The increase in full year R&D expenses was primarily due to the termination of ALRN-6924 in 2023 and additional clinical programs acquired as part of the 2023 merger with Lung Therapeutics, Inc. General and Administrative (G&A) Expenses: G&A expenses for the quarter ended December 31, 2024, were $2.5 million compared to $5.3 million for the quarter ended December 31, 2023. G&A expenses for the full-year 2024 were $13.9 million, compared to $11.4 million for the prior year. The increase in full year G&A expenses was primarily due to increased headcount associated with the 2023 merger of Lung Therapeutics, Inc. and severance expense following the merger closure and increased facilities and other expenses as a result of the merger, offset by a decrease in professional fees. Net Loss: Net loss for the quarter ended December 31, 2024 was $41.0 million, compared to $7.3 million for the quarter ended December 31, 2023. The basic and diluted net loss per share for the quarter ended December 31, 2024, was $1.89 compared to a net loss per share of $1.54 for the quarter ended December 31, 2023. The increase in net loss in 2024 is mainly due to a non-cash impairment charge of $37.0 million related to aligning the carrying value of LTI-01 to the fair value. Excluding the non-cash charge, net loss per share for the fourth quarter ended December 31, 2024, was a loss of $0.26. The basic and diluted net loss per share for the full-year 2024 was $3.51, or an adjusted net loss per share of $1.53 excluding the non-cash impairment charge, compared to a net loss per share of $3.42 for the full-year 2023. About Rein Therapeutics Rein Therapeutics is a clinical-stage biopharmaceutical company advancing a novel pipeline of first-in-class therapies to address significant unmet medical needs in orphan pulmonary and fibrosis indications. Rein's lead product candidate, LTI-03, is a novel, synthetic peptide with a dual mechanism targeting alveolar epithelial cell survival as well as inhibition of profibrotic signaling. Rein's second product candidate, LTI-01, is a proenzyme that has completed Phase 1b and Phase 2a clinical trials for the treatment of loculated pleural effusions. LTI-01 has received Orphan Drug Designation in the U.S. and E.U. and Fast Track Designation in the U.S. For more information, please visit the company's website at reintx.com, or follow them on LinkedIn and X. Forward-Looking Statements This press release may contain forward-looking statements of Rein Therapeutics, Inc. ("Rein", the "Company", "we", "our" or "us") within the meaning of the Private Securities Litigation Reform Act of 1995, including statements with respect to: the timing and expectation of a Phase 2 clinical trial of LTI-03; and future expectations, plans and prospects for the Company. We use words such as "anticipate," "believe," "estimate," "expect," "hope," "intend," "may," "plan," "predict," "project," "target," "potential," "would," "can," "could," "should," "continue," and other words and terms of similar meaning to help identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks and uncertainties related to: changes in applicable laws or regulations; the possibility that the Company may be adversely affected by other economic, business, and/or competitive factors, including risks inherent in pharmaceutical research and development, such as: adverse results in the Company's drug discovery; preclinical and clinical development activities; the risk that the results of preclinical studies and early clinical trials may not be replicated in later clinical trials, including in a Phase 2 trial of LTI-03, or that partial results of a trial will be indicative of the full results of the trial; the Company's ability to enroll patients in its clinical trials; and the risk that any of its clinical trials may not commence, continue or be completed on time, or at all; decisions made by the U.S. Food and Drug Administration and other regulatory authorities; investigational review boards at clinical trial sites and publication review bodies with respect to its development candidates; the Company's ability to obtain, maintain and enforce intellectual property rights for its platform and development candidates; competition; the Company's ability to obtain additional funding and the sufficiency of the Company's cash resources, after financing, to fund its planned activities for the periods anticipated and the Company's ability to manage unplanned cash requirements; and general economic and market conditions; as well as the risks and uncertainties discussed in the "Risk Factors" section of the Company's Annual Report on Form 10-K for the year ended December 31, 2024, which is on file with the United States Securities and Exchange Commission (the "SEC") and in subsequent filings that the Company files with the SEC. These forward-looking statements should not be relied upon as representing the Company's view as of any date subsequent to the date of this press release, and we expressly disclaim any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Rein Investor Relations & Media Contact: Argot Partners [email protected] 212-600-1902 SOURCE Rein Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Biologics Licensing Application (BLA) for clemidsogene lanparvovec (RGX-121) submitted and on track for potential FDA approval 2H 2025; strategic partnership with Nippon Shinyaku aims to expand potential access and commercial opportunity in MPS II and MPS I Pivotal trial of RGX-202 for Duchenne Muscular Dystrophy progressing rapidly; enrollment completion expected in 2025 with BLA filing in mid-2026 AbbVie-partnered retinal franchise continues advancing; pivotal data evaluating the safety and efficacy of the subretinal delivery of surabgene lomparvovec (ABBV-RGX-314) in patients with wet age-related macular degeneration