Novartis Secures FDA Traditional Approval for Fabhalta in Primary IgAN; GSK Reports Mixed Phase III Results for Camlipixant in Refractory Chronic Cough; Merck Secures FDA Approval for LIPFENDRA; Innovent Reports Positive Phase II Results for Soficinib in Vitiligo; Lilly Signs Deal to Acquire AtaiBeckley for Mental Health Therapies

Novartis Wins FDA Traditional Approval for Fabhalta to Treat Primary IgAN

Novartis announced that the US Food and Drug Administration (FDA) has granted traditional approval to Fabhalta (iptacopan) for slowing the progression of kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk of disease progression. The first-in-class complement inhibitor received the approval under the FDA’s priority review pathway, following its accelerated approval in August 2024 for reducing proteinuria in patients with primary IgAN.

Commenting on the approval, Dana Rizk, MD, Professor of Medicine in the Division of Nephrology at the University of Alabama at Birmingham and a member of the APPLAUSE-IgAN Steering Committee, emphasized that IgAN is a chronic immune-mediated kidney disorder that can ultimately lead to kidney failure and significantly affect patients’ quality of life. She noted that slowing the loss of kidney function is a key therapeutic objective and highlighted the approval of Fabhalta as further validation of targeting complement activation and other underlying disease mechanisms to help preserve renal function.

IgAN is among the most common autoimmune kidney diseases, with approximately 25 new cases diagnosed annually per million people worldwide. Patients with persistent proteinuria face a substantial risk of disease progression, with nearly half advancing to kidney failure within 10 to 20 years after diagnosis. Many of these individuals ultimately require dialysis or kidney transplantation, creating a considerable long-term clinical and healthcare burden.

According to Bonnie Schneider, Director and Co-Founder of the IgA Nephropathy Foundation, the approval represents an important advancement for the IgAN community. She stated that the availability of Fabhalta offers renewed optimism by providing patients and their families with a treatment capable of helping preserve kidney function and improving future disease management.

The FDA’s decision was supported by findings from the Phase III APPLAUSE-IgAN trial. Over two years, Fabhalta demonstrated a statistically significant and clinically meaningful benefit in preserving kidney function, with an annualized mean decline in estimated glomerular filtration rate (eGFR) of 3.0 mL/min/1.73 m² per year, compared with 5.7 mL/min/1.73 m² per year among patients receiving placebo. The therapy also consistently showed superior performance across multiple key renal efficacy endpoints.

The Phase III study further confirmed Fabhalta’s favorable safety profile, consistent with earlier clinical findings. The most frequently reported adverse events included abdominal pain, dizziness, and nausea. As with other complement inhibitors, Fabhalta carries a risk of serious infections caused by encapsulated bacteria and is therefore available only through a Risk Evaluation and Mitigation Strategy (REMS) program, which requires patients to receive appropriate vaccinations before initiating treatment.

GSK Unveils Phase III CALM-1 and CALM-2 Data Evaluating Camlipixant in Refractory Chronic Cough

GSK plc has announced results from its Phase III CALM-1 and CALM-2 clinical trials evaluating the efficacy and safety of camlipixant, a P2X3 receptor antagonist, in adults with refractory chronic cough (RCC), a condition with limited treatment options and significant unmet medical need.

In the CALM-1 study, camlipixant administered at 50 mg twice daily achieved the primary endpoint by significantly reducing 24-hour cough frequency compared with placebo after 12 weeks of treatment. However, the CALM-2 trial did not demonstrate a statistically significant improvement for the same dose at week 24. The lower 25 mg twice-daily dose failed to meet the primary endpoint in either trial. Additionally, neither study achieved the predefined goals for key secondary outcomes, including assessments using the Chronic Cough Diary (CCD). Safety findings were comparable across both studies, with treatment-related adverse events occurring at similar rates and severity in the camlipixant and placebo groups.

