Corneal Dystrophies, Hereditary

Completes Transition to Immune-Mediated Disease Focused Company Highlights Additional Data from Phase 1b Primary Membranous Nephropathy (pMN) Study with plans to Advance to Late Phase Development in 2025 Outlines Plans for Clinical Trials in Systemic Lupus Erythematosus (SLE) and Immune Thrombocytopenia (ITP) with First Patients Dosed Targeted for 1H25 Cash runway through 2027 Expected to Enable Delivery of Key Value Inflection Points Virtual Investor Event to be Held at 12:00 p.m. ET Today WELLESLEY HILLS, Mass., Oct. 15, 2024 (GLOBE NEWSWIRE) -- Climb Bio, Inc. (Nasdaq: CLYM), will be hosting a virtual investor event, today, October 15, 2024, from 12:00 p.m. to 2:00 p.m. ET. The event will feature members of Climb Bio’s management team who will provide an update on the Company’s development strategy for budoprutug, an anti-CD19 monoclonal antibody with potential to treat a broad range of B-cell mediated diseases. The event will also feature Dr. Frank Cortazar, Director of the New York Vasculitis and Glomerular Center and Chief of Clinical Research at St. Peter's Health Partners, who was the primary investigator in the Phase 1b trial of budoprutug in pMN. Dr. Cortazar will present additional data from the completed pMN trial and share clinical data from a single patient with Minimal Change Disease (MCD) treated with budoprutug. “Our mission at Climb Bio is to develop better treatments for the approximately 50 million patients in the U.S. and many more globally living with immune-mediated diseases,” said Aoife Brennan, President and CEO of Climb Bio. “We are off to a great start with some world class talent and a cornerstone asset, budoprutug. We believe CD19 is the optimal target for B-cell mediated diseases and that an antibody is the modality with the broadest potential to help patients. We are thrilled today to share our development strategy and promising preclinical and clinical data that underscore our enthusiasm for budoprutug’s potential.” Dr. Cortazar added, “I know firsthand the challenges of managing patients with pMN and the need for new treatment options. The patients in the pMN trial demonstrated high rates of complete clinical remission, as well as rapid reductions in B-cells and anti-PLA2R antibodies, two key biomarkers for pMN. These data potentially support a significant advancement in our fight against this challenging condition, and I am hopeful to continue to help advance this innovative therapy for those in need.” Event Highlights Rational and data driven clinical development strategy The Company’s strategic approach to developing budoprutug will consider indications across three opportunity sets, including; IgG4-mediated diseases, such as pMN;Diseases caused by IgG1-3 that impact a single organ system, such as ITP; andComplex systemic diseases, such as SLE. Budoprutug is a differentiated anti-CD19 monoclonal antibody with potential for potent B-cell depletion The Company will share data supporting budoprutug’s potential for deep and durable depletion of both peripheral and tissue-resident B-cells in patients with immune-mediated diseases In a transgenic mouse model expressing human CD19, budoprutug achieved dose-dependent B-cell depletion in bone marrow, lymph nodes, and spleen.Doses as low as 100 mg induced B-cell depletion to undetectable levels in pMN patients treated with budoprutug. Continued advancement of subcutaneous formulation of budoprutug Budoprutug has been successfully formulated above 175 mg/ml while maintaining low viscosity, creating an opportunity to pursue a subcutaneous dosing form that potentially features a low volume injection.The Company plans to continue to advance the subcutaneous formulation clinical program, with non-clinical data expected in the first half of 2025. Additional promising Phase 1b Study Results in pMN Dr. Frank Cortazar, Director of the New York Nephrology Vasculitis and Glomerular Center, will present additional data from the Phase 1b study of budoprutug in patients with pMN.The study demonstrated the following for the 5 patients who received all 4 doses of budoprutug: Complete remission of proteinuria achieved by 3/5 (60%) of patients.Rapid and significant reductions in anti-PLA2R autoantibodies, a key driver of pMN, with serological remission occurring in the 3 patients that were PLA2R positive at baseline.Complete and sustained B-cell depletion was observed in all patients, with undetectable levels of B-cells occurring after just two doses of study drug as low as 100 mg.Budoprutug