are expected in 2026 and planning of diabetic retinopathy pivotal study continues Conference call today at 4:30 p.m. ET ROCKVILLE, Md., March 13, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and recent operational highlights for the fourth quarter and year ended December 31, 2024. "REGENXBIO is on the cusp of delivering potential first- or best-in-class gene therapies to market. In 2025, we have already submitted our first BLA and will accelerate late-stage development and advance commercial preparations for multiple potential product launches," said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. "RGX-202 is progressing rapidly through pivotal study, continues to demonstrate a differentiated profile, and remains on track to be the next gene therapy in Duchenne. Our partnership with AbbVie continues to advance multiple large, global commercial opportunities. Now partnered with Nippon Shinyaku, RGX-121 is poised to bring another meaningful revenue stream to REGENXBIO if approved in the coming months. We remain focused on bringing multiple potentially transformative new medicines to patients." PROGRAM HIGHLIGHTS AND MILESTONES Neurodegenerative Disease: Clemidsogene lanparvovec (RGX-121) is a potentially first-in-class treatment for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, being developed and potentially commercialized in partnership with Nippon Shinyaku. In March 2025, REGENXBIO completed its Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA), seeking accelerated approval of clemidsogene lanparvovec. REGENXBIO expects potential approval of clemidsogene lanparvovec in the second half of 2025. FDA approval could result in receipt of a Priority Review Voucher (PRV). REGENXBIO retains full rights to the PRV. Clemidsogene lanparvovec remains on track to be the potential first gene therapy and one-time treatment approved for MPS II. Neuromuscular Disease: RGX-202 is designed to deliver a differentiated, novel microdystrophin gene for improved function and outcomes for patients living with Duchenne. The pivotal Phase I/II/III AFFINITY DUCHENNE® trial is ongoing in ambulatory patients and will enroll approximately 30 patients aged 1+ in the U.S. and Canada. In November 2024, REGENXBIO announced the first patient had been dosed in the pivotal phase. The pivotal trial is nearly 50% enrolled, and REGENXBIO expects to complete enrollment in the study in 2025, share top line data in the first half of 2026, and submit a BLA under the accelerated approval pathway in mid-2026. Results to date from the ongoing Phase I/II trial demonstrate a favorable safety profile with no SAEs or AEs of special interest, robust microdystrophin expression, and improved functional outcomes at 9 and 12 months, supporting the potential of RGX-202 to be a differentiated gene therapy in Duchenne. The latest results were published in November 2024 (press release). REGENXBIO expects to share additional Phase I/II biomarker data at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, including the first biomarker data from the cohort of patients aged 1-3. The company expects to share additional efficacy and safety data, including additional functional data, in the first half of 2025. Retinal Disease: Surabgene lomparvovec (sura-vec, ABBV-RGX-314), developed in collaboration with AbbVie, is potentially the first-in-class treatment for wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR). Sura-vec for the Treatment of DR (Suprachoroidal Delivery) AbbVie and REGENXBIO announced in January 2025 that they will plan a Phase III clinical program. The program is expected to support global regulatory submissions. The Phase II ALTITUDE® trial is enrolling a cohort of patients with center-involved diabetic macular edema (DME). Patients will receive a one-time, in-office injection of sura-vec at dose level 4 (1.5x10e12 GC/eye) with short course prophylactic steroid eye drops. Sura-vec for the Treatment of Wet AMD (Subretinal Delivery) Enrollment is ongoing in the ATMOSPHERE® and ASCENT™ pivotal trials. REGENXBIO and AbbVie expect to share topline results in 2026. Enrollment in the Phase II fellow eye sub-study evaluating the subretinal delivery of sura-vec in patients with bilateral wet AMD is complete. Positive results from this study were presented at the 2024 American Academy of Ophthalmology annual meeting, supporting the potential of sura-vec to treat bilateral disease at an expected commercial launch (press release). Sura-vec for the Treatment of Wet AMD (Suprachoroidal Delivery) The Phase II AAVIATE® trial continues enrolling a new cohort to evaluate sura-vec at dose level 4 (1.5x10e12 GC/eye). Patients in this cohort will also receive short course prophylactic steroid eye drops. CORPORATE UPDATES In March 2025, REGENXBIO announced the successful closing of its strategic partnership with Nippon Shinyaku to develop and commercialize RGX-121 for the treatment of MPS II and RGX-111 for MPS I in the United States and Asia. Per the agreement, REGENXBIO will receive $110 million up front and up to an additional $700 million if certain milestones are achieved. REGENXBIO announced the promotions of two key leaders: Ram Palanki, PharmD, from Executive Vice President of Commercial Strategy and Operations to Executive Vice President, Chief Commercial Officer. Dr. Palanki joined the company in 2018. Craig Malzahn from Senior Vice President, Technical Operations, to Executive Vice President, Product Development and Chief Technology Officer. Mr. Malzahn joined the company in 2019. FINANCIAL RESULTS Cash Position: Cash, cash equivalents and marketable securities were $244.9 million as of December 31, 2024, compared to $314.1 million as of December 31, 2023. The decrease was primarily driven by cash used to fund operating activities during the year ended December 31, 2024, and