On September 2, 2025 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported a global licensing and collaboration agreement with Novartis for ARO-SNCA, Arrowhead’s preclinical stage siRNA therapy against alpha-synuclein for the treatment of synucleinopathies, such as Parkinson’s Disease, and for other additional collaboration targets that will utilize Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform (Press release, Arrowhead Research Corporation, SEP 2, 2025, View Source [SID1234655634]). Upon closing, Arrowhead will receive $200 million as an upfront payment and is eligible to receive up to $2 billion in potential milestone payments plus royalties on commercial sales.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"With a robust neuroscience pipeline and clear commitment to neurodegenerative diseases and genetic medicines, Novartis is a compelling partner for Arrowhead in the CNS space," said Christopher Anzalone, Ph.D., President and CEO at Arrowhead. "Our TRiMTM platform has generated impressive preclinical results demonstrating delivery to CNS, including distribution to deep brain regions, after subcutaneous administration. The potential translation of these results in upcoming clinical trials would represent an important leap forward for neurodegenerative diseases and gene targets in the CNS that have been historically difficult to address. We look forward to working with Novartis to bring ARO-SNCA for the treatment of synucleinopathies, such as Parkinson’s Disease, into clinical trials as soon as possible and to collaborate on additional programs in the future."

Fiona Marshall, Ph.D., President of Biomedical Research at Novartis added, "Neurodegenerative conditions such as Parkinson’s Disease affect millions of patients worldwide. Novartis aspires to transform the lives of patients and families living with these diseases, by advancing medicines that significantly alter the course of the disease. We believe that one way to effectively target core drivers in Parkinson’s and other neurodegenerative diseases requires completely novel approaches to deliver RNA medicines to the brain. We see Arrowhead’s TRiMTM technology as having great potential to achieve the type of widespread and effective delivery in key brain structures that will be necessary to see the full benefit of RNA medicines in neurodegeneration."

Summary of License and Collaboration Agreement
Under the terms of the agreement, Novartis will receive an exclusive worldwide license to research, develop, manufacture, and commercialize ARO-SNCA, a preclinical stage program that utilizes Arrowhead’s TRiMTM platform for subcutaneous administration and delivery to the CNS designed to target the gene that encodes the alpha-synuclein protein as a potential treatment for patients with Parkinson’s Disease, and other synucleinopathies. Novartis will select additional collaboration targets outside of Arrowhead’s current pipeline to be developed using the TRiMTM platform.
For all licensed programs under the agreement, Arrowhead will conduct and complete preclinical research activities necessary to enable a clinical trial application (CTA) filing. Novartis will then assume sole control over development, manufacturing, medical affairs, and commercialization activities.

Summary Financial Terms
Upon closing, Novartis will make a $200 million upfront payment to Arrowhead. Arrowhead is also eligible to receive development, regulatory, and sales milestone payments of up to $2 billion. Arrowhead is further eligible to receive tiered royalties on commercial sales up to the low double digits.
The transaction is expected to close in the second half of 2025, subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions.
Gibson, Dunn & Crutcher LLP is serving as legal advisor to Arrowhead.

On September 2, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that the time of management’s presentation at the H.C. Wainwright 27th Annual Global Investment Conference, being held September 8th – 10th, 2025 in New York City, has been changed to 10:30 AM ET on September 9 (Press release, Anixa Biosciences, SEP 2, 2025, https://ir.anixa.com/news/detail/1096/anixa-biosciences-announces-time-change-for-presentation-at-the-h-c-wainwright-27th-annual-global-investment-conference [SID1234655633]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Mike Catelani, President and CFO of Anixa, will deliver a presentation and will be available for one-on-one meetings during the conference.