was generally well-tolerated, with no reported drug-related serious adverse events. Full data from this study will be presented in a poster presentation on October 24th at the American Society of Nephrology’s Kidney Week 2024 being held in San Diego, CA.Dr. Cortazar will also share data from a single patient trial for MCD showing the MCD patient was able to fully taper steroid and remains in complete remission 18 months following final administration of budoprutug, given as 4 doses over 6 months, 600mg cumulative dose. Initiation of Phase 1b clinical trial in SLE planned for the first half of 2025, subject to regulatory clearance SLE is a complex, chronic systemic disease opportunity affecting multiple organ systems, leading to significant morbidity and mortality that affects approximately 200,000 to 300,000 people in the U.S.Design of Phase 1b trial in SLE: An open-label trial in patients with active lupus is planned.Endpoints will include clinical response, decline in pathogenic autoantibodies, and assessment of B-cell subsets upon recovery.The trial will utilize informative serum biomarkers to accelerate proof-of-concept, optimize dosing, and benchmark against other B-cell depleting therapies. Initiation of Phase 2 clinical trial in ITP planned for the first half of 2025, subject to regulatory clearance ITP is an IgG 1-3 immune-mediated disorder affecting an estimated 60,000 adults in the U.S. and where there is compelling proof-of-concept validating the clinical rationale for using B-cell depletion therapies.Design of Phase 2 trial in ITP: Single-arm, open-label study in adult patients with chronic ITP is planned.Primary endpoint will measure platelet improvement along with other disease parameters.A unique "treat-to-target" approach is included in the study protocol to allow for personalized redosing based on individual patient needs. Corporate Update and Upcoming Milestones As of September 30, 2024, the Company had cash, cash equivalents and marketable securities of approximately $218 million, which is expected to fund operations through 2027.Initiation of clinical trials in SLE and ITP with first patients dosed targeted for the first half of 2025, subject to regulatory clearance.Advancement of subcutaneous formulation clinical program, with non-clinical data expected in the first half of 2025.Advancement of pMN clinical program to late phase development expected in 2025. Event DetailsClimb Bio, Inc. will host its virtual investor event, today, October 15, 2024, from 12:00 p.m. to 2:00 p.m. ET. To attend the virtual event, please register here. The live webcast of the event, as well as a replay, will be available under "Events and Presentations" in the Investors section of the Company's website: climbbio.com. About Climb Bio, Inc. Climb Bio, Inc. is a clinical-stage biotechnology company developing therapeutics for patients with immune-mediated diseases. The Company’s lead product candidate, budoprutug, is an anti-CD19 monoclonal antibody that has demonstrated B-cell depletion and has potential to treat a broad range of B-cell mediated diseases. For more information, please visit climbbio.com. Forward-Looking StatementsThis press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including without limitation statements regarding: future expectations, plans and prospects for Climb Bio; the anticipated benefits of the acquisition of Tenet Medicines, Inc.; expectations regarding budoprutug’s therapeutic benefits, clinical potential and clinical development; the trial design for the planned clinical trials of budoprutug; plans to optimize the administration of budoprutug; the anticipated timelines for initiating clinical trials of budoprutug; the sufficiency of Climb Bio’s cash resources for the period anticipated and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “will,” “working” and similar expressions. Forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. Climb Bio may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. These risks and uncertainties include, but are not limited to, important risks and uncertainties associated with: the ability of Climb Bio to timely and successfully achieve or recognize the anticipated benefits of its acquisition of Tenet Medicines, Inc.; changes in applicable laws or regulation; the possibility that Climb Bio may be adversely affected by other economic, business and/or competitive factors; Climb Bio’s ability to advance budoprutug on the timelines expected or at all and to obtain and maintain necessary approvals from the