RLYB-116

NEW HAVEN, Conn.--( BUSINESS WIRE )--Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company translating scientific advances into transformative therapies for patients with devastating rare diseases, today highlighted its 2024 accomplishments and announced its anticipated key milestones for 2025. “With our team’s innovation and execution throughout 2024, Rallybio is well positioned to create meaningful value in 2025,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. “We are planning for key readouts from our RLYB212 and RLYB116 clinical programs and expecting important advancements in our REV102 program throughout the year. We believe these developments will provide further evidence of the value our programs can ultimately bring to patients. With these accomplishments and continued financial discipline, the Company is poised for significant momentum in 2025 and even greater success in the years to come.” 2024 Accomplishments RLYB212 Obtained clinical trial application approvals and initiated the first-ever Phase 2 dose confirmation trial in pregnant women at higher risk for HPA-1a alloimmunization and fetal and neonatal alloimmune thrombocytopenia (FNAIT) Screened more than 14,000 pregnant women through January 1, 2025 in the Company’s ongoing FNAIT natural history study Presented results of an epidemiological analysis demonstrating FNAIT risk across racially and ethnically diverse populations, indicating that more than 30,000 pregnancies each year are at higher risk for FNAIT, at the NORD Summit and ASHG Published Phase 1b proof-of-concept results, in addition to the modeling and simulations that support the RLYB212 dose regimen for the Phase 2 trial RLYB116 Successfully completed manufacturing process enhancements, which are expected to further improve the tolerability of RLYB116 Presented biomarker characterization analyses indicating that RLYB116 led to a greater degree of complement inhibition in the Phase 1 MAD study than initially reported REV102 Advanced REV102, an ENPP1 inhibitor for the treatment of patients with hypophosphatasia (HPP) which was discovered in partnership with Recursion Pharmaceuticals Presented data at ASBMR from an early lead ENPP1 inhibitor, REV101, in a mouse model of later-onset HPP demonstrating a 30% reduction in inorganic pyrophosphate (PPi), a key biomarker that is elevated in HPP and contributes to poor bone mineralization RLYB332 Presented preclinical data for RLYB332 at ASH, including favorable pharmacodynamic (PD) data, supporting RLYB332 as a long-acting, potentially best-in-class therapy for the treatment of diseases of iron overload FNAIT Natural History Study Update Rallybio plans to conclude screening in the FNAIT natural history study in the United States and Canada as of January 31, 2025. The Company will focus resources on advancing the RLYB212 Phase 2 trial across sites in Europe, including initiation of dosing in the sentinel participant and collection of natural history data in a sub-study of the Phase 2 trial. Participants identified as having higher risk for HPA-1a alloimmunization and FNAIT in the ongoing Phase 2 trial who do not receive RLYB212 are eligible to enroll in the natural history sub-study of the Phase 2 trial. Anticipated 2025 Key Milestones RLYB212 Initiate dosing of sentinel participant in the Phase 2 trial in the second quarter of 2025 Present interim data from the FNAIT natural history study in mid-2025 Report interim safety and pharmacokinetic (PK) data from the Phase 2 trial sentinel participant in the third quarter of 2025 Completion of pregnancy, with safety and PK data readout, from the Phase 2 trial sentinel participant in the fourth quarter of 2025 RLYB116 Initiate confirmatory clinical PK/PD study in the second quarter of 2025 Cohort 1 data readout from clinical PK/PD study in the third quarter of 2025 Cohort 2 data readout from clinical PK/PD study in the fourth quarter of 2025 REV102 Initiate investigational new drug application (IND)-enabling studies in 2025 to support the initiation of a Phase 1 study in 2026 Report REV102 data from a preclinical model of later-onset HPP in the second half of 2025 About Rallybio Rallybio (NASDAQ: RLYB) is a clinical-stage biotechnology company with a mission to develop and commercialize life-transforming therapies for patients with severe and rare diseases. Rallybio has built a broad pipeline of promising product candidates aimed at addressing diseases with unmet medical needs in areas of maternal fetal health, complement dysregulation, hematology, and metabolic disorders. The Company has two clinical stage programs: RLYB212, an anti-HPA-1a antibody for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT) and RLYB116, a C5 inhibitor with the potential to treat several diseases of complement dysregulation, as well as additional programs in preclinical development. Rallybio is headquartered in New Haven, Connecticut. For more information, please visit www.rallybio.com and follow us on LinkedIn and Twitter . Forward-Looking Statements This press release contains forward-looking statements that are based on our management’s beliefs and assumptions and currently available information. All statements, other than statements of historical facts contained in this press release are forward-looking statements. In some cases, forward-looking statements can be identified by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements concerning the timing of dosing the sentinel participant in the RLYB212 Phase 2 trial, the timing of initiating the RLYB116 confirmatory PK/PD study and the date when data is available, including data for Cohorts 1 and 2, the timing of initiation of IND-enabling activities for REV102, whether anticipated 2025 milestones will result in meaningful value appreciation, whether RLYB116 will demonstrate improved tolerability at higher doses with complete and sustained complement inhibition, the timing of disclosure and characterization of preclinical data for REV102, the timing of completion of pregnancy, and data readout, for the sentinel participant for the RLYB212 Phase 2 trial, the timing of data releases for the Company’s programs, including interim data from the FNAIT natural history study, interim safety and pharmacokinetic data from the sentinel participant in the RLYB212 Phase 2 trial, and REV102 data from a preclinical model of later-onset HPP. The forward-looking statements in this press release are only predictions and are based largely on management’s current expectations and projections about future events and financial trends that management believes may affect Rallybio’s business, financial condition and results of operations. These forward-looking statements speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions, including, but not limited to, our ability to successfully initiate and conduct our planned clinical trials, including the FNAIT natural history study, and the Phase 2 trial for RLYB212, and complete such clinical trials and obtain results on our expected timelines, or at all, whether our cash resources will be sufficient to fund our operating expenses and capital expenditure requirements and whether we will be successful raising additional capital, our ability to enter into strategic partnerships or other arrangements, competition from other biotechnology and pharmaceutical companies, and those risks and uncertainties described in Rallybio’s filings with the U.S. Securities and Exchange Commission (SEC), including Rallybio’s Quarterly Report on Form 10-Q for the period ended September 30, 2024, and subsequent filings with the SEC. The events and circumstances reflected in our forward-looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward-looking statements. Except as required by applicable law, we are not obligated to publicly update or revise any forward-looking statements contained in this press release, whether as a result of any new information, future events, changed circumstances or otherwise.

