RLYB-212

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 reported financial results for the third quarter ended September 30, 2025, and provided an update on recent company developments. “In the third quarter, we continued to execute with discipline and focus, advancing our lead program, RLYB116, and achieving a key clinical milestone with the completion of dosing in Cohort 1 in our confirmatory PK/PD Phase 1 study,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. “In parallel, we strengthened our balance sheet with non-dilutive capital received from the sale of our interest in REV102. With the second cohort of the RLYB116 study progressing, we remain on track to report data in the fourth quarter of 2025 and are increasingly confident in RLYB116's potential to be an effective therapeutic. Our internal and external market assessments further validate the importance of advancing this program, as we plan for the next stage in development and in RLYB116’s potential to address serious unmet needs in complement-mediated diseases.” Recent Business Highlights and Upcoming Milestones: Corporate Updates In the third quarter of 2025, Rallybio generated a total of $20 million pursuant to its agreement with Recursion Pharmaceuticals for the sale of its interest in REV102, an investigational ENPP1 inhibitor in development for the treatment of hypophosphatasia (HPP). The total included $7.5 million from an upfront payment and $12.5 million related to the initiation of additional preclinical studies. RLYB116 Program In September 2025, Rallybio completed dosing of Cohort 1 in the RLYB116 Phase 1 confirmatory pharmacokinetic/pharmacodynamic (PK/PD) clinical trial, which is planned for development in two hematologic conditions with significant unmet need: immune platelet transfusion refractoriness (PTR) and refractory antiphospholipid syndrome (APS).The Phase 1 study is designed to demonstrate complete and sustained complement inhibition as well as favorable tolerability. Data from Cohort 1 support the advancement of RLYB116 as a differentiated therapeutic and enables progression to Cohort 2. Rallybio is on track to report data from the study in the fourth quarter of 2025. RLYB332 Program Rallybio continues to evaluate plans for future development of RLYB332, a long-acting, monoclonal anti-matriptase-2 antibody with the potential to be a best-in-class treatment for diseases of iron overload. Preclinical data have demonstrated superior impact on PD parameters, including serum iron, unsaturated iron binding capacity, and transferrin saturation, relative to comparator molecules. Third Quarter 2025 Financial Results Revenue: Revenue was $0.2 million for the third quarter of 2025, compared to $0.3 million for the same period in 2024. The decrease in revenue for the third quarter of 2025 was related to the collaboration agreement with Johnson & Johnson in the third quarter of 2024 and the recognition of revenue related to the collaboration's performance obligations. Research & Development (R&D) Expenses: R&D expenses were $4.1 million for the third quarter of 2025, compared to $8.2 million for the same period in 2024. The decrease in R&D expenses were primarily due to a decrease in development costs related to RLYB212 and other program candidates, in addition to a decrease in payroll and personnel-related expenses, largely related to the Company's workforce reduction announced in May 2025; offset by an increase in development costs related to RLYB116. General & Administrative (G&A) Expenses: G&A expenses were $3.0 million for the third quarter of 2025, compared to $4.1 million for the same period in 2024. The decrease in G&A expenses were primarily due to a decrease in payroll and personnel-related expenses, largely related to lower headcount as compared to the same period in 2024. Net Loss and Net Loss Per Common Share: Rallybio reported a net income of $16.0 million, or $0.36 per common share, for the third quarter of 2025 compared to a net loss of $11.5 million, or $0.26 per common share, for the same period in 2024. Cash Position: As of September 30, 2025, cash, cash equivalents, and marketable securities were $59.3 million. Rallybio expects that its cash, cash equivalents and marketable securities will be sufficient to support operations through 2027. 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 pipeline of promising product candidates aimed at addressing diseases with unmet medical need in areas of complement dysregulation and hematology. The Company’s lead program, RLYB116, is a differentiated C5 inhibitor with the potential to treat diseases of complement dysregulation, with an initial focus on immune platelet transfusion refractoriness and refractory antiphospholipid syndrome. Rallybio’s pipeline also includes RLYB332, a preclinical long-acting matriptase-2 antibody for the treatment of diseases of iron overload. Rallybio is headquartered in New Haven, Connecticut. For more information, please visit www.rallybio.com and follow us on LinkedIn. 