Roche Announces Positive Interim Results for Sefaxersen in IgA Nephropathy; Lilly’s Olumiant Receives FDA Approval for Pediatric Patients With Severe Alopecia Areata; AbbVie’s JUVMO Receives U.S. FDA Approval for Parkinson’s Disease; Biologics License Application for Ifinatamab Deruxtecan Withdrawn in Extensive-Stage Small Cell Lung Cancer; Mirum Pharmaceuticals Reports Primary Endpoint Success in Phase 3 AZURE-1 Study of Brelovitug in Chronic Hepatitis Delta Virus

Roche Reports Positive Interim Data Showing Sefaxersen Significantly Reduces Proteinuria in IgA Nephropathy

Roche announced positive prespecified interim results from the ongoing Phase III IMAgINATION study evaluating investigational sefaxersen in adults with primary IgA nephropathy (IgAN). The trial achieved its primary endpoint, demonstrating statistically significant and clinically meaningful reductions in proteinuria with sefaxersen compared with placebo at week 37, as assessed by the 24-hour urine protein-to-creatinine ratio (UPCR). Proteinuria is an important marker of kidney damage, and reductions in UPCR are strongly associated with the preservation of kidney function over the long term. Sefaxersen is being developed as a once-monthly subcutaneous injection intended to support self-administration by people living with IgAN. Its safety and tolerability profile remained consistent with previously reported findings, with no new safety signals identified.

Levi Garraway, MD, PhD, Roche’s Chief Medical Officer and Head of Global Product Development, said the positive interim Phase III findings demonstrate the potential of sefaxersen to affect an important surrogate marker of kidney function in people with IgAN. He noted that the treatment could potentially provide a new option for slowing disease progression and reducing the long-term burden of dialysis or kidney transplantation.

Bonnie Schneider, Director and Co-Founder of the IgA Nephropathy Foundation, highlighted the uncertainty faced by patients and families dealing with the potential progression of IgAN to kidney failure, dialysis, or transplantation. She said the positive IMAgINATION results offer hope that emerging therapies may help preserve kidney function and contribute to changes in the treatment landscape.

The IMAgINATION study will continue under blinded conditions to assess changes in kidney function over two years, with estimated glomerular filtration rate (eGFR) at week 105 serving as the measure. Roche plans to present the interim findings at an upcoming medical congress and submit the data to health authorities, with the aim of making sefaxersen available to patients as quickly as possible.

IgA nephropathy, also known as Berger’s disease, is a chronic, progressive autoimmune disorder that can lead to end-stage kidney disease in up to 50% of patients within 20 years of diagnosis. The disease is commonly diagnosed before the age of 40 and affects at least 25 adults per million people worldwide each year. IgAN is the most common form of primary glomerulonephritis, an inflammation of the kidney’s glomeruli, and represents a significant cause of chronic kidney disease and kidney failure.

Lilly’s Olumiant Gains FDA Approval for Patients 12 and Older With Severe Alopecia Areata

Eli Lilly and Company announced that the U.S. Food and Drug Administration (FDA) has approved Olumiant® (baricitinib), a once-daily oral therapy, for the treatment of severe alopecia areata in pediatric patients aged 12 years and older. This decision expands Olumiant’s U.S. prescribing label and builds on its established indications for adults with moderately to severely active rheumatoid arthritis and severe AA.

Alopecia areata is a chronic, immune-mediated condition characterized by unpredictable hair loss. The disease affects up to 2% of individuals at some point during their lifetime, with nearly 40% of cases developing before the age of 20.²˒³ AA that begins during childhood or adolescence may be more severe and can result in extensive hair loss.³

“Today’s approval represents an important milestone for adolescents living with severe alopecia areata and their families, who have historically faced limited treatment options,” said Adrienne Brown, executive vice president and president of Lilly Immunology. “The visible and often extensive hair loss associated with severe alopecia areata can significantly affect patients, particularly adolescents during an important stage of their lives. Extending Olumiant’s availability to patients aged 12 and older provides an important treatment option and underscores Lilly’s commitment to advancing care for people affected by immune-mediated diseases.”