Welcome to Endpoints News’ manufacturing briefs, where we bring you essential news on new builds, collaborations, recalls and more. National Resilience is replacing its CEO effective immediately with COO William Marth, who will take over from Rahul Singhvi. Marth previously served as CEO of Curia Global, known then as AMRI, as well as CEO of Teva Pharmaceutical’s US division. Moderna has opened its new mRNA manufacturing facility in Australia within the campus of Monash University. The new site will be able to make 100 million vaccine doses a year, and the first dose is anticipated to be produced next year. Around 140 new “direct” jobs will be created at the site. Reckitt Benckiser is pouring £155 million ($200 million) into a new facility in Wilson, NC, to make its over-the-counter flu and cold drug products, the company said Tuesday. This will be Reckitt’s largest OTC manufacturing site in the US and will create 300 new jobs. CDMO Cambrex is partnering with Eli Lilly’s biotech innovative arm, dubbed Lilly Catalyze360-ExploR&D, to offer its services to Lilly’s biotech partners, according to a Thursday release. Agenus has revealed its planning to make layoffs as a part of its wider plan to reduce costs, the company said Thursday. The company said Monday it is restructuring its manufacturing sites to become a biologics CDMO. Nature’s Toolbox has secured $15 million from JP Morgan to fund the development of its manufacturing platforms to make biomaterials, the company said Wednesday. Altasciences is the manufacturer for several of Metsera’s weight loss candidates, the company said on Nov. 28. The CDMO has produced the biotech’s Phase 1/2 GLP-1 asset, dubbed MET-097i. It is also making two other candidates for Metsera: a long-acting amylin analog (MET-233i) and an oral GLP-1 drug (MET-002). Rallybio is moving its C5 inhibitor into a confirmatory study next year following manufacturing improvements, the company said Monday. After data analysis and changes to the production of the drug, named RLYB116, the study will be completed in the second quarter of 2025. GE Healthcare is gaining full ownership of the radiopharma manufacturer Nihon Medi-Physics by buying Sumitomo Chemical’s 50% stake in the company, according to a Sunday release. The transaction is expected to close early next year. Telix IsoTherapeutics has renewed its supply agreement with clinical-stage radiopharma Plus Therapeutics, according to a Tuesday release. Telix will continue to supply rhenium-186 isotopes for Plus’ lead candidate, obisbemeda, which is being studied for CNS tumours. Eckert & Ziegler is supplying the radiopharma company Ariceum Therapeutics with actinium-225 and lutetium-177 for its lead asset, satoreotide, under study for hard-to-treat cancers. KBI Biopharma will manufacture Alanis Therapeutics’ preclinical antibody that is being investigated for myelodysplastic syndromes and acute myeloid leukemia, according to a Tuesday release. CELLforCURE by SEQENS is partnering with clinical-stage biotech Smart Immune, according to a Tuesday release. The CDMO will make the biotech’s Phase 1/2 asset, SMART101, for acute leukemia or primary immunodeficiencies. NorthX Biologics will make Amarna Therapeutics’ clinical-stage gene therapy called Nimvec AM510, which is being investigated for type 1 diabetes, according to a Tuesday announcement. API manufacturer Antheia will receive $12 million from the US Department of Health and Human Services over two years to manufacture an unnamed pharma ingredient domestically, according to a Tuesday release. Lonza’s ADC company Synaffix has entered a licensing agreement with DNA sequencer Illumina, the companies said Wednesday. Synaffix’s so-called “metal-free clock chemistry” tech will be used by Illumina for its DNA sequencing products. Chemical manufacturer LG Chem is collaborating with microbiology biotech Acies Bio on sustainable commercial production of chemicals in high demand, the companies said Thursday. The UK Health Security Agency has signed a contract with CSL Seqirus to manufacture five million doses of human H5 influenza vaccines for the UK, the agency said Tuesday. The Brazilian research organization Oswaldo Cruz Foundation signed a memorandum of understanding with Belgian companies Quantoom Biosciences and Univercells to boost production of RNA therapeutics in Belgium, according to a Friday release. Gavi, the Vaccine Alliance, is investing $23.4 million to help vaccinate children in Sudan, in partnership with Save the Children and UNICEF, according to a Monday release. The funding will in part be used to improve vaccine storage and cold supply chains. With additional reporting by Katherine Lewin