Telitacicept

Annualized eGFR slope of -0.6 vs. -5.7 mL/min/1.73 m²/year for placebo, a 5.1 mL/min/1.73 m²/year treatment difference (p<0.001) 89% and 81% reductions in risk of confirmed ≥30% and ≥40% eGFR declines, respectively Proteinuria reductions sustained through 104 weeks following transition to every-other-week dosing; favorable safety profile BOSTON, Oct. 22, 2026 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today announced final two-year results from the Phase 3 TELIGAN trial evaluating telitacicept in adults with IgA nephropathy (IgAN), conducted in China by its collaborator, RemeGen Co., Ltd (HKEX: 9995, SHA: 688331). TELIGAN met the Stage B primary endpoint, demonstrating a statistically significant difference in annualized estimated glomerular filtration rate (eGFR) slope through 104 weeks compared with placebo. The data were presented today in an oral presentation (TH-OR111) during the Featured High-Impact Clinical Trials plenary session at the American Society of Nephrology (ASN) Kidney Week 2026 in Denver, Colorado. The 104-week findings build on the previously reported 39-week results, published in The New England Journal of Medicine in May 2026, and extend the evidence from proteinuria reduction to longer-term preservation of kidney function and reduced incidence of clinically relevant kidney disease progression events. “At 104 weeks, TELIGAN demonstrated a substantial reduction in proteinuria and important evidence of durable kidney protection,” said Jean-Paul Kress, M.D., Chairman and Chief Executive Officer of Vor Bio. “An annualized eGFR slope approaching zero, lower rates of kidney function deterioration, and sustained proteinuria reductions following the transition to every-other-week dosing together reinforce the clinical potential of telitacicept. These findings add to the growing body of evidence supporting dual BAFF/APRIL inhibition as we continue to advance our global Phase 3 programs in myasthenia gravis and Sjögren’s disease.” TELIGAN is a multicenter, randomized, double-blind, placebo-controlled Phase 3 trial that enrolled 318 adults with biopsy-confirmed IgAN receiving stable background supportive therapy. Patients were randomized 1:1 to telitacicept 240 mg or placebo. Treatment was administered subcutaneously once weekly through Week 39 (Stage A), followed by every-other-week dosing from Week 40 through Week 104 (Stage B). The Stage A primary endpoint assessed change in 24-hour urine protein-to-creatinine ratio (24h-UPCR) at Week 39; the Stage B primary endpoint assessed annualized eGFR slope through Week 104. Key Findings from the Final 104-Week Analysis: Kidney function preservation: Annualized eGFR slope through 104 weeks was -0.6 mL/min/1.73 m²/year (95% CI: -1.7 to 0.4) for telitacicept versus -5.7 mL/min/1.73 m²/year (95% CI: -7.0 to -4.5) for placebo, representing a between-group difference of 5.1 mL/min/1.73 m²/year (95% CI: 3.5 to 6.7; p<0.001). Reduced kidney disease progression: Telitacicept was associated with an 89% lower risk of confirmed ≥30% eGFR decline (HR 0.11) and an 81% lower risk of confirmed ≥40% eGFR decline (HR 0.19) relative to placebo. No patient in the telitacicept group experienced a prespecified composite kidney failure endpoint event, compared with five in the placebo group, including two who required dialysis. Durable proteinuria reduction: At Week 104, 24h-UPCR decreased 60.3% from baseline in the telitacicept group compared with a 0.9% increase in the placebo group, representing a reported placebo-adjusted reduction of 60.7%. At Week 104, 44.0% of telitacicept-treated patients achieved 24h-UPCR <0.5 g/g versus 8.2% in the placebo group. Reductions in proteinuria were maintained after the transition to every-other-week dosing. Reduction in pathogenic IgA1: At Week 104, serum galactose-deficient IgA1 (Gd-IgA1) decreased 47.79% from baseline with telitacicept, compared with a 19.06% increase with placebo, consistent with the drug’s dual BAFF/APRIL mechanism. Safety: Treatment-emergent adverse events occurred in 95.0% of telitacicept-treated patients and 89.3% of placebo-treated patients. Serious adverse events occurred in 8.2% versus 18.2%, respectively. Overall infections were reported in 67.3% of each group, and serious infections in 1.9% versus 4.4%. Injection-site reactions were more frequent with telitacicept (56.0% versus 18.9%), with most mild to moderate in severity. In June 2026, telitacicept received conditional approval in China for the reduction of proteinuria in adults with primary IgAN at risk of disease progression. RemeGen is responsible for the development and commercialization of telitacicept in China, while Vor Bio holds exclusive rights to develop and commercialize telitacicept outside Greater China. About IgA Nephropathy IgA nephropathy (IgAN) is one of the most common primary glomerular diseases worldwide and a leading cause of chronic kidney disease. It is characterized by deposition of IgA-containing immune complexes in the kidneys, resulting in inflammation, proteinuria, hematuria, and progressive loss of kidney function. Overproduction of Gd-IgA1 is a central driver of IgAN, and despite available therapies, patients remain at risk of progression to kidney failure. About Telitacicept Telitacicept is a novel recombinant fusion protein designed to treat autoimmune diseases through dual inhibition of BLyS (BAFF) and APRIL - two cytokines essential to B cell and plasma cell survival. This dual-target mechanism reduces autoreactive B cells and autoantibody production, key drivers of autoimmune pathology. Telitacicept is approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), generalized myasthenia gravis (gMG), IgA nephropathy (IgAN), and Sjögren’s disease (SjD). Vor Bio is advancing telitacicept in global Phase 3 trials in gMG and SjD to support potential regulatory approvals in the United States, Europe, and Japan. About Vor Bio Vor Bio is a clinical-stage biotechnology company transforming the treatment of autoimmune diseases. The Company is focused on rapidly advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development and potential commercialization to address serious autoantibody-driven conditions worldwide. For more information visit . Vor Bio routinely posts information that may be important to investors in the “Investors” section of its website. The Company encourages investors to consult that section of its website regularly. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “design,” “expect,” “intend,” “may,” “ongoing,” “plan,” “potential,” “should,” “update,” “will,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the therapeutic and clinical potential of telitacicept, including its potential benefits in IgAN and other autoimmune diseases, its potential to preserve kidney function and slow disease progression, and the durability of its effects; the potential of dual BAFF/APRIL inhibition; the conditional approval of telitacicept in China and any conversion to full approval; Vor Bio’s development and commercialization plans, including potential regulatory approvals in the United States, Europe, and Japan; and other statements that are not historical fact. Vor 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. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including that results from the TELIGAN trial, which RemeGen conducted solely in China, may not be predictive of results in other populations or in our ongoing or future clinical trials, and that the FDA and other regulators outside China may not accept data generated in China; that results of earlier clinical trials or analyses may not be predictive of future results, and that data presented at medical meetings may change following additional analysis or peer review; the risk of unexpected adverse events or safety findings; that conditional approvals in China may be withdrawn or not converted to full approval; our reliance on RemeGen, including for clinical data and the supply of telitacicept, and our ability to maintain our license with RemeGen; competition from other therapies, including other BAFF and/or APRIL inhibitors; that the data for our product candidates may not be sufficient for obtaining regulatory approval to commercialize products; we may not be able to execute our business plans, including meeting our planned clinical and regulatory milestones and timelines; and possible limitations of financial and other resources. The clinical trial results described in this press release are based on information reported by RemeGen; Vor Bio has not independently verified these data. These and other risks are described in greater detail under the caption “Risk Factors” included in Vor Bio’s most recent annual or quarterly report and in other reports it has filed or may the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Vor Bio expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as may be required by law. CONTACT: Media & Investor Contacts: Carl Mauch cmauch@vorbio.com

Cosette Pharmaceuticals has achieved a corporate milestone, securing an FDA approval for a legacy Daiichi Sankyo drug in its first nod under the new drug application (NDA) pathway. The approval is for a liquid formulation of Evoxac, which was originally marketed by Daiichi Sankyo in capsules, providing an alternative treatment option for managing dry mouth symptoms in patients living with Sjögren’s disease. While the drug was approved under an NDA, Cosette relied partially on existing safety and efficacy data from the legacy capsule formulation through the FDA’s 505(b)(2) pathway. This allowed the New Jersey company to bypass a full-scale clinical development program. Cosette expects the new oral solution to hit the U.S. market by the end of 2026 and offer a better option for patients who may struggle with swallowing capsules. “We have built Cosette by acquiring and strengthening trusted brands,” Cosette’s CEO Apurva Saraf said in an Oct. 20 statement. “Today, we are demonstrating that we can also create value through our own product development capabilities.” Evoxac was part of a multi-product transaction Cosette penned with Daiichi back in 2022, as the Japanese pharma shifted its focus to oncology by shedding some legacy medicines. A portfolio of Daiichi cardiovascular disease therapies—Benicar, Welchol, Azor, Tribenzor and Effient—also found their new home at Cosette. Back then, all those drugs registered annual sales of about $123 million. Sjögren’s is a systemic autoimmune condition characterized by immune-mediated attacks on moisture-producing glands, leading to debilitating dry eyes and dry mouth alongside fatigue and joint pain. Existing therapies like Evoxac are approved to manage specific symptoms of Sjögren’s, but no FDA-approved therapies are available to address the underlying autoimmune cause of the disease. The dry spell in Sjögren’s disease-modifying therapies is ending. Novartis is expecting an FDA decision for ianalumab, its investigational drug targeting the B cell-activating factor (BAFF) receptor, after a pair of phase 3 wins . Last month, Amgen announced that its dazodalibep, a fusion protein targeting the CD40 ligand, also succeeded in a phase 3 trial in patients with moderate-to-severe Sjögren’s. The company will likely need a second phase 3 win to file for an approval. Outside the U.S., Chinese authorities approved RemeGen’s telitacicept for Sjögren’s in June, making it the first regulatory approval anywhere for the indication. The BAFF/APRIL dual inhibitor proved its ability to address both systemic disease manifestations and symptoms in a China phase 3 trial. The drug is being developed by Vor Bio outside China, with a global phase 3 in Sjögren’s underway. In contrast, argenx wasn’t so lucky as its FcRn block Vyvgart Hytrulo suffered a surprise phase 3 failure in Sjögren’s earlier this month.