After reviewing the combined findings from the CALM programme, GSK concluded that the treatment’s clinical benefit was insufficient to meaningfully improve the management of refractory chronic cough and has therefore discontinued further development of camlipixant for this indication. The company plans to present and publish the Phase III trial data to support ongoing research and understanding of RCC.

Meanwhile, GSK will continue the ongoing Phase IIb BALANCE trial (NCT07519395), which is investigating the efficacy and safety of camlipixant in adults with irritable bowel syndrome with diarrhoea (IBS-D) and irritable bowel syndrome with mixed bowel habits (IBS-M).

Merck’s LIPFENDRA Receives FDA Approval as the First Oral PCSK9 Inhibitor to Lower LDL-C

Merck has received U.S. FDA approval for LIPFENDRA® (enlicitide) 20 mg tablets as an adjunct to diet and exercise for lowering low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including those with heterozygous familial hypercholesterolemia (HeFH). LIPFENDRA is the first FDA-approved oral PCSK9 inhibitor and a first-in-class macrocyclic peptide therapy designed to reduce LDL-C, commonly referred to as “bad cholesterol.”

Commenting on the approval, Dr. Dean Y. Li, president of Merck Research Laboratories, highlighted that LIPFENDRA combines PCSK9 inhibition with innovative macrocyclic peptide technology to deliver significant LDL-C reductions in a convenient once-daily oral formulation. He noted that the approval represents an important milestone in expanding treatment options for adults with elevated LDL-C and acknowledged the collaborative regulatory review process that supported its approval.

The FDA decision was supported by findings from the Phase III CORALreef Lipids and CORALreef HeFH studies. In the CORALreef Lipids trial, LIPFENDRA achieved a 56% reduction in LDL-C versus placebo at 24 weeks, while a post-hoc analysis using revised data handling methods showed a 60% reduction from baseline after excluding biologically implausible baseline LDL-C values. In the CORALreef HeFH study, treatment with LIPFENDRA reduced LDL-C by 59% compared with placebo at the same time point. Across both studies, the therapy also lowered other atherogenic lipid markers linked to atherosclerotic cardiovascular disease (ASCVD), including non-HDL cholesterol and apolipoprotein B (ApoB).

LIPFENDRA demonstrated a safety profile comparable to placebo in the CORALreef Lipids study. In patients with HeFH enrolled in CORALreef HeFH, diarrhea and dizziness were reported more frequently among those receiving LIPFENDRA than placebo, although treatment discontinuation due to adverse events occurred at similar rates in both treatment groups.

According to Dr. Ann Marie Navar, associate professor of medicine in the Division of Cardiology at UT Southwestern Medical Center and a lead investigator of the CORALreef Lipids trial, elevated LDL-C remains a key contributor to ASCVD, the world’s leading cause of death. She emphasized that the strong LDL-C reductions observed across both Phase III studies mark the introduction of the first oral PCSK9 inhibitor available for cholesterol lowering.

Merck is also conducting an ongoing cardiovascular outcomes study to evaluate whether LIPFENDRA can reduce cardiovascular morbidity and mortality. At present, its effect on these long-term clinical outcomes has not yet been established.

Innovent Biologics’ Phase II Vitiligo Trial of TYK2 inhibitor Soficinib Meets Primary Endpoint

Innovent Biologics announced that its proprietary next-generation oral TYK2 inhibitor, soficitinib (ICP-332), achieved the primary endpoint in a Phase II/III clinical study evaluating the therapy for adults with non-segmental vitiligo. The randomized, double-blind, placebo-controlled, parallel-group, adaptive, multicenter trial was designed to assess the efficacy and safety of soficitinib in patients with non-segmental vitiligo. The study includes an initial Phase II portion followed by a Phase III stage.