Details of the presentation are as follows:

Event: H.C. Wainwright 27th Annual Global Investment Conference
Date: September 9, 2025
Time: 10:30 AM ET
Location: Kennedy I – 4th Floor, Lotte New York Palace Hotel
Webcast: View Source

On September 2, 2025 Alpha Tau Medical Ltd. (Nasdaq: DRTS, DRTSW) ("Alpha Tau"), the developer of the innovative alpha-radiation cancer therapy Alpha DaRT reported that the first patient has been treated in its U.S. multi-center pancreatic cancer pilot study, known as IMPACT (Intratumoral Pancreatic Alpha Combination Trial), evaluating the safety, feasibility, and efficacy of Alpha DaRT in combination with chemotherapy for patients with newly diagnosed unresectable locally advanced or metastatic pancreatic adenocarcinoma (Press release, Alpha Tau Medical, SEP 2, 2025, View Source [SID1234655632]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Pancreatic cancer is the third leading cause of cancer-related death in the United States, with approximately 66,000 new cases diagnosed annually. Tragically, up to 87% of these patients are considered inoperable at diagnosis due to either locally advanced disease or distant metastases. These patients face a dismal prognosis, with limited benefit from existing systemic therapies.

Uzi Sofer, CEO of Alpha Tau, stated, "With the vast majority of pancreatic cancer patients deemed inoperable at diagnosis, the need for innovation is urgent. The initiation of the IMPACT trial in the U.S. marks an important step by exploring how Alpha DaRT, with its ultra-high dose and localized alpha radiation, might complement chemotherapy in treating this terrible disease. This pilot study is a key part of our broader strategy to bring Alpha DaRT to cancer patients with some of the highest unmet needs."

The first patient was treated for unresectable pancreatic cancer at the University Cancer Centers in Houston by a multidisciplinary team including the Principal Investigator, Radiation Oncologist Dr. Mark D’Andrea MD FACRO and Gastroenterologist Dr. Isaac Raijman MD.

Dr. D’Andrea noted, "The Alpha DaRT sources are designed to emit powerful alpha particles that travel only a short range in tissue. This is ideal for pancreatic tumors, which are surrounded by critical structures. Its biological effectiveness may offer a new way to achieve local control in a conformal manner in one session, instead of a more lengthy treatment seen with conventional radiation therapy. It also offers the potential for activation of a systemic response of the treatment outside of the initial treated area. This trial gives us the opportunity to further evaluate this novel modality in the treatment of one of the most deadly and challenging cancers we face."

Dr. Raijman added that the "Alpha DaRT sources were delivered into the pancreatic tumor under real-time endoscopic ultrasound guidance, enabling a seamless and accurate delivery through a minimally invasive approach. This non-surgical technique makes it possible to reach deeply located tumors with precision, and with potentially less side effects. This is an exciting time for endoscopists to explore a new and promising interventional option for the treatment of such a devasting disease."

"This study incorporates a thoughtful design based on our pre-clinical work to explore the integration of Alpha DaRT with chemotherapy in both locally advanced and metastatic pancreatic cancer," commented Dr. Robert Den, MD, Chief Medical Officer of Alpha Tau. "We are focused on generating high-quality clinical data on safety and early efficacy to inform our future development path. Our goal is to eventually offer patients a localized treatment option with the potential to enhance both local control and overall outcomes. This trial builds on Alpha Tau’s expanding clinical program aimed at solid tumors with limited local treatment options and aligns with the company’s mission to develop curative technologies that deliver alpha radiation precisely where it matters most."

About the IMPACT Study

The IMPACT study aims to enroll up to 30 patients, comprising 15 patients with inoperable locally advanced disease and 15 patients with metastatic disease, across multiple centers in the U.S., Canada and Israel.

Eligible patients must have newly diagnosed, histologically confirmed pancreatic adenocarcinoma and must be inoperable, non-irradiated, and either chemotherapy-naïve or within the first four cycles of their initial chemotherapy regimen. Patients who have undergone prior surgery or received radiation are excluded.

Patients will continue receiving their standard-of-care chemotherapy throughout the study (mFOLFIRINOX), and Alpha DaRT sources will be implanted into the primary tumor using ultrasound-guided endoscopy. Follow-up will continue up to 6 months after enrollment.

