Proteinuria

Company is advancing portfolio of novel therapies that aim to redefine the standard of care for patients with autoimmune diseases Lead candidate JADE-001 on track to enter the clinic in second half of 2025, with initial data expected in first half of 2026 The combined company will operate as “Jade Biosciences, Inc.” and will trade on Nasdaq under the ticker symbol “JBIO” April 28, 2025 -- Jade Biosciences, Inc. (“Jade” or the “Company”), a biotechnology company focused on developing best-in-class therapies for autoimmune diseases, today announced the completion of its previously announced merger with Aerovate Therapeutics, Inc. (“Aerovate”). The combined company will operate as Jade Biosciences, Inc., with its shares expected to begin trading on the Nasdaq Capital Market on April 29, 2025, under the ticker symbol “JBIO.” Immediately prior to the merger, Jade successfully completed a previously announced, oversubscribed private investment round that resulted in total gross proceeds of approximately $300 million from a syndicate of healthcare investors led by Fairmount, Venrock Healthcare Capital Partners, and a large investment firm, with participation from Deep Track Capital, Braidwell LP, Driehaus Capital Management, Frazier Life Sciences, RA Capital Management, Great Point Partners, Soleus Capital, Avidity Partners, Blackstone Multi-Asset Investing, Logos Capital, Deerfield Management, OrbiMed, and Samsara BioCapital, among other leading investment management firms. The proceeds include the conversion of $95 million in previously issued convertible notes, plus interest and premiums thereon. Pursuant to the terms of the previously disclosed merger agreement, each outstanding share of Jade common stock was converted into 0.6311 shares of common stock of the combined company, as adjusted for the reverse stock split of Aerovate common stock at a ratio of 1-for-35 shares, effected immediately prior to the merger. In the reverse stock split, every 35 shares of Aerovate common stock outstanding were combined and reclassified into 1 share of Aerovate common stock. The new CUSIP number for the combined company following the reverse stock split and merger is 008064206. In connection with the closing of the merger, Aerovate will distribute a previously announced special cash dividend of approximately $2.40 per share of common stock to pre-merger Aerovate stockholders of record as of April 25, 2025. Following completion of the reverse stock split, private placement, and merger, there are approximately 60.6 million shares of the combined company’s common stock outstanding on a fully-diluted basis, or approximately 52.6 million shares excluding shares underlying equity awards. “We’ve made remarkable progress since our founding, assembling a talented team with deep expertise in drug development for autoimmune diseases,” said Tom Frohlich, Chief Executive Officer of Jade. “With a strong financial foundation, we aim to advance best-in-class therapeutics for autoimmune diseases, offering more patient-friendly dosing regimens with the potential of better outcomes. Our lead program, JADE-001, is just one example that reflects our commitment to providing patients with better solutions.” JADE-001 is an investigational anti-A PRoliferation-Inducing Ligand (APRIL) monoclonal antibody for the treatment of Immunoglobulin A (IgA) nephropathy (IgAN), an autoimmune condition in which the kidney is damaged by pathogenic IgA containing immune complexes, leading to proteinuria and kidney function decline, and potentially progressing to end-stage kidney disease requiring dialysis or transplantation. Designed to block the APRIL protein, JADE-001 targets an underlying cause of IgAN, offering the potential to reduce pathogenic IgA levels, decrease proteinuria, and preserve kidney function over the long term. Incorporating half-life extension technology, JADE-001 is engineered to enable dosing at intervals of at least eight weeks, with the potential to significantly alleviate the burden of treatment, particularly considering that IgAN is often diagnosed in young adulthood and requires lifelong care. JADE-001 is anticipated to enter the clinic in the second half of 2025, with initial data expected in the first half of 2026. In addition, Jade has initiated preclinical development of JADE-002 and JADE-003, two undisclosed antibody programs. IgAN is a chronic autoimmune disease in which an abnormal form of IgA (a type of antibody produced by the body) is deposited in the kidney and causes kidney damage. Over time, kidney function can decline and may lead to kidney failure requiring dialysis or kidney transplant. IgAN is often diagnosed in young adulthood, and there are currently no approved treatments for IgAN that reduce the abnormal form of IgA and stabilize kidney function. APRIL, or “A Proliferation-Inducing Ligand,” is a protein in the body that is necessary for immune cells to make IgA antibodies. Too much APRIL can lead to the overproduction of IgA, including an abnormal IgA variant that can result in immune complex formation which can be harmful to the kidney. Jade Biosciences is focused on developing best-in-class therapies to address critical unmet needs in autoimmune diseases. Its lead asset, JADE-001, targets the cytokine APRIL for immunoglobulin A nephropathy, with initiation of a first-in-human clinical trial expected in the second half of 2025. Jade’s pipeline also includes two undisclosed antibody discovery programs, JADE-002 and JADE-003, currently in preclinical development. Jade was launched based on assets licensed from Paragon Therapeutics, an antibody discovery engine founded by Fairmount. The content above comes from the network. if any infringement, please contact us to modify.

