Machado-Joseph Disease

BEIJING, China I March 24, 2025 I SineuGene Therapeutics Co., Ltd. (“SineuGene”), a clinical-stage biotech company pioneering innovative therapies for neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for SNUG01 — a first-in-class TRIM72 (Tripartite Motif Protein 72)-targeted gene therapy candidate for amyotrophic lateral sclerosis (ALS). The clearance authorizes a global Phase I/IIa clinical trial designed to evaluate SNUG01’s safety, tolerability, and preliminary efficacy in adults with ALS through a dose-escalation and expansion study. ALS is a rapidly progressive neurodegenerative disorder marked by motor neuron degeneration in the brain and spinal cord, leading to muscle weakness, atrophy, and eventually respiratory failure. Median survival time post-diagnosis is 3–5 years. Despite being the most prevalent adult-onset motor neuron disease, existing therapies offer limited clinical benefits, slowing progression only marginally and leaving no curative options for patients. SNUG01 leverages groundbreaking research carried out by Dr. Yichang Jia’s laboratory at Tsinghua University, whose team identified TRIM72 as a multifunctional neuroprotectant. The therapy utilizes a recombinant adeno-associated virus serotype 9 (rAAV9) capsid to deliver the human TRIM72 gene via intrathecal administration. Preclinical studies demonstrate TRIM72’s ability to counteract ALS pathogenesis through several synergistic mechanisms such as reducing oxidative stress by scavenging reactive oxygen species (ROS), restoring mitochondrial homeostasis, suppressing stress granule dysregulation, inhibiting neuroinflammatory cascades, and enhancing neuronal membrane repair capacity In an investigator-initiated trial (IIT) at Peking University Third Hospital, SNUG01 demonstrated a favorable safety and tolerability profile, along with early efficacy signals in both functional clinical assessments and key biomarkers of neurodegeneration. These results enhance the translational validation of TRIM72, bridging target discovery, preclinical models, and human proof-of-concept. Unlike mutation-targeting gene therapies, SNUG01 leverages multiple neuroprotective mechanisms, offering a critical advantage for over 90% of ALS patients with sporadic (non-familial) disease. This unique multi-target approach positions SNUG01 as a first-in-class candidate for the majority of ALS cases, addressing an urgent unmet need in this underserved population. SineuGene will partner with a leading global consortium of academic and clinical institutions to conduct a multi-regional clinical trial, expediting validation of SNUG01’s safety and therapeutic efficacy profile across a diverse ALS patient populations. About SineuGene SineuGene Therapeutics Co., Ltd., often referred to as “SineuGene,” is a biotechnology company focused on developing innovative therapies for neurological diseases. Established in late 2021, the company builds on over a decade of scientific research conducted by Dr. Yichang Jia’s team from School of Medicine, Tsinghua University. SineuGene uses advanced technologies such as adeno-associated virus (AAV) vector-mediated gene delivery and editing and antisense oligonucleotide (ASO)- based gene expression regulation. The company has created a diverse pipeline that targets major neurological diseases, including Amyotrophic Lateral Sclerosis (ALS), stroke, Parkinson’s disease, Alzheimer’s disease, Spinocerebellar Ataxia type 3, and Huntington’s disease. SineuGene is dedicated to transforming groundbreaking research into effective treatments, aiming to meet the unmet medical needs in the field of neurology. Website: www.sineugene.com SOURCE: SineuGene Therapeutics