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 of Cohort 2 in the RLYB116 confirmatory PK/PD study and the periods in which data from the trial are expected to be available, whether the PK/PD confirmatory study will demonstrate improved tolerability and complete and sustained inhibition of terminal complement, whether RLYB116 will be effective in treating a broad range of complement-mediated diseases, the potential commercial opportunity for RLYB116, the potential for future development of RLYB332, the potential for RLYB332 to be a best-in-class treatment for diseases of iron overload, and the Company's cash runway. 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 RLYB116 PK/PD confirmatory study, 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 June 30, 2025, 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. Financial Tables RALLYBIO CORPORATION SELECTED CONDENSED CONSOLIDATED FINANCIAL INFORMATION Condensed Consolidated Statements of Operations and Comprehensive Income (Loss) (Unaudited) FOR THE THREE MONTHS ENDED SEPTEMBER 30, FOR THE NINE MONTHS ENDED SEPTEMBER 30, (in thousands, except share and per share amounts) 2025 2024 2025 2024 Revenue: Collaboration and license revenue $ 212 $ 299 $ 636 $ 598 Total revenue 212 299 636 598 Operating expenses: Research and development 4,143 8,240 15,942 34,122 General and administrative 2,994 4,125 11,346 15,364 Total operating expenses 7,137 12,365 27,288 49,486 Loss from operations (6,925 ) (12,066 ) (26,652 ) (48,888 ) Other income: Interest income 462 986 1,629 3,405 Gain on sale of joint venture and other income 22,479 251 22,771 561 Total other income, net 22,941 1,237 24,400 3,966 Gain (loss) before equity in losses of joint venture 16,016 (10,829 ) (2,252 ) (44,922 ) Loss on investment in joint venture — 637 874 1,809 Net income (loss) $ 16,016 $ (11,466 ) $ (3,126 ) $ (46,731 ) Net income (loss) per common share, basic and diluted $ 0.36 $ (0.26 ) $ (0.07 ) $ (1.08 ) Weighted-average common shares outstanding, basic and diluted 45,058,591 44,593,221 44,892,485 43,170,177 Other comprehensive gain (loss): Net unrealized gain (loss) on marketable securities 7 240 (44 ) 154 Other comprehensive gain (loss) 7 240 (44 ) 154 Comprehensive gain (loss) $ 16,023 $ (11,226 ) $ (3,170 ) $ (46,577 ) Condensed Consolidated Balance Sheets (Unaudited) (in thousands) SEPTEMBER 30, 2025 DECEMBER 31, 2024 Cash, cash equivalents and marketable securities $ 59,319 $ 65,511 Total assets 67,661 68,108 Total liabilities 4,627 6,454 Total stockholders' equity 63,034 61,654

iStock, marchmeena29 Azafaros will use the Series B haul to push lead asset nizubaglustat into late-stage studies for Niemann-Pick disease Type C and GM1/GM2 gangliosidoses later this year. Azafaros has brought home €132 million ($148 million) in series B capital to advance a pipeline of therapies against rare lysosomal storage disorders. The money will help the Dutch biotech run two Phase III programs for the investigational small molecule drug nizubaglustat, which is being tested for Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses. The company expects to launch both late-stage trials later this year, according to a Tuesday release . Both of Azafaros’ disease targets are rare neurometabolic conditions characterized by enzyme deficiencies, leading to the abnormal accumulation of toxic substances. NPC , for instance, involves the build-up of lipids in various tissues throughout the body, including the brain and liver. Symptoms of NPC vary from patient to patient, with some experiencing difficulties swallowing and walking. In some cases, NPC can be fatal within months of birth. GM1 and GM2 gangliosidoses involve the build-up of specific types of glycolipids—fatty molecules with a sugar moiety attached. These conditions affect the peripheral and central nervous system and, when left unchecked, can turn fatal. Common symptoms include progressive neurodegeneration, cognitive decline and impaired motor function. Designed to be taken orally, nizubaglustat has a “dual mode of action” and “the potential to offer disease-modifying” effects, according to Azafaros’ website , though the biotech did not reveal what the drug’s specific target is. Aside from funding the late-stage development of nizubaglustat, Azafaros will also use the funds from Tuesday’s series B to expand its pipeline into other indications. The financing round was led by Jeito Capital and Forbion Growth. Azafaros’ series B round is welcome news to the broader rare disease space, which in recent weeks has been hit with financial troubles and clinical setbacks. In April, for instance, Rallybio shelved its monoclonal antibody RLYB212, which was being developed for the rare bleeding disorder fetal and neonatal alloimmune thrombocytopenia, after a disappointing Phase II showing. Last week, Rallybio cut off 40% of its workforce. Meanwhile, in March, Sarepta Therapeutics took a 22% stock hit after a patient taking its Duchenne muscular dystrophy gene therapy Elevidys died. Not all is bleak, however. In mid-April, California’s Glycomine closed a $115 million Series C financing round, which will help bankroll Phase IIb assessments for its lead asset GLM101 in phosphomannomutase-2 congenital disorder of glycosylation. A couple of weeks later, Novartis acquired Regulus Therapeutics and its investigational oligonucleotide farabursen, being tested for autosomal dominant polycystic kidney disease, for a potential $1.7 billion.