Satellos Reports Promising Efficacy Signals for Novel DMD Treatment in Adult Patients
• Satellos Bioscience's investigational treatment for Duchenne muscular dystrophy (DMD) has demonstrated encouraging efficacy signals in adult patients, offering potential new options in a challenging therapeutic area.
• The development comes amid setbacks in the DMD field, including Pfizer's withdrawal of its gene therapy fordadistrogene movaparvovec following a Phase III failure and a Phase II patient fatality.
• The global DMD treatment market is projected to grow substantially from $2.3 billion in 2023 to $5.2 billion by 2033 across major markets, primarily driven by Elevidys and Santhera Pharmaceuticals' Agamree.
Tourmaline Bio's Heart Disease Antibody Shows Promise in Phase 2, But Shares Fall Amid Competition Concerns
• Tourmaline Bio's antibody drug pacibekitug demonstrated significant reduction in cardiovascular risk biomarkers compared to placebo in Phase 2 trials, but shares fell 18% as investors question its competitive edge against Novo Nordisk's offering.
• Schrödinger announced CFO Geoffrey Porges will leave the company amid near-record low share prices, coinciding with plans to lay off 7% of staff as part of cost-cutting measures.
• CRISPR Therapeutics is expanding its technological capabilities through a $95 million deal with Sirius Therapeutics to develop SRSD107, a novel siRNA blood thinner designed to minimize bleeding risk.
REGENXBIO Secures $250 Million Royalty Monetization Deal to Advance Gene Therapy Pipeline
• REGENXBIO has closed a non-dilutive royalty bond agreement with Healthcare Royalty for up to $250 million, receiving $150 million upfront that extends its cash runway into early 2027.
• The deal monetizes select royalties from ZOLGENSMA for SMA and payments from gene therapies for MPS disorders, while REGENXBIO retains other funding opportunities including a potential Priority Review Voucher.
• This strategic financing supports REGENXBIO's late-stage pipeline development, including RGX-121 for MPS II, RGX-202 for Duchenne muscular dystrophy, and ABBV-RGX-314 for wet AMD.
FDA's Cell and Gene Therapy Champion Peter Marks Departs, Leaving Industry at Critical Juncture
• Peter Marks, head of FDA's Center for Biologics Evaluation and Research since 2016, has resigned, leaving cell and gene therapy developers without their biggest regulatory advocate during a challenging investment period.
• Under Marks' leadership, the FDA approved dozens of cell and gene therapies including the first gene therapy, first cellular treatment for cancer, and first CRISPR gene editing medicine, establishing flexible regulatory frameworks for these novel treatments.
• Despite concerns about regulatory uncertainty following Marks' departure, incoming FDA Commissioner Marty Makary has signaled support for conditional approval pathways for rare disease treatments where randomized controlled trials aren't feasible.
Wave Life Sciences to Seek FDA Approval for Duchenne Muscular Dystrophy Drug Following Promising Phase 2 Results
• Wave Life Sciences will pursue FDA accelerated approval for WVE-N531, an exon 53 skipping therapy for Duchenne muscular dystrophy, following positive Phase 2 trial results showing increased dystrophin production.
• The experimental treatment demonstrated substantial improvements in muscle health with a statistically significant improvement in "time to rise" functional tests compared to historical controls through 48 weeks of treatment.
• If approved, WVE-N531 could offer a potential monthly dosing regimen and become a new therapeutic option for approximately 8-10% of Duchenne patients with specific genetic mutations amenable to exon 53 skipping.
Adverum Launches Groundbreaking Phase 3 ARTEMIS Trial for One-Time Gene Therapy in Wet AMD
• Adverum Biotechnologies initiates ARTEMIS, the first-ever registrational intravitreal gene therapy trial for wet AMD, evaluating Ixo-vec against standard aflibercept treatment in 284 patients.
• The innovative trial design includes both treatment-naïve and previously treated patients, comparing a single Ixo-vec injection to regular aflibercept dosing with a primary endpoint of non-inferior vision outcomes.
• Current data shows 42% of wet AMD patients discontinue treatment after 2 years, highlighting the potential impact of Ixo-vec's one-time administration approach for long-term vision preservation.
Clearside's CLS-AX Phase 2b Trial Data Shows Promise in Treatment-Naïve AMD Patients
• New subgroup analyses from Clearside Biomedical's ODYSSEY Phase 2b trial provide strategic insights for upcoming Phase 3 program in neovascular age-related macular degeneration.
• Data presented at the Angiogenesis 2025 meeting supports focusing on treatment-naïve patients and implementing specific visual acuity criteria for future trials.
• The 36-week ODYSSEY trial employed a robust design, being randomized, double-masked, and active-controlled across multiple centers.
REGENXBIO and Nippon Shinyaku Partner to Advance Gene Therapies for MPS I and MPS II
• REGENXBIO and Nippon Shinyaku have entered an exclusive partnership to develop and commercialize RGX-121 for MPS II and RGX-111 for MPS I.
• Nippon Shinyaku will lead commercialization in the U.S. and Asia, while REGENXBIO will continue to lead clinical development and manufacturing.
• The agreement includes an upfront payment of $110 million to REGENXBIO, with potential milestone payments reaching up to $700 million.