Results from the Phase II segment demonstrated significant improvements in facial repigmentation after 24 weeks of treatment. Patients receiving 80 mg of soficitinib once daily experienced a 38.8% reduction from baseline in the Facial Vitiligo Area Scoring Index (F-VASI), while those receiving the 120 mg once-daily dose achieved a 41.2% reduction. In comparison, the placebo arm recorded only a 2.2% reduction. Both treatment groups showed statistically significant superiority over placebo (P<0.0001).

The investigational therapy also exhibited a favorable safety and tolerability profile, consistent with findings from earlier clinical studies, with no new safety concerns identified during the trial. Innovent Biologics stated that comprehensive efficacy and safety findings will be presented at upcoming international scientific meetings and published in peer-reviewed journals.

Soficitinib is an investigational oral TYK2 inhibitor selectively targeting the TYK2 pathway and has been independently developed by Innovent Biologics for the treatment of T-cell-mediated autoimmune disorders. Beyond vitiligo, the company is advancing the candidate across multiple dermatological indications, including atopic dermatitis, psoriasis, chronic urticaria, and nodular prurigo. TYK2 belongs to the Janus kinase (JAK) family and is an important regulator of the JAK-STAT signaling pathway involved in inflammatory and immune-mediated diseases.

Commenting on the results, Dr. Cui Jisong, Co-founder, Chairman, and Chief Executive Officer of Innovent Biologics, stated that the successful achievement of the primary endpoint in the Phase II study marks an important milestone for soficitinib. He added that the next-generation oral TYK2 inhibitor has the potential to offer patients with vitiligo an innovative treatment option that combines improved efficacy, a favorable safety profile, and convenient oral administration.

Vitiligo is a chronic autoimmune skin disorder characterized by the destruction of melanocytes, leading to depigmented white patches on the skin. The condition affects an estimated 0.5% to 2% of the global population and typically requires long-term disease management. Current treatment strategies focus on halting disease progression, promoting repigmentation, and maintaining therapeutic benefits to minimize the risk of recurrence.

Lilly to Acquire AtaiBeckley to Expand Treatment-Resistant Depression Pipeline

Eli Lilly and Company has entered into a definitive agreement to acquire AtaiBeckley Inc., a biotechnology company focused on developing rapid-acting neuroplastogens. AtaiBeckley’s portfolio includes several clinical-stage candidates alongside a discovery pipeline of next-generation therapies. Its lead candidate, BPL-003 (mebufotenin benzoate), is an intranasal synthetic 5-MeO-DMT therapy being investigated for treatment-resistant depression (TRD), a condition affecting millions of individuals in the United States.

Research suggests that treatment-resistant depression and other severe psychiatric disorders may be associated with impaired synaptic plasticity, limiting the brain’s ability to establish and reinforce neural connections involved in mood regulation. AtaiBeckley’s therapeutic approach is designed to restore synaptic connectivity and stimulate the formation of new neural pathways, providing a differentiated mechanism of action compared with traditional antidepressants that primarily modulate neurotransmitter activity.

Under the agreement, Lilly will acquire all outstanding AtaiBeckley shares for $6.75 per share in cash at closing, with shareholders also eligible to receive up to $2.50 per share through a Contingent Value Right (CVR). The CVR provides milestone-based cash payments, including $1.00 per share upon the initiation of a Phase III trial of VLS-01 before the fourth anniversary of closing, $0.50 per share following U.S. regulatory approval and DEA rescheduling of BPL-003 before the fifth anniversary, and $1.00 per share upon U.S. regulatory approval and DEA rescheduling of VLS-01 before the seventh anniversary. The upfront consideration values AtaiBeckley at approximately $2.8 billion, with the CVR offering up to an additional $1.0 billion in potential value, although these milestone payments are not guaranteed.

The acquisition does not require financing and is anticipated to close during the third quarter of 2026, subject to shareholder approval, regulatory clearances, and other customary closing conditions. The cash purchase price represents an approximately 40% premium over AtaiBeckley’s 30-day volume-weighted average share price through July 15, 2026. The transaction has received unanimous approval from the boards of directors of both Lilly and AtaiBeckley.