INOVIO’s VOCERYQ Receives FDA Accelerated Approval for Adult RRP; AbbVie Submits Etentamig Biologics License Application to FDA for R/R Multiple Myeloma; Setara Biosciences Raises $90 Million Series A to Advance Rare Disease Precision Medicines; BioMarin Expands Fabry Disease Portfolio With Exclusive Global License for Next-Generation ERT; Pfizer’s ELREXFIO Advances Multiple Myeloma Treatment With Positive Phase 3 Results

INOVIO Announces Accelerated FDA Approval for VOCERYQ in Adult RRP

INOVIO announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to VOCERYQ (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd), formerly known as INO-3107, for reducing the frequency of surgical interventions in adults with recurrent respiratory papillomatosis (RRP). The approval was granted under the FDA’s accelerated approval pathway, based on the achievement of a complete response, defined as no requirement for surgical intervention during the 12 months following treatment in the efficacy population. Continued approval for this indication may be dependent on the confirmation and further characterization of clinical benefits through one or more confirmatory studies.

The approval is based on findings from INOVIO’s RRP-001 clinical study, which assessed VOCERYQ in 32 adults with RRP who had undergone at least two surgeries during the year preceding treatment. No additional surgical excisions of papillomas were required during the VOCERYQ treatment period specified in the study protocol. Among the 32 participants, 26 had undergone at least three surgical procedures to remove papillomas in the 12 months before treatment and constituted the efficacy population. The median number of surgical excisions in this group during the year before the first dose was 4.5, ranging from 3 to 8. Following 12 months of follow-up, seven of these 26 patients (27%; 95% CI: 12–48%) achieved a complete response, defined as requiring no surgical removal of papillomas. This outcome is considered unlikely to occur during the natural course of untreated RRP. Comprehensive clinical findings for VOCERYQ have been published in Nature Communications and The Laryngoscope, a leading publication for specialists managing RRP.

Kim McClellan, President of the RRP Foundation, highlighted the approval as a significant advancement toward providing patients with access to effective nonsurgical treatment alternatives. She emphasized the importance of incorporating patient perspectives into drug development and noted that individuals with RRP have historically faced repeated surgical procedures to manage their condition. McClellan acknowledged INOVIO, the participating patients, and clinical investigators for their contributions to VOCERYQ’s development, expressing optimism that the therapy could further reshape the RRP treatment landscape. She also underscored the Foundation’s commitment to collaborating with clinicians, regulatory authorities, and healthcare industry partners to improve the quality of life for people living with RRP.

VOCERYQ demonstrated a favorable tolerability profile, with the most frequently reported adverse reactions occurring at an incidence greater than 5% and including injection-site reactions, headache, fatigue, hypertension, nausea, and dizziness. Most treatment-related adverse events were transient and classified as Grade 1 or Grade 2, with no common Grade 4 or Grade 5 adverse events reported. All 32 participants completed the study. Additional important safety information has also been provided.

INOVIO is expected to conduct a confirmatory study to further establish VOCERYQ’s clinical benefit following accelerated approval. The FDA has required the company to submit a draft study protocol by the end of 2026, with the objective of reaching agreement on a finalized protocol by mid-2027.

VOCERYQ received approval for administration in combination with CELLECTRA® 5PSP, INOVIO’s proprietary DNA medicine delivery device. The company anticipates that VOCERYQ will become available for ordering and shipment within approximately 60 days, with broader commercial field deployment expected during the first quarter of 2027.

AbbVie Submits Regulatory Application for Etentamig in Adults With R/R Multiple Myeloma

AbbVie announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval for etentamig, a second-generation BCMA × CD3 bispecific antibody T-cell engager administered intravenously for the treatment of adults with relapsed or refractory multiple myeloma.

BCMA-targeted bispecific antibodies and CAR-T therapies have substantially transformed the multiple myeloma treatment landscape. Nevertheless, their widespread utilization may be constrained by potential adverse events, including cytokine release syndrome, infections, and neurotoxicity, alongside the need for specialized treatment administration and monitoring. Furthermore, therapeutic options become increasingly limited as patients experience relapse in later treatment lines. These challenges collectively underscore the need for additional therapeutic approaches that target disease biology while facilitating effective treatment delivery across diverse healthcare settings.

According to Daejin Abidoye, M.D., vice president and therapeutic area head of oncology, solid tumor and hematology at AbbVie, multiple myeloma is a chronic hematologic malignancy that significantly impacts patients’ lives, making it essential to develop therapies that combine efficacy with practical treatment delivery. He noted that this regulatory milestone represents progress toward potentially introducing an innovative, patient-focused treatment option characterized by a manageable safety profile and the convenience of monthly dosing, addressing the needs of patients across different care settings.

The BLA submission is supported by findings from the global, multicenter, randomized Phase 3 CERVINO trial, which compared etentamig with investigator-selected standard available therapies (SAT) in patients with triple-class-exposed relapsed or refractory multiple myeloma who had previously received at least two lines of treatment. AbbVie had announced the study’s topline results earlier in the year.

Setara Biosciences Announces $90 Million Series A to Expand Its Precision Medicine Pipeline

Setara Biosciences, Inc. announced the successful completion of a $90 million Series A financing round. The company was established following Jazz Pharmaceuticals’ acquisition of Actio Biosciences, which included its lead investigational candidate ABS-1230 for KCNT1-related epilepsy. Setara plans to develop Actio’s remaining pipeline, primarily its clinical-stage small-molecule TRPV4 inhibitor STAR-0871 (previously ABS-0871), targeting Charcot-Marie-Tooth disease type 2C (CMT2C) and overactive bladder, alongside several earlier-stage discovery programs. The financing was backed by existing investors Deerfield Management, Canaan, Droia Ventures, EcoR1, Euclidean Capital, and Regeneron Ventures, with Jazz Pharmaceuticals joining as a new shareholder in connection with the Actio acquisition.