The FDA is yet to approve a therapy specifically for FSGS. \n Orphan drug specialist Amicus Therapeutics is paying Dimerix $30 million cash for U.S. rights to the phase 3 prospect DMX-200, establishing it as a challenger to Travere Therapeutics in a rare kidney disease. New Jersey-based Amicus’ hunt for de-risked assets that could address a significant unmet medical need led it to Australia. Melbourne-based Dimerix began a phase 3 study of the CCR2 inhibitor DMX-200 in focal segmental glomerulosclerosis (FSGS) kidney disease in 2022. The Australian biotech recently aligned with the FDA on the use of proteinuria as an appropriate primary endpoint for traditional approval. Dimerix reported interim results on the effect of DMX-200 on proteinuria last year. With enrollment well underway and another interim analysis coming up, data that could support filings for FDA approval is in sight.As the program advances, Amicus could pay Dimerix up to $75 million in development and regulatory milestones. A further $35 million is tied to the first sale of the drug. Beyond that, Amicus has committed up to $410 million in sales milestones and $40 million tied to potential future indications.   The FDA is yet to approve a therapy specifically for FSGS. That could change soon. Travere filed for FDA approval of Filspari in the indication this March. Amicus’ willingness to bet on DMX-200 despite the potential of a rival that is near approval reflects a belief that there is room for multiple approaches in FSGS.Jeffrey Castelli, chief development officer at Amicus, said on an earnings call Thursday that DMX-200 “is specifically targeting the inflammatory signaling in the damaged kidney.” Other molecules, in contrast, are focused on hemodynamics. Some patients will respond better to one drug than the other, Castelli said, and physicians will try to figure out which is the right approach for each individual.  “With DMX-200, there are ways you can look at how much the inflammatory component is driving things. Those are going to be the patients we\'re going to see respond best in the trials, and those are going to be the patients that I think doctors will want to focus on,” Castelli said. ChemoCentryx, now part of Amgen, was developing a CCR2 inhibitor with Vifor Fresenius Medical Care Renal Pharma until a phase 2 flop scuttled the FSGS program in 2020. Castelli discussed the differences between DMX-200 and earlier candidates, contrasting attempts to directly inhibit MCP-1 binding to the downstream approach of Dimerix’s molecule. “What happened with traditional binding inhibitors is MCP-1 couldn\'t dock. It actually led to a rebound effect where MCP-1 levels dramatically increase, which then would compete with the inhibitor and kind of lead to a muting effect,” Castelli said. “The beauty of the downstream blocking is that MCP-1 can still dock. That leads to MCP-1 degradation.”Amicus believes the mechanism of DMX-200 will “really maximize the ability to shut down the monocyte inflammation,” Castelli said. The downstream effects will also allow “monocytes to continue their job elsewhere ... so they can still fight infections as they should outside of the kidney.”

Amicus licenses exclusive U.S. rights to commercialize DMX-200 for the treatment of Focal Segmental Glomerulosclerosis (FSGS) DMX-200 is in a pivotal Phase 3 trial for FSGS, a rare and fatal kidney disease with no FDA-approved therapies Dimerix successfully completed Type C meeting with the FDA in March 2025, aligning on proteinuria as the primary endpoint for approval Dimerix to receive US$30 million (~AU$48 million) upfront payment, up to US$560 million (~AU$892 million) for success-based milestone payments, in addition to tiered royalties on DMX-200 net U.S. sales MELBOURNE, Australia and PRINCETON, NJ, USA I April 30, 2025 I Dimerix Limited (ASX: DXB, “Dimerix”) and Amicus Therapeutics (Nasdaq: FOLD, “Amicus”) today announced that the two companies have entered into an exclusive license agreement for the commercialization of Dimerix’ Phase 3 drug candidate DMX-200 for all indications, including FSGS, in the United States (U.S.). Dimerix retains all rights to commercialize DMX-200 in all territories other than those already exclusively licensed. DMX-200 is a small molecule inhibitor of the chemokine receptor 2 (CCR2) under development in a pivotal Phase 3 study, ACTION3, for the treatment of Focal Segmental Glomerulosclerosis (FSGS) kidney disease. In early 2024, Dimerix reported positive interim results from the ACTION3 trial in FSGS showing DMX-200 was performing better than placebo in reducing proteinuria with no safety concerns to date. Full enrollment of ACTION3 is expected by year-end 2025. An additional blinded interim analysis is planned once the revised primary and secondary endpoints have been pre-specified in the protocol and agreed with the FDA. In a March 2025 Type C meeting, Dimerix successfully aligned with the FDA on proteinuria as an appropriate primary endpoint for traditional marketing approval for DMX-200. “Amicus is thrilled to enter into this collaboration with Dimerix to bring DMX-200 to patients in the U.S., and we are incredibly impressed by their achievements to date. We look forward to leveraging our regulatory, commercial, medical, and advocacy capabilities to bring this potentially transformative treatment to people living with FSGS in the U.S.,” said Bradley Campbell, President and Chief Executive Officer, Amicus Therapeutics. “This licensing agreement represents a major step forward in our strategy to strengthen our portfolio and fully aligns with our mission to develop and deliver transformative medicines for people living with rare