The FDA’s approval was supported by 36-week results from the adolescent cohort of the BRAVE-AA-PEDS trial, involving patients aged 12 to under 18 years. The study represents the first and largest Phase 3 trial specifically designed to assess a treatment in pediatric patients with severe AA.⁴ At study entry, patients had a median Severity of Alopecia Tool (SALT) score of 100, reflecting complete scalp hair loss, and had been living with AA for an average of 6.4 years.¹

After 36 weeks of treatment with once-daily Olumiant:

• At least 80% scalp hair coverage, defined as a SALT score of ≤20, was achieved by 42% of patients receiving Olumiant 4 mg and 27% receiving Olumiant 2 mg, compared with 5% of patients receiving placebo.
• At least 90% scalp hair coverage, defined as a SALT score of ≤10, was achieved by 37% of patients treated with Olumiant 4 mg and 21% treated with Olumiant 2 mg, compared with 2% of those receiving placebo.
• Eyebrow and eyelash hair coverage also improved among patients treated with Olumiant 4 mg who had substantial eyebrow and eyelash hair loss at baseline.

The safety profile observed in pediatric patients aged 12 years and older with severe AA was consistent with the established safety profile of Olumiant in adults with severe AA.¹ The FDA-approved prescribing information includes a boxed warning regarding the risks of serious infections, mortality, malignancies, major adverse cardiovascular events (MACE), and thrombosis.

Olumiant is available in 4 mg, 2 mg, and 1 mg tablets.¹ The recommended starting dose is 2 mg once daily, which may be increased to 4 mg once daily when the treatment response is inadequate. For patients with nearly complete or complete scalp hair loss, with or without substantial eyebrow or eyelash hair loss, treatment with 4 mg once daily may be considered. Once an adequate response is achieved with the 4 mg dose, the dosage should be reduced to 2 mg once daily.

AbbVie Secures FDA Approval for JUVMO™ (tavapadon) in Parkinson’s Disease

The US Food and Drug Administration (FDA) has approved AbbVie’s next-generation dopamine-based therapy for Parkinson’s disease, tavapadon, potentially supporting the company’s $8.7 billion acquisition of its original developer, Cerevel Therapeutics.

According to an update on the FDA’s website, tavapadon, which AbbVie will commercialize under the brand name Juvmo, was approved on 25 September to improve motor function in adults with Parkinson’s disease. The therapy is a partial agonist of the dopamine D1 and D5 receptors.

Unlike conventional dopamine-based treatments such as levodopa, which interact with multiple dopamine receptor types, Cerevel developed Juvmo to selectively target D1 receptors, which play a more direct role in motor function regulation. This selective mechanism could potentially limit non-motor side effects associated with activity at D2 and D3 receptors. Combined with its extended half-life, Juvmo is designed to provide a more targeted and potentially optimized approach to dopamine-based treatment for Parkinson’s disease.

Although AbbVie and the FDA have not publicly disclosed the specific evidence underlying Juvmo’s approval for Parkinson’s disease, the decision likely reflects positive findings from the Phase III TEMPO programme (NCT04201093; NCT04223193; NCT04542499; NCT04760769). The programme evaluated tavapadon using both flexible- and fixed-dose regimens in patients across different stages and clinical presentations of Parkinson’s disease. According to research published in Diseases, Juvmo significantly improved motor symptoms among patients in the TEMPO-1 and TEMPO-2 trials. In TEMPO-3, the therapy also significantly reduced motor fluctuations when used in combination with levodopa compared with levodopa alone.

Ifinatamab Deruxtecan Application Withdrawn for Certain Patients with Previously Treated Extensive-Stage SCLC

Daiichi Sankyo and Merck have voluntarily withdrawn their Biologics License Application (BLA) seeking accelerated approval in the U.S. for ifinatamab deruxtecan (I-DXd) in adults with extensive-stage small cell lung cancer (ES-SCLC) whose disease has progressed on or after platinum-based chemotherapy.

The withdrawal follows discussions with the U.S. Food and Drug Administration (FDA), which determined that the data supporting the application, including findings from the Phase 2 IDeate-Lung01 trial, did not meet the requirements necessary to support accelerated approval for the proposed indication.