U.S. Food and Drug Administration and other regulatory authorities; obtaining and maintaining the necessary approvals from investigational review boards at clinical trial sites and independent data safety monitoring boards; replicating in clinical trials positive results found in early-stage clinical trials of budoprutug; competing successfully with other companies that are seeking to develop treatments for systemic lupus erythematosus, immune thrombocytopenia and membranous nephropathy and other immune-mediated diseases; maintaining or protecting intellectual property rights related to budoprutug and/or its other product candidates; managing expenses; and raising the substantial additional capital needed, on the timeline necessary, to continue development of budoprutug and any other product candidates Climb Bio may develop. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Climb Bio’s actual results to differ materially from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in Climb Bio’s most recent filings with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Climb Bio’s views as of the date hereof and should not be relied upon as representing Climb Bio’s views as of any date subsequent to the date hereof. Climb Bio anticipates that subsequent events and developments will cause Climb Bio’s views to change. However, while Climb Bio may elect to update these forward-looking statements at some point in the future, Climb Bio specifically disclaims any obligation to do so, except as required by law. InvestorsChris Brinzey ICR Westwickechris.brinzey@westwicke.com 339-970-2843 MediaJon YuICR Westwickejon.yu@westwicke.com475-395-5375

MENLO PARK, Calif., Oct. 8, 2024 /PRNewswire/ -- Emmetrope Ophthalmics, LLC ("Emmecell"), a clinical-stage biotechnology company pioneering the discovery and development of cell-based therapies for the treatment of serious eye diseases, today announced the successful completion of the last patient last visit in its phase 1 extension study EMME-001, a 21-patient randomized, double-masked, US based, multi-center trial assessing the safety and efficacy of EO2002 for the treatment of corneal edema. This milestone marks a significant step forward in the development of an advanced treatment option aimed at improving visual function for patients suffering from Fuchs dystrophy, pseudophakic bullous keratopathy, and other causes of corneal edema. EO2002 is a first-in-class, non-surgical cell therapy incorporating Emmecell's exclusive Magnetic Cell Delivery (MCD) nanoparticle platform. EO2002 was studied in EMME-001 as a treatment for corneal edema without the need for invasive and high-risk surgical procedures, such as corneal transplantation or endothelial scraping. This straightforward intracameral injection is designed as a therapeutic intervention for the approximately 90% of patients with symptomatic corneal edema who do not have disease severe enough to warrant the risks of corneal transplantation surgery. Preliminary data from the phase 1 trial indicate improvement in best correct visual acuity, reduction in central corneal thickness, and no significant safety concerns, demonstrating the therapy's promise as a viable treatment alternative for individuals suffering with symptomatic corneal edema. Additional detailed topline results are expected to be reported in the next month for all subjects treated across three doses of EO2002. "The prospect of being able to treat patients without surgery would be a game-changer for patients suffering with corneal edema," said Ellen Koo, MD, professor of ophthalmology at the Bascom Palmer Eye Institute, University of Miami, and an investigator in the study. "Currently, for patients with severe enough disease, we perform corneal transplant surgery, which requires a single donor to treat a single eye. Cell therapy offers the opportunity to treat hundreds of eyes from a single donor cornea, and with EO2002's non-surgical approach, we could effectively help the much larger population of patients suffering with symptomatic corneal edema without surgery." "Completion of this trial represents a significant milestone for Emmecell, and it reflects the incredible enthusiasm from our investigators to help develop a non-surgical approach to treat corneal endothelial dysfunction," said Jeffrey L. Goldberg, MD, PhD, Emmecell co-founder, and professor and chair of ophthalmology at Stanford University. "Potential corneal endothelial cell injections could be used to treat the entire spectrum of corneal edema." Using its proprietary MCD platform, Emmecell is able to overcome some of the biggest challenges in regenerative medicine, namely how to deliver, retain and integrate cells to the appropriate target location. In addition to its lead clinical program in corneal edema, Emmecell has two other programs in earlier-stage development to treat age-related macular degeneration and glaucoma. For media inquiries, please contact: Noelia Kunzevitzky, PhD Chief Operating Officer Emmecell (650) 769-4232 [email protected] SOURCE Emmecell WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