diseases.” “We are delighted to partner with Amicus in the United States. The Amicus team has a remarkable history of successfully delivering rare disease medicines to those in need. Their expertise and resources will be crucial to help achieve our mutual objective of commercializing this innovative treatment,” said Dr. Nina Webster, CEO and Managing Director of Dimerix. “I’m grateful to the dedicated Dimerix team, trial participants, and investigators for their continued commitment to developing a new therapy for patients with FSGS who currently have a poor prognosis and very limited treatment options.” Dimerix will continue to fund and execute the ACTION3 study, and Amicus will be responsible for submission and maintenance of the regulatory dossier in the United States, as well as all costs of commercialization activities. Additionally, Amicus will have the exclusive rights to develop DMX-200 in other future indications in the United States. Amicus and Dimerix will form a Joint Steering Committee to align the development and commercialization of DMX-200 in FSGS in U.S. The agreement otherwise contains terms common for an arrangement of this kind. In exchange for these rights, Dimerix will receive a US$30 million (~AU$48 million) upfront payment. The next potential milestone payment is based on positive data from the Phase 3 trial in FSGS. In total, Dimerix is eligible to receive potential success-based development and regulatory milestone payments of up to US$75 million (~AU$119 million) until FDA approval of DMX-200 in FSGS, US$35 million (~AU$56 million) on first sale, commercial sales milestone payments of up to US$410 million (AU$653 million), and tiered royalties from the low-teens to low-twenties percentages of DMX-200 net sales in the U.S. In addition, Dimerix is eligible to receive up to US$40 million (~AU$64 million) in milestone payments for potential future indications. The upfront payment from Amicus will be funded with cash on hand. All contracted financial terms are denominated in U.S. dollars. Evercore Partners International LLP is acting as exclusive financial advisor to Dimerix, and Cooleys LLP is serving as Dimerix legal advisor. Wilson Sonsini Goodrich & Rosati is serving as Amicus legal advisor. Authorized for lodgment with ASX by the Board of Dimerix. About ACTION3 Phase 3 Study The Phase 3 study, which is titled “Angiotensin II Type 1 Receptor (AT1R) & Chemokine Receptor 2 (CCR2) Targets for Inflammatory Nephrosis”, or ACTION3 for short, is a pivotal (Phase 3), multi-center, randomized, double-blind, placebo-controlled study of the efficacy and safety of DMX-200 in patients with FSGS who are receiving a stable dose of an angiotensin II receptor blocker (ARB). Once the ARB dose is stable, patients will be randomized to receive either DMX-200 (120 mg capsule twice daily) or placebo. The single Phase 3 trial in FSGS patients has two interim analysis points built in that are designed to capture evidence of proteinuria and kidney function (eGFR slope) during the trial, aimed at generating sufficient evidence to support marketing approval. Further information about the study can be found on ClinicalTrials.gov (Study Identifier: NCT05183646) or Australian New Zealand Clinical Trials Registry (ANZCTR) (Study Identifier ACTRN12622000066785). About DMX-200 DMX-200 is a chemokine receptor (CCR2) antagonist administered to patients already receiving an angiotensin II type I receptor (AT1R) blocker, the standard of care treatment for hypertension and kidney disease. DMX-200 is protected by granted patents in various territories until 2032, with patent applications submitted globally that may extend patent protection to 2042, in addition to Orphan Drug Designation granted by the FDA in the United States. About FSGS FSGS is a rare, serious kidney disorder characterized by progressive scarring (sclerosis) in parts of the glomeruli—the kidney’s filtering units. This scarring leads to proteinuria, progressive loss of kidney function, and often end-stage renal disease. FSGS is increasingly understood to have an inflammatory component, with monocyte and macrophage activation contributing to glomerular injury. In the United States, more than 40,000 people are estimated to be living with FSGS, including both adults and children. There are no therapies specifically approved for FSGS in the U.S., and management relies on non-specific immunosuppressive and supportive therapies. In patients with progressive or treatment-resistant FSGS, the average time from diagnosis to end-stage kidney disease can be as short as five years. Even among those who undergo kidney transplantation, disease recurrence occurs in up to 60% of cases, underscoring the urgent need for new, disease-modifying treatments. About Dimerix Limited Dimerix (ASX: DXB) is a clinical-stage biopharmaceutical company working to improve the lives of patients with inflammatory diseases, including kidney diseases. Dimerix is currently focused on developing its proprietary Phase 3 product candidate DMX-200, for Focal Segmental Glomerulosclerosis (FSGS) kidney disease, and is also developing DMX-700 for respiratory disease. DMX-200 and DMX-700 were both identified using Dimerix’ proprietary assay, Receptor Heteromer Investigation Technology (Receptor-HIT), which is a scalable and globally applicable technology platform enabling the understanding of receptor interactions to rapidly screen and identify new drug opportunities. For more information, please visit the company’s website at www.dimerix.com and follow on X and LinkedIn . About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com , and follow on X and LinkedIn . SOURCE: Amicus Therapeutics