• RGX-121 and RGX-111 are AAV-based gene therapies designed to address the underlying enzyme deficiencies in MPS II and MPS I, respectively.
Focused Ultrasound Shows Promise in Alzheimer's Treatment
• A clinical trial using focused ultrasound to treat Alzheimer’s disease has shown promising results, marking a potential new direction in treating this debilitating condition.
• The study, published in the Journal of Neurosurgery, highlights the noninvasive nature of focused ultrasound as a key advantage in Alzheimer's treatment.
• Researchers are optimistic about the potential of this approach to offer a new therapeutic avenue for patients suffering from Alzheimer's disease.
• Further research is needed to fully understand the long-term effects and optimal use of focused ultrasound in Alzheimer's treatment.
Arvinas Advances Vepdegestrant into Phase 3 Trials for Breast Cancer and Updates Pipeline Milestones
• Arvinas plans to initiate two Phase 3 trials in 2025 for vepdegestrant in ER+/HER2- metastatic breast cancer, one in the first-line setting with atirmociclib and another in the second-line setting with a CDK4/6 inhibitor.
• Topline data from the Phase 3 VERITAC-2 monotherapy trial of vepdegestrant in second-line-plus ER+/HER2- metastatic breast cancer is anticipated in the first quarter of 2025.
• Arvinas is set to present initial data from the Phase 1 trial of ARV-393 in B-cell lymphomas and file an IND application for a novel PROTAC KRAS G12D degrader in 2025.
• Phase 1 trial with PROTAC LRRK2 degrader ARV-102 in patients with Parkinson’s disease has been initiated, with data expected to be presented in the first half of 2025.
Regenxbio Expands MPS I Gene Therapy Trial After Initial Data Review
Regenxbio has decided to expand its phase 1/2 trial for RGX-111, a gene therapy targeting mucopolysaccharidosis type I (MPS I), following promising initial data showing clinical activity. The therapy aims to deliver a functional copy of the IDUA gene to the central nervous system, offering a potential one-shot treatment for this rare inherited disease.
Sarepta Seeks Accelerated Approval for DMD Gene Therapy SRP-9001
• Sarepta Therapeutics has submitted SRP-9001 (delandistrogene moxeparvovec) to the FDA for accelerated approval to treat ambulatory Duchenne muscular dystrophy (DMD) patients.
• The filing is based on positive data from early-stage studies, showing improvements in clinical function and a consistent safety profile, while awaiting Phase 3 EMBARK results.
• SRP-9001, a one-time gene therapy, delivers a shortened dystrophin gene via an AAV vector, addressing the underlying genetic defect in DMD patients.
• If approved, SRP-9001 would offer a one-time treatment option for DMD, contrasting with Sarepta's existing chronic exon-skipping therapies.
Elevidys Gene Therapy Shows Sustained Benefits in Duchenne Muscular Dystrophy Patients
• Sarepta Therapeutics' Elevidys demonstrates sustained benefits and disease stabilization in ambulatory Duchenne muscular dystrophy (DMD) patients, according to Phase 3 EMBARK trial results.
• Crossover-treated patients showed a 2.34-point improvement on the North Star Ambulatory Assessment (NSAA) compared to matched external controls after 52 weeks of Elevidys treatment.
• Patients treated with Elevidys in Part 1 of EMBARK maintained clinically meaningful improvements in NSAA, Time to Rise (TTR), and 10-meter walk/run (10MWR) at two years.
• Muscle biopsies showed consistent micro-dystrophin expression, and MRI scans indicated minimal muscle pathology progression, reinforcing Elevidys's long-term efficacy and safety.
Sozinibercept Shows Promise in Improving Visual Outcomes for nAMD Patients
• Sozinibercept, a novel VEGF-C/D inhibitor, is under investigation for neovascular age-related macular degeneration (nAMD) to improve visual acuity beyond current VEGF-A therapies.
• Phase 2b trials showed that sozinibercept combined with ranibizumab led to a statistically significant additional gain of 3.4 letters in BCVA compared to ranibizumab alone at 24 weeks.
• Ongoing Phase 3 trials (ShORe and COAST) are evaluating sozinibercept in combination with ranibizumab or aflibercept, with potential regulatory filings planned after 12-month efficacy analysis.
• Next-generation nAMD therapies like sozinibercept aim to target multiple pathways beyond VEGF-A to enhance vision and quality of life for patients, addressing the limitations of current treatments.
Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025
• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD.
• EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration.
• Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option.
• Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.
CytomX Therapeutics Reports Promising Interim Phase 1 Data for CX-2051 Antibody-Drug Conjugate
• CytomX Therapeutics has announced encouraging interim Phase 1 data for CX-2051, their novel antibody-drug conjugate targeting EpCAM-expressing solid tumors, showing early signs of clinical activity.
• The dose-escalation study demonstrated a manageable safety profile with dose-limiting toxicities observed only at higher dose levels, supporting continued development of the therapeutic candidate.
• Preliminary efficacy signals were observed across multiple tumor types, with several patients achieving stable disease, suggesting potential clinical benefit in difficult-to-treat EpCAM-positive malignancies.
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