Patient enrollment remains ongoing in the Phase 3 IDeate-Lung02 trial, which is evaluating the efficacy and safety of ifinatamab deruxtecan against physician’s choice of chemotherapy, including amrubicin, lurbinectedin, or topotecan, in patients with relapsed ES-SCLC following disease progression after one prior line of platinum-based chemotherapy.

Daiichi Sankyo noted that enrollment in IDeate-Lung02 is nearing completion and stated that the company plans to assess the potential for a future regulatory submission to the FDA and other global health authorities based on the trial results.

Merck said that although the current dataset does not support approval at this stage, the companies will continue evaluating the potential role of ifinatamab deruxtecan in ES-SCLC and other difficult-to-treat cancers. The companies also acknowledged the contributions of patients, their families, and investigators involved in the clinical development program.

Beyond IDeate-Lung02, ifinatamab deruxtecan is being evaluated in two additional Phase 3 studies in advanced or metastatic cancers: IDeate-Prostate01 in castration-resistant prostate cancer (CRPC) and IDeate-Esophageal01 in esophageal squamous cell carcinoma (ESCC).

Mirum’s Brelovitug Meets Primary Endpoint in Phase 3 AZURE-1 Trial for Chronic Hepatitis Delta Virus

Mirum Pharmaceuticals, Inc. announced that the Phase 3 portion of the AZURE-1 study met its primary endpoint in evaluating brelovitug, an investigational fully human monoclonal antibody targeting hepatitis B surface antigen (HBsAg), for the treatment of chronic hepatitis delta virus (HDV) infection. Additionally, 48-week results from the Phase 2b portion of AZURE-1 showed progressively deeper viral suppression, with a higher proportion of patients achieving HDV RNA below the lower limit of quantification (LLOQ; <10 IU/mL) or target not detected (TND), along with increased rates of alanine aminotransferase (ALT) normalization. AZURE-1 is one of two pivotal Phase 3 trials expected to support Mirum’s U.S. registration package for brelovitug.

According to Nancy Shulman, M.D., Executive Vice President of Clinical Development at Mirum, the findings support the potential of brelovitug as a well-tolerated and convenient single-agent treatment capable of achieving both substantial viral suppression and ALT normalization, with responses continuing to deepen through 48 weeks. The results were observed across a broad patient population, including individuals with significant liver inflammation, cirrhosis, and clinically significant portal hypertension. Mirum expects to report topline results from the AZURE-4 study later in 2026 and plans to advance toward a Biologics License Application (BLA) submission in the first half of 2027.

Norah Terrault, M.D., MPH, Professor of Medicine and Chief of the Division of GI and Liver at the Keck School of Medicine of USC and an AZURE-1 investigator, noted that effective management of chronic HDV requires controlling both viral replication and liver inflammation to reduce the risk of progression to cirrhosis, liver cancer, and liver failure. As ALT is a marker of liver injury, the combination of virologic response and ALT normalization observed in the study may be relevant for patients requiring long-term treatment, particularly those with aggressive forms of viral hepatitis.

Chari A. Cohen, DrPH, MPH, President of the Hepatitis B Foundation, emphasized that chronic hepatitis delta is a lifelong condition and highlighted the need for treatment options that provide sustained efficacy while maintaining safety and tolerability, particularly for patients with advanced disease who may have limited therapeutic choices.

The Phase 3 portion of AZURE-1 enrolled 153 treatment-naive patients who were randomized in a 2:2:1 ratio to receive brelovitug 300 mg once weekly (QW) through self-administered subcutaneous (SC) injection, brelovitug 900 mg once every four weeks (Q4W) through SC injection, or delayed treatment beginning at Week 24. The global trial included a broad patient population, including individuals with advanced disease.

At Week 24, brelovitug achieved the study’s primary endpoint. A combined virologic response—defined as at least a 2 log10 reduction in HDV RNA from baseline or undetectable HDV RNA (<LLOQ, TND)—and ALT normalization was achieved by 56% of patients receiving 300 mg QW and 45% receiving 900 mg Q4W, compared with 0% in the delayed-treatment group. Both comparisons versus delayed treatment were statistically significant (p<0.0001).