PIONEER study expands the investigation of atacicept into a broad definition of IgA nephropathy and into multiple autoimmune glomerular diseases, supported by the disease-modifying potential of BAFF/APRIL dual inhibition;Multiple regulatory and clinical milestones expected over the next 18 months; Announcements made at Vera’s R&D Day in New York, where the company’s management team was joined by academic leaders Jonathan Barratt, Richard Lafayette, and Brad Rovin BRISBANE, Calif., Oct. 02, 2024 (GLOBE NEWSWIRE) -- Vera Therapeutics, Inc. (Nasdaq: VERA), a late clinical-stage biotechnology company focused on developing and commercializing transformative treatments for patients with serious immunological diseases, today announced expansion of its development pipeline for its lead asset, atacicept. This program is expected to build on the positive data reported to date from the ongoing ORIGIN Phase 2b and 3 clinical program developing atacicept to treat patients with IgAN, by extending into a broader population of IgAN and other autoimmune kidney indications. “Based on the positive clinical data announced over the past year, we have a greater understanding of atacicept’s disease-modifying mechanism of action and potential to be a best-in-class treatment option for patients with IgAN. We’re committed to providing long-term access to atacicept for all ORIGIN participants, and the PIONEER study will expand that opportunity to a significantly greater number of patients with IgAN,” said Marshall Fordyce, M.D., Founder and CEO of Vera Therapeutics. “We believe that B cell modulation through BAFF/APRIL dual inhibition has the potential to transform the treatment landscape for other autoimmune diseases, including autoimmune forms of primary membranous nephropathy, focal segmental glomerulosclerosis, and minimal change disease.” “We view this expansion of our pipeline as highly complementary to our lead program in IgAN. As such, we remain focused on completing the pivotal clinical program for atacicept in IgAN. We look forward to keeping everyone apprised of our progress, as we have a number of significant milestones planned across our pipeline,” concluded Dr. Fordyce. ORIGIN Extend – The company plans to initiate a study in Q4 2024 that will provide ORIGIN participants with extended access to atacicept prior to commercial availability in their region, as well as an opportunity to capture longer-term data. PIONEER Study – In 2025, the company plans to initiate a study evaluating the efficacy and safety of atacicept in: Expanded IgAN populations – The first set of cohorts will include adults with low kidney function (eGFR 20 to <30 mL/min/1.73 m2), low (UPCR <1.0 g/g) or high proteinuria (UPCR ≥5.0 g/g), or IgAN recurrence after kidney transplant; adolescents at high risk of progression (UPCR ≥0.3 g/g); as well as adolescents and adults with IgA vasculitis nephritis.Anti-PLA2R and anti-nephrin podocytopathies – The PIONEER study will expand to additional autoimmune glomerular diseases characterized by the presence of antibodies to glomerular antigens, including primary membranous nephropathy (pMN), focal segmental glomerulosclerosis (FSGS), and minimal change disease (MCD). These new indications represent a significant potential opportunity for atacicept, with the combined peak prevalence of IgAN and autoimmune-driven PMN, FSGS, and MCD in the US estimated at ~230,000. The company believes atacicept may have therapeutic potential in additional rheumatologic and hematologic indications. Vera’s management team was joined by Jonathan Barratt, MD, PhD, FRCP (University of Leicester), Richard Lafayette, MD, FACP (Stanford University Medical Center), and Brad Rovin, MD, FACP, FASN (Ohio State University Wexner Medical Center). A replay of the event is available on the Investor Calendar of the company’s website at https://ir.veratx.com or (click here). The R&D Day event was held in advance of the anticipated 96-week data from the Phase 2b ORIGIN study of