was partially offset by $131.1 million of aggregate net proceeds received from the follow-on public offering of the Company's common stock and pre-funded warrants completed in March 2024. Revenues: Revenues were $21.2 million and $83.3 million for the three months and full year ended December 31, 2024, respectively, compared to $22.2 million and $90.2 million for the three months and full year ended December 31, 2023, respectively. The decreases were primarily attributable to Zolgensma royalty revenues, which decreased from $85.3 million in 2023 to $81.5 million in 2024. Research and Development Expenses: Research and development expenses were $50.4 million and $208.5 million for the three months and full year ended December 31, 2024, respectively, compared to $55.7 million and $232.3 million for the three months and full year ended December 31, 2023, respectively. The decrease for the full year ended December 31, 2024 was primarily attributable to lower personnel-related costs, manufacturing and clinical supply costs for lead product candidates and early-stage research and development activities, and was partially offset by increases in clinical trial expenses for ABBV-RGX-314 and RGX-202. The decrease for the fourth quarter of 2024 was largely driven by personnel-related costs and clinical trial expenses as compared to the fourth quarter of 2023. General and Administrative Expenses: General and administrative expenses were $20.1 million and $76.6 million for the three months and full year ended December 31, 2024, respectively, compared to $19.1 million and $88.5 million for the three months and full year ended December 31, 2023, respectively. The decrease for the full year ended December 31, 2024 was primarily attributable professional services, consulting fees and other corporate overhead expenses. The increase for the fourth quarter of 2024 was largely driven by stock-based compensation expense as compared to the fourth quarter of 2023. Net Loss: Net loss was $51.2 million, or $1.01 basic and diluted net loss per share, for the three months ended December 31, 2024, compared to a net loss of $62.9 million, or $1.43 basic and diluted net loss per share, for the three months ended December 31, 2023. Net loss was $227.1 million, or $4.59 basic and diluted net loss per share, for the year ended December 31, 2024, compared to a net loss of $263.5 million, or $6.02 basic and diluted net loss per share, for the year ended December 31, 2023. FINANCIAL GUIDANCE REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $244.9 million as of December 31, 2024 and the $110.0 million upfront payment to be received under the Nippon Shinyaku collaboration to fund its operations into the second half of 2026. This cash runway guidance is based on the Company's current operational plans and excludes the impact of any material payments that may potentially be received from partners or licensees upon the achievement of development or regulatory milestones, or upon the approval or commercialization of product candidates. CONFERENCE CALL In connection with this announcement, REGENXBIO will host a conference call and webcast at 4:30 p.m. ET today. Listeners can register for the webcast via this link. Analysts wishing to participate in the question and answer session should use this link. A replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time. ABOUT REGENXBIO Inc. REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit . FORWARD-LOOKING STATEMENTS This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, clinical trials, costs and cash flow. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timing or likelihood of payments from AbbVie or Nippon Shinyaku, the monetization of any priority review voucher, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2024, which will be filed with the U.S. Securities and Exchange Commission (SEC) in the first quarter of 2025, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at . All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Zolgensma® is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO. CONTACTS: Dana Cormack Corporate Communications [email protected] George E. MacDougall Investor Relations [email protected] SOURCE REGENXBIO Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Theriva Biologics has $11.6 million to hand, which it expects to keep the lights on until the third quarter.\n Theriva Biologics is putting more of its eggs in the VCN-01 basket as it unveils a change-up in its R&D focus.Last fall, SYN-004, also known as ribaxamase, finished a second cohort part of a phase 1b/2a test in allogeneic hematopoietic cell transplant patients for the prevention of acute graft-versus-host-disease. The Maryland biotech has since been given the regulatory sign-off to move the β-lactamase enzyme into a third cohort.But not so fast: In its full-year financials posted March 6, Theriva said it could only do that with new “grant funding, or [by finding] a licensee or partner,” given its limited cash supply. Instead, the focus of its current funding and research teams will now be VCN-01, an oncolytic adenovirus that is in multiple early or midstage trials for several conditions, including metastatic pancreatic ductal adenocarcinoma and refractory retinoblastoma.The former test is currently in phase 2 trials with key data expected in the coming months, and the biotech seeks to move on to phase 3 should results go its way.In refractory retinoblastoma, meanwhile, VCN-01 has seen positive phase 1 data, according to Theriva, which hopes to work with “key opinion leaders to develop a potential pivotal trial protocol for discussion with regulatory agencies.”Theriva has $11.6 million in cash, which it expects to keep the lights on until the third quarter of this year. Shares in the biotech were up more than 7% after-hours Thursday.