The primary objectives of the study are to assess the feasibility of Alpha DaRT source implantation and to evaluate its safety and tolerability, specifically monitoring for Grade 3 or higher adverse events related to the device. Secondary objectives include evaluating local tumor response using RECIST criteria and/or volumetric imaging, measuring time to local progression, monitoring progression-free survival and overall survival, and collecting patient-reported outcomes related to pain control. In addition, patients with inoperable locally advanced disease will be evaluated based on the percentage of patients who become surgically resectable after treatment with Alpha DaRT. Additional information about the IMPACT trial can be found at View Source

On September 2, 2025 Alivexis, Inc. (Headquartered in Minato-ku, Tokyo; CEO S. Roy Kimura) reported they have received a notice from Astellas Pharma Inc. ("Astellas") to exercise the option right granted under a Research Collaboration Agreement, executed on June 17th 2024 (Press release, Alivexis, SEP 2, 2025, View Source [SID1234655631]). This agreement focused on identifying small molecule compounds targeting a new drug target selected by Astellas, utilizing Alivexis’ drug discovery platform ModBind and other advanced technologies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The joint research collaboration aimed to accelerate the discovery of novel functional modulators for a previously uncharacterized drug target selected by Astellas. Leveraging our computational drug discovery platform, including ModBind, the Alivexis team led the integrated drug discovery researching, including conducting in silico evaluations and developing experimental assays to evaluate compound efficacy. Under the terms of the collaboration agreement, Astellas was granted the option to acquire rights to the research deliverables.

About ModBind.
Alivexis has established a computational drug discovery platform that greatly accelerates small molecule drug discovery, which includes physics-based molecular dynamics simulations using GPUs (Graphics Processing Units), large-scale virtual screening algorithms, and deep learning models. With the help of their computational drug discovery platform, Alivexis has already delivered several clinical candidate molecules for both in-house drug discovery projects and external collaborations. Among Alivexis’ various computational drug discovery tools, the newly developed ModBind is a molecular simulations-based algorithm that can predict the efficacy of drug candidate compounds with high accuracy, yet performs hundreds to thousands of times faster than the other state-of-the-art technologies in the field. ModBind is based on a theoretical approach that is fundamentally different from industry standard simulations-based prediction technologies. One significant advantage of ModBind is that it is an absolute predictor of ligand efficacy and does not require known reference compounds, which are usually necessary for other methodologies. Therefore, ModBind is useful in all stages of preclinical drug discovery – from screening large random chemical libraries for initial hit finding to delivering clinical candidates in the lead-optimization stage. This capability has already been proven by Alivexis’ in-house research and external collaborations and is contributing to the progression of many drug discovery projects.

【CEO S. Roy Kimura’s Comments】
"We are extremely pleased that Astellas Pharma has elected to exercise its option to obtain the rights for the small molecule compounds that were discovered through our ModBind drug discovery collaboration. Despite being a difficult target with no previously reported lead compounds, our ModBind simulation technology has successfully identified compounds worth further study. This result further demonstrates the power of our ModBind platform to rapidly identify promising drug lead compounds. Beyond identifying new drug candidates for other promising disease targets, we are currently applying ModBind to develop a foundation AI model for drug discovery and generate very large synthetic datasets to train target-specific generative AI models. We believe that the combination of simulation and AI (ModBindxAI) is the key to unlocking efficient drug discovery. "

On September 2, 2025 Aligos Therapeutics, Inc. (Nasdaq: ALGS) a clinical stage biopharmaceutical company focused on improving patient outcomes through best-in-class therapies for liver and viral diseases, reported that members of management will present at the H.C. Wainwright 27th Annual Global Investment Conference being held September 8-10, 2025 (Press release, Aligos Therapeutics, SEP 2, 2025, View Source [SID1234655629]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A webcast of the on demand presentation will be accessible by visiting the Presentation & Events section on the "Investors" page of Aligos’ website at www.aligos.com. A replay of the webcast will be available following the presentation for at least 30 days.