atacicept in IgAN, which will be presented as a late-breaking oral presentation at the American Society of Nephrology Kidney Week 2024. About VeraVera Therapeutics is a late clinical-stage biotechnology company focused on developing treatments for serious immunological diseases. Vera’s mission is to advance treatments that target the source of immunological diseases in order to change the standard of care for patients. Vera’s lead product candidate is atacicept, a fusion protein self-administered as a subcutaneous injection once weekly that blocks both B-cell Activating Factor (BAFF) and A PRoliferation-Inducing Ligand (APRIL), which stimulate B cells and plasma cells to produce autoantibodies contributing to certain autoimmune diseases, including IgAN, also known as Berger’s disease, and lupus nephritis. In addition, Vera is evaluating additional diseases where the reduction of autoantibodies by atacicept may prove medically useful. Vera is also developing MAU868, a monoclonal antibody designed to neutralize infection with BK virus (BKV), a polyomavirus that can have devastating consequences in certain settings such as kidney transplant. Vera retains all global developmental and commercial rights to atacicept and MAU868. For more information, please visit www.veratx.com. About AtaciceptAtacicept is an investigational recombinant fusion protein that contains the soluble transmembrane activator and calcium-modulating cyclophilin ligand interactor (TACI) receptor that binds to the cytokines B-cell activating factor (BAFF) and A PRoliferation-Inducing Ligand (APRIL). These cytokines are members of the tumor necrosis factor family that promote B-cell survival and autoantibody production associated with certain autoimmune diseases, including IgAN and lupus nephritis. The Phase 2b ORIGIN clinical trial of atacicept in IgAN met its primary and key secondary endpoints, with statistically significant and clinically meaningful proteinuria reductions and stabilization of eGFR versus placebo through 36 weeks. The safety profile during the randomized period was comparable between atacicept and placebo. Through 72 weeks, atacicept demonstrated further reductions in Gd-IgA1, hematuria, and proteinuria, as well as stabilization of eGFR reflecting a profile consistent with that of the general population without IgAN. Atacicept has received FDA Breakthrough Therapy Designation for the treatment of IgAN, which reflects the FDA’s determination that, based on an assessment of data from the Phase 2b ORIGIN clinical trial, atacicept may demonstrate substantial improvement on a clinically significant endpoint over available therapies for patients with IgAN. Vera believes atacicept is positioned for best-in-class potential, targeting B cells and plasma cells to reduce autoantibodies and having been administered to more than 1,500 patients in clinical studies across different indications. Forward-looking StatementsStatements contained in this press release regarding matters, events or results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, Vera’s expectations regarding the expansion of its development pipeline for atacicept, atacicept’s potential to be a best-in-class treatment for patients with IgAN, Vera’s expectations regarding the potential for B cell modulation through BAFF/APRIL dual inhibition to transform the treatment landscape for certain autoimmune diseases, Vera’s plans to initiate a study in the fourth quarter of 2024 providing extended access to atacicept to ORIGIN participants, Vera’s plans to initiate the PIONEER study in 2025, Vera’s anticipated presentations of clinical trial data, and Vera’s product candidates, strategy, and regulatory matters. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “expanded,” “substantial,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Vera’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks related to the regulatory approval process, results of earlier clinical trials may not be obtained in later clinical trials, preliminary results may not be predictive of topline results, risks and uncertainties associated with Vera’s business in general, the impact of macroeconomic and geopolitical events, and the other risks described in Vera’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Vera undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. For more information, please contact: Investor Contact:Joyce AllaireLifeSci Advisors212-915-2569jallaire@lifesciadvisors.com Media Contact:Madelin HawtinLifeSci CommunicationsMHawtin@lifescicomms.com