STAR-0871 represents the company’s lead development program and is being investigated as a potential first-in-class oral small-molecule TRPV4 inhibitor for TRPV4-associated neuromuscular disorders, including CMT2C, as well as overactive bladder. CMT2C is a rare inherited condition affecting motor and sensory functions, with an estimated prevalence of approximately 2,500 individuals in the U.S. Meanwhile, overactive bladder affects tens of millions of Americans, while currently available therapies provide limited efficacy for many patients.

CMT2C results from gain-of-function mutations in the TRPV4 gene, leading to excessive activity of the TRPV4 ion channel. STAR-0871 is designed to selectively inhibit this channel and directly target the underlying genetic mechanism of the disease. Considering TRPV4’s involvement in additional physiological processes, Setara also anticipates that STAR-0871 could offer therapeutic benefits in more common disorders, including overactive bladder.

Setara has completed the healthy-volunteer component of the Phase 1 clinical trial evaluating STAR-0871. The company intends to initiate a Phase 1b patient study, designated THERON, in individuals with CMT2C in 2027. In support of its development for this indication, STAR-0871 has received Rare Pediatric Disease, Orphan Drug, and Fast Track designations from the U.S. FDA. Additionally, the company plans to launch a Phase 2a clinical study to assess STAR-0871 in patients with overactive bladder. Beyond its primary candidate, Setara is advancing a pipeline of early-stage discovery initiatives directed toward genetic epilepsies and additional therapeutic indications, including migraine.

BioMarin Expands Fabry Disease Development Strategy With Exclusive Global ERT Licensing Agreement

BioMarin Pharmaceutical Inc. announced the execution of an exclusive global licensing agreement with GC Biopharma Corp. and Hanmi Pharm Co., Ltd. for the development and commercialization of HM15421/GC1134A, an investigational long-acting enzyme replacement therapy (ERT) being developed for Fabry disease.

HM15421/GC1134A is a recombinant human alpha-galactosidase A (α-Gal A) enzyme therapy currently undergoing evaluation in a Phase 1/2 clinical trial involving patients with Fabry disease. The candidate has the potential to become the first subcutaneous enzyme replacement therapy available for this condition.

Alexander Hardy, president and chief executive officer of BioMarin, stated that the collaboration underscores the company’s increasing dedication to the Fabry disease community, further strengthening its commitment following the acquisition of Amicus earlier in the year. He highlighted HM15421/GC1134A’s potential to introduce a novel treatment approach and become a meaningful therapeutic option for patients. Hardy also emphasized that the candidate complements BioMarin’s expanding clinical pipeline, encompassing both internally developed and externally sourced programs, and could enable the company to serve a broader patient population beyond individuals with mutations eligible for treatment with GALAFOLD.

Under the agreement, BioMarin will provide an upfront payment of $70 million to GC Biopharma and Hanmi. The two companies will additionally be eligible to receive up to $405 million in aggregate development and commercial milestone payments, along with tiered royalties, subject to the achievement of specified milestones.

Preclinical investigations of HM15421/GC1134A demonstrated differentiated therapeutic effects across several important parameters, including kidney function, vascular health, and peripheral nervous system outcomes. The candidate was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) in 2024. Upon completion of the ongoing Phase 1/2 clinical study, BioMarin will assume exclusive responsibility for subsequent clinical development, manufacturing, and commercialization of HM15421/GC1134A in all markets outside South Korea. GC Biopharma and Hanmi will retain commercialization rights within South Korea.

Pfizer Announces Positive Phase 3 Results for ELREXFIO in Multiple Myeloma

Pfizer Inc. announced positive topline findings from the Phase 3 MagnetisMM-32 trial, which investigated ELREXFIO® (elranatamab) as a monotherapy in adults with relapsed or refractory multiple myeloma (RRMM) who had received at least one previous treatment line incorporating an anti-CD38 agent (daratumumab or isatuximab) and lenalidomide.

At the planned interim efficacy analysis, MagnetisMM-32 achieved its primary endpoint of progression-free survival (PFS), evaluated through blinded independent central review (BICR). ELREXFIO demonstrated a statistically significant and clinically meaningful decrease in the risk of disease progression or death compared with investigator-selected treatment regimens, including elotuzumab, pomalidomide, and dexamethasone (EloPd); pomalidomide, bortezomib, and dexamethasone (PVd); or carfilzomib and dexamethasone (Kd). The study incorporated one of the most comprehensive standard-of-care comparator approaches among Phase 3 BCMA-directed bispecific antibody trials in this patient population, allowing investigators to select from three established therapeutic combinations.

At the time of the analysis, over 75% of patients treated with ELREXFIO remained alive without disease progression, with the estimated PFS rate reaching 68% at 18 months.¹ Overall survival (OS), a key secondary endpoint, had not yet matured sufficiently for analysis, and patient follow-up is ongoing until the next planned OS assessment.

The safety findings observed with ELREXFIO in MagnetisMM-32 were consistent with its established safety profile, with no previously unidentified safety concerns reported. Infection prevention measures implemented from the initiation of treatment helped reduce infection-related risks, including severe infections. Grade 3 or higher infections occurred in 27.5% of patients receiving ELREXFIO compared with 18.1% in the investigator-selected treatment group. Grade 5 infections were reported in 0.8% and 1.6% of patients, respectively.

Pfizer intends to submit the MagnetisMM-32 findings to regulatory authorities worldwide, with comprehensive results expected to be presented at a future scientific conference and published in a medical journal.