PASADENA, Calif.--( BUSINESS WIRE )--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the global licensing and collaboration agreement with Sarepta Therapeutics (NASDAQ: SRPT) announced on November 26, 2024, has now closed. Closing of the transaction was subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions. Summary Financial Terms Upon closing, Arrowhead receives a $500 million upfront payment and $325 million through the purchase by Sarepta of Arrowhead common stock priced at $27.25 per share. Arrowhead will also receive $250 million to be paid in annual installments of $50 million over 5 years. Arrowhead also has the potential to receive $300 million in near-term payments associated with the continued enrollment of certain cohorts of a Phase 1/2 study of ARO-DM1, which Arrowhead is on track to achieve during the next 12 months. Arrowhead is eligible to receive development milestone payments of between $110 million and $410 million per program and sales milestone payments of between $500 million and $700 million per program. Arrowhead is also eligible to receive tiered royalties on commercial sales up to the low double digits. Summary of Programs under License and Collaboration Agreement Clinical Stage ARO-DUX4, which is designed to target the gene that encodes the DUX4 protein as a potential treatment for patients with facioscapulohumeral muscular dystrophy type 1, currently dosing patients in a Phase 1/2 clinical study. ARO-DM1, which is designed to reduce expression of the dystrophia myotonica protein kinase (DMPK) gene in skeletal muscle as a potential treatment for patients with type 1 myotonic dystrophy, currently dosing patients in a Phase 1/2 clinical study. ARO-MMP7, which is designed to reduce expression of matrix metalloproteinase 7 (MMP7) in the lung as a potential treatment for idiopathic pulmonary fibrosis, currently dosing patients in a Phase 1/2 clinical study. ARO-ATXN2, which is designed to silence expression of the toxic ATXN2 protein in the CNS as a potential treatment for spinocerebellar ataxia 2 (SCA2), currently in a Phase 1/2 study that is open for enrollment. Preclinical Stage ARO-HTT for patients Huntington’s disease expected to be CTA-ready in 2025 ARO-ATXN1 for patients with spinocerebellar ataxia 1 (SCA1) expected to be CTA-ready in 2026 ARO-ATXN3 for patients with spinocerebellar ataxia 3 (SCA3) expected to be CTA-ready in 2026 Discovery During the five-year term, Sarepta can propose up to six new CNS or muscle targets for which Arrowhead will perform discovery and preclinical development. Sarepta would then receive an exclusive license to those programs and be responsible for clinical development and commercialization. Drug Manufacturing Under the agreement, Arrowhead will manufacture clinical drug supply for all programs arising out of the license and collaboration, and commercial drug product for the four programs currently in clinical trials. About Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit www.arrowheadpharma.com , or follow us on X (formerly Twitter) at @ArrowheadPharma , LinkedIn , Facebook , and Instagram . To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts . Safe Harbor Statement under the Private Securities Litigation Reform Act: This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline or product candidates, including anticipated regulatory submissions and clinical program results, prospects or benefits of our collaborations with other companies, or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the impact of the ongoing COVID-19 pandemic on our business, the safety and efficacy of our product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances. Source: Arrowhead Pharmaceuticals, Inc.

WOODBRIDGE, Conn.--( BUSINESS WIRE )--Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for spinocerebellar ataxia type 3 (SCA3), a neurodegenerative disorder with no current treatment. Cure Rare Disease launched its SCA3 program in 2021 to develop an antisense oligonucleotide (ASO) therapy, with initial funding support provided by Gregory Klassen, a patient living with SCA3. In 2022 and 2023, in collaboration with Leiden University Medical Center and the Cure Rare Disease team, several in vivo studies were conducted in a disease mouse model, demonstrating functional improvement. A clinical candidate was identified through non-GLP toxicology studies conducted with Charles River Labs. In 2024, a Type B pre-IND meeting with the Food and Drug Administration (FDA) provided regulatory guidance ahead of manufacturing scale-up and clinical trial design. With CIRM funding support, Cure Rare Disease will complete manufacturing scale-up, conduct IND-enabling toxicology studies and submit an investigational new drug (IND) application to initiate early clinical trials. The trials will be led by Principal Investigator Dr. Susan Perlman at UCLA. "This program is a powerful example of what can be achieved when patients, organizations, researchers and governing bodies come together with a shared mission. From Greg Klassen’s initial support to our collaborations with leading scientists and clinicians, industry partners and regulatory guidance from the FDA, the development of a therapy for SCA3 has been a truly unified effort,” said Cure Rare Disease CEO and founder Richard Horgan. “We are incredibly appreciative of CIRM’s support in helping us take this program into the clinic, proving that rare disease treatments can be brought to patients through our model of collective dedication and innovation." About SCA3 Spinocerebellar ataxia type 3 (SCA3) is a rare neuromuscular disease with a prevalence of one to five in 100,000 people. It is the most common form of spinocerebellar ataxia. The disease is caused by mutations in the ATXN3 gene, which codes for the enzyme ataxin-3. Ataxin-3 is found throughout the body and helps destroy and remove damaged proteins. SCA3 results from a mutation that causes a toxic accumulation of trinucleotide repeats. About Cure Rare Disease (CRD) Cure Rare Disease (CRD) is a nonprofit biotechnology company focused on developing genetic medicines for rare and ultra-rare diseases. CRD is committed to accelerating the development of treatments for individuals with rare diseases through a patient-centric approach to research and development. The company's programs include gene therapy and antisense oligonucleotide (ASO) treatments for a range of rare diseases. For more information, visit www.cureraredisease.org . About the California Institute for Regenerative Medicine (CIRM) The California Institute for Regenerative Medicine (CIRM) is a funding agency established by Californians to accelerate regenerative medicine research and deliver treatments for patients with unmet medical needs. Established in 2004 through the passage of Proposition 71, CIRM was initially funded with $3 billion from the state of California to support ongoing research. In 2020, Proposition 14 provided an additional $5.5 billion to continue the agency’s work. CIRM has funded billions in stem cell and genetic research, as well as development programs. Through its research, infrastructure and education programs, CIRM aims to transform regenerative medicine, stimulate economic growth and improve the lives of diverse communities throughout the state. For more information, visit www